Document and Entity Information
Document and Entity Information - USD ($) | 12 Months Ended | ||
Dec. 31, 2021 | Mar. 10, 2022 | Jun. 30, 2021 | |
Cover [Abstract] | |||
Document Type | 10-K | ||
Amendment Flag | false | ||
Document Period End Date | Dec. 31, 2021 | ||
Document Fiscal Year Focus | 2021 | ||
Document Fiscal Period Focus | FY | ||
Trading Symbol | AVRO | ||
Entity Registrant Name | AVROBIO, INC. | ||
Entity Central Index Key | 0001681087 | ||
Current Fiscal Year End Date | --12-31 | ||
Entity Filer Category | Non-accelerated Filer | ||
Entity Common Stock, Shares Outstanding | 43,696,327 | ||
Entity Public Float | $ 336,928,742 | ||
Entity Emerging Growth Company | true | ||
Entity Ex Transition Period | false | ||
Entity Small Business | true | ||
Entity Well-known Seasoned Issuer | No | ||
Entity Current Reporting Status | Yes | ||
Entity Voluntary Filers | No | ||
Entity Shell Company | false | ||
Entity File Number | 001-38537 | ||
Entity Tax Identification Number | 81-0710585 | ||
Entity Address, Address Line One | One Kendall Square | ||
Entity Address, Address Line Two | Building 300 | ||
Entity Address, Address Line Three | Suite 201 | ||
Entity Address, City or Town | Cambridge | ||
Entity Address, State or Province | MA | ||
Entity Address, Postal Zip Code | 02139 | ||
City Area Code | 617 | ||
Local Phone Number | 914-8420 | ||
Document Annual Report | true | ||
Document Transition Report | false | ||
Entity Incorporation, State or Country Code | DE | ||
Entity Interactive Data Current | Yes | ||
Title of 12(b) Security | Common Stock, $0.0001 par value per share | ||
Security Exchange Name | NASDAQ | ||
ICFR Auditor Attestation Flag | false | ||
Auditor Firm ID | 42 | ||
Auditor Name | Ernst & Young LLP | ||
Auditor Location | Boston, Massachusetts | ||
Documents Incorporated by Reference | DOCUMENTS INCORPORATED BY REFERENCE Part III of this Annual Report on Form 10-K incorporates by reference certain information from the registrant’s definitive Proxy Statement for its 2022 annual meeting of shareholders, which the registrant intends to file pursuant to Regulation 14A with the Securities and Exchange Commission not later than 120 days after the registrant’s fiscal year end of December 31, 2021. Except with respect to information specifically incorporated by reference in this Form 10-K, the Proxy Statement is not deemed to be filed as part of this Form 10-K. |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Current assets: | ||
Cash and cash equivalents | $ 189,567 | $ 259,682 |
Prepaid expenses and other current assets | 9,578 | 7,560 |
Total current assets | 199,145 | 267,242 |
Property and equipment, net | 4,126 | 3,064 |
Restricted cash | 492 | 492 |
Other assets | 74 | 436 |
Total assets | 203,837 | 271,234 |
Current liabilities: | ||
Accounts payable | 3,486 | 2,682 |
Accrued expenses and other current liabilities | 15,638 | 13,693 |
Deferred rent | 262 | 239 |
Total current liabilities | 19,386 | 16,614 |
Note payable, net of discount | 14,945 | |
Deferred rent, net of current portion | 30 | 276 |
Total liabilities | 34,361 | 16,890 |
Commitments and contingencies (Note 6) | ||
Stockholders’ equity: | ||
Preferred stock, $0.0001 par value; 10,000 shares authorized and no shares issued or outstanding as of December 31, 2021 and 2020 | ||
Common stock, $0.0001 par value; 150,000 shares authorized as of December 31, 2021 and 2020; 43,652 and 41,569 shares issued and outstanding as of December 31, 2021 and 2020, respectively | 4 | 4 |
Additional paid-in capital | 553,014 | 518,756 |
Accumulated deficit | (383,542) | (264,416) |
Total stockholders’ equity | 169,476 | 254,344 |
Total liabilities and stockholders' equity | $ 203,837 | $ 271,234 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - $ / shares | Dec. 31, 2021 | Dec. 31, 2020 |
Statement Of Financial Position [Abstract] | ||
Preferred stock, par value | $ 0.0001 | $ 0.0001 |
Preferred stock, authorized | 10,000,000 | 10,000,000 |
Preferred stock, issued | 0 | 0 |
Preferred stock, outstanding | 0 | 0 |
Common stock, par value | $ 0.0001 | $ 0.0001 |
Common stock, authorized | 150,000,000 | 150,000,000 |
Common stock, issued | 41,569,000 | 43,652,000 |
Common stock, outstanding | 41,569,000 | 43,652,000 |
Consolidated Statements of Oper
Consolidated Statements of Operations and Comprehensive Loss - USD ($) shares in Thousands, $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Operating expenses: | ||
Research and development | $ 83,114 | $ 87,236 |
General and administrative | 35,727 | 32,992 |
Total operating expenses | 118,841 | 120,228 |
Loss from operations | (118,841) | (120,228) |
Other (expense) income: | ||
Interest (expense) income, net | (224) | 719 |
Other expense, net | (61) | (203) |
Total other (expense) income, net | (285) | 516 |
Net loss | (119,126) | (119,712) |
Comprehensive loss | $ (119,126) | $ (119,712) |
Net loss per share —basic and diluted | $ (2.78) | $ (3.31) |
Weighted-average number of common shares outstanding—basic and diluted | 42,854 | 36,206 |
Consolidated Statements of Stoc
Consolidated Statements of Stockholders' Equity - USD ($) $ in Thousands | Total | Public Offering [Member] | ATM Facility [Member] | Common Stock [Member] | Common Stock [Member]Public Offering [Member] | Common Stock [Member]ATM Facility [Member] | Additional Paid-in Capital [Member] | Additional Paid-in Capital [Member]Public Offering [Member] | Additional Paid-in Capital [Member]ATM Facility [Member] | Accumulated Deficit [Member] |
Beginning balance at Dec. 31, 2019 | $ 186,013 | $ 3 | $ 330,714 | $ (144,704) | ||||||
Beginning balance, shares at Dec. 31, 2019 | 31,643,000 | |||||||||
Vesting of restricted stock awards and units, shares | 31,000 | |||||||||
Exercise of stock options | 270 | 270 | ||||||||
Exercise of stock options, shares | 148,000 | |||||||||
Issuance of common stock, net of offering costs | $ 163,849 | $ 8,130 | $ 1 | $ 163,848 | $ 8,130 | |||||
Issuance of common stock upon public offering, net of offering costs, shares | 9,350,000 | 384,000 | ||||||||
Issuance of common stock under 2018 employee stock purchase plan | 166 | 166 | ||||||||
Issuance of common stock under 2018 employee stock purchase plan, shares | 13,000 | |||||||||
Stock-based compensation expense | 15,628 | 15,628 | ||||||||
Net loss | (119,712) | (119,712) | ||||||||
Ending balance at Dec. 31, 2020 | 254,344 | $ 4 | 518,756 | (264,416) | ||||||
Ending balance, shares at Dec. 31, 2020 | 41,569,000 | |||||||||
Vesting of restricted stock awards and units, shares | 1,000 | |||||||||
Exercise of stock options | $ 920 | 920 | ||||||||
Exercise of stock options, shares | 226,180 | 226,000 | ||||||||
Issuance of common stock, net of offering costs | $ 14,550 | $ 14,550 | ||||||||
Issuance of common stock upon public offering, net of offering costs, shares | 1,829,000 | |||||||||
Issuance of common stock under 2018 employee stock purchase plan | $ 209 | 209 | ||||||||
Issuance of common stock under 2018 employee stock purchase plan, shares | 27,000 | |||||||||
Stock-based compensation expense | 18,579 | 18,579 | ||||||||
Net loss | (119,126) | (119,126) | ||||||||
Ending balance at Dec. 31, 2021 | $ 169,476 | $ 4 | $ 553,014 | $ (383,542) | ||||||
Ending balance, shares at Dec. 31, 2021 | 43,652,000 |
Consolidated Statements of St_2
Consolidated Statements of Stockholders' Equity (Parenthetical) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Public Offering [Member] | ||
Issuance of common stock upon public offering, offering costs | $ 697 | |
ATM Facility [Member] | ||
Issuance of common stock upon public offering, offering costs | $ 47 | $ 67 |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Cash flows from operating activities: | ||
Net loss | $ (119,126) | $ (119,712) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Stock-based compensation expense | 18,579 | 15,628 |
Depreciation and amortization expense | 1,399 | 1,207 |
Deferred rent expense | (223) | (183) |
Other | 49 | 20 |
Changes in operating assets and liabilities: | ||
Prepaid expenses and other current assets | (2,018) | 1,098 |
Other assets | 362 | 84 |
Accounts payable | 804 | (881) |
Accrued expenses and other current liabilities | 2,149 | 3,936 |
Net cash used in operating activities | (98,025) | (98,803) |
Cash flows from investing activities: | ||
Purchases of property and equipment | (2,461) | (1,177) |
Net cash used in investing activities | (2,461) | (1,177) |
Cash flows from financing activities: | ||
Proceeds from long-term debt | 15,000 | |
Payments of issuance cost related to long-term debt | (103) | |
Exercise of stock options | 920 | 270 |
Net cash provided by financing activities | 30,371 | 172,619 |
Net (decrease) increase in cash, cash equivalents and restricted cash | (70,115) | 72,639 |
Cash, cash equivalents and restricted cash at beginning of period | 260,174 | 187,535 |
Cash, cash equivalents and restricted cash at end of period | 190,059 | 260,174 |
Supplemental Cash: | ||
Interest paid | 98 | |
Supplemental disclosure of non-cash investing and financing activities: | ||
Common stock offering costs incurred but unpaid at period end | 205 | |
Reconciliation of cash, cash equivalents and restricted cash reported within the consolidated balance sheets: | ||
Cash and cash equivalents, end of period | 189,567 | 259,682 |
Restricted cash | 492 | 492 |
Cash, cash equivalents and restricted cash at end of period | 190,059 | 260,174 |
2018 Employee Stock Purchase Plan [Member] | ||
Cash flows from financing activities: | ||
Proceeds from issuance of common stock, net of offering costs | 209 | 166 |
Public Offering [Member] | ||
Cash flows from financing activities: | ||
Proceeds from issuance of common stock, net of offering costs | (205) | 164,053 |
ATM Facility [Member] | ||
Cash flows from financing activities: | ||
Proceeds from issuance of common stock, net of offering costs | $ 14,550 | $ 8,130 |
Nature of the Business
Nature of the Business | 12 Months Ended |
Dec. 31, 2021 | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | |
Nature of the Business | 1. Nature of the Business AVROBIO, Inc. (the “Company” or “AVROBIO”) is a clinical-stage gene therapy company focused on developing potentially curative ex vivo The Company is subject to risks and uncertainties common to early-stage companies in the biotechnology industry, including but not limited to, risks associated with completing preclinical studies and clinical trials, receiving regulatory approvals for product candidates, development by competitors of new biopharmaceutical products, dependence on key personnel, protection of proprietary technology, compliance with government regulations and the ability to secure additional capital to fund operations. Product candidates currently under development will require significant additional research and development efforts, including preclinical and clinical testing and regulatory approval, prior to commercialization. These efforts require significant amounts of additional capital, adequate personnel and infrastructure and extensive compliance-reporting capabilities. Even if the Company’s product development efforts are successful, it is uncertain when, if ever, the Company will realize revenue from product sales. The Company has devoted substantially all of its efforts to research and development, business planning, acquiring operating assets, seeking protection for its technology and product candidates, and raising capital. Since inception, the Company has funded its operations through sales of preferred stock and common stock and a term loan facility. As of December 31, 2021, the Company had an accumulated deficit of $383,542. Although the Company has incurred recurring losses and expects to continue to incur losses for the foreseeable future, the Company expects that its existing cash and cash equivalents on hand as of December 31, 2021 of $189,567 will be sufficient to fund current planned operations and capital expenditure requirements for at least the next twelve months from the filing date of this Annual Report on Form 10-K with the SEC. However, the future viability of the Company is dependent on its ability to raise additional capital to finance its operations. The Company’s inability to raise capital as and when needed could have a negative impact on its financial condition and ability to pursue its business strategies. There can be no assurance that the current operating plan will be achieved or that additional funding will be available on terms acceptable to the Company, or at all. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 12 Months Ended |
Dec. 31, 2021 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies Basis of Presentation The accompanying consolidated financial statements have been prepared in conformity with accounting principles generally accepted in the United States of America (“GAAP”). Any reference in these notes to applicable guidance is meant to refer to the authoritative United States generally accepted accounting principles as found in the Accounting Standards Codification (“ASC”) and Accounting Standards Update (“ASU”) of the Financial Accounting Standards Board (“FASB”). Principles of Consolidation The accompanying consolidated financial statements include the accounts of AVROBIO, Inc. and its wholly owned subsidiaries. All intercompany transactions and balances have been eliminated in consolidation. Segment Information Operating segments are identified as components of an enterprise about which separate discrete financial information is available for evaluation by the chief operating decision maker, or decision-making group, in making decisions on how to allocate resources and assess performance. The Company’s chief operating decision maker is the chief executive officer (“CEO”). The Company and the CEO view the Company’s operations and manage its business as one operating segment. All material long-lived assets of the Company reside in the United States. Use of Estimates The preparation of financial statements in conformity with GAAP requires that the Company make estimates and judgments that may affect the reported amounts of assets, liabilities and expenses and the related disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of expenses during the reporting periods On an on ‑going basis, the Company evaluates its estimates, judgments and methodologies. The Company bases its estimates on historical experience and on various other assumptions that are believed to be reasonable, the results of which form the basis for making judgments about the carrying values of assets and liabilities. Actual results may differ from these estimates. Changes in estimates are reflected in reported results in the period in which they become known. Cash and Cash Equivalents The Company considers all highly liquid investments purchased with original maturities of three months or less at acquisition to be cash equivalents. As of December 31, 2021 and 2020, Concentrations of Credit Risk The Company has no significant off-balance sheet risk, such as foreign exchange contracts, option contracts, or other foreign hedging arrangements. Financial instruments that potentially expose the Company to concentrations of credit risk consist primarily of cash, cash equivalents and restricted cash. Periodically, the Company maintains deposits in accredited financial institutions in excess of federally insured limits. The Company deposits its cash and cash equivalents in financial institutions that it believes have high credit quality and has not experienced any losses on such accounts and does not believe it is exposed to any unusual credit risk beyond the normal credit risk associated with commercial banking relationships. Fair Value Measurements Certain assets and liabilities of the Company are carried at fair value under GAAP. Fair value is defined as the exchange price that would be received for an asset or paid to transfer a liability (an exit price) in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants on the measurement date. Valuation techniques used to measure fair value must maximize the use of observable inputs and minimize the use of unobservable inputs. Financial assets and liabilities carried at fair value are to be classified and disclosed in one of the following three levels of the fair value hierarchy, of which the first two are considered observable and the last is considered unobservable: • Level 1— Fair values are determined utilizing prices (unadjusted) in active markets for identical assets or liabilities that the Company has the ability to access. • Level 2—Observable inputs (other than Level 1 quoted prices), such as quoted prices in active markets for similar assets or liabilities, quoted prices in markets that are not active for identical or similar assets or liabilities, or other inputs that are observable or can be corroborated by observable market data. • Level 3—Unobservable inputs that are supported by little or no market activity that are significant to determining the fair value of the assets or liabilities, including pricing models, discounted cash flow methodologies and similar techniques. The carrying amounts of the Company’s financial instruments, which include cash equivalents, accounts payable, and