Document and Entity Information
Document and Entity Information - shares | 3 Months Ended | |
Mar. 31, 2021 | May 06, 2021 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Mar. 31, 2021 | |
Document Fiscal Year Focus | 2021 | |
Document Fiscal Period Focus | Q1 | |
Trading Symbol | AVRO | |
Entity Registrant Name | AVROBIO, INC. | |
Entity Central Index Key | 0001681087 | |
Current Fiscal Year End Date | --12-31 | |
Entity Filer Category | Non-accelerated Filer | |
Entity Common Stock, Shares Outstanding | 41,766,122 | |
Entity Emerging Growth Company | true | |
Entity Ex Transition Period | false | |
Entity Small Business | true | |
Entity Current Reporting Status | Yes | |
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 Quarterly 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, $0.0001 par value per share | |
Security Exchange Name | NASDAQ |
Condensed Consolidated Balance
Condensed Consolidated Balance Sheets - USD ($) $ in Thousands | Mar. 31, 2021 | Dec. 31, 2020 |
Current assets: | ||
Cash and cash equivalents | $ 233,027 | $ 259,682 |
Prepaid expenses and other current assets | 9,037 | 7,560 |
Total current assets | 242,064 | 267,242 |
Property and equipment, net | 2,930 | 3,064 |
Restricted cash | 492 | 492 |
Other assets | 426 | 436 |
Total assets | 245,912 | 271,234 |
Current liabilities: | ||
Accounts payable | 2,698 | 2,682 |
Accrued expenses and other current liabilities | 9,822 | 13,693 |
Deferred rent | 248 | 239 |
Total current liabilities | 12,768 | 16,614 |
Deferred rent, net of current portion | 214 | 276 |
Total liabilities | 12,982 | 16,890 |
Commitments and contingencies (Note 6) | ||
Stockholders’ equity: | ||
Preferred stock, $0.0001 par value; 10,000 shares authorized; no shares issued or outstanding | ||
Common stock, $0.0001 par value; 150,000 shares authorized; 41,766 and 41,569 shares issued and outstanding as of March 31, 2021 and December 31, 2020, respectively | 4 | 4 |
Additional paid-in capital | 524,241 | 518,756 |
Accumulated deficit | (291,315) | (264,416) |
Total stockholders’ equity | 232,930 | 254,344 |
Total liabilities and stockholders’ equity | $ 245,912 | $ 271,234 |
Condensed Consolidated Balanc_2
Condensed Consolidated Balance Sheets (Parenthetical) - $ / shares | Mar. 31, 2021 | Dec. 31, 2020 |
Statement Of Financial Position [Abstract] | ||
Preferred stock, par value | $ 0.0001 | $ 0.0001 |
Preferred stock, authorized | 10,000,000 | 10,000,000 |
Preferred stock, issued | 0 | 0 |
Preferred stock, outstanding | 0 | 0 |
Common stock, par value | $ 0.0001 | $ 0.0001 |
Common stock, authorized | 150,000,000 | 150,000,000 |
Common stock, issued | 41,766,000 | 41,569,000 |
Common stock, outstanding | 41,766,000 | 41,569,000 |
Condensed Consolidated Statemen
Condensed Consolidated Statements of Operations and Comprehensive Loss - USD ($) shares in Thousands, $ in Thousands | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Operating expenses: | ||
Research and development | $ 18,527 | $ 18,274 |
General and administrative | 8,357 | 8,315 |
Total operating expenses | 26,884 | 26,589 |
Loss from operations | (26,884) | (26,589) |
Other (expense) income: | ||
Interest income | 10 | 632 |
Other expense | (25) | (16) |
Total other (expense) income, net | (15) | 616 |
Net loss | (26,899) | (25,973) |
Comprehensive loss | $ (26,899) | $ (25,973) |
Net loss per share — basic and diluted | $ (0.65) | $ (0.77) |
Weighted-average number of common shares outstanding — basic and diluted | 41,618 | 33,667 |
Condensed Consolidated Statem_2
Condensed Consolidated Statements of Stockholders' Equity - USD ($) $ in Thousands | Total | Common Stock [Member] | Additional Paid-in Capital [Member] | Accumulated Deficit [Member] |
Beginning balance at Dec. 31, 2019 | $ 186,013 | $ 3 | $ 330,714 | $ (144,704) |
Beginning balance, shares at Dec. 31, 2019 | 31,643,000 | |||
Stock-based compensation expense | 2,822 | 2,822 | ||
Exercise of stock options | 35 | 35 | ||
Exercise of stock options, shares | 7,000 | |||
Vesting of restricted stock awards and units, shares | 11,000 | |||
Issuance of common stock, net of offering costs | 93,628 | $ 1 | 93,627 | |
Issuance of common stock, net of offering costs, shares | 4,350,000 | |||
Net loss | (25,973) | (25,973) | ||
Ending balance at Mar. 31, 2020 | 256,525 | $ 4 | 427,198 | (170,677) |
Ending balance, shares at Mar. 31, 2020 | 36,011,000 | |||
Beginning balance at Dec. 31, 2020 | 254,344 | $ 4 | 518,756 | (264,416) |
Beginning balance, shares at Dec. 31, 2020 | 41,569,000 | |||
Stock-based compensation expense | 4,635 | 4,635 | ||
Exercise of stock options | $ 850 | 850 | ||
Exercise of stock options, shares | 196,872 | 197,000 | ||
Net loss | $ (26,899) | (26,899) | ||
Ending balance at Mar. 31, 2021 | $ 232,930 | $ 4 | $ 524,241 | $ (291,315) |
Ending balance, shares at Mar. 31, 2021 | 41,766,000 |
Condensed Consolidated Statem_3
Condensed Consolidated Statements of Stockholders' Equity (Parenthetical) $ in Thousands | 3 Months Ended |
Mar. 31, 2020USD ($) | |
Statement Of Stockholders Equity [Abstract] | |
Issuance of common stock upon public offering, offering costs | $ 419 |
Condensed Consolidated Statem_4
Condensed Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Cash flows from operating activities: | ||
Net loss | $ (26,899) | $ (25,973) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Depreciation and amortization expense | 291 | 283 |
Stock-based compensation expense | 4,635 | 2,822 |
Deferred rent expense | (52) | (53) |
Changes in operating assets and liabilities: | ||
Prepaid expenses and other current assets | (1,477) | 976 |
Other assets | 10 | 319 |
Accounts payable | (26) | (1,443) |
Accrued expenses and other current liabilities | (3,821) | 635 |
Net cash used in operating activities | (27,339) | (22,434) |
Cash flows from investing activities: | ||
Purchases of property and equipment | (115) | (577) |
Net cash used in investing activities | (115) | (577) |
Cash flows from financing activities: | ||
Proceeds from exercise of stock options | 850 | 35 |
Proceeds from issuance of common shares upon completion of public offering, net of offering costs | 93,628 | |
Other financing activities | (51) | |
Net cash provided by financing activities | 799 | 93,663 |
Net (decrease) increase in cash, cash equivalents and restricted cash | (26,655) | 70,652 |
Cash, cash equivalents and restricted cash at beginning of period | 260,174 | 187,535 |
Cash, cash equivalents and restricted cash at end of period | 233,519 | 258,187 |
Supplemental disclosure of non-cash investing and financing activities: | ||
Purchases of property and equipment included in accounts payable and accrued expenses | 42 | |
Common stock offering costs incurred but unpaid at period end | 249 | |
Reconciliation of cash, cash equivalents and restricted cash reported within the condensed consolidated balance sheets: | ||
Cash and cash equivalents | 233,027 | 257,695 |
Restricted cash | 492 | 492 |
Cash, cash equivalents and restricted cash at end of period | $ 233,519 | $ 258,187 |
Nature of the Business
Nature of the Business | 3 Months Ended |
Mar. 31, 2021 | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | |
Nature of the Business | 1. Nature of the Business AVROBIO, Inc. (the “Company” or “AVROBIO”) is a clinical-stage gene therapy company focused on developing potentially curative ex vivo The Company is subject to risks and uncertainties common to clinical-stage companies in the biotechnology industry, including but not limited to, risks associated with completing preclinical studies and clinical trials, receiving regulatory approvals for product candidates, development by competitors of new biopharmaceutical products, dependence on key personnel, protection of proprietary technology, compliance with government regulations and the ability to secure additional capital to fund operations. Product candidates currently under development will require significant additional research and development efforts, including preclinical and clinical testing and regulatory approval, prior to commercialization. These efforts require significant amounts of additional capital, adequate personnel and infrastructure and extensive compliance-reporting capabilities. Even if the Company’s product development efforts are successful, it is uncertain when, if ever, the Company will realize revenue from product sales. The Company has incurred recurring losses since its inception, including net losses of $26,899 and $25,973 for the three months ended March 31, 2021 and 2020, respectively. In addition, as of March 31, 2021, the Company had an accumulated deficit of $291,315. The Company has primarily funded these losses through the proceeds from sales of common and preferred stock. 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 March 31, 2021 of $233,027 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 Quarterly Report on Form 10-Q 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 | 3 Months Ended |
