Cover Page
Cover Page - USD ($) | 12 Months Ended | ||
Dec. 31, 2019 | Mar. 09, 2020 | Jun. 28, 2019 | |
Cover [Abstract] | |||
Document Type | 10-K | ||
Amendment Flag | false | ||
Document Period End Date | Dec. 31, 2019 | ||
Document Fiscal Year Focus | 2019 | ||
Document Fiscal Period Focus | FY | ||
Trading Symbol | TBIO | ||
Entity Registrant Name | Translate Bio, Inc. | ||
Entity Central Index Key | 0001693415 | ||
Current Fiscal Year End Date | --12-31 | ||
Entity Current Reporting Status | Yes | ||
Entity Shell Company | false | ||
Entity Filer Category | Accelerated Filer | ||
Entity Small Business | true | ||
Entity Emerging Growth Company | true | ||
Entity Ex Transition Period | true | ||
Entity Address, State or Province | DE | ||
Title of 12(b) Security | Common Stock | ||
Entity Interactive Data Current | Yes | ||
Security Exchange Name | NASDAQ | ||
Entity Public Float | $ 486,315,359 | ||
Entity Voluntary Filers | No | ||
Entity Common Stock, Shares Outstanding | 60,029,454 | ||
Entity Well-known Seasoned Issuer | No |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Current assets: | ||
Cash and cash equivalents | $ 84,580 | $ 55,199 |
Short-term investments | 104,098 | 88,904 |
Short-term collaboration receivables | 4,596 | 833 |
Prepaid expenses and other current assets | 9,391 | 3,641 |
Restricted cash | 950 | 1,025 |
Total current assets | 203,615 | 149,602 |
Property and equipment, net | 12,539 | 10,245 |
Right-of-use assets, net | 10,400 | |
Goodwill | 21,359 | 21,359 |
Intangible assets, net | 85,536 | 106,445 |
Other assets | 2,752 | |
Total assets | 336,201 | 287,651 |
Current liabilities: | ||
Accounts payable | 15,968 | 5,168 |
Accrued expenses | 7,072 | 6,547 |
Current portion of deferred revenue | 18,100 | 2,572 |
Current portion of operating lease liability | 530 | |
Total current liabilities | 41,670 | 14,287 |
Contingent consideration | 103,655 | 103,642 |
Deferred revenue, net of current portion | 25,256 | 41,841 |
Deferred tax liabilities | 481 | |
Deferred rent | 2,105 | |
Operating lease liability, net of current portion | 12,084 | |
Total liabilities | 182,665 | 162,356 |
Commitments and contingencies (Notes 3, 4 and 14) | ||
Stockholders' equity (deficit): | ||
Preferred stock, $0.001 par value; 10,000,000 shares authorized as of December 31, 2019 and 2018, respectively; no shares issued and outstanding as of December 31, 2019 and 2018 | ||
Common stock, $0.001 par value; 200,000,000 shares authorized as of December 31, 2019 and 2018; 60,022,067 shares and 45,139,955 shares issued and outstanding as of December 31, 2019 and 2018, respectively | 60 | 45 |
Additional paid-in capital | 512,231 | 371,257 |
Accumulated deficit | (359,496) | (246,203) |
Accumulated other comprehensive income | 741 | 196 |
Total stockholders' equity (deficit) | 153,536 | 125,295 |
Total liabilities and stockholders' equity (deficit) | $ 336,201 | $ 287,651 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - $ / shares | Dec. 31, 2019 | Dec. 31, 2018 |
Statement of Financial Position [Abstract] | ||
Preferred stock, par value | $ 0.001 | $ 0.001 |
Preferred stock, shares authorized | 10,000,000 | 10,000,000 |
Preferred stock, shares Issued | 0 | 0 |
Preferred stock, shares outstanding | 0 | 0 |
Common stock, par value | $ 0.001 | $ 0.001 |
Common stock, shares authorized | 200,000,000 | 200,000,000 |
Common stock, shares issued | 60,022,067 | 45,139,955 |
Common stock, shares outstanding | 60,022,067 | 45,139,955 |
Consolidated Statements of Oper
Consolidated Statements of Operations - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Income Statement [Abstract] | ||
Collaboration revenue | $ 7,804 | $ 1,420 |
Operating expenses: | ||
Research and development | 76,369 | 58,024 |
General and administrative | 28,632 | 22,606 |
Change in fair value of contingent consideration | 13 | 25,020 |
Impairment of intangible asset | 18,559 | |
Total operating expenses | 123,573 | 105,650 |
Loss from operations | (115,769) | (104,230) |
Other income (expense): | ||
Interest income | 2,010 | 1,323 |
Other expense | (20) | (53) |
Total other income (expense), net | 1,990 | 1,270 |
Loss before benefit from income taxes | (113,779) | (102,960) |
Benefit from income taxes | 486 | 5,565 |
Net loss | (113,293) | (97,395) |
Accretion of redeemable convertible preferred stock to redemption value | (644) | |
Net loss attributable to common stockholders | $ (113,293) | $ (98,039) |
Net loss per share attributable to common stockholders—basic and diluted | $ (2.20) | $ (3.64) |
Weighted average common shares outstanding—basic and diluted | 51,445,539 | 26,945,508 |
Consolidated Statements of Comp
Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Statement of Comprehensive Income [Abstract] | ||
Net loss | $ (113,293) | $ (97,395) |
Other comprehensive income (loss): | ||
Unrealized gains on available-for-sale securities, net of tax of $0 | 545 | 117 |
Comprehensive loss | $ (112,748) | $ (97,278) |
Consolidated Statements of Co_2
Consolidated Statements of Comprehensive Loss (Parenthetical) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Statement of Comprehensive Income [Abstract] | ||
Unrealized gains on available-for-sale securities, net of tax | $ 0 | $ 0 |
Consolidated Statements of Rede
Consolidated Statements of Redeemable Convertible Preferred Stock and Stockholders' Equity (Deficit) - USD ($) $ in Thousands | Total | Common Stock [Member] | Additional Paid-in Capital [Member] | Accumulated Deficit [Member] | Accumulated Other Comprehensive Income [Member] | Redeemable Convertible Preferred Stock [Member] |
Beginning balances at Dec. 31, 2017 | $ (93,515) | $ 10 | $ 55,204 | $ (148,808) | $ 79 | |
Beginning balance, Shares at Dec. 31, 2017 | 9,582,791 | |||||
Redeemable convertible preferred stock, beginning balance at Dec. 31, 2017 | $ 192,896 | |||||
Redeemable convertible preferred stock, shares outstanding, beginning balance at Dec. 31, 2017 | 142,288,292 | |||||
Accretion of redeemable convertible preferred stock to redemption value | (644) | (644) | $ 644 | |||
Conversion of redeemable convertible preferred stock to common stock | 193,540 | $ 26 | 193,514 | $ (193,540) | ||
Conversion of redeemable convertible preferred stock to common stock, Shares | 25,612,109 | (142,288,292) | ||||
Issuance of common stock in connection with IPO and SHOE, net of commissions and offering costs | 113,192 | $ 9 | 113,183 | |||
Issuance of common stock in connection with IPO and SHOE, net of commissions and offering costs ,Shares | 9,714,371 | |||||
Issuance of common stock in full settlement of contingent consideration anti-dilution liability (Note 5) | 2,387 | 2,387 | ||||
Issuance of common stock in full settlement of contingent consideration anti-dilution liability (Note 5) ,Shares | 183,619 | |||||
Issuance of common stock due to the aggregation of fractional shares in connection with the reverse stock split ,Shares | 52 | |||||
Forfeited restricted common stock | 2 | 2 | ||||
Forfeited restricted common stock, Shares | (2,446) | |||||
Exercise of stock options | 278 | 278 | ||||
Exercise of stock options, Shares | 49,459 | |||||
Stock-based compensation expense | 7,333 | 7,333 | ||||
Unrealized gains on available-for-sale securities | 117 | 117 | ||||
Net loss | (97,395) | (97,395) | ||||
Ending balances at Dec. 31, 2018 | $ 125,295 | $ 45 | 371,257 | (246,203) | 196 | |
Ending balance, shares at Dec. 31, 2018 | 45,139,955 | |||||
Redeemable convertible preferred stock, shares outstanding, beginning balance at Dec. 31, 2018 | 0 | |||||
Issuance of common stock in connection with private placement, net of placement agent fees and offering costs | $ 44,134 | $ 6 | 44,128 | |||
Issuance of common stock in connection with private placement, net of placement agent fees and offering costs,shares | 5,582,940 | |||||
Issuance of common stock in connection with public offering, net of underwriting discounts and commissions and offering costs | 84,011 | $ 9 | 84,002 | |||
Issuance of common stock in connection with public offering, net of underwriting discounts and commissions and offering costs ,Shares | 9,000,000 | |||||
Issuance of common stock in connection with a former employee letter agreement (Note 10) | 847 | 847 | ||||
Issuance of common stock in connection with a former employee letter agreement (Note 10), Shares | 67,406 | |||||
Forfeited restricted common stock | (2) | (2) | ||||
Forfeited restricted common stock, Shares | (1,783) | |||||
Exercise of stock options | 1,512 | 1,512 | ||||
Exercise of stock options, Shares | 233,549 | |||||
Stock-based compensation expense | 10,487 | 10,487 | ||||
Unrealized gains on available-for-sale securities | 545 | 545 | ||||
Net loss | (113,293) | (113,293) | ||||
Ending balances at Dec. 31, 2019 | $ 153,536 | $ 60 | $ 512,231 | $ (359,496) | $ 741 | |
Ending balance, shares at Dec. 31, 2019 | 60,022,067 |
Consolidated Statements of Cash
Consolidated Statements of Cash flows - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Cash flows from operating activities: | ||
Net loss | $ (113,293) | $ (97,395) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Depreciation and amortization expense | 4,732 | 2,841 |
Stock-based compensation expense | 11,334 | 7,333 |
Impairment of intangible asset | 18,559 | |
Change in fair value of contingent consideration | 13 | 25,020 |
Deferred income tax benefit | (486) | (5,565) |
Changes in operating assets and liabilities: | ||
Short-term collaboration receivables | (3,763) | (833) |
Prepaid expenses and other assets | (8,495) | (1,346) |
Right-of-use assets | 484 | |
Accounts payable | 9,928 | 658 |
Accrued expenses | 257 | 2,271 |
Deferred rent | 470 | |
Lease liability | (375) | |
Deferred revenue | (1,057) | 44,413 |
Net cash used in operating activities | (82,162) | (22,133) |
Cash flows from investing activities: | ||
Purchases of investments | (174,271) | (136,694) |
Sales and maturities of investments | 159,622 | 57,984 |
Purchases of property and equipment | (3,537) | (6,580) |
Net cash used in investing activities | (18,186) | (85,290) |
Cash flows from financing activities: | ||
Proceeds from public offering, net of underwriting discounts and commissions | 84,600 | |
Payments of public offering costs | (589) | |
Proceeds from private placement, net of placement agent fees | 44,608 | |
Payments of private placement offering costs | (477) | |
Proceeds from initial public offering of common stock, net of underwriting discounts and commissions | 117,447 | |
Payments of initial public offering costs | (4,102) | |
Proceeds from option exercises | 1,512 | 278 |
Net cash provided by financing activities | 129,654 | 113,623 |
Net increase in cash, cash equivalents and restricted cash | 29,306 | 6,200 |
Cash, cash equivalents and restricted cash at beginning of period | 56,224 | 50,024 |
Cash, cash equivalents and restricted cash at end of period | 85,530 | 56,224 |
Cash, cash equivalents and restricted cash at end of period: | ||
Cash and cash equivalents | 84,580 | 55,199 |
Restricted cash | 950 | 1,025 |
Total cash, cash equivalents and restricted cash at end of period | 56,224 | 50,024 |
Supplemental disclosure of non-cash investing and financing activities: | ||
Purchases of property and equipment included in accounts payable and accrued expenses | 1,189 | 49 |
Issuance of common stock in connection with a former employee letter agreement (Note 10) | $ 847 | |
Issuance of common stock as settlement of contingent consideration liability | 2,387 | |
Accretion of redeemable convertible preferred stock to redemption value | $ 644 |
Nature of the Business and Basi
Nature of the Business and Basis of Presentation | 12 Months Ended |
Dec. 31, 2019 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Nature of the Business and Basis of Presentation | 1. Nature of the Business and Basis of Presentation Translate Bio, Inc. (the “Company”) is a clinical-stage messenger RNA (“mRNA”) therapeutics company developing a new class of potentially transformative medicines to treat diseases caused by protein or gene dysfunction. Using its proprietary mRNA therapeutic platform (“MRT platform”), the Company creates mRNA that encodes functional proteins. The Company’s mRNA is designed to be delivered to the target cell where the cell’s own machinery recognizes it and translates it, restoring or augmenting protein function to treat or prevent disease. The Company is primarily focused on applying its MRT platform to treat pulmonary diseases caused by insufficient protein production or where production of proteins can modify disease. The Company is developing MRT5005 for the treatment of cystic fibrosis (“CF”). The Company is conducting a Phase 1/2 clinical trial to evaluate the safety and tolerability of single and multiple-ascending doses of MRT5005. Percent predicted forced expiratory volume in one second (“ppFEV 1 pre-defined one-month mid-dose 1 mid-dose In September 2019, the Company announced its decision to discontinue the development of MRT5201, a liver targeted treatment for ornithine transcarbamylase (“OTC”) deficiency (see Notes 4 and 5). The Company’s decision to discontinue the development of MRT5201 for OTC deficiency was based on data from preclinical studies in 2019, the results of which did not support desired pharmacokinetic and safety profile for advancement of the program. The Company is subject to risks 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 and the ability to secure additional capital to fund operations. Product candidates currently under development will require significant additional research and development efforts, including 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 product development efforts are successful, it is uncertain when, if ever, the Company will realize significant revenue from product sales. The accompanying consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States of America (“GAAP”) and include the accounts of the Company and its two wholly owned subsidiaries, Translate Bio MA, Inc. and Translate Bio Securities Corporation, from their date of incorporation. All intercompany accounts and transactions have been eliminated in consolidation. Acquisition of Shire’s MRT Program In December 2016, the Company entered into an asset purchase agreement (as amended in June 2018, the “Shire Agreement”) with Shire Human Genetic Therapies, Inc. (“Shire”), a subsidiary of Takeda Pharmaceutical Company Ltd., pursuant to which Shire sold equipment to and assigned to the Company all of its rights to certain patent rights, permits, real property leases, contracts, regulatory documentation, books and records, and materials related to Shire’s mRNA therapy platform (the “MRT Program”), including its cystic fibrosis transmembrane conductance regulator program. Reverse Stock Split I mpany effected a one-for-5.5555 reverse stock split of its issued and outstanding sh Sales of Common Stock I On May 3, 2019, the Company issued and sold 5,582,940 shares of its common stock in a private placement at a price per share of $8.50, resulting in gross proceeds of $47.5 million before deducting placement agent fees of $2.8 million and other offering expenses of $0.6 million. On July 3, 2019, the Company filed a universal shelf registration statement on Form S-3 - On July 3, 2019, the Company entered into an Open Market Sale Agreement SM to $50.0 million. The Company has not sold any shares under the Sales Agreement as of March 9, 2020. Sales of common stock through Jefferies may be made by any method that is deemed an “at the market” offering as defined in Rule 415 promulgated under its normal trading and sales practices to sell the common stock based upon the Company’s instructions. The Company is not obligated to make any sales of its common stock under the Sales Agreement. Any shares sold would be pursuant to the 2019 Shelf. Sanofi Pasteur Collaboration and Licensing Agreement In 2018, the Company entered into a collaboration and license agreement with Sanofi Pasteur Inc. (“Sanofi”), the vaccines global business unit of Sanofi S.A., to develop mRNA vaccines for up to five infectious disease pathogens (the “Sanofi Agreement”). Under the Sanofi Agreement, the Company and Sanofi are jointly conducting research and development activities to advance mRNA vaccines and mRNA vaccine platform development during a three-year research term, which may be extended by mutual agreement. Following the research term, the Company is obligated to manufacture clinical product for Sanofi, which the Company estimates may take up to eight years to complete. The Company is eligible to receive up to $805.0 million in payments, including an upfront payment of $45.0 million, which the Company received in 2018; certain development, regulatory and sales-related milestones across several vaccine targets; and option exercise fees if Sanofi exercises its option related to development of vaccines for additional pathogens. The Company is also eligible to receive reimbursable development costs and tiered royalty payments associated with worldwide sales of the developed vaccines, if any (see Note 3). Going Concern In accordance with Accounting Standards Update (“ASU”) No. 2014-15, Disclosure of Uncertainties about an Entity’s Ability to Continue as a Going Concern (Subtopic 205-40) The Company’s financial statements have been prepared on the basis of continuity of operations, realization of assets and the satisfaction of liabilities in the ordinary course of business. Through December 31, 2019, the Company has funded its operations with proceeds from the sale of redeemable convertible preferred stock and the sale of bridge units, which ultimately converted into shares of the Company’s common stock, the proceeds from the IPO, the upfront payment received under the Sanofi Agreement, the proceeds from a private placement of the Company’s common stock and the proceeds from a public offering of the Company’s common stock. The Company has incurred recurring losses and cash outflows from operations since its inception, including net losses of $113.3 million and $97.4 million for the years ended December 31, 2019 and 2018, respectively. In addition, the Company had an accumulated deficit of $359.5 million as of December 31, 2019. The Company expects to continue to generate operating losses for the foreseeable future. As of March 12, 2020, the date of issuance of these consolidated financial statements, the Company expects that its cash, cash equivalents and short-term investments of $188.7 million as of December 31, 2019 will be sufficient to fund its operating expenses and capital expenditure requirements into the second quarter of 2021. The future viability of the Company beyond that point is dependent on the Company’s ability to raise additional capital to finance its operations. Although the Company has been successful in raising capital in the past, there is no assurance that it will be successful in obtaining such additional financing on terms acceptable to the Company, if at all. The Company expects that its expenses will increase in connection with its ongoing business activities. As a result, the Company will need substantial additional funding to support its continuing operations and pursue its growth strategy. Until such time as the Company can generate significant revenue from products sales, if ever, it expects to finance its operations through the sale of equity, debt financing or other capital sources, including collaborations with other companies or other strategic transactions. The Company may be unable to raise additional funds or enter into such other agreements or arrangements when needed on favorable terms, or at all. If the Company is unable to obtain funding, the Company will 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, or the Company may be unable to continue operations. Based on its recurring losses and cash outflows from operations since inception, expectation of continuing operating losses and cash outflows from operations for the foreseeable future and the need to raise additional capital to finance its future operations, the Company concluded that there i s |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 12 Months Ended |
