Document and Entity Information
Document and Entity Information - shares | 9 Months Ended | |
Sep. 30, 2020 | Oct. 30, 2020 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Entity Registrant Name | AKOUOS, INC. | |
Document Period End Date | Sep. 30, 2020 | |
Entity Central Index Key | 0001722271 | |
Entity Current Reporting Status | Yes | |
Entity Filer Category | Non-accelerated Filer | |
Current Fiscal Year End Date | --12-31 | |
Entity Common Stock, Shares Outstanding | 34,382,665 | |
Amendment Flag | false | |
Document Fiscal Year Focus | 2020 | |
Document Fiscal Period Focus | Q3 | |
Entity Shell Company | false | |
Entity Small Business | true | |
Entity Emerging Growth Company | true | |
Entity Interactive Data Current | Yes | |
Entity Ex Transition Period | false |
CONSOLIDATED BALANCE SHEETS
CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands | Sep. 30, 2020 | Dec. 31, 2019 |
Current assets: | ||
Cash and cash equivalents | $ 178,622 | $ 25,078 |
Marketable securities | 141,452 | |
Prepaid expenses and other current assets | 2,154 | 1,061 |
Total current assets | 322,228 | 26,139 |
Property and equipment, net | 12,667 | 11,492 |
Operating lease right-of-use assets | 6,035 | 6,214 |
Restricted cash | 1,317 | 1,317 |
Total assets | 342,247 | 45,162 |
Current liabilities: | ||
Accounts payable | 817 | 339 |
Accrued expenses and other current liabilities | 6,730 | 4,962 |
Operating lease liabilities | 1,071 | 641 |
Total current liabilities | 8,618 | 5,942 |
Operating lease liabilities, net of current portion | 12,321 | 13,143 |
Other long‑term liabilities | 188 | |
Total liabilities | 20,939 | 19,273 |
Commitments and contingencies (See Note 11) | ||
Convertible preferred stock (Series Seed, Seed 1, A, and B), $0.0001 par value; no shares authorized at September 30, 2020 and 178,349,005 shares authorized at December 31, 2019; no shares issued and outstanding at September 30, 2020 and 178,349,005 shares issued and outstanding at December 31, 2019 | 58,690 | |
Stockholders’ equity (deficit): | ||
Common stock, $0.0001 par value; 200,000,000 shares authorized at September 30, 2020 and 235,000,000 shares authorized at December 31, 2019; 34,374,348 shares issued and outstanding at September 30, 2020; and 997,165 shares issued and outstanding at December 31, 2019 | 3 | |
Additional paid-in capital | 390,473 | 323 |
Accumulated other comprehensive income | 8 | |
Accumulated deficit | (69,176) | (33,124) |
Total stockholders’ equity (deficit) | 321,308 | (32,801) |
Total liabilities, convertible preferred stock and stockholders’ equity | $ 342,247 | $ 45,162 |
CONSOLIDATED BALANCE SHEETS (Pa
CONSOLIDATED BALANCE SHEETS (Parenthetical) - $ / shares | Sep. 30, 2020 | Dec. 31, 2019 |
CONSOLIDATED BALANCE SHEETS | ||
Convertible preferred stock, Par value | $ 0.0001 | $ 0.0001 |
Convertible preferred stock, Shares authorized | 0 | 178,349,005 |
Convertible preferred stock, Shares issued | 0 | 178,349,005 |
Convertible preferred stock, Shares outstanding | 0 | 178,349,005 |
Preferred stock, Par value | $ 0.0001 | $ 0.0001 |
Preferred stock, Shares authorized | 5,000,000 | 0 |
Preferred stock, Shares issued | 0 | 0 |
Preferred stock, Shares outstanding | 0 | 0 |
Common stock, Par value | $ 0.0001 | $ 0.0001 |
Common stock, Shares authorized | 200,000,000 | 235,000,000 |
Common stock, Shares issued | 34,374,348 | 997,165 |
Common stock, Shares outstanding | 34,374,348 | 997,165 |
CONSOLIDATED STATEMENT OF OPERA
CONSOLIDATED STATEMENT OF OPERATIONS AND COMPREHENSIVE LOSS - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2020 | Sep. 30, 2019 | Sep. 30, 2020 | Sep. 30, 2019 | |
Operating expenses: | ||||
Research and development | $ 8,641 | $ 4,377 | $ 26,612 | $ 11,886 |
General and administrative | 4,478 | 887 | 9,646 | 2,267 |
Total operating expenses | 13,119 | 5,264 | 36,258 | 14,153 |
Loss from operations | (13,119) | (5,264) | (36,258) | (14,153) |
Other income (expense): | ||||
Change in fair value of preferred stock tranche liability | (3,013) | (2,260) | ||
Interest income | 21 | 70 | 201 | 298 |
Other income (expense), net | 9 | (3) | 5 | (8) |
Total other income (expense), net | 30 | (2,946) | 206 | (1,970) |
Net loss and comprehensive loss | $ (13,089) | $ (8,210) | $ (36,052) | $ (16,123) |
Net loss per share attributable to common stockholders, basic and diluted | $ (0.85) | $ (13.21) | $ (3.01) | $ (27.48) |
Weighted-average common shares outstanding, basic and diluted | 15,334,241 | 621,581 | 11,991,870 | 586,728 |
Unrealized gain on marketable securities | $ 8 | $ 8 | ||
Total other comprehensive income | 8 | 8 | ||
Total comprehensive loss | $ (13,081) | $ (8,210) | $ (36,044) | $ (16,123) |
CONSOLIDATED STATEMENTS OF CONV
CONSOLIDATED STATEMENTS OF CONVERTIBLE PREFERRED STOCK AND STOCKHOLDERS' EQUITY/(DEFICIT) - USD ($) $ in Thousands | Common Stock | Additional Paid-In Capital | Accumulated Other Comprehensive Income | Accumulated Deficit | Total |
Balances at beginning of period at Dec. 31, 2018 | $ 27,647 | ||||
Convertible preferred stock, Shares outstanding, beginning of period at Dec. 31, 2018 | 103,015,190 | ||||
Balances at end of period at Mar. 31, 2019 | $ 27,647 | ||||
Convertible preferred stock, Shares outstanding, end of period at Mar. 31, 2019 | 103,015,190 | ||||
Balances at beginning of period at Dec. 31, 2018 | $ 154 | $ (7,383) | $ (7,229) | ||
Balances at beginning of period (in shares) at Dec. 31, 2018 | 935,887 | ||||
Increase (Decrease) in Stockholders' Equity [Roll Forward] | |||||
Issuance of common stock upon exercise of stock options (in shares) | 197 | ||||
Vesting of restricted common stock from early-exercised stock options and sales of restricted common stock | 7 | 7 | |||
Stock-based compensation expense | 18 | 18 | |||
Net loss | (5,909) | (5,909) | |||
Balances at end of period at Mar. 31, 2019 | 179 | (13,292) | (13,113) | ||
Balances at end of period (in shares) at Mar. 31, 2019 | 936,084 | ||||
Balances at beginning of period at Dec. 31, 2018 | $ 27,647 | ||||
Convertible preferred stock, Shares outstanding, beginning of period at Dec. 31, 2018 | 103,015,190 | ||||
Balances at end of period at Sep. 30, 2019 | $ 58,690 | ||||
Convertible preferred stock, Shares outstanding, end of period at Sep. 30, 2019 | 178,349,005 | ||||
Balances at beginning of period at Dec. 31, 2018 | 154 | (7,383) | $ (7,229) | ||
Balances at beginning of period (in shares) at Dec. 31, 2018 | 935,887 | ||||
Increase (Decrease) in Stockholders' Equity [Roll Forward] | |||||
Net loss | (16,123) | ||||
Balances at end of period at Sep. 30, 2019 | 229 | (23,506) | (23,277) | ||
Balances at end of period (in shares) at Sep. 30, 2019 | 980,237 | ||||
Balances at beginning of period at Mar. 31, 2019 | $ 27,647 | ||||
Convertible preferred stock, Shares outstanding, beginning of period at Mar. 31, 2019 | 103,015,190 | ||||
Balances at end of period at Jun. 30, 2019 | $ 27,647 | ||||
Convertible preferred stock, Shares outstanding, end of period at Jun. 30, 2019 | 103,015,190 | ||||
Balances at beginning of period at Mar. 31, 2019 | 179 | (13,292) | $ (13,113) | ||
Balances at beginning of period (in shares) at Mar. 31, 2019 | 936,084 | ||||
Increase (Decrease) in Stockholders' Equity [Roll Forward] | |||||
Issuance of common stock upon exercise of stock options | 13 | 13 | |||
Issuance of common stock upon exercise of stock options (in shares) | 16,608 | ||||
Vesting of restricted common stock from early-exercised stock options and sales of restricted common stock | (9) | (9) | |||
Stock-based compensation expense | 18 | 18 | |||
Net loss | (2,004) | (2,004) | |||
Balances at end of period at Jun. 30, 2019 | 201 | (15,296) | (15,095) | ||
Balances at end of period (in shares) at Jun. 30, 2019 | 952,692 | ||||
Increase (Decrease) in Temporary Equity [Roll Forward] | |||||
Issuance of convertible preferred stock, net | $ 25,016 | ||||
Issuance of convertible preferred stock, net (in shares) | 75,333,815 | ||||
Reclassification of preferred stock tranche liability upon settlement | $ 6,027 | ||||
Balances at end of period at Sep. 30, 2019 | $ 58,690 | ||||
Convertible preferred stock, Shares outstanding, end of period at Sep. 30, 2019 | 178,349,005 | ||||
Increase (Decrease) in Stockholders' Equity [Roll Forward] | |||||
Issuance of common stock upon exercise of stock options | 43 | $ 43 | |||
Issuance of common stock upon exercise of stock options (in shares) | 27,545 | ||||
Vesting of restricted common stock from early-exercised stock options and sales of restricted common stock | (36) | (36) | |||
Stock-based compensation expense | 21 | 21 | |||
Net loss | (8,210) | (8,210) | |||
Balances at end of period at Sep. 30, 2019 | 229 | (23,506) | (23,277) | ||
Balances at end of period (in shares) at Sep. 30, 2019 | 980,237 | ||||
Balances at beginning of period at Dec. 31, 2019 | $ 58,690 | ||||
Convertible preferred stock, Shares outstanding, beginning of period at Dec. 31, 2019 | 178,349,005 | ||||
Increase (Decrease) in Temporary Equity [Roll Forward] | |||||
Issuance of convertible preferred stock, net | $ 104,837 | ||||
Issuance of convertible preferred stock, net (in shares) | 221,399,223 | ||||
Balances at end of period at Mar. 31, 2020 | $ 163,527 | ||||
Convertible preferred stock, Shares outstanding, end of period at Mar. 31, 2020 | 399,748,228 | ||||
Balances at beginning of period at Dec. 31, 2019 | 323 | (33,124) | $ (32,801) | ||
Balances at beginning of period (in shares) at Dec. 31, 2019 | 997,165 | ||||
Increase (Decrease) in Stockholders' Equity [Roll Forward] | |||||
Issuance of common stock upon exercise of stock options | 7 | 7 | |||
Issuance of common stock upon exercise of stock options (in shares) | 13,630 | ||||
Vesting of restricted common stock from early-exercised stock options and sales of restricted common stock | 12 | 12 | |||
Stock-based compensation expense | 111 | 111 | |||
Net loss | (10,440) | (10,440) | |||
Balances at end of period at Mar. 31, 2020 | 453 | (43,564) | (43,111) | ||
Balances at end of period (in shares) at Mar. 31, 2020 | 1,010,795 | ||||
Balances at beginning of period at Dec. 31, 2019 | $ 58,690 | ||||
Convertible preferred stock, Shares outstanding, beginning of period at Dec. 31, 2019 | 178,349,005 | ||||
Convertible preferred stock, Shares outstanding, end of period at Sep. 30, 2020 | 0 | ||||
Balances at beginning of period at Dec. 31, 2019 | 323 | (33,124) | $ (32,801) | ||
Balances at beginning of period (in shares) at Dec. 31, 2019 | 997,165 | ||||
Increase (Decrease) in Stockholders' Equity [Roll Forward] | |||||
Net loss | (36,052) | ||||
Unrealized gain (loss) on marketable securities | 8 | ||||
Balances at end of period at Sep. 30, 2020 | $ 3 | 390,473 | $ 8 | (69,176) | 321,308 |
Balances at end of period (in shares) at Sep. 30, 2020 | 34,374,348 | ||||
Balances at beginning of period at Mar. 31, 2020 | $ 163,527 | ||||
Convertible preferred stock, Shares outstanding, beginning of period at Mar. 31, 2020 | 399,748,228 | ||||
Increase (Decrease) in Temporary Equity [Roll Forward] | |||||
Conversion of convertible preferred stock to common stock | $ (163,527) | ||||
Conversion of convertible preferred stock, shares | (399,748,228) | ||||
Balances at beginning of period at Mar. 31, 2020 | 453 | (43,564) | $ (43,111) | ||
Balances at beginning of period (in shares) at Mar. 31, 2020 | 1,010,795 | ||||
Increase (Decrease) in Stockholders' Equity [Roll Forward] | |||||
Issuance of common stock upon exercise of stock options | 31 | 31 | |||
Issuance of common stock upon exercise of stock options (in shares) | 15,181 | ||||
Vesting of restricted common stock from early-exercised stock options and sales of restricted common stock | 7 | 7 | |||
Stock-based compensation expense | 490 | 490 | |||
Issuance of common stock upon completion of initial public offering, net of commissions, underwriting discounts and offering costs | $ 1 | 223,849 | 223,850 | ||
Issuance of common stock upon completion of initial public offering, net of commissions, underwriting discounts and offering costs, shares | 14,375,000 | ||||
Conversion of Stock, Amount Issued | $ 2 | 163,510 | 163,512 | ||
Conversion of Stock, Shares Issued | 18,969,672 | ||||
Net loss | (12,523) | (12,523) | |||
Balances at end of period at Jun. 30, 2020 | $ 3 | 388,340 | (56,087) | $ 332,256 | |
Balances at end of period (in shares) at Jun. 30, 2020 | 34,370,648 | ||||
Convertible preferred stock, Shares outstanding, end of period at Sep. 30, 2020 | 0 | ||||
Increase (Decrease) in Stockholders' Equity [Roll Forward] | |||||
Issuance of common stock upon exercise of stock options | 3 | $ 3 | |||
Issuance of common stock upon exercise of stock options (in shares) | 3,700 | ||||
Vesting of restricted common stock from early-exercised stock options and sales of restricted common stock | 54 | 54 | |||
Stock-based compensation expense | 2,076 | 2,076 | |||
Net loss | (13,089) | (13,089) | |||
Unrealized gain (loss) on marketable securities | 8 | 8 | |||
Balances at end of period at Sep. 30, 2020 | $ 3 | $ 390,473 | $ 8 | $ (69,176) | $ 321,308 |
Balances at end of period (in shares) at Sep. 30, 2020 | 34,374,348 |
CONSOLIDATED STATEMENTS OF CO_2
CONSOLIDATED STATEMENTS OF CONVERTIBLE PREFERRED STOCK AND STOCKHOLDERS' DEFICIT (Parenthetical) - USD ($) $ in Thousands | 1 Months Ended | 3 Months Ended | 9 Months Ended | |
Feb. 29, 2020 | Mar. 31, 2020 | Sep. 30, 2019 | Sep. 30, 2020 | |
CONSOLIDATED STATEMENTS OF CONVERTIBLE PREFERRED STOCK AND STOCKHOLDERS' EQUITY/(DEFICIT) | ||||
Issuance cost | $ 200 | $ 228 | $ 34 | $ 3,419 |
CONSOLIDATED STATEMENTS OF CASH
CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | |
Sep. 30, 2019 | Sep. 30, 2020 | Sep. 30, 2019 | |
Cash flows from operating activities: | |||
Net loss | $ (8,210) | $ (36,052) | $ (16,123) |
Adjustments to reconcile net loss to net cash used in operating activities: | |||
Depreciation and amortization expense | 100 | 1,433 | 172 |
Net amortization of premiums and accretion of discounts on marketable securities | (10) | ||
Amortization of operating lease right-of-use assets | 179 | 254 | |
Change in fair value of preferred stock tranche liability | 3,013 | 2,260 | |
Stock-based compensation expense | 2,677 | 57 | |
Changes in operating assets and liabilities: | |||
Prepaid expenses and other current assets | (1,093) | (36) | |
Accounts payable | 348 | 290 | |
Operating lease liabilities | (392) | 351 | |
Accrued expenses and other current liabilities | 2,582 | 1,282 | |
Other long-term liabilities | (62) | ||
Net cash used in operating activities | (30,328) | (11,555) | |
Cash flows from investing activities: | |||
Purchases of property and equipment | (3,234) | (929) | |
Purchases of marketable securities | (141,434) | ||
Net cash used in investing activities | (144,668) | (929) | |
Cash flows from financing activities: | |||
Proceeds from issuance of convertible preferred stock, net of issuance costs paid | 104,837 | 25,016 | |
Proceeds from initial public offering of common stock | 227,269 | ||
Payments of finance lease obligations | (189) | (74) | |
Proceeds from exercise of stock options and sale of restricted common stock | 42 | 56 | |
Payments of initial public offering costs | (34) | (3,419) | |
Net cash provided by financing activities | 328,540 | 24,998 | |
