Document and Entity Information
Document and Entity Information - shares | 9 Months Ended | |
Sep. 30, 2022 | Nov. 04, 2022 | |
Document and Entity Information | ||
Document Type | 10-Q | |
Document Quarterly Report | true | |
Document Period End Date | Sep. 30, 2022 | |
Document Transition Report | false | |
Entity File Number | 001-39231 | |
Entity Registrant Name | PASSAGE BIO, INC. | |
Entity Incorporation, State or Country Code | DE | |
Entity Tax Identification Number | 82-2729751 | |
Entity Address, Address Line One | One Commerce Square | |
Entity Address, Address Line Two | 2005 Market Street, 39th Floor | |
Entity Address, City or Town | Philadelphia | |
Entity Address, State or Province | PA | |
Entity Address, Postal Zip Code | 19103 | |
City Area Code | 267 | |
Local Phone Number | 866-0311 | |
Title of 12(b) Security | Common Stock | |
Trading Symbol | PASG | |
Security Exchange Name | NASDAQ | |
Entity Current Reporting Status | Yes | |
Entity Interactive Data Current | Yes | |
Entity Filer Category | Non-accelerated Filer | |
Entity Small Business | true | |
Entity Emerging Growth Company | true | |
Entity Ex Transition Period | false | |
Entity Shell Company | false | |
Entity Common Stock, Shares Outstanding | 54,553,263 | |
Entity Central Index Key | 0001787297 | |
Current Fiscal Year End Date | --12-31 | |
Document Fiscal Year Focus | 2022 | |
Document Fiscal Period Focus | Q3 | |
Amendment Flag | false |
Balance Sheets
Balance Sheets - USD ($) $ in Thousands | Sep. 30, 2022 | Dec. 31, 2021 |
Current assets: | ||
Cash and cash equivalents | $ 62,568 | $ 128,965 |
Marketable securities | 151,190 | 186,808 |
Prepaid expenses and other current assets | 1,509 | 1,726 |
Prepaid research and development | 7,257 | 7,567 |
Total current assets | 222,524 | 325,066 |
Property and equipment, net | 22,918 | 23,806 |
Right of use assets - operating leases | 19,890 | |
Other assets | 4,720 | 6,204 |
Total assets | 270,052 | 355,076 |
Current liabilities: | ||
Accounts payable | 4,506 | 9,448 |
Accrued expenses and other current liabilities | 13,861 | 20,050 |
Operating lease liabilities | 3,252 | |
Total current liabilities | 21,619 | 29,498 |
Operating lease liabilities - noncurrent | 24,037 | |
Deferred rent | 6,921 | |
Total liabilities | 45,656 | 36,419 |
Commitments and Contingencies (note 9) | ||
Stockholders' equity: | ||
Common stock, $0.0001 par value: 300,000,000 shares authorized; 54,546,596 shares issued and outstanding at September 30, 2022 and 54,244,996 shares issued and outstanding at December 31, 2021 | 5 | 5 |
Additional paidin capital | 691,194 | 675,346 |
Accumulated other comprehensive income (loss) | (1,468) | (413) |
Accumulated deficit | (465,335) | (356,281) |
Total stockholders' equity | 224,396 | 318,657 |
Total liabilities and stockholders' equity | $ 270,052 | $ 355,076 |
Balance Sheets (Parenthetical)
Balance Sheets (Parenthetical) - $ / shares | Sep. 30, 2022 | Dec. 31, 2021 |
Balance Sheets | ||
Common stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 |
Common stock, shares authorized | 300,000,000 | 300,000,000 |
Common stock, shares issued | 54,546,596 | 54,244,996 |
Common stock, shares outstanding | 54,546,596 | 54,244,996 |
Statements of Operations and Co
Statements of Operations and Comprehensive Loss - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2022 | Sep. 30, 2021 | Sep. 30, 2022 | Sep. 30, 2021 | |
Operating expenses: | ||||
Research and development | $ 15,362 | $ 26,623 | $ 68,396 | $ 84,705 |
Acquired inprocess research and development | 1,500 | 5,500 | 3,000 | 7,000 |
General and administrative | 10,664 | 14,978 | 38,754 | 42,864 |
Loss from operations | (27,526) | (47,101) | (110,150) | (134,569) |
Interest income, net | 825 | 186 | 1,096 | 337 |
Net loss | $ (26,701) | $ (46,915) | $ (109,054) | $ (134,232) |
Per share information: | ||||
Net loss per share of common stock, basic | $ (0.49) | $ (0.87) | $ (2.01) | $ (2.53) |
Net loss per share of common stock, diluted | $ (0.49) | $ (0.87) | $ (2.01) | $ (2.53) |
Weighted average common shares outstanding, basic | 54,473,945 | 53,995,560 | 54,379,397 | 53,080,645 |
Weighted average common shares outstanding, diluted | 54,473,945 | 53,995,560 | 54,379,397 | 53,080,645 |
Comprehensive loss: | ||||
Net loss | $ (26,701) | $ (46,915) | $ (109,054) | $ (134,232) |
Unrealized gain (loss) on marketable securities | 110 | 14 | (1,055) | 45 |
Comprehensive loss | $ (26,591) | $ (46,901) | $ (110,109) | $ (134,187) |
Statements of Stockholders' Equ
Statements of Stockholders' Equity - USD ($) $ in Thousands | Common stock | Additional paid-in capital | Accumulated other comprehensive income (loss) | Accumulated deficit | Total |
Balance at Beginning of period at Dec. 31, 2020 | $ 4 | $ 475,617 | $ (12) | $ (170,895) | $ 304,714 |
Balance at Beginning of period (in shares) at Dec. 31, 2020 | 45,614,807 | ||||
Stockholders' equity | |||||
Vesting of early exercise option awards | 45 | 45 | |||
Vesting of early exercise option awards (in shares) | 211,865 | ||||
Exercise of stock options and vesting of restricted stock units | 229 | 229 | |||
Exercise of stock options and vesting of restricted stock units (in shares) | 101,479 | ||||
Issuance of shares in connection with employee stock purchase plan | 505 | 505 | |||
Issuance of shares in connection with employee stock purchase plan (in shares) | 38,348 | ||||
Sale of common stock, net of issuance costs | $ 1 | 165,804 | 165,805 | ||
Sale of common stock, net of issuance costs (in shares) | 8,050,000 | ||||
Unrealized gain (loss) on marketable securities | 45 | 45 | |||
Share-based compensation expense | 25,116 | 25,116 | |||
Net loss | (134,232) | (134,232) | |||
Balance at end of period at Sep. 30, 2021 | $ 5 | 667,316 | 33 | (305,127) | 362,227 |
Balance at end of period (in shares) at Sep. 30, 2021 | 54,016,499 | ||||
Balance at Beginning of period at Jun. 30, 2021 | $ 5 | 660,457 | 19 | (258,212) | 402,269 |
Balance at Beginning of period (in shares) at Jun. 30, 2021 | 53,956,380 | ||||
Stockholders' equity | |||||
Vesting of early exercise option awards (in shares) | 19,373 | ||||
Exercise of stock options and vesting of restricted stock units | 44 | 44 | |||
Exercise of stock options and vesting of restricted stock units (in shares) | 40,746 | ||||
Unrealized gain (loss) on marketable securities | 14 | 14 | |||
Share-based compensation expense | 6,815 | 6,815 | |||
Net loss | (46,915) | (46,915) | |||
Balance at end of period at Sep. 30, 2021 | $ 5 | 667,316 | 33 | (305,127) | 362,227 |
Balance at end of period (in shares) at Sep. 30, 2021 | 54,016,499 | ||||
Balance at Beginning of period at Dec. 31, 2021 | $ 5 | 675,346 | (413) | (356,281) | 318,657 |
Balance at Beginning of period (in shares) at Dec. 31, 2021 | 54,244,996 | ||||
Stockholders' equity | |||||
Exercise of stock options and vesting of restricted stock units | 128 | 128 | |||
Exercise of stock options and vesting of restricted stock units (in shares) | 146,056 | ||||
Issuance of shares in connection with employee stock purchase plan | 243 | 243 | |||
Issuance of shares in connection with employee stock purchase plan (in shares) | 155,544 | ||||
Unrealized gain (loss) on marketable securities | (1,055) | (1,055) | |||
Share-based compensation expense | 15,477 | 15,477 | |||
Net loss | (109,054) | (109,054) | |||
Balance at end of period at Sep. 30, 2022 | $ 5 | 691,194 | (1,468) | (465,335) | 224,396 |
Balance at end of period (in shares) at Sep. 30, 2022 | 54,546,596 | ||||
Balance at Beginning of period at Jun. 30, 2022 | $ 5 | 688,253 | (1,578) | (438,634) | 248,046 |
Balance at Beginning of period (in shares) at Jun. 30, 2022 | 54,463,235 | ||||
Stockholders' equity | |||||
Exercise of stock options and vesting of restricted stock units | 79 | 79 | |||
Exercise of stock options and vesting of restricted stock units (in shares) | 83,361 | ||||
Unrealized gain (loss) on marketable securities | 110 | 110 | |||
Share-based compensation expense | 2,862 | 2,862 | |||
Net loss | (26,701) | (26,701) | |||
Balance at end of period at Sep. 30, 2022 | $ 5 | $ 691,194 | $ (1,468) | $ (465,335) | $ 224,396 |
Balance at end of period (in shares) at Sep. 30, 2022 | 54,546,596 |
Statements of Stockholders' E_2
Statements of Stockholders' Equity (Parenthetical) $ in Thousands | 9 Months Ended |
Sep. 30, 2021 USD ($) | |
Statements of Stockholders' Equity | |
Issuance cost | $ 669 |
Statements of Cash Flows
Statements of Cash Flows - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2022 | Sep. 30, 2021 | |
Cash flows used in operating activities: | ||
Net loss | $ (109,054) | $ (134,232) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Acquired inprocess research and development | 3,000 | 7,000 |
Depreciation and amortization | 2,733 | 874 |
Sharebased compensation | 15,477 | 25,116 |
Amortization of premium and discount on marketable securities, net | 1,206 | 2,235 |
Deferred rent | 1,618 | |
Changes in operating assets and liabilities: | ||
Prepaid expenses and other current assets, and other assets | 1,701 | (66) |
Prepaid research and development | 310 | 2,026 |
Right of use assets and operating lease liabilities | 478 | |
Accounts payable | (4,979) | 2,085 |
Accrued expenses and other current liabilities | (5,061) | (2,760) |
Net cash used in operating activities | (94,189) | (96,104) |
Cash flows provided by (used in) investing activities: | ||
Purchases of marketable securities | (116,304) | (169,860) |
Sales or maturities of marketable securities | 149,661 | 145,974 |
Purchases of technology licenses | (3,000) | (7,500) |
Purchases of property and equipment | (1,800) | (10,823) |
Net cash provided by (used in) investing activities | 28,557 | (42,209) |
Cash flows provided by (used in) financing activities: | ||
Proceeds from issuance of common stock, net of offering costs | 165,805 | |
Payment of offering costs | (338) | |
Proceeds from the exercise of stock options | 128 | 229 |
Proceeds from the issuance of common stock under employee stock purchase plan | 243 | 505 |
Payments for insurance premium financing | (1,136) | |
Net cash provided by (used in) financing activities | (765) | 166,201 |
Net increase (decrease) in cash and cash equivalents | (66,397) | 27,888 |
Cash and cash equivalents at beginning of year | 128,965 | 135,002 |
Cash and cash equivalents at end of period | 62,568 | 162,890 |
Supplemental disclosure of noncash investing and financing activities: | ||
Unrealized gain (loss) on marketable securities | (1,055) | 45 |
Property and equipment in accounts payable and accrued expenses and other current liabilities | 45 | 2,254 |
Property and equipment in deferred rent | 2,769 | |
