UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

WASHINGTON, D.C. 20549

FORM 10-Q

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

FOR THE QUARTERLY PERIOD ENDED SEPTEMBER 30, ~~2022~~2023

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

For the transition period from to

COMMISSION FILE NUMBER 001-36576

MARINUS PHARMACEUTICALS, INC.

(Exact name of registrant as specified in its charter)


Delaware		20-0198082
(State or other jurisdiction of incorporation or organization)		(I.R.S. Employer Identification No.)

5 Radnor Corporate Center, Suite 500

100 Matsonford Rd

Radnor, PA 19087

(Address of registrant’s principal executive offices, including zip code)

Registrant’s telephone number, including area code: (484) 801-4670

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	MRNS	Nasdaq Global Market

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☐ No.

Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted 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 such files). ☒ Yes ☐ ~~No.~~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.


Large accelerated filer ☐	Accelerated filer	☐

Non-accelerated filer ☒	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 ☒ ~~No.~~No

The number of outstanding shares of the registrant’s common stock, par value $0.001 per share, as of ~~October 31, 2022,~~November 3, 2023, was: ~~37,196,644.~~54,573,581.

Table of Contents

MARINUS PHARMACEUTICALS, INC. AND SUBSIDIARY

INDEX TO FORM 10-Q

FOR THE QUARTER ENDED SEPTEMBER 30, ~~2022~~2023

PART I – FINANCIAL INFORMATION
Item 1.	Consolidated Financial Statements (unaudited)
	Consolidated Balance Sheets as of September 30, ~~2022~~2023 and December 31, 20212	3
	Consolidated Statements of Operations and Comprehensive (Loss) Income ~~(Loss)~~ for the three and nine months ended September 30, ~~2022~~2023 and ~~2021~~2022	4
	Consolidated Statements of Cash Flows for the nine months ended September 30, ~~2022~~2023 and ~~2021~~2022	5
	Consolidated Statements of Stockholders’ Equity for the three and nine months ended September 30, ~~2022~~2023 and ~~2021~~2022	6
	Notes to Consolidated Financial Statements	7
Item 2.	Management’s Discussion and Analysis of Financial Condition and Results of Operations	2326
Item 3.	Quantitative and Qualitative Disclosures About Market Risk	4452
Item 4.	Controls and Procedures	4552

PART II – OTHER INFORMATION
Item 1.	Legal Proceedings	4653
Item 1A.	Risk Factors	4653
Item 2.	Unregistered Sales of Equity Securities and Use of Proceeds	5154
Item 3.	Defaults Upon Senior Securities	5154
Item 4.	Mine Safety Disclosures	5154
Item 5.	Other Information	5154
Item 6.	Exhibits	5255
	Signatures	5356

Unless the context otherwise requires, all references in this Quarterly Report on Form 10-Q to the “Company,” “Marinus,” “we,” “us,” and “our” include Marinus Pharmaceuticals, Inc. and its wholly owned subsidiary, Marinus Pharmaceuticals Emerald Limited, an Ireland company.

Table of Contents

PART I

FINANCIAL INFORMATION

Item 1. Consolidated Financial Statements

MARINUS PHARMACEUTICALS, INC. AND SUBSIDIARY

CONSOLIDATED BALANCE SHEETS

(in thousands, except share and per share amounts)

(unaudited)

September 30,

December 31,

2022

2021

ASSETS







Current assets:

Cash and cash equivalents

$
168,249

$
122,927

Accounts receivable, net

3,398

2,629

Inventory

104

—

Contract asset

—

557

Prepaid expenses and other current assets

5,255

5,565

Total current assets

177,006

131,678

Property and equipment, net

5,520

2,499

Other assets

2,315

2,663

Total assets

$
184,841

$
136,840

LIABILITIES AND STOCKHOLDERS’ EQUITY

Current liabilities:

Accounts payable

$
1,622

$
3,126

Refund liability

—

21,233

Accrued expenses

19,428

16,207

Total current liabilities

21,050

40,566

Notes payable, net of deferred financing costs

70,780

40,809

Contract liability

9,499

—

Other long-term liabilities

1,508

1,979

Total liabilities

102,837

83,354

Stockholders’ equity:

Series A convertible preferred stock, $0.001 par value; 25,000,000 shares authorized, 4,300 shares issued and outstanding at September 30, 2022 and 4,575 shares issued and outstanding at December 31, 2021

4,043

4,302

Common stock, $0.001 par value; 150,000,000 shares authorized, 37,203,951 issued and 37,196,644 outstanding at September 30, 2022 and 36,797,561 issued and 36,790,254 outstanding at December 31, 2021

37

37

Additional paid-in capital

474,133

459,852

Treasury stock at cost, 7,307 shares at September 30, 2022 and December 31, 2021

—

—

Accumulated deficit

(396,209)

(410,705)

Total stockholders’ equity

82,004

53,486

Total liabilities and stockholders’ equity

$
184,841

$
136,840



	September 30,		December 31,
	2023		2022
ASSETS
Current assets:
Cash and cash equivalents	$	140,437	$	240,551
Short-term investments		35,919		—
Accounts receivable, net		4,281		6,348
Inventory		2,736		77
Prepaid expenses and other current assets		11,667		5,402
Total current assets		195,040		252,378
Property and equipment, net		3,909		4,236
Other assets		1,857		2,904
Total assets	$	200,806	$	259,518
LIABILITIES AND STOCKHOLDERS’ EQUITY
Current liabilities:
Accounts payable	$	2,626	$	4,461
Current portion of notes payable		7,700		—
Current portion of revenue interest financing payable		1,901		1,020
Accrued expenses		18,328		19,536
Total current liabilities		30,555		25,017
Notes payable, net of deferred financing costs		64,783		71,018
Revenue interest financing payable, net of deferred financing costs		32,855		29,857
Contract liabilities, net		17,105		16,285
Other long-term liabilities		971		1,341
Total liabilities		146,269		143,518
Stockholders’ equity:
Series A convertible preferred stock, $0.001 par value; 25,000,000 shares authorized, no shares issued and outstanding at September 30, 2023; 4,300 shares issued and outstanding at December 31, 2022		—		4,043
Common stock, $0.001 par value; 150,000,000 shares authorized, 54,580,797 issued and 54,573,490 outstanding at September 30, 2023 and 49,650,074 issued and 49,642,767 outstanding at December 31, 2022		55		50
Additional paid-in capital		584,710		542,428
Treasury stock at cost, 7,307 shares at September 30, 2023 and December 31, 2022		—		—
Accumulated other comprehensive loss		(71)		—
Accumulated deficit		(530,157)		(430,521)
Total stockholders’ equity		54,537		116,000
Total liabilities and stockholders’ equity	$	200,806	$	259,518

See accompanying notes to consolidated financial statements.

Table of Contents

MARINUS PHARMACEUTICALS, INC. AND SUBSIDIARY

CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE (LOSS) INCOME ~~(LOSS)~~

(in thousands, except share and per share amounts)

(unaudited)

Three Months Ended September 30,

Nine Months Ended September 30,


2022

2021

2022

2021

Revenue:



Product revenue, net

$
555

$
—

$
555

$
—

Federal contract revenue

1,785

1,127

5,088

4,838

Collaboration revenue


—


8,987

12,673


8,987

Total revenue

2,340

10,114

18,316

13,825

Expenses:

Research and development

19,002

18,353

58,488

55,506

Selling, general and administrative

13,389

9,452

42,187

26,656

Cost of product revenue

48

—

48

—

Cost of collaboration revenue

—

1,478

—

1,478

Cost of IP license fee

—

—

1,169

—

Total expenses

32,439

29,283

101,892

83,640

Loss from operations

(30,099)

(19,169)

(83,576)

(69,815)

Interest income

514

17

610

57

Interest expense

(2,634)

(678)

(6,982)

(1,029)

Gain from sale of priority review voucher, net

107,375

—

107,375

—

Other (expense) income, net

(114)

323

(1,179)

316

Income (loss) before income taxes

75,042

(19,507)

16,248

(70,471)

Provision for income taxes

(1,752)

—

(1,752)

—

Net income (loss) and comprehensive income (loss)

$
73,290

$
(19,507)

$
14,496

$
(70,471)

Net income allocated to preferred shareholders

1,656

—

336

—

Net income (loss) applicable to common shareholders

$
71,634

$
(19,507)

$
14,160

$
(70,471)

Per share information:

Net income (loss) per share of common stock—basic

$
1.93

$
(0.53)

$
0.38

$
(1.92)

Net income (loss) per share of common stock—diluted

$
1.89

$
(0.53)

$
0.37

$
(1.92)

Basic weighted average shares outstanding

37,202,269

36,744,591

37,084,060

36,667,472

Diluted weighted average shares outstanding

37,910,511

36,744,591

38,393,754

36,667,472


	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022

Revenue:
Product revenue, net	$	5,429	$	555	$	13,010	$	555
Federal contract revenue		1,891		1,785		10,753		5,088
Collaboration revenue		18		—		36		12,673
Total revenue		7,338		2,340		23,799		18,316

Expenses:
Research and development		23,661		19,002		73,006		58,488
Selling, general and administrative		14,868		13,389		45,794		42,187
Cost of product revenue		455		48		1,047		48
Cost of IP license fee		—		—		—		1,169
Total expenses		38,984		32,439		119,847		101,892
Loss from operations		(31,646)		(30,099)		(96,048)		(83,576)
Interest income		1,895		514		6,366		610
Interest expense		(4,242)		(2,634)		(12,597)		(6,982)
Gain from sale of priority review voucher, net		—		107,375		—		107,375
Other income (expense), net		1,021		(114)		1,105		(1,179)
(Loss) income before income taxes		(32,972)		75,042		(101,174)		16,248

(Provision) benefit for income taxes		—		(1,752)		1,538		(1,752)
Net (loss) income	$	(32,972)	$	73,290	$	(99,636)	$	14,496

Net income allocated to preferred shareholders		—		1,656		—		336
Net (loss) income applicable to common shareholders	$	(32,972)	$	71,634	$	(99,636)	$	14,160

Per share information:
Net (loss) income per share of common stock—basic	$	(0.61)	$	1.93	$	(1.89)	$	0.38
Net (loss) income per share of common stock—diluted	$	(0.61)	$	1.89	$	(1.89)	$	0.37
Basic weighted average shares outstanding		53,920,109		37,202,269		52,755,114		37,084,060
Diluted weighted average shares outstanding		53,920,109		37,910,511		52,755,114		38,393,754

Other comprehensive income (loss):
Unrealized gain (loss) on available-for-sale securities		43		—		(71)		—
Total comprehensive (loss) income	$	(32,929)	$	73,290	$	(99,707)	$	14,496

See accompanying notes to consolidated financial statements.

Table of Contents

MARINUS PHARMACEUTICALS, INC. AND SUBSIDIARY

CONSOLIDATED STATEMENTS OF CASH FLOWS

(in thousands)

(unaudited)

Nine Months Ended September 30,

2022

2021

Cash flows from operating activities







Net income (loss)

$
14,496

$
(70,471)

Adjustments to reconcile net loss to net cash used in operating activities:

Gain from sale of PRV, net of transaction costs

(107,375)

—

Depreciation and amortization

336

262

Amortization of debt issuance costs

1,133

319

Stock-based compensation expense

11,091

10,867

Amortization of net contract asset/liability

(1,000)

(41)

Noncash lease expense

194

232

Noncash lease liability

252

237

Write off of fixed assets

169

—

Issuance of common stock for cost of license agreement

1,169

—

Unrealized loss on foreign currency transactions

930

—

Changes in operating assets and liabilities:

Refund liability

(22,163)

21,828

Net contract asset/liability

11,057

(1,018)

Prepaid expenses and other current assets, non-current assets, inventory and accounts receivable

(2,313)

(453)

Accounts payable, accrued expenses and other long term-liabilities

1,052

4,511

Net cash used in operating activities

(90,972)

(33,727)

Cash flows from investing activities

Proceeds from sale of PRV, net of transaction costs

107,375

—

Maturities of short-term investments

—

1,474

Deposit on property and equipment

—

(1,329)

Purchases of property and equipment

(1,682)

(839)

Net cash provided by (used in) investing activities

105,693

(694)

Cash flows from financing activities

Proceeds from exercise of stock options

1,763

902

Proceeds from notes payable, net of upfront fee

28,838

40,259

Financing costs, paid

—

(148)

Net cash provided by financing activities

30,601

41,013

Net increase in cash and cash equivalents

45,322

6,592

Cash and cash equivalents—beginning of period

122,927

138,509

Cash and cash equivalents—end of period

$
168,249

$
145,101

Supplemental disclosure of cash flow information

Debt issuance costs included in accrued expenses

$
—

$
900

Property and equipment in accrued expenses

$
—

$
107

Property and equipment in deposits placed in service

$
1,665

$
—


	Nine Months Ended September 30,
	2023		2022
Cash flows from operating activities
Net (loss) income	$	(99,636)	$	14,496
Adjustments to reconcile net (loss) income to net cash used in operating activities:
Gain from sale of PRV, net of transaction costs		—		(107,375)
Depreciation and amortization		426		336
Amortization of debt issuance costs		1,659		1,133
Accretion of revenue interest financing debt		4,421		—
Amortization of discount on short-term investments		(995)		—
Stock-based compensation expense		11,638		11,091
Amortization of net contract asset/liability		(1,259)		(1,000)
Noncash lease expense		146		194
Noncash lease liability		300		252
Write off of fixed assets		61		169
Issuance of common stock for cost of license agreement		—		1,169
Unrealized loss on foreign currency transactions		—		930
Changes in operating assets and liabilities:
Refund liability		—		(22,163)
Net contract asset/liability		2,079		11,057
Prepaid expenses and other current assets, non-current assets, inventory and accounts receivable		(6,290)		(2,313)
Accounts payable and accrued expenses		(3,559)		1,052
Net cash used in operating activities		(91,009)		(90,972)
Cash flows from investing activities
Proceeds from sale of PRV, net of transaction costs		—		107,375
Proceeds from sale of property and equipment		9		—
Maturities of short-term investments		17,000		—
Purchases of short-term investments		(51,995)		—
Purchases of property and equipment		(85)		(1,682)
Net cash (used in) provided by investing activities		(35,071)		105,693
Cash flows from financing activities
Proceeds from exercise of stock options		783		1,763
Proceeds from notes payable, net of fees		—		28,838
Payments of revenue interesting financing debt		(737)		—
Proceeds from equity offerings, net of offering costs		25,920		—
Net cash provided by financing activities		25,966		30,601
Net (decrease) increase in cash and cash equivalents		(100,114)		45,322
Cash and cash equivalents—beginning of period		240,551		122,927
Cash and cash equivalents—end of period	$	140,437	$	168,249
Supplemental disclosure of cash flow information
Unrealized loss on short-term investments	$	(71)	$	—
Financing costs	$	97	$	—
Cash paid for interest during the period	$	6,540	$	—
Cash paid for income taxes during the period	$	903	$	—
Property and equipment in deposits placed in service	$	—	$	1,665

See accompanying notes to consolidated financial statements.

Table of Contents

MARINUS PHARMACEUTICALS, INC. AND SUBSIDIARY

CONSOLIDATED STATEMENTS OF ~~STOCKHOLDERS’~~ EQUITY

(in ~~thousands, except share amounts)~~thousands)

(unaudited)

Series A

Additional

Total

Convertible Preferred Stock

Common Stock

Paid-in

Treasury Stock

Accumulated

Stockholders’


Shares

Amount

Shares

Amount

Capital

Shares

Amount

Deficit

Equity

Balance, December 31, 2020

4,753

$
4,469

36,578,460

$
37

$
444,622

7,307

$
—

$
(311,929)

$
137,199

Stock-based compensation expense

—

—

—

—

5,035

—

—

—

5,035

Exercise of stock options

—

—

55,030

—

244

—

—

—

244

Financing costs

—

—

—

—

(3)

—

—

—

(3)

Net Loss

—

—

—

—

—

—

—

(27,141)

(27,141)

Balance, March 31, 2021

4,753

$
4,469

36,633,490

$
37

$
449,898

7,307

$
—

$
(339,070)

$
115,334

Stock-based compensation expense

—

—

—

—

2,991

—

—

—

2,991

Exercise of stock options

—

—

63,312

—

557

—

—

—

557

Conversion of convertible preferred stock into common

(178)

(167)

35,600

—

167

—

—

—

—

Net loss

—

—

—

—

—

—

—

(23,823)

(23,823)

Balance, June 30, 2021

4,575

$
4,302

36,732,402

$
37

$
453,613

7,307

$
—

$
(362,893)

$
95,059

Stock-based compensation expense

—

—

—

—

2,841

—

—

—

2,841

Exercise of stock options

—

—

18,799

—

101

—

—

—

101

Net loss

—

—

—

—

—

—

—

(19,507)

(19,507)

Balance, September 30, 2021

4,575

$
4,302

36,751,201

$
37

$
456,555

7,307

—

(382,400)

78,494

Balance, December 31, 2021

4,575

$
4,302

36,790,254

$
37

$
459,852

7,307

$
—

$
(410,705)

$
53,486

Stock-based compensation expense

—

—

—

—

3,379

—

—

—

3,379

Exercise of stock options

—

—

225,165

—

1,733

—

—

—

1,733

Issuance of stock related to IP license agreement with Ovid

—

—

123,255

—

1,168

—

—

—

1,168

Net Loss

—

—

—

—

—

—

—

(19,361)

(19,361)

Balance, March 31, 2022

4,575

$
4,302

37,138,674

$
37

$
466,132

7,307

$
—

$
(430,066)

$
40,405

Stock-based compensation expense

—

—

—

—

3,817

—

—

—

3,817

Issuance of common stock (vesting of restricted shares)

—

—

2,508

—

—

—

—

—

—

Exercise of stock options

—

—

2,968

—

14

—

—

—

14

Conversion of convertible preferred stock into common

(275)

(259)

55,000

—

259

—

—

—

—

Net loss

—

—

—

—

—

—

—

(39,433)

(39,433)

Balance, June 30, 2022

4,300

$
4,043

37,199,150

$
37

$
470,222

7,307

$
—

$
(469,499)

$
4,803

Stock-based compensation expense

—

—

—

—

3,895

—

—

—

3,895

Exercise of stock options

—

—

3,304

—

16

—

—

—

16

Net settlement of restricted shares

—

—

(5,810)

—

—

—

—

—

—

Net income

—

—

—

—

—

—

—

73,290

73,290

Balance, September 30, 2022

4,300

$
4,043

37,196,644

$
37

$
474,133

7,307

—

(396,209)

82,004


												Accumulated
	Series A						Additional					Other				Total
	Convertible Preferred Stock			Common Stock			Paid-in		Treasury Stock			Comprehensive		Accumulated		Stockholders’
	Shares	Amount		Shares	Amount		Capital		Shares	Amount		Income		Deficit		Equity
Balance, December 31, 2021	4,575	$	4,302	36,790,254	$	37	$	459,852	7,307	$	—	$	—	$	(410,705)	$	53,486
Stock-based compensation expense	—		—	—		—		3,378	—		—		—		—		3,378
Exercise of stock options	—		—	225,165		—		1,733	—		—		—		—		1,733
Issuance of stock related to IP license agreement with Ovid	—		—	123,255		—		1,169	—		—		—		—		1,169
Net Loss	—		—	—		—		—	—		—		—		(19,361)		(19,361)
Balance, March 31, 2022	4,575	$	4,302	37,138,674	$	37	$	466,132	7,307	$	—		—	$	(430,066)	$	40,405
Stock-based compensation expense	—		—	—		—		3,817	—		—		—		—		3,817
Net issuance of common stock in connection with the vesting of restricted stock	—		—	2,508		—		—	—		—		—		—		—
Exercise of stock options	—		—	2,968		—		14	—		—		—		—		14
Conversion of convertible preferred stock into common	(275)		(259)	55,000		—		259	—		—		—		—		—
Net loss	—		—	—		—		—	—		—		—		(39,433)		(39,433)
Balance, June 30, 2022	4,300	$	4,043	37,199,150	$	37	$	470,222	7,307	$	—		—	$	(469,499)	$	4,803
Stock-based compensation expense	—		—	—		—		3,895	—		—		—		—		3,895
Exercise of stock options	—		—	3,304		—		16	—		—		—		—		16
Net settlement of restricted shares	—		—	(5,810)		—		—	—		—		—		—		—
Conversion of convertible preferred stock into common	—		—	—		—		—	—		—		—		—		—
Net income	—		—	—		—		—	—		—		—		73,290		73,290
Balance, September 30, 2022	4,300	$	4,043	37,196,644	$	37	$	474,133	7,307		—	$	—		(396,209)		82,004

Balance, December 31, 2022	4,300	$	4,043	49,642,767	$	50	$	542,428	7,307	$	—	$	—	$	(430,521)	$	116,000
Stock-based compensation expense	—		—	—		—		3,741	—		—		—		—		3,741
Net issuance of common stock in connection with the vesting of restricted stock	—		—	22,350		—		—	—		—		—		—		—
Unrealized gain on short-term investments	—		—	—		—		—	—		—		74		—		74
Net loss	—		—	—		—		—	—		—		—		(34,730)		(34,730)
Balance, March 31, 2023	4,300	$	4,043	49,665,117	$	50	$	546,169	7,307	$	—	$	74	$	(465,251)	$	85,085
Stock-based compensation expense	—		—	—		—		3,891	—		—		—		—		3,891
Net issuance of common stock in connection with the vesting of restricted stock	—		—	11,625		—		—	—		—		—		—		—
Exercise of stock options	—		—	72,440		—		485	—		—		—		—		485
Conversion of convertible preferred stock into common	(4,300)		(4,043)	860,000		1		4,042	—		—		—		—		—
Unrealized loss on short-term investments	—		—	—		—		—	—		—		(188)		—		(188)
Net loss	—		—	—		—		—	—		—		—		(31,934)		(31,934)
Balance, June 30, 2023	—	$	—	50,609,182	$	51	$	554,587	7,307	$	—		(114)	$	(497,185)	$	57,339
Stock-based compensation expense	—		—	—		—		4,006	—		—		—		—		4,006
Exercise of stock options	—		—	50,338		—		298	—		—		—		—		298
Net issuance of common stock in connection with the vesting of restricted stock	—		—	224,170		—		—	—		—		—		—		—
Issuance of common stock in connection with at-the-market facility offering (average price of $7.17 per share), net of expenses of $529	—		—	3,689,800		4		25,819	—		—		—		—		25,823
Unrealized gain on short-term investments	—		—	—		—		—	—		—		43		—		43
Net loss	—		—	—		—		—	—		—		—		(32,972)		(32,972)
Balance, September 30, 2023	—	$	—	54,573,490	$	55	$	584,710	7,307		—	$	(71)		(530,157)		54,537

See accompanying notes to consolidated financial statements.

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MARINUS PHARMACEUTICALS, INC. AND SUBSIDIARY

NOTES TO CONSOLIDATED FINANCIAL STATEMENTS

1. Description of the Business and Liquidity

We are a commercial-stage pharmaceutical company dedicated to the development of innovative therapeutics for the treatment of seizure disorders, including rare genetic epilepsies and status epilepticus.On March 18, 2022, the ~~U.S~~U.S. Food and Drug Administration (FDA) approved our new drug application (NDA) for the use of ~~ZTALMY®~~ZTALMY^® (ganaxolone) oral suspension CV for the treatment of seizures associated with ~~cyclin-dependent kinase-like~~Cyclin-dependent Kinase-like 5 (CDKL5) ~~deficiency disorder~~Deficiency Disorder (CDD) in patients 2two years of age and older. In June 2022, the U.S. Drug Enforcement Administration (DEA) published an interim final rule in the Federal Register placing ganaxolone and its salts in schedule V (CV) of the Controlled Substances Act (CSA). , which rule became final on December 9, 2022. ZTALMY, our first FDA approved product, became available for commercial sale and shipment ~~to patients with a prescription in the U.S.~~ in the third quarter of 2022.On July 28, 2023, the European Commission (EC) granted marketing authorization for ZTALMY for the adjunctive treatment of epileptic seizures associated with CDD in patients two to 17 years of age. ZTALMY may be continued in patients 18 years of age and older. With the EC marketing authorization granted for ZTALMY, Orion Corporation (Orion), our commercialization partner for ZTALMY in Europe, has announced it has begun preparations for the launch of ZTALMY, including engaging in the required processes for obtaining pricing and reimbursement approval in the various European countries. The pricing and reimbursement process can be time consuming and may delay Orion’s commercial launch of ZTALMY in one or more European countries. We ~~also plan to develop~~are developing ganaxolone for the treatment of other rare genetic epilepsies, including ~~tuberous sclerosis complex~~Tuberous Sclerosis Complex (TSC), and for the treatment of refractory status epilepticus ~~(SE)~~(RSE). We are developing ganaxolone in formulations for two different routes of administration: intravenous (IV) and oral. The different formulations are intended to maximize potential therapeutic applications of ganaxolone for adult and pediatric patient populations, in both acute and chronic care, and for both acute hospital care and chronic at home-administration settings. While the precise mechanism by which ganaxolone exerts its therapeutic effects in the treatment of seizures is unknown, its anticonvulsant effects are thought to result from positive allosteric modulation of the gamma-aminobutyric acid type A (GABA_A) receptor in the central nervous system (CNS). Ganaxolone is a synthetic analog of allopregnanolone, an endogenous neurosteroid. Ganaxolone acts at both synaptic and extrasynaptic GABA_A receptors, a target known for its anti-seizure, antidepressant and anxiolytic potential.

~~The continued global spread of~~ COVID-19 ~~has impacted~~affected our clinical operations and timelines. For example, our ~~Phase 3~~ Randomized Therapy In Status Epilepticus ~~Trial (RAISE trial) in refractory status epilepticus (RSE)~~(RAISE) trial for RSE is conducted in hospitals, primarily intensive care units in academic medical centers, which ~~have~~ experienced high rates of COVID-19 admissions. Several of these sites participating in the RAISE trial ~~have~~ experienced COVID-related difficulties, including staff turnover and the need to devote significant resources to patients with COVID-19, which ~~has~~ resulted in site initiation and enrollment delays for the RAISE trial. Given these COVID-19-related challenges and ~~a temporary pause beginning~~the interruption in ~~February 2022 of the RAISE trial after routine monitoring of stability batches of clinical~~drug supply ~~material indicated that it became necessary to reduce the shelf life to less than the anticipated 24-months to meet product stability testing specifications,~~in mid-2022, we previously adjusted ~~the~~our expectation for our top-line data readout for the RAISE ~~trial to the second half of 2023.~~trial. In May 2022, we resumed screening and recruitment for the RAISE trial. ~~In addition,~~We now expect our ~~ganaxolone clinical trials~~interim analysis with top-line data readout for the RAISE trial to be available in the ~~outpatient setting may be negatively impacted~~second quarter of 2024, if ~~patients and their caregivers do not want to participate while~~ the ~~COVID-19 pandemic persists. The duration and severity of~~pre-defined stopping criteria from the ~~pandemic and its long-term impact on our business~~planned interim analysis are ~~uncertain at this time.~~met.

Liquidity

~~Other~~Since inception, other than for the three months ended September 30, 2022 ~~we have not generated any product revenues, and excluding the~~due to a one-time net gain from the sale of ~~the Rare Pediatric Disease (RPD)~~our Priority Review Voucher (PRV) ~~resulting in~~, we have incurred net ~~income~~losses and negative cash flows from our operations. We incurred a net loss of $99.6 million for the ~~three and~~ nine months ended September 30, ~~2022, we have incurred operating losses since inception.~~2023. There is no assurance that profitable operations will ~~ever~~ be achieved in the future, and if achieved, could be sustained on a continuing basis. In addition, development activities, clinical and preclinical testing, and commercialization of ganaxolone (in indications other than CDD in the U.S.) will require significant additional financing. Our accumulated deficit as of September 30, ~~2022~~2023 was ~~$396.2~~$530.2 million, and we expect to incur substantial losses in future periods. We plan to finance our future operations with a combination of proceeds from the issuance of equity securities, the issuance of debt, government funding, collaborations, licensing transactions and other commercial transactions or other sources, and revenues from ~~future~~ product ~~sales, if any.~~

In July 2022, we entered into an asset purchase agreement with Novo Nordisk, Inc. to sell the PRV awarded to us by the FDA as a result of the RPD Designation for ganaxolone for the treatment of CDD, pursuant to which Novo Nordisk, Inc. paid us $110.0 million upon the closing of the transaction in the third quarter of 2022.sales. We have not generated positive cash flows from operations, and there are no assurances that we will be ~~successful in obtaining an adequate level of financing for the commercialization and continued development of ganaxolone.~~

~~On July 30, 2021, we entered into a collaboration agreement (the Orion Collaboration Agreement) with Orion Corporation (Orion),~~ whereby Orion received exclusive rights to commercialize the oral and IV dose formulations of ganaxolone in the European Economic Area, United Kingdom and Switzerland in multiple seizure disorders, including CDD, TSC and RSE. Under the agreement, we received a €25 million ($29.6 million) upfront fee and are eligible to receive up to an additional €97 million in research and development reimbursement and cash milestone payments based on specific clinical and commercial achievements, as well as tiered royalty payments based on net sales ranging from the low double digits to the high teens for the oral programs and the low double-digits to the low twenties for the IV programs.

