UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

Form 10-Q

(Mark One)

☒
x			QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the quarterly period ended ~~September 30, 2017~~

March 31, 2023

☐
o			TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the transition period from to

__________to __________

Commission file number: 001-36183

Eiger BioPharmaceuticals, Inc.

(Exact Name of Registrant as Specified in its Charter)

~~Delaware~~		~~33-0971591~~

(650) 272-6138

(Registrant’s Telephone Number, Including Area Code)

Securities registered pursuant to Section 12(b) of the Act:


Title of each class	Trading Symbol(s)	Name of each exchange on which registered
Common Stock (par value $0.001 per share)	EIGR	The Nasdaq Stock Market LLC

Indicate by check mark whether the registrant: (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yes ☒x No ☐

Indicate by check mark whether the registrant has submitted electronically, ~~and posted on its corporate Web site, if any,~~ every Interactive Data File required to be submitted ~~and posted~~ pursuant to Rule 405 of Regulation S-T (§232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit ~~and post~~ such files). Yes ☒x No ☐

Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company or an emerging growth company. See the definitions of “large accelerated filer”, “accelerated filer”, “smaller reporting company” and “emerging growth company” in Rule 12b-2 of the Exchange Act. ~~(Check one)~~:

Large accelerated filer

☐

Accelerated filer

☐

Non-accelerated filer

☐ ~~(Do not check if a smaller reporting company)~~

Smaller reporting company

☒

Emerging growth company

☒

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☒

Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes ☐o No ☒

As of ~~November 3, 2017,~~May 8, 2023, the number of outstanding shares of the registrant’s common stock, par value $0.001 per share, was ~~10,516,406.~~

44,296,417.

Our independent registered public accounting firm is KPMG LLP, San Francisco, CA, Auditor ID: 185

Table of Contents

EIGER BIOPHARMACEUTICALS, INC.

TABLE OF CONTENTS

	~~Page No.~~
		Page No.
PART I. FINANCIAL INFORMATION
Item 1. Financial Statements:			3
Condensed Consolidated Balance Sheets as of ~~September 30, 2017~~March 3 1 , 2023 (unaudited) and December 31, ~~2016~~2022			3
Condensed Consolidated Statements of Operations for the three ~~and nine~~ months ended ~~September 30, 2017~~March 31, 2023 and ~~2016~~2022 (unaudited)			4
Condensed Consolidated Statements of Comprehensive Loss for the three months ended Mar ch 3 1 , 202 3 and ~~nine~~202 2 (unaudited)		5
Condensed Consolidated Statements of Stockholders’ Equity for the three months ended ~~September 30, 2017~~March 31, 2023 and ~~2016~~2022 (unaudited)			5 6
Condensed Consolidated Statements of Cash Flows for the ~~nine~~ three months ended ~~September 30, 2017~~March 31, 2023 and ~~2016~~2022 (unaudited)			6 7
Notes to the Condensed Consolidated Financial Statements (unaudited)			7 8
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations			15 20
Item 3. Quantitative and Qualitative Disclosures About Market Risk			22 27
Item 4. Controls and Procedures			22 27

PART II. OTHER INFORMATION			28
Item 1. Legal Proceedings			23 28
Item 1A. Risk Factors			23 28
Item 2. Unregistered Sales of Equity Securities and Use of Proceeds			47 69
Item 3. Defaults Upon Senior Securities			47 69
Item 4. Mine Safety Disclosures			48 69
Item 5. Other Information			48 69
Item 6. Exhibits			49 70
Signatures			50 71

In this Quarterly Report on Form 10-Q, “we,” “our,” “us,” “Eiger,” and “the Company” refer to Eiger ~~Biopharmaceuticals,~~BioPharmaceuticals, Inc. Eiger, Eiger ~~Biopharmaceuticals,~~BioPharmaceuticals, the Eiger logo and other trade names, trademarks or service marks of Eiger are the property of Eiger ~~Biopharmaceuticals,~~BioPharmaceuticals, Inc. This Quarterly Report on Form 10-Q contains references to our trademarks and to trademarks belonging to other entities. Trade names, trademarks and service marks of other companies appearing in this Quarterly Report on Form 10-Q are the property of their respective holders. We do not intend our use or display of other companies’ trade names or trademarks to imply a relationship with, or endorsement or sponsorship of us by any other companies.

Table of Contents

PART I. ~~FINANCI~~ALFINANCIAL INFORMATION

~~ITEM 1.~~

ITEM 1. FINANCIAL STATEMENTS

Eiger BioPharmaceuticals, Inc.

Condensed Consolidated Balance Sheets

(In thousands)

	September 30,			December 31,
	2017			2016				March 31, 2023		December 31, 2022
	(Unaudited)							(Unaudited)
Assets							Assets
Current assets:							Current assets:
Cash and cash equivalents	$	13,078		$	27,756		Cash and cash equivalents	$	15,181	$	25,798
Marketable securities		19,264			32,180
Short-term debt securities							Short-term debt securities	60,091		73,150
Accounts receivable, net							Accounts receivable, net	3,891		1,749
Inventories							Inventories	5,338		2,853
Prepaid expenses and other current assets		783			581		Prepaid expenses and other current assets	12,410		13,985
Total current assets		33,125			60,517		Total current assets	96,911		117,535
Property and equipment, net		90			76		Property and equipment, net	804		696
Operating lease right-of-use assets							Operating lease right-of-use assets	446		561
Other assets		66			143		Other assets	1,067		1,347
Total assets	$	33,281		$	60,736		Total assets	$	99,228	$	120,139
Liabilities and Stockholders’ Equity							Liabilities and Stockholders’ Equity
Current liabilities:							Current liabilities:
Accounts payable	$	4,167		$	2,639		Accounts payable	$	11,858	$	8,975
Accrued liabilities		1,566			2,649		Accrued liabilities	11,706		15,655
Current portion of operating lease liabilities							Current portion of operating lease liabilities	435		491
Total current liabilities		5,733			5,288		Total current liabilities	23,999		25,121
Long term debt, net		14,993			14,727
Debt, net of current portion							Debt, net of current portion	39,989		39,625
Operating lease liabilities							Operating lease liabilities	1		83
Total liabilities		20,726			20,015		Total liabilities	63,989		64,829
Stockholders’ equity:							Stockholders’ equity:
Common stock		8			8		Common stock	44		44
Additional paid-in capital		120,462			117,086		Additional paid-in capital	495,342		492,759
Accumulated other comprehensive loss		(2	)		(15	)	Accumulated other comprehensive loss	(170)		(300)
Accumulated deficit		(107,913	)		(76,358	)	Accumulated deficit	(459,977)		(437,193)
Total stockholders’ equity		12,555			40,721		Total stockholders’ equity	35,239		55,310
Total liabilities and stockholders’ equity	$	33,281		$	60,736		Total liabilities and stockholders’ equity	$	99,228	$	120,139

The accompanying notes are an integral part of these unaudited condensed consolidated financial statements.

Table of Contents

Eiger BioPharmaceuticals, Inc.

Condensed Consolidated Statements of Operations

(Unaudited)

(In thousands, except share and per share amounts)

Three Months Ended

Nine Months Ended

September 30,

September 30,

2017

2016

2017

2016

Operating expenses:

Research and development

$
6,145

$
8,072

$
21,740

$
23,637

General and administrative

2,727

3,264

9,195

9,574

Total operating expenses

8,872

11,336

30,935

33,211

Loss from operations

(8,872
)

(11,336
)

(30,935
)

(33,211
)
Interest expense

(388
)

—

(1,129
)

(685
)
Interest income

98

—

321

—

Other income (expense), net

(8
)

(34
)

188

(423
)
Net loss

$
(9,170
)

$
(11,370
)

$
(31,555
)

$
(34,319
)
Net loss per common share, basic and diluted

$
(1.10
)

$
(1.49
)

$
(3.77
)

$
(6.58
)
Weighted-average common shares outstanding, basic and diluted

8,372,934

7,622,963

8,366,880

5,218,099


	Three Months Ended March 31,
	2023		2022
Product revenue, net	$	4,118	$	2,673
Costs and operating expenses:
Cost of sales	118		110
Research and development	16,748		17,570
Selling, general and administrative	9,515		6,813
Total costs and operating expenses	26,381		24,493
Loss from operations	(22,263)		(21,820)
Interest expense	(1,285)		(886)
Interest income	711		45
Other (expense) income, net	55		27
Loss before provision for income taxes	(22,782)		(22,634)
Provision for income taxes	2		9
Net loss	$	(22,784)	$	(22,643)
Net loss per common share:
Basic and diluted	$	(0.52)	$	(0.64)
Weighted-average common shares outstanding:
Basic and diluted	44,145,635		35,253,147

The accompanying notes are an integral part of these unaudited condensed consolidated financial statements.

Table of Contents

Eiger BioPharmaceuticals, Inc.

Condensed Consolidated Statements of Comprehensive Loss

(Unaudited)

(In thousands)

	Three Months Ended						Nine Months Ended
	September 30,						September 30,							Three Months Ended March 31,
	2017			2016			2017			2016				2023		2022
Net loss	$	(9,170	)	$	(11,370	)	$	(31,555	)	$	(34,319	)	Net loss	$	(22,784)	$	(22,643)
Other comprehensive loss:													Other comprehensive loss:
Unrealized gain on marketable securities, net		4			—			13			—
Unrealized gain (loss) on available-for-sale debt securities, net													Unrealized gain (loss) on available-for-sale debt securities, net	167		(373)
Foreign currency translation adjustment													Foreign currency translation adjustment	(37)		—
Comprehensive loss	$	(9,166	)	$	(11,370	)	$	(31,542	)	$	(34,319	)	Comprehensive loss	$	(22,654)	$	(23,016)

The accompanying notes are an integral part of these unaudited condensed consolidated financial statements.

Table of Contents

Eiger BioPharmaceuticals, Inc.

Condensed Consolidated Statements of ~~Cash Flow~~

Stockholders’ Equity

(Unaudited)

(In ~~thousands)~~

thousands, except share amounts)

Nine Months Ended

September 30,

2017

2016

Operating activities

Net loss

$
(31,555
)

$
(34,319
)
Adjustments to reconcile net loss to net cash used in operating activities:

Depreciation and amortization

30

15

Amortization of premiums on marketable securities

(63
)

—

Non-cash interest expense

266

685

Stock-based compensation

3,223

1,751

Gain on intellectual property sale

(200
)

—

Change in fair value of obligation to issue shares to Eiccose

—

204

Change in fair value of warrant liability

—

165

Issuance of common stock in connection with a license agreement

—

3,172

Change in operating assets and liabilities:

Prepaid expenses and other current assets

(202
)

(5
)
Other non-current assets

77

(88
)
Accounts payable

1,528

2,135

Accrued and other liabilities

(1,083
)

(547
)
Net cash used in operating activities

(27,979
)

(26,832
)
Investing activities

Purchase of marketable securities

(22,533
)

—

Proceeds from maturities of marketable securities

35,525

—

Proceeds from intellectual property sale

200

—

Proceeds from merger transaction

—

28,018

Purchase of property and equipment

(44
)

(53
)
Net cash provided by investing activities

13,148

27,965

Financing activities

Proceeds from issuance of common stock upon private placement, net of issuance cost

—

32,108

Proceeds from issuance of common stock upon public offering, net of issuance cost

—

18,229

Proceeds from issuance of common stock upon ESPP purchase

142

—

Proceeds from issuance of common stock upon options exercises

11

39

Proceeds from issuance of common stock upon warrants exercises

—

7

Net cash provided by financing activities

153

50,383

Net (decrease) increase in cash and cash equivalents

(14,678
)

51,516

Cash and cash equivalents at beginning of period

27,756

4,778

Cash and cash equivalents at end of period

$
13,078

$
56,294

Supplemental disclosures of non-cash investing and financing information:

Non-cash activities:

Conversion of warrant liability to common stock upon private placement

$
—

$
1,050

Issuance of common stock in connection with a license agreement

$
—

$
3,172

Issuance of common stock to Eiccose upon private placement

$
—

$
1,661

Non-cash net liabilities assumed in reverse merger

$
—

$
671

Conversion of convertible promissory note to common stock upon private
placement

$
—

$
6,129

Conversion of preferred stock to common stock upon reverse merger

$
—

$
22,567


	Common Stock			Additional Paid-In Capital		Accumulated Other Comprehensive Income (Loss)		Accumulated Deficit		Total Stockholders’ Equity
	Shares	Amount
Balance at December 31, 2022	44,074,284	$	44	$	492,759	$	(300)	$	(437,193)	$	55,310
Issuance of common stock upon ESPP purchase	29,986	—		41		—		—		41
Issuance of common stock upon release of restricted stock units and performance stock units	192,147	—		—		—		—		—
Stock-based compensation expense	—	—		2,542		—		—		2,542
Unrealized gain on available-for-sale debt securities, net	—	—		—		167		—		167
Cumulative translation adjustment	—	—		—		(37)		—		(37)
Net loss	—	—		—		—		(22,784)		(22,784)
Balance at March 31, 2023	44,296,417	$	44	$	495,342	$	(170)	$	(459,977)	$	35,239


	Common Stock			Additional Paid-In Capital		Accumulated Other Comprehensive Loss		Accumulated Deficit		Total Stockholders’ Equity
	Shares	Amount
Balance at December 31, 2021	34,568,821	$	35	$	412,930	$	(149)	$	(340,417)	$	72,399
Issuance of common stock upon offering at-the-market, net of $1,288 of commissions	5,841,786	6		45,604		—		—		45,610
Issuance of common stock upon exercise of stock options	15,995	—		144		—		—		144
Vesting of common stock issued under Product Development Agreement	—	—		19		—		—		19
Issuance of common stock upon ESPP purchase	18,130	—		64		—		—		64
Issuance of common stock upon release of restricted stock units	85,106	—		—		—		—		—
Stock-based compensation expense	—	—		2,047		—		—		2,047
Unrealized loss on available-for-sale debt securities, net	—	—		—		(373)		—		(373)
Net loss	—	—		—		—		(22,643)		(22,643)
Balance at March 31, 2022	40,529,838	$	41	$	460,808	$	(522)	$	(363,060)	$	97,267

The accompanying notes are an integral part of these unaudited condensed consolidated financial statements.

Table of Contents

Eiger BioPharmaceuticals, Inc.

Condensed Consolidated Statements of Cash Flow

(Unaudited)

(In thousands)


	Three Months Ended March 31,
	2023		2022
Operating activities
Net loss	$	(22,784)	$	(22,643)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation and amortization	74		73
Inventory write down	10		—
Amortization of debt securities premiums and discounts	(374)		330
Non-cash interest expense	364		200
Reduction in the carrying amount of right-of-use assets	115		132
Common stock issued under Product Development Agreement	—		19
Stock-based compensation	2,542		2,047
Change in operating assets and liabilities:
Accounts receivable	(2,130)		289
Inventories	(860)		666
Prepaid expenses and other current assets	1,401		(854)
Other assets	454		(397)
Accounts payable	1,553		1,269
Accrued liabilities	(4,232)		560
Operating lease liabilities	(138)		(150)
Net cash used in operating activities	(24,005)		(18,459)
Investing activities
Purchase of debt securities available-for-sale	(19,388)		(10,902)
Proceeds from maturities of debt securities available-for-sale	32,987		7,900
Purchase of property and equipment	(216)		—
Net cash provided by (used in) investing activities	13,383		(3,002)
Financing activities
Issuance of common stock upon offering at-the-market, net of commissions	—		45,610
Proceeds from issuance of common stock upon stock option exercises	—		144
Proceeds from issuance of common stock upon ESPP purchase	41		64
Common stock offering costs	—		(16)
Net cash provided by financing activities	41		45,802
Effect of foreign exchange on cash and cash equivalents	(36)		—
Net (decrease) increase in cash and cash equivalents	(10,617)		24,341
Cash and cash equivalents at beginning of period	25,798		22,221
Cash and cash equivalents at end of period	$	15,181	$	46,562

Supplemental disclosure of cash flow information:
Interest paid	$	920	$	686
Income taxes paid	$	—	$	41

The accompanying notes are an integral part of these unaudited condensed consolidated financial statements.

Eiger BioPharmaceuticals, Inc.

Notes to the Condensed Consolidated Financial Statements

(Unaudited)

1.	~~Description of Business~~

1. Description of Business

Eiger BioPharmaceuticals, Inc. (the ~~“Company”)~~Company or Eiger) was incorporated in the State of Delaware on November 6, 2008. Eiger is a ~~clinical-stage~~commercial-stage biopharmaceutical company ~~committed~~focused on the development of innovative therapies for hepatitis delta virus (HDV), the most severe form of viral hepatitis, and other serious diseases. All five of the Company’s rare disease programs have been granted Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA).

The Eiger HDV platform includes two first-in-class therapies in Phase 3 that target critical host processes involved in viral replication. Lonafarnib is a first-in-class, oral farnesylation inhibitor, and peginterferon lambda is a first-in-class, type III, interferon.

The Company is also developing avexitide, a well-characterized peptide, as a treatment for congenital hyperinsulinism (HI), an ultra-rare pediatric metabolic disorder, and post-bariatric hypoglycemia (PBH), a debilitating and potentially life-threatening condition. Avexitide has completed Phase 2 for both indications, and Phase 3 study startup activities for HI have been initiated.

The FDA approved the Company’s first commercial product, Zokinvy® (lonafarnib), to ~~bringing to market novel products~~reduce risk of mortality of Hutchinson-Gilford progeria syndrome (HGPS) and for ~~the~~ treatment of ~~orphan diseases.~~ processing-deficient progeroid laminopathies (PL), with either heterozygous LMNA mutation with progerin-like protein accumulation, or homozygous or compound heterozygous ZMPSTE24 mutations, on November 20, 2020. Collectively known as progeria, HGPS and PL are ultra-rare, fatal, genetic premature aging diseases that accelerate mortality in young patients. In July 2022, the Company announced that the European Commission (EC) granted Eiger a centralized marketing authorization (MA). The EC's centralized MA is valid in all 27 EU member states plus Iceland, Liechtenstein, and Norway. In August 2022, the Medicine and Healthcare products Regulatory Agency (MHRA) granted approval in the UK.

The Company ~~has built a diverse portfolio~~commercially launched Zokinvy in the U.S. in January 2021 and started to record product revenue in the first quarter of ~~well-characterized product candidates with~~2021. The first European sales were recognized in the ~~potential to address diseases for which the unmet medical need is high, the biology for treatment is clear, and for which an effective therapy is urgently needed.~~ fourth quarter of 2022.

The Company’s principal operations are based in Palo Alto, California, with subsidiaries in Delaware, Ireland, England and itWales. The Company operates in one segment.

Liquidity

As of ~~September 30, 2017,~~March 31, 2023, the Company had ~~$13.1~~$75.3 million of cash, cash equivalents and short-term securities, comprised of $15.2 million of cash and cash equivalents ~~$19.3~~and $60.1 million of short-term ~~marketable~~debt securities available-for-sale. The Company had an accumulated deficit of ~~$107.9~~$460.0 million and negative cash flows from operating ~~activities.~~activities as of March 31, 2023. As the Company continues to incur losses, its transition to profitability will depend on the successful development, approval, and commercialization of product candidates and on the achievement of sufficient revenues to support its cost structure. The Company ~~expects~~may never achieve profitability, and until it does, the Company will need to continue to ~~incur losses for the next several years.~~

On October 31, 2017, the Company issued 2,132,961 shares of common stock pursuant to an underwriting agreement with BTIG, LLC at a public offering price of $10.00 per share, for gross proceeds of $21,329,610, which resulted in approximately $19.5 million of net proceeds to the Company after deducting underwriting fees and offering expenses. See Note11.

raise additional capital.

Management believes that the currently available resources will be sufficient to fund its planned operations for at least the next 12 months following the issuance date of these ~~unaudited~~ condensed consolidated financial statements.

However, if the Company’s anticipated operating results are not achieved in future periods, the Company believes that planned expenditures may need to be reduced or it would be required to raise funding in order to fund the operations. Additionally, the Company’s ability to raise additional capital may be adversely impacted by potential worsening global economic conditions and disruptions to, and volatility in, the credit and financial markets in the United States and worldwide.

2.	~~Summary of Significant Accounting Policies~~

2. Summary of Significant Accounting Policies

Basis of Presentation

The ~~unaudited~~ condensed consolidated financial statements include the accounts of Eiger BioPharmaceuticals, Inc. and its wholly owned subsidiaries, EBPI Merger Inc., EB Pharma LLC, ~~and~~ Eiger BioPharmaceuticals Europe Limited, and EigerBio Europe Limited, have been prepared in accordance with accounting principles generally accepted in the United States of America, (“

(U.S. ~~GAAP”)~~GAAP) and ~~following~~follow the requirements of the Securities and Exchange Commission ~~(the “SEC”)~~(SEC) for ~~interim~~annual reporting. As permitted under those rules, certain footnotes or other financial information that are normally required by U.S. GAAP can be condensed or omitted. These financial statements have been prepared on the same basis as the Company’s annual financial statements and, in the opinion of management, reflect all adjustments, consisting only of normal recurring adjustments, which are necessary for a fair statement of the Company’s financial information. These interim results are not necessarily indicative of the results to be expected for the year ending December 31, 2017 or for any other interim period or for any other future year. The balance sheet as of December 31, 2016, has been derived from audited consolidated financial statements at that date but does not include all of the information required by U.S. GAAP for complete financial statements. All intercompany balances and transactions have been eliminated in consolidation.

The accompanying unaudited condensed consolidated financial statements and related financial information should be read in conjunction with the audited consolidated financial statements and the related notes thereto contained in the Company’s Annual Report on Form 10-K for the year ended December 31, 2016, filed with the Securities and Exchange Commission on March 23, 2017.

Use of Estimates

The preparation of ~~unaudited~~ condensed consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of revenue and expenses during the reporting period. On an ongoing basis, the Company evaluates its ~~estimates, including those related to clinical trial accrued liabilities, stock-based compensation and income taxes.~~estimates. The Company bases its estimates on historical experience and on various other market-specific and relevant assumptions that the Company believes to be reasonable under the circumstances. Actual results could differ from those estimates.

Concentrations of Risk

~~Short-Term Marketable~~

Financial instruments that potentially subject the Company to a concentration of credit risk consists of cash, cash equivalents and investments. The Company’s cash is held by financial institutions in the United States and Ireland. Amounts on deposit may at times exceed federally insured limits. Management believes that the financial institutions are financially sound, and accordingly, minimal credit risk exists with respect to the financial institution.

The Company relies on one supply chain for each of its product candidates. If any of the single source suppliers in any of the supply chains fail to satisfy the Company’s requirements on a timely basis, it could suffer delays in its clinical development programs and activities which could adversely affect its operating results.

Two customers accounted for approximately 51 percent and 49 percent of the Company’s accounts receivable as of March 31, 2023. Two customers accounted for approximately 58 percent and 42 percent of the Company’s accounts receivable as of December 31, 2022. Two customers accounted for approximately 76 percent and 24 percent of product revenue during the three months ended March 31, 2023. One customer accounted for approximately 100 percent of product revenue during the three months ended March 31, 2022. Three vendors accounted for approximately 15 percent, 12 percent and 12 percent of Company's accounts payable as of March 31, 2023. Two vendors accounted for approximately 12 percent and 11 percent of Company's accounts payable as of December 31, 2022.

Foreign Currency Exchange

Foreign Currency Transaction Risk

The foreign currency transaction risk relates to changes in exchange rates on monetary assets, liabilities, revenues and expenses held at Eiger BioPharmaceuticals Europe Limited. Gains and losses on foreign currency transactions result primarily from monetary assets, liabilities, revenues and expenses denominated in Euro. Aggregated transaction gains for three months ended March 31, 2023 were $0.1 million. The Company expects the foreign currency gain/loss to continue to fluctuate as long as the Company continue to hold monetary assets and liabilities at [the subsidiary level] [its subsidiaries in Ireland and England and Wales]. Market uncertainty could potentially lead to significant volatility with foreign currency exchange rates, which could result in additional foreign currency gain/loss.

Foreign Currency Translation Risk

The foreign currency translation risk relates to the translation of the foreign consolidated subsidiaries' assets, liabilities, revenues and expenses from the subsidiaries’ functional currency to the U.S. dollar at each reporting date. Fluctuations in exchange rates may impact the amount of assets, liabilities, revenues and expenses reported on the consolidated balance sheets and consolidated statements of operations. The financial statements of the Company’s foreign subsidiaries, which have a functional currency other than the U.S. dollar, are translated into U.S. dollars using a current exchange rate. Gains and losses resulting from this translation are recognized as a foreign currency translation adjustment within accumulated other comprehensive loss, which is a component of stockholders' equity and comprehensive income (loss). Aggregate translation losses, net of tax, for the three months ended March 31, 2023 and March 31, 2022 were $37,000 and $0, respectively.

Debt Securities

~~Short-term marketable~~

All securities are short-term in nature and consist of debt securities classified as available-for-sale and have maturities greater than 90 days, but less than 365 days from the date of acquisition. ~~All short-term marketable~~The Company’s debt securities consist of

available-for-sale securities that are ~~carried at~~classified as Level 2 because their value is based on valuations using significant inputs derived from, or corroborated by, observable market data. The Company evaluates, on a quarterly basis, its available-for-sale debt securities for potential impairment. For available-for-sale debt securities in an unrealized loss position, the Company assesses whether such declines are due to credit related factors such as changes to the rating of the security by a ratings agency, market conditions and supportable forecasts of economic and market conditions, among others. If the fair value ~~based upon quoted market prices. Unrealized gains~~of available-for-sale debt securities is less than the amortized cost basis, the Company assesses whether it has plans to sell the security or it is more likely than not it will be required to sell any available-for-sale debt security before recovery of its amortized cost basis. If either condition is met, the security’s amortized cost basis is written down to fair value and ~~losses on available-for-sale securities are excluded from earnings and are reported~~is recognized through other (expense) income, net. If neither condition is met, declines as a ~~component~~result of ~~accumulated~~credit losses, if any, are recognized as an allowance for credit loss, limited to the amount of unrealized loss, through other (expense) income, net. Any portion of the unrealized loss that is not a result of a credit loss, is recognized in other comprehensive loss. The cost of available-for-sale securities sold is based on the specific-identification method. Realized gains and losses on the sale of ~~marketable~~debt securities are determined using the specific-identification method and recorded in other ~~expense,~~(expense) income, net.

Accounts Receivable

Accounts receivable represent amounts billed to the Company’s customers, net of an allowance for credit losses. Trade accounts receivable are recorded at invoiced amounts and do not bear interest. The allowance for credit losses reflects the Company’s best estimate of probable losses inherent in the receivable portfolio determined based on various factors, including age of the ~~accompanying unaudited condensed consolidated statements~~outstanding invoice, credit quality of ~~operations.~~

the customer, historical experience, current economic conditions, and management’s expectations of future economic conditions. The Company regularly reviews the adequacy of the allowance for credit losses by considering the age of each outstanding invoice and the collection history of each customer to determine the appropriate amount of allowance for credit losses.

The Company had no allowance for credit losses as of March 31, 2023 and December 31, 2022. The Company had no credit losses for the three months ended March 31, 2023 and 2022.

Inventories

Inventories are stated at the lower of cost, determined based on actual costs, or estimated net realizable value, on a first-in, first-out basis. Inventories consist of raw materials, work-in-process, and finished goods.

Prior to regulatory approval of the Company’s product candidates, expenses incurred to manufacture drug products are recorded as research and development expense. The Company begins capitalizing these expenses as inventory upon regulatory approval.

The Company periodically assesses the recoverability of its inventory and reduces the carrying value of the inventory when items are determined to be obsolete, defective or in excess of forecasted sales requirements. Inventory write-downs for excess, defective, and obsolete inventory are recorded as a cost of sales.

There have been $10,000 in write-downs of inventories for the three months ended March 31, 2023. There was no write-down of inventories for the three months ended March 31, 2022.

Revenue Recognition

The Company recognizes revenue upon transfer of control of promised products to customers in an amount that reflects the consideration it expects to receive in exchange for those products.To determine revenue recognition for contracts with customers, the Company performs the following five-step approach: (i) identify the contract, or contracts, with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when, or as, the performance obligation is satisfied. The five-step model is only applied to contracts when it is probable that the Company will collect substantially all of the consideration it is entitled to in exchange for the goods transferred to a customer.

Product Revenue

The Company’s product revenue consists of sales of Zokinvy, which received FDA approval in November 2020, EC centralized MA in July 2022, and MHRA approval in August 2022. Zokinvy was launched commercially in the United States in January 2021 and in Europe in November 2022. Prior to 2021, the Company had no product revenue. In the

United States, the Company sells Zokinvy to a single specialty pharmacy provider that subsequently dispenses the product directly to patients. The Company discloses revenue on a total basis without further disaggregation. Additionally, the Company does not have any contract assets or liabilities, other than accounts receivable, related to its product revenue.

In June 2021, the French National Agency for Medicines and Health Products Safety (ANSM) granted Zokinvy (lonafarnib) a Temporary Authorizations for Use (Autorisation Temporaire d'Utilisation or ATU) for an early access program for a term of one year. The Company has received a one year extension of the ATU program and expects the program to continue until commercial reimbursement of Zokinvy is approved in France. In the context of this program, the Company sells product to a distributor who in turn ships product to pharmacies after receiving requests from physicians for patients in France. In November 2021, the Company began distributing and recognizing revenue from sales of Zokinvy (lonafarnib) through a reimbursed early access program in France. The Company recorded revenue of $0.5 million from sales of product under the ATU program for the three months ended March 31, 2023. There was no revenue from sale of product under the ATU program for the three months ended March 31, 2022.

The Company recognizes product revenue when a customer obtains control of its product, which occurs at a point in time, typically upon delivery to a customer as the delivery of the product is the Company’s only performance obligation. Shipping and handling activities are fulfillment activities rather than a separate performance obligation and are recorded in cost of sales.

Product revenue is recorded at the net sales price (transaction price), which includes estimates of variable consideration resulting from rebates, prompt payment discounts, co-payment assistance, and returns. Amounts related to such items are estimated at contract inception and updated at the end of each reporting period as additional information becomes available. The amount of variable consideration may be constrained and is included in the transaction price only to the extent it is probable that a significant reversal of cumulative revenue recognized will not occur when the uncertainty associated with the variable consideration is resolved. Product revenue is recorded after considering the impact of the following variable consideration amounts along with the constraint at the time of revenue recognition:

Rebates: The Company’s product is subject to government mandated rebates for Medicaid Drug Rebate Program, Medicare Part D Prescription Drug Benefit Program, and other government health care programs in the United States. Rebate amounts are based upon contractual agreements or legal requirements with public sector benefit providers. The Company uses the expected-value method for estimating these rebates based on statutory discount rates and expected utilization. The expected utilization of rebates is estimated based on expected coverage of identified patients. Estimates for these rebates are adjusted quarterly to reflect the most recent information. The Company records an accrued liability for unpaid rebates related to products for which control has been transferred to a customer.

Prompt payment discounts: The Company provides a discount to a customer if it pays for purchases within 30 days. The Company expects that its customers will earn prompt payment discounts and uses the most likely amount method for estimating such discounts. As a result, when revenues are recognized, the Company deducts the full amount of the prompt payment discounts from total product revenues and records these discounts as a reduction of accounts receivable.

Co-payment assistance: The Company provides co-payment assistance to patients who have commercial insurance and meet certain eligibility requirements. The Company uses the expected-value method for estimating co-payment assistance based on estimates of program redemption using data provided by third-party administrators. Estimates for the co-payment assistance are adjusted quarterly to reflect actual experience. The Company records an accrued liability for unredeemed co-payment assistance related to products for which control has been transferred to a customer.

Product returns: The Company offers limited product return rights and generally allows for the return of product that is damaged or defective, or within a few months prior to and up to a few months after the product expiration date. The Company considers several factors in the estimation of potential product returns, including expiration dates of the product shipped, the limited product return rights, third-party data in monitoring channel inventory levels, shelf life of the product, and other relevant factors.

Cost of Sales

Cost of sales consists primarily of direct and indirect costs related to the manufacturing of Zokinvy for commercial sale, including third-party manufacturing costs, packaging services, freight, storage costs, and write down of inventories.

Accrued Research and Development Costs

The Company accrues for estimated costs of research and development activities conducted by third-party service providers, which include the conduct of preclinical and clinical studies, and contract manufacturing activities. The Company records the estimated costs of research and development activities based upon the estimated amount of services provided but not yet invoiced and includes these costs in accrued liabilities in the ~~unaudited~~accompanying condensed consolidated balance sheets and within research and development ~~expense~~expenses in the ~~unaudited~~accompanying condensed consolidated statements of operations. The Company accrues for these costs based on factors such as estimates of the work completed and in accordance with agreements established with its third-party service providers. The Company makes judgments and estimates in determining the accrued liabilities balance in each reporting period. As actual costs become known, the Company adjusts its accrued liabilities.

~~Net Loss per Share~~

~~Basic net~~

Recent Adopted Accounting Pronouncements

In June 2016, the Financial Accounting Standards Board (FASB) issued Accounting Standards Updates (ASU) No. 2016-13, Financial Instruments—Credit Losses (Topic 326). ASU 2016-13 requires an entity to utilize a new impairment model that requires measurement and recognition of expected credit losses for most financial assets and certain other instruments, including but not limited to available-for-sale debt securities. Credit losses relating to available-for-sale debt securities will be recorded through an allowance for credit losses rather than as a direct write-down to the security. The new guidance requires the use of forward-looking expected credit loss ~~per share of common stock is calculated by dividing~~models based on historical experience, current conditions, and reasonable and supportable forecasts that affect the net loss by the weighted average number of shares of common stock outstanding during the period, without consideration for potentially dilutive securities. Since the Company was in a loss position for all periods presented, diluted net loss per share is the same as basic net loss per share for all periods as the inclusion of all potential common shares outstanding would have been anti-dilutive.

~~The Weighted-average common shares outstanding for the three and nine months periods ended September 30, 2016, are impacted by the weighted effect~~collectability of the ~~conversion or issuance~~reported amount, which may result in earlier recognition of ~~6,668,045 common shares in conjunction with~~credit losses under the ~~closing of the Merger Agreement on March 22, 2016.~~

The following table sets forth the outstanding potentially dilutive securities which have been excluded in the calculation of diluted net loss per share because including such securities would be anti-dilutive (in common stock equivalent shares):

Three Months Ended

Nine Months Ended

September 30,

September 30,

2017

2016

2017

2016

Options to purchase common stock

1,530,394

1,124,044

1,530,394

1,124,044

Warrants to purchase common stock

10,180

10,180

10,180

10,180

Total

1,540,574

1,134,224

1,540,574

1,134,224

~~Recent Accounting Pronouncements~~

new guidance. In ~~February 2016,~~April 2019, the FASB issued ASU ~~2016-02,~~ ~~Leases (Topic 842),~~ No. 2019-04, Codification Improvements to Topic 326, Financial Instruments – Credit Losses, Topic 815, Derivatives and Hedging, and Topic 825, Financial Instruments,which ~~requires lessees to recognize most leases on their balance sheet. The standard requires use of the modified retrospective transition method, with elective relief, which requires application~~clarifies and corrects certain unintended applications of the guidance contained in each of the amended Topics. Additionally, in May 2019, the FASB issued ASU No. 2019-05, Financial Instruments – Credit Losses (Topic 326), which provides an option to irrevocably elect to measure certain individual financial assets at fair value instead of amortized cost. In November 2019, the FASB issued ASU No. 2019-10, Financial Instruments – Credit Losses (Topic 326), Derivatives and Hedging (Topic 815), and Leases (Topic 842), whichdeferred the effective date for ~~all periods presented. The new standard will be effective~~ASU No. 2016-13 for smaller reporting companies to fiscal years beginning after December 15, ~~2018. Early adoption is permitted.~~ ~~The Company is currently in the process of evaluating the impact that the standard will have on its consolidated financial statements.~~

~~In March 2016, the FASB issued ASU 2016-09,~~ ~~Compensation-Stock Compensation (Topic 718): Improvements to Employee Share-Based Payment Accounting, which is intended to simplify several aspects of the accounting for employee share-based payment transactions,~~2022, including the accounting and reporting of income taxes, the determination of forfeiture rates, classification of awards as either equity or liabilities, and classification on the statement of cash flows. ASU 2016-09 is effective for fiscal years and interim periods within those ~~years beginning after December 15, 2016 and early adoption is permitted.~~fiscal years. The Company ~~has~~ adopted this ~~ASU in in the first quarter of 2017.~~ Asguidance on a ~~result of adopting this standard, the Company made an accounting policy election to account for forfeitures as they occur.~~ ~~This change did not have a~~modified-retrospective basis effective January 1, 2023 and noted no material impact to the Company’s condensed consolidated financial statements.

3.	~~Fair Value Measurements~~

3. Fair Value Measurements

Fair value accounting is applied for all financial assets and liabilities that are recognized or disclosed at fair value in the financial statements on a recurring basis (at least annually). ~~At September 30, 2017~~As of March 31, 2023 and December 31, ~~2016,~~2022, the carrying amount of cash equivalents, accounts receivable, prepaid expenses and other current assets, accounts payable and accrued liabilities approximated their ~~estimate~~estimated fair value due to their relatively short maturities. Management believes the terms of ~~long term~~its long-term debt reflect current market conditions for an instrument with similar terms and maturity, therefore the carrying value of the Company’s debt approximated its fair value.