Net increase in cash, cash equivalents and restricted cash | 153,544 | 12,514 | |
Cash, cash equivalents and restricted cash at beginning of period | 26,395 | 24,340 | |
Cash, cash equivalents and restricted cash at end of period | 36,854 | 179,939 | 36,854 |
Supplemental cash flow information: | |||
Cash paid for interest | 5 | 8 | |
Supplemental disclosure of non‑cash investing and financing information: | |||
Purchases of property and equipment included in accounts payable and accrued expenses | 157 | ||
Settlement of preferred stock tranche liability | 6,027 | ||
Operating lease liabilities arising from obtaining right-of-use assets | 6,550 | ||
Leasehold improvements paid by lessor | 4,922 | ||
Vesting of common stock subject to repurchase | 73 | (38) | |
Reconciliation of cash, cash equivalents and restricted cash: | |||
Total cash, cash equivalents and restricted cash shown in the statement of cash flows | $ 36,854 | $ 179,939 | $ 36,854 |
Nature of the Business and Basi
Nature of the Business and Basis of Presentation | 9 Months Ended |
Sep. 30, 2020 | |
Nature of the Business and Basis of Presentation | |
Nature of the Business and Basis of Presentation | 1. Nature of the Business and Basis of Presentation Akouos, Inc., together with its consolidated subsidiary (the “Company” or “Akouos”), is a precision genetic medicine company dedicated to its mission of developing gene therapies with the potential to restore, improve, and preserve high‑acuity physiologic hearing for individuals worldwide who live with disabling hearing loss. The Company was formed as a limited liability corporation under the laws of the Commonwealth of Massachusetts in March 2016 under the name Akouos, LLC and converted into a corporation under the laws of the State of Delaware in November 2016 under the name Akouos, Inc. The Company is subject to risks and uncertainties common to early‑stage companies in the biotechnology industry, including, but not limited to, development by competitors of new technological innovations, dependence on key personnel, protection of proprietary technology, compliance with government regulations, the impact of the COVID‑19 coronavirus, and the ability to secure additional capital to fund operations. Product candidates currently under development will require significant additional research and development efforts, including extensive preclinical and clinical testing and regulatory approval prior to commercialization. These efforts require significant amounts of additional capital, adequate personnel and infrastructure, and extensive compliance‑reporting capabilities. Even if the Company’s development efforts are successful, it is uncertain when, if ever, the Company will realize significant revenue from product sales. On June 18, 2020, the Company effected a one-for-21.073 reverse stock split of its issued and outstanding shares of common stock and a proportional adjustment to the existing conversion ratios for each series of the Company’s convertible preferred stock. Accordingly, all share and per share amounts for all periods presented in the accompanying condensed consolidated financial statements and notes thereto have been adjusted retroactively, where applicable, to reflect this reverse stock split and adjustment of the preferred stock conversion ratios. On June 30, 2020, the Company completed its initial public offering of its common stock and issued and sold 14,375,000 shares of its common stock, at a public offering price of $17.00 per share, for gross proceeds of $244.4 million, or net proceeds of $223.8 million after deducting underwriting discounts, commissions, and offering expenses. Upon the closing of the Company’s initial public offering on June 30, 2020, all shares of convertible preferred stock automatically converted into 18,969,672 shares of common stock. The accompanying condensed consolidated financial statements have been prepared on the basis of continuity of operations, realization of assets, and the satisfaction of liabilities and commitments in the ordinary course of business. The Company has primarily funded its operations with proceeds from sales of convertible preferred stock and proceeds from the Company’s initial public offering of common stock. The Company has incurred losses and negative cash flows from operations since its inception. As of September 30, 2020, the Company had an accumulated deficit of $69.2 million. The Company expects that its operating losses and negative cash flows will continue for the foreseeable future. The Company expects that its existing cash, cash equivalents and marketable securities will be sufficient to fund its operating expenses and capital expenditure requirements for at least 12 months from the issuance date of the interim condensed consolidated financial statements. The Company expects to seek additional funding through public and private equity financings, debt financings, collaborations, licensing arrangements, and/or strategic alliances. The Company may not be able to obtain financing on acceptable terms, or at all, and the Company may not be able to enter into collaborations or other such arrangements. The terms of any financing may adversely affect the holdings or the rights of the Company’s stockholders. If the Company is unable to obtain funding, the Company could be forced to delay, reduce, or eliminate some or all of its research and development programs, product portfolio expansion, or commercialization efforts, which could adversely affect its business prospects. Although management continues to pursue these plans, there is no assurance that the Company will be successful in obtaining sufficient funding on terms acceptable to the Company to fund continuing operations, if at all. Impact of the COVID‑19 Coronavirus The COVID‑19 pandemic, which began in December 2019 and has spread worldwide, has caused many governments to implement measures to slow the spread of the outbreak. The outbreak and government measures taken in response have had and will continue to have a significant impact, both direct and indirect, on businesses and commerce, as worker shortages have occurred, supply chains have been disrupted, and facilities and production have been suspended. The future progression of the pandemic and its effects on the Company’s business and operations are uncertain. The COVID‑19 pandemic may affect the Company’s ability to initiate and complete preclinical studies, delay the initiation of its planned clinical trial or future clinical trials, disrupt regulatory activities, or have other adverse effects on its business and operations. In particular, the Company and its contract manufacturing organizations (“CMOs”) and contract research organizations (“CROs”) may face disruptions that may affect the Company’s ability to initiate and complete preclinical studies, manufacturing disruptions, and delays at clinical trial sites. The pandemic has already caused significant disruptions in the financial markets, and may continue to cause such disruptions, which could impact the Company’s ability to raise additional funds to support its operations. Moreover, the pandemic has significantly impacted economies worldwide and could result in adverse effects on the Company’s business and operations. The Company is monitoring the potential impact of the COVID‑19 pandemic on its business and financial statements. To date, the Company has not experienced material business disruptions or incurred impairment losses in the carrying values of its assets as a result of the pandemic and it is not aware of any specific related event or circumstance that would require it to revise its estimates reflected in these condensed consolidated financial statements. The extent to which the COVID‑19 pandemic will directly or indirectly impact the Company’s business, results of operations and financial condition, including planned and future clinical trials and research and development costs, will depend on future developments that are highly uncertain, including as a result of new information that may emerge concerning COVID‑19, the actions taken to contain or treat it, and the duration and intensity of the related effects. Basis of Presentation The accompanying condensed consolidated financial statements reflect the operations of the Company and its wholly owned, domestic subsidiary. Intercompany balances and transactions have been eliminated in consolidation. The accompanying condensed consolidated financial statements have been prepared in conformity with generally accepted accounting principles in the United States of America (“GAAP”). Any reference in these notes to applicable guidance is meant to refer to the authoritative GAAP as found in the Accounting Standards Codification (“ASC”) and Accounting Standards Update (“ASU”) of the Financial Accounting Standards Board (“FASB”). |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 9 Months Ended |
Sep. 30, 2020 | |
Summary of Significant Accounting Policies | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies Summary of Significant Accounting Policies The significant accounting policies and estimates used in the preparation of the accompanying consolidated financial statements are described in the Company’s audited consolidated financial statements for the year ended December 31, 2019 included in the Company’s final prospectus for its initial public offering filed pursuant to Rule 424(b)(4) under the Securities Act of 1933, as amended, with the Securities and Exchange Commission, or the SEC, on June 26, 2020. There have been no material changes in the Company’s significant accounting policies during the nine months ended September 30, 2020. Use of Estimates The preparation of the Company’s consolidated financial statements in conformity with 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 revenue and expenses during the reporting periods. Significant estimates and assumptions reflected in these condensed consolidated financial statements include, but are not limited to, the accrual of research and development expenses and the valuations of common stock, stock options, and a preferred stock tranche liability. 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. On an ongoing basis, management evaluates its estimates, as there are changes in circumstances, facts, and experience. Changes in estimates are recorded in the period in which they become known. Actual results may differ from those estimates or assumptions. Unaudited Interim Financial Information The accompanying condensed consolidated balance sheet as of September 30, 2020, the condensed consolidated statements of operations and comprehensive loss for the three and nine months ended September 30, 2020 and 2019, the condensed consolidated statement of cash flows for the nine months ended September 30, 2020 and 2019 and the condensed consolidated statements of convertible preferred stock and stockholders’ equity (deficit) for the three and nine months ended September 30, 2020 and 2019 are unaudited. The unaudited interim condensed consolidated financial statements have been prepared on the same basis as the audited annual consolidated financial statements and, in the opinion of management, reflect all adjustments, which include only normal recurring adjustments, necessary for the fair statement of the Company’s financial position as of September 30, 2020 and necessary for the fair statement of the Company’s financial position as of September 30, 2020 and the results of its operations for the three and nine months ended September 30, 2020 and 2019 and its cash flows for the nine months ended September 30, 2020 and 2019. The financial data and other information disclosed in these notes related to the three and nine months ended September 30, 2020 and 2019 are also unaudited. The results for the three and nine months ended September 30, 2020 are not necessarily indicative of results to be expected for the year ending December 31, 2020, any other interim periods, or any future year or period. Concentrations of Credit Risk and of Significant Suppliers Financial instruments that potentially expose the Company to concentrations of credit risk consist primarily of cash, cash equivalents and marketable securities. The Company maintains its cash and cash equivalents at one accredited financial institution. The Company’s cash equivalents as of September 30, 2020 and December 31, 2019 consisted of U.S. government money market funds. Marketable securities consist of U.S. Treasury notes with maturities of less than twelve months. The Company does not believe that it is subject to unusual credit risk beyond the normal credit risk associated with commercial banking relationships. The Company is dependent on third‑party vendors for its product candidates. In particular, the Company relies, and expects to continue to rely, on a small number of vendors to manufacture materials and components required for the production of its product candidates. These programs could be adversely affected by a significant interruption in the manufacturing process. Cash Equivalents The Company considers all highly liquid investments with a remaining maturity when purchased of three months or less to be cash equivalents. Restricted Cash In connection with the Company’s lease agreement entered into in December 2018, the Company is required to maintain a letter of credit of $1.3 million for the benefit of the landlord. As of September 30, 2020 and December 31, 2019, this amount was classified as restricted cash (non‑current) in the condensed consolidated balance sheets. The Company is required to maintain a separate cash balance of less than $0.1 million pledged as collateral for a credit card account. As of September 30, 2020 and December 31, 2019, this amount was classified as restricted cash (non‑current) on the condensed consolidated balance sheets. Marketable Securities Marketable securities represent holdings of available-for-sale marketable debt securities in accordance with the Company’s investment policy. Marketable securities that mature within one-year from the balance sheet date are classified as current in the condensed consolidated balance sheets. Investments in marketable securities are recorded at fair value, with any unrealized gains and losses reported within accumulated other comprehensive income (loss) as a separate component of stockholders’ equity (deficit) until realized or until a determination is made that an other-than-temporary decline in market value has occurred. The amortized cost of debt securities is adjusted for amortization of premiums and accretion of discounts to maturity. Such amortization and accretion, together with interest on securities sold, is determined based on the specific identification method and any realized gains or losses on the sale of investments are reflected as a component of other income (expense). Fair Value Measurements Certain assets and liabilities of the Company are carried at fair value under 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. Financial assets and financial 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—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 Company’s cash equivalents, marketable securities and preferred