Vesting of early exercise option awards | $ 45 | |
Right of use assets recognized upon the adoption of Topic 842 | (20,375) | |
Operating lease liabilities recognized upon the adoption of Topic 842 | $ 27,296 |
Nature of Operations
Nature of Operations | 9 Months Ended |
Sep. 30, 2022 | |
Nature of Operations | |
Nature of Operations | 1. Nature of Operations Passage Bio, Inc., or the Company, a Delaware corporation incorporated in July 2017, is a clinical stage genetic medicines company focused on developing transformative therapies for central nervous system, or CNS disorders, with limited or no approved treatment options. The Company has a strategic research collaboration with the Trustees of the University of Pennsylvania’s, or Penn, Gene Therapy Program, or GTP, that provides the Company with access to one of the premier research institutions in the world for the discovery and preclinical development of genetic medicine product candidates, and exclusive rights to certain CNS indications. Under this collaboration, GTP conducts discovery and preclinical activities enabling Investigational New Drug, or IND, applications and the Company conducts all clinical development, manufacturing, regulatory strategy, and commercialization activities under the agreement. The Company also has a collaboration agreement and a development services and clinical supply agreement with Catalent Maryland, Inc., or Catalent, |
Risks and Liquidity
Risks and Liquidity | 9 Months Ended |
Sep. 30, 2022 | |
Risks and Liquidity | |
Risks and Liquidity | 2. Risks and Liquidity The Company has incurred recurring losses and negative cash flows from operations since inception and had an accumulated deficit of $465.3 million as of September 30, 2022. The Company anticipates incurring additional losses until such time, if ever, that it can generate significant sales of its product candidates currently in development. Substantial additional capital will be needed by the Company to fund its operations and to develop its product candidates. In January 2021, the Company closed a follow-on public offering in which the Company issued and sold 8,050,000 shares of its common stock at a public offering price of $22.00 per share for net proceeds of $165.8 million after deducting underwriting discounts, commissions and other offering expenses. The Company’s operations have consisted primarily of organizing the Company, securing financing, developing licensed technology, performing research, and conducting preclinical studies and clinical trials. The Company faces risks associated with early-stage biotechnology companies whose product candidates are in development. Product candidates currently under development will require significant additional research and development efforts, including extensive preclinical and clinical testing, establishing manufacturing capacity and regulatory approval prior to commercialization. These efforts require significant amounts of additional capital for the Company to complete its research and development, achieve its research and development objectives, defend its intellectual property rights, and recruit and retain skilled personnel and key members of management. Even if the Company’s product development efforts are successful, it is uncertain when, if ever, the Company will realize revenue from product sales. The Company plans to seek additional funding through public or private equity offerings, debt financings, other collaborations, strategic alliances and licensing arrangements. The Company may not be able to obtain financing on acceptable terms, or at all, and the Company may not be able to enter into strategic alliances or other arrangements on favorable terms, or at all. The terms of any financing may adversely affect the holdings or the rights of the Company’s stockholders. If the Company is unable to obtain funding or prospects of funding are unfavorable, the Company could be required to further delay, reduce or eliminate research and development programs, product portfolio expansion or future commercialization efforts, which could adversely affect its business prospects. In March and November 2022, the Company announced workforce reductions and plans to prioritize research and development programs to reduce operating expenses and to extend its cash runway. In accordance with Accounting Standards Update, or ASU, No. 2014-15 , Disclosure of Uncertainties about an Entity’s Ability to Continue as a Going Concern its forecasted operating expenses and capital expenditure requirements for at least the next twelve months from the issuance date of these financial statements. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 9 Months Ended |
Sep. 30, 2022 | |
Summary of Significant Accounting Policies | |
Summary of Significant Accounting Policies | 3. Summary of Significant Accounting Policies The Company’s complete summary of significant accounting policies can be found in “Note 3. Summary of Significant Accounting Policies” in the audited financial statements included in the Company’s Annual Report filed on Form 10-K for the year ended December 31, 2021. Basis of Presentation The accompanying unaudited financial statements have been prepared in accordance with generally accepted accounting principles in the United States, or GAAP. Any reference in these notes to applicable guidance is meant to refer to GAAP as found in the Accounting Standards Codification, or ASC, and Accounting Standards Updates promulgated by the Financial Accounting Standards Board, or FASB. Interim Financial Statements The accompanying unaudited interim financial statements have been prepared from the books and records of the Company in accordance with GAAP for interim financial information and Rule 10-01 of Regulation S-X promulgated by the United States Securities and Exchange Commission, or SEC, which permits reduced disclosures for interim periods. All adjustments, consisting only of normal recurring adjustments, necessary for a fair presentation of the accompanying balance sheets, statements of operations, stockholders’ equity, and cash flows have been made. Although these interim financial statements do not include all of the information and footnotes required for complete annual financial statements, management believes the disclosures are adequate to make the information presented not misleading. Unaudited interim results of operations and cash flows are not necessarily indicative of the results that may be expected for the full year. Unaudited interim financial statements and footnotes should be read in conjunction with the audited financial statements and footnotes included in the Company’s 2021 Annual Report filed on Form 10-K. Use of Estimates The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and contingent liabilities at the date of the financial statements and the reported amounts of expenses during the reporting period. Actual results could differ from those estimates. Estimates and assumptions are periodically reviewed and the effects of the revisions are reflected in the accompanying financial statements in the period they are determined to be necessary. Fair Value of Financial Instruments Management believes that the carrying amounts of the Company’s financial instruments, including cash equivalents, prepaid expenses, and accounts payable, approximate fair value due to the short-term nature of those instruments. Concentration of credit risk Financial instruments that potentially subject the Company to significant concentrations of credit risk consist primarily of cash, cash equivalents, and marketable securities. The Company maintains deposits in federally insured financial institutions in excess of federally insured limits. The Company has not experienced any losses in such accounts and believes it is not exposed to significant risk on its cash, cash equivalents, and marketable securities. Cash and cash equivalents The Company considers all highly-liquid investments that have maturities of three months or less when acquired to be cash equivalents. Cash equivalents as of September 30, 2022 consisted of money market funds, certificates of deposits, corporate debt securities and commercial paper. Cash consists of cash deposits at banking institutions. Marketable securities The Company classifies its marketable securities as available-for-sale, which include commercial paper, certificates of deposit, corporate debt securities and United States, or U.S., government debt securities with original maturities of greater than three months. These securities are carried at fair market value, with unrealized gains and losses reported in comprehensive loss and accumulated other comprehensive income (loss) within stockholders’ equity. Gains or losses on marketable securities sold are recognized on the specific identification method. Share-based compensation The Company measures share-based awards at their grant-date fair value and records compensation expense on a straight-line basis over the vesting period of the awards. Estimating the fair value of share-based awards requires the input of subjective assumptions, including, for stock options, stock price volatility. The Company accounts for forfeitures for stock option awards as they occur. The Company uses the Black-Scholes option pricing model to value its stock option awards. The assumptions used in estimating the fair value of share-based awards represent management's estimate and involve inherent uncertainties and the application of management's judgment. As a result, if factors change and management uses different assumptions, share-based compensation expense could be materially different for future awards. The expected term of the stock options is estimated using the "simplified method," as the Company has limited historical information from which to develop reasonable expectations about future exercise patterns and post-vesting employment termination behavior for its stock option grants. The simplified method is the midpoint between the vesting period and the contractual term of the option. For stock price volatility, the Company uses a composite of comparable public company data as a basis for its expected volatility to calculate the fair value of option grants. The risk-free rate is based on the U.S. Treasury yield curve commensurate with the expected life of the option. Net Loss Per Share Basic net loss per share of common stock is computed by dividing net loss by the weighted-average number of shares of common stock outstanding during each period. Diluted loss per share of common stock includes the effect, if any, from the potential exercise or conversion