Mar. 31, 2021 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies Basis of Presentation The accompanying condensed consolidated financial statements (the “unaudited condensed 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”). The unaudited condensed consolidated financial statements have been prepared on the same basis as the audited annual consolidated financial statements as of and for the year ended December 31, 2020, and, in the opinion of management, reflect all adjustments, consisting of normal recurring adjustments, necessary for the fair presentation of the Company’s financial position as of March 31, 2021, and the results of its operations for the three months ended March 31, 2021 and 2020, its statements of stockholders’ equity for the three months ended March 31, 2021 and 2020 and its statement of cash flows for the three months ended March 31, 2021 and 2020. The results for the three months ended March 31, 2021 are not necessarily indicative of the results to be expected for the year ending December 31, 2021, any other interim periods, or any future year or period. These interim financial statements should be read in conjunction with the audited financial statements as of and for the year ended December 31, 2020, and the notes thereto, which are included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2020, filed with the SEC on March 18, 2021. The unaudited condensed consolidated financial statements reflect the application of certain significant accounting policies as described below and elsewhere in these notes to the unaudited condensed consolidated financial statements. As of March 31, 2021, there have been no changes to the Company’s significant accounting policies as described in the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2020. Segment Information Operating segments are identified as components of an enterprise about which separate discrete financial information is available for evaluation by the chief operating decision maker, or decision-making group, in making decisions on how to allocate resources and assess performance. The Company’s chief operating decision maker is the chief executive officer (“CEO”). The Company and the CEO view the Company’s operations and manage its business as one operating segment. All material long-lived assets of the Company reside in the United States. Use of Estimates The preparation of the unaudited condensed consolidated financial statements in conformity with GAAP requires that the Company make estimates and judgments that may affect the reported amounts of assets, liabilities and expenses and the related disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of expenses during the reporting periods. On an ongoing basis, the Company evaluates its estimates, judgments and methodologies. The Company bases its estimates on historical experience and on various other assumptions that are believed to be reasonable, the results of which form the basis for making judgments about the carrying values of assets and liabilities. Actual results may differ from these estimates. Changes in estimates are reflected in reported results in the period in which they become known. Significant estimates relied upon in preparing the unaudited condensed consolidated financial statements include the determination of the fair value of share-based awards issued and the estimation of accrued research and development expenses. Stock-based Compensation For stock-based awards issued to employees and members of the Company’s board of directors (the “Board”) for their services on the Board, the Company measures the estimated fair value of the stock-based award on the date of grant and recognizes compensation expense for those awards over the requisite service period, which is generally the vesting period of the respective award. The Company issues stock-based awards with only service-based vesting conditions and records the expense for these awards using the straight-line method. The Company has not issued any stock-based awards with performance- or market-based vesting conditions. The Company accounts for forfeitures as they occur. Prior to the adoption of Accounting Standards Update (“ASU”) No. 2018-07, Compensation-Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting The Company classifies stock-based compensation expense in its consolidated statements of operations and comprehensive loss in the same manner in which the award recipient’s cash compensation costs are classified. Given the absence of an active market for the Company’s common stock prior to its initial public offering (“IPO”), the Company and the Board, the members of which the Company believes have extensive business, finance, and venture capital experience, were required to estimate the fair value of the Company’s common stock at the time of each grant of a stock-based award. The Company and the Board determined the estimated fair value of the Company’s equity instruments based on a number of factors, including external market conditions affecting the biotechnology industry sector. The Company and the Board utilized various valuation methodologies in accordance with the framework of the American Institute of Certified Public Accountants’ Technical Practice Aid, Valuation of Privately-Held Company Equity Securities Issued as Compensation likelihood of achieving a liquidity event for the common stock underlying the stock-based awards, such as an IPO or sale of the Company, given prevailing market conditions. 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, 2020, the Company determined the volatility for awards granted up through December 31, 2020 based on an analysis of reported data for a group of guideline companies that issued options with substantially similar terms. Beginning with options granted in 2021, the Company estimates its expected stock volatility using a weighted-average calculation based on the historical volatility of the Company and publicly traded peer companies and expects to continue to do so until such time as it has adequate historical data regarding the volatility of its own traded stock price. peer group of companies and began utilizing the Company’s historical volatility in fiscal year 2021 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 its IPO or such earlier time that it is no longer an “emerging growth company”. 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. Recently Issued Accounting Pronouncements In February 2016, the FASB issued ASU No. 2016-02, (Topic 842) Leases, he Company currently expects that its operating lease commitments will be subject to the new standard and recognized as right-of-use assets and operating lease liabilities upon adoption of this standard, which will increase the Company’s total assets and total liabilities. In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments—Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments , or 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. ASU 2016-13 is effective for non-EGCs for fiscal years beginning December 15, 2019 and interim periods within those fiscal years, and will be effective for the Company for fiscal years beginning after December 15, 2022 and interim periods within those fiscal years, assuming the Company remains an EGC. Early adoption is permitted. The Company is currently evaluating the effects the adoption of ASU 2016-13 may have on its financial statements. In November 2019, the FASB issued ASU 2019-11, “ Codification Improvements to Topic 326, Financial Instruments – Credit Losses ”, or ASU 2019-11. ASU 2019-11 is an accounting pronouncement that amends ASU 