UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

Form 10-Q

Form10-Q

(Mark One)

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

For the quarterly period ended SeptemberJune 30, 20172020

or

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

For the transition period fromto.

Commission File Number001-36510

ZAFGEN,LARIMAR THERAPEUTICS, INC.

(Exact name of registrant as specified in its charter)

 

Delaware

Delaware

20-3857670

(State or other jurisdiction of

incorporation or organization)

(IRS Employer

Identification No.)

Zafgen, Inc.

175 Portland Street, 4th FloorThree Bala Plaza East, Suite 506

Boston, Massachusetts 02114Bala Cynwyd, PA 19004

(Address of principal executive offices, including zip code)

Registrant’s Telephone Number, Including Area Code:

(617)622-4003 (844) 511-9056

 

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

 

Title of each class

Trading Symbol(s)

Name of each exchange on which registered

Common Stock

LRMR

The Nasdaq Global Market

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

Indicate by check mark whether the registrant has submitted electronically 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.

 

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

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 OctoberJuly 31, 2017,2020, there were 27,489,45715,356,206 shares of the registrant’s Common Stock, $0.001 par value per share, outstanding.

 

 

 


FORWARD-LOOKING STATEMENTS

This Quarterly Report on Form10-Q, Report, or Quarterly Report, contains forward-looking statements that involve riskswithin the meaning of Section 27A of the Securities Act of 1933, as amended (the “Securities Act”), and uncertainties. We make suchSection 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), about us and our subsidiaries. These forward-looking statements pursuantare intended to be covered by the safe harbor provisions offor forward-looking statements provided by the Private Securities Litigation Reform Act of 1995 and other federal securities laws. All1995. Forward-looking statements other thanare not statements of historical facts contained in this Quarterly Report arefact, and can be identified by the use of forward-looking statements. In some cases, you can identify forward-looking statements by terminology such as “believes,” “expects,” “may,” “will,” “could,” “should,” “expects,” “intends,“projects,” “plans,” “anticipates,“goal,“believes,“targets,” “potential,” “estimates,” “predicts,“pro forma,“potential,“seeks,“continue”“intends” or “anticipates” or the negative of these termsthereof or other comparable terminology. These forward-lookingForward-looking statements include, but are not limited to, statements about:

concerning:

the accuracy of

our estimates regarding expenses, future revenues,results of operations, financial position, research and development costs, capital requirements and our needs for additional financing;

how long we can continue to fund our operations with our existing cash, forecastscash equivalents and capital requirements;marketable debt securities;

our plans to commercializeZGN-1061 as a treatment for type 2 diabetes and other related metabolic disorders;

our ability to successfully advanceZGN-1061 into later-stageoptimize and scale CTI-1601 or any other product candidate’s manufacturing process and to manufacture sufficient quantities of clinical trials;and, if approved, commercial supplies of CTI-1601;

our ability to dissociate effectsrealize any value from CTI-1601 and any other product candidate we may develop in the future and nonclinical programs being developed and anticipated to be developed in light of methionine aminopeptidase 2,inherent risks and difficulties involved in successfully bringing product candidates to market and the risk that products will not achieve broad market acceptance;

delays in our anticipated clinical timelines, patient recruitment and milestones for CTI-1601, including those associated with COVID-19;

uncertainties in obtaining successful clinical results for CTI-1601 or MetAP2, inhibitors from prothrombotic effects orany other adverse events observedproduct candidate that we may develop in clinical development of beloranib;the future and unexpected costs that may result therefrom;

our ability to provide a compelling argument for improved safety ofZGN-1061comply with regulatory requirements applicable to our business and other novel MetAP2 inhibitors relative to first generation compounds, including beloranib;

regulatory and political developments in the United States and foreign countries;

the performanceuncertainties associated with the clinical development and regulatory approval for CTI-1601 or any other product candidate that we may develop in the future, including potential delays in the commencement, enrollment and completion of our third-party contract manufacturersclinical trials;

the difficulties and clinical research organizations;

our ability to obtainexpenses associated with obtaining and maintain intellectual property protectionmaintaining regulatory approval for our proprietary assets;CTI‑1601 or any other product candidate we may develop in the future, and the indication and labeling under any such approval;

the size and growth of the potential markets for ourCTI-1601 or any other product candidates and our ability to serve those markets;

candidate that we may develop in the future, the rate and degree of market acceptance of ourCTI-1601 or any other product candidates for any indication once approved;

candidate that we may develop in the future and our ability to obtain additional financing when needed;serve those markets;

the success of competing therapies and products that are or become available for available;

our ability to obtain and maintain patent protection and defend our intellectual property rights against third-parties;

the indications thatperformance of third-parties upon which we are pursuing;depend, including third-party contract research organizations, or CROs, and third-party suppliers, manufacturers, group purchasing organizations, distributors and logistics providers;

our ability to maintain our relationships, profitability and contracts with our key commercial partners;

our ability to recruit or retain key scientific, technical, commercial, and management personnel or to retain our executive officers;

 


our ability to comply with stringent U.S. and foreign government regulations in the manufacturing of pharmaceutical products, including good manufacturing practice compliance and other relevant regulatory authorities;

our ability to maintain proper functionality and security of our internal computer and information systems and prevent or avoid cyber-attacks, malicious intrusion, breakdown, destruction, loss of key scientificdata privacy or management personnel;other significant disruption; and

the extent to which health epidemics and other outbreaks of communicable diseases, including the recent outbreak COVID-19, disrupt our operations, the operations of third parties on which we rely or the operations of regulatory agencies we interact with in the development of CTI-1601.

other risks and uncertainties, including those listed under Part II, Item 1A. Risk Factors.

You should assume that the information appearing in this report is accurate as its date only. Any forward-looking statements in this Quarterly Report reflect our current views with respect to future events or to our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by these forward-looking statements. Factors that mayThe factors the could cause actual resultsor contribute to differ materially from current expectationssuch differences include, among other things,but are not limited to, those discussed in our Current Report on Form 8-K/A filed on June 26, 2020, and those listed under Part II, Item 1A. Risk Factors and elsewhere inof this Quarterly Report. New factors emerge from time to time, and it is not possible for us to predict which factors will arise. In addition, we cannot assess the impact of each factor on our business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Except as required by law, we assume no obligation to update or revise these forward-looking statements after the date of this report for any reason, even if new information becomes available in the future.

This Quarterly Report also contains estimates, projections and other information concerning our industry, our business, 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.


Zafgen, Inc.

INDEX

 


Larimar Therapeutics, Inc.

INDEX

Page

PART I - FINANCIAL INFORMATION

Item 1.1

Financial Statements (unaudited)

3

Condensed Consolidated Balance Sheets as of SeptemberJune 30, 20172020 and December 31, 20162019

3

Condensed Consolidated Statements of Operations and Comprehensive Loss for the threeThree and nine months ended SeptemberSix Months Ended June 30, 20172020 and 20162019

4

Condensed Consolidated Statements of Changes in Stockholders Equity (Deficit) for the Three and Six Months Ended June 30, 2020 and 2019

5

Condensed Consolidated Statements of Cash Flows for the nine months ended SeptemberSix Months Ended June 30, 20172020 and 20162019

5

6

Notes to Condensed Consolidated Financial Statements

6

7

Item 2.

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

15

22

Item 3.

Quantitative and Qualitative Disclosures About Market Risk

28

30

Item 4.

Management’s Evaluation of our Disclosure Controls and Procedures

28

30

PART II - OTHER INFORMATION

Item 1.

Legal Proceedings

28

32

Item 1A.

Risk Factors

29

32

Item 6.

Exhibits

51

34

Signatures

53

36

PART I – FINANCIAL INFORMATIONOn May 28, 2020, Larimar Therapeutics, Inc. (formerly known as Zafgen, Inc.) (“Larimar”), completed its reverse merger with Chondrial Therapeutics, Inc. (“Chondrial”), in accordance with the terms of the Agreement and Plan of Merger, dated as of December 17, 2019, as amended, by and among Larimar, Chondrial, a wholly-owned subsidiary of Larimar, Zordich Merger Sub, Inc. (“Merger Sub”) and Chondrial Holdings, LLC (“Holdings”), the sole stockholder of Chondrial ( the “Merger Agreement”), pursuant to which Merger Sub merged with and into Chondrial, with Chondrial surviving as a wholly owned subsidiary of Larimar (the “Merger”).

For accounting purposes, the Merger is treated as a “reverse asset acquisition” under generally acceptable accounting principles in the United States (“U.S. GAAP”) and Chondrial is considered the accounting acquirer. Accordingly, Chondrial’s historical results of operations replace Larimar’s historical results of operations for all periods prior to the Merger and, for all periods following the Merger, the results of operations of the combined company are included in the Company’s financial statements.

This quarterly report on Form 10-Q relates to the Company’s quarter ended June 30, 2020, which includes the date of the completion of the Merger and is therefore the Company’s first periodic report that includes results of operations for the combined company, including Chondrial.

 

Unless the context otherwise requires, references to the “Company,” the “combined company” “we,” “our” or “us” in this report refer to Larimar Therapeutics, Inc. and its subsidiaries, references to “Larimar” refer to the Company following the completion of the Merger, and references to “Zafgen” refer to the Company prior to the completion of the Merger.


PART I-FINANCIAL INFORMATION

Item 1.

Financial Statements

ZAFGEN,LARIMAR THERAPEUTICS, INC.

CONDENSED CONSOLIDATED BALANCE SHEETS

(In thousands, except share and per share data)

(Unaudited)

 

  September 30, December 31, 

 

June 30,

 

 

December 31,

 

  2017 2016 

 

2020

 

 

2019

 

Assets

   

 

 

 

 

 

 

 

 

Current assets:

   

 

 

 

 

 

 

 

 

Cash and cash equivalents

  $19,193  $32,352 

 

$

112,673

 

 

$

1,009

 

Marketable securities

   73,987  96,842 

Tax incentive receivable

   469  347 

Marketable debt securities

 

 

1,011

 

 

 

 

Prepaid expenses and other current assets

   2,007  1,358 

 

 

5,427

 

 

 

3,741

 

  

 

  

 

 

Total current assets

   95,656  130,899 

 

 

119,111

 

 

 

4,750

 

Property and equipment, net

   551  661 

 

 

675

 

 

 

274

 

Operating lease right-of-use assets

 

 

4,252

 

 

 

87

 

Restricted cash

 

 

1,339

 

 

 

 

Other assets

   57  61 

 

 

80

 

 

 

90

 

  

 

  

 

 

Total assets

  $96,264  $131,621 

 

$

125,457

 

 

$

5,201

 

  

 

  

 

 

Liabilities and Stockholders’ Equity

   

Liabilities and Stockholders’ Equity (Deficit)

 

 

 

 

 

 

 

 

Current liabilities:

   

 

 

 

 

 

 

 

 

Accounts payable

  $2,676  $2,572 

 

$

2,258

 

 

$

3,539

 

Accrued expenses

   3,090  3,733 

 

 

3,796

 

 

 

2,259

 

Notes payable, current

   1,268  3,589 
  

 

  

 

 

Operating lease liabilities, current

 

 

591

 

 

 

97

 

Total current liabilities

   7,034  9,894 

 

 

6,645

 

 

 

5,895

 

  

 

  

 

 

Operating lease liabilities

 

 

6,268

 

 

 

 

Total liabilities

   7,034  9,894 

 

 

12,913

 

 

 

5,895

 

  

 

  

 

 

Commitments and contingencies (Note 7)

   

Commitments and contingencies (See Note 9)

 

 

 

 

 

 

 

 

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 June 30, 2020 and December 31, 2019; no shares issued and

outstanding as of June 30, 2020 and December 31, 2019

 

 

 

 

 

 

Common stock, $0.001 par value per share; 115,000,000 shares

authorized as of June 30, 2020 and December 31, 2019; 15,356,206 and

6,091,250 shares issued and outstanding as of June 30, 2020 and

December 31, 2019, respectively

 

 

15

 

 

 

6

 

Additionalpaid-in capital

   365,723  359,329 

 

 

153,668

 

 

 

22,432

 

Accumulated deficit

   (276,492 (237,549

 

 

(41,136

)

 

 

(23,132

)

Accumulated other comprehensive loss

   (28 (80

 

 

(3

)

 

 

 

  

 

  

 

 

Total stockholders’ equity

   89,230  121,727 
  

 

  

 

 

Total liabilities and stockholders’ equity

  $96,264  $131,621 
  

 

  

 

 

Total stockholders’ equity (deficit)

 

 

112,544

 

 

 

(694

)

Total liabilities and stockholders’ equity (deficit)

 

$

125,457

 

 

$

5,201

 

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


LARIMAR THERAPEUTICS, INC.

ZAFGEN, INC.

CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS

(In thousands, except share and per share data)

(Unaudited)

 

 

Three Months Ended June 30,

 

 

Six Months Ended June 30,

 

  Three Months Ended
September 30,
 Nine Months Ended
September 30,
 
  2017 2016 2017 2016 

Revenue

  $—    $—    $—    $—   
  

 

  

 

  

 

  

 

 

 

2020

 

 

2019

 

 

2020

 

 

2019

 

Operating expenses:

     

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Research and development

   9,723  10,001  29,928  32,661 

 

$

8,907

 

 

$

3,128

 

 

$

13,914

 

 

$

7,350

 

General and administrative

   3,117  4,830  9,713  15,089 

 

 

2,492

 

 

 

576

 

 

 

4,159

 

 

 

1,078

 

  

 

  

 

  

 

  

 

 

Total operating expenses

   12,840  14,831  39,641  47,750 

 

 

11,399

 

 

 

3,704

 

 

 

18,073

 

 

 

8,428

 

  

 

  

 

  

 

  

 

 

Loss from operations

   (12,840 (14,831 (39,641 (47,750

 

 

(11,399

)

 

 

(3,704

)

 

 

(18,073

)

 

 

(8,428

)

  

 

  

 

  

 

  

 

 

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, net

 

 

69

 

 

 

 

 

 

69

 

 

 

 

Net loss

  $(12,585 $(14,675 $(38,943 $(47,439

 

$

(11,330

)

 

$

(3,704

)

 

$

(18,004

)

 

$

(8,428

)

  

 

  

 

  

 

  

 

 

Net loss per share, basic and diluted

  $(0.46 $(0.54 $(1.42 $(1.74

 

$

(1.21

)

 

$

(0.61

)

 

$

(2.33

)

 

$

(1.38

)

  

 

  

 

  

 

  

 

 

Weighted average common shares outstanding, basic and diluted

   27,483,550  27,322,907  27,414,314  27,286,323 

 

 

9,381,412

 

 

 

6,091,250

 

 

 

7,736,331

 

 

 

6,091,250

 

  

 

  

 

  

 

  

 

 

Comprehensive loss:

     

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Net loss

  $(12,585 $(14,675 $(38,943 $(47,439

 

$

(11,330

)

 

$

(3,704

)

 

$

(18,004

)

 

$

(8,428

)

Other comprehensive loss:

     

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Unrealized gain (loss) on marketable securities

   13  (52 52  141 
  

 

  

 

  

 

  

 

 

Total other comprehensive income (loss)

   13  (52 52  141 
  

 

  

 

  

 

  

 

 

Unrealized loss on marketable debt securities

 

 

(3

)

 

 

 

 

 

(3

)

 

 

 

Total other comprehensive loss

 

 

(3

)

 

 

 

 

 

(3

)

 

 

 

Total comprehensive loss

  $(12,572 $(14,727 $(38,891 $(47,298

 

$

(11,333

)

 

$

(3,704

)

 

$

(18,007

)

 

$

(8,428

)

  

 

  

 

  

 

  

 

 

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


LARIMAR THERAPEUTICS, INC.

ZAFGEN, INC.

CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWSCHANGES IN

STOCKHOLDERS’ EQUITY (DEFICIT)

(In thousands)thousands, except share data)

(Unaudited)

 

   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 
  

 

 

  

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Accumulated

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Additional

 

 

 

 

 

 

Other

 

 

Total

 

 

 

Common Stock

 

 

Paid-in

 

 

Accumulated

 

 

Comprehensive

 

 

Stockholders’

 

 

 

Shares

 

 

Par Value

 

 

Capital

 

 

Deficit

 

 

Loss

 

 

Equity

 

Balances as of December 31, 2019

 

 

6,091,250

 

 

$

6

 

 

$

22,432

 

 

$

(23,132

)

 

$

 

 

$

(694

)

Capital contributions from related party

 

 

 

 

 

 

 

 

9,595

 

 

 

 

 

 

 

 

 

9,595

 

Stock-based compensation expense

 

 

 

 

 

 

 

 

29

 

 

 

 

 

 

 

 

 

29

 

Net loss

 

 

 

 

 

 

 

 

 

 

 

(6,674

)

 

 

 

 

 

(6,674

)

Balances as of March 31, 2020

 

 

6,091,250

 

 

 

6

 

 

 

32,056

 

 

 

(29,806

)

 

 

 

 

 

2,256

 

Capital contributions from related party

 

 

 

 

 

 

 

 

8,400

 

 

 

 

 

 

 

 

 

8,400

 

Merger with Zafgen Inc.

 

 

3,124,337

 

 

 

3

 

 

 

37,116

 

 

 

 

 

 

 

 

 

37,119

 

Private Placement of common shares and pre-funded warrants, net of transaction costs

 

 

6,140,619

 

 

 

6

 

 

 

75,344

 

 

 

 

 

 

 

 

 

75,350

 

Stock-based compensation expense

 

 

 

 

 

 

 

 

752

 

 

 

 

 

 

 

 

 

752

 

Unrealized loss on marketable debt securities

 

 

 

 

 

 

 

 

 

 

 

 

 

 

(3

)

 

 

(3

)

Net loss

 

 

 

 

 

 

 

 

 

 

 

(11,330

)

 

 

 

 

 

(11,330

)

Balances as of June 30, 2020

 

 

15,356,206

 

 

 

15

 

 

 

153,668

 

 

 

(41,136

)

 

 

(3

)

 

 

112,544

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Balances as of December 31, 2018

 

 

6,091,250

 

 

 

6

 

 

 

2,908

 

 

 

 

 

 

 

 

 

2,914

 

Capital contributions from related party

 

 

 

 

 

 

 

 

3,000

 

 

 

 

 

 

 

 

 

3,000

 

Stock-based compensation expense

 

 

 

 

 

 

 

 

34

 

 

 

 

 

 

 

 

 

34

 

Net loss

 

 

 

 

 

 

 

 

 

 

 

(4,724

)

 

 

 

 

 

(4,724

)

Balances as of March 31, 2019

 

 

6,091,250

 

 

 

6

 

 

 

5,942

 

 

 

(4,724

)

 

 

 

 

 

1,224

 

Capital contributions from related party

 

 

 

 

 

 

 

 

2,990

 

 

 

 

 

 

 

 

 

2,990

 

Stock-based compensation expense

 

 

 

 

 

 

 

 

33

 

 

 

 

 

 

 

 

 

33

 

Net loss

 

 

 

 

 

 

 

 

 

 

 

(3,704

)

 

 

 

 

 

(3,704

)

Balances as of June 30, 2019

 

 

6,091,250

 

 

$

6

 

 

$

8,965

 

 

$

(8,428

)

 

$

 

 

$

543

 

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


LARIMAR THERAPEUTICS, INC.

ZAFGEN, INC.CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS

(In thousands)

(Unaudited)

 

 

Six Months Ended June 30,

 

 

 

2020

 

 

2019

 

Cash flows from operating activities:

 

 

 

 

 

 

 

 

Net loss

 

$

(18,004

)

 

$

(8,428

)

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

 

 

 

 

 

 

 

 

Stock-based compensation expense

 

 

781

 

 

 

67

 

Depreciation expense

 

 

55

 

 

 

38

 

Changes in operating assets and liabilities:

 

 

 

 

 

 

 

 

Prepaid expenses and other current assets

 

 

(1,760

)

 

 

(1,324

)

Accounts payable

 

 

(3,284

)

 

 

653

 

Accrued expenses

 

 

1,067

 

 

 

11

 

Right-of-use assets

 

 

89

 

 

 

39

 

Operating lease liabilities

 

 

(85

)

 

 

(26

)

Other assets

 

 

21

 

 

 

5

 

Net cash used in operating activities:

 

 

(21,120

)

 

 

(8,965

)

Cash flows from investing activities:

 

 

 

 

 

 

 

 

Purchase of property and equipment

 

 

(58

)

 

 

(33

)

Cash, cash equivalents, and restricted cash acquired in connection with the Merger

 

 

41,934

 

 

 

 

Merger transaction costs

 

 

(1,233

)

 

 

 

Net cash provided by (used in) investing activities

 

 

40,643

 

 

 

(33

)

Cash flows from financing activities:

 

 

 

 

 

 

 

 

Capital contribution from related party

 

 

17,995

 

 

 

5,990

 

Proceeds from sale of common stock and prefunded warrants, net of issuance costs

 

 

75,485

 

 

 

 

Net cash provided by financing activities

 

 

93,480

 

 

 

5,990

 

Net increase (decrease) in cash, cash equivalents and restricted cash

 

 

113,003

 

 

 

(3,008

)

Cash, cash equivalents and restricted cash at beginning of period

 

 

1,009

 

 

 

4,396

 

Cash, cash equivalents and restricted cash at end of period

 

$

114,012

 

 

$

1,388

 

Supplemental disclosure of non-cash investing and financing activities:

 

 

 

 

 

 

 

 

Fair value of net assets acquired in the Merger, including $1.0 million of marketable debt securities and excluding cash acquired

 

$

(4,815

)

 

$

 

Leased assets obtained in exchange for new operating lease liabilities

 

$

448

 

 

$

 

Offering costs included in accounts payable and accrued expenses

 

$

135

 

 

$

 

Merger transaction costs included in accounts payable and accrued expenses

 

$

65

 

 

$

 

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


LARIMAR THERAPEUTICS, INC.

NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS

(Unaudited)

1.

Organization, Nature of the Business, COVID-19 Risk and Basis of Presentation

1. Nature of the Business and Basis of Presentation

Zafgen,Larimar Therapeutics, Inc., together with its subsidiaries (the “Company” or the Company, was incorporated on November 22, 2005 under the laws of the State of Delaware. The Company“Larimar”), is a clinical stage biopharmaceutical company dedicatedleveraging its proprietary knowledge 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 novel therapeutics that treat the underlying biological mechanisms through the methionine aminopeptidase 2 (“MetAP2”) pathway.develop a therapeutic treatment for mitochondrial disorders which currently have no cure. The Company has pioneeredfocused on Friedreich’s Ataxia, which is a progressive disease that affects multiple body systems, particularly the study of MetAP2 inhibitors in both commonbrain and rare forms of obesity. Theheart. CTI-1601, the Company’s lead product candidate isZGN-1061,in Phase 1 clinical development, utilizes a novel fumagillin-class MetAP2 inhibitor administered by subcutaneous injection, whichcell penetrant peptide to deliver frataxin, the protein deficient in Friedreich’s Ataxia, to the mitochondria where it is currently being profiled for its utilitybelieved to be processed into mature frataxin and becomes active in the treatment of type 2 diabetes and other related metabolic disorders. Since its inception, the Company has devoted substantially all of its efforts to research and development, recruiting management, acquiring operating assets and raising capital. Currently, the Company is focusing its personnel and financial resources onZGN-1061 and the discovery of novel and highly differentiated MetAP2 inhibitors.mitochondrial metabolism.

The Company is subject to risks and uncertainties common to pre-commercialization 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 governmentgovernmental regulations and the needability to obtainsecure additional financing. Productcapital to fund operations. Drug candidates currently under development will require significant additional research and development efforts, including extensive preclinicalnonclinical 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 productdrug development efforts are successful, it is uncertain when, if ever, the Company will generaterealize significant revenue from product sales. The Company operates in an environment

In March 2020, the World Health Organization declared the outbreak of rapid change in technologyCOVID-19, a novel strain of Coronavirus, a global pandemic. This outbreak is causing major disruptions to businesses and substantial competition from pharmaceutical and biotechnology companies. In addition, the Company is dependent upon the services 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 itsfollow-on offering in January 2015. Until such time, if ever,markets worldwide as the Company can generate substantial product revenue,virus spreads. The extent of the Company expectseffect on the Company’s operational and financial performance will depend on future developments, including the duration, spread and intensity of the pandemic, and governmental, regulatory and private sector responses, all of which are uncertain and difficult to finance its cash needs through a combination of equity offerings, debt financings, collaborations, strategic alliances, licensing arrangements and other sources of funding. Ifpredict. Although the Company is unable to raise additional funds through equity, debt financings or licensing arrangements when needed,estimate the Company may be requiredfinancial effect of the pandemic at this time, if the pandemic continues to delay, limit, reduce or terminate product development or future commercialization efforts or grant rights to develop and market products or product candidates thatevolve into a severe worldwide crisis, it could have a material adverse effect on 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, 2017 will be sufficient to fund its anticipated levelCompany’s business, results of operations, financial condition and capital expenditurescash flows. The financial statements do not reflect any adjustments as a result of the pandemic.

The pandemic resulted in the temporary stoppage of the Company’s Phase 1 clinical trial studying CTI-1601 in patients with Friedreich’s Ataxia after the completion of two cohorts. The Company has since resumed the SAD Phase 1 clinical trial in July 2020. The Company is conducting the clinical trial at one clinical trial site. Because Friedreich’s Ataxia is a rare disease, there are a limited number of patients in close proximity to the clinical trial site and clinical trial patients travel from throughout the United States to the clinical trial site to participate. After dosing, patients remain in isolation in the clinical research unit for a period of at least one yeartime. The travel advisories and risk of infection related to COVID-19 have presented increased risks to patients traveling to the Company’s clinical trial site for dosing and the Company expects to incur additional clinical trial costs to safely transport and isolate patients participating in the trial. While top line results from the issuance dateongoing Phase 1 clinical trials were originally expected by the end of 2020, the delay in the clinical trial timeline caused by the ongoing impact of COVID-19 resulted in top line results now being expected in the first half of 2021. The Company may experience additional delays in clinical trial timelines as a result of additional travel and hospital restrictions related to the COVID-19 pandemic which may be imposed, including as a result of resurgences of COVID-19 cases in certain geographic areas.

Merger with Zafgen

On December 17, 2019, Zafgen, Inc. (“Zafgen”), Chondrial Therapeutics Inc. (“Chondrial”), Zordich Merger Sub, Inc. (“Merger Sub”) and Chondrial Holdings, LLC (“Holdings”), the sole stockholder of Chondrial, entered into an Agreement and Plan of Merger, as amended on March 9, 2020 (the “Merger Agreement”), pursuant to which Merger Sub merged with and into Chondrial, with Chondrial surviving as a wholly owned subsidiary of the Company and the surviving corporation of the merger (the “Merger”).

The transaction was accounted for as a reverse acquisition in accordance with accounting principles generally accepted in the United States of America ("GAAP"). Under this Quarterly Report.method of accounting, Chondrial was deemed to be the accounting acquirer for financial reporting purposes. This determination was primarily based on the facts that, immediately following the Merger: (1) shareholders of Chondrial own a substantial majority of the voting rights of


the combined company; (2) the majority of the board of directors of the combined company is composed of directors designated by Chondrial under the terms of the merger; and (3) existing members of Chondrial management will be the management of the combined company. Because Chondrial has been determined to be the accounting acquirer in the Merger, but not the legal acquirer, the Merger is deemed a reverse acquisition under the guidance of the Financial Accounting Standards Board (“FASB”) Accounting Standards Codification (“ASC”) Topic 805, Business Combinations. As a result, the historical financial statements of Chondrial are the historical financial statements of the combined company. As the Merger has been accounted for as an asset acquisition, goodwill has not been recorded within the condensed combined balance sheet.

The Merger was completed on May 28, 2020 pursuant to the terms of the Merger Agreement. In addition, immediately prior to the closing of the Merger, Zafgen effected a 1-for-12 reverse stock split (the “Reverse Stock Split”) of Zafgen’s common stock, par value $0.001 per share (the “Zafgen Common Stock”). At the effective time of the Merger (the “Effective Time”), each share of Chondrial’s common stock, par value $0.001 per share (“Chondrial Common Stock”), outstanding immediately prior to the Effective Time was converted into the right to receive shares of Zafgen based on an exchange ratio set forth in the Merger Agreement. At the Effective Time following the Reverse Stock Split, the exchange ratio was determined to be 60,912.5005 shares of Zafgen Common Stock for each share of Chondrial Common Stock (the “Exchange Ratio”). At the closing of the Merger on May 28, 2020, Zafgen issued an aggregate of 6,091,250 shares of its common stock to Holdings (the “Merger Shares”), based on the Exchange Ratio after giving effect to the Reverse Stock Split described below. Holdings subsequently distributed the Merger Shares to its members.

In addition, all outstanding options exercisable for common units of Holdings became options exercisable for the shares of common stock of Zafgen based on the conversion factor discussed within the Merger Agreement. In connection with the Merger, Zafgen changed its name to Larimar Therapeutics, Inc. Following the closing of the Merger, Chondrial Therapeutics, Inc. became a wholly-owned subsidiary of the Company. As used herein, the words “the Company” refers to, for periods following the Merger, Larimar, together with its subsidiaries, and for periods prior to the Merger, Chondrial Therapeutics Inc., and its direct and indirect subsidiaries, as applicable.

Basis of Presentation

The condensed consolidated financial statements include the accounts of the CompanyLarimar and its wholly owned subsidiaries, Chondrial Therapeutics Inc., Chondrial Therapeutics IP LLC, Zafgen Securities Corporation, Zafgen Australia Pty Limited and Zafgen Animal Health, LLC. All intercompany balances and transactions have been eliminated. The accompanying condensed consolidated financial statements have been prepared in conformity with GAAP. Unless otherwise noted, all references to common stock share and per share amounts have also been adjusted to reflect the Exchange Ratio.

Reverse Stock Split

On May 28, 2020, immediately prior to the closing of the Merger, Zafgen effected the Reverse Stock Split. Accordingly, all share and per share amounts for all periods presented in the accompanying consolidated financial statements and notes thereto have been adjusted retroactively, where applicable, to reflect the Reverse Stock Split. No fractional shares were issued in connection with the Reverse Stock Split. Unless otherwise noted, all references to common stock share and per share amounts have also been adjusted to reflect the Exchange Ratio.

Going Concern Assessment

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 consolidated financial statements are issued. As of the issuance date of these condensed consolidated financial statements, the Company expects that its cash and cash equivalents will be sufficient to fund its forecasted operating expenses and capital expenditure requirements for at least the next twelve months from the issuance date of these financial statements.

Since its inception, the Company has incurred significant operating losses and negative cash flows from operations. 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, will need additional capital to fund its future operations, which it may raise through a combination of equity offerings, debt financings,


other third-party funding, marketing and distribution arrangements, other collaborations, strategic alliances and licensing arrangements.

The Company has funded its operations to date primarily with proceeds from sales of common stock, prefunded warrants for the purchase of common stock and contributions from Holdings. In 2020, the Company completed the Merger and acquired $42.9 million of cash, cash equivalents, restricted cash and marketable debt securities that were held by Zafgen immediately prior to the Merger. The Company also raised $75.4 million, net of offering costs, through a private offering of common stock and prefunded warrants to purchase shares of common stock in connection with and immediately after the closing of the Merger. In addition, in 2020, prior to the Merger, the Company received $18.0 million in capital contributions from Holdings.