accrued expenses, approximated their fair values as of December 31, 2021 and 2020 due to the short‑term nature of these instruments. The Company has evaluated the estimated fair value of financial instruments using available market information. The use of different market assumptions, estimation methodologies, or both, could have a significant effect on the estimated fair value amounts. See Note 4 “ Fair Value of Financial Assets and Liabilities” Property and Equipment Property and equipment are recorded at cost. Depreciation and amortization is calculated using the straight-line method over the following estimated useful lives of the assets: Estimated Useful Life Laboratory and office equipment 5 years Computer equipment 2 years Leasehold improvements Lesser of lease term or 10 years Impairment of Long-Lived Assets Long-lived assets consist of property and equipment. Long-lived assets to be held and used are tested for recoverability whenever events or changes in business circumstances indicate that the carrying amount of the assets may not be fully recoverable. Factors that the Company considers in deciding when to perform an impairment review include significant underperformance of the business in relation to expectations, significant negative industry or economic trends and significant changes or planned changes in the use of the assets. If an impairment review is performed to evaluate a long-lived asset group for recoverability, the Company compares forecasts of undiscounted cash flows expected to result from the use and eventual disposition of the long-lived asset group to its carrying value. An impairment loss would be recognized when estimated undiscounted future cash flows expected to result from the use of an asset group are less than its carrying amount. The impairment loss would be based on the excess of the carrying value of the impaired asset group over its fair value, determined based on discounted cash flows. The Company did not record any impairment loss during the years ended . Leases The Company categorizes leases at their inception as either operating or capital leases. On certain lease arrangements, the Company may receive rent holidays or other incentives. The Company recognizes lease costs on a straight-line basis once control of the space is achieved, without regard to deferred payment terms, such as rent holidays, that defer the commencement date of required payments or escalating payment amounts. The difference between required lease payments and rent expense has been recorded as deferred rent and accrued expenses and other current liabilities in the accompanying consolidated balance sheets. Additionally, incentives received are treated as a reduction of costs over the term of the agreement, as they are considered an inseparable part of the lease agreement. Comprehensive Income (Loss) Comprehensive income (loss) is defined as the change in stockholders’ equity of a business enterprise during a period from transactions and other events and circumstances from non-owner sources. Comprehensive income (loss) includes net income (loss) as well as other changes in stockholders’ equity (deficit) which includes certain changes in equity that are excluded from net income (loss). Comprehensive loss has been disclosed in the accompanying consolidated statements of operations and comprehensive loss and equals the Company’s net loss for all periods presented. Foreign Currency Translation The functional currency of the Company’s international operations in Canada and Australia is the U.S. dollar. Accordingly, all operating assets and liabilities of these international subsidiaries are remeasured into U.S. dollars using the exchange rates in effect at the balance sheet date or historical rates, as appropriate, while expenses are remeasured into U.S. dollars at the average rates in effect during the period. Any differences resulting from the remeasurement of assets, liabilities, and operations of the Canadian and Australian subsidiaries are recorded within other (expense) income, net in the consolidated statements of operations and comprehensive loss. During the years ended December 31, 2021 and 2020, the Company recorded foreign exchange losses of $61 and $203, respectively, in other expense Research and Development Expenses Research and development costs are expensed as incurred. Research and development expenses consist of costs incurred in performing research and development activities, including salaries, stock-based compensation and benefits, facilities costs, depreciation, third-party license fees, and external costs of outside vendors engaged to conduct preclinical development activities and clinical trials as well as to manufacture research and development materials. Non-refundable prepayments for goods or services that will be used or rendered for future research and development activities are deferred and capitalized. Such amounts are recognized as an expense as the goods are delivered or the related services are performed or until it is no longer expected that the goods will be delivered or the services rendered. The Company has entered into various research and development related contracts with parties both inside and outside of the United States. The payments related to these agreements are recorded as research and development expenses as incurred. The Company records accrued liabilities for estimated ongoing research costs. When evaluating the adequacy of the accrued liabilities, the Company analyzes progress of the studies or clinical trials, including the phase or completion of events, invoices received and contracted costs. Significant judgments and estimates are made in determining the accrued balances at the end of any reporting period. Actual results could differ from the Company’s estimates. The Company’s historical accrual estimates have not been materially different from the actual costs. Stock-Based Compensation For stock-based awards issued to employees and members of the Company’s board of directors (the “Board”) for their services on the Board, the Company measures the estimated fair value of the stock-based award on the date of grant and recognizes compensation expense for those awards over the requisite service period, which is generally the vesting period of the respective award. The Company issues stock-based awards with only service-based vesting conditions and records the expense for these awards using the straight-line method. The Company has not issued any stock-based awards with performance- or market-based vesting conditions. The Company accounts for forfeitures as they occur. Prior to the adoption of Accounting Standards Update (“ASU”) No. 2018-07, Compensation-Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting The Company classifies stock-based compensation expense in its consolidated statements of operations and comprehensive loss in the same manner in which the award recipient’s cash compensation costs are classified. Given the absence of an active market for the Company’s common stock prior to its initial public offering (“IPO”), the Company and the Board, the members of which the Company believes have extensive business, finance, and venture capital experience, were required to estimate the fair value of the Company’s common stock at the time of each grant of a stock-based award. The Company and the Board determined the estimated fair value of the Company’s equity instruments based on a number of factors, including external market conditions affecting the biotechnology industry sector. The Company and the Board utilized various valuation methodologies in accordance with the framework of the American Institute of Certified Public Accountants’ Technical Practice Aid, Valuation of Privately-Held Company Equity Securities Issued as Compensation The fair value of each stock option award is estimated on the date of grant using the Black-Scholes option pricing model. As there was no public market for its common stock prior to June 21, 2018, which was the first day of trading, and as the trading history of the Company’s common stock was limited through December 31, 2021, the Company determined the volatility for awards granted based on an analysis of reported data for a group of guideline companies that issued options with substantially similar terms. The expected volatility has been determined using a weighted-average of the historical volatility measures of this group of guideline companies. The Company expects to continue to do so until such time as it has adequate historical data regarding the volatility of its own traded stock price. The expected term of the Company’s stock options has been determined utilizing the “simplified” method for awards that qualify as “plain-vanilla” options. The risk-free interest rate is determined by reference to the U.S. Treasury yield curve in effect at the time of grant of the award for time periods approximately equal to the expected term of the award. The Company has not paid, and does not anticipate paying, cash dividends on its common stock; therefore, the expected dividend yield is assumed to be zero. Income Taxes Deferred tax assets and liabilities are determined on the basis of the differences between the consolidated financial statements and tax basis of assets and liabilities using enacted tax rates in effect for the year in which the differences are expected to reverse. Changes in deferred tax assets and liabilities are recorded in the provision for income taxes. The Company assesses the likelihood that its deferred tax assets will be recovered from future taxable income and, to the extent it believes, based upon the weight of available evidence, that it is more likely than not that all or a portion of the deferred tax assets will not be realized, a valuation allowance is established. The Company accounts for uncertain tax positions recognized in the consolidated financial statements by prescribing a more-likely-than-not threshold for financial statement recognition and measurement of a tax position taken or expected to be taken in a tax return. 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 Basic net loss per share is computed by dividing net loss by the weighted-average number of shares of common stock outstanding during the period, without consideration of potential dilutive securities. Diluted net loss per share is computed by adjusting the weighted-average shares outstanding for the potential dilutive effects of common stock equivalents outstanding during the period calculated in accordance with the treasury stock method. For purposes of the diluted net loss per share calculation, stock options and restricted stock units are considered to be common stock equivalents but have been excluded from the calculation of diluted net loss per share, as their effect would be anti-dilutive for all periods presented. Therefore, basic and diluted net loss per share were the same for all periods presented. Subsequent Event Considerations The Company considers events or transactions that occur after the balance sheet date but prior to the issuance of the consolidated financial statements to provide additional evidence for certain estimates or to identify matters that require additional disclosure. Subsequent events have been evaluated as required. See Note 14. Emerging Growth Company Status The Company is an “emerging growth company,” as defined in the Jumpstart Our Business Startups Act, or JOBS Act, and may take advantage of certain exemptions from various reporting requirements that are applicable to other public companies that are not emerging growth companies. The Company may take advantage of these exemptions until the Company is no longer an “emerging growth company.” Section 107 of the JOBS Act provides that an “emerging growth company” can take advantage of the extended transition period afforded by the JOBS Act for the implementation of new or revised accounting standards. The Company has elected to use the extended transition period for complying with new or revised accounting standards and as a result of this election, its consolidated financial statements may not be comparable to companies that comply with public company effective dates. The Company may take advantage of these exemptions up until the last day of the fiscal year following the fifth anniversary of an offering or such earlier time that it is no longer an “emerging growth company.” Recently Issued Accounting Pronouncements In February 2016, the FASB issued ASU No. 2016-02, (Topic 842) Leases, he Company currently expects that its operating lease commitments will be subject to the new standard and recognized as right-of-use assets and operating lease liabilities upon adoption of this standard, which will increase the Company’s total assets and total liabilities. In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments—Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments In Codification Improvements to Topic 326, Financial Instruments – Credit Losses Financial Instruments – Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments As a result of the Company having elected the extended transition period for complying with new or revised accounting standards pursuant to Section 107(b) of the JOBS Act, 2016-13 and ASU 2019-11 are effective for the Company for fiscal years beginning after December 15, 2022, and interim periods within those fiscal years. In December 2019, the FASB issued ASU No. 2019-12, Simplifying the Accounting for Income Taxes |
License Agreements
License Agreements | 12 Months Ended |
Dec. 31, 2021 | |
License Agreements [Abstract] | |
License Agreements | 3. License Agreements Agreement with The University of Manchester On September 30, 2020, the Company entered into an agreement (“MPSII License Agreement”) with The University of Manchester, England (“UoM”), whereby UoM granted to the Company an exclusive worldwide license under certain patent and other intellectual property rights, subject to certain retained rights, to develop, commercialize and sell an ex vivo lentiviral gene therapy for use in the treatment of Hunter syndrome, or mucopolysaccharidosis type II (“MPSII”) As part of the agreement, the Company is obligated to make milestone payments of up to an aggregate of $80,000 upon the achievement of specified development and regulatory milestones, to pay royalties, on a product-by-product and country- by-country basis, of a mid-single digit percentage based on net sales of products licensed under the agreement and to pay a low double - digit percentage of any sublicense fees received by the Company. The next anticipated payment milestones under the MPSII License Agreement include $ 2,000 , which would become due following the date of regulatory approval of the clinical trial application for the investigator-sponsored Phase 1/2 clinical trial sponsored by UoM, and $ 4,000 , upon the dosing of the first patient in the investigator-sponsored Phase 1/2 clinical trial sponsored by UoM. Unless terminated earlier, the agreement expires upon the later of 15 years from the effective date or the expiration of the last valid claim of the licensed patents, subject to certain surviving rights and obligations. UoM and the Company can each terminate the agreement in the event of the bankruptcy or insolvency of the other party, or a material breach by the other party and failure to cure such breach within a certain period of time. UoM has the right to terminate the agreement in the event of certain actions relating to challenge or opposition to the licensed intellectual property brought by the Company or its affiliates or sublicensees. Concurrently with the MPSII License Agreement, the Company entered into a collaborative research funding agreement with UoM (“CRFA”). Under the CRFA, the Company has agreed to fund the budgeted costs of an investigator-sponsored Phase 1/2 clinical trial to be sponsored by UoM in connection with the development activities under the MPSII License Agreement, which are currently estimated to equal approximately £9,900 in the aggregate For the years ended December 31, 2021 and 2020, the Company recognized $1,437 and $565, respectively, of costs related to the CRFA. Agreements with University Health Network (“UHN”) Fabry License Agreement— On January 27, 2016, the Company entered into an agreement with UHN, pursuant to which UHN granted the Company an option to enter into an exclusive license under the UHN intellectual property related to Fabry disease in accordance with the pre-negotiated licensing terms. On November 4, 2016, the Company exercised its option and entered into a license agreement with UHN, pursuant to which UHN granted the Company an exclusive worldwide license under certain intellectual property rights and a non-exclusive worldwide license under certain know-how, in each case subject to certain retained rights, to develop, commercialize and sell products for use in the treatment of Fabry disease. In addition, for three years following the execution of the agreement, UHN granted the Company an exclusive option to obtain a license under certain improvements to the licensed intellectual property rights as well as an option to negotiate a license under certain other improvements. Under this agreement, the Company paid an