Document and Entity Information
Document and Entity Information - shares | 9 Months Ended | |
Sep. 30, 2020 | Oct. 30, 2020 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Sep. 30, 2020 | |
Document Fiscal Year Focus | 2020 | |
Document Fiscal Period Focus | Q3 | |
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 | 36,450,950 | |
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 | Sep. 30, 2020 | Dec. 31, 2019 |
Current assets: | ||
Cash and cash equivalents | $ 219,546 | $ 187,043 |
Prepaid expenses and other current assets | 9,440 | 8,658 |
Total current assets | 228,986 | 195,701 |
Property and equipment, net | 3,312 | 3,696 |
Restricted cash | 492 | 492 |
Other assets | 465 | 625 |
Total assets | 233,255 | 200,514 |
Current liabilities: | ||
Accounts payable | 2,311 | 3,949 |
Accrued expenses and other current liabilities | 22,525 | 9,854 |
Deferred rent | 216 | 214 |
Total current liabilities | 25,052 | 14,017 |
Deferred rent, net of current portion | 331 | 484 |
Total liabilities | 25,383 | 14,501 |
Commitments and contingencies (Note 11) | ||
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; 36,449 and 31,643 shares issued and outstanding as of September 30, 2020 and December 31, 2019, respectively | 4 | 3 |
Additional paid-in capital | 444,153 | 330,714 |
Accumulated deficit | (236,285) | (144,704) |
Total stockholders’ equity | 207,872 | 186,013 |
Total liabilities and stockholders’ equity | $ 233,255 | $ 200,514 |
Condensed Consolidated Balanc_2
Condensed Consolidated Balance Sheets (Parenthetical) - $ / shares | Sep. 30, 2020 | Dec. 31, 2019 |
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 | 36,449,000 | 31,643,000 |
Common stock, outstanding | 36,449,000 | 31,643,000 |
Condensed Consolidated Statemen
Condensed Consolidated Statements of Operations and Comprehensive Loss - USD ($) shares in Thousands, $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2020 | Sep. 30, 2019 | Sep. 30, 2020 | Sep. 30, 2019 | |
Operating expenses: | ||||
Research and development | $ 28,509 | $ 13,042 | $ 67,649 | $ 37,755 |
General and administrative | 8,209 | 5,022 | 24,515 | 14,621 |
Total operating expenses | 36,718 | 18,064 | 92,164 | 52,376 |
Loss from operations | (36,718) | (18,064) | (92,164) | (52,376) |
Other income (expense): | ||||
Interest income | 12 | 925 | 706 | 2,146 |
Other expense | (74) | (6) | (123) | (73) |
Total other income (expense), net | (62) | 919 | 583 | 2,073 |
Net loss | (36,780) | (17,145) | (91,581) | (50,303) |
Comprehensive loss | $ (36,780) | $ (17,145) | $ (91,581) | $ (50,303) |
Net loss per share attributable to common stockholders—basic and diluted (Note 10) | $ (1.01) | $ (0.57) | $ (2.59) | $ (1.93) |
Weighted-average number of common shares outstanding used in computing net loss per share attributable to common stockholders—basic and diluted | 36,444 | 30,297 | 35,409 | 26,019 |
Condensed Consolidated Statem_2
Condensed Consolidated Statements of Stockholders' Equity - USD ($) $ in Thousands | Total | Public Offering [Member] | ATM Facility [Member] | Common Stock [Member] | Common Stock [Member]Public Offering [Member] | Common Stock [Member]ATM Facility [Member] | Additional Paid-in Capital [Member] | Additional Paid-in Capital [Member]Public Offering [Member] | Additional Paid-in Capital [Member]ATM Facility [Member] | Accumulated Deficit [Member] |
Beginning balance at Dec. 31, 2018 | $ 122,184 | $ 2 | $ 193,921 | $ (71,739) | ||||||
Beginning balance, shares at Dec. 31, 2018 | 23,807,000 | |||||||||
Stock-based compensation expense | 1,455 | 1,455 | ||||||||
Exercise of stock options | 252 | 252 | ||||||||
Exercise of stock options, shares | 117,000 | |||||||||
Vesting of restricted stock awards and units, shares | 30,000 | |||||||||
Net loss | (17,103) | (17,103) | ||||||||
Ending balance at Mar. 31, 2019 | 106,788 | $ 2 | 195,628 | (88,842) | ||||||
Ending balance, shares at Mar. 31, 2019 | 23,954,000 | |||||||||
Beginning balance at Dec. 31, 2018 | 122,184 | $ 2 | 193,921 | (71,739) | ||||||
Beginning balance, shares at Dec. 31, 2018 | 23,807,000 | |||||||||
Net loss | (50,303) | |||||||||
Ending balance at Sep. 30, 2019 | 206,760 | $ 3 | 328,799 | (122,042) | ||||||
Ending balance, shares at Sep. 30, 2019 | 31,607,000 | |||||||||
Beginning balance at Mar. 31, 2019 | 106,788 | $ 2 | 195,628 | (88,842) | ||||||
Beginning balance, shares at Mar. 31, 2019 | 23,954,000 | |||||||||
Stock-based compensation expense | 1,663 | 1,663 | ||||||||
Exercise of stock options | 238 | 238 | ||||||||
Exercise of stock options, shares | 110,000 | |||||||||
Vesting of restricted stock awards and units, shares | 31,000 | |||||||||
Net loss | (16,055) | (16,055) | ||||||||
Ending balance at Jun. 30, 2019 | 92,634 | $ 2 | 197,529 | (104,897) | ||||||
Ending balance, shares at Jun. 30, 2019 | 24,095,000 | |||||||||
Stock-based compensation expense | 1,797 | 1,797 | ||||||||
Exercise of stock options | 9 | 9 | ||||||||
Exercise of stock options, shares | 6,000 | |||||||||
Vesting of restricted stock awards and units, shares | 31,000 | |||||||||
Issuance of common stock, net of offering costs | $ 129,465 | $ 1 | $ 129,464 | |||||||
Issuance of common stock, net of offering costs, shares | 7,475,000 | |||||||||
Net loss | (17,145) | (17,145) | ||||||||
Ending balance at Sep. 30, 2019 | 206,760 | $ 3 | 328,799 | (122,042) | ||||||
Ending balance, shares at Sep. 30, 2019 | 31,607,000 | |||||||||
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, 2019 | $ 186,013 | $ 3 | 330,714 | (144,704) | ||||||
Beginning balance, shares at Dec. 31, 2019 | 31,643,000 | |||||||||
Exercise of stock options, shares | 38,761 | |||||||||
Net loss | $ (91,581) | |||||||||
Ending balance at Sep. 30, 2020 | 207,872 | $ 4 | 444,153 | (236,285) | ||||||
Ending balance, shares at Sep. 30, 2020 | 36,449,000 | |||||||||
Beginning balance at Mar. 31, 2020 | 256,525 | $ 4 | 427,198 | (170,677) | ||||||
Beginning balance, shares at Mar. 31, 2020 | 36,011,000 | |||||||||
Stock-based compensation expense | 3,899 | 3,899 | ||||||||
Exercise of stock options | 61 | 61 | ||||||||
Exercise of stock options, shares | 25,000 | |||||||||
Vesting of restricted stock awards and units, shares | 10,000 | |||||||||
Issuance of common stock, net of offering costs | $ 8,130 | $ 8,130 | ||||||||
Issuance of common stock, net of offering costs, shares | 384,000 | |||||||||
Issuance of common stock under the 2018 employee stock purchase plan, shares | 2,000 | |||||||||
Net loss | (28,828) | (28,828) | ||||||||
Ending balance at Jun. 30, 2020 | 239,787 | $ 4 | 439,288 | (199,505) | ||||||
Ending balance, shares at Jun. 30, 2020 | 36,432,000 | |||||||||
