Document and Entity Information
Document and Entity Information - shares | 3 Months Ended | |
Mar. 31, 2021 | May 03, 2021 | |
Cover page [Abstract] | ||
Entity Registrant Name | Rocket Pharmaceuticals, Inc. | |
Entity Central Index Key | 0001281895 | |
Current Fiscal Year End Date | --12-31 | |
Document Type | 10-Q | |
Amendment Flag | false | |
Document Quarterly Report | true | |
Document Period End Date | Mar. 31, 2021 | |
Document Fiscal Year Focus | 2021 | |
Document Fiscal Period Focus | Q1 | |
Document Transition Report | false | |
Entity File Number | 001-36829 | |
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 | 646 | |
Local Phone Number | 440-9100 | |
Title of 12(b) Security | Common Stock, $0.01 par value | |
Trading Symbol | RCKT | |
Security Exchange Name | NASDAQ | |
Entity Current Reporting Status | Yes | |
Entity Interactive Data Current | Yes | |
Entity Filer Category | Large Accelerated Filer | |
Entity Small Business | false | |
Entity Emerging Growth Company | false | |
Entity Shell Company | false | |
Entity Common Stock, Shares Outstanding | 63,396,954 |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) $ in Thousands | Mar. 31, 2021 | Dec. 31, 2020 |
Current assets: | ||
Cash and cash equivalents | $ 252,540 | $ 297,098 |
Investments | 213,813 | 185,621 |
Prepaid expenses and other assets | 5,469 | 4,626 |
Total current assets | 471,822 | 487,345 |
Property and equipment, net | 19,833 | 19,206 |
Goodwill | 30,815 | 30,815 |
Restricted cash | 1,461 | 1,568 |
Deposits | 455 | 455 |
Operating lease right-of-use assets | 1,787 | 914 |
Finance lease right-of-use asset | 50,311 | 50,521 |
Total assets | 576,484 | 590,824 |
Current liabilities: | ||
Accounts payable and accrued expenses | 33,168 | 25,472 |
Convertible notes, net of unamortized discount, current | 4,986 | 4,875 |
Operating lease liabilities, current | 683 | 626 |
Finance lease liability, current | 1,655 | 1,644 |
Total current liabilities | 40,492 | 32,617 |
Convertible notes, net of unamortized discount, non-current | 35,544 | 35,066 |
Operating lease liabilities, non-current | 1,299 | 498 |
Finance lease liabilities, non-current | 19,028 | 18,988 |
Other liabilities | 122 | 136 |
Total liabilities | 96,485 | 87,305 |
Commitments and contingencies (Note 11) | ||
Stockholders' equity: | ||
Common stock, $0.01 par value, 120,000,000 shares authorized; 61,987,799 and 60,996,367 shares issued and outstanding at March 31, 2021 and December 31, 2020, respectively | 619 | 610 |
Additional paid-in capital | 842,477 | 825,794 |
Accumulated other comprehensive loss | (75) | (42) |
Accumulated deficit | (363,022) | (322,843) |
Total stockholders' equity | 479,999 | 503,519 |
Total liabilities and stockholders' equity | 576,484 | 590,824 |
Series A Convertible Preferred Shares [Member] | ||
Stockholders' equity: | ||
Preferred stock | 0 | 0 |
Series B Convertible Preferred Shares [Member] | ||
Stockholders' equity: | ||
Preferred stock | $ 0 | $ 0 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - $ / shares | Mar. 31, 2021 | Dec. 31, 2020 |
Stockholders' 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) | 61,987,799 | 60,996,367 |
Common stock, shares outstanding (in shares) | 61,987,799 | 60,996,367 |
Series A Convertible Preferred Shares [Member] | ||
Stockholders' 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 Shares [Member] | ||
Stockholders' 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 | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Consolidated Statements of Operations [Abstract] | ||
Revenue | $ 0 | $ 0 |
Operating expenses: | ||
Research and development | 28,542 | 16,957 |
General and administrative | 10,680 | 7,163 |
Total operating expenses | 39,222 | 24,120 |
Loss from operations | (39,222) | (24,120) |
Research and development incentives | 500 | 0 |
Interest expense | (1,729) | (1,573) |
Interest and other income net | 911 | 967 |
(Amortization of premium) accretion of discount on investments - net | (639) | 62 |
Net loss | $ (40,179) | $ (24,664) |
Net loss per share attributable to common stockholders - basic (in dollars per share) | $ (0.65) | $ (0.45) |
Net loss per share attributable to common stockholders - diluted (in dollars per share) | $ (0.65) | $ (0.45) |
Weighted-average common shares outstanding - basic (in shares) | 61,574,405 | 54,883,120 |
Weighted-average common shares outstanding - diluted (in shares) | 61,574,405 | 54,883,120 |
Consolidated Statements of Comp
Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Consolidated Statements of Comprehensive Loss [Abstract] | ||
Net loss | $ (40,179) | $ (24,664) |
Other comprehensive loss | ||
Net unrealized loss on investments | (33) | (95) |
Total comprehensive loss | $ (40,212) | $ (24,759) |
Consolidated Statements of Stoc
Consolidated Statements of Stockholders' Equity - USD ($) $ in Thousands | Common Stock [Member] | Treasury Stock [Member] | Additional Paid-In Capital [Member] | Accumulated Other Comprehensive Loss [Member] | Accumulated Deficit [Member] | Total |
Beginning Balance at Dec. 31, 2019 | $ 548 | $ (53) | $ 489,925 | $ 20 | $ (183,143) | $ 307,297 |
Beginning Balance (in shares) at Dec. 31, 2019 | 54,773,061 | |||||
Issuance of common stock pursuant to exercise of stock options | $ 3 | 0 | (3) | 0 | 0 | 0 |
Issuance of common stock pursuant to exercise of stock options (in shares) | 386,974 | |||||
Share repurchase | $ 0 | 0 | (72) | 0 | 0 | (72) |
Share repurchase (in shares) | (3,000) | |||||
Sale of treasury stock | $ 0 | 53 | 0 | 0 | 0 | 53 |
Sale of treasury stock (in shares) | 0 | |||||
Issuance of treasury stock pursuant to exercise of stock options | $ 0 | (429) | 0 | 0 | 0 | (429) |
Issuance of treasury stock pursuant to exercise of stock options (in shares) | 0 | |||||
Unrealized comprehensive loss on investments | $ 0 | 0 | 0 | (95) | 0 | (95) |
Share-based compensation | 0 | 0 | 3,961 | 0 | 0 | 3,961 |
Net loss | 0 | 0 | 0 | 0 | (24,664) | (24,664) |
Ending Balance at Mar. 31, 2020 | $ 551 | (429) | 493,811 | (75) | (207,807) | 286,051 |
Ending Balance (in shares) at Mar. 31, 2020 | 55,157,035 | |||||
Beginning Balance at Dec. 31, 2020 | $ 610 | 0 | 825,794 | (42) | (322,843) | 503,519 |
Beginning Balance (in shares) at Dec. 31, 2020 | 60,996,367 | |||||
Issuance of common stock pursuant to exercise of stock options | $ 9 | 0 | 8,783 | 0 | 0 | 8,792 |
Issuance of common stock pursuant to exercise of stock options (in shares) | 991,432 | |||||
Unrealized comprehensive loss on investments | $ 0 | 0 | 0 | (33) | 0 | (33) |
Share-based compensation | 0 | 0 | 7,900 | 0 | 0 | 7,900 |
Net loss | 0 | 0 | 0 | 0 | (40,179) | (40,179) |
Ending Balance at Mar. 31, 2021 | $ 619 | $ 0 | $ 842,477 | $ (75) | $ (363,022) | $ 479,999 |
Ending Balance (in shares) at Mar. 31, 2021 | 61,987,799 |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Operating Activities: | ||
Net loss | $ (40,179) | $ (24,664) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Accretion of discount on convertible notes | 589 | 844 |
Depreciation and amortization expense | 674 | 118 |
Write down of property and equipment | 0 | 63 |
Stock-based compensation | 7,900 | 3,961 |
Accretion of discount (amortization of premium) on investments, net | 620 | (63) |
Changes in operating assets and liabilities: | ||
Prepaid expenses and other assets | (842) | (271) |
Accounts payable and accrued expenses | 7,047 | (1,932) |
Operating lease liabilities | (15) | (452) |
Finance lease liability | (63) | 151 |
Other long term liabilities | (14) | 0 |
Net cash used in operating activities | (24,283) | (22,245) |
Investing activities: | ||
Purchases of investments | (103,815) | (23,481) |
Proceeds from maturities of investments | 74,969 | 56,805 |
Payments made to acquire right of use asset | 0 | (530) |
Purchases of property and equipment | (328) | (5,207) |
Purchases of internal use software | 0 | (88) |
Net cash (used in) provided by investing activities | (29,174) | 27,499 |
Financing activities: | ||
Issuance of common stock, pursuant to exercise of stock options | 8,792 | 0 |
Common stock repurchase | 0 | (72) |
Proceeds from sale of treasury stock | 0 | 53 |
Convertible notes refinancing costs to the lender | 0 | (98) |
Net cash provided by (used in) financing activities | 8,792 | (117) |