option fee of CAD$20, an upfront license fee of CAD$75, plus the annual license maintenance fee for the first year. Thereafter, the Company is also required to pay UHN future annual license maintenance fees until the first sale of a licensed product in certain markets. The Company is also obligated to make future milestone payments in an aggregate amount of up to CAD$2,450 upon the achievement of specified milestones as well as royalties on a country-by-country basis of a low to mid-single-digit percentage of annual net sales of licensed products and a lower single-digit royalty percentage in certain circumstances. Additionally, the Company has agreed to pay a low double-digit royalty percentage of all sublicensing revenue. The agreement requires the Company to meet certain performance milestones within specified timeframes. UHN may terminate the agreement if the Company fails to meet these performance milestones despite using commercially reasonable efforts and the Company is unable to reach agreement with UHN on revised timeframes. The Company’s royalty obligation expires on a licensed product-by-licensed product and country-by-country basis upon the latest to occur of the expiration or termination of the last valid claim under the licensed intellectual property rights in such country, the tenth anniversary of the first commercial sale of such licensed product in such country and the expiration of any applicable regulatory exclusivity in such country. Unless terminated earlier, the agreement expires upon the expiration of the Company’s royalty obligation for all licensed products. UHN can terminate the agreement if the Company fails to make any payments within a specified period after receiving written notice of such failure, or in the event that the Company fails to obtain or maintain insurance. Either the Company or UHN may terminate the license agreement in the event of a material breach by the other party and failure to cure such breach within a certain period of time. The Company can voluntarily terminate the agreement with prior notice to UHN. For the years ended December 31, 2021 and 2020, the Company recorded research and development expense related to this agreement with UHN of $209 and $344, respectively, which consists of reimbursable funded study trial costs and license maintenance fees. No milestone fees were incurred related to the Fabry license agreement in the years ended December 31, 2021 and 2020. Interleukin 12 License Agreement— On January 27, 2016, the Company entered into an exclusive license agreement with UHN, pursuant to which UHN granted the Company a license to certain patent rights for the commercial development, manufacture, distribution and use of any products or processes resulting from development of those patent rights related to Interleukin 12. Upon execution of this agreement, the Company paid an upfront license fee of CAD$264. In addition, as part of the initial consideration for the license, the Company issued to UHN 1,161,665 shares of the Company’s common stock and agreed to pay UHN up to $2,000 upon the closing of an IPO if certain criteria are met The agreement requires the Company to meet certain diligence requirements based upon specified milestones. The agreement expires on the later of the date the last patent rights expire in the last country or ten years from the date of first sale. UHN can terminate the agreement if the Company fails to make any payments within a specified period after receiving written notice of such failure, or in the event that the Company fails to obtain or maintain insurance. The Company can voluntarily terminate the agreement with prior notice to UHN. Either the Company or UHN may terminate the license agreement in the event of a material breach by the other party and failure to cure such breach within a certain period of time. For the years ended December 31, 2021 and 2020, the Company recorded research and development expense related to this agreement with UHN of $39 and $37, respectively, which consists of license maintenance fees. No milestone fees were incurred related to the Interleukin 12 license agreement in the years ended December 31, 2021 and 2020. Agreement with BioMarin Pharmaceutical Inc. (“BioMarin”) On August 31, 2017, the Company entered into a license agreement with BioMarin, pursuant to which BioMarin granted the Company an exclusive worldwide license under certain intellectual property rights owned or controlled by BioMarin to develop, commercialize and sell products for use in the treatment of Pompe disease. The license agreement was amended in February 2018 and again in January 2020 to, among things, provide that BioMarin would supply the Company with certain technology materials. As consideration for this agreement, the Company paid an upfront license fee of $500 in cash and issued 233,765 shares of Series B Preferred Stock to BioMarin at the time of the Company’s Series B Preferred Stock financing in January 2018. The Company is also obligated to make future milestone payments of up to $13,000 upon the achievement of certain specified milestones and agreed to pay BioMarin royalties of a low single-digit percentage of net sales of licensed products sold by the Company or its affiliates covered by patent rights in a relevant country. No expenses related to the license were recorded for the years ended December 31, 2021 and 2020. Unless terminated earlier, the agreement expires upon the expiration of the Company’s royalty obligation for all licensed products throughout the world. BioMarin and the Company can terminate the agreement in the event of a material breach by the other party and failure to cure such breach within a certain period of time. The Company may terminate the agreement at will upon written notice to BioMarin. BioMarin has the right to terminate the agreement upon the Company’s bankruptcy or insolvency, or in the event of any challenge or opposition to the licensed patent rights or related actions brought by the Company or its affiliates or sublicensees, or if the Company, its affiliates or sublicensees knowingly assist a third-party in challenging or otherwise opposing the licensed patent rights, except as required under a court order or subpoena. Agreement with Papillon Therapeutics, Inc. (previously GenStem Therapeutics, Inc. ) On October 2, 2017, the Company entered into a license agreement with GenStem, pursuant to which GenStem granted the Company an exclusive worldwide license, subject to certain retained rights, under certain intellectual property rights owned or controlled by GenStem to develop, commercialize and sell products for use in the treatment of cystinosis. Under this agreement, the Company paid an upfront license fee of $1,000 and is required to make payments upon completion of certain milestones up to an aggregate of $16,000. The Company also agreed to pay GenStem a tiered mid to high single-digit royalty percentage on annual net sales of licensed products as well as a low double-digit percentage of sublicense income received from certain third-party licensees. The Company’s royalty obligation expires on a licensed product-by-licensed product and country-by-country basis on the eleventh anniversary of the first commercial sale of such licensed product in such country or the expiration of the last valid claim under the licensed patent rights covering such licensed product in such country, whichever is later. Unless terminated earlier, the agreement expires upon the expiration of the Company’s royalty obligation for all licensed products throughout the world. GenStem and the Company can terminate the agreement in the event of a material breach by the other party and failure to cure such breach within a certain period of time. The Company may terminate the agreement at will upon the specified prior written notice to GenStem. In October 2021, the Company received noticed that the license agreement with GenStem had been assigned to Papillon Therapeutics, Inc. (“Papillon”). No expenses related to the license were recorded for the years ended December 31, 2021 and 2020. Agreement with Lund University Rights Holders On November 17, 2016, the Company entered into a license agreement with affiliates of Lund University, along with certain other relevant rights holders that may be added from time to time, pursuant to which such rights holders granted to the Company an exclusive worldwide license, subject to certain retained rights, under certain intellectual property rights to develop, commercialize and sell products in any and all uses relevant to Gaucher disease. As consideration for the license, the Company is required to make payments in connection with the achievement of certain milestones up to an aggregate of $550. The agreement expires on the latest of (i) the twentieth anniversary of the end of a certain research project the Company is funding pursuant to an agreement with Lund University, (ii) the expiration of the term of any patent filed on the licensed rights that covers a licensed product, (iii) the expiration of any applicable marketing exclusivity right and (iv) such time that neither the Company nor any sublicensees, partners or contractors are commercializing a licensed product. Either the Company or the rights holders acting together may terminate the license agreement if the other such party commits a material breach and fails to cure such breach within a certain period of time, or if the other party enters into liquidation, becomes insolvent, or enters into composition or statutory reorganization proceedings. No expenses related to the license were recorded for the years ended December 31, 2021 and 2020. |
Fair Value of Financial Assets
Fair Value of Financial Assets and Liabilities | 12 Months Ended |
Dec. 31, 2021 | |
Fair Value Disclosures [Abstract] | |
Fair Value of Financial Assets and Liabilities | 4. Fair Value of Financial Assets and Liabilities The following table presents information about the Company’s financial assets and liabilities measured at fair value on a recurring basis and indicates the level of the fair value hierarchy utilized to determine such fair values as of December 31, 2021 and 2020: Fair Value Measurements as of December 31, 2021 Level 1 Level 2 Level 3 Total Assets: Cash equivalents—money market funds $ 189,332 $ — $ — $ 189,332 $ 189,332 $ — $ — $ 189,332 Fair Value Measurements as of December 31, 2020 Level 1 Level 2 Level 3 Total Assets: Cash equivalents—money market funds $ 44,416 $ — $ — $ 44,416 $ 44,416 $ — $ — $ 44,416 During the years ended December 31, 2021 and 2020, there were no transfers between levels. |
Supplemental Balance Sheet Info
Supplemental Balance Sheet Information | 12 Months Ended |
Dec. 31, 2021 | |
Balance Sheet Related Disclosures [Abstract] | |
Supplemental Balance Sheet Information | 5. Supplemental Balance Sheet Information Prepaid expenses and Other Current Assets Prepaid expenses and other current assets consisted of the following: December 31, 2021 2020 Tax incentive refund $ 2,697 $ 2,558 Prepaid research and development expenses 4,496 3,145 Prepaid insurance 112 973 Prepaid compensation benefits 575 609 Interest income receivable — 4 Other current assets 1,698 271 Prepaid expenses and other current assets $ 9,578 $ 7,560 Property and equipment, net Property and equipment, net consisted of the following: December 31, 2021 2020 Laboratory and office equipment $ 6,162 $ 3,965 Leasehold improvements 1,635 1,377 Computer equipment 149 143 7,946 5,485 Less: Accumulated depreciation and amortization (3,820 ) (2,421 ) Property and equipment, net $ 4,126 $ 3,064 Depreciation and amortization expense for the years ended December 31, 2021 and 2020 was $1,399 and $1,207, respectively. Restricted Cash As of December 31, 2021 and 2020, the Company had restricted cash as presented in the table below, which consists of cash used to secure letters of credit for the benefit of the landlord in connection with the Company’s lease agreements. The cash will be restricted until the termination or modification of the lease arrangement. December 31, 2021 2020 Restricted cash $ 492 $ 492 Accrued Expenses Accrued expenses consisted of the following: December 31, 2021 2020 Compensation and benefit costs $ 5,579 $ 6,402 Research and development expenses 8,882 5,847 Consulting and professional fees 999 1,096 Other liabilities 178 348 $ 15,638 $ 13,693 |
Commitments and Contingencies
Commitments and Contingencies | 12 Months Ended |
Dec. 31, 2021 | |
Commitments And Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 6. Commitments and Contingencies Lease Agreements On January 12, 2018, the Company entered into a lease agreement for office space located in Cambridge, Massachusetts. The lease agreement expires in January 2023. The annual lease payments are subject to a 3% increase each year. The Company recognizes rent expense on a straight-line basis over the lease period and has recorded deferred rent for rent expense incurred but not yet paid. The Company received a tenant incentive allowance of $842 in 2018. Such incentive allowance is being amortized as a reduction of rent expense on a straight-line basis over the lease period. In accordance with the lease agreement, the Company is required to maintain a security deposit of $209, which was recorded in restricted cash as of December 31, 2021 and 2020. On August 31, 2018, the Company entered into a sub-lease agreement for lab space located in Cambridge Massachusetts, United States, which was set to expire in October 2020. On June 9, 2020, the Company amended the terms of the sublease, which was set to expire in April 2022. Effective January 1, 2022, the Company amended the terms of the sublease, which is now set to expire in April 2023. The annual lease payments are subject to a 5% increase each year. In accordance with the lease agreement, the Company is required to maintain a security deposit of $283, which was recorded in restricted cash as of December 31, 2021 and 2020. On June 1, 2020, the Company entered into a lease agreement for office space located in Toronto, Ontario, Canada, which is set to expire in June 2025. The annual lease payments are fixed for years 1 and 2, and then subject to a 6.67% increase for years 3 through 5. In accordance with the lease agreement, the Company is required to maintain a security deposit of CAD$27, which was recorded in other long-term assets as of December 31, 2021 and 2020. The Company recorded rent expense of $2,648 and $2,352 during the years ended December 31, 2021 and 2020, respectively. The following table summarizes the future minimum lease payments due under operating leases as of December 31, 2021: Year Ending December 31, 2022 $ 1,297 2023 208 2024 147 2025 73 $ 1,725 Other Funding Commitments As of December 31, 2021, the Company had several ongoing clinical and non-clinical studies for its various pipeline programs. The Company enters into contracts in the normal course of business with contract research organizations and clinical sites for the conduct of clinical trials, professional consultants for expert advice and other vendors for clinical supply manufacturing or other services. These contracts are generally cancellable, with notice, at the Company’s option and do not have significant cancellation penalties. Guarantees The Company enters into certain agreements with other parties in the ordinary course of business that contain indemnification provisions. These typically include agreements with directors and officers, business partners, contractors, landlords and clinical sites. Under these provisions, the Company generally indemnifies and holds harmless the indemnified party for losses suffered or incurred by the indemnified party as a result of the Company’s activities. These indemnification provisions generally survive termination of the underlying agreement. The maximum potential amount of future payments the Company could be required to make under these indemnification provisions is unlimited. However, to date the Company has not incurred material costs to defend lawsuits or settle claims related to these indemnification provisions. As a result, the estimated fair value of these obligations is minimal. Litigation The Company, from time to time, may be party to litigation arising in the ordinary course of business. The Company was not subject to any material legal proceedings during the years ended December 31, 2021 and 2020, and to the best of its knowledge, no material legal proceedings are currently pending or threatened. Other The Company is also party to various agreements, principally relating to licensed technology, that require future payments relating to milestones not met as of December 31, 2021 and 2020, or royalties on future sales of specified products. No milestone or royalty payments under these agreements are expected to be payable in the immediate future. See Note 3 “Licenses Agreements” The Company enters into standard indemnification agreements in the ordinary course of business. Pursuant to the agreements, the Company agrees to indemnify, hold harmless, and to reimburse the indemnified party for losses suffered or incurred by the indemnified party, generally the Company’s business partners, in connection with any U.S. patent or any copyright or other intellectual property infringement claim by any third-party with respect to the Company’s products. The term of these indemnification agreements is generally perpetual any time after execution of the agreement. The maximum potential amount of future payments the Company could be required to make under these indemnification agreements is unlimited. The Company has never incurred costs to defend lawsuits or settle claims related to these indemnification agreements. |