Assets and liabilities recorded at fair value on a recurring basis in the ~~unaudited~~ condensed consolidated balance sheets are categorized based upon the level of judgment associated with the inputs used to measure their fair values. Fair value is defined as the exchange price that would be received for an asset or an exit price that would be paid to transfer a liability in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants on the measurement date. The authoritative guidance on fair value measurements establishes a three-tier fair value hierarchy for disclosure of fair value measurements as follows:

Level 1: Inputs are unadjusted, quoted prices in active markets for identical assets or liabilities at the measurement date;

Level 2: Inputs are observable, unadjusted quoted prices in active markets for similar assets or liabilities, unadjusted quoted prices for identical or similar assets or liabilities in markets that are not active, or other inputs that are observable or can be corroborated by observable market data for substantially the full term of the related assets or liabilities; and

Level 3: Unobservable inputs that are significant to the measurement of the fair value of the assets or liabilities that are supported by little or no market data.

The Company’s money market funds are classified as Level 1 because they are valued using quoted market prices. The Company’s ~~marketable~~debt securities and commercial paper consist of available-for-sale securities and are classified as Level 2 because their value is based on valuations using significant inputs derived from or corroborated by observable market data. There were no assets or liabilities classified as Level 3 as of ~~September 30, 2017~~March 31, 2023 and December 31, ~~2016.~~

2022.

There were no transfers ~~between Level 1, Level 2~~into or out of Level 3 of the fair value hierarchy during the periods presented.

The following tables present the fair value hierarchy for assets and liabilities measured at fair value (in thousands):


	September 30, 2017									March 31, 2023
	Level 1		Level 2		Level 3		Total			Level 1		Level 2		Level 3		Total
Financial assets:									Financial assets:
Money market fund	$	8,016	$	—	$	—	$	8,016
Money market funds									Money market funds	$	12,631	$	—	$	—	$	12,631
Corporate debt securities		—		13,031		—		13,031	Corporate debt securities	—		19,783		—		19,783
Commercial paper		—		8,233		—		8,233	Commercial paper	—		4,990		—		4,990
U.S. government bonds									U.S. government bonds	—		35,318		—		35,318
Total	$	8,016	$	21,264	$	—	$	29,280	Total	$	12,631	$	60,091	$	—	$	72,722


	December 31, 2016									December 31, 2022
	Level 1		Level 2		Level 3		Total			Level 1		Level 2		Level 3		Total
Financial assets:									Financial assets:
Money market fund	$	9,657	$	—	$	—	$	9,657
Money market funds									Money market funds	$	11,546	$	—	$	—	$	11,546
Commercial paper									Commercial paper	—		8,913		—		8,913
Corporate debt securities		—		11,469		—		11,469	Corporate debt securities	—		28,642		—		28,642
Commercial paper		—		22,891		—		22,891
U.S. government bonds									U.S. government bonds	—		39,563		—		39,563
Total	$	9,657	$	34,360	$	—	$	44,017	Total	$	11,546	$	77,118	$	—	$	88,664

There were no financial liabilities as of ~~September 30, 2017~~March 31, 2023 and December 31, ~~2016.~~

2022.

The following ~~table summarizes~~tables summarize the estimated value of the Company’s cash equivalents and ~~marketable~~debt securities and the gross unrealized holding gains and losses (in thousands):


	September 30, 2017										March 31, 2023
	Amortized cost		Unrealized gain		Unrealized loss			Estimated Fair Value			Amortized cost		Unrealized gain		Unrealized loss		Estimated Fair Value
Cash equivalents:										Cash equivalents:
Money market fund	$	8,016	$	—	$	—		$	8,016
Commercial paper		2,000		—		—			2,000
Money market funds										Money market funds	$	12,631	$	—	$	—	$	12,631
Total cash equivalents	$	10,016	$	—	$	—		$	10,016	Total cash equivalents	$	12,631	$	—	$	—	$	12,631
Marketable securities:
Debt securities:										Debt securities:
Corporate debt securities	$	13,033	$	—	$	(2	)	$	13,031	Corporate debt securities	$	19,820	$	—	$	(37)	$	19,783
Commercial paper		6,233		—		—			6,233	Commercial paper	4,990		—		—		4,990
Total marketable securities	$	19,266	$	—	$	(2	)	$	19,264
U.S. government bonds										U.S. government bonds	35,314		8		(4)		35,318
Total debt securities										Total debt securities	$	60,124	$	8	$	(41)	$	60,091
Classified as:										Classified as:
Cash equivalents										Cash equivalents							$	12,631
Short-term debt securities										Short-term debt securities							60,091
																	$	72,722

December 31, 2016

Amortized cost

Unrealized gain

Unrealized loss

Estimated Fair Value

Cash equivalents:

Money market fund

$
9,657

$
—

$
—

$
9,657

Corporate debt securities

2,180

—

—

2,180

Total cash equivalents

$
11,837

$
—

$
—

$
11,837

Marketable securities:

Corporate debt securities

$
9,294

$
—

$
(5
)

$
9,289

Commercial paper

22,901

3

(13
)

22,891

Total marketable securities

$
32,195

$
3

$
(18
)

$
32,180

~~As of September 30, 2017, and December 31, 2016, the contractual maturity of the available-for-sale marketable securities is less than one year.~~ ~~The Company periodically reviews the available-for-sale investments for~~ ~~other-than-temporary~~ impairment loss. The Company considers factors such as the duration, severity and the reason for the decline in value, the potential recovery period and its intent to sell. For debt securities, it also considers whether (i) it is more likely ~~than not that the Company will be required to sell the debt securities before recovery of~~ ~~their~~ ~~amortized cost basis, and (ii) the amortized cost basis cannot be recovered as a result of credit losses.~~


	December 31, 2022
	Amortized cost		Unrealized gain		Unrealized loss		Estimated Fair Value
Cash equivalents:
Money market funds	$	11,546	$	—	$	—	$	11,546
Commercial paper	3,968		—		—		3,968
Total cash equivalents	$	15,514	$	—	$	—	$	15,514
Debt securities:
U.S. government bonds	$	39,646	$	3	$	(86)	$	39,563
Corporate debt securities	28,759		—		(117)		28,642
Commercial paper	4,945		—		—		4,945
Total debt securities	$	73,350	$	3	$	(203)	$	73,150
Classified as:
Cash equivalents							$	15,514
Short-term debt securities							73,150
							$	88,664

During the three ~~and nine~~ months ended ~~September 30, 2017,~~March 31, 2023, the Company did not recognize any other-than-temporary impairment losses. ~~All marketable~~The Company determined that the decline in fair value of debt securities ~~with~~was not due to credit-related factors, and no allowance for expected credit losses was recorded as of March 31, 2023. There were unrealized losses of $33,000 as of March 31, 2023, and no unrealized losses have been in athe loss position for ~~less~~more than ~~twelve~~ 12 months.

However, the Company is planning to hold these securities until maturity and expects to recover the amortized cost basis.

4.	~~Accrued Liabilities~~

4. Balance Sheet Components

Inventories

Inventories consist of the following (in thousands):


	March 31, 2023		December 31, 2022
Raw materials	$	4,290	$	1,703
Work-in-progress	834		884
Finished goods	214		266
Total inventories	$	5,338	$	2,853

Prepaid Expenses and Other Current Assets

Prepaid expenses and other current assets consist of the following (in thousands):


	March 31, 2023		December 31, 2022
Prepaid contract manufacturing costs	$	2,653	$	3,542
Short term deposits	4,743		4,542
Prepaid research costs	3,438		2,822
Prepaid insurance	147		586
Prepaid marketing	454		753
Other	975		1,740
Total prepaid expenses and other current assets	$	12,410	$	13,985

Accrued Liabilities

Accrued liabilities consist of the following (in thousands):

	September 30,		December 31,
	2017		2016			March 31, 2023		December 31, 2022
Compensation and related benefits	$	1,088	$	1,299	Compensation and related benefits	$	3,755	$	2,101
Consulting costs		175		106
Contract research costs		144		834	Contract research costs	2,724		4,188
Contract manufacturing costs		50		122	Contract manufacturing costs	1,906		6,167
Franchise tax		27		97
Product revenue reserves					Product revenue reserves	1,688		1,373
Other		82		191	Other	1,633		1,826
Total accrued liabilities	$	1,566	$	2,649	Total accrued liabilities	$	11,706	$	15,655

~~Debt~~

5. Bristol-Meyers Squibb License Agreement

On April 20, 2016, the Company and Bristol-Myers Squibb Company (BMS) entered into a License Agreement (the BMS License Agreement) and a Common Stock Purchase Agreement (the BMS Purchase Agreement).

Under the BMS License Agreement, BMS granted the Company an exclusive, worldwide, license to research, develop, manufacture, and sell products containing PEG-interferon Lambda-1a (peginterferon lambda or the Licensed Product) for all therapeutic and diagnostic uses in humans and animals. The Company is responsible for the development and commercialization of the Licensed Product at its sole cost and expense. The Company paid BMS $2.0 million and issued 157,587 shares of its common stock at an aggregate fair value of $3.2 million in April 2016. The BMS License Agreement also includes development and regulatory milestone payments totaling $61.0 million and commercial sales milestones of up to $128.0 million. The Company is obligated to pay BMS annual net sales royalties in the range of mid-single to mid-teens, depending on net sales levels. In ~~December 2016,~~fourth quarter of 2020, the Company recorded in research and development expense a $3.0 million milestone, triggered on successful demonstration of proof of concept, as defined by the BMS License Agreement, in a Phase 2 clinical trial. In March 2022, the Company recorded a $5.0 million milestone expense in research and development, which was related to the initiation of a Phase 3 clinical trial, as defined under the BMS License Agreement.

6. Debt

Innovatus Term Loan

On June 1, 2022 (Closing Date), the Company entered into ~~an aggregate $25.0~~a term loan and security agreement (Innovatus Loan) with Innovatus Life Sciences Lending Fund I, LP (Innovatus), providing for up to $75.0 million ~~loan~~funded in three tranches with ~~Oxford Finance LLC (the “Oxford Loan”).~~a maturity date of August 31, 2027. The loan matures on July 1, 2021. The Company borrowed $15.0 million in December 2016 (“Tranche A”). The remaining $10.0 million (“Tranche B”) will be available to the Company upon achievement of positive top line data from the lonafarnib Phase 2 trial in HDV, which was achieved in the fourth quarter of 2016, plus positive top line Phase 2 data from at least onefloating per annum interest rate of the ~~following programs: (i) Lambda in HDV, (ii) Exendin 9-39 in PBH based on the Company’s own IND, (iii) ubenimex in PAH, or (iv) ubenimex in Lymphedema.~~

~~The Oxford~~Innovatus Loan ~~bears interest at a floating rate per annum~~is equal to the sum of (a) the greater of ~~either~~(i) the ~~30-day U.S. Dollar LIBOR reported~~Prime Rate published in the Money Rates section of the Wall Street Journal (or any successor thereto) and (ii) 3.5%, plus ~~6.41% or 6.95%.~~(b) 3.75%; provided that, at the election of the Borrower, up to 2.25% of such rate shall be payable in-kind until the third anniversary of the closing date. The Company is required to ~~repay the Tranche A in 18~~make monthly ~~interest only~~interest-only payments followed by 36 equal monthly payments of principal and interest commencing on the first day of the month following the funding of Tranche A. If the Company receives the Tranche B funds, then the interest only period is extended by six months followed by 30 equal monthly payments of principal plus accrued interest. At the time of final payment,through July 1, 2027, after which the Company is required to ~~pay an exit fee of 7.5%~~make monthly amortizing payments, with the remaining balance of the ~~original~~ principal ~~balance~~plus accrued and unpaid interest due at maturity. 2.25% of ~~each tranche, which will be $1.1 million~~the interest is payable in-kind for ~~Tranche A. The Company recorded as a liability and debt discount~~ the ~~exit fee at the origination~~first three years of the term ~~loan. In addition,~~by increasing the ~~Company incurred loan origination fees and debt issuance costs of $0.3 million which were recorded as a~~ ~~direct deduction from the carrying amount~~principal balance. Prepayments of the ~~related debt liability~~.loan, in whole or in part, will be subject to an early prepayment fee which declines each year until the third anniversary date of the Closing Date, after which no prepayment fee is required. The Company is also required to pay ~~a 5.0% success~~an exit fee ~~within 30 days following the FDA’s approval~~upon any payment or prepayment equal to 6.5% of the ~~Company’s first product. This fee is enforceable within 10 years from the funding of Tranche A. In connection with the execution~~aggregate principal amount of the tranches funded under the Innovatus Loan. The Innovatus Loan ~~Agreement,~~contains customary representations, warranties, events of default and covenants of the Company ~~agreed~~and its subsidiaries, including a requirement to ~~certain customary representations~~maintain a cash balance of not less than 5% of the aggregate principal amount of funded and ~~warranties.~~

outstanding loan terms at all times. The ~~loan~~Innovatus Loan is secured by ~~the~~ perfected first priority liens on the Company's assets, including a commitment by the Company to not allow any liens to be placed upon the ~~Company’s~~Company's intellectual property. The Oxford Loan includes customary events of default, including failure to pay amounts due, breaches of covenants and warranties, material adverse effect events, certain cross defaults and judgments, and insolvency. If the Company is unable to comply with these covenants or if the Company defaults on any portion of the outstanding borrowings, the lenders can also impose a 5.0% penalty and restrict access to additional borrowings under the loan and security agreement.

The Company was funded $40.0 million in ~~compliance with~~June 2022 on the ~~terms~~Closing Date under Tranche A. The remaining $35.0 million is divided into two tranches (Tranche B and Tranche C). The $17.5 million under each of Tranche B and Tranche C will be available for a period commencing on the ~~Oxford Loan as~~later of ~~September~~(a) the first date that the Company achieves certain development and regulatory milestones applicable to each Tranche and (b) November 1, 2022. Both Tranche B and Tranche C draw periods end on the earlier of (a) June 30, ~~2017 and December 31, 2016.~~

2024 or (b) an event of default. The Company is ~~permitted~~currently eligible to ~~make voluntary prepayments of~~draw the ~~Oxford Loan with a prepayment fee, calculated~~$17.5 million under Tranche B, but has not done so as of ~~the loan origination date, equal to (i) 3.0% of the loan prepaid during the first 12 months, (ii) 2.0% of the loan prepaid in months 13-24 and (iii) 1.0% of the loan prepaid thereafter.~~ March 31, 2023.

The Company ~~is required to make~~identified a number of embedded derivatives that require bifurcation from the Innovatus Loan. These embedded features include mandatory ~~prepayments of the outstanding loan~~prepayment upon ~~the acceleration by lender following the occurrence of~~ an event of default ~~along~~or change in control and contingent rate increases. However, the fair value of these embedded features was deemed to be immaterial on the date of issuance. At each subsequent reporting period, the Company will reassess the fair value of the embedded features and will record a liability if the fair value of the features becomes material.

In connection with the issuance of the Innovatus Loan, the Company recorded a debt discount of $0.2 million and capitalized debt issuance costs of $1.1 million. The discount and issuance costs will be amortized over the life of the loan. Interest expense for the Innovatus Loan for the three months ended March 31, 2023 was $1.3 million, and is inclusive of non-cash amortization of the debt discount and debt issuance costs and accretion of final payment. The carrying amount of the Innovatus Loan at March 31, 2023 was $40.0 million. The carrying amount of the Innovatus Loan approximates fair value given its recent issuance. The effective interest rate for the Innovatus Loan was 13.35% as of March 31, 2023.

Additionally, in connection with entering into the Innovatus Loan, the Company entered into a Stock Purchase Agreement with Innovatus for the sale of common stock with an aggregate value of $5.0 million. On June 1, 2022, the Company issued 749,053 shares of common stock to Innovatus at a per share purchase price of $6.6751, the preceding five-day volume weighted average price per share.

A portion of the loan proceeds were used to repay in full theapproximately $33.5 million of aggregate principal amount, unpaid interest, and exit fees in connection with loans outstanding owed to Oxford Finance LLC (the Oxford Loan) by the Company.

Oxford Term Loan

On June 1, 2022, upon entering into the Innovatus Loan, the Company repaid the Oxford Loan, including (i) the $30.0 million outstanding principal balances, (ii) $0.2 million in accrued and unpaid interest, and (iii) other final payments consisting of $3.3 million, for a total payment of $33.5 million. The Company recorded a loss of $1.1 million on early extinguishment of the ~~final payment,~~debt related to the ~~prepayment fee~~unamortized debt premium, discount, and ~~any~~cost of issuance, which was recognized as a component of other ~~obligations that are due and payable at~~(expense) income, net in the ~~time~~condensed consolidated statement of ~~prepayment.~~

operations.

The Company accounts for the amortization of the debt discount utilizing the effective interest method. ~~The Company recorded interest expense of $0.3 million and $0.8 million for the three and nine months ended September 30, 2017. Long-term debt~~Debt and unamortized discount balances are as follows (in thousands):

September 30,

December 31,

2017

2016

Face value of term loan

$
15,000

$
15,000

Exit fee

1,125

1,125

Unamortized debt discount associated with exit fee, debt issuance costs and loan
origination fees

(1,132
)

(1,398
)
Term loan, net

$
14,993

$
14,727


	March 31, 2023		December 31, 2022
Face value of debt	$	40,756	$	40,531
Exit fee	2,600		2,600
Unamortized debt discount associated with exit fee, debt issuance costs and loan origination fees	(3,367)		(3,506)
Total debt, net	$	39,989	$	39,625

6.	~~Sale of Assets~~

7. Stock-Based Compensation

In ~~May 2017,~~June 2016, the Company’s Board of Directors adopted and in August 2016 the Company’s stockholders approved the Amended and Restated 2013 Equity Incentive Plan (Restated 2013 Plan). As of March 31, 2023, there were 2,232,008 shares available for grant under the Restated 2013 Plan.

During the second quarter of 2021, the Company ~~and Theragene Pharmaceuticals, Inc. (“Theragene”) entered into an asset purchase agreement (“Theragene APA”), whereby~~approved the ~~Company sold all of the assets related~~2021 Inducement Plan to MYDICAR including any related intellectual property for a cash payment of $0.2 million and 475,000 shares of common stock of Theragene. At any time after the Theragene APA execution date and until Theragene has received cumulative gross proceeds of $4.0 million (“Proceeds Date”) from all equity financing transactions occurring after the Theragene APA execution date, if Theragene issues additional shares of its common stock without consideration or for a consideration per share less than $6.00 per share then Theragene will issue additionalreserve 850,000 shares of its common stock to be used exclusively for grants of awards to individuals that were not previously employees or directors of the Company ~~concurrently~~as a material inducement to such individuals’ entry into employment with ~~such issue, in an amount such that the per share consideration multiplied by the aggregate number of common stock shares issued to~~ the Company ~~will equal $2.85 million. Additionally,~~within the ~~Company may exercise a put option at any time after the Proceeds Date, where upon written notice from the Company, Theragene will repurchase the 225,000 shares~~meaning of ~~its common stock held by the Company (“Option Shares”) at an aggregate purchase price equal to the greater of $1.35 million or the aggregate fair market value~~Rule 5635(c)(4) of the Option Shares as of the date of the receipt notice. The Company is also eligible to receive contingent consideration up to a maximum $45.0 million in cash, based upon Theragene achieving certain specified future milestones. In addition, the Company is also eligible to receive up to 8% royalties on net sales of any future Theragene products covered by or involving the related patents or know-how until the 20^th ~~anniversary of the Theragene APA.~~

~~The Company has determined that the sale of the MYDICAR assets qualify as an asset sale and not a business.~~

Nasdaq Listing Rules. As of ~~September 30, 2017,~~March 31, 2023, there were 1,510,000 shares remaining and available to be issued under the Company received a cash payment of $0.2 million, which was recognized as other income. Concurrently with the execution of the Theragene APA, the Company became an owner of 475,000 shares of common stock of Theragene and a put option for 225,000 shares of common stock of Theragene, which were recognized as a cost method investment with carrying value of zero.

2021 Inducement Plan.

7.	~~Stock Based Compensation~~

The following table summarizes stock option activity under the Company’s ~~stock based~~stock-based compensation ~~plan~~plans during the ~~nine~~three months ended ~~September 30, 2017~~March 31, 2023 (in thousands, except option and share data):

Shares
Available for
Grant

Number of
Options

Weighted-
Average
Exercise
Price

Weighted-
Average
Remaining
Contractual
Life
(in Years)

Aggregate
Intrinsic
Value

Outstanding as of December 31, 2016

646,778

1,212,044

$
14.06

$
2,414

Additional options authorized

417,883

Granted

(652,200
)

652,200

$
10.91

Exercised

(5,688
)

$
2.06

Canceled and forfeited

328,162

(328,162
)

$
13.89

Outstanding as of September 30, 2017

740,623

1,530,394

$
12.80

8.31

$
2,529

Vested and exercisable as of September 30, 2017

601,330

$
14.28

7.32

$
1,286


	Number of Options	Weighted- Average Exercise Price		Weighted- Average Remaining Contractual Life (in Years)	Aggregate Intrinsic Value
Outstanding as of December 31, 2022	6,143,183	$	9.00	6.00	$	—
Granted	2,234,978	$	1.48
Forfeited	(327,523)	$	5.20
Outstanding as of March 31, 2023	8,050,638	$	7.22	4.68	$	—
Vested and exercisable as of March 31, 2023	4,228,656	$	9.92	4.19	$	—

~~Stock Options Granted to Employees~~

During the three ~~and nine~~ months ended ~~September 30, 2017, the Company granted employees stock options for 56,000~~March 31, 2023 and ~~579,700 shares, respectively. The~~ ~~weighted-average grant date fair value of these options was $7.45 and $7.66 for the three and nine months ended September 30, 2017, respectively.~~ ~~During the three and nine months ended September 30, 2016, the Company granted employees stock options for 814,028 shares~~ ~~and~~2022, the weighted-average grant date fair value of ~~these~~ options ~~was $10.58.~~

granted were $1.48 and $3.23 per share, respectively.

The Company records stock-based compensation of stock options granted ~~to employees~~ by estimating the fair value of stock-based awards using the Black-Scholes option pricing model and amortizes the fair value of the stock-based awards granted over the applicable vesting period of the awards on a straight-line basis. The fair value of ~~employee~~ stock options was estimated using the following weighted-average assumptions:


	Three Months Ended September 30,			Nine Months Ended September 30,			Three Months Ended March 31,
	2017		2016	2017	2016		2023	2022
Expected term (in years)	5.77-6.08		5.27-6.08	5.27-6.08	5.27-6.08	Expected term (in years)	0.86-6.08	5.27-6.08
Volatility		79%	73.9%-77.1%	79.0%-80.0%	73.9%-77.1%	Volatility	90.21%-156.25%	68.71%-70.75%
Risk free interest rate	1.93%-2.01%		1.21%-1.31%	1.63%-2.23%	1.21%-1.31%	Risk free interest rate	3.56%-4.85%	1.76%-2.41%
Dividend yield	—		—	—	—	Dividend yield	—	—

Restricted Stock ~~Options Granted to Non-Employees~~

~~The Company grants stock options to non-employees in exchange for services rendered.~~ Units and Performance Stock Units

During the three ~~and nine~~ months ended ~~September 30, 2017,~~March 31, 2023, the Company granted ~~to non-employees~~15,000 performance stock ~~options for 20,000~~units (PSUs), with a weighted-average grant date fair value of $2.26 per share. There were no PSUs granted during the three months ended March 31, 2022. The performance-based metrics include the achievement of certain revenue targets and ~~72,500 shares, respectively.~~clinical and regulatory milestones. During the three ~~and nine~~ months ended ~~September 30, 2016,~~March 31, 2023 and 2022, the Company ~~granted to non-employees stock options for zero~~recorded $0.2 million and ~~48,544 shares, respectively. Stock-based~~$0.1 million, respectively, of stock-based compensation expense related to ~~stock options granted to non-employees~~the PSUs as it is ~~recognized~~expected that all milestones will be achieved by the target dates. This expense is included in selling, general and administrative expenses. As of March 31, 2023, other than 15,000 PSUs for which vesting was accelerated in connection with the modification noted below, no PSUs have vested as the performance-based metrics of the PSUs have not yet been achieved.

During the three months ended March 31, 2023 and 2022, the Company granted 39,167 and 298,150 restricted stock ~~options are earned and will fluctuate as the estimated~~units (RSUs), respectively, with a weighted-average grant date fair value of $2.26 and $5.22 per share, respectively. In relation to the ~~common stock fluctuates until~~RSUs granted, the ~~awards vest. The~~ Company ~~believes that the estimated fair value of the stock options is more readily measurable than the fair value of the services rendered.~~

~~The fair value of the stock options granted~~ ~~to non-employees~~ ~~is estimated at each reporting date using the Black-Scholes option-pricing model~~ ~~using similar assumptions as for employees except that the expected term is based on the options’ remaining contractual term instead of the simplified method:~~

Three Months Ended September 30,

Nine Months Ended September 30,

2017

2016

2017

2016
Remaining contractual term (in years)

6.0-10.0

7.0-9.67

6.0-10.0

7.0-10.0
Volatility

83.6%-94.8%

89.4%-96.4%

83.6%-97.7%

84.4%-96.4%
Risk free interest rate

1.90%-2.33%

1.40%-1.60%

1.90%-2.42%

1.37%-2.04%
Dividend yield

—

—

—

—

~~Stock-Based Compensation Expense~~

~~Total~~recognized $0.4 million and $0.4 million in stock-based compensation expense ~~recognized~~ for ~~options granted to employees~~the three months ended March 31, 2023 and ~~non-employees was as follows (in thousands):~~

Three Months Ended September 30,

Nine Months Ended September 30,

2017

2016

2017

2016

Research and development

$
273

$
126

$
771

$
428

General and administrative

680

1,114

2,452

1,323

Total

$
953

$
1,240

$
3,223

$
1,751

2022, respectively, which were included in selling, general and administrative expenses. As of ~~September 30, 2017,~~March 31, 2023, the total unrecognized compensation expense related to unvested ~~employee~~RSUs and PSUs was $2.5 million, which the Company expects to recognize over an estimated weighted-average period of 2.0 years.

The following table summarizes RSU and PSU activity and weighted average grant date fair value for the three months ended March 31, 2023:


	Shares	Weighted- Average Grant Date Fair Value
Unvested shares as of December 31, 2022	641,407	$	7.08
Granted	54,167	$	2.26
Vested	(192,147)	$	5.12
Forfeited	(13,456)	$	7.44
Unvested shares as of March 31, 2023	489,971	$	7.31

Awards Modification

On February 6, 2023, the Company entered into a separation agreement and general release with David Cory, the Company's former President and Chief Executive Officer. Pursuant to the separation agreement, 50% of Mr. Cory's unvested equity awards were accelerated to vest on the date the separation agreement was executed. Additionally, the exercise period for Mr. Cory's vested awards was extended, including his accelerated awards. The stock compensation recognized related to these modifications was $0.9 million, which is reflected in selling, general and administrative expenses.

Stock-Based Compensation Expense

Total stock-based compensation expense recognized was as follows (in thousands):


	Three Months Ended March 31,
	2023		2022
Research and development	$	834	$	625
Selling, general and administrative	1,708		1,422
Total	$	2,542	$	2,047

As of March 31, 2023, the total unrecognized compensation expense related to unvested options was ~~$7.5~~$9.7 million, which the Company expects to recognize over an estimated weighted average period of ~~2.9~~2.7 years.

8.	~~Income Taxes~~

8. Income Taxes

The Company’s provision for income taxes was approximately $2,000 and $9,000 for the three months ended March 31, 2023 and 2022, respectively, with an effective tax rate of 0.01% and 0.04% for the three months ended March 31, 2023 and 2022, respectively. The effective tax rate in each period differs from the U.S. statutory tax rate primarily due to the valuation allowances on the Company’s deferred tax assets as it is more likely than not that some or all of the Company’s deferred tax assets will not be realized. The tax expense recorded for the three ~~and nine~~ months ended ~~September 30,~~March 31, 2023, and 2022, respectively, relates to state taxes.

9. Commitments and Contingencies

Lease Agreements

In October 2017, the Company entered into a non-cancelable operating facility lease agreement for 8,029 square feet of office space located at 2155 Park Boulevard in Palo Alto, California. The lease commenced on March 1, 2018 and was ~~zero due~~ to expire in February 2023. The lease had a three-year renewal option prior to expiration. The lease included rent escalation clauses throughout the lease term. In October 2017, the Company provided a security deposit of $0.3 million. In February 2023, the Company amended the lease to extend the lease by one year with a one year renewal option. The extended lease commenced on March 1, 2023 and expires on February 28, 2024. The Company accounted for the amendment as a lease modification in accordance with ASC Topic 842. The Company also has additional operating leases that are included in its lease accounting but are not considered material for disclosure.

The maturities of the Company’s ~~loss position and full valuation allowance. This is consistent with~~operating lease liabilities as of March 31, 2023 were as follows (in thousands):


Undiscounted lease payments	March 31, 2023
Remaining in 2023	$	375
2024	84
2025	1
Total undiscounted payments	460
Less: imputed interest	24
Present value of future lease payments	436
Less: current portion of operating lease liabilities	435
Operating lease liabilities	$	1

Rent expense recognized for the ~~zero-tax expense~~Company’s operating leases was $0.1 million for the three ~~and nine~~ months ended ~~September 30, 2016.~~

9.	~~Legal Matters~~

~~In July 2015, following Celladon’s announcements~~March 31, 2023 and 2022. Under the terms of the ~~negative CUPID 2 data and~~lease agreements, the ~~suspension of further research and development activities and~~Company is also responsible for certain variable lease payments that are not included in the ~~subsequent declines~~measurement of the ~~price~~lease liability. Variable lease payments for the operating leases were $15,000 and $23,000 for the three months ended March 31, 2023 and 2022, respectively.

The operating cash outflows for the operating lease liabilities were $0.2 million for the three months ended March 31, 2023 and 2022. As of ~~its common stock, three~~March 31, 2023 and December 31, 2022, the weighted-average remaining lease terms were 0.9 years and 1.2 years, and weighted-average discount rates were 12.82% and 12.82%, respectively.

Legal Matters

On November 8, 2022 a putative securities class ~~actions were~~action complaint was filed in the ~~U.S.~~United States District Court for the ~~Southern~~Northern District of California ~~against Celladon and certain of its current and former officers. The complaints generally alleged~~alleging that the ~~defendants~~company and two former executives violated Sections 10(b) and 20(a) of the Securities Exchange Act ~~of 1934, as amended (the “Exchange Act”), by making materially false~~ and ~~misleading statements~~SEC Rule 10b-5. The complaint alleged generally that between March 2021 and October 2022 material misstatements and omissions were made to shareholders regarding the ~~clinical trial program~~TOGETHER study of peginterferon lambda for ~~MYDICAR, thereby artificially inflating~~ the ~~price~~treatment of ~~Celladon’s common stock.~~COVID-19 as well as the likelihood of FDA approval of an Emergency Use Authorization for peginterferon lambda. The ~~complaints sought unspecified monetary damages and other relief, including attorneys’ fees. On December 9, 2015, the district court consolidated the three putative securities class actions and~~Court appointed a lead plaintiff ~~to represent the putative class. The lead plaintiff filed a consolidated amended complaint~~ on ~~February 29, 2016.~~

March 2, 2023. On October 7, 2016, the district court granted defendants’ motion to dismiss the consolidated amended complaint and granted leave to amend within 60 days from the date of the district court’s order. The lead plaintiff subsequently filed a notice of intent not to amend the consolidated amended complaint and instead indicated that it intended to appeal the district court’s decision. On December 9, 2016, the district court closed the case.

~~On December 28, 2016,~~April 10, 2023, the lead plaintiff filed a notice ~~to the United States Court~~ of Appeals for the Ninth Circuit appealing the district court’s order dismissing the consolidated amended complaint. On May 5, 2017, the lead plaintiff and appellant filed his opening appellate brief. On July 5, 2017, defendants filed their answering appellate brief.

It is possible that additional suits will be filed, or allegations made by stockholders, with respect to these same or other matters and also naming the Company and/or Celladon’s former officers and directors as defendants. The Company believes that it has meritorious defenses and intends to defend these lawsuits vigorously. Due to the early stagevoluntary dismissal without prejudice.

10. Net Loss Per Share

Basic net loss per share of ~~these proceedings, the Company is not able to predict or reasonably estimate the ultimate outcome or possible losses relating to these claims.~~

~~10.~~

~~Commitments and Contingencies~~

~~Lease Agreement~~

In March 2017, the Company entered into a non-cancelable facility lease agreement for an office facility in Palo Alto, California. The lease commenced on April 1, 2017 and expires 12 months after the commencement date. The lease has one twelve-month renewal option prior to expiration and includes rent escalation clauses through the lease term. In April 2017, the Company provided a security deposit of $27,000 as collateral for the lease. The ~~future minimum rent payable under the new lease agreement is approximately $0.3 million during the next year.~~

~~There were no other changes in commitments and contingencies during the three and nine months ended September 30, 2017.~~

~~11.~~

~~Subsequent Events~~

On October 27, 2017, the Company entered into an underwriting agreement (the “Underwriting Agreement”) with BTIG, LLC (the “Underwriter”) relating to the offering, issuance and sale (the “Offering”) of 1,854,749 shares of the Company’s common stock ~~par value $0.001 per share. The price in~~is calculated by dividing the ~~Offering was $10.00 per share. The Underwriter had a 30-day option to purchase up to an additional 278,212~~net loss by the weighted average number of shares of common stock ~~which~~outstanding during the period, without consideration for potentially dilutive securities. Since the Company was ~~exercised on October 27, 2017. On October~~in a loss position for the three months ended March 31, ~~2017, pursuant to~~2023 and 2022, diluted net loss per share is the ~~Underwriting Agreement,~~same as basic net loss per share as the ~~Underwriters purchased~~inclusion of all potential common shares ofoutstanding would have been anti-dilutive. Dilutive potential common stock ~~from~~equivalents include the ~~Company at a price~~assumed exercise, vesting and issuance of ~~$9.40~~employee stock awards using the treasury stock method.

The following table sets forth the outstanding potentially dilutive securities which have been excluded in the calculation of diluted net loss per share ~~which resulted in approximately $19.5 million of net proceeds to the Company after deducting the underwriting discount and offering expenses payable by the Company. The shares of~~because including such securities would be anti-dilutive (in common stock ~~are listed on The NASDAQ Global Market. All~~equivalent shares):


	Three Months Ended March 31,
	2023	2022
Options to purchase common stock	8,050,638	6,490,451
Restricted and Performance stock units (unvested)	489,971	802,819
ESPP	291,887	87,553
Total	8,832,496	7,380,823

ITEM 2. Management’s Discussion and Analysis of ~~the shares in the Offering were sold by the Company. Pursuant to the terms~~Financial Condition and Results of the Underwriting Agreement, each of the Company’s directors and officers have entered into “lock-up” agreements with Underwriter that generally prohibit, without prior written consent of BTIG, LLC, the sale, transfer or other disposition of securities of the Company for a period ending 75 days after October 27, 2017. The Offering was made pursuant to the Company’s effective shelf registration statement on Form S-3 (Registration No. 333-212114) filed with the Securities and Exchange Commission (the “SEC”) on June 17, 2016 and declared effective on August 4, 2016.

Operations

~~ITEM 2.~~

~~Management’s~~ ~~Discussion and Analysis of~~ ~~Financial Condition and Results of Operations~~

You should read the following discussion and analysis of ~~Eiger’s~~Eiger BioPharmaceuticals, Inc.'s (Eiger) financial condition and results of operations together with our unaudited condensed consolidated financial statements and related notes thereto included in Part I, Item 1 of this Quarterly Report on Form 10-Q, ~~Eiger’s~~and our consolidated financial statements and related notes thereto for the year ended December 31, ~~2016,~~2022, included in our Annual Report on Form 10-K filed with the Securities and Exchange Commission (“SEC”) on March ~~23, 2017.~~17, 2023. This discussion and other parts of this report contain forward-looking statements that involve risks and uncertainties, such as our plans, objectives, expectations, intentions and beliefs. Our actual results could differ materially from those discussed in these forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, those identified below and those discussed in the section entitled “Risk Factors” included elsewhere in this report.

Forward-Looking Statements

This Quarterly Report contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”). These statements involve known and unknown risks, uncertainties and other factors that may cause our actual results, performance or achievements to be materially different from any future results, performances or achievements expressed or implied by the forward-looking statements In some cases, forward-looking statements are identified by words such as “believe,” “will,” “may,” “estimate,” “continue,” “anticipate,” “intend,” “should,” “plan,” “expect,” “predict,” “could,” “potentially” or the negative of these terms or similar expressions. You should read these statements carefully because they discuss future expectations, contain projections of future results of operations or financial condition, or state other “forward-looking” information. These statements relate to, among other ~~thing,~~things, our future plans, objectives, expectations, intentions, the potential for our programs, the timing of our clinical trials and financial performance and the assumptions that underlie these statements. These forward-looking statements are subject to certain risks and uncertainties that could cause actual results to differ materially from those anticipated in the forward-looking statements. Factors that might cause such a difference include, but are not limited to, those discussed in this Quarterly Report on Form 10-Q in Part II, Item 1A — “Risk Factors,” and elsewhere in this Quarterly Report on Form 10-Q. Forward-looking statements are based on our management’s beliefs and assumptions and on information currently available to our management. These statements, like all statements in this Quarterly Report on Form 10-Q, speak only as of their date, and we undertake no obligation to update or revise these statements in light of future developments. We caution investors that our business and financial performance are subject to substantial risks and uncertainties.

