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

Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, anon-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” inRule 12b-2 of the Exchange Act.

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 inRule 12b-2 of the Exchange Act). Yes ☐ No ☒

As of ~~October 31, 2017,~~November 8, 2022, there were ~~27,489,457~~43,269,200 shares of the registrant’s Common Stock, $0.001 par value per share, outstanding.

Statements made in this Quarterly Report on Form10-Q ~~Report,~~ that are not statements of historical or ~~Quarterly Report, contains forward-looking statements that involve risks and uncertainties. We make such forward-looking statements pursuant to~~current facts are “forward-looking statements” within the ~~safe harbor provisions~~meaning of the Private Securities Litigation Reform Act of ~~1995~~1995. Forward-looking statements discuss our business, operations and ~~other federal securities laws. All statements other than statements of historical facts contained in this Quarterly Report are forward-looking statements.~~financial performance and conditions, as well as our plans, objectives and expectations for our business operations and financial performance and condition. In some cases, you can identify forward-looking statements by terminology such as “aim,” “anticipate,” “assume,” “believe,” “contemplate,” “continue,” “could,” “design,” “due,” “estimate,” “expect,” “goal,” “intend,” “may,” “objective,” “plan,” “predict,” “positioned,” “potential,” “seek,” “should,” “target,” “will,” ~~“should,” “expects,” “intends,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “potential,” “continue”~~“would” and other similar expressions that are predictions of or indicate future events and future trends, or the negative of these terms or other comparable terminology. In addition, statements that “we believe” or similar statements reflect our beliefs and opinions on the relevant subject only. These forward-looking statements, which are subject to risks, uncertainties and assumptions about us, may include ~~but are not limited to, statements about:~~

You should understand that the following important factors could affect our future results and could cause those results or other outcomes to differ materially from those expressed or implied in our forward-looking statements:

~~Any~~These forward-looking statements are based on management’s current expectations, estimates, forecasts and projections about our business and the industry in ~~this Quarterly Report reflect our current views with respect to~~which we operate, and management’s beliefs and assumptions are not guarantees of future ~~events~~performance or ~~to our future financial performance~~development and involve known and unknown risks, uncertainties and other factors that ~~may cause~~are in some cases beyond our ~~actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by~~control. In light of the significant uncertainties in these forward-looking statements. Factors that may cause actual results to differ materially from current expectations include, among other things, those listed under Part II, Item 1A. Risk Factors and elsewhere in this Quarterly Report. Given these uncertainties,statements, you should not ~~place undue reliance~~rely upon forward-looking statements as predictions of future events. Although we believe the expectations reflected in the forward-looking statements are reasonable, the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements may not be achieved or occur at all. The factors that could cause or contribute to such differences include, but are not limited to, those discussed in our Annual Report on ~~these~~Form 10-K filed on March 25, 2022. All forward-looking ~~statements. Except~~statements are applicable only as of the date on which they were made and, except as required by law, we ~~assume~~undertake no obligation to publicly update ~~or revise these~~any forward-looking statements, ~~for any reason, even if~~whether as a result of new information, ~~becomes available in~~future events or otherwise after the ~~future.~~

~~This~~date of this Quarterly Report ~~also contains estimates, projections~~on Form 10-Q or to reflect the occurrence of any unanticipated events. Comparisons of results for current and ~~other information concerning our industry, our business,~~any prior periods are not intended to express any future trends or indications of future performance, unless expressed as such, and the markets for certain diseases, including data regarding the estimated size of those markets, and the incidence and prevalence of certain medical conditions. Information that is based on estimates, forecasts, projections, market research or similar methodologies is inherently subject to uncertainties and actual events or circumstances may differ materially from events and circumstances reflected in this information. Unless otherwise expressly stated, we obtained this industry, business, market and other data from reports, research surveys, studies and similar data prepared by market research firms and other third parties, industry, medical and general publications, government data and similar sources.


	September 30,			December 31,			September 30,			December 31,
	2017			2016			2022			2021
Assets
Current assets:
Cash and cash equivalents	$	19,193		$	32,352		$	86,047		$	70,097
Marketable securities		73,987			96,842
Tax incentive receivable		469			347
Marketable debt securities								38,652			—
Prepaid expenses and other current assets		2,007			1,358			2,428			2,107

Total current assets		95,656			130,899			127,127			72,204
Property and equipment, net		551			661			909			1,049
Operating lease right-of-use assets								2,997			3,406
Restricted cash								1,339			1,339
Other assets		57			61			643			669

Total assets	$	96,264		$	131,621		$	133,015		$	78,667

Liabilities and Stockholders’ Equity
Current liabilities:
Accounts payable	$	2,676		$	2,572		$	849		$	1,660
Accrued expenses		3,090			3,733			7,939			6,592
Notes payable, current		1,268			3,589

Operating lease liabilities, current								632			594
Total current liabilities		7,034			9,894			9,420			8,846

Operating lease liabilities								4,933			5,408
Total liabilities		7,034			9,894			14,353			14,254

Commitments and contingencies (Note 7)
Commitments and contingencies (See Note 8)
Stockholders’ equity:
Preferred stock; $0.001 par value per share; 5,000,000 shares authorized as of September 30, 2017 and December 31, 2016; no shares issued and outstanding as of September 30, 2017 and December 31, 2016		—			—
Common stock, $0.001 par value per share; 115,000,000 shares authorized as of September 30, 2017 and December 31, 2016; 27,483,925 and 27,332,551 shares issued and outstanding as of September 30, 2017 and December 31, 2016, respectively		27			27
Preferred stock; $0.001 par value per share; 5,000,000 shares authorized as of September 30, 2022 and December 31, 2021; no shares issued and outstanding as of September 30, 2022 and December 31, 2021								—			—
Common stock, $0.001 par value per share; 115,000,000 shares authorized as of September 30, 2022 and December 31, 2021; 43,269,200 shares and 17,710,450 shares issued and outstanding as of September 30, 2022 and December 31, 2021, respectively								43			18
Additionalpaid-in capital		365,723			359,329			260,839			180,645
Accumulated deficit		(276,492	)		(237,549	)		(142,180	)		(116,250	)
Accumulated other comprehensive loss		(28	)		(80	)		(40	)		—

Total stockholders’ equity		89,230			121,727			118,662			64,413

Total liabilities and stockholders’ equity	$	96,264		$	131,621		$	133,015		$	78,667

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


	Three Months Ended September 30,						Nine Months Ended September 30,						Three Months Ended September 30,						Nine Months Ended September 30,
	2017			2016			2017			2016
Revenue	$	—		$	—		$	—		$	—
													2022			2021			2022			2021
Operating expenses:
Research and development		9,723			10,001			29,928			32,661		$	5,582		$	14,028		$	17,032		$	32,104
General and administrative		3,117			4,830			9,713			15,089			2,931			2,702			9,055			9,275

Total operating expenses		12,840			14,831			39,641			47,750			8,513			16,730			26,087			41,379

Loss from operations		(12,840	)		(14,831	)		(39,641	)		(47,750	)		(8,513	)		(16,730	)		(26,087	)		(41,379	)

Other income (expense):
Interest income		266			230			740			664
Interest expense		(31	)		(132	)		(157	)		(432	)
Foreign currency transaction gains (losses), net		20			58			115			79

Total other income (expense), net		255			156			698			311

Other income (expense), net														193			(75	)		157			(123	)
Net loss	$	(12,585	)	$	(14,675	)	$	(38,943	)	$	(47,439	)	$	(8,320	)	$	(16,805	)	$	(25,930	)	$	(41,502	)

Net loss per share, basic and diluted	$	(0.46	)	$	(0.54	)	$	(1.42	)	$	(1.74	)	$	(0.37	)	$	(0.92	)	$	(1.32	)	$	(2.48	)

Weighted average common shares outstanding, basic and diluted		27,483,550			27,322,907			27,414,314			27,286,323			22,228,228			18,287,924			19,649,558			16,768,458

Comprehensive loss:
Net loss	$	(12,585	)	$	(14,675	)	$	(38,943	)	$	(47,439	)	$	(8,320	)	$	(16,805	)	$	(25,930	)	$	(41,502	)
Other comprehensive loss:
Unrealized gain (loss) on marketable securities		13			(52	)		52			141

Unrealized gain (loss) on marketable debt securities														17			(1	)		(40	)		(1	)
Total other comprehensive income (loss)		13			(52	)		52			141			17			(1	)		(40	)		(1	)

Total comprehensive loss	$	(12,572	)	$	(14,727	)	$	(38,891	)	$	(47,298	)	$	(8,303	)	$	(16,806	)	$	(25,970	)	$	(41,503	)

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

1. ~~Nature~~

Description of ~~the~~ Business and Basis of Presentation

~~Zafgen,~~Larimar Therapeutics, Inc., together with its subsidiary (the “Company” or ~~the Company, was incorporated on November 22, 2005 under the laws of the State of Delaware. The Company~~“Larimar”), is a ~~biopharmaceutical~~clinical-stage biotechnology company ~~dedicated to significantly improving the health and well-being of patients affected by type 2 diabetes, rare diseases and other metabolic diseases. The Company is~~ focused on developing treatments for patients suffering from complex rare diseases using its novel therapeutics that treat the underlying biological mechanisms through the methionine aminopeptidase 2 (“MetAP2”) pathway. The Company has pioneered the study of MetAP2 inhibitors in both common and rare forms of obesity. The Company’scell penetrating peptide technology platform. Larimar's lead product candidate, CTI-1601, is~~ZGN-1061,~~ a ~~novel fumagillin-class MetAP2 inhibitor~~subcutaneously administered, ~~by subcutaneous injection,~~recombinant fusion protein intended to deliver human frataxin ("FXN"), an essential protein, to the mitochondria of patients with Friedreich’s ataxia. Friedreich’s ataxia is a rare, progressive and fatal disease in which ~~is currently being profiled for~~patients are unable to produce sufficient FXN due to a genetic abnormality.

In May 2021, Larimar reported positive topline data from its ~~utility~~Phase 1 Friedreich’s ataxia, ("FA") program after completing dosing of the single ascending dose ("SAD") trial in December 2020 and of the ~~treatment of type 2 diabetes~~multiple ascending dose ("MAD") trial in March 2021.

Also in May 2021, the United States Food and ~~other related metabolic disorders. Since its inception,~~Drug Administration ("FDA") placed a clinical hold on the Company's CTI-1601 clinical program after the Company ~~has devoted substantially all~~notified the agency of ~~its efforts~~mortalities at the highest dose levels of the 26-week non-human primate toxicology study that was designed to ~~research~~support extended dosing of patients with CTI-1601. At the time the hold was placed, Larimar had no interventional clinical trials with patients enrolling or enrolled.

In February 2022, in response to the Company's complete response to the clinical hold discussed above, the FDA stated that it was maintaining the clinical hold and ~~development, recruiting management, acquiring operating assets~~that additional data are needed to resolve the clinical hold. Larimar subsequently submitted a request for an FDA Type C meeting, which was granted and ~~raising capital. Currently,~~was held in July 2022. Following the Type C meeting, the Company ~~is focusing~~submitted a complete response to the FDA.

In September 2022, the FDA cleared the initiation of the 25 mg cohort of a Phase 2, four-week, placebo-controlled, dose exploration trial of CTI-1601 in Friedreich’s ataxia (FA) patients. In a written communication to Larimar, the FDA indicated it was lifting its ~~personnel~~full clinical hold on the CTI-1601 program and ~~financial resources onZGN-1061~~imposing a partial hold. With this decision, the FDA cleared CTI-1601 for a return to the clinic and the ~~discovery~~initiation of ~~novel and highly differentiated MetAP2 inhibitors.~~a 25 mg dose cohort of a phase 2 dose exploration study. The conduct of additional cohorts and/or other clinical trials of CTI-1601 in this study are subject to approval by the FDA. Larimar does not know when, or if, the partial clinical hold will be lifted.

The Company is subject to risks and uncertainties common to pre-commercial companies in the biotechnology industry, including, but not limited to, development by competitors of new technological innovations, dependence on key personnel, protection of proprietary technology, ~~dependence on key personnel,~~ compliance with ~~government~~governmental regulations, failure to secure regulatory approval for its drug candidates or any other product candidates and the ~~need~~ability to ~~obtain~~secure additional ~~financing. Product~~capital to fund its operations. Drug candidates currently under development will require ~~significant additional research and development efforts, including~~ extensive ~~preclinical~~non-clinical and clinical testing and regulatory approval prior to commercialization. These efforts require significant amounts of additional capital, adequate personnel, and infrastructure and extensive compliance-reporting capabilities.

The Company’s product candidates are all in the research and development stage. There can be no assurance that the Company’s research and development will be successfully completed, that adequate protection for the Company’s intellectual property will be obtained, that any product candidates developed will obtain necessary government regulatory approval or that any approved products will be commercially viable. Even if ~~the Company’s product~~our drug development efforts are successful, it is uncertain when, if ever, ~~the Company~~we will ~~generate~~realize significant revenue from product sales. ~~The Company operates~~

In late 2019, an outbreak of a novel strain of the Coronavirus 2019 Disease (COVID-19) was identified and infections have been found in ~~an environment~~a number of ~~rapid change~~countries around the world, including the United States. In 2020, the pandemic resulted in ~~technology~~a four-month temporary stoppage of the Company’s CTI-1601Phase 1 clinical trials in patients with Friedreich’s ataxia. Both of the Company's SAD and ~~substantial competition from pharmaceutical~~MAD clinical trials were completed in 2021. In the future, the COVID-19 pandemic and ~~biotechnology companies. In addition,~~responsive measures thereto may result in negative impacts on the Company, ~~is dependent upon~~including possible delays in our clinical and regulatory activities. We cannot be certain what the ~~services~~overall impact of ~~its employees and consultants.~~

~~The Company has incurred losses and negative cash flows from operations since its inception. As of September 30, 2017,~~ the Company had an accumulated deficit of $276.5 million. From its inception through September 30, 2017, the Company received net proceeds of $333.3 million from the sales of redeemable convertible preferred stock, the issuance of convertible promissory notes, the proceeds from its initial public offering (“IPO”) in June 2014 and its~~follow-on~~ offering in January 2015. Until such time, if ever, as the Company can generate substantial product revenue, the Company expects to finance its cash needs through a combination of equity offerings, debt financings, collaborations, strategic alliances, licensing arrangements and other sources of funding. If the Company is unable to raise additional funds through equity, debt financings or licensing arrangements when needed, the Company may be required to delay, limit, reduce or terminate product development or future commercialization efforts or grant rights to develop and market products or product candidates that the Company would otherwise prefer to develop and market itself. Based on its current operating plans, the Company believes its cash, cash equivalents and marketable securities of $93.2 million as of September 30, 2017COVID-19 pandemic will be ~~sufficient~~on our business and it has the potential to ~~fund its anticipated level~~materially adversely affect our business, financial condition, results of operations, and ~~capital expenditures for a period~~prospects.

Basis of ~~at least one year from the issuance date of this Quarterly Report.~~Presentation

The condensed consolidated financial statements include the accounts of ~~the Company~~Larimar and its wholly owned ~~subsidiaries Zafgen Securities Corporation, Zafgen Australia Pty Limited, and Zafgen Animal Health, LLC.~~subsidiary. All intercompany balances and transactions have been eliminated. The accompanying condensed consolidated financial statements have been prepared in conformity with GAAP.

Going Concern Assessment

The Company’s condensed consolidated financial statements have been presented on the basis that it will continue as a going concern, which contemplates the realization of assets and satisfaction of liabilities in the normal course of business.

Since its inception, the Company has incurred significant recurring operating losses and negative cash flows from operations. The Company has incurred net losses of $25.9 million and $41.5 million for the nine months ended September 30, 2022 and 2021, respectively. In addition, as of September 30, 2022, the Company had an accumulated deficit of $142.2 million. The Company expects to continue to generate operating losses for the foreseeable future. As of September 30, 2022, the Company had approximately $124.7 million of cash, cash equivalents and marketable debt securities available for use to fund its operations and capital requirements.

The Company has funded its operations to date primarily with proceeds from sales of common stock, proceeds from the sale of prefunded warrants for the purchase of common stock, the acquisition in 2020 of cash, cash equivalents, restricted cash, cash equivalents and marketable debt securities upon the merger with Zafgen, Inc. and prior to the 2020 merger with Zafgen, capital contributions from Chondrial Holdings, LLC.

In accordance with Accounting Standards Update (“ASU”) No. 2014-15, Disclosure of Uncertainties about an Entity’s Ability to Continue as a Going Concern, the Company has evaluated whether there are certain conditions and events, considered in the aggregate, that raise substantial doubt about the Company’s ability to continue as a going concern within one year after the date that the condensed consolidated financial statements are issued. As of the issuance date of these condensed consolidated financial statements, the Company expects its cash, cash equivalents and marketable debt securities will be sufficient to fund its forecasted operating expenses and capital expenditure requirements, for at least twelve months from the issuance of these financial statements. If the timing of our clinical assumptions were delayed or if there were other forecasted assumption changes that negatively impact our operating plan, the Company could reduce expenditures in order to further extend cash resources.

The Company has not yet commercialized any products and does not expect to generate revenue from the commercial sale of any products for several years, if at all. The Company expects that its research and development and general and administrative expenses will continue to increase and, as a result, that it will need additional capital to fund its future operating and capital requirements. Until the Company can generate substantial revenue, if ever, management continuously evaluates different strategies to obtain the required funding for future operations. These strategies include seeking additional funding through a combination of public or private equity offerings, debt financings, collaborations and licensing arrangements or other sources. The incurrence of indebtedness would result in increased fixed payment obligations and the Company may be required to agree to certain restrictive covenants, such as limitations on its ability to incur additional debt, limitations on its ability to acquire, sell or license intellectual property rights, minimum required cash balances and other operating restrictions that could adversely impact the Company's ability to conduct its business. Any additional fundraising efforts may divert the Company's management from their day-to-day activities, which may adversely affect its ability to develop and commercialize its product candidates.

There can be no assurance that the Company will be able to raise sufficient additional capital on acceptable terms, if at all. If such additional financing is not available on satisfactory terms, or is not available in sufficient amounts, or if the Company does not have sufficient authorized shares, the Company may be required to delay, limit, or eliminate the development of business opportunities and its ability to achieve its business objectives, its competitiveness, and its business, financial condition, and results of operations will be materially adversely affected. The Company could also be required to seek funds through arrangements with collaborative partners or otherwise at an earlier stage than otherwise would be desirable and it may be required to relinquish rights to some of its technologies or product candidates or otherwise agree to terms unfavorable to it, any of which may have a material adverse effect on the Company's business, operating results and prospects. In addition, geopolitical unrest, the impact of the COVID-19 pandemic and/or the potential impact of other health crises on the global financial markets may reduce the Company's ability to access capital, which could negatively affect its liquidity and ability to continue as a going concern.

If the Company is unable to obtain sufficient funding when needed and/or on acceptable terms, the Company may be required to significantly curtail, delay or discontinue one or more of its research and development programs, the manufacture of clinical and commercial supplies, product portfolio expansion or pre commercialization efforts, which could adversely affect its business prospects, or the Company may be unable to continue operations.

Summary of Significant Accounting Policies

Unaudited Interim Financial Information

The condensed consolidated balance sheet as of December 31, ~~2016~~2021 was derived from the Company’s audited financial statements but does not include all disclosures required by ~~accounting principles generally accepted in the United States of America, or~~ GAAP. The accompanying unaudited condensed consolidated financial statements as of September 30, ~~2017~~2022 and for the three and nine months ended September 30, ~~2017~~2022 and ~~2016,~~2021, have been prepared by the Company pursuant to the rules and regulations of the Securities and Exchange Commission ~~or SEC,~~(“SEC”) for interim financial statements. Certain information and footnote disclosures normally included in financial statements prepared in accordance with GAAP have been condensed or omitted pursuant to such rules and regulations. However, the Company believes that the disclosures are adequate to make the information presented not misleading. These condensed consolidated financial statements should be read in conjunction with the Company’s audited consolidated financial statements and the notes thereto for the year ended December 31, ~~2016,~~2021 included in the Company’s Annual Report onForm 10-K ~~for the year ended December 31, 2016, on file~~ filed with the ~~SEC.~~ SEC on March 25, 2022.

In the opinion of management, all adjustments, consisting only of normal

recurring adjustments necessary for a fair statement of the Company’s condensed consolidated financial position as of September 30, ~~2017 and~~2022, condensed consolidated results of operations ~~and cash flows~~ for the three and nine months ended September 30, ~~2017~~2022 and ~~2016~~condensed consolidated statement of cash flows for the nine months ended September 30, 2022 have been made. The results of operations for the three and nine months ended September 30, ~~2017~~2022 are not necessarily indicative of the results of operations that may be expected for the year ending December 31, ~~2017.~~2022.

~~2. Summary of Significant Accounting Policies~~

Use of Estimates

The preparation of condensed consolidated financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities ~~the~~and disclosure of contingent assets and liabilities ~~as of~~at the date of the ~~condensed consolidated~~ financial statements and the reported amounts of expenses during the reporting ~~periods.~~period. Significant estimates and assumptions reflected in these condensed consolidated financial statements include, but are not limited to, the accrual of research and development ~~expenses and~~expense, the recording as prepaid expense of payments made in advance of the actual provision of goods or services, valuation of stock-based ~~awards. Estimates are periodically reviewed~~awards and valuation of leases. Due to inherent uncertainty involved in ~~light of~~making estimates, actual results reported in future periods may be affected by changes in ~~circumstances, facts~~these estimates. On an ongoing basis, the Company evaluates its estimates and ~~experience. Actual results could differ from the Company’s estimates.~~assumptions.

Research and Development Costs

~~Marketable securities~~

~~Marketable securities consist~~Costs associated with internal research and development and external research and development services, including drug development, clinical studies and non-clinical studies, are expensed as incurred. Research and development expenses include costs for salaries, employee benefits, subcontractors, stock-based compensation, facility-related expenses, third-party license fees, laboratory supplies, and external costs of ~~investments with original maturities greater than 90 days.~~outside vendors engaged to conduct discovery, non-clinical and clinical development activities, and clinical trials as well as to manufacture clinical trial materials, depreciation and other costs. The Company ~~has classified its investments with maturities beyond one year as short term,~~recognizes external research and development costs based on ~~their highly liquid nature~~an evaluation of the progress to completion of specific tasks using information provided to the Company by its service providers.

Nonrefundable advance payments for goods or services to be received in the future for use in research and ~~because such marketable securities represent~~development activities are recorded as prepaid expenses. Such prepaid expenses are recognized as an expense when the ~~investment~~goods have been delivered or the related services have been performed, or when it is no longer expected that the goods will be delivered, or the services rendered.

Upfront payments, milestone payments and annual maintenance fees under license agreements are currently expensed in the period in which they are incurred.

Patent Costs

All patent-related costs incurred in connection with filing and prosecuting patent applications are expensed as incurred due to the uncertainty about the recovery of ~~cash that is available for current operations.~~ the expenditure. Amounts incurred are classified as general and administrative expenses.

Stock-Based Compensation

The Company ~~considers its investment portfolio of investments asavailable-for-sale.~~ ~~Accordingly, these investments are recorded at fair value, which is based on quoted market prices. Unrealized gains~~measures all stock-based awards granted to employees, directors and losses are reported as a component of accumulated other comprehensive income (loss) in stockholders’ equity. Realized gains and losses and declines in value judged to be other than temporary are included as a component of other income (expense), netnon-employee consultants based on the ~~specific identification method. When determining whether a decline in~~fair value on the date of grant using the Black-Scholes option-pricing model. Compensation expense of those awards is ~~other than temporary,~~recognized over the requisite service period, which is the vesting period of the respective award. Typically, the Company issues awards with only service-based vesting conditions and records the expense for these awards using the straight-line method. The Company accounts for forfeitures as they occur.

The Company classifies stock-based compensation expense in its condensed consolidated statements of operations and comprehensive loss in the same manner in which the award recipient’s payroll costs are classified or in which the award recipient’s service payments are classified.

Prior to May 28, 2020, the Company had been a private company and lacked company-specific historical and implied volatility information for its common stock. Therefore, the Company estimates its expected common stock price volatility based on the historical volatility of publicly traded peer companies and expects to continue to do so until it has adequate historical data regarding the volatility of its own traded stock price. The expected term of the Company’s stock options has been determined utilizing the “simplified” method for awards that qualify as “plain-vanilla” options. The risk-free interest rate is determined by reference to the U.S. Treasury yield curve in effect at the time of grant of the award for time periods approximately equal to the expected term of the award. Expected dividend yield considers ~~various factors, including whether~~the fact that the Company has never paid cash dividends on common stock and does not expect to pay any cash dividends in the intent to sell the security, and whether it is more likely than not that the Company will be required to sell the security prior to recovery of its amortized cost basis. Fair value is determined based on quoted market prices.foreseeable future.

Net ~~Income (Loss)~~Loss Per Share

Basic net ~~income (loss)~~loss per share is computed by dividing the net ~~income (loss)~~loss attributable to common stockholders by the weighted average number of common shares outstanding for the period. Basic shares outstanding includes the weighted average effect of the Company’s prefunded warrants issued in June 2020, the exercise of which requires little or no consideration for the delivery of shares of common stock. Basic and diluted weighted average shares of common stock outstanding for the three and nine months ended September 30, 2022 and 2021 includes the weighted average effect of 628,403 prefunded warrants for the purchase of shares of common stock, for which the remaining unfunded exercise price is $0.01 per share.