Stock-based compensation expense | 4,805 | 4,805 | ||||||||
Exercise of stock options | 60 | 60 | ||||||||
Exercise of stock options, shares | 7,000 | |||||||||
Vesting of restricted stock awards and units, shares | 10,000 | |||||||||
Net loss | (36,780) | (36,780) | ||||||||
Ending balance at Sep. 30, 2020 | $ 207,872 | $ 4 | $ 444,153 | $ (236,285) | ||||||
Ending balance, shares at Sep. 30, 2020 | 36,449,000 |
Condensed Consolidated Statem_3
Condensed Consolidated Statements of Stockholders' Equity (Parenthetical) - USD ($) $ in Thousands | 3 Months Ended | ||
Jun. 30, 2020 | Mar. 31, 2020 | Sep. 30, 2019 | |
Public Offering [Member] | |||
Issuance of common stock upon public offering, offering costs | $ 419 | $ 525 | |
ATM Facility [Member] | |||
Issuance of common stock upon public offering, offering costs | $ 67 |
Condensed Consolidated Statem_4
Condensed Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2020 | Sep. 30, 2019 | Sep. 30, 2020 | Sep. 30, 2019 | |
Cash flows from operating activities: | ||||
Net loss | $ (91,581) | $ (50,303) | ||
Adjustments to reconcile net loss to net cash used in operating activities: | ||||
Depreciation and amortization expense | $ 314 | $ 215 | 898 | 605 |
Stock-based compensation expense | 11,526 | 4,915 | ||
Deferred rent expense | (151) | (123) | ||
Changes in operating assets and liabilities: | ||||
Prepaid expenses and other current assets | (782) | (3,616) | ||
Other assets | 160 | (13) | ||
Accounts payable | (1,263) | (1,127) | ||
Accrued expenses and other current liabilities | 12,878 | 577 | ||
Net cash used in operating activities | (68,315) | (49,085) | ||
Cash flows from investing activities: | ||||
Purchases of property and equipment | (1,096) | (819) | ||
Net cash used in investing activities | (1,096) | (819) | ||
Cash flows from financing activities: | ||||
Proceeds from exercise of stock options | 156 | 499 | ||
Net cash provided by financing activities | 101,914 | 129,964 | ||
Net increase in cash, cash equivalents and restricted cash | 32,503 | 80,060 | ||
Cash, cash equivalents and restricted cash at beginning of period | 187,535 | 126,794 | ||
Cash, cash equivalents and restricted cash at end of period | 220,038 | 206,854 | 220,038 | 206,854 |
Supplemental disclosure of non-cash investing and financing activities: | ||||
Purchases of property and equipment included in accounts payable and accrued expenses | 80 | |||
Reconciliation of cash, cash equivalents and restricted cash reported within the condensed consolidated balance sheets: | ||||
Cash and cash equivalents | 219,546 | 206,362 | 219,546 | 206,362 |
Restricted cash | 492 | 492 | 492 | 492 |
Cash, cash equivalents and restricted cash at end of period | $ 220,038 | $ 206,854 | 220,038 | 206,854 |
Public Offering [Member] | ||||
Cash flows from financing activities: | ||||
Proceeds from issuance of common shares, net of offering costs | 93,628 | $ 129,465 | ||
ATM Facility [Member] | ||||
Cash flows from financing activities: | ||||
Proceeds from issuance of common shares, net of offering costs | $ 8,130 |
Nature of the Business
Nature of the Business | 9 Months Ended |
Sep. 30, 2020 | |
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 $91,581 and $50,303 for the nine months ended September 30, 2020 and 2019, respectively. In addition, as of September 30, 2020, the Company had an accumulated deficit of $236,285. 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 September 30, 2020 of $219,546 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 | 9 Months Ended |
Sep. 30, 2020 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies Basis of Presentation The accompanying 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 interim 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, 2019, 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 September 30, 2020, and the results of its operations for the three and nine months ended September 30, 2020 and 2019, its statements of stockholders’ equity for the three and nine months ended September 30, 2020 and 2019 and its statement of cash flows for the nine months ended September 30, 2020 and 2019. The results for the three and nine months ended September 30, 2020 are not necessarily indicative of the results to be expected for the year ending December 31, 2020, 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, 2019, and the notes thereto, which are included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2019, filed with the SEC on March 16, 2020. The accompanying 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 September 30, 2020, 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, 2019, except as it related to the adoption of new accounting standards during the first nine months of 2020 as discussed below. Reclassifications Certain reclassifications have been made to the prior year condensed consolidated balance sheet to conform to the current year presentation, which reflect a reclassification from other assets to restricted cash for $492 and from other current liabilities to deferred rent for $231. These reclassifications have no impact on the Company’s net loss or cash flows. Use of Estimates The preparation of consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, 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. Actual results may differ from these estimates under different assumptions or conditions. 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 initial public offering (“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 Adopted Accounting Pronouncements In November 2018, the FASB issued ASU No. 2018-18, Collaborative Arrangements (Topic 808)—Clarifying the Interaction between Topic 808 and Topic 606 In August 2018, the FASB issued ASU No. 2018-15, Intangible-Goodwill and Other Internal-Use Software (Subtopic 350-40), In August 2018, t he FASB issued ASU No. 2018-13, Fair Value Measurement (Topic 820): Disclosure Framework—Changes to the Disclosure Requirements for Fair Value Measurement , or ASU 2018-13. The amendments in ASU 2018-13 eliminate, add, and modify certain disclosure requirements for fair value measurements. The amendments are effective for interim and annual reporting periods beginning after December 15, 2019, with early adoption permitted for either the entire ASU or only the provisions that eliminate or modify requirements. The amendments with respect to changes in unrealized gains and losses, the range and weighted-average of significant unobservable inputs used to develop Level 3 fair value measurements, and the narrative description of measurement uncertainty are to be applied prospectively. All other