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On May 11, 2021 (Closing Date), we entered into a Credit Agreement and Guaranty (as amended by that certain letter agreement on May 17, 2021 and as further amended by that certain letter agreement on May 23, 2022, the Credit Agreement), and as further amended by that certain Limited Consent and First Amendment to Credit Agreement on October 28, 2022 (the Credit Agreement Amendment) with Oaktree Fund Administration, LLC, as administrative agent and the lenders party thereto that provides for a ~~five-year~~ ~~senior secured term loan facility~~successful in obtaining an ~~aggregate principal amount~~adequate level of ~~up to $100.0 million available to us in four~~ ~~tranches (collectively, the Term Loans). As of September 30, 2022, we have drawn a total of~~ ~~$75.0 million of the Term Loans pursuant to the Credit Agreement, with a remaining undrawn principal balance of $25.0 million, which is available through December 31, 2023 and is~~ ~~subject to the achievement of certain milestone events. Refer to Note 9. Notes Payable for additional information.~~

In September 2020, we entered into a contract (BARDA Contract) with the Biomedical Advanced Research and Development Authority (BARDA), a division of the U.S. Department of Health and Human Services’ Office of the Assistant Secretary for Preparedness and Response. Under the BARDA Contract, we received an award of up to an estimated $51 million for development of IV-administered ganaxolonefinancing for the ~~treatment~~continued development and commercialization of RSE. The BARDA Contract provides for funding to support, on a cost-sharing basis, the completion of a Phase 3 clinical trial of IV-administered ganaxolone in patients with RSE, which covers the RAISE trial, funding of pre-clinical studies to evaluate IV-administered ganaxolone as an effective treatment for RSE due to chemical nerve gas agent exposure, and funding of certain ganaxolone manufacturing scale-up and regulatory activities. In March 2022, we entered into an amendment with BARDA to extend the end date of our base performance period for funding under the BARDA Contract from September 1, 2022 to December 31, 2023. In September 2022, we entered into an amendment with BARDA that, among other things, (i) provides for the exercise of BARDA’s option under the BARDA Contract to support U.S. onshoring of the manufacturing capabilities for ganaxolone active pharmaceutical ingredient (API) (Option 2), (ii) changes the end of date of our performance period under Option 2 from December 31, 2026 to July 31, 2025, (iii) increases the government cost share amount under Option 2 from approximately $11.5 million to approximately $12.3 million, and (iv) increases our cost share amount under Option 2 from approximately $4.9 million to approximately $5.3 million.

~~The BARDA Contract consists of an approximately~~ ~~two-year~~ base period, and an extension period through December 31, 2023, during which BARDA will provide up to approximately $21 million of funding for the RAISE trial on a cost share basis and funding of additional preclinical studies of ganaxolone in nerve agent exposure models. Following successful completion of the RAISE trial and preclinical studies in the base period and extension period, the BARDA Contract provides for approximately $31 million of additional BARDA funding for three options in support of ganaxolone manufacturing, supply chain, clinical, regulatory and toxicology activities, including the $12.3 million exercise of Option 2 as described above. Under the BARDA Contract, we will be responsible for cost sharing in the amount of approximately $33 million and BARDA will be responsible for approximately $52 million, if all development options are completed. The contract period-of-performance (base and extension periods plus option exercises) is up to approximately five years.ganaxolone.

Management’s operating plan, which underlies the analysis of our ability to continue as a going concern, involves the estimation of the amount and timing of future cash inflows and outflows. Actual results could vary from the operating plan. We follow the provisions of Financial Accounting Standards Board (FASB) Accounting Standards Codification (ASC) Topic 205-40, Presentation of Financial Statements—Going Concern, which requires management to assess our ability to continue as a going concern within one year after the date the financial statements are issued. ~~For the year ended December 31, 2021, the three months ended March 31, 2022, and the three and six months ended June 30, 2022, we identified conditions and events~~We believe that ~~raised substantial doubt about~~ our ~~ability to continue as a going concern as our~~existing cash, ~~and~~ cash equivalents ~~for each~~and short-term investments as of ~~these periods were not~~September 30, 2023, will be sufficient to fund ~~operations~~our operating expenses and capital expenditure requirements, as well as maintain the minimum cash balance required under our debt facility, for the one-year period after ~~each of~~ the ~~dates such~~date the financial statements ~~were~~are issued. ~~In August 2022,~~However, we ~~secured~~will need to secure additional funding in the future, from one or more equity or debt financings, government funding, collaborations, licensing transactions, other commercial transactions or other sources in order to carry out all of ~~$107.4 million in net proceeds from the closing of the PRV transaction~~our commercialization and planned research and development activities with ~~Novo Nordisk, Inc. In October 2022, we entered into a royalty monetization agreement with Sagard Healthcare Partners, pursuant~~respect to ~~which we received a total of $32.5 million upfront in return for royalty payments on U.S. net sales of~~ ganaxolone. We believe our cash and cash equivalents on hand as of September 30, 2022, inclusive of the $32.5 million payment from Sagard Healthcare Partners and excluding the $15.0 million liquidity requirement associated with our Note Payable (Note 9), is sufficient to fund operations for at least the next twelve months from the issuance of these financial statements.

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2. Summary of Significant Accounting Policies

Basis of Presentation

The accompanying unaudited interim consolidated financial statements include the accounts of Marinus Pharmaceuticals, Inc. (a Delaware corporation) as well as the accounts of Marinus Pharmaceuticals Emerald Limited (an Ireland company incorporated in February 2021), a wholly owned subsidiary requiring consolidation. Marinus Pharmaceuticals Emerald Limited serves as a corporate presence in the European Union for regulatory purposes. The unaudited interim consolidated financial statements included herein have been prepared pursuant to the rules and regulations of the Securities and Exchange Commission (SEC). Accordingly, they do not include all information and disclosures necessary for a presentation of our financial position, results of operations and cash flows in conformity with generally accepted accounting principles in the U.S. (GAAP) for annual financial statements. In the opinion of management, these unaudited interim consolidated financial statements reflect all adjustments, consisting primarily of normal recurring accruals, necessary for a fair presentation of our financial position and results of operations and cash flows for the periods presented. The results of operations for interim periods are not necessarily indicative of the results for the full year. These unaudited interim consolidated financial statements should be read in conjunction with the audited financial statements for the year ended December 31, ~~2021~~2022 and accompanying notes thereto included in our Annual Report on Form 10-K filed with the SEC on March ~~24, 2022.~~9, 2023.

Use of Estimates

The preparation of financial statements in conformity with 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 income and expenses during the reporting period. Actual results could differ from such estimates.

Product Revenue, net

We recognize ZTALMY revenue in accordance with ASC 606 – Revenue from contracts with customers. Our revenue recognition analysis consists of the following steps: (i) identification of the promised goods in the contract; (ii) determination of whether the promised goods are performance obligations, including whether they are capable of being distinct; (iii) measurement of the transaction price, including the constraint on variable consideration; (iv) allocation of the transaction price to the performance obligations; and (v) recognition of revenue as we satisfy each performance obligation.

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contract with Orsini has a single performance obligation to deliver ZTALMY upon receipt of a purchase order, which is satisfied when Orsini receives ZTALMY. We recognize ZTALMY revenue at the point in time when control of ZTALMY is transferred to Orsini, which is upon delivery to Orsini. The transaction price that we recognize for ZTALMY revenue includes an estimate of variable consideration. Shipping and handling costs to Orsini are recorded as selling, general and administrative expenses. The components of variable consideration include:

Trade Discounts and Allowances. We provide an incentive prompt payment discount to Orsini as explicitly stated in the contract with Orsini. This discount is recorded as a reduction of ZTALMY revenue and accounts receivable in the period in which the related ZTALMY revenue is recognized. We estimate the amount of variable consideration for all discounts and allowances using the expected value method.

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will promptly notify Orsini and will reimburse Orsini for direct administrative expenses incurred in connection with the recall as well as the cost of replacement product.

Government Rebates. We are subject to discount obligations under state Medicaid programs, Medicare, and ~~Medicare.~~the Tricare Retail Refund Program. We estimate reserves related to these discount programs and record these obligations in the same period the related revenue is recognized, resulting in a reduction of product revenue.

Patient Assistance. We offer a voluntary co-pay patient assistance program intended to provide financial assistance to eligible patients with a prescription drug co-payment required by payors and coupon programs for cash payors. The calculation of the current liability for this assistance is based on an estimate of claims and the cost per claim that we expect to receive associated with ZTALMY that has been recognized as revenue but remains in the distribution channel inventories at the end of each reporting period.

Federal Contract Revenue

We recognize federal contract revenue from the BARDA Contract in the period in which the allowable research and development expenses are incurred, and receivables associated with this revenue are included within accounts receivable on our interim consolidated balance sheets. This revenue is not within the scope of ASC 606 – Revenue from contracts with customers.

~~Trade Receivables,~~Short-term Investments

We classify our short-term investments as available-for-sale securities, which include U.S. government agency debt securities and U.S. treasury debt securities with original maturities of greater than three months. These securities are carried at fair market value, with unrealized gains and losses reported in comprehensive loss and accumulated other comprehensive income (loss) within stockholders’ equity. All of our investments were short-term in nature as of September 30, 2023.

Accounts Receivable, net

Net trade receivables related to ZTALMY sales, which are recorded in net ~~Accounts~~accounts receivable on the consolidated balance sheets, were approximately ~~$0.2~~$2.3 million and $1.3 million as of September 30, ~~2022. There were no net trade receivables related to ZTALMY as of~~2023 and December 31, ~~2021.~~2022, respectively. As of both September 30, ~~2022~~2023 and December 31, ~~2021,~~2022, we had no allowance for doubtful accounts. An allowance for doubtful accounts is determined based on our assessment of the ~~credit worthiness~~creditworthiness and financial condition of our ~~customer,~~customers, aging of receivables, as well as the general economic environment. Any allowance would reduce the net receivables to the amount that is expected to be collected. Payment terms for Orsini are approximately 30 days from the shipment date.

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Excluding net trade receivables, accounts receivable represents amounts due to us under the BARDA contract for valid expenditures expected to be reimbursed to us under the terms of the BARDA contract and current amounts due to us from Orion Corporation (Orion) under the collaboration agreement (Note 12).

Inventory

Inventories are recorded using actual costs and may consist of raw materials (ganaxolone API), work in process and finished goods. We began capitalizing inventory related to ZTALMY subsequent to the March 2022 FDA approval of ZTALMY, as the related costs were expected to be recoverable through the commercialization and subsequent sale of ZTALMY. Prior to FDA approval of ZTALMY, costs estimated at approximately $2 million for commercially saleable product and materials were incurred and included in research and development expenses. As a result, cost of product revenues related to ZTALMY will initially reflect a lower average per unit cost of materials ~~over~~into approximately the ~~next approximately 24 months~~first half of 2024, as previously expensed inventory is utilized for commercial production and sold to customers.

Debt Issuance Costs

Debt issuance costs incurred in connection with Note payable (Note 9)10) and revenue interest financing payable (Note 11) are amortized to interest expense over the term of the respective financing arrangement using the effective-interest method. Debt issuance costs, net of related amortization, are deducted from the carrying value of the related debt.

Contract ~~Liability~~Liabilities, net

When consideration is received, or such consideration is unconditionally due, from a customer prior to completing our performance obligation to the customer under the terms of a contract, a contract liability is recorded. Contract liabilities expected to be recognized as revenue or a reduction of expense within the 12 months following the balance sheet date are classified as current liabilities. Contract liabilities not expected to be recognized as revenue within

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the 12 months following the balance sheet date are classified as long-term liabilities. In accordance with ASC 210-20, our contract ~~liability is~~liabilities are partially offset by aour contract ~~asset~~assets as further discussed in Note ~~10.~~12.

Liability Related to Revenue Interest Financing and Non-Cash Interest Expense

In October 2022, we recognized a liability related to the Revenue Interest Financing Agreement with Sagard Healthcare Royalty Partners, LP (Sagard) under ASC 470-10 Debt and ASC 835-30 Interest - Imputation of Interest. The initial funds we received from Sagard pursuant to the terms of the Revenue Interest Financing Agreement were recorded as a liability and will be accreted under the effective interest method upon the estimated amount of future royalty payments to be made pursuant to the Revenue Interest Financing Agreement. The issuance costs were recorded as a direct deduction to the carrying amount of the liability and will be amortized under the effective interest method over the estimated period the liability will be repaid as further discussed in Note 11. We estimated the total amount of future product revenue to be generated over the life of the Revenue Interest Financing Agreement, and a significant increase or decrease in these estimates could materially impact the liability balance and the related interest expense. If the timing or amounts of any estimated future revenue and related payments change, we will prospectively adjust the effective interest and the related amortization of the liability and related issuance costs.

Collaboration and Licensing Revenue

We may enter into collaboration and licensing arrangements for research and development, manufacturing, and commercialization activities with counterparties for the development and commercialization of our product candidates. These arrangements may contain multiple components, such as (i) licenses, (ii) research and development activities, and (iii) the manufacturing of certain material. Payments pursuant to these arrangements may include non-refundable and refundable payments, payments upon the achievement of significant regulatory, development and commercial milestones, sales of product at certain agreed-upon amounts, and royalties on product sales. The amount of variable consideration is constrained until it is probable that the revenue is not at a significant risk of reversal in a future period.

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In determining the appropriate amount of revenue to be recognized as we fulfill our obligations under a collaboration agreement, we perform the following steps: (i) identification of the promised goods or services in the contract; (ii) determination of whether the promised goods or services are performance obligations, including whether they are capable of being distinct; (iii) measurement of the transaction price, including the constraint on variable consideration; (iv) allocation of the transaction price to the performance obligations; and (v) recognition of revenue as we satisfy each performance obligation.

We must develop estimates and assumptions that require judgment to determine the underlying stand-alone selling price for each performance obligation, which determines how the transaction price is allocated among the performance obligations. The estimation of the stand-alone selling price may include such estimates as forecasted revenues and costs, development timelines, discount rates and probabilities of regulatory and commercial success. We also apply significant judgment when evaluating whether contractual obligations represent distinct performance obligations, allocating transaction price to performance obligations within a contract, determining when performance obligations have been met, assessing the recognition and future reversal of variable consideration and determining and applying appropriate methods of measuring progress for performance obligations satisfied over time.

3. Cash, Cash Equivalents and Short-Term Investments

As of September 30, 2023, our cash and cash equivalents included $1.2 million of cash accounts in banking institutions and $139.3 million in money market funds. As of December 31, 2022, our cash and cash equivalents included $10.6 million of cash accounts in banking institutions and $230.0 million in money market funds. Our cash and cash equivalents are maintained in federally insured financial institutions in excess of the federally insured limit. Included in other assets at September 30, 2023 was $0.2 million of accrued interest receivable related to our short-term investments.

The following table provides details regarding our portfolio of short-term investments (in thousands) as of September 30, 2023:


	Amortized Cost		Unrealized Gains		Unrealized Losses		Fair Value
U.S. Treasury securities	$	28,624	$	97	$	(155)	$	28,566
U.S. Government Agency securities		7,366		21		(34)		7,353
Total	$	35,990	$	118	$	(189)	$	35,919

We did not have any short-term investments as of December 31, 2022.

4. Fair Value Measurements

FASB accounting guidance defines fair value as the price that would be received to sell an asset or paid to transfer a liability (the exit price) in an orderly transaction between market participants at the measurement date. The accounting guidance outlines a valuation framework and creates a fair value hierarchy in order to increase the consistency and comparability of fair value measurements and the related disclosures. In determining fair value, we use quoted prices and observable inputs. Observable inputs are inputs that market participants would use in pricing the asset or liability based on market data obtained from independent sources.

The fair value hierarchy is broken down into three levels based on the source of inputs as follows:

●

Level 1 — Valuations based on unadjusted quoted prices in active markets for identical assets or liabilities.

●

Level 2 — Valuations based on observable inputs and quoted prices in active markets for similar assets and liabilities.

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●

Level 3 — Valuations based on inputs that are unobservable and models that are significant to the overall fair value measurement.

If the inputs used to measure fair value fall within different levels of the hierarchy, the category level is based on the lowest priority level input that is significant to the fair value measurement of the instrument. As of ~~both~~ September 30, ~~2022~~2023, all of our financial assets and liabilities were classified as Level 1 or Level 2 valuations. As of December 31, ~~2021,~~2022, all of our financial assets and liabilities were classified as Level 1 valuations.

~~Table~~We estimate the fair values of ~~Contents~~our financial instruments categorized as Level 2 in the fair value hierarchy, including U.S. Treasury securities and U.S. Government Agency securities, by taking into consideration valuations obtained from third-party pricing services. The pricing services use industry standard valuation models, including both income- and market-based approaches, for which all significant inputs are observable, either directly or indirectly, to estimate fair value. These inputs include reported trades of and broker/dealer quotes on the same or similar securities, benchmark yields, issuer credit spreads, benchmark securities, and other observable inputs. We obtain a single price for each financial instrument and do not adjust the prices obtained from the pricing service.

The following fair value hierarchy table presents information about each major category of our financial assets and liabilities measured at fair value on a recurring basis (in thousands):


	Level 1		Level 2		Level 3		Total		Level 1		Level 2		Level 3		Total
September 30, 2022
September 30, 2023
Assets
Cash									$	1,161	$	—	$	—	$	1,161
Money market funds (cash equivalents)										139,276		—		—		139,276
U.S. Treasury securities										—		28,566		—		28,566
U.S. Government Agency securities										—		7,353		—		7,353
Total assets									$	140,437	$	35,919	$	—	$	176,356
December 31, 2022
Assets
Cash	$	5,575	$	—	$	—	$	5,575	$	10,569	$	—	$	—	$	10,569
Money market funds (cash equivalents)		162,674		—		—		162,674		229,982		—		—		229,982
Total assets	$	168,249	$	—	$	—	$	168,249	$	240,551	$	—	$	—	$	240,551
December 31, 2021
Assets
Cash	$	2,360	$	—	$	—	$	2,360
Money market funds (cash equivalents)		120,567		—		—		120,567
Total assets	$	122,927	$	—	$	—	$	122,927

4.5. Inventory

Inventories are stated at actual ~~costs. At September 30, 2022, inventory~~costs and consisted of the following (in thousands):

September 30,

2022

Raw materials

$
—

Work in process

—

Finished goods

104

Total Inventories

$
104

~~There were no inventories at December 31, 2021.~~

~~5. Accrued Expenses~~

~~Accrued expenses consisted of the following (in thousands):~~

September 30,

December 31,

2022

2021

Payroll and related costs

$
6,466

$
5,830

Clinical trials and drug development

7,601

8,217

Professional fees

1,066

1,311

Accrued tax provision

1,752

—

Costs related to sales and marketing

1,207

—

Short-term lease liabilities

616

556

Other

720

293

Total accrued expenses

$
19,428

$
16,207


	September 30,		December 31,
	2023		2022
Raw materials	$	1,410	$	—
Work in process		571		—
Finished goods		755		77
Total Inventories	$	2,736	$	77

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6. Accrued Expenses

Accrued expenses consisted of the following (in thousands):


	September 30,		December 31,
	2023		2022
Payroll and related costs	$	7,159	$	7,061
Clinical trials and drug development		5,303		5,725
Professional fees		594		1,417
Accrued tax provision		—		2,445
Third-party commercial expenses		3,708		1,880
Short-term lease liabilities		704		637
Other		860		371
Total accrued expenses	$	18,328	$	19,536

7. Net (Loss) Income ~~(Loss)~~ Per Share of Common Stock

Basic net (loss) income ~~(loss)~~ per share of common stock is computed by dividing net (loss) income ~~(loss)~~ attributable to common stockholders by the weighted average number of shares of common stock outstanding during each period, without consideration for potential dilutive shares of common stock. Diluted net (loss) income ~~(loss)~~ per share of common stock includes the effect, if any, from the potential exercise or conversion of securities, such as convertible preferred stock, stock options, and unvested restricted stock, which would result in the issuance of incremental shares of common stock. Diluted net (loss) income ~~(loss)~~ per share of common stock is computed by dividing the net (loss) income ~~(loss)~~ attributable to common stockholders by the weighted-average number of common share equivalents outstanding for the period determined using the treasury-stock method and if-converted method, as applicable. Basic and diluted net (loss) income ~~(loss)~~ per share attributable to common stockholders is presented in conformity with the two-class method required for participating securities, which include convertible preferred stock.

Under the two-class method, undistributed earnings are allocated to common stock and convertible preferred stock to the extent that each preferred security may share in earnings as if all of the earnings for the period had been distributed. The total earnings allocated to common stock is then divided by the number of outstanding shares to which the earnings are allocated to determine the earnings per share. The two-class method is not applicable during periods with a net loss, as the holders of the convertible preferred stock have no obligation to fund losses.

The pre-funded warrants to purchase common stock issued in connection with the November 2022 offering are included in the calculation of basic and diluted net loss per share as the exercise price of $0.001 per share is non-substantive and is virtually assured. The pre-funded warrants are more fully described in Note 8.

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The computations for basic and diluted net (loss) income ~~(loss)~~ per share were as follows (in thousands, except per-share data):

Three Months Ended

Nine Months Ended

September 30,

September 30,

2022

2021

2022

2021

Numerator






Net income (loss)

$
73,290

$
(19,507)

$
14,496

$
(70,471)

Less: Net income attributable to preferred shareholders

(1,656)

—

(336)

—

Net income (loss) attributable to common shareholders

71,634

(19,507)

14,160

(70,471)

Denominator

Basic weighted average shares outstanding

37,202,269

36,744,591

37,084,060

36,667,472

Effect of dilutive securities

708,242

—

1,309,694

—

Diluted weighted average shares outstanding

37,910,511

36,744,591

38,393,754

36,667,472

Basic net income (loss) per share of common stock

$
1.93

$
(0.53)

$
0.38

$
(1.92)

Diluted net income (loss) per share of common stock

$
1.89

$
(0.53)

$
0.37

$
(1.92)


	Three Months Ended				Nine Months Ended
	September 30,				September 30,
	2023			2022	2023		2022
Numerator
Net (loss) income	$	(32,972)	$	73,290	$	(99,636)	$	14,496
Less: Net income attributable to preferred shareholders		—		(1,656)		—		(336)
Net (loss) income attributable to common shareholders		(32,972)		71,634		(99,636)		14,160

Denominator
Basic weighted average shares outstanding		53,920,109		37,202,269		52,755,114		37,084,060
Effect of dilutive securities		—		708,242		—		1,309,694
Diluted weighted average shares outstanding		53,920,109		37,910,511		52,755,114		38,393,754


Basic net (loss) income per share of common stock	$	(0.61)	$	1.93	$	(1.89)	$	0.38
Diluted net (loss) income per share of common stock	$	(0.61)	$	1.89	$	(1.89)	$	0.37

The following potentially dilutive securities have been excluded from the computation of diluted weighted-average shares of common stock outstanding prior to the use of the two-class method, as they would be anti-dilutive:


	Three Months Ended		Nine Months Ended		Three Months Ended		Nine Months Ended
	September 30,		September 30,		September 30,		September 30,
	2022	2021	2022	2021	2023	2022	2023	2022
Convertible preferred stock	—	915,000	—	915,000
Restricted stock awards and restricted stock units	—	37,000	—	37,000	1,265,316	—	1,265,316	—
Stock options	4,735,841	4,625,768	4,566,687	4,625,768	7,188,661	4,735,841	7,188,661	4,566,687
	4,735,841	5,577,768	4,566,687	5,577,768	8,453,977	4,735,841	8,453,977	4,566,687

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7.8. Stockholders’ Equity

In 2005, we adopted the 2005 Stock Option and Incentive Plan (2005 Plan) that authorizes us to grant stock options, restricted stock and other equity-based awards. As of September 30, ~~2022,~~2023, 577 options to purchase shares of common stock were outstanding pursuant to grants in connection with the 2005 Plan. No additional shares are available for issuance under the 2005 Plan. The amount, terms of grants, and exercisability provisions are determined and set by our board of directors.

Effective August 2014, we adopted our 2014 Equity Incentive Plan, as amended (2014 Plan), that authorizes us to grant stock options, restricted stock, and other equity-based awards, subject to adjustment in accordance with the 2014 Plan. As of September 30, ~~2022, 3,833,770~~2023, 5,063,925 options to purchase shares of common stock were outstanding pursuant to grants in connection with the 2014 Plan, and ~~888,309~~775,794 shares of common stock were available for future issuance. The amount, terms of grants, and exercisability provisions are determined and set by our board of directors. In accordance with the 2014 Plan, on January 1, ~~2022,~~2023, the shares of common stock available for future grants under the 2014 Plan was increased ~~to 2,343,330.~~by 2,020,111.

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Stock Options

There were ~~5,790,813~~7,188,661 stock options outstanding as of September 30, ~~2022~~2023 at a weighted average exercise price of ~~$10.99~~$9.75 per share, including ~~1,956,466~~2,124,159 stock options outstanding outside of the 2014 Plan, granted as inducements to new employees. During the nine months ended September 30, ~~2022, 1,878,607~~2023, 1,971,291 options were granted to employees and directors at a weighted average exercise price of ~~$9.06~~$6.34 per share. Of the options granted, ~~1,230,471~~1,550,766 options were granted pursuant to the 2014 Plan and ~~648,136~~420,525 were granted outside of the 2014 Plan as inducements for new employees.

~~Total compensation cost recognized for all stock option awards in the statements of operations is as follows (in thousands):~~

Three Months Ended

Nine Months Ended

September 30,

September 30,

2022

2021

2022

2021

Research and development

$
1,210

$
1,060

$
3,615

$
3,333

Selling, general and administrative

2,178

1,700

6,202

7,317

Total

$
3,388

$
2,760

$
9,817

$
10,650

Restricted Stock and Restricted Stock Units

All issued and outstanding restricted shares of common stock are time-based, and become vested within two years of the grant date, pursuant to the 2014 Plan. Compensation expense is recorded ratably over the requisite service period. Compensation expense related to restricted stock is measured based on the fair value using the closing market price of our common stock on the date of the grant. As of September 30, ~~2022,~~2023, we ~~had~~ ~~12,500 outstanding~~did not have any restricted shares of ~~restricted~~ common ~~stock.~~stock outstanding.

During the nine months ended September 30, ~~2022,~~2023, we granted ~~801,028~~974,940 restricted stock units, which vest within ~~four~~three years of the grant date, pursuant to the 2014 Plan. As of September 30, ~~2022,~~2023, we had ~~710,732~~1,265,316 restricted stock units outstanding.

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Total compensation cost recognized for all stock options, restricted stock awards and restricted stock units in the statements of operations is as follows (in thousands):


	Three Months Ended				Nine Months Ended				Three Months Ended				Nine Months Ended
	September 30,				September 30,				September 30,				September 30,
	2022		2021		2022		2021		2023		2022		2023		2022
Research and development	$	184	$	—	$	454	$	—	$	1,519	$	1,394	$	4,329	$	4,069
Selling, general and administrative		323		81		820		217		2,487		2,501		7,309		7,022
Total	$	507	$	81	$	1,274	$	217	$	4,006	$	3,895	$	11,638	$	11,091

Preferred Stock

As of September 30, ~~2022, 4,300~~2023 all shares of our Series A Convertible Preferred Stock (Preferred Stock) had been converted and none remained ~~outstanding, convertible into 860,000 shares of our common stock. During~~outstanding. In the nine months ended September 30, ~~2022, 275~~2023, 4,300 shares of our Preferred ~~Stock~~stock were converted into ~~55,000~~860,000 shares of our common stock.

Stock Issued in Connection with Ovid License Agreement

On March 29, 2022, pursuant to an exclusive patent license agreement with Ovid Therapeutics Inc. (Ovid), we issued 123,255 shares of our common stock to Ovid. The shares were issued in reliance upon the exemption from the registration requirements of the Securities Act of 1933, as amended (the Securities Act) provided by Section 4(a)(2) of the Securities Act and Regulation D thereunder as sales by an issuer not involving any public offering (see Part II, Item 2. Unregistered Sales of Equity Securities and Use of Proceeds). The fair value of these shares is reflected in operating expenses for the nine months ended September 30, 2022.

Underwritten Public Offering

In connection with an underwritten public offering in November 2022 and the closing of the related exercise of the underwriters’ option in December 2022, we issued a total of 12,421,053 shares of common stock and 2,105,264 pre-funded warrants (the Pre-funded Warrants) resulting in aggregate net proceeds, after underwriting discounts and commissions in the public offering and fees, of $64.5 million. The exercise price and the number of shares of common stock issuable upon exercise of each Pre-funded Warrant are subject to appropriate adjustment in the event of certain stock dividends and distributions, stock splits, stock combinations, reclassifications or similar events affecting the

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common stock as well as upon any distribution of assets, including cash, stock or other property, to our stockholders. The Pre-funded Warrants are exercisable at any time, will not expire and are exercisable in cash or by means of a cashless exercise. A holder of Pre-funded Warrants may not exercise such Pre-funded Warrants if the holder, together with its affiliates, would beneficially own more than 9.99% of the number of shares of common stock outstanding immediately after giving effect to such exercise. A holder of Pre-funded Warrants may increase or decrease this percentage not in excess of 19.99% by providing at least 61 days’ prior notice to us.

Sales Pursuant to Equity Distribution Agreement

On July 9, 2020, we entered into an Equity Distribution Agreement (EDA) with JMP Securities LLC (JMP), as amended by the March 31, 2023 Amendment No. 1 to the EDA (Amended Agreement), to create an at the market equity program under which we from time to time may offer and sell shares of our common stock without a maximum aggregate offering price. The Amended Agreement was entered into in connection with our filing of a Registration Statement on Form S-3 (File No. 333-271041) with the SEC (the 2023 Registration Statement), which includes a prospectus supplement covering the offering, issuance and sale by us of up to $75,000,000 of shares of common stock that may be issued and sold under the Amended Agreement. Subject to the terms and conditions of the Amended Agreement, JMP will be entitled to a commission of up to 3.0% of the gross proceeds from each sale of shares of our common stock. In the nine months ended September 30, 2023, we sold approximately 3.7 million shares of our common stock pursuant to the Amended Agreement, which consisted of net proceeds of approximately $25.9 million. We did not sell any shares of our common stock during the nine months ended September 30, 2022 under the EDA.

8.9. Leases

We have entered into one operating lease for real estate. This lease has a term of 78 months, and includes renewal terms which can extend the lease ~~terms~~term by 60 months, ~~(which~~which we include in the lease ~~terms~~term when it is reasonably certain that we will exercise the ~~option).~~option. As of September 30, ~~2022,~~2023, our operating lease had a remaining lease term of 3624 months. The right-of-use (ROU) asset is included in ~~"Other assets"~~“Other assets” on our interim consolidated balance sheets as of ~~both~~ September 30, ~~2022~~2023 and December 31, ~~2021,~~2022, and represents our right to use the underlying asset for the lease term. Our obligations to make lease payments are included in ~~"Accrued expenses"~~both “Accrued expenses” and ~~"Other~~“Other long-term ~~liabilities"~~liabilities” on our interim consolidated balance sheets as of ~~both~~ September 30, ~~2022~~2023 and December 31, ~~2021, respectively.~~2022. The ROU asset was initially measured at cost, which comprises the initial amount of the lease liability adjusted for lease payments made at or before the lease commencement date, plus any initial direct costs incurred, less any lease incentives received. The ROU asset is subsequently measured throughout the lease term at the carrying amount of the lease liability, plus initial direct costs, plus (minus) any prepaid (accrued) lease payments, less the unamortized balance of lease incentives received.