Dec. 31, 2019 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies 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 consolidated financial statements, and the reported amounts of revenues and expenses during the reporting period. Actual results could differ from those estimates. Significant estimates and assumptions reflected in these consolidated financial statements include, but are not limited to, the accrual for research and development expenses, the revenue recognized from collaboration agreements, the valuation of common stock and stock-based awards, the valuation of assets acquired and liabilities assumed in business combinations, and the impairment of identifiable intangible assets and goodwill. Estimates are periodically reviewed in light of changes in circumstances, facts and experience. Changes in estimates are recorded in the period in which they become known. Actual results could differ from those estimates. Cash and Cash Equivalents All highly liquid investments purchased with an original maturity date of three months or less at the date of purchase are considered to be cash equivalents. Cash equivalents consisted of money market funds as of December 31, 2019 and 2018. Investments The Company’s debt security investments are classified as available-for-sale The Company evaluates its investments with unrealized losses for other-than-temporary impairment. When assessing investments for other-than-temporary declines in value, the Company considers such factors as, among other things, how significant the decline in value is as a percentage of the original cost, how long the market value of the investment has been less than its original cost, the Company’s ability and intent to retain the investment for a period of time sufficient to allow for any anticipated recovery in fair value and market conditions in general. If any adjustment to fair value reflects a decline in the value of the investment that the Company considers to be “other than temporary,” the Company reduces the investment to fair value through a charge to the statement of operations. No such adjustments were necessary during the periods presented. The Company’s investments as of December 31, 2019 and 2018 had original maturities of less than one year. Concentrations of Credit Risk Financial instruments that potentially subject the Company to concentrations of credit risk consist principally of cash and cash equivalents as well as short-term investments. Cash, cash equivalents and short-term investments consist of demand deposits, money market funds and U.S. government agency bonds. The Company generally maintains balances in various operating accounts with financial institutions that management believes to be of high credit quality, in amounts that may exceed federally insured limits. The Company has not experienced any losses related to its cash, cash equivalents and short-term investments and does not believe that it is subject to unusual credit risk beyond the normal credit risk associated with commercial banking relationships. Restricted Cash In connection with its operating lease commitments, the Company issued letters of credit collateralized by cash deposits that are classified as restricted cash in the consolidated balance sheets. Restricted cash amounts have been classified as current assets based on the release dates of the restrictions under the letters of credit, which occur annually. 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 lives of each asset. Estimated useful lives are periodically assessed to determine if changes are appropriate. Upon retirement or sale, the related cost and accumulated depreciation and amortization are removed from the accounts and any resulting gain or loss is included in the consolidated statements of operations. Repair and maintenance costs are expensed as incurred. The estimated useful lives of the Company’s property and equipment are as follows: Estimated Useful Life Laboratory equipment 5 years Computer equipment 3 years Office equipment 5 years Leasehold improvements Shorter of lease term or 10 years Costs for capital assets not yet placed into service are capitalized as construction-in-progress Property and equipment 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 an asset group for recoverability, the Company compares the forecasted undiscounted cash flows expected to result from the use and eventual disposition of the asset group to its carrying value. An impairment loss would be recognized when estimated undiscounted future cash flows expected to result from the use and eventual disposition of an asset group are less than its carrying amount. The impairment loss would be based on the excess of the carrying value of the impaired asset group over its fair value, determined based on discounted cash flows using market participant assumptions. The Company recorded an impairment loss on property and equipment during the year ended December 31, 2019 ; , Revenue Recognition The terms of the Company’s collaboration agreements may include consideration such as non-refundable The Company had no revenue prior to the Sanofi Agreement, therefore the adoption of Financial Accounting Standards Board (“FASB”) Accounting Standards Codification (“ASC”) 606, Revenue from Contracts with Customers The Company recognizes the transaction price allocated to upfront license payments as revenue upon delivery of the license to the customer and resulting ability of the customer to use and benefit from the license, if the license is determined to be distinct from the other performance obligations identified in the contract. If the license is considered to not be distinct from other performance obligations, the Company utilizes judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied (i) at a point in time, but only for licenses determined to be distinct from other performance obligations in the contract, or (ii) over time, and, if over time, the appropriate method of measuring progress for purposes of recognizing revenue from license payments. The Company evaluates the measure of progress each reporting period and, if necessary, adjusts the measure of performance and related revenue recognition. The Sanofi Agreement entitles the Company to additional payments upon the achievement of performance-based milestones. These milestones are generally categorized into three types: development milestones, generally based on the advancement of the Company’s pipeline and initiation of clinical trials; regulatory milestones, generally based on the submission, filing or approval of regulatory applications such as a new drug application (“NDA”) in the United States; and sales-based milestones, generally based on meeting specific thresholds of sales in certain geographic areas. The Company is also eligible to receive from Sanofi tiered royalty payments on worldwide net sales of mRNA vaccines. For each collaboration that includes development milestone payments, the Company evaluates whether it is probable that the consideration associated with each milestone will not be subject to a significant reversal in the cumulative amount of revenue recognized. Amounts that meet this threshold are included in the transaction price using the most likely amount method, whereas amounts that do not meet this threshold are considered constrained and excluded from the transaction price until they meet this threshold. Milestones tied to regulatory approval, and therefore not within the Company’s control, are considered constrained until such approval is received. Upfront and ongoing development milestones per its collaboration agreements are not subject to refund if the development activities are not successful. At the end of each subsequent reporting period, the Company re-evaluates catch-up ASC 606 requires the Company to allocate the arrangement consideration on a relative standalone selling price basis for each performance obligation after determining the transaction price of the contract and identifying the performance obligations to which that amount should be allocated. The relative standalone selling price is defined in ASC 606 as the price at which an entity would sell a promised good or service separately to a customer. If other observable transactions in which the Company has sold the same performance obligation separately are not available, the Company is required to estimate the standalone selling price of each performance obligation. Key assumptions to determine the standalone selling price may include forecasted revenues, development timelines, reimbursement rates for personnel costs, discount rates and probabilities of technical and regulatory success. Whenever the Company determines that a contract should be accounted for as a combined performance obligation it will utilize the cost-to-cost cost-to-cost The Company evaluates its collaborative agreements for proper classification in the consolidated statements of operations based on the nature of the underlying activity. Transactions between collaborators recorded in the Company’s consolidated statements of operations are recorded on either a gross or net basis, depending on the characteristics of the collaborative relationship. For revenue generating arrangements where the Company, as a vendor, provides consideration to a licensor or collaborator, as a customer, the Company applies the accounting standard that governs such transactions. This standard addresses the accounting for revenue arrangements where both the vendor and the customer make cash payments to each other for services and/or products. A payment to a customer is presumed to be a reduction of the transaction price unless the Company receives an identifiable benefit for the payment and it can reasonably estimate the fair value of the benefit received. Payments to a customer that are deemed a reduction of the transaction price are recorded first as a reduction of revenue, to the extent of both cumulative revenue recorded to date and probable future revenues, which include any unamortized deferred revenue balances, under all arrangements with such customer, and then as an expense. Payments that are not deemed to be a reduction of the transaction price are recorded as an expense. Consideration that does not meet the requirements to satisfy the above revenue recognition criteria is a contract liability and is recorded as deferred revenue in the consolidated balance sheets. Although the Company follows detailed guidelines in measuring revenue, certain judgments affect the application of its revenue policy. For example, in connection with the Sanofi Agreement, the Company has recorded short-term and long-term deferred revenue on its consolidated balance sheets based on the Company’s best estimate of when such revenue will be recognized. Short-term deferred revenue consists of amounts that are expected to be recognized as revenue in the next 12 months. Amounts that the Company expects will not be recognized within the next 12 months are classified as long-term deferred revenue. The estimate of deferred revenue also reflects management’s estimate of the periods of the Company’s involvement in certain of its collaborations. The Company’s performance obligations under these collaborations consist of participation on steering committees and the performance of other research and development services. In certain instances, the timing of satisfying these obligations can be difficult to estimate. Accordingly, the Company’s estimates may change in the future. Such changes to estimates would result in a change in revenue recognition amounts. If these estimates and judgments change over the course of these agreements, it may affect the timing and amount of revenue that the Company will recognize and record in future periods. At December 31, 2019, the Company had short-term and long-term deferred revenue of $18.1 million and $25.3 million, respectively, related to the Sanofi Agreement. Under ASC 606, the Company will recognize revenue and record a receivable when it fulfills its performance obligations under the Sanofi Agreement. When no further performance obligation is required to be satisfied, the Company will recognize revenue for the portion satisfied and record a receivable. Amounts are recorded as short-term collaboration receivables when the Company’s right to consideration is unconditional. The Company does not assess whether a contract has a significant financing component if the expectation at contract inception is that the period between payment by the customer and the transfer of the promised goods or services to the customer will be one year or less. The Company expenses incremental costs of obtaining a contract as and when incurred if the expected amortization period of the asset that the Company would have recognized is one year or less or the amount is immaterial. At December 31, 2019, the Company has not capitalized any costs to obtain any of its contracts. In-Process The fair value of in-process The fair value of an IPR&D intangible asset is typically determined using an income approach whereby management forecasts the net cash flows expected to be generated by the asset over its estimated useful life. The net cash flows reflect the asset’s stage of completion, the probability of technical success, the projected costs to complete, expected market competition, and an assessment of the asset’s life-cycle. The net cash flows are then adjusted to present value by applying an appropriate discount rate that reflects the risk factors associated with the cash flow streams. Indefinite-lived IPR&D is not subject to amortization, but is tested annually for impairment or more frequently if there are indicators of impairment. The Company tests its indefinite-lived IPR&D annually for impairment on October 1st. In testing indefinite-lived IPR&D for impairment, the Company has the option to first assess qualitative factors to determine whether the existence of events or circumstances would indicate that it is more likely than not that its fair value is less than its carrying amount, or the Company can perform a quantitative impairment analysis to determine the fair value of the indefinite-lived IPR&D without performing a qualitative assessment. Qualitative factors that the Company considers 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 the Company chooses to first assess qualitative factors and the Company determines that it is more likely than not that the fair value of the indefinite-lived IPR&D is less than its carrying amount, the Company would then determine the fair value of the indefinite-lived IPR&D. Under either approach, if the fair value of the indefinite-lived IPR&D is less than its carrying amount, an impairment charge is recognized in the consolidated statements of operations. In September 2019, the Company discontinued development of MRT5201, which was an indicator of impairment, and, as a result, the Company retested the indefinite-lived IPR&D related to the OTC deficiency program for impairment. The Company will not be investing any additional funds in this program and has reallocated all resources previously dedicated to the OTC deficiency program to other programs within the Company. The Company determined that there was no residual value to the indefinite-lived IPR&D related to the OTC deficiency program and, as a result, the Company recorded an impairment charge of $ million during the year ended December 31, 2019, representing the entire value of the indefinite-lived IPR&D related to the OTC deficiency program. During the year ended December 31, 2018, the Company did not recognize any impairment charges related to its definite-lived IPR&D (see Note 4). Definite-lived IPR&D is recorded at fair value and amortized over the greater of economic consumption or on a straight-line basis over its estimated useful life. Definite-lived IPR&D is tested for recoverability whenever events or changes in business circumstances indicate that the carrying amount of the assets may not be fully recoverable. If an impairment review is performed to evaluate an asset group for recoverability, the Company compares the forecasted undiscounted cash flows expected to result from the use and eventual disposition of the asset group to its carrying value. An impairment loss would be recognized when estimated undiscounted future cash flows expected to result from the use and eventual disposition of an asset group are less than its carrying amount. The impairment loss would be based on the excess of the carrying value of the impaired asset group over its fair value, determined based on discounted cash flows using market participant assumptions. Goodwill Goodwill represents the excess of the fair value of the consideration transferred over the fair value of the net tangible and identifiable intangible assets acquired in a business combination. Goodwill is not subject to amortization, but is tested annually for impairment or more frequently if there are indicators of impairment. The Company tests its goodwill annually for impairment on October 1st. The Company has determined that there is a single reporting unit for purposes of testing goodwill for impairment. In testing goodwill for impairment, the Company has the option to first assess qualitative factors to determine whether the existence of events or circumstances would indicate that it is more likely than not that the fair value of the reporting unit was less than its carrying amount, or the Company can perform a two-step two-step two-step 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 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 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 and short-term investments are carried at fair value, determined based on Level 2 inputs in the fair value hierarchy described above (see Note 5). The Company’s contingent consideration liability is carried at fair value, determined based on Level 3 inputs in the fair value hierarchy described above (see Note 5). The carrying values of the Company’s short-term collaboration receivables, prepaid expenses and other current assets, accounts payable, accrued expenses and other short-term liabilities approximate their fair values due to the short-term nature of these assets and liabilities. Segment Information The Company manages its operations as a single operating segment for the purposes of assessing performance and making operating decisions. The Company’s primary focus is on the advancement of the Company’s MRT platform to treat diseases caused by protein or gene dysfunction. Research and Development Costs Costs associated with internal research and development and external research and development services, including drug development and preclinical studies, are expensed as incurred. Research and development expenses include costs for salaries, employee benefits, subcontractors, facility-related expenses, depreciation and amortization, including amortization related to definite-lived IPR&D intangible assets, stock-based compensation, third-party license fees, laboratory supplies, and external costs of outside vendors engaged to conduct discovery, preclinical and clinical development activities and clinical trials as well as to manufacture clinical trial materials, and other costs. The Company recognizes external research and development costs based on an evaluation of the progress to completion of specific tasks using information provided to the Company by its service providers. 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. Such prepaid expenses are recognized as an expense when the services have been performed or the goods have been delivered, or when it is no longer expected that the goods will be delivered or the services rendered. Upfront payments, milestone payments (other than those deemed contingent consideration in a business combination) and annual maintenance fees under license agreements are expensed in the period in which they are incurred. Research and Development Contract Costs and Accruals The Company has entered into various research and development-related contracts with companies both inside and outside of the United States. The related costs are recorded as research and development expenses as incurred. The Company records accruals for estimated ongoing research and development costs. When evaluating the adequacy of the accrued liabilities, the Company analyzes progress of the studies or clinical trials, including the phase or completion of events, invoices received and contracted costs. Significant judgments and estimates are made in determining the accrued balances at the end of any reporting period. Actual results could differ materially from the Company’s estimates. The Company’s historical accrual estimates 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 all stock-based awards granted to employees, directors and, effective January 1, 2019, non-employee Through December 31, 2018, for stock-based awards granted to non-employee non-employee The Company classifies stock-based compensation expense in its consolidated statements of operations 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. Classification and Accretion of Redeemable Convertible Preferred Stock The Company has classified its redeemable convertible preferred stock outside of stockholders’ equity (deficit) because the shares contained certain redemption features that were not solely within the control of the Company. Costs incurred in connection with the issuance of each series of redeemable convertible preferred stock were recorded as a reduction of gross proceeds from issuance. The carrying values of redeemable convertible preferred stock were accreted to their redemption values through a charge to additional paid-in 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. The Company’s only element of other comprehensive income (loss) was unrealized gains (losses) on U.S. government agency bonds, which are classified as available-for-sale-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 statement 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 more-likely-than-not Net Income (Loss) per Share The Company follows the two-class two-class two-class Basic net income (loss) per share attributable to common stockholders is computed by dividing the net income (loss) attributable to common stockholders by the weighted average number of shares of common stock outstanding for the period. Diluted net income (loss) 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 shares of common stock outstanding for the period, including potential dilutive shares of common stock. For purposes of this calculation, outstanding stock options, unvested restricted common stock and redeemable convertible preferred stock are considered potential dilutive shares of common stock. The Company’s preferred