Document and Entity Information
Document and Entity Information - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2018 | Feb. 28, 2019 | Jun. 30, 2018 | |
Document And Entity Information [Abstract] | |||
Document Type | 10-K | ||
Amendment Flag | false | ||
Document Period End Date | Dec. 31, 2018 | ||
Document Fiscal Year Focus | 2,018 | ||
Document Fiscal Period Focus | FY | ||
Trading Symbol | ADVM | ||
Entity Registrant Name | Adverum Biotechnologies, Inc. | ||
Entity Central Index Key | 1,501,756 | ||
Current Fiscal Year End Date | --12-31 | ||
Entity Well-known Seasoned Issuer | No | ||
Entity Current Reporting Status | Yes | ||
Entity Voluntary Filers | No | ||
Entity Filer Category | Accelerated Filer | ||
Entity Emerging Growth Company | true | ||
Entity Ex Transition Period | true | ||
Entity Small Business | true | ||
Entity Shell Company | false | ||
Entity Common Stock, Shares Outstanding | 63,201,009 | ||
Entity Public Float | $ 294.8 |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) $ in Thousands | Dec. 31, 2018 | Dec. 31, 2017 |
Current assets: | ||
Cash and cash equivalents | $ 154,949 | $ 70,519 |
Short-term investments | 50,130 | 119,966 |
Prepaid expenses and other current assets | 3,675 | 3,256 |
Total current assets | 208,754 | 193,741 |
Property and equipment, net | 3,586 | 3,024 |
Restricted cash | 999 | |
Deposit and other non-current assets | 156 | 140 |
Intangible asset | 5,000 | |
Total assets | 213,495 | 201,905 |
Current liabilities: | ||
Accounts payable | 1,707 | 1,731 |
Accrued expenses and other current liabilities | 8,784 | 6,964 |
Deferred rent, current portion | 228 | 129 |
Deferred revenue, current portion | 1,850 | |
Total current liabilities | 10,719 | 10,674 |
Long-term liabilities: | ||
Deferred rent, net of current portion | 1,366 | 222 |
Deferred revenue, net of current portion | 5,250 | |
Deferred tax liability, non-current | 1,250 | |
Other non-current liabilities | 243 | 481 |
Total liabilities | 12,328 | 17,877 |
Commitments and contingencies (Note 10) | ||
Stockholders’ equity: | ||
Preferred stock, $0.0001 par value, 5,000,000 shares authorized; no shares issued and outstanding | ||
Common stock, $0.0001 par value, 300,000,000 shares authorized at December 31, 2018 and 2017; 62,965,468 and 49,015,339 shares issued and outstanding at December 31, 2018 and 2017, respectively | 6 | 5 |
Additional paid-in capital | 522,503 | 439,048 |
Accumulated other comprehensive loss | (799) | (963) |
Accumulated deficit | (320,543) | (254,062) |
Total stockholders’ equity | 201,167 | 184,028 |
Total liabilities and stockholders’ equity | $ 213,495 | $ 201,905 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - $ / shares | Dec. 31, 2018 | Dec. 31, 2017 |
Statement Of Financial Position [Abstract] | ||
Preferred stock, par value | $ 0.0001 | $ 0.0001 |
Preferred stock, shares authorized | 5,000,000 | 5,000,000 |
Preferred stock, shares issued | 0 | 0 |
Preferred stock, shares outstanding | 0 | 0 |
Common stock, par value | $ 0.0001 | $ 0.0001 |
Common stock, shares authorized | 300,000,000 | 300,000,000 |
Common stock, shares issued | 62,965,468 | 49,015,339 |
Common stock, shares outstanding | 62,965,468 | 49,015,339 |
Consolidated Statements of Oper
Consolidated Statements of Operations and Comprehensive Loss - USD ($) shares in Thousands | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Income Statement [Abstract] | ||
Collaboration and license revenue | $ 1,612,000 | $ 1,849,000 |
Type of Revenue [Extensible List] | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember |
Operating expenses: | ||
Research and development | $ 50,133,000 | $ 39,839,000 |
General and administrative | 24,560,000 | 20,857,000 |
Impairment of goodwill and intangible assets | 5,000,000 | |
Total operating expenses | 79,693,000 | 60,696,000 |
Operating loss | (78,081,000) | (58,847,000) |
Other income: | ||
Other income, net | 4,204,000 | 2,700,000 |
Net loss before income taxes | (73,877,000) | (56,147,000) |
Income tax benefit | 1,250,000 | 0 |
Net loss | (72,627,000) | (56,147,000) |
Other comprehensive loss: | ||
Net unrealized gain (loss) on marketable securities | 168,000 | (182,000) |
Foreign currency translation adjustment | (4,000) | (774,000) |
Comprehensive loss | $ (72,463,000) | $ (57,103,000) |
Net loss per share - basic and diluted | $ (1.18) | $ (1.29) |
Weighted-average common shares outstanding-basic and diluted | 61,375 | 43,661 |
Consolidated Statements of Stoc
Consolidated Statements of Stockholders' Equity - USD ($) $ in Thousands | Total | Common Stock [Member] | Additional Paid-in Capital [Member] | Accumulated Other Comprehensive Income (Loss) [Member] | Accumulated Deficit [Member] |
Balance at Dec. 31, 2016 | $ 215,600 | $ 4 | $ 413,518 | $ (7) | $ (197,915) |
Balance, shares at Dec. 31, 2016 | 41,805,009 | ||||
Issuance of common stock net of issuance costs | 16,519 | $ 1 | 16,518 | ||
Issuance of common stock net of issuance costs, shares | 5,130,339 | ||||
Remeasurement of contingent common stock warrant in consideration for services | 60 | 60 | |||
Stock-based compensation expense | 8,723 | 8,723 | |||
Common stock issued upon exercise of stock options | 367 | 367 | |||
Common stock issued upon exercise of stock options, shares | 1,808,696 | ||||
Common stock issued under employee stock purchase plan | 175 | 175 | |||
Common stock issued under employee stock purchase plan, shares | 74,642 | ||||
Common stock issued upon release of restricted stock units, shares | 307,610 | ||||
Restricted stock surrendered for taxes | (313) | (313) | |||
Restricted stock surrendered for taxes, shares | (110,957) | ||||
Net unrealized gain (loss) on marketable securities | (182) | (182) | |||
Foreign currency translation adjustment | (774) | (774) | |||
Net loss | (56,147) | (56,147) | |||
Balance at Dec. 31, 2017 | 184,028 | $ 5 | 439,048 | (963) | (254,062) |
Balance, shares at Dec. 31, 2017 | 49,015,339 | ||||
Issuance of common stock net of issuance costs | 70,187 | $ 1 | 70,186 | ||
Issuance of common stock net of issuance costs, shares | 11,642,128 | ||||
Adoption of Topic 606 | 6,146 | 6,146 | |||
Stock-based compensation expense | 13,432 | 13,432 | |||
Common stock issued upon exercise of stock options | 688 | 688 | |||
Common stock issued upon exercise of stock options, shares | 1,606,137 | ||||
Common stock issued under employee stock purchase plan | 340 | 340 | |||
Common stock issued under employee stock purchase plan, shares | 120,475 | ||||
Common stock issued upon release of restricted stock units, shares | 773,965 | ||||
Restricted stock surrendered for taxes | (1,191) | (1,191) | |||
Restricted stock surrendered for taxes, shares | (192,576) | ||||
Net unrealized gain (loss) on marketable securities | 168 | 168 | |||
Foreign currency translation adjustment | (4) | (4) | |||
Net loss | (72,627) | (72,627) | |||
Balance at Dec. 31, 2018 | $ 201,167 | $ 6 | $ 522,503 | $ (799) | $ (320,543) |
Balance, shares at Dec. 31, 2018 | 62,965,468 |
Consolidated Statements of St_2
Consolidated Statements of Stockholders' Equity (Parenthetical) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Statement Of Stockholders Equity [Abstract] | ||
Issuance costs | $ 4,140 | $ 230 |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Cash flows from operating activities: | ||
Net loss | $ (72,627) | $ (56,147) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Depreciation and amortization | 1,750 | 2,096 |
Stock-based compensation expense | 13,432 | 8,723 |
Amortization of premium on marketable securities | 24 | 593 |
Accreted interest on BPI | 95 | |
Impairment of goodwill and intangible assets | 5,000 | |
Non-cash research and development expense | 60 | |
Other | (17) | 10 |
Changes in operating assets and liabilities: | ||
Accounts receivable, net | 886 | |
Prepaid expenses and other current assets | (793) | (491) |
Deposit and other long-term assets | (16) | |
Accounts payable | (67) | 333 |
Accrued expenses and other current liabilities | 215 | 487 |
Restructuring liabilities | (25) | |
Deferred revenue | (953) | (1,849) |
Deferred rent | 1,243 | (97) |
Deferred tax liability | (1,250) | |
Net cash used in operating activities | (53,964) | (45,421) |
Cash flows from investing activities: | ||
Purchases of marketable securities | (78,726) | (209,787) |
Sales of marketable securities | 1,003 | |
Maturities of marketable securities | 148,979 | 87,596 |
Purchases of property and equipment | (809) | (1,016) |
Net cash provided by (used in) provided by investing activities | 69,444 | (122,204) |
Cash flows from financing activities: | ||
Proceeds from offering of common stock, net of issuance costs | 70,187 | 16,519 |
Proceeds from issuance of common stock pursuant to option exercises | 688 | 367 |
Taxes paid related to net share settlement of restricted stock units | (1,191) | (313) |
Proceeds from employee stock purchase plan | 340 | 175 |
Repayment of BPI loan | (175) | |
Proceeds from a financing arrangement | 100 | |
Net cash provided by financing activities | 69,949 | 16,748 |
Effect of foreign currency exchange rate on cash and cash equivalents | (774) | |
Net increase (decrease) in cash and cash equivalents and restricted cash | 85,429 | (151,651) |
Cash and cash equivalents and restricted cash at beginning of period | 70,519 | 222,170 |
Cash and cash equivalents and restricted cash at end of period | 155,948 | 70,519 |
Supplemental schedule of noncash investing and financing information | ||
Fixed assets in accounts payable and current liabilities | $ 1,616 | $ 115 |
Description of the Business
Description of the Business | 12 Months Ended |
Dec. 31, 2018 | |
Accounting Policies [Abstract] | |
Description of the business | 1. Description of the business Nature of Business —Adverum Biotechnologies, Inc. (the “Company” or “Adverum”) was incorporated in Delaware on July 17, 2006 and is headquartered in Menlo Park, California. The Company is a clinical-stage gene therapy company targeting unmet medical need in ocular and rare diseases. The Company develops gene therapy product candidates designed to provide durable efficacy by inducing sustained expression of a therapeutic protein. The Company’s core capabilities include clinical development, novel vector discovery, and in-house manufacturing expertise, specifically in scalable process development, assay development, and current Good Manufacturing Practices (“cGMP”) quality control. Adverum is advancing a pipeline of gene therapy product candidates designed to treat wet age-related macular degeneration (“AMD”) as well as rare diseases. Since the Company’s inception, it has devoted its efforts principally to performing research and development activities, including conducting preclinical studies, early clinical trials, filing patent applications, obtaining regulatory agreements, hiring personnel, and raising capital to support these activities The Company has not generated any revenue from the sale of products since its inception. The Company has experienced net losses since its inception and had an accumulated deficit of $320.5 million as of December 31, 2018. The Company expects to incur losses and have negative net cash flows from operating activities as it engages in further research and development activities. The Company believes that it has sufficient funds to continue operations into 2021. In May 2016, the Company completed the acquisition of all of the outstanding shares of Annapurna Therapeutics SAS (“Annapurna”), a privately-held French gene therapy company, in accordance with the terms of the acquisition agreement (the “Annapurna acquisition”) dated as of January 29, 2016, as amended on April 6, 2016. As a result, Annapurna is now a wholly owned subsidiary of the Company. Upon completion of the Annapurna acquisition, the Company changed its name to “Adverum Biotechnologies, Inc.” The Company’s shares of common stock listed on The Nasdaq Global Market, previously trading through the close of business on May 11, 2016 under the ticker symbol “AAVL,” commenced trading on The Nasdaq Global Market under the ticker symbol “ADVM” on May 12, 2016. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 12 Months Ended |
Dec. 31, 2018 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of significant accounting policies Basis of Presentation and Principles of Consolidation — The Company’s consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States of America (“U.S. GAAP”) and pursuant to the rules and regulations of the Securities and Exchange Commission (“SEC”). The consolidated financial statements include the accounts of the Company and its wholly-owned subsidiaries. All inter-company transactions and balances have been eliminated in consolidation. Use of Estimates —The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets, liabilities, and the disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of income and expenses during the reporting period. The Company bases estimates and assumptions on historical experience when available and on various factors that it believes to be reasonable under the circumstances. On an ongoing basis, the Company evaluates its estimates and assumptions, including those related to research and development expense accruals, stock-based compensation expense, income taxes, intangible asset, fair values of financial instruments and fair value of common stock warrants. The Company’s actual results may differ from these estimates under different assumptions or conditions. There have been no significant changes from the Company’s original estimates in any periods presented. Foreign Currency Translation —The Company’s consolidated financial statements are prepared in U.S. dollars. The Company’s foreign subsidiaries use the Euro and Australian dollar as their functional currencies and maintain their records in their local currencies, except its Ireland subsidiary that uses the U.S. dollar as its functional currency. Assets and liabilities are re-measured at exchange rates in effect at the end of the reporting period for the Company’s French and Australian subsidiaries, and at historical exchange rates for its Irish subsidiary. Equity is measured at historical rates and income and expenses are re-measured at average exchange rates for the reporting period. The resulting foreign currency translation adjustment is recorded in accumulated other comprehensive loss in the consolidated balance sheet. Transactions denominated in foreign currency are translated at exchange rates at the date of transaction with foreign currency gains (losses) recorded in other income (expense), net in the consolidated statements of operations and comprehensive loss. Cash and Cash Equivalents —The Company considers all highly liquid investments purchased with original maturities of three months or less at the date of purchase to be cash equivalents. Cash and cash equivalents include cash held in banks, money market accounts and highly liquid debt securities. Cash equivalents are stated at fair value. Restricted Cash —Restricted cash primarily consists of cash collateral to letter of credit provided to the landlord in relation to a lease agreement (see Note 10). Short-Term Investments —All short-term investments, which consist of debt securities and certificates of deposit, have been classified as “available for sale” and are carried at fair value. Unrealized gains and losses, net of any related tax effects, are excluded from earnings and are included in other comprehensive loss and reported as a separate component of stockholders’ equity until realized. Realized gains and losses and declines in value judged to be other than temporary, if any, on available-for-sale securities are included in other income (expense), net in the Company’s consolidated statements of operations and comprehensive loss. The cost of securities sold is based on the specific-identification method. The amortized cost of securities is adjusted for amortization of premiums and accretion of discounts to maturity. Interest on short-term investments is included in other income, net in the Company’s consolidated statements of operations and comprehensive loss. In accordance with the Company’s investment policy, management invests to diversify credit risk and only invests in securities with high credit quality, including U.S. government securities. The Company periodically evaluates whether declines in the fair value of its investments below their cost are other than temporary. The evaluation includes consideration of the cause of the impairment, including the creditworthiness of the security issuers, the number of securities in an unrealized loss position, the severity and duration of the unrealized losses, whether the Company has the intent to sell the securities, and whether it is more likely than not that the Company will be required to sell the securities before the recovery of their amortized cost basis. If the Company determines that the decline in fair value of an investment is below its accounting basis and this decline is other than temporary, the Company would reduce the carrying value of the security it holds and records a loss for the amount of such decline. The Company has not recorded any realized losses or declines in value judged to be other than temporary on its investments. Segment Reporting —The Company operates and manages its business as one reporting and operating segment, which is the business of developing and commercializing gene therapeutics. The Company’s chief executive officer, who is the chief operating decision maker, reviews financial information on an aggregate basis for purposes of allocating resources and evaluating financial performance. Concentrations of Credit Risk and Other Uncertainties —Financial instruments that potentially subject the Company to significant concentrations of credit risk consists primarily of cash, cash equivalents and short-term investments. Risks associated with cash, cash equivalents, short-term investments are mitigated by the Company’s investment policy, which limits the Company’s investing to only those marketable securities rated at least A-1/P-1 Short Term Rating and A/A2 Long Term Rating, as determined by independent credit rating agencies. Management believes that the Company is not exposed to significant credit risk. The Company is subject to certain risks and uncertainties, including, but not limited to changes in any of the following areas that the Company believes could have a material adverse effect on future financial position or results of operations: ability to obtain future financing; regulatory approval and market acceptance of, and reimbursement for, the Company’s product candidates; performance of third-party clinical research organizations and manufacturers; development of sales channels; protection of the intellectual property; litigation or claims against the Company based on intellectual property, patent, product, regulatory or other factors; and the Company’s ability to attract and retain employees necessary to support the growth. Property and Equipment —Property and equipment are recorded at cost, net of accumulated depreciation and amortization. Depreciation is recorded using the straight-line method over the estimated useful lives of the assets, generally three to five years. Leasehold improvements are capitalized and amortized over the shorter period of their expected lives or the lease term. Major replacements and improvements are capitalized, while general repairs and maintenance are expensed as incurred. Valuation of Long-Lived Assets and Purchased Intangible Assets —The Company evaluates the carrying value of amortizable long‑lived assets, whenever events, or changes in business circumstances or the planned use of long‑lived assets indicate that their carrying amounts may not be fully recoverable or that their useful lives are no longer appropriate. If these facts and circumstances exist, the Company assesses for recovery by comparing the carrying values of long‑lived assets with their future undiscounted net cash flows. If the comparison indicates that impairment exists, long‑lived