As of ~~both~~ September 30, ~~2022~~2023 and December 31, ~~2021,~~2022, ROU assets were ~~$1.4~~$1.0 million and ~~$1.7~~$1.3 million, respectively, and operating lease liabilities were ~~$2.1~~$1.5 million and ~~$2.5~~$2.0 million, respectively. We have entered into various short-term operating leases, primarily for clinical trial equipment, with an initial term of twelve months or less. These leases are not recorded on our balance sheets. All operating lease expense is recognized on a straight-line basis over the lease term. During each of the three months ended September 30, ~~2022~~2023 and ~~2021,~~2022, we recognized $0.1 million ~~and $0.2 million, respectively,~~ in total lease costs. During each of the nine months ended September 30, ~~2022~~2023 and ~~2021,~~2022, we recognized $0.4 million ~~and $0.5 million, respectively,~~ in total ~~lease~~least costs. In all periods, we recognized less than ~~$0.1~~$0.1 million in short-term lease costs related to short-term operating leases.

Because the rate implicit in each lease is not readily determinable, we use our incremental borrowing rate to determine the present value of the lease payments. The weighted average incremental borrowing rate used to determine the initial value of ROU assets and lease liabilities was 11.0%, derived from a corporate yield curve based on a synthetic credit rating model using a market signal analysis. We have certain contracts for real estate which may contain lease and non-lease components which we have elected to treat as a single lease component.

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ROU assets for operating leases are periodically reduced by impairment losses. We use the long-lived assets impairment guidance in ASC Subtopic 360-10, Property, Plant, and Equipment – Overall, to determine whether an ROU

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asset is impaired, and if so, the amount of the impairment loss to recognize. As of ~~both~~ September 30, ~~2022~~2023 and December 31, ~~2021,~~2022, we have not recognized any impairment losses for our ROU assets.

We monitor for events or changes in circumstances that require a reassessment of our lease. When a reassessment results in the remeasurement of a lease liability, a corresponding adjustment is made to the carrying amount of the corresponding ROU asset unless doing so would reduce the carrying amount of the ROU asset to an amount less than zero. In that case, the amount of the adjustment that would result in a negative ROU asset balance is recorded in our interim consolidated statements of operations and comprehensive loss.

Maturities of operating lease liabilities as of September 30, ~~2022~~2023 were as follows (in thousands):




Remaining three months of 2022	$	203
2023		823
Remaining three months of 2023			$	206
2024		840		840
2025		642		642
Thereafter		—
		2,508		1,688
Less: imputed interest		(384)		(180)
Total lease liabilities	$	2,124	$	1,508

Current operating lease liabilities	$	616	$	704
Non-current operating lease liabilities		1,508		804
Total lease liabilities	$	2,124	$	1,508

9.10. Notes Payable

On May 11, 2021 (Closing Date) and as amended on May 17, 2021, ~~and~~ May 23, 2022 ~~(the Credit Agreement Amendment)~~and October 28, 2022 (Credit Agreement) we entered into the Credit Agreement with Oaktree Fund Administration, LLC as administrative agent (Oaktree) and the lenders party thereto (collectively, the Lenders) that ~~provides~~provided for a five-year senior secured term loan facility in an aggregate original principal amount of up to ~~$100.0~~$125.0 million that was available to us in ~~four~~five tranches (collectively, the Term Loans). On October 28, 2022, we entered into that certain Limited Consent and First Amendment to Credit Agreement in connection with the Revenue Interest Financing Agreement. Refer to Note 12. Subsequent Events for further information.

Upon entering into the Credit Agreement in May 2021, we borrowed $15.0 million in term loans from the Lenders (Tranche A-1 Term Loans); upon receipt of written acceptance by the FDA of our NDA filing relating to the use of ganaxolone in CDD in September 2021 we borrowed $30.0 million of tranche A-2 term loans from the Lenders (Tranche A-2 Term Loans); and in March 2022, we borrowed $30.0 million in term loans from the Lenders that became available as a result of the approval by the FDA of ZTALMY oral suspension for the treatment of seizures associated with CDD in patients two years of age and older (Tranche B Term Loans). In May 2022, we entered into an amendment to extend the commitment date for the tranche C ~~term loans~~Term Loans (Tranche C Term Loans) commitment from June 30, 2023 to December 31, 2023, and to eliminate the commitment fees associated with the Tranche C Term Loans. Also in May 2022, we delivered to Oaktree a separate notice of commitment termination with respect to the tranche D term loans (Tranche D Term Loans) commitment. ~~Under~~In October 2022, we entered into an amendment to, among other things, allow for the ~~terms~~consummation of a revenue interest financing agreement (Revenue Interest Financing Agreement) with Sagard Health Royalty Partners (Sagard) and the transactions thereunder. In addition, the October 2022 amendment increased the exit fee due by us upon any repayment, whether as a prepayment or a scheduled repayment, of the principal of the loans under the Credit Agreement from 2.00% to 2.67%. In August 2023, we ~~may, at our sole discretion, borrow from~~delivered to Oaktree a separate notice of commitment termination with respect to the ~~Lenders up to an additional~~ $25.0 million ~~in term~~of Tranche C Term Loans commitment. Following the termination of the Tranche C Term Loan Commitment, the loans ~~subject to~~under the ~~following milestone event:~~Credit Agreement consist of $75.0 million of previously drawn Term Loans with no additional funds available thereunder.

●

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~~million and net proceeds to us of at least $36.0 million. In addition, the availability of this tranche is subject to~~ either our current Phase 3 trial in RSE or a Phase 3 trial in TSC achieving statistical significance (p value < 0.05) across all primary endpoints and ganaxolone must be generally well tolerated, with a safety profile generally consistent with previous clinical trials.

~~In addition, the~~The Credit Agreement contains a minimum liquidity covenant that requires us to maintain cash and cash equivalents of at least $15.0 million from the funding date of the Tranche B Term Loans until the maturity of the Term Loans.

The Term Loans will be guaranteed by certain of our future subsidiaries (Guarantors). Our obligations under the Credit Agreement are secured by a pledge of substantially all of our assets and will be secured by a pledge of substantially all of the assets of the Guarantors.

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until the Maturity Date. We are also required to make quarterly principal payments beginning on June 30, 2024 in an amount equal to 5.0% of the aggregate amount of the Term Loans outstanding on June 30, 2024, and continuing until the Maturity Date. On the Maturity Date, we are required to pay in full all outstanding Term Loans and other amounts owed under the Credit Agreement.

At the time of borrowing any tranche of the Term Loans, we ~~are~~were required to pay an upfront fee of 2.0% of the aggregate principal amount borrowed at that time. In addition, a commitment fee of 75 basis points per annum began to accrue on each of the tranche B, C, and D commitments for the period beginning 120 days after the funding date of the Tranche A-2 Term Loans, and continued until the applicable tranche was either funded or terminated, at which time the related commitment fees were due. The Tranche A-2 Term Loans were funded on September 27, 2021, and as such, we began accruing the commitment fees for tranche B, C, and D Term Loans 120 days later, on January 25, 2022. We drew down the additional $30.0 million of Tranche B Term Loans in March 2022, and paid less than $0.1 million in commitment fees related to Tranche B Term Loans. The May 2022 amendment eliminated the commitment fees related to the Tranche C Term Loans, and separately, we terminated the Tranche D Term Loans in May ~~2022. We did not incur any commitment fees for the three months ended September 30,~~ 2022 and ~~will not incur any additional commitment fees~~the Tranche C Term Loans in ~~the future.~~August 2023.

We may prepay all or any portion of the Term Loans, and are required to make mandatory prepayments of the Term Loans from the proceeds of asset sales, casualty and condemnation events, and prohibited debt issuances, subject to certain exceptions. All mandatory and voluntary prepayments of the Term Loans are subject to prepayment premiums equal to (i) 4% of the principal prepaid plus a “make-whole” amount equal to the interest that would have accrued through May 11, 2023 if prepayment ~~occurs~~occurred on or before May 11, 2023, (ii) 4% of the principal prepaid if prepayment occurs after May 11, 2023 but on or before May 11, 2024, or (iii) 2% of the principal prepaid if prepayment occurs after May 11, 2024 but on or before May 11, 2025. If prepayment occurs after May 11, 2025, no prepayment premium is due.

We are also required to make mandatory prepayments of the Term Loans upon an event of default under the Credit Agreement resulting from the occurrence of a change of control. ~~These mandatory prepayments are subject to a prepayment premium equal to~~ ~~10.0% of the principal prepaid if the prepayment occurs after May 11, 2022 but on or before May 11, 2023.~~

In addition, we are required to pay an exit fee in an amount equal to ~~2.0%~~2.67% of all principal repaid, whether as a mandatory prepayment, voluntary prepayment, or a scheduled repayment. OnPrior to the October ~~28,~~ 2022 ~~we entered into that certain Limited Consent and First Amendment~~amendment to the Credit Agreement, ~~in connection with the Revenue Interest Financing Agreement, which increased~~ the exit fee ~~from~~was 2.0% ~~to 2.67%~~. ~~Refer to Note 12. Subsequent Events for further information.~~The increase in the exit fee resulted in an additional $0.5 million of debt issuance costs that are classified as a contra-liability on the consolidated balance sheets and is being recognized as interest expense over the term of the loan using the effective interest method.

In addition to the minimum liquidity covenant, we are subject to a number of affirmative and restrictive covenants under the Credit Agreement, including limitations on our ability and our subsidiaries’ abilities, among other things, to incur additional debt, grant or permit additional liens, make investments and acquisitions, merge or consolidate

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with others, dispose of assets, pay dividends and distributions, and enter into affiliate transactions, subject to certain exceptions. As of September 30, ~~2022,~~2023, we were in compliance with all covenants.

Upon the occurrence of certain events, including but not limited to our failure to satisfy our payment obligations under the Credit Agreement, the breach of certain of our other covenants under the Credit Agreement, the occurrence of cross defaults to other indebtedness, or defaults related to enforcement action by the FDA or other Regulatory Authority or recall of ganaxolone, Oaktree and the Lenders will have the right, among other remedies, to accelerate all amounts outstanding under the Term Loans and declare all principal, interest, and outstanding fees immediately due and payable.

In March 2022, we borrowed $30.0 million upon the approval by the FDA of ZTALMY for CDD and incurred debt issuance costs of $1.8 million, including the exit fee of $0.6 million, that are classified as contra-liabilities on our consolidated balance sheets and are being recognized as interest expenses over the term of the loan using the effective interest method.

In September 2021, we borrowed $30.0 million upon receipt of written acceptance by the FDA of our NDA filing relating to the use of ganaxolone in the treatment of CDD and incurred debt issuance costs of $1.2 million,

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including the exit fee of $0.6 million, that are classified as contra-liabilities on our consolidated balance sheets and are being recognized as interest expenses over the term of the loan using the effective-interest method.

In May 2021, we borrowed $15.0 million upon entering into the Credit Agreement and incurred debt issuance costs of $4.4 million, including the exit fee of $0.3 million, that are classified as a contra-liabilities on the consolidated balance sheet and are being recognized as interest expenses over the term of the loan using the effective-interest method.

For the nine months ended September 30, ~~2022,~~2023, we recognized interest expense of ~~$7.0~~$8.0 million, of which ~~$5.7~~$6.5 million was interest on the Term Loans ~~$1.2~~and $1.5 million was non-cash interest expense related to the amortization of debt issuance ~~costs, and $0.1 million was non-cash interest expense related to the commitment fee.~~costs.

The following table summarizes the composition of Notes payable as reflected on the consolidated balance sheet as of September 30, ~~2022~~2023 (in thousands):


Gross proceeds	$	75,000	$	75,000
Contractual exit fee		1,500		2,003
Unamortized debt discount and issuance costs		(5,720)		(4,520)
Total	$	70,780
Total note payable			$	72,483

Current portion of note payable				7,700
Non-current portion of note payable				64,783
Total note payable			$	72,483

The aggregate maturities of Notes payable as of September 30, ~~2022~~2023 are as follows (in thousands):


Remainder of 2022	$	—
2023		—
Remainder of 2023			$	—
2024		11,250		11,250
2025		15,000		15,000
2026 and thereafter		48,750
2026				48,750
Total	$	75,000	$	75,000

11. Revenue Interest Financing Agreement

On October 28, 2022 (Closing Date), we entered into the Revenue Interest Financing Agreement with Sagard pursuant to which we received $32.5 million (Investment Amount) to provide funding for our development and commercialization of ganaxolone and related pharmaceutical products, including the commercial launch of ZTALMY, and for working capital and general administrative purposes.

In exchange for the Investment Amount, we have agreed to make quarterly payments to Sagard (Payments) as follows: (i) for each calendar quarter from and after the Closing Date through and including the quarter ended June 30, 2026, an amount equal to 7.5% of (a) our U.S. net sales of ZTALMY and all other pharmaceutical products that contain ganaxolone (Net Sales), in each case with any dosage form, dosing regimen, or strength, or any improvements related thereto (collectively, Included Products) and (b) certain other payments received by us in connection with the manufacture, development and sale of the Included Products in the U.S. (Other Included Payments, and, together with Net Sales, Product Revenue); and (ii) for each calendar quarter following the calendar quarter ended June 30, 2026, an amount equal to (x) 15.0% of the first $100 million in annual Product Revenue of the Included Products and (y) 7.5% of annual Product Revenue of the Included Products in excess of $100 million.

The Payments are subject to a hard cap equal to 190% of the Investment Amount (Hard Cap) or $61.8 million. Sagard’s right to receive payments will terminate when Sagard has received payments in respect of the Included Products, including any additional payments described below, equal to the Hard Cap. Further, we have the right to make voluntary prepayments to Sagard, and such payments will be credited against the Hard Cap.

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The obligations under the Revenue Interest Financing Agreement, including the Payments, will be guaranteed by certain of our future subsidiaries that are required to become a party thereto as guarantors (Guarantors). Our obligations under the Revenue Interest Financing Agreement and the guarantee of such obligations are secured, subject to customary permitted liens and other agreed upon exceptions and subject to an intercreditor agreement with Oaktree as administrative agent for the lenders under our credit agreement (as described below, the Credit Agreement), by a pledge of substantially all of our and the Guarantors’ assets that relate to, or are used or held for use for, the development, manufacture, use and/or commercialization of ZTALMY and all other pharmaceutical products that contain ganaxolone in the U.S., including the Product Revenue, pursuant to the terms of the Security Agreement dated as of the Closing Date by and among us, the Guarantors from time to time party thereto, and Sagard (Security Agreement).

At any time, we have the right, but not the obligation (Call Option), to repurchase all, but not less than all, of Sagard’s interest in the Payments at a repurchase price (Put/Call Price) equal to: (a) on or before the third anniversary of the Closing Date, 160% of the Investment Amount; (b) after the third anniversary but on or prior to the fourth anniversary of the Closing Date, 180% of the Investment Amount; and (c) after the fourth anniversary of the Closing Date, 190% of the Investment Amount, in each case, less the aggregate of all of our payments in respect of the Payments made to Sagard prior to such date.

The Revenue Interest Financing Agreement contains certain restrictions on our and our subsidiaries’ abilities, among other things, to incur additional debt, grant or permit additional liens, make investments and acquisitions, dispose of assets, pay dividends and distributions and enter into affiliate transactions, in each case, subject to certain exceptions. In addition, the Revenue Interest Financing Agreement contains a financial covenant that requires us to maintain at all times cash and cash equivalents in certain deposit accounts in an amount at least equal to (i) from the Closing Date until the repayment of the loans under the Credit Agreement, $15.0 million and (ii) thereafter, $10.0 million.

In connection with the Revenue Interest Financing Agreement, on the Closing Date, we entered into an amendment to the Credit Agreement with Oaktree, which is fully described in Note 10.

Issuance costs pursuant to the Revenue Interest Financing Agreement consisted primarily of advisory and legal fees and totaled $2.6 million. These issuance costs were recorded as a direct deduction to the carrying amount of the liability and will be amortized under the effective interest method over the estimated period the liability will be repaid. For the nine months ended September 30, 2023, we estimated an effective annual interest rate of approximately 18%. Over the course of the Revenue Interest Financing Agreement, the actual interest rate will be affected by the amount and timing of net ZTALMY revenue recognized and changes in the timing of forecasted net ZTALMY revenue. On a quarterly basis, we will reassess the expected timing of the net ZTALMY revenue, recalculate the amortization and effective interest rate and adjust the accounting prospectively as needed.

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The following table summarizes the activity of the Revenue Interest Financing Agreement for the nine months ended September 30, 2023 (in thousands):



Revenue Interest Financing Balance at December 31, 2022	$	30,877
Non-cash interest expense in the nine months ended September 30, 2023		4,421
Amortization of debt discount in the nine months ended September 30, 2023		194
Payments made in the nine months ended September 30, 2023		(736)
Revenue Interest Financing Balance at September 30, 2023	$	34,756

Current portion of revenue interest financing liability	$	1,901
Long-term portion of revenue interest financing liability		32,855
Revenue Interest Financing Balance at September 30, 2023	$	34,756

12. Collaboration Revenue

Orion Collaboration Agreement

In July 2021, we entered into a collaboration agreement ~~(the Orion~~(Orion Collaboration Agreement) with ~~Orion Corporation (Orion).~~Orion. The Orion Collaboration Agreement falls under the scope of ASC Topic 808, Collaborative Arrangements (ASC 808) as both parties are active participants in the arrangement that are exposed to significant risks and rewards. While this arrangement is in the scope of ASC 808, we analogize to ASC 606 for some aspects of this arrangement, including for the delivery of a good or service (i.e., a unit of account). Revenue recognized by analogizing to ASC 606 is recorded as collaboration revenue on the consolidated statements of operations.

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Under the terms of the Orion Collaboration Agreement, we granted Orion an exclusive, royalty-bearing, sublicensable license to certain of our intellectual property rights with respect to commercializing biopharmaceutical products incorporating our product candidate ganaxolone (Licensed Products) in the European Economic Area, the United Kingdom and Switzerland (collectively, the Territory) for the diagnosis, prevention and treatment of certain human diseases, disorders or conditions (Field), initially in the indications of CDD, TSC and RSE. We will be responsible for the continued development of Licensed Products and regulatory interactions related thereto, including conducting and sponsoring all clinical trials, provided that Orion may conduct certain post-approval studies in the Territory. Orion will be responsible, at Orion’s sole cost and expense, for the commercialization of any Licensed Product in the Field in the Territory.

Under the terms of the Orion Collaboration Agreement, we received a €25.0 million ($29.6 million) upfront payment from Orion in July 2021. In connection with the upfront fee, we agreed to provide Orion with the results of a planned genotoxicity study on the M2 metabolite of ganaxolone, a “Combined Micronucleus & Comet study in vivo.” In May 2022, the final study report was received, which confirmed that no genotoxicity was found, as measured by formation of micronuclei in the bone marrow or comet morphology in the liver. In the event that the results of the study were positive, based on the criteria set forth in the study’s protocol, Orion would have had the right to terminate the Orion Collaboration Agreement within ninety (90) days after its receipt of the final study report, and we would have been required to refund Orion seventy-five percent (75%) of the upfront fee. We are eligible to receive up to an additional €97 million in research and development reimbursement and cash milestone payments based on specific clinical and commercial achievements, as well as tiered royalty payments based on net sales ranging from the low double-digits to high teens for the oral programs and the low double-digits to low 20s for the IV program. Also, as part of the overall arrangement, we have agreed to supply the Licensed Products to Orion at an agreed upon price.

The Orion Collaboration Agreement shall remain effective until the date of expiration of the last to expire Royalty Term, which is defined as the period beginning on the date of the first commercial sale Licensed Product in such country and ending on the latest to occur of (a) the tenth (10th) anniversary of the first commercial sale of Licensed Product in such country, (b) the expiration of the last-to-expire licensed patent covering the manufacture, use or sale of such Licensed Product in such country, and (c) the expiration of regulatory exclusivity period, if any, for such Licensed

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Product in such country. The Orion Collaboration Agreement has a term of at least ten (10) years since a commercial sale has yet to occur. The Orion Collaboration Agreement allows for termination in certain specific events, such as material breach, in the event Orion challenges the validity, enforceability or scope of the licensed patent rights, termination for forecast failure, insolvency and force majeure, none of which are probable at contract inception.

In accordance with the guidance, we identified the following commitments under the arrangement: (i) exclusive rights to develop, use, sell, have sold, offer for sale and import any product comprised of Licensed Product (License), (ii) development and regulatory activities (Development and Regulatory Activities), and (iii) requirement to supply Orion with the Licensed Product at an agreed upon price (Supply of Licensed Product). We determined that these three commitments represent distinct performance obligations for purposes of recognizing revenue or reducing expense, which we will recognize such revenue or expense, as applicable, as we fulfill these performance obligations.

At contract inception, we determined that the non-refundable portion of the upfront payment plus the research and development reimbursement constitutes the transaction price as of the outset of the Orion Collaboration Agreement. The refundable portion of the upfront payment and the future potential regulatory and development milestone payments were fully constrained at contract inception as the risk of significant revenue reversal related to these amounts had not yet been resolved. During ~~the nine months ended September 30,~~ 2022, the refundable portion of the upfront payment was determined to be included in the transaction price as the final genotoxicity study on the M2 metabolite of ganaxolone was received as described ~~above.~~above, and the remaining $12.7 million of the upfront payment was recorded as collaboration revenue in the year ended December 31, 2022. The achievement of the future potential milestones is not within our control and is subject to certain research and development success and therefore ~~carry~~carries significant uncertainty. As a result of the July 2023 EC approval of ZTALMY oral suspension for the adjunctive treatment of epileptic seizures associated with CDD in patients two to 17 years of age, we are now eligible under the Orion Collaboration Agreement to receive a commercial milestone payment of 10 million Euro, if commercial sales of ZTALMY commence in the Territory, due upon the earlier of (1) the first commercial sale of ZTALMY within two of a select set of countries consisting of Germany, France, Italy, Spain, and the United Kingdom or (2) the 18-month anniversary of the first commercial sale of ZTALMY in the Territory. We will reevaluate the likelihood of achieving these milestones at the end of each reporting period and adjust the transaction price in the period the risk is resolved. In addition, we will recognize any consideration related to sales-based milestones and royalties when the subsequent sales occur since those payments relate primarily to the License, which was delivered by us to Orion upon entering into the Orion Collaboration Agreement. ~~We recorded~~

$12.7 million of the $21.2 million refundable portion of the upfront payment as collaboration revenue in the nine months ended September 30, 2022, and $9.5 million was recorded as a long-term liability as of September 30, 2022.

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The transaction price was allocated to the three performance obligations based on the estimated stand-alone selling prices at contract inception. The stand-alone selling price of the License was based on a discounted cash flow approach and considered several factors including, but not limited to, discount rate, development timeline, regulatory risks, estimated market demand and future revenue potential using an adjusted market approach. The stand-alone selling price of the Development and Regulatory Activities and the Supply of Licensed Product was estimated using the expected cost-plus margin approach.

As of ~~the agreement date in July 2021,~~December 31, 2022, we allocated the transaction price to the performance obligations as described below and recorded the ~~$9.0~~remaining $12.7 million ~~transaction price associated with~~of the ~~License~~upfront payment as ~~revenue.~~collaboration revenue during the year ended December 31, 2022. During ~~2021,~~2022, we amortized ~~$0.1~~$1.1 million of the transaction price associated with the Development and Regulatory Services as a reduction of research and development costs. These reductions to transaction price resulted in a total contract liability of ~~$6.6~~$15.1 million atas of December 31, ~~2021.~~2022. In accordance with ASC 210-20, the contract liability of ~~$6.6~~$15.1 million was offset by a contract asset of ~~$7.2~~$5.1 million related to the reimbursement of research and development costs, resulting in a net contract ~~asset~~liability of ~~$0.6~~$10.0 million atas of December 31, ~~2021.~~2022.

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Transaction Price and Net Contract ~~Asset at~~Liability as of December 31, ~~2021:~~2022:


			Cumulative Collaboration						Cumulative Collaboration
	Transaction		Revenue Recognized		Contract		Transaction		Revenue Recognized		Contract
	Price		as of December 31, 2021		Liability		Price		as of December 31, 2022		Liability
License	$	8,987	$	8,987	$	-	$	21,660	$	21,660	$	-
Development and Regulatory Services		2,787		106		2,681		6,717		1,158		5,559
Supply of Licensed Product		3,943		-		3,943		9,503		-		9,503
	$	15,717	$	9,093		6,624	$	37,880	$	22,818	$	15,062

Less Total Contract Asset						7,181						5,079
Net Contract Asset					$	557
Net Contract Liability											$	9,983

During the nine months ended September 30, 2022, the refundable portion of the upfront payment was determined to be included in the transaction price as the final genotoxicity study on the M2 metabolite of ganaxolone was received as described above. As such, the refundable portion of the upfront payment of €18.8 million ($21.2 million) was allocated to the purchase price as shown below, resulting in a total purchase price of $37.9 million. Of the $21.2 million refundable portion of the upfront payment, we recorded $12.7 million of collaboration revenue in the nine months ended September 30, 2022. During the nine months ended September 30, 2022,2023, we amortized ~~$1.1~~$1.2 million of the transaction price associated with the Development and Regulatory Services as a reduction of research and development costs. These reductions to the transaction price resulted in a total contract liability of ~~$15.1~~$13.8 million atas of September 30, ~~2022.~~2023. In accordance with ASC 210-20, the contract liability of ~~$15.1~~$13.8 million is offset by a contract asset of ~~$5.6~~$3.5 million related to the reimbursement of research and development costs, resulting in a net contract liability of ~~$9.5~~$10.3 million atas of September 30, ~~2022.~~2023.

Transaction Price and Net Contract Liability atas of September 30, ~~2022:~~2023:


			Cumulative Collaboration						Cumulative Collaboration
	Transaction		Revenue Recognized		Contract		Transaction		Revenue Recognized		Contract
	Price		as of September 30, 2022		Liability		Price		as of September 30, 2023		Liability
License	$	21,660	$	21,660	$	-	$	21,660	$	21,660	$	-
Development and Regulatory Services		6,717		1,106		5,611		6,717		2,417		4,300
Supply of Licensed Product		9,503		-		9,503		9,503		-		9,503
	$	37,880	$	22,766		15,114	$	37,880	$	24,077	$	13,803

Less Total Contract Asset						5,615						3,464
Net Contract Liability					$	9,499					$	10,339

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~~In 2021, we~~We incurred $2.0 million of incremental costs in connection with obtaining the Orion Collaboration Agreement. These contract acquisition costs were allocated consistent with the transaction price, resulting in $1.1 million of expense recorded to selling, general and administrative expense commensurate with the recognition of the License performance obligation and $0.9 million recorded as capitalized contract costs, included in other current assets and other assets, which are being amortized as Development and Regulatory Services and Supply of Licensed Product obligations are met. Cost of collaboration revenue of $1.5 million for the three and nine months ended September 30, 2021 represents a one-time fee paid to Purdue Neuroscience Company (Purdue) related to that certain license agreement entered into in September 2004, and subsequently amended and restated in May 2008, between us and Purdue, and was paid in conjunction with the Orion Collaboration Agreement.

We reevaluate the transaction price and the total estimated costs expected to be incurred to satisfy the performance obligations and ~~adjusts~~adjust the deferred revenue at the end of each reporting period. Such changes will result in a change to the amount of collaboration revenue recognized and deferred revenue.

~~11. Income taxes~~Tenacia Collaboration Agreement

~~We account for income taxes under the liability method in accordance with ASC Topic 740,~~ ~~Income Taxes~~. Deferred tax assets and liabilities are determined based on the difference between the financial statement and tax bases of assets and liabilities as measured by the enacted tax rates which will be in effect when these differences reverse. Deferred tax expense (benefit) is the result of changes in deferred tax assets and liabilities. Deferred income tax assets and liabilities are adjusted to recognize the effects of changes in tax laws or enacted tax rates in the period during which they are signed into law.

The Tax Cuts and Jobs Act passed in 2017 included a provision which would require taxpayers to capitalize and amortize U.S.-based research & experimentation (R&E) expenses over a period of five years and non-U.S. R&E expenses over 15 years effective for tax years beginning after December 31, 2021 pursuant to Internal Revenue Code Section 174. As a result of the capitalization of R&E expenses, income generated from the sale of the PRV, ~~and limitations related to the utilization of state net operating losses,~~ ~~we have a current income tax expense of $1.8~~ ~~million for the period ended September 30,~~On November 16, 2022 attributable to state income taxes. Although Congress is considering legislation that would defer the amortization requirement to later years, we have no assurance that the provision will be repealed or otherwise modified. As of September 30, 2022, we do not estimate a federal income tax liability due to the utilization of Net Operating Losses (NOLs) after taking into consideration Internal Revenue Code Section 382 limitations related to changes in ownership.

We have significant deferred tax assets, a substantial amount of which result from operating loss carryforwards. We routinely evaluate our ability to realize the benefits of these assets to determine whether it is more likely than not that such benefit will be realized. Based on the history of losses generated, we believe as of September 30, 2022, it is more likely than not that our remaining deferred tax assets will not be realized. Accordingly, we maintain a full valuation allowance on our net deferred tax assets.

~~12. Subsequent Events~~

~~On October 28, 2022 (the Closing~~(Effective Date), we entered into a ~~revenue interest financing agreement (the Revenue Interest Financing~~Collaboration and Supply Agreement (Tenacia Collaboration Agreement) with ~~Sagard Healthcare Royalty Partners, LP (Sagard)~~ ~~pursuant~~Tenacia Biotechnology (Shanghai) Co., Ltd. (Tenacia). The Tenacia Collaboration Agreement falls under the scope of ASC Topic 808, Collaborative Arrangements (ASC 808) as both parties are active participants in the arrangement that are exposed to ~~which~~significant risks and rewards. While this arrangement is in the scope of ASC 808, we ~~received $32.5 million (the Investment Amount)~~ analogize to ~~provide funding~~ASC 606 for ~~our development and commercialization~~some aspects of ~~ganaxolone and related pharmaceutical products,~~this arrangement, including ~~the commercial launch of ZTALMY, and for working capital and general administrative purposes.~~

~~In exchange~~ for the ~~Investment Amount, we have agreed~~delivery of a good or service (i.e., a unit of account). Revenue recognized by analogizing to ~~make quarterly payments to Sagard (the Payments)~~ASC 606 is recorded as ~~follows: (i) for each calendar quarter from and after~~collaboration revenue on the ~~Closing Date through and including the quarter ended June 30, 2026, an amount equal to 7.5%~~consolidated statements of (a) our U.S. net sales of ZTALMY and all other pharmaceutical products that contain ganaxolone (Net Sales), in each case with any dosage form, dosing regimen, or strength, or any improvements related thereto (collectively, the Included Products) and (b) certain other payments received by us in connection with theoperations.

