Document and Entity Information
Document and Entity Information - shares | 6 Months Ended | |
Jun. 30, 2020 | Aug. 05, 2020 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Jun. 30, 2020 | |
Document Fiscal Year Focus | 2020 | |
Document Fiscal Period Focus | Q2 | |
Trading Symbol | PRVL | |
Entity Registrant Name | Prevail Therapeutics Inc. | |
Entity Central Index Key | 0001714798 | |
Current Fiscal Year End Date | --12-31 | |
Entity Filer Category | Non-accelerated Filer | |
Entity Small Business | true | |
Entity Emerging Growth Company | true | |
Entity Ex Transition Period | true | |
Entity Shell Company | false | |
Entity Current Reporting Status | Yes | |
Entity Address, State or Province | NY | |
Entity Common Stock, Shares Outstanding | 34,214,851 | |
Title of 12(b) Security | Common Stock, par value $0.0001 per share | |
Security Exchange Name | NASDAQ | |
Entity Interactive Data Current | Yes | |
Entity File Number | 001-38939 | |
Entity Tax Identification Number | 82-2129632 | |
Entity Address, Address Line One | 430 East 29th Street | |
Entity Address, Address Line Two | Suite 1520 | |
Entity Address, City or Town | New York | |
Entity Address, Postal Zip Code | 10016 | |
City Area Code | 917 | |
Local Phone Number | 336-9310 | |
Document Quarterly Report | true | |
Document Transition Report | false | |
Entity Incorporation, State or Country Code | DE |
Balance Sheets (Unaudited)
Balance Sheets (Unaudited) - USD ($) $ in Thousands | Jun. 30, 2020 | Dec. 31, 2019 |
CURRENT ASSETS: | ||
Cash and cash equivalents | $ 111,065 | $ 168,051 |
Investments | 6,458 | 0 |
Prepaid expenses and other current assets | 4,620 | 6,410 |
Total current assets | 122,143 | 174,461 |
Property and equipment, net | 2,698 | 2,549 |
Investments | 13,674 | 0 |
Operating lease right-of-use assets | 9,355 | 10,001 |
Other long-term assets | 2,730 | 0 |
Restricted cash | 91 | 91 |
TOTAL ASSETS | 150,691 | 187,102 |
CURRENT LIABILITIES: | ||
Accounts payable | 1,981 | 5,162 |
Accrued expenses and other current liabilities | 9,435 | 5,330 |
Operating lease liabilities | 1,447 | 1,341 |
Total current liabilities | 12,863 | 11,833 |
Long-term operating lease liabilities | 9,173 | 9,927 |
TOTAL LIABILITIES | 22,036 | 21,760 |
COMMITMENTS AND CONTINGENCIES (Note 13) STOCKHOLDERS’ EQUITY | 0 | 0 |
Preferred stock - $0.0001 par value, 10,000,000 shares authorized as of June 30, 2020 and December 31, 2019, respectively; no shares issued as of June 30, 2020 and December 31, 2019, respectively | 0 | 0 |
Common stock - $0.0001 par value, 200,000,000 shares authorized as of June 30, 2020 and December 31, 2019, respectively, 34,214,851 and 34,138,750 shares issued and outstanding as of June 30, 2020 and December 31, 2019, respectively | 3 | 3 |
Additional paid-in capital | 253,430 | 249,441 |
Accumulated deficit | (124,777) | (84,102) |
Accumulated other comprehensive loss | (1) | 0 |
Total stockholders’ equity | 128,655 | 165,342 |
TOTAL LIABILITIES AND STOCKHOLDERS’ EQUITY | $ 150,691 | $ 187,102 |
Balance Sheets (Parenthetical)
Balance Sheets (Parenthetical) (Unaudited) - $ / shares | Jun. 30, 2020 | Dec. 31, 2019 |
Statement Of Financial Position [Abstract] | ||
Preferred stock, par value | $ 0.0001 | $ 0.0001 |
Preferred stock, shares authorized | 10,000,000 | 10,000,000 |
Preferred stock, shares issued | 0 | 0 |
Common stock, par value | $ 0.0001 | $ 0.0001 |
Common stock, shares authorized | 200,000,000 | 200,000,000 |
Common stock, shares issued | 34,214,851 | 34,138,750 |
Common stock, shares outstanding | 34,214,851 | 34,138,750 |
Statements of Operations and Co
Statements of Operations and Comprehensive Loss (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2020 | Jun. 30, 2019 | Jun. 30, 2020 | Jun. 30, 2019 | |
Operating Expenses: | ||||
Research and development | $ 12,943 | $ 11,955 | $ 24,360 | $ 20,366 |
General and administrative | 9,208 | 3,713 | 17,070 | 5,598 |
Operating expenses | (22,151) | (15,668) | (41,430) | (25,964) |
Other income | 0 | 0 | 210 | 0 |
Interest income, net | 51 | 565 | 545 | 916 |
Total other income | 51 | 565 | 755 | 916 |
Net loss | (22,100) | (15,103) | (40,675) | (25,048) |
Other comprehensive loss | ||||
Net unrealized loss on investments | (1) | 0 | (1) | 0 |
Total other comprehensive loss | (1) | 0 | (1) | 0 |
Comprehensive loss | $ (22,101) | $ (15,103) | $ (40,676) | $ (25,048) |
Net loss per share | ||||
Basic and Diluted | $ (0.66) | $ (0.58) | $ (1.22) | $ (1.05) |
Weighted average shares outstanding: | ||||
Basic and Diluted | 33,467,346 | 26,212,356 | 33,367,344 | 23,945,198 |
Statements of Changes in Redeem
Statements of Changes in Redeemable Convertible Preferred Stock and Stockholders' Equity (Deficit) (Unaudited) - USD ($) $ in Thousands | Total | Series Seed Preferred Stock | Series A Preferred Stock | Series B Preferred Stock | Common Stock | Additional Paid-In Capital | Accumulated Deficit | Accumulated Other Comprehensive Loss [Member] |
Balance at Dec. 31, 2018 | $ (18,417) | $ 1 | $ 2,496 | $ (20,914) | ||||
Balance, temporary equity, shares at Dec. 31, 2018 | 6,480,000 | 8,997,085 | ||||||
Balance, temporary equity at Dec. 31, 2018 | $ 3,524 | $ 76,186 | ||||||
Balance, shares at Dec. 31, 2018 | 7,209,000 | |||||||
Issuance of Preferred Stock | $ 49,834 | |||||||
Issuance of Preferred Stock, shares | 3,958,046 | |||||||
Stock-based compensation | 1,721 | 1,721 | ||||||
Conversion of convertible preferred stock into common stock upon the closing of initial public offering | 129,543 | $ (3,524) | $ (76,186) | $ (49,834) | $ 1 | 129,542 | ||
Conversion of convertible preferred stock into common stock upon the closing of initial public offering, shares | (6,480,000) | (8,997,085) | (3,958,046) | 19,435,131 | ||||
Issuance of common stock upon closing of initial public offering, net of issuance costs | 113,236 | $ 1 | 113,235 | |||||
Issuance of common stock upon closing of initial public offering, net of issuance costs, shares | 7,353,000 | |||||||
Exercise of stock options | 4 | 4 | ||||||
Exercise of stock options, shares | 24,063 | |||||||
Net loss | (25,048) | (25,048) | ||||||
Balance at Jun. 30, 2019 | 201,039 | $ 3 | 246,998 | (45,962) | ||||
Balance, shares at Jun. 30, 2019 | 34,021,194 | |||||||
Balance at Mar. 31, 2019 | (27,750) | $ 1 | 3,108 | (30,859) | ||||
Balance, temporary equity, shares at Mar. 31, 2019 | 6,480,000 | 8,997,085 | 3,958,046 | |||||
Balance, temporary equity at Mar. 31, 2019 | $ 3,524 | $ 76,186 | $ 49,834 | |||||
Balance, shares at Mar. 31, 2019 | 7,209,000 | |||||||
Stock-based compensation | 1,109 | 1,109 | ||||||
Conversion of convertible preferred stock into common stock upon the closing of initial public offering | 129,543 | $ (3,524) | $ (76,186) | $ (49,834) | $ 1 | 129,542 | ||
Conversion of convertible preferred stock into common stock upon the closing of initial public offering, shares | (6,480,000) | (8,997,085) | (3,958,046) | 19,435,131 | ||||
Issuance of common stock upon closing of initial public offering, net of issuance costs | 113,236 | $ 1 | 113,235 | |||||
Issuance of common stock upon closing of initial public offering, net of issuance costs, shares | 7,353,000 | |||||||
Exercise of stock options | 4 | 4 | ||||||
Exercise of stock options, shares | 24,063 | |||||||
Net loss | (15,103) | (15,103) | ||||||
Balance at Jun. 30, 2019 | 201,039 | $ 3 | 246,998 | (45,962) | ||||
Balance, shares at Jun. 30, 2019 | 34,021,194 | |||||||
Balance at Dec. 31, 2019 | $ 165,342 | $ 3 | 249,441 | (84,102) | ||||
Balance, shares at Dec. 31, 2019 | 34,138,750 | 34,138,750 | ||||||
Stock-based compensation | $ 3,881 | 3,881 | ||||||
Conversion of convertible preferred stock into common stock upon the closing of initial public offering, shares | 19,435,131 | |||||||
Exercise of stock options | 108 | 108 | ||||||
Exercise of stock options, shares | 76,101 | |||||||
Net loss | (40,675) | (40,675) | ||||||
Net unrealized loss on investments | (1) | $ (1) | ||||||
Balance at Jun. 30, 2020 | $ 128,655 | $ 3 | 253,430 | (124,777) | (1) | |||
Balance, shares at Jun. 30, 2020 | 34,214,851 | 34,214,851 | ||||||
Balance at Mar. 31, 2020 | $ 148,461 | $ 3 | 251,135 | (102,677) | ||||
Balance, shares at Mar. 31, 2020 | 34,196,456 | |||||||
Stock-based compensation | 2,249 | 2,249 | ||||||
Exercise of stock options | 46 | 46 | ||||||
Exercise of stock options, shares | 18,395 | |||||||
Net loss | (22,100) | (22,100) | ||||||
Net unrealized loss on investments | (1) | (1) | ||||||
Balance at Jun. 30, 2020 | $ 128,655 | $ 3 | $ 253,430 | $ (124,777) | $ (1) | |||
Balance, shares at Jun. 30, 2020 | 34,214,851 | 34,214,851 |
Statements of Changes in Rede_2
Statements of Changes in Redeemable Convertible Preferred Stock and Stockholders' Equity (Deficit) (Parenthetical) (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | |
Jun. 30, 2019 | Mar. 31, 2019 | Jun. 30, 2019 | |
Series B Preferred Stock | |||
Issuance costs | $ 166 | ||
Initial Public Offering | |||
Issuance costs | $ 3,016 | $ 3,016 |
Statements of Cash Flows (Unaud
Statements of Cash Flows (Unaudited) - USD ($) $ in Thousands | 6 Months Ended | |
Jun. 30, 2020 | Jun. 30, 2019 | |
Cash flows from operating activities | ||
Net loss | $ (40,675) | $ (25,048) |
Adjustments to reconcile net loss to net cash used in operating activities | ||
Net amortization of premiums and accretion of discounts on available-for-sale securities | 12 | |
Depreciation | 225 | 93 |
Stock-based compensation | 3,881 | 1,721 |
Changes in operating assets and liabilities | ||
Prepaid expenses and other current assets | 1,790 | (3,690) |
Accrued interest on investments | (15) | |
Other long-term assets | (2,689) | |
Operating lease right-of-use asset | 646 | 1,018 |
Accounts payable | (3,181) | 762 |
Accrued expenses and other current liabilities | 4,064 | 3,078 |
Operating lease liabilities | (648) | (388) |
Net cash used in operating activities | (36,590) | (22,454) |
Cash flows from investing activities | ||
Purchases of property and equipment | (374) | (1,538) |
Purchases of investments | (20,130) | |
Net cash used in investing activities | (20,504) | (1,538) |
Cash flows from financing activities | ||
Proceeds from issuance of common stock in initial public offering | 116,251 | |
Payment of issuance costs for common stock in initial public offering | (3,016) | |
Proceeds from exercise of stock options | 108 | 4 |
Net cash provided by financing activities | 108 | 163,073 |
Net (decrease) increase in cash, cash equivalents and restricted cash | (56,986) | 139,081 |
Cash, cash equivalents, and restricted cash at beginning of period | 168,142 | 63,105 |
Cash, cash equivalents, and restricted cash at end of period | 111,156 | 202,186 |
Supplemental disclosure of non-cash investing and financing activities | ||
Conversion of preferred stock to common stock upon the initial public offering | 129,543 | |
Issuance costs associated with public offering included in accrued expenses and other current liabilities | $ 41 | |
Series B Preferred Stock | ||
Cash flows from financing activities | ||
Proceeds from issuance of Series B preferred stock | 50,000 | |
Payment of issuance costs for Series B preferred stock | $ (166) |
Statements of Cash Flows (Paren
Statements of Cash Flows (Parenthetical) (Unaudited) - USD ($) $ in Thousands | Jun. 30, 2020 | Jun. 30, 2019 |
Reconciliation of cash, cash equivalents and restricted cash reported within the Balance Sheets: | ||
Cash and cash equivalents | $ 111,065 | $ 202,095 |
Restricted cash | 91 | 91 |
Total cash, cash equivalents and restricted cash | $ 111,156 | $ 202,186 |
Nature of the Business
Nature of the Business | 6 Months Ended |
Jun. 30, 2020 | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | |
Nature of the Business | 1. NATURE OF THE BUSINESS Prevail Therapeutics Inc., or the Company, was incorporated in the State of Delaware on July 6, 2017. The Company is a clinical-stage gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with devastating neurodegenerative diseases The Company is applying a precision medicine approach to neurodegeneration by studying its gene therapies in genetically defined patient populations. The Company believes this will increase the probability of creating disease-modifying therapies that improve patients’ lives. The Company’s lead clinical-stage programs are PR001 for the treatment of Parkinson’s disease with GBA1 mutations, or PD-GBA, and neuronopathic (Type 2 or Type 3) Gaucher disease, or nGD, and PR006 for the treatment of frontotemporal dementia with GRN mutations, or FTD-GRN. The Company is also focused on developing a broad pipeline of gene therapies for a range of neurodegenerative diseases, including PR004 for the treatment of synucleinopathies, and other programs. The Company is subject to a number of risks common to early-stage companies in the biotechnology industry. Principal among these risks are the uncertainties in the development process, development of the same or similar technological innovations by competitors, protection of proprietary technology, dependence on key personnel, compliance with government regulations and approval requirements, and the need to obtain additional financing to fund operations. Product candidates currently under development will require significant additional research and development efforts, including extensive pre-clinical and clinical testing and regulatory approval, prior to commercialization. These efforts require significant amounts of additional capital, adequate personnel infrastructure, and extensive compliance-reporting capabilities. There can be no assurance that the Company’s research and development will be successfully completed, that adequate protection for the Company’s technology will be obtained, that any products developed will obtain necessary government regulatory approval, or that any approved products will be commercially viable. The Company operates in an environment of rapid change in technology and substantial competition from pharmaceutical and biotechnology companies. In addition, the Company is dependent upon the services of its employees, consultants and contractors. Stock Split - In June 2019, the Board of Directors of the Company approved a 1.62-for-one forward stock-split of the Company’s outstanding shares of common stock, convertible preferred stock and options outstanding and available for future issuance. The stock