Document and Entity Information
Document and Entity Information - USD ($) | 12 Months Ended | ||
Dec. 31, 2019 | Feb. 24, 2020 | Jun. 30, 2019 | |
Cover [Abstract] | |||
Document Type | 10-K | ||
Amendment Flag | false | ||
Document Period End Date | Dec. 31, 2019 | ||
Document Fiscal Year Focus | 2019 | ||
Document Fiscal Period Focus | FY | ||
Trading Symbol | MYOK | ||
Entity Registrant Name | MYOKARDIA, INC. | ||
Entity Central Index Key | 0001552451 | ||
Current Fiscal Year End Date | --12-31 | ||
Entity Well-known Seasoned Issuer | Yes | ||
Entity Current Reporting Status | Yes | ||
Entity Voluntary Filers | No | ||
Entity Filer Category | Large Accelerated Filer | ||
Entity Small Business | false | ||
Entity Emerging Growth Company | false | ||
Entity Shell Company | false | ||
Title of 12(b) Security | Common Stock | ||
Entity Common Stock, Shares Outstanding | 46,574,538 | ||
Entity Public Float | $ 2,271,068,236 | ||
Entity File Number | 001-37609 | ||
Entity Incorporation, State or Country Code | DE | ||
Entity Tax Identification Number | 44-5500552 | ||
Entity Interactive Data Current | Yes | ||
Security Exchange Name | NASDAQ | ||
Entity Address, Address Line One | 1000 Sierra Point Parkway | ||
Entity Address, City or Town | Brisbane | ||
Entity Address, State or Province | CA | ||
Entity Address, Postal Zip Code | 94005 | ||
City Area Code | 650 | ||
Local Phone Number | 741-0900 | ||
Document Annual Report | true | ||
Document Transition Report | false | ||
Documents Incorporated by Reference | DOCUMENTS INCORPORATED BY REFERENCE Part III of this Annual Report on Form 10-K incorporates information by reference to portions of the definitive proxy statement for the registrant’s 2020 Annual Meeting of Stockholders, to be filed within 120 days of the registrant’s fiscal year ended December 31, 2019. |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Current assets | ||
Cash and cash equivalents | $ 101,436 | $ 246,122 |
Short-term investments | 314,691 | 68,564 |
Prepaid expenses and other current assets | 7,709 | 4,760 |
Total current assets | 423,836 | 319,446 |
Property and equipment, net | 15,743 | 5,138 |
Operating lease right-of-use assets | 417 | |
Long-term investments | 14,153 | 80,148 |
Restricted cash and other | 1,945 | 2,521 |
Total assets | 456,094 | 407,253 |
Current liabilities | ||
Accounts payable | 6,237 | 2,946 |
Accrued liabilities | 41,292 | 20,758 |
Prepayment from collaboration partner | 12,973 | |
Operating lease liabilities - current | 383 | |
Total current liabilities | 47,912 | 36,677 |
Other long-term liabilities | 1,908 | 9 |
Total liabilities | 49,820 | 36,686 |
Commitments and contingencies (Note 7) | ||
Stockholders’ equity | ||
Preferred stock, $0.0001 par value; 5,000,000 shares authorized; none issued and outstanding | 0 | 0 |
Common stock, $0.0001 par value, 150,000,000 shares authorized at December 31, 2019 and 2018; 46,379,073 and 40,288,949 shares issued and outstanding at December 31, 2019 and 2018, respectively | 5 | 4 |
Additional paid-in capital | 884,486 | 573,183 |
Accumulated other comprehensive income (loss) | 549 | (67) |
Accumulated deficit | (478,766) | (202,553) |
Total stockholders’ equity | 406,274 | 370,567 |
Total liabilities and stockholders’ equity | $ 456,094 | $ 407,253 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - $ / shares | Dec. 31, 2019 | Dec. 31, 2018 |
Statement Of Financial Position [Abstract] | ||
Preferred stock, par value | $ 0.0001 | $ 0.0001 |
Preferred stock, shares authorized | 5,000,000 | 5,000,000 |
Preferred stock, shares issued | 0 | 0 |
Preferred stock, shares outstanding | 0 | 0 |
Common stock, par value | $ 0.0001 | $ 0.0001 |
Common stock, shares authorized | 150,000,000 | 150,000,000 |
Common stock, shares issued | 46,379,073 | 40,288,949 |
Common stock, shares outstanding | 46,379,073 | 40,288,949 |
Consolidated Statements of Oper
Consolidated Statements of Operations and Comprehensive Loss - USD ($) | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Income Statement [Abstract] | |||
Collaboration and license revenue | $ 0 | $ 33,558,000 | $ 11,442,000 |
Operating expenses: | |||
Repurchase of royalty rights | 80,000,000 | ||
Research and development | 146,171,000 | 68,774,000 | 48,136,000 |
Selling, general and administrative | 61,663,000 | 38,435,000 | 21,973,000 |
Total operating expenses | 287,834,000 | 107,209,000 | 70,109,000 |
Loss from operations | (287,834,000) | (73,651,000) | (58,667,000) |
Interest and other income, net | 11,621,000 | 5,953,000 | 1,657,000 |
Net loss | (276,213,000) | (67,698,000) | (57,010,000) |
Other comprehensive income (loss) | 616,000 | 125,000 | (200,000) |
Comprehensive loss | $ (275,597,000) | $ (67,573,000) | $ (57,210,000) |
Net loss per share, basic and diluted | $ (6.17) | $ (1.76) | $ (1.74) |
Weighted average number of shares used to compute net loss per share, basic and diluted | 44,765,496 | 38,386,906 | 32,832,514 |
Consolidated Statements of Stoc
Consolidated Statements of Stockholders' Equity - USD ($) $ in Thousands | Total | Common Stock | Additional Paid-In Capital | Accumulated Other Comprehensive Income/ (Loss) | Accumulated Deficit |
Beginning balance at Dec. 31, 2016 | $ 145,382 | $ 3 | $ 223,208 | $ 8 | $ (77,837) |
Beginning balance, shares at Dec. 31, 2016 | 31,428,998 | ||||
Issuance of common stock during offering, net of issuance costs, value | 133,862 | $ 1 | 133,861 | ||
Issuance of common stock during offering, net of issuance costs, shares | 4,025,000 | ||||
Issuance of common stock under equity incentive and employee stock purchase plans, value | 2,365 | 2,365 | |||
Issuance of common stock under equity incentive and employee stock purchase plans, shares | 400,754 | ||||
Repurchase of early exercised stock options, value | (45) | (45) | |||
Repurchase of early exercised stock options, shares | (41,961) | ||||
Vesting of early exercised stock options and restricted stock | 184 | 184 | |||
Stock-based compensation | 6,138 | 6,138 | |||
Cumulative effect adjustment upon adoption | ASU 2016-09 | 8 | (8) | |||
Unrealized gains/(losses) | (200) | (200) | |||
Net loss | (57,010) | (57,010) | |||
Ending balance at Dec. 31, 2017 | 230,676 | $ 4 | 365,719 | (192) | (134,855) |
Ending balance, shares at Dec. 31, 2017 | 35,812,791 | ||||
Issuance of common stock during offering, net of issuance costs, value | 181,863 | 181,863 | |||
Issuance of common stock during offering, net of issuance costs, shares | 3,961,147 | ||||
Issuance of common stock under equity incentive and employee stock purchase plans, value | 6,208 | 6,208 | |||
Issuance of common stock under equity incentive and employee stock purchase plans, shares | 516,008 | ||||
Repurchase of early exercised stock options, value | (1) | (1) | |||
Repurchase of early exercised stock options, shares | (997) | ||||
Vesting of early exercised stock options and restricted stock | 53 | 53 | |||
Stock-based compensation | 19,341 | 19,341 | |||
Unrealized gains/(losses) | 125 | 125 | |||
Net loss | (67,698) | (67,698) | |||
Ending balance at Dec. 31, 2018 | 370,567 | $ 4 | 573,183 | (67) | (202,553) |
Ending balance, shares at Dec. 31, 2018 | 40,288,949 | ||||
Issuance of common stock during offering, net of issuance costs, value | 271,224 | $ 1 | 271,223 | ||
Issuance of common stock during offering, net of issuance costs, shares | 5,663,750 | ||||
Issuance of common stock under equity incentive and employee stock purchase plans, value | 7,071 | 7,071 | |||
Issuance of common stock under equity incentive and employee stock purchase plans, shares | 426,374 | ||||
Vesting of early exercised stock options and restricted stock | 15 | 15 | |||
Stock-based compensation | 32,994 | 32,994 | |||
Unrealized gains/(losses) | 616 | 616 | |||
Net loss | (276,213) | (276,213) | |||
Ending balance at Dec. 31, 2019 | $ 406,274 | $ 5 | $ 884,486 | $ 549 | $ (478,766) |
Ending balance, shares at Dec. 31, 2019 | 46,379,073 |
Consolidated Statements of St_2
Consolidated Statements of Stockholders' Equity (Parenthetical) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Common Stock | |||
Issuance costs | $ 17,638 | $ 12,233 | $ 9,025 |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Cash flow from operating activities: | |||
Net loss | $ (276,213) | $ (67,698) | $ (57,010) |
Adjustments to reconcile net loss to net cash used in operating activities: | |||
Stock-based compensation expense | 32,994 | 19,341 | 6,138 |
Depreciation | 1,948 | 1,567 | 1,294 |
Amortization of discount on investments | (1,619) | (279) | 85 |
Loss on disposal of equipment | 4 | ||
Change in operating assets and liabilities: | |||
Receivable from collaboration partner | 1,013 | 43,987 | |
Prepaid expenses and other current assets | (2,612) | (2,884) | (482) |
Operating lease right-of-use assets | 2,618 | ||
Other long-term assets | 290 | (296) | (59) |
Accounts payable | 3,064 | 671 | 367 |
Accrued liabilities | 11,665 | 8,945 | 2,968 |
Prepayment from collaboration partner | (12,973) | 8,541 | 4,432 |
Operating lease liabilities | (2,838) | ||
Other long-term liabilities | 1,907 | (178) | (120) |
Deferred revenue | (33,558) | (11,442) | |
Net cash used in operating activities | (241,769) | (64,815) | (9,838) |
Cash flow from investing activities: | |||
Purchases of investments | (259,897) | (132,475) | (44,044) |
Sales of investments | 4,000 | 8,000 | 4,000 |
Maturities of investments | 78,000 | 28,000 | 4,000 |
Purchases of property and equipment | (3,264) | (3,373) | (1,517) |
Proceeds from sale of equipment | 10 | ||
Net cash used in investing activities | (181,161) | (99,848) | (37,551) |
Cash flow from financing activities: | |||
Proceeds from issuance of common stock in follow-on offerings, net of issuance and financing costs | 271,224 | 181,863 | 133,862 |
Proceeds from exercise of stock options and employee stock purchase plan | 7,071 | 6,208 | 2,365 |
Payments of prior period offering costs | (38) | ||
Net cash provided by financing activities | 278,295 | 188,071 | 136,189 |
Net (decrease) increase in cash, cash equivalents and restricted cash | (144,635) | 23,408 | 88,800 |
Cash, cash equivalents and restricted cash, beginning of period | 248,265 | 224,857 | 136,057 |
Cash, cash equivalents and restricted cash, end of period | 103,630 | 248,265 | 224,857 |
Non-cash investing and financing activities: | |||
Vesting of early exercised options and restricted stock | 15 | 53 | 184 |
Unpaid portion of property and equipment purchases included in period-end accounts payable and accrued liabilities | $ 9,756 | $ 468 | $ 283 |
Organization
Organization | 12 Months Ended |
Dec. 31, 2019 | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | |
Organization | 1. Organization MyoKardia, Inc. (the Company) is a clinical-stage biopharmaceutical company pioneering a precision medicine approach to discover, develop and commercialize targeted therapies for the treatment of serious and neglected rare cardiovascular diseases. The Company’s initial focus is on the treatment of cardiomyopathies, a group of diseases of the heart muscle. MyoKardia’s pipeline includes mavacamten and MYK-224, which are being studied for the treatment of hypertrophic cardiomyopathy, LUS-1, being studied for the treatment of diseases of diastolic dysfunction and danicamtiv (formerly MYK-491) and ACT-1, being studied for the treatment of diseases of systolic dysfunction. MyoKardia’s most advanced programs are: mavacamten, which is in four clinical trials including a Phase 3 study in patients with hypertrophic cardiomyopathy (HCM); danicamtiv, which recently completed a Phase 2a multiple-ascending dose study in patients with stable systolic heart failure and is being advanced to a Phase 2 study in patients with genetic dilated cardiomyopathy; and MYK-224, which is in a Phase 1 randomized, placebo-controlled study in healthy volunteers. The Company was incorporated on June 8, 2012 in Delaware. As of December 31, 2019, its corporate headquarters and operations were located in South San Francisco, California and as of the date of this filing its corporate headquarters and operations are located in Brisbane, California. Liquidity The Company has incurred significant operating losses since inception and has an accumulated deficit of $478.8 million as of December 31, 2019. The Company has relied on its ability to fund its operations through private and public equity financings and to a lesser extent, through a license and collaboration arrangement with a collaboration partner, Sanofi S.A. (Sanofi) via its subsidiary, Aventis, Inc. As discussed further in Note 3, the collaboration agreement ended on December 31, 2018 and the Company had no revenues relating to its Sanofi collaboration in 2019, nor has it received reimbursements of research and development expenses after June 30, 2019. The Company has not yet received regulatory approval to commercialize or sell any product and does not have customers. Management expects operating losses and negative operating cash flows to continue for the foreseeable future. As the Company continues to incur losses, a transition to profitability is dependent upon the successful development, approval, and commercialization of the Company’s products and product candidates and the achievement of a level of revenues adequate to support its cost structure. The Company’s ultimate success depends on the outcome of its research and development activities and anticipates the need to raise additional capital to fully implement its business plan. The Company intends to raise such capital through the issuance of additional equity, debt and/or strategic alliances with partner companies. As of December 31, 2019, the Company had $430.3 million of cash, cash equivalents and short and long-term investments, which management believes will be sufficient to meet the Company’s anticipated operating and capital expenditure requirements for the twelve months following the date of issuance of these financial statements. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 12 Months Ended |
Dec. 31, 2019 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies Basis of Presentation and Consolidation The consolidated financial statements of the Company include the Company’s accounts and have been prepared in conformity with accounting principles generally accepted in the United States of America (US GAAP) and include the Company’s accounts and those of its wholly-owned subsidiaries MyoKardia Australia Pty Ltd and MyoKardia Netherlands B.V Use of Estimates The preparation of consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, disclosure of contingent assets and liabilities and the reported amounts of revenues and expenses in the consolidated financial statements and the accompanying notes. On an ongoing basis, management evaluates its estimates, including those related to clinical trials accrued liabilities, income tax valuation allowance and stock-based compensation. Management bases its estimates on historical experience and on various other market-specific and relevant assumptions that management believes to be reasonable under the circumstances. Actual results could differ from those estimates. Segments The Company operates and manages its business as one reportable and operating segment, which is the business of developing and commercializing therapeutics. The Company’s chief executive officer, who is the chief operating decision maker, reviews financial information on an aggregate basis for purposes of allocating and evaluating financial performance. All revenues have been earned in the United States of America and all long-lived assets are maintained in the United States of America. Cash and Cash Equivalents Cash and cash equivalents consist of cash and other highly liquid investments with original maturities of three months or less from the date of purchase. At December 31, 2019 and 2018, the Company’s cash and cash equivalents were comprised of funds held in checking accounts, interest-bearing money market accounts, money market funds and commercial paper. Reconciliation of Cash, Cash Equivalents, and Restricted Cash as Reported in Consolidated Statements of Cash Flows Cash as reported in the consolidated statements of cash flows includes the aggregate amounts of cash, cash equivalents and restricted cash as presented on the consolidated balance sheets. Restricted cash at December 31, 2019 and 2018 represents cash balances held as security in connection with the Company’s facility lease agreements. The following table provides a reconciliation of cash, cash equivalents, and restricted cash within the consolidated balance sheets to the total shown in the consolidated statements of cash flows As of December 31, 2019 2018 Cash and cash equivalents $ 101,436 $ 246,122 Restricted cash included in prepaid expenses and other current assets 337 — Restricted cash included in restricted cash and other 1,857 2,143 Total cash, cash equivalents and restricted cash shown in the consolidated statements of cash flows $ 103,630 $ 248,265 Concentration of Credit Risk Financial instruments that potentially subject the Company to a concentration of credit risk consist of cash, cash equivalents and investments. All of the Company’s cash and cash equivalents are held at financial institutions that management believes are of high credit quality. Such deposits may, at times, exceed