Document and Entity Information
Document and Entity Information - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2021 | Mar. 23, 2022 | Jun. 30, 2021 | |
Cover [Abstract] | |||
Document Type | 10-K | ||
Amendment Flag | false | ||
Document Period End Date | Dec. 31, 2021 | ||
Document Fiscal Year Focus | 2021 | ||
Document Fiscal Period Focus | FY | ||
Trading Symbol | LRMR | ||
Entity Registrant Name | LARIMAR THERAPEUTICS, INC. | ||
Entity Central Index Key | 0001374690 | ||
Current Fiscal Year End Date | --12-31 | ||
Entity Filer Category | Non-accelerated Filer | ||
Entity Small Business | true | ||
Entity Emerging Growth Company | false | ||
Entity Common Stock, Shares Outstanding | 17,710,450 | ||
Entity Current Reporting Status | Yes | ||
Entity Shell Company | false | ||
Entity File Number | 001-36510 | ||
Entity Tax Identification Number | 20-3857670 | ||
Entity Address, Address Line One | Three Bala Plaza East | ||
Entity Address, Address Line Two | Suite 506 | ||
Entity Address, City or Town | Bala Cynwyd | ||
Entity Address, State or Province | PA | ||
Entity Address, Postal Zip Code | 19004 | ||
City Area Code | 844 | ||
Local Phone Number | 511-9056 | ||
Title of 12(b) Security | Common Stock, par value $0.001 per share | ||
Security Exchange Name | NASDAQ | ||
Entity Interactive Data Current | Yes | ||
Document Annual Report | true | ||
Document Transition Report | false | ||
Entity Incorporation State Country Code | DE | ||
Entity Well-known Seasoned Issuer | No | ||
Entity Voluntary Filers | No | ||
Entity Public Float | $ 94.4 | ||
ICFR Auditor Attestation Flag | false | ||
Documents Incorporated by Reference | Part III of this Annual Report on Form 10-K incorporates certain information by reference from the registrant’s proxy statement for the 2022 annual meeting of shareholders to be filed no later than 120 days after the end of the registrant’s fiscal year ended December 31, 2021. | ||
Auditor Firm ID | 238 | ||
Auditor Name | PricewaterhouseCoopers LLP | ||
Auditor Location | Philadelphia, Pennsylvania, USA |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 |
Current assets: | |||
Cash and cash equivalents | $ 70,097 | $ 68,148 | |
Marketable debt securities | 24,490 | ||
Prepaid expenses and other current assets | 2,107 | 5,314 | |
Total current assets | 72,204 | 97,952 | |
Property and equipment, net | 1,049 | 1,040 | |
Operating lease right-of-use assets | 3,406 | 3,936 | |
Restricted cash | 1,339 | 1,339 | |
Other assets | 669 | 419 | |
Total assets | 78,667 | 104,686 | |
Current liabilities: | |||
Accounts payable | 1,660 | 2,634 | |
Accrued expenses | 6,592 | 5,843 | |
Operating lease liabilities, current | 594 | 515 | |
Total current liabilities | 8,846 | 8,992 | |
Operating lease liabilities | 5,408 | 6,002 | |
Total liabilities | 14,254 | 14,994 | |
Commitments and contingencies (See Note 9) | |||
Stockholders’ equity: | |||
Preferred stock; $0.001 par value per share; 5,000,000 shares authorized as of December 31, 2021 and December 31, 2020; no shares issued and outstanding as of December 31, 2021 and December 31, 2020 | |||
Common stock, $0.001 par value per share; 115,000,000 shares authorized as of December 31, 2021 and December 31, 2020; 17,710,450 and 15,367,730 shares issued and outstanding as of December 31, 2021 and December 31, 2020, respectively | 18 | 15 | |
Additional paid-in capital | 180,645 | 155,290 | |
Accumulated deficit | (116,250) | (65,614) | |
Accumulated other comprehensive gain | 1 | ||
Total stockholders' equity | 64,413 | 89,692 | $ (694) |
Total liabilities and stockholders' equity | $ 78,667 | $ 104,686 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - $ / shares | Dec. 31, 2021 | Dec. 31, 2020 |
Statement of Financial Position [Abstract] | ||
Preferred stock, par value per share | $ 0.001 | $ 0.001 |
Preferred stock, shares authorized | 5,000,000 | 5,000,000 |
Preferred stock, shares issued | 0 | 0 |
Preferred stock, shares outstanding | 0 | 0 |
Common stock, par value per share | $ 0.001 | $ 0.001 |
Common stock, shares authorized | 115,000,000 | 115,000,000 |
Common stock, shares issued | 17,710,450 | 15,367,730 |
Common stock, shares outstanding | 17,710,450 | 15,367,730 |
Consolidated Statements of Oper
Consolidated Statements of Operations and Comprehensive Loss - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Operating expenses: | ||
Research and development | $ 38,396 | $ 31,407 |
General and administrative | 12,069 | 11,397 |
Total operating expenses | 50,465 | 42,804 |
Loss from operations | (50,465) | (42,804) |
Other income (expense), net | (171) | 322 |
Net loss | $ (50,636) | $ (42,482) |
Net loss per share, basic | $ (2.95) | $ (3.57) |
Net loss per share, diluted | $ (2.95) | $ (3.57) |
Weighted average common shares outstanding, basic | 17,164,284 | 11,883,155 |
Weighted average common shares outstanding, diluted | 17,164,284 | 11,883,155 |
Comprehensive income (loss): | ||
Net loss | $ (50,636) | $ (42,482) |
Other comprehensive loss: | ||
Unrealized gain (loss) on marketable debt securities | (1) | 1 |
Total other comprehensive income (loss) | (1) | 1 |
Total comprehensive loss | $ (50,637) | $ (42,481) |
Consolidated Statements of Chan
Consolidated Statements of Changes in Stockholders' Equity (Deficit) - USD ($) $ in Thousands | Total | Common Stock [Member] | Additional Paid-in Capital [Member] | Accumulated Deficit [Member] | Accumulated Other Comprehensive Loss [Member] |
Balances at Dec. 31, 2019 | $ (694) | $ 6 | $ 22,432 | $ (23,132) | |
Beginning balance, shares at Dec. 31, 2019 | 6,091,250 | ||||
Capital contributions from related party | 17,995 | 17,995 | |||
Merger with Zafgen Inc. | 37,119 | $ 3 | 37,116 | ||
Merger with Zafgen Inc., Shares | 3,124,337 | ||||
Private Placement, net of transaction costs | 74,850 | $ 6 | 74,844 | ||
Shares issued to private placement, Shares | 6,105,359 | ||||
Issuance of Common Stock to placement agent for advisory fees in lieu of cash and other issuances of common stock | 742 | 742 | |||
Issuance of common stock to placement agent for advisory fees in lieu of cash and other issuances of common stock, Shares | 46,784 | ||||
Stock-based compensation expense | 2,161 | 2,161 | |||
Unrealized gain (loss) on marketable debt securities | 1 | $ 1 | |||
Net loss | (42,482) | (42,482) | |||
Balances at Dec. 31, 2020 | 89,692 | $ 15 | 155,290 | (65,614) | 1 |
Ending balance, shares at Dec. 31, 2020 | 15,367,730 | ||||
Issuance of Common Stock. Net | 19,885 | $ 3 | 19,882 | ||
Issuance of Common Stock. Net, shares | 2,342,720 | ||||
Stock-based compensation expense | 5,473 | 5,473 | |||
Unrealized gain (loss) on marketable debt securities | (1) | $ (1) | |||
Net loss | (50,636) | (50,636) | |||
Balances at Dec. 31, 2021 | $ 64,413 | $ 18 | $ 180,645 | $ (116,250) | |
Ending balance, shares at Dec. 31, 2021 | 17,710,450 |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Cash flows from operating activities: | ||
Net loss | $ (50,636) | $ (42,482) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Stock-based compensation expense | 5,473 | 2,161 |
Gain on disposal of fixed asset | (1) | |
Depreciation expense | 326 | 155 |
Amortization of premium (discount) on marketable securities | (14) | 10 |
Changes in operating assets and liabilities: | ||
Prepaid expenses and other current assets | 3,207 | (1,647) |
Accounts payable | (974) | (3,288) |
Accrued expenses | 749 | 3,232 |
Right-of-use assets | 530 | 405 |
Operating lease liabilities | (515) | (427) |
Other assets | (250) | (318) |
Net cash used in operating activities: | (42,105) | (42,199) |
Cash flows from investing activities: | ||
Purchase of property and equipment | (333) | (62) |
Purchase of marketable debt securities | (8,248) | (24,486) |
Maturities and sales of marketable debt securities | 32,750 | 1,000 |
Cash, cash equivalents, and restricted cash acquired in connection with the Merger | 41,934 | |
Merger transaction costs | (1,296) | |
Net cash provided by investing activities | 24,169 | 17,090 |
Cash flows from financing activities: | ||
Capital contributions from related party | 17,995 | |
Proceeds from sale of common stock and prefunded warrants, net of issuance costs | 19,885 | 75,592 |
Net cash provided by financing activities | 19,885 | 93,587 |
Net increase in cash, cash equivalents and restricted cash | 1,949 | 68,478 |
Cash, cash equivalents and restricted cash at beginning of period | 69,487 | 1,009 |
Cash, cash equivalents and restricted cash at end of period | $ 71,436 | 69,487 |
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) | |
Property and equipment included in accounts payable and accrued expenses | 460 | |
Leased assets obtained in exchange for new operating lease liabilities | $ 448 |
Consolidated Statements of Ca_2
Consolidated Statements of Cash Flows (Parenthetical) $ in Millions | 12 Months Ended |
Dec. 31, 2020USD ($) | |
Statement of Cash Flows [Abstract] | |
Marketable debt securities acquired | $ 1 |
Description of Business and Org
Description of Business and Organization | 12 Months Ended |
Dec. 31, 2021 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Description of Business and Organization | 1. Description of Business and Organization Larimar Therapeutics, Inc., together with its subsidiaries (the “Company” or “Larimar”), is a clinical-stage biotechnology company focused on developing treatments for patients suffering from complex rare diseases using its novel cell penetrating peptide technology platform. Larimar's lead product candidate, CTI-1601, is a subcutaneously administered, 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 sufficient FXN due to a genetic abnormality. In May 2021, Larimar reported positive topline data from its Phase 1 Friedreich’s ataxia, ("FA") program after completing dosing of the single ascending dose, ("SAD"), trial in December 2020 and of the multiple ascending dose, ("MAD"), trial in March 2021. Data from these trials demonstrate proof-of-concept by showing that daily subcutaneous injections of CTI-1601 for up to 13 days resulted in dose-dependent increases in FXN levels from baseline compared to placebo in all evaluated tissues (buccal cells, skin, and platelets). FXN levels achieved in peripheral tissues (buccal cells) following daily 50 mg and 100 mg subcutaneous injections of CTI-1601 were at or in excess of FXN levels that would be expected in phenotypically normal heterozygous carriers. There were no serious adverse events, ("SAEs"), associated with either the MAD or SAD trials. To support extended dosing of patients with CTI-1601, we conducted a 26-week NHP toxicology study in 2021. In May 2021 we notified the FDA of certain mortalities which occurred at the highest dose levels in the then-ongoing study. On May 25, 2021 the FDA placed a clinical hold on the CTI-1601 clinical program. In the clinical hold letter, the FDA stated that it needed to review a full study report from the then-ongoing NHP study and that we may not initiate additional interventional clinical trials until we have submitted such report and received notification from the FDA that additional clinical trials may commence. At the time of the FDA clinical hold, we had no interventional clinical trials with patients enrolled or enrolling and as of the date of this report, do not have interventional clinical trials with patients enrolled or enrolling. The Company submitted a complete response to the clinical hold in January 2022. In February 2022, the Company received feedback from the FDA following its submission of the complete response. The FDA stated that it was maintaining its clinical hold at this time and requested additional information. The Company is evaluating how best to respond to their request. The Company does not know when, or if, the clinical hold will be lifted. The Company is subject to risks and uncertainties common to pre-commercial companies in the biotechnology industry, including, but not limited to, development by competitors of new technological innovations, dependence on key personnel, protection of proprietary technology, compliance with governmental regulations, failure to secure regulatory approval for its drug candidates or any other product candidates and the ability to secure additional capital to fund its operations. Drug candidates currently under development will require extensive non-clinical and clinical testing and regulatory approval prior to commercialization. These efforts require significant amounts of additional capital, adequate personnel, and infrastructure and extensive compliance-reporting capabilities. Even if our drug development efforts are successful, it is uncertain when, if ever, we will realize significant revenue from product sales. In March 2020, the World Health Organization ("WHO"), declared the outbreak of COVID-19, a novel strain of Coronavirus, a global pandemic. The pandemic resulted in the temporary stoppage of the Company’s CTI-1601 Phase 1 clinical trials in patients with Friedreich’s ataxia in March 2020. In July 2020, the Company resumed these clinical trials, and has since completed both of the Company's SAD and MAD clinical trials. Since being discovered, new variants of COVID-19 have emerged. In November 2021, the Omicron variant was identified in South Africa, and has been deemed a variant of concern by the WHO. In addition to the Omicron variant, the WHO has deemed four other variants to be variants of concern: the Alpha variant, the Beta variant, the Gamma variant, and the Delta variant. All of the aforementioned variants have at least one of the following characteristics resulting in the WHO deeming them variants of concern: an increase in transmissibility or detrimental change in COVID-19 epidemiology, an increase in virulence or change in clinical disease presentation, or a decrease in effectiveness of public health and social measures or available diagnostics, vaccines, or therapeutics. Vaccines manufactured by Moderna, Pfizer and Johnson & Johnson were introduced late in the fourth quarter of 2020 and became widely available by the end of the first quarter of 2021. While the vaccines have proven effective in reducing the severity and mortality of COVID-19, including the variants that have evolved to date, the overall vaccination rate has not reached the level required for herd immunity in some areas of the country. Further, additional doses of vaccines may be required and individuals may refuse or fail to receive one or more such doses. The incidence of variants of COVID-19 has been increasing, particularly among unvaccinated individuals, and the Omicron variant has proven to be more easily spread than earlier variants. The low vaccination rate, the spread of the variants and the evolution of additional mutations against which the current vaccines may prove ineffective could again result in major disruptions to businesses and markets worldwide. Furthermore, the incidence of "breakthrough" infections even among vaccinated individuals have been increasing, resulting in the need for booster doses which could make COVID-19 a long-term infectious disease concern. The Company’s business, results of operations, financial condition and cash flows could be materially and adversely affected. Specifically, the Company could experience additional delays in future clinical trial timelines as a result of additional travel and hospital restrictions related to the COVID-19 pandemic and the efforts to mitigate it which may be imposed or could experience supply shortages or manufacturer shutdowns impacting the manufacture of drug substance or drug product, which could also impact clinical trial timelines. The financial statements do not reflect any adjustments as a result of the pandemic. 