Note Payable
Note Payable | 12 Months Ended |
Dec. 31, 2021 | |
Debt Disclosure [Abstract] | |
Note Payable | 7. Note Payable On November 2, 2021 (the “Closing Date”), the Company entered into a Loan and Security Agreement (the “Loan Agreement”) with Silicon Valley Bank (“SVB”) pursuant to which a term loan in an aggregate principal amount of up to $50,000 (the “Term Loan Facility”) is available to the Company in three tranches, subject to certain terms and conditions. The first tranche of $15,000 was advanced to the Company on the Closing Date. Subject to the terms and conditions of the Loan Agreement, the first tranche allows the Company to borrow an additional $15,000 through October 31, 2023. Upon satisfaction of certain milestones, the second and third tranches are available under the Term Loan Facility which allows the Company to borrow an additional amount up to $10,000 in each tranche through October 31, 2023. Additionally, the Company may seek to borrow up to an additional $15,000 at the sole discretion of the lender through the term of the Loan Agreement. The Loan Agreement matures on October 1, 2026 (the “Maturity Date”). The Company is required to pay an end of term fee (“End of Term Charge”) equal to 9.00% of the aggregate principal amount of the Term Loan advances upon repayment. Advances under the Term Loan Facility will bear interest at a rate equal to the greater of either (i) the Prime Rate (as reported in The Wall Street Journal) plus 4.85%, and (ii) 8.10%. The Company will make interest only payments through November 1, 2024. Following the interest only period, the Company will repay the principal balance and interest of the advances in equal monthly installments through October 1, 2026. The Company may prepay advances under the Loan Agreement, in whole or in part, at any time subject to a prepayment charge (the “Prepayment Premium”) equal to: (a) 1.50% of amounts so prepaid, if such prepayment occurs during the first year following the Closing Date; (b) 1.00% of the amount so prepaid, if such prepayment occurs during the second year following the Closing Date, and (c) 0.00% of the amount so prepaid, if such prepayment occurs after the second year following the Closing Date. Upon prepayment or repayment of all or any of the term loans under the Term Loan Facility, the Company will pay (in addition to any Prepayment Premium) an end of term charge of 9.0% of the aggregate funded amount under the Term Loan Facility. The Term Loan Facility is secured by substantially all of the Company’s assets, other than the Company’s intellectual property. The Company has agreed to not pledge or secure its intellectual property to others. The End of Term Charge is recorded as a debt discount with an initial carrying balance of $1,350. During the year ended December 31, 2021 the Company recognized $103 of debt issuance costs related to legal expenses that has been included in the debt discount balance. The debt discount costs are being accreted to the principal amount of debt and being amortized from the date of issuance through the Maturity Date to interest expense using the effective-interest rate method. The effective interest rate of the outstanding debt under the Loan Agreement is approximately 11.12 %. As of December 31, 2021 the carrying value of the note payable consists of the following: December 31, 2021 (in thousands) Note payable, including End of Term Charge 16,350 Debt discount, net of accretion (1,405 ) Note payable, net of discount, long-term 14,945 As of December 31, 2021, the future principal payments due under the arrangement, excluding interest and the end of term charge, are as follows: Year Ending December 31, Principle 2022 $ — 2023 — 2024 1,875 2025 7,500 2026 5,625 Total $ 15,000 During the year ended December 31, 2021, the Company recognized $203 of interest expense related to the Loan Agreement, which is reflected in other (expense) income, net on the consolidated statements of operations and comprehensive loss. |
Common Stock
Common Stock | 12 Months Ended |
Dec. 31, 2021 | |
Equity [Abstract] | |
Common Stock | 8. Common Stock As of December 31, 2021 and 2020, the authorized capital stock of the Company included 150,000,000 shares of common stock, $0.0001 par value, and 10,000,000 shares of undesignated preferred stock. As of December 31, 2021 and 2020, no undesignated shares of preferred stock were outstanding. In accordance to the Fourth Amended and Restated Certificate of Incorporation, the holders of the common stock shall have the exclusive right to vote for the election of directors of the Company and on all other matters requiring stockholder action, each outstanding share entitling the holder thereof to one vote on each matter properly submitted to the stockholders of the Company for their vote; provided, however, that, except as otherwise required by law, holders of common stock, as such, shall not be entitled to vote on any amendment to any amendment to a certificate of designations of any series of undesignated preferred stock that alters or changes the powers, preferences, rights or other terms of one or more outstanding series of undesignated preferred stock if the holders of such affected series of undesignated preferred stock are entitled to vote, either separately or together with the holders of one or more other such series, on such amendment pursuant to a certificate of designations of any series of undesignated preferred stock. Through December 31, 2021, no cash dividends have been declared or paid. Public Offerings In July 2019, the Company closed an underwritten public offering of 7,475,000 shares of its common stock at a public offering price of $18.50 per share (the “July 2019 Follow-on Offering”), which included 975,000 shares of the Company’s common stock resulting from the full exercise of the underwriters’ option to purchase additional shares at the public offering price, less underwriting discounts and commissions. The net proceeds to the Company from the July 2019 Follow-on Offering, after deducting underwriting discounts and commissions and other offering expenses payable by the Company, were $129,464. In February 2020, the Company closed an underwritten public offering of shares of its common stock at a public offering price of $ 23.00 per share (the “February 2020 Follow-on Offering”) . The net proceeds to the Company from the February 2020 Follow-on Offering, after deducting underwriting discounts and commissions and other offering expenses payable by the Company, were $ . In June 2020, the Company sold an aggregate of 384,140 shares of common stock under its “at-the-market” facility (the “ATM Facility”) for net proceeds, after deducting commissions and other offering expenses payable by the Company, of $8,130. In November 2020, the Company closed an underwritten public offering of 5,000,000 shares of its common stock at a public offering price of $15.00 per share (the “November 2020 Follow-on Offering”). The net proceeds to the Company from the November 2020 Follow-on Offering, after deducting underwriting discounts and commissions and other offering expenses payable by the Company, were $70,221. As of December 31, 2021, approximately $26,549 of common stock remained available for future issuance under the ATM Facility. Common Stock Reserved for Future Issuance As of December 31, 2021 and 2020, the Company has reserved the following shares of common stock for future issuance: December 31, 2021 2020 Shares reserved for exercise of outstanding stock options 7,423,777 5,559,545 Shares reserved for vesting of restricted stock units 599,850 1,198 Shares reserved for issuance under the 2018 Stock Option and Incentive Plan 2,583,736 3,877,478 Shares reserved for issuance under the 2018 Employee Stock Purchase Plan 1,151,010 762,900 Shares reserved for issuance under the 2019 Inducement Plan 412,686 365,050 Shares reserved for issuance under the 2020 Inducement Plan 1,637,000 1,700,000 Total shares of authorized common stock reserved for future issuance 13,808,059 12,266,171 |
Stock-Based Compensation
Stock-Based Compensation | 12 Months Ended |
Dec. 31, 2021 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Stock-Based Compensation | 9. Stock-Based Compensation Amended and Restated 2015 Stock Option and Grant Plan The Company’s Amended and Restated 2015 Stock Option and Grant Plan, (the “2015 Plan”) provides for the Company to issue restricted stock awards and restricted stock units, or to grant incentive stock options or non-statutory stock options. Incentive stock options may be granted only to the Company’s employees including officers and members of the Board who are also employees. Restricted stock awards and restricted stock units and non- statutory stock options may be granted to employees, members of the Board, outside advisors, and consultants of the Company. The total number of common shares that may be issued under the 2015 Plan was 2,008,564 shares. Following the IPO, no further grants have been made under 2015 plan. Shares that expire, are terminated, surrendered or cancelled under the 2015 Plan without having been fully exercised will be available for future awards under the 2018 Plan (as defined below). In addition, shares of common stock that are tendered to the Company by a participant to exercise an award are added to the number of shares of common stock available for future awards. The 2015 Plan is administered by the Board. Equity awards granted to employees and members of the Board typically vest over four years. 2018 Stock Option and Incentive Plan The Company’s 2018 Stock Option and Incentive Plan (the “2018 Plan”) was adopted by the Board on June 1, 2018 and approved by stockholders on June 7, 2018 and became effective upon the effectiveness of the Company’s Registration Statement on Form S-1. The 2018 Plan replaced the 2015 Plan as the Board determined not to make additional awards under the 2015 Plan following the pricing of the Company’s IPO. The 2018 Plan allows the Board, compensation committee or other designated committee to make equity-based and cash-based incentive awards to its officers, employees, directors and other key persons (including consultants). The Company initially reserved 616,300 shares of its common stock for the issuance of awards under the 2018 Plan. The 2018 Plan provides that the number of shares reserved and available for issuance under the plan will automatically increase each January 1, beginning on January 1, 2019, by 4% of the outstanding number of shares of our common stock on the immediately preceding December 31, or such lesser number of shares as determined by its Board or compensation committee (the “Plan Evergreen”). This number is subject to adjustment in the event of a stock split, stock dividend or other change in its capitalization. On April 16, 2020, the Board adopted an amendment to the 2018 Plan (the “Amendment”), to (i) increase the number of shares of common stock currently reserved for issuance under the 2018 Plan by 3,300,000 shares and (ii) automatically terminate the 2018 Plan’s annual increase (or “evergreen”) provision after January 2022. The Amendment was approved by the Board on June 4, 2020 and the Company’s stockholders on June 4, 2020. The number of shares of common stock available for future grant under the 2018 Plan was 2,583,736 as of December 31, 2021, which does not include the shares added to the 2018 Plan reserve on January 1, 2022 as a result of the Plan Evergreen for the year ended December 31, 2021. During the years ended December 31, 2021 and 2020, the Company granted options to purchase 4,253,232 and, 3,019,663 shares, respectively, of common stock to employees, nonemployees and members of the Board. 2018 Employee Stock Purchase Plan The Company’s 2018 Employee Stock Purchase Plan (the “ESPP”) was adopted by the Board on June 1, 2018 and approved by stockholders on June 7, 2018 and became effective upon the effectiveness of the Company’s Registration Statement on Form S-1. The ESPP is intended to qualify as an “employee stock purchase plan” within the meaning of Section 423(b) of the Code. The ESPP initially reserves and authorizes the issuance of up to a total of 223,200 shares of common stock to participating employees. The ESPP provides that the number of shares reserved and available for issuance will automatically increase each January 1, beginning on January 1, 2019 and each January 1 thereafter through January 1, 2028, by the least of (i) 1% of the outstanding number of shares of our common stock on the immediately preceding December 31; (ii) 1,115,700 shares or (iii) such number of shares as determined by the ESPP administrator (the “ESPP Evergreen”). The number of shares reserved under the ESPP is subject to adjustment in the event of a stock split, stock dividend or other change in our capitalization. During the years ended December 31, 2021 and 2020, the Company issued 27,580 and 13,425 shares, respectively of common stock. The total number of shares of common stock available for future grant was 1,151,010 as of December 31, 2021, which does not include the shares added to the ESPP reserve on January 1, 2022 as a result of the ESPP Evergreen for the year ended December 31, 2021. 2019 Inducement Plan The Company’s 2019 Inducement Plan (the “2019 Plan”) was adopted by the Board on December 11, 2019. The purpose of the 2019 Plan is to allow the Company to grant equity awards to new employees as inducements material to such new employee’s acceptance of employment with the Company. The Company intends that the shares underlying the 2019 Plan be reserved for persons to whom the Company may issue securities without stockholder approval as an inducement pursuant to Rule 5635(c)(4) of the Nasdaq marketplace rules. The Company initially reserved 1,800,000 shares of its common stock for the issuance of awards under the 2019 Plan. The number of shares of common stock available for future grant under the 2019 Plan was 412,686 as of December 31, 2021 . 2020 Inducement Plan The Company’s 2020 Inducement Plan (the “2020 Plan”) was adopted by the Board on December 9, 2020. The purpose of the 2020 Plan is to allow the Company to grant equity awards to new employees as inducements material to such new employee’s acceptance of employment with the Company. The Company intends that the shares underlying the 2020 Plan be reserved for persons to whom the Company may issue securities without stockholder approval as an inducement pursuant to Rule 5635(c)(4) of the Nasdaq marketplace rules. The Company initially reserved 1,700,000 shares of its common stock for the issuance of awards under the 2020 Plan. The number of shares of common stock available for future grant under the 2020 Plan was 1,637,000 as of December 31, 2021. Stock Option Valuation The assumptions that the Company used to determine the grant-date fair value of stock options granted to employees and members of the Board were as follows, presented on a weighted-average basis: Year Ended December 31, 2021 2020 Expected option life (years) 6.06 6.00 Risk-free interest rate 0.80 % 0.76 % Expected volatility 81.22 % 76.02 % Expected dividend yield — % — % The following table summarizes the Company’s stock option activity for the year ended December 31, 2021: Number of Options Weighted- Average Exercise Price Weighted- Average Remaining Contractual Term (Years) Aggregate Intrinsic Value Outstanding as of December 31, 2020 5,559,545 $ 14.97 7.65 $ 13,511 Granted 4,253,232 $ 12.64 Exercised (226,180 ) $ 4.07 Cancelled or forfeited (2,162,820 ) $ 16.55 Outstanding as of December 31, 2021 7,423,777 $ 13.54 6.79 $ 1,468,969 Exercisable as of December 31, 2021 2,690,230 $ 12.87 5.69 $ 1,466,939 The aggregate intrinsic value of stock options is calculated as the difference between the exercise price of the underlying stock options and the estimated fair value of the Company’s common stock for those stock options that had exercise prices lower than the estimated fair value of the Company’s common stock. The aggregate intrinsic value of options exercised during the years ended December 31, 2021 and 2020 was $1,826 and $2,077, respectively. The weighted-average grant-date fair value of the Company’s stock options granted during the years ended December 31, 2021 and 2020 was $8.70 and $12.28, respectively. Restricted Common Stock The Company has granted restricted common stock (or restricted stock awards) with time-based vesting conditions to certain employees of the Company. shares of restricted stock awards may not be sold or transferred by the holder. These restrictions lapse according to the time-based vesting conditions of each award. The Company has the option to repurchase the restricted stock awards at the original purchase price if the grantee terminates its working relationship with the Company prior to the vesting date. There were no unvested restricted stock awards as of December 31, 2021 . Restricted stock units Restricted stock units represent an unsecured promise to grant at no cost a set number of shares of common stock upon vesting. With respect to restricted stock units, recipients are not entitled to cash dividends and have no voting rights during the vesting period. The following table summarizes the Company’s restricted stock award and restricted stock unit activity for the year ended December 31, 2021: Number of Shares Weighted- Average Grant Date Fair Value Issued and unvested as of December 31, 2020 1,198 $ 15.65 Granted 771,600 $ 9.63 Vested (575 ) $ 15.65 Forfeited, cancelled or expired (172,373 ) $ 9.63 Issued and unvested as of December 31, 2021 599,850 $ 9.64 The total fair value of restricted stock awards and restricted stock units vested during the years ended December 31, 2021 and 2020 was $5 and $29, respectively. Stock-Based Compensation Stock-based compensation expense was allocated as follows: Year Ended December 31, 2021 2020 Research and development $ 6,996 $ 8,207 General and administrative 11,583 7,421 Total stock-based compensation expense $ 18,579 $ 15,628 As of December 31, 2021, total unrecognized compensation cost related to unvested stock-based awards was $43,331, which is expected to be recognized over a weighted-average period of 2.82 years. As of December 31, 2020, the Company modified certain awards in conjunction with employee terminations. The modification provided for the extension of the post-employment exercise period. The modifications resulted in approximately $417 of additional research and development expenses for the year ended December 31, 2020. |