stock tranche liability are carried at fair value, determined according to the fair value hierarchy described above (see Note 3). The carrying values of the Company’s accounts payable and accrued expenses approximate their fair values due to the short‑term nature of these liabilities. Property and Equipment Property and equipment are stated at cost less accumulated depreciation and amortization. Depreciation and amortization expense is recognized using the straight‑line method over the estimated useful life of each asset as follows: Estimated Useful Life Laboratory equipment 5 years Furniture and fixtures 4 years Leasehold improvements Shorter of term of lease or 15 years Costs for capital assets not yet placed into service are capitalized as construction‑in‑progress and depreciated once placed into service. Upon retirement or sale, the cost of assets disposed of and the related accumulated depreciation and amortization are removed from the accounts and any resulting gain or loss is included in loss from operations. Expenditures for repairs and maintenance that do not improve or extend the life of the respective assets are charged to expense as incurred. Classification and Accretion of Convertible Preferred Stock The Company’s convertible preferred stock was classified outside of stockholders’ equity (deficit) on the condensed consolidated balance sheets because the holders of such shares had liquidation rights in the event of a deemed liquidation that, in certain situations, was not solely within the control of the Company and would require the redemption of the then‑outstanding convertible preferred stock. The Company’s Series Seed, Series Seed 1, Series A, and Series B convertible preferred stock were not redeemable, except in the event of a deemed liquidation. Because the occurrence of a deemed liquidation event was not currently probable, the carrying values of the convertible preferred stock were not being accreted to their redemption values. Subsequent adjustments to the carrying values to the convertible preferred stock would be made only when a deemed liquidation event became probable. Upon the closing of the Company’s initial public offering on June 30, 2020, all outstanding shares of convertible preferred stock automatically converted into 18,969,672 shares of common stock. Impairment of Long‑Lived Assets Long‑lived assets consist of property and equipment. Long‑lived assets to be held and used 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 group for recoverability, the Company compares forecasts of undiscounted cash flows expected to result from the use and eventual disposition of the long‑lived asset group to its carrying value. An impairment loss would be recognized in loss from operations when estimated undiscounted future cash flows expected to result from the use of an asset group are less than its carrying amount. Impairment is measured based on the excess of the carrying value of the related assets over the fair value of such assets. The Company did not record any impairment losses on long‑lived assets during the three and nine months ended September 30, 2020 and 2019. Segment Information The Company manages its operations as a single segment for the purposes of assessing performance and making operating decisions. The Company’s focus is on developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals with hearing loss. All of the Company’s long‑lived assets are located in the United States. Government Contracts The Company has received funding from government contracts that reimburse the Company for certain allowable costs for funded projects. For contracts with government agencies, when the Company has concluded that it is the principal in conducting the research and development expenses but where the funding arrangement is not considered core to the Company’s ongoing operations, the Company classifies the recognized funding received as other income in its condensed consolidated statement of operations and comprehensive loss. Research and Development Costs Costs for research and development activities are expensed in the period in which they are incurred. Research and development expenses consist of costs incurred in performing research and development activities, including salaries and bonuses, stock‑based compensation, employee benefits, facilities costs, laboratory supplies, depreciation and amortization, manufacturing expenses, and external costs of vendors engaged to conduct research and preclinical development activities as well as the cost of licensing technology. Upfront payments under license agreements are expensed upon receipt of the license, and annual maintenance fees under license agreements are expensed in the period in which they are incurred. Milestone payments under license agreements are accrued, with a corresponding expense being recognized, in the period in which the milestone is determined to be probable of achievement and the related amount is reasonably estimable. Nonrefundable advance payments for goods or services to be received in the future for use in research and development activities are recorded as prepaid expenses. The prepaid amounts are expensed as the related goods are delivered or the services are performed. Research, Development, and Manufacturing Contract Costs and Accruals The Company has entered into various research, development, and manufacturing contracts with research institutions and other companies. These agreements are generally cancelable, and related costs are recorded as research and development expenses as incurred. The Company records accruals for estimated ongoing research and development costs. When billing terms under these contracts do not coincide with the timing of when the work is performed, the Company is required to make estimates of outstanding obligations to those third parties as of period end. Any accrual estimates are based on a number of factors, including the Company’s knowledge of the progress towards completion of the research, development, and manufacturing activities, invoicing to date under the contracts, communication from the research institutions and other companies of any actual costs incurred during the period that have not yet been invoiced, and the costs included in the contracts. Significant judgments and estimates may be made in determining the accrued balances at the end of any reporting period. Actual results could differ from the estimates made by the Company. The historical accrual estimates made by the Company have not been materially different from the actual costs. Patent Costs All patent‑related costs incurred in connection with filing and prosecuting patent applications are expensed as incurred due to the uncertainty about the recovery of the expenditure. Amounts incurred are classified as general and administrative expenses. Stock‑Based Compensation The Company measures stock options with service‑based vesting granted to employees, non‑employees, and directors based on the fair value on the date of grant using the Black‑Scholes option‑pricing model. The Company measures restricted common stock awards using the difference, if any, between the purchase price per share of the award and the fair value of the Company’s common stock at the date of grant. Compensation expense for employee awards is recognized over the requisite service period, which is generally the vesting period of the award. Compensation expense for non‑employee awards is recognized in the same manner as if the Company had paid cash in exchange for the goods or services, which is generally the vesting period of the award. The Company uses the straight‑line method to record the expense of awards with only service‑based vesting conditions. The Company has not granted awards with performance‑ or market‑based vesting conditions. The Company accounts for forfeitures of share‑based awards as they occur. The Company classifies stock‑based compensation expense in its condensed consolidated statements of operations and comprehensive loss in the same manner in which the award recipient’s payroll costs are classified or in which the award recipient’s service payments are classified. Comprehensive Loss Comprehensive loss includes net loss as well as other changes in stockholders’ equity (deficit) that result from transactions and economic events other than those with stockholders, which for the three and nine months ended September 30, 2020 consists of unrealized gains on marketable securities. 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 consolidated financial statements or in the Company’s tax returns. Deferred tax assets and liabilities are determined on the basis of the differences between the financial statements and tax basis of assets and liabilities using enacted tax rates in effect for the year in which the differences are expected to reverse. Changes in deferred tax assets and liabilities are recorded in the provision for income taxes. The Company assesses the likelihood that its deferred tax assets will be recovered from future taxable income and, to the extent it believes, based upon the weight of available evidence, that it is more likely than not that all or a portion of the deferred tax assets will not be realized, a valuation allowance is established through a charge to income tax expense. Potential for recovery of deferred tax assets is evaluated by estimating the future taxable profits expected and considering prudent and feasible tax planning strategies. The Company accounts for uncertainty in income taxes recognized in the consolidated financial statements by applying a two‑step process to determine the amount of tax benefit to be recognized. First, the tax position must be evaluated to determine the likelihood that it will be sustained upon external examination by the taxing authorities. If the tax position is deemed more‑likely‑than‑not to be sustained, the tax position is then assessed to determine the amount of benefit to recognize in the consolidated financial statements. The amount of the benefit that may be recognized is the largest amount that has a greater than 50% likelihood of being realized upon ultimate settlement. The provision for income taxes includes the effects of any resulting tax reserves, or unrecognized tax benefits, that are considered appropriate as well as the related net interest and penalties. The Company’s policy is to recognize interest and/or penalties related to income tax matters in income tax expense. The Company had no amounts accrued for interest and penalties on its condensed consolidated balance sheets at September 30, 2020 and December 31, 2019. Net Loss per Share The Company follows the two‑class method when computing net income (loss) per share as the Company has issued shares that meet the definition of participating securities. The two‑class method determines net income (loss) per share for each class of common and participating securities according to dividends declared or accumulated and participation rights in undistributed earnings. The two‑class method requires income (loss) available to common stockholders for the period to be allocated between common and participating securities based upon their respective rights to share in undistributed earnings as if all income (loss) for the period had been distributed. Basic net income (loss) per share attributable to common stockholders is computed by dividing net income (loss) attributable to common stockholders by the weighted‑average number of common shares outstanding for the period. Diluted net income (loss) per share attributable to common stockholders is computed by adjusting net income (loss) attributable to common stockholders to reallocate undistributed earnings based on the potential impact of dilutive securities. Diluted net income (loss) per share attributable to common stockholders is computed by dividing the diluted net income (loss) attributable to common stockholders by the weighted‑average number of common shares outstanding for the period, including potential dilutive common shares. For purposes of this calculation, the Company’s outstanding stock options, unvested restricted common stock, and convertible preferred stock are considered potential dilutive common shares. The Company’s participating securities contractually entitle the holders of such securities to participate in dividends but do not contractually require the holders of such securities to participate in losses of the Company. Accordingly, in periods in which the Company reports a net loss, such losses are not allocated to such participating securities. In periods in which the Company reports a net loss attributable to common stockholders, diluted net loss per share attributable to common stockholders is the same as basic net loss per share attributable to common stockholders, since dilutive common shares are not assumed to have been issued if their effect is anti‑dilutive. The Company reported a net loss attributable to common stockholders for the three and nine months ended September 30, 2020 and 2019. Recently Adopted Accounting Pronouncements In August 2018, the FASB issued ASU No. 2018‑13, Fair Value Measurement (Topic 820): Disclosure Requirements for Fair Value Measurement (“ASU 2018‑13”), which modifies the existing disclosure requirements for fair value measurements in Topic 820. The new disclosure requirements include disclosure related to changes in unrealized gains or losses included in other comprehensive income (loss) for recurring Level 3 fair value measurements held at the end of each reporting period and the explicit requirement to disclose the range and weighted‑average of significant unobservable inputs used for Level 3 fair value measurements. The other provisions of ASU 2018‑13 include eliminated and modified disclosure requirements. An entity is permitted to early adopt any removed or modified disclosures upon issuance of ASU 2018‑13 and delay adoption of the additional disclosures until their effective date. For all entities, this guidance is required to be adopted for annual periods beginning after December 15, 2019, including interim periods within those fiscal years. Early adoption is permitted. The Company adopted ASU 2018‑13 as of the required effective date of January 1, 2020, and the adoption did not have a material impact on the Company’s consolidated financial statements. Recently Issued Accounting Pronouncements From time to time, new accounting pronouncements are issued by the FASB or other standard‑setting bodies that the Company adopts as of the specified effective date. The Company qualifies as an “emerging growth company” as defined in the Jumpstart Our Business Startups Act of 2012 and has elected not to “opt out” of the extended transition related to complying with new or revised accounting standards, which means that when a standard is issued or revised and it has different application dates for public and non‑public companies, the Company can adopt the new or revised standard at the time non‑public companies adopt the new or revised standard and can do so until such time that the Company either (i) irrevocably elects to “opt out” of such extended transition period or (ii) no longer qualifies as an emerging growth company. The Company may choose to early adopt any new or revised accounting standards whenever such early adoption is permitted for non‑public companies. In June 2016, the FASB issued ASU No. 2016‑13, Financial Instruments—Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments (“ASU 2016‑13”), which requires the measurement and recognition of expected credit losses for financial assets held at amortized cost. ASU 2016‑13 replaces the existing incurred loss impairment model with an expected loss model. It also eliminates the concept of other‑than‑temporary impairment and requires credit losses related to available‑for‑sale debt securities to be recorded through an allowance for credit losses rather than as a reduction in the amortized cost basis of the securities. These changes may result in earlier recognition of credit losses. In November 2018, the FASB issued ASU No. 2018‑19, Codification Improvements to Topic 326, Financial Instruments—Credit Losses , which narrowed the scope and changed the effective date for non‑public entities for ASU 2016‑13. The FASB subsequently issued supplemental guidance within ASU No. 2019‑05, Financial Instruments—Credit Losses (Topic 326): Targeted Transition Relief (“ASU 2019‑05”). ASU 2019‑05 provides an option to irrevocably elect the fair value option for certain financial assets previously measured at amortized cost basis. For public entities that are SEC filers, excluding entities eligible to be smaller reporting companies, ASU 2016‑13 is effective for annual periods beginning after December 15, 2019, including interim periods within those fiscal years. For all other entities, ASU 2016‑13 is effective for annual periods beginning after December 15, 2022, including interim periods within those fiscal years. Early adoption is permitted. The Company is currently evaluating the impact that the adoption of ASU 2016‑13 will have on its consolidated financial statements. |
