Document and Entity Information
Document and Entity Information - USD ($) | 12 Months Ended | ||
Dec. 31, 2019 | Mar. 06, 2020 | Jun. 28, 2019 | |
Cover [Abstract] | |||
Document Type | 10-K | ||
Amendment Flag | false | ||
Document Period End Date | Dec. 31, 2019 | ||
Document Fiscal Year Focus | 2019 | ||
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 | Accelerated Filer | ||
Entity Common Stock, Shares Outstanding | 36,030,495 | ||
Entity Public Float | $ 289,225,075 | ||
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, $0.0001 par value per share | ||
Security Exchange Name | NASDAQ | ||
Documents Incorporated by Reference | DOCUMENTS INCORPORATED BY REFERENCE Part III of this Annual Report on Form 10-K incorporates by reference certain information from the registrant’s definitive Proxy Statement for its 2020 annual meeting of shareholders, which the registrant intends to file pursuant to Regulation 14A with the Securities and Exchange Commission not later than 120 days after the registrant’s fiscal year end of December 31, 2019. Except with respect to information specifically incorporated by reference in this Form 10-K, the Proxy Statement is not deemed to be filed as part of this Form 10-K. |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Current assets: | ||
Cash and cash equivalents | $ 187,043 | $ 126,302 |
Prepaid expenses and other current assets | 8,658 | 3,718 |
Total current assets | 195,701 | 130,020 |
Property and equipment, net | 3,696 | 2,634 |
Other assets | 1,117 | 825 |
Total assets | 200,514 | 133,479 |
Current liabilities: | ||
Accounts payable | 3,949 | 2,784 |
Accrued expenses and other current liabilities | 10,068 | 7,822 |
Total current liabilities | 14,017 | 10,606 |
Deferred rent, net of current portion | 484 | 689 |
Total liabilities | 14,501 | 11,295 |
Commitments and contingencies (Note 14) | ||
Stockholders’ equity: | ||
Preferred stock, $0.0001 par value; 10,000,000 shares authorized and no shares issued or outstanding as of December 31, 2019 and 2018 | ||
Common stock, $0.0001 par value; 150,000,000 shares authorized as of December 31, 2019 and 2018; 31,673,058 and 23,959,903 shares issued as of December 31, 2019 and 2018, respectively; 31,642,806 and 23,806,628 shares outstanding as of December 31, 2019 and 2018, respectively | 3 | 2 |
Additional paid-in capital | 330,714 | 193,921 |
Accumulated deficit | (144,704) | (71,739) |
Total stockholders’ equity | 186,013 | 122,184 |
Total liabilities and stockholders' equity | $ 200,514 | $ 133,479 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - $ / shares | Dec. 31, 2019 | Dec. 31, 2018 |
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 | 31,673,058 | 23,959,903 |
Common stock, outstanding | 31,642,806 | 23,806,628 |
Consolidated Statements of Oper
Consolidated Statements of Operations and Comprehensive Loss - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Operating expenses: | ||
Research and development | $ 54,974 | $ 35,095 |
General and administrative | 20,835 | 11,148 |
Total operating expenses | 75,809 | 46,243 |
Loss from operations | (75,809) | (46,243) |
Other income (expense): | ||
Interest income | 2,934 | 1,726 |
Change in fair value of preferred stock warrant liability | (162) | |
Change in fair value of derivative liability | (1,629) | |
Other expense | (90) | (53) |
Total other income (expense), net | 2,844 | (118) |
Net loss | (72,965) | (46,361) |
Comprehensive loss | (72,965) | (46,361) |
Reconciliation of net loss to net loss attributable to common stockholders: | ||
Net loss | (72,965) | (46,361) |
Accretion of issuance costs on redeemable convertible preferred stock | (2,243) | |
Net loss attributable to common stockholders—basic and diluted | $ (72,965) | $ (48,604) |
Net loss per share attributable to common stockholders—basic and diluted (Note 13) | $ (2.66) | $ (3.62) |
Weighted-average number of common shares used in computing net loss per share attributable to common stockholders—basic and diluted | 27,432,489 | 13,435,478 |
Consolidated Statements of Rede
Consolidated Statements of Redeemable Convertible Preferred Stock and Stockholders' (Deficit) Equity - USD ($) $ in Thousands | Total | IPO [Member] | Series Seed Redeemable Convertible Preferred Stock [Member] | Series A Redeemable Convertible Preferred Stock [Member] | Series B Redeemable Convertible Preferred Stock [Member] | Common Stock [Member] | Common Stock [Member]IPO [Member] | Additional Paid-in Capital [Member] | Additional Paid-in Capital [Member]IPO [Member] | Accumulated Deficit [Member] |
Beginning balance at Dec. 31, 2017 | $ (23,135) | $ 1,500 | $ 25,000 | $ 339 | $ (23,474) | |||||
Beginning balance, shares at Dec. 31, 2017 | 3,333,333 | 31,450,499 | 2,305,173 | |||||||
Issuance of redeemable convertible preferred stock, | $ 58,257 | |||||||||
Issuance of redeemable convertible preferred stock, shares | 28,285,557 | |||||||||
Issuance of series B redeemable convertible preferred stock to settle accrued liability of license cost | $ 500 | |||||||||
Issuance of series B redeemable convertible preferred stock to settle accrued liability of license cost, shares | 233,765 | |||||||||
Accretion of issuance costs related to redeemable convertible preferred stock | (2,243) | $ 2,243 | (339) | (1,904) | ||||||
Issuance of common stock upon IPO/public offering, net of issuance/offering costs | $ 104,013 | $ 1 | $ 104,012 | |||||||
Issuance of common stock upon IPO/public offering, net of issuance/offering costs, shares | 6,035,151 | |||||||||
Stock-based compensation expense | 2,196 | 2,196 | ||||||||
Reclassification of warrants to purchase redeemable convertible stock into warrants to purchase common stock | 197 | 197 | ||||||||
Conversion of redeemable convertible preferred stock into common stock | 87,500 | $ (1,500) | $ (25,000) | $ (61,000) | $ 1 | 87,499 | ||||
Conversion of redeemable convertible preferred stock into common stock, shares | (3,333,333) | (31,450,499) | (28,519,322) | 15,320,213 | ||||||
Exercise of warrants to purchase common stock, shares | 6,091 | |||||||||
Vesting of restricted stock awards, shares | 123,026 | |||||||||
Exercise of stock options | 17 | 17 | ||||||||
Exercise of stock options, shares | 16,974 | |||||||||
Net loss | (46,361) | (46,361) | ||||||||
Ending balance at Dec. 31, 2018 | 122,184 | $ 2 | 193,921 | (71,739) | ||||||
Ending balance, shares at Dec. 31, 2018 | 23,806,628 | |||||||||
Issuance of common stock upon IPO/public offering, net of issuance/offering costs | $ 129,465 | $ 1 | $ 129,464 | |||||||
Issuance of common stock upon IPO/public offering, net of issuance/offering costs, shares | 7,475,000 | |||||||||
Issuance of common stock under 2018 employee stock purchase plan | 27 | 27 | ||||||||
Issuance of common stock under 2018 employee stock purchase plan, shares | 1,671 | |||||||||
Stock-based compensation expense | 6,800 | 6,800 | ||||||||
Vesting of restricted stock awards, shares | 123,024 | |||||||||
Exercise of stock options | $ 502 | 502 | ||||||||
Exercise of stock options, shares | 236,483 | 236,483 | ||||||||
Net loss | $ (72,965) | (72,965) | ||||||||
Ending balance at Dec. 31, 2019 | $ 186,013 | $ 3 | $ 330,714 | $ (144,704) | ||||||
Ending balance, shares at Dec. 31, 2019 | 31,642,806 |
Consolidated Statements of Re_2
Consolidated Statements of Redeemable Convertible Preferred Stock and Stockholders' (Deficit) Equity (Parenthetical) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Statement Of Stockholders Equity [Abstract] | ||
Issuance of redeemable convertible preferred stock, net of issuance costs | $ 2,243 | |
Issuance of common stock upon IPO, net of issuance costs | $ 525 | $ 2,628 |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows - USD ($) | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Cash flows from operating activities: | ||
Net loss | $ (72,965,000) | $ (46,361,000) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Stock-based compensation expense | 6,800,000 | 2,196,000 |
Depreciation and amortization expense | 850,000 | 380,000 |
Amortization of deferred offering costs | 37,000 | |
Impairment loss of property and equipment | 0 | 235,000 |
Deferred rent expense | (172,000) | (105,000) |
Change in fair value of preferred stock warrant liability | 162,000 | |
Change in fair value of derivative liability | 1,629,000 | |
Changes in operating assets and liabilities: | ||
Prepaid expenses and other current assets | (4,888,000) | (3,037,000) |
Other assets | (241,000) | (115,000) |
Accounts payable | 1,028,000 | 2,018,000 |
Accrued expenses and other current liabilities | 1,920,000 | 5,313,000 |
Net cash used in operating activities | (67,668,000) | (37,648,000) |
Cash flows from investing activities: | ||
Purchases of property and equipment | (1,585,000) | (1,832,000) |
Net cash used in investing activities | (1,585,000) | (1,832,000) |
Cash flows from financing activities: | ||
Proceeds from issuance of redeemable convertible preferred stock, net of issuance costs | 58,257,000 | |
Proceeds from issuance of common shares upon completion of public offering, net of offering costs | 129,465,000 | 104,013,000 |
Payment of dilution liability | (2,000,000) | |
Exercise of stock options | 502,000 | 17,000 |
Proceeds from the issuance of shares under the employee stock purchase plan | 27,000 | |
Net cash provided by financing activities | 129,994,000 | 160,287,000 |
Net increase in cash, cash equivalents and restricted cash | 60,741,000 | 120,807,000 |
Cash, cash equivalents and restricted cash at beginning of period | 126,794,000 | 5,987,000 |
Cash, cash equivalents and restricted cash at end of period | 187,535,000 | 126,794,000 |
Supplemental disclosure of non-cash investing and financing activities: | ||
Purchases of property and equipment included in accounts payable and accrued expenses | 582,000 | 256,000 |
Property and equipment held for sale | 19,000 | |
Deferred offering costs included in accrued expenses and accounts payable | $ 104,000 | |
Purchase of property and equipment paid for by landlord | 842,000 | |
Accretion of issuance costs related to redeemable convertible preferred stock | $ 2,243,000 |
Nature of the Business
Nature of the Business | 12 Months Ended |
Dec. 31, 2019 | |
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. Through December 31, 2019, the Company has funded its operations primarily with proceeds from the sale of Seed redeemable convertible preferred stock (the “Series Seed Preferred Stock”), series A redeemable convertible preferred stock (the “Series A Preferred Stock”) and series B redeemable convertible preferred stock (the “Series B Preferred Stock”), (the Series Seed Preferred Stock, the Series A Preferred Stock and the Series B Preferred Stock are collectively referred to as the “Preferred Stock”) and common stock through the Company’s initial public offering (“IPO”). The Company has incurred recurring losses since its inception, including net losses of $72,965 and $46,361 for the years ended December 31, 2019 and 2018, respectively. In addition, as of December 31, 2019, the Company had an accumulated deficit of $144,704. 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, 2019 of $187,043, together with net proceeds from the follow on public offering completed in February 2020 (“the February 2020 Follow-On Offering”) (see Note 17) 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, 2019 | |
