Document and Entity Information
Document and Entity Information - shares | 9 Months Ended | |
Sep. 30, 2021 | Oct. 28, 2021 | |
Document and Entity Information | ||
Document Type | 10-Q | |
Document Quarterly Report | true | |
Document Transition Report | false | |
Document Period End Date | Sep. 30, 2021 | |
Entity File Number | 001-37625 | |
Entity Registrant Name | Voyager Therapeutics, Inc. | |
Entity Incorporation, State or Country Code | DE | |
Entity Tax Identification Number | 46-3003182 | |
Entity Address, Address Line One | 75 Sidney Street | |
Entity Address, City or Town | Cambridge | |
Entity Address, State or Province | MA | |
Entity Address, Postal Zip Code | 02139 | |
City Area Code | 857 | |
Local Phone Number | 259-5340 | |
Title of 12(b) Security | Common Stock, $0.001 par value | |
Trading Symbol | VYGR | |
Security Exchange Name | NASDAQ | |
Entity Current Reporting Status | Yes | |
Entity Interactive Data Current | Yes | |
Entity Filer Category | Accelerated Filer | |
Entity Small Business | false | |
Entity Emerging Growth Company | false | |
Entity Shell Company | false | |
Entity Central Index Key | 0001640266 | |
Amendment Flag | false | |
Current Fiscal Year End Date | --12-31 | |
Document Fiscal Year Focus | 2021 | |
Document Fiscal Period Focus | Q3 | |
Entity Common Stock, Shares Outstanding | 37,957,396 |
Condensed Consolidated Balance
Condensed Consolidated Balance Sheets - USD ($) $ in Thousands | Sep. 30, 2021 | Dec. 31, 2020 |
Current assets: | ||
Cash and cash equivalents | $ 111,475 | $ 104,440 |
Marketable securities, current | 10,024 | 76,698 |
Related party collaboration receivable | 1,165 | 8,012 |
Prepaid expenses and other current assets | 3,540 | 8,619 |
Total current assets | 126,204 | 197,769 |
Property and equipment, net | 22,992 | 25,435 |
Deposits and other non-current assets | 1,779 | 2,316 |
Operating lease, right-of-use asset | 33,799 | 36,064 |
Total assets | 184,774 | 261,584 |
Current liabilities: | ||
Accounts payable | 592 | 634 |
Accrued expenses | 11,324 | 14,205 |
Other current liabilities | 5,248 | 4,198 |
Deferred revenue, current | 9,537 | 7,729 |
Total current liabilities | 26,701 | 26,766 |
Deferred revenue, non-current | 29,639 | 36,088 |
Other non-current liabilities | 41,012 | 44,410 |
Total liabilities | 97,352 | 107,264 |
Commitments and contingencies (see note 8) | ||
Stockholders' equity: | ||
Preferred stock $0.001 par value: 5,000,000 shares authorized at September 30, 2021 and December 31, 2020; no shares issued and outstanding at September 30, 2021 and December 31, 2020 | ||
Common stock, $0.001 par value: 120,000,000 shares authorized at September 30, 2021 and December 31, 2020; 37,781,786 and 37,368,027 shares issued and outstanding at September 30, 2021 and December 31, 2020, respectively | 38 | 37 |
Additional paid-in capital | 440,325 | 430,324 |
Accumulated other comprehensive loss | (128) | (134) |
Accumulated deficit | (352,813) | (275,907) |
Total stockholders' equity | 87,422 | 154,320 |
Total liabilities and stockholders' equity | $ 184,774 | $ 261,584 |
Condensed Consolidated Balanc_2
Condensed Consolidated Balance Sheets (Parenthetical) - $ / shares | Sep. 30, 2021 | Dec. 31, 2020 |
Condensed Consolidated Balance Sheets | ||
Preferred Stock, par value (in dollars per share) | $ 0.001 | $ 0.001 |
Preferred Stock, Shares Authorized | 5,000,000 | 5,000,000 |
Preferred Stock, Shares Issued | 0 | 0 |
Preferred Stock, Shares Outstanding | 0 | 0 |
Common stock, par value (in dollars per share) | $ 0.001 | $ 0.001 |
Common Stock, Shares Authorized | 120,000,000 | 120,000,000 |
Common Stock, Shares, Issued | 37,781,786 | 37,368,027 |
Common Stock, Shares, Outstanding | 37,781,786 | 37,368,027 |
Condensed Consolidated Statemen
Condensed Consolidated Statements of Operations and Comprehensive Income (Loss) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2021 | Sep. 30, 2020 | Sep. 30, 2021 | Sep. 30, 2020 | |
Condensed Consolidated Statements of Operations and Comprehensive Income (Loss) | ||||
Collaboration revenue | $ 1,482 | $ 117,843 | $ 9,342 | $ 164,591 |
Operating expenses: | ||||
Research and development | 17,914 | 25,039 | 59,767 | 86,757 |
General and administrative | 8,714 | 8,277 | 28,895 | 26,721 |
Total operating expenses | 26,628 | 33,316 | 88,662 | 113,478 |
Operating (loss) income | (25,146) | 84,527 | (79,320) | 51,113 |
Other income (expense): | ||||
Interest income | 121 | 254 | 253 | 1,578 |
Other (expense) income | (112) | 830 | 2,161 | (24) |
Total other income, net | 9 | 1,084 | 2,414 | 1,554 |
Net (loss) income | (25,137) | 85,611 | (76,906) | 52,667 |
Other comprehensive (loss) income | ||||
Net unrealized (loss) gain on available-for-sale securities | (2) | (205) | 6 | 44 |
Total other comprehensive (loss) income | (2) | (205) | 6 | 44 |
Comprehensive (loss) income | $ (25,139) | $ 85,406 | $ (76,900) | $ 52,711 |
Net (loss) income per share, basic | $ (0.67) | $ 2.30 | $ (2.04) | $ 1.42 |
Net (loss) income per share, diluted | $ (0.67) | $ 2.27 | $ (2.04) | $ 1.40 |
Weighted-average common shares outstanding, basic | 37,780,547 | 37,242,504 | 37,623,309 | 37,079,242 |
Weighted-average common shares outstanding, diluted | 37,780,547 | 37,672,328 | 37,623,309 | 37,500,155 |
Condensed Consolidated Statem_2
Condensed Consolidated Statements of Stockholders' Equity - USD ($) $ in Thousands | Common Stock | Additional Paid-in Capital | Accumulated Other Income (Loss) | Accumulated Deficit | Total |
Stockholders' Equity, Beginning Balance at Dec. 31, 2019 | $ 37 | $ 412,227 | $ (104) | $ (312,648) | $ 99,512 |
Shares, Outstanding, Beginning Balance at Dec. 31, 2019 | 36,865,116 | ||||
Exercises of vested stock options | 34 | 34 | |||
Exercises of vested stock options (in shares) | 3,035 | ||||
Vesting of restricted stock units (in shares) | 108,600 | ||||
Stock based compensation expense | 3,949 | 3,949 | |||
Unrealized gain (loss) on available for-sale securities, net of tax | 525 | 525 | |||
Net (loss) income | (24,263) | (24,263) | |||
Stockholders' Equity, Ending Balance at Mar. 31, 2020 | $ 37 | 416,210 | 421 | (336,911) | 79,757 |
Shares, Outstanding, Ending Balance at Mar. 31, 2020 | 36,976,751 | ||||
Stockholders' Equity, Beginning Balance at Dec. 31, 2019 | $ 37 | 412,227 | (104) | (312,648) | 99,512 |
Shares, Outstanding, Beginning Balance at Dec. 31, 2019 | 36,865,116 | ||||
Unrealized gain (loss) on available for-sale securities, net of tax | 44 | ||||
Net (loss) income | 52,667 | ||||
Stockholders' Equity, Ending Balance at Sep. 30, 2020 | $ 37 | 426,240 | (60) | (259,981) | 166,236 |
Shares, Outstanding, Ending Balance at Sep. 30, 2020 | 37,262,703 | ||||
Stockholders' Equity, Beginning Balance at Mar. 31, 2020 | $ 37 | 416,210 | 421 | (336,911) | 79,757 |
Shares, Outstanding, Beginning Balance at Mar. 31, 2020 | 36,976,751 | ||||
Exercises of vested stock options | 1,651 | 1,651 | |||
Exercises of vested stock options (in shares) | 160,478 | ||||
Vesting of restricted stock units (in shares) | 21,403 | ||||
Issuance of common stock under ESPP | 638 | 638 | |||
Issuance of common stock under ESPP (in shares) | 44,995 | ||||
Stock based compensation expense | 3,795 | 3,795 | |||
Unrealized gain (loss) on available for-sale securities, net of tax | (276) | (276) | |||
Net (loss) income | (8,681) | (8,681) | |||
Stockholders' Equity, Ending Balance at Jun. 30, 2020 | $ 37 | 422,294 | 145 | (345,592) | 76,884 |
Shares, Outstanding, Ending Balance at Jun. 30, 2020 | 37,203,627 | ||||
Exercises of vested stock options | 510 | 510 | |||
Exercises of vested stock options (in shares) | 50,774 | ||||
Vesting of restricted stock units (in shares) | 8,302 | ||||
Stock based compensation expense | 3,436 | 3,436 | |||
Unrealized gain (loss) on available for-sale securities, net of tax | (205) | (205) | |||
Net (loss) income | 85,611 | 85,611 | |||
Stockholders' Equity, Ending Balance at Sep. 30, 2020 | $ 37 | 426,240 | (60) | (259,981) | 166,236 |
Shares, Outstanding, Ending Balance at Sep. 30, 2020 | 37,262,703 | ||||
Stockholders' Equity, Beginning Balance at Dec. 31, 2020 | $ 37 | 430,324 | (134) | (275,907) | 154,320 |
Shares, Outstanding, Beginning Balance at Dec. 31, 2020 | 37,368,027 | ||||
Exercises of vested stock options | $ 1 | 27 | 28 | ||
Exercises of vested stock options (in shares) | 3,811 | ||||
Vesting of restricted stock units (in shares) | 184,217 | ||||
Stock based compensation expense | 3,498 | 3,498 | |||
Unrealized gain (loss) on available for-sale securities, net of tax | 11 | 11 | |||
Net (loss) income | (21,649) | (21,649) | |||
Stockholders' Equity, Ending Balance at Mar. 31, 2021 | $ 38 | 433,849 | (123) | (297,556) | 136,208 |
Shares, Outstanding, Ending Balance at Mar. 31, 2021 | 37,556,055 | ||||
Stockholders' Equity, Beginning Balance at Dec. 31, 2020 | $ 37 | 430,324 | (134) | (275,907) | $ 154,320 |
Shares, Outstanding, Beginning Balance at Dec. 31, 2020 | 37,368,027 | ||||
Exercises of vested stock options (in shares) | 3,811 | ||||
Unrealized gain (loss) on available for-sale securities, net of tax | $ 6 | ||||
Net (loss) income | (76,906) | ||||
Stockholders' Equity, Ending Balance at Sep. 30, 2021 | $ 38 | 440,325 | (128) | (352,813) | 87,422 |
Shares, Outstanding, Ending Balance at Sep. 30, 2021 | 37,781,786 | ||||
Stockholders' Equity, Beginning Balance at Mar. 31, 2021 | $ 38 | 433,849 | (123) | (297,556) | 136,208 |
Shares, Outstanding, Beginning Balance at Mar. 31, 2021 | 37,556,055 | ||||
Vesting of restricted stock units (in shares) | 114,412 | ||||
Issuance of common stock under ESPP | 580 | 580 | |||
Issuance of common stock under ESPP (in shares) | 101,752 | ||||
Stock based compensation expense | 3,871 | 3,871 | |||
Unrealized gain (loss) on available for-sale securities, net of tax | (3) | (3) | |||
Net (loss) income | (30,120) | (30,120) | |||
Stockholders' Equity, Ending Balance at Jun. 30, 2021 | $ 38 | 438,300 | (126) | (327,676) | 110,536 |
Shares, Outstanding, Ending Balance at Jun. 30, 2021 | 37,772,219 | ||||
Vesting of restricted stock units (in shares) | 9,567 | ||||
Stock based compensation expense | 2,025 | 2,025 | |||
Unrealized gain (loss) on available for-sale securities, net of tax | (2) | (2) | |||
Net (loss) income | (25,137) | (25,137) | |||
Stockholders' Equity, Ending Balance at Sep. 30, 2021 | $ 38 | $ 440,325 | $ (128) | $ (352,813) | $ 87,422 |
Shares, Outstanding, Ending Balance at Sep. 30, 2021 | 37,781,786 |
Condensed Consolidated Statem_3
Condensed Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2021 | Sep. 30, 2020 | |
Cash flow from operating activities | ||
Net (loss) income | $ (76,906) | $ 52,667 |
Adjustments to reconcile net loss (income) to net cash used in operating activities: | ||
Stock-based compensation expense | 9,670 | 11,432 |
Depreciation | 3,683 | 2,830 |
Amortization of premiums and discounts on marketable securities | 324 | (57) |
Other non-cash items | (2,460) | 24 |
Changes in operating assets and liabilities: | ||
Related party collaboration receivable | 6,847 | 5,807 |
Prepaid expenses and other current assets | 1,731 | 1,379 |
Operating lease, right-of-use asset | 2,265 | 2,341 |
Other non-current assets | 69 | 207 |
Accounts payable | (42) | (684) |
Accrued expenses | (2,912) | (3,440) |
Operating lease liabilities | (2,348) | (2,370) |
Lease incentive benefit | 3,947 | |
Deferred revenue | (4,641) | (148,822) |
Net cash used in operating activities | (64,720) | (74,739) |
Cash flow from investing activities | ||
Purchases of property and equipment | (1,262) | (8,541) |
Proceeds from sale of equipment | 31 | |
Purchases of marketable securities | (24,979) | |