Net change in cash, cash equivalents and restricted cash | (44,665) | 5,137 |
Cash, cash equivalents and restricted cash at beginning of period | 298,666 | 186,908 |
Cash, cash equivalents and restricted cash at end of period | 254,001 | 192,045 |
Supplemental disclosure of non-cash financing and investing activities: | ||
Accrued purchases of property and equipment | 337 | 2,796 |
Accrued purchases of internal use software | 311 | 174 |
Retirement of treasury stock | 0 | 72 |
Witholding tax payable on shares witheld in treasury stock | 0 | 429 |
Unrealized loss on investments | (33) | (95) |
Finance lease right of use asset and lease liability | 0 | 20,179 |
Reclassification of construction in process to finance right of use asset | 325 | 26,465 |
Supplemental cash flow information: | ||
Cash paid for interest | $ 1,347 | $ 1,495 |
Nature of Business
Nature of Business | 3 Months Ended |
Mar. 31, 2021 | |
Nature of Business [Abstract] | |
Nature of Business | 1. Nature of Business Rocket Pharmaceuticals, Inc. (“Rocket” or the “Company”) is a clinical-stage, multi-platform biotechnology company focused on the development of first, only and best-in-class gene therapies, with direct on-target mechanism of action and clear clinical endpoints, for rare and devastating diseases. Rocket has four ex vivo ” in vivo |
Risks and Liquidity
Risks and Liquidity | 3 Months Ended |
Mar. 31, 2021 | |
Risks and Liquidity [Abstract] | |
Risks and Liquidity | 2. Risks and Liquidity T he Company has not generated any revenue and has incurred losses since inception. The Company’s operations are subject to certain risks and uncertainties, including, among others, uncertainty of drug candidate development, technological uncertainty, uncertainty regarding patents and proprietary rights, the absence of 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 companie 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 as of March 31, 2021. As of March 31, 2021, the Company ha s of cash, cash equivalents and investments . During the year ended December 31, 2020, the Company received net proceeds of $ million from a public offering of shares of common stock. Rocket expects such resources will be sufficient to fund its operating expenses and capital expenditure requirements into the second half of 2023. In April 2021, the Company called for the redemption of the remaining $ million principal balance of its 2022 Convertible Senior Notes due 2022 which were converted into common stock (See Notes 7 and 16). 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. |
Basis of Presentation, Principl
Basis of Presentation, Principles of Consolidation and Summary of Significant Accounting Policies | 3 Months Ended |
Mar. 31, 2021 | |
Basis of Presentation, Principles of Consolidation and Summary of Significant Accounting Policies [Abstract] | |
Basis of Presentation, Principles of Consolidation and Summary of Significant Accounting Policies | 3. Basis of Presentation, Principles of Consolidation and Summary of Significant Accounting Policies Basis of Presentation The accompanying unaudited interim consolidated financial statements should be read in conjunction with the Company’s consolidated financial statements for the year ended December included in the Annual Report on Form -K filed with the Securities and Exchange Commission (“SEC”) on March (“ Form -K”). The unaudited interim consolidated financial statements have been prepared on the same basis as the audited annual financial statements and, in the opinion of management, reflect all adjustments, which include only normal recurring adjustments, necessary for the fair statement of the Company’s consolidated financial position as of March and the results of its operations and its cash flows for the months ended March The financial data and other information disclosed in these consolidated notes related to the months ended March and are unaudited. The results for the months ended March are not necessarily indicative of results to be expected for the year ending December and any other interim periods or any future year or period. 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 (“U.S. GAAP”). All intercompany accounts have been eliminated in consolidation. Use of Estimates The preparation of the consolidated financial statements in conformity with U.S. 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 (“R&D”) 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 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 finance and operating leases (see Note “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: March 31, 2021 December 31 2020 Cash and cash equivalents $ 252,540 $ 297,098 Restricted cash 1,461 1,568 $ 254,001 $ 298,666 Significant Accounting Policies The significant accounting policies used in the preparation of these consolidated financial statements for the months ended March are consistent with those disclosed in Note to the consolidated financial statements in the Form -K, except as noted below. 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 three months ended March 31, 2021 and 2020, respectively, the Company recorded research and development incentive income of $0.5 million and $0, respectively. 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 for the three months ended March 31, 2021 or are expected to have a significant effect on the consolidated financial statements |
Fair Value of Financial Instrum
Fair Value of Financial Instruments | 3 Months Ended |
Mar. 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 March 31 2021 Level 1 Level 2 Level 3 Total Assets: Cash and cash equivalents: Money market mutual funds $ 235,899 $ - $ - $ 235,899 235,899 - - 235,899 Investments: United States Treasury securities 107,566 - - 107,566 Corporate Bonds - 100,247 - 100,247 Municipal Bonds - 6,000 - 6,000 107,566 106,247 - 213,813 $ 343,465 $ 106,247 $ - $ 449,712 Fair Value Measurements as of December 31, 2020 Using: Level 1 Level 2 Level 3 Total Assets: Cash and 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 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 Government, Corporate, Municipal and Agency Bonds as Level assets as these assets are not traded in an active market and have been valued through a -party pricing service based on quoted prices for similar assets. The fair value of the Convertible Notes and the Convertible Notes as of March was and , respectively (see Note . |
Property and Equipment, Net
Property and Equipment, Net | 3 Months Ended |
Mar. 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: March 31, 2021 December 31, 2020 Laboratory equipment $ 8,770 $ 7,807 Machinery and equipment 9,973 9,933 Computer equipment 218 218 Furniture and fixtures 1,867 1,880 Leasehold improvements 29 29 Internal use software 1,696 1,385 22,553 21,252 Less: accumulated depreciation and amortization (2,720 ) (2,046 ) $ 19,833 $ 19,206 During the three months ended March 31, 2021 and 2020, the Company recognized $0.7 million and $0.1 million of depreciation and amortization expense, respectively. |
Accounts Payable and Accrued Ex
Accounts Payable and Accrued Expenses | 3 Months Ended |
Mar. 31, 2021 | |
Accounts Payable and Accrued Expenses [Abstract] | |
Accounts Payable and Accrued Expenses | 6. Accounts Payable and Accrued Expenses At March 31, 2021 and December 31, 2020, the Company’s accounts payable and accrued expenses consisted of the following: March 31, December 31, 2021 2020 Research and development $ 22,675 $ 14,962 Property and equipment 337 1,456 Right of use asset 325 - Employee compensation 6,187 4,875 Accrued interest 449 1,122 Government grant payable 597 590 Professional fees 1,338 1,332 Internal use software 311 300 Other 949 835 $ 33,168 $ 25,472 |
Convertibles Notes
Convertibles Notes | 3 Months Ended |
Mar. 31, 2021 | |
Convertibles Notes [Abstract] | |