Overview

We are a ~~clinical stage~~commercial-stage biopharmaceutical company focused on ~~bringing~~the development of innovative therapies for hepatitis delta virus (HDV) and other serious diseases. All five of our rare disease programs have FDA Breakthrough Therapy designation.

Our HDV platform includes two first-in-class therapies in Phase 3 that target critical host processes involved in viral replication. Lonafarnib is a first-in-class, oral farnesylation inhibitor, and peginterferon lambda is a first-in-class, type III, interferon.

D-LIVR is the pivotal Phase 3 study of lonafarnib boosted with ritonavir, alone or in combination with peginterferon alfa-2a, for HDV. The study completed enrollment of 407 patients, and we announced topline Week 48 data in December 2022, with Week 72 data expected by end of the second quarter 2023. LIMT-2 is the pivotal Phase 3 study of peginterferon lambda for HDV. The study has completed screening and is expected to ~~market novel~~complete randomization of approximately 150 patients by end of the second quarter 2023.

We are also developing avexitide, a first in class, well-characterized GLP-1 antagonist, as a targeted treatment for two metabolic diseases with high unmet medical needs and no approved therapies: congenital hyperinsulinism (HI) and post-bariatric hypoglycemia (PBH). Avexitide has completed Phase 2 for both indications, and we initiated Phase 3 study start-up activities for HI in 2022. In connection with our Phase 3 start-up activities, we observed low levels of product-related impurities in the finished drug product. Although not unusual for this class of compounds, we are working with our Contract Manufacturing Organizations (CMOs) to control and qualify these materials and plan to initiate dosing in Phase 3 when an adequate supply of materials with a sufficient shelf-life has been released.

The FDA approved our first commercial product, ~~candidates~~Zokinvy (lonafarnib), to reduce the risk of mortality of Hutchinson-Gilford progeria syndrome (HGPS) and for treatment of processing-deficient progeroid laminopathies (PL) with either heterozygous LMNA mutation with progerin-like protein accumulation, or homozygous or compound heterozygous ZMPSTE24 mutations, on November 20, 2020. Collectively known as progeria, these are ultra-rare and rapidly fatal genetic conditions of accelerated aging in children. On July 20, 2022, we announced that the European Commission (EC) granted Eiger a centralized marketing authorization (MA) under the exceptional circumstances procedure for Zokinvy for the treatment of ~~orphan diseases. Since~~progeria. The MA is subject to the EMA’s continued review on an annual basis of new efficacy and safety information which may become available. The EC's centralized MA is valid in all 27 EU member states plus Iceland, Liechtenstein, and Norway. Regulatory review is ongoing by the UK's Medicine and Healthcare products Regulatory Agency (MHRA) as part of the UK's European Commission Decision Reliance Procedure, which has been extended to apply across Great Britain until December 31, 2023.

We commercially launched Zokinvy in the U.S. in January 2021 and in Europe in November 2022 and started to record product revenue in the first quarter of 2021. The European Commission's centralized marketing authorization is valid in all 27 EU member states as well as Iceland, Liechtenstein, and Norway. Our first European sales were recognized in the fourth quarter of 2022.

We have initiated a formal program prioritization assessment with a focus on enhancing long-term shareholder value while fulfilling the promise of advancing our founding in 2008, we have worked with investigators at Stanford University and evaluated a number of potential development candidates from pharmaceutical companies to comprise a pipeline of novel product candidates. Our resulting pipeline includes four Phase 2 candidates addressing four distinct orphan diseases. The programs have several aspects in common: the disease targets represent conditions of high medical need which are inadequately treated by current standard of care; the therapeutic approaches are supported by an understanding of disease biology and mechanism as elucidated by our academic research relationships; prior clinical experience with thehigh-potential product candidates ~~guides an understanding of safety; and the development paths leverage the experience and capabilities of our experienced, commercially-focused management team. The pipeline includes lonafarnib~~ for Hepatitis Delta Virus, or HDV, lambda for HDV, exendin 9-39 for Post-Bariatric Hypoglycemia, or PBH, and ubenimex for Pulmonary Arterial Hypertension, or PAH, and lymphedema. We plan to deliver data from all ongoing Phase 2 clinical trials over the course of the next fifteen months.

patients with serious diseases.

We have ~~no products approved for commercial sale and have not generated any revenue from product sales. We have never been profitable and have~~historically incurred operating losses in each year since inception, and we ~~do not anticipate that we will achieve profitability in~~expect to incur losses for the ~~near term. Our~~foreseeable future. We had a net ~~losses were $31.6~~loss of $22.8 million and ~~$34.3~~$22.6 million for the ~~nine~~three months ended ~~September 30, 2017~~March 31, 2023 and ~~2016,~~2022, respectively. As of ~~September 30, 2017,~~March 31, 2023, we had an accumulated deficit of ~~$107.9~~$460.0 million. Substantially all of our operating losses have resulted from expenses incurred in connection with our research and development programs and from selling, general and administrative costs associated with our operations.

We expect to incur significant expenses and increasing operating losses for at least the next several years as we initiate and continue the clinical development of, and seek regulatory approval for, and potentially commercialize our product candidates, which will require the addition of new personnel and ~~add personnel necessary~~upgrades to operate as a public company with an advanced clinical candidate pipeline of products. In addition, we are now operating as a publicly traded company following the merger with Celladon in 2016, and we have and will be hiring additional financial and other personnel, upgrading our ~~financial~~ information ~~systems and incurring costs associated with operating as a public company.~~technology systems. We expect that our operating losses will fluctuate significantly from quarter to quarter and year to year due to timing of clinical development programs and efforts to achieve additional regulatory ~~approval.~~

~~Merger~~approvals. We have initiated a formal program prioritization assessment with ~~Celladon~~

a focus on enhancing long-term shareholder value while fulfilling the promise of advancing our high-potential product candidates for patients with serious diseases.

Recent Developments

On ~~March 22, 2016,~~April 13, 2023, we ~~completed~~ announced the ~~merger between Private Eiger~~appointments of William G. Kachioff, as Chief Financial Officer, and ~~Celladon in accordance with the terms of the Agreement~~James A. Vollins as General Counsel, Chief Compliance Officer and ~~Plan of Merger, dated as of November 18, 2015, by and among Private Eiger, Celladon and Celladon Merger Sub, Inc., or the Merger. Also on March 22, 2016, in connection with, and prior~~Corporate Secretary, to the ~~completion~~Company’s management team.

Prior to joining Eiger, Mr. Kachioff has served as interim CFO at Aduro Biotech (now Chinook Therapeutics) and CFO at GenomeDx Biosciences (now Veracyte), Biocept, Althea Technologies (now Ajinomoto Bio-Pharma) and MicroIslet.

Prior to joining Eiger, Mr. Vollins has served as General Counsel, Chief Compliance Officer and Corporate Secretary at BioDelivery Sciences International, Inc. and Bio Products Laboratory Limited.

Financial Operations Overview

Product Revenue, Net

Our product revenue, net consists of sales of Zokinvy® (lonafarnib) for HGPS and processing-deficient progeroid laminopathies in the ~~Merger, we effected~~United States and under a ~~fifteen for one reverse stock split~~reimbursed early access program, or cohort ATU program, in France. In November 2022, sales of Zokinvy launched commercially in Europe through our ~~common stock, or the Reverse Stock Split,~~wholly owned subsidiary in Ireland.

Cost of Sales

Cost of sales consists primarily of direct and ~~changed our name to “Eiger BioPharmaceuticals, Inc.”~~

On November 18, 2015, in connection with the Merger, we entered into a subscription agreement, or the “Subscription Agreement” with investors for the sale of shares of our common stock, or the “Private Placement”, which closed on March 22, 2016.

Immediately prior to and in connection with the Merger, each share of Private Eiger’s preferred stock outstanding was converted into shares of Private Eiger’s common stock at an exchange ratio of one share of common stock for each share of preferred stock.

Under the terms of the Merger Agreement, at the effective time of the Merger, Celladon issued shares of common stock to Private Eiger stockholders, at an exchange ratio of approximately 0.09 shares of common stock, after taking into account the Reverse Stock Split, in exchange for each share of Private Eiger’s common stock outstanding immediately priorindirect costs related to the ~~Merger. The exchange ratio was calculated by a formula that was determined through arms-length negotiations between Celladon~~manufacturing of Zokinvy for commercial sale, including third-party manufacturing costs, third party logistics costs, write down of inventories, and Private Eiger. Immediately after the Merger, the former Private Eiger equity holders beneficially owned approximately 78% of post-merger Eiger’s common stock. The Merger was accounted for as a reverse asset acquisition.

~~Financial Operations Overview~~

other period costs.

Research and Development Expenses

Research and development expenses represent costs incurred to conduct research and development, such as the development of our product candidates. We recognize all research and development costs as they are incurred. Research and development expenses consist primarily of the following:

•expenses incurred under agreements with consultants, contract research organizations and clinical trial sites that conduct research and development activities on our behalf;

•laboratory and vendor expenses related to the execution of clinical trials;

•contract manufacturing expenses, primarily for the production of clinical trial supplies;

•license fees associated with our license agreements; and

•internal costs that are associated with activities performed by our research and development organization and generally benefit multiple programs. These costs are not separately allocated by product candidate. Unallocated internal research and development costs consist primarily of:

◦personnel costs, which include salaries, benefits and stock-based compensation expense;

◦allocated facilities and other expenses, which include expenses for rent and maintenance of facilities and depreciation expense; and

◦regulatory expenses and technology license fees related to development activities.

The largest component of our operating expenses has historically been the investment in clinical trials, including contract manufacturing ~~capabilities,~~arrangements, clinical trial material related costs and other research and development activities. However, we do not allocate internal research and development costs, such as salaries, benefits, stock-based compensation expense and indirect costs to product candidates on a program-specific basis. The following table shows our research and development expenses for the three ~~and nine~~ months ended ~~September 30, 2017~~March 31, 2023 and ~~2016~~2022 (in thousands):

Three Months Ended September 30,

Nine Months Ended September 30,

2017

2016

2017

2016

Product candidates:

Lonafarnib HDV

$
715

$
1,020

$
3,105

$
3,771

Lambda HDV

719

648

2,141

6,103

Exendin 9-39 PBH

721

1,104

2,540

1,919

Ubenimex PAH

1,994

3,189

7,633

7,299

Ubenimex Lymphedema

500

1,164

1,359

1,787

Internal research and development costs

1,496

947

4,962

2,758

Total research and development expense

$
6,145

$
8,072

$
21,740

$
23,637


	Three Months Ended March 31,
	2023		2022
Product candidates:
Lonafarnib	$	4,559	$	5,970
Peginterferon lambda	4,704		7,352
Avexitide	1,839		612
Internal research and development costs	5,646		3,636
Total research and development expense	$	16,748	$	17,570

We expect research and development expenses will continue to be significant and may increase in the future as we advance our product candidates into and through later stage clinical trials and pursue regulatory approvals, which will require a significant investment in regulatory support and contract manufacturing and clinical trial material related costs. In addition, in the future we ~~continue to~~may evaluate opportunities to acquire or in-license other product candidates and technologies, which may result in higher research and development expenses due to license ~~fee~~fees and/or milestone payments.

The process of conducting clinical trials necessary to obtain regulatory approval is costly and time consuming. We may never succeed in timely developing and achieving regulatory approval for our product candidates. The probability of success of our product candidates may be affected by numerous factors, including clinical data, competition, intellectual property rights, manufacturing capability and commercial viability. As a result, we are unable to determine the duration and completion costs of our development projects or when and to what extent we will generate revenue from the commercialization and sale of any of our product candidates.

The COVID-19 pandemic presents additional risks and uncertainties associated with developing drugs,as further described in Part II, Item 1A, “Risk Factors”.

Selling, General and Administrative Expenses

~~General~~

Selling, general and administrative expenses consist of personnel costs, allocated expenses, ~~and~~ expenses for outside professional services, including legal, audit, accounting services, insurance costs and costs associated with being a public ~~company.~~company, and commercial related expenses. Personnel costs consist of salaries, benefits and stock-based compensation. Allocated expenses consist of facilities and other expenses, which include direct and allocated expenses for rent and maintenance of facilities, depreciation expense and other supplies. ~~During the nine months ended September 30, 2016, we incurred incremental expenses as a result of the Merger. As a result of becoming a public company following completion of the Merger,~~Our expenses include costs related to compliance with the rules and regulations of the SEC and ~~NASDAQ, additional~~Nasdaq, insurance, investor relations, banking fees and other administrative expenses and professional services.

We expect our selling, general and administrative expenses to increase in the future due to sales and marketing activities from the commercialization of our product candidates.

Interest Expense

Interest expense ~~for the three and nine months ended September 30, 2017,~~ consists of interest and amortization of the debt discount related to the Oxford Loan borrowing in December 2016. Interest expense for the nine months ended September 30, 2016, consists of interest and amortization of the debt discount related to the outstanding convertible promissory notes issued in November 2015 and then converted into common stock in March 2016, or “the Notes”.

on our long-term borrowings.

Interest Income

Interest income consists of ~~interests~~interest earned on our investments in ~~marketable~~debt securities and cash equivalents.

~~Other Income (Expense), Net~~

~~Other income, net for the three and nine months ended September 30, 2017, primarily consists of the payment received for MYDICAR sale.~~

Other expense, net for the nine months ended September 30, 2016, consists of the change in fair value of the obligation to issue common stock to Eiccose and the change in fair value of warrant liability.

The change in fair value of the obligation to issue common stock to Eiccose was related to our obligation to issue shares to Eiccose upon the closing of the next round of financing that resulted in at least $25.0 million in gross proceeds to us. Upon the closing of the Private Placement on March 22, 2016, we issued to Eiccose 96,300 fully vested shares of our common stock in settlement of this obligation. In connection with this transaction we remeasured the fair value of the obligation to issue common stock at the settlement date and the change in fair value of $0.2 million was recognized within other expense, net during the nine months ended September 30, 2016. Upon the settlement of the obligation with the issuance of shares on March 22, 2016, the liability was reclassified to common stock and additional paid-in capital within stockholders’ equity.

In connection with our issuance of the Notes, we issued warrants to the noteholders to purchase shares of our common stock at an exercise price of $0.11 per share, on a post-Merger and post-Reverse Stock Split basis, or the Warrants. The number of shares into which the Warrants could be exercised was equal to the warrant coverage amount divided by the per share price of the equity securities sold in a qualified financing and thus was accounted for as a liability. Upon the closing of the Private Placement on March 22, 2016, the number of shares of common stock issuable upon exercise of the Warrants was fixed and the fair value remeasured at that date, and the warrants were automatically exercised. During the nine months ended September 30, 2016, we recognized a loss related to the change in fair value of the warrant liability of $0.2 million. The warrant liability was reclassified to common stock and additional paid-in capital within stockholders’ equity, upon the exercise of the Warrants and issuance of shares on March 22, 2016.

Critical Accounting Policies and Estimates

Our management’s discussion and analysis of financial condition and results of operations is based on our unaudited condensed consolidated financial statements, which have been prepared in accordance with generally accepted accounting principles in the United States. The preparation of these unaudited condensed consolidated financial statements requires us to make estimates and judgments that affect the reported amounts of assets, liabilities revenues, and expenses. On an ongoing basis, we evaluate these estimates and judgments. We base our estimates on historical experience and on various assumptions that we believe to be reasonable under the circumstances. These estimates and assumptions form the basis for making judgments about the carrying values of assets and liabilities and the recording of revenues and expenses that are not readily apparent from other sources. Actual results may differ materially from these estimates.

There ~~have been~~were no material changes to our critical accounting policies ~~during the nine months ended September 30, 2017~~ as ~~compared to the critical accounting policies~~ disclosed in our 2022 Annual Report on Form 10-K ~~for the year ended December 31, 2016.~~

filed with SEC on March 17, 2023.

Results of Operations

Comparison of the Three Months Ended ~~September 30, 2017~~March 31, 2023 and ~~2016~~

2022

Three Months Ended September 30,

$

%

2017

2016

Change

Change

Operating expenses:

Research and development

$
6,145

$
8,072

$
(1,927
)

24
%
General and administrative

2,727

3,264

(537
)

16
%
Total operating expenses

8,872

11,336

(2,464
)

Loss from operations

(8,872
)

(11,336
)

2,464

Interest expense

(388
)

—

(388
)

100
%
Interest income

98

—

98

100
%
Other income (expense), net

(8
)

(34
)

26

*%

Net loss

$
(9,170
)

$
(11,370
)

$
2,200

19
%
The following table summarizes results of operations for the three months ended March 31, 2023 and 2022 (in thousands):

*	~~Percentage not meaningful~~


	Three Months Ended March 31,				$ Change
	2023		2022
Product revenue, net	$	4,118	$	2,673	$	1,445	54	%
Total revenue	4,118		2,673		1,445		54	%
Costs and operating expenses:
Cost of sales	118		110		8		7	%
Research and development	16,748		17,570		(822)		(5)	%
Selling, general and administrative	9,515		6,813		2,702		40	%
Total costs and operating expenses	26,381		24,493		1,888		8	%
Loss from operations	(22,263)		(21,820)		(443)		2	%
Interest expense	(1,285)		(886)		(399)		45	%
Interest income	711		45		666		*
Other (expense) income, net	55		27		28		104	%
Loss before provision for income taxes	(22,782)		(22,634)		(148)		1	%
Provision for income taxes	2		9		(7)		(78)	%
Net loss	$	(22,784)	$	(22,643)	$	(141)	1	%

*Percentage not meaningful.

Product revenue, net

Product revenue, net increased by $1.4 million for the three months ended March 31, 2023 compared to the same period in 2022. The increase was primarily due to $0.6 million in Germany sales compared to no such sales for the same period in 2022, an increase of $0.5 million in France ATU sales, and an increase of $0.3 million in U.S. sales during the quarter.

Cost of sales

Cost of sales was flat for the three months ended March 31, 2023 compared to the same period in 2022.

Research and development expenses

Research and development expenses decreased by ~~$2.0 million to $6.1~~$0.8 million for the three months ended ~~September 30, 2017, from $8.1 million for~~March 31, 2023 compared to the same period in ~~2016.~~2022. The decrease was primarily due to a ~~$2.6~~$4.7 million decrease in ~~clinical~~milestone expenses related to the Phase 3 LIMT-2 study of peginterferon lambda for HDV under the BMS License Agreement, which occurred in March 2022, and ~~drug supply expenditures due to decreased program activity. The~~a $0.2 million decrease ~~was~~in outside services across programs including consulting and advisory services. These decreases were partially offset by a ~~$0.3~~$2.3 million increase in clinical and contract manufacturing expenditures, a $1.6 million increase in headcount related expenses, including stock-based compensation, and a $0.2 million increase in operational overhead costs.

Selling, general and administrative expenses

Selling, general and administrative expenses increased by $2.7 million to $9.5 million for the three months ended March 31, 2023, from $6.8 million for the same period in 2022. The increase was primarily due to a $1.3 million increase in outside services, including consulting, advisory and accounting services, a $1.3 million increase in compensation and personnel related expenses, including stock-based compensation, due to an increase in headcount, and ~~severance payments, $0.2 million in milestone payments to two Stanford University inventors, and a~~ $0.1 million increase in ~~facility and insurance~~other operating expenses, ~~related~~all to ~~increase in operating activity.~~

~~General and administrative expenses~~

~~General and administrative expenses decreased by $0.6 million to $2.7 million for~~support the three months ended September 30, 2017 from $3.3 million for the same period in 2016. The decrease was primarily due to a $0.4 million decrease in stock-based compensation expense due to changes in headcount and a higher level of stock-based compensation expense in the comparative third quarter 2016, and a $0.2 million decrease in legal, consulting, advisory and accounting services due to the incrementalCompany's operations.

Interest expenses ~~incurred as a result of the Merger in the first quarter 2016.~~

~~Interest expense~~

Interest expense increased by $0.4 million for the three months ended ~~September 30, 2017, and consisted~~March 31, 2023 compared to the same period in 2022. The increase was primarily due to an increase in the outstanding principal balance of ~~interest and amortization of the debt discount~~our long-term borrowings related to the ~~Oxford Loan borrowings in December 2016. There was no interest expense~~Innovatus loan.

Interest income

Interest income increased by $0.7 million for the three months ended ~~September 30, 2016.~~

~~Interest income~~

~~Interest income decreased by $0.1 million for the three months ended September 30, 2017, due~~March 31, 2023 compared to the interest earned on our investments in marketable securities and cash equivalents. There was no interest income earned for the three months ended September 30, 2016, as funds were not invested until the fourth quarter in 2016.

~~Comparison of the Nine Months Ended September 30, 2017 and 2016~~

Nine Months Ended September 30,

$

%

2017

2016

Change

Change

Operating expenses:

Research and development

$
21,740

$
23,637

$
(1,897
)

8
%
General and administrative

9,195

9,574

(379
)

4
%
Total operating expenses

30,935

33,211

(2,276
)

Loss from operations

(30,935
)

(33,211
)

2,276

Interest expense

(1,129
)

(685
)

(444
)

65
%
Interest income

321

—

321

100
%
Other income (expense), net

188

(423
)

611

144
%
Net loss

$
(31,555
)

$
(34,319
)

$
2,764

8
%

~~Research and development expenses~~

~~Research and development expenses decreased by $1.9 million to $21.7 million for the nine months ended September 30, 2017 from $23.6 million for~~ the same period in ~~2016.~~2022. The ~~decrease~~increase was primarily ~~due to a~~ ~~$5.2 million upfront payment under our License Agreement with Bristol Myers Squibb Company~~ that was recognized in the second quarter 2016. There were no similar payments during the same period in 2017. The decrease was offset by a $1.6 million increase in compensation and personnel related expenses due to an increase in headcount, a $0.9 million increase in clinical expenditures due to increased program activity, a $0.4 million increase in stock-based compensation expense due to an increase in headcount, $0.2 million in milestone payments to two Stanford University inventors, and a $0.2 million increase in facility and insurance expenses related to increase in operating activity.

~~General and administrative expenses~~

~~General and administrative expenses~~interest rates.

Provision for income taxes

Provision for income taxes decreased by ~~$0.4 million~~$7,000 compared to ~~$9.2 million for the nine months ended September 30, 2017 from $9.6 million for~~ the same period in ~~2016.~~2022. The ~~decrease~~change was primarily due to a $2.4 million decrease in legal, consulting, advisory and accounting services due to the incremental expenses incurred as a result of the Merger in the first quarter 2016. The decrease was partially offset by $1.1 million increase in stock-based compensation expense and a $0.6 million increase in compensation and personnel related expenses due to an increase in headcount, a $0.2 million increase in facility and insurance expenses and a $0.1 million increase in franchise tax expense ~~due to increased operating activity.~~

~~Interest expense~~

Interest expense increased by $0.4 million to $1.1 million for the nine months ended September 30, 2017 from $0.7 million for the same period in 2016. Interest expense in 2017 consisted of interest and amortization of the debt discount related to the Oxford Loan borrowings in December 2016. Interest expense in 2016 consisted of interest and amortization of the debt discount related to the Notes outstanding prior to their conversion into common stock in March 2016.

state taxes.

~~Interest income~~

Interest income increased by $0.3 million for the nine months ended September 30, 2017, due to the interest earned on our investments in marketable securities and cash equivalents. There was no interest income earned for the nine months ended September 30, 2016, as funds were not invested until the fourth quarter in 2016.

~~Other income (expense), net~~

Other income (expense), net increased by $0.6 million to $0.2 million of other income, net for the nine months ended September 30, 2017 from $0.4 million of other expense, net for the same period in 2016. Other income in 2017 primarily consisted of the payment received from Theragene for MYDICAR sale. Other expense in 2016 primarily consisted of the change in fair value of the obligation to issue common stock to Eiccose and the change in fair value of warrant liability, that were settled upon the Merger.

Liquidity and Capital Resources

Sources of Liquidity

As of ~~September 30, 2017,~~March 31, 2023, we had ~~$13.1~~$75.3 million of cash, cash equivalents and short-term debt securities, comprised of $15.2 million of cash and cash equivalents ~~$19.3~~and $60.1 million of ~~marketable~~short-term debt securities available-for-sale, and an accumulated deficit of ~~$107.9~~$460.0 million.

On ~~October~~June 1, 2022, we entered into a loan and security agreement with Innovatus Life Sciences Lending Fund I, LP (Innovatus), providing for up to $75.0 million funded in three tranches with a maturity date of August 31, ~~2017,~~2027. We were funded $40.0 million in June 2022 at the closing, of which approximately $33.5 million was used to pay off loans outstanding owed to Oxford Finance LLC (the Oxford Loan). We recorded a loss of $1.1 million on early extinguishment of the debt which was recognized as a component of Other (expense) income, net in the consolidated statement of operations and comprehensive loss. As part of the agreement with Innovatus, we are required to maintain a cash balance of

not less than 5% of the aggregate principal amount of funded and outstanding term loans at all times. We are currently eligible to draw the $17.5 million under Tranche B, but have not done so as of March 31, 2023.

Additionally, in connection with entering into the Innovatus Loan, we entered into a Stock Purchase Agreement with Innovatus for the sale of common stock with an aggregate value of $5.0 million. On June 1, 2022, we issued ~~2,132,961~~749,053 shares of common stock to Innovatus at a per share purchase price of $6.6751. Refer to Note 6 of our unaudited condensed consolidated financial statements included within Item 1 of this Quarterly Report on Form 10-Q for additional details.

On March 25, 2022, we entered into a new Open Market Sale AgreementSM with Jefferies, pursuant to ~~an underwriting agreement with BTIG, LLC at~~which we can sell up to a ~~public offering price~~maximum of ~~$10.00 per share,~~$50.0 million of our common stock in offerings that are deemed “at the market” offerings as defined in Rule 415 under the Securities Act, under Eiger’s effective shelf registration statement (the 2022 ATM Facility). In April 2022, we completed offerings from the 2022 ATM facility for ~~gross~~a total of 2,686,288 shares of our common stock resulting in net proceeds of ~~$21,329,610, which resulted in approximately $19.5~~$20.8 million ~~of net proceeds to the Company~~, after deducting ~~underwriting fees and offering expenses.~~ commissions costs. No additional offerings were completed since April 2022. As of March 31, 2023, there was approximately $28.7 million remaining under the 2022 ATM Facility for future issuance.

We believe that the currently available resources will be sufficient to fund our planned operations for at least the next 12 months following the issuance date of these unaudited condensed consolidated financial statements. However, if our anticipated operating results are not achieved in future periods, we believe that planned expenditures may need to be reduced ~~in order to extend the time period over which the then-available resources~~or we would be ~~able~~required to raise funding in order to fund our operations.

Additionally, our ability to raise additional capital may be adversely impacted by potential worsening global economic conditions and the recent disruptions to, and volatility in, the credit and financial markets in the United States and worldwide resulting from the COVID-19 pandemic, among other events.

Our primary uses of cash are to fund operating expenses, including research and development expenditures and selling, general and administrative expenditures. Cash used to fund operating expenses is impacted by the timing of when we pay these expenses, as reflected in the change in outstanding accounts payable and accrued expenses.

Future Funding Requirements

~~We have~~

Prior to 2021, we had not generated any ~~revenue from~~ product ~~sales.~~revenue. We ~~do not know when, or if, we will generate any revenue from~~launched our first commercial product, ~~sales.~~Zokinvy, in January 2021. We ~~do not expect to generate any revenue from product sales unless and until we obtain regulatory approval for and commercialize any of our product candidates. At the same time, we~~ expect our expenses to increase in connection with our ongoing development and manufacturing activities, particularly as we continue the research, development, manufacture and clinical trials of, and seek regulatory approval for our product candidates.

Our primary uses of capital are, and we expect will continue to be, funding ~~research efforts and~~ the development of our product candidates, sales and marketing costs for commercialization of Zokinvy and other product candidates, compensation and related expenses, hiring additional staff, including clinical, scientific, operational, manufacturing, financial, and management personnel, and costs associated with operating as a public company. We expect to incur substantial expenditures in the foreseeable future for the development and potential commercialization of our product candidates.

We plan to continue to fund losses from operations and capital funding needs through future equity and/or debt financings, as well as potential additional collaborations or strategic partnerships with other companies. As a result of economic conditions, general global economic uncertainty, political change and other factors, we do not know whether additional capital will be available when needed, or that, if available, we will be able to obtain additional capital on reasonable terms. If we are unable to raise additional capital due to the volatile global financial markets, general economic uncertainty or other factors, we may need to curtail planned development or commercialization activities. The sale of additional equity, including pursuant to the 2022 ATM Facility, or convertible debt could result in additional dilution to our stockholders. The incurrence of indebtedness would result in debt service obligations and could result in operating and financing covenants that would restrict our operations. We can provide no assurance that financing will be available in the amounts we need or on terms acceptable to us, if at all. If we are not able to secure adequate additional funding we may be forced to delay, make reductions in spending, extend payment terms with suppliers, liquidate assets where possible, and/or suspend or curtail planned programs. Any of these actions could materially harm our business.

Cash Flows

The following table summarizes our cash flows for the periods indicated (in thousands):


	Nine Months Ended September 30,							Three Months Ended March 31,
	2017			2016				2023		2022
Net cash used in operating activities	$	(27,979	)	$	(26,832	)	Net cash used in operating activities	$	(24,005)	$	(18,459)
Net cash provided by investing activities		13,148			27,965
Net cash provided by (used) in investing activities							Net cash provided by (used) in investing activities	13,383		(3,002)
Net cash provided by financing activities		153			50,383		Net cash provided by financing activities	41		45,802
Net (decrease) increase in cash and cash equivalents	$	(14,678	)	$	51,516
Effect of foreign exchange on cash and cash equivalents							Effect of foreign exchange on cash and cash equivalents	(36)		—
Net increase in cash and cash equivalents							Net increase in cash and cash equivalents	$	(10,617)	$	24,341

Cash flows from operating activities

Cash used in operating activities for the ~~nine~~three months ended ~~September 30, 2017~~March 31, 2023 was ~~$28.0~~$24.0 million, ~~consisting~~which primarily consisted of a net loss of ~~$31.6~~$22.8 million, ~~which was partially offset by~~ stock-based compensation expense of ~~$3.2~~$2.5 million, non-cash interest expense of $0.4 million, amortization of debt securities discount of $0.4 million, reduction in the carrying amount of right-of-use assets of $0.1 million and depreciation and amortization of $0.1 million. Additionally, cash used in operating activities reflected changes in net operating assets ~~and liabilities primarily~~ due to an increase of ~~$1.5~~$2.1 million in accounts ~~payable. The increase was partially offset by $1.1~~receivable, a decrease of $2.7 million ~~decrease~~ in accounts payable and accrued ~~and other~~ liabilities ~~associated with~~primarily due to the timing of ~~payments.~~

payments, an increase of $0.9 million in inventories and a decrease of $0.1 million in operating lease liabilities partially offset by a decrease of $1.4 million in prepaid expenses and other current assets and a decrease of $0.5 million in other assets.

Cash used in operating activities for the ~~nine~~three months ended ~~September 30, 2016~~March 31, 2022 was ~~$26.8~~$18.4 million, which primarily ~~consisting~~consisted of a net loss of ~~$34.3~~$22.6 million, ~~offset by $3.2 million expense related to a non-cash issuance of common stock to Bristol-Meyers Squibb in connection with the BMS License Agreement, $1.8 million of~~ stock-based compensation expense ~~$0.7~~of $2.0 million, amortization of debt securities discount of $0.3 million, non-cash interest expense ~~related to the convertible promissory notes outstanding prior to their conversion into common stock in March 2016~~of $0.2 million, depreciation and ~~$0.4~~amortization of $0.1 million ~~change in fair value~~and amortization of ~~warrant liability and obligation to issue shares to Eiccose.~~operating lease right-of-use assets of $0.1 million. Additionally, cash used in operating activities reflected changes in net operating assets ~~and liabilities primarily~~of $1.4 million due to an increase of ~~$1.6~~$1.9 million in accounts payable and accrued liabilities due to timing of payments, a decrease of $0.7 million in inventory and a decrease of $0.3 million in accounts receivable, which were partially offset by an increase of $0.9 million in prepaid expenses and other ~~liabilities primarily associated with~~current assets, an increase of $0.4 million in ~~business activity.~~

other assets and a decrease of $0.2 million in operating lease liabilities.

Cash flows from investing activities

~~Cash~~ provided by investing activities was $13.1 million for the nine months ended September 30, 2017, and primarily consisted of $35.5 million proceeds from maturities of marketable securities and $0.2 million proceeds from MYDICAR sale, which was partially offset by $22.5 million purchase of marketable securities.

Cash provided by investing activities for the ~~nine~~three months ended ~~September 30, 2016~~March 31, 2023 was $13.4 million, primarily ~~resulted~~consisting of $33.0 million of proceeds from ~~the $28.0~~maturities of debt securities, which were partially offset by $19.4 million of purchases of debt securities and $0.2 million of ~~cash received upon~~purchases of property and equipment.

Cash used in investing activities was $3.0 million for the ~~consummation~~three months ended March 31, 2022, primarily consisting of ~~the Merger.~~

$10.9 million of purchases of debt securities, which were partially offset by $7.9 million of proceeds from maturities of debt securities.

Cash flows from financing activities

Cash provided by financing activities for the ~~nine~~three months ended ~~September 30, 2017~~ ~~primarily~~ ~~consisted~~March 31, 2023 was $41,000 of proceeds from ~~the~~ ~~purchase~~ issuance of common stock ~~under our ESPP.~~

upon ESPP purchases.

Cash provided by financing activities for the ~~nine~~three months ended ~~September 30, 2016~~March 31, 2022 was $45.8 million, which primarily consisted of ~~$32.1~~$45.6 million of net proceeds from the issuance of common stock inunder the ~~private placement, net of issuance costs,~~ 2020 ATM Facility and ~~net proceeds of $18.2~~$0.2 million ~~from the issuance~~ of common stock ~~in the underwritten public offering on August 23, 2016, after deducting underwriting discounts~~upon stock option exercises and ~~commissions and expenses payable by us.~~

ESPP purchases.

Contractual Obligations and Other Commitments

~~The following table summarizes our~~

Our contractual obligations atas of March 31, 2023 have not materially changed from what we presented in our Annual Report on Form 10-K for the year ended December 31, ~~2016 (in thousands):~~

Payments due by period

Total

Less
than
1 year

1 – 3
Years

3 – 5
Years

More
than
5 years

Operating lease obligations (1)

$
401

$
330

$
71

$
—

$
—

Term loan debt (2)

$
15,000

$
—

$
12,083

$
2,917

$
—

Interest on term loan debt (3)

$
4,378

$
979

$
2,205

$
1,194

$
—

Total

$
19,779

$
1,309

$
14,359

$
4,111

$
—

~~(1)~~

~~Represents future rent payments under two Palo Alto facility lease contracts.~~

2022 filed with the SEC on March 17, 2023.

~~(2)~~

~~Represents the Oxford first tranche Loan of $15.0 million.~~

~~(3)~~

~~Includes an exit fee on the Oxford Loan of $1.125 million due at maturity.~~

In March 2017, we entered into a non-cancelable facility lease agreement for an office facility in Palo Alto, California. The lease commenced on April 1, 2017 and expires 12 months after the commencement date. The lease has one 12-month renewal option prior to expiration and includes rent escalation clauses through the lease term. In April 2017, we provided a security deposit of $27,000 as collateral for the lease. The ~~operating lease obligation amounts above do not reflect our future minimum rent payable under the new lease agreement of approximately $0.3 million during the next year.~~

We are obligated to make future payments to third parties under asset purchase and license agreements, including royalties and payments that become due and payable on the achievement of certain development and commercialization milestones. We have not included these potential payment obligations in the table above as the amount and timing of such payments are not known.

Off-Balance Sheet Arrangements

During the periods presented, we did not have, nor do we currently have, any off-balance sheet arrangements as defined under the rules of the SEC and do not have any holdings in variable interest entities.

~~ITEM 3.~~

ITEM 3. Quantitative ~~and Qualitative Disclosures about Market Risk~~

~~As of September 30, 2017, we had market risk exposure related to our cash~~ and ~~cash equivalents.~~ Qualitative Disclosures about Market Risk

We ~~had cash and cash equivalents of $13.1 million and $19.3 million of short-term marketable securities. Such interest-earning instruments carry~~are a degree of interest rate risk; however, historical fluctuations of interest income have not been significant. We have not been exposed nor do we anticipate being exposed to material risks due to changes in interest rates. A hypothetical 10% change in interest rates during anysmaller reporting company as defined by Rule 12b-2 of the ~~periods presented would~~Securities Exchange Act of 1934 and are not ~~have had a material impact on our unaudited condensed consolidated financial statements.~~

~~Financial instruments that potentially subject us~~required to concentration of credit risk consist of cash and cash equivalents. We place our cash and cash equivalents with high credit quality financial institutions and pursuant to our investment policy, we limitprovide the amount of credit exposure with any one financial institution. Deposits held with banks may exceed the amount of insurance provided on such deposits. We have not experienced any losses on our deposits of cash and cash equivalents.