Proceeds from sales and maturities of marketable securities | 72,632 | 168,000 |
Net cash provided by investing activities | 71,370 | 134,511 |
Cash flow from financing activities | ||
Proceeds from the exercise of stock options | 28 | 2,195 |
Proceeds from the purchase of common stock under ESPP | 357 | 482 |
Net cash provided by financing activities | 385 | 2,677 |
Net increase in cash, cash equivalents, and restricted cash | 7,035 | 62,449 |
Cash, cash equivalents, and restricted cash beginning of period | 106,219 | 86,777 |
Cash, cash equivalents, and restricted cash end of period | 113,254 | 149,226 |
Supplemental disclosure of cash and non-cash activities | ||
Capital expenditures incurred but not yet paid | $ 22 | $ 454 |
Nature of business
Nature of business | 9 Months Ended |
Sep. 30, 2021 | |
Nature of business | |
Nature of business | 1. Nature of business Voyager Therapeutics, Inc. (the “Company”) is a gene therapy company focused on developing life-changing treatments and next-generation platform technologies. The Company is focused on diseases where the Company believes a single dose adeno-associated virus (“AAV”) gene therapy approach that either increases or decreases the production of a specific protein can either halt or slow disease progression or reduce the symptom severity, therefore providing clinically meaningful impact to patients. The Company’s gene therapy platforms enable it to engineer, optimize, manufacture and deliver its AAV-based gene therapies that the Company believes have the potential to safely provide durable efficacy following a single administration. The Company’s team of experts in the fields of AAV gene therapy and neuroscience first identifies and selects diseases with target tissues that have tropism for AAV gene therapy. The Company then engineers and optimizes AAV vectors for delivery of the virus payload to the targeted tissue or cells. The Company believes its single dose gene therapies have the potential to be delivered directly, with targeted infusions or systemically, in conjunction with its novel capsids. The Company is identifying novel AAV capsids, outer viral protein shells that enclose genetic material of a virus payload. The Company’s team has developed a proprietary system called TRACER TM in vivo evolution of AAV capsids with cell-specific transduction properties in wild-type animals. The Company is also applying the TRACER system towards further capsid variant libraries and selection for tropism and transduction in additional cell and tissue types, such as cardiac and skeletal muscle. The Company’s quality and manufacturing processes employ an established system capable of enabling production of high quality AAV vectors at clinical scale. In addition to the Company’s capsid screening platform, the Company has developed a vectorized antibody platform which the Company believes will overcome many of the challenges of passive immunization. The Company’s business strategy focuses on discovering, developing, manufacturing and commercializing its gene therapy programs. As part of this strategy, the Company has developed core competencies specific to AAV gene therapy development and manufacturing. This business strategy also includes business development activities that may include in-licensing activities or partnering certain programs in certain geographies with collaborators, as the Company has demonstrated through its ongoing collaboration with Neurocrine Biosciences, Inc. (“Neurocrine”) under a collaboration agreement that became effective in January 2019 (the “Neurocrine Collaboration Agreement”), or out-licensing activities including license agreements related to the Company’s AAV capsids The Company has a history of incurring annual net operating losses. As of September 30, 2021, the Company had an accumulated deficit of $352.8 million. The Company has not generated any product revenue and has financed its operations primarily through public offerings and private placements of its equity securities and funding from its prior collaborations with Sanofi Genzyme Corporation (“Sanofi Genzyme”) and AbbVie Biotechnology Ltd and AbbVie Ireland Unlimited Company (collectively, “AbbVie”) and its ongoing collaboration with Neurocrine, and, in October 2021, its licensing agreement with Pfizer Inc. (“Pfizer”). Through September 30, 2021, the Company has raised approximately $640.0 million of proceeds from sales of convertible preferred stock and common stock, including its initial public offering and follow-on public offering, and proceeds from collaboration agreements. As of September 30, 2021, the Company had cash, cash equivalents, and marketable debt securities of $121.5 million. Based upon its current operating plan, the Company expects that its existing cash, cash equivalents, and marketable debt securities will be sufficient to meet the Company’s planned operating expenses and capital expenditure re quirements There can be no assurance that the Company will be able to obtain additional debt or equity financing or generate product revenue or revenue from collaboration partners on terms acceptable to the Company, on a timely basis or at all. The failure of the Company to obtain sufficient funds on acceptable terms when needed could have a material adverse effect on the Company’s business, results of operations, and financial condition. |
Summary of significant accounti
Summary of significant accounting policies | 9 Months Ended |
Sep. 30, 2021 | |
Summary of significant accounting policies | |
Summary of significant accounting policies | 2. Summary of significant accounting policies and basis of presentation Basis of Presentation The accompanying unaudited condensed consolidated financial statements of the Company have been prepared in accordance with accounting principles generally accepted in the United States (“GAAP”) for interim financial reporting and as required by Regulation S-X, Rule 10-01. Accordingly, they do not include all of the information and footnotes required by GAAP for complete financial statements. For further information, refer to the consolidated financial statements and footnotes included in the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2020 as filed with the Securities and Exchange Commission (“SEC”) on February 25, 2021. These interim condensed consolidated financial statements, in the opinion of management, reflect all normal recurring adjustments necessary for a fair presentation of the Company’s financial position and results of operations for the periods presented. 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 Updates (“ASU”) of the Financial Accounting Standards Board (“FASB”). Principles of Consolidation The unaudited interim consolidated financial statements include the accounts of the Company and its wholly owned subsidiary as disclosed in Note 2, under the headings “Summary of Significant Accounting Policies” and “Basis of Presentation”, within the “Notes to Consolidated Financial Statements” accompanying the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2020. Intercompany accounts and transactions have been eliminated. Use of Estimates The preparation of consolidated financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the amounts reported in the consolidated financial statements and accompanying notes. On an ongoing basis, the Company’s management evaluates its estimates, which include, but are not limited to, estimates related to revenue recognition, accrued expenses, stock-based compensation expense, and income taxes. The Company bases its estimates on historical experience and other market specific or other relevant assumptions that it believes to be reasonable under the circumstances. Actual results may differ from those estimates or assumptions. Certain reclassifications have been made to prior periods to conform to current period presentation. Summary of Significant Accounting Policies There have been no changes in the Company's significant accounting policies as described in Note 2, “Summary of Significant Accounting Policies,” within the “Notes to Consolidated Financial Statements” accompanying the Company’s Annual Report on Form 10-K for the year ended December 31, 2020. |
Fair value measurements
Fair value measurements | 9 Months Ended |
Sep. 30, 2021 | |
Fair value measurements | |
Fair value measurements | 3. Fair value measurements Assets and liabilities measured at fair value on a recurring basis as of September 30, 2021 and December 31, 2020 are as follows: Quoted Prices Significant in Active Other Significant Markets for Observable Unobservable Identical Assets Inputs Inputs Assets Total (Level 1) (Level 2) (Level 3) September 30, 2021 (in thousands) Money market funds included in cash and cash equivalents $ 105,359 $ 105,359 $ — $ — Marketable securities - U.S. Treasury notes 10,024 10,024 — — Total $ 115,383 $ 115,383 $ — $ — December 31, 2020 Money market funds included in cash and cash equivalents $ 103,992 $ 103,992 $ — $ — Marketable securities: U.S. Treasury notes 70,342 70,342 — — Equity securities 6,356 6,356 — — Total marketable securities $ 76,698 $ 76,698 $ — $ — Warrants to purchase equity securities 3,816 — 3,816 — Total $ 184,506 $ 180,690 $ 3,816 $ — The Company measures the fair value of money market funds, U.S. Treasury notes and equity securities based on quoted prices in active markets for identical securities. The Level 2 equity securities included warrants used to purchase equity securities that were valued using the Black-Scholes model. The Black-Scholes option pricing model requires inputs based on certain subjective assumptions, including (a) the expected stock price volatility, (b) the calculation of expected term of the awards, (c) the risk-free interest rate, and (d) expected dividends. All warrants were exercised, and the shares of common stock received following exercise were subsequently sold, by the Company during the three months ended June 30, 2021. |
Cash, cash equivalents, restric
Cash, cash equivalents, restricted cash, and available-for-sale marketable securities | 9 Months Ended |
Sep. 30, 2021 | |
Cash, cash equivalents, restricted cash, and available-for-sale marketable securities | |
Cash, cash equivalents, restricted cash, and available-for-sale marketable securities | 4. Cash, cash equivalents, restricted cash, and available-for-sale marketable securities Cash, cash equivalents, and marketable securities included the following at September 30, 2021 and December 31, 2020: Amortized Unrealized Unrealized Fair Cost Gains Losses Value (in thousands) As of September 30, 2021 Money market funds included in cash and cash equivalents $ 105,359 $ — $ — $ 105,359 Marketable securities - U.S. Treasury notes 10,024 — 10,024 Total money market funds and marketable securities $ 115,383 $ — $ — $ 115,383 As of December 31, 2020 Money market funds included in cash and cash equivalents $ 103,992 $ — $ — $ 103,992 Marketable securities: U.S. Treasury notes 70,348 — 6 70,342 Equity securities 1,220 5,136 — 6,356 Total marketable securities $ 71,568 $ 5,136 $ 6 $ 76,698 Total money market funds and marketable securities $ 175,560 $ 5,136 $ 6 $ 180,690 All of the Company’s marketable debt securities as of September 30, 2021, have a contractual maturity of one year or less. The following table provides a reconciliation of cash, cash equivalents, and restricted cash within the condensed consolidated balance sheets that sum to the total of the same such amounts shown in the condensed consolidated statements of cash flows: As of September 30, As of December 31, 2021 2020 (in thousands) Cash and cash equivalents $ 111,475 $ 104,440 Restricted cash included in deposits and other non-current assets 1,779 1,779 Total cash, cash equivalents, and restricted cash $ 113,254 $ 106,219 |