Diluted net ~~income (loss)~~loss per share attributable to common stockholders is computed by dividing the diluted net ~~income (loss)~~loss attributable to common stockholders by the weighted average number of common shares, including ~~potential~~potentially dilutive common shares assuming the dilutive effect of outstanding stock options and unvested restricted common shares, as determined using the treasury stock method. For periods in which the Company has reported net losses, diluted net loss per common share attributable to common stockholders is the same as basic net loss per common share attributable to common stockholders, since dilutive common shares are not assumed to have been issued if their effect is antidilutive.

The Company excluded ~~the following~~3,136,776 and 2,545,962 common stock equivalents outstanding as of September 30, ~~2017~~2022 and ~~2016,~~2021, respectively, from the computation of diluted net loss per share for the three and nine months ended September 30, ~~2017~~2022 and ~~2016~~2021 because they had an anti-dilutive impact due to the net loss incurred for the ~~periods:~~periods presented.

   As of September 30,
   2017    2016
Options to purchase common stock
   3,963,480    3,220,415
Unvested restricted common stock
   5,533    6,637




   3,969,013    3,227,052

Recently Issued and Adopted Accounting Pronouncements

~~In May 2014,~~From time to time, new accounting guidance is issued by the ~~Financial Accounting Standards Board (the “FASB”) issued ASUNo. 2014-09,~~~~Revenue from Contracts with Customers~~ ~~(Topic 606), which supersedes all existing revenue recognition requirements, including most industry-specific guidance. The new~~FASB or other standard ~~requires a company to recognize revenue when it transfers goods or services to customers in an amount~~setting bodies that reflects the consideration that the company expects to receive for those goods or services. The FASB has continued to issue accounting standards updates to clarify and provide implementation guidance related to Revenue from Contracts with Customers, including~~ASU 2016-08~~~~, Revenue from Contract with Customers: Principal versus Agent Considerations~~,~~ASU 2016-10,~~~~Revenue from Contracts with Customers: Identifying Performance Obligations and Licensing~~~~, andASU 2016-12,~~~~Revenue from Contracts with Customers: Narrow-Scope Improvements and Practical Expedients.~~~~These amendments address a number of areas, including the entity’s identification of its performance obligations in a contract, collectability,non-cash~~ ~~consideration, presentation of sales tax and an entity’s evaluation~~is adopted by us as of the ~~nature~~effective date or, in some cases where early adoption is permitted, in advance of ~~its promise to grant a license of intellectual property~~the effective date. We have assessed the recently issued guidance that is not yet effective and ~~whether or not that revenue is recognized over time or at a point in time. These new standards will be effective for~~believe the ~~Company beginning January 1, 2018. The Company early adopted the standard as of January 1, 2017, however there is no impact of this~~ new guidance ~~on its condensed consolidated financial statements as it does~~will not ~~currently~~ have ~~any revenue generating arrangements.~~

~~In February 2016, the FASB issued ASUNo. 2016-02,~~~~Leases~~. This guidance will require that lease arrangements longer than 12 months result in an entity recognizing an asset and liability equal to the present value of the lease payments in the statement of financial position. This guidance is effective for annual periods beginning after December 15, 2018, and interim periods therein. This standard requires a modified retrospective transition approach for all leases existing at, or entered into after, the date of initial application, with an option to use certain transition relief. Early adoption is permitted. The Company is evaluating the effect that this guidance will have on its condensed consolidated financial statements.

~~In March 2016, the FASB issued ASUNo. 2016-09,~~~~Improvements to Employee Share-Based Payment Accounting~~. This standard was adopted on January 1, 2017 on a modified retrospective basis. As a result the Company has made an accounting policy election to account for forfeitures as they occur. The adoption of this guidance also requires excess tax benefits and tax deficiencies be recorded in the income statement as opposed to additional~~paid-in~~ ~~capital when the awards vest or are settled. The adoption of this guidance had an immaterial~~material impact on the ~~Company’s~~ condensed consolidated results of operations, cash flows or financial ~~statements as of and for the three and nine months ended September 30, 2017.~~position.

~~In August 2016, the FASB issued ASUNo. 2016-15,~~~~Statement of Cash Flows: Classification of Certain Cash Receipts and Cash Payments~~. This guidance addresses the presentation and classification of certain cash receipts and cash payments in the statement of cash flows. The standard will be effective for annual periods beginning after December 15, 2017, and interim periods therein. Early adoption is permitted. The Company is evaluating the effect that this guidance will have on its condensed consolidated financial statements.11

~~In May 2017, the FASB issued ASUNo. 2017-09,~~~~Compensation – Stock Compensation: Scope of Modification Accounting~~. This guidance addresses which changes to the terms or conditions of a share-based payment award require an entity to apply modification accounting. The standard will be effective for annual periods beginning after December 15, 2017, and interim periods therein. Early adoption is permitted. The Company is evaluating the effect that this guidance will have on its condensed consolidated financial statements.

Fair Value Measurements and Marketable ~~Securities~~

debt securities

Fair Value Measurements

The ~~following tables present information about the~~ Company’s ~~financial~~ assets and liabilities that ~~have been~~are measured at fair value on a recurring basis as of September 30, ~~2017~~2022 and December 31, ~~2016,~~2021 are measured in accordance with the standards of ASC 820, Fair Value Measurements and ~~indicate the~~Disclosures, which establishes a three-level valuation hierarchy for measuring fair value and expands financial statement disclosures about fair value measurements. The valuation hierarchy is based on the transparency of ~~the hierarchy of~~inputs to the valuation inputs utilized to determine such fair value. In general, fair values determined by Level 1 inputs utilize quoted prices (unadjusted) in active markets for identical assets or liabilities. Fair value determined by Level 2 inputs utilize observable inputs other than Level 1 prices, such as quoted prices, for similar assets or liabilities, quoted market prices in markets that are not active or other inputs that are observable or can be corroborated by observable market data for substantially the full term of ~~the related assets or liabilities. Fair values determined by Level 3 inputs are unobservable data points for the~~an asset or liability as of the measurement date. The three levels are defined as follows:


Level – 1	Inputs to the valuation methodology are quoted prices (unadjusted) for identical assets or liabilities in active markets.

Level – 2	Inputs to the valuation methodology include quoted prices for similar assets and liabilities in active markets, and inputs that are observable for the asset or liability, either directly or indirectly, for substantially the full term of the financial instrument.

Level – 3	Inputs to the valuation methodology are unobservable and significant to the fair value measurement.

The Company’s financial instruments consist primarily of cash, cash equivalents, marketable debt securities, accounts payable and ~~include situations where there is little, if any, market activity for~~accrued liabilities. For accounts payable and accrued liabilities, the ~~asset or liability. During the nine months ended~~carrying amounts of these financial instruments as of September 30, ~~2017, there~~2022 and December 31, 2021 were ~~no transfers between Level 1 and Level 2 financial assets.~~considered representative of their fair values due to their short term to maturity.

The following tables summarize the Company’s cash equivalents and marketable debt securities as of September 30, ~~2017~~2022 and cash equivalents as of December 31, ~~2016:~~2021:


	September 30, 2017								Total		Quoted Prices in Active Markets (Level 1)		Significant Other Observable Inputs (Level 2)		Significant Unobservable Inputs (Level 3)
			Quoted		Significant				(in thousands)
September 30, 2022
Cash equivalents:
Money market funds									$	84,034		84,034	$	—	$	—
Total cash equivalents										84,034		84,034		—		—
			Prices in		Other		Significant
Marketable debt securities:
U.S. Government and agency securities										38,652		—		38,652		—
Total marketable debt securities										38,652		—		38,652		—
Total cash equivalents and marketable debt securities									$	122,686	$	84,034	$	38,652	$	—
			Active		Observable		Unobservable
			Markets		Inputs		Inputs
	Total		(Level 1)		(Level 2)		(Level 3)
	(in thousands)
December 31, 2021
Cash equivalents:
Money market funds	$	13,823	$	13,823	$	—	$	—	$	6,137	$	6,137	$	—	$	—
Commercial paper		3,499		—		3,499		—		7,549		7,549		—		—

Corporate bonds										1,219		1,219		—		—
Total cash equivalents		17,322		13,823		3,499		—		14,905		14,905		—		—


Marketable securities:
Corporate bonds		58,747		—		58,747		—
Commercial paper		14,760		—		14,760		—
Certificates of deposit		480		—		480		—

Total marketable securities		73,987		—		73,987		—

Total cash equivalents and marketable securities	$	91,309	$	13,823	$	77,486	$	—

   December 31, 2016
   Total    Quoted
Prices in
Active
Markets
(Level 1)    Significant
Other
Observable
Inputs
(Level 2)    Significant
Unobservable
Inputs
(Level 3)
   (in thousands)
Cash equivalents:

Money market funds
   $ 22,091    $ 22,091    $ —      $ —
Commercial paper
   2,997    —      2,997    —
Corporate bonds
   1,500    —      1,500    —








Total cash equivalents
   26,588    22,091    4,497    —








Marketable securities:

Corporate bonds
   69,622    —      69,622    —
Commercial paper
   27,220    —      27,220    —








Total marketable securities
   96,842    —      96,842    —








Total cash equivalents and marketable securities
   $ 123,430    $ 22,091    $ 101,339    $ —

The ~~carrying amounts reflected~~accrued interest receivable related to the Company’s investments was $0.2 million as of September 30, 2022 and is included in prepaid expenses and other current assets on the condensed consolidated balance ~~sheets for tax incentive~~sheet. There was no accrued interest receivable ~~accounts payable,~~ and ~~accrued expenses approximate~~December 31, 2021.

The Company classifies its money market funds which are valued based on quoted market prices in active markets with no valuation adjustment, as Level 1 assets within the fair value ~~due~~hierarchy.

The Company classifies its investments in U.S. treasury securities corporate commercial paper, and corporate debt securities as Level 2 assets within the fair value hierarchy. The fair values of these investments are estimated by taking into consideration valuations obtained from third-party pricing services. The pricing services utilize 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, issuer credit spreads, benchmark securities, prepayment/default projections based on historical data and other observable inputs.

As of September 30, 2022 and December 31, 2021, the unrealized losses for available-for-sale investments were non-credit related, and the Company does not intend to sell the investments that were in an unrealized loss position, nor will it be required to sell those investments before recovery of their ~~short-term maturities. The carrying value~~amortized cost basis, which may be maturity. As of September 30, 2022 and December 31, 2021, no allowances for credit losses for the Company’s ~~outstanding notes payable approximates fair value (a Level 2 fair value measurement), reflecting interest rates currently available~~investments were recorded. During the three and nine months ended September 30, 2022 and 2021, the Company did not recognize any impairment losses related to ~~the Company.~~

investments.

Marketable ~~Securities~~debt securities

The following ~~tables summarize~~table summarizes the ~~Company’s~~Company's marketable debt securities as of September 30, ~~2017 and~~2022. The Company held no marketable debt securities as of December 31, ~~2016:~~2021.

   September 30, 2017
      Gross    Gross    Fair
Value
   Amortized    Unrealized    Unrealized
   Cost    Gains    Losses
   (in thousands)
Assets:

Corporate bonds (due within 1 year)
   $ 58,772    $ —      $ (25 )    $ 58,747
Commercial paper (due within 1 year)
   14,763       (3 )    14,760
Certificates of deposit (due within 1 year)
   480    —      —      480








   $ 74,015    $ —      $ (28 )    $ 73,987


	Amortized Cost		Gross Unrealized Gains		Gross Unrealized Losses			Fair Value
	(in thousands)
September 30, 2022
Assets:
U.S. Government and agency securities		38,692		2		(42	)		38,652
	$	38,692	$	2	$	(42	)	$	38,652

Fixed Assets

~~5. Notes Payable~~

The Company has outstanding amounts due under a loan and security agreement with Oxford Finance LLC and Midcap Financial, or the 2014 Credit Facility, entered into in March 2014. All promissory notes issued under the 2014 Credit Facility are collateralized by substantially allFixed assets, net consisted of the ~~Company’s personal property, other than its intellectual property. There are no financial covenants associated with~~following:


		September 30,			December 31,
	Useful Life	2022			2021
		(in thousands)
Computer equipment	5 years	$	66		$	66
Lab equipment	5 years		1,192			1,092
Furniture and fixtures	7 years		456			456
Leasehold improvements	lease term		31			31
			1,745			1,645
Less: Accumulated depreciation			(836	)		(596	)
		$	909		$	1,049

Depreciation expense during the 2014 Credit Facility; however, there are negative covenants restricting the Company’s activities, including limitations on dispositions, mergers or acquisitions; encumbering or granting a security interest in its intellectual property; incurring indebtedness or liens; paying dividends; making certain investments;three and ~~certain other business transactions.~~

~~During the three~~nine months ended September 30, ~~2017~~2022 was $0.1 million and ~~2016,~~$0.2 million, respectively. Depreciation expense during the three and nine months ended September 30, 2021 was also $0.1 million and $0.2 million, respectively.

Stockholders’ Equity and Stock Options

Common Stock and Prefunded Warrants

As of September 30, 2022, the Company’s Certificate of Incorporation, as amended and restated, authorized the Company ~~recognized less than $0.1~~to issue up to 115,000,000 shares of $0.001 par value common stock, of which 43,269,200 shares were issued and outstanding, and up to 5,000,000 shares of $0.001 par value undesignated preferred stock, of which no shares were issued or outstanding. The voting, dividend, and liquidation rights of the holders of the Company’s common stock are subject to and qualified by the rights, powers, and preferences of the holders of the preferred stock. Each share of common stock entitles the holder to one vote on all matters submitted to a vote of the Company’s stockholders. Common stockholders are entitled to receive dividends, as may be declared by the board of directors of the Company (the “Board”), if any. No cash dividends have been declared or paid to date.

On May 28, 2020, the Company entered into a securities purchase agreement with certain accredited investors (the “Purchasers”) for the sale by the Company in a private placement of 6,105,359 shares of the Company’s common stock and prefunded warrants to purchase an aggregate of 628,403 shares of the Company’s common stock, for a price of $11.88 per share of the common stock and $11.87 per prefunded warrant. The prefunded warrants are exercisable at an exercise price of $0.01 and are exercisable indefinitely. The Purchasers may exercise the prefunded warrants on a cashless basis in the event that there is no effective registration statement covering the resale of the shares of common stock underlying the prefunded warrants on the date in which the Company is required to deliver the shares. The private placement closed on June 1, 2020. The aggregate gross proceeds for the issuance and sale of the common stock and prefunded warrants were $80.0 million; transaction costs totaled $4.6 million and ~~$0.1 million, respectively,~~resulted in net proceeds of ~~interest expense related~~$75.4 million. The Company’s Registration Statement on Form S-3, filed with the SEC on June 26, 2020, registered the resale of 6,105,359 shares of common stock sold and the 628,403 shares of common stock underlying the prefunded warrants. MTS Health Partners served as placement agent to the ~~2014 Credit Facility.~~Company in connection with the private placement. As partial compensation for these services, the Company issued MTS Health Partners 35,260 shares of common stock.

In July 2021, the Company sold 2,342,720 shares under an “at-the-market” equity distribution agreement (the "Prior ATM Agreement") with Piper Sandler & Co. for net proceeds of $19.9 million. The Prior ATM Agreement was terminated in November 2022 in connection with the establishment of a new "at-the-market' offering program (see 2022 ATM Agreement below). An aggregate of 2,354,244 shares of common stock were sold pursuant to the Prior ATM Agreement for net proceeds of $20.1 million

In September 2022, the Company sold 25,558,750 shares of common stock at a public offering price of $3.15 per share and received net proceeds, net of underwriting discounts and commissions and offering costs of $75.2 million.

2022 ATM Agreement

On November 10, 2022, the Company entered into a sales agreement (the "ATM Agreement") with an investment bank (Guggenheim Securities, LLC) in connection with the establishment of an “at-the-market” offering program under which the Company may sell up to an aggregate of $50.0 million of shares of common stock (the “ATM Shares”) from time to time.

Under the ATM Agreement, the Company sets the parameters for the sale of ATM Shares, including the number of ATM Shares to be issued, the time period during which sales are requested to be made, limitations on the number of ATM Shares that may be sold in any one trading day and any minimum price below which sales may not be made. Sales of the ATM Shares, if any, under the ATM Agreement may be made in transactions that are deemed to be “at-the-market offerings” as defined in Rule 415 under the Securities Act. The Company pays its investment

bank a commission equal to 3.0% of the gross proceeds of any ATM Shares sold through its investment bank under the ATM Agreement and reimburses the investment bank for certain specified expenses. The ATM Agreement contains customary representations, warranties and agreements by the Company, indemnification obligations of the Company and its investment bank, other customary obligations of the parties and termination provisions. The Company has no obligation to sell any of the ATM Shares and may at any time suspend offers under the ATM Agreement.

The ATM Shares will be offered and sold pursuant to the Company’s Registration Statement on Form S-3, filed by the Company on November 10, 2022 (the “Registration Statement”), and the sales agreement prospectus that forms a part of such Registration Statement, following such time, if ever, as the Registration Statement is declared effective by the SEC. As of the date of this Quarterly Report on Form 10-Q, no ATM Shares have been sold pursuant to the ATM Agreement.

2020 Equity Incentive Plan

The Board adopted the 2020 Equity Incentive Plan (the "2020 Plan") on July 16, 2020 and the stockholders of the Company approved the 2020 Plan on September 29, 2020. The 2020 Plan replaces the predecessor plans (the "Prior Plans") that the Company assumed following its merger with Zafgen in May 2020. Options outstanding under the Prior Plans will remain outstanding, unchanged, and subject to the terms of the Prior Plans and the respective award agreements, and no further awards will be made under the Prior Plans. However, if any award previously granted under the Prior Plans, expires, terminates, is canceled, or is forfeited for any reason after the approval of the 2020 Plan, the shares subject to that award will be added to the 2020 Plan share pool so that they can be utilized for new grants under the 2020 Plan.

The 2020 Plan provides for the grant of incentive stock options (“ISOs”), nonstatutory stock options (“NSOs”), stock appreciation rights, restricted stock awards, restricted stock unit awards, and cash or other stock-based awards. ISOs may be granted only to the Company’s employees, including the Company’s officers, and the employees of the Company’s affiliates. All other awards may be granted to the Company’s employees, including the Company’s officers, the Company’s non-employee directors and consultants, and the employees and consultants of the Company’s affiliates.

The maximum number of shares that may be issued in respect of any awards under the 2020 Plan is the sum of: (i) 1,700,000 shares plus (ii) an annual increase on January 1, 2021 and each anniversary of such date thereafter through January 1, 2030, equal to the lesser of (A) 4% of the shares issued and outstanding on the last day of the immediately preceding fiscal year, or (B) such smaller number of shares as determined by the Board (collectively, the “Plan Limit”). The maximum aggregate number of shares that may be issued under the 2020 Plan is 8,000,000 over the ten-year term of the 2020 Plan. During the nine months ended September 30, ~~2017~~2022 and ~~2016, the Company recognized $0.2 million~~twelve months ended December 31, 2021, respectively, options to purchase 130,516 and ~~$0.4 million, respectively, of interest expense related to the 2014 Credit Facility. The effective annual interest rate of the outstanding debt~~56,966 shares issued under the ~~2014 Credit Facility is approximately 10.8%.~~

~~6. Stock-Based Awards~~

~~The Company grants equity awards under its 2014 Stock Option~~Prior Plans were cancelled and Incentive Plan, or the 2014 Stock Option Plan, and is authorized to issue common stock under its 2014 Employee Stock Purchase Plan. The Company also has outstanding stock-based awards under its Amended and Restated 2006 Stock Option Plan but is no longer granting awards under this plan. As of September 30, 2017, 2,236,781 shares arebecame available for grant under the ~~2014 Stock Option~~2020 Plan.

As permitted by the 2020 Plan, ~~including 1,093,302~~the Company added 614,709 and 708,418 shares ~~automatically added~~available for grant to the ~~2014 Stock Option~~2020 Plan on January 1, ~~2017~~2021 and January 1, 2022, respectively. As of September 30, 2022, 991,687 shares of common stock were available for grant under the 2020 Plan.

Stock Valuation

The following table presents, on a weighted average basis, the assumptions used in the Black-Scholes option-pricing model to determine the grant-date fair value of stock options granted to employees:


	September 30,	December 31,
	2022	2021
Risk-free interest rate	2.13%	0.89%
Expected term (in years)	6.21	6.19
Expected volatility	89%	91%
Dividend yield	0.00%	0.00%

Stock Options

The following table summarizes the Company’s stock option activity for the nine months ended September 30, 2022 (amounts in millions, except for share and per share data):



				Weighted		Weighted Average		Aggregate
				Average		Remaining		Intrinsic
	Number of			Exercise		Contractual		Value (a)
	Shares			Price		Term (in years)		(in millions)
Outstanding as of December 31, 2021		2,523,305		$	18.88		7.6
Granted		868,450			6.99
Forfeited/Expired		(254,979	)		59.60
Outstanding as of September 30, 2022		3,136,776		$	12.27		8.1	$	0.1
Exercisable as of September 30, 2022		1,329,897		$	14.81		7.2	$	—
Vested and expected to vest as of September 30, 2022		3,136,776		$	12.27		8.1	$	0.1

(a)

The aggregate intrinsic value is calculated as the difference between the exercise price of the underlying options and the fair value of the common stock for the options that were "in the money" at September 30, 2022.

2022 Option Grants

During the nine months ended September 30, 2022, the Company granted options to purchase 868,450 shares of common stock to employees and directors of the Company under the 2020 Plan. The options have an exercise price equal to the closing stock price as of the grant date. Of the 868,450 options granted in 2022, 826,950 were granted to employees and vest over four years with 25% vesting on the first anniversary of the grant and the remainder vesting in equal monthly installments thereafter. The remaining 41,500 options were annual grants to the Company's directors and vest one year from the grant date. The weighted-average grant date fair value of options granted during the nine months ended September 30, 2022 was $5.24.

Stock-Based Compensation

Stock-based compensation expense was classified in the condensed consolidated statements of operations as follows:


	Three Months Ended September 30,				Nine Months Ended September 30,
	2022		2021		2022		2021

Research and development	$	702	$	567	$	2,050	$	1,520
General and administrative		965		896		2,926		2,473
	$	1,667	$	1,463	$	4,976	$	3,993

As of September 30, 2022, total unrecognized compensation expense related to unvested stock options was $14.0 million, which is expected to be recognized over a weighted average period of 2.36 years.

Commitments and Contingencies

Intellectual Property Licenses

The Company is party to an exclusive License Agreement (the “WFUHS License”), dated November 30, 2016, as amended, with Wake Forest University Health Sciences (“WFUHS”) and an exclusive License Agreement (the “IU License”), dated November 30, 2016, as amended, with Indiana University (“IU”). Such agreements provide for a transferable, worldwide license to certain patent rights regarding technology used by the Company with respect to the development of CTI-1601.

In partial consideration for the right and license granted under these agreements, the Company will pay each of WFUHS and IU a royalty of a low single digit percentage of net sales of licensed products depending on whether there is a valid patent covering such products. As additional consideration for these agreements, the Company is obligated to pay each of WFUHS and IU certain milestone payments of up to $2.6 million in the aggregate upon the achievement of certain developmental milestones, commencing on the enrollment of the first patient in a Phase 1 clinical trial. The Company enrolled the first patient in its SAD trial on December 11, 2019 and paid WFUHS and IU less than $0.1 million. The Company will also pay each of WFUHS and IU sublicensing fees ranging from a high-single digit to a low double-digit percentage of sublicense consideration depending on the Company’s achievement of certain regulatory milestones as of the time of receipt of the sublicense consideration. The Company is also obligated to reimburse WFUHS and IU for patent-related expenses. In the event that the Company disputes the validity of any of the licensed patents, the royalty rate would be tripled during such dispute. The Company is also obligated to pay to IU a minimum annual royalty of less than $0.1 million per annum starting in the 2020 calendar year for the term of the agreement.

In the event that the Company is required to pay IU consideration, then the Company may deduct 20% of such IU consideration on a dollar-for-dollar basis from the consideration due to WFUHS. In the event that the Company is required to pay WFUHS consideration, then the Company may deduct 60% of such WFUHS consideration on a dollar-for-dollar basis from the consideration due to IU.

Both agreements continue from their effective date through the last to expire of the licensed patents unless earlier terminated by either party in accordance with their terms.

Leases

On May 28, 2020, the Company acquired a non-cancellable operating lease for approximately 17,705 square feet of office space (the “Premises”). The lease expires on October 30, 2029.As part of the agreement, the Company is required to maintain a letter of credit, which upon signing was $1.3 million and is classified as restricted cash within the condensed consolidated financial statements. In addition to the base rent, the Company is also responsible for its share of operating expenses, electricity and real estate taxes, which costs are not included in the determination of the leases’ right-of-use assets or lease liabilities. The right-of-use asset is being amortized to other income/(expense) over the remaining lease term as a result of ~~a provision in~~ the ~~2014 Stock Option Plan.~~sublease described below.

