Document and Entity Information
Document and Entity Information - USD ($) $ in Billions | 12 Months Ended | ||
Dec. 31, 2021 | Feb. 22, 2022 | Jun. 30, 2021 | |
Cover [Abstract] | |||
Document Type | 10-K | ||
Amendment Flag | false | ||
Document Annual Report | true | ||
Document Period End Date | Dec. 31, 2021 | ||
Current Fiscal Year End Date | --12-31 | ||
Document Fiscal Year Focus | 2021 | ||
Document Fiscal Period Focus | FY | ||
Document Transition Report | false | ||
Entity File Number | 001-36829 | ||
Entity Registrant Name | Rocket Pharmaceuticals, Inc. | ||
Entity Central Index Key | 0001281895 | ||
Entity Incorporation, State or Country Code | DE | ||
Entity Tax Identification Number | 04-3475813 | ||
Entity Address, Address Line One | 9 Cedarbrook Drive | ||
Entity Address, City or Town | Cranbury | ||
Entity Address, State or Province | NJ | ||
Entity Address, Postal Zip Code | 08512 | ||
City Area Code | 609 | ||
Local Phone Number | 659-8001 | ||
Title of 12(b) Security | Common Stock, $0.01 par value | ||
Trading Symbol | RCKT | ||
Security Exchange Name | NASDAQ | ||
Entity Well-known Seasoned Issuer | No | ||
Entity Voluntary Filers | No | ||
Entity Current Reporting Status | Yes | ||
Entity Interactive Data Current | Yes | ||
Entity Filer Category | Large Accelerated Filer | ||
Entity Small Business | false | ||
Entity Emerging Growth Company | false | ||
ICFR Auditor Attestation Flag | true | ||
Entity Shell Company | false | ||
Entity Public Float | $ 1.9 | ||
Entity Common Stock, Shares Outstanding | 64,505,889 | ||
Auditor Firm ID | 274 | ||
Auditor Name | EISNERAMPER LLP | ||
Auditor Location | Iselin, New Jersey |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Current assets: | ||
Cash and cash equivalents | $ 232,694 | $ 297,098 |
Investments | 156,046 | 185,621 |
Prepaid expenses and other current assets | 3,319 | 4,626 |
Total current assets | 392,059 | 487,345 |
Property and equipment, net | 22,299 | 19,206 |
Goodwill | 30,815 | 30,815 |
Restricted cash | 1,343 | 1,568 |
Deposits | 455 | 455 |
Operating lease right-of-use assets | 1,569 | 914 |
Finance lease right-of-use asset | 48,480 | 50,521 |
Total assets | 497,020 | 590,824 |
Current liabilities: | ||
Accounts payable and accrued expenses | 19,615 | 25,472 |
Convertible notes, net of unamortized discount, current | 0 | 4,875 |
Operating lease liabilities, current | 863 | 626 |
Finance lease liability, current | 1,689 | 1,644 |
Total current liabilities | 22,167 | 32,617 |
Convertible notes, net of unamortized discount, non-current | 0 | 35,066 |
Operating lease liabilities, non-current | 905 | 498 |
Finance lease liability, non-current | 19,144 | 18,988 |
Other liabilities | 80 | 136 |
Total liabilities | 42,296 | 87,305 |
Commitments and contingencies (Note 12) | ||
Stockholders' equity: | ||
Common stock, $0.01 par value, 120,000,000 shares authorized; 64,505,889 and 60,996,367 shares issued and outstanding at December 31, 2021 and 2020, respectively | 645 | 610 |
Additional paid-in capital | 946,152 | 825,794 |
Accumulated other comprehensive loss | (161) | (42) |
Accumulated deficit | (491,912) | (322,843) |
Total stockholders' equity | 454,724 | 503,519 |
Total liabilities and stockholders' equity | 497,020 | 590,824 |
Series A Convertible Preferred Stock [Member] | ||
Stockholders' equity: | ||
Preferred stock | 0 | 0 |
Series B Convertible Preferred Stock [Member] | ||
Stockholders' equity: | ||
Preferred stock | $ 0 | $ 0 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - $ / shares | Dec. 31, 2021 | Dec. 31, 2020 |
Shareholders' equity: | ||
Preferred stock, par value (in dollars per share) | $ 0.01 | $ 0.01 |
Preferred stock, shares authorized (in shares) | 5,000,000 | 5,000,000 |
Common stock, par value (in dollars per share) | $ 0.01 | $ 0.01 |
Common stock, shares authorized (in shares) | 120,000,000 | 120,000,000 |
Common stock, shares issued (in shares) | 64,505,889 | 60,996,367 |
Common stock, shares outstanding (in shares) | 64,505,889 | 60,996,367 |
Series A Convertible Preferred Stock [Member] | ||
Shareholders' equity: | ||
Preferred stock, shares authorized (in shares) | 300,000 | 300,000 |
Preferred stock, shares issued (in shares) | 0 | 0 |
Preferred stock, shares outstanding (in shares) | 0 | 0 |
Series B Convertible Preferred Stock [Member] | ||
Shareholders' equity: | ||
Preferred stock, shares authorized (in shares) | 300,000 | 300,000 |
Preferred stock, shares issued (in shares) | 0 | 0 |
Preferred stock, shares outstanding (in shares) | 0 | 0 |
Consolidated Statements of Oper
Consolidated Statements of Operations - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Consolidated Statements of Operations [Abstract] | |||
Revenue | $ 0 | $ 0 | $ 0 |
Operating expenses: | |||
Research and development | 125,476 | 105,438 | 57,907 |
General and administrative | 41,772 | 28,865 | 18,244 |
Total operating expenses | 167,248 | 134,303 | 76,151 |
Loss from operations | (167,248) | (134,303) | (76,151) |
Research and development incentives | 1,000 | 0 | 250 |
Interest expense | (2,977) | (6,967) | (5,958) |
Interest and other income, net | 3,068 | 2,150 | 3,414 |
(Amortization of premium) accretion of discount on investments - net | (2,912) | (580) | 1,175 |
Net loss | $ (169,069) | $ (139,700) | $ (77,270) |
Net loss per share attributable to common stockholders - basic (in dollars per share) | $ (2.67) | $ (2.52) | $ (1.58) |
Net loss per share attributable to common stockholders - diluted (in dollars per share) | $ (2.67) | $ (2.52) | $ (1.58) |
Weighted-average common shares outstanding - basic (in shares) | 63,235,417 | 55,380,740 | 49,010,358 |
Weighted-average common shares outstanding - diluted (in shares) | 63,235,417 | 55,380,740 | 49,010,358 |
Consolidated Statements of Comp
Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Consolidated Statements of Comprehensive Loss [Abstract] | |||
Net loss | $ (169,069) | $ (139,700) | $ (77,270) |
Other comprehensive loss | |||
Net unrealized (loss) gain on investments | (119) | (62) | 147 |
Total comprehensive loss | $ (169,188) | $ (139,762) | $ (77,123) |
Consolidated Statements of Chan
Consolidated Statements of Changes in Stockholders' Equity - USD ($) $ in Thousands | Common Stock [Member] | Treasury Stock [Member] | Additional Paid-In Capital [Member] | Accumulated Other Comprehensive Income/(Loss) [Member] | Accumulated Deficit [Member] | Total |
Beginning Balance at Dec. 31, 2018 | $ 452 | $ (668) | $ 300,253 | $ (127) | $ (105,873) | $ 194,037 |
Beginning Balance (in shares) at Dec. 31, 2018 | 45,194,736 | |||||
Issuance of common stock, net of issuance costs | $ 96 | 0 | 177,664 | 0 | 0 | 177,760 |
Issuance of common stock, net of issuance costs (in shares) | 9,568,000 | |||||
Issuance of common stock pursuant to exercise of stock options | $ 1 | 0 | 30 | 0 | 0 | 31 |
Issuance of common stock pursuant to exercise of stock options (in shares) | 110,325 | |||||
Treasury stock purchases | $ 0 | (725) | 0 | 0 | (726) | |
Treasury stock purchases | (1) | |||||
Treasury stock purchases (in shares) | 0 | |||||
Issuance of treasury stock pursuant to exercise of stock options | $ 0 | (397) | 0 | 0 | 0 | (397) |
Issuance of treasury stock pursuant to exercise of stock options (in shares) | 0 | |||||
Retirement of treasury stock | $ (1) | 1,393 | (1,392) | 0 | 0 | 0 |
Retirement of treasury stock (in shares) | (100,000) | |||||
Sale of treasury stock | $ 0 | 344 | 0 | 0 | 0 | 344 |
Sale of treasury stock (in shares) | 0 | |||||
Unrealized comprehensive gain (loss) on investments | $ 0 | 0 | 0 | 147 | 0 | 147 |
Share-based compensation | 0 | 0 | 13,371 | 0 | 0 | 13,371 |
Net loss | 0 | 0 | 0 | 0 | (77,270) | (77,270) |
Ending Balance at Dec. 31, 2019 | $ 548 | (53) | 489,925 | 20 | (183,143) | 307,297 |
Ending Balance (in shares) at Dec. 31, 2019 | 54,773,061 | |||||
Issuance of common stock, net of issuance costs | $ 53 | 0 | 280,710 | 0 | 0 | 280,763 |
Issuance of common stock, net of issuance costs (in shares) | 5,339,286 | |||||
Issuance of common stock pursuant to exercise of stock options | $ 6 | 0 | 2,552 | 0 | 0 | 2,558 |
Issuance of common stock pursuant to exercise of stock options (in shares) | 586,857 | |||||
Issuance of treasury stock pursuant to exercise of stock options | $ 0 | (614) | 0 | 0 | 0 | (614) |
Issuance of treasury stock pursuant to exercise of stock options (in shares) | 0 | |||||
Issuance of common stock pursuant to exercise of warrant (in shares) | 1,601 | |||||
Issuance of common stock pursuant to conversion of notes | $ 3 | 0 | 7,626 | 0 | 0 | 7,629 |
Issuance of common stock pursuant to conversion of notes (in shares) | 298,562 | |||||
Issuance of warrants | 26,562 | 26,562 | ||||
Stock repurchase | $ 0 | 0 | (72) | 0 | 0 | (72) |
Stock repurchase (in shares) | (3,000) | |||||
Sale of treasury stock | $ 0 | 667 | (76) | 0 | 0 | 591 |
Sale of treasury stock (in shares) | 0 | |||||
Unrealized comprehensive gain (loss) on investments | $ 0 | 0 | 0 | (62) | 0 | (62) |
Share-based compensation | 0 | 0 | 18,567 | 0 | 0 | 18,567 |
Net loss | 0 | 0 | 0 | 0 | (139,700) | (139,700) |
Ending Balance at Dec. 31, 2020 | $ 610 | 0 | 825,794 | (42) | (322,843) | 503,519 |
Ending Balance (in shares) at Dec. 31, 2020 | 60,996,367 | |||||
Issuance of common stock, net of issuance costs | $ 8 | 0 | 26,346 | 0 | 0 | 26,354 |
Issuance of common stock, net of issuance costs (in shares) | 812,516 | |||||
Issuance of common stock pursuant to exercise of stock options | $ 12 | 0 | 11,315 | 0 | 0 | 11,327 |
Issuance of common stock pursuant to exercise of stock options (in shares) | 1,209,960 | |||||
Issuance of common stock pursuant to conversion of notes | $ 15 | 0 | 40,679 | 0 | 0 | 40,694 |
Issuance of common stock pursuant to conversion of notes (in shares) | 1,487,046 | |||||
Issuance of warrants | 12,781 | 12,781 | ||||
Unrealized comprehensive gain (loss) on investments | $ 0 | 0 | 0 | (119) | 0 | (119) |
Share-based compensation | 0 | 0 | 29,237 | 0 | 0 | 29,237 |
Net loss | 0 | 0 | 0 | 0 | (169,069) | (169,069) |
Ending Balance at Dec. 31, 2021 | $ 645 | $ 0 | $ 946,152 | $ (161) | $ (491,912) | $ 454,724 |
Ending Balance (in shares) at Dec. 31, 2021 | 64,505,889 |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Operating Activities: | |||
Net loss | $ (169,069) | $ (139,700) | $ (77,270) |
Adjustments to reconcile net loss to net cash used in operating activities: | |||
Accretion of discount on convertible notes | 753 | 2,758 | 3,602 |
Depreciation and amortization of property and equipment | 3,240 | 1,145 | 426 |
Amortization of right of use asset | 2,133 | 5,105 | 0 |
Write down of property and equipment, net | 261 | 419 | 0 |
Stock-based compensation | 29,237 | 18,567 | 13,371 |
Expense in connection with warrant issuances | 12,781 | 26,562 | 0 |
Accretion of discount (amortization of premium) on investments, net | 2,887 | 580 | (1,066) |
Changes in operating assets and liabilities: | |||
Prepaid expenses and other assets | 1,307 | (1,517) | (917) |
Accounts payable and accrued expenses | (4,827) | 11,015 | (2,722) |
Operating lease liabilities | (11) | (139) | (87) |
Finance lease liability | 201 | 452 | 0 |
Other long term liabilities | (56) | 113 | 0 |
Net cash used in operating activities | (121,163) | (74,640) | (64,663) |
Investing activities: | |||
Purchases of investments | (245,875) | (209,343) | (184,298) |
Proceeds from maturities of investments | 272,443 | 141,811 | 168,556 |
Payments made to acquire right of use asset | (95) | (8,452) | 0 |
Purchases of property and equipment | (7,620) | (20,607) | (23,269) |
Net cash provided by (used in) investing activities | 18,853 | (96,591) | (39,011) |
Financing activities: | |||
Issuance of common stock, net of issuance costs | 26,354 | 280,763 | 177,760 |
Issuance of common stock, pursuant to exercise of stock options | 11,327 | 2,558 | 31 |
Common stock repurchase | 0 | (72) | 0 |
Proceeds from sale of treasury stock | 0 | 591 | 344 |
Payment of withholding tax on option exercises | 0 | (614) | (344) |
Convertible notes refinancing costs to the lender | 0 | (237) | 0 |
Net cash provided by financing activities | 37,681 | 282,989 | 177,791 |
Net change in cash, cash equivalents and restricted cash | (64,629) | 111,758 | 74,117 |
Cash, cash equivalents and restricted cash at beginning of period | 298,666 | 186,908 | 112,791 |
Cash, cash equivalents and restricted cash at end of period | 234,037 | 298,666 | 186,908 |
Supplemental disclosure of non-cash financing and investing activities: | |||
Accrued purchases of property and equipment | 728 | 1,756 | 4,650 |
Retirement of treasury stock | 0 | 0 | 1,393 |
Treasury stock purchases paid in prior year | 0 | 0 | 726 |
Withholding tax payable on shares withheld in treasury stock | 0 | 0 | 53 |
Unrealized (loss) gain on investments | (119) | (62) | 147 |
Conversion of 2021 and 2022 convertible notes into common stock | 40,694 | 7,629 | 0 |
Finance lease right of use asset and lease liability | 0 | 20,179 | 0 |
Reclassification of construction in process to finance right of use asset | 98 | 26,465 | 0 |
Supplemental cash flow information: | |||
Cash paid for interest | 148 | 2,960 | 2,990 |
Cash paid for income taxes | $ 0 | $ 0 | $ 26 |
Nature of Business and Basis of
Nature of Business and Basis of Presentation | 12 Months Ended |
Dec. 31, 2021 | |
Nature of Business and Basis of Presentation [Abstract] | |
Nature of Business and Basis of Presentation | 1. Nature of Business and Basis of Presentation Rocket Pharmaceuticals, Inc. (“Rocket” or the “Company”) is ex vivo ” Of these, both the Phase 2 FA program and the Phase 1/2 LAD-I program are in registration-enabling studies in the United States (“U.S.”) and Europe (“EU”). In addition, in the U.S., Rocket has a clinical stage in vivo adeno-associated virus (“AAV”) program for Danon disease, a multi-organ lysosomal-associated disorder leading to early death due to heart failure. Additional discovery efforts on a gene therapy program for the less common FA subtypes C and G is ongoing. The Company has global commercialization and development rights to all of these product candidates under royalty-bearing license agreements Effective December 2021, the Company made a decision to no longer pursue Rocket-sponsored clinical evaluation of RP-L401; this program is to be returned to academic innovators. The Company has opted to focus available resources towards advancement of RP-A501, RP-L102, RP-L201 and RP-L301, based on the clinical data to date and potential for therapeutic advancement in these severe disorders of childhood and young adulthood. |
Risks and Liquidity
Risks and Liquidity | 12 Months Ended |
Dec. 31, 2021 | |
Risks and Liquidity [Abstract] | |
Risks and Liquidity | 2. Risks and Liquidity The Company has not generated any revenue and has incurred losses since inception. Operations of the Company are subject to certain risks and uncertainties, including, among others, uncertainty of drug candidate development, technological uncertainty, uncertainty regarding patents and proprietary rights, having no commercial manufacturing experience, marketing or sales capability or experience, dependency on key personnel, compliance with government regulations and the need to obtain additional financing. Drug candidates currently under development will require significant additional research and development efforts, including extensive preclinical and clinical testing and regulatory approval, prior to commercialization. These efforts require significant amounts of additional capital, adequate personnel infrastructure, and extensive compliance-reporting capabilities. The Company’s product candidates are in the development and clinical stage. There can be no assurance that the Company’s research and development will be successfully completed, that adequate protection for the Company’s intellectual property will be obtained, that any products developed will obtain necessary government approval or that any approved products will be commercially viable. Even if the Company’s product development efforts are successful, it is uncertain when, if ever, the Company will generate significant revenue from product sales. The Company operates in an environment of rapid change in technology and substantial competition from pharmaceutical and biotechnology companies. The Company’s consolidated financial statements have been prepared on the basis of continuity of operations, realization of assets and the satisfaction of liabilities in the ordinary course of business. The Company has experienced negative cash flows from operations and had an accumulated deficit of $491.9 million as of December 31, 2021. As of December 31, 2021, the Company has $388.7 million of cash, cash equivalents and investments. During the year ended December 31, 2021, the Company issued and sold 812,516 shares of its common stock at a purchase price of $32.48 per share for aggregate net proceeds of approximately $26.4 million in a private placement transaction to a fund affiliated with RTW Investments, LP., the Company’s largest shareholder (see Note 15) . The Compan half of In April the Company called for the redemption of the remaining principal balance of the Company’s , Convertible Senior Notes due 2022 which were converted into common stock. On August holders of of the Convertible Notes converted the remaining remaining balance of the Convertible Notes into common stock (see Note . As of December none of the Convertible Notes or Convertible Notes were outstanding. In the longer term, the future viability of the Company is dependent on its ability to generate cash from operating activities or to raise additional capital to finance its operations. The Company’s failure to raise capital as and when needed could have a negative impact on its financial condition and ability to pursue its business strategies. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 12 Months Ended |
Dec. 31, 2021 | |
