Cover Page
Cover Page - shares | 9 Months Ended | |
Sep. 30, 2019 | Nov. 04, 2019 | |
Cover page. | ||
Document Type | 10-Q | |
Document Quarterly Report | true | |
Document Period End Date | Sep. 30, 2019 | |
Document Transition Report | false | |
Entity File Number | 001-37833 | |
Entity Registrant Name | Audentes Therapeutics, Inc. | |
Entity Incorporation, State or Country Code | DE | |
Entity Tax Identification Number | 46-1606174 | |
Entity Address, Address Line One | 600 California Street, 17th Floor | |
Entity Address, City or Town | San Francisco | |
Entity Address, State or Province | CA | |
Entity Address, Postal Zip Code | 94108 | |
City Area Code | 415 | |
Local Phone Number | 818-1001 | |
Title of 12(b) Security | Common Stock, par value $0.00001 per share | |
Trading Symbol | BOLD | |
Security Exchange Name | NASDAQ | |
Entity Current Reporting Status | Yes | |
Entity Interactive Data Current | Yes | |
Entity Filer Category | Large Accelerated Filer | |
Entity Small Business | false | |
Entity Emerging Growth Company | false | |
Entity Shell Company | false | |
Entity Common Stock, Shares Outstanding | 45,783,265 | |
Amendment Flag | false | |
Document Fiscal Year Focus | 2019 | |
Document Fiscal Period Focus | Q3 | |
Entity Central Index Key | 0001628738 | |
Current Fiscal Year End Date | --12-31 |
Condensed Consolidated Balance
Condensed Consolidated Balance Sheets - USD ($) $ in Thousands | Sep. 30, 2019 | Dec. 31, 2018 |
Current assets: | ||
Cash and cash equivalents | $ 63,590 | $ 144,349 |
Short-term investments | 287,874 | 269,958 |
Prepaid expenses and other current assets | 4,919 | 5,465 |
Total current assets | 356,383 | 419,772 |
Restricted cash - long-term | 3,748 | 3,748 |
Property and equipment, net | 38,165 | 32,099 |
Right-of-use assets | 24,859 | |
Goodwill | 3,631 | 3,631 |
Intangible assets | 8,000 | 8,000 |
Other assets | 6,722 | 5,305 |
Total assets | 441,508 | 472,555 |
Current liabilities: | ||
Accounts payable | 9,905 | 8,123 |
Accrued liabilities | 14,550 | 12,928 |
Operating lease liabilities | 3,399 | |
Contingent acquisition consideration payable | 0 | 2,345 |
Deferred rent | 456 | |
Total current liabilities | 27,854 | 23,852 |
Deferred rent - long-term | 4,720 | |
Asset retirement obligation - long-term | 231 | 215 |
Operating lease liabilities - long-term | 26,490 | |
Contingent acquisition consideration payable - long-term | 2,460 | 0 |
Deferred tax liability, net | 1,014 | 1,014 |
Total liabilities | 58,049 | 29,801 |
Stockholders' equity: | ||
Preferred stock, $0.00001 par value, 10,000,000 shares authorized as of September 30, 2019 (unaudited) and December 31, 2018; 0 shares issued and outstanding as of September 30, 2019 (unaudited) and December 31, 2018, respectively | 0 | 0 |
Common stock, $0.00001 par value, 300,000,000 shares authorized as of September 30, 2019 (unaudited) and December 31, 2018; 45,740,575 and 43,546,786 shares issued and outstanding as of September 30, 2019 (unaudited) and December 31, 2018, respectively | 0 | 0 |
Additional paid-in capital | 842,599 | 762,284 |
Accumulated deficit | (459,366) | (319,470) |
Accumulated other comprehensive income (loss) | 226 | (60) |
Total stockholders' equity | 383,459 | 442,754 |
Total liabilities and stockholders' equity | $ 441,508 | $ 472,555 |
Condensed Consolidated Balanc_2
Condensed Consolidated Balance Sheets (Parenthetical) - $ / shares | Sep. 30, 2019 | Dec. 31, 2018 |
Statement of Financial Position [Abstract] | ||
Preferred stock, par value (USD per share) | $ 0.00001 | $ 0.00001 |
Preferred stock, shares authorized (in shares) | 10,000,000 | 10,000,000 |
Preferred stock, shares issued (in shares) | 0 | 0 |
Preferred stock, shares outstanding (in shares) | 0 | 0 |
Common stock, par value (USD per share) | $ 0.00001 | $ 0.00001 |
Common stock, shares authorized (in shares) | 300,000,000 | 300,000,000 |
Common stock, shares issued (in shares) | 45,740,575 | 43,546,786 |
Common stock, shares outstanding (in shares) | 45,740,575 | 43,546,786 |
Condensed Consolidated Statemen
Condensed Consolidated Statements of Operations and Comprehensive Loss - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2019 | Sep. 30, 2018 | Sep. 30, 2019 | Sep. 30, 2018 | |
Operating expenses: | ||||
Research and development | $ 37,636 | $ 29,918 | $ 114,772 | $ 76,157 |
General and administrative | 10,189 | 7,817 | 31,960 | 20,617 |
Total operating expenses | 47,825 | 37,735 | 146,732 | 96,774 |
Loss from operations | (47,825) | (37,735) | (146,732) | (96,774) |
Interest income, net | 2,112 | 1,509 | 6,937 | 3,662 |
Other expense, net | (28) | (65) | (101) | (117) |
Net loss | (45,741) | (36,291) | (139,896) | (93,229) |
Unrealized (losses) gains on investments, net | 0 | (4) | 286 | 33 |
Comprehensive loss | $ (45,741) | $ (36,295) | $ (139,610) | $ (93,196) |
Net loss per share, basic and diluted (USD per share) | $ (1) | $ (0.97) | $ (3.14) | $ (2.57) |
Weighted-average number of shares used in computing net loss per share, basic and diluted (in shares) | 45,543,354 | 37,359,877 | 44,538,676 | 36,302,803 |
Condensed Consolidated Statem_2
Condensed Consolidated Statements of Stockholders' Equity Statement - USD ($) $ in Thousands | Total | Common Stock | Additional Paid-in Capital | Accumulated Deficit | Accumulated Other Comprehensive Income (Loss) |
Beginning balance (in shares) at Dec. 31, 2017 | 29,901,368 | ||||
Beginning balance at Dec. 31, 2017 | $ 156,598 | $ 0 | $ 347,327 | $ (190,649) | $ (80) |
Increase (Decrease) in Stockholders' Equity [Roll Forward] | |||||
Issuance of common stock upon exercise of stock options (in shares) | 183,692 | ||||
Issuance of common stock upon exercise of stock options | 807 | 807 | |||
Stock-based compensation expense | 3,385 | 3,385 | |||
Issuance of common stock, net of issuance costs (in shares) | 6,612,500 | ||||
Issuance of common stock, net of issuance costs | 217,237 | 217,237 | |||
Net loss | (25,571) | (25,571) | |||
Unrealized gain (loss) on investments, net | (14) | (14) | |||
Ending balance (in shares) at Mar. 31, 2018 | 36,697,560 | ||||
Ending balance at Mar. 31, 2018 | 352,442 | $ 0 | 568,756 | (216,220) | (94) |
Beginning balance (in shares) at Dec. 31, 2017 | 29,901,368 | ||||
Beginning balance at Dec. 31, 2017 | 156,598 | $ 0 | 347,327 | (190,649) | (80) |
Increase (Decrease) in Stockholders' Equity [Roll Forward] | |||||
Net loss | (93,229) | ||||
Ending balance (in shares) at Sep. 30, 2018 | 37,453,129 | ||||
Ending balance at Sep. 30, 2018 | 310,555 | $ 0 | 594,480 | (283,878) | (47) |
Beginning balance (in shares) at Mar. 31, 2018 | 36,697,560 | ||||
Beginning balance at Mar. 31, 2018 | 352,442 | $ 0 | 568,756 | (216,220) | (94) |
Increase (Decrease) in Stockholders' Equity [Roll Forward] | |||||
Issuance of common stock upon exercise of stock options (in shares) | 141,828 | ||||
Issuance of common stock upon exercise of stock options | 1,139 | 1,139 | |||
Issuance of common stock upon exercise of warrants (in shares) | 5,800 | ||||
Stock-based compensation expense | 4,097 | 4,097 | |||
Issuance of common stock, net of issuance costs (in shares) | 400,024 | ||||
Issuance of common stock, net of issuance costs | 14,602 | 14,602 | |||
Net loss | (31,367) | (31,367) | |||
Unrealized gain (loss) on investments, net | 51 | 51 | |||
Ending balance (in shares) at Jun. 30, 2018 | 37,245,212 | ||||
Ending balance at Jun. 30, 2018 | 340,964 | $ 0 | 588,594 | (247,587) | (43) |
Increase (Decrease) in Stockholders' Equity [Roll Forward] | |||||
Issuance of common stock upon exercise of stock options (in shares) | 207,917 | ||||
Issuance of common stock upon exercise of stock options | 1,253 | 1,253 | |||
Stock-based compensation expense | 4,633 | 4,633 | |||
Net loss | (36,291) | (36,291) | |||
Unrealized gain (loss) on investments, net | (4) | (4) | |||
Ending balance (in shares) at Sep. 30, 2018 | 37,453,129 | ||||
Ending balance at Sep. 30, 2018 | $ 310,555 | $ 0 | 594,480 | (283,878) | (47) |
Beginning balance (in shares) at Dec. 31, 2018 | 43,546,786 | 43,546,786 | |||
Beginning balance at Dec. 31, 2018 | $ 442,754 | $ 0 | 762,284 | (319,470) | (60) |
Increase (Decrease) in Stockholders' Equity [Roll Forward] | |||||
Issuance of common stock upon exercise of stock options (in shares) | 213,891 | ||||
Issuance of common stock upon exercise of stock options | 3,005 | 3,005 | |||
Stock-based compensation expense | 5,500 | 5,500 | |||
Net loss | (49,391) | (49,391) | |||
Unrealized gain (loss) on investments, net | 127 | 127 | |||
Ending balance (in shares) at Mar. 31, 2019 | 43,760,677 | ||||
Ending balance at Mar. 31, 2019 | $ 401,995 | $ 0 | 770,789 | (368,861) | 67 |
Beginning balance (in shares) at Dec. 31, 2018 | 43,546,786 | 43,546,786 | |||
Beginning balance at Dec. 31, 2018 | $ 442,754 | $ 0 | 762,284 | (319,470) | (60) |
Increase (Decrease) in Stockholders' Equity [Roll Forward] | |||||
Issuance of common stock upon exercise of stock options (in shares) | 698,790 | ||||
Net loss | $ (139,896) | ||||
Ending balance (in shares) at Sep. 30, 2019 | 45,740,575 | 45,740,575 | |||
Ending balance at Sep. 30, 2019 | $ 383,459 | $ 0 | 842,599 | (459,366) | 226 |
Beginning balance (in shares) at Mar. 31, 2019 | 43,760,677 | ||||
Beginning balance at Mar. 31, 2019 | 401,995 | $ 0 | 770,789 | (368,861) | 67 |
Increase (Decrease) in Stockholders' Equity [Roll Forward] | |||||
Issuance of common stock upon exercise of stock options (in shares) | 141,316 | ||||
Issuance of common stock upon exercise of stock options | 1,673 | 1,673 | |||
Issuance of common stock pursuant to ESPP purchases (in shares) | 38,328 | ||||
Issuance of common stock pursuant to ESPP purchases | 767 | 767 | |||
Issuance of common stock upon vesting of restricted stock units, net of shares withheld for employee taxes (in shares) | 24,439 | ||||
Issuance of common stock upon vesting of restricted stock units, net of shares withheld for employee taxes | (567) | (567) | |||
Stock-based compensation expense | 6,131 | 6,131 | |||
Issuance of common stock, net of issuance costs (in shares) | 1,419,351 | ||||
Issuance of common stock, net of issuance costs | 54,308 | 54,308 | |||
Net loss | (44,764) | (44,764) | |||
Unrealized gain (loss) on investments, net | 159 | 159 | |||
Ending balance (in shares) at Jun. 30, 2019 | 45,384,111 | ||||
Ending balance at Jun. 30, 2019 | 419,702 | $ 0 | 833,101 | (413,625) | 226 |
Increase (Decrease) in Stockholders' Equity [Roll Forward] | |||||
Issuance of common stock upon exercise of stock options (in shares) | 343,583 | ||||
Issuance of common stock upon exercise of stock options | 3,950 | 3,950 | |||
Issuance of common stock upon vesting of restricted stock units, net of shares withheld for employee taxes (in shares) | 11,081 | ||||
Issuance of common stock upon vesting of restricted stock units, net of shares withheld for employee taxes | (229) | (229) | |||
Stock-based compensation expense | 5,712 | 5,712 | |||
Issuance of common stock, net of issuance costs (in shares) | 1,800 | ||||
Issuance of common stock, net of issuance costs | 65 | 65 | |||
Net loss | $ (45,741) | (45,741) | |||