stock contractually entitles the holders of such shares to participate in dividends but does not contractually require the holders of such shares to participate in losses of the Company. Accordingly, in periods in which the Company reports a net loss attributable to common stockholders, 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 shares of common stock 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 years ended December 31, 2019 and 2018. Recently Adopted Accounting Pronouncements In February 2016, the FASB issued ASU 2016-02, right-of-use 2016-02 The Company elected the permitted practical expedients within ASC 842, which allowed the Company to not reassess previous accounting conclusions around whether arrangements are or contain leases, and carried forward both the historical classification of leases and the treatment of initial direct costs. In addition, the Company elected to exclude leases with an initial term of one year or less in the recognized ROU assets and lease liabilities. Adoption of the new standard resulted in the recording of ROU assets and related lease liabilities of approximately $10.9 million and $13.0 million, respectively, as of January 1, 2019. The standard did not materially impact the Company’s consolidated net earnings and had no impact on cash flows. Refer to Note 13 for the additional disclosures required by ASC 842. The Company determines if an arrangement is a lease at inception. For leases where the Company is the lessee, ROU assets represent the Company’s right to use an underlying asset for the lease term and lease liabilities represent an obligation to make lease payments arising from the lease. ROU assets and lease liabilities are recognized at the lease commencement date based on the present value of lease payments over the lease term. As most of the Company’s leases do not provide an implicit interest rate, the Company uses its incremental borrowing rate, which are the rates incurred to borrow on a collateralized basis over a term equal to the lease payments in a similar economic environment, in determining the present value of lease payments. The lease terms used to calculate the ROU asset and related lease liability include options to extend or terminate the lease when it is reasonably certain that the Company will exercise that option. Lease expense for operating leases is recognized on a straight-line basis over the lease term as an operating expense. The Company has lease agreements which require payments for lease and non-lease In July 2017, the FASB issued ASU No. 2017-11, Earnings Per Share (Topic 260), Distinguishing Liabilities from Equity (Topic 480), Derivatives and Hedging (Topic 815) I. Accounting for Certain Financial Instruments with Down Round Features II. Replacement of the Indefinite Deferral for Mandatorily Redeemable Financial Instruments of Certain Nonpublic Entities and Certain Mandatorily Redeemable Noncontrolling Interests with a Scope Exception . In June 2018, the FASB issued ASU No. 2018-07, Compensation-Stock Compensation (Topic 718) non-employees. non-employee non-employee Recently Issued Accounting Pronouncements In June 2016, the FASB issued guidance on the Measurement of Credit Losses on Financial Instruments. The guidance requires that credit losses be reported using an expected losses model rather than the incurred losses model that is currently used, and establishes additional disclosures related to credit risks. For available-for-sale 3 In January 2017, the FASB issued ASU No. 2017-04, Intangibles—Goodwill and Other: Simplifying the Test for Goodwill Impairment (Topic 350) In August 2018, the FASB issued ASU No. 2018-13, Fair Value Measurement (Topic 820): Disclosure Framework—Changes to the Disclosure Requirements for Fair Value Measurement In November 2018, the FASB issued ASU No. 2018-18, Collaborative Arrangements (Topic 808) Revenue Recognition Collaborative Arrangements In December 2019, the FASB issued ASU No. 2019-12, Income Taxes-Simplifying the Accounting for Income Taxes step-up |
Collaboration Agreement
Collaboration Agreement | 12 Months Ended |
Dec. 31, 2019 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Sanofi Collaboration and License Agreement | 3. Collaboration Agreement Sanofi Collaboration and License Agreement In 2018, the Company entered into the Sanofi Agreement to develop mRNA vaccines and mRNA vaccine platform development for up to five infectious disease pathogens (the “Licensed Fields”). Under the Sanofi Agreement, the Company and Sanofi have agreed to collaborate to perform certain research and development activities to advance mRNA vaccines and mRNA vaccine platform development during a three-year research term, which may be extended by mutual agreement. Following the research term, the Company is obligated to manufacture clinical product for Sanofi, which the Company estimates may take up to eight years to complete. The collaboration activities will be subject to a collaboration plan to be updated annually. Under the Sanofi Agreement, the Company received an upfront payment of $45.0 million and is eligible for certain potential milestone and option payments, each as further described below. In addition, the Company is eligible to receive from Sanofi tiered royalty payments on worldwide net sales of mRNA vaccines. Under the Sanofi Agreement, the Company and Sanofi created a governance structure, including committees and working groups, to manage the activities under the collaboration. If the Company and Sanofi do not mutually agree on certain decisions, Sanofi would be able to break a deadlock without the Company’s consent. The collaboration includes an estimated budget. Sanofi is responsible for paying reimbursable development costs, including the Company’s employee costs, out-of-pocket Under the terms of the Sanofi Agreement, the Company has granted to Sanofi exclusive, worldwide licenses under applicable patents, patent applications, know-how The Company and Sanofi retain the rights to perform their respective obligations and exercise their respective rights under the Sanofi Agreement, and Sanofi may grant sublicenses to affiliates or third parties. Sanofi has also granted the Company non-exclusive, Sanofi has sole responsibility for all commercialization activities for mRNA vaccines in the Licensed Fields and is obligated to bear all costs in connection with any such commercialization. The Company and Sanofi intend to enter into a supply agreement pursuant to which the Company would be responsible for manufacturing certain non-clinical The Sanofi Agreement provides that the Company is eligible to receive aggregate potential payments of up to $805.0 million from Sanofi, which includes the $45.0 million upfront payment the Company received in 2018, potential milestone payments and potential option exercise payments. Sanofi will also pay the Company $5.0 million with respect to each additional Licensed Field for which it exercises an option. Sanofi has also agreed to pay the Company milestone payments upon the achievement of specified development, regulatory and commercialization milestones. In particular, the Company is entitled to receive development and regulatory milestone payments of up to $63.0 million per Licensed Field and sales milestone payments of up to $85.0 million per Licensed Field. In addition, the Company is entitled to receive a $10.0 million milestone payment from Sanofi following completion of the technology and process transfer. Sanofi has agreed to pay the Company a tiered royalty on worldwide net sales of all mRNA vaccines within each Licensed Field ranging from a high single-digit percentage to a low teens percentage, depending on quarterly net sales by Sanofi, its affiliates and its sublicensees. The royalty paid to the Company can be reduced with respect to a product once the relevant licensed patent rights expire or if additional licensed technology is required, but the royalty payments generally may not fall below the Company’s royalty obligations to third parties plus a royalty of a low single-digit percentage. Royalty payments under the Sanofi Agreement are payable on a product-by-product country-by-country The Sanofi Agreement provides that it will remain in effect until terminated in accordance with its terms. Either the Company or Sanofi may terminate the Sanofi Agreement in its entirety if the other party is subject to certain insolvency proceedings. Either party may terminate the Sanofi Agreement in its entirety or with respect to a particular Licensed Field, country or product if the other party materially breaches the Sanofi Agreement and the breach remains uncured for a specified period, which may be extended in certain circumstances. Sanofi may also terminate the Sanofi Agreement in its entirety or with respect to a particular Licensed Field, country or product for safety reasons or for convenience, in each case after a specified notice period. After termination of the Sanofi Agreement, Sanofi may continue to manufacture and commercialize the terminated products for a specified period of time, subject to Sanofi’s payment obligations. Accounting Under ASC 606 In determining the appropriate amount of revenue to be recognized under ASC 606 , The Company identified the following promised goods or services contained in the Sanofi Agreement: (i) the license it conveyed to Sanofi with respect to the Licensed Fields, (ii) the licensed know-how non-clinical non-cash Under ASC 606, at the end of each reporting period, the Company re-evaluates re-evaluated re-evaluate non-refundable out-of-pocket Under ASC 606, the Company recognized revenue using the cost-to-cost cost-to-cost The following table summarizes the Company’s collaboration revenue (in thousands): Year Ended December 31, 2019 2018 Collaboration revenue $ 7,804 $ 1,420 The following table presents the balance of the Company’s contract liabilities (in thousands): December 31, December 31, Contract liabilities Deferred revenue $ 43,356 $ 44,413 The Company considers the total consideration expected to be earned in the next 12 months for services to be performed as short-term deferred revenue, and consideration that is expected to be earned subsequent to 12 months from the balance sheet date as long-term deferred revenue. The Company expects to complete its obligations and recognize all net revenues from the collaboration over eight years. Revenue recognized from contract liabilities was $1.1 million and $0 during the years ended December 31, 2019 and 2018, respectively. |
Intangible Assets and Goodwill
Intangible Assets and Goodwill | 12 Months Ended |
Dec. 31, 2019 | |
Goodwill and Intangible Assets Disclosure [Abstract] | |
Intangible Assets and Goodwill | 4. Intangible Assets and Goodwill Intangible Assets, Net The acquisition of Shire’s MRT Program was accounted for in accordance with the acquisition method of accounting for business combinations. The total purchase consideration transferred was allocated to the tangible and identifiable intangible assets acquired based on their estimated fair values. The tables below present the Company’s definite-lived intangible assets that are subject to amortization and indefinite-lived intangible assets: December 31, 2019 Estimated Gross Carrying Amount Accumulated Amortization Impairment Charge Net Carrying Amount (In thousands) Definite-lived intangible assets: MRT 8 years $ 45,992 $ (2,747 ) $ — $ 43,245 Indefinite-lived intangible assets: IPR&D—CF Indefinite 42,291 — — 42,291 IPR&D—OTC Indefinite 18,559 — (18,559 ) — Total intangible assets, net $ 106,842 $ (2,747 ) $ (18,559 ) $ 85,536 December 31, 2018 Estimated Gross Carrying Amount Accumulated Amortization Impairment Charge Net Carrying Amount (In thousands) Definite-lived intangible assets: MRT 8 years $ 45,992 $ (397 ) $ — $ 45,595 Indefinite-lived intangible assets: IPR&D—CF Indefinite 42,291 — — 42,291 IPR&D—OTC Indefinite 18,559 — — 18,559 Total intangible assets, net $ 106,842 $ (397 ) $ — $ 106,445 Identifiable intangible assets acquired in the acquisition of Shire’s MRT Program consisted of IPR&D, which included ongoing projects that could further the Company’s preclinical and clinical development activities related to CF, OTC deficiency and other potential rare diseases. As of the date of acquisition, the IPR&D was determined to be indefinite-lived. Upon commencement of the Sanofi Agreement, the IPR&D — MRT intangible asset was reclassified from indefinite-lived to definite-lived intangible assets and the Company began amortization of this intangible asset. Amortization will be recorded over an estimated eight-year period based on an economic consumption model. For the years ended December 31, 2019 and 2018, the Company recorded amortization expense of $2.3 million and $0.4 million, respectively, related to the definite-lived MRT intangible asset. The estimated aggregate amortization expense for each of the five succeeding fiscal years is $9.0 million, $9.9 million, $4.2 million, $2.3 million and $2.5 million for the years ending December 31, 2020, 2021, 2022, 2023 and 2024, respectively. Indefinite-lived IPR&D is not subject to amortization, but is tested annually for impairment or more frequently if there are indicators of impairment. The Company tests its indefinite-lived IPR&D annually for impairment on October 1st. The Company determined that the discontinuation of the development of MRT5201 was an indicator of impairment and , in non-refundable The amounts payable by the Company for incurred costs related to the OTC deficiency program were immaterial as of December 31, 2019. Goodwill The excess of the fair value of the consideration transferred over the fair value of identifiable assets acquired in the acquisition of Shire’s MRT Program was allocated to goodwill in the amount of $21.4 million. There have been no changes to the carrying amount of goodwill during the year ended December 31, 2019. Goodwill is not subject to amortization, but is tested annually for impairment or more frequently if there are indicators of impairment. The Company tests its goodwill annually for impairment on October 1 st During the years ended December 31, 2019 and 2018, the Company did not recognize any impairment charges related to goodwill. |
Fair Value of Financial Assets
Fair Value of Financial Assets and Liabilities | 12 Months Ended |
Dec. 31, 2019 | |
Fair Value Disclosures [Abstract] | |
Fair Value of Financial Assets and Liabilities | 5. Fair Value of Financial Assets and Liabilities The following tables present information about the Company’s financial assets and liabilities measured at fair value on a recurring basis (in thousands): Fair Value Measurements as of December 31, 2019 Using: Level 1 Level 2 Level 3 Total Assets: Money market funds $ — $ 56,591 $ — $ 56,591 U.S. government agency bonds — 104,098 — 104,098 $ — $ 160,689 $ — $ 160,689 Liabilities: Contingent consideration $ — $ — $ 103,655 $ 103,655 $ — $ — $ 103,655 $ 103,655 Fair Value Measurements as of December 31, 2018 Using: Level 1 Level 2 Level 3 Total Assets: Money market funds $ — $ 23,318 $ — $ 23,318 U.S. government agency bonds — 88,904 — 88,904 $ — $ 112,222 $ — $ 112,222 Liabilities: Contingent consideration $ — $ — $ 103,642 $ 103,642 $ — $ — $ 103,642 $ 103,642 During the years ended December 31, 2019 and 2018, there were no transfers between Level 1, Level 2 and Level 3. Cash equivalents as of December 31, 2019 and 2018 consisted of money market funds totaling $56.6 million and $23.3 million, respectively. The money market funds were valued using inputs observable in active markets for similar securities, which represent a Level 2 measurement in the fair value hierarchy. The Company’s short-term investments as of December 31, 2019 and 2018 consisted of U.S. government agency bonds and were classified as available-for-sale Valuation of Contingent Consideration The contingent consideration liability related to the acquisition of Shire’s MRT Program in 2016 was classified as Level 3 measurement within the fair value hierarchy. The Company may be required to pay future consideration to Shire contingent upon the achievement of potential future milestones and earnout payments. The fair value of the liability to make potential future milestone and earnout payments was estimated by the Company at each reporting date based, in part, on the results of a third-party valuation using a discounted cash flow analysis based on various assumptions, including the probability of achieving specified events, discount rates, and the period of time until earnout payments are payable and the conditions triggering the milestone payments are met. The actual settlement of contingent consideration could differ from current estimates based on the actual occurrence of these specified events. The following tables presents the unobservable inputs and fair value of the components of the contingent consideration (dollar amounts in thousands): Unobservable Inputs Fair Value at Projected Year of Payment December 31, December 31, Earnout payments 2026 – $ 96,097 $ 94,999 Milestone payments 2026 – 7,558 8,643 $ 103,655 $ 103,642 The discount rate used in the third-party valuation was 13.5% and 14.5% as of December 31, 2019 and 2018, respectively. The following table presents a roll-forward of the total acquisition-related contingent consideration liability (in thousands): Fair Value Balance as of December 31, 2018 $ 103,642 Discontinuation of MRT5201 (23,174 ) Increase in fair value of contingent consideration 23,187 Balance as of December 31, 2019 $ 103,655 The net increase in the fair value of contingent consideration during the year ended December 31, 2019 was primarily due to the continued progress of MRT5005, the time value of money due to the passage of time and a decrease in the discount rate. The increase was offset by a decrease in the fair value of contingent consideration due to a decision to discontinue the development of MRT5201 in September 2019, which resulted in the removal of the $23.2 million in contingent consideration liability related to this program. |
Property and Equipment, Net
Property and Equipment, Net | 12 Months Ended |
Dec. 31, 2019 | |
Property, Plant and Equipment [Abstract] | |
Property and Equipment, Net | 6. Property and Equipment, Net Property and equipment, net consisted of the following (in thousands): December 31, December 31, Laboratory equipment $ 9,044 $ 7,012 Computer equipment 779 686 Office equipment 883 836 Leasehold improvements 5,635 5,635 Construction in progress 3,460 959 19,801 15,128 Less: Accumulated depreciation and amortization (7,262 ) (4,883 ) $ 12,539 $ 10,245 Depreciation and amortization expense related to property and equipment was $2.4 million for the each of the years ended December 31, 2019 and 2018. |
Accrued Expenses
Accrued Expenses | 12 Months Ended |
Dec. 31, 2019 | |
Payables and Accruals [Abstract] | |
Accrued Expenses | 7. Accrued Expenses Accrued expenses consisted of the following (in thousands): December 31, December 31, Accrued employee compensation and benefits $ 3,547 $ 2,933 Accrued external research and development expenses 1,763 1,901 Accrued consultant and professional fees 1,390 977 Other 372 736 $ 7,072 $ 6,547 |
Redeemable Convertible Preferre
Redeemable Convertible Preferred Stock | 12 Months Ended |
Dec. 31, 2019 | |
Temporary Equity Disclosure [Abstract] | |
Redeemable Convertible Preferred Stock | 8. Redeemable Convertible Preferred Stock As of December 31, 2017, the Company had 142,288,292 shares of redeemable convertible preferred stock issued and outstanding which were redeemable and convertible by the holders under specified conditions. The redeemable convertible preferred stock was classified outside of stockholders’ equity (deficit) because the shares contained redemption features that were not solely within the control of the Company. Upon the closing of the Company’s IPO i |
Common Stock
Common Stock | 12 Months Ended |
Dec. 31, 2019 | |
Equity [Abstract] | |
Common Stock | 9. Common Stock On July 2, 2018, the Company filed its restated certificate of incorporation, which authorizes the Company to issue up to 200,000,000 shares of common stock, $0.001 par value per share. As of December 31, 2019 and 2018, there were 60,022,067 and 45,139,955 shares of common stock issued and outstanding, respectively. Each share of common stock entitles the holder to one vote for each share of common stock held. Common stockholders are entitled to receive dividends, as declared by the Company’s Board of Directors (the “Board of Directors”). These dividends are subject to the preferential dividend rights of the holders of the Company’s preferred stock. Through December 31, 2018 and 2017, no cash dividends have been declared or paid. |
Incentive Stock Options and Res
Incentive Stock Options and Restricted Stock | 12 Months Ended |
Dec. 31, 2019 | |
Disclosure of Compensation Related Costs, Share-based Payments [Abstract] | |