Convertibles Notes | 7. Convertibles Notes 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 principal amount of $52.0 million, (the “2021 Convertible Notes”). The 2021 Convertible Notes were issued in 2016 and were scheduled to mature on August 1, 2021 (the “Maturity Date”). The 2021 Convertible Notes are unsecured and accrue interest at a rate of 5.75% per annum and interest is payable semi-annually on February 1 and August 1 of each year. Each holder of the 2021 Convertible Notes (“Holder”) has 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. The Conversion Rate is subject to adjustment from time to time upon the occurrence of certain events, including the issuance of stock dividends and payment of cash dividends. The Company, at its option, may redeem for cash all or any portion of the 2021 Convertible Notes if the last reported sale price of a share of the Company’s common stock is equal to or greater than 200% of the Conversion Rate for the 2021 Convertible Notes then in effect for at least 20 trading days (whether or not consecutive) during any 30 consecutive trading day period (including the last trading day of such period) ending within the five The 2021 Convertible Notes are considered a hybrid financial instrument consisting of a fixed interest rate “host” and various embedded features that required evaluation as potential embedded derivatives under FASB ASC 815, Derivatives and Hedging 2022 Convertible Notes On February 20, 2020 and , August 2022 Additionally, the Company repurchased shares of its common stock that have been retired for an aggregate amount of $ from certain holders of the Convertible Notes participating in the exchange transactions in privately negotiated, private transactions. Also pursuant to the Exchange Agreements, on , the Company exchanged $ million aggregate principal amount of the Convertible Notes for (a) $ million aggregate principal amount of its newly issued Convertible Senior Notes due (an exchange ratio equal to Convertible Notes per exchanged Convertible Notes) and (b) approximately $ to pay the accrued and unpaid interest on the exchanged Convertible Notes from, and including, , to, but excluding, the closing date of the exchange transaction, adjusted to take into account the unearned accrued interest on the Convertible Notes from, and including, , to, but excluding, the closing date of the exchange transaction. The Convertible Notes were issued in a private placement exempt from registration in reliance on Section (a)( ) of the Securities Act of , as amended (the “Securities Act”). The Convertible Notes issued in the exchange transaction were issued as additional notes pursuant to the Indenture, dated as of , between the Company and Wilmington Trust, National Association, as trustee, as supplemented by the Supplemental Indenture, dated as of , governing the Convertible Notes. The conversion rate for the 2022 Convertible Notes is initially 31.1876 shares of the Company’s common stock per 1.00 principal amount of 2022 Convertible Notes, which is equivalent to an initial conversion price of approximately $32.06 per share of common stock and is subject to adjustment under the terms of the 2022 Convertible Notes. The Company may redeem for cash all or any portion of the 2022 Convertible Notes, at its option, if the last reported sale price of its common stock is equal to or greater than 130% of the conversion price then in effect for at least 20 trading days (whether or not consecutive) during any 30 consecutive trading day period (including the last trading day of such period) ending within the five 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 balance of the 2022 Convertible Notes (See Note 16) as the Company’s stock price traded above the Conversion Rate for at least 20 trading days during a 30-day consecutive trading period. The table below summarizes the carrying value of the 2021 Convertible Notes as of March 31, 2021 and December 31, 2020: March 31, 2021 December 31, 2020 Principal amount $ 5,150 $ 5,150 Discount (164 ) (275 ) Carrying value $ 4,986 $ 4,875 Accretion of the 2021 Convertible Notes discount was $0.1 million and $ million for the months ended , respectively The table below summarizes the carrying value of the 2022 Convertible Notes as of March 31, 2021 and December 31, 2020: March 31, 2021 December 31, 2020 Principal amount $ 38,350 $ 38,350 Discount (2,806 ) (3,284 ) Carrying value $ 35,544 $ 35,066 Accretion of the 2022 Convertible Notes discount was $0.5 million and $0.2 million for the three months ended March 31, 2021 and 2020, respectively. |
Stock Based Compensation
Stock Based Compensation | 3 Months Ended |
Mar. 31, 2021 | |
Stock Based Compensation [Abstract] | |
Stock Based Compensation | 8. Stock Based Compensation 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: Three Months Ended March 31 2021 2020 Risk-free interest rate 0.62 % 1.48 % Expected term (in years) 5.87 5.87 Expected volatility 69.41 % 77.75 % Expected dividend yield 0.00 % 0.00 % Exercise price $ 36.99 $ 22.47 Fair value of common stock $ 36.99 $ 22.47 The following table summarizes stock option activity for the three months ended March 31, 2021 under the Second Amended and Restated 2014 Stock Option and Incentive Plan: Number of Shares Weighted Average Exercise Price Weighted Average Contractual Term (Years) Aggregate Intrinsic Value Outstanding as of December 31, 2020 11,050,931 $ 9.10 6.55 $ 504,079 Granted 916,575 60.95 10.64 Exercised (991,432 ) 8.31 51,892 Cancelled (83,244 ) 31.82 Outstanding as of March 31, 2021 10,892,830 $ 13.42 6.57 $ 349,406 Options vested and exercisable as of March 31, 2021 7,856,137 $ 5.70 5.55 $ 300,452 Options unvested as of March 31, 2021 3,036,693 $ 33.17 9.20 The weighted average grant-date fair value per share of stock options granted during the three months ended March 31, 2021 and 2020 was $36.99 and $15.03, respectively. The total fair value of options vested during the three months ended March 31, 2021 and 2020 was $8.3 million and $17.3 million, respectively. Stock-Based Compensation Stock-based compensation expense recognized by award type was as follows: Three Months Ended March 31, 2021 2020 Stock options $ 7,826 $ 3,961 Restricted stock units 74 - Total share based compensation expense $ 7,900 $ 3,961 Stock-based compensation expense by classification included within the consolidated statements of operations and comprehensive loss was as follows : Three Months Ended March 31, 2021 2020 Research and development $ 2,916 $ 1,735 General and administrative 4,984 2,226 Total share based compensation expense $ 7,900 $ 3,961 As of March 31, 2021, the Company had an aggregate of $55.7 million of unrecognized stock-based compensation expense, which is expected to be recognized over the weighted average period of 3.01 years. Restricted Stock The following table summarizes our RSU activity for the three months ended March 31, 2021: Number of Shares Weighted Average Grant Date Fair Value Unvested as of December 31 2020 20,000 $ 25.06 Granted 3,500 62.32 Vested - - Forfeited - - Unvested as of March 31 2021 23,500 30.61 |
Stockholders' Equity
Stockholders' Equity | 3 Months Ended |
Mar. 31, 2021 | |
Stockholders' Equity [Abstract] | |
Stockholders' Equity | 9. Stockholders’ Equity The Company had warrants outstanding as of both March 31, 2021 and December 31, 2020, convertible into 7,051 and 603,386 shares of common stock at an exercise price of $24.82 and $57.11 per share, respectively which will expire on June 28, 2023 and December 21, 2030, respectively. |
Net Loss Per Share
Net Loss Per Share | 3 Months Ended |
Mar. 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: Three Months Ended March 31, 2021 2020 Numerator: Net loss attributable to common stockholders $ (40,179 ) $ (24,664 ) Denominator: Weighted-average common shares outstanding - basic and diluted 61,574,405 54,883,120 Net loss per share attributable to common stockholders - basic and diluted $ (0.65 ) $ (0.45 ) 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: Three Months Ended March 31, 2021 2020 Shares issuable upon conversion of the 2021 Convertible Notes 160,536 394,327 Shares issuable upon conversion of the 2022 Convertible Notes 1,195,449 1,226,621 Warrants exercisable for common shares 610,437 14,102 Options to purchase common shares 10,892,830 10,538,890 12,859,252 12,173,940 |
Commitments and Contingencies
Commitments and Contingencies | 3 Months Ended |
Mar. 31, 2021 | |
Commitments and Contingencies [Abstract] | |