Ending balance (in shares) at Sep. 30, 2019 | 45,740,575 | 45,740,575 | |||
Ending balance at Sep. 30, 2019 | $ 383,459 | $ 0 | $ 842,599 | $ (459,366) | $ 226 |
Condensed Consolidated Statem_3
Condensed Consolidated Statements of Stockholders' Equity (Parenthetical) - USD ($) $ in Thousands | 3 Months Ended | |||
Sep. 30, 2019 | Jun. 30, 2019 | Jun. 30, 2018 | Mar. 31, 2018 | |
Statement of Stockholders' Equity [Abstract] | ||||
Issuance of common stock, issuance costs | $ 2 | $ 1,695 | $ 292 | $ 14,201 |
Condensed Consolidated Statem_4
Condensed Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2019 | Sep. 30, 2018 | |
Cash flows from operating activities: | ||
Net loss | $ (139,896) | $ (93,229) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Depreciation and amortization | 5,732 | 3,948 |
Amortization of right-of-use assets | 1,966 | |
Stock-based compensation | 17,343 | 12,115 |
Accretion of discount on marketable securities | (2,878) | (856) |
Change in fair value of contingent acquisition consideration payable | 115 | (2,327) |
Other | 21 | 83 |
Changes in operating assets and liabilities: | ||
Prepaid expenses and other current assets | 591 | (1,212) |
Other assets | (1,417) | (3,260) |
Accounts payable | 70 | (724) |
Accrued liabilities | 1,548 | 4,635 |
Deferred rent | 0 | 1,332 |
Operating lease liabilities | (2,112) | |
Net cash used in operating activities | (118,917) | (79,495) |
Cash flows from investing activities: | ||
Purchases of property and equipment | (10,017) | (8,074) |
Proceeds from maturities of marketable securities | 381,351 | 127,880 |
Purchases of marketable securities | (396,103) | (209,952) |
Net cash used in investing activities | (24,769) | (90,146) |
Cash flows from financing activities: | ||
Proceeds from exercise of stock options and ESPP purchases | 9,350 | 3,199 |
Proceeds from issuance of common stock, net of issuance costs | 54,373 | 231,724 |
Tax paid related to net share settlement of equity awards | (796) | 0 |
Net cash provided by financing activities | 62,927 | 234,923 |
Net (decrease) increase in cash, cash equivalents and restricted cash | (80,759) | 65,282 |
Cash, cash equivalents and restricted cash at beginning of period | 148,097 | 42,661 |
Cash, cash equivalents and restricted cash at end of period | 67,338 | 107,943 |
Noncash investing and financing activities: | ||
Change in accounts payable and accrued liabilities related to property and equipment purchases | 1,786 | 2,200 |
Change in prepaid expenses and other current assets related to equity issuances and option exercises | 45 | $ 0 |
Right-of-use assets obtained in exchange for lease obligations | $ 5,155 |
Organization and Basis of Prese
Organization and Basis of Presentation | 9 Months Ended |
Sep. 30, 2019 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Organization and Basis of Presentation | Organization and Basis of Presentation Audentes Therapeutics, Inc., or the Company, was incorporated in the State of Delaware on November 13, 2012. The Company is an AAV-based genetic medicines company focused on developing and commercializing innovative products for patients living with serious rare neuromuscular diseases. The Company operates in one business segment, with its corporate headquarters located in San Francisco, California and its manufacturing and research operations located in South San Francisco, California. The accompanying consolidated financial statements include the accounts of Audentes Therapeutics, Inc., and its wholly owned subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. Need for Additional Capital The Company has incurred net losses from operations since inception and as of September 30, 2019 , had an accumulated deficit of $459.4 million. The Company expects that its development activities will continue to generate operating losses over the next several years. Common Stock Sales Agreement In March 2018, the Company filed an automatic universal shelf registration statement. Pursuant to the registration statement, the Company entered into an “at-the-market” program and sales agreement, or ATM, with Cowen and Company, LLC., or Cowen, under which the Company may, from time to time, offer and sell common stock having an aggregate offering value of up to $150.0 million . Upon delivery of a placement notice and subject to the terms and conditions of the sales agreement, Cowen would use its commercially reasonable efforts to sell the shares from time to time, based upon the Company’s instructions. Sales of the Company’s common stock, if any, would be made at market prices by any method that is deemed to be an “at the market offering” as defined in Rule 415 under the Securities Act of 1933, as amended, including sales made directly on the Nasdaq Stock Market, or Nasdaq, or any other existing trading market for the common stock, at market prices or as otherwise agreed with Cowen. Under the sales agreement, Cowen would be entitled to a commission of up to 3.0% of the gross proceeds per share sold. The Company has no obligation to sell any shares under the sales agreement and may, at any time, suspend offers under the sales agreement or terminate the sales agreement by giving written notice as specified in the sales agreement. During the nine months ended September 30, 2019 , the Company sold 1,421,151 shares of common stock under the ATM for aggregate net proceeds of $54.4 million . As of September 30, 2019, there was approximately $78.6 million (excluding commissions) available for future sales pursuant to the ATM. Liquidity As of September 30, 2019 , the Company had approximately $351.5 million of cash, cash equivalents and marketable securities, consisting of $63.6 million of cash and cash equivalents and $287.9 million of marketable securities. The Company believes that its balance of cash, cash equivalents and investments as of September 30, 2019 is sufficient to fund its current operational plan for at least the next twelve months from the issuance of these financial statements, though it may pursue additional capital through one or more equity offerings, debt financings or other third-party funding, including potential strategic alliances and licensing or collaboration arrangements. If financing is not available at adequate levels or on acceptable terms, the Company may need to reevaluate its operating plans. In addition, if the Company’s anticipated operating results are not achieved in future periods, planned expenditures may need to be reduced in order to extend the time period over which the then-available resources would be able to fund the Company’s operations. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 9 Months Ended |
Sep. 30, 2019 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | Summary of Significant Accounting Policies The Company's significant accounting policies are detailed in Note 2 of the “Notes to Consolidated Financial Statements” in the Company’s Annual Report on Form 10-K for the year ended December 31, 2018 . Except as detailed below, there have been no material changes to the Company’s significant accounting policies during the nine months ended September 30, 2019, as compared to the significant accounting policies disclosed in Note 2 - Significant Accounting Policies included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2018. Leases The Company determines if an arrangement is or contains a lease at inception by assessing whether the arrangement contains an identified asset and whether it has the right to control the identified asset. Right-of-use (ROU) assets represent the Company's right to use an underlying asset for the lease term and lease liabilities represent the Company's obligation to make lease payments arising from the lease. ROU assets and lease liabilities are recognized at the lease commencement date based on the present value of future lease payments over the lease term. As the implicit rate in the Company's leases is unknown, the Company uses its incremental borrowing rate based on the information available at the lease commencement date in determining the present value of future lease payments. The Company gives consideration to its credit risk, term of the lease, total lease payments and adjust for the impacts of collateral, as necessary, when calculating its incremental borrowing rates. The ROU asset is based on the measurement of the lease liability and also includes any lease payments made prior to or on lease commencement and excludes lease incentives and initial direct costs incurred, as applicable. The lease terms may include options to extend or terminate the lease when it is reasonably certain the Company will exercise any such options. Rent expense for the Company's operating leases is recognized on a straight-line basis over the lease term. The Company has lease agreements with lease and non-lease components. The Company has elected to not separate lease and non-lease components for any leases within its existing classes of assets and, as a result, accounts for the lease and non-lease components as a single lease component. The Company has also elected to not apply the recognition requirement to any leases within its existing classes of assets with a term of 12 months or less. Basis of Preparation The accompanying unaudited interim condensed consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States of America, or U.S. GAAP, and applicable rules and regulations of the Securities and Exchange Commission, or SEC, regarding interim financial reporting. As permitted under those rules, certain footnotes or other financial information that are normally required by U.S. GAAP have been condensed or omitted, and accordingly the balance sheet as of December 31, 2018 has been derived from audited financial statements at that date but does not include all of the information required by U.S. GAAP for complete financial statements. These unaudited interim condensed consolidated financial statements have been prepared on the same basis as the Company’s annual financial statements and, in the opinion of management, reflect all adjustments (consisting only of normal recurring adjustments) that are necessary for a fair presentation of the Company’s financial information. The results of operations for the three and nine months ended September 30, 2019 are not necessarily indicative of the results to be expected for the year ending December 31, 2019 or for any other interim period or for any other future year. The accompanying unaudited interim condensed consolidated financial statements and related financial information should be read in conjunction with the audited financial statements and the related notes thereto for the year ended December 31, 2018 included in the Company’s audited financial statements filed in its Annual Report on Form 10-K for the year ended December 31, 2018 . Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities, as of the date of the financial statements, and the reported amounts of any expenses during the reporting period. On an ongoing basis, management evaluates its estimates, including those related to accrued liabilities, acquisition contingent consideration, income taxes, and stock-based compensation. Management bases its estimates on historical experience, and on various other market-specific relevant assumptions that management believes to be reasonable, under the circumstances. Actual results may differ from those estimates. Concentration of Credit Risk Financial instruments that potentially subject the Company to a concentration of credit risk consist of cash and cash equivalents and marketable securities. The Company invests in a variety of financial instruments and in accordance with its investment policy, limits the amount of credit exposure with any one issuer, industry or geographic area for investments other than instruments backed by the U.S. federal government. Concentration of Manufacturing and Third-Party Services Risk The Company is subject to certain risks with respect to sources of supply of manufactured materials and drug product for use in its preclinical studies and clinical trials. Due to the technical aspects of manufacturing drug product for gene therapies, there exist few alternative sources of manufacturing. The Company is reliant upon its own internal manufacturing capability and a small number of third-party vendors to produce drug product in sufficient quantities and quality to conduct its research and development activities. Accounting Pronouncements Adopted in 2019 In August 2018, the Financial Accounting Standards Board, or FASB, issued ASU 2018-15, Intangibles - Goodwill and Other-Internal Use Software (Subtopic 350-40): Customer’s Accounting for Implementation Costs Incurred in a Cloud Computing Arrangement That is a Service Contract. This guidance requires companies to apply the internal-use software guidance in ASC 350-40 to implementation costs incurred in a hosting arrangement that is a service contract to determine whether to capitalize certain implementation costs or expense them as incurred. This guidance is effective for fiscal years, and interim periods within those fiscal years, beginning after December 15, 2019. Early adoption was permitted and the Company early adopted the standard on January 1, 2019. Adoption of the standard did not have a material impact on the Company's condensed consolidated financial statements. See Note 5 for further disclosure. In June 2018, the FASB issued ASU 2018-07, Compensation - Stock Compensation (Topic 718), Improvements to Nonemployee Share-Based Payment Accounting , which expands the scope of Topic 718 to include share-based payment transactions for acquiring goods and services from nonemployees. An entity should apply the requirements of Topic 718 to nonemployee awards except for specific guidance on inputs to an option pricing model and the attribution of cost. The measurement of equity-classified nonemployee awards is fixed at the grant date, and entities would measure the cost of awards subject to a performance condition using the outcome that is probable at the balance sheet date. The Company adopted this standard on January 1, 2019. As a result of adopting this standard, the Company no longer remeasures equity-classified nonemployee awards. The adoption of this new standard did not result in material impact on the Company's condensed consolidated financial statements. In February 2016, the FASB issued ASU No. 2016-02, Leases (Topic 842) , which establishes a comprehensive new lease accounting model. Under the new guidance, at the commencement date, lessees are required to recognize a lease liability with a corresponding right-of-use (ROU) asset. Effective January 1, 2019, the Company adopted Topic 842 using the modified retrospective approach provided by ASU 2018-11. Results for reporting periods beginning January 1, 2019 are presented under Topic 842, while prior period amounts were not adjusted and continue to be presented in accordance with the Company’s historical accounting under Topic 840, Leases . The Company elected certain practical expedients permitted under the transition guidance, including the election to carryforward historical lease classification. The Company also elected the short-term lease practical expedient, which allowed the Company to not recognize leases with a term of less than twelve months on its consolidated balance sheets. In addition, the Company elected the lease and non-lease components practical expedient, which allowed the Company to calculate the present value of the fixed payments without performing an allocation of lease and non-lease components. The impact of the adoption of Topic 842 on the accompanying Condensed Consolidated Balance Sheet as of January 1, 2019 was as follows: December 31, 2018 Effect of Adoption January 1, 2019 (in thousands) Operating lease right-of-use assets $ — $ 21,669 $ 21,669 Liabilities: Operating leases $ — $ 2,541 $ 2,541 Deferred rent $ 456 $ (456 ) $ — Operating lease liabilities - long-term $ — $ 24,304 $ 24,304 Deferred rent and asset retirement obligation - long-term $ 4,935 $ (4,720 ) $ 215 Adoption of the new standard resulted in recording operating lease right-of-use assets and operating lease liabilities of approximately $24.9 million and $29.9 million , respectively, on the Company’s condensed consolidated balance sheet as of September 30, 2019 . However, the adoption of the new standard did not have an impact on the Company’s beginning accumulated deficit, statement of operations or cash flows. For additional information regarding the Company’s leases, see Note 9 in the notes to the condensed consolidated financial statements. Accounting Pronouncements Not Yet Adopted In August 2018, the FASB issued ASU 2018-13, Fair Value Measurement (Topic 820) , which modifies, removes and adds certain disclosure requirements on fair value measurements based on the FASB Concepts Statement, Conceptual Framework for Financial Reporting—Chapter 8: Notes to Financial Statements. This guidance is effective for fiscal years, and interim periods within those fiscal years, beginning after December 15, 2019. The amendments on changes in unrealized gains and losses, the range and weighted average of significant unobservable inputs used to develop Level 3 fair value measurements and the narrative description of measurement uncertainty should be applied prospectively for only the most recent interim or annual period presented in the initial fiscal year of adoption. All other amendments should be applied retrospectively to all periods presented upon their effective date. Early adoption is permitted upon issuance of ASU 2018-13. An entity is permitted to early adopt any removed or modified disclosures upon issuance of ASU 2018-13 and delay adoption of the additional disclosures until their effective date. It is the Company’s expectation that adoption of this pronouncement will not have a material impact to its condensed consolidated financial statements. In January 2017, the FASB issued ASU 2017-04, Intangibles - Goodwill and Other (Topic 350): Simplifying the Test for Goodwill Impairment , which eliminates Step 2 from the goodwill impairment test. In addition, ASU 2017-04 eliminates the requirements for any reporting unit with a zero or negative carrying amount to perform a qualitative assessment and, if it fails that qualitative test, to perform Step 2 of the goodwill impairment test. ASU 2017-04 is effective for fiscal years beginning after December 15, 2019, with early adoption permitted. The Company is currently evaluating the impact that the standard will have on its consolidated financial statements and related disclosures. It is the Company’s expectation that adoption of this pronouncement will not have a material impact to its consolidated financial statements and related disclosures. |
Investments
Investments | 9 Months Ended |
Sep. 30, 2019 | |
Investments, Debt and Equity Securities [Abstract] | |
Investments | Investments Investments consist of available-for-sale marketable securities as follows (in thousands): September 30, 2019 Amortized Cost Unrealized Gains Unrealized Losses Fair Value (Unaudited) Money market funds $ 22,647 $ — $ — $ 22,647 Commercial paper 92,750 7 (3 ) 92,754 Corporate securities 111,201 178 — 111,379 U.S. treasury bills 10,760 2 — 10,762 U.S. government agency securities 61,367 35 (1 ) 61,401 U.S. agency bonds 37,579 8 — 37,587 Total available-for-sale securities $ 336,304 $ 230 $ (4 ) $ 336,530 December 31, 2018 Amortized Cost Unrealized Gains Unrealized Losses Fair Value Money market funds $ 33,399 $ — $ — $ 33,399 Commercial paper 144,578 — — 144,578 Corporate securities 82,670 8 (39 ) 82,639 U.S. treasury bills 60,573 — (8 ) 60,565 U.S. government agency securities 15,219 — — 15,219 U.S. agency bonds 51,411 — (22 ) 51,389 U.S. agency discount securities 18,716 — — 18,716 Total available-for-sale securities $ 406,566 $ 8 $ (69 ) $ 406,505 The following table summarizes the classification of the available-for-sale securities on the Company's condensed consolidated balance sheets (in thousands): September 30, 2019 December 31, 2018 (Unaudited) Cash and cash equivalents $ 48,656 $ 136,547 Short-term investments 287,874 269,958 Total $ 336,530 $ 406,505 The Company does not intend to sell the investments that are in an unrealized loss position, and it is unlikely that it will be required to sell the investments before recovery of the investments' amortized cost basis, which may be maturity. The unrealized losses were the result of interest rate fluctuations affecting the value of the underlying instruments and the Company determined that the unrealized losses at September 30, 2019 |
Fair Value Measurements
Fair Value Measurements | 9 Months Ended |
Sep. 30, 2019 | |
Fair Value Disclosures [Abstract] | |