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 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) former shareholders of Chondrial owned a substantial majority of the voting rights of the combined company; (2) the majority of the board of directors of the combined company was composed of directors designated by Chondrial under the terms of the Merger Agreement; and (3) existing members of Chondrial management constituted 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 consolidated 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 Common Stock 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. In December 2020, Chondrial Therapeutics was legally merged into Larimar Therapeutics, Inc. 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 consolidated financial statements include the accounts of Larimar and its wholly owned subsidiaries. All intercompany balances and transactions have been eliminated. The accompanying 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. Going Concern The Company’s consolidated financial statements have been presented on the basis that it will continue as a going concern, which contemplates the realization of assets and satisfaction of liabilities in the normal course of business. Since its inception, the Company has incurred significant recurring operating losses and negative cash flows from operations. The Company has incurred net losses of $ 50.6 million and $ 42.5 million for the years ended December 31, 2021, and 2020, respectively. In addition, as of December 31, 2021, the Company had an accumulated deficit of $ 116.3 million . The Company expects to continue to generate operating losses for the foreseeable future. As of December 31, 2021, the Company had approximately $ 70.1 million of cash and cash equivalents available for use to fund its operations and capital requirements. The Company has funded its operations to date primarily with proceeds from sales of common stock, prefunded warrants for the purchase of common stock and, prior to the merger with Zafgen described above, contributions from Holdings. In June 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 June 2020. In August 2020, the Company entered into an Equity Distribution Agreement (the “ATM Agreement”) with an investment bank in connection with the establishment of an “at-the-market” offering program under which the Company could sell up to an aggregate of $ 50,000,000 of shares of its common stock from time to time through this investment bank as sales agent. In July 2021, the Company sold 2,342,720 shares pursuant to the ATM Agreement for gross proceeds of $ 20.5 million. As of March 23, 2022, $ 29.2 million of additional shares of common stock remained available for sale by the Company under the ATM Agreement. See Note 8 for a further discussion of the ATM Agreement. 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 consolidated financial statements, the Company expects 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 of these financial statements. If the timing of our clinical assumptions were delayed or if there were other forecasted assumption changes that negatively impact our operating plan, the Company could reduce expenditures in order to further extend cash resources. The Company has not yet commercialized any products and does not expect to generate revenue from the commercial sale of any products for several years, if at all. The Company expects that its research and development and general and administrative expenses will continue to increase and, as a result, will need additional capital to fund its future operating and capital requirements. Management is currently evaluating different strategies to obtain the required funding for future operations. Until the Company can generate substantial revenue, if ever, the Company expects to seek additional funding through a combination of public or private equity offerings, debt financings, collaborations and licensing arrangements or other sources. The incurrence of indebtedness would result in increased fixed payment obligations and the Company may be required to agree to certain restrictive covenants, such as limitations on its ability to incur additional debt, limitations on its ability to acquire, sell or license intellectual property rights and other operating restrictions that could adversely impact the Company's ability to conduct our business. Any additional fundraising efforts may divert the Company's management from their day-to-day activities, which may adversely affect its ability to develop and commercialize our product candidates. There can be no assurance that the Company will be able to raise sufficient additional capital on acceptable terms or at all. If such additional financing is not available on satisfactory terms, or is not available in sufficient amounts, or the Company does not have sufficient authorized shares, the Company may be required to delay, limit, or eliminate the development of business opportunities and its ability to achieve its business objectives, its competitiveness, and its business, financial condition, and results of operations will be materially adversely affected. The Company could also be required to seek funds through arrangements with collaborative partners or otherwise at an earlier stage than otherwise would be desirable and it may be required to relinquish rights to some of its technologies or product candidates or otherwise agree to terms unfavorable to it, any of which may have a material adverse effect on the Company's business, operating results and prospects . In addition, geopolitical unrest including the potential impact of the Russian invasion of Ukraine, the possibility that the conflict could expand beyond eastern Europe, the impact of the COVID-19 pandemic and/or other health crises on the global financial markets may reduce the Company's ability to access capital, which could negatively affect its liquidity and ability to continue as a going concern. If the Company is unable to obtain funding when needed and/or on acceptable terms, the Company may be required to significantly curtail, delay or discontinue one or more of its research and development programs, the manufacture of clinical and commercial supplies, product portfolio expansion or pre commercialization efforts, which could adversely affect its business prospects, or the Company may be unable to continue operations. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 12 Months Ended |
Dec. 31, 2021 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies Use of Estimates The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of expenses during the reporting period. Significant estimates and assumptions reflected in these consolidated financial statements include, but are not limited to, the accrual of research and development expense, valuation of stock-based awards and valuation of leases. Due to inherent uncertainty involved in making estimates, actual results reported in future periods may be affected by changes in these estimates. On an ongoing basis, the Company evaluates its estimates and assumptions. Concentrations of Credit Risk and Significant Suppliers Financial instruments that potentially subject the Company to concentrations of credit risk consist principally of cash, cash equivalents and marketable debt securities. 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, cash equivalents and marketable debt securities. The Company is highly dependent on third-party manufacturers to supply products for research and development activities in its programs, to scale and optimize their manufacturing processes and, ultimately, to provide commercial supply. The Company relies and expects to continue to rely on a small number of manufacturers to supply it with its requirements for drug substance and formulated drugs related to these programs. The drug substance which is in frozen liquid form for CTI-1601 is currently manufactured for the Company by a third-party manufacturer, and the frozen liquid form of drug product is made at another manufacturer. The Company is undertaking a program with a third manufacturer to begin to produce a lyophilized version of the drug product from the same drug substance, that, once available, the Company intends to use in certain of its future planned clinical trials. The Company’s research and development programs could be adversely affected by a significant interruption in these manufacturing services or in the supply of drug substance 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 commercial paper, corporate bonds and money market funds as of December 31, 2021 and 2020 . Marketable debt securities Marketable debt securities consist of debt investments with original maturities greater than ninety days. The Company classifies its marketable debt securities as available-for-sale. Accordingly, these investments are recorded at fair value, which is based on quoted market prices. When 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 are reported as a component of accumulated other comprehensive income in stockholders’ equity. Credit losses are recognized through the use of an allowance for credit losses account and subsequent improvements in expected credit losses are recognized as a reversal of the allowance account. If the Company has the intent to sell the security or if it is more likely than not that the Company will be required to sell the security prior to recovery of its amortized cost basis, 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. Property and Equipment Property and equipment are stated at cost less accumulated depreciation. Depreciation expense is recognized using the straight-line method over a five or seven-year estimated useful life for equipment, furniture and fixtures and office equipment. Leasehold improvements are amortized over the shorter of the asset life or the term of the lease agreement. Expenditures for repairs and maintenance of assets are charged to expense as incurred. Upon retirement or sale, the cost and related accumulated depreciation of assets disposed of are removed from the accounts and any resulting gain or loss is included in loss from operations. Impairment of Long-Lived Assets Long-lived assets consist of property and equipment, net¸ and net operating lease assets. Long-lived assets to be held and used are tested for recoverability whenever events or changes in business circumstances indicate that the carrying amount of the assets may not be fully recoverable. Factors that the Company considers in deciding when to perform an impairment review include significant underperformance of the business in relation to expectations, significant negative industry or economic trends, and significant changes or planned changes in the use of the assets. If an impairment review is performed to evaluate a long-lived asset for recoverability, the Company compares forecasts of undiscounted cash flows expected to result from the use and eventual disposition of the long-lived asset to its carrying value. Any impairment loss, if indicated, is measured as the amount by which the carrying amount of the asset exceeds the estimated fair value of the asset. 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, clinical studies and non-clinical 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, non-clinical 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 key 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 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 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 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. Comprehensive Loss Comprehensive loss includes net loss as well as other changes in stockholders’ equity that result from transactions and economic events other than those with stockholders. For the years ended December 31, 2021 and 2020 , the Company’s only element of other comprehensive income (loss) was unrealized gain (loss) on marketable securities. Net Loss Per Share Basic net loss per share is computed by dividing the net 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 twelve months ended December 31, 2021 and 2020 includes the weighted average effect of 628,403 prefunded warrants for the purchase of shares of common stock for which the remaining unfunded exercise price is $ 0.01 per share. Diluted net loss per share attributable to common stockholders is computed by dividing the diluted net loss attributable to common stockholders by the weighted average number of common shares, including potentially dilutive common shares assuming the dilutive effect of outstanding stock options and unvested restricted common shares, as determined using the treasury stock method. For periods in which the Company has reported net losses, diluted net loss per common share attributable to common stockholders is the same as basic net loss per common share attributable to common stockholders, since dilutive common shares are not assumed to have been issued if their effect is antidilutive. The Company excluded 2,523,305 and 2,008,902 options to purchase common stock, outstanding as of December 31, 2021 and 2020 respectively, from the computation of diluted net loss per share for the twelve months ended December 31, 2021 and 2020, respectively, because they had an anti-dilutive impact due to the net loss incurred for the periods. 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 June 2016, the FASB issued ASU No. 2016-13, Financial Instruments—Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments . The FASB subsequently issued amendments to ASU 2016-13. This standard requires entities 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 available-for-sale debt securities to the amount by which carrying value exceeds fair value and requires the reversal of previously recognized credit losses if fair value increases. The Company adopted the standard on January 1, 2020 . The adoption of this standard did not have a material impact on the Company’s consolidated financial statements or related disclosures. In August 2018, the FASB issued ASU No. 2018-13, Fair Value Measurement (Topic 820): Disclosure Framework—Changes to the Disclosure Requirements for Fair Value Measurement . This standard modifies certain disclosure requirements on fair value measurements. This standard became effective and was adopted by the Company on January 1, 2020 . The adoption of this standard did not have a material impact on the Company’s disclosures. |