2016-13, “ Financial Instruments – Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments .” The amendments update guidance on reporting credit losses for financial assets. These amendments affect loans, debt securities, trade receivables, net investments in leases, off balance sheet credit exposures, reinsurance receivables, and any other financial assets not excluded from the scope that have the contractual right to receive cash. The amendments in both ASU 2016-13 and ASU 2019-11 are effective for annual reporting periods beginning after December 15, 2019, including interim periods within those fiscal years. As a result of the Company having elected the extended transition period for complying with new or revised accounting standards pursuant to Section 107(b) of the JOBS Act, 2016-13 and ASU 2019-11 are effective for the Company for fiscal years beginning after December 15, 2022, and interim periods within those fiscal years. The Company is currently evaluating ASU 2016-13 and ASU 2019-11 and their impact on its consolidated financial statements and financial statement disclosures. In December 2019, the FASB issued ASU No. 2019-12, Simplifying the Accounting for Income Taxes , or ASU 2019-12. ASU 2019-12 eliminates certain exceptions related to the approach for intraperiod tax allocation, the methodology for calculating income taxes in an interim period and the recognition of deferred tax liabilities for outside basis differences. It also clarifies and simplifies other aspects of the accounting for income taxes. ASU 2019-12 is effective for non-EGCs for fiscal years beginning after December 15, 2020 and interim periods within those fiscal years and will be effective for the Company for fiscal years beginning after December 15, 2021 and interim periods beginning after December 15, 2022, assuming the Company remains an EGC. Early adoption is permitted. The Company is currently evaluating the effects the adoption of ASU 2019-12 may have on its financial statements. |
License Agreements
License Agreements | 3 Months Ended |
Mar. 31, 2021 | |
License Agreements [Abstract] | |
License Agreements | 3. License Agreements Agreement with The University of Manchester On September 30, 2020, the Company entered into an agreement (“MPSII License Agreement”) with The University of Manchester, England (“UoM”), whereby UoM granted to the Company an exclusive worldwide license under certain patent and other intellectual property rights, subject to certain retained rights, to develop, commercialize and sell an ex vivo lentiviral gene therapy for use in the treatment of Hunter syndrome, or mucopolysaccharidosis type II (“MPSII”) As part of the agreement, the Company is obligated to make milestone payments of up to an aggregate of $80,000 upon the achievement of specified development and regulatory milestones, to pay royalties, on a product-by-product and country-by-country basis, of a mid-single digit percentage based on net sales of products licensed under the agreement and to pay a low double digit percentage of any sublicense fees received by the Company. The next anticipated payment milestones under the MPSII License Agreement include $2,000, which would become due following the date of regulatory approval of the clinical trial application for the investigator-sponsored Phase 1/2 clinical trial sponsored by UoM, and $4,000, upon the dosing of the first patient in the investigator-sponsored Phase 1/2 clinical trial sponsored by UoM. Unless terminated earlier, the agreement expires upon the later of 15 years from the effective date or the expiration of the last valid claim of the licensed patents, subject to certain surviving rights and obligations. Concurrently with the MPSII License Agreement, the Company entered into a collaborative research funding agreement with UoM (“CRFA”). Under the CRFA, the Company has agreed to fund the budgeted costs of an investigator-sponsored Phase 1/2 clinical trial to be sponsored by UoM in connection with the development activities under the MPSII License Agreement, which are currently estimated to equal approximately £9,100 in the aggregate For the three months ended March 31, 2021 and March 31, 2020, the Company incurred no expenses related to the CRFA. Agreements with University Health Network (“UHN”) Fabry License Agreement— On January 27, 2016, the Company entered into an agreement with UHN, pursuant to which UHN granted the Company an option to enter into an exclusive license under the UHN intellectual property related to Fabry disease in accordance with the pre-negotiated licensing terms. On November 4, 2016, the Company exercised its option and entered into a license agreement with UHN, pursuant to which UHN granted the Company an exclusive worldwide license under certain intellectual property rights and a non-exclusive worldwide license under certain know-how, in each case subject to certain retained rights, to develop, commercialize and sell products for use in the treatment of Fabry disease. In addition, for three years following the execution of the agreement, UHN granted the Company an exclusive option to obtain a license under certain improvements to the licensed intellectual property rights as well as an option to negotiate a license under certain other improvements. Under this agreement, the Company paid an option fee of CAD $20, an upfront license fee of CAD $75, plus the annual license maintenance fee for the first year. Thereafter, the Company is also required to pay UHN future annual license maintenance fees until the first sale of a licensed product in certain markets. The Company is also obligated to make future milestone payments in an aggregate amount of up to CAD $2,450 upon the achievement of specified milestones as well as royalties on a country-by-country basis of a low to mid-single-digit percentage of annual net sales of licensed products and a lower single-digit royalty percentage in certain circumstances. Additionally, the Company has agreed to pay a low double-digit royalty percentage of all sublicensing revenue. The agreement requires the Company to meet certain performance milestones within specified timeframes. UHN may terminate the agreement if the Company fails to meet these performance milestones despite using commercially reasonable efforts and the Company is unable to reach agreement with UHN on revised timeframes. The Company’s royalty obligation expires on a licensed product-by-licensed product and country-by-country basis upon the latest to occur of the expiration or termination of the last valid claim under the licensed intellectual property rights in such country, the tenth anniversary of the first commercial sale of such licensed product in such country and the expiration of any applicable regulatory exclusivity in such country. Unless terminated earlier, the agreement expires upon the expiration of the Company’s royalty obligation for all licensed products. UHN can terminate the agreement if the Company fails to make any payments within a specified period after receiving written notice of such failure, or in the event that the Company fails to obtain or maintain insurance. Either the Company or UHN may terminate the license agreement in the event of a material breach by the other party and failure to cure such breach within a certain period of time. The Company can voluntarily terminate the agreement with prior notice to UHN. For the three months ended March 31, 2021 and 2020, the Company recorded research and development expense related to this agreement with UHN of $44 and $0, respectively, which consists of reimbursable funded study trial costs. No milestone or maintenance fees were incurred related to this agreement in the three months ended March 31, 2021 and 2020. Interleukin 12 License Agreement— On January 27, 2016, the Company entered into an exclusive license agreement with UHN, pursuant to which UHN granted the Company a license to certain patent rights for the commercial development, manufacture, distribution and use of any products or processes resulting from development of those patent rights related to Interleukin 12. Upon execution of this agreement, the Company paid an upfront license fee of CAD $264. In addition, as part of the initial consideration for the license, the Company issued to UHN 1,161,665 shares of the Company’s common stock and agreed to pay UHN up to $2,000 upon the closing of an IPO if certain criteria are met. The fair value of the shares issued to UHN of $480 and the upfront fee was expensed upon the execution of the agreement. Upon the closing of the IPO in 2018, as the criteria were met, the Company paid UHN $2,000. 