Summary of Significant Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 3. Summary of Significant Accounting Policies Principles of Consolidation The consolidated financial statements represent the consolidation of the accounts of the Company and its subsidiaries in conformity with accounting principles generally accepted in the United States (“US GAAP”). All intercompany accounts have been eliminated in consolidation. Use of Estimates The preparation of the consolidated financial statements in conformity with US 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 goodwill impairment, the accrual of research and development expenses, the valuation of equity transactions, and stock-based awards. Changes in estimates and assumptions are reflected in reported results in the period in which they become known. Actual results could differ from those estimates. Cash, Cash Equivalents and Restricted Cash Cash, cash equivalents and restricted cash consists of bank deposits, certificates of deposit and money market accounts with financial institutions. Cash equivalents are carried at cost which approximates fair value due to their short-term nature and which the Company believes do not have a material exposure to credit risk. The Company considers all highly liquid investments with maturities of three months or less from the date of purchase to be cash equivalents. The Company’s cash and cash equivalent accounts, at times, may exceed federally insured limits. The Company has not experienced any losses in such accounts. Restricted cash consists of deposits collateralizing letters of credit issued by a bank in connection with the Company’s operating leases (see Note 12 “Commitments and Contingencies” for additional disclosures) and a deposit collateralizing a letter of credit issued by a bank supporting the Company’s corporate credit card. Cash, cash equivalents and restricted cash consist of the following: December 31, 2021 December 31, 2020 Cash and cash equivalents $ 232,694 $ 297,098 Restricted cash 1,343 1,568 $ 234,037 $ 298,666 Government Grants Research and development expense is presented net of reimbursements from the California Institute for Regenerative Medicine (“CIRM”), which are recognized over the period necessary to match the reimbursement with the related costs when it is probable that the Company has complied with the CIRM conditions and will receive the reimbursement. During the years ended December 31, 2021, 2020, and 2019, the Company offset $0.1 million, $3.6 million and $1.2 million of CIRM grant funds against research and development (“R&D”) expenses (See Note 14 “CIRM Grant” for additional disclosure). Concentrations of credit risk and off-balance sheet risk Financial instruments that subject the Company to credit risk primarily consist of cash and cash equivalents and available-for-sale securities. The Company maintains its cash and cash equivalent balances with high-quality financial institutions and, consequently, the Company believes that such funds are subject to minimal credit risk. The Company’s marketable securities consist of U.S. Treasury securities, and Corporate, Government Municipal and Agency Bonds. The Company has adopted an investment policy that limits the amounts the Company may invest in any one type of investment and requires all investments held by the Company to be at least AA+/Aa1 rated, thereby reducing credit risk exposure. Investments Investments consist of investments in United States Treasury securities and Corporate, Municipal and Agency Bonds. Management determines the appropriate classification of these securities at the time they are acquired and evaluates the appropriateness of such classifications at each balance sheet date. The Company classifies its investments as available-for-sale pursuant to Financial Accounting Standards Board (“FASB”) Accounting Standard Codification (“ASC”) 320, Investments—Debt and Equity Securities Goodwill Business combinations are accounted for under the acquisition method. The total cost of an acquisition is allocated to the underlying identifiable net assets, based on their respective estimated fair values as of the acquisition date. Determining the fair value of assets acquired and liabilities assumed requires management’s judgment and often involves the use of significant estimates and assumptions, including assumptions with respect to future cash inflows and outflows, discount rates, asset lives and market multiples, among other items. Assets acquired and liabilities assumed are recorded at their estimated fair values. The excess of the purchase price over the estimated fair values of the net assets acquired is recorded as goodwill. Goodwill is tested for impairment annually as of December 31, or more frequently when events or changes in circumstances indicate that the asset might be impaired. Examples of such events or circumstances include, but are not limited to, a significant adverse change in legal or business climate, an adverse regulatory action or unanticipated competition. The Company has one segment and one reporting unit and as such review’s goodwill for impairment at the consolidated level. When testing goodwill, the Company has the option to first assess qualitative factors for reporting units that carry goodwill. The qualitative assessment includes assessing the totality of relevant events and circumstances that affect the fair value or carrying value of the reporting unit. These events and circumstances include macroeconomic conditions, industry and competitive environment conditions, overall financial performance, reporting unit specific events and market considerations. The Company also considers recent valuations of the reporting unit, including the magnitude of the difference between the most recent fair value estimate and the carrying value, as well as both positive and adverse events and circumstances, and the extent to which each of the events and circumstances identified may affect the comparison of a reporting unit’s fair value with its carrying value. If the qualitative assessment results in a conclusion that it is more likely than not that the fair value of a reporting unit exceeds the carrying value, then no further testing is performed for that reporting unit. The Company performed the qualitative assessment of its goodwill and determined that it is more likely than not that the fair value of a reporting unit exceeds the carrying value of the reporting unit. As a result, the Company has determined there was no goodwill impairment as of and for the years ended December 31, 2021, 2020 and 2019. Property and Equipment Property and equipment are stated at cost less accumulated depreciation. Depreciation expense is recognized using the straight-line method over the useful life of the asset. The estimated useful lives are three Impairment of Long-Lived Assets The Company reviews long-lived assets for impairment whenever events or changes in circumstances indicate that the asset’s carrying amount may not be recoverable. The Company conducted our long-lived asset impairment analyses in accordance with ASC 360-10-15, “Impairment or Disposal of Long-Lived Assets.” ASC 360-10-15 requires us to group assets and liabilities at the lowest level for which identifiable cash flows are largely independent of the cash flows of other assets and liabilities and evaluate the asset group against the sum of the undiscounted future cash flows. If the undiscounted cash flows do not indicate the carrying amount of the asset is recoverable, an impairment charge is measured as the amount by which the carrying amount of the asset group exceeds its fair value based on discounted cash flow analysis or appraisals. There is no impairment of long-lived assets as of and for the years ended December 31, 2021, 2020 and 2019. Fair Value Measurements The Company is required to disclose information on all assets and liabilities reported at fair value that enables an assessment of the inputs used in determining the reported fair values. FASB ASC 820, Fair Value Measurements and Disclosures Level 1—Valuations based on unadjusted quoted prices in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date. Level 2—Valuations based on quoted prices for similar assets or liabilities in markets that are not active or for which all significant inputs are observable, either directly or indirectly. Level 3—Valuations that require inputs that reflect the Company’s own assumptions that are both significant to the fair value measurement and unobservable. To the extent that valuation is based on models or inputs that are less observable or unobservable in the market, the determination of fair value requires more judgment. Accordingly, the degree of judgment exercised by the Company in determining fair value is greatest for instruments categorized in Level 3. A financial instrument’s level within the fair value hierarchy is based on the lowest level of any input that is significant to the fair value measurement. The fair value of the Company’s financial instruments, including cash and cash equivalents, restricted cash, deposits, accounts payable and accrued expenses approximate their respective carrying values due to the short-term nature of these instruments. Research and Development Expenses R&D costs, which include salaries and staff costs, license costs, manufacturing and development costs, clinical trial expenses, regulatory and scientific consulting fees, as well as contract research, and stock-based compensation expense, are accounted for in accordance with ASC Topic 730, Research and Development (“ASC 730”). The Company does not currently have any commercial biopharmaceutical products and does not expect to have any for several years, if at all. Accordingly, R&D costs are expensed as incurred. While certain of the Company’s R&D costs may have future benefits, the policy of expensing all R&D expenditures is predicated on the fact that the Company has no history of successful commercialization of product candidates to base any estimate of the number of future periods that would be benefited. Foreign Currency Transactions Certain transactions during the years ended December 31, 2021, 2020 and 2019 are denominated in Euros and British pounds. Gains and losses on foreign currency transactions were not significant for the years ended December 31, 2021, 2020 and 2019. Treasury Stock The Company records treasury stock at cost. Stock-Based Compensation The Company issues stock-based awards to employees and non-employees, generally in the form of stock options and restricted stock units. The Company accounts for its stock-based compensation awards in accordance with FASB ASC Topic 718, Compensation—Stock Compensation (“ASC 718”). ASC 718 requires all stock-based payments, including grants of stock options and restricted stock units and modifications to existing stock options, to be recognized in the consolidated statements of operations and comprehensive loss based on their fair values. The Company measures the compensation expense of employee and nonemployee services received in exchange for an award of equity instruments based on the fair value of the award on the grant date. That cost is recognized on a straight-line basis over the period during which the employee and nonemployee is required to provide service in exchange for the award. The fair value of options on the date of grant is calculated using the Black-Scholes option pricing model based on key assumptions such as stock price, expected volatility and expected term. The Company’s estimates of these assumptions are primarily based on the trading price of the Company’s stock, historical data, peer company data and judgment regarding future trends and factors. The Company classifies stock-based compensation expense in its consolidated statement of operations in the same manner in which the award recipient’s payroll costs and services are classified or in which the award recipient’s service payments are classified. The Company recognizes compensation expense for at least the portion of awards that are vested. Forfeitures are accounted for as they occur. NYS Life Sciences Research and Development Tax Credit New York State allows investors and owners of emerging technology companies focused on biotechnology to claim a tax credit against their New York State Tax return for certain expenditures incurred in New York State, including applicable R&D related expenditures. The credit is recognized as research and development incentives when the eligibility and amount has been approved by New York State. During the years ended December 31, 2021, 2020 and 2019, the Company recorded research and development incentive income of $1.0 million, $0, and $0, respectively related to the NYS Life Sciences Research and Development Tax Credit. NYC Biotechnology Tax Credit Program New York City allowed investors and owners of emerging technology companies focused on biotechnology to claim a tax credit against the General Corporation Tax and Unincorporated Business Tax for amounts paid or incurred for certain facilities, operations, and employee training in New York City. The credit was recognized as research and development incentives when approved by New York City of the eligibility for the credit and the credit amount. During the years ended December 31, 2021, 2020 and 2019, the Company recorded research and development incentive income of $ , $ , and $ million, respectively related to the NYC Biotechnology Program. Income Taxes The Company accounts for income taxes under the asset and liability method. The Company recognizes deferred tax assets and liabilities for the future tax consequences attributable to differences between the financial statement carrying amounts of existing assets and liabilities and their respective tax bases, as well as for operating loss and tax credit carryforwards. The Company measures deferred tax assets and liabilities using enacted tax rates expected to apply to taxable income in the years in which the Company expects to recover or settle those temporary differences. The Company recognizes the effect of a change in tax rates on deferred tax assets and liabilities in the results of operations in the period that includes the enactment date. The Company reduces the measurement of a deferred tax asset, if necessary, by a valuation allowance if it is more likely than not that the Company will not realize some or all of the deferred tax asset. The Company’s deferred tax assets relate primarily to its net operating loss carryforwards and other balance sheet differences. In accordance with ASC 740 “Income Taxes”, the Company recorded a full valuation allowance to fully offset the net deferred tax asset because it is not more likely than not that the Company will realize future benefits associated with these deferred tax assets at December 31, 2021 and 2020. The Company accounts for uncertainty in income taxes recognized in the 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 financial statements. The amount of the benefit that may be recognized is the largest amount that has a greater than 50% likelihood of being realized upon ultimate settlement. The provision for income taxes includes the effects of any resulting tax reserves, or unrecognized tax benefits, that are considered appropriate as well as the related net interest and penalties. Net Loss Per Share The Company calculates net loss per share in accordance with FASB ASC 260, Earnings per Share. Segment Reporting Operating segments are defined as components of an enterprise about which separate discrete information is available for evaluation by the chief operating decision maker, or decision-making group, in deciding how to allocate resources and in assessing performance. The Company views its operations and manages its business in one operating segment. Comprehensive Loss Comprehensive loss is defined as the change in equity of a business enterprise during a period from transactions, and other events and circumstances from non-owner sources and consists of net loss and changes in unrealized gains and losses on investments. Reclassifications Certain reclassifications have been made to the prior year financial statements in order to conform to the current year’s presentation. Recent Accounting Pronouncements In June 2016, the FASB issued ASU 2016-13, Financial Instruments – Credit Losses (Topic 362): Measurement of Credit Losses on Financial Statements (“ASU 2016-13”). The new standard requires that expected credit losses relating to financial assets measured on an amortized cost basis and available-for-sale debt securities be recorded through an allowance for credit losses. It also 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 also requires the reversal of previously recognized credit losses if fair value increases. The targeted transition relief standard allows filers an option to irrevocably elect the fair value option of ASC 825-10, Financial Instruments-Overall, applied on an instrument-by-instrument basis for eligible instruments. The new standard was effective beginning January 1, 2021. The adoption of ASU 2016-13, and related updates, did not have a material impact on the Company’s consolidated financial position and results of operations. There were no other recent accounting pronouncements that impacted the Company or are expected to have a significant effect on the consolidated financial statements. |
Fair Value of Financial Instrum
Fair Value of Financial Instruments | 12 Months Ended |
Dec. 31, 2021 | |
Fair Value of Financial Instruments [Abstract] | |
Fair Value of Financial Instruments | 4. Fair Value of Financial Instruments Items measured at fair value on a recurring basis are the Company’s investments. The following table sets forth the Company’s financial instruments that were measured at fair value on a recurring basis by level within the fair value hierarchy: Fair Value Measurements as of December 31, 2021 Using: Level 1 Level 2 Level 3 Total Assets: Cash equivalents: Money market mutual funds $ 179,900 $ - $ - $ 179,900 179,900 - - 179,900 Investments: United States Treasury securities 44,045 - - 44,045 Corporate Bonds - 96,696 - 96,696 Municipal Bonds - 6,000 - 6,000 Agency Bonds - 9,305 - 9,305 44,045 112,001 - 156,046 $ 223,945 $ 112,001 $ - $ 335,946 Fair Value Measurements as of December 31, 2020 Using: Level 1 Level 2 Level 3 Total Assets: Cash equivalents: Money market mutual funds $ 193,312 $ - $ - $ 193,312 United States Treasury securities 62,497 - - 62,497 Corporate Bonds - 501 - 501 Agency Bonds - 8,015 - 8,015 255,809 8,516 - 264,325 Investments: United States Treasury securities 112,328 - - 112,328 Corporate Bonds - 63,710 - 63,710 Municipal Bonds - 6,000 - 6,000 Agency Bonds - 3,583 - 3,583 112,328 73,293 - 185,621 $ 368,137 $ 81,809 $ - $ 449,946 The Company classifies its money market mutual funds and U.S. Treasury securities as Level 1 assets under the fair value hierarchy, as these assets have been valued using quoted market prices in active markets without any valuation adjustment. The Company classifies its Corporate, Municipal and Agency Bonds as Level 2 assets as these assets are not traded in an active market and have been valued through a third-party pricing service based on quoted prices for similar assets. |