Fair Value Measurements | Fair Value Measurements Fair value accounting is applied for all financial assets and liabilities that are recognized or disclosed at fair value in the financial statements on a recurring basis (at least annually). Financial instruments include cash and cash equivalents, restricted cash, accounts payable and accrued liabilities that approximate fair value due to their relatively short maturities. Assets and liabilities recorded at fair value on a recurring basis in the balance sheets are categorized based upon the level of judgment associated with the inputs used to measure their fair values. Fair value is defined as the exchange price that would be received for an asset or an exit price that would be paid to transfer a liability in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants on the measurement date. The authoritative guidance on fair value measurements establishes a three-tier fair value hierarchy for disclosure of fair value measurements as follows: Level 1 – Inputs are unadjusted, quoted prices in active markets for identical assets or liabilities at the measurement date; Level 2 – Inputs are observable, unadjusted quoted prices in active markets for similar assets or liabilities, unadjusted quoted prices for identical or similar assets or liabilities 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 related assets or liabilities; and Level 3 – Unobservable inputs that are significant to the measurement of the fair value of the assets or liabilities that are supported by little or no market data. Assets Measured at Fair Value Financial assets subject to fair value measurements on a recurring basis and the level of inputs used in such measurements are as follows (in thousands): September 30, 2019 Fair Value Measurements Using Total Level 1 Level 2 Level 3 (Unaudited) Money market funds $ 22,647 $ 22,647 $ — $ — Commercial paper 92,754 — 92,754 — Corporate securities 111,379 — 111,379 — U.S. treasury bills 10,762 — 10,762 — U.S. government agency securities 61,401 — 61,401 — U.S. agency bonds 37,587 — 37,587 — Total financial assets $ 336,530 $ 22,647 $ 313,883 $ — December 31, 2018 Fair Value Measurements Using Total Level 1 Level 2 Level 3 Money market funds $ 33,399 $ 33,399 $ — $ — Commercial paper 144,578 — 144,578 — Corporate securities 82,639 — 82,639 — U.S. treasury bills 60,565 — 60,565 — U.S. government agency securities 15,219 — 15,219 — U.S. agency bonds 51,389 — 51,389 — U.S. agency discount securities 18,716 — 18,716 — Total financial assets $ 406,505 $ 33,399 $ 373,106 $ — The financial assets listed above do not include the Company’s operating cash of $14.9 million and $7.8 million as of September 30, 2019 and December 31, 2018 , respectively. Liabilities Measured at Fair Value In August 2015, the Company acquired Cardiogen Sciences, Inc., or Cardiogen, a biotechnology company focused on the discovery and development of AAV gene therapy products for rare, inherited arrhythmogenic diseases. Pursuant to the terms of the acquisition, upon first dosing of a patient in a human clinical study involving AT307 for the treatment of CASQ2-CPVT, the Company is obligated to pay to former Cardiogen shareholders $4.2 million in common stock plus an additional $5.8 million in either cash or common stock, at the Company’s election, for aggregate contingent consideration of $10.0 million . The Company recorded a contingent consideration payable that is estimated using a probability-based income approach utilizing an appropriate discount rate. Key assumptions used by management to estimate the fair value of contingent acquisition consideration payable include estimated probability of occurrence, the estimated timing of when the milestone may be attained and assumed discount period and discount rate, which are Level 3 inputs. Changes in the fair value of the contingent acquisition consideration payable, resulting from management’s revision of key assumptions, are recorded in research and development expense in the consolidated statement of operations and comprehensive loss. The probability-based income approach used by management to estimate the fair value of the contingent acquisition consideration is most sensitive to changes in the estimated probability of occurrence. The following is a summary of the contingent acquisition consideration payable recorded in the accompanying consolidated balance sheets (in thousands): Amount (Unaudited) Balance, December 31, 2018 $ 2,345 Change in fair value of contingent acquisition consideration payable 115 Balance, September 30, 2019 $ 2,460 |
Balance Sheet Components
Balance Sheet Components | 9 Months Ended |
Sep. 30, 2019 | |
Property, Plant and Equipment [Abstract] | |
Balance Sheet Components | Balance Sheet Components Property and Equipment, Net Property and equipment, net, consist of the following (in thousands): September 30, 2019 December 31, 2018 (Unaudited) Furniture and office equipment $ 2,439 $ 1,898 Computer equipment 1,391 1,019 Software 526 513 Leasehold improvements 23,453 19,607 Laboratory equipment 11,694 9,570 Manufacturing equipment 8,129 6,619 Construction in progress and deposits on equipment 6,640 3,320 Total property and equipment 54,272 42,546 Less accumulated depreciation and amortization (16,107 ) (10,447 ) Property and equipment, net $ 38,165 $ 32,099 Property and equipment depreciation and amortization expense for the three months ended September 30, 2019 and 2018 was $2.0 million and $1.5 million , respectively. Property and equipment depreciation and amortization for the nine months ended September 30, 2019 and 2018 was $5.7 million and $3.9 million , respectively. Capitalized Implementation Costs of a Hosting Arrangement The Company implemented a new enterprise resource planning, or ERP, system in July 2019. The ERP system is a cloud-based hosting arrangement that is a service contract. The Company early and prospectively adopted ASU 2018-15, Intangibles - Goodwill and Other-Internal Use Software (Subtopic 350-40): Customer’s Accounting for Implementation Costs Incurred in a Cloud Computing Arrangement That is a Service Contract in the classification of costs incurred in connection with the implementation of this hosted ERP system. Based on the guidance, the Company expensed all costs (internal and external) that were incurred in the planning and post-implementation operation stages and capitalized approximately $2.5 million in costs related to the application development stage. The capitalized costs are amortized on a straight-line basis over the non-cancelable contract term of five years . As of September 30, 2019, approximately $0.5 million and $1.9 million of the capitalized costs were classified in current and noncurrent assets, respectively. The Company began amortizing the capitalized implementation costs on July 1, 2019, which was the date the ERP system was placed in production and ready for its intended use. Amortization expense for the three and nine months ended September 30, 2019 was $0.1 million . Accrued Liabilities Accrued liabilities consist of the following (in thousands): September 30, 2019 December 31, 2018 (Unaudited) Accrued payroll and related expenses $ 8,310 $ 8,581 Accrued research and development expenses 4,692 3,317 Accrued construction in progress 74 71 Accrued general and administrative expenses 1,474 959 Total accrued liabilities $ 14,550 $ 12,928 |
Commitments and Contingencies
Commitments and Contingencies | 9 Months Ended |
Sep. 30, 2019 | |
Commitments and Contingencies Disclosure [Abstract] | |
Commitments and contingencies | Commitments and Contingencies In February 2019, the Company entered into an exclusive license agreement with Nationwide Children's Hospital related to the Company's AT702 program. Pursuant to the agreement, the Company paid an upfront fee of $7.0 million and will be obligated to make certain milestone and royalty payments upon the achievement of developmental, regulatory and net sales milestones. As of September 30, 2019 , the Company is subject to contingent payments upon the achievement of certain development, regulatory and commercial milestones, totaling up to approximately $270.9 million across all of its licensing agreements. Of this amount, $74.0 million relates to the Company’s Crigler-Najjar and CASQ2 -CPVT programs, for which the Company previously announced plans to explore outlicensing opportunities to continue development activities . |
Stock Compensation
Stock Compensation | 9 Months Ended |
Sep. 30, 2019 | |
Share-based Payment Arrangement [Abstract] | |
Stock Compensation | Stock Compensation Stock-based Compensation Expense Stock-based compensation expense by category was as follows for the three and nine months ended September 30, 2019 and 2018 (in thousands): Three Months Ended September 30, Nine Months Ended September 30, 2019 2018 2019 2018 (Unaudited) Research and development $ 3,048 $ 2,611 $ 9,465 $ 6,930 General and administrative 2,664 2,022 7,878 5,185 Total stock-based compensation expense $ 5,712 $ 4,633 $ 17,343 $ 12,115 Stock Options The following table summarizes option activity for the nine months ended September 30, 2019 : Number of Options Outstanding Weighted- Average Exercise Price Per Option Balance, December 31, 2018 4,894,201 $ 18.79 Options granted 1,581,183 $ 28.04 Options exercised (698,790 ) $ 12.35 Options forfeited (664,866 ) $ 24.29 Balance, September 30, 2019 5,111,728 $ 21.82 The fair value of stock options granted to employees was estimated using a Black-Scholes option pricing model with the following assumptions: Three Months Ended September 30, Nine Months Ended September 30, 2019 2018 2019 2018 Expected term (in years) 6.0-6.1 6.1 5.5-6.1 5.5-6.1 Expected volatility 62-63% 70 % 62-68% 70-76% Risk-free interest rate 1.4-1.9% 2.8-3.0% 1.4-2.6% 2.3-3.0% Expected dividend yield — % — % — % — % Restricted Stock Units In January 2019, the Company's compensation committee of the board of directors approved the commencement of granting restricted stock units, or RSUs, to our employees. RSUs are share awards that entitle the holder to receive freely tradable shares of the Company's common stock upon the completion of a specific period of continued service. RSUs are generally subject to forfeiture if employment terminates prior to the release of vesting restrictions. RSUs granted are valued at the market price of the Company's common stock on the date of grant. The Company recognizes stock-based compensation expense for the fair value of RSUs on a straight-line basis over the requisite service period of these awards. The following table summarizes activity of RSUs granted to employees with service-based vesting during the nine months ended September 30, 2019: Number of RSUs Outstanding Weighted- Average Grant Date Fair Value Per RSU Balance, December 31, 2018 — RSUs granted 571,530 $ 28.59 RSUs vested (58,156 ) $ 24.89 RSUs forfeited (62,468 ) $ 24.94 Balance, September 30, 2019 450,906 $ 29.57 2016 Employee Stock Purchase Plan The Company's 2016 Employee Stock Purchase Plan, or the 2016 ESPP, was adopted on July 19, 2016 and commenced on May 1, 2018. The Company initially reserved 210,000 shares of common stock for issuance under the 2016 ESPP. The number of shares reserved for issuance under the 2016 ESPP increases automatically on January 1 of each calendar year through January 1, 2028 by the number of shares equal to 1% of the total outstanding shares of the Company’s common stock as of the immediately preceding December 31 . Under the 2016 ESPP, employees may purchase common stock through payroll deductions at a price equal to 85% of the lower of the fair market value at the beginning of the offering period or at the end of each applicable purchase period. The 2016 ESPP generally provides for offering periods of six months in duration with purchase periods ending on either May 15 or November 15. Contributions under the 2016 ESPP are limited to a maximum of 15% of an employee’s eligible compensation and purchases are settled with common stock from the ESPP’s previously authorized and available pool of shares. The expense for the three and nine months ended September 30, 2019 was based on the fair value of rights granted upon the commencement of an offering and calculated using the following assumptions: for the three and nine months ended September 30, 2019 expected term in years was 0.5 ; volatility was 54.6% and 54.6-62.2% , respectively; the risk-free interest rate was 2.4% and 2.4-2.5% , respectively; and no dividend yield. |