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 three months ended March 31, 2021 and 2020, the Company recorded research and development expense related to this agreement with UHN of $39 and $35, respectively, which consists of license maintenance fees. No milestone fees were incurred related to this agreement in the three months ended March 31, 2021 and 2020. Agreement with BioMarin Pharmaceutical Inc. (“BioMarin”) On August 31, 2017, the Company entered into a license agreement with BioMarin, pursuant to which BioMarin granted the Company an exclusive worldwide license under certain intellectual property rights owned or controlled by BioMarin to develop, commercialize and sell products for use in the treatment of Pompe disease. The license agreement was amended in February 2018 and again in January 2020 to, among things, provide that BioMarin would supply the Company with certain technology materials. As consideration for this agreement, the Company paid an upfront license fee of $500 in cash and issued 233,765 shares of Series B Preferred Stock to BioMarin at the time of the Company’s Series B Preferred Stock financing in January 2018. The Company has 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 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 has recognized no expenses related to the license for the three months ended March 31, 2021 and 2020. Unless terminated earlier, the agreement expires upon the expiration of the Company’s royalty obligation for all licensed products throughout the world. BioMarin and the Company can terminate the agreement in the event of a material breach by the other party and failure to cure such breach within a certain period of time. The Company may terminate the agreement at will upon written notice to BioMarin. BioMarin has the right to terminate the agreement upon the Company’s bankruptcy or insolvency, or in the event of any challenge or opposition to the licensed patent rights or related actions brought by the Company or its affiliates or sublicensees, or if the Company, its affiliates or sublicensees knowingly assist a third-party in challenging or otherwise opposing the licensed patent rights, except as required under a court order or subpoena. Agreement with 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 has recognized no research and development expenses related to milestone payments for the three months ended March 31, 2021 and 2020. Agreement with Lund University Rights Holders On November 17, 2016, the Company entered into a license agreement with affiliates of Lund University, along with certain other relevant rights holders that may be added from time to time, pursuant to which such rights holders granted to the Company an exclusive worldwide license, subject to certain retained rights, under certain intellectual property rights to develop, commercialize and sell products in any and all uses relevant to Gaucher disease. As consideration for the license, the Company is required to make payments in connection with the achievement of certain milestones up to an aggregate of $550. The agreement expires on the latest of (i) the twentieth anniversary of the end of a certain research project the Company is funding pursuant to an agreement with Lund University, (ii) the expiration of the term of any patent filed on the licensed rights that covers a licensed product, (iii) the expiration of any applicable marketing exclusivity right and (iv) such time that neither the Company nor any sublicensees, partners or contractors are commercializing a licensed product. Either the Company or the rights holders acting together may terminate the license agreement if the other such party commits a material breach and fails to cure such breach within a certain period of time, or if the other party enters into liquidation, becomes insolvent, or enters into composition or statutory reorganization proceedings. The Company has recognized no expenses related to this agreement for the three months ended March 31, 2021 and 2020. |
Fair Value Measurement
Fair Value Measurement | 3 Months Ended |
Mar. 31, 2021 | |
Fair Value Disclosures [Abstract] | |
Fair Value Measurement | 4. Fair Value Measurement The following table presents information about the Company’s financial assets 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 March 31, 2021 and December 31, 2020: Fair Value Measurements as of March 31, 2021 Level 1 Level 2 Level 3 Total Assets: Cash equivalents — $ 231,651 $ — $ — $ 231,651 $ 231,651 $ — $ — $ 231,651 Fair Value Measurements as of December 31, 2020 Level 1 Level 2 Level 3 Total Assets: Cash equivalents — money market funds $ 44,416 $ — $ — $ 44,416 $ 44,416 $ — $ — $ 44,416 The fair value of cash equivalents was determined through quoted prices by third-party pricing services. During the three months ended March 31, 2021, there were no transfers between levels. |
Supplemental Balance Sheet Info
Supplemental Balance Sheet Information | 3 Months Ended |
Mar. 31, 2021 | |
Balance Sheet Related Disclosures [Abstract] | |
Supplemental Balance Sheet Information | 5. Supplemental Balance Sheet Information Prepaid expenses and other current assets Prepaid expenses and other current assets consisted of the following: March 31, 2021 December 31, 2020 Tax incentive refund $ 2,739 $ 2,558 Prepaid research and development expenses 4,297 3,145 Prepaid insurance 692 973 Prepaid compensation benefits 922 609 Other current assets 387 275 $ 9,037 $ 7,560 Property and equipment, net Property and equipment, net consisted of the following: March 31, 2021 December 31, 2020 Laboratory and office equipment $ 4,105 $ 3,965 Leasehold improvements 1,394 1,377 Computer equipment 143 143 5,642 5,485 Less: Accumulated depreciation and amortization (2,712 ) (2,421 ) Property and equipment, net $ 2,930 $ 3,064 Depreciation and amortization expense was $291 and $283 for the three months ended March 31, 2021 and 2020. Restricted Cash As of March 31, 2021 and December 31, 2020, the Company had restricted cash as presented in the table below, which consists of cash used to secure letters of credit for the benefit of the landlord in connection with the Company’s lease agreements. The cash will be restricted until the termination or modification of the lease arrangement. March 31, 2021 December 31, 2020 Restricted cash $ 492 $ 492 Accrued Expenses Accrued expenses and other current liabilities consisted of the following: March 31, 2021 December 31, 2020 Compensation and benefit costs $ 3,460 $ 6,402 Research and development expenses 5,151 5,847 Consulting and professional fees 942 1,096 Other liabilities 269 348 $ 9,822 $ 13,693 |
Commitments and Contingencies
Commitments and Contingencies | 3 Months Ended |
Mar. 31, 2021 | |
Commitments And Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 6. Commitments and Contingencies 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 three months ended March 31, 2021 and 2020 and to the best of the Company’s 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 March 31, 2021 and December 31, 2020, or royalties on future sales. No milestone or royalty payments under these agreements are expected to be payable in the immediate future, except as disclosed in Note 3 “License Agreements.” The Company enters into standard indemnification agreements in the ordinary course of business. Pursuant to the agreements, the Company agrees to indemnify, hold harmless, and to reimburse the indemnified party for losses suffered or incurred by the indemnified party, generally the Company’s business partners, in connection with any U.S. patent or any copyright or other intellectual property infringement claim by any third-party with respect to the Company’s products. Further, the Company indemnifies its directors and officers who are, or were, serving at the Company’s request in such capacities. The Company’s maximum exposure under these arrangements is unknown as of March 31, 2021. The Company does not anticipate recognizing any significant losses relating to these arrangements. 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. |