federally insured limits. The Company invests in a variety of financial instruments, such as, but not limited to, corporate debt and United States Treasury and Government agency securities, and by policy, limits the amount of credit exposure with any one financial institution or commercial issuer. The Company has not experienced any credit losses on its investments. Revenue Recognition The Company did not recognize revenues during the year ended December 31, 2019. As discussed in Note 3, in the years ended December 31, 2018 and 2017 the Company generated revenue from its collaboration and license agreement with its former collaboration partner, Sanofi. The collaboration and license agreement included non-refundable upfront license fees, reimbursement of research and development costs and contingent consideration payments based on the achievement of defined collaboration objectives. To date, the Company has not recognized revenue from sales of its product candidates. Effective January 1, 201 8 , the Company adopted A c counting Standards Codification Topic 606, Revenue from Contracts with Customers (ASC 606 ) using the full retrospective transition method. This standard applies to all contracts with customers, except for contracts that are within the scope of other standards, such as leases, insurance, certain collaboration arrangements and financial instruments. Under ASC 606, the Company recognizes revenue when its customer obtains control of promised goods or services, in an amount that reflects the consideration which the Company expects to receive in exchange for those goods or services. To determine revenue recognition for arrangements that the Company determines are within the scope of ASC 606, the Company performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) the Company satisfies a performance obligation. The Company only applies the five-step model to contracts when it is probable that Company will collect the consideration it is entitled to in exchange for the goods or services it transfers to the customer. At contract inception, once the contract is determined to be within the scope of ASC 606, the Company assesses the goods or services promised within each contract and determines those that are performance obligations and assesses whether each promised good or service is distinct. The Company then recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) the performance obligation is satisfied. Research and Development Expenses Research and development costs are expensed as incurred and consist of salaries and benefits, lab supplies and facility costs, and fees paid to others that conduct certain research and development activities on the Company’s behalf. Under the terms of the Company’s collaboration agreement with Sanofi, which terminated effective December 31, 2018, and as discussed in Note 3, the Company and Sanofi shared qualified research and development expenses that were jointly incurred to develop certain of the Company’s product candidates. Qualified costs consisted of internal and external research and development expenses including employee costs and direct out-of-pocket costs that were specifically identifiable or reasonably and directly related to the development of these candidates. Examples of qualified costs included those incurred for clinical trials, preparation for regulatory approval, manufacture or purchase of product for use in trials. Clinical Trials Accrued Liabilities The Company’s clinical trials accrued liabilities are a component of research and development expenses and based on patient enrollment and related costs at clinical investigator sites as well as estimates for the services received and efforts expended pursuant to contracts with clinical research organizations (CROs) that conduct and manage clinical trials on the Company’s behalf. Management estimates clinical trials accrued liabilities for services the Company has not yet been invoiced or otherwise notified of the actual cost based on the services performed, pursuant to contracts with research institutions and CROs that conduct and manage preclinical studies and clinical trials on its behalf. Management estimates these expenses based on discussions with the Company’s internal clinical management personnel and CROs as to the progress or stage of completion of trials or services and the contracted fees to be paid for such services. Assumptions used by management in developing the estimate include the progress or stage of completion and patient enrollment of the clinical trials. Recently Adopted Accounting Pronouncements – Leases Effective January 1, 2019, the Company adopted Accounting Standards Codification Topic 842, Leases (ASC 842), which requires lessees to recognize a right-of-use asset (ROU) and a lease liability on the balance sheet for all leases except for short-term leases with a lease term of twelve months or less. For lessees, leases continue to be classified as either operating or finance leases in the income statement. Lessor accounting is similar to the prior model but updated to align with certain changes to the lessee model. Lessors continue to classify leases as operating, direct financing or sales-type leases. The Company elected to adopt ASC 842 under the transition method that allows for the application of the new guidance at the beginning of the adoption period without recasting comparative periods. The Company also elected transition practical expedients to the implementation of the lease standard, as follows: (1) the Company did not reassess whether any expired or existing contracts, which had commenced before January 1, 2019, the date of adoption, are or contain leases (2) the Company did not reassess the lease classification for any expired or existing leases and (3) the Company did not reassess the initial direct costs for any existing leases. All of the Company’s leases are operating leases for property, which historically have been accounted for as operating leases, and under ASC 842 were also determined to be operating leases. The Company also reviewed its open contracts as of the date of adoption and determined that none had terms and conditions that would represent ROU assets or liabilities that would be considered embedded leases. Upon adoption, the Company recognized ROU assets and related lease liabilities totaling $2.1 million, representing the present value of future lease payments of each lease utilizing the Company’s incremental borrowing rate (IBR), which is the estimated borrowing rate of a collateralized loan over the remaining term of the lease. A deferred rent amount of $0.2 million as of December 31, 2018 was also reclassified to the ROU assets, reducing the carrying value to $1.9 million. Recently Adopted Accounting Pronouncements – Other In December 2019, the FASB issued ASU 2019-12, Income Taxes (Topic 740): Simplifying the Accounting for Income Taxes In June 2018, the FASB issued ASU No. 2018-07 (Topic 718), Compensation – Stock Compensation (ASU 2018-07) In February 2018, the FASB issued ASU No. 2018-05 (Topic 740) Income Taxes Recently Issued Accounting Pronouncements Not Yet Adopted In November 2018, the FASB issued ASU 2018-18 (Topic 808), Clarifying the Interaction Between Topic 808 and Topic 606, which provides guidance on how to assess whether certain transactions between collaborative arrangement participants should be accounted for within the revenue recognition standard. The ASU also provides more comparability in the presentation of revenue for certain transactions between collaborative arrangement participants. It accomplishes this by allowing organizations to only present units of account in collaborative arrangements that are within the scope of the revenue recognition standard together with revenue accounted for under the revenue recognition standard. The parts of the collaborative arrangement that are not in the scope of the revenue recognition standard should be presented separately from revenue accounted for under the revenue recognition standard. For public companies, the amendments in ASU 2018-18 are effective for fiscal years beginning after December 15, 2019, and interim periods within those fiscal years. Early adoption is permitted. The Company has evaluated this amendment and it is not expected to have a material impact to the Company’s financial statements. In August 2018, the FASB issued ASU 2018-13 (Topic 820), Fair Value Measurement In June 2016, the FASB issued ASU No. 2016-13 (Topic 326), Financial Instruments –Measurement of Credit Losses on Financial Instruments |
Collaboration and License Reven
Collaboration and License Revenue | 12 Months Ended |
Dec. 31, 2019 | |
Collaboration And License Agreement Disclosure [Abstract] | |
Collaboration and License Revenue | 3. Collaboration and License Revenue The Company has not recorded revenues in the year ended December 31, 2019, and all revenues in the years ended December 31, 2018 and 2017, respectively, relate to an exclusive License and Collaboration Agreement (Collaboration Agreement) with Aventis Inc., a wholly-owned subsidiary of Sanofi. The Collaboration Agreement provided for the research, development and potential commercialization of pharmaceutical products for the treatment, prevention and diagnosis of hypertrophic and dilated cardiomyopathy, as well as potential additional indications. Revenues for the two years ended December 31, 2018 and 2017, respectively, relate to a $45.0 million payment received from Sanofi in January 2017 as a non-refundable continuation payment for such research and development. The $45.0 million was recognized pro-rata over the two-year period ending December 31, 2018 based on a cost-based input method and was recorded proportionally in relation to the Company’s research and development effort over those periods. The Collaboration Agreement provided for a termination clause whereby on or before December 31, 2018, Sanofi was required to notify the Company of its intent to continue the collaboration and on December 31, 2018 Sanofi notified the Company it was not continuing the collaboration; however, certain royalty rights remained with Sanofi. In July 2019, the Company and Sanofi entered into a Termination Agreement and the Company reacquired these U.S. royalty rights for $80.0 million, of which $50.0 million was paid immediately and $30.0 million was deposited into escrow to be paid to Sanofi by June 30, 2020 without conditions. The Company also paid $4.3 million to Sanofi for certain of its assets related to the danicamtiv program. Neither the Company nor Sanofi have further obligations under the Collaboration Agreement that would prevent the payment of the escrowed amount. As a result of the Termination Agreement, there are no further rights or obligations between the parties. Revenue Recognition in 2018 and 2017 The Company implemented ASC 606 using the full retrospective transition method effective January 1, 2018 and revised its revenue for the years ended December 31, 2016 and 2017 accordingly. The Company evaluated the Collaboration Agreement under ASC 606 and determined that it had the following performance obligations to Sanofi: 1. the licenses of intellectual property for each of the HCM-1, HCM-2 and DCM-1 compound development programs, and 2. the performance of research and development services, including regulatory support, for each of the three programs. The Company considered whether the licenses had standalone functionality and were capable of being distinct; however, given the fact that the research and development services were of such a specialized nature that could only be performed by the Company and Sanofi could not benefit from the intellectual property licenses without the Company’s performance, the Company determined that the intellectual property licenses were not distinct from the research and development services and thus the license and research and development services for each program were combined into three separate performance obligations. Contract Term For revenue recognition purposes, the Company determined that the Collaboration Agreement was a period to period contract for which the Company had enforceable rights and obligations from inception through the initial term of December 31, 2016. Sanofi had the right to terminate the Collaboration Agreement prior to December 31, 2016 or to extend the contract term through December 31, 2018. If Sanofi had elected to terminate the agreement, the termination would have taken effect on December 31, 2016 and all licensed rights would have reverted to the Company. The Company did not have any obligation to reimburse Sanofi any portion of the payments received if Sanofi had terminated the agreement. In December 2016, Sanofi elected to continue the Collaboration Agreement for an extended term ending December 31, 2018 and made a $45.0 million continuation payment to the Company in January 2017. The Company determined that the extended term was to be treated as a separate contract because such an extension was not probable at the inception of the contract, the extension represented additional goods and services, and such activities were priced commensurate to the effort required and did not involve any significant discount. It was also concluded that the extended term provided the Company with enforceable rights and obligations for the two-year period ended December 31, 2018. Because Sanofi retained the option in the Collaboration Agreement to extend the arrangement, neither party was committed to perform and the contract did not have enforceable rights and obligations beyond December 31, 2018. Transaction Price The Company’s assessment of the transaction price included an analysis of amounts to which it was expected to be entitled for providing goods or services to the customer. The extended term (from January 1, 2017 to December 31, 2018) had a fixed fee of $45.0 million, paid by Sanofi contemporaneously with the notice of continuation of the contract. The Company therefore determined that the transaction price for this extended term was $45.0 million and this amount As noted above, the Collaboration Agreement included up to $45.0 million in funding from Sanofi of approved in-kind research and clinical activities. Sanofi was the decision maker on how to provide these services and such services were used in the development of joint program technology which is co-owned by both parties. As such the Company concluded that these in-kind contributions did not constitute consideration paid by Sanofi to the Company. Any consideration related to sales-based royalties were to be recognized when the related sales occurred and therefore have also been excluded from the transaction price. Methodology for Recognition Since the Company determined that the three performance obligations were satisfied over time, the Company selected a single revenue recognition method that it believed most faithfully depicts the Company’s performance in transferring control of the services. ASC 606 allows entities to choose between two methods to measure progress toward complete satisfaction of a performance obligation: 1. Output methods - recognize revenue on the basis of direct measurements of the value to the customer of the goods or services transferred to date relative to the remaining goods or services promised under the contract (e.g. surveys of performance completed to date, appraisals of results achieved, milestones reached, time elapsed, and units produced, or units delivered); or 2. Input methods - recognize revenue on the basis of the entity’s efforts or inputs to the satisfaction of a performance obligation (e.g., resources consumed, labor hours expended, costs incurred, or time elapsed) relative to the total expected inputs to the satisfaction of that performance obligation. The Company utilized a cost-based input method to measure proportional performance and calculated the corresponding amount of revenue to recognize. The Company believed this was the best measure of progress because other measures did not reflect how the Company executed its performance obligations under the contract with Sanofi. In applying the cost-based input methods of revenue recognition, the Company used actual costs incurred relative to budgeted costs to fulfill the combined performance obligations. Revenue was recognized based on actual costs incurred as a percentage of total actual and budgeted costs as the Company completed its performance obligations, which were fulfilled on December 31, 2018. A cost-based input method of revenue recognition requires management to make estimates of costs to complete the Company’s performance obligations. In making such estimates, significant judgment is required to evaluate assumptions related to cost estimates. The cumulative effect of revisions to estimated costs to complete the Company’s performance obligations were recorded in the period in which changes were identified and amounts could be reasonably estimated. For the years ended December 31, 2019, 2018 and 2017, the Company recognized zero, $33.6 The Company will not recognize any further revenue from the Collaboration Agreement. There were no contract assets or liabilities during the year ended December 31, 2019. The following table presents changes in the Company’s contract assets and liabilities, which excludes research and