Commitments and Contingencies | 11. Commitments and Contingencies The Company determines if an arrangement is a lease at inception. Operating and finance leases are presented in the Company’s consolidated balance sheet as right-of-use assets from leases, current lease liabilities and long-term lease liabilities. Certain of the Company’s lease agreements contain renewal options; however, the Company does not recognize right-of-use assets or lease liabilities for renewal periods unless it is determined that the Company is reasonably certain of renewing the lease at inception or when a triggering event occurs. As the Company’s leases do not provide an implicit rate, the Company estimated the incremental borrowing rate in calculating the present value of the lease payments using an estimate of the Company’s collateralized borrowing rate for debt with a similar term. The Company has utilized its incremental borrowing rate based on the long-term borrowing costs of comparable companies in the biotechnology industry. Since the Company elected to account for each lease component and its associated non-lease components as a single combined lease component, all contract consideration was allocated to the combined lease component. Some of the Company’s lease agreements contain rent escalation clauses (including index-based escalations). The Company recognizes the minimum rental expense on a straight-line basis based on the fixed components of a lease arrangement. The Company will amortize this expense over the term of the lease beginning with the lease commencement date. Variable lease components represent amounts that are not fixed in nature and are not tied to an index or rate and are recognized as incurred. 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. 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 NJ Lease Agreement is estimated to be approximately $29.3 million over the 15-year term of the NJ Lease Agreement. 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 March 31, 2021 and December 31, 2020. 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 in March 2020, 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 o f . During the months ended , the Company reclassified an additional $ million bringing the aggregate reclassification to $ million of construction costs in progress as part of the right of use asset Interest associated with the financing lease was $ million and $ million for the months ended , respectively and is recorded as interest expense on the consolidated statements of operations. Operating Leases 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”) with a bank for $0.9 million which expired on June 30, 2020 and renewed automatically for a one-year period until its expiration date of July 31, 2021. 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 down to $0.8 million. The Empire LOC serves as the Company’s security deposit on the lease in which the landlord is the beneficiary. The Company has accounted for the ESB Lease Amendment as a modification to the ESB Lease Agreement. The Company remeasured 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 March 31, 2021 and December 31, 2020. This security deposit was reduced to $0.8 million in March 2021 in connection with the ESB Lease Amendment. 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 months ended . Rental income is netted against rent expense in the consolidated statement of operations. Rent expense was $0.3 million for the three months ended . The total restricted cash balance for the Company’s operating and finance leases at and was $ million and $ million, respectively. Lease cost March 31, 2021 Operating lease cost $ 196 Finance lease cost Amortization of right of use assets 535 Interest on lease liablities 459 Total lease cost $ 1,190 The following table summarizes the maturity of the Company’s operating and finance lease liabilities on an undiscounted cash flow basis and a reconciliation to the operating and finance lease liabilities as of March 31, 2021: Maturity of operating lease liabilities March 31, 2021 2021 544 2022 860 2023 488 2024 208 Total lease payments $ 2,100 Less: interest (118 ) Total operating lease liabilities $ 1,982 Maturity of finance lease liability March 31, 2021 2021 1,235 2022 1,689 2023 1,736 2024 1,791 Thereafter 48,768 Total lease payments $ 55,219 Less: interest (34,536 ) Total finance lease liability $ 20,683 Leases March 31, 2021 Operating right-of-use assets $ 1,787 Operating current lease liabilities 683 Operating noncurrent lease liabilities 1,299 Total operating lease liabilities $ 1,982 Finance right-of-use assets $ 50,311 Finance current lease liability 1,655 Finance noncurrent lease liability 19,028 Total finance lease liability $ 20,683 Other information Cash paid for amounts included in the measurement of lease liabilities: Operating cash flows from operating leases $ 226 Cash flows from finance lease $ 408 Weighted-average remaining lease term - operating leases 2.7 Weighted-average remaining lease term - finance lease 23.4 Weighted-average discount rate - operating leases 4.90 % Weighted-average discount rate - finance lease 8.96 % Litigation From time to time, the Company may be subject to other 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 | 3 Months Ended |
Mar. 31, 2021 | |
Agreements Related to Intellectual Property [Abstract] | |
Agreements Related to Intellectual Property | 12. Agreements Related to Intellectual Property The Company 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. |
CIRM Grants
CIRM Grants | 3 Months Ended |
Mar. 31, 2021 | |
CIRM Grants [Abstract] | |
CIRM Grants | 13. 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 gene therapy for LAD-I. Proceeds from the grant will help fund clinical trial costs as well as manufactured drug product for Phase I/II 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 , 2020 , the Company met the next CIRM milestone and recorded a receivable, included in prepaid and other assets in the consolidated balance sheet, and a reduction of research and development expenses of $ million 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 lentiviral vector (LVV)-based gene therapy, RP-L401, for the treatment of IMO. The Company received a $1.1 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. The Company recorded a reduction of research and development expense of $0.1 million for the three months ended March 31, 2021. |
Related Party Transactions
Related Party Transactions | 3 Months Ended |
Mar. 31, 2021 | |
Related Party Transactions [Abstract] | |
Related Party Transactions | 14. Related Party Transactions During April 2018 , the and 2020 , r In , 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 options with a fair value of $ million. In , the Company entered into a consulting agreement with the spouse of 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. In December 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. There were no warrants issued for the three months ended March 31, 2021. |
401(k) Savings Plan
401(k) Savings Plan | 3 Months Ended |
Mar. 31, 2021 | |
401(k) Savings Plan [Abstract] | |
401(k) Savings Plan | 15. 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 the safe harbor match of 4% of employee contributions to the Plan, subject to certain limitations. The Company’s matching contribution for the three months ended March 31, 2021 and 2020 $ million, respectively. |
Subsequent Events
Subsequent Events | 3 Months Ended |
Mar. 31, 2021 | |
Subsequent Events [Abstract] | |
Subsequent Events | 16. Subsequent Events On April 26, 2021, the Company completed a full redemption of its 2022 Convertible Notes prior to the redemption date. Holders of approximately $38.4 remaining million principal amount of the 2022 Convertible Notes converted such notes in accordance with the terms of the governing indenture into approximately 1.3 million shares of the Company’s common stock and cash in lieu of fractional shares. Following the redemption, approximately $5.2 million aggregate principal amount of the 2021 Convertible Notes remain outstanding. |
Basis of Presentation, Princi_2
Basis of Presentation, Principles of Consolidation and Summary of Significant Accounting Policies (Policies) | 3 Months Ended |
Mar. 31, 2021 | |