Stockholders' Equity
Stockholders' Equity | 3 Months Ended |
Mar. 31, 2021 | |
Equity [Abstract] | |
Stockholders' Equity | 7. Stockholders’ Equity C ommon Stock As of March 31, 2021 and December 31, 2020, the authorized capital stock of the Company included 150,000,000 shares of common stock, $0.0001 par value and 10,000,000 shares of undesignated preferred stock. As of March 31, 2021 and December 31, 2020, no undesignated preferred stock was outstanding. As of March 31, 2021, no cash dividends have been declared or paid. Common Stock Reserved for Future Issuance As of March 31, 2021 and December 31, 2020, the Company has reserved the following shares of common stock for future issuance: March 31, 2021 December 31, 2020 Shares reserved for exercise of outstanding stock options 7,663,046 5,559,545 Shares reserved for vesting of restricted stock units 1,055 1,198 Shares reserved for issuance under the 2018 Stock Option and Grant Plan 3,433,617 3,877,478 Shares reserved for issuance under the 2018 Employee Stock Purchase Plan 1,178,590 762,900 Shares reserved for issuance under the 2019 Inducement Plan 171,300 365,050 Shares reserved for issuance under the 2020 Inducement Plan 1,700,000 1,700,000 Total shares of authorized common stock reserved for future issuance 14,147,608 12,266,171 |
Stock-Based Compensation
Stock-Based Compensation | 3 Months Ended |
Mar. 31, 2021 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Stock-Based Compensation | 8. Stock-based Compensation 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 of directors were as follows, presented on a weighted-average basis: Three Months Ended March 31, 2021 2020 Expected option life (years) 6.00 6.00 Risk-free interest rate 0.63 % 0.96 % Expected volatility 80.67 % 75.49 % Expected dividend yield — % — % The following table summarizes the Company’s stock option activity for the three months ended March 31, 2021: Number of Options Weighted- Average Exercise Price Weighted- Average Remaining Contractual Term (Years) Aggregate Intrinsic Value Outstanding as of December 31, 2020 5,559,545 $ 14.97 7.65 $ 13,511 Granted 2,359,416 $ 15.66 Exercised (196,872 ) $ 4.32 Cancelled or forfeited (59,043 ) $ 17.88 Outstanding as of March 31, 2021 7,663,046 $ 15.45 8.36 $ 9,922 Exercisable as of March 31, 2021 2,254,165 $ 12.75 6.68 $ 8,626 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 three months ended March 31, 2021 and 2020 was $1,729 and $133, respectively. The weighted-average grant-date fair value of the Company’s stock options granted during the three months ended March 31, 2021 and 2020 was $10.69 and $13.68, respectively. Restricted Stock Units The following table summarizes the Company’s restricted common stock units for the three months ended March 31, 2021: Number of Shares Weighted- Average Grant Date Fair Value Issued and unvested as of December 31, 2020 1,198 $ 15.65 Granted — Vested (143 ) $ 15.65 Forfeited, cancelled or expired — Issued and unvested as of March 31, 2021 1,055 $ 15.65 The total fair value of restricted stock units vested during the three months ended March 31, 2021 and 2020 was $2 and $15, respectively. Stock-Based Compensation Stock-based compensation expense was allocated as follows: Three Months Ended March 31, 2021 2020 Research and development $ 2,115 $ 1,443 General and administrative 2,520 1,379 Total stock-based compensation expense $ 4,635 $ 2,822 As of March 31, 2021, total unrecognized compensation cost related to the unvested stock-based awards was $56,945, which is expected to be recognized over a weighted-average period of 3.23 years. |
Net Loss Per Share
Net Loss Per Share | 3 Months Ended |
Mar. 31, 2021 | |
Earnings Per Share [Abstract] | |
Net Loss Per Share | 9. Net Loss Per Share 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 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 for the periods indicated: Three Months Ended March 31, 2021 2020 Options to purchase common stock 7,663,046 5,310,482 Restricted common stock 1,055 21,798 |
Related Party Transactions
Related Party Transactions | 3 Months Ended |
Mar. 31, 2021 | |
Related Party Transactions [Abstract] | |
Related Party Transactions | 10. Related Party Transactions UHN For the three months ended March 31, 2021 and 2020, the Company recognized $39 and $35, respectively, of research and development expense related to the license agreements with UHN. Refer to Note 3 “ License Agreements Others For the three months ended March 31, 2021 and 2020, the Company recorded expenses of $641 and $360, respectively, related to a sublease to rent lab space, provided by an entity affiliated with a member of the Company’s board of directors. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 3 Months Ended |
Mar. 31, 2021 | |
Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation The accompanying condensed consolidated financial statements (the “unaudited condensed 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”). The unaudited condensed consolidated financial statements have been prepared on the same basis as the audited annual consolidated financial statements as of and for the year ended December 31, 2020, and, in the opinion of management, reflect all adjustments, consisting of normal recurring adjustments, necessary for the fair presentation of the Company’s financial position as of March 31, 2021, and the results of its operations for the three months ended March 31, 2021 and 2020, its statements of stockholders’ equity for the three months ended March 31, 2021 and 2020 and its statement of cash flows for the three months ended March 31, 2021 and 2020. The results for the three months ended March 31, 2021 are not necessarily indicative of the results to be expected for the year ending December 31, 2021, any other interim periods, or any future year or period. These interim financial statements should be read in conjunction with the audited financial statements as of and for the year ended December 31, 2020, and the notes thereto, which are included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2020, filed with the SEC on March 18, 2021. The unaudited condensed consolidated financial statements reflect the application of certain significant accounting policies as described below and elsewhere in these notes to the unaudited condensed consolidated financial statements. As of March 31, 2021, there have been no changes to the Company’s significant accounting policies as described in the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2020. |
Segment Information | Segment Information Operating segments are identified as components of an enterprise about which separate discrete financial information is available for evaluation by the chief operating decision maker, or decision-making group, in making decisions on how to allocate resources and assess performance. The Company’s chief operating decision maker is the chief executive officer (“CEO”). The Company and the CEO view the Company’s operations and manage its business as one operating segment. All material long-lived assets of the Company reside in the United States. |
Use of Estimates | Use of Estimates The preparation of the unaudited condensed consolidated financial statements in conformity with GAAP requires that the Company make estimates and judgments that may affect the reported amounts of assets, liabilities and expenses and the related disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of expenses during the reporting periods. On an ongoing basis, the Company evaluates its estimates, judgments and methodologies. The Company bases its estimates on historical experience and on various other assumptions that are believed to be reasonable, the results of which form the basis for making judgments about the carrying values of assets and liabilities. Actual results may differ from these estimates. Changes in estimates are reflected in reported results in the period in which they become known. Significant estimates relied upon in preparing the unaudited condensed consolidated financial statements include the determination of the fair value of share-based awards issued and the estimation of accrued research and development expenses. |