Income Taxes
Income Taxes | 9 Months Ended |
Sep. 30, 2019 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | Income Taxes The Company did not record a federal or state income tax provision or benefit for the three and nine months ended September 30, 2019 and 2018 as it has incurred net losses since inception. In addition, the net deferred tax assets generated from net operating losses have been fully reserved as the Company believes it is not more likely than not that the benefit will be realized. |
Leases
Leases | 9 Months Ended |
Sep. 30, 2019 | |
Leases [Abstract] | |
Leases | Leases Under Topic 842, operating lease expense is generally recognized evenly over the term of the lease. The Company has various non-cancelable lease agreements for our office and manufacturing spaces with lease periods expiring between 2023 and 2027. The Company’s lease terms may include options to extend or terminate the leases. The lease term represents the period up to the early termination date unless it is reasonably certain that the Company will not exercise the early termination option. For certain leases, the Company has options to extend the lease term for additional periods ranging from five to eight years . These renewal options are not considered in the remaining lease term unless it is reasonably certain that the Company will exercise such options. Certain leases include rental payments that are adjusted periodically based on changes in consumer price and other indexes. For the three and nine months ended September 30, 2019, the Company recorded operating lease costs of $1.5 million and $4.4 million , respectively. Under the terms of the lease agreements, the Company is also responsible for certain variable lease payments that are not included in the measurement of the lease liability. Variable lease costs for the three and nine months ended September 30, 2019 were $0.2 million and $0.7 million , respectively, primarily related to common area maintenance, taxes, utilities and insurance with the Company's operating leases. Cash paid for amounts included in the measurement of operating lease liabilities for the three and nine months ended September 30, 2019 were $1.6 million and $4.4 million , respectively. As of September 30, 2019 , maturities of lease liabilities for the following five fiscal years and thereafter were as follows (in thousands except lease term and discount rate): Amount Remainder of 2019 $ 1,627 2020 6,692 2021 6,901 2022 7,130 2023 5,847 Thereafter 13,582 Total lease payments 41,779 Less: Imputed interest (11,714 ) Tenant improvement not yet received (176 ) Present value of operating lease liabilities $ 29,889 Current operating lease liabilities $ 3,399 Operating lease liabilities - long-term $ 26,490 Weighted-average remaining lease term (in years) 6.1 Weighted-average discount rate 11.0 % As the Company elected to apply the provisions of Topic 842 on a prospective basis, the following comparative period disclosure is being presented in accordance with Topic 840. The future minimum commitments under the Company's leases as of December 31, 2018, were as follows: Amount (in thousands) 2019 $ 6,073 2020 6,692 2021 6,901 2022 7,130 2023 5,847 Thereafter 13,024 Total minimum lease payments $ 45,667 |
Net Loss per Share
Net Loss per Share | 9 Months Ended |
Sep. 30, 2019 | |
Earnings Per Share [Abstract] | |
Net Loss per Share | Net Loss per Share Basic net loss per share is calculated by dividing net loss by the weighted-average number of common shares outstanding during the period and excludes any potential dilutive effects of common stock equivalents. Diluted net loss per share is computed giving effect to all potential dilutive common shares, including common stock issuable upon exercise of stock options, convertible preferred stock, and unvested restricted common stock. As the Company had net losses for the three and nine months ended September 30, 2019 and 2018 , all potential common shares were determined to be anti-dilutive and were therefore excluded from the calculation of diluted net loss per share. The following table sets forth the computation of basic and diluted net loss per share of common stock during the three and nine months ended September 30, 2019 and 2018 : Three Months Ended September 30, Nine Months Ended September 30, 2019 2018 2019 2018 (in thousands, except per share data) Net loss $ (45,741 ) $ (36,291 ) $ (139,896 ) $ (93,229 ) Weighted-average number of shares used in computing net loss per share 45,543,354 37,359,877 44,538,676 36,302,803 Net loss per share, basic and diluted $ (1.00 ) $ (0.97 ) $ (3.14 ) $ (2.57 ) The following outstanding shares of common stock equivalents were excluded from the computation of diluted net loss per share of common stock for the periods presented because including them would have been anti-dilutive: Nine Months Ended September 30, 2019 2018 Stock options to purchase common stock 5,111,728 5,029,052 Restricted stock units 450,906 — 5,562,634 5,029,052 |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 9 Months Ended |
Sep. 30, 2019 | |
Accounting Policies [Abstract] | |
Leases | Leases The Company determines if an arrangement is or contains a lease at inception by assessing whether the arrangement contains an identified asset and whether it has the right to control the identified asset. Right-of-use (ROU) assets represent the Company's right to use an underlying asset for the lease term and lease liabilities represent the Company's obligation to make lease payments arising from the lease. ROU assets and lease liabilities are recognized at the lease commencement date based on the present value of future lease payments over the lease term. As the implicit rate in the Company's leases is unknown, the Company uses its incremental borrowing rate based on the information available at the lease commencement date in determining the present value of future lease payments. The Company gives consideration to its credit risk, term of the lease, total lease payments and adjust for the impacts of collateral, as necessary, when calculating its incremental borrowing rates. The ROU asset is based on the measurement of the lease liability and also includes any lease payments made prior to or on lease commencement and excludes lease incentives and initial direct costs incurred, as applicable. The lease terms may include options to extend or terminate the lease when it is reasonably certain the Company will exercise any such options. Rent expense for the Company's operating leases is recognized on a straight-line basis over the lease term. The Company has lease agreements with lease and non-lease components. The Company has elected to not separate lease and non-lease components for any leases within its existing classes of assets and, as a result, accounts for the lease and non-lease components as a single lease component. The Company has also elected to not apply the recognition requirement to any leases within its existing classes of assets with a term of 12 months or less. |
Basis of Presentation | Basis of Preparation The accompanying unaudited interim condensed consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States of America, or U.S. GAAP, and applicable rules and regulations of the Securities and Exchange Commission, or SEC, regarding interim financial reporting. As permitted under those rules, certain footnotes or other financial information that are normally required by U.S. GAAP have been condensed or omitted, and accordingly the balance sheet as of December 31, 2018 has been derived from audited financial statements at that date but does not include all of the information required by U.S. GAAP for complete financial statements. These unaudited interim condensed consolidated financial statements have been prepared on the same basis as the Company’s annual financial statements and, in the opinion of management, reflect all adjustments (consisting only of normal recurring adjustments) that are necessary for a fair presentation of the Company’s financial information. The results of operations for the three and nine months ended September 30, 2019 are not necessarily indicative of the results to be expected for the year ending December 31, 2019 or for any other interim period or for any other future year. The accompanying unaudited interim condensed consolidated financial statements and related financial information should be read in conjunction with the audited financial statements and the related notes thereto for the year ended December 31, 2018 included in the Company’s audited financial statements filed in its Annual Report on Form 10-K for the year ended December 31, 2018 . |
Use of Estimates | Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities, as of the date of the financial statements, and the reported amounts of any expenses during the reporting period. On an ongoing basis, management evaluates its estimates, including those related to accrued liabilities, acquisition contingent consideration, income taxes, and stock-based compensation. Management bases its estimates on historical experience, and on various other market-specific relevant assumptions that management believes to be reasonable, under the circumstances. Actual results may differ from those estimates. |
Concentration of Credit Risk | Concentration of Credit Risk Financial instruments that potentially subject the Company to a concentration of credit risk consist of cash and cash equivalents and marketable securities. The Company invests in a variety of financial instruments and in accordance with its investment policy, limits the amount of credit exposure with any one issuer, industry or geographic area for investments other than instruments backed by the U.S. federal government. |
Concentration of Manufacturing and Third-Party Services Risk | Concentration of Manufacturing and Third-Party Services Risk The Company is subject to certain risks with respect to sources of supply of manufactured materials and drug product for use in its preclinical studies and clinical trials. Due to the technical aspects of manufacturing drug product for gene therapies, there exist few alternative sources of manufacturing. The Company is reliant upon its own internal manufacturing capability and a small number of third-party vendors to produce drug product in sufficient quantities and quality to conduct its research and development activities. |