401(k) Savings Plan
401(k) Savings Plan | 12 Months Ended |
Dec. 31, 2021 | |
Compensation And Retirement Disclosure [Abstract] | |
401(k) Savings Plan | 10. 401(k) Savings Plan The Company established a defined contribution savings plan under Section 401(k) of the Internal Revenue Code. Eligible employees may make pretax contributions to the 401(k) Plan up to statutory limits. At the election of the Board, the Company may elect to match employee contributions. Currently, the Company makes matching contributions at a rate of 50% of the first 6% of employee contributions. The Company recorded $593 and $468 of expenses related to its 401(k) match for the years ended December 31, 2021 and 2020, respectively. |
Income Taxes
Income Taxes | 12 Months Ended |
Dec. 31, 2021 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | 11. Income Taxes For the years ended December 31, 2021 and 2020, the Company did not record a current or deferred income tax expense or (benefit) due to current and historical losses incurred by the Company. The Company’s operations are predominantly based in the United States and the Company’s foreign subsidiaries generated de minimis A reconciliation of income tax expense (benefit) computed at the statutory federal income tax rate to the Company’s effective tax rate as reflected in the consolidated financial statements is as follows: Year Ended December 31, 2021 2020 Federal income tax expense at statutory rate 21.0 % 21.0 % State income taxes, net of federal benefit 6.0 % 5.6 % Permanent differences -1.2 % -0.6 % Foreign rate differential 0.1 % 0.1 % Research and development tax credits 2.0 % 2.3 % Change in valuation allowance -27.9 % -28.3 % Provision to Return 0.0 % -0.1 % Effective income tax rate 0.0 % 0.0 % Deferred taxes are recognized for temporary differences between the basis of assets and liabilities for financial statement and income tax purposes. The significant components of the Company’s deferred tax assets and liabilities are comprised of the following: December 31, 2021 2020 Deferred tax assets: U.S., foreign and state net operating loss carryforwards $ 84,102 $ 57,705 Research and development credits 7,821 4,383 Capitalized start up and organizational costs 26 29 Equity based compensation 3,926 2,944 Licensing agreements 3,749 4,004 Accruals and other 1,376 1,587 Total deferred tax assets 101,000 70,652 Valuation allowance (100,893 ) (70,492 ) Net deferred tax assets $ 107 $ 160 Deferred tax liabilities: Property and equipment $ (107 ) $ (160 ) Total deferred tax liabilities $ — $ — The Company has evaluated the positive and negative evidence bearing upon the realizability of its deferred tax assets. As of December 31, 2021 and 2020, based on the Company’s history of operating losses, the Company has concluded that it is not more likely than not that the benefit of its deferred tax assets will be realized. Accordingly, the Company has provided a full valuation allowance for deferred tax assets as of December 31, 2021 and 2020. The valuation allowance increased by $30,401 and $32,066 during the years ended December 31, 2021 and 2020, respectively, due primarily to net operating losses generated. As of December 31, 2021 and 2020, the Company had U.S. federal net operating loss carryforwards of $312,967 and $213,991, respectively, that may be available to offset future income tax liabilities. The U.S. federal tax operating loss carryforwards of approximately $17,743 will expire at various dates through 2037. Approximately $295,224 of the U.S. federal tax operating losses can be carried forward indefinitely. As of December 31, 2021 and 2020, the Company also had U.S. state net operating loss carryforwards of $290,500 and $201,642, respectively, which may be available to offset future taxable income. These losses expire at various dates beginning in 2041. As of December 31, 2021 and 2020, the Company had federal research and development tax credit carryforwards of $6,234 and $3,677, respectively. Included in the $6,234 of federal tax credit carryforwards are $1,959 of orphan drug credits. Through the year ended December 31, 2020 the Company qualifies for, and has elected to, apply part of its federal research credits against its payroll tax liability in accordance with certain provisions of the Internal Revenue Code. The amount applied towards the Company’s payroll tax liability is capped at $250 per year. The federal research credits generated in excess of the $250 cap are able to be carried forward for 20 years. As of December 31, 2021 and 2020, the Company had state research and development tax credit carryforwards of approximately $1,959 and $894, respectively, available to reduce future tax liabilities which expire at various dates beginning in 2037. For all years through December 31, 2021, the Company generated research credits but has not conducted a study to document the qualified activities. This study may result in an adjustment to the Company’s research and development credit carryforwards. As the Company carries out extensive research and development activities, it seeks to benefit from the Australian research and development tax credit cash rebate regime. Under this regime, the Company’s Australian subsidiary may be eligible to surrender the trading losses that arise from its research and development activities for a payable tax credit of up to approximately 43.5% of eligible research and development expenditures. Qualifying expenditures largely comprise employment costs for research staff, consumables, certain internal overhead costs and subcontracted expenditures as part of research projects for which the Company does not receive income. For the year ended December 31, 2021 and 2020, the Company recorded $1,210 and $589, respectively, in research and development tax credits as an offset to research and development expenses in the consolidated statement of operations and comprehensive loss. Under the provisions of the Internal Revenue Code, the net operating loss and tax credit carryforwards are subject to review and possible adjustment by the Internal Revenue Service and state tax authorities. Net operating loss and tax credit carryforwards may become subject to an annual limitation in the event of certain cumulative changes in the ownership interest of significant shareholders over a three-year The Company files income tax returns in the United States, Australia and Canada, and in several states. The foreign, federal and state income tax returns are generally subject to tax examinations for the tax years ended December 31, 2016 through December 31, 2020. To the extent the Company has tax attribute carryforwards, the tax years in which the attribute was generated may still be adjusted upon examination by foreign tax authorities, the Internal Revenue Service, or state tax authorities to the extent utilized in a future period. |
Net Loss per Share
Net Loss per Share | 12 Months Ended |
Dec. 31, 2021 | |
Earnings Per Share [Abstract] | |
Net Loss per Share | 12. Net Loss per Share For purposes of the diluted net loss per share calculation, stock options, unvested restricted stock awards and unvested restricted stock units are considered to be common stock equivalents but have been excluded from the calculation of diluted net loss per share, as their effect would be anti-dilutive for all periods presented. Therefore, the weighted-average number of common shares outstanding used to calculate both basic and diluted net loss per share attributable to common stockholders is the same. The following potentially dilutive common stock equivalents, presented based on amounts outstanding at each period end, were excluded from the computation of diluted net loss per share attributable to common stockholders for the periods indicated: Year Ended December 31, 2021 2020 Options to purchase common stock 7,423,777 5,559,545 Restricted stock awards and units 599,850 1,198 |
Related Party Transactions
Related Party Transactions | 12 Months Ended |
Dec. 31, 2021 | |
Related Party Transactions [Abstract] | |
Related Party Transactions | 13. Related Party Transactions UHN In connection with the Company’s entry into a license agreement with UHN on January 27, 2016, the Company issued UHN 1,161,665 shares of its common stock. Upon the closing of the IPO in 2018, as UHN’s fully-diluted percentage ownership of the Company was reduced within a range of specified percentages, the Company was obligated to pay UHN an amount of $2,000, which was paid in July 2018. For the years ended December 31, 2021 and 2020, the Company recognized $248 and $381, respectively, of research and development expense related to the license agreements with UHN. Refer to Note 3 “License Agreements” Others For the years ended December 31, 2021 and 2020, the Company recorded expenses of $1,523 and $1,488, respectively, related to a sublease to rent lab space, provided by an entity affiliated with a member of the board. |
Subsequent Events
Subsequent Events | 12 Months Ended |
Dec. 31, 2019 | |
Subsequent Events [Abstract] | |
Subsequent Events | 14. Subsequent Events The Company has completed an evaluation of all subsequent events after the audited balance sheet date of December 31, 2021 through the filing date of this Annual Report on Form 10-K with the SEC, to ensure that these consolidated financial statements include appropriate disclosure of events both recognized in the consolidated financial statements as of December 31, 2021, and events which occurred subsequently but were not recognized in the consolidated financial statements. The Company has concluded that no subsequent events have occurred that require disclosure, except as disclosed within these consolidated financial statements and as described below. In January 2022 the Company announced the deprioritization of AVR-RD-01, its investigational gene therapy program for Fabry disease. This decision was made due to several factors, including new clinical data showing variable engraftment patterns from the five most recently dosed patients in the Company’s Phase 2 clinical trial of AVR-RD-01 for the treatment of Fabry disease, which the Company refers to as the FAB-GT clinical trial. The emergence of such new data would have significantly extended the program’s development timeline. That development, coupled with an increasingly challenging market and regulatory environment for Fabry disease, were among the primary factors leading to the Company’s deprioritization of its Fabry program. As a result of the deprioritization, the Company has stopped enrollment of its Phase 2 FAB-GT clinical trial and will focus on its other pipeline programs. In connection with the deprioritization of AVR-RD-01 noted above, in January, 2022, the Company approved changes to the Company’s organization as well as a broader operational cost reduction plan. As part of this plan, the Company approved a reduction in the Company’s workforce by approximately 23% across different areas and functions in the Company (the “Workforce Reduction”). The Workforce Reduction is expected to be substantially complete in the first quarter of 2022. Affected employees were offered separation benefits, including severance payments. The Company estimates that the severance and termination-related costs will total approximately $2.5 million in the aggregate and expects to record these charges in the first quarter of 2022. The Company expects that payments of these costs will be substantially complete through the end of the first quarter of 2022. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 12 Months Ended |
Dec. 31, 2021 | |
Accounting Policies [Abstract] | |
Basis of presentation | Basis of Presentation The accompanying consolidated financial statements have been prepared in conformity with accounting principles generally accepted in the United States of America (“GAAP”). Any reference in these notes to applicable guidance is meant to refer to the authoritative United States generally accepted accounting principles as found in the Accounting Standards Codification (“ASC”) and Accounting Standards Update (“ASU”) of the Financial Accounting Standards Board (“FASB”). |
Principles of Consolidation | Principles of Consolidation The accompanying consolidated financial statements include the accounts of AVROBIO, Inc. and its wholly owned subsidiaries. All intercompany transactions and balances have been eliminated in consolidation. |
Segment Information | Segment Information Operating segments are identified as components of an enterprise about which separate discrete financial information is available for evaluation by the chief operating decision maker, or decision-making group, in making decisions on how to allocate resources and assess performance. The Company’s chief operating decision maker is the chief executive officer (“CEO”). The Company and the CEO view the Company’s operations and manage its business as one operating segment. All material long-lived assets of the Company reside in the United States. |
Use of Estimates | Use of Estimates The preparation of financial statements in conformity with GAAP requires that the Company make estimates and judgments that may affect the reported amounts of assets, liabilities and expenses and the related disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of expenses during the reporting periods On an on ‑going basis, the Company evaluates its estimates, judgments and methodologies. The Company bases its estimates on historical experience and on various other assumptions that are believed to be reasonable, the results of which form the basis for making judgments about the carrying values of assets and liabilities. Actual results may differ from these estimates. Changes in estimates are reflected in reported results in the period in which they become known. |
Cash and Cash Equivalents | Cash and Cash Equivalents The Company considers all highly liquid investments purchased with original maturities of three months or less at acquisition to be cash equivalents. As of December 31, 2021 and 2020, |
Concentrations of Credit Risk | Concentrations of Credit Risk The Company has no significant off-balance sheet risk, such as foreign exchange contracts, option contracts, or other foreign hedging arrangements. Financial instruments that potentially expose the Company to concentrations of credit risk consist primarily of cash, cash equivalents and restricted cash. Periodically, the Company maintains deposits in accredited financial institutions in excess of federally insured limits. The Company deposits its cash and cash equivalents in financial institutions that it believes have high credit quality and has not experienced any losses on such accounts and does not believe it is exposed to any unusual credit risk beyond the normal credit risk associated with commercial banking relationships. |