The Company recorded stock-based compensation expense related to stock options and restricted common stock in the following expense categories within its condensed consolidated statements of operations:

   Three Months
Ended
September 30,    Nine Months Ended
September 30,
   2017    2016    2017    2016
   (in thousands)    (in thousands)
Research and development
   $ 896    $ 878    $ 2,629    $ 2,708
General and administrative
   $ 1,200    1,438    3,570    5,218








   $ 2,096    $ 2,316    $ 6,199    $ 7,926

~~7. Commitments and Contingencies~~

~~Leases~~

The Company has a lease for office space in Boston, Massachusetts, effective as of July 28, 2014, with an initial term expiring July 31, 2017 and an option to extend the lease for three additional years. In March 2015,On October 27, 2020, the Company entered into ~~an operating lease~~a sublease agreement (the “Sublease”) with Massachusetts Municipal Association, Inc. (the “Subtenant”), whereby the Company sublet the entire Premises to the Subtenant. The initial term of the Sublease commenced on December 4, 2020 and continues until October 30, 2029. In connection with the Sublease, the Company evaluated the need for ~~additional office space in Boston, Massachusetts, effective as of April 15, 2015, with~~impairment under ASC 360 and determined there was no impairment.

The Sublease provided for an initial annual base rent of $0.8 million, which increases annually up to a maximum annual base rent of $1.0 million. The Subtenant also is responsible for paying to the Company future increases in operating costs (commencing on January 1, 2022), future increases in annual tax costs (commencing July 1, 2021) and all utility costs (commencing March 1, 2021) attributable to the Premises during the term ~~expiring~~of the Sublease. As part of the Sublease, the subtenant deposited a letter of credit in the amount of $0.8 million to assure their performance under the sublease. If there are no uncured events of default under the sublease, the amount of this security deposit decreases over time to $0.4 million on ~~July 31, 2017, and two options to extend~~the sixth anniversary of the Sublease. The Company records sublease income on this ~~lease for three additional years each. In October 2015,~~sublease on a straight-line basis as a component of other income/(expense).

On November 5, 2018, the Company entered into an operating lease for office and lab space in ~~San Diego, California,~~Philadelphia, Pennsylvania, effective as of ~~October~~January 1, ~~2015, with a term~~2019, and expiring on ~~September 30, 2019, and~~December 31, 2020 with an option to extend ~~this~~the lease for ~~five~~two additional years. ~~In January 2017,~~On August 4, 2020, the Company ~~extended~~executed the ~~leases for both office spaces in Boston, Massachusetts with new terms expiring on July 31, 2020. The Company also has the~~first option to extend ~~both of these leases in Boston, Massachusetts~~the lease for an additional ~~three years. With the landlord’s consent,~~year, expiring on December 31, 2021. On August 9, 2021, the Company ~~has subleased 2,976 square feet of office space in Boston, Massachusetts~~executed the remaining option to extend the lease for an ~~unrelated third party which began~~additional year, expiring on ~~January 1, 2017 and expires on September 30, 2018.~~December 31, 2022. The Company ~~expects~~has determined this lease extension qualifies as a short-term lease and have applied the accounting policy election to ~~receive approximately $0.2~~not record the related right-of-use asset and lease liabilities.

Expense arising from operating leases was $0.1 million ~~in sublease rental income~~ and $0.2 millionduring the ~~sublease period, which is recorded as an offset to rental expense in the condensed consolidated statements of operations.~~

~~Future minimum lease payments for its operating leases as of September 30, 2017 were as follows:~~

Year Ending December 31,

(in thousands)
2017 (October to December)
   $ 118
2018
   479
2019
   464
2020
   226


   $ 1,287

~~During the~~ three ~~months ended September 30, 2017~~ and ~~2016, the Company recognized $0.1 million of rental expense related to office space. During the~~ nine months ended September 30, ~~2017~~2022 and ~~2016, the Company recognized $0.3~~also $0.1 million ~~of rental expense related to office space.~~

~~Intellectual Property Licenses~~

~~The Company has acquired exclusive rights to develop patented compounds~~ and ~~relatedknow-how~~ for beloranib under two licensing agreements with two third parties in the course of its research and development activities. The licensing rights obligate the Company to make payments to the licensors for license fees, milestones, license maintenance fees and royalties. The Company is also responsible for patent prosecution costs.

~~As of September 30, 2017, the Company is obligated to make additional milestone payments of up to $12.3~~$0.2 million ~~upon reaching certainpre-commercialization~~ milestones, such as clinical trials and government approvals (including the U.S. Food and Drug Administration, or FDA, approval of a New Drug Application, or NDA), and up to $12.5 million upon reaching certain product commercialization milestones related to the development of beloranib. Under one of the license agreements, the Company is also obligated to pay up to $1.3 million with respect to each subsequent licensed product, if any, that is a new chemical entity. In addition, the Company will owe single-digit royalties on sales of commercial products developed using these licensed technologies, if any.

~~There were no milestones achieved~~ during the three orand nine months ended September 30, ~~2017 or 2016.~~2021, respectively. For operating leases, the weighted-average remaining lease term for leases at September 30, 2022 and December 31, 2021 was 7.0 and 7.6 years, respectively. For operating leases, the weighted average discount rate for leases at September 30, 2022 and December 31, 2021 was 11.0%. The Company is also obligated to pay to the licensors a percentage of fees received if and when the Company sublicenses the technology. As of September 30, 2017, the Company has not yet developed a commercial product using the licensed technologies and it has not entered into any ~~sublicense agreements for the technologies.~~financing leases.

~~Indemnification Agreements~~

~~In the ordinary course~~Maturities of business, the Company may provide indemnification of varying scope and terms to vendors, lessors, business partners, and other parties with respect to certain matters including, but not limited to, losses arising out of breach of such agreements or from intellectual property infringement claims made by third parties. In addition, the Company has entered into indemnification agreements with members of the management team and the board of directors of the Company that will require the Company, among other things, to indemnify them against certainlease liabilities ~~that may arise by reason of their status or service as directors or officers. The maximum potential amount of future payments the Company could be required to make~~due under these ~~indemnification~~lease agreements is, in many cases, unlimited. To date, the Company has not incurred any material costs as a result of such indemnifications. The Company does not believe that the outcome of any claims under indemnification arrangements will have a material effect on its financial position, results of operations or cash flows, and it has not accrued any liabilities related to such obligations in its condensed consolidated financial statements as of September 30, ~~2017.~~

2022 are as follows:


	Operating
(in thousands)	Leases
Three months ending December 31, 2022	$	302
Year ended December 31, 2023		1,146
Year ended December 31, 2024		1,065
Year ended December 31, 2025		1,083
Year ended December 31, 2026		1,101
Thereafter		3,213
Total lease payments		7,910
Less: imputed interest		(2,345	)
Present value of lease liabilities	$	5,565

Legal Proceedings

The Company accrues a liability for legal contingencies when it believes that it is both probable that a liability has been incurred and that the Company can reasonably estimate the amount of the loss. The Company reviews these accruals and adjusts them to reflect ongoing negotiations, settlements, rulings, advice of legal counsel and other relevant information. To the extent new information is obtained and the views on the probable outcomes of claims, suits, assessments, investigations or legal proceedings change, changes in the Company’s accrued liabilities would be recorded in the period in which such determination is made. In addition, in accordance with the relevant authoritative guidance, for any matters in which the likelihood of material loss is at least reasonably possible, the Company will provide disclosure of the possible loss or range of loss. If a reasonable estimate cannot be made, however, the Company will provide disclosure to that effect. The Company expenses legal costs as they are incurred.

On October 21, 2015, a purported stockholder of the Company filed a putative class action lawsuit in the U.S. District Court for the District of Massachusetts, against the Company and Thomas E. Hughes, captioned Aviad Bessler v. Zafgen, Inc. and Thomas E. Hughes,~~No. 1:15-cv-13618.~~ ~~An amended complaint was filed on February 22, 2016. The amended complaint alleges violations of Sections 10(b) and 20(a) of the Securities Exchange Act of 1934 and SEC Rule10b-5~~ based on allegedly false and misleading statements and omissions regarding the Company’s clinical trials for its drug beloranib. On August 9, 2016, the District Court granted the motion to dismiss and dismissed the amended complaint with prejudice. On August 12, 2016, plaintiffs filed a notice of appeal to the First Circuit Court of Appeals and on April 7, 2017, the dismissal with prejudice was affirmed.

The Company may periodically become subject to other legal proceedings and claims arising in connection with ongoing business activities, including claims or disputes related to patents that have been issued or that are pending in the field of research on which the Company is focused. The Company is not currently a party to any litigation, nor is management aware of any ~~material claims~~pending or threatened litigation against the Company, that it believes would materially affect the Company's business, operating results, financial condition or cash flows.

Item 2. Management’s Discussion and Analysis of ~~December 2016. The following table summarizes the restructuring reserve for the periods indicated:~~

   Nine Months
Ended
September 30,
2017    Year Ended
December 31,
2016
   (in thousands)
Restructuring reserve beginning balance
   $ 376    $ —
Restructuring expenses incurred during the period
   24    1,223
Amounts paid during the period
   (400 )    (847 )




Restructuring reserve ending balance
   $ —      $ 376

In October 2017, the Company granted its newly appointed Chief Executive Officer two inducement stock option grants, which were not granted under the Company’s 2014 Stock Option and Incentive Plan: (i) an option to purchase 550,000 shares of the Company’s common stock with standard vesting terms, and (ii) an additional option to purchase 1,100,000 shares of the Company’s common stock, with such option shares vesting in accordance with certain stock price appreciation and other financial performance goals.

Additionally, in October 2017, the Company granted its President and Chief Scientific Officer an option to purchase 275,000 shares of the Company’s common stock under the Company’s 2014 Stock Option and Incentive Plan. Such option will vest in accordance with certain stock price appreciation performance goals.

~~Item 2.~~

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

The following discussion and analysis of our financial condition and results of operations should be read in conjunction with our condensed consolidated financial statements and related notes appearing elsewhere in this Quarterly Report onForm 10-Q or (“Quarterly ~~Report,~~Report”), and the audited consolidated financial statements and notes thereto and management’s discussion and analysis of financial condition and results of operations for the year ended December 31, ~~2016~~2021 included in our Annual Report onForm 10-K ~~for~~ filed with the ~~year ended December 31, 2016.~~Securities and Exchange Commission (“SEC”) on March 25, 2022 ("2021 Annual Report"). Some of the information contained in this discussion and analysis or set forth elsewhere in this Quarterly Report, including information with respect to our plans and strategy for our business, includes forward-looking statements that involve risks, uncertainties, and ~~uncertainties. As~~assumptions. These statements are based on our beliefs and expectations about future outcomes and are subject to risks and uncertainties that could cause our actual results to differ materially from anticipated results. We undertake no obligation to publicly update these forward-looking statements, whether as a result of ~~many factors, including those factors set forth~~new information, future events or otherwise. You should read the “Risk Factors” section included in our 2021 Annual Report and our subsequently filed Quarterly Reports on Form 10-Q in addition to the ~~‘‘Risk Factors’’ section~~"Risk Factors" and “Cautionary Note Regarding Forward-Looking Statements” sections of this Quarterly Report ~~our~~for a discussion of important factors that could cause actual results ~~could~~to differ materially from the results described in or implied by ~~these forward-looking statements.~~

Our actual results and timing of certain events may differ materially from the results discussed, projected, anticipated, or indicated in any forward-looking statements. We caution you that forward-looking statements are not guarantees of future performance and that our actual results of operations, financial condition and liquidity, and the development of the industry in which we operate may differ materially from the forward-looking statements contained in ~~this Quarterly Report. In addition, even if our results of operations, financial condition~~the following discussion and liquidity, and the development of the industry in which we operate are consistent with the forward-looking statements contained in this Quarterly Report, they may not be predictive of results or developments in future periods.analysis.

The following information and any forward-looking statements should be considered in light of factors discussed elsewhere in this Quarterly Report, including those risks identified under the Risk Factors section.Overview

We caution readers not to place undue reliance on any forward-looking statements made by us, which speak only as of the date they are made. We disclaim any obligation, except as specifically required by law and the rules of the SEC, to publicly update or revise any such statements to reflect any change in our expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements.

We are a ~~biopharmaceutical~~clinical-stage biotechnology company ~~dedicated to significantly improving the health and well-being of patients affected by type 2 diabetes, rare diseases and other metabolic diseases. We are~~ focused on developing treatments for patients suffering from complex rare diseases using our novel therapeutics that treat the underlying biological mechanisms through the methionine aminopeptidase 2, or MetAP2, pathway. We have pioneered the study of MetAP2 inhibitors in both common and rare forms of obesity.cell penetrating peptide technology platform. Our lead product candidate, CTI-1601, is~~ZGN-1061,~~ a ~~novel fumagillin-class MetAP2 inhibitor~~subcutaneously administered, ~~by subcutaneous injection,~~recombinant fusion protein intended to deliver human frataxin (“FXN”), an essential protein, to the mitochondria of patients with Friedreich’s ataxia ("FA"). FA is a rare, progressive, and fatal disease in which patients are unable to produce enough FXN due to a genetic abnormality. There is currently ~~being profiled~~no effective therapy for ~~its utility~~FA.

We have completed two Phase 1 clinical trials in patients with FA. We have received an orphan drug designation, fast track designation and rare pediatric disease designation, from the U.S. Food and Drug Administration (the "FDA") for CTI‑1601. In addition, we received orphan drug designation for CTI-1601 from the European Commission and a Priority Medicines ("PRIME") designation from the European Medicines Agency ("EMA"). The receipt of such designations or positive opinions may not result in a faster development process, review or approval compared to products considered for approval under conventional FDA or EMA procedures and does not assure ultimate approval by the FDA or EMA.

Our cell penetrating peptide technology platform, which enables a therapeutic molecule to cross a cell membrane in order to reach intracellular targets, has the potential to enable the treatment of ~~type 2 diabetes~~other rare and ~~other related metabolic disorders.~~orphan diseases. We ~~have completed a Phase 1 clinical trial~~intend to use our proprietary platform to target additional orphan indications characterized by deficiencies in or alterations of~~ZGN-1061~~ ~~in the Netherlands, which was comprised of a single ascending dose,~~ intracellular content or ~~SAD, portion and a multiple ascending dose, or MAD, portion. This clinical trial evaluatedZGN-1061~~ for safety, tolerability and pharmacokinetics while also gaining an early indication of efficacy over four weeks of treatment. We reported positive top line results from this clinical trial in May 2017 and presented the full data package at the American Diabetes Association’s 77th Annual Scientific Sessions in June 2017. In this Phase 1 assessment~~ZGN-1061~~ demonstrated rapid drug absorption and clearance in line with criteria established in advance for the molecule, and had a favorable tolerability profile with no safety signals identified, including no evidence of prothrombotic effects. The data show that~~ZGN-1061~~ treatment causes improvements across multiple metabolic measures consistent with MetAP2 inhibition, and patients in the clinical trial experienced mean weight loss of up to approximately one pound per week. In the third quarter of 2017, we advanced~~ZGN-1061~~ into a Phase 2 clinical trial in both Australia and New Zealand, in patients with type 2 diabetes who are obese and are failing to respond adequately to current therapies. This trial is expected to enroll approximately 120 patients.activity.

~~ZGN-1061~~ acts through potent inhibition of MetAP2, an enzyme that modulates the activity of key cellular processes that control metabolism. MetAP2 inhibitors work, at least in part, by directing MetAP2 binding to cellular stress and growth factor mediators, thereby reducing the tone of signals that drive lipid synthesis by the liver and fat storage throughout the body. In this manner, MetAP2 inhibition serves the purpose of~~re-establishing~~ balance to the ways the body stores and metabolizes fat and glucose. MetAP2 inhibitors reduce the production of new fatty acid molecules by the liver and help convert stored fats into useful energy, while reducing hunger. In animal models that mimic aspects of type 2 diabetes, these processes lead to improvement of glycemic control independent of weight loss.

~~ZGN-1061~~ was discovered by our researchers as part of a multi-year campaign to identify novel compounds for development in the treatment of type 2 diabetes, and that avoided limiting preclinical safety concerns observed with beloranib, including teratogenicity and effects on testicular function. To date, the compound has similar efficacy, potency, and range of activity in animal models as beloranib, but displays highly differentiated properties and improved safety margins in preclinical studies, supporting the value of the compound as a more highly optimized MetAP2 inhibitor. Further, the compound displays improved safety margins in~~non-clinical~~ ~~safety studies relative to beloranib for effects on coagulation in dogs, an effect that correlates with reduced impact on endothelial cell proliferation~~~~in vitro~~.

Since our inception, ~~in November 2005,~~ we have devoted substantially all of our resources to developing~~ZGN-1061,~~ ~~beloranib,ZGN-839~~ ~~and additional MetAP2 inhibitors,~~ CTI-1601, building our intellectual property portfolio, developing ~~our supply chain,~~third-party manufacturing capabilities, business planning, raising capital, and providing general and administrative support for such operations. ~~From~~

On May 20, 2021 we announced that we had received an EMA PRIME designation for CTI-1601 in FA. Through PRIME, the EMA offers early and proactive support to medicine developers to optimize the generation of robust data on a medicine’s benefits and risks and enable accelerated assessment of medicines applications so that these medicines can reach patients earlier. The PRIME designation was based on both pre-clinical data as well as tolerability data from the CTI-1601 Phase 1 program in patients with FA.

In May 2021, we reported positive topline data from our ~~inception through~~Phase 1 FA program after completing dosing of the single ascending dose ("SAD") trial in December 2020 and of the multiple ascending dose ("MAD") trial in March 2021. Data from these trials demonstrate proof-of-concept by showing that daily subcutaneous injections of CTI-1601 for up to 13 days resulted in dose-dependent increases in FXN levels from baseline compared to placebo in all evaluated tissues (buccal cells, skin, and platelets). FXN levels achieved in peripheral tissues (buccal cells) following daily 50 mg and 100 mg subcutaneous injections of CTI-1601 were at or in excess of FXN levels that would be expected in phenotypically normal heterozygous carriers. There were no serious adverse events ("SAEs") associated with either the MAD or SAD trials.

Also in May 2021, the FDA placed a clinical hold on our ~~initial public offering,~~CTI-1601 clinical program after we notified the agency of mortalities at the highest dose levels of the 26-week non-human primate toxicology study that was designed to support extended dosing of patients with CTI-1601. At the time the hold was placed, we had no interventional clinical trials with patients enrolling or ~~IPO,~~enrolled.

In February 2022, in ~~June 2014,~~response to the complete response we ~~received gross~~submitted to the FDA, the FDA stated that it was maintaining the clinical hold and that additional data are needed to resolve the clinical hold. We subsequently submitted a request to the FDA for a Type C meeting, which was granted and was held in July 2022. We submitted a complete response incorporating additional information requested by the FDA at the meeting as well as information on the proposed study in August 2022.

In September 2022, following the Type C meeting and the submission of our complete response, the FDA cleared the initiation of the 25 mg cohort of a Phase 2, four-week, placebo-controlled, dose exploration trial of CTI-1601 in Friedreich’s ataxia (FA) patients. In connection therewith, the FDA lifted its full clinical hold on the CTI-1601 program and imposed a partial clinical hold. This trial is designed to further characterize CTI-1601’s safety and PD and PK profiles to provide information about the preferred long-term dose and dose regimen. We have since initiated the Phase 2 dose exploration trial, with top-line data expected in the second half of 2023. In connection with the FDA’s partial clinical hold, the FDA will review the data from the 25 mg cohort prior to our being authorized to initiate a second cohort and/or other clinical trials. Larimar does not know when, or if the partial clinical hold will be lifted.

We have funded our operations to date primarily with proceeds ~~of $104.0 million~~ from sales of redeemable convertible preferred stock and, to a lesser extent, through the issuances of convertible promissory notes. During June 2014, we completed our IPO with net proceeds of $102.7 million after deducting underwriting discounts and commissions paid by us. On January 28, 2015, we completed a~~follow-on~~ ~~offering of our~~ common stock, ~~with net~~ proceeds from the sale of ~~$130.0 million after deducting underwriting discounts and commissions paid by us.~~

We have never generated any revenue and have incurred net losses in each year since our inception. We have an accumulated deficit of $276.5 million as of September 30, 2017. Our net loss was $38.9 millionprefunded warrants for the ~~nine months ended September 30, 2017 and $57.9 million for~~purchase of common stock, the ~~year ended December 31, 2016. These losses have resulted principally from costs incurred~~acquisition in ~~connection within-licensing~~2020 of beloranib, research and development activities and general and administrative costs associated with our operations. We expect to incur significant expenses and operating losses for the foreseeable future.

~~We expect to continue to incur expenses in connection with our ongoing activities, if and as we:~~

As a result, we will need additional financing to support our continuing operations. Until such time that we can generate significant revenue from product sales, if ever, we expect to finance our operations through a combination of public, private equity, debt financings, or other sources, which may include collaborations with third parties. Arrangements with collaborators or others may require us to relinquish rights to certain of our technologies or product candidates. In addition, we may never successfully complete development of any of our product candidates, obtain adequate patent protection for our technology, obtain necessary regulatory approval for our product candidates or achieve commercial viability for any approved product candidates. Adequate additional financing may not be available to us on acceptable terms, or at all. Our failure to raise capital as and when needed would have a negative impact on our financial condition and our ability to pursue our business strategy. We will need to generate significant revenue to achieve profitability, and we may never do so.

~~We expect that our existing~~cash, cash equivalents, restricted cash, cash equivalents and marketable debt securities upon the merger with Zafgen, Inc. and, prior to the 2020 merger with Zafgen, capital contributions from Chondrial Holdings, LLC.

In August 2020, we entered into an Equity Distribution Agreement ("the Prior ATM Agreement") with an investment bank in connection with the establishment of an “at-the-market” offering program providing for the sale of up to an aggregate of 50,000,000 of shares of our common stock from time to time through the investment bank as sales agent.

In July 2021, we sold 2,342,720 shares pursuant to the Prior ATM Agreement for net proceeds of $19.9 million, after issuance costs. The Prior ATM Agreement was terminated in November 2022 in connection with the establishment of a new "at-the-market" offering program.

In November 2022, we entered into a Sales Agreement (the "ATM Agreement") with an investment bank in connection with the establishment of an “at-the-market” offering program providing for the sale of up to an aggregate of $50.0 million of shares of our common stock from time to time through this investment bank as sales agent.

In September ~~30, 2017, will enable us to fund our operating expenses and capital expenditure requirements~~2022, the Company sold 25,558,750 shares of common stock, for a ~~period~~price of ~~at least one year from~~$3.15 per share, in an underwritten offering for net proceeds of $75.2 million.

In late 2019, an outbreak of a novel strain of the ~~issuance date~~Coronavirus 2019 Disease (COVID-19) was identified and infections have been found in a number of ~~this Quarterly Report. See “—Liquidity~~countries around the world, including the United States. In 2020, the pandemic resulted in a four-month temporary stoppage of the Company’s CTI-1601Phase 1 clinical trials in patients with Friedreich’s ataxia. Both of the Company's SAD and ~~Capital Resources.”~~MAD clinical trials were completed in 2021. In the future, the COVID-19 pandemic and responsive measures thereto may result in negative impacts on the Company, including possible delays in our clinical and regulatory activities. We cannot be certain what the overall impact of the COVID-19 pandemic will be on our business and it has the potential to materially adversely affect our business, financial condition, results of operations, and prospects.

WeTo date, we have not generated any revenue from product sales, ~~since our inception,~~ and do not expect to generate any revenue from the sale of products in the ~~near~~foreseeable future. If our development efforts result in clinical success and regulatory approval or collaboration agreements with third parties for our product candidates, we may generate revenue from those product candidates or collaborations.

The majority of our operating expenses since inception have consisted primarily of research and development ~~activities,~~ and general and administrative costs.

Research and development expenses, which consist primarily of costs associated with our product research and development efforts, are expensed as incurred. Research and development expenses consist primarily of:

WeCosts for certain activities, such as manufacturing, non-clinical studies and clinical trials are ~~currently primarily focused~~generally recognized based on ~~developingZGN-1061~~the evaluation of the progress of completion of specific tasks using information and ~~other early research activities~~data provided by our vendors and typically use our employee, consultant and infrastructure resources across our research and development programs. We track outsourced development costs by product candidate or development program, but we do not allocate personnel costs, external consultant costs, payments made under our licensing agreements or other internal costs to specific development programs or product candidates unless the payments are specifically identifiable to a development program or product candidate. We record our research and development expenses net of any research and development tax incentives we are entitled to receive from government authorities.

collaborators. Research and development activities are central to our business. ~~Product candidates~~We expect to increase our investment in ~~later stages of clinical~~research and development ~~generally have higher development costs than those~~ in ~~earlier stages of clinical development, primarily due~~order to ~~the increased size and duration of later-stage~~advance CTI-1601 through additional clinical trials. WeAs a result, we expect that our research and development expenses will increase in the foreseeable future as we pursue ~~later stages of~~ clinical development of ~~our~~CTI-1601 and/or any other product ~~candidates.~~candidates we develop.

At this time, we cannot ~~determine with certainty~~reasonably estimate or know the ~~duration~~nature, timing and ~~completion~~estimated costs of the ~~current~~efforts that will be necessary to complete the clinical and commercial development of CTI-1601 or ~~future clinical trials of our~~any other product candidates orwe develop. We are also unable to predict when, if ~~when, or to what extent we~~ever, material net cash inflows will ~~generate revenue~~commence from ~~the commercialization and sale of any of our product candidates that obtain regulatory approval. We may never succeed in achieving regulatory approval for any~~sales of our product candidates. The duration, costs, and timing of clinical trials and development of ~~our~~CTI-1601 or any other product candidates we develop will depend on a variety of factors, including:

A change in the outcome of any of these variables with respect to the development of a product candidate could ~~mean a significant~~significantly change in the costs, timing and ~~timing~~viability associated with the development of that product candidate. For example, if the FDA or another regulatory authority were to require us to conduct additional non-clinical or clinical trials beyond those that we currently anticipate will be required for the completion of clinical development of a product candidate, or if we experience significant delays in enrollment in any of our clinical trials, we could be required to expend significant additional financial resources and time on the completion of clinical development.