Accrued expenses
Accrued expenses | 9 Months Ended |
Sep. 30, 2021 | |
Accrued expenses | |
Accrued expenses | 5. Accrued expenses Accrued expenses as of September 30, 2021 and December 31, 2020 consist of the following: As of September 30, As of December 31, 2021 2020 (in thousands) Employee compensation costs $ 5,862 $ 5,857 Research and development costs 4,068 6,624 Accrued goods and services 326 496 Professional services 1,068 1,228 Total $ 11,324 $ 14,205 |
Lease obligation
Lease obligation | 9 Months Ended |
Sep. 30, 2021 | |
Lease obligation | |
Lease obligation | 6. Lease obligation Operating Leases As of September 30, 2021, the Company has leases for office and lab space at 75 and 64 Sidney Street in Cambridge, Massachusetts through November 30, 2026. In March 2020, the Company entered into an agreement to lease additional laboratory and office space at 75 Hayden Avenue in Lexington, Massachusetts through January 31, 2031. The Company gained control of and occupied the space in November 2020. In September 2021, the Company entered into an agreement with BioNTech US, Inc. to sublease part of the office and lab space leased by the Company at 75 Sidney Street in Cambridge, Massachusetts (the “Sublease Agreement”). The sublease term is for approximately 3.3 years and the Company expects to receive $8.5 million from the sublessee over the term of the sublease. The sublease did not relieve the Company of its original obligation under the lease, and therefore the Company did not adjust the operating lease right-of-use asset as a result of the sublease and accounted for the sublease as a separate lease. Sublease payments received are classified within operating expenses to offset the related operating lease payments. The Company received leasehold improvement incentives from the landlord totaling $5.3 million for the 75 Sidney Street and 64 Sidney Street leases and $5.6 million of leasehold improvement incentives from the landlord for the 75 Hayden Avenue lease. The leasehold improvements have been capitalized as fixed assets and the Company recorded the incentives as a component of its right-of-use assets and is amortizing them as a reduction of lease expense over the respective lease terms. The Company’s lease agreements require the Company to maintain a cash deposit or irrevocable letter of credit in the aggregate amount of $1.8 million payable to its landlords as security for the performance of its obligations under the leases. These amounts are recorded as restricted cash and included in deposits and other non-current assets in the accompanying condensed consolidated balance sheets. The following table summarizes the Company’s significant contractual obligations under operating leases as of payment due date by period as of September 30, 2021: Total Minimum Lease Payments (in thousands) 2021 (remainder of year) 2,117 2022 8,698 2023 8,958 2024 9,227 2025 9,644 Thereafter 19,945 Total future minimum lease payments $ 58,589 Less: imputed interest (13,330) Total lease liability $ 45,259 Reported as: Other current liabilities $ 5,248 Other non-current liabilities 40,011 Total lease liability $ 45,259 During the three and nine months ended September 30, 2021, the Company incurred lease expense of $1.7 million and $5.4 million, respectively, for operating leases. During the three and nine months ended September 30, 2020, the Company incurred lease expense of $1.6 million and $5.0 million, respectively, for operating leases. As of September 30, 2021, the weighted average remaining lease term was 6.2 years and the weighted average incremental borrowing rate used to determine the operating lease liability was 7.9%. The following table summarizes the operating sublease income generated under the Sublease Agreement for the three and nine months ended September 30, 2021 and 2020: Three Months Ended Nine Months Ended September 30, September 30, 2021 2020 2021 2020 Operating sublease income (in thousands) $ 204 $ — $ 204 $ — |
Other liabilities
Other liabilities | 9 Months Ended |
Sep. 30, 2021 | |
Other liabilities | |
Other liabilities | 7. Other liabilities As of September 30, 2021 and December 31, 2020, other current and non-current liabilities consisted of the following: As of September 30, As of December 31, 2021 2020 (in thousands) Other current liabilities Lease liability 5,248 4,198 Total other current liabilities $ 5,248 $ 4,198 Other non-current liabilities Lease liability $ 40,011 $ 43,409 Other 1,001 1,001 Total other non-current liabilities $ 41,012 $ 44,410 Strategic Restructuring On August 6, 2021, the board of directors of the Company approved a strategic restructuring plan to eliminate a portion of its workforce as part of an initiative to reduce expenses and enhance operations. The strategic restructuring plan was approved in connection with its portfolio reevaluation efforts and its strategic shift to invest additional resources in the Company’s novel capsid development efforts. During the three and nine months ended September 30, 2021, the Company incurred restructuring costs of approximately $2.0 million, which consists of severance-related costs. Approximately $0.9 million of these restructuring costs were paid as of September 30, 2021. |
Commitments and contingencies
Commitments and contingencies | 9 Months Ended |
Sep. 30, 2021 | |
Commitments and contingencies | |
Commitments and contingencies | 8. Commitments and contingencies Significant Agreements Neurocrine Collaboration Agreement Summary of Agreement In January 2019, the Company signed the Neurocrine Collaboration Agreement for the research, development and commercialization of certain of its AAV gene therapy products. The Neurocrine Collaboration Agreement became effective in March 2019. Under the Neurocrine Collaboration Agreement, the Company agreed to collaborate on the conduct of four collaboration programs (the “Neurocrine Programs”) which include: (i) the VY-AADC (NBIb-1817) for Parkinson’s disease (the “VY-AADC Program”), (ii) the VY-FXN01 for Friedreich’s ataxia (the “FA Program”) (collectively with the VY-AADC Program, the “Legacy Programs”), and (iii) two programs to be determined by the Company and Neurocrine at a later date (the “Discovery Programs”) In June 2019, in conjunction with the termination of the collaboration agreement with Sanofi Genzyme (the “Sanofi Genzyme Collaboration Agreement”), the Company gained ex-U.S. rights to the FA Program . The Company’s ex-U.S. rights to the FA Program were subsequently transferred to Neurocrine under the terms of the Neurocrine Collaboration Agreement. To facilitate the transfer of the ex-U.S. rights to the FA Program to Neurocrine, the Company and Neurocrine executed an amendment to the Neurocrine Collaboration Agreement (the “June 2019 Modification”), and Neurocrine paid $5.0 million to the Company . There were no other changes in pricing or scope of the obligations required to be performed under the Neurocrine Collaboration Agreement In February 2021, Neurocrine notified the Company that it had elected to terminate the Neurocrine Collaboration Agreement solely with regards to the VY-AADC Program, effective August 2, 2021 (the “Neurocrine VY-AADC Program Termination Effective Date”). The Neurocrine Collaboration Agreement remains in full force and effect for each other program thereunder. As a result of the termination, subsequent to the Neurocrine VY-AADC Program Termination Effective Date, Neurocrine is no longer be obligated to reimburse the Company for research and development activities related to the VY-AADC Program. Under the terms of the Neurocrine Collaboration Agreement, the Company originally agreed to collaborate with Neurocrine on, and to grant, exclusive, royalty-bearing, non-transferable, sublicensable licenses to certain of its intellectual property rights, for all human and veterinary diagnostic, prophylactic, and therapeutic uses, for the research, development, and commercialization of gene therapy products (the “Collaboration Products”) on a worldwide basis under (i) the VY-AADC Program; (ii) the FA Program; and (iii) each Discovery Program. As a result of the termination of the Neurocrine Collaboration Agreement with regards to the VY-AADC Program, in accordance with the terms of the Neurocrine Collaboration Agreement, the licenses granted by the Company to Neurocrine regarding the VY-AADC Program have expired, and the Company has regained worldwide intellectual property rights regarding the VY-AADC Program, in each case as of the VY-AADC Termination Effective Date. Pursuant to development plans agreed by the parties, which are overseen by a joint steering committee (“JSC”), the Company has operational responsibility, subject to certain exceptions, for the conduct of each Neurocrine Program prior to the occurrence of a specified event for such Neurocrine Program (a “Transition Event”), as described below, and is required to use commercially reasonable efforts to develop the corresponding Collaboration Products. Neurocrine has agreed to be responsible for all costs incurred by the Company in conducting these activities for each Neurocrine Program, in accordance with an agreed budget for each Neurocrine Program. If the Company breaches its development responsibilities or in certain circumstances upon a change in control, Neurocrine has the right but not the obligation to assume the activities under such Neurocrine Program. U pon the occurrence of a Transition Event for each Neurocrine Program, Neurocrine has agreed to assume responsibility for development, manufacturing and commercialization activities for such Neurocrine Program from the Company and to pay milestones and royalties on future net sales as described further below. As a result of Neurocrine’s termination of the Neurocrine Collaboration Agreement with respect to the VY-AADC Program, the Transition Event with respect to the VY-AADC Program is no longer applicable. The Transition Events for the remaining programs are (i) with respect to the FA Program, the Company’s receipt of topline data for the initial Phase 1 clinical trial for an FA Program product candidate; and (ii) with respect to each Discovery Program, the preparation by the Company and the approval by Neurocrine of an investigational new drug (“IND”) application to be filed with the U.S. Food and Drug Administration (the “FDA”) by Neurocrine for the first development candidate in such Discovery Program. For the FA Program, the Company was granted the option (the “FA Co-Co Option”) to co-develop and co-commercialize the FA Program upon the occurrence of a specified event (a “FA Co-Co Trigger Event”). The Company agreed, upon its exercise of the FA Co-Co Option, to enter into a cost- and profit-sharing arrangement with Neurocrine (the “FA Co-Co Agreement”), and (i) jointly develop and commercialize the Collaboration Products for the FA Program (“FA Collaboration Products”), (ii) share in its costs, profits and losses, and (iii) forfeit certain milestones and royalties on net sales in the United States during the effective period of the FA Co-Co Agreement. The FA Co-Co Trigger Event is the receipt of topline data for the initial Phase 1 clinical trial for an FA Program product candidate. Under the Neurocrine Collaboration Agreement, subject to exceptions specified therein, the Company and Neurocrine agreed that profits and losses under the Company’s FA Co-Co Option would be allocated 60% to Neurocrine and 40% to the Company for any FA Collaboration Product. The parties agreed that FA Co-Co Agreement would provide the