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Under the terms of the Tenacia Collaboration Agreement, we granted Tenacia an exclusive, royalty-bearing, sublicensable license to certain of our intellectual property rights to develop, commercialize and otherwise exploit certain products incorporating certain oral and intravenous formulations of our product candidate ganaxolone (Licensed Products) in Mainland China, Hong Kong, Macau and Taiwan (collectively, Territory) for the diagnosis, prevention and treatment of certain human diseases, disorders or conditions (Field), initially for the treatment of cyclin-dependent kinase-like 5 deficiency disorder, tuberous sclerosis complex and SE (including RSE) (collectively, the Initial Indications). The collaboration can be expanded to include additional indications and formulations of ganaxolone pursuant to a right of first negotiation.

Under the terms of the Tenacia Collaboration Agreement, Tenacia agreed to pay us an upfront cash payment of $10 million (the Upfront Fee) within ~~manufacture,~~forty-five (45) days after the Effective Date, which was paid in December 2022. In addition to the Upfront Fee, Tenacia has agreed to make cash payments to us upon the achievement of certain development, regulatory and sales-based milestones related to (i) the Initial Indications and (ii) the first new formulation or pro-drug of ganaxolone or any back-up compound of ganaxolone in a new indication (Selected Product) for which the parties amend the Tenacia Collaboration Agreement in connection with Tenacia’s exercise of its right of first negotiation and for which there is no other Licensed Product approved in China (for clarity, the milestone payments under this clause (ii) will only apply to one Selected Product), up to an aggregate amount of $256 million. Of the milestones, $15 million relates to regulatory approvals with separate milestones related to each of oral and intravenous formulations and the Selected Product, and an aggregate of $241 million of sales-based milestones are connected to annual revenue thresholds specific to each of the oral, intravenous and Selected Product formulations of ganaxolone. Tenacia has further agreed to pay us tiered royalty payments based on annual net sales of Licensed Products ranging from the low double digits to the mid-teens for each of the oral formulation, intravenous formulation and Selected Product formulation of Licensed Products. Tenacia’s obligations to pay royalties to us with respect to sales of a Licensed Product in each particular jurisdiction of the Territory will commence on the date of first commercial sale in such jurisdiction and expire upon the latest of (i) ten years following the first commercial sale of such Licensed Product in such jurisdiction, (ii) the ~~Included~~expiration of the last-to-expire valid claim of any licensed patent rights that covers such Licensed Product in such jurisdiction and (iii) the expiration of all regulatory exclusivities for such Licensed Product in such jurisdiction. Royalty payments are subject to reduction in specified circumstances as set forth in the Tenacia Collaboration Agreement, including if net sales decrease by a certain percentage after the introduction of a generic product.

Tenacia will be primarily responsible for the development of Licensed Products in the ~~U.S. (the Other Included Payments,~~Territory and ~~together~~regulatory interactions related thereto, including conducting and sponsoring clinical studies in the Field in the Territory to support regulatory filings in the Territory. All regulatory approvals filed by Tenacia in the Territory will be in the name of and owned by us unless otherwise required by applicable law, in which case such regulatory approvals would be in the name of and owned by Tenacia for the benefit of us. We and Tenacia agreed to enter into clinical and commercial supply agreements pursuant to which we will supply Tenacia with ~~Net Sales,~~its requirements of Licensed Products necessary for Tenacia to develop and commercialize Licensed Products in the Field in the Territory. The parties entered into the clinical and commercial supply agreement in May 2023. The agreement contains pricing, delivery, acceptance, payment, termination, forecasting, and other terms consistent with the Tenacia Collaboration Agreement, as well as certain quality assurance, indemnification, liability and other standard industry terms. Tenacia will be responsible for, at Tenacia’s sole cost and expense, obtaining regulatory approval and commercializing the Licensed Product ~~Revenue); and (ii) for each calendar quarter following~~in the ~~calendar quarter ended June 30, 2026, an amount equal to (x) 15.0%~~Field in Mainland China. Tenacia is enrolling patients in our Phase 3 randomized, double blind, placebo-controlled trial (TrustTSC trial) of adjunctive ganaxolone.

The term of the ~~first $100 million~~Tenacia Collaboration Agreement extends for so long as royalties are payable anywhere in ~~annual Product Revenue~~the Territory. Subject to the terms of the ~~Included Products and (y) 7.5% of annual Product Revenue of the Included Products in excess of $100 million.~~

The Payments are subject to a hard cap equal to 190% of the Investment Amount (the Hard Cap). Sagard’s right to receive payments will terminate when Sagard has received payments in respect of the Included Products, including any additional payments described below, equal to the Hard Cap. Further, we have the right to make voluntary prepayments to Sagard, and such payments will be credited against the Hard Cap.

If Sagard has not received aggregate payments equaling at least 100% of the Investment Amount by December 31, 2027 or at least 190% of the Investment Amount by December 31, 2032 (each, a Minimum Amount), then we will be obligated to make a cash payment to Sagard in an amount sufficient to gross up Sagard up to the applicable Minimum Amount withinTenacia Collaboration Agreement, (i) for a specified period of time after ~~each reference date.~~the Effective Date, Tenacia may terminate the Tenacia Collaboration Agreement in its entirety for any or no reason upon written notice to us, and (ii) either party may terminate the Tenacia Collaboration Agreement for the other party’s material breach following a cure period or insolvency.

~~The obligations~~In accordance with the guidance, we identified the following commitments under the ~~Revenue Interest Financing Agreement, including~~arrangement: (i) grant to Tenacia the ~~Payments, will be guaranteed by certain of our future subsidiaries that are required~~exclusive rights to ~~become a party thereto as guarantors (the Guarantors). Our obligations under~~develop, commercialize and otherwise exploit Licensed Product in the ~~Revenue Interest Financing Agreement~~Field in the Territory (License) and (ii) requirement to supply Tenacia with the ~~guarantee of such obligations are secured, subject to customary permitted liens and other~~Licensed Product at an agreed upon ~~exceptions and subject to an intercreditor agreement with Oaktree as administrative agent~~price (Supply of Licensed Product). We determined that these two commitments represent distinct performance obligations for the lenders under our credit agreement (as described below, the Credit Agreement), by a pledge of substantially all of our and the Guarantors’ assets that relate to, or are used or held for use for, the development, manufacture, use and/or commercialization of ZTALMY and all other pharmaceutical products that contain ganaxolone in the United States, including the Product Revenue, pursuant to the terms of the Security Agreement dated as of the Closing Date by and among us, the Guarantors from time to time party thereto, and Sagard (the Security Agreement).

At any time, we have the right, but not the obligation (the Call Option), to repurchase all, but not less than all, of Sagard’s interest in the Payments at a repurchase price (the Put/Call Price) equal to: (a) on or before the third anniversary of the Closing Date, 160% of the Investment Amount; (b) after the third anniversary but on or prior to the fourth anniversary of the Closing Date, 180% of the Investment Amount; and (c) after the fourth anniversary of the Closing Date, 190% of the Investment Amount, in each case, less the aggregate of all of our payments in respect of the Payments made to Sagard prior to such date.

In connection with the Revenue Interest Financing Agreement, on the Closing Date, we entered into that certain Limited Consent and First Amendment to Credit Agreement (the Credit Agreement Amendment) to our previously disclosed Credit Agreement and Guaranty dated as of May 11, 2021 with Oaktree, as the administrative agent (the Administrative Agent) and the lenders party thereto (as amended by that certain Letter Agreement re: Minimum Liquidity Amount dated May 17, 2021 and as further amended by that certain Amendment dated May 23, 2022, the Credit Agreement) to, among other things, allow for the consummation of the Revenue Interest Financing Agreement and the transactions thereunder. In addition, the Credit Agreement Amendment increases the exit fee due by us upon any repayment, whether as a prepayment or a scheduled repayment, of the principal of the loans under the Credit Agreement from 2.00% to 2.67%.

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of recognizing revenue or reducing expense, which it will recognize such revenue or expense, as applicable, as it fulfills these performance obligations.

The transaction price was allocated to the two performance obligations based on the estimated stand-alone selling prices at contract inception. The stand-alone selling price of the License was based on a discounted cash flow approach and considered several factors including, but not limited to, discount rate, development timeline, regulatory risks, estimated market demand and future revenue potential using an adjusted market approach. The stand-alone selling price of the Supply of Licensed Product was estimated using the expected cost-plus margin approach.

As of December 31, 2022, we allocated the transaction price to the performance obligations as described below. There was no activity in the three and nine months ended September 30, 2023. The cumulative collaboration revenue recognized as of September 30, 2023 is $3.0 million, which was the $3.0 million transaction price associated with the License as revenue for the year ended December 31, 2022. No license revenue was recorded in the three and nine months ended September 30, 2023. There was a total contract liability of $7.0 million as of both September 30, 2023 and December 31, 2022. In accordance with ASC 210-20, the contract liability of $7.0 million is offset by a contract asset of $0.7 million, resulting in a net contract liability of $6.3 million as of both September 30, 2023 and December 31, 2022.

Transaction Price and Net Contract Liability as of September 30, 2023 and December 31, 2022:


			Cumulative Collaboration
	Transaction		Revenue Recognized		Contract
	Price		as of September 30, 2023 and December 31, 2022		Liability
License	$	2,998	$	2,998	$	-
Supply of Licensed Product		7,002		-		7,002
	$	10,000	$	2,998	$	7,002

Less Total Contract Asset						700
Net Contract Liability					$	6,302

In December 2022, we incurred $1.0 million of incremental costs in obtaining the Tenacia Collaboration Agreement. These contract acquisition costs were allocated consistent with the transaction price, resulting in $0.1 million of expense recorded to selling, general and administrative expense and $0.2 million recorded to cost of collaboration revenue in the period ended December 31, 2022, commensurate with the recognition of the License performance obligation, and $0.7 million recorded as capitalized contract costs, which will be amortized as Supply of License Product obligations are met.

We reevaluate the transaction price and the total estimated costs expected to be incurred to satisfy the performance obligations and adjust the deferred revenue at the end of each reporting period. Such changes will result in a change to the amount of collaboration revenue recognized and deferred revenue.

Biologix Distribution and Supply Agreement

In May 2023, we entered into an exclusive distribution and supply agreement (Biologix Agreement) with Biologix FZCo (Biologix), whereby Biologix has the right to distribute and sell ganaxolone inAlgeria, Bahrain, Egypt, Iraq, Jordan, Kingdom of Saudi Arabia, Kuwait, Lebanon, Libya, Morocco, Oman, Qatar, Tunisia and United Arab Emirates. In exchange for distribution rights, we will be the exclusive supplier of our products to Biologix on terms set forth in the respective agreements in exchange for a negotiated purchase price for the products. Upon execution of the Biologix Agreement, we received an upfront payment of $0.5 million which is to be recognized over the term of the agreement. We may be entitled to additional fees upon regulatory milestones. In the three and nine months ended September 30, 2023, we recorded less than $0.1 million of collaboration revenue related to the Biologix Agreement. There was a total contract liability of $0.5 million at September 30, 2023. As the Biologix Agreement was entered into in May 2023, there was no contract liability at December 31, 2022.

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Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations

Cautionary Note Regarding Forward-Looking Statements

This Quarterly Report on Form 10-Q contains forward-looking statements, within the meaning of the U.S. Private Securities Litigation Reform Act of 1995, that involve substantial risks and uncertainties. In some cases, you can identify forward-looking statements by the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “objective,” “ongoing,” “plan,” “predict,” “project,” “potential,” “should,” “will,” or “would,” and or the negative of these terms, or other comparable terminology intended to identify statements about the future. These statements involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to be materially different from the information expressed or implied by these forward-looking statements. Although we believe that we have a reasonable basis for each forward-looking statement contained in this Quarterly Report on Form 10-Q, we caution you that these statements are based on a combination of facts and factors currently known by us and our expectations of the future, about which we cannot be certain.

The forward-looking statements in this Quarterly Report on Form 10-Q include, among other things, statements about:

●

our plans to continue to successfully commercialize ganaxolone in Cyclin-dependent Kinase-like 5 Deficiency Disorder (CDD) in the ~~United States;~~U.S.;

●

our plans to meet our post-approval commitments to the U.S. Food and Drug Administration (FDA) for ganaxolone;

●

our ~~plans to achieve regulatory approval for~~expectations regarding the commercialization of ganaxolone in the ~~European Union (EU), and~~EU, including the ~~expected~~ timing thereof;

●

our ability to develop ganaxolone for additional indications, including Refractory Status Epilepticus (RSE)~~, Established Status Epilepticus (ESE)~~, Tuberous Sclerosis Complex (TSC) and Lennox Gastaut Syndrome (LGS);

●

the status, timing and results of preclinical studies and clinical trials;

●

the design of and enrollment in clinical trials, availability of data from ongoing clinical trials, expectations for regulatory approvals, orand the attainment of clinical trial results that will be supportive of regulatory approvals;

●

the potential benefits of ganaxolone, including in indications other than CDD;

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the timing of seeking marketing approval of ganaxolone in specific additional indications;

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our ability to maintain marketing approval for ganaxolone in CDD and obtain regulatory approval for ganaxolone in other indications;

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the possibility that we expand the targeted indication footprint and explore new potential formulations of ganaxolone;

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our estimates of expenses and future revenue and profitability;

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our estimates regarding our capital requirements and our needs for additional financing;

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our estimates of the size of the potential markets for ganaxolone;

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our expectations regarding our ~~collaboration~~collaborations with Orion Corporation (Orion), Tenacia Biotechnology (Shanghai) Co., Ltd. (Tenacia), and Biologix FZCo (Biologix) including the expected ~~amount~~amounts and ~~timing~~timings of milestone, royalty and other payments, including research and development reimbursement ~~milestone, royalty and other payments~~if applicable, pursuant thereto;

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our ability to attract collaborators with acceptable development, regulatory and commercial expertise;

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the benefits ~~to be~~and contractual requirements derived from corporate collaborations, license agreements, and other collaborative or acquisition efforts, including those relating to the development and commercialization of ganaxolone;

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sources of revenue, including expected future sales of ganaxolone in CDD, revenue contributions from our contract (BARDA Contract) with the Biomedical Advanced Research and Development Authority (BARDA), corporate collaborations, license agreements, and other collaborative efforts for the development and commercialization of ganaxolone for CDD and in other indications being developed for ganaxolone;

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our ~~eligibility to receive funding under the remaining debt tranche available under the Credit Agreement with Oaktree;~~

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~~our~~ ability to create and maintain an effective sales and marketing infrastructure where we elect to market and sell ganaxolone directly;

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the pricing and the timing and amount of reimbursement for ganaxolone;

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the success of other competing therapies that may become available;

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the manufacturing capacity and supply for ganaxolone;

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the possibility that third parties, such as Ovid Therapeutics, Inc. (Ovid), may initiate legal proceedings alleging that we are infringing their intellectual property rights, the outcome of which would be uncertain and could harm our business;

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our belief that the amended protocol for the Phase 3 Randomized Therapy In Status Epilepticus Trial (RAISE trial) will facilitate the enrollment of patients transferred to the intensive care unit (ICU) from other hospitals or the emergency room, who may already have received high doses of anesthetic medication for less than 18 hours;

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~~our expectation that the majority of clinical sites participating in the RAISE trial will have adopted the RAISE protocol amendment by the end of 2022;~~

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the possibility that we expand and diversify our product pipeline through acquisitions of additional drug candidates that fit our business strategy;

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our belief that our existing cash and cash equivalents will be sufficient to fund our operating expenses and capital expenditure requirements, ~~and~~as well as maintain the minimum cash balance required under our debt facility, ~~into~~for at least twelve months from the ~~first quarter of 2024, inclusive of the $32.5 million payment from Sagard Healthcare Partners;~~date these financial statements are issued;

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our ability to maintain and protect our intellectual property rights;

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our results of operations, financial condition, liquidity, prospects, and growth strategies;

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our ability to, among other actions, secure additional financing or strategic transactions and continue as a going concern;

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the extent to which our business may be adversely impacted by the effects of the COVID-19 coronavirus pandemic or by other pandemics, epidemics or outbreaks;

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the enforceability of the exclusive forum provisions in our fourth amended and restated certificate of incorporation; and

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the industry in which we operate and trends which may affect the industry or us.

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You should refer to Part II Item 1A. Risk Factors of this Quarterly Report on ~~this~~ Form 10-Q and Part I Item 1A. Risk Factors of our Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC) on March ~~24, 2022~~9, 2023 for a discussion of important factors that may cause our actual results to differ materially from those expressed or implied by our forward-looking statements. As a result of these factors, we cannot assure you that the forward-looking statements in this Quarterly Report on Form 10-Q will prove to be accurate. Furthermore, if our forward-looking statements prove to be inaccurate, the inaccuracy may be material. In light of the significant uncertainties in these forward-looking statements, you should not regard these statements as a representation or warranty by us or any other person that we will achieve our objectives and plans in any specified time frame or at all. We undertake no obligation to publicly update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

You should read this Quarterly Report on Form 10-Q and the documents that we reference in this Quarterly Report on Form 10-Q and have filed as exhibits to this Quarterly Report on Form 10-Q completely and with the understanding that our actual future results may be materially different from what we expect. We qualify all of our forward-looking statements by these cautionary statements.

The following Management’s Discussion and Analysis of Financial Condition and Results of Operations should be read in conjunction with: (i) the interim consolidated financial statements and related notes thereto which are included in this Quarterly Report on Form 10-Q; and (ii) our annual financial statements for the year ended December 31, ~~2021~~2022 which are included in our Annual Report on Form 10-K filed with the SEC on March ~~24, 2022.~~9, 2023.

Overview

We are a commercial-stage pharmaceutical company dedicated to the development of innovative therapeutics for the treatment of seizure disorders, including rare genetic epilepsies and status epilepticus. On March 18, 2022, the ~~FDA~~U.S. Food and Drug Administration (FDA) approved our new drug application (NDA) for the use of ZTALMY^® (ganaxolone) oral suspension CV for the treatment of seizures associated with ~~CDD~~Cyclin-dependent Kinase-like 5 (CDKL5) Deficiency Disorder (CDD) in patients 2two years of age and older. In June 2022, the United States Drug Enforcement Administration (DEA) published an interim final rule in the Federal Register placing ganaxolone and its salts in schedule V (CV) of the Controlled Substances Act (CSA). ZTALMY, our first FDA approved product, became available for commercial sale and shipment ~~to patients with a prescription in the U.S.~~ in the third quarter of 2022. On July 28, 2023, the European Commission (EC) granted marketing authorization for ZTALMY for the adjunctive treatment of epileptic seizures associated with CDD in patients two to 17 years of age. ZTALMY may be continued in patients 18 years of age and older. We ~~also plan to develop~~are developing ganaxolone for the treatment of other rare genetic epilepsies, including ~~TSC,~~Tuberous Sclerosis Complex (TSC), and for the treatment of ~~status epilepticus (SE)~~Refractory Status Epilepticus (RSE). ~~While the precise mechanism by which~~We are developing ganaxolone exerts its therapeutic effects in the treatment of seizures associated with CDD is unknown, its anticonvulsant effects are thought to result from positive allosteric modulation of the gamma-aminobutyric acid type A (GABA_A~~) receptor in the Central Nervous System (CNS). Ganaxolone is a synthetic analog of allopregnanolone, an endogenous neurosteroid. Ganaxolone is being developed~~ in formulations for two different routes of administration: intravenous (IV) and oral. The different formulations are intended to maximize potential therapeutic applications of ganaxolone for adult and pediatric patient populations, in both acute and chronic care, and for both ~~in-patient~~acute hospital care and ~~self-administered~~chronic at home-administration settings. While the precise mechanism by which ganaxolone exerts its therapeutic effects in the treatment of seizures is unknown, its anticonvulsant effects are thought to result from positive allosteric modulation of the gamma-aminobutyric acid type A (GABA_A) receptor in the central nervous system (CNS). Ganaxolone is a synthetic analog of allopregnanolone, an endogenous neurosteroid. Ganaxolone acts at both synaptic and extrasynaptic GABA_A receptors, a target known for its anti-seizure, antidepressant and anxiolytic potential.

COVID-19

~~The continued global spread of~~ COVID-19 ~~has impacted~~affected our clinical operations and timelines. For example, our ~~RAISE~~Randomized Therapy In Status Epilepticus (RAISE) trial for RSE is conducted in hospitals, primarily intensive care units in academic medical centers, which ~~have~~ experienced high rates of COVID-19 admissions. Several of these sites participating in the RAISE trial ~~have~~ experienced COVID-related difficulties, including staff turnover and the need to devote significant resources to patients with

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COVID-19, which ~~has~~ resulted in site initiation and enrollment delays for the RAISE trial. Given these COVID-19-related challenges and ~~our recent~~the interruption in drug supply in mid-2022, we previously adjusted our expectation for our top-line data readout for the RAISE ~~trial to the second half of 2023.~~trial. In May 2022, we resumed screening and recruitment for the RAISE trial. ~~In addition,~~We now expect our ~~ganaxolone clinical trials~~interim analysis with top-line data readout for the RAISE trial to be available in the ~~outpatient setting may be negatively impacted~~second quarter of 2024, if ~~patients and their caregivers do not want to participate while~~ the ~~COVID-19 pandemic persists. The duration and severity~~pre-defined stopping criteria from the planned interim analysis are met.

Table of ~~the pandemic and its long-term impact on our business are uncertain at this time.~~Contents

Our Products and Product Candidates

~~ZTALMY®~~ZTALMY^® (ganaxolone) oral suspension CV

ZTALMY is an oral suspension given three times per day that we have developed for the treatment of ~~CDD.~~CDD-associated seizures. ZTALMY was approved by the FDA in March 2022 for the treatment of seizures associated with CDD in patients 2 years of age and older. ~~In June 2022, the DEA published an interim final rule in the Federal Register placing ganaxolone and its salts in schedule V of the CSA.~~ ZTALMY, our first FDA approved product, became available for commercial sale and shipment ~~to patients with a prescription in the U.S.~~ in the third quarter of 2022. We recorded ZTALMY net ~~U.S.~~ product revenue ~~related to ZTALMY~~ of ~~$0.6~~$5.4 million and $13.0 million in the three and nine months ended September 30, ~~2022.~~2023, respectively. On July 28, 2023, the EC granted marketing authorization for ZTALMY for the adjunctive treatment of epileptic seizures associated with CDD in patients two to 17 years of age. ZTALMY may be continued in patients 18 years of age and older. With the EC marketing authorization granted for ZTALMY, Orion, our commercialization partner for ZTALMY in Europe, announced it has begun preparations for the launch of ZTALMY, including engaging in the required processes for obtaining pricing and reimbursement approval in the various European countries. The pricing and reimbursement process can be time-consuming and may delay Orion’s commercial launch of ZTALMY in one or more European countries.

CDD is a serious and rare genetic disorder that is caused by a mutation of the CDKL5 gene, located on the X chromosome. CDD is a severely debilitating and potentially fatal genetic condition, which occurs with an estimated frequency of 1:40,000 live births in the U.S. It predominantly affects females and is characterized by early onset, difficult to control seizures and severe neurodevelopmental impairment. The CDKL5 gene encodes proteins essential for normal brain structure and function. Most children affected by CDD have neurodevelopmental deficits such as difficulty walking, talking and taking care of themselves. Many also suffer from scoliosis, gastrointestinal dysfunction or sleep disorders. Genetic testing is available to determine if a patient has a mutation in the CDKL5 gene.

In June 2017, we were granted FDA orphan drug designation for ganaxolone for the treatment of CDD. ~~Additionally, in November 2019,~~The designation provides the ~~European Medical Agency’s (EMA) Committee~~drug developer with a seven-year period of U.S. marketing exclusivity, as well as tax credits for ~~Orphan Medicinal Products (COMP)~~clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance and waiver of Prescription Drug User Fee Act filing fees. In July 2020, the FDA granted ~~orphan drug designation~~Rare Pediatric Disease Designation (RPD Designation) for ganaxolone for the treatment of ~~CDD. In July 2020, the FDA granted RPD Designation for ganaxolone for the treatment of~~ CDD. The FDA grants RPD Designation for diseases that affect fewer than 200,000 people in the U.S. and in which the serious or life-threatening manifestations occur primarily in individuals 18 years of age and younger. ~~The~~Upon FDA approval of ZTALMY in CDD in March 2022, the FDA awarded us a Rare Pediatric Disease Priority Review Voucher (PRV), which we monetized in August 2022 for $110.0 million in cash.

In November 2019, the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) granted orphan drug designation for ganaxolone for the treatment of CDD. Prior to the grant of the marketing authorization, the COMP is required to determine whether the orphan drug designation criteria are still met. On May 26, 2023, the COMP provided a positive opinion to maintain the orphan drug designation for ganaxolone in CDD in the EU.

The US and EC approvals of ZTALMY in CDD are based on data from ~~the~~a Phase 3 ~~Marigold~~ double-blind placebo-controlled trial (Marigold Study), in which 101 patients were randomized and treated with ZTALMY. ~~Patients~~Clinical trial patients receiving ZTALMY showed a median 30.7% reduction in 28-day major motor seizure frequency, compared to a median 6.9% reduction for those receiving placebo, achieving the trial’s primary endpoint (p=0.0036). InAt two years in the ~~Marigold~~ open label extension ~~study,~~phase of the Marigold Study, patients (n=50) treated with ZTALMY ~~for at least 12 months (n=48)~~ experienced a median ~~49.6%~~48.2% reduction in major motor seizure frequency. ~~On October 13~~^, 2022, we presented two posters at the Child Neurology Society Meeting from our Phase 3 Marigold clinical trial of ZTALMY, including open label extension data showing continued seizure reduction over a two-year period. Although 24-month data was available for only 16 of the 54 patients remaining in the open-label extension, the reduction in Major Motor Seizure Frequency for this subset continued to improve, with a median reduction of 53% at the 24-month mark versus a 30.7% reduction at the conclusion of the double-blind phase. The discontinuation rate was about 30% during the first year of the open label phase but declined to about 10% during the second. These data ~~in total~~ suggest that patients who remain on treatment long-term may demonstrate continued reductions in seizure frequency. ~~In the clinical development program, ZTALMY demonstrated efficacy, safety and tolerability with the~~The most common adverse ~~reactions (AEs) (incidence >5% and at least twice the rate of placebo)~~events in the ~~ZTALMY~~double-blind portion of the Marigold Study were somnolence (36.0% in the ganaxolone group ~~being somnolence,~~compared to 15.7% in the placebo group), pyrexia (18.0% and 7.8%, respectively) and salivary hypersecretion (6.0% and ~~seasonal allergy.~~2.0%, respectively).

We own families of patents and pending patent applications that claim certain formulations of ganaxolone and cover ~~ZTALMY, and~~ certain therapeutic uses of ganaxolone, including for treating CDD. The 20-year terms for patents, and applications that issue as patents, in these families run from 2026 through 2042, absent any available patent term adjustments or extensions. We have also licensed from Ovid certain patents that claim certain therapeutic uses of ganaxolone for the treatment of CDD. The licensed patents include a granted U.S. patent, and pending applications in the U.S. and Europe.

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The 20-year term for these licensed patents and applications that issue as patents will run through 2037, absent anany available patent term adjustments.

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~~Priority Review Voucher.~~ ~~As a result of the RPD Designation for ganaxolone for the treatment of~~ CDD, the FDA awarded us a PRV on March 18, 2022 in connection with the approval of the use of ZTALMY in CDD. On July 13, 2022, we entered into an asset purchase agreement (the PRV Asset Purchase Agreement) with Novo Nordisk Inc., pursuant to which we agreed to sell the PRV to Novo Nordisk, Inc. for $110.0 million, payable in cash, upon the closing of the transaction. In August 2022, the transaction closed and we received $110.0 million from Novo Nordisk, Inc. We intend to use the proceeds from the sale of the PRV for general corporate purposes, including continuing to advance our clinical pipeline and for the commercial launch of ZTALMY.

U.S. Commercial Strategy. Since ZTALMY was approved by the FDA, we have been focused on the implementation and execution of an integrated launch plan to make ZTALMY available to U.S. CDD patients through a specialty pharmacy. Key launch strategies have included and continue to include: (1) establishing our supply chain network and quality management system to assure product is available to patients; (2) driving clinical awareness of ZTALMY as the first and only FDA approved product indicated specifically for seizures associated with CDD; (3) deploying our field sales force to target physicians who treat this rare pediatric patient population; (4) engaging commercial and government payers with the objective of obtaining insurance coverage; and (5) developing our internal capabilities (such as Finance, Human Resources, Information Technology, Data Analytics and Compliance) to support our first launch as a commercial company.

U.S. Marketing Strategy. Our marketing strategy is to reinforce that seizures are central to the constellation of CDD symptoms, establish ZTALMY as central to the comprehensive management of seizures associated with CDD, and ensure that patients have seamless access to ZTALMY from prescription through fulfillment. Our ~~“Now Approved”~~ marketing campaign for ZTALMY is ~~live,~~active, and our integrated commercial launch activities initiated in the third quarter of 2022.

U.S. Sales Strategy. Our U.S. commercial sales ~~organization is in place, including~~force includes 16 regional account managers experienced in rare ~~disease as our commercial sales force.~~disease. Our field force is targeting identified key accounts and centers of excellence for CDD. Based on our market research, we estimate the addressable patient population for ZTALMY in CDD in the U.S. is approximately 2,000 patients. As this is the first product approved by the FDA specifically for seizures associated with CDD and the International Classification of Diseases, Tenth Revision (ICD10) code for CDD was established in ~~2020,~~2021, there is limited data available for this specific market. We have strengthened both our market access and field force teams, and both payer and customer engagement are ~~underway.~~ongoing.