Merger Accounting
Merger Accounting | 12 Months Ended |
Dec. 31, 2021 | |
Business Combinations [Abstract] | |
Merger Accounting | 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 giving effect to the Reverse Stock Split. In addition, pursuant to the terms of the 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 aggregate of 328,770 shares of the Company’s common stock after giving effect to the Reverse Stock Split. The total purchase price paid in the Merger was 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 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 the fair value of the assets acquired. The following summarizes the 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 |
Fair Value Measurements and Mar
Fair Value Measurements and Marketable Debt Securities | 12 Months Ended |
Dec. 31, 2021 | |
Fair Value Disclosures [Abstract] | |
Fair Value Measurements and Marketable Debt Securities | 4. Fair Value Measurements and Marketable Debt Securities Fair Value Measurements The Company’s assets and liabilities that are measured at fair value on a recurring basis as of December 31, 2021 and December 31, 2020 are measured in accordance with the standards of ASC 820, Fair Value Measurements and Disclosures , which establishes a three-level valuation hierarchy for measuring fair value and expands financial statement disclosures about fair value measurements. The valuation hierarchy is based on upon the transparency of inputs to the valuation of an asset or liability as of the measurement date. The three levels are defined as follows: Level – 1 Inputs to the valuation methodology are quoted prices (unadjusted) for identical assets or liabilities in active markets. Level – 2 Inputs to the valuation methodology include quoted prices for similar assets and liabilities in active markets, and inputs that are observable for the asset or liability, either directly or indirectly, for substantially the full term of the financial instrument. Level – 3 Inputs to the valuation methodology are unobservable and significant to the fair value measurement. The Company’s financial instruments consist primarily of cash and cash equivalents, marketable securities, accounts payable and accrued liabilities. For accounts payable and accrued liabilities, the carrying amounts of these financial instruments as of December 31, 2021 and 2020 were 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 December 31, 2021 and 2020: Total Quoted Significant Significant (in thousands) December 31, 2021 Cash equivalents: Money market funds $ 6,137 $ 6,137 $ — $ — Commercial paper 7,549 7,549 — — Corporate bonds 1,219 1,219 — — Total cash equivalents 14,905 14,905 — — December 31, 2020 Cash equivalents: Money market funds $ 4,229 $ 4,229 $ — $ — Commercial paper 6,499 — 6,499 — Corporate bonds 1,907 — 1,907 — Total cash equivalents 12,635 4,229 8,406 — Marketable securities: U.S Government securities 2,005 — 2,005 — Commercial paper 22,485 — 22,485 — Total marketable debt securities 24,490 — 24,490 — Total cash equivalents and marketable debt securities $ 37,125 $ 4,229 $ 32,896 $ — Marketable Debt Securities The following tables summarize the Company’s marketable debt securities as of December 31, 2020 . There were no marketable debt securities as of December 31, 2021: Amortized Gross Gross Fair Value (in thousands) December 31, 2020 Assets: U.S Government securities (due within 1 year) 2,005 — — 2,005 Commercial paper (due within 1 year) 22,484 2 ( 1 ) 22,485 $ 24,489 $ 2 $ ( 1 ) $ 24,490 |
Prepaid Expenses and Other Curr
Prepaid Expenses and Other Current Assets | 12 Months Ended |
Dec. 31, 2021 | |
Prepaid Expense and Other Assets [Abstract] | |
Prepaid Expenses and Other Current Assets | 5. Prepaid Expenses and Other Current Assets Prepaid expenses and other current assets consisted of the following: December 31, December 31, 2021 2020 (in thousands) Prepaid research and development expenses $ 676 $ 4,460 Prepaid insurance 944 571 Payroll tax receivable 208 32 Other prepaid expenses and other assets 279 251 $ 2,107 $ 5,314 |
Fixed Assets
Fixed Assets | 12 Months Ended |
Dec. 31, 2021 | |
Assets [Abstract] | |
Fixed Assets | 6. Fixed Assets Fixed assets, net consisted of the following: December 31, December 31, Useful Life 2021 2020 (in thousands) Computer equipment 5 years $ 66 $ 66 Lab equipment 5 years 1,092 849 Furniture and fixtures 7 years 456 460 Leasehold improvements lease term 31 — 1,645 1,375 Less: Accumulated depreciation ( 596 ) ( 335 ) $ 1,049 $ 1,040 Depreciation expense was $ 0.2 million for the years ended December 31, 2021 and 2020 , respectively. |
Accrued Expenses
Accrued Expenses | 12 Months Ended |
Dec. 31, 2021 | |
Payables and Accruals [Abstract] | |
Accrued Expenses | 7. Accrued Expenses Accrued expenses consisted of the following: December 31, December 31, 2021 2020 (in thousands) Accrued research and development expenses $ 5,042 $ 3,409 Accrued payroll and related expenses 1,098 1,350 Accrued professional fees — 924 Accrued other 452 160 $ 6,592 $ 5,843 |
Stockholders_ Equity and Stock
Stockholders’ Equity and Stock Options | 12 Months Ended |
Dec. 31, 2021 | |
Stockholders' Equity Note [Abstract] | |
Stockholders' Equity and Stock Options | 8. Stockholders’ Equity and Stock Options Common Stock and Prefunded warrants As of December 31, 2021 , the Company’s Certificate of Incorporation, as amended and restated, authorized the Company to issue up to 115,000,000 shares of $ 0.001 par value common stock, of which 17,710,450 shares were issued and outstanding, and up to 5,000,000 shares of $ 0.001 par value undesignated preferred stock, of which no shares were issued or outstanding. The voting, dividend and liquidation rights of the holders of the Company’s common stock are subject to and qualified by the rights, powers and preferences of the holders of the preferred stock. Each share of common stock entitles the holder to one vote on all matters submitted to a vote of the Company’s stockholders . Common stockholders are entitled to receive dividends, as may be declared by the board of directors of the Company (the “Board”), if any. No cash dividends have been declared or paid to date. On May 28, 2020, the Company entered into a securities purchase agreement with certain accredited investors (the “Purchasers”) for the sale by the Company in a private placement of 6,105,359 shares of the Company’s common stock and prefunded warrants to purchase an aggregate of 628,403 shares of the Company’s common stock, for a price of $ 11.88 per share of the common stock and $ 11.87 per prefunded warrant. The prefunded warrants are exercisable at an exercise price of $ 0.01 and are exercisable indefinitely. The Purchasers may exercise the prefunded warrants on a cashless basis in the event that there is no effective registration statement covering the resale of the shares of common stock underlying the prefunded warrants on the date in which the Company is required to deliver the shares. The private placement closed on June 1, 2020. The aggregate gross proceeds for the issuance and sale of the common stock and prefunded warrants were $ 80.0 million; transaction costs totaled $ 4.6 million and 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, the Company issued MTS Health Partners 35,260 shares of common stock. Equity Distribution Agreement On August 14, 2020, the Company entered into the ATM Agreement with an investment bank, in connection with the establishment of an “at-the-market” offering program under which the Company may sell up to an aggregate of $ 50,000,000 of shares of common stock (the “ATM Shares”) from time to time (the “Offering”). Under the ATM Agreement, the Company sets the parameters for the sale of ATM Shares, including the number of ATM Shares to be issued, the time period during which sales are requested to be made, limitations on the number of ATM Shares that may be sold in any one trading day and any minimum price below which sales may not be made. Sales of the ATM Shares, if any, under the ATM Agreement may be made in transactions that are deemed to be “at-the-market offerings” as defined in Rule 415 under the Securities Act. The Company pays its investment bank a commission equal to 3.0 % of the gross proceeds of any ATM Shares sold through its investment bank under the ATM Agreement and reimburses investment bank for certain specified expenses. The Agreement contains customary representations, warranties and agreements by the Company, indemnification obligations of the Company and its investment bank, other customary obligations of the parties and termination provisions. The Company has no obligation to sell any of the ATM Shares and may at any time suspend offers under the ATM Agreement. In July 2021, the Company sold 2,342,720 shares under the ATM Agreement for net proceeds of $ 19.9 million, after issuance costs. As of December 31, 2021 , 2,354,244 shares have been sold under the ATM Agreement for net proceeds of $ 20.1 million, after issuance costs. As of March 23, 2022, approximately $ 29.2 million of additional shares of common stock remained available for sale by the Company under the ATM Agreement. Summary of Plans Upon completion of the Merger with Zafgen, Zafgen’s 2014 Stock Option and Incentive Plan (the “2014 Plan”) and Zafgen’s 2006 Stock Option Plan (the “2006 Plan” and together with the 2014 Plan the “Prior Plans”) were assumed by the Company. As described below, the Company adopted a new equity incentive plan in July 2020 that was approved by the stockholders in September 2020. These three plans are administered by the Board or, at the discretion of the Board, by a committee of the Board. 2020 Equity Incentive Plan The Board adopted the 2020 Equity Incentive Plan (the "2020 Plan") on July 16, 2020 and the stockholders of the Company approved the 2020 Plan on September 29, 2020. The 2020 Plan replaces the 2014 Plan. Option outstanding under the Prior Plans will remain outstanding, unchanged and subject to the terms of the Prior Plans and the respective award agreements, and no further awards will be made under the 2014 Plan. However, if any award previously granted under the Prior Plans, expires, terminates, is canceled or is forfeited for any reason after the approval of the 2020 Plan, the shares subject to that award will be added to the 2020 Plan share pool so that they can be utilized for new grants under the 2020 Plan. The 2020 Plan provides for the grant of incentive stock options (“ISOs”), nonstatutory stock options (”NSOs”), stock appreciation rights, restricted stock awards, restricted stock unit awards, and cash or other stock-based awards. ISOs may be granted only to the Company’s employees, including the Company’s officers, and the employees of the Company’s affiliates. All other awards may be granted to the Company’s employees, including the Company’s officers, the Company’s non-employee directors and consultants, and the employees and consultants of the Company’s affiliates. The maximum number of shares that may be issued in respect of any awards under the 2020 Plan is the sum of: (i) 1,700,000 shares plus (ii) an annual increase on January 1, 2021 and each anniversary of such date thereafter through January 1, 2030, equal to the lesser of (A) 4 % of the shares issued and outstanding on the last day of the immediately preceding fiscal year, and (B) such smaller number of shares as determined by the Board (collectively, the “Plan Limit”). The maximum aggregate number of shares that may be issued under the 2020 Plan is 8,000,000 over the ten-year term of the 2020 Plan. In 2021, options to purchase 56,966 shares issued under the Prior Plans were cancelled and became available for grant under the 2020 Plan. As of December 31, 2021 , 993,419 shares of common stock were available for grant under the 2020 Plan. As permitted by the 2020 Plan, the Company added 708,418 shares available for grant under the 2020 Plan on January 1, 2022 increasing the maximum number of shares of the Company’s common stock that may be issued under the 2020 Plan to 1,701,837 shares. 2014 Stock Option and Incentive Plan and 2006 Stock Option Plan In 2014, the Board and stockholders of Zafgen adopted the 2014 Plan. The 2014 Plan provided 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. As the 2020 Plan was adopted by the Company and approved by the Company’s stockholders, no further awards will be made under the Prior Plans. 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 a 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 corresponding capital contribution from Holdings. From January 1, 2020 through the Merger date Holdings did not issue 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 weighted average exercise price of $ 12.14 per share were substituted for the 202,392 options to purchase Common Units, with a weighted average exercise price of $ 10.36 per Common Unit, that were outstanding immediately prior to the Merger. The Company treated 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 on the Merger date, the remaining $ 0.5 million is recognized over the remaining vesting term with the original grant date fair value remaining of less than $ 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: 2021 2020 Risk-free interest rate 0.89 % 0.37 % Expected term (in years) 6.19 6.08 Expected volatility 91 % 91 % Dividend yield 0.00 % 0.00 % Stock Options The following table summarizes the Company’s stock option activity for the twelve months ended December 31, 2021 (amounts in millions, except for share and per share data): Weighted Weighted Average Aggregate Average Remaining Intrinsic Number of Exercise Contractual Value (a) Shares Price Term (in years) (in millions) Outstanding as of December 31, 2020 2,008,902 $ 22.31 7.9 Granted 626,750 15.56 Forfeited/Expired ( 112,347 ) 61.80 Outstanding as of December 31, 2021 2,523,305 $ 18.88 7.6 $ 0.2 Exercisable as of December 31, 2021 1,027,858 $ 26.52 5.9 $ — Vested and expected to vest as of December 31, 2021 2,523,305 $ 18.88 7.6 $ 0.2 (a) The aggregate intrinsic value is calculated as the difference between the exercise price of the underlying options and the fair value of the common stock for the options that were in the money at December 31, 2021 . 2021 Option Grants During the year ended December 31, 2021 , the Company granted options to purchase 626,750 shares of common stock to employees and directors under the 2020 Plan. The options have an exercise price equal to the closing stock price as of the grant date. Of the 626,750 options granted in 2021, 576,950 were granted to employees and vest over four years , with 25 % vesting on the first anniversary of the grant and the remainder vesting in equal monthly installments thereafter. The remaining 49,800 options were annual grants to the Company's directors and vest one year from the grant date. January 2022 Option Grants On January 18, 2022, the Company granted options to purchase 582,750 shares of common stock to employees under the 2020 Plan. The options have an exercise price equal to 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. Stock-Based Compensation Stock-based compensation expense was classified in the consolidated statements of operations as follows: Year Ended December 31, 2021 2020 (in thousands) Research and development $ 2,104 $ 788 General and administrative 3,369 1,373 $ 5,473 $ 2,161 As of December 31, 2021 , total unrecognized compensation expense related to unvested stock options and restricted stock units was $ 14.6 million, which is expected to be recognized over a weighted average period of 2.66 years. |