Incentive Stock Options and Restricted Stock | 10. Incentive Stock Options and Restricted Stock 2018 Equity Incentive Plan On March 7, 2018, the Board of Directors, subject to stockholder approval, adopted, and on June 15, 2018, its stockholders approved, the 2018 Equity Incentive Plan (the “2018 Plan”), which became effective on June 27, 2018. The 2018 Plan provides for the grant of incentive stock options, non-qualified The number of shares initially reserved for issuance under the 2018 Plan is the sum of 2,512,187, plus the number of shares (up to 1,013,167 shares) equal to the sum of (i) the number of shares remaining available for issuance under the 2016 Stock Incentive Plan, as amended, (the “2016 Plan”), upon the effectiveness of the 2018 Plan, which was 360,514 shares, and (ii) the number of shares of common stock subject to outstanding awards under the 2016 Plan that expire, terminate or are otherwise surrendered, canceled, 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 2018 Plan will automatically increase on the first day of each fiscal year, beginning with the fiscal year ending December 31, 2019 and continuing for each fiscal year until, and including, the fiscal year ending December 31, 2028, by an amount equal to the lowest of (i) 3,349,582 shares, (ii) 4% of the outstanding shares of common stock on such date and (iii) an amount determined by the Board of Directors. Accordingly, on January 1, 2019, the number of shares of common stock that may be issued under that 2018 Plan increased by 1,805,598 shares of common stock and through December 31, 2019, a total of 151,548 shares issued under the 2016 Plan have been cancelled for a total of 4,829,847 shares of common stock reserved for issuance under the 2018 Plan as of December 31, 2019. The shares of common stock underlying any awards that are forfeited, canceled, 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 2018 Plan will be added back to the shares of common stock available for issuance under the 2018 Plan. The 2018 Plan is administered by the Board of Directors. The exercise prices, vesting periods and other restrictions are determined at the discretion of the Board of Directors, except that the exercise price per share of options may not be less than 100% of the fair market value of the common stock on the date of grant. Stock options awarded under the 2018 Plan expire 10 years after the grant date, unless the Board of Directors sets a shorter term. Awards granted to employees, officers, members of the Board of Directors and consultants typically vest over a period of one to four years. Typically, unvested stock options are forfeited upon the recipient ceasing to provide services to the Company. 2018 Employee Stock Purchase Plan On March 7, 2018, the Board of Directors, subject to stockholder approval, adopted, and on June 15, 2018, its stockholders approved the 2018 Employee Stock Purchase Plan (the “2018 ESPP”), which became effective on June 27, 2018. A total of 418,697 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 2018 ESPP will automatically increase on the first day of each fiscal year, beginning with the fiscal year commencing on January 1, 2019 and continuing for each fiscal year until, and including, the fiscal year commencing on January 1, 2029, by an amount equal to the lowest of (i) 837,395 shares, (ii) 1% of the outstanding shares of common stock on such date and (iii) an amount determined by the Board of Directors. Accordingly, on January 1, 2019, the number of shares of common stock that may be issued under the 2018 ESPP increased by 451,399 shares for a total of 870,096 shares of common stock reserved for issuance under this plan. As of December 31, 2019, no shares had been issued under the 2018 ESPP. 2016 Stock Incentive Plan The 2016 Plan provides for the grant of stock options, stock appreciation rights, restricted stock and restricted stock units. Shares that are expired, terminated, surrendered or canceled under the 2016 Plan without having been exercised will be available for future grants of awards under the 2018 Plan. In addition, shares of common stock that are tendered to the Company by a participant to exercise an award are added to the number of shares of common stock available for the grant of awards under the 2018 Plan. The 2016 Plan is administered by the Board of Directors. The exercise prices, vesting periods and other restrictions were determined at the discretion of the Board of Directors, except that the exercise price per share of options could not be less than 100% of the fair market value of the common stock on the date of grant. Stock options awarded under the 2016 Plan expire 10 years after the grant date, unless the Board of Directors set a shorter term. Stock options and restricted stock granted to employees, officers, members of the Board of Directors and consultants typically vest over a four-year period. Upon the effectiveness of the 2018 Plan on June 27, 2018, no further awards will be made under the 2016 Plan, but awards outstanding under the 2016 Plan will continue to be governed by their existing terms. Stock Options The following table summarizes the Company’s stock option activity since December 31, 2018 (in thousands, except share and per share amounts): Number of Shares Weighted Average Exercise Price Weighted Average Remaining Contractual Term Intrinsic Value (in years) Outstanding as of December 31, 2018 6,236,006 $ 7.78 8.74 $ 1,104 Granted 2,825,200 $ 8.55 Exercised (233,549 ) $ 6.48 Forfeited (181,279 ) $ 7.89 Outstanding as of December 31, 2019 8,646,378 $ 8.06 8.42 $ 3,687 Exercisable as of December 31, 2019 3,457,794 $ 7.67 7.96 $ 2,234 Vested and expected to vest as of December 31, 2019 8,646,378 $ 8.06 8.42 $ 3,687 The aggregate intrinsic value of options is calculated as the difference between the exercise price of the options and the fair value of the Company’s common stock for those options that had exercise prices lower than the fair value of the Company’s common stock. The aggregate intrinsic value of stock options exercised during the years ended December 31, 2019 and 2018 was $0.6 million and $0.1 million, respectively. The weighted average grant-date fair value per share of stock options granted was $5.59 and $5.95 during the years ended December 31, 2019 and 2018, respectively. Stock Option Valuation The fair value of stock option grants is estimated using the Black-Scholes option-pricing model. The Company completed its IPO in June 2018 and therefore lacks company-specific historical and implied volatility information. As a result, 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 expected term of stock options granted to non-employees is equal to the contractual term of the option award. 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. The following table presents, on a weighted average basis, the assumptions used in the Black-Scholes option-pricing model to determine the grant-date fair value of stock options granted to employees and directors: Year Ended December 31, 2019 2018 Risk-free interest rate 2.36 % 2.81 % Expected term (in years) 6.0 6.0 Expected volatility 73.0 % 75.5 % Expected dividend yield 0 % 0 % Restricted Common Stock The following table summarizes the Company’s restricted stock activity since December 31, 2018: Number of Shares Weighted Average Grant-Date Fair Value Unvested restricted common stock outstanding as of December 31, 2018 219,148 $ 1.27 Forfeited restricted common stock (1,783 ) $ 1.28 Vested restricted common stock (183,197 ) $ 1.27 Unvested restricted common stock outstanding as of December 31, 2019 34,168 $ 1.28 Stock-Based Compensation Stock-based compensation expense was classified in the consolidated statements of operations as follows (in thousands): Year Ended December 31, 2019 2018 Research and development expenses $ 4,786 $ 3,756 General and administrative expenses 6,548 3,577 $ 11,334 $ 7,333 Included in general and administrative stock-based compensation expense during the year ended December 31, 2019 is $0.8 million related to the issuance of 67,406 shares of common stock in connection with a former employee letter agreement. As of December 31, 2019, total unrecognized compensation cost related to the unvested stock-based awards was $24.3 million, which is expected to be recognized over weighted average periods of 2.4 years. |
Income Taxes
Income Taxes | 12 Months Ended |
Dec. 31, 2019 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | 11. Income Taxes During the years ended December 31, 2019 and 2018, the Company recognized an income tax benefit of $ 0.5 million and $5.6 million, respectively, which resulted from a reduction in the deferred tax liabilities recorded as part of the Company’s acquisition of the MRT Program. All of the Company’s operating losses since inception have been generated in the United States . A reconciliation of the U.S. federal statutory income tax rate to the Company’s effective income tax rate is as follows: Year Ended December 31, 2019 2018 U.S. federal statutory income tax rate (21.0 )% (21.0 )% State income taxes, net of federal benefit (6.6 ) (6.9 ) Research and development tax credits and orphan drug credit (6.8 ) (5.5 ) Stock-based compensation 0.5 0.5 Other permanent differences 0.2 0.3 Change in deferred tax asset valuation allowance 33.6 27.7 Other (0.3 ) (0.4 ) Effective income tax rate (0.4 )% (5.3 )% Components of the Company’s deferred tax assets and liabilities were a s December 31, 2019 2018 Deferred tax assets: Net operating loss carryforwards $ 61,460 $ 50,917 Research and development tax credit and orphan drug credit carryforwards 21,830 13,581 Intangibles 3,607 — Deferred revenue 11,845 — Depreciation and amortization 2,253 2,380 Accrued expenses and other 5,354 2,894 Total deferred tax assets 106,349 69,772 Deferred tax liabilities: Indefinite-lived intangible assets — (2,109 ) Total deferred tax liabilities — (2,109 ) Valuation allowance (106,346 ) (68,143 ) Net deferred tax assets (liabilities) $ 3 $ (480 ) As of December 31, 2019, the Company had federal net operating loss carryforwards of $229.3 million, of which $122.1 million will, if not utilized, begin to expire in 2031, and state net operating loss carryforwards of $210.6 million, which will, if not utilized, begin to expire in 2031. Federal net operating loss carryforwards generated after 2017 will be carried forward indefinitely and could be used to offset up to 80% of taxable income of each future tax year . As of December 31, 2019, the Company had federal and state research and development tax credits carryforwards of $6.5 million and $2.7 million, respectively, which will, if not utilized, begin to expire in 0.3 . Utilization of the net operating loss carryforwards and research and development tax credit carryforwards may be subject to a substantial annual limitation under Section 382 of the Internal Revenue Code of 1986, as amended, due to ownership changes that have occurred previously or that could occur in the future. These ownership changes may limit the amount of carryforwards that can be utilized annually to offset future taxable income. In general, an ownership change, as defined by Section 382, results from transactions increasing the ownership of certain stockholders or public groups in the stock of a corporation by more than 50% over a three-year tax-exempt In addition, the Company has not conducted a study of its research and development tax credit carryforwards. This study may result in an adjustment to the Company’s research and development tax credit carryforwards. Until a study is completed and any adjustment is known, no amounts are being presented as an uncertain tax position. The Company has evaluated the positive and negative evidence bearing upon its ability to realize the deferred tax assets. Management has considered the Company’s history of cumulative net losses incurred since inception and its lack of commercialization of any products or generation of any revenue from product sales since inception and has concluded that it is more likely than not that the Company will not realize all of the benefits of the deferred tax assets. At December 31, 2019, the Company maintained a full valuation allowance against its net deferred tax assets with the exception of alternative minimum tax credit carryforwards, which will be fully refundable. At December 31, 2018, the Company maintained a full valuation allowance with the exception of $0.8 million related primarily to indefinite-lived net operating loss carryforwards. Management reevaluates the positive and negative evidence at each reporting period . Changes in the valuation allowance for deferred tax assets during the years ended December 31, 2019 and 2018 related primarily to the increase in net operating loss carryforwards and research and development tax credit carryforwards in 2019 and 2018, and were as follows (in thousands): Year Ended December 31, 2019 2018 Valuation allowance at beginning of year $ (68,143 ) $ (39,605 ) Increases recorded to income tax provision (38,203 ) (28,538 ) Valuation allowance at end of year $ (106,346 ) $ (68,143 ) The Company had not recorded any amounts for unrecognized tax benefits as of December 31, 2019 and 2018. The Company files income tax returns in the U.S. and Massachusetts. The federal and state returns are generally subject to tax examinations for the tax years ended December 31, 2016 to the present. There are currently no pending tax examinations. To the extent the Company has tax attribute carryforwards, the tax years in which the attribute was generated may still be adjusted upon examination by the Internal Revenue Service and state tax authorities to the extent utilized in a future or prior period. The Company’s policy is to record interest and penalties related to income taxes as part of its income tax provision. |
Net Loss per Share
Net Loss per Share | 12 Months Ended |
Dec. 31, 2019 | |
Earnings Per Share [Abstract] | |
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): Year Ended December 31, 2019 2018 Numerator: Net loss $ (113,293 ) $ (97,395 ) Accretion of redeemable convertible preferred stock to redemption value — (644 ) Net loss attributable to common stockholders $ (113,293 ) $ (98,039 ) Denominator: Weighted average common shares outstanding—basic and diluted 51,445,539 26,945,508 Net loss per share attributable to common stockholders—basic and diluted $ (2.20 ) $ (3.64 ) The Company excluded 124,697 shares and 372,301 shares of restricted common stock, presented on a weighted average basis, from the calculations of basic net loss per share attributable to common stockholders for the years ended December 31, 2019 and 2018, respectively, because those shares had not vested. The Company’s potentially dilutive securities, which include stock options, unvested restricted common stock and redeemable convertible preferred stock, have been excluded from the computation of diluted net loss per share attributable to common stockholders as the effect would be to reduce the net loss per share. Therefore, the weighted average number of shares of common stock 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 shares of common stock, 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: December 31, 2019 2018 Options to purchase common stock 8,646,378 6,236,006 Unvested restricted common stock 34,168 219,148 8,680,546 6,455,154 |
Leases
Leases | 12 Months Ended |
Dec. 31, 2019 | |
Leases [Abstract] | |
Leases | 13. Leases The Company is a lessee under two operating leases comprising a commercial real estate lease and an equipment lease. Real Estate Lease In June 2017, the Company entered into an operating lease for office and laboratory space at its headquarters in Lexington, Massachusetts. The Company occupies approximately 59,000 square feet of space under a 10-year Equipment Lease In March 2018, the Company entered into an operating lease for communications equipment for use at its office and laboratory space in Lexington, Massachusetts. The term of the lease is five years, expiring in March 2023. Accounting Under ASC 842 The Company excludes leases with an initial term of one year or less in the recognized ROU assets and lease liabilities. The Company recognizes lease expense for these leases on a straight-line basis over the lease term. For lease agreements entered into or reassessed after the adoption of ASC 842, lease and non-lease The components of lease cost were as follows (dollar amounts in thousands): Year Ended Lease cost Operating lease cost ̥$ 2,692 Short-term lease cost — Total lease cost $ 2,692 Other information Operating cash flows from operating leases $ 2,583 Operating lease liabilities arising from obtaining right-of-use assets — Weighted-average remaining lease term 8 years Weighted-average discount rate 17.5 % During the year ended December 31, 2018, the Company recorded rent expense of $3.0 million. As of December 31, 2019, maturities of operating lease liabilities are as follows (in thousands): December 31, 2019 2020 $ 2,659 2021 2,737 2022 2,818 2023 2,860 2024 2,937 2025 and thereafter 10,160 Total future minimum lease payments 24,171 Less: imputed interest (11,557 ) Present value of lease liabilities $ 12,614 As of December 31, 2018, minimum rental commitments under the real estate lease was as follows (in thousands): December 31, 2018 2019 $ 2,534 2020 2,610 2021 2,688 2022 2,769 2023 2,852 2024 and thereafter 13,096 Total future minimum lease payments $ 26,549 As most of the Company’s leases do not provide an implicit rate, the Company uses its incremental borrowing rate which are the rates incurred to borrow on a collateralized basis over a term equal to the lease payments in a similar economic environment in determining the present value of lease payments. The Company used the incremental borrowing rate on January 1, 2019 for operating leases that commenced prior to that date. Suite Retention and Development Agreement In September 2019, the Company entered into a suite retention and development agreement with Albany Molecular Research, Inc. (“AMRI”) under which a series of cleanroom suites will be built at AMRI’s manufacturing facility (“AMRI Agreement”) in accordance with the Company’s objectives. The Company will have exclusive use of the space once the build-out build-out build-out build-out (“Build-Out Build-Out Build-Out Build-Out build-out build-out build-out |
Commitments and Contingencies
Commitments and Contingencies | 12 Months Ended |
Dec. 31, 2019 | |
Commitments and Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 14. Commitments and Contingencies Research, Supply and License Agreements Roche Master Supply Agreement The Company is a party to a master supply agreement with Roche Diagnostics Corporation (“Roche”) pursuant to which Roche will custom manufacture certain products for the Company. The agreement requires the Company to purchase from Roche specified manufactured products and the related raw materials in an amount equal to the greater of (i) quantities of raw materials in the Company’s annual forecast to be purchased or (ii) 80% of the Company’s demand for products as the same or similar type (the “Purchase Commitment”). In June 2017, the Company exercised its option under the agreement to extend the agreement through December 31, 2024. In September 2018, the Company and Roche amended the agreement to remove and replace the Purchase Commitment for certain manufactured products and related raw materials supplied by Roche. The agreement, as amended, specifies a minimum purchase requirement for certain custom manufactured products. As of December 31, 2019, the Company’s purchase commitments under the agreement totaled $14.4 million, with $0.5 million committed as payments in 2020 and $3.5 million committed as payments each year from 2021 to 2024. Included in accounts payable in the Company’s consolidated balance sheets as of December 31, 2019 was $10.1 million related to this agreement. Research and development expenses related to this agreement totaled $12.5 million and $5.0 million during the years ended December 31, 2019 and 2018, respectively. MIT Research Agreement In September 2019, the Company entered into a research agreement with the Massachusetts Institute of Technology (“MIT”) pursuant to which the Company is obligated to reimburse MIT up to $4.1 million for specified direct and indirect costs to be incurred from January 2020 through December 2022 for specified research activities conducted for the Company (the “2019 MIT Agreement”). As of December 31, 2019, the Company had paid MIT $0.3 million towards the total committed amount. There were no research and development expenses related to this agreement during the year ended December 31, 2019. The 2019 MIT Agreement expires in December 2022 and may be extended thereafter by mutual agreement of the parties. In November 2016, the Company entered into a research agreement with MIT pursuant to which the Company was obligated to reimburse MIT in an amount up to $3.1 million for specified direct and indirect costs incurred through October 2019 in specified research activities conducted for the Company (the “2016 MIT Agreement”). As of December 31, 2019 and 2018, the Company had paid MIT $3.1 million and $2.5 million, respectively, of the total committed amount. As amended, the 2016 MIT Agreement expired in October 2019. Research and development expenses related to this agreement totaled $0.7 million and $1.2 million during the years ended December 31, 2019 and 2018, respectively. MIT Exclusive Patent License Agreement The Company is a party to an exclusive patent license agreement with MIT pursuant to which the Company received an exclusive license under the licensed patent rights to develop, manufacture and commercialize any product containing both (i) any RNA sequences, including mRNA, that encode a protein or peptide suitable for human therapeutic use which may include operably linked non-coding non-coding The Company has the right to grant sublicenses under this license. The patent rights licensed to the Company by MIT include claims