Document and Entity Information
Document and Entity Information - shares | 6 Months Ended | |
Jun. 30, 2022 | Aug. 01, 2022 | |
Document and Entity Information | ||
Document Type | 10-Q | |
Document Quarterly Report | true | |
Document Transition Report | false | |
Document Period End Date | Jun. 30, 2022 | |
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 | 64 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, $0.001 par value | |
Trading Symbol | VYGR | |
Security Exchange Name | NASDAQ | |
Entity Current Reporting Status | Yes | |
Entity Interactive Data Current | Yes | |
Entity Filer Category | Accelerated Filer | |
Entity Small Business | true | |
Entity Emerging Growth Company | false | |
Entity Shell Company | false | |
Entity Central Index Key | 0001640266 | |
Amendment Flag | false | |
Current Fiscal Year End Date | --12-31 | |
Document Fiscal Year Focus | 2022 | |
Document Fiscal Period Focus | Q2 | |
Entity Common Stock, Shares Outstanding | 38,647,592 |
Condensed Consolidated Balance
Condensed Consolidated Balance Sheets - USD ($) $ in Thousands | Jun. 30, 2022 | Dec. 31, 2021 |
Current assets: | ||
Cash and cash equivalents | $ 78,329 | $ 117,433 |
Marketable securities, current | 69,727 | 15,106 |
Related party collaboration receivable | 151 | 732 |
Prepaid expenses and other current assets | 3,421 | 3,427 |
Total current assets | 151,628 | 136,698 |
Property and equipment, net | 19,071 | 21,920 |
Deposits and other non-current assets | 1,515 | 1,779 |
Operating lease, right-of-use asset | 16,391 | 33,458 |
Total assets | 188,605 | 193,855 |
Current liabilities: | ||
Accounts payable | 1,218 | 574 |
Accrued expenses | 7,185 | 10,950 |
Other current liabilities | 2,526 | 5,571 |
Deferred revenue, current | 87,003 | 33,886 |
Total current liabilities | 97,932 | 50,981 |
Deferred revenue, non-current | 7,880 | 8,210 |
Other non-current liabilities | 22,741 | 39,609 |
Total liabilities | 128,553 | 98,800 |
Commitments and contingencies (see note 8) | ||
Stockholders' equity: | ||
Preferred stock $0.001 par value: 5,000,000 shares authorized at June 30, 2022 and December 31, 2021; no shares issued and outstanding at June 30, 2022 and December 31, 2021 | ||
Common stock, $0.001 par value: 120,000,000 shares authorized at June 30, 2022 and December 31, 2021; 38,439,251 and 37,918,395 shares issued and outstanding at June 30, 2022 and December 31, 2021, respectively | 38 | 38 |
Additional paid-in capital | 447,888 | 442,259 |
Accumulated other comprehensive loss | (364) | (138) |
Accumulated deficit | (387,510) | (347,104) |
Total stockholders' equity | 60,052 | 95,055 |
Total liabilities and stockholders' equity | $ 188,605 | $ 193,855 |
Condensed Consolidated Balanc_2
Condensed Consolidated Balance Sheets (Parenthetical) - $ / shares | Jun. 30, 2022 | Dec. 31, 2021 |
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 | 38,439,251 | 37,918,395 |
Common Stock, Shares, Outstanding | 38,439,251 | 37,918,395 |
Condensed Consolidated Statemen
Condensed Consolidated Statements of Operations and Comprehensive Loss - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2022 | Jun. 30, 2021 | Jun. 30, 2022 | Jun. 30, 2021 | |
Condensed Consolidated Statements of Operations and Comprehensive Loss | ||||
Collaboration revenue | $ 712 | $ 1,357 | $ 1,371 | $ 7,858 |
Operating expenses: | ||||
Research and development | 12,527 | 19,505 | 26,876 | 41,851 |
General and administrative | 7,552 | 10,437 | 15,211 | 20,181 |
Total operating expenses | 20,079 | 29,942 | 42,087 | 62,032 |
Operating loss | (19,367) | (28,585) | (40,716) | (54,174) |
Other income (expense) : | ||||
Interest income | 219 | 48 | 271 | 132 |
Other (expense) income | 61 | (1,583) | 39 | 2,273 |
Total other income (expense), net | 280 | (1,535) | 310 | 2,405 |
Net loss | (19,087) | (30,120) | (40,406) | (51,769) |
Other comprehensive (loss) income | ||||
Net unrealized (loss) gain on available-for-sale securities | (141) | (3) | (226) | 8 |
Total other comprehensive (loss) income | (141) | (3) | (226) | 8 |
Comprehensive loss | $ (19,228) | $ (30,123) | $ (40,632) | $ (51,761) |
Net loss per share, basic | $ (0.50) | $ (0.80) | $ (1.06) | $ (1.38) |
Net loss per share, diluted | $ (0.50) | $ (0.80) | $ (1.06) | $ (1.38) |
Weighted-average common shares outstanding, basic | 38,298,426 | 37,581,381 | 38,183,192 | 37,543,387 |
Weighted-average common shares outstanding, diluted | 38,298,426 | 37,581,381 | 38,183,192 | 37,543,387 |
Condensed Consolidated Statem_2
Condensed Consolidated Statements of Stockholders' Equity - USD ($) $ in Thousands | Common Stock | Additional Paid-in Capital | Accumulated Other Comprehensive Income (Loss) | Accumulated Deficit | Total |
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 | 11 | 11 | |||
Net loss | (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 | ||||
Unrealized gain (loss) on available-for-sale securities | 8 | ||||
Net loss | (51,769) | ||||
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 | ||||
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 | (3) | (3) | |||
Net loss | (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 | ||||
Stockholders' Equity, Beginning Balance at Dec. 31, 2021 | $ 38 | 442,259 | (138) | (347,104) | 95,055 |
Shares, Outstanding, Beginning Balance at Dec. 31, 2021 | 37,918,395 | ||||
Exercises of vested stock options | 12 | 12 | |||
Exercises of vested stock options (in shares) | 11,484 | ||||
Vesting of restricted stock units (in shares) | 312,090 | ||||
Stock based compensation expense | 2,268 | 2,268 | |||
Unrealized gain (loss) on available-for-sale securities | (85) | (85) | |||
Net loss | (21,319) | (21,319) | |||
Stockholders' Equity, Ending Balance at Mar. 31, 2022 | $ 38 | 444,539 | (223) | (368,423) | 75,931 |
Shares, Outstanding, Ending Balance at Mar. 31, 2022 | 38,241,969 | ||||
Stockholders' Equity, Beginning Balance at Dec. 31, 2021 | $ 38 | 442,259 | (138) | (347,104) | $ 95,055 |
Shares, Outstanding, Beginning Balance at Dec. 31, 2021 | 37,918,395 | ||||
Exercises of vested stock options (in shares) | 74,496 | ||||
Unrealized gain (loss) on available-for-sale securities | $ (226) | ||||
Net loss | (40,406) | ||||
Stockholders' Equity, Ending Balance at Jun. 30, 2022 | $ 38 | 447,888 | (364) | (387,510) | 60,052 |
Shares, Outstanding, Ending Balance at Jun. 30, 2022 | 38,439,251 | ||||
Stockholders' Equity, Beginning Balance at Mar. 31, 2022 | $ 38 | 444,539 | (223) | (368,423) | 75,931 |
Shares, Outstanding, Beginning Balance at Mar. 31, 2022 | 38,241,969 | ||||
Exercises of vested stock options | 575 | 575 | |||
Exercises of vested stock options (in shares) | 63,012 | ||||
Vesting of restricted stock units (in shares) | 32,165 | ||||
Issuance of common stock under ESPP | 313 | 313 | |||
Issuance of common stock under ESPP (in shares) | 102,105 | ||||
Stock based compensation expense | 2,460 | 2,460 | |||
Unrealized gain (loss) on available-for-sale securities | (141) | (141) | |||
Net loss | (19,087) | (19,087) | |||
Stockholders' Equity, Ending Balance at Jun. 30, 2022 | $ 38 | $ 447,888 | $ (364) | $ (387,510) | $ 60,052 |
Shares, Outstanding, Ending Balance at Jun. 30, 2022 | 38,439,251 |
Condensed Consolidated Statem_3
Condensed Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 6 Months Ended | |
Jun. 30, 2022 | Jun. 30, 2021 | |
Cash flow from operating activities | ||
Net loss | $ (40,406) | $ (51,769) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Stock-based compensation expense | 4,809 | 7,592 |
Depreciation | 4,016 | 2,459 |
Amortization of premiums and discounts on marketable securities | 2 | 213 |
Other non-cash items | (2,469) | (2,460) |
Changes in operating assets and liabilities: | ||
Related party collaboration receivable | 581 | 6,254 |
Prepaid expenses and other current assets | 6 | 893 |
Operating lease, right-of-use asset | 2,556 | 1,922 |
Other non-current assets | 69 | |
Accounts payable | 644 | 156 |
Accrued expenses | (3,652) | (3,357) |
Operating lease liabilities | (2,647) | (1,891) |
Other non-current liabilities | (287) | |
Deferred revenue | 52,787 | (4,092) |
Net cash provided by (used in) operating activities | 15,941 | (44,011) |
Cash flow from investing activities | ||