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. Use of Estimates The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the consolidated financial statements and the reported amounts of revenue and expenses during the reporting periods. Significant estimates and assumptions reflected in these consolidated financial statements include, but are not limited to, the accrual for research and development expenses, stock-based compensation expense, the valuation of equity and derivative instruments and the recoverability of the Company’s net deferred tax assets and related valuation allowance. Estimates are periodically reviewed in light of changes in circumstances, facts and experience. Changes in estimates are recorded in the period in which they become known. Actual results could differ materially from those estimates. Cash and Cash Equivalents The Company considers all highly liquid investments purchased with original maturities of 90 days or less at acquisition to be cash equivalents. Cash and cash equivalents include cash held in banks and amounts held in interest-bearing money market accounts. Cash equivalents are carried at cost, which approximates their fair market value. Restricted Cash As of both December 31, 2019 and 2018, restricted cash consisted of $492 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. Deferred Issuance Costs The Company capitalizes certain legal, professional accounting and other third-party fees that are directly associated with in-process equity financings as deferred issuance costs until such financings are consummated. After consummation of the equity financing, these costs are recorded as a reduction of the proceeds generated as a result of the offering. Should the planned equity financing be abandoned, the deferred issuance costs will be expensed immediately as a charge to operating expenses in the consolidated statements of operations. As of December 31, 2019, the Company recorded deferred issuance costs of $133 within other assets on the consolidated balance sheet related to a follow-on offering of common stock completed in February 2020. There were no amounts deferred as of December 31, 2018. 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 and software 2 years Leasehold improvements Lesser of lease term or 10 years Upon disposal, retirement or sale, the cost of assets disposed of and the related accumulated depreciation are removed from the accounts and any resulting gain or loss is included in the results of operations. Expenditures for repairs and maintenance that do not improve or extend the lives of the respective assets are charged to expense as incurred. 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. During the year ended December 31, 2018, the Company recorded an impairment loss of $235 on long-lived assets related to leasehold improvements. The Company did not record any impairment loss during the year ended December 31, 2019. Fair Value Measurements Certain assets and liabilities of the Company are carried at fair value under GAAP (see Note 3). 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—Quoted prices in active markets for identical assets or liabilities. • 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 and cash equivalents, restricted cash, accounts payable, and accrued expenses, approximated their fair values at December 31, 2019 and 2018 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 3 for further discussion. 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. Research and Development Costs 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 the 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, 2019, 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. See Note 10 for the assumptions used by the Company in determining the grant date fair value of stock-based awards granted, as well as a summary of the stock-based award activity under the Company’s stock-based compensation plan for the year ended December 31, 2019. Warrant to Purchase Preferred Stock Prior to the IPO, the Company classified the warrant for the purchase of shares of its redeemable convertible preferred stock (see Note 7) as a liability on its consolidated balance sheets as the warrant was a free-standing financial instrument that may require the Company to transfer assets upon exercise. The preferred stock warrant liability was initially recorded at fair value upon the date of issuance and was subsequently remeasured to fair value at each reporting date. Changes in the fair value of the warrant to purchase preferred stock were recognized as a component of other income (expense), net in the consolidated statements of operations and comprehensive loss. The Company utilized the Black-Scholes option-pricing model, which incorporates assumptions and estimates, to value the warrant. The Company assessed these assumptions and estimates on a quarterly basis as additional information impacting the assumptions was obtained. Estimates and assumptions impacting the fair value measurement included the fair value per share of the underlying redeemable convertible preferred stock issuable upon exercise of the warrant, the remaining contractual term of the warrant, the risk-free interest rate, the expected dividend yield and the expected volatility of the price of the underlying redeemable convertible preferred stock. Upon the IPO, the warrant to purchase preferred stock was converted to a warrant to purchase common stock. The carrying amount of the warrant to purchase preferred stock as of the date of IPO was transferred to the account of additional paid in capital. No further revaluation was needed for the warrant to purchase common stock. 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’ (deficit) equity which includes certain changes in equity that are excluded from net income (loss). Comprehensive loss has been disclosed in the accompanying 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 income (expense), net in the consolidated statements of operations and comprehensive loss. During the years ended December 31, 2019 and 2018, the Company recorded foreign exchange losses of $90 and $51, respectively, in other expense. 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. 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 other 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. Net Income (Loss) per Share Attributable to Common Stockholders Net income (loss) per share attributable to common stockholders is determined using the two-class method, which includes the weighted-average number of shares of common stock outstanding during the period and other securities that participate in dividends (a participating security). In periods of income, the redeemable convertible preferred stock would be considered participating securities because the shares include rights to participate in dividends with the common stock; however, the redeemable convertible preferred stock is not considered a participating security in periods of loss as they do not have an obligation to share in the Company’s net losses. Under the two-class method, basic net income (loss) per share attributable to common stockholders is computed by dividing the net income (loss) attributable to common stockholders by the weighted-average number of shares of common stock outstanding during the period. Diluted net income (loss) per share attributable to common stockholders is computed using the more dilutive of (1) the two-class method or (2) the if-converted method. The Company allocates net income first to the holders of Preferred Stock based on dividend rights under the Company’s certificate of incorporation and then to preferred and common stockholders based on ownership interests. 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. The Company has evaluated all subsequent events and determined that there are no material recognized or unrecognized subsequent events requiring disclosure, other than as disclosed in these notes to the consolidated financial statements. See Note 17 for further information. 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 Adopted Accounting Pronouncements In June 2018, the FASB issued ASU No. 2018-07, Compensation-Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting Compensation-Stock Compensation In May 2014, the FASB issued ASU No. 2014-09, Revenue from Contracts with Customers (Topic 606) In May 2017, the FASB issued ASU No. 2017-09, Compensation—Stock Compensation (Topic 718): Scope of Modification Accounting In November 2016, the FASB issued ASU No. 2016-18, Statement of Cash Flows (Topic 230) Restricted Cash December 31, 2019 2018 Cash and cash equivalents $ 187,043 $ 126,302 Other assets 492 492 Cash, cash equivalents and restricted cash $ 187,535 $ 126,794 Recently Issued Accounting Pronouncements In December 2019, the FASB issued ASU No. 2019-12, Simplifying the Accounting for Income Taxes In August 2018, the FASB issued ASU No. 2018-13, Fair Value Measurement (Topic 820): Disclosure Framework—Changes to the Disclosure Requirements for Fair Value Measurement In August 2018, the FASB issued ASU No. 2018-15, Intangible-Goodwill and Other Internal-Use Software (Subtopic 350-40) (“ASU 2018-15”). ASU 2018-15 updates guidance regarding accounting for implementation costs associated with a cloud computing arrangement that is a service contract. The amendments under ASU 2018-15 are effective for interim and annual fiscal periods beginning after December 15, 2019, with early adoption permitted. The Company does not expect the adoption of ASU 2018-15 to have a material impact on its consolidated financial statements. In November 2018, the FASB issued ASU No. 2018-18, Collaborative Arrangements (Topic 808)—Clarifying the Interaction between Topic 808 and Topic 606 (“ASU 2018-18”). The amendments in ASU 2018-18 clarify that certain transactions between collaborative arrangement participants should be accounted for as revenue under ASC 606 when the collaborative arrangement participant is a customer in the context of a unit of account. The amendments under ASU 2018-18 are effective for interim and annual fiscal periods beginning after December 15, 2019, with early adoption permitted. The amendments in ASU 2018-18 should be applied retrospectively to the date of initial application of ASC 606. The Company does not expect the adoption of ASU 2018-18 to have a material impact on its consolidated financial statements. In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments—Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments (“ASU 2016-13”). ASU 2016-13 requires that credit losses be reported as an allowance using an expected losses model, representing the entity's current estimate of credit losses expected to be incurred. The accounting guidance currently in effect is based on an incurred loss model. For available-for-sale debt securities with unrealized losses, this standard now requires allowances to be recorded instead of reducing the amortized cost of the investment. The amendments under ASU 2016-13 are effective for interim and annual fiscal periods beginning after December 15, 2019. The Company does not expect the adoption of ASC 2016-13 to have a material impact on its consolidated financial statements. In February 2016, the FASB issued ASU No. 2016-02, (Topic 842) Leases (“ASU 2016-02”). ASU 2016-02 requires an entity to recognize assets and liabilities arising from a lease for both financing and operating leases. The ASU will also require new qualitative and quantitative disclosures to help investors and other financial statement users better understand the amount, timing, and uncertainty of cash flows arising from leases. For public entities, ASU 2016-02 is effective for fiscal years beginning after December 15, 2018. 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, ASU 2016-02 is effective for the Company for the year ended December 31, 2021, and all interim periods thereafter. Early adoption is permitted. The Company is currently evaluating the impact that the adoption of ASU 2016-02 will have on its consolidated financial statements. |
Fair Value of Financial Assets
Fair Value of Financial Assets and Liabilities | 12 Months Ended |
Dec. 31, 2019 | |
Fair Value Disclosures [Abstract] | |