of securities, such as stock options, which would result in the issuance of incremental shares of common stock. For diluted net loss per share, the weighted-average number of shares of common stock is the same for basic net loss per share due to the fact that when a net loss exists, dilutive securities are not included in the calculation as the impact is anti-dilutive. The following potentially dilutive securities have been excluded from the computation of diluted weighted-average shares of common stock outstanding, as they would be anti-dilutive: Nine Months Ended 2022 2021 Stock options 11,738,877 9,581,659 Unvested restricted stock units 409,500 238,333 Employee stock purchase plan 62,488 49,528 12,210,865 9,869,520 Recently Adopted Accounting Pronouncements In February 2016, the FASB issued ASU No. 2016-02, “ Leases” Recently Issued Accounting Pronouncements In June 2016, the FASB issued ASU No. 2016-13, “ Financial Instruments-Credit Losses: Measurement of Credit Losses on Financial Instruments ”, or ASU 2016-13, which replaces the incurred loss impairment methodology under current U.S. GAAP with a methodology that reflects expected credit losses and requires consideration of a broader range of reasonable and supportable information to inform credit loss estimates. ASU 2016-13 was subsequently updated by ASU No. 2019-04, “ Codification Improvements to Topic 326, Financial Instruments—Credit Losses, Topic 815, Derivatives and Hedging, and Topic 825, Financial Instruments ”, to clarify that entities should include recoveries when estimating the allowance for credit losses. This guidance is effective for the Company for fiscal years, and interim periods within those fiscal years, beginning after December 15, 2022 and must be adopted using a modified retrospective approach, with certain exceptions. The Company is currently evaluating the impact of this standard on its financial statements and related disclosures and does not expect a material impact to financial statements or disclosures . |
Cash, cash equivalents and mark
Cash, cash equivalents and marketable securities | 9 Months Ended |
Sep. 30, 2022 | |
Cash, cash equivalents and marketable securities | |
Cash, cash equivalents and marketable securities | 4. Cash, cash equivalents and marketable securities The following table provides details regarding the Company’s portfolio of cash and cash equivalents: Cost or (in thousands) Amortized cost Unrealized gains Unrealized losses Fair value September 30, 2022: Cash accounts in banking institutions $ 25,453 $ - $ - $ 25,453 Money market funds 15,721 - - 15,721 Certificates of deposit 7,032 - (2) 7,030 Corporate debt securities 554 - (1) 553 Commercial paper 13,817 - (6) 13,811 Total $ 62,576 $ - $ (9) $ 62,568 December 31, 2021 Cash accounts in banking institutions $ 44,549 $ - $ - $ 44,549 Money market funds 84,416 - - 84,416 Total $ 128,965 $ - $ - $ 128,965 The following table provides details regarding the Company’s portfolio of marketable securities: (in thousands) Amortized cost Unrealized gains Unrealized losses Fair value September 30, 2022: Certificates of deposit $ 22,012 $ 2 $ (96) $ 21,918 Commercial paper 49,451 1 (118) 49,334 Corporate debt securities 79,188 - (1,198) 77,990 U.S. government securities 1,998 - (50) 1,948 Total $ 152,649 $ 3 $ (1,462) $ 151,190 December 31, 2021 Certificates of deposit $ 5,296 $ - $ - $ 5,296 Commercial paper 26,503 4 (4) 26,503 Corporate debt securities 145,577 10 (418) 145,169 U.S. government securities 1,996 - (8) 1,988 Non-U.S. government securities 7,849 4 (1) 7,852 Total $ 187,221 $ 18 $ (431) $ 186,808 |
Fair Value of Financial Instrum
Fair Value of Financial Instruments | 9 Months Ended |
Sep. 30, 2022 | |
Fair Value of Financial Instruments | |
Fair Value of Financial Instruments | 5. Fair Value of Financial Instruments Fair value is the price that could be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants. Fair value determination in accordance with applicable accounting guidance requires that a number of significant judgments be made. Additionally, fair value is used on a nonrecurring basis to evaluate assets for impairment or as required for disclosure purposes by applicable accounting guidance on disclosures about fair value of financial instruments. Depending on the nature of the assets and liabilities, various valuation techniques and assumptions are used when estimating fair value. The carrying amounts of certain of the Company’s financial instruments, including prepaid expense and accounts payable are shown at cost, which approximates fair value due to the short-term nature of these instruments. The Company follows the provisions of FASB ASC Topic 820, Fair Value Measurement ● Level 1: Unadjusted quoted prices in active markets that are accessible at the measurement date for identical, unrestricted assets or liabilities. ● Level 2: Quoted prices in markets that are not active, or inputs which are observable, either directly or indirectly, for substantially the full term of the asset or liabilities. ● Level 3: Prices or valuation techniques that require inputs that are both significant to the fair value measurement and unobservable (i.e., supported by little or no market activity). The following fair value hierarchy table presents information about the Company’s assets measured at fair value on a recurring basis. Included within cash and cash equivalents on the balance sheet, but excluded from the fair value hierarchy table, are cash deposits held at financial institutions: Fair value measurement at reporting date using Quoted prices in active Significant markets for other Significant identical observable unobservable assets inputs inputs (in thousands) (Level 1) (Level 2) (Level 3) September 30, 2022: Assets Cash and cash equivalents: Certificates of deposit $ - $ 7,030 $ - Money market funds 15,721 - $ - Commercial paper - 13,811 - Corporate debt securities - 553 - Total cash and cash equivalents 15,721 21,394 - Marketable securities: Certificates of deposit - 21,918 - Commercial paper - 49,334 - Corporate debt securities - 77,990 - U.S. government securities - 1,948 - Total marketable securities - 151,190 - Total financial assets $ 15,721 $ 172,584 $ - December 31, 2021 Assets Cash and cash equivalents: Money market funds $ 84,416 $ - $ - Total cash and cash equivalents 84,416 - - Marketable securities: Certificates of deposit - 5,296 - Commercial paper - 26,503 - Corporate debt securities - 145,169 - U.S. government securities - 1,988 - Non-U.S. government securities - 7,852 - Total marketable securities - 186,808 - Total financial assets $ 84,416 $ 186,808 $ - |
Property and Equipment, net
Property and Equipment, net | 9 Months Ended |
Sep. 30, 2022 | |
Property and Equipment, net | |
Property and Equipment, net | 6. Property and Equipment, net Property and Equipment, net, consist of the following: (in thousands) September 30, 2022 December 31, 2021 Laboratory equipment $ 9,753 $ 8,916 Office equipment 601 621 Computer hardware and software 1,089 1,028 Furniture and fixtures 1,203 1,487 Leasehold improvements 13,487 13,409 Construction in progress 993 822 Total property and equipment 27,126 26,283 Accumulated depreciation (4,208) (2,477) $ 22,918 $ 23,806 |
Accrued Expenses and Other Curr
Accrued Expenses and Other Current Liabilities | 9 Months Ended |
Sep. 30, 2022 | |
Accrued Expenses and Other Current Liabilities | |
Accrued Expenses and Other Current Liabilities | 7. Accrued Expenses and Other Current Liabilities Accrued expenses and other current liabilities consisted of the following: (in thousands) September 30, 2022 December 31, 2021 Professional fees $ 636 $ 877 Compensation and related benefits 7,006 10,014 Research and development 5,640 8,498 Property and equipment 8 161 Other 571 500 $ 13,861 $ 20,050 |
Leases
Leases | 9 Months Ended |
Sep. 30, 2022 | |
Leases | |
Leases | 8. Leases On January 1, 2022, the Company adopted ASU No. 2016-02, Leases The Company was not required to record a cumulative effect adjustment upon adoption as the Company did not capitalize any material initial direct costs nor were any contracts reassessed leading to changes in the terms or contractual payments of historical arrangements that would impact expense recognition, however, the Company eliminated $3.2 million of deferred rent liabilities and $3.8 million of tenant improvement allowances as of January 1, 2022 related to the Leases as these liabilities are reflected in the operating lease ROU assets. The Company used incremental borrowing rates, or IBRs, of 9.0% and 10.0% to discount the operating lease liabilities for the Lease Agreement and the Laboratory Lease Agreement, respectively. The Company’s IBRs were quoted by an unrelated third-party lender and reflect a collateralized borrowing with similar terms and amounts as the Leases. The Company is party to a lease agreement for office space, or the Lease Agreement, in Philadelphia, Pennsylvania. The Lease Agreement commenced in February 2021 and is expected to expire in December 2031. The Company has an option to extend the term of the Lease Agreement by up to two five-year terms. This option to extend was not recognized as part of the Company's measurement of the ROU asset and operating lease liability as of September 30, 2022. The landlord provided the Company with a tenant improvement allowance of $2.8 million, for which the related expenditures were paid directly by the landlord. The Company is also party to a lease agreement for laboratory space, or the Laboratory Lease Agreement, in Hopewell, New Jersey. The laboratory is initially focused on state-of-the-art analytical capabilities, assay development and validation, and clinical product testing to support both viral vector manufacturing and clinical development. The Laboratory Lease Agreement commenced in March 2021 and is expected to expire in February 2036. The Company has an option to extend the term of the Laboratory Lease Agreement by up to two five-year terms. This option to extend was not recognized as part of the Company's measurement of the ROU asset and operating lease liability as of September 30, 2022. The landlord provided the Company with a tenant improvement allowance of The following table summarizes the Company’s operating leases: Nine Months Ended ($ in thousands) September 30, 2022 Operating lease cost $ 2,487 Cash paid for amounts included in the measurement of operating cash flows from operating leases $ 2,009 Weighted-average discount rate 9.7% Weighted-average remaining lease term (years) 12.4 The future minimum lease payments under the Company’s operating lease arrangements as of September 30, 2022 are as follows: (in thousands) 2022 (remaining) $ 845 2023 3,450 2024 3,548 2025 3,648 2026 3,751 Thereafter 33,507 Total undiscounted lease payments 48,749 Less: imputed interest (21,460) Total lease liabilities $ 27,289 The Company recognized operating lease costs of $0.8 million during both the three months ended September 30, 2022 and 2021 related to the leases. The Company recognized operating lease costs of $2.5 million during both the nine months ended September 30, 2022 and 2021 related to the leases. The future minimum lease payments under the Company’s operating lease arrangements as of December 31, 2021 were as follows: (in thousands) 2022 $ 2,884 2023 3,453 2024 3,550 2025 3,651 2026 3,754 Thereafter 33,534 $ 50,826 |