Commitments and Contingencies
Commitments and Contingencies | 12 Months Ended |
Dec. 31, 2021 | |
Commitments and Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 9. Commitments and Contingencies Intellectual Property Licenses The Company is party to an exclusive License Agreement (the “WFUHS License”), dated November 30, 2016 with Wake Forest University Health Sciences (“WFUHS”) and an exclusive License Agreement (the “IU License”), dated November 30, 2016, as amended, with Indiana University (“IU”). Such agreements provide for a transferable, worldwide license to certain patent rights regarding technology used by the Company with respect to the development of CTI-1601. In partial consideration for the right and license granted under these agreements, the Company will pay each of WFUHS and IU a royalty of a low single digit percentage of net sales of licensed products depending on whether there is a valid patent covering such products. As additional consideration for these agreements, the Company is obligated to pay each of WFUHS and IU certain milestone payments of up to $ 2.6 million in the aggregate upon the achievement of certain developmental milestones, commencing on the enrollment of the first patient in a Phase 1 clinical trial. The Company 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 twelve months ended December 31, 2021 and 2020 , no milestones were achieved and no milestone expense was recognized. Both agreements continue from their effective date through the last to expire of the licensed patents unless earlier terminated by either party in accordance with their terms. Leases On August 8, 2019, the Company entered into an operating lease for office space in Bala Cynwyd, Pennsylvania, effective as of December 15, 2019, for a period of three years and six months with an option to extend the lease for three additional years. Due to required tenant improvements to be completed by the landlord, the Company did not take immediate possession of the leased property and the lease term commenced on February 15, 2020 . In the quarter ended March 31, 2020, the Company recorded 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 (the “Premises”). The lease expires on October 30, 2029. As part of the agreement, the Company is required to maintain a letter of credit, which upon signing was $ 1.3 million and is classified as restricted cash within the consolidated financial statements. In addition to the base rent, the Company is also responsible for its share of operating expenses, electricity and real estate taxes, which costs are not included in the determination of the leases’ right-of-use assets or lease liabilities. Starting in 2021, the right-of-use asset is being amortized to other income (expense) over the remaining lease term as a result of the sublease agreement discussed below. On October 27, 2020, the Company entered into a sublease agreement (the “Sublease”) with Massachusetts Municipal Association, Inc. (the “Subtenant”), whereby the Company sublet the entire Premises to the Subtenant. The term of the Sublease commenced on December 4, 2020 and continues until October 30, 2029 . In connection with the Sublease, the Company evaluated the need for impairment under ASC 360 and determined there was no impairment. The Sublease provides for an initial annual base rent of $ 0.8 million, which increases annually up to a maximum annual base rent of $ 1.0 million. The Subtenant also is responsible for paying to the Company future increases in operating costs (commencing on January 1, 2022), future increases in annual tax costs (commencing July 1, 2021) and all utility costs (commencing March 1,2021) attributable to the Premises during the term of the Sublease. As part of the Sublease, the subtenant deposited a letter of credit in the amount of $ 0.8 million to assure their performance under the sublease. If there are no uncured events of default under the sublease, the amount of this security deposit decreases over time to $ 0.4 million on the sixth anniversary of the Sublease. The Company records sublease income on the Sublease on a straight-line basis as a component of other income (expense). On November 5, 2018, the Company entered into an operating lease for office and lab space in Philadelphia, Pennsylvania, effective as of January 1, 2019 , and expiring on December 31, 2020 with an option to extend the lease for each of the two additional years. On August 4, 2020, the Company executed the first option to extend the lease for an additional year, expiring on December 31, 2021 . On August 9, 2021, the Company executed the remaining option to extend the lease for an additional year, expiring on December 31, 2022 . The Company determined this lease extension qualifies as a short-term lease and have applied the accounting policy election to not record the related right-of-use asset and lease liabilities. Expense arising from operating leases was $ 0.3 million and $ 0.7 million during the twelve months ended December 31, 2021 and 2020, respectively. For operating leases, the weighted-average remaining lease term for leases at December 31, 2021 and 2020 was 7.6 and 8.6 years, respectively. For operating leases, the weighted average discount rate for leases at December 31, 2021 and 2020 was 11.0 %. The Company has not entered into any financing leases. Maturities of lease liabilities due under these lease agreements as of December 31, 2021 are as follows: Year Ending December 31, Operating (in thousands) Leases 2022 $ 1,197 2023 1,146 2024 1,065 2025 1,083 2026 1,101 Thereafter 3,213 Total lease payments 8,805 Less: imputed interest ( 2,803 ) Present value of lease liabilities $ 6,002 Legal Proceedings The Company is not currently a party to any litigation, nor is management aware of any pending or threatened litigation against the Company, that it believes would materially affect the Company’s business, operating results, financial condition or cash flows. |
Income Taxes
Income Taxes | 12 Months Ended |
Dec. 31, 2021 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | 10. Income Taxes During the years ended December 31, 2021, and 2020 , the Company recorded no income tax benefits for the net operating losses incurred in each year due to its uncertainty of realizing a benefit from those items. The domestic and foreign components of loss before income taxes are as follows. Years ended December 31, 2021 2020 Domestic $ ( 50,617 ) $ ( 42,608 ) Foreign ( 19 ) 126 $ ( 50,636 ) $ ( 42,482 ) A reconciliation of the U.S. federal statutory income tax rate to the Company’s effective income tax rate is as follows: Year Ended December 31, 2021 2020 Federal statutory income tax rate 21.0 % 21.0 % State taxes, net of federal benefit 3.4 7.8 Change in state tax rate ( 14.5 ) — Federal and state research and development tax credit 7.9 13.5 Nondeductible permanent differences ( 0.6 ) ( 0.2 ) Change in deferred tax asset valuation allowance ( 17.2 ) ( 42.1 ) Effective income tax rate 0.0 % 0.0 % Net deferred tax assets as of December 31, 2021 and 2020 consisted of the following: 2021 2020 Deferred tax assets: Capitalized R&D Expenses $ 65,781 $ 73,014 Stock Based Compensation 1,458 490 Net operating Loss Carryforwards 43,219 32,081 Tax credit carryforwards 12,912 8,904 Other Temporary Differences 24 50 Fixed Assets & Intangibles 44 75 Operating Lease Liability 1,477 1,786 Total deferred tax assets $ 124,915 $ 116,400 Deferred tax liabilities: Operating Right of Use Asset ( 899 ) ( 1,081 ) Total deferred tax liabilities $ ( 899 ) $ ( 1,081 ) Less: Valuation allowance $ ( 124,016 ) $ ( 115,319 ) Net deferred tax assets / (liabilities) $ — $ — Changes in the valuation allowance for deferred tax assets during the year ended December 31, 2021 related primarily to the increase in net operating loss carryforwards and tax credit carryforwards and a decrease other deferred tax assets associated with a reduction in the Company’s effective state rate. The change in the valuation allowance during the year ended December 31, 2020 related primarily to deferred tax assets acquired as a result of the Merger with Zafgen, increase in net operating loss carryforwards, and tax credit carryforwards, Changes to the valuation allowance were as follows: Years ended December 31, 2021 2020 Valuation allowance as of the beginning of the year $ 115,319 $ 11,735 Increases acquired as a result of the Merger with Zafgen — 85,689 Increases recorded to income tax provision 8,697 17,895 Valuation Allowance at end of Year $ 124,016 $ 115,319 As of December 31, 2021 , the Company had net operating loss carryforwards that expire for federal, foreign and state income tax purposes of $ 158.8 million, $ 1.2 million and $ 131.3 million, respectively. The federal and state operating losses begin to expire in 2026 and 2030 , while the foreign net loss carryforward can be carried forward indefinitely. As of December 31, 2021 , the Company had federal net operating loss carryforwards that were generated after December 31, 2017 of $ 120.0 million that do not expire, however these carryforwards are limited to 80 % of the taxable income in any one tax period. As of December 31, 2021 , the Company also had available tax credit carryforwards for federal and state income tax purposes of $ 12.9 million which begin to expire in 2039 . Utilization of the pre-Merger net operating loss carryforwards attributable to Zafgen, of approximately $ 33.5 million, are subject to a substantial annual limitation under Section 382 of the Internal Revenue Code of 1986 due to ownership changes occurred during the tax year associated with the Merger. In addition to the limitation of the pre-Merger NOL’s of Zafgen, the net capitalized R&D deferred tax assets in the amount of $ 73.0 million is subject to the built-in loss rules under Section 382 and may not be realized if the underlying asset associated with the R&D is disposed within five years of the Merger, or May 28, 2025 . In general, an ownership change, as defined by Section 382, results from transactions increasing the ownership of certain stockholders or public groups in the stock of a corporation by more than 50% over a three-year period. The ownership changes will limit the amount of pre-merger Zafgen carryforwards that can be utilized annually to offset future taxable income with an annual limitation of approximately $ 35 thousand per year. The Company has reduced their NOL and R&D tax credit deferred tax assets associated with the pre-Merger Zafgen operations as a result of the 382 analysis. The Company has not conducted a study to assess whether a change of control has occurred or whether there have been multiple changes of control since inception associated with the pre-Merger Chondrial tax attributes. If the Company experienced a change of control, as defined by Section 382, at any time since inception, utilization of the pre-Merger Chondrial net operating loss carryforwards or tax credit carryforwards would be subject to an annual limitation under Section 382, which is determined by first multiplying the value of the Company’s stock at the time of the ownership change by the applicable long-term tax-exempt rate, and then could be subject to additional adjustments, as required. Any limitation may result in expiration of a portion of the net operating loss carryforwards or tax credit carryforwards before utilization. Further, until a study is completed, and any limitation is known, no amounts are being presented as an uncertain tax position. As of December 31, 2021 and 2020 , the Company’s net deferred tax asset balance before the valuation allowance was $ 124.0 million and $ 115.3 million, respectively, and was comprised principally of net operating loss carryforwards, capitalized research and development expenses and tax credit carryforwards. During the years ended December 31, 2021 and 2020, gross deferred tax assets increased due to deferred tax assets acquired as a result of the Merger with Zafgen, additional net operating loss carryforwards, research and development tax credits generated. The Company has evaluated the positive and negative evidence bearing upon its ability to realize the deferred tax assets. Management has considered the Company’s history of cumulative net losses incurred since inception and its lack of commercialization of any products or generation of any revenue from product sales since inception and has concluded that it is more likely than not that the Company will not realize the benefits of the deferred tax assets. Accordingly, a full valuation allowance has been established against the deferred tax assets as of December 31, 2021 and 2020. Management reevaluates the positive and negative evidence at each reporting period. The Company has no t recorded any amounts for unrecognized tax benefits as of December 31, 2021 and 2020 . The Company files tax returns as prescribed by the tax laws of the jurisdictions in which it operates. In the normal course of business, the Company is subject to examination by federal and state jurisdictions, where applicable. There are currently no pending income tax examinations. The Company’s tax years are still open under statute from 2016 to the present. Earlier years may be examined to the extent that tax credit or net operating loss carryforwards are used in future periods. The Company’s policy is to record interest and penalties related to income taxes as part of its income tax provision. |