development reimbursements under the cost sharing plan further discussed below, for the year ended December 31, 2018 (in thousands): Year Ended December 31, 2018 Balance at Beginning of Period Additions Deductions Balance at End of Period Contract liabilities: Deferred revenue $ 33,558 $ — $ (33,558 ) $ — Cost Sharing During the years ended December 31, 2019, 2018 and 2017, the Company received research and development cost reimbursements from Sanofi under the terms of the Collaboration Agreement. Since the inception of the Collaboration Agreement and up until December 31, 2018, Sanofi had been conditionally responsible for reimbursing the Company for: (i) one half or more of the registration program plan (RPP) costs after clinical proof-of-concept had been established for the lead compound under each of the HCM-1 and HCM-2 programs; and (ii) if the Company had initiated a clinical trial of a compound under a proof-of-concept development plan and not terminated its development thereof and if an additional compound had been identified as a development candidate for the same program, the Company was entitled to full reimbursement of pre-proof-of-concept (pre-POC) (iii) research and development costs on development candidates mutually identified as such additional compounds, with the objective of conducting IND-enabling studies and clinical trials on such candidate. Effective October 2017, and through June 30, 2019, Sanofi shared RPP costs for the mavacamten program pursuant to the Collaboration Agreement termination terms. Registration program costs approved by the Company and Sanofi included amounts incurred relating to clinical trials, development and manufacturing of, and obtaining regulatory approvals for mavacamten, and included direct employee costs and direct out-of-pocket costs incurred, by or on behalf of a party, specifically identifiable or reasonably and directly allocable to those activities. Pursuant to the additional compounds provisions of the Collaboration Agreement, in August 2018 Sanofi agreed to reimburse the Company for eligible costs it incurred in the development of the MYK-224 compound, which had been identified as an additional compound under the HCM-1 program. Eligible costs were subject to review and approval under the same procedures as under the RPP program; reimbursable costs consisted of research and development activities agreed to by the Company and Sanofi that were negotiated and budgeted prior to the application for reimbursement. Reimbursements for this compound continued through March 31, 2019, in accordance to the Collaboration Agreement termination terms. Estimated reimbursements were invoiced to Sanofi before each interim period based on budgeted amounts. For the RPP program, these estimates consisted of one half of the Company’s mavacamten development budget in excess of Sanofi’s mavacamten development budget each interim period and the entire MYK-224 budget which was reimbursable in full. Actual amounts received from Sanofi were applied to the applicable interim period to reduce the Company’s research and development expenses. Due to the termination of the license agreement effective December 31, 2018, the Company has not received reimbursements for the MYK-224 compound for any periods subsequent to April 1, 2019 and for the mavacamten compound subsequent to July 1, 2019. The Company recorded $18.5 Year Ended December 31, 2019 2018 2017 Balance at beginning of year $ 12,973 $ 4,432 $ — Additions for advance billings — — 1,013 Payments received from Sanofi 5,521 31,659 10,697 Actual expenses incurred (18,494 ) (23,118 ) (7,278 ) Balance at end of year $ — $ 12,973 $ 4,432 |
Fair Value Measurements
Fair Value Measurements | 12 Months Ended |
Dec. 31, 2019 | |
Fair Value Disclosures [Abstract] | |
Fair Value Measurements | 4. Fair Value Measurements Fair value accounting is applied for all financial assets and liabilities, including short-term and long-term investments, and non-financial assets and liabilities that are recognized or disclosed at fair value in the consolidated financial statements on a recurring basis (at least annually). The carrying amount of the Company’s financial instruments, including accounts payable and accrued liabilities and other current liabilities approximate fair value due to their short-term maturities. Marketable securities are stated at their estimated fair values. The c ounterparties to the agreements relating to the Company’s investment securities consist of the U.S. Treasury, governmental agencies, various major corporat ions and financial institutions with high credit standing . The carrying amounts for financial instruments consisting of cash and cash equivalents, receivable from collaboration partner , accounts payable and accrued liabilities approximate fair value due to their short maturities. The accounting guidance for fair value provides a framework for measuring fair value, clarifies the definition of fair value and expands disclosures regarding fair value measurements. Fair value is defined as the price that would be received to sell an asset or paid to transfer a liability (an exit price) in an orderly transaction between market participants at the reporting date. The accounting guidance establishes a three-tiered hierarchy, which prioritizes the inputs used in the valuation methodologies in measuring fair value as follows: Level 1—Inputs are unadjusted, quoted prices in active markets for identical assets or liabilities at the measurement date. Level 2—Inputs other than quoted market prices included in Level 1 are either directly or indirectly observable for the asset or liability through correlation with market data at the measurement date and for the duration of the instrument’s anticipated life. Level 3—Inputs reflect management’s best estimate of what market participants would use in pricing the asset or liability at the measurement date. Consideration is given to the risk inherent in the valuation technique and the risk inherent in the inputs to the model. All investments have been classified as “available-for-sale” and are carried at fair value based upon quoted market prices or pricing models for similar securities at period end. Generally, those investments with contractual maturities greater than 12 months are considered long-term investments. Unrealized gains and losses, deemed temporary in nature, are reported as a component of accumulated other comprehensive loss, net of tax, on the consolidated balance sheets. A decline in the fair value of any security below cost that is deemed other than temporary results in a charge to earnings and the corresponding establishment of a new cost basis for the security. The amortized cost of securities is adjusted for amortization of premiums and accretion of discounts to maturity. Dividend and interest income are recognized when earned. Realized gains and losses are included in earnings and are derived using the specific identification method for determining the cost of securities sold. The following table sets forth the Company’s financial instruments that were measured at fair value on a recurring basis by level within the fair value hierarchy (in thousands): Fair Value Measurements at December 31, 2019 Total Level 1 Level 2 Level 3 Assets Money market funds $ 100,441 $ 100,441 $ — $ — U.S. government agency obligations 134,055 — 134,055 — Corporate securities 194,789 — 194,789 — Total $ 429,285 $ 100,441 $ 328,844 $ — Fair Value Measurements at December 31, 2018 Total Level 1 Level 2 Level 3 Assets Money market funds $ 245,194 $ 245,194 $ — $ — U.S. government agency obligations 85,033 — 85,033 — Corporate securities 63,679 — 63,679 — Total $ 393,906 $ 245,194 $ 148,712 $ — The following table is a summary of amortized cost, unrealized gain and loss, and fair value (in thousands) of the Company’s marketable securities by contractual maturities: Fair Value Measurements at December 31, 2019 Amortized Cost Unrealized Gain Unrealized Loss Fair Value Cash equivalents (due within 90 days) $ 100,440 $ 1 $ — $ 100,441 Short-term investments (due within one year) 314,181 523 (13 ) 314,691 Long-term investments (due between one and two years) 14,110 47 (4 ) 14,153 Total $ 428,731 $ 571 $ (17 ) $ 429,285 Fair Amortized Cost Unrealized Gain Unrealized Loss Fair Value Cash equivalents (due within 90 days) $ 245,194 $ — $ — $ 245,194 Short-term investments (due within one year) 68,656 — (92 ) 68,564 Long-term investments (due between one and two years) 80,118 98 (68 ) 80,148 Total $ 393,968 $ 98 $ (160 ) $ 393,906 The Company recognizes transfers between levels of the fair value hierarchy as of the end of the reporting period. There were no transfers within the hierarchy during the years ended December 31, 2019 and 2018. There were no material realized gains or losses on available-for- sale securities during the periods presented. |
Balance Sheet Components
Balance Sheet Components | 12 Months Ended |
Dec. 31, 2019 | |
Balance Sheet Related Disclosures [Abstract] | |
Balance Sheet Components | 5. Balance Sheet Components Property and Equipment Property and equipment are stated at cost and depreciated using the straight-line method over the estimated useful lives of the assets, ranging from two to five years. Leasehold improvements are amortized over the shorter of their estimated useful lives or the related lease term. Upon retirement or sale, the cost and related accumulated depreciation are removed from the consolidated balance sheet and the resulting gain or loss is reflected in the consolidated statement of operations and comprehensive loss. Property and equipment consists of the following (in thousands): December 31, 2019 2018 Scientific equipment $ 10,642 $ 9,126 Furniture and equipment 2,572 1,248 Capitalized software 389 302 Leasehold improvements 509 451 Construction in progress 9,568 — Total 23,680 11,127 Less: Accumulated depreciation (7,937 ) (5,989 ) Property and equipment, net $ 15,743 $ 5,138 Depreciation expense was $1.9 million, $1.6 million and $1.3 million for the years ended December 31, 2019, 2018 and 2017, respectively. Construction in progress consists of leasehold improvements made to the Company’s Brisbane, California facility in preparation for occupancy in January 2020. The Company reviews long-lived assets, including property and equipment, for impairment whenever events or changes in business circumstances indicate that the carrying amount of the assets may not be fully recoverable. An impairment charge would be recorded when estimated undiscounted future cash flows expected to result from the use of the asset and its eventual disposition are less than its carrying amount. Impairment, if any, is assessed using discounted cash flows or other appropriate measures of fair value. Through December 31, 2019, there have been no such impairment charges. Accrued Liabilities Accrued liabilities consist of the following (in thousands): December 31, 2019 2018 Clinical trials accrued liabilities $ 11,494 $ 6,272 Outside services 6,592 4,631 Payroll-related liabilities 11,724 8,151 Construction in progress 9,139 — Other 2,343 1,704 Total accrued liabilities $ 41,292 $ 20,758 Construction in progress consists of leasehold improvements made to the Company’s Brisbane, California facility in preparation for occupancy in January 2020. |
Leases
Leases | 12 Months Ended |
Dec. 31, 2019 | |
Lessee Disclosure [Abstract] | |
Leases | 6. Leases The Company determines if an arrangement is or contains a lease at inception. Operating lease right-of-use (ROU) assets and liabilities are presented separately on our consolidated balance sheets. The Company does not have any finance leases. Operating lease ROU assets and operating lease liabilities are recognized based on the present value of the future minimum lease payments over the lease term beginning at the commencement date. As the Company’s leases do not provide enough information to determine an implicit interest rate, the Company determines its incremental borrowing rate based on the rate of interest that the Company would have to pay to borrow on a collateralized basis over a similar term in a similar economic environment. The operating lease ROU assets also include any lease payments made and excludes lease incentives and initial direct costs incurred. Lease terms may include options to extend or terminate the lease when it is reasonably certain that the Company will exercise that option. Lease expense for minimum lease payments is recognized on a straight-line basis over the lease term. As of December 31, 2019, the Company has operating leases for approximately 56,500 square feet of office and lab space in two separate facilities in South San Francisco, California (the “Existing Facilities”). All of the lease agreements associated with the Existing Facilities expire on or before April 30, 2020 and there are no options to extend the leases. The Company does not plan to cancel the existing lease agreements for its Existing Facilities prior to their respective expiration dates. Information related to operating leases as of December 31, 2019 and upon adoption of ASC 842 on January 1, 2019 is as follows (in thousands, except for percentages and years): December 31, 2019 January 1, 2019 Assets Operating lease right-of-use assets $ 417 $ 1,940 Liabilities Operating lease liabilities - current $ 383 $ 2,126 Weighted average remaining lease term (years) 0.3 1.0 Weighted average discount rate 6 % 6 % Information related to operating lease activity during the year ended December 31, 2019 follows (in thousands): Year Ended December 31, 2019 Operating lease right-of-use assets obtained in exchange for lease obligations $ 1,095 Operating lease rental expense $ 2,760 Operating lease payments $ 3,007 Future annual payments of operating lease liabilities as of December 31, 2019 are as follows (in thousands): Year ending December 31: Amount 2020 387 Total future lease payments 387 Less: imputed interest (4 ) Total operating lease liabilities $ 383 In September 2018, the Company entered into a noncancelable operating lease (the “Lease”) for approximately 129,800 square feet of space in Brisbane, California (the “New Facility”). As of December 31, 2019, the Company has capitalized $9.1 million of tenant improvements as construction in progress within property and equipment. The lease commencement date was in January 2020 and the Company will record the ROU asset and lease liability on its balance sheet in accordance with ASC 842 in the first quarter of 2020. The Company will also reclass the amount included as tenant improvement within property and equipment on the December 31, 2019 balance sheet to leasehold improvements within property and equipment and amortize it over the lease term of 10 years. The Lease grants the Company an option to extend the Lease for an additional 10-year period. As of December 31, 2019, future minimum rental payments under the Lease during the 10-year term are $93.2 million in the aggregate. The Lease further provides that the Company is obligated to pay to the landlord certain costs, including taxes and operating expenses. In September 2018, the Company provided a standby letter of credit of $1.9 million as security for its obligations under the Lease. This standby letter of credit is classified on the balance sheet as restricted cash and other. Future annual minimum operating lease payments due under the Lease are as follows (in thousands): Year ending December 31: Amount 2020 5,454 2021 8,461 2022 8,757 2023 9,063 2024 9,381 Thereafter 52,063 Total $ 93,179 The adoption of ASC 842 did not materially affect the amount or timing of operating lease rent expense to be recognized during the year ended December 31, 2019 as compared to accounting under the prior guidance. Operating lease r ent expense for the year ended December 31, 2019, 2018 and 2017 was $2.8 million, $2.1 million and $1.4 million, respectively, which is included in operating expenses on the Company’s consolidated statements of operations and comprehensive loss. The operating leases require the Company to share in prorated operating expenses and property taxes based upon actual amounts incurred; those amounts are not fixed for future periods and, therefore, are not included in the future commitments listed above. |
Commitments and Contingencies
Commitments and Contingencies | 12 Months Ended |
Dec. 31, 2019 | |