amendments are to be applied retrospectively to all periods presented. The Company adopted ASU 2018-13 during the quarter ended March 31, 2020 . The adoption did not have a material impact on the condensed consolidated financial statements. Recently Issued Accounting Pronouncements In February 2016, the FASB issued ASU No. 2016-02, (Topic 842) Leases, he Company currently expects that its operating lease commitments will be subject to the new standard and recognized as right-of-use assets and operating lease liabilities upon adoption of this standard, which will increase the Company’s total assets and total liabilities. In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments—Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments In Codification Improvements to Topic 326, Financial Instruments – Credit Losses Financial Instruments – Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments As a result of the Company having elected the extended transition period for complying with new or revised accounting standards pursuant to Section 107(b) of the JOBS Act, 2016-13 and ASU 2019-11 are effective for the Company for fiscal years beginning after December 15, 2020, and interim periods within fiscal years beginning after December 15, 2021. In December 2019, the FASB issued ASU No. 2019-12, Simplifying the Accounting for Income Taxes |
Fair Value Measurement
Fair Value Measurement | 9 Months Ended |
Sep. 30, 2020 | |
Fair Value Disclosures [Abstract] | |
Fair Value Measurement | 3. 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 September 30, 2020 and December 31, 2019: Fair Value Measurements as of September 30, 2020 Level 1 Level 2 Level 3 Total Assets: Money market funds $ 219,306 $ — $ — $ 219,306 $ 219,306 $ — $ — $ 219,306 Fair Value Measurements as of December 31, 2019 Level 1 Level 2 Level 3 Total Assets: Money market funds $ 186,797 $ — $ — $ 186,797 $ 186,797 $ — $ — $ 186,797 The fair value of cash equivalents was determined through quoted prices by third-party pricing services. During the three and nine months ended September 30, 2020, there were no transfers between levels. |
Prepaid Expenses and Other Curr
Prepaid Expenses and Other Current Assets | 9 Months Ended |
Sep. 30, 2020 | |
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: September 30, 2020 December 31, 2019 Tax incentive refund $ 2,366 $ 1,916 Prepaid research and development costs 3,528 4,915 Prepaid insurance 1,762 897 Prepaid compensation and benefit costs 621 362 Other current assets 1,163 568 $ 9,440 $ 8,658 |
Property and Equipment, Net
Property and Equipment, Net | 9 Months Ended |
Sep. 30, 2020 | |
Property Plant And Equipment [Abstract] | |
Property and Equipment, Net | 5. Property and Equipment, Net Property and equipment, net consisted of the following: September 30, 2020 December 31, 2019 Laboratory and office equipment $ 3,923 $ 3,456 Leasehold improvements 1,378 1,340 Computer equipment and software 143 134 5,444 4,930 Less: Accumulated depreciation and amortization (2,132 ) (1,234 ) $ 3,312 $ 3,696 Depreciation and amortization expense was $314 and $215 for the three months ended September 30, 2020 and 2019, respectively and $898 and $605 for the nine months ended September 30, 2020 and 2019. |
Accrued Expenses and Other Curr
Accrued Expenses and Other Current Liabilities | 9 Months Ended |
Sep. 30, 2020 | |
Payables And Accruals [Abstract] | |
Accrued Expenses and Other Current Liabilities | 6. Accrued Expenses and Other Current Liabilities Accrued expenses and other current liabilities consisted of the following: September 30, 2020 December 31, 2019 Compensation and benefit costs $ 5,761 $ 3,028 Research and development costs 7,540 5,794 Consulting and professional fees 1,028 917 License agreement 8,000 — Other liabilities 196 115 $ 22,525 $ 9,854 |
Stockholders' Equity
Stockholders' Equity | 9 Months Ended |
Sep. 30, 2020 | |
Equity [Abstract] | |
Stockholders' Equity | 7. Stockholders’ Equity C ommon Stock As of September 30, 2020 and December 31, 2019, 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 September 30, 2020 and December 31, 2019, no undesignated preferred stock was outstanding. In accordance with 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 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. As of September 30, 2020, no cash dividends have been declared or paid. Common Stock Reserved for Future Issuance As of September 30, 2020 and December 31, 2019, the Company has reserved the following shares of common stock for future issuance: September 30, 2020 December 31, 2019 Shares reserved for vesting of restricted stock awards — 30,252 Shares reserved for exercise of outstanding stock options 5,658,890 3,414,445 Shares reserved for vesting of restricted stock units 1,342 2,300 Shares reserved for issuance under the 2018 Stock Option and Grant Plan 3,541,042 332,513 Shares reserved for issuance under the 2018 Employee Stock Purchase Plan 774,088 459,595 Shares reserved for issuance under the 2019 Inducement Plan 831,550 1,800,000 Total shares of authorized common stock reserved for future issuance 10,806,912 6,039,105 |
Stock-Based Compensation
Stock-Based Compensation | 9 Months Ended |
Sep. 30, 2020 | |
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: Nine Months Ended September 30, 2020 2019 Expected option life (years) 6.00 5.97 Risk-free interest rate 0.84 % 2.40 % Expected volatility 75.85 % 81.25 % Expected dividend yield — % — % The following table summarizes the Company’s stock option activity for the nine months ended September 30, 2020: Number of Options Weighted- Average Exercise Price Weighted- Average Remaining Contractual Term (Years) Aggregate Intrinsic Value Outstanding as of December 31, 2019 3,414,445 $ 12.08 8.43 $ 29,353 Granted 2,485,413 $ 19.82 Exercised (38,761 ) $ 4.05 Cancelled or forfeited (202,207 ) $ 20.07 Outstanding as of September 30, 2020 5,658,890 $ 15.25 8.43 $ 13,481 Exercisable as of September 30, 2020 1,754,340 $ 8.92 7.06 $ 11,057 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 nine months ended September 30, 2020 and 2019 was $595 and $3,446, respectively. The weighted-average grant-date fair value of the Company’s stock options granted during the nine months ended September 30, 2020 and 2019 was $12.98 and $11.77, respectively. Restricted Common Stock The following table summarizes the Company’s restricted common stock activity for the nine months ended September 30, 2020: Number of Shares Weighted- Average Grant Date Fair Value Issued and unvested as of December 31, 2019 32,552 $ 1.50 Granted — Vested (31,210 ) $ 0.89 Forfeited, cancelled or expired — Issued and