Stock-Based Compensation | Stock-based Compensation For stock-based awards issued to employees and members of the Company’s board of directors (the “Board”) for their services on the Board, the Company measures the estimated fair value of the stock-based award on the date of grant and recognizes compensation expense for those awards over the requisite service period, which is generally the vesting period of the respective award. The Company issues stock-based awards with only service-based vesting conditions and records the expense for these awards using the straight-line method. The Company has not issued any stock-based awards with performance- or market-based vesting conditions. The Company accounts for forfeitures as they occur. Prior to the adoption of Accounting Standards Update (“ASU”) No. 2018-07, Compensation-Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting The Company classifies stock-based compensation expense in its consolidated statements of operations and comprehensive loss in the same manner in which the award recipient’s cash compensation costs are classified. Given the absence of an active market for the Company’s common stock prior to its initial public offering (“IPO”), the Company and the Board, the members of which the Company believes have extensive business, finance, and venture capital experience, were required to estimate the fair value of the Company’s common stock at the time of each grant of a stock-based award. The Company and the Board determined the estimated fair value of the Company’s equity instruments based on a number of factors, including external market conditions affecting the biotechnology industry sector. The Company and the Board utilized various valuation methodologies in accordance with the framework of the American Institute of Certified Public Accountants’ Technical Practice Aid, Valuation of Privately-Held Company Equity Securities Issued as Compensation likelihood of achieving a liquidity event for the common stock underlying the stock-based awards, such as an IPO or sale of the Company, given prevailing market conditions. 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, 2020, the Company determined the volatility for awards granted up through December 31, 2020 based on an analysis of reported data for a group of guideline companies that issued options with substantially similar terms. Beginning with options granted in 2021, the Company estimates its expected stock volatility using a weighted-average calculation based on the historical volatility of the Company and publicly traded peer companies and expects to continue to do so until such time as it has adequate historical data regarding the volatility of its own traded stock price. peer group of companies and began utilizing the Company’s historical volatility in fiscal year 2021 |
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 its IPO or such earlier time that it is no longer an “emerging growth company”. |
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. |
Recently Issued Accounting Pronouncements | Recently Issued Accounting Pronouncements In February 2016, the FASB issued ASU No. 2016-02, (Topic 842) Leases, he Company currently expects that its operating lease commitments will be subject to the new standard and recognized as right-of-use assets and operating lease liabilities upon adoption of this standard, which will increase the Company’s total assets and total liabilities. In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments—Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments , or 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. ASU 2016-13 is effective for non-EGCs for fiscal years beginning December 15, 2019 and interim periods within those fiscal years, and will be effective for the Company for fiscal years beginning after December 15, 2022 and interim periods within those fiscal years, assuming the Company remains an EGC. Early adoption is permitted. The Company is currently evaluating the effects the adoption of ASU 2016-13 may have on its financial statements. In November 2019, the FASB issued ASU 2019-11, “ Codification Improvements to Topic 326, Financial Instruments – Credit Losses ”, or ASU 2019-11. ASU 2019-11 is an accounting pronouncement that amends ASU 2016-13, “ Financial Instruments – Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments .” The amendments update guidance on reporting credit losses for financial assets. These amendments affect loans, debt securities, trade receivables, net investments in leases, off balance sheet credit exposures, reinsurance receivables, and any other financial assets not excluded from the scope that have the contractual right to receive cash. The amendments in both ASU 2016-13 and ASU 2019-11 are effective for annual reporting periods beginning after December 15, 2019, including interim periods within those fiscal years. As a result of the Company having elected the extended transition period for complying with new or revised accounting standards pursuant to Section 107(b) of the JOBS Act, 2016-13 and ASU 2019-11 are effective for the Company for fiscal years beginning after December 15, 2022, and interim periods within those fiscal years. The Company is currently evaluating ASU 2016-13 and ASU 2019-11 and their impact on its consolidated financial statements and financial statement disclosures. In December 2019, the FASB issued ASU No. 2019-12, Simplifying the Accounting for Income Taxes , or ASU 2019-12. ASU 2019-12 eliminates certain exceptions related to the approach for intraperiod tax allocation, the methodology for calculating income taxes in an interim period and the recognition of deferred tax liabilities for outside basis differences. It also clarifies and simplifies other aspects of the accounting for income taxes. ASU 2019-12 is effective for non-EGCs for fiscal years beginning after December 15, 2020 and interim periods within those fiscal years and will be effective for the Company for fiscal years beginning after December 15, 2021 and interim periods beginning after December 15, 2022, assuming the Company remains an EGC. Early adoption is permitted. The Company is currently evaluating the effects the adoption of ASU 2019-12 may have on its financial statements. |
Fair Value Measurement (Tables)
Fair Value Measurement (Tables) | 3 Months Ended |
Mar. 31, 2021 | |
Fair Value Disclosures [Abstract] | |
Summary of Financial Assets Measured at Fair Value on Recurring Basis | The following table presents information about the Company’s financial assets 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 March 31, 2021 and December 31, 2020: Fair Value Measurements as of March 31, 2021 Level 1 Level 2 Level 3 Total Assets: Cash equivalents — $ 231,651 $ — $ — $ 231,651 $ 231,651 $ — $ — $ 231,651 Fair Value Measurements as of December 31, 2020 Level 1 Level 2 Level 3 Total Assets: Cash equivalents — money market funds $ 44,416 $ — $ — $ 44,416 $ 44,416 $ — $ — $ 44,416 |
Supplemental Balance Sheet In_2
Supplemental Balance Sheet Information (Tables) | 3 Months Ended |
Mar. 31, 2021 | |
Balance Sheet Related Disclosures [Abstract] | |
Summary of Prepaid Expenses and Other Current Assets | Prepaid expenses and other current assets consisted of the following: March 31, 2021 December 31, 2020 Tax incentive refund $ 2,739 $ 2,558 Prepaid research and development expenses 4,297 3,145 Prepaid insurance 692 973 Prepaid compensation benefits 922 609 Other current assets 387 275 $ 9,037 $ 7,560 |
Summary of Property and Equipment, Net | Property and equipment, net consisted of the following: March 31, 2021 December 31, 2020 Laboratory and office equipment $ 4,105 $ 3,965 Leasehold improvements 1,394 1,377 Computer equipment 143 143 5,642 5,485 Less: Accumulated depreciation and amortization (2,712 ) (2,421 ) Property and equipment, net $ 2,930 $ 3,064 |
Summary of Restricted Cash | As of March 31, 2021 and December 31, 2020, the Company had restricted cash as presented in the table below, which consists of cash used to secure letters of credit for the benefit of the landlord in connection with the Company’s lease agreements. The cash will be restricted until the termination or modification of the lease arrangement. March 31, 2021 December 31, 2020 Restricted cash $ 492 $ 492 |