General and administrative expenses ~~consist~~(consisting primarily of personnel costs, ~~consisting~~composed of salaries, related benefits and stock-based ~~compensation, of~~compensation), costs related to our executive, finance, ~~business~~information technology, and ~~corporate development and~~costs related to other administrative functions. General and administrative expenses also include ~~travel expenses, allocated facility-related costs not otherwise included in research and development expenses,~~ insurance expenses and professional fees for auditing, tax, and legal services, including legal expenses to pursue patent protection offor our intellectual property. We expect that our general and administrative expenses will ~~decrease during 2017~~increase in the foreseeable future as ~~compared~~we hire additional employees to ~~2016 primarily due to reduced headcount~~implement, improve and scale our operational, financial, commercial and management systems.

Our condensed consolidated financial statements are prepared in accordance with GAAP. The preparation of our condensed consolidated financial statements and related ~~costs.~~

~~Interest income.~~ ~~Interest income consists~~disclosures requires us to make estimates and assumptions that affect the reported amount of ~~interest earned~~assets, liabilities, costs and expenses, and related disclosures. We believe that the estimates and assumptions involved in the accounting policies described below may have the greatest potential impact on our ~~cash equivalents~~condensed consolidated financial statements and, ~~marketable securities.~~therefore, consider these to be our critical accounting policies. We evaluate these estimates and assumptions on an ongoing basis. Our ~~interest income has not been significant due to low interest earned on invested balances. We anticipate that our interest income will decrease as we incur operating losses.~~actual results may differ from these estimates under different assumptions and conditions.

~~Interest expense.~~ ~~Interest expense relates to outstanding borrowings under the 2014 Credit Facility, consisting~~22

As part of the ~~stated interest~~process of ~~8.1% per year due~~preparing our condensed consolidated financial statements, we are required to estimate our accrued research and development expenses and evaluate payments made to vendors in advance of actual work activities being performed. This process involves reviewing open contracts and purchase orders, communicating with our personnel and outside vendors to identify services that have been performed on ~~outstanding borrowings,~~our behalf and estimating the level of service performed and the associated costs incurred for the services when we have not yet been invoiced or otherwise notified of the actual costs. The majority of our service providers invoice us in arrears for services performed, on a ~~final~~pre-determined schedule or when contractual milestones are met. We make estimates of our accrued expenses as of each balance sheet date in our condensed consolidated financial statements based on facts and circumstances known to us at that time. Examples of estimated accrued research and development expenses include fees paid to:

We base our expenses related to clinical trials on our estimates of the services received and efforts expended pursuant to contracts with multiple CROs or CMOs that conduct and manage clinical trials or manufacture clinical trial material on our behalf. The financial terms of these agreements are subject to negotiation, vary from contract to contract and may result in uneven payment flows. There may be instances in which payments made to our vendors will exceed the level of 6%services provided and result in a prepayment of ~~amounts drawn down that is being recorded~~the clinical expense, non-clinical expense, or manufacturing activities. Payments under some of these contracts depend on factors such as interest expense over the term through the maturity date using the effective-interest method, the amortization of deferred financing costs, the accretion of debt discounts relating to the 2014 Credit Facility, and a fee which was paid to the lender upon the completion of ~~our IPO.~~

~~Foreign currency transaction gains (losses), net.~~ ~~Foreign currency transaction gains (losses), net consists~~clinical trial milestones. In accruing service fees, we estimate the time period over which services will be performed, enrollment of patients, number of sites activated and the level of effort to be expended in each period. In accruing CMO costs, we estimate the time period that manufacturing will be completed, the achievement of milestones and the percentage of completion of each specific CMO agreement. If the actual timing of the ~~realized and unrealized gains and losses~~performance of services or the level of effort varies from ~~foreign currency-denominated cash balances, vendor payables andtax-related~~ ~~receivables from~~our estimate, we adjust the ~~Australian government. We currently~~accrual or prepaid accordingly. Although we do not ~~engage~~expect our estimates to be materially different from amounts actually incurred, our understanding of the status and timing of services performed relative to the actual status and timing of services performed may vary and may result in ~~hedging activities related~~us recognizing adjustments in future periods as additional information becomes available.

We measure all stock-based awards granted to employees, directors and non-employee consultants based on the fair value on the date of grant using the Black-Scholes option-pricing model. Compensation expense of those awards is recognized over the requisite service period, which is generally the vesting period of the respective award. Typically, we issue awards with only service-based vesting conditions and record the expense for these awards using the straight-line method. We account for forfeitures as they occur.

We classify stock-based compensation expense in our ~~foreign currency-denominated receivables~~condensed consolidated statements of operations and ~~payables; as such,~~comprehensive loss in the same manner in which the award recipient’s payroll costs are classified or in which the award recipient’s service payments are classified.

Prior to May 28, 2020, we ~~cannot predict~~were a private company and lacked company-specific historical and implied volatility information for our common stock. Therefore, we estimate our expected common stock price volatility based on the ~~impact~~historical volatility of ~~future foreign currency transaction gains~~publicly traded peer companies and ~~losses on our operating results. See “—Quantitative and Qualitative Disclosures about Market Risk.”~~

~~Since our inception in 2005,~~expect to continue to do so until we have ~~not recorded any~~adequate historical data regarding the volatility of our own traded stock price. The expected term of our stock options have been determined utilizing the “simplified” method for awards that qualify as “plain-vanilla” options. The risk-free interest rate is determined by reference to the U.S. ~~federal or state income tax benefits~~Treasury yield curve in effect at the time of grant of the award for time periods approximately equal to the ~~net losses~~expected term of the award. Expected dividend yield considers the fact that we have ~~incurred in each year or our earned tax credits, due~~never paid cash dividends on common stock and do not expect to our uncertainty of realizing a benefit from those items. As of December 31, 2016, we had net operating loss carryforwards for federal and state income tax purposes of $49.1 million and $35.9 million, respectively, which begin to expire in 2026 and 2030, respectively. As of December 31, 2016, we did not record deferred tax assets of $12.8 million (gross) that were attributable to stock option exercises which will be recorded as an increase in additional paid in capital once they are realized in accordance with accounting for stock-based compensation awards. These deductions are not reflectedpay any cash dividends in the federal and state net operating loss carryforwards and the capitalized research and development expense deferred tax assets in the amounts of $9.4 million, $7.2 million, and $3.4 million, respectively. As of December 31, 2016, we also had available tax credit carryforwards for federal and state income tax purposes of $13.1 million and $1.9 million, respectively, which begin to expire in 2026 and 2021, respectively.foreseeable future.

Comparison of ~~the Three Months Ended~~three months ended September 30, ~~2017~~2022 and ~~2016~~2021

The following table summarizes our results of operations for the three months ended September 30, ~~2017~~2022 and ~~2016:~~2021:

Research and development expenses for the three months ended September 30, ~~2017~~2022 decreased ~~$0.3~~$8.4 million compared to the three months ended September 30, ~~2016.~~2021. The decrease in research and development expenses was primarily ~~due to~~driven by a ~~$3.3~~decrease of $6.7 million in drug manufacturing costs, a decrease of $1.0 million in ~~our beloranib program and~~nonclinical development costs, a decrease of $0.5 million ~~associated with our unallocated expenses,~~in consulting expenditures, a decrease of $0.5 million in clinical expense and a decrease of $0.3 million in internal lab costs. These decreases were partially offset by ~~increased costs~~an increase of ~~$1.9~~$0.4 million ~~related to work on discovery and screening~~in personnel expense and an increase of ~~$1.7~~$0.1 million ~~related to ourZGN-1061~~ ~~program.~~

~~Costs~~in non-cash, stock-based compensation expense associated with ~~our discovery~~stock option grants made in 2021 and ~~screening programs increased $1.9 million over the prior period as during the 2017 period we had considerable costs related to discovery work for our early stage programs.~~2022.

~~Costs associated with ourZGN-1061~~ ~~program increased period over period by $1.7 million. In the third quarter of 2016 we initiated a Phase 1 clinical trial forZGN-1061~~ which completed recruiting and dosing patients in the first quarter of 2017. The overall increase is primarily due to additional preclinical studies as well as drug product and drug substance activities as we have commenced a Phase 2 clinical trial in the third quarter of 2017 in both Australia and New Zealand, for which we plan to enroll approximately 120 patients for 12 weeks of dosing. The expenses during the 2016 period were for the Phase 1 clinical trial of~~ZGN-1061~~ ~~in the Netherlands, which was comprised of 39 patients in a single ascending dose, or SAD, portion and 29 patients in a multiple ascending dose, or MAD, portion.~~

Of the decrease in cost associated with our beloranib program, clinical trials costs and related expenses decreased by $2.0 million period over period and preclinical and manufacturing costs decreased $1.4 million period over period, both as a result of the suspension of our beloranib program in July 2016. We suspended the development of our beloranib program, however safety and~~close-out~~ ~~visits were still being conducted for patients in our U.S. Phase 3 clinical trial in patients with PWS.~~

Unallocated expenses decreased period over period primarily due to a decrease in personnel related costs of $0.6 million. Personnel related expenses decreased primarily from a reduction in the number of employees for the second quarter of the 2017 as compared to 2016, as we had a reduction in workforce which took place in July 2016.

General and administrative expenses for the three months ended September 30, ~~2017 decreased $1.7~~2022 increased $0.2 million compared to the three months ended September 30, ~~2016.~~2021. The ~~decrease~~increase in general and administrative expense was ~~due to decreases in personnel related costs of $1.2 million, professional fees~~primarily driven by an increase of $0.2 million in professional fees associated with legal and~~non-cash~~ ~~stock-based compensation expense~~ accounting services and an increase of $0.2 ~~million. Personnel related expenses decreased~~million in personnel expense, partially offset by a decrease of $0.2 million in operational costs primarily due to a reduction in the number of employees for the second quarter of 2017 as compared to 2016, as we had a reduction in workforce which took place in July 2016. Professional fees decreased primarily due to lower legal fees as the plaintiffs for the class action lawsuit had filed a notice of appeal to the First Circuit Court of Appeals and on April 7, 2017, the dismissal with prejudice was affirmed.~~Non-cash~~ ~~stock compensation decreased due mainly to forfeited stock options~~ related to ~~the reduction in workforce in July 2016, as well as a lower stock option grant price for the 2017 annual grant.~~insurance, technology and recruiting services.

~~Interest expense.~~ ~~Interest expense for the three~~Comparison of nine months ended September 30, ~~2017~~2022 and 2016 of less than $0.1 million and $0.1 million, respectively, was related to interest expense on our outstanding borrowings under the 2014 Credit Facility. Interest expense consists primarily of the stated interest of 8.1% per year due on outstanding borrowings. It also includes expense related to the final payment of 6% of amounts drawn down that is being recorded over the term through the maturity date using the effective-interest method and the amortization of deferred financing costs and debt discounts relating to the 2014 Credit Facility.2021

~~Interest income.~~ ~~Interest income of $0.3 million and $0.2 million, respectively, for the three months ended September 30, 2017 and 2016 was related to interest earned on our marketable securities balances.~~

~~Foreign currency transaction gains (losses), net.~~ We had foreign currency transaction gains of less than $0.1 million and $0.1 million for the three months ended September 30, 2017 and 2016, respectively. Foreign currency transaction gains and losses consisted of the realized and unrealized gains and losses from foreign currency-denominated cash balances, vendor payables and~~tax-related~~ ~~receivables from the Australian government, generally reflecting the fluctuation of the Australian dollar relative to the U.S. dollar.~~

The following table summarizes our results of operations for the nine months ended September 30, ~~2017~~2022 and ~~2016:~~2021:

Research and development expenses for the nine months ended September 30, ~~2017~~2022 decreased ~~$2.7~~$15.1 million compared to the nine months ended September 30, ~~2016.~~2021. The decrease in research and development expenses was primarily ~~due to~~driven by a ~~$12.1~~decrease of $9.0 million in drug manufacturing costs, a decrease of $3.5 million in ~~our beloranib program~~clinical trial expense, a decrease of $3.3 million in nonclinical development costs, a decrease of $0.5 million in consulting expenses and a decrease of ~~$3.1~~$0.4 million ~~associated with our unallocated expenses~~in internal lab costs. These decreases were partially offset by ~~increased costs of $9.6 million related to ourZGN-1061~~ ~~program, as well as~~ an increase of $0.8 million in ~~discovery~~personnel expense and ~~screening expenses~~an increase of ~~$3.7 million.~~

~~Costs~~$0.5 million in non-cash, stock-based compensation expense associated with ~~ourZGN-1061~~ ~~program increased period over period by $9.6 million. In the third quarter of 2016 we initiated the Phase 1 clinical trial forZGN-1061~~ ~~which completed recruiting~~stock option grants made in 2021 and dosing patients in the first quarter of 2017. The overall increase is primarily due to additional preclinical studies as well as drug product and drug substance activities as we commenced our Phase 2 clinical trial in the third quarter of 2017 in both Australia and New Zealand, for which we plan to enroll 120 patients in a 12 week clinical trial. The expenses during the 2016 period were for startup costs for the Phase 1 clinical trial of~~ZGN-1061~~ ~~in the Netherlands, which was comprised of 39 patients in a single ascending dose, or SAD, portion and 29 patients in a multiple ascending dose, or MAD, portion.~~2022.

Of the decrease in costs associated with our beloranib program, clinical trials and related expenses decreased by $7.6 million period over period and preclinical and manufacturing costs decreased $4.5 million period over period, both as a result of the suspension of our beloranib program in July 2016. During the nine months ended September 30, 2016, we reported topline clinical data from our Phase 2b clinical trial in patients with severe obesity complicated by type 2 diabetes and our U.S. Phase 3 clinical trial in patients with PWS. Prior to the FDA placing the investigational new drug application, or IND, for beloranib on full clinical hold in December 2015, we suspended dosing of patients in the randomized portion of both of these trials in October 2015. In July 2016, we announced the suspension of our beloranib program.

Unallocated expenses decreased period over period primarily due to a decrease of $1.3 million in consultants, as well as a decrease in personnel related costs of $1.3 million. Consultant costs decreased primarily related to our beloranib program which we suspended in July 2016. Personnel related expenses decreased primarily from a reduction in the number of employees for the 2017 period as compared to the 2016 period, as we had a reduction in workforce which took place in July 2016. Other unallocated expenses decreased due to lower travel expenses and facilities expenses.

General and administrative expenses for the nine months ended September 30, ~~2017~~2022 decreased ~~$5.4~~$0.2 million compared to the nine months ended September 30, ~~2016.~~2021. The decrease in general and administrative expense was ~~due~~primarily driven by a decrease of $0.5 million in operational costs primarily related to ~~decreases~~technology and recruiting services and a decrease of $0.4 million in ~~personnel related costs~~professional fees primarily associated with legal and consulting expense, partially offset by an increase of ~~$2.1~~$0.5 million~~non-cash~~ in stock-based compensation expense of $1.6 million and professional fees of $1.4 million. Personnel related expenses decreased primarily from a reduction in the number of employees for the 2017 period as compared to the 2016 period, from the reduction in workforce which took place in July 2016.~~Non-cash~~ ~~stock-based compensation expense decreased due mainly to forfeited stock options related to the reduction in workforce in July 2016, as well as a lower~~associated with stock option grant price for the 2017 annual grant. Professional fees decreased primarily due to lower legal fees as the plaintiffs for the class action lawsuit had filed a notice of appeal to the First Circuit Court of Appealsgrants made in 2021 and ~~on April 7, 2017, the dismissal with prejudice was affirmed. The decrease was also due to lower commercial readiness costs~~2022 and ~~investor relations and public relations costs in the 2017 period as compared to the same period in 2016, primarily as a result of suspending development of beloranib in July 2016.~~

~~Interest expense.~~ ~~Interest expense for the nine months ended September 30, 2017 and 2016~~an increase of $0.2 million and $0.4 million, respectively, was related to interest expense on our outstanding borrowings under the 2014 Credit Facility. Interest expense consists primarily of the stated interest of 8.1% per year due on outstanding borrowings. It also includes expense related to the final payment of 6% of amounts drawn down that is being recorded over the term through the maturity date using the effective-interest method and the amortization of deferred financing costs and debt discounts relating to the 2014 Credit Facility.in personnel expense.

~~Interest income.~~ ~~Interest income of $0.7 million for the nine months ended September 30, 2017 and 2016 was related to interest earned on our marketable securities balances.~~

~~Foreign currency transaction gains (losses), net.~~ We had foreign currency transaction gains of $0.1 million for the nine months ended September 30, 2017 and 2016. Foreign currency transaction gains and losses consisted of the realized and unrealized gains and losses from foreign currency-denominated cash balances, vendor payables and~~tax-related~~ ~~receivables from the Australian government, generally reflecting the fluctuation of the Australian dollar relative to the U.S. dollar.~~

As of September 30, 2017, we had cash, cash equivalents and marketable securities totaling $93.2 million. We invest our cash in money market funds, certificates of deposit, commercial paper and corporate bonds, with the primary objectives to preserve principal, provide liquidity and maximize income without significantly increasing risk.

Since our inception, ~~in November 2005,~~ we have not generated any revenue from any sources, including from product sales, and have incurred ~~recurring net losses. As of September 30, 2017, we had an accumulated deficit of $276.5 million. From~~significant operating losses and negative cash flows from our inception through our IPO in June 2014, we received gross proceeds of $104.0 million from sales of redeemable convertible preferred stock and, to a lesser extent, through the issuances of convertible promissory notes and a loan security agreement. During June 2014, we completed our IPO with net proceeds of $102.7 million after deducting underwriting discounts and commissions paid by us. On January 28, 2015, we completed a~~follow-on~~ ~~offering of our common stock, with net proceeds of $130.0 million after deducting underwriting discounts and commissions paid by us.~~

operations. We have ~~outstanding amounts under the 2014 Credit Facility, which we entered into in March 2014. All promissory notes issued under the 2014 Credit Facility are collateralized by~~devoted substantially all of our ~~personal property, other than~~resources to developing CTI-1601, building our intellectual property. There are no financial covenants associated with the 2014 Credit Facility; however, there are negative covenants restricting our activities, including limitations on dispositions, mergers or acquisitions; encumbering or granting a security interest in our intellectual property; incurring indebtedness or liens; paying dividends; making certain investments;property portfolio, developing third-party manufacturing capabilities, business planning, capital raising, and ~~certain other business transactions.~~providing general and administrative support for such operations.

Upon entering into this 2014 Credit Facility, we were obligated to make monthly, interest-only payments on any term loans funded under the 2014 Credit Facility until December 1, 2014 and, thereafter, to pay 36 consecutive, equal monthly installments of principal and interest from January 1, 2015 through December 1, 2017. As per the terms of the agreement, in June 2014, upon the completion of our IPO, the term of monthly, interest-only payments was extended until June 1, 2015. Outstanding term loans under the 2014 Credit Facility bear interest at an annual rate of 8.1%. In addition, a final payment equal to 6.0% of any amounts drawn under the facility is due upon the earlier of the maturity date, acceleration of the term loans or prepayment of all or part of the term loans. We were also obligated to pay a separate fee upon any initial public offering; a sale of substantially all of our assets; or a merger, reorganization or sale of our voting equity securities where existing voting stockholders hold less than 50% of voting equity securities after such a transaction.Cash Flows

The following table summarizes our sources and uses of cash for each of the periods presented below:

During the nine months ended September 30, ~~2017,~~2022, operating activities used ~~$33.9~~$20.9 million of cash, resulting from our net loss of ~~$38.9~~$25.9 million, ~~partially offset by~~adjusted for noncash expenses of $5.2 million and changes in our operating assets and liabilities resulting in a source of ~~$1.2 million andnon-cash~~ ~~charges~~cash of ~~$6.2~~$0.1 million. Our net loss was primarily attributed to research and development activities related to our~~ZGN-1061~~ CTI-1601 program and our general and administrative ~~expenses. Our netnon-cash~~ ~~charges during the nine months ended September 30, 2017, consisted~~expenses as described above. Noncash expenses are primarily of stock-based compensation expense of $6.2 million. Net cash used in changes in our operating assets and liabilities during the nine months ended September 30, 2017, consisted primarily of a $0.6 million decrease in accrued expenses as well as an increase in prepaid expenses and other current assets of $0.6 million.expense.

During the nine months ended September 30, ~~2016,~~2021, operating activities used ~~$43.3~~$34.2 million of cash, resulting from our net loss of ~~$47.4~~$41.5 million, ~~and changes in our operating assets and liabilities~~adjusted for noncash expenses of ~~$5.0 million, partially offset bynon-cash~~ ~~charges of $9.1~~$4.2 million. Our net loss was primarily ~~attributed~~attributable to research and development activities related to our ~~beloranib program, ourZGN-1061~~CTI-1601 program and our general and administrative ~~expenses. Our netnon-cash~~ ~~charges during~~expenses as described above. Noncash expenses primarily include stock-based compensation expense. For the nine months ended September 30, ~~2016, consisted primarily~~2021 the increase in accrued expenses and prepaid expenses, partially offset by the paydown of ~~stock-based compensation expense~~accounts payable resulted in a source of ~~$8.1~~cash of $3.1 million.

Net cash ~~used in changes in our operating assets and liabilities during the nine months ended September 30, 2016, consisted primarily of a $5.0 million decrease in accounts payable and accrued expenses.~~

During the nine months ended September 30, ~~2017,~~2022, investing activities ~~provided $23.0~~used $38.7 million of cash, ~~resulting~~including $61.6 million in purchases of new marketable debt securities and $0.1 million in purchases of property and equipment, partially offset by a $23.0 million increase from ~~proceeds from sales and~~ maturities of marketable ~~securities of $112.2 million, offset primarily by the use of cash for purchases of marketable securities of $89.2 million.~~debt securities.

During the nine months ended September 30, ~~2016,~~2021, investing activities provided ~~$34.5~~$17.7 million of cash, resulting from ~~proceeds from sales and~~ maturities of marketable debt securities of ~~$141.4~~$26.3 million, partially offset by ~~the use of cash for~~$8.3 million in purchases of new marketable ~~securities of $106.2 million and purchases of property and equipment of $0.7 million.~~debt securities.

During the nine months ended September 30, ~~2017,~~2022, financing activities ~~used $2.4~~provided $75.6 million ~~for payments related to our notes payable, partially offset by $0.2 million received~~of cash from ~~proceeds relating to the exercise~~sale of common stock ~~options and common stock purchased under our 2014 Employee Stock Purchase Plan.~~in an underwritten public offering, net of issuance costs.

During the nine months ended September 30, ~~2016,~~2021 financing activities ~~used $2.2~~provided $19.9 million ~~for payments related to our notes payable, partially offset by $0.2 million received~~of net cash, from ~~proceeds relating to the exercise~~sale of common stock ~~options and common stock purchased~~ under ~~our 2014 Employee Stock Purchase Plan.~~

~~ZGN-1061~~ ~~has completed Phase 1 clinical development, therefore we~~We have not yet commercialized any products and do not expect to ~~continue~~generate revenue from the commercial sale of any products for several years, if at all.

We have to date incurred net losses. We incurred net losses of approximately $25.9 million and $41.5 million for the nine months ended September 30, 2022 and 2021, respectively. As of September 30, 2022, we had an accumulated deficit of $142.2 million and cash, cash equivalents and marketable debt securities of $124.7 million, excluding restricted cash of $1.3 million.

Losses have resulted principally from costs incurred in connection with research and development activities, and general and administrative costs associated with the development of CTI-1601 and our operations. We expect to incur significant expenses and operating losses for the foreseeable ~~future. We anticipate that~~future as we ~~will~~expect to continue to incur expenses in connection with our ongoing activities, if and as we:

We ~~expect~~believe that based on our ~~existing~~current operating plan our cash, cash equivalents and marketable debt securities ~~as of September 30, 2017,~~ will ~~enable us to~~be able fund ~~our~~ operating expenses and capital expenditure requirements for ~~a period of~~ at least ~~one year~~the next twelve months from the ~~issuance date~~filing of ~~this Quarterly Report. We have based this estimate on~~these financial statements with the SEC. If we encounter unexpected delays in our clinical trials or if there are other unanticipated changes to our operating plan from our current assumptions that ~~may prove to be wrong, and~~negatively impact our operations, we may ~~use~~reduce expenditures in order to further extend our ~~available capital resources sooner than we currently expect. Because of the numerous risks and uncertainties associated with the development ofZGN-1061~~ and our other research and development activities and because the extent to which we may enter into collaborations with third parties for the development of our product candidates is unknown, we are unable to estimate the amounts of increased capital outlays and operating expenses associated with completing the research and development of our product candidates. Our future capital requirements for~~ZGN-1061~~ ~~will depend on many factors, including:~~

existing cash resources. Until ~~such time, if ever, as~~ we can generate substantial ~~product~~ revenue, if ever, we expect to ~~finance our cash needs~~seek additional funding through a combination of public or private equity offerings, debt financings, collaborations ~~strategic alliances~~ and licensing arrangements. To the extent that we raise additional capital through the sale of equity or convertible debt securities, the ownership interest of our stockholders will be diluted, and the terms of these securities may include liquidationarrangements or other ~~preferences that adversely affect the rights~~sources. The incurrence of ~~our common stockholders. Debt financing, if available,~~indebtedness would result in increased fixed payment obligations and we may ~~involve agreements that include~~be required to agree to certain restrictive covenants, ~~limiting or restricting~~such as limitations on our ability to ~~take specific actions, such as incurring~~incur additional debt, ~~making capital expenditures~~minimum cash balances, limitations on our ability to acquire, sell or ~~declaring dividends~~license intellectual property rights and other operating restrictions that could adversely impact our ability to conduct our business. Any additional fundraising efforts may ~~require the issuance of warrants,~~divert our management from their day-to-day activities, which ~~could potentially dilute their ownership interest. If~~may adversely affect our ability to develop and commercialize our product candidates.