Commitments And Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 7. Commitments and Contingencies Purchase Commitments The Company conducts product research and development programs through a combination of internal and collaborative programs that include, among others, arrangements with universities, contract research organizations and clinical research sites. The Company has contractual arrangements with these organizations; however, these contracts are generally cancelable on 30 days’ notice and the obligations under these contracts are largely based on services performed. Contingencies From time to time, the Company may have contingent liabilities that arise in the ordinary course of business activities. The Company accrues for such a liability when it is probable that future expenditures will be made, and such expenditures can be reasonably estimated. There were no contingent liabilities requiring accrual or disclosure as of December 31, 2019 and 2018. Guarantees and Indemnifications The Company enters into standard indemnification arrangements in the ordinary course of business. Pursuant to certain of these arrangements, the Company indemnifies, holds harmless, and agrees to reimburse the indemnified parties for losses suffered or incurred by the indemnified party, in connection with any trade secret, copyright, patent or other intellectual property infringement claim by any third-party with respect to the Company’s technology. The term of these indemnification arrangements is generally perpetual. The maximum potential amount of future payments the Company could be required to make under these agreements is not determinable because it involves claims that may be made against the Company in the future but have not yet been made. The Company indemnifies its officers and directors for certain events or occurrences, subject to certain limits, while the officer or director is or was serving at the Company’s request in such capacity, as permitted under Delaware law and in accordance with its certificate of incorporation and bylaws, and agreements providing for indemnification entered into with its officers and directors. The term of the indemnification period lasts as long as an officer or director may be subject to any proceeding arising out of acts or omissions of such officer or director in such capacity. The maximum amount of potential future indemnification of directors and officers is unlimited; however, the Company currently holds director and officer liability insurance. This insurance allows the transfer of risk associated with its exposure and may enable it to recover a portion of any future amounts paid. The Company believes that the fair value of these indemnification obligations is minimal. Accordingly, the Company has not recognized any liabilities relating to these obligations for any period presented. |
Stockholders' Equity
Stockholders' Equity | 12 Months Ended |
Dec. 31, 2019 | |
Stockholders Equity Note [Abstract] | |
Stockholders' Equity | 8. Stockholders’ Equity On March 8, 2018, the Company filed a Registration Statement on Form S-3ASR (the “2018 Shelf Registration Statement”) covering the potential offering, issuance, and sale of an indeterminate amount of common stock, preferred stock, debt securities, warrants and/or units. In March 2019, the Company completed a follow-on offering under the 2018 Shelf Registration Statement in which the Company issued 5,663,750 shares of common stock at a price of $51.00 per share, including 738,750 shares sold directly to the underwriters upon exercise of their option to purchase up to 738,750 shares of the Company’s common stock within 30 days of the offering. During the year ended December 31, 2019, the Company received proceeds totaling approximately $271.2 million from the offering, net of underwriting discounts and commissions and offering expenses. Common Stock Reserved for Issuance The Company has reserved shares of common stock, on an as-if-converted basis, for issuance as follows: December 31, 2019 December 31, 2018 Options and awards issued and outstanding 5,315,254 3,864,407 Shares available for issuance under 2015 Stock Option and Incentive Plan 685,435 904,785 Shares available for issuance under 2015 Employee Stock Purchase Plan 1,140,541 780,716 Total 7,141,230 5,549,908 Preferred stock As amended in November 2015, the Company's Certificate of Incorporation authorizes 5,000,000 shares of preferred stock at a par value of $0.0001 per share. As of December 31, 2019, and 2018, no preferred stock was issued or outstanding. |
Stock-Based Compensation
Stock-Based Compensation | 12 Months Ended |
Dec. 31, 2019 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Stock-Based Compensation | 9. Stock-Based Compensation In June 2012, the Company adopted the 2012 Equity Incentive Plan (as amended, the 2012 Plan). The 2012 Plan provides for the granting of incentive stock options, nonstatutory stock options, restricted stock units (RSUs), stock bonuses and rights to acquire restricted stock to employees, officers, directors and consultants. Incentive stock options may be granted with exercise prices of not less than 100% of the estimated fair value of the common stock and nonstatutory stock options may be granted with an exercise price of not less than 85% of the estimated fair value of the common stock on the date of grant. Stock options granted to a stockholder owning more than 10% of the voting stock must have an exercise price of not less than 110% of the estimated fair value of the common stock on the date of grant. The Board of Directors determines the estimated fair value of common stock. Stock options were generally granted with terms of up to ten years and vest over a period of four years. Upon the exercise of options, the Company issues new common stock from its authorized shares. Effective with the Company’s initial public offering in August 2015, the Company no longer issues shares from this plan and all cancelled or forfeited shares are returned to the 2015 Stock Option and Incentive Plan. In October 2015, the Company’s Board of Directors and stockholders adopted the 2015 Stock Option and Incentive Plan (the 2015 Plan) and the 2015 Employee Stock Purchase Plan (2015 ESPP) and The Company began issuing RSUs to employees under the 2015 Plan during the year ended December 31, 2018. RSUs settle into shares of common stock upon vesting, generally over a four-year . During the year ended December 31, 2019, the Company also granted certain key employees performance-based RSUs. The Company accounts for stock-based compensation arrangements with employees in accordance with ASC 718, Stock Compensation. Stock-based compensation expense for performance stock options and RSUs is based on the probability of achieving certain performance criteria, as defined in the individual grant agreement. The Company estimates the number of performance options and RSUs ultimately expected to vest and recognizes stock-based compensation expense for those options and RSUs expected to vest when it becomes probable that the performance criteria will be met. The fair value of a stock-based award is recognized over the period during which a grantee is required to provide services in exchange for the option or RSU award, known as the requisite service period (usually the vesting period), on a straight-line basis. The Company accounts for forfeitures as they occur. Equity instruments issued to non-employees are recorded at their fair value on the grant date. The fair value of options granted to consultants is expensed over the non-employees’ vesting period. Non-employee stock-based compensation expense was not material for all periods presented. Estimating the fair value of equity-settled awards as of the grant date using valuation models, such as the Black-Scholes option pricing model, is affected by assumptions regarding a number of complex variables. Changes in the assumptions can materially affect the fair value and ultimately how much stock-based compensation expense is recognized. These inputs are subjective and generally require significant analysis and judgment to develop. Expected Term —The expected term assumption represents the weighted-average period that the Company’s stock-based awards are expected to be outstanding. The expected term of the Company’s options exceeds the number of years the Company has been a publicly held corporation; therefore, the Company has opted to use the “simplified method” for estimating the expected term of the options. The simplified method is calculated as average of the vesting term and the original contractual term of the option. Expected Volatility —For all stock options granted to date, the volatility data was estimated based on a study of the Company’s trading history and that of its publicly traded industry peer companies. For purposes of identifying these peer companies, the Company considered the industry, stage of development, size and financial leverage of potential comparable companies. Expected Dividend —The Black-Scholes valuation model calls for a single expected dividend yield as an input. The Company currently has no history or expectation of paying cash dividends on its common stock. Risk-Free Interest Rate—The risk-free interest rate is based on the yield available on U.S. Treasury zero-coupon issues similar in duration to the expected term of the equity-settled award. In October 2015, the Company adopted the 2015 ESPP, which provides eligible employees with the opportunity to acquire an ownership interest in the Company through periodic payroll deductions, based on a six-month look-back period, at a price equal to the lesser of 85% of the fair market value of the common stock at either the first business day or last business day of the relevant offering period, provided that no more than 2,500 shares of common stock may be purchased by any one employee during each offering period. The 2015 ESPP is intended to constitute an “employee stock purchase plan” under Section 423(b) of the Internal Revenue Code of 1986, as amended. The 2015 ESPP may be terminated by the Company’s board of directors at any time. A total of 255,000 shares of common stock were initially reserved for issuance under the 2015 ESPP, subject to an annual increase on January 1 of each year beginning on January 1, 2017. Effective January 1, 2020 and 2019, the Company reserved an additional 463,790 402,889 Options The following table summarizes stock option activity and related information for the periods presented below: Shares Subject to Outstanding Options Weighted Average Exercise Price Per Option Weighted Average Remaining Contractual Term (in years) Aggregate Intrinsic Value (in Balance at December 31, 2018 3,701,461 $ 26.40 Options granted 1,371,130 43.27 Options exercised (337,846 ) 15.78 Options canceled (160,587 ) 38.75 Balance at December 31, 2019 4,574,158 $ 31.81 7.7 $ 187,879 Exercisable at December 31, 2019 2,380,560 $ 23.21 7.0 $ 118,246 Vested and expected to vest at December 31, 2019 4,574,158 $ 31.81 7.7 $ 187,879 The aggregate intrinsic value of options was calculated as the difference between the exercise price of the options and the estimated fair value of common stock. The aggregate intrinsic value of options exercised was $14.3 million, $21.1 million and $9.8 million for the years ended December 31, 2019, 2018 and 2017, respectively. The weighted‑average grant date fair value of options granted under the Company’s stock plans in the years ended December 31, 2019, 2018 and 2017 34.70 per share, respectively. As of December 31, 2019, total unamortized stock-based compensation relating to options was $55.3 million, which is expected to be recognized over the average remaining vesting period of 2.4 years. Year Ended December 31, 2019 2018 2017 Risk-free interest rate 1.59% - 2.54% 2.54% - 2.98% 1.92% - 2.27% Expected life (in years) 6.1% 5.3 - 6.1 5.3 - 6.1 Volatility 65% - 69% 69% - 75% 71% - 75% Dividend yield 0% 0% 0% Restricted Stock Units The following table summarizes RSU activity and related information for the period presented below: Shares Subject to Outstanding Awards Weighted Average Grant Date Fair Value Weighted Average Remaining Contractual Term (in years) Aggregate Intrinsic Value (in Balance at December 31, 2018 162,946 $ 53.37 RSUs awarded 643,814 45.36 RSUs released (42,214 ) 53.08 RSUs forfeited (23,450 ) 48.23 Balance at December 31, 2019 741,096 $ 46.59 2.2 $ 54,015 The weighted -average grant-date fair value of RSUs granted during the years ended December 31, 2019 and 2018 was $45.36 and $53.27, respectively. The total fair value of RSUs vested during the years ended December 31, 2019, 2018 and 2017 was $2.0 million, $167,000 and zero, respectively. As of December 31, 2019, total unamortized stock-based compensation relating to RSUs was $27.9 million, which is expected to be recognized over the average remaining vesting period of 3.1 years. Stock-Based Compensation Stock-based compensation expense, net of forfeitures, as applicable for the years ended December 31, 2019, 2018 and 2017, is reflected in the statements of operations and comprehensive loss as follows (in thousands): Year Ended December 31, 2019 2018 2017 Research and development $ 14,673 $ 8,144 $ 2,752 Selling, general and administrative $ 18,321 11,197 3,386 Total stock-based compensation $ 32,994 $ 19,341 $ 6,138 During the years ended December 31, 2018, 2017, and 2016, the Company recognized $340,000, $248,000 and $174,000 in stock-based compensation expense relating to stock options issued with performance-based vesting criteria, including the achievement of certain clinical and regulatory development milestones related to product candidates as well as commercial milestones. Recognition of stock-based compensation expense associated with these performance-based stock options commences when the performance condition is considered probable of achievement, using management’s best estimates, which consider the inherent risk and uncertainty regarding the future outcomes of the activities described by the milestones. |
Net Loss per Share
Net Loss per Share | 12 Months Ended |
Dec. 31, 2019 | |
Earnings Per Share [Abstract] | |
Net Loss per Share | 10. Net Loss per Share The following table sets forth the computation of basic and diluted net loss per share (in thousands, except share and per share data): Year Ended December 31, 2019 2018 2017 Numerator Net loss $ (276,213 ) $ (67,698 ) $ (57,010 ) Denominator Weighted average shares outstanding 44,767,581 38,466,233 33,098,571 Less: weighted average shares subject to repurchase (2,085 ) (79,327 ) (266,057 ) Weighted average shares used to compute basic and diluted net loss per share 44,765,496 38,386,906 32,832,514 Net loss per share, basic and diluted $ (6.17 ) $ (1.76 ) $ (1.74 ) Basic net loss per share is computed by dividing the net loss by the weighted average number of common shares outstanding for the period. Diluted net loss per share is computed by dividing the net loss by the weighted average number of common shares and potentially dilutive securities for the period, determined using the treasury-stock method and the as-if converted method, for convertible securities, if inclusion of these is dilutive. Because the Company has reported a net loss for all periods presented, diluted net loss per common share is the same as basic net loss per common share for those periods. The following outstanding shares of potentially dilutive securities were excluded from the computation of diluted net loss per share for the periods presented because including them would have been antidilutive: As of December 31, 2019 2018 2017 Common stock subject to repurchase — 9,790 81,373 Options and awards issued and outstanding 5,315,254 3,864,407 2,964,549 As of December 31, 2019, the Company has contributions from plan participants of $0.4 million under the 2015 ESPP, which if converted, would be equivalent to 10,000 shares based on 85% of the stock price at the beginning of the offering period. As of December 31, 2018, the Company had contributions from plan participants of $0.3 million under the 2015 ESPP, which if converted, would be equivalent to 5,700 shares based on 85% of the stock price at the beginning of the offering period. |
Employee Benefit Plan
Employee Benefit Plan | 12 Months Ended |
Dec. 31, 2019 | |
Employee Benefit Plans [Abstract] | |
Employee Benefit Plan | 11. Employee Benefit Plan The Company sponsors a 401(k) Plan, which stipulates that eligible employees can elect to contribute to the 401(k) Plan, subject to certain limitations of eligible compensation. |
Income Taxes
Income Taxes | 12 Months Ended |