assets are written down to their respective fair values based on discounted cash flows. Significant management judgment is required in the forecasting of future operating results that is used in the preparation of expected undiscounted cash flows. If management’s assumptions about future operating results were to change as a result of events or circumstances, the Company may be required to record an impairment loss on these assets. There were no impairment indicators noted for the Company’s amortizable long-lived assets, fixed assets, in the year ended December 31, 2017. The Company also evaluates the carrying value of intangible asset (not subject to amortization) related to in‑process research and development (“IPR&D”) asset, which is considered to be indefinite‑lived until the completion or abandonment of the associated research and development efforts. Accordingly, amortization of the IPR&D assets will not occur until the product reaches commercialization. During the period the intangible asset is considered indefinite‑lived, it will be tested for impairment on an annual basis, as well as between annual tests if the Company becomes aware of any events occurring or changes in circumstances that would indicate that the fair value of the IPR&D asset is less than its carrying amount. If and when development is complete, which generally occurs when regulatory approval to market the product is obtained, the associated IPR&D asset would be deemed definite‑lived and would then be amortized based on its estimated useful life at that point in time based on respective patent term. If a potential impairment exists, an impairment loss is measured as the excess of the asset’s carrying value over its fair value. During the year ended December 31, 2018, the Company recorded an impairment charge of $5.0 million related to its intangible assets. (see Note 3). Financial Liabilities — During the year ended December 31, 2016, the Company entered into a sponsored research agreement with The Alpha-1 Project, Inc. (the “TAP”) with an embedded derivative, the Company elected to account for this financial liability at fair value and recorded as other non-current liabilities in its consolidated balance sheets (see Note 5). The change in fair value is recorded within other income, net in the Company’s consolidated statement of operations and comprehensive loss. Revenue Recognition —The Company has primarily generated revenue through license, research and collaboration arrangements with its strategic partners. Effective January 1, 2018, the Company adopted the new revenue standards under Topic 606 using the modified retrospective approach. Results for reporting periods beginning after January 1, 2018 are presented under Topic 606, while prior period results are not adjusted and continue to be reported in accordance with the revenue standards under Topic 605. Under Topic 606, the Company recognizes revenue when its customer obtains control of promised goods or services, in an amount that reflects the consideration which the Company expects to receive in exchange for those goods or services. To determine revenue recognition for arrangements that the Company determines are within the scope of ASC 606, the Company performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) the Company satisfies a performance obligation. At contract inception, once the contract is determined to be within the scope of ASC 606, the Company assesses the goods or services promised within each contract and determines those that are performance obligations. The Company then recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) the performance obligation is satisfied. The Company’s license and collaboration arrangements with Regeneron Pharmaceuticals, Inc. (“Regeneron”), Editas Medicine, Inc. (“Editas”), and GenSight Biologics (“GenSight”) are within the scope of Topic 606. Upon the adoption of Topic 606, the Company recorded a net decrease of $6.1 million to its deferred revenue and opening accumulated deficit as of January 1, 2018 for the cumulative effect of the adoption. The effect of the adoption is summarized for the Company’s license and collaboration agreements as follows: Collaboration and License Revenue Collaboration Agreement with Regeneron — Under Topic 606, the transaction price at contract inception was determined to be $8.0 million, which was related to the non-refundable upfront payment for license and research services. The arrangement also provides for additional payments to the Company when certain development and regulatory milestones are achieved. Because these milestone payments are not within the control of the Company and are not considered probable of being achieved until the events occur, the Company did not include them in the transaction price at contract inception. The transaction price of $8.0 million at contract inception was allocated to two performance obligations. The Company’s deferred revenue associated with its Regeneron collaboration agreement as of December 31, 2017 under Topic 605 was $6.5 million. As a result of adopting Topic 606, the Company recorded a $6.5 million reduction to its deferred revenue and opening accumulated deficit during the three months ended March 31, 2018 as the performance obligations associated with the Regeneron deferred revenue were satisfied as of January 1, 2018. There was no outstanding deferred revenue associated with Regeneron as of March 31, 2018 or December 31, 2018. Collaboration Agreement with Editas — Under Topic 606, the transaction price at contract inception was determined to be $1.0 million, which was related to the non-refundable upfront payment for license and research services. The arrangement provides for additional payments to the Company when certain development and regulatory milestones are achieved. Because these milestone payments are not within the control of the Company and are not considered probable of being achieved until the events occur, the Company did not include them in the transaction price at contract inception. The transaction price of $1.0 million at contract inception was allocated to a single performance obligation. The Company’s deferred revenue associated with its Editas collaboration agreement as of December 31, 2017 under Topic 605 was $0.5 million. As a result of adopting Topic 606, the Company recorded an increase of $0.4 million to its deferred revenue and opening accumulated deficit during the three months ended March 31, 2018 due to differences in the timing of recognition under Topic 606. During the year ended December 31, 2018, the Company recognized revenue of $1.4 million associated with the Editas collaboration agreement. There was no outstanding deferred revenue associated with Editas as of December 31, 2018. License Agreement with GenSight — On February 2014, the Company entered into an agreement with GenSight, where the Company granted GenSight a non-exclusive license to its proprietary AAV.7m8 vector. Under the agreement, the Company is eligible to receive development, regulatory and commercial milestones. Also, the Company is eligible to receive low to mid-single digit royalties on sales of GenSight’s licensed products. During the year ended December 31, 2018, GenSight achieved a clinical development milestone pursuant to the agreement. This milestone was previously constrained under Topic 606. The Company earned a $0.2 million milestone payment, which was recognized as revenue in the consolidated statement of operations and comprehensive loss for the year ended December 31, 2018. Under Topic 605, the Company’s revenue for the year ended December 31, 2018 would have been $2.2 million. Research and Development Expenses —Research and development expenses are charged to expense as incurred. Research and development expenses include primarily personnel-related costs, stock-based compensation expense, laboratory supplies, consulting costs, external contract research and development expenses, including expenses incurred under agreements with CROs, the cost of acquiring, developing and manufacturing clinical trial materials, and overhead expenses, such as rent, equipment depreciation, insurance and utilities. Advance payments for goods or services for future research and development activities are deferred and expensed as the goods are delivered or the related services are performed. The Company estimates preclinical studies and clinical trial expenses based on the services performed pursuant to contracts with research institutions and clinical research organizations that conduct and manage preclinical studies and clinical trials on the Company’s behalf. In accruing service fees, the Company estimates the time period over which services will be performed and the level of effort to be expended in each period. These estimates are based on communications with the third-party service providers and the Company’s estimates of accrued expenses and on information available at each balance sheet date. If the actual timing of the performance of services or the level of effort varies from the estimate, the Company will adjust the accrual accordingly. There have been no significant changes from the Company’s original estimates in any of the periods presented. Fair Value Measurements —Fair value is defined as the price that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants at the measurement date. As such, fair value is a market-based measurement that should be determined based on assumptions that market participants would use in pricing an asset or liability. The carrying amounts of the Company’s financial instruments, including cash equivalents, prepaid and other current assets, accounts payable, accrued expenses and other current liabilities approximate their fair values due to their short-term maturities. Refer to Note 5 for the methodologies and assumptions used in valuing financial instruments. Stock-Based Compensation Expense —Stock-based compensation expense related to stock awards to employees is measured at fair value of the award on the date of the grant. The Company estimates the grant-date fair value, and the resulting stock-based compensation expense, using the Black-Scholes valuation model for stock options and using intrinsic value, which is the closing price of its common stock on the date of the grant, for the restricted stock units (“RSUs”). The fair value of the award that is ultimately expected to vest is recognized as expense on a straight-line basis over the requisite service period, which is generally the vesting period. The Black-Scholes valuation model requires the use of following assumptions: Expected Term —The expected term assumption represents the period that the Company’s stock-based awards are expected to be outstanding and is determined using the simplified method. Expected Volatility —Expected volatility is estimated using comparable public companies’ volatility for similar terms. Expected Dividend —The Black-Scholes valuation model calls for a single expected dividend yield as an input. The Company has never paid dividends and has no plans to pay dividends. Risk-Free Interest Rate —The risk-free interest rate is based on the U.S. Treasury zero-coupon issues in effect at the time of grant for periods corresponding with the expected term of option. Stock-based compensation expense related to awards granted to non-employees is recognized based on the then-current fair value at each measurement date over the associated service period of the award, which is generally the vesting term, using the accelerated attribution method. The fair value of non-employee stock options is estimated using the Black-Scholes valuation model with assumptions generally consistent with those used for employee stock options, with the exception of the expected term, which is the remaining contractual life at each measurement date. Refer to Note 12 for more information on assumptions used in estimating stock-based compensation expense. Income Taxes —The Company accounts for income taxes using the asset and liability method. The Company recognizes deferred tax assets and liabilities for the expected future tax consequences of events that have been included in the financial statements or tax returns. Deferred tax assets and liabilities are determined based on the difference 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. In evaluating the ability to recover its deferred income tax assets, the Company considers all available positive and negative evidence, including its operating results, ongoing tax planning and forecasts of future taxable income on a jurisdiction-by-jurisdiction basis. In the event the Company determines that it would be able to realize its deferred income tax assets in the future in excess of their net recorded amount, it would make an adjustment to the valuation allowance that would reduce the provision for income taxes. Conversely, in the event that all or part of the net deferred tax assets are determined not to be realizable in the future, an adjustment to the valuation allowance would be charged to earnings in the period when such determination is made. As of December 31, 2018 and 2017, the Company has recorded a full valuation allowance on its deferred tax assets. Tax benefits related to uncertain tax positions are recognized when it is more likely than not that a tax position will be sustained during an audit. Interest and penalties related to unrecognized tax benefits are included within the provision for income tax. Comprehensive Loss —Comprehensive loss comprises net loss and other comprehensive income (loss). Other comprehensive income (loss) consists of foreign currency translation adjustments related to translation of the financial statements of the Company’s Australia and France subsidiaries and unrealized gain (loss) on marketable securities. The Company did not have reclassifications from other comprehensive income (loss) to the income (loss) during the years ended December 31, 2018 and 2017. Basic and Diluted Net Loss Per Share — Basic net loss per share is calculated by dividing the net loss by the weighted-average number of shares of common stock outstanding for the period. Diluted net loss per share is computed by giving effect to all potential dilutive common stock equivalents outstanding for the period using the treasury stock method. Outstanding stock options, RSUs, ESPP and warrants are considered to be common stock equivalents and are only included in the calculation of diluted net loss per share when their effect is dilutive. Recent Accounting Standard Update Not Yet Effective — In February 2016, the Financial Accounting Standards Board (“FASB”) issued ASU No. 2016-2, Leases, which amends the current guidance on leasing activities to provide more transparency and comparability, and requires that all leases be recognized by lessees on their balance sheet as a right-of-use asset and corresponding lease liability, which are currently accounted for as operating leases. The new standard is effective for fiscal years and interim periods within those fiscal years beginning after December 15, 2018, with early adoption permitted. We adopted the new standard using the modified retrospective approach as of January 1, 2019 and expect to record a lease liability in the range of $24.0 million to $27.0 million, and a right‑to‑use asset of approximately $23.0 million to $26.0 million, and no adjustment to the accumulated deficit. While the Company continues to evaluate the effect of the standard, the Company anticipates that the adoption will result in a material increase in assets and liabilities on its consolidated balance sheet and will not have a material impact on the consolidated statement of operations or statement of cash flows. In June 2018, the FASB issued ASU 2018-07, “Compensation – Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting” that expands the scope of ASC Topic 718 to include share-based payment transactions for acquiring goods and services from nonemployees. An entity should apply the requirements of ASC Topic 718 to nonemployee awards except for certain exemptions specified in the amendment. The guidance is effective for fiscal years beginning after December 15, 2018, including interim reporting periods within that fiscal year. Early adoption is permitted, but no earlier than an entity’s adoption date of Topic 606. The Company is currently evaluating the impact of the guidance on its consolidated financial statements. |
Impairment Evaluation for Goodw
Impairment Evaluation for Goodwill and Intangible Assets | 12 Months Ended |
Dec. 31, 2018 | |
Goodwill And Intangible Assets Disclosure [Abstract] | |
Impairment Evaluation for Goodwill and Intangible Assets | 3. Impairment Evaluation for Goodwill and Intangible Assets The Company recorded In-process Research and Development (“IPR&D”) intangible assets upon the acquisition of Annapurna in May 2016. The carrying value of the IPR&D intangible asset was $5.0 million as of June 30, 2018. The Company evaluates indefinite lived intangible assets for impairment on an annual basis or more frequently if indicators of impairment exist. As the Company recorded goodwill and IPR&D intangible assets upon the Annapurna acquisition, the Company is required to test goodwill and indefinite lived intangible assets for impairment on an annual basis or more frequently if indicators of impairment exist. The Company operates as one reporting unit and goodwill was allocated to this reporting unit. During the year ended December 31, 2018, the Company identified an impairment indicator related to the intangible asset and performed an impairment analysis. On October 30, 2018, the Company decided to discontinue the development of ADVM‑043. The Company recorded an impairment charge of $5.0 million on IPR&D assets related to the Company’s intangible asset for ADVM‑043. This amount was recorded in Impairment of intangible assets on the Company’s consolidated statements of operations and comprehensive loss. |
Cash Equivalents and Short-Term
Cash Equivalents and Short-Term Investments | 12 Months Ended |
Dec. 31, 2018 | |
Cash And Cash Equivalents [Abstract] | |
Cash Equivalents and Short-Term Investments | 4. Cash Equivalents and Short-Term Investments The following is a summary of the Company’s cash equivalents and short-term investments (in thousands): December 31, 2018 Amortized Cost Basis Unrealized Gains Unrealized Loses Estimated Fair Value Money market funds $ 126 $ — $ — $ 126 U.S. government and agency securities 25,792 1 (4 ) 25,789 Commercial paper 147,606 — — 147,606 Corporate bonds 27,778 5 (17 ) 27,766 Certificates of deposit 1,420 — — 1,420 Total cash equivalents and short-term investments 202,722 6 (21 ) 202,707 Less: Cash equivalents (152,577 ) — — (152,577 ) Total short-term investments $ 50,145 $ 6 $ (21 ) $ 50,130 December 31, 2017 Money market funds $ 65 $ — $ — $ 65 U.S. government and agency securities 58,351 — (145 ) 58,206 Commercial paper 71,427 — — 71,427 Corporate bonds 38,354 1 (38 ) 38,317 Certificates of deposit 9,731 — — 9,731 Total cash equivalents and short-term investments 177,928 1 (183 ) 177,746 Less: Cash equivalents (57,780 ) — — (57,780 ) Total short-term investments $ 120,148 $ 1 $ (183 ) $ 119,966 Management determined that the gross unrealized losses on the Company’s marketable securities as of December 31, 2018 were temporary in nature. Therefore, none of the Company’s marketable securities were other-than-temporarily impaired as of December 31, 2018. All investments as at December 31, 2018 have a remaining maturity of one year or less. |
Fair Value Measurements and Fai
Fair Value Measurements and Fair Value of Financial Instruments | 12 Months Ended |
Dec. 31, 2018 | |
Fair Value Disclosures [Abstract] | |