Fair Value Measurements | Fair Value Measurements Certain assets and liabilities of the Company are carried at fair value under GAAP. Fair value is defined as the exchange price that would be received for an asset or paid to transfer a liability (an exit price) in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants on the measurement date. Valuation techniques used to measure fair value must maximize the use of observable inputs and minimize the use of unobservable inputs. Financial assets and liabilities carried at fair value are to be classified and disclosed in one of the following three levels of the fair value hierarchy, of which the first two are considered observable and the last is considered unobservable: • Level 1— Fair values are determined utilizing prices (unadjusted) in active markets for identical assets or liabilities that the Company has the ability to access. • Level 2—Observable inputs (other than Level 1 quoted prices), such as quoted prices in active markets for similar assets or liabilities, quoted prices in markets that are not active for identical or similar assets or liabilities, or other inputs that are observable or can be corroborated by observable market data. • Level 3—Unobservable inputs that are supported by little or no market activity that are significant to determining the fair value of the assets or liabilities, including pricing models, discounted cash flow methodologies and similar techniques. The carrying amounts of the Company’s financial instruments, which include cash equivalents, accounts payable, and accrued expenses, approximated their fair values as of December 31, 2021 and 2020 due to the short‑term nature of these instruments. The Company has evaluated the estimated fair value of financial instruments using available market information. The use of different market assumptions, estimation methodologies, or both, could have a significant effect on the estimated fair value amounts. See Note 4 “ Fair Value of Financial Assets and Liabilities” |
Property and Equipment | Property and Equipment Property and equipment are recorded at cost. Depreciation and amortization is calculated using the straight-line method over the following estimated useful lives of the assets: Estimated Useful Life Laboratory and office equipment 5 years Computer equipment 2 years Leasehold improvements Lesser of lease term or 10 years |
Impairment of Long-Lived Assets | Impairment of Long-Lived Assets Long-lived assets consist of property and equipment. Long-lived assets to be held and used are tested for recoverability whenever events or changes in business circumstances indicate that the carrying amount of the assets may not be fully recoverable. Factors that the Company considers in deciding when to perform an impairment review include significant underperformance of the business in relation to expectations, significant negative industry or economic trends and significant changes or planned changes in the use of the assets. If an impairment review is performed to evaluate a long-lived asset group for recoverability, the Company compares forecasts of undiscounted cash flows expected to result from the use and eventual disposition of the long-lived asset group to its carrying value. An impairment loss would be recognized when estimated undiscounted future cash flows expected to result from the use of an asset group are less than its carrying amount. The impairment loss would be based on the excess of the carrying value of the impaired asset group over its fair value, determined based on discounted cash flows. The Company did not record any impairment loss during the years ended . |
Leases | Leases The Company categorizes leases at their inception as either operating or capital leases. On certain lease arrangements, the Company may receive rent holidays or other incentives. The Company recognizes lease costs on a straight-line basis once control of the space is achieved, without regard to deferred payment terms, such as rent holidays, that defer the commencement date of required payments or escalating payment amounts. The difference between required lease payments and rent expense has been recorded as deferred rent and accrued expenses and other current liabilities in the accompanying consolidated balance sheets. Additionally, incentives received are treated as a reduction of costs over the term of the agreement, as they are considered an inseparable part of the lease agreement. |
Comprehensive Income (Loss) | Comprehensive Income (Loss) Comprehensive income (loss) is defined as the change in stockholders’ equity of a business enterprise during a period from transactions and other events and circumstances from non-owner sources. Comprehensive income (loss) includes net income (loss) as well as other changes in stockholders’ equity (deficit) which includes certain changes in equity that are excluded from net income (loss). Comprehensive loss has been disclosed in the accompanying consolidated statements of operations and comprehensive loss and equals the Company’s net loss for all periods presented. |
Foreign Currency Translation | Foreign Currency Translation The functional currency of the Company’s international operations in Canada and Australia is the U.S. dollar. Accordingly, all operating assets and liabilities of these international subsidiaries are remeasured into U.S. dollars using the exchange rates in effect at the balance sheet date or historical rates, as appropriate, while expenses are remeasured into U.S. dollars at the average rates in effect during the period. Any differences resulting from the remeasurement of assets, liabilities, and operations of the Canadian and Australian subsidiaries are recorded within other (expense) income, net in the consolidated statements of operations and comprehensive loss. During the years ended December 31, 2021 and 2020, the Company recorded foreign exchange losses of $61 and $203, respectively, in other expense |
Research and Development Expenses | Research and Development Expenses Research and development costs are expensed as incurred. Research and development expenses consist of costs incurred in performing research and development activities, including salaries, stock-based compensation and benefits, facilities costs, depreciation, third-party license fees, and external costs of outside vendors engaged to conduct preclinical development activities and clinical trials as well as to manufacture research and development materials. Non-refundable prepayments for goods or services that will be used or rendered for future research and development activities are deferred and capitalized. Such amounts are recognized as an expense as the goods are delivered or the related services are performed or until it is no longer expected that the goods will be delivered or the services rendered. The Company has entered into various research and development related contracts with parties both inside and outside of the United States. The payments related to these agreements are recorded as research and development expenses as incurred. The Company records accrued liabilities for estimated ongoing research costs. When evaluating the adequacy of the accrued liabilities, the Company analyzes progress of the studies or clinical trials, including the phase or completion of events, invoices received and contracted costs. Significant judgments and estimates are made in determining the accrued balances at the end of any reporting period. Actual results could differ from the Company’s estimates. The Company’s historical accrual estimates have not been materially different from the actual costs. |
Stock-Based Compensation | Stock-Based Compensation For stock-based awards issued to employees and members of the Company’s board of directors (the “Board”) for their services on the Board, the Company measures the estimated fair value of the stock-based award on the date of grant and recognizes compensation expense for those awards over the requisite service period, which is generally the vesting period of the respective award. The Company issues stock-based awards with only service-based vesting conditions and records the expense for these awards using the straight-line method. The Company has not issued any stock-based awards with performance- or market-based vesting conditions. The Company accounts for forfeitures as they occur. Prior to the adoption of Accounting Standards Update (“ASU”) No. 2018-07, Compensation-Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting The Company classifies stock-based compensation expense in its consolidated statements of operations and comprehensive loss in the same manner in which the award recipient’s cash compensation costs are classified. Given the absence of an active market for the Company’s common stock prior to its initial public offering (“IPO”), the Company and the Board, the members of which the Company believes have extensive business, finance, and venture capital experience, were required to estimate the fair value of the Company’s common stock at the time of each grant of a stock-based award. The Company and the Board determined the estimated fair value of the Company’s equity instruments based on a number of factors, including external market conditions affecting the biotechnology industry sector. The Company and the Board utilized various valuation methodologies in accordance with the framework of the American Institute of Certified Public Accountants’ Technical Practice Aid, Valuation of Privately-Held Company Equity Securities Issued as Compensation The fair value of each stock option award is estimated on the date of grant using the Black-Scholes option pricing model. As there was no public market for its common stock prior to June 21, 2018, which was the first day of trading, and as the trading history of the Company’s common stock was limited through December 31, 2021, the Company determined the volatility for awards granted based on an analysis of reported data for a group of guideline companies that issued options with substantially similar terms. The expected volatility has been determined using a weighted-average of the historical volatility measures of this group of guideline companies. The Company expects to continue to do so until such time as it has adequate historical data regarding the volatility of its own traded stock price. The expected term of the Company’s stock options has been determined utilizing the “simplified” method for awards that qualify as “plain-vanilla” options. The risk-free interest rate is determined by reference to the U.S. Treasury yield curve in effect at the time of grant of the award for time periods approximately equal to the expected term of the award. The Company has not paid, and does not anticipate paying, cash dividends on its common stock; therefore, the expected dividend yield is assumed to be zero. |
Income Taxes | Income Taxes Deferred tax assets and liabilities are determined on the basis of the differences between the consolidated financial statements and tax basis of assets and liabilities using enacted tax rates in effect for the year in which the differences are expected to reverse. Changes in deferred tax assets and liabilities are recorded in the provision for income taxes. The Company assesses the likelihood that its deferred tax assets will be recovered from future taxable income and, to the extent it believes, based upon the weight of available evidence, that it is more likely than not that all or a portion of the deferred tax assets will not be realized, a valuation allowance is established. The Company accounts for uncertain tax positions recognized in the consolidated financial statements by prescribing a more-likely-than-not threshold for financial statement recognition and measurement of a tax position taken or expected to be taken in a tax return. 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 Basic net loss per share is computed by dividing net loss by the weighted-average number of shares of common stock outstanding during the period, without consideration of potential dilutive securities. Diluted net loss per share is computed by adjusting the weighted-average shares outstanding for the potential dilutive effects of common stock equivalents outstanding during the period calculated in accordance with the treasury stock method. For purposes of the diluted net loss per share calculation, stock options and restricted stock units are considered to be common stock equivalents but have been excluded from the calculation of diluted net loss per share, as their effect would be anti-dilutive for all periods presented. Therefore, basic and diluted net loss per share were the same for all periods presented. |
Subsequent Event Considerations | Subsequent Event Considerations The Company considers events or transactions that occur after the balance sheet date but prior to the issuance of the consolidated financial statements to provide additional evidence for certain estimates or to identify matters that require additional disclosure. Subsequent events have been evaluated as required. See Note 14. |
Emerging Growth Company Status | Emerging Growth Company Status The Company is an “emerging growth company,” as defined in the Jumpstart Our Business Startups Act, or JOBS Act, and may take advantage of certain exemptions from various reporting requirements that are applicable to other public companies that are not emerging growth companies. The Company may take advantage of these exemptions until the Company is no longer an “emerging growth company.” Section 107 of the JOBS Act provides that an “emerging growth company” can take advantage of the extended transition period afforded by the JOBS Act for the implementation of new or revised accounting standards. The Company has elected to use the extended transition period for complying with new or revised accounting standards and as a result of this election, its consolidated financial statements may not be comparable to companies that comply with public company effective dates. The Company may take advantage of these exemptions up until the last day of the fiscal year following the fifth anniversary of an offering or such earlier time that it is no longer an “emerging growth company.” |
Recently Issued Accounting Pronouncements | Recently Issued Accounting Pronouncements In February 2016, the FASB issued ASU No. 2016-02, (Topic 842) Leases, he Company currently expects that its operating lease commitments will be subject to the new standard and recognized as right-of-use assets and operating lease liabilities upon adoption of this standard, which will increase the Company’s total assets and total liabilities. In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments—Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments In Codification Improvements to Topic 326, Financial Instruments – Credit Losses Financial Instruments – Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments As a result of the Company having elected the extended transition period for complying with new or revised accounting standards pursuant to Section 107(b) of the JOBS Act, 2016-13 and ASU 2019-11 are effective for the Company for fiscal years beginning after December 15, 2022, and interim periods within those fiscal years. In December 2019, the FASB issued ASU No. 2019-12, Simplifying the Accounting for Income Taxes |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Accounting Policies [Abstract] | |
Schedule of Property and Equipment Estimated Useful Life | Property and equipment are recorded at cost. Depreciation and amortization is calculated using the straight-line method over the following estimated useful lives of the assets: Estimated Useful Life Laboratory and office equipment 5 years Computer equipment 2 years Leasehold improvements Lesser of lease term or 10 years |
Fair Value of Financial Asset_2
Fair Value of Financial Assets and Liabilities (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Fair Value Disclosures [Abstract] | |
Summary of Financial Assets and Liabilities Measured at Fair Value on Recurring Basis | The following table presents information about the Company’s financial assets and liabilities measured at fair value on a recurring basis and indicates the level of the fair value hierarchy utilized to determine such fair values as of December 31, 2021 and 2020: Fair Value Measurements as of December 31, 2021 Level 1 Level 2 Level 3 Total Assets: Cash equivalents—money market funds $ 189,332 $ — $ — $ 189,332 $ 189,332 $ — $ — $ 189,332 Fair Value Measurements as of December 31, 2020 Level 1 Level 2 Level 3 Total Assets: Cash equivalents—money market funds $ 44,416 $ — $ — $ 44,416 $ 44,416 $ — $ — $ 44,416 |
Supplemental Balance Sheet In_2
Supplemental Balance Sheet Information (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Balance Sheet Related Disclosures [Abstract] | |
Summary of Prepaid Expenses and Other Current Assets | Prepaid expenses and other current assets consisted of the following: December 31, 2021 2020 Tax incentive refund $ 2,697 $ 2,558 Prepaid research and development expenses 4,496 3,145 Prepaid insurance 112 973 Prepaid compensation benefits 575 609 Interest income receivable — 4 Other current assets 1,698 271 Prepaid expenses and other current assets $ 9,578 $ 7,560 |
Summary of Property and Equipment, Net | Property and equipment, net consisted of the following: December 31, 2021 2020 Laboratory and office equipment $ 6,162 $ 3,965 Leasehold improvements 1,635 1,377 Computer equipment 149 143 7,946 5,485 Less: Accumulated depreciation and amortization (3,820 ) (2,421 ) Property and equipment, net $ 4,126 $ 3,064 |
Summary of Restricted Cash | As of December 31, 2021 and 2020, the Company had restricted cash as presented in the table below, which consists of cash used to secure letters of credit for the benefit of the landlord in connection with the Company’s lease agreements. The cash will be restricted until the termination or modification of the lease arrangement. December 31, 2021 2020 Restricted cash $ 492 $ 492 |