split became effective on June 7, 2019. Accordingly, all share and per share amounts for all periods presented in the accompanying financial statements and notes thereto have been retroactively adjusted, where applicable, to reflect this forward stock split. Shares of common stock underlying outstanding stock options and other equity instruments were proportionately increased and the respective per share value and exercise prices, if applicable, were proportionately decreased in accordance with the terms of the agreements governing such securities. Initial Public Offering - In June 2019, the Company completed its initial public offering, or IPO, whereby the Company sold an aggregate of 7,353,000 shares of its common stock at a price of $17.00 per share. The shares began trading on The Nasdaq Global Select Market on June 20, 2019. The aggregate net proceeds received by the Company from the offering were approximately $113.0 million, after deducting underwriting discounts and commissions and offering expenses payable by the Company of $12.0 million. Upon the closing of the IPO, all outstanding shares of redeemable convertible preferred stock converted into 19,435,131 shares of common stock. Additionally, the Company is authorized to issue 200,000,000 shares of common stock and 10,000,000 shares of preferred stock. On June 24, 2019, the Company filed an Amended and Restated Certificate of Incorporation with the Secretary of State of the State of Delaware, in connection with the closing of the IPO. Liquidity and Capital Resources- Since its inception, the Company has incurred operating losses and has consistently used cash to fund operations. The Company has not recognized any revenue to date, devoting its efforts and capital resources to research and development of its product candidates. The Company’s activities have been primarily funded by the sale of shares of convertible preferred stock and common stock (see Note 8). The Company manages its capital resources to ensure the Company will continue as a going concern while maximizing the return to stockholders through the optimization of the debt and equity balances. The Company’s cash and cash equivalents as of June 30, 2020 and December 31, 2019 were $111.1 million and $168.1 million respectively and the Company held investments of $20.1 million as of June 30, 2020 that are available to be sold for cash. In March 2019, the Company raised an aggregate of $49.8 million of net proceeds from its Series B Preferred Stock financing. In June 2019, the Company completed its IPO whereby the Company sold an aggregate of 7,353,000 shares of its common stock for aggregate net proceeds of approximately $113.0 million. The Company estimates that its cash and cash equivalents and available-for-sale investments balances as of June 30, 2020, will be sufficient to enable it to fund its operating expenses and capital expenditure requirements for at least 12 months from the issuance date of these financial statements. This estimate is based on assumptions that may prove to be incorrect, and the Company could use its available capital resources sooner than currently expected. Changing circumstances could cause the Company to consume capital resources sooner than currently anticipated, and the Company may need to spend more than currently planned due to circumstances beyond its control. |
Significant Accounting Policies
Significant Accounting Policies | 6 Months Ended |
Jun. 30, 2020 | |
Accounting Policies [Abstract] | |
Significant Accounting Policies | 2. SIGNIFICANT ACCOUNTING POLICIES Basis of Presentation— The Company’s unaudited interim financial statements have been prepared by the Company in accordance with accounting principles generally accepted in the United States, or U.S. GAAP, for interim information and pursuant to the rules and regulations of the U.S. Securities and Exchange Commission, or the SEC, for reporting on Form 10-Q. Accordingly, certain information and footnote disclosures normally included in financial statements prepared in accordance with U.S. GAAP have been omitted pursuant to such rules and regulations. These unaudited interim financial statements should be read in conjunction with the audited financial statements as of and for the year ended December 31, 2019, and the notes thereto, which are included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2019. The COVID-19 pandemic has interrupted business operations across the globe. Estimates and assumptions about future events and their effects cannot be determined with certainty and therefore require the exercise of judgment. As of the date of issuance of these financial statements, the Company is not aware of any specific event or circumstance that would require the Company to update its estimates, assumptions and judgments or revise the reported amounts of assets and liabilities or the disclosure of contingent assets and liabilities. These estimates, however, may change as new events occur and additional information is obtained, and are recognized in the financial statements as soon as they become known. Use of Estimates —The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the financial statements, and the reported amounts of expenses during the reporting period. Significant estimates in the financial statements include, but are not limited to stock-based compensation, operating lease right-of-use assets and liabilities, the recoverability of the Company’s net deferred tax assets and related valuation allowance, and accrued expenses related to expenses incurred for research and development from external vendors. The Company bases its estimates on historical experience, known trends and other market-specific or other relevant factors that it believes to be reasonable under the circumstances. The Company tracks the progress of its various research and development studies and manufacturing projects to ensure related prepaid expenses and accrued expenses are in line with progress of each. On an ongoing basis, management evaluates its estimates, as there are changes in circumstances, facts and experience. Actual results may differ materially from those estimates or assumptions. Cash, Cash Equivalents and Restricted Cash —The Company’s cash and cash equivalents include short-term highly liquid investments which are readily convertible into cash. These investments include money market securities and commercial paper with maturities of three months or less when acquired. The Company’s institutional money market accounts permit daily redemption and the fair values of these investments are based upon the quoted prices in active markets provided by the holding financial institutions, which are considered Level 1 inputs in the fair value hierarchy, as described below. The Company had cash and cash equivalents of $111.1 million as of June 30, 2020. Restricted cash represents cash on deposit with a financial institution as collateral in support of a letter of credit outstanding in favor of the Company’s landlord for office space. The restricted cash balance has been excluded from the cash balance and is classified as non-current restricted cash on the balance sheets as the lease expires after June 30, 2021. Investments — The Company classifies investments in debt securities with remaining maturities when purchased of greater than three months as available-for-sale. Investments with a remaining maturity date greater than one year are classified as non-current. Available-for-sale securities are maintained by investment managers and consist of corporate bonds and U.S. government agency securities. Available-for-sale securities are carried at fair value with the unrealized gains and losses included in other comprehensive loss as a component of stockholders’ equity until realized. The Company had $20.1 million in total available-for-sale debt securities as of June 30, 2020. Any premium or discount arising at purchase is amortized and/or accreted to interest income and/or expensed over the life of the instrument. If any adjustment to fair value reflects a decline in the value of the investment, the Company considers all available evidence to evaluate the extent to which the decline is “other-than-temporary” and, if so, marks the investment to market through a change to the Company’s statement of operations and comprehensive loss. The Company does not intend to sell the investments and it is not more likely than not that the Company will be required to sell the investments before recovery of their amortized costs bases, which may be maturity. Realized gains and losses are included in the statement of operations and comprehensive loss in interest income, net and on a specific-identification basis. There were no realized gains or losses on investments for the three and six months ended June 30, 2020. There were no investments that had been in an unrealized loss position for more than 12 months as of June 30, 2020 Concentration of Credit Risk —The Company maintains cash deposits in excess of government-provided insurance limits. The Company maintains its cash balances with two accredited financial institutions, and accordingly, such funds are not exposed to significant credit risk. Leases —The Company determines if an arrangement is a lease at inception. Operating leases are included in operating lease right-of-use, or ROU, assets, operating lease liabilities, and long-term operating lease liabilities in the Company’s balance sheets. ROU assets represent the Company’s right to use an underlying asset for the lease term and lease liabilities represent the Company’s obligation to make lease payments arising from the lease. Operating lease ROU assets and liabilities are recognized at commencement date based on the present value of lease payments over the lease term. As the Company’s leases do not provide a readily determinable implicit rate, the Company’s uses its incremental borrowing rate based on the information available at commencement date in determining the present value of lease payments. The operating lease ROU asset also includes any lease prepayments, offset by lease incentives. The Company’s facilities operating leases have lease and non-lease components for which the Company has elected to apply the practical expedient and account for each lease component and related non-lease component as one single component. Operating lease cost is recognized on a straight-line basis over the lease term. Property and Equipment, Net —Property and equipment are stated at cost less accumulated depreciation. Depreciation expense is recognized using the straight-line method over the estimated useful life of the asset, ranging from 3-7 years as follows: Fixed Asset Type Estimated useful life Laboratory Equipment 7 Years Leasehold Improvements Lesser of useful life or remaining lease term Computer Equipment 3 Years Furniture and Fixtures 7 Years Expenditures for repairs and maintenance of assets are charged to expense as incurred, while major betterments are capitalized. Upon retirement or sale, the cost and related accumulated depreciation of assets disposed of are removed from the accounts and any resulting gain or loss is included in the statements of operations and comprehensive loss. Impairment of Long-Lived Assets —Long-lived assets, comprised of property and equipment, to be held and used and the right-of-use asset associated with the Company’s leased office space are tested for recoverability whenever events or changes in business circumstances indicate that the carrying amount of the assets may not be fully recoverable. Factors that the Company considers in deciding when to perform an impairment review include significant underperformance of the business in relation to expectations, significant negative industry or economic trends and significant changes or planned changes in the use of the assets. If an impairment review is performed to evaluate a long-lived asset for recoverability, the Company compares forecasts of undiscounted cash flows expected to result from the use and eventual disposition of the long-lived asset to its carrying value. An impairment loss would be recognized when estimated undiscounted future cash flows expected to result from the use of an asset are less than its carrying amount. The impairment loss would be based on the excess of the carrying value of the impaired asset over its current fair value. To date, the Company has not recorded any impairment losses on long-lived assets. Other Long-Term Assets – At June 30, 2020 the Company had other long-term assets that primarily consisted of advanced payments made to the contract research organization responsible for conducting the Company’s PROPEL, PROVIDE and PROCLAIM clinical trials. Comprehensive Loss — During the three and six months ended June 30, 2020, the Company recorded unrealized gains and losses associated with its available-for sale debt securities in other comprehensive loss. Fair Value Measurements —Certain assets and liabilities are carried at fair value under U.S. GAAP. Fair value is defined as the exchange price that would be received for an asset or paid to transfer a liability (an exit price) in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants on the measurement date. Valuation techniques used to measure fair value must maximize the use of observable inputs and minimize the use of unobservable inputs. A framework is used for measuring fair value utilizing a three-tier hierarchy that prioritizes the inputs to valuation techniques used to measure fair value. Financial assets and liabilities carried at fair value are to be classified and disclosed in one of the following three levels of the fair value hierarchy, of which the first two are considered observable and the last is considered unobservable: • Level 1 —Unadjusted quoted prices in active markets for identical assets or liabilities. • Level 2 —Observable inputs (other than Level 1 quoted prices), such as quoted prices in active markets for similar assets or liabilities, quoted prices in markets that are not active for identical or similar assets or liabilities, or other inputs that are observable or can be corroborated by observable market data. • Level 3 —Unobservable inputs that are supported by little or no market activity and that are significant to determining the fair value of the assets or liabilities, including pricing