Dec. 31, 2019 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | 12. Income Taxes The Company accounts for income taxes under the asset and liability method. Current income tax expense or benefit represents the amount of income taxes expected to be payable or refundable for the current year. Deferred income tax assets and liabilities are determined based on differences between the financial statement reporting and tax bases of assets and liabilities and net operating loss and credit carryforwards and are measured using the enacted tax rates and laws that will be in effect when such items are expected to reverse. Deferred income tax assets are reduced, as necessary, by a valuation allowance when management determines it is more likely than not that some or all of the tax benefits will not be realized. For each of the years ended December 31, 2019, 2018 and 2017, the effective income tax rate and tax provision from continuing operations was 0% The following is the reconciliation between the statutory federal income tax rate and the Company’s effective tax rate: Year Ended December 31, 2019 2018 2017 Federal statutory income tax rate 21.0 % 21.0 % 34.0 % State taxes (tax effected) 7.3 8.6 7.1 Non-deductible expenses and other (0.5 ) 2.5 2.0 Research and development reimbursements 3.2 5.9 6.0 Change in valuation allowance (31.0 ) (38.0 ) (24.0 ) Tax Act – net deferred tax rate change — — (25.1 ) Total — % — % — % As of December 31, 2019 and 2018, the components of the Company’s deferred tax assets are as follows (in thousands): As of December 31, 2019 2018 Deferred tax assets: Net operating loss carryforwards 98,445 49,689 Research and development reimbursement carryforwards 23,061 12,908 Stock-based compensation 8,861 4,349 Start-up costs 22,896 1,237 Depreciation (1,027 ) (959 ) Other 2,646 1,858 Total deferred tax assets 154,882 69,082 Less: valuation allowance (154,882 ) (69,082 ) Net deferred tax assets — — The Company’s primary deferred tax assets of $98.4 million and $49.7 million at December 31, 2019 and 2018, respectively, relate to its net operating loss carryforwards (NOLs). Based on a history of cumulative losses in recent periods and consideration of other available positive and negative evidence, the Company has recorded a full valuation allowance to offset the deferred tax assets for both periods presented. As of December 31, 2019, the Company had approximately $351.1 million and $354.0 million of federal and state net operating losses, respectively, that will begin to expire in 2032. As of December 31, 2019, the Company had approximately $7.2 million and $5.9 million of federal and state research and development tax credit carryovers, respectively. If not utilized, the federal credit carryforward will expire in 2032, and the state credit carryforward does not expire. As of December 31, 2019, the Company had approximately $18.2 million of federal orphan tax credit carryovers, which will begin to expire in 2036 if not utilized. The valuation allowance increased by approximately $85.8 million, $ 25.7 Under Sections 382 and 383 of the Internal Revenue Code of 1986, as amended (the Code) if a corporation undergoes an “ownership change,” generally defined as a greater than 50% change (by value) in its equity ownership over a rolling three-year As of December 31, 2019, and 2018, the Company did not have a liability related to unrecognized tax benefits. All unrecognized tax benefits have been netted against the research and development and orphan drug credit carryforwards deferred tax asset. The Company records interest and penalties related to unrecognized tax benefits within interest and other income, net. As of December 31, 2019, and 2018, the Company had not accrued any interest or penalties related to unrecognized tax benefits. The Company is subject to U.S. federal and California income tax assessment for years beginning in 2012 and Australia beginning in 2015. However, since the Company has incurred federal and California net operating losses every year since inception, all of its income tax returns are subject to examination and adjustments by the Internal Revenue Service for at least three years and by the California Franchise Tax Board for four years following the year in which the tax attributes are utilized. The Company does not believe that there will be a material change in its unrecognized tax positions over the next twelve months. There is no amount of unrecognized tax benefit that, if recognized, would affect the effective tax rate. Uncertain Tax Positions The Company accounts for uncertain tax positions in accordance with ASC 740-10, Accounting for Uncertainty in Income Taxes. The Company includes penalties and interest expense related to income taxes as a component of interest and other income, net; there were no interest or penalties accrued at December 31, 2019 and 2018. The Company has not been audited by the Internal Revenue Service, any state tax authority, or foreign tax authorities. It is subject to taxation in the United States and Australia. Because of the net operating loss, research credit carryforwards, and orphan drug tax credit carryforwards, substantially all of its tax years, from 2012 to 2019, remain open to U.S. federal and California tax examinations. The statute of limitation in Australia is four years. At December 31, 2019, 2018 and 2017, the Company's reserve for unrecognized tax benefits is approximately $7.5 million, $3.8 million and $2.5 million, respectively. Due to the full valuation allowance at December 31, 2019, current adjustments to the unrecognized benefits will have no impact to the Company's effective income tax rate. A reconciliation of the beginning and ending amount of unrecognized tax benefits is as follows (in thousands): For the Year Ended December 31, 2019 2018 2017 Beginning balance $ 3,810 $ 2,471 $ 1,474 Increases (decreases) of unrecognized tax benefits related to prior year 380 289 (97 ) Increases of unrecognized tax benefits related to current year 3,314 1,050 1,094 Ending balance $ 7,504 $ 3,810 $ 2,471 The Company does not anticipate material changes to its uncertain tax positions through the next twelve months. |
Quarterly Financial Data (unaud
Quarterly Financial Data (unaudited) | 12 Months Ended |
Dec. 31, 2019 | |
Quarterly Financial Data [Abstract] | |
Quarterly Financial Data (unaudited) | 13. Quarterly Financial Data (unaudited) The following table summarizes the unaudited quarterly financial data for the last two fiscal years (in thousands, except per share data): (in thousands, except share and per share amounts) First Second Third Fourth Quarter Quarter Quarter Quarter 2019 Revenues $ — $ — $ — $ — Total operating expenses $ 39,741 $ 41,564 $ 145,118 $ 61,411 Net loss $ (37,470 ) $ (38,174 ) $ (141,802 ) $ (58,767 ) Net loss per common share, basic and diluted $ (0.93 ) $ (0.83 ) $ (3.07 ) $ (1.27 ) Weighted average number of shares, basic and diluted 40,506,313 46,065,901 46,133,068 46,278,409 2018 Revenues $ 5,331 $ 6,639 $ 9,188 $ 12,400 Total operating expenses $ 23,931 $ 26,130 $ 26,867 $ 30,281 Net loss $ (17,820 ) $ (18,413 ) $ (15,789 ) $ (15,676 ) Net loss per common share, basic and diluted $ (0.50 ) $ (0.49 ) $ (0.39 ) $ (0.39 ) Weighted average number of shares, basic and diluted 35,827,235 37,440,024 40,116,644 40,259,575 |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 12 Months Ended |
Dec. 31, 2019 | |
Accounting Policies [Abstract] | |
Basis of Presentation and Consolidation | Basis of Presentation and Consolidation The consolidated financial statements of the Company include the Company’s accounts and have been prepared in conformity with accounting principles generally accepted in the United States of America (US GAAP) and include the Company’s accounts and those of its wholly-owned subsidiaries MyoKardia Australia Pty Ltd and MyoKardia Netherlands B.V |
Use of Estimates | Use of Estimates The preparation of consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, disclosure of contingent assets and liabilities and the reported amounts of revenues and expenses in the consolidated financial statements and the accompanying notes. On an ongoing basis, management evaluates its estimates, including those related to clinical trials accrued liabilities, income tax valuation allowance and stock-based compensation. Management bases its estimates on historical experience and on various other market-specific and relevant assumptions that management believes to be reasonable under the circumstances. Actual results could differ from those estimates. |
Segments | Segments The Company operates and manages its business as one reportable and operating segment, which is the business of developing and commercializing therapeutics. The Company’s chief executive officer, who is the chief operating decision maker, reviews financial information on an aggregate basis for purposes of allocating and evaluating financial performance. All revenues have been earned in the United States of America and all long-lived assets are maintained in the United States of America. |
Cash and cash equivalents | Cash and Cash Equivalents Cash and cash equivalents consist of cash and other highly liquid investments with original maturities of three months or less from the date of purchase. At December 31, 2019 and 2018, the Company’s cash and cash equivalents were comprised of funds held in checking accounts, interest-bearing money market accounts, money market funds and commercial paper. |
Reconciliation of Cash, Cash Equivalents, and Restricted Cash as Reported in Consolidated Statements of Cash Flows | Reconciliation of Cash, Cash Equivalents, and Restricted Cash as Reported in Consolidated Statements of Cash Flows Cash as reported in the consolidated statements of cash flows includes the aggregate amounts of cash, cash equivalents and restricted cash as presented on the consolidated balance sheets. Restricted cash at December 31, 2019 and 2018 represents cash balances held as security in connection with the Company’s facility lease agreements. The following table provides a reconciliation of cash, cash equivalents, and restricted cash within the consolidated balance sheets to the total shown in the consolidated statements of cash flows As of December 31, 2019 2018 Cash and cash equivalents $ 101,436 $ 246,122 Restricted cash included in prepaid expenses and other current assets 337 — Restricted cash included in restricted cash and other 1,857 2,143 Total cash, cash equivalents and restricted cash shown in the consolidated statements of cash flows $ 103,630 $ 248,265 |
Concentration of Credit Risk | Concentration of Credit Risk Financial instruments that potentially subject the Company to a concentration of credit risk consist of cash, cash equivalents and investments. All of the Company’s cash and cash equivalents are held at financial institutions that management believes are of high credit quality. Such deposits may, at times, exceed federally insured limits. The Company invests in a variety of financial instruments, such as, but not limited to, corporate debt and United States Treasury and Government agency securities, and by policy, limits the amount of credit exposure with any one financial institution or commercial issuer. The Company has not experienced any credit losses on its investments. |
Revenue Recognition | Revenue Recognition The Company did not recognize revenues during the year ended December 31, 2019. As discussed in Note 3, in the years ended December 31, 2018 and 2017 the Company generated revenue from its collaboration and license agreement with its former collaboration partner, Sanofi. The collaboration and license agreement included non-refundable upfront license fees, reimbursement of research and development costs and contingent consideration payments based on the achievement of defined collaboration objectives. To date, the Company has not recognized revenue from sales of its product candidates. Effective January 1, 201 8 , the Company adopted A c counting Standards Codification Topic 606, Revenue from Contracts with Customers (ASC 606 ) using the full retrospective transition method. This standard applies to all contracts with customers, except for contracts that are within the scope of other standards, such as leases, insurance, certain collaboration arrangements and financial instruments. Under ASC 606, the Company recognizes revenue when its customer obtains control of promised goods or services, in an amount that reflects the consideration which the Company expects to receive in exchange for those goods or services. To determine revenue recognition for arrangements that the Company determines are within the scope of ASC 606, the Company performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) the Company satisfies a performance obligation. The Company only applies the five-step model to contracts when it is probable that Company will collect the consideration it is entitled to in exchange for the goods or services it transfers to the customer. At contract inception, once the contract is determined to be within the scope of ASC 606, the Company assesses the goods or services promised within each contract and determines those that are performance obligations and assesses whether each promised good or service is distinct. The Company then recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) the performance obligation is satisfied. Revenue Recognition in 2018 and 2017 The Company implemented ASC 606 using the full retrospective transition method effective January 1, 2018 and revised its revenue for the years ended December 31, 2016 and 2017 accordingly. The Company evaluated the Collaboration Agreement under ASC 606 and determined that it had the following performance obligations to Sanofi: 1. the licenses of intellectual property for each of the HCM-1, HCM-2 and DCM-1 compound development programs, and 2. the performance of research and development services, including regulatory support, for each of the three programs. The Company considered whether the licenses had standalone functionality and were capable of being distinct; however, given the fact that the research and development services were of such a specialized nature that could only be performed by the Company and Sanofi could not benefit from the intellectual property licenses without the Company’s performance, the Company determined that the intellectual property licenses were not distinct from the research and development services and thus the license and research and development services for each program were combined into three separate performance obligations. |
Research and Development Expenses | Research and Development Expenses Research and development costs are expensed as incurred and consist of salaries and benefits, lab supplies and facility costs, and fees paid to others that conduct certain research and development activities on the Company’s behalf. Under the terms of the Company’s collaboration agreement with Sanofi, which terminated effective December 31, 2018, and as discussed in Note 3, the Company and Sanofi shared qualified research and development expenses that were jointly incurred to develop certain of the Company’s product candidates. Qualified costs consisted of internal and external research and development expenses including employee costs and direct out-of-pocket costs that were specifically identifiable or reasonably and directly related to the development of these candidates. Examples of qualified costs included those incurred for clinical trials, preparation for regulatory approval, manufacture or purchase of product for use in trials. |
Clinical Trials Accrued Liabilities | Clinical Trials Accrued Liabilities The Company’s clinical trials accrued liabilities are a component of research and development expenses and based on patient enrollment and related costs at clinical investigator sites as well as estimates for the services received and efforts expended pursuant to contracts with clinical research organizations (CROs) that conduct and manage clinical trials on the Company’s behalf. Management estimates clinical trials accrued liabilities for services the Company has not yet been invoiced or otherwise notified of the actual cost based on the services performed, pursuant to contracts with research institutions and CROs that conduct and manage preclinical studies and clinical trials on its behalf. Management estimates these expenses based on discussions with the Company’s internal clinical management personnel and CROs as to the progress or stage of completion of trials or services and the contracted fees to be paid for such services. Assumptions used by management in developing the estimate include the progress or stage of completion and patient enrollment of the clinical trials. |
Recently Adopted Accounting Pronouncements | Recently Adopted Accounting Pronouncements – Leases Effective January 1, 2019, the Company adopted Accounting Standards Codification Topic 842, Leases (ASC 842), which requires lessees to recognize a right-of-use asset (ROU) and a lease liability on the balance sheet for all leases except for short-term leases with a lease term of twelve months or less. For lessees, leases continue to be classified as either operating or finance leases in the income statement. Lessor accounting is similar to the prior model but updated to align with certain changes to the lessee model. Lessors continue to classify leases as operating, direct financing or sales-type leases. The Company elected to adopt ASC 842 under the transition method that allows for the application of the new guidance at the beginning of the adoption period without recasting comparative periods. The Company also elected transition practical expedients to the implementation of the lease standard, as follows: (1) the Company did not reassess whether any expired or existing contracts, which had commenced before January 1, 2019, the date of adoption, are or contain leases (2) the Company did not reassess the lease classification for any expired or existing leases and (3) the Company did not reassess the initial direct costs for any existing leases. All of the Company’s leases are operating leases for property, which historically have been accounted for as operating leases, and under ASC 842 were also determined to be operating leases. The Company also reviewed its open contracts as of the date of adoption and determined that none had terms and conditions that would represent ROU assets or liabilities that would be considered embedded leases. Upon adoption, the Company recognized ROU assets and related lease liabilities totaling $2.1 million, representing the present value of future lease payments of each lease utilizing the Company’s incremental borrowing rate (IBR), which is the estimated borrowing rate of a collateralized loan over the remaining term of the lease. A deferred rent amount of $0.2 million as of December 31, 2018 was also reclassified to the ROU assets, reducing the carrying value to $1.9 million. Recently Adopted Accounting Pronouncements – Other In December 2019, the FASB issued ASU 2019-12, Income Taxes (Topic 740): Simplifying the Accounting for Income Taxes In June 2018, the FASB issued ASU No. 2018-07 (Topic 718), Compensation – Stock Compensation (ASU 2018-07) In February 2018, the FASB issued ASU No. 2018-05 (Topic 740) Income Taxes |