Purchases of property and equipment | (1,280) | (602) |
Purchases of marketable securities | (54,848) | |
Proceeds from sales and maturities of marketable securities | 57,632 | |
Net cash (used in) provided by investing activities | (56,128) | 57,030 |
Cash flow from financing activities | ||
Proceeds from the exercise of stock options | 587 | 28 |
Proceeds from the purchase of common stock under ESPP | 232 | 357 |
Net cash provided by financing activities | 819 | 385 |
Net (decrease) increase in cash, cash equivalents, and restricted cash | (39,368) | 13,404 |
Cash, cash equivalents, and restricted cash beginning of period | 119,212 | 106,219 |
Cash, cash equivalents, and restricted cash end of period | 79,844 | 119,623 |
Supplemental disclosure of cash and non-cash activities | ||
Capital expenditures incurred but not yet paid | $ 113 | $ 111 |
Nature of business
Nature of business | 6 Months Ended |
Jun. 30, 2022 | |
Nature of business | |
Nature of business | 1. Nature of business Voyager Therapeutics, Inc. (the “Company”) is a gene therapy and neuroscience company developing life-changing treatments and next-generation adeno-associated virus (“AAV”) capsids. The Company focuses on diseases where it believes a single dose AAV gene therapy or an antibody can either halt or slow disease progression or reduce 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 it believes have the potential to safely provide durable efficacy. The Company’s team of experts in the field of AAV gene therapy and neuroscience first identifies and selects diseases in which the Company believes an AAV gene therapy or an antibody will answer a high unmet medical need, be supported by target validation, offer an efficient path to human proof of biology, present robust preclinical pharmacology, and offer strong commercial potential. The Company then engineers and optimizes either an AAV vector for delivery of the virus payload to the targeted tissue or cells or a passive antibody for systemic delivery. The Company is identifying proprietary AAV capsids, the outer viral protein shells that enclose genetic material of a virus payload. The Company’s team has developed a proprietary AAV capsid discovery platform called TRACER TM in vivo The Company is also applying the TRACER discovery platform to identify capsid variant libraries and facilitate the selection of capsids with tropism and transduction in additional cell and tissue types. The Company is actively engaged in discussions with multiple parties to make TRACER capsids available to third parties for use in their drug development programs through potential licensing and other arrangements. In addition to the Company’s TRACER discovery platform, the Company has developed a vectorized antibody platform which the Company believes will overcome many of the challenges of passive immunization. However, the Company has also seen promising preclinical results from the passive immunization approach and the Company is investigating both passive immunization and vectorized approaches to administer proprietary antibodies that selectively target pathological tau to address tauopathies. The Company’s business strategy focuses on discovering, developing, manufacturing and commercializing its gene therapy and antibody programs. As part of this strategy, the Company has developed core competencies specific to AAV gene therapy and antibody development and manufacturing. This business strategy also includes business development activities that may include in-licensing activities or partnering certain programs in specific 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 TRACER capsids such as the Company’s October 2021 licensing agreement with Pfizer Inc. The Company is devoting substantially all of its efforts to product research and development, market development, and raising capital. The Company is subject to risks common to companies in the biotechnology and gene therapy industries, including but not limited to, the need to obtain sufficient capital to continue to fund its operations, risks of failure of preclinical studies and clinical trials, the need to obtain marketing approval for its product candidates, the need to successfully commercialize and gain market acceptance of its product candidates, dependence on key personnel, protection of proprietary information and technology, protection against data breaches and other cybersecurity threats, compliance with government regulations, development by competitors of technological innovations, and ability to transition from pilot-scale manufacturing to large-scale production of products. The Company has a history of incurring annual net operating losses. As of June 30, 2022, the Company had an accumulated deficit of $387.5 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 fees, milestone payments, and cost reimbursements associated with 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, its licensing agreement with Pfizer, and its licensing agreement with Novartis. Through June 30, 2022, the Company has raised approximately $724.0 million of proceeds from sales of convertible preferred stock and common stock, including its initial public offering and follow-on public offering, and from its collaboration and license agreements. As of June 30, 2022, the Company had cash, cash equivalents, and marketable securities of $148.1 million. Based upon its current operating plan, the Company expects that its existing cash, cash equivalents, and marketable securities at June 30, 2022, together with amounts expected to be received as reimbursement for development costs under the Neurocrine Collaboration Agreement, 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 on terms acceptable to the Company or generate product revenue or revenue from collaboration partners, 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 | 6 Months Ended |
Jun. 30, 2022 | |
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. 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, 2021 as filed with the Securities and Exchange Commission (“SEC”) on March 8, 2022. 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 heading “Summary of Significant Accounting Policies - 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, 2021. 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, 2021. |
Fair value measurements
Fair value measurements | 6 Months Ended |
Jun. 30, 2022 | |
Fair value measurements | |
Fair value measurements | 3. Fair value measurements Assets and liabilities measured at fair value on a recurring basis as of June 30, 2022 and December 31, 2021 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) June 30, 2022 (in thousands) Money market funds included in cash and cash equivalents $ 75,562 $ 75,562 $ — $ — Marketable securities - U.S. Treasury notes 69,727 69,727 — — Total $ 145,288 $ 145,288 $ — $ — December 31, 2021 Money market funds included in cash and cash equivalents $ 100,305 $ 100,305 $ — $ — Marketable securities - U.S. Treasury notes 15,106 15,106 Total $ 115,411 $ 115,411 $ — $ — The Company measures the fair value of money market funds and U.S. Treasury notes based on quoted prices in active markets for identical securities. |
Cash, cash equivalents, restric
Cash, cash equivalents, restricted cash, and available-for-sale marketable securities | 6 Months Ended |
Jun. 30, 2022 | |