We carry out some of our clinical development and supportive activities in foreign countries and payments may be due in foreign currencies. We do not participate in any foreign currency hedging activities and we do not have any other derivative financial instruments. We did not recognize any significant exchange rate losses during the three and nine months ended September 30, 2017.

information under this item.

~~ITEM 4.~~

ITEM 4. Controls and Procedures

Evaluation of Disclosure Controls and Procedures

~~Our management, with the participation of our Chief Executive Officer and Chief Financial Officer, has evaluated the effectiveness of our~~

We are responsible for maintaining disclosure controls and procedures, ~~(as~~as defined in Rules 13a-15(e) and 15d-15(e) under the ~~Securities~~Exchange Act. Disclosure controls and procedures are controls and other procedures designed to ensure that the information required to be disclosed by us in the reports that we file or submit under the Exchange Act is recorded, processed, summarized, and reported within the time periods specified in the SEC’s rules and forms. Disclosure controls and procedures include, without limitation, controls and procedures designed to ensure that information required to be disclosed by us in the reports that we file or submit under the Exchange Act is accumulated and communicated to our management, including our principal executive officer and our principal financial officer, as appropriate to allow timely decisions regarding required disclosure.

Based on our management’s evaluation (with the participation of ~~1934,~~our principal executive officer and our principal financial officer) of our disclosure controls and procedures as ~~amended),~~required by Rule 13a-15 under the Exchange Act, our principal executive officer and our principal financial officer have concluded that our disclosure controls and procedures were effective to achieve their stated purpose as of March 31, 2023, the end of the period covered by this Quarterly Report on Form 10-Q. Based on such evaluation, our Chief Executive Officer and Chief Financial Officer have concluded that, as of the end of the period covered by this quarterly report, our disclosure controls and procedures were, in design and operation, effective.

report.

Changes in Internal Control

over Financial Reporting

There were no ~~material~~ changes in our internal control over financial reporting (as defined in Rules 13a-15(f) and 15d-15(f) under the Exchange Act) that occurred during the three ~~and nine~~ months ended ~~September 30, 2017,~~March 31, 2023 that have materially affected, or are reasonably likely to materially affect, our internal control over financial reporting.

~~Limitations on Effectiveness of Controls and Procedures~~

In designing and evaluating the disclosure controls and procedures and internal control over financial reporting, management recognizes that any controls and procedures, no matter how well designed and operated, can provide only reasonable assurance of achieving the desired control objectives. In addition, the design of disclosure controls and procedures and internal control over financial reporting must reflect the fact that there are resource constraints and that management is required to apply judgment in evaluating the benefits of possible controls and procedures relative to their costs.

PART II. OTHER INFORMATION

~~ITEM 1.~~

ITEM 1. LEGAL PROCEEDINGS

~~See Note 9~~

We do not list all routine litigation matters with which we are a party. We discuss below certain pending matters. In determining whether to discuss a pending matter, we consider both quantitative and qualitative factors to assess materiality, such as, among others, the amount of damages alleged and the nature of other relief sought, if specified; our view of the ~~Notes~~merits of the claims and of the strength of our defenses; and whether the action purports to be, or is, a class action the jurisdiction in which the proceeding is pending.

Schoen v. Eiger BioPharmaceuticals, Inc., et al., Case No. 22-cv-06985

On November 8, 2022 a putative securities class action complaint was filed in the United States District Court for the Northern District of California alleging that the company and two former executives violated Sections 10(b) and 20(a) of the Securities Exchange Act and SEC Rule 10b-5. The complaint alleged generally that between March 2021 and October 2022, material misstatements and omissions were made to shareholders regarding the TOGETHER study of peginterferon lambda for the treatment of COVID-19 as well as the likelihood of FDA authorization of an Emergency Use Authorization for peginterferon lambda. The Court appointed a lead plaintiff on March 2, 2023. On April 10, 2023, the lead plaintiff filed a notice of voluntary dismissal without prejudice.

The Progeria Research Foundation, Inc. v. Eiger BioPharmaceuticals, Inc. Arbitration

On November 15, 2022, the Company received a demand for arbitration (Demand) from claimant The Progeria Research Foundation, Inc. (PRF) asserting two claims under a May 15, 2018 Collaboration and Supply Agreement (the PRF Collaboration Agreement) between the parties. PRF has alleged that the Company breached an obligation to supply quantities of a drug as requested by PRF. PRF also has a claim for declaratory relief regarding the grant of licenses under the PRF Collaboration Agreement. On January 18, 2023, the Company filed a response to the ~~unaudited Condensed Consolidated Financial Statements contained within this Quarterly Report~~Demand denying PRF’s claims, contesting the arbitrability of PRF’s claim for declaratory relief, and asserting a counterclaim for declaratory relief related to the contractual provision underlying PRF’s original drug supply claim. To give the parties an opportunity to discuss a potential negotiated resolution of their dispute, the arbitration has been suspended through the end of 2023. As a result, all arbitration activities are now on ~~Form 10-Q~~hold, and the final hearing, originally scheduled for ~~a further discussion of our legal proceedings.~~

May 9 – 12, 2023, in Boston, Massachusetts, has been taken off calendar.

~~ITEM 1A.~~

ITEM 1A. RISK FACTORS

You should carefully consider the following risk factors, as well as the other information in this Quarterly Report on Form 10-Q, and in our other public filings. The occurrence of any of the following risks could harm our business, financial condition, results of operations and/or growth prospects or cause our actual results to differ materially from those contained in forward-looking statements we have made in this Quarterly Report on Form 10-Q and those we may make from time to time. You should consider all of the risk factors described when evaluating our business.

Risk Factor Summary

Below is a summary of material factors that make an investment in our common stock speculative or risky. Importantly, this summary does not address all of the risks and uncertainties that we face. Additional discussion of the risks and uncertainties summarized in this risk factor summary, as well as other risks and uncertainties that we face, can be found under “Risk Factors” in Part II, Item 1A of this Quarterly Report. The below summary is qualified in its entirety by that more complete discussion of such risks and uncertainties. You should carefully consider the risks and uncertainties described under “Risk Factors” in Part II, Item 1A of this Quarterly Report as part of your evaluation of an investment in our common stock.

•We are a commercial-stage biopharmaceutical company with additional product candidates in clinical development and a limited operating history. We have ~~marked~~incurred net losses in each year since our inception. We have one U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA)-approved product for commercial sale, Zokinvy (lonafarnib), and prior to 2021, have never generated any product revenue and may never be profitable.

•We are dependent on the success of our product candidates, which are in various stages of clinical development. We cannot give any assurance that we will generate data for any of our product candidates sufficient to receive regulatory approval and without regulatory approval we will not be able to market our product candidates.

•Prior to the approval of our new drug application (NDA) for Zokinvy® (lonafarnib) to reduce the risk of mortality in HGPS, and for treatment of processing-deficient progeroid laminopathies with either heterozygous LMNA mutation with progerin-like protein accumulation, or homozygous or compound heterozygous ZMPSTE24 mutations, we had not submitted an application for approval or obtained FDA approval for any product. We may not be able to obtain FDA approval of any future NDA or Biologics License Application (BLA) for our product candidates, which would prohibit commercialization.

•Our business strategy is based upon obtaining and maintaining Orphan Drug designation for our product candidates. If we are unable to obtain or maintain Orphan Drug designation or regulatory approval, our business would be substantially harmed.

•Our future success depends in part on our ability to attract, retain, and motivate qualified personnel.

•Product development involves a lengthy and expensive process with an ~~asterisk (*) those risk factors below~~uncertain outcome, and results of earlier studies may not be predictive of future study results.

•We rely on clinical studies of our product candidates in order to obtain regulatory approval. We may find it difficult to enroll patients in our clinical studies given the limited number of patients who have the diseases for which our product candidates are being studied.

•If clinical studies of our product candidates fail to demonstrate safety, efficacy, purity and/or potency to the satisfaction of the FDA or similar regulatory authorities outside the United States or do not otherwise produce positive results, we may incur additional costs or experience delays in completing, or ultimately be unable to complete, the development and commercialization of our product candidates.

•We rely on third parties to conduct our clinical studies, manufacture our product candidates and perform other services. Our ability to obtain regulatory approval or commercialize our product candidates and our business could be impaired if these collaborations are unsuccessful.

•If the market opportunities for our product candidates are smaller than we believe they are, we may not meet our revenue expectations. Because the patient populations in the market for our product candidates may be small, we must be able to successfully identify patients and acquire a significant market share to achieve profitability and growth.

•We face intense competition and rapid technological change and the possibility that ~~reflect significant changes~~our competitors may develop therapies that are similar, more advanced or more effective than ours.

•We currently have limited marketing and sales capabilities for the commercialization of our product candidates.

•The commercial success of any of our current or future product candidates will depend upon the degree of market acceptance by physicians, patients, third-party payors and others in the medical community. Sales of our products depend substantially on the extent to which the costs of our product candidates will be paid for or reimbursed by healthcare management organizations or government authorities or third-party payors.

•We are currently conducting and will continue to conduct clinical trials for our product candidates outside the United States, which could expose us to risks that could have a material adverse effect on our business, including risks in connection with the actions taken by the Russian Federation in Ukraine and surrounding areas.

•We may not be able to develop peginterferon lambda for COVID-19 or file for a temporary use authorization with a foreign regulatory agency.

•We intend to rely on a combination of exclusivity from Orphan Drug designation and our patent rights for our product candidates. If we are unable to maintain exclusivity from the ~~risk factors included~~combination of these approaches, then our ability to compete effectively in our ~~Annual Report~~markets may be harmed.

•The annual reassessment by the EMA of the risk-benefit balance for Zokinvy including information on ~~Form 10-K~~the safe and effective use may not be positive, which could lead to a variation, suspension, revocation of our marketing authorization or requirement to fulfil additional specific obligations.

•If we are unable to maintain effective proprietary rights for our product candidates, or if the ~~year ended December 31, 2016.~~

scope of the intellectual property protection is not sufficiently broad, our competitors could develop and commercialize product candidates similar or identical to ours.

•We may be unsuccessful in obtaining or maintaining necessary rights to our product candidates through acquisitions and in-licenses. If we fail to comply with obligations in the agreements under which we license intellectual property and other rights from third parties or otherwise experience disruptions to our business relationships with our licensors, we could lose license rights that are important to our business.

•We may not be successful in any efforts to identify, license, discover, develop or commercialize additional product candidates.

•Healthcare legislative reform measures may have a material adverse effect on our business and results of operations. If we fail to comply with environmental, health and safety laws and regulations, we could become subject to fines or penalties or incur costs.

•We face potential product liability, and, if successful claims are brought against us, we may incur substantial liability and costs.

•We are dependent upon information technology systems and any failure or security breach of such systems could result in a material disruption in the development of our product candidates or other business operations as well as result in statutory or contractual obligations or otherwise expose us to liability.

•Our future success depends in part on our ability to attract, retain, and motivate qualified personnel.

•The current COVID-19 pandemic has and may continue to adversely affect our financial condition and our business as well as those of third parties on which we rely on significant manufacturing, clinical or other business operations.

Risks Related to our Financial Condition, Integration and Capital Requirements

We have incurred losses since our inception, have a limited operating history on which to assess our business, and anticipate that we will continue to incur significant losses for the foreseeable future.*

We are a ~~clinical development-stage~~commercial-stage biopharmaceutical company with a limited operating history. We have incurred net losses in each year since our inception. For the ~~nine~~three months ended ~~September 30, 2017~~March 31, 2023 and ~~2016,~~2022, we reported a net loss of ~~$31.6~~$22.8 million and ~~$34.3~~$22.6 million, respectively. As of ~~September 30, 2017,~~March 31, 2023, we had an accumulated deficit of ~~approximately $107.9~~$460.0 million. Our prior losses, combined with expected future losses, have had and may continue to have an adverse effect on our stockholders’ equity and working capital.

We believe that ~~the~~our currently available resources will be sufficient to fund our planned operations for at least the next 12 months following the issuance date of these ~~unaudited condensed~~ consolidated financial statements. Our assumption does not reflect the possibility that we may not be able to access a portion of our existing cash, cash equivalents and short-term securities due to market and other conditions. For example, on March 10, 2023, the Federal Deposit Insurance Corporation (FDIC) was appointed receiver of Silicon Valley Bank (SVB), where we maintain an operating account with a balance that exceeded the FDIC insurance limit. Although, the cash in our accounts at Silicon Valley Bank did not impact our operations or our conclusion that we have sufficient capital to fund our operations for at least the next 12 months, we cannot be certain that future market corrections or other conditions will not impact our ability to access our cash, cash equivalents and short-term securities. On March 12, 2023, the U.S. Treasury Department, the Federal Reserve and the FDIC jointly announced enabling actions that fully protect all depositors of SVB and that such depositors would have access to all of their funds starting March 13, 2023. On March 26, 2023, the FDIC announced that First Citizens BancShares would acquire the commercial banking business of SVB. We continue to maintain an operating account at SVB and have begun measures to reduce our deposit concentration risk. If other banks and financial institutions enter receivership or become insolvent in the future in response to financial conditions affecting the banking system and financial markets, our ability to access our existing cash, cash equivalents and short-term securities may be threatened.

In addition, we will continue to require substantial additional capital to continue our clinical development, manufacturing and regulatory approval efforts and potential commercialization activities. Accordingly, we will need to raise substantial additional capital to continue to fund our operations. The ~~amount~~amounts and timing of our future funding requirements will depend on many factors, including our ability to achieve regulatory approval and the pace and results of our clinical development efforts. Failure to raise capital as and when needed, on favorable terms or at all, would have a negative impact on our financial condition and our ability to develop our product candidates.

We have devoted substantially all of our financial resources to identify, acquire, and develop our product candidates, including manufacturing of clinical supplies, conducting clinical studies and providing selling, general and administrative

support for our operations. To date, we have financed our operations primarily through the sale of equity securities and debt facilities. The amount of our future net losses will depend, in part, on the rate of our future expenditures and our ability to obtain funding through equity or debt financings, strategic collaborations, or grants. Biopharmaceutical product development is a highly speculative undertaking and involves a substantial degree of risk. We expect losses to increase as we advance ~~five Phase 2~~our clinical development programs ~~for potentially four indications. While we have not yet commenced pivotal~~in various clinical studies, particularly the D-LIVR pivotal study, to support the submission of an NDA for ~~any product candidate and it~~lonafarnib-based regimens for use in an HDV indication. We may need significant additional resources in order to aggressively move lonafarnib-based regimens forward successfully based on the discussions with the FDA. It may be several years, if ever, before we complete pivotal clinical studies and have aadditional product ~~candidate~~candidates approved for ~~commercialization, we~~commercialization. We expect to invest significant funds into ~~these~~our clinical candidates ~~to determine the potential~~ to advance these compounds to potential regulatory approval.

If we obtain regulatory approval to market aone or more additional product ~~candidate,~~candidates, our future revenue will depend upon the size of any markets in which our product candidates may receive approval, and our ability to achieve sufficient market acceptance, pricing, reimbursement from third-party payors, and adequate market share for our product candidates in those markets. Even if we obtain adequate market share for our product candidates, because the potential markets in which our product candidates may ultimately receive regulatory approval could be very small, we may never become profitable despite obtaining such market share and acceptance of our products.

We have also agreed with The Progeria Research Foundation to make Zokinvy available to progeria (HGPS and processing-deficient progeroid laminopathies) patients under an expanded access program that may not result in payment to us. Future clinical trials of new therapies for progeria conducted by third parties may also result in patients converting from commercially reimbursed Zokinvy to product provided through clinical trials and result in lower revenues received by us.

We expect to continue to incur significant expenses and increasing operating losses for the foreseeable future and our expenses will increase substantially if and as we:

•continue the clinical development of our product candidates;

•in-license or acquire additional product candidates;

•undertake the manufacturing or have manufactured our product candidates;

•advance our programs into larger, more expensive clinical studies;

•initiate additional nonclinical, clinical, or other studies for our product candidates;

•

~~initiate additional nonclinical, clinical, or other studies for our product candidates;~~

•commercialize and provide expanded access to Zokinvy for the treatment of HGPS and processing-deficient PL

•identify ~~educate~~ and develop potential commercial opportunities, such as ~~lonafarnib for HDV,~~lonafarnib-based regimens and peginterferon lambda for HDV, ~~exendin 9-39~~and avexitide for ~~PBH~~HI and ~~ubenimex for PAH and lymphedema;~~

PBH;

•seek regulatory and marketing approvals and reimbursement for our product candidates;

•establish a sales, marketing, and distribution infrastructure to commercialize any products for which we may obtain marketing approval and market ourselves;

•seek to identify, assess, acquire, and/or develop other product candidates;

•make milestone, royalty or other payments under third-party license agreements;

•develop and educate HDV markets;

•seek to maintain, protect, and expand our intellectual property portfolio;

•seek to attract and retain skilled personnel;

•create additional infrastructure to support our operations as a public company and our product development and planned future commercialization efforts; and

experience any delays or encounter issues with the development and potential for regulatory approval of our clinical candidates such as safety issues, clinical trial accrual delays, longer follow-up for planned studies, additional major studies, or supportive studies necessary to support marketing approval.

Further, the net losses we incur may fluctuate significantly from quarter to quarter and year to year, such that a period-to-period comparison of our results of operations may not be a representative indication of our future performance.

~~We have~~

Prior to 2021, we never generated any ~~revenue from~~ product ~~sales~~revenue and may never be profitable.

We have ~~no products~~one product approved for commercialization in the U.S. and ~~have never generated any revenue.~~EU for two ultra-rare diseases. Zokinvy works to (i) reduce the risk of mortality in HGPS, and (ii) treat processing-deficient progeroid laminopathies with either heterozygous LMNA mutation with progerin-like protein accumulation, or homozygous or compound heterozygous ZMPSTE24 mutations. Zokinvy was approved by the FDA in November 2020 and launched commercially in the U.S. in January 2021. In July 2022, our MAA for Zokinvy was approved by the EC, under the exceptional circumstances procedure. The Marketing Authorization (MA) is subject to the EMA’s continued review on an annual basis of new efficacy and safety information which may become available. Our ability to generate substantial revenue and achieve profitability depends on our ability to (i) obtain the regulatory and marketing approvals necessary to commercialize Zokinvy in foreign jurisdictions, alone or with strategic collaboration partners, and (ii) to successfully complete the development of, and obtain the regulatory and marketing approvals necessary to commercialize, ~~one or~~ more of our product ~~candidates.~~candidates in the U.S. or foreign jurisdictions. We do not anticipate generating ~~revenue from~~significant product ~~sales~~revenue for the foreseeable future. Our ability to generate future ~~revenue from~~ product ~~sales~~revenue depends heavily on our success in many areas, including, but not limited to:

•completing research and development of our product candidates;

•obtaining additional and maintaining current regulatory and marketing approvals for our product candidates;

•manufacturing product candidates and establishing and maintaining supply and manufacturing relationships with third parties that meet regulatory requirements and our supply needs in sufficient quantities to meet market demand for our product candidates, if approved;

•marketing, launching and commercializing product candidates for which we obtain regulatory and marketing approval, either directly or with a collaborator or distributor;

•gaining market acceptance of our product candidates as treatment options;

•addressing any competing products;

•protecting and enforcing our intellectual property rights, including patents, trade secrets, and know-how;

•negotiating favorable terms in and maintaining any collaboration, licensing, or other arrangements into which we may enter;

•obtaining reimbursement or pricing for our product candidates that supports profitability; and

•attracting, hiring, and retaining qualified personnel.

Even if ~~one or more of the~~we obtain additional product ~~candidates that we develop is approved~~approvals for commercial sale, we anticipate incurring significant costs associated with commercializing any approved product candidate. Our current pipeline of product candidates has been in-licensed from third parties and we will have to develop or acquire manufacturing capabilities in order to continue development and potential commercialization of our product candidates. Additionally, if we are not able to generate sufficient revenue from the sale of any approved products, we may never become profitable.

Raising additional capital may cause dilution to our stockholders, restrict our operations or require us to relinquish rights.

To the extent that we raise additional capital through the sale of equity, including pursuant to the 2022 ATM Facility, debt or other securities convertible into equity, ~~your~~stockholder ownership interest will be diluted, and the terms of these new securities may include liquidation or other preferences that adversely affect your rights as a common stockholder such as the ~~debt facility~~Loan and Security Agreement we entered into with ~~Oxford Finance~~Innovatus Life Sciences Lending Fund I, LP (Innovatus) in ~~December 2016. This~~June 2022 (the Innovatus Loan). The Innovatus Loan was a ~~$25.0~~$75.0 million debt financing arrangement with ~~Oxford Finance~~Innovatus wherein we borrowed the first tranche of ~~$15.0~~$40.0 million upon closing of the debt financing in December 2016. Our ability to access the final $10.0 million under the loan and security agreement is subject to our ability to achieve certain clinical development milestones, which we may not be able to meet and which and could adversely affect our liquidity.June 2022. The ~~loan~~Innovatus Loan is secured by ~~the~~ perfected first priority liens on ~~the Company's assets, including a commitment by the Company to not allow any liens to be placed upon the Company’s intellectual property.~~our assets. The ~~Oxford~~Innovatus Loan includes customary events of default, including failure to pay amounts due, breaches of covenants and warranties, material adverse effect events, certain cross defaults and judgments, and insolvency. If the Company is unable to comply with these covenants or if the Company default on any portion of the outstanding borrowings, the lenders can also impose a 5.0% penalty and restrict access to additional borrowings under the loan and security agreement.

If we raise additional funds through collaborations, strategic alliances or licensing arrangements with third parties, we may have to relinquish valuable rights to our product candidates or future revenue streams or grant licenses on terms that are not favorable to us. Our ability to raise additional capital may be adversely impacted by worsening global economic conditions and continuing disruptions to and volatility in the credit and financial markets in the U.S. and worldwide resulting from the ongoing COVID-19 pandemic. We cannot assure you that ~~we will~~ we will be able to obtain additional funding if and when necessary to fund our entire portfolio of product candidates to meet our projected plans. If we are unable to obtain funding

on a timely basis, we may be required to delay or discontinue one or more of our development programs or the commercialization of any product candidates or be unable to expand our operations or otherwise capitalize on potential business opportunities, which could materially affect our business, financial condition, and results of operations.

Covenants in ~~our loan and security agreement~~the Innovatus Loan restrict our business and operations in many ways, and if we do not effectively ~~manage~~comply with our covenants, our financial conditions and results of operations could be adversely affected. In addition, we may not meet the milestones required to access the final loan available under the agreement and may also not provide sufficient cash to meet the repayment obligations of our debt incurred under the loan and security agreement.

~~Our loan and security agreement with Oxford Finance LLC~~

The Innovatus Loan provides for up to ~~$25.0~~$75.0 million in term loans due on ~~July 1, 2021,~~August 31, 2027, of which ~~$15.0~~$40.0 million in ~~term loans has been borrowed to date.~~principal is outstanding as of March 31, 2023. All of our current and future assets, ~~except for intellectual property, are secured for~~secure our borrowings under the ~~loan and security agreement.~~Innovatus Loan. The ~~loan and security agreement~~Innovatus Loan requires that we comply with certain covenants applicable to us, including among other things, covenants restricting dispositions, changes in business, management, ownership or business locations, mergers or acquisitions, indebtedness, encumbrances, distributions, investments, transactions with affiliates and subordinated debt, any of which could restrict our business and operations, particularly our ability to respond to changes in our business or to take specified actions to take advantage of certain business opportunities that may be presented to us. Our failure to comply with any of the covenants could result in a default under the ~~loan and security agreement,~~Innovatus Loan, which could permit the lenders to declare all or part of any outstanding borrowings to be immediately due and ~~payable, or to refuse to permit additional borrowings under the loan and security agreement.~~payable. If we are unable to repay those amounts, the lenders under the ~~loan and security agreement~~Innovatus Loan could proceed against the collateral granted to them to secure that debt, ~~which~~and our inability to use or dispose of those assets would seriously harm our business. In addition, should we be unable to comply with these covenants or if we default on any portion of our outstanding borrowings, the lenders can also impose a 5.0% penalty, ~~and~~ restrict access to additional borrowings under the loan and security ~~agreement. Moreover, our ability to access~~agreement, and accelerate the ~~final $10.0 million~~maturity of the debt. Any default under the ~~loan and security agreement is subject to our ability to achieve certain clinical development milestones, which we may not be able to meet and which and could adversely~~Innovatus Loan would materially affect our liquidity. In addition, although we expect to borrow additional funds under the loan and security agreement, before we do so, we must first satisfy ourselves that we will have access to future alternate sources of capital, including cash flow from our own operations, equity capital markets or debt capital markets in order to repay any principal borrowed, which we may be unable to do, in which case, our liquidity and ability to fund our operations ~~may~~and complete our planned clinical trials and regulatory filings would be substantially impaired.

Risks Related to the Development of our Product Candidates

We are ~~heavily~~ dependent on the success of our product candidates, which are in ~~the early~~various stages of clinical development. Certain of our product candidates have produced results in academic settings to date or for other indications than those that we contemplate, and we cannot give any assurance that we will generate data for any of our product candidates sufficient to receive regulatory approval in our planned indications, which will be required before they can be commercialized.*

To date, we have invested substantially all of our efforts and financial resources to identify, acquire, and develop our portfolio of product candidates. Our future success is dependent on our ability to ~~successfully~~ further develop, obtain regulatory ~~approval~~approvals for, and commercialize one or more of these product candidates. ~~We currently generate no~~Our NDA for Zokinvy to reduce the risk of mortality in HGPS, and for treatment of processing-deficient progeroid laminopathies (PL) with either heterozygous LMNA mutation with progerin-like protein accumulation, or homozygous or compound heterozygous ZMPSTE24 mutations, was approved in November 2020. Prior to the U.S. Zokinvy commercial launch in 2021, we had not generated revenue from sales of any ~~drugs,~~products and we may never be able to develop or commercialize additional product candidates. In addition, we have a ~~product candidate.~~

commitment to provide access to Zokinvy for patients with HGPS and PL with either heterozygous LMNA mutation with progerin-like protein accumulation, or homozygous or compound heterozygous ZMPSTE24 mutations, for no or minimal cost to those patients.

WeWith respect to potential commercial products, we currently have ~~five Phase 2 development programs focused on four separate indications. For one of our~~two product candidates ~~ubenimex, data to date has~~that are in Phase 3 clinical development, lonafarnib-based regimens for HDV and peginterferon lambda for HDV. Phase 3 study startup activities for avexitide in HI have been developed for use in indications other than those that we have rights to or in which we plan to develop the product candidate and most of the data has been developed for a dose lower than in our current studies. Similarly, some of our lonafarnib results to date rely on an assay that has not been validated or accepted by the FDA to assess the potential efficacy of the product. While we have undertaken confirmation with an European Medical Agency approved assay, there can be no assurance that the data we have seen to dateinitiated. It may be ~~replicated in a FDA validated assay,~~years before our studies are completed, and new studies are initiated, if ~~available.~~

at all.

We provide our geographically diverse clinical sites with good clinical practice protocols. We review and monitor the execution of our protocols at our various sites in an effort to understand those protocols are being followed. There can be no assurance that the data we develop for our product candidates in our planned indications will be sufficient or complete enough to obtain regulatory approval.

~~In addition, none~~ Likewise, there can be no assurance that the data obtained from foreign clinical trial sites in studies not conducted under an investigational new drug application, or IND, will be accepted in support of ~~our product candidates have advanced into a pivotal study~~an application for ~~our proposed indications and it~~regulatory approval or authorization for usein the U.S. Similarly, data obtained from foreign clinical trial sites may not be ~~years before such study is initiated and completed, if at all.~~ accepted by other foreign regulatory authorities in support of an application for regulatory approval or authorization for use in these jurisdictions.

•We are not permitted to market or promote any of our product candidates before we receive regulatory approval from the FDA or comparable foreign regulatory authorities, and we may never receive such regulatory approval for any of our current product candidates. We cannot be certain that any of our product candidates will be successful in clinical studies or receive regulatory approval. Further, our product candidates may not receive

regulatory approval even if they are successful in clinical studies. If we do not receive regulatory approvals for our product candidates, we may not be able to continue our operations.

We may not be able to obtain FDA approval of any future NDA or BLA for our product candidates.

The clinical development, manufacturing, labeling, packaging, storage, recordkeeping, advertising, promotion, export, import, marketing and distribution and other possible activities relating to lonafarnib-based regimens, peginterferon lambda, avexitide and any other product candidate that we may develop in the future are subject to extensive regulation in the United States. Prior to the approval of our NDA for Zokinvy to reduce the risk of mortality in HGPS, and for treatment of PL with either heterozygous LMNA mutation with progerin-like protein accumulation, or homozygous or compound heterozygous ZMPSTE24 mutations, we had not submitted an application for approval or obtained FDA approval for any product.

Approval of an NDA or a BLA is not guaranteed, and the approval process is an expensive and uncertain process that may take several years. The FDA and foreign regulatory entities also have substantial discretion in the approval process. The number and types of preclinical studies and clinical trials that will be required for approval varies depending on the product candidate, the disease or the condition that the product candidate is designed to target and the regulations applicable to any particular product candidate. Data are subject to varying interpretation and the FDA may not agree that our clinical data support that any of our product candidates are safe and effective for the proposed therapeutic use. Despite the time and expense associated with preclinical studies and clinical trials, failure can occur at any stage, and we could encounter problems that require us to repeat or perform additional preclinical studies or clinical trials or generate additional chemistry, manufacturing and controls data, including product stability data. In previous studies, ECG abnormalities were observed in our lonafarnib program. The FDA and similar foreign authorities could delay, limit or deny approval of a product candidate, and may ultimately approve the product for narrower indications or with unfavorable labeling that would impede our commercialization of the product.

Approval procedures vary among countries and can involve additional product testing and additional administrative review periods, including obtaining reimbursement and pricing approval in select markets. The time required to obtain approval in other countries might differ from that required to obtain FDA approval. The regulatory approval process in other countries may include all of the risks associated with FDA approval as well as additional, presently unanticipated, risks. Regulatory approval in one country does not ensure regulatory approval in another, but a failure or delay in obtaining regulatory approval in one country may negatively impact the regulatory process in others, including the risk that our product candidates may not be approved for all indications requested and that such approval may be subject to limitations on the indicated uses for which the product may be marketed.

Our business strategy is based upon obtaining ~~orphan drug~~and maintaining Orphan Drug designation for our product candidates, which is an uncertain process. The regulatory approval processes of the FDA and comparable foreign authorities are lengthy, time consuming, and inherently unpredictable. If we are unable to obtain ~~orphan drug~~or maintain Orphan Drug designation or regulatory approval for our product candidates, our business ~~will~~would be substantially harmed.*

Our approach to identifying and developing product candidates depends, in large part, on our ability to obtain ~~orphan drug~~and maintain Orphan Drug designation from regulatory authorities in major markets. Without the potential protection of this regulatory exclusivity upon approval, many of our product candidates would otherwise not justify investment. In addition, the Company actively pursues new patents on all product candidates, such as the patent already issued covering PAH, that could potentially extend the market exclusivity beyond the available exclusivity provided by the orphan indication. While we assess the potential for obtaining ~~orphan drug~~Orphan Drug designation at the time that we contemplate the acquisition of product candidates and we intend to timely file for such designation, there can be no assurance that we will obtain ~~orphan drug~~Orphan Drug designation or be able to successfully meet the regulatory requirements to maintain that designation with the planned clinical trials for our product candidates. Failure to obtain ~~orphan drug~~and maintain Orphan Drug designation would make our product candidates significantly less competitive and potentially not viable investments for further development. Although we ~~already obtained orphan drug~~currently have Orphan Drug designation for ~~four~~some of our product candidates in ~~three~~multiple targeted indications, failure to demonstrate significant benefit over existing approved ~~drugs~~products in pivotal clinical trials may lead to marketing approval but without qualifying for ~~orphan drug~~Orphan Drug protection in some regions, such as in Europe.

The time required to obtain approval by the FDA and comparable foreign authorities is unpredictable, typically takes many years following the commencement of clinical studies and depends upon numerous factors. In addition, approval policies, regulations, or the type and amount of clinical data necessary to gain approval may change during the course of a product candidate’s clinical development and may vary among jurisdictions, which may cause delays in the approval or the decision not to approve an application. We have ~~not~~ obtained U.S. and EMA regulatory approval for ~~any~~one product, ~~candidate,~~Zokinvy, and

it is possible that none of our ~~existing~~current product candidates or any future product candidates we may seek to develop ~~in the future~~ will ever obtain regulatory approval.

~~Applications~~

Future applications for our product candidates could fail to receive regulatory approval for many reasons, including but not limited to the following:

•the FDA or comparable foreign regulatory authorities may disagree with the design, size or implementation of our clinical studies;

•the population studied in the clinical program may not be sufficiently broad or representative to assure safety in the full population for which we seek approval;

•the FDA or comparable foreign regulatory authorities may disagree with our interpretation of data from our development efforts;

•the data collected from clinical studies of our product candidates may not be sufficient or complete or meet the regulatory requirements to support the submission of an NDA, BLA, or other submission or to obtain regulatory approval in the United States or foreign jurisdictions;

•

the data collected from clinical studies of our product candidates may not be sufficient or complete or meet the regulatory requirements to support the submission of a new drug application, or NDA, or other submission or to obtain regulatory approval in the United States or foreign jurisdictions. For example, we identified significant good clinical practice (GCP) violations in the LOWR HDV – 1 and LOWR HDV – 2 studies. We alerted the FDA to the discovery of GCP violations. These findings may bring into question the validity of the data set and may require repeat clinical studies to confirm the observations in question. Further studies may be needed to confirm the rigor, robustness and validity of any problematic safety and efficacy data identified;

•the FDA or comparable foreign regulatory authorities may find failures in our manufacturing processes, validation procedures and specifications, or facilities of our third-party manufacturers with which we contract for clinical and commercial supplies that may delay or limit our ability to obtain regulatory approval for our product candidates; and

•the approval policies or regulations of the FDA or comparable foreign regulatory authorities may significantly change in a manner rendering our NDA, BLA or other submission insufficient for approval.

The lengthy and uncertain regulatory approval process, as well as the unpredictability of the results of clinical studies, may result in our failing to obtain additional regulatory approval to market any of our product candidates or to be significantly delayed from our expectations for potential approval, which would significantly harm our business, results of operations, and prospects. In addition, although we have obtained ~~orphan drug~~Orphan Drug designation for ~~four~~five of our ~~product candidates in our planned indications~~development programs to date, there can be no assurance that the FDA or comparable foreign regulatory authorities will grant our similar status for our other proposed development indications or other product candidates in the future.

Although the FDA has granted Rare Pediatric Disease designation to avexitide for the treatment of congenital hyperinsulinism, NDA approval for this program may not meet the eligibility criteria for a priority review voucher.

Our avexitide compound has received Rare Pediatric Disease (RPD) designation from the FDA for the treatment of Congenital Hyperinsulinism (HI). The FDA defines a “rare pediatric disease” as a serious or life-threatening disease in which the serious or life-threatening manifestations primarily affect patients under the age of 18 years, that is, a disease or condition that affects fewer than 200,000 individuals in the United States, or a patient population greater than 200,000 in the United States when there is no reasonable expectation that the cost of developing and making available the product in the United States will be recovered from sales in the United States for that drug or biological product. Specifically, under this program, a sponsor who receives an approval for a drug or biologic for a “rare pediatric disease” may qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product. The sponsor of a rare pediatric disease product receiving a priority review voucher may transfer (including by sale) the voucher to another sponsor. The voucher may be further transferred any number of times before the voucher is used, as long as the sponsor making the transfer has not yet submitted the application. The FDA may also revoke any priority review voucher if the rare pediatric disease product for which the voucher was awarded is not marketed in the U.S. within one year following the date of approval. In addition, the priority review voucher is only awarded to an NCE, thus if a compound is approved first for an indication that is not a rare pediatric disease the compound may not be eligible to receive the voucher. While we obtained and sold the priority review voucher issued upon approval of Zokinvy to reduce the risk of mortality in HGPS, and for treatment of PL with either heterozygous LMNA mutation with progerin-like protein accumulation, or homozygous or compound heterozygous ZMPSTE24 mutations, there can be no assurance that we will be successful in obtaining approval for avexitide for the treatment of HI, or that a priority review voucher will be issued at the time of any such approval.

Congress has only authorized the Rare Pediatric Disease Priority Review Voucher Program until September 30, 2024. However, if a product candidate receives RPD designation before September 30, 2024, it is eligible to receive a voucher if it is approved before September 30, 2026. Avexitide may not be approved by that date, or at all, and, therefore, we may not

be in a position to obtain a priority review voucher prior to expiration of the program, unless Congress further reauthorizes the program.

There is no assurance we will receive a rare pediatric disease priority review voucher. Also, although priority review vouchers may be sold or transferred to third parties, there is no guarantee that we will be able to realize any value if we were to sell a priority review voucher.

Although we have received Breakthrough Therapy designations, this may not lead to a faster development, regulatory review or approval process, and it does not increase the likelihood of receiving marketing approval in the United States.

We have received Breakthrough Therapy designation for lonafarnib and peginterferon lambda for the treatment of HDV, and for avexitide for the treatment of HI and PBH. A Breakthrough Therapy is defined as a therapy that is intended, alone or in combination with one or more other therapies, to treat a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the therapy may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development. For therapies that have been designated as breakthrough therapies, interaction and communication between the FDA and the sponsor of the trial can help to identify the most efficient path for clinical development while minimizing the number of patients placed in ineffective control regimens. Therapies designated as breakthrough therapies by the FDA may also be eligible for priority review and accelerated approval.