Recently Issued Accounting Pronouncements Not Yet Adopted | Recently Issued Accounting Pronouncements Not Yet Adopted In November 2018, the FASB issued ASU 2018-18 (Topic 808), Clarifying the Interaction Between Topic 808 and Topic 606, which provides guidance on how to assess whether certain transactions between collaborative arrangement participants should be accounted for within the revenue recognition standard. The ASU also provides more comparability in the presentation of revenue for certain transactions between collaborative arrangement participants. It accomplishes this by allowing organizations to only present units of account in collaborative arrangements that are within the scope of the revenue recognition standard together with revenue accounted for under the revenue recognition standard. The parts of the collaborative arrangement that are not in the scope of the revenue recognition standard should be presented separately from revenue accounted for under the revenue recognition standard. For public companies, the amendments in ASU 2018-18 are effective for fiscal years beginning after December 15, 2019, and interim periods within those fiscal years. Early adoption is permitted. The Company has evaluated this amendment and it is not expected to have a material impact to the Company’s financial statements. In August 2018, the FASB issued ASU 2018-13 (Topic 820), Fair Value Measurement In June 2016, the FASB issued ASU No. 2016-13 (Topic 326), Financial Instruments –Measurement of Credit Losses on Financial Instruments |
Fair Value Measurements | Fair Value Measurements Fair value accounting is applied for all financial assets and liabilities, including short-term and long-term investments, and non-financial assets and liabilities that are recognized or disclosed at fair value in the consolidated financial statements on a recurring basis (at least annually). The carrying amount of the Company’s financial instruments, including accounts payable and accrued liabilities and other current liabilities approximate fair value due to their short-term maturities. Marketable securities are stated at their estimated fair values. The c ounterparties to the agreements relating to the Company’s investment securities consist of the U.S. Treasury, governmental agencies, various major corporat ions and financial institutions with high credit standing . The carrying amounts for financial instruments consisting of cash and cash equivalents, receivable from collaboration partner , accounts payable and accrued liabilities approximate fair value due to their short maturities. The accounting guidance for fair value provides a framework for measuring fair value, clarifies the definition of fair value and expands disclosures regarding fair value measurements. Fair value is defined as the price that would be received to sell an asset or paid to transfer a liability (an exit price) in an orderly transaction between market participants at the reporting date. The accounting guidance establishes a three-tiered hierarchy, which prioritizes the inputs used in the valuation methodologies in measuring fair value as follows: Level 1—Inputs are unadjusted, quoted prices in active markets for identical assets or liabilities at the measurement date. Level 2—Inputs other than quoted market prices included in Level 1 are either directly or indirectly observable for the asset or liability through correlation with market data at the measurement date and for the duration of the instrument’s anticipated life. Level 3—Inputs reflect management’s best estimate of what market participants would use in pricing the asset or liability at the measurement date. Consideration is given to the risk inherent in the valuation technique and the risk inherent in the inputs to the model. |
Property and Equipment | Property and Equipment Property and equipment are stated at cost and depreciated using the straight-line method over the estimated useful lives of the assets, ranging from two to five years. Leasehold improvements are amortized over the shorter of their estimated useful lives or the related lease term. Upon retirement or sale, the cost and related accumulated depreciation are removed from the consolidated balance sheet and the resulting gain or loss is reflected in the consolidated statement of operations and comprehensive loss. |
Leases | Leases The Company determines if an arrangement is or contains a lease at inception. Operating lease right-of-use (ROU) assets and liabilities are presented separately on our consolidated balance sheets. The Company does not have any finance leases. Operating lease ROU assets and operating lease liabilities are recognized based on the present value of the future minimum lease payments over the lease term beginning at the commencement date. As the Company’s leases do not provide enough information to determine an implicit interest rate, the Company determines its incremental borrowing rate based on the rate of interest that the Company would have to pay to borrow on a collateralized basis over a similar term in a similar economic environment. The operating lease ROU assets also include any lease payments made and excludes lease incentives and initial direct costs incurred. Lease terms may include options to extend or terminate the lease when it is reasonably certain that the Company will exercise that option. Lease expense for minimum lease payments is recognized on a straight-line basis over the lease term. |
Stock-Based Compensation | Stock-Based Compensation The Company accounts for stock-based compensation arrangements with employees in accordance with ASC 718, Stock Compensation. Stock-based compensation expense for performance stock options and RSUs is based on the probability of achieving certain performance criteria, as defined in the individual grant agreement. The Company estimates the number of performance options and RSUs ultimately expected to vest and recognizes stock-based compensation expense for those options and RSUs expected to vest when it becomes probable that the performance criteria will be met. The fair value of a stock-based award is recognized over the period during which a grantee is required to provide services in exchange for the option or RSU award, known as the requisite service period (usually the vesting period), on a straight-line basis. The Company accounts for forfeitures as they occur. Equity instruments issued to non-employees are recorded at their fair value on the grant date. The fair value of options granted to consultants is expensed over the non-employees’ vesting period. Non-employee stock-based compensation expense was not material for all periods presented. |
Income Taxes | Income Taxes The Company accounts for income taxes under the asset and liability method. Current income tax expense or benefit represents the amount of income taxes expected to be payable or refundable for the current year. Deferred income tax assets and liabilities are determined based on differences between the financial statement reporting and tax bases of assets and liabilities and net operating loss and credit carryforwards and are measured using the enacted tax rates and laws that will be in effect when such items are expected to reverse. Deferred income tax assets are reduced, as necessary, by a valuation allowance when management determines it is more likely than not that some or all of the tax benefits will not be realized. |
Transaction Price | Transaction Price The Company’s assessment of the transaction price included an analysis of amounts to which it was expected to be entitled for providing goods or services to the customer. The extended term (from January 1, 2017 to December 31, 2018) had a fixed fee of $45.0 million, paid by Sanofi contemporaneously with the notice of continuation of the contract. The Company therefore determined that the transaction price for this extended term was $45.0 million and this amount As noted above, the Collaboration Agreement included up to $45.0 million in funding from Sanofi of approved in-kind research and clinical activities. Sanofi was the decision maker on how to provide these services and such services were used in the development of joint program technology which is co-owned by both parties. As such the Company concluded that these in-kind contributions did not constitute consideration paid by Sanofi to the Company. Any consideration related to sales-based royalties were to be recognized when the related sales occurred and therefore have also been excluded from the transaction price. |
Methodology of Revenue Recognition | Methodology for Recognition Since the Company determined that the three performance obligations were satisfied over time, the Company selected a single revenue recognition method that it believed most faithfully depicts the Company’s performance in transferring control of the services. ASC 606 allows entities to choose between two methods to measure progress toward complete satisfaction of a performance obligation: 1. Output methods - recognize revenue on the basis of direct measurements of the value to the customer of the goods or services transferred to date relative to the remaining goods or services promised under the contract (e.g. surveys of performance completed to date, appraisals of results achieved, milestones reached, time elapsed, and units produced, or units delivered); or 2. Input methods - recognize revenue on the basis of the entity’s efforts or inputs to the satisfaction of a performance obligation (e.g., resources consumed, labor hours expended, costs incurred, or time elapsed) relative to the total expected inputs to the satisfaction of that performance obligation. The Company utilized a cost-based input method to measure proportional performance and calculated the corresponding amount of revenue to recognize. The Company believed this was the best measure of progress because other measures did not reflect how the Company executed its performance obligations under the contract with Sanofi. In applying the cost-based input methods of revenue recognition, the Company used actual costs incurred relative to budgeted costs to fulfill the combined performance obligations. Revenue was recognized based on actual costs incurred as a percentage of total actual and budgeted costs as the Company completed its performance obligations, which were fulfilled on December 31, 2018. A cost-based input method of revenue recognition requires management to make estimates of costs to complete the Company’s performance obligations. In making such estimates, significant judgment is required to evaluate assumptions related to cost estimates. The cumulative effect of revisions to estimated costs to complete the Company’s performance obligations were recorded in the period in which changes were identified and amounts could be reasonably estimated. |
Registration Program Plan and Pre-POC Cost Sharing | Cost Sharing During the years ended December 31, 2019, 2018 and 2017, the Company received research and development cost reimbursements from Sanofi under the terms of the Collaboration Agreement. Since the inception of the Collaboration Agreement and up until December 31, 2018, Sanofi had been conditionally responsible for reimbursing the Company for: (i) one half or more of the registration program plan (RPP) costs after clinical proof-of-concept had been established for the lead compound under each of the HCM-1 and HCM-2 programs; and (ii) if the Company had initiated a clinical trial of a compound under a proof-of-concept development plan and not terminated its development thereof and if an additional compound had been identified as a development candidate for the same program, the Company was entitled to full reimbursement of pre-proof-of-concept (pre-POC) (iii) research and development costs on development candidates mutually identified as such additional compounds, with the objective of conducting IND-enabling studies and clinical trials on such candidate. Effective October 2017, and through June 30, 2019, Sanofi shared RPP costs for the mavacamten program pursuant to the Collaboration Agreement termination terms. Registration program costs approved by the Company and Sanofi included amounts incurred relating to clinical trials, development and manufacturing of, and obtaining regulatory approvals for mavacamten, and included direct employee costs and direct out-of-pocket costs incurred, by or on behalf of a party, specifically identifiable or reasonably and directly allocable to those activities. Pursuant to the additional compounds provisions of the Collaboration Agreement, in August 2018 Sanofi agreed to reimburse the Company for eligible costs it incurred in the development of the MYK-224 compound, which had been identified as an additional compound under the HCM-1 program. Eligible costs were subject to review and approval under the same procedures as under the RPP program; reimbursable costs consisted of research and development activities agreed to by the Company and Sanofi that were negotiated and budgeted prior to the application for reimbursement. Reimbursements for this compound continued through March 31, 2019, in accordance to the Collaboration Agreement termination terms. Estimated reimbursements were invoiced to Sanofi before each interim period based on budgeted amounts. For the RPP program, these estimates consisted of one half of the Company’s mavacamten development budget in excess of Sanofi’s mavacamten development budget each interim period and the entire MYK-224 budget which was reimbursable in full. Actual amounts received from Sanofi were applied to the applicable interim period to reduce the Company’s research and development expenses. Due to the termination of the license agreement effective December 31, 2018, the Company has not received reimbursements for the MYK-224 compound for any periods subsequent to April 1, 2019 and for the mavacamten compound subsequent to July 1, 2019. |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Accounting Policies [Abstract] | |
Summary of Cash Reported in Consolidated Statements of Cash Flows | The following table provides a reconciliation of cash, cash equivalents, and restricted cash within the consolidated balance sheets to the total shown in the consolidated statements of cash flows As of December 31, 2019 2018 Cash and cash equivalents $ 101,436 $ 246,122 Restricted cash included in prepaid expenses and other current assets 337 — Restricted cash included in restricted cash and other 1,857 2,143 Total cash, cash equivalents and restricted cash shown in the consolidated statements of cash flows $ 103,630 $ 248,265 |
Collaboration and License Rev_2
Collaboration and License Revenue (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Collaboration And License Agreement Disclosure [Abstract] | |
Changes in Contract Assets and Liabilities | The following table presents changes in the Company’s contract assets and liabilities, which excludes research and development reimbursements under the cost sharing plan further discussed below, for the year ended December 31, 2018 (in thousands): Year Ended December 31, 2018 Balance at Beginning of Period Additions Deductions Balance at End of Period Contract liabilities: Deferred revenue $ 33,558 $ — $ (33,558 ) $ — |
Schedule Prepayments and Reimbursed Research and Development Expenses | The following table presents the Sanofi prepayments and reimbursed research and development expenses for the years ended December 31, 2019, 2018 and 2017 (in thousands): Year Ended December 31, 2019 2018 2017 Balance at beginning of year $ 12,973 $ 4,432 $ — Additions for advance billings — — 1,013 Payments received from Sanofi 5,521 31,659 10,697 Actual expenses incurred (18,494 ) (23,118 ) (7,278 ) Balance at end of year $ — $ 12,973 $ 4,432 |
Fair Value Measurements (Tables
Fair Value Measurements (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Fair Value Disclosures [Abstract] | |
Summary of Fair Value Assets Measured on Recurring Basis | All investments have been classified as “available-for-sale” and are carried at fair value based upon quoted market prices or pricing models for similar securities at period end. Generally, those investments with contractual maturities greater than 12 months are considered long-term investments. Unrealized gains and losses, deemed temporary in nature, are reported as a component of accumulated other comprehensive loss, net of tax, on the consolidated balance sheets. A decline in the fair value of any security below cost that is deemed other than temporary results in a charge to earnings and the corresponding establishment of a new cost basis for the security. The amortized cost of securities is adjusted for amortization of premiums and accretion of discounts to maturity. Dividend and interest income are recognized when earned. Realized gains and losses are included in earnings and are derived using the specific identification method for determining the cost of securities sold. The following table sets forth the Company’s financial instruments that were measured at fair value on a recurring basis by level within the fair value hierarchy (in thousands): Fair Value Measurements at December 31, 2019 Total Level 1 Level 2 Level 3 Assets Money market funds $ 100,441 $ 100,441 $ — $ — U.S. government agency obligations 134,055 — 134,055 — Corporate securities 194,789 — 194,789 — Total $ 429,285 $ 100,441 $ 328,844 $ — Fair Value Measurements at December 31, 2018 Total Level 1 Level 2 Level 3 Assets Money market funds $ 245,194 $ 245,194 $ — $ — U.S. government agency obligations 85,033 — 85,033 — Corporate securities 63,679 — 63,679 — Total $ 393,906 $ 245,194 $ 148,712 $ — |