U.S. Market Access. We have established a cross-functional payer and reimbursement account team with the objective of obtaining and maintaining reimbursement (coverage) of ~~ZTALMY.~~ZTALMY in the U.S. We are focusing our efforts on reimbursement from commercial payers where pharmacy benefit managers (PBMs) control the majority of commercial pharmacy-benefit lives and government payers, primarily Medicaid for the target population for CDD. We expect approximately ~~60%~~50% of the CDD patient population will access primary coverage through ~~both~~ Fee-for-Service ~~and~~or Managed Medicaid, with the remaining ~~40%~~approximately 50% accessing primary coverage through commercial ~~coverage,~~payers, with the top PBMs having significant influence. For the three and nine months ended September 30, 2022, we received over 50 CDD prescription enrollment forms, of which more than 30 were for new commercial patients not previously treated with ZTALMY. The prescribing and fulfillment process for ZTALMY is managed through ZTALMY One™, a comprehensive patient support program. Enrollment in the program offers various support and information to help caregivers and patients prescribed ZTALMY access their ZTALMY prescription and assist in determining eligibility for and access to co-pay support or free drug programs.

U.S. Specialty Pharmacy. We are utilizing Orsini Pharmaceutical Services, LLC (Orsini), a specialty pharmacy, to provide services for U.S. patients, including patient enrollment, benefit verification and investigation, prior authorization support, patient education and drug counseling, dispensing of product and shipment coordination. We recorded our first sales of ZTALMY to Orsini in the ~~three months ended September 30,~~third quarter of 2022.

U.S. Specialty Distributor. We are utilizing ASD Specialty Healthcare, LLC (ASD), a specialty distributor, to provide distribution services in the U.S. in connection with ZTALMY to institutional inpatient pharmacies, U.S. governmental customers, including any Department of Veterans Affairs or Department of Defense sites, and Kaiser Permanente facilities.

Infrastructure. We continue to enhance our internal capabilities and processes to support a commercial stage company. We have implemented a healthcare compliance program to guide our compliance with rules and regulations regarding pharmaceutical sales.

Manufacture of Commercial Supply. We have executed commercial supply agreements for ganaxolone active pharmaceutical ingredient (API) with our current manufacturer and also with our current supplier for finished bulk drug

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product. Additionally, we have executed a master supply agreement with a second API supplier to undertake certain process development activities and, ~~subsequently to~~if successful, provide commercial supplies of API and/or API intermediates.

Regulated as a Controlled Substance in the U.S. On June 1, 2022, the ~~DEA~~Drug Enforcement Administration (DEA) published an interim final rule in the Federal Register placing ganaxolone and its salts in schedule V of the ~~CSA.~~Controlled Substances Act (CSA), which rule became final December 9, 2022. Under the CSA, drugs are classified into five (5) distinct categories or schedules depending upon the drug’s acceptable medical use and the drug’s abuse or dependency potential. Schedule V is defined by the DEA as drugs with lower potential for abuse than schedule IV and consist of preparations containing limited quantities of certain narcotics. ZTALMY became available for commercial sale and shipment ~~to patients with a prescription in the U.S.~~ in the third quarter of 2022. As a controlled substance, ganaxolone is subject to the applicable CSA requirements such as registration, security, recordkeeping and reporting, storage manufacturing, distribution, importation and other requirements.

FDA Post-Marketing Requirements. In connection with ~~the~~ FDA approval of ZTALMY for CDD, we have ~~a number of~~several post-marketing commitments. The Phase 1 renal impairment study commitment was completed and submitted to the FDA in May ~~2022. The~~2022, the Phase 1 hepatic impairment study and the thorough QTc study ~~was~~were completed and ~~we expect it will be~~ submitted to the FDA in December 2022 and the ~~fourth quarter of 2022.~~extractable/leachable study results on the container closure system were submitted to the FDA in July 2023. The remaining post-marketing requirements include: ~~a phase 1 hepatic impairment study;~~ 2-year carcinogenicity studies of ganaxolone and the major human unconjugated plasma metabolite, M2, in rats; a 26-week carcinogenicity of ganaxolone in transgenic mice; a juvenile animal toxicity study of ~~the major human unconjugated plasma metabolite,~~ M2 in rats; ~~extractable/leachable study results on the container closure system;~~ a CNS distribution study of the ~~M47~~M17 metabolite in rats; and in vitro studies to assess the drug interaction potential of ~~M47~~the M17 metabolite. We expect to be able to complete these remaining required FDA studies within the requested FDA timeframe.

Managed Access Program (MAPS). We have recently initiated a global managed access program to support physician access to ZTALMY for appropriate patients with seizures associated with CDD in geographies where there is no available patient access, local regulatory criteria and program eligibility are satisfied, and we do not already have a commercial distribution relationship in place.

Marketing Authorization Application

In August 2021, the Committee for Medicinal Products for Human Use (CHMP) of the EMA granted our request for accelerated assessment of ganaxolone for the treatment of seizures associated with CDD. The marketing authorization application (MAA) for ganaxolone was submitted to the EMA on October 11, 2021 and on October 28, 2021, we received formal notification from the EMA that the CDD MAA was validated. With this validation, the EMA began its formal review of the MAA under the centralized ~~procedure~~procedure. On May 26, 2023, the CHMP adopted a positive opinion recommending approval of ZTALMY and on July 28, 2023, the EC approved ZTALMY oral suspension for the adjunctive treatment of epileptic seizures associated with CDD in patients two to 17 years of age. ZTALMY may be continued in patients 18 years of age and older. The EC decision is applicable to all 27 EU member states plus Iceland, Norway and Liechtenstein. ZTALMY is the first treatment in the EU indicated for the treatment of seizures associated with CDD.

EC Post-Authorization Measures. In connection with the EC approval of ZTALMY for CDD, we have several post-marketing authorization measures. In September 2023, the clinical study report (CSR) for Study 1042-HME-1001 and the ganaxolone Steady-State Metabolite Study report were submitted. The remaining post-marketing authorization measures include: completing the final Study 1042-CDD-3001 CSR with the open-label trial completion; participating in Study LLF001 (CANDID observational study) and providing annual updates; participating in the CDD-IPR-CDD-0 CDKL5 Deficiency Disorder International Patient Registry and providing six monthly updates; conducting a toxicity study with a sediment dwelling organism and an updated Environmental Risk Assessment; developing a sodium benzoate-free suspension and assessing the compatibility of the ~~EU, Norway, Iceland,~~oral suspension with food, drinks, enteral tubes, shake time and ~~Liechtenstein.~~

~~In February 2022,~~stand time; conducting M17 in vitro drug-drug interaction (DDI) Studies; conducting a M17 in vivo pharmacokinetic (PK) Study with Brain Penetrance; conducting a 26-Week Oral Gavage Toxicity Study of M2; conducting a M2 Embryo-fetal Development study; and conducting a 26-week Oral Gavage Carcinogenicity Study of ganaxolone and M2. The EMA also requested weight of evidence (WoE) assessments to evaluate the ~~MAA was converted to~~need for a standard review and we reached an agreement with the EMA to extend the Day 120 clock stop by three months to allow sufficient time to respond to questions received as part of the review process. In May 2022, we submitted a request for discussion to the CHMP to extend the Day 120 clock stop by an additional four months in order to allow sufficient time to conduct the non-clinical testing requested by the EMA and to respond to questions received as part of the review process. The CHMP agreed with our proposal for the extension of the Day 120 clock stop. As a result, we expect to submit complete responses to the EMA by the end of November 2022, and we expect the CHMP’s opinion on the MAA by the end of the first quarter of 2023. Further delays in the review and approval process could occur if we are not able to timely or adequately respond to all EMA requests or if the EMA does not agree that our responses are adequate to address its questions.

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carcinogenicity study in rats with ganaxolone, a 2-year carcinogenicity study in rats with M2, and a juvenile toxicity study with M2. We expect to be able complete the remaining required studies within the requested EMA timeframe.

Our Pipeline

We are developing ganaxolone in indications where there is a mechanistic rationale for ganaxolone to provide a benefit, including the following indications:

Status Epilepticus (SE)

SE is a life-threatening condition characterized by continuous, prolonged seizures or rapidly recurring seizures without intervening recovery of consciousness. If SE is not treated urgently, permanent neuronal damage may occur, which contributes to high rates of morbidity and mortality. Patients with SE who do not respond to first-line benzodiazepine treatment are classified as having ~~ESE,~~Established Status Epilepticus (ESE) and those who then progress to and subsequently fail at least one second-line antiepileptic drug (AED) are classified as having RSE. In RSE, synaptic GABA_A receptors are internalized into the neuron, resulting in decreased responsiveness to drugs such as benzodiazepines. RSE unresponsiveness to one or more second-line AEDs requires treatment with IV anesthesia to terminate seizures and prevent neuronal injury and other complications. The IV anesthetic is increased to a level that induces deep coma and is maintained at that rate for 24 hours or more. SE that recurs following an attempted wean of IV anesthesia is classified as super refractory status epilepticus (SRSE). In April 2016, we were granted FDA orphan drug designation for the IV formulation of ganaxolone for the treatment of SE, which includes RSE.

In January 2021, we enrolled the first patient in the Phase 3 pivotal RAISE trial. The RAISE trial is a randomized, double-blind, placebo-controlled clinical trial in patients with RSE. We expect approximately 8070 trial sites in hospitals, primarily across the U.S. and Canada, to participate. The RAISE trial is designed to enroll approximately 124 patients, ~~who will~~to be randomized to receive ganaxolone or placebo added to ~~standard-of-care.~~standard of care. With this number of patients, the trial is designed to provide over 90% power to detect a 30% efficacy difference between ganaxolone and placebo.

~~Upon preliminary review~~ We reached alignment with the FDA on a protocol amendment, including a proposal for an interim analysis when two-thirds of the ~~baseline characteristics of~~ patients (approximately 82) have completed the trial. We anticipate reaching the enrollment target for the interim analysis in the first quarter of 2024 with topline data now expected in the second quarter of 2024, if the pre-defined stopping criteria from the planned interim analysis are met. We believe positive interim RAISE trial ~~we noted that, as of September 30, 2022, the patient population is comparable to the population evaluated~~results could be adequate for regulatory filing purposes in our Phase 2 study. In particular, the mean ages, baseline Status Epilepsy Severity Score (STESS) results, pretreatment seizure burdens and baseline seizure frequencies are similar, as is the proportion of patients with a prior history of epilepsy. This is preliminary data and may not be representative of the demographics of patients upon full enrollment of the RAISE trial.

RSE.

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The co-primary endpoints for the RAISE trial are (1) proportion of patients with RSE who experience seizure cessation within 30 minutes of treatment initiation without other medications for SE treatment, and (2) proportion of patients with no progression to IV anesthesia for 36 hours following initiation of the ~~study~~trial drug. In June 2022, we announced that we amended the protocol for the RAISE trial to expand eligibility criteria to support recruitment. We broadened the inclusion criteria to permit patients previously treated with up to 18 hours of high-dose IV anesthesia to qualify for the ~~study,~~trial, rather than excluding patients treated with anesthetics at high doses for any duration. We believe this will facilitate the enrollment of patients transferred to the ICU from other hospitals or the emergency room, who may already have received high doses of anesthetic medication for less than 18 hours. We expect that the vast majority of clinical sites participating in the trial will have adopted the RAISE protocol amendment by the end of 2022. We reached alignment with the FDA on the protocol amendment, including a proposal for a potential interim analysis when two-thirds of the patients (approximately 82) have completed the study.

Several academic medical centers and intensive care units participating in the RAISE trial ~~have~~ experienced COVID-related difficulties, including staff turnover and the need to devote significant resources to patients with COVID-19, which ~~has~~ resulted in site initiation and enrollment delays. Additionally, in February 2022, we temporarily paused the RAISE trial after routine monitoring of stability batches of clinical supply material indicated that it became necessary to reduce the shelf life to less than the anticipated 24 months to meet product stability testing specifications. We notified the FDA of this issue and our plans to proactively pause the trial, and we subsequently provided additional information to the FDA to support resuming trial activities. In May 2022, we announced that the trial had resumed utilizing new batches of the current IV formulation of ~~ganaxolone. We have~~ganaxolone, and we implemented a reduced shelf life of 12 months. In agreement with the FDA, ganaxolone clinical supplies with the current IV formulation will be stored under refrigerated conditions for the entire duration of clinical use. Based on ongoing stability data, the shelf life of the current IV formulation has been updated to 18 months under refrigerated conditions, which was submitted in the subsequent IND amendment in February 2023. We ~~anticipate manufacturing~~manufactured the IV ganaxolone formulation with a new buffer ~~by end of the fourth quarter of 2022,~~and are targeting a shelf life of 24 months at ~~least 24 months.~~room temperature, pending the results of ongoing stability monitoring. The FDA agreed that in principle a buffer change in the ganaxolone IV formulation is ~~acceptable.~~

~~We are working closely with key investigators and site coordinators~~acceptable but requested additional information be submitted prior to ~~support enrollment efficiencies at existing RAISE study sites and are also increasing~~use of the ~~number of U.S. centers participating~~new formulation in the ~~trial. Additionally, we plan~~clinical trials. An IND amendment was submitted to ~~expand~~ the ~~study to~~FDA in May 2023. All sites ~~in Canada and Australia. Consistent~~have been resupplied with the ~~prior announcement,~~new formulation, which we ~~expect our top-line data readout for the RAISE trial~~believe will not require refrigeration and is expected to ~~be available in the second half of 2023.~~have a two-year shelf life.

Planning continues for a separate Phase 3 RSE trial to support an MAA in Europe (RAISE ~~II)~~II trial). We gained alignment on the ~~study~~trial design at a meeting with the EMA in the first quarter of 2021. Due to the delay in clinical trial supply mentioned for the RAISE trial, the RAISE II trial initiation is planned for the ~~second half~~fourth quarter of 2023. The RAISE II trial will be a double blind, placebo-controlled pivotal registration trial expected to enroll 70 patients who have failed first-line benzodiazepine treatment and at least one second-line AED. Patients will receive either ganaxolone or placebo, administered in combination with a standard-of-care second-line AED. The simultaneous administration of a standard-of-care AED with the ~~study medication~~trial drug is expected to provide data complementary to that from the RAISE ~~study.~~trial. There are two additional key differences between the RAISE and RAISE II trials. First, rather than specifying progression to IV anesthesia as a treatment failure, under the RAISE II protocol any escalation of care will constitute a treatment failure. This could be IV anesthesia or another second-line IV AED. Second, the primary analysis for the RAISE II trial will be a responder analysis, with response defined as SE cessation within 30 minutes and no escalation of care within 36 hours, rather than the co-primary endpoints in the RAISE ~~study,~~trial, which require statistical significance to be achieved independently on both the 30-minute and 36-hour outcomes.

~~The FDA has indicated alignment on the overall trial design for a third SE study,~~We have recently decided to discontinue the RESET trial, a Phase 2 ~~study~~trial evaluating ganaxolone in the treatment of ~~ESE.~~ESE, and focus our resources for IV ganaxolone on completing the RAISE trial and accelerating the RAISE II trial and further investigate potential development opportunities in SRSE. The RESET trial proved complex and costly to enroll.

SRSE is a life-threatening neurological emergency with high morbidity and mortality. We do not currently have a clinical trial ongoing in SRSE, however, physicians have received ganaxolone for 21 patients in SRSE under emergency investigational new drug (EIND) applications that have been approved by the FDA. We plan to ~~begin U.S. enrollment by~~engage with the ~~end~~FDA to request input on potential development of 2022. The RESET trial will enroll patients with convulsive SE presenting to emergency departments, and will be conducted under Exception from Informed Consent (EFIC) guidelines. The RESET trial will consist of two phases: an initial open-label, dose optimization phase and a subsequent double-blind placebo-controlled phase. In the open-label portionganaxolone for SRSE in light of the ~~trial, sequential cohorts will receive IV ganaxolone for varying durations and at different doses. The dosing for each cohort will depend on~~encouraging data generated from patients treated under the ~~efficacy and tolerability seen in the previous one, with the optimal dose and duration of ganaxolone incorporated in the double-blind phase of the study~~EINDs to follow. We expect that the double-blind placebo-controlled phase will enroll approximately 80 ESE patients randomized equally to IV ganaxolone or placebo added to a standard-of-care AED. The primary efficacy endpoint will be the absence of electrographic (rapid EEG) evidence of SE or recurrence of

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~~generalized convulsions at 1 hour after the initiation of treatment. We are targeting data from the first dose-finding cohort of the RESET trial by the end of 2023.~~date.

In September 2021, the ~~United States~~U.S. Patent and Trademark Office (USPTO) granted us a patent on a method of treating SE, including dosing regimens. This issued patent expires in 2040. That patent is a member of a patent family we own

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that includes pending patent applications that claim certain therapeutic regimens for the treatment of SE, including RSE, using intravenous ganaxolone. OThe USPTO granted us a second patent for SE on June 20, 2023, which includes claims related to our clinical therapeutic regimen for the treatment of SE using IV ganaxolone. These new claims cover therapeutic regiments in which high doses of ganaxolone are administered, which we believe is relevant for some patients, and strengthens our intellectual property portfolio for the treatment of SE, including SRSE, using ganaxolone.

In July ~~26,~~ 2022, the ~~United States~~USPTO issued U.S. Patent ~~and Trademark Office (USPTO) issued a patent~~No. 11,395,817 (Ovid ‘817 Patent) to Ovid Therapeutics, Inc. (Ovid) with claims that encompass our product candidate for the treatment of SE. On March 15, 2023, we filed a petition seeking post-grant review (PGR) of the Ovid ‘817 Patent with the U.S. Patent and Trademark Office Patent Trial and Appeal Board (PTAB). Our petition for PGR argues that the claims of the Ovid ‘817 Patent are unpatentable on multiple grounds. Ovid filed a preliminary response to our petition on June 20, 2023. In Ovid’s reply to our request for PGR, Ovid disclaimed claims 1-21, 23 and 24 of the Ovid ‘817 Patent, which has the effect of erasing these claims from the patent irrespective of the outcome of the PGR. On August 17, 2023, the PTAB issued a decision granting institution of our petition seeking PGR of the Ovid ‘817 Patent. In instituting the PGR, the PTAB stated that it was more likely than not that we would be able to invalidate the remaining claims (22 and 25-31) of the Ovid ‘817 Patent during the proceeding. The next steps in the PGR are replies by us and Ovid as well as depositions of the experts. The oral arguments are currently scheduled for May 22, 2024. The decision to institute is not a final decision on the patentability of the claims. The final decision will be based on the full record developed during the proceeding. If we do not prevail in the PGR proceeding, the decision can be appealed to the Court of Appeals for the Federal Circuit. If an appeal is not successful, our ability to challenge the Ovid ‘817 Patent in court will be limited in certain respects.

The Ovid ‘817 Patent claims cover the use of ganaxolone in the treatment of SE and do not cover or impact our marketing and sales of ZTALMY for the treatment of seizures associated with CDD. If we prevail in the PGR, the Ovid ‘817 Patent will not be enforceable against us.

Ovid may file a lawsuit against us alleging infringement of its ~~patents and/or we may challenge the validity of Ovid’s patents with the USPTO or through the courts.~~patents. Any such ~~proceeding,~~proceedings, in the PTAB or courts, regardless of ~~its~~their outcome, would likely result in the expenditure of significant financial resources and the diversion of management’s time and resources. In addition, any such proceeding may cause negative publicity, adversely impact patients, and we may be prohibited from marketing or selling ganaxolone for SE, including RSE, ~~and ESE~~ during such proceedings or if we are not successful in such proceedings. If Ovid does decide to bring an infringement lawsuit, we do not expect that it will be filed before a commercial launch of ganaxolone for ~~SE,~~ RSE ~~or ESE~~ based upon the “safe harbor” provisions of the Drug Price Competition and Patent Term Restoration Act of 1984 ~~(the Hatch-Waxman~~(Hatch-Waxman Act). We may need to acquire or obtain a license to ~~the~~certain Ovid patents to market or sell ganaxolone for ~~SE,~~ RSE, ~~and ESE,~~ which may not be available on commercially acceptable terms or at all. If we are not able to acquire the certain Ovid patents or negotiate a license on acceptable terms, and if our product is determined to infringe Ovid’s patents and ~~the~~such patents are determined to be valid, then we may be forced to pay Ovid royalties, damages and costs, or we may be prevented from commercializing ganaxolone for ~~SE,~~ RSE ~~and ESE~~ altogether, which would have a material adverse impact on our business.

Tuberous Sclerosis Complex (TSC)

TSC is a rare genetic disorder that ~~affects many organs by causing, typically~~causes non-malignant tumors in the brain, skin, kidney, heart, eyes, and lungs.Rarely, patients may develop malignant tumors of the kidney, breast or thyroid gland. The condition is caused by inherited mutations in either the TSC1 or TSC2 gene. It occurs with a frequency of approximately 1:6,000 live births, with a mutation being found in 85% of patients. While the disease phenotype can be extremely variable, epilepsy occurs with a frequency of up to 85%. TSC is a leading cause of genetic epilepsy, often manifesting in the first year of life as either focal seizures or infantile spasms. There are currently few disease-specific treatments approved for seizures in TSC. Orphan drug designation for ganaxolone for the treatment in TSC was granted by the FDA in August 2021 and by the EMA in October 2021.

In August 2021, we announced top-line data from our open-label Phase 2 trial ~~(the CALM~~(CALM trial) evaluating the safety and efficacy of adjunctive oral ganaxolone in 23 patients with seizures associated with TSC. The CALM trial enrolled 23 patients ages 2 to 32 who entered a four-week baseline period followed by a 12-week treatment period, during which they received up to 600 mg of ganaxolone (oral liquid suspension) three times a day. Patients who met eligibility criteria were able to continue ganaxolone treatment during a 24-week extension. The primary endpoint was the

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percent change in 28-day TSC-associated seizure frequency during the 12-week treatment period relative to the four-week baseline period. Secondary outcome measures included the percentage of patients experiencing a greater than or equal to 50% reduction in 28-day TSC-associated seizure frequency through the end of the 12-week treatment period compared to the 4-week baseline period.

The primary endpoint showed a median 16.6% reduction in 28-day frequency of TSC-associated seizures relative to the four-week baseline period. A secondary endpoint showed that the proportion of patients that achieved at least a 50% seizure reduction was 30.4%. During the trial, patients with focal seizures (n=19) showed a median 25.2% reduction in focal seizure frequency. Ganaxolone was generally well-tolerated with somnolence reported as the most common AE. In addition, one serious adverse event (SAE) of worsening seizures occurred, which was assessed by the investigator as ~~treatment-related.~~treatment related. Four patients discontinued the trial due to AEs. Additionally, the data from the trial suggested that in patients on concomitant Epidiolex, early elevation of ganaxolone blood levels occurred and appeared to be linked to greater somnolence. The interpretation of these findings is limited by the small sample size and open-label design of the ~~study.~~trial. A formal Phase 1 drug-drug interaction ~~study is ongoing to assess whether there is an~~trial was completed in the fourth quarter of 2022, demonstrating a lack of significant interaction between ganaxolone and Epidiolex. Additionally, the titration schedule for all subjects in the Phase 3 TSC trial has been adjusted to maximize tolerability.

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In response to our request for an End of Phase 2 meeting with the FDA regarding a proposed Phase 3 TSC trial, the FDA provided written responses to our questions in lieu of a meeting. We believe the written responses show overall alignment on the clinical development plan in TSC. We believe that, based on the FDA’s written responses, and with the FDA approval of CDD, a single trial could serve as necessary support for regulatory approval for TSC in the U.S. In response to our request for Protocol Assistance, which is a special form of scientific advice available for developers of designated orphan medicines for rare diseases, the EMA provided written feedback in December 2021 in lieu of a meeting. We believe the written responses from the EMA, like those from the FDA, show overall alignment on the clinical development plan in TSC. After commencing site initiations in the first quarter of 2022 and dosing the first patient in the second quarter of 2022, we are actively ~~screening~~enrolling patients in the U.S. and Western Europe for ~~enrollment in a~~this global Phase 3 randomized, double blind, placebo-controlled trial (TrustTSC trial) of adjunctive ganaxolone in approximately ~~160 TSC~~128 patients. With this number of patients, the trial is designed to provide 90% power to detect a 25% efficacy difference between ganaxolone and placebo. We expect to expand the trial to include up to 8090 sites, including several TSC centers of excellence, predominantly in the U.S., Western Europe, Canada, Australia, China and Israel. The primary endpoint for the TrustTSC trial is percent change in 28-day frequency of TSC-associated seizures. We plan to announce top-line data from the TrustTSC trial ~~in the first quarter of 2024.~~by mid-2024.

In July 2023, the USPTO granted us a patent on a method of treating TSC-related epilepsy by administering oral ganaxolone. This issued patent expires in 2040. This patent is a member of a patent family we own that includes pending patent applications that claim certain therapeutic regimens for the treatment of TSC.

Second-Generation Formulation, ~~Prodrug~~Clinical Development ~~and~~in Lennox-Gastaut Syndrome (LGS) and Prodrug Development

Top-line data from a single ascending dose (SAD) Phase 1 trial ~~with~~in healthy volunteers utilizing the first candidate for a second-generation formulation of ganaxolone ~~were announced in the second quarter~~demonstrated linear pharmacokinetic (PK) properties at doses of ~~2022, including pharmacokinetic (PK)~~up to 1200 mg and PK characteristics that may allow for twice-daily dosing. Additionally, as shown in the figure below, while area under the curve (AUC), which quantifies the total exposure to drug over a period of time, demonstrated linear kinetics through the full range dosed, C_max, which is the maximum serum concentration that a drug achieves after administration, showed less substantial increases at the higher end of the dose range. These findings suggest that efficacy could be improved using higher doses of ganaxolone without corresponding increases in dose-related side effects, such as somnolence. This would permit greater individualization of dosing to optimize efficacy and tolerability.

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We believe that the data support further clinical development of this formulation of ~~ganaxolone,~~ganaxolone. We received preliminary feedback from the FDA on the design of the multiple ascending dose (MAD) trial. The FDA requested additional data, including the SAD clinical trial report, prior to initiating the MAD trial. The information was provided to the FDA and, ~~an additional Phase 1 cohort~~upon review, the FDA indicated that we may proceed with the proposed trial. We initiated the MAD trial in the third quarter of ~~assessing~~2023 with preliminary data expected by the ~~pharmacokinetics~~end of ~~a second-generation oral formulation of ganaxolone is planned.~~2023.

The development of ganaxolone prodrug compounds continues to advance, with lead oral and IV candidates selected, and Phase 1 data targeted for 2024.

We plan to pursue the development of the second-generation formulation of ganaxolone for LGS, a severe form of epilepsy that typically begins between one and eight years of age. Affected children have neurodevelopmental impairments and intractable seizures, including focal, atonic, tonic, generalized tonic-clonic and atypical absence seizures. In March 2023, the FDA granted orphan drug designation to ganaxolone for the treatment of LGS. This designation applies to the active moiety of ganaxolone and is not dependent on the formulation. Given the overlap in seizure types and etiologies with other disorders where ganaxolone has potential to reduce seizures, such as CDD and TSC, we believe that LGS represents a promising opportunity for ganaxolone development. We expect to finalize the clinical program design for LGS in the first quarter of 2024, pending results of the MAD trial that we initiated in the third quarter of 2023.

On September 27, 2023, the USPTO issued a Notice of Allowance in an Ovid patent application with claims that encompass our product candidate for the treatment of LGS. This patent is scheduled to issue on November 7, 2023. The claims in the Ovid LGS patent application cover the use of ganaxolone in the treatment of LGS and do not cover or impact the use of ganaxolone in any other indication. Ovid may file a lawsuit against us alleging infringement of its LGS patent claims. Any such proceeding, regardless of the outcome, would likely result in the expenditure of significant financial resources and the diversion of management’s time and resources. In addition, any such proceeding may cause negative publicity, adversely impact patients, and we may be prohibited from marketing or selling ganaxolone for LGS during such proceeding or if we are ~~planning~~not successful in such proceedings. If Ovid does decide to ~~utilize~~bring an infringement lawsuit, we do not expect that it will be filed before a ~~second-generation formulation~~commercial launch of ganaxolone for LGS based upon the “safe harbor” provisions of the Drug Price Competition and Patent Term Restoration Act of 1984 (Hatch-Waxman Act). We may need to acquire or obtain a license to the Ovid LGS ~~development program, with~~patent to market or sell ganaxolone for LGS, which may not be available on commercially acceptable terms or at all. If we are not able to acquire the Ovid LGS patent or negotiate a ~~Phase 2 trial targeted~~license on acceptable terms, and if our product is determined to ~~begin in 2023.~~

infringe Ovid’s patent and such patent is determined to be valid, then we may be forced to pay Ovid royalties, damages and costs, or we may be prevented from commercializing ganaxolone for LGS altogether, which would have a material adverse impact on our business.

Operations

Our operations to date have consisted primarily of organizing and staffing our company, ~~and~~ developing ganaxolone, including conducting preclinical studies and clinical trials, and raising capital. We have funded our operations primarily through sales of equity and debt securities. ZTALMY, our first FDA approved product, became available for commercial sale and shipment ~~to patients with a prescription in the U.S.~~ in the third quarter of 2022. We recorded ~~$0.6~~$5.4 million and $13.0 million of net ZTALMY sales in the three months ended September 30, 2022. Other than for the three months ended September 30, 2022, we have incurred operating losses since inception and have not generated any product sales revenue. Excluding the one-time gain from the sale of the PRV resulting in net income in the three and nine months ended September 30, 2023, respectively. Since inception, other than for

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the three months ended September 30, 2022 due to a one-time net gain from the sale of our PRV, we have ~~not~~incurred net losses and negative cash flows from our operations. We have generated limited product revenues, and there is no assurance that profitable operations will be achieved ~~profitable operations.~~in the future, and if achieved, could be sustained on a continuing basis. We incurred net losses of $33.0 million and $99.6 million for the three and nine months ended September 30, 2023, respectively. Due to the one-time receipt of proceeds from the sale of the PRV ~~of $110.0 million~~ in the third quarter of 2022, we generated net income of $73.3 million and $14.5 million for the three and nine months ended September 30, ~~2022. We incurred a net loss of $70.5 million for the nine months ended September 30, 2021.~~2022, respectively. Our accumulated deficit as of September 30, ~~2022~~2023 was ~~$396.2~~$530.2 million, and we expect to continue to incur substantial losses in future periods. We anticipate that our operating expenses will increase substantially as we carry out all of our planned commercialization and continued research and development activities with respect to ganaxolone.