unvested as of September 30, 2020 1,342 $ 15.65 The total fair value of restricted common stock vested during the nine months ended September 30, 2020 and 2019 was $28 and $44, respectively. Stock-Based Compensation Stock-based compensation expense was allocated as follows: Three Months Ended September 30, Nine Months Ended September 30, 2020 2019 2020 2019 Research and development $ 2,686 $ 889 $ 6,095 $ 2,649 General and administrative 2,119 908 5,431 2,265 Total stock-based compensation expense $ 4,805 $ 1,797 $ 11,526 $ 4,914 As of September 30, 2020, total unrecognized compensation cost related to the unvested stock-based awards was $41,143, which is expected to be recognized over a weighted-average period of 3.08 years. During the three months ended September 30, 2020, the Company modified certain awards in conjunction with an employee’s termination. The modification provided for the extension of the post-employment exercise period. The modifications resulted in approximately $274 of additional research and development expenses during the three and nine months ended September 30, 2020. |
License Agreements
License Agreements | 9 Months Ended |
Sep. 30, 2020 | |
Text Block [Abstract] | |
License Agreements | 9. License Agreements Agreement with The University of Manchester On September 30, 2020, the Company entered into an agreement (“MPSII License Agreement”) with The University of Manchester, England (“UoM”), whereby UoM granted to the Company an exclusive worldwide license under certain patent and other intellectual property rights, subject to certain retained rights, to develop, commercialize and sell an ex vivo lentiviral gene therapy for use in the treatment of Hunter syndrome, or mucopolysaccharidosis type II (“MPSII”) As part of the agreement, the Company is obligated to make milestone payments of up to an aggregate of $80,000 upon the achievement of specified development and regulatory milestones, to pay royalties, on a product-by-product and country-by-country basis, of a mid-single digit percentage based on net sales of products licensed under the agreement and to pay a low double digit percentage of any sublicense fees received by the Company. The next anticipated payment milestones under the MPSII License Agreement include $2,000, which would become due following the date of regulatory approval of the clinical trial application for the investigator-sponsored Phase 1/2 clinical trial sponsored by UoM, and $4,000, upon the dosing of the first patient in the investigator-sponsored Phase 1/2 clinical trial sponsored by UoM. Unless terminated earlier, the agreement expires upon the later of 15 years from the effective date or the expiration of the last valid claim of the licensed patents, subject to certain surviving rights and obligations. UoM and the Company can each terminate the agreement in the event of the bankruptcy or insolvency of the other party, or a material breach by the other party and failure to cure such breach within a certain period of time. UoM has the right to terminate the agreement in the event of certain actions relating to challenge or opposition to the licensed intellectual property brought by the Company or its affiliates or sublicensees. Concurrently with the MPSII License Agreement, the Company entered into a collaborative research funding agreement with UoM (“CRFA”). Under the CRFA, the Company has agreed to fund the budgeted costs of an investigator-sponsored Phase 1/2 clinical trial to be sponsored by UoM in connection with the development activities under the MPSII License Agreement, which are currently estimated to equal approximately £9,100 in the aggregate As of September 30, 2020, the Company recognized no costs related to the CRFA. Agreements with UHN Fabry License Agreement— On January 27, 2016, the Company entered into an agreement with the University Health Network (“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. 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 September 30, 2020 and 2019, the Company recorded research and development expense related to this agreement with UHN of $65 and $415, respectively, which consists of reimbursable funded study trial costs. For the nine months ended September 30, 2020 and 2019, the Company recorded research and development expense related to this agreement with UHN of $174 and $741, respectively, which consists of reimbursable funded study trial costs. No milestone or maintenance fees were incurred related to the Fabry license agreement in the nine months ended September 30, 2020 and 2019. 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 its IPO if certain criteria were met. This obligation was considered a derivative instrument and was initially recorded at fair value. 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 September 30, 2020 and 2019, the Company did not record research and development expense related to this agreement with UHN. 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. As consideration for this agreement, the Company paid an upfront license fee of $500 in cash and issued 233,765 shares of preferred stock to BioMarin in January 2018. The Company is also obligated to make future milestone payments of up to $13,000 upon the achievement of certain specified milestones and agreed to pay BioMarin royalties of a low single-digit percentage of net sales of licensed products sold by the Company or its affiliates covered by patent rights in a relevant country No milestone fees related to the license were recorded for the nine months ended September 30, 2020 and 2019. 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. In November 2019, the Company made a payment of $2,000 in connection with the dosing of the first patient in the investigator-sponsored Phase 1/2 clinical trial of AVR-RD-04 in cystinosis in the United States. The next anticipated payment under this Agreement is $2,000, which would become due following the dosing of the first patient in the first pivotal clinical trial of AVR-RD-04 in cystinosis in the United States. 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 milestone fees related to the license were recorded for the nine months ended September 30, 2020 and 2019. |
Net Loss per Share Attributable
Net Loss per Share Attributable to Common Stockholders | 9 Months Ended |
Sep. 30, 2020 | |
Earnings Per Share [Abstract] | |
Net Loss per Share Attributable to Common Stockholders | 10. 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: Nine Months Ended September 30, 2020 2019 Options to purchase common stock 5,658,890 3,145,428 Restricted common stock 1,342 63,313 |
Commitments and Contingencies