Fair Value Measurements
Fair Value Measurements | 9 Months Ended |
Sep. 30, 2020 | |
Fair Value Measurements | |
Fair Value Measurements | 3. Fair Value Measurements The Company’s financial assets measured at fair value on a recurring basis by level within the fair value hierarchy at September 30, 2020 and December 31, 2019 are summarized as follows (in thousands): Fair Value Measurements at September 30, 2020 Using: Level 1 Level 2 Level 3 Total Assets: Cash equivalents: Money market funds $ 177,940 $ — $ — $ 177,940 Restricted cash: Money market funds 1,292 — — 1,292 Marketable securities: U.S. Treasury notes — 141,452 — 141,452 $ 179,232 $ 141,452 $ — $ 320,684 Fair Value Measurements at December 31, 2019 Using: Level 1 Level 2 Level 3 Total Assets: Cash equivalents: Money market funds $ 24,281 $ — $ — $ 24,281 Restricted cash: Money market funds 1,292 — — 1,292 $ 25,573 $ — $ — $ 25,573 U.S. government money market funds were valued by the Company based on quoted market prices, which represent a Level 1 measurement within the fair value hierarchy. As of September 30, 2020, the Company’s marketable securities consisted of U.S. Treasury notes, which were valued based on Level 2 inputs. In determining the fair value of its U.S. Treasury notes, the Company relied on quoted prices for similar securities in active markets or other inputs that are observable or can be corroborated by observable market data. During the three and nine months ended September 30, 2020 and 2019 there were no transfers between Level 1, Level 2, and Level 3. |
Marketable Securities
Marketable Securities | 9 Months Ended |
Sep. 30, 2020 | |
Investments, Debt and Equity Securities [Abstract] | |
Marketable securities | 4. Marketable Securities As of September 30, 2020, the fair value of available-for-sale marketable debt securities by type of security was as follows (in thousands): September 30, 2020 Gross Gross Amortized Unrealized Unrealized Fair Cost Gain Loss Value Assets Marketable securities: U.S. Treasury notes $ 141,444 $ 8 $ — $ 141,452 $ 141,444 $ 8 $ — $ 141,452 All available-for-sale securities have contractual maturities of less than one year. At December 31, 2019, the Company did not hold any marketable securities. |
Property and Equipment, Net
Property and Equipment, Net | 9 Months Ended |
Sep. 30, 2020 | |
Property and Equipment, Net | |
Property and Equipment, Net | 5. Property and Equipment, Net Property and equipment, net consisted of the following (in thousands): September 30, 2020 December 31, 2019 Laboratory equipment $ 4,623 $ 2,536 Furniture and fixtures 461 358 Leasehold improvements 8,915 8,915 Construction in progress 495 77 14,494 11,886 Less: Accumulated depreciation and amortization (1,827) (394) $ 12,667 $ 11,492 Depreciation and amortization expense for the three and nine months ended September 30, 2020 was approximately $0.5 million and $1.4 million, respectively. Depreciation and amortization expense for the three and nine months ended September 30, 2019 was approximately $0.1 million and $0.2 million, respectively. |
Accrued Expenses and Other Curr
Accrued Expenses and Other Current Liabilities | 9 Months Ended |
Sep. 30, 2020 | |
Accrued Expenses and Other Current Liabilities | |
Accrued Expenses and Other Current Liabilities | 6. Accrued Expenses and Other Current Liabilities Accrued expenses and other current liabilities consisted of the following (in thousands): September 30, 2020 December 31, 2019 Accrued external research, development, and manufacturing expenses $ 3,156 $ 2,605 Accrued employee compensation and benefits 1,581 1,157 Accrued professional fees 1,497 67 Payments due for leasehold improvements — 756 Other 496 377 $ 6,730 $ 4,962 |
Convertible Preferred Stock
Convertible Preferred Stock | 9 Months Ended |
Sep. 30, 2020 | |
Convertible Preferred Stock | |
Convertible Preferred Stock | 7. Convertible Preferred Stock The Company issued Series Seed convertible preferred stock (the “Series Seed preferred stock”), Series Seed 1 convertible preferred stock (the “Series Seed 1 preferred stock”), Series A convertible preferred stock (the “Series A preferred stock”), and Series B convertible preferred stock (the “Series B preferred stock,” and collectively with the Series Seed preferred stock, the Series Seed 1 preferred stock, and the Series A preferred stock, the “Preferred Stock”). In February 2020, the Company issued and sold 221,399,223 shares of Series B preferred stock, at a price of $0.47455 per share, for gross proceeds of $105.1 million. The Company incurred issuance costs in connection with this transaction of $0.2 million. Upon the closing of the Company’s initial public offering on June 30, 2020, all outstanding shares of Preferred Stock automatically converted into 18,969,672 shares of common stock. |
Stockholders' Equity
Stockholders' Equity | 9 Months Ended |
Sep. 30, 2020 | |
Stockholders' Equity | |
Stockholders' Equity | 8. Stockholders’ Equity Common Stock As of September 30, 2020 and December 31, 2019, the Company’s certificate of incorporation, as amended and restated, authorized the Company to issue 200,000,000 and 235,000,000 shares, respectively, of common stock $0.0001 par value per share. On June 18, 2020, the Company effected a one-for-21.073 reverse stock split of its issued and outstanding shares of common stock and a proportional adjustment to the existing conversion ratios for each series of the Company’s convertible preferred stock. Accordingly, all share and per share amounts for all periods presented in the accompanying condensed consolidated financial statements and notes thereto have been adjusted retroactively, where applicable, to reflect this reverse stock split and adjustment of the preferred stock conversion ratios. On June 30, 2020, the Company completed its initial public offering of its common stock and issued and sold 14,375,000 shares of common stock, at a public offering price of $17.00 per share, for gross proceeds of $244.4 million, or net proceeds of $223.8 million after deducting underwriting discounts, commissions, and offering expenses. Preferred Stock As of September 30, 2020, the Company’s certificate of incorporation, as amended and restated, authorized the Company to issue 5,000,000 shares of preferred stock, $0.0001 par value per share. As of September 30, 2020, no shares of preferred stock were issued or outstanding. . |
Stock_Based Compensation
Stock‑Based Compensation | 9 Months Ended |
Sep. 30, 2020 | |
Stock‑Based Compensation | |
Stock-Based Compensation | 9. Stock‑Based Compensation 2016 Stock Plan The Company’s 2016 Stock Plan (the “2016 Plan”) provides for the Company to grant incentive stock options or non‑qualified stock options, restricted stock, restricted stock units, and other equity awards to employees, directors, and consultants of the Company. The 2016 Plan is administered by the board of directors or, at the discretion of the board of directors, by a committee of the board of directors. The board of directors may also delegate to one or more officers of the Company the power to grant awards to employees and certain officers of the Company. The exercise prices, vesting, and other restrictions are determined at the discretion of the board of directors, or its committee or any such officer if so delegated. Stock options granted under the 2016 Plan with service‑based vesting conditions generally vest over four years and expire after ten years. As of December 31, 2019, the total number of shares of common stock that were issuable under the 2016 Plan was 1,508,669 shares, of which 363,350 shares remained available for future issuance as of December 31, 2019. During the nine months ended September 30, 2020 the Company increased the number of shares of common stock authorized for issuance under the 2016 Plan from 1,508,669 shares to 3,722,685 shares. As of September 30, 2020, no shares remained available for future issuance under the 2016 Plan. 2020 Stock Plan On May 28, 2020, the Company's board of directors adopted, and on June 17, 2020 its stockholders approved, the 2020 Stock Plan (the "2020 Plan"). The 2020 Plan provides for the grant of incentive stock options, nonstatutory stock options, stock appreciation rights, restricted stock awards, restricted stock units, and other stock-based awards. The number of shares initially reserved for issuance under the 2020 Plan is the sum of 4,294,594, plus the number of shares (up to 3,622,691 shares) equal to the sum of (i) the number of shares remaining available for issuance under the 2016 Plan upon the effectiveness of the 2020 Plan and (ii) the number of shares of common stock subject to outstanding awards granted under the 2016 Plan that expire, terminate or are otherwise surrendered, cancelled, forfeited or repurchased by the Company at their original issuance price pursuant to a contractual repurchase right. The number of shares of common stock that may be issued under the 2020 Plan will automatically increase on the first day of each fiscal year, beginning with the fiscal year ending December 31, 2021 and continuing for each fiscal year until, and including, the fiscal year ending December 31, 2030, equal to the lowest of (i) 2,728,610 shares, (ii) 4% of the number of shares of common stock outstanding on such date, and (iii) an amount determined by the Company's board of directors. The shares of common stock underlying any awards that are forfeited, cancelled, held back upon exercise or settlement of an award to satisfy the exercise price or tax withholding, repurchased or are otherwise terminated by the Company under the 2020 Plan will be added back to the shares of common stock available for issuance under the 2020 Plan. As of September 30, 2020, the total number of shares of common stock available for issuance under the 2020 Plan is 5,035,409 shares. 2020 Employee Stock Purchase Plan On May 28, 2020, the Company's board of directors adopted, and on June 17, 2020 its stockholders approved, the 2020 Employee Stock Purchase Plan (the "2020 ESPP"). A total of 360,651 shares of common stock were initially reserved for issuance under this plan. The number of shares of common stock that may be issued under the 2020 ESPP will automatically increase on the first day of each fiscal year, beginning with the fiscal year commencing on January 1, 2021 and continuing for each fiscal year until, and including, the fiscal year commencing on January 1, 2031, equal to the lowest of (i) 640,630 shares, (ii) 1% of the number of shares of common stock outstanding on such date, and (iii) an amount determined by the Company's board of directors. Stock Option Valuation The fair value of stock option grants is estimated using the Black‑Scholes option‑pricing model. The Company historically has been a private company and 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 such time as it has adequate historical data regarding the volatility of its own traded stock price. For options with service‑based vesting conditions, the expected term of the Company’s stock options has been determined utilizing the “simplified” method for awards that qualify as “plain‑vanilla” options. The risk‑free interest rate is determined by reference to the U.S. Treasury yield curve in effect at the time of grant of the award for time periods approximately equal to the expected term of the award. Expected dividend yield is based on the fact that the Company has never paid cash dividends and does not expect to pay any cash dividends in the foreseeable future. Stock Options The following table summarizes the Company’s stock option activity since December 31, 2019: Weighted‑ Weighted‑ Average Average Aggregate Number of Exercise Contractual Intrinsic Shares Price Term Value (in years) (in thousands) Outstanding as of December 31, 2019 961,751 $ 1.69 9.5 $ 524 Granted 1,989,654 8.67 Exercised (32,511) 1.27 Forfeited (57,766) 3.32 Outstanding as of September 30, 2020 2,861,128 $ 6.54 $ 46,726 Vested and expected to vest as of September 30, 2020 2,861,128 $ 6.54 $ 46,726 Options exercisable as of September 30, 2020 349,736 $ 3.58 $ 6,745 The weighted‑average grant‑date fair value of stock options granted during the three months ended September 30, 2020 and 2019 was $17.81 per share and $1.22 per share respectively, and during the nine months ended September 30, 2020 and 2019 was $7.08 per share and $1.10 per share, respectively. Early Exercise of Stock Options into Restricted Stock Certain option grants permit option holders to elect to exercise unvested options in exchange for unvested common stock. The options that are exercised prior to vesting will continue to vest according to the respective option agreement, and such unvested shares are subject to repurchase by the Company at the optionee’s original exercise price in the event the optionee’s service with the Company voluntarily or involuntarily terminates. A summary of the Company’s unvested common stock from option early exercises that is subject to repurchase by the Company is as follows: Shares Unvested restricted common stock as of December 31, 2019 102,896 Vested (48,615) Unvested restricted common stock as of September 30, 2020 54,281 Proceeds from the early exercise of options are recorded as a liability within accrued expenses and other current liabilities on the condensed consolidated balance sheet. The liability for