Basis of Presentation, Principles of Consolidation and Summary of Significant Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation The accompanying unaudited interim consolidated financial statements should be read in conjunction with the Company’s consolidated financial statements for the year ended December included in the Annual Report on Form -K filed with the Securities and Exchange Commission (“SEC”) on March (“ Form -K”). The unaudited interim consolidated financial statements have been prepared on the same basis as the audited annual financial statements and, in the opinion of management, reflect all adjustments, which include only normal recurring adjustments, necessary for the fair statement of the Company’s consolidated financial position as of March and the results of its operations and its cash flows for the months ended March The financial data and other information disclosed in these consolidated notes related to the months ended March and are unaudited. The results for the months ended March are not necessarily indicative of results to be expected for the year ending December and any other interim periods or any future year or period. |
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 (“U.S. 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 U.S. 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 (“R&D”) 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 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 finance and operating leases (see Note “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: March 31, 2021 December 31 2020 Cash and cash equivalents $ 252,540 $ 297,098 Restricted cash 1,461 1,568 $ 254,001 $ 298,666 |
Significant Accounting Policies | Significant Accounting Policies The significant accounting policies used in the preparation of these consolidated financial statements for the months ended March are consistent with those disclosed in Note to the consolidated financial statements in the Form -K, except as noted below. |
NYS Life Sciences Research and Development Tax Credit | 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 three months ended March 31, 2021 and 2020, respectively, the Company recorded research and development incentive income of $0.5 million and $0, respectively. |
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 for the three months ended March 31, 2021 or are expected to have a significant effect on the consolidated financial statements |
Basis of Presentation, Princi_3
Basis of Presentation, Principles of Consolidation and Summary of Significant Accounting Policies (Tables) | 3 Months Ended |
Mar. 31, 2021 | |
Basis of Presentation, Principles of Consolidation and 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 finance and operating leases (see Note “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: March 31, 2021 December 31 2020 Cash and cash equivalents $ 252,540 $ 297,098 Restricted cash 1,461 1,568 $ 254,001 $ 298,666 |
Fair Value of Financial Instr_2
Fair Value of Financial Instruments (Tables) | 3 Months Ended |
Mar. 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 March 31 2021 Level 1 Level 2 Level 3 Total Assets: Cash and cash equivalents: Money market mutual funds $ 235,899 $ - $ - $ 235,899 235,899 - - 235,899 Investments: United States Treasury securities 107,566 - - 107,566 Corporate Bonds - 100,247 - 100,247 Municipal Bonds - 6,000 - 6,000 107,566 106,247 - 213,813 $ 343,465 $ 106,247 $ - $ 449,712 Fair Value Measurements as of December 31, 2020 Using: Level 1 Level 2 Level 3 Total Assets: Cash and 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) | 3 Months Ended |
Mar. 31, 2021 | |
Property and Equipment, Net [Abstract] | |
Property and Equipment | The Company’s property and equipment consisted of the following: March 31, 2021 December 31, 2020 Laboratory equipment $ 8,770 $ 7,807 Machinery and equipment 9,973 9,933 Computer equipment 218 218 Furniture and fixtures 1,867 1,880 Leasehold improvements 29 29 Internal use software 1,696 1,385 22,553 21,252 Less: accumulated depreciation and amortization (2,720 ) (2,046 ) $ 19,833 $ 19,206 |
Accounts Payable and Accrued _2
Accounts Payable and Accrued Expenses (Tables) | 3 Months Ended |
Mar. 31, 2021 | |
Accounts Payable and Accrued Expenses [Abstract] | |
Accounts Payable and Accrued Expenses | At March 31, 2021 and December 31, 2020, the Company’s accounts payable and accrued expenses consisted of the following: March 31, December 31, 2021 2020 Research and development $ 22,675 $ 14,962 Property and equipment 337 1,456 Right of use asset 325 - Employee compensation 6,187 4,875 Accrued interest 449 1,122 Government grant payable 597 590 Professional fees 1,338 1,332 Internal use software 311 300 Other 949 835 $ 33,168 $ 25,472 |
Convertibles Notes (Tables)
Convertibles Notes (Tables) | 3 Months Ended |
Mar. 31, 2021 | |
2021 Convertible Notes [Member] | |
Debt Instrument [Line Items] | |
Carrying Value of Convertible Notes | The table below summarizes the carrying value of the 2021 Convertible Notes as of March 31, 2021 and December 31, 2020: March 31, 2021 December 31, 2020 Principal amount $ 5,150 $ 5,150 Discount (164 ) (275 ) Carrying value $ 4,986 $ 4,875 |
2022 Convertible Notes [Member] | |
Debt Instrument [Line Items] | |
Carrying Value of Convertible Notes | The table below summarizes the carrying value of the 2022 Convertible Notes as of March 31, 2021 and December 31, 2020: March 31, 2021 December 31, 2020 Principal amount $ 38,350 $ 38,350 Discount (2,806 ) (3,284 ) Carrying value $ 35,544 $ 35,066 |
Stock Based Compensation (Table
Stock Based Compensation (Tables) | 3 Months Ended |
Mar. 31, 2021 | |
Stock Based Compensation [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: Three Months Ended March 31 2021 2020 Risk-free interest rate 0.62 % 1.48 % Expected term (in years) 5.87 5.87 Expected volatility 69.41 % 77.75 % Expected dividend yield 0.00 % 0.00 % Exercise price $ 36.99 $ 22.47 Fair value of common stock $ 36.99 $ 22.47 |
Stock Option Activity | The following table summarizes stock option activity for the three months ended March 31, 2021 under the Second Amended and Restated 2014 Stock Option and Incentive Plan: Number of Shares Weighted Average Exercise Price Weighted Average Contractual Term (Years) Aggregate Intrinsic Value Outstanding as of December 31, 2020 11,050,931 $ 9.10 6.55 $ 504,079 Granted 916,575 60.95 10.64 Exercised (991,432 ) 8.31 51,892 Cancelled (83,244 ) 31.82 Outstanding as of March 31, 2021 10,892,830 $ 13.42 6.57 $ 349,406 Options vested and exercisable as of March 31, 2021 7,856,137 $ 5.70 5.55 $ 300,452 Options unvested as of March 31, 2021 3,036,693 $ 33.17 9.20 |
Stock-Based Compensation Expense by Award Type | Stock-based compensation expense recognized by award type was as follows: Three Months Ended March 31, 2021 2020 Stock options $ 7,826 $ 3,961 Restricted stock units 74 - Total share based compensation expense $ 7,900 $ 3,961 |
Stock-Based Compensation Expense | Stock-based compensation expense by classification included within the consolidated statements of operations and comprehensive loss was as follows : Three Months Ended March 31, 2021 2020 Research and development $ 2,916 $ 1,735 General and administrative 4,984 2,226 Total share based compensation expense $ 7,900 $ 3,961 |
RSU Activity | The following table summarizes our RSU activity for the three months ended March 31, 2021: Number of Shares Weighted Average Grant Date Fair Value Unvested as of December 31 2020 20,000 $ 25.06 Granted 3,500 62.32 Vested - - Forfeited - - Unvested as of March 31 2021 23,500 30.61 |
Net Loss Per Share (Tables)
Net Loss Per Share (Tables) | 3 Months Ended |
Mar. 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: Three Months Ended March 31, 2021 2020 Numerator: Net loss attributable to common stockholders $ (40,179 ) $ (24,664 ) Denominator: Weighted-average common shares outstanding - basic and diluted 61,574,405 54,883,120 Net loss per share attributable to common stockholders - basic and diluted $ (0.65 ) $ (0.45 ) |
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: Three Months Ended March 31, 2021 2020 Shares issuable upon conversion of the 2021 Convertible Notes 160,536 394,327 Shares issuable upon conversion of the 2022 Convertible Notes 1,195,449 1,226,621 Warrants exercisable for common shares 610,437 14,102 Options to purchase common shares 10,892,830 10,538,890 12,859,252 12,173,940 |