Property and Equipment, Net
Property and Equipment, Net | 12 Months Ended |
Dec. 31, 2021 | |
Property and Equipment, Net [Abstract] | |
Property and Equipment, Net | 5. Property and Equipment, Net The Company’s property and equipment consisted of the following: December 31, 2021 December 31, 2020 Laboratory equipment $ 12,600 $ 7,807 Machinery and equipment 10,432 9,933 Computer equipment 218 218 Furniture and fixtures 1,963 1,880 Leasehold improvements 407 29 Internal use software 1,902 1,385 27,522 21,252 Less: accumulated depreciation and amortization (5,223 ) (2,046 ) $ 22,299 $ 19,206 Depreciation and amortization during the years ended December 31, 2021, 2020, and 2019 was $ million, $1.1 million and $ million, respectively. |
Accounts Payable and Accrued Ex
Accounts Payable and Accrued Expenses | 12 Months Ended |
Dec. 31, 2021 | |
Accounts Payable and Accrued Expenses [Abstract] | |
Accounts Payable and Accrued Expenses | 6. Accounts Payable and Accrued Expenses At December 31, 2021 and 2020, the Company’s accounts payable and accrued expenses consisted of the following: December 31, 2021 December 31, 2020 Research and development $ 12,082 $ 14,962 Property and equipment 725 1,756 Employee compensation 4,533 4,875 Accrued interest - 1,122 Government grant payable 597 590 Professional fees 1,196 1,332 Other 482 835 $ 19,615 $ 25,472 |
Convertible Notes Payable
Convertible Notes Payable | 12 Months Ended |
Dec. 31, 2021 | |
Convertible Notes Payable [Abstract] | |
Convertible Notes Payable | 7. Convertible Notes Payable 2021 Convertible Notes On January 4, 2018, in connection with its reverse merger with Inotek Pharmaceuticals, Corporation (“Inotek”), the Company assumed the obligations of Inotek under its outstanding convertible notes, with an aggregate original principal amount of $52.0 million, (the “2021 Convertible Notes”). The 2021 Convertible Notes were issued in 2016 and were initially scheduled to mature on August 1, 2021 (the “Maturity Date”). The 2021 Convertible Notes were unsecured and accrued interest at a rate of 5.75% per annum and interest was payable semi-annually on February 1 and August 1 of each year. Each holder of the 2021 Convertible Notes (“Holder”) had the option until the close of business on the second business day immediately preceding the Maturity Date to convert all, or any portion, of the 2021 Convertible Notes held by it at a conversion rate of 31.1876 shares of the Company’s common stock per $1.00 principal amount of 2021 Convertible Notes (the “Conversion Rate”) which is $32.08 per share. On August 2, 2021, holders of the 2021 Convertible Notes converted the $5.15 million remaining balance of the 2021 Convertible Notes into 160,614 shares of the Company’s common. As of December 31, 2021, none of the 2021 Convertible Notes were outstanding. 2022 Convertible Notes On February 20, 2020, and June 5, 2020, the Company entered into separate, privately negotiated exchange agreements (the “Exchange Agreements”) with certain holders of the 2021 Convertible Notes. Pursuant to the Exchange Agreements, on February 20, 2020, the Company exchanged approximately $39.35 million aggregate principal amount of the 2021 Convertible Notes for (a) approximately $39.35 million aggregate principal amount of 6.25% Convertible Senior Notes due August 2022 The conversion rate for the 2022 Convertible Notes was initially 31.1876 shares of the Company’s common stock per $1.00 principal amount of 2022 Convertible Notes, which was equivalent to an initial conversion price of approximately $32.06 per share of common stock. The Company may have redeemed for cash all or any portion of the 2022 Convertible Notes, at its option, if the last reported sale price of its common stock was equal to or greater than 130% of the conversion price then in effect for at least 20 trading days during any 30 consecutive trading day period. In December 2020, $8.5 million principal amount, representing a carrying value of $7.6 million of the 2022 Convertible Notes was converted into 298,562 shares of the Company’s common stock. On April 26, 2021, the Company called for the redemption of the remaining $38.4 million principal amount of the 2022 Convertible Notes as the Company’s stock price traded above the Conversion Rate for at least 20 trading days during a 30-day consecutive trading period. On April 26, 2021, the Company redeemed in full the 2022 Convertible Notes prior to the redemption date. Holders of approximately $38.4 million remaining principal amount of the 2022 Convertible Notes converted such notes in accordance with the terms of the Exchange Agreements into approximately 1.3 million shares of the Company’s common stock and cash in lieu of fractional shares. In accordance with ASC 470- Debt , the settlement of the 2022 Convertible Notes is accounted for as a conversion since the 2022 Convertible Notes did not include a beneficial conversion feature and the carrying amount of the 2022 Convertible Notes. The table below summarizes the carrying value of the 2021 and 2022 Convertible Notes as of December 31, 2021 and 2020: 2021 Notes 2022 Notes December 31, 2021 December 31, 2020 December 31, 2021 December 31, 2020 Principal amount $ - $ 5,150 $ - $ 38,350 Discount - (275 ) - (3,284 ) Carrying value $ - $ 4,875 $ - $ 35,066 Accretion of the 2021 Convertible Notes discount was $0.3 million, $1.3 million, and $3.6 million for the years ended December 31, 2021, 2020, and 2019, respectively. Accretion of the 2022 Convertible Notes discount was $0.5 million and $1.5 million for the years ended December 31, 2021 and December 31, 2020. |
Shareholders' Equity
Shareholders' Equity | 12 Months Ended |
Dec. 31, 2021 | |
Stockholders' Equity [Abstract] | |
Shareholders' Equity | 8. Stockholders’ Equity Common Stock The Company is currently authorized to issue up to shares of $ par value common stock. All issued shares of common stock are entitled to vote on a 1 share/ vote basis. Second Amended and Restated 2014 Stock Option and Incentive Plan In March 2018, Rocket’s Board of Directors approved the Second Amended and Restated 2014 Stock Option and Incentive Plan (the “Revised 2014 Plan”) which was approved by the Company’s shareholders at the Annual Meeting held on June 25, 2018. Treasury Stock During fiscal 2019, the Company recorded treasury stock of $ million for shares withheld to pay the payroll tax liability of an option exercise . There was no treasury stock as of December 31, 2021 and 2020. Private Placement On August 27, 2021, the Company entered into a Securities Purchase Agreement (the “Purchase Agreement”) with a fund affiliated with RTW Investments, LP, the Company’s largest shareholder (the “Purchaser”), pursuant to which the Company agreed to sell and issue to the Purchaser, in a private placement (the “Private Placement”), 812,516 shares of the Company’s common stock at a purchase price of $32.48 per share for aggregate net proceeds of approximately $26.4 million after deducting estimated offering expenses payable. The Private Placement closed on August 31, 2021. Public Offerings On December 14, 2020, the Company completed a public offering of shares of common stock, which includes the full exercise of the underwriters’ option to purchase an additional shares of our common stock, at a public offering price of $ per share. The gross proceeds to Rocket from the public offering were approximately $ million, net of $ million of offering costs, commissions, legal and other expenses for net proceeds from the offering of $ |
Stock-Based Awards
Stock-Based Awards | 12 Months Ended |
Dec. 31, 2021 | |
Stock-Based Awards [Abstract] | |
Stock-Based Awards | 9. Stock-Based Awards Stock Option Valuation The weighted average assumptions that the Company used in the Black-Scholes pricing model to determine the fair value of the stock options granted to employees, non-employees and directors were as follows: Years Ended December 31, 2021 2020 2019 Risk-free interest rate 0.83 % 1.00 % 2.26 % Expected term (in years) 5.84 5.84 5.81 Expected volatility 69.27 % 76.98 % 75.71 % Expected dividend yield 0.00 % 0.00 % 0.00 % Exercise price $ 51.20 $ 22.96 $ 14.47 Fair value of common stock $ 51.20 $ 22.96 $ 14.47 The following table summarizes stock option activity for the years ended December 31, 2021 and 2020, under the Revised 2014 Plan: Number of Shares Weighted Average Exercise Price Weighted Average Contractual Term (Years) Aggregate Intrinsic Value Outstanding as of December 31, 2019 9,763,541 $ 5.95 6.96 $ 164,021 Granted 2,193,546 22.96 8.97 Exercised (586,857 ) 4.82 13,494 Cancelled (319,299 ) 15.82 Outstanding as of December 31, 2020 11,050,931 $ 9.10 6.55 $ 504,079 Granted 1,671,759 51.20 8.58 Exercised (1,209,960 ) 9.32 54,487 Cancelled (368,969 ) 35.87 Outstanding as of December 31, 2021 11,143,761 $ 14.51 5.95 $ 128,817 Options vested and exercisable as of December 31, 2021 8,692,898 $ 7.31 5.11 $ 127,571 Options unvested as of December 31, 2021 2,450,863 $ 40.01 8.93 The weighted average grant-date fair value per share of stock options granted during the years ended December 31, 2021, 2020 and 2019 was $31.07, $15.10, and $ , The total fair value of options vested during the years ended December 31, 2021, 2020 and 2019 was $22.6 million, $15.6 million and $63.1 million, respectively. Stock-Based Compensation Stock-based compensation expense recognized by award type is as follows: Years Ended December 31, 2021 2020 2019 Stock options $ 28,811 $ 18,527 $ 13,371 Restricted stock units 426 40 - Total share based compensation expense $ 29,237 $ 18,567 $ 13,371 Stock-based compensation expense by classification included within the consolidated statements of operations and comprehensive loss was as follows: Years Ended December 31, 2021 2020 2019 Research and development $ 11,954 $ 7,355 $ 6,153 General and administrative 17,283 11,212 7,218 Total share based compensation expense $ 29,237 $ 18,567 $ 13,371 As of December 31, 2021 and 2020, the Company had an aggregate of $46.3 million and $30.4 million of unrecognized stock-based compensation expense, which is expected to be recognized over the weighted average period of 1.8 year s and 2.1 years, respectively. Restricted Stock The following table summarizes our RSU activity for the years ended December 31, 2021 and 2020: Number of Shares Weighted Average Grant Date Fair Value Unvested as of December 31, 2019 - $ - Granted 20,000 25.06 Exercised - Forfeited - Unvested as of December 31, 2020 20,000 25.06 Granted 3,500 62.32 Exercised - Forfeited - Unvested as of December 31, 2021 23,500 30.61 The intrinsic vested during the years ended December 31, 2021 and 2020 was $ million and $ million. As of December 31, 2021, there was approximately $ million of unrecognized compensation cost granted. This amount is expected to be recognized over a weighted average period of Warrants A summary of the warrants outstanding at December 31, 2021 is as follows: Price Outstanding Grant Date Expiration Date 24.42 7,051 June 28, 2013 June 28, 2023 57.11 603,386 December 21, 2020 December 21, 2030 33.63 301,291 August 9, 2021 August 9, 2031 22.51 153,155 December 17, 2021 December 17, 2031 22.51 153,155 December 17, 2021 December 17, 2031 Total 1,218,038 The following table below is the summary of changes in warrants to purchase common stock for the years ended December 31, 2021 and 2020: Number of Warrant Shares Outstanding Exercise Price per Share Balance as of December 31, 2019 14,102 $ 24.42 Granted 603,386 $ 57.11 Exercised (7,051 ) $ - Balance as of December 31, 2020 610,437 Granted August 2021 301,291 $ 33.63 Granted December 2021 306,310 $ 22.51 Exercised - $ - Balance as of December 31, 2021 1,218,038 The Company issued warrants to a related party during the years ended December 31, 2021 and 2020 and incurred a non-cash R&D expense of $12.8 million and $26.6 million respectively (see Note 15- Related Party Transactions). During the year ended December 31, 2020, 7,051 of warrants were exercised into 1,601 shares of common stock. The fair value of the 2021 and 2020 warrants was calculated using the Black-Scholes fair value pricing model with the following inputs: Years Ended December 31, 2021 2020 Risk-free interest rate 1.37 % 0.95 % Expected term (in years) 10.00 10.00 Expected volatility 70.25 % 74.20 % Expected dividend yield 0.00 % 0.00 % Exercise price $ 28.07 $ 57.11 Fair value of common stock $ 28.07 $ 57.11 |
Net Loss Per Share
Net Loss Per Share | 12 Months Ended |
Dec. 31, 2021 | |
Net Loss Per Share [Abstract] | |
Net Loss Per Share | 10. Net Loss Per Share Basic and diluted net loss per share attributable to common stockholders was calculated as follows: For the Years Ended December 31, 2021 2020 2019 Numerator: Net loss attributable to common stockholders $ (169,069 ) $ (139,700 ) $ (77,270 ) Denominator: Weighted-average common shares outstanding - basic and diluted 63,235,417 55,380,740 49,010,358 Net loss per share attributable to common stockholders - basic and diluted $ (2.67 ) $ (2.52 ) $ (1.58 ) The Company excluded the following potential shares of common stock, presented based on amounts outstanding at each period end, from the computation of diluted net loss per share attributable to common stockholders for the periods indicated because including them would have had an anti-dilutive effect: For the Years Ended December 31, 2021 2020 2019 Shares issuable upon conversion of the 2021 Convertible Notes - 160,536 1,620,948 Shares issuable upon conversion of the 2022 Convertible Notes - 1,195,449 - Warrants exercisable for common shares 1,218,038 610,437 14,102 Restricted stock units exercisable for common shares 23,500 - - Options to purchase common shares 11,143,761 11,050,931 9,608,537 12,385,299 13,017,353 11,243,587 |
Income Taxes
Income Taxes | 12 Months Ended |
Dec. 31, 2021 | |
Income Taxes [Abstract] | |
Income Taxes | 11. Income Taxes No provision for federal or state income taxes was recorded during the years ended December 31, 2021, 2020 and 2019, as the Company incurred operating losses and maintains a full valuation allowance against its net deferred tax assets. A reconciliation of income tax benefit computed at the statutory federal income tax rate to income taxes as reflected in the financial statements is as follows: For the years ended December 31, 2021 2020 2019 U.S. federal tax at statutory rate 21.0 % 21.0 % 21.0 % Foreign rate differential (13.0 )% 0.0 % 0.0 % Change in state tax apportionment 0.1 % 0.1 % (17.4 )% Stock compensation 1.5 % 0.9 % (0.7 )% Transfer pricing adjustments (22.8 )% 0.0 % 0.0 % Valuation allowance 4.6 % (35.0 )% (5.1 )% Tax credits 8.7 % 13.5 % 0.0 % Other (0.2 )% (0.5 )% 2.1 % Effective tax rate 0 % 0 % 0 % The significant components of the Company’s deferred income tax assets and liabilities after applying the enacted corporate tax rates are as follows: At December 31, 2021 2020 2019 Deferred income tax assets (liabilities) R&D Credits $ 35,766 $ 42,613 $ 18,471 Net operating losses ("NOL") and credit carryforwards 26,789 21,359 2,517 Capitalized research and development costs 19,753 23,179 27,652 Stock-based compensation 11,552 7,406 3,896 Debt discount - (748 ) (1,460 ) Warrants 8,382 5,585 - Other (8,881 ) 1,189 643 Valuation allowance (93,361 ) (100,583 ) (51,719 ) Net deferred income tax asset $ - $ - $ - As of December 31, 2021, the Company had federal and state net operating loss carryforwards of approximately $118.2 million and $30.9 million, respectively, which begin to expire in 2026 2038 As required by ASC 740, Income Taxes Under Internal Revenue Code Section 382, if a corporation undergoes an “ownership change,” the corporation’s ability to use its pre-change NOL carryforwards and other pre-change tax attributes to offset its post-change income may be limited. The Company has completed a study to assess whether an ownership change has occurred or whether there have been multiple ownership changes since the Company became a “loss corporation” as defined in Section 382. The Company experienced multiple ownership changes occurring in 2005, 2007, 2015, and 2018. The ownership change has and will continue to subject our pre-ownership change net operating loss carryforwards to an annual limitation, which will significantly restrict our ability to use them to offset taxable income in periods following the ownership change. In general, the annual use limitation equals the aggregate value of our stock at the time of the ownership change multiplied by a specified tax-exempt interest rate. As a result of the ownership change, the Company is limited to an approximate $ million annual limitation on our ability to utilize our pre-merger NOL’s and R&D Credits. Due to this limitation, approximately $ million of the $ million pre-merger Federal NOL will expire unutilized as the cumulative limitation amount over a carryforward period is $ million. Additionally, $ million of Federal R&D Credits will expire unutilized. As a result, the Company has reduced its deferred tax assets related to the Federal NOL and Federal R&D Credits by an aggregate of $ million which is offset by the corresponding decrease in the valuation allowance. The company evaluated intercompany transfer pricing agreements. Based on a review of the 2018, 2019 and 2020 tax years, the Company determined that a markup of 10% should have been applied to R&D expenses paid for on behalf of Rocket Pharmaceuticals, Ltd by Rocket Pharmaceuticals, Inc. The net impact was to reduce the Company’s net operating losses for 2018, 2019 and 2020. No income tax expense was recorded as a result of these adjustments. The calculation of our tax liabilities involves dealing with uncertainties in the application of complex tax laws and regulations for both federal taxes and the many states in which Rocket operates or does business in. ASC 740 states that a tax benefit from an uncertain tax position may be recognized when it is more likely than not that the position will be sustained upon examination, including resolutions of any related appeals or litigation processes, on the basis of the technical merits. The Company records uncertain tax positions as liabilities in accordance with ASC 740 and adjusts these liabilities when our judgment changes as a result of the evaluation of new information not previously available. Because of the complexity of some of these uncertainties, the ultimate resolution may result in a payment that is materially different from our current estimate of the unrecognized tax benefit liabilities. These differences will be reflected as increases or decreases to income tax expense in the period in which new information is available. As of December 31, 2021, 2020 and 2019, the Company has not recorded any uncertain tax positions in its financial statements. The Company recognizes interest and penalties related to unrecognized tax benefits on the income tax expense line in the accompanying consolidated statement of operations. As of December 31, 2021, 2020 and 2019, no accrued interest or penalties are included on the related tax liability line in the consolidated balance sheets. The Company was audited by New York City for the 2017 tax year and the audit was completed with no change to the 2017 New York City tax return. |