Company the right to terminate for any reason upon prior written notice to Neurocrine and Neurocrine the right to terminate in certain circumstances upon change of control. The Company’s research and development activities under the Neurocrine Collaboration Agreement are to be conducted pursuant to plans agreed to by the parties, on a program-by-program basis, and overseen by the JSC, as detailed in the Neurocrine Collaboration Agreement. The parties have agreed on a list of up to eight target genes (the “Targets”) from which Neurocrine had the right to nominate Targets for the two Discovery Programs. The Targets nominated for the Discovery Programs were approved by a consensus of the JSC or the executive officers. The Neurocrine Collaboration Agreement provides for an upfront non-refundable payment of $115.0 million, as well as for aggregate development and regulatory milestone payments from Neurocrine to the Company for Collaboration Products under (i) the VY-AADC Program of up to $170.0 million; (ii) the FA Program of up to $195.0 million, and (iii) each of the two Discovery Programs of up to $130.0 million per Discovery Program. The Company may be entitled to receive aggregate commercial milestone payments for each Collaboration Product of up to $275.0 million, subject to an aggregate cap on commercial milestone payments across all Neurocrine Programs of $1.1 billion. As a result of Neurocrine’s termination of the Neurocrine Collaboration Agreement with respect to the VY-AADC Program, the Company is no longer entitled to receive the development, regulatory and commercial milestone payments related to the VY-AADC Program upon the achievement of specified milestones. Furthermore, in connection with the Neurocrine Collaboration Agreement, Neurocrine purchased 4,179,728 shares of the Company’s common stock at a price of $11.9625 per share, for an aggregate purchase price of $50.0 million. Neurocrine also agreed to pay the Company royalties, based on future net sales of the Collaboration Products. Such royalty percentages, for net sales in and outside the United States, as applicable, range (i) for the VY-AADC Program, from the mid-teens to low thirties and the low-teens to low twenties, respectively; (ii) for the FA Program, from the low-teens to high-teens and high-single digits to mid-teens, respectively; and (iii) for each Discovery Program, from the high-single digits to mid-teens and mid-single digits to low-teens, respectively. On a country-by-country and program-by-program basis, royalty payments would commence on the first commercial sale of a Collaboration Product and terminate on the later of (a) the expiration of the last patent covering the Collaboration Product or its method of use in such country, (b) ten years from the first commercial sale of the Collaboration Product in such country and (c) the expiration of regulatory exclusivity in such country (the “Royalty Term”). Royalty payments may be reduced by up to 50% in specified circumstances, including expiration of patents rights related to a Collaboration Product, approval of biosimilar products in a given country or required payment of licensing fees to third parties related to the development and commercialization of any Collaboration Product. As a result of Neurocrine’s termination of the Neurocrine Collaboration Agreement with respect to the VY-AADC Program, the Company is no longer entitled to receive royalties related to the VY-AADC Program. Additionally, the licenses granted to Neurocrine shall automatically convert to fully paid-up, non-royalty bearing, perpetual, irrevocable, exclusive licenses on a country-by-country and product-by-product basis upon the expiration of the Royalty Term applicable to such Collaboration Product in such country. Under the terms of the Neurocrine Collaboration Agreement and subject to specified exceptions therein, each party owns the entire right, title and interest in and to all intellectual property rights made solely by its employees or agents in the course of the collaboration. The parties jointly own all rights, title and interest in and to all intellectual property rights made or invented jointly by employees or agents of both parties. During the term of the Neurocrine Collaboration Agreement, neither party nor any of its respective affiliates is permitted to directly or indirectly exploit any AAV-based gene therapy products directed to a Target to which a Collaboration Product is directed, subject to specified exceptions including the parties’ conduct of basic research activities. Unless earlier terminated, the Neurocrine Collaboration Agreement expires on the later of (i) the expiration of the last to expire royalty term with respect to a Collaboration Product in all countries in the relevant territory or (ii) the expiration or termination of any FA Co-Co Agreement. Neurocrine may terminate the Neurocrine Collaboration Agreement in its entirety or on a program-by-program or country-by-country basis by providing at least (x) 180-day advance notice if such notice is provided prior to the first commercial sale of the Collaboration Product to which the termination applies or (y) one-year advance notice if such notice is provided after the first commercial sale of the Collaboration Product to which the termination applies. The Company may terminate the Neurocrine Collaboration Agreement, subject to specified conditions, if Neurocrine challenges the validity or enforceability of certain of the Company’s intellectual property rights. Subject to a cure period, either party may terminate the Neurocrine Collaboration Agreement in the event of a material breach by the other party in whole or in part, subject to specified conditions. Upon termination in certain cases, Neurocrine has agreed to grant to the Company licenses to certain Neurocrine intellectual property, subject to a negotiation between the parties to establish royalty rates for use of such intellectual property. In the event of a breach by the Company with respect to a Neurocrine Program, if such termination were to occur after a Transition Event, then (i) with respect to the FA Program, if an FA Co-Co Agreement is in effect, Neurocrine can terminate the FA Co-Co Agreement for such program and the Company would no longer have co- development and co-commercialization rights with respect to the FA Collaboration Products and (ii) subject to any license agreements, Neurocrine would no longer have any obligations with respect to any Collaboration Products resulting from such program. Termination of VY-AADC Program As described above, as of the Neurocrine VY-AADC Program Termination Effective Date, the license granted by the Company to Neurocrine thereunder regarding the VY-AADC Program expired, the Company regained worldwide intellectual property rights regarding the VY-AADC Program, and the restrictions on the Company to develop, manufacture or commercialize a gene therapy product directed to the Target of the VY-AADC Program terminated, in each case in accordance with the terms of the Neurocrine Collaboration Agreement. As of the Neurocrine VY-AADC Program Termination Effective Date, Neurocrine no longer is obligated to reimburse the Company for research and development activities related to the VY-AADC Program, and the Company is no longer entitled to receive future milestone or royalty payments related to the VY-AADC Program. The Company continues to believe that the VY-AADC program may hold promise for Parkinson’s disease patients as evidenced by the positive multi-year safety and efficacy data from the two Phase 1b clinical trials presented in September 2020 at the MDS Virtual Congress. As a result of portfolio reevaluation efforts and a strategic shift to invest in novel capsid development efforts, however, the Company has determined that it will not advance the VY-AADC Program on its own. The Company is exploring potential options for continuing the future development and commercialization of the VY-AADC Program as a partnered program. Accounting Analysis At inception, the Neurocrine Collaboration Agreement included the following performance obligations: (i) research and development services for each Legacy Program combined with a development and commercialization license for each such program and (ii) research and development services for each Discovery Program combined with a development and commercialization license for each program. The research services and license on a program by program basis are not distinct as Neurocrine cannot benefit from such license on its own or from other resources commonly available in the industry, without the corresponding research services due to the unique and specialized expertise of the Company that is not readily available in the marketplace. The Company identified $92.4 million of fixed transaction price consisting of the $115.0 million upfront fee and $5.0 million payment from the June 2019 Modification, offset by a discount of $27.6 million related to the $50.0 million equity investment of 4,179,728 shares when measured at fair value on the date of issuance. The Company is also entitled to reimbursement of costs incurred by the Company prior to the Transition Events associated with each Neurocrine Program. These amounts are determinable based on program plans and budgets, and the Company has a contractual right to the payment of costs incurred under the agreed upon program plans. The Company utilized the most likely amount approach and estimated the expected cost reimbursement to be $431.1 million at inception. The Company concluded that these amounts do not require a constraint and are included in the transaction price at inception. The Company considers this estimate at each reporting date and updates the estimate based on information available. As of September 30, 2021, the estimate of the expected reimbursement is The Company allocated the fixed transaction price to the separate performance obligations based on the relative standalone selling price of each performance obligation or in the case of certain variable consideration to one or more performance obligations. The estimated standalone selling prices for performance obligations, that include a license and research services, were developed using the estimated selling price of the license, using comparable and market data, and an estimate of the overall effort to perform the research services along with a reasonable profit for research services. The Company has concluded that the variable consideration related to the cost reimbursement of each program will be allocated to each respective program as the cost reimbursement relates specifically to the respective program services being performed under the Neurocrine Collaboration Agreement. The reimbursement of research services is considered to be at a market rate and the allocation of the fixed consideration to all of the performance obligations depicts the estimated amounts in which it would expect to receive for these obligations, absent the variable consideration related to the research reimbursement. The total variable consideration allocated to each program related to the expected cost reimbursement was as follows as of September 30, 2021 Performance Obligation Amount (in thousands) Variable Consideration VY-AADC Program $ 53,397 FA Program 88,533 Discovery Program 1 72,782 Discovery Program 2 70,040 Total $ 284,752 Based on the relative standalone selling price allocation, the allocation of the transaction price, exclusive of the variable consideration allocated to the individual performance obligations, to the separate performance obligations was as follows: Performance Obligation