unvested common stock subject to repurchase is then reclassified to additional paid‑in capital as the Company’s repurchase right lapses. The shares purchased by the employees and directors pursuant to the early exercise of stock options are not deemed, for accounting purposes, to be outstanding until those shares have vested. As of September 30, 2020, the liability related to the payments for unvested shares from early‑exercised options was less than $0.1 million. As of December 31, 2019 the liability related to the payments for unvested shares from early‑exercised options was $0.1 million. Restricted Common Stock Awards The Company has both (i) granted restricted stock awards, with the recipient not paying for the shares of common stock, and (ii) issued and sold restricted stock, with the recipient purchasing the common stock at its fair value per share. In both circumstances, the restricted shares of common stock have service‑based vesting conditions and unvested shares are either subject to forfeiture by the employee or subject to repurchase by the Company, at the lesser of holder’s original purchase price or fair value, in the event the holder’s service with the Company voluntarily or involuntarily terminates. Service‑based restricted stock awards generally vest over four years. Proceeds from the issuance and sale of restricted common stock are recorded as a liability within accrued expenses and other current liabilities on the condensed consolidated balance sheet. The liability for unvested common stock subject to repurchase is then reclassified to additional paid‑in capital as the Company’s repurchase right lapses. Shares of restricted common stock granted or sold to employees and directors are not deemed, for accounting purposes, to be outstanding until those shares have vested. The Company did not grant or sell restricted common stock awards during 2019 or the nine months ended September 30, 2020. As of September 30, 2020 and December 31, 2019, the liability related to the payments received for shares of unvested restricted stock was less than $0.1 million. The following table summarizes the Company’s restricted common stock award activity for the nine months ended September 30, 2020: Weighted‑ Average Grant‑Date Shares Fair Value Unvested restricted common stock as of December 31, 2019 209,742 $ 0.0443 Vested (87,986) 0.0400 Unvested restricted common stock as of September 30, 2020 121,756 $ 0.0500 The total fair value of restricted common stock vested during the three and nine months ended September 30, 2020 was less than $0.1 million. Stock‑Based Compensation The Company records compensation cost for all share‑based payment arrangements, including employee, director, and consultant stock options and restricted stock. The Company recorded stock‑based compensation expense in the following expense categories of its consolidated statements of operations and comprehensive loss (in thousands): Three Months Ended Nine Months Ended September 30, September 30, 2020 2019 2020 2019 Research and development expenses $ 399 $ 12 $ 617 $ 30 General and administrative expenses 1,677 9 2,060 27 $ 2,076 $ 21 $ 2,677 $ 57 In connection with the termination of an employee in July 2020, the Company accelerated the vesting of 1,541 shares of restricted stock and 60,948 unvested options, of which 22,684 shares are held by such employee. The modification of these awards resulted in the recognition of $1.1 million of stock-based compensation expense during the three and nine months ended September 30, 2020, which was classified as general and administrative expenses. As of September 30, 2020, there was $13.6 million of unrecognized compensation cost related to unvested stock options, which is expected to be recognized over a weighted-average period of 2.25 years. |
License Agreements
License Agreements | 9 Months Ended |
Sep. 30, 2020 | |
License Agreements | |
License Agreements | 10. License Agreements License Agreement with Massachusetts Eye and Ear In October 2017, the Company entered into a license agreement with Massachusetts Eye and Ear Infirmary and the Schepens Eye Research Institute, Inc. (collectively referred to as “MEE”) (the “MEE License”). Under the MEE License, the Company received an exclusive, non‑transferable, sublicensable, worldwide, royalty‑bearing license to certain patent rights and know‑how, including rights related to adeno-associated virus, or AAV, ancestral technology, including AAVAnc80, to use, research, develop, manufacture, and commercialize licensed products in the treatment, diagnosis, and prevention of any and all balance disorders, including hearing disorders of the inner ear, in each case, with a total prevalence in the United States of less than 3,000 patients, and an exclusive, non‑transferable, sublicensable right and license under MEE’s rights, title, and interest in certain patents co‑owned by MEE and Children’s Medical Center Corporation to use, research, develop, manufacture, and commercialize licensed products. The Company is obligated to use commercially reasonable efforts to develop and commercialize the MEE licensed products, including filing an investigational new drug application (“IND”) in the United States or investigational medicinal product dossier (“IMPD”) in any country in the European Union or an equivalent application in any country within 18 months of completion of specified toxicology studies for a licensed product. Upon entering into the MEE License in 2017, the Company issued to MEE shares of the Company’s common stock then having a fair value of $0.1 million. The Company is obligated to make aggregate milestone payments to MEE of up to $17.7 million upon the achievement of specified development, regulatory, and sales milestones. The Company is also obligated to pay tiered royalties of a mid to high single‑digit percentage based on annual net sales of licensed products by the Company and any of its affiliates and sublicensees. Royalties will be paid by the Company on a licensed product‑by‑licensed product and country‑by‑country basis beginning after the first commercial sale of an MEE licensed product and lasting until the later of (i) the expiration of the last valid claim in the licensed patents or (ii) ten years after the first commercial sale of such MEE licensed product (the “MEE Royalty Term”). The MEE License remains in effect until the last expiration date of the last to expire MEE Royalty Term, unless terminated earlier. The Company has the right to terminate the MEE License at will, with or without cause, by 90 days’ advance written notice to MEE or upon MEE’s material breach of the MEE License, provided that MEE does not cure such material breach within a specified period. MEE has the right to terminate the MEE License in its entirety if (i) the Company fails to make any payment due within a specified period after MEE notifies the Company of such failure, (ii) the Company or its affiliates challenge the validity of the licensed patent rights, (iii) the Company fails to maintain required insurance, or (iv) the Company becomes insolvent or bankrupt. MEE also has the right to terminate the Company’s rights to specific intellectual property rights it has licensed to the Company under the MEE License if the Company materially breaches certain diligence obligations and does not cure within a specified period after written notice from MEE. During the three and nine months ended September 30, 2020 and 2019 the Company did not make any payments to MEE or recognize any research and development expenses under the MEE License. Sublicense Agreement with Lonza Houston, Inc. In October 2017, the Company entered into a sublicense agreement with Lonza Houston, Inc. (“Lonza”), as amended in December 2018 (the “Lonza Sublicense”). Under the agreement, the Company received an exclusive, non‑transferable, sublicensable, worldwide, royalty‑bearing sublicense to certain patent rights and know‑how related to AAV ancestral technology, including AAVAnc80, to use, research, develop, manufacture, and commercialize licensed products for the treatment, diagnosis, and prevention of any and all balance disorders or diseases pertaining to the inner ear and/or any and all hearing diseases or disorders, including hearing disorders of the inner ear, but excluding all such disorders or diseases with a total prevalence in the United States of less than 3,000 patients. The Company is obligated to use commercially reasonable efforts to develop and commercialize the Lonza sublicensed products, including filing an IND in the United States or IMPD in any country in the European Union or an equivalent application in any country within 18 months of completion of specified toxicology studies for a licensed product. Upon entering into the Lonza Sublicense in 2017, the Company issued to Lonza shares of the Company’s common stock then having a fair value of $0.1 million. The Company is obligated to make aggregate milestone payments to Lonza of up to $18.5 million upon the achievement of specified development, regulatory, and sales milestones. The Company is also obligated to pay tiered royalties of a mid to high single‑digit percentage based on annual net sales of licensed products by the Company and any of its affiliates and sublicensees as denoted in the Lonza Sublicense. Royalties will be paid by the Company on a licensed product‑by‑licensed product and country‑by‑country basis beginning after the first commercial sale of a Lonza sublicensed product and lasting until the later of (i) the expiration of the last valid claim in the patent or (ii) ten years after the first commercial sale of such Lonza sublicensed product (the “Lonza Royalty Term”). The Lonza Sublicense remains in effect until the last expiration date of the last to expire Lonza Royalty Term, unless terminated earlier. The Company has the right to terminate the Lonza Sublicense at will, with or without cause, by 90 days’ advance written notice to Lonza or upon Lonza’s material breach of the Lonza Sublicense, provided that Lonza does not cure such material breach within a specified period. Lonza has the right to terminate the Lonza Sublicense in its entirety if (i) the Company fails to make any payment due within a specified period after Lonza notifies the Company of such failure, (ii) the Company or its affiliates challenge the validity of the sublicensed patent rights, (iii) the Company fails to maintain required insurance, or (iv) the Company becomes insolvent or bankrupt. Lonza also has the right to terminate the Company’s rights to specific intellectual property rights it has sublicensed to the Company under the Lonza Sublicense if the Company materially breaches certain diligence obligations and does not cure within a specified period after written notice from Lonza. During the three and nine months ended September 30, 2020 and 2019 the Company did not make any payments to Lonza or recognize any research and development expenses under the Lonza Sublicense. |
Commitments and Contingencies
Commitments and Contingencies | 9 Months Ended |
Sep. 30, 2020 | |
Commitments and Contingencies | |
Commitments and Contingencies | 11. Commitments and Contingencies 401(k) Plan The Company has a defined‑contribution plan under Section 401(k) of the Internal Revenue Code of 1986 (the “401(k) Plan”). The 401(k) Plan covers all employees who meet defined minimum age and service requirements and allows participants to defer a portion of their annual compensation on a pre‑tax basis. As currently established, the Company is not required to make, and to date has not made, any contributions to the 401(k) Plan. Indemnification Agreements In the ordinary course of business, the Company may provide indemnification of varying scope and terms to vendors, lessors, CROs, business partners, and other parties with respect to certain matters including, but not limited to, losses arising out of breach of such agreements or from intellectual property infringement claims made by third parties. In addition, the Company has entered into indemnification agreements with members of its board of directors and certain of its executive officers that will require the Company, among other things, to indemnify them against certain liabilities that may arise by reason of their status or service as directors or officers. The maximum potential amount of future payments the Company could be required to make under these indemnification agreements is, in many cases, unlimited. The Company has not incurred any material costs as a result of such indemnifications and is not currently aware of any indemnification claims. Legal Proceedings The Company is not currently party to any material legal proceedings. At each reporting date, the Company evaluates whether or not a potential loss amount or a potential range of loss is probable and reasonably estimable under the provisions of the authoritative guidance that addresses accounting for contingencies. The Company expenses as incurred the costs related to such legal proceedings. |
Net Loss per Share
Net Loss per Share | 9 Months Ended |
Sep. 30, 2020 | |
Net Loss per Share | |
Net Loss per Share | 12. Net Loss per Share Net Loss per Share Basic and diluted net loss per share attributable to common stockholders was calculated as follows (in thousands, except share and per share amounts): Three Months Ended September 30, Nine Months Ended September 30, 2020 2019 2020 2019 Numerator: Net loss attributable to common stockholders $ (13,089) $ (8,210) $ (36,052) $ (16,123) Denominator: Weighted‑average common shares outstanding, basic and diluted 15,334,241 621,581 11,991,870 586,728 Net loss per share attributable to common stockholders, basic and diluted $ (0.85) $ (13.21) $ (3.01) $ (27.48) The Company’s potential dilutive securities have been excluded from the computation of diluted net loss per share as the effect would be to reduce the net loss per share. Therefore, the weighted‑average number of common shares outstanding used to calculate both basic and diluted net loss per share attributable to common stockholders is the same. The Company excluded the following potential common shares, presented based on amounts outstanding at each period end, from the computation of diluted net loss per share attributable to common stockholders for the periods indicated because including them would have had an anti‑dilutive effect: September 30, 2020 2019 Convertible preferred stock (as converted to common stock) — 18,969,672 Unvested restricted common stock 176,037 295,360 Stock options to purchase common stock 2,861,128 398,726 3,037,165 19,663,758 |
Subsequent Events
Subsequent Events | 9 Months Ended |
Sep. 30, 2020 | |
Subsequent Events | |
Subsequent Events | 13. Subsequent Events Grant of stock options under the 2020 Plan The Company has evaluated subsequent events for recognition, remeasurement and disclosure purposes through the date which the unaudited consolidated financial statements were issued. On October 1, 2020, the Company granted options for the purchase of an aggregate of 642,780 shares of common stock, at an exercise price of $22.45 per share. The aggregate grant-date fair value of these options was $11.3 million, which is expected to be recognized as stock-based compensation expense over a period of approximately four years. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 9 Months Ended |