If the Company is unable to obtain future funding when needed, the Company may be forced to delay, reduce or eliminate some or all of its research and development programs, product portfolio expansion or pre‑commercialization efforts, which could adversely affect its business prospects, or the Company may be unable to continue operations. There is no assurance that the Company will be successful in obtaining sufficient funding on terms acceptable to the Company to fund continuing operations, if at all.

2.

Summary of Significant Accounting Policies

Unaudited Interim Financial Information

The condensed consolidated balance sheet as of December 31, 20162019 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 SeptemberJune 30, 20172020 and for the three and ninesix months ended SeptemberJune 30, 20172020 and 2016,2019, 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, 2016,2019 included in the Company’s AnnualCurrent Report onForm 10-K, for the year ended December 31, 2016,8-K/A filed on file with the SEC.June 26, 2020. 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 SeptemberJune 30, 20172020 and condensed consolidated results of operations and cash flows for the three and ninesix months ended SeptemberJune 30, 20172020 and 20162019 have been made. The results of operations for the three and ninesix months ended SeptemberJune 30, 20172020 are not necessarily indicative of the results of operations that may be expected for the year ending December 31, 2017.

2. Summary of Significant Accounting Policies2020.

Use of Estimates

The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities theand disclosure of contingent assets and liabilities as ofat 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 theexpense, valuation of stock-based awards. Estimates are periodically reviewedawards and valuation of leases. Due to inherent uncertainty involved in light ofmaking estimates, actual results reported in future periods may be affected by changes in circumstances, factsthese estimates. On an ongoing basis, the Company evaluates its estimates and experience. Actual resultsassumptions.

Concentrations of Credit Risk and Significant Suppliers

Financial instruments that potentially subject the Company to concentrations of credit risk consist principally of cash and cash equivalents. The Company generally maintains cash balances in various operating accounts at financial institutions that management believes to be of high credit quality in amounts that may exceed federally insured limits. The Company has not experienced losses related to its cash and cash equivalents.


The Company is highly dependent on third-party manufacturers to supply products for research and development activities in its programs. The Company relies and expects to continue to rely on a small number of manufacturers to supply it with its requirements for the active pharmaceutical ingredients and formulated drugs related to these programs. These programs could differ frombe adversely affected by a significant interruption in these manufacturing services or in the Company’s estimates.supply of active pharmaceutical ingredients and formulated drugs.

Cash and Cash Equivalents

The Company considers all highly liquid investments with maturities of three months or less at the date of purchase to be cash equivalents. Cash equivalents consisted of money market funds, U.S. government securities and corporate bonds as of June 30, 2020. As of December 31, 2019, the Company did not have cash equivalents.

Marketable debt securities

Marketable debt securities consist of debt investments with original maturities greater than 90ninety days. The Company has classifiedclassifies its investments with maturities beyond one yearmarketable debt securities as short term, based on their highly liquid nature and because such marketable securities represent the investment of cash that is available for current operations. 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. UnrealizedWhen the fair value is below the amortized cost the amount of the expected credit loss is estimated. The credit-related impairment amount is recognized in net income; the remaining impairment amount and unrealized gains and losses are reported as a component of accumulated other comprehensive income (loss) in stockholders’ equity. Realized gainsCredit losses are recognized through the use of an allowance for credit losses account and subsequent improvements in expected credit losses and declines in value judged to be other than temporary are includedrecognized as a componentreversal of other income (expense), net based on the specific identification method. When determining whether a decline in value is other than temporary, the Company considers various factors, including whetherallowance account. If the Company has the intent to sell the security and whetheror it is more likely than not that the Company will be required to sell the security prior to recovery of its amortized cost basis. Fairbasis, the allowance for credit loss is written off and the excess of the amortized cost basis of the asset over its fair value is recorded in net income.

Segment Information

The Company manages its operations as a single operating segment for the purposes of assessing performance and making operating decisions. The Company’s focus is on the research, development and commercialization of novel therapeutics for the treatment of rare diseases.

Research and Development Costs

Costs associated with internal research and development and external research and development services, including drug development and nonclinical studies, are expensed as incurred. Research and development expenses include costs for salaries, employee benefits, subcontractors, facility-related expenses, depreciation, stock-based compensation, third-party license fees, laboratory supplies, and external costs of outside vendors engaged to conduct discovery, nonclinical and clinical development activities and clinical trials as well as to manufacture clinical trial materials, and other costs. The Company recognizes external research and development costs based on 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 development activities are recorded as prepaid expenses. Such prepaid expenses are recognized as an expense when the 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 the expenditure. Amounts incurred are classified as general and administrative expenses.

Stock-Based Compensation

The Company measures all stock-based awards granted to employees, non-employee consultants and directors 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, the Company issues awards with only service-based and market-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 the fact that the Company has never paid cash dividends on common stock and does not expect to pay any cash dividends in the foreseeable future.

Income Taxes

The Company accounts for income taxes using the asset and liability method, which requires the recognition of deferred tax assets and liabilities for the expected future tax consequences of events that have been recognized in the consolidated financial statements or in the Company’s tax returns. Deferred tax assets and liabilities are determined based on quoted market prices.the difference between the financial statement and tax basis of assets and liabilities using enacted tax rates in effect for the year in which the differences are expected to reverse. Changes in deferred tax assets and liabilities are recorded in the provision for income taxes. The Company assesses the likelihood that its deferred tax assets will be recovered from future taxable income and, to the extent it believes, based upon the weight of available evidence, that it is more likely than not that all or a portion of the deferred tax assets will not be realized, a valuation allowance is established through a charge to income tax expense. Potential for recovery of deferred tax assets is evaluated by estimating the future taxable profits expected and considering prudent and feasible tax planning strategies.

The Company accounts for uncertainty in income taxes recognized in the consolidated financial statements by applying a two-step process to determine the amount of tax benefit to be recognized. First, the tax position must be evaluated to determine the likelihood that it will be sustained upon external examination by the taxing authorities. If the tax position is deemed more-likely-than-not to be sustained, the tax position is then assessed to determine the amount of benefit to recognize in the consolidated financial statements. The amount of the benefit that may be recognized is the largest amount that has a greater than 50% likelihood of being realized upon ultimate settlement. The provision for income taxes includes the effects of any resulting tax reserves, or unrecognized tax benefits, that are considered appropriate as well as the related net interest and penalties.

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 six months ended June 30, 2020 includes the weighted average effect of 628,403 prefunded warrants for the purchase of shares of common stock, which were issued in June 2020, and 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 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 common stock equivalents, outstanding as of SeptemberJune 30, 20172020 and 2016,2019, from the computation of diluted net loss per share for the three and six months ended SeptemberJune 30, 20172020 and 20162019 because they had an anti-dilutive impact due to the net loss incurred for the periods:

 

  As of September 30, 

 

As of June 30,

 

  2017   2016 

 

2020

 

 

2019

 

Options to purchase common stock

   3,963,480    3,220,415 

 

 

720,067

 

 

 

 

Unvested restricted common stock

   5,533    6,637 

 

 

6,957

 

 

 

 

  

 

   

 

 

 

 

727,024

 

 

 

 

   3,969,013    3,227,052 
  

 

   

 

 

Prior to the Merger the Company did not have options to purchase common stock or unvested restricted common stock to exclude from the calculation of earnings per share as all outstanding options were for common units of Holdings that upon the Merger converted into options exercisable for the shares of common stock of the Company.

Recently Issued and Adopted Accounting Pronouncements

In May 2014, 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 standard requires a company to recognize revenue when it transfers goods or services to customers in an amount 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, includingASU 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 of the nature of its promise to grant a license of intellectual property and whether or not that revenue is recognized over time or at a point in time. These new standards will be effective for 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 not currently have any revenue generating arrangements.

In FebruaryJune 2016, the FASB issued ASUNo. 2016-02,2016-13, Financial Instruments—Credit Losses (Topic 326): Measurement of Credit Losses on Financial InstrumentsLeases. This guidance will require that lease arrangements longer than 12 months result in an entity recognizing an asset and liability equalThe FASB subsequently issued amendments 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.ASU 2016-13. This standard requires a modified retrospective transition approachentities to estimate an expected lifetime credit loss on financial assets ranging from short-term trade accounts receivable to long-term financings and report credit losses using an expected losses model rather than the incurred losses model that was previously used, and establishes additional disclosures related to credit risks. For available-for-sale debt securities with unrealized losses, the standard now requires allowances to be recorded instead of reducing the amortized cost of the investment. This standard limits the amount of credit losses to be recognized for all leases existing at, or entered into after,available-for-sale debt securities to the dateamount by which carrying value exceeds fair value and requires the reversal of initial application, with an option to use certain transition relief. Early adoption is permitted.previously recognized credit losses if fair value increases. The Company is evaluatingadopted 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.2020. The adoption of this guidance also requires excess tax benefits and tax deficiencies be recorded in the income statement as opposed to additionalpaid-in capital when the awards vest or are settled. The adoption of this guidance had an immaterialstandard did not have a material impact on the Company’s condensed consolidated financial statements as of and for the three and nine months ended September 30, 2017.related disclosures.

In August 2016,2018, the FASB issued ASUNo. 2016-15,2018-13, Fair Value Measurement (Topic 820): Disclosure Framework—Changes to the Disclosure Requirements for Fair Value MeasurementStatement of Cash Flows: Classification of Certain Cash Receipts and Cash Payments. This guidance addresses the presentation and classification ofstandard modifies certain cash receipts and cash payments in the statement of cash flows. Thedisclosure requirements on fair value measurements. This standard will bebecame effective for annual periods beginningthe Company on January 1, 2020. The adoption of this standard did not have a material impact on the Company’s disclosures.

3.

Merger Accounting

On May 28, 2020, the Company completed its merger with Zafgen. Based on the Exchange Ratio, immediately following the Merger, former Zafgen stockholders, Zafgen option holders and other persons holding securities or other rights directly or indirectly convertible, exercisable or exchangeable for Zafgen Common Stock (collectively, the “Zafgen Securityholders”) owned approximately 34% of the outstanding capital stock of the combined company, and Holdings, the former Chondrial stockholder, owned approximately 66% of the outstanding capital stock of the combined company. At the closing of the Merger, all shares of Chondrial Common Stock were exchanged for an aggregate of 6,091,250 shares of Zafgen Common Stock, after December 15, 2017, and interim periods therein. Early adoption is permitted. The Company is evaluatinggiving effect to the effect that this guidance will have on its condensed consolidated financial statements.Reverse Stock Split.

In May 2017, the FASB issued ASUNo. 2017-09,Compensation – Stock Compensation: Scope of Modification Accounting. This guidance addresses which changesaddition, pursuant to the terms or conditions of a share-based payment award requirethe Merger Agreement, the Company assumed all outstanding stock options to purchase shares of Zafgen common stock at the closing of the Merger. At the closing of the Merger, such stock options became options to purchase an entityaggregate of 328,770 shares of the Company’s common stock after giving effect to apply modification accounting. the Reverse Stock Split.


The standard will be effectivetotal purchase price paid in the Merger has been allocated to the tangible and intangible assets acquired and liabilities assumed of Zafgen based on their fair values as of the completion of the Merger.Transaction costs primarily included bank fees and professional fees associated with legal counsel, auditors and printers. The following summarizes the purchase price paid in the Merger (in thousands, except share and per share amounts):

Number of shares of the combined organization owned by Zafgen stockholders(1)

 

 

3,124,337

 

Multiplied by the fair value per share of Zafgen common stock(2)

 

$

11.88

 

Fair value of consideration issued in effect of the Merger

 

$

37,119

 

Transaction costs

 

$

1,715

 

Purchase price:

 

$

38,834

 

(1)

The number of shares of 3,124,337 represents the historical 37,492,044 shares of Zafgen common stock outstanding immediately prior to the closing of the Merger, adjusted for the Reverse Stock Split.

(2)

Based on the last reported sale price of Zafgen common stock on the Nasdaq Global Market on May 28, 2020, the closing date of the Merger, and after giving effect to the Reverse Stock Split.

The allocation of the purchase price for annual periods beginning after December 15, 2017,the Merger was based on estimates of the fair value of the net assets acquired, which was then adjusted for the difference between the purchase price and interim periods therein. Early adoption is permitted.the fair value of the assets acquired. The Company is evaluatingfollowing summarizes the effect that this guidance will have on its condensed consolidated financial statements.

allocation of the purchase price to the net tangible and intangible assets acquired (in thousands):

Cash and cash equivalents

 

$

40,595

 

Marketable debt securities

 

 

1,014

 

Other current and noncurrent assets

 

 

357

 

Property and equipment, net

 

 

398

 

Restricted cash

 

 

1,339

 

Right-of-use asset

 

 

3,806

 

Current liabilities

 

 

(2,685

)

Lease liability, net of current portion

 

 

(5,990

)

Purchase price

 

$

38,834

 

3.

4.

Fair Value Measurements and Marketable Debt Securities

Fair Value Measurements

The following tables present information about the Company’s financial assets and liabilities that have beenare measured at fair value on a recurring basis as of SeptemberJune 30, 20172020 and December 31, 2016,2019 are measured in accordance with the standards of ASC 820, Fair Value Measurements and indicate theDisclosures, 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 upon the transparency of the hierarchy ofinputs 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 thean 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 and include situations where there is little, if any, market activity forcash equivalents, accounts payable and accrued liabilities. For accounts payable and accrued liabilities, the asset or liability. During the nine months ended Septembercarrying amounts of these financial instruments as of June 30, 2017, there2020 and December 31, 2019 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 SeptemberJune 30, 20172020, there were no cash equivalents and marketable debt securities as of December 31, 2016:2019:

 

   September 30, 2017 
       Quoted   Significant     
       Prices in   Other   Significant 
       Active   Observable   Unobservable 
       Markets   Inputs   Inputs 
   Total   (Level 1)   (Level 2)   (Level 3) 
   (in thousands) 

Cash equivalents:

        

Money market funds

  $13,823   $13,823   $—     $—   

Commercial paper

   3,499    —      3,499    —   
  

 

 

   

 

 

   

 

 

   

 

 

 

Total cash equivalents

   17,322    13,823    3,499    —   
  

 

 

   

 

 

   

 

 

   

 

 

 

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 in the condensed consolidated balance sheets for tax incentive receivable, accounts payable, and accrued expenses approximate fair value due to their short-term maturities. The carrying value of the Company’s outstanding notes payable approximates fair value (a Level 2 fair value measurement), reflecting interest rates currently available to the Company.

 

 

June 30, 2020

 

 

 

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

 

$

33,546

 

 

$

33,546

 

 

$

 

 

$

 

        U.S. Government securities

 

 

902

 

 

 

 

 

 

902

 

 

 

 

        Corporate bonds

 

 

1,977

 

 

 

 

 

 

1,977

 

 

 

 

             Total cash equivalents

 

 

36,425

 

 

 

33,546

 

 

 

2,879

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Marketable securities:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

        Corporate bonds

 

 

1,011

 

 

 

 

 

 

1,011

 

 

 

 

              Total marketable debt securities

 

 

1,011

 

 

 

 

 

 

1,011

 

 

 

 

   Total cash equivalents and marketable debt securities

 

$

37,436

 

 

$

33,546

 

 

$

3,890

 

 

$

 

Marketable Debt Securities

The following tables summarize the Company’s marketable debt securities as of SeptemberJune 30, 2017 and2020. There were no marketable debt securities as of December 31, 2016:2019:

 

  September 30, 2017 
      Gross   Gross   Fair
Value
 
  Amortized   Unrealized   Unrealized   

 

June 30, 2020

 

  Cost   Gains   Losses   

 

Amortized

Cost

 

 

Gross

Unrealized

Gains

 

 

Gross

Unrealized

Losses

 

 

Fair Value

 

  (in thousands) 

 

(in thousands)

 

Assets:

        

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Corporate bonds (due within 1 year)

  $58,772   $—     $(25  $58,747 

 

$

1,014

 

 

$

 

 

$

(3

)

 

$

1,011

 

Commercial paper (due within 1 year)

   14,763      (3   14,760 

Certificates of deposit (due within 1 year)

   480    —      —      480 
  

 

   

 

   

 

   

 

 

 

$

1,014

 

 

$

 

 

$

(3

)

 

$

1,011

 

  $74,015   $—     $(28  $73,987 
  

 

   

 

   

 

   

 

 

As of June 30, 2020, the Company did not have an allowance for credit losses.