Commitments and Contingencies
Commitments and Contingencies | 12 Months Ended |
Dec. 31, 2021 | |
Commitments and Contingencies [Abstract] | |
Commitments and Contingencies | 12. Commitments and Contingencies The Finance Lease The Company has a lease for a facility in Cranbury, New Jersey, consisting of 103,720 square feet of space including areas for offices, process development, research and development laboratories and 50,000 square feet dedicated to AAV Current Good Manufacturing Practice (“cGMP”) manufacturing facilities to support the Company’s pipeline. A smaller area within this facility was originally leased in August 2018, and the lease was amended in June 2019 to include the full building (such lease, as amended, the “NJ Lease Agreement”). The NJ Lease Agreement has a 15-year term from September 1, 2019, with an option to renew for two consecutive five-year renewal terms. The Company determined the lease commencement date was reached on March 15, 2020, when the construction of all landlord owned improvements had been substantially completed and when the Company began including its leasehold improvements on the balance sheet and move equipment into the space. Upon commencement of the NJ Lease Agreement, the Company recognized total right-of-use assets of $47.7 million, with a corresponding lease liability of $20.2 million. The Company reclassified $26.5 million of construction costs in progress and $1.1 million of prepaid rent as part of the right-of-use asset upon the lease commencement date of March 15, 2020. During the year ended December 31, 2021, the Company reclassified an additional $0.1 million of construction costs in progress bringing the aggregate reclassification through December 31, 2021, of $32.1 million of construction costs in progress as part of the right of use asset. Estimated rent payments for the NJ Lease Agreement are $1.2 million per annum, payable in monthly installments, depending upon the nature of the leased space, and subject to annual base rent increases of 3%. The total commitment under the lease is estimated to be approximately $29.3 million over the 15-year term of the lease. The Company paid a cash security deposit of $0.3 million to the landlord in connection with the NJ Lease Agreement which has been reflected in deposits in the consolidated balance sheets as of December 31, 2021 and 2020. The total restricted cash balance for the Company’s operating and finance leases at December 31, 2021 and 2020 was $0.8 million and $1.1 million, respectively. Operating Leases On March 31, 2016 the Company entered into a lease agreement for its office and laboratory space at the Alexandria Center for Life Sciences in New York, New York with an initial term ending on (the “NY Lease Agreement”). Effective May 31, 2020 the Company terminated the NY Lease Agreement and recorded a loss on lease termination of for the year ended December 31, 2020 On June 7, 2018, the Company entered into a three-year lease agreement for office space in the Empire State Building in New York, NY (the “ESB Lease Agreement”). In connection with the ESB Lease Agreement, the Company established an irrevocable standby letter of credit (the “Empire LOC”) for $0.9 million. On March 26, 2021, the Company entered in Amendment No. 1 to the ESB Lease Agreement (“ESB Lease Amendment”) that extended the term of the lease agreement to June 30, 2024, reduced the rent payments going forward, and reduced the Empire LOC to $0.8 million. The Empire LOC serves as the Company’s security deposit on the lease in which the landlord is the beneficiary and expires August 29, 2024. The Company has accounted for the ESB Lease Amendment as a modification to the ESB Lease Agreement and remeasured the lease liability and adjusted the operating lease right of use asset by $1.1 million. The Company has a certificate of deposit of $0.8 million and $0.9 million with a bank as collateral for the Empire LOC which is classified as part of restricted cash in the consolidated balance sheets as of December 31, 2021 and December 31, 2020, respectively. On January 4, 2018, in connection with the Reverse Merger, the Company assumed an operating lease for Inotek’s former headquarters in Lexington, Massachusetts, with a term ending in February 2023 Rent expense was $1.2 million, $1.1 million, and $0.9 million for the years ended December 31, 2021, 2020 and 2019, respectively. Lease cost December 31, 2021 Operating lease cost $ 645 Finance lease cost Amortization of right of use assets 2,140 Interest on lease liablities 1,845 Total lease cost $ 4,630 The following table summarizes the maturity of the Company’s lease liabilities on an undiscounted cash flow basis and a reconciliation to the operating lease liabilities recognized on the Company’s balance sheet as of December 31, 2021: Maturity of operating lease liabilities December 31, 2021 2022 917 2023 548 2024 269 2025 64 2026 54 Total lease payments $ 1,852 Less: interest (84 ) Total operating lease liabilities $ 1,768 Maturity of finance lease liability December 31, 2021 2022 1,689 2023 1,736 2024 1,791 2025 1,856 2026 1,912 Thereafter 45,000 Total lease payments $ 53,984 Less: interest (33,151 ) Total finance lease liability $ 20,833 Leases December 31, 2021 Operating right-of-use assets $ 1,569 Operating current lease liabilities 863 Operating noncurrent lease liabilities 905 Total operating lease liabilities $ 1,768 Finance right-of-use assets $ 48,480 Finance current lease liability 1,689 Finance noncurrent lease liability 19,144 Total finance lease liability $ 20,833 Other information Cash paid for amounts included in the measurement of lease liabilities: Operating cash flows from operating leases $ 655 Cash flows from finance lease $ 1,644 Weighted-average remaining lease term - operating leases 2.5 years Weighted-average remaining lease term - finance lease 22.7 years Weighted-average discount rate - operating leases 4.46 % Weighted-average discount rate - finance lease 8.96 % Litigation From time to time, the Company may be subject to various legal proceedings and claims that arise in the ordinary course of its business activities. Although the results of litigation and claims cannot be predicted with certainty, the Company does not believe it is party to any other claim or litigation the outcome of which, if determined adversely to the Company, would individually or in the aggregate be reasonably expected to have a material adverse effect on its business. Regardless of the outcome, litigation can have an adverse impact on the Company because of defense and settlement costs, diversion of management resources and other factors. Indemnification Arrangements Pursuant to its bylaws and as permitted under Delaware law, the Company has indemnification obligations to directors, officers, employees or agents of the Company or anyone serving in these capacities. The maximum potential amount of future payments the Company could be required to pay is unlimited. The Company has insurance that reduces its monetary exposure and would enable it to recover a portion of any future amounts paid. As a result, the Company believes that the estimated fair value of these indemnification commitments is minimal. Throughout the normal course of business, the Company has agreements with vendors that provide goods and services required by the Company to run its business. In some instances, vendor agreements include language that requires the Company to indemnify the vendor from certain damages caused by the Company’s use of the vendor’s goods and/or services. The Company has insurance that would allow it to recover a portion of any future amounts that could arise from these indemnifications. As a result, the Company believes that the estimated fair value of these indemnification commitments is minimal. |
Agreements Related to Intellect
Agreements Related to Intellectual Property | 12 Months Ended |
Dec. 31, 2021 | |
Agreements Related to Intellectual Property [Abstract] | |
Agreements Related to Intellectual Property | 13. Agreements Related to Intellectual Property The Company, directly and through its subsidiary Spacecraft , LLC, has various license and research and collaboration arrangements. The transactions principally resulted in the acquisition of rights to intellectual property which is in the preclinical phase and has not been tested for safety or feasibility. In all cases, the Company did not acquire tangible assets, processes, protocols or operating systems. The Company expenses the acquired intellectual property rights as of the acquisition date on the basis that the cost of intangible assets purchased from others for use in research and development activities, has no alternative future uses. License Agreement for Danon Disease with UCSD In February 2017, the Company entered into a License Agreement with The Regents of the University of California, represented by its San Diego campus (“UCSD”), under which UCSD granted us an exclusive, sublicensable, worldwide license to certain intellectual property rights for the treatment of lysosomal storage diseases, including Danon disease. In exchange for the license, the Company became obligated to make an up-front payment, certain clinical and commercial milestone payments, royalty payments (on net sales of products covered by a valid claim within the licensed intellectual property), maintenance fees and sublicense revenue payments. The upfront license fee of $0.05 million was expensed as research and development costs. The Company is obligated to make aggregate milestone payments of up to $1.5 million to UCSD upon the achievement of specified development and regulatory milestones for the treatment of Danon disease. A reduced schedule of milestone payments applies to achieving the same milestones for additional indications. With respect to any commercialized products covered by the agreement, the Company is obligated to pay a low single digit percentage royalty on net sales, subject to specified adjustments. If it enters into a sublicense agreement with a sublicensee, it will be obligated to pay a portion of any consideration received from such sublicensees in specified circumstances. The Company is also subject to certain diligence milestones for development of a product using the intellectual property licensed from UCSD under this agreement. The term of the license agreement with UCSD is through the expiration of the licensed patents, some of which are still in the pending application phase. REGENXBIO, Inc. License On November 19, 2018, the Company entered into a license agreement with REGENXBIO Inc. (“RGNX”), pursuant to which the Company obtained an exclusive license for all U.S. patents and patent applications related to RGNX’s NAV AAV-9 vector for the treatment of Danon disease in humans by in vivo Under the terms of the license agreement, the Company is obligated to use commercially reasonable efforts to develop, commercialize, market, promote and sell products incorporating the Licensed Patents (“Licensed Products”). Unless the license agreement is terminated earlier as provided below, the license from RGNX expires on a country-by-country, Licensed Product-by-Licensed Product basis until the later of the expiration date of the last to expire of the last valid claim of the applicable Licensed Patent or ten years after the first commercial sale of a Licensed Product in such country. The license agreement provides that RGNX may terminate the agreement upon a material breach by the Company if the Company does not cure such breach within a specified notice period if the Company commences a challenge against RGNX or certain of its licensors to declare or render invalid or unenforceable the licensed patents or upon the Company’s bankruptcy or insolvency. The Company may terminate the agreement in its entirety or terminate one or more of the licensed vectors at any time upon six months’ notice. The Company’s Option Right expires four years from the date of the license agreement. In consideration for the rights granted to the Company under the license agreement, the Company made an upfront payment to RGNX of $ million included as research and development expenses. A fee of $ million per additional vector would be due if the Company exercises its Option Right to purchase additional vectors. The license agreement provides for royalties payable to RGNX in the high-single digits to low-teens on net sales levels of Licensed Products during the royalty term. If successful, the Company will be required to make milestone payments to RGNX of up to $ million for each Licensed Product upon the achievement of specified clinical development and regulatory milestones in the U.S. and European Union. In addition, the Company shall pay RGNX of the payment fees received from a priority review voucher issued in connection with or otherwise related to a Licensed Product. These royalty obligations are subject to specified reductions if additional licenses from third parties are required. The Company must also pay RGNX a portion of all non-royalty sublicense income (if any) received from sublicensees. The Company paid a $ million license fee payment under the RGNX agreement upon the dosing of the first Danon patient in 2019. There were additional milestones achieved or related payments made during the years ended December |
CIRM Grants
CIRM Grants | 12 Months Ended |
Dec. 31, 2021 | |
CIRM Grant [Abstract] | |
CIRM Grants | 14. CIRM Grants LAD-I CIRM Grant On April 30, 2019, the California Institute for Regenerative Medicine (“CIRM”) awarded the Company up to $6.5 million under a CLIN2 grant award to support the clinical development of its LVV-based gene therapy for RP-L201. Proceeds from the grant will help fund clinical trial costs as well as manufactured drug product for Phase 1/2 patients enrolled at the U.S. clinical site, University of California, Los Angeles (“UCLA”) Mattel Children’s Hospital, led by principal investigator Donald Kohn, M.D., UCLA Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA. In 2019, the Company received the first two grants from CIRM in the aggregate of $1.2 million which were included as an offset against R&D expenses. In 2020, the Company met additional CIRM milestones and received an additional $1.1 million milestone which was recorded as a reduction of R&D expenses in 2020. The Company received the additional milestone payments of $1.1 million and $1.0 million in January and April of 2021, respectively. As of December 31, 2021, the Company did not meet the next milestone and therefore no receivable has been recorded. IMO CIRM Grant On November 12, 2020, the CIRM awarded the Company up to $3.7 million under a CLIN2 grant award to support the clinical development of its LVV-based gene therapy, RP-L401, for the treatment of IMO. The Company received $1.0 million pursuant to the grant on January 4, 2021, related to the CIRM IMO award and recorded a receivable, included in prepaid and other assets in the consolidated balance sheet, and a reduction of research and development expenses of $0.9 million as of December 31, 2020. |
Related Party Transactions
Related Party Transactions | 12 Months Ended |
Dec. 31, 2021 | |
Related Party Transactions [Abstract] | |