that cover the Company’s customized lipid-based nanoparticles used for delivery of coding RNA components in its MRT platform, including products that may be developed under the Company’s collaboration with Sanofi. Under the license agreement, the Company is obligated to make annual license maintenance payments to MIT, payable on January 1 of each calendar year, of up to $0.2 million, which may be credited against royalties subsequently due on net sales of licensed products earned in the same calendar year. The Company paid annual license maintenance fees of $0.2 million and $0.1 million, to MIT during the years ended December 31, 2019 and 2018, respectively. The Company is also obligated to make milestone payments to MIT aggregating up to $1.375 million upon the achievement of specified clinical and regulatory milestones with respect to each licensed product and $1.250 million upon the Company’s first commercial sale of each licensed product, and to pay royalties of a low single-digit percentage to MIT based on the Company’s, and any of its affiliates’ and sublicensees’, net sales of licensed products. The royalties are payable on a product-by-product country-by-country last-to-expire MIT-licensed paid the Company’s The agreement MIT has the right to terminate the agreement if the Company fails to pay amounts when due or otherwise materially breaches the agreement and fails to cure such nonpayment or breach within specified cure periods or in the event the Company ceases to carry on its business related to the agreement. In the event of a termination due to the Company’s breach caused by a due diligence failure of a licensed product, but where the Company has fulfilled its obligations with respect to a different licensed product, MIT may not terminate the agreement with respect to the different licensed product. MIT may immediately terminate the agreement if the Company or any of its affiliates brings specified patent challenges against MIT or assists others in bringing a patent challenge against MIT. The Company has the right to terminate the agreement for its convenience at any time on three months’ prior written notice to MIT and payment of all amounts due to MIT through the date of termination. The Company’s patent rights, and the rights of its affiliates and sublicensees, in specified licensed products may also terminate if the Company, its affiliates or MIT receives a request from a third party to develop such licensed product for which the Company is unable to, within nine months of receiving notice of any such request, either demonstrate that the Company has initiated a fully funded project for the commercial development of such licensed product, and provide a business plan with acceptable milestones; demonstrate that the licensed product proposed by such third party would be competitive with a licensed product for which the Company has initiated a fully funded project; or enter into a sublicense agreement with such third party on commercially reasonable terms, and, in each case, MIT, in its sole discretion, grants a license to such third party for the specified patent rights. Research and development expenses related to this agreement totaled $0.2 million and $0.1 million during the years ended December 31, 2019 and 2018, respectively. As of December 31, 2019 and 2018, amounts payable by the Company related to this agreement totaled $0 and $0.7 million, respectively. Indemnification Agreements In the ordinary course of business, the Company may provide indemnification of varying scope and terms to vendors, lessors, 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 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. To date, the Company has not incurred any material costs as a result of such indemnifications. The Company does not believe that the outcome of any claims under indemnification arrangements will have a material effect on its financial position, results of operations or cash flows, and it has not accrued any liabilities related to such obligations in its consolidated financial statements as of December 31, 2019 and 2018. Legal Proceedings The Company is not a party to any litigation and does not have contingency reserves established for any litigation liabilities. |
Related Party Transactions
Related Party Transactions | 12 Months Ended |
Dec. 31, 2019 | |
Related Party Transactions [Abstract] | |
Related Party Transactions | 15. Related Party Transactions Private Placement In connection with a private placement of the Company’s common stock in May 2019, entities affiliated with Baupost Group, L.L.C. (“Baupost”), a substantial stockholder, purchased 2,352,941 shares of the Company’s common stock at a price per share of $8.50 for an aggregate purchase price of $20.0 million. Public Offering In connection with a public offering of the Company’s common stock in September 2019, Baupost purchased 5,000,000 shares of the Company’s common stock at a price per share of $10.00 for an aggregate purchase price of $50.0 million. |
Benefit Plans
Benefit Plans | 12 Months Ended |
Dec. 31, 2019 | |
Retirement Benefits [Abstract] | |
Benefit Plans | 16. Benefit Plans The Company has established a defined-contribution retirement plan under Section 401(k) of the Code. This plan covers all employees who meet minimum age and service requirements and allows participants to defer a portion of their annual compensation on a pre-tax |
Selected Quarterly Financial In
Selected Quarterly Financial Information (Unaudited) | 12 Months Ended |
Dec. 31, 2019 | |
Selected Quarterly Financial Information [Abstract] | |
Selected Quarterly Financial Information | 17. Selected Quarterly Financial Information (Unaudited) The following table contains quarterly financial information for 2019 and 2018 (in thousands, except per share amounts). The Company believes that the following information reflects all normal recurring adjustments necessary for a fair statement of the information for the periods presented. The operating results for any quarter are not necessarily indicative of results for any future period. 2019 First Quarter Second Quarter Third Quarter Fourth Quarter Total Collaboration revenue $ 1,474 $ 1,174 $ 1,266 $ 3,890 $ 7,804 Total operating expenses 35,679 29,364 22,901 35,629 123,573 Loss from operations (34,205 ) (28,190 ) (21,635 ) (31,739 ) (115,769 ) Net loss (33,198 ) (27,832 ) (21,227 ) (31,036 ) (113,293 ) Net loss per share applicable to common stockholders—basic and diluted $ (0.74 ) $ (0.57 ) $ (0.41 ) $ (0.48 ) $ (2.20 ) 2018 First Quarter Second Quarter Third Quarter Fourth Quarter Total Collaboration revenue $ — $ — $ 238 $ 1,182 $ 1,420 Total operating expenses 22,389 29,062 45,719 8,480 105,650 Loss from operations (22,389 ) (29,062 ) (45,481 ) (7,298 ) (104,230 ) Net loss (21,209 ) (27,503 ) (42,646 ) (6,037 ) (97,395 ) Net loss per share applicable to common stockholders—basic and diluted $ (2.35 ) $ (3.04 ) $ (0.97 ) $ (0.13 ) $ (3.64 ) During the three months ended December 31, 2018, the fair value of contingent consideration decreased by $14.6 million as a result of an increase in the discount rate. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 12 Months Ended |
Dec. 31, 2019 | |
Accounting Policies [Abstract] | |
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 consolidated financial statements, and the reported amounts of revenues and expenses during the reporting period. Actual results could differ from those estimates. Significant estimates and assumptions reflected in these consolidated financial statements include, but are not limited to, the accrual for research and development expenses, the revenue recognized from collaboration agreements, the valuation of common stock and stock-based awards, the valuation of assets acquired and liabilities assumed in business combinations, and the impairment of identifiable intangible assets and goodwill. Estimates are periodically reviewed in light of changes in circumstances, facts and experience. Changes in estimates are recorded in the period in which they become known. Actual results could differ from those estimates. |
Cash and Cash Equivalents | Cash and Cash Equivalents All highly liquid investments purchased with an original maturity date of three months or less at the date of purchase are considered to be cash equivalents. Cash equivalents consisted of money market funds as of December 31, 2019 and 2018. |
Investments | Investments The Company’s debt security investments are classified as available-for-sale The Company evaluates its investments with unrealized losses for other-than-temporary impairment. When assessing investments for other-than-temporary declines in value, the Company considers such factors as, among other things, how significant the decline in value is as a percentage of the original cost, how long the market value of the investment has been less than its original cost, the Company’s ability and intent to retain the investment for a period of time sufficient to allow for any anticipated recovery in fair value and market conditions in general. If any adjustment to fair value reflects a decline in the value of the investment that the Company considers to be “other than temporary,” the Company reduces the investment to fair value through a charge to the statement of operations. No such adjustments were necessary during the periods presented. The Company’s investments as of December 31, 2019 and 2018 had original maturities of less than one year. |
Concentrations of Credit Risk | Concentrations of Credit Risk Financial instruments that potentially subject the Company to concentrations of credit risk consist principally of cash and cash equivalents as well as short-term investments. Cash, cash equivalents and short-term investments consist of demand deposits, money market funds and U.S. government agency bonds. The Company generally maintains balances in various operating accounts with financial institutions that management believes to be of high credit quality, in amounts that may exceed federally insured limits. The Company has not experienced any losses related to its cash, cash equivalents and short-term investments and does not believe that it is subject to unusual credit risk beyond the normal credit risk associated with commercial banking relationships. |
Restricted Cash | Restricted Cash In connection with its operating lease commitments, the Company issued letters of credit collateralized by cash deposits that are classified as restricted cash in the consolidated balance sheets. Restricted cash amounts have been classified as current assets based on the release dates of the restrictions under the letters of credit, which occur annually. |
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 lives of each asset. Estimated useful lives are periodically assessed to determine if changes are appropriate. Upon retirement or sale, the related cost and accumulated depreciation and amortization are removed from the accounts and any resulting gain or loss is included in the consolidated statements of operations. Repair and maintenance costs are expensed as incurred. The estimated useful lives of the Company’s property and equipment are as follows: Estimated Useful Life Laboratory equipment 5 years Computer equipment 3 years Office equipment 5 years Leasehold improvements Shorter of lease term or 10 years Costs for capital assets not yet placed into service are capitalized as construction-in-progress Property and equipment 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 an asset group for recoverability, the Company compares the forecasted undiscounted cash flows expected to result from the use and eventual disposition of the asset group to its carrying value. An impairment loss would be recognized when estimated undiscounted future cash flows expected to result from the use and eventual disposition of an asset group are less than its carrying amount. The impairment loss would be based on the excess of the carrying value of the impaired asset group over its fair value, determined based on discounted cash flows using market participant assumptions. The Company recorded an impairment loss on property and equipment during the year ended December 31, 2019 ; , |
Revenue Recognition | Revenue Recognition The terms of the Company’s collaboration agreements may include consideration such as non-refundable The Company had no revenue prior to the Sanofi Agreement, therefore the adoption of Financial Accounting Standards Board (“FASB”) Accounting Standards Codification (“ASC”) 606, Revenue from Contracts with Customers The Company recognizes the transaction price allocated to upfront license payments as revenue upon delivery of the license to the customer and resulting ability of the customer to use and benefit from the license, if the license is determined to be distinct from the other performance obligations identified in the contract. If the license is considered to not be distinct from other performance obligations, the Company utilizes judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied (i) at a point in time, but only for licenses determined to be distinct from other performance obligations in the contract, or (ii) over time, and, if over time, the appropriate method of measuring progress for purposes of recognizing revenue from license payments. The Company evaluates the measure of progress each reporting period and, if necessary, adjusts the measure of performance and related revenue recognition. The Sanofi Agreement entitles the Company to additional payments upon the achievement of performance-based milestones. These milestones are generally categorized into three types: development milestones, generally based on the advancement of the Company’s pipeline and initiation of clinical trials; regulatory milestones, generally based on the submission, filing or approval of regulatory applications such as a new drug application (“NDA”) in the United States; and sales-based milestones, generally based on meeting specific thresholds of sales in certain geographic areas. The Company is also eligible to receive from Sanofi tiered royalty payments on worldwide net sales of mRNA vaccines. For each collaboration that includes development milestone payments, the Company evaluates whether it is probable that the consideration associated with each milestone will not be subject to a significant reversal in the cumulative amount of revenue recognized. Amounts that meet this threshold are included in the transaction price using the most likely amount method, whereas amounts that do not meet this threshold are considered constrained and excluded from the transaction price until they meet this threshold. Milestones tied to regulatory approval, and therefore not within the Company’s control, are considered constrained until such approval is received. Upfront and ongoing development milestones per its collaboration agreements are not subject to refund if the development activities are not successful. At the end of each subsequent reporting period, the Company re-evaluates catch-up ASC 606 requires the Company to allocate the arrangement consideration on a relative standalone selling price basis for each performance obligation after determining the transaction price of the contract and identifying the performance obligations to which that amount should be allocated. The relative standalone selling price is defined in ASC 606 as the price at which an entity would sell a promised good or service separately to a customer. If other observable transactions in which the Company has sold the same performance obligation separately are not available, the Company is required to estimate the standalone selling price of each performance obligation. Key assumptions to determine the standalone selling price may include forecasted revenues, development timelines, reimbursement rates for personnel costs, discount rates and probabilities of technical and regulatory success. Whenever the Company determines that a contract should be accounted for as a combined performance obligation it will utilize the cost-to-cost cost-to-cost The Company evaluates its collaborative agreements for proper classification in the consolidated statements of operations based on the nature of the underlying activity. Transactions between collaborators recorded in the Company’s consolidated statements of operations are recorded on either a gross or net basis, depending on the characteristics of the collaborative relationship. For revenue generating arrangements where the Company, as a vendor, provides consideration to a licensor or collaborator, as a customer, the Company applies the accounting standard that governs such transactions. This standard addresses the accounting for revenue arrangements where both the vendor and the customer make cash payments to each other for services and/or products. A payment to a customer is presumed to be a reduction of the transaction price unless the Company receives an identifiable benefit for the payment and it can reasonably estimate the fair value of the benefit received. Payments to a customer that are deemed a reduction of the transaction price are recorded first as a reduction of revenue, to the extent of both cumulative revenue recorded to date and probable future revenues, which include any unamortized deferred revenue balances, under all arrangements with such customer, and then as an expense. Payments that are not deemed to be a reduction of the transaction price are recorded as an expense. Consideration that does not meet the requirements to satisfy the above revenue recognition criteria is a contract liability and is recorded as deferred revenue in the consolidated balance sheets. Although the Company follows detailed guidelines in measuring revenue, certain judgments affect the application of its revenue policy. For example, in connection with the Sanofi Agreement, the Company has recorded short-term and long-term deferred revenue on its consolidated balance sheets based on the Company’s best estimate of when such revenue will be recognized. Short-term deferred revenue consists of amounts that are expected to be recognized as revenue in the next 12 months. Amounts that the Company expects will not be recognized within the next 12 months are classified as long-term deferred revenue. The estimate of deferred revenue also reflects management’s estimate of the periods of the Company’s involvement in certain of its collaborations. The Company’s performance obligations under these collaborations consist of participation on steering committees and the performance of other research and development services. In certain instances, the timing of satisfying these obligations can be difficult to estimate. Accordingly, the Company’s estimates may change in the future. Such changes to estimates would result in a change in revenue recognition amounts. If these estimates and judgments change over the course of these agreements, it may affect the timing and amount of revenue that the Company will recognize and record in future periods. At December 31, 2019, the Company had short-term and long-term deferred revenue of $18.1 million and $25.3 million, respectively, related to the Sanofi Agreement. Under ASC 606, the Company will recognize revenue and record a receivable when it fulfills its performance obligations under the Sanofi Agreement. When no further performance obligation is required to be satisfied, the Company will recognize revenue for the portion satisfied and record a receivable. Amounts are recorded as short-term collaboration receivables when the Company’s right to consideration is unconditional. The Company does not assess whether a contract has a significant financing component if the expectation at contract inception is that the period between payment by the customer and the transfer of the promised goods or services to the customer will be one year or less. The Company expenses incremental costs of obtaining a contract as and when incurred if the expected amortization period of the asset that the Company would have recognized is one year or less or the amount is immaterial. At December 31, 2019, the Company has not capitalized any costs to obtain any of its contracts. |