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 June 30, 2022 and December 31, 2021: Amortized Unrealized Unrealized Fair Cost Gains Losses Value (in thousands) As of June 30, 2022 Money market funds included in cash and cash equivalents $ 75,562 $ — $ — $ 75,562 Marketable securities - U.S. Treasury notes 69,962 — (236) 69,727 Total money market funds and marketable securities $ 145,524 $ — $ (236) $ 145,288 As of December 31, 2021 Money market funds included in cash and cash equivalents $ 100,305 — — $ 100,305 Marketable securities - U.S. Treasury notes 15,117 — (11) 15,106 Total money market funds and marketable securities $ 115,422 $ — $ (11) $ 115,411 All of the Company’s marketable securities as of June 30, 2022, have a contractual maturity of one year or less. The Company reviews investments whenever the fair value of an investment is less than the amortized cost and evidence indicates that an investment’s carrying amount is not recoverable within a reasonable period of time. In connection with these investments, the Company evaluates whether the decline in fair value has resulted from credit losses or other factors, considering the extent to which fair value is less than amortized cost, any changes to the rating of the security by a rating agency, and adverse conditions specifically related to the security, among other factors. If this assessment indicates that a credit loss exists, the present value of cash flows expected to be collected from the security is compared to the amortized cost basis of the security. If the present value of cash flows expected to be collected is less than the amortized cost basis, a credit loss exists and an allowance for credit losses is recorded for the credit loss on the condensed consolidated balance sheet, limited by the amount that the fair value is less than the amortized cost basis. Any impairment that is not related to credit is recognized in other comprehensive (loss) income. Changes in the allowance for credit losses are recorded as a provision for (or reversal of) credit loss expense in general and administrative expenses within the condensed consolidated statement of operations. Losses are charged against the allowance when the Company believes the uncollectability of an available-for-sale security is confirmed or when either of the criteria regarding intent or requirement to sell is met. The Company held $70.0 million and $15.1 million marketable securities that were in an unrealized loss position as of June 30, 2022 and December 31, 2021, respectively. The unrealized losses at June 30, 2022 and December 31, 2021 were attributable to changes in interest rates and the unrealized losses do not represent credit losses. The Company does not intend to sell these securities and it is not more likely than not that it will be required to sell them before recovery of their amortized cost basis. 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 June 30, As of December 31, 2022 2021 (in thousands) Cash and cash equivalents $ 78,329 $ 117,433 Restricted cash included in deposits and other non-current assets 1,515 1,779 Total cash, cash equivalents, and restricted cash $ 79,844 $ 119,212 |
Accrued expenses
Accrued expenses | 6 Months Ended |
Jun. 30, 2022 | |
Accrued expenses | |
Accrued expenses | 5. Accrued expenses Accrued expenses as of June 30, 2022 and December 31, 2021 consist of the following: As of June 30, As of December 31, 2022 2021 (in thousands) Employee compensation costs $ 2,800 $ 5,022 Research and development costs 3,662 3,719 Professional services 344 727 Accrued goods and services 379 1,482 Total $ 7,185 $ 10,950 |
Lease obligation
Lease obligation | 6 Months Ended |
Jun. 30, 2022 | |
Lease obligation | |
Lease obligation | 6. Lease obligation Operating Leases As of June 30, 2022, the Company has a lease for office and lab space at 64 Sidney Street in Cambridge, Massachusetts through November 30, 2026 and a lease for additional laboratory and office space at 75 Hayden Avenue in Lexington, Massachusetts through January 31, 2031. In September 2021, the Company entered into an agreement with BioNTech US, Inc. (“BioNTech US”) to sublease part of the office and lab space leased by the Company at 75 Sidney Street in Cambridge, Massachusetts (the “Sublease Agreement”) at that time. The sublease term was for approximately 3.3 years. 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. On June 22, 2022 the Company entered into a Lease Termination Agreement (the “Lease Termination Agreement”) and terminated the lease for office and lab space at 75 Sidney Street (the “75 Sidney Street Lease”), effective immediately. The 75 Sidney Street Lease was previously scheduled to terminate, in accordance with its terms, on November 30, 2026. In connection with the Lease Termination Agreement, the Company also entered into a Sublease Termination Agreement (the “Sublease Termination Agreement”) and terminated the Sublease Agreement with BioNTech US. The Company did not incur any termination penalties in connection with the Lease Termination Agreement or Sublease Termination Agreement. The Company derecognized the related right-of-use asset of approximately $14.5 million and the operating lease liabilities of $17.0 million, accordingly, resulting in a gain of $2.5 million in the three-month period ended June 30, 2022. 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. Approximately $0.3 million relates to the letter of credit held for the 75 Sidney Street Lease, which is included in prepaid expenses and other current assets in the accompanying condensed balance sheets. The remaining $1.5 million is recorded as restricted cash and included in deposits and other non-current assets in the accompanying condensed consolidated balance sheets. During the three and six months ended June 30, 2022, the Company incurred lease expenses of $1.4 million and $2.8 million, respectively, for operating leases. During the three and six months ended June 30, 2021, the Company incurred lease expenses of $1.9 million and $3.7 million, respectively, for operating leases. As of June 30, 2022, the weighted average remaining lease term was 6.5 years and the weighted average incremental borrowing rate used to determine the operating lease liability was 7.4%. The following table summarizes the operating sublease income generated under the Sublease Agreement for the three and six months ended June 30, 2022 and 2021: Three Months Ended Six Months Ended June 30, June 30, 2022 2021 2022 2021 Operating sublease income (in thousands) $ 690 $ — $ 1,380 $ — |
Other liabilities
Other liabilities | 6 Months Ended |
Jun. 30, 2022 | |
Other liabilities | |
Other liabilities | 7. Other liabilities As of June 30, 2022 and December 31, 2021, other current and non-current liabilities consisted of the following: As of June 30, As of December 31, 2022 2021 (in thousands) Other current liabilities Lease liability 2,526 5,571 Total other current liabilities $ 2,526 $ 5,571 Other non-current liabilities Lease liability $ 21,741 $ 38,608 Other 1,000 1,001 Total other non-current liabilities $ 22,741 $ 39,609 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 TRACER capsid development efforts. During the year ended December 31, 2021, the Company incurred restructuring costs of approximately $2.6 million, which consists of severance-related costs. Substantially all costs have been paid as of June 30, 2022. |
Commitments and contingencies
Commitments and contingencies | 6 Months Ended |
Jun. 30, 2022 | |
Commitments and contingencies | |
Commitments and contingencies | 8. Commitments and contingencies Significant Agreements Neurocrine Collaboration Agreement Summary of Agreement In March 2019, the Company entered into the Neurocrine Collaboration Agreement for the research, development and commercialization of certain of its AAV gene therapy products. Under the Neurocrine Collaboration Agreement, the Company agreed to collaborate on the conduct of four collaboration programs (the “Neurocrine Programs”) which include: (i) VY-AADC (NBIb-1817) for Parkinson’s disease (the “VY-AADC Program”), (ii) 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, Neurocrine is no longer 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. Upon 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 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. Under the Neurocrine Collaboration Agreement, the parties committed to agree 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 Company and Neurocrine completed the nomination process, and the JSC has approved two Targets for development under the Discovery Programs. The two Targets are currently under development. 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, which the Company is no longer eligible to receive in light of the partial termination of the Neurocrine Collaboration Agreement; (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. 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. 