Fair Value of Financial Assets and Liabilities | 3. 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, 2019 and 2018: Fair Value Measurements as of December 31, 2019 Using: Level 1 Level 2 Level 3 Total Assets: Money market funds $ 186,797 $ — $ — $ 186,797 Restricted cash 492 — — 492 $ 187,289 $ — $ — $ 187,289 Fair Value Measurements as of December 31, 2018 Using: Level 1 Level 2 Level 3 Total Assets: Money market funds $ 126,047 $ — $ — $ 126,047 Restricted cash 492 — — 492 $ 126,539 $ — $ — $ 126,539 During the years ended December 31, 2019 and 2018, there were no transfers between Level 1, Level 2 and Level 3. Valuation of the Warrant to Purchase Preferred Stock In connection with a loan and security agreement in 2017 (the “Loan Agreement”) (Note 7), the Company agreed to issue a warrant to the lender for the purchase shares of Series A Preferred Stock. The fair value of the warrant to purchase preferred stock was determined based on significant inputs not observable in the market, which represents a Level 3 measurement within the fair value hierarchy. In order to determine the fair value of the warrant to purchase preferred stock, the Company utilized available facts and circumstances to estimate the number of shares of Series A Preferred Stock for which the warrant would ultimately be exercisable. The Company then used the Black-Scholes option-pricing model, which incorporates assumptions and estimates, to value the warrant to purchase preferred stock. Estimates and assumptions impacting the fair value measurement included the fair value of the underlying shares of Series A Preferred Stock, the remaining contractual term of the warrant, risk-free interest rate, expected dividend yield and expected volatility of the price of the underlying preferred stock. The Company determined the fair value of the underlying preferred stock based on various valuation methodologies. The Company lacks company-specific historical and implied volatility information of its stock. Therefore, it estimated its expected stock volatility based on the historical volatility of publicly traded guideline companies for a term equal to the remaining contractual term of the warrant. The risk-free interest rate was determined by reference to the U.S. Treasury yield curve for time periods approximately equal to the remaining contractual term of the warrant. The Company estimated no expected dividend yield based on the fact that the Company has never paid or declared dividends and does not intend to do so in the foreseeable future. Upon the IPO, the warrant to purchase preferred stock was converted to a warrant to purchase common stock. The carrying amount of warrant to purchase preferred stock as of the date of IPO was transferred to the account of additional paid in capital. The following table sets forth a summary of changes in the fair value of the Company’s preferred stock warrant liability for which fair value was determined by Level 3 inputs: Warrant Liability Balance as of December 31, 2017 $ 35 Change in fair value 162 Conversion of preferred stock warrant to common stock warrant (197 ) Balance as of December 31, 2018 $ — Valuation of Derivative In January 2016, in connection with a license agreement entered into with University Health Network (“UHN”), and as part of the initial consideration for the license, the Company issued 1,161,665 shares of common stock to UHN pursuant to a stock purchase agreement (the “Stock Purchase Agreement”). The Stock Purchase Agreement contained a provision requiring the Company to make a cash payment to UHN of up to $2,000 if UHN’s fully diluted ownership is reduced within specified percentages as part of an initial public offering by the Company. The Company concluded the anti-dilution feature represented a derivative instrument and should be measured at fair value, with changes in fair value recognized as a gain or loss to other income (expense), net in the consolidated statements of operations and comprehensive loss. On June 21, 2018, in connection with the Company’s IPO, the Company remeasured the fair value of the derivative to $2,000 as the Company was required to pay the dilution payment as mentioned above, which the Company paid in July 2018. An increase in fair value of $1,629 was recorded in other expense in the accompanying consolidated statement of operations and comprehensive loss for the year ended December 31, 2018. The following table sets forth a summary of changes in the fair value of the Company’s derivative liability for which fair value is determined by Level 3 inputs: Derivative Liability Balance as of December 31, 2017 $ 371 Change in fair value 1,629 Payment (2,000 ) Balance as of December 31, 2018 $ — |
Prepaid Expenses and Other Curr
Prepaid Expenses and Other Current Assets | 12 Months Ended |
Dec. 31, 2019 | |
Text Block [Abstract] | |
Prepaid Expenses and Other Current Assets | 4. Prepaid Expenses and Other Current Assets Prepaid expenses and other current assets consisted of the following: December 31, 2019 2018 Tax incentive refund $ 1,916 $ 1,325 Prepaid research and development costs 4,915 981 Prepaid insurance 897 316 Interest income receivable 224 220 Prepaid rent 86 81 Other current assets 620 795 $ 8,658 $ 3,718 |
Property and Equipment, Net
Property and Equipment, Net | 12 Months Ended |
Dec. 31, 2019 | |
Property Plant And Equipment [Abstract] | |
Property and Equipment, Net | 5. Property and Equipment, Net Property and equipment, net consisted of the following: December 31, 2019 2018 Laboratory and office equipment $ 3,456 $ 1,624 Leasehold improvements 1,340 1,260 Computer equipment and software 134 134 4,930 3,018 Less: Accumulated depreciation and amortization (1,234 ) (384 ) $ 3,696 $ 2,634 Depreciation and amortization expense for the years ended December 31, 2019 and 2018 was $850 and $380, respectively. |
Accrued Expenses
Accrued Expenses | 12 Months Ended |
Dec. 31, 2019 | |
Payables And Accruals [Abstract] | |
Accrued Expenses | 6. Accrued Expenses Accrued expenses consisted of the following: December 31, 2019 2018 Compensation and benefit costs $ 3,028 $ 2,616 Research and development costs 5,794 3,969 Consulting and professional fees 917 320 Other liabilities 329 917 $ 10,068 $ 7,822 |
Loan Agreement and Warrant to P
Loan Agreement and Warrant to Purchase Preferred Stock | 12 Months Ended |
Dec. 31, 2019 | |
Statement Of Stockholders Equity [Abstract] | |
Loan Agreement and Warrant to Purchase Preferred Stock | 7. Loan Agreement and Warrant to Purchase Preferred Stock On June 23, 2017, the Company entered into the Loan Agreement with a lender, which provided for the issuance of term loans of up to $10,000, subject to the achievement of various development and corporate milestones. Any outstanding principal amounts under the Loan Agreement accrued interest at a floating per annum rate equal to the greater of 1% and the “prime rate,” as defined in the Loan Agreement, minus 3%. As of October 31, 2018, the Company had not drawn down from the facility and the Loan Agreement expired on October 31, 2018. In connection with the Loan Agreement, the Company agreed to issue a warrant to the lender for the purchase of up to 188,702 shares of the Company’s Series A Preferred Stock with an exercise price of $0.7949 per share. The warrant expires on June 22, 2027. The warrant was initially exercisable for the purchase of up to 28,305 shares of Series A Preferred Stock and could become exercisable for up to an additional 160,397 shares of Series A Preferred Stock based on the amounts drawn under the Loan Agreement. On the issuance date of the warrant, the Company recorded a deferred financing cost and a liability for the warrant to purchase preferred stock in the Company’s consolidated balance sheet equal to the issuance-date fair value of the warrant. Upon closing of the IPO, the carrying amount of the warrant to purchase preferred stock as of the date of IPO was transferred to the account of additional paid in capital (Note 3). The Company recognized a loss of $162 in the consolidated statements of operations and comprehensive loss for the year ended December 31, 2018 related to the change in fair value of the warrant. On July 13, 2018, the lender of the Loan Agreement exercised its common stock warrants to purchase 6,850 shares of common stock on a net basis, resulting in the issuance of 6,091 shares of common stock. |
Redeemable Convertible Preferre
Redeemable Convertible Preferred Stock | 12 Months Ended |
Dec. 31, 2019 | |
Text Block [Abstract] | |
Redeemable Convertible Preferred Stock | 8. Redeemable Convertible Preferred Stock Prior to the IPO, the authorized capital stock of the Company included 63,491,857 shares of $0.0001 par value preferred stock, of which 3,333,333 shares have been designated as Series Seed Preferred Stock, 31,639,202 shares have been designated as Series A Preferred Stock and 28,519,322 shares have been designated as Series B Preferred Stock. On January 19, 2018, the Company entered into a stock purchase agreement for the sale of 28,285,557 shares of Series B Preferred Stock for $2.1389 per share. The total gross proceeds received were $60,500 and issuance costs were $2,243. In addition, the Company issued 233,765 shares of Series B Preferred Stock to BioMarin as required by the Company’s license agreement with BioMarin (Note 11). Upon closing of the IPO, all outstanding shares of Preferred Stock were converted into 15,320,213 shares of common stock. The holders of the Company’s Preferred Stock had certain voting, dividend, and redemption rights, as well as liquidation preferences and conversion privileges. All rights, preferences, and privileges associated with the preferred stock were terminated at the time of the Company’s IPO in conjunction with the conversion of all outstanding shares of Preferred Stock into shares of common stock. |
Common Stock
Common Stock | 12 Months Ended |
Dec. 31, 2019 | |
Federal Home Loan Banks [Abstract] | |
Common Stock | 9. Common Stock As of December 31, 2019 and 2018, the authorized capital stock of the Company included 150,000,000 shares of common stock, $0.0001 par value, and 10,000 shares of undesignated preferred stock. As of December 31, 2019 and 2018, 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, 2019, no cash dividends have been declared or paid. Initial Public Offering On June 20, 2018, the Company’s registration statement on Form S-1 relating to its IPO was declared effective by the SEC. The IPO closed on June 25, 2018 and the Company issued and sold 5,247,958 common shares at a public offering price of $19.00 per share for net proceeds of $90,103 after deducting underwriting discounts and commissions of $6,980 and other offering expenses of approximately $2,628. Simultaneously, on June 25, 2018, the Company issued and sold 787,193 additional common shares, pursuant to the full exercise of the underwriters’ option to purchase additional shares, for net proceeds of $13,910 after deducting underwriting discounts and commissions of $1,047. The aggregate net proceeds to the Company from the IPO, after deducting underwriting discounts and commissions and other offering costs, were $104,013. Follow-On Public Offering 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, 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 “July 2019 Follow-on Offering”). 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 approximately $129,500. Common Stock Reserved for Future Issuance At December 31, 2019 and 2018, the Company has reserved the following shares of common stock for future issuance: December 31, 2019 2018 Shares reserved for vesting of restricted stock awards 30,252 153,276 Shares reserved for exercise of outstanding stock options 3,414,445 2,164,101 Shares reserved for vesting of restricted stock units 2,300 Shares reserved for issuance under the 2018 Stock Option and Incentive Plan 332,513 385,561 Shares reserved for issuance under the 2018 Employee Stock Purchase Plan 459,595 223,200 Shares reserved for issuance under the 2019 Inducement Plan 1,800,000 — Total shares of authorized common stock reserved for future issuance 6,039,105 2,926,138 |
Stock-Based Compensation
Stock-Based Compensation | 12 Months Ended |