In-Process Research and Development | In-Process The fair value of in-process The fair value of an IPR&D intangible asset is typically determined using an income approach whereby management forecasts the net cash flows expected to be generated by the asset over its estimated useful life. The net cash flows reflect the asset’s stage of completion, the probability of technical success, the projected costs to complete, expected market competition, and an assessment of the asset’s life-cycle. The net cash flows are then adjusted to present value by applying an appropriate discount rate that reflects the risk factors associated with the cash flow streams. Indefinite-lived IPR&D is not subject to amortization, but is tested annually for impairment or more frequently if there are indicators of impairment. The Company tests its indefinite-lived IPR&D annually for impairment on October 1st. In testing indefinite-lived IPR&D for impairment, the Company has the option to first assess qualitative factors to determine whether the existence of events or circumstances would indicate that it is more likely than not that its fair value is less than its carrying amount, or the Company can perform a quantitative impairment analysis to determine the fair value of the indefinite-lived IPR&D without performing a qualitative assessment. Qualitative factors that the Company considers 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 the Company chooses to first assess qualitative factors and the Company determines that it is more likely than not that the fair value of the indefinite-lived IPR&D is less than its carrying amount, the Company would then determine the fair value of the indefinite-lived IPR&D. Under either approach, if the fair value of the indefinite-lived IPR&D is less than its carrying amount, an impairment charge is recognized in the consolidated statements of operations. In September 2019, the Company discontinued development of MRT5201, which was an indicator of impairment, and, as a result, the Company retested the indefinite-lived IPR&D related to the OTC deficiency program for impairment. The Company will not be investing any additional funds in this program and has reallocated all resources previously dedicated to the OTC deficiency program to other programs within the Company. The Company determined that there was no residual value to the indefinite-lived IPR&D related to the OTC deficiency program and, as a result, the Company recorded an impairment charge of $ million during the year ended December 31, 2019, representing the entire value of the indefinite-lived IPR&D related to the OTC deficiency program. During the year ended December 31, 2018, the Company did not recognize any impairment charges related to its definite-lived IPR&D (see Note 4). |
Goodwill | Goodwill Goodwill represents the excess of the fair value of the consideration transferred over the fair value of the net tangible and identifiable intangible assets acquired in a business combination. Goodwill is not subject to amortization, but is tested annually for impairment or more frequently if there are indicators of impairment. The Company tests its goodwill annually for impairment on October 1st. The Company has determined that there is a single reporting unit for purposes of testing goodwill for impairment. In testing goodwill for impairment, the Company has the option to first assess qualitative factors to determine whether the existence of events or circumstances would indicate that it is more likely than not that the fair value of the reporting unit was less than its carrying amount, or the Company can perform a two-step two-step two-step |
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 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 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 and short-term investments are carried at fair value, determined based on Level 2 inputs in the fair value hierarchy described above (see Note 5). The Company’s contingent consideration liability is carried at fair value, determined based on Level 3 inputs in the fair value hierarchy described above (see Note 5). The carrying values of the Company’s short-term collaboration receivables, prepaid expenses and other current assets, accounts payable, accrued expenses and other short-term liabilities approximate their fair values due to the short-term nature of these assets and liabilities. |
Segment Information | Segment Information The Company manages its operations as a single operating segment for the purposes of assessing performance and making operating decisions. The Company’s primary focus is on the advancement of the Company’s MRT platform to treat diseases caused by protein or gene dysfunction. |
Research and Development Costs | Research and Development Costs Costs associated with internal research and development and external research and development services, including drug development and preclinical studies, are expensed as incurred. Research and development expenses include costs for salaries, employee benefits, subcontractors, facility-related expenses, depreciation and amortization, including amortization related to definite-lived IPR&D intangible assets, stock-based compensation, third-party license fees, laboratory supplies, and external costs of outside vendors engaged to conduct discovery, preclinical and clinical development activities and clinical trials as well as to manufacture clinical trial materials, and other costs. The Company recognizes external research and development costs based on an evaluation of the progress to completion of specific tasks using information provided to the Company by its service providers. 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. Such prepaid expenses are recognized as an expense when the services have been performed or the goods have been delivered, or when it is no longer expected that the goods will be delivered or the services rendered. Upfront payments, milestone payments (other than those deemed contingent consideration in a business combination) and annual maintenance fees under license agreements are expensed in the period in which they are incurred. |
Research and Development Contract Costs and Accruals | Research and Development Contract Costs and Accruals The Company has entered into various research and development-related contracts with companies both inside and outside of the United States. The related costs are recorded as research and development expenses as incurred. The Company records accruals for estimated ongoing research and development costs. When evaluating the adequacy of the accrued liabilities, the Company analyzes progress of the studies or clinical trials, including the phase or completion of events, invoices received and contracted costs. Significant judgments and estimates are made in determining the accrued balances at the end of any reporting period. Actual results could differ materially from the Company’s estimates. The Company’s historical accrual estimates 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 all stock-based awards granted to employees, directors and, effective January 1, 2019, non-employee Through December 31, 2018, for stock-based awards granted to non-employee non-employee The Company classifies stock-based compensation expense in its consolidated statements of operations 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. |
Classification and Accretion of Redeemable Convertible Preferred Stock | Classification and Accretion of Redeemable Convertible Preferred Stock The Company has classified its redeemable convertible preferred stock outside of stockholders’ equity (deficit) because the shares contained certain redemption features that were not solely within the control of the Company. Costs incurred in connection with the issuance of each series of redeemable convertible preferred stock were recorded as a reduction of gross proceeds from issuance. The carrying values of redeemable convertible preferred stock were accreted to their redemption values through a charge to additional paid-in |
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. The Company’s only element of other comprehensive income (loss) was unrealized gains (losses) on U.S. government agency bonds, which are classified as available-for-sale-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 statement 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 more-likely-than-not |
Net Income (Loss) per Share | Net Income (Loss) per Share The Company follows the two-class two-class two-class Basic net income (loss) per share attributable to common stockholders is computed by dividing the net income (loss) attributable to common stockholders by the weighted average number of shares of common stock outstanding for the period. Diluted net income (loss) 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 shares of common stock outstanding for the period, including potential dilutive shares of common stock. For purposes of this calculation, outstanding stock options, unvested restricted common stock and redeemable convertible preferred stock are considered potential dilutive shares of common stock. The Company’s preferred stock contractually entitles the holders of such shares to participate in dividends but does not contractually require the holders of such shares to participate in losses of the Company. Accordingly, in periods in which the Company reports a net loss attributable to common stockholders, 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 shares of common stock 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 years ended December 31, 2019 and 2018. |
Recently Adopted Accounting Pronouncement | Recently Adopted Accounting Pronouncements In February 2016, the FASB issued ASU 2016-02, right-of-use 2016-02 The Company elected the permitted practical expedients within ASC 842, which allowed the Company to not reassess previous accounting conclusions around whether arrangements are or contain leases, and carried forward both the historical classification of leases and the treatment of initial direct costs. In addition, the Company elected to exclude leases with an initial term of one year or less in the recognized ROU assets and lease liabilities. Adoption of the new standard resulted in the recording of ROU assets and related lease liabilities of approximately $10.9 million and $13.0 million, respectively, as of January 1, 2019. The standard did not materially impact the Company’s consolidated net earnings and had no impact on cash flows. Refer to Note 13 for the additional disclosures required by ASC 842. The Company determines if an arrangement is a lease at inception. For leases where the Company is the lessee, ROU assets represent the Company’s right to use an underlying asset for the lease term and lease liabilities represent an obligation to make lease payments arising from the lease. ROU assets and lease liabilities are recognized at the lease commencement date based on the present value of lease payments over the lease term. As most of the Company’s leases do not provide an implicit interest rate, the Company uses its incremental borrowing rate, which are the rates incurred to borrow on a collateralized basis over a term equal to the lease payments in a similar economic environment, in determining the present value of lease payments. The lease terms used to calculate the ROU asset and related lease liability include options to extend or terminate the lease when it is reasonably certain that the Company will exercise that option. Lease expense for operating leases is recognized on a straight-line basis over the lease term as an operating expense. The Company has lease agreements which require payments for lease and non-lease In July 2017, the FASB issued ASU No. 2017-11, Earnings Per Share (Topic 260), Distinguishing Liabilities from Equity (Topic 480), Derivatives and Hedging (Topic 815) I. Accounting for Certain Financial Instruments with Down Round Features II. Replacement of the Indefinite Deferral for Mandatorily Redeemable Financial Instruments of Certain Nonpublic Entities and Certain Mandatorily Redeemable Noncontrolling Interests with a Scope Exception . In June 2018, the FASB issued ASU No. 2018-07, Compensation-Stock Compensation (Topic 718) non-employees. non-employee non-employee |
Recently Issued Accounting Pronouncements | Recently Issued Accounting Pronouncements In June 2016, the FASB issued guidance on the Measurement of Credit Losses on Financial Instruments. The guidance requires that credit losses be reported using an expected losses model rather than the incurred losses model that is currently used, and establishes additional disclosures related to credit risks. For available-for-sale 3 In January 2017, the FASB issued ASU No. 2017-04, Intangibles—Goodwill and Other: Simplifying the Test for Goodwill Impairment (Topic 350) In August 2018, the FASB issued ASU No. 2018-13, Fair Value Measurement (Topic 820): Disclosure Framework—Changes to the Disclosure Requirements for Fair Value Measurement In November 2018, the FASB issued ASU No. 2018-18, Collaborative Arrangements (Topic 808) Revenue Recognition Collaborative Arrangements In December 2019, the FASB issued ASU No. 2019-12, Income Taxes-Simplifying the Accounting for Income Taxes step-up |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Accounting Policies [Abstract] | |
Property And Equipment Estimated Useful Life | The estimated useful lives of the Company’s property and equipment are as follows: Estimated Useful Life Laboratory equipment 5 years Computer equipment 3 years Office equipment 5 years Leasehold improvements Shorter of lease term or 10 years |
Collaboration Agreement (Tables
Collaboration Agreement (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Summary of Collaboration Revenue | The following table summarizes the Company’s collaboration revenue (in thousands): Year Ended December 31, 2019 2018 Collaboration revenue $ 7,804 $ 1,420 |
Balance of Contract Liabilities Related to Collaboration Agreements | The following table presents the balance of the Company’s contract liabilities (in thousands): December 31, December 31, Contract liabilities Deferred revenue $ 43,356 $ 44,413 |
Intangible Assets and Goodwill
Intangible Assets and Goodwill (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Goodwill and Intangible Assets Disclosure [Abstract] | |
Summary of Definite-Lived Intangible Assets Subject to Amortization and Indefinite-Lived Intangible Assets | December 31, 2019 Estimated Gross Carrying Amount Accumulated Amortization Impairment Charge Net Carrying Amount (In thousands) Definite-lived intangible assets: MRT 8 years $ 45,992 $ (2,747 ) $ — $ 43,245 Indefinite-lived intangible assets: IPR&D—CF Indefinite 42,291 — — 42,291 IPR&D—OTC Indefinite 18,559 — (18,559 ) — Total intangible assets, net $ 106,842 $ (2,747 ) $ (18,559 ) $ 85,536 December 31, 2018 Estimated Gross Carrying Amount Accumulated Amortization Impairment Charge Net Carrying Amount (In thousands) Definite-lived intangible assets: MRT 8 years $ 45,992 $ (397 ) $ — $ 45,595 Indefinite-lived intangible assets: IPR&D—CF Indefinite 42,291 — — 42,291 IPR&D—OTC Indefinite 18,559 — — 18,559 Total intangible assets, net $ 106,842 $ (397 ) $ — $ 106,445 |
Fair Value of Financial Asset_2
Fair Value of Financial Assets and Liabilities (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Fair Value Disclosures [Abstract] | |
Schedule of Financial Assets and Liabilities Measured at Fair Value on Recurring Basis | The following tables present information about the Company’s financial assets and liabilities measured at fair value on a recurring basis (in thousands): Fair Value Measurements as of December 31, 2019 Using: Level 1 Level 2 Level 3 Total Assets: Money market funds $ — $ 56,591 $ — $ 56,591 U.S. government agency bonds — 104,098 — 104,098 $ — $ 160,689 $ — $ 160,689 Liabilities: Contingent consideration $ — $ — $ 103,655 $ 103,655 $ — $ — $ 103,655 $ 103,655 Fair Value Measurements as of December 31, 2018 Using: Level 1 Level 2 Level 3 Total Assets: Money market funds $ — $ 23,318 $ — $ 23,318 U.S. government agency bonds — 88,904 — 88,904 $ — $ 112,222 $ — $ 112,222 Liabilities: Contingent consideration $ — $ — $ 103,642 $ 103,642 $ — $ — $ 103,642 $ 103,642 |
Schedule of Unobservable Inputs and Fair Value Components of Contingent Consideration | The following tables presents the unobservable inputs and fair value of the components of the contingent consideration (dollar amounts in thousands): Unobservable Inputs Fair Value at Projected Year of Payment December 31, December 31, Earnout payments 2026 – $ 96,097 $ 94,999 Milestone payments 2026 – 7,558 8,643 $ 103,655 $ 103,642 |
Schedule of Total Acquisition Related Contingent Consideration Liability | The following table presents a roll-forward of the total acquisition-related contingent consideration liability (in thousands): Fair Value Balance as of December 31, 2018 $ 103,642 Discontinuation of MRT5201 (23,174 ) Increase in fair value of contingent consideration 23,187 Balance as of December 31, 2019 $ 103,655 |
Property and Equipment, Net (Ta
Property and Equipment, Net (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Property, Plant and Equipment [Abstract] | |
Schedule of Property and Equipment, Net | Property and equipment, net consisted of the following (in thousands): December 31, December 31, Laboratory equipment $ 9,044 $ 7,012 Computer equipment 779 686 Office equipment 883 836 Leasehold improvements 5,635 5,635 Construction in progress 3,460 959 19,801 15,128 Less: Accumulated depreciation and amortization (7,262 ) (4,883 ) $ 12,539 $ 10,245 |
Accrued Expenses (Tables)
Accrued Expenses (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Payables and Accruals [Abstract] | |
Schedule of Accrued Expenses | Accrued expenses consisted of the following (in thousands): December 31, December 31, Accrued employee compensation and benefits $ 3,547 $ 2,933 Accrued external research and development expenses 1,763 1,901 Accrued consultant and professional fees 1,390 977 Other 372 736 $ 7,072 $ 6,547 |
Incentive Stock Options and R_2
Incentive Stock Options and Restricted Stock (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Summary of Stock Option Activity | The following table summarizes the Company’s stock option activity since December 31, 2018 (in thousands, except share and per share amounts): Number of Shares Weighted Average Exercise Price Weighted Average Remaining Contractual Term Intrinsic Value (in years) Outstanding as of December 31, 2018 6,236,006 $ 7.78 8.74 $ 1,104 Granted 2,825,200 $ 8.55 Exercised (233,549 ) $ 6.48 Forfeited (181,279 ) $ 7.89 Outstanding as of December 31, 2019 8,646,378 $ 8.06 8.42 $ 3,687 Exercisable as of December 31, 2019 3,457,794 $ 7.67 7.96 $ 2,234 Vested and expected to vest as of December 31, 2019 8,646,378 $ 8.06 8.42 $ 3,687 |
Summary of Assumptions Used in Black-Scholes Option-Pricing Model to Determine Grant-Date Fair Value of Stock Option Granted | The following table presents, on a weighted average basis, the assumptions used in the Black-Scholes option-pricing model to determine the grant-date fair value of stock options granted to employees and directors: Year Ended December 31, 2019 2018 Risk-free interest rate 2.36 % 2.81 % Expected term (in years) 6.0 6.0 Expected volatility 73.0 % 75.5 % Expected dividend yield 0 % 0 % |
Summary of Restricted Stock Activity | The following table summarizes the Company’s restricted stock activity since December 31, 2018: Number of Shares Weighted Average Grant-Date Fair Value Unvested restricted common stock outstanding as of December 31, 2018 219,148 $ 1.27 Forfeited restricted common stock (1,783 ) $ 1.28 Vested restricted common stock (183,197 ) $ 1.27 Unvested restricted common stock outstanding as of December 31, 2019 34,168 $ 1.28 |
Summary of Stock-Based Compensation Expense | Stock-based compensation expense was classified in the consolidated statements of operations as follows (in thousands): Year Ended December 31, 2019 2018 Research and development expenses $ 4,786 $ 3,756 General and administrative expenses 6,548 3,577 $ 11,334 $ 7,333 |
Income Taxes (Tables)
Income Taxes (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Income Tax Disclosure [Abstract] | |
Schedule of Reconciliation of U.S. Federal Statutory Income Tax Rate and Effective Income Tax Rate | A reconciliation of the U.S. federal statutory income tax rate to the Company’s effective income tax rate is as follows: Year Ended December 31, 2019 2018 U.S. federal statutory income tax rate (21.0 )% (21.0 )% State income taxes, net of federal benefit (6.6 ) (6.9 ) Research and development tax credits and orphan drug credit (6.8 ) (5.5 ) Stock-based compensation 0.5 0.5 Other permanent differences 0.2 0.3 Change in deferred tax asset valuation allowance 33.6 27.7 Other (0.3 ) (0.4 ) Effective income tax rate (0.4 )% (5.3 )% |
Schedule of Components of Deferred Tax Assets and Liabilities | Components of the Company’s deferred tax assets and liabilities were a s December 31, 2019 2018 Deferred tax assets: Net operating loss carryforwards $ 61,460 $ 50,917 Research and development tax credit and orphan drug credit carryforwards 21,830 13,581 Intangibles 3,607 — Deferred revenue 11,845 — Depreciation and amortization 2,253 2,380 Accrued expenses and other 5,354 2,894 Total deferred tax assets 106,349 69,772 Deferred tax liabilities: Indefinite-lived intangible assets — (2,109 ) Total deferred tax liabilities — (2,109 ) Valuation allowance (106,346 ) (68,143 ) Net deferred tax assets (liabilities) $ 3 $ (480 ) |
Summary of Changes in Valuation Allowance for Deferred Tax Assets | Changes in the valuation allowance for deferred tax assets during the years ended December 31, 2019 and 2018 related primarily to the increase in net operating loss carryforwards and research and development tax credit carryforwards in 2019 and 2018, and were as follows (in thousands): Year Ended December 31, 2019 2018 Valuation allowance at beginning of year $ (68,143 ) $ (39,605 ) Increases recorded to income tax provision (38,203 ) (28,538 ) Valuation allowance at end of year $ (106,346 ) $ (68,143 ) |
Net Loss per Share (Tables)
Net Loss per Share (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Earnings Per Share [Abstract] | |
Summary of Basic and Diluted Net Loss Per Share Attributable to Common Stockholders | Basic and diluted net loss per share attributable to common stockholders was calculated as follows (in thousands, except share and per share amounts): Year Ended December 31, 2019 2018 Numerator: Net loss $ (113,293 ) $ (97,395 ) Accretion of redeemable convertible preferred stock to redemption value — (644 ) Net loss attributable to common stockholders $ (113,293 ) $ (98,039 ) Denominator: Weighted average common shares outstanding—basic and diluted 51,445,539 26,945,508 Net loss per share attributable to common stockholders—basic and diluted $ (2.20 ) $ (3.64 ) |