Fair Value Measurements and Fair Value of Financial Instruments | 5. Fair Value Measurements and Fair Value of Financial Instruments The authoritative guidance on fair value measurements establishes a three-tier fair value hierarchy for disclosure of fair value measurements as follows: Level 1: Quoted prices in active markets for identical assets or liabilities. Level 2: Observable inputs other than Level 1 prices, such as quoted prices for similar assets or liabilities, quoted prices in markets that are not active, or other inputs that are observable or can be corroborated by observable market data for substantially the full term of the assets or liabilities. Level 3: Unobservable inputs that are supported by little or no market activity and that are significant to the fair value of the assets or liabilities. Assets and liabilities measured at fair value are classified in their entirety based on the lowest level of input that is significant to the fair value measurement. The Company’s assessment of the significance of a particular input to the fair value measurement in its entirety requires management to make judgments and consider factors specific to the asset or liability. The fair value of Level 1 securities is determined using quoted prices in active markets for identical assets. Level 1 securities consist of highly liquid money market funds. Financial assets and liabilities are considered Level 2 when their fair values are determined using inputs that are observable in the market or can be derived principally from or corroborated by observable market data such as pricing for similar securities, recently executed transactions, cash flow models with yield curves, and benchmark securities. In addition, Level 2 financial instruments are valued using comparisons to like-kind financial instruments and models that use readily observable market data as their basis. U.S. government and agency securities, commercial paper, corporate bond and certificates of deposit are valued primarily using market prices of comparable securities, bid/ask quotes, interest rate yields and prepayment spreads and are included in Level 2. During the periods presented, the Company has not changed the manner in which it values liabilities that are measured at estimated fair value using Level 3 inputs. There were no transfers within the hierarchy during the years ended December 31, 2018 and 2017. The following table summarizes, for assets and liabilities recorded at fair value, the respective fair value and the classification by level of input within the fair value hierarchy as described above (in thousands): Quoted Prices Significant Other Significant Total In Observable Inputs Unobservable Inputs December 31, 2018 Carrying (Level 1) (Level 2) (Level 3) Money market funds $ 126 $ 126 $ — $ — U.S. government and agency securities 25,792 — 25,792 — Commercial paper 147,606 — 147,606 — Corporate bonds 27,778 — 27,778 — Certificates of deposit 1,420 — 1,420 — Total cash equivalents and short-term investments $ 202,722 $ 126 $ 202,596 $ — Other noncurrent liability: Financing arrangement $ — $ — $ — $ — December 31, 2017 Assets: Money market funds $ 65 $ 65 $ — $ — U.S. government and agency securities 58,206 — 58,206 — Commercial paper 71,427 — 71,427 — Corporate bonds 38,317 — 38,317 — Certificates of deposit 9,731 — 9,731 — Total cash equivalents and short-term investments $ 177,746 $ 65 $ 177,681 $ — Other noncurrent liability: Financing arrangement $ 157 $ — $ — $ 157 In August 2016, the Company entered into a financing arrangement with the TAP for a total amount of up to $0.3 million (the “TAP financing”), of which $0 and approximately $0.2 million was outstanding as of December 31, 2018 and 2017, respectively (see Note 9). The Company elected the fair value option to account for this financing arrangement. The fair value of the financing arrangement was determined based on the expected value approach and is classified as Level 3 within the fair value hierarchy. The Company determined that the changes in the fair value were immaterial during the years ended December 31, 2018 and 2017. The key unobservable inputs in the valuation model include timing of milestones, probability of achievement of development and commercial milestones, and a discount factor. The TAP financing liability was remeasured to $0 due to the Company’s decision to discontinue the A1AT program during the year ended December 31, 2018. Non-financial assets such as intangible assets, property, plant, and equipment are evaluated for impairment and adjusted to their fair value using Level 3 inputs, only when impairment is recognized. Fair values are considered Level 3 when management makes significant assumptions in developing a discounted cash flow model based upon a number of considerations, including projections of revenues, earnings and a discount rate. |
Significant Agreements
Significant Agreements | 12 Months Ended |
Dec. 31, 2018 | |
Research And Development [Abstract] | |
Significant Agreements | 6. Significant Agreements Editas — In January 2018, the Company entered into an agreement to amend its collaboration, option and license agreement with Editas. The Company originally entered into an agreement with Editas in August 2016 pursuant to which the Company and Editas collaborate on certain studies using AAV vectors in connection with Editas’ genome editing technology and the Company grants to Editas an exclusive option to obtain certain exclusive rights to use the Company’s proprietary vectors in up to five ophthalmic indications. In January 2018, the Company and Editas extended the research collaboration, option and license agreement. In consideration for extending the agreement, Editas made a one-time, non-refundable cash payment of $0.5 million to the Company in February 2018. In June 2018, the Company and Editas entered into a subsequent amendment to the agreement to extend the Research Period and First Option Exercise Date (each as defined in the collaboration, option, and license agreement with Editas, as amended). Under the terms of the agreement, as amended, Editas had until November 2018 to exercise the option with respect to a designated initial indication, which Editas declined to do. With respect to the four other indications, Editas may exercise the option until August 2020, provided that the option will expire in August 2019 if Editas has not exercised one option with respect to any other indication by such date. Upon Editas’ timely exercise of the option with respect to the first additional indication for which Editas timely exercises its option, Editas will pay us a $1.5 million fee. Upon each subsequent exercise of the option, Editas will pay us a $1.0 million fee per indication. If Editas elects to develop a product using certain of our proprietary vectors, we will be eligible to receive up to $15.5 million in development and commercialization milestone payments for such product, and tiered royalties between the mid-single digits and low teens on net sales of such product, subject to certain adjustments. Unless earlier terminated, the agreement will be in effect until the later of the expiration of the option exercise period or the expiration of the royalty term of the last product. At any time after the option is first exercised, Editas may terminate the agreement for convenience in its entirety or on an indication-by-indication or country-by-country basis, upon prior written notice to us. We may also terminate the agreement if Editas challenges our patents relating to our proprietary vectors and does not withdraw such challenge within a defined period of time. In addition, either party may terminate the agreement with written notice upon a bankruptcy of the other party or upon an uncured material breach by the other party. Under Topic 606, the transaction price is $1.5 million related to the $1.0 million non-refundable upfront payment for license and research services at contract inception and the one-time, non-refundable cash payment of $0.5 million made by Editas in February 2018 in consideration for extending the agreement. The arrangement provides for additional payments to the Company when certain development and regulatory milestones are achieved. Because these milestone payments are not within the control of the Company and are not considered probable of being achieved until the events occur, the Company did not include them in the transaction price. The transaction price of $1.5 million was allocated to a single performance obligation, research and development services. During the year ended December 31, 2018, the Company recognized revenue of $1.4 million associated with the Editas collaboration agreement. The Company had no deferred revenue balance as of December 31, 2018. |
Property and Equipment, Net
Property and Equipment, Net | 12 Months Ended |
Dec. 31, 2018 | |
Property Plant And Equipment [Abstract] | |
Property and Equipment, Net | 7. Property and Equipment, Net Property and equipment, net consists of the following: December 31, 2018 2017 (In thousands) Computer equipment and software $ 646 $ 535 Laboratory equipment 5,470 4,956 Furniture and fixtures 678 552 Leasehold improvements 1,602 1,549 Construction in progress 1,612 105 Total property and equipment 10,008 7,697 Less accumulated depreciation and amortization (6,422 ) (4,673 ) Property and equipment, net $ 3,586 $ 3,024 Depreciation and amortization expense related to property and equipment was $1.8 million and $2.1 million for the years ended December 31, 2018 and 2017, respectively. |
Accrued Expenses and Other Curr
Accrued Expenses and Other Current Liabilities | 12 Months Ended |
Dec. 31, 2018 | |
Payables And Accruals [Abstract] | |
Accrued Expenses and Other Current Liabilities | 8. Accrued Expenses and Other Current Liabilities Accrued expenses and other current liabilities consist of the following: December 31, 2018 2017 (In thousands) Compensation expense $ 2,944 $ 2,259 Accrued preclinical costs 289 1,255 Accrued professional fees 2,291 2,295 Accrued clinical and process development costs 1,561 910 Other 1,699 245 Total accrued expenses and other current liabilities $ 8,784 $ 6,964 |
Financing Arrangements
Financing Arrangements | 12 Months Ended |
Dec. 31, 2018 | |
Debt Disclosure [Abstract] | |
Financing Arrangements | 9. Financing Arrangements Banque Publique d’Investissement (“BPI France”) Agreement In August 2015, BPI France granted Annapurna a €0.8 million interest-free conditional advance, of which €0.5 million was outstanding as of December 31, 2016. Payments are scheduled in equal quarterly amounts of €25,000 from September 30, 2017 to June 30, 2022. This payment schedule will be modified if the Company will receive revenue from license or product sales before advances are paid in full. The Company calculated 7% imputed interest expense on these advances that was recorded as a discount at the issuance date. The discount is amortized as an interest expense over the life of the advances. As of December 31, 2018, the total carrying value, which approximates the fair value, of the conditional advance was $0.3 million, of which $0.2 million was recorded within other non-current liabilities and $0.1 million within accrued expenses and other current liabilities in the Company’s consolidated balance sheets. As of December 31, 2017, the total carrying value, which approximates the fair value, of the conditional advance was $0.4 million, of which $0.3 million was recorded within other non-current liabilities and $0.1 million within accrued expenses and other current liabilities in the Company’s consolidated balance sheets. The TAP Agreement In July 2016, the Company entered into a sponsored research agreement with The TAP in which the TAP will fund the Company’s A1AT research activities of up to $0.3 million in cash in three different tranches. The Company may repay up to 4.5 times the received amount if and when certain product approval and sales milestones are achieved. In September 2016, the Company received $0.1 million and issued a warrant to purchase 10,000 shares of its common stock exercisable anytime during five years from the issuance date at an exercise price of $4.33 per share (the “TAP warrant). In December 2017, the Company achieved a milestone which entitled the Company to receive $0.1 million. For the valuation details of the TAP financing, refer to Note 5. The following table presents the TAP financing activity: Years ended December 31, 2018 2017 (In thousands) Balance of TAP financing liability as of the beginning of the year (1) $ 157 $ 74 Funding (2) — 100 Gain on fair value of TAP financing liability ( 3 ) (157 ) (17 ) Balance of TAP financing liability as of the end of the year $ — $ 157 ___________ (1) Recorded within other non-current liabilities in the Company’s consolidated balance sheets. (2) Recorded as a receivable as of December 31, 2017. Payment was received in January 2018. (3) Recorded within other non-current liabilities in the Company’s consolidated balance sheet and other income, net in the Company consolidated statement of operations and comprehensive loss. |
Commitments and Contingencies
Commitments and Contingencies | 12 Months Ended |
Dec. 31, 2018 | |
Commitments And Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 10. Commitments and Contingencies Facility Lease Agreement On June 28, 2018, the Company entered into a lease on a new facility with office, laboratory, and manufacturing space, which will serve as the Company’s new corporate headquarters. The term of the lease is ten years and also provides for two options to extend the lease term for a period of seven years each. The Company is obligated to make lease payments totaling approximately $49.3 million over the initial term of the lease. Under the lease, the Company will receive a tenant improvement allowance for the costs associated with the design, development and construction of tenant improvements for the leased facility. The Company has provided the landlord with a letter of credit in the amount of $1.0 million. The security for the letter of credit of $1.0 million is classified as restricted cash under long term assets on the balance sheet. The Company leases its Menlo Park office building under a non-cancelable lease agreement, which expires on May 8, 2020. The Company may extend this lease for up to four years. The lease agreement provides for an escalation of rent payments each year. The Company records rent expense on a straight-line basis over the term of the lease. As of December 31, 2018, future minimum commitments under the Company’s facility operating leases were as follows: Years ended December 31, Future Commitments (In thousands) 2019 $ 3,344 2020 4,221 2021 4,683 2022 4,846 2023 5,016 Thereafter 28,837 Total minimum lease payments $ 50,947 Rent expense recognized under the operating lease, including additional rent charges for utilities, parking, maintenance, and real estate taxes was $3.3 million and $1.8 million for the years ended December 31, 2018 and 2017, respectively. Contractual Obligations As of December 31, 2018, the Company had a contractual obligation of approximately $0.7 million for a contract manufacturing with a vendor Collaborations and License Agreements The Company is a party to various agreements, principally relating to licensed technology that requires future payments relating to milestones or royalties on future sales of specified products. Through December 31, 2018, none of the goals had been achieved under the license agreements and no milestones were accrued or payable. Because the achievement of these milestones is not fixed and determinable, such commitments have not been included on the Company’s consolidated balance sheets. Aggregate annual maintenance fees payments were approximately $0.1 million and $0.5 million for each of the years ended December 31, 2018, and 2017. The Company was a party to a master services agreement (“MSA”) with Cornell University (“Cornell”) originally established in August 2014 and amended in December 2015. Under the MSA, Cornell provided assistance in regulatory affairs, overall project management, and parameter development. The MSA, as amended, provided for the Company to pay Cornell $13.3 million ratably over four years for these services as services were performed. In December 2016, the Company informed Cornell that the Company decided to terminate the MSA for material breach, effective January 6, 2017. Subsequently, Cornell informed the Company that it disputes the validity of the Company’s termination of the MSA. Although the Company intends to defend the validity of the termination of the MSA, the Company recorded $2.0 million of estimated costs associated with the termination of the MSA during the year-ended December 31, 2017. This MSA included services relating to gene therapy programs directed to A1AT deficiency, HAE and severe allergy. The Company’s license agreements with Cornell remained in effect despite the termination of the MSA. Guarantees and Indemnifications In the normal course of business, the Company enters into contracts and agreements that contain a variety of representations and warranties and provide for indemnification for certain liabilities. The exposure under these agreements is unknown because it involves claims that may be made against the Company in the future but have not yet been made. To date, the Company has not paid any claims or been required to defend any action related to its indemnification obligations. However, the Company may record charges in the future as a result of these indemnification obligations. The Company also has indemnification obligations to its directors and executive officers for specified events or occurrences, subject to some limits, while they are serving at the Company’s request in such capacities. There have been no claims to date and the Company believes the fair value of these indemnification agreements is minimal. Accordingly, the Company has not recorded any liabilities for these agreements as of December 31, 2018 and 2017. Legal Proceedings From time to time, the Company may become involved in litigation and other legal actions. The Company estimates the range of liability related to any pending litigation where the amount and range of loss can be estimated. The Company records its best estimate of a loss when the loss is considered probable. Where a liability is probable and there is a range of estimated loss with no best estimate in the range, the Company records a charge equal to at least the minimum estimated liability for a loss contingency when both of the following conditions are met: (i) information available prior to issuance of the financial statements indicates that it is probable that a liability had been incurred at the date of the financial statements and (ii) the range of loss can be reasonably estimated. In July 2015, three securities class action lawsuits were filed against the Company and certain of its officers in the United States District Court for the Northern District of California (“U.S. District Court”), each on behalf of a purported class of persons and entities who purchased or otherwise acquired the Company’s publicly traded securities between July 31, 2014 and June 15, 2015. The lawsuits asserted claims under the Securities Exchange Act of 1934, as amended (the “Exchange Act”) and Securities Act of 1933, as amended (the “Securities Act”) and alleged that the defendants who are no longer with the Company made materially false and misleading statements and omitted allegedly material information related to, among other things, the Phase 2a clinical trial for AVA-101, a product candidate which is no longer being developed, and the prospects of AVA-101. The complaints sought unspecified damages, attorneys’ fees and other costs. In December 2015, a putative securities class action lawsuit was filed against the Company, the Company’s board of directors, underwriters of the Company’s January 13, 2015, follow-on public stock offering, and two of the Company’s institutional stockholders, in the Superior Court of the State of California for the County of San Mateo (“San Mateo Superior Court”). The complaint alleged that, in connection with the Company’s follow-on stock offering, the same defendants violated the Securities Act by allegedly making materially false and misleading statements and by allegedly omitting material information related to the Phase 2a clinical trial for AVA- 101 and the prospects of AVA-101. The complaint sought unspecified compensatory and rescissory damages, attorneys’ fees and other costs. The plaintiff has dismissed the two institutional stockholder defendants. In March 2017, the Company reached an agreement to settle the asserted actions. The proposed aggregate amount of the settlement is $13.0 million, of which $1.0 million was contributed by the Company to cover its indemnification obligations to the underwriters, and the remainder was contributed by the Company’s insurers. The Company and the defendants have denied and continue to deny each and all of the claims alleged in the actions, and the settlement does not assign or reflect any admission of fault, wrongdoing or liability as to any defendant. Notice of the settlement was provided to shareholders in the fall of 2017, and no shareholder objected to the settlement. In January 2018, the San Mateo Superior Court entered a judgment and order finally approving the settlement. On February 5, 2018, the U.S. District Court entered an order dismissing the consolidated federal action with prejudice. The Company recorded $1.0 million as general and administrative expense during the three months ended March 31, 2017, when the amount and time of settlement became estimable and probable. |