Commitments and Contingencies
Commitments and Contingencies | 9 Months Ended |
Sep. 30, 2022 | |
Commitments and Contingencies. | |
Commitments and Contingencies | 9. Commitments and Contingencies Amended and Restated Research, Collaboration and License Arrangement with Penn The Company has a research, collaboration and licensing agreement with Penn, as amended, or the Penn Agreement, for research and development collaborations and exclusive license rights to patents for certain products and technologies. Under the Penn Agreement, the Company has obligations to fund certain research relating to the preclinical development of selected products in research programs as well as the exploratory research program in non-rare and/or non-monogenic, or large CNS indications, initially Alzheimer’s Disease, or AD, and Temporal Lobe Epilepsy, or TLE. In addition, the Company will fund discovery research conducted by Penn through August 3, 2026 and will receive exclusive rights, subject to certain limitations, to technologies resulting from the discovery research for the Company’s products developed with GTP, such as novel capsids, toxicity reduction technologies and delivery, and formulation improvements. This funding commitment for the discovery research is $5.0 million annually, paid in quarterly increments of $1.3 million through August 3, 2026. The Penn Agreement includes an exploratory research program focused on discovering targets and novel gene therapy candidates for large CNS indications, initially focused on AD and TLE, and can be expanded to other large CNS diseases upon mutual agreement. The initial term of the exploratory research program is until August 2024, which term can be extended by mutual agreement. During such term, the Company will have an exclusive right of first negotiation to include additional targets to the exploratory research program within the agreed upon large CNS indications. Under the exploratory research program, the Company will have the right to further develop and commercialize any gene therapy product candidates specific for those selected targets within AD and TLE (and any future large CNS diseases that are mutually agreed upon) that may arise from the exploratory research programs on substantially the same terms of the current Penn Agreement. Under the Penn Agreement, the Company has eight remaining options available to commence additional licensed programs for CNS indications and has until August 3, 2026, to exercise these options. If the Company were to exercise any of these options, it would owe Penn a non-refundable upfront fee of $1.0 million per product indication, with $0.5 million due upfront and another $0.5 million fee owed upon a further developmental milestone. The Company has the obligation to fund certain research relating to the preclinical development of each licensed program. The Penn Agreement requires that the Company make payments of up to (i) $16.5 million per product candidate for rare, monogenic disorders in the aggregate and (ii) $39.0 million per product candidate in the aggregate arising from the exploratory program for large CNS indications, initially AD and TLE and such other mutually agreed upon large CNS indications. Each payment will be due upon the achievement of specific development milestone events by such licensed product for a first indication, reduced development milestone payments for the second and third indications and no development milestone payments for subsequent indications. In addition, on a product-by-product basis, the Company is obligated to make up to $55.0 million in sales milestone payments on each licensed product based on annual sales of the licensed product in excess of defined thresholds. Upon successful commercialization of a product using the licensed technology, the Company is obligated to pay to Penn, on a licensed product-by-licensed product and country-by-country basis, tiered royalties (subject to customary reductions) in the mid-single digits on annual worldwide net sales of such licensed product. In addition, the Company is obligated to pay to Penn a percentage of sublicensing income, ranging from the mid-single digits to low double digits, for sublicenses under the Penn Agreement. The agreement will expire on a licensed product-by-licensed product and country-by-country basis upon the later of (i) the expiration of the last valid claim of the licensed patent rights that covers the exploitation of such licensed product in such country, and (ii) the expiration of the royalty period. In addition, the Company will pay a tiered transaction fee of 1-2% of the net proceeds upon certain change of control events. During the three months ended September 30, 2022, the Company made payments under the Penn Agreement for acquired in-process research and development of During the three months ended September 30, 2021, the Company made payments under the Penn Agreement for acquired in-process research and development of During the nine months ended September 30, 2022, the Company made payments under the Penn Agreement for acquired in-process research and development of $3.0 million related to the achievement of development milestones. During the nine months ended September 30, 2021, the Company made payments under the Penn Agreement for acquired in-process research and development of Catalent Agreements In June 2019, the Company entered into a collaboration agreement, or the Collaboration Agreement, with Catalent Maryland Inc., or Catalent. As part of the Collaboration Agreement, the Company will pay an annual fee for five years ending in 2025 for the use of the Clean Room Suite. In April 2020, the Company entered into a development services and clinical supply agreement, or the Manufacturing and Supply Agreement, with Catalent to secure clinical scale manufacturing capacity for batches of active pharmaceutical ingredients for the Company’s gene therapy product candidates. The Manufacturing and Supply Agreement confirms the terms contemplated by the Collaboration Agreement. The Collaboration Agreement continues to be in effect pursuant to its terms. Under the terms of the Manufacturing and Supply Agreement, Catalent has agreed to manufacture batches of drug product for the Company’s gene therapy product candidates at the Clean Room Suite at a Catalent facility provided for in the Collaboration Agreement. The Manufacturing and Supply Agreement provides for a term of five years which period may be extended once, at the Company’s option, for an additional five-year period. The Manufacturing and Supply Agreement also includes minimum annual purchase commitments. The Company has the right to terminate the Manufacturing and Supply Agreement for convenience or other reasons specified in the Manufacturing and Supply Agreement upon prior written notice. If the Company terminates the Manufacturing and Supply Agreement, it will be obligated to pay an early termination fee to Catalent. Under both the Collaboration Agreement and the Manufacturing and Supply Agreement, the Company has an annual minimum commitment of $10.6 million per year owed to Catalent for five years from the validation of the Clean Room, subject to certain inflationary adjustments. Employment Agreements The Company has entered into employment agreements with key personnel providing for compensation and, in certain circumstances, severance and acceleration of vesting in stock-based compensation awards, as described in the respective employment agreements. Patent Infringement Claim On February 18, 2020, the Company received a letter from REGENXBIO Inc., or Regenx, which stated its view that the use of the Company’s AAVhu68 capsid infringes patent claims to which Regenx has an exclusive license and which expire in 2024. Regenx also stated that it has exclusive licenses to various pending patent applications regarding the use of AAV vectors administered via intra-cisterna magna injection, and that these applications may lead to issued claims that Regenx believes may, if issued, cover the Company’s planned method of administration for the Company’s lead product candidates. The Company believes it has valid defenses to the issued claims set forth by Regenx relating to AAVhu68. Further, the prosecution of pending patent applications is highly uncertain, and it is unclear whether any patents will be issued from these pending Regenx patent applications at all, much less with claims that are relevant to the administration of the Company’s product candidates. Regenx also requested information regarding the Company’s relationship with Dr. Wilson while he was serving as an advisor to Regenx. Regenx's letter also offers to discuss licensing the applicable patent portfolios from them. In April 2020, the Company responded to Regenx indicating that it does not believe it requires a license to any of the specified Regenx patents or patent applications at this time, and that it found that Dr. Wilson’s relationship with the Company was consistent with his obligations to Regenx. The Company will continue to monitor the situation and, if necessary, take appropriate actions, which may include responding to further correspondence from Regenx, and engaging in discussions with Regenx regarding their claims. If any such patents were enforceable and such claims were ultimately successful, the Company might require a license to continue to use and sell any product candidates using such AAV vector. In March 2022, Penn and Regenx entered into a letter agreement that involved a release of current claims against certain Penn laboratory sponsors, including the Company, and a covenant not to sue certain Penn laboratory sponsors, including the Company, on certain future claims related to certain patent rights and other matters. The Company believes that the release and covenant provide the Company a full release of the matters raised by Regenx against the Company in the February 18, 2020 letter relating to AAVhu68 and Dr. Wilson’s relationship with the Company. |