Schedule of Potential Securities Excluded from Computation of Earnings Per Share | The Company excluded the following potential shares of common stock, 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: December 31, 2019 2018 Options to purchase common stock 8,646,378 6,236,006 Unvested restricted common stock 34,168 219,148 8,680,546 6,455,154 |
Leases (Tables)
Leases (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Leases [Abstract] | |
Schedule of Lease Cost | The components of lease cost were as follows (dollar amounts in thousands): Year Ended Lease cost Operating lease cost ̥$ 2,692 Short-term lease cost — Total lease cost $ 2,692 Other information Operating cash flows from operating leases $ 2,583 Operating lease liabilities arising from obtaining right-of-use assets — Weighted-average remaining lease term 8 years Weighted-average discount rate 17.5 % |
Schedule of maturities of operating lease liabilities | As of December 31, 2019, maturities of operating lease liabilities are as follows (in thousands): December 31, 2019 2020 $ 2,659 2021 2,737 2022 2,818 2023 2,860 2024 2,937 2025 and thereafter 10,160 Total future minimum lease payments 24,171 Less: imputed interest (11,557 ) Present value of lease liabilities $ 12,614 |
Schedule of Future Minimum Rental Commitments for Operating Leases | As of December 31, 2018, minimum rental commitments under the real estate lease was as follows (in thousands): December 31, 2018 2019 $ 2,534 2020 2,610 2021 2,688 2022 2,769 2023 2,852 2024 and thereafter 13,096 Total future minimum lease payments $ 26,549 |
Selected Quarterly Financial _2
Selected Quarterly Financial Information (Unaudited) (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Selected Quarterly Financial Information [Abstract] | |
Schedule of Selected Quarterly Financial Information | The following table contains quarterly financial information for 2019 and 2018 (in thousands, except per share amounts). The Company believes that the following information reflects all normal recurring adjustments necessary for a fair statement of the information for the periods presented. The operating results for any quarter are not necessarily indicative of results for any future period. 2019 First Quarter Second Quarter Third Quarter Fourth Quarter Total Collaboration revenue $ 1,474 $ 1,174 $ 1,266 $ 3,890 $ 7,804 Total operating expenses 35,679 29,364 22,901 35,629 123,573 Loss from operations (34,205 ) (28,190 ) (21,635 ) (31,739 ) (115,769 ) Net loss (33,198 ) (27,832 ) (21,227 ) (31,036 ) (113,293 ) Net loss per share applicable to common stockholders—basic and diluted $ (0.74 ) $ (0.57 ) $ (0.41 ) $ (0.48 ) $ (2.20 ) 2018 First Quarter Second Quarter Third Quarter Fourth Quarter Total Collaboration revenue $ — $ — $ 238 $ 1,182 $ 1,420 Total operating expenses 22,389 29,062 45,719 8,480 105,650 Loss from operations (22,389 ) (29,062 ) (45,481 ) (7,298 ) (104,230 ) Net loss (21,209 ) (27,503 ) (42,646 ) (6,037 ) (97,395 ) Net loss per share applicable to common stockholders—basic and diluted $ (2.35 ) $ (3.04 ) $ (0.97 ) $ (0.13 ) $ (3.64 ) |
Nature of the Business and Ba_2
Nature of the Business and Basis of Presentation - Additional Information (Detail) $ / shares in Units, $ in Thousands | May 03, 2019USD ($)$ / sharesshares | Jun. 30, 2018 | Jul. 31, 2018USD ($)$ / sharesshares | Dec. 31, 2019USD ($) | Sep. 30, 2019USD ($)shares | Jun. 30, 2019USD ($) | Mar. 31, 2019USD ($) | Dec. 31, 2018USD ($)Disease | Sep. 30, 2018USD ($) | Jun. 30, 2018USD ($) | Mar. 31, 2018USD ($) | Dec. 31, 2019USD ($)Subsidiary | Dec. 31, 2018USD ($)Disease | Sep. 20, 2019$ / shares | Jul. 03, 2019USD ($) |
Initial Public Offering [Line Items] | |||||||||||||||
Number of wholly owned subsidiaries | Subsidiary | 2 | ||||||||||||||
Reverse stock split description | one-for-5.5555 reverse stock split | ||||||||||||||
Reverse stock split ratio | 0.1800 | ||||||||||||||
Net proceeds from IPO | $ 113,200 | ||||||||||||||
Payments of other offering expenses | $ 4,102 | ||||||||||||||
Accumulated deficit | $ (359,496) | $ 359,500 | $ (246,203) | $ (359,496) | (246,203) | ||||||||||
Net loss | $ (31,036) | (21,227) | $ (27,832) | $ (33,198) | $ (6,037) | $ (42,646) | $ (27,503) | $ (21,209) | $ (113,293) | $ (97,395) | |||||
Cash, cash equivalents and short-term investments | 188,700 | ||||||||||||||
Sale from time to time of equity and debt securities | $ 250,000 | ||||||||||||||
Sanofi Pasteur Collaboration and Licensing Agreement [Member] | Sanofi Pasteur Inc [Member] | |||||||||||||||
Initial Public Offering [Line Items] | |||||||||||||||
Number of infectious disease pathogens | Disease | 5 | 5 | |||||||||||||
Period of research term | 3 years | ||||||||||||||
Upfront payment received | $ 45,000 | $ 45,000 | |||||||||||||
Sanofi Pasteur Collaboration and Licensing Agreement [Member] | Sanofi Pasteur Inc [Member] | Maximum [Member] | |||||||||||||||
Initial Public Offering [Line Items] | |||||||||||||||
Extended period of research term | 8 years | ||||||||||||||
Receivable from collaboration | 805,000 | ||||||||||||||
Open Market Sale Agreement [Member] | |||||||||||||||
Initial Public Offering [Line Items] | |||||||||||||||
Market sale aggreement of common stock | $ 50,000 | ||||||||||||||
Public Offering [Member] | |||||||||||||||
Initial Public Offering [Line Items] | |||||||||||||||
Payments of other offering expenses | $ 600 | ||||||||||||||
Issuance of common, shares | shares | 9,000,000 | ||||||||||||||
Gross proceeds from issuance of public offering | $ 90,000 | ||||||||||||||
Underwriting discounts and commissions | $ 5,400 | ||||||||||||||
Common stock issued and sold, per share | $ / shares | $ 10 | ||||||||||||||
IPO [Member] | |||||||||||||||
Initial Public Offering [Line Items] | |||||||||||||||
Issuance of common, shares | shares | 9,714,371 | ||||||||||||||
Common stock issued and sold, per share | $ / shares | $ 13 | ||||||||||||||
Private Placement [Member] | |||||||||||||||
Initial Public Offering [Line Items] | |||||||||||||||
Gross proceeds from issuance of private placement | $ 47,500 | ||||||||||||||
Payments of other offering expenses | $ 600 | ||||||||||||||
Issuance of common, shares | shares | 5,582,940 | ||||||||||||||
Common stock issued and sold, per share | $ / shares | $ 8.50 | ||||||||||||||
Placement agent fees | $ 2,800 |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies - Additional Information (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Jan. 01, 2019 | Dec. 31, 2018 | |
Indefinite-lived Intangible Assets [Line Items] | |||
Current portion of deferred revenue | $ 18,100 | $ 2,572 | |
Deferred revenue, net of current portion | 25,256 | $ 41,841 | |
Right-of-use assets, net | 10,400 | ||
Operating lease, liability | 12,614 | ||
indefinite lived intangible assets Impairment charge | 18,559 | ||
ASU 2016-02 [Member] | |||
Indefinite-lived Intangible Assets [Line Items] | |||
Current portion of deferred revenue | 18,100 | ||
Deferred revenue, net of current portion | 25,300 | ||
Right-of-use assets, net | $ 10,900 | ||
Operating lease, liability | $ 13,000 | ||
In-Process Research and Development [Member] | OTC Deficiency [Member] | Shire's MRT Program [Member] | |||
Indefinite-lived Intangible Assets [Line Items] | |||
indefinite lived intangible assets Impairment charge | $ 18,600 |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies - Estimated Useful Lives of Property and Equipment (Detail) | 12 Months Ended |
Dec. 31, 2019 | |
Laboratory Equipment [Member] | |
Property Plant And Equipment Estimated Useful Lives [Line Items] | |
Property plant and equipment estimated useful life | 5 years |
Computer Equipment [Member] | |
Property Plant And Equipment Estimated Useful Lives [Line Items] | |
Property plant and equipment estimated useful life | 3 years |
Office Equipment [Member] | |
Property Plant And Equipment Estimated Useful Lives [Line Items] | |
Property plant and equipment estimated useful life | 5 years |
Leasehold Improvements [Member] | |
Property Plant And Equipment Estimated Useful Lives [Line Items] | |
Property plant and equipment estimated useful life | Shorter of lease term or 10 years |
Collaboration Agreement - Addit
Collaboration Agreement - Additional Information (Detail) - Sanofi Agreement [Member] $ in Millions | 3 Months Ended | 12 Months Ended | |
Mar. 31, 2019USD ($) | Dec. 31, 2019USD ($) | Dec. 31, 2018USD ($)Disease | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||
Number of infectious disease pathogens for vaccine development | Disease | 5 | ||
Period of research term | 3 years | ||
Upfront payment received | $ 45 | ||
Additional fee per added pathogen option | $ 5 | ||
Maximum development and regulatory milestone payment receivable | 63 | ||
Technology and process transfer milestone payment receivable | 10 | ||
Royalty payment term | 10 years | ||
Reduction in transaction price | $ 10 | ||
Non-refundable upfront payment | 45 | ||
Estimated reimbursable employee cost | 32.6 | ||
Estimated reimbursable development cost | 54.5 | ||
Estimated milestone payments | $ 19 | ||
Revenue recognizing period | 8 years | ||
Revenue recognized from contract liabilities | $ 1.1 | 0 | |
Maximum [Member] | |||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||
Extended period of research term | 8 years | ||
Receivable from collaboration | 805 | ||
Sales milestone payment receivable | $ 85 |
Collaboration Agreement - Summa
Collaboration Agreement - Summary of Collaboration Revenue (Detail) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | ||||||
Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Dec. 31, 2018 | Sep. 30, 2018 | Dec. 31, 2019 | Dec. 31, 2018 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | ||||||||
Collaboration revenue | $ 3,890 | $ 1,266 | $ 1,174 | $ 1,474 | $ 1,182 | $ 238 | $ 7,804 | $ 1,420 |
Collaboration Agreement - Balan
Collaboration Agreement - Balance of Contract Liabilities Related to Collaboration Agreements (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Contract liabilities | ||
Deferred revenue | $ 43,356 | $ 44,413 |
Intangible Assets and Goodwil_2
Intangible Assets and Goodwill - Summary of Definite-Lived Intangible Assets Subject to Amortization and Indefinite-Lived Intangible Assets (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Finite Lived Intangible Assets [Line Items] | ||
Definite-lived intangible assets, accumulated amortization | $ (2,747) | $ (397) |
Definite-lived intangible assets, Impairment Charge | (18,559) | |
Total intangible assets, gross carrying amount | 106,842 | 106,842 |
Total intangible assets, net carrying amount | $ 85,536 | $ 106,445 |
In-Process Research and Development [Member] | MRT Product [Member] | Shire's MRT Program [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Definite-lived intangible assets, estimated life | 8 years | 8 years |
Definite-lived intangible assets, gross carrying amount | $ 45,992 | $ 45,992 |
Definite-lived intangible assets, accumulated amortization | (2,747) | (397) |
Definite-lived intangible assets, net carrying amount | 43,245 | 45,595 |
In-Process Research and Development [Member] | Cystic Fibrosis [Member] | Shire's MRT Program [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Indefinite-lived intangible assets, gross carrying amount/net carrying amount | 42,291 | 42,291 |
In-Process Research and Development [Member] | OTC Deficiency [Member] | Shire's MRT Program [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Definite-lived intangible assets, Impairment Charge | (18,559) | |
Indefinite-lived intangible assets, gross carrying amount/net carrying amount | $ 18,559 | $ 18,559 |
Intangible Assets and Goodwil_3
Intangible Assets and Goodwill - Additional Information (Detail) - USD ($) | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Finite Lived Intangible Assets [Line Items] | ||
Impairment of Intangible Assets, Finite-lived | $ 0 | |
indefinite lived intangible assets Impairment charge | 18,559,000 | |
Goodwill, Impairment Charges | 0 | $ 0 |
Goodwill | 21,359,000 | 21,359,000 |
Goodwill changes | 0 | |
Shire's MRT Program [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Goodwill | 21,400,000 | |
Shire's MRT Program [Member] | Sanofi Agreement [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Estimated amortization expense of intangible assets for 2020 | 9,000,000 | |
Estimated amortization expense of intangible assets for 2021 | 9,900,000 | |
Estimated amortization expense of intangible assets for 2022 | 4,200,000 | |
Estimated amortization expense of intangible assets for 2023 | 2,300,000 | |
Estimated amortization expense of intangible assets for 2024 | 2,500,000 | |
OTC Deficiency Program [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Amortization of Intangible Assets | 2,300,000 | $ 400,000 |
OTC Deficiency [Member] | Prepaid Expenses and Other Current Assets [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
short-term receivables | 900,000 | |
In Process Research and Development [Member] | OTC Deficiency [Member] | Shire's MRT Program [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
indefinite lived intangible assets Impairment charge | $ 18,559,000 |
Fair Value of Financial Asset_3
Fair Value of Financial Assets and Liabilities - Schedule of Financial Assets and Liabilities Measured at Fair Value on Recurring Basis (Detail) - Fair Value, Measurements, Recurring - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Assets: | ||
Total, assets | $ 160,689 | $ 112,222 |
Liabilities: | ||
Total, liabilities | 103,655 | 103,642 |
Fair Value, Inputs, Level 2 | ||
Assets: | ||
Total, assets | 160,689 | 112,222 |
Fair Value, Inputs, Level 3 | ||
Liabilities: | ||
Total, liabilities | 103,655 | 103,642 |
Money Market Funds | ||
Assets: | ||
Total, assets | 56,591 | 23,318 |
Money Market Funds | Fair Value, Inputs, Level 2 | ||
Assets: | ||
Total, assets | 56,591 | 23,318 |
U.S. Government Agency Bonds | ||
Assets: | ||
Total, assets | 104,098 | 88,904 |
U.S. Government Agency Bonds | Fair Value, Inputs, Level 2 | ||
Assets: | ||
Total, assets | 104,098 | 88,904 |
Contingent Consideration | ||
Liabilities: | ||
Total, liabilities | 103,655 | 103,642 |
Contingent Consideration | Fair Value, Inputs, Level 3 | ||
Liabilities: | ||
Total, liabilities | $ 103,655 | $ 103,642 |
Fair Value of Financial Asset_4
Fair Value of Financial Assets and Liabilities - Schedule of Unobservable Inputs and Fair Value Components of Contingent Consideration (Detail) - Contingent Consideration - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Fair Value Assets Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Fair Value at | $ 103,655 | $ 103,642 |
Earnout Payments | ||
Fair Value Assets Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Fair Value at | $ 96,097 | $ 94,999 |
Earnout Payments | Minimum [Member] | ||
Fair Value Assets Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Projected Year of Payment | 2026 | 2026 |
Earnout Payments | Maximum [Member] | ||
Fair Value Assets Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Projected Year of Payment | 2039 | 2039 |
Milestone Payments | ||
Fair Value Assets Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Fair Value at | $ 7,558 | $ 8,643 |
Milestone Payments | Minimum [Member] | ||
Fair Value Assets Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Projected Year of Payment | 2026 | 2026 |
Milestone Payments | Maximum [Member] | ||
Fair Value Assets Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Projected Year of Payment | 2030 | 2030 |
Fair Value of Financial Asset_5
Fair Value of Financial Assets and Liabilities - Schedule of Total Acquisition Related Contingent Consideration Liability (Detail) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2019 | Dec. 31, 2018 | |
Business Acquisition, Contingent Consideration [Line Items] | |||
Beginning Balance | $ 103,642 | ||
Discontinuation of MRT5201 and Increase in fair value of contingent consideration | $ 14,600 | 13 | $ 25,020 |
Ending Balance | $ 103,642 | 103,655 | $ 103,642 |
Discontinuation of MRT5201 | |||
Business Acquisition, Contingent Consideration [Line Items] | |||
Discontinuation of MRT5201 and Increase in fair value of contingent consideration | (23,174) | ||
Contingent Consideration | |||
Business Acquisition, Contingent Consideration [Line Items] | |||
Discontinuation of MRT5201 and Increase in fair value of contingent consideration | $ 23,187 |
Fair Value of Financial Asset_6
Fair Value of Financial Assets and Liabilities - Additional Information (Detail) | 3 Months Ended | 12 Months Ended | |
Dec. 31, 2018USD ($) | Dec. 31, 2019USD ($) | Dec. 31, 2018USD ($) | |
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | |||
Cash and cash equivalents | $ 55,199,000 | $ 84,580,000 | $ 55,199,000 |
Short-term investments amortized cost | 103,400,000 | ||
Short-term investments unrealized gain | 700,000 | ||
Short-term investments fair value | 104,100,000 | ||
Increase decrease in contingent consideration liability | $ 14,600,000 | $ 13,000 | $ 25,020,000 |
Contingent Consideration | |||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | |||
Discount Rate | 14.5 | 13.5 | 14.5 |
Increase decrease in contingent consideration liability | $ 23,187,000 | ||
Minimum [Member] | |||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | |||
Short term investments maturity period | 1 year | ||
Money Market Funds | Fair Value, Inputs, Level 2 | |||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | |||
Cash and cash equivalents | $ 23,300,000 | $ 56,600,000 | $ 23,300,000 |
Fair Value, Measurements, Recurring | |||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | |||
Fair Value, assets transfers into (out of) Level 3 | 0 | 0 | |
Fair value,assets transfers from Level 2 to Level 1 | 0 | 0 | 0 |
Fair value,assets transfers from Level 1 to Level 2 | 0 | 0 | 0 |
Fair value, liabilities transfers from Level 1 to Level 2 | 0 | 0 | 0 |
Fair value, liabilities transfers from Level 2 to Level 1 | $ 0 | 0 | 0 |
Fair Value, liabilities transfers into (out of) Level 3 | 0 | $ 0 | |
In Process Research and Development [Member] | OTC Deficiency [Member] | Shires Mrt Program [Member] | |||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | |||
Increase decrease in contingent consideration liability | $ 23,200,000 |
Property and Equipment, Net - S
Property and Equipment, Net - Schedule of Property and Equipment, Net (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Property Plant And Equipment [Line Items] | ||
Property and equipment, gross | $ 19,801 | $ 15,128 |
Less: Accumulated depreciation and amortization | (7,262) | (4,883) |
Property and equipment, net | 12,539 | 10,245 |
Laboratory Equipment [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, gross | 9,044 | 7,012 |
Computer Equipment [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, gross | 779 | 686 |
Office Equipment [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, gross | 883 | 836 |
Leasehold Improvements [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, gross | 5,635 | 5,635 |
Construction In Progress [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, gross | $ 3,460 | $ 959 |
Property and Equipment, Net - A
Property and Equipment, Net - Additional Information (Detail) - USD ($) $ in Millions | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Property, Plant and Equipment [Abstract] | ||
Depreciation and amortization expense | $ 2.4 | $ 2.4 |
Accrued Expenses - Schedule of
Accrued Expenses - Schedule of Accrued Expenses (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Payables and Accruals [Abstract] | ||
Accrued employee compensation and benefits | $ 3,547 | $ 2,933 |
Accrued external research and development expenses | 1,763 | 1,901 |
Accrued consultant and professional fees | 1,390 | 977 |
Other | 372 | 736 |
Total accrued expenses | $ 7,072 | $ 6,547 |
Redeemable Convertible Prefer_2
Redeemable Convertible Preferred Stock - Additional Information (Detail) - shares | Dec. 31, 2019 | Dec. 31, 2018 | Jul. 31, 2018 | Dec. 31, 2017 |
Temporary Equity [Line Items] | ||||
Preferred Shares Issued | 0 | 0 | ||
Preferred Shares Outstanding | 0 | 0 | ||
Conversion of redeemable convertible preferred stock to common stock, Shares | 25,612,109 | |||
Preferred Shares Authorized | 0 | 0 | ||
Convertible Preferred Stock Subject to Mandatory Redemption | ||||
Temporary Equity [Line Items] | ||||
Preferred Shares Issued | 142,288,292 | |||
Preferred Shares Outstanding | 142,288,292 |
Common Stock - Additional Infor
Common Stock - Additional Information (Detail) - USD ($) | 12 Months Ended | |||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | Jul. 02, 2018 | |
Class of Stock [Line Items] | ||||
Common stock, shares authorized | 200,000,000 | 200,000,000 | 200,000,000 | |
Common stock, par value | $ 0.001 | $ 0.001 | ||
Common stock voting rights | Each share of common stock entitles the holder to one vote for each share of common stock held. | |||
Cash dividend paid shares | $ 0 | $ 0 | ||
Common stock, shares issued | 60,022,067 | 45,139,955 | ||
Common stock, shares outstanding | 60,022,067 | 45,139,955 |
Incentive Stock Options and R_3
Incentive Stock Options and Restricted Stock - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Thousands | Jan. 01, 2019 | Jun. 15, 2018 | Mar. 07, 2018 | Dec. 31, 2019 | Dec. 31, 2018 |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Stock-based compensation expense | $ 847 | ||||