Designation as a breakthrough therapy is within the discretion of the FDA. Accordingly, even if we believe one of our product candidates meets the criteria for designation as a breakthrough therapy, the FDA may disagree and instead determine not to make such designation. The Breakthrough Therapy designations we have obtained may not result in faster development processes, reviews or approvals compared to therapies considered for approval under conventional FDA procedures and does not assure ultimate approval by the FDA. In addition, the FDA may later decide that any of our development programs no longer meet the conditions for qualification or decide that the time period for FDA review or approval will not be shortened. In addition, even if one or more of our product candidates qualify as breakthrough therapies, the FDA may later decide that the product no longer meets the conditions for qualification. Thus, even though we intend to seek Breakthrough Therapy designation for some or all of our future product candidates for the treatment of various conditions, there can be no assurance that we will receive any such Breakthrough Therapy designation.

We may submit an application for our product candidates under the Accelerated Approval Pathway. If we are unable to obtain approval or licensure of our product candidates through the Accelerated Approval Program in the United States, we may be required to conduct additional nonclinical and clinical studies and trials beyond those that we currently contemplate, which could increase the expense of obtaining, reduce the likelihood of obtaining and/or delay the timing of obtaining, necessary marketing approval. Even if we receive approval from the FDA through the Accelerated Approval Program, if any required confirmatory post-marketing trial does not verify clinical benefit, or if we do not comply with rigorous post-marketing requirements, the FDA may seek to withdraw the approval.

We may submit an NDA or BLA for our product candidates under the Accelerated Approval Pathway. If we are unable to obtain approval of our product candidates through the Accelerated Approval Program in the United States, we may be required to conduct additional nonclinical and clinical studies and trials beyond those that we currently contemplate, which could increase the expense of obtaining, reduce the likelihood of obtaining and/or delay the timing of obtaining, necessary marketing approval. Even if we receive approval from the FDA through the Accelerated Approval Program, if any required confirmatory post-marketing trial does not verify clinical benefit, or if we do not comply with rigorous post-marketing requirements, the FDA may seek to withdraw the approval.

We may seek approval under the Accelerated Approval pathway for our lonafarnib and peginterferon lambda products for the treatment of HDV. For any approval to market a product, we must provide the FDA and foreign regulatory agencies with clinical data that adequately demonstrate the safety, efficacy, purity and/or potency of the product for the indication applied for in the NDA, BLA, or other respective regulatory filings. The Accelerated Approval Program is one of several approaches used by the FDA to make prescription drugs and biologics more rapidly available for the treatment of serious or life-threatening diseases. Section 506(c) of the Federal Food, Drug and Cosmetic Act (FDCA) provides that the FDA may grant accelerated approval to “a product for a serious or life-threatening condition upon a determination that the product has an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit, or on a clinical endpoint that can be measured earlier than irreversible morbidity or mortality, that is reasonably likely to predict an effect on irreversible morbidity or mortality or other clinical benefit, taking into account the severity, rarity, or prevalence of the condition and the availability or lack of alternative treatments.” Approval through the Accelerated Approval Program is typically subject,

however, to the requirement that the applicant conduct additional post-marketing clinical trials to verify and describe the product’s clinical benefit. Typically, clinical benefit is verified when post-marketing clinical trials show that the product provides a clinically meaningful positive therapeutic effect, that is, an effect on how a patient feels, functions, or survives. The FDA may require that these studies be underway prior to Accelerated Approval pursuant to the Food and Drug Omnibus Reform Act of 2022 (FDORA). If such confirmatory post-marketing trial fails to confirm the product’s clinical profile or risks and benefits, the FDA may withdraw its approval of the product. The FDA has broad discretion with regard to approval through the Accelerated Approval Program, and even if we believe that the Accelerated Approval Program is appropriate for our product candidates, we cannot assure you that the FDA will ultimately agree. The FDA may also change its policies with respect over Accelerated Approval over time. For example, in March 2023, the FDA announced the availability of draft guidance on “Clinical Trial Considerations to Support Accelerated Approval of Oncology Therapeutics,” in which the agency outlined, and invited public comment on, its “preferred approach” of randomized controlled trials, including those that provide for longer term follow-up that could fulfill a post-marketing requirement to verify clinical benefit. In that draft guidance, the FDA acknowledged that historically, single-arm trial designs and response endpoints have most commonly been used in oncology, but noted that such trials have limitations. Furthermore, even if we do obtain approval through the Accelerated Approval Program, we may not experience a faster development process, review or approval compared to conventional FDA procedures.

Our lonafarnib-based regimens and peginterferon lambda products for the treatment of HDV are in Phase 3 studies with endpoints intended to support accelerated approval. The primary endpoint for the D-LIVR study, the Phase 3 study of lonafarnib-based regimens, is a composite of a > 2 log reduction in HDV RNA and ALT normalization at end of 48 weeks of treatment and is intended to support accelerated approval. The primary endpoint for the LIMT-2 study, the Phase 3 study of peginterferon lambda, is a durable virologic response, defined as HDV RNA below the limit of quantitation at 24 weeks post-treatment, and is intended to support accelerated approval. The study endpoints were previously achieved in Phase 2 studies and are consistent with FDA guidance on the development of treatments for HDV. While these proposed endpoints are designed to be consistent with FDA guidance, there is no assurance that approval will be granted on a timely basis, or at all. FDA may disagree that the design of, or results from, our studies support accelerated approval. Additionally, the FDA could require us to conduct further studies or trials prior to granting approval of any type, including by determining that approval through the Accelerated Approval Program is not appropriate and that our clinical trials may not be used to support approval through the conventional pathway. We might not be able to fulfill the FDA’s requirements in a timely manner, which would cause delays, or approval might not be granted because our submission is deemed incomplete by the FDA. There also can be no assurance that after subsequent FDA feedback we will continue to pursue approval through the Accelerated Approval Program. A failure to obtain approval through the Accelerated Approval Program could result in a longer time period to obtain approval of our product candidates, could increase the cost of their development, could delay our ability to commercialize our products and could significantly harm our financial position and competitive position in the marketplace.

Even if we receive approval for one or more of our product candidates through the Accelerated Approval Program, we will be subject to rigorous post-marketing requirements, possibly including the completion of one or more confirmatory post-marketing trials as the FDA may require, to verify the clinical benefit of the product, and submission to the FDA of all promotional materials prior to their dissemination. The FDA could seek to withdraw the approval for multiple reasons, including if we fail to conduct any required confirmatory post-marketing trial with due diligence, our confirmatory post-marketing trial does not confirm the predicted clinical benefit, other evidence shows that the product is not safe or effective under the conditions of use, or we disseminate promotional materials that are found by the FDA to be false and misleading.

Moreover, Congress has recently enacted potential changes to the Accelerated Approval Program that could impact our ability to obtain an Accelerated Approval, or increase the burdens associated with post marketing requirements in the event we do obtain an Accelerated Approval. In particular, FDA must specify certain conditions for required post approval studies for products that receive Accelerated Approval, which may include enrollment targets and milestones, including the target date for study completion, by the time the product is approved. FDA may also require post approval studies to be underway at the time of Accelerated Approval or within a specified time period following Accelerated Approval for such products.

Any delay in obtaining, or inability to obtain, approval through the Accelerated Approval Program, or any issues in maintaining approval granted under the Accelerated Approval Program, would delay or prevent commercialization of our products, and would materially adversely affect our business, financial condition, results of operations, cash flows and prospects

Product development involves a lengthy and expensive process with an uncertain outcome, and results of earlier studies may not be predictive of future study results.*

Clinical testing is expensive and generally takes many years to complete, and the outcome is inherently uncertain. Failure can occur at any time during the clinical study process. The results of preclinical studies and early clinical studies of our product candidates may not be predictive of the results of larger, later-stage controlled clinical studies. Product candidates that have shown promising results in early-stage clinical studies may still suffer significant setbacks in subsequent clinical studies. Our clinical studies to date have been conducted on a small number of patients in limited numbers of clinical sites and in academic settings or for other indications. We will have to conduct larger, well-controlled studies in our proposed indications to verify the results obtained to date and to support any regulatory submissions for further clinical development. A number of companies in the biopharmaceutical industry have suffered significant setbacks in advanced clinical studies due to lack of efficacy or adverse safety profiles despite promising results in earlier, smaller clinical studies. Moreover, clinical data are often susceptible to varying interpretations and analyses. We do not know whether any Phase 2, Phase 3, or other clinical studies we have conducted or may conduct will demonstrate consistent or adequate safety, efficacy, ~~and safety~~purity and/or potency with respect to the proposed indication for use sufficient to obtain regulatory approval to receive regulatory approval or market our ~~drug~~product candidates. For example, in 2018 we ~~identified significant GCP violations~~announced negative results from two of our Phase 2 clinical trials of ubenimex in ~~the LOWR HDV – 1~~two different indications, and ~~LOWR HDV – 2 studies. We alerted the FDA to the discovery~~as a result we have terminated further development of GCP violations. These findings may call into question the validity of the data set and may require repeat clinical studies to confirm the observations in question. Further studies may be needed to confirm the rigor, robustness and validity of any problematic safety and efficacy data identified.

ubenimex.

We may find it difficult to enroll patients in our clinical studies given the limited number of patients who have the diseases for which our product candidates are being studied. Difficulty in enrolling patients could delay or prevent clinical studies of our product candidates.

Identifying and qualifying patients to participate in clinical studies of our product candidates is essential to our success. The timing of our clinical studies depends in part on the rate at which we can recruit patients to participate in clinical trials of our product candidates, and we may experience delays in our clinical studies if we encounter difficulties in enrollment.

The eligibility criteria of our planned clinical studies may further limit the available eligible study participants as we expect to require that patients have specific characteristics that we can measure or meet the criteria to assure their conditions are appropriate for inclusion in our clinical studies. We may not be able to identify, recruit, and enroll a sufficient number of patients to complete our clinical studies in a timely manner because of the perceived risks and benefits of the product candidate under study, the availability and efficacy of competing therapies and clinical studies, and the willingness of physicians to participate in our planned clinical studies. For example, early in the COVID-19 pandemic, certain clinical study sites that were scheduled to open were delayed in activating and other sites suspended randomization of subjects for a period of time. Future pandemic restrictions could result in delays of our clinical trials, including LIMT-2. If patients are unwilling to participate in our clinical studies for any reason, the timeline for conducting studies and obtaining regulatory approval of our product candidates may be delayed.

If we experience delays in the completion of, or termination of, any clinical study of our product candidates, the commercial prospects of our product candidates could be harmed, and our ability to generate product revenue from any of these product candidates could be delayed or prevented. In addition, any delays in completing our clinical studies would likely increase our overall costs, impair product candidate development and jeopardize our ability to obtain regulatory approval relative to our current plans. Any of these occurrences may harm our business, financial condition, and prospects significantly.

Clinical studies are costly, time consuming and inherently risky, and we may fail to demonstrate safety, ~~and~~ efficacy, purity and/or potency to the satisfaction of applicable regulatory authorities.

Clinical development is expensive, time consuming and involves significant risk. We cannot guarantee that any clinical studies will be conducted as planned or completed on schedule, if at all. A failure of one or more clinical studies can occur at any stage of development. Events that may prevent successful or timely completion of clinical development include but are not limited to:

•inability to generate satisfactory preclinical, toxicology, or other in vivo or in vitro data or diagnostics to support the initiation or continuation of clinical studies necessary for product approval;

•delays in reaching agreement on acceptable terms with ~~CROs~~contract research organizations (CROs) and clinical study sites, the terms of which can be subject to extensive negotiation and may vary significantly among different CROs and clinical study sites;

•delays in obtaining required Institutional Review Board ~~or IRB,~~(IRB) approval at each clinical study site;

•failure to permit the conduct of a study by regulatory authorities, after review of an investigational new drug ~~or IND,~~(IND) or equivalent foreign application or amendment;

•delays in recruiting qualified patients, or patients dropping out of, in our clinical ~~studies;~~

studies, including as a result of the evolving COVID-19 global pandemic;

•feasibility of continuous trial execution in countries impacted by war, geopolitical conflict and other humanitarian crises;

•failure by clinical sites or our CROs or other third parties to adhere to clinical study requirements or report complete findings, such as for the LOWR HDV – 1 and LOWR HDV – 2 studies, where we have identified significant GCP violations that may impact the data set and information that we plan to submit to the FDA and may require repeat studies to confirm the rigor, robustness and validity of safety and efficacy signals seen in these studies;

findings;

•failure to perform the clinical studies in accordance with the FDA’s ~~GCP~~good clinical practice (GCP) requirements, or applicable foreign regulatory guidelines;

~~patients dropping out of our clinical studies;~~

•occurrence of adverse events associated with our product candidates;

•changes in regulatory requirements and guidance that require amending or submitting new clinical protocols;

•the cost of clinical studies of our product candidates;

•negative or inconclusive results from our clinical trials which may result in our deciding, or regulators requiring us, to conduct additional clinical studies or abandon development programs in other ongoing or planned indications for a product candidate; and

•delays in reaching agreement on acceptable terms with third-party manufacturers and the time for manufacture of sufficient quantities of our product candidates for use in clinical studies.

Any inability to successfully complete clinical development and obtain regulatory approval could result in additional costs to us or impair our ability to generate revenue. Clinical study delays could also shorten any periods during which our products have patent protection and may allow competitors to develop and bring products to market before we do, which could impair our ability to obtain ~~orphan drug~~or maintain Orphan Drug designation exclusivity and to successfully commercialize our product candidates and may harm our business and results of operations.

Given we are not able to receive an Emergency Use Authorization (EUA) from FDA in the U.S., we may not be able to receive a similar temporary use authorization from foreign regulatory agencies. If we do receive an EUA or similar temporary use authorization, absent a full marketing authorization for that indication, our ability to sell our products would be revoked when the COVID-19 public health emergency terminates.

On October 5, 2022, we announced that, following feedback from FDA, we will not submit an EUA application for peginterferon lambda for the treatment of patients with mild-to-moderate COVID-19. If we decide to submit a future application for an EUA, we cannot predict whether FDA will grant an EUA. If we do not receive an EUA from FDA, we will not be able to commercialize future products and may be required to conduct additional clinical trials for an EUA. Obtaining such an authorization is dependent upon a number of factors, which are not under our control. Even if an EUA is received, we also cannot predict how long, if ever, an EUA would remain in place.

We do intend to pursue similar temporary use authorizations in non-U.S. jurisdictions.

Various regulatory pathways are available in jurisdictions outside the United States to make products available for emergency use. For example, regulatory authorities in certain EU Member States may temporarily authorize the distribution of an unauthorized drug in response to the suspected or confirmed spread of pathogenic agents such as the virus which is causing COVID-19. Obtaining such a temporary authorization is dependent upon a number of factors, which are not under our control. If such authorizations would be granted, they would only apply for a limited period of time. We might thus no longer be authorized to distribute our product under these authorizations if that time limit expired or the pandemic terminates.

The regulatory authorities in the EU or in other jurisdictions outside the U.S. may grant a conditional marketing authorization for medicinal products intended for the treatment of seriously debilitating or life-threatening diseases prior to the submission of comprehensive clinical data if that treatment is of major therapeutic advantage to the patients concerned or no other authorized treatment is available. In emergency situations, such a conditional marketing authorization may also be granted for these medicinal products where comprehensive preclinical or pharmaceutical data have not been supplied.

These conditional marketing authorizations are subject to specific conditions (e.g., completing on-going studies or conducting new studies) which must be fulfilled within a timeline specified in the marketing authorization. These marketing authorizations are valid for a short period of time (e.g., one year) which can usually be renewed. If we would apply for such a conditional marketing authorization the regulatory authority concerned may reject our application because it considers that the benefit-risk balance of our product is not favorable or it judges it unlikely that we would be able to provide comprehensive data. If we would obtain such a conditional marketing authorization we may not be able to complete (timely) the studies which the regulatory authority imposed as a condition for the marketing authorization or the data collected in the course of these studies may indicate that our product does not have a favorable benefit-risk profile. As a result, we may not be able to continue distributing our product because the conditional marketing authorization has been revoked or not renewed, or the regulatory authority refused finally to grant a regular marketing authorization.

Regulatory authorities in the EU or other jurisdictions outside the EU may make a product available for compassionate reasons to a group of patients with a chronically or seriously debilitating disease or whose disease is considered to be life-threatening, and who cannot be treated satisfactorily with an authorized medicinal product provided that the product is subject of an application for marketing authorization or is undergoing clinical trials. If we would request a regulatory authority to make our product available to patient under these conditions the regulatory authority may reject our request if, for example, it considers that the patients concerned can be treated satisfactorily with other products that are already authorized.

Programs which make products available under the conditions mentioned above are usually authorized for a limited period of time (e.g., one year). Regulatory authorities may not renew expiring authorizations for these programs if we terminate prematurely a clinical trial with our product or decide not to submit or to withdraw an application for marketing authorization in the jurisdiction concerned. That may, for example, happen because pharmacovigilance data or other data collected during our clinical trials indicate that our product does not have the appropriate benefit-risk balance.

Our product candidates may cause undesirable side effects or have other properties that could delay or prevent their regulatory approval, limit the commercial profile of an approved label, or result in significant negative consequences following marketing approval, if any.*

Undesirable side effects, or unforeseen side effects due to incomplete or inadequate safety data collection from LOWR HDV – 1 and LOWR HDV – 2 clinical studies, caused by our product candidates could cause us or regulatory authorities to interrupt, delay, or terminate or conduct additional or larger clinical studies or even if approved, result in a restrictive label or delay regulatory approval by the FDA or comparable foreign authorities.

~~For example, our~~Our lonafarnib product candidate has been studied in thousands of oncology patients, and the most common non-hematologic adverse events of any grade were gastrointestinal system disorders (nausea, anorexia, diarrhea and vomiting), weight loss, fatigue and rash. Use of Zokinvy to reduce the risk of mortality in HGPS, and for treatment of processing-deficient progeroid laminopathies with either heterozygous LMNA mutation with progerin-like protein accumulation, or homozygous or compound heterozygous ZMPSTE24 mutations, has been reported to cause ECG abnormalities, but these ECG abnormalities have not resulted in a risk of mortality for these patients. There is no guarantee that additional or more severe side effects or other properties will not be identified through ongoing clinical studies by other uses of lonafarnib for other indications or our own clinical trials. ~~Additionally, while we have a license~~Our peginterferon lambda product candidate is well-characterized and has been studied in thousands of HBV and HCV patients, and the most common adverse events seen are moderate headache, pyrexia, fatigue, and myalgia. ALT flares that were seen result from vigorous antiviral immunological response to ~~another farnesyltransferase inhibitor compound, tipifarnib, from Janssen Pharmaceutica, N.V.,~~treatment, not due to direct hepatotoxicity. There is no guarantee that additional or ~~Janssen, Janssen has granted rights to tipifarnib to Kura Oncology, Inc., or Kura, in oncology and negative results or undesirable~~more severe side effects ~~from Kura’s~~will not be identified through ongoing clinical ~~trials~~studies for ~~a compound with a similar mechanism~~other uses of ~~action may negatively impact the perception of lonafarnib for anti-viral indications. Merck may also grant rights to other anti-viral or potentially other indications to other third parties.~~peginterferon lambda. Undesirable side effects, other properties, and negative results for other indications may negatively impact the development and potential for approval of our product candidates for our proposed indications.

For example, the ECG abnormalities seen with lonafarnib in HGPS and PL patients has the potential to impact the labeling for lonafarnib-based regimens for HDV. Our avexitide product candidate has been studied in 39 HI patients and over 70 PBH patients, and the most common adverse events are injection site bruising/reaction, nausea, and headache. There is no guarantee that additional or more severe side effects will not be identified through ongoing clinical studies for other uses of avexitide in clinical trials.

Additionally, even if one or more of our current product candidates receives marketing approval, and we or others later may identify undesirable side effects caused by such products, potentially significant negative consequences could result, including but not limited to:

•regulatory authorities may withdraw approvals of such products;

•regulatory authorities may require additional warnings on the label;

•we may be required to create a Risk Evaluation and Mitigation Strategy ~~or REMS,~~(REMS) plan, which could include a medication guide outlining the risks of such side effects for distribution to patients, a communication plan for healthcare providers, and/or other elements to assure safe use;

•we could be sued and held liable for harm caused to patients; and

•our reputation may suffer.

Any of these events could prevent us from achieving or maintaining market acceptance of a product candidate, even if approved, and could significantly harm our business, results of operations, and prospects.

~~Even~~

We are subject to ongoing regulatory requirements related to the U.S. and EU approvals of Zokinvy, and if we obtain additional regulatory ~~approval~~approvals for a product candidate, we will ~~remain~~be subject to additional ongoing regulatory requirements.

If our product candidates are approved, they will be subject to ongoing regulatory requirements with respect to manufacturing, labeling, packaging, storage, advertising, promotion, sampling, record-keeping, conduct of post-marketing studies, and submission of safety, efficacy and other post-approval information, including both federal and state requirements in the United States and requirements of comparable foreign regulatory authorities.

Manufacturers and manufacturers’ facilities are required to continuously comply with FDA and comparable foreign regulatory authority requirements, including ensuring that quality control and manufacturing procedures conform to current Good Manufacturing Practices ~~or cGMP,~~(cGMP) regulations and corresponding foreign regulatory manufacturing requirements. As such, we and our contract manufacturers will be subject to continual review and inspections to assess compliance with cGMP and adherence to commitments made in any NDA, ~~or marketing authorization application,~~BLA, or MAA.

Any regulatory approvals that we receive for our product candidates may be subject to limitations on the approved indicated uses for which the product candidate may be marketed or to the conditions of approval, or contain requirements for potentially costly post-marketing testing, including Phase IV4 clinical trials, and surveillance to monitor the safety, ~~and~~ efficacy, purity and/or potency of the product candidate. We will be required to report certain adverse reactions and production problems, if any, to the FDA and comparable foreign regulatory authorities.

Any new legislation addressing ~~drug~~product safety issues could result in delays in product development or commercialization, or increased costs to assure compliance. If our original marketing approval for a product candidate was obtained through an accelerated approval pathway, we could be required to conduct a successful post-marketing clinical study ~~in order~~ to confirm the clinical benefit for our products. An unsuccessful post-marketing study or failure to complete such a study could result in the withdrawal of marketing approval.

If a regulatory agency discovers previously unknown problems with a product, such as adverse events of unanticipated severity or frequency, or problems with the facility where the product is manufactured, or disagrees with the promotion, marketing, or labeling of a product, the regulatory agency may impose restrictions on that product or us, including requiring withdrawal of the product from the market. If we fail to comply with applicable regulatory requirements, a regulatory agency or enforcement authority may, among other things:

•issue warning letters;

•impose civil or criminal penalties;

•suspend or withdraw regulatory approval;

•suspend any of our ongoing clinical studies;

•refuse to approve pending applications or supplements to approved applications submitted by us;

•impose restrictions on our operations, including closing our contract manufacturers’ facilities; or

•require a product recall.

In addition, prescription drugs and biologics may be promoted only for the approved indications in accordance with the approved label. The FDA and other agencies actively enforce the laws and regulations prohibiting the promotion of off-label uses, and a company that is found to have improperly promoted off-label may be subject to significant liability. However, physicians may, in their independent medical judgment, prescribe legally available products for off-label uses. The FDA does not regulate the behavior of physicians in their choice of treatments, but the FDA does restrict manufacturer’s communications on the subject of off-label use of their products.

Any government investigation of alleged violations of law would be expected to require us to expend significant time and resources in response and could generate adverse publicity. Any failure to comply with ongoing regulatory requirements may significantly and adversely affect our ability to develop and commercialize our products and the value of us and our operating results would be adversely affected.

We rely on third parties to conduct our clinical studies, manufacture our product candidates and perform other services. If these third parties do not successfully perform and comply with regulatory requirements, we may not be able to successfully complete clinical development, obtain regulatory approval or commercialize our product candidates and our business could be substantially harmed.*

We have relied upon and plan to continue to rely upon investigators and third-party CROs to conduct, monitor and manage our ongoing clinical programs. We rely on these parties for execution of clinical studies and manage and control only certain aspects of their activities. We remain responsible for ensuring that each of our studies is conducted in accordance with the applicable protocol, legal, regulatory, and scientific standards, and our reliance on the CROs does not relieve us of our regulatory responsibilities. We, our investigators, and our CROs and other vendors are required to comply with all applicable laws, regulations and guidelines, including those required by the FDA and comparable foreign regulatory authorities for all of our product candidates in clinical development. If we or any of our investigators, CROs or vendors fail to comply with applicable laws, regulations and guidelines, the results generated in our clinical studies may be deemed unreliable and the FDA or comparable foreign regulatory authorities may require us to perform additional studies before approving our marketing applications. For example, we identified significant GCP violations in the LOWR HDV – 1 and LOWR HDV – 2 studies. We alerted the FDA to the discovery or GCP violations. These findings may call into question the validity of the data set and may require repeat clinical studies to confirm the observations in question. Further studies may be needed to confirm the rigor, robustness and validity of any problematic safety and efficacy data identified. We cannot assure you that our CROs and other vendors will meet these requirements, or that upon inspection by any regulatory authority, such regulatory authority will determine that efforts, including any of our clinical studies, comply with applicable requirements. Our failure to comply with these laws, regulations and guidelines may require us to repeat clinical studies or conduct larger additional studies, which would be costly and delay the regulatory approval process.

If any of our relationships with investigators or third-party CROs terminate, we may not be able to enter into arrangements with alternative CROs in a timely manner or do so on commercially reasonable terms. In addition, our CROs may not prioritize our clinical studies relative to those of other customers and any turnover in personnel or delays in the allocation of CRO employees by the CRO may negatively affect our clinical studies. If investigators or CROs do not successfully carry out their contractual duties or obligations or meet expected deadlines, our clinical studies may be delayed or terminated, and we may not be able to meet our current plans with respect to our product candidates. CROs may also involve higher costs than anticipated, which could negatively affect our financial condition and operations.

In addition, we do not currently have, nor do we plan to establish, the capability to manufacture product candidates for use in the conduct of our clinical studies or in support of our commercialization of potential products, and we lack the resources and the capability to manufacture any of our product candidates on a clinical or commercial scale without the use of third-party manufacturers. We plan to rely on third-party manufacturers and their responsibilities will include purchasing from third-party suppliers the materials necessary to produce our product candidates for our clinical studies and regulatory approval. There are expected to be a limited number of suppliers for the active ingredients and other materials that we expect to use to manufacture our product candidates, and we may not be able to identify alternative suppliers to prevent a possible disruption of the manufacture of our product candidates for our clinical studies, and, if approved, ultimately for commercial sale. Although we generally do not expect to begin a clinical study unless we believe we have a sufficient supply of a product candidate to complete the study, any significant delay or discontinuity in the supply of a product candidate, or the active ingredient or other material components in the manufacture of the product candidate, could delay completion of our clinical studies and potential timing for regulatory approval of our product candidates, which would harm our business and results of operations.

For example, in connection with Phase 3 startup activities for Avexitide, we observed low levels of product-related impurities in the finished drug product. Although not unusual for this class of compounds, we are working with our CMOs to control and qualify these materials and plan to initiate dosing in Phase 3 when an adequate supply of materials with a sufficient shelf-life has been released.

With respect to our lonafarnib program, we procured an inventory of product from Merck to supply our initial clinical study needs. In 2016, we transferred the manufacturing of drug substance and drug product to our third-party contractors. The material used for lonafarnib HDV pivotal trials, ongoing progeria clinical studies and expanded access program, and commercial Zokinvy supply are sourced from Eiger-controlled CMOs. These same vendors are currently under development for commercial qualification. Materials used for our avexitide clinical trials are also sourced from CMOs. Our vendors have successfully made GMP batches for our ~~future~~ clinical ~~studies. With respect~~studies, however, our trials could be delayed if quality, stability, or other issues occur in relation to ~~our lambda program, as part~~the manufacture of the license agreement, we obtained a substantial inventory of product from BMS sufficient to initiate our clinical trials. During 2017 we transferred the manufacturing technology to our third-party vendors. With respect to our ubenimex programs we have relied on Nippon Kayakuany unique batch. If these CMOs are not able to provide us with sufficient quantities of product for our clinical trials or in support of our commercialization of potential products on a timely basis, or at all, whether due to ~~conduct~~production shortages or other supply interruptions resulting from any delay, our

clinical trials ~~in 2016 and 2017 and have now completed~~or regulatory approval may be delayed, or could impair our ability to generate revenue from the ~~process~~sale of ~~transferring the manufacturing of ubenimex to our third-party vendors in the United States and~~ such ~~drug~~ product ~~will be adequate to complete ongoing and future clinical studies.~~

candidate.

We rely and expect to continue to rely on third parties to manufacture our clinical product supplies, and if those third parties fail to obtain approval of government regulators, fail to provide us with sufficient quantities of ~~drug~~ product, or fail to do so at acceptable quality levels or prices our product candidates could be stopped, delayed, or made less profitable.

We do not currently have nor do we plan to acquire the infrastructure or capability internally to manufacture our clinical supplies for use in the conduct of our clinical trials, and we lack the resources and the capability to manufacture any of our product candidates on a clinical or commercial scale. We currently rely on outside vendors to source raw materials and manufacture our clinical supplies of our product candidates and plan to continue relying on third parties to manufacture our product candidates on a commercial scale, if approved.

The facilities used by our contract manufacturers to manufacture our product candidates ~~must~~will be ~~approved~~subject to pre-approval inspection by the FDA ~~pursuant to inspections~~ that will be conducted after we submit our marketing applications to the ~~FDA.~~FDA or comparable foreign regulatory authorities. We do not control the manufacturing process of, and are completely dependent on, our contract manufacturing partners for compliance with the regulatory requirements, known as cGMPs, for manufacture of our product candidates. If our contract manufacturers cannot successfully manufacture material that conforms to our specifications and the strict regulatory requirements of the FDA, EMA or others, ~~they will~~our future applications may not be ~~able to secure and/or maintain~~approved by regulatory ~~approval for their manufacturing facilities. In addition, we~~authorities, which would significantly delay our commercialization plans and increase our costs. We have no control over the ability of our contract manufacturers to maintain adequate quality control, quality assurance and qualified ~~personnel.~~personnel, and in the past we have experienced quality control issues with product manufactured by our contract manufacturers. If the FDA or a comparable foreign regulatory authority does not approve these facilities for the manufacture of our product candidates or if it withdraws any such approval in the future, we may need to find alternative manufacturing facilities, which would significantly impact our ability to develop, obtain regulatory approval for or market our product candidates, if approved.

We do not yet have sufficient information to reliably estimate the cost of the commercial manufacturing of our product candidates, and the actual cost to manufacture our product candidates could materially and adversely affect the commercial viability of our product candidates. As a result, we may ~~never~~not be able to develop aadditional commercially viable ~~product.~~

products.

In addition, our reliance on third-party manufacturers exposes us to the following additional risks:

•We may be unable to identify manufacturers on acceptable terms or at all;

•Our third-party manufacturers might be unable to timely formulate and manufacture our product or produce the quantity and quality required to meet our clinical and commercial needs, if any;

•Contract manufacturers may not be able to execute our manufacturing procedures appropriately;

•Our future contract manufacturers may not perform as agreed or may not remain in the contract manufacturing business for the time required to supply our clinical trials or to successfully produce, store and distribute our products;

•Manufacturers are subject to ongoing periodic unannounced inspection by the FDA and corresponding state agencies to ensure strict compliance with cGMP and other government regulations and corresponding foreign standards. We do not have control over third-party manufacturers’ compliance with these regulations and standards;

•We may not own, or may have to share, the intellectual property rights to any improvements made by our third-party manufacturers in the manufacturing process for our product candidates; and

•

~~We may not own, or may have to share, the intellectual property rights to any improvements made by our third-party manufacturers in the manufacturing process for our product candidates; and~~

•Our third-party manufacturers could breach or terminate their agreement with us.

Each of these risks could delay our clinical trials, the approval ifof any of our product candidates by the FDA or comparable foreign regulatory authorities or the commercialization of our product candidates or result in higher costs or deprive us of potential product revenue. In addition, we rely on third parties to perform release testing on our product candidates prior to delivery to patients. If these tests are not conducted appropriately ~~conducted~~ and test data ~~are~~is not reliable, patients could be put at risk of serious harm and could result in product liability suits.

The ~~manufacture~~manufacturing of medical products is complex and requires significant expertise and capital investment, including the development of advanced manufacturing techniques and process controls. Manufacturers of biologic products often

encounter difficulties in production, particularly in scaling up and validating initial production and absence of contamination. These problems include difficulties with production costs and yields, quality control, including stability of the product, quality assurance testing, operator error, shortages of qualified personnel, as well as compliance with strictly enforced federal, state and foreign regulations. Furthermore, if contaminants are discovered in our supply of our product candidates or in the manufacturing facilities, such manufacturing facilities may need to be closed for an extended period of time to investigate and remedy the contamination. We cannot assure you that any stability or other issues relating to the manufacture of our product candidates will not occur in the future. Additionally, our manufacturers may experience manufacturing difficulties due to resource constraints or as a result of labor disputes or unstable political environments. If our manufacturers were to encounter any of these difficulties, or otherwise fail to comply with their contractual obligations, our ability to provide our product candidates to patients in clinical trials would be jeopardized. Any delay or interruption in the supply of clinical trial supplies could delay the completion of clinical trials, increase the costs associated with maintaining clinical trial programs and, depending upon the period of delay, require us to commence new clinical trials at additional expense or terminate clinical trials completely.

If the market opportunities for our product candidates are smaller than we believe they are, we may not meet our revenue expectations and, even assuming approval of a product candidate, our business may suffer. Because the patient populations in the market for our product candidates may be small, we must be able to successfully identify patients and acquire a significant market share to achieve profitability and growth.

We focus our product development principally on treatments for ~~orphan~~rare and ultra-rare diseases. Given the small number of patients who have the diseases that we are targeting, our eligible patient population and pricing estimates may differ significantly from the actual market addressable by our product candidate. Our projections of both the number of people who have these diseases, as well as the subset of people with these diseases who have the potential to benefit from treatment with our product candidates, are based on our beliefs and estimates. These estimates have been derived from a variety of sources, including the scientific literature, patient foundations, or market research, and may prove to be incorrect. Further, new studies may change the estimated incidence or prevalence of these diseases. The number of patients may turn out to be lower than expected. For example, ~~for lonafarnib and lambda, HDV is associated with hepatitis B virus infection, which is a pre-requisite for the replication of HDV. Although~~although we believe that ~~the~~our lonafarnib-based regimens and peginterferon lambda data are supportive of ~~the increased severity of hepatitis in the presence of hepatitis D and hepatitis B virus co-infection compared to hepatitis B alone,~~antiviral activity against HDV, there can be no assurance that our clinical trials will successfully address this condition. Likewise, the potentially addressable patient population for each of our product candidates may be limited or may not be amenable to treatment with our product candidates, and new patients may become increasingly difficult to identify or gain access to, which would adversely affect our results of operations and our business.

Moreover, we expect that the sales of Zokinvy to patients with progeria will have limited profits given the ultra-rare nature of these diseases.

We face intense competition and rapid technological change and the possibility that our competitors may develop therapies that are similar, more advanced, or more effective than ours, which may adversely affect our financial condition and our ability to successfully commercialize our product candidates.

The biotechnology and pharmaceutical industries are intensely competitive and subject to rapid and significant technological change. We are currently aware of various existing therapies that may compete with our product candidates. For example, we have competitors both in the United States and internationally, including multinational pharmaceutical companies, specialty pharmaceutical companies and biotechnology companies. Some of the pharmaceutical and biotechnology companies we expect to compete with include Gilead ~~Merck, Roche, Actelion/Johnson~~Sciences, Vir Biotechnology, Janssen Pharmaceuticals, Replicor, Zealand Pharmaceuticals, Xeris Pharmaceuticals, Rezolute, Hanmi Pharmaceutical, and ~~Johnson, Replicor, Arrowhead, Novartis, Xoma, Reata and Arena~~Crinetics Pharmaceuticals as well as other smaller companies or biotechnology startups and large multinational pharmaceutical companies. Many of our competitors have substantially greater financial, technical, and other resources, such as larger research and development staff and experienced marketing and manufacturing organizations. Additional mergers and acquisitions in the biotechnology and pharmaceutical industries may result in even more resources being concentrated in our competitors. As a result, these companies may obtain regulatory approval more rapidly than we are able to and may be more effective in selling and marketing their products as well. Smaller or early-stage companies may also prove to be significant competitors, particularly through collaborative arrangements with large, established companies. Competition may increase further as a result of advances in the commercial applicability of technologies and greater availability of

capital for investment in these industries. Our competitors may succeed in developing, acquiring, or licensing on an exclusive basis, products that are more effective or less costly than any product candidate that we may develop, or achieve earlier patent protection, regulatory approval, product commercialization, and market penetration than we do. Additionally, technologies developed by our competitors may render our potential product candidates uneconomical or obsolete, and we may not be successful in marketing our product candidates against competitors.

~~We currently~~

Although we commercially launched Zokinvy following its FDA approval, we have limited marketing and sales experience. If we are unable to establish sales and marketing capabilities or enter into agreements with third parties to market and sell our product candidates, we may be unable to generate any revenue.