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 cost 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. During the fourth quarter of 2021, the Company further revised the estimate of the expected reimbursement to approximately $80 million based on expectations resulting from decisions made at the JSC meeting in the fourth quarter of 2021, which are expected to result in significantly less research and development services being provided by the Company under the Neurocrine Collaboration Agreement. As of June 30, 2022, the estimate of the expected reimbursement is $79.7 million based on expectations as of such date. Additional consideration to be paid to the Company upon reaching certain milestones are excluded from the transaction price at inception due to the uncertainty of achieving the development and regulatory milestones. 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, which 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 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 June 30, 2022 Performance Obligation Amount (in thousands) Variable Consideration VY-AADC Program $ 53,863 FA Program 17,088 Discovery Program 1 5,355 Discovery Program 2 3,362 Total $ 79,668 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 six months ended June 30, 2022, the Company recognized $0.7 million and $1.4 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 six months ended June 30, 2021, the Company recognized $1.4 million and $7.9 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 six months ended June 30, 2022: Balance at Balance at December 31, 2021 Additions Deductions June 30, 2022 ( in thousands) Related party collaboration receivable $ 732 $ 358 (939) $ 151 Contract liabilities: Deferred revenue $ 12,096 $ - $ (1,214) $ 10,881 The change in the receivable balance for the six months ended June 30, 2022 is primarily driven by amounts owed to the Company for research and development services provided, offset by amounts collected from Neurocrine during the period. 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 six months ended June 30, 2022. 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. Pfizer Option and License Agreement Summary of Agreement On October 1, 2021, the Company entered into an option and license agreement with Pfizer (the “Pfizer License Agreement”), pursuant to which the Company has granted Pfizer options to receive an exclusive license (the Pfizer License Options”) to certain TRACER capsids to develop and commercialize certain AAV gene therapy candidates comprised of a capsid and specified Pfizer transgenes (the “Pfizer Transgenes”). Under the terms of the Pfizer License Agreement, Pfizer intends to evaluate the potential use of the capsids in combination with up to two Pfizer Transgenes to help treat respective central nervous system and cardiovascular diseases. Under the Pfizer License Agreement, the Company has agreed to provide Pfizer with certain quantities of materials encoding specified existing capsids for Pfizer’s evaluation. During the research term, which extends until October 1, 2022, or, in the event Pfizer exercises a Pfizer License Option, until October 1, 2024, the Company may, at its sole discretion and expense, conduct additional research activities to identify additional proprietary capsids that may be useful for AAV gene therapies for the treatment of central nervous system or cardiovascular diseases. The Company has agreed to disclose to Pfizer, on a rolling basis, the performance characteristics identified during the research term for all such capsid candidates. Following such disclosure, Pfizer has the right, in its sole discretion, to select any capsid candidate for evaluation to determine its interest in exercising a Pfizer License Option with respect to such capsid candidate. Pfizer may exercise up to two Pfizer License Options, provided that it may exercise only one Pfizer License Option for each Pfizer Transgene. The Company has granted Pfizer, effective upon Pfizer’s exercise of a Pfizer License Option, with respect to a capsid candidate for the Pfizer Transgene identified therein, an exclusive, worldwide license, with the right to sublicense, under certain of the Company’s intellectual property, the rights to develop and commercialize the applicable licensed capsid as incorporated into products containing the corresponding Pfizer Transgene (the “Pfizer Licensed Products”). Additionally, upon such Pfizer License Option exercise, the Company and Pfizer have agreed that the Company shall provide certain additional know-how that has not been previously provided to Pfizer to enable Pfizer to exploit such licensed capsid and the corresponding Pfizer Transgene for use in a Pfizer Licensed Product. Upon the exercise of a Pfizer License Option, until October 1, 2024, while the Company is not obligated to conduct additional research activities upon option exercise to identify additional proprietary capsids that may be useful for AAV gene therapies for the treatment of central nervous system or cardiovascular diseases, it has agreed to continue to disclose to Pfizer, on a rolling basis, the performance characteristics identified for all such capsid candidates, if and when available. Pfizer may, during the research term conduct additional evaluation of such capsid candidates and has the right to substitute any other capsid candidate for the capsid it previously elected to license when it exercised the Pfizer License Option. Under the Pfizer License Agreement, Pfizer is solely responsible for, and has sole decision-making authority with respect to, development and commercialization of the Pfizer Licensed Products. In the event Pfizer exercises a Pfizer License Option, Pfizer is required to use commercially reasonable efforts to develop and obtain regulatory approval for at least one Pfizer Licensed Product for each Pfizer Transgene for which Pfizer has exercised its Pfizer License Option in (i) the United States and (ii) at least one of the following countries: the United Kingdom, France, Germany, Italy, Spain and Japan (each of which, a “Pfizer Major Market Country”), subject to certain limitations. Pfizer is also required to use commercially reasonable efforts to commercialize each Pfizer Licensed Product in the United States and at least one Pfizer Major Market Country where Pfizer or its designated affiliates or sublicensees has received regulatory approval for such Pfizer Licensed Product, subject to certain limitations. Under the terms of the Pfizer License Agreement, Pfizer paid the Company an upfront payment of $30.0 million in October 2021. Pfizer has also agreed to pay the Company, upon each Pfizer License Option exercise, a fee of $10.0 million. Following each Pfizer License Option exercise, with respect to a Pfizer Transgene, the Company is also eligible to receive specified development, regulatory, and commercialization milestone payments of up to an aggregate of $115.0 million for the first corresponding Pfizer Licensed Product to achieve the corresponding milestone. On a Pfizer Licensed Product-by-Pfizer Licensed Product basis, the Company is also eligible to receive (a) specified sales milestone payments of up to an aggregate of $175.0 million per Pfizer Licensed Product and (b) tiered, escalating royalties in the mid- to high-single-digit percentages of annual net sales of each Pfizer Licensed Product. The royalties are subject to potential reductions in customary circumstances including patent claim expiration, payments for certain third-party licenses, and biosimilar market penetration, subject to specified limits. Under the terms of the Pfizer License Agreement, each the Company and Pfizer owns the entire right, title, and interest in and to all patents or know-how controlled by such party and existing as of or before the effective date of the Pfizer License Agreement, or invented, developed, created, generated or acquired solely by or on behalf of such party after such effective date. Subject to certain specified exceptions, any patents and know-how that are invented or otherwise developed jointly by or on behalf of the parties during the term of the Pfizer License Agreement and in the course of the Company’s and Pfizer’s activities under the Pfizer License Agreement will follow inventorship under U.S. patent law. Subject to certain limitations and