models, discounted cash flow methodologies and similar techniques. The following table summarizes assets measured at fair value on a recurring basis at June 30, 2020 and December 31, 2019 (and does not include cash of $0.2 million and $0.2 million, respectively): Active Markets (Level 1) Observable Inputs (Level 2) Unobservable Inputs (Level 3) June 30, 2020 (in thousands) Cash and cash equivalents: Money Market Funds $ 110,846 $ — $ — Investments: Corporate bonds 6,458 — — U.S. treasury securities 13,674 — — Restricted cash: Money Market Funds 91 — — Total $ 131,069 $ — $ — Active Markets (Level 1) Observable Inputs (Level 2) Unobservable Inputs (Level 3) December 31, 2019 (in thousands) Cash and cash equivalents: Money Market Funds $ 167,805 $ — $ — Restricted cash: Money Market Funds 91 — — Total $ 167,896 $ — $ — There have been no changes to the valuation methods utilized by the Company, nor were there transfers between Level 1, Level 2 and Level 3 investments during the three and six months ended June 30, 2020. there were no financial instruments classified as Level 3 investments. Research and Development Costs —Research and development costs are expensed as incurred. Research and development expenses consist principally of personnel costs, including salaries, stock-based compensation, and benefits for research and development employees, costs related to third-party contract research organizations, contract development and manufacturing organizations, other third-party research service providers, third-party license fees, other direct and indirect operational costs related to the Company’s research and development activities, including facility-related expenses. Non-refundable research and development advance payments are capitalized and expensed as the related goods are delivered or services are performed. Stock-Based Compensation —The Company measures all stock options and other stock-based awards granted to employees, directors, consultants and other nonemployees based on the fair value on the date of the grant and recognizes compensation expense of those awards over the requisite service period, which is generally the vesting period of the respective award. The Company recognizes forfeitures at the time forfeitures occur. The Company classifies stock-based compensation expense in its statement of operations and comprehensive loss in the same way the payroll costs or service payments are classified for the related stock-based award recipient. The fair value of each stock option grant is estimated on the date of grant using the Black-Scholes option pricing model. The Company lacks company specific historical and implied volatility information. Therefore, it estimates its expected stock volatility based on the historical volatility of a publicly-traded set of peer companies and expects to continue to do so until it has adequate historical data regarding the volatility of its own traded stock price. Income Taxes —The Company accounts for income taxes using the asset and liability method, which requires the recognition of deferred tax assets and liabilities for the expected future tax consequences of events that have been recognized in the financial statements or the Company’s tax returns. Under this method, deferred tax assets and liabilities are determined on the basis of the differences between the financial statements and tax basis of the assets and liabilities using enacted tax rates in effect for the year in which the differences are expected to reverse. The effect of a change in tax rates on deferred tax assets and liabilities is recognized in income in the period that includes the enactment date. Changes in deferred tax assets and liabilities are recorded in the provision for income taxes. The Company recognizes deferred tax assets to the extent that the Company believes that these assets are more likely than not to be realized. In making such a determination, management considers all available positive and negative evidence, including future reversals of existing taxable temporary differences, projected future taxable income, tax-planning strategies, and results of recent operations. Valuation allowances are provided, if based upon the weight of available evidence, it is more likely than not that some or all of the deferred tax assets will not be realized. If management determines that the Company would be able to realize its deferred tax assets in the future in excess of their net recorded amount, management would make an adjustment to the deferred tax asset valuation allowance, which would reduce the provision for income taxes. The Company records uncertain tax positions in accordance with ASC 740 on the basis of a two-step process in which (1) management determines whether it is more likely than not that the tax positions will be sustained on the basis of the technical merits of the position and (2) for those tax positions that meet the more-likely-than-not recognition threshold, management recognizes the largest amount of tax benefit that is more than 50 percent likely to be realized upon ultimate settlement with the related tax authority. The Company provides reserves for potential payments of tax to various tax authorities related to uncertain tax positions. These reserves are based on a determination of whether and how much of a tax benefit taken by the Company in its tax filings or positions is more likely than not to be realized following resolution of any potential contingencies related to the tax benefit. Potential interest related to the underpayment of income taxes will be classified as a component of interest expense and any related penalties will be classified in operating expenses in the statement of operations and comprehensive loss. On March 27, 2020, President Trump signed into law the Coronavirus Aid, Relief and Economic Security ( ) . The , among other things, includes provisions relating to refundable payroll tax credits, deferment of employer social security payments, net operating loss carryback periods, alternative minimum tax credit refunds, and modifications to the net interest deduction limitations. While the Company continues to examine the impacts the may have on its business, it does not expect it will have a material impact to its financial statements. Net Loss per Common Share— Basic net loss per common share is computed by dividing net loss applicable to common stockholders by the weighted-average number of common stock outstanding during the period. Diluted net loss per common share is computed by dividing the net loss applicable to common stockholders by the diluted weighted-average number of common stock outstanding during the period. In periods in which the Company reports a net loss, all common stock equivalents are deemed anti-dilutive such that basic net loss per common share and diluted net loss per common share are equivalent (Note 12). Segment Reporting— Operating segments are identified as components of an enterprise for which separate discrete financial information is available for evaluation by the Company’s Chief Operating Decision Maker in making decisions regarding resource allocation and assessing performance. To date, the Company has viewed its operations and manages its business as one operating segment. Recent Accounting Pronouncements (Adopted) — In August 2018, the FASB issued ASC 2018-13 Fair Value Measurement – Disclosure Framework-Changes to the Disclosure Requirement for Fair Value Measurement, or ASU 2018-13. The amendments in ASU 2018-13 modify the disclosure requirements on fair value measurements in ASC 820, Fair Value Measurement, based on the concepts in the FASB Concepts Statement, including the consideration of costs and benefits. The ASU is effective for fiscal years beginning after December 15, 2019, including interim periods within those years. The Company has adopted this guidance effective January 1, 2020. The adoption of the guidance did not have a material impact on the Company’s financial statements. In August 2018, the FASB issued ASU No. 2018-15, Intangible-Goodwill and Other Internal-Use Software (Subtopic 350-40) The Company has adopted this guidance effective January 1, 2020. The adoption of the guidance did not have a material impact on the Company’s financial statements. |
License Agreements
License Agreements | 6 Months Ended |
Jun. 30, 2020 | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | |
License Agreements | 3. LICENSE AGREEMENTS REGENXBIO Inc. In August 2017, the Company entered into a License Agreement, or the REGENXBIO GBA1 License, with REGENXBIO Inc., or REGENXBIO. Under the terms of the REGENXBIO GBA1 License, REGENXBIO granted the Company an exclusive, worldwide license under certain patents and patent applications to make, have made, use, import, sell and offer for sale products for the treatment of disease, including but not limited to Parkinson’s disease and Gaucher disease, whether or not caused by mutations in the gene that produces the GBA1 enzyme in humans by in vivo gene therapy using AAV9 delivering the gene (or any portion thereof) encoding for GBA1. As consideration for the licensed rights under the REGENXBIO GBA1 License, the Company issued 2,430,000 shares of its common stock with an aggregate fair value of $0.4 million in a concurrent private placement to REGENXBIO. The Company is obligated, pursuant to the REGENXBIO GBA1 License, to pay REGENXBIO: (1) an annual maintenance fee; (2) mid-to high-single digit royalty percentages on net sales of licensed products, subject to reduction in specified circumstances; and (3) mid-teen to low-twenties royalty percentages of any sublicense fees the Company receives from sublicensees for the licensed intellectual property rights. The REGENXBIO GBA1 License will expire on a country-by-country, licensed product-by-licensed product basis upon the later of (1) the expiration, lapse, abandonment or invalidation of the last valid claim of the In May 2018, the Company entered into a license agreement, or the REGENXBIO Option Genes License, with REGENXBIO pursuant to which REGENXBIO granted the Company three distinct exclusive options for specified genes, or the Option Genes, exercisable at the Company’s sole discretion through May 10, 2019. Each option represented the right to obtain an exclusive, worldwide license under certain patents and patent applications to make, have made, use, import, sell and offer for sale products for the treatment or prevention of disease, including but not limited to Parkinson’s disease, whether or not caused by mutations in any Option Gene that is the subject of the applicable license, in humans by in vivo gene therapy using AAV9 delivering the applicable licensed Option Gene and/or RNA interference or antisense modalities that target the applicable licensed Option Gene. The Company also received a non-exclusive, royalty-free, worldwide research license to perform research and development activities for each Option Gene solely for purposes of evaluating whether to exercise the applicable option. In April 2019, the Company exercised all of the options under the REGENXBIO Option Genes License and paid the additional up-front fee of $0.6 million per option for an aggregate of $1.8 million to REGENXBIO. In May 2020, the Company paid annual maintenance fees of approximately $0.1 million per option for an aggregate total of approximately $0.2 million to REGENXBIO. Under the terms of the REGENXBIO Option Genes License, the Company is required to pay REGENXBIO: (1) an annual maintenance fee; (2) mid- to high-single digit royalty percentages on net sales of the licensed product, subject to reduction in specified circumstances; and (3) mid-teen to low-twenties royalty percentages of any sublicense fees the Company receives from sublicensees for the licensed intellectual property rights. If a licensed product includes the GBA1 gene and otherwise would be subject to royalties under the REGENXBIO GBA1 License, then royalties for that licensed product will only be due under the REGENXBIO Option Genes License. The REGENXBIO Option Genes License will expire on a country-by-country, licensed product-by-licensed product basis upon the later of (1) the expiration, lapse, abandonment or invalidation of the last valid claim of the licensed intellectual property and (2) seven years from the first commercial sale of each licensed product. |
Fair Value of Financial Instrum
Fair Value of Financial Instruments | 6 Months Ended |
Jun. 30, 2020 | |
Fair Value Disclosures [Abstract] | |
Fair Value of Financial Instruments | 4. FAIR VALUE OF FINANCIAL INSTRUMENTS The Company’s financial instruments consist of cash equivalents, short and long-term investments, accounts payable and accrued expenses. Fair value estimates of these instruments are made at a specific point in time, based on relevant market information. The fair value of cash equivalents, which consisted of money-market funds, and short and long-term investments were determined using Level 1 inputs reflecting quoted prices in active markets. The carrying amount of accounts payable and accrued expenses as reported on the balance sheets as of June 30, 2020 and December 31, 2019, approximates fair value, due to the short-term duration of these instruments. As of June 30, 2020 and December 31, 2019 there were no financial instruments classified as Level 3 investments. Further, during the three and six months ended June 30, 2020 and 2019 there were no transfers between Level 1, Level 2, and Level 3. |
Investments
Investments | 6 Months Ended |
Jun. 30, 2020 | |
Investments [Abstract] | |
Investments | 5. INVESTMENTS As of June 30, 2020, the fair value of investments by type of security was as follows: Description Amortized Cost Unrealized Gains Unrealized Losses Fair Value (in thousands) Corporate bonds $ 6,458 $ — $ — $ 6,458 U.S. government agency treasuries and securities 13,675 — (1 ) 13,674 Total $ 20,133 $ — $ (1 ) $ 20,132 The estimated fair value and amortized cost of the Company’s available-for-sale securities by contractual maturity are summarized as follows: June 30, 2020 Amortized Cost Fair Value (in thousands) Due in one year or less $ 6,458 $ 6,458 Due between one year and five years 13,675 13,674 Total $ 20,133 $ 20,132 |
Property and Equipment, Net
Property and Equipment, Net | 6 Months Ended |
Jun. 30, 2020 | |
Property Plant And Equipment [Abstract] | |