We anticipate that our expenses will increase substantially as we:

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conduct multiple later stage clinical trials in targeted indications;

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continue the research, development and scale-up manufacturing capabilities to optimize ganaxolone and dose forms for which we may obtain regulatory approval;

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continue to establish and implement sales, marketing and distribution capabilities to commercialize ganaxolone;

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conduct other preclinical studies and clinical trials to support the filing of NDAs with the FDA, MAAs with the EMA and other marketing authorization filings with regulatory agencies in other countries;

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acquire the rights to other product candidates and fund their development;

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maintain, expand and protect our global intellectual property portfolio;

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hire additional clinical, manufacturing, scientific and commercial personnel; and

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add operational, financial and management information systems and personnel, including personnel to support our drug development efforts.

We had cash, ~~and~~ cash equivalents and short-term investments of ~~$168.2~~$176.4 million atas of September 30, ~~2022, which included the $110.0 million we received from Novo Nordisk, Inc. in the third quarter of 2022 upon the closing of the PRV transaction.~~2023. We believe that our existing cash, ~~and~~ cash equivalents ~~on hand~~and short-term investments as of September 30, ~~2022 and the $32.5 million payment from Sagard Healthcare Partners received in October 2022~~2023 will be sufficient to fund our operating expenses and capital expenditure requirements, ~~and~~as well as maintain the minimum cash balance required under our debt facility, ~~into~~for at least twelve months from the ~~first quarter of 2024.~~date these financial statements are issued. However, we will need to secure additional funding in the future, from one or more equity or debt financings, government funding, collaborations, licensing transactions, other commercial transactions or other sources in order to carry out all of our commercialization and planned research and development activities with respect to ganaxolone.

Financial Overview

Product Revenue, net

Our first FDA approved product, ZTALMY, became available for commercial sale and shipment ~~to patients with a prescription in the U.S.~~ in the third quarter of 2022. We have three customers, one ~~customer,~~of which, Orsini Pharmaceutical Services, LLC (Orsini), a specialty pharmacy that dispenses ZTALMY directly to ~~patients.~~patients, represents over 99% of our ZTALMY revenue to date. Our contract with Orsini has a single performance obligation to deliver ZTALMY upon receipt of a purchase order, which is satisfied when Orsini receives ZTALMY. We recognize ZTALMY revenue at the point in time when control of ZTALMY is transferred to Orsini, which is upon delivery to Orsini. The transaction price that we recognize for ZTALMY revenue includes an estimate of variable consideration. Shipping and handling costs to Orsini are recorded as selling, general and administrative expenses. The components of variable consideration include:

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Trade Discounts and Allowances. We provide ancontractual discounts, including incentive prompt payment ~~discount to Orsini as explicitly stated in the contract with Orsini. This discount is~~discounts and chargebacks. Each of these potential discounts are recorded as a reduction of ZTALMY revenue and accounts receivable in the period in which the related ZTALMY revenue is recognized. We estimate the amount of variable consideration for all discounts and allowances using the expected value method.

Product Returns and Recall. We provide for ZTALMY returns in accordance with our Return Good Policy. We estimate the amount of ZTALMY that may be returned using the expected value method, and we present this amount as a reduction of ZTALMY revenue in the period the related ZTALMY revenue is recognized. In the event of a recall, we will promptly notify Orsini and will reimburse Orsini for direct administrative expenses incurred in connection with the recall as well as the cost of replacement product.

Government Rebates. We are subject to discount obligations under state Medicaid programs, ~~and Medicare.~~Medicare, the Tricare Retail Refund Program. We estimate reserves related to these discount programs and record these obligations in the same period the related revenue is recognized, resulting in a reduction of product revenue.

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Federal Contract Revenue

In September 2020, we ~~and BARDA~~ entered into a contract (BARDA Contract) with the Biomedical Advanced Research and Development Authority (BARDA), a division of the U.S. Department of Health and Human Services’ Office of the Assistant Secretary for Preparedness and Response. Under the BARDA Contract, ~~under which~~ we received an award of up to an estimated $51 million for development of IV-administered ganaxolone for the treatment of RSE. The BARDA Contract provides for funding to support, on a cost-sharing basis, the completion of a Phase 3 clinical trial of IV-administered ganaxolone in patients with RSE, which covers the RAISE trial, funding of pre-clinical studies to evaluate IV-administered ganaxolone as an effective treatment for RSE due to chemical nerve gas agent exposure, and funding of certain ganaxolone manufacturing scale-up and regulatory activities. In March 2022, we entered into an amendment with BARDA to extend the end date of our base performance period for funding under the BARDA Contract from September 1, 2022 to December 31, 2023. In September 2022, we entered into an amendment with BARDA that, among other things, (i) provides for the exercise of BARDA’s option under the BARDA Contract to support U.S. onshoring of the manufacturing capabilities for ganaxolone API (Option 2), (ii) changes the end of date of our performance period under Option 2 from December 31, 2026 to July 31, 2025, (iii) increases the government cost share amount under Option 2 from approximately $11.5 million to approximately $12.3 million, and (iv) increases our cost share amount under Option 2 from approximately $4.9 million to approximately $5.3 million. In September 2023, we entered into an amendment with BARDA to extend the end date of our base performance period for funding under the BARDA Contract from December 31, 2023 to September 30, 2024.

The BARDA Contract consists of an approximately ~~two-year~~four-year base period, ~~and an~~including the extension ~~period through December 31, 2023,~~periods, during which BARDA will provide up to approximately $21 million of funding for the RAISE trial on a cost share basis and funding of additional preclinical studies of ganaxolone in nerve agent exposure models. Following successful completion of the RAISE trial and preclinical studies in the base period and extension ~~period,~~periods, the BARDA Contract provides for approximately $31 million of additional BARDA funding for three options in support of ganaxolone manufacturing, supply chain, clinical, regulatory and toxicology activities, including the $12.3 million exercise of Option 2 as described above. Under the BARDA Contract, we will be responsible for cost sharing in the amount of approximately $33 million and BARDA will be responsible for approximately $52 million if all development options are completed. The contract period-of-performance (base period plus option exercises) is up to approximately five years.

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We recognize federal contract revenue from the BARDA Contract in the period in which the allowable research and development expenses are incurred. We expect federal contract revenue to increase as the costs associated with our RAISE trial increase.

Collaboration Revenue

In July 2021, we ~~and Orion~~ entered into a collaboration agreement ~~(the Orion~~(Orion Collaboration Agreement). with Orion. Under the terms of the Orion Collaboration Agreement, we granted Orion an exclusive, royalty-bearing, sublicensable license to certain of our intellectual property rights with respect to commercializing biopharmaceutical products incorporating ganaxolone (Licensed Products) in the European Economic Area, the United Kingdom and Switzerland (collectively, the Territory) for the diagnosis, prevention and treatment of certain human diseases, disorders or conditions (Field), initially in the indications of CDD, TSC and RSE.

Under the terms of the Orion Collaboration Agreement, we received a €25.0 million ($29.6 million) upfront payment from Orion in July 2021. We are eligible to receive up to an additional ~~€97.0~~€97 million in ~~research and development~~R&D reimbursement and cash milestone payments based on specific clinical and commercial ~~achievements, as well as tiered royalty payments based on net sales ranging from the low double-digits to high teens for the oral programs and the low double-digits to low 20s for the IV program.~~achievements. Also, as part of the overall arrangement, we have agreed to supply the Licensed Products to Orion at an agreed upon price.

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We identified the following commitments under the arrangement: (i) exclusive rights to develop, use, sell, have sold, offer for sale and import any product comprised of Licensed Product (License); (ii) development and regulatory activities (Development and Regulatory Activities); and (iii) requirement to supply Orion with the Licensed Product at an agreed upon price (Supply of Licensed Product). We determined that these three commitments represent distinct performance obligations for purposes of recognizing revenue and will recognize license and collaboration revenue or a reduction of expense as we fulfill each performance obligation.

On November 16, 2022, we entered into a Collaboration and Supply Agreement (Tenacia Collaboration Agreement) with Tenacia. Under the terms of the Tenacia Collaboration Agreement, we granted Tenacia an exclusive, royalty-bearing, sublicensable license to certain of our intellectual property rights to develop, commercialize and otherwise exploit certain products incorporating certain oral and intravenous formulations of the our product candidate ganaxolone (Licensed Products) in Mainland China, Hong Kong, Macau and Taiwan (collectively, Territory) for the diagnosis, prevention and treatment of certain human diseases, disorders or conditions (Field), initially for the treatment of CDD, TSC and SE (including RSE) (collectively, Initial Indications). The collaboration can be expanded to include additional indications and formulations of ganaxolone pursuant to a right of first negotiation.

Under the terms of the Tenacia Collaboration Agreement, Tenacia agreed to pay us an upfront cash payment of $10 million (Upfront Fee) within forty-five (45) days after the Effective Date, which was received in December 2022. In addition to the Upfront Fee, Tenacia has agreed to make cash payments to us upon the achievement of certain development, regulatory and sales-based milestones related to (i) the Initial Indications and (ii) the first new formulation or pro-drug of ganaxolone or any back-up compound of ganaxolone in a new indication (Selected Product) for which the parties amend the Tenacia Collaboration Agreement in connection with Tenacia’s exercise of its right of first negotiation and for which there is no other Licensed Product approved in China (for clarity, the milestone payments under this clause (ii) will only apply to one Selected Product), up to an aggregate amount of $256 million. Of the milestones, $15 million relates to regulatory approvals with separate milestones related to each of oral and intravenous formulations and the Selected Product, and an aggregate of $241 million of sales-based milestones are connected to annual revenue thresholds specific to each of the oral, intravenous and Selected Product formulations of ganaxolone. Tenacia has further agreed to pay us tiered royalty payments based on annual net sales of Licensed Products ranging from the low double digits to the mid-teens for each of the oral formulation, intravenous formulation and Selected Product formulation of Licensed Products. Tenacia’s obligations to pay royalties to us with respect to sales of a Licensed Product in each particular jurisdiction of the Territory will commence on the date of first commercial sale in such jurisdiction and expire upon the latest of (i) ten years following the first commercial sale of such Licensed Product in such jurisdiction, (ii) the expiration of the last-to-expire valid claim of any licensed patent rights that covers such Licensed Product in such jurisdiction and (iii) the expiration of all regulatory exclusivities for such Licensed Product in such jurisdiction. Royalty payments are subject to reduction in specified circumstances as set forth in the Tenacia Collaboration Agreement, including if net sales decrease by a certain percentage after the introduction of a generic product.

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Tenacia will be primarily responsible for the development of Licensed Products in the Territory and regulatory interactions related thereto, including conducting and sponsoring clinical studies in the Field in the Territory to support regulatory filings in the Territory. All regulatory approvals filed by Tenacia in the Territory will be in the name of and owned by us unless otherwise required by applicable law, in which case such regulatory approvals would be in the name of and owned by Tenacia for the benefit of us. We and Tenacia agreed to enter into clinical and commercial supply agreements pursuant to which we will supply Tenacia with its requirements of Licensed Products necessary for Tenacia to develop and commercialize Licensed Products in the Field in the Territory. The parties entered into the clinical and commercial supply agreement in May 2023. The agreement contains pricing, delivery, acceptance, payment, termination, forecasting, and other terms consistent with the Tenacia Collaboration Agreement, as well as certain quality assurance, indemnification, liability and other standard industry terms. Tenacia will be responsible for, at Tenacia’s sole cost and expense, obtaining regulatory approval and commercializing the Licensed Product in the Field in Mainland China.Tenacia is enrolling patients in our Phase 3 randomized, double blind, placebo-controlled trial (TrustTSC trial) of adjunctive ganaxolone.

The term of the Tenacia Collaboration Agreement extends for so long as royalties are payable anywhere in the Territory. Subject to the terms of the Tenacia Collaboration Agreement, (i) for a specified period of time after the Effective Date, Tenacia may terminate the Tenacia Collaboration Agreement in its entirety for any or no reason upon written notice to us, and (ii) either party may terminate the Tenacia Collaboration Agreement for the other party’s material breach following a cure period or insolvency.

In accordance with the guidance, we identified the following commitments under the arrangement: (i) grant to Tenacia the exclusive rights to develop, commercialize and otherwise exploit Licensed Product in the Field in the Territory (License) and (ii) requirement to supply Tenacia with the Licensed Product at an agreed upon price (Supply of Licensed Product). We determined that these two commitments represent distinct performance obligations for purposes of recognizing revenue or reducing expense, which it will recognize such revenue or expense, as applicable, as it fulfills these performance obligations.

We have also entered into agreements for commercialization of ganaxolone in other territories with (i) NovaMedica LLC (NovaMedica), whereby NovaMedica has the right to market and sell ganaxolone in Armenia, Azerbaijan, Belarus, Georgia, Kazakhstan, Kyrgyzstan, Moldova, Russia, Tajikistan, Turkmenistan, Ukraine and Uzbekistan, and (ii) Biologix FZCo (Biologix), whereby Biologix has the right to distribute and sell ganaxolone inAlgeria, Bahrain, Egypt, Iraq, Jordan, Kingdom of Saudi Arabia, Kuwait, Lebanon, Libya, Morocco, Oman, Qatar, Tunisia and United Arab Emirates. In exchange for distribution rights, we will be the exclusive supplier of our products to NovaMedica and Biologix on terms set forth in the respective agreements in exchange for a negotiated purchase price for the products. As of the first quarter 2023, we have initiated limited sales of ZTALMY to NovaMedica to support on-going early access programs associated with patients from the Marigold Study. We continue to assess opportunities in other markets to further expand the distribution and commercialization of ganaxolone globally.

Research and Development Expenses

Our research and development expenses consist primarily of costs incurred for the development of ganaxolone, which include:

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employee-related expenses, including salaries, benefits, travel and stock-based compensation expense;

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expenses incurred under agreements with clinical research organizations (CROs) and investigative sites that conduct our clinical trials and preclinical studies;

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the cost of acquiring, developing and manufacturing clinical trial materials;

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facilities, depreciation and other expenses, which include direct and allocated expenses for rent and maintenance of facilities, ~~insurance~~ and other ~~supplies;~~

supplies and services;

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costs associated with preclinical activities and regulatory operations; and

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costs associated with developing new formulations and prodrugs of ganaxolone.

We expense research and development costs when we incur them. We record costs for some development activities, such as clinical trials, based on an evaluation of the progress to completion of specific tasks using data such as patient enrollment, clinical site activations and information our vendors provide to us.

We have and will incur substantial costs beyond our present and planned clinical trials in order to file an NDA and Supplemental NDAs, or MAAs outside the U.S., for ganaxolone for various clinical indications, and in each case, the nature, design, size and cost of further clinical trials and other studies will depend in large part on the outcome of preceding studies and trials and discussions with regulators. It is difficult to determine with certainty the costs and duration of our current or future clinical trials and preclinical studies, or if, when or to what extent we will generate revenue from the commercialization and sale of ganaxolone if we obtain regulatory approval. We may never succeed in achieving regulatory approval for ganaxolone. The duration, costs and timing of clinical trials and development of ganaxolone will depend on a variety of factors, including the uncertainties of future clinical trials and preclinical studies, uncertainties in clinical trial enrollment rate and significant and changing government regulation.

In addition, the probability of success for our clinical programs will depend on numerous factors, including competition, manufacturing capability and commercial viability. See the “Risk Factors” section of our Annual Report on Form 10-K filed on March 9, 2023 for more information with respect to such factors. Our commercial success depends upon attaining significant market acceptance, if approved, among physicians, patients, healthcare payers and the medical community. We will determine which programs to pursue and how much to fund each program in response to the scientific and clinical success, as well as an assessment of commercial potential.

Selling, General and Administrative Expenses

Selling, general and administrative expenses consist principally of salaries and related costs for executive, commercial and other administrative personnel and consultants, including stock-based compensation and travel expenses. Other selling, general and administrative expenses include professional fees for commercial, legal, patent review, consulting and accounting services. Selling, general and administrative expenses are expensed when incurred.

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Cost of Product Revenue

Cost of product revenue includes the cost of inventory sold, which includes direct manufacturing and supply chain costs. Also included in cost of product revenue are royalty payments owed to Purdue Neuroscience Company (Purdue) and Ovid in accordance with the respective license agreements. We began capitalizing inventory related to ZTALMY subsequent to the March 2022 FDA approval of ZTALMY, as the related costs were expected to be recoverable through the commercialization and subsequent sale of ZTALMY. Prior to FDA approval of ZTALMY, costs estimated at approximately $2 million for commercially saleable product and materials were incurred and included in research and development expenses. As a result, cost of product revenues related to ZTALMY will initially reflect a lower average per unit cost of materials into approximately the first half of 2024, as previously expensed inventory is utilized for commercial production and sold to customers.

Cost of ~~Collaboration Revenue~~

Cost of collaboration revenue represents a one-time fee paid to Purdue related to that certain license agreement entered into in September 2004 and subsequently amended and restated in May 2008 between us and Purdue. This fee was paid in conjunction with our Orion Collaboration Agreement in the third quarter of 2021.

IP License Fee ~~Expenses~~

In March 2022, we entered into an exclusive patent license agreement (License Agreement) with Ovid. Under the License Agreement, we have an exclusive, non-transferable (except as provided in the License Agreement), royalty-bearing, sublicensable license under certain of Ovid’s patent(s) and patent applications to develop, make, have made, commercialize, promote, distribute, sell, offer for sale and import, ganaxolone, including any analogues or derivatives, including its salts, and pharmaceutical formulations of the foregoing (Licensed Products), in the U.S., the member states of the EU, Iceland, Lichtenstein, Norway, the United Kingdom, and Switzerland (Territory) for the treatment of CDD in humans (Field). Under the License Agreement, we have the sole right and responsibility for, and control over, all development, manufacturing, and commercialization activities, including all regulatory activities, with respect to the

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Licensed Products in the Field in the Territory. In addition, all regulatory approvals and related filings with respect to the Licensed Products in the Field in the Territory will be in the name of, and be owned solely by, us. We were required, at Ovid’s option exercisable in accordance with the License Agreement, to (i) pay to Ovid the sum of $1.5 million in cash; or (ii) issue to Ovid 123,255 shares of our common stock, which option to obtain shares of our common stock was exercisable within the five-business day period following the filing of our Annual Report on Form 10-K for the year ended December 31, 2021 on March 24, 2022. On March 29, 2022, we issued 123,255 shares of our common stock to Ovid, per Ovid’s option in accordance with the License Agreement. As such, we recorded $1.2 million of IP license fee expenses related to the Ovid License Agreement in the nine months ended September 30, 2022.

The License Agreement also provides for payment of royalties by us to Ovid in the low single digits on net sales by us, our affiliates and sublicensees, of Licensed Products in the Field in the Territory. Such royalties are subject to reduction in the event of generic competition in accordance with the License Agreement. We may terminate the License Agreement at any time without cause on thirty days’ prior written notice. Either party may terminate the License Agreement for the other party’s material breach or insolvency subject to certain cure periods. Also, Ovid has the right to terminate the License Agreement if there has not been a first commercial sale of any Licensed Products in the Field in the Territory on or before June 30, 2025. In the event of termination, all licenses granted under the License Agreement will terminate.

Interest Income

Interest income consists principally of interest income earned on cash and cash equivalents and investment balances.

Interest Expense

Interest expense consists of interest expense and amortization of debt discount ~~and commitment fees~~ related to our Notes ~~Payable.~~

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~~Gain from Sale of Priority Review Voucher, net~~

~~In the third quarter of 2022, we recognized a one-time gain, net of transaction costs, from the sale of the PRV to Novo Nordisk, Inc. Refer to Notes 1~~Payable and ~~2 in the accompanying notes to consolidated financial statements for further details.~~our Revenue Interest Financing Payable.

Other Income (Expense) ~~Income,~~, net

Other ~~expense~~income and ~~income~~expense consists principally of gains or losses on disposal of fixed assets held for sale, foreign currency ~~translation, and~~transactions, fair value ~~adjustments.~~adjustments and other non-operational gains and losses.

~~Provision~~Benefit (Provision) for Income Taxes

~~Due~~We recorded a $1.5 million benefit for income taxes due to the identification of a discrete item of tax determined upon preparation of our 2022 tax return in the nine months ended September 30, 2023. We did not record a benefit or provision for income taxes in the three months ended September 30, 2023. We recorded a $1.8 million provision for income taxes in the three and nine months ended September 30, 2022 due to net income related to the one-time receipt of proceeds from the sale of the ~~PRV of $110.0 million in the third quarter of 2022, we generated net income for the three and nine months ended September 30, 2022. As a result~~PRV. , ~~we have recorded current income tax expense in the three and nine months ended September 30, 2022 attributable to state income taxes.~~

Results of Operations

Product Revenue, net

We recognized ~~$0.6~~$5.4 million and $13.0 million of ZTALMY net product revenue ~~related to ZTALMY sales~~ for the three and nine months ended September 30, ~~2022. As~~2023, respectively. We recognized $0.6 million of ZTALMY ~~our first FDA approved product, became available for commercial sale and shipment to patients with a prescription in the U.S. in the third quarter of 2022, we did not recognize any~~net product revenue infor both the three and nine months ended September 30, ~~2021.~~2022.

Federal Contract Revenue

We recognized $1.9 million and $10.8 million of federal contract revenue for the three and nine months ended September 30, 2023 as a result of the BARDA Contract. We recognized $1.8 million and $5.1 million of federal contract revenue for the three and nine months ended September 30, 2022, respectively as a result of the BARDA Contract. ~~We recognized $1.1 million and $4.8~~The

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increase in the nine months ended September 30, 2023 compared to the 2022 period primarily relates to expenses incurred in connection with on-going validation of a new third-party supplier of ganaxolone API.

Collaboration Revenue

Collaboration revenue, which resulted from our agreement with Biologix, was less than $0.1 million for the three and nine months ended September 30, ~~2021, respectively, as a result of~~2023. No collaboration revenue was recorded in the ~~BARDA Contract.~~

~~Collaboration Revenue~~

three months ended September 30, 2022. Collaboration revenue was $12.7 million for the nine months ended September 30, 2022 as a result of revenue recognition related to the previously refundable upfront payment pursuant to the Orion Collaboration Agreement. ~~We did not recognize any collaboration revenue for the three months ended September 30, 2022.~~ In connection with the upfront fee related to the Orion Collaboration Agreement, we agreed to provide Orion with the results of an ongoing genotoxicity study. In ~~February~~May 2022, the ~~verified draft~~final study report showed that no genotoxicity was found, as measured by formation of micronuclei in the bone marrow or comet morphology in the liver. ~~These results were formalized in the final study report received in May 2022 and, as~~As a result of the study’s findings, we ~~are~~were not required to refund Orion any of the upfront fee and Orion does not have the right to terminate the Orion Collaboration Agreement based on the study outcome. During the nine months ended September 30, 2022, we allocated the previously refundable portion of the upfront payment to the transaction price and recognized the related revenue. ~~We recognized $9.0 million of collaboration revenue for the three and nine months ended September 30, 2021 upon entering into the Orion Collaboration Agreement.~~

Research and Development Expenses

We record direct research and development expenses, consisting principally of external costs, such as fees paid to investigators, consultants, central laboratories and CROs in connection with our clinical trials, and costs related to manufacturing, to specific product development programs. We do not allocate costs related to purchasing clinical trial materials, employee and contractor-related costs, costs associated with our facility expenses, including depreciation or other indirect costs, to specific product programs because these costs are deployed across multiple product programs under research and development and, as such, are not separately classified. The table below shows our research and

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development expenses incurred with respect to each active program, in thousands. The primary drivers of our research and development expenditures are currently in our product development programs in CDD, RSE, ~~TSC~~ and ~~ESE.~~TSC. We did not allocate research and development expenses to any other specific product development programs during the periods presented (in thousands):


	Three Months Ended				Nine Months Ended				Three Months Ended				Nine Months Ended
	September 30,				September 30,				September 30,				September 30,
	2022		2021		2022		2021		2023		2022		2023		2022
CDKL5 deficiency disorder (1)	$	909	$	1,968	$	2,973	$	6,529	$	406	$	909	$	2,461	$	2,973
PCDH19-related epilepsy (2)		—		628		1,063		2,070		120		—		245		1,063
Tuberous Sclerosis (3)		2,590		1,093		6,897		2,347		3,491		2,590		10,865		6,897
Drug Development - Suspension (4)		1,115		1,779		2,770		6,653
Drug Development – Suspension (4)										1,267		1,115		2,425		2,770
Oral Indications Subtotal		4,614		5,468		13,703		17,599		5,284		4,614		15,996		13,703

Status epilepticus (5)		2,661		1,777		6,484		4,265		4,117		2,661		10,697		6,484
Drug Development - IV (6)		708		723		4,688		3,267
Drug Development – IV (6)										1,305		708		10,733		4,688
IV Indications Subtotal		3,369		2,500		11,172		7,532		5,422		3,369		21,430		11,172

Other research and development (7)		2,356		3,679		7,751		9,818		2,193		2,356		4,021		7,751
Indirect research and development (8)		8,663		6,706		25,862		20,557		10,762		8,663		31,559		25,862
Total	$	19,002	$	18,353	$	58,488	$	55,506	$	23,661	$	19,002	$	73,006	$	58,488

(1)

The decrease in the three ~~and~~months ended September 30, 2023 compared to the 2022 period was due primarily to decreased clinical trial activity. The decrease in the nine months ended September 30, ~~2022~~2023 compared to the ~~2021~~2022 period was also due primarily to decreased clinical trial activity, but was partially offset by increased costs due to increased clinical site close-out activities related to the Marigold Study and increased activity associated with the MAA application and review in the first quarter of 2023.

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(2)

The increase in the three months ended September 30, 2023 compared to the 2022 period was due primarily to ~~more significant regulatory and statistical analysis expenses associated with our NDA filing preparation than in the prior period and reduced~~increased clinical trial ~~activity in the current period.~~

~~(2)~~

close-out costs. The decrease in the ~~three and~~ nine months ended September 30, ~~2022~~2023 compared to the ~~2021~~2022 period was due to reduced clinical activity, specifically completion of the open label extension portion of the PCDH-19 trial.

(3)

The increase in the three and nine months ended September 30, ~~2022~~2023 compared to the ~~2021~~2022 period was due primarily to increased ~~activity in the 2022 period from the~~ Phase 3 TSC trial ~~start-up,~~activity, including increased global site activity, as compared to more limited Phase ~~2 activities~~3 activity in the ~~relevant 2021~~2022 period.

(4)

The ~~decrease~~increase in the three ~~and nine~~ months ended September 30, ~~2022~~2023 compared to the ~~2021~~2022 period was due primarily to higher manufacturing ~~costs~~development activity related to clinical trial batches than in the prior 2022 period. The decrease in the nine months ended September 30, 2023 compared to the 2022 period was due primarily to higher manufacturing development activity related to pre-validation and registration batches in the ~~prior~~2022 period, ~~compared~~partially offset by the higher manufacturing development activity related to ~~the relevant 2022 period.~~clinical trial batches.

(5)

The increase in the three and nine months ended September 30, ~~2022~~2023 compared to the ~~2021~~2022 period was due primarily to increased ~~costs related to the RESET~~RAISE and RAISE II Phase 3 trial ~~with no comparable costs in the relevant 2021 period, as well as increased costs related to the RAISE trial.~~activity.

(6)

The increase in the three months ended September 30, 2023 compared to the 2022 period was due primarily to the completion of registration batches with no comparable costs in the prior 2022 period. The increase in the nine months ended September 30, ~~2022~~2023 compared to the ~~2021~~2022 period was due primarily to ~~set-up fees at~~start-up costs associated with validation of a new ~~third party manufacturer and ongoing stability testing. Costs remained consistent for~~third-party supplier of ganaxolone API with no comparable costs in the ~~three months ended September 30,~~prior 2022 ~~compared to the three months ended September 30, 2021.~~period.

(7)

Other research and development expenses include external expenses associated with preclinical ~~and clinical~~ development of ganaxolone, including safety studies, stability studies, preclinical studies, including animal toxicology and pharmacology studies, and other professional fees. The decrease in the three and nine months ended September 30, ~~2022~~2023 compared to the ~~2021~~2022 period was due primarily to ~~toxicology and other safety study activities. The decrease in the nine months ended September 30, 2022 compared to the 2021 period was due primarily to~~decreased activities associated with the Phase 1 clinical trials.

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(8)

Indirect research and development expenses include personnel costs and non-study specific research and development costs. The ~~increase~~increases in the three and nine months ended September 30, ~~2022~~2023 compared to the ~~2021 period was~~2022 periods were primarily related to increased personnel costs in support of our increased activity in preclinical, clinical, and manufacturing activities.

Selling, General and Administrative Expenses

Selling, general and administrative expenses were $14.9 million and $45.8 million for the three and nine months ended September 30, 2023, respectively, compared to $13.4 million and $42.2 million for the three and nine months ended September 30, 2022, ~~respectively, compared to $9.5 million and $26.7 million for the three and nine months ended September 30, 2021,~~ respectively. The primary drivers of the increase for the three months ended September 30, 2023 compared to 2022 were ~~$2.7~~$0.5 million in increased ~~personnel and training costs, $0.8 million in increased commercialization preparation costs, $0.7 million in increased noncash stock-based compensation~~commercial costs, $0.5 million in increased consulting costs, ~~and $0.5~~$0.4 million in increased ~~software related expenses, partially offset by a $1.1 million decrease due to contract acquisition~~personnel costs ~~related to our Orion Collaboration Agreement in the three months ended September 30, 2021~~ and ~~a $0.2 million decrease in general costs.~~net other cost increases of $0.1 million. The primary drivers of the increase for the nine months ended September 30, 2023 compared to 2022 were ~~$7.5~~$3.4 million in increased personnel ~~and training~~ costs, ~~$5.6~~$1.5 million in increased ~~commercialization preparation costs, $1.9 million in increased~~professional fees, consulting costs ~~$1.0~~and other general costs, $0.6 million in increased software related ~~expenses, $0.8 million in increased travel and meeting~~ costs and $0.3 million in increased ~~general costs,~~stock-based compensation costs. These increases were partially offset by ~~a $1.1~~$2.2 million ~~decrease~~in decreased commercial costs, primarily due to ~~contract acquisition costs related to our Orion Collaboration Agreement~~significant commercial-launch preparedness activities in 2022.