exceptions, the Company agreed (i) during the research term, not to conduct any internal program or program on behalf of a third party that is directed to development or commercialization of any capsid candidates, or grant any third party or affiliate any right or license under the Company’s rights in such capsid candidates to exploit any therapeutic product, in combination with any Pfizer Transgene in any indication for therapeutic, diagnostic and prophylactic human and veterinary use; and (ii) after Pfizer’s exercise of a Pfizer License Option, not to grant any third party or affiliate any right or license under the Company’s patents to exploit any licensed capsid in combination with any Pfizer Transgene. Unless earlier terminated, the Pfizer License Agreement expires on the earlier to occur of (i) the first anniversary of the effective date of the Pfizer License Agreement, if no Pfizer License Option is exercised, and (ii) the expiration of the last-to-expire royalty term with respect to all Pfizer Licensed Products in all countries if at least one Pfizer License Option is exercised. Subject to a cure period, either party may terminate the Pfizer License Agreement, in whole or in part, subject to specified conditions, in the event of the other party’s uncured material breach. Pfizer may also terminate the Pfizer License Agreement, in whole or in part, subject to specified conditions, for the Company’s insolvency, the occurrence of a violation of global trade control laws, or for the Company’s noncompliance with certain anti-bribery or anti-corruption covenants. Pfizer may also terminate the Pfizer License Agreement, in whole or in part, for any or no reason upon ninety days’ written notice to us. Upon certain terminations for cause by Pfizer, the licenses that the Company has granted to Pfizer under the Pfizer License Agreement shall become irrevocable and perpetual, and all milestone payments and royalties that would have otherwise been payable by Pfizer under such licenses had the Pfizer License Agreement remained in effect would be substantially reduced. Accounting Analysis The Company assessed the promised goods and services under the Pfizer License Agreement, in accordance with ASC 606, and determined that the Pfizer License Agreement contains two performance obligations consisting of two material rights, one for each of the Pfizer License Options. The Company concluded that each Pfizer License Option provides a material right as consideration for each option is less than the amount that the Company would otherwise have expected to receive outside the context of the contract. The promises at inception do not include the underlying goods or services that would be delivered upon exercise of the option, but rather represent the value to the customer of having the right to exercise the Pfizer License Option at the specified exercise fee. Upon the exercise of a Pfizer License Option, until October 1, 2024, while the Company is not obligated to conduct additional research activities upon option exercise to identify additional proprietary capsids that may be useful for AAV gene therapies for the treatment of central nervous system or cardiovascular diseases, it has agreed to continue to disclose to Pfizer, on a rolling basis, the performance characteristics identified for all such capsid candidates, if and when available. Pfizer may, conduct additional evaluation of such capsid candidates and has the right to substitute any other capsid candidate for the capsid it previously elected to license when it exercised the Pfizer License Option. The Company determined that this promise to provide Pfizer the ability to evaluate and potentially substitute other capsid candidates for the capsid it previously elected to license when it exercised the Pfizer License Option, if and when available, is an additional performance obligation in the arrangement (“the Pfizer Substitution Right Performance Obligation”). The Company received a nonrefundable, upfront payment of $30.0 million as consideration under the Pfizer License Agreement, which represents the transaction price at inception. Additional consideration to be paid to the Company upon exercise of the Pfizer License Options or upon reaching certain milestones are excluded from the transaction price as they relate to option fees and milestones that could only be achieved subsequent to an option exercise. The Company allocated the transaction price to the two material rights based on their relative standalone selling prices. The estimated standalone selling price for each material right was based on an adjusted market assessment approach. The Company concluded that the market would be willing to pay an equal amount for each Pfizer License Option on a standalone basis. The Company reached th |
Stock-based compensation
Stock-based compensation | 6 Months Ended |
Jun. 30, 2022 | |
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 loss is as follows: Three Months Ended Six Months Ended June 30, June 30, 2022 2021 2022 2021 (in thousands) Research and development $ 781 $ 1,336 $ 1,582 $ 2,706 General and administrative 1,709 2,655 3,227 4,886 Total stock-based compensation expense $ 2,490 $ 3,991 $ 4,809 $ 7,592 Stock-based compensation expense by type of award included within the condensed consolidated statements of operations and comprehensive loss was as follows: Three Months Ended Six Months Ended June 30, June 30, 2022 2021 2022 2021 (in thousands) Stock options $ 1,618 $ 2,480 $ 3,095 $ 5,002 Restricted stock awards and units 842 1,392 1,634 2,366 Employee stock purchase plan awards 30 120 81 223 Total stock-based compensation expense $ 2,490 $ 3,991 4,809 7,592 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 six months ended June 30, 2022 was as follows: Weighted Average Grant Date Fair Value Units Per Unit Unvested restricted stock units as of December 31, 2021 806,379 $ 7.26 Granted 739,080 $ 3.79 Vested (344,255) 7.45 Forfeited (169,839) $ 5.06 Unvested restricted stock units as of June 30, 2022 1,031,365 $ 5.08 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. All of the restricted stock units granted in the six months ended June 30, 2022 vest in equal amounts, annually over three As of June 30, 2022, the Company had unrecognized stock-based compensation expense related to its unvested restricted stock units of $4.0 million, which is expected to be recognized over the remaining average vesting period of 2.0 years. Stock Options The following is a summary of stock option activity for the six months ended June 30, 2022: Weighted Remaining Aggregate Average Contractual Intrinsic Exercise Life Value Shares Price (in years) (in thousands) Outstanding at December 31, 2021 5,013,193 $ 12.69 5.6 $ 1,165 Granted 2,965,075 $ 4.95 Exercised (74,496) $ 7.90 Cancelled or forfeited (1,274,172) $ 12.42 Outstanding at June 30, 2022 6,629,600 $ 9.33 7.2 $ 5,914 Exercisable at June 30, 2022 3,378,945 $ 12.29 5.3 $ 1,575 As of June 30, 2022, the Company had unrecognized stock-based compensation expense related to its unvested stock options of $13.0 million which is expected to be recognized over the remaining weighted-average vesting period of 3.1 years. |
Net loss per share
Net loss per share | 6 Months Ended |
Jun. 30, 2022 | |
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 June 30, 2022 2021 Unvested restricted common stock awards 137,255 156,863 Unvested restricted common stock units 1,031,365 1,170,702 Outstanding stock options 6,629,600 5,919,375 Total 7,798,220 7,246,940 Basic net loss per share for the three and six months ended June 30, 2022 and 2021 is the same as diluted net loss per share as shown on the Company’s condensed consolidated statement of operations. |
Related party transactions
Related party transactions | 6 Months Ended |
Jun. 30, 2022 | |
Related party transactions | |