Related Party Transactions | 15. Related Party Transactions During April 2018, the Company entered into an agreement with a member of the Board of Directors for business development consulting services. Payments for the services under the agreement are $ per quarter, and the Company may terminate the agreement with days’ notice. The Company incurred expenses for each of the years ended December 31, 2021, 2020 and 2019, relating to services provided under this agreement. In September 2021, the Company entered into a consulting agreement with a member of the Board of Directors for pipeline development, new asset evaluation, and corporate strategy. In lieu of cash for services to be provided under the consulting agreement during its term, the Company granted the board member options to purchase common stock with a fair value of $ million. In October 2020, the Company entered into a consulting agreement with the spouse of one of the Company’s executive officers for information technology advisory services. In exchange for the services provided under the agreement, the Company granted restricted stock units which vest over a period. On August 27, 2021, the Company entered into a Securities Purchase Agreement (the “Purchase Agreement”) with a fund affiliated with RTW Investments, LP, the Company’s largest shareholder (the “Purchaser”), pursuant to which it agreed to sell and issue to the Purchaser, in a private placement (the “Private Placement”), 812,516 shares of the Company’s common stock at a purchase price of $32.48 per share for aggregate net proceeds of approximately $26.4 million to the Company before deducting estimated offering expenses payable by the Company. The Private Placement closed on August 31, 2021. In addition, concurrently with the execution of the Purchase Agreement, the Company entered into a registration rights agreement with the Purchaser, pursuant to which the Company agreed, following demand by the Purchaser, to file with the Securities and Exchange Commission a Registration Statement on Form S-3 covering the resale of shares of common stock held by the Purchaser as promptly as reasonably practicable following such demand, and in any event within 60 days of such demand. On December 21, 2020, the Company entered into a consulting agreement with a related party. Pursuant to the consulting agreement, the related party provides certain business development and asset identification consulting services to the Company. The term of the consulting agreement is three years and may be terminated with 60 days’ notice by either party. In exchange for the business development services to be provided under the agreement, the Company issued a warrant exercisable for 603,386 shares of Common Stock. Pursuant to the consulting agreement, the related party is entitled to receive additional warrants exercisable for common stock upon identification of new assets for the Company to in-license. The Company recorded a non-cash R&D expense of $26.6 million for the year ended December 31, 2020, related to the issuance of the Common Stock warrant. On August 9, 2021, the Company issued a warrant exercisable for 301,291 shares of common stock to the same related party for business development and asset identification consulting services ("August 2021 Warrant"). The Company recorded a non-cash R&D expense of $7.6 million during year ended December 31, 2021, related to the issuance of the August 2021 warrant. On December 17, 2021, the Company issued warrants exercisable for 153,155 and 153,155 shares of common stock, respectively to the same related party for business development and asset identification consulting services (“December 2021 Warrants”). The Company recorded a non-cash R&D expense of $5.2 million during year ended December 31, 2021, related to the issuance of the December 2021 warrant. Total non-cash R&D expense of $12.8 million during the year ended December 31, 2021, related to the issuance of the August 2021 and December 2021 warrants. |
401(k) Savings Plan
401(k) Savings Plan | 12 Months Ended |
Dec. 31, 2021 | |
401(k) Savings Plan [Abstract] | |
401(k) Savings Plan | 16. 401(k) Savings Plan The Company has a defined contribution savings plan (the “Plan”) under Section 401(k) of the Internal Revenue Code of 1986. This Plan covers substantially all employees who meet minimum age and service requirements and allows participants to defer a portion of their annual compensation on a pre-tax basis. Company contributions to the Plan may be made at the discretion of the Company’s Board of Directors. The Company has elected to match 4% of employee contributions to the Plan, subject to certain limitations. The Company’s matching contribution for the years ended December 31, 2021, 2020, and 2019 was $0.6 million, $0.3 million, and $0.2 million, respectively. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 12 Months Ended |
Dec. 31, 2021 | |
Summary of Significant Accounting Policies [Abstract] | |
Principles of Consolidation | Principles of Consolidation The consolidated financial statements represent the consolidation of the accounts of the Company and its subsidiaries in conformity with accounting principles generally accepted in the United States (“US GAAP”). All intercompany accounts have been eliminated in consolidation. |
Use of Estimates | Use of Estimates The preparation of the consolidated financial statements in conformity with US 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 goodwill impairment, the accrual of research and development expenses, the valuation of equity transactions, and stock-based awards. Changes in estimates and assumptions are reflected in reported results in the period in which they become known. Actual results could differ from those estimates. |
Cash, Cash Equivalents and Restricted Cash | Cash, Cash Equivalents and Restricted Cash Cash, cash equivalents and restricted cash consists of bank deposits, certificates of deposit and money market accounts with financial institutions. Cash equivalents are carried at cost which approximates fair value due to their short-term nature and which the Company believes do not have a material exposure to credit risk. The Company considers all highly liquid investments with maturities of three months or less from the date of purchase to be cash equivalents. The Company’s cash and cash equivalent accounts, at times, may exceed federally insured limits. The Company has not experienced any losses in such accounts. Restricted cash consists of deposits collateralizing letters of credit issued by a bank in connection with the Company’s operating leases (see Note 12 “Commitments and Contingencies” for additional disclosures) and a deposit collateralizing a letter of credit issued by a bank supporting the Company’s corporate credit card. Cash, cash equivalents and restricted cash consist of the following: December 31, 2021 December 31, 2020 Cash and cash equivalents $ 232,694 $ 297,098 Restricted cash 1,343 1,568 $ 234,037 $ 298,666 |
Government Grants | Government Grants Research and development expense is presented net of reimbursements from the California Institute for Regenerative Medicine (“CIRM”), which are recognized over the period necessary to match the reimbursement with the related costs when it is probable that the Company has complied with the CIRM conditions and will receive the reimbursement. During the years ended December 31, 2021, 2020, and 2019, the Company offset $0.1 million, $3.6 million and $1.2 million of CIRM grant funds against research and development (“R&D”) expenses (See Note 14 “CIRM Grant” for additional disclosure). |
Concentrations of Credit Risk and Off-balance Sheet Risk | Concentrations of credit risk and off-balance sheet risk Financial instruments that subject the Company to credit risk primarily consist of cash and cash equivalents and available-for-sale securities. The Company maintains its cash and cash equivalent balances with high-quality financial institutions and, consequently, the Company believes that such funds are subject to minimal credit risk. The Company’s marketable securities consist of U.S. Treasury securities, and Corporate, Government Municipal and Agency Bonds. The Company has adopted an investment policy that limits the amounts the Company may invest in any one type of investment and requires all investments held by the Company to be at least AA+/Aa1 rated, thereby reducing credit risk exposure. |
Investments | Investments Investments consist of investments in United States Treasury securities and Corporate, Municipal and Agency Bonds. Management determines the appropriate classification of these securities at the time they are acquired and evaluates the appropriateness of such classifications at each balance sheet date. The Company classifies its investments as available-for-sale pursuant to Financial Accounting Standards Board (“FASB”) Accounting Standard Codification (“ASC”) 320, Investments—Debt and Equity Securities |
Goodwill | Goodwill Business combinations are accounted for under the acquisition method. The total cost of an acquisition is allocated to the underlying identifiable net assets, based on their respective estimated fair values as of the acquisition date. Determining the fair value of assets acquired and liabilities assumed requires management’s judgment and often involves the use of significant estimates and assumptions, including assumptions with respect to future cash inflows and outflows, discount rates, asset lives and market multiples, among other items. Assets acquired and liabilities assumed are recorded at their estimated fair values. The excess of the purchase price over the estimated fair values of the net assets acquired is recorded as goodwill. Goodwill is tested for impairment annually as of December 31, or more frequently when events or changes in circumstances indicate that the asset might be impaired. Examples of such events or circumstances include, but are not limited to, a significant adverse change in legal or business climate, an adverse regulatory action or unanticipated competition. The Company has one segment and one reporting unit and as such review’s goodwill for impairment at the consolidated level. When testing goodwill, the Company has the option to first assess qualitative factors for reporting units that carry goodwill. The qualitative assessment includes assessing the totality of relevant events and circumstances that affect the fair value or carrying value of the reporting unit. These events and circumstances include macroeconomic conditions, industry and competitive environment conditions, overall financial performance, reporting unit specific events and market considerations. The Company also considers recent valuations of the reporting unit, including the magnitude of the difference between the most recent fair value estimate and the carrying value, as well as both positive and adverse events and circumstances, and the extent to which each of the events and circumstances identified may affect the comparison of a reporting unit’s fair value with its carrying value. If the qualitative assessment results in a conclusion that it is more likely than not that the fair value of a reporting unit exceeds the carrying value, then no further testing is performed for that reporting unit. The Company performed the qualitative assessment of its goodwill and determined that it is more likely than not that the fair value of a reporting unit exceeds the carrying value of the reporting unit. As a result, the Company has determined there was no goodwill impairment as of and for the years ended December 31, 2021, 2020 and 2019. |
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 the useful life of the asset. The estimated useful lives are three |
Impairment of Long-Lived Assets | Impairment of Long-Lived Assets The Company reviews long-lived assets for impairment whenever events or changes in circumstances indicate that the asset’s carrying amount may not be recoverable. The Company conducted our long-lived asset impairment analyses in accordance with ASC 360-10-15, “Impairment or Disposal of Long-Lived Assets.” ASC 360-10-15 requires us to group assets and liabilities at the lowest level for which identifiable cash flows are largely independent of the cash flows of other assets and liabilities and evaluate the asset group against the sum of the undiscounted future cash flows. If the undiscounted cash flows do not indicate the carrying amount of the asset is recoverable, an impairment charge is measured as the amount by which the carrying amount of the asset group exceeds its fair value based on discounted cash flow analysis or appraisals. There is no impairment of long-lived assets as of and for the years ended December 31, 2021, 2020 and 2019. |
Fair Value Measurements | Fair Value Measurements The Company is required to disclose information on all assets and liabilities reported at fair value that enables an assessment of the inputs used in determining the reported fair values. FASB ASC 820, Fair Value Measurements and Disclosures Level 1—Valuations based on unadjusted quoted prices in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date. Level 2—Valuations based on quoted prices for similar assets or liabilities in markets that are not active or for which all significant inputs are observable, either directly or indirectly. Level 3—Valuations that require inputs that reflect the Company’s own assumptions that are both significant to the fair value measurement and unobservable. To the extent that valuation is based on models or inputs that are less observable or unobservable in the market, the determination of fair value requires more judgment. Accordingly, the degree of judgment exercised by the Company in determining fair value is greatest for instruments categorized in Level 3. A financial instrument’s level within the fair value hierarchy is based on the lowest level of any input that is significant to the fair value measurement. The fair value of the Company’s financial instruments, including cash and cash equivalents, restricted cash, deposits, accounts payable and accrued expenses approximate their respective carrying values due to the short-term nature of these instruments. |
Research and Development Expenses | Research and Development Expenses R&D costs, which include salaries and staff costs, license costs, manufacturing and development costs, clinical trial expenses, regulatory and scientific consulting fees, as well as contract research, and stock-based compensation expense, are accounted for in accordance with ASC Topic 730, Research and Development (“ASC 730”). The Company does not currently have any commercial biopharmaceutical products and does not expect to have any for several years, if at all. Accordingly, R&D costs are expensed as incurred. While certain of the Company’s R&D costs may have future benefits, the policy of expensing all R&D expenditures is predicated on the fact that the Company has no history of successful commercialization of product candidates to base any estimate of the number of future periods that would be benefited. |
Foreign Currency Transactions | Foreign Currency Transactions Certain transactions during the years ended December 31, 2021, 2020 and 2019 are denominated in Euros and British pounds. Gains and losses on foreign currency transactions were not significant for the years ended December 31, 2021, 2020 and 2019. |
Treasury Stock | Treasury Stock The Company records treasury stock at cost. |
Stock-Based Compensation | Stock-Based Compensation The Company issues stock-based awards to employees and non-employees, generally in the form of stock options and restricted stock units. The Company accounts for its stock-based compensation awards in accordance with FASB ASC Topic 718, Compensation—Stock Compensation (“ASC 718”). ASC 718 requires all stock-based payments, including grants of stock options and restricted stock units and modifications to existing stock options, to be recognized in the consolidated statements of operations and comprehensive loss based on their fair values. The Company measures the compensation expense of employee and nonemployee services received in exchange for an award of equity instruments based on the fair value of the award on the grant date. That cost is recognized on a straight-line basis over the period during which the employee and nonemployee is required to provide service in exchange for the award. The fair value of options on the date of grant is calculated using the Black-Scholes option pricing model based on key assumptions such as stock price, expected volatility and expected term. The Company’s estimates of these assumptions are primarily based on the trading price of the Company’s stock, historical data, peer company data and judgment regarding future trends and factors. The Company classifies stock-based compensation expense in its consolidated statement of operations in the same manner in which the award recipient’s payroll costs and services are classified or in which the award recipient’s service payments are classified. The Company recognizes compensation expense for at least the portion of awards that are vested. Forfeitures are accounted for as they occur. |
Tax Credits and Income Taxes | NYS Life Sciences Research and Development Tax Credit New York State allows investors and owners of emerging technology companies focused on biotechnology to claim a tax credit against their New York State Tax return for certain expenditures incurred in New York State, including applicable R&D related expenditures. The credit is recognized as research and development incentives when the eligibility and amount has been approved by New York State. During the years ended December 31, 2021, 2020 and 2019, the Company recorded research and development incentive income of $1.0 million, $0, and $0, respectively related to the NYS Life Sciences Research and Development Tax Credit. NYC Biotechnology Tax Credit Program New York City allowed investors and owners of emerging technology companies focused on biotechnology to claim a tax credit against the General Corporation Tax and Unincorporated Business Tax for amounts paid or incurred for certain facilities, operations, and employee training in New York City. The credit was recognized as research and development incentives when approved by New York City of the eligibility for the credit and the credit amount. During the years ended December 31, 2021, 2020 and 2019, the Company recorded research and development incentive income of $ , $ , and $ million, respectively related to the NYC Biotechnology Program. Income Taxes The Company accounts for income taxes under the asset and liability method. The Company recognizes deferred tax assets and liabilities for the future tax consequences attributable to differences between the financial statement carrying amounts of existing assets and liabilities and their respective tax bases, as well as for operating loss and tax credit carryforwards. The Company measures deferred tax assets and liabilities using enacted tax rates expected to apply to taxable income in the years in which the Company expects to recover or settle those temporary differences. The Company recognizes the effect of a change in tax rates on deferred tax assets and liabilities in the results of operations in the period that includes the enactment date. The Company reduces the measurement of a deferred tax asset, if necessary, by a valuation allowance if it is more likely than not that the Company will not realize some or all of the deferred tax asset. The Company’s deferred tax assets relate primarily to its net operating loss carryforwards and other balance sheet differences. In accordance with ASC 740 “Income Taxes”, the Company recorded a full valuation allowance to fully offset the net deferred tax asset because it is not more likely than not that the Company will realize future benefits associated with these deferred tax assets at December 31, 2021 and 2020. The Company accounts for uncertainty in income taxes recognized in the 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 financial statements. The amount of the benefit that may be recognized is the largest amount that has a greater than 50% likelihood of being realized upon ultimate settlement. The provision for income taxes includes the effects of any resulting tax reserves, or unrecognized tax benefits, that are considered appropriate as well as the related net interest and penalties. |