Common Stock Warrants
Common Stock Warrants | 12 Months Ended |
Dec. 31, 2018 | |
Warrants And Rights Note Disclosure [Abstract] | |
Common Stock Warrants | 11. The Lions Eye Institute In connection with the Company’s research and collaboration agreement, as amended, with LEI (the “LEI Agreement”), the Company agreed to issue a warrant to purchase a certain number of the Company’s common stock upon the achievement of each milestones as set forth in the LEI Agreement. During the year ended December 31, 2015, the Company issued a warrant to purchase 40,000 shares of its common stock with an exercise price of $10.51 per share to LEI. This common stock warrant is exercisable immediately, and expires on October 15, 2020. The estimated fair value of this warrant was approximately $0.2 million and was recorded within research and development expenses in the Company’s consolidated statement of operations and comprehensive loss and additional paid-in capital in the Company’s balance sheet for the year ended December 31, 2015. The fair value of the warrant was calculated using the Black-Scholes valuation model, and was based on the closing price of common stock on the issuance date of $8.35 per share, contractual term of the warrant of 5 years, a risk-free interest rate of 1.34%, an expected volatility of 75% and a 0% expected dividend yield. Additionally, in September 2017, the Company issued a warrant to purchase 40,000 shares of its common stock with an exercise price of $3.65 per share to LEI. This common stock warrant is exercisable immediately, and expires on September 29, 2022. The estimated fair value of this warrant was approximately $0.1 million and the fair value of this warrant was recorded as research and development expenses in the Company’s consolidated statement of operations and comprehensive loss and additional paid-in capital in the Company’s consolidated balance sheet for the year ended December 31, 2017. The fair value of the warrant was calculated using the Black-Scholes valuation model, and was based on the closing price of common stock on the issuance date of $3.65 per share, contractual term of the warrant of 5 years, a risk-free interest rate of 1.89%, an expected volatility of 91% and a 0% expected dividend yield. TAP Warrant. In July 2016, in connection with the TAP financing agreement (see Note 9), the Company issued a warrant to purchase 10,000 shares of its common stock exercisable anytime during five years from the issuance date at an exercise price of $4.33 per share. The estimated fair value of this warrant was $26,000 at the issuance date using the Black-Scholes valuation model with the following assumptions: exercise price of $4.33 per share, expected term of the warrant of 5 years, a risk-free interest rate of 1.07%, an expected volatility of 72% and a 0% expected dividend. The fair value of TAP warrant was recorded to other non-current liabilities and additional paid-in capital in the Company’s consolidated balance sheet for the year ended December 31, 2016. |
Stock Plans
Stock Plans | 12 Months Ended |
Dec. 31, 2018 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Stock Plans | 12. Stock Plans On December 26, 2006, the Company adopted the 2006 Equity Incentive Plan, which was amended by the board of directors on November 15, 2012 (the “2006 Plan”). The 2006 Plan allowed for the granting of incentive stock options (“ISOs”) and non-qualified stock options (“NSOs”) to the employees, members of the board of directors and consultants of the Company. ISOs were granted only to the Company’s employees, including officers and directors who are also employees. NSOs were granted to the employees and consultants. In July 2014, the Company’s board of directors and its stockholders approved the establishment of the 2014 Equity Incentive Award Plan (the “2014 Plan”). Options may no longer be issued under the 2006 Plan after July 30, 2014. In addition, the 2014 Plan provides for annual increases in the number of shares available for issuance thereunder on the first business day of each fiscal year, beginning with the year ended December 31, 2015, equal to four percent (4%) of the number of shares of the Company’s common stock outstanding as of such date or a lesser number of shares as determined by the Company’s board of directors. In October 2017, the Company adopted the 2017 Inducement Plan (the “Inducement Plan”). The Company reserved 600,000 shares for issuance pursuant to stock options and RSUs under the Inducement Plan. The only persons eligible to receive grants of stock options and RSUs under Inducement Plan are individuals who satisfy the standards for inducement grants under Nasdaq guidance that is, generally, a person not previously an employee or director of Adverum, or following a bona fide period of non-employment, as an inducement material to the individual's entering into employment with Adverum. The 2006 Plan, 2014 Plan and Inducement Plan are referred to collectively as the Plans. As of December 31, 2018, a total of 19,353,469 shares of common stock were reserved for issuance and 2,695,347 shares were available for future grants under the Plans. Stock Options Stock options under the 2014 Plan and the Inducement Plan may be granted for periods of up to 10 years and at prices no less than 100% of the estimated fair value of the shares on the date of grant as determined by the board of directors, provided, however, that the exercise price of an ISO and NSO granted to a 10% stockholder may not be less than 110% of the estimated fair value of the shares on the date of grant. Stock options granted to employees and non-employees generally vest ratably over four years. The following table summarizes stock option activity under the Company’s stock plans and related information: (In thousands, except exercise prices and years) Options Outstanding Weighted- Average Exercise Price Weighted- Average Remaining Contract Life (in years) Aggregate Intrinsic Value (a) Balance at December 31, 2016 7,449 $ 4.46 8.4 $ 11,837 Granted 1,989 2.85 Exercised (1,808 ) 0.20 Cancelled/forfeited (935 ) 8.87 Balance at December 31, 2017 6,695 $ 4.51 7.4 $ 9,539 Granted 2,049 5.94 Exercised (1,606 ) 0.43 Cancelled/forfeited (691 ) 5.90 Balance at December 31, 2018 6,447 $ 5.83 7.6 $ 3,594 Vested and expected to vest as of December 31, 2018 6,447 $ 5.83 7.6 $ 3,594 Exercisable at December 31, 2018 3,321 $ 6.63 6.7 $ 2,630 (a) In June 2017, the Company granted 150,000 stock options outside the Plans to its certain executive officers. The total intrinsic value of stock options exercised during the years ended December 31, 2018 and 2017 were $8.2 million and $4.8 million, respectively. Stock Options Granted to Employees. The fair value of each stock option issued to employees was estimated at the date of grant using the Black-Scholes valuation model with the following weighted-average assumptions: Options Employee Stock Purchase Plan Years ended December 31, Years ended December 31, 2018 2017 2018 2017 Expected volatility 80% 82% 78% 52% Expected term (in years) 6.0 6.0 0.5 0.5 Expected dividend yield — — — — Risk-free interest rate 2.8% 1.9% 2.3% 1.3% The weighted-average fair values of options granted during the years ended December 31, 2018 and 2017, were $4.15 and $1.99, respectively. As of December 31, 2018, there was $6.5 million of unrecognized stock-based compensation expense related to employee stock options that is expected to be recognized over a weighted-average period of 2.2 years. Stock Options Granted to Non-Employees. Stock-based compensation expense related to stock options granted to non-employees is recognized as the stock options are earned. The Company believes that the estimated fair value of the stock options is more readily measurable than the fair value of the services rendered. The following table presents the weighted-average assumptions used in the Black-Scholes valuation model to estimate the fair value of non-employee stock options: Years ended December 31, 2018 2017 Expected volatility 77% 84% Expected term (in years) 7.7 8.8 Expected dividend yield — — Risk-free interest rate 2.7% 2.3% As of December 31, 2018, unrecognized stock-based compensation expense related to unvested non-employees stock options was approximately $0.1 million, which is expected to be recognized over a weighted-average period of 1.1 years, based on the estimated fair value at December 31, 2018. RSUs RSUs are share awards that entitle the holder to receive freely tradable shares of the Company’s common stock upon vesting. The fair value of RSUs is based upon the closing sales price of the Company’s common stock on the grant date. RSUs granted to employees generally vest over a two to four-year period. The following table summarizes the RSU activity under the Company’s stock plans and related information: (In thousands, except grant date fair value and years) Number of Units (in thousands) Weighted- Average Grant Date Fair Value (in dollars) Weighted- Average Remaining Contractual Term (in years) Balance at December 31, 2016 1,049 $ 5.47 1.7 Granted 2,543 2.81 Vested and released (307 ) 6.43 Forfeited (770 ) 3.58 Balance at December 31, 2017 2,515 $ 3.24 1.6 Granted 1,381 5.94 Vested and released (774 ) (3.46 ) Forfeited (725 ) (4.28 ) Balance at December 31, 2018 2,397 $ 9.23 4.8 The weighted-average grant-date fair value of RSUs granted during the years ended December 31, 2018 and 2017, were $5.94 and $2.81, respectively. During the years ended December 31, 2018 and 2017, total fair value of RSUs vested was $2.7 million and $2.0 million, respectively. The number of RSUs vested includes shares of common stock that the Company withheld on behalf of employees to satisfy the minimum statutory tax withholding requirements. As of December 31, 2018, there was $7.4 million of unrecognized compensation cost related to unvested RSUs that is expected to be recognized over a weighted-average period of 2.5 years. ESPP In July 2014, the Company approved the establishment of the 2014 Employee Stock Purchase Plan (the “ESPP”). The Company reserved 208,833 shares of its common stock for issuance and provided for annual increases in the number of shares available for issuance on the first business day of each fiscal year, beginning in 2015, equal to the lesser of one percent (1%) of the number of shares of the Company’s common stock outstanding as of such date or a number of shares as determined by the Company’s board of directors. During the years ended December 31, 2018, 120,475 shares were issued under the ESPP. As of December 31, 2018, a total of 1,331,773 shares of common stock were available for future issuance under the ESPP. As of December 31, 2018, unrecognized compensation cost related to the ESPP was immaterial. Stock-Based Compensation Recognized in the Consolidated Statement of Operations and Comprehensive Loss The following table presents, by operating expense, the Company’s stock-based compensation expense: Years ended December 31, 2018 2017 (In thousands) Research and development $ 4,820 $ 5,253 General and administrative 8,612 3,470 Total share-based compensation expense $ 13,432 $ 8,723 During the year ended December 31, 2018, the Company recorded approximately $4.1 million of stock-based compensation expense as a result of the modification of the vesting and exercisability of stock awards associated with the departure of two of its executives. |
401(k) Savings Plan
401(k) Savings Plan | 12 Months Ended |
Dec. 31, 2018 | |
Compensation And Retirement Disclosure [Abstract] | |
401(k) Savings Plan | 13. 401(k) Savings Plan The Company established a defined-contribution savings plan under Section 401(k) of the Code. The 401(k) Plan covers all employees who meet defined minimum age and service requirements, and allows participants to defer a portion of their annual compensation on a pretax basis. The amount of contributions that the Company made to the 401(k) Plan during the year ended December 31, 2018 and 2017 was $0.4 million and $0.3 million, respectively. |
Income Taxes
Income Taxes | 12 Months Ended |
Dec. 31, 2018 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | 14. Income Taxes The Company recorded $1.3 million income tax benefit related to the change in the deferred tax liabilities balance due to intangible assets impairment recognized in the third quarter of 2018 and no income tax benefit or expense were recorded for the year ended December 31, 2017. The following table presents domestic and foreign components of loss before provision for income taxes: Years ended December 31, 2018 2017 (In thousands) U.S. $ (45,024 ) $ (36,923 ) Foreign (28,853 ) (19,224 ) Loss before income taxes $ (73,877 ) $ (56,147 ) A reconciliation of income tax expense computed at the statutory federal income tax rate of 21% and 34% during the year ended December 31, 2018 and 2017, respectively, to income taxes as reflected in the financial statements is as follows: Years ended December 31, 2018 2017 (In thousands) Federal income tax expense at statutory rate $ (15,514 ) $ (19,090 ) Non-deductible foreign research expenses — 19 Stock compensation (1,512 ) (76 ) Non-deductible expenses 75 61 Other 41 — Research and development tax credits (1,215 ) (507 ) Change in valuation allowance 11,219 7,935 Foreign rate differential (586 ) 1,495 Rate change — 10,163 Change in uncertain tax positions 6,242 — Total tax benefit $ (1,250 ) $ — Deferred income taxes reflect the net tax effects of temporary differences between the carrying amounts of assets and liabilities for financial reporting purposes and the amounts used for income tax purposes. The following table presents significant components of the Company’s deferred tax assets: As of December 31, 2018 2017 (In thousands) Deferred tax assets: Net operating loss carryforwards $ 37,247 $ 27,142 Accruals, reserve and other 827 2,586 Stock-based compensation 4,549 3,623 Tax credit carryforwards 4,161 1,774 Property and equipment 310 274 Intangibles 38 64 Other 122 — Total deferred tax assets before valuation allowance 47,254 35,463 Valuation allowance (47,254 ) (35,463 ) Total deferred tax assets — — Deferred tax liabilities: IPR&D — (1,250 ) Total deferred tax liabilities $ — $ (1,250 ) Net deferred tax assets $ — $ — On December 22, 2017, the U.S. government enacted comprehensive tax legislation, commonly known as the Tax Cuts and Jobs Act of 2017 (the “Act”), which significantly reforms the Internal Revenue Code of 1986, as amended. The Act contains broad and complex changes to corporate taxation, including in part reduction of the U.S. federal corporate tax rate from 35% to 21%, requires companies to pay a one-time transition tax on earnings of certain foreign subsidiaries that were previously considered permanently reinvested, and creates new taxes on certain foreign sourced earnings. As of December 31, 2017, the Company was able to determine a reasonable estimate, namely the one-time transition tax and the remeasurement of deferred tax at the new tax rate. The Company did not recognize any provisional tax expense due to its significant operating losses. The effect on the Company’s deferred tax balance due to the change of net tax rate was fully offset by its valuation allowance. On December 22, 2017 the SEC staff issued SAB 118, which allowed the Company to record provisional amounts during a measurement period not to extend beyond one year of the enactment date. Staff Accounting Bulletin No. 118 ("SAB 118") was issued to address the application of U.S. GAAP in situations when a registrant does not have the necessary information available, prepared, or analyzed (including computations) in reasonable detail to complete the accounting for certain income tax effects of the Act. In accordance with SAB 118, the Company has completed its analysis during the fourth quarter of 2018 noting no material adjustments from the provisional amounts recorded in the prior year. In addition, the guidance indicates that either accounting for deferred taxes related to GILTI or to treat any taxes on future GILTI inclusions as period cost are both acceptable methods subject to an accounting policy election. The Company has elected to treat future GILTI inclusions if any as period costs. The Company has evaluated the positive and negative evidence bearing upon the realizability of its deferred tax assets. Based on the Company’s history of operating losses, the Company has concluded that it is more likely than not that the benefit of its deferred tax assets will not be realized. Accordingly, the Company has provided a full valuation allowance for deferred tax assets as of December 31, 2018 and 2017. The valuation allowance increased approximately $11.8 million and $12.0 million during the years ended December 31, 2018 and 2017, respectively. As of December 31, 2018, the Company had U.S. federal net operating losses (“NOLs”) carryforwards of approximately $100.0 million to offset any future federal income. Approximately $57.0 million of NOLs expire at various years beginning with 2036. As of December 31, 2018, the Company also had U.S. state NOL carryforwards of approximately $61.1 million to offset any future state income. U.S. state NOLs expire at various years beginning with 2036. At December 31, 2018, the Company also had approximately $65.0 million of foreign net operating loss carryforwards which may be available to offset future foreign income; these carryforwards do not expire. As of December 31, 2018, the Company had federal research and development tax credit carryforwards of approximately $3.2 million available to reduce future tax liabilities which expire at various years beginning with 2036. As of December 31, 2018, the Company had state credit carryforwards of approximately $3.3 million available to reduce future tax liabilities which do not expire. Under Section 382 of the Internal Revenue Code of 1986, as amended (Code), the Company’s ability to utilize NOL carryforwards or other tax attributes, such as research tax credits, in any taxable year may be limited if the Company experiences an “ownership change.” Generally, a Section 382 ownership change occurs if there is a cumulative increase of more than 50 percentage points in the stock ownership of one or more stockholders or groups of stockholders who owns at least 5% of a corporation’s stock within a specified testing period. Similar rules may apply under state tax laws. The Company believes that it has experienced ownership changes under Section 382, which will result in limitations in the Company’s ability to utilize net operating losses and credits. As a result, the amount of the NOLs and research and development credit carryforwards presented in the Company’s consolidated financial statements are limited and will expire unutilized, and the Company removed a significant amount of NOLs and credits from its deferred taxes. The Company files income tax returns in the U.S. Federal, state, and foreign jurisdictions. The federal, state and foreign income tax returns are open under the statute of limitations subject to tax examinations for the tax years ended December 31, 2013 through December 31, 2018. 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, state or foreign tax authorities to the extent utilized in a future period. The Company has total unrecognized tax benefits as of December 31, 2018 and 2017 of approximately $8.8 million and $2.7 million, respectively. No amount of the unrecognized tax benefits, if recognized, would reduce the Company’s annual effective tax rate because the benefits are in the form of deferred tax assets for which a full valuation allowance has been recorded. The Company does not anticipate a significant change to its unrecognized tax benefits over the next twelve months. A reconciliation of the unrecognized tax benefits is as follows: Years ended December 31, 2018 2017 (In thousands) Unrecognized tax benefits as of the beginning of the year $ 2,745 $ 2,157 Increase (decrease) related to prior year tax provisions 1,941 — Increase related to current year tax provisions 4,119 588 Unrecognized tax benefits as of the end of the year $ 8,805 $ 2,745 The Company recognizes interest and penalties related to uncertain tax positions in income tax expense. As of December 31, 2018, and 2017, the Company had no accrued interest or penalties related to uncertain tax positions and no amounts have been recognized in the Company’s consolidated statements of operations and comprehensive loss. There are no ongoing examinations by taxing authorities at this time. |