Related party transactions | 11. Related-party transactions During the three and six months ended June 30, 2022, the Company received scientific advisory board and other 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 six months ended June 30, 2022, was $82,425 and $92,325, respectively . During the six months ended June 30, 2022, the Company received advisory services related to strategic planning, operations, and management from Alfred Sandrock, M.D., Ph.D., the Company’s current President and Chief Executive Officer and a member of the Company’s Board of Directors, before he commenced service in the capacity of President and Chief Executive Officer in March 2022. The total amount of fees paid to Dr. Sandrock for services provided was $60,000 for the six months ended June 30, 2022. 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 June 30, 2022, the Company had approximately $0.2 million in related party collaboration receivables associated with Neurocrine. |
Summary of significant accoun_2
Summary of significant accounting policies (Policies) | 6 Months Ended |
Jun. 30, 2022 | |
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. 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, 2021 as filed with the Securities and Exchange Commission (“SEC”) on March 8, 2022. 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 heading “Summary of Significant Accounting Policies - 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, 2021. 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. |
Summary of Significant Accounting Policies | 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, 2021. |
Fair value measurements (Tables
Fair value measurements (Tables) | 6 Months Ended |
Jun. 30, 2022 | |
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 June 30, 2022 and December 31, 2021 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) June 30, 2022 (in thousands) Money market funds included in cash and cash equivalents $ 75,562 $ 75,562 $ — $ — Marketable securities - U.S. Treasury notes 69,727 69,727 — — Total $ 145,288 $ 145,288 $ — $ — December 31, 2021 Money market funds included in cash and cash equivalents $ 100,305 $ 100,305 $ — $ — Marketable securities - U.S. Treasury notes 15,106 15,106 Total $ 115,411 $ 115,411 $ — $ — |
Cash, cash equivalents, restr_2
Cash, cash equivalents, restricted cash, and available-for-sale marketable securities (Tables) | 6 Months Ended |
Jun. 30, 2022 | |
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 June 30, 2022 and December 31, 2021: Amortized Unrealized Unrealized Fair Cost Gains Losses Value (in thousands) As of June 30, 2022 Money market funds included in cash and cash equivalents $ 75,562 $ — $ — $ 75,562 Marketable securities - U.S. Treasury notes 69,962 — (236) 69,727 Total money market funds and marketable securities $ 145,524 $ — $ (236) $ 145,288 As of December 31, 2021 Money market funds included in cash and cash equivalents $ 100,305 — — $ 100,305 Marketable securities - U.S. Treasury notes 15,117 — (11) 15,106 Total money market funds and marketable securities $ 115,422 $ — $ (11) $ 115,411 |
Reconciliation of cash, cash equivalents, and restricted cash | As of June 30, As of December 31, 2022 2021 (in thousands) Cash and cash equivalents $ 78,329 $ 117,433 Restricted cash included in deposits and other non-current assets 1,515 1,779 Total cash, cash equivalents, and restricted cash $ 79,844 $ 119,212 |
Accrued expenses (Tables)
Accrued expenses (Tables) | 6 Months Ended |
Jun. 30, 2022 | |
Accrued expenses | |
Schedule of accrued expenses | As of June 30, As of December 31, 2022 2021 (in thousands) Employee compensation costs $ 2,800 $ 5,022 Research and development costs 3,662 3,719 Professional services 344 727 Accrued goods and services 379 1,482 Total $ 7,185 $ 10,950 |
Lease obligation (Tables)
Lease obligation (Tables) | 6 Months Ended |
Jun. 30, 2022 | |
Lease obligation | |
Summary of operating sublease income | Three Months Ended Six Months Ended June 30, June 30, 2022 2021 2022 2021 Operating sublease income (in thousands) $ 690 $ — $ 1,380 $ — |
Other liabilities (Tables)
Other liabilities (Tables) | 6 Months Ended |
Jun. 30, 2022 | |
Other liabilities | |
Summary of other current and non-current liabilities | As of June 30, As of December 31, 2022 2021 (in thousands) Other current liabilities Lease liability 2,526 5,571 Total other current liabilities $ 2,526 $ 5,571 Other non-current liabilities Lease liability $ 21,741 $ 38,608 Other 1,000 1,001 Total other non-current liabilities $ 22,741 $ 39,609 |
Commitments and contingencies (
Commitments and contingencies (Tables) | 6 Months Ended |
Jun. 30, 2022 | |
Schedule of collaboration receivables and contract liabilities | Balance at Balance at December 31, 2021 Additions Deductions June 30, 2022 ( in thousands) Related party collaboration receivable $ 732 $ 358 (939) $ 151 Contract liabilities: Deferred revenue $ 12,096 $ - $ (1,214) $ 10,881 |
Neurocrine Collaboration Agreement | |
Schedule of allocation of variable consideration | Performance Obligation Amount (in thousands) Variable Consideration VY-AADC Program $ 53,863 FA Program 17,088 Discovery Program 1 5,355 Discovery Program 2 3,362 Total $ 79,668 |
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) | 6 Months Ended |
Jun. 30, 2022 | |
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 loss is as follows: Three Months Ended Six Months Ended June 30, June 30, 2022 2021 2022 2021 (in thousands) Research and development $ 781 $ 1,336 $ 1,582 $ 2,706 General and administrative 1,709 2,655 3,227 4,886 Total stock-based compensation expense $ 2,490 $ 3,991 $ 4,809 $ 7,592 |
Summary of stock-based compensation expense by type of award | Three Months Ended Six Months Ended June 30, June 30, 2022 2021 2022 2021 (in thousands) Stock options $ 1,618 $ 2,480 $ 3,095 $ 5,002 Restricted stock awards and units 842 1,392 1,634 2,366 Employee stock purchase plan awards 30 120 81 223 Total stock-based compensation expense $ 2,490 $ 3,991 4,809 7,592 |
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 six months ended June 30, 2022 was as follows: Weighted Average Grant Date Fair Value Units Per Unit Unvested restricted stock units as of December 31, 2021 806,379 $ 7.26 Granted 739,080 $ 3.79 Vested (344,255) 7.45 Forfeited (169,839) $ 5.06 Unvested restricted stock units as of June 30, 2022 1,031,365 $ 5.08 |
Summary of stock option activity | The following is a summary of stock option activity for the six months ended June 30, 2022: Weighted Remaining Aggregate Average Contractual Intrinsic Exercise Life Value Shares Price (in years) (in thousands) Outstanding at December 31, 2021 5,013,193 $ 12.69 5.6 $ 1,165 Granted 2,965,075 $ 4.95 Exercised (74,496) $ 7.90 Cancelled or forfeited (1,274,172) $ 12.42 Outstanding at June 30, 2022 6,629,600 $ 9.33 7.2 $ 5,914 Exercisable at June 30, 2022 3,378,945 $ 12.29 5.3 $ 1,575 |
Net loss per share (Tables)
Net loss per share (Tables) | 6 Months Ended |
Jun. 30, 2022 | |
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 June 30, 2022 2021 Unvested restricted common stock awards 137,255 156,863 Unvested restricted common stock units 1,031,365 1,170,702 Outstanding stock options 6,629,600 5,919,375 Total 7,798,220 7,246,940 |
Nature of business (Details)
Nature of business (Details) - USD ($) $ in Thousands | Jun. 30, 2022 | Dec. 31, 2021 |
Nature of business | ||
Accumulated deficit | $ (387,510) | $ (347,104) |
Proceeds raised | 724,000 | |
Cash, cash equivalents, and marketable debt securities | $ 148,100 |
Fair value measurements - Asset
Fair value measurements - Assets and liabilities measured on a recurring basis - (Details) - USD ($) $ in Thousands | Jun. 30, 2022 | Dec. 31, 2021 |
Assets, Fair Value Disclosure [Abstract] | ||
Cash and cash equivalents | $ 78,329 | $ 117,433 |
Fair Value, Measurements, Recurring | ||
Assets, Fair Value Disclosure [Abstract] | ||
Total assets measured at fair value | 145,288 | 115,411 |
Level 1 | Fair Value, Measurements, Recurring | ||
Assets, Fair Value Disclosure [Abstract] | ||
Total assets measured at fair value | 145,288 | 115,411 |
U.S. Treasury notes | Fair Value, Measurements, Recurring | ||
Assets, Fair Value Disclosure [Abstract] | ||
Marketable securities | 69,727 | 15,106 |
U.S. Treasury notes | Level 1 | Fair Value, Measurements, Recurring | ||
Assets, Fair Value Disclosure [Abstract] | ||
Marketable securities | 69,727 | 15,106 |
Money market funds | ||
Assets, Fair Value Disclosure [Abstract] | ||
Cash and cash equivalents | 75,562 | 100,305 |
Money market funds | Fair Value, Measurements, Recurring | ||