Sep. 30, 2020 | |
Summary of Significant Accounting Policies | |
Use of Estimates | Use of Estimates The preparation of the Company’s consolidated financial statements in conformity with 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 revenue and expenses during the reporting periods. Significant estimates and assumptions reflected in these condensed consolidated financial statements include, but are not limited to, the accrual of research and development expenses and the valuations of common stock, stock options, and a preferred stock tranche liability. 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. On an ongoing basis, management evaluates its estimates, as there are changes in circumstances, facts, and experience. Changes in estimates are recorded in the period in which they become known. Actual results may differ from those estimates or assumptions. |
Unaudited Interim Financial Information | Unaudited Interim Financial Information The accompanying condensed consolidated balance sheet as of September 30, 2020, the condensed consolidated statements of operations and comprehensive loss for the three and nine months ended September 30, 2020 and 2019, the condensed consolidated statement of cash flows for the nine months ended September 30, 2020 and 2019 and the condensed consolidated statements of convertible preferred stock and stockholders’ equity (deficit) for the three and nine months ended September 30, 2020 and 2019 are unaudited. The unaudited interim condensed consolidated financial statements have been prepared on the same basis as the audited annual consolidated financial statements and, in the opinion of management, reflect all adjustments, which include only normal recurring adjustments, necessary for the fair statement of the Company’s financial position as of September 30, 2020 and necessary for the fair statement of the Company’s financial position as of September 30, 2020 and the results of its operations for the three and nine months ended September 30, 2020 and 2019 and its cash flows for the nine months ended September 30, 2020 and 2019. The financial data and other information disclosed in these notes related to the three and nine months ended September 30, 2020 and 2019 are also unaudited. The results for the three and nine months ended September 30, 2020 are not necessarily indicative of results to be expected for the year ending December 31, 2020, any other interim periods, or any future year or period. |
Concentrations of Credit Risk and of Significant Suppliers | Concentrations of Credit Risk and of Significant Suppliers Financial instruments that potentially expose the Company to concentrations of credit risk consist primarily of cash, cash equivalents and marketable securities. The Company maintains its cash and cash equivalents at one accredited financial institution. The Company’s cash equivalents as of September 30, 2020 and December 31, 2019 consisted of U.S. government money market funds. Marketable securities consist of U.S. Treasury notes with maturities of less than twelve months. The Company does not believe that it is subject to unusual credit risk beyond the normal credit risk associated with commercial banking relationships. The Company is dependent on third‑party vendors for its product candidates. In particular, the Company relies, and expects to continue to rely, on a small number of vendors to manufacture materials and components required for the production of its product candidates. These programs could be adversely affected by a significant interruption in the manufacturing process. |
Cash Equivalents | Cash Equivalents The Company considers all highly liquid investments with a remaining maturity when purchased of three months or less to be cash equivalents. |
Restricted Cash | Restricted Cash In connection with the Company’s lease agreement entered into in December 2018, the Company is required to maintain a letter of credit of $1.3 million for the benefit of the landlord. As of September 30, 2020 and December 31, 2019, this amount was classified as restricted cash (non‑current) in the condensed consolidated balance sheets. The Company is required to maintain a separate cash balance of less than $0.1 million pledged as collateral for a credit card account. As of September 30, 2020 and December 31, 2019, this amount was classified as restricted cash (non‑current) on the condensed consolidated balance sheets. |
Marketable Securities | Marketable Securities Marketable securities represent holdings of available-for-sale marketable debt securities in accordance with the Company’s investment policy. Marketable securities that mature within one-year from the balance sheet date are classified as current in the condensed consolidated balance sheets. Investments in marketable securities are recorded at fair value, with any unrealized gains and losses reported within accumulated other comprehensive income (loss) as a separate component of stockholders’ equity (deficit) until realized or until a determination is made that an other-than-temporary decline in market value has occurred. The amortized cost of debt securities is adjusted for amortization of premiums and accretion of discounts to maturity. Such amortization and accretion, together with interest on securities sold, is determined based on the specific identification method and any realized gains or losses on the sale of investments are reflected as a component of other income (expense). |
Fair Value Measurements | Fair Value Measurements Certain assets and liabilities of the Company are carried at fair value under 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. Financial assets and financial 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—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 Company’s cash equivalents, marketable securities and preferred stock tranche liability are carried at fair value, determined according to the fair value hierarchy described above (see Note 3). The carrying values of the Company’s accounts payable and accrued expenses approximate their fair values due to the short‑term nature of these liabilities. |
Property and Equipment | Property and Equipment Property and equipment are stated at cost less accumulated depreciation and amortization. Depreciation and amortization expense is recognized using the straight‑line method over the estimated useful life of each asset as follows: Estimated Useful Life Laboratory equipment 5 years Furniture and fixtures 4 years Leasehold improvements Shorter of term of lease or 15 years Costs for capital assets not yet placed into service are capitalized as construction‑in‑progress and depreciated once placed into service. Upon retirement or sale, the cost of assets disposed of and the related accumulated depreciation and amortization are removed from the accounts and any resulting gain or loss is included in loss from operations. Expenditures for repairs and maintenance that do not improve or extend the life of the respective assets are charged to expense as incurred. |
Classification and Accretion of Convertible Preferred Stock | Classification and Accretion of Convertible Preferred Stock The Company’s convertible preferred stock was classified outside of stockholders’ equity (deficit) on the condensed consolidated balance sheets because the holders of such shares had liquidation rights in the event of a deemed liquidation that, in certain situations, was not solely within the control of the Company and would require the redemption of the then‑outstanding convertible preferred stock. The Company’s Series Seed, Series Seed 1, Series A, and Series B convertible preferred stock were not redeemable, except in the event of a deemed liquidation. Because the occurrence of a deemed liquidation event was not currently probable, the carrying values of the convertible preferred stock were not being accreted to their redemption values. Subsequent adjustments to the carrying values to the convertible preferred stock would be made only when a deemed liquidation event became probable. Upon the closing of the Company’s initial public offering on June 30, 2020, all outstanding shares of convertible preferred stock automatically converted into 18,969,672 shares of common stock. |
Impairment of Long-Lived Assets | Impairment of Long‑Lived Assets Long‑lived assets consist of property and equipment. Long‑lived assets to be held and used 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 group for recoverability, the Company compares forecasts of undiscounted cash flows expected to result from the use and eventual disposition of the long‑lived asset group to its carrying value. An impairment loss would be recognized in loss from operations when estimated undiscounted future cash flows expected to result from the use of an asset group are less than its carrying amount. Impairment is measured based on the excess of the carrying value of the related assets over the fair value of such assets. The Company did not record any impairment losses on long‑lived assets during the three and nine months ended September 30, 2020 and 2019. |
Segment Information | Segment Information The Company manages its operations as a single segment for the purposes of assessing performance and making operating decisions. The Company’s focus is on developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals with hearing loss. All of the Company’s long‑lived assets are located in the United States. |
Government Contracts | Government Contracts The Company has received funding from government contracts that reimburse the Company for certain allowable costs for funded projects. For contracts with government agencies, when the Company has concluded that it is the principal in conducting the research and development expenses but where the funding arrangement is not considered core to the Company’s ongoing operations, the Company classifies the recognized funding received as other income in its condensed consolidated statement of operations and comprehensive loss. |
Research and Development Costs | Research and Development Costs Costs for research and development activities are expensed in the period in which they are incurred. Research and development expenses consist of costs incurred in performing research and development activities, including salaries and bonuses, stock‑based compensation, employee benefits, facilities costs, laboratory supplies, depreciation and amortization, manufacturing expenses, and external costs of vendors engaged to conduct research and preclinical development activities as well as the cost of licensing technology. Upfront payments under license agreements are expensed upon receipt of the license, and annual maintenance fees under license agreements are expensed in the period in which they are incurred. Milestone payments under license agreements are accrued, with a corresponding expense being recognized, in the period in which the milestone is determined to be probable of achievement and the related amount is reasonably estimable. Nonrefundable advance payments for goods or services to be received in the future for use in research and development activities are recorded as prepaid expenses. The prepaid amounts are expensed as the related goods are delivered or the services are performed. |
Research, Development, and Manufacturing Contract Costs and Accruals | Research, Development, and Manufacturing Contract Costs and Accruals The Company has entered into various research, development, and manufacturing contracts with research institutions and other companies. These agreements are generally cancelable, and related costs are recorded as research and development expenses as incurred. The Company records accruals for estimated ongoing research and development costs. When billing terms under these contracts do not coincide with the timing of when the work is performed, the Company is required to make estimates of outstanding obligations to those third parties as of period end. Any accrual estimates are based on a number of factors, including the Company’s knowledge of the progress towards completion of the research, development, and manufacturing activities, invoicing to date under the contracts, communication from the research institutions and other companies of any actual costs incurred during the period that have not yet been invoiced, and the costs included in the contracts. Significant judgments and estimates may be made in determining the accrued balances at the end of any reporting period. Actual results could differ from the estimates made by the Company. The historical accrual estimates made by the Company have not been materially different from the actual costs. |
Patent Costs | Patent Costs All patent‑related costs incurred in connection with filing and prosecuting patent applications are expensed as incurred due to the uncertainty about the recovery of the expenditure. Amounts incurred are classified as general and administrative expenses. |
Stock-Based Compensation | Stock‑Based Compensation The Company measures stock options with service‑based vesting granted to employees, non‑employees, and directors based on the fair value on the date of grant using the Black‑Scholes option‑pricing model. The Company measures restricted common stock awards using the difference, if any, between the purchase price per share of the award and the fair value of the Company’s common stock at the date of grant. Compensation expense for employee awards is recognized over the requisite service period, which is generally the vesting period of the award. Compensation expense for non‑employee awards is recognized in the same manner as if the Company had paid cash in exchange for the goods or services, which is generally the vesting period of the award. The Company uses the straight‑line method to record the expense of awards with only service‑based vesting conditions. The Company has not granted awards with performance‑ or market‑based vesting conditions. The Company accounts for forfeitures of share‑based awards as they occur. The Company classifies stock‑based compensation expense in its condensed consolidated statements of operations and comprehensive loss in the same manner in which the award recipient’s payroll costs are classified or in which the award recipient’s service payments are classified. |
Comprehensive Loss | Comprehensive Loss Comprehensive loss includes net loss as well as other changes in stockholders’ equity (deficit) that result from transactions and economic events other than those with stockholders, which for the three and nine months ended September 30, 2020 consists of unrealized gains on marketable securities. |