Related Party Transactions
Related Party Transactions | 12 Months Ended |
Dec. 31, 2021 | |
Related Party Transactions [Abstract] | |
Related Party Transactions | 11. Related Party Transactions In November 2016, the Company entered into a consulting agreement with Mark Payne, M.D (the “Consulting Engagement”). Dr. Payne was a director of Chondrial at that time, a full-time employee of IU and one of the inventors of the licensed IU intellectual property, and as such is entitled to a certain share of the revenues received by IU under the IU License. Pursuant to the terms of his consulting agreement the Company agreed to pay Dr. Payne $ 0.1 million per year over the term of the agreement and granted Dr. Payne 123,853 restricted Common Units in Holdings. On November 30, 2016, 30 % immediately vested and was associated with Chondrial Therapeutics IP, LLC (“IP LLC”) becoming a subsidiary of Holdings, which was subsequently contributed to the Company on December 31, 2018. The remaining 70 % vested ratably over 48 months beginning on December 1, 2016. The consulting agreement had a four-year term, subject to earlier termination. On November 30, 2020, the Company entered into a 1-month extension of the Consulting Engagement, expiring on December 31, 2020 .On January 1, 2021, the Company entered into a new consulting agreement with Mark Payne, M.D. which extended the term of the Consulting Engagement for a four-year term beginning on January 1, 2021. During each of the twelve months ended December 31, 2021 and 2020 , the Company recognized $ 0.1 million, related to this consulting agreement, recorded as research and development expense 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 (the "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 in order to fund the Company’s operations. Under a November 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 gross 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 Restated 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 twelve months ended December 31, 2020, Holdings provided the Company non-interest bearing, permanent funding from the above Series A and Series B preferred unit transactions, totaling $ 18.0 million which has been recorded as capital contributions with the balance of combined equity and additional paid in capital on the consolidated balance sheets and consolidated statements of changes in stockholders’ equity for each respective period. No contributions were made by Holdings subsequent to the Merger. During 2020, the Company purchased a piece of laboratory equipment for $ 0.5 million from a supplier of which one the Company's directors is also a current director. During 2021, the Company purchased a piece of laboratory equipment and lab supplies for a cumulative $ 0.1 million from the same supplier. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 12 Months Ended |
Dec. 31, 2021 | |
Accounting Policies [Abstract] | |
Merger with Zafgen | 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 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) former shareholders of Chondrial owned a substantial majority of the voting rights of the combined company; (2) the majority of the board of directors of the combined company was composed of directors designated by Chondrial under the terms of the Merger Agreement; and (3) existing members of Chondrial management constituted 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 consolidated 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 Common Stock 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. In December 2020, Chondrial Therapeutics was legally merged into Larimar Therapeutics, Inc. 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 | Basis of Presentation The consolidated financial statements include the accounts of Larimar and its wholly owned subsidiaries. All intercompany balances and transactions have been eliminated. The accompanying 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 | 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. |
Going Concern | Going Concern The Company’s consolidated financial statements have been presented on the basis that it will continue as a going concern, which contemplates the realization of assets and satisfaction of liabilities in the normal course of business. Since its inception, the Company has incurred significant recurring operating losses and negative cash flows from operations. The Company has incurred net losses of $ 50.6 million and $ 42.5 million for the years ended December 31, 2021, and 2020, respectively. In addition, as of December 31, 2021, the Company had an accumulated deficit of $ 116.3 million . The Company expects to continue to generate operating losses for the foreseeable future. As of December 31, 2021, the Company had approximately $ 70.1 million of cash and cash equivalents available for use to fund its operations and capital requirements. The Company has funded its operations to date primarily with proceeds from sales of common stock, prefunded warrants for the purchase of common stock and, prior to the merger with Zafgen described above, contributions from Holdings. In June 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 June 2020. In August 2020, the Company entered into an Equity Distribution Agreement (the “ATM Agreement”) with an investment bank in connection with the establishment of an “at-the-market” offering program under which the Company could sell up to an aggregate of $ 50,000,000 of shares of its common stock from time to time through this investment bank as sales agent. In July 2021, the Company sold 2,342,720 shares pursuant to the ATM Agreement for gross proceeds of $ 20.5 million. As of March 23, 2022, $ 29.2 million of additional shares of common stock remained available for sale by the Company under the ATM Agreement. See Note 8 for a further discussion of the ATM Agreement. 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 consolidated financial statements, the Company expects 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 of these financial statements. If the timing of our clinical assumptions were delayed or if there were other forecasted assumption changes that negatively impact our operating plan, the Company could reduce expenditures in order to further extend cash resources. The Company has not yet commercialized any products and does not expect to generate revenue from the commercial sale of any products for several years, if at all. The Company expects that its research and development and general and administrative expenses will continue to increase and, as a result, will need additional capital to fund its future operating and capital requirements. Management is currently evaluating different strategies to obtain the required funding for future operations. Until the Company can generate substantial revenue, if ever, the Company expects to seek additional funding through a combination of public or private equity offerings, debt financings, collaborations and licensing arrangements or other sources. The incurrence of indebtedness would result in increased fixed payment obligations and the Company may be required to agree to certain restrictive covenants, such as limitations on its ability to incur additional debt, limitations on its ability to acquire, sell or license intellectual property rights and other operating restrictions that could adversely impact the Company's ability to conduct our business. Any additional fundraising efforts may divert the Company's management from their day-to-day activities, which may adversely affect its ability to develop and commercialize our product candidates. There can be no assurance that the Company will be able to raise sufficient additional capital on acceptable terms or at all. If such additional financing is not available on satisfactory terms, or is not available in sufficient amounts, or the Company does not have sufficient authorized shares, the Company may be required to delay, limit, or eliminate the development of business opportunities and its ability to achieve its business objectives, its competitiveness, and its business, financial condition, and results of operations will be materially adversely affected. The Company could also be required to seek funds through arrangements with collaborative partners or otherwise at an earlier stage than otherwise would be desirable and it may be required to relinquish rights to some of its technologies or product candidates or otherwise agree to terms unfavorable to it, any of which may have a material adverse effect on the Company's business, operating results and prospects . In addition, geopolitical unrest including the potential impact of the Russian invasion of Ukraine, the possibility that the conflict could expand beyond eastern Europe, the impact of the COVID-19 pandemic and/or other health crises on the global financial markets may reduce the Company's ability to access capital, which could negatively affect its liquidity and ability to continue as a going concern. If the Company is unable to obtain funding when needed and/or on acceptable terms, the Company may be required to significantly curtail, delay or discontinue one or more of its research and development programs, the manufacture of clinical and commercial supplies, product portfolio expansion or pre commercialization efforts, which could adversely affect its business prospects, or the Company may be unable to continue operations. |
Use of Estimates | Use of Estimates The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of expenses during the reporting period. Significant estimates and assumptions reflected in these consolidated financial statements include, but are not limited to, the accrual of research and development expense, valuation of stock-based awards and valuation of leases. Due to inherent uncertainty involved in making estimates, actual results reported in future periods may be affected by changes in these estimates. On an ongoing basis, the Company evaluates its estimates and assumptions. |
Concentrations of Credit Risk and Significant Suppliers | Concentrations of Credit Risk and Significant Suppliers Financial instruments that potentially subject the Company to concentrations of credit risk consist principally of cash, cash equivalents and marketable debt securities. 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, cash equivalents and marketable debt securities. The Company is highly dependent on third-party manufacturers to supply products for research and development activities in its programs, to scale and optimize their manufacturing processes and, ultimately, to provide commercial supply. The Company relies and expects to continue to rely on a small number of manufacturers to supply it with its requirements for drug substance and formulated drugs related to these programs. The drug substance which is in frozen liquid form for CTI-1601 is currently manufactured for the Company by a third-party manufacturer, and the frozen liquid form of drug product is made at another manufacturer. The Company is undertaking a program with a third manufacturer to begin to produce a lyophilized version of the drug product from the same drug substance, that, once available, the Company intends to use in certain of its future planned clinical trials. The Company’s research and development programs could be adversely affected by a significant interruption in these manufacturing services or in the supply of drug substance and formulated drugs. |
Cash and Cash Equivalents | 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 commercial paper, corporate bonds and money market funds as of December 31, 2021 and 2020 . |
Marketable Debt Securities | Marketable debt securities Marketable debt securities consist of debt investments with original maturities greater than ninety days. The Company classifies its marketable debt securities as available-for-sale. Accordingly, these investments are recorded at fair value, which is based on quoted market prices. When 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 are reported as a component of accumulated other comprehensive income in stockholders’ equity. Credit losses are recognized through the use of an allowance for credit losses account and subsequent improvements in expected credit losses are recognized as a reversal of the allowance account. If the Company has the intent to sell the security or if it is more likely than not that the Company will be required to sell the security prior to recovery of its amortized cost basis, 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. |
Property and Equipment | Property and Equipment Property and equipment are stated at cost less accumulated depreciation. Depreciation expense is recognized using the straight-line method over a five or seven-year estimated useful life for equipment, furniture and fixtures and office equipment. Leasehold improvements are amortized over the shorter of the asset life or the term of the lease agreement. Expenditures for repairs and maintenance of assets are charged to expense as incurred. Upon retirement or sale, the cost and related accumulated depreciation of assets disposed of are removed from the accounts and any resulting gain or loss is included in loss from operations. |