Assets, Fair Value Disclosure [Abstract] | ||
Cash and cash equivalents | 75,562 | 100,305 |
Money market funds | Level 1 | Fair Value, Measurements, Recurring | ||
Assets, Fair Value Disclosure [Abstract] | ||
Cash and cash equivalents | $ 75,562 | $ 100,305 |
Cash, cash equivalents, restr_3
Cash, cash equivalents, restricted cash and available-for-sale marketable securities - Cash, Cash Equivalents, and Marketable Securities - (Details) - USD ($) $ in Thousands | 6 Months Ended | |
Jun. 30, 2022 | Dec. 31, 2021 | |
Amortized Cost Basis [Abstract] | ||
Cash and Cash Equivalents, at Carrying Value | $ 78,329 | $ 117,433 |
Total money market funds and marketable securities | 145,524 | 115,422 |
Fair Value Disclosure [Abstract] | ||
Total money market funds and marketable securities | 145,288 | 115,411 |
Unrealized Gain and Loss [Abstract] | ||
Total money market funds and marketable securities | (236) | (11) |
Debt securities, unrealized loss position | 70,000 | 15,100 |
Money market funds | ||
Amortized Cost Basis [Abstract] | ||
Cash and Cash Equivalents, at Carrying Value | 75,562 | 100,305 |
U.S. Treasury notes | ||
Amortized Cost Basis [Abstract] | ||
Marketable securities, amortized cost | 69,962 | 15,117 |
Fair Value Disclosure [Abstract] | ||
Marketable securities, fair value | 69,727 | 15,106 |
Unrealized Gain and Loss [Abstract] | ||
Marketable securities, unrealized losses | $ (236) | $ (11) |
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 | Jun. 30, 2022 | Dec. 31, 2021 | Jun. 30, 2021 | Dec. 31, 2020 |
Cash, cash equivalents, restricted cash, and available-for-sale marketable securities | ||||
Cash and cash equivalents | $ 78,329 | $ 117,433 | ||
Restricted cash included in deposits and other noncurrent assets | 1,515 | 1,779 | ||
Total cash, cash equivalents, and restricted cash | $ 79,844 | $ 119,212 | $ 119,623 | $ 106,219 |
Accrued expenses (Details)
Accrued expenses (Details) - USD ($) $ in Thousands | Jun. 30, 2022 | Dec. 31, 2021 |
Accrued expenses | ||
Employee compensation costs | $ 2,800 | $ 5,022 |
Research and development costs | 3,662 | 3,719 |
Professional services | 344 | 727 |
Accrued goods and services | 379 | 1,482 |
Total | $ 7,185 | $ 10,950 |
Lease obligation (Details)
Lease obligation (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||||
Jun. 30, 2022 | Jun. 30, 2021 | Jun. 30, 2022 | Jun. 30, 2021 | Dec. 31, 2021 | Sep. 30, 2021 | |
Operating lease, right-of-use asset | $ 2,556 | $ 1,922 | ||||
Operating lease liabilities | (2,647) | (1,891) | ||||
Restricted cash included in deposits and other noncurrent assets | $ 1,515 | 1,515 | $ 1,779 | |||
Lease expense | $ 1,400 | $ 1,900 | $ 2,800 | $ 3,700 | ||
Weighted average remaining lease term | 6 years 6 months | 6 years 6 months | ||||
Weighted average incremental borrowing rate | 7.40% | 7.40% | ||||
Total minimum lease payments | ||||||
Current lease liabilities | $ 2,526 | $ 2,526 | 5,571 | |||
Non-current lease liabilities | 21,741 | 21,741 | $ 38,608 | |||
75 Sidney Street | ||||||
Lease term | 3 years 3 months 18 days | |||||
Operating lease, right-of-use asset | 14,500 | |||||
Operating lease liabilities | 17,000 | |||||
Gain on termination of lease | 2,500 | |||||
Restricted cash included in deposits and other noncurrent assets | 1,800 | 1,800 | ||||
Deposits and other non-current assets | 75 Sidney Street | ||||||
Restricted cash included in deposits and other noncurrent assets | 1,500 | 1,500 | ||||
Other current assets | 75 Sidney Street | ||||||
Restricted cash included in deposits and other noncurrent assets | $ 300 | $ 300 |
Lease obligation - Operating su
Lease obligation - Operating sublease income (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended |
Jun. 30, 2022 | Jun. 30, 2022 | |
Lease obligation | ||
Operating sublease income | $ 690 | $ 1,380 |
Other liabilities (Details)
Other liabilities (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Jun. 30, 2022 | |
Other current liabilities | ||
Lease liability | $ 5,571 | $ 2,526 |
Total other current liabilities | 5,571 | 2,526 |
Other non-current liabilities | ||
Lease liability | 38,608 | 21,741 |
Other | 1,001 | 1,000 |
Total other non-current liabilities | 39,609 | $ 22,741 |
Restructuring costs | $ 2,600 |
Commitments and contingencies -
Commitments and contingencies - Neurocrine Collaboration Agreement - (Details) $ / shares in Units, $ in Thousands | 1 Months Ended | 3 Months Ended | 6 Months Ended | ||||
Jun. 30, 2019 USD ($) shares | Mar. 31, 2019 USD ($) item $ / shares shares | Jun. 30, 2022 USD ($) | Dec. 31, 2021 USD ($) | Jun. 30, 2021 USD ($) | Jun. 30, 2022 USD ($) | Jun. 30, 2021 USD ($) | |
Allocation of Transaction Price | |||||||
Revenue recognized | $ 1,214 | ||||||
Deferred revenue | $ 10,881 | $ 12,096 | 10,881 | ||||
Related party collaboration receivable | 151 | 732 | 151 | ||||
Neurocrine Collaboration Agreement | FA Program | |||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||
Percentage of profit or loss under co-co option | 40% | ||||||
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 | ||||||
Discovery programs under development | 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 | $ 5,000 | $ 50,000 | |||||
Price per share | $ / shares | $ 11.9625 | ||||||
Aggregate maximum milestone payments to be received from collaborative partner | $ 170,000 | ||||||
Estimated cost reimbursement | 431,100 | $ 80,000 | 79,700 | ||||
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 | ||||||
Equity investment | 50,000 | ||||||
Allocation of Transaction Price | |||||||
Allocation of variable consideration | 79,668 | 79,668 | |||||
Allocation of fixed consideration | 92,400 | 92,382 | 92,382 | ||||
Revenue recognized | 700 | $ 1,400 | 1,400 | $ 7,900 | |||
Cumulative catch up of revenue recognized | 900 | ||||||
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% | ||||||
Neurocrine Collaboration Agreement | Neurocrine | VY-AADC | |||||||
Allocation of Transaction Price | |||||||
Allocation of variable consideration | 53,863 | 53,863 | |||||
Allocation of fixed consideration | 49,045 | 49,045 | |||||
Neurocrine Collaboration Agreement | Neurocrine | FA Program | |||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||
Percentage of profit or loss under co-co option | 60% | ||||||
Allocation of Transaction Price | |||||||
Allocation of variable consideration | 17,088 | 17,088 | |||||
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 | 5,355 | 5,355 | |||||
Allocation of fixed consideration | 14,443 | 14,443 | |||||
Neurocrine Collaboration Agreement | Neurocrine | Discovery program 2 | |||||||
Allocation of Transaction Price | |||||||
Allocation of variable consideration | 3,362 | 3,362 | |||||
Allocation of fixed consideration | $ 8,247 | $ 8,247 | |||||
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 | 6 Months Ended |
Jun. 30, 2022 USD ($) | |
Commitments and contingencies | |
Related party collaboration receivables, beginning balance | $ 732 |
Additions | 358 |
Deductions | (939) |
Related party collaboration receivables, ending balance | 151 |
Deferred revenue, beginning balance | 12,096 |
Deductions | (1,214) |
Deferred revenue, ending balance | $ 10,881 |
Commitments and contingencies_3
Commitments and contingencies - Pfizer Option and License Agreement - (Details) $ in Thousands | 6 Months Ended | |||
Oct. 01, 2021 USD ($) item | Jun. 30, 2022 USD ($) | Mar. 04, 2022 USD ($) | Dec. 31, 2021 USD ($) | |
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||
Revenue recognized | $ | $ 1,214 | |||
Deferred revenue | $ | 10,881 | $ 12,096 | ||
Pfizer | Option and license agreement | ||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||
Number of transgenes | 2 | |||
Number of options | 1 | |||
Number of options exercised | 0 | |||
Number of performance obligations | 2 | |||
Number of material rights | 2 | |||
Upfront payment | $ | $ 30,000 | |||
Number of license options | 1 | |||
Allocation of transaction price | $ | $ 15,000 | |||
Aggregate milestone payments, if exercise rights | $ | $ 10,000 | |||
Deferred revenue | $ | $ 30,000 | |||
Number of days written notice required by reporting entity to terminate agreement | 90 days | |||