Dec. 31, 2019 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Stock-Based Compensation | 10. 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 will be made under 2015 plan. Shares that expire, are terminated, surrendered or canceled 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. The number of options available for future grant under the 2018 Plan was 332,513 as of December 31, 2019, which does not include the shares added to the 2018 Plan reserve on January 1, 2020 as a result of the Plan Evergreen for the year ended December 31, 2019. During the years ended December 31, 2019 and 2018, the Company granted options to purchase 1,677,967 and, 1,277,528 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, 2019 and 2018, the Company issued 1,671 and 0 shares, respectively of common stock. The total number of common shares that may be issued under the ESPP was 461,266 shares as of December 31, 2019, of which 459,595 shares remained available for future grant, and which does not include the shares added to the ESPP reserve on January 1, 2020 as a result of the ESPP Evergreen for the year ended December 31, 2019. 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 options available for future grant under the 2019 Plan was 1,800,000 as of December 31, 2019. 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, 2019 2018 Expected option life (years) 5.62 6.06 Risk-free interest rate 2.09 % 2.74 % Expected volatility 76.83 % 81.60 % Expected dividend yield — % — % The following table summarizes the Company’s stock option activity for the year ended December 31, 2019: Number of Options Weighted- Average Exercise Price Weighted- Average Remaining Contractual Term (Years) Aggregate Intrinsic Value Outstanding as of December 31, 2018 2,164,101 $ 7.75 8.77 $ 23,083 Granted 1,667,967 $ 16.50 Exercised (236,483 ) $ 2.12 Cancelled or forfeited (181,140 ) $ 14.13 Outstanding as of December 31, 2019 3,414,445 $ 12.08 8.43 $ 29,353 Exercisable as of December 31, 2019 977,229 $ 5.69 7.26 $ 14,702 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, 2019 and 2018 was $3,514 and $410, respectively. The weighted-average grant-date fair value of the Company’s stock options granted during the years ended December 31, 2019 and 2018 was $11.61 and $10.91, respectively. Restricted Common Stock The Company has granted restricted common stock with time-based vesting conditions to certain employees of the Company. The purchase price of the restricted stock awards are determined by the Board. Unvested shares of restricted common stock 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. The following table summarizes the Company’s restricted common stock activity for the year ended December 31, 2019: Number of Shares Weighted- Average Grant Date Fair Value Issued and unvested as of December 31, 2018 153,275 $ 0.42 Granted 2,300 $ 15.65 Vested (123,023 ) $ 0.42 Forfeited, canceled or expired — $ — Issued and unvested as of December 31, 2019 32,552 $ 1.50 The total fair value of restricted common stock vested during the years ended December 31, 2019 and 2018 was $50 and $52, respectively. Stock-Based Compensation Stock-based compensation expense was allocated as follows: Year Ended December 31, 2019 2018 Research and development $ 3,646 $ 920 General and administrative 3,154 1,276 Total stock-based compensation expense $ 6,800 $ 2,196 As of December 31, 2019, total unrecognized compensation cost related to unvested stock-based awards was $22,878, which is expected to be recognized over a weighted-average period of 3.14 years. |
License Agreements
License Agreements | 12 Months Ended |
Dec. 31, 2019 | |
Text Block [Abstract] | |
License Agreements | 11. License Agreements Agreements with 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, 2019 and 2018, the Company recorded research and development expense related to this agreement with UHN of $783 and $15, 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, 2019 and 2018. 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. The fair value of the shares issued to UHN of $480 and the upfront fee was expensed upon the execution of the agreement. In addition, the Company agreed to pay UHN up to $2,000 upon the closing of an IPO if certain criteria are met. This obligation was considered a derivative instrument and was initially recorded at fair value of $49 (Note 3). The Company is also required to pay UHN future annual license maintenance fees of CAD $50 on each anniversary of the effective date of the license agreement prior to expiration or termination and potential future milestone payments of up to CAD $19,275 upon the achievement of specified clinical and regulatory milestones. The Company also agreed to pay UHN royalties of a low single-digit percentage of net sales of licensed products sold by the Company. If the Company grants any sublicense rights under the license agreement, the Company has agreed to pay UHN a low double-digit royalty percentage of any sublicense income received by the Company. 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, 2019 and 2018, the Company recorded research and development expense related to this agreement with UHN of $38 and $41, 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, 2019 and 2018. 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. Both the upfront cash payment of $500 and the value of the shares Series B Preferred Stock issued of $500 were recorded as research and development expense during the year ended December 31, 2017. 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. The Company recorded research and development expense related to this agreement with BioMarin of $1,000 related to the license for the year ended December 31, 2017. No expenses related to the license were recorded for the years ended December 31, 2019 and 2018. 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 GenStem Therapeutics, Inc. (“GenStem”) 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. The Company recorded research and development expense of $1,000 for the year ended December 31, 2017, which consisted of upfront fees related to the license. For the year ended December 31, 2019, the Company recorded research and development expense of $2,000 , which related to milestone payments. No expense related to the license was recorded for the year ended December 31, 2018. 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, 2019 and 2018. |
Income Taxes
Income Taxes | 12 Months Ended |
Dec. 31, 2019 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | 12. Income Taxes For the years ended December 31, 2019 and 2018, 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, 2019 2018 Federal income tax expense at statutory rate 21.0 % 21.0 % State income taxes, net of federal benefit 5.2 % 5.2 % Permanent differences -0.9 % -0.8 % U.S.—TCJA 0.0 % 0.0 % Foreign rate differential 0.2 % 0.2 % Research and development tax credits 2.1 % 1.1 % Change in valuation allowance -27.6 % -26.5 % Provision to Return 0.0 % -0.2 % 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, 2019 2018 Deferred tax assets: U.S., foreign and state net operating loss carryforwards $ 33,594 $ 15,815 Research and development credits 1,716 454 Capitalized start up and organizational costs 32 35 Equity based compensation 543 185 Derivative liability — — Licensing agreements 1,756 1,258 Accruals and other 993 948 Total deferred tax assets 38,634 18,695 Valuation allowance (38,425 ) (18,457 ) Net deferred tax assets $ 209 $ 238 Deferred tax liabilities: Property and equipment $ (209 ) $ (238 ) 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, 2019 and 2018, 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, 2019 and 2018. The valuation allowance increased $19,968 and $11,991 during the years ended December 31, 2019 and 2018, respectively, due primarily to net operating losses generated. As of December 31, 2019 and 2018, the Company had U.S. federal net operating loss carryforwards of $121,836 and $57,279, 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 $104,093 of the U.S. federal tax operating losses can be carried forward indefinitely. As of December 31, 2019 and 2018, the Company also had U.S. state net operating loss carryforwards of $115,129 and $54,212, respectively, which may be available to offset future taxable income. These losses expire at various dates through 2039. As of December 31, 2019 and 2018, the Company had foreign net operating loss carryforwards of $2,440 and $1,201, respectively. The foreign net operating losses can be carried forward indefinitely. As of December 31, 2019 and 2018, the Company has federal research and development tax credit carryforwards of $1,547 and $339, respectively. Included in the $1,547 of federal tax credit carryforwards are $530 of orphan drug credits. 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, 2019 and 2018, the Company had state research and development tax credit carryforwards of approximately $214 and $146, respectively, available to reduce future tax liabilities which expire at various dates through 2033. For all years through December 31, 2019, 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, AVROBIO Australia, 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, 2019, the Company recorded $0.9 million in research and development tax credits as an offset to research and development expenses. 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 period in excess of 50 percent, as defined under Sections 382 and 383 of the Internal Revenue Code, respectively, as well as similar state provisions. This could limit the amount of tax attributes that can be utilized annually to offset future tax liabilities. The amount of the annual limitation is determined based on the value of the Company immediately prior to the ownership change. Subsequent ownership changes may further affect the limitation in future years. The Company has completed numerous financings since its inception, which may have resulted in a change in control as defined by Sections 382 and 383 of the Internal Revenue Code, or could result in a change in control in the future. 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, 2018. 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 Attributable
Net Loss per Share Attributable to Common Stockholders | 12 Months Ended |
Dec. 31, 2019 | |
Earnings Per Share [Abstract] | |
Net Loss per Share Attributable to Common Stockholders | 13. Net Loss per Share Attributable to Common Stockholders For purposes of the diluted net loss per share calculation, stock options and unvested restricted stock 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, 2019 2018 Options to purchase common stock 3,414,445 2,164,101 Restricted common stock 32,552 153,276 |
Commitments and Contingencies
Commitments and Contingencies | 12 Months Ended |
Dec. 31, 2019 | |
Commitments And Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 14. Commitments and Contingencies Lease Agreements In August 2017, the Company entered into a sub-lease agreement for laboratory space located in Cambridge Massachusetts, United States, which expires in August 2020. 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. On December 4, 2018, the Company terminated the sub-lease without any penalty. The leasehold improvement cost was written off to rental expenses during the year ended December 31, 2018. 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, with a landlord who is an affiliate of the landlord of the Company’s prior lease facility. 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 other assets as of December 31, 2019 and 2018. In contemplation of this agreement, the Company terminated its prior lease agreement. On August 31, 2018, the Company entered into a sub-lease agreement for lab space located in Cambridge Massachusetts, United States, which expires in October 2020. The annual lease payments are subject to a 3% increase each year. In accordance with the lease agreement, the Company is required to maintain a security deposit of $283, which was recorded in other assets as of December 31, 2019 and 2018. The Company recorded rent expense of $2,257 and $1,295 during the years ended December 31, 2019 and 2018, respectively. The following table summarizes the future minimum lease payments due under operating leases as of December 31, 2019: Year Ending December 31, 2020 $ 1,563 2021 705 2022 726 2023 61 $ 3,055 Legal Proceedings 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, 2019 and 2018, 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 at December 31, 2019 and 2018, 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 11 for discussion of these arrangements. 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. |