Property and Equipment, Net | 6. PROPERTY AND EQUIPMENT, NET Property and equipment, net, consisted of the following, as of: June 30, 2020 December 31, 2019 (in thousands) Laboratory equipment $ 2,104 $ 1,730 Leasehold improvements 887 887 Computer equipment 36 36 Furniture and fixtures 219 218 Gross property and equipment 3,246 2,871 Less: Accumulated depreciation (548 ) (322 ) Property and equipment, net $ 2,698 $ 2,549 |
Accrued Expenses and Other Curr
Accrued Expenses and Other Current Liabilities | 6 Months Ended |
Jun. 30, 2020 | |
Accrued Expenses And Other Current Liabilities [Abstract] | |
Accrued Expenses and Other Current Liabilities | 7. ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES Accrued expenses and other current liabilities consisted of the following: June 30, 2020 December 31, 2019 (in thousands) Employee-related $ 2,062 $ 2,577 Clinical 2,700 — Research and development 793 1,595 Professional fees 3,639 634 Other 241 524 Total accrued expenses and other current liabilities $ 9,435 $ 5,330 |
Redeemable Convertible Preferre
Redeemable Convertible Preferred Stock and Common Stock | 6 Months Ended |
Jun. 30, 2020 | |
Temporary Equity Disclosure [Abstract] | |
Redeemable Convertible Preferred Stock and Common Stock | 8. REDEEMABLE CONVERTIBLE PREFERRED STOCK AND COMMON STOCK General In March 2019, the Company authorized the sale and issuance of 3,958,046 shares of Series B Preferred Stock with a par value per share of $0.0001 at a price of $12.63 per share for aggregate net proceeds of $49.8 million. Issuance costs were $0.2 million. In June 2019, the Board of Directors of the Company approved a 1.62-for-one forward stock split of the Company’s outstanding shares of common stock and convertible preferred stock. Accordingly, all share and per share amounts for all periods presented in the accompanying financial statements and notes thereto have been retroactively adjusted, where applicable, to reflect this forward stock split. Shares of common stock underlying outstanding stock options and other equity instruments were proportionately increased and the respective per share value and exercise prices, if applicable, were proportionately decreased in accordance with the terms of the agreements governing such securities. |
Leases
Leases | 6 Months Ended |
Jun. 30, 2020 | |
Leases [Abstract] | |
Leases | 9. LEASES Lease Agreements —The Company leases office and laboratory space in its New York City location under an operating lease agreement entered in September 2017, with an original term of 49 months. In connection with the lease, the Company paid a security deposit of less than $0.1 million in the form of an unconditional and irrevocable letter of credit, which is secured with cash on deposit classified as restricted cash. The original lease was initially modified in October 2018, which extended the original term of the lease and included space on two additional floors. The Company accounted for the new agreement as a modification of the original agreement and recorded an additional right-of-use asset and corresponding lease liability based on the incremental borrowing rate determined as of the effective date of the modified lease. The additional floors were recognized as additional lease components. One of these lease components provided an incentive allowance to reimburse the Company for the cost of qualified leasehold improvement. During the year ended December 31, 2019, the Company incurred qualified costs of $0.4 million which will be recognized over the remaining lease term. The Company was reimbursed for these qualified costs in February 2020. In July 2019, the Company entered into a second modification to further extend the term to 76 months, rent additional space and surrender a portion of the originally leased space. The Company accounted for the new agreement as a modification of the existing agreement with the additional space recognized as a separate lease component. The Company recorded an additional right-of-use asset and a corresponding lease liability calculated based on the incremental borrowing rate determined as of the effective date of the second lease modification. The agreement does not include any options to extend or terminate the lease, and no restrictions or covenants are imposed by the lease agreement. The Company identified and assessed the following significant assumption in recognizing the right-of-use assets and corresponding liabilities: • Incremental borrowing rate— The Company’s lease agreement does not provide a readily determinable implicit rate. As the Company does not have any external borrowings for comparable terms of the lease, the Company estimated the incremental borrowing rate based on the credit quality of the Company and by comparing interest rates available in the market for similar borrowings adjusted for the impact of collateral over the term of the lease. The Company is required to pay for operating costs, including insurance, maintenance, and taxes, which are billed annually based on the Company’s share of the total rentable square footage. These additional charges are considered variable lease cost and are recognized in the period in which the costs are incurred. The components of the lease expense were as follows: Three Months Ended June 30, Six Months Ended June 30, 2020 2019 2020 2019 (in thousands) Operating lease cost $ 569 $ 441 $ 1,138 $ 900 Variable lease cost 196 138 363 277 Total lease cost $ 765 $ 579 $ 1,501 $ 1,177 Weighted-average remaining lease term 5.3 years 5.7 years 5.3 years 5.7 years Weighted-average discount rate 9.50 % 9.00 % 9.50 % 9.00 % Cash paid for amounts included in the measurement of the lease liabilities, net of qualified costs, were $1.1 million for the six months ended June 30, 2020. Cash paid for amounts included in the measurement of the lease liabilities were $0.3 million for the six months ended June 30, 2019, respectively. As of June 30, 2020 and December 31, 2019, the maturities of the Company’s remaining operating lease liabilities were as follows: June 30, 2020 December 31, 2019 (in thousands) 2020 $ 1,159 $ 2,299 2021 2,379 2,379 2022 2,463 2,463 2023 2,549 2,549 2024 2,638 2,638 Thereafter 2,263 2,263 Present value adjustment (2,831 ) (3,322 ) Present value of lease payments $ 10,620 $ 11,269 |
Stock-Based Compensation
Stock-Based Compensation | 6 Months Ended |
Jun. 30, 2020 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Stock-Based Compensation | 10. STOCK-BASED COMPENSATION In August 2017, the Company adopted the Prevail Therapeutics Inc. 2017 Equity Incentive Plan or the 2017 Plan under which the Company granted incentive stock options, nonqualified stock options, stock appreciate rights, or SARs, restricted stock, unrestricted stock, restricted stock awards, performance awards, or other awards that are convertible into or based on Company stock. The maximum number of shares that could have been be issued under the 2017 Plan was 2,511,000 shares as of December 31, 2017. In March 2018, the Company amended the 2017 Plan and increased In June 2019, the Company adopted the Prevail Therapeutics Inc. 2019 Equity Incentive Plan or the 2019 Plan, which superseded the 2017 Equity Incentive Plan, under which the Company may grant incentive stock options, nonqualified stock options, SARs, restricted stock, unrestricted stock, restricted stock awards, performance awards or other awards that are convertible into or based on Company stock. The aggregate number of shares that may be issued pursuant to stock awards under the 2019 Plan were 3,078,831 and 2,756,692 as of June 30, 2020 and December 31, 2019, respectively. The number of shares reserved for issuance under the 2019 Plan will automatically increase on January 1 of each year, through and including January 1, 2029, by 4% of the total number of shares of the Company’s common stock outstanding on December 31st of the preceding calendar year, or a lesser number of shares determined by the Company’s Board of Directors. In June 2019 the Company adopted the 2019 Employee Stock Purchase Plan or the 2019 ESPP, under which the Company may permit employees to purchase shares of common stock. The maximum number of shares that may be issued under the 2019 ESPP was 671,387 shares as of June 30, 2020. The number of shares reserved for issuance under the 2019 ESPP will automatically increase on January 1 of each year, through and including January 1, 2029, by the lesser of (1) 1% of the total number of shares of the Company’s common stock outstanding on December 31 of the preceding calendar year, (2) 1,500,000 shares or (3) such lesser number of shares as determined by the Company’s Board of Directors. No purchases were made pursuant to this plan as of June 30, 2020. The exercise price for a stock option awarded under the 2019 Plan shall not be less than 100% of the fair market value of the Company’s common stock on the date of grant. Typically, options granted under the 2019 Plan vest 25% on the one-year grant date anniversary and monthly thereafter over the following three years and expire ten years from the date of grant. Stock Options The following tables summarize stock option activity under the 2017 Plan and the 2019 Plan: Number of Awards Weighted Average Exercise Price Weighted Average Remaining Contractual Life (Years) Aggregate Intrinsic Value (in thousands except share data) Outstanding, December 31, 2019 5,681,422 $ 3.60 8.6 $ 69,219 Granted 1,132,600 16.82 Exercised (76,101 ) 1.42 Cancelled/Forfeited (89,189 ) 3.12 Outstanding, June 30, 2020 6,648,732 $ 5.91 8.4 $ 62,035 Exercisable, June 30, 2020 2,459,316 2.46 8.0 30,595 Vested and expected to vest, June 30, 2020 6,648,732 5.91 8.4 62,035 As of June 30, 2020, the total unrecognized compensation expense related to unvested employee and non-employee options was $24.6 million, which the Company expects to recognize over an estimated weighted-average period of 2.9 years. The Company uses the Black-Scholes option pricing model to estimate the fair value of stock options on the date of grant. The Company determined the assumptions for the Black-Scholes option-pricing valuation model as discussed below. Each of these inputs is subjective and generally requires significant judgment to determine. The weighted average fair value and assumptions used to determine the fair value of stock options granted was as follows: Three Months Ended June 30, Six Months Ended June 30, 2020 2019 2020 2019 Grant date fair value $ 11.95 $ 7.26 $ 12.11 $ 6.18 Expected term 5.8 6.1 6.0 6.1 Risk-free interest rate 0.4 % 2.3 % 1.2 % 2.4 % Expected volatility 87.1 % 79.2 % 86.1 % 79.1 % Dividend rate — — — — Expected Term — The expected term represents the period that the stock-based awards are expected to be outstanding. As the Company does not have sufficient historical experience for determining the expected term of the stock option awards granted, the Company based its expected term for awards issued to employees and non-employees using the simplified method, which is presumed to be the midpoint between the vesting date and the end of the contractual term. Risk-Free Interest Rate — The risk-free interest rate is based on the U.S. Treasury yield curve in effect at the date of grant for zero-coupon U.S. Treasury constant maturity notes with terms approximately equal to the stock-based awards’ expected term. Expected Volatility — Since the Company does not have a trading history of common stock, the expected volatility was derived from the average historical stock volatilities of the common stock of several public companies within the industry that the Company considers to be comparable to its business over a period equivalent to the expected term of the stock-based awards. Dividend Rate — The expected dividend rate is zero as the Company has not paid and does not anticipate paying any dividends in the foreseeable future. Fair Value of Common Stock — Prior to the IPO, the fair value of the shares of common stock underlying the stock-based awards were determined by the Company’s Board of Directors with input from management. Since there was no public market for the common stock prior to June 20, 2019, the Company’s Board of Directors had determined the fair value of the common stock at the time of grant of the stock-based award by considering a number of objective and subjective factors, including having valuations of the common stock performed by a third-party valuation specialist. The fair value of the common stock is now determined by the public market. Restricted Stock As of June 30, 2020, and December 31, 2019, 2,349,000 shares of common stock are subject to a repurchase right by the Company. Non-vested Restricted Stock Award (RSA) Outstanding The following table presents a summary of the Company’s non-vested restricted stock award activity under all plans and related information for the six months ended June 30, 2020: Number of Restricted Stock Awards Outstanding Weighted Average Grant Date Fair Value Per Share Non-vested restricted stock awards outstanding as of December 31, 2019 978,759 $ 0.18 Restricted stock awards vested (293,622 ) 0.18 Non-vested restricted stock awards outstanding as of June 30, 2020 685,137 0.18 Three Months Ended June 30, Six Months Ended June 30, 2020 2019 2020 2019 (in thousands) Aggregate grant date fair value of restricted stock awards vested $ 25 $ 25 $ 51 $ 51 Restricted stock awards are generally granted at the fair market value of the Company’s common stock on the date of grant and vest 25% after the first year and monthly thereafter over the following three years. Forfeitures are based on actual forfeitures in the given period. There were $0.1 million of total unrecognized compensation cost related to non-vested restricted stock awards granted under the Company’s equity incentive plans as of June 30, 2020. This cost is expected to be recognized over a weighted-average period of 1.1 years. Total stock-based compensation expense is recognized for restricted stock and stock options granted to employees and non-employees and has been reported in the Company’s statements of operations and comprehensive loss as follows: Three Months Ended June 30, Six Months Ended June 30, 2020 2019 2020 2019 (in thousands) Research and development $ 1,112 $ 553 $ 1,935 $ 1,065 General and administrative 1,137 556 1,947 656 Total stock-based compensation expense $ 2,249 $ 1,109 $ 3,881 $ 1,721 Convertible Preferred Stock Immediately prior to the closing of the Company’s IPO, 19,435,131 shares of outstanding convertible preferred stock converted into 19,435,131 shares of common stock. Preferred Stock The Company’s amended and restated certificate of incorporation, which became effective upon the completion of the IPO, authorizes 10,000,000 shares of preferred stock, of which no shares were issued or outstanding as of June 30, 2020. |