Share-Based Compensation
Share-Based Compensation | 9 Months Ended |
Sep. 30, 2022 | |
Share-Based Compensation | |
Share-Based Compensation | 10. Share-Based Compensation Equity Incentive Plan The Company has three equity incentive plans: the 2018 Equity Incentive Plan, as amended, or the 2018 Plan, the 2020 Equity Incentive Plan, or the Incentive Plan, and the 2021 Equity Inducement Plan, or the Inducement Plan. New awards can only be granted under the Incentive Plan and the Inducement Plan. The total number of shares authorized under the Incentive Plan as of September 30, 2022 was 10,370,926. Of this amount, 3,910,223 shares were available for future grants as of September 30, 2022, which includes awards previously issued under the 2018 Plan that were forfeited and available for issuance under the Incentive Plan. The number of shares of the Company’s common stock that may be issued pursuant to rights granted under the Incentive Plan shall automatically increase on January 1st of each year, commencing on January 1, 2021 and continuing for ten years, in an amount equal to five percent of the total number of shares of the Company’s common stock outstanding on December 31st of the preceding calendar year, subject to the discretion of the board of directors or compensation committee to determine a lesser number of shares shall be added for such year. As a result, on January 1, 2022, the number of shares reserved for issuance under the Incentive Plan increased by 2,712,249 shares. The Incentive Plan provides for the granting of common stock, incentive stock options, nonqualified stock options, restricted stock awards, and/or stock appreciation rights to employees, directors, and other persons, as determined by the Company’s board of directors. The Company’s stock options awarded to date under the Incentive Plan vest based on requisite service period, generally over four-year periods, and have a term of ten years. The total number of shares authorized under the Inducement Plan as of September 30, 2022 was 2,000,000, as a result of an increase to the shares authorized for issuance in February 2022. Of this amount, 865,500 shares were available for future grants as of September 30, 2022. The Inducement Plan provides for the granting of nonqualified stock options and restricted stock awards to employees hired by the Company, as determined by the Company’s board of directors. The Company’s stock options awarded to date under the Inducement Plan vest based on requisite service period and have a term of ten years. The Company’s restricted stock units awarded to date under the Inducement Plan vest based on requisite service period and have a term based on each award agreement. The Company measures share-based awards at their grant-date fair value and records compensation expense on a straight-line basis over the vesting period of the awards. The Company recorded share-based compensation expense in the following expense categories in its accompanying statements of operations for the period presented: Three Months Ended September 30, Nine Months Ended September 30, (in thousands) 2022 2021 2022 2021 Research and development $ 568 $ 2,306 $ 6,528 $ 13,155 General and administrative 2,294 4,509 8,949 11,961 $ 2,862 $ 6,815 $ 15,477 $ 25,116 During the three months ended September 30, 2022, there was no expense related to the modifications of awards. During the three months ended September 30, 2021, the Company modified certain awards and recognized an additional $0.2 million in general and administrative expenses related to the modifications. During the nine months ended September 30, 2022, the Company modified certain awards and recognized an additional $0.4 million in general and administrative expenses related to the modifications. During the nine months ended September 30, 2021, the Company modified certain awards and recognized an additional $6.1 million in research and development expenses and $0.2 million in general and administrative expenses related to the modifications. The following table summarizes stock option activity for the nine months ended September 30, 2022: Weighted Weighted average average remaining Number of exercise price contractual shares per share term (years) Outstanding at January 1, 2022 9,416,998 $ 13.72 8.6 Granted 5,707,241 3.37 Exercised (126,056) 1.02 Forfeited (3,259,306) 11.65 Outstanding at September 30, 2022 11,738,877 $ 9.40 7.3 Vested and Exercisable at September 30, 2022 5,510,147 $ 11.95 5.4 Vested or expected to vest at September 30, 2022 11,738,877 $ 9.40 7.3 The weighted-average grant date fair value of options granted was $2.52 and $13.64 for the nine months ended September 30, 2022 and 2021, respectively. As of September 30, 2022, the total unrecognized compensation expense related to unvested stock option awards was $32.6 million, which the Company expects to recognize over a weighted-average period of 2.4 years. The fair value of each option was estimated on the date of grant using the weighted average assumptions in the table below: Nine Months Ended September 30, 2022 2021 Expected volatility 92.3 % 98.9 % Risk‑free interest rate 2.6 % 0.9 % Expected term 5.8 years 6.0 years Expected dividend yield — — Restricted Stock Units The Company issues restricted stock units, or RSUs, to employees that vest over periods as determined by the board of directors. Any unvested shares are forfeited upon termination of services. The fair value of the RSUs is equal to the fair market value price of the Company’s common stock on the date of grant. Compensation expense is recognized on a straight-line basis over the vesting period of the RSUs. The following table summarizes activity related to RSU awards during the nine months ended September 30, 2022: Weighted average Number of shares grant date fair value Unvested balance at January 1, 2022 290,500 $ 14.78 Granted 261,500 4.21 Vested (20,000) 17.40 Forfeited (122,500) 12.86 Unvested balance at September 30, 2022 409,500 $ 8.48 As of September 30, 2022, the total unrecognized expense related to all RSUs was $2.5 million, which the Company expects to recognize over a weighted-average period of 2.3 years. Employee Stock Purchase Plan The Company’s 2020 Employee Stock Purchase Plan, or the ESPP, became effective on February 28, 2020. The ESPP authorizes the issuance of up to 1,435,619 shares of the Company’s common stock. Of this amount, 1,168,841 were available for future grants as of September 30, 2022. The number of shares of the Company’s common stock that may be issued pursuant to rights granted under the ESPP shall automatically increase on January 1st of each year and continuing for ten years, in an amount equal to one percent of the total number of shares of the Company’s common stock outstanding on December 31st of the preceding calendar year, subject to the discretion of the board of directors or compensation committee to determine a lesser number of shares shall be added for such year. As a result, on January 1, 2022, the number of shares reserved for issuance under the ESPP increased by 542,449 shares, resulting in a total of 1,435,619 shares authorized for issuance. Under the ESPP, eligible employees can purchase the Company’s common stock through accumulated payroll deductions at such times as are established by the compensation committee. Eligible employees may purchase the Company’s common stock at 85% of the lower of the fair market value of the Company’s common stock on the first day of the offering period or on the last day of the offering period. The offering periods under the ESPP have a duration of six months, with periods ending in May and November of each calendar year. Eligible employees may contribute up to 15% of their eligible compensation. Under the ESPP, a participant may not accrue rights to purchase more than $25,000 worth of the Company’s common stock for each calendar year in which such right is outstanding or purchase more than 4,000 shares of the Company’s common stock in any single offering period. In accordance with the guidance in ASC 718-50 – Compensation – Stock Compensation , the ability to purchase shares of the Company’s common stock at 85% of the lower of the price on the first day of the offering period or the last day of the offering period (i.e. the purchase date) represents an option and therefore, the ESPP is a compensatory plan under this guidance. Accordingly, share-based compensation expense is determined based on the option’s grant-date fair value as estimated by applying the Black Scholes option-pricing model and is recognized over the withholding period. The Company recognized share-based compensation expense of de minimus and $0.1 million during the three months ended September 30, 2022 and 2021, respectively, and $0.2 million and $0.3 million during the nine months ended September 30, 2022 and 2021, respectively, related to the ESPP. |
Subsequent Events
Subsequent Events | 9 Months Ended |
Sep. 30, 2022 | |
Subsequent Events. | |
Subsequent Events | 11. Subsequent Events In November 2022, the Company announced plans to stop further clinical development of PBKR03 for Krabbe disease. Additionally, the Company announced a 23 percent reduction to its workforce, which will result in severance related expenses within the three months ending December 31, 2022. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 9 Months Ended |
Sep. 30, 2022 | |
Summary of Significant Accounting Policies | |
Basis of Presentation | Basis of Presentation The accompanying unaudited financial statements have been prepared in accordance with generally accepted accounting principles in the United States, or GAAP. Any reference in these notes to applicable guidance is meant to refer to GAAP as found in the Accounting Standards Codification, or ASC, and Accounting Standards Updates promulgated by the Financial Accounting Standards Board, or FASB. |
Interim Financial Statements | Interim Financial Statements The accompanying unaudited interim financial statements have been prepared from the books and records of the Company in accordance with GAAP for interim financial information and Rule 10-01 of Regulation S-X promulgated by the United States Securities and Exchange Commission, or SEC, which permits reduced disclosures for interim periods. All adjustments, consisting only of normal recurring adjustments, necessary for a fair presentation of the accompanying balance sheets, statements of operations, stockholders’ equity, and cash flows have been made. Although these interim financial statements do not include all of the information and footnotes required for complete annual financial statements, management believes the disclosures are adequate to make the information presented not misleading. Unaudited interim results of operations and cash flows are not necessarily indicative of the results that may be expected for the full year. Unaudited interim financial statements and footnotes should be read in conjunction with the audited financial statements and footnotes included in the Company’s 2021 Annual Report filed on Form 10-K. |