Commitments and Contingencies (
Commitments and Contingencies (Tables) | 3 Months Ended |
Mar. 31, 2021 | |
Commitments and Contingencies [Abstract] | |
Lease Cost | Lease cost March 31, 2021 Operating lease cost $ 196 Finance lease cost Amortization of right of use assets 535 Interest on lease liablities 459 Total lease cost $ 1,190 |
Maturities of Operating Lease Liabilities | The following table summarizes the maturity of the Company’s operating and finance lease liabilities on an undiscounted cash flow basis and a reconciliation to the operating and finance lease liabilities as of March 31, 2021: Maturity of operating lease liabilities March 31, 2021 2021 544 2022 860 2023 488 2024 208 Total lease payments $ 2,100 Less: interest (118 ) Total operating lease liabilities $ 1,982 |
Maturity of Finance Lease Liability | Maturity of finance lease liability March 31, 2021 2021 1,235 2022 1,689 2023 1,736 2024 1,791 Thereafter 48,768 Total lease payments $ 55,219 Less: interest (34,536 ) Total finance lease liability $ 20,683 |
Balance Sheet Information Related to Leases | Leases March 31, 2021 Operating right-of-use assets $ 1,787 Operating current lease liabilities 683 Operating noncurrent lease liabilities 1,299 Total operating lease liabilities $ 1,982 Finance right-of-use assets $ 50,311 Finance current lease liability 1,655 Finance noncurrent lease liability 19,028 Total finance lease liability $ 20,683 |
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 $ 226 Cash flows from finance lease $ 408 Weighted-average remaining lease term - operating leases 2.7 Weighted-average remaining lease term - finance lease 23.4 Weighted-average discount rate - operating leases 4.90 % Weighted-average discount rate - finance lease 8.96 % |
Nature of Business (Details)
Nature of Business (Details) | 3 Months Ended |
Mar. 31, 2021Program | |
Nature of Business [Abstract] | |
Number of clinical-stage programs | 4 |
Risks and Liquidity (Details)
Risks and Liquidity (Details) - USD ($) $ in Thousands | Apr. 26, 2021 | Mar. 31, 2021 | Dec. 31, 2020 | Feb. 20, 2020 |
Risks and Liquidity [Abstract] | ||||
Accumulated deficit | $ (363,022) | $ (322,843) | ||
Cash, cash equivalents and investments | $ 466,400 | |||
2022 Convertible Notes [Member] | ||||
Shareholders' Equity Disclosure [Abstract] | ||||
Convertible senior notes, interest rate, stated percentage | 6.25% | 6.25% | ||
Debt instrument maturity date | Aug. 31, 2022 | |||
2022 Convertible Notes [Member] | Subsequent Event [Member] | ||||
Shareholders' Equity Disclosure [Abstract] | ||||
Redemption of principal balance | $ 38,400 | |||
Convertible senior notes, interest rate, stated percentage | 6.25% | |||
IPO [Member] | Common Stock [Member] | ||||
Shareholders' Equity Disclosure [Abstract] | ||||
Net proceeds, offering amount | $ 280,800 | |||
Common stock shares issued (in shares) | 5,339,286 |
Basis of Presentation, Princi_4
Basis of Presentation, Principles of Consolidation and Summary of Significant Accounting Policies (Details) - USD ($) $ in Thousands | 3 Months Ended | |||
Mar. 31, 2021 | Mar. 31, 2020 | Dec. 31, 2020 | Dec. 31, 2019 | |
Cash, Cash Equivalents and Restricted Cash [Abstract] | ||||
Cash and cash equivalents | $ 252,540 | $ 297,098 | ||
Restricted cash | 1,461 | 1,568 | ||
Total | 254,001 | $ 192,045 | $ 298,666 | $ 186,908 |
NYS Life Sciences Research and Development Tax Credit [Abstract] | ||||
Research and development incentive income | $ 500 | $ 0 |
Fair Value of Financial Instr_3
Fair Value of Financial Instruments (Details) - USD ($) $ in Thousands | Mar. 31, 2021 | Dec. 31, 2020 |
2021 Convertible Notes [Member] | ||
Additional Information in Fair Value Disclosure [Abstract] | ||
Fair value convertible note | $ 8,100 | |
2022 Convertible Notes [Member] | ||
Additional Information in Fair Value Disclosure [Abstract] | ||
Fair value convertible note | 53,100 | |
Recurring [Member] | ||
Assets [Abstract] | ||
Cash and cash equivalents | 235,899 | $ 264,325 |
Investments | 213,813 | 185,621 |
Fair value of financial instruments | 449,712 | 449,946 |
Recurring [Member] | Money Market Mutual Funds [Member] | ||
Assets [Abstract] | ||
Cash and cash equivalents | 235,899 | 193,312 |
Recurring [Member] | United States Treasury Securities [Member] | ||
Assets [Abstract] | ||
Cash and cash equivalents | 62,497 | |
Investments | 107,566 | 112,328 |
Recurring [Member] | Corporate Bonds [Member] | ||
Assets [Abstract] | ||
Cash and cash equivalents | 501 | |
Investments | 100,247 | 63,710 |
Recurring [Member] | Municipal Bonds [Member] | ||
Assets [Abstract] | ||
Investments | 6,000 | 6,000 |
Recurring [Member] | Agency Bonds [Member] | ||
Assets [Abstract] | ||
Cash and cash equivalents | 8,015 | |
Investments | 3,583 | |
Recurring [Member] | Level 1 [Member] | ||
Assets [Abstract] | ||
Cash and cash equivalents | 235,899 | 255,809 |
Investments | 107,566 | 112,328 |
Fair value of financial instruments | 343,465 | 368,137 |
Recurring [Member] | Level 1 [Member] | Money Market Mutual Funds [Member] | ||
Assets [Abstract] | ||
Cash and cash equivalents | 235,899 | 193,312 |
Recurring [Member] | Level 1 [Member] | United States Treasury Securities [Member] | ||
Assets [Abstract] | ||
Cash and cash equivalents | 62,497 | |
Investments | 107,566 | 112,328 |
Recurring [Member] | Level 1 [Member] | Corporate Bonds [Member] | ||
Assets [Abstract] | ||
Cash and cash equivalents | 0 | |
Investments | 0 | 0 |
Recurring [Member] | Level 1 [Member] | Municipal Bonds [Member] | ||
Assets [Abstract] | ||
Investments | 0 | 0 |
Recurring [Member] | Level 1 [Member] | Agency Bonds [Member] | ||
Assets [Abstract] | ||
Cash and cash equivalents | 0 | |
Investments | 0 | |
Recurring [Member] | Level 2 [Member] | ||
Assets [Abstract] | ||
Cash and cash equivalents | 0 | 8,516 |
Investments | 106,247 | 73,293 |
Fair value of financial instruments | 106,247 | 81,809 |
Recurring [Member] | Level 2 [Member] | Money Market Mutual Funds [Member] | ||
Assets [Abstract] | ||
Cash and cash equivalents | 0 | 0 |
Recurring [Member] | Level 2 [Member] | United States Treasury Securities [Member] | ||
Assets [Abstract] | ||
Cash and cash equivalents | 0 | |
Investments | 0 | 0 |
Recurring [Member] | Level 2 [Member] | Corporate Bonds [Member] | ||
Assets [Abstract] | ||
Cash and cash equivalents | 501 | |
Investments | 100,247 | 63,710 |
Recurring [Member] | Level 2 [Member] | Municipal Bonds [Member] | ||
Assets [Abstract] | ||
Investments | 6,000 | 6,000 |
Recurring [Member] | Level 2 [Member] | Agency Bonds [Member] | ||
Assets [Abstract] | ||
Cash and cash equivalents | 8,015 | |
Investments | 3,583 | |
Recurring [Member] | Level 3 [Member] | ||
Assets [Abstract] | ||
Cash and cash equivalents | 0 | 0 |
Investments | 0 | 0 |
Fair value of financial instruments | 0 | 0 |
Recurring [Member] | Level 3 [Member] | Money Market Mutual Funds [Member] | ||
Assets [Abstract] | ||
Cash and cash equivalents | 0 | 0 |
Recurring [Member] | Level 3 [Member] | United States Treasury Securities [Member] | ||
Assets [Abstract] | ||
Cash and cash equivalents | 0 | |
Investments | 0 | 0 |
Recurring [Member] | Level 3 [Member] | Corporate Bonds [Member] | ||
Assets [Abstract] | ||
Cash and cash equivalents | 0 | |
Investments | 0 | 0 |
Recurring [Member] | Level 3 [Member] | Municipal Bonds [Member] | ||
Assets [Abstract] | ||
Investments | $ 0 | 0 |
Recurring [Member] | Level 3 [Member] | Agency Bonds [Member] | ||
Assets [Abstract] | ||
Cash and cash equivalents | 0 | |
Investments | $ 0 |
Property and Equipment, Net (De
Property and Equipment, Net (Details) - USD ($) $ in Thousands | 3 Months Ended | ||
Mar. 31, 2021 | Mar. 31, 2020 | Dec. 31, 2020 | |
Property and Equipment [Abstract] | |||
Property and equipment, gross | $ 22,553 | $ 21,252 | |
Less: accumulated depreciation and amortization | (2,720) | (2,046) | |
Property and equipment, net | 19,833 | 19,206 | |
Depreciation and amortization expense | 674 | $ 118 | |
Laboratory Equipment [Member] | |||
Property and Equipment [Abstract] | |||
Property and equipment, gross | 8,770 | 7,807 | |
Machinery and Equipment [Member] | |||
Property and Equipment [Abstract] | |||