Related Party Transactions
Related Party Transactions | 6 Months Ended |
Jun. 30, 2020 | |
Related Party Transactions [Abstract] | |
Related Party Transactions | 11. RELATED PARTY TRANSACTIONS Since the Company’s inception in July 2017, the Company has engaged in transactions with related parties, which included OrbiMed Private Investments VI, LP In August 2017, OrbiMed Private Investments VI, LP In August 2017, the Company entered into a Patent License Agreement and Stock Purchase Agreement with REGENXBIO. See Note 3. In April 2019, the Company exercised all of the options under the REGENXBIO Option Gene License and paid the additional up-front fee of $0.6 million per option, or an aggregate of $1.8 million, to REGENXBIO which was recorded as research and development expense for the three and six month periods ended June 30, 2019. Additionally, in May 2020 in accordance with the REGENXBIO GBA1 License, the Company paid an annual maintenance fee of approximately $0.2 million, which was also recorded as research and development expense, for the three and six month periods ended June 30, 2019. |
Net Loss Per Share
Net Loss Per Share | 6 Months Ended |
Jun. 30, 2020 | |
Earnings Per Share [Abstract] | |
Net Loss Per Share | 12. NET LOSS PER SHARE The following outstanding potentially dilutive common stock equivalents have been excluded from the calculation of diluted net loss per share for the periods presented due to their anti-dilutive effect: June 30, 2020 2019 Common stock options issued and outstanding 6,648,732 5,782,090 Restricted stock subject to future vesting 685,137 1,272,381 Total 7,333,869 7,054,471 Three Months Ended June 30, Six Months Ended June 30, 2020 2019 2020 2019 (in thousands except share data) Net loss $ (22,100 ) $ (15,103 ) $ (40,675 ) $ (25,048 ) Weighted-average number of shares-basic and diluted 33,467,346 26,212,356 33,367,344 23,945,198 Net loss per share—basic and diluted (0.66 ) (0.58 ) (1.22 ) (1.05 ) |
Commitments and Contingencies
Commitments and Contingencies | 6 Months Ended |
Jun. 30, 2020 | |
Commitments And Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 13. COMMITMENTS AND CONTINGENCIES Contingencies —From time to time, the Company may be involved in disputes or regulatory inquiries that arise in the ordinary course of business. When the Company determines that a loss is both probable and reasonably estimable, a liability is recorded and disclosed if the amount is material to the financial statements taken as a whole. When a material loss contingency is only reasonably possible, the Company does not record a liability, but instead discloses the nature and the amount of the claim, and an estimate of the loss or range of loss, if such an estimate can reasonably be made. The Company did not have accruals established for any litigation liabilities as of June 30, 2020 and December 31, 2019. In June 2019, the Company received a letter on behalf of Alector, Inc., or Alector, a biopharmaceutical company employing antibodies for the treatment of neurodegeneration, stating concerns regarding whether confidential information of Alector was used in connection with work on behalf of the company and patents and patent applications filed on behalf of the Company, as well as alleging that Alector has certain rights to the Company’s patents and patent applications. Following the Company’s response to Alector’s claims, Alector served Dr. Abeliovich with a demand for arbitration in which it made similar allegations to those stated in its letter to the Company. In the demand, Alector sought relief from Dr. Abeliovich in the form of monetary damages, equitable relief in the form of an injunction against Dr. Abeliovich’s activities that may involve Alector’s confidential information and an assignment to Alector of rights to certain of the Company’s patents and patent applications. The arbitration hearings were completed on July 15, 2020, and the Company expects a decision from the arbitrator at the end of the third quarter or in the fourth quarter of 2020. The Company is indemnifying Dr. Abeliovich in connection with the arbitration pursuant to his indemnification agreement with the Company. The Company does not deem a potential loss probable or estimable as of June 30, 2020 and has not recorded an accrual. In addition, given the uncertainty inherent in litigation and arbitration, we do not believe it is possible to develop an estimate of reasonably possible loss for this matter. We do not expect the outcome of this matter to have a material adverse effect on our business, operating results or financial condition . However, potential liabilities are subject to change due to new developments, changes in settlement strategy or the impact of evidentiary requirements, which could cause us to pay damage awards or settlements (or become subject to equitable remedies) that could have a material adverse effect on our business, operating results or financial condition in the periods recognized or paid. The Company is also party to various agreements, principally relating to licensed technology, that require future payments relating to milestones, or royalties on future sales of specified products. No material milestone or royalty payments under these agreements are expected to be payable in the immediate future. See Note 3 for further details of these agreements. |
Subsequent Event
Subsequent Event | 6 Months Ended |
Jun. 30, 2020 | |
Subsequent Events [Abstract] | |
Subsequent Event | 14. SUBSEQUENT EVENT In July 2020, the Company received $3.0 million from an insurance claim as partial coverage for legal expenses incurred from the ongoing arbitration matter disclosed in Note 13. On August 11, 2020, the Company entered into an Open Market Sale Agreement SM |
Significant Accounting Polici_2
Significant Accounting Policies (Policies) | 6 Months Ended |
Jun. 30, 2020 | |
Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation— The Company’s unaudited interim financial statements have been prepared by the Company in accordance with accounting principles generally accepted in the United States, or U.S. GAAP, for interim information and pursuant to the rules and regulations of the U.S. Securities and Exchange Commission, or the SEC, for reporting on Form 10-Q. Accordingly, certain information and footnote disclosures normally included in financial statements prepared in accordance with U.S. GAAP have been omitted pursuant to such rules and regulations. These unaudited interim financial statements should be read in conjunction with the audited financial statements as of and for the year ended December 31, 2019, and the notes thereto, which are included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2019. The COVID-19 pandemic has interrupted business operations across the globe. Estimates and assumptions about future events and their effects cannot be determined with certainty and therefore require the exercise of judgment. As of the date of issuance of these financial statements, the Company is not aware of any specific event or circumstance that would require the Company to update its estimates, assumptions and judgments or revise the reported amounts of assets and liabilities or the disclosure of contingent assets and liabilities. These estimates, however, may change as new events occur and additional information is obtained, and are recognized in the financial statements as soon as they become known. |
Use of Estimates | Use of Estimates —The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the financial statements, and the reported amounts of expenses during the reporting period. Significant estimates in the financial statements include, but are not limited to stock-based compensation, operating lease right-of-use assets and liabilities, the recoverability of the Company’s net deferred tax assets and related valuation allowance, and accrued expenses related to expenses incurred for research and development from external vendors. The Company bases its estimates on historical experience, known trends and other market-specific or other relevant factors that it believes to be reasonable under the circumstances. The Company tracks the progress of its various research and development studies and manufacturing projects to ensure related prepaid expenses and accrued expenses are in line with progress of each. On an ongoing basis, management evaluates its estimates, as there are changes in circumstances, facts and experience. Actual results may differ materially from those estimates or assumptions. |
Cash, Cash Equivalents and Restricted Cash | Cash, Cash Equivalents and Restricted Cash —The Company’s cash and cash equivalents include short-term highly liquid investments which are readily convertible into cash. These investments include money market securities and commercial paper with maturities of three months or less when acquired. The Company’s institutional money market accounts permit daily redemption and the fair values of these investments are based upon the quoted prices in active markets provided by the holding financial institutions, which are considered Level 1 inputs in the fair value hierarchy, as described below. The Company had cash and cash equivalents of $111.1 million as of June 30, 2020. Restricted cash represents cash on deposit with a financial institution as collateral in support of a letter of credit outstanding in favor of the Company’s landlord for office space. The restricted cash balance has been excluded from the cash balance and is classified as non-current restricted cash on the balance sheets as the lease expires after June 30, 2021. |
Investments | Investments — The Company classifies investments in debt securities with remaining maturities when purchased of greater than three months as available-for-sale. Investments with a remaining maturity date greater than one year are classified as non-current. Available-for-sale securities are maintained by investment managers and consist of corporate bonds and U.S. government agency securities. Available-for-sale securities are carried at fair value with the unrealized gains and losses included in other comprehensive loss as a component of stockholders’ equity until realized. The Company had $20.1 million in total available-for-sale debt securities as of June 30, 2020. Any premium or discount arising at purchase is amortized and/or accreted to interest income and/or expensed over the life of the instrument. If any adjustment to fair value reflects a decline in the value of the investment, the Company considers all available evidence to evaluate the extent to which the decline is “other-than-temporary” and, if so, marks the investment to market through a change to the Company’s statement of operations and comprehensive loss. The Company does not intend to sell the investments and it is not more likely than not that the Company will be required to sell the investments before recovery of their amortized costs bases, which may be maturity. Realized gains and losses are included in the statement of operations and comprehensive loss in interest income, net and on a specific-identification basis. There were no realized gains or losses on investments for the three and six months ended June 30, 2020. There were no investments that had been in an unrealized loss position for more than 12 months as of June 30, 2020 |
Concentration of Credit Risk | Concentration of Credit Risk —The Company maintains cash deposits in excess of government-provided insurance limits. The Company maintains its cash balances with two accredited financial institutions, and accordingly, such funds are not exposed to significant credit risk. |
Leases | Leases —The Company determines if an arrangement is a lease at inception. Operating leases are included in operating lease right-of-use, or ROU, assets, operating lease liabilities, and long-term operating lease liabilities in the Company’s balance sheets. ROU assets represent the Company’s right to use an underlying asset for the lease term and lease liabilities represent the Company’s obligation to make lease payments arising from the lease. Operating lease ROU assets and liabilities are recognized at commencement date based on the present value of lease payments over the lease term. As the Company’s leases do not provide a readily determinable implicit rate, the Company’s uses its incremental borrowing rate based on the information available at commencement date in determining the present value of lease payments. The operating lease ROU asset also includes any lease prepayments, offset by lease incentives. The Company’s facilities operating leases have lease and non-lease components for which the Company has elected to apply the practical expedient and account for each lease component and related non-lease component as one single component. Operating lease cost is recognized on a straight-line basis over the lease term. |
Property and Equipment, Net | Property and Equipment, Net —Property and equipment are stated at cost less accumulated depreciation. Depreciation expense is recognized using the straight-line method over the estimated useful life of the asset, ranging from 3-7 years as follows: Fixed Asset Type Estimated useful life Laboratory Equipment 7 Years Leasehold Improvements Lesser of useful life or remaining lease term Computer Equipment 3 Years Furniture and Fixtures 7 Years Expenditures for repairs and maintenance of assets are charged to expense as incurred, while major betterments are capitalized. Upon retirement or sale, the cost and related accumulated depreciation of assets disposed of are removed from the accounts and any resulting gain or loss is included in the statements of operations and comprehensive loss. |