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 consolidated financial statements or in the Company’s tax returns. Deferred tax assets and liabilities are determined on the basis of the differences between the financial statements and tax basis of assets and liabilities using enacted tax rates in effect for the year in which the differences are expected to reverse. Changes in deferred tax assets and liabilities are recorded in the provision for income taxes. The Company assesses the likelihood that its deferred tax assets will be recovered from future taxable income and, to the extent it believes, based upon the weight of available evidence, that it is more likely than not that all or a portion of the deferred tax assets will not be realized, a valuation allowance is established through a charge to income tax expense. Potential for recovery of deferred tax assets is evaluated by estimating the future taxable profits expected and considering prudent and feasible tax planning strategies. The Company accounts for uncertainty in income taxes recognized in the consolidated financial statements by applying a two‑step process to determine the amount of tax benefit to be recognized. First, the tax position must be evaluated to determine the likelihood that it will be sustained upon external examination by the taxing authorities. If the tax position is deemed more‑likely‑than‑not to be sustained, the tax position is then assessed to determine the amount of benefit to recognize in the consolidated financial statements. The amount of the benefit that may be recognized is the largest amount that has a greater than 50% likelihood of being realized upon ultimate settlement. The provision for income taxes includes the effects of any resulting tax reserves, or unrecognized tax benefits, that are considered appropriate as well as the related net interest and penalties. The Company’s policy is to recognize interest and/or penalties related to income tax matters in income tax expense. The Company had no amounts accrued for interest and penalties on its condensed consolidated balance sheets at September 30, 2020 and December 31, 2019. |
Net Loss per Share | Net Loss per Share The Company follows the two‑class method when computing net income (loss) per share as the Company has issued shares that meet the definition of participating securities. The two‑class method determines net income (loss) per share for each class of common and participating securities according to dividends declared or accumulated and participation rights in undistributed earnings. The two‑class method requires income (loss) available to common stockholders for the period to be allocated between common and participating securities based upon their respective rights to share in undistributed earnings as if all income (loss) for the period had been distributed. Basic net income (loss) per share attributable to common stockholders is computed by dividing net income (loss) attributable to common stockholders by the weighted‑average number of common shares outstanding for the period. Diluted net income (loss) per share attributable to common stockholders is computed by adjusting net income (loss) attributable to common stockholders to reallocate undistributed earnings based on the potential impact of dilutive securities. Diluted net income (loss) per share attributable to common stockholders is computed by dividing the diluted net income (loss) attributable to common stockholders by the weighted‑average number of common shares outstanding for the period, including potential dilutive common shares. For purposes of this calculation, the Company’s outstanding stock options, unvested restricted common stock, and convertible preferred stock are considered potential dilutive common shares. The Company’s participating securities contractually entitle the holders of such securities to participate in dividends but do not contractually require the holders of such securities to participate in losses of the Company. Accordingly, in periods in which the Company reports a net loss, such losses are not allocated to such participating securities. In periods in which the Company reports a net loss attributable to common stockholders, diluted net loss per share attributable to common stockholders is the same as basic net loss per share attributable to common stockholders, since dilutive common shares are not assumed to have been issued if their effect is anti‑dilutive. The Company reported a net loss attributable to common stockholders for the three and nine months ended September 30, 2020 and 2019. |
Recently Adopted and Issued Accounting Pronouncements | Recently Adopted Accounting Pronouncements In August 2018, the FASB issued ASU No. 2018‑13, Fair Value Measurement (Topic 820): Disclosure Requirements for Fair Value Measurement (“ASU 2018‑13”), which modifies the existing disclosure requirements for fair value measurements in Topic 820. The new disclosure requirements include disclosure related to changes in unrealized gains or losses included in other comprehensive income (loss) for recurring Level 3 fair value measurements held at the end of each reporting period and the explicit requirement to disclose the range and weighted‑average of significant unobservable inputs used for Level 3 fair value measurements. The other provisions of ASU 2018‑13 include eliminated and modified disclosure requirements. An entity is permitted to early adopt any removed or modified disclosures upon issuance of ASU 2018‑13 and delay adoption of the additional disclosures until their effective date. For all entities, this guidance is required to be adopted for annual periods beginning after December 15, 2019, including interim periods within those fiscal years. Early adoption is permitted. The Company adopted ASU 2018‑13 as of the required effective date of January 1, 2020, and the adoption did not have a material impact on the Company’s consolidated financial statements. Recently Issued Accounting Pronouncements From time to time, new accounting pronouncements are issued by the FASB or other standard‑setting bodies that the Company adopts as of the specified effective date. The Company qualifies as an “emerging growth company” as defined in the Jumpstart Our Business Startups Act of 2012 and has elected not to “opt out” of the extended transition related to complying with new or revised accounting standards, which means that when a standard is issued or revised and it has different application dates for public and non‑public companies, the Company can adopt the new or revised standard at the time non‑public companies adopt the new or revised standard and can do so until such time that the Company either (i) irrevocably elects to “opt out” of such extended transition period or (ii) no longer qualifies as an emerging growth company. The Company may choose to early adopt any new or revised accounting standards whenever such early adoption is permitted for non‑public companies. In June 2016, the FASB issued ASU No. 2016‑13, Financial Instruments—Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments (“ASU 2016‑13”), which requires the measurement and recognition of expected credit losses for financial assets held at amortized cost. ASU 2016‑13 replaces the existing incurred loss impairment model with an expected loss model. It also eliminates the concept of other‑than‑temporary impairment and requires credit losses related to available‑for‑sale debt securities to be recorded through an allowance for credit losses rather than as a reduction in the amortized cost basis of the securities. These changes may result in earlier recognition of credit losses. In November 2018, the FASB issued ASU No. 2018‑19, Codification Improvements to Topic 326, Financial Instruments—Credit Losses , which narrowed the scope and changed the effective date for non‑public entities for ASU 2016‑13. The FASB subsequently issued supplemental guidance within ASU No. 2019‑05, Financial Instruments—Credit Losses (Topic 326): Targeted Transition Relief (“ASU 2019‑05”). ASU 2019‑05 provides an option to irrevocably elect the fair value option for certain financial assets previously measured at amortized cost basis. For public entities that are SEC filers, excluding entities eligible to be smaller reporting companies, ASU 2016‑13 is effective for annual periods beginning after December 15, 2019, including interim periods within those fiscal years. For all other entities, ASU 2016‑13 is effective for annual periods beginning after December 15, 2022, including interim periods within those fiscal years. Early adoption is permitted. The Company is currently evaluating the impact that the adoption of ASU 2016‑13 will have on its consolidated financial statements. |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 9 Months Ended |
Sep. 30, 2020 | |
Summary of Significant Accounting Policies | |
Schedule of estimated useful lives | Estimated Useful Life Laboratory equipment 5 years Furniture and fixtures 4 years Leasehold improvements Shorter of term of lease or 15 years |
Fair Value Measurements (Tables
Fair Value Measurements (Tables) | 9 Months Ended |
Sep. 30, 2020 | |
Fair Value Measurements | |
Schedule of fair value hierarchy for its assets and liabilities that are measured at fair value on a recurring basis | Fair Value Measurements at September 30, 2020 Using: Level 1 Level 2 Level 3 Total Assets: Cash equivalents: Money market funds $ 177,940 $ — $ — $ 177,940 Restricted cash: Money market funds 1,292 — — 1,292 Marketable securities: U.S. Treasury notes — 141,452 — 141,452 $ 179,232 $ 141,452 $ — $ 320,684 Fair Value Measurements at December 31, 2019 Using: Level 1 Level 2 Level 3 Total Assets: Cash equivalents: Money market funds $ 24,281 $ — $ — $ 24,281 Restricted cash: Money market funds 1,292 — — 1,292 $ 25,573 $ — $ — $ 25,573 |
Marketable Securities (Tables)
Marketable Securities (Tables) | 9 Months Ended |
Sep. 30, 2020 | |
Investments, Debt and Equity Securities [Abstract] | |
Schedule of the fair value of available-for-sale marketable debt securities by type of security | September 30, 2020 Gross Gross Amortized Unrealized Unrealized Fair Cost Gain Loss Value Assets Marketable securities: U.S. Treasury notes $ 141,444 $ 8 $ — $ 141,452 $ 141,444 $ 8 $ — $ 141,452 |
Property and Equipment, Net (Ta
Property and Equipment, Net (Tables) | 9 Months Ended |
Sep. 30, 2020 | |
Property and Equipment, Net | |
Schedule of property and equipment, net | September 30, 2020 December 31, 2019 Laboratory equipment $ 4,623 $ 2,536 Furniture and fixtures 461 358 Leasehold improvements 8,915 8,915 Construction in progress 495 77 14,494 11,886 Less: Accumulated depreciation and amortization (1,827) (394) $ 12,667 $ 11,492 |
Accrued Expenses and Other Cu_2
Accrued Expenses and Other Current Liabilities (Tables) | 9 Months Ended |
Sep. 30, 2020 | |
Accrued Expenses and Other Current Liabilities | |
Schedule of accrued expenses and other current liabilities | September 30, 2020 December 31, 2019 Accrued external research, development, and manufacturing expenses $ 3,156 $ 2,605 Accrued employee compensation and benefits 1,581 1,157 Accrued professional fees 1,497 67 Payments due for leasehold improvements — 756 Other 496 377 $ 6,730 $ 4,962 |
Stock_Based Compensation (Table
Stock‑Based Compensation (Tables) | 9 Months Ended |
Sep. 30, 2020 | |
Stock‑Based Compensation | |
Schedule of stock option activity | Weighted‑ Weighted‑ Average Average Aggregate Number of Exercise Contractual Intrinsic Shares Price Term Value (in years) (in thousands) Outstanding as of December 31, 2019 961,751 $ 1.69 9.5 $ 524 Granted 1,989,654 8.67 Exercised (32,511) 1.27 Forfeited (57,766) 3.32 Outstanding as of September 30, 2020 2,861,128 $ 6.54 $ 46,726 Vested and expected to vest as of September 30, 2020 2,861,128 $ 6.54 $ 46,726 Options exercisable as of September 30, 2020 349,736 $ 3.58 $ 6,745 |
Schedule of unvested common stock from option early exercises | Shares Unvested restricted common stock as of December 31, 2019 102,896 Vested (48,615) Unvested restricted common stock as of September 30, 2020 54,281 |
Schedule of restricted common stock award activity | Weighted‑ Average Grant‑Date Shares Fair Value Unvested restricted common stock as of December 31, 2019 209,742 $ 0.0443 Vested (87,986) 0.0400 Unvested restricted common stock as of September 30, 2020 121,756 $ 0.0500 |
Schedule of stock based compensation expense | Three Months Ended Nine Months Ended September 30, September 30, 2020 2019 2020 2019 Research and development expenses $ 399 $ 12 $ 617 $ 30 General and administrative expenses 1,677 9 2,060 27 $ 2,076 $ 21 $ 2,677 $ 57 |
Net Loss per Share (Tables)
Net Loss per Share (Tables) | 9 Months Ended |
Sep. 30, 2020 | |
Net Loss per Share | |
Schedule of net loss per share | Three Months Ended September 30, Nine Months Ended September 30, 2020 2019 2020 2019 Numerator: Net loss attributable to common stockholders $ (13,089) $ (8,210) $ (36,052) $ (16,123) Denominator: Weighted‑average common shares outstanding, basic and diluted 15,334,241 621,581 11,991,870 586,728 Net loss per share attributable to common stockholders, basic and diluted $ (0.85) $ (13.21) $ (3.01) $ (27.48) |
Schedule of securities excluded from computation of net loss per share | September 30, 2020 2019 Convertible preferred stock (as converted to common stock) — 18,969,672 Unvested restricted common stock 176,037 295,360 Stock options to purchase common stock 2,861,128 398,726 3,037,165 19,663,758 |
Nature of the Business and Ba_2
Nature of the Business and Basis of Presentation (Details) $ / shares in Units, $ in Thousands | Jun. 30, 2020USD ($)$ / sharesshares | Jun. 18, 2020 | Jun. 30, 2020USD ($)$ / sharesshares | Sep. 30, 2020USD ($) | Dec. 31, 2019USD ($) |
Temporary Equity [Line Items] | |||||
Value of shares issued | $ 223,850 | ||||
Accumulated deficit | $ (69,176) | $ (33,124) | |||
Common Stock | |||||
Temporary Equity [Line Items] | |||||
Reverse stock split ratio | 21.073 | ||||
Shares issued | shares | 14,375,000 | 14,375,000 | |||
Share price | $ / shares | $ 17 | $ 17 | |||
Value of shares issued | $ 223,800 | $ 1 | |||
Shares issued, gross proceeds | $ 244,400 |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies (Details) $ in Millions | 9 Months Ended | |
Sep. 30, 2020USD ($)Institution | Dec. 31, 2019USD ($) | |
Summary of Significant Accounting Policies | ||
Number of financial institutions in which the company maintains cash and cash equivalents | Institution | 1 | |
Letter of credit related to lease agreement | $ 1.3 | $ 1.3 |
Cash collateral for credit card account | $ 0.1 | $ 0.1 |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies - Property and Equipment (Details) | 9 Months Ended |
Sep. 30, 2020 | |
Laboratory equipment | |
Property and Equipment | |
Property, Plant and Equipment, Useful Life | 5 years |
Furniture and fixtures | |
Property and Equipment | |
Property, Plant and Equipment, Useful Life | 4 years |
Leasehold improvements | |
Property and Equipment | |
Property, Plant and Equipment, Useful Life | 15 years |
Summary of Significant Accoun_6
Summary of Significant Accounting Policies - Preferred stock, etc. (Details) - USD ($) $ in Millions | Jun. 30, 2020 | Sep. 30, 2020 | Jun. 30, 2020 | Sep. 30, 2019 | Sep. 30, 2020 | Sep. 30, 2019 | Dec. 31, 2019 |
Summary of Significant Accounting Policies [Line Items] | |||||||
Impairment losses on long-lived assets | $ 0 | $ 0 | $ 0 | $ 0 | |||
Amounts accrued for interest and penalties | $ 0 | $ 0 | $ 0 | ||||
Common Stock | |||||||
Summary of Significant Accounting Policies [Line Items] | |||||||
Common stock shares converted | 18,969,672 | 18,969,672 |