Property and equipment, gross | 9,973 | 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 | 1,867 | 1,880 | |
Leasehold Improvements [Member] | |||
Property and Equipment [Abstract] | |||
Property and equipment, gross | 29 | 29 | |
Internal use Software [Member] | |||
Property and Equipment [Abstract] | |||
Property and equipment, gross | $ 1,696 | $ 1,385 |
Accounts Payable and Accrued _3
Accounts Payable and Accrued Expenses (Details) - USD ($) $ in Thousands | Mar. 31, 2021 | Dec. 31, 2020 |
Accounts Payable and Accrued Expenses [Abstract] | ||
Research and development | $ 22,675 | $ 14,962 |
Property and equipment | 337 | 1,456 |
Right of use asset | 325 | 0 |
Employee compensation | 6,187 | 4,875 |
Accrued interest | 449 | 1,122 |
Government grant payable | 597 | 590 |
Professional fees | 1,338 | 1,332 |
Internal use software | 311 | 300 |
Other | 949 | 835 |
Total | $ 33,168 | $ 25,472 |
Convertibles Notes (Details)
Convertibles Notes (Details) | Apr. 26, 2021USD ($)d | Feb. 20, 2020USD ($)$ / sharesshares | Mar. 31, 2021USD ($)d$ / shares | Mar. 31, 2020USD ($) | Dec. 31, 2020USD ($)shares | Jun. 12, 2020USD ($) | Jan. 04, 2018USD ($) |
Debt [Abstract] | |||||||
Aggregate amount of common stock shares repurchased | $ 72,000 | ||||||
Carrying value of convertible notes [Abstract] | |||||||
Carrying value | $ 35,544,000 | $ 35,066,000 | |||||
Accretion of debt discount | $ 589,000 | 844,000 | |||||
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 | ||||||
Convertible Note exchange | 1 | ||||||
Conversion price (in dollars per share) | $ / shares | $ 32.08 | ||||||
Percentage of common stock conversion price | 200.00% | ||||||
Number of trading days, not consecutive | d | 20 | ||||||
Number of consecutive trading days | d | 30 | ||||||
Number of trading days immediately preceding notice of redemption | 5 days | ||||||
Redemption price percentage | 100.00% | ||||||
Effective interest rate | 15.30% | ||||||
Debt instrument, aggregate principal amount exchanged | $ 39,350,000 | $ 7,500,000 | |||||
Debt instrument, percentage of aggregate outstanding principal | 76.00% | ||||||
Accrued and unpaid interest | $ 100,000 | 11,000,000 | |||||
Number of common stock shares repurchased (in shares) | shares | 3,000 | ||||||
Aggregate amount of common stock shares repurchased | $ 71,670,000 | ||||||
Carrying value of convertible notes [Abstract] | |||||||
Principal amount | $ 5,150,000 | 5,150,000 | $ 7,500,000 | $ 52,000,000 | |||
Discount | (164,000) | (275,000) | |||||
Carrying value | 4,986,000 | 4,875,000 | |||||
Accretion of debt discount | $ 100,000 | 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 | ||||||
Convertible Note exchange | 1 | ||||||
Conversion price (in dollars per share) | $ / shares | $ 32.06 | ||||||
Percentage of common stock conversion price | 130.00% | ||||||
Number of trading days, not consecutive | d | 20 | ||||||
Number of consecutive trading days | d | 30 | ||||||
Number of trading days immediately preceding notice of redemption | 5 days | ||||||
Effective interest rate | 15.30% | ||||||
Conversion of convertible notes into common stock, principal amount | 8,500,000 | ||||||
Conversion of convertible notes into common stock | $ 7,600,000 | ||||||
Convertible Notes converted into shares of common stock (in shares) | shares | 298,562 | ||||||
Carrying value of convertible notes [Abstract] | |||||||
Principal amount | $ 39,350,000 | $ 38,350,000 | $ 38,350,000 | ||||
Discount | (2,806,000) | (3,284,000) | |||||
Carrying value | 35,544,000 | $ 35,066,000 | |||||
Accretion of debt discount | $ 500,000 | $ 200,000 | |||||
2022 Convertible Notes [Member] | Subsequent Event [Member] | |||||||
Debt [Abstract] | |||||||
Convertible senior notes, interest rate, stated percentage | 6.25% | ||||||
Number of trading days, not consecutive | d | 20 | ||||||
Number of consecutive trading days | d | 30 | ||||||
Redemption of principal balance | $ 38,400,000 |
Stock Based Compensation, Share
Stock Based Compensation, Share Option Valuation (Details) - Stock Options [Member] - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 12 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | Dec. 31, 2020 | |
Stock Option Activity [Roll Forward] | |||
Outstanding at beginning of period (in shares) | 11,050,931 | ||
Granted (in shares) | 916,575 | ||
Exercised (in shares) | (991,432) | ||
Cancelled (in shares) | (83,244) | ||
Outstanding at end of period (in shares) | 10,892,830 | 11,050,931 | |
Options vested and exercisable at end of period (in shares) | 7,856,137 | ||
Options unvested at ending of period (in shares) | 3,036,693 | ||
Weighted Average Exercise Price [Abstract] | |||
Outstanding at beginning of period (in dollars per share) | $ 9.10 | ||
Granted (in dollars per share) | 60.95 | ||
Exercised (in dollars per share) | 8.31 | ||
Cancelled (in dollars per share) | 31.82 | ||
Outstanding at end of period (in dollars per share) | 13.42 | $ 9.10 | |
Options vested and exercisable at end of period (in dollars per share) | 5.70 | ||
Options unvested at ending (in dollars per share) | $ 33.17 | ||
Weighted-Average Remaining Contractual Term [Abstract] | |||
Outstanding | 6 years 6 months 25 days | 6 years 6 months 18 days | |
Granted | 10 years 7 months 20 days | ||
Options vested and exercisable | 5 years 6 months 18 days | ||
Options unvested | 9 years 2 months 12 days | ||
Aggregate Intrinsic Value [Abstract] | |||
Outstanding at beginning of period | $ 504,079 | ||
Exercised | 51,892 | ||
Outstanding at end of period | 349,406 | $ 504,079 | |
Options vested and exercisable | $ 300,452 | ||
Weighted average grant date fair value of shares granted (in dollars per share) | $ 36.99 | $ 15.03 | |
Total fair value of options vested | $ 8,300 | $ 17,300 | |
Employees and Directors [Member] | |||
Weighted-Average Assumptions [Abstract] | |||
Risk-free interest rate | 0.62% | 1.48% | |
Expected term | 5 years 10 months 13 days | 5 years 10 months 13 days | |
Expected volatility | 69.41% | 77.75% | |
Expected dividend yield | 0.00% | 0.00% | |
Exercise price (in dollars per share) | $ 36.99 | $ 22.47 | |
Fair value of common stock (in dollars per share) | $ 36.99 | $ 22.47 |
Stock Based Compensation, Stock
Stock Based Compensation, Stock-Based Compensation (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Stock-Based Compensation Expense [Abstract] | ||
Total share based compensation expense | $ 7,900 | $ 3,961 |
Unrecognized share-based compensation cost | $ 55,700 | |
Weighted average period expected to recognize unrecognized share-based compensation cost | 3 years 3 days | |
Research and Development [Member] | ||
Stock-Based Compensation Expense [Abstract] | ||
Total share based compensation expense | $ 2,916 | 1,735 |
General and Administrative [Member] | ||
Stock-Based Compensation Expense [Abstract] | ||
Total share based compensation expense | 4,984 | 2,226 |
Stock Options [Member] | ||
Stock-Based Compensation Expense [Abstract] | ||
Total share based compensation expense | 7,826 | 3,961 |
Restricted Stock Units [Member] | ||
Stock-Based Compensation Expense [Abstract] | ||
Total share based compensation expense | $ 74 | $ 0 |
Stock-Based Awards, Restricted
Stock-Based Awards, Restricted Stock Units (Details) - Restricted Stock Units (RSU) [Member] | 3 Months Ended |
Mar. 31, 2021$ / sharesshares | |
Number of Shares [Roll Forward] | |
Unvested, beginning balance (in shares) | shares | 20,000 |
Granted (in shares) | shares | 3,500 |
Vested (in shares) | shares | 0 |
Forfeited (in shares) | shares | 0 |
Unvested, ending balance (in shares) | shares | 23,500 |
Weighted Average Grant Date Fair Value [Abstract] | |
Unvested, beginning balance (in dollars per share) | $ / shares | $ 25.06 |
Granted (in dollars per share) | $ / shares | 62.32 |
Vested (in dollars per share) | $ / shares | 0 |
Forfeited (in dollars per share) | $ / shares | 0 |
Unvested, ending balance (in dollars per share) | $ / shares | $ 30.61 |
Stockholders' Equity (Details)
Stockholders' Equity (Details) - $ / shares | Mar. 31, 2021 | Dec. 31, 2020 |
Shareholders' Equity Disclosure [Abstract] | ||
Warrants outstanding (in shares) | 7,051 | 603,386 |
Warrant One [Member] | ||
Shareholders' Equity Disclosure [Abstract] | ||
Exercise price of warrants (in dollars per share) | $ 24.82 | |