Net Loss per Share
Net Loss per Share | 12 Months Ended |
Dec. 31, 2018 | |
Earnings Per Share [Abstract] | |
Net Loss per Share | 15. Net Loss per Share The following common stock equivalents outstanding at the end of the periods presented were excluded from the calculation of diluted net loss per share for the periods indicated because including them would have had an anti-dilutive effect: As of December 31, 2018 2017 (In thousands) Stock options 6,447 6,695 Restricted stock units 2,397 2,515 ESPP 62 71 Warrants to purchase common stock 90 90 8,996 9,371 |
Selected Quarterly Financial In
Selected Quarterly Financial Information | 12 Months Ended |
Dec. 31, 2018 | |
Quarterly Financial Information Disclosure [Abstract] | |
Selected Quarterly Financial Information | 16. Selected Quarterly Financial Information (Unaudited) The Company’s quarterly consolidated results of operations are shown below: Quarterly Results of Operations Three Months Ended March 31, 2018 June 30, 2018 September 30, 2018 December 31, 2018 (In thousands, except per share amounts) Revenue $ 216 $ 493 $ 833 $ 70 Total operating expenses (1) (18,162 ) (20,396 ) (24,306 ) (16,829 ) Net loss (17,200 ) (18,810 ) (20,958 ) (15,659 ) Basic and diluted net loss per share $ (0.30 ) $ (0.30 ) $ (0.34 ) $ (0.25 ) Quarterly Results of Operations Three Months Ended March 31, 2017 June 30, 2017 September 30, 2017 December 31, 2017 (In thousands, except per share amounts) Revenue $ 462 $ 463 $ 463 $ 461 Total operating expenses (1) (17,050 ) (12,556 ) (15,034 ) (16,056 ) Net loss (16,099 ) (11,430 ) (13,829 ) (14,789 ) Basic and diluted net loss per share (0.38 ) (0.27 ) (0.32 ) (0.32 ) (1) During the year ended December 31, 2017, the Company recorded a total of $2.0 million of estimated costs associated with the termination of MSA with Cornell University (see Note 10). During the three months ended March 31, 2017, the Company recorded a total of $2.3 million of these estimated costs, which was subsequently adjusted by $0.3 million during the three months ended June 30, 2017. Basic and diluted net loss per share is computed independently for each of the quarters presented. Therefore, the sum of quarterly basic and diluted per share amounts may not equal annual basic and diluted net loss per share amounts. |
Summary of significant accoun_2
Summary of significant accounting policies (Policies) | 12 Months Ended |
Dec. 31, 2018 | |
Accounting Policies [Abstract] | |
Basis of Presentation and Principles of Consolidation | Basis of Presentation and Principles of Consolidation — The Company’s consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States of America (“U.S. GAAP”) and pursuant to the rules and regulations of the Securities and Exchange Commission (“SEC”). The consolidated financial statements include the accounts of the Company and its wholly-owned subsidiaries. All inter-company transactions and balances have been eliminated in consolidation. |
Use of Estimates | Use of Estimates —The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets, liabilities, and the disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of income and expenses during the reporting period. The Company bases estimates and assumptions on historical experience when available and on various factors that it believes to be reasonable under the circumstances. On an ongoing basis, the Company evaluates its estimates and assumptions, including those related to research and development expense accruals, stock-based compensation expense, income taxes, intangible asset, fair values of financial instruments and fair value of common stock warrants. The Company’s actual results may differ from these estimates under different assumptions or conditions. There have been no significant changes from the Company’s original estimates in any periods presented. |
Foreign Currency Translation | Foreign Currency Translation —The Company’s consolidated financial statements are prepared in U.S. dollars. The Company’s foreign subsidiaries use the Euro and Australian dollar as their functional currencies and maintain their records in their local currencies, except its Ireland subsidiary that uses the U.S. dollar as its functional currency. Assets and liabilities are re-measured at exchange rates in effect at the end of the reporting period for the Company’s French and Australian subsidiaries, and at historical exchange rates for its Irish subsidiary. Equity is measured at historical rates and income and expenses are re-measured at average exchange rates for the reporting period. The resulting foreign currency translation adjustment is recorded in accumulated other comprehensive loss in the consolidated balance sheet. Transactions denominated in foreign currency are translated at exchange rates at the date of transaction with foreign currency gains (losses) recorded in other income (expense), net in the consolidated statements of operations and comprehensive loss. |
Cash and Cash Equivalents | Cash and Cash Equivalents —The Company considers all highly liquid investments purchased with original maturities of three months or less at the date of purchase to be cash equivalents. Cash and cash equivalents include cash held in banks, money market accounts and highly liquid debt securities. Cash equivalents are stated at fair value. |
Restricted Cash | Restricted Cash —Restricted cash primarily consists of cash collateral to letter of credit provided to the landlord in relation to a lease agreement (see Note 10). |
Short-Term Investments | Short-Term Investments —All short-term investments, which consist of debt securities and certificates of deposit, have been classified as “available for sale” and are carried at fair value. Unrealized gains and losses, net of any related tax effects, are excluded from earnings and are included in other comprehensive loss and reported as a separate component of stockholders’ equity until realized. Realized gains and losses and declines in value judged to be other than temporary, if any, on available-for-sale securities are included in other income (expense), net in the Company’s consolidated statements of operations and comprehensive loss. The cost of securities sold is based on the specific-identification method. The amortized cost of securities is adjusted for amortization of premiums and accretion of discounts to maturity. Interest on short-term investments is included in other income, net in the Company’s consolidated statements of operations and comprehensive loss. In accordance with the Company’s investment policy, management invests to diversify credit risk and only invests in securities with high credit quality, including U.S. government securities. The Company periodically evaluates whether declines in the fair value of its investments below their cost are other than temporary. The evaluation includes consideration of the cause of the impairment, including the creditworthiness of the security issuers, the number of securities in an unrealized loss position, the severity and duration of the unrealized losses, whether the Company has the intent to sell the securities, and whether it is more likely than not that the Company will be required to sell the securities before the recovery of their amortized cost basis. If the Company determines that the decline in fair value of an investment is below its accounting basis and this decline is other than temporary, the Company would reduce the carrying value of the security it holds and records a loss for the amount of such decline. The Company has not recorded any realized losses or declines in value judged to be other than temporary on its investments. |
Segment Reporting | Segment Reporting —The Company operates and manages its business as one reporting and operating segment, which is the business of developing and commercializing gene therapeutics. The Company’s chief executive officer, who is the chief operating decision maker, reviews financial information on an aggregate basis for purposes of allocating resources and evaluating financial performance. |
Concentrations of Credit Risk and Other Uncertainties | Concentrations of Credit Risk and Other Uncertainties —Financial instruments that potentially subject the Company to significant concentrations of credit risk consists primarily of cash, cash equivalents and short-term investments. Risks associated with cash, cash equivalents, short-term investments are mitigated by the Company’s investment policy, which limits the Company’s investing to only those marketable securities rated at least A-1/P-1 Short Term Rating and A/A2 Long Term Rating, as determined by independent credit rating agencies. Management believes that the Company is not exposed to significant credit risk. The Company is subject to certain risks and uncertainties, including, but not limited to changes in any of the following areas that the Company believes could have a material adverse effect on future financial position or results of operations: ability to obtain future financing; regulatory approval and market acceptance of, and reimbursement for, the Company’s product candidates; performance of third-party clinical research organizations and manufacturers; development of sales channels; protection of the intellectual property; litigation or claims against the Company based on intellectual property, patent, product, regulatory or other factors; and the Company’s ability to attract and retain employees necessary to support the growth. |
Property and Equipment | Property and Equipment —Property and equipment are recorded at cost, net of accumulated depreciation and amortization. Depreciation is recorded using the straight-line method over the estimated useful lives of the assets, generally three to five years. Leasehold improvements are capitalized and amortized over the shorter period of their expected lives or the lease term. Major replacements and improvements are capitalized, while general repairs and maintenance are expensed as incurred. |
Valuation of Long-Lived Assets and Purchased Intangible Assets | Valuation of Long-Lived Assets and Purchased Intangible Assets —The Company evaluates the carrying value of amortizable long‑lived assets, whenever events, or changes in business circumstances or the planned use of long‑lived assets indicate that their carrying amounts may not be fully recoverable or that their useful lives are no longer appropriate. If these facts and circumstances exist, the Company assesses for recovery by comparing the carrying values of long‑lived assets with their future undiscounted net cash flows. If the comparison indicates that impairment exists, long‑lived assets are written down to their respective fair values based on discounted cash flows. Significant management judgment is required in the forecasting of future operating results that is used in the preparation of expected undiscounted cash flows. If management’s assumptions about future operating results were to change as a result of events or circumstances, the Company may be required to record an impairment loss on these assets. There were no impairment indicators noted for the Company’s amortizable long-lived assets, fixed assets, in the year ended December 31, 2017. The Company also evaluates the carrying value of intangible asset (not subject to amortization) related to in‑process research and development (“IPR&D”) asset, which is considered to be indefinite‑lived until the completion or abandonment of the associated research and development efforts. Accordingly, amortization of the IPR&D assets will not occur until the product reaches commercialization. During the period the intangible asset is considered indefinite‑lived, it will be tested for impairment on an annual basis, as well as between annual tests if the Company becomes aware of any events occurring or changes in circumstances that would indicate that the fair value of the IPR&D asset is less than its carrying amount. If and when development is complete, which generally occurs when regulatory approval to market the product is obtained, the associated IPR&D asset would be deemed definite‑lived and would then be amortized based on its estimated useful life at that point in time based on respective patent term. If a potential impairment exists, an impairment loss is measured as the excess of the asset’s carrying value over its fair value. During the year ended December 31, 2018, the Company recorded an impairment charge of $5.0 million related to its intangible assets. (see Note 3). |
Financial Liabilities | Financial Liabilities — During the year ended December 31, 2016, the Company entered into a sponsored research agreement with The Alpha-1 Project, Inc. (the “TAP”) with an embedded derivative, the Company elected to account for this financial liability at fair value and recorded as other non-current liabilities in its consolidated balance sheets (see Note 5). The change in fair value is recorded within other income, net in the Company’s consolidated statement of operations and comprehensive loss. |
Revenue Recognition | Revenue Recognition —The Company has primarily generated revenue through license, research and collaboration arrangements with its strategic partners. Effective January 1, 2018, the Company adopted the new revenue standards under Topic 606 using the modified retrospective approach. Results for reporting periods beginning after January 1, 2018 are presented under Topic 606, while prior period results are not adjusted and continue to be reported in accordance with the revenue standards under Topic 605. Under Topic 606, the Company recognizes revenue when its customer obtains control of promised goods or services, in an amount that reflects the consideration which the Company expects to receive in exchange for those goods or services. To determine revenue recognition for arrangements that the Company determines are within the scope of ASC 606, the Company performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) the Company satisfies a performance obligation. At contract inception, once the contract is determined to be within the scope of ASC 606, the Company assesses the goods or services promised within each contract and determines those that are performance obligations. The Company then recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) the performance obligation is satisfied. The Company’s license and collaboration arrangements with Regeneron Pharmaceuticals, Inc. (“Regeneron”), Editas Medicine, Inc. (“Editas”), and GenSight Biologics (“GenSight”) are within the scope of Topic 606. Upon the adoption of Topic 606, the Company recorded a net decrease of $6.1 million to its deferred revenue and opening accumulated deficit as of January 1, 2018 for the cumulative effect of the adoption. The effect of the adoption is summarized for the Company’s license and collaboration agreements as follows: Collaboration and License Revenue Collaboration Agreement with Regeneron — Under Topic 606, the transaction price at contract inception was determined to be $8.0 million, which was related to the non-refundable upfront payment for license and research services. The arrangement also provides for additional payments to the Company when certain development and regulatory milestones are achieved. Because these milestone payments are not within the control of the Company and are not considered probable of being achieved until the events occur, the Company did not include them in the transaction price at contract inception. The transaction price of $8.0 million at contract inception was allocated to two performance obligations. The Company’s deferred revenue associated with its Regeneron collaboration agreement as of December 31, 2017 under Topic 605 was $6.5 million. As a result of adopting Topic 606, the Company recorded a $6.5 million reduction to its deferred revenue and opening accumulated deficit during the three months ended March 31, 2018 as the performance obligations associated with the Regeneron deferred revenue were satisfied as of January 1, 2018. There was no outstanding deferred revenue associated with Regeneron as of March 31, 2018 or December 31, 2018. Collaboration Agreement with Editas — Under Topic 606, the transaction price at contract inception was determined to be $1.0 million, which was related to the non-refundable upfront payment for license and research services. The arrangement provides for additional payments to the Company when certain development and regulatory milestones are achieved. Because these milestone payments are not within the control of the Company and are not considered probable of being achieved until the events occur, the Company did not include them in the transaction price at contract inception. The transaction price of $1.0 million at contract inception was allocated to a single performance obligation. The Company’s deferred revenue associated with its Editas collaboration agreement as of December 31, 2017 under Topic 605 was $0.5 million. As a result of adopting Topic 606, the Company recorded an increase of $0.4 million to its deferred revenue and opening accumulated deficit during the three months ended March 31, 2018 due to differences in the timing of recognition under Topic 606. During the year ended December 31, 2018, the Company recognized revenue of $1.4 million associated with the Editas collaboration agreement. There was no outstanding deferred revenue associated with Editas as of December 31, 2018. License Agreement with GenSight — On February 2014, the Company entered into an agreement with GenSight, where the Company granted GenSight a non-exclusive license to its proprietary AAV.7m8 vector. Under the agreement, the Company is eligible to receive development, regulatory and commercial milestones. Also, the Company is eligible to receive low to mid-single digit royalties on sales of GenSight’s licensed products. During the year ended December 31, 2018, GenSight achieved a clinical development milestone pursuant to the agreement. This milestone was previously constrained under Topic 606. The Company earned a $0.2 million milestone payment, which was recognized as revenue in the consolidated statement of operations and comprehensive loss for the year ended December 31, 2018. Under Topic 605, the Company’s revenue for the year ended December 31, 2018 would have been $2.2 million. |