Commitments and Contingencies | 9 Months Ended |
Sep. 30, 2020 | |
Commitments And Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 11. Commitments and Contingencies Lease Agreement On June 1, 2020, the Company entered into a lease agreement for office space located in Toronto, Ontario, Canada, which is set to expire in June 2025. The annual lease payments are fixed for years 1 and 2, and then subject to a 6.67% increase for years 2 through 5. As a result, the Company’s lease obligations through 2025 increased by CAD $865 during the nine months ended September 30, 2020. 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 nine months ended September 30, 2020 and 2019 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 September 30, 2020 and December 31, 2019, 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 9. See Note 9 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. 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 at September 30, 2020 . 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. |
Related Party Transactions
Related Party Transactions | 9 Months Ended |
Sep. 30, 2020 | |
Related Party Transactions [Abstract] | |
Related Party Transactions | 12. Related Party Transactions UHN For the three months ended September 30, 2020 and 2019, the Company recognized $65 and $415, respectively, of research and development expense related to the license agreements with UHN. For the nine months ended September 30, 2020 and 2019, the Company recognized $212 and $779, respectively, of research and development expense related to the license agreements with UHN (Note 9). Others For three months ended September 30, 2020 and 2019, the Company recorded expenses of $355 and $369, respectively, related to a sublease to rent lab space, provided by an entity affiliated with a member of the Company’s board of directors. For the nine months ended September 30, 2020 and 2019, the Company recorded expenses of $1,081 and $1,090, 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) | 9 Months Ended |
Sep. 30, 2020 | |
Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation The accompanying 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 interim 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, 2019, 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 September 30, 2020, and the results of its operations for the three and nine months ended September 30, 2020 and 2019, its statements of stockholders’ equity for the three and nine months ended September 30, 2020 and 2019 and its statement of cash flows for the nine months ended September 30, 2020 and 2019. The results for the three and nine months ended September 30, 2020 are not necessarily indicative of the results to be expected for the year ending December 31, 2020, 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, 2019, and the notes thereto, which are included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2019, filed with the SEC on March 16, 2020. The accompanying 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 September 30, 2020, 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, 2019, except as it related to the adoption of new accounting standards during the first nine months of 2020 as discussed below. |
Reclassifications | Reclassifications Certain reclassifications have been made to the prior year condensed consolidated balance sheet to conform to the current year presentation, which reflect a reclassification from other assets to restricted cash for $492 and from other current liabilities to deferred rent for $231. These reclassifications have no impact on the Company’s net loss or cash flows. |
Use of Estimates | Use of Estimates The preparation of consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, 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. Actual results may differ from these estimates under different assumptions or conditions. |
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 initial public offering (“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 Adopted Accounting Pronouncements | Recently Adopted Accounting Pronouncements In November 2018, the FASB issued ASU No. 2018-18, Collaborative Arrangements (Topic 808)—Clarifying the Interaction between Topic 808 and Topic 606 In August 2018, the FASB issued ASU No. 2018-15, Intangible-Goodwill and Other Internal-Use Software (Subtopic 350-40), In August 2018, t he FASB issued ASU No. 2018-13, Fair Value Measurement (Topic 820): Disclosure Framework—Changes to the Disclosure Requirements for Fair Value Measurement , or ASU 2018-13. The amendments in ASU 2018-13 eliminate, add, and modify certain disclosure requirements for fair value measurements. The amendments are effective for interim and annual reporting periods beginning after December 15, 2019, with early adoption permitted for either the entire ASU or only the provisions that eliminate or modify requirements. The amendments with respect to changes in unrealized gains and losses, the range and weighted-average of significant unobservable inputs used to develop Level 3 fair value measurements, and the narrative description of measurement uncertainty are to be applied prospectively. All other amendments are to be applied retrospectively to all periods presented. The Company adopted ASU 2018-13 during the quarter ended March 31, 2020 . The adoption did not have a material impact on the condensed consolidated financial statements. |
Recently Issued Accounting Pronouncements | Recently Issued Accounting Pronouncements In February 2016, the FASB issued ASU No. 2016-02, (Topic 842) Leases, he Company currently expects that its operating lease commitments will be subject to the new standard and recognized as right-of-use assets and operating lease liabilities upon adoption of this standard, which will increase the Company’s total assets and total liabilities. In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments—Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments In Codification Improvements to Topic 326, Financial Instruments – Credit Losses Financial Instruments – Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments As a result of the Company having elected the extended transition period for complying with new or revised accounting standards pursuant to Section 107(b) of the JOBS Act, 2016-13 and ASU 2019-11 are effective for the Company for fiscal years beginning after December 15, 2020, and interim periods within fiscal years beginning after December 15, 2021. In December 2019, the FASB issued ASU No. 2019-12, Simplifying the Accounting for Income Taxes |
Fair Value Measurement (Tables)