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Interest Income

Interest income was $1.9 million and $6.4 million for the three and nine months ended September 30, ~~2021 and a~~2023, respectively, compared to $0.5 million ~~decrease in noncash stock-based compensation costs. Of such decrease, $2.1~~and $0.6 million ~~was~~for the three and nine months ended September 30, 2022, respectively. The increase is due to ~~modifications of stock options recorded~~the increase in ~~the first quarter of 2021 in connection with a severance agreement with our former Chief Financial Officer.~~cash, cash equivalent and short-term investments and increased yield.

Interest Expense

Interest expense was $4.2 million and $12.6 million for the three and nine months ended September 30, 2023, respectively, compared to $2.6 million and $7.0 million for the three and nine months ended September 30, 2022, respectively. Interest expense for the nine months ended September 30, 2023 included $6.5 million of interest paid and $1.5 million of debt amortization in connection with our Note Payable (Note 10 in accompanying notes to consolidated financial statements), and $4.4 million of non-cash interest expense and $0.2 million of debt amortization related to our revenue interest financing payable (Note 11 in accompanying notes to consolidated financial statements). Interest expense for the nine months ended September 30, 2022 included $5.7 million of interest paid, $1.2 million of debt amortization, and $0.1 million related to commitment fees paid in connection with our ~~Notes payable (Note 9 in accompanying notes to consolidated financial statements). Interest expense~~Note Payable.

Other Income (Expense), net

Other income was ~~$0.7~~$1.0 million and ~~$1.0~~$1.1 million for the three and nine months ended September 30, ~~2021, respectively.~~

~~Gain from Sale of Priority Review Voucher, net~~

In2023, respectively, due primarily to the third quarter of 2022, we recognized a one-time gain of $107.4 million from the sale of the PRV to Novo Nordisk, Inc. The gain was recorded net of transaction costs of approximately $2.6 million. Refer to Notes 1 and 2 in the accompanying notes to consolidated financial statements for further details.

~~Other (Expense) Income, net~~

IRS Employee Retention Tax Credit program. Other expense was $0.1 million and $1.2 million for the three and nine months ended September 30, 2022, respectively, which consisted principally of foreign currency ~~translation,~~transaction losses, losses on fixed assets held for sale, and fair value adjustments. ~~Other income (expense) recorded~~

Liquidity and Capital Resources

Since inception, other than for the three months ended September 30, 2022 due to a one-time net gain from the sale of our PRV, we have incurred net losses and negative cash flows from our operations. We incurred a net loss of $99.6 million for the ~~three and~~ nine months ended September 30, ~~2021 was not material.~~

~~Provision for Income Taxes~~

2023. Due to the one-time receipt of proceeds from the sale of the PRV of $110.0 million in the third quarter of 2022, we generated net income of ~~$73.3 million and~~ $14.5 million for the ~~three and~~ nine months ended September 30, ~~2022, respectively. As a result~~, ~~we have a current income tax expense of $1.8 million for the period ended September 30, 2022 attributable to state income taxes.~~

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~~Liquidity and Capital Resources~~

Other than for the three months ended September 30, 2022, we have incurred net losses and negative cash flows from our operations since inception. Due to the one-time receipt of proceeds from the sale of the PRV of $110.0 million in the third quarter of 2022, we generated net income of $73.3 million and $14.5 million for the three and nine months ended September 30, 2022, respectively. We generated a net loss of $19.5 million and $70.5 million for the three and nine months ended September 30, 2021.2022. Our cash used in operating activities was $91.0 million for the nine months ended September 30, ~~2022~~2023 compared to ~~$33.7~~$91.0 million for the nine months ended September 30, ~~2021.~~2022. Historically, we have financed our operations principally through the sale of common stock, notes payable, preferred stock and convertible debt.

In July 2022, we entered into the PRV Asset Purchase Agreement to sell our PRV, pursuant to which Novo Nordisk, Inc. paid us $110.0 million upon the closing of the transaction. ~~At September 30,~~In August 2022, ~~we had cash and cash equivalents of $168.2 million.~~

~~On July 14, 2022, we announced that we had entered into a definitive agreement to sell a Rare Pediatric Disease Priority Review Voucher (PRV) for $110 million. Thereafter,~~ we received a letter ~~dated August 1, 2022~~ from Purdue in which Purdue claimed that it was owed $5.5 million by us from the sale of the PRV pursuant to ~~a 2008 agreement between~~the Purdue ~~and us.~~License Agreement. Our position communicated to Purdue is that we do not owe Purdue any of the proceeds from the sale of the PRV. No associated payment by us has been made, and Purdue has not filed a specific claim to date.

In November 2022, in connection with an underwritten public offering of 10,526,316 shares of our common stock, pre-funded warrants to purchase 2,105,264 shares of common stock and the exercise of an option of 1,894,737 shares of common stock, we received approximately $64.5 million in total net proceeds after taking into account the exercise of the underwriters’ option, in each case deducting the underwriting discounts and commissions and after deducting offering expenses paid or payable by us. Additionally, in November 2022, we received an upfront payment of $32.5 million pursuant to the Revenue Interest Financing Agreement with Sagard, and in December 2022, we received an upfront payment of $10.0 million in connection with the Tenacia Collaboration Agreement.

In September 2023, in connection with the amended Equity Distribution Agreement (EDA) with JMP Securities LLC (JMP), we received net proceeds totaling approximately $25.9 million from the sale of 3.7 million shares of our common stock at an average price of $7.17 per share.

As of September 30, 2023, we had cash, cash equivalents and short-term investments of $176.4 million. We believe that our existing cash, cash equivalents and short-term investments as of September 30, 2023 will be sufficient to

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fund our operating expenses and capital expenditure requirements, as well as maintain the minimum cash balance required under our debt facility, for at least twelve months from the date these financial statements are issued. However, we will need to secure additional funding in the future, from one or more equity or debt financings, government funding, collaborations, licensing transactions, other commercial transactions or other sources in order to carry out all of our commercialization and planned research and development activities with respect to ganaxolone.

Orion Commercialization Agreement

On July 30, 2021, we entered into the Orion Collaboration Agreement, whereby Orion received exclusive rights to commercialize the oral and IV dose formulations of ganaxolone in the European Economic Area, United Kingdom and Switzerland (collectively, Territory) in multiple seizure disorders, including CDD, TSC and RSE. Under the agreement, we received a €25 million ($29.6 million) upfront ~~fee and~~fee. We are eligible to receive up to an additional €97 million in research and development reimbursement and cash milestone payments based on specific clinical and commercial achievements, as well as tiered royalty payments based on net sales ranging from the low ~~double digits~~double-digits to ~~the~~ high teens for the oral programs and the low double-digits to ~~the~~ low ~~twenties~~20s for the IV ~~programs.~~program. As a result of the July 2023 EC approval of ZTALMY oral suspension for the adjunctive treatment of epileptic seizures associated with CDD in patients two to 17 years of age, we are now eligible under the Orion Collaboration Agreement to receive a commercial milestone payment of 10 million Euro, if commercial sales of ZTALMY commence in the Territory, upon the earlier of (1) the first commercial sale of ZTALMY within two of a select set of countries consisting of Germany, France, Italy, Spain, and the United Kingdom or (2) the 18-month anniversary of the first commercial sale of ZTALMY in the Territory. Also, as part of the overall arrangement, we have agreed to supply the Licensed Products to Orion at an agreed upon price.

Tenacia Commercialization Agreement

On November 16, 2022, we entered into a collaboration and supply agreement with Tenacia, pursuant to which we granted Tenacia an exclusive, royalty-bearing, sublicensable license to certain of our intellectual property rights to develop, commercialize and otherwise exploit certain products incorporating certain oral and intravenous formulations of our product candidate ganaxolone (Licensed Products) in Mainland China, Hong Kong, Macau and Taiwan (collectively, Territory) for the diagnosis, prevention and treatment of certain human diseases, disorders or conditions (Field), initially for the treatment of cyclin-dependent kinase-like 5 deficiency disorder, tuberous sclerosis complex and SE (including RSE) (collectively, Initial Indications). The collaboration can be expanded to include additional indications and formulations of ganaxolone pursuant to a right of first negotiation.

In connection with the execution of the Tenacia Collaboration Agreement, Tenacia agreed to pay us an upfront cash payment of $10 million (Upfront Fee) within forty-five (45) days after the Effective Date and payment was received in December 2022. In addition to the Upfront Fee, Tenacia has agreed to make cash payments to us upon the achievement of certain development, regulatory and sales-based milestones related to (i) the Initial Indications and (ii) the first new formulation or pro-drug of ganaxolone or any back-up compound of ganaxolone in a new indication (Selected Product) for which the parties amend the Tenacia Collaboration Agreement in connection with Tenacia’s exercise of its right of first negotiation and for which there is no other Licensed Product approved in China (for clarity, the milestone payments under this clause (ii) will only apply to one Selected Product), up to an aggregate amount of $256 million. Of the milestones, $15 million relates to regulatory approvals with separate milestones related to each of oral and intravenous formulations and the Selected Product, and an aggregate of $241 million of sales-based milestones are connected to annual revenue thresholds specific to each of the oral, intravenous and Selected Product formulations of ganaxolone.

Tenacia has further agreed to pay us tiered royalty payments based on annual net sales of Licensed Products ranging from the low double digits to the mid-teens for each of the oral formulation, intravenous formulation and Selected Product formulation of Licensed Products. Tenacia’s obligations to pay royalties to us with respect to sales of a Licensed Product in each particular jurisdiction of the Territory will commence on the date of first commercial sale in such jurisdiction and expire upon the latest of (i) ten years following the first commercial sale of such Licensed Product in such jurisdiction, (ii) the expiration of the last-to-expire valid claim of any licensed patent rights that covers such Licensed Product in such jurisdiction and (iii) the expiration of all regulatory exclusivities for such Licensed Product in such jurisdiction. Royalty payments are subject to reduction in specified circumstances as set forth in the Tenacia

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Collaboration Agreement, including if net sales decrease by a certain percentage after the introduction of a generic product.

Biologix Distribution and Supply Agreement

In May 2023, we entered into an exclusive distribution and supply agreement (Biologix Agreement) with Biologix, whereby Biologix has the right to distribute and sell ganaxolone inAlgeria, Bahrain, Egypt, Iraq, Jordan, Kingdom of Saudi Arabia, Kuwait, Lebanon, Libya, Morocco, Oman, Qatar, Tunisia and United Arab Emirates. In exchange for distribution rights, we will be the exclusive supplier of our products to Biologix on terms set forth in the Biologix Agreement in exchange for a negotiated purchase price for the products. Upon execution of the Biologix Agreement, we received an upfront payment of $0.5 million which is to be recognized over the term of the agreement. We may be entitled to additional fees upon regulatory milestones. In the nine months ended September 30, 2023, we recorded less than $0.1 million of collaboration revenue related to the Biologix Agreement.

NovaMedica Agreement

In June 2013, we entered into a clinical development and collaboration agreement with NovaMedica LLC (NovaMedica) for commercialization of ganaxolone, and in December 2022, as amended in May 2023, we entered into a manufacturing and supply agreement with NovaMedica, whereby NovaMedica has the right to market and sell ganaxolone in Armenia, Azerbaijan, Belarus, Georgia, Kazakhstan, Kyrgyzstan, Moldova, Russia, Tajikistan, Turkmenistan, Ukraine and Uzbekistan. In exchange for distribution rights, we will be the exclusive supplier of our products to NovaMedica on terms set forth in the NovaMedica Agreement in exchange for a negotiated purchase price for the products. As of the first quarter of 2023, we have initiated limited sales of ZTALMY to NovaMedica to support on-going early access programs associated with patients from the Marigold Study.

Oaktree Credit Agreement

On May 11, 2021 (Closing Date) and as amended on May 17, 2021, May 23, 2022 and October 28, 2022 (Credit Agreement), we entered into athe Credit Agreement and Guaranty (as amended by that certain letter agreement on May 17, 2021 and as further amended by that certain letter agreement on May 23, 2022 (the Credit Agreement), and as further amended by that certain Limited Consent and First Amendment to Credit Agreement on October 28, 2022 (the Credit Agreement Amendment) with Oaktree Fund Administration, LLC as administrative agent (Oaktree) and the lenders party thereto (collectively, ~~the~~ Lenders) that ~~provides~~provided for a five-year senior secured term loan facility in an aggregate original principal amount of up to ~~$100.0~~$125.0 million that was available to us in ~~four~~five tranches (collectively, ~~the~~ Term Loans). ~~Upon entering into the Credit Agreement in May 2021,~~As of September 30, 2023, we ~~borrowed~~had drawn on three tranches with no additional funds available thereunder.

We received $15.0 million ~~in term loans from the Lenders (Tranche~~of Tranche A-1 Term ~~Loans), upon receipt~~Loans on the Closing Date, $30.0 million of ~~written~~Tranche A-2 Term Loans in September 2021 after formal acceptance by the FDA of ~~our~~an NDA filing relating to the use of ganaxolone in the treatment of CDD, ~~in September 2021, we borrowed~~and $30.0 million of Tranche ~~A-2~~B Term Loans ~~from the Lenders (Tranche A-2 Term Loans), and~~ in March 2022 ~~we borrowed $30.0 million in term loans from the Lenders that became available as a result of the~~after FDA approval ~~by the FDA~~ of ZTALMY ~~oral suspension~~ for the treatment of seizures associated with CDD in patients two years of age and older (Tranche B Term Loans). In May 2022, we entered into an amendment to extend the commitment date for the tranche C term loans (Tranche C Term Loans) commitment from June 30, 2023 to December 31, 2023, and to eliminate the commitment fees associated with the Tranche C Term Loans. In May 2022, we also delivered to the Oaktree a separate notice of commitment termination with respect to the tranche D term loans (Tranche D Term Loans) commitment. Under the terms of the Credit Agreement, we may, at our sole discretion, borrow from the Lenders up to an additional $25.0 million in Term Loans subject to the following milestone event:CDD.

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Through December 31, 2023, $25.0 million of Tranche C Term Loans will be available for draw if we complete one or more financings (including through the issuance of common stock, convertible debt, subordinated debt, a synthetic royalty or a sublicense) resulting in gross proceeds to us of at least $40.0 million and net proceeds to us of at least $36.0 million. In addition, the availability of this tranche is subject to either our current Phase 3 trial in RSE or a Phase 3 trial in TSC achieving statistical significance

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~~(p value < 0.05) across all primary endpoints and ganaxolone must be generally well tolerated, with a safety profile generally consistent with previous clinical trials.~~

The Term Loans mature on May 11, 2026 (Maturity Date). The Term Loans bear interest at a fixed per annum rate (subject to increase during an event of default) of 11.50%, and we are required to make quarterly interest payments until the Maturity Date. We are also required to make quarterly principal payments beginning on June 30, 2024 in an amount equal to 5.0% of the aggregate amount of the Term Loans outstanding on June 30, 2024, and continuing until the Maturity Date. On the Maturity Date, we are required to pay in full all outstanding Term Loans and other amounts owed under the Credit Agreement.

At the time of borrowing any tranche of the Term Loans, we ~~are~~were required to pay an upfront fee of 2.0% of the aggregate principal amount borrowed at that time. In addition, a commitment fee of 75 basis points per annum began to accrue on each of the tranche B, C and D commitments for the period beginning 120 days after the funding date of the Tranche A-2 Term Loans, and continued to accrue until the applicable tranche was either funded or terminated, at which time the related commitment fees were due. The Tranche A-2 Term Loans were funded on September 27, 2021, and as such, we began accruing the commitment fees for tranche B, C, and D term loans 120 days later, on January 25, 2022. We drew down the additional $30.0 million of Tranche B Term Loans in March 2022, and paid less than $0.1 million in commitment fees related to Tranche B Term Loans. The May 2022 amendment eliminated the commitment fees related to the Tranche C Term Loans, and separately, we terminated the Tranche D Term Loans in May 2022. As of September 30, 2022, we did not have any accruals for commitment fees related to the Term Loans, and we will not incur additional commitment fees in the future.

In connection with the Revenue Interest Financing Agreement, on October 28, 2022, we entered into an amendment to the Credit Agreement ~~Amendment~~ to, among other things, allow for the consummation of the Revenue Interest Financing Agreement and the transactions ~~thereunder.~~thereunder, and paid $0.3 million in administrative fees in connection with the execution of the amendment. In addition, the ~~Credit Agreement Amendment~~amendment increased the exit fee due by us upon any repayment, whether

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as a prepayment or a scheduled repayment, of the principal of the loans under the Credit Agreement from 2.00% to 2.67%.

Sagard Financing Agreement

In October 2022, we entered into a revenue interest financing agreement (the Revenue Interest Financing Agreement) with Sagard Healthcare Royalty Partners, LP (Sagard) pursuant to which we received $32.5 million.

In exchange for the Investment Amount, we have agreed to make quarterly payments to Sagard (Payments) as follows: (i) for each calendar quarter from and after the closing date of such financing through and including the quarter ended June 30, 2026, an amount equal to 7.5% of (a) our U.S. net sales of ZTALMY and all other pharmaceutical products that contain ganaxolone (Net Sales), in each case with any dosage form, dosing regimen, or strength, or any improvements related thereto (collectively, Included Products) and (b) certain other payments received by us in connection with the manufacture, development and sale of the Included Products in the U.S. (Other Included Payments, and, together with Net Sales, Product Revenue); and (ii) for each calendar quarter following the calendar quarter ended June 30, 2026, an amount equal to (x) 15.0% of the first $100 million in annual Product Revenue of the Included Products and (y) 7.5% of annual Product Revenue of the Included Products in excess of $100 million.

The Payments are subject to a hard cap equal to 190% ($61.8 million) of the Investment Amount (Hard Cap). Sagard’s right to receive payments will terminate when Sagard has received payments in respect of the Included Products, including any additional payments described below, equal to the Hard Cap. Further, we have the right to make voluntary prepayments to Sagard, and such payments will be credited against the Hard Cap.

BARDA Contract

In September 2020, we and BARDA entered into the BARDA Contract, under which we received an award of up to an estimated $51 million for development of IV-administered ganaxolone for the treatment of RSE. The BARDA Contract provides for funding to support, on a cost-sharing basis, the completion of a Phase 3 clinical trial of IV-administered ganaxolone in patients with RSE, which covers the RAISE trial, funding of pre-clinical studies to evaluate IV-administered ganaxolone as an effective treatment for RSE due to chemical nerve gas agent exposure, and funding of certain ganaxolone manufacturing scale-up and regulatory activities. In March 2022, we entered into an amendment with BARDA to extend the end date of our base performance period for funding under the BARDA Contract from September 1, 2022 to December 31, 2023. In September 2022, we entered into an amendment with BARDA that, among other things, (i) provides for the exercise of BARDA’s option under the BARDA Contract to support U.S. onshoring of the manufacturing capabilities for ganaxolone API (Option 2), (ii) changes the end of date of our performance period under Option 2 from December 31, 2026 to July 31, 2025, (iii) increases the government cost share amount under Option 2 from approximately $11.5 million to approximately $12.3 million, and (iv) increases our cost share amount under Option 2 from approximately $4.9 million to approximately $5.3 million. In September 2023, we entered into an amendment with BARDA to extend the end date of our base performance period for funding under the BARDA Contract from December 31, 2023 to September 30, 2024.

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approximately $33 million and BARDA will be responsible for approximately $52 million if all development options are completed. The contract period-of-performance (base period plus option exercises) is up to approximately five years.

41Equity Financings

~~Table~~In connection with the closing of ~~Contents~~an equity financing in November 2022 and the December 2022 exercise of the related underwriters’ option, we issued a total of 12,421,053 shares of common stock and 2,105,264 pre-funded warrants to purchase common stock in an underwritten public offering resulting in aggregate net proceeds of $64.5 million, after deducting the underwriting discounts and commissions and offering expenses paid or payable by us.

Equity Distribution Agreement

~~In October 2017,~~On July 9, 2020, we entered into an Equity Distribution Agreement ~~(Prior EDA)~~(EDA) with JMP Securities LLC (JMP), ~~under which JMP,~~ as ~~our exclusive agent, at our discretion and at such times that we may determine from time~~amended by the March 31, 2023 Amendment No. 1 to ~~time, may sell over a three-year period from~~ the ~~execution of the agreement up to a maximum of $50 million of shares of our common stock. On July 9, 2020, we entered into a new Equity Distribution Agreement (New EDA) with JMP~~EDA (Amended Agreement), to create an at the market equity program under which we from time to time may offer and sell shares of our common stock ~~having an~~without a maximum aggregate offering ~~price~~price. The Amended Agreement was entered into in connection with our filing of a Registration Statement on Form S-3 (File No. 333-271041) with the SEC (the 2023 Registration Statement), which includes a prospectus supplement covering the offering, issuance and sale by us of up to ~~$60 million through or to JMP.~~$75,000,000 of shares of common stock that may be issued and sold under the Amended Agreement. Subject to the terms and conditions of the ~~New EDA, JMP will use its commercially reasonable efforts to sell shares of our common stock from time to time, based upon our instructions.~~Amended Agreement, JMP will be entitled to a commission of up to 3.0% of the gross proceeds from each sale of shares of our common stock. ~~The New EDA superseded and terminated~~In the ~~Prior EDA effective immediately upon effectiveness~~nine months ended September 30, 2023, we sold 3.7 million shares of our ~~shelf registration statement on Form S-3 (File No. 333-239780) filed with~~common stock pursuant to the ~~SEC on July 9, 2020 and declared effective by the SEC on July 27, 2020.~~Amended Agreement, which consisted of net proceeds of approximately $25.9 million at an average price of $7.17 per share. We did not sell any shares of our common stock during the ~~three and~~ nine months ended September 30, 2022 ~~or during the year ended December 31, 2021~~ under the ~~New~~ EDA.

IP License Agreement

In March 2022, we entered into the License Agreement with Ovid. Under the License Agreement, we have an exclusive, non-transferable (except as provided in the License Agreement), royalty-bearing, sublicensable license under certain of Ovid’s patent(s) and patent applications to develop, make, have made, commercialize, promote, distribute, sell, offer for sale and import, ganaxolone, including any analogues or derivatives, including its salts, and pharmaceutical formulations of the foregoing (Licensed Products), in the U.S., the member states of the EU, Iceland, Lichtenstein, Norway, the United Kingdom, and Switzerland (Territory) for the treatment of CDD in humans (Field). Under the License Agreement, we have the sole right and responsibility for, and control over, all development, manufacturing, and commercialization activities, including all regulatory activities, with respect to the Licensed Products in the Field in the Territory. In addition, all regulatory approvals and related filings with respect to the Licensed Products in the Field in the Territory will be in the name of, and be owned solely by, us. We were required, at Ovid’s option exercisable in accordance with the License Agreement, to (i) pay to Ovid the sum of $1.5 million in cash; or (ii) issue to Ovid 123,255 shares of our common stock, which option to obtain shares of our common stock was exercisable within the five-business day period following the filing of our Annual Report on Form 10-K for the year ended December 31, 2021 on March 24, 2022. On March 29, 2022, we issued 123,255 shares of our common stock to Ovid, per Ovid’s option in accordance with the License Agreement. As such, we recorded $1.2 million of IP license fee expenses related to the Ovid License Agreement in the nine months ended September 30, 2022.

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Cash Flows

Operating Activities. Cash used in operating activities ~~increased to~~was $91.0 million for the nine months ended September 30, ~~2022~~2023 and remained consistent compared to ~~$33.7~~ $91.0 million for the same period in ~~2021.~~2022. Excluding the noncash impacts primarily related to depreciation and amortization, debt issuance costs, accretion of revenue interest financing and amortization of discounts on short-term investments, stock-based compensation, cost of license agreement and changes in the net contract assets/liabilities related to the Orion, ~~Collaboration Agreement,~~Tenacia and Biologix collaboration agreements, the change in cash used in operating activities for the nine months ended September 30, ~~2022 compared~~2023 to the same period in ~~2021,~~2022, was primarily the result of decreases in the changes in accounts payable, accrued expenses, ~~and~~ other long term-liabilities, prepaid expenses, other current assets, inventory and accounts receivable and an increase in operating expenses.

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Investing Activities. Cash used in investing activities for the nine months ended September 30, 2023 primarily represents $52.0 million in purchases of short-term investments and partially offset by $17.0 million in maturities of short-term investments. Cash provided by investing activities for the nine months ended September 30, 2022 represents net proceeds of $107.4 million from the sale of the PRV, partially offset by $1.7 million in purchases of property and equipment.

Financing Activities. Cash ~~used in investing~~provided by financing activities for the nine months ended September 30, ~~2021~~2023 represents ~~$2.2~~$25.9 million in ~~deposits on and purchases~~net proceeds related to the sale of ~~property and equipment,~~common stock in connection with the EDA as amended, $0.8 million in proceeds from the exercise of stock options, partially offset by $0.7 million of payments in connection with the ~~maturity of short-term investments of $1.5 million.~~

Revenue Interest Financing ~~Activities.~~Agreement with Sagard. Cash provided by financing activities for the nine months ended September 30, 2022 includes $28.8 million in proceeds from notes payable, net of issuance costs, and $1.8 million in proceeds from the exercise of stock options. Cash provided by financing activities for the nine months ended September 30, 2021 includes $40.3 million in proceeds from notes payable, net of issuance costs and $0.9 million in proceeds from the exercise of stock options, partially offset by $0.1 million of debt issuance costs.

Funding Requirements

~~Other~~Since inception, other than for the three months ended September 30, 2022 asdue to a ~~result of the~~ one-time ~~receipt of proceeds~~net gain from the sale of our PRV, we have incurred net losses and negative cash flows from our operations. We incurred a net loss of $99.6 million for the ~~PRV~~nine months ended September 30, 2023. We have generated limited product revenues, and there is no assurance that profitable operations will be achieved in the ~~third quarter of 2022 to Novo Nordisk, Inc., we have not~~future, and if achieved, ~~profitability since our inception, and we expect to continue to incur net losses for the foreseeable future.~~could be sustained on a continuing basis.

We had cash, ~~and~~ cash equivalents and short-term investments of ~~$168.2 at~~$176.4 million as of September 30, ~~2022.~~2023. We believe that our existing cash, ~~and~~ cash equivalents ~~on hand~~and short-term investments as of September 30, ~~2022 and the $32.5 million payment from Sagard Healthcare Partners received in October 2022~~2023 will be sufficient to fund our operating expenses and capital expenditure requirements, ~~and~~as well as maintain the minimum cash balance required under our debt facility, ~~into~~for at least twelve months from the ~~first quarter of 2024.~~date these financial statements are issued. However, we will need to secure additional funding in the future, from one or more equity or debt financings, government funding, collaborations, licensing transactions, other ~~strategic~~commercial transactions or other sources in order to carry out all of our commercialization and planned research and development activities with respect to ganaxolone. In order to meet these additional cash requirements, we may seek to sell additional equity or convertible debt securities that may result in dilution to our stockholders, or engage in federal contracts or other partnerships. If we raise additional funds through the issuance of convertible debt securities, these securities could have rights senior to those of our common stock and could contain covenants that restrict our operations. There can be no assurance that we will be able to obtain additional equity or debt financing on terms acceptable to us, if at all. Further, the continued spread of COVID-19 has also led to severe disruption and volatility in the global capital markets, which could increase our cost of capital and adversely affect our ability to access the capital markets in the future. Our failure to obtain sufficient funds on acceptable terms when needed could have a negative impact on our business, results of operations, and financial condition.

Our future capital requirements will depend on many factors, including:

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the ~~timing and success of our commercialization of ZTALMY;~~

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~~the ongoing and planned development, formulation and commercialization activities related to ganaxolone;~~

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~~the~~ effects of the COVID-19 pandemic on our business, the medical community and the global economy;

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the results of our preclinical studies and clinical trials;

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the development, formulation and commercialization activities related to ganaxolone, including ZTALMY;

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the scope, progress, results and costs of researching and developing ganaxolone, including ZTALMY, or any other future product candidates, and conducting preclinical studies and clinical trials;

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the timing of, and the costs involved in, obtaining regulatory approvals for ganaxolone, including ZTALMY, or any other future product candidates;

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the cost of commercialization activities for ZTALMY in CDD in the U.S., including marketing, sales and distribution costs;

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the cost of commercialization activities for ZTALMY, ganaxolone in any other indications, or any other future product candidates, ~~that~~ are approved for sale, including marketing, sales and distribution costs;

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the cost of manufacturing and formulating ganaxolone, or any other future product candidates, to internal and regulatory standards for use in preclinical studies, clinical trials and, if approved, commercial sale;

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our ability to establish and maintain strategic collaborations, licensing or other arrangements and the financial terms of such agreements;

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our ability to receive funding under the BARDA Contract;

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our expectations regarding the amount and timing of milestone and royalty payments pursuant to our exclusive license ~~agreement~~agreements with Orion ~~for the commercialization of ganaxolone in Europe;~~

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~~our eligibility for the additional debt tranche under the Credit Agreement with Oaktree;~~and Tenacia.

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any product liability, infringement or other lawsuits related to ~~our product candidates~~ZTALMY or other indications being developed for ganaxolone and, if approved, products;

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capital needed to attract and retain skilled personnel;

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the costs involved in preparing, filing, prosecuting, maintaining, defending and enforcing patent claims, including litigation costs and the outcome of such litigation;

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~~our ability to resolve any potential disputes with Purdue relating to the sale of the PRV;~~ and

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the timing, receipt and amount of sales of, or royalties on, ZTALMY in CDD and on future approved products, ~~including payments payable to Ovid.~~if any.

Please see the Risk Factors section included in our Annual Report on Form 10-K for the year ended December 31, ~~2021~~2022 filed with the SEC on March ~~24, 2022~~9, 2023 for additional risks associated with our substantial capital requirements.

Off-Balance Sheet Arrangements

We do not have any off-balance sheet arrangements that have or are reasonably likely to have a current or future effect on our financial condition, changes in financial condition, revenues or expenses, results of operations, liquidity, capital expenditures or capital resources that are material to investors.

Discussion of Critical Accounting Policies and Significant Judgments and Estimates

The preparation of financial statements in conformity with GAAP requires us to use judgment in making certain estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities and the reported amounts of revenues and expenses in our financial statements and accompanying notes. Critical accounting policies are those that are most important to the portrayal of our financial condition and results of operations and require difficult, subjective and complex judgments by management in order to make estimates about the effect of matters that are inherently uncertain. During the three months ended September 30, ~~2022,~~2023, there were no

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significant changes to our critical accounting policies from those described in our annual financial statements for the year ended December 31, ~~2021,~~2022, which we included in our Annual Report on Form 10-K and was filed with the SEC on March ~~24, 2022.~~9, 2023.