Summary of Fair Value Measurement of Available-for-sale Securities | The following table is a summary of amortized cost, unrealized gain and loss, and fair value (in thousands) of the Company’s marketable securities by contractual maturities: Fair Value Measurements at December 31, 2019 Amortized Cost Unrealized Gain Unrealized Loss Fair Value Cash equivalents (due within 90 days) $ 100,440 $ 1 $ — $ 100,441 Short-term investments (due within one year) 314,181 523 (13 ) 314,691 Long-term investments (due between one and two years) 14,110 47 (4 ) 14,153 Total $ 428,731 $ 571 $ (17 ) $ 429,285 Fair Amortized Cost Unrealized Gain Unrealized Loss Fair Value Cash equivalents (due within 90 days) $ 245,194 $ — $ — $ 245,194 Short-term investments (due within one year) 68,656 — (92 ) 68,564 Long-term investments (due between one and two years) 80,118 98 (68 ) 80,148 Total $ 393,968 $ 98 $ (160 ) $ 393,906 |
Balance Sheet Components (Table
Balance Sheet Components (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Balance Sheet Related Disclosures [Abstract] | |
Summary of Property and Equipment | Property and equipment consists of the following (in thousands): December 31, 2019 2018 Scientific equipment $ 10,642 $ 9,126 Furniture and equipment 2,572 1,248 Capitalized software 389 302 Leasehold improvements 509 451 Construction in progress 9,568 — Total 23,680 11,127 Less: Accumulated depreciation (7,937 ) (5,989 ) Property and equipment, net $ 15,743 $ 5,138 |
Summary of Accrued Liabilities | Accrued liabilities consist of the following (in thousands): December 31, 2019 2018 Clinical trials accrued liabilities $ 11,494 $ 6,272 Outside services 6,592 4,631 Payroll-related liabilities 11,724 8,151 Construction in progress 9,139 — Other 2,343 1,704 Total accrued liabilities $ 41,292 $ 20,758 |
Leases (Tables)
Leases (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Lessee Disclosure [Abstract] | |
Schedule of Information Related to Operating Leases Up on ASC 842 | Information related to operating leases as of December 31, 2019 and upon adoption of ASC 842 on January 1, 2019 is as follows (in thousands, except for percentages and years): December 31, 2019 January 1, 2019 Assets Operating lease right-of-use assets $ 417 $ 1,940 Liabilities Operating lease liabilities - current $ 383 $ 2,126 Weighted average remaining lease term (years) 0.3 1.0 Weighted average discount rate 6 % 6 % |
Schedule of Information Related to Operating Lease Activity | Information related to operating lease activity during the year ended December 31, 2019 follows (in thousands): Year Ended December 31, 2019 Operating lease right-of-use assets obtained in exchange for lease obligations $ 1,095 Operating lease rental expense $ 2,760 Operating lease payments $ 3,007 |
Schedule of Future Annual Payment of Operating Lease Liabilities | Future annual payments of operating lease liabilities as of December 31, 2019 are as follows (in thousands): Year ending December 31: Amount 2020 387 Total future lease payments 387 Less: imputed interest (4 ) Total operating lease liabilities $ 383 Future annual minimum operating lease payments due under the Lease are as follows (in thousands): Year ending December 31: Amount 2020 5,454 2021 8,461 2022 8,757 2023 9,063 2024 9,381 Thereafter 52,063 Total $ 93,179 |
Stockholders' Equity (Tables)
Stockholders' Equity (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Equity [Abstract] | |
Schedule of Common Stock Reserved for Issuance | The Company has reserved shares of common stock, on an as-if-converted basis, for issuance as follows: December 31, 2019 December 31, 2018 Options and awards issued and outstanding 5,315,254 3,864,407 Shares available for issuance under 2015 Stock Option and Incentive Plan 685,435 904,785 Shares available for issuance under 2015 Employee Stock Purchase Plan 1,140,541 780,716 Total 7,141,230 5,549,908 |
Stock-Based Compensation (Table
Stock-Based Compensation (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Summary of Stock Option and Related Information | The following table summarizes stock option activity and related information for the periods presented below: Shares Subject to Outstanding Options Weighted Average Exercise Price Per Option Weighted Average Remaining Contractual Term (in years) Aggregate Intrinsic Value (in Balance at December 31, 2018 3,701,461 $ 26.40 Options granted 1,371,130 43.27 Options exercised (337,846 ) 15.78 Options canceled (160,587 ) 38.75 Balance at December 31, 2019 4,574,158 $ 31.81 7.7 $ 187,879 Exercisable at December 31, 2019 2,380,560 $ 23.21 7.0 $ 118,246 Vested and expected to vest at December 31, 2019 4,574,158 $ 31.81 7.7 $ 187,879 |
Schedule of Assumptions for Black-Scholes Option-Pricing Model Used in Determining Fair Value of Time-Based and Performance-Based Options Granted to Employees | The following table illustrates the assumptions for the Black-Scholes option-pricing model used in determining the fair value of time-based and performance-based options granted to employees: Year Ended December 31, 2019 2018 2017 Risk-free interest rate 1.59% - 2.54% 2.54% - 2.98% 1.92% - 2.27% Expected life (in years) 6.1% 5.3 - 6.1 5.3 - 6.1 Volatility 65% - 69% 69% - 75% 71% - 75% Dividend yield 0% 0% 0% |
Summary of RSU Activity and Related Information | The following table summarizes RSU activity and related information for the period presented below: Shares Subject to Outstanding Awards Weighted Average Grant Date Fair Value Weighted Average Remaining Contractual Term (in years) Aggregate Intrinsic Value (in Balance at December 31, 2018 162,946 $ 53.37 RSUs awarded 643,814 45.36 RSUs released (42,214 ) 53.08 RSUs forfeited (23,450 ) 48.23 Balance at December 31, 2019 741,096 $ 46.59 2.2 $ 54,015 |
Schedule of Stock-Based Compensation Expense, Net of Forfeitures | Stock-based compensation expense, net of forfeitures, as applicable for the years ended December 31, 2019, 2018 and 2017, is reflected in the statements of operations and comprehensive loss as follows (in thousands): Year Ended December 31, 2019 2018 2017 Research and development $ 14,673 $ 8,144 $ 2,752 Selling, general and administrative $ 18,321 11,197 3,386 Total stock-based compensation $ 32,994 $ 19,341 $ 6,138 |
Net Loss per Share (Tables)
Net Loss per Share (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Earnings Per Share [Abstract] | |
Computation of Basic and Diluted Net Loss per Share | The following table sets forth the computation of basic and diluted net loss per share (in thousands, except share and per share data): Year Ended December 31, 2019 2018 2017 Numerator Net loss $ (276,213 ) $ (67,698 ) $ (57,010 ) Denominator Weighted average shares outstanding 44,767,581 38,466,233 33,098,571 Less: weighted average shares subject to repurchase (2,085 ) (79,327 ) (266,057 ) Weighted average shares used to compute basic and diluted net loss per share 44,765,496 38,386,906 32,832,514 Net loss per share, basic and diluted $ (6.17 ) $ (1.76 ) $ (1.74 ) |
Summary of Potentially Dilutive Securities Excluded from Computation of Diluted Net Loss per Share | The following outstanding shares of potentially dilutive securities were excluded from the computation of diluted net loss per share for the periods presented because including them would have been antidilutive: As of December 31, 2019 2018 2017 Common stock subject to repurchase — 9,790 81,373 Options and awards issued and outstanding 5,315,254 3,864,407 2,964,549 |
Income Taxes (Tables)
Income Taxes (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Income Tax Disclosure [Abstract] | |
Schedule of Reconciliation between Statutory Federal Income Tax Rate and Effective Tax Rate | The following is the reconciliation between the statutory federal income tax rate and the Company’s effective tax rate: Year Ended December 31, 2019 2018 2017 Federal statutory income tax rate 21.0 % 21.0 % 34.0 % State taxes (tax effected) 7.3 8.6 7.1 Non-deductible expenses and other (0.5 ) 2.5 2.0 Research and development reimbursements 3.2 5.9 6.0 Change in valuation allowance (31.0 ) (38.0 ) (24.0 ) Tax Act – net deferred tax rate change — — (25.1 ) Total — % — % — % |
Components of Company's Deferred Tax Assets | As of December 31, 2019 and 2018, the components of the Company’s deferred tax assets are as follows (in thousands): As of December 31, 2019 2018 Deferred tax assets: Net operating loss carryforwards 98,445 49,689 Research and development reimbursement carryforwards 23,061 12,908 Stock-based compensation 8,861 4,349 Start-up costs 22,896 1,237 Depreciation (1,027 ) (959 ) Other 2,646 1,858 Total deferred tax assets 154,882 69,082 Less: valuation allowance (154,882 ) (69,082 ) Net deferred tax assets — — |
Schedule of Reconciliation of Beginning and Ending Amount of Unrecognized Tax Benefits | A reconciliation of the beginning and ending amount of unrecognized tax benefits is as follows (in thousands): For the Year Ended December 31, 2019 2018 2017 Beginning balance $ 3,810 $ 2,471 $ 1,474 Increases (decreases) of unrecognized tax benefits related to prior year 380 289 (97 ) Increases of unrecognized tax benefits related to current year 3,314 1,050 1,094 Ending balance $ 7,504 $ 3,810 $ 2,471 |
Quarterly Financial Data (una_2
Quarterly Financial Data (unaudited) (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Quarterly Financial Data [Abstract] | |
Summary of Unaudited Quarterly Financial Data | The following table summarizes the unaudited quarterly financial data for the last two fiscal years (in thousands, except per share data): (in thousands, except share and per share amounts) First Second Third Fourth Quarter Quarter Quarter Quarter 2019 Revenues $ — $ — $ — $ — Total operating expenses $ 39,741 $ 41,564 $ 145,118 $ 61,411 Net loss $ (37,470 ) $ (38,174 ) $ (141,802 ) $ (58,767 ) Net loss per common share, basic and diluted $ (0.93 ) $ (0.83 ) $ (3.07 ) $ (1.27 ) Weighted average number of shares, basic and diluted 40,506,313 46,065,901 46,133,068 46,278,409 2018 Revenues $ 5,331 $ 6,639 $ 9,188 $ 12,400 Total operating expenses $ 23,931 $ 26,130 $ 26,867 $ 30,281 Net loss $ (17,820 ) $ (18,413 ) $ (15,789 ) $ (15,676 ) Net loss per common share, basic and diluted $ (0.50 ) $ (0.49 ) $ (0.39 ) $ (0.39 ) Weighted average number of shares, basic and diluted 35,827,235 37,440,024 40,116,644 40,259,575 |
Organization - Additional Infor
Organization - Additional Information (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | ||
Date of incorporation | Jun. 8, 2012 | |
Accumulated deficit | $ 478,766 | $ 202,553 |
Cash, cash equivalents and short and long-term investments | $ 430,300 |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies - Additional Information (Details) | 3 Months Ended | 12 Months Ended | ||||||
Dec. 31, 2018USD ($) | Sep. 30, 2018USD ($) | Jun. 30, 2018USD ($) | Mar. 31, 2018USD ($) | Dec. 31, 2019USD ($)Segment | Dec. 31, 2018USD ($) | Dec. 31, 2017USD ($) | Jan. 01, 2019USD ($) | |
Summary Of Significant Accounting Policies [Line Items] | ||||||||
Number of reportable and operating segment | Segment | 1 | |||||||
Revenue recognized | $ 12,400,000 | $ 9,188,000 | $ 6,639,000 | $ 5,331,000 | $ 0 | $ 33,558,000 | $ 11,442,000 | |
Operating lease, liability | 383,000 | |||||||
Deferred rent | $ 200,000 | $ 200,000 | ||||||
Operating lease right-of-use assets | $ 417,000 | $ 1,940,000 | ||||||
ASU 842 | ||||||||
Summary Of Significant Accounting Policies [Line Items] | ||||||||
Operating lease, liability | 2,100,000 | |||||||
Operating lease, Right of use asset, before reclassification | 2,100,000 | |||||||
Operating lease right-of-use assets | $ 1,900,000 |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies - Summary of Cash Reported in Consolidated Statements of Cash Flows (Details) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 |
Accounting Policies [Abstract] | ||||
Cash and cash equivalents | $ 101,436 | $ 246,122 | ||
Restricted cash included in prepaid expenses and other current assets | 337 | |||
Restricted cash included in restricted cash and other | 1,857 | 2,143 | ||
Total cash, cash equivalents and restricted cash shown in the consolidated statements of cash flows | $ 103,630 | $ 248,265 | $ 224,857 | $ 136,057 |
Collaboration and License Rev_3
Collaboration and License Revenue - Additional Information (Details) - USD ($) | 1 Months Ended | 12 Months Ended | 24 Months Ended | |||
Jul. 31, 2019 | Jan. 31, 2017 | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2018 | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||
Repurchase of royalty rights | $ 80,000,000 | |||||
Deferred revenue | $ 33,558,000 | |||||
Contract assets | 0 | |||||
Contract liabilities | 0 | |||||
Collaborative Agreement | Sanofi (Aventis Inc.) | ||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||
Non-refundable continuation payment received | $ 45,000,000 | |||||
Revenue recognized on pro-rata basis to research and development effort | $ 45,000,000 | |||||
Continuation payment, amount received | $ 45,000,000 | |||||
Fixed fee | 45,000,000 | |||||
Transaction price for extended term | 45,000,000 | |||||
Collaboration and license revenue | 0 | $ 33,600,000 | 11,400,000 | |||
Deferred revenue | 0 | |||||
Reduction in research and development expenses due to RPP reimbursements | $ 18,500,000 | $ 23,100,000 | $ 7,300,000 | |||
Collaborative Agreement | Sanofi (Aventis Inc.) | Maximum | ||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||
Funding from approved in-kind research and clinical activities | $ 45,000,000 | |||||
Termination Agreement | Sanofi (Aventis Inc.) | Royalty Rights | ||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||
Payment received for research and development | $ 80,000,000 | |||||
Repurchase of royalty rights | 50,000,000 | |||||
Transferred to escrow | 30,000,000 | |||||
Research and development asset reacquired | $ 4,300,000 |
Collaboration and License Rev_4
Collaboration and License Revenue - Changes in Contract Assets and Liabilities (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Contract liabilities: | ||
Deferred revenue, Balance at Beginning of Period | $ 33,558 | |
Deferred revenue, Deductions | $ (33,558) | $ (11,442) |
Deferred revenue, Balance at End of Period | $ 33,558 |
Collaboration and License Rev_5
Collaboration and License Revenue - Schedule of Prepayments and Reimbursed Research and Development Expenses (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||
Balance at beginning of year | $ 12,973 | ||
Balance at end of year | $ 12,973 | ||
Collaborative Agreement | Aventis Inc | |||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||
Balance at beginning of year | 12,973 | 4,432 | |
Additions for advance billings | $ 1,013 | ||
Payments received from Sanofi | 5,521 | 31,659 | 10,697 |
Actual expenses incurred | $ (18,494) | (23,118) | (7,278) |
Balance at end of year | $ 12,973 | $ 4,432 |
Fair Value Measurements - Summa
Fair Value Measurements - Summary of Fair Value Assets Measured on Recurring Basis (Details) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Assets | ||
Assets fair value | $ 429,285 | $ 393,906 |
Money market funds | ||
Assets | ||
Assets fair value | 100,441 | 245,194 |
Fair Value Measurements on Recurring Basis | Level 1 | ||
Assets | ||
Assets fair value | 100,441 | 245,194 |
Fair Value Measurements on Recurring Basis | Level 2 | ||
Assets | ||
Assets fair value | 328,844 | 148,712 |
Fair Value Measurements on Recurring Basis | Money market funds | Level 1 | ||
Assets | ||
Assets fair value | 100,441 | 245,194 |
U.S. government agency obligations | ||
Assets | ||
Assets fair value | 134,055 | 85,033 |
U.S. government agency obligations | Fair Value Measurements on Recurring Basis | Level 2 | ||
Assets | ||
Assets fair value | 134,055 | 85,033 |
Corporate securities | ||
Assets | ||
Assets fair value | 194,789 | 63,679 |
Corporate securities | Fair Value Measurements on Recurring Basis | Level 2 | ||
Assets | ||
Assets fair value | $ 194,789 | $ 63,679 |
Fair Value Measurements - Sum_2
Fair Value Measurements - Summary of Fair Value Measurement of Available-for-sale Securities (Details) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | $ 428,731 | $ 393,968 |
Unrealized Gain | 571 | 98 |
Unrealized Loss | (17) | (160) |
Fair Value | 429,285 | 393,906 |
Cash equivalents (due within 90 days) | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 100,440 | 245,194 |
Unrealized Gain | 1 | |
Fair Value | 100,441 | 245,194 |
Short-term investments (due within one year) | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 314,181 | 68,656 |
Unrealized Gain | 523 | |
Unrealized Loss | (13) | (92) |