Impairment of Long-Lived Assets | Impairment of Long-Lived Assets Long-lived assets consist of property and equipment, net¸ and net operating lease assets. Long-lived assets to be held and used are tested for recoverability whenever events or changes in business circumstances indicate that the carrying amount of the assets may not be fully recoverable. Factors that the Company considers in deciding when to perform an impairment review include significant underperformance of the business in relation to expectations, significant negative industry or economic trends, and significant changes or planned changes in the use of the assets. If an impairment review is performed to evaluate a long-lived asset for recoverability, the Company compares forecasts of undiscounted cash flows expected to result from the use and eventual disposition of the long-lived asset to its carrying value. Any impairment loss, if indicated, is measured as the amount by which the carrying amount of the asset exceeds the estimated fair value of the asset. |
Segment Information | 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 | Research and Development Costs Costs associated with internal research and development and external research and development services, including drug development, clinical studies and non-clinical studies, are expensed as incurred. Research and development expenses include costs for salaries, employee benefits, subcontractors, facility-related expenses, depreciation, stock-based compensation, third-party license fees, laboratory supplies, and external costs of outside vendors engaged to conduct discovery, non-clinical 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 key 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 | 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 | 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 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 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 | 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 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. |
Comprehensive Loss | Comprehensive Loss Comprehensive loss includes net loss as well as other changes in stockholders’ equity that result from transactions and economic events other than those with stockholders. For the years ended December 31, 2021 and 2020 , the Company’s only element of other comprehensive income (loss) was unrealized gain (loss) on marketable securities. |
Net Loss Per Share | Net Loss Per Share Basic net loss per share is computed by dividing the net 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 twelve months ended December 31, 2021 and 2020 includes the weighted average effect of 628,403 prefunded warrants for the purchase of shares of common stock for which the remaining unfunded exercise price is $ 0.01 per share. Diluted net loss per share attributable to common stockholders is computed by dividing the diluted net loss attributable to common stockholders by the weighted average number of common shares, including potentially dilutive common shares assuming the dilutive effect of outstanding stock options and unvested restricted common shares, as determined using the treasury stock method. For periods in which the Company has reported net losses, diluted net loss per common share attributable to common stockholders is the same as basic net loss per common share attributable to common stockholders, since dilutive common shares are not assumed to have been issued if their effect is antidilutive. The Company excluded 2,523,305 and 2,008,902 options to purchase common stock, outstanding as of December 31, 2021 and 2020 respectively, from the computation of diluted net loss per share for the twelve months ended December 31, 2021 and 2020, respectively, because they had an anti-dilutive impact due to the net loss incurred for the periods. 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 | Recently Issued and Adopted Accounting Pronouncements In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments—Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments . The FASB subsequently issued amendments to ASU 2016-13. This standard requires entities 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 available-for-sale debt securities to the amount by which carrying value exceeds fair value and requires the reversal of previously recognized credit losses if fair value increases. The Company adopted the standard on January 1, 2020 . The adoption of this standard did not have a material impact on the Company’s consolidated financial statements or related disclosures. In August 2018, the FASB issued ASU No. 2018-13, Fair Value Measurement (Topic 820): Disclosure Framework—Changes to the Disclosure Requirements for Fair Value Measurement . This standard modifies certain disclosure requirements on fair value measurements. This standard became effective and was adopted by the Company on January 1, 2020 . The adoption of this standard did not have a material impact on the Company’s disclosures. |
Merger Accounting (Tables)
Merger Accounting (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Business Combinations [Abstract] | |
Summary of Purchase Price Paid in Merger | 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. |
Summary of Allocation of Purchase Price to Net Tangible and Intangible Assets Acquired | The allocation of the purchase price for 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 the fair value of the assets acquired. The following summarizes the 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 |
Fair Value Measurements and M_2
Fair Value Measurements and Marketable Debt Securities (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Fair Value Disclosures [Abstract] | |
Summary of Cash Equivalents and Marketable Debt Securities | The following tables summarize the Company’s cash equivalents and marketable debt securities as of December 31, 2021 and 2020: Total Quoted Significant Significant (in thousands) December 31, 2021 Cash equivalents: Money market funds $ 6,137 $ 6,137 $ — $ — Commercial paper 7,549 7,549 — — Corporate bonds 1,219 1,219 — — Total cash equivalents 14,905 14,905 — — December 31, 2020 Cash equivalents: Money market funds $ 4,229 $ 4,229 $ — $ — Commercial paper 6,499 — 6,499 — Corporate bonds 1,907 — 1,907 — Total cash equivalents 12,635 4,229 8,406 — Marketable securities: U.S Government securities 2,005 — 2,005 — Commercial paper 22,485 — 22,485 — Total marketable debt securities 24,490 — 24,490 — Total cash equivalents and marketable debt securities $ 37,125 $ 4,229 $ 32,896 $ — |
Summary of Marketable Debt Securities | The following tables summarize the Company’s marketable debt securities as of December 31, 2020 . There were no marketable debt securities as of December 31, 2021: Amortized Gross Gross Fair Value (in thousands) December 31, 2020 Assets: U.S Government securities (due within 1 year) 2,005 — — 2,005 Commercial paper (due within 1 year) 22,484 2 ( 1 ) 22,485 $ 24,489 $ 2 $ ( 1 ) $ 24,490 |
Prepaid Expenses and Other Cu_2
Prepaid Expenses and Other Current Assets (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Prepaid Expense and Other Assets [Abstract] | |
Schedule of Prepaid Expenses and Other Current Assets | Prepaid expenses and other current assets consisted of the following: December 31, December 31, 2021 2020 (in thousands) Prepaid research and development expenses $ 676 $ 4,460 Prepaid insurance 944 571 Payroll tax receivable 208 32 Other prepaid expenses and other assets 279 251 $ 2,107 $ 5,314 |
Fixed Assets (Tables)
Fixed Assets (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Assets [Abstract] | |
Schedule of Fixed Assets, Net | Fixed assets, net consisted of the following: December 31, December 31, Useful Life 2021 2020 (in thousands) Computer equipment 5 years $ 66 $ 66 Lab equipment 5 years 1,092 849 Furniture and fixtures 7 years 456 460 Leasehold improvements lease term 31 — 1,645 1,375 Less: Accumulated depreciation ( 596 ) ( 335 ) $ 1,049 $ 1,040 |
Accrued Expenses (Tables)
Accrued Expenses (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Payables and Accruals [Abstract] | |
Schedule of Accrued Expenses | Accrued expenses consisted of the following: December 31, December 31, 2021 2020 (in thousands) Accrued research and development expenses $ 5,042 $ 3,409 Accrued payroll and related expenses 1,098 1,350 Accrued professional fees — 924 Accrued other 452 160 $ 6,592 $ 5,843 |
Stockholders_ Equity and Stoc_2
Stockholders’ Equity and Stock Options (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Stockholders' Equity Note [Abstract] | |
Assumptions used to Determine Fair Value of Stock Options Granted | 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: 2021 2020 Risk-free interest rate 0.89 % 0.37 % Expected term (in years) 6.19 6.08 Expected volatility 91 % 91 % Dividend yield 0.00 % 0.00 % |
Summary of Stock Option Activity | The following table summarizes the Company’s stock option activity for the twelve months ended December 31, 2021 (amounts in millions, except for share and per share data): Weighted Weighted Average Aggregate Average Remaining Intrinsic Number of Exercise Contractual Value (a) Shares Price Term (in years) (in millions) Outstanding as of December 31, 2020 2,008,902 $ 22.31 7.9 Granted 626,750 15.56 Forfeited/Expired ( 112,347 ) 61.80 Outstanding as of December 31, 2021 2,523,305 $ 18.88 7.6 $ 0.2 Exercisable as of December 31, 2021 1,027,858 $ 26.52 5.9 $ — Vested and expected to vest as of December 31, 2021 2,523,305 $ 18.88 7.6 $ 0.2 (a) The aggregate intrinsic value is calculated as the difference between the exercise price of the underlying options and the fair value of the common stock for the options that were in the money at December 31, 2021 . |
Summary of Stock-Based Compensation Expense | Stock-based compensation expense was classified in the consolidated statements of operations as follows: Year Ended December 31, 2021 2020 (in thousands) Research and development $ 2,104 $ 788 General and administrative 3,369 1,373 $ 5,473 $ 2,161 |
Commitments and Contingencies (
Commitments and Contingencies (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Commitments and Contingencies Disclosure [Abstract] | |
Schedule of Maturities of Lease Liabilities Due Under Lease Agreements | Maturities of lease liabilities due under these lease agreements as of December 31, 2021 are as follows: Year Ending December 31, Operating (in thousands) Leases 2022 $ 1,197 2023 1,146 2024 1,065 2025 1,083 2026 1,101 Thereafter 3,213 Total lease payments 8,805 Less: imputed interest ( 2,803 ) Present value of lease liabilities $ 6,002 |
Income Taxes (Tables)
Income Taxes (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Income Tax Disclosure [Abstract] | |
Schedule of Domestic and Foreign Components of Loss Before Income Taxes | The domestic and foreign components of loss before income taxes are as follows. Years ended December 31, 2021 2020 Domestic $ ( 50,617 ) $ ( 42,608 ) Foreign ( 19 ) 126 $ ( 50,636 ) $ ( 42,482 ) |
Reconciliation of Federal Statutory Income Tax Rate | A reconciliation of the U.S. federal statutory income tax rate to the Company’s effective income tax rate is as follows: Year Ended December 31, 2021 2020 Federal statutory income tax rate 21.0 % 21.0 % State taxes, net of federal benefit 3.4 7.8 Change in state tax rate ( 14.5 ) — Federal and state research and development tax credit 7.9 13.5 Nondeductible permanent differences ( 0.6 ) ( 0.2 ) Change in deferred tax asset valuation allowance ( 17.2 ) ( 42.1 ) Effective income tax rate 0.0 % 0.0 % |
Schedule of Net Deferred Tax Assets | Net deferred tax assets as of December 31, 2021 and 2020 consisted of the following: 2021 2020 Deferred tax assets: Capitalized R&D Expenses $ 65,781 $ 73,014 Stock Based Compensation 1,458 490 Net operating Loss Carryforwards 43,219 32,081 Tax credit carryforwards 12,912 8,904 Other Temporary Differences 24 50 Fixed Assets & Intangibles 44 75 Operating Lease Liability 1,477 1,786 Total deferred tax assets $ 124,915 $ 116,400 Deferred tax liabilities: Operating Right of Use Asset ( 899 ) ( 1,081 ) Total deferred tax liabilities $ ( 899 ) $ ( 1,081 ) Less: Valuation allowance $ ( 124,016 ) $ ( 115,319 ) Net deferred tax assets / (liabilities) $ — $ — |
Summary of Changes in Valuation Allowance for Deferred Tax Assets | The change in the valuation allowance during the year ended December 31, 2020 related primarily to deferred tax assets acquired as a result of the Merger with Zafgen, increase in net operating loss carryforwards, and tax credit carryforwards, Changes to the valuation allowance were as follows: Years ended December 31, 2021 2020 Valuation allowance as of the beginning of the year $ 115,319 $ 11,735 Increases acquired as a result of the Merger with Zafgen — 85,689 Increases recorded to income tax provision 8,697 17,895 Valuation Allowance at end of Year $ 124,016 $ 115,319 |
Description of Business and O_2
Description of Business and Organization - Additional Information (Detail) | Aug. 14, 2020USD ($) | Jun. 30, 2020USD ($) | May 28, 2020$ / sharesshares | Mar. 23, 2022USD ($) | Jul. 31, 2021USD ($)shares | Dec. 31, 2021USD ($)$ / sharesshares | Dec. 31, 2020USD ($)$ / sharesshares |
Common stock, par value per share | $ / shares | $ 0.001 | $ 0.001 | |||||
Common stock, shares issued | shares | 17,710,450 | 15,367,730 | |||||
Net loss | $ 50,636,000 | $ 42,482,000 | |||||
Accumulated deficit | (116,250,000) | (65,614,000) | |||||
Cash and cash equivalents | $ 70,097,000 | $ 68,148,000 | |||||
Private Offering [Member] | |||||||
Proceeds from issuance common stock and warrants net offering costs | $ 75,400,000 | ||||||
Common Stock [Member] | |||||||
Issuance of common stock | shares | 2,342,720 | ||||||
Common Stock [Member] | ATM Offering Program [Member] | |||||||
Common stock, shares issued | shares | 2,354,244 | ||||||
Issuance of common stock | shares | 2,342,720 | ||||||
Gross proceeds from issuance of common stock | $ 20,500,000 | ||||||
Common Stock [Member] | Subsequent Event [Member] | ATM Offering Program [Member] | |||||||
Gross sales proceeds under sales agreement remaining amount | $ 29,200,000 | ||||||
Maximum [Member] | Common Stock [Member] | ATM Offering Program [Member] | |||||||
Aggregate sale of shares of our common stock | $ 50,000,000 | ||||||
Zafgen [Member] | |||||||
Reverse stock split of common stock | 1-for-12 | ||||||
Common stock, par value per share | $ / shares | $ 0.001 | ||||||
Cash, cash equivalents, restricted cash and marketable debt securities acquired merger | $ 42,900,000 | ||||||
Zafgen [Member] | Merger Agreement [Member] | |||||||
Exchange rate of shares issued per share | 60,912.5005 | ||||||
Common stock, shares issued | shares | 6,091,250 | ||||||
Chondrial [Member] | Merger Agreement [Member] | |||||||