Impairment of Long-Lived Assets | Impairment of Long-Lived Assets —Long-lived assets, comprised of property and equipment, to be held and used and the right-of-use asset associated with the Company’s leased office space are tested for recoverability whenever events or changes in business circumstances indicate that the carrying amount of the assets may not be fully recoverable. Factors that the Company considers in deciding when to perform an impairment review include significant underperformance of the business in relation to expectations, significant negative industry or economic trends and significant changes or planned changes in the use of the assets. If an impairment review is performed to evaluate a long-lived asset for recoverability, the Company compares forecasts of undiscounted cash flows expected to result from the use and eventual disposition of the long-lived asset to its carrying value. An impairment loss would be recognized when estimated undiscounted future cash flows expected to result from the use of an asset are less than its carrying amount. The impairment loss would be based on the excess of the carrying value of the impaired asset over its current fair value. To date, the Company has not recorded any impairment losses on long-lived assets. |
Other Long-Term Assets | Other Long-Term Assets – At June 30, 2020 the Company had other long-term assets that primarily consisted of advanced payments made to the contract research organization responsible for conducting the Company’s PROPEL, PROVIDE and PROCLAIM clinical trials. |
Comprehensive Loss | Comprehensive Loss — During the three and six months ended June 30, 2020, the Company recorded unrealized gains and losses associated with its available-for sale debt securities in other comprehensive loss. |
Fair Value Measurements | Fair Value Measurements —Certain assets and liabilities are carried at fair value under U.S. GAAP. Fair value is defined as the exchange price that would be received for an asset or paid to transfer a liability (an exit price) in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants on the measurement date. Valuation techniques used to measure fair value must maximize the use of observable inputs and minimize the use of unobservable inputs. A framework is used for measuring fair value utilizing a three-tier hierarchy that prioritizes the inputs to valuation techniques used to measure fair value. Financial assets and liabilities carried at fair value are to be classified and disclosed in one of the following three levels of the fair value hierarchy, of which the first two are considered observable and the last is considered unobservable: • Level 1 —Unadjusted quoted prices in active markets for identical assets or liabilities. • Level 2 —Observable inputs (other than Level 1 quoted prices), such as quoted prices in active markets for similar assets or liabilities, quoted prices in markets that are not active for identical or similar assets or liabilities, or other inputs that are observable or can be corroborated by observable market data. • Level 3 —Unobservable inputs that are supported by little or no market activity and that are significant to determining the fair value of the assets or liabilities, including pricing models, discounted cash flow methodologies and similar techniques. The following table summarizes assets measured at fair value on a recurring basis at June 30, 2020 and December 31, 2019 (and does not include cash of $0.2 million and $0.2 million, respectively): Active Markets (Level 1) Observable Inputs (Level 2) Unobservable Inputs (Level 3) June 30, 2020 (in thousands) Cash and cash equivalents: Money Market Funds $ 110,846 $ — $ — Investments: Corporate bonds 6,458 — — U.S. treasury securities 13,674 — — Restricted cash: Money Market Funds 91 — — Total $ 131,069 $ — $ — Active Markets (Level 1) Observable Inputs (Level 2) Unobservable Inputs (Level 3) December 31, 2019 (in thousands) Cash and cash equivalents: Money Market Funds $ 167,805 $ — $ — Restricted cash: Money Market Funds 91 — — Total $ 167,896 $ — $ — There have been no changes to the valuation methods utilized by the Company, nor were there transfers between Level 1, Level 2 and Level 3 investments during the three and six months ended June 30, 2020. there were no financial instruments classified as Level 3 investments. |
Research and Development Costs | Research and Development Costs —Research and development costs are expensed as incurred. Research and development expenses consist principally of personnel costs, including salaries, stock-based compensation, and benefits for research and development employees, costs related to third-party contract research organizations, contract development and manufacturing organizations, other third-party research service providers, third-party license fees, other direct and indirect operational costs related to the Company’s research and development activities, including facility-related expenses. Non-refundable research and development advance payments are capitalized and expensed as the related goods are delivered or services are performed. |
Stock-Based Compensation | Stock-Based Compensation —The Company measures all stock options and other stock-based awards granted to employees, directors, consultants and other nonemployees based on the fair value on the date of the grant and recognizes compensation expense of those awards over the requisite service period, which is generally the vesting period of the respective award. The Company recognizes forfeitures at the time forfeitures occur. The Company classifies stock-based compensation expense in its statement of operations and comprehensive loss in the same way the payroll costs or service payments are classified for the related stock-based award recipient. The fair value of each stock option grant is estimated on the date of grant using the Black-Scholes option pricing model. The Company lacks company specific historical and implied volatility information. Therefore, it estimates its expected stock volatility based on the historical volatility of a publicly-traded set of peer companies and expects to continue to do so until it has adequate historical data regarding the volatility of its own traded stock price. |
Income Taxes | Income Taxes —The Company accounts for income taxes using the asset and liability method, which requires the recognition of deferred tax assets and liabilities for the expected future tax consequences of events that have been recognized in the financial statements or the Company’s tax returns. Under this method, deferred tax assets and liabilities are determined on the basis of the differences between the financial statements and tax basis of the assets and liabilities using enacted tax rates in effect for the year in which the differences are expected to reverse. The effect of a change in tax rates on deferred tax assets and liabilities is recognized in income in the period that includes the enactment date. Changes in deferred tax assets and liabilities are recorded in the provision for income taxes. The Company recognizes deferred tax assets to the extent that the Company believes that these assets are more likely than not to be realized. In making such a determination, management considers all available positive and negative evidence, including future reversals of existing taxable temporary differences, projected future taxable income, tax-planning strategies, and results of recent operations. Valuation allowances are provided, if based upon the weight of available evidence, it is more likely than not that some or all of the deferred tax assets will not be realized. If management determines that the Company would be able to realize its deferred tax assets in the future in excess of their net recorded amount, management would make an adjustment to the deferred tax asset valuation allowance, which would reduce the provision for income taxes. The Company records uncertain tax positions in accordance with ASC 740 on the basis of a two-step process in which (1) management determines whether it is more likely than not that the tax positions will be sustained on the basis of the technical merits of the position and (2) for those tax positions that meet the more-likely-than-not recognition threshold, management recognizes the largest amount of tax benefit that is more than 50 percent likely to be realized upon ultimate settlement with the related tax authority. The Company provides reserves for potential payments of tax to various tax authorities related to uncertain tax positions. These reserves are based on a determination of whether and how much of a tax benefit taken by the Company in its tax filings or positions is more likely than not to be realized following resolution of any potential contingencies related to the tax benefit. Potential interest related to the underpayment of income taxes will be classified as a component of interest expense and any related penalties will be classified in operating expenses in the statement of operations and comprehensive loss. On March 27, 2020, President Trump signed into law the Coronavirus Aid, Relief and Economic Security ( ) . The , among other things, includes provisions relating to refundable payroll tax credits, deferment of employer social security payments, net operating loss carryback periods, alternative minimum tax credit refunds, and modifications to the net interest deduction limitations. While the Company continues to examine the impacts the may have on its business, it does not expect it will have a material impact to its financial statements. |
Net Loss per Common Share | Net Loss per Common Share— Basic net loss per common share is computed by dividing net loss applicable to common stockholders by the weighted-average number of common stock outstanding during the period. Diluted net loss per common share is computed by dividing the net loss applicable to common stockholders by the diluted weighted-average number of common stock outstanding during the period. In periods in which the Company reports a net loss, all common stock equivalents are deemed anti-dilutive such that basic net loss per common share and diluted net loss per common share are equivalent (Note 12). |
Segment Reporting | Segment Reporting— Operating segments are identified as components of an enterprise for which separate discrete financial information is available for evaluation by the Company’s Chief Operating Decision Maker in making decisions regarding resource allocation and assessing performance. To date, the Company has viewed its operations and manages its business as one operating segment. |
Recently Issued Accounting Pronouncements (Adopted) | Recent Accounting Pronouncements (Adopted) — In August 2018, the FASB issued ASC 2018-13 Fair Value Measurement – Disclosure Framework-Changes to the Disclosure Requirement for Fair Value Measurement, or ASU 2018-13. The amendments in ASU 2018-13 modify the disclosure requirements on fair value measurements in ASC 820, Fair Value Measurement, based on the concepts in the FASB Concepts Statement, including the consideration of costs and benefits. The ASU is effective for fiscal years beginning after December 15, 2019, including interim periods within those years. The Company has adopted this guidance effective January 1, 2020. The adoption of the guidance did not have a material impact on the Company’s financial statements. In August 2018, the FASB issued ASU No. 2018-15, Intangible-Goodwill and Other Internal-Use Software (Subtopic 350-40) The Company has adopted this guidance effective January 1, 2020. The adoption of the guidance did not have a material impact on the Company’s financial statements. |
Significant Accounting Polici_3
Significant Accounting Policies (Tables) | 6 Months Ended |
Jun. 30, 2020 | |
Accounting Policies [Abstract] | |
Schedule of Estimated Useful Lives of Property and Equipment | Depreciation expense is recognized using the straight-line method over the estimated useful life of the asset, ranging from 3-7 years as follows: Fixed Asset Type Estimated useful life Laboratory Equipment 7 Years Leasehold Improvements Lesser of useful life or remaining lease term Computer Equipment 3 Years Furniture and Fixtures 7 Years |
Schedule of Fair Value, Assets and Liabilities Measured on Recurring Basis | The following table summarizes assets measured at fair value on a recurring basis at June 30, 2020 and December 31, 2019 (and does not include cash of $0.2 million and $0.2 million, respectively): Active Markets (Level 1) Observable Inputs (Level 2) Unobservable Inputs (Level 3) June 30, 2020 (in thousands) Cash and cash equivalents: Money Market Funds $ 110,846 $ — $ — Investments: Corporate bonds 6,458 — — U.S. treasury securities 13,674 — — Restricted cash: Money Market Funds 91 — — Total $ 131,069 $ — $ — Active Markets (Level 1) Observable Inputs (Level 2) Unobservable Inputs (Level 3) December 31, 2019 (in thousands) Cash and cash equivalents: Money Market Funds $ 167,805 $ — $ — Restricted cash: Money Market Funds 91 — — Total $ 167,896 $ — $ — |
Investments (Table)
Investments (Table) | 6 Months Ended |
Jun. 30, 2020 | |
Investments [Abstract] | |
Schedule of Investments | As of June 30, 2020, the fair value of investments by type of security was as follows: Description Amortized Cost Unrealized Gains Unrealized Losses Fair Value (in thousands) Corporate bonds $ 6,458 $ — $ — $ 6,458 U.S. government agency treasuries and securities 13,675 — (1 ) 13,674 Total $ 20,133 $ — $ (1 ) $ 20,132 |
Schedule of Contractual Maturities of Debt Securities | The estimated fair value and amortized cost of the Company’s available-for-sale securities by contractual maturity are summarized as follows: June 30, 2020 Amortized Cost Fair Value (in thousands) Due in one year or less $ 6,458 $ 6,458 Due between one year and five years 13,675 13,674 Total $ 20,133 $ 20,132 |
Property and Equipment, Net (Ta
Property and Equipment, Net (Tables) | 6 Months Ended |
Jun. 30, 2020 | |
Property Plant And Equipment [Abstract] | |
Summary of Property and Equipment, Net | Property and equipment, net, consisted of the following, as of: June 30, 2020 December 31, 2019 (in thousands) Laboratory equipment $ 2,104 $ 1,730 Leasehold improvements 887 887 Computer equipment 36 36 Furniture and fixtures 219 218 Gross property and equipment 3,246 2,871 Less: Accumulated depreciation (548 ) (322 ) Property and equipment, net $ 2,698 $ 2,549 |
Accrued Expenses and Other Cu_2