Although certain of our employees may have marketed, launched and sold other pharmaceutical products in the past while employed at other companies, we have nolimited recent experience selling and marketing our product candidates and we currently have noa small sales and marketing ~~or sales~~ organization. To successfully commercialize ~~any~~Zokinvy and additional products that may result from our development programs, we will need to invest in and ~~develop~~expand these capabilities, either on our own or with others, which would be expensive, difficult and time consuming. Any failure or delay in the timely development of our internal commercialization capabilities could adversely impact the potential for success of our products.

Further, given our ~~lack of prior~~recent, limited experience in marketing and selling biopharmaceutical products, we may rely on future collaborators to commercialize our products. If collaborators do not commit sufficient resources to commercialize our future products and we are unable to develop the necessary marketing and sales capabilities on our own, we will be unable to generate sufficient product revenue to sustain or grow our business. We may be competing with companies that currently have extensive and well-funded marketing and sales operations, in particular in the markets our product candidates are intended to address. Without appropriate capabilities, whether directly or through third-party collaborators, we may be unable to compete successfully against these more established companies.

In addition, we have established an expanded access program in order to make Zokinvy available for patients with progeria, which requires additional resources and costs to support.

The commercial success of any of our current or future product ~~candidate~~candidates will depend upon the degree of market acceptance by physicians, patients, third-party payors, and others in the medical community.

Even with the approvals from the FDA and comparable foreign regulatory authorities, the commercial success of our products will depend in part on the health care providers, patients, and third-party payors accepting our product candidates as medically useful, cost-effective, and safe. Any product that we bring to the market may not gain market acceptance by physicians, patients, third-party payors and other health care providers. The degree of market acceptance of any of our products will depend on a number of factors, including without limitation:

•the timing of our receipt of any marketing and commercialization licensures;

•the terms of any licensures and the countries in which licensures are obtained;

•the safety, efficacy, purity and/or potency of the product as demonstrated in clinical studies and potential advantages over competing treatments;

•the prevalence and severity of the disease and any side effects;

•the clinical indications for which approval is granted, including any limitations or warnings contained in a product’s approved labeling;

•the convenience and ease of administration;

•the cost of treatment;

•the willingness of the patients and physicians to accept these ~~therapies;~~

therapies or any new methods of administration;

•the marketing, sales and distribution support for the product;

•the publicity concerning our products or competing products and treatments;

•the success of our physician education programs;

•availability of alternative effective treatments for the disease indications our product candidates are intended to treat and

the relative risks, benefits and costs of those treatments; and

•the pricing and availability of third-party insurance coverage and reimbursement.

Even if a product displays a favorable safety, efficacy, ~~and safety~~purity and/or potency profile upon approval, market acceptance of the product remains uncertain. Efforts to educate the medical community and third-party payors on the benefits of the products may require significant investment and resources and may never be successful. If our products fail to achieve an adequate level of acceptance by physicians, patients, third-party payors, and other health care providers, we will not be able to generate sufficient revenue to become or remain profitable.

Failure to obtain or maintain adequate reimbursement or insurance coverage for new or current products could limit our ability to market those products and decrease our ability to generate revenue.

We cannot be sure that reimbursement will be available for our products and, if coverage and reimbursement are available, what the level of reimbursement will be. Coverage may also be more limited than the indications for which the product is approved by the FDA or comparable foreign regulatory authorities. Reimbursement may impact the demand for, or the price of, our products.

The pricing, coverage and reimbursement of our products must be sufficient to support our commercial efforts and other development programs and the availability and adequacy of coverage and reimbursement by governmental and private payors are essential for most patients to be able to afford expensive treatments, particularly in ~~orphan drug~~Orphan Drug designated indications where the eligible patient population is small. Sales of our ~~product candidates~~products will depend substantially, both domestically and abroad, on the extent to which the costs of our product candidates will be paid for or reimbursed by health maintenance, managed care, pharmacy benefit and similar healthcare management organizations, or government authorities, private health insurers, and other third-party payors. If coverage and reimbursement are not available, or are available only in limited amounts, we may have to subsidize or provide products for free or we may not be able to successfully commercialize our products.

For example, Zokinvy for patients with HGPS and processing-deficient progeroid laminopathies provided under an expanded access program may not result in reimbursement.

In addition, there is significant uncertainty related to the insurance coverage and reimbursement for newly approved products. In the United States, the principal decisions about coverage and reimbursement for new drugs are typically made by the Centers for Medicare & Medicaid Services ~~or CMS,~~(CMS), an agency within the U.S. Department of Health and Human Services (HHS), as CMS decides whether and to what extent a new drug will be covered and reimbursed under Medicare. Private payors ~~tend to~~often follow the coverage reimbursement policies established by CMS to a substantial degree. It is difficult to predict what CMS and other payors will decide with respect to ~~reimbursement~~coverage for products such as ours and what reimbursement ~~codes~~ our products may receive.

The process for determining whether a third-party payor will provide coverage for a drug product typically is separate from the process for setting the price of a drug product or for establishing the reimbursement rate that the payor will pay for the drug product once coverage is approved. Third-party payors may limit coverage to specific drug products on an approved list, also known as a formulary, which might not include all of the FDA-approved drugs for a particular indication. A decision by a third-party payor not to cover Zokinvy or any of our product candidates once approved could reduce utilization of such products and have a material adverse effect on our sales, results of operations and financial condition. Moreover, a third-party payor’s decision to provide coverage for a drug product does not imply that adequate reimbursement will be available. Adequate third-party payor reimbursement may not be available to enable us to maintain price levels sufficient to realize an appropriate return on our investment in product development. Additionally, coverage and reimbursement for drug products can differ significantly from payor to payor. One third-party payor’s decision to cover a particular medical product or service does not ensure that other payors will also provide coverage for the medical product or service or will provide coverage at an adequate reimbursement rate. As a result, the coverage determination process will require us to provide scientific and clinical support for the use of our products to each payor separately and will be a time-consuming process.

Third-party payors are increasingly challenging the prices charged for medical products and services, examining the medical necessity and reviewing the cost-effectiveness of drug products and medical services, in addition to questioning safety and efficacy. If third-party payors do not consider our products to be cost-effective compared to other available therapies, they may not cover our products after FDA approval or, if they do, the level of payment may not be sufficient to allow us to sell our products at a profit. Additionally, coverage policies and third‑party reimbursement rates may change at any time. Therefore, even if favorable coverage and reimbursement status is attained for one or more of our products, less favorable coverage policies and reimbursement rates may be implemented in the future. If we are unable to obtain and maintain sufficient third‑party coverage and adequate reimbursement, the commercial success of our products may be greatly hindered and our financial condition and results of operations may be materially and adversely affected.

Outside the United States, international operations are generally subject to extensive governmental price controls and other price-restrictive regulations, and we believe the increasing emphasis on cost-containment initiatives in Europe, Canada, and other countries has and will continue to put pressure on the pricing and usage of products. In many countries, the prices of products are subject to varying price control mechanisms as part of national health systems. Price controls or other changes in pricing regulation could restrict the amount that we are able to charge for our products. Accordingly, in markets outside the United States, the potential revenue may be insufficient to generate commercially reasonable revenue and profits.

Moreover, increasing efforts by governmental and third-party payors in the United States and abroad to limit or reduce healthcare costs may result in restrictions on coverage and the level of reimbursement for new products and, as a result, ~~they may not cover~~inadequate coverage or ~~provide adequate~~ payment for our products.

In the EU, coverage and reimbursement status of our products, if approved, are provided for by the national laws of the EU Member States. In the EU, pricing and reimbursement schemes vary widely from Member State to Member State. Some Member States provide that products can only be effectively marketed after a reimbursement price has been agreed. Some Member States may require the completion of additional studies in order to compare the cost-effectiveness of a particular drug candidate to currently available therapies (so called health technology assessment, or HTA) in order to obtain reimbursement or pricing approval. EU Member States may approve a specific price for a product or it may instead adopt a system of direct or indirect controls on the profitability of the Company placing the product on the market. Other Member States may allow companies to fix their own prices for products, but monitor and control prescription volumes and issue guidance to physicians to limit prescriptions. Recently, many countries in the EU have increased the amount of discounts required on pharmaceuticals and these efforts could continue as countries attempt to manage healthcare expenditures, especially in light of the severe fiscal and debt crises experienced by many countries in the EU. The downward pressure on healthcare costs in general, particularly prescription drugs, has become intense. As a result, increasingly high barriers are being erected to the entry of new products. Political, economic and regulatory developments may further complicate pricing negotiations, and pricing negotiations may continue after reimbursement has been obtained. Reference pricing used by various EU Member States, and parallel trade (arbitrage between low-priced and high-priced Member States), can further reduce prices. There can be no assurance that any country that has price controls or reimbursement limitations for drug products will allow favorable reimbursement and pricing arrangements for any of our products, if approved in those countries.

We expect to experience pricing pressures in connection with products due to ~~the increasing trend toward managed~~increased and continued efforts to limit or reduce healthcare ~~including the increasing influence of health maintenance organizations and additional legislative changes.~~spending. The downward pressure on healthcare costs in general, particularly prescription drugs, has and is expected to continue to increase in the future. As a result, profitability of our products may be more difficult to achieve even if they receive regulatory approval.

We intend to rely on a combination of exclusivity from ~~orphan drug~~Orphan Drug designation as well as patent rights for our product candidates and any future product candidates. If we are unable to obtain or maintain exclusivity from the combination of these approaches, we may not be able to compete effectively in our markets.

Our business strategy is to focus on product candidates for which ~~orphan drug~~Orphan Drug designation may be obtained in the major markets of the world. In addition, we rely or will rely upon a combination of patents, trade secret protection, and confidentiality agreements to protect the intellectual property related to our technologies and product candidates. ~~For example, the portfolio of patents licensed from Merck expires before the anticipated launch date of the lonafarnib product candidate.~~ Our success depends in large part on our and our licensors’ ability to obtain regulatory exclusivity and maintain patent and other intellectual property protection in the United States and in other countries with respect to our proprietary technology and products.

Under the Orphan Drug Act, the FDA may designate a product as an ~~orphan drug~~Orphan Drug if it is intended to treat a rare disease or condition, defined as a patient population of fewer than 200,000 in the United States, or a patient population greater than 200,000 in the United States where there is no reasonable expectation that the cost of developing the ~~drug~~product will be recovered from sales in the United States. In the European Union (the EU), the EMA’s Committee for Orphan Medicinal Products ~~or COMP,~~(COMP) grants ~~orphan drug~~Orphan Drug designation to promote the development of products that are intended for the diagnosis, prevention, or treatment of a life-threatening or chronically debilitating condition affecting not more than five in 10,000 persons in the ~~European Union.~~EU. Additionally, designation is granted for products intended for the diagnosis, prevention, or treatment of a life-threatening, seriously debilitating or serious and chronic condition when, without incentives, it is unlikely that sales of the drug in the ~~European Union~~EU would be sufficient to justify the necessary investment in developing the drug or biological product or where there is no satisfactory method of diagnosis, prevention, or treatment, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition.

In the United States, ~~orphan drug~~Orphan Drug designation entitles a party to financial incentives such as opportunities for grant funding towards clinical trial costs, tax advantages, and user-fee waivers. In addition, if a product receives the first FDA approval for the indication for which it has ~~orphan drug~~Orphan Drug designation, the product is entitled to ~~orphan drug~~Orphan Drug exclusivity, which means the FDA may not approve any other application to market the same ~~drug~~product for the same indication for a period of seven years, except in limited circumstances, such as a showing of clinical superiority over the product with orphan exclusivity or where the manufacturer is unable to assure sufficient product quantity.

Congress is considering updates to the Orphan Drug provisions of the FDCA in response to a recent decision by the U.S. Court of Appeals for the 11th Circuit. Any changes to the Orphan Drug provisions could change our opportunities for, or likelihood of success in obtaining, Orphan Drug exclusivity and would materially adversely affect our business, financial condition, results of operations, cash flows and prospects.

In the ~~European Union, orphan drug~~EU, Orphan Drug designation entitles a party to financial incentives such as reduction of fees or fee waivers and ten years of market exclusivity following drug or biological product approval. This period may be reduced to six years if the ~~orphan drug~~Orphan Drug designation criteria are no longer met, including where it is shown that the product is sufficiently profitable not to justify maintenance of market exclusivity.

Because the extent and scope of patent protection for our products may in some cases be limited, ~~orphan drug~~Orphan Drug designation is especially important for our products for which ~~orphan drug~~Orphan Drug designation may be available. For eligible ~~drugs,~~products, we plan to rely on the exclusivity period under the Orphan Drug Act to maintain a competitive position. If we do not obtain ~~orphan drug~~or maintain Orphan Drug exclusivity for our drug ~~products~~ and ~~biologic~~biological products that do not have broad patent protection, our competitors may then sell the same drug to treat the same condition sooner than if we had obtained ~~orphan drug~~Orphan Drug exclusivity and our revenue will be reduced.

Even though we have ~~orphan drug designation~~Orphan Drug designations for ~~lonafarnib~~each of our development programs in the United States and ~~Europe,~~the EU, we may not be the first to obtain marketing approval for any particular orphan indication due to the uncertainties associated with developing pharmaceutical products. Further, even if we obtain ~~orphan drug~~Orphan Drug exclusivity for a product, that exclusivity may not effectively protect the product from competition because different ~~drugs~~products with different active moieties can be approved for the same condition. Even after an ~~orphan drug~~Orphan Drug is approved, the FDA or EMA can subsequently approve the same ~~drug~~product with the same active moiety for the same condition if the FDA or EMA concludes that the later ~~drug~~product is safer, more effective, or makes a major contribution to patient care. Orphan ~~drug~~Drug designation neither shortens the development time or regulatory review time of a product candidate nor gives the product candidate any advantage in the regulatory review or approval process.

We have sought to protect our proprietary position by filing patent applications in the United States and abroad related to our product candidates that are important to our business. This process is expensive and time consuming, and we may not be able to file and prosecute all necessary or desirable patent applications at a reasonable cost or in a timely manner. It is also possible that we will fail to identify patentable aspects of our research and development output before it is too late to obtain patent protection.

The patent position of biotechnology and pharmaceutical companies generally is highly uncertain and involves complex legal and factual questions for which legal principles remain unsolved. The patent applications that we own or ~~in-licenses~~in-licensed may fail to result in issued patents with claims that cover our product candidates in the United States or in other foreign countries. There is no assurance that all potentially relevant prior art relating to our patents and patent applications has been found, which can invalidate a patent or prevent a patent from issuing from a pending patent application. Even if patents do successfully issue, and even if such patents cover our product candidates, third parties may challenge their validity, enforceability, or scope, which may result in such patents being narrowed, found unenforceable or invalidated. Furthermore, even if they are unchallenged, our patents and patent applications may not adequately protect our intellectual property, provide exclusivity for our product candidates, or prevent others from designing around our claims. Any of these outcomes could impair our ability to prevent competition from third parties, which may have an adverse impact on our business.

We, independently or together with our licensors, have filed several patent applications covering various aspects of our product candidates. We cannot offer any assurances about which, if any, patents will issue, the breadth of any such patent or whether any issued patents will be found invalid and unenforceable or will be threatened by third parties. Any successful opposition to these patents or any other patents owned by or licensed to us after patent issuance could deprive us of rights necessary for the successful commercialization of any product candidates that we may develop. Further, if we encounter delays in regulatory approvals, the period of time during which we could market a product candidate under patent protection could be reduced.

Although we have licensed a number of patents covering methods of use and certain compositions of matter, we do not have complete patent protection for our product candidates. For example, in connection with the ~~patent coverage for lonafarnib expires before~~arbitration with PRF described in Part I, Item 3 Legal Proceedings below, PRF is indirectly challenging the ~~anticipated launch date.~~validity of our in-license of certain patents covering the methods of treating HGPS and progeroid laminopathies. Likewise, most of the patents or applications covering products that we have licensed in from Stanford have limited protection outside of the United States. Therefore, a competitor could develop the same or similar product that may compete with our product candidate.

Certain of our product licenses are limited to specified indications or therapeutic areas which may result in the same compound being developed and commercialized by a third party whom we have no control over or rights against. This may result in safety data, pricing or off label uses from that third party’s product that may negatively affect the development and commercialization of our product candidates. For example, Kura has an exclusive license to tipifarnib for use in cancer indications while we have a license for anti-viral indications. As a result of Kura’s right to use the same compound in a different indication, it is possible that development and sales may impact our product development and commercialization efforts. If we cannot obtain and maintain effective protection of exclusivity from our regulatory efforts and intellectual property rights, including patent protection, for our product candidates, we may not be able to compete effectively, and our business and results of operations would be harmed.

The annual reassessment by the EMA of the risk-benefit balance, including information on the safe and effective use, for Zokinvy may not be positive, leading to a variation, suspension, revocation of our MA or fulfillment of additional specific obligations.

On July 20, 2022, we announced that the European Commission (EC) granted an MA under the exceptional circumstances procedure for Zokinvy to treat patients aged 12 months and older with HGPS and PL. The EC authorization follows the positive opinion granted by the CHMP in May 2022 which found that the risk-benefit balance for Zokinvy is favorable to recommend the granting of a MA although the rarity of the disease means that it was not possible to obtain complete information on Zokinvy during the assessment process. As a result, the MA was issued under the exceptional circumstances procedure and subject to the EMA’s continued review on an annual basis of new efficacy and safety information which may become available.

Under Article 14(8) Regulation (EC) No 726/2004, products for which the applicant can demonstrate that comprehensive data (in line with the requirements laid down in Annex I of Directive 2001/83/EC, as amended) cannot be provided (due to specific reasons foreseen in the legislation) might be eligible for marketing authorization under exceptional circumstances. This type of authorization is reviewed annually to reassess the risk-benefit balance. The fulfillment of any specific procedures/obligations imposed as part of the marketing authorization under exceptional circumstances is aimed at the provision of information on the safe and effective use of the product.

It is possible that the annual reassessment by the EMA of the risk-benefit balance including information on the safe and effective use for Zokinvy may not be positive. This could lead to the variation, suspension, revocation of our MA for Zokinvy in the EU, or lead to additional specific commitments or conditions being fulfilled.

We may not have sufficient patent term protections for our products to effectively protect our business.

Patents have a limited term. In the United States, the statutory expiration of a patent is generally 20 years after it is filed. Although various extensions may be available, the life of a patent, and the protection it affords, is limited. Even if patents covering our product candidates are obtained, once the patent life has expired for a product, we may be open to competition from generic medications. In addition, upon issuance in the United States any patent term can be adjusted based on certain delays caused by the applicant(s) or the United States Patent and Trademark Office ~~or USPTO.~~(USPTO). For example, a patent term can be reduced based on certain delays caused by the patent applicant during patent prosecution.

Patent term extensions under the Hatch-Waxman Act in the United States and under supplementary protection certificates in ~~Europe~~the EU may be available to extend the patent or data exclusivity terms of products. With respect to ~~ubenimex, lonafarnib,~~lonafarnib-based regimens, peginterferon lambda and ~~exendin 9-39,~~avexitide, a substantial portion of the potential commercial opportunity will likely rely on patent term extensions, and we cannot provide any assurances that any such patent term extensions will be obtained and, if so, for how long. As a result, we may not be able to maintain exclusivity for our products for an extended period after regulatory approval, which would negatively impact our business and results of operations. If we do not have sufficient patent terms or regulatory exclusivity to protect our products, our business and results of operations will be adversely affected.

Patent laws and rule changes could increase the uncertainties and costs surrounding the prosecution of our patent applications and the enforcement or defense of our issued patents.

Changes in either the patent laws or interpretation of the patent laws in the United States and other countries may diminish the value of our patents or narrow the scope of our patent protection. The laws of foreign countries may not protect our rights to the same extent as the laws of the United States. Publications of discoveries in the scientific literature often lag behind the actual discoveries, and patent applications in the United States and other jurisdictions are typically not published until 18 months after filing, or in some cases not at all. We therefore cannot be certain that it or our licensors were the first to make the invention claimed in our owned and licensed patents or pending applications, or that we or our licensor were the first to file for patent protection of such inventions. Assuming the other requirements for patentability are met, in the

United States prior to March 15, 2013, the first to make the claimed invention is entitled to the patent, while outside the United States, the first to file a patent application is entitled to the patent. After March 15, 2013, under the Leahy-Smith America Invents Act ~~or the~~(the Leahy-Smith ~~Act,~~Act) enacted on September 16, 2011, the United States has moved to a first to file system. The Leahy-Smith Act also includes a number of significant changes that affect the way patent applications will be prosecuted and may also affect patent litigation. The effects of these changes are currently unclear as the USPTO must still implement various regulations, the courts have yet to address any of these provisions and the applicability of the act and new regulations on specific patents discussed herein have not been determined and would need to be reviewed. In general, the Leahy-Smith Act and its implementation could increase the uncertainties and costs surrounding the prosecution of our patent applications and the enforcement or defense of our issued patents, all of which could have a material adverse effect on our business and financial condition.

If we are unable to maintain effective proprietary rights for our product candidates or any future product candidates, we may not be able to compete effectively in our markets.

In addition to the protection afforded by patents, we rely on trade secret protection and confidentiality agreements to protect proprietary know-how that is not patentable or that we elect not to patent, processes for which patents are difficult to enforce and any other elements of our product candidate discovery and development processes that involve proprietary know-how, information or technology that is not covered by patents. However, trade secrets can be difficult to protect. We seek to protect our proprietary technology and processes, in part, by entering into confidentiality agreements with our employees, consultants, scientific advisors, and contractors. We also seek to preserve the integrity and confidentiality of our data and trade secrets by maintaining physical security of our premises and physical and electronic security of our information technology systems. While we have confidence in these individuals, organizations and systems, agreements or security measures may be breached, and we may not have adequate remedies for any breach. In addition, our trade secrets may otherwise become known or be independently discovered by competitors.

Although we expect all of our employees and consultants to assign their inventions to us, and all of our employees, consultants, advisors, and any third parties who have access to our proprietary know-how, information, or technology to enter into confidentiality agreements, we cannot provide any assurances that all such agreements have been duly executed or that our trade secrets and other confidential proprietary information will not be disclosed or that competitors will not otherwise gain access to our trade secrets or independently develop substantially equivalent information and techniques. Misappropriation or unauthorized disclosure of our trade secrets could impair our competitive position and may have a material adverse effect on our business. Additionally, if the steps taken to maintain our trade secrets are deemed inadequate, we may have insufficient recourse against third parties for misappropriating the trade secret.

Third-party claims of intellectual property infringement may prevent or delay our development and commercialization efforts.

Our commercial success depends in part on our avoiding infringement of the patents and proprietary rights of third parties. There have been many lawsuits and other proceedings involving patent and other intellectual property rights in the biotechnology and pharmaceutical industries, including patent infringement lawsuits, interferences, oppositions, and reexamination proceedings before the USPTO and corresponding foreign patent offices. Numerous U.S. and foreign issued patents and pending patent applications, which are owned by third parties, exist in the fields in which we are developing product candidates. As the biotechnology and pharmaceutical industries expand and more patents are issued, the risk increases that our product candidates may be subject to claims of infringement of the patent rights of third parties.

Third parties may assert that we are ~~employing~~using or exploiting their proprietary technology without authorization. There may be third-party patents or patent applications with claims to materials, formulations, methods of manufacture, or methods for treatment related to the use or manufacture of our product candidates. ~~We have conducted~~Even if we conduct freedom to operate analyses, we would expect to do so only with respect to ~~only~~ certain of our product candidates ~~and therefore we do not know whether~~as they move through development. Accordingly, there ~~are any~~may be third-party patents that would impair our ability to commercialize ~~these~~ product ~~candidates. We also~~candidates and we cannot ~~guarantee that any of our analyses are complete and thorough, nor can we be sure~~assure you that we ~~have identified each and every patent and pending application~~could obtain a license, or even if available, whether such license might be obtained on commercially reasonable terms. Even in ~~the United States and abroad~~those situations where we conduct a freedom to operate analysis, there can be no assurance that iswe would identify all relevant or necessary patents and patent applications that may apply to the manufacture and commercialization of our product candidates. Because patent applications can take many years to issue, there may be currently pending patent applications that may later result in issued patents that our product candidates may ~~infringe.~~

~~In addition, third parties may obtain~~infringe, and if patents inissue with respect to any such application and we become aware of such issuance, we would have to determine its impact on our efforts to develop and commercialize our product candidates and the ~~future and claim that use of our technologies infringes upon these patents.~~ strategy for obtaining a license or contesting any such issued patent.

If any third-party patents were held by a court of competent jurisdiction to cover aspects of our formulations, the manufacturing process of any of our product candidates, methods of use, any molecules formed during the manufacturing process or any final product itself, the holders of any such patents may be able to block our ability to commercialize such product candidate unless we obtained a license under the applicable patents, or until such patents expire or are finally determined to be invalid or unenforceable. Such a license may not be available on commercially reasonable terms, or at all.

~~Parties~~

If we fail to obtain a license, then parties making claims against us may obtain injunctive or other equitable relief, which could effectively block our ability to further develop and commercialize one or more of our product candidates. Defense of these claims, regardless of their merit, would involve substantial litigation expense and would be a substantial diversion of employee resources from our business. In the event of a successful claim of infringement against us, we may have to pay substantial damages, including treble damages and attorneys’ fees for willful infringement, pay royalties, redesign our infringing products or obtain one or more licenses from third parties, which may be impossible or require substantial time and monetary expenditure.

We may not be successful in meeting our diligence obligations under our existing license agreements necessary to maintain our product candidate licenses in effect. In addition, if required in order to commercialize our product candidates, we may be unsuccessful in obtaining or maintaining necessary rights to our product candidates through acquisitions and in-licenses.

We currently have rights to the intellectual property, through licenses from third parties and under patents that we do not own, to develop and commercialize our product candidates. Because our programs may require the use of proprietary rights held by third parties, the growth of our business will likely depend in part on our ability to maintain in effect these proprietary rights. For example, we have certain specified diligence obligations under our ~~Stanford~~ license ~~agreements~~agreement for ~~our ubenimex and lonafarnib product candidates.~~lonafarnib. We may not be able to achieve the required diligence milestones in a timely manner, which may result in ~~a right of termination by Stanford,~~the license agreement being terminated, and we may be unable to successfully negotiate an extension or waiver of those termination rights. Any termination of license agreements with third parties with respect to our product candidates would be expected to negatively impact our business prospects.

We may be unable to acquire or in-license any compositions, methods of use, processes, or other third-party intellectual property rights from third parties that we identify as necessary for our product candidates. The licensing and acquisition of third-party intellectual property rights is a competitive area, and a number of more established companies are also pursuing strategies to license or acquire third-party intellectual property rights that we may consider attractive. These established companies may have a competitive advantage over us due to their size, cash resources, and greater clinical development and commercialization capabilities. In addition, companies that perceive us to be a competitor may be unwilling to assign or license rights to us. Even if we are able to license or acquire third-party intellectual property rights that are necessary for our product candidates, there can be no assurance that they will be available on favorable terms.

We collaborate with U.S. and foreign academic institutions to identify product candidates, accelerate our research and conduct development. Typically, these institutions have provided us with an option to negotiate an exclusive license to any of the institution’s rights in the patents or other intellectual property resulting from the collaboration. Regardless of such option, we may be unable to negotiate a license within the specified timeframe or under terms that are acceptable to us. If we are unable to do so, the institution may offer the intellectual property rights to other parties, potentially blocking our ability to pursue a program of interest to us.

If we are unable to successfully obtain and maintain rights to required third-party intellectual property, we may have to abandon development of that product candidate or pay additional amounts to the third party, and our business and financial condition could suffer.

Our product candidates marketed under NDAs may be subject to generic competition.

Under the Hatch-Waxman Act, a pharmaceutical manufacturer may file an abbreviated new drug application ~~or ANDA,~~(ANDA) seeking approval of a generic copy of an approved innovator product marketed under an NDA. Generally, in place of clinical studies intended to demonstrate safety or effectiveness, an ANDA applicant usually needs only to submit data demonstrating that its product has the same active ingredient(s), strength, dosage form, route of administration and that it is bioequivalent to the branded product. Under the Hatch-Waxman Act, a manufacturer may also submit an NDA under section 505(b)(2) that references the FDA’s finding of safety and effectiveness of a previously approved drug. A 505(b)(2) NDA product may be for a new or improved version of the original innovator product.

Innovative small molecule drugs may be eligible for certain periods of regulatory ~~exclusivity (e.g.,~~exclusivity. The FDCA provides a period of five years of non-patent exclusivity for a new drug containing a new chemical ~~entities,~~entity (NCE). During the exclusivity period, the FDA may not accept for review an ANDA or a 505(b)(2) NDA submitted by another company for another version of such product candidate where the applicant does not own or have a legal right of reference to all the data required for approval. However, an application may be submitted after four years if it contains a certification of patent invalidity or non-infringement. The FDCA also provides three years of marketing exclusivity for ~~changes~~an NDA, 505(b)(2) NDA or supplement to an approved ~~drug requiring a~~NDA if new clinical ~~study, seven years~~investigations, other than bioavailability studies, that were conducted or sponsored by the applicant are deemed by the FDA to be essential to the approval of the application, for ~~orphan drugs), which preclude~~example, for new indications, dosages or strengths of an existing product candidate. This three-year exclusivity covers only the conditions associated with the new clinical investigations and does not prohibit the FDA from approving ANDAs for product candidates containing the original active agent for other conditions of use. Five-year and three-year exclusivity will not delay the submission or approval ~~(or in some circumstances, FDA filing~~of a full NDA. However, an applicant submitting a full NDA would be required to conduct or obtain a right of reference to all of the nonclinical studies and ~~review of) an ANDA or 505(b)(2) NDA relying~~adequate and well-controlled clinical trials necessary to demonstrate safety and effectiveness.

Manufacturers may seek to launch these generic drugs following the expiration of the marketing exclusivity period, even if we still have patent protection for our drug. Competition that our drug candidates may face from generic versions of our drug candidates could materially and adversely impact our future revenue, profitability and cash flows and substantially limit our ability to obtain a return on the ~~FDA’s finding~~investments we have made in those drug candidates. Our future revenues, profitability and cash flows could also be materially and adversely affected and our ability to obtain a return on the investments we have made in those drug candidates may be substantially limited if our drug candidates, if and when approved, are not afforded the appropriate periods of ~~safety and effectiveness for the innovative drug.~~ non-patent exclusivity.

In addition to the benefits of regulatory exclusivity, an innovator NDA holder may have patents claiming the active ingredient, product formulation or an approved use of the drug, which would be listed with the product in the FDA publication, “Approved Drug Products with Therapeutic Equivalence Evaluations,” known as the “Orange Book.” If there are patents listed in the Orange Book, a generic applicant that seeks to market its product before expiration of the patents must include in the ANDA or 505(b)(2) what is known as a “Paragraph IV certification,” challenging the validity or enforceability of, or claiming non-infringement of, the listed patent or patents. Notice of the certification must be given to the innovator, too, and if within 45 days of receiving notice the innovator sues to protect its patents, approval of the ANDA is stayed for 30 months, or as lengthened or shortened by the court.

If there are patents listed for our ~~product candidates~~products in the Orange Book after approval by the FDA, ANDAs and 505(b)(2) NDAs with respect to those ~~product candidates~~products would be required to include a certification as to each listed patent indicating whether the ANDA applicant does or does not intend to challenge the patent. We cannot predict whether any patents issuing from our pending patent applications will be eligible for listing in the Orange Book, how any generic competitor would address such patents, whether we would sue on any such patents, or the outcome of any such suit.

We may not be successful in securing or maintaining proprietary patent protection in the United States and/or in other countries for products and technologies we develop or license. Moreover, if any patents that are granted and listed in the Orange Book are successfully challenged by way of a Paragraph IV certification and subsequent litigation, the affected product could more immediately face generic competition and its sales would likely decline materially. Should sales decline, we may have to write off a portion or all of the intangible assets associated with the affected product and our results of operations and cash flows could be materially and adversely affected.

Our biological product candidates for which we intend to seek licensure may face competition from biosimilars.

The Patient Protection and Affordable Care Act, as amended by the Health Care and Education Reconciliation Act, collectively the ACA, includes a subtitle called the Biologics Price Competition and Innovation Act of 2009, or BPCIA, which created an abbreviated licensure pathway for biological products that are biosimilar to or interchangeable with an FDA-licensed reference biological product. Under the BPCIA, an application for a biosimilar product may not be submitted to the FDA until four years following the date that the reference product was first licensed by the FDA. In addition, the licensure of a biosimilar product may not be made effective by the FDA until 12 years from the date on which the reference product was first licensed. During this 12-year period of exclusivity, another company may still market a competing version of the reference product if the FDA approves a full BLA for the competing product containing the sponsor’s own preclinical data and data from adequate and well-controlled clinical trials to demonstrate the safety, purity and potency of their product. The law is complex and is still being interpreted and implemented by the FDA. As a result, its ultimate impact, implementation, and meaning are subject to uncertainty. While it is uncertain when such processes intended to

implement BPCIA may be fully adopted by the FDA, any such processes could have an adverse effect on the future commercial prospects for our product candidates.

There is a risk that any product candidates we may develop that are licensed as a biological product under a BLA would not qualify for the 12-year period of exclusivity or that this exclusivity could be shortened due to congressional action or otherwise, or that the FDA will not consider any product candidates we may develop to be reference products for competing products, potentially creating the opportunity for competition sooner than anticipated. In addition, the approval of a biologic product biosimilar to one of our products could have a material adverse impact on our business as it may be significantly less costly to bring to market and may be priced significantly lower than our products. Moreover, the extent to which a biosimilar, once approved, will be substituted for any one of the reference products in a way that is similar to traditional generic substitution for non-biological products is not yet clear, and will depend on a number of marketplace and regulatory factors that are still developing.

Other aspects of the BPCIA, some of which may impact the BPCIA exclusivity provisions, have also been the subject of recent litigation, including litigation challenging the constitutionality of the ACA. For example, in December 2018, a federal district court ruled that the ACA, without the “individual mandate” penalty (which was repealed by Congress as part of the Tax Cuts and Jobs Act), is unconstitutional in its entirety. In December 2019, the U.S. Court of Appeals for the 5th Circuit upheld the district court ruling that the individual mandate provisions are unconstitutional and remanded the case back to the district court for further analysis of whether such provisions could be severed from the remainder of the ACA. On June 17, 2021, the U.S. Supreme Court dismissed the case without specifically ruling on the constitutionality of the ACA. There may, however, be other efforts to challenge, repeal, or replace the ACA in the future. We continue to evaluate the effect that the ACA and its possible repeal and replacement has (or may have) on our business and exclusivity under the BPCIA. It is uncertain the extent to which any such changes may impact our business or financial condition.

The patent protection and patent prosecution for some of our product candidates is dependent on third parties.

While we normally seek and gain the right to fully prosecute the patents relating to our product candidates, there may be times when patents relating to our product candidates are controlled by our licensors. This is the case with our agreements with Stanford and Nippon Kayaku, each of whom is primarily responsible for the prosecution of patents and patent applications licensed to us under the applicable collaboration agreements. If they or any of our future licensors fail to appropriately and broadly prosecute and maintain patent protection for patents covering any of our product candidates, our ability to develop and commercialize those product candidates may be adversely affected and we may not be able to prevent competitors from making, using, importing, and selling competing products. In addition, even where we now have the right to control patent prosecution of patents and patent applications, we have licensed from third parties, we may still be adversely affected or prejudiced by actions or inactions of our licensors in effect from actions prior to us assuming control over patent prosecution.

If we fail to comply with obligations in the agreements under which we license intellectual property and other rights from third parties or otherwise experience disruptions to our business relationships with our licensors, we could lose license rights that are important to our business.

We are a party to a number of intellectual property license and supply agreements that are important to our business and expects to enter into additional license agreements in the future. Our existing agreements impose, and we expect that future license agreements will impose, various diligence, milestone payment, royalty, purchasing, supply and other obligations on us. If we fail to comply with our obligations under these agreements, or we are subject to a bankruptcy, our agreements may be subject to termination by the licensor, in which event we would not be able to develop, manufacture or market products covered by the license or subject to supply commitments.

Although we are not currently involved in any intellectual property litigation, we may be involved in lawsuits to protect or enforce our patents or the patents of our licensors, which could be expensive, time consuming, and unsuccessful.

Competitors may infringe our patents or the patents of our licensors. Although we are not currently involved in any intellectual property litigation, if we or one of our licensing partners were to initiate legal proceedings against a third party to enforce a patent covering one of our product candidates, the defendant could counterclaim that the patent covering our product candidate is invalid and/or unenforceable. In patent litigation in the United States, defendant counterclaims alleging invalidity and/or unenforceability are commonplace. Grounds for a validity challenge could be an alleged failure to meet any of several statutory requirements, including lack of novelty, obviousness, or non-enablement. Grounds for an unenforceability assertion could be an allegation that someone connected with prosecution of the patent withheld relevant

information from the USPTO, or made a misleading statement, during prosecution. The outcome following legal assertions of invalidity and unenforceability is unpredictable.

Interference proceedings provoked by third parties or brought by us or declared by the USPTO may be necessary to determine the priority of inventions with respect to our patents or patent applications or those of our licensors. An unfavorable outcome could require us to cease using the related technology or to attempt to license rights to it from the prevailing party. Our business could be harmed if the prevailing party does not offer us a license on commercially reasonable terms. Our defense of litigation or interference proceedings may fail and, even if successful, may result in substantial costs and distract our management and other employees. In addition, the uncertainties associated with litigation could have a material adverse effect on our ability to raise the funds necessary to continue our clinical trials, continue our research programs, license necessary technology from third parties, or enter into development partnerships that would help us bring our product candidates to market.