Accounting Pronouncements Adopted and Note Yet Adopted in 2019 | Accounting Pronouncements Not Yet Adopted In August 2018, the FASB issued ASU 2018-13, Fair Value Measurement (Topic 820) , which modifies, removes and adds certain disclosure requirements on fair value measurements based on the FASB Concepts Statement, Conceptual Framework for Financial Reporting—Chapter 8: Notes to Financial Statements. This guidance is effective for fiscal years, and interim periods within those fiscal years, beginning after December 15, 2019. The amendments on changes in unrealized gains and losses, the range and weighted average of significant unobservable inputs used to develop Level 3 fair value measurements and the narrative description of measurement uncertainty should be applied prospectively for only the most recent interim or annual period presented in the initial fiscal year of adoption. All other amendments should be applied retrospectively to all periods presented upon their effective date. Early adoption is permitted upon issuance of ASU 2018-13. An entity is permitted to early adopt any removed or modified disclosures upon issuance of ASU 2018-13 and delay adoption of the additional disclosures until their effective date. It is the Company’s expectation that adoption of this pronouncement will not have a material impact to its condensed consolidated financial statements. In January 2017, the FASB issued ASU 2017-04, Intangibles - Goodwill and Other (Topic 350): Simplifying the Test for Goodwill Impairment , which eliminates Step 2 from the goodwill impairment test. In addition, ASU 2017-04 eliminates the requirements for any reporting unit with a zero or negative carrying amount to perform a qualitative assessment and, if it fails that qualitative test, to perform Step 2 of the goodwill impairment test. ASU 2017-04 is effective for fiscal years beginning after December 15, 2019, with early adoption permitted. The Company is currently evaluating the impact that the standard will have on its consolidated financial statements and related disclosures. It is the Company’s expectation that adoption of this pronouncement will not have a material impact to its consolidated financial statements and related disclosures. Accounting Pronouncements Adopted in 2019 In August 2018, the Financial Accounting Standards Board, or FASB, issued ASU 2018-15, Intangibles - Goodwill and Other-Internal Use Software (Subtopic 350-40): Customer’s Accounting for Implementation Costs Incurred in a Cloud Computing Arrangement That is a Service Contract. This guidance requires companies to apply the internal-use software guidance in ASC 350-40 to implementation costs incurred in a hosting arrangement that is a service contract to determine whether to capitalize certain implementation costs or expense them as incurred. This guidance is effective for fiscal years, and interim periods within those fiscal years, beginning after December 15, 2019. Early adoption was permitted and the Company early adopted the standard on January 1, 2019. Adoption of the standard did not have a material impact on the Company's condensed consolidated financial statements. See Note 5 for further disclosure. In June 2018, the FASB issued ASU 2018-07, Compensation - Stock Compensation (Topic 718), Improvements to Nonemployee Share-Based Payment Accounting , which expands the scope of Topic 718 to include share-based payment transactions for acquiring goods and services from nonemployees. An entity should apply the requirements of Topic 718 to nonemployee awards except for specific guidance on inputs to an option pricing model and the attribution of cost. The measurement of equity-classified nonemployee awards is fixed at the grant date, and entities would measure the cost of awards subject to a performance condition using the outcome that is probable at the balance sheet date. The Company adopted this standard on January 1, 2019. As a result of adopting this standard, the Company no longer remeasures equity-classified nonemployee awards. The adoption of this new standard did not result in material impact on the Company's condensed consolidated financial statements. In February 2016, the FASB issued ASU No. 2016-02, Leases (Topic 842) , which establishes a comprehensive new lease accounting model. Under the new guidance, at the commencement date, lessees are required to recognize a lease liability with a corresponding right-of-use (ROU) asset. Effective January 1, 2019, the Company adopted Topic 842 using the modified retrospective approach provided by ASU 2018-11. Results for reporting periods beginning January 1, 2019 are presented under Topic 842, while prior period amounts were not adjusted and continue to be presented in accordance with the Company’s historical accounting under Topic 840, Leases . The Company elected certain practical expedients permitted under the transition guidance, including the election to carryforward historical lease classification. The Company also elected the short-term lease practical expedient, which allowed the Company to not recognize leases with a term of less than twelve months on its consolidated balance sheets. In addition, the Company elected the lease and non-lease components practical expedient, which allowed the Company to calculate the present value of the fixed payments without performing an allocation of lease and non-lease components. |
Schedule of New Accounting Pron
Schedule of New Accounting Pronouncements and Changes in Accounting Principles (Tables) | 9 Months Ended |
Sep. 30, 2019 | |
Accounting Policies [Abstract] | |
Schedule of New Accounting Pronouncements and Changes in Accounting Principles | The impact of the adoption of Topic 842 on the accompanying Condensed Consolidated Balance Sheet as of January 1, 2019 was as follows: December 31, 2018 Effect of Adoption January 1, 2019 (in thousands) Operating lease right-of-use assets $ — $ 21,669 $ 21,669 Liabilities: Operating leases $ — $ 2,541 $ 2,541 Deferred rent $ 456 $ (456 ) $ — Operating lease liabilities - long-term $ — $ 24,304 $ 24,304 Deferred rent and asset retirement obligation - long-term $ 4,935 $ (4,720 ) $ 215 |
Investments (Tables)
Investments (Tables) | 9 Months Ended |
Sep. 30, 2019 | |
Investments, Debt and Equity Securities [Abstract] | |
Schedule of Available-for-Sale Securities | Investments consist of available-for-sale marketable securities as follows (in thousands): September 30, 2019 Amortized Cost Unrealized Gains Unrealized Losses Fair Value (Unaudited) Money market funds $ 22,647 $ — $ — $ 22,647 Commercial paper 92,750 7 (3 ) 92,754 Corporate securities 111,201 178 — 111,379 U.S. treasury bills 10,760 2 — 10,762 U.S. government agency securities 61,367 35 (1 ) 61,401 U.S. agency bonds 37,579 8 — 37,587 Total available-for-sale securities $ 336,304 $ 230 $ (4 ) $ 336,530 December 31, 2018 Amortized Cost Unrealized Gains Unrealized Losses Fair Value Money market funds $ 33,399 $ — $ — $ 33,399 Commercial paper 144,578 — — 144,578 Corporate securities 82,670 8 (39 ) 82,639 U.S. treasury bills 60,573 — (8 ) 60,565 U.S. government agency securities 15,219 — — 15,219 U.S. agency bonds 51,411 — (22 ) 51,389 U.S. agency discount securities 18,716 — — 18,716 Total available-for-sale securities $ 406,566 $ 8 $ (69 ) $ 406,505 |
Classification of Available-for-sale Securities | The following table summarizes the classification of the available-for-sale securities on the Company's condensed consolidated balance sheets (in thousands): September 30, 2019 December 31, 2018 (Unaudited) Cash and cash equivalents $ 48,656 $ 136,547 Short-term investments 287,874 269,958 Total $ 336,530 $ 406,505 |
Fair Value Measurements (Tables
Fair Value Measurements (Tables) | 9 Months Ended |
Sep. 30, 2019 | |
Fair Value Disclosures [Abstract] | |
Fair Value of Financial Assets | Financial assets subject to fair value measurements on a recurring basis and the level of inputs used in such measurements are as follows (in thousands): September 30, 2019 Fair Value Measurements Using Total Level 1 Level 2 Level 3 (Unaudited) Money market funds $ 22,647 $ 22,647 $ — $ — Commercial paper 92,754 — 92,754 — Corporate securities 111,379 — 111,379 — U.S. treasury bills 10,762 — 10,762 — U.S. government agency securities 61,401 — 61,401 — U.S. agency bonds 37,587 — 37,587 — Total financial assets $ 336,530 $ 22,647 $ 313,883 $ — December 31, 2018 Fair Value Measurements Using Total Level 1 Level 2 Level 3 Money market funds $ 33,399 $ 33,399 $ — $ — Commercial paper 144,578 — 144,578 — Corporate securities 82,639 — 82,639 — U.S. treasury bills 60,565 — 60,565 — U.S. government agency securities 15,219 — 15,219 — U.S. agency bonds 51,389 — 51,389 — U.S. agency discount securities 18,716 — 18,716 — Total financial assets $ 406,505 $ 33,399 $ 373,106 $ — |
Summary of Contingent Acquisition Consideration Payable | The following is a summary of the contingent acquisition consideration payable recorded in the accompanying consolidated balance sheets (in thousands): Amount (Unaudited) Balance, December 31, 2018 $ 2,345 Change in fair value of contingent acquisition consideration payable 115 Balance, September 30, 2019 $ 2,460 |
Balance Sheet Components (Table
Balance Sheet Components (Tables) | 9 Months Ended |
Sep. 30, 2019 | |
Property, Plant and Equipment [Abstract] | |
Property and Equipment, Net | Property and equipment, net, consist of the following (in thousands): September 30, 2019 December 31, 2018 (Unaudited) Furniture and office equipment $ 2,439 $ 1,898 Computer equipment 1,391 1,019 Software 526 513 Leasehold improvements 23,453 19,607 Laboratory equipment 11,694 9,570 Manufacturing equipment 8,129 6,619 Construction in progress and deposits on equipment 6,640 3,320 Total property and equipment 54,272 42,546 Less accumulated depreciation and amortization (16,107 ) (10,447 ) Property and equipment, net $ 38,165 $ 32,099 |
Accrued Liabilities | Accrued liabilities consist of the following (in thousands): September 30, 2019 December 31, 2018 (Unaudited) Accrued payroll and related expenses $ 8,310 $ 8,581 Accrued research and development expenses 4,692 3,317 Accrued construction in progress 74 71 Accrued general and administrative expenses 1,474 959 Total accrued liabilities $ 14,550 $ 12,928 |
Stock Compensation (Tables)
Stock Compensation (Tables) | 9 Months Ended |
Sep. 30, 2019 | |
Share-based Payment Arrangement [Abstract] | |
Stock-based Compensation Expense by Category | Stock-based compensation expense by category was as follows for the three and nine months ended September 30, 2019 and 2018 (in thousands): Three Months Ended September 30, Nine Months Ended September 30, 2019 2018 2019 2018 (Unaudited) Research and development $ 3,048 $ 2,611 $ 9,465 $ 6,930 General and administrative 2,664 2,022 7,878 5,185 Total stock-based compensation expense $ 5,712 $ 4,633 $ 17,343 $ 12,115 |
Summary of Option Activity | The following table summarizes option activity for the nine months ended September 30, 2019 : Number of Options Outstanding Weighted- Average Exercise Price Per Option Balance, December 31, 2018 4,894,201 $ 18.79 Options granted 1,581,183 $ 28.04 Options exercised (698,790 ) $ 12.35 Options forfeited (664,866 ) $ 24.29 Balance, September 30, 2019 5,111,728 $ 21.82 |
Fair Value of Stock Options, Valuation Assumptions | The fair value of stock options granted to employees was estimated using a Black-Scholes option pricing model with the following assumptions: Three Months Ended September 30, Nine Months Ended September 30, 2019 2018 2019 2018 Expected term (in years) 6.0-6.1 6.1 5.5-6.1 5.5-6.1 Expected volatility 62-63% 70 % 62-68% 70-76% Risk-free interest rate 1.4-1.9% 2.8-3.0% 1.4-2.6% 2.3-3.0% Expected dividend yield — % — % — % — % |