Net Loss Per Share | Net Loss Per Share The Company calculates net loss per share in accordance with FASB ASC 260, Earnings per Share. |
Segment Reporting | Segment Reporting Operating segments are defined as components of an enterprise about which separate discrete information is available for evaluation by the chief operating decision maker, or decision-making group, in deciding how to allocate resources and in assessing performance. The Company views its operations and manages its business in one operating segment. |
Comprehensive Loss | Comprehensive Loss Comprehensive loss is defined as the change in equity of a business enterprise during a period from transactions, and other events and circumstances from non-owner sources and consists of net loss and changes in unrealized gains and losses on investments. |
Reclassification | Reclassifications Certain reclassifications have been made to the prior year financial statements in order to conform to the current year’s presentation. |
Recent Accounting Pronouncements | Recent Accounting Pronouncements In June 2016, the FASB issued ASU 2016-13, Financial Instruments – Credit Losses (Topic 362): Measurement of Credit Losses on Financial Statements (“ASU 2016-13”). The new standard requires that expected credit losses relating to financial assets measured on an amortized cost basis and available-for-sale debt securities be recorded through an allowance for credit losses. It also 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 also requires the reversal of previously recognized credit losses if fair value increases. The targeted transition relief standard allows filers an option to irrevocably elect the fair value option of ASC 825-10, Financial Instruments-Overall, applied on an instrument-by-instrument basis for eligible instruments. The new standard was effective beginning January 1, 2021. The adoption of ASU 2016-13, and related updates, did not have a material impact on the Company’s consolidated financial position and results of operations. There were no other recent accounting pronouncements that impacted the Company or are expected to have a significant effect on the consolidated financial statements. |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Summary of Significant Accounting Policies [Abstract] | |
Cash, Cash Equivalents and Restricted Cash | Restricted cash consists of deposits collateralizing letters of credit issued by a bank in connection with the Company’s operating leases (see Note 12 “Commitments and Contingencies” for additional disclosures) and a deposit collateralizing a letter of credit issued by a bank supporting the Company’s corporate credit card. Cash, cash equivalents and restricted cash consist of the following: December 31, 2021 December 31, 2020 Cash and cash equivalents $ 232,694 $ 297,098 Restricted cash 1,343 1,568 $ 234,037 $ 298,666 |
Fair Value of Financial Instr_2
Fair Value of Financial Instruments (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Fair Value of Financial Instruments [Abstract] | |
Fair Value of Financial Instruments Measured on Recurring Basis | Items measured at fair value on a recurring basis are the Company’s investments. The following table sets forth the Company’s financial instruments that were measured at fair value on a recurring basis by level within the fair value hierarchy: Fair Value Measurements as of December 31, 2021 Using: Level 1 Level 2 Level 3 Total Assets: Cash equivalents: Money market mutual funds $ 179,900 $ - $ - $ 179,900 179,900 - - 179,900 Investments: United States Treasury securities 44,045 - - 44,045 Corporate Bonds - 96,696 - 96,696 Municipal Bonds - 6,000 - 6,000 Agency Bonds - 9,305 - 9,305 44,045 112,001 - 156,046 $ 223,945 $ 112,001 $ - $ 335,946 Fair Value Measurements as of December 31, 2020 Using: Level 1 Level 2 Level 3 Total Assets: Cash equivalents: Money market mutual funds $ 193,312 $ - $ - $ 193,312 United States Treasury securities 62,497 - - 62,497 Corporate Bonds - 501 - 501 Agency Bonds - 8,015 - 8,015 255,809 8,516 - 264,325 Investments: United States Treasury securities 112,328 - - 112,328 Corporate Bonds - 63,710 - 63,710 Municipal Bonds - 6,000 - 6,000 Agency Bonds - 3,583 - 3,583 112,328 73,293 - 185,621 $ 368,137 $ 81,809 $ - $ 449,946 |
Property and Equipment, Net (Ta
Property and Equipment, Net (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Property and Equipment, Net [Abstract] | |
Property and Equipment | The Company’s property and equipment consisted of the following: December 31, 2021 December 31, 2020 Laboratory equipment $ 12,600 $ 7,807 Machinery and equipment 10,432 9,933 Computer equipment 218 218 Furniture and fixtures 1,963 1,880 Leasehold improvements 407 29 Internal use software 1,902 1,385 27,522 21,252 Less: accumulated depreciation and amortization (5,223 ) (2,046 ) $ 22,299 $ 19,206 |
Accounts Payable and Accrued _2
Accounts Payable and Accrued Expenses (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Accounts Payable and Accrued Expenses [Abstract] | |
Accounts Payable and Accrued Expenses | At December 31, 2021 and 2020, the Company’s accounts payable and accrued expenses consisted of the following: December 31, 2021 December 31, 2020 Research and development $ 12,082 $ 14,962 Property and equipment 725 1,756 Employee compensation 4,533 4,875 Accrued interest - 1,122 Government grant payable 597 590 Professional fees 1,196 1,332 Other 482 835 $ 19,615 $ 25,472 |
Convertible Notes Payable (Tabl
Convertible Notes Payable (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Convertible Notes Payable [Abstract] | |
Carrying Value of Convertible Notes | The table below summarizes the carrying value of the 2021 and 2022 Convertible Notes as of December 31, 2021 and 2020: 2021 Notes 2022 Notes December 31, 2021 December 31, 2020 December 31, 2021 December 31, 2020 Principal amount $ - $ 5,150 $ - $ 38,350 Discount - (275 ) - (3,284 ) Carrying value $ - $ 4,875 $ - $ 35,066 |
Stock-Based Awards (Tables)
Stock-Based Awards (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Stock-Based Awards [Abstract] | |
Weighted-Average Assumptions for Stock Options | The weighted average assumptions that the Company used in the Black-Scholes pricing model to determine the fair value of the stock options granted to employees, non-employees and directors were as follows: Years Ended December 31, 2021 2020 2019 Risk-free interest rate 0.83 % 1.00 % 2.26 % Expected term (in years) 5.84 5.84 5.81 Expected volatility 69.27 % 76.98 % 75.71 % Expected dividend yield 0.00 % 0.00 % 0.00 % Exercise price $ 51.20 $ 22.96 $ 14.47 Fair value of common stock $ 51.20 $ 22.96 $ 14.47 |
Stock Option Activity | The following table summarizes stock option activity for the years ended December 31, 2021 and 2020, under the Revised 2014 Plan: Number of Shares Weighted Average Exercise Price Weighted Average Contractual Term (Years) Aggregate Intrinsic Value Outstanding as of December 31, 2019 9,763,541 $ 5.95 6.96 $ 164,021 Granted 2,193,546 22.96 8.97 Exercised (586,857 ) 4.82 13,494 Cancelled (319,299 ) 15.82 Outstanding as of December 31, 2020 11,050,931 $ 9.10 6.55 $ 504,079 Granted 1,671,759 51.20 8.58 Exercised (1,209,960 ) 9.32 54,487 Cancelled (368,969 ) 35.87 Outstanding as of December 31, 2021 11,143,761 $ 14.51 5.95 $ 128,817 Options vested and exercisable as of December 31, 2021 8,692,898 $ 7.31 5.11 $ 127,571 Options unvested as of December 31, 2021 2,450,863 $ 40.01 8.93 |
Stock-Based Compensation Expense by Award Type | Stock-based compensation expense recognized by award type is as follows: Years Ended December 31, 2021 2020 2019 Stock options $ 28,811 $ 18,527 $ 13,371 Restricted stock units 426 40 - Total share based compensation expense $ 29,237 $ 18,567 $ 13,371 |
Stock-Based Compensation Expense | Stock-based compensation expense by classification included within the consolidated statements of operations and comprehensive loss was as follows: Years Ended December 31, 2021 2020 2019 Research and development $ 11,954 $ 7,355 $ 6,153 General and administrative 17,283 11,212 7,218 Total share based compensation expense $ 29,237 $ 18,567 $ 13,371 |
RSU Activity | The following table summarizes our RSU activity for the years ended December 31, 2021 and 2020: Number of Shares Weighted Average Grant Date Fair Value Unvested as of December 31, 2019 - $ - Granted 20,000 25.06 Exercised - Forfeited - Unvested as of December 31, 2020 20,000 25.06 Granted 3,500 62.32 Exercised - Forfeited - Unvested as of December 31, 2021 23,500 30.61 |
Warrants Outstanding and Changes in Warrants to Purchase Common Stock | A summary of the warrants outstanding at December 31, 2021 is as follows: Price Outstanding Grant Date Expiration Date 24.42 7,051 June 28, 2013 June 28, 2023 57.11 603,386 December 21, 2020 December 21, 2030 33.63 301,291 August 9, 2021 August 9, 2031 22.51 153,155 December 17, 2021 December 17, 2031 22.51 153,155 December 17, 2021 December 17, 2031 Total 1,218,038 The following table below is the summary of changes in warrants to purchase common stock for the years ended December 31, 2021 and 2020: Number of Warrant Shares Outstanding Exercise Price per Share Balance as of December 31, 2019 14,102 $ 24.42 Granted 603,386 $ 57.11 Exercised (7,051 ) $ - Balance as of December 31, 2020 610,437 Granted August 2021 301,291 $ 33.63 Granted December 2021 306,310 $ 22.51 Exercised - $ - Balance as of December 31, 2021 1,218,038 |
Fair Value Pricing Model of Warrants | The fair value of the 2021 and 2020 warrants was calculated using the Black-Scholes fair value pricing model with the following inputs: Years Ended December 31, 2021 2020 Risk-free interest rate 1.37 % 0.95 % Expected term (in years) 10.00 10.00 Expected volatility 70.25 % 74.20 % Expected dividend yield 0.00 % 0.00 % Exercise price $ 28.07 $ 57.11 Fair value of common stock $ 28.07 $ 57.11 |
Net Loss Per Share (Tables)
Net Loss Per Share (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Net Loss Per Share [Abstract] | |
Basic and Diluted Net Loss Per Share | Basic and diluted net loss per share attributable to common stockholders was calculated as follows: For the Years Ended December 31, 2021 2020 2019 Numerator: Net loss attributable to common stockholders $ (169,069 ) $ (139,700 ) $ (77,270 ) Denominator: Weighted-average common shares outstanding - basic and diluted 63,235,417 55,380,740 49,010,358 Net loss per share attributable to common stockholders - basic and diluted $ (2.67 ) $ (2.52 ) $ (1.58 ) |
Antidilutive Securities Excluded from Computation of Diluted Net Loss Per Share | The Company excluded the following potential shares of common stock, presented based on amounts outstanding at each period end, from the computation of diluted net loss per share attributable to common stockholders for the periods indicated because including them would have had an anti-dilutive effect: For the Years Ended December 31, 2021 2020 2019 Shares issuable upon conversion of the 2021 Convertible Notes - 160,536 1,620,948 Shares issuable upon conversion of the 2022 Convertible Notes - 1,195,449 - Warrants exercisable for common shares 1,218,038 610,437 14,102 Restricted stock units exercisable for common shares 23,500 - - Options to purchase common shares 11,143,761 11,050,931 9,608,537 12,385,299 13,017,353 11,243,587 |
Income Taxes (Tables)
Income Taxes (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Income Taxes [Abstract] | |
Reconciliation of Income Tax Benefit Computed at Statutory Federal Income Tax Rate to Income Taxes | A reconciliation of income tax benefit computed at the statutory federal income tax rate to income taxes as reflected in the financial statements is as follows: For the years ended December 31, 2021 2020 2019 U.S. federal tax at statutory rate 21.0 % 21.0 % 21.0 % Foreign rate differential (13.0 )% 0.0 % 0.0 % Change in state tax apportionment 0.1 % 0.1 % (17.4 )% Stock compensation 1.5 % 0.9 % (0.7 )% Transfer pricing adjustments (22.8 )% 0.0 % 0.0 % Valuation allowance 4.6 % (35.0 )% (5.1 )% Tax credits 8.7 % 13.5 % 0.0 % Other (0.2 )% (0.5 )% 2.1 % Effective tax rate 0 % 0 % 0 % |
Deferred Tax Assets and Liabilities | The significant components of the Company’s deferred income tax assets and liabilities after applying the enacted corporate tax rates are as follows: At December 31, 2021 2020 2019 Deferred income tax assets (liabilities) R&D Credits $ 35,766 $ 42,613 $ 18,471 Net operating losses ("NOL") and credit carryforwards 26,789 21,359 2,517 Capitalized research and development costs 19,753 23,179 27,652 Stock-based compensation 11,552 7,406 3,896 Debt discount - (748 ) (1,460 ) Warrants 8,382 5,585 - Other (8,881 ) 1,189 643 Valuation allowance (93,361 ) (100,583 ) (51,719 ) Net deferred income tax asset $ - $ - $ - |
Commitments and Contingencies (
Commitments and Contingencies (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Commitments and Contingencies [Abstract] | |
Lease Cost | Rent expense was $1.2 million, $1.1 million, and $0.9 million for the years ended December 31, 2021, 2020 and 2019, respectively. Lease cost December 31, 2021 Operating lease cost $ 645 Finance lease cost Amortization of right of use assets 2,140 Interest on lease liablities 1,845 Total lease cost $ 4,630 |
Maturities of Operating Lease Lease Liabilities | The following table summarizes the maturity of the Company’s lease liabilities on an undiscounted cash flow basis and a reconciliation to the operating lease liabilities recognized on the Company’s balance sheet as of December 31, 2021: Maturity of operating lease liabilities December 31, 2021 2022 917 2023 548 2024 269 2025 64 2026 54 Total lease payments $ 1,852 Less: interest (84 ) Total operating lease liabilities $ 1,768 |
Maturity of Finance Lease Liability | Maturity of finance lease liability December 31, 2021 2022 1,689 2023 1,736 2024 1,791 2025 1,856 2026 1,912 Thereafter 45,000 Total lease payments $ 53,984 Less: interest (33,151 ) Total finance lease liability $ 20,833 |
Balance Sheet Information Related to Leases | Leases December 31, 2021 Operating right-of-use assets $ 1,569 Operating current lease liabilities 863 Operating noncurrent lease liabilities 905 Total operating lease liabilities $ 1,768 Finance right-of-use assets $ 48,480 Finance current lease liability 1,689 Finance noncurrent lease liability 19,144 Total finance lease liability $ 20,833 |
Lease Related to Cash Flow Information, Lease Term and Discount Rate | Other information Cash paid for amounts included in the measurement of lease liabilities: Operating cash flows from operating leases $ 655 Cash flows from finance lease $ 1,644 Weighted-average remaining lease term - operating leases 2.5 years Weighted-average remaining lease term - finance lease 22.7 years Weighted-average discount rate - operating leases 4.46 % Weighted-average discount rate - finance lease 8.96 % |
Nature of Business and Basis _2
Nature of Business and Basis of Presentation (Details) | 12 Months Ended |
Dec. 31, 2021Program | |
Nature of Business and Basis of Presentation [Abstract] | |
Number of clinical-stage programs | 3 |
Risks and Liquidity (Details)
Risks and Liquidity (Details) - USD ($) $ / shares in Units, $ in Thousands | Aug. 27, 2021 | Aug. 02, 2021 | Apr. 26, 2021 | Dec. 31, 2020 | Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | Jun. 30, 2021 | Mar. 31, 2021 | Jun. 12, 2020 | Feb. 20, 2020 | Jan. 04, 2018 |
Risks and Liquidity [Abstract] | ||||||||||||
Accumulated deficit | $ (322,843) | $ (491,912) | $ (322,843) | |||||||||
Cash, cash equivalents and investments | 388,700 | |||||||||||
Shareholders' Equity Disclosure [Abstract] | ||||||||||||
Issuance of common stock, net of issuance costs | 26,354 | 280,763 | $ 177,760 | |||||||||
Conversion of convertible notes into common stock | 40,694 | 7,629 | $ 0 | |||||||||
2022 Convertible Notes [Member] | ||||||||||||
Shareholders' Equity Disclosure [Abstract] | ||||||||||||
Redemption of principal balance | $ 38,400 | 38,350 | $ 0 | 38,350 | $ 38,400 | $ 39,350 | ||||||
Convertible senior notes, interest rate, stated percentage | 6.25% | 6.25% | ||||||||||
Debt instrument maturity date | Aug. 31, 2022 | |||||||||||
Conversion of convertible notes into common stock | $ 38,400 | 7,600 | ||||||||||
Outstanding amount | $ 0 | |||||||||||
2021 Convertible Notes [Member] | ||||||||||||
Shareholders' Equity Disclosure [Abstract] | ||||||||||||
Redemption of principal balance | $ 5,150 | $ 0 | $ 5,150 | $ 5,150 | $ 7,500 | $ 52,000 | ||||||
Convertible senior notes, interest rate, stated percentage | 5.75% | 6.25% | ||||||||||
Debt instrument maturity date | Aug. 1, 2021 | |||||||||||
Conversion of convertible notes into common stock | $ 5,150 | |||||||||||
Outstanding amount | $ 0 | |||||||||||
Common Stock [Member] | ||||||||||||
Shareholders' Equity Disclosure [Abstract] | ||||||||||||
Common stock shares issued and sold (in shares) | 812,516 | 5,339,286 | 9,568,000 | |||||||||
Private Placement [Member] | ||||||||||||
Shareholders' Equity Disclosure [Abstract] | ||||||||||||
Issuance of common stock, net of issuance costs | $ 26,400 | |||||||||||
Private Placement [Member] | Common Stock [Member] | ||||||||||||