5.

Prepaid Expenses and Other Current Assets

Prepaid expenses and other current assets consisted of the following:

 

   December 31, 2016 
       Gross   Gross   Fair
Value
 
   Amortized   Unrealized   Unrealized   
   Cost   Gains   Losses   
   (in thousands) 

Assets:

        

Corporate bonds (due within 1 year)

  $68,777   $—     $(54  $68,723 

Corporate bonds (due after 1 year through 2 years)

   901    —      (2   899 

Commercial paper (due within 1 year)

   27,244    —      (24   27,220 
  

 

 

   

 

 

   

 

 

   

 

 

 
  $96,922   $—     $(80  $96,842 
  

 

 

   

 

 

   

 

 

   

 

 

 

 

 

June 30,

 

 

December 31,

 

 

 

2020

 

 

2019

 

 

 

(in thousands)

 

Prepaid research and development expenses

 

$

4,926

 

 

$

3,099

 

Capitalized transaction costs

 

 

 

 

 

419

 

Research and development tax credit sale receivable

 

 

 

 

 

82

 

Payroll tax receivable

 

 

55

 

 

 

76

 

Other prepaid expenses and other assets

 

 

446

 

 

 

65

 

 

 

$

5,427

 

 

$

3,741

 

4.

Capitalized transaction costs as of December 31, 2019 consists of capitalized legal and proxy fees incurred by the Company, related to the Merger. These costs were included in the purchase price allocation when accounting for the Merger.


6.

Fixed Assets

Fixed assets, net consisted of the following:

 

 

 

 

June 30,

 

 

December 31,

 

 

 

Useful Life

 

2020

 

 

2019

 

 

 

 

 

(in thousands)

 

Computer equipment

 

5 years

 

$

41

 

 

$

14

 

Lab equipment

 

5 years

 

 

389

 

 

 

389

 

Furniture and fixtures

 

7 years

 

 

479

 

 

 

50

 

 

 

 

 

 

909

 

 

 

453

 

Less: Accumulated depreciation

 

 

 

 

(234

)

 

 

(179

)

 

 

 

 

$

675

 

 

$

274

 

Depreciation expense during the three and six months ended June 30, 2020 and 2019 was less than $0.1 million.

7.

Accrued Expenses

Accrued expenses consisted of the following as of September 30, 2017 and December 31, 2016:following:

 

 

June 30,

 

 

December 31,

 

  September 30,   December 31, 

 

2020

 

 

2019

 

  2017   2016 

 

(in thousands)

 

  (in thousands) 

Accrued research and development expenses

 

$

2,290

 

 

$

1,295

 

Accrued payroll and related expenses

  $1,525   $2,008 

 

516

 

 

119

 

Accrued research and development expenses

   1,223    892 

Accrued professional fees

   267    347 

 

526

 

 

337

 

Accrued restructuring

   —      376 

Accrued other

   75    110 

 

464

 

 

508

 

  

 

   

 

 

 

$

3,796

 

 

$

2,259

 

  $3,090   $3,733 
  

 

   

 

 


8.

Stockholders’ Equity and Stock Options

Common Stock and Prefunded warrants

5. Notes PayableAs of June 30, 2020, the Company’s Certificate of Incorporation, as amended and restated, authorized the Company to issue 115,000,000 of $0.001 par value common stock and 5,000,000 of $0.001 par value preferred stock. 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 will be 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 resulted in net proceeds of $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 Company in connection with the private placement. As partial compensation for these services, we issued MTS Health Partners 35,260 shares of our common stock.

Restricted Common Units

In November 2016, Holdings granted 123,853 Restricted Common Units of Holdings to Dr. Mark Payne (see Note 10) with an aggregate grant date fair value of approximately $0.5 million. Thirty percent (30%) of the award vested upon issuance with the remaining seventy percent (70%) vesting ratably over the next 48 months as long as services were continued to be provided as stipulated in the consulting agreement. 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 allrecognized compensation expense of the Company’s personal property, other than its intellectual property. There are no financial covenants associated with 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; and certain other business transactions.

During the three months ended September 30, 2017 and 2016, the Company recognized less than $0.1 million on a graded vesting basis in research and development expense during each of the three and six months ended June 30, 2020 and 2019. As of June 30, 2020, the Company expects to recognize less than $0.1 million respectively, of interest expense related toover the 2014 Credit Facility. During the nine months ended September 30, 2017 and 2016,remaining five-month vesting period. In accordance with ASC 718, Compensation—Stock Compensation, the Company recognized $0.2 million and $0.4 million, respectively, of interest expense related to the 2014 Credit Facility. The effective annual interest ratehas recorded costs incurred as stock-based compensation with a corresponding capital contribution from Holdings as such employees are working on behalf of the outstanding debt underCompany.

Summary of Plans

Upon completion of the 2014 Credit Facility is approximately 10.8%.

6. Stock-Based Awards

The Company grants equity awards under itsMerger with Zafgen, Zafgen’s 2014 Stock Option and Incentive Plan or the 2014 Stock Option Plan,(the “2014 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 RestatedZafgen’s 2006 Stock Option Plan but(the “2006 Plan”) were assumed by the Company. These plans are administered by the Board or, at the discretion of the Board, by a committee of the Board.

2014 Stock Option and Incentive Plan and 2006 Stock Option Plan

In 2014, the Board and shareholders of Zafgen adopted the 2014 Plan. The 2014 Plan provides for the grant of stock options, stock appreciation rights, restricted stock awards, restricted stock units, unrestricted stock awards, performance-share awards, cash-based awards and dividend equivalent rights to employees, members of the Board and consultants of the Company. The number of shares initially reserved for issuance under the 2014 Plan was 180,685 shares of common stock. The number of shares reserved for issuance may be increased by the number of shares under the previously authorized 2006 Plan that are not needed to fulfill the Company’s obligations for awards issued under the 2006 Plan as a result of forfeiture, expiration, cancellation, termination or net issuances of awards thereunder. The number of shares of common stock that may be issued under the 2014 Plan is no longer granting awards under this plan.also subject to increase on the first day of each fiscal year by the lesser of (i) 4% of the Company’s outstanding shares of common stock as of that date, or (ii) an amount determined by the Board. As of SeptemberJune 30, 2017, 2,236,7812020, 505,893 shares areof common stock were available for grant under the 2014 Stock Option Plan, including 1,093,302124,821 shares of common stock automatically added to the 2014 Stock Option Plan on January 1, 20172020.


2016 Equity and Incentive Plan

Under the 2016 Equity Plan adopted by Holdings on November 30, 2016, (the “2016 Equity Incentive Plan”), the Board of Managers of Holdings (the “Board of Managers”) or committee thereof was authorized to issue 122,133 Common Units of Holdings or combination of Common Units, Common Unit options or profit interest units. On March 23, 2018, the Board of Managers increased the number of Common Units reserved for grant and issuance pursuant to the 2016 Plan from 122,133 to 138,133 and on April 29, 2019 increased the number of Common Units reserved for grant and issuance pursuant to the 2016 Plan by an additional 101,500 to 239,633. The Company has recorded costs incurred as stock-based compensation with a resultcorresponding capital contribution from Holdings.

During the three and six months ended June 30, 2020 Holdings did not issue options to purchase Common Units to employees of the Company. During the three and six months ended June 30, 2019 Holdings issued 59,236 options to purchase Common Units to employees of the Company.

The Company assumed all of the outstanding and unexercised options to purchase units of Holdings upon consummation of the Merger. Pursuant to the terms of the Merger Agreement, options to purchase 330,818 shares of the Company’s common stock at a provision inweighted average exercise price was $12.14 per share were substituted for the 2014 Stock Option Plan.202,392 options to purchase Common Units, with a weighted average exercise price was $10.36 per Common Unit, that were outstanding immediately prior to the Merger.

The Company recorded stock-basedtreated the conversion as a modification pursuant to ASC 718, Compensation—Stock Compensation, and calculated the pre and post-modification value of the options. The increase in fair value of the options was calculated to be $1.2 million. As $0.7 million related to vested options the expense was recognized immediately and the remaining $0.5 million will be recognized over the remaining vesting term with the original grant date fair value remaining of $0.1 million.

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:

 

 

2020

 

 

2019

 

Risk-free interest rate

 

0.47%

 

 

2.00%

 

Expected term (in years)

 

 

6.25

 

 

 

6.25

 

Expected volatility

 

90%

 

 

77%

 

Dividend yield

 

0.00%

 

 

0.00%

 

Stock Options

The following table summarizes the Company’s stock option activity for the six months ended June 30, 2020:

 

 

 

 

 

 

Weighted

 

 

Average

 

 

 

 

 

 

 

 

 

 

 

Average

 

 

Remaining

 

 

Aggregate

 

 

 

Number of

 

 

Exercise

 

 

Contractual

 

 

Intrinsic

 

 

 

Shares

 

 

Price

 

 

Term

 

 

Value

 

Outstanding as of December 31, 2019

 

 

202,392

 

 

$

10.36

 

 

 

7.5

 

 

$

 

Assumed as part of the Merger with Zafgen

 

 

328,770

 

 

 

74.80

 

 

 

 

 

 

 

 

 

Modification of stock options

 

 

128,426

 

 

 

14.96

 

 

 

 

 

 

 

 

 

Granted

 

 

60,479

 

 

 

11.88

 

 

 

 

 

 

 

 

 

Outstanding as of June 30, 2020

 

 

720,067

 

 

$

40.73

 

 

 

5.4

 

 

$

0.4

 

Exercisable as of June 30, 2020

 

 

535,922

 

 

$

50.34

 

 

 

4.2

 

 

$

0.2

 

Vested and expected to vest as of June 30, 2020

 

 

720,067

 

 

$

40.73

 

 

 

5.4

 

 

$

0.4

 

July 2020 Option Grants

On July 16, 2020, the Company granted options to purchase 489,295 shares of common stock to employees under the 2014 Plan. The options have an exercise price equal to $11.90, which was the closing stock price as of the grant date, and vest over four years, with 25% vesting on the first anniversary of the grant and the remainder vesting in equal monthly installments thereafter.


In addition, on July 16, 2020 the Company granted options to purchase 735,100 shares of common stock to employees and directors under the Company’s 2020 Equity Incentive Plan (the “2020 Plan”). The 2020 Plan was approved by the Company’s Board of Directors, upon the recommendation of the Compensation Committee of the Board on July 16, 2020. The options granted to employees have an exercise price equal to $11.90, which was the closing stock price as of the grant date, 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 options granted to directors have an exercise price equal to $11.90, which was the closing stock price as of the grant date, and vest monthly in equal installments over three years. The Company plans to submit the 2020 Plan to the Company’s stockholders for approval. If the 2020 Plan is not approved by the stockholders of the Company by July 16, 2021, the 2020 Plan and the options granted thereunder will expire.

Option Grants with market-based vesting conditions

In October 2017, Zafgen granted 45,833 common stock options that vest on the third anniversary of the grant date upon achievement by the Company of minimum common stock prices for 20 consecutive days during the period between the first anniversary of the grant date and the third anniversary of the grant date. As of the Effective Time the options had not achieved the minimum common stock prices and remain unvested.

Stock-Based Compensation

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

 

 

Three Months Ended June 30,

 

 

Six Months Ended June 30,

 

 

 

2020

 

 

2019

 

 

2020

 

 

2019

 

 

 

(in thousands)

 

Research and development

 

$

199

 

 

$

17

 

 

$

211

 

 

$

36

 

General and administrative

 

 

553

 

 

 

16

 

 

 

570

 

 

 

31

 

 

 

$

752

 

 

$

33

 

 

$

781

 

 

$

67

 


As of June 30, 2020, total unrecognized compensation expense related to unvested stock options and restricted common stock units was $1.1 million, which is expected to be recognized over a weighted average period of 3.08 years.

9.

Commitments

Intellectual Property Licenses

The Company is party to a License Agreement (the “WFUHS License”), dated November 30, 2016 with Wake Forest University Health Sciences (“WFUHS”) and a 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 $2.2 million in the followingaggregate upon the achievement of certain developmental milestones, commencing on the enrollment of the first patient in a Phase 1 clinical trial. 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.

During the three months and six months ended June 30, 2020 and 2019, no milestones were achieved and no expense categories within its condensed consolidated statementswas recognized. Both agreements continue from their effective date through the last to expire 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 Contingenciesthe licensed patents unless earlier terminated by either party.

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, the Company entered into an operating lease for additional office space in Boston, Massachusetts, effective as of April 15, 2015, with an initial term expiring on July 31, 2017, and two options to extend this lease for three additional years each. In October 2015,On August 8, 2019, the Company entered into an operating lease for office space in San Diego, California,Bala Cynwyd, Pennsylvania, effective as of October 1, 2015,December 15, 2019, for a period of three years and six months with a term expiring on September 30, 2019, and an option to extend thisthe lease for fivethree additional years. In January 2017,Due to required tenant improvements to be completed by the landlord, the Company extendeddid not take possession of the leases for both office spaces in Boston, Massachusetts with new terms expiringleased property and the lease term commenced on JulyFebruary 15, 2020. In the quarter ended March 31, 2020. The Company also has the option to extend both of these leases in Boston, Massachusetts for an additional three years. With the landlord’s consent,2020, the Company has subleased 2,976recorded an operating lease right-of-use asset and operating lease liability of $0.4 million.

On May 28, 2020, as part of the Merger with Zafgen, the Company acquired a non-cancellable operating lease for approximately 17,705 square feet of office space inat 3 Center Plaza, Boston, Massachusetts to an unrelated third party which beganMassachusetts. The lease commenced on January 1, 2017June 21, 2019 and expires on September 30, 2018. The Company expects to receiveafter a term of approximately $0.2 million in sublease rental income during 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 three124 months ended September 30, 2017 and 2016, the Company recognized $0.1 millionhas an option to extend the lease for 60 additional months. As part of rental expense related to office space. During the nine months ended September 30, 2017 and 2016,agreement, the Company recognized $0.3is required to maintain a letter of credit, which upon signing was $1.3 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 inis classified as restricted cash within the course of its research and development activities. The licensing rights obligate the Company to make paymentsconsolidated financial statements. In addition to the licensors for license fees, milestones, license maintenance fees and royalties. Thebase rent, the Company is also responsible for patent prosecution costs.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 Company has ceased using the leased space and is actively seeking to obtain a subtenant. The right-of-use asset is being amortized to rent expense over the remaining lease term.

As of September 30, 2017,On November 5, 2018, the Company is obligatedentered into an operating lease for office and lab space in Philadelphia, Pennsylvania, effective as of January 1, 2019, and expiring on December 31, 2020 with an option to makeextend the lease for two additional milestone payments of up to $12.3years.

Expense arising from operating leases was $0.1 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 or nineand six months ended SeptemberJune 30, 2017 or 2016.2020 and less than $0.1 million during the three and six months ended June 30, 2019. For operating leases, the weighted-average remaining lease term for leases at June 30, 2020 and December 31, 2019 was 9.0 and 3.3 years,


respectively. For operating leases, the weighted average discount rate for leases at June 30, 2020 and December 31, 2019 was 11.0% and 12.0%, respectively. 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 sublicensefinancing leases.