Furthermore, because of the substantial amount of discovery required in connection with intellectual property litigation, there is a risk that some of our confidential information could be compromised by disclosure during this type of litigation. There could also be public announcements of the results of hearings, motions, or other interim proceedings or developments. If securities analysts or investors perceive these results to be negative, it could have a material adverse effect on the price of our common stock.

We may be subject to claims that our employees, consultants or independent contractors have wrongfully used or disclosed confidential information of third parties or that our employees have wrongfully used or disclosed alleged trade secrets of their former employers.

We employ individuals who were previously employed at universities or other biotechnology or pharmaceutical companies, including our competitors or potential competitors. Although we have written agreements and make every effort to ensure that our employees, consultants and independent contractors do not use the proprietary information or intellectual property rights of others in their work forums, and we are not currently subject to any claims that our employees, consultants or independent contractors have wrongfully used or disclosed confidential information of third parties, we may in the future be subject to such claims. Litigation may be necessary to defend against these claims. If we fail in defending any such claims, in addition to paying monetary damages, we may lose valuable intellectual property rights or personnel, which could adversely impact our business. Even if we are successful in defending against such claims, litigation could result in substantial costs and be a distraction to management and other employees.

We may not be able to protect our intellectual property rights throughout the world.

Filing, prosecuting, and defending patents on product candidates in all countries throughout the world would be prohibitively expensive, and our intellectual property rights in some countries outside the United States can be less extensive than those in the United States. Likewise, certain of our license agreements, for example for ubenimex, do not include patents or patent applications outside of the United States as our licensor elected not to file in foreign jurisdictions. In addition, the laws of some foreign countries do not protect intellectual property rights to the same extent as federal and state laws in the United States. Competitors may use our technologies in jurisdictions where we have not obtained patent protection to develop their own products and may also export infringing products to territories where we have patent protection, but enforcement is not as strong as that in the United States. These products may compete with our products and our patents or other intellectual property rights may not be effective or sufficient to prevent them from competing.

Many companies have encountered significant problems in protecting and defending intellectual property rights in foreign jurisdictions. The legal systems of certain countries, particularly certain developing countries, do not favor the enforcement of patents, trade secrets, and other intellectual property protection, particularly those relating to biotechnology products, which could make it difficult for us to stop the infringement of our patents or marketing of competing products in violation of our proprietary rights generally. Proceedings to enforce our patent rights in foreign jurisdictions, whether or not successful, could result in substantial costs and divert our efforts and attention from other aspects of our business, could put our patents at risk of being invalidated or interpreted narrowly and our patent applications at risk of not issuing and could provoke third parties to assert claims against us. We may not prevail in any lawsuits that we initiate, and the damages or other remedies awarded, if any, may not be commercially meaningful. Accordingly, our efforts to enforce our intellectual property rights around the world may be inadequate to obtain a significant commercial advantage from the intellectual property that we develop or license.

Risks Related to our Business Operations

We have previously identified a material weakness in our internal control over financial reporting and our current management team may fail to maintain an effective system of internal control, which may result in material misstatements of our financial statements or cause us to fail to meet our periodic reporting obligations as a public company.

In connection with the audit of our consolidated financial statements for the years ended December 31, 2015 and 2014 as a private company, we and our independent auditors identified a material weakness in our internal control over financial reporting. A material weakness is a deficiency, or a combination of deficiencies, in internal control over financial reporting such that there is a reasonable possibility that a material misstatement of the subject company’s annual or interim financial statements will not be prevented or detected on a timely basis. Our lack of sufficient accounting personnel resulted in the identification of a material weakness in our internal control over financial reporting. Specifically, the material weakness that was identified related to a lack of sufficient accounting resources and personnel that had limited our ability to adequately segregate duties, perform sufficient review and approval of manual journal entries posted to the general ledger, establish defined accounting policies and procedures or perform timely reviews of account reconciliations or accounting estimates.

During 2016, we implemented measures to improve our internal control over financing reporting to address the underlying causes of the previously identified material weakness, including (i) the hiring of our Controller and other accounting personnel, (ii) establishing segregation of duties for review and approval of manual journal entries, (iii) establishing accounting policies and procedures, (iv) performing timely reviews of account reconciliations and accounting estimates, and (v) implementing appropriate disclosure controls and procedures. We believe the remediation steps outlined above were sufficient to remediate the previously identified material weakness in internal control over financial reporting as discussed above.

Our failure to implement and maintain effective internal control over financial reporting could result in errors in our financial statements that could result in a restatement of our financial statements or cause us to fail to meet our reporting obligations. In addition, although we are not currently required to formally test our internal controls for attestation, we are required as an “emerging growth company” to report on our internal controls. After we are no longer an emerging growth company, any testing by us conducted in connection with Section 404 of the Sarbanes-Oxley Act, or the subsequent testing by our independent registered public accounting firm, may reveal deficiencies in our internal controls over financial reporting that are deemed to be material weaknesses or that may require prospective or retroactive changes to our consolidated financial statements or identify other areas for further attention or improvement. Inadequate internal controls could also cause investors to lose confidence in our reported financial information, which could have a negative effect on the trading price of our common stock.

Our future success depends in part on our ability to attract, retain, and motivate qualified personnel.

On December 14, 2022, David Cory resigned as our President and Chief Executive Officer and ~~to attract, retain,~~a member of our Board, and ~~motivate other qualified personnel.~~

on January 3, 2023, Sriram Ryali notified the Company of his resignation as the Company’s Chief Financial Officer, effective January 20, 2023, and on February 17, 2023, Erik Atkisson resigned as our General Counsel, Corporate Secretary and Chief Compliance Officer. The Company has also experienced recent turnover in its financial reporting function.

The Board has appointed Dr. David Apelian as the Company’s Interim Chief Executive Officer, and Dr. Apelian will remain a member of the Board. We are highly dependent on ~~David Cory, our President and~~Dr. Apelian until we retain a permanent Chief Executive Officer. On April 13, 2023, Eiger announced the appointments of William G. Kachioff as Chief Financial Officer, and James A. Vollins as General Counsel, Chief Compliance Officer and Corporate Secretary, to the ~~loss of whose services may adversely impact the achievement of our objectives. Mr. Cory could leave our employment at any time, as he is an “at will” employee.~~ Company's management team.

Recruiting and retaining other qualified employees, consultants, and advisors for our business, including scientific and technical personnel, will also be critical to our success. There is currently a shortage of highly qualified personnel in our industry, which is likely to continue. As a result, competition for personnel is intense and the turnover rate can be high. We may not be able to attract and retain personnel on acceptable terms given the competition among numerous pharmaceutical and biotechnology companies for individuals with similar skill sets. In addition, failure to succeed in development and commercialization of our product candidates may make it more challenging to recruit and retain qualified personnel. The inability to recruit and retain qualified personnel, or the loss of the services of ~~Mr. Cory~~Dr. Apelian or other members of our management team, or the failure to hire a permanent Chief Executive Officer and other qualified management personnel, may impede the progress of our research, development, and commercialization objectives and would negatively impact our ability to ~~succeed in our in-licensing strategy.~~

succeed.

We will need to expand our organization and we may experience difficulties in managing this growth, which could disrupt our operations.*

As of ~~September 30, 2017,~~March 31, 2023, we had 1751 full-time employees. As our development and commercialization plans and strategies develop, we expect to need additional managerial, operational, manufacturing, sales, marketing, financial, legal, and other resources. Our management may need to divert a disproportionate amount of their attention away from our day-to-day activities and devote a substantial amount of time to managing these growth activities. We may not be able to effectively manage the expansion of our operations, which may result in weaknesses in our infrastructure, operational mistakes, loss of business opportunities, loss of employees, and reduced productivity among remaining employees. Our expected growth could require significant capital expenditures and may divert financial resources from other projects, such as the development of additional product candidates. If our management is unable to effectively manage our growth, our expenses may increase more than expected, our ability to generate and/or grow revenue could be reduced and we may not be able to implement our business strategy. Our future financial performance and our ability to commercialize product candidates and compete effectively will depend, in part, on our ability to effectively manage any future growth.

Failure to comply with existing or future laws and regulations related to privacy or data security could lead to government enforcement actions (which could include civil or criminal fines or penalties), private litigation, other liabilities, and/or adverse publicity. Compliance or the failure to comply with such laws could increase the costs of our products and services, could limit their use or adoption, and could otherwise negatively affect our operating results and business.

Regulation of data processing is evolving as federal, state, and foreign governments continue to adopt new, or modify existing, laws and regulations addressing data privacy and security, and the collection, processing, storage, transfer, and use of data. We and our partners may be subject to current, new, or modified federal, state, and foreign data protection laws and regulations (e.g., laws and regulations that address data privacy and data security including, without limitation, health data). These new or proposed laws and regulations are subject to differing interpretations and may be inconsistent among jurisdictions, and guidance on implementation and compliance practices are often updated or otherwise revised, which adds to the complexity of processing personal data. These and other requirements could require us or our partners to incur additional costs to achieve compliance, limit our competitiveness, necessitate the acceptance of more onerous obligations in our contracts, restrict our ability to use, store, transfer, and process data, impact our or our partners’ ability to process or use data in order to support the provision of our products or services, affect our or our partners’ ability to offer our products and services in certain locations, or cause regulators to reject, limit or disrupt our clinical trial activities.

In the United States, numerous federal and state laws and regulations, including state data breach notification laws, state health information privacy laws, and federal and state consumer protection laws (e.g., Section 5 of the Federal Trade Commission Act) that govern the collection, use, disclosure, and protection of health-related and other personal information apply to our operations or the operations of our partners. In addition, we may receive unintended health information in error from third parties (including research institutions from which we may obtain clinical trial data) that are subject to privacy and security requirements under the federal Health Insurance Portability and Accountability Act of 1996 (HIPAA) as amended by the Health Information Technology for Economic and Clinical Health Act of 2009 (HITECH). Depending on the facts and circumstances, we could be subject to significant penalties if we obtain, use, or disclose individually identifiable health information in a manner that is not authorized or permitted by HIPAA. On December 10, 2020, the Office of Civil Rights within the Department of Health and Human Services issued proposed revisions to the HIPAA Privacy Rule aimed at reducing regulatory burdens that may exist in discouraging coordination of care and patient access to their health information, among other changes. While a final rule has not yet been issued, if adopted, these proposed changes may require us to update our policies and procedures to comply with the new requirements. In particular, several state laws have recently been passed or amended to significantly expand privacy rights and obligations of businesses that process personal information, including identifiable sensitive health information. For example, in June 2018, California enacted the California Consumer Privacy Act (CCPA), which went into effect on January 1, 2020. The CCPA gives California residents expanded rights to access and require deletion of their personal information, opt out of certain personal information sharing, and receive detailed information about how their personal information is used. The CCPA provides for civil penalties for violations, as well as a private right of action for data breaches that may increase data breach litigation. The California Privacy Rights Act of 2020 (CPRA), which expands the CCPA, was passed in the election on November 3, 2020. The CPRA will, among other things, give consumers the ability to limit use of information deemed to be sensitive, increase the maximum penalties for violations concerning consumers under age 16, and establish the California Privacy Protection Agency to implement and enforce the new law and impose administrative fines. Following the CPRA, Virginia and Colorado have enacted similar, but not completely consistent, comprehensive privacy legislation that will also go into effect in January and July 2023, respectively. Many other states are considering similar legislation.

Aspects of these new state privacy laws, and their interpretation and enforcement, remain uncertain. The potential effects of these new and evolving state privacy laws are far-reaching and may require us to modify our data processing practices and policies and to incur substantial costs and expenses in an effort to comply. Although the California, Virginia and Colorado laws include exemptions for certain clinical trials data and HIPAA protected health information, the law may increase our compliance costs and potential liability with respect to other personal information we collect about California residents, and those health-data related exceptions may evolve through amendment or regulatory interpretation. The state privacy law developments, moreover, have prompted a number of additional proposals for new federal and state privacy legislation that, if passed, could increase our potential liability, increase our compliance costs and adversely affect our business.

Foreign data protection laws, including, without limitation, the EU GDPR that took effect in May 2018, and EU Member State data protection legislation, may also apply to health-related and other personal information obtained from individuals. The EU GDPR has been transposed into the national laws of the United Kingdom by virtue of section 3 of the European Union (Withdrawal) Act 2018 as the UK GDPR (together, the EU GDPR and the UK GDPR, the GDPR). The EU GDPR has direct effect in all EU Member States and has extraterritorial effect where organizations outside of the EU process personal information of individuals in the EU in relation to the offering of goods or services to those individuals (targeting test) or the monitoring of their behavior (monitoring test). The UK GDPR has a similar extraterritorial test for organizations outside of the UK processing personal information of individuals in the UK in relation to the offering of goods or services to those individuals or the monitoring of their behavior. As such, the GDPR applies to us to the extent we are established in an EU Member State or the UK or we meet the requirements of either the targeting test or the monitoring test. These laws impose strict obligations on businesses, including to: (i) implement administrative, physical, technical, and organizational safeguards to protect personal information; (ii) establish an appropriate and valid legal basis for processing personal information; (iii) comply with accountability transparency requirements regarding the processing of personal information, which require controllers to demonstrate and record compliance with the GDPR and to provide more detailed information to data subjects regarding processing; (iv) comply with data protection rights of data subjects including a right of access to and rectification of personal information, a right to obtain restriction of processing or to object to processing of personal information, a right to ask for a copy of personal information to be provided to a third party in a useable format and erasing personal information in certain circumstances; (v) report certain personal data breaches to the relevant supervisory authority without undue delay (and no later than 72 hours, where feasible); (vi) obtain explicit consent for collection of sensitive personal information such as health data; and (vii) consider data protection as any new products or services are developed and to limit the amount of personal information processed.

The EU GDPR restricts the transfer of personal information from the European Economic Area (EEA) to the United States and other countries that the European Commission does not recognize as having “adequate” data protection laws unless the parties to the transfer have implemented an appropriate data transfer mechanism in accordance with the EU GDPR. The UK GDPR has similar restrictions on transfers of personal information from the United Kingdom to countries that the UK does not recognize as having “adequate” data protection laws in the United Kingdom (as discussed below) and Switzerland impose similar restrictions. One of the primary mechanisms allowing United States companies to import personal information from Europe had been certification to the EU-U.S. Privacy Shield and Swiss-U.S. Privacy Shield frameworks administered by the United States Department of Commerce. However, in July 2020, the Court of Justice of the EU (CJEU) invalidated the EU-U.S. Privacy Shield, and subsequent regulatory guidance required additional compliance efforts to analyze international data flows and take steps to ensure adequate protections for personal data transferred to the United States and other certain jurisdictions, including by implementing supplementary measures that provide privacy protections in addition to those provided under the Standard Contractual Clauses (SCCs). Moreover, new versions of the European Commission’s Standard Contractual Clauses, now the primary mechanism for the lawful transfer of personal information transfers from Europe and/or the United Kingdom to the United States or other countries, have been released requiring additional compliance and implementation efforts. The United Kingdom is also expected to publish its own set of SCCs in early 2022 for transfers of personal data outside of the United Kingdom. Similarly, the Swiss Federal Data Protection and Information Commissioner announced that the Swiss-U.S. Privacy Shield Framework is inadequate for personal information transfers from Switzerland to the United States in light of the CJEU’s July 2020 decision, and also raised questions about the viability of the older version of the Standard Contractual Clauses. As such, any transfers by us or our vendors of personal information from Europe may not comply with European data protection law, may increase our exposure to the EU GDPR’s heightened sanctions for violations of its cross-border data transfer restrictions and may reduce demand from companies subject to European data protection laws.

Moreover, where we rely on SCCs, we must now evaluate and implement supplementary measures that provide privacy protections additional to those provided under SCCs. This evaluation will, in particular, include an assessment as to whether the types of personal data transferred pursuant to SCCs may be subject to government surveillance in the data importer’s country and an assessment as to whether the data importer can meet its contractual obligations under the SCCs. This may have implications for our cross-border data flows and may result in compliance costs. Inability to import personal information from the EEA, United Kingdom or Switzerland may also restrict our clinical trial activities in Europe; limit our ability to collaborate with CROs, service providers, contractors and other companies subject to European data protection laws; and require us to increase our data processing capabilities in Europe at significant expense.

Additionally, other countries outside of Europe continue to enact or are considering enacting similar cross-border data transfer restrictions and laws requiring local data residency, which could increase the cost and complexity of delivering our services and operating our business. For example, Brazil enacted the General Data Protection Law (Lei Geral de Proteção de Dados Pessoais or LGPD) (Law No. 13,709/2018), which broadly regulates the processing of personal information and imposes compliance obligations and penalties comparable to those of the EU GDPR.

Under the EU GDPR, regulators may impose substantial fines and penalties for non-compliance. Companies that violate the EU GDPR can face fines of up to the greater of 20 million Euros or 4% of their consolidated worldwide annual turnover (revenue) and restrictions or prohibitions on data processing. The EU also confers a private right of action on data subjects and consumer associations to lodge complaints with supervisory authorities, seek judicial remedies and obtain compensation for damages resulting from violations of the EU GDPR. The EU GDPR has increased our responsibility and liability in relation to personal information that we process, requiring us to put in place additional mechanisms to ensure compliance with the EU GDPR and other EU and international data protection rules. There may also be a risk that the measures will not be implemented correctly or that individuals within the business will not be fully compliant with the required procedures. Similarly, the UK GDPR introduces fines of up to the greater of £17.5 million or up to 4% of their consolidated worldwide annual turnover (revenue).

Failure to comply with U.S. and international data protection laws and regulations could result in government enforcement actions (which could include civil or criminal penalties, fines or sanctions), private litigation, and/or adverse publicity and could negatively affect our operating results and business. Moreover, patients about whom we or our partners obtain information, as well as the providers who share this information with us, may contractually limit our ability to use and disclose the information. Claims that we have violated individuals’ privacy rights, failed to comply with data protection laws, or breached our contractual obligations related to security or privacy, even if we are not found liable, could be expensive and time-consuming to defend and could result in adverse publicity that could harm our business. Compliance with data protection laws may be time-consuming, require additional resources and could result in increased expenses,

reduce overall demand for our products and services and make it more difficult to meet expectations of or commitments to customers or partners.

Any of these matters could materially adversely affect our business, financial condition, or operational results.

Failure in our information technology and storage systems or our security measures, including without limitation, data breaches, or inadequacy of our business continuity and disaster recovery plans and procedures, could significantly disrupt the operation of our business.

Our ability to execute our business plan and maintain operations depends on the continued and uninterrupted performance of our, and our third-party vendors’, information technology or(IT) systems, and the availability of data related to our products, services and operations. IT ~~systems. IT~~ systems and data are vulnerable to risks and damages from a variety of sources, including catastrophe or natural disaster, telecommunications or network failures, malicious human acts, ~~and~~breaches of security, cyber-attacks, loss of power or other natural ~~disasters.~~or man-made events. Moreover, despite network security and back-up measures, ~~some of~~we and our vendors frequently defend against and ~~our~~respond to data security attacks and incidents, and vendors’ servers are potentially vulnerable to physical or electronic break-ins, computer viruses, software vulnerabilities, ransomware attacks and similar disruptive problems. If our business continuity and disaster recovery plans and procedures were disrupted, inadequate or unsuccessful in the event of a problem, we could experience a material adverse interruption of our operations.

Specifically, data security breaches, whether inadvertent or intentional, by employees or others, may expose proprietary information, trade secrets, personal information, clinical trial data or other sensitive data to unauthorized persons, impact the integrity, availability or confidentiality of our IT systems or data (including, but not limited to, data loss), or disrupt or interrupt our IT systems or operations. Our partners and vendors face similar risks and any security breach of their systems could adversely affect our security posture. Malicious attacks by third parties are of ever-increasing sophistication and can be made by groups and individuals with a wide range of motives (including, but not limited to, industrial espionage and financial motivation prompted by the enormous growth in ransomware over the past several years) and expertise, including organized criminal groups, “hacktivists,” nation states and others. Foreign, federal, and state laws or regulations allows for the imposition of civil liability, fines and/or corrective action on entities that improperly use or disclose the personal information of individuals, including through a data security breach. Accordingly, data security breaches experienced by us, our collaborators or contractors could lead to significant fines, required corrective action, loss of trade secrets or other intellectual property, or could lead to the public exposure of personally identifiable information (including sensitive personal information) of our employees, collaborators, clinical trial patients, and others. A data security breach or privacy violation that leads to disclosure or modification of or prevents access to personal information, including personally identifiable information, patient information or protected health information, could result in civil liability, harm our reputation, compel us to comply with federal and/or state breach notification laws, subject us to mandatory corrective action, require us to verify the correctness of database contents and otherwise subject us to liability under laws and regulations that protect personal data, resulting in increased costs or loss of revenue. If we are unable to adequately prevent, detect or respond to data security breaches or privacy violations, or implement satisfactory remedial measures in the wake of a data security incident, our operations could be disrupted, and we may suffer civil liability to our customers or individuals, loss of reputation, financial loss and other regulatory penalties because of lost or misappropriated information, including sensitive patient data, or our clinical trials may be adversely impacted from data loss resulting in delayed regulatory approvals or other operational impacts. In addition, these breaches and other inappropriate access events can be difficult to detect, and any delay in identifying and responding to them may lead to increased harm of the type described above. Moreover, the prevalent use of mobile devices or other remote working activity that access and process confidential information remotely increases the risk of data security breaches, which could lead to the loss of confidential information, trade secrets or other intellectual property. While we have implemented security measures designed to protect our data security and information technology systems, no set of security measures is infallible, and these measures may not prevent such events.

For example, in March 2021, we learned we were the victim of a business email compromise during which an unauthorized party gained access to the email account of an employee in our finance department. The incident resulted in a net loss of approximately $0.3 million. Based on our investigation, the incident was financially motivated and impacted a single email account. In response to the incident, we conducted a review of our corporate information technology and email policies and have implemented additional security measures.

Despite precautionary measures to prevent anticipated and unanticipated problems, including data breaches, there can be no assurance that our efforts to protect our data and information technology systems will prevent breakdowns or breaches in

our systems (or that of our third-party providers). Such events could affect our IT systems, sustained or repeated system failures that interrupt our ability to generate, use and maintain data or our IT systems could adversely affect our ability to operate our ~~business.~~

business and result in increased costs or loss of revenue, other financial and reputational harm to us, theft of trade secrets and other proprietary information, legal claims or proceedings, liability under laws that protect the privacy of personal information and regulatory penalties.

The financial exposure from the events referenced above could either not be insured against or not be fully covered through any insurance that we maintain. There can be no assurance that the limitations of liability in our contracts would be enforceable or adequate or would otherwise protect us from liabilities or damages as a result of the events referenced above.

We may not be successful in any efforts to identify, license, discover, develop or commercialize additional product candidates.

Although a substantial amount of our effort will focus on the continued clinical testing, potential approval, and commercialization of our existing product candidates, the success of our business is also expected to depend in part upon our ability to identify, license, discover, develop, or commercialize additional product candidates. Research programs to identify new product candidates require substantial technical, financial, and human resources. We may focus our efforts and resources on potential programs or product candidates that ultimately prove to be unsuccessful. Our research programs or licensing efforts may fail to yield additional product candidates for clinical development and commercialization for a number of reasons, including but not limited to the following:

•our research or business development methodology or search criteria and process may be unsuccessful in identifying potential product candidates;

•we may not be able or willing to assemble sufficient resources to acquire or discover additional product candidates;

•our product candidates may not succeed in preclinical or clinical testing;

•our potential product candidates may be shown to have harmful side effects or may have other characteristics that may make the products unmarketable or unlikely to receive marketing approval;

•competitors may develop alternatives that render our product candidates obsolete or less attractive;

•product candidates we develop may be covered by third parties’ patents or other exclusive rights;

•the market for a product candidate may change during our program so that such a product may become unreasonable to continue to develop;

•a product candidate may not be capable of being produced in commercial quantities at an acceptable cost, or at all; and

•a product candidate may not be accepted as safe and effective by patients, the medical community, or third-party payors.

If any of these events occur, we may be forced to abandon our development efforts for a program or programs, or we may not be able to identify, license, discover, develop, or commercialize additional product candidates, which would have a material adverse effect on our business and could potentially cause us to cease operations.

Healthcare legislative reform measures may have a material adverse effect on our business and results of operations.*

In the United States, there have been and continue to be a number of legislative initiatives to ~~contain~~reform the delivery and payment for healthcare ~~costs.~~items and services. For example, in March 2010, the Patient Protection and Affordable Care Act ~~as amended by the Health Care and Education Reconciliation Act, or the ACA,~~(ACA) was passed, which substantially ~~changes~~changed the way health care is financed by both governmental and private insurers, and significantly impacts the U.S. pharmaceutical industry. ~~Some of~~This law was designed to expand access to health insurance coverage for uninsured and underinsured individuals while at the ~~provisions of the Affordable Care Act have yet to be fully implemented, and since its enactment, there have been judicial and Congressional challenges to numerous provisions~~same time containing overall healthcare costs. The framework of the ACA and other healthcare reforms continues to evolve as ~~well as recent efforts by~~a result of executive, legislative, regulatory, and administrative developments. For example, for calendar years 2021 and 2022, the ~~Trump administration~~American Rescue Plan Act of 2021 temporarily increased premium tax credit assistance established under the ACA to ~~repeal or replace~~help eligible individuals cover premiums for health insurance purchased through the health insurance marketplace and removed the 400% federal poverty level limit that otherwise applies for purposes of eligibility to receive premium tax credits. The Inflation Reduction Act of 2022(IRA) extended this increased tax credit assistance and removal of the 400% federal poverty limit through 2025. There have also

been legal challenges to certain aspects of the ACA. ~~Since January 2017, President Trump has signed two Executive Orders designed~~On June 17, 2021, the U.S. Supreme Court dismissed a recent judicial challenge to ~~delay the implementation of any certain provisions of~~ the ACA ~~or otherwise circumvent some of~~brought by several states without specifically ruling on the requirements for health insurance mandated by the ACA. The Trump administration has also announced that it will discontinue the payment of cost-sharing reduction, or CSR, payments to insurance companies until Congress approves the appropriation of funds for the CSR payments. The loss of the CSR payments is expected to increase premiums on certain policies issued by qualified health plans under the ACA. ~~A bipartisan bill to appropriate funds for CSR payments has been introduced in the Senate, but the future of that bill is uncertain.~~ ~~Further, each chamber of Congress has put forth multiple bills designed to repeal or repeal and replace portions~~constitutionality of the ACA. ~~Although none~~It is unclear how the healthcare reform measures of ~~these measures~~the Biden administration or other efforts, if any, to challenge, repeal, amend or replace the ACA will impact the ACA and our business.

Also, there has been ~~enacted by Congress to date, Congress may consider other legislation to repeal and replace elements~~heightened governmental scrutiny over pharmaceutical pricing practices in light of the ~~ACA.~~rising cost of prescription drugs and biologics. Such scrutiny has resulted in Congressional and federal agency inquiries regarding pricing and related practices, as well as proposed and enacted federal and state legislation and regulation designed to, among other things, bring more transparency to product pricing, review the relationship between pricing and manufacturer patient programs, and reform government program reimbursement methodologies for products. For example, the American Rescue Plan Act of 2021 included among its provisions a sunset of the ACA’s cap on pharmaceutical manufacturers’ rebate liability under the Medicaid Drug Rebate Program. Previously, under the ACA, manufacturers’ rebate liability was capped at 100% of the average manufacturer price for a covered outpatient drug. Effective January 1, 2024, manufacturers’ Medicaid Drug Rebate Program rebate liability will no longer be capped, potentially resulting in a manufacturer paying more in Medicaid Drug Rebate Program rebates than it receives on the sale of certain covered outpatient drugs. The Biden Administration has continued to support legislative reforms to lower prescription drug prices. In August 2022, President Biden signed into law the IRA, which implements substantial changes to the Medicare program, including drug pricing reforms and changes to the Medicare Part D benefit design.Among other reforms, the Inflation Reduction Act of 2022 imposes inflation rebates on drug manufacturers for products reimbursed under Medicare Parts B and D if the prices of those products increase faster than inflation; implements changes to the Medicare Part D benefit that, beginning in 2025, will cap benefit annual out-of-pocket spending at $2,000, while imposing new discount obligations for pharmaceutical manufacturers; and, beginning in 2026, establishes a “maximum fair price” for a fixed number of pharmaceutical and biological products covered under Medicare Parts B and D following a price negotiation process with the Centers for Medicare and Medicaid Services. The IRA explicitly excludes from price negotiation orphan drugs designated for only one rare disease or condition and for which the only approved indication is for such disease or condition. However, those drugs with multiple orphan designations are not explicitly excluded from drug price negotiation. CMS continues to release guidance implementing the IRA and the Biden administration continues to direct the Department of Health and Human Services to consider new healthcare payment and delivery models that would lower drug costs and promote access to innovative therapies for beneficiaries enrolled in the Medicare and Medicaid programs.We cannot predict the likelihood, nature or extent of healthcare or drug pricing reforms that may arise from future legislation or administration action nor can we predict how, or to what extent, the drug pricing policies or future healthcare reforms will affect our products. However, we expect these initiatives to increase pressure on drug pricing.

At the state level, legislatures have increasingly passed legislation and implemented regulations designed to control pharmaceutical and biological product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product access and marketing cost disclosure and transparency measures, and, in some cases, designed to encourage importation from other countries and bulk purchasing. Any ~~repeal and replace~~ legislation at the state or federal level aimed at further healthcare reform may have the effect of limiting the amounts that government agencies will pay for healthcare products and services. ~~Policy~~For example, an emerging trend at the state level is the establishment of prescription drug affordability boards, some of which will prospectively permit certain states to establish upper payment limits for drugs that the state has determined to be “high-cost”.In addition, policy changes, including potential modification or repeal of all or parts of the ~~Affordable Care Act~~ACA or the implementation of new health care legislation, could result in significant changes to the health care system, which may prevent us from being able to generate revenue, attain profitability or

commercialize our drugs. We expect that additional state and federal healthcare reform measures will be adopted in the future, any of which could limit the amounts that federal and state governments will pay for healthcare products and services, which could result in reduced demand or lower pricing for our product candidates, or additional pricing pressures.

In the United States, the ~~European Union~~EU and other potentially significant markets for our product candidates, government authorities and third-party payors are increasingly attempting to limit or regulate the price of medical products and services, particularly for new and innovative products and therapies, which has resulted in lower average selling prices. For example, in the United States, there have been several recent Congressional inquiries and proposed bills designed to, among other things, bring more transparency to drug pricing, review the relationship between pricing and manufacturer patient programs, and reform government program reimbursement methodologies for drugs. Furthermore, theThe increased emphasis on managed healthcare in the United States and on country and regional pricing and reimbursement controls in the ~~European Union~~EU will put additional pressure on product pricing, reimbursement and usage, which may adversely affect our future product sales and results of operations. These pressures can arise from rules and practices of managed care groups, judicial decisions and governmental laws and regulations related to Medicare, Medicaid and healthcare reform, pharmaceutical reimbursement policies and pricing in general.

Further, it is possible that additional governmental action is taken in response to the COVID-19 pandemic.

We may be subject, directly or indirectly, to foreign, federal and state healthcare fraud and abuse laws, false claims laws, and health information privacy and security laws. If we are unable to comply, or ~~has~~have not fully complied, with such laws, itwe could face substantial ~~penalties.~~

~~If we obtain FDA approval for any of our product candidates and begin commercializing those products in the United States, our~~penalties, sanctions or other liability.

Our operations may be subject to various foreign, federal and state fraud and abuse laws, including, without limitation, the federal Anti-Kickback Statute, the federal False Claims Act, ~~and~~ physician sunshine laws, the EU GDPR and/or the UK GDPR and other regulations. These laws may impact, among other things, our ~~proposed~~research, sales, marketing, education and ~~education~~patient assistance programs. In addition, we may be subject to patient privacy regulation by ~~both the~~foreign, federal, ~~government~~ and ~~the states~~state governments in which we conduct our business. The laws that may affect our ability to operate include:

•the federal Anti-Kickback Statute, which prohibits, among other things, persons from knowingly and willfully soliciting, receiving, offering or paying anything of value as remuneration, directly or indirectly, to induce, or in return for, the purchase or recommendation of an item or service reimbursable under a federal healthcare program, such as the Medicare and Medicaid programs;

•federal civil and criminal false claims laws and civil monetary penalty laws, which prohibit, among other things, individuals or entities from knowingly presenting, or causing to be presented, claims for payment from Medicare, Medicaid, or other third-party payors that are false or fraudulent;

~~the federal Health Insurance Portability and Accountability Act of 1996, or~~ •HIPAA, which created new federal criminal statutes that prohibit executing a scheme to defraud any healthcare benefit program and making false statements relating to healthcare matters;

•HIPAA ~~as amended by the Health Information Technology~~ and ~~Clinical Health Act, or HITECH, and our~~its implementing regulations ~~which imposes~~impose certain requirements on certain covered entity healthcare providers, health plans, and healthcare clearinghouse and their business associates that perform certain services involving the use or disclosure of individually identifiable health information as well as their covered subcontractors, relating to the privacy, security, and transmission of individually identifiable health information;

•The Physician Payments Sunshine Act, which requires manufacturers of drugs, devices, biologics, and medical supplies to report annually to ~~the U.S. Department of Health and Human Services~~HHS information related to payments and other transfers of value to physicians ~~other healthcare providers,~~(defined to include doctors, dentists, optometrists, podiatrists, and chiropractors), teaching hospitals, physician assistants, nurse practitioners, clinical nurse specialists, anesthesiologist assistants, certified registered nurse anesthetists, and certified nurse midwives, as well as ownership and investment interests held by physicians ~~and other healthcare providers~~ and their immediate family members and applicable group purchasing organizations;

•the Federal Food, Drug, and

Cosmetic Act (FDCA), which, among other things, prohibits the adulteration and misbranding of drugs and biological products;

•state law equivalents of each of the above federal laws, such as anti-kickback and false claims laws that may apply to items or services reimbursed by any third-party payors, including commercial insurers, state laws that require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government, or otherwise restrict payments that may be made to healthcare providers and other potential referral sources; state laws that require drug manufacturers to report information related to payments and other transfers of value to physicians and other healthcare providers or marketing ~~expenditures,~~expenditures; state and local laws requiring the registration of pharmaceutical sales representatives; and state laws governing the privacy and security of health information in certain circumstances, many of which differ from each other in significant ways and may not have the same effect, thus complicating compliance ~~efforts.~~

efforts; and federal and state laws requiring the disclosure of certain drug pricing information; and

•the EU GDPR and/or the UK GDPR (together, GDPR) and other EU Member State or English data protection legislation, which require data controllers and processors to (i) implement administrative, physical, technical, and organizational safeguards to protect personal information; (ii) establish an appropriate and valid legal basis for processing personal information (iii) comply with accountability transparency requirements regarding the processing of personal information, which require controllers to demonstrate and record compliance with the GDPR and to provide more detailed information to data subjects regarding processing; (iv) comply with data protection rights of data subjects including a right of access to and rectification of personal information, a right to obtain restriction of processing or to object to processing of personal information, a right to ask for a copy of personal information to be provided to a third party in a useable format and erasing personal information in certain circumstances; (v) report certain personal data breaches to the relevant supervisory authority without undue delay (and no later than 72 hours, where feasible); (vi) obtain explicit consent for collection of sensitive personal information such as health data; and (vii) consider data protection as any new products or services are developed and to limit the amount of personal information processed. In addition, the GDPR prohibits the international transfer of personal information outside of the EU and/or the UK including to the U.S., unless made to a country

deemed to have adequate data privacy laws by the European Commission and/or the UK or a data transfer mechanism in accordance with the GDPR (as applicable) has been put in place.

Because of the breadth of these laws and the narrowness of the statutory exceptions and safe harbors available, it is possible that some of our business activities could be subject to challenge under one or more of such laws. In addition, recent health care reform legislation has strengthened these laws.

If our operations are found to be in violation of any of the laws described above or any other governmental regulations that apply, we may be subject to penalties, including significant civil, criminal and ~~criminal~~administrative penalties, damages, disgorgement, fines, sanctions, exclusion from participation in government health care programs, such as Medicare and Medicaid, imprisonment, corporate integrity oversight and reporting obligations, contractual damages, reputational harm, diminished profits and future earnings, and the curtailment or restructuring of our operations, any of which could adversely affect our ability to operate our business and our results of operations.

If we fail to comply with our reporting and payment obligations under the Medicaid Drug Rebate Program or other governmental pricing programs in the US, we could be subject to additional rebate or discount requirements, fines, sanctions and exposure under other laws which could have an adverse effect on our business, results of operations and financial condition.

The Company’s product is subject to government mandated rebates under the Medicaid Drug Rebate Program and other government health care programs in the United States. These programs generally require us to pay rebates or otherwise provide discounts to government payors in connection with drugs that are dispensed to beneficiaries of these programs. In some cases, such as with the Medicaid Drug Rebate Program, rebates are calculated based on pricing metrics we are required to report on a monthly and quarterly basis to the government agencies that administer the programs. These pricing metrics are complex and may vary across products and programs. Additionally, the requirements of government health care programs change frequently and are often subject to interpretation by regulatory agencies and the courts. Tracking these updates and maintaining compliance will be costly and time consuming. Further, errors in the submission of our pricing data, failure to make necessary pricing disclosures, and/or overcharging government payors (or failing to identify overpayments) could result in allegations against us under the federal False Claims Act and other laws and regulations, as well as subject us to potential termination of our rebate agreement with CMS. Responding to government investigations or enforcement actions and/or making necessary refunds to government payors would be expensive and time consuming and could have an adverse effect on our business, results of operations and financial condition.