Amount (in thousands) Fixed Consideration VY-AADC Program $ 49,045 FA Program 20,647 Discovery Program 1 14,443 Discovery Program 2 8,247 Total $ 92,382 The Company recognizes the transaction price associated with each performance obligation on a proportional performance basis over the period of service using input-based measurements such as costs incurred to date, to estimate proportion performed, and remeasures its progress towards completion at the end of each reporting period. The Company determined the partial termination of the Neurocrine Collaboration Agreement with respect to the VY-AADC Program represented a modification of the arrangement under ASC 606 and that the remaining fixed transaction price at the Neurocrine VY-AADC Program Termination Effective Date of $42.2 million should be re-allocated to the FA Program and Discovery Program 1 and 2 based on their standalone selling prices. Accordingly, the Company recorded a cumulative adjustment to revenue of approximately $0.9 million on the partially satisfied remaining performance obligations, as the remaining services to be performed under each of the performance obligations are not distinct from the services prior to the modification. The Company determined that reasonable changes to the Company’s estimates of standalone selling prices for the FA Program, Discovery Program 1 and Discovery Program 2 performance obligations did not have a material impact on the re-allocation or the amount of revenue recorded pursuant to the cumulative catch-up adjustment. During the three and nine months ended September 30, 2021, the Company recognized $1.5 million and $9.3 million, respectively, of revenue associated with its collaboration with Neurocrine related to research and development services performed during the period and the corresponding cost reimbursement receivable. During the three and nine months ended September 30, 2020, the Company recognized $11.2 million and $50.1 million of revenue, respectively, associated with its collaboration with Neurocrine related to research and development services performed during the period and the corresponding cost reimbursement receivable. The following table presents changes in the balances of the Company’s related party collaboration receivables and contract liabilities during the nine months ended September 30, 2021: Balance at Balance at December 31, 2020 Additions Deductions September 30, 2021 ( in thousands) Related party collaboration receivable $ 8,012 $ 6,418 $ (13,265) $ 1,165 Contract liabilities: Deferred revenue $ 43,689 $ - $ (4,512) $ 39,176 The change in the receivable balance for the nine months ended September 30, 2021 is primarily driven by amounts owed to the Company for research and development services provided, offset by amounts collected from Neurocrine during the period. As of September 30, 2021, there was $39.2 million of deferred revenue related to the Neurocrine Collaboration Agreement, which is classified as either current or non-current in the accompanying condensed consolidated balance sheet based on the period the services are expected to be delivered. Costs incurred relating to the Company’s collaboration programs under the Neurocrine Collaboration Agreement consist of internal and external research and development costs, which primarily include: salaries and benefits, lab supplies, preclinical research studies, clinical studies, consulting services, and commercial development. These costs are included in research and development expenses in the Company’s condensed consolidated statements of operations during the three and nine months ended September 30, 2021. The Company incurred approximately $0.8 million of costs to obtain the Neurocrine Collaboration Agreement which were payable only upon the close of the deal and therefore considered incremental costs of obtaining a contract with a customer and capitalized. The costs are recorded in prepaid expenses and other non-current assets and are being amortized over the period in which the research services will be provided. Other Agreements The Company has entered into various agreements with contract research organizations and institutions to license intellectual property. In consideration for the rights licensed under such agreements, the Company generally made upfront payments, which were recorded as research and development expense as the acquired technologies were considered in-process research and development. The license agreements obligate the Company to make additional payments that are contingent upon specific clinical trial and regulatory approval milestones being achieved as well as royalties on future product sales. The agreements to license intellectual property include potential milestone payments that are dependent upon the development of products licensed under the agreements and contingent upon the achievement of clinical trial or regulatory approval milestones. The Company reached a milestone related to first patient dosing on the RESTORE-1 Phase 2 clinical trial which resulted in a $0.1 million milestone payment to one of its licensors in 2019. The Company can generally terminate the license agreements upon 30 to 90 days’ prior written notice. Additionally, certain license agreements require the Company to reimburse the licensor for certain past and ongoing patent related expenses. During the year ended December 31, 2016, the Company entered into a research and development funding arrangement with a non-profit organization that provides up to $4.0 million in funding to the Company upon the achievement of clinical and development milestones. The agreement provides that the Company repay amounts received under certain circumstances including termination of the agreement, and to pay an amount up to 2.6 times the funding received upon successful development and commercialization of any products developed. During the year ended December 31, 2017, the Company earned a milestone payment of $1.0 million. The Company has evaluated the arrangement and has concluded that it represents a research and development financing arrangement as it is probable that the Company will repay amounts received under the arrangement. As a result, the $1.0 million earned to date is recorded as a non-current liability in the condensed consolidated balance sheet. Litigation Except as described below, the Company was not a party to any material legal matters or claims as of September 30, 2021 or December 31, 2020. The Company did not have contingency reserves established for any litigation liabilities as of September 30, 2021 or December 31, 2020. On January 22, 2021, a putative class action lawsuit was filed in the U.S. District Court for the Eastern District of New York against the Company and certain of its current and former officers and directors, captioned Karp v. Voyager Therapeutics, Inc. et al. , No. 1:21-cv-00381. The complaint generally alleged that the defendants violated Sections 10(b) and/or 20(a) of the Securities Exchange Act of 1934 and Rule 10b-5 promulgated thereunder by making material misstatements or omissions concerning the Company’s Huntington’s disease program and the Company’s investigational new drug application for VY-HTT01. On April 19, 2021, the court appointed the lead plaintiff for the action, and on April 30, 2021, the action was transferred to the U.S. District Court for the District of Massachusetts (where it was assigned case number 1:21-cv-10727). On July 2, 2021, the lead plaintiff voluntarily dismissed the action without prejudice against all defendants and as to all claims. This matter is no longer pending. |
Stock-based compensation
Stock-based compensation | 9 Months Ended |
Sep. 30, 2021 | |
Stock-based compensation | |
Stock-based compensation | 9. Stock-based compensation Stock-Based Compensation Expense Total compensation cost recognized for all stock-based compensation awards in the condensed consolidated statements of operations and comprehensive income (loss) is as follows: Three Months Ended Nine Months Ended September 30, September 30, 2021 2020 2021 2020 (in thousands) Research and development $ 702 $ 1,524 $ 3,409 $ 4,973 General and administrative 1,375 2,008 6,261 6,459 Total stock-based compensation expense $ 2,077 $ 3,532 $ 9,670 $ 11,432 Stock-based compensation expense by type of award included within the condensed consolidated statements of operations and comprehensive income (loss) was as follows: Three Months Ended Nine Months Ended September 30, September 30, 2021 2020 2021 2020 (in thousands) Stock options $ 1,372 $ 2,696 $ 6,375 $ 8,834 Restricted stock awards and units 651 740 3,018 2,345 Employee stock purchase plan awards 54 96 277 252 Total stock-based compensation expense $ 2,077 $ 3,532 9,670 11,432 Restricted Stock Units A summary of the status of and changes in unvested restricted stock unit activity under the Company’s equity award plans for the nine months ended September 30, 2021 was as follows: Weighted Average Grant Date Fair Value Units Per Unit Unvested restricted stock units as of December 31, 2020 638,471 $ 12.74 Granted 1,264,651 $ 6.20 Vested (308,196) 12.88 Forfeited (647,661) $ 9.08 Unvested restricted stock units as of September 30, 2021 947,265 $ 7.52 Stock-based compensation of restricted stock units is based on the fair value of the Company’s common stock on the date of grant and is recognized over the vesting period. Restricted stock units granted by the Company typically vest in equal amounts, annually over three years. In the nine months ended September 30, 2021, the Company granted 534,651 restricted stock units vesting in equal amounts, annually over three years, and 730,000 restricted stock units vesting in equal amounts, annually over two years. All of the restricted stock units granted in the nine months ended September 30, 2020 vest in equal amounts, annually over three years. The stock-based compensation expense related to restricted stock units and awards was $0.7 million and $3.0 million for the three and nine months ended September 30, 2021, respectively. The stock-based compensation expense related to restricted stock units and awards was $0.7 million and $2.3 million for the three and nine months ended September 30, 2020, respectively. As of September 30, 2021, the Company had unrecognized stock-based compensation expense related to its unvested restricted stock units of $0.3 million, which is expected to be recognized over the remaining average vesting period of 1.7 years. Stock Options The following is a summary of stock option activity for the nine months ended September 30, 2021: Weighted Remaining Aggregate Average Contractual Intrinsic Exercise Life Value Shares Price (in years) (in thousands) Outstanding at December 31, 2020 5,485,078 $ 14.77 Granted 1,411,929 $ 6.57 Exercised (3,811) $ 7.27 Cancelled or forfeited (1,487,124) $ 13.41 Outstanding at September 30, 2021 5,406,072 $ 13.01 6.0 $ — Exercisable at September 30, 2021 3,750,020 $ 14.48 4.7 $ — As of September 30, 2021, the Company had unrecognized stock-based compensation expense related to its unvested stock options of $9.4 million which is expected to be recognized over the remaining weighted-average vesting period of 2.4 years. |
Net loss per share
Net loss per share | 9 Months Ended |
Sep. 30, 2021 | |
Net loss per share | |