Common stock, par value per share | $ / shares | $ 0.001 |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies - Additional Information (Detail) - $ / shares | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Summary Of Significant Accounting Policies [Line Items] | ||
Percentage of likelihood to be realized upon ultimate settlement, description | 50.00% | |
Warrant issued | 628,403 | 628,403 |
Warrant exercise price | $ 0.01 | $ 0.01 |
Accounting Standards Update 2016-13 [Member] | ||
Summary Of Significant Accounting Policies [Line Items] | ||
Change in Accounting Principle, Accounting Standards Update, Adopted [true false] | true | |
Change in Accounting Principle, Accounting Standards Update, Adoption Date | Jan. 1, 2020 | |
Change in Accounting Principle, Accounting Standards Update, Immaterial Effect [true false] | true | |
Accounting Standards Update 2018-13 [Member] | ||
Summary Of Significant Accounting Policies [Line Items] | ||
Change in Accounting Principle, Accounting Standards Update, Adopted [true false] | true | |
Change in Accounting Principle, Accounting Standards Update, Adoption Date | Jan. 1, 2020 | |
Change in Accounting Principle, Accounting Standards Update, Immaterial Effect [true false] | true | |
Options to Purchase Common Stock [Member] | ||
Summary Of Significant Accounting Policies [Line Items] | ||
Total potentially dilutive shares | 2,523,305 | 2,008,902 |
Equipment, Furniture and Fixtures and Office Equipment [Member] | Minimum [Member] | ||
Summary Of Significant Accounting Policies [Line Items] | ||
Estimated useful life | 5 years | |
Equipment, Furniture and Fixtures and Office Equipment [Member] | Maximum [Member] | ||
Summary Of Significant Accounting Policies [Line Items] | ||
Estimated useful life | 7 years |
Merger Accounting - Additional
Merger Accounting - Additional Information (Detail) - shares | May 28, 2020 | Dec. 31, 2021 | Dec. 31, 2020 |
Business Acquisition [Line Items] | |||
Common stock, shares issued | 17,710,450 | 15,367,730 | |
Zafgen [Member] | |||
Business Acquisition [Line Items] | |||
Capital stock ownership percentage | 34.00% | ||
Zafgen [Member] | Options to Purchase Common Stock [Member] | |||
Business Acquisition [Line Items] | |||
Options to purchase stock | 328,770 | ||
Chondrial Therapeutics Inc. [Member] | |||
Business Acquisition [Line Items] | |||
Capital stock ownership percentage | 66.00% | ||
Merger Agreement [Member] | Zafgen [Member] | |||
Business Acquisition [Line Items] | |||
Common stock, shares issued | 6,091,250 |
Merger Accounting - Summary of
Merger Accounting - Summary of Purchase Price Paid in Merger (Detail) - USD ($) $ / shares in Units, $ in Thousands | May 28, 2020 | Dec. 31, 2021 | Dec. 31, 2020 | May 27, 2020 | |
Business Acquisition [Line Items] | |||||
Purchase price: | $ 38,834 | ||||
Common stock outstanding | 17,710,450 | 15,367,730 | |||
Zafgen [Member] | |||||
Business Acquisition [Line Items] | |||||
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 | ||||
Common stock outstanding | 37,492,044 | ||||
[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. |
Merger Accounting - Summary o_2
Merger Accounting - Summary of Allocation of Purchase Price to Net Tangible and Intangible Assets Acquired (Detail) $ in Thousands | May 28, 2020USD ($) |
Business Combinations [Abstract] | |
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 |
Fair Value Measurements and M_3
Fair Value Measurements and Marketable Debt Securities - Additional Information (Detail) $ in Thousands | Dec. 31, 2021USD ($) |
Fair Value Disclosures [Abstract] | |
Marketable debt securities | $ 0 |
Fair Value Measurements and M_4
Fair Value Measurements and Marketable Debt Securities - Summary of Cash Equivalents and Marketable Debt Securities (Detail) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Cash equivalents | $ 14,905 | $ 12,635 |
Marketable debt securities | 24,490 | |
Total cash equivalents and marketable debt securities | 37,125 | |
Money Market Funds [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Cash equivalents | 6,137 | 4,229 |
Commercial Paper [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Cash equivalents | 7,549 | 6,499 |
Marketable debt securities | 22,485 | |
Corporate Bonds [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Cash equivalents | 1,219 | 1,907 |
U.S Government Securities [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Marketable debt securities | 2,005 | |
Quoted Prices in Active Markets, (Level 1) [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Cash equivalents | 14,905 | 4,229 |
Total cash equivalents and marketable debt securities | 4,229 | |
Quoted Prices in Active Markets, (Level 1) [Member] | Money Market Funds [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Cash equivalents | 6,137 | 4,229 |
Quoted Prices in Active Markets, (Level 1) [Member] | Commercial Paper [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Cash equivalents | 7,549 | |
Quoted Prices in Active Markets, (Level 1) [Member] | Corporate Bonds [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Cash equivalents | $ 1,219 | |
Significant Other Observable Inputs (Level 2) [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Cash equivalents | 8,406 | |
Marketable debt securities | 24,490 | |
Total cash equivalents and marketable debt securities | 32,896 | |
Significant Other Observable Inputs (Level 2) [Member] | Commercial Paper [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Cash equivalents | 6,499 | |
Marketable debt securities | 22,485 | |
Significant Other Observable Inputs (Level 2) [Member] | Corporate Bonds [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Cash equivalents | 1,907 | |
Significant Other Observable Inputs (Level 2) [Member] | U.S Government Securities [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Marketable debt securities | $ 2,005 |
Fair Value Measurements and M_5
Fair Value Measurements and Marketable Debt Securities - Summary of Marketable Debt Securities (Detail) $ in Thousands | Dec. 31, 2020USD ($) |
Schedule Of Available For Sale Securities [Line Items] | |
Amortized Cost | $ 24,489 |
Gross Unrealized Gains | 2 |
Gross Unrealized Losses | (1) |
Fair Value | 24,490 |
U.S Government securities (due within 1 year) [Member] | |
Schedule Of Available For Sale Securities [Line Items] | |
Amortized Cost | 2,005 |
Fair Value | 2,005 |
Commercial paper (due within 1 year) [Member] | |
Schedule Of Available For Sale Securities [Line Items] | |
Amortized Cost | 22,484 |
Gross Unrealized Gains | 2 |
Gross Unrealized Losses | (1) |
Fair Value | $ 22,485 |
Prepaid Expenses and Other Cu_3
Prepaid Expenses and Other Current Assets - Schedule of Prepaid Expenses and Other Current Assets (Detail) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Prepaid Expense and Other Assets [Abstract] | ||
Prepaid research and development expenses | $ 676 | $ 4,460 |
Prepaid insurance | 944 | 571 |
Payroll tax receivable | 208 | 32 |
Other prepaid expenses and other assets | 279 | 251 |
Total prepaid expenses and other current assets | $ 2,107 | $ 5,314 |
Fixed Assets - Schedule of Fixe
Fixed Assets - Schedule of Fixed Assets, Net (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Fixed assets, gross | $ 1,645 | $ 1,375 |
Less: Accumulated depreciation | (596) | (335) |
Fixed assets, net | 1,049 | 1,040 |
Computer Equipment [Member] | ||
Fixed assets, gross | $ 66 | 66 |
Estimated useful life | 5 years | |
Lab Equipment [Member] | ||
Fixed assets, gross | $ 1,092 | 849 |
Estimated useful life | 5 years | |
Furniture and Fixtures [Member] | ||
Fixed assets, gross | $ 456 | $ 460 |
Estimated useful life | 7 years | |
Leasehold Improvements [Member] | ||
Fixed assets, gross | $ 31 | |
Fixed assets, useful life | lease term |
Fixed Assets - Additional Infor
Fixed Assets - Additional Information (Detail) - USD ($) $ in Millions | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Assets [Abstract] | ||
Depreciation expense | $ 0.2 | $ 0.2 |
Accrued Expenses - Schedule of
Accrued Expenses - Schedule of Accrued Expenses (Detail) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Payables and Accruals [Abstract] | ||
Accrued research and development expenses | $ 5,042 | $ 3,409 |
Accrued payroll and related expenses | 1,098 | 1,350 |
Accrued professional fees | 924 | |
Accrued other | 452 | 160 |
Total accrued expenses | $ 6,592 | $ 5,843 |
Stockholders Equity and Stock O
Stockholders Equity and Stock Options - Additional Information (Detail) - USD ($) | Jan. 18, 2022 | Jan. 01, 2022 | Aug. 14, 2020 | May 28, 2020 | Apr. 29, 2019 | Mar. 23, 2022 | Jul. 31, 2021 | Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2021 | Mar. 23, 2018 | Nov. 30, 2016 | Dec. 31, 2014 |
Undesignated preferred stock, shares authorized | 5,000,000 | 5,000,000 | 5,000,000 | ||||||||||
Undesignated preferred stock, par value | $ 0.001 | $ 0.001 | $ 0.001 | ||||||||||
Undesignated preferred stock, shares issued | 0 | 0 | 0 | ||||||||||
Undesignated preferred stock, shares outstanding | 0 | 0 | 0 | ||||||||||
Common stock, shares authorized | 115,000,000 | 115,000,000 | 115,000,000 | ||||||||||
Common stock, par value | $ 0.001 | $ 0.001 | $ 0.001 | ||||||||||
Common stock, shares issued | 17,710,450 | 15,367,730 | 17,710,450 | ||||||||||
Common stock, shares outstanding | 17,710,450 | 15,367,730 | 17,710,450 | ||||||||||
Dividend paid | $ 0 | $ 0 | |||||||||||
Common stock voting rights | Each share of common stock entitles the holder to one vote on all matters submitted to a vote of the Company’s stockholders | ||||||||||||
Issuance of warrants | 628,403 | 628,403 | 628,403 | ||||||||||
Warrants exercise price | $ 0.01 | $ 0.01 | $ 0.01 | ||||||||||
Proceeds from sale of common stock and prefunded warrants, net of issuance costs | $ 19,885,000 | $ 75,592,000 | |||||||||||
Options to purchase shares of common stock granted | 626,750 | ||||||||||||
Unrecognized compensation expense | $ 14,600,000 | $ 14,600,000 | |||||||||||
Unrecognized compensation expense recognized period | 2 years 7 months 28 days | ||||||||||||
2016 Equity and Incentive Plan [Member] | |||||||||||||
Number of options to purchase common unit prior to conversion | 202,392 | ||||||||||||
Number of options to purchase common unit post conversion | 330,818 | ||||||||||||
Weighted average exercise price post conversion | $ 12.14 | ||||||||||||
Weighted average exercise price prior to conversion | $ 10.36 | ||||||||||||
Increase in fair value | $ 1,200,000 | ||||||||||||
2016 Equity and Incentive Plan [Member] | Vested Recognized Immediately [Member] | |||||||||||||
Increase in fair value | 700,000 | ||||||||||||
2016 Equity and Incentive Plan [Member] | Nonvested Recognize Over Remaining Term [Member] | |||||||||||||
Increase in fair value | 500,000 | ||||||||||||
2014 Stock Option and Incentive Plan [Member] | |||||||||||||
Shares reserved for future issuance | 180,685 | ||||||||||||
2020 Equity Incentive Plan [Member] | |||||||||||||
Share-based compensation arrangement by share-based payment award, description | The maximum number of shares that may be issued in respect of any awards under the 2020 Plan is the sum of: (i) 1,700,000 shares plus (ii) an annual increase on January 1, 2021 and each anniversary of such date thereafter through January 1, 2030, equal to the lesser of (A) 4% of the shares issued and outstanding on the last day of the immediately preceding fiscal year, and (B) such smaller number of shares as determined by the Board (collectively, the “Plan Limit”). | ||||||||||||
Minimum level of maximum number of shares allowed to be issued | 1,700,000 | ||||||||||||
Percentage of outstanding shares | 4.00% | ||||||||||||
2020 Equity Incentive Plan [Member] | From Prior Plans [Member] | |||||||||||||
Shares available for grant | 56,966 | 56,966 | |||||||||||
2020 Equity Incentive Plan [Member] | Employees and Directors [Member] | |||||||||||||
Options to purchase shares of common stock granted | 626,750 | ||||||||||||
2020 Equity Incentive Plan [Member] | Employees [Member] | |||||||||||||
Options to purchase shares of common stock granted | 576,950 | ||||||||||||
Vesting period of stock option | 4 years | ||||||||||||
2020 Equity Incentive Plan [Member] | Director [Member] | |||||||||||||
Options to purchase shares of common stock granted | 49,800 | ||||||||||||
Vesting period of stock option | 1 year | ||||||||||||
2020 Equity Incentive Plan [Member] | First Anniversary [Member] | Employees [Member] | |||||||||||||
Stock option vesting percentage | 25.00% | ||||||||||||
2020 Equity Incentive Plan [Member] | Subsequent Event [Member] | Employees [Member] | |||||||||||||
Options to purchase shares of common stock granted | 582,750 | ||||||||||||
Vesting period of stock option | 4 years | ||||||||||||
2020 Equity Incentive Plan [Member] | Subsequent Event [Member] | First Anniversary [Member] | Employees [Member] | |||||||||||||
Stock option vesting percentage | 25.00% | ||||||||||||
Maximum [Member] | 2016 Equity and Incentive Plan [Member] | |||||||||||||
Aggregate grant fair value | $ 100,000 | ||||||||||||
Maximum [Member] | 2020 Equity Incentive Plan [Member] | Incentive Stock Options [Member] | |||||||||||||
Maximum number of shares that may be issued under stock option incentive plan | 8,000,000 | 8,000,000 | |||||||||||
Common Stock [Member] | |||||||||||||
Issuance of common stock | 2,342,720 | ||||||||||||
Common Stock [Member] | 2020 Equity Incentive Plan [Member] | |||||||||||||
Shares available for grant | 993,419 | 993,419 | |||||||||||
Term of plan | 10 years | ||||||||||||
Common Stock [Member] | 2020 Equity Incentive Plan [Member] | Subsequent Event [Member] | |||||||||||||
Increase in shares reserved for future issuance | 708,418 | ||||||||||||
Maximum number of shares that may be issued under stock option incentive plan | 1,701,837 | ||||||||||||
Common Units [Member] | 2016 Equity and Incentive Plan [Member] | |||||||||||||
Increase in shares reserved for future issuance | 101,500 | ||||||||||||
Maximum number of shares that may be issued under stock option incentive plan | 239,633 | 138,133 | 122,133 | ||||||||||
Private Placement [Member] | |||||||||||||
Issuance of common stock | 6,105,359 | ||||||||||||
Share price per share | $ 11.88 | ||||||||||||
Issuance of warrants | 628,403 | ||||||||||||
Warrants price per share | $ 11.87 | ||||||||||||
Gross proceeds | $ 80,000,000 | ||||||||||||
Warrants exercise price | $ 0.01 | ||||||||||||
Transaction cost | $ 4,600,000 | ||||||||||||
Proceeds from issuance common stock and warrants net offering costs | $ 75,400,000 | ||||||||||||
Private Placement [Member] | MTS Health Partners [Member] | |||||||||||||
Issuance of common stock | 35,260 | ||||||||||||