Schedule of RSU Activity | The following table summarizes activity of RSUs granted to employees with service-based vesting during the nine months ended September 30, 2019: Number of RSUs Outstanding Weighted- Average Grant Date Fair Value Per RSU Balance, December 31, 2018 — RSUs granted 571,530 $ 28.59 RSUs vested (58,156 ) $ 24.89 RSUs forfeited (62,468 ) $ 24.94 Balance, September 30, 2019 450,906 $ 29.57 |
Leases (Tables)
Leases (Tables) | 9 Months Ended |
Sep. 30, 2019 | |
Leases [Abstract] | |
Future Minimum Lease Payments - Topic 842 | As of September 30, 2019 , maturities of lease liabilities for the following five fiscal years and thereafter were as follows (in thousands except lease term and discount rate): Amount Remainder of 2019 $ 1,627 2020 6,692 2021 6,901 2022 7,130 2023 5,847 Thereafter 13,582 Total lease payments 41,779 Less: Imputed interest (11,714 ) Tenant improvement not yet received (176 ) Present value of operating lease liabilities $ 29,889 Current operating lease liabilities $ 3,399 Operating lease liabilities - long-term $ 26,490 Weighted-average remaining lease term (in years) 6.1 Weighted-average discount rate 11.0 % |
Future Minimum Lease Payments - Topic 840 | As the Company elected to apply the provisions of Topic 842 on a prospective basis, the following comparative period disclosure is being presented in accordance with Topic 840. The future minimum commitments under the Company's leases as of December 31, 2018, were as follows: Amount (in thousands) 2019 $ 6,073 2020 6,692 2021 6,901 2022 7,130 2023 5,847 Thereafter 13,024 Total minimum lease payments $ 45,667 |
Net Loss per Share (Tables)
Net Loss per Share (Tables) | 9 Months Ended |
Sep. 30, 2019 | |
Earnings Per Share [Abstract] | |
Computation of Basic and Diluted Net Loss per Share of Common Stock | The following table sets forth the computation of basic and diluted net loss per share of common stock during the three and nine months ended September 30, 2019 and 2018 : Three Months Ended September 30, Nine Months Ended September 30, 2019 2018 2019 2018 (in thousands, except per share data) Net loss $ (45,741 ) $ (36,291 ) $ (139,896 ) $ (93,229 ) Weighted-average number of shares used in computing net loss per share 45,543,354 37,359,877 44,538,676 36,302,803 Net loss per share, basic and diluted $ (1.00 ) $ (0.97 ) $ (3.14 ) $ (2.57 ) |
Anti-Dilutive Common Stock Equivalents Excluded from Computation of Diluted Net Loss per Share | The following outstanding shares of common stock equivalents were excluded from the computation of diluted net loss per share of common stock for the periods presented because including them would have been anti-dilutive: Nine Months Ended September 30, 2019 2018 Stock options to purchase common stock 5,111,728 5,029,052 Restricted stock units 450,906 — 5,562,634 5,029,052 |
Organization and Basis of Pre_2
Organization and Basis of Presentation (Details) | 1 Months Ended | 9 Months Ended | |
Mar. 31, 2018USD ($) | Sep. 30, 2019USD ($)segmentshares | Dec. 31, 2018USD ($) | |
Subsidiary, Sale of Stock [Line Items] | |||
Number of operating business segment | segment | 1 | ||
Accumulated deficit | $ 459,366,000 | $ 319,470,000 | |
Cash, cash equivalents and marketable securities | 351,500,000 | ||
Cash and cash equivalents | 63,590,000 | $ 144,349,000 | |
Marketable securities | 287,900,000 | ||
At-the-Market | |||
Subsidiary, Sale of Stock [Line Items] | |||
Common stock available for sale | $ 150,000,000 | $ 78,600,000 | |
Sale of stock, commission percentage | 3.00% | ||
Sale of stock, number of shares sold (in shares) | shares | 1,421,151 | ||
Net proceeds from sale of stock | $ 54,400,000 |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies Adjustment for Adoption (Details) - USD ($) $ in Thousands | Sep. 30, 2019 | Jan. 01, 2019 | Dec. 31, 2018 |
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||
Right-of-use assets | $ 24,859 | $ 21,669 | |
Liabilities: | |||
Operating lease liabilities | 3,399 | 2,541 | |
Deferred rent | $ 456 | ||
Operating lease liabilities - long-term | 26,490 | 24,304 | |
Deferred rent and asset retirement obligation - long-term | 215 | $ 4,935 | |
Operating lease liability | $ 29,889 | ||
Accounting Standards Update 2016-02 | |||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||
Right-of-use assets | 21,669 | ||
Liabilities: | |||
Operating lease liabilities | 2,541 | ||
Deferred rent | (456) | ||
Operating lease liabilities - long-term | 24,304 | ||
Deferred rent and asset retirement obligation - long-term | $ (4,720) |
Investments - Schedule of Avail
Investments - Schedule of Available-for-Sale Securities (Details) - USD ($) $ in Thousands | Sep. 30, 2019 | Dec. 31, 2018 |
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | $ 336,304 | $ 406,566 |
Unrealized Gains | 230 | 8 |
Unrealized Losses | (4) | (69) |
Fair Value | 336,530 | 406,505 |
Money market funds | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 22,647 | 33,399 |
Unrealized Gains | 0 | 0 |
Unrealized Losses | 0 | 0 |
Fair Value | 22,647 | 33,399 |
Commercial paper | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 92,750 | 144,578 |
Unrealized Gains | 7 | 0 |
Unrealized Losses | (3) | 0 |
Fair Value | 92,754 | 144,578 |
Corporate securities | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 111,201 | 82,670 |
Unrealized Gains | 178 | 8 |
Unrealized Losses | 0 | (39) |
Fair Value | 111,379 | 82,639 |
U.S. treasury bills | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 10,760 | 60,573 |
Unrealized Gains | 2 | 0 |
Unrealized Losses | 0 | (8) |
Fair Value | 10,762 | 60,565 |
U.S. government agency securities | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 61,367 | 15,219 |
Unrealized Gains | 35 | 0 |
Unrealized Losses | (1) | 0 |
Fair Value | 61,401 | 15,219 |
U.S. agency bonds | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 37,579 | 51,411 |
Unrealized Gains | 8 | 0 |
Unrealized Losses | 0 | (22) |
Fair Value | $ 37,587 | 51,389 |
U.S. agency discount securities | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 18,716 | |
Unrealized Gains | 0 | |
Unrealized Losses | 0 | |
Fair Value | $ 18,716 |
Investments - Classification of
Investments - Classification of Available-for-sale Investments (Details) - USD ($) $ in Thousands | Sep. 30, 2019 | Dec. 31, 2018 |
Debt Securities, Available-for-sale [Line Items] | ||
Available-for-sale securities | $ 336,530 | $ 406,505 |
Cash and cash equivalents | ||
Debt Securities, Available-for-sale [Line Items] | ||
Available-for-sale securities | 48,656 | 136,547 |
Short-term investments | ||
Debt Securities, Available-for-sale [Line Items] | ||
Available-for-sale securities | $ 287,874 | $ 269,958 |
Fair Value Measurements - Fair
Fair Value Measurements - Fair Value of Financial Assets (Details) - Recurring - USD ($) $ in Thousands | Sep. 30, 2019 | Dec. 31, 2018 |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | $ 336,530 | $ 406,505 |
Fair Value Measurements Using Level 1 | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | 22,647 | 33,399 |
Fair Value Measurements Using Level 2 | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | 313,883 | 373,106 |
Fair Value Measurements Using Level 3 | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | 0 | 0 |
Money market funds | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | 22,647 | 33,399 |
Money market funds | Fair Value Measurements Using Level 1 | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | 22,647 | 33,399 |
Money market funds | Fair Value Measurements Using Level 2 | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | 0 | 0 |
Money market funds | Fair Value Measurements Using Level 3 | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | 0 | 0 |
Commercial paper | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | 92,754 | 144,578 |
Commercial paper | Fair Value Measurements Using Level 1 | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | 0 | 0 |
Commercial paper | Fair Value Measurements Using Level 2 | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | 92,754 | 144,578 |
Commercial paper | Fair Value Measurements Using Level 3 | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | 0 | 0 |
Corporate securities | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | 111,379 | 82,639 |
Corporate securities | Fair Value Measurements Using Level 1 | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | 0 | 0 |
Corporate securities | Fair Value Measurements Using Level 2 | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | 111,379 | 82,639 |
Corporate securities | Fair Value Measurements Using Level 3 | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | 0 | 0 |
U.S. treasury bills | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | 10,762 | 60,565 |
U.S. treasury bills | Fair Value Measurements Using Level 1 | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | 0 | 0 |
U.S. treasury bills | Fair Value Measurements Using Level 2 | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | 10,762 | 60,565 |
U.S. treasury bills | Fair Value Measurements Using Level 3 | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | 0 | 0 |
U.S. government agency securities | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | 61,401 | 15,219 |
U.S. government agency securities | Fair Value Measurements Using Level 1 | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | 0 | 0 |
U.S. government agency securities | Fair Value Measurements Using Level 2 | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | 61,401 | 15,219 |
U.S. government agency securities | Fair Value Measurements Using Level 3 | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | 0 | 0 |
U.S. agency bonds | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | 37,587 | 51,389 |
U.S. agency bonds | Fair Value Measurements Using Level 1 | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | 0 | 0 |
U.S. agency bonds | Fair Value Measurements Using Level 2 | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | 37,587 | 51,389 |
U.S. agency bonds | Fair Value Measurements Using Level 3 | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | $ 0 | 0 |
U.S. agency discount securities | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | 18,716 | |
U.S. agency discount securities | Fair Value Measurements Using Level 1 | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | 0 | |
U.S. agency discount securities | Fair Value Measurements Using Level 2 | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | 18,716 | |
U.S. agency discount securities | Fair Value Measurements Using Level 3 | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Financial assets | $ 0 |
Fair Value Measurements - Addit
Fair Value Measurements - Additional Information (Details) - USD ($) $ in Thousands | Sep. 30, 2019 | Dec. 31, 2018 | Aug. 31, 2015 |
Business Acquisition, Contingent Consideration [Line Items] | |||
Cash held in primary operating bank accounts | $ 14,900 | $ 7,800 | |
Contingent consideration | $ 2,460 | $ 2,345 | $ 10,000 |
Common Stock | |||
Business Acquisition, Contingent Consideration [Line Items] | |||
Contingent consideration | 4,200 | ||