Net loss per share | 10. Net loss per share The following table sets forth the outstanding potentially dilutive securities that have been excluded in the calculation of diluted net loss per share because to include them would be anti-dilutive: As of September 30, 2021 2020 Unvested restricted common stock awards 156,863 117,647 Unvested restricted common stock units 947,265 — Outstanding stock options 5,406,072 4,013,244 Total 6,510,200 4,130,891 Basic net income (loss) and diluted weighted-average shares outstanding are as follows for the three and nine months ended September 30, 2021 and 2020. Three Months Ended September 30, Nine Months Ended September 30, 2021 2020 2021 2020 (in thousands, except share data) Numerator: Net (loss) income $ (25,137) $ 85,611 $ (76,906) $ 52,667 Denominator for basic net (loss) income per share: Weighted average shares outstanding-basic 37,780,547 37,242,504 37,623,309 37,079,242 Denominator for diluted net (loss) income per share: Weighted average shares outstanding 37,780,547 37,242,504 37,623,309 37,079,242 Common stock options and restricted stock units — 429,824 — 420,913 Weighted average shares outstanding-diluted 37,780,547 37,672,328 37,623,309 37,500,155 |
Related party transactions
Related party transactions | 9 Months Ended |
Sep. 30, 2021 | |
Related party transactions | |
Related party transactions | 11. Related-party transactions During the three and nine months ended September 30, 2021 and 2020, the Company received board and scientific advisory services from one of its prior executives, Dinah Sah, Ph.D., the Company’s former Chief Scientific Officer. The total amount of fees paid to Dr. Sah for services provided during the three and nine months ended September 30, 2021, was . During the three and nine months ended September 30, 2021, the Company received consulting and advisor services from Third Rock Ventures. The total fees paid was de minimus. Under the Neurocrine Collaboration Agreement, the Company and Neurocrine have agreed to conduct research, development and commercialization activities for certain of the Company’s AAV gene therapy products (Note 8). Amounts due from Neurocrine are reflected as related party collaboration receivables. As of September 30, 2021, the Company had approximately |
Subsequent Events
Subsequent Events | 9 Months Ended |
Sep. 30, 2021 | |
Subsequent Events | |
Subsequent Events | 12. Subsequent Events On October 1, 2021, the Company entered into an option and license agreement with Pfizer pursuant to which the Company has granted Pfizer options to receive an exclusive license to novel capsids generated from the Company’s TRACER screening technology to develop and commercialize certain AAV gene therapy candidates comprised of a novel capsid and specified transgenes to help treat respective central nervous system and cardiovascular diseases. Under the terms of this agreement, the Company received an upfront payment of $30.0 million and is eligible to receive future option exercise payments of $10.0 million upon each of up to two option exercises; specified development, regulatory, and commercialization milestone payments following each option exercise of up to an aggregate of $115.0 million for the first licensed product to achieve such milestones; specified sales milestone payments of up to an aggregate of $175.0 million per licensed product; and tiered, escalating royalties in the mid- to high-single digit percentages of annual net sales of each licensed product. |
Summary of significant accoun_2
Summary of significant accounting policies (Policies) | 9 Months Ended |
Sep. 30, 2021 | |
Summary of significant accounting policies | |
Basis of Presentation | Basis of Presentation The accompanying unaudited condensed consolidated financial statements of the Company have been prepared in accordance with accounting principles generally accepted in the United States (“GAAP”) for interim financial reporting and as required by Regulation S-X, Rule 10-01. Accordingly, they do not include all of the information and footnotes required by GAAP for complete financial statements. For further information, refer to the consolidated financial statements and footnotes included in the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2020 as filed with the Securities and Exchange Commission (“SEC”) on February 25, 2021. These interim condensed consolidated financial statements, in the opinion of management, reflect all normal recurring adjustments necessary for a fair presentation of the Company’s financial position and results of operations for the periods presented. 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 Updates (“ASU”) of the Financial Accounting Standards Board (“FASB”). |
Principles of Consolidation | Principles of Consolidation The unaudited interim consolidated financial statements include the accounts of the Company and its wholly owned subsidiary as disclosed in Note 2, under the headings “Summary of Significant Accounting Policies” and “Basis of Presentation”, within the “Notes to Consolidated Financial Statements” accompanying the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2020. Intercompany accounts and transactions have been eliminated. |
Use of Estimates | Use of Estimates The preparation of consolidated financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the amounts reported in the consolidated financial statements and accompanying notes. On an ongoing basis, the Company’s management evaluates its estimates, which include, but are not limited to, estimates related to revenue recognition, accrued expenses, stock-based compensation expense, and income taxes. The Company bases its estimates on historical experience and other market specific or other relevant assumptions that it believes to be reasonable under the circumstances. Actual results may differ from those estimates or assumptions. Certain reclassifications have been made to prior periods to conform to current period presentation. |
Recent Accounting Pronouncements | Summary of Significant Accounting Policies There have been no changes in the Company's significant accounting policies as described in Note 2, “Summary of Significant Accounting Policies,” within the “Notes to Consolidated Financial Statements” accompanying the Company’s Annual Report on Form 10-K for the year ended December 31, 2020. |
Fair value measurements (Tables
Fair value measurements (Tables) | 9 Months Ended |
Sep. 30, 2021 | |
Fair value measurements | |
Assets and liabilities measured at fair value on a recurring basis | Assets and liabilities measured at fair value on a recurring basis as of September 30, 2021 and December 31, 2020 are as follows: Quoted Prices Significant in Active Other Significant Markets for Observable Unobservable Identical Assets Inputs Inputs Assets Total (Level 1) (Level 2) (Level 3) September 30, 2021 (in thousands) Money market funds included in cash and cash equivalents $ 105,359 $ 105,359 $ — $ — Marketable securities - U.S. Treasury notes 10,024 10,024 — — Total $ 115,383 $ 115,383 $ — $ — December 31, 2020 Money market funds included in cash and cash equivalents $ 103,992 $ 103,992 $ — $ — Marketable securities: U.S. Treasury notes 70,342 70,342 — — Equity securities 6,356 6,356 — — Total marketable securities $ 76,698 $ 76,698 $ — $ — Warrants to purchase equity securities 3,816 — 3,816 — Total $ 184,506 $ 180,690 $ 3,816 $ — |
Cash, cash equivalents, restr_2
Cash, cash equivalents, restricted cash, and available-for-sale marketable securities (Tables) | 9 Months Ended |
Sep. 30, 2021 | |
Cash, cash equivalents, restricted cash, and available-for-sale marketable securities | |
Schedule of money market funds and marketable securities | Cash, cash equivalents, and marketable securities included the following at September 30, 2021 and December 31, 2020: Amortized Unrealized Unrealized Fair Cost Gains Losses Value (in thousands) As of September 30, 2021 Money market funds included in cash and cash equivalents $ 105,359 $ — $ — $ 105,359 Marketable securities - U.S. Treasury notes 10,024 — 10,024 Total money market funds and marketable securities $ 115,383 $ — $ — $ 115,383 As of December 31, 2020 Money market funds included in cash and cash equivalents $ 103,992 $ — $ — $ 103,992 Marketable securities: U.S. Treasury notes 70,348 — 6 70,342 Equity securities 1,220 5,136 — 6,356 Total marketable securities $ 71,568 $ 5,136 $ 6 $ 76,698 Total money market funds and marketable securities $ 175,560 $ 5,136 $ 6 $ 180,690 |
Reconciliation of cash, cash equivalents, and restricted cash | As of September 30, As of December 31, 2021 2020 (in thousands) Cash and cash equivalents $ 111,475 $ 104,440 Restricted cash included in deposits and other non-current assets 1,779 1,779 Total cash, cash equivalents, and restricted cash $ 113,254 $ 106,219 |
Accrued expenses (Tables)
Accrued expenses (Tables) | 9 Months Ended |
Sep. 30, 2021 | |
Accrued expenses | |
Schedule of accrued expenses | As of September 30, As of December 31, 2021 2020 (in thousands) Employee compensation costs $ 5,862 $ 5,857 Research and development costs 4,068 6,624 Accrued goods and services 326 496 Professional services 1,068 1,228 Total $ 11,324 $ 14,205 |
Lease obligation (Tables)
Lease obligation (Tables) | 9 Months Ended |
Sep. 30, 2021 | |
Lease obligation | |
Summary of significant contractual obligations under operating leases | The following table summarizes the Company’s significant contractual obligations under operating leases as of payment due date by period as of September 30, 2021: Total Minimum Lease Payments (in thousands) 2021 (remainder of year) 2,117 2022 8,698 2023 8,958 2024 9,227 2025 9,644 Thereafter 19,945 Total future minimum lease payments $ 58,589 Less: imputed interest (13,330) Total lease liability $ 45,259 Reported as: Other current liabilities $ 5,248 Other non-current liabilities 40,011 Total lease liability $ 45,259 |
Summary of operating sublease income | Three Months Ended Nine Months Ended September 30, September 30, 2021 2020 2021 2020 Operating sublease income (in thousands) $ 204 $ — $ 204 $ — |
Other liabilities (Tables)
Other liabilities (Tables) | 9 Months Ended |
Sep. 30, 2021 | |
Other liabilities | |
Summary of other current and non-current liabilities | As of September 30, As of December 31, 2021 2020 (in thousands) Other current liabilities Lease liability 5,248 4,198 Total other current liabilities $ 5,248 $ 4,198 Other non-current liabilities Lease liability $ 40,011 $ 43,409 Other 1,001 1,001 Total other non-current liabilities $ 41,012 $ 44,410 |
Commitments and contingencies (
Commitments and contingencies (Tables) | 9 Months Ended |
Sep. 30, 2021 | |
Schedule of collaboration receivables and contract liabilities | Balance at Balance at December 31, 2020 Additions Deductions September 30, 2021 ( in thousands) Related party collaboration receivable $ 8,012 $ 6,418 $ (13,265) $ 1,165 Contract liabilities: Deferred revenue $ 43,689 $ - $ (4,512) $ 39,176 |
Neurocrine Collaboration Agreement | |
Schedule of allocation of variable consideration | Performance Obligation Amount (in thousands) Variable Consideration VY-AADC Program $ 53,397 FA Program 88,533 Discovery Program 1 72,782 Discovery Program 2 70,040 Total $ 284,752 |
Schedule of allocation of fixed consideration | Performance Obligation Amount (in thousands) Fixed Consideration VY-AADC Program $ 49,045 FA Program 20,647 Discovery Program 1 14,443 Discovery Program 2 8,247 Total $ 92,382 |
Stock-based compensation (Table
Stock-based compensation (Tables) | 9 Months Ended |
Sep. 30, 2021 | |
Stock-based compensation | |
Compensation cost recognized for all stock-based compensation awards | Total compensation cost recognized for all stock-based compensation awards in the condensed consolidated statements of operations and comprehensive income (loss) is as follows: Three Months Ended Nine Months Ended September 30, September 30, 2021 2020 2021 2020 (in thousands) Research and development $ 702 $ 1,524 $ 3,409 $ 4,973 General and administrative 1,375 2,008 6,261 6,459 Total stock-based compensation expense $ 2,077 $ 3,532 $ 9,670 $ 11,432 |