Summary of Accrued Expenses | Accrued expenses and other current liabilities consisted of the following: March 31, 2021 December 31, 2020 Compensation and benefit costs $ 3,460 $ 6,402 Research and development expenses 5,151 5,847 Consulting and professional fees 942 1,096 Other liabilities 269 348 $ 9,822 $ 13,693 |
Stockholders' Equity (Tables)
Stockholders' Equity (Tables) | 3 Months Ended |
Mar. 31, 2021 | |
Equity [Abstract] | |
Summary of Common Stock Reserved for Future Issuance | As of March 31, 2021 and December 31, 2020, the Company has reserved the following shares of common stock for future issuance: March 31, 2021 December 31, 2020 Shares reserved for exercise of outstanding stock options 7,663,046 5,559,545 Shares reserved for vesting of restricted stock units 1,055 1,198 Shares reserved for issuance under the 2018 Stock Option and Grant Plan 3,433,617 3,877,478 Shares reserved for issuance under the 2018 Employee Stock Purchase Plan 1,178,590 762,900 Shares reserved for issuance under the 2019 Inducement Plan 171,300 365,050 Shares reserved for issuance under the 2020 Inducement Plan 1,700,000 1,700,000 Total shares of authorized common stock reserved for future issuance 14,147,608 12,266,171 |
Stock-Based Compensation (Table
Stock-Based Compensation (Tables) | 3 Months Ended |
Mar. 31, 2021 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Summary of Assumptions that Used to Determine Grant-Date Fair Value of Stock Options Granted to Employees and Members of Board of Directors | The assumptions that the Company used to determine the grant-date fair value of stock options granted to employees and members of the board of directors were as follows, presented on a weighted-average basis: Three Months Ended March 31, 2021 2020 Expected option life (years) 6.00 6.00 Risk-free interest rate 0.63 % 0.96 % Expected volatility 80.67 % 75.49 % Expected dividend yield — % — % |
Summary of Stock Option Activity | The following table summarizes the Company’s stock option activity for the three months ended March 31, 2021: Number of Options Weighted- Average Exercise Price Weighted- Average Remaining Contractual Term (Years) Aggregate Intrinsic Value Outstanding as of December 31, 2020 5,559,545 $ 14.97 7.65 $ 13,511 Granted 2,359,416 $ 15.66 Exercised (196,872 ) $ 4.32 Cancelled or forfeited (59,043 ) $ 17.88 Outstanding as of March 31, 2021 7,663,046 $ 15.45 8.36 $ 9,922 Exercisable as of March 31, 2021 2,254,165 $ 12.75 6.68 $ 8,626 |
Summary of Restricted Common Stock Units | The following table summarizes the Company’s restricted common stock units for the three months ended March 31, 2021: Number of Shares Weighted- Average Grant Date Fair Value Issued and unvested as of December 31, 2020 1,198 $ 15.65 Granted — Vested (143 ) $ 15.65 Forfeited, cancelled or expired — Issued and unvested as of March 31, 2021 1,055 $ 15.65 |
Schedule of Stock-based Compensation Expense | Stock-based compensation expense was allocated as follows: Three Months Ended March 31, 2021 2020 Research and development $ 2,115 $ 1,443 General and administrative 2,520 1,379 Total stock-based compensation expense $ 4,635 $ 2,822 |
Net Loss Per Share (Tables)
Net Loss Per Share (Tables) | 3 Months Ended |
Mar. 31, 2021 | |
Earnings Per Share [Abstract] | |
Schedule of Dilutive Common Stock Equivalents Excluded from Computation of Diluted Net Loss per Share | The following potentially dilutive common stock equivalents, presented based on amounts outstanding at each period end, were excluded from the computation of diluted net loss per share for the periods indicated: Three Months Ended March 31, 2021 2020 Options to purchase common stock 7,663,046 5,310,482 Restricted common stock 1,055 21,798 |
Nature of the Business - Additi
Nature of the Business - Additional Information (Detail) - USD ($) $ in Thousands | 3 Months Ended | ||
Mar. 31, 2021 | Mar. 31, 2020 | Dec. 31, 2020 | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | |||
Net loss | $ 26,899 | $ 25,973 | |
Accumulated deficit | 291,315 | $ 264,416 | |
Cash and cash equivalents | $ 233,027 | $ 257,695 | $ 259,682 |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies - Additional Information (Detail) | 3 Months Ended |
Mar. 31, 2021Segment | |
Significant Accounting Policies [Abstract] | |
Number of operating segment | 1 |
License Agreements - Additional
License Agreements - Additional Information (Detail) £ in Thousands, $ in Thousands | Sep. 30, 2020USD ($) | Sep. 30, 2020GBP (£) | Oct. 02, 2017USD ($) | Aug. 31, 2017USD ($)shares | Nov. 17, 2016USD ($) | Jan. 27, 2016USD ($) | Jan. 27, 2016CAD ($)shares | Mar. 31, 2021USD ($)shares | Mar. 31, 2020USD ($) | Dec. 31, 2020USD ($)shares | Dec. 31, 2018USD ($) |
License Agreement [Line Items] | |||||||||||
Accrued upfront license | $ 0 | $ 0 | |||||||||
Common stock, issued | shares | 41,766,000 | 41,569,000 | |||||||||
Fair value of shares issued | 93,628,000 | ||||||||||
Research and development | $ 18,527,000 | 18,274,000 | |||||||||
Preferred stock, issued | shares | 0 | 0 | |||||||||
University of Manchester Agreement [Member] | MPSII License Agreement [Member] | |||||||||||
License Agreement [Line Items] | |||||||||||
Upfront license fee | $ 8,000,000 | ||||||||||
Milestone payments | $ 80,000,000 | ||||||||||
Milestone payment paid | 2,000,000 | ||||||||||
Milestone payment due | $ 4,000,000 | ||||||||||
Agreement expiration period | 15 years | ||||||||||
Agreement expiration description | upon the later of 15 years from the effective date or the expiration of the last valid claim of the licensed patent | ||||||||||
Research and development expense | £ | £ 9,100 | ||||||||||
UHN Agreement [Member] | |||||||||||
License Agreement [Line Items] | |||||||||||
Research and development expense | $ 44,000 | 0 | |||||||||
Milestone fees | 0 | 0 | |||||||||
UHN Agreement [Member] | Fabry License Agreement [Member] | |||||||||||
License Agreement [Line Items] | |||||||||||
Upfront license fee | $ 75 | ||||||||||
Milestone payments | 2,450 | ||||||||||
Option fee | 20 | ||||||||||
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, issued | shares | 1,161,665 | ||||||||||
Fair value of shares issued | $ 480,000 | ||||||||||
Payments upon closing of an initial public offering | $ 2,000,000 | $ 2,000,000 | |||||||||
Research and development | 39,000 | 35,000 | |||||||||
BioMarin Pharmaceutical Inc [Member] | |||||||||||
License Agreement [Line Items] | |||||||||||
Upfront license fee | $ 500,000 | ||||||||||
Milestone payments | $ 13,000,000 | ||||||||||
Preferred stock, issued | shares | 233,765 | ||||||||||
Expenses related to license | 0 | 0 | |||||||||
GenStem Therapeutics Inc [Member] | |||||||||||
License Agreement [Line Items] | |||||||||||
Upfront license fee | $ 1,000,000 | ||||||||||
Milestone payments | $ 16,000,000 | ||||||||||
Research and development | 0 | 0 | |||||||||
Lund University Rights Holders Agreement [Member] | |||||||||||
License Agreement [Line Items] | |||||||||||
Milestone payments | $ 550,000 | ||||||||||
Expenses related to license | $ 0 | $ 0 |
Fair Value Measurement - Summar
Fair Value Measurement - Summary of Financial Assets Measured at Fair Value on Recurring Basis (Detail) - Fair Value on Recurring Basis [Member] - USD ($) $ in Thousands | Mar. 31, 2021 | Dec. 31, 2020 |
Assets Fair Value Disclosure | ||
Assets Fair Value Disclosure | $ 231,651 | $ 44,416 |
Money Market Funds [Member] | Cash Equivalents [Member] | ||
Assets Fair Value Disclosure | ||
Assets Fair Value Disclosure | 231,651 | 44,416 |
Level 1 [Member] | ||
Assets Fair Value Disclosure | ||
Assets Fair Value Disclosure | 231,651 | 44,416 |
Level 1 [Member] | Money Market Funds [Member] | Cash Equivalents [Member] | ||
Assets Fair Value Disclosure | ||
Assets Fair Value Disclosure | $ 231,651 | $ 44,416 |
Fair Value Measurement - Additi
Fair Value Measurement - Additional Information (Detail) | Mar. 31, 2021USD ($) |
Fair Value Disclosures [Abstract] | |
Fair value input transfer between levels | $ 0 |
Supplemental Balance Sheet In_3
Supplemental Balance Sheet Information - Summary of Prepaid Expenses and Other Current Assets (Detail) - USD ($) $ in Thousands | Mar. 31, 2021 | Dec. 31, 2020 |
Supplemental Balance Sheet Information Disclosures [Abstract] | ||
Tax incentive refund | $ 2,739 | $ 2,558 |