Fair Value Measurement (Tables) | 9 Months Ended |
Sep. 30, 2020 | |
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 September 30, 2020 and December 31, 2019: Fair Value Measurements as of September 30, 2020 Level 1 Level 2 Level 3 Total Assets: Money market funds $ 219,306 $ — $ — $ 219,306 $ 219,306 $ — $ — $ 219,306 Fair Value Measurements as of December 31, 2019 Level 1 Level 2 Level 3 Total Assets: Money market funds $ 186,797 $ — $ — $ 186,797 $ 186,797 $ — $ — $ 186,797 |
Prepaid Expenses and Other Cu_2
Prepaid Expenses and Other Current Assets (Tables) | 9 Months Ended |
Sep. 30, 2020 | |
Text Block [Abstract] | |
Summary of Prepaid Expenses and Other Current Assets | Prepaid expenses and other current assets consisted of the following: September 30, 2020 December 31, 2019 Tax incentive refund $ 2,366 $ 1,916 Prepaid research and development costs 3,528 4,915 Prepaid insurance 1,762 897 Prepaid compensation and benefit costs 621 362 Other current assets 1,163 568 $ 9,440 $ 8,658 |
Property and Equipment, Net (Ta
Property and Equipment, Net (Tables) | 9 Months Ended |
Sep. 30, 2020 | |
Property Plant And Equipment [Abstract] | |
Summary of Property and Equipment, Net | Property and equipment, net consisted of the following: September 30, 2020 December 31, 2019 Laboratory and office equipment $ 3,923 $ 3,456 Leasehold improvements 1,378 1,340 Computer equipment and software 143 134 5,444 4,930 Less: Accumulated depreciation and amortization (2,132 ) (1,234 ) $ 3,312 $ 3,696 |
Accrued Expenses and Other Cu_2
Accrued Expenses and Other Current Liabilities (Tables) | 9 Months Ended |
Sep. 30, 2020 | |
Payables And Accruals [Abstract] | |
Summary of Accrued Expenses and Other Current Liabilities | Accrued expenses and other current liabilities consisted of the following: September 30, 2020 December 31, 2019 Compensation and benefit costs $ 5,761 $ 3,028 Research and development costs 7,540 5,794 Consulting and professional fees 1,028 917 License agreement 8,000 — Other liabilities 196 115 $ 22,525 $ 9,854 |
Stockholders' Equity (Tables)
Stockholders' Equity (Tables) | 9 Months Ended |
Sep. 30, 2020 | |
Equity [Abstract] | |
Summary of Common Stock Reserved for Future Issuance | As of September 30, 2020 and December 31, 2019, the Company has reserved the following shares of common stock for future issuance: September 30, 2020 December 31, 2019 Shares reserved for vesting of restricted stock awards — 30,252 Shares reserved for exercise of outstanding stock options 5,658,890 3,414,445 Shares reserved for vesting of restricted stock units 1,342 2,300 Shares reserved for issuance under the 2018 Stock Option and Grant Plan 3,541,042 332,513 Shares reserved for issuance under the 2018 Employee Stock Purchase Plan 774,088 459,595 Shares reserved for issuance under the 2019 Inducement Plan 831,550 1,800,000 Total shares of authorized common stock reserved for future issuance 10,806,912 6,039,105 |
Stock-Based Compensation (Table
Stock-Based Compensation (Tables) | 9 Months Ended |
Sep. 30, 2020 | |
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: Nine Months Ended September 30, 2020 2019 Expected option life (years) 6.00 5.97 Risk-free interest rate 0.84 % 2.40 % Expected volatility 75.85 % 81.25 % Expected dividend yield — % — % |
Summary of Stock Option Activity | The following table summarizes the Company’s stock option activity for the nine months ended September 30, 2020: Number of Options Weighted- Average Exercise Price Weighted- Average Remaining Contractual Term (Years) Aggregate Intrinsic Value Outstanding as of December 31, 2019 3,414,445 $ 12.08 8.43 $ 29,353 Granted 2,485,413 $ 19.82 Exercised (38,761 ) $ 4.05 Cancelled or forfeited (202,207 ) $ 20.07 Outstanding as of September 30, 2020 5,658,890 $ 15.25 8.43 $ 13,481 Exercisable as of September 30, 2020 1,754,340 $ 8.92 7.06 $ 11,057 |
Summary of Restricted Stock Unit Activity | The following table summarizes the Company’s restricted common stock activity for the nine months ended September 30, 2020: Number of Shares Weighted- Average Grant Date Fair Value Issued and unvested as of December 31, 2019 32,552 $ 1.50 Granted — Vested (31,210 ) $ 0.89 Forfeited, cancelled or expired — Issued and unvested as of September 30, 2020 1,342 $ 15.65 |
Schedule of Stock-based Compensation Expense | Stock-based compensation expense was allocated as follows: Three Months Ended September 30, Nine Months Ended September 30, 2020 2019 2020 2019 Research and development $ 2,686 $ 889 $ 6,095 $ 2,649 General and administrative 2,119 908 5,431 2,265 Total stock-based compensation expense $ 4,805 $ 1,797 $ 11,526 $ 4,914 |
Net Loss per Share Attributab_2
Net Loss per Share Attributable to Common Stockholders (Tables) | 9 Months Ended |
Sep. 30, 2020 | |
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: Nine Months Ended September 30, 2020 2019 Options to purchase common stock 5,658,890 3,145,428 Restricted common stock 1,342 63,313 |
Nature of the Business - Additi
Nature of the Business - Additional Information (Detail) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | |||||||
Sep. 30, 2020 | Jun. 30, 2020 | Mar. 31, 2020 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Sep. 30, 2020 | Sep. 30, 2019 | Dec. 31, 2019 | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | |||||||||
Net loss | $ (36,780) | $ (28,828) | $ (25,973) | $ (17,145) | $ (16,055) | $ (17,103) | $ (91,581) | $ (50,303) | |
Accumulated deficit | (236,285) | (236,285) | $ (144,704) | ||||||
Cash and cash equivalents | $ 219,546 | $ 206,362 | $ 219,546 | $ 206,362 | $ 187,043 |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies - Additional Information (Detail) - USD ($) $ in Thousands | Sep. 30, 2020 | Dec. 31, 2019 |
New Accounting Pronouncements Or Change In Accounting Principle [Line Items] | ||
Restricted cash | $ 492 | $ 492 |
Deferred rent | $ 231 | |
ASU No. 2018-18 [Member] | ||
New Accounting Pronouncements Or Change In Accounting Principle [Line Items] | ||
Change in accounting principle, accounting standards update, adopted [true false] | true | |
Change in accounting principle, accounting standards update, adoption date | Jan. 1, 2020 | |
Change in accounting principle, accounting standards update, immaterial effect [true false] | true | |
ASU No. 2018-15 [Member] | ||
New Accounting Pronouncements Or Change In Accounting Principle [Line Items] | ||
Change in accounting principle, accounting standards update, adopted [true false] | true | |
Change in accounting principle, accounting standards update, adoption date | Jan. 1, 2020 | |
Change in accounting principle, accounting standards update, immaterial effect [true false] | true | |