The withdrawal of the United Kingdom from the EU, commonly referred to as “Brexit,” may adversely impact our ability to obtain regulatory approvals of our product candidates in the EU, result in restrictions or imposition of taxes and duties for importing our product candidates into the EU, and may require us to incur additional expenses in order to develop, manufacture and commercialize our product candidates in the EU.

In June 2016, the electorate in the UK voted in favor of leaving the EU (commonly referred to as “Brexit”). Thereafter, in March 2017, the country formally notified the EU of its intention to withdraw pursuant to Article 50 of the Lisbon Treaty and the UK formally left the EU on January 31, 2020. A transition period began on February 1, 2020, during which EU pharmaceutical law remained applicable to the UK. This transition period ended on December 31, 2020. The EU-UK Trade and Cooperation Agreement (TCA) was agreed on December 24, 2020. The UK and EU agreed that the TCA would apply provisionally from January 1, 2021, the TCA was ratified on April 30, 2021 and came into force on May 1, 2021.

Since the regulatory framework in the UK covering the quality, safety and efficacy of pharmaceutical products, clinical trials, marketing authorization, commercial sales and distribution of pharmaceutical products is derived from EU Directives and Regulations, Brexit could materially impact the future regulatory regime which applies to products and the approval of product candidates in the UK, as UK legislation now has the potential to diverge from EU legislation. It remains to be seen how Brexit will fully impact regulatory requirements for product candidates and products in the UK in the long-term.

At present, the regulatory framework for medicines that existed before the end of the transition period has effectively been preserved in UK domestic legislation as ‘retained EU law’ which has prevented substantial divergence to the regulation of medicines so far. However, the UK Government has now introduced the Retained EU Law (Revocation and Reform) Bill 2022 which intends to end the “special status” of retained EU law under UK law. If enacted into law, this would facilitate departure from retained EU law, and may lead to greater regulatory divergence between the EU and UK in the future. In comparison, as matters currently stand, under the terms of the Northern Ireland Protocol (the NIP), implemented by the EU (Withdrawal Agreement) Act 2020, Northern Ireland is treated for the same purposes as if it were still an EU Member

State, and must remain aligned to the EU single market and customs rules. The UK government has also introduced a bill ‘Northern Ireland Protocol Bill’ which if enacted into law would enable the government to unilaterally disapply parts of the NIP which may lead to changes to the regulatory environment in Northern Ireland, and may trigger retaliatory measures against the UK by the EU. However, on February 27, 2023, the UK government and the EU Commission reached political agreement on a package of proposed amendments to the NIP (Windsor Framework). According to the UK government, if these proposals are implemented, this would lead to a “carve-out from EU rules” and, amongst other measures, the UK medicines regulator (MHRA) would be responsible for approving medicines for the whole UK market, meaning that medicinal products available in Northern Ireland will not have to comply with EU law but rather with UK law, so medicinal products would be able to freely circulate between Northern Ireland and Great Britain. Furthermore, the UK government has said that it will withdraw the Northern Ireland Protocol Bill under the Windsor Framework if it is implemented. The future regulatory framework for medicines in Northern Ireland will remain uncertain until the proposals are passed into law. The EU-UK Trade and Cooperation Agreement allows for future deviation from the current regulatory framework and it is not known if and/or when any deviations may occur, which may have an impact on Eiger’s business.

Companies now need to comply with a separate UK regulatory legal framework in order to commercialize medicinal products in Great Britain (namely, England, Wales and Scotland, as EU law currently applies in Northern Ireland). For instance, Great Britain will no longer be covered by centralized marketing authorizations (as matters stand, under the Northern Irish Protocol, centralized marketing authorizations will continue to be recognized in Northern Ireland). All medicinal products with a current centralized marketing authorization were automatically converted to Great Britain marketing authorizations on January 1, 2021. For a period of three years from January 1, 2021, the MHRA may rely on a decision taken by the European Commission on the approval of a new marketing authorization in the centralized procedure, in order to more quickly grant a new Great Britain marketing authorization. A separate application will, however, still be required. The MHRA has also introduced new procedures aimed at accelerating regulatory approvals and time to market, including ‘rolling reviews’ and the Innovative Licensing and Access Pathway (ILAP) although eligibility requirements apply.

The TCA and the proposed Bills allow for future deviation from the current regulatory framework and it is not known if and/or when any deviations may occur, which may have an impact on development, manufacture, marketing authorization, commercial sales and distribution of pharmaceutical products.

Our employees, principal investigators, consultants and commercial partners may engage in misconduct or other improper activities, including non-compliance with regulatory standards and requirements and insider trading.

We are exposed to the risk of fraudulent conduct or other illegal activity by our employees, independent contractors, principal investigators, consultants, commercial partners and vendors. Misconduct by these parties could include intentional, reckless and/or negligent conduct that fails to: comply with the regulations of the FDA and non-U.S. regulators, provide accurate information to the FDA and non-U.S. regulators, comply with healthcare fraud and abuse laws in the United States and abroad, report financial information or data accurately or disclose unauthorized activities to us. In particular, promotion, sales, marketing and certain business arrangements in the healthcare industry are subject to extensive laws intended to prevent fraud, misconduct, kickbacks, self-dealing and other abusive practices. These laws may restrict or prohibit a wide range of pricing, discounting, marketing and promotion, sales commission, customer incentive programs and other business arrangements. Such misconduct could also involve the improper use of information obtained in the course of patient recruitment or clinical trials, which could result in regulatory sanctions and cause serious harm to our reputation. We have adopted a code of business conduct and ethics applicable to all of our employees, but it is not always possible to identify and deter employee misconduct, and the precautions we take to detect and prevent this activity may not be effective in controlling unknown or unmanaged risks or losses or in protecting us from governmental investigations or other actions or lawsuits stemming from a failure to comply with these laws or regulations. If any such actions are instituted against us, and we are not successful in defending ourselves or asserting our rights, those actions could have a significant impact on our business, including the imposition of significant fines or other sanctions.

We face potential product liability, and, if successful claims are brought against us, we may incur substantial liability and costs. If the use or misuse of our product candidates harm patients or is perceived to harm patients even when such harm is unrelated to our product candidates, our regulatory approvals could be revoked or otherwise negatively impacted, and we could be subject to costly and damaging product liability claims.

The use or misuse of our product candidates in clinical trials and the sale of any products for which we obtain marketing approval exposes us to the risk of product liability claims. Product liability claims might be brought against us by consumers, healthcare providers, pharmaceutical companies or others selling or otherwise coming into contact with our

products. There is a risk that our product candidates may induce adverse events. If we cannot successfully defend against product liability claims, we could incur substantial liability and costs. In addition, regardless of merit or eventual outcome, product liability claims may result in:

•impairment of our business reputation;

•initiation of investigations by regulators;

•withdrawal of clinical trial participants;

•costs due to related litigation;

•distraction of management’s attention from our primary business;

•substantial monetary awards to patients or other claimants;

•the inability to commercialize our product candidates;

•product recalls, withdrawals or labeling, marketing or promotional restrictions; and

•decreased demand for our product candidates, if approved for commercial sale.

We believe our current product liability insurance coverage is appropriate in light of our clinical programs; however, we may not be able to maintain insurance coverage at a reasonable cost or in sufficient amounts to protect us against losses due to liability. If and when we obtain marketing approval for product candidates, we intend to increase our insurance coverage to include the sale of commercial products; however, we may be unable to obtain product liability insurance on commercially reasonable terms or in adequate amounts. On occasion, large judgments have been awarded in class action lawsuits based on drugs or medical treatments that had unanticipated adverse effects. A successful product liability ~~claim~~claims or series of claims brought against us could cause our stock price to decline and, if judgments exceed our insurance coverage, could adversely affect our results of operations and business.

Patients with the diseases targeted by our product candidates are often already in severe and advanced stages of disease and have both known and unknown significant pre-existing and potentially life-threatening health risks. During the course of treatment, patients may suffer adverse events, including death, for reasons that may or may not be related to our product candidates. Such events could subject us to costly litigation, require us to pay substantial amounts of money to injured patients, delay, negatively impact or end our opportunity to receive or maintain regulatory approval to market our products, or require us to suspend or abandon our commercialization efforts. Even in a circumstance in which we do not believe that an adverse event is related to our products, the investigation into the circumstance may be time-consuming or inconclusive. These investigations may interrupt our sales efforts, delay our regulatory approval process in other countries, or impact and limit the type of regulatory approvals our product candidates receive or maintain. As a result of these factors, a product liability claim, even if successfully defended, could have a material adverse effect on our business, financial condition or results of operations.

If we fail to comply with environmental, health and safety laws and regulations, we could become subject to fines or penalties or incur costs that could have a material adverse effect on the success of our business.

Our research and development activities and our third-party manufacturers’ and suppliers’ activities involve the controlled storage, use, and disposal of hazardous materials, including the components of our product candidates and other hazardous compounds. We and our manufacturers and suppliers are subject to laws and regulations governing the use, manufacture, storage, handling, and disposal of these hazardous materials. In some cases, these hazardous materials and various wastes resulting from their use are stored at our and our manufacturers’ facilities pending their use and disposal. We cannot eliminate the risk of contamination, which could cause an interruption of our commercialization efforts, research and development efforts and business operations, environmental damage resulting in costly clean-up and liabilities under applicable laws and regulations governing the use, storage, handling, and disposal of these materials and specified waste products. Although we believe that the safety procedures utilized by our licensors and our third-party manufacturers for handling and disposing of these materials generally comply with the standards prescribed by these laws and regulations, we cannot guarantee that this is the case or eliminate the risk of accidental contamination or injury from these materials. In such an event, we may be held liable for any resulting damages and such liability could exceed our resources and state or federal or other applicable authorities may curtail our use of certain materials and/or interrupt our business operations. Furthermore, environmental laws and regulations are complex, change frequently, and have tended to become more stringent. We cannot predict the impact of such changes and cannot be certain of our future compliance. We do not currently carry biological or hazardous waste insurance coverage.

We are currently conducting and will continue to conduct clinical trials in foreign countries, which could expose us to risks that could have a material adverse effect on the success of our business and the delivery of clinical trial data.*

We have conducted in the past and are currently conducting clinical trials in the United ~~States; Canada; Sydney, Australia; Ankara, Turkey; Hannover, Germany; Karachi, Pakistan; Auckland,~~States, Canada, Australia, Turkey, Germany, Pakistan, New Zealand, Mongolia, Spain, France, Bulgaria, Romania, Taiwan, Sweden, Italy, Belgium, Switzerland, United Kingdom, Greece, Moldova, Ukraine, Russia, and ~~Jerusalem and Beersheba,~~ Israel, and accordingly, we are subject to risks associated with doing business globally, including commercial, political, and financial risks. Emerging regions, such as Eastern Europe, Latin America, Asia, and Africa, as well as more developed markets, such as the United Kingdom, France, Germany, and Australia, provide clinical study opportunities for us. In addition, we are subject to potential disruption caused by military conflicts; potentially unstable governments or legal systems; civil or political upheaval or unrest; local labor policies and conditions; possible expropriation, nationalization, or confiscation of assets; problems with repatriation of foreign earnings; economic or trade sanctions; closure of markets to imports; anti-American sentiment; terrorism or other types of violence in or outside the United States; health pandemics; and a significant reduction in global travel. For example, both Turkey and Pakistan are key regions for clinical activity relating to Hepatitis Delta Virus, and further outbreaks of violence and political instability in the region could disrupt our clinical operations or otherwise limit our ability to access or conduct clinical studies in those regions. Certain countries have closed their borders due to COVID-19 preventing activation of clinical sites. Actions taken by the Russian Federation in Ukraine and surrounding areas have destabilized the region and caused the adoption of comprehensive sanctions by, among others, the EU, the United States and the UK, which restrict a wide range of trade and financial dealings with Russia and Russian persons, as well as certain regions in Ukraine, including by imposing stricter export controls, prohibiting dealings with major Russian banks and credit institutions, and prohibiting trade with the Donetsk, Luhansk, Kherson and Zaporizhzhia regions of Ukraine. In addition, clinical site initiation and patient enrollment may be delayed, and we may not be able to access sites for initiation, monitoring and data collection in regions affected by the Russian invasion of Ukraine, including due to the prioritization of hospital resources away from clinical trials or as a result of government-imposed curfews, warfare, violence or other governmental action or events that restrict movement. Some patients may not be able to comply with clinical trial protocols if the conflict impedes patient movement or interrupts healthcare services. We could also experience disruptions in our supply chain or limits to our ability to obtain sufficient materials for our products in certain regions. Our success will depend, in part, on our ability to overcome the challenges we encounter with respect to these risks and other factors affecting U.S. companies with global operations. If our global clinical trials were to experience significant disruption due to these risks or for other reasons, it could have a material adverse effect on our ~~financial results.~~

operations and prospects.

We or the third parties upon whom we depend may be adversely affected by earthquakes, natural epidemics or other natural disasters and our business continuity and disaster recovery plans may not adequately protect us from a serious disaster.

~~Our~~

Earthquakes, health epidemics or other natural disasters could severely disrupt our operations and have a material adverse effect on our business. If a natural disaster, power outage or other event occurred that prevented us from using all or a significant portion of our headquarters, damaged critical infrastructure, such as the manufacturing facilities of our third-party CMOs, or otherwise disrupted operations, it may be difficult or, in certain cases, impossible for us to continue our business for a substantial period of time. For example, in December 2019, an outbreak of a novel strain of coronavirus (COVID-19) originated in Wuhan, China. Since certain starting materials of certain of our products obtained from third-party chemical suppliers are manufactured in China and Japan, an outbreak of communicable diseases in the region, or the perception that such an outbreak could occur, and the measures taken by the governments of countries affected, could adversely affect our business, financial condition or results of operations by limiting our ability to manufacture product within or outside for example China, Japan, Italy, Canada, and the United States, forcing temporary closure of facilities that we rely upon or increasing the costs associated with obtaining starting materials and then clinical supplies of our product candidates. The extent to which the coronavirus impacts our results will depend on future developments, which are highly uncertain and cannot be accurately predicted, including new information which may emerge concerning the severity of the coronavirus and the actions to contain the coronavirus or treat its impact, among others. In addition, our corporate headquarters ~~are~~is located in the San Francisco Bay Area, which has in the past experienced severe earthquakes, ~~and~~ other natural ~~disasters.~~disasters, and an outbreak of COVID-19. We do not carry earthquake insurance. Earthquakes or other natural disasters could severely disrupt our operations or those of our collaborators, and have a material adverse effect on our business, results of operations, financial condition, and prospects. If a natural disaster, terrorist attack, power outage, or other event occurred that prevented us from using or damaged critical elements of our business and operations (such as the manufacturing facilities of our third-party contract manufacturers) our business may be disrupted for a substantial period of time. We have limited ~~or no~~ disaster recovery and business continuity plans in place currently and our business would be impaired in the event of a serious disaster or similar event. We may incur substantial expenses to develop and implement any disaster recovery and business continuity plans, which could have a material adverse effect on our business.

~~Risks Related~~

Our business is currently adversely affected by and could be materially adversely affected in the future by the effects of disease outbreaks, epidemics and pandemics including the evolving effects of the COVID-19 outbreak. We have a significant number of clinical trial sites in countries that have been directly affected by COVID-19. We depend on manufacturing operations for various stages of our supply chain in countries that have been directly affected by COVID-19. COVID-19 continues to ~~Celladon’s Historical Business Operations~~

Weadversely affect our business and could materially and adversely affect our operations and those of our manufacturers and other third parties with whom we conduct business.

Our business has been adversely affected by COVID-19 and could be materially and adversely impacted by COVID-19 or other health epidemics in regions where we have significant manufacturing facilities, concentrations of clinical trial sites or other business operations. The ongoing effects of the pandemic and government and other responses thereto, including shelter-in-place orders and our work-from-home policies, may continue to negatively impact productivity, disrupt our business and delay our clinical programs and timelines, the magnitude of which will depend, in part, on the length and severity of any restrictions and other limitations on our ability to conduct our business in the ordinary course. These and similar, and perhaps more severe, disruptions in our operations could negatively impact our business, operating results and financial condition.

Additionally, some of our suppliers of certain materials used in the production of our drug products are located in China, Japan, Canada, Italy and the ~~subject~~United States. While many of ~~securities class action lawsuits that were filed against Celladon in 2015, and additional securities litigation~~these materials may be ~~brought against us~~obtained by more than one supplier, including suppliers outside of China, Japan, Canada, Italy and the United States, port closures and other restrictions resulting from the coronavirus outbreak in the future.*

region may disrupt our supply chain or limit our ability to obtain sufficient materials for our drug products.

In ~~July 2015, following Celladon’s announcements~~addition, our clinical trials have been and may continue to be affected by the COVID-19 pandemic. Site initiation and patient enrollment has been delayed, due to prioritization of hospital resources toward the COVID-19 pandemic, travel restrictions imposed by governments, and the inability to access sites for initiation and monitoring.

Further, as a result of the ~~negative CUPID 2~~COVID-19 pandemic, the extent and length of which is uncertain, we may be required to develop and implement additional clinical study policies and procedures designed to help protect study participants from the COVID-19 virus, which may include using telemedicine visits, remote monitoring of patients and clinical sites, and measures to ensure that data from clinical studies that may be disrupted as result of the pandemic are collected pursuant to the study protocol and consistent with GCPs, with any material protocol deviation reviewed and approved by the site IRB.

The FDA and EMA have in the past suspended, may continue to suspend, prioritize or delay certain foreign inspections, and if there continues to be a suspension or delay in inspections, our product application reviews and potential approvals could be impacted or delayed. Further, ongoing surges in COVID-19 case numbers with the emergence of new variants and sub-variants have contributed to interruptions in FDA’s surveillance capabilities, and such disruptions may persist in the future as a result of future surges. We cannot predict whether, and when, FDA will decide to pause or resume inspections due to the COVID-19 pandemic.

Regulatory authorities outside the United States may adopt similar restrictions or other policy measures in response to the COVID-19 pandemic, including providing guidance regarding the conduct of clinical trials. If global health concerns continue to prevent the FDA or other regulatory authorities from conducting their regular inspections, or impact reviews or other regulatory activities, it could significantly impact the ability of the FDA or other foreign regulatory authorities to timely review and process our regulatory submissions, which could have a material adverse effect on our business.

While we expect the COVID-19 pandemic to continue to adversely affect our business operations, the extent of the impact on our clinical development and regulatory efforts and the ~~suspension~~value of ~~further research~~ and ~~development activities~~market for our common stock will depend on future developments that are highly uncertain and cannot be predicted with confidence at this time, such as the ~~subsequent declines~~ultimate duration of the ~~price of its common stock, three putative class actions were filed~~pandemic, travel restrictions, quarantines, social distancing and business closure requirements in the U.S. ~~District Court for~~and in other countries, and the ~~Southern District~~effectiveness of ~~California against Celladon~~actions taken globally to contain and ~~certain~~treat COVID-19. In addition, to the extent the ongoing COVID-19 pandemic adversely affects our business and results of ~~its current and former officers. The complaints generally alleged that~~operations, it may also have the ~~defendants violated Sections 10(b) and 20(a)~~effect of heightening many of the ~~Securities Exchange Act of 1934, as amended, or the Exchange Act, by making materially false~~other risks and misleading statements regarding the clinical trial program for MYDICAR, thereby artificially inflating the price of Celladon’s common stock. The complaints sought unspecified monetary damages and other relief, including attorneys’ fees. On December 9, 2015, the district court consolidated the three putative securities class actions and appointed a lead plaintiff to represent the putative class. The lead plaintiff filed a consolidated amended complaint on February 29, 2016.

On October 7, 2016, the district court granted defendants’ motion to dismiss the consolidated amended complaint and granted leave to amend within 60 days from the date of the district court’s order. The lead plaintiff subsequently filed a notice of intent not to amend the consolidated amended complaint and instead indicated that it intended to appeal the district court’s decision. On December 9, 2016, the district court closed the case.

On December 28, 2016, the lead plaintiff filed a notice to the United States Court of Appeals for the Ninth Circuit appealing the district court’s order dismissing the consolidated amended complaint. On May 5, 2017, lead plaintiff filed his opening brief. On July 5, 2017, defendants filed their answering appellate brief.

It is possible that additional suits will be filed, or allegations made by stockholders, with respect to these same or other matters and also naming us and/or our former officers and directors as defendants. We believe that we have meritorious defenses and intend to defend these lawsuits vigorously. Due to the early stage of these proceedings, we are not able to predict or reasonably estimate the ultimate outcome or possible losses relating to these claims. While we and Celladon’s former directors and officers have a separate liability insurance policy dedicated to any claims that may arise from premerger events, there is no assurance that the coverage will be sufficient. In addition, any such litigation could resultuncertainties described elsewhere in ~~substantial costs and a diversion of our management’s attention and resources, which could harm our business.~~

this ‘‘Risk Factors’’ section.

Risks Related to Ownership of our Common Stock

The market price of our common stock has been and may continue to be highly volatile, and you may not be able to resell some or all of your shares at a desired market price.

The market price of our common stock has been and is likely to continue to be volatile. Our stock price could be subject to wide fluctuations in response to a variety of factors, including the following:

•results or delays in preclinical studies or clinical trials;

•our decision to initiate a clinical trial, not to initiate a clinical trial or to terminate an existing clinical trial;

•unanticipated serious safety concerns related to the use of any of our product candidates;

•reports of adverse events in other gene therapy products or clinical trials of such products;

•inability to obtain additional funding;

•any delay in filing an IND, NDA, BLA, or ~~NDA~~MAA for any of our product candidates and any adverse development or perceived adverse development with respect to the FDA’s review of that IND, NDA, or ~~NDA;~~

BLA(or the EMA’s review of that MAA);

•our ability to obtain regulatory approvals for our product candidates, and delays or failures to obtain such approvals;

•failure of any of our product candidates, if approved, to achieve commercial success;

•failure to obtain ~~orphan drug~~or maintain Orphan Drug designation;

•failure to maintain our existing third-party license and supply agreements;

•failure by our licensors to prosecute, maintain, or enforce our intellectual property rights;

•changes in laws or regulations applicable to our product candidates;

•any inability to obtain adequate supply of our product candidates or the inability to do so at acceptable prices;

•adverse regulatory authority decisions;

•

~~adverse regulatory authority decisions;~~

•introduction of new products, services, or technologies by our competitors;

•failure to meet or exceed financial and development projections we may provide to the public;

•failure to meet or exceed the financial and development projections of the investment community;

•the perception of the pharmaceutical industry by the public, legislatures, regulators, and the investment community;

•announcements of significant acquisitions, strategic partnerships, joint ventures, or capital commitments by us or our competitors;

•disputes or other developments relating to proprietary rights, including patents, litigation matters, and our ability to obtain patent protection for our technologies;

•additions or departures of key personnel;

•significant lawsuits, including patent or stockholder litigation;

•if securities or industry analysts do not publish research or reports about our business, or if they issue an adverse or misleading opinions regarding our business and stock;

•changes in the market valuations of similar companies;

•general market or macroeconomic conditions;

•sales of our common stock by us or our stockholders in the future;

•trading volume of our common stock;

•announcements by commercial partners or competitors of new commercial products, clinical progress or the lack thereof, significant contracts, commercial relationships or capital commitments;

•adverse publicity relating to the hepatitis market generally, including with respect to other products and potential products in such markets;

•the introduction of technological innovations or new therapies that compete with potential products of ours;

•changes in the structure of health care payment systems; and

•period-to-period fluctuations in our financial results.

Moreover, the stock markets in general have experienced substantial volatility that has often been unrelated to the operating performance of individual ~~companies.~~companies, including very recently in connection with the ongoing COVID-19 pandemic, which has resulted in decreased stock prices for many companies notwithstanding the lack of a fundamental change in their underlying business models or prospects. These broad market ~~fluctuations~~and industry factors, including potentially worsening economic conditions and other adverse effects or developments relating to the ongoing COVID-19 pandemic, may also adversely affect the trading price of our common stock.

In the past, following periods of volatility in the market price of a company’s securities, stockholders have often instituted class action securities litigation against those companies. Such litigation, if instituted, could result in substantial costs and diversion of management attention and resources, which could significantly harm our profitability and reputation.

We will incur costs and demands upon management as a result of complying with the laws and regulations affecting public companies.

We have incurred and will continue to incur significant legal, accounting and other expenses associated with public company reporting requirements. We also incur costs associated with corporate governance requirements, including requirements under the Sarbanes-Oxley Act, as well as rules implemented by the SEC and The ~~NASDAQ~~Nasdaq Stock Market LLC. These rules and regulations impose significant legal and financial compliance costs and make some activities more time-consuming and costly. For example, our management team consists of certain executive officers who have not previously managed and operated a public company. These executive officers and other personnel will need to devote substantial time to gaining expertise regarding operations as a public company and compliance with applicable laws and regulations. In addition, it may be more difficult for us to attract and retain qualified individuals to serve on our board of directors or as executive officers, which may adversely affect investor confidence and could cause our business or stock price to suffer.

Anti-takeover provisions in our charter documents and under Delaware law could make an acquisition of us more difficult and may prevent attempts by our stockholders to replace or remove our management.

Provisions in our certificate of incorporation and bylaws may delay or prevent an acquisition or a change in management. These provisions include a classified board of directors, a prohibition on actions by written consent of our stockholders and the ability of the board of directors to issue preferred stock without stockholder approval. In addition, because we are incorporated in Delaware, we are governed by the provisions of Section 203 of the ~~DGCL,~~Delaware General Corporation Law, which prohibits stockholders owning in excess of 15% of our voting stock from merging or combining with us. Although we believe these provisions collectively will provide for an opportunity to receive higher bids by requiring potential acquirers to negotiate with our board of directors, they would apply even if the offer may be considered beneficial by some stockholders. In addition, these provisions may frustrate or prevent any attempts by our stockholders to replace or remove then current management by making it more difficult for stockholders to replace members of the board of directors, which is responsible for appointing the members of management.

We do not anticipate that we will pay any cash dividends in the foreseeable future.

We expect to retain our future earnings to fund the development and growth of our business. As a result, capital appreciation, if any, of our common stock will be the sole source of gain for our stockholders, if any, for the foreseeable future.

Future sales of shares by existing stockholders could cause our stock price to decline.

If existing stockholders sell, or indicate an intention to sell, substantial amounts of our common stock in the public market after legal restrictions on resale lapse, the trading price of our common stock could decline. Certain of our existing stockholders, including ~~Vivo Ventures Fund VI, L.P.~~Columbia Threadneedle Investments, 683 Capital Management, BlackRock Institutional Trust, and ~~Interwest Partners X, L.P.,~~The Vanguard Group, and their respective affiliated entities, own substantial ownership interest in our common stock and any decision to sell a significant number of shares may negatively impact the price of our common stock.

The ownership of our common stock is highly concentrated, and it may prevent stockholders from influencing significant corporate decisions and may result in conflicts of interest that could cause our stock price to decline.

Our executive officers, directors and 5% stockholders and their affiliates beneficially own or control a significant portion of the outstanding shares of our common stock. Accordingly, these executive officers, directors, 5% stockholders and their affiliates, acting as a group, have substantial influence over the outcome of corporate actions requiring stockholder approval, including the election of directors, any merger, consolidation or sale of all or substantially all of our assets or any other significant corporate transactions. These stockholders may also delay or prevent a change of control of us, even if such a change of control would benefit our other stockholders. The significant concentration of stock ownership may adversely affect the trading price of our common stock due to investors’ perception that conflicts of interest may exist or arise.

~~Because the Merger resulted in an ownership change under Section 382 of the Internal Revenue Code our~~

Our net operating loss carryforwards and certain other tax attributes are now subject to limitations.

Our federal and state net operating loss (NOL) carry-forwards will begin to expire, if not utilized, beginning in 2030 for federal income tax purposes and 2028 for California state income tax purposes. These NOL carry-forwards could expire unused and be unavailable to offset future income tax liabilities. Under legislation enacted in 2017, informally titled the Tax Cuts and Jobs Act, as modified by the Coronavirus Aid, Relief, and Economic Security Act (CARES Act), U.S. federal net operating loss carryforwards generated in tax years beginning after December 31, 2017 may be carried forward indefinitely but, in the case of tax years beginning after December 31, 2020, may only be used to offset 80% of taxable income annually. In addition, California has enacted A.B. 85 which imposed limits on the usability of California state net operating losses and certain tax credits in tax years beginning after 2019 and before 2023. Such limitations could result in the expiration of portions of our net operating loss and tax credit carryforwards before utilization. On February 9, 2022, Senate Bill No. 113 was signed into California law and reinstates the net operating loss deduction, and removes the above-described temporary limitation on allowable credits, for taxable years beginning on or after January 1, 2022. Moreover, if a corporation undergoes an ~~“ownership change”~~ownership change within the meaning of Section 382 of the ~~Internal Revenue~~ Code ~~of 1986, as amended, or Section 382,~~(Section 382) the corporation’s ~~net operating loss~~NOL carryforwards and certain other tax attributes arising from before the ownership change are subject to limitations on use after the ~~ownership~~“ownership change.” In general, an ownership change occurs if there is a cumulative change in the corporation’s equity ownership by certain stockholders that exceeds fifty percentage points over a rolling three-year period. Similar rules may apply under state tax laws. ~~The recent Merger~~Our merger with Celladon resulted in such an ownership change and, accordingly, ~~both parties’ net operating loss~~Celladon’s NOL carryforwards and certain other tax attributes will be subject to further limitations on their use. WeIn addition, we assessed whether Eiger had an ownership change, as defined by Section 382 of the Code, as a result of the Merger and other stock issuances that occurred from our formation through December 31, ~~2016.~~2020. Based upon this assessment, ~~no reduction was~~we have experienced ownership changes on April 20, 2016, October 18, 2018 and December 31, 2020. Due to these ownership changes, reductions were made to ~~the federal~~our NOL and ~~state NOL carryforwards or federal and state~~ tax credit carryforwards under these rules. Additional ownership changes in the future could result in additional limitations on ~~the combined organization’s~~our net operating loss and tax credit carryforwards. Consequently, even if we achieve profitability, we may not be able to utilize a material portion of our ~~net operating loss~~NOL carryforwards and other tax attributes, which could have a material adverse effect on cash flow and results of operations.

A full valuation allowance has been provided for the entire amount of our remaining net operating loss.

~~ITEM 2.~~

ITEM 2. UNREGISTERED SALES OF EQUITY SECURITIES AND USE OF PROCEEDS

~~None~~

None.

~~ITEM 3.~~

ITEM 3. DEFAULTS UPON SENIOR SECURITIES

~~None.~~

~~ITEM 4.~~

~~MINE SAFETY DISCLOSURES~~

None.

ITEM 4. MINE SAFETY DISCLOSURES

Not applicable.

~~ITEM 5.~~

ITEM 5. OTHER INFORMATION

None.

ITEM 6. EXHIBITS

~~ITEM 6.~~

E~~XHIBITS~~

Exhibit Number						Description of Document

3.1						Amended and Restated Certificate of Incorporation of Celladon Corporation (incorporated by reference to Exhibit 3.1 to the Current Report on Form 8-K of Celladon Corporation, filed with the SEC on February 10, 2014).

3.2						Amended and Restated Bylaws of Celladon Corporation (incorporated by reference to Exhibit 3.2 to the Current Report on Form 8-K, filed with the SEC on February 10, 2014).

3.3						Agreement and Plan of Merger and Reorganization, dated as of November 18, 2015, by and among Celladon Corporation, Celladon Merger Sub, Inc., and Eiger BioPharmaceuticals, Inc. (incorporated by reference to Exhibit 2.1 to the Current Report on Form 8-K filed with the SEC on November 19, 2015).

3.4						Certificate of Amendment of Amended and Restated Certificate of Incorporation of Celladon Corporation (incorporated by reference to Annex D to the Registration Statement on Form S-4, as amended (File No. 333-208521), originally filed with the SEC on December 14, 2015).

~~3.4~~ 3.5						Certificate of Amendment of Amended and Restated Certificate of Incorporation of Celladon Corporation (incorporated by reference to Annex E to the Registration Statement on Form S-4, as amended (File No. 333-208521), originally filed with the SEC on December 14, 2015).

~~31.1~~ 10.1						Separation Agreement and General Rel e ase, dated as of February 6, 2023, by and between Eiger BioPharmaceuticals, Inc. and David A. Cory (incorporated by reference to Exhibit 10.41 to the Annual Report on Form 10-K (File No. 001-36183) filed with the SEC on March 17, 2023).

10.2						Offer Letter Agreement, dated as January 9, 2023, by and between Eiger BioPharmaceuticals, Inc. and David Apelian (incorporated by reference to Exhibit 10.42 to the Annual Report on Form 10-K (File No. 001-36183) filed with the SEC on March 17, 2023).

10.3						Second Amendment to Lease, dated February 16, 2023, by and between Eiger BioPharmaceuticals, Inc. and the McDonald Family Co. LLC. (incorporated by reference to Exhibit 10.43 to the Annual Report on Form 10-K (File No. 001-36183) filed with the SEC on March 17, 2023).

31.1**						Certification of Principal Executive Officer required by Rule 13a-14(a) or Rule 15d-14(a).

~~31.2~~ 31.2**						Certification of Principal Financial Officer required by Rule 13a-14(a) or Rule 15d-14(a).

32.1+						~~Certification~~Certifications of Principal Executive Officer and Principal Financial Officer required by Rule 13a-14(b) or Rule 15d-14(b) and Section 1350 of Chapter 63 of Title 18 of the United States Code (18 U.S.C. §1350).

~~101.INS~~ 101.INS**						Inline XBRL Instance ~~Document~~ Document- the instance document does not appear in the Interactive Data File because its XBRL tags are embedded within the Inline XBRL document.

~~101.SCH~~ 101.SCH**						Inline XBRL Taxonomy Extension Schema Document

~~101.CAL~~ 101.CAL**						Inline XBRL Taxonomy Extension Calculation Linkbase Document

~~101.DEF~~ 101.DEF**						Inline XBRL Taxonomy Extension Definition Linkbase Document

~~101.LAB~~ 101.LAB**						Inline XBRL Taxonomy Extension Label Linkbase Document

~~101.PRE~~ 101.PRE**						Inline XBRL Taxonomy Extension Presentation Linkbase Document

104						The cover page from the Company’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2023, has been formatted in Inline XBRL.

+	This certification accompanies the Quarterly Report pursuant to Section 906 of the Sarbanes-Oxley Act of 2002 and shall not be deemed “filed” by the Registrant for purposes of Section 18 of the Securities Exchange Act of 1934, as amended.

__________________________

+ This certification accompanies the Quarterly Report pursuant to Section 906 of the Sarbanes-Oxley Act of 2002 and shall not be deemed “filed” by the Registrant for purposes of Section 18 of the Securities Exchange Act of 1934, as amended.

~~SIGNATURES~~

** Filed herewith.

SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this ~~Quarterly Report on Form 10-Q~~report to be signed on its behalf by the undersigned thereunto duly authorized.


	Eiger BioPharmaceuticals, Inc.

Date: ~~November 9, 2017~~ May 11, 2023	By:	~~By:~~		/s/ David ~~A. Cory~~ Apelian
				David ~~A. Cory~~ Apelian
				Director, ~~President and~~Interim Chief Executive Officer
				(Principal Executive Officer)

			Eiger BioPharmaceuticals, Inc.

Date: ~~November 9, 2017~~ May 11, 2023	By:	~~By:~~		/s/ ~~James Welch~~ William G. Kachioff
				~~James Welch~~ William G. Kachioff
				Chief Financial Officer
				(Principal Financial Officer)

	Shares Available for Grant			Number of Options			Weighted- Average Exercise Price		Weighted- Average Remaining Contractual Life (in Years)		Aggregate Intrinsic Value
Outstanding as of December 31, 2016		646,778			1,212,044		$	14.06			$	2,414
Additional options authorized		417,883
Granted		(652,200	)		652,200		$	10.91
Exercised					(5,688	)	$	2.06
Canceled and forfeited		328,162			(328,162	)	$	13.89
Outstanding as of September 30, 2017		740,623			1,530,394		$	12.80		8.31	$	2,529
Vested and exercisable as of September 30, 2017					601,330		$	14.28		7.32	$	1,286

	Payments due by period
	Total		Less than 1 year		1 – 3 Years		3 – 5 Years		More than 5 years
Operating lease obligations (1)	$	401	$	330	$	71	$	—	$	—
Term loan debt (2)	$	15,000	$	—	$	12,083	$	2,917	$	—
Interest on term loan debt (3)	$	4,378	$	979	$	2,205	$	1,194	$	—
Total	$	19,779	$	1,309	$	14,359	$	4,111	$	—

(State or Other Jurisdiction of

Incorporation or Organization)

(I.R.S. Employer

Identification No.)

~~350 Cambridge Ave, Suite 350~~
2155 Park Boulevard
Palo Alto, CA

(Address of Principal Executive Offices)

(Zip Code)