Accrued Expenses and Other Current Liabilities (Tables) | 6 Months Ended |
Jun. 30, 2020 | |
Accrued Expenses And Other Current Liabilities [Abstract] | |
Schedule of Accrued Expenses and Other Current Liabilities | Accrued expenses and other current liabilities consisted of the following: June 30, 2020 December 31, 2019 (in thousands) Employee-related $ 2,062 $ 2,577 Clinical 2,700 — Research and development 793 1,595 Professional fees 3,639 634 Other 241 524 Total accrued expenses and other current liabilities $ 9,435 $ 5,330 |
Leases (Tables)
Leases (Tables) | 6 Months Ended |
Jun. 30, 2020 | |
Leases [Abstract] | |
Summary of Components of Operating Lease Expense | The components of the lease expense were as follows: Three Months Ended June 30, Six Months Ended June 30, 2020 2019 2020 2019 (in thousands) Operating lease cost $ 569 $ 441 $ 1,138 $ 900 Variable lease cost 196 138 363 277 Total lease cost $ 765 $ 579 $ 1,501 $ 1,177 Weighted-average remaining lease term 5.3 years 5.7 years 5.3 years 5.7 years Weighted-average discount rate 9.50 % 9.00 % 9.50 % 9.00 % |
Summary of Maturities of Remaining Operating Lease Liabilities | As of June 30, 2020 and December 31, 2019, the maturities of the Company’s remaining operating lease liabilities were as follows: June 30, 2020 December 31, 2019 (in thousands) 2020 $ 1,159 $ 2,299 2021 2,379 2,379 2022 2,463 2,463 2023 2,549 2,549 2024 2,638 2,638 Thereafter 2,263 2,263 Present value adjustment (2,831 ) (3,322 ) Present value of lease payments $ 10,620 $ 11,269 |
Stock-Based Compensation (Table
Stock-Based Compensation (Tables) | 6 Months Ended |
Jun. 30, 2020 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Summary of Stock Option Activity | The following tables summarize stock option activity under the 2017 Plan and the 2019 Plan: Number of Awards Weighted Average Exercise Price Weighted Average Remaining Contractual Life (Years) Aggregate Intrinsic Value (in thousands except share data) Outstanding, December 31, 2019 5,681,422 $ 3.60 8.6 $ 69,219 Granted 1,132,600 16.82 Exercised (76,101 ) 1.42 Cancelled/Forfeited (89,189 ) 3.12 Outstanding, June 30, 2020 6,648,732 $ 5.91 8.4 $ 62,035 Exercisable, June 30, 2020 2,459,316 2.46 8.0 30,595 Vested and expected to vest, June 30, 2020 6,648,732 5.91 8.4 62,035 |
Schedule of Assumptions to Estimate Fair Value of Stock Options Using Black-Scholes Option Pricing Model | The weighted average fair value and assumptions used to determine the fair value of stock options granted was as follows: Three Months Ended June 30, Six Months Ended June 30, 2020 2019 2020 2019 Grant date fair value $ 11.95 $ 7.26 $ 12.11 $ 6.18 Expected term 5.8 6.1 6.0 6.1 Risk-free interest rate 0.4 % 2.3 % 1.2 % 2.4 % Expected volatility 87.1 % 79.2 % 86.1 % 79.1 % Dividend rate — — — — |
Summary of Non-vested Restricted Stock Award Activity | The following table presents a summary of the Company’s non-vested restricted stock award activity under all plans and related information for the six months ended June 30, 2020: Number of Restricted Stock Awards Outstanding Weighted Average Grant Date Fair Value Per Share Non-vested restricted stock awards outstanding as of December 31, 2019 978,759 $ 0.18 Restricted stock awards vested (293,622 ) 0.18 Non-vested restricted stock awards outstanding as of June 30, 2020 685,137 0.18 Three Months Ended June 30, Six Months Ended June 30, 2020 2019 2020 2019 (in thousands) Aggregate grant date fair value of restricted stock awards vested $ 25 $ 25 $ 51 $ 51 |
Summary of Stock-Based Compensation Expense | Total stock-based compensation expense is recognized for restricted stock and stock options granted to employees and non-employees and has been reported in the Company’s statements of operations and comprehensive loss as follows: Three Months Ended June 30, Six Months Ended June 30, 2020 2019 2020 2019 (in thousands) Research and development $ 1,112 $ 553 $ 1,935 $ 1,065 General and administrative 1,137 556 1,947 656 Total stock-based compensation expense $ 2,249 $ 1,109 $ 3,881 $ 1,721 |
Net Loss Per Share (Tables)
Net Loss Per Share (Tables) | 6 Months Ended |
Jun. 30, 2020 | |
Earnings Per Share [Abstract] | |
Schedule of Potentially Dilutive Common Stock Equivalents Excluded from Calculation of Diluted Net Loss Per Share | The following outstanding potentially dilutive common stock equivalents have been excluded from the calculation of diluted net loss per share for the periods presented due to their anti-dilutive effect: June 30, 2020 2019 Common stock options issued and outstanding 6,648,732 5,782,090 Restricted stock subject to future vesting 685,137 1,272,381 Total 7,333,869 7,054,471 |
Schedule of Basic and Diluted Net Loss Per Share | Three Months Ended June 30, Six Months Ended June 30, 2020 2019 2020 2019 (in thousands except share data) Net loss $ (22,100 ) $ (15,103 ) $ (40,675 ) $ (25,048 ) Weighted-average number of shares-basic and diluted 33,467,346 26,212,356 33,367,344 23,945,198 Net loss per share—basic and diluted (0.66 ) (0.58 ) (1.22 ) (1.05 ) |
Nature of the Business - Additi
Nature of the Business - Additional Information (Details) | 1 Months Ended | 3 Months Ended | 6 Months Ended | |||
Jun. 30, 2019USD ($)$ / sharesshares | Mar. 31, 2019USD ($)$ / shares | Jun. 30, 2019USD ($)$ / sharesshares | Jun. 30, 2020USD ($)shares | Jun. 30, 2019USD ($)$ / sharesshares | Dec. 31, 2019USD ($)shares | |
Nature Of Business [Line Items] | ||||||
Entity incorporation date | Jul. 6, 2017 | |||||
Description of stock split | 1.62-for-one forward stock-split | |||||
Stock split ratio | 1.62 | |||||
Underwriting discounts, commissions and estimated offering expenses | $ 3,016,000 | |||||
Common stock, shares authorized | shares | 200,000,000 | 200,000,000 | ||||
Preferred stock, shares authorized | shares | 10,000,000 | 10,000,000 | ||||
Cash and cash equivalents | $ 202,095,000 | $ 202,095,000 | $ 111,065,000 | 202,095,000 | $ 168,051,000 | |
Proceeds from issuance of common stock in initial public offering | $ 116,251,000 | |||||
Series B Preferred Stock | ||||||
Nature Of Business [Line Items] | ||||||
Shares issued, price per share | $ / shares | $ 12.63 | |||||
Underwriting discounts, commissions and estimated offering expenses | $ 200,000 | |||||
Net proceeds from issuance of convertible preferred stock | $ 49,800,000 | |||||
Available for sale | ||||||
Nature Of Business [Line Items] | ||||||
Investment, Available for sale | $ 20,100 | |||||
Common Stock | ||||||
Nature Of Business [Line Items] | ||||||
Stock issued during period, shares, new issues | shares | 7,353,000 | 7,353,000 | ||||
Initial Public Offering | ||||||
Nature Of Business [Line Items] | ||||||
Common stock, shares authorized | shares | 200,000,000 | 200,000,000 | 200,000,000 | |||
Preferred stock, shares authorized | shares | 10,000,000 | 10,000,000 | 10,000,000 | |||
Proceeds from issuance of common stock in initial public offering | $ 113,000,000 | |||||
Initial Public Offering | Common Stock | ||||||
Nature Of Business [Line Items] | ||||||
Stock issued during period, shares, new issues | shares | 7,353,000 | |||||
Shares issued, price per share | $ / shares | $ 17 | $ 17 | $ 17 | |||
Aggregate net proceeds received from offering | $ 113,000,000 | |||||
Underwriting discounts, commissions and estimated offering expenses | $ 12,000,000 | |||||
Redeemable convertible preferred stock converted into share | shares | 19,435,131 |
Significant Accounting Polici_4
Significant Accounting Policies - Additional Information (Details) | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2020USD ($) | Jun. 30, 2020USD ($)Segment | Dec. 31, 2019USD ($) | Jun. 30, 2019USD ($) | |
Significant Accounting Policies [Line Items] | ||||
Cash and cash equivalents | $ 111,065,000 | $ 111,065,000 | $ 168,051,000 | $ 202,095,000 |
Debt securities, available for sale | 20,100,000 | 20,100,000 | ||
Realized investments gains (losses) | 0 | 0 | ||
Net unrealized loss on investments | 0 | 0 | ||
Fair value of cash | 200,000 | 200,000 | 200,000 | |
Transfers of assets between Level 1 and Level 2 | 0 | 0 | 0 | 0 |
Transfers of assets between Level 2 and Level 1 | 0 | 0 | 0 | 0 |
Transfers of liabilities between Level 1 and Level 2 | 0 | 0 | 0 | 0 |
Transfers of liabilities between Level 2 and Level 1 | 0 | 0 | 0 | 0 |
Transfers of assets between Level 1 and Level 3 | 0 | 0 | 0 | 0 |
Transfers of assets between Level 3 and Level 1 | 0 | 0 | 0 | 0 |
Transfers of assets between Level 2 and Level 3 | 0 | 0 | 0 | 0 |
Transfers of assets between Level 3 and Level 2 | 0 | 0 | 0 | 0 |
Transfers of liabilities between Level 1 and Level 3 | 0 | 0 | 0 | 0 |
Transfers of liabilities between Level 3 and Level 1 | 0 | 0 | 0 | 0 |
Transfers of liabilities between Level 2 and Level 3 | 0 | 0 | 0 | 0 |
Transfers of liabilities between Level 3 and Level 2 | 0 | $ 0 | 0 | $ 0 |
Number of operating segment | Segment | 1 | |||
Fair Value, Inputs, Level 3 | ||||
Significant Accounting Policies [Line Items] | ||||
Financial instruments, owned, at fair value | $ 0 | $ 0 | $ 0 | |
Minimum | ||||
Significant Accounting Policies [Line Items] | ||||
Property and equipment useful life | 3 years | |||
Largest amount of tax benefit | 50.00% | |||
Maximum | ||||
Significant Accounting Policies [Line Items] | ||||
Property and equipment useful life | 7 years |
Significant Accounting Polici_5
Significant Accounting Policies - Schedule of Estimated Useful Lives of Property and Equipment (Details) | 6 Months Ended |
Jun. 30, 2020 | |
Laboratory Equipment | |
Property Plant And Equipment [Line Items] | |
Property and equipment useful life | 7 years |
Leasehold Improvements | |
Property Plant And Equipment [Line Items] | |
Property and equipment useful life | Lesser of useful life or remaining lease term |
Computer Equipment | |
Property Plant And Equipment [Line Items] | |
Property and equipment useful life | 3 years |
Furniture and Fixtures | |
Property Plant And Equipment [Line Items] | |
Property and equipment useful life | 7 years |
Significant Accounting Polici_6
Significant Accounting Policies - Schedule of Assets Measured at Fair Value (Details) - Fair Value, Recurring Basis - Active Markets (Level 1) - USD ($) $ in Thousands | Jun. 30, 2020 | Dec. 31, 2019 |
Fair Value Balance Sheet Grouping Financial Statement Captions [Line Items] | ||
Total | $ 131,069 | $ 167,896 |
Money Market Funds | ||
Fair Value Balance Sheet Grouping Financial Statement Captions [Line Items] | ||
Cash and cash equivalents | 110,846 | 167,805 |
Restricted cash | 91 | $ 91 |
US Treasury Securities | ||
Fair Value Balance Sheet Grouping Financial Statement Captions [Line Items] | ||
Investments | 13,674 | |
Corporate Bonds | ||
Fair Value Balance Sheet Grouping Financial Statement Captions [Line Items] | ||
Investments | $ 6,458 |
License Agreements - Additional
License Agreements - Additional Information (Details) - USD ($) $ in Thousands | 1 Months Ended | 3 Months Ended | 6 Months Ended | 12 Months Ended | |||
May 31, 2020 | Apr. 30, 2019 | Jun. 30, 2020 | Jun. 30, 2019 | Jun. 30, 2020 | Jun. 30, 2019 | Dec. 31, 2017 | |
Research And Development Arrangement Contract To Perform For Others [Line Items] | |||||||
Common stock, fair value | $ 113,236 | $ 113,236 | |||||
Research and development | $ 12,943 | $ 11,955 | $ 24,360 | $ 20,366 | |||
REGENXBIO GBA1 | |||||||
Research And Development Arrangement Contract To Perform For Others [Line Items] | |||||||
Licenses to be expired | 7 years | ||||||
REGENXBIO Option Genes License | |||||||
Research And Development Arrangement Contract To Perform For Others [Line Items] | |||||||
Expenses incurred for annual maintenance fee | $ 100 | ||||||
Maintenance Fee | $ 200 | ||||||
License | REGENXBIO GBA1 | |||||||
Research And Development Arrangement Contract To Perform For Others [Line Items] | |||||||
Stock issued during period, shares, new issues | 2,430,000 | ||||||
Common stock, fair value | $ 400 | ||||||
License | REGENXBIO Option Genes License | |||||||
Research And Development Arrangement Contract To Perform For Others [Line Items] | |||||||
Additional upfront fee paid | $ 600 | ||||||
Research and development | $ 1,800 |
Fair Value of Financial Instr_2
Fair Value of Financial Instruments - Additional Information (Details) - USD ($) | Jun. 30, 2020 | Dec. 31, 2019 | Jun. 30, 2019 |
Fair Value Balance Sheet Grouping Financial Statement Captions [Line Items] | |||
Transfers of assets between Level 1 and Level 2 | $ 0 | $ 0 | $ 0 |
Transfers of assets between Level 2 and Level 1 | 0 | 0 | 0 |
Transfers of liabilities between Level 1 and Level 2 | 0 | 0 | 0 |
Transfers of liabilities between Level 2 and Level 1 | 0 | 0 | 0 |
Transfers of assets between Level 1 and Level 3 | 0 | 0 | 0 |
Transfers of assets between Level 3 and Level 1 | 0 | 0 | 0 |
Transfers of assets between Level 2 and Level 3 | 0 | 0 | 0 |
Transfers of assets between Level 3 and Level 2 | 0 | 0 | 0 |
Transfers of liabilities between Level 1 and Level 3 | 0 | 0 | 0 |
Transfers of liabilities between Level 3 and Level 1 | 0 | 0 | 0 |
Transfers of liabilities between Level 2 and Level 3 | 0 | 0 | 0 |
Transfers of liabilities between Level 3 and Level 2 | 0 | 0 | $ 0 |
Fair Value, Inputs, Level 3 | |||
Fair Value Balance Sheet Grouping Financial Statement Captions [Line Items] | |||
Financial instruments, owned, at fair value | $ 0 | $ 0 |
Investments -Schedule of Invest
Investments -Schedule of Investments (Details) $ in Thousands | Jun. 30, 2020USD ($) |
Schedule Of Invested Securities [Line Items] | |
Amortized Cost | $ 20,133 |
Unrealized Losses | (1) |
Fair Value | 20,132 |
Corporate Bonds | |
Schedule Of Invested Securities [Line Items] | |
Amortized Cost | 6,458 |
Fair Value | 6,458 |
US Government Agencies Debt Securities | |
Schedule Of Invested Securities [Line Items] | |
Amortized Cost | 13,675 |
Unrealized Losses | (1) |
Fair Value | $ 13,674 |
Investments -Schedule of Contra
Investments -Schedule of Contractual Maturities of Debt Securities (Details) $ in Thousands | Jun. 30, 2020USD ($) |
Amortized Cost | |
Due in one year or less | $ 6,458 |
Due between one year and five years | 13,675 |
Total | 20,133 |
Fair Value | |
Due in one year or less | 6,458 |
Due between one year and five years | 13,674 |
Total | $ 20,132 |
Property and Equipment, Net - S
Property and Equipment, Net - Summary of Property and Equipment, Net (Details) - USD ($) $ in Thousands | Jun. 30, 2020 | Dec. 31, 2019 |