ATM Offering Program [Member] | Common Stock [Member] | |||||||||||||
Common stock, shares issued | 2,354,244 | 2,354,244 | |||||||||||
Issuance of common stock | 2,342,720 | ||||||||||||
Sales agreement, description | The Company has no obligation to sell any of the ATM Shares and may at any time suspend offers under the ATM Agreement. | ||||||||||||
Proceeds from sale of common stock and prefunded warrants, net of issuance costs | $ 19,900,000 | $ 20,100,000 | |||||||||||
ATM Offering Program [Member] | Common Stock [Member] | Subsequent Event [Member] | |||||||||||||
Gross sales proceeds under sales agreement remaining amount | $ 29,200,000 | ||||||||||||
ATM Offering Program [Member] | Common Stock [Member] | Maximum [Member] | |||||||||||||
Aggregate sale of shares of our common stock | $ 50,000,000 | ||||||||||||
ATM Offering Program [Member] | Piper Sandler & Co [Member] | Common Stock [Member] | |||||||||||||
Percentage of compensation for services equal to gross proceeds | 3.00% |
Stockholders Equity and Stock_2
Stockholders Equity and Stock Options - Assumptions used to Determine Fair Value of Stock Options Granted (Details) | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Stockholders' Equity Note [Abstract] | ||
Risk-free interest rate | 0.89% | 0.37% |
Expected term (in years) | 6 years 2 months 8 days | 6 years 29 days |
Expected volatility | 91.00% | 91.00% |
Dividend yield | 0.00% | 0.00% |
Stockholders Equity and Stock_3
Stockholders Equity and Stock Options - Summary of Stock Option Activity (Details) - USD ($) $ / shares in Units, $ in Millions | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | ||
Stockholders' Equity Note [Abstract] | |||
Number of shares, Outstanding | 2,008,902 | ||
Number of shares, Granted | 626,750 | ||
Number of shares, Forfeited/Expired | (112,347) | ||
Number of shares, Outstanding | 2,523,305 | 2,008,902 | |
Number of shares, Exercisable as of December 31, 2021 | 1,027,858 | ||
Number of shares, Vested and expected to vest as of December 31, 2021 | 2,523,305 | ||
Weighted average exercise price, balance | $ 22.31 | ||
Weighted average exercise price, Granted | 15.56 | ||
Weighted average exercise price, Forfeited/Expired | 61.80 | ||
Weighted average exercise price, balance | 18.88 | $ 22.31 | |
Weighted average exercise price, Exercisable as of December 31, 2021 | 26.52 | ||
Weighted average exercise price, Vested and expected to vest as of December 31, 2021 | $ 18.88 | ||
Weighted average remaining contractual term, outstanding | 7 years 7 months 6 days | 7 years 10 months 24 days | |
Weighted average remaining contractual term, Exercisable as of December 31, 2021 | 5 years 10 months 24 days | ||
Weighted average remaining contractual term, Vested and expected to vest as of December 31, 2021 | 7 years 7 months 6 days | ||
Aggregate intrinsic value, outstanding | [1] | $ 0.2 | |
Aggregate intrinsic value, Vested and expected to vest as of December 31, 2021 | [1] | $ 0.2 | |
[1] | The aggregate intrinsic value is calculated as the difference between the exercise price of the underlying options and the fair value of the common stock for the options that were in the money at December 31, 2021 . |
Stockholders Equity and Stock_4
Stockholders Equity and Stock Options - Summary of Stock-Based Compensation Expense (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Total stock-based compensation expense | $ 5,473 | $ 2,161 |
Research and Development [Member] | ||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Total stock-based compensation expense | 2,104 | 788 |
General and Administrative [Member] | ||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Total stock-based compensation expense | $ 3,369 | $ 1,373 |
Commitments and Contingencies -
Commitments and Contingencies - Additional Information (Detail) $ in Thousands | Aug. 09, 2021 | Oct. 27, 2020 | Aug. 14, 2020 | May 28, 2020USD ($)ft² | Aug. 08, 2019 | Nov. 05, 2018 | Dec. 31, 2021USD ($) | Dec. 31, 2020USD ($) | Mar. 31, 2020USD ($) |
Other Commitments [Line Items] | |||||||||
Milestone expenses | $ 0 | $ 0 | |||||||
Milestone payments due | 0 | 0 | |||||||
Operating lease right-of-use assets | 3,406 | $ 3,936 | |||||||
Operating lease liability | $ 6,002 | ||||||||
Weighted average remaining lease term in years | 7 years 7 months 6 days | 8 years 7 months 6 days | |||||||
Weighted average discount rate | 11.00% | 11.00% | |||||||
Lease expenses | $ 300 | $ 700 | |||||||
Office [Member] | Pennsylvania [Member] | |||||||||
Other Commitments [Line Items] | |||||||||
Operating lease term | 3 years 6 months | ||||||||
Lease extension period | 3 years | ||||||||
Operating lease right-of-use assets | $ 400 | ||||||||
Operating lease liability | $ 400 | ||||||||
Lease term commencement date | Feb. 15, 2020 | ||||||||
Office [Member] | Massachusetts [Member] | |||||||||
Other Commitments [Line Items] | |||||||||
Area of office space | ft² | 17,705 | ||||||||
Office [Member] | Massachusetts [Member] | Letter of Credit [Member] | |||||||||
Other Commitments [Line Items] | |||||||||
Restricted cash | $ 1,300 | ||||||||
Office Sublease [Member] | Massachusetts [Member] | First Sublease Year [Member] | |||||||||
Other Commitments [Line Items] | |||||||||
Annual base rent | 800 | ||||||||
Office Sublease [Member] | Massachusetts [Member] | First Sublease Year [Member] | Letter of Credit [Member] | |||||||||
Other Commitments [Line Items] | |||||||||
Lease security deposits letters of credit | 800 | ||||||||
Office Sublease [Member] | Massachusetts [Member] | Final Sublease Year [Member] | |||||||||
Other Commitments [Line Items] | |||||||||
Annual base rent | 1,000 | ||||||||
Office Sublease [Member] | Massachusetts [Member] | Sixth Sublease Year [Member] | Letter of Credit [Member] | |||||||||
Other Commitments [Line Items] | |||||||||
Lease security deposits letters of credit | $ 400 | ||||||||
Office Sublease [Member] | Massachusetts [Member] | |||||||||
Other Commitments [Line Items] | |||||||||
Lease term commencement date | Dec. 4, 2020 | ||||||||
Lease term expiration date | Oct. 30, 2029 | ||||||||
Office and Lab [Member] | Pennsylvania [Member] | |||||||||
Other Commitments [Line Items] | |||||||||
Lease extension period | 2 years | ||||||||
Lease term commencement date | Jan. 1, 2019 | ||||||||
Lease term expiration date | Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2020 | ||||||
WFUHS [Member] | |||||||||
Other Commitments [Line Items] | |||||||||
License agreement consideration deduction percentage | 60.00% | ||||||||
WFUHS [Member] | Maximum [Member] | |||||||||
Other Commitments [Line Items] | |||||||||
Milestone payments | $ 2,600 | ||||||||
IU [Member] | |||||||||
Other Commitments [Line Items] | |||||||||
License agreement consideration deduction percentage | 20.00% | ||||||||
IU [Member] | Maximum [Member] | |||||||||
Other Commitments [Line Items] | |||||||||
Milestone payments | $ 2,600 | ||||||||
Annual royalty pay obligation | $ 100 |
Commitments and Contingencies_2
Commitments and Contingencies - Schedule of Maturities of Lease Liabilities Due Under Lease Agreements (Detail) $ in Thousands | Dec. 31, 2021USD ($) |
Leases [Abstract] | |
2022 | $ 1,197 |
2023 | 1,146 |
2024 | 1,065 |
2025 | 1,083 |
2026 | 1,101 |
Thereafter | 3,213 |
Total lease payments | 8,805 |
Less: imputed interest | (2,803) |
Present value of lease liabilities | $ 6,002 |
Income Taxes - Additional Infor
Income Taxes - Additional Information (Detail) - USD ($) | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Income Taxes Disclosure [Line Items] | ||
Income tax benefit | $ 0 | $ 0 |
Operating loss carryforwards, minimum percentage of taxable income in tax period | 80.00% | |
Net capitalized R&D deferred tax assets | $ 65,781,000 | 73,014,000 |
Net deferred tax assets before the valuation allowance | 124,000,000 | 115,300,000 |
Unrecognized tax benefits | 0 | $ 0 |
Zafgen [Member] | ||
Income Taxes Disclosure [Line Items] | ||
Net operating loss carryforwards | 33,500,000 | |
Net capitalized R&D deferred tax assets | $ 73,000,000 | |
Net capitalized R&D deferred tax assets realization description | is subject to the built-in loss rules under Section 382 and may not be realized if the underlying asset associated with the R&D is disposed within five years of the Merger, or May 28, 2025 | |
Net operating loss carryforwards, change in ownership, annual utilization limit | $ 35,000 | |
Earliest Tax Year [Member] | ||
Income Taxes Disclosure [Line Items] | ||
Open tax year | 2016 | |
Domestic Tax Authority [Member] | ||
Income Taxes Disclosure [Line Items] | ||
Net operating loss carryforwards | $ 158,800,000 | |
Net operating loss carryforwards, beginning of expiration period | 2026 | |
Domestic Tax Authority [Member] | No Expiration [Member] | ||
Income Taxes Disclosure [Line Items] | ||
Net operating loss carryforwards | $ 120,000,000 | |
Operating loss carryforwards, description | carryforwards are limited to 80% of the taxable income in any one tax period. | |
Foreign Tax Authority [Member] | ||
Income Taxes Disclosure [Line Items] | ||
Net operating loss carryforwards | $ 1,200,000 | |
State and Local Jurisdiction [Member] | ||
Income Taxes Disclosure [Line Items] | ||
Net operating loss carryforwards | $ 131,300,000 | |
Net operating loss carryforwards, beginning of expiration period | 2030 | |
Federal and State [Member] | ||
Income Taxes Disclosure [Line Items] | ||
Tax credit carryforward | $ 12,900,000 | |
Research and development tax credit carry forwards, beginning of expiration period | 2039 |
Income Taxes - Schedule of Dome
Income Taxes - Schedule of Domestic and Foreign Components of Loss Before Income Taxes (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Income Tax Disclosure [Abstract] | ||
Domestic | $ (50,617) | $ (42,608) |
Foreign | (19) | 126 |
Loss before income taxes | $ (50,636) | $ (42,482) |
Income Taxes - Reconciliation o
Income Taxes - Reconciliation of Federal Statutory Income Tax Rate (Detail) | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Income Tax Disclosure [Abstract] | ||
Federal statutory income tax rate | 21.00% | 21.00% |
State taxes, net of federal benefit | 3.40% | 7.80% |
Change in state tax rate | (14.50%) | |
Federal and state research and development tax credit | 7.90% | 13.50% |
Nondeductible permanent differences | (0.60%) | (0.20%) |
Change in deferred tax asset valuation allowance | (17.20%) | (42.10%) |
Effective income tax rate | 0.00% | 0.00% |
Income Taxes - Schedule of Net
Income Taxes - Schedule of Net Deferred Tax Assets (Detail) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 |
Deferred tax assets: | |||
Capitalized R&D Expenses | $ 65,781 | $ 73,014 | |
Stock Based Compensation | 1,458 | 490 | |
Net operating Loss Carryforwards | 43,219 | 32,081 | |
Tax credit carryforwards | 12,912 | 8,904 | |
Other Temporary Differences | 24 | 50 | |
Fixed Assets & Intangibles | 44 | 75 | |
Operating Lease Liability | 1,477 | 1,786 | |
Total deferred tax assets | 124,915 | 116,400 | |
Deferred tax liabilities: | |||
Operating Right of Use Asset | (899) | (1,081) | |
Total deferred tax liabilities | (899) | (1,081) | |
Less: Valuation allowance | (124,016) | (115,319) | $ (11,735) |
Net deferred tax assets / (liabilities) | $ 0 | $ 0 |
Income Taxes - Summary of Chang
Income Taxes - Summary of Changes in the Valuation Allowance for Deferred Tax Assets (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Income Taxes Disclosure [Line Items] | ||
Valuation allowance as of the beginning of the year | $ 115,319 | $ 11,735 |
Increases recorded to income tax provision | 8,697 | 17,895 |
Valuation Allowance at end of Year | $ 124,016 | 115,319 |
Zafgen [Member] | ||
Income Taxes Disclosure [Line Items] | ||
Increases acquired as a result of the Merger with Zafgen | $ 85,689 |
Related Party Transactions - Ad
Related Party Transactions - Additional Information (Details) - USD ($) $ in Thousands | Jan. 16, 2020 | Nov. 21, 2019 | Nov. 30, 2016 | Nov. 30, 2020 | Nov. 30, 2016 | Dec. 31, 2021 | Dec. 31, 2020 |
Related Party Transaction [Line Items] | |||||||
Research and development expense | $ 38,396 | $ 31,407 | |||||
Capital contributions from related party | 17,995 | ||||||
Laboratory Equipment [Member] | |||||||
Related Party Transaction [Line Items] | |||||||
Purchased of equipment | 100 | 500 | |||||
Holdings [Member] | Series A Preferred Unit Purchase Agreement [Member] | |||||||
Related Party Transaction [Line Items] | |||||||
Gross proceeds from sale of equity | $ 35,600 | ||||||
Capital contributions from related party | 35,600 | ||||||
Holdings [Member] | Series B Bridge Unit Purchase Agreement [Member] | |||||||
Related Party Transaction [Line Items] | |||||||
Capital contributions from related party | $ 10,000 | ||||||
Holdings [Member] | Series B Bridge Unit Purchase Agreement [Member] | Maximum [Member] | |||||||
Related Party Transaction [Line Items] | |||||||
Gross proceeds from sale of equity | $ 10,000 | ||||||
Holdings [Member] | Second Series B Bridge Unit Purchase Agreement [Member] | |||||||
Related Party Transaction [Line Items] | |||||||
Capital contributions from related party | $ 11,400 | ||||||
Holdings [Member] | Second Series B Bridge Unit Purchase Agreement [Member] | Maximum [Member] | |||||||
Related Party Transaction [Line Items] | |||||||
Gross proceeds from sale of equity | $ 15,000 | ||||||
Holdings [Member] | Series A and Series B [Member] | |||||||
Related Party Transaction [Line Items] | |||||||
Capital contributions from related party | 18,000 | ||||||
Consulting Agreement [Member] | |||||||
Related Party Transaction [Line Items] | |||||||
Amount to be paid for consulting services | $ 100 | ||||||
Research and development expense | $ 100 | $ 100 | |||||
Period of agreement | 4 years | ||||||
Extented period of agreement | 1 month | ||||||
Agreement expiration date | Dec. 31, 2020 | ||||||
Consulting Agreement [Member] | Holdings [Member] | |||||||
Related Party Transaction [Line Items] | |||||||
Restricted common units granted | 123,853 | ||||||
Award vesting | 30.00% | ||||||
Consulting Agreement [Member] | Future Services [Member] | Vests Over Forty Eight Months [Member] | |||||||
Related Party Transaction [Line Items] | |||||||
Award vesting | 70.00% | ||||||
Consulting Agreement [Member] | Future Services [Member] | Remaining Restricted Common Units [Member] | |||||||
Related Party Transaction [Line Items] | |||||||
Vesting period of stock option | 48 months | ||||||
New Consulting Agreement [Member] | |||||||
Related Party Transaction [Line Items] | |||||||
Period of agreement | 4 years |