Research and Development Expenses | Research and Development Expenses —Research and development expenses are charged to expense as incurred. Research and development expenses include primarily personnel-related costs, stock-based compensation expense, laboratory supplies, consulting costs, external contract research and development expenses, including expenses incurred under agreements with CROs, the cost of acquiring, developing and manufacturing clinical trial materials, and overhead expenses, such as rent, equipment depreciation, insurance and utilities. Advance payments for goods or services for future research and development activities are deferred and expensed as the goods are delivered or the related services are performed. The Company estimates preclinical studies and clinical trial expenses based on the services performed pursuant to contracts with research institutions and clinical research organizations that conduct and manage preclinical studies and clinical trials on the Company’s behalf. In accruing service fees, the Company estimates the time period over which services will be performed and the level of effort to be expended in each period. These estimates are based on communications with the third-party service providers and the Company’s estimates of accrued expenses and on information available at each balance sheet date. If the actual timing of the performance of services or the level of effort varies from the estimate, the Company will adjust the accrual accordingly. There have been no significant changes from the Company’s original estimates in any of the periods presented. |
Fair Value Measurements | Fair Value Measurements —Fair value is defined as the price that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants at the measurement date. As such, fair value is a market-based measurement that should be determined based on assumptions that market participants would use in pricing an asset or liability. The carrying amounts of the Company’s financial instruments, including cash equivalents, prepaid and other current assets, accounts payable, accrued expenses and other current liabilities approximate their fair values due to their short-term maturities. Refer to Note 5 for the methodologies and assumptions used in valuing financial instruments. |
Stock-Based Compensation Expense | Stock-Based Compensation Expense —Stock-based compensation expense related to stock awards to employees is measured at fair value of the award on the date of the grant. The Company estimates the grant-date fair value, and the resulting stock-based compensation expense, using the Black-Scholes valuation model for stock options and using intrinsic value, which is the closing price of its common stock on the date of the grant, for the restricted stock units (“RSUs”). The fair value of the award that is ultimately expected to vest is recognized as expense on a straight-line basis over the requisite service period, which is generally the vesting period. The Black-Scholes valuation model requires the use of following assumptions: Expected Term —The expected term assumption represents the period that the Company’s stock-based awards are expected to be outstanding and is determined using the simplified method. Expected Volatility —Expected volatility is estimated using comparable public companies’ volatility for similar terms. Expected Dividend —The Black-Scholes valuation model calls for a single expected dividend yield as an input. The Company has never paid dividends and has no plans to pay dividends. Risk-Free Interest Rate —The risk-free interest rate is based on the U.S. Treasury zero-coupon issues in effect at the time of grant for periods corresponding with the expected term of option. Stock-based compensation expense related to awards granted to non-employees is recognized based on the then-current fair value at each measurement date over the associated service period of the award, which is generally the vesting term, using the accelerated attribution method. The fair value of non-employee stock options is estimated using the Black-Scholes valuation model with assumptions generally consistent with those used for employee stock options, with the exception of the expected term, which is the remaining contractual life at each measurement date. Refer to Note 12 for more information on assumptions used in estimating stock-based compensation expense. |
Income Taxes | Income Taxes —The Company accounts for income taxes using the asset and liability method. The Company recognizes deferred tax assets and liabilities for the expected future tax consequences of events that have been included in the financial statements or tax returns. Deferred tax assets and liabilities are determined based on the difference 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. In evaluating the ability to recover its deferred income tax assets, the Company considers all available positive and negative evidence, including its operating results, ongoing tax planning and forecasts of future taxable income on a jurisdiction-by-jurisdiction basis. In the event the Company determines that it would be able to realize its deferred income tax assets in the future in excess of their net recorded amount, it would make an adjustment to the valuation allowance that would reduce the provision for income taxes. Conversely, in the event that all or part of the net deferred tax assets are determined not to be realizable in the future, an adjustment to the valuation allowance would be charged to earnings in the period when such determination is made. As of December 31, 2018 and 2017, the Company has recorded a full valuation allowance on its deferred tax assets. Tax benefits related to uncertain tax positions are recognized when it is more likely than not that a tax position will be sustained during an audit. Interest and penalties related to unrecognized tax benefits are included within the provision for income tax. |
Comprehensive Loss | Comprehensive Loss —Comprehensive loss comprises net loss and other comprehensive income (loss). Other comprehensive income (loss) consists of foreign currency translation adjustments related to translation of the financial statements of the Company’s Australia and France subsidiaries and unrealized gain (loss) on marketable securities. The Company did not have reclassifications from other comprehensive income (loss) to the income (loss) during the years ended December 31, 2018 and 2017. |
Basic and Diluted Net Loss Per Share | Basic and Diluted Net Loss Per Share — Basic net loss per share is calculated by dividing the net loss by the weighted-average number of shares of common stock outstanding for the period. Diluted net loss per share is computed by giving effect to all potential dilutive common stock equivalents outstanding for the period using the treasury stock method. Outstanding stock options, RSUs, ESPP and warrants are considered to be common stock equivalents and are only included in the calculation of diluted net loss per share when their effect is dilutive. |
Recent Accounting Standard Update Not Yet Effective | Recent Accounting Standard Update Not Yet Effective — In February 2016, the Financial Accounting Standards Board (“FASB”) issued ASU No. 2016-2, Leases, which amends the current guidance on leasing activities to provide more transparency and comparability, and requires that all leases be recognized by lessees on their balance sheet as a right-of-use asset and corresponding lease liability, which are currently accounted for as operating leases. The new standard is effective for fiscal years and interim periods within those fiscal years beginning after December 15, 2018, with early adoption permitted. We adopted the new standard using the modified retrospective approach as of January 1, 2019 and expect to record a lease liability in the range of $24.0 million to $27.0 million, and a right‑to‑use asset of approximately $23.0 million to $26.0 million, and no adjustment to the accumulated deficit. While the Company continues to evaluate the effect of the standard, the Company anticipates that the adoption will result in a material increase in assets and liabilities on its consolidated balance sheet and will not have a material impact on the consolidated statement of operations or statement of cash flows. In June 2018, the FASB issued ASU 2018-07, “Compensation – Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting” that expands the scope of ASC Topic 718 to include share-based payment transactions for acquiring goods and services from nonemployees. An entity should apply the requirements of ASC Topic 718 to nonemployee awards except for certain exemptions specified in the amendment. The guidance is effective for fiscal years beginning after December 15, 2018, including interim reporting periods within that fiscal year. Early adoption is permitted, but no earlier than an entity’s adoption date of Topic 606. The Company is currently evaluating the impact of the guidance on its consolidated financial statements. |
Cash Equivalents and Short-Te_2
Cash Equivalents and Short-Term Investments (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Cash And Cash Equivalents [Abstract] | |
Summary of Cash Equivalents and Short-term Investments | The following is a summary of the Company’s cash equivalents and short-term investments (in thousands): December 31, 2018 Amortized Cost Basis Unrealized Gains Unrealized Loses Estimated Fair Value Money market funds $ 126 $ — $ — $ 126 U.S. government and agency securities 25,792 1 (4 ) 25,789 Commercial paper 147,606 — — 147,606 Corporate bonds 27,778 5 (17 ) 27,766 Certificates of deposit 1,420 — — 1,420 Total cash equivalents and short-term investments 202,722 6 (21 ) 202,707 Less: Cash equivalents (152,577 ) — — (152,577 ) Total short-term investments $ 50,145 $ 6 $ (21 ) $ 50,130 December 31, 2017 Money market funds $ 65 $ — $ — $ 65 U.S. government and agency securities 58,351 — (145 ) 58,206 Commercial paper 71,427 — — 71,427 Corporate bonds 38,354 1 (38 ) 38,317 Certificates of deposit 9,731 — — 9,731 Total cash equivalents and short-term investments 177,928 1 (183 ) 177,746 Less: Cash equivalents (57,780 ) — — (57,780 ) Total short-term investments $ 120,148 $ 1 $ (183 ) $ 119,966 |
Fair Value Measurements and F_2
Fair Value Measurements and Fair Value of Financial Instruments (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Fair Value Disclosures [Abstract] | |
Financial Assets and Liabilities Measured and Recognized at Fair Value | The following table summarizes, for assets and liabilities recorded at fair value, the respective fair value and the classification by level of input within the fair value hierarchy as described above (in thousands): Quoted Prices Significant Other Significant Total In Observable Inputs Unobservable Inputs December 31, 2018 Carrying (Level 1) (Level 2) (Level 3) Money market funds $ 126 $ 126 $ — $ — U.S. government and agency securities 25,792 — 25,792 — Commercial paper 147,606 — 147,606 — Corporate bonds 27,778 — 27,778 — Certificates of deposit 1,420 — 1,420 — Total cash equivalents and short-term investments $ 202,722 $ 126 $ 202,596 $ — Other noncurrent liability: Financing arrangement $ — $ — $ — $ — December 31, 2017 Assets: Money market funds $ 65 $ 65 $ — $ — U.S. government and agency securities 58,206 — 58,206 — Commercial paper 71,427 — 71,427 — Corporate bonds 38,317 — 38,317 — Certificates of deposit 9,731 — 9,731 — Total cash equivalents and short-term investments $ 177,746 $ 65 $ 177,681 $ — Other noncurrent liability: Financing arrangement $ 157 $ — $ — $ 157 |
Property and Equipment, Net (Ta
Property and Equipment, Net (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Property Plant And Equipment [Abstract] | |
Schedule of Property and Equipment, Net | Property and equipment, net consists of the following: December 31, 2018 2017 (In thousands) Computer equipment and software $ 646 $ 535 Laboratory equipment 5,470 4,956 Furniture and fixtures 678 552 Leasehold improvements 1,602 1,549 Construction in progress 1,612 105 Total property and equipment 10,008 7,697 Less accumulated depreciation and amortization (6,422 ) (4,673 ) Property and equipment, net $ 3,586 $ 3,024 |
Accrued Expenses and Other Cu_2
Accrued Expenses and Other Current Liabilities (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Payables And Accruals [Abstract] | |
Schedule of Accrued Expenses and Other Current Liabilities | Accrued expenses and other current liabilities consist of the following: December 31, 2018 2017 (In thousands) Compensation expense $ 2,944 $ 2,259 Accrued preclinical costs 289 1,255 Accrued professional fees 2,291 2,295 Accrued clinical and process development costs 1,561 910 Other 1,699 245 Total accrued expenses and other current liabilities $ 8,784 $ 6,964 |
Financing Arrangements (Tables)
Financing Arrangements (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Debt Disclosure [Abstract] | |
Schedule of TAP Financing Activity | The following table presents the TAP financing activity: Years ended December 31, 2018 2017 (In thousands) Balance of TAP financing liability as of the beginning of the year (1) $ 157 $ 74 Funding (2) — 100 Gain on fair value of TAP financing liability ( 3 ) (157 ) (17 ) Balance of TAP financing liability as of the end of the year $ — $ 157 ___________ (1) Recorded within other non-current liabilities in the Company’s consolidated balance sheets. (2) Recorded as a receivable as of December 31, 2017. Payment was received in January 2018. (3) Recorded within other non-current liabilities in the Company’s consolidated balance sheet and other income, net in the Company consolidated statement of operations and comprehensive loss. |
Commitments and Contingencies (
Commitments and Contingencies (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Commitments And Contingencies Disclosure [Abstract] | |
Schedule of Future Minimum Commitments under Facility Operating Leases | As of December 31, 2018, future minimum commitments under the Company’s facility operating leases were as follows: Years ended December 31, Future Commitments (In thousands) 2019 $ 3,344 2020 4,221 2021 4,683 2022 4,846 2023 5,016 Thereafter 28,837 Total minimum lease payments $ 50,947 |
Stock Plans (Tables)
Stock Plans (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Schedule of Fair Value of Option Issued to Employees Valuation Assumptions | The fair value of each stock option issued to employees was estimated at the date of grant using the Black-Scholes valuation model with the following weighted-average assumptions: Options Employee Stock Purchase Plan Years ended December 31, Years ended December 31, 2018 2017 2018 2017 Expected volatility 80% 82% 78% 52% Expected term (in years) 6.0 6.0 0.5 0.5 Expected dividend yield — — — — Risk-free interest rate 2.8% 1.9% 2.3% 1.3% |
Summary of Restricted Stock Units Activity | The following table summarizes the RSU activity under the Company’s stock plans and related information: (In thousands, except grant date fair value and years) Number of Units (in thousands) Weighted- Average Grant Date Fair Value (in dollars) Weighted- Average Remaining Contractual Term (in years) Balance at December 31, 2016 1,049 $ 5.47 1.7 Granted 2,543 2.81 Vested and released (307 ) 6.43 Forfeited (770 ) 3.58 Balance at December 31, 2017 2,515 $ 3.24 1.6 Granted 1,381 5.94 Vested and released (774 ) (3.46 ) Forfeited (725 ) (4.28 ) Balance at December 31, 2018 2,397 $ 9.23 4.8 |
Stock-Based Compensation Expense | The following table presents, by operating expense, the Company’s stock-based compensation expense: Years ended December 31, 2018 2017 (In thousands) Research and development $ 4,820 $ 5,253 General and administrative 8,612 3,470 Total share-based compensation expense $ 13,432 $ 8,723 |
Non Employee Stock Option [Member] | |
Schedule of Non-Employee Stock Option Plan Valuation Assumptions | The following table presents the weighted-average assumptions used in the Black-Scholes valuation model to estimate the fair value of non-employee stock options: Years ended December 31, 2018 2017 Expected volatility 77% 84% Expected term (in years) 7.7 8.8 Expected dividend yield — — Risk-free interest rate 2.7% 2.3% |
2006 and 2014 Equity Incentive Plan [Member] | |
Summary of Stock Options Activity | The following table summarizes stock option activity under the Company’s stock plans and related information: (In thousands, except exercise prices and years) Options Outstanding Weighted- Average Exercise Price Weighted- Average Remaining Contract Life (in years) Aggregate Intrinsic Value (a) Balance at December 31, 2016 7,449 $ 4.46 8.4 $ 11,837 Granted 1,989 2.85 Exercised (1,808 ) 0.20 Cancelled/forfeited (935 ) 8.87 Balance at December 31, 2017 6,695 $ 4.51 7.4 $ 9,539 Granted 2,049 5.94 Exercised (1,606 ) 0.43 Cancelled/forfeited (691 ) 5.90 Balance at December 31, 2018 6,447 $ 5.83 7.6 $ 3,594 Vested and expected to vest as of December 31, 2018 6,447 $ 5.83 7.6 $ 3,594 Exercisable at December 31, 2018 3,321 $ 6.63 6.7 $ 2,630 (a) |
Income Taxes (Tables)
Income Taxes (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Income Tax Disclosure [Abstract] | |
Schedule of Components of Loss Before Income Taxes | The following table presents domestic and foreign components of loss before provision for income taxes: Years ended December 31, 2018 2017 (In thousands) U.S. $ (45,024 ) $ (36,923 ) Foreign (28,853 ) (19,224 ) Loss before income taxes $ (73,877 ) $ (56,147 ) |
Schedule of Reconciliation of Income Tax Expense Computed Statutory Federal Income Tax and Financial Statements | A reconciliation of income tax expense computed at the statutory federal income tax rate of 21% and 34% during the year ended December 31, 2018 and 2017, respectively, to income taxes as reflected in the financial statements is as follows: Years ended December 31, 2018 2017 (In thousands) Federal income tax expense at statutory rate $ (15,514 ) $ (19,090 ) Non-deductible foreign research expenses — 19 Stock compensation (1,512 ) (76 ) Non-deductible expenses 75 61 Other 41 — Research and development tax credits (1,215 ) (507 ) Change in valuation allowance 11,219 7,935 Foreign rate differential (586 ) 1,495 Rate change — 10,163 Change in uncertain tax positions 6,242 — Total tax benefit $ (1,250 ) $ — |
Schedule of Deferred Tax Assets | The following table presents significant components of the Company’s deferred tax assets: As of December 31, 2018 2017 (In thousands) Deferred tax assets: Net operating loss carryforwards $ 37,247 $ 27,142 Accruals, reserve and other 827 2,586 Stock-based compensation 4,549 3,623 Tax credit carryforwards 4,161 1,774 Property and equipment 310 274 Intangibles 38 64 Other 122 — Total deferred tax assets before valuation allowance 47,254 35,463 Valuation allowance (47,254 ) (35,463 ) Total deferred tax assets — — Deferred tax liabilities: IPR&D — (1,250 ) Total deferred tax liabilities $ — $ (1,250 ) Net deferred tax assets $ — $ — |
Schedule of Unrecognized Tax Benefits | A reconciliation of the unrecognized tax benefits is as follows: Years ended December 31, 2018 2017 (In thousands) Unrecognized tax benefits as of the beginning of the year $ 2,745 $ 2,157 Increase (decrease) related to prior year tax provisions 1,941 — Increase related to current year tax provisions 4,119 588 Unrecognized tax benefits as of the end of the year $ 8,805 $ 2,745 |
Net Loss per Share (Tables)
Net Loss per Share (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Earnings Per Share [Abstract] | |
Schedule of Antidilutive Common Stock Equivalents Excluded from Calculation of Diluted Net Loss Per Share | The following common stock equivalents outstanding at the end of the periods presented were excluded from the calculation of diluted net loss per share for the periods indicated because including them would have had an anti-dilutive effect: As of December 31, 2018 2017 (In thousands) Stock options 6,447 6,695 Restricted stock units 2,397 2,515 ESPP 62 71 Warrants to purchase common stock 90 90 8,996 9,371 |
Selected Quarterly Financial _2