Fair Value Measurements - Recur
Fair Value Measurements - Recurring Basis (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | |||
Sep. 30, 2020 | Sep. 30, 2019 | Sep. 30, 2020 | Sep. 30, 2019 | Dec. 31, 2019 | |
Fair Value, Transfers Between Level 1 and Level 2, Description and Policy [Abstract] | |||||
Transfer of fair value asset, Level 1 into Level 2 | $ 0 | $ 0 | $ 0 | $ 0 | |
Transfer of fair value asset, Level 2 into Level 1 | 0 | 0 | 0 | 0 | |
Transfer of fair value asset, into Level 3 | 0 | 0 | 0 | 0 | |
Transfer of fair value asset, out of Level 3 | 0 | 0 | 0 | 0 | |
Transfer of fair value liability, Level 1 into Level 2 | 0 | 0 | 0 | 0 | |
Transfer of fair value liability Level 2 into Level 1 | 0 | 0 | 0 | 0 | |
Transfer of fair value liability, into Level 3 | 0 | 0 | 0 | 0 | |
Transfer of fair value liability, out of Level 3 | 0 | $ 0 | 0 | $ 0 | |
Recurring | |||||
Assets: | |||||
Assets | 320,684 | 320,684 | $ 25,573 | ||
Recurring | Money market funds | |||||
Assets: | |||||
Cash equivalents | 177,940 | 177,940 | 24,281 | ||
Restricted cash | 1,292 | 1,292 | 1,292 | ||
Recurring | US Treasury Securities [Member] | |||||
Assets: | |||||
Marketable securities | 141,452 | 141,452 | |||
Recurring | Level 1 | |||||
Assets: | |||||
Assets | 179,232 | 179,232 | 25,573 | ||
Recurring | Level 1 | Money market funds | |||||
Assets: | |||||
Cash equivalents | 177,940 | 177,940 | 24,281 | ||
Restricted cash | 1,292 | 1,292 | $ 1,292 | ||
Recurring | Level 2 | |||||
Assets: | |||||
Assets | 141,452 | 141,452 | |||
Recurring | Level 2 | US Treasury Securities [Member] | |||||
Assets: | |||||
Marketable securities | $ 141,452 | $ 141,452 |
Marketable Securities (Details)
Marketable Securities (Details) $ in Thousands | Sep. 30, 2020USD ($) |
Debt Securities, Available-for-sale [Line Items] | |
Marketable securities, Amortized Cost | $ 141,444 |
Unrealized gain | 8 |
Fair market value | 141,452 |
US Treasury Securities [Member] | |
Debt Securities, Available-for-sale [Line Items] | |
Marketable securities, Amortized Cost | 141,444 |
Unrealized gain | 8 |
Fair market value | $ 141,452 |
Property and Equipment, Net (De
Property and Equipment, Net (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | |||
Sep. 30, 2020 | Sep. 30, 2019 | Sep. 30, 2020 | Sep. 30, 2019 | Dec. 31, 2019 | |
Property and Equipment | |||||
Property and equipment, Gross | $ 14,494 | $ 14,494 | $ 11,886 | ||
Less: Accumulated depreciation and amortization | (1,827) | (1,827) | (394) | ||
Property and Equipment, Net | 12,667 | 12,667 | 11,492 | ||
Depreciation and amortization expense | 500 | $ 100 | 1,433 | $ 172 | |
Laboratory equipment | |||||
Property and Equipment | |||||
Property and equipment, Gross | 4,623 | 4,623 | 2,536 | ||
Furniture and fixtures | |||||
Property and Equipment | |||||
Property and equipment, Gross | 461 | 461 | 358 | ||
Leasehold improvements | |||||
Property and Equipment | |||||
Property and equipment, Gross | 8,915 | 8,915 | 8,915 | ||
Construction in progress | |||||
Property and Equipment | |||||
Property and equipment, Gross | $ 495 | $ 495 | $ 77 |
Accrued Expenses and Other Cu_3
Accrued Expenses and Other Current Liabilities (Details) - USD ($) $ in Thousands | Sep. 30, 2020 | Dec. 31, 2019 |
Accrued Expenses and Other Current Liabilities | ||
Accrued external research, development, and manufacturing expenses | $ 3,156 | $ 2,605 |
Accrued employee compensation and benefits | 1,581 | 1,157 |
Accrued professional fees | 1,497 | 67 |
Payments due for leasehold improvements | 756 | |
Other | 496 | 377 |
Total | $ 6,730 | $ 4,962 |
Convertible Preferred Stock (De
Convertible Preferred Stock (Details) - USD ($) $ / shares in Units, $ in Thousands | Jun. 30, 2020 | Feb. 29, 2020 | Jun. 30, 2020 | Mar. 31, 2020 | Sep. 30, 2019 | Sep. 30, 2020 | Sep. 30, 2019 |
Temporary Equity [Line Items] | |||||||
Gross proceeds received from sale of convertible preferred stock | $ 104,837 | $ 25,016 | |||||
Issuance costs | $ 200 | $ 228 | $ 34 | $ 3,419 | |||
Series B preferred stock | |||||||
Temporary Equity [Line Items] | |||||||
Shares issued | 221,399,223 | ||||||
Share price | $ 0.47455 | ||||||
Gross proceeds received from sale of convertible preferred stock | $ 105,100 | ||||||
Common Stock | |||||||
Temporary Equity [Line Items] | |||||||
Shares issued | 14,375,000 | 14,375,000 | |||||
Share price | $ 17 | $ 17 | |||||
Common stock shares converted | 18,969,672 | 18,969,672 |
Stockholders' Equity (Details)
Stockholders' Equity (Details) $ / shares in Units, $ in Thousands | Jun. 30, 2020USD ($)$ / sharesshares | Jun. 18, 2020 | Jun. 30, 2020USD ($)$ / sharesshares | Sep. 30, 2020$ / sharesshares | Dec. 31, 2019$ / sharesshares |
Class of Stock [Line Items] | |||||
Common Stock, Shares Authorized | 200,000,000 | 235,000,000 | |||
Common Stock, Par or Stated Value Per Share | $ / shares | $ 0.0001 | $ 0.0001 | |||
Issuance of common stock upon completion of initial public offering, net of commissions, underwriting discounts and offering costs | $ | $ 223,850 | ||||
Preferred Stock, Shares Authorized | 5,000,000 | 0 | |||
Preferred Stock, Par or Stated Value Per Share | $ / shares | $ 0.0001 | $ 0.0001 | |||
Preferred Stock, Shares Issued | 0 | 0 | |||
Preferred Stock, Shares Outstanding | 0 | 0 | |||
Common Stock | |||||
Class of Stock [Line Items] | |||||
Reverse stock split ratio | 21.073 | ||||
Issuance of common stock upon completion of initial public offering, net of commissions, underwriting discounts and offering costs, shares | 14,375,000 | 14,375,000 | |||
Share Price | $ / shares | $ 17 | $ 17 | |||
Shares issued, gross proceeds | $ | $ 244,400 | ||||
Issuance of common stock upon completion of initial public offering, net of commissions, underwriting discounts and offering costs | $ | $ 223,800 | $ 1 |
Stock-Based Compensation (Detai
Stock-Based Compensation (Details) - shares | Jun. 17, 2020 | Sep. 30, 2020 | Dec. 31, 2019 |
2016 Plan | |||
Stock-Based Compensation | |||
Number of shares authorized for issuance | 3,722,685 | 1,508,669 | |
Remaining shares available for issuance | 0 | 363,350 | |
2016 Plan | Stock option | |||
Stock-Based Compensation | |||
Vesting period | 4 years | ||
Expiration period | 10 years | ||
2020 Stock Plan | |||
Stock-Based Compensation | |||
Number of shares authorized for issuance | 5,035,409 | ||
Number of shares authorized, fixed portion | 4,294,594 | ||
Number of shares authorized, variable portion | 3,622,691 | ||
Annual increase in shares reserved for issuance | 2,728,610 | ||
Annual increase in shares reserved for issuance, as a percent of stock outstanding | 4.00% | ||
2020 Employee Stock Purchase Plan | |||
Stock-Based Compensation | |||
Number of shares authorized, fixed portion | 360,651 | ||
Annual increase in shares reserved for issuance | 640,630 | ||
Annual increase in shares reserved for issuance, as a percent of stock outstanding | 1.00% |
Stock_Based Compensation - Stoc
Stock‑Based Compensation - Stock option activity (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 9 Months Ended | 12 Months Ended | ||
Sep. 30, 2020 | Sep. 30, 2019 | Sep. 30, 2020 | Sep. 30, 2019 | Dec. 31, 2019 | |
Share-based Compensation Arrangement by Share-based Payment Award, Options, Additional Disclosures [Abstract] | |||||
Weighted-average grant-date fair value of stock options granted | $ 17.81 | $ 1.22 | $ 7.08 | $ 1.10 | |
Stock option | |||||
Number of Shares | |||||
Outstanding as at beginning of period (in shares) | 961,751 | ||||
Granted (in shares) | 1,989,654 | ||||
Exercised (in shares) | (32,511) | ||||
Forfeited (in shares) | (57,766) | ||||
Outstanding as at end of period (in shares) | 2,861,128 | 2,861,128 | 961,751 | ||
Vested and expected to vest as at end of period (in shares) | 2,861,128 | 2,861,128 | |||
Options exercisable as at end of period (in shares) | 349,736 | 349,736 | |||
Weighted Average Exercise Price | |||||
Weighted average exercise price outstanding as at beginning of period (in dollars per share) | $ 1.69 | ||||
Weighted average exercise price, Granted (in dollars per share) | 8.67 | ||||
Weighted average exercise price, Exercised (in dollars per share) | 1.27 | ||||
Weighted average exercise price, Forfeited (in dollars per share) | 3.32 | ||||
Weighted average exercise price, Outstanding as at end of period (in dollars per share) | $ 6.54 | 6.54 | $ 1.69 | ||
Vested and expected to vest as at end of period (in dollars per share) | 6.54 | 6.54 | |||
Options exercisable as at end of period (in dollars per share) | $ 3.58 | $ 3.58 | |||
Share-based Compensation Arrangement by Share-based Payment Award, Options, Additional Disclosures [Abstract] | |||||
Contractual term, Outstanding (in years) | 9 years 2 months 12 days | 9 years 6 months | |||
Contractual term, Vested and expected to vest (in years) | 9 years 2 months 12 days | ||||
Contractual term, Options exercisable (in years) | 8 years 7 months 6 days | ||||
Average intrinsic value, Outstanding | $ 46,726 | $ 46,726 | $ 524 | ||
Average intrinsic value, Vested and expected to vest | 46,726 | 46,726 | |||
Average intrinsic value, Exercisable | $ 6,745 | $ 6,745 |
Stock_Based Compensation - Earl
Stock‑Based Compensation - Early exercise of stock options into restricted stock (Details) - USD ($) $ in Millions | 9 Months Ended | 12 Months Ended |
Sep. 30, 2020 | Dec. 31, 2019 | |
Stock-Based Compensation | ||
Liability for unvested shares | $ 0.1 | |
Maximum | ||
Stock-Based Compensation | ||
Liability for unvested shares | $ 0.1 | |
Restricted stock from early exercise of options | ||
Stock-Based Compensation | ||
Unvested restricted common stock at beginning of period | 102,896 | |
Vested | (48,615) | |
Unvested restricted common stock at end of period | 54,281 | 102,896 |
Stock_Based Compensation - Rest
Stock‑Based Compensation - Restricted common stock award activity (Details) - USD ($) $ / shares in Units, $ in Millions | 3 Months Ended | 9 Months Ended | 12 Months Ended |
Sep. 30, 2020 | Sep. 30, 2020 | Dec. 31, 2019 | |
Restricted stock awards | |||
Shares | |||
Unvested restricted common stock at beginning of period | 209,742 | ||
Vested | (87,986) | ||
Unvested restricted common stock at end of period | 121,756 | 121,756 | 209,742 |
Weighted Average Grant Date Fair Value | |||
Unvested restricted common stock at beginning of period | $ 0.0443 | ||
Vested | 0.0400 | ||
Unvested restricted common stock at end of period | $ 0.0500 | $ 0.0500 | $ 0.0443 |
Restricted stock awards | Maximum | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Liability for unvested awards | $ 0.1 | $ 0.1 | $ 0.1 |
Weighted Average Grant Date Fair Value | |||
Total fair value of restricted common stock vested | $ 0.1 | $ 0.1 | |
Service‑based restricted stock | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Vesting period | 4 years | ||
Issued in period | 0 | 0 |
Stock_Based Compensation - St_2
Stock‑Based Compensation - Stock based compensation expense (Details) - USD ($) $ in Thousands | Jul. 20, 2020 | Sep. 30, 2020 | Sep. 30, 2019 | Sep. 30, 2020 | Sep. 30, 2019 |
Stock-Based Compensation | |||||
Stock-based compensation expense | $ 2,076 | $ 21 | $ 2,677 | $ 57 | |
Accelerated vesting, expense recognized | 1,100 | 1,100 | |||
Total unrecognized stock‑based compensation expense related to the unvested stock‑based awards | 13,600 | $ 13,600 | |||
Unrecognized stock-based compensation expense, weighted average period | 2 years 3 months | ||||
Stock option | |||||
Stock-Based Compensation | |||||
Accelerated vesting, number of shares | 60,948 | ||||
Stock option | Terminated Employee [Member] | |||||
Stock-Based Compensation | |||||
Accelerated vesting, number of shares | 22,684 | ||||
Restricted stock awards | |||||
Stock-Based Compensation | |||||
Accelerated vesting, number of shares | 1,541 | ||||
Research and development expenses | |||||
Stock-Based Compensation | |||||
Stock-based compensation expense | 399 | 12 | $ 617 | 30 | |
General and administrative expenses | |||||
Stock-Based Compensation | |||||
Stock-based compensation expense | $ 1,677 | $ 9 | $ 2,060 | $ 27 |
License Agreement (Details)
License Agreement (Details) | 1 Months Ended | 3 Months Ended | 9 Months Ended | ||
Oct. 31, 2017USD ($)item | Sep. 30, 2020USD ($) | Sep. 30, 2019USD ($) | Sep. 30, 2020USD ($) | Sep. 30, 2019USD ($) | |
License Agreements | |||||
Research and development | $ 8,641,000 | $ 4,377,000 | $ 26,612,000 | $ 11,886,000 | |
MEE License | |||||
License Agreements | |||||
Number of domestic patients | item | 3,000 | ||||
IND filing period | 18 months | ||||
Fair value of stock | $ 100,000 | ||||
Milestone payment amount | 0 | 0 | 0 | 0 | |
Period for start commercial sale of product | 10 years | ||||
Number of days for advance written notice | 90 days | ||||
Research and development | 0 | 0 | 0 | 0 | |
MEE License | Maximum | |||||
License Agreements | |||||
Milestone payment amount | $ 17,700,000 | ||||
Lonza Sublicense | |||||
License Agreements | |||||
Number of domestic patients | item | 3,000 | ||||
IND filing period | 18 months | ||||
Fair value of stock | $ 100,000 | ||||
Milestone payment amount | 0 | 0 | 0 | 0 | |
Period for start commercial sale of product | 10 years | ||||
Number of days for advance written notice | 90 days | ||||
Research and development | $ 0 | $ 0 | $ 0 | $ 0 | |
Lonza Sublicense | Maximum | |||||
License Agreements | |||||
Milestone payment amount | $ 18,500,000 |
Net Loss per Share (Details)
Net Loss per Share (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 9 Months Ended | ||||||
Sep. 30, 2020 | Jun. 30, 2020 | Mar. 31, 2020 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Sep. 30, 2020 | Sep. 30, 2019 | |
Net Loss per Share | ||||||||
Net loss attributable to common stockholders | $ (13,089) | $ (12,523) | $ (10,440) | $ (8,210) | $ (2,004) | $ (5,909) | $ (36,052) | $ (16,123) |
Weighted-average common shares outstanding, basic and diluted | 15,334,241 | 621,581 | 11,991,870 | 586,728 | ||||
Net loss per share attributable to common stockholders, basic and diluted | $ (0.85) | $ (13.21) | $ (3.01) | $ (27.48) |
Net Loss per Share - Antidiluti
Net Loss per Share - Antidilutive securities (Details) - shares | 9 Months Ended | |
Sep. 30, 2020 | Sep. 30, 2019 | |
Net Loss per Share and Unaudited Pro Forma Net Loss per Share | ||
Securities excluded from computation of earnings per share amount | 3,037,165 | 19,663,758 |
Convertible preferred stock | ||
Net Loss per Share and Unaudited Pro Forma Net Loss per Share | ||
Securities excluded from computation of earnings per share amount | 18,969,672 | |
Restricted stock awards | ||
Net Loss per Share and Unaudited Pro Forma Net Loss per Share | ||
Securities excluded from computation of earnings per share amount | 176,037 | 295,360 |
Stock option | ||
Net Loss per Share and Unaudited Pro Forma Net Loss per Share | ||
Securities excluded from computation of earnings per share amount | 2,861,128 | 398,726 |
Subsequent Events (Details)
Subsequent Events (Details) - Subsequent event $ / shares in Units, $ in Millions | Oct. 01, 2020USD ($)$ / sharesshares |
Subsequent Event [Line Items] | |
Granted (in shares) | shares | 642,780 |
Exercise price | $ / shares | $ 22.45 |
Aggregate grant-date fair value of options | $ | $ 11.3 |
Vesting period | 4 years |