Property Plant And Equipment [Line Items] | ||
Gross property and equipment | $ 3,246 | $ 2,871 |
Less: Accumulated depreciation | (548) | (322) |
Property and equipment, net | 2,698 | 2,549 |
Laboratory Equipment | ||
Property Plant And Equipment [Line Items] | ||
Gross property and equipment | 2,104 | 1,730 |
Leasehold Improvements | ||
Property Plant And Equipment [Line Items] | ||
Gross property and equipment | 887 | 887 |
Computer Equipment | ||
Property Plant And Equipment [Line Items] | ||
Gross property and equipment | 36 | 36 |
Furniture and Fixtures | ||
Property Plant And Equipment [Line Items] | ||
Gross property and equipment | $ 219 | $ 218 |
Accrued Expenses and Other Cu_3
Accrued Expenses and Other Current Liabilities - Schedule of Accrued Expenses and Other Current Liabilities (Details) - USD ($) $ in Thousands | Jun. 30, 2020 | Dec. 31, 2019 |
Accrued Expenses And Other Current Liabilities [Abstract] | ||
Employee-related | $ 2,062 | $ 2,577 |
Clinical | 2,700 | 0 |
Research and development | 793 | 1,595 |
Professional fees | 3,639 | 634 |
Other | 241 | 524 |
Total accrued expenses and other current liabilities | $ 9,435 | $ 5,330 |
Redeemable Convertible Prefer_2
Redeemable Convertible Preferred Stock and Common Stock - Additional Information (Details) $ / shares in Units, $ in Thousands | 1 Months Ended | 3 Months Ended | 6 Months Ended | |
Jun. 30, 2019shares | Mar. 31, 2019USD ($)$ / sharesshares | Jun. 30, 2019shares | Jun. 30, 2019USD ($)shares | |
Temporary Equity [Line Items] | ||||
Issuance costs | $ | $ 3,016 | |||
Description of stock split | 1.62-for-one forward stock-split | |||
Stock split ratio | 1.62 | |||
Conversion of convertible preferred stock into common stock upon the closing of initial public offering, shares | shares | 19,435,131 | |||
Series B Preferred Stock | ||||
Temporary Equity [Line Items] | ||||
Preferred stock, shares authorized | shares | 3,958,046 | |||
Preferred stock, par value | $ / shares | $ 0.0001 | |||
Shares issued, price per share | $ / shares | $ 12.63 | |||
Aggregate net proceeds from issuance of stock | $ | $ 49,800 | |||
Issuance costs | $ | $ 200 | |||
Conversion of convertible preferred stock into common stock upon the closing of initial public offering, shares | shares | (3,958,046) | (3,958,046) |
Leases - Additional Information
Leases - Additional Information (Details) - USD ($) | 6 Months Ended | 12 Months Ended | ||
Jun. 30, 2020 | Jun. 30, 2019 | Dec. 31, 2019 | Sep. 30, 2017 | |
Lessee Lease Description [Line Items] | ||||
Operating lease arrangement, original term | 49 months | |||
Lessee, operating lease, option to extend | false | |||
Lessee, operating lease, option to terminate | false | |||
Qualified costs incurred | $ 400,000 | |||
Cash paid for amounts included in measurement of lease liabilities | $ 1,100,000 | $ 300,000 | ||
Maximum | ||||
Lessee Lease Description [Line Items] | ||||
Security deposit | $ 100,000 |
Leases - Summary of Components
Leases - Summary of Components of Lease Expense (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2020 | Jun. 30, 2019 | Jun. 30, 2020 | Jun. 30, 2019 | |
Leases [Abstract] | ||||
Operating lease cost | $ 569 | $ 441 | $ 1,138 | $ 900 |
Variable lease cost | 196 | 138 | 363 | 277 |
Total lease cost | $ 765 | $ 579 | $ 1,501 | $ 1,177 |
Weighted-average remaining lease term | 5 years 3 months 18 days | 5 years 8 months 12 days | 5 years 3 months 18 days | 5 years 8 months 12 days |
Weighted-average discount rate | 9.50% | 9.00% | 9.50% | 9.00% |
Leases - Summary of Maturities
Leases - Summary of Maturities of Remaining Operating Lease Liabilities (Details) - USD ($) $ in Thousands | Jun. 30, 2020 | Dec. 31, 2019 |
Leases [Abstract] | ||
2020 | $ 1,159 | $ 2,299 |
2021 | 2,379 | 2,379 |
2022 | 2,463 | 2,463 |
2023 | 2,549 | 2,549 |
2024 | 2,638 | 2,638 |
Thereafter | 2,263 | 2,263 |
Present value adjustment | (2,831) | (3,322) |
Present value of lease payments | $ 10,620 | $ 11,269 |
Stock-Based Compensation - Addi
Stock-Based Compensation - Additional Information (Details) - USD ($) $ in Millions | 1 Months Ended | 3 Months Ended | 6 Months Ended | ||||||
Jun. 30, 2019 | Apr. 30, 2019 | Dec. 31, 2018 | Mar. 31, 2018 | Jun. 30, 2019 | Jun. 30, 2020 | Jun. 30, 2019 | Dec. 31, 2019 | Dec. 31, 2017 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||||||
Conversion of convertible preferred stock into common stock upon the closing of initial public offering, shares | 19,435,131 | ||||||||
Preferred stock, shares authorized | 10,000,000 | 10,000,000 | |||||||
Preferred stock, shares issued | 0 | 0 | |||||||
Preferred stock, shares outstanding | 0 | ||||||||
Common Stock | |||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||||||
Conversion of convertible preferred stock into common stock upon the closing of initial public offering, shares | 19,435,131 | 19,435,131 | 19,435,131 | ||||||
Convertible Preferred Stock | |||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||||||
Preferred stock, shares outstanding | 19,435,131 | ||||||||
Stock Option | |||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||||||
Unrecognized compensation expense | $ 24.6 | ||||||||
Unrecognized compensation expense, estimated weighted-average period for recognition | 2 years 10 months 24 days | ||||||||
Dividend rate | 0.00% | ||||||||
Restricted Stock | |||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||||||
Repurchase of common stock | 2,349,000 | 2,349,000 | |||||||
Restricted Stock Awards | |||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||||||
Vesting period | 3 years | ||||||||
Unrecognized compensation expense, estimated weighted-average period for recognition | 1 year 1 month 6 days | ||||||||
Unrecognized stock-based compensation expense | $ 0.1 | ||||||||
After the First Year and Monthly Thereafter | Restricted Stock Awards | |||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||||||
Vesting percentage | 25.00% | ||||||||
2017 Equity Incentive Plan | |||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||||||
Increase in number of issuance of shares | 6,029,733 | 4,862,027 | 4,003,427 | ||||||
2017 Equity Incentive Plan | Maximum | |||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||||||
Number of issuance of shares | 2,511,000 | ||||||||
2019 Equity Incentive Plan | |||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||||||
Annual shares increase for future issuance by percentage | 4.00% | ||||||||
Vesting period | 3 years | ||||||||
Expiration period | 10 years | ||||||||
2019 Equity Incentive Plan | After the First Year and Monthly Thereafter | |||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||||||
Vesting percentage | 25.00% | ||||||||
2019 Equity Incentive Plan | Maximum | |||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||||||
Number of issuance of shares | 3,078,831 | 2,756,692 | |||||||
Exercise price as a percentage of grant date fair value | 100.00% | ||||||||
2019 Employee Stock Purchase Plan | |||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||||||
Annual shares increase for future issuance by percentage | 1.00% | ||||||||
Annual increase in ordinary shares for available for future issuance | 1,500,000 | ||||||||
Number of shares Purchase | 0 | ||||||||
2019 Employee Stock Purchase Plan | Maximum | |||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||||||
Number of issuance of shares | 671,387 |
Stock-Based Compensation - Summ
Stock-Based Compensation - Summary of Stock Option Activity (Details) - Stock Option $ / shares in Units, $ in Thousands | 6 Months Ended | 12 Months Ended |
Jun. 30, 2020USD ($)$ / sharesshares | Dec. 31, 2019USD ($)$ / sharesshares | |
Number of Awards | ||
Outstanding beginning balance, Number of Awards | shares | 5,681,422 | |
Granted, Number of Awards | shares | 1,132,600 | |
Exercised, Number of Awards | shares | (76,101) | |
Cancelled/Forfeited, Number of Awards | shares | (89,189) | |
Outstanding ending balance, Number of Awards | shares | 6,648,732 | 5,681,422 |
Exercisable, Number of Awards | shares | 2,459,316 | |
Vested and expected to vest, Number of Awards | shares | 6,648,732 | |
Weighted Average Exercise Price | ||
Outstanding beginning balance, Weighted Average Exercise Price | $ / shares | $ 3.60 | |
Granted, Weighted Average Exercise Price | $ / shares | 16.82 | |
Exercised, Weighted Average Exercise Price | $ / shares | 1.42 | |
Cancelled/Forfeited, Weighted Average Exercise Price | $ / shares | 3.12 | |
Outstanding ending balance, Weighted Average Exercise Price | $ / shares | 5.91 | $ 3.60 |
Exercisable, Weighted Average Exercise Price | $ / shares | 2.46 | |
Vested and expected to vest, Weighted Average Exercise Price | $ / shares | $ 5.91 | |
Weighted Average Remaining Contractual Life (Years) | ||
Outstanding, Weighted-Average Remaining Contractual Life (Years) | 8 years 4 months 24 days | 8 years 7 months 6 days |
Exercisable, Weighted-Average Remaining Contractual Life (Years) | 8 years | |
Vested and expected to vest, Weighted-Average Remaining Contractual Life (Years) | 8 years 4 months 24 days | |
Aggregate Intrinsic Value | ||
Outstanding, Aggregate Intrinsic Value | $ | $ 62,035 | $ 69,219 |
Exercisable, Aggregate Intrinsic Value | $ | 30,595 | |
Vested and expected to vest, Aggregate Intrinsic Value | $ | $ 62,035 |
Stock-Based Compensation - Sche
Stock-Based Compensation - Schedule of Assumptions to Estimate Fair Value of Stock Options Using Black-Scholes Option Pricing Model (Details) - Stock Option - $ / shares | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2020 | Jun. 30, 2019 | Jun. 30, 2020 | Jun. 30, 2019 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Grant date fair value | $ 11.95 | $ 7.26 | $ 12.11 | $ 6.18 |
Expected term | 5 years 9 months 18 days | 6 years 1 month 6 days | 6 years | 6 years 1 month 6 days |
Risk-free interest rate | 0.40% | 2.30% | 1.20% | 2.40% |
Expected volatility | 87.10% | 79.20% | 86.10% | 79.10% |
Dividend rate | 0.00% |
Stock-Based Compensation - Su_2
Stock-Based Compensation - Summary of Non-vested Restricted Stock Award Activity (Details) - Restricted Stock Awards - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2020 | Jun. 30, 2019 | Jun. 30, 2020 | Jun. 30, 2019 | |
Number of Restricted Stock Awards Outstanding | ||||
Non-vested restricted stock awards outstanding, Beginning | 978,759 | |||
Restricted stock awards vested | (293,622) | |||
Non-vested restricted stock awards outstanding, Ending | 685,137 | 685,137 | ||
Weighted Average Grant Date Fair Value Per Share | ||||
Non-vested restricted stock awards outstanding, Beginning | $ 0.18 | |||
Restricted stock awards vested | 0.18 | |||
Non-vested restricted stock awards outstanding, Ending | $ 0.18 | $ 0.18 | ||
Aggregate grant date fair value of restricted stock awards vested | $ 25 | $ 25 | $ 51 | $ 51 |
Stock-Based Compensation - Su_3
Stock-Based Compensation - Summary of Stock-Based Compensation Expense (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2020 | Jun. 30, 2019 | Jun. 30, 2020 | Jun. 30, 2019 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Total stock-based compensation expense | $ 2,249 | $ 1,109 | $ 3,881 | $ 1,721 |
Research and Development | Employees and Non-Employees | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Total stock-based compensation expense | 1,112 | 553 | 1,935 | 1,065 |
General and Administrative | Employees and Non-Employees | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Total stock-based compensation expense | $ 1,137 | $ 556 | $ 1,947 | $ 656 |
Related Party Transactions - Ad
Related Party Transactions - Additional Information (Details) - USD ($) $ in Millions | 1 Months Ended | 3 Months Ended | 6 Months Ended |
May 31, 2020 | Jun. 30, 2019 | Jun. 30, 2019 | |
Research and Development | |||
Related Party Transaction [Line Items] | |||
Expenses incurred for annual maintenance fee | $ 0.2 | $ 0.2 | |
REGENXBIO Option Genes License | |||
Related Party Transaction [Line Items] | |||
Expenses incurred for annual maintenance fee | $ 0.1 | ||
REGENXBIO Option Genes License | Research and Development | |||
Related Party Transaction [Line Items] | |||
Additional upfront fee paid | 0.6 | 0.6 | |
Aggregate payments to related parties | $ 1.8 | $ 1.8 |
Net Loss Per Share - Schedule o
Net Loss Per Share - Schedule of Potentially Dilutive Common Stock Equivalents Excluded from Calculation of Diluted Net Loss Per Share (Details) - shares | 6 Months Ended | |
Jun. 30, 2020 | Jun. 30, 2019 | |
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Antidilutive securities excluded from computation of earnings per share (in shares) | 7,333,869 | 7,054,471 |
Common stock options issued and outstanding | ||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Antidilutive securities excluded from computation of earnings per share (in shares) | 6,648,732 | 5,782,090 |
Restricted stock subject to future vesting | ||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Antidilutive securities excluded from computation of earnings per share (in shares) | 685,137 | 1,272,381 |
Net Loss Per Share - Schedule_2
Net Loss Per Share - Schedule of Basic and Diluted Net Loss per Share (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2020 | Jun. 30, 2019 | Jun. 30, 2020 | Jun. 30, 2019 | |
Numerator: | ||||
Net loss | $ (22,100) | $ (15,103) | $ (40,675) | $ (25,048) |
Denominator: | ||||
Weighted-average number of shares-basic and diluted | 33,467,346 | 26,212,356 | 33,367,344 | 23,945,198 |
Net loss per share—basic and diluted | $ (0.66) | $ (0.58) | $ (1.22) | $ (1.05) |
Commitments And Contingencies -
Commitments And Contingencies - Additional Information (Details) - USD ($) | Jun. 30, 2020 | Dec. 31, 2019 |
Commitments And Contingencies Disclosure [Abstract] | ||
Estimated litigation liability | $ 0 | $ 0 |
Subsequent Event - Additional I
Subsequent Event - Additional Information (Details) - USD ($) $ in Thousands | Aug. 11, 2020 | Jul. 31, 2020 | Jun. 30, 2019 | Jun. 30, 2019 |
Subsequent Event [Line Items] | ||||
Common stock, aggregate offering price | $ 113,236 | $ 113,236 | ||
Subsequent Event | ||||
Subsequent Event [Line Items] | ||||
Amount received from insurance claim | $ 3,000 | |||
Subsequent Event | Open Market Sale Agreement | Maximum | Jefferies LLC | ||||
Subsequent Event [Line Items] | ||||
Common stock, aggregate offering price | $ 75,000 |