Shareholders' Equity Disclosure [Abstract] | ||||||||||||
Common stock shares issued and sold (in shares) | 812,516 | 812,516 | ||||||||||
Share price (in dollars per share) | $ 32.48 | $ 32.48 | ||||||||||
Issuance of common stock, net of issuance costs | $ 26,400 | $ 26,400 |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies, Cash, Cash Equivalents and Restricted Cash (Details) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 |
Cash, Cash Equivalents and Restricted Cash [Abstract] | ||||
Cash and cash equivalents | $ 232,694 | $ 297,098 | ||
Restricted cash | 1,343 | 1,568 | ||
Total | $ 234,037 | $ 298,666 | $ 186,908 | $ 112,791 |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies, Government Grants (Details) - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Government Grants [Abstract] | |||
Receivable of offset grants against expenses | $ 0.1 | $ 3.6 | $ 1.2 |
Summary of Significant Accoun_6
Summary of Significant Accounting Policies, Investments (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Investments [Abstract] | |||
Realized gains (losses) on investment | $ 0 | $ 0 | $ 0 |
Unrealized gain on investments | $ (119) | $ (62) | $ 147 |
Summary of Significant Accoun_7
Summary of Significant Accounting Policies, Goodwill (Details) $ in Millions | 12 Months Ended | ||
Dec. 31, 2021USD ($)Segment | Dec. 31, 2020USD ($) | Dec. 31, 2019USD ($) | |
Goodwill [Abstract] | |||
Number of operating segment | 1 | ||
Number of reporting unit | 1 | ||
Goodwill impairment | $ | $ 0 | $ 0 | $ 0 |
Summary of Significant Accoun_8
Summary of Significant Accounting Policies, Property and Equipment (Details) | 12 Months Ended |
Dec. 31, 2021 | |
Internal use Software [Member] | |
Property and Equipment [Abstract] | |
Estimated useful lives | 6 years |
Minimum [Member] | |
Property and Equipment [Abstract] | |
Estimated useful lives | 3 years |
Maximum [Member] | |
Property and Equipment [Abstract] | |
Estimated useful lives | 15 years |
Summary of Significant Accoun_9
Summary of Significant Accounting Policies, Impairment of Long-Lived Assets (Details) - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Impairment of Long-Lived Assets [Abstract] | |||
Impairment of long-lived assets | $ 0 | $ 0 | $ 0 |
Summary of Significant Accou_10
Summary of Significant Accounting Policies, Tax Credits (Details) - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
NYS Life Sciences Research and Development Tax Credit [Member] | |||
Tax Credits [Abstract] | |||
Research and development incentive income | $ 1 | $ 0 | $ 0 |
NYC Biotechnology Tax Credit Program [Member] | |||
Tax Credits [Abstract] | |||
Research and development incentive income | $ 0 | $ 0 | $ 0.3 |
Fair Value of Financial Instr_3
Fair Value of Financial Instruments (Details) - Recurring [Member] - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Assets [Abstract] | ||
Cash equivalents | $ 179,900 | $ 264,325 |
Investments | 156,046 | 185,621 |
Fair value of financial instruments | 335,946 | 449,946 |
Money Market Mutual Funds [Member] | ||
Assets [Abstract] | ||
Cash equivalents | 179,900 | 193,312 |
United States Treasury Securities [Member] | ||
Assets [Abstract] | ||
Cash equivalents | 62,497 | |
Investments | 44,045 | 112,328 |
Corporate Bonds [Member] | ||
Assets [Abstract] | ||
Cash equivalents | 501 | |
Investments | 96,696 | 63,710 |
Municipal Bonds [Member] | ||
Assets [Abstract] | ||
Investments | 6,000 | 6,000 |
Agency Bonds [Member] | ||
Assets [Abstract] | ||
Cash equivalents | 8,015 | |
Investments | 9,305 | 3,583 |
Level 1 [Member] | ||
Assets [Abstract] | ||
Cash equivalents | 179,900 | 255,809 |
Investments | 44,045 | 112,328 |
Fair value of financial instruments | 223,945 | 368,137 |
Level 1 [Member] | Money Market Mutual Funds [Member] | ||
Assets [Abstract] | ||
Cash equivalents | 179,900 | 193,312 |
Level 1 [Member] | United States Treasury Securities [Member] | ||
Assets [Abstract] | ||
Cash equivalents | 62,497 | |
Investments | 44,045 | 112,328 |
Level 1 [Member] | Corporate Bonds [Member] | ||
Assets [Abstract] | ||
Cash equivalents | 0 | |
Investments | 0 | 0 |
Level 1 [Member] | Municipal Bonds [Member] | ||
Assets [Abstract] | ||
Investments | 0 | 0 |
Level 1 [Member] | Agency Bonds [Member] | ||
Assets [Abstract] | ||
Cash equivalents | 0 | |
Investments | 0 | 0 |
Level 2 [Member] | ||
Assets [Abstract] | ||
Cash equivalents | 0 | 8,516 |
Investments | 112,001 | 73,293 |
Fair value of financial instruments | 112,001 | 81,809 |
Level 2 [Member] | Money Market Mutual Funds [Member] | ||
Assets [Abstract] | ||
Cash equivalents | 0 | 0 |
Level 2 [Member] | United States Treasury Securities [Member] | ||
Assets [Abstract] | ||
Cash equivalents | 0 | |
Investments | 0 | 0 |
Level 2 [Member] | Corporate Bonds [Member] | ||
Assets [Abstract] | ||
Cash equivalents | 501 | |
Investments | 96,696 | 63,710 |
Level 2 [Member] | Municipal Bonds [Member] | ||
Assets [Abstract] | ||
Investments | 6,000 | 6,000 |
Level 2 [Member] | Agency Bonds [Member] | ||
Assets [Abstract] | ||
Cash equivalents | 8,015 | |
Investments | 9,305 | 3,583 |
Level 3 [Member] | ||
Assets [Abstract] | ||
Cash equivalents | 0 | 0 |
Investments | 0 | 0 |
Fair value of financial instruments | 0 | 0 |
Level 3 [Member] | Money Market Mutual Funds [Member] | ||
Assets [Abstract] | ||
Cash equivalents | 0 | 0 |
Level 3 [Member] | United States Treasury Securities [Member] | ||
Assets [Abstract] | ||
Cash equivalents | 0 | |
Investments | 0 | 0 |
Level 3 [Member] | Corporate Bonds [Member] | ||
Assets [Abstract] | ||
Cash equivalents | 0 | |
Investments | 0 | 0 |
Level 3 [Member] | Municipal Bonds [Member] | ||
Assets [Abstract] | ||
Investments | 0 | 0 |
Level 3 [Member] | Agency Bonds [Member] | ||
Assets [Abstract] | ||
Cash equivalents | 0 | |
Investments | $ 0 | $ 0 |
Property and Equipment, Net (De
Property and Equipment, Net (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Property and Equipment [Abstract] | |||
Property and equipment, gross | $ 27,522 | $ 21,252 | |
Less: accumulated depreciation and amortization | (5,223) | (2,046) | |
Property and equipment, net | 22,299 | 19,206 | |
Depreciation and amortization | 3,240 | 1,145 | $ 426 |
Laboratory Equipment [Member] | |||
Property and Equipment [Abstract] | |||
Property and equipment, gross | 12,600 | 7,807 | |
Machinery and Equipment [Member] | |||
Property and Equipment [Abstract] | |||
Property and equipment, gross | 10,432 | 9,933 | |
Computer Equipment [Member] | |||
Property and Equipment [Abstract] | |||
Property and equipment, gross | 218 | 218 | |
Furniture and Fixtures [Member] | |||
Property and Equipment [Abstract] | |||
Property and equipment, gross | 1,963 | 1,880 | |
Leasehold Improvements [Member] | |||
Property and Equipment [Abstract] | |||
Property and equipment, gross | 407 | 29 | |
Internal use Software [Member] | |||
Property and Equipment [Abstract] | |||
Property and equipment, gross | $ 1,902 | $ 1,385 |
Accounts Payable and Accrued _3
Accounts Payable and Accrued Expenses (Details) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Accounts Payable and Accrued Expenses [Abstract] | ||
Research and development | $ 12,082 | $ 14,962 |
Property and equipment | 725 | 1,756 |
Employee compensation | 4,533 | 4,875 |
Accrued interest | 0 | 1,122 |
Government grant payable | 597 | 590 |
Professional fees | 1,196 | 1,332 |
Other | 482 | 835 |
Total | $ 19,615 | $ 25,472 |
Convertible Notes Payable (Deta
Convertible Notes Payable (Details) | Aug. 02, 2021USD ($)shares | Apr. 26, 2021USD ($)dshares | Feb. 20, 2020USD ($)$ / shares | Dec. 31, 2020USD ($)shares | Dec. 31, 2021USD ($)d$ / shares | Dec. 31, 2020USD ($) | Dec. 31, 2019USD ($) | Jun. 30, 2021USD ($) | Mar. 31, 2021USD ($) | Jun. 12, 2020USD ($) | Jan. 04, 2018USD ($) |
Debt [Abstract] | |||||||||||
Conversion of convertible notes into common stock | $ 40,694,000 | $ 7,629,000 | $ 0 | ||||||||
Carrying value of convertible notes [Abstract] | |||||||||||
Carrying value | $ 35,066,000 | 0 | 35,066,000 | ||||||||
Accretion of debt discount | $ 753,000 | 2,758,000 | 3,602,000 | ||||||||
2021 Convertible Notes [Member] | |||||||||||
Debt [Abstract] | |||||||||||
Debt instrument maturity date | Aug. 1, 2021 | ||||||||||
Convertible senior notes, interest rate, stated percentage | 5.75% | 6.25% | |||||||||
Debt conversion ratio | 31.1876 | ||||||||||
Conversion per principal amount of debt | $ 1,000 | ||||||||||
Conversion price (in dollars per share) | $ / shares | $ 32.08 | ||||||||||
Conversion of convertible notes into common stock | $ 5,150,000 | ||||||||||
Convertible Notes converted into shares of common stock (in shares) | shares | 160,614 | ||||||||||
Convertible notes, outstanding | $ 0 | ||||||||||
Debt instrument, aggregate principal amount exchanged | $ 39,350,000 | $ 7,500,000 | |||||||||
Carrying value of convertible notes [Abstract] | |||||||||||
Principal amount | 5,150,000 | 0 | 5,150,000 | $ 5,150,000 | $ 7,500,000 | $ 52,000,000 | |||||
Discount | (275,000) | 0 | (275,000) | ||||||||
Carrying value | 4,875,000 | 0 | 4,875,000 | ||||||||
Accretion of debt discount | $ 300,000 | 1,300,000 | $ 3,600,000 | ||||||||
2022 Convertible Notes [Member] | |||||||||||
Debt [Abstract] | |||||||||||
Debt instrument maturity date | Aug. 31, 2022 | ||||||||||
Convertible senior notes, interest rate, stated percentage | 6.25% | 6.25% | |||||||||
Debt conversion ratio | 31.1876 | ||||||||||
Conversion per principal amount of debt | $ 1,000 | ||||||||||
Conversion price (in dollars per share) | $ / shares | $ 32.06 | ||||||||||
Conversion of convertible notes into common stock | $ 38,400,000 | $ 7,600,000 | |||||||||
Convertible Notes converted into shares of common stock (in shares) | shares | 1,300,000 | 298,562 | |||||||||
Convertible notes, outstanding | $ 0 | ||||||||||
Percentage of common stock conversion price | 130.00% | ||||||||||
Number of trading days, not consecutive | d | 20 | 20 | |||||||||
Number of consecutive trading days | d | 30 | 30 | |||||||||
Conversion of convertible notes into common stock, principal amount | $ 8,500,000 | ||||||||||
Carrying value of convertible notes [Abstract] | |||||||||||
Principal amount | $ 38,400,000 | $ 39,350,000 | 38,350,000 | $ 0 | 38,350,000 | $ 38,400,000 | |||||
Discount | (3,284,000) | 0 | (3,284,000) | ||||||||
Carrying value | $ 35,066,000 | 0 | 35,066,000 | ||||||||
Accretion of debt discount | $ 500,000 | $ 1,500,000 |
Shareholders' Equity (Details)
Shareholders' Equity (Details) $ / shares in Units, $ in Thousands | Aug. 27, 2021USD ($)$ / sharesshares | Dec. 14, 2020USD ($)$ / sharesshares | Dec. 12, 2019USD ($) | Dec. 31, 2021USD ($)Vote$ / sharesshares | Dec. 31, 2020USD ($)$ / sharesshares | Dec. 31, 2019USD ($)shares |
Common Stock [Abstract] | ||||||
Common stock, shares authorized (in shares) | shares | 120,000,000 | 120,000,000 | ||||
Common stock, par value (in dollars per share) | $ / shares | $ 0.01 | $ 0.01 | ||||
Number of voting right for each share | Vote | 1 | |||||
Treasury Stock [Abstract] | ||||||
Shares withheld to pay the payroll tax liability | $ 0 | $ 614 | $ 344 | |||
Private Placement and Public Offerings [Abstract] | ||||||
Issuance of common stock, net of issuance costs | 26,354 | 280,763 | 177,760 | |||
Net proceeds from offering | $ 11,327 | $ 2,558 | $ 31 | |||
Private Placement [Member] | ||||||
Private Placement and Public Offerings [Abstract] | ||||||
Issuance of common stock, net of issuance costs | $ 26,400 | |||||
Common Stock [Member] | ||||||
Private Placement and Public Offerings [Abstract] | ||||||
Common stock shares issued (in shares) | shares | 812,516 | 5,339,286 | 9,568,000 | |||
Common Stock [Member] | Private Placement [Member] | ||||||
Private Placement and Public Offerings [Abstract] | ||||||
Common stock shares issued (in shares) | shares | 812,516 | 812,516 | ||||
Share price (in dollars per share) | $ / shares | $ 32.48 | $ 32.48 | ||||
Issuance of common stock, net of issuance costs | $ 26,400 | $ 26,400 | ||||
Common Stock [Member] | Public Offering [Member] | ||||||
Private Placement and Public Offerings [Abstract] | ||||||
Common stock shares issued (in shares) | shares | 5,339,286 | |||||
Share price (in dollars per share) | $ / shares | $ 56 | |||||
Gross proceeds, offering amount | $ 299,000 | |||||
Offering costs | 18,200 | |||||
Net proceeds from offering | $ 280,800 | |||||
Common Stock [Member] | Exercise of Underwriters' Option [Member] | ||||||
Private Placement and Public Offerings [Abstract] | ||||||
Common stock shares issued (in shares) | shares | 696,428 | |||||
Treasury Stock [Member] | ||||||
Treasury Stock [Abstract] | ||||||
Shares withheld to pay the payroll tax liability | $ 100 | |||||
Treasury stock (in shares) | shares | 0 | 0 |
Stock-Based Awards, Stock Optio
Stock-Based Awards, Stock Option Valuation (Details) - Stock Options [Member] - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Stock Option Activity [Roll Forward] | |||
Outstanding at beginning of period (in shares) | 11,050,931 | 9,763,541 | |
Granted (in shares) | 1,671,759 | 2,193,546 | |
Exercised (in shares) | (1,209,960) | (586,857) | |
Cancelled (in shares) | (368,969) | (319,299) | |
Outstanding at end of period (in shares) | 11,143,761 | 11,050,931 | 9,763,541 |
Options vested and exercisable at end of period (in shares) | 8,692,898 | ||
Options unvested at end of period (in shares) | 2,450,863 | ||
Weighted Average Exercise Price [Abstract] | |||
Outstanding at beginning of period (in dollars per share) | $ 9.10 | $ 5.95 | |
Granted (in dollars per share) | 51.20 | 22.96 | |
Exercised (in dollars per share) | 9.32 | 4.82 | |
Cancelled (in dollars per share) | 35.87 | 15.82 | |
Outstanding at end of period (in dollars per share) | 14.51 | $ 9.10 | $ 5.95 |
Options vested and exercisable at end of period (in dollars per share) | 7.31 | ||
Options unvested at end of period (in dollars per share) | $ 40.01 | ||
Weighted-Average Remaining Contractual Term [Abstract] | |||
Outstanding | 5 years 11 months 12 days | 6 years 6 months 18 days | 6 years 11 months 15 days |
Granted | 8 years 6 months 29 days | 8 years 11 months 19 days | |
Options vested and exercisable | 5 years 1 month 9 days | ||
Options unvested | 8 years 11 months 4 days | ||
Aggregate Intrinsic Value [Abstract] | |||
Outstanding at beginning of period | $ 504,079 | $ 164,021 | |
Exercised | 54,487 | 13,494 | |
Outstanding at end of period | 128,817 | $ 504,079 | $ 164,021 |
Options vested and exercisable | $ 127,571 | ||
Weighted average grant date fair value of shares granted (in dollars per share) | $ 31.07 | $ 15.10 | $ 9.58 |
Total fair value of options vested | $ 22,600 | $ 15,600 | $ 63,100 |
Employees and Directors [Member] | |||
Fair Value Assumptions [Abstract] | |||
Risk-free interest rate | 0.83% | 1.00% | 2.26% |
Expected term (in years) | 5 years 10 months 2 days | 5 years 10 months 2 days | 5 years 9 months 21 days |
Expected volatility | 69.27% | 76.98% | 75.71% |
Expected dividend yield | 0.00% | 0.00% | 0.00% |
Exercise price (in dollars per share) | $ 51.20 | $ 22.96 | $ 14.47 |
Fair value of common stock (in dollars per share) | $ 51.20 | $ 22.96 | $ 14.47 |
Stock-Based Awards, Stock-Based
Stock-Based Awards, Stock-Based Compensation (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Stock-Based Compensation [Abstract] | |||
Share based compensation expense | $ 29,237 | $ 18,567 | $ 13,371 |
Unrecognized share-based compensation cost | $ 46,300 | $ 30,400 | |
Weighted average period expected to recognize unrecognized share-based compensation cost | 1 year 9 months 18 days | 2 years 1 month 6 days | |
Research and Development [Member] | |||
Stock-Based Compensation [Abstract] | |||
Share based compensation expense | $ 11,954 | $ 7,355 | 6,153 |
General and Administrative [Member] | |||
Stock-Based Compensation [Abstract] | |||
Share based compensation expense | 17,283 | 11,212 | 7,218 |
Stock Options [Member] | |||
Stock-Based Compensation [Abstract] | |||
Share based compensation expense | 28,811 | 18,527 | 13,371 |
Restricted Stock Units [Member] | |||
Stock-Based Compensation [Abstract] | |||
Share based compensation expense | 426 | $ 40 | $ 0 |
Unrecognized share-based compensation cost | $ 500 | ||
Weighted average period expected to recognize unrecognized share-based compensation cost | 2 years 9 months 18 days |
Stock-Based Awards, Restricted
Stock-Based Awards, Restricted Stock Units (Details) $ / shares in Units, $ in Millions | 12 Months Ended | |
Dec. 31, 2021USD ($)$ / sharesshares | Dec. 31, 2020USD ($)$ / sharesshares | |
Number of Shares [Roll Forward] | ||
Exercised (in shares) | 7,051 | |
Weighted Average Grant Date Fair Value [Abstract] | ||
Unrecognized share-based compensation cost | $ | $ 46.3 | $ 30.4 |
Weighted average period expected to recognize unrecognized share-based compensation cost | 1 year 9 months 18 days | 2 years 1 month 6 days |
Restricted Stock Units (RSU) [Member] | ||
Number of Shares [Roll Forward] | ||
Beginning balance (in shares) | 20,000 | 0 |
Granted (in shares) | 3,500 | 20,000 |
Exercised (in shares) | 0 | 0 |
Forfeited (in shares) | 0 | 0 |
Ending balance (in shares) | 23,500 | 20,000 |
Weighted Average Grant Date Fair Value [Abstract] | ||
Beginning balance (in dollars per share) | $ / shares | $ 25.06 | $ 0 |
Granted (in dollars per share) | $ / shares | 62.32 | 25.06 |
Ending balance (in dollars per share) | $ / shares | $ 30.61 | $ 25.06 |
Intrinsic value | $ | $ 0.4 | $ 0.5 |
Unrecognized share-based compensation cost | $ | $ 0.5 | |
Weighted average period expected to recognize unrecognized share-based compensation cost | 2 years 9 months 18 days |
Stock-Based Awards, Warrants (D
Stock-Based Awards, Warrants (Details) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | 12 Months Ended | ||||||
Dec. 31, 2021 | Aug. 31, 2021 | Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | Dec. 17, 2021 | Aug. 09, 2021 | Dec. 21, 2020 | |
Warrants [Abstract] | ||||||||
Outstanding (in shares) | 1,218,038 | 1,218,038 | ||||||
Number of Warrant Shares Outstanding and Exercisable [Abstract] | ||||||||
Exercised (in shares) | (7,051) | |||||||
Expense in connection with warrant issue | $ 12,781 | $ 26,562 | $ 0 | |||||
Warrants [Member] | ||||||||
Warrants [Abstract] | ||||||||