Use of Estimates | Use of Estimates The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and contingent liabilities at the date of the financial statements and the reported amounts of expenses during the reporting period. Actual results could differ from those estimates. Estimates and assumptions are periodically reviewed and the effects of the revisions are reflected in the accompanying financial statements in the period they are determined to be necessary. |
Fair Value of Financial Instruments | Fair Value of Financial Instruments Management believes that the carrying amounts of the Company’s financial instruments, including cash equivalents, prepaid expenses, and accounts payable, approximate fair value due to the short-term nature of those instruments. |
Concentration of credit risk | Concentration of credit risk Financial instruments that potentially subject the Company to significant concentrations of credit risk consist primarily of cash, cash equivalents, and marketable securities. The Company maintains deposits in federally insured financial institutions in excess of federally insured limits. The Company has not experienced any losses in such accounts and believes it is not exposed to significant risk on its cash, cash equivalents, and marketable securities. |
Cash and cash equivalents | Cash and cash equivalents The Company considers all highly-liquid investments that have maturities of three months or less when acquired to be cash equivalents. Cash equivalents as of September 30, 2022 consisted of money market funds, certificates of deposits, corporate debt securities and commercial paper. Cash consists of cash deposits at banking institutions. |
Marketable securities | Marketable securities The Company classifies its marketable securities as available-for-sale, which include commercial paper, certificates of deposit, corporate debt securities and United States, or U.S., government debt securities with original maturities of greater than three months. These securities are carried at fair market value, with unrealized gains and losses reported in comprehensive loss and accumulated other comprehensive income (loss) within stockholders’ equity. Gains or losses on marketable securities sold are recognized on the specific identification method. |
Share-based compensation | Share-based compensation The Company measures share-based awards at their grant-date fair value and records compensation expense on a straight-line basis over the vesting period of the awards. Estimating the fair value of share-based awards requires the input of subjective assumptions, including, for stock options, stock price volatility. The Company accounts for forfeitures for stock option awards as they occur. The Company uses the Black-Scholes option pricing model to value its stock option awards. The assumptions used in estimating the fair value of share-based awards represent management's estimate and involve inherent uncertainties and the application of management's judgment. As a result, if factors change and management uses different assumptions, share-based compensation expense could be materially different for future awards. The expected term of the stock options is estimated using the "simplified method," as the Company has limited historical information from which to develop reasonable expectations about future exercise patterns and post-vesting employment termination behavior for its stock option grants. The simplified method is the midpoint between the vesting period and the contractual term of the option. For stock price volatility, the Company uses a composite of comparable public company data as a basis for its expected volatility to calculate the fair value of option grants. The risk-free rate is based on the U.S. Treasury yield curve commensurate with the expected life of the option. |
Net Loss Per Share | Net Loss Per Share Basic net loss per share of common stock is computed by dividing net loss by the weighted-average number of shares of common stock outstanding during each period. Diluted loss per share of common stock includes the effect, if any, from the potential exercise or conversion of securities, such as stock options, which would result in the issuance of incremental shares of common stock. For diluted net loss per share, the weighted-average number of shares of common stock is the same for basic net loss per share due to the fact that when a net loss exists, dilutive securities are not included in the calculation as the impact is anti-dilutive. The following potentially dilutive securities have been excluded from the computation of diluted weighted-average shares of common stock outstanding, as they would be anti-dilutive: Nine Months Ended 2022 2021 Stock options 11,738,877 9,581,659 Unvested restricted stock units 409,500 238,333 Employee stock purchase plan 62,488 49,528 12,210,865 9,869,520 |
Recently Adopted and Issued Accounting Pronouncements | Recently Adopted Accounting Pronouncements In February 2016, the FASB issued ASU No. 2016-02, “ Leases” Recently Issued Accounting Pronouncements In June 2016, the FASB issued ASU No. 2016-13, “ Financial Instruments-Credit Losses: Measurement of Credit Losses on Financial Instruments ”, or ASU 2016-13, which replaces the incurred loss impairment methodology under current U.S. GAAP with a methodology that reflects expected credit losses and requires consideration of a broader range of reasonable and supportable information to inform credit loss estimates. ASU 2016-13 was subsequently updated by ASU No. 2019-04, “ Codification Improvements to Topic 326, Financial Instruments—Credit Losses, Topic 815, Derivatives and Hedging, and Topic 825, Financial Instruments ”, to clarify that entities should include recoveries when estimating the allowance for credit losses. This guidance is effective for the Company for fiscal years, and interim periods within those fiscal years, beginning after December 15, 2022 and must be adopted using a modified retrospective approach, with certain exceptions. The Company is currently evaluating the impact of this standard on its financial statements and related disclosures and does not expect a material impact to financial statements or disclosures . |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 9 Months Ended |
Sep. 30, 2022 | |
Summary of Significant Accounting Policies | |
Schedule of antidilutive securities excluded from computation of earnings per share | Nine Months Ended 2022 2021 Stock options 11,738,877 9,581,659 Unvested restricted stock units 409,500 238,333 Employee stock purchase plan 62,488 49,528 12,210,865 9,869,520 |
Cash, cash equivalents and ma_2
Cash, cash equivalents and marketable securities (Tables) | 9 Months Ended |
Sep. 30, 2022 | |
Cash, cash equivalents and marketable securities | |
Schedule of details regarding the Company's portfolio of cash and cash equivalents | Cost or (in thousands) Amortized cost Unrealized gains Unrealized losses Fair value September 30, 2022: Cash accounts in banking institutions $ 25,453 $ - $ - $ 25,453 Money market funds 15,721 - - 15,721 Certificates of deposit 7,032 - (2) 7,030 Corporate debt securities 554 - (1) 553 Commercial paper 13,817 - (6) 13,811 Total $ 62,576 $ - $ (9) $ 62,568 December 31, 2021 Cash accounts in banking institutions $ 44,549 $ - $ - $ 44,549 Money market funds 84,416 - - 84,416 Total $ 128,965 $ - $ - $ 128,965 |
Schedule of details regarding the Company's portfolio of marketable securities | (in thousands) Amortized cost Unrealized gains Unrealized losses Fair value September 30, 2022: Certificates of deposit $ 22,012 $ 2 $ (96) $ 21,918 Commercial paper 49,451 1 (118) 49,334 Corporate debt securities 79,188 - (1,198) 77,990 U.S. government securities 1,998 - (50) 1,948 Total $ 152,649 $ 3 $ (1,462) $ 151,190 December 31, 2021 Certificates of deposit $ 5,296 $ - $ - $ 5,296 Commercial paper 26,503 4 (4) 26,503 Corporate debt securities 145,577 10 (418) 145,169 U.S. government securities 1,996 - (8) 1,988 Non-U.S. government securities 7,849 4 (1) 7,852 Total $ 187,221 $ 18 $ (431) $ 186,808 |
Fair Value of Financial Instr_2
Fair Value of Financial Instruments (Tables) | 9 Months Ended |
Sep. 30, 2022 | |
Fair Value of Financial Instruments | |
Summary of assets measured at fair value on a recurring basis | Fair value measurement at reporting date using Quoted prices in active Significant markets for other Significant identical observable unobservable assets inputs inputs (in thousands) (Level 1) (Level 2) (Level 3) September 30, 2022: Assets Cash and cash equivalents: Certificates of deposit $ - $ 7,030 $ - Money market funds 15,721 - $ - Commercial paper - 13,811 - Corporate debt securities - 553 - Total cash and cash equivalents 15,721 21,394 - Marketable securities: Certificates of deposit - 21,918 - Commercial paper - 49,334 - Corporate debt securities - 77,990 - U.S. government securities - 1,948 - Total marketable securities - 151,190 - Total financial assets $ 15,721 $ 172,584 $ - December 31, 2021 Assets Cash and cash equivalents: Money market funds $ 84,416 $ - $ - Total cash and cash equivalents 84,416 - - Marketable securities: Certificates of deposit - 5,296 - Commercial paper - 26,503 - Corporate debt securities - 145,169 - U.S. government securities - 1,988 - Non-U.S. government securities - 7,852 - Total marketable securities - 186,808 - Total financial assets $ 84,416 $ 186,808 $ - |
Property and Equipment, net (Ta
Property and Equipment, net (Tables) | 9 Months Ended |
Sep. 30, 2022 | |
Property and Equipment, net | |
Schedule of property and equipment, net | (in thousands) September 30, 2022 December 31, 2021 Laboratory equipment $ 9,753 $ 8,916 Office equipment 601 621 Computer hardware and software 1,089 1,028 Furniture and fixtures 1,203 1,487 Leasehold improvements 13,487 13,409 Construction in progress 993 822 Total property and equipment 27,126 26,283 Accumulated depreciation (4,208) (2,477) $ 22,918 $ 23,806 |
Accrued Expenses and Other Cu_2
Accrued Expenses and Other Current Liabilities (Tables) | 9 Months Ended |
Sep. 30, 2022 | |
Accrued Expenses and Other Current Liabilities | |