Summary of Accrued Expenses | Accrued expenses consisted of the following: December 31, 2021 2020 Compensation and benefit costs $ 5,579 $ 6,402 Research and development expenses 8,882 5,847 Consulting and professional fees 999 1,096 Other liabilities 178 348 $ 15,638 $ 13,693 |
Commitments and Contingencies (
Commitments and Contingencies (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Commitments And Contingencies Disclosure [Abstract] | |
Summary of Future Minimum Lease Payments Due Under Operating Leases | The following table summarizes the future minimum lease payments due under operating leases as of December 31, 2021: Year Ending December 31, 2022 $ 1,297 2023 208 2024 147 2025 73 $ 1,725 |
Note Payable (Tables)
Note Payable (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Debt Disclosure [Abstract] | |
Carrying Value of Note Payable | As of December 31, 2021 the carrying value of the note payable consists of the following: December 31, 2021 (in thousands) Note payable, including End of Term Charge 16,350 Debt discount, net of accretion (1,405 ) Note payable, net of discount, long-term 14,945 |
Future Principal Payments | As of December 31, 2021, the future principal payments due under the arrangement, excluding interest and the end of term charge, are as follows: Year Ending December 31, Principle 2022 $ — 2023 — 2024 1,875 2025 7,500 2026 5,625 Total $ 15,000 |
Common Stock (Tables)
Common Stock (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Equity [Abstract] | |
Summary of Common Stock Reserved for Future Issuance | As of December 31, 2021 and 2020, the Company has reserved the following shares of common stock for future issuance: December 31, 2021 2020 Shares reserved for exercise of outstanding stock options 7,423,777 5,559,545 Shares reserved for vesting of restricted stock units 599,850 1,198 Shares reserved for issuance under the 2018 Stock Option and Incentive Plan 2,583,736 3,877,478 Shares reserved for issuance under the 2018 Employee Stock Purchase Plan 1,151,010 762,900 Shares reserved for issuance under the 2019 Inducement Plan 412,686 365,050 Shares reserved for issuance under the 2020 Inducement Plan 1,637,000 1,700,000 Total shares of authorized common stock reserved for future issuance 13,808,059 12,266,171 |
Stock-Based Compensation (Table
Stock-Based Compensation (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Summary of Assumptions that Used to Determine Grant-Date Fair Value of Stock Options Granted to Employees and Members of Board | The assumptions that the Company used to determine the grant-date fair value of stock options granted to employees and members of the Board were as follows, presented on a weighted-average basis: Year Ended December 31, 2021 2020 Expected option life (years) 6.06 6.00 Risk-free interest rate 0.80 % 0.76 % Expected volatility 81.22 % 76.02 % Expected dividend yield — % — % |
Summary of Stock Option Activity | The following table summarizes the Company’s stock option activity for the year ended December 31, 2021: Number of Options Weighted- Average Exercise Price Weighted- Average Remaining Contractual Term (Years) Aggregate Intrinsic Value Outstanding as of December 31, 2020 5,559,545 $ 14.97 7.65 $ 13,511 Granted 4,253,232 $ 12.64 Exercised (226,180 ) $ 4.07 Cancelled or forfeited (2,162,820 ) $ 16.55 Outstanding as of December 31, 2021 7,423,777 $ 13.54 6.79 $ 1,468,969 Exercisable as of December 31, 2021 2,690,230 $ 12.87 5.69 $ 1,466,939 |
Summary of Restricted Stock Award and Restricted Stock Unit Activity | The following table summarizes the Company’s restricted stock award and restricted stock unit activity for the year ended December 31, 2021: Number of Shares Weighted- Average Grant Date Fair Value Issued and unvested as of December 31, 2020 1,198 $ 15.65 Granted 771,600 $ 9.63 Vested (575 ) $ 15.65 Forfeited, cancelled or expired (172,373 ) $ 9.63 Issued and unvested as of December 31, 2021 599,850 $ 9.64 |
Schedule of Stock-based Compensation Expense | Stock-based compensation expense was allocated as follows: Year Ended December 31, 2021 2020 Research and development $ 6,996 $ 8,207 General and administrative 11,583 7,421 Total stock-based compensation expense $ 18,579 $ 15,628 |
Income Taxes (Tables)
Income Taxes (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Income Tax Disclosure [Abstract] | |
Schedule of Reconciliation of Income Tax Expense (Benefit) | A reconciliation of income tax expense (benefit) computed at the statutory federal income tax rate to the Company’s effective tax rate as reflected in the consolidated financial statements is as follows: Year Ended December 31, 2021 2020 Federal income tax expense at statutory rate 21.0 % 21.0 % State income taxes, net of federal benefit 6.0 % 5.6 % Permanent differences -1.2 % -0.6 % Foreign rate differential 0.1 % 0.1 % Research and development tax credits 2.0 % 2.3 % Change in valuation allowance -27.9 % -28.3 % Provision to Return 0.0 % -0.1 % Effective income tax rate 0.0 % 0.0 % |
Schedule of Deferred Tax Assets and Liabilities | Deferred taxes are recognized for temporary differences between the basis of assets and liabilities for financial statement and income tax purposes. The significant components of the Company’s deferred tax assets and liabilities are comprised of the following: December 31, 2021 2020 Deferred tax assets: U.S., foreign and state net operating loss carryforwards $ 84,102 $ 57,705 Research and development credits 7,821 4,383 Capitalized start up and organizational costs 26 29 Equity based compensation 3,926 2,944 Licensing agreements 3,749 4,004 Accruals and other 1,376 1,587 Total deferred tax assets 101,000 70,652 Valuation allowance (100,893 ) (70,492 ) Net deferred tax assets $ 107 $ 160 Deferred tax liabilities: Property and equipment $ (107 ) $ (160 ) Total deferred tax liabilities $ — $ — |
Net Loss per Share (Tables)
Net Loss per Share (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Earnings Per Share [Abstract] | |
Schedule of Dilutive Common Stock Equivalents Excluded from Computation of Diluted Net Loss per Share | The following potentially dilutive common stock equivalents, presented based on amounts outstanding at each period end, were excluded from the computation of diluted net loss per share attributable to common stockholders for the periods indicated: Year Ended December 31, 2021 2020 Options to purchase common stock 7,423,777 5,559,545 Restricted stock awards and units 599,850 1,198 |
Nature of the Business - Additi
Nature of the Business - Additional Information (Detail) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | ||
Accumulated deficit | $ (383,542) | $ (264,416) |
Cash and cash equivalents | $ 189,567 | $ 259,682 |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies - Additional Information (Detail) | 12 Months Ended | |
Dec. 31, 2021USD ($)Segment | Dec. 31, 2020USD ($) | |
Significant Of Accounting Policies [Line Items] | ||
Number of operating segment | Segment | 1 | |
Impairment loss of long-lived assets | $ 0 | $ 0 |
Foreign exchange losses | $ 61,000 | $ 203,000 |
Expected Dividend Yield [Member] | ||
Significant Of Accounting Policies [Line Items] | ||
Expected cash dividend yield | 0 |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies - Schedule of Property and Equipment Estimated Useful Life (Detail) | 12 Months Ended |
Dec. 31, 2021 | |
Laboratory and Office Equipment [Member] | |
Property, Plant and Equipment [Line Items] | |
Property and equipment estimated useful life | 5 years |
Computer Equipment [Member] | |
Property, Plant and Equipment [Line Items] | |
Property and equipment estimated useful life | 2 years |
Leasehold Improvements [Member] | |
Property, Plant and Equipment [Line Items] | |
Property and equipment estimated useful life | 10 years |
Property and equipment estimated useful life | Lesser of lease term |
License Agreements - Additional
License Agreements - Additional Information (Detail) £ in Thousands, $ in Thousands | Sep. 30, 2020USD ($) | Sep. 30, 2020GBP (£) | Oct. 02, 2017USD ($) | Jan. 27, 2016USD ($) | Jan. 27, 2016CAD ($)shares | Aug. 31, 2017USD ($)shares | Dec. 31, 2021USD ($)shares | Dec. 31, 2020USD ($)shares | Dec. 31, 2018USD ($) | Nov. 17, 2016USD ($) |
License Agreement [Line Items] | ||||||||||
Collaborative agreement expenses | $ 1,437,000 | $ 565,000 | ||||||||
Common stock, issued | shares | 41,569,000 | 43,652,000 | ||||||||
Research and development | $ 83,114,000 | $ 87,236,000 | ||||||||
Preferred stock issued | shares | 0 | 0 | ||||||||
University of Manchester Agreement [Member] | MPSII License Agreement [Member] | ||||||||||
License Agreement [Line Items] | ||||||||||
Upfront license fee | $ 8,000,000 | |||||||||
Milestone payments payable | $ 80,000,000 | |||||||||
Milestone payment paid | 2,000,000 | |||||||||
Milestone payment due | $ 4,000,000 | |||||||||
Agreement Expiration Period | 15 years | |||||||||
Agreement Expiration Description | upon the later of 15 years from the effective date or the expiration of the last valid claim of the licensed patents | |||||||||
Research and development expense | £ | £ 9,900 | |||||||||
UHN Agreement [Member] | ||||||||||
License Agreement [Line Items] | ||||||||||
Research and development expense | $ 209,000 | 344,000 | ||||||||
Milestone fees | 0 | 0 | ||||||||
UHN Agreement [Member] | Fabry License Agreement [Member] | ||||||||||
License Agreement [Line Items] | ||||||||||
Upfront license fee | $ 75 | |||||||||
Milestone payments payable | 2,450 | |||||||||
Option fee | 20 | |||||||||
UHN Agreement [Member] | Interleukin 12 License Agreement [Member] | ||||||||||
License Agreement [Line Items] | ||||||||||
Upfront license fee | 264 | |||||||||
Milestone payments payable | 19,275 | |||||||||
Milestone fees | 0 | 0 | ||||||||
Annual maintenance fees | $ 50 | |||||||||
Common stock, issued | shares | 1,161,665 | |||||||||
Fair value of shares issued | $ 480,000 | |||||||||
Payments upon closing of an initial public offering | $ 2,000,000 | $ 2,000,000 | ||||||||
Research and development | 39,000 | 37,000 | ||||||||
BioMarin Pharmaceutical Inc [Member] | ||||||||||
License Agreement [Line Items] | ||||||||||
Upfront license fee | $ 500,000 | |||||||||
Preferred stock issued | shares | 233,765 | |||||||||
Milestone payments | 13,000,000 | |||||||||
Expenses related to license | 0 | 0 | ||||||||
GenStem Therapeutics Inc [Member] | ||||||||||
License Agreement [Line Items] | ||||||||||
Upfront license fee | $ 1,000,000 | |||||||||
Milestone payments payable | $ 16,000,000 | |||||||||
Expenses related to license | 0 | |||||||||
Lund University Rights Holders Agreement [Member] | ||||||||||
License Agreement [Line Items] | ||||||||||
Milestone payments payable | $ 550,000 | |||||||||
Expenses related to license | $ 0 | $ 0 |
Fair Value of Financial Asset_3
Fair Value of Financial Assets and Liabilities - Summary of Financial Assets and Liabilities Measured at Fair Value on Recurring Basis (Detail) - Fair Value on Recurring Basis [Member] - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Assets Fair Value Disclosure | ||
Assets Fair Value Disclosure | $ 189,332 | $ 44,416 |
Money Market Funds [Member] | Cash Equivalents [Member] | ||
Assets Fair Value Disclosure | ||
Assets Fair Value Disclosure | 189,332 | 44,416 |
Level 1 [Member] | ||
Assets Fair Value Disclosure | ||
Assets Fair Value Disclosure | 189,332 | 44,416 |
Level 1 [Member] | Money Market Funds [Member] | Cash Equivalents [Member] | ||
Assets Fair Value Disclosure | ||
Assets Fair Value Disclosure | $ 189,332 | $ 44,416 |
Fair Value of Financial Asset_4
Fair Value of Financial Assets and Liabilities - Additional Information (Detail) - USD ($) | Dec. 31, 2021 | Dec. 31, 2020 |
Fair Value Disclosures [Abstract] | ||
Fair value input transfer between levels | $ 0 | $ 0 |
Supplemental Balance Sheet In_3
Supplemental Balance Sheet Information - Summary of Prepaid Expenses and Other Current Assets (Detail) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Supplemental Balance Sheet Information Disclosures [Abstract] | ||
Tax incentive refund | $ 2,697 | $ 2,558 |
Prepaid research and development expenses | 4,496 | 3,145 |
Prepaid insurance | 112 | 973 |
Prepaid compensation benefits | 575 | 609 |
Interest income receivable | 4 | |
Other current assets | 1,698 | 271 |
Prepaid expenses and other current assets | $ 9,578 | $ 7,560 |
Supplemental Balance Sheet In_4
Supplemental Balance Sheet Information - Summary of Property and Equipment, Net (Detail) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Property, Plant and Equipment [Line Items] | ||
Property plant and equipment, gross | $ 7,946 | $ 5,485 |
Less: Accumulated depreciation and amortization | (3,820) | (2,421) |
Property and equipment, net | 4,126 | 3,064 |
Laboratory and Office Equipment [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property plant and equipment, gross | 6,162 | 3,965 |
Leasehold Improvements [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property plant and equipment, gross | 1,635 | 1,377 |
Computer Equipment [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property plant and equipment, gross | $ 149 | $ 143 |
Supplemental Balance Sheet In_5
Supplemental Balance Sheet Information - Additional Information (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Supplemental Balance Sheet Information Disclosures [Abstract] | ||
Depreciation and amortization expense | $ 1,399 | $ 1,207 |
Supplemental Balance Sheet In_6
Supplemental Balance Sheet Information - Summary of Restricted Cash (Detail) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Restricted Cash And Investments [Abstract] | ||
Restricted cash | $ 492 | $ 492 |
Supplemental Balance Sheet In_7
Supplemental Balance Sheet Information - Summary of Accrued Expenses (Detail) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Accrued Liabilities Current [Abstract] | ||
Compensation and benefit costs | $ 5,579 | $ 6,402 |
Research and development expenses | 8,882 | 5,847 |
Consulting and professional fees | 999 | 1,096 |
Other liabilities | 178 | 348 |
Accrued expenses | $ 15,638 | $ 13,693 |
Commitments and Contingencies -
Commitments and Contingencies - Additional Information (Detail) $ in Thousands, $ in Thousands | Jan. 01, 2022 | Jun. 09, 2020 | Jun. 01, 2020 | Aug. 31, 2018 | Jan. 12, 2018USD ($) | Dec. 31, 2021USD ($) | Dec. 31, 2020USD ($) | Dec. 31, 2021CAD ($) | Dec. 31, 2020CAD ($) |
Commitments And Contingencies Disclosure [Line Items] | |||||||||
Lease agreement expiration month and year | 2022-04 | 2025-06 | 2020-10 | 2023-01 | |||||
Percentage of annual increase in rent | 6.67% | 3.00% | |||||||
Tenant incentive allowance | $ 842 | ||||||||
Annual lease payments, description | The annual lease payments are fixed for years 1 and 2, and then subject to a 6.67% increase for years 3 through 5 | ||||||||
Rent expense | $ 2,648 | $ 2,352 | |||||||
Subsequent Event [Member] | |||||||||
Commitments And Contingencies Disclosure [Line Items] | |||||||||
Lease agreement expiration month and year | 2023-04 | ||||||||
Percentage of annual increase in rent | 5.00% | ||||||||
Restricted Cash [Member] | |||||||||
Commitments And Contingencies Disclosure [Line Items] | |||||||||
Security deposit in connection with lease | 283 | 283 | |||||||
Other Long-term Assets [Member] | |||||||||
Commitments And Contingencies Disclosure [Line Items] | |||||||||
Security deposit in connection with lease | $ 27 | $ 27 | |||||||
Cambridge, Massachusetts | Restricted Cash [Member] | |||||||||
Commitments And Contingencies Disclosure [Line Items] | |||||||||
Security deposit in connection with lease | $ 209 | $ 209 |
Commitments and Contingencies_2
Commitments and Contingencies - Summary of Future Minimum Lease Payments Due Under Operating Leases (Detail) $ in Thousands | Dec. 31, 2021USD ($) |
Commitments And Contingencies Disclosure [Abstract] | |
2022 | $ 1,297 |
2023 | 208 |
2024 | 147 |
2025 | 73 |
Total | $ 1,725 |
Note Payable - Additional Infor
Note Payable - Additional Information (Details) $ in Thousands | Nov. 02, 2021USD ($)Tranche | Dec. 31, 2021USD ($) |
Debt Disclosure [Line Items] | ||
Debt Issuance Costs, Net | $ 1,405 | |
Term Loan Facility [Member] | Silicon Valley Bank [Member] | ||
Debt Disclosure [Line Items] | ||
Loan maturity date | Oct. 1, 2026 | |
End of term charge as percentage on principal amount | 9.00% | |
Loan, interest rate | 8.10% | |
Term Loan Facility [Member] | Silicon Valley Bank [Member] | Prime Rate [Member] | ||
Debt Disclosure [Line Items] | ||
Loan, interest rate | 4.85% | |
Term Loan Prepayment During First Year [Member] | Silicon Valley Bank [Member] | ||
Debt Disclosure [Line Items] | ||
Line of credit facility prepayment of premium percentage | 1.50% | |
Term Loan Prepayment During Second Year [Member] | Silicon Valley Bank [Member] | ||
Debt Disclosure [Line Items] | ||
Line of credit facility prepayment of premium percentage | 1.00% | |
Term Loan Prepayment After Second Year [Member] | Silicon Valley Bank [Member] | ||
Debt Disclosure [Line Items] | ||
Line of credit facility prepayment of premium percentage | 0.00% | |
Loan Agreement [Member] | Silicon Valley Bank [Member] | ||