Maturities of lease liabilities due under these lease agreements for the technologies.as of June 30, 2020 are as follows:

Indemnification Agreements

Year Ending December 31,

 

Operating

 

(in thousands)

Leases

 

2020 (July - December)

 

$

588

 

2021

 

 

1,177

 

2022

 

 

1,197

 

2023

 

 

1,146

 

2024

 

 

1,065

 

Thereafter

 

 

5,397

 

Total lease payments

 

 

10,570

 

Less: Imputed interest

 

 

(3,711

)

Present value of lease liabilities

 

$

6,859

 

10.

Related Party Transactions

In the ordinary course of business,November 2016, the Company may provide indemnificationentered into a consulting agreement with Mark Payne, M.D (the “Consulting Engagement”). Dr. Payne was a director of varying scopeChondrial at that time, a full-time employee of IU and terms to vendors, lessors, business partners, and other parties with respect to certain matters including, but not limited to, losses arising outone of breachthe inventors of such agreements or fromthe licensed IU intellectual property, infringement claims made by third parties. In addition, the Company has entered into indemnification agreements with membersand as such is entitled to a certain share of the management team andrevenues received by IU under the board of directors of the Company that will require the Company, among other things,IU License. Pursuant to indemnify them against certain 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 under these indemnification 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.

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 aware of any material claims as of September 30, 2017.

8. Retirement Plan

The Company has a Savings Incentive Match Plan, or SIMPLE IRA, for employees. Under the terms of the plan,his consulting agreement the Company contributes 2% of an employee’s annual base salary, upagreed to a maximumpay Dr. Payne $0.1 million per year over the term of the annual Internal Revenue Service compensation limits, for all full-time employees.

agreement and granted Dr. Payne 123,853 restricted Common Units in Holdings. On November 30, 2016, 30% vested and was associated with Chondrial Therapeutics IP, LLC (“IP LLC”) becoming a subsidiary of Holdings, which subsequently contributed to the Company on December 31, 2018. The remaining 70% is associated with future services (see Note 8) vesting ratably over 48 months beginning on December 1, 2016. The consulting agreement has a four-year term, subject to earlier termination. During the three and six months ended SeptemberJune 30, 20172020 and 2016,2019, the Company recognized less than $0.1 million, of expense related to its contributions to the plan. During the nine months ended September 30, 2017 and 2016, the Company recognized $0.1 million and $0.2 million, respectively, of expense related to its contributions to the plan.

9. Australia Research and Development Tax Incentive

The Company’s wholly owned subsidiary, Zafgen Australia Pty Limited, which conducts corethis consulting agreement, recorded as research and development activities on behalfexpense in the Statement of Operations.

The funding to the Company originated from Holdings’ sale of Series A Preferred Units and Series B convertible preferred units with Deerfield Private Design Fund IV, L.P., Deerfield Private Design Fund III, L.P. and Deerfield Health Innovations Fund, L.P. (together, the “Deerfield Funds”), and certain other purchasers, from inception through May 28, 2020 and the contribution of the proceeds received by Holdings on such sales to the Company is eligiblein order to receivefund the Company’s operations.

Under a 45% refundable tax incentiveNovember 30, 2016 Series A Preferred Unit Purchase Agreement, as amended on September 8, 2017, November 15, 2017, November 14, 2018 and April 29, 2019, Holdings sold Series A Preferred Units for qualified researchgross proceeds of $35.6 million. The gross proceeds of $35.6 million were contributed to the Company.

On November 21, 2019 (as amended on December 20, 2019), Holdings entered into a Second Amended and development activities throughRestated LLC Agreement and entered into a Series B Bridge Unit Purchase Agreement with the Deerfield Funds and certain other purchasers to sell Series B convertible preferred units (“Series B Bridge Units”) for gross proceeds of up to $10.0 million. The gross proceeds of $10.0 million were contributed to the Company.


On January 16, 2020, Holdings entered into a Third Amended and Restated LLC Agreement and entered into a Second Series B Bridge Unit Purchase Agreement with the Deerfield Funds and certain other purchasers to sell Second Series B convertible preferred units (“Second Series B Bridge Units”) for gross proceeds of up to $15.0 million. The gross proceeds of $11.4 million were contributed to the Company.

During the six months ended June 30, 2020 and the year December 31, 20162019, Holdings provided the Company non-interest bearing, permanent funding from the above Series A and 43.5% beginning January 1, 2017. For the three months ended September 30, 2017 and 2016, $0.2Series B preferred unit transactions, totaling $18.0 million and nil,$19.4 million, respectively, waswhich has been recorded as a reduction to researchcapital contributions with the balance of combined equity and development expensesadditional paid in capital on the condensed consolidated balance sheets and condensed consolidated statements of operations. Forchanges in stockholders’ equity for each respective period. No contributions were made by Holdings subsequent to the nine months ended September 30, 2017 and 2016, $0.5 million and $0.3 million, respectively, were recorded as a reductionMerger.

11.

Subsequent Events

The Company has entered into an Equity Distribution Agreement (the “ATM Program”) with an investment banking firm, pursuant to research and development expenses in the condensed consolidated statements of operations. The refund is denominated in Australian dollars and, therefore, the related receivable isre-measured into U.S. dollars as of each reporting date. For the three months ended September 30, 2017 and 2016,which the Company recorded in its condensed consolidated statements of operations unrealized foreign currency exchange gains of less than $0.1 million related to this tax incentive receivable. For the nine months ended September 30, 2017 and 2016, the Company recorded in its condensed consolidated statements of operations unrealized foreign currency exchange gains of less than $0.1 million and $0.1 million, respectively, related to this tax incentive receivable. As of September 30, 2017 and December 31, 2016, the Company’s tax incentive receivable from the Australian government was $0.5 million and $0.3 million, respectively.

10. Restructuring

On July 19, 2016, the Company announced that following a comprehensive reviewmay sell shares of its assets and clinical programs,common stock through the investment banker as well as feedback from regulatory authorities, thesales agent for aggregate proceeds of up to $50.0 million. The Company refocused its resources on development of a differentiated MetAP2 inhibitor,ZGN-1061. As part of the strategic restructuring, the Company reorganized its operations to align with its new priorities focused onZGN-1061 development. The Company’s workforce was reduced by approximately 31% as 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 
  

 

 

   

 

 

 

11. Subsequent Events

In October 2017, the Company granted its newly appointed Chief Executive Officer two inducement stock option grants, which werehas not grantedsold any shares 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.ATM Program.

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, 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, 20162019 included in our AnnualCurrent Report onForm 10-K for8-K filed with the year ended December 31, 2016.Securities and Exchange Commission (“SEC”), on June 2, 2020, as amended on June 26, 2020. 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. Asassumptions. 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 forthnew information, future events or otherwise. You should read the “Risk Factors” section included in our Current Report on Form 8-K filed with the ‘‘Risk Factors’’ sectionSEC on June 2, 2020, as amended on June 26, 2020, and the "Risk Factors" and “Forward-Looking Statements” sections of this Quarterly Report ouron Form 10-Q for a discussion of important factors that could cause actual results couldto 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 conditionthe 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.

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.

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.analysis.

Overview

We are a biopharmaceuticalclinical-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, isZGN-1061, a novel fumagillin-class MetAP2 inhibitorsubcutaneously 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 patients are unable to produce enough FXN due to a genetic abnormality. There is currently no effective therapy for Friedreich’s Ataxia. CTI-1601 is currently being profiledevaluated in Phase 1 clinical trials in patients with Friedreich’s Ataxia. We have received orphan drug status, fast track designation and rare pediatric disease designation, from the U.S. Food and Drug Administration (the “FDA”), for its utilityCTI‑1601. In addition, the European Medicines Agency (“EMA”) Committee for Orphan Medicinal Products (“COMP”) issued a positive opinion on the Company’s application for orphan drug designation for CTI-1601. 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 diabetesother rare and other related metabolic disorders.orphan diseases. We have completed a Phase 1 clinical trialintend to use our proprietary platform to target additional orphan indications characterized by deficiencies in or alterations ofZGN-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 assessmentZGN-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 thatZGN-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 advancedZGN-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 ofre-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 innon-clinical safety studies relative to beloranib for effects on coagulation in dogs, an effect that correlates with reduced impact on endothelial cell proliferationin vitro.

Since our inception, in November 2005, we have devoted substantially all of our resources to developingZGN-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 our inception through our initial public offering, or 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. 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 afollow-on offering of our common stock, with net proceeds of $130.0 million after deducting underwriting discounts and commissions paid by us.

We have never generated any revenue and have, to date, incurred net losses. We incurred net losses in each year since our inception. We haveof approximately $18.0 million and $8.4 million for the six months ended June 30, 2020, and 2019, respectively. As of June 30, 2020, we had an accumulated deficit of $276.5$41.1 million asand a cash, cash equivalents and marketable debt securities balance of September 30, 2017. Our net loss was $38.9 million for the nine months ended September 30, 2017 and $57.9 million for the year ended December 31, 2016.$113.7 million. These losses have resulted principally from costs incurred in connection within-licensing of beloranib, research and development activities, in-licensing of technology 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:

Continue to advance the development ofZGN-1061 CTI-1601 through Phase 2 and later-stageadditional clinical trials;

seekSeek to identify and advance development of additional product candidates into clinical development and indications for our product candidates;

seekSeek to obtain regulatory approvalsapproval for our product candidates;


Identify, acquire or in-license other product candidates and technologies;

Maintain, leverage and expand our intellectual property portfolio; and

add

Expand our operational, financial and management information systems;

addsystems and personnel, including personnel to support our productclinical development and future commercialization;commercialization efforts and

maintain, leverage and expand our intellectual property portfolio.operations as a public company.

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 equity, 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 ourits 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 expectbelieve that, based on our existingcurrent operating plan, our cash and cash equivalents and marketable securities as of September 30, 2017,the filing date will enable us to fund our operating expenses and capital expenditure requirementsoperations for a period of at least one yeartwelve months from the issuance of these interim financial statements.

Merger with Zafgen

On December 17, 2019, we entered into an Agreement and Plan of Merger (the “Merger Agreement”) with Zordich Merger Sub, Inc. (“Merger Sub”), our wholly-owned subsidiary, Chondrial Therapeutics Inc. (“Chondrial”), and Chondrial Holding, LLC (“Holdings”) pursuant to which the Merger Sub would merge with and into Chondrial, with Chondrial surviving the merger as our wholly owned subsidiary (the “Merger”). The Merger was completed on May 28, 2020 pursuant to the terms of the Merger Agreement.

Pursuant to the terms of the Merger Agreement, upon closing of the Merger, all of Chondrial’s outstanding common stock was exchanged for our common stock and all outstanding options exercisable for units of Holdings were exchanged for options to purchase our common stock. In addition, immediately prior to the closing of the Merger, we effected a 1 for 12 reverse stock split (the “Reverse Stock Split”) and changed our name from Zafgen, Inc. to Larimar Therapeutics, Inc. Following the Merger, the business conducted by Chondrial became our primary business.

The business combination was accounted for as a reverse acquisition in accordance with U.S. generally accepted accounting principles (“GAAP”). Under this method of accounting, Chondrial was deemed to be the accounting acquirer for financial reporting purposes. This determination was primarily based on the facts that, immediately following the merger: (i) Chondrial’s stockholders own a substantial majority of the voting rights in the combined company, (ii) the majority of the board of directors of the combined company is composed of directors designated by Chondrial under the terms of the Merger Agreement and (iii) existing members of Chondrial management will be the management of the combined company. Accordingly, for accounting purposes, the business combination was treated as the equivalent of Chondrial issuing stock to acquire Zafgen’s net assets. As a result, as of the closing date of the Merger, Zafgen’s net assets were recorded at their acquisition-date fair values, which were then adjusted for the difference between the purchase price and the fair value of the assets acquired, in the financial statements of Chondrial and the reported operating results prior to the business combination are those of Chondrial. As the Merger has been accounted for as an asset acquisition, goodwill has not been recorded within the condensed combined balance sheet.

Private Placement

On May 28, 2020, we entered into a Securities Purchase Agreement with certain accredited investors for the sale by us in a private placement of 6,105,359 shares of our common stock (the “Private Placement Shares”), and pre-funded warrants to purchase an aggregate of 628,403 shares of our common stock (the “Pre-funded Warrants”). The Pre-Funded Warrants are immediately exercisable at an exercise price for $0.01 and are exercisable indefinitely. We refer to this Quarterly Report. See “—Liquiditysale herein as the Private Placement.


The Private Placement closed on June 1, 2020. The aggregate gross proceeds for the issuance and Capital Resources.”sale of the Private Placement Shares and Pre-Funded common stock Warrants were $80.0 million and, after deducting certain of our expenses, the net proceeds we received in the Private Placement were $75.4 million. We intend to use the net proceeds from the Private Placement for research and development of our product candidates, working capital and general corporate purposes.

Financial Operations Overview

Revenue

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 nearforeseeable 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.

Operating Expenses

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

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:

personnelemployee related costs, including salaries, related benefits and stock-based compensation expenses for employees engaged in scientific research and development functions;

third-party contract costs relating to research, formulation, manufacturing, preclinicalnonclinical studies and clinical trial activities;

external costs of outside consultants;

payments made under our third-party licensing agreements;

sponsored research agreements;

laboratory consumables; and

allocated facility-related costs.

We are currently primarily focused on developingZGN-1061 and other early research activities and typically use our employee, consultant and infrastructure resources across our researchResearch and development programs. We track outsourced development costs are expensed as incurred. Costs for certain activities, such as manufacturing, nonclinical studies and clinical trials are generally recognized based on the evaluation of the progress of completion of specific tasks using information and data provided 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 researchvendors 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 candidatesWe expect to increase our investment in later stages of clinicalresearch and development generally have higher development costs than those in earlier stages of clinical development, primarily dueorder to the increased size and duration of later-stageadvance 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 ourCTI-1601 or any other product candidates.candidates we develop.

WeAt this time, we cannot determine with certaintyreasonably estimate or know the durationnature, timing and completionestimated costs of the currentefforts that will be necessary to complete the development of CTI-1601 or future clinical trials of ourany other product candidates orwe develop. We are also unable to predict when, if when, or to what extent weever, material net cash inflows will generate revenuecommence from the commercialization and sale of any of our product candidates that obtain regulatory approval. We may never succeed in achieving regulatory approval for anysales of our product candidates. The duration, costs, and timing of clinical trials and development of ourCTI-1601 or any other product candidates we develop will depend on a variety of factors, including:

the scope, rate of progress and expense of clinical trials and other research and development activities, including the ongoing impact of COVID-19 on these activities;

clinical trial results;

uncertainties in clinical trial enrollment rate or design;

significant and changing government regulation;

the timing and receipt of any regulatory approvals; and

the U.S. Food and Drug Administration, or FDA’s or other regulatory authority’s influence on clinical trial design.design;


establishing manufacturing capabilities or making arrangements with third-party manufacturers and risk involved with development of manufacturing processes, FDA pre-approval inspection practices and successful completion of manufacturing batches for clinical development and other regulatory purposes;

obtain and maintain patent and trade secret protection and regulatory exclusivity for our product candidates; and

our ability to retain key research and development personnel.

A change in the outcome of any of these variables with respect to the development of a product candidate could mean a significantsignificantly change in the costs, timing and timingviability associated with the development of that product candidate. For example, if the FDA, or another regulatory authority were to require us to conduct 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

General and administrative expenses consist primarily of personnel costs, consisting of salaries, related benefits and stock-based compensation, of our executive, finance, business and corporate developmentinformation technology, and 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 of our intellectual property. We expect that our general and administrative expenses will decrease during 2017increase in the foreseeable future as comparedwe hire additional employees to 2016 primarily due to reduced headcountimplement and improve our operational, financial and management systems. Additionally, as a publicly-traded company, we will incur significant additional legal, accounting and other expenses that we did not incur as a privately-held company.