Schedule of accrued liabilities and other current liabilities | (in thousands) September 30, 2022 December 31, 2021 Professional fees $ 636 $ 877 Compensation and related benefits 7,006 10,014 Research and development 5,640 8,498 Property and equipment 8 161 Other 571 500 $ 13,861 $ 20,050 |
Leases (Tables)
Leases (Tables) | 9 Months Ended |
Sep. 30, 2022 | |
Leases | |
Schedule of operating lease cost | Nine Months Ended ($ in thousands) September 30, 2022 Operating lease cost $ 2,487 Cash paid for amounts included in the measurement of operating cash flows from operating leases $ 2,009 Weighted-average discount rate 9.7% Weighted-average remaining lease term (years) 12.4 |
Schedule of future minimum rental payments for operating leases | (in thousands) 2022 (remaining) $ 845 2023 3,450 2024 3,548 2025 3,648 2026 3,751 Thereafter 33,507 Total undiscounted lease payments 48,749 Less: imputed interest (21,460) Total lease liabilities $ 27,289 (in thousands) 2022 $ 2,884 2023 3,453 2024 3,550 2025 3,651 2026 3,754 Thereafter 33,534 $ 50,826 |
Share-Based Compensation (Table
Share-Based Compensation (Tables) | 9 Months Ended |
Sep. 30, 2022 | |
Share-Based Compensation | |
Schedule of share-based compensation expense category | Three Months Ended September 30, Nine Months Ended September 30, (in thousands) 2022 2021 2022 2021 Research and development $ 568 $ 2,306 $ 6,528 $ 13,155 General and administrative 2,294 4,509 8,949 11,961 $ 2,862 $ 6,815 $ 15,477 $ 25,116 |
Summary of stock option activity plan | Weighted Weighted average average remaining Number of exercise price contractual shares per share term (years) Outstanding at January 1, 2022 9,416,998 $ 13.72 8.6 Granted 5,707,241 3.37 Exercised (126,056) 1.02 Forfeited (3,259,306) 11.65 Outstanding at September 30, 2022 11,738,877 $ 9.40 7.3 Vested and Exercisable at September 30, 2022 5,510,147 $ 11.95 5.4 Vested or expected to vest at September 30, 2022 11,738,877 $ 9.40 7.3 |
Schedule of weighted average assumptions applied to options | Nine Months Ended September 30, 2022 2021 Expected volatility 92.3 % 98.9 % Risk‑free interest rate 2.6 % 0.9 % Expected term 5.8 years 6.0 years Expected dividend yield — — |
Summary of activity related to restricted stock unit | Weighted average Number of shares grant date fair value Unvested balance at January 1, 2022 290,500 $ 14.78 Granted 261,500 4.21 Vested (20,000) 17.40 Forfeited (122,500) 12.86 Unvested balance at September 30, 2022 409,500 $ 8.48 |
Risks and Liquidity (Details)
Risks and Liquidity (Details) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | ||
Jan. 31, 2021 | Sep. 30, 2022 | Dec. 31, 2021 | |
Risks And Liquidity [Line Items] | |||
Accumulated deficit | $ (465,335) | $ (356,281) | |
Follow-on public offering | |||
Risks And Liquidity [Line Items] | |||
Shares issued | 8,050,000 | ||
Share price | $ 22 | ||
Net proceeds | $ 165,800 |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies (Details) - USD ($) $ in Thousands | 9 Months Ended | ||
Sep. 30, 2022 | Sep. 30, 2021 | Jan. 01, 2022 | |
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | |||
Amount of antidilutive securities excluded from computation of earnings per share | 12,210,865 | 9,869,520 | |
Operating lease liability | $ 27,289 | ||
Right-of-use assets | $ 19,890 | ||
Accounting Standards Update 2016-02 | |||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | |||
Operating lease liability | $ 27,300 | ||
Right-of-use assets | $ 20,400 | ||
Stock options (including shares subject to repurchase) | |||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | |||
Amount of antidilutive securities excluded from computation of earnings per share | 11,738,877 | 9,581,659 | |
Unvested restricted stock units | |||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | |||
Amount of antidilutive securities excluded from computation of earnings per share | 409,500 | 238,333 | |
Employee stock purchase plan | |||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | |||
Amount of antidilutive securities excluded from computation of earnings per share | 62,488 | 49,528 |
Cash, cash equivalents and ma_3
Cash, cash equivalents and marketable securities - Portfolio of Cash and Cash Equivalents (Details) - USD ($) $ in Thousands | Sep. 30, 2022 | Dec. 31, 2021 |
Cash and cash equivalents | ||
Cost or Amortized cost | $ 62,576 | $ 128,965 |
Unrealized losses | (9) | |
Fair value | 62,568 | 128,965 |
Cash accounts in banking institutions | ||
Cash and cash equivalents | ||
Cost or Amortized cost | 25,453 | 44,549 |
Fair value | 25,453 | 44,549 |
Certificates of deposit | ||
Cash and cash equivalents | ||
Cost or Amortized cost | 7,032 | |
Unrealized losses | (2) | |
Fair value | 7,030 | |
Money market funds | ||
Cash and cash equivalents | ||
Cost or Amortized cost | 15,721 | 84,416 |
Fair value | 15,721 | $ 84,416 |
Commercial paper | ||
Cash and cash equivalents | ||
Cost or Amortized cost | 13,817 | |
Unrealized losses | (6) | |
Fair value | 13,811 | |
Corporate debt securities | ||
Cash and cash equivalents | ||
Cost or Amortized cost | 554 | |
Unrealized losses | (1) | |
Fair value | $ 553 |
Cash, cash equivalents and ma_4
Cash, cash equivalents and marketable securities - Portfolio of Marketable Securities (Details) - USD ($) $ in Thousands | Sep. 30, 2022 | Dec. 31, 2021 |
Debt Securities, Available-for-sale, Fair Value to Amortized Cost [Abstract] | ||
Amortized cost | $ 152,649 | $ 187,221 |
Unrealized gains | 3 | 18 |
Unrealized losses | (1,462) | (431) |
Fair value | 151,190 | 186,808 |
Certificates of deposit | ||
Debt Securities, Available-for-sale, Fair Value to Amortized Cost [Abstract] | ||
Amortized cost | 22,012 | 5,296 |
Unrealized gains | 2 | |
Unrealized losses | (96) | |
Fair value | 21,918 | 5,296 |
Commercial paper | ||
Debt Securities, Available-for-sale, Fair Value to Amortized Cost [Abstract] | ||
Amortized cost | 49,451 | 26,503 |
Unrealized gains | 1 | 4 |
Unrealized losses | (118) | (4) |
Fair value | 49,334 | 26,503 |
Corporate debt securities | ||
Debt Securities, Available-for-sale, Fair Value to Amortized Cost [Abstract] | ||
Amortized cost | 79,188 | 145,577 |
Unrealized gains | 10 | |
Unrealized losses | (1,198) | (418) |
Fair value | 77,990 | 145,169 |
U.S. government securities | ||
Debt Securities, Available-for-sale, Fair Value to Amortized Cost [Abstract] | ||
Amortized cost | 1,998 | 1,996 |
Unrealized losses | (50) | (8) |
Fair value | $ 1,948 | 1,988 |
Non-U.S. government securities | ||
Debt Securities, Available-for-sale, Fair Value to Amortized Cost [Abstract] | ||
Amortized cost | 7,849 | |
Unrealized gains | 4 | |
Unrealized losses | (1) | |
Fair value | $ 7,852 |
Fair Value of Financial Instr_3
Fair Value of Financial Instruments (Details) - USD ($) $ in Thousands | Sep. 30, 2022 | Dec. 31, 2021 |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | $ 62,568 | $ 128,965 |
Marketable securities | 151,190 | 186,808 |
Certificates of deposit | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Marketable securities | 21,918 | 5,296 |
Commercial paper | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Marketable securities | 49,334 | 26,503 |
Corporate debt securities | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Marketable securities | 77,990 | 145,169 |
U.S. government securities | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Marketable securities | 1,948 | 1,988 |
Non-U.S. government securities | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Marketable securities | 7,852 | |
Money market funds | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | 15,721 | 84,416 |
Certificates of deposit | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | 7,030 | |
Commercial paper | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | 13,811 | |
Corporate debt securities | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | 553 | |
Level 1 | Recurring | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | 15,721 | 84,416 |
Total financial assets | 15,721 | 84,416 |
Level 1 | Recurring | Money market funds | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | 15,721 | 84,416 |
Level 2 | Recurring | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | 21,394 | |
Marketable securities | 151,190 | 186,808 |
Total financial assets | 172,584 | 186,808 |
Level 2 | Recurring | Certificates of deposit | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Marketable securities | 21,918 | 5,296 |
Level 2 | Recurring | Commercial paper | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Marketable securities | 49,334 | 26,503 |
Level 2 | Recurring | Corporate debt securities | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Marketable securities | 77,990 | 145,169 |
Level 2 | Recurring | U.S. government securities | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Marketable securities | 1,948 | 1,988 |
Level 2 | Recurring | Non-U.S. government securities | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Marketable securities | $ 7,852 | |
Level 2 | Recurring | Certificates of deposit | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | 7,030 | |
Level 2 | Recurring | Commercial paper | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | 13,811 | |
Level 2 | Recurring | Corporate debt securities | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | $ 553 |
Property and Equipment, net (De
Property and Equipment, net (Details) - USD ($) $ in Thousands | Sep. 30, 2022 | Dec. 31, 2021 |
Property, Plant and Equipment [Line Items] | ||
Total property and equipment | $ 27,126 | $ 26,283 |
Accumulated depreciation | (4,208) | (2,477) |
Total, property plant and equipment net | 22,918 | 23,806 |
Laboratory equipment | ||
Property, Plant and Equipment [Line Items] | ||
Total property and equipment | 9,753 | 8,916 |
Office equipment | ||
Property, Plant and Equipment [Line Items] | ||
Total property and equipment | 601 | 621 |
Computer hardware and software. | ||
Property, Plant and Equipment [Line Items] | ||
Total property and equipment | 1,089 | 1,028 |
Furniture and fixtures | ||
Property, Plant and Equipment [Line Items] | ||
Total property and equipment | 1,203 | 1,487 |
Leasehold improvements | ||
Property, Plant and Equipment [Line Items] | ||
Total property and equipment | 13,487 | 13,409 |
Construction in progress | ||
Property, Plant and Equipment [Line Items] | ||
Total property and equipment | $ 993 | $ 822 |
Accrued Expenses and Other Cu_3
Accrued Expenses and Other Current Liabilities (Details) - USD ($) $ in Thousands | Sep. 30, 2022 | Dec. 31, 2021 |
Accrued Expenses and Other Current Liabilities | ||
Professional fees | $ 636 | $ 877 |
Compensation and related benefits | 7,006 | 10,014 |