There can be no assurance that we raise additional funds through collaborations, strategic alliances or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs or grant licenses on terms that may notwill be ~~favorable to us. If we are unable~~able to raise sufficient additional ~~funds through equity,~~capital on acceptable terms, if at all. If such additional financing is not available on satisfactory terms, or ~~debt financings when needed,~~is not available in sufficient amounts, or we do not have sufficient authorized shares, we may be required to delay, limit, or eliminate the development of business opportunities and our ability to achieve our business objectives, our competitiveness, and our business, financial condition, and results of operations will be materially adversely affected. We could also be required to seek funds through arrangements with collaborative partners or otherwise at an earlier stage than otherwise would be desirable and we may be required to relinquish rights to some of our technologies or product candidates or otherwise agree to terms unfavorable to us, any of which may have a material adverse effect on our business, operating results and prospects. In addition, geopolitical unrest, the impact of the COVID-19 pandemic and/or the potential impact of other health crises on the global financial markets may reduce our ability to access capital, which could negatively affect our liquidity and ability to continue as a going concern.

If we are unable to obtain sufficient funding when needed and/or ~~terminate~~on acceptable terms, we may be required to significantly curtail, delay or discontinue one or more of our research and development programs, the manufacture of clinical and commercial supplies, product ~~development~~portfolio expansion or ~~future~~pre commercialization efforts, which could adversely affect our business prospects, or ~~grant~~we may be unable to continue operations. Certain restrictive covenants, such as limitations on our ability to incur additional debt, limitations on our ability to acquire, sell or license intellectual property rights and other operating restrictions that could adversely impact our ability to conduct our business. Any additional fundraising efforts may divert our management from their day-to-day activities, which may adversely affect our ability to develop and ~~marketZGN-1061~~ ~~that we would otherwise prefer to develop and market ourselves.~~commercialize our product candidates.

Since our inception in 2005, we have not recorded any U.S. federal or state income tax benefits for the net losses we have incurred in each year or our earned tax credits, due to our uncertainty of realizing a benefit from those items. As of December 31, 2016, we had net operating loss carryforwards for federal and state income tax purposes of $49.1 million and $35.9 million, respectively, which begin to expire in 2026 and 2030, respectively. As of December 31, 2016, we did not record deferred tax assets of $12.8 million (gross) that were attributable to stock option exercises which will be recorded as an increase in additional paid in capital once they are realized in accordance with accounting for stock-based compensation awards. These deductions are not reflected in the federal and state net operating loss carryforwards and the capitalized research and development expense deferred tax assets in the amounts of $9.4 million, $7.2 million, and $3.4 million, respectively. As of December 31, 2016, we also had available tax credit carryforwards for federal and state income tax purposes of $13.1 million and $1.9 million, respectively, which begin to expire in 2026 and 2021, respectively. We have not completed a study to assess whether an ownership change, generally defined as a greater than 50% change (by value) in the equity ownership of our corporate entity over a three-year period, has occurred or whether there have been multiple ownership changes since our inception, due to the significant costs and complexities associated with such studies. Accordingly, our ability to utilize our tax carryforwards may be limited. Additionally, U.S. tax laws limit the time during which these carryforwards may be utilized against future taxes. As a result, we may not be able to take full advantage of these carryforwards for federal and state tax purposes.

During the nine months ended September 30, 2017, with the exception of the lease extensions described below, there have been no material changes to our contractual obligations and commitments outside the ordinary course of business from those described under the heading “Management’s Discussion and Analysis of Financial Condition and Results of Operations—Contractual Obligations and Commitments” in our Annual Report on Form~~10-K~~ ~~for the year ended December 31, 2016, our Quarterly Report on Form10-Q~~ ~~for the period ended March 31, 2017, as filed with the SEC on May 9, 2017, and our Quarterly Report on Form10-Q~~ for the period ended June 30, 2017, as filed with the SEC on August 9, 2017. As of July 31, 2017, we negotiated the financial terms of the lease extensions for both of our office spaces in Boston, Massachusetts with additional minimum lease payments of $0.2 million for the remainder of 2017, $0.4 million for the years 2018 and 2019 and $0.2 million for 2020.

During the periods presented we did not have, and we currently do not ~~currently~~ have, anyoff-balance sheet arrangements, as defined under ~~Securities and Exchange Commission~~applicable SEC rules, such as relationships with unconsolidated entities or financial partnerships, which are often referred to as structured finance or special purpose entities, established for the purpose of facilitating financing transactions that are not required to be reflected on our balance sheets.

Our condensed consolidated financial statements are prepared in accordance with GAAP. The preparation of our financial statements and related disclosures requires us to make estimates, assumptions and judgments that affect the reported amount of assets, liabilities, revenue, costs and expenses, and related disclosures. We believe that of our critical accounting policies which are described under the heading “Management’s Discussion and Analysis of Financial Condition and Results of Operations—Critical Accounting Policies and Significant Judgments and Estimates” in our Annual Report on~~Form 10-K~~ ~~for the year ended December 31, 2016, the following accounting policies involve the most judgment and complexity:~~

Accordingly, we believe the policies set forth above are critical to fully understanding and evaluating our financial condition and results of operations. If actual results or events differ materially from the estimates, judgments and assumptions used by us in applying these policies, our reported financial condition and results of operations could be materially affected. There have been no material changes to our critical accounting policies since December 31, 2016.

Please read Note 2 to our condensed consolidated financial statements included in Part I of Item 1 ~~“Financial Statements and Supplementary Data,”~~ of this Quarterly Report on Form 10-Q for a description of recent accounting pronouncements applicable to our business.

Item 3. Quantitative and ~~marketable securities~~Qualitative Disclosures about Market Risk

We are a "smaller reporting company" as of September 30, 2017 consisted of cash, corporate bonds, commercial paper, certificates of deposits, and money market accounts. The primary objectives of our investment activities are to preserve principal, provide liquidity and maximize income without significantly increasing risk. Our primary exposure to market risk is interest income sensitivity, which is affecteddefined by ~~changes in the general level~~Rule 12b-2 of ~~U.S. interest rates. If market interest rates were to increase immediately and uniformly by 50 basis points, orone-half~~ ~~of a percentage point, from levels as of September 30, 2017, the net fair value of our interest-sensitive financial instruments would have resulted in a hypothetical decline of $0.1 million.~~

Foreign currency transaction exposure results primarily from transactions with our contract research organizations, or CROs, and other providers related to our clinical trials that are denominated in currencies other than the functional currency of the legal entity in which the transaction is recorded by us, primarily the Australian dollar. Any transaction gains or losses resulting from currency fluctuations is recorded on a separate line in our condensed consolidated statement of operations. Net foreign currency transaction gains of $0.1 million and less than $0.1 million, were recorded for the nine months ended September 30, 2017 and 2016, respectively.

Currently, our largest foreign currency exposures are those with respect to the Australian dollar. Relative to foreign currency exposures existing as of September 30, 2017, a 10% unfavorable movement in foreign currency exchange rates would expose us to an increased net loss. For the nine months ended September 30, 2017, we estimated that a 10% unfavorable movement in foreign currency exchange rates would have increased our net loss by $0.1 million. This amount is based on a sensitivity analysis performed on our financial position as of September 30, 2017. We have experienced and will continue to experience fluctuations in our net income (loss) as a result of revaluing our assets and liabilities that are not denominated in the functional currency of the entity that recorded the asset or liability. At this time, we do not hedge our foreign currency risk.

~~We maintain disclosure controls and procedures (as defined in Rules13a-15(e)~~ or~~15d-15(e)~~ ~~under~~ the Securities Exchange Act of 1934, as amended or(the "Exchange Act") and are not required to provide the information under this item.

We maintain “disclosure controls and procedures,” as such term is defined in Rules 13a-15(e) and 15d-15(e) under the Exchange ~~Act)~~Act, that are designed to ensure that information required to be disclosed by us in ~~the~~ reports that we file or submit under the Exchange Act is ~~(1)~~ recorded, processed, summarized, and reported within the time periods specified in ~~the SEC’s~~SEC rules and forms, and ~~(2)~~that such information is accumulated and communicated to our management, including our ~~principal executive officer~~Chief Executive Officer and ~~principal financial officer, as appropriate,~~our Chief Financial Officer, to allow timely decisions regarding required disclosure.

~~Based on this evaluation, our management,~~The design of any disclosure controls and procedures also is based in part upon certain assumptions about the likelihood of future events, and there can be no assurance that any design will succeed in achieving its stated goals under all potential future conditions.

With respect to the quarter ended September 30, 2022, under the supervision and with the participation of our ~~principal executive officer~~management, we conducted an evaluation of the effectiveness of the design and ~~principal financial officer, evaluated the effectiveness~~operations of our disclosure controls and ~~procedures as of the end of the period covered by~~procedures. Based upon this ~~report. In designing~~evaluation, our Chief Executive Officer and ~~evaluating~~Chief Financial Officer have concluded that our disclosure controls and procedures are effective.

Management does not expect that our ~~management recognizes that any controls~~internal control over financial reporting will prevent or detect all errors and ~~procedures,~~all fraud. A control system, no matter how well ~~designed~~conceived and operated, can provide only reasonable, not absolute, assurance that the objectives of ~~achieving~~the control systems are met. Further, the design of a control system must reflect the fact that there are resource constraints, and the benefits of controls must be considered relative to their ~~objectives, and management necessarily applies its judgment~~costs. Because of the inherent limitations in ~~evaluating the cost-benefit relationship~~a cost-effective control system, no evaluation of possible controls and procedures. Our principal executive officer and principal financial officer have concluded based upon the evaluation described above that, as of September 30, 2017, our disclosure controls and procedures were effective at the reasonable assurance level.

~~We continue to review and document our disclosure controls and procedures, including our~~ internal ~~controls and procedures for~~control over financial reporting can provide absolute assurance that misstatements due to error or fraud will not occur or that all control issues and ~~may from time to time make changes aimed at enhancing their effectiveness and to ensure that our systems evolve with our business.~~instances of fraud, if any, have been or will be detected.

~~During the three months ended September 30, 2017, there have been~~There were no changes in our internal control over financial reporting (as defined in Rules13a-15(f) or~~15d-15(d)~~ and 15d-15(f) under the Exchange Act) that occurred during the fiscal quarter ended September 30, 2022 which have materially affected, or are reasonably likely to materially affect, our internal control over financial reporting.

~~On October 21, 2015, a purported stockholder of the Company filed a putative class action lawsuit~~From time to time, we are subject to claims in ~~the U.S. District Court for the District of Massachusetts, against the Company and Thomas E. Hughes, captioned Aviad Bessler v. Zafgen, Inc. and Thomas E. Hughes,No. 1:15-cv-13618.~~ ~~An amended complaint was filed on February 22, 2016. The amended complaint alleges violations of~~

~~Sections 10(b) and 20(a) of the Securities Exchange Act of 1934 and SEC Rule10b-5~~ based on allegedly false and misleading statements and omissions regarding our clinical trials for beloranib. On August 9, 2016, the District Court granted the motion to dismiss and dismissed the amended complaint with prejudice. On August 12, 2016, plaintiffs filed a notice of appeal to the First Circuit Court of Appeals and, on April 7, 2017, the dismissal with prejudice was affirmed.

~~In the future, we may become party to~~ legal ~~matters and claims~~proceedings arising in the ~~ordinary~~normal course of ~~business,~~business. To our knowledge, during the ~~resolution~~three months ended September 30, 2022, there were no, and as of ~~which we do not anticipate would~~the date of this Quarterly Report, there are no, threatened or pending legal actions that could reasonably be expected to have a material adverse ~~impact~~effect on our business, financial ~~position,~~condition, results of operations or cash flows.

~~Investing in our common stock involves a high degree of risk.~~ You should carefully consider the risk factors described in our 2021 Annual Report under the caption “Item 1A. Risk Factors.” The risks described ~~below, as well as the other information in this Quarterly Report onForm 10-Q,~~ ~~or Quarterly Report, and~~ in our ~~other public filings before making an investment decision. Our business, prospects, financial condition, or operating results could be harmed by any of these~~2021 Annual Report are not the only risks ~~as well as other~~facing our company. Additional risks and uncertainties not currently known to us or that we currently consider immaterial. If any such risks or uncertainties actually occur, our business, financial condition or operating results could differ materially from the plans, projections and other forward-looking statements included in the section titled “Management’s Discussion and Analysis of Financial Condition and Results of Operations” and elsewhere in this Quarterly Report and in our other public filings. The trading price of our common stock could decline due to any of these risks, and as a result, you may lose all or part of your investment.

~~Risks Related to Product Development, Regulatory Approval and Commercialization~~

~~We currently depend almost entirely on the success of one product candidate,ZGN-1061,~~ ~~which has completed Phase 1 and is currently in Phase 2 clinical development. We cannot be certain that we will be able to obtain regulatory approval forZGN-1061,~~ ~~or successfully commercializeZGN-1061~~ ~~if approved.~~

~~We currently have only one product candidate in clinical stage of development,ZGN-1061,~~ which has completed Phase 1 clinical development in the Netherlands and recently started Phase 2 clinical development, and our business currently depends almost entirely on its successful clinical development, regulatory approval and commercialization. We currently have no drug products for sale and may never be able to develop marketable drug products. In order to conduct clinical trials in the United States we need to file an Investigational New Drug Application, or IND with the U.S. Food and Drug Administration, or FDA. Because our business is almost entirely dependent upon this one product candidate, any setback in our pursuit of regulatory approval for~~ZGN-1061~~ would have a material adverse effect on our business and prospects. The clinical trials of our product candidates are, and the manufacturing and marketing of our product candidates will be, subject to extensive and rigorous review and regulation by numerous government authorities in the United States and in other countries where we intend to test and, if approved, market any product candidate. Before obtaining regulatory approvals for the commercial sale of any product candidate, we must demonstrate through preclinical testing and clinical trials that the product candidate is safe and effective for use in each target indication. This process can take many years and will likely include post-marketing studies, or PMS, post-marketing requirements, or PMRs, and surveillance such as Risk Evaluation and Mitigation Strategies, or REMS, which will require the expenditure of substantial resources beyond the proceeds we currently have on hand.

~~Furthermore, we are not permitted to marketZGN-1061~~ in the United States until we receive approval of a New Drug Application, or NDA, from the FDA, or in any foreign countries until we receive the requisite marketing approval from such countries. Development of diabetes drugs requires approximately 2,500 subjects randomized to active doses of the product with 1,300 to 1,500 subjects exposed for a year and 300 to 500 subjects exposed for 18 months in order to estimate the safety of the drug in an NDA. In addition, it is anticipated that the FDA may require that their guidance for assessment of cardiovascular risk with diabetes products be followed which may require testing of 5,000 to 10,000 subjects. Meeting the requirements of the FDA or certain European regulatory authorities may require that we conduct additional pivotal clinical trials. Accordingly, obtaining approval of an NDA or Marketing Authorization Application, or MAA, is a complex, lengthy, expensive and uncertain process.

~~The FDA and certain European regulatory authorities may delay, limit or deny approval ofZGN-1061~~ ~~for many reasons, including, among others:~~

~~Any of these factors, many of which are beyond our control, could jeopardize our ability to obtain and/or maintain regulatory approval for and successfully marketZGN-1061.~~ Of the large number of drugs in development in the United States, only a small percentage will successfully complete the FDA regulatory approval process and be commercialized. Accordingly, even if we are able to obtain the requisite financing to continue to fund our development and clinical trials, we cannot assure you that~~ZGN-1061~~ ~~or any other of our product candidates will be successfully developed or commercialized.~~

~~Favorable results from preclinical studies and our Phase 1 clinical trial ofZGN-1061~~ ~~are not necessarily predictive of the results of additional preclinical studies or later-stage clinical trials ofZGN-1061.~~ ~~Given the thrombosis findings in humans treated with beloranib, development costs forZGN-1061~~ ~~may be higher and we may be unable to successfully develop, obtain regulatory approval for and commercializeZGN-1061.~~

~~Favorable results from our preclinical studies ofZGN-1061~~ ~~and the Phase 1 clinical trial may not necessarily be predictive of the results from ongoing and later-stage clinical trials. To date we have shown thatZGN-1061~~ ~~has similar potency against the MetAP2 target and similar activity in mouse and rat models of obesity compared to beloranib. Toxicology studies in rats and dogs have shown thatZGN-1061~~ is not exhibiting any testicular safety signals or activation of thrombosis-related biochemical markers, and displays an appreciable margin for embryofetal toxicity, testicular toxicity, prothrombotic effects and other previously observed issues for MetAP2 inhibitors such as hematological and neuronal toxicities with a small therapeutic margin and no margin for embryofetal toxicity. Further in our Phase 1 clinical trial,~~ZGN-1061~~ demonstrated rapid drug absorption and clearance in line with criteria established in advance for the molecule, and has a favorable tolerability profile with no safety signals identified, including no evidence of prothrombotic effects. The data show that~~ZGN-1061~~ treatment causes improvements across multiple metabolic measures consistent with MetAP2 inhibition, and patients in the clinical trial experienced mean weight loss of up to approximately one pound per week. However, we can provide no assurance that the results of our preclinical studies and Phase 1 clinical trial of~~ZGN-1061~~ ~~will be replicated in ongoing or later-stage clinical trials ofZGN-1061~~ or other preclinical studies. Many companies in the pharmaceutical and biotechnology industries have suffered significant setbacks in clinical trials after achieving positive results in preclinical and early-stage clinical development. In particular, we have suffered significant setbacks in later-stage clinical trials of our former lead product candidate, beloranib, after achieving positive results in preclinical and clinical development, and we cannot be certain that we will not face similar setbacks in our development of~~ZGN-1061.~~ The setbacks in later-stage clinical development have been caused by, among other things, preclinical findings made while clinical trials were underway or safety or efficacy observations made in clinical trials, including previously unreported or ununderstood adverse events. Moreover, preclinical and clinical data are often susceptible to varying interpretations and analyses, and many companies that believed their product candidates performed satisfactorily in preclinical studies and clinical trials nonetheless failed to obtain FDA and/or EMA approval. If we fail to produce positive results in our later-stage clinical trials of~~ZGN-1061,~~ the development timeline and regulatory approval and commercialization prospects for our lead product candidate, and, correspondingly, our business and financial prospects, would be materially adversely affected.

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

Undesirable side effects caused by our product candidates could cause us or regulatory authorities such as the FDA to interrupt, delay or halt clinical trials and could result in a more restrictive label or the delay or denial of regulatory approval by the FDA or other regulatory authorities. For example, common adverse events observed in patients treated with our first generation MetAP2 inhibitor, beloranib, versus placebo included diarrhea, injection site bruising, dizziness, decreased appetite, anxiety and sleep disturbances (insomnia principally manifested as delayed onset of sleep and abnormal dreams), among others. In addition, an imbalance in the number of thrombotic events observed in patients treated with beloranib as compared to patients on placebo in our clinical trials was observed. We may see similar adverse events with~~ZGN-1061~~ ~~as we saw with beloranib, and therefore, we will study these parameters in preclinical and clinical development ofZGN-1061.~~ ~~In our Phase 1 clinical trial ofZGN-1061,~~ ~~the most common adverse events reported were mild gastrointestinal issues (comparable betweenZGN-1061~~ ~~and placebo groups), headache and procedural-related irritation.~~

~~Further, ifZGN-1061~~ receives marketing approval and we or others identify undesirable side effects caused by the product (or any other similar product) after the approval, a number of potentially significant negative consequences could result, including:

Any of these events could prevent us from achieving or maintaining market acceptance of the affected product candidate and could substantially increase the costs of commercializing our product candidates and significantly impact our ability to successfully commercialize our product candidates and generate revenues.

~~Failures or delays in the commencement or completion of our planned clinical trials ofZGN-1061~~ ~~could result in increased costs to us and could delay, prevent or limit our ability to generate revenue and continue our business.~~

~~ZGN-1061~~ has completed Phase 1 clinical development in the Netherlands, has recently started Phase 2 clinical development in Australia and New Zealand and will require substantial further clinical development before we can submit an NDA to the FDA or an MAA to the EMA for its marketing approval.

Despite the guidance we may receive from the FDA, the EMA, or other applicable regulatory authorities including Australia and New Zealand, any of these regulatory authorities can change their positions on the acceptability of our clinical trial designs or the clinical endpoints selected, which may require us to complete additional clinical trials or impose stricter approval conditions than we currently expect. Successful completion of such clinical trials is a prerequisite to submitting an NDA to the FDA and an MAA to the EMA and, consequently, the ultimate approval and commercial marketing of~~ZGN-1061.~~ We do not know whether any clinical trials will begin or be completed on schedule, if at all, as the commencement and completion of clinical trials can be delayed or prevented for a number of reasons, including, among others:

Clinical trials may also be delayed or terminated as a result of ambiguous or negative interim results. In addition, a clinical trial may be suspended or terminated by us, the FDA, other regulatory authorities, the IRBsor ethics committees, at the sites where the IRBs or ethics committees are overseeing a clinical trial, a data monitoring committee, or DMC, overseeing the clinical trial at issue or other regulatory authorities due to a number of factors, including, among others:

~~Changes in regulatory requirements, FDA guidance or guidance from EMA, Australia or New Zealand or unanticipated events during our clinical trials ofZGN-1061~~ ~~may occur, which may result in changes to clinical trial protocols or additional clinical trial requirements, which could result in increased costs to us and could delay our development timeline.~~

Changes in regulatory requirements, FDA guidance or guidance from EMA or unanticipated events during our clinical trials may force us to adjust our clinical program. The FDA, the EMA, or the applicable regulatory authorities in Australia and New Zealand may impose additional clinical trial and/or preclinical study requirements. For instance, the FDA issued draft guidance on developing products for weight management in February 2007, and issued draft guidance on developing products for the treatment of diabetes in February 2008 but these guidance documents may be revised at any time. In December 2008, FDA established guidance on evaluating cardiovascular risk of new therapies for the treatment of type 2 diabetes. Amendments to our clinical trial protocols would require resubmission to the FDA, the EMA, or the applicable regulatory authorities in Australia and New Zealand as well as IRBs and ethics committees for review and approval, which may adversely impact the cost, timing or successful completion of a clinical trial. If we experience delays completing, or if we terminate, any of our clinical trials, or if we are required to conduct additional clinical trials and/or preclinical studies, the commercial prospects for~~ZGN-1061~~ ~~may be harmed and our ability to generate product revenue will be delayed.~~

~~We rely, and expect that we will continue to rely, on third parties to conduct any future clinical trials forZGN-1061.~~ ~~If these third parties do not successfully carry out their contractual duties or meet expected deadlines, we may not be able to develop and obtain regulatory approval for or commercializeZGN-1061~~ ~~and our business could be substantially harmed.~~

~~We enter into agreements with third-party CROs to provide monitors for and to manage data for our ongoing clinical trials. We rely heavily on these parties for execution of clinical trials forZGN-1061~~ and control only certain aspects of their activities. As a result, we have less direct control over the conduct, timing and completion of these clinical trials and the management of data developed through the clinical trials than would be the case if we were relying entirely upon our own staff. Communicating with outside parties can also be challenging, potentially leading to mistakes as well as difficulties in coordinating activities. Outside parties may:

These factors may materially adversely affect the willingness or ability of third parties to conduct our clinical trials and may subject us to unexpected cost increases that are beyond our control. Nevertheless, we are responsible for ensuring that each of our clinical trials is conducted in accordance with the applicable protocol, legal and regulatory requirements and scientific standards, and our reliance on CROs does not relieve us of our regulatory responsibilities. We and our CROs are required to comply with requirements for Good Clinical Practice, or GCPs, which are legal requirements enforced by the FDA, the Competent Authorities of the Member States of the European Economic Area and comparable foreign regulatory authorities for any products in clinical development. The FDA enforces these GCP regulations through periodic inspections of clinical trial sponsors, principal investigators and clinical trial sites, IRBs, and other vendors that may be involved in the clinical development of new products. If we or our investigators or CROs fail to comply with applicable GCPs, the clinical data generated in our clinical trials may be deemed unreliable and the FDA or comparable foreign regulatory authorities may require us to perform additional clinical trials before approving our marketing applications. We cannot assure you that, upon inspection, the FDA will determine that any of our clinical trials comply with GCPs. In addition, our clinical trials must be conducted with products produced under current Good Manufacturing Practices, or cGMPs’ regulations, to assure the identity, strength, quality, and purity of our drug product candidates being used in the clinical trials, as well as the~~to-be-marketed~~ formulation and product. Our failure or the failure of our CROs and/or contract manufacturing organizations, or CMOs, to comply with these regulations may require us to repeat clinical trials, which would delay the regulatory approval process and could also subject us to enforcement action, up to and including, civil and criminal penalties.