Critical Accounting Policies and Significant Judgments and Estimates

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

Other Income (Expense)

Interest income. Interest income consistsdisclosures requires us to make estimates and assumptions that affect the reported amount of interest earnedassets, 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 equivalentscondensed 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, consistingResearch and Development Expenses

As part of the stated interestprocess of 8.1% per year duepreparing our condensed consolidated financial statements, we are required to estimate our accrued research and development expenses. 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, onfinalpre-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:

CROs, in connection with clinical trials;

vendors in connection with nonclinical development activities;

contract manufacturing organizations in connection with the production of preclinical and clinical trial materials; and

vendors related to product candidate manufacturing, development and distribution of clinical supplies.

We base our expenses related to clinical trials on our estimates of the services received and efforts expended pursuant to contracts with multiple CROs that conduct and manage clinical trials 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 recordedthe clinical expense, nonclinical 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 consistsclinical 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. If the actual timing of the realizedperformance of services or the level of effort varies from our estimate, we adjust the accrual or prepaid accordingly. Although we do not expect our estimates to be materially different from amounts actually incurred, our understanding of the status and unrealized gainstiming of services performed relative to the actual status and lossestiming of services performed may vary and may result in us recognizing adjustments in future periods as additional information becomes available.

Stock-Based Compensation

We measure all stock-based awards granted to employees, non-employee consultants and directors 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 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, we had been 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 historical volatility of publicly traded peer companies and expect to continue to do so until we have 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. 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 the fact that we have never paid cash dividends on common stock and do not expect to pay any cash dividends in the foreseeable future.

COVID-19 Update

In March 2020, the World Health Organization declared the outbreak of COVID-19, a novel strain of Coronavirus, a global pandemic. This outbreak is causing major disruptions to businesses and markets worldwide as the virus spreads. The extent of the effect on our operational and financial performance will depend on future developments, including the duration, spread and intensity of the pandemic, and governmental, regulatory and private sector responses, all of which are uncertain and difficult to predict. Although we are unable to estimate the financial effect of the pandemic at this time, if the pandemic continues to evolve into a severe worldwide crisis, it could have a material adverse effect on our business, results of operations, financial condition and cash flows. The financial statements do not reflect any adjustments as a result of the pandemic.

The pandemic resulted in the temporary stoppage of our CTI-1601 in a single ascending dose (referred to as “SAD”) Phase 1 clinical trial in patients with Friedreich’s Ataxia. We have since resumed the SAD Phase 1 clinical trial in July 2020. We are conducting the clinical trial at one clinical trial site. Because Friedreich’s Ataxia is a rare disease, there are a limited number of patients in close proximity to the clinical trial site and clinical trial patients travel from foreign currency-denominated cash balances, vendor payablesthroughout the United States to the clinical trial site to participate. After dosing, patients remain in isolation in the clinical research unit for a period of time. The travel advisories andtax-related receivables risk of infection related to COVID-19 have presented increased risks to patients traveling to our clinical trial site for dosing and we expect to incur additional clinical trial costs to safely transport and isolate patients participating in the trial. In addition, additional stoppages or delays in the trial could result from new developments with respect to COVID-19. While top line results from the Australian government.SAD and the planned multiple ascending dose (referred to as “MAD”) ongoing Phase 1 clinical trials were originally expected by the end of 2020, the delay in the clinical trial timeline caused by the ongoing impact of COVID-19 resulted in top line results now being expected in the first half of 2021. We currently do not engagemay experience additional delays in hedging activitiesclinical trial timelines as a result of additional travel and hospital restrictions related to our foreign currency-denominated receivables and payables;the


COVID-19 pandemic which may be imposed, including as such, we cannot predict the impacta result of future foreign currency transaction gains and losses on our operating results. See “—Quantitative and Qualitative Disclosures about Market Risk.”

Income Taxes

Since our inceptionresurgences of COVID-19 cases 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.certain geographic areas.

Results of Operations

Comparison of the Three Months Ended Septemberthree months ended June 30, 20172020 and 20162019

The following table summarizes our results of operations for the three months ended SeptemberJune 30, 20172020 and 2016:2019:

 

   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 June 30,

 

 

 

 

 

 

 

 

 

 

 

Increase

 

 

 

2020

 

 

2019

 

 

(Decrease)

 

 

 

(in thousands)

 

Statement of Operations Data:

 

 

 

 

 

 

 

 

 

 

 

 

Operating expenses:

 

 

 

 

 

 

 

 

 

 

 

 

Research and development

 

$

8,907

 

 

$

3,128

 

 

$

5,779

 

General and administrative

 

 

2,492

 

 

 

576

 

 

 

1,916

 

Total operating expenses

 

 

11,399

 

 

 

3,704

 

 

 

7,695

 

Loss from operations

 

 

(11,399

)

 

 

(3,704

)

 

 

(7,695

)

Other income, net

 

 

69

 

 

 

 

 

 

69

 

Net loss

 

$

(11,330

)

 

$

(3,704

)

 

$

(7,626

)

Research and development expenses

   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
  

 

 

   

 

 

   

 

 

 

Research and development expenses for the three months ended SeptemberJune 30, 2017 decreased $0.32020 increased $5.8 million compared to the three months ended SeptemberJune 30, 2016. 2019. The decreaseincrease was primarily due to a $3.3$5.0 million decreaseincrease in external development costs for CTI-1601, a $0.4 million increase in personnel related costs due to headcount additions in our beloranib programresearch and development functions and a decrease$0.2 million increase in stock-based compensation due to the modification of $0.5stock-options that converted from common unit options in Holdings to options to purchase our common stock. The $5.0 million associated with our unallocated expenses, partially offset by increasedincrease in external development costs was primarily attributed to incremental costs incurred for the further development of $1.9 million related to work on discovery and screening and an increase of $1.7 million related to ourZGN-1061 program.

Costs associated with our discovery and screening programs increased $1.9 million overCTI-1601. Specifically, during late 2019 the prior period as during the 2017 period we had considerable costs related to discovery work for our early stage programs.

Costs associated with ourZGN-1061 program increased period over period by $1.7 million. In the third quarter of 2016 we initiatedfirst patients were dosed in a Phase 1 SAD clinical trial forZGN-1061 which completed recruiting and dosing patients inof CTI-1601. The trial was ongoing during the first quarter of 2017. The overall increase is primarily2020 but was paused due to additional preclinicalissues related to COVID-19. During July 2020, we resumed the SAD clinical trial. In addition, there was an increase in third-party manufacturing of CTI-1601 and toxicology 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 expensesCTI-1601 during the 2016 period were for the Phase 1 clinical trial ofZGN-1061three months ended June 30, 2020. 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 andclose-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

   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
  

 

 

   

 

 

   

 

 

 

General and administrative expenses for the three months ended SeptemberJune 30, 2017 decreased $1.72020 increased $1.9 million compared to the three months ended SeptemberJune 30, 2016.2019. The decreaseincrease was due to decreases in personnel related costs of $1.2 million, professional fees of $0.2 million andnon-cash stock-based compensation expense of $0.2 million. Personnel related expenses decreased primarily due to a reduction$1.0 million increase 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. Professionalprofessional fees decreasedthat is primarily due to loweran increase in accounting and audit fees due primarily to the Merger, an increase in legal fees as the plaintiffs for the class action lawsuit had filedassociated with intellectual property filings and costs of being 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.

Other income (expense), net

Interest expense. Interest expense forpublic company during the three months ended SeptemberJune 30, 2017 and 20162020. The increase was also the result of less than $0.1a $0.5 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 yearincrease in stock-based compensation due on outstanding borrowings. It also includes expense related to the final paymentmodification of 6%stock-options that converted from common unit options in Holdings to options to purchase our common stock.


Results of amounts drawn down that is being recorded over the term through the maturity date using the effective-interest method and the amortizationOperations

Comparison of deferred financing costs and debt discounts relating to the 2014 Credit Facility.

Interest income. Interest income of $0.3 million and $0.2 million, respectively, for the threesix months ended SeptemberJune 30, 20172020 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 andtax-related receivables from the Australian government, generally reflecting the fluctuation of the Australian dollar relative to the U.S. dollar.

Results of Operations

Comparison of the Nine Months Ended September 30, 2017 and 20162019

The following table summarizes our results of operations for the ninesix months ended SeptemberJune 30, 20172020 and 2016:2019:

 

   Nine Months Ended September 30, 
           Increase 
   2017   2016   (Decrease) 
   (in thousands) 

Statement of Operations Data:

      

Revenue

  $—     $—     $—   
  

 

 

   

 

 

   

 

 

 

Operating expenses:

      

Research and development

   29,928    32,661    (2,733

General and administrative

   9,713    15,089    (5,376
  

 

 

   

 

 

   

 

 

 

Total operating expenses

   39,641    47,750    (8,109
  

 

 

   

 

 

   

 

 

 

Loss from operations

   (39,641   (47,750   8,109 
  

 

 

   

 

 

   

 

 

 

Other income (expense):

      

Interest income

   740    664    76 

Interest expense

   (157   (432   275 

Foreign currency transaction gains (losses), net

   115    79    36 
  

 

 

   

 

 

   

 

 

 

Total other income (expense), net

   698    311    387 
  

 

 

   

 

 

   

 

 

 

Net loss

  $(38,943  $(47,439  $8,496 
  

 

 

   

 

 

   

 

 

 

 

 

Six Months Ended June 30,

 

 

 

 

 

 

 

 

 

 

 

Increase

 

 

 

2020

 

 

2019

 

 

(Decrease)

 

 

 

(in thousands)

 

Statement of Operations Data:

 

 

 

 

 

 

 

 

 

 

 

 

Operating expenses:

 

 

 

 

 

 

 

 

 

 

 

 

Research and development

 

$

13,914

 

 

$

7,350

 

 

$

6,564

 

General and administrative

 

 

4,159

 

 

 

1,078

 

 

 

3,081

 

Total operating expenses

 

 

18,073

 

 

 

8,428

 

 

 

9,645

 

Loss from operations

 

 

(18,073

)

 

 

(8,428

)

 

 

(9,645

)

Other income, net

 

 

69

 

 

 

 

 

 

69

 

Net loss

 

$

(18,004

)

 

$

(8,428

)

 

$

(9,576

)

Research and development expenses

   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
  

 

 

   

 

 

   

 

 

 

Research and development expenses for the ninesix months ended SeptemberJune 30, 2017 decreased $2.72020 increased $6.6 million compared to the ninesix months ended SeptemberJune 30, 2016. 2019. The decreaseincrease was primarily due to a $12.1$5.4 million decreaseincrease in external development costs for CTI-1601 and a $0.7 million increase in personnel related costs due to headcount additions in our beloranib programresearch and a decrease of $3.1development functions. The $5.4 million associated with our unallocated expenses partially offset by increased costs of $9.6 million related to ourZGN-1061 program, as well as an increase in discovery and screening expensesexternal development costs was primarily attributed to incremental costs incurred for the further development of $3.7 million.

Costs associated with ourZGN-1061 program increased period over period by $9.6 million. In the third quarter of 2016 we initiated the Phase 1 clinicalCTI-1601. The trial forZGN-1061 which completed recruiting and dosing patients inwas ongoing during the first quarter of 2017. The overall increase is primarily2020 but was paused due to additional preclinical studies as well as drug product and drug substance activities asissues related to COVID-19. During July 2020, we commenced our Phase 2 clinical trial inresumed the third quarter of 2017 in both Australia and New Zealand, for which we plan to enroll 120 patients in a 12 weekSAD clinical trial. The expensesIn addition, there was in an increase in third-party manufacturing of CTI-1601 and toxicology studies for CTI-1601 during the 2016 period were for startup costs for the Phase 1 clinical trial ofZGN-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 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 ninesix months ended SeptemberJune 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.2020.

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

   Nine Months Ended September 30, 
           Increase 
   2017   2016   (Decrease) 
   (in thousands) 

Personnel related

  $1,761   $3,848   $(2,087

Non-cash stock-based compensation

   3,570    5,219    (1,649

Professional fees

   3,048    4,446    (1,398

Other

   1,334    1,576    (242
  

 

 

   

 

 

   

 

 

 

Total general and administrative expenses

  $9,713   $15,089   $(5,376
  

 

 

   

 

 

   

 

 

 

General and administrative expenses for the ninesix months ended SeptemberJune 30, 2017 decreased $5.42020 increased $3.1 million compared to the ninesix months ended SeptemberJune 30, 2016. 2019. The decreaseincrease was due to decreases in personnel related costs of $2.1 million,non-cash 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 stock option grant price for the 2017 annual grant. Professional fees decreased primarily due to lowera $2.1 million increase in professional fees that is primarily due to an increase in accounting and audit fees due to the Merger, an increase in legal fees asassociated with intellectual property filings and costs of being a public company during the plaintiffs forsix months ended June 30, 2020. The increase was also the class action lawsuit had filedresult of a notice of appeal$0.5 million increase in stock-based compensation due to the First Circuit Courtmodification of Appeals and on April 7, 2017, the dismissal with prejudice was affirmed. The decrease was also duestock-options that converted from common unit options in Holdings to lower commercial readiness costs and investor relations and public relations costs in the 2017 period as comparedoptions to the same period in 2016, primarily as a result of suspending development of beloranib in July 2016.

Other income (expense), net

Interest expense. Interest expense for the nine months ended September 30, 2017 and 2016 of $0.2 million and $0.4 million, respectively, was related to interest expense onpurchase 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.

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 andtax-relatedcommon stock. receivables from the Australian government, generally reflecting the fluctuation of the Australian dollar relative to the U.S. dollar.

Liquidity and Capital Resources

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. Fromsignificant 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 afollow-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 bydevoted substantially all of our personal property, other thanresources 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:

 

   Nine Months Ended
September 30,
 
   2017   2016 
   (in thousands) 

Cash used in operating activities

  $(33,948  $(43,309

Cash provided by investing activities

   22,957    34,534 

Cash used in financing activities

   (2,168   (1,959
  

 

 

   

 

 

 

Net decrease in cash and cash equivalents

  $(13,159  $(10,734
  

 

 

   

 

 

 

 

 

Six Months Ended June 30,

 

 

 

2020

 

 

2019

 

 

 

(in thousands)

 

Net cash used in operating activities

 

$

(21,120

)

 

$

(8,965

)

Net cash provided by (used in) investing activities

 

 

40,643

 

 

 

(33

)

Net cash provided by financing activities

 

 

93,480

 

 

 

5,990

 

Net increase (decrease) in cash, cash equivalents and restricted cash

 

$

113,003

 

 

$

(3,008

)


Net cash used in operating activities

During the ninesix months ended SeptemberJune 30, 2017,2020, operating activities used $33.9$21.1 million of cash, resulting from our net loss of $38.9$18.0 million partially offset by, adjusted for noncash expenses of $0.8 million and changes in our operating assets and liabilities of $1.2 million andnon-cash$4.0 million. charges of $6.2 million. Our net loss was primarily attributed to research and development activities related to ourZGN-1061 CTI-1601 program and our general and administrative expenses. Our netnon-cash charges during the nine months ended September 30, 2017, consistedexpenses as described above. Noncash expenses primarily ofinclude stock-based compensation expense of $6.2 million. Net cash usedexpense. The change in changes in our operating assets and liabilities during the nine months ended September 30, 2017, consistedwas primarily of a $0.6 million decrease in accrued expenses as well asdue to an increase in accounts payable, accrued expenses and prepaid expenses and other current assets of $0.6 million.due to the growth in our operating activities.

During the ninesix months ended SeptemberJune 30, 2016,2019, operating activities used $43.3$9.0 million of cash, resulting from our net loss of $47.4$8.4 million, adjusted for noncash expenses of $0.1 million and changes in our operating assets and liabilities of $5.0$0.6 million partially offset bynon-cash charges of $9.1 million.. Our net loss was primarily attributed to research and development activities related to our beloranibCTI-1601 program ourZGN-1061 program, and our general and administrative expenses. Our netnon-cash charges during the nine months ended September 30, 2016, consisted primarily of stock-based compensation expense of $8.1 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.expenses as described above.