Research and development | 5,640 | 8,498 |
Property and equipment | 8 | 161 |
Other | 571 | 500 |
Accrued expenses and other current liabilities | $ 13,861 | $ 20,050 |
Leases (Details)
Leases (Details) $ in Thousands | 3 Months Ended | 9 Months Ended | ||||
Sep. 30, 2022 USD ($) item | Sep. 30, 2021 USD ($) | Sep. 30, 2022 USD ($) item | Sep. 30, 2021 USD ($) | Jan. 01, 2022 USD ($) | Dec. 31, 2021 USD ($) | |
Lessee, Lease, Description [Line Items] | ||||||
Rent expense | $ 800 | $ 800 | $ 2,500 | $ 2,500 | ||
Deferred rent | $ 3,200 | $ 6,921 | ||||
Tenant improvement allowances liability | 3,800 | |||||
Operating lease liability | 27,289 | 27,289 | ||||
Right-of-use assets | 19,890 | 19,890 | ||||
Future minimum lease payments | ||||||
2022 (remaining) | 845 | 845 | ||||
2023 | 3,450 | 3,450 | 2,884 | |||
2024 | 3,548 | 3,548 | 3,453 | |||
2025 | 3,648 | 3,648 | 3,550 | |||
2026 | 3,751 | 3,751 | 3,651 | |||
2026 | 3,754 | |||||
Thereafter | 33,534 | |||||
Thereafter | 33,507 | 33,507 | ||||
Total Undiscounted lease payments | 48,749 | 48,749 | $ 50,826 | |||
Less: imputed interest | (21,460) | (21,460) | ||||
Total Lease Liabilities | 27,289 | 27,289 | ||||
Other current assets | ||||||
Lessee, Lease, Description [Line Items] | ||||||
Tenant improvements | $ 100 | $ 100 | ||||
Accounting Standards Update 2016-02 | ||||||
Lessee, Lease, Description [Line Items] | ||||||
Operating lease liability | 27,300 | |||||
Right-of-use assets | 20,400 | |||||
Future minimum lease payments | ||||||
Total Lease Liabilities | $ 27,300 | |||||
Operating Leases For Office Space | ||||||
Lessee, Lease, Description [Line Items] | ||||||
Incremental borrowing rate | 9% | 9% | ||||
Operating Leases For Laboratory Space | ||||||
Lessee, Lease, Description [Line Items] | ||||||
Incremental borrowing rate | 10% | 10% | ||||
Maximum | Operating Leases For Office Space | ||||||
Lessee, Lease, Description [Line Items] | ||||||
Number of extension terms | item | 2 | 2 | ||||
Extended term of new lease agreement | 5 years | |||||
Tenant improvement allowance | $ 2,800 | $ 2,800 | ||||
Maximum | Operating Leases For Laboratory Space | ||||||
Lessee, Lease, Description [Line Items] | ||||||
Number of extension terms | item | 2 | 2 | ||||
Extended term of new lease agreement | 5 years | |||||
Tenant improvement allowance | $ 1,300 | $ 1,300 |
Leases - Operating lease cost (
Leases - Operating lease cost (Details) $ in Thousands | 9 Months Ended |
Sep. 30, 2022 USD ($) | |
Leases | |
Operating lease cost | $ 2,487 |
Cash paid for amounts included in the measurement of operating cash flows from operating leases | $ 2,009 |
Weighted-average discount rate | 9.70% |
Weighted-average remaining lease term (years) | 12 years 4 months 24 days |
Commitments and Contingencies -
Commitments and Contingencies - Collaboration Agreements (Details) $ in Millions | 1 Months Ended | 3 Months Ended | 9 Months Ended | 12 Months Ended | |||||
Aug. 03, 2020 USD ($) Option | Aug. 31, 2021 | Apr. 30, 2020 USD ($) | Jun. 30, 2019 | Sep. 30, 2022 USD ($) | Sep. 30, 2021 USD ($) | Sep. 30, 2022 USD ($) | Sep. 30, 2021 USD ($) | Dec. 31, 2021 USD ($) | |
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||||
Development milestone payment | $ 0 | $ 0 | |||||||
Catalent Maryland, Inc. (Catalent) | |||||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||||
Annual fee payable, term | 5 years | ||||||||
Period for which annual fee is payable | 5 years | ||||||||
Term of manufacturing and supply agreement | 5 years | ||||||||
Additional extension term of manufacturing and supply agreement | 5 years | ||||||||
Minimum amount agreed to purchase in batches of drug product | $ 10.6 | ||||||||
Amended and restated agreement with Penn | |||||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||||
Annual fee payable | $ 5 | ||||||||
Quarterly fee payable | $ 1.3 | ||||||||
Number of CNS indications | Option | 8 | ||||||||
Upfront payments | $ 1 | ||||||||
Upfront fee payable upon clinical candidate designation | 0.5 | ||||||||
Upfront fee due | $ 0.5 | ||||||||
Payments for technology license fee | $ 5 | $ 5 | |||||||
Additional technology license fee paid | 1.5 | 3 | $ 0.5 | $ 1.5 | |||||
Payment made on option exercise | $ 0.5 | $ 0.5 | |||||||
Amount payable per product candidate arising from the exploratory program for large CNS indications | 39 | 39 | |||||||
Sales milestone payments | 55 | 55 | |||||||
Amended and restated agreement with Penn | Minimum | |||||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||||
Collaboration agreement, tiered transaction fee percent | 1% | ||||||||
Amended and restated agreement with Penn | Maximum | |||||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||||
Collaboration agreement, tiered transaction fee percent | 2% | ||||||||
Amount payable per product candidate for rare, monogenic disorders | $ 16.5 | $ 16.5 |
Share-Based Compensation - Equi
Share-Based Compensation - Equity Incentive Plan & ESPP (Details) | 3 Months Ended | 9 Months Ended | ||||
Jan. 01, 2022 shares | Feb. 28, 2020 USD ($) shares | Sep. 30, 2022 USD ($) shares | Sep. 30, 2021 USD ($) | Sep. 30, 2022 USD ($) plan shares | Sep. 30, 2021 USD ($) | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||
Number of equity incentive plans | plan | 3 | |||||
Number of shares purchased | 4,000 | |||||
Share-based compensation expense | $ | $ 2,862,000 | $ 6,815,000 | $ 15,477,000 | $ 25,116,000 | ||
Equity Incentive Plan 2020 | ||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||
Shares authorized | 10,370,926 | 10,370,926 | ||||
Shares available for future grant | 3,910,223 | 3,910,223 | ||||
Period till which there is automatic increase in rights granted under the Plan | 10 years | |||||
Percent of the total number of shares of the Company's common stock outstanding | 5% | |||||
Term of award | 10 years | |||||
Vesting period | 4 years | |||||
Additional number of common shares reserved for future issuance | 2,712,249 | |||||
Equity Inducement Plan 2021 | ||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||
Shares authorized | 2,000,000 | 2,000,000 | ||||
Shares available for future grant | 865,500 | 865,500 | ||||
Term of award | 10 years | |||||
Employee Stock Purchase Plan | ||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||
Shares authorized | 1,435,619 | 1,435,619 | ||||
Shares available for future grant | 1,168,841 | 1,168,841 | ||||
Additional number of common shares reserved for future issuance | 542,449 | |||||
Period till which there is automatic increase in rights granted under ESPP | 10 years | |||||
Percentage of common stock outstanding | 1% | |||||
Percentage of stock purchase price | 85% | |||||
Percentage of maximum employee contribution | 15% | |||||
Maximum amount of common stock that can be purchased | $ | $ 25,000 | |||||
Share-based compensation expense | $ | $ 100,000 | $ 100,000 | $ 200,000 | $ 300,000 |
Share-Based Compensation - Shar
Share-Based Compensation - Share-based compensation expense (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2022 | Sep. 30, 2021 | Sep. 30, 2022 | Sep. 30, 2021 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Share-based compensation expense | $ 2,862 | $ 6,815 | $ 15,477 | $ 25,116 |
Share-based compensation expense due to modifications | 0 | |||
Research and development | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Share-based compensation expense | 568 | 2,306 | 6,528 | 13,155 |
Share-based compensation expense due to modifications | 6,100 | |||
General and administrative | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Share-based compensation expense | $ 2,294 | 4,509 | 8,949 | 11,961 |
Share-based compensation expense due to modifications | $ 200 | $ 400 | $ 200 |
Share-Based Compensation - Stoc
Share-Based Compensation - Stock Option Activity (Details) - USD ($) $ / shares in Units, $ in Millions | 9 Months Ended | 12 Months Ended | |
Sep. 30, 2022 | Sep. 30, 2021 | Dec. 31, 2021 | |
Number of shares | |||
Outstanding, Beginning of period | 9,416,998 | ||
Granted | 5,707,241 | ||
Exercised | (126,056) | ||
Forfeited | (3,259,306) | ||
Outstanding, Ending of period | 11,738,877 | 9,416,998 | |
Vested and Exercisable at September 30, 2022 | 5,510,147 | ||
Vested or expected to vest at September 30, 2022 | 11,738,877 | ||
Weighted average exercise price per share | |||
Outstanding, Beginning of period | $ 13.72 | ||
Granted | 3.37 | ||
Exercised | 1.02 | ||
Forfeited | 11.65 | ||
Outstanding, Ending of period | 9.40 | $ 13.72 | |
Vested and Exercisable at September 30, 2022 | 11.95 | ||
Vested or expected to vest at September 30, 2022 | $ 9.40 | ||
Weighted average remaining contractual term (years) | |||
Outstanding | 7 years 3 months 18 days | 8 years 7 months 6 days | |
Vested and Exercisable at September 30, 2022 | 5 years 4 months 24 days | ||
Vested or expected to vest at September 30, 2022 | 7 years 3 months 18 days | ||
Weighted average grant date fair value of options granted | $ 2.52 | $ 13.64 | |
Unrecognized compensation expense related to unvested stock option awards | $ 32.6 | ||
Weighted average period for recognition | 2 years 4 months 24 days |
Share-Based Compensation - Assu
Share-Based Compensation - Assumptions Used in Determining Fair Value & Early exercise of Stock Options (Details) | 9 Months Ended | |
Sep. 30, 2022 | Sep. 30, 2021 | |
Share-based Compensation Arrangement by Share-based Payment Award, Fair Value Assumptions and Methodology [Abstract] | ||
Expected volatility | 92.30% | 98.90% |
Risk-free interest rate | 2.60% | 0.90% |
Expected term | 5 years 9 months 18 days | 6 years |
Share-Based Compensation - Rest
Share-Based Compensation - Restricted Stock Units (Details) - Unvested restricted stock units - USD ($) $ / shares in Units, $ in Millions | 9 Months Ended | |
Sep. 30, 2022 | Dec. 31, 2021 | |
Number of shares | ||
Unvested balance, Beginning | 290,500 | |
Granted | 261,500 | |
Vested | (20,000) | |
Forfeited | (122,500) | |
Unvested balance, Ending | 409,500 | |
Weighted average grant date fair value | ||
Unvested balance, Weighted average grant date fair value, Beginning | $ 8.48 | $ 14.78 |
Granted, Weighted average grant date fair value | 4.21 | |
Vested, Weighted average grant date fair value | 17.40 | |
Forfeited, Weighted average grant date fair value | 12.86 | |
Unvested balance, Weighted average grant date fair value, Ending | $ 8.48 | |
Total unrecognized expense | $ 2.5 | |
Weighted-average remaining contractual term | 2 years 3 months 18 days |
Subsequent Events (Details)
Subsequent Events (Details) | Nov. 30, 2022 |
Subsequent Events | |
Subsequent Events | |
Percent of percent reduction to our workforce | 23% |