Summary of stock-based compensation expense by type of award | Three Months Ended Nine Months Ended September 30, September 30, 2021 2020 2021 2020 (in thousands) Stock options $ 1,372 $ 2,696 $ 6,375 $ 8,834 Restricted stock awards and units 651 740 3,018 2,345 Employee stock purchase plan awards 54 96 277 252 Total stock-based compensation expense $ 2,077 $ 3,532 9,670 11,432 |
Summary of status and changes in unvested restricted stock | A summary of the status of and changes in unvested restricted stock unit activity under the Company’s equity award plans for the nine months ended September 30, 2021 was as follows: Weighted Average Grant Date Fair Value Units Per Unit Unvested restricted stock units as of December 31, 2020 638,471 $ 12.74 Granted 1,264,651 $ 6.20 Vested (308,196) 12.88 Forfeited (647,661) $ 9.08 Unvested restricted stock units as of September 30, 2021 947,265 $ 7.52 |
Summary of stock option activity | The following is a summary of stock option activity for the nine months ended September 30, 2021: Weighted Remaining Aggregate Average Contractual Intrinsic Exercise Life Value Shares Price (in years) (in thousands) Outstanding at December 31, 2020 5,485,078 $ 14.77 Granted 1,411,929 $ 6.57 Exercised (3,811) $ 7.27 Cancelled or forfeited (1,487,124) $ 13.41 Outstanding at September 30, 2021 5,406,072 $ 13.01 6.0 $ — Exercisable at September 30, 2021 3,750,020 $ 14.48 4.7 $ — |
Net loss per share (Tables)
Net loss per share (Tables) | 9 Months Ended |
Sep. 30, 2021 | |
Net loss per share | |
Outstanding potentially dilutive securities excluded in the calculation of diluted net loss per share | The following table sets forth the outstanding potentially dilutive securities that have been excluded in the calculation of diluted net loss per share because to include them would be anti-dilutive: As of September 30, 2021 2020 Unvested restricted common stock awards 156,863 117,647 Unvested restricted common stock units 947,265 — Outstanding stock options 5,406,072 4,013,244 Total 6,510,200 4,130,891 |
Schedule of weighted average number of shares, basic and diluted | Three Months Ended September 30, Nine Months Ended September 30, 2021 2020 2021 2020 (in thousands, except share data) Numerator: Net (loss) income $ (25,137) $ 85,611 $ (76,906) $ 52,667 Denominator for basic net (loss) income per share: Weighted average shares outstanding-basic 37,780,547 37,242,504 37,623,309 37,079,242 Denominator for diluted net (loss) income per share: Weighted average shares outstanding 37,780,547 37,242,504 37,623,309 37,079,242 Common stock options and restricted stock units — 429,824 — 420,913 Weighted average shares outstanding-diluted 37,780,547 37,672,328 37,623,309 37,500,155 |
Nature of business (Details)
Nature of business (Details) - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2021 | Dec. 31, 2020 | |
Nature of business | ||
Accumulated deficit | $ (352,813) | $ (275,907) |
Proceeds raised | 640,000 | |
Cash, cash equivalents, and marketable debt securities | $ 121,500 |
Fair value measurements - Asset
Fair value measurements - Assets measured on a recurring basis - (Details) - USD ($) $ in Thousands | Sep. 30, 2021 | Dec. 31, 2020 |
Assets, Fair Value Disclosure [Abstract] | ||
Cash and cash equivalents | $ 111,475 | $ 104,440 |
Fair Value, Measurements, Recurring | ||
Assets, Fair Value Disclosure [Abstract] | ||
Marketable securities | 76,698 | |
Total assets measured at fair value | 115,383 | 184,506 |
Level 1 | Fair Value, Measurements, Recurring | ||
Assets, Fair Value Disclosure [Abstract] | ||
Marketable securities | 76,698 | |
Total assets measured at fair value | 115,383 | 180,690 |
Level 2 | Fair Value, Measurements, Recurring | ||
Assets, Fair Value Disclosure [Abstract] | ||
Total assets measured at fair value | 3,816 | |
U.S. Treasury notes | Fair Value, Measurements, Recurring | ||
Assets, Fair Value Disclosure [Abstract] | ||
Marketable securities | 10,024 | 70,342 |
U.S. Treasury notes | Level 1 | Fair Value, Measurements, Recurring | ||
Assets, Fair Value Disclosure [Abstract] | ||
Marketable securities | 10,024 | 70,342 |
Equity securities | Fair Value, Measurements, Recurring | ||
Assets, Fair Value Disclosure [Abstract] | ||
Marketable securities | 6,356 | |
Equity securities | Level 1 | Fair Value, Measurements, Recurring | ||
Assets, Fair Value Disclosure [Abstract] | ||
Marketable securities | 6,356 | |
Warrants | Fair Value, Measurements, Recurring | ||
Assets, Fair Value Disclosure [Abstract] | ||
Warrants to purchase equity securities | 3,816 | |
Warrants | Level 2 | Fair Value, Measurements, Recurring | ||
Assets, Fair Value Disclosure [Abstract] | ||
Warrants to purchase equity securities | 3,816 | |
Money market funds | ||
Assets, Fair Value Disclosure [Abstract] | ||
Cash and cash equivalents | 105,359 | 103,992 |
Money market funds | Fair Value, Measurements, Recurring | ||
Assets, Fair Value Disclosure [Abstract] | ||
Cash and cash equivalents | 105,359 | 103,992 |
Money market funds | Level 1 | Fair Value, Measurements, Recurring | ||
Assets, Fair Value Disclosure [Abstract] | ||
Cash and cash equivalents | $ 105,359 | $ 103,992 |
Cash, cash equivalents, restr_3
Cash, cash equivalents, restricted cash and available-for-sale marketable securities (Details) - USD ($) $ in Thousands | 9 Months Ended | 12 Months Ended |
Sep. 30, 2021 | Dec. 31, 2020 | |
Amortized Cost Basis [Abstract] | ||
Cash and Cash Equivalents, at Carrying Value | $ 111,475 | $ 104,440 |
Total marketable securities, amortized cost | 71,568 | |
Total money market funds and marketable securities | 115,383 | 175,560 |
Fair Value Disclosure [Abstract] | ||
Cash and cash equivalents | 111,475 | 104,440 |
Total marketable securities | 76,698 | |
Total money market funds and marketable securities | 115,383 | 180,690 |
Unrealized Gain and Loss [Abstract] | ||
Total marketable securities, unrealized gains | 5,136 | |
Total money market funds and marketable securities | 5,136 | |
Total marketable securities, unrealized losses | 6 | |
Total money market funds and marketable securities | 6 | |
Money market funds | ||
Amortized Cost Basis [Abstract] | ||
Cash and Cash Equivalents, at Carrying Value | 105,359 | 103,992 |
Fair Value Disclosure [Abstract] | ||
Cash and cash equivalents | 105,359 | 103,992 |
U.S. Treasury notes | ||
Amortized Cost Basis [Abstract] | ||
Marketable securities, amortized cost | 10,024 | 70,348 |
Fair Value Disclosure [Abstract] | ||
Marketable securities, fair value | $ 10,024 | 70,342 |
Unrealized Gain and Loss [Abstract] | ||
Marketable securities, unrealized losses | 6 | |
Equity securities | ||
Amortized Cost Basis [Abstract] | ||
Marketable equity securities, amortized cost | 1,220 | |
Fair Value Disclosure [Abstract] | ||
Marketable equity securities, fair value | 6,356 | |
Unrealized Gain and Loss [Abstract] | ||
Equity securities, unrealized gains | $ 5,136 | |
Maximum | ||
Unrealized Gain and Loss [Abstract] | ||
Debt securities, contractual maturity period | 1 year |
Cash, cash equivalents, restr_4
Cash, cash equivalents, restricted cash and available-for-sale marketable securities - Reconciliation of cash, cash equivalents, and restricted cash (Details) - USD ($) $ in Thousands | Sep. 30, 2021 | Dec. 31, 2020 | Sep. 30, 2020 | Dec. 31, 2019 |
Cash, cash equivalents, restricted cash, and available-for-sale marketable securities | ||||
Cash and cash equivalents | $ 111,475 | $ 104,440 | ||
Restricted cash included in deposits and other noncurrent assets | 1,779 | 1,779 | ||
Total cash, cash equivalents, and restricted cash | $ 113,254 | $ 106,219 | $ 149,226 | $ 86,777 |
Accrued expenses (Details)
Accrued expenses (Details) - USD ($) $ in Thousands | Sep. 30, 2021 | Dec. 31, 2020 |
Accrued expenses | ||
Employee compensation costs | $ 5,862 | $ 5,857 |
Research and development costs | 4,068 | 6,624 |
Accrued goods and services | 326 | 496 |
Professional services | 1,068 | 1,228 |
Total | $ 11,324 | $ 14,205 |
Lease obligation (Details)
Lease obligation (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | |||
Sep. 30, 2021 | Sep. 30, 2020 | Sep. 30, 2021 | Sep. 30, 2020 | Dec. 31, 2020 | |
Lease expense | $ 1,700 | $ 1,600 | $ 5,400 | $ 5,000 | |
Restricted cash included in deposits and other noncurrent assets | $ 1,779 | $ 1,779 | $ 1,779 | ||
Weighted average remaining lease term | 6 years 2 months 12 days | 6 years 2 months 12 days | |||
Weighted average incremental borrowing rate | 7.90% | 7.90% | |||
Total minimum lease payments | |||||
2021 (remainder of year) | $ 2,117 | $ 2,117 | |||
2022 | 8,698 | 8,698 | |||
2023 | 8,958 | 8,958 | |||
2024 | 9,227 | 9,227 | |||
2025 | 9,644 | 9,644 | |||
Thereafter | 19,945 | 19,945 | |||
Total future minimum lease payments | 58,589 | 58,589 | |||
Less: imputed interest | (13,330) | (13,330) | |||
Total lease liability | 45,259 | 45,259 | |||
Current lease liabilities | $ 5,248 | $ 5,248 | 4,198 | ||
Operating Lease, Liability, Current, Statement of Financial Position [Extensible List] | Other Liabilities, Current | Other Liabilities, Current | |||
Non-current lease liabilities | $ 40,011 | $ 40,011 | $ 43,409 | ||
Operating Lease, Liability, Noncurrent, Statement of Financial Position [Extensible List] | Other Liabilities, Noncurrent | Other Liabilities, Noncurrent | |||
75 and 64 Sidney Street | |||||
Leasehold improvements | $ 5,300 | $ 5,300 | |||
75 Sidney Street | |||||
Lease term | 3 years 3 months 18 days | 3 years 3 months 18 days | |||
Financial obligations from sublessee | $ 8,500 | $ 8,500 | |||
75 Hayden Avenue | |||||
Entitled leasehold improvements | 5,600 | 5,600 | |||
Deposits and other non-current assets | |||||
Restricted cash included in deposits and other noncurrent assets | $ 1,800 | $ 1,800 |
Lease obligation - Operating su
Lease obligation - Operating sublease income (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended |
Sep. 30, 2021 | Sep. 30, 2021 | |
Lease obligation | ||
Operating sublease income | $ 204 | $ 204 |
Other liabilities (Details)
Other liabilities (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | |
Sep. 30, 2021 | Sep. 30, 2021 | Dec. 31, 2020 | |
Other current liabilities | |||
Lease liability | $ 5,248 | $ 5,248 | $ 4,198 |
Total other current liabilities | 5,248 | 5,248 | 4,198 |
Other non-current liabilities | |||
Lease liability | 40,011 | 40,011 | 43,409 |
Other | 1,001 | 1,001 | 1,001 |
Total other non-current liabilities | 41,012 | 41,012 | $ 44,410 |
Restructuring costs | $ 2,000 | 2,000 | |
Restructuring costs paid | $ 900 |
Commitments and contingencies -
Commitments and contingencies - Neurocrine Collaboration Agreement - (Details) $ / shares in Units, $ in Thousands | 1 Months Ended | 3 Months Ended | 9 Months Ended | ||||
Jun. 30, 2019USD ($)shares | Mar. 31, 2019USD ($)item$ / sharesshares | Sep. 30, 2021USD ($) | Sep. 30, 2020USD ($) | Sep. 30, 2021USD ($) | Sep. 30, 2020USD ($) | Dec. 31, 2020USD ($) | |
Allocation of Transaction Price | |||||||
Revenue recognized | $ 4,512 | ||||||
Deferred revenue | $ 39,176 | 39,176 | $ 43,689 | ||||
Related party collaboration receivable | 1,165 | 1,165 | $ 8,012 | ||||
Neurocrine | FA Program | |||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||
Percentage of profit or loss under co-co option | 60.00% | ||||||
Neurocrine Collaboration Agreement | FA Program | |||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||
Percentage of profit or loss under co-co option | 40.00% | ||||||
Neurocrine Collaboration Agreement | Neurocrine | |||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||