Related Party Transactions
Related Party Transactions | 12 Months Ended |
Dec. 31, 2019 | |
Related Party Transactions [Abstract] | |
Related Party Transactions | 15. 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, 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. See Note 3 for further discussion on the accounting treatment for this provision. For the years ended December 31, 2019 and 2018, the Company recognized $821 and $56, respectively, of research and development expense related to the license agreements with UHN. Refer to Note 11 for additional information regarding the UHN license agreements. Others For the years ended December 31, 2019 and 2018, the Company recorded expenses of $1,451 and $348, respectively, related to a sublease to rent lab space, provided by an entity affiliated with a member of the board. For the years ended December 31, 2019 and 2018, the Company recorded expenses of $0 and $40, respectively, related to consulting services provided by an entity affiliated with an officer of the Company and a member of the Board. |
Benefit Plans
Benefit Plans | 12 Months Ended |
Dec. 31, 2019 | |
Postemployment Benefits [Abstract] | |
Benefit Plans | 16. Benefit Plans The Company established a defined contribution savings plan under Section 401(k) of the Internal Revenue Code. This plan covers substantially all employees who meet minimum age and service requirements and allows participants to defer a portion of their annual compensation on a pre-tax basis. In August 2018, the Company’s Board approved a company matching contribution of up to 3%, effective October 1, 2018. The Company made matching contributions of $224 and $35 to the plan during the years ended December 31, 2019 and 2018, respectively. |
Subsequent Events
Subsequent Events | 12 Months Ended |
Dec. 31, 2019 | |
Subsequent Events [Abstract] | |
Subsequent Events | 17. Subsequent Events On December 20, 2019, the Company filed a shelf registration statement on Form S-3 with the SEC (the “December 2019 Shelf,” which covers the offering, issuance and sale by the Company of up to an aggregate of $250.0 million of the Company’s common stock, preferred stock, debt securities, warrants and/or units. The December 2019 Shelf was declared effective by the SEC on January 14, 2020. In February 2020, the Company closed an underwritten public offering of 4,350,000 shares of its common stock at a public offering price of $23.00 per share, less underwriting discounts and commissions. The net proceeds to the Company from this offering, after deducting underwriting discounts and commissions and other estimated offering expenses payable by the Company, were approximately $93,547. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 12 Months Ended |
Dec. 31, 2019 | |
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. |
Use of Estimates | Use of Estimates The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the consolidated financial statements and the reported amounts of revenue and expenses during the reporting periods. Significant estimates and assumptions reflected in these consolidated financial statements include, but are not limited to, the accrual for research and development expenses, stock-based compensation expense, the valuation of equity and derivative instruments and the recoverability of the Company’s net deferred tax assets and related valuation allowance. Estimates are periodically reviewed in light of changes in circumstances, facts and experience. Changes in estimates are recorded in the period in which they become known. Actual results could differ materially from those estimates. |
Cash and Cash Equivalents | Cash and Cash Equivalents The Company considers all highly liquid investments purchased with original maturities of 90 days or less at acquisition to be cash equivalents. Cash and cash equivalents include cash held in banks and amounts held in interest-bearing money market accounts. Cash equivalents are carried at cost, which approximates their fair market value. |
Restricted Cash | Restricted Cash As of both December 31, 2019 and 2018, restricted cash consisted of $492 |
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. |
Deferred Issuance Costs | Deferred Issuance Costs The Company capitalizes certain legal, professional accounting and other third-party fees that are directly associated with in-process equity financings as deferred issuance costs until such financings are consummated. After consummation of the equity financing, these costs are recorded as a reduction of the proceeds generated as a result of the offering. Should the planned equity financing be abandoned, the deferred issuance costs will be expensed immediately as a charge to operating expenses in the consolidated statements of operations. As of December 31, 2019, the Company recorded deferred issuance costs of $133 within other assets on the consolidated balance sheet related to a follow-on offering of common stock completed in February 2020. There were no amounts deferred as of December 31, 2018. |
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 and software 2 years Leasehold improvements Lesser of lease term or 10 years Upon disposal, retirement or sale, the cost of assets disposed of and the related accumulated depreciation are removed from the accounts and any resulting gain or loss is included in the results of operations. Expenditures for repairs and maintenance that do not improve or extend the lives of the respective assets are charged to expense as incurred. |
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. During the year ended December 31, 2018, the Company recorded an impairment loss of $235 on long-lived assets related to leasehold improvements. The Company did not record any impairment loss during the year ended December 31, 2019. |
Fair Value Measurements | Fair Value Measurements Certain assets and liabilities of the Company are carried at fair value under GAAP (see Note 3). 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—Quoted prices in active markets for identical assets or liabilities. • 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 and cash equivalents, restricted cash, accounts payable, and accrued expenses, approximated their fair values at December 31, 2019 and 2018 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 3 for further discussion. |
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. |
Research and Development Costs | Research and Development Costs 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 the 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, 2019, 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. See Note 10 for the assumptions used by the Company in determining the grant date fair value of stock-based awards granted, as well as a summary of the stock-based award activity under the Company’s stock-based compensation plan for the year ended December 31, 2019. |
Warrant to Purchase Preferred Stock | Warrant to Purchase Preferred Stock Prior to the IPO, the Company classified the warrant for the purchase of shares of its redeemable convertible preferred stock (see Note 7) as a liability on its consolidated balance sheets as the warrant was a free-standing financial instrument that may require the Company to transfer assets upon exercise. The preferred stock warrant liability was initially recorded at fair value upon the date of issuance and was subsequently remeasured to fair value at each reporting date. Changes in the fair value of the warrant to purchase preferred stock were recognized as a component of other income (expense), net in the consolidated statements of operations and comprehensive loss. The Company utilized the Black-Scholes option-pricing model, which incorporates assumptions and estimates, to value the warrant. The Company assessed these assumptions and estimates on a quarterly basis as additional information impacting the assumptions was obtained. Estimates and assumptions impacting the fair value measurement included the fair value per share of the underlying redeemable convertible preferred stock issuable upon exercise of the warrant, the remaining contractual term of the warrant, the risk-free interest rate, the expected dividend yield and the expected volatility of the price of the underlying redeemable convertible preferred stock. Upon the IPO, the warrant to purchase preferred stock was converted to a warrant to purchase common stock. The carrying amount of the warrant to purchase preferred stock as of the date of IPO was transferred to the account of additional paid in capital. No further revaluation was needed for the warrant to purchase common stock. |
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’ (deficit) equity which includes certain changes in equity that are excluded from net income (loss). Comprehensive loss has been disclosed in the accompanying 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 income (expense), net in the consolidated statements of operations and comprehensive loss. During the years ended December 31, 2019 and 2018, the Company recorded foreign exchange losses of $90 and $51, respectively, in other expense. |
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. |
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 other 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. |
Net Income (Loss) per Share Attributable to Common Stockholders | Net Income (Loss) per Share Attributable to Common Stockholders Net income (loss) per share attributable to common stockholders is determined using the two-class method, which includes the weighted-average number of shares of common stock outstanding during the period and other securities that participate in dividends (a participating security). In periods of income, the redeemable convertible preferred stock would be considered participating securities because the shares include rights to participate in dividends with the common stock; however, the redeemable convertible preferred stock is not considered a participating security in periods of loss as they do not have an obligation to share in the Company’s net losses. Under the two-class method, basic net income (loss) per share attributable to common stockholders is computed by dividing the net income (loss) attributable to common stockholders by the weighted-average number of shares of common stock outstanding during the period. Diluted net income (loss) per share attributable to common stockholders is computed using the more dilutive of (1) the two-class method or (2) the if-converted method. The Company allocates net income first to the holders of Preferred Stock based on dividend rights under the Company’s certificate of incorporation and then to preferred and common stockholders based on ownership interests. |
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. The Company has evaluated all subsequent events and determined that there are no material recognized or unrecognized subsequent events requiring disclosure, other than as disclosed in these notes to the consolidated financial statements. See Note 17 for further information. |
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 Adopted Accounting Pronouncements | Recently Adopted Accounting Pronouncements In June 2018, the FASB issued ASU No. 2018-07, Compensation-Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting Compensation-Stock Compensation In May 2014, the FASB issued ASU No. 2014-09, Revenue from Contracts with Customers (Topic 606) In May 2017, the FASB issued ASU No. 2017-09, Compensation—Stock Compensation (Topic 718): Scope of Modification Accounting In November 2016, the FASB issued ASU No. 2016-18, Statement of Cash Flows (Topic 230) Restricted Cash December 31, 2019 2018 Cash and cash equivalents $ 187,043 $ 126,302 Other assets 492 492 Cash, cash equivalents and restricted cash $ 187,535 $ 126,794 |