ASU No. 2018-13 [Member] | ||
New Accounting Pronouncements Or Change In Accounting Principle [Line Items] | ||
Change in accounting principle, accounting standards update, adopted [true false] | true | |
Change in accounting principle, accounting standards update, adoption date | Jan. 1, 2020 | |
Change in accounting principle, accounting standards update, immaterial effect [true false] | true |
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 | Sep. 30, 2020 | Dec. 31, 2019 |
Assets Fair Value Disclosure | ||
Assets Fair Value Disclosure | $ 219,306 | $ 186,797 |
Money Market Funds [Member] | ||
Assets Fair Value Disclosure | ||
Assets Fair Value Disclosure | 219,306 | 186,797 |
Level 1 [Member] | ||
Assets Fair Value Disclosure | ||
Assets Fair Value Disclosure | 219,306 | 186,797 |
Level 1 [Member] | Money Market Funds [Member] | ||
Assets Fair Value Disclosure | ||
Assets Fair Value Disclosure | $ 219,306 | $ 186,797 |
Fair Value Measurement - Additi
Fair Value Measurement - Additional Information (Detail) | Sep. 30, 2020USD ($) |
Fair Value Disclosures [Abstract] | |
Fair value input transfer between levels | $ 0 |
Prepaid Expenses and Other Cu_3
Prepaid Expenses and Other Current Assets - Summary of Prepaid Expenses and Other Current Assets (Detail) - USD ($) $ in Thousands | Sep. 30, 2020 | Dec. 31, 2019 |
Prepaid Expense And Other Assets Current [Abstract] | ||
Tax incentive refund | $ 2,366 | $ 1,916 |
Prepaid research and development costs | 3,528 | 4,915 |
Prepaid insurance | 1,762 | 897 |
Prepaid compensation and benefit costs | 621 | 362 |
Other current assets | 1,163 | 568 |
Total | $ 9,440 | $ 8,658 |
Property and Equipment, Net - S
Property and Equipment, Net - Summary of Property and Equipment, Net (Detail) - USD ($) $ in Thousands | Sep. 30, 2020 | Dec. 31, 2019 |
Property, Plant and Equipment [Line Items] | ||
Property plant and equipment, gross | $ 5,444 | $ 4,930 |
Less: Accumulated depreciation and amortization | (2,132) | (1,234) |
Property plant and equipment, net | 3,312 | 3,696 |
Laboratory and Office Equipment [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property plant and equipment, gross | 3,923 | 3,456 |
Leasehold Improvements [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property plant and equipment, gross | 1,378 | 1,340 |
Computer Equipment and Software [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property plant and equipment, gross | $ 143 | $ 134 |
Property and Equipment, Net - A
Property and Equipment, Net - Additional Information (Detail) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2020 | Sep. 30, 2019 | Sep. 30, 2020 | Sep. 30, 2019 | |
Property Plant And Equipment [Abstract] | ||||
Depreciation and amortization expense | $ 314 | $ 215 | $ 898 | $ 605 |
Accrued Expenses and Other Cu_3
Accrued Expenses and Other Current Liabilities - Summary of Accrued Expenses and Other Current Liabilities (Detail) - USD ($) $ in Thousands | Sep. 30, 2020 | Dec. 31, 2019 |
Accrued Liabilities Current [Abstract] | ||
Compensation and benefit costs | $ 5,761 | $ 3,028 |
Research and development costs | 7,540 | 5,794 |
Consulting and professional fees | 1,028 | 917 |
License agreement | 8,000 | |
Other liabilities | 196 | 115 |
Accrued expenses | $ 22,525 | $ 9,854 |
Stockholders' Equity - Addition
Stockholders' Equity - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Thousands | 9 Months Ended | |
Sep. 30, 2020 | Dec. 31, 2019 | |
Equity [Abstract] | ||
Common stock, shares authorized | 150,000,000 | 150,000,000 |
Common stock, par value | $ 0.0001 | $ 0.0001 |
Undesignated preferred stock | 10,000,000 | 10,000,000 |
Undesignated preferred stock outstanding | 0 | 0 |
Cash dividends | $ 0 |
Stockholders' Equity - Summary
Stockholders' Equity - Summary of Common Stock Reserved for Future Issuance (Detail) - shares | Sep. 30, 2020 | Dec. 31, 2019 |
Class of Stock [Line Items] | ||
Total shares of authorized common stock reserved for future issuance | 10,806,912 | 6,039,105 |
Restricted Stock [Member] | ||
Class of Stock [Line Items] | ||
Total shares of authorized common stock reserved for future issuance | 30,252 | |
Employee Stock Option [Member] | ||
Class of Stock [Line Items] | ||
Total shares of authorized common stock reserved for future issuance | 5,658,890 | 3,414,445 |
Restricted Stock Units [Member] | ||
Class of Stock [Line Items] | ||
Total shares of authorized common stock reserved for future issuance | 1,342 | 2,300 |
2018 Stock Option and Incentive Plan [Member] | ||
Class of Stock [Line Items] | ||
Total shares of authorized common stock reserved for future issuance | 3,541,042 | 332,513 |
2018 Employee Stock Purchase Plan [Member] | ||
Class of Stock [Line Items] | ||
Total shares of authorized common stock reserved for future issuance | 774,088 | 459,595 |
2019 Inducement Plan [Member] | ||
Class of Stock [Line Items] | ||
Total shares of authorized common stock reserved for future issuance | 831,550 | 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 of Directors (Detail) | 9 Months Ended | |
Sep. 30, 2020 | Sep. 30, 2019 | |
Share Based Compensation Arrangement By Share Based Payment Award Fair Value Assumptions And Methodology [Abstract] | ||
Expected option life (years) | 6 years | 5 years 11 months 19 days |
Risk-free interest rate | 0.84% | 2.40% |
Expected volatility | 75.85% | 81.25% |
Stock-Based Compensation - Su_2
Stock-Based Compensation - Summary of Stock Option Activity (Detail) $ / shares in Units, $ in Thousands | 9 Months Ended | 12 Months Ended |
Sep. 30, 2020USD ($)$ / sharesshares | Dec. 31, 2019USD ($)$ / sharesshares | |
Share Based Compensation Arrangement By Share Based Payment Award Options Outstanding Roll Forward | ||
Number of options, Outstanding beginning balance | shares | 3,414,445 | |
Number of options, Granted | shares | 2,485,413 | |
Number of options, Exercised | shares | (38,761) | |
Number of options, Cancelled or forfeited | shares | (202,207) | |
Number of options, Outstanding ending balance | shares | 5,658,890 | 3,414,445 |
Number of options, Exercisable | shares | 1,754,340 | |
Weighted average exercise price, Outstanding beginning balance | $ / shares | $ 12.08 | |
Weighted average exercise price, Granted | $ / shares | 19.82 | |
Weighted average exercise price, Exercised | $ / shares | 4.05 | |
Weighted average exercise price, Cancelled or forfeited | $ / shares | 20.07 | |
Weighted average exercise price, Outstanding ending balance | $ / shares | 15.25 | $ 12.08 |
Weighted average exercise price, Exercisable | $ / shares | $ 8.92 | |