Net cash provided by (used in) investing activities

During the ninesix months ended SeptemberJune 30, 2017,2020, investing activities provided $23.0$40.6 million of cash, resulting from proceedsa $41.9 million increase from salesour Merger, which was offset by transaction costs associated with the Merger of $1.2 million and maturities$0.1 million from the purchase of marketable securities of $112.2 million, offset primarily by the use of cash for purchases of marketable securities of $89.2 million.equipment.

During the ninesix months ended SeptemberJune 30, 2016,2019, investing activities provided $34.5used less than $0.1 million of cash resulting from proceeds from sales and maturities of marketable securities of $141.4 million, offset by the use of cash for purchases of marketable securities of $106.2 million and purchases of property and equipment of $0.7 million.laboratory equipment.

Net cash used inprovided by financing activities

During the ninesix months ended SeptemberJune 30, 2017,2020, financing activities used $2.4provided $93.5 million for payments related to our notes payable, partially offset by $0.2 million receivedof cash that was the result of the proceeds from proceeds relating to the exercisesale of common stock options and prefunded common stock purchased under our 2014 Employee Stock Purchase Plan.warrants, net of issuance costs, from the Private Placement of $75.5 million and contributions from Holdings of $18.0 million.

During the ninesix months ended SeptemberJune 30, 2016,2019, net cash provided by financing activities used $2.2of $6.0 million for payments related to our notes payable, partially offset by $0.2 million receivedwas the result of contributions from proceeds relating to the exercise of common stock options and common stock purchased under our 2014 Employee Stock Purchase Plan.Holdings.

Operating Capital Requirements

ZGN-1061 has completedCTI-1601 is currently in Phase 1 clinical development, therefore we expect to continue to incur significant expenses and operating losses for the foreseeable future. We anticipate that we will continue to incur expenses, if and as we:

we seek to:

Continue to advance the development ofZGN-1061 CTI-1601 through Phase 2additional clinical development and if successful, later-stagetrials, including the cost of clinical trials;materials as well as manufacturing scale up costs;

seekSeek to identify and advance development of additional product candidates into clinical development and indications for our product candidates;

seekSeek to obtain regulatory approvals for our product candidates;

Identify, acquire or in-license other product candidates and technologies;

add

Maintain, leverage and expand our intellectual property portfolio; and

Expand our operational, financial and management information systems;

addsystems and personnel, including personnel to support our productclinical development and future commercialization;commercialization efforts and

maintain, leverage and expand our intellectual property portfolio.operations as a public company.

We expect to continue to generate operating losses for the foreseeable future. We completed the Merger on May 28, 2020 which, upon closing, provided cash, cash equivalents, restricted cash and marketable debt securities of $42.9 million concurrent with the Private Placement which provided additional net proceeds of $75.4 million. We believe that, based on our existingcurrent operating plan, our cash, cash equivalents and marketable debt securities as of September 30, 2017,the filing date, we will enable usbe able to fund our operating expenses and capital expenditure requirementsoperations for a period of at least one year from the issuance date of this Quarterly Report. We have based this estimate on assumptions that may prove to be wrong, and we may use 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 forZGN-1061 will depend on many factors, including:twelve months.

the costs, timing and outcome of regulatory review;

the costs of future research and development activities, including clinical trials;

the costs of preparing, filing and prosecuting patent applications, maintaining and enforcing our intellectual property rights and defending intellectual property-related claims;

the extent to which we acquire orin-license other products, product candidates, or technologies; and

our ability to establish any future collaboration arrangements on favorable terms, if at all.

Until such time, if ever, as we can generate substantial product revenue, we expect to finance our cash needsseek additional funding through a combination of equity offerings, debt financings, other third-party funding, marketing and distribution arrangements, and other 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 liquidation or other preferences that adversely affect the rights of our common stockholders. Debt financing, if available, 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 and may require the issuance of warrants, which could potentially dilute their ownership interest. If 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 thatWe may not be favorableable to us. If we are unable to raise additional funds through equity,obtain financing on acceptable terms, or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to developat all, and marketZGN-1061 that we would otherwise prefer to develop and market ourselves.

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.enter into collaborations or other arrangements.


Contractual Obligations and Commitments

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 Form10-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 financialThe terms of any financing may adversely affect the lease extensions for bothholdings or our existing stockholders’ rights. If we are unable to obtain additional funding, we will be forced to delay, reduce or eliminate some or all 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 2018research and 2019 and $0.2 million for 2020.development programs, product portfolio expansion or commercialization efforts, which would adversely affect our business, or we may be unable to continue operations.

Off-Balance Sheet Arrangements

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 CommissionSEC 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.

Application of Critical Accounting Policies

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 onForm 10-K for the year ended December 31, 2016, the following accounting policies involve the most judgment and complexity:

accrued research and development costs; and

stock-based compensation.

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.

Recently Issued Accounting Pronouncements

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.

Other Company Information

None.

Item 3.

Quantitative and Qualitative DisclosureDisclosures about Market Risk

Interest Rate Fluctuation RiskNot applicable.

Our cash, cash equivalents, and marketable securities 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 affected by changes in the general level 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 Exchange Risk

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.

Item 4.

Management’s Evaluation of our Disclosure Controls and Procedures

We maintain disclosure controls and procedures (as defined in Rules13a-15(e) or15d-15(e) under the Securities Exchange Act of 1934, as amended, or the Exchange Act) that are designed to ensure that information required to be disclosed 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 rules and forms and (2) accumulated and communicated to our management, including our principal executive officer and principal financial officer, as appropriate, to allow timely decisions regarding required disclosure.

Based on this evaluation, ourOur management, with the participation of our principal executive officer and principal financial officer, evaluated the effectiveness of our disclosure controls and procedures as of the end of the period covered by this report. In designing and evaluating our disclosure controls and procedures, our management recognizes that any controls and procedures, no matter how well designed and operated, can provide only reasonable assurance of achieving their objectives, and management necessarily applies its judgment in evaluating the cost-benefit relationship of possible controls and procedures.

Our principal executive officer and principal financial officer have concluded based upon the evaluation described above that as of SeptemberJune 30, 2017,2020, our disclosure controls and procedures were not effective at the reasonable assurance level.level due the material weakness in internal control over financial reporting previously disclosed in our Form 8-K/A dated June 26, 2020.

Notwithstanding the identified material weaknesses, management, including our principal executive officer and principal financial officer, have determined, based on the procedures we have performed, that the condensed consolidated financial statements included in this Quarterly Report on Form 10-Q fairly represent in all material respects our financial condition, results of operations and cash flows at June 30, 2020 and for the periods presented in accordance with U.S. GAAP.

Previously identified material weaknesses in internal control over financial reporting

As part of the audit of our consolidated financial statements as of and for the years ended December 31, 2019 and 2018, we identified material weaknesses in our internal control over financial reporting. A material weakness is a deficiency, or a combination of deficiencies, in internal control over financial reporting, such that there is a reasonable possibility that a material misstatement of our financial statements will not be prevented or detected on a timely basis. If we are unable to remediate these material weaknesses, or if we identify additional material weaknesses in the future or otherwise fail to maintain an effective system of internal controls, we may not be able to accurately or timely report our financial condition or results of operations.


The material weaknesses we identified were as follows:

We did not maintain an effective control environment commensurate with our financial reporting requirements. We lacked a sufficient number of professionals with an appropriate level of accounting and controls knowledge, training and experience to appropriately analyze, record and disclose accounting matters timely, completely and accurately. Additionally, the limited personnel resulted in our inability to consistently establish appropriate authorities and responsibilities in pursuit of our financial reporting objectives, as demonstrated by, amongst other things, our insufficient segregation of duties in our finance and accounting functions. This material weakness contributed to the following material weakness.

We did not design and maintain adequate controls over the preparation and review of certain account reconciliations and journal entries. Specifically, we did not design and maintain controls to ensure (i) appropriate segregation of duties in the preparation and review of account reconciliations and journal entries, and (ii) account reconciliations and journal entries were reviewed at the appropriate level of precision. This material weakness resulted in adjustments to prepaid expenses and accrued expenses which were identified and recorded as part of the audit of our consolidated financial statements as of and for the years ended December 31, 2019 and 2018.

Each of these control deficiencies could result in a misstatement of our accounts or disclosures that would result in a material misstatement of our consolidated financial statements that would not be prevented or detected, and accordingly, we determined these control deficiencies constitute material weaknesses.

Remediation Plan

We are in the process of implementing measures designed to improve our internal control over financial reporting and remediate the control deficiencies that led to these material weaknesses, including hiring additional finance and accounting personnel and initiating design and implementation of controls to enhance our internal controls over financial reporting including the establishment of formal accounting policies and procedures. In particular, we have hired a Chief Financial Officer and Vice President, Controller and retained as consultants certain finance and accounting personnel that were previously employed by Zafgen during the second quarter of 2020, to supplement our accounting and finance department during a transition period.

We believe the measures described above will remediate the control deficiencies we have identified and strengthen our internal control over financial reporting. We are committed to continuing to improve our internal control processes and will continue to review, optimize and documentenhance our disclosure financial reporting controls and procedures, includingprocedures. As we continue to evaluate and work to improve our internal controls and procedures for control over financial reporting, we may take additional measures to address control deficiencies, or we may modify certain of the remediation measures described above. The material weakness will not be considered remediated until the applicable controls operate for a sufficient period of time and may from time to time make changes aimed at enhancing their effectiveness and to ensuremanagement has concluded, through testing, that our systems evolve with our business.these controls are operating effectively.

Changes in Internal Control Over Financial Reporting

During the three months ended September 30, 2017,Except as described above, there have been no changes in our internal control over financial reporting (as defined in Rules13a-15(f) or15d-15(d) underduring the Exchange Act)three months ended June 30, 2020 that have materially affected, or are reasonably likely to materially affect, our internal control over financial reporting.


PART II – OTHER- OTHER INFORMATION

Item 1.

Legal Proceedings

On October 21, 2015, a purported stockholder of the Company filed a putative class action lawsuitFrom 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 claimsproceedings arising in the ordinarynormal course of business, the resolution of which we do not anticipate wouldbusiness. To our knowledge, there are no threatened or pending legal actions that could reasonably be expected to have a material adverse impacteffect on our business, financial position,condition, results of operations or cash flows.

Item 1A.

Risk Factors

InvestingThere have been no material changes to the risk factors described in our common stock involves a high degree of risk. You should carefully considerCurrent Report on Form 8-K filed with the SEC on June 2, 2020, as amended on June 26, 2020. The risks described below, as well as the other information in this Quarterly Report onForm 10-Q, or Quarterlyour Annual Report and insuch Current Report on Form 8-K are not the only risks facing our other public filings before making an investment decision. Our business, prospects, financial condition, or operating results could be harmed by any of these risks, as well as other risksCompany. Additional risk 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 forZGN-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:

the FDA may not accept our IND forZGN-1061 or may put it on clinical hold;

we may not be able to demonstrate thatZGN-1061 is safe and effective to the satisfaction of the FDA and the European Medicines Agency, or EMA;

the results of our clinical trials may not meet the level of statistical or clinical significance required by the FDA and EMA for marketing approval;

the FDA and EMA may disagree with the number, design, size, duration, conduct or implementation of our clinical trials;

the FDA and EMA may require that we conduct additional clinical trials or preclinical studies;

the FDA and EMA may not approve the formulation, labeling or specifications ofZGN-1061;

the contract research organizations, or CROs, that we retain to conduct our clinical trials may take actions outside of our control that materially adversely impact our clinical trials;

the FDA and EMA may find the data from preclinical studies and clinical trials insufficient to demonstrate thatZGN-1061’s clinical and other benefits outweigh its safety risks;

the FDA and EMA may disagree with our interpretation of data from our preclinical studies and clinical trials;

the FDA and EMA may not accept data generated at our clinical trial sites;

if and when our NDA is submitted and reviewed by an FDA advisory committee, the FDA may have difficulties scheduling an advisory committee meeting in a timely manner or the advisory committee may recommend against approval of our application or may recommend that the FDA require, as a condition of approval, additional preclinical studies or clinical trials, limitations on approved labeling or distribution and use restrictions;

the FDA could require development of a REMS as a condition of approval or post-approval, or may not agree with our proposed REMS, or may impose additional requirements that limit the promotion, advertising, distribution, or sales ofZGN-1061;

the FDA and EMA may find deficiencies with or not approve the manufacturing processes or facilities of third-party manufacturers with which we contract; or

the FDA and EMA may change their approval policies or adopt new regulations.

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 thatZGN-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 thatZGN-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 ofZGN-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 ofZGN-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 ofZGN-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 withZGN-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:

regulatory authorities may request that we withdraw the product from the market or may limit their approval of the product through labeling or other means;

regulatory authorities may require the addition of labeling statements, such as a “black box” warning or a contraindication or a precaution;

we may be required to change the way the product is distributed or administered, conduct additional clinical trials or change the labeling of the product;

we may decide to remove the products from the marketplace;

we could be sued and held liable for injury caused to individuals exposed to or taking our product candidates; and

our reputation may suffer.

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 ofZGN-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:

the FDA, EMA or other governing bodies in Europe or Australia and New Zealand may deny permission to pursue clinical trials and/or indications we want to initiate;

delays in regulatory filings or receiving regulatory approvals of INDs, or clinical trial authorization applications, or CTAs, that may be required;

unfavorable results from our preclinical and /ornon-clinical studies, the FDA, the EMA or the applicable regulatory authorities in Australia or New Zealand, may require additional preclinical and /ornon-clinical studies;

delays in reaching or failing to reach agreement on acceptable terms with prospective CROs and clinical trial sites, the terms of which can be subject to extensive negotiation and may vary significantly among different CROs and trial sites;

inadequate quantity or quality of a product candidate or other materials necessary to conduct clinical trials, for example delays in the manufacturing of sufficient supply of finished drug product;

difficulties obtaining Institutional Review Board, or IRB, and/or ethics committee approval to conduct a clinical trial at a prospective site or sites in the United States, the European Union, Australia or New Zealand;

challenges in recruiting and enrolling patients to participate in clinical trials, including the size and nature of the patient population, the proximity of patients to clinical trial sites, eligibility criteria for the clinical trial, the nature of the clinical trial protocol, the availability of approved effective treatments for the relevant disease and competition from other clinical trial programs for similar indications;

severe or unexpected drug-related side effects experienced by patients in a clinical trial, including side effects previously identified in our previous clinical trials for beloranib;

the FDA, the EMA, or the applicable regulatory authorities in Australia and New Zealand may disagree with our clinical trial designs, our interpretation of data from clinical trials, or may change the requirements for approval even after it has reviewed and commented on the design for our clinical trials;

difficulties in retaining or recruiting clinical investigators and/or patients in our ongoing or future clinical trials;

reports from preclinical,non-clinical or clinical testing of other weight loss therapies that raise safety or efficacy concerns; and

difficulties retaining patients who have enrolled in a clinical trial but may be prone to withdraw due to rigors of the clinical trial, lack of efficacy, side effects, screening and monitoring measures, personal issues or loss of interest.

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:

failure to conduct the clinical trial in accordance with regulatory requirements or our clinical protocols;

inspection of the clinical trial operations or trial sites by the FDA, the EMA, or the applicable regulatory authorities in Australia and New Zealand that reveals deficiencies or violations that require us to undertake corrective action, including the imposition of a partial clinical hold or a full clinical hold;

unforeseen safety issues, including any that could be identified in ournon-clinical or preclinical studies, adverse side effects or lack of effectiveness;

changes in government regulations or administrative actions;

problems with clinical supply materials; and

lack of adequate funding to continue the clinical trial.