~~Although we design our clinical trials forZGN-1061,~~ investigators and CROs conduct all of the clinical trials. As a result, many important aspects of our drug development programs are outside of our direct control. In addition, the investigators or CROs may not perform all of their obligations under arrangements with us or in compliance with regulatory requirements, but we remain responsible and are subject to enforcement action that may include civil penalties up to and including criminal prosecution for any violations of FDA laws and regulations during the conduct of our clinical trials. If the investigators or CROs do not perform clinical trials in a satisfactory manner, breach their obligations to us, or fail to comply with regulatory requirements, the development and commercialization of~~ZGN-1061~~ ~~may be delayed or our development program materially and irreversibly harmed. We cannot control the amount and timing of resources these investigators or CROs devote to our program orZGN-1061.~~ If we are unable to rely on clinical data collected by our investigators or CROs, we could be required to repeat, extend the duration of, or increase the size of our clinical trials and this could significantly delay commercialization and require significantly greater expenditures.

If any of our relationships with these third-party investigators or CROs terminate, we may not be able to enter into arrangements with alternative investigators or CROs in a timely manner, or at all. If investigators or CROs do not successfully carry out their contractual duties or obligations or meet expected deadlines, if they needdeem to be replaced or if the quality or accuracy of the clinical data they obtain is compromised due to the failure to adhere to our clinical protocols, regulatory requirements or for other reasons, any such clinical trialsimmaterial also may ~~be extended, delayed or terminated, and we may not be able to obtain regulatory approval for or successfully commercializeZGN-1061.~~ ~~As a result, our financial results and the commercial prospects forZGN-1061~~ ~~in the subject indication would be harmed, our costs could increase and our ability to generate revenue could be delayed.~~

~~We rely completely on third-party suppliers to manufacture our clinical drug supplies forZGN-1061,~~ ~~and we intend to rely on third parties to produce commercial supplies ofZGN-1061~~ ~~and preclinical, clinical and commercial supplies of any future product candidate.~~

~~We do not currently have, nor do we currently plan to acquire, the infrastructure or capability to internally manufacture our clinical drug supply ofZGN-1061,~~ or any future product candidates, for use in the conduct of our preclinical studies and clinical trials, and we lack the internal resources and the capability to manufacture any product candidates on a clinical or commercial scale. The CMOs used to manufacture the active drug substance and final drug product must be approved by our quality assurance unit and inspected by the FDA and other comparable foreign regulatory agencies.

We rely on our CMOs to comply with cGMPs for manufacture of raw materials, active drug substance and finished drug products. If our CMOs cannot successfully manufacture material that conforms to our specifications and the regulatory requirements of the FDA or applicable foreign regulatory agencies, the CMOs will not be able to secure and/or maintain regulatory approval for their manufacturing facilities. While we manage our quality expectations through an audit program for our vendors and suppliers, we have no direct control over our CMOs’ ability to maintain adequate quality control, quality assurance and qualified personnel. Furthermore, our CMOs are engaged with third party vendors to supply and/or manufacture starting materials or components for them, which exposes our CMOs to regulatory risks for the production of such materials and components. As a result, failure to satisfy the regulatory requirements for the production of those materials and components may affect the regulatory clearance of our CMOs’ facilities generally. If the FDA or an applicable foreign regulatory agency does not approve these facilities for the manufacture of our product candidates or if it withdraws its approval in the future, we may need to find alternative manufacturing facilities, which would adversely impact our ability to develop, obtain regulatory approval for or market our product candidates.

~~We rely completely on third-party suppliers to manufacture our preclinical and clinical drug supplies forZGN-1061.~~ ~~Currently each batch ofZGN-1061~~ is individually contracted under a work order, which is governed by a quality and service agreement. The current drug substance manufacturing process will support preclinical studies and early clinical trials and will be further optimized to support advanced clinical development and commercialization. Current drug substance, including key starting material, in inventory, is expected to support Phase 2 clinical trials. A new formulation with longer shelf life has been developed and manufactured to support Phase 2 clinical development.

~~Even if we receive marketing approval forZGN-1061~~ ~~in the United States, we may never receive regulatory approval to marketZGN-1061~~ ~~outside of the United States.~~

~~We intend to pursue marketing approval ofZGN-1061~~ in the United States, the European Union and in other countries worldwide. In order to market any product outside of the United States, we must establish and comply with the numerous and varying safety, efficacy and other regulatory requirements of other countries, including potential additional clinical trials and/or preclinical studies. Approval procedures vary among countries and can involve additional product candidate testing and additional administrative review periods. The time required to obtain approvals in other countries might differ from that required to obtain FDA approval. The marketing approval processes in other countries may implicate all of the risks detailed above regarding FDA approval in the United States as well as other risks. In particular, in many countries outside of the United States, products must receive pricing and reimbursement approval before the product can be commercialized. Obtaining this approval can result in substantial delays in bringing products to market in such countries. In addition, on March 20, 2017, the United Kingdom government started the process to leave the European Union by April 2019, or Brexit. The effects of Brexit will depend on any agreements the United Kingdom makes to retain access to European Union markets either during a transitional period or more permanently. Brexit could lead to legal uncertainty and potentially divergent national laws and regulation as the United Kingdom determines which European Union laws to replace or replicate. Marketing approval in one country does not necessarily ensure marketing approval in another, but a failure or delay in obtaining marketing approval in one country may have a negative effect on the regulatory process or commercial activities in others. Failure to obtain marketing approval in other countries or any delay or other setback in obtaining such approval would impair our ability to market~~ZGN-1061~~ ~~in such foreign markets. Any such impairment would reduce the size of our potential market, which could have a material adverse impact on our business, results of operations and prospects.~~

~~Even if we receive marketing approval forZGN-1061,~~ ~~it may not achieve broad market acceptance, which would limit the revenue that we generate from its sales.~~

~~The commercial success ofZGN-1061,~~ ~~if developed and approved for marketing by the FDA or EMA or other applicable regulatory authorities, will depend upon the awareness and acceptance ofZGN-1061~~ ~~among the medical community, including physicians, patients, advocacy groups and healthcare payors. Market acceptance ofZGN-1061,~~ ~~if approved, will depend on a number of factors, including, among others:~~

If~~ZGN-1061~~ ~~is approved but does not achieve an adequate level of acceptance by patients, advocacy groups, physicians and payors, we may not generate sufficient revenue fromZGN-1061~~ ~~to become or remain profitable. Before granting reimbursement approval, healthcare payors may require us to demonstrate that, in addition to treating type 2 diabetes in patients,ZGN-1061~~ ~~also provides incremental health benefits to patients. Our efforts to educate the medical community and third-party payors about the benefits ofZGN-1061~~ ~~may require significant resources and may never be successful.~~

~~If we are unable to establish sales and marketing capabilities or enter into agreements with third parties to market and sellZGN-1061,~~ ~~we may not be able to generate any revenue.~~

~~We do not currently have an established infrastructure for the sales, marketing and distribution of pharmaceutical products. In order to marketZGN-1061,~~ ~~if approved by the FDA or any other regulatory body, we must build our sales, marketing, managerial and othernon-technical~~ capabilities or make arrangements with third parties to perform these services. If we are unable to establish adequate sales, marketing and distribution capabilities, whether independently or with third parties, or if we are unable to do so on commercially reasonable terms, our business, results of operations, financial condition and prospects will be materially adversely affected.

~~Even if we receive marketing approval forZGN-1061,~~ ~~we may still face future development and regulatory difficulties.~~

~~Even if we receive marketing approval forZGN-1061,~~ ~~regulatory authorities may still impose significant restrictions onZGN-1061’s~~ ~~indicated uses or marketing or impose ongoing requirements for potentially costly post-approval studies.ZGN-1061~~ will also be subject to ongoing FDA and EMA requirements governing the labeling, packaging, storage and promotion of the product and recordkeeping and submission of safety and other post-market information. The FDA has significant post-market authority, including, for example, the authority to require labeling changes based on new safety information and to require post-market studies or clinical trials to evaluate serious safety risks related to the use of a drug. The FDA also has the authority to require, as part of an NDA or post-approval, the submission of a REMS. Any REMS required by the FDA may lead to increased costs to assure compliance with new post-approval regulatory requirements and potential requirements or restrictions on the sale of approved products, all of which could lead to lower sales volume and revenue. Additionally, the FDA may require a PMS and/or PMRs, that could represent and result in additional restrictions and/or limitations for the product.

Manufacturers of drug products and their facilities are subject to continual review and periodic inspections by the FDA and other regulatory authorities for compliance with cGMPs and other regulations. If we or a regulatory agency discover problems with~~ZGN-1061,~~ ~~such as adverse events of unanticipated severity or frequency, or problems with the facility whereZGN-1061~~ ~~is manufactured, a regulatory agency may impose restrictions onZGN-1061,~~ ~~the manufacturer or us, including requiring withdrawal ofZGN-1061~~ ~~from the market or suspension of manufacturing. If we or the manufacturing facilities forZGN-1061~~ ~~fail to comply with applicable regulatory requirements, a regulatory agency may, among other things:~~

~~Competing technologies could emerge, including devices and surgical procedures, adversely affecting our opportunity to generate revenue from the sale ofZGN-1061.~~

The biotechnology and pharmaceutical industries are intensely competitive and subject to rapid and significant technological change. We have competitors in a number of jurisdictions, many of which have substantially greater name recognition, commercial infrastructures and financial, technical and personnel resources than we have. Established competitors may invest heavily to quickly discover and develop novel compounds that could make~~ZGN-1061~~ obsolete or uneconomical. Any new product that competes with an approved product may need to demonstrate compelling advantages in efficacy, convenience, tolerability and safety to be commercially successful. Other competitive factors, including generic competition, could force us to lower prices or could result in reduced sales. In addition, new products developed by others could emerge as competitors to~~ZGN-1061.~~ ~~If we are not able to compete effectively against our current and future competitors, our business will not grow and our financial condition and operations will suffer.~~

~~Our future growth depends, in part, on our ability to penetrate foreign markets, where we would be subject to additional regulatory burdens and other risks and uncertainties.~~

~~Our future profitability will depend, in part, on our ability to commercializeZGN-1061~~ ~~in foreign markets for which we may rely on collaborations with third parties. If we commercializeZGN-1061~~ ~~in foreign markets, we would be subject to additional risks and uncertainties, including:~~

~~Foreign sales ofZGN-1061~~ ~~could also be adversely affected by the imposition of governmental controls, political and economic instability, trade restrictions and changes in tariffs.~~

~~We are subject to healthcare laws and regulations, which could expose us to criminal sanctions, civil penalties, contractual damages, reputational harm and diminished profits and future earnings.~~

~~Healthcare providers, physicians and others will play a primary role in the recommendation and prescription ofZGN-1061,~~ if approved. Our future arrangements with third-party payors will expose us to broadly applicable fraud and abuse and other healthcare laws and regulations that may constrain the business or financial arrangements and relationships through which we market, sell and distribute~~ZGN-1061,~~ ~~if we obtain marketing approval. Restrictions under applicable federal and state healthcare laws and regulations include the following:~~

Ensuring that our future business arrangements with third parties comply with applicable healthcare laws and regulations could be costly. It is possible that governmental authorities will conclude that our business practices do not comply with current or future statutes, regulations or case law involving applicable fraud and abuse or other healthcare laws and regulations. If our operations, including anticipated activities to be conducted by our sales team, were found to be in violation of any of these laws or any other governmental regulations that may apply to us, we may be subject to significant civil, criminal and administrative penalties, damages, fines and exclusion from government funded healthcare programs, such as Medicare and Medicaid, any of which could substantially disrupt our operations. If any of the physicians or other providers or entities with whom we expect to do business is found not to be in compliance with applicable laws, they may be subject to criminal, civil or administrative sanctions, including exclusions from government funded healthcare programs.

~~The FDA and other regulatory agencies actively enforce the laws and regulations prohibiting the promotion ofoff-label~~ ~~uses. If we are found to have improperly promotedoff-label~~ ~~uses, we may become subject to significant liability.~~

~~The FDA and other regulatory agencies strictly regulate the promotional claims that may be made about prescription products, such asZGN-1061,~~ if approved. In particular, a product may not be promoted for uses that are not approved by the FDA or such other regulatory agencies as reflected in the product’s approved labeling. If we receive marketing approval for~~ZGN-1061,~~ ~~physicians may nevertheless prescribeZGN-1061~~ ~~to their patients in a manner that is inconsistent with the approved label. If we are found to have promoted suchoff-label~~ uses, we may become subject to significant liability. The federal government has levied large civil and criminal fines against companies for alleged improper promotion and has enjoined several companies from engaging in~~off-label~~ promotion and required that they enter into corporate integrity agreements with the Office of Inspector General of the Department of Health and Human Services, or OIG. The FDA has also requested that companies enter into consent decrees or permanent injunctions under which specified promotional conduct is changed or curtailed. If we cannot successfully manage the promotion of~~ZGN-1061,~~ ~~if approved, we could become subject to significant liability, which would materially adversely affect our business and financial condition.~~

~~Even if approved, reimbursement policies could limit our ability to sellZGN-1061.~~

~~If approved by regulatory authorities, market acceptance and sales ofZGN-1061~~ will depend on reimbursement policies and may be affected by healthcare reform measures. Government authorities and third-party payors, such as private health insurers and health maintenance organizations, decide which medications they will pay for and establish reimbursement levels for those medications. Cost containment is a primary concern in the U.S. healthcare industry and elsewhere. Government authorities and these third-party payors have attempted to control costs by limiting coverage and the amount of reimbursement for particular medications. We cannot be sure that reimbursement will be available for~~ZGN-1061~~ ~~and, if reimbursement is available, the level of such reimbursement. Reimbursement may impact the demand for, or the price of,ZGN-1061.~~ ~~If reimbursement is not available or is available only at limited levels, we may not be able to successfully commercializeZGN-1061.~~

In some foreign countries, particularly in Canada and European countries, the pricing of prescription pharmaceuticals is subject to strict governmental control. In these countries, pricing negotiations with governmental authorities can take six to 12 months or longer after the receipt of regulatory approval and product launch. To obtain favorable reimbursement for the indications sought or pricing approval in some countries, we may be required to conduct a clinical trial that compares the cost-effectiveness of~~ZGN-1061~~ ~~with other available therapies. If reimbursement forZGN-1061~~ is unavailable in any country in which we seek reimbursement, if it is limited in scope or amount, if it is conditioned upon our completion of additional clinical trials, or if pricing is set at unsatisfactory levels, our operating results could be materially adversely affected.

~~Our development programs for our product candidates, which are primarily related toZGN-1061,~~ ~~may require substantial financial resources and may ultimately be unsuccessful.~~

~~Our lead product candidateZGN-1061~~ has completed Phase 1 clinical development and is currently in Phase 2 clinical development, and there are a number of FDA and certain European regulatory requirements that we must satisfy before we can commence late-stage clinical trials of~~ZGN-1061.~~ Satisfaction of these requirements will entail substantial time, effort and financial resources. We may never satisfy these requirements. We believe that our cash, cash equivalents and marketable securities will be sufficient to fund operations for a period of at least one year from the issuance date of this Quarterly Report, but we will need to raise more funds to continue development and commercialization of~~ZGN-1061~~ and our other product candidates, which may not be easily available. Furthermore, any time, effort and financial resources we expend on our other early-stage development programs may adversely affect our ability to continue development and commercialization of~~ZGN-1061,~~ and we may never commence clinical trials of such development programs despite expending significant resources in pursuit of their development. If we do commence clinical trials of our other potential product candidates, such product candidates may never be approved by the FDA or other regulatory authorities.

~~If we are unable to adequately protect our proprietary technology or maintain issued patents which are sufficient to protectZGN-1061~~ ~~or future product candidates, others could compete against us more directly, which would have a material adverse impact on our business, results of operations, financial condition and prospects.~~

Our commercial success will depend in part on our success in obtaining and maintaining issued patents and other intellectual property rights in the United States and elsewhere and protecting our proprietary technology. If we do not adequately protect our intellectual property and proprietary technology, competitors may be able to use our technologies and erode or negate any competitive advantage we may have, which could harm our business and ability to achieve profitability. Our owned patent application relates to compositions of matter and methods of use of~~ZGN-1061.~~

As of October 31, 2017, we own one issued U.S patent, one pending U.S. patent application, one pending Patent Cooperation Treaty, or PCT, patent application, and two pending U.S. provisional patent applications that relate to~~ZGN-1061.~~

As of October 31, 2017, we own 15 issued U.S. patents, and 9 pending U.S. patent applications with pending foreign counterpart applications, all of which relate to our internal efforts to discover novel MetAP2 inhibitors.

~~We cannot provide any assurances that any of our pending patent applications that mature into issued patents will include claims with a scope sufficient to protectZGN-1061~~ and our other product candidates. Other parties have developed technologies that may be related or competitive to our approach, and may have filed or may file patent applications and may have received or may receive patents that may overlap or conflict with our patent applications, either by claiming the same methods or formulations or by claiming subject matter that could dominate our patent position. The patent positions of biotechnology and pharmaceutical companies, including our patent position, involve complex legal and factual questions, and, therefore, the issuance, scope, validity and enforceability of any patent claims that we may obtain cannot be predicted with certainty. Patents, if issued, may be challenged, deemed unenforceable, invalidated, or circumvented. U.S. patents and patent applications may also be subject to interference proceedings, ~~ex parte~~ ~~reexamination, or~~ ~~inter partes~~ ~~review proceedings, supplemental examination and challenges in district court.~~

Patents may be subjected to opposition, post-grant review, or comparable proceedings lodged in various foreign, both national and regional, patent offices. These proceedings could result in either loss of the patent or denial of the patent application or loss or reduction in the scope of one or more of the claims of the patent or patent application. In addition, such proceedings may be costly. Thus, any patents that we may own or exclusively license may not provide any protection against competitors. Furthermore, an adverse decision in an interference proceeding can result in a third party receiving the patent right sought by us, which in turn could affect our ability to develop, market or otherwise commercialize~~ZGN-1061~~ ~~and our other product candidates.~~

Furthermore, though an issued patent is presumed valid and enforceable, its issuance is not conclusive as to its validity or its enforceability and it may not provide us with adequate proprietary protection or competitive advantages against competitors with similar products. Competitors may also be able to design around our patents. Other parties may develop and obtain patent protection for more effective technologies, designs or methods. The laws of some foreign countries do not protect our proprietary rights to the same extent as the laws of the United States, and we may encounter significant problems in protecting our proprietary rights in these countries. If these developments were to occur, they could have a material adverse effect on our potential future sales.

Our ability to enforce our patent rights depends on our ability to detect infringement. It is difficult to detect infringers who do not advertise the components that are used in their products. Moreover, it may be difficult or impossible to obtain evidence of infringement in a competitor’s or potential competitor’s product. Any litigation to enforce or defend our patent rights, even if we were to prevail, could be costly and time-consuming and would divert the attention of our management and key personnel from our business operations. We may not prevail in any lawsuits that we initiate and the damages or other remedies awarded if we were to prevail may not be commercially meaningful.

In addition, proceedings to enforce or defend our patents could put our patents at risk of being invalidated, held unenforceable, or interpreted narrowly. Such proceedings could also provoke third parties to assert claims against us, including that some or all of the claims in one or more of our patents are invalid or otherwise unenforceable. If any of our patents covering~~ZGN-1061~~ are invalidated or found unenforceable, our financial position and results of operations would be materially and adversely impacted. In addition, if a court found that valid, enforceable patents held by third parties covered~~ZGN-1061,~~ ~~our financial position and results of operations would also be materially and adversely impacted.~~

~~The degree of future protection for our proprietary rights is uncertain, and we cannot ensure that:~~

~~We rely upon unpatented trade secrets, unpatentedknow-how~~ and continuing technological innovation to develop and maintain our competitive position, which we seek to protect, in part, by confidentiality agreements with our employees and our collaborators and consultants. We also have agreements with our employees and selected consultants that obligate them to assign their inventions to us and have~~non-compete~~ agreements with some, but not all, of our consultants. It is possible that technology relevant to our business will be independently developed by a person that is not a party to such an agreement. Furthermore, if the employees and consultants who are parties to these agreements breach or violate the terms of these agreements, we may not have adequate remedies for any such breach or violation, and we could lose our trade secrets through such breaches or violations. Further, our trade secrets could otherwise become known or be independently discovered by our competitors.

~~We may infringe the intellectual property rights of others, which may prevent or delay our product development efforts and stop us from commercializing or increase the costs of commercializingZGN-1061,~~ ~~if approved.~~

Our success will depend in part on our ability to operate without infringing the intellectual property and proprietary rights of third parties. We cannot assure you that our business, products and methods do not or will not infringe the patents or other intellectual property rights of third parties.

~~The pharmaceutical industry is characterized by extensive litigation regarding patents and other intellectual property rights. Other parties may allege thatZGN-1061~~ or the use of our technologies infringes patent claims or other intellectual property rights held by them or that we are employing their proprietary technology without authorization. Patent and other types of intellectual property litigation can involve complex factual and legal questions, and their outcome is uncertain. Any claim relating to intellectual property infringement that is successfully asserted against us may require us to pay substantial damages, including treble damages and attorneys’ fees if we are found to be willfully infringing another party’s patents, for past use of the asserted intellectual property and royalties and other consideration going forward if we are forced to take a license. In addition, if any such claim were successfully asserted against us and we could not obtain such a license, we may be forced to stop or delay developing, manufacturing, selling or otherwise commercializing~~ZGN-1061.~~

Even if we are successful in these proceedings, we may incur substantial costs and divert management time and attention in pursuing these proceedings, which could have a material adverse effect on us. If we are unable to avoid infringing the patent rights of others, we may be required to seek a license, defend an infringement action or challenge the validity of the patents in court, or redesign our products. Patent litigation is costly and time consuming. We may not have sufficient resources to bring these actions to a successful conclusion. In addition, intellectual property litigation or claims could force us to do one or more of the following:

~~Any of these risks coming to fruition could have a material adverse effect on our business, results of operations, financial condition and prospects.~~

~~We may be subject to claims challenging the inventorship or ownership of our patents and other intellectual property.~~

We may also be subject to claims that former employees, collaborators or other third parties have an ownership interest in our patents or other intellectual property. Litigation may be necessary to defend against these and other claims challenging inventorship or ownership. If we fail in defending any such claims, in addition to paying monetary damages, we may lose valuable intellectual property rights, such as exclusive ownership of, or right to use, valuable intellectual property. Such an outcome could have a material adverse effect on our business. Even if we are successful in defending against such claims, litigation could result in substantial costs and be a distraction to management and other employees.

Obtaining and maintaining our patent protection depends on compliance with various procedural, document submission, fee payment and other requirements imposed by governmental patent agencies, and our patent protection could be reduced or eliminated for~~non-compliance~~ ~~with these requirements.~~

The U.S. Patent and Trademark Office, or U.S. PTO, and various foreign governmental patent agencies require compliance with a number of procedural, documentary, fee payment and other provisions during the patent process. There are situations in which noncompliance can result in abandonment or lapse of a patent or patent application, resulting in partial or complete loss of patent rights in the relevant jurisdiction. In such an event, competitors might be able to enter the market earlier than would otherwise have been the case.

~~We may be involved in lawsuits to protect or enforce our patents or the patents of our licensors, which could be expensive, time-consuming and unsuccessful.~~

Competitors may infringe our patents or the patents of our licensors. To counter infringement or unauthorized use, we may be required to file infringement claims, which can be expensive and time-consuming. In addition, in an infringement proceeding, a court may decide that a patent of ours or our licensors is not valid, is unenforceable and/or is not infringed, or may refuse to stop the other party from using the technology at issue on the grounds that our patents do not cover the technology in question. An adverse result in any litigation or defense proceedings could put one or more of our patents at risk of being invalidated or interpreted narrowly and could put our patent applications at risk of not issuing.

Interference proceedings provoked by third parties or brought by us may be necessary to determine the priority of inventions with respect to our patents or patent applications or those of our licensors. An unfavorable outcome could require us to cease using the related technology or to attempt to license rights to it from the prevailing party. Our business could be harmed if the prevailing party does not offer us a license on commercially reasonable terms. Our defense of litigation or interference proceedings may fail and, even if successful, may result in substantial costs and distract our management and other employees. We may not be able to prevent, alone or with our licensors, misappropriation of our intellectual property rights, particularly in countries where the laws may not protect those rights as fully as in the United States.

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

~~Issued patents covering our product candidates could be found invalid or unenforceable if challenged in court.~~

If we or one of our licensing partners initiated legal proceedings against a third party to enforce a patent covering our product candidate, the defendant could counterclaim that the patent covering our product candidate is invalid and/or unenforceable. In patent litigation in the United States, defendant counterclaims alleging invalidity and/or unenforceability are commonplace. Grounds for a validity challenge include alleged failures to meet any of several statutory requirements, including lack of novelty, obviousness or~~non-enablement.~~ Grounds for unenforceability assertions include allegations that someone connected with prosecution of the patent withheld relevant information from the U.S. PTO, or made a misleading statement, during prosecution. Third parties may also raise similar claims before administrative bodies in the United States or abroad, even outside the context of litigation. Such mechanisms include~~re-examination,~~ post grant review and equivalent proceedings in foreign jurisdictions, e.g., opposition proceedings. Such proceedings could result in revocation or amendment of our patents in such a way that they no longer cover our product candidates or competitive products. The outcome following legal assertions of invalidity and unenforceability is unpredictable. With respect to validity, for example, we cannot be certain that there is no invalidating prior act, of which we and the patent examiner were unaware during prosecution. If a defendant were to prevail on a legal assertion of invalidity and/or unenforceability, we would lose at least part, and perhaps all, of the patent protection on our product candidates. Such a loss of patent protection would have a material adverse impact on our business.