Number of collaboration programs | item | 4 | ||||||
Number of discovery programs | item | 2 | ||||||
Programs added later | item | 2 | ||||||
Number of targets | item | 8 | ||||||
Upfront payment | $ 115,000 | $ 115,000 | |||||
Purchase of common stock, shares | shares | 4,179,728 | 4,179,728 | |||||
Purchase of common stock | $ 50,000 | $ 50,000 | |||||
Price per share | $ / shares | $ 11.9625 | ||||||
Estimated cost reimbursement | $ 431,100 | 284,700 | |||||
Termination period | 10 years | ||||||
Period of advance notice for termination prior to first commercial sale | 180 days | ||||||
Period of advance notice for termination after first commercial sale | 1 year | ||||||
Discount related to equity investment | 27,600 | ||||||
Allocation of Transaction Price | |||||||
Allocation of variable consideration | 284,752 | 284,752 | |||||
Allocation of fixed consideration | 92,400 | 92,382 | 92,382 | ||||
Revenue recognized | 1,500 | $ 11,200 | 9,300 | $ 50,100 | |||
Cumulative catch up of revenue recognized | 900 | ||||||
Deferred revenue | 39,200 | 39,200 | |||||
Costs to obtain collaboration agreement | 800 | 800 | |||||
Consideration received | $ 5,000 | ||||||
Neurocrine Collaboration Agreement | Neurocrine | Maximum | |||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||
Percentage of reduction in royalty payments | 50.00% | ||||||
Neurocrine Collaboration Agreement | Neurocrine | VY-AADC | |||||||
Allocation of Transaction Price | |||||||
Allocation of variable consideration | 53,397 | 53,397 | |||||
Allocation of fixed consideration | 49,045 | 49,045 | |||||
Neurocrine Collaboration Agreement | Neurocrine | FA Program | |||||||
Allocation of Transaction Price | |||||||
Allocation of variable consideration | 88,533 | 88,533 | |||||
Allocation of fixed consideration | 20,647 | 20,647 | |||||
Neurocrine Collaboration Agreement | Neurocrine | Discovery and FA Programs | |||||||
Allocation of Transaction Price | |||||||
Allocation of fixed consideration | 42,200 | 42,200 | |||||
Neurocrine Collaboration Agreement | Neurocrine | Discovery program 1 | |||||||
Allocation of Transaction Price | |||||||
Allocation of variable consideration | 72,782 | 72,782 | |||||
Allocation of fixed consideration | 14,443 | 14,443 | |||||
Neurocrine Collaboration Agreement | Neurocrine | Discovery program 2 | |||||||
Allocation of Transaction Price | |||||||
Allocation of variable consideration | 70,040 | 70,040 | |||||
Allocation of fixed consideration | $ 8,247 | $ 8,247 | |||||
Neurocrine Collaboration Agreement | Neurocrine | Development and regulatory milestones | VY-AADC | |||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||
Aggregate maximum milestone payments to be received from collaborative partner | $ 170,000 | ||||||
Neurocrine Collaboration Agreement | Neurocrine | Development and regulatory milestones | FA Program | |||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||
Aggregate maximum milestone payments to be received from collaborative partner | 195,000 | ||||||
Neurocrine Collaboration Agreement | Neurocrine | Development and regulatory milestones | Discovery program 1 | |||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||
Aggregate maximum milestone payments to be received from collaborative partner | 130,000 | ||||||
Neurocrine Collaboration Agreement | Neurocrine | Commercial Milestone | |||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||
Per Milestone, maximum milestone payments to be received from collaborative partner | 275,000 | ||||||
Aggregate maximum milestone payments to be received from collaborative partner | $ 1,100,000 |
Commitments and contingencies_2
Commitments and contingencies - Collaboration receivables and contract liabilities - (Details) $ in Thousands | 9 Months Ended |
Sep. 30, 2021USD ($) | |
Commitments and contingencies | |
Related party collaboration receivables, beginning balance | $ 8,012 |
Additions | 6,418 |
Deductions | (13,265) |
Related party collaboration receivables, ending balance | 1,165 |
Deferred revenue, beginning balance | 43,689 |
Deductions | (4,512) |
Deferred revenue, ending balance | $ 39,176 |
Commitments and contingencies_3
Commitments and contingencies - Other Agreements - (Details) $ in Millions | 9 Months Ended | 12 Months Ended | ||
Sep. 30, 2021 | Dec. 31, 2019USD ($) | Dec. 31, 2016USD ($)multiple | Dec. 31, 2017USD ($) | |
Various contract research organizations and institutions | ||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||
Milestone payable upon achievement | $ 0.1 | |||
Various contract research organizations and institutions | Minimum | ||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||
Number of days written notice required by reporting entity to terminate agreement | 30 days | |||
Various contract research organizations and institutions | Maximum | ||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||
Number of days written notice required by reporting entity to terminate agreement | 90 days | |||
Non-profit organization agreement | ||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||
Maximum funding | $ 4 | |||
Funding multiple | multiple | 2.6 | |||
Milestone payment liability | $ 1 |
Stock-based compensation - Comp
Stock-based compensation - Compensation expense (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2021 | Sep. 30, 2020 | Sep. 30, 2021 | Sep. 30, 2020 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Stock-based compensation. | $ 2,077 | $ 3,532 | $ 9,670 | $ 11,432 |
Stock options | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Stock-based compensation. | 1,372 | 2,696 | 6,375 | 8,834 |
Unvested restricted common stock units | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Stock-based compensation. | 651 | 740 | 3,018 | 2,345 |
Employee stock purchase plan | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Stock-based compensation. | 54 | 96 | 277 | 252 |
Research and development | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Stock-based compensation. | 702 | 1,524 | 3,409 | 4,973 |
General and administrative | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Stock-based compensation. | $ 1,375 | $ 2,008 | $ 6,261 | $ 6,459 |
Stock-based compensation - Rest
Stock-based compensation - Restricted Stock - (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2021 | Sep. 30, 2020 | Sep. 30, 2021 | Sep. 30, 2020 | |
Restricted stock disclosures | ||||
Stock-based compensation. | $ 2,077 | $ 3,532 | $ 9,670 | $ 11,432 |
Unvested restricted common stock units | ||||
Shares | ||||
Balance, beginning (in shares) | 638,471 | |||
Granted (in shares) | 1,264,651 | |||
Vested (in shares) | (308,196) | |||
Forfeited (in shares) | (647,661) | |||
Balance, ending (in shares) | 947,265 | 947,265 | ||
Weighted Average Grant Date Fair Value Per Share | ||||
Balance, beginning (in dollars per share) | $ 12.74 | |||
Granted (in dollars per share) | 6.20 | |||
Vested (in dollars per share) | 12.88 | |||
Forfeited (in dollars per share) | 9.08 | |||
Balance, ending (in dollars per share) | $ 7.52 | $ 7.52 | ||
Restricted stock disclosures | ||||
Vesting period | 3 years | 3 years | ||
Stock-based compensation. | $ 651 | $ 740 | $ 3,018 | $ 2,345 |
Unrecognized stock-based compensation expense | $ 300 | $ 300 | ||
Remaining weighted-average remaining vesting period | 1 year 8 months 12 days | |||
Vesting period, tranche one | Unvested restricted common stock units | ||||
Shares | ||||
Granted (in shares) | 534,651 | |||
Restricted stock disclosures | ||||
Vesting period | 3 years | |||
Vesting period, tranche two | Unvested restricted common stock units | ||||
Shares | ||||
Granted (in shares) | 730,000 | |||
Restricted stock disclosures | ||||
Vesting period | 2 years |
Stock-based compensation - Stoc
Stock-based compensation - Stock Options - (Details) $ / shares in Units, $ in Millions | 9 Months Ended |
Sep. 30, 2021USD ($)$ / sharesshares | |
Share-based Compensation Arrangement by Share-based Payment Award, Options, Outstanding [Roll Forward] | |
Outstanding, beginning balance (in shares) | shares | 5,485,078 |
Granted (in shares) | shares | 1,411,929 |
Exercised (in shares) | shares | (3,811) |
Cancelled or forfeited (in shares) | shares | (1,487,124) |
Outstanding, ending balance (in shares) | shares | 5,406,072 |
Exercisable (in shares) | shares | 3,750,020 |
Weighted Average Exercise Price | |
Outstanding (in dollars per share) | $ / shares | $ 14.77 |
Granted (in dollars per share) | $ / shares | 6.57 |
Exercised (in dollars per share) | $ / shares | 7.27 |
Cancelled or forfeited (in dollars per share) | $ / shares | 13.41 |
Outstanding (in dollars per share) | $ / shares | 13.01 |
Exercisable (in dollars per share) | $ / shares | $ 14.48 |
Remaining Contractual Life | |
Outstanding | 6 years |
Exercisable | 4 years 8 months 12 days |
Stock options | |
Options disclosures | |
Unrecognized stock-based compensation expense | $ | $ 9.4 |
Remaining weighted-average remaining vesting period | 2 years 4 months 24 days |
Net loss per share (Details)
Net loss per share (Details) - shares | 9 Months Ended | |
Sep. 30, 2021 | Sep. 30, 2020 | |
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Antidilutive securities | 6,510,200 | 4,130,891 |
Unvested restricted common stock awards | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Antidilutive securities | 156,863 | 117,647 |
Unvested restricted common stock units | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Antidilutive securities | 947,265 | |
Stock options | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Antidilutive securities | 5,406,072 | 4,013,244 |
Net loss per share - Basic and
Net loss per share - Basic and diluted weighted average shares outstanding (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2021 | Sep. 30, 2020 | Sep. 30, 2021 | Sep. 30, 2020 | |
Numerator: | ||||
Net (loss) income | $ (25,137) | $ 85,611 | $ (76,906) | $ 52,667 |
Denominator for basic net (loss) income per share: | ||||
Weighted average shares outstanding-basic | 37,780,547 | 37,242,504 | 37,623,309 | 37,079,242 |
Denominator for diluted net (loss) income per share: | ||||
Weighted average shares outstanding-basic | 37,780,547 | 37,242,504 | 37,623,309 | 37,079,242 |
Common stock options and restricted stock units | 429,824 | 420,913 | ||
Weighted average shares outstanding-diluted | 37,780,547 | 37,672,328 | 37,623,309 | 37,500,155 |
Related party transactions (Det
Related party transactions (Details) - USD ($) | 3 Months Ended | 9 Months Ended | |||
Sep. 30, 2021 | Sep. 30, 2020 | Sep. 30, 2021 | Sep. 30, 2020 | Dec. 31, 2020 | |
Related party transactions | |||||
Related party collaboration receivable | $ 1,165,000 | $ 1,165,000 | $ 8,012,000 | ||
Dr. Sah | Board and scientific advisory services | |||||
Related party transactions | |||||
Total amount of services received | 30,000 | $ 200,000 | 120,000 | $ 300,000 | |
Neurocrine | Collaboration arrangement | |||||
Related party transactions | |||||
Related party collaboration receivable | $ 1,200,000 | $ 1,200,000 |
Subsequent events - (Details)
Subsequent events - (Details) - Pfizer - Option and license agreement - Subsequent Event $ in Millions | Oct. 01, 2021USD ($)item |
Subsequent events | |
Upfront payment | $ 30 |
Number of exercise options | item | 2 |
Aggregate milestone payments, if exercise rights | $ 10 |
Development, regulatory and commercialization milestone | |
Subsequent events | |
Aggregate maximum milestone payments to be received from collaborative partner | 115 |
Sales milestone | |
Subsequent events | |
Aggregate maximum milestone payments to be received from collaborative partner | $ 175 |