Recently Issued Accounting Pronouncements | Recently Issued Accounting Pronouncements In December 2019, the FASB issued ASU No. 2019-12, Simplifying the Accounting for Income Taxes In August 2018, the FASB issued ASU No. 2018-13, Fair Value Measurement (Topic 820): Disclosure Framework—Changes to the Disclosure Requirements for Fair Value Measurement In August 2018, the FASB issued ASU No. 2018-15, Intangible-Goodwill and Other Internal-Use Software (Subtopic 350-40) (“ASU 2018-15”). ASU 2018-15 updates guidance regarding accounting for implementation costs associated with a cloud computing arrangement that is a service contract. The amendments under ASU 2018-15 are effective for interim and annual fiscal periods beginning after December 15, 2019, with early adoption permitted. The Company does not expect the adoption of ASU 2018-15 to have a material impact on its consolidated financial statements. In November 2018, the FASB issued ASU No. 2018-18, Collaborative Arrangements (Topic 808)—Clarifying the Interaction between Topic 808 and Topic 606 (“ASU 2018-18”). The amendments in ASU 2018-18 clarify that certain transactions between collaborative arrangement participants should be accounted for as revenue under ASC 606 when the collaborative arrangement participant is a customer in the context of a unit of account. The amendments under ASU 2018-18 are effective for interim and annual fiscal periods beginning after December 15, 2019, with early adoption permitted. The amendments in ASU 2018-18 should be applied retrospectively to the date of initial application of ASC 606. The Company does not expect the adoption of ASU 2018-18 to have a material impact on its consolidated financial statements. In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments—Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments (“ASU 2016-13”). ASU 2016-13 requires that credit losses be reported as an allowance using an expected losses model, representing the entity's current estimate of credit losses expected to be incurred. The accounting guidance currently in effect is based on an incurred loss model. For available-for-sale debt securities with unrealized losses, this standard now requires allowances to be recorded instead of reducing the amortized cost of the investment. The amendments under ASU 2016-13 are effective for interim and annual fiscal periods beginning after December 15, 2019. The Company does not expect the adoption of ASC 2016-13 to have a material impact on its consolidated financial statements. In February 2016, the FASB issued ASU No. 2016-02, (Topic 842) Leases (“ASU 2016-02”). ASU 2016-02 requires an entity to recognize assets and liabilities arising from a lease for both financing and operating leases. The ASU will also require new qualitative and quantitative disclosures to help investors and other financial statement users better understand the amount, timing, and uncertainty of cash flows arising from leases. For public entities, ASU 2016-02 is effective for fiscal years beginning after December 15, 2018. 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, ASU 2016-02 is effective for the Company for the year ended December 31, 2021, and all interim periods thereafter. Early adoption is permitted. The Company is currently evaluating the impact that the adoption of ASU 2016-02 will have on its consolidated financial statements. |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
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 and software 2 years Leasehold improvements Lesser of lease term or 10 years |
Schedule Of Cash And Cash Equivalents And Restricted Cash | The cash, cash equivalents and restricted cash balances as of December 31, 2019 and 2018, which are presented in the Company’s consolidated statements of cash flows subsequent to the adoption of ASU 2016-18, consisted of the following: December 31, 2019 2018 Cash and cash equivalents $ 187,043 $ 126,302 Other assets 492 492 Cash, cash equivalents and restricted cash $ 187,535 $ 126,794 |
Fair Value of Financial Asset_2
Fair Value of Financial Assets and Liabilities (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
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, 2019 and 2018: Fair Value Measurements as of December 31, 2019 Using: Level 1 Level 2 Level 3 Total Assets: Money market funds $ 186,797 $ — $ — $ 186,797 Restricted cash 492 — — 492 $ 187,289 $ — $ — $ 187,289 Fair Value Measurements as of December 31, 2018 Using: Level 1 Level 2 Level 3 Total Assets: Money market funds $ 126,047 $ — $ — $ 126,047 Restricted cash 492 — — 492 $ 126,539 $ — $ — $ 126,539 |
Warrants to Purchase Redeemable Convertible Preferred Stock [Member] | |
Summary of Changes in Fair Value Determined by Level 3 Inputs | The following table sets forth a summary of changes in the fair value of the Company’s preferred stock warrant liability for which fair value was determined by Level 3 inputs: Warrant Liability Balance as of December 31, 2017 $ 35 Change in fair value 162 Conversion of preferred stock warrant to common stock warrant (197 ) Balance as of December 31, 2018 $ — |
Derivative Liabilities [Member] | |
Summary of Changes in Fair Value Determined by Level 3 Inputs | The following table sets forth a summary of changes in the fair value of the Company’s derivative liability for which fair value is determined by Level 3 inputs: Derivative Liability Balance as of December 31, 2017 $ 371 Change in fair value 1,629 Payment (2,000 ) Balance as of December 31, 2018 $ — |
Prepaid Expenses and Other Cu_2
Prepaid Expenses and Other Current Assets (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Text Block [Abstract] | |
Summary of Prepaid Expenses and Other Current Assets | Prepaid expenses and other current assets consisted of the following: December 31, 2019 2018 Tax incentive refund $ 1,916 $ 1,325 Prepaid research and development costs 4,915 981 Prepaid insurance 897 316 Interest income receivable 224 220 Prepaid rent 86 81 Other current assets 620 795 $ 8,658 $ 3,718 |
Property and Equipment, Net (Ta
Property and Equipment, Net (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Property Plant And Equipment [Abstract] | |
Summary of Property and Equipment, Net | Property and equipment, net consisted of the following: December 31, 2019 2018 Laboratory and office equipment $ 3,456 $ 1,624 Leasehold improvements 1,340 1,260 Computer equipment and software 134 134 4,930 3,018 Less: Accumulated depreciation and amortization (1,234 ) (384 ) $ 3,696 $ 2,634 |
Accrued Expenses (Tables)
Accrued Expenses (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Payables And Accruals [Abstract] | |
Summary of Accrued Expenses | Accrued expenses consisted of the following: December 31, 2019 2018 Compensation and benefit costs $ 3,028 $ 2,616 Research and development costs 5,794 3,969 Consulting and professional fees 917 320 Other liabilities 329 917 $ 10,068 $ 7,822 |
Common Stock (Tables)
Common Stock (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Federal Home Loan Banks [Abstract] | |
Summary of Common Stock Reserved for Future Issuance | At December 31, 2019 and 2018, the Company has reserved the following shares of common stock for future issuance: December 31, 2019 2018 Shares reserved for vesting of restricted stock awards 30,252 153,276 Shares reserved for exercise of outstanding stock options 3,414,445 2,164,101 Shares reserved for vesting of restricted stock units 2,300 Shares reserved for issuance under the 2018 Stock Option and Incentive Plan 332,513 385,561 Shares reserved for issuance under the 2018 Employee Stock Purchase Plan 459,595 223,200 Shares reserved for issuance under the 2019 Inducement Plan 1,800,000 — Total shares of authorized common stock reserved for future issuance 6,039,105 2,926,138 |
Stock-Based Compensation (Table
Stock-Based Compensation (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
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, 2019 2018 Expected option life (years) 5.62 6.06 Risk-free interest rate 2.09 % 2.74 % Expected volatility 76.83 % 81.60 % Expected dividend yield — % — % |
Summary of Stock Option Activity | The following table summarizes the Company’s stock option activity for the year ended December 31, 2019: Number of Options Weighted- Average Exercise Price Weighted- Average Remaining Contractual Term (Years) Aggregate Intrinsic Value Outstanding as of December 31, 2018 2,164,101 $ 7.75 8.77 $ 23,083 Granted 1,667,967 $ 16.50 Exercised (236,483 ) $ 2.12 Cancelled or forfeited (181,140 ) $ 14.13 Outstanding as of December 31, 2019 3,414,445 $ 12.08 8.43 $ 29,353 Exercisable as of December 31, 2019 977,229 $ 5.69 7.26 $ 14,702 |
Summary of Restricted Stock Unit Activity | The following table summarizes the Company’s restricted common stock activity for the year ended December 31, 2019: Number of Shares Weighted- Average Grant Date Fair Value Issued and unvested as of December 31, 2018 153,275 $ 0.42 Granted 2,300 $ 15.65 Vested (123,023 ) $ 0.42 Forfeited, canceled or expired — $ — Issued and unvested as of December 31, 2019 32,552 $ 1.50 |
Schedule of Stock-based Compensation Expense | Stock-based compensation expense was allocated as follows: Year Ended December 31, 2019 2018 Research and development $ 3,646 $ 920 General and administrative 3,154 1,276 Total stock-based compensation expense $ 6,800 $ 2,196 |
Income Taxes (Tables)
Income Taxes (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
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, 2019 2018 Federal income tax expense at statutory rate 21.0 % 21.0 % State income taxes, net of federal benefit 5.2 % 5.2 % Permanent differences -0.9 % -0.8 % U.S.—TCJA 0.0 % 0.0 % Foreign rate differential 0.2 % 0.2 % Research and development tax credits 2.1 % 1.1 % Change in valuation allowance -27.6 % -26.5 % Provision to Return 0.0 % -0.2 % 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, 2019 2018 Deferred tax assets: U.S., foreign and state net operating loss carryforwards $ 33,594 $ 15,815 Research and development credits 1,716 454 Capitalized start up and organizational costs 32 35 Equity based compensation 543 185 Derivative liability — — Licensing agreements 1,756 1,258 Accruals and other 993 948 Total deferred tax assets 38,634 18,695 Valuation allowance (38,425 ) (18,457 ) Net deferred tax assets $ 209 $ 238 Deferred tax liabilities: Property and equipment $ (209 ) $ (238 ) Total deferred tax liabilities $ — $ — |
Net Loss per Share Attributab_2
Net Loss per Share Attributable to Common Stockholders (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
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, 2019 2018 Options to purchase common stock 3,414,445 2,164,101 Restricted common stock 32,552 153,276 |
Commitments and Contingencies (
Commitments and Contingencies (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Commitments And Contingencies Disclosure [Abstract] | |
Schedule Of Future Minimum Rental Payment Under Operating Lease | The following table summarizes the future minimum lease payments due under operating leases as of December 31, 2019: Year Ending December 31, 2020 $ 1,563 2021 705 2022 726 2023 61 $ 3,055 |
Nature of the Business - Additi
Nature of the Business - Additional Information (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | ||
Net loss | $ (72,965) | $ (46,361) |
Accumulated deficit | (144,704) | (71,739) |
Cash and cash equivalents | $ 187,043 | $ 126,302 |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies - Additional Information (Detail) | 12 Months Ended | |
Dec. 31, 2019USD ($)Segment | Dec. 31, 2018USD ($) | |
Significant Of Accounting Policies [Line Items] | ||
Restricted cash | $ 492,000 | $ 492,000 |
Deferred issuance costs | 133,000,000 | 0 |
Impairment loss of property and equipment | $ 0 | 235,000 |