Exercise price per share (in dollars per share) | $ 24.42 | $ 22.51 | $ 33.63 | $ 57.11 | ||||
Number of Warrant Shares Outstanding and Exercisable [Abstract] | ||||||||
Beginning balance (in shares) | 610,437 | 14,102 | ||||||
Granted (in shares) | 306,310 | 301,291 | 603,386 | |||||
Exercised (in shares) | 0 | (7,051) | ||||||
Ending balance (in shares) | 1,218,038 | 1,218,038 | 610,437 | 14,102 | ||||
Fair Value Assumptions [Abstract] | ||||||||
Risk-free interest rate | 1.37% | 0.95% | ||||||
Expected term (in years) | 10 years | 10 years | ||||||
Expected volatility | 70.25% | 74.20% | ||||||
Expected dividend yield | 0.00% | 0.00% | ||||||
Exercise price (in dollars per share) | $ 28.07 | $ 28.07 | $ 57.11 | |||||
Fair value of common stock (in dollars per share) | 28.07 | 28.07 | $ 57.11 | |||||
Common Stock [Member] | ||||||||
Number of Warrant Shares Outstanding and Exercisable [Abstract] | ||||||||
Issuance of common stock pursuant to exercise of warrant (in shares) | 1,601 | |||||||
22.42 [Member] | ||||||||
Warrants [Abstract] | ||||||||
Exercise price per share (in dollars per share) | $ 24.42 | $ 24.42 | ||||||
Outstanding (in shares) | 7,051 | 7,051 | ||||||
Grant date | Jun. 28, 2013 | |||||||
Expiration date | Jun. 28, 2023 | Jun. 28, 2023 | ||||||
57.11 [Member] | ||||||||
Warrants [Abstract] | ||||||||
Exercise price per share (in dollars per share) | $ 57.11 | $ 57.11 | ||||||
Outstanding (in shares) | 603,386 | 603,386 | ||||||
Grant date | Dec. 21, 2020 | |||||||
Expiration date | Dec. 21, 2030 | Dec. 21, 2030 | ||||||
33.63 [Member] | ||||||||
Warrants [Abstract] | ||||||||
Exercise price per share (in dollars per share) | $ 33.63 | $ 33.63 | ||||||
Outstanding (in shares) | 301,291 | 301,291 | ||||||
Grant date | Aug. 9, 2021 | |||||||
Expiration date | Aug. 9, 2031 | Aug. 9, 2031 | ||||||
22.51 [Member] | ||||||||
Warrants [Abstract] | ||||||||
Exercise price per share (in dollars per share) | $ 22.51 | $ 22.51 | ||||||
Outstanding (in shares) | 153,155 | 153,155 | ||||||
Grant date | Dec. 17, 2021 | |||||||
Expiration date | Dec. 17, 2031 | Dec. 17, 2031 | ||||||
22.51 [Member] | ||||||||
Warrants [Abstract] | ||||||||
Exercise price per share (in dollars per share) | $ 22.51 | $ 22.51 | ||||||
Outstanding (in shares) | 153,155 | 153,155 | ||||||
Grant date | Dec. 17, 2021 | |||||||
Expiration date | Dec. 17, 2031 | Dec. 17, 2031 |
Net Loss Per Share (Details)
Net Loss Per Share (Details) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Numerator [Abstract] | |||
Net loss attributable to common stockholders | $ (169,069) | $ (139,700) | $ (77,270) |
Denominator [Abstract] | |||
Weighted-average common shares outstanding - basic (in shares) | 63,235,417 | 55,380,740 | 49,010,358 |
Weighted-average common shares outstanding - diluted (in shares) | 63,235,417 | 55,380,740 | 49,010,358 |
Net loss per share attributable to common stockholders - basic (in dollars per share) | $ (2.67) | $ (2.52) | $ (1.58) |
Net loss per share attributable to common stockholders - diluted (in dollars per share) | $ (2.67) | $ (2.52) | $ (1.58) |
Weighted Average Number of Shares Outstanding Reconciliation [Abstract] | |||
Warrants exercisable for common shares (in shares) | 1,218,038 | 610,437 | 14,102 |
Restricted stock units exercisable for common shares (in shares) | 23,500 | 0 | 0 |
Options to purchase common shares (in shares) | 11,143,761 | 11,050,931 | 9,608,537 |
Weighted Average Number Diluted Shares Outstanding (in shares) | 12,385,299 | 13,017,353 | 11,243,587 |
Convertible Notes 2021 [Member] | |||
Weighted Average Number of Shares Outstanding Reconciliation [Abstract] | |||
Shares issuable upon conversion of Convertible Notes (in shares) | 0 | 160,536 | 1,620,948 |
Convertible Notes 2022 [Member] | |||
Weighted Average Number of Shares Outstanding Reconciliation [Abstract] | |||
Shares issuable upon conversion of Convertible Notes (in shares) | 0 | 1,195,449 | 0 |
Income Taxes, Net Loss by Juris
Income Taxes, Net Loss by Jurisdiction (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Federal [Member] | |||
Details of Income Taxes [Abstract] | |||
Provision for income taxes | $ 0 | $ 0 | $ 0 |
State [Member] | |||
Details of Income Taxes [Abstract] | |||
Provision for income taxes | $ 0 | $ 0 | $ 0 |
Income Taxes, Reconciliation Be
Income Taxes, Reconciliation Between Effective Tax Rates and Statutory Rates (Details) | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Reconciliation of Income Tax Benefit Computed at Statutory Federal Income Tax Rate to Income Taxes [Abstract] | |||
U.S. federal tax at statutory rate | 21.00% | 21.00% | 21.00% |
Foreign rate differential | (13.00%) | 0.00% | 0.00% |
Change in state tax apportionment | 0.10% | 0.10% | (17.40%) |
Stock compensation | 1.50% | 0.90% | (0.70%) |
Transfer pricing adjustments | (22.80%) | 0.00% | 0.00% |
Valuation allowance | 4.60% | (35.00%) | (5.10%) |
Tax credits | 8.70% | 13.50% | 0.00% |
Other | (0.20%) | (0.50%) | 2.10% |
Effective tax rate | 0.00% | 0.00% | 0.00% |
Income Taxes, Deferred Tax Asse
Income Taxes, Deferred Tax Assets and Liabilities (Details) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 |
Deferred income tax assets (liabilities) [Abstract] | |||
R&D Credits | $ 35,766 | $ 42,613 | $ 18,471 |
Net operating losses ("NOL") and credit carryforwards | 26,789 | 21,359 | 2,517 |
Capitalized research and development costs | 19,753 | 23,179 | 27,652 |
Stock-based compensation | 11,552 | 7,406 | 3,896 |
Debt discount | 0 | (748) | (1,460) |
Warrants | 8,382 | 5,585 | 0 |
Other | 1,189 | 643 | |
Other | (8,881) | ||
Valuation allowance | (93,361) | (100,583) | (51,719) |
Net deferred income tax asset | $ 0 | $ 0 | $ 0 |
Income Taxes, NOL Carryforwards
Income Taxes, NOL Carryforwards and Tax Credits (Details) - USD ($) $ in Thousands | 12 Months Ended | |||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2017 | |
Operating Loss Carryforwards [Abstract] | ||||
R&D Credits | $ 35,766 | $ 42,613 | $ 18,471 | |
Increase in valuation allowance | (7,200) | $ 48,900 | ||
Annual limitations under section 382 of IRC | $ 1,700 | |||
Tax Credit Carryforward [Abstract] | ||||
Transfer pricing adjustments | (22.80%) | 0.00% | 0.00% | |
Uncertain Tax Positions [Abstract] | ||||
Uncertain tax positions | $ 0 | $ 0 | $ 0 | |
Income Tax Penalties and Interest Accrued [Abstract] | ||||
Accrued interest or penalties | 0 | 0 | 0 | |
Income tax adjustments from prior periods | $ 0 | |||
Research and Development [Member] | ||||
Tax Credit Carryforward [Abstract] | ||||
Transfer pricing adjustments | 10.00% | |||
Income tax expense | $ 0 | |||
Federal [Member] | ||||
Operating Loss Carryforwards [Abstract] | ||||
NOL carryforwards | $ 118,200 | $ 127,100 | ||
NOL carryforwards, expiration date | Dec. 31, 2026 | |||
Net operating loss not subject to expiration | $ 85,700 | |||
R&D Credits | $ 35,800 | |||
Tax credit, expiration date | Dec. 31, 2038 | |||
Net operating loss, subject to expiration | $ 35,800 | $ 91,200 | ||
Cumulative limitation period | 20 years | |||
Tax Credit Carryforward [Abstract] | ||||
Income tax expense | $ 0 | 0 | 0 | |
Federal [Member] | Research and Development [Member] | ||||
Tax Credit Carryforward [Abstract] | ||||
Tax credit carryforwards, subject to expiration | 4,900 | |||
State [Member] | ||||
Operating Loss Carryforwards [Abstract] | ||||
NOL carryforwards | $ 30,900 | |||
NOL carryforwards, expiration date | Dec. 31, 2026 | |||
Tax Credit Carryforward [Abstract] | ||||
Income tax expense | $ 0 | $ 0 | $ 0 |
Commitments and Contingencies,
Commitments and Contingencies, Finance Lease (Details) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021USD ($)ft²LeaseAgreement | Dec. 31, 2020USD ($) | Mar. 15, 2020USD ($) | |
Finance Lease [Abstract] | |||
Total lease payments | $ 53,984 | ||
Cash security deposit | 455 | $ 455 | |
Restricted cash | 1,343 | 1,568 | |
Finance lease, right-of-use assets | 48,480 | 50,521 | |
Finance lease, liability | 20,833 | ||
Operating and Finance Leases [Member] | |||
Finance Lease [Abstract] | |||
Restricted cash | $ 800 | 1,100 | |
NJ Lease Agreement [Member] | |||
Finance Lease [Abstract] | |||
Area of lease | ft² | 103,720 | ||
Term of finance lease agreement | 15 years | ||
Number of options to renew lease agreement | LeaseAgreement | 2 | ||
Term of renewal of finance lease agreement | 5 years | ||
Estimated rent payments | $ 1,200 | ||
Percentage of annual increase in base rent | 3.00% | ||
Total lease payments | $ 29,300 | ||
Cash security deposit | 300 | $ 300 | |
Finance lease, right-of-use assets | $ 47,700 | ||
Finance lease, liability | 20,200 | ||
Reclassification of construction costs in progress to right-of-use assets | 100 | 26,500 | |
Reclassification of prepaid rent to right-of-use assets | $ 1,100 | ||
Aggregate amount for reclassification of construction costs in progress to right-of-use assets | $ 32,100 | ||
AAV Current Good Manufacturing Practice (cGMP) [Member] | |||
Finance Lease [Abstract] | |||
Area of lease | ft² | 50,000 |
Commitments and Contingencies_2
Commitments and Contingencies, Operating Leases (Details) - USD ($) $ in Millions | Mar. 26, 2021 | May 31, 2020 | Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | Jun. 07, 2018 |
Operating Leases [Abstract] | ||||||
Rent expense | $ 1.2 | $ 1.1 | $ 0.9 | |||
NY Lease Agreement [Member] | ||||||
Operating Leases [Abstract] | ||||||
Loss on lease termination | $ (76) | |||||
Lease expiration date | Jul. 31, 2022 | |||||
ESB Lease Agreement [Member] | ||||||
Operating Leases [Abstract] | ||||||
Term of lease agreement | 3 years | |||||
Letter of credit | $ 0.9 | |||||
Certificate of deposit | $ 0.8 | 0.9 | ||||
Lease expiration date | Aug. 29, 2024 | |||||
ESB Lease Agreement Amendment [Member] | ||||||
Operating Leases [Abstract] | ||||||
Letter of credit | $ 0.8 | |||||
Operating lease right of use asset, adjustment | $ 1.1 | |||||
Lease expiration date | Jun. 30, 2024 | |||||
Inotek Lexington Massachusetts Lease Agreement [Member] | ||||||
Operating Leases [Abstract] | ||||||
Lease expiration date | Feb. 28, 2023 | |||||
Rental income received under sublease agreement | $ 0.4 | $ 0.4 | $ 0.3 |
Commitments and Contingencies_3
Commitments and Contingencies, Details of Operating and Finance Lease (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Lease cost [Abstract] | ||
Operating lease cost | $ 645 | |
Finance lease cost [Abstract] | ||
Amortization of right of use assets | 2,140 | |
Interest on lease liabilities | 1,845 | |
Total lease cost | 4,630 | |
Maturities of operating lease liabilities [Abstract] | ||
2022 | 917 | |
2023 | 548 | |
2024 | 269 | |
2025 | 64 | |
2026 | 54 | |
Total lease payments | 1,852 | |
Less: interest | (84) | |
Total operating lease liabilities | 1,768 | |
Maturity of finance lease liability [Abstract] | ||
2022 | 1,689 | |
2023 | 1,736 | |
2024 | 1,791 | |
2025 | 1,856 | |
2026 | 1,912 | |
Thereafter | 45,000 | |
Total lease payments | 53,984 | |
Less: interest | (33,151) | |
Total finance lease liability | 20,833 | |
Lease assets and liabilities [Abstract] | ||
Operating right-of-use assets | 1,569 | $ 914 |
Operating current lease liabilities | 863 | 626 |
Operating noncurrent lease liabilities | 905 | 498 |
Total operating lease liabilities | 1,768 | |
Finance lease, right-of-use assets | 48,480 | 50,521 |
Finance current lease liability | 1,689 | 1,644 |
Finance noncurrent lease liability | 19,144 | $ 18,988 |
Total finance lease liability | 20,833 | |
Cash paid for amounts included in the measurement of lease liabilities [Abstract] | ||
Operating cash flows from operating leases | 655 | |
Cash flows from finance lease | $ 1,644 | |
Weighted-average remaining lease term - operating leases | 2 years 6 months | |
Weighted-average remaining lease term - finance lease | 22 years 8 months 12 days | |
Weighted-average discount rate - operating leases | 4.46% | |
Weighted-average discount rate - finance lease | 8.96% |
Agreements Related to Intelle_2
Agreements Related to Intellectual Property (Details) $ in Thousands, $ / Vector in Millions | 1 Months Ended | 12 Months Ended | |
Feb. 28, 2017USD ($) | Dec. 31, 2021USD ($)MilestonePatent$ / Vector | Dec. 31, 2020Milestone | |
License Agreement for Danon Disease [Member] | |||
License Agreement [Abstract] | |||
Research and development costs | $ 50 | ||
License Agreement for Danon Disease [Member] | Maximum [Member] | |||
License Agreement [Abstract] | |||
Potential future obligation | $ 1,500 | ||
REGENXBIO, Inc. License [Member] | |||
License Agreement [Abstract] | |||
Research and development costs | $ 7,000 | ||
Potential future obligation | $ 13,000 | ||
Term of agreement | 10 years | ||
Termination notice period | 6 months | ||
Number of patents | Patent | 2 | ||
Options right expiry term | 4 years | ||
Fee payable (per additional vector) | $ / Vector | 2 | ||
Royalties payable on fees received | 20.00% | ||
Payment for License Fee | $ 1,000 | ||
Number of additional milestones achieved | Milestone | 0 | 0 |
CIRM Grants (Details)
CIRM Grants (Details) $ in Millions | Apr. 01, 2021USD ($) | Jan. 04, 2021USD ($) | Dec. 31, 2021USD ($)Grant | Dec. 31, 2019USD ($) | Dec. 31, 2020USD ($) | Nov. 12, 2020USD ($) | Apr. 30, 2019USD ($) |
LAD-I CIRM Grant [Member] | |||||||
CIRM Grant [Abstract] | |||||||
Grant award for clinical development support | $ 6.5 | ||||||
Amount received from grant for eligible costs incurred | $ 1.2 | ||||||
Grant receivable included in prepaid and other assets | $ 1.1 | ||||||
Number of grants | Grant | 2 | ||||||
Milestone payments received | $ 1 | $ 1.1 | |||||
IMO CIRM Grant [Member] | |||||||
CIRM Grant [Abstract] | |||||||
Grant award for clinical development support | $ 3.7 | ||||||
Amount received from grant for eligible costs incurred | $ 1 | ||||||
Grant receivable included in prepaid and other assets | $ 0.9 | ||||||
Decrease in research and development expense | $ 0.1 |
Related Party Transactions (Det
Related Party Transactions (Details) - USD ($) $ / shares in Units, $ in Thousands | Aug. 27, 2021 | Dec. 31, 2021 | Aug. 31, 2021 | Oct. 31, 2020 | Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | Dec. 17, 2021 | Aug. 09, 2021 | Dec. 21, 2020 |
Related Party Transaction [Abstract] | ||||||||||
Expense in connection with warrant issue | $ 12,781 | $ 26,562 | $ 0 | |||||||
Issuance of common stock, net of issuance costs | $ 26,354 | $ 280,763 | $ 177,760 | |||||||
Private Placement [Member] | ||||||||||
Related Party Transaction [Abstract] | ||||||||||
Issuance of common stock, net of issuance costs | $ 26,400 | |||||||||
Period to file S-3 following demand by purchaser | 60 days | |||||||||
Common Stock [Member] | ||||||||||
Related Party Transaction [Abstract] | ||||||||||
Issuance of common stock, net of issuance costs (in shares) | 812,516 | 5,339,286 | 9,568,000 | |||||||
Common Stock [Member] | Private Placement [Member] | ||||||||||
Related Party Transaction [Abstract] | ||||||||||
Issuance of common stock, net of issuance costs (in shares) | 812,516 | 812,516 | ||||||||
Share price (in dollars per share) | $ 32.48 | $ 32.48 | $ 32.48 | |||||||
Issuance of common stock, net of issuance costs | $ 26,400 | $ 26,400 | ||||||||
Restricted Stock Units (RSU) [Member] | ||||||||||
Related Party Transaction [Abstract] | ||||||||||
Shares granted (in shares) | 3,500 | 20,000 | ||||||||
Warrants [Member] | ||||||||||
Related Party Transaction [Abstract] | ||||||||||
Shares granted (in shares) | 306,310 | 301,291 | 603,386 | |||||||
Warrant issued (in shares) | 12,800,000 | 12,800,000 | ||||||||
Consulting Agreement, Business Development Services [Member] | ||||||||||
Related Party Transaction [Abstract] | ||||||||||
Termination notice period for business development consulting services agreement | 60 days | |||||||||
Term of consulting agreement | 3 years | |||||||||
Consulting Agreement, Business Development Services [Member] | Warrants [Member] | ||||||||||
Related Party Transaction [Abstract] | ||||||||||
Warrant issued (in shares) | 603,386 | |||||||||
Expense in connection with warrant issue | $ 26,600 | |||||||||
August 2021 Warrant [Member] | Common Stock [Member] | ||||||||||
Related Party Transaction [Abstract] | ||||||||||
Expense in connection with warrant issue | $ 7,600 | |||||||||
August 2021 Warrant [Member] | Consulting Agreement, Business Development Services [Member] | Common Stock [Member] | ||||||||||
Related Party Transaction [Abstract] | ||||||||||
Warrant issued (in shares) | 301,291 | |||||||||
December 2021 Warrants [Member] | Common Stock [Member] | ||||||||||
Related Party Transaction [Abstract] | ||||||||||
Expense in connection with warrant issue | 5,200 | |||||||||
December 2021 Warrants [Member] | Consulting Agreement, Business Development Services [Member] | Common Stock [Member] | ||||||||||
Related Party Transaction [Abstract] | ||||||||||
Warrant issued (in shares) | 153,155 | |||||||||
December 2021 Warrants [Member] | Consulting Agreement, Asset Identification Services [Member] | Common Stock [Member] | ||||||||||
Related Party Transaction [Abstract] | ||||||||||
Warrant issued (in shares) | 153,155 | |||||||||
Member of the Board of Directors - One [Member] | ||||||||||
Related Party Transaction [Abstract] | ||||||||||
Business development consulting services expense (per quarter) | $ 28 | |||||||||
Termination notice period for business development consulting services agreement | 14 days | |||||||||
Related party expenses | $ 100 | $ 100 | $ 100 | |||||||
Member of the Board of Directors - Two [Member] | ||||||||||
Related Party Transaction [Abstract] | ||||||||||
Term of consulting agreement | 1 year | |||||||||
Number of options granted (in shares) | 20,000 | |||||||||
Fair value amount of options granted | $ 400 | |||||||||
Spouse of Executive officer [Member] | Restricted Stock Units (RSU) [Member] | ||||||||||
Related Party Transaction [Abstract] | ||||||||||
Shares granted (in shares) | 10,000 | |||||||||
Vesting period | 3 years |
401(k) Savings Plan (Details)
401(k) Savings Plan (Details) - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
401(k) Savings Plan [Abstract] | |||
Percentage of matching employee contributions | 4.00% | ||
Matching employee contributions | $ 0.6 | $ 0.3 | $ 0.2 |