Debt Disclosure [Line Items] | ||
Loan, interest rate | 11.12% | |
Debt discount | $ 1,350 | |
Loan Agreement [Member] | Silicon Valley Bank [Member] | Legal Expenses [Member] | ||
Debt Disclosure [Line Items] | ||
Debt Issuance Costs, Net | 103 | |
Loan Agreement [Member] | Silicon Valley Bank [Member] | ||
Debt Disclosure [Line Items] | ||
Interest expense recognized | $ 203 | |
Loan Agreement [Member] | Term Loan Facility [Member] | Silicon Valley Bank [Member] | ||
Debt Disclosure [Line Items] | ||
Borrowing amount | $ 50,000 | |
Debt instrument number of tranches | Tranche | 3 | |
Proceeds from loan agreement | $ 15,000 | |
Additional borrowing amount | 15,000 | |
Line of credit facility additional discretionary | $ 15,000 | |
Loan maturity date | Oct. 1, 2026 | |
End of term charge as percentage on principal amount | 9.00% | |
Loan Agreement [Member] | Term Loan Facility [Member] | Silicon Valley Bank [Member] | Regulatory Milestone [Member] | ||
Debt Disclosure [Line Items] | ||
Additional borrowing amount | $ 10,000 |
Note Payable - Carrying Value o
Note Payable - Carrying Value of Note Payable (Details) $ in Thousands | Dec. 31, 2021USD ($) |
Debt Disclosure [Abstract] | |
Note payable, including End of Term Charge | $ 16,350 |
Debt discount, net of accretion | (1,405) |
Note payable, net of discount, long-term | $ 14,945 |
Note Payable - Future Principal
Note Payable - Future Principal Payments (Details) $ in Thousands | Dec. 31, 2021USD ($) |
Debt Disclosure [Abstract] | |
2024 | $ 1,875 |
2025 | 7,500 |
2026 | 5,625 |
Total | $ 15,000 |
Common Stock - Additional Infor
Common Stock - Additional Information (Detail) - USD ($) | 1 Months Ended | 12 Months Ended | |||||
May 31, 2021 | Nov. 30, 2020 | Jun. 30, 2020 | Feb. 29, 2020 | Jul. 31, 2019 | Dec. 31, 2021 | Dec. 31, 2020 | |
Class of Stock [Line Items] | |||||||
Common stock, shares authorized | 150,000,000 | 150,000,000 | |||||
Common stock, par value | $ 0.0001 | $ 0.0001 | |||||
Undesignated preferred stock | 10,000,000 | 10,000,000 | |||||
Undesignated shares of preferred stock outstanding | 0 | 0 | |||||
Cash dividends | $ 0 | ||||||
July 2019 Follow-on Offering [Member] | |||||||
Class of Stock [Line Items] | |||||||
Proceeds from issuance of common stock, net of offering costs | $ 129,464,000 | ||||||
July 2019 Follow-on Offering [Member] | Common Stock [Member] | |||||||
Class of Stock [Line Items] | |||||||
Common stock, shares issued and sold | 7,475,000 | ||||||
Issuance price per shares | $ 18.50 | ||||||
Over-Allotment Option [Member] | Common Stock [Member] | |||||||
Class of Stock [Line Items] | |||||||
Common stock, shares issued and sold | 975,000 | ||||||
February 2020 Follow-on Offering [Member] | |||||||
Class of Stock [Line Items] | |||||||
Proceeds from issuance of common stock, net of offering costs | $ 93,627,000 | ||||||
February 2020 Follow-on Offering [Member] | Common Stock [Member] | |||||||
Class of Stock [Line Items] | |||||||
Common stock, shares issued and sold | 4,350,000 | ||||||
Issuance price per shares | $ 23 | ||||||
ATM Facility [Member] | |||||||
Class of Stock [Line Items] | |||||||
Common stock, shares issued and sold | 1,829,268 | 384,140 | |||||
Proceeds from issuance of common stock, net of offering costs | $ 14,550,000 | $ 8,130,000 | 14,550,000 | $ 8,130,000 | |||
Common stock remained available for future issuance | $ 26,549,000 | ||||||
ATM Facility [Member] | Common Stock [Member] | |||||||
Class of Stock [Line Items] | |||||||
Common stock, shares issued and sold | 1,829,000 | 384,000 | |||||
November 2020 Follow-on Offering [Member] | |||||||
Class of Stock [Line Items] | |||||||
Proceeds from issuance of common stock, net of offering costs | $ 70,221,000 | ||||||
November 2020 Follow-on Offering [Member] | Common Stock [Member] | |||||||
Class of Stock [Line Items] | |||||||
Common stock, shares issued and sold | 5,000,000 | ||||||
Issuance price per shares | $ 15 |
Common Stock - Summary of Commo
Common Stock - Summary of Common Stock Reserved for Future Issuance (Detail) - shares | Dec. 31, 2021 | Dec. 31, 2020 | Apr. 16, 2020 |
Class of Stock [Line Items] | |||
Total shares of authorized common stock reserved for future issuance | 13,808,059 | 12,266,171 | |
Employee Stock Option [Member] | |||
Class of Stock [Line Items] | |||
Total shares of authorized common stock reserved for future issuance | 7,423,777 | 5,559,545 | |
Restricted Stock Units [Member] | |||
Class of Stock [Line Items] | |||
Total shares of authorized common stock reserved for future issuance | 599,850 | 1,198 | |
2018 Stock Option and Incentive Plan [Member] | |||
Class of Stock [Line Items] | |||
Total shares of authorized common stock reserved for future issuance | 2,583,736 | 3,877,478 | 3,300,000 |
2018 Employee Stock Purchase Plan [Member] | |||
Class of Stock [Line Items] | |||
Total shares of authorized common stock reserved for future issuance | 1,151,010 | 762,900 | |
2019 Inducement Plan [Member] | |||
Class of Stock [Line Items] | |||
Total shares of authorized common stock reserved for future issuance | 412,686 | 365,050 | |
2020 Inducement Plan [Member] | |||
Class of Stock [Line Items] | |||
Total shares of authorized common stock reserved for future issuance | 1,637,000 | 1,700,000 |
Stock-Based Compensation - Addi
Stock-Based Compensation - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Apr. 16, 2020 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||
Common stock reserved for issuance | 13,808,059 | 12,266,171 | |
Number of options granted | 4,253,232 | ||
Aggregate intrinsic value of options exercised | $ 1,826 | $ 2,077 | |
Options Granted, Weighted-average grant-date fair value | $ 8.70 | $ 12.28 | |
Unvested restricted stock awards | 599,850 | 1,198 | |
Total fair value of restricted stock awards and restricted stock units vested | $ 5 | $ 29 | |
Unrecognized stock-based compensation expenses | $ 43,331 | ||
Unrecognized stock-based compensation expense, period for recognition | 2 years 9 months 25 days | ||
Additional research and development expenses | $ 417 | ||
Restricted Stock [Member] | |||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||
Unvested restricted stock awards | 0 | ||
2018 Stock Option and Incentive Plan [Member] | |||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||
Number of common shares that may be issued under the plan | 616,300 | ||
Common shares available for future grant | 2,583,736 | ||
Number of shares of common stock outstanding, increase, percentage | 4.00% | ||
Common stock reserved for issuance | 2,583,736 | 3,877,478 | 3,300,000 |
Number of options granted | 4,253,232 | 3,019,663 | |
2015 Stock Option and Grant Plan [Member] | |||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||
Number of common shares that may be issued under the plan | 2,008,564 | ||
Common shares available for future grant | 0 | ||
Option vesting period | 4 years | ||
2018 Employee Stock Purchase Plan [Member] | |||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||
Common shares available for future grant | 1,151,010 | ||
Employee Stock Purchase Plan, description | The ESPP provides that the number of shares reserved and available for issuance will automatically increase each January 1, beginning on January 1, 2019 and each January 1 thereafter through January 1, 2028, by the least of (i) 1% of the outstanding number of shares of our common stock on the immediately preceding December 31; (ii) 1,115,700 shares or (iii) such number of shares as determined by the ESPP administrator (the “ESPP Evergreen”). | ||
Number of common shares issued | 27,580 | 13,425 | |
2018 Employee Stock Purchase Plan [Member] | Maximum [Member] | |||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||
Number of common shares that may be issued under the plan | 223,200 | ||
Number of shares of common stock outstanding, increase, percentage | 1.00% | ||
Number of common shares that may increase under the plan | 1,115,700 | ||
2019 Inducement Plan [Member] | |||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||
Number of common shares that may be issued under the plan | 1,800,000 | ||
Common shares available for future grant | 412,686 | ||
2020 Inducement Plan [Member] | |||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||
Number of common shares that may be issued under the plan | 1,700,000 | ||
Common shares available for future grant | 1,637,000 |
Stock-Based Compensation - Summ
Stock-Based Compensation - Summary of Assumptions that Used to Determine Grant-Date Fair Value of Stock Options Granted to Employees and Members of Board (Detail) | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Share Based Compensation Arrangement By Share Based Payment Award Fair Value Assumptions And Methodology [Abstract] | ||
Expected option life (years) | 6 years 21 days | 6 years |
Risk-free interest rate | 0.80% | 0.76% |
Expected volatility | 81.22% | 76.02% |
Stock-Based Compensation - Su_2
Stock-Based Compensation - Summary of Stock Option Activity (Detail) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Share Based Compensation Arrangement By Share Based Payment Award Options Outstanding Roll Forward | ||
Number of options, Outstanding beginning balance | 5,559,545 | |
Number of options, Granted | 4,253,232 | |
Number of options, Exercised | (226,180) | |
Number of options, Cancelled or forfeited | (2,162,820) | |
Number of options, Outstanding ending balance | 7,423,777 | 5,559,545 |
Number of options, Exercisable | 2,690,230 | |
Weighted average exercise price, Outstanding beginning balance | $ 14.97 | |
Weighted average exercise price, Granted | 12.64 | |
Weighted average exercise price, Exercised | 4.07 | |
Weighted average exercise price, Cancelled or forfeited | 16.55 | |
Weighted average exercise price, Outstanding ending balance | 13.54 | $ 14.97 |
Weighted average exercise price, Exercisable | $ 12.87 | |
Weighted average remaining contractual term, Outstanding balance | 6 years 9 months 14 days | 7 years 7 months 24 days |
Weighted average remaining contractual term, Exercisable | 5 years 8 months 8 days | |
Aggregate intrinsic value, Outstanding balance | $ 1,468,969 | $ 13,511 |
Aggregate intrinsic value, Exercisable | $ 1,466,939 |
Stock-Based Compensation - Su_3
Stock-Based Compensation - Summary of Restricted Stock Award and Restricted Stock Unit Activity (Detail) | 12 Months Ended |
Dec. 31, 2021$ / sharesshares | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Number of shares, Issued and unvested beginning balance | shares | 1,198 |
Number of shares, Granted | shares | 771,600 |
Number of shares, Vested | shares | (575) |
Number of shares, Forfeited, cancelled or expired | shares | (172,373) |
Number of shares, Issued and unvested ending balance | shares | 599,850 |
Weighted average grant date fair value, Issued and unvested, beginning balance | $ / shares | $ 15.65 |
Weighted average grant date fair value, Granted | $ / shares | 9.63 |
Weighted average grant date fair value, Vested | $ / shares | 15.65 |
Weighted average grant date fair value, Forfeited, cancelled or expired | $ / shares | 9.63 |
Weighted average grant date fair value, Issued and unvested, ending balance | $ / shares | $ 9.64 |
Stock-Based Compensation - Sche
Stock-Based Compensation - Schedule of Stock-based Compensation Expense (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||
Total stock-based compensation expense | $ 18,579 | $ 15,628 |
Research and Development Expense [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||
Total stock-based compensation expense | 6,996 | 8,207 |
General and Administrative Expense [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||
Total stock-based compensation expense | $ 11,583 | $ 7,421 |
401(k) Savings Plan - Additiona
401(k) Savings Plan - Additional Information (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Compensation And Retirement Disclosure [Abstract] | ||
Defined contribution plan, description | Currently, the Company makes matching contributions at a rate of 50% of the first 6% of employee contributions. | |
Employer matching contribution, percent of match | 50.00% | |
Percent of employee contributions | 6.00% | |
Defined contribution plan, expense | $ 593 | $ 468 |
Income Taxes - Additional Infor
Income Taxes - Additional Information (Detail) - USD ($) | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Income Taxes [Line Items] | ||
Deferred income tax expense (benefit) | $ 0 | $ 0 |
Deferred tax assets change in valuation allowance | 30,401,000 | 32,066,000 |
Deferred tax assets, operating loss carryforwards, domestic | 312,967,000 | $ 213,991,000 |
Deferred tax assets, operating loss carryforwards, not subject to expiration | 295,224,000 | |
Payroll tax liability capped per year | 250,000 | |
Income tax reconciliation tax credit research | $ 250,000 | |
Income tax credit carryforward expiration period | 20 years | |
Research and development tax credits | 2.00% | 2.30% |
Percentage of cumulative changes in ownership interest subject to annual limitation | 50.00% | |
Period of cumulative changes in ownership interest | 3 years | |
U.S. federal [Member] | ||
Income Taxes [Line Items] | ||
Research and development tax credits carryforwards | $ 6,234,000 | $ 3,677,000 |
Australia [Member] | ||
Income Taxes [Line Items] | ||
Income tax reconciliation tax credit research | $ 1,210,000 | 589,000 |
Australia [Member] | Maximum [Member] | ||
Income Taxes [Line Items] | ||
Research and development tax credits | 43.50% | |
Orphan Drug Credit [Member] | U.S. federal [Member] | ||
Income Taxes [Line Items] | ||
Research and development tax credits carryforwards | $ 1,959,000 | |
Expiration Through 2037 [Member] | ||
Income Taxes [Line Items] | ||
Deferred tax assets, operating loss carryforwards, subject to expiration | 17,743,000 | |
Expiration Through 2039 [Member] | ||
Income Taxes [Line Items] | ||
Deferred tax assets, operating loss carryforwards, state | 290,500,000 | 201,642,000 |
Expiration Through 2033 [Member] | State [Member] | ||
Income Taxes [Line Items] | ||
Research and development tax credits carryforwards | $ 1,959,000 | $ 894,000 |
Income Taxes - Schedule of Reco
Income Taxes - Schedule of Reconciliation of Income Tax Expense (Benefit) (Detail) | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Income Tax Disclosure [Abstract] | ||
Federal income tax expense at statutory rate | 21.00% | 21.00% |
State income taxes, net of federal benefit | 6.00% | 5.60% |
Permanent differences | (1.20%) | (0.60%) |
Foreign rate differential | 0.10% | 0.10% |
Research and development tax credits | 2.00% | 2.30% |
Change in valuation allowance | (27.90%) | (28.30%) |
Provision to Return | 0.00% | (0.10%) |
Effective income tax rate | 0.00% | 0.00% |
Income Taxes - Schedule of Defe
Income Taxes - Schedule of Deferred Tax Assets and Liabilities (Detail) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Deferred tax assets: | ||
U.S., foreign and state net operating loss carryforwards | $ 84,102 | $ 57,705 |
Research and development credits | 7,821 | 4,383 |
Capitalized start up and organizational costs | 26 | 29 |
Equity based compensation | 3,926 | 2,944 |
Licensing agreements | 3,749 | 4,004 |
Accruals and other | 1,376 | 1,587 |
Total deferred tax assets | 101,000 | 70,652 |
Valuation allowance | (100,893) | (70,492) |
Net deferred tax assets | 107 | 160 |
Deferred tax liabilities: | ||
Property and equipment | $ (107) | $ (160) |
Net Loss per Share - Schedule o
Net Loss per Share - Schedule of Dilutive Common Stock Equivalents Excluded from Computation of Diluted Net Loss per Share (Detail) - shares | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Options to Purchase Common Stock [Member] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Antidilutive securities excluded from computation of earnings per share amount | 7,423,777 | 5,559,545 |
Restricted Stock Awards and Units [Member] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Antidilutive securities excluded from computation of earnings per share amount | 599,850 | 1,198 |
Related Party Transactions - Ad
Related Party Transactions - Additional Information (Detail) - USD ($) | Jan. 27, 2016 | Jul. 31, 2018 | Dec. 31, 2021 | Dec. 31, 2020 |
Related Party Transaction [Line Items] | ||||
Common stock issued for initial consideration for license | 771,600 | |||
Research and development expense | $ 83,114,000 | $ 87,236,000 | ||
License Agreement [Member] | ||||
Related Party Transaction [Line Items] | ||||
Provision for maximum cash payment | $ 2,000,000 | |||
Research and development expense | 248,000 | 381,000 | ||
License Agreement [Member] | Stock Purchase Agreement [Member] | ||||
Related Party Transaction [Line Items] | ||||
Common stock issued for initial consideration for license | 1,161,665 | |||
Sub-lease Agreement [Member] | Officers and Board Members [Member] | ||||
Related Party Transaction [Line Items] | ||||
Expense related to related party | $ 1,523,000 | $ 1,488,000 |
Subsequent Events - Additional
Subsequent Events - Additional Information (Detail) - Forecast [Member] $ in Millions | 3 Months Ended |
Mar. 31, 2022USD ($) | |
Subsequent Event [Line Items] | |
Expected workforce reduction percentage | 23.00% |
Severance and termination-related costs | $ 2.5 |