Prepaid research and development expenses | 4,297 | 3,145 |
Prepaid insurance | 692 | 973 |
Prepaid compensation benefits | 922 | 609 |
Other current assets | 387 | 275 |
Total | $ 9,037 | $ 7,560 |
Supplemental Balance Sheet In_4
Supplemental Balance Sheet Information - Summary of Property and Equipment, Net (Detail) - USD ($) $ in Thousands | Mar. 31, 2021 | Dec. 31, 2020 |
Property Plant And Equipment [Line Items] | ||
Property plant and equipment, gross | $ 5,642 | $ 5,485 |
Less: Accumulated depreciation and amortization | (2,712) | (2,421) |
Property and equipment, net | 2,930 | 3,064 |
Laboratory and Office Equipment [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property plant and equipment, gross | 4,105 | 3,965 |
Leasehold Improvements [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property plant and equipment, gross | 1,394 | 1,377 |
Computer Equipment [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property plant and equipment, gross | $ 143 | $ 143 |
Supplemental Balance Sheet In_5
Supplemental Balance Sheet Information - Additional Information (Detail) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Supplemental Balance Sheet Information Disclosures [Abstract] | ||
Depreciation and amortization expense | $ 291 | $ 283 |
Supplemental Balance Sheet In_6
Supplemental Balance Sheet Information - Summary of Restricted Cash (Detail) - USD ($) $ in Thousands | Mar. 31, 2021 | Dec. 31, 2020 |
Restricted Cash And Investments [Abstract] | ||
Restricted cash | $ 492 | $ 492 |
Supplemental Balance Sheet In_7
Supplemental Balance Sheet Information - Summary of Accrued Expenses (Detail) - USD ($) $ in Thousands | Mar. 31, 2021 | Dec. 31, 2020 |
Accrued Liabilities Current [Abstract] | ||
Compensation and benefit costs | $ 3,460 | $ 6,402 |
Research and development expenses | 5,151 | 5,847 |
Consulting and professional fees | 942 | 1,096 |
Other liabilities | 269 | 348 |
Accrued expenses | $ 9,822 | $ 13,693 |
Stockholders' Equity - Addition
Stockholders' Equity - Additional Information (Detail) - USD ($) | 3 Months Ended | ||
Mar. 31, 2021 | Dec. 31, 2020 | Mar. 31, 2020 | |
Equity [Abstract] | |||
Common stock, authorized | 150,000,000 | 150,000,000 | 150,000,000 |
Common stock, par value | $ 0.0001 | $ 0.0001 | $ 0.0001 |
Undesignated preferred stock | 10,000,000 | 10,000,000 | |
Undesignated shares of preferred stock outstanding | 0 | 0 | |
Cash dividends | $ 0 |
Stockholders' Equity - Summary
Stockholders' Equity - Summary of Common Stock Reserved for Future Issuance (Detail) - shares | Mar. 31, 2021 | Dec. 31, 2020 |
Class Of Stock [Line Items] | ||
Total shares of authorized common stock reserved for future issuance | 14,147,608 | 12,266,171 |
Employee Stock Option [Member] | ||
Class Of Stock [Line Items] | ||
Total shares of authorized common stock reserved for future issuance | 7,663,046 | 5,559,545 |
Restricted Stock Units [Member] | ||
Class Of Stock [Line Items] | ||
Total shares of authorized common stock reserved for future issuance | 1,055 | 1,198 |
2018 Stock Option and Incentive Plan [Member] | ||
Class Of Stock [Line Items] | ||
Total shares of authorized common stock reserved for future issuance | 3,433,617 | 3,877,478 |
2018 Employee Stock Purchase Plan [Member] | ||
Class Of Stock [Line Items] | ||
Total shares of authorized common stock reserved for future issuance | 1,178,590 | 762,900 |
2019 Inducement Plan [Member] | ||
Class Of Stock [Line Items] | ||
Total shares of authorized common stock reserved for future issuance | 171,300 | 365,050 |
2020 Inducement Plan [Member] | ||
Class Of Stock [Line Items] | ||
Total shares of authorized common stock reserved for future issuance | 1,700,000 | 1,700,000 |
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 of Directors (Detail) | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Share Based Compensation Arrangement By Share Based Payment Award Fair Value Assumptions And Methodology [Abstract] | ||
Expected option life (years) | 6 years | 6 years |
Risk-free interest rate | 0.63% | 0.96% |
Expected volatility | 80.67% | 75.49% |
Stock-Based Compensation - Su_2
Stock-Based Compensation - Summary of Stock Option Activity (Detail) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 12 Months Ended |
Mar. 31, 2021 | Dec. 31, 2020 | |
Share Based Compensation Arrangement By Share Based Payment Award Options Outstanding Roll Forward | ||
Number of options, Outstanding beginning balance | 5,559,545 | |
Number of options, Granted | 2,359,416 | |
Number of options, Exercised | (196,872) | |
Number of options, Cancelled or forfeited | (59,043) | |
Number of options, Outstanding ending balance | 7,663,046 | 5,559,545 |
Number of options, Exercisable | 2,254,165 | |
Weighted average exercise price, Outstanding beginning balance | $ 14.97 | |
Weighted average exercise price, Granted | 15.66 | |
Weighted average exercise price, Exercised | 4.32 | |
Weighted average exercise price, Cancelled or forfeited | 17.88 | |
Weighted average exercise price, Outstanding ending balance | 15.45 | $ 14.97 |
Weighted average exercise price, Exercisable | $ 12.75 | |
Weighted average remaining contractual term, Outstanding balance | 8 years 4 months 9 days | 7 years 7 months 24 days |
Weighted average remaining contractual term, Exercisable | 6 years 8 months 4 days | |
Aggregate intrinsic value, Outstanding balance | $ 9,922 | $ 13,511 |
Aggregate intrinsic value, Exercisable | $ 8,626 |
Stock-Based Compensation - Addi
Stock-Based Compensation - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | ||
Aggregate intrinsic value of options exercised | $ 1,729 | $ 133 |
Options Granted, Weighted-average grant-date fair value | $ 10.69 | $ 13.68 |
Total fair value of restricted stock units vested | $ 2 | $ 15 |
Unrecognized stock-based compensation expenses | $ 56,945 | |
Unrecognized stock-based compensation expense, period for recognition | 3 years 2 months 23 days |
Stock-Based Compensation - Su_3
Stock-Based Compensation - Summary of Restricted Common Stock Units (Detail) - Restricted Stock Units [Member] | 3 Months Ended |
Mar. 31, 2021$ / sharesshares | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
Number of shares, Issued and unvested beginning balance | shares | 1,198 |
Number of shares, Vested | shares | (143) |
Number of shares, Issued and unvested ending balance | shares | 1,055 |
Weighted average grant date fair value, Issued and unvested, beginning balance | $ / shares | $ 15.65 |
Weighted average grant date fair value, Vested | $ / shares | 15.65 |
Weighted average grant date fair value, Issued and unvested, ending balance | $ / shares | $ 15.65 |
Stock-Based Compensation - Sche
Stock-Based Compensation - Schedule of Stock-based Compensation Expense (Detail) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||
Total stock-based compensation expense | $ 4,635 | $ 2,822 |
Research and Development Expense [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||
Total stock-based compensation expense | 2,115 | 1,443 |
General and Administrative Expense [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||
Total stock-based compensation expense | $ 2,520 | $ 1,379 |
Net Loss Per Share - Schedule o
Net Loss Per Share - Schedule of Dilutive Common Stock Equivalents Excluded from Computation of Diluted Net Loss per Share (Detail) - shares | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Options to Purchase Common Stock [Member] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Antidilutive securities excluded from computation of earnings per share amount | 7,663,046 | 5,310,482 |
Restricted Common Stock [Member] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Antidilutive securities excluded from computation of earnings per share amount | 1,055 | 21,798 |
Related Party Transactions - Ad
Related Party Transactions - Additional information (Detail) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Related Party Transaction [Line Items] | ||
Research and development expense | $ 18,527 | $ 18,274 |
License Agreement [Member] | ||
Related Party Transaction [Line Items] | ||
Research and development expense | 39 | 35 |
Sub-lease Agreement [Member] | Officers and Board Members [Member] | ||
Related Party Transaction [Line Items] | ||
Expense related to related party | $ 641 | $ 360 |