Number of operating segments | Segment | 1 | |
Foreign exchange losses | $ 90,000 | $ 51,000 |
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 - Estimated Useful Lives of Property and Equipment (Detail) | 12 Months Ended |
Dec. 31, 2019 | |
Laboratory and Office Equipment [Member] | |
Property, Plant and Equipment [Line Items] | |
Property and equipment estimated useful life | 5 years |
Computer Equipment and Software [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 |
Summary of Significant Accoun_6
Summary of Significant Accounting Policies - Statements of cash flows subsequent to the adoption of ASU (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 |
Cash And Cash Equivalents [Abstract] | |||
Cash and cash equivalents | $ 187,043 | $ 126,302 | |
Other assets | 492 | 492 | |
Cash, cash equivalents and restricted cash | $ 187,535 | $ 126,794 | $ 5,987 |
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 ($) | Dec. 31, 2019 | Dec. 31, 2018 |
Assets Fair Value Disclosure | ||
Assets Fair Value Disclosure | $ 187,289 | $ 126,539 |
Level 1 [Member] | ||
Assets Fair Value Disclosure | ||
Assets Fair Value Disclosure | 187,289 | 126,539 |
Money Market Funds [Member] | ||
Assets Fair Value Disclosure | ||
Assets Fair Value Disclosure | 186,797 | 126,047 |
Money Market Funds [Member] | Level 1 [Member] | ||
Assets Fair Value Disclosure | ||
Assets Fair Value Disclosure | 186,797 | 126,047 |
Restricted Cash [Member] | ||
Assets Fair Value Disclosure | ||
Assets Fair Value Disclosure | 492 | 492 |
Restricted Cash [Member] | Level 1 [Member] | ||
Assets Fair Value Disclosure | ||
Assets Fair Value Disclosure | $ 492 | $ 492 |
Fair Value of Financial Asset_4
Fair Value of Financial Assets and Liabilities - Additional Information (Detail) - USD ($) | Jan. 27, 2016 | Jul. 31, 2018 | Dec. 31, 2019 | Dec. 31, 2018 |
Fair Value of Assets and Liabilities Measured on Non Recurring Basis [Line Items] | ||||
Fair value input transfer between levels | $ 0 | $ 0 | ||
Common stock issued for initial consideration for license | 2,300 | |||
Increase in fair value of derivative | $ (1,629,000) | |||
IPO [Member] | ||||
Fair Value of Assets and Liabilities Measured on Non Recurring Basis [Line Items] | ||||
Dilution payment | $ 2,000,000 | |||
License Agreement [Member] | ||||
Fair Value of Assets and Liabilities Measured on Non Recurring Basis [Line Items] | ||||
Provision for maximum cash payment | $ 2,000,000 | $ 2,000,000 | ||
License Agreement [Member] | Stock Purchase Agreement [Member] | ||||
Fair Value of Assets and Liabilities Measured on Non Recurring Basis [Line Items] | ||||
Common stock issued for initial consideration for license | 1,161,665 |
Fair Value of Financial Asset_5
Fair Value of Financial Assets and Liabilities - Summary of Changes in Fair Value of Preferred Stock Warrant Liability for Fair Value Determined by Level 3 Inputs (Detail) - Level 3 [Member] - Warrants to Purchase Redeemable Convertible Preferred Stock [Member] $ in Thousands | 12 Months Ended |
Dec. 31, 2018USD ($) | |
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items] | |
Beginning balance | $ 35 |
Change in fair value | 162 |
Conversion of preferred stock warrant to common stock warrant | $ (197) |
Fair Value of Financial Asset_6
Fair Value of Financial Assets and Liabilities - Summary of Changes in Fair Value of Derivative Liability for Fair Value Determined by Level 3 Inputs (Detail) - Level 3 [Member] - Derivative Liabilities [Member] $ in Thousands | 12 Months Ended |
Dec. 31, 2018USD ($) | |
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items] | |
Beginning balance | $ 371 |
Change in fair value | 1,629 |
Payment | $ (2,000) |
Prepaid Expenses and Other Cu_3
Prepaid Expenses and Other Current Assets - Summary of Prepaid Expenses and Other Current Assets (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Prepaid Expense And Other Assets Current [Abstract] | ||
Tax incentive refund | $ 1,916 | $ 1,325 |
Prepaid research and development costs | 4,915 | 981 |
Prepaid insurance | 897 | 316 |
Interest income receivable | 224 | 220 |
Prepaid rent | 86 | 81 |
Other current assets | 620 | 795 |
Total | $ 8,658 | $ 3,718 |
Property and Equipment, Net - S
Property and Equipment, Net - Summary of Property and Equipment, Net (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Property, Plant and Equipment [Line Items] | ||
Property plant and equipment, gross | $ 4,930 | $ 3,018 |
Less: Accumulated depreciation and amortization | (1,234) | (384) |
Property plant and equipment, net | 3,696 | 2,634 |
Laboratory and Office Equipment [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property plant and equipment, gross | 3,456 | 1,624 |
Leasehold Improvements [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property plant and equipment, gross | 1,340 | 1,260 |
Computer Equipment and Software [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property plant and equipment, gross | $ 134 | $ 134 |
Property and Equipment, Net - A
Property and Equipment, Net - Additional Information (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Property Plant And Equipment [Abstract] | ||
Depreciation and amortization expense | $ 850 | $ 380 |
Accrued Expenses - Summary of A
Accrued Expenses - Summary of Accrued Expenses (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Accrued Liabilities Current [Abstract] | ||
Compensation and benefit costs | $ 3,028 | $ 2,616 |
Research and development costs | 5,794 | 3,969 |
Consulting and professional fees | 917 | 320 |
Other liabilities | 329 | 917 |
Accrued expenses | $ 10,068 | $ 7,822 |
Loan Agreement and Warrant to_2
Loan Agreement and Warrant to Purchase Preferred Stock - Additional Information (Detail) - USD ($) | Jul. 13, 2018 | Jun. 23, 2017 | Dec. 31, 2019 | Dec. 31, 2018 |
Class of Warrant or Right [Line Items] | ||||
Maximum issuance of term loans | $ 10,000,000 | |||
Loan expiration date | Oct. 31, 2018 | |||
Loss related to change in fair value of warrant | $ 162,000 | |||
Purchase of common stock | 6,850 | |||
Issuance of common stock | 6,091 | 31,673,058 | 23,959,903 | |
Series A Preferred Stock [Member] | ||||
Class of Warrant or Right [Line Items] | ||||
Warrant to purchase shares | 188,702 | |||
Warrant exercise price | $ 0.7949 | |||
Warrant expiration date | Jun. 22, 2027 | |||
Shares purchase upon exercise of warrant | 28,305 | |||
Additional shares purchase upon exercise of warrant | 160,397 | |||
Prime Rate [Member] | ||||
Class of Warrant or Right [Line Items] | ||||
Floating interest rate per annum | 3.00% | |||
Minimum [Member] | ||||
Class of Warrant or Right [Line Items] | ||||
Floating interest rate per annum | 1.00% |
Redeemable Convertible Prefer_2
Redeemable Convertible Preferred Stock - Additional Information (Detail) - USD ($) | Mar. 31, 2018 | Jan. 19, 2018 | Dec. 31, 2018 | Dec. 31, 2019 | Jun. 25, 2018 |
Temporary Equity [Line Items] | |||||
Redeemable convertible preferred stock, shares authorized | 63,491,857 | ||||
Redeemable convertible preferred stock, par value | $ 0.0001 | ||||
Proceeds from issuance of redeemable convertible preferred stock | $ 58,257,000 | ||||
IPO [Member] | |||||
Temporary Equity [Line Items] | |||||
Redeemable convertible preferred stock, sales price per share | $ 19 | ||||
Outstanding shares of preferred stock converted into common stock | 15,320,213 | ||||
Series Seed Redeemable Convertible Preferred Stock [Member] | |||||
Temporary Equity [Line Items] | |||||
Redeemable convertible preferred stock, shares authorized | 3,333,333 | ||||
Series A Redeemable Convertible Preferred Stock [Member] | |||||
Temporary Equity [Line Items] | |||||
Redeemable convertible preferred stock, shares authorized | 31,639,202 | ||||
Series B Redeemable Convertible Preferred Stock [Member] | |||||
Temporary Equity [Line Items] | |||||
Redeemable convertible preferred stock, shares authorized | 28,519,322 | ||||
Redeemable convertible preferred stock, shares issued and sold | 28,285,557 | ||||
Redeemable convertible preferred stock, sales price per share | $ 2.1389 | ||||
Proceeds from issuance of redeemable convertible preferred stock | $ 60,500,000 | ||||
Redeemable convertible preferred stock, net of issuance costs | $ 2,243 | ||||
Series B Redeemable Convertible Preferred Stock [Member] | BioMarin Pharmaceutical Inc [Member] | |||||
Temporary Equity [Line Items] | |||||
Redeemable convertible preferred stock, shares issued and sold | 233,765 |
Common Stock - Additional Infor
Common Stock - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Thousands | Jun. 25, 2018 | Jul. 31, 2019 | Dec. 31, 2019 | Dec. 31, 2018 |
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 | 10,000 | ||
Undesignated shares of preferred stock outstanding | 0 | 0 | ||
Cash dividends | $ 0 | |||
Net proceeds from issuance of common stock | $ 129,465 | $ 104,013 | ||
IPO [Member] | ||||
Class of Stock [Line Items] | ||||
Common stock, shares issued and sold | 5,247,958 | |||
Issuance price per shares | $ 19 | |||
Net proceeds from issuance of common stock | $ 90,103 | |||
Net proceeds after deducting underwriting discounts and commissions and offering costs | 6,980 | |||
Other offering expenses | $ 2,628 | |||
Over-Allotment Option [Member] | ||||
Class of Stock [Line Items] | ||||
Common stock, shares issued and sold | 787,193 | |||
Net proceeds from issuance of common stock | $ 13,910 | |||
Net proceeds after deducting underwriting discounts and commissions and offering costs | 1,047 | |||
Other offering expenses | $ 104,013 | |||
Over-Allotment Option [Member] | Common Stock [Member] | ||||
Class of Stock [Line Items] | ||||
Common stock, shares issued and sold | 975,000 | |||
July 2019 Follow-on Offering [Member] | ||||
Class of Stock [Line Items] | ||||
Net proceeds from issuance of common stock | $ 129,500 | |||
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 |
Common Stock - Summary of Commo
Common Stock - Summary of Common Stock Reserved for Future Issuance (Detail) - shares | Dec. 31, 2019 | Dec. 31, 2018 |
Class of Stock [Line Items] | ||
Total shares of authorized common stock reserved for future issuance | 6,039,105 | 2,926,138 |
Restricted Stock [Member] | ||
Class of Stock [Line Items] | ||
Total shares of authorized common stock reserved for future issuance | 30,252 | 153,276 |
Restricted Stock Units [Member] | ||
Class of Stock [Line Items] | ||
Total shares of authorized common stock reserved for future issuance | 2,300 | |
Employee Stock Option [Member] | ||
Class of Stock [Line Items] | ||
Total shares of authorized common stock reserved for future issuance | 3,414,445 | 2,164,101 |
2018 Stock Option and Incentive Plan [Member] | ||
Class of Stock [Line Items] | ||
Total shares of authorized common stock reserved for future issuance | 332,513 | 385,561 |
2018 Employee Stock Purchase Plan [Member] | ||
Class of Stock [Line Items] | ||
Total shares of authorized common stock reserved for future issuance | 459,595 | 223,200 |
2019 Inducement Plan [Member] | ||
Class of Stock [Line Items] | ||
Total shares of authorized common stock reserved for future issuance | 1,800,000 |
Stock-Based Compensation - Addi
Stock-Based Compensation - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Number of options granted | 1,667,967 | |
Aggregate intrinsic value of options exercised | $ 3,514 | $ 410 |
Options Granted, Weighted-average grant-date fair value | $ 11.61 | $ 10.91 |
Total fair value of restricted common stock vested | $ 50 | $ 52 |
Unrecognized stock-based compensation expenses | $ 22,878 | |
Unrecognized stock-based compensation expense, period for recognition | 3 years 1 month 20 days | |
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 | 332,513 | |
Number of shares of common stock outstanding, increase, percentage | 4.00% | |
Number of options granted | 1,677,967 | 1,277,528 |
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] | ||
Number of common shares that may be issued under the plan | 461,266 | |
Common shares available for future grant | 459,595 | |
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 | 1,671 | 0 |