Warrants expiry date | Jun. 28, 2023 | |
Warrant Two [Member] | ||
Shareholders' Equity Disclosure [Abstract] | ||
Exercise price of warrants (in dollars per share) | $ 57.11 | |
Warrants expiry date | Dec. 21, 2030 |
Net Loss Per Share (Details)
Net Loss Per Share (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Numerator [Abstract] | ||
Net loss attributable to common stockholders | $ (40,179) | $ (24,664) |
Denominator [Abstract] | ||
Weighted-average common shares outstanding - basic (in shares) | 61,574,405 | 54,883,120 |
Weighted-average common shares outstanding - diluted (in shares) | 61,574,405 | 54,883,120 |
Net loss per share attributable to common stockholders - basic (in dollars per share) | $ (0.65) | $ (0.45) |
Net loss per share attributable to common stockholders - diluted (in dollars per share) | $ (0.65) | $ (0.45) |
Weighted Average Number of Shares Outstanding Reconciliation [Abstract] | ||
Warrants exercisable for common shares (in shares) | 610,437 | 14,102 |
Options to purchase common shares (in shares) | 10,892,830 | 10,538,890 |
Weighted average number diluted shares outstanding (in shares) | 12,859,252 | 12,173,940 |
Convertible Notes 2021 [Member] | ||
Weighted Average Number of Shares Outstanding Reconciliation [Abstract] | ||
Shares issuable upon conversion of Convertible Notes (in shares) | 160,536 | 394,327 |
Convertible Notes 2022 [Member] | ||
Weighted Average Number of Shares Outstanding Reconciliation [Abstract] | ||
Shares issuable upon conversion of Convertible Notes (in shares) | 1,195,449 | 1,226,621 |
Commitments and Contingencies,
Commitments and Contingencies, Finance Lease (Details) $ in Thousands | 3 Months Ended | |||
Mar. 31, 2021USD ($)ft²LeaseAgreement | Mar. 31, 2020USD ($) | Dec. 31, 2020USD ($) | Mar. 15, 2020USD ($) | |
Finance Lease [Abstract] | ||||
Total lease payments | $ 55,219 | |||
Cash security deposit | 455 | $ 455 | ||
Finance right-of-use assets | 50,311 | 50,521 | ||
Finance lease, liability | 20,683 | |||
Finance lease, interest expense | $ 459 | |||
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 right-of-use assets | $ 47,700 | |||
Finance lease, liability | 20,200 | |||
Reclassification of construction costs in progress to right-of-use assets | 300 | 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 | 32,400 | |||
Finance lease, interest expense | $ 500 | $ 200 | ||
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 Thousands | Mar. 26, 2021 | Mar. 31, 2021 | Mar. 31, 2020 | Dec. 31, 2020 | Jun. 07, 2018 |
Operating Leases [Abstract] | |||||
Rent expense | $ 300 | $ 300 | |||
Restricted cash | $ 1,461 | $ 1,568 | |||
ESB Lease Agreement [Member] | |||||
Operating Leases [Abstract] | |||||
Term of lease agreement | 3 years | ||||
Letter of credit | $ 900 | ||||
Letter of credit expiration date | Jun. 30, 2020 | ||||
LOC automatic renewal period | 1 year | ||||
Lease expiration date | Jul. 31, 2021 | ||||
Certificate of deposit | $ 800 | 900 | |||
ESB Lease Agreement Amendment [Member] | |||||
Operating Leases [Abstract] | |||||
Letter of credit | 800 | ||||
Lease expiration date | Jun. 30, 2024 | ||||
Operating lease right of use asset, adjustment | $ 1,100 | ||||
Certificate of deposit | $ 800 | ||||
Inotek Lexington Massachusetts Lease Agreement [Member] | |||||
Operating Leases [Abstract] | |||||
Lease expiration date | Feb. 28, 2023 | ||||
Rental income received under sublease agreement | $ 100 | $ 100 | |||
Operating and Finance Leases [Member] | |||||
Operating Leases [Abstract] | |||||
Restricted cash | $ 1,000 | $ 1,100 |
Commitments and Contingencies_3
Commitments and Contingencies, Details of Operating and Finance Lease (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2021 | Dec. 31, 2020 | |
Lease cost [Abstract] | ||
Operating lease cost | $ 196 | |
Finance lease cost [Abstract] | ||
Amortization of right of use assets | 535 | |
Interest on lease liabilities | 459 | |
Total lease cost | 1,190 | |
Maturity of operating lease liabilities [Abstract] | ||
2021 | 544 | |
2022 | 860 | |
2023 | 488 | |
2024 | 208 | |
Total lease payments | 2,100 | |
Less: interest | (118) | |
Total operating lease liabilities | 1,982 | |
Maturity of finance lease liability [Abstract] | ||
2021 | 1,235 | |
2022 | 1,689 | |
2023 | 1,736 | |
2024 | 1,791 | |
Thereafter | 48,768 | |
Total lease payments | 55,219 | |
Less: interest | (34,536) | |
Total finance lease liability | 20,683 | |
Lease assets and liabilities [Abstract] | ||
Operating right-of-use assets | 1,787 | $ 914 |
Operating current lease liabilities | 683 | 626 |
Operating noncurrent lease liabilities | 1,299 | 498 |
Total operating lease liabilities | 1,982 | |
Finance right-of-use assets | 50,311 | 50,521 |
Finance current lease liability | 1,655 | 1,644 |
Finance noncurrent lease liability | 19,028 | $ 18,988 |
Total finance lease liability | 20,683 | |
Cash paid for amounts included in the measurement of lease liabilities [Abstract] | ||
Operating cash flows from operating leases | 226 | |
Cash flows from finance lease | $ 408 | |
Weighted-average remaining lease term - operating leases | 2 years 8 months 12 days | |
Weighted-average remaining lease term - finance lease | 23 years 4 months 24 days | |
Weighted-average discount rate - operating leases | 4.90% | |
Weighted-average discount rate - finance lease | 8.96% |
CIRM Grants (Details)
CIRM Grants (Details) $ in Millions | Apr. 01, 2021USD ($) | Jan. 04, 2021USD ($) | Dec. 31, 2019USD ($)Grant | Mar. 31, 2021USD ($) | Dec. 31, 2020USD ($) | Nov. 12, 2020USD ($) | Apr. 30, 2019USD ($) |
LAD-I CIRM Grant [Member] | |||||||
CIRM Grants [Abstract] | |||||||
Grant award for clinical development support | $ 6.5 | ||||||
Number of grants | Grant | 2 | ||||||
Amount received from grant for eligible costs incurred | $ 1.2 | ||||||
Grant receivable included in prepaid and other assets | $ 1 | $ 1.1 | |||||
Milestone payments received | $ 1.1 | ||||||
IMO CIRM Grant [Member] | |||||||
CIRM Grants [Abstract] | |||||||
Grant award for clinical development support | $ 3.7 | ||||||
Amount received from grant for eligible costs incurred | $ 1.1 | ||||||
Grant receivable included in prepaid and other assets | $ 0.1 | $ 0.9 | |||||
Subsequent Event [Member] | LAD-I CIRM Grant [Member] | |||||||
CIRM Grants [Abstract] | |||||||
Milestone payments received | $ 1 |
Related Party Transactions (Det
Related Party Transactions (Details) - USD ($) $ in Thousands | 1 Months Ended | 3 Months Ended | |||
Oct. 31, 2020 | Mar. 31, 2021 | Mar. 31, 2020 | Dec. 31, 2020 | Dec. 21, 2020 | |
Related Party Transaction [Abstract] | |||||
Termination notice period for business development consulting services agreement | 60 days | ||||
Term of consulting agreement | 3 years | ||||
Warrant issued (in shares) | 7,051 | 603,386 | |||
Restricted Stock Units [Member] | |||||
Related Party Transaction [Abstract] | |||||
Shares granted (in shares) | 3,500 | ||||
Warrants Exercisable for Common Shares [Member] | |||||
Related Party Transaction [Abstract] | |||||
Warrant issued (in shares) | 0 | 603,386 | |||
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 | $ 28 | $ 28 | |||
Member of the Board of Directors - Two [Member] | |||||
Related Party Transaction [Abstract] | |||||
Term of consulting agreement | 1 year | ||||
Number of options granted (in shares) | 9,784 | ||||
Fair value amount of options granted | $ 100 | ||||
Spouse of Executive officer [Member] | Restricted Stock Units [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 | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
401(k) Savings Plan [Abstract] | ||
Percentage of matching employee contributions | 4.00% | |
Matching employee contributions | $ 0.2 | $ 0.1 |
Subsequent Events (Details)
Subsequent Events (Details) - Subsequent Event [Member] shares in Millions, $ in Millions | Apr. 26, 2021USD ($)shares |
2021 Convertible Notes [Member] | |
Convertible Notes [Abstract] | |
Convertible notes, outstanding | $ 5.2 |
2022 Convertible Notes [Member] | |
Convertible Notes [Abstract] | |
Redemption of principal balance | $ 38.4 |
2022 Convertible Notes [Member] | Common Stock [Member] | |
Convertible Notes [Abstract] | |
Common stock issued for convertible notes (in shares) | shares | 1.3 |