Pfizer | Development, regulatory and commercialization milestone | Option and license agreement | ||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||
Aggregate maximum milestone payments to be received from collaborative partner | $ | $ 115,000 | |||
Pfizer | Sales milestone | Option and license agreement | ||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||
Aggregate maximum milestone payments to be received from collaborative partner | $ | $ 175,000 | |||
Minimum | Pfizer | Option and license agreement | ||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||
Number of countries received regulatory approval | 1 | |||
Number of major market countries received regulatory approval | 1 | |||
Maximum | Pfizer | Option and license agreement | ||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||
Number of options exercised | 1 |
Commitments and contingencies_4
Commitments and contingencies - Novartis Option and License Agreement (Details) $ in Thousands | 6 Months Ended | ||
Mar. 04, 2022 USD ($) item | Jun. 30, 2022 USD ($) | Dec. 31, 2021 USD ($) | |
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||
Revenue recognized | $ | $ 1,214 | ||
Deferred revenue | $ | 10,881 | $ 12,096 | |
Novartis Pharma, AG | Novartis License Agreement | |||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||
Number of transgenes | 3 | ||
Number of additional targets | 2 | ||
Upfront payment | $ | $ 54,000 | ||
Additional fee agreed to pay | $ | 18,000 | ||
Aggregate maximum milestone payments to be received from collaborative partner | $ | 125,000 | ||
Aggregate milestone payments, if exercise rights | $ | $ 12,500 | ||
Number of license options | 3 | ||
Number of license options on additional targets | 5 | ||
Number of license option exercise for each target | 1 | ||
Number of Alliance Managers | 2 | ||
Number of options | 1 | ||
Number of major market countries received regulatory approval | 3 | ||
Number of options exercised | 0 | ||
Number of days written notice required by reporting entity to terminate agreement | 90 days | ||
Number of performance obligations | 3 | ||
Number of material rights | 3 | ||
Allocation of transaction price | $ | $ 18,000 | ||
Revenue recognized | $ | $ 54,000 | ||
Novartis Pharma, AG | Novartis License Agreement | Maximum | |||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||
Number of options exercised | 1 | ||
Novartis Pharma, AG | Sales milestone | Novartis License Agreement | |||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||
Aggregate maximum milestone payments to be received from collaborative partner | $ | $ 175,000 |
Commitments and contingencies_5
Commitments and contingencies - Other Agreements - (Details) - Non-profit organization agreement $ in Millions | 12 Months Ended | |
Dec. 31, 2016 USD ($) multiple | Dec. 31, 2017 USD ($) | |
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 | 6 Months Ended | ||
Jun. 30, 2022 | Jun. 30, 2021 | Jun. 30, 2022 | Jun. 30, 2021 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Stock-based compensation. | $ 2,490 | $ 3,991 | $ 4,809 | $ 7,592 |
Stock options | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Stock-based compensation. | 1,618 | 2,480 | 3,095 | 5,002 |
Unvested restricted common stock units | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Stock-based compensation. | 842 | 1,392 | 1,634 | 2,366 |
Employee stock purchase plan | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Stock-based compensation. | 30 | 120 | 81 | 223 |
Research and development | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Stock-based compensation. | 781 | 1,336 | 1,582 | 2,706 |
General and administrative | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Stock-based compensation. | $ 1,709 | $ 2,655 | $ 3,227 | $ 4,886 |
Stock-based compensation - Rest
Stock-based compensation - Restricted Stock - (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2022 | Jun. 30, 2021 | Jun. 30, 2022 | Jun. 30, 2021 | |
Restricted stock disclosures | ||||
Stock-based compensation. | $ 2,490 | $ 3,991 | $ 4,809 | $ 7,592 |
Unvested restricted common stock units | ||||
Shares | ||||
Balance, beginning (in shares) | 806,379 | |||
Granted (in shares) | 739,080 | |||
Vested (in shares) | (344,255) | |||
Forfeited (in shares) | (169,839) | |||
Balance, ending (in shares) | 1,031,365 | 1,031,365 | ||
Weighted Average Grant Date Fair Value Per Share | ||||
Balance, beginning (in dollars per share) | $ 7.26 | |||
Granted (in dollars per share) | 3.79 | |||
Vested (in dollars per share) | 7.45 | |||
Forfeited (in dollars per share) | 5.06 | |||
Balance, ending (in dollars per share) | $ 5.08 | $ 5.08 | ||
Restricted stock disclosures | ||||
Vesting period | 3 years | |||
Stock-based compensation. | $ 842 | $ 1,392 | $ 1,634 | $ 2,366 |
Unrecognized stock-based compensation expense | $ 4,000 | $ 4,000 | ||
Remaining weighted-average remaining vesting period | 2 years | |||
Minimum | Unvested restricted common stock units | ||||
Restricted stock disclosures | ||||
Vesting period | 3 years | |||
Maximum | Unvested restricted common stock units | ||||
Restricted stock disclosures | ||||
Vesting period | 4 years |
Stock-based compensation - Stoc
Stock-based compensation - Stock Options - (Details) $ / shares in Units, $ in Thousands | 6 Months Ended | 12 Months Ended |
Jun. 30, 2022 USD ($) $ / shares shares | Dec. 31, 2021 USD ($) $ / shares shares | |
Share-based Compensation Arrangement by Share-based Payment Award, Options, Outstanding [Roll Forward] | ||
Outstanding, beginning balance (in shares) | shares | 5,013,193 | |
Granted (in shares) | shares | 2,965,075 | |
Exercised (in shares) | shares | (74,496) | |
Cancelled or forfeited (in shares) | shares | (1,274,172) | |
Outstanding, ending balance (in shares) | shares | 6,629,600 | 5,013,193 |
Exercisable (in shares) | shares | 3,378,945 | |
Weighted Average Exercise Price | ||
Outstanding (in dollars per share) | $ / shares | $ 12.69 | |
Granted (in dollars per share) | $ / shares | 4.95 | |
Exercised (in dollars per share) | $ / shares | 7.90 | |
Cancelled or forfeited (in dollars per share) | $ / shares | 12.42 | |
Outstanding (in dollars per share) | $ / shares | 9.33 | $ 12.69 |
Exercisable (in dollars per share) | $ / shares | $ 12.29 | |
Remaining Contractual Life | ||
Outstanding | 7 years 2 months 12 days | 5 years 7 months 6 days |
Exercisable | 5 years 3 months 18 days | |
Aggregate Intrinsic Value | ||
Outstanding | $ | $ 5,914 | $ 1,165 |
Exercisable | $ | 1,575 | |
Stock options | ||
Options disclosures | ||
Unrecognized stock-based compensation expense | $ | $ 13,000 | |
Remaining weighted-average remaining vesting period | 3 years 1 month 6 days |
Net loss per share (Details)
Net loss per share (Details) - shares | 6 Months Ended | |
Jun. 30, 2022 | Jun. 30, 2021 | |
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Antidilutive securities | 7,798,220 | 7,246,940 |
Unvested restricted common stock awards | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Antidilutive securities | 137,255 | 156,863 |
Unvested restricted common stock units | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Antidilutive securities | 1,031,365 | 1,170,702 |
Stock options | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Antidilutive securities | 6,629,600 | 5,919,375 |
Related party transactions (Det
Related party transactions (Details) | 3 Months Ended | 6 Months Ended | |||
Jun. 30, 2022 USD ($) individual | Jun. 30, 2021 USD ($) individual | Jun. 30, 2022 USD ($) individual | Jun. 30, 2021 USD ($) individual | Dec. 31, 2021 USD ($) | |
Related party transactions | |||||
Related party collaboration receivable | $ 151,000 | $ 151,000 | $ 732,000 | ||
Dr. Sah | Board and scientific advisory services | |||||
Related party transactions | |||||
Number of individuals providing services | individual | 1,000 | 1,000 | 1,000 | 1,000 | |
Total amount of services received | $ 82,425 | $ 60,000 | $ 92,325 | $ 90,000 | |
Dr. Sandrock | Consulting and advisor services | |||||
Related party transactions | |||||
Total amount of services received | 60,000 | ||||
Neurocrine | Collaboration arrangement | |||||
Related party transactions | |||||
Related party collaboration receivable | $ 200,000 | $ 200,000 |