Common stock, shares issued | 60,022,067 | 45,139,955 | |||
Unrecognized compensation cost related to unvested stock-based awards | $ 24,300 | ||||
Unrecognized compensation cost, period for recognition | 2 years 4 months 24 days | ||||
Stock Options [Member] | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Intrinsic value of stock options, exercised | $ 600 | $ 100 | |||
Weighted average grant-date fair value | $ 5.59 | $ 5.95 | |||
2018 Stock Incentive Plan [Member] | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Number of common shares reserved for issuance | 4,829,847 | ||||
Number of shares remaining available for issuance | 360,514 | ||||
Increase in common stock issued | 1,805,598 | ||||
Percentage threshold of outstanding shares under the plan | 4.00% | ||||
Percentage of exercise price per share of fair market value | 100.00% | ||||
Expiration period of stock options after grant date | 10 years | ||||
2018 Stock Incentive Plan [Member] | Minimum [Member] | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Number of stock issued during period under the plan | 3,349,582 | ||||
Stock options for purchase of common stock held, exercisable period | 1 year | ||||
2018 Stock Incentive Plan [Member] | Maximum [Member] | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Stock options for purchase of common stock held, exercisable period | 4 years | ||||
2018 Stock Incentive Plan [Member] | Share-based Compensation Award, Tranche One [Member] | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Number of common shares reserved for issuance | 2,512,187 | ||||
2018 Stock Incentive Plan [Member] | Share-based Compensation Award, Tranche Two [Member] | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Number of common shares reserved for issuance | 1,013,167 | ||||
2018 Employee Stock Purchase Plan [Member] | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Number of common shares reserved for issuance | 870,096 | 418,697 | |||
Increase in common stock issued | 451,399 | ||||
Percentage threshold of outstanding shares under the plan | 1.00% | ||||
Common stock, shares issued | 0 | ||||
2018 Employee Stock Purchase Plan [Member] | Minimum [Member] | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Number of stock issued during period under the plan | 837,395 | ||||
2016 Stock Incentive Plan [Member] | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Sharebased Compensation Arrangement by Sharebased Payment Award Shares Cancelled in Period | 151,548 | ||||
Percentage of exercise price per share of fair market value | 100.00% | ||||
Expiration period of stock options after grant date | 10 years | ||||
Stock options for purchase of common stock held, exercisable period | 4 years | ||||
Former Employee [Member] | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Issue of shares to former employee satisfaction claims | 67,406 | ||||
Former Employee [Member] | General and Administrative Expense [Member] | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Stock-based compensation expense | $ 800 |
Incentive Stock Options and R_4
Incentive Stock Options and Restricted Stock - Summary of Stock Option Activity (Detail) - Stock Options [Member] - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Number of shares | ||
Number of shares, beginning balance | 6,236,006 | |
Number of shares, granted | 2,825,200 | |
Number of shares, exercised | (233,549) | |
Number of shares, forfeited | (181,279) | |
Number of shares, ending balance | 8,646,378 | 6,236,006 |
Number of shares, exercisable | 3,457,794 | |
Number of shares, vested and expected to vest, ending balance | 8,646,378 | |
Weighted average exercise price | ||
Weighted average exercise price, beginning balance | $ 7.78 | |
Weighted average exercise price, granted | 8.55 | |
Weighted average exercise price, exercised | 6.48 | |
Weighted average exercise price, forfeited | 7.89 | |
Weighted average exercise price, ending balance | 8.06 | $ 7.78 |
Weighted average exercise price, exercisable | 7.67 | |
Weighted average exercise price, vested and expected to vest, ending balance | $ 8.06 | |
Weighted average remaining contractual term | ||
Weighted average remaining contractual term, ending balance | 8 years 5 months 1 day | 8 years 8 months 26 days |
Weighted average remaining contractual term, exercisable | 7 years 11 months 15 days | |
Weighted average remaining contractual term, vested and expected to vest, ending balance | 8 years 5 months 1 day | |
Intrinsic value | ||
Intrinsic value, ending balance | $ 3,687 | $ 1,104 |
Intrinsic value, exercisable | 2,234 | |
Intrinsic value, vested and expected to vest, ending balance | $ 3,687 |
Incentive Stock Option and Rest
Incentive Stock Option and Restricted Stock - Summary of Assumptions Used in Black-Scholes Option-Pricing Model to Determine Grant-Date Fair Value of Stock Option Granted (Detail) - Employees And Directors [Member] - Stock Options [Member] | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Risk-free interest rate | 2.36% | 2.81% |
Expected term (in years) | 6 years | 6 years |
Expected volatility | 73.00% | 75.50% |
Expected dividend yield | 0.00% | 0.00% |
Incentive Stock Option and Re_2
Incentive Stock Option and Restricted Stock - Summary of Restricted Stock Activity (Detail) - Restricted Common Stock [Member] | 12 Months Ended |
Dec. 31, 2019$ / sharesshares | |
Number of shares | |
Number of shares, beginning balance | shares | 219,148 |
Number of shares, forfeited | shares | (1,783) |
Number of shares, vested | shares | (183,197) |
Number of shares, ending balance | shares | 34,168 |
Weighted average grant-date fair value | |
Weighted average grant-date fair value, beginning balance | $ / shares | $ 1.27 |
Weighted average grant-date fair value, forfeited | $ / shares | 1.28 |
Weighted average grant-date fair value, vested | $ / shares | 1.27 |
Weighted average grant-date fair value, ending balance | $ / shares | $ 1.28 |
Incentive Stock Option and Re_3
Incentive Stock Option and Restricted Stock - Summary of Stock-Based Compensation Expense (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Stock-based compensation expense | $ 11,334 | $ 7,333 |
Research and Development Expenses [Member] | ||
Stock-based compensation expense | 4,786 | 3,756 |
General and Administrative Expenses [Member] | ||
Stock-based compensation expense | $ 6,548 | $ 3,577 |
Income Taxes - Additional Infor
Income Taxes - Additional Information (Detail) - USD ($) | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Operating Loss Carryforwards [Line Items] | ||
Income tax benefits | $ (486,000) | $ (5,565,000) |
orphan drug tax credit carryforwards | $ 13,000,000 | |
Orphan drug tax credit carryforwards expiration year | 2037 | |
Increase in ownership of certain stockholders or public groups in the stock as result of transactions, specified period | 3 years | |
Indefinite-lived net operating loss carryforwards | $ 800,000 | |
Unrecognized tax benefits | $ 0 | 0 |
Income tax examination, description | The federal and state returns are generally subject to tax examinations for the tax years ended December 31, 2016 to the present. | |
Tax cuts and jobs act of 2017, net operating losses carryforward, maximum percentage of taxable income | 80.00% | |
Federal [Member] | ||
Operating Loss Carryforwards [Line Items] | ||
Operating loss carryforwards | $ 229,300,000 | |
Operating loss carryforwards subject to expiration | $ 122,100,000 | |
Operating loss carryforwards, expiration date | 2031 | |
Research and development tax credit carryforward, amount | $ 6,500,000 | |
Research and development tax credit carryforward expiration year | 2032 | |
State [Member] | ||
Operating Loss Carryforwards [Line Items] | ||
Operating loss carryforwards | $ 210,600,000 | |
Operating loss carryforwards, expiration date | 2031 | |
Research and development tax credit carryforward, amount | $ 2,700,000 | |
Research and development tax credit carryforward expiration year | 2028 | |
Investment tax credit carryforwards amount | $ 300,000 | |
Investment tax credit carryforward expiration year | 2020 | |
Shires Mrt Program [Member] | ||
Operating Loss Carryforwards [Line Items] | ||
Income tax benefits | $ 500,000 | $ 5,600,000 |
Minimum [Member] | ||
Operating Loss Carryforwards [Line Items] | ||
Increase in ownership of certain stockholders or public groups in the stock as result of transactions | 50.00% |
Income Taxes - Schedule of Reco
Income Taxes - Schedule of Reconciliation of U.S. Federal Statutory Income Tax Rate and Effective Income Tax Rate (Detail) | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
U.S. federal statutory income tax rate | (21.00%) | (21.00%) |
State income taxes, net of federal benefit | (6.60%) | (6.90%) |
Research and development tax credits and orphan drug credit | (6.80%) | (5.50%) |
Stock-based compensation | 0.50% | 0.50% |
Other permanent differences | 0.20% | 0.30% |
Change in deferred tax asset valuation allowance | 33.60% | 27.70% |
Other | (0.30%) | (0.40%) |
Effective income tax rate | (0.40%) | (5.30%) |
Income Taxes - Schedule of Comp
Income Taxes - Schedule of Components of Deferred Tax Assets and Liabilities (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 |
Deferred tax assets: | |||
Net operating loss carryforwards | $ 61,460 | $ 50,917 | |
Research and development tax credit and orphan drug credit carryforwards | 21,830 | 13,581 | |
Intangibles | 3,607 | ||
Deferred revenue | 11,845 | ||
Depreciation and amortization | 2,253 | 2,380 | |
Accrued expenses and other | 5,354 | 2,894 | |
Total deferred tax assets | 106,349 | 69,772 | |
Deferred tax liabilities: | |||
Indefinite-lived intangible assets | (2,109) | ||
Total deferred tax liabilities | (2,109) | ||
Valuation allowance | (106,346) | (68,143) | $ (39,605) |
Net deferred tax assets | $ 3 | ||
Net deferred tax (liabilities) | $ (480) |
Income Taxes - Summary of Chang
Income Taxes - Summary of Changes in Valuation Allowance for Deferred Tax Assets (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Valuation Allowance [Line Items] | ||
Valuation allowance at beginning of year | $ (68,143) | $ (39,605) |
Valuation allowance at end of year | (106,346) | (68,143) |
Increases Recorded to Income Tax Provision [Member] | ||
Valuation Allowance [Line Items] | ||
Increases/decreases in valuation allowance | $ (38,203) | $ (28,538) |
Net Loss per Share - Summary of
Net Loss per Share - Summary of Basic and Diluted Net Loss Per Share Attributable to Common Stockholders (Detail) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 12 Months Ended | ||||||||
Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2019 | Dec. 31, 2018 | |
Numerator: | ||||||||||
Net loss | $ (31,036) | $ (21,227) | $ (27,832) | $ (33,198) | $ (6,037) | $ (42,646) | $ (27,503) | $ (21,209) | $ (113,293) | $ (97,395) |
Accretion of redeemable convertible preferred stock to redemption value | (644) | |||||||||
Net loss attributable to common stockholders | $ (113,293) | $ (98,039) | ||||||||
Denominator: | ||||||||||
Weighted average common shares outstanding—basic and diluted | 51,445,539 | 26,945,508 | ||||||||
Net loss per share attributable to common stockholders—basic and diluted | $ (0.48) | $ (0.41) | $ (0.57) | $ (0.74) | $ (0.13) | $ (0.97) | $ (3.04) | $ (2.35) | $ (2.20) | $ (3.64) |
Net Loss per Share - Additional
Net Loss per Share - Additional Information (Detail) - shares | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Earnings Per Share, Basic, by Common Class, Including Two Class Method [Line Items] | ||
Antidilutive securities excluded from computation of basic net loss per share | 8,680,546 | 6,455,154 |
Restricted Common Stock [Member] | ||
Earnings Per Share, Basic, by Common Class, Including Two Class Method [Line Items] | ||
Antidilutive securities excluded from computation of basic net loss per share | 34,168 | 219,148 |
Restricted Common Stock [Member] | Weighted Average [Member] | ||
Earnings Per Share, Basic, by Common Class, Including Two Class Method [Line Items] | ||
Antidilutive securities excluded from computation of basic net loss per share | 124,697 | 372,301 |
Net Loss per Share - Summary _2
Net Loss per Share - Summary of Potential Common Shares Excluded from Computation of Diluted Net Loss per Share Attributable to Common Stockholders (Detail) - shares | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Earnings Per Share, Basic, by Common Class, Including Two Class Method [Line Items] | ||
Antidilutive securities excluded from computation of basic net loss per share | 8,680,546 | 6,455,154 |
Stock Options [Member] | ||
Earnings Per Share, Basic, by Common Class, Including Two Class Method [Line Items] | ||
Antidilutive securities excluded from computation of basic net loss per share | 8,646,378 | 6,236,006 |
Restricted Common Stock [Member] | ||
Earnings Per Share, Basic, by Common Class, Including Two Class Method [Line Items] | ||
Antidilutive securities excluded from computation of basic net loss per share | 34,168 | 219,148 |
Leases - Additional Information
Leases - Additional Information (Details) $ in Millions | 1 Months Ended | 12 Months Ended | |
Mar. 31, 2018 | Jun. 30, 2017USD ($)ft² | Dec. 31, 2019USD ($)Lease | |
Lessee Lease Description [Line Items] | |||
Number of operating leases | Lease | 2 | ||
Remaining term of lease | 8 years | ||
Rent expense | $ 3 | ||
Lexington, Massachusetts [Member] | |||
Lessee Lease Description [Line Items] | |||
Office and laboratory space | ft² | 59,000 | ||
Lease, term of contract | 5 years | 10 years | |
Monthly lease payments | $ 0.2 | ||
Lease expiration period | Mar. 31, 2023 | Mar. 31, 2023 | |
Percentage of annual increase in operating lease | 3.00% | ||
Cash deposit collateral | $ 1 | ||
Operating Lease Term Of Contract | 5 years | 10 years | |
Suite Retention And Development Agreement [Member] | |||
Lessee Lease Description [Line Items] | |||
Commitment to Build Out Cost | $ 6 | ||
Shared Overage cost commitment | $ 11 | ||
Maximum [Member] | Lexington, Massachusetts [Member] | |||
Lessee Lease Description [Line Items] | |||
Remaining term of lease | 9 years | ||
Albany Molecular Research, Inc. ("AMRI") [Member] | Suite Retention And Development Agreement [Member] | |||
Lessee Lease Description [Line Items] | |||
Lease, term of contract | 5 years | ||
Monthly lease payments | $ 1 | ||
Payment terms of Build-Out Costs | In the event the Build-Out Costs exceed $6.0 million, the Company and AMRI will share overage costs equally, up to $11.0 million | ||
Operating Lease Term Of Contract | 5 years | ||
Operating Lease Renewal Term | 3 years | ||
Operating lease option to extend description | the Company has the right to extend for an additional three years | ||
Percentage Of Increase In The Monthly Rental Expense | 3.00% | ||
Albany Molecular Research, Inc. ("AMRI") [Member] | Suite Retention And Development Agreement [Member] | Other Noncurrent Assets [Member] | |||
Lessee Lease Description [Line Items] | |||
Build Out Costs paid | $ 2.8 | ||
Albany Molecular Research, Inc. ("AMRI") [Member] | Suite Retention And Development Agreement [Member] | Prepaid Expenses and Other Current Assets [Member] | |||
Lessee Lease Description [Line Items] | |||
Payments For Purchase Of Deliverables | $ 1 |
Leases - Schedule of Lease Cost
Leases - Schedule of Lease Cost (Details) $ in Thousands | 12 Months Ended |
Dec. 31, 2019USD ($) | |
Lease Cost | |
Operating lease cost | $ 2,692 |
Short-term lease cost | 0 |
Total lease cost | 2,692 |
Operating cash flows from operating leases | 2,583 |
Operating lease liabilities arising from obtaining right-of-use assets | $ 0 |
Weighted-average remaining lease term | 8 years |
Weighted-average discount rate | 17.50% |
Leases - Schedule of Maturities
Leases - Schedule of Maturities of Operating Lease Liabilities (Detail) $ in Thousands | Dec. 31, 2019USD ($) |
Leases [Abstract] | |
2019 | $ 2,659 |
2020 | 2,737 |
2021 | 2,818 |
2022 | 2,860 |
2023 | 2,937 |
2024 and thereafter | 10,160 |
Total future minimum lease payments | 24,171 |
Less: imputed interest | (11,557) |
Present value of lease liabilities | $ 12,614 |
Leases - Schedule of Future Min
Leases - Schedule of Future Minimum Rental Commitments for Operating Leases (Detail) $ in Thousands | Dec. 31, 2018USD ($) |
Leases [Abstract] | |
2019 | $ 2,534 |
2020 | 2,610 |
2021 | 2,688 |
2022 | 2,769 |
2023 | 2,852 |
2024 and thereafter | 13,096 |
Total future minimum lease payments | $ 26,549 |
Commitments and Contingencies -
Commitments and Contingencies - Additional Information (Detail) - USD ($) $ in Thousands | 1 Months Ended | 12 Months Ended | ||
Sep. 30, 2019 | Nov. 30, 2016 | Dec. 31, 2019 | Dec. 31, 2018 | |
Loss Contingencies [Line Items] | ||||
Research agreement, payable amount | $ 1,763 | $ 1,901 | ||
Research and development | $ 76,369 | 58,024 | ||
Research agreement, expiration period | 2022-12 | |||
Research agreement, payment | $ 300 | |||
Roche Diagnostics Corporation Master Supply Agreement [Member] | ||||
Loss Contingencies [Line Items] | ||||
Raw material to purchase as percentage of demand | 80.00% | |||
Agreement extended date | Dec. 31, 2024 | |||
Commitment amount | $ 14,400 | |||
Purchase commitments, year 2020 | 500 | |||
Purchase commitments, year 2021 | 3,500 | |||
Purchase commitments, year 2022 | 3,500 | |||
Purchase commitments, year 2023 | 3,500 | |||
Purchase commitments, year 2024 | 3,500 | |||
Research and development | 12,500 | 5,000 | ||
MIT Research Agreement [Member] | ||||
Loss Contingencies [Line Items] | ||||
Research agreement, payable amount | 0 | 700 | ||
Research and development | 700 | 1,200 | ||
Research agreement, committed amount | $ 4,100 | $ 3,100 | ||
Research agreement, expiration period | 2022-12 | 2019-10 | ||
Research agreement, payment | 3,100 | 2,500 | ||
MIT Exclusive Patent License Agreement [Member] | ||||
Loss Contingencies [Line Items] | ||||
Research and development | 200 | 100 | ||
Annual license maintenance payments | 200 | |||
Payments of annual license maintenance fees | 200 | $ 100 | ||
MIT Exclusive Patent License Agreement [Member] | Sanofi Pasteur Inc [Member] | ||||
Loss Contingencies [Line Items] | ||||
Upfront payment received | 700 | |||
MIT Exclusive Patent License Agreement [Member] | Milestone Payment One [Member] | ||||
Loss Contingencies [Line Items] | ||||
License agreement, milestone payments | 1,375 | |||
MIT Exclusive Patent License Agreement [Member] | Milestone Payment Two [Member] | ||||
Loss Contingencies [Line Items] | ||||
License agreement, milestone payments | 1,250 | |||
Accounts Payable | Roche Diagnostics Corporation Master Supply Agreement [Member] | ||||
Loss Contingencies [Line Items] | ||||
Purchase commitments | $ 10,100 |
Related Party Transactions - Ad
Related Party Transactions - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Millions | 1 Months Ended | 12 Months Ended | ||
Sep. 30, 2019 | Dec. 31, 2019 | Sep. 20, 2019 | May 03, 2019 | |
Private Placement [Member] | ||||
Related Party Transaction [Line Items] | ||||
Sale of Stock, Price Per Share | $ 8.50 | |||
Private Placement [Member] | Baupost Group LLC [Member] | ||||
Related Party Transaction [Line Items] | ||||
Sale of Stock, Number of Shares Issued in Transaction | 2,352,941 | |||
Sale of Stock, Price Per Share | $ 8.50 | |||
Sale of Stock, Consideration Received on Transaction | $ 20 | |||
Public Offering [Member] | ||||
Related Party Transaction [Line Items] | ||||
Sale of Stock, Price Per Share | $ 10 | |||
Public Offering [Member] | Baupost Group LLC [Member] | ||||
Related Party Transaction [Line Items] | ||||
Sale of Stock, Number of Shares Issued in Transaction | 5,000,000 | |||
Sale of Stock, Price Per Share | $ 10 | |||
Sale of Stock, Consideration Received on Transaction | $ 50 |
Benefit Plans - Additional Info
Benefit Plans - Additional Information (Detail) - USD ($) $ in Millions | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Retirement Benefits [Abstract] | ||
Employer's discretionary contribution to the plan | $ 0.4 | $ 0.3 |
Selected Quarterly Financial _3
Selected Quarterly Financial Information (Unaudited) - Schedule of Quarterly Financial Information (Detail) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 12 Months Ended | ||||||||
Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2019 | Dec. 31, 2018 | |
Selected Quarterly Financial Information [Abstract] | ||||||||||
Collaboration revenue | $ 3,890 | $ 1,266 | $ 1,174 | $ 1,474 | $ 1,182 | $ 238 | $ 7,804 | $ 1,420 | ||
Total operating expenses | 35,629 | 22,901 | 29,364 | 35,679 | 8,480 | 45,719 | $ 29,062 | $ 22,389 | 123,573 | 105,650 |
Loss from operations | (31,739) | (21,635) | (28,190) | (34,205) | (7,298) | (45,481) | (29,062) | (22,389) | (115,769) | (104,230) |
Net loss | $ (31,036) | $ (21,227) | $ (27,832) | $ (33,198) | $ (6,037) | $ (42,646) | $ (27,503) | $ (21,209) | $ (113,293) | $ (97,395) |
Net loss per share attributable to common stockholders—basic and diluted | $ (0.48) | $ (0.41) | $ (0.57) | $ (0.74) | $ (0.13) | $ (0.97) | $ (3.04) | $ (2.35) | $ (2.20) | $ (3.64) |
Selected Quarterly Financial _4
Selected Quarterly Financial Information (Unaudited) - Additional Information (Detail) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2019 | Dec. 31, 2018 | |
Selected Quarterly Financial Information [Abstract] | |||
Decrease in fair value of contingent consideration | $ 14,600 | $ 13 | $ 25,020 |