Selected Quarterly Financial Information (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Quarterly Financial Information Disclosure [Abstract] | |
Schedule of Quarterly Consolidated Results of Operations | The Company’s quarterly consolidated results of operations are shown below: Quarterly Results of Operations Three Months Ended March 31, 2018 June 30, 2018 September 30, 2018 December 31, 2018 (In thousands, except per share amounts) Revenue $ 216 $ 493 $ 833 $ 70 Total operating expenses (1) (18,162 ) (20,396 ) (24,306 ) (16,829 ) Net loss (17,200 ) (18,810 ) (20,958 ) (15,659 ) Basic and diluted net loss per share $ (0.30 ) $ (0.30 ) $ (0.34 ) $ (0.25 ) Quarterly Results of Operations Three Months Ended March 31, 2017 June 30, 2017 September 30, 2017 December 31, 2017 (In thousands, except per share amounts) Revenue $ 462 $ 463 $ 463 $ 461 Total operating expenses (1) (17,050 ) (12,556 ) (15,034 ) (16,056 ) Net loss (16,099 ) (11,430 ) (13,829 ) (14,789 ) Basic and diluted net loss per share (0.38 ) (0.27 ) (0.32 ) (0.32 ) (1) During the year ended December 31, 2017, the Company recorded a total of $2.0 million of estimated costs associated with the termination of MSA with Cornell University (see Note 10). During the three months ended March 31, 2017, the Company recorded a total of $2.3 million of these estimated costs, which was subsequently adjusted by $0.3 million during the three months ended June 30, 2017. |
Description of the Business - A
Description of the Business - Additional Information (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | ||
Date of incorporation | Jul. 17, 2006 | |
Accumulated deficit | $ (320,543) | $ (254,062) |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies - Additional Information (Detail) | 1 Months Ended | 3 Months Ended | 12 Months Ended | ||||||||||||
Feb. 28, 2018USD ($) | Aug. 31, 2016USD ($) | May 31, 2014USD ($) | Dec. 31, 2018USD ($) | Sep. 30, 2018USD ($) | Jun. 30, 2018USD ($) | Mar. 31, 2018USD ($) | Dec. 31, 2017USD ($) | Sep. 30, 2017USD ($) | Jun. 30, 2017USD ($) | Mar. 31, 2017USD ($) | Dec. 31, 2018USD ($)Segment | Dec. 31, 2017USD ($) | Jan. 01, 2019USD ($) | Jan. 01, 2018USD ($) | |
Summary Of Significant Accounting Policies [Line Items] | |||||||||||||||
Number of reportable segments | Segment | 1 | ||||||||||||||
Number of operating segments | Segment | 1 | ||||||||||||||
Impairment losses of amortizable long-lived assets | $ 0 | ||||||||||||||
Retained earnings (accumulated deficit) | $ (320,543,000) | $ (254,062,000) | $ (320,543,000) | (254,062,000) | |||||||||||
Collaboration and license revenue | $ 70,000 | $ 833,000 | $ 493,000 | $ 216,000 | $ 461,000 | $ 463,000 | $ 463,000 | $ 462,000 | $ 1,612,000 | $ 1,849,000 | |||||
Type of Revenue [Extensible List] | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember | |||||
Editas Medicine Inc [Member] | |||||||||||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||||||||||
Initial payments received | $ 500,000 | ||||||||||||||
Collaboration and license revenue | $ 1,400,000 | ||||||||||||||
Type of Revenue [Extensible List] | us-gaap:LicenseAndServiceMember | ||||||||||||||
Regeneron Corporation [Member] | |||||||||||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||||||||||
Deferred revenue | $ 0 | $ 0 | $ 0 | ||||||||||||
Accounting Standards Update 2014-09 [Member] | Editas Medicine Inc [Member] | |||||||||||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||||||||||
Deferred revenue | $ 0 | 0 | |||||||||||||
Initial payments received | $ 500,000 | ||||||||||||||
Collaboration and license revenue | $ 1,400,000 | ||||||||||||||
Type of Revenue [Extensible List] | us-gaap:LicenseAndServiceMember | ||||||||||||||
Accounting Standards Update 2014-09 [Member] | Upfront Payment Arrangement [Member] | Editas Medicine Inc [Member] | |||||||||||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||||||||||
Deferred revenue | $ 1,000,000 | ||||||||||||||
Initial payments received | $ 1,000,000 | ||||||||||||||
Accounting Standards Update 2014-09 [Member] | Regeneron Corporation [Member] | Upfront Payment Arrangement [Member] | |||||||||||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||||||||||
Deferred revenue | $ 8,000,000 | ||||||||||||||
Initial payments received | $ 8,000,000 | ||||||||||||||
ASU No. 2016-2, Leases [Member] | Subsequent Event [Member] | |||||||||||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||||||||||
Adjustment to accumulated deficit | $ 0 | ||||||||||||||
Difference between Revenue Guidance in Effect before and after Topic 606 [Member] | Accounting Standards Update 2014-09 [Member] | |||||||||||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||||||||||
Retained earnings (accumulated deficit) | $ 6,100,000 | ||||||||||||||
Deferred revenue | $ (6,100,000) | ||||||||||||||
Difference between Revenue Guidance in Effect before and after Topic 606 [Member] | Accounting Standards Update 2014-09 [Member] | Editas Medicine Inc [Member] | |||||||||||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||||||||||
Retained earnings (accumulated deficit) | (400,000) | ||||||||||||||
Deferred revenue | 400,000 | ||||||||||||||
Difference between Revenue Guidance in Effect before and after Topic 606 [Member] | Accounting Standards Update 2014-09 [Member] | Regeneron Corporation [Member] | |||||||||||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||||||||||
Retained earnings (accumulated deficit) | 6,500,000 | ||||||||||||||
Deferred revenue | $ (6,500,000) | ||||||||||||||
Calculated under Revenue Guidance in Effect before Topic 606 [Member] | Editas Medicine Inc [Member] | |||||||||||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||||||||||
Collaboration and license revenue | $ 2,200,000 | ||||||||||||||
Type of Revenue [Extensible List] | us-gaap:LicenseAndServiceMember | ||||||||||||||
Calculated under Revenue Guidance in Effect before Topic 606 [Member] | GenSight Biologics [Member] | License Agreement [Member] | |||||||||||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||||||||||
Collaboration and license revenue | $ 200,000 | ||||||||||||||
Calculated under Revenue Guidance in Effect before Topic 606 [Member] | Accounting Standards Update 2014-09 [Member] | Editas Medicine Inc [Member] | |||||||||||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||||||||||
Deferred revenue | $ 500,000 | $ 500,000 | |||||||||||||
Calculated under Revenue Guidance in Effect before Topic 606 [Member] | Accounting Standards Update 2014-09 [Member] | Regeneron Corporation [Member] | |||||||||||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||||||||||
Deferred revenue | $ 6,500,000 | $ 6,500,000 | |||||||||||||
IPR&D [Member] | |||||||||||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||||||||||
Impairment of intangible assets | $ 5,000,000 | ||||||||||||||
Minimum [Member] | |||||||||||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||||||||||
Estimated useful lives of assets | 3 years | ||||||||||||||
Minimum [Member] | ASU No. 2016-2, Leases [Member] | Subsequent Event [Member] | |||||||||||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||||||||||
Right-to-use asset | 23,000,000 | ||||||||||||||
Lease liability | 24,000,000 | ||||||||||||||
Maximum [Member] | |||||||||||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||||||||||
Estimated useful lives of assets | 5 years | ||||||||||||||
Maximum [Member] | ASU No. 2016-2, Leases [Member] | Subsequent Event [Member] | |||||||||||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||||||||||
Right-to-use asset | 26,000,000 | ||||||||||||||
Lease liability | $ 27,000,000 |
Impairment Evaluation for Goo_2
Impairment Evaluation for Goodwill and Intangible Assets - Additional Information (Details) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2018USD ($)Segment | Jun. 30, 2018USD ($) | Dec. 31, 2017USD ($) | |
Finite Lived Intangible Assets [Line Items] | |||
Intangible asset | $ 5,000 | ||
IPR&D [Member] | |||
Finite Lived Intangible Assets [Line Items] | |||
Impairment charge | $ 5,000 | ||
Annapurna Therapeutics SAS [Member] | IPR&D [Member] | |||
Finite Lived Intangible Assets [Line Items] | |||
Intangible asset | $ 5,000 | ||
Number of reporting units | Segment | 1 | ||
Impairment charge | $ 5,000 |
Cash Equivalents and Short-Te_3
Cash Equivalents and Short-Term Investments - Summary of Cash Equivalents and Short-term Investments (Detail) - USD ($) $ in Thousands | Dec. 31, 2018 | Dec. 31, 2017 |
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost Basis | $ 202,722 | $ 177,928 |
Unrealized Gains | 6 | 1 |
Unrealized Loses | (21) | (183) |
Estimated Fair Value | 202,707 | 177,746 |
Less: Cash Equivalents, Amortized Cost Basis | (154,949) | (70,519) |
Amortized Cost Basis | 50,145 | 120,148 |
Unrealized Gains | 6 | 1 |
Unrealized Loses | (21) | (183) |
Estimated Fair Value | 50,130 | 119,966 |
Money Market Funds [Member] | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost Basis | 126 | 65 |
Estimated Fair Value | 126 | 65 |
Commercial Paper [Member] | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost Basis | 147,606 | 71,427 |
Estimated Fair Value | 147,606 | 71,427 |
Cash Equivalents [Member] | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Less: Cash Equivalents, Amortized Cost Basis | (152,577) | (57,780) |
Less: Cash Equivalents, Unrealized Gains | 0 | 0 |
Less: Cash Equivalents, Unrealized Loses | 0 | 0 |
Less: Cash Equivalents, Estimated Fair Value | (152,577) | (57,780) |
U.S. Government Agency Securities [Member] | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost Basis | 25,792 | 58,351 |
Unrealized Gains | 1 | |
Unrealized Loses | (4) | (145) |
Estimated Fair Value | 25,789 | 58,206 |
Corporate Bonds [Member] | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost Basis | 27,778 | 38,354 |
Unrealized Gains | 5 | 1 |
Unrealized Loses | (17) | (38) |
Estimated Fair Value | 27,766 | 38,317 |
Certificates of Deposit [Member] | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost Basis | 1,420 | 9,731 |
Estimated Fair Value | $ 1,420 | $ 9,731 |
Cash Equivalents and Short-Te_4
Cash Equivalents and Short-Term Investments - Additional Information (Detail) | 12 Months Ended |
Dec. 31, 2018USD ($) | |
Available For Sale Securities [Abstract] | |
Marketable securities other-than-temporarily impaired | $ 0 |
Remaining maturity period of investments | 1 year |
Fair Value Measurements and F_3
Fair Value Measurements and Fair Value of Financial Instruments - Additional Information (Detail) - USD ($) | 12 Months Ended | ||
Dec. 31, 2018 | Dec. 31, 2017 | Aug. 31, 2016 | |
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | |||
Liabilities transferred within Level 3 | $ 0 | $ 0 | |
The Alpha-1 Project, Inc. [Member] | |||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | |||
Financing liability remeasured due to discontinue of A1AT program | 0 | ||
The Alpha-1 Project, Inc. [Member] | Significant Unobservable Inputs (Level 3) [Member] | |||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | |||
Amount borrowed under financing arrangement | $ 300,000 | ||
Proceeds from financing arrangement | $ 0 | $ 200,000 |
Fair Value Measurements and F_4
Fair Value Measurements and Fair Value of Financial Instruments - Financial Assets and Liabilities Measured and Recognized at Fair Value on Recurring Basis (Detail) - USD ($) $ in Thousands | Dec. 31, 2018 | Dec. 31, 2017 |
Assets: | ||
Assets | $ 202,722 | $ 177,746 |
Corporate Bonds [Member] | ||
Assets: | ||
Assets | 27,778 | 38,317 |
Financing Arrangement [Member] | ||
Other noncurrent liability: | ||
Other noncurrent liability | 157 | |
Money Market Funds [Member] | ||
Assets: | ||
Assets | 126 | 65 |
Commercial Paper [Member] | ||
Assets: | ||
Assets | 147,606 | 71,427 |
Certificates of Deposit [Member] | ||
Assets: | ||
Assets | 1,420 | 9,731 |
U.S. Government and Agency Securities [Member] | ||
Assets: | ||
Assets | 25,792 | 58,206 |
Quoted Prices in Active Markets (Level 1) [Member] | ||
Assets: | ||
Assets | 126 | 65 |
Quoted Prices in Active Markets (Level 1) [Member] | Money Market Funds [Member] | ||
Assets: | ||
Assets | 126 | 65 |
Significant Other Observable Inputs (Level 2) [Member] | ||
Assets: | ||
Assets | 202,596 | 177,681 |
Significant Other Observable Inputs (Level 2) [Member] | Corporate Bonds [Member] | ||
Assets: | ||
Assets | 27,778 | 38,317 |
Significant Other Observable Inputs (Level 2) [Member] | Commercial Paper [Member] | ||
Assets: | ||
Assets | 147,606 | 71,427 |
Significant Other Observable Inputs (Level 2) [Member] | Certificates of Deposit [Member] | ||
Assets: | ||
Assets | 1,420 | 9,731 |
Significant Other Observable Inputs (Level 2) [Member] | U.S. Government and Agency Securities [Member] | ||
Assets: | ||
Assets | $ 25,792 | 58,206 |
Significant Unobservable Inputs (Level 3) [Member] | Financing Arrangement [Member] | ||
Other noncurrent liability: | ||
Other noncurrent liability | $ 157 |
Significant Agreements - Additi
Significant Agreements - Additional Information (Detail) - USD ($) | 1 Months Ended | 3 Months Ended | 12 Months Ended | |||||||||
Feb. 28, 2018 | Aug. 31, 2016 | Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2017 | Sep. 30, 2017 | Jun. 30, 2017 | Mar. 31, 2017 | Dec. 31, 2018 | Dec. 31, 2017 | |
License Agreement [Line Items] | ||||||||||||
Revenue recognized | $ 70,000 | $ 833,000 | $ 493,000 | $ 216,000 | $ 461,000 | $ 463,000 | $ 463,000 | $ 462,000 | $ 1,612,000 | $ 1,849,000 | ||
Type of Revenue [Extensible List] | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember | ||
Editas Medicine Inc [Member] | ||||||||||||
License Agreement [Line Items] | ||||||||||||
Initial payments received | $ 500,000 | |||||||||||
Collaboration, option and license agreement fee receivable per indication | $ 1,000,000 | |||||||||||
Range of tiered royalties receivable on net sales | between the mid-single digits and low teens | |||||||||||
Revenue recognized | $ 1,400,000 | |||||||||||
Type of Revenue [Extensible List] | us-gaap:LicenseAndServiceMember | |||||||||||
Deferred revenue | $ 0 | $ 0 | ||||||||||
Editas Medicine Inc [Member] | Topic 606 [Member] | ||||||||||||
License Agreement [Line Items] | ||||||||||||
Initial payments received | $ 500,000 | |||||||||||
Collaboration, option and license agreement fee receivable per indication | 1,500,000 | |||||||||||
Non-refundable upfront payment received | 1,000,000 | |||||||||||
Revenue recognized | $ 1,400,000 | |||||||||||
Type of Revenue [Extensible List] | us-gaap:LicenseAndServiceMember | |||||||||||
Editas Medicine Inc [Member] | Additional Indication [Member] | ||||||||||||
License Agreement [Line Items] | ||||||||||||
Collaboration, option and license agreement fee receivable per indication | 1,500,000 | |||||||||||
Editas Medicine Inc [Member] | Maximum [Member] | ||||||||||||
License Agreement [Line Items] | ||||||||||||
Potential milestone earnings | $ 15,500,000 |
Property and Equipment, Net - S
Property and Equipment, Net - Schedule of Property and Equipment, Net (Detail) - USD ($) $ in Thousands | Dec. 31, 2018 | Dec. 31, 2017 |
Property, Plant and Equipment [Line Items] | ||
Total property and equipment | $ 10,008 | $ 7,697 |
Less accumulated depreciation and amortization | (6,422) | (4,673) |
Property and equipment, net | 3,586 | 3,024 |
Computer Equipment and Software [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Total property and equipment | 646 | 535 |
Laboratory Equipment [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Total property and equipment | 5,470 | 4,956 |
Furniture and Fixtures [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Total property and equipment | 678 | 552 |
Leasehold Improvements [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Total property and equipment | 1,602 | 1,549 |
Construction in Progress [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Total property and equipment | $ 1,612 | $ 105 |
Property and Equipment, Net - A
Property and Equipment, Net - Additional Information (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Property Plant And Equipment [Abstract] | ||
Depreciation and amortization expense | $ 1,750 | $ 2,096 |
Accrued Expenses and Other Cu_3
Accrued Expenses and Other Current Liabilities - Schedule of Accrued Expenses and Other Current Liabilities (Detail) - USD ($) $ in Thousands | Dec. 31, 2018 | Dec. 31, 2017 |
Accrued Expenses And Other Current Liabilities [Abstract] | ||
Compensation expense | $ 2,944 | $ 2,259 |
Accrued preclinical costs | 289 | 1,255 |
Accrued professional fees | 2,291 | 2,295 |
Accrued clinical and process development costs | 1,561 | 910 |
Other | 1,699 | 245 |
Total accrued expenses and other current liabilities | $ 8,784 | $ 6,964 |
Financing Arrangements - Additi
Financing Arrangements - Additional Information (Detail) | 1 Months Ended | 12 Months Ended | |||||
Dec. 31, 2017USD ($) | Sep. 30, 2016USD ($)$ / sharesshares | Jul. 31, 2016USD ($)Tranche$ / sharesshares | Dec. 31, 2018USD ($) | Dec. 31, 2018EUR (€) | Dec. 31, 2016EUR (€) | Aug. 31, 2015EUR (€) | |
Line Of Credit Facility [Line Items] | |||||||
Proceeds from financing arrangement | $ 100,000 | ||||||
The Alpha-1 Project, Inc. [Member] | |||||||
Line Of Credit Facility [Line Items] | |||||||
Potential milestone earnings | $ 100,000 | ||||||
Number Of Tranches | Tranche | 3 | ||||||
Proceeds from financing arrangement | $ 100,000 | ||||||
The Alpha-1 Project, Inc. [Member] | Common Shares [Member] | |||||||
Line Of Credit Facility [Line Items] | |||||||
Number of shares issued on exercise of warrants | shares | 10,000 | 10,000 | |||||
Exercise price | $ / shares | $ 4.33 | $ 4.33 | |||||
Expiration period | 5 years | 5 years | |||||
The Alpha-1 Project, Inc. [Member] | Maximum [Member] | |||||||
Line Of Credit Facility [Line Items] | |||||||
Potential milestone earnings | $ 300,000 | ||||||
Expected percentage of milestone repayments | 450.00% | ||||||
BPI France [Member] | |||||||
Line Of Credit Facility [Line Items] | |||||||
Interest free conditional advance, maximum borrowing capacity | € | € 800,000 | ||||||
Interest free conditional advance | 400,000 | $ 300,000 | € 500,000 | ||||
Interest free conditional advance, frequency of payments | Quarterly | Quarterly | |||||
Interest free conditional advance, periodic payment | € | € 25,000 | ||||||
Interest free conditional advance, beginning date of periodic payment | Sep. 30, 2017 | Sep. 30, 2017 | |||||
Interest free conditional advance, ending date of periodic payment | Jun. 30, 2022 | Jun. 30, 2022 | |||||
Interest free conditional advance, imputed interest rate | 7.00% | ||||||
BPI France [Member] | Other Noncurrent Liabilities [Member] | |||||||
Line Of Credit Facility [Line Items] | |||||||
Interest free conditional advance | 300,000 | $ 200,000 | |||||
BPI France [Member] | Accrued Expenses and Other Current Liabilities [Member] | |||||||
Line Of Credit Facility [Line Items] | |||||||
Interest free conditional advance | $ 100,000 | $ 100,000 |
Financing Arrangements - Summar
Financing Arrangements - Summary of TAP Financing Activity (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Line Of Credit Facility [Line Items] | ||
Balance of TAP financing liability as of the beginning of the year | $ 481 | |
Balance of TAP financing liability as of the end of the year | 243 | $ 481 |
TAP [Member] | ||
Line Of Credit Facility [Line Items] | ||
Balance of TAP financing liability as of the beginning of the year | 157 | 74 |
Funding | 100 | |
Gain on fair value of TAP financing liability | $ (157) | (17) |
Balance of TAP financing liability as of the end of the year | $ 157 |
Commitments and Contingencies -
Commitments and Contingencies - Additional Information (Detail) | 1 Months Ended | 3 Months Ended | 12 Months Ended | |||
Mar. 31, 2017USD ($) | Dec. 31, 2015USD ($)Claim | Jul. 31, 2015Claim | Mar. 31, 2017USD ($) | Dec. 31, 2018USD ($)Claim | Dec. 31, 2017USD ($)Claim | |
Other Commitments [Line Items] | ||||||
Lease renewal term | 4 years | |||||
Total minimum lease payments | $ 50,947,000 | |||||
Restricted cash, noncurrent | $ 999,000 | |||||
Lease expiration date | May 8, 2020 | |||||