We do not seek to protect our intellectual property rights in all jurisdictions throughout the world and we may not be able to adequately enforce our intellectual property rights even in the jurisdictions where we seek protection.

Filing, prosecuting and defending patents on product candidates in all countries and jurisdictions throughout the world would be prohibitively expensive, and our intellectual property rights in some countries outside the United States could be less extensive than those in the United States. In addition, the laws of some foreign countries do not protect intellectual property rights to the same extent as federal and state laws in the United States. Consequently, we may not be able to prevent third parties from practicing our inventions in all countries outside the United States, or from selling or importing products made using our inventions in and into the United States or other jurisdictions. Competitors may use our technologies in jurisdictions where we have not obtained patent protection to develop their own products and further, may export otherwise infringing products to territories where we have patent protection, but enforcement is not as strong as that in the United States. These products may compete with our products and our patents or other intellectual property rights may not be effective or sufficient to prevent them from competing.

Many companies have encountered significant problems in protecting and defending intellectual property rights in foreign jurisdictions. The legal systems of certain countries, particularly certain developing countries, do not favor the enforcement of patents and other intellectual property protection, particularly those relating to biopharmaceuticals, which could make it difficult for us to stop the infringement of our patents or marketing of competing products in violation of our proprietary rights generally. For example, an April 2014 report from the Office of the United States Trade Representative identified a number of countries, including India and China, where challenges to the procurement and enforcement of patent rights have been reported. Several countries, including India and China, have been listed in the report every year since 1989. Proceedings to enforce our patent rights in foreign jurisdictions could result in substantial costs and divert our efforts and attention from other aspects of our business, could put our patents at risk of being invalidated or interpreted narrowly, could put our patent applications at risk of not issuing and could provoke third parties to assert claims against us. We may not prevail in any lawsuits that we initiate and the damages or other remedies awarded, if any, may not be commercially meaningful. Accordingly, our efforts to enforce our intellectual property rights around the world may be inadequate to obtain a significant commercial advantage from the intellectual property that we develop or license.

We are dependent on licensed intellectual property for certain early-stage product candidates. If we were to lose our rights to licensed intellectual property, we may not be able to continue developing or commercializing such product candidates, if approved.

We have an exclusive license with Children’s Medical Center Corporation, pursuant to which we exclusively licensed certain patent rights relating to decreasing the growth of fat tissue, on a worldwide basis. We may enter into additional licenses to third-party intellectual property that are necessary or useful to our business. Current or future licensors may also allege that we have breached our license agreement and may accordingly seek to terminate our license with them. In addition, current or future licensors may decide to terminate our license at will. If successful, this could result in our loss of the right to use the licensed intellectual property, which could materially adversely affect our ability to develop and commercialize a product candidate or product, if approved, as well as harm our competitive business position and our business prospects.

~~We have not yet registered trademarks for a commercial trade name forZGN-1061~~ ~~and failure to secure such registrations could adversely affect our business.~~

~~We have not yet registered trademarks for a commercial trade name forZGN-1061.~~ Any future trademark applications may be rejected during trademark registration proceedings. Although we would be given an opportunity to respond to those rejections, we may be unable to overcome such rejections. In addition, in the U.S. PTO and in comparable agencies in many foreign jurisdictions, third parties are given an opportunity to oppose pending trademark applications and to seek to cancel registered trademarks. Opposition or cancellation proceedings may be filed against our trademarks, and our trademarks may not survive such proceedings. Moreover, any name we propose to use with our product candidates in the United States must be approved by the FDA, regardless of whether we have registered it, or applied to register it, as a trademark. The FDA typically conducts a review of proposed product names, including an evaluation of potential for confusion with other product names. If the FDA objects to any of our proposed proprietary product names, we may be required to expend significant additional resources in an effort to identify a suitable substitute name that would qualify under applicable trademark laws, not infringe the existing rights of third parties and be acceptable to the FDA.

~~If we do not obtain additional protection under the Hatch-Waxman Amendments and similar foreign legislation by extending the patent terms and obtaining data exclusivity forZGN-1061,~~ ~~our business may be materially harmed.~~

~~Depending upon the timing, duration and specifics of development and FDA marketing approval ofZGN-1061,~~ one or more of our U.S. patents may be eligible for limited patent term restoration under the Drug Price Competition and Patent Term Restoration Act of 1984, referred to as the Hatch-Waxman Amendments. The Hatch-Waxman Amendments permit a patent restoration term of up to five years as compensation for patent term lost during product development and the FDA regulatory review process. However, we may not be granted an extension because of, for example, failing to apply within applicable deadlines, failing to apply prior to expiration of relevant patents or otherwise failing to satisfy applicable requirements. Moreover, the applicable time period or the scope of patent protection afforded could be less than we request. If we are unable to obtain patent term extension or restoration or the term of any such extension is less than we request, our competitors may obtain approval of competing products following our patent expiration, and our ability to generate revenues could be materially adversely affected.

~~Changes in U.S. patent law could diminish the value of patents in general, thereby impairing our ability to protect our products.~~

The United States has recently enacted and is currently implementing the America Invents Act of 2011, which is wide-ranging patent reform legislation. Further, the U.S. Supreme Court has ruled on several patent cases in recent years, either narrowing the scope of patent protection available in certain circumstances or weakening the rights of patent owners in certain situations. In addition to increasing uncertainty with regard to our ability to obtain future patents, this combination of events has created uncertainty with respect to the value of patents, once obtained. Depending on decisions by the U.S. Congress, the federal courts and the U.S. PTO, the laws and regulations governing patents could change in unpredictable ways that would weaken our ability to obtain new patents or to enforce our existing patents or future patents.

~~We may be subject to damages resulting from claims that we or our employees have wrongfully used or disclosed alleged trade secrets of their former employers.~~

Our employees have been previously employed at other biotechnology or pharmaceutical companies, including our competitors or potential competitors. Although we are not aware of any claims currently pending against us, we may be subject to claims that these employees or we have inadvertently or otherwise used or disclosed trade secrets or other proprietary information of the former employers of our employees. Litigation may be necessary to defend against these claims. Even if we are successful in defending against these claims, litigation could result in substantial costs and be a distraction to management. If we fail in defending such claims, in addition to paying money claims, we may lose valuable intellectual property rights or personnel. A loss of key personnel or their work product could hamper or prevent our ability to develop and commercialize~~ZGN-1061~~ ~~and other product candidates, which would~~ materially adversely affect our business, financial condition or future results.

Item 2. Unregistered Sales of Equity Securities and ~~results~~Use of ~~operations.~~

On November 10, 2022, we reduced the size of our organization, and we may encounter difficulties in managing this development and restructuring, which could disrupt our operations. In addition, we may not achieve anticipated benefits and savings from the reduction.

In July 2016, our board of directors approved the suspension of further development of beloranib and a restructuring plan, pursuant to which our workforce was reduced by approximately 31% as of December 2016. The workforce reduction resulted in the loss of longer-term employees, the loss of institutional knowledge and expertise and the reallocation and combination of certain roles and responsibilities across the organization, all of which could adversely affect our operations. Given the complexity of our business, we must continue to implement and improve our managerial, operational and financial systems, manage our facilities and continue to recruit and retain qualified personnel. This will be made more challenging given the workforce reduction described above. As a result, our management may need to divert a disproportionate amount of its attention away from our~~day-to-day~~ activities, and devote a substantial amount of time to managing these activities. Further, the restructuring and possible additional cost containment measures may yield unintended consequences, such as attrition beyond our intended workforce reduction and reduced employee morale. In addition, we may not achieve anticipated benefits from the workforce reduction. Due to our limited resources, we may not be able to effectively manage our operations or recruit and retain qualified personnel, which may result in weaknesses in our infrastructure and operations, risks that we may not be able to comply with legal and regulatory requirements, and loss of employees and reduced productivity among remaining employees. For example, the workforce reduction may negatively impact our clinical and regulatory functions, which would have a negative impact on our ability to successfully develop, and ultimately, commercialize~~ZGN-1061. If~~ our management is unable to effectively manage this transition and workforce reduction and additional cost containment measures, our expenses may be more than expected and we may not be able to implement our business strategy. As a result, our future financial performance and our ability to commercialize~~ZGN-1061~~ ~~successfully would be negatively affected.~~

Our future success depends on our ability to retain our executive officers, and particularly our current Chief Executive Officer, President and Chief Scientific Officer, and to attract, retain and motivate qualified personnel.

~~We are highly dependent on Mr. Jeffrey Hatfield, our Chief Executive Officer and Dr. Thomas E. Hughes, our President and Chief Scientific Officer. We have~~ entered into ~~a severance and change in control agreement~~the ATM Agreement with each of Mr. Hatfield and Dr. Hughes, but either executive may terminate his employment with us at any time. Although we do not have any reason to believe that we will lose the services of Mr. Hatfield or Dr. Hughes in the foreseeable future, the loss of either executive’s services might impede the achievement of our research, development and commercialization objectives. We also do not have any~~key-man~~ ~~life insurance on Mr. Hatfield or Dr. Hughes.~~

Our success also depends upon the principal members of our executive, medical and development teams. We have entered into a severance and change in control agreement with our executive officers and department vice president level employees, but they may terminate their employment with us at any time. The loss of the services of any of these persons might impede the achievement of our development and commercialization objectives.

~~With any change in leadership, there is also a risk to retention of employees, as well as the potential for disruption to business operations, initiatives, plans and strategies.~~

We rely on consultants and advisors, including scientific and clinical advisors, to assist us in formulating our development and commercialization strategy. Our consultants and advisors may be employed by employers other than us and may have commitments under consulting or advisory contracts with other entities that may limit their availability to us and may not be subject to our standard~~non-compete~~ agreements. Recruiting and retaining qualified scientific personnel and sales and marketing personnel will also be critical to our success. We may not be able to attract and retain these personnel on acceptable terms given the workforce reduction and competition among numerous pharmaceutical and biotechnology companies for similar personnel. We also experience competition for the hiring of scientific personnel from universities and research institutions. Failure to succeed in clinical trials may make it more challenging to recruit and retain qualified scientific personnel.

Our employees may engage in misconduct or other improper activities, including violating applicable regulatory standards and requirements or engaging in insider trading, which could significantly harm our business.

~~We are exposed to the risk of employee fraud or other misconduct. Misconduct by employees could include intentional failures to comply with the regulations of the FDA and applicablenon-U.S.~~ ~~regulators, provide accurate information to the FDA and applicablenon-U.S.~~ regulators, comply with healthcare fraud and abuse laws and regulations in the United States and abroad, report financial information or data accurately or disclose unauthorized activities to us. In particular, sales, marketing and business arrangements in the healthcare industry are subject to extensive laws and regulations intended to prevent fraud, misconduct, kickbacks, self-dealing and other abusive practices. These laws and regulations restrict or prohibit a wide range of pricing, discounting, marketing and promotion, sales commission, customer incentive programs and other business arrangements. Employee

misconduct could also involve the improper use of, including trading on, information obtained in the course of clinical trials, which could result in regulatory sanctions and serious harm to our reputation. We have adopted an insider trading policy and a code of conduct, but it is not always possible to identify and deter employee misconduct, and the precautions we take to detect and prevent this activity may be ineffective in controlling unknown or unmanaged risks or losses or in protecting us from governmental investigations or other actions or lawsuits stemming from a failure to comply with these laws or regulations. If any such actions are instituted against us, and we are not successful in defending ourselves or asserting our rights, those actions could have a significant impact on our business, including the imposition of significant fines or other sanctions.

~~We face potential product liability exposure, and, if claims are brought against us, we may incur substantial liability.~~

~~The use of beloranib andZGN-1061~~ ~~in clinical trials and the sale ofZGN-1061,~~ if developed and approved, exposes us to the risk of product liability claims. Product liability claims might be brought against us by patients, healthcare providers or others selling or otherwise coming into contact with~~ZGN-1061.~~ For example, we may be sued if any product we develop allegedly causes injury or death or is found to be otherwise unsuitable during product testing, manufacturing, marketing or sale. Any such product liability claims may include allegations of defects in manufacturing, defects in design, a failure to warn of dangers inherent in the product, including as a result of interactions with alcohol or other drugs, negligence, strict liability and a breach of warranties. Claims could also be asserted under state consumer protection acts. If we become subject to product liability claims and cannot successfully defend ourselves against them, we could incur substantial liabilities. In addition, regardless of merit or eventual outcome, product liability claims may result in, among other things:

We maintain product liability insurance coverage for our clinical trials with a $10.0 million annual aggregate coverage limit. Nevertheless, our insurance coverage may be insufficient to reimburse us for any expenses or losses we may suffer. Moreover, in the future, we may not be able to maintain insurance coverage at a reasonable cost or in sufficient amounts to protect us against losses, including if insurance coverage becomes increasingly expensive. If and when we obtain marketing approval for~~ZGN-1061,~~ we intend to expand our insurance coverage to include the sale of commercial products; however, we may not be able to obtain this product liability insurance on commercially reasonable terms. Large judgments have been awarded in class action lawsuits based on drugs that had unanticipated side effects. The cost of any product liability litigation or other proceedings, even if resolved in our favor, could be substantial, particularly in light of the size of our business and financial resources. A product liability claim or series of claims brought against us could cause our stock price to decline and, if we are unsuccessful in defending such a claim or claims and the resulting judgments exceed our insurance coverage, our financial condition, business and prospects could be materially adversely affected.

We must maintain effective internal control over financial reporting, and if we are unable to do so, the accuracy and timeliness of our financial reporting may be adversely affected, which could have a material adverse effect on our business and stock price.

We currently are an “emerging growth company,” as defined in the Jumpstart our Business Startups Act of 2012, or the JOBS Act, and we have taken advantage of certain exemptions from various reporting requirements that are applicable to other public companies that are not “emerging growth companies” including not being required to comply with the auditor attestation requirements of Section 404(b) of the Sarbanes-Oxley Act.

We must maintain effective internal control over financial reporting in order to accurately and timely report our results of operations and financial condition. In addition, as a public company, the Sarbanes-Oxley Act of 2002, or the Sarbanes-Oxley Act, requires, among other things, that we assess the effectiveness of our disclosure controls and procedures quarterly and the effectiveness of our internal control over financial reporting at the end of each fiscal year.

The rules governing the standards that must be met for our management to assess our internal control over financial reporting pursuant to Section 404 of the Sarbanes-Oxley Act are complex and require significant documentation, testing and possible remediation. These stringent standards require that our audit committee be advised and regularly updated on management’s review of

internal control over financial reporting. Our management may not be able to effectively and timely implement controls and procedures that adequately respond to the increased regulatory compliance and reporting requirements that are applicable to us as a public company. If we fail to staff our accounting and finance function adequately or maintain internal control over financial reporting adequate to meet the demands that will be placed upon us as a public company, including the requirements of the Sarbanes-Oxley Act, our business and reputation may be harmed and our stock price may decline. Furthermore, investor perceptions of us may be adversely affected, which could cause a decline in the market price of our common stock.

~~Our ability to use our net operating loss carryforwards and certain tax credit carryforwards may be subject to limitation.~~

Since our inception in 2005, we have not recorded any U.S. federal or state income tax benefits for the net losses we have incurred in each year or our earned tax credits, due to our uncertainty of realizing a benefit from those items. As of December 31, 2016, we had net operating loss carryforwards for federal and state income tax purposes of $49.1 million and $35.9 million, respectively, which begin to expire in 2026 and 2030, respectively. As of December 31, 2016, we did not record deferred tax assets of $12.8 million (gross) that were attributable to stock option exercises which will be recorded as an increase in additional paid in capital once they are realized in accordance with accounting for stock-based compensation awards. These deductions are not reflected in the federal and state net operating loss carryforwards and the capitalized research and development expense deferred tax assets in the amounts of $9.4 million, $7.2 million, and $3.4 million, respectively. As of December 31, 2016, we also had available tax credit carryforwards for federal and state income tax purposes of $13.1 million and $1.9 million, respectively, which begin to expire in 2026 and 2021, respectively. Under Section 382 of the Internal Revenue Code of 1986, as amended, or the Code, changes in our ownership may limit the amount of our net operating loss carryforwards and tax credit carryforwards that could be utilized annually to offset our future taxable income, if any. This limitation would generally apply in the event of a cumulative change in ownership of our company of more than 50% within a three-year period. Any such limitation may significantly reduce our ability to utilize our net operating loss carryforwards and tax credit carryforwards before they expire. Our~~follow-on~~ public offering, initial public offering, or IPO, private placements and other transactions that have occurred since our inception, may trigger such an ownership change pursuant to Section 382. Any such limitation, whether as the result of our~~follow-on~~ public offering, IPO, prior private placements, sales of our common stock by our existing stockholders or additional sales of our common stock by us, could have a material adverse effect on our results of operations in future years. We have not completed a study to assess whether an ownership change for purposes of Section 382 has occurred, or whether there have been multiple ownership changes since our inception, due to the significant costs and complexities associated with such study.

~~Unfavorable global economic conditions could adversely affect our business, financial condition or results of operations.~~

Our results of operations could be adversely affected by general conditions in the global economy and in the global financial markets. A severe or prolonged economic downturn could result in a variety of risks to our business, including, weakened demand for our product candidates and our ability to raise additional capital when needed on acceptable terms, if at all. A weak or declining economy could also strain our suppliers, possibly resulting in supply disruption, or cause our customers to delay making payments for our services. Any of the foregoing could harm our business and we cannot anticipate all of the ways in which the current economic climate and financial market conditions could adversely impact our business.

~~Our internal computer systems, or those of our third-party CROs or other contractors or consultants, may fail or suffer security breaches, which could result in a material disruption of ourZGN-1061~~ ~~or other product candidate development programs.~~

Despite the implementation of security measures, our internal computer systems and those of our third-party CROs and other contractors and consultants are vulnerable to damage from computer viruses, unauthorized access, natural disasters, terrorism, war and telecommunication and electrical failures. While we have not experienced any such system failure or accident, if such an event were to occur and cause interruptions in our operations, it could result in a material disruption of our programs. For example, the loss of clinical trial data for~~ZGN-1061~~ could result in delays in our regulatory approval efforts and significantly increase our costs to recover or reproduce the data. To the extent that any disruption or security breach results in a loss of or damage to our data or applications or other data or applications relating to our technology or product candidates, or inappropriate disclosure of confidential or proprietary information, we could incur liabilities and the further development of~~ZGN-1061~~ ~~could be delayed.~~

~~We may not be successful in our efforts to identify or discover additional product candidates.~~

~~The success of our business depends primarily upon our ability to identify, develop and commercialize products based on our MetAP2 platform. AlthoughZGN-1061~~ is currently in clinical development, our research programs may fail to identify other potential product candidates for clinical development for a number of reasons. Our research methodology may be unsuccessful in identifying potential product candidates or our potential product candidates may be shown to have harmful side effects or may have other characteristics that may make the products unmarketable or unlikely to receive marketing approval.

If any of these events occur, we may be forced to abandon our development efforts for a program or programs, which would have a material adverse effect on our business and could potentially cause us to cease operations. Research programs to identify new product candidates require substantial technical, financial and human resources. We may focus our efforts and resources on potential programs or product candidates that ultimately prove to be unsuccessful.

We may seek to establish collaborations and, if we are not able to establish them on commercially reasonable terms, we may have to alter our development and commercialization plans or expand our internal efforts and growth.

Our drug development programs and the potential commercialization of our product candidates will require substantial additional cash to fund expenses. For some of our product candidates, we may decide to collaborate with pharmaceutical and biotechnology companies for the development and potential commercialization of those product candidates in some or all markets.

We face significant competition in seeking appropriate collaborators. Whether we reach a definitive agreement for a collaboration will depend, among other things, upon our assessment of the collaborator’s resources and expertise, the terms and conditions of the proposed collaboration and the proposed collaborator’s evaluation of a number of factors. Those factors may include the design or results of clinical trials, the likelihood of approval by the FDA or similar regulatory authorities outside the United States, the potential market for the applicable product candidate, the costs and complexities of manufacturing and delivering such product candidate to patients, the potential of competing products, the existence of uncertainty with respect to our ownership of technology, which can exist if there is a challenge to such ownership without regard to the merits of the challenge and industry and market conditions generally. The collaborator may also consider alternative product candidates or technologies for similar indications that may be available to collaborate on and whether such collaboration could be more attractive than the one with us for our product candidate. The terms of any collaboration or other arrangements that we may establish may not be favorable to us.

We may also be restricted under existing license agreements from entering into future agreements on certain terms with potential collaborators. Collaborations are complex and time-consuming to negotiate and document. In addition, there have been a significant number of recent business combinations among large pharmaceutical companies that have resulted in a reduced number of potential future collaborators.

We may not be able to negotiate collaborations on a timely basis, on acceptable terms, or at all. If we are unable or unwilling to do so, we may have to curtail the development of the product candidate for which we are seeking to collaborate, reduce or delay its development program or one or more of our other development programs, delay its potential commercialization in some or all markets or reduce the scope of any sales or marketing activities, or increase our expenditures and undertake development or commercialization activities at our own expense, including potentially increasing our infrastructure and investment outside the United States. If we elect to increase our expenditures to fund development or commercialization activities on our own, we will need to obtain additional capital, which may not be available to us on acceptable terms or at all. If we do not have sufficient funds, we may not be able to further develop our product candidates or bring them to market and generate product revenue. In addition such efforts may require diversion of a disproportionate amount of our attention away from other~~day-to-day~~ ~~activities, and require devotion of a substantial amount of our time to managing these expansion activities.~~

In addition, any future collaborations that we enter into may not be successful. The success of our collaboration arrangements will depend heavily on the efforts and activities of our collaborators. Collaborators generally have significant discretion in determining the efforts and resources that they will apply to these collaborations. Disagreements between parties to a collaboration arrangement regarding clinical development and commercialization matters can lead to delays in the development process or commercializing the applicable product candidate and, in some cases, termination of the collaboration arrangement. These disagreements can be difficult to resolve if neither of the parties has final decision-making authority. Collaborations with pharmaceutical or biotechnology companies and other third parties often are terminated or allowed to expire by the other party. Any such termination or expiration would adversely affect us financially and could harm our business reputation.

~~We may acquire businesses or products, or form strategic alliances, in the future, and we may not realize the benefits of such acquisitions or alliances.~~

We may acquire additional businesses or products, form strategic alliances or create joint ventures with third parties that we believe will complement or augment our existing business. If we acquire businesses with promising markets or technologies, we may not be able to realize the benefit of such transactions if we are unable to successfully integrate such businesses with our existing operations and company culture. We may encounter numerous difficulties in developing, manufacturing and marketing any new products resulting from a strategic alliance or acquisition that delay or prevent us from realizing their expected benefits or enhancing our business. We cannot assure you that, following any such transaction, we will achieve the expected synergies to justify the transaction.

~~We have not generated any revenue from product sales. We have incurred significant operating losses since our inception, and anticipate that we will incur continued losses for the foreseeable future.~~

Biopharmaceutical product development is a highly speculative undertaking and involves a substantial degree of risk. Our operations to date have been limited primarily to organizing and staffing our company and conducting research and development activities for beloranib,~~ZGN-1061,ZGN-839~~ ~~and additional MetAP2 inhibitors. We have never generated any revenue from product sales. We have not obtained regulatory approvals for any of our product candidates.~~

Since our inception and until July 2016, we focused substantially all of our efforts and financial resources on developing beloranib, which was in Phase 3 clinical development for our lead indication of the treatment of hyperphagia and obesity in patients with Prader-Willi Syndrome, or PWS, and Phase 2 clinical development for the treatment of obesity in patients with hypothalamic injury-associated obesity, or HIAO. In December 2015, the FDA put the beloranib IND on full clinical hold. Due to the uncertainties, costs and risks associated with the development of beloranib, in July 2016, we suspended further development of beloranib and directed our efforts and financial resources to developing~~ZGN-1061.~~ ~~In October 2016, we suspended our development ofZGN-839~~ ~~in order to focus all of our resources to developingZGN-1061~~ ~~and the discovery and development of novel and highly differentiated MetAP2 inhibitors.~~

~~We have funded our operations to date through proceeds from sales of redeemable convertible preferred stock, convertible debt and proceeds from our IPO andfollow-on~~ public offering, and have incurred losses in each year since our inception. Our net losses were $38.9 million for the nine months ended September 30, 2017 and $57.9 million for the year ended December 31, 2016. As of September 30, 2017, we had an accumulated deficit of $276.5 million. Substantially all of our operating losses resulted from costs incurredGuggenheim Securities, LLC (“Guggenheim Securities”), in connection with ~~our development programs for beloranib,ZGN-1061~~ ~~andZGN-839,~~ ~~early research activities, licensing milestone fees and from general and administrative costs associated with our operations. We expect to incur increasing levels~~the establishment of ~~operating losses over the next several years and for the foreseeable future. Our prior losses, combined with expected future losses, have had and will continue to have~~ an ~~adverse effect on our stockholders’ equity and working capital. We expect our research and development expenses will increase over time in connection with our clinical trials ofZGN-1061,~~ ~~and of any other product candidates~~“at-the-market” offering program under which we may ~~choose~~sell up to ~~pursue. In addition, if and when we obtain marketing approval forZGN-1061,~~ we will incur significant sales, marketing and outsourced manufacturing expenses. We will continue to incur additional costs associated with operating as a public company. As a result, we expect to continue to incur significant operating losses that would increase over time for the foreseeable future. Becausean aggregate of the numerous risks and uncertainties associated with developing pharmaceutical products, we are unable to predict the extent of any future losses or when we will become profitable, if at all. Even if we do become profitable, we may not be able to sustain or increase our profitability on a quarterly or annual basis.