Weighted average remaining contractual term, Outstanding balance | 8 years 5 months 4 days | 8 years 5 months 4 days |
Weighted average remaining contractual term, Exercisable | 7 years 21 days | |
Aggregate intrinsic value, Outstanding balance | $ | $ 13,481 | $ 29,353 |
Aggregate intrinsic value, Exercisable | $ | $ 11,057 |
Stock-Based Compensation - Addi
Stock-Based Compensation - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 9 Months Ended | |
Sep. 30, 2020 | Sep. 30, 2020 | Sep. 30, 2019 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |||
Aggregate intrinsic value of options exercised | $ 595 | $ 3,446 | |
Options Granted, Weighted-average grant-date fair value | $ 12.98 | $ 11.77 | |
Total fair value of restricted common stock vested | $ 28 | $ 44 | |
Unrecognized stock-based compensation expenses | $ 41,143 | $ 41,143 | |
Unrecognized stock-based compensation expense, period for recognition | 3 years 29 days | ||
Additional research and development expenses | $ 274 | $ 274 |
Stock-Based Compensation - Su_3
Stock-Based Compensation - Summary of Restricted Stock Unit Activity (Detail) | 9 Months Ended |
Sep. 30, 2020$ / sharesshares | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Number of shares, Issued and unvested beginning balance | shares | 32,552 |
Number of shares, Vested | shares | (31,210) |
Number of shares, Issued and unvested ending balance | shares | 1,342 |
Weighted average grant date fair value, Issued and unvested, beginning balance | $ / shares | $ 1.50 |
Weighted average grant date fair value, Vested | $ / shares | 0.89 |
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 | 9 Months Ended | ||
Sep. 30, 2020 | Sep. 30, 2019 | Sep. 30, 2020 | Sep. 30, 2019 | |
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||||
Total stock-based compensation expense | $ 4,805 | $ 1,797 | $ 11,526 | $ 4,914 |
Research and Development Expense [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||||
Total stock-based compensation expense | 2,686 | 889 | 6,095 | 2,649 |
General and Administrative Expense [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||||
Total stock-based compensation expense | $ 2,119 | $ 908 | $ 5,431 | $ 2,265 |
License Agreements - Additional
License Agreements - Additional Information (Detail) $ in Thousands | Oct. 02, 2017USD ($) | Nov. 17, 2016USD ($) | Jan. 27, 2016USD ($) | Jan. 27, 2016CAD ($)shares | Nov. 30, 2019USD ($) | Aug. 31, 2017USD ($)shares | Sep. 30, 2020USD ($)shares | Sep. 30, 2019USD ($) | Sep. 30, 2020USD ($)shares | Sep. 30, 2020GBP (£) | Sep. 30, 2019USD ($) | Dec. 31, 2019shares |
License Agreement [Line Items] | ||||||||||||
Accrued upfront license | $ 0 | $ 0 | ||||||||||
Common stock, shares issued | shares | 36,449,000 | 36,449,000 | 31,643,000 | |||||||||
Research and development | $ 28,509,000 | $ 13,042,000 | $ 67,649,000 | $ 37,755,000 | ||||||||
Preferred stock issued | shares | 0 | 0 | 0 | |||||||||
University of Manchester Agreement [Member] | MPSII License Agreement [Member] | ||||||||||||
License Agreement [Line Items] | ||||||||||||
Upfront license fee | $ 8,000 | $ 8,000 | ||||||||||
Milestone payments | 80,000 | |||||||||||
Milestone payment paid | 2,000 | |||||||||||
Milestone payment due | $ 4,000 | |||||||||||
Agreement Expiration Period | 15 years | 15 years | ||||||||||
Agreement Expiration Description | upon the later of 15 years from the effective date or the expiration of the last valid claim of the licensed patents | upon the later of 15 years from the effective date or the expiration of the last valid claim of the licensed patents | ||||||||||
Research and development expense | £ | £ 9,100 | |||||||||||
UHN Agreement [Member] | ||||||||||||
License Agreement [Line Items] | ||||||||||||
Research and development expense | 65,000 | 415,000 | $ 174,000 | 741,000 | ||||||||
Milestone fees | 0 | 0 | ||||||||||
Annual maintenance 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, shares issued | shares | 1,161,665,000 | |||||||||||
Fair value of shares issued | $ 480,000 | |||||||||||
Payments upon closing of an initial public offering | $ 2,000,000 | |||||||||||
Research and development | $ 0 | $ 0 | 38,000 | 38,000 | ||||||||
BioMarin Pharmaceutical Inc [Member] | ||||||||||||
License Agreement [Line Items] | ||||||||||||
Upfront license fee | $ 500,000 | |||||||||||
Milestone payments | 13,000,000 | |||||||||||
Preferred stock issued | shares | 233,765,000 | |||||||||||
Expenses related to license | 0 | 0 | ||||||||||
GenStem Therapeutics Inc [Member] | ||||||||||||
License Agreement [Line Items] | ||||||||||||
Upfront license fee | $ 1,000,000 | |||||||||||
Milestone payment paid | $ 2,000,000 | |||||||||||
Milestone payment due | 2,000,000 | |||||||||||
Expenses related to license | 0 | 0 | ||||||||||
Milestone payments | $ 16,000,000 | |||||||||||
Lund University Rights Holders Agreement [Member] | ||||||||||||
License Agreement [Line Items] | ||||||||||||
Expenses related to license | $ 0 | $ 0 | ||||||||||
Milestone payments | $ 550,000 |
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 | 9 Months Ended | |
Sep. 30, 2020 | Sep. 30, 2019 | |
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 | 5,658,890 | 3,145,428 |
Restricted Stock [Member] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Antidilutive securities excluded from computation of earnings per share amount | 1,342 | 63,313 |
Commitments and Contingencies -
Commitments and Contingencies - Additional Information (Detail) - CAD ($) $ in Thousands | Jun. 01, 2020 | Sep. 30, 2020 |
Commitments And Contingencies Disclosure [Line Items] | ||
Lease agreement expiration month and year | 2025-06 | |
Annual lease payments, description | The annual lease payments are fixed for years 1 and 2, and then subject to a 6.67% increase for years 2 through 5 | |
Percentage of annual increase in rent | 6.67% | |
Increase in lease obligations | $ 865 | |
Other Long-term Assets [Member] | ||
Commitments And Contingencies Disclosure [Line Items] | ||
Security deposit in connection with lease | $ 27 |
Related Party Transactions - Ad
Related Party Transactions - Additional information (Detail) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2020 | Sep. 30, 2019 | Sep. 30, 2020 | Sep. 30, 2019 | |
Related Party Transaction [Line Items] | ||||
Research and development expense | $ 28,509 | $ 13,042 | $ 67,649 | $ 37,755 |
License Agreement [Member] | ||||
Related Party Transaction [Line Items] | ||||
Research and development expense | 65 | 415 | 212 | 779 |
Sub-lease Agreement [Member] | Officers and Board Members [Member] | ||||
Related Party Transaction [Line Items] | ||||
Expense related to related party | $ 355 | $ 369 | $ 1,081 | $ 1,090 |