Rent expense | $ 3,300,000 | $ 1,800,000 | ||||
Contractual obligation for manufacturing materials | 700,000 | |||||
Accrued royalties | 0 | |||||
Annual maintenance fee payable | $ 100,000 | $ 500,000 | ||||
Type of Cost, Good or Service [Extensible List] | us-gaap:MaintenanceMember | us-gaap:MaintenanceMember | ||||
Claims paid to date related to indemnification issues | $ 0 | $ 0 | ||||
Number of claims to date | Claim | 1 | 3 | 0 | 0 | ||
Accruals or expenses related to indemnification issues | $ 0 | $ 0 | ||||
Proposed aggregate amount of settlement | $ 13,000,000 | |||||
General and administrative expense | 24,560,000 | 20,857,000 | ||||
Indemnification Obligations [Member] | ||||||
Other Commitments [Line Items] | ||||||
Proposed aggregate amount of settlement | $ 1,000,000 | |||||
General and administrative expense | $ 1,000,000 | |||||
Cornell University [Member] | Master Service Agreement [Member] | ||||||
Other Commitments [Line Items] | ||||||
Lease term | 4 years | |||||
Total minimum lease payments | $ 13,300,000 | |||||
Estimated costs associated with the termination | $ 2,300,000 | $ 2,000,000 | ||||
Letter of Credit [Member] | ||||||
Other Commitments [Line Items] | ||||||
Security deposit | $ 1,000,000 | |||||
New Corporate Headquarters [Member] | ||||||
Other Commitments [Line Items] | ||||||
Lease entered date | Jun. 28, 2018 | |||||
Lease term | 10 years | |||||
Lease renewal term | 7 years | |||||
Lease description | The term of the lease is ten years and also provides for two options to extend the lease term for a period of seven years each. | |||||
Total minimum lease payments | $ 49,300,000 |
Commitments and Contingencies_2
Commitments and Contingencies - Schedule of Future Minimum Commitments under Facility Operating Leases (Detail) $ in Thousands | Dec. 31, 2018USD ($) |
Commitments And Contingencies Disclosure [Abstract] | |
2,019 | $ 3,344 |
2,020 | 4,221 |
2,021 | 4,683 |
2,022 | 4,846 |
2,023 | 5,016 |
Thereafter | 28,837 |
Total minimum lease payments | $ 50,947 |
Common Stock Warrants - Additio
Common Stock Warrants - Additional Information (Detail) | 1 Months Ended | 12 Months Ended | ||
Sep. 30, 2017USD ($)$ / sharesshares | Sep. 30, 2016$ / sharesshares | Jul. 31, 2016USD ($)$ / sharesshares | Dec. 31, 2015USD ($)$ / sharesshares | |
The Alpha-1 Project, Inc. [Member] | ||||
Class Of Warrant Or Right [Line Items] | ||||
Fair value of common stock price per share | $ 4.33 | |||
Value of warrants | $ | $ 26,000 | |||
The Alpha-1 Project, Inc. [Member] | Common Shares [Member] | ||||
Class Of Warrant Or Right [Line Items] | ||||
Warrant to purchase shares of common stock | shares | 10,000 | 10,000 | ||
Exercise price | $ 4.33 | $ 4.33 | ||
Expiration period | 5 years | 5 years | ||
Contractual Term [Member] | The Alpha-1 Project, Inc. [Member] | ||||
Class Of Warrant Or Right [Line Items] | ||||
Contractual term of warrant | 5 years | |||
Risk-Free Interest Rate [Member] | The Alpha-1 Project, Inc. [Member] | ||||
Class Of Warrant Or Right [Line Items] | ||||
Fair value of obligation | 1.07 | |||
Expected Volatility Rate [Member] | The Alpha-1 Project, Inc. [Member] | ||||
Class Of Warrant Or Right [Line Items] | ||||
Fair value of obligation | 72 | |||
Expected Dividend Yield [Member] | The Alpha-1 Project, Inc. [Member] | ||||
Class Of Warrant Or Right [Line Items] | ||||
Fair value of obligation | 0 | |||
LEI [Member] | ||||
Class Of Warrant Or Right [Line Items] | ||||
Warrant to purchase shares of common stock | shares | 40,000 | 40,000 | ||
Exercise price | $ 3.65 | $ 10.51 | ||
Warrant expiration date | Sep. 29, 2022 | Oct. 15, 2020 | ||
Fair value of obligation to issue warrant | $ | $ 100,000 | $ 200,000 | ||
Fair value of common stock price per share | $ 3.65 | $ 8.35 | ||
LEI [Member] | Contractual Term [Member] | ||||
Class Of Warrant Or Right [Line Items] | ||||
Contractual term of warrant | 5 years | 5 years | ||
LEI [Member] | Risk-Free Interest Rate [Member] | ||||
Class Of Warrant Or Right [Line Items] | ||||
Fair value of obligation | 1.89 | 1.34 | ||
LEI [Member] | Expected Volatility Rate [Member] | ||||
Class Of Warrant Or Right [Line Items] | ||||
Fair value of obligation | 91 | 75 | ||
LEI [Member] | Expected Dividend Yield [Member] | ||||
Class Of Warrant Or Right [Line Items] | ||||
Fair value of obligation | 0 | 0 |
Stock Plans - Additional Inform
Stock Plans - Additional Information (Detail) $ / shares in Units, $ in Millions | 1 Months Ended | 12 Months Ended | |||
Jun. 30, 2017shares | Jul. 31, 2014shares | Dec. 31, 2018USD ($)Executive$ / sharesshares | Dec. 31, 2017USD ($)$ / shares | Oct. 31, 2017shares | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||
Total intrinsic value of stock options exercised | $ | $ 8.2 | $ 4.8 | |||
Weighted-average fair values of options granted | $ / shares | $ 4.15 | $ 1.99 | |||
Unrecognized stock-based compensation expense related to employees' awards | $ | $ 6.5 | ||||
Unrecognized stock-based compensation, weighted-average period | 2 years 2 months 12 days | ||||
One time share based payment charge related to cancellation of unvested stock option of CEO | $ | $ 4.1 | ||||
Number of departure executives | Executive | 2 | ||||
Executives [Member] | |||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||
Number of stock options granted | 150,000 | ||||
Plans [Member] | |||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||
Common stock shares authorized for issuance | 19,353,469 | ||||
Shares available for future grants | 2,695,347 | ||||
Non Employee Stock Option [Member] | |||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||
Stock options granted period | 7 years 8 months 12 days | 8 years 9 months 18 days | |||
Unrecognized stock-based compensation expense related to employees' awards | $ | $ 0.1 | ||||
Unrecognized stock-based compensation, weighted-average period | 1 year 1 month 6 days | ||||
Restricted Stock Units [Member] | |||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||
Unrecognized stock-based compensation, weighted-average period | 2 years 6 months | ||||
Weighted-average grant date fair value of RSUs granted | $ / shares | $ 5.94 | $ 2.81 | |||
Total fair values of RSUs vested | $ | $ 2.7 | $ 2 | |||
Unrecognized compensation cost | $ | $ 7.4 | ||||
Restricted Stock Units [Member] | Maximum [Member] | |||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||
Restricted stock units granted to employees vesting period | 4 years | ||||
Restricted Stock Units [Member] | Minimum [Member] | |||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||
Restricted stock units granted to employees vesting period | 2 years | ||||
2014 Equity Incentive Award Plan [Member] | |||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||
Percentage of annual increase in number of shares available for future issuance | 4.00% | ||||
2017 Inducement Plan [Member] | Stock Options and Restricted Stock Units [Member] | |||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||
Common stock shares available for future grant | 600,000 | ||||
2014 Plan And Inducement Plan [Member] | |||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||
Stock options granted description | Stock options under the 2014 Plan and the Inducement Plan may be granted for periods of up to 10 years and at prices no less than 100% of the estimated fair value of the shares on the date of grant as determined by the board of directors, provided, however, that the exercise price of an ISO and NSO granted to a 10% stockholder may not be less than 110% of the estimated fair value of the shares on the date of grant. | ||||
Stock options granted to employees and non-employees vesting period | 4 years | ||||
2014 Plan And Inducement Plan [Member] | Maximum [Member] | |||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||
Stock options granted period | 10 years | ||||
2014 Employee Stock Purchase Plan [Member] | |||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||
Common stock shares available for future grant | 208,833 | ||||
Shares available for future grants | 1,331,773 | ||||
Percentage increase in shares issued | 1.00% | ||||
Common stock, shares issued | 120,475 |
Stock Plans - Summary of Stock
Stock Plans - Summary of Stock Options Activity (Detail) - 2006 Equity Incentive Plan [Member] - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Share-based Compensation Arrangement by Share-based Payment Award, Options, Nonvested, Options Outstanding [Roll Forward] | |||
Options Outstanding, Beginning Balance | 6,695,000 | 7,449,000 | |
Options Outstanding, Granted | 2,049,000 | 1,989,000 | |
Options Outstanding, Exercised | (1,606,000) | (1,808,000) | |
Options Outstanding, Cancelled/forfeited | (691,000) | (935,000) | |
Options Outstanding, Ending Balance | 6,447,000 | 6,695,000 | 7,449,000 |
Options Outstanding, Vested and expected to vest | 6,447,000 | ||
Options Outstanding, Exercisable | 3,321,000 | ||
Share-based Compensation Arrangement by Share-based Payment Award, Options, Outstanding, Weighted Average Exercise Price [Abstract] | |||
Weighted-Average Exercise Price, Beginning Balance | $ 4.51 | $ 4.46 | |
Weighted-Average Exercise Price, Granted | 5.94 | 2.85 | |
Weighted-Average Exercise Price, Exercised | 0.43 | 0.20 | |
Weighted-Average Exercise Price, Cancelled/forfeited | 5.90 | 8.87 | |
Weighted-Average Exercise Price, Ending Balance | 5.83 | $ 4.51 | $ 4.46 |
Weighted-Average Exercise Price, Vested and expected to vest | 5.83 | ||
Weighted-Average Exercise Price, Exercisable | $ 6.63 | ||
Share-based Compensation Arrangement by Share-based Payment Award, Options, Additional Disclosures [Abstract] | |||
Weighted-Average Remaining Contractual Life (In years) | 7 years 7 months 6 days | 7 years 4 months 24 days | 8 years 4 months 24 days |
Weighted-Average Remaining Contractual Life (In years), Vested and expected to vest | 7 years 7 months 6 days | ||
Weighted-Average Remaining Contractual Life (In years), Exercisable | 6 years 8 months 12 days | ||
Aggregate Intrinsic Value, Options outstanding | $ 3,594 | $ 9,539 | $ 11,837 |
Aggregate Intrinsic Value, Vested and expected to vest | 3,594 | ||
Aggregate Intrinsic Value, Exercisable | $ 2,630 |
Stock Plans - Summary of Stoc_2
Stock Plans - Summary of Stock Options Activity (Parenthetical) (Detail) | 12 Months Ended |
Dec. 31, 2018$ / shares | |
Equity [Abstract] | |
Aggregate intrinsic value of option per share | $ 3.15 |
Stock Plans - Schedule of Emplo
Stock Plans - Schedule of Employees Stock Purchase Plan Valuation Assumptions (Detail) | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Employee Stock Option [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Expected volatility | 80.00% | 82.00% |
Expected term (in years) | 6 years | 6 years |
Risk-free interest rate | 2.80% | 1.90% |
Employee Stock Purchase Plan [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Expected volatility | 78.00% | 52.00% |
Expected term (in years) | 6 months | 6 months |
Risk-free interest rate | 2.30% | 1.30% |
Non Employee Stock Option [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Expected volatility | 77.00% | 84.00% |
Expected term (in years) | 7 years 8 months 12 days | 8 years 9 months 18 days |
Risk-free interest rate | 2.70% | 2.30% |
Stock Plans - Summary of Restri
Stock Plans - Summary of Restricted Stock Units Activity (Detail) - Restricted Stock Units [Member] - $ / shares | 12 Months Ended | ||
Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Number of Units, Beginning Balance | 2,515,000 | 1,049,000 | |
Number of Units, Granted | 1,381,000 | 2,543,000 | |
Number of Units, Vested and released | (774,000) | (307,000) | |
Number of Units, Forfeited | (725,000) | (770,000) | |
Number of Units, Ending Balance | 2,397,000 | 2,515,000 | 1,049,000 |
Weighted-Average Grant-Date Fair Value, Beginning Balance | $ 3.24 | $ 5.47 | |
Weighted-Average Grant-Date Fair Value, Granted | 5.94 | 2.81 | |
Weighted-Average Grant-Date Fair Value, Vested and released | 3.46 | 6.43 | |
Weighted-Average Grant-Date Fair Value, Forfeited | 4.28 | 3.58 | |
Weighted-Average Grant-Date Fair Value, Ending Balance | 9.23 | 3.24 | $ 5.47 |
Weighted-Average Grant-Date Fair Value, Vested and released | (3.46) | (6.43) | |
Weighted-Average Grant-Date Fair Value, Forfeited | $ (4.28) | $ (3.58) | |
Weighted-Average Remaining Contractual Term (In years) | 4 years 9 months 18 days | 1 year 7 months 6 days | 1 year 8 months 12 days |
Stock Plans - Stock-Based Compe
Stock Plans - Stock-Based Compensation Expense (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||
Total share-based compensation expense | $ 13,432 | $ 8,723 |
Research and Development [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||
Total share-based compensation expense | 4,820 | 5,253 |
General and Administrative [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||
Total share-based compensation expense | $ 8,612 | $ 3,470 |
401(k) Savings Plan - Additiona
401(k) Savings Plan - Additional Information (Detail) - USD ($) $ in Millions | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Compensation And Retirement Disclosure [Abstract] | ||
Contribution by company | $ 0.4 | $ 0.3 |
Income Taxes - Additional Infor
Income Taxes - Additional Information (Detail) - USD ($) | 3 Months Ended | 12 Months Ended | ||
Sep. 30, 2018 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Income Taxes [Line Items] | ||||
Income tax benefit of expense | $ 1,300,000 | $ 1,250,000 | $ 0 | |
Statutory federal income tax rate | 21.00% | 34.00% | ||
Provisional tax expense | $ 0 | |||
Increase (decrease) in valuation allowance due to net operating loss | 11,800,000 | $ 12,000,000 | ||
Unrecognized tax benefits | 8,805,000 | 2,745,000 | $ 2,157,000 | |
Unrecognized tax benefits, if recognized would effective annual tax rate | 0 | |||
Accrued interest or penalties related to uncertain tax positions | 0 | 0 | ||
Interest or penalties recognized | $ 0 | $ 0 | ||
Minimum [Member] | ||||
Income Taxes [Line Items] | ||||
Tax examinations, year under examination | 2,013 | |||
Maximum [Member] | ||||
Income Taxes [Line Items] | ||||
Tax examinations, year under examination | 2,018 | |||
Domestic Tax Authority [Member] | ||||
Income Taxes [Line Items] | ||||
NOL carryforwards | $ 100,000,000 | |||
NOL carryforwards subject to expiration | $ 57,000,000 | |||
NOL carryforwards expiration | expire at various years beginning with 2036 | |||
Domestic Tax Authority [Member] | Research Tax Credit Carryforward [Member] | ||||
Income Taxes [Line Items] | ||||
Tax credit carryforwards | $ 3,200,000 | |||
Tax credit carryforwards expiration | expire at various years beginning with 2036 | |||
State credit carryforwards [Member] | ||||
Income Taxes [Line Items] | ||||
NOL carryforwards | $ 61,100,000 | |||
NOL carryforwards expiration | expire at various years beginning with 2036 | |||
State credit carryforwards [Member] | Research Tax Credit Carryforward [Member] | ||||
Income Taxes [Line Items] | ||||
Tax credit carryforwards | $ 3,300,000 | |||
Foreign Tax Authority [Member] | ||||
Income Taxes [Line Items] | ||||
NOL carryforwards | $ 65,000,000 |
Income Taxes - Schedule of Comp
Income Taxes - Schedule of Components of Loss Before Income Taxes (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Income Tax Disclosure [Abstract] | ||
U.S. | $ (45,024) | $ (36,923) |
Foreign | (28,853) | (19,224) |
Net loss before income taxes | $ (73,877) | $ (56,147) |
Income Taxes - Schedule of Reco
Income Taxes - Schedule of Reconciliation of Income Tax Expense Computed Statutory Federal Income Tax and Financial Statements (Detail) - USD ($) | 3 Months Ended | 12 Months Ended | |
Sep. 30, 2018 | Dec. 31, 2018 | Dec. 31, 2017 | |
Income Tax Disclosure [Abstract] | |||
Federal income tax expense at statutory rate | $ (15,514,000) | $ (19,090,000) | |
Non-deductible foreign research expenses | 19,000 | ||
Stock compensation | (1,512,000) | (76,000) | |
Non-deductible expenses | 75,000 | 61,000 | |
Other | 41,000 | ||
Research and development tax credits | (1,215,000) | (507,000) | |
Change in valuation allowance | 11,219,000 | 7,935,000 | |
Foreign rate differential | (586,000) | 1,495,000 | |
Rate change | 10,163,000 | ||
Change in uncertain tax positions | 6,242,000 | ||
Total tax benefit | $ (1,300,000) | $ (1,250,000) | $ 0 |
Income Taxes - Schedule of Defe
Income Taxes - Schedule of Deferred Tax Assets (Detail) - USD ($) $ in Thousands | Dec. 31, 2018 | Dec. 31, 2017 |
Deferred tax assets: | ||
Net operating loss carryforwards | $ 37,247 | $ 27,142 |
Accruals, reserve and other | 827 | 2,586 |
Stock-based compensation | 4,549 | 3,623 |
Tax credit carryforwards | 4,161 | 1,774 |
Property and equipment | 310 | 274 |
Intangibles | 38 | 64 |
Other | 122 | |
Total deferred tax assets before valuation allowance | 47,254 | 35,463 |
Valuation allowance | $ (47,254) | (35,463) |
Deferred tax liabilities: | ||
IPR&D | (1,250) | |
Total deferred tax liabilities | $ (1,250) |
Income Taxes - Schedule of Unre
Income Taxes - Schedule of Unrecognized Tax Benefits (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Income Tax Disclosure [Abstract] | ||
Unrecognized tax benefits as of the beginning of the year | $ 2,745 | $ 2,157 |
Increase (decrease) related to prior year tax provisions | 1,941 | |
Increase related to current year tax provisions | 4,119 | 588 |
Unrecognized tax benefits as of the end of the year | $ 8,805 | $ 2,745 |
Net Loss per Share - Schedule o
Net Loss per Share - Schedule of Antidilutive Common Stock Equivalents Excluded from Calculation of Diluted Net Loss Per Share (Detail) - shares shares in Thousands | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Antidilutive common stock equivalents excluded from calculation of diluted net loss per share | 8,996 | 9,371 |
Stock Options [Member] | ||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Antidilutive common stock equivalents excluded from calculation of diluted net loss per share | 6,447 | 6,695 |
Restricted Stock Units [Member] | ||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Antidilutive common stock equivalents excluded from calculation of diluted net loss per share | 2,397 | 2,515 |
ESPP [Member] | ||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Antidilutive common stock equivalents excluded from calculation of diluted net loss per share | 62 | 71 |
Warrants to Purchase Common Stock [Member] | ||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Antidilutive common stock equivalents excluded from calculation of diluted net loss per share | 90 | 90 |
Selected Quarterly Financial _3
Selected Quarterly Financial Information - Schedule of Quarterly Consolidated Results of Operations (Detail) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 12 Months Ended | ||||||||
Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2017 | Sep. 30, 2017 | Jun. 30, 2017 | Mar. 31, 2017 | Dec. 31, 2018 | Dec. 31, 2017 | |
Quarterly Financial Information Disclosure [Abstract] | ||||||||||
Revenue | $ 70 | $ 833 | $ 493 | $ 216 | $ 461 | $ 463 | $ 463 | $ 462 | $ 1,612 | $ 1,849 |
Type of Revenue [Extensible List] | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember |
Total operating expenses | $ (16,829) | $ (24,306) | $ (20,396) | $ (18,162) | $ (16,056) | $ (15,034) | $ (12,556) | $ (17,050) | $ (79,693) | $ (60,696) |
Net loss | $ (15,659) | $ (20,958) | $ (18,810) | $ (17,200) | $ (14,789) | $ (13,829) | $ (11,430) | $ (16,099) | $ (72,627) | $ (56,147) |
Basic and diluted net loss per share | $ (0.25) | $ (0.34) | $ (0.30) | $ (0.30) | $ (0.32) | $ (0.32) | $ (0.27) | $ (0.38) | $ (1.18) | $ (1.29) |
Selected Quarterly Financial _4
Selected Quarterly Financial Information - Schedule of Quarterly Consolidated Results of Operations (Parenthetical) (Detail) - Cornell University [Member] - Master Service Agreement [Member] - USD ($) $ in Millions | 3 Months Ended | 12 Months Ended | |
Jun. 30, 2017 | Mar. 31, 2017 | Dec. 31, 2017 | |
Schedule Of Quarterly Financial Information [Line Items] | |||
Estimated costs associated with the termination | $ 2.3 | $ 2 | |
Estimated costs adjustments associated with the termination | $ 0.3 |