Our ability to become profitable depends upon our ability to generate revenue. To date, we have not generated any revenue from any of our product candidates, and we do not know when, or if, we will generate any revenue. We do not expect to generate significant revenue unless and until we obtain marketing approval of, and begin to sell,~~ZGN-1061.~~ ~~Our ability to generate revenue depends on a number of factors, including, but not limited to, our ability to:~~

~~Absent our entering into a collaboration or partnership agreement, we expect to incur significant sales and marketing costs when we prepare to commercializeZGN-1061.~~ ~~Even if we initiate and successfully complete our clinical trials ofZGN-1061,~~ ~~andZGN-1061~~ ~~is approved for commercial sale, and despite expending these costs,ZGN-1061~~ may not be a commercially successful drug. We may not achieve profitability soon after generating product sales, if ever. If we are unable to generate sufficient product revenue, we will not become profitable and may be unable to continue operations without continued funding.

We will need to raise additional funding, which may not be available on acceptable terms, or at all. Failure to obtain this necessary capital when needed may force us to delay, limit or terminate our product development efforts or other operations.

~~Developing small molecule products is expensive, and we expect our research and development expenses to increase substantially in connection with our ongoing activities, particularly as we advanceZGN-1061~~ ~~into later stage clinical trials. Depending on the status of regulatory approval or, if approved, commercialization ofZGN-1061~~ ~~or any of our other product candidates, as well as the progress we make in sellingZGN-1061~~ or any of our other product candidates, we will require additional capital to fund operating needs thereafter. We may also need to raise additional funds sooner if we choose to pursue additional indications and/or geographies for~~ZGN-1061~~ ~~or our other product candidates or otherwise expand more rapidly than we presently anticipate.~~

As of September 30, 2017, our cash, cash equivalents and marketable securities were $93.2 million. We expect that our cash, cash equivalents and marketable securities will be sufficient to fund our current operations for a period of at least one year from the issuance date of this Quarterly Report. However, our operating plan may change as a result of many factors currently unknown to us, and we may need to seek additional funds sooner than planned, through public or private equity or debt financings, government or other third-party funding, marketing and distribution arrangements and other collaborations, strategic alliances and licensing arrangements or a combination of these approaches. In any event, we will require additional capital to obtain regulatory approval for, and to commercialize, our product candidates. Raising funds in the current economic environment may present additional challenges. Even if we believe we have sufficient funds for our current or future operating plans, we may seek additional capital if market conditions are favorable or if we have specific strategic considerations.

~~Any additional fundraising efforts may divert our management from theirday-to-day~~ activities, which may adversely affect our ability to develop and commercialize our product candidates. In addition, we cannot guarantee that future financing will be available in sufficient amounts or on terms acceptable to us, if at all. Moreover, the terms of any financing may adversely affect the holdings or the rights of our stockholders and the issuance of additional securities, whether equity or debt, by us, or the possibility of such issuance, may cause the market price of our shares to decline. The sale of additional equity or convertible securities would dilute all of our stockholders. The incurrence of indebtedness would result in increased fixed payment obligations and we may be required to agree to certain restrictive covenants, such as limitations on our ability to incur additional debt, limitations on our ability to acquire, sell or license intellectual property rights and other operating restrictions that could adversely impact our ability to conduct our business. We could also be required to seek funds through arrangements with collaborative partners or otherwise at an earlier stage than otherwise would be desirable and we may be required to relinquish rights to some of our technologies or product candidate or otherwise agree to terms unfavorable to us, any of which may have a material adverse effect on our business, operating results and prospects.

If we are unable to obtain funding on a timely basis, we may be required to significantly curtail, delay or discontinue one or more of our research or development programs or the commercialization of any product candidate or be unable to expand our operations or otherwise capitalize on our business opportunities, as desired, which could materially affect our business, financial condition and results of operations.

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

We may seek additional capital through a combination of private and public equity offerings, debt financings, collaborations and strategic and licensing arrangements. To the extent that we raise additional capital through the sale of common stock or securities convertible or exchangeable into common stock, a stockholder’s ownership interest in our company will be diluted. In addition, the terms of any such securities may include liquidation or other preferences that materially adversely affect the rights of our stockholders. Debt financing, if available, would increase our fixed payment obligations and may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends. If we raise additional funds through collaboration, strategic partnerships and licensing arrangements with third parties, we may have to relinquish valuable rights to~~ZGN-1061~~ ~~or other product candidates, our intellectual property, future revenue streams or grant licenses on terms that are not favorable to us.~~

~~The market price~~$50.0 million of shares of our common stock ~~similar~~(the “ATM Shares”) from time to time through Guggenheim Securities, as sales agent.

[Under the ~~market price of shares of common stock of other biopharmaceutical companies, is subject to wide fluctuations. From January 1, 2017 to September 30, 2017~~ATM Agreement, we will set the daily closing price of our common stock on the NASDAQ Global Market ranged from a high of $5.08 to a low of $3.24 and will continue to be subject to wide fluctuations in response to many risk factors listed in this section, and others beyond our control, including:

These and other market and industry factors may cause the market price and demand for our common stock to fluctuate substantially, regardless of our actual operating performance, which may limit or prevent investors from readily selling their shares of common stock and may otherwise negatively affect the liquidity of our common stock. In addition, the stock market in general, and NASDAQ listed and biopharmaceutical companies in particular, have experienced extreme price and volume fluctuations that have often been unrelated or disproportionate to the operating performance of these companies. In the past, when the market price of a stock has been volatile, holders of that stock have instituted securities class action litigation against the company that issued the stock.

On October 21, 2015, a purported stockholder of the Company filed a putative class action lawsuit in the U.S. District Court for the District of Massachusetts, against the Company and Thomas E. Hughes, captioned Aviad Bessler v. Zafgen, Inc. and Thomas E. Hughes,~~No. 1:15-cv-13618.~~ ~~An amended complaint was filed on February 22, 2016. The amended complaint alleges violations of Sections 10(b) and 20(a) of the Securities Exchange Act of 1934 and SEC Rule10b-5~~ based on allegedly false and misleading statements and omissions regarding our clinical trials for beloranib. On August 9, 2016, the District Court granted the motion to dismiss and dismissed the amended complaint with prejudice. On August 12, 2016, plaintiffs filed a notice of appeal to the First Circuit Court of Appeals and, on April 7, 2017 the dismissal with prejudice was affirmed.

~~Our executive officers, directors, and principal stockholders exercise significant control over our company.~~

As of October 31, 2017, the existing holdings of our executive officers, directors, principal stockholders and their affiliates, including investment funds affiliated with Atlas Ventures, or Atlas, and entities affiliated with Fidelity Investment (FMR LLC), or Fidelity, represent beneficial ownership, in the aggregate, of approximately 37.2% of our common stock. As a result, these stockholders, if they act together, are able to influence our management and affairs and control the outcome of matters submitted to our stockholders for approval, including the election of directors and any sale, merger, consolidation, or sale of all or substantially all of our assets. The concentration of voting power among these stockholders may have an adverse effect on the price of our common stock. In addition, this concentration of ownership might adversely affect the market price of our common stock by:

~~Future sales of our common stock may cause our stock price to decline.~~

Sales of a substantial number of shares of our common stock in the public market or the perception that these sales might occur could significantly reduce the market price of our common stock and impair our ability to raise adequate capital through the sale of ~~additional equity securities.~~

WeATM Shares, including the number of ATM Shares to be issued, the time period during which sales are requested to be made, limitations on the number of ATM Shares that may be ~~at an increased risk of securities class action litigation.~~

~~Historically, securities class action litigation has often been brought against a company following a decline~~sold in ~~the market~~any one trading day and any minimum price ~~of its securities. This risk is especially relevant for us because biotechnology and pharmaceutical companies have experienced significant stock price volatility in recent years.~~

~~On October 21, 2015, a purported stockholder~~below which sales may not be made. Sales of the Company filed a putative class action lawsuit in the U.S. District Court for the District of Massachusetts, against the Company and Thomas E. Hughes, captioned Aviad Bessler v. Zafgen, Inc. and Thomas E. Hughes,~~No. 1:15-cv-13618.~~ ~~An amended complaint was filed on February 22, 2016. The amended complaint alleges violations of Sections 10(b) and 20(a) of the Securities Exchange Act of 1934 and SEC Rule10b-5~~ based on allegedly false and misleading statements and omissions regarding our clinical trials for beloranib. On August 9, 2016, the District Court granted the motion to dismiss and dismissed the amended complaint with prejudice. On August 12, 2016, plaintiffs filed a notice of appealATM Shares, if any, pursuant to the ~~First Circuit Court of Appeals and, on April 7, 2017 the dismissal with prejudice was affirmed.~~

We are an “emerging growth company” and have availed ourselves of reduced disclosure requirements applicable to emerging growth companies, which could make our common stock less attractive to investors.

We are an “emerging growth company,” as defined in the JOBS Act, and we have taken advantage of certain exemptions from various reporting requirements that are applicable to other public companies that are not “emerging growth companies” including not being required to comply with the auditor attestation requirements of Section 404(b) of the Sarbanes-Oxley Act, reduced disclosure obligations regarding executive compensation in our periodic reports and proxy statements, and exemptions from the requirements of holding a nonbinding advisory vote on executive compensation and shareholder approval of any golden parachute payments not previously approved. In addition, Section 107 of the JOBS Act also provides that an emerging growth company can take advantage of the extended transition period provided in Section 7(a)(2)(B) of the Securities Act, for complying with new or revised accounting standards. In other words, an emerging growth company can delay the adoption of certain accounting standards until those standards would otherwise apply to private companies. However, we are electing not to take advantage of such extended transition period, and as a result we will comply with new or revised accounting standards on the relevant dates on which adoption of such standards is required for~~non-emerging~~ growth companies. Section 107 of the JOBS Act provides that our decision to not take advantage of the extended transition period for complying with new or revised accounting standards is irrevocable. We cannot predict if investors will find our common stock less attractive because we may rely on any of the exemptions available under the JOBS Act. If some investors find our common stock less attractive as a result, thereATM Agreement may be a less active trading market for our common stock and our stock price may be more volatile. We may take advantage of these reporting exemptions until we are no longer an emerging growth company. We will remain an emerging growth company until the earliest of (i) the last day of the fiscal yearmade in which we have total annual gross revenue of $1.0 billion or more; (ii) December 31, 2019; (iii) the date on which we have issued more than $1.0 billion in nonconvertible debt during the previous three years; and (iv) the date on which wetransactions that are deemed to be ~~a large accelerated filer~~“at-the-market offerings” as defined in Rule 415 under the ~~rules~~Securities Act of 1933, as amended. We will pay Guggenheim Securities a commission equal to 3.0% of the ~~SEC.~~

gross proceeds of any ATM Shares sold through Guggenheim Securities under the ATM Agreement and have agreed to reimburse Guggenheim Securities for certain specified expenses. The ATM Agreement contains customary representations, warranties and agreements by us, indemnification obligations of ourselves and Guggenheim Securities, other obligations of the parties and termination provisions. We have never paid dividends on our common stock and we do not anticipate paying any dividends in the foreseeable future. Consequently, any gains from an investment in our common stock will likely depend on whether the price of our common stock increases.

~~We have not paid dividends on~~no obligation to sell any of ~~our common stock to date~~the ATM Shares, and ~~we currently intend to retain all of our future earnings, if~~may at any ~~to fund~~time suspend offers under the ~~development and growth of our business. As a result, capital appreciation, if any, of our common stock~~ATM Agreement.]

The ATM Shares will be the sole source of gains for our common stockholders for the foreseeable future. Consequently, in the foreseeable future, our common stockholders will likely only experience a gain from their investment in our common stock if the price of our common stock increases.

If equity research analysts do not continue to publish research or reports about our business or if they issue unfavorable commentary or downgrade our common stock, the price of our common stock could decline.

~~The trading market for our common stock relies in part on the research~~offered and reports that equity research analysts publish about us and our business. We do not control these analysts. The price of our common stock could decline if one or more equity analysts downgrade our common stock or if analysts issue other unfavorable commentary or cease publishing reports about us or our business.

~~Anti-takeover provisions contained in our amended and restated certificate of incorporation and amended and restated bylaws, as well as provisions of Delaware law, could impair a takeover attempt.~~

Our amended and restated certificate of incorporation, amended and restated bylaws and Delaware law contain provisions which could have the effect of rendering more difficult, delaying or preventing an acquisition deemed undesirable by our board of directors. Our corporate governance documents include provisions:

~~These provisions, alone or together, could delay or prevent hostile takeovers and changes in control or changes in our management.~~the Registration Statement.

As a Delaware corporation, we are also subject to provisions of Delaware law, including Section 203 of the Delaware General Corporation law, which prevents some stockholders holding more than 15% of our outstanding common stock from engaging in certain business combinations without approval of the holders of substantially all of our outstanding common stock.

Any provision of our amended and restated certificate of incorporation, amended and restated bylaws or Delaware law that has the effect of delaying or deterring a change in control could limit the opportunity for our stockholders to receive a premium for their shares of our common stock, and could also affect the price that some investors are willing to pay for our common stock.29

The exhibits filed as part of this Quarterly Report are set forth on the Exhibit Index, which is incorporated herein by reference.

Pursuant to the requirements ~~of Section 13 or 15(d)~~ of the Securities Exchange Act of 1934, as amended, the registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized.


Delaware	20-3857670
(State or other jurisdiction of incorporation or organization)	(IRS Employer Identification No.)

Three Bala Plaza East, Suite 506	19004
Bala Cynwyd, PA	(zip code)
(Address of principal executive offices)


Large accelerated filer	☐	☐	Accelerated filer	☒ ☐
Non-accelerated filer	☒
~~Non-accelerated~~ ~~filer~~		~~☐ (Do not check if a smaller reporting company)~~	Emerging growth company	☒ ☐

Smaller reporting company	☒	☐


			Page
					~~Page~~
PART I –- FINANCIAL INFORMATION

Item 1.1	Financial Statements (unaudited)			3

	Condensed Consolidated Balance Sheets as of September 30, ~~2017~~2022 and December 31, ~~2016~~2021			3

	Condensed Consolidated Statements of Operations and Comprehensive Loss for the three and nine months ended September 30, ~~2017~~2022 and ~~2016~~2021			4

	Condensed Consolidated Statements of Changes in Stockholders' Equity for the three and nine months ended September 30, 2022 and 2021		5

	Condensed Consolidated Statements of Cash Flows for the nine months ended September 30, ~~2017~~2022 and ~~2016~~2021			5	7

	Notes to Condensed Consolidated Financial Statements			6	8

Item 2.	Management’s Discussion and Analysis of Financial Condition and Results of Operations			15	19

Item 3.	Quantitative and Qualitative Disclosures About Market Risk			28

Item 4.	~~Management’s Evaluation of our Disclosure~~ Controls and Procedures			28


PART II –- OTHER INFORMATION

Item 1.	Legal Proceedings			28	29

Item 1A.	Risk Factors			29
Item 2.	Unregistered Sales of Equity Securities and Use of Proceeds		29
Item 6.3.	~~Exhibits~~Defaults Upon Senior Securities			51	29
Item 4.	Mine Safety Disclosures		29
~~Signatures~~ Item 5.	Other Information	53	29


Item 1.6.	~~Financial Statements~~	Exhibits	30

Signatures			31


	Nine Months Ended September 30,
	2017			2016
Cash flows from operating activities:
Net loss	$	(38,943	)	$	(47,439	)
Adjustments to reconcile net loss to net cash used in operating activities
Stock-based compensation expense		6,199			8,106
Non-cash interest expense		13			33
Depreciation expense		138			151
Loss on disposal of research and development equipment		—			328
Unrealized foreign currency transaction gains		(31	)		(161	)
Premium on marketable securities, net		(297	)		(299	)
Amortization of premium on marketable securities		219			934
Changes in operating assets and liabilities:
Prepaid expenses and other current assets		(649	)		256
Tax incentive receivable		(91	)		(250	)
Accounts payable		104			(3,557	)
Accrued expenses		(610	)		(1,411	)

Net cash used in operating activities		(33,948	)		(43,309	)

Cash flows from investing activities:
Proceeds from sales and maturities of marketable securities		112,164			141,366
Purchases of marketable securities		(89,179	)		(106,172	)
Purchases of property and equipment		(28	)		(660	)

Net cash provided by investing activities		22,957			34,534

Cash flows from financing activities:
Repayments of notes payable		(2,363	)		(2,179	)
Proceeds from exercise of common stock options and employee stock purchase plan		195			220

Net cash used in financing activities		(2,168	)		(1,959	)

Net decrease in cash and cash equivalents		(13,159	)		(10,734	)
Cash and cash equivalents at beginning of period		32,352			35,595

Cash and cash equivalents at end of period	$	19,193		$	24,861

Supplemental disclosure of cash flow information:
Cash paid for interest	$	130		$	314

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

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


										Accumulated
					Additional					Other			Total
	Common Stock				Paid-in		Accumulated			Comprehensive			Stockholders’
	Shares		Par Value		Capital		Deficit			Loss			Equity
Balances as of December 31, 2021		17,710,450	$	18	$	180,645	$	(116,250	)	$	—		$	64,413
Stock-based compensation expense		—		—		1,635		—			—			1,635
Net loss		—		—		—		(8,943	)		—			(8,943	)
Balances as of March 31, 2022		17,710,450	$	18	$	182,280	$	(125,193	)	$	—		$	57,105
Stock-based compensation expense		—		—		1,675		—			—			1,675
Unrealized gain on marketable debt securities		—		—		—		—			(57	)		(57	)
Net loss		—		—		—		(8,667	)		—			(8,667	)
Balances as of June 30, 2022		17,710,450	$	18	$	183,955	$	(133,860	)	$	(57	)	$	50,056
Stock-based compensation expense		—		—		1,666		—			—			1,666
Issuance of Common Stock, net		25,558,750		25		75,218		—			—			75,243
Unrealized gain on marketable debt securities		—		—		—		—			17			17
Net loss		—		—		—		(8,320	)		—			(8,320	)
Balances as of September 30, 2022		43,269,200	$	43	$	260,839	$	(142,180	)	$	(40	)	$	118,662


	As of September 30,
	2017		2016
Options to purchase common stock		3,963,480		3,220,415
Unvested restricted common stock		5,533		6,637

		3,969,013		3,227,052


	December 31, 2016
	Total		Quoted Prices in Active Markets (Level 1)		Significant Other Observable Inputs (Level 2)		Significant Unobservable Inputs (Level 3)
	(in thousands)
Cash equivalents:
Money market funds	$	22,091	$	22,091	$	—	$	—
Commercial paper		2,997		—		2,997		—
Corporate bonds		1,500		—		1,500		—

Total cash equivalents		26,588		22,091		4,497		—

Marketable securities:
Corporate bonds		69,622		—		69,622		—
Commercial paper		27,220		—		27,220		—

Total marketable securities		96,842		—		96,842		—

Total cash equivalents and marketable securities	$	123,430	$	22,091	$	101,339	$	—


	September 30, 2017
			Gross		Gross			Fair Value
	Amortized		Unrealized		Unrealized
	Cost		Gains		Losses
	(in thousands)
Assets:
Corporate bonds (due within 1 year)	$	58,772	$	—	$	(25	)	$	58,747
Commercial paper (due within 1 year)		14,763				(3	)		14,760
Certificates of deposit (due within 1 year)		480		—		—			480

	$	74,015	$	—	$	(28	)	$	73,987


	Three Months Ended September 30,				Nine Months Ended September 30,
	2017		2016		2017		2016
	(in thousands)				(in thousands)
Research and development	$	896	$	878	$	2,629	$	2,708
General and administrative	$	1,200		1,438		3,570		5,218

	$	2,096	$	2,316	$	6,199	$	7,926


Year Ending December 31,
(in thousands)
2017 (October to December)	$	118
2018		479
2019		464
2020		226

	$	1,287


	Nine Months Ended September 30, 2017			Year Ended December 31, 2016
	(in thousands)
Restructuring reserve beginning balance	$	376		$	—
Restructuring expenses incurred during the period		24			1,223
Amounts paid during the period		(400	)		(847	)

Restructuring reserve ending balance	$	—		$	376


	Three Months Ended September 30,
							Increase
	2017			2016			(Decrease)
	(in thousands)
Statement of Operations Data:
Revenue	$	—		$	—		$	—

Operating expenses:
Research and development		9,723			10,001			(278	)
General and administrative		3,117			4,830			(1,713	)

Total operating expenses		12,840			14,831			(1,991	)

Loss from operations		(12,840	)		(14,831	)		1,991

Other income (expense):
Interest income		266			230			36
Interest expense		(31	)		(132	)		101
Foreign currency transaction gains (losses), net		20			58			(38	)

Total other income (expense), net		255			156			99

Net loss	$	(12,585	)	$	(14,675	)	$	2,090


	Three Months Ended September 30,
					Increase
	2017		2016		(Decrease)
	(in thousands)
Direct research and development expenses by program:
ZGN-1061:
Preclinical and manufacturing	$	2,827	$	1,529	$	1,298
Clinical trials		1,152		704		448

Subtotal		3,979		2,233		1,746

Discovery and screening		1,887		4		1,883

Beloranib:
Preclinical and manufacturing		86		1,467		(1,381	)
Clinical trials		24		1,982		(1,958	)

Subtotal		110		3,449		(3,339	)

ZGN-839		—		108		(108	)

Subtotal		5,976		5,794		182

Unallocated expenses:
Personnel related		1,822		2,421		(599	)
Non-cash stock-based compensation		896		877		19
Consultants		638		457		181
Other		391		452		(61	)

Subtotal		3,747		4,207		(460	)

Total research and development expenses	$	9,723	$	10,001	$	(278	)


	Nine Months Ended September 30,
					Increase
	2017		2016		(Decrease)
	(in thousands)
Direct research and development expenses by program:
ZGN-1061:
Preclinical and manufacturing	$	10,695	$	3,742	$	6,953
Clinical trials		3,581		981		2,600

Subtotal		14,276		4,723		9,553

Discovery and screening		3,956		269		3,687

Beloranib:
Preclinical and manufacturing		676		5,132		(4,456	)
Clinical trials		64		7,686		(7,622	)

Subtotal		740		12,818		(12,078	)

ZGN-839		—		834		(834	)

Subtotal		18,972		18,644		328

Unallocated expenses:
Personnel related		5,525		6,867		(1,342	)
Non-cash stock-based compensation		2,629		2,707		(78	)
Consultants		1,675		2,935		(1,260	)
Other		1,127		1,508		(381	)

Subtotal		10,956		14,017		(3,061	)

Total research and development expenses	$	29,928	$	32,661	$	(2,733	)


	Three Months Ended September 30,
					Increase
	2017		2016		(Decrease)
	(in thousands)
Personnel related	$	548	$	1,787	$	(1,239	)
Non-cash stock-based compensation		1,200		1,439		(239	)
Professional fees		929		1,162		(233	)
Other		440		442		(2	)

Total general and administrative expenses	$	3,117	$	4,830	$	(1,713	)


Exhibit No.		Description

31.1*		Certification of Principal Executive Officer pursuant to Rule13a-14(a) orRule 15d-14(a) of the Securities Exchange Act of 1934, as adopted pursuant to Section 302 of the Sarbanes-Oxley Act of 2002.

31.2*		Certification of Principal Financial Officer pursuant toRule 13a-14(a) orRule 15d-14(a) of the Securities Exchange Act of 1934, as adopted pursuant to Section 302 of the Sarbanes-Oxley Act of 2002.

32.1**		Certification of Principal Executive Officer and Principal Financial Officer pursuant to 18 U.S.C. Section 1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002.

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

101.SCH*		Inline XBRL Taxonomy Extension Schema Document.

101.CAL*		Inline XBRL Taxonomy Extension Calculation Document.

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

101.LAB*		Inline XBRL Taxonomy Extension Labels Linkbase Document.

101.PRE*		Inline XBRL Taxonomy Extension Presentation Link Document.
104		Cover Page Interactive Data File (formatted as inline XBRL and contained in Exhibit 101)


	LARIMAR THERAPEUTICS, INC.
			~~ZAFGEN, INC.~~
Date: November 10, 2022	By:	/s/ Carole S. Ben-Maimon, M.D.
~~Date: November 7, 2017~~		~~By:~~	~~/s/ Jeffrey Hatfield~~ Carole S. Ben-Maimon, M.D.
			~~Jeffrey Hatfield~~
				President and Chief Executive Officer (Principal Executive Officer)

Date: November ~~7, 2017~~10, 2022	By:	~~By:~~	/s/ ~~Patricia L. Allen~~Michael Celano
			~~Patricia L. Allen~~ Michael Celano
				Chief Financial Officer (Principal Financial and Accounting Officer)