Item 3. Quantitative and Qualitative Disclosures About Market Risk

We are a smaller reporting company as defined by Rule 12b-2 of the Securities Exchange Act of 1934, as amended (Exchange Act) and are not required to provide the information under this item.

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Item 4. Controls and Procedures

(a) 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 disclosure controls and procedures (as such term is defined in Rules 13a-15(e) and 15d-15(e) under the Exchange Act) as of the end of the period covered by this Quarterly Report on Form 10-Q 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 SEC rules and forms, and that information required to be disclosed in the reports we file or submit under the Exchange Act is accumulated and communicated to our management, including our Chief Executive Officer and Chief Financial Officer, to allow timely decisions regarding required disclosures. Management recognizes that any controls and procedures, no matter how well designed and operated, can provide only reasonable assurance of achieving their objectives and management necessarily applies its judgment in evaluating the cost benefit relationship of possible controls and procedures. Based on such evaluation, our Chief Executive Officer and Chief Financial Officer have concluded that our disclosure controls and procedures were effective at the reasonable assurance level as of September 30, ~~2022.~~2023.

(b) Changes in Internal Control Over Financial Reporting

There was no change in our internal control over financial reporting identified in connection with the evaluation required by Rule 13a-15(d) and 15d-15(d) of the Exchange Act that occurred during the quarter ended September 30, ~~2022~~2023 that has materially affected, or is reasonably likely to materially affect, our internal control over financial reporting.

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PART II

OTHER INFORMATION

Item 1. Legal Proceedings

From time to time, we may become subject to litigation and claims arising in the ordinary course of business. We are not currently a party to any material legal proceedings, and we are not aware of any pending or threatened legal proceedings against us that we believe could have a material adverse effect on our business, operating results or financial condition.

Item 1A. Risk Factors

Except as set forth below, there have been no material changes to the risk factors previously disclosed in our Annual Report on Form 10-K for the fiscal year ended December 31, ~~2021.~~

Our failure to comply with the covenants or other terms of the Credit Agreement, including as a result of events beyond our control, could result in a default under the Credit Agreement or Revenue Interest Financing Agreement that could materially and adversely affect the ongoing viability of our business.

On May 11, 2021 (Credit Agreement Closing Date), we entered into a Credit Agreement and Guaranty (as amended by that certain letter agreement on May 17, 2021, that certain letter agreement on May 23, 2022 and that certain Limited Consent and First Amendment to Credit Agreement on October 28, 2022, the Credit Agreement) with Oaktree Fund Administration, LLC, as administrative agent (Oaktree) and the lenders party thereto (collectively, the Lenders) that provides for a five-year senior secured term loan facility in an aggregate original principal amount of up to $100.0 million, consisting of (i) tranche A-1 term loans in an aggregate principal amount of $15.0 million advanced on the Credit Agreement Closing Date; (ii) tranche A-2 term loans in an aggregate principal amount of $30.0 million advanced on September 27, 2021; (iii) tranche B term loans in an aggregate principal amount of $30.0 million advanced on March 30, 2022; and (iv) tranche C term loans in an aggregate principal amount of $25.0 million (collectively, the Term Loans). Our ability to draw each tranche of the Term Loans is subject to the satisfaction of certain conditions applicable to each tranche as specified in the Credit Agreement. The Term Loans bear interest at a fixed per annum rate (subject to increase during an event of default) of 11.50% and are scheduled to mature on the fifth anniversary of the Credit Agreement Closing Date (Maturity Date). In addition, at the time of funding of any tranche of the Term Loans, we are required to pay an upfront fee of 2.0% of the aggregate principal amount being funded. We are required to make quarterly interest payments until the Maturity Date. We are also required to make principal payments, which are payable in quarterly installments beginning on the last day of the first quarter ending after the third anniversary of the Credit Agreement Closing Date, in an amount equal to 5.0% of the aggregate amount of the Term Loans outstanding on the date of the first such quarterly principal payment and continuing until the Maturity Date, on which date all outstanding Term Loans and other amounts owed under the Credit Agreement will be required to be paid in full. A commitment fee of 75 basis points per annum will accrue on the tranche C commitments for the period beginning 120 days after the funding date of the tranche A-2 term loans until the applicable tranche is either funded or terminated. The Term Loans will be guaranteed by certain of our future subsidiaries. Our obligations under the Credit Agreement and the guarantee of such obligations are secured, subject to customary permitted liens and other agreed upon exceptions and subject to an intercreditor agreement with Sagard Healthcare Royalty Partners, LP (Sagard), by a pledge of substantially all of our assets and will be secured by a pledge of substantially all of the assets of the future guarantors. The Credit Agreement contains various covenants that limit our ability to engage in specified types of transactions without Oaktree's prior consent, as well as a financial covenant that requires us to maintain at all times cash and cash equivalents in certain deposit accounts in an amount at least equal to (i) from the funding date of the tranche A-2 term loans until the funding date of the tranche B term loans, $20.0 million, and (ii) from the funding of the tranche B term loans until the Maturity Date, $15.0 million.

Oaktree may elect to accelerate the repayment of all unpaid principal of the Term Loans, accrued interest and other amounts owed under the Credit Agreement upon consummation of a specified change of control transaction or the occurrence of certain events of default (as specified in the Credit Agreement), including, among other things:

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~~our default in a payment obligation under the Credit Agreement;~~

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~~our breach of the restrictive covenants or other terms of the Credit Agreement;~~

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~~our breach of reporting obligations;~~

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~~our failure to properly maintain the collateral;~~

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~~certain regulatory actions that cause an ongoing delay in commercialization of ganaxolone and which could reasonably be expected to result in a material adverse effect;~~

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~~a recall of ganaxolone that could reasonably be expected to result in a material adverse effect;~~

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following the sale of ganaxolone by us in the U.S. to treat Cyclin-dependent Kinase-like 5 Deficiency Disorder (CDD), an injunction against the sale or manufacture of ganaxolone for more than 45 days that could, after the termination of such 45-day period, reasonably be expected to result in a material adverse effect; and

●

~~certain specified insolvency and bankruptcy-related events.~~

Subject to any applicable cure period set forth in the Credit Agreement, all amounts outstanding with respect to the Term Loans (principal and accrued interest), as well as any applicable prepayment premiums, interest “make-whole” payments or exit fees, would become due and payable (i) immediately, in the case of a payment or bankruptcy event of default or (ii) in the case of any other event of default, upon the request of Lenders holding at least a majority of the outstanding Term Loans and Term Loan commitments, at a default interest rate of 13.50%. Our assets or cash flow may not be sufficient to fully repay our obligations under the Term Loans if the obligations thereunder are accelerated upon any events of default. The duration and magnitude of any negative impact from the COVID-19 pandemic on ganaxolone commercialization, development or net revenues could also affect our ability to meet the requirements to draw on one or more of the Term Loan tranches and to remain in compliance with our liquidity financial covenant. Further, if we are unable to repay, refinance or restructure our obligations under the Term Loans, Oaktree on behalf of the Lenders could proceed to protect and enforce their rights under the Credit Agreement and other loan documents by exercising such remedies (including foreclosure on the assets securing our obligations under the Credit Agreement and the other loan documents) as are available to Oaktree and the Lenders and in respect thereof under applicable law, either by suit in equity or by action at law, or both, whether for specific performance of any covenant or other agreement contained in the Credit Agreement or other loan documents or in aid of the exercise of any power granted in the Credit Agreement or other loan documents. The foregoing would materially and adversely affect the ongoing viability of our business.

On October 28, 2022 (the RIFA Closing Date), we entered into a revenue interest financing agreement (the Revenue Interest Financing Agreement) with Sagard Healthcare Royalty Partners, LP (Sagard) pursuant to which Sagard agreed to pay $32.5 million (the Investment Amount) to the Company to provide funding for the Company’s development and commercialization of ganaxolone and related pharmaceutical products, including the commercial launch of ZTALMY, and for working capital and general administrative purposes.

In exchange for the Investment Amount, the Company has agreed to make quarterly payments to Sagard (the Payments) as follows: (i) for each calendar quarter from and after the RIFA Closing Date through and including the quarter ended June 30, 2026, an amount equal to 7.5% of (a) the Company’s U.S. net sales of ZTALMY and all other pharmaceutical products that contain ganaxolone (Net Sales), in each case with any dosage form, dosing regimen, or strength, or any improvements related thereto (collectively, the Included Products); and (b) payments received by the Company in connection with the manufacture, development and sale of Included Products in the U.S., including in connection with any out-licensing of U.S. rights to any Included Product (Other Included Payments, and together with Net Sales, Product Revenue), and (ii) for each calendar quarter following the calendar quarter ended June 30, 2026, an amount equal to (x) 15.0% of the first $100.0 million in annual Product Revenues of the Included Products and (y) 7.5% of annual Product Revenues of the Included Products in excess of $100.0 million.

~~Table of Contents~~

The Payments are subject to a hard cap equal to 190% of the Investment Amount (the Hard Cap). Sagard’s right to receive payments will terminate when Sagard has received payments in respect of the Included Products, including any additional payments described below, equal to the Hard Cap. Further, the Company has the right to make voluntary prepayments to Sagard, and such payments will be credited against the Hard Cap.

The obligations under the Revenue Interest Financing Agreement, including the Payments, will be guaranteed by certain of the Company’s future subsidiaries (Subsidiaries) that are required to become a party thereto as guarantors (the Guarantors). The Company’s obligations under the Revenue Interest Financing Agreement and the guarantee of such obligations are secured, subject to customary permitted liens and other agreed upon exceptions and subject to an intercreditor agreement with Oaktree as administrative agent for the lenders under the Credit Agreement, by a pledge of substantially all of the Company’s and the Guarantors’ assets that relate to, or are used or held for use for, the development, manufacture, use and/or commercialization of ZTALMY and all other pharmaceutical products that contain ganaxolone in the U.S., including the Product Revenue, pursuant to the terms of the Security Agreement dated as of the RIFA Closing Date by and among the Company, the Guarantors from time to time party thereto, and Sagard (the Security Agreement).

At any time, the Company has the right, but not the obligation (the Call Option), to repurchase all, but not less than all, of Sagard’s interest in the Payments at a repurchase price (the Put/Call Price) equal to: (a) on or before the third anniversary of the RIFA Closing Date, 160% of the Investment Amount; (b) after the third anniversary but on or prior to the fourth anniversary of the RIFA Closing Date, 180% of the Investment Amount; and (c) after the fourth anniversary of the RIFA Closing Date, 190% of the Investment Amount, in each case, less the aggregate of all of the payments of the Company in respect of the Payments made to Sagard prior to such date.

The Revenue Interest Financing Agreement contains certain restrictions on the Company’s and its Subsidiaries’ abilities, among other things, to incur additional debt, grant or permit additional liens, make investments and acquisitions, dispose of assets, pay dividends and distributions and enter into affiliate transactions, in each case, subject to certain exceptions. In addition, the Revenue Interest Financing Agreement contains a financial covenant that requires the Company to maintain at all times cash and cash equivalents in certain deposit accounts in an amount at least equal to (i) from the RIFA Closing Date until the repayment of the loans under the Credit Agreement, $15.0 million and (ii) thereafter, $10.0 million.

In addition, the Revenue Interest Financing Agreement provides that if certain events occur, including certain bankruptcy events, a change of control, non-payment of Payments, divestiture of rights to commercialize Included Products in the U.S., divestiture of certain assets related to the Included Products (subject to customary carve-outs), and (subject to applicable cure periods) non-compliance with the covenants in the Revenue Interest Financing Agreement, Sagard has the right, but not the obligation, to require the Company to repurchase all, but not less than all, of Sagard’s interest in the Payments at the Put/Call Price. Our assets or cash flow may not be sufficient to fully repurchase all of Sagard’s interest in the Payments if such obligation is triggered upon any events of default. The duration and magnitude of any negative impact from the COVID-19 pandemic on ganaxolone commercialization, development or net revenues could also affect our ability to remain in compliance with our liquidity financial covenant. Further, if we are unable to repay, refinance or restructure our obligations under the Revenue Interest Financing Agreement, Sagard could proceed to protect and enforce its rights under the Revenue Interest Financing Agreement and other transaction documents by exercising such remedies (including foreclosure on the assets securing our obligations under the Revenue Interest Financing Agreement and the other transaction documents) as are available to Sagard and in respect thereof under applicable law, either by suit in equity or by action at law, or both, whether for specific performance of any covenant or other agreement contained in the Revenue Interest Financing Agreement or other transaction documents or in aid of the exercise of any power granted in the Revenue Interest Financing Agreement or other transaction documents. The foregoing would materially and adversely affect the ongoing viability of our business.

~~Table of Contents~~

~~If we are unable to satisfy certain conditions in our Credit Agreement, we will be unable to draw down the remaining amount of the term loan facility.~~

For our Credit Agreement, we must satisfy certain conditions to be eligible to draw down the tranche C term loans of $25.0 million. The tranche C term loans of $25.0 million may be drawn by us on or before December 31, 2023, provided that we satisfy certain conditions described in the Credit Agreement, including (i) the completion of one or more financings, including through the issuance of common stock, convertible debt, subordinated debt, a synthetic royalty or a sublicense in which we receive gross proceeds in an aggregate amount of at least $40.0 million and net proceeds in an aggregate amount of at least $36.0 million and (ii) either our current Phase 3 RAISE trial or a Phase 3 trial in tuberous sclerosis complex (TSC) achieving statistical significance (p value < 0.05) across all primary endpoints and ganaxolone being generally well tolerated, with a safety profile generally consistent with previous clinical trials. If we are unable to satisfy those conditions, we would not be able to draw down the tranche of loans and may not be able to obtain alternative financing on commercially reasonable terms or at all.

~~Our Credit Agreement and Revenue Interest Financing Agreement contains restrictions that limit our flexibility in operating our business.~~

The Credit Agreement and the Revenue Interest Financing Agreement contain various covenants that limit our ability to engage in specified types of transactions without the prior consent of Oaktree and the Lenders holding a majority of the Term Loan commitments and/or Sagard, as applicable,. These covenants limit our ability to, among other things:

●

~~sell, transfer, lease or dispose of our assets;~~

●

~~create, incur or assume additional indebtedness;~~

●

~~encumber or permit liens on certain of our assets;~~

●

~~make restricted payments, including paying dividends on, repurchasing or making distributions with respect to our common stock;~~

●

~~make specified investments (including acquisitions, loans and advances);~~

●

~~consolidate, merge, sell or otherwise dispose of all or substantially all of our assets;~~

●

~~enter into certain transactions with our affiliates;~~

●

~~grant certain license rights related to our products, technology and other intellectual property rights;~~

●

in the case of the Credit Agreement, permit our cash and cash equivalents held in certain deposit accounts to at any time be less than (i) from the funding of the tranche A-2 term loans until the funding of the tranche B term loans, $20.0 million and (ii) from the funding date of the tranche B term loans until the Maturity Date, $15.0 million; and

●

in the case of the Revenue Interest Financing Agreement, permit our cash and cash equivalents held in certain deposit accounts to be less than (i) from the RIFA Closing Date until the repayment of the loans under the Credit Agreement, $15.0 million and (ii) thereafter, $10.0 million.

The covenants in our Credit Agreement, Revenue Interest Financing Agreement and related security agreements may limit our ability to take certain actions that may be in our long-term best interests. In the event that we breach one or more covenants, Oaktree and/or Sagard may choose to declare an event of default and (i) in the case of the Credit Agreement, require that we immediately repay all amounts outstanding under the Credit Agreement, plus penalties and interest, terminate the Lenders’ commitments to fund any undrawn Term Loan tranches and foreclose on the collateral

~~Table of Contents~~

granted to them to secure the obligations under the Credit Agreement and the other loan documents and/or (ii) in the case of the Revenue Interest Financing Agreement, require that we repurchase all, but not less than all, of Sagard’s interest in the Payments and foreclose on the collateral granted to them to secure the obligations under the Revenue Interest Financing Agreement and the other transaction documents. Such repayment could have a material adverse effect on our business, operating results and financial condition.2022.

Third parties, such as Ovid Therapeutics, Inc., may initiate legal proceedings alleging that we are infringing their intellectual property rights, the outcome of which would be uncertain and could harm our business.

Our commercial success depends upon our ability to develop, manufacture, market and sell our products and product candidates, all of which contain ganaxolone, ~~if approved,~~ and to use our related technologies. We may become party to, or threatened with, adversarial proceedings or litigation regarding intellectual property rights with respect to one or more of our products, including interference or derivation proceedings before the ~~USPTO.~~U.S. Patent and Trademark Office (USPTO). Third parties may assert infringement claims against us based on existing patents or patents that may be granted in the future. If we are found to infringe a third party’s intellectual property rights, we could be required to obtain a license from such third party to continue commercializing one or more of our products. However, we may not be able to obtain any required license on commercially reasonable terms or at all. Under certain circumstances, we could be forced, including by court order, to cease commercializing one or more of our products. In addition, in any such proceeding or litigation, we could be found liable for monetary damages. A finding of infringement could prevent us from commercializing one or more of our products or force us to cease some of our business operations, which could materially harm our business. Any claims by third parties that we have misappropriated their confidential information or trade secrets could have a similar negative impact on our business.

While our product candidates are in preclinical studies and clinical trials, we believe that the use of our product candidates in these preclinical studies and clinical trials falls within the scope of the exemptions provided by 35 U.S.C. Section 271(e) in the ~~United States,~~U.S., which exempts from patent infringement liability activities reasonably related to the development and submission of information to the FDA (Federal Development Patent Infringement Exemption). As our product candidates progress toward commercialization, the possibility of a patent infringement claim against us increases. While ganaxolone itself is off patent, we attempt to ensure that our product candidates and the methods we employ to manufacture ganaxolone do not infringe other parties’ patents and other proprietary rights. There can be no assurance they do not, however, and competitors or other parties may assert that we infringe their proprietary rights in any event.

On July 26, 2022, the USPTO issued ~~a patent~~U.S. Patent No. 11,395,817 (Ovid ‘817 Patent) to Ovid Therapeutics, Inc. (Ovid) with claims that encompass our product candidate for the treatment of SE. On March 15, 2023, we filed a petition seeking post-grant review (PGR) of the Ovid ‘817 Patent with the U.S. Patent and Trademark Office Patent Trial and Appeal Board (PTAB). Our petition for PGR argues that the claims of the Ovid ‘817 Patent are unpatentable on multiple grounds. Ovid filed a preliminary response to our petition on June 20, 2023. In Ovid’s reply to our request for PGR, Ovid disclaimed claims 1-21, 23 and 24 of the Ovid ‘817 Patent, which has the effect of erasing these claims from the patent irrespective of the outcome of the PGR. On August 17, 2023, the PTAB issued a decision granting institution of our petition seeking PGR of the Ovid ‘817 Patent. In instituting the PGR, the PTAB stated that it was more likely than not that we would be able to invalidate the remaining claims (22 and 25-31) of the Ovid ‘817 Patent during the proceeding. The next steps in the PGR are replies by us and Ovid as well as depositions of the experts. The oral arguments are currently scheduled for May 22, 2024. The decision to institute is not a final decision on the patentability of the claims. The final decision will be based on the full record developed during the proceeding. If we do not prevail in

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the PGR proceeding, the decision can be appealed to the Court of Appeals for the Federal Circuit. If an appeal is not successful, our ability to challenge the Ovid ‘817 Patent in court will be limited in certain respects. The Ovid ‘817 Patent claims cover the use of ganaxolone in the treatment of SE and do not cover or impact our marketing and sales of ZTALMY for the treatment of seizures associated with CDD.

On September 27, 2023, the USPTO issued a Notice of Allowance in an Ovid patent application with claims that encompass our product candidate for the treatment of LGS. The patent is scheduled to issue on November 7, 2023. The claims in this Ovid LGS patent application cover the use of ganaxolone for the treatment of LGS and do not cover or impact the use of ganaxolone in any other indication.

Ovid may file a lawsuit against us alleging infringement of its ~~patents and/or we may challenge the validity of Ovid’s patents with the USPTO or through the courts.~~patents. Any such proceeding, in the PTAB or courts, regardless of ~~its~~their outcome, would likely result in the expenditure of significant financial resources and the diversion of management’s time and resources. In addition, any such proceeding may cause negative publicity, adversely impact patients, and we may be prohibited from marketing or selling ganaxolone for SE, RSE and ~~ESE~~LGS during such proceedings or if we are not successful in such proceedings. If Ovid does decide to bring an infringement lawsuit, we do not expect that it will be filed before a commercial launch of ganaxolone for SE, RSE or ~~ESE~~LGS based upon the “safe harbor” provisions of the Hatch-Waxman Act. We may need to acquire or obtain a license to ~~the~~certain Ovid patents to market or sell ganaxolone for SE, RSE or ~~ESE,~~LGS, which may not be available on commercially acceptable terms or at all. If we are not able to acquire ~~the~~certain Ovid patents or negotiate a license on acceptable terms, and if our product is determined to infringe Ovid’s patents and ~~the~~such patents are determined to be valid, then we may be forced to pay Ovid royalties, damages and costs, or we may be prevented from commercializing ganaxolone for SE, RSE and ~~ESE~~LGS altogether, which would have a material adverse impact on our business.

We have multiple ganaxolone drug products in development, and until such products are approved by regulatory authorities, there remains the risk that the drug product quality requirements may not support continued clinical investigation and result in delays or termination of such clinical studies, and product approvals.

We currently have multiple ganaxolone drug products in clinical development, including an oral suspension, IV solution and a new formulation for which top-line data from a Phase 1 trial with healthy volunteers were announced in the second quarter of 2022 and for which an additional Phase 1 cohort assessing the pharmacokinetics (PK) is planned.

~~Table of Contents~~

While we strive to develop a full understanding of manufacturing processes used, as well as the resultant product quality attributes, there is a risk that problems may arise over the course of development which could render a given drug product non-viable. Such problems could relate to manufacturing reproducibility, scale-up challenges, drug product chemical or physical stability issues. Related quality requirements may not support continued clinical investigation and result in delays or termination of such clinical studies and product approvals. Such quality requirements can include physical and chemical attributes of the drug product, stability and shelf life, microbial and other contamination, including adverse impact of drug product packaging and administration devices. These problems could result in unacceptable manufacturing economics, or direct concerns related to drug product safety or efficacy. For example, we announced in February 2022 a product supply interruption for our IV ganaxolone clinical supplies. Routine monitoring of stability batches of IV clinical supply material showed visible particulates of aluminum phosphate in the drug solution, which led to a pause in recruitment for the RAISE trial. In May 2022, we announced that the RAISE trial had resumed utilizing new batches of the current IV formulation of ganaxolone. In connection with the resumption of the trial and in consultation with the FDA, we have implemented a 12-month shelf life; refrigerated storage conditions for the entire duration of clinical use, including storage at the clinical sites; and frequent testing for visible particles. If we experience issues with product quality requirements and we are unable to resolve these issues in a timely manner or at all, we may need to delay or terminate our RAISE or other clinical trials with the current IV formulation, which could further delay our clinical development plans and future product approvals.

Item 2. Unregistered Sales of Equity Securities and Use of Proceeds

~~The following table provides certain information with respect to purchase of our common stock during the three and nine months ended September 30, 2022:~~None.

Maximum Number

Total Number of

(or Approximate Dollar Value)

Total Number

Average

Shares Purchased as Part

of Shares That May

of Shares

Price Paid

of Publicly Announced

Yet Be Purchased
Period

Purchased (1)

Per Share

Plans or Programs

Under the Plans or Programs
January 1, 2022 through January 31, 2022

-

$
-

-

$
-
February 1, 2022 through February 28, 2022

-

-

-

-
March 1, 2022 through March 31, 2022

1,236

8.59

-

-
April 1, 2022 through April 30, 2022

2,251

7.93

-

-
May 1, 2022 through May 31, 2022

-

-

-

-
June 1, 2022 through June 30, 2022

1,505

12.68

-

-
July 1, 2022 through July 31, 2022

-

-

-

-
August 1, 2022 through August 31, 2022

-

-

-

-
September 1, 2022 through September 30, 2022

5,810

13.16

-

-
Total

10,802

$
11.48

-

$
-

~~(1)~~

~~Represents shares of common stock withheld to satisfy taxes associated with the vested of restricted stock~~

Item 3. Defaults upon Senior Securities

None.

Item 4. Mine Safety Disclosures

Not applicable.

Item 5. Other Information

None.

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Item 6. ~~Exhibits~~Exhibits

Exhibit Number		Exhibit Description

3.1		Fourth Amended and Restated Certificate of Incorporation. (Incorporated by reference to Exhibit 3.1 to Form 8-K current report filed on August 7, 2014.)
3.2		Certificate of Amendment of the Fourth Amended and Restated Certificate of Incorporation. (Incorporated by reference to Exhibit 3.1 to Form 8-K current report filed on April 2, 2020.)
3.3		Certificate of Amendment of the Fourth Amended and Restated Certificate of Incorporation. (Incorporated by reference to Exhibit 3.1 to Form 8-K current report filed on May 27, 2020.)
3.4		Certificate of Amendment of the Fourth Amended and Restated Certificate of Incorporation. (Incorporated by reference to Exhibit 3.1 to Form 8-K current report filed on September 22, 2020.)
3.5		Certificate of Amendment of the Fourth Amended and Restated Certificate of Incorporation. (Incorporated by reference to Exhibit 3.2 to Form 8-K current report filed on September 22, 2020.)
3.6		Amended and Restated By-laws. (Incorporated by reference to Exhibit 3.2 to Form 8-K current report filed on August 7, 2014.)
3.7		Certificate of Designations, Preferences and Rights of Series A Participating Convertible Preferred Stock. (Incorporated by reference to Exhibit 3.1 to Form 8-K current report filed on December 13, 2019.)
3.8		Delaware Certificate of Change of Registered ~~Agent~~Agent. (Incorporated by reference to Exhibit 3.8 to Form 10-Q quarterly report filed on May 12, 2022.)
4.1		Specimen Certificate evidencing shares of Marinus Pharmaceuticals, Inc.’s common stock. (Incorporated by reference to Exhibit 4.1 to Form S-1/A registration statement filed on July 18, 2014.)
~~10.1*+~~4.2		~~Asset~~Form of Pre-funded Warrant to Purchase ~~Agreement, dated July 13, 2022, by and between Marinus Pharmaceuticals, Inc. and Novo Nordisk Inc.~~Common Stock. (Incorporated by ~~referenced~~reference to Exhibit ~~10.1~~4.1 to Form 8-K current report filed on ~~July 14,~~November 10, 2022.)
~~10.2+~~		Amendment No. 2, dated September 21, 2022, by and between Marinus Pharmaceuticals, Inc. and the Biomedical Advanced Research and Development Authority, a division of the U.S. Department of Health and Human Services’ Office of the Assistant Secretary for Preparedness and Response. (Incorporated by reference to Exhibit 10.1 to Form 8-K current report filed on September 23, 2022.)
~~10.3+~~		~~Revenue Interest Financing Agreement, dated October 28, 2022, by and between Marinus Pharmaceuticals, Inc. and Sagard Healthcare Royalty Partners, LP. (Filed herewith.)~~
~~10.4+~~		~~Security Agreement dated October 28, 2022, by and among Marinus Pharmaceuticals, Inc. and Sagard Healthcare Royalty Partners, LP. (Filed herewith.)~~
~~10.5+~~		Limited Consent and First Amendment to Credit Agreement, dated October 28, 2022, by and among Marinus Pharmaceuticals, Inc., as Grantor, and Oaktree Fund Administration, LLC, as Administrative Agent. (Filed herewith.)
31.1		Certification of Chief Executive Officer pursuant to Rule 13a-14(a) or 15a-14(a) under the Exchange Act (filed herewith.)
31.2		Certification of Chief Financial Officer pursuant to Rule 13a-14(a) or 15a-14(a) under the Exchange Act (filed herewith.)
32.1		Certification Pursuant to 18 U.S.C. Section 1350 of principal executive officer and principal financial officer (furnished herewith.)
101.INS		XBRL Instance 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		XBRL Taxonomy Extension Schema
101.CAL		XBRL Taxonomy Extension Calculation Linkbase
101.DEF		XBRL Taxonomy Extension Definition Linkbase
101.LAB		XBRL Taxonomy Extension Labels Linkbase
101.PRE		XBRL Taxonomy Extension Presentation Linkbase
104		Cover Page Interactive Data File formatted as Inline XBRL and contained in Exhibit 101

* Portions of this exhibit have been omitted in compliance with Item 601 of Regulation S-K.

~~+ Schedules have been omitted pursuant to Item 601(a)(5) of Regulation S-K. The registrant undertakes to furnish supplemental copies of any of the omitted schedules upon request by the SEC.~~

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Table of Contents

SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned thereunto duly authorized.

Signature	Title	Date

/s/ SCOTT BRAUNSTEIN, M.D.	President, ~~and~~ Chief Executive Officer ~~(principal executive officer)~~(Principal Executive Officer), Chairman of the Board and Director	November 7, ~~2022~~2023
Scott Braunstein, M.D.		November 7, ~~2022~~2023

/s/ STEVEN PFANSTIEL	Chief Operating Officer, Chief Financial Officer and Treasurer ~~(principal financial~~(Principal Financial and ~~accounting officer)~~Accounting Officer)	November 7, ~~2022~~2023
Steven Pfanstiel		November 7, ~~2022~~2023

5356



					Maximum Number
				Total Number of	(or Approximate Dollar Value)
	Total Number	Average		Shares Purchased as Part	of Shares That May
	of Shares	Price Paid		of Publicly Announced	Yet Be Purchased
Period	Purchased (1)	Per Share		Plans or Programs	Under the Plans or Programs
January 1, 2022 through January 31, 2022	-	$	-	-	$	-
February 1, 2022 through February 28, 2022	-		-	-		-
March 1, 2022 through March 31, 2022	1,236		8.59	-		-
April 1, 2022 through April 30, 2022	2,251		7.93	-		-
May 1, 2022 through May 31, 2022	-		-	-		-
June 1, 2022 through June 30, 2022	1,505		12.68	-		-
July 1, 2022 through July 31, 2022	-		-	-		-
August 1, 2022 through August 31, 2022	-		-	-		-
September 1, 2022 through September 30, 2022	5,810		13.16	-		-
Total	10,802	$	11.48	-	$	-