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 forZGN-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:

have staffing difficulties;

fail to comply with contractual obligations;

experience regulatory compliance issues;

undergo changes in priorities or become financially distressed; or

form relationships with other entities, some of which may be our competitors.

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 theto-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 ofZGN-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 marketZGN-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:

the relative convenience and ease of subcutaneous injections as the necessary method of administration ofZGN-1061;

the prevalence and severity of any adverse side effects associated withZGN-1061;

limitations or warnings contained in the labeling approved forZGN-1061 by the FDA, EMA, or other regulatory authorities, such as a “black box” warning;

availability of alternative treatments, including a number of competitive type 2 diabetes therapies already approved or expected to be commercially launched in the near future;

the willingness of the target patient population to try new therapies and of physicians to prescribe these therapies;

the strength of marketing and distribution support and timing of market introduction of competitive products;

publicity concerning our products or competing products and treatments;

pricing;

the effectiveness of our sales and marketing strategies;

our ability to increase awareness ofZGN-1061 through marketing efforts;

our ability to obtain sufficient third-party coverage or reimbursement;

the willingness of patients to payout-of-pocket in the absence of third-party coverage; and

the likelihood that the FDA may require development of a REMS, as a condition of approval or post-approval or may not agree with our proposed REMS or may impose additional requirements that limit the promotion, advertising, distribution or sales ofZGN-1061.

IfZGN-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 withZGN-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:

issue warning letters or untitled letters;

seek an injunction or impose civil or criminal penalties or monetary fines;

suspend or withdraw marketing approval;

suspend any ongoing clinical trials;

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

suspend or impose restrictions on operations, including costly new manufacturing requirements; or

seize or detain products, refuse to permit the import or export of products, or request that we initiate a product recall.

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 makeZGN-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 toZGN-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:

our customers’ ability to obtain reimbursement forZGN-1061 in foreign markets;

our inability to directly control commercial activities because we are relying on third parties;

the burden of complying with complex and changing foreign regulatory, tax, accounting and legal requirements;

different medical practices and customs in foreign countries affecting acceptance in the marketplace;

import or export licensing requirements;

longer accounts receivable collection times;

longer lead times for shipping;

language barriers for technical training;

reduced protection of intellectual property rights in some foreign countries;

foreign currency exchange rate fluctuations; and

the interpretation of contractual provisions governed by foreign laws in the event of a contract dispute.

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 distributeZGN-1061, if we obtain marketing approval. Restrictions under applicable federal and state healthcare laws and regulations include the following:

The federal Anti-Kickback Statute prohibits, among other things, persons from knowingly and willfully soliciting, offering, receiving or paying remuneration, directly or indirectly, in cash or in kind, to induce or reward either the referral of an individual for, or the purchase, order or recommendation of, any good or service, for which payment may be made under federal healthcare programs such as Medicare and Medicaid.

The federal false claims laws impose criminal and civil penalties, including those from civil whistleblower or qui tam actions pursuant to the federal False Claims Act, against individuals or entities for knowingly presenting, or causing to be presented, to the federal government, claims for payment that are false or fraudulent or making a false statement to avoid, decrease, or conceal an obligation to pay money to the federal government.

The federal Health Insurance Portability and Accountability Act of 1996, as amended by the Health Information Technology for Economic and Clinical Health Act, imposes criminal and civil liability for executing a scheme to defraud any healthcare benefit program and also imposes obligations, including mandatory contractual terms, with respect to safeguarding the privacy, security and transmission of individually identifiable health information.

The federal false statements statute prohibits knowingly and willfully falsifying, concealing or covering up a material fact or making any materially false statement in connection with the delivery of or payment for healthcare benefits, items or services.

The federal transparency requirements, sometimes referred to as the “Sunshine Act,” under the Patient Protection and Affordable Care Act, require manufacturers of drugs, devices, biologics, and medical supplies to report to the Department of Health and Human Services information related to physician payments and other transfers of value and physician ownership and investment interests.

Analogous state laws and regulations, such as state anti-kickback and false claims laws and transparency laws, may apply to sales or marketing arrangements and claims involving healthcare items or services reimbursed bynon-governmental third-party payors, including private insurers, and some state laws require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government in addition to requiring drug manufacturers to report information related to payments to physicians and other healthcare providers or marketing expenditures and drug pricing.

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 forZGN-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 inoff-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 ofZGN-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 forZGN-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 ofZGN-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 ofZGN-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 ofZGN-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 ofZGN-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.

Risks Relating to Our Intellectual Property Rights

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 ofZGN-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 toZGN-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 commercializeZGN-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 coveringZGN-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 coveredZGN-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:

any of our patents, or any of our pending patent applications, if issued, will include claims having a scope sufficient to protectZGN-1061 or any other products or product candidates;

any of our pending patent applications will issue as patents;

we will be able to successfully develop and commercializeZGN-1061, if approved, before our relevant patents expire;

we were the first to make the inventions covered by each of our patents and pending patent applications;

we were the first to file patent applications for these inventions;

others will not develop similar or alternative technologies that do not infringe our patents;

any of our patents will be found to ultimately be valid and enforceable;

any patents issued to us will provide a basis for an exclusive market for our commercially viable products, will provide us with any competitive advantages or will not be challenged by third parties;

we will develop additional proprietary technologies or product candidates that are separately patentable; or

that our commercial activities or products will not infringe upon the patents of others.

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 havenon-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 commercializingZGN-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:

cease developing, selling or otherwise commercializingZGN-1061;

pay substantial damages for past use of the asserted intellectual property;

obtain a license from the holder of the asserted intellectual property, which license may not be available on reasonable terms, if at all; and

in the case of trademark claims, redesign or rename the trademarks or trade names of our product candidates to avoid infringing the intellectual property rights of third parties, which may not be possible and, even if possible, could be costly and time-consuming.

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 fornon-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 ornon-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 includere-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 commercializeZGN-1061 and other product candidates, which would materially adversely affect our business, financial condition and resultsor future results.

Item 2.

Unregistered Sales of Equity Securities and Use of Proceeds

There have been no sales of operations.

General Company-Related Risks

In 2016, we reducedunregistered securities other than as previously disclosed by 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 ourday-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 weaknessesCompany in our infrastructureCurrent Reports on Form 8-K as filed with the SEC, and operations, risks that we may not be ableas set forth below:

MTS Health Partners served as placement agent 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, commercializeZGN-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 commercializeZGN-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 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 anykey-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 standardnon-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 complyconnection 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 precautionsPrivate Placement. As partial compensation for these services, 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 withZGN-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:

withdrawal of patients from our clinical trials;

substantial monetary awards to patients or other claimants;

decreased demand forZGN-1061 or any future product candidates following marketing approval, if obtained;

damage to our reputation and exposure to adverse publicity;

increased FDA warnings on product labels;

litigation costs;

distraction of management’s attention from our primary business;

loss of revenue; and

the inability to successfully commercializeZGN-1061 or any future product candidates, if approved.

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 forZGN-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 priceissued MTS Health Partners 35,260 shares 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 The issuance of realizing a benefitthese securities were exempt 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. Underregistration under Section 3824(a)(2) of the Internal Revenue CodeSecurities Act or Regulation D promulgated thereunder, in that the transaction was by an issuer not involving any public offering.

Our Registration Statement on Form S-3, filed with the SEC on June 26, 2020, registered the resale of 1986, as amended, or the Code, changes in our ownership may limit the amount35,260 shares 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. Ourfollow-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 ourfollow-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 studyissued to assess whetherMTS Health Partners.

Item 3.

Defaults Upon Senior Securities

None.

Item 4.

Mine Safety Disclosures

Not applicable.

Item 5.

Other Information

On August 14, 2020, we entered into 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 associatedEquity Distribution Agreement (the “Agreement”) 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 forZGN-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 ofZGN-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 otherday-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.

Risks Related to Our Financial Position and Need for Capital

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 developingZGN-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 incurredPiper Sandler & Co. (“Piper Sandler”), 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 levelsthe 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 choosesell 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:

initiate and successfully complete clinical trials that meet their clinical endpoints;

initiate and successfully complete all safety studies required to obtain U.S. and foreign marketing approval forZGN-1061 in the indications we are pursuing;

commercializeZGN-1061, if developed and approved, by developing a sales force or entering into collaborations with third parties; and

achieve market acceptance ofZGN-1061 in the medical community and with third-party payors.

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 forZGN-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 toZGN-1061 or other product candidates, our intellectual property, future revenue streams or grant licenses on terms that are not favorable to us.

Risks Related to Our Common Stock

We expect that our stock price may fluctuate significantly.

The market price$50,000,000 of shares of our common stock similar(the “ATM Shares”) from time to time through Piper Sandler, as sales agent (the “Offering”).

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, 2017Agreement, 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:

plansparameters for progress of, or results from preclinical studies and clinical trials ofZGN-1061 and/or other product candidates;

the failure of the FDA to accept our IND forZGN-1061;

the failure of the FDA or the EMA to approveZGN-1061;

our ability to establish an adequate safety margin and profile forZGN-1061 or other product candidates, including risk of serious thromboembolic events;

announcements of new products, technologies, commercial relationships, acquisitions or other events by us or our competitors;

the success or failure of other type 2 diabetes or weight loss therapies;

regulatory or legal developments in the United States and other countries;

failure ofZGN-1061, if successfully developed and approved, to achieve commercial success;

fluctuations in stock market prices and trading volumes of similar companies;

general market conditions and overall fluctuations in U.S. equity markets;

variations in our quarterly operating results;

changes in our financial guidance or securities analysts’ estimates of our financial performance;

changes in accounting principles;

our ability to raise additional capital and the terms on which we can raise it;

sales of large blocks of our common stock, including sales by our executive officers, directors and significant stockholders;

additions or departures of key personnel;

discussion of us or our stock price by the press and by online investor communities; and

other risks and uncertainties described in these risk factors.

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:

delaying, deferring or preventing a change of control of us;

impeding a merger, consolidation, takeover or other business combination involving us; or

discouraging a potential acquirer from making a tender offer or otherwise attempting to obtain control of us.

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 declinesold in the marketany 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 stockholderbelow 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 appeal 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 ofATM Shares, if 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 fornon-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, thereAgreement 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 rulesSecurities Act. We pay Piper Sandler a commission equal to 3.0% of the SEC.

gross proceeds of any ATM Shares sold through Piper Sandler under the Agreement and have agreed to reimburse the Piper Sandler for certain specified expenses. The Agreement contains customary representations, warranties and agreements by us, indemnification obligations of ourselves and Piper Sandler, 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 onno obligation to sell any of our common stock to datethe ATM Shares, and we currently intend to retain all of our future earnings, ifmay at any to fundtime suspend offers under the development and growth of our business. As a result, capital appreciation, if any, of our common stockAgreement.

The ATM Shares will be offered and sold pursuant to the sole sourceCompany’s Registration Statement on Form S-3 filed by the Company on August 14, 2020 (the “Registration Statement”) and the sales agreement prospectus that forms a part of gains for our common stockholders forsuch Registration Statement, following such time as the foreseeable future. Consequently,Registration Statement is declared effective by the Securities and Exchange Commission.


The description of the Agreement does not purport to be complete and is qualified in its entirety by reference to the foreseeable future, our common stockholdersAgreement, a copy of which will likely only experience a gain from their investment in our common stock ifbe filed with the price of our common stock increases.SEC as an exhibit to the Registration Statement.

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 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:

 

creating a classified board of directors whose members serve staggered three-year terms;

authorizing “blank check” preferred stock, which could be issued by our board of directors without stockholder approval and may contain voting, liquidation, dividend, and other rights superior to our common stock;

limiting the liability of, and providing indemnification to, our directors and officers;

limiting the ability of our stockholders to call and bring business before special meetings;

requiring advance notice of stockholder proposals for business to be conducted at meetings of our stockholders and for nominations of candidates for election to our board of directors;

controlling the procedures for the conduct and scheduling of board of directors and stockholder meetings; and

providing our board of directors with the express power to postpone previously scheduled annual meetings and to cancel previously scheduled special meetings.

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

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.

Item 6.

Exhibits

Exhibits

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

EXHIBIT INDEX

 

Exhibit

No.

Description

    3.1

Certificate of Amendment of Ninth Amended and Restated Certificate of Incorporation Of Zafgen, Inc. related to the Reverse Stock Split, dated May 28, 2020 (incorporated by reference to Exhibit 3.1 of the Company’s Current Report on Form 8-K filed on June 2, 2020).

    3.2

Certificate of Amendment of Ninth Amended and Restated Certificate of Incorporation Of Zafgen, Inc. related to the Name Change, dated May 28, 2020 (incorporated by reference to Exhibit 3.1 of the Company’s Current Report on Form 8-K filed on June 2, 2020).

    4.1

Form of Company Pre-funded Warrant to Purchase Common Stock by and among the Company and certain investors (incorporated by reference to Exhibit 4.1 of the Company’s Current Report on Form 8-K filed on June 2, 2020).

  10.1

Securities Purchase Agreement, dated as of May 28, 2020, by and among the Company and the investors listed on the Schedule of Investors attached thereto (incorporated by reference to Exhibit 10.1 of the Company’s Current Report on Form 8-K filed on June 2, 2020).

  10.2

Registration Rights Agreement, dated as of June 1, 2020, by and among the Company and certain Investors (incorporated by reference to Exhibit 10.2 of the Company’s Current Report on Form 8-K filed on June 2, 2020).

  10.3

Registration Rights Agreement, dated as of June 8, 2020, by and among the Company and certain Investors (incorporated by reference to Exhibit 10.3 of the Company’s Registration Statement on Form S-3 filed on June 26, 2020).

  10.4

Form of Indemnification Agreement between the Company and its directors (incorporated by reference to Exhibit 10.3 of the Company’s Current Report on Form 8-K filed on June 2, 2020).

  10.5

Employment Agreement, dated June 1, 2020, by and between the Company and Michael Celano (incorporated by reference to Exhibit 10.4 of the Company’s Current Report on Form 8-K filed on June 2, 2020).

  10.6*+

License Agreement, by and between the Company and Wake Forest University Health Sciences, effective as of November 30, 2016.

  10.7*+

Amendment 1 to License Agreement, by and between the Company and Wake Forest University Health Sciences, effective as of November 28, 2017.

  10.8*+

Amendment 2 to License Agreement, by and between the Company and Wake Forest University Health Sciences, effective as of March 29, 2019.

  10.9*+

License Agreement, by and between the Company and Indian University Research and Technology Corporation, effective as of November 30, 2016.

  10.10*+

First Amendment to License Agreement, by and between the Company, the Trustee of Indiana University and Indiana University Research and Technology Corporation, effective as of August 16, 2019.

  10.11

Notice of Substitute Option Grant between the Company and a certain Optionee (incorporated by reference to Exhibit 4.5 of the Company’s Registration Statement on Form S-8 filed on June 26, 2020).


  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*

XBRL Instance Document.

101.SCH*

XBRL Taxonomy Extension Schema Document.

101.CAL*

XBRL Taxonomy Extension Calculation Document.

101.DEF*

XBRL Taxonomy Extension Definition Linkbase Document.

101.LAB*

XBRL Taxonomy Extension Labels Linkbase Document.

101.PRE*

XBRL Taxonomy Extension Presentation Link Document.

 

*

Filed herewith.

**

Furnished herewith.

+

Certain confidential portions (indicated by brackets and asterisks) have been omitted from this exhibit.


SIGNATURESSIGNATURES

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.

 

LARIMAR THERAPEUTICS, INC.

ZAFGEN, INC.

Date: August 14, 2020

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, 2017August 14, 2020

By:

By:

/s/ Patricia L. AllenMichael Celano

Patricia L. Allen

Michael Celano

Chief Financial Officer

(Principal Financial and Accounting Officer)

 

5336