Cash or Common Stock | |||
Business Acquisition, Contingent Consideration [Line Items] | |||
Contingent consideration | $ 5,800 |
Fair Value Measurements - Summa
Fair Value Measurements - Summary of Contingent Acquisition Consideration Payable Recorded in Accompanying Consolidated Balance Sheets (Details) - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2019 | Sep. 30, 2018 | |
Contingent Consideration [Roll Forward] | ||
Beginning balance | $ 2,345 | |
Change in fair value of contingent acquisition consideration payable | 115 | $ (2,327) |
Ending balance | $ 2,460 |
Balance Sheet Components - Prop
Balance Sheet Components - Property and Equipment, Net (Details) - USD ($) $ in Thousands | Sep. 30, 2019 | Dec. 31, 2018 |
Property, Plant and Equipment [Line Items] | ||
Property and equipment | $ 54,272 | $ 42,546 |
Less accumulated depreciation and amortization | (16,107) | (10,447) |
Property and equipment, net | 38,165 | 32,099 |
Furniture and office equipment | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment | 2,439 | 1,898 |
Computer equipment | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment | 1,391 | 1,019 |
Software | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment | 526 | 513 |
Leasehold improvements | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment | 23,453 | 19,607 |
Laboratory equipment | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment | 11,694 | 9,570 |
Manufacturing equipment | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment | 8,129 | 6,619 |
Construction in progress and deposits on equipment | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment | $ 6,640 | $ 3,320 |
Balance Sheet Components - Addi
Balance Sheet Components - Additional Information (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2019 | Sep. 30, 2018 | Sep. 30, 2019 | Sep. 30, 2018 | |
Capitalized Contract Cost [Line Items] | ||||
Depreciation and amortization expense | $ 2,000 | $ 1,500 | $ 5,732 | $ 3,948 |
Hosting arrangement, implementation costs capitalized | $ 2,500 | 2,500 | ||
Hosting arrangement, service contract, term | 5 years | |||
Hosting arrangement, implementation costs amortization | $ 100 | 100 | ||
Current assets | ||||
Capitalized Contract Cost [Line Items] | ||||
Hosting arrangement, implementation costs capitalized, net | 500 | 500 | ||
Noncurrent assets | ||||
Capitalized Contract Cost [Line Items] | ||||
Hosting arrangement, implementation costs capitalized, net | $ 1,900 | $ 1,900 |
Balance Sheet Components - Accr
Balance Sheet Components - Accrued Liabilities (Details) - USD ($) $ in Thousands | Sep. 30, 2019 | Dec. 31, 2018 |
Property, Plant and Equipment [Abstract] | ||
Accrued payroll and related expenses | $ 8,310 | $ 8,581 |
Accrued research and development expenses | 4,692 | 3,317 |
Accrued construction in progress | 74 | 71 |
Accrued general and administrative expenses | 1,474 | 959 |
Total accrued liabilities | $ 14,550 | $ 12,928 |
Commitments and Contingencies -
Commitments and Contingencies - Additional Information (Details) - USD ($) $ in Millions | 1 Months Ended | |
Feb. 28, 2019 | Sep. 30, 2019 | |
Commitments and Contingencies Disclosure [Abstract] | ||
Upfront fee consideration for licensed rights | $ 7 | |
Contingent payments (up to) | $ 270.9 | |
Contingent payments, Crigler-Najjar and CASQ2-CPVT Programs | $ 74 |
Stock Compensation - Stock-base
Stock Compensation - Stock-based Compensation Expense by Category (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2019 | Sep. 30, 2018 | Sep. 30, 2019 | Sep. 30, 2018 | |
Share-based Payment Arrangement, Expensed and Capitalized, Amount [Line Items] | ||||
Stock-based compensation expense | $ 5,712 | $ 4,633 | $ 17,343 | $ 12,115 |
Research and development | ||||
Share-based Payment Arrangement, Expensed and Capitalized, Amount [Line Items] | ||||
Stock-based compensation expense | 3,048 | 2,611 | 9,465 | 6,930 |
General and administrative | ||||
Share-based Payment Arrangement, Expensed and Capitalized, Amount [Line Items] | ||||
Stock-based compensation expense | $ 2,664 | $ 2,022 | $ 7,878 | $ 5,185 |
Stock Compensation - Summary of
Stock Compensation - Summary of Option Activity (Details) | 9 Months Ended |
Sep. 30, 2019$ / sharesshares | |
Number of Options Outstanding | |
Beginning Balance (in shares) | shares | 4,894,201 |
Options granted (in shares) | shares | 1,581,183 |
Options exercised (in shares) | shares | (698,790) |
Options forfeited (in shares) | shares | (664,866) |
Ending Balance (in shares) | shares | 5,111,728 |
Weighted- Average Exercise Price Per Option | |
Beginning Balance (in USD per share) | $ / shares | $ 18.79 |
Options granted (in USD per share) | $ / shares | 28.04 |
Options exercised (in USD per share) | $ / shares | 12.35 |
Options forfeited (in USD per share) | $ / shares | 24.29 |
Ending Balance (in USD per share) | $ / shares | $ 21.82 |
Stock Compensation - Fair Value
Stock Compensation - Fair Value of Stock Options, Valuation Assumptions (Details) - Employee Stock Option | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2019 | Sep. 30, 2018 | Sep. 30, 2019 | Sep. 30, 2018 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Expected term | 6 years 1 month 6 days | |||
Expected volatility | 70.00% | |||
Expected volatility, minimum | 62.00% | 62.00% | 70.00% | |
Expected volatility, maximum | 63.00% | 68.00% | 76.00% | |
Risk-free interest rate, minimum | 1.40% | 2.80% | 1.40% | 2.30% |
Risk-free interest rate, maximum | 1.90% | 3.00% | 2.60% | 3.00% |
Expected dividend yield | 0.00% | 0.00% | 0.00% | 0.00% |
Minimum | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Expected term | 6 years | 5 years 6 months | 5 years 6 months | |
Maximum | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Expected term | 6 years 1 month 6 days | 6 years 1 month 6 days | 6 years 1 month 6 days |
Stock Compensation - RSU Activi
Stock Compensation - RSU Activity (Details) - Restricted stock units | 9 Months Ended |
Sep. 30, 2019$ / sharesshares | |
Number of RSUs Outstanding | |
Beginning balance (in shares) | 0 |
RSUs granted (in shares) | 571,530 |
RSUs vested (in shares) | (58,156) |
RSUs forfeited (in shares) | (62,468) |
Ending balance (in shares) | 450,906 |
Share-based Compensation Arrangement by Share-based Payment Award, Equity Instruments Other than Options, Nonvested, Weighted Average Grant Date Fair Value [Abstract] | |
RSUs granted (in USD per share) | $ / shares | $ 28.59 |
RSUs vested (in USD per share) | $ / shares | 24.89 |
RSUs forfeited (in USD per share) | $ / shares | 24.94 |
Ending balance (in USD per share) | $ / shares | $ 29.57 |
Stock Compensation - Additional
Stock Compensation - Additional Information (Details) - 2016 Employee Stock Purchase Plan - Employee Stock Purchase Plan - shares | May 01, 2018 | Sep. 30, 2019 | Sep. 30, 2019 |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Shares reserved for issuance (in shares) | 210,000 | ||
Proportion of outstanding shares of common stock | 1.00% | ||
Purchase of common stock through payroll deductions expressed in percentage of fair market value | 85.00% | ||
Common stock offering period | 6 months | ||
Maximum percentage of an employee's eligible compensation | 15.00% | ||
Fair values of rights granted, expected term | 6 months | 6 months | |
Expected volatility | 54.60% | ||
Risk-free interest rate | 2.40% | ||
Expected volatility, minimum | 54.60% | ||
Expected volatility, maximum | 62.20% | ||
Risk-free interest rate, minimum | 2.40% | ||
Risk-free interest rate, maximum | 2.50% | ||
Fair values of rights granted, expected dividend yield | 0.00% | 0.00% |
Income taxes - Additional Infor
Income taxes - Additional Information (Details) - USD ($) | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2019 | Sep. 30, 2018 | Sep. 30, 2019 | Sep. 30, 2018 | |
Income Tax Disclosure [Abstract] | ||||
Federal income tax provision (benefit) | $ 0 | $ 0 | $ 0 | $ 0 |
State income tax provision (benefit) | $ 0 | $ 0 | $ 0 | $ 0 |
Leases - Lease Cost (Details)
Leases - Lease Cost (Details) $ in Millions | 3 Months Ended | 9 Months Ended |
Sep. 30, 2019USD ($) | Sep. 30, 2019USD ($) | |
Lessee, Lease, Description [Line Items] | ||
Total operating lease cost | $ 1.5 | $ 4.4 |
Variable lease cost | 0.2 | 0.7 |
Cash payments for operating lease liabilities | $ 1.6 | $ 4.4 |
Minimum | ||
Lessee, Lease, Description [Line Items] | ||
Lessee, lease, renewal term | 5 years | 5 years |
Maximum | ||
Lessee, Lease, Description [Line Items] | ||
Lessee, lease, renewal term | 8 years | 8 years |
Leases - Maturities of Lease Li
Leases - Maturities of Lease Liabilities (Details) - USD ($) $ in Thousands | Sep. 30, 2019 | Jan. 01, 2019 | Dec. 31, 2018 |
Future Minimum Lease Payments - Topic 842 | |||
Remainder of 2019 | $ 1,627 | ||
2020 | 6,692 | ||
2021 | 6,901 | ||
2022 | 7,130 | ||
2023 | 5,847 | ||
Thereafter | 13,582 | ||
Total lease payments | 41,779 | ||
Imputed interest | (11,714) | ||
Tenant improvement not yet received | (176) | ||
Present value of operating lease liabilities | 29,889 | ||
Current operating lease liabilities | 3,399 | $ 2,541 | |
Operating lease liabilities - long-term | $ 26,490 | $ 24,304 | |
Weighted-average remaining lease term (in years) | 6 years 1 month 6 days | ||
Weighted-average discount rate | 11.00% | ||
Future Minimum Lease Payments - Topic 840 | |||
2019 | $ 6,073 | ||
2020 | 6,692 | ||
2021 | 6,901 | ||
2022 | 7,130 | ||
2023 | 5,847 | ||
Thereafter | 13,024 | ||
Total minimum lease payments | $ 45,667 |
Net Loss per Share - Computatio
Net Loss per Share - Computation of Basic and Diluted Net Loss Per Share of Common Stock (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 9 Months Ended | ||||||
Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Sep. 30, 2019 | Sep. 30, 2018 | |
Earnings Per Share [Abstract] | ||||||||
Net loss | $ (45,741) | $ (44,764) | $ (49,391) | $ (36,291) | $ (31,367) | $ (25,571) | $ (139,896) | $ (93,229) |
Weighted-average number of shares used in computing net loss per share (in shares) | 45,543,354 | 37,359,877 | 44,538,676 | 36,302,803 | ||||
Net loss per share, basic and diluted (USD per share) | $ (1) | $ (0.97) | $ (3.14) | $ (2.57) |
Net Loss per Share - Anti-Dilut
Net Loss per Share - Anti-Dilutive Common Stock Equivalents Excluded from Computation of Diluted Net Loss Per Share (Details) - shares | 9 Months Ended | |
Sep. 30, 2019 | Sep. 30, 2018 | |
Earnings Per Share, Basic, by Common Class, Including Two Class Method [Line Items] | ||
Anti-dilutive common stock equivalents excluded from computation of diluted net loss (in shares) | 5,562,634 | 5,029,052 |
Stock options to purchase common stock | ||
Earnings Per Share, Basic, by Common Class, Including Two Class Method [Line Items] | ||
Anti-dilutive common stock equivalents excluded from computation of diluted net loss (in shares) | 5,111,728 | 5,029,052 |
Restricted stock units | ||
Earnings Per Share, Basic, by Common Class, Including Two Class Method [Line Items] | ||
Anti-dilutive common stock equivalents excluded from computation of diluted net loss (in shares) | 450,906 | 0 |