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 | 1,800,000 |
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, 2019 | Dec. 31, 2018 | |
Share Based Compensation Arrangement By Share Based Payment Award Fair Value Assumptions And Methodology [Abstract] | ||
Expected option life (years) | 5 years 7 months 13 days | 6 years 21 days |
Risk-free interest rate | 2.09% | 2.74% |
Expected volatility | 76.83% | 81.60% |
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, 2019 | Dec. 31, 2018 | |
Share Based Compensation Arrangement By Share Based Payment Award Options Outstanding Roll Forward | ||
Number of options, Outstanding beginning balance | 2,164,101 | |
Number of options, Granted | 1,667,967 | |
Number of options, Exercised | (236,483) | |
Number of options, Cancelled or forfeited | (181,140) | |
Number of options, Outstanding ending balance | 3,414,445 | 2,164,101 |
Number of options, Exercisable | 977,229 | |
Weighted average exercise price, Outstanding beginning balance | $ 7.75 | |
Weighted average exercise price, Granted | 16.50 | |
Weighted average exercise price, Exercised | 2.12 | |
Weighted average exercise price, Cancelled or forfeited | 14.13 | |
Weighted average exercise price, Outstanding ending balance | 12.08 | $ 7.75 |
Weighted average exercise price, Exercisable | $ 5.69 | |
Weighted average remaining contractual term, Outstanding balance | 8 years 5 months 4 days | 8 years 9 months 7 days |
Weighted average remaining contractual term, Exercisable | 7 years 3 months 3 days | |
Aggregate intrinsic value, Outstanding balance | $ 29,353 | $ 23,083 |
Aggregate intrinsic value, Exercisable | $ 14,702 |
Stock-Based Compensation - Su_3
Stock-Based Compensation - Summary of Restricted Stock Unit Activity (Detail) | 12 Months Ended |
Dec. 31, 2019$ / sharesshares | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Number of shares, Issued and unvested beginning balance | shares | 153,275 |
Number of shares, Granted | shares | 2,300 |
Number of shares, Vested | shares | (123,023) |
Number of shares, Issued and unvested ending balance | shares | 32,552 |
Weighted average grant date fair value, Issued and unvested, beginning balance | $ / shares | $ 0.42 |
Weighted average grant date fair value, Granted | $ / shares | 15.65 |
Weighted average grant date fair value, Vested | $ / shares | 0.42 |
Weighted average grant date fair value, Issued and unvested, ending balance | $ / shares | $ 1.50 |
Stock-Based Compensation - Sche
Stock-Based Compensation - Schedule of Stock-based Compensation Expense (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||
Total stock-based compensation expense | $ 6,800 | $ 2,196 |
Research and Development Expense [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||
Total stock-based compensation expense | 3,646 | 920 |
General and Administrative Expense [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||
Total stock-based compensation expense | $ 3,154 | $ 1,276 |
License Agreements - Additional
License Agreements - Additional Information (Detail) $ in Thousands | Oct. 02, 2017USD ($) | Nov. 17, 2016USD ($) | Jan. 27, 2016USD ($)shares | Jan. 27, 2016CAD ($) | Aug. 31, 2017USD ($)shares | Dec. 31, 2019USD ($)shares | Dec. 31, 2018USD ($)shares | Dec. 31, 2017USD ($) | Jul. 13, 2018shares | Jan. 27, 2016CAD ($)shares |
License Agreement [Line Items] | ||||||||||
Common stock, shares issued | shares | 31,673,058 | 23,959,903 | 6,091 | |||||||
Research and development | $ 54,974,000 | $ 35,095,000 | ||||||||
Preferred stock issued | shares | 0 | 0 | ||||||||
UHN Agreement [Member] | ||||||||||
License Agreement [Line Items] | ||||||||||
Research and development expense | $ 783,000 | $ 15,000 | ||||||||
Milestone Fees | 0 | 0 | ||||||||
UHN Agreement [Member] | Fabry License Agreement [Member] | ||||||||||
License Agreement [Line Items] | ||||||||||
Option fee | $ 20 | |||||||||
Upfront license fee | $ 75 | |||||||||
Milestone payments | 2,450 | |||||||||
UHN Agreement [Member] | Interleukin 12 License Agreement [Member] | ||||||||||
License Agreement [Line Items] | ||||||||||
Upfront license fee | 264 | |||||||||
Milestone payments | 19,275 | |||||||||
Milestone Fees | 0 | 0 | ||||||||
Annual maintenance fees | $ 50 | |||||||||
Common stock, shares issued | shares | 1,161,665 | 1,161,665 | ||||||||
Fair value of shares issued | $ 480 | |||||||||
Payments upon closing of an initial public offering | 2,000,000 | |||||||||
Fair value of derivative liability | $ 49,000 | |||||||||
Research and development | 38,000 | 41,000 | ||||||||
BioMarin Pharmaceutical Inc [Member] | ||||||||||
License Agreement [Line Items] | ||||||||||
Upfront license fee | $ 500,000 | |||||||||
Research and development | $ 1,000,000 | |||||||||
Milestone payments | 13,000,000 | |||||||||
Upfront cash payment | 500,000 | |||||||||
Expenses related to license | 0 | 0 | ||||||||
BioMarin Pharmaceutical Inc [Member] | Series B Preferred Stock [Member] | ||||||||||
License Agreement [Line Items] | ||||||||||
Research and development | 500,000 | |||||||||
Preferred stock issued | shares | 233,765 | |||||||||
GenStem Therapeutics Inc [Member] | ||||||||||
License Agreement [Line Items] | ||||||||||
Upfront license fee | $ 1,000,000 | |||||||||
Research and development | 2,000,000 | $ 1,000,000 | ||||||||
Milestone payments | $ 16,000,000 | |||||||||
Expenses related to license | 0 | |||||||||
Lund University Rights Holders Agreement [Member] | ||||||||||
License Agreement [Line Items] | ||||||||||
Milestone payments | $ 550 | |||||||||
Expenses related to license | $ 0 | $ 0 |
Income Taxes - Additional Infor
Income Taxes - Additional Information (Detail) - USD ($) | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Deferred income tax expense (benefit) | $ 0 | $ 0 |
Deferred tax assets change in valuation allowance | 19,968,000 | 11,991,000 |
Deferred tax assets, operating loss carryforwards, domestic | 121,836,000 | 57,279,000 |
Deferred tax assets, operating loss carryforwards, not subject to expiration | 104,093,000 | |
Deferred tax assets, operating loss carryforwards, foreign | 2,440,000 | $ 1,201,000 |
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 | 2.10% | 1.10% |
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] | ||
Research and development tax credits carryforwards | $ 1,547,000 | $ 339,000 |
Australia [Member] | ||
Income tax reconciliation tax credit research | $ 900,000 | |
Australia [Member] | Maximum [Member] | ||
Research and development tax credits | 43.50% | |
Orphan Drug Credit [Member] | U.S. federal [Member] | ||
Research and development tax credits carryforwards | $ 530,000 | |
Expiration Through 2037 [Member] | ||
Deferred tax assets, operating loss carryforwards, subject to expiration | 17,743,000 | |
Expiration Through 2039 [Member] | ||
Deferred tax assets, operating loss carryforwards, state | 115,129,000 | 54,212,000 |
Expiration Through 2033 [Member] | State [Member] | ||
Research and development tax credits carryforwards | $ 214,000 | $ 146,000 |
Income Taxes - Schedule of Reco
Income Taxes - Schedule of Reconciliation of Income Tax Expense (Benefit) (Detail) | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Income Tax Disclosure [Abstract] | ||
Federal income tax expense at statutory rate | 21.00% | 21.00% |
State income taxes, net of federal benefit | 5.20% | 5.20% |
Permanent differences | (0.90%) | (0.80%) |
U.S.—TCJA | 0.00% | 0.00% |
Foreign rate differential | 0.20% | 0.20% |
Research and development tax credits | 2.10% | 1.10% |
Change in valuation allowance | (27.60%) | (26.50%) |
Provision to Return | 0.00% | (0.20%) |
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, 2019 | Dec. 31, 2018 |
Deferred tax assets: | ||
U.S., foreign and state net operating loss carryforwards | $ 33,594 | $ 15,815 |
Research and development credits | 1,716 | 454 |
Capitalized start up and organizational costs | 32 | 35 |
Equity based compensation | 543 | 185 |
Licensing agreements | 1,756 | 1,258 |
Accruals and other | 993 | 948 |
Total deferred tax assets | 38,634 | 18,695 |
Valuation allowance | (38,425) | (18,457) |
Net deferred tax assets | 209 | 238 |
Deferred tax liabilities: | ||
Property and equipment | (209) | (238) |
Total deferred tax liabilities | $ 0 | $ 0 |
Net Loss per Share Attributab_3
Net Loss per Share Attributable to Common Stockholders - Schedule of Dilutive Common Stock Equivalents Excluded from Computation of Diluted Net Loss per Share (Detail) - shares | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Employee Stock Option [Member] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Antidilutive securities excluded from computation of earnings per share amount | 3,414,445 | 2,164,101 |
Restricted Stock [Member] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Antidilutive securities excluded from computation of earnings per share amount | 32,552 | 153,276 |
Commitments and Contingencies -
Commitments and Contingencies - Additional Information (Detail) - USD ($) $ in Thousands | Jan. 12, 2018 | Aug. 31, 2018 | Aug. 31, 2017 | Dec. 31, 2019 | Dec. 31, 2018 |
Commitments And Contingencies Disclosure [Line Items] | |||||
Lease agreement expiration date | Jan. 31, 2023 | ||||
Percentage of annual increase in rent | 3.00% | ||||
Tenant incentive allowance | $ 842 | ||||
Security deposit in connection with lease | $ 209 | $ 209 | |||
Rent expense | 2,257 | 1,295 | |||
Sub-lease Agreement [Member] | |||||
Commitments And Contingencies Disclosure [Line Items] | |||||
Lease agreement expiration date | Oct. 31, 2020 | Aug. 31, 2020 | |||
Percentage of annual increase in rent | 3.00% | 3.00% | |||
Security deposit in connection with lease | $ 283 | $ 283 |
Commitments and Contingencies_2
Commitments and Contingencies - Schedule Of Future Minimum Rental Payment Under Operating Lease (Detail) $ in Thousands | Dec. 31, 2019USD ($) |
Commitments And Contingencies Disclosure [Abstract] | |
2020 | $ 1,563 |
2021 | 705 |
2022 | 726 |
2023 | 61 |
Total | $ 3,055 |
Related Party Transactions - Ad
Related Party Transactions - Additional information (Detail) - USD ($) | Jan. 27, 2016 | Jul. 31, 2018 | Dec. 31, 2019 | Dec. 31, 2018 |
Related Party Transaction [Line Items] | ||||
Common stock issued for initial consideration for license | 2,300 | |||
Research and development expense | $ 54,974,000 | $ 35,095,000 | ||
Officers and Board Members [Member] | Consulting Services Agreement [Member] | ||||
Related Party Transaction [Line Items] | ||||
Expense related to related party | 0 | 40,000 | ||
License Agreement [Member] | ||||
Related Party Transaction [Line Items] | ||||
Provision for maximum cash payment | $ 2,000,000 | $ 2,000,000 | ||
Research and development expense | 821,000 | 56,000 | ||
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 | $ 1,451,000 | $ 348,000 |
Benefit Plans - Additional Info
Benefit Plans - Additional Information (Detail) - USD ($) $ in Thousands | 1 Months Ended | 12 Months Ended | |
Aug. 31, 2018 | Dec. 31, 2019 | Dec. 31, 2018 | |
Defined Benefit Plans [Abstract] | |||
Employer matching contribution, percent of match | 3.00% | ||
Employer contributions to defined contribution savings plans | $ 224 | $ 35 |
Subsequent Events - Additional
Subsequent Events - Additional Information (Detail) - USD ($) | 1 Months Ended | 12 Months Ended | ||
Feb. 29, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | Jan. 14, 2020 | |
Subsequent Event [Line Items] | ||||
Net proceeds from issuance of common stock | $ 129,465,000 | $ 104,013,000 | ||
Subsequent Event [Member] | ||||
Subsequent Event [Line Items] | ||||
Shelf registration maximum equity offering price | $ 250,000,000 | |||
Subsequent Event [Member] | Underwritten Public Offering [Member] | ||||
Subsequent Event [Line Items] | ||||
Net proceeds from issuance of common stock | $ 93,547,000 | |||
Subsequent Event [Member] | Common Stock [Member] | Underwritten Public Offering [Member] | ||||
Subsequent Event [Line Items] | ||||
Common stock, shares issued and sold | 4,350,000 | |||
Issuance price per shares | $ 23 |