Fair Value | 314,691 | 68,564 |
Long-term investments (due between one and two years) | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 14,110 | 80,118 |
Unrealized Gain | 47 | 98 |
Unrealized Loss | (4) | (68) |
Fair Value | $ 14,153 | $ 80,148 |
Fair Value Measurements - Addit
Fair Value Measurements - Additional Information (Details) - USD ($) | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Fair Value Disclosures [Abstract] | ||
Transfers between level 1 to level 2 | $ 0 | $ 0 |
Transfers between level 2 to level 1 | 0 | 0 |
Realized losses on available-for-sale securities | 0 | 0 |
Realized gains on available-for-sale securities | $ 0 | $ 0 |
Balance Sheet Components - Addi
Balance Sheet Components - Additional Information (Details) - USD ($) | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Property Plant And Equipment [Line Items] | |||
Property plant and equipment basis of valuation | cost | ||
Property plant and equipment depreciation methods | the straight-line method over the estimated useful lives of the assets | ||
Depreciation expense | $ 1,948,000 | $ 1,567,000 | $ 1,294,000 |
Impairment of long-lived assets | $ 0 | ||
Minimum | |||
Property Plant And Equipment [Line Items] | |||
Property plant and equipment useful life | 2 years | ||
Maximum | |||
Property Plant And Equipment [Line Items] | |||
Property plant and equipment useful life | 5 years | ||
Leasehold improvements | |||
Property Plant And Equipment [Line Items] | |||
Property plant and equipment estimated useful lives | shorter of their estimated useful lives or the related lease term |
Balance Sheet Components - Summ
Balance Sheet Components - Summary of Property and Equipment (Details) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Property Plant And Equipment [Line Items] | ||
Property and equipment | $ 23,680 | $ 11,127 |
Less: Accumulated depreciation | (7,937) | (5,989) |
Property and equipment, net | 15,743 | 5,138 |
Scientific Equipment | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment | 10,642 | 9,126 |
Furniture and Equipment | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment | 2,572 | 1,248 |
Capitalized Software | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment | 389 | 302 |
Leasehold Improvements | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment | 509 | $ 451 |
Construction in Progress | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment | $ 9,568 |
Balance Sheet Components - Su_2
Balance Sheet Components - Summary of Accrued Liabilities (Details) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Balance Sheet Related Disclosures [Abstract] | ||
Clinical trials accrued liabilities | $ 11,494 | $ 6,272 |
Outside services | 6,592 | 4,631 |
Payroll-related liabilities | 11,724 | 8,151 |
Construction in progress | 9,139 | |
Other | 2,343 | 1,704 |
Total accrued liabilities | $ 41,292 | $ 20,758 |
Leases - Additional Information
Leases - Additional Information (Details) $ in Millions | 1 Months Ended | 12 Months Ended | ||
Sep. 30, 2018USD ($)ft² | Dec. 31, 2019USD ($)ft²Facility | Dec. 31, 2018USD ($) | Dec. 31, 2017USD ($) | |
Lessee Lease Description [Line Items] | ||||
Operating lease rent expense | $ 2.8 | $ 2.1 | $ 1.4 | |
New Facility | ||||
Lessee Lease Description [Line Items] | ||||
Area of Real Estate Property | ft² | 129,800 | |||
Rent commencement date | 2020-01 | |||
Lease period | 10 years | 10 years | ||
Additional period of extension in lease contract | 10 years | |||
Aggregate future minimum rental payments due | $ 93.2 | |||
Standby letter of credit | $ 1.9 | |||
New Facility | Construction in Progress | ||||
Lessee Lease Description [Line Items] | ||||
Tenant improvements capitalized | $ 9.1 | |||
Existing Facilities | ||||
Lessee Lease Description [Line Items] | ||||
Area of Real Estate Property | ft² | 56,500 | |||
Facilities available for operating lease | Facility | 2 | |||
Lease expiration date | Apr. 30, 2020 |
Leases - Schedule of Informatio
Leases - Schedule of Information Related to Operating Leases Up on Adoption of ASC 842 (Details) - USD ($) $ in Thousands | Dec. 31, 2019 | Jan. 01, 2019 |
Operating Leases [Abstract] | ||
Operating lease right-of-use assets | $ 417 | $ 1,940 |
Operating lease liabilities - current | $ 383 | $ 2,126 |
Weighted average remaining lease term (years) | 3 months 18 days | 1 year |
Weighted average discount rate | 6.00% | 6.00% |
Leases - Schedule of Informat_2
Leases - Schedule of Information Related to Operating Lease Activity (Details) $ in Thousands | 12 Months Ended |
Dec. 31, 2019USD ($) | |
Lease Cost [Abstract] | |
Operating lease right-of-use assets obtained in exchange for lease obligations | $ 1,095 |
Operating lease rental expense | 2,760 |
Operating lease payments | $ 3,007 |
Leases - Future Annual Payments
Leases - Future Annual Payments of Operating Lease Liabilities (Details) $ in Thousands | Dec. 31, 2019USD ($) |
Operating Lease Liabilities Payments Due [Abstract] | |
2020 | $ 387 |
Total future lease payments | 387 |
Less: imputed interest | (4) |
Total operating lease liabilities | $ 383 |
Leases - Schedule of Future Ann
Leases - Schedule of Future Annual Minimum Operating Lease Payments Due Under Leases for the New Facility (Details) $ in Thousands | Dec. 31, 2019USD ($) |
Lessee Lease Description [Line Items] | |
2020 | $ 387 |
Total future lease payments | 387 |
New Facility | |
Lessee Lease Description [Line Items] | |
2020 | 5,454 |
2021 | 8,461 |
2022 | 8,757 |
2023 | 9,063 |
2024 | 9,381 |
Thereafter | 52,063 |
Total future lease payments | $ 93,179 |
Commitments and Contingencies -
Commitments and Contingencies - Additional Information (Details) - USD ($) | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Commitments And Contingencies Disclosure [Abstract] | ||
Purchase commitment cancellation notice period | 30 days | |
Contingent liability for accrual | $ 0 | $ 0 |
Stockholders' Equity - Addition
Stockholders' Equity - Additional Information (Details) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | 12 Months Ended | ||
Mar. 31, 2019 | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Class Of Stock [Line Items] | ||||
Proceeds from offering, net of underwriting discounts and commissions and offering expenses | $ 271,224 | $ 181,863 | $ 133,862 | |
Preferred stock, shares authorized | 5,000,000 | 5,000,000 | ||
Preferred stock, par value | $ 0.0001 | $ 0.0001 | ||
Preferred stock, shares issued | 0 | 0 | ||
Preferred stock, shares outstanding | 0 | 0 | ||
Follow-On-Offering | ||||
Class Of Stock [Line Items] | ||||
Common stock shares issued | 5,663,750 | |||
Common stock shares issued, price per share | $ 51 | |||
Stock repurchase offering period | 30 days | |||
Proceeds from offering, net of underwriting discounts and commissions and offering expenses | $ 271,200 | |||
Over Allotment Option | ||||
Class Of Stock [Line Items] | ||||
Common stock shares issued | 738,750 |
Stockholders' Equity - Schedule
Stockholders' Equity - Schedule of Common Stock Reserved for Issuance (Details) - shares | Dec. 31, 2019 | Jan. 01, 2019 | Dec. 31, 2018 |
Class Of Stock [Line Items] | |||
Shares reserved for future issuance, shares | 7,141,230 | 5,549,908 | |
Options and Awards Issued and Outstanding | |||
Class Of Stock [Line Items] | |||
Shares reserved for future issuance, shares | 5,315,254 | 3,864,407 | |
2015 Stock Option and Incentive Plan | |||
Class Of Stock [Line Items] | |||
Shares reserved for future issuance, shares | 685,435 | 1,611,557 | 904,785 |
2015 Employee Stock Purchase Plan | |||
Class Of Stock [Line Items] | |||
Shares reserved for future issuance, shares | 1,140,541 | 780,716 |
Stock-Based Compensation - Addi
Stock-Based Compensation - Additional Information (Details) - USD ($) | 1 Months Ended | 12 Months Ended | ||||||
Oct. 31, 2015 | Jun. 30, 2012 | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | Jan. 01, 2020 | Jan. 01, 2019 | Dec. 31, 2016 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Shares reserved for issuance, shares | 7,141,230 | 5,549,908 | ||||||
Aggregate intrinsic value of options exercised | $ 14,300,000 | $ 21,100,000 | $ 9,800,000 | |||||
Weighted average grant date fair value of options granted | $ 27.02 | $ 34.70 | $ 9.92 | |||||
Unamortized stock-based compensation | $ 55,300,000 | |||||||
Stock option average expected recognition period | 2 years 4 months 24 days | |||||||
Unrecognized share based compensation expense | $ 340,000 | $ 248,000 | $ 174,000 | |||||
Stock Options | ||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Shares reserved for issuance, shares | 5,315,254 | 3,864,407 | ||||||
RSU | ||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Stock option average expected recognition period | 3 years 1 month 6 days | |||||||
Weighted average grant date fair value of RSUs granted | $ 45.36 | $ 53.27 | ||||||
Total fair value vested | $ 2,000,000 | $ 167,000 | $ 0 | |||||
Unamortized stock-based compensation relating to RSUs | $ 27,900,000 | |||||||
2012 Equity Incentive Plan | Incentive Stock Options | Minimum | ||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Percentage of estimated fair value of common stock | 100.00% | |||||||
2012 Equity Incentive Plan | Nonstatutory Stock Options | Minimum | ||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Percentage of estimated fair value of common stock | 85.00% | |||||||
2012 Equity Incentive Plan | Stock Options | ||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Terms of options granted | 10 years | |||||||
Vesting period | 4 years | |||||||
2012 Equity Incentive Plan | Stock Options | Minimum | ||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Percentage of estimated fair value of common stock | 110.00% | |||||||
2012 Equity Incentive Plan | Stock Options | Maximum | ||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Employee owning voting rights of stock options | 10.00% | |||||||
2015 Stock Option and Incentive Plan | ||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Vesting period | 4 years | |||||||
Shares reserved for issuance, shares | 685,435 | 904,785 | 1,611,557 | |||||
Number of shares reserved for issuance under the option plan, description | (i) the number of shares represented by awards outstanding under the Company’s 2012 Equity Incentive Plan that are forfeited or lapse unexercised and which following the pricing date are not issued under the 2012 Plan, and (ii) an annual increase on January 1 of each year beginning on January 1, 2017. | |||||||
2015 Stock Option and Incentive Plan | Subsequent Event | ||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Shares reserved for issuance, shares | 1,855,162 | |||||||
2015 Stock Option and Incentive Plan | IPO | ||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Shares reserved for issuance, shares | 1,650,000 | |||||||
2015 Equity Incentive Plan | Employee Stock Purchase Plan | ||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Percentage of estimated fair value of common stock | 85.00% | |||||||
Shares reserved for issuance, shares | 255,000 | 402,889 | ||||||
Number of common stock purchased by one employee | 2,500 | |||||||
2015 Equity Incentive Plan | Employee Stock Purchase Plan | Subsequent Event | ||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Shares reserved for issuance, shares | 463,790 |
Stock-Based Compensation - Summ
Stock-Based Compensation - Summary of Stock Option and Related Information (Details) $ / shares in Units, $ in Thousands | 12 Months Ended |
Dec. 31, 2019USD ($)$ / sharesshares | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Shares Subject to Outstanding Options, Beginning Balance | shares | 3,701,461 |
Shares Subject to Outstanding Options, granted | shares | 1,371,130 |
Shares Subject to Outstanding Options, exercised | shares | (337,846) |
Shares Subject to Outstanding Options, canceled | shares | (160,587) |
Shares Subject to Outstanding Options, Ending Balance | shares | 4,574,158 |
Shares Subject to Outstanding Options, Exercisable | shares | 2,380,560 |
Shares Subject to Outstanding Options, Vested and expected to vest | shares | 4,574,158 |
Weighted Average Exercise Price per Share, Beginning Balance | $ / shares | $ 26.40 |
Weighted Average Exercise Price Per Share, granted | $ / shares | 43.27 |
Weighted Average Exercise Price per Share, exercised | $ / shares | 15.78 |
Weighted Average Exercise Price Per Share, cancelled | $ / shares | 38.75 |
Weighted Average Exercise Price per Share, Ending Balance | $ / shares | 31.81 |
Weighted Average Exercise Price Per Share, Exercisable | $ / shares | 23.21 |
Weighted Average Exercise Price Per Share, Vested and expected to vest | $ / shares | $ 31.81 |
Weighted Average Remaining Contractual Term | 7 years 8 months 12 days |
Weighted Average Remaining Contractual Term, Exercisable | 7 years |
Weighted Average Remaining Contractual Term, Vested and expected to vest | 7 years 8 months 12 days |
Aggregate Intrinsic Value, Beginning Balance | $ | $ 187,879 |
Aggregate Intrinsic Value, Exercisable | $ | 118,246 |
Aggregate Intrinsic Value, Vested and expected to vest | $ | $ 187,879 |
Stock-Based Compensation - Sche
Stock-Based Compensation - Schedule of Assumptions for Black-Scholes Option-Pricing Model Used in Determining Fair Value of Time-Based and Performance-Based Options Granted to Employees (Details) | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||
Risk-free interest rate, minimum | 1.59% | 2.54% | 1.92% |
Expected life (in years) | 6 years 1 month 6 days | 5 years 3 months 18 days | 5 years 3 months 18 days |
Volatility, minimum | 65.00% | 69.00% | 71.00% |
Dividend yield | 0.00% | 0.00% | 0.00% |
Maximum | |||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||
Risk-free interest rate, maximum | 2.54% | 2.98% | 2.27% |
Expected life (in years) | 6 years 1 month 6 days | 6 years 1 month 6 days | |
Volatility, maximum | 69.00% | 75.00% | 75.00% |
Stock-Based Compensation - Su_2
Stock-Based Compensation - Summary of RSU Activity and Related Information (Details) - Restricted Stock Units $ / shares in Units, $ in Thousands | 12 Months Ended |
Dec. 31, 2019USD ($)$ / sharesshares | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
Shares Subject to Outstanding Awards, Beginning Balance | shares | 162,946 |
Shares Subject to Outstanding Awards, RSUs awarded | shares | 643,814 |
Shares Subject to Outstanding Awards, RSUs released | shares | (42,214) |
Shares Subject to Outstanding Awards, RSUs forfeited | shares | (23,450) |
Shares Subject to Outstanding Awards, Ending Balance | shares | 741,096 |
Weighted Average Grant Date Fair Value. Beginning Balance | $ / shares | $ 53.37 |
Weighted Average Grant Date Fair Value. RSUs awarded | $ / shares | 45.36 |
Weighted Average Grant Date Fair Value. RSUs released | $ / shares | 53.08 |
Weighted Average Grant Date Fair Value. RSUs forfeited | $ / shares | 48.23 |
Weighted Average Grant Date Fair Value. Ending Balance | $ / shares | $ 46.59 |
Weighted Average Remaining Contractual Term | 2 years 2 months 12 days |
Aggregate Intrinsic Value | $ | $ 54,015 |
Stock-Based Compensation - Sc_2
Stock-Based Compensation - Schedule of Stock-Based Compensation Expense, Net of Forfeitures (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | |||
Stock-based compensation expense | $ 32,994 | $ 19,341 | $ 6,138 |
Research and development | |||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | |||
Stock-based compensation expense | 14,673 | 8,144 | 2,752 |
Selling, general and administrative | |||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | |||
Stock-based compensation expense | $ 18,321 | $ 11,197 | $ 3,386 |
Net Loss per Share - Computatio
Net Loss per Share - Computation of Basic and Diluted Net Loss per Share (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Numerator | |||||||||||
Net loss | $ (276,213) | $ (67,698) | $ (57,010) | ||||||||
Denominator | |||||||||||
Weighted average shares outstanding | 44,767,581 | 38,466,233 | 33,098,571 | ||||||||
Less: weighted average shares subject to repurchase | (2,085) | (79,327) | (266,057) | ||||||||
Weighted average shares used to compute basic and diluted net loss per share | 46,278,409 | 46,133,068 | 46,065,901 | 40,506,313 | 40,259,575 | 40,116,644 | 37,440,024 | 35,827,235 | 44,765,496 | 38,386,906 | 32,832,514 |
Net loss per share, basic and diluted | $ (1.27) | $ (3.07) | $ (0.83) | $ (0.93) | $ (0.39) | $ (0.39) | $ (0.49) | $ (0.50) | $ (6.17) | $ (1.76) | $ (1.74) |
Net Loss per Share - Summary of
Net Loss per Share - Summary of Potentially Dilutive Securities Excluded from Computation of Diluted Net Loss per Share (Details) - shares | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Common stock subject to repurchase | |||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | |||
Antidilutive securities excluded from earnings per share | 9,790 | 81,373 | |
Options and Awards Issued and Outstanding | |||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | |||
Antidilutive securities excluded from earnings per share | 5,315,254 | 3,864,407 | 2,964,549 |
Net Loss per Share - Additional
Net Loss per Share - Additional Information (Details) - 2015 Employee Stock Purchase Plan - USD ($) $ in Millions | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Earnings Per Share [Line Items] | ||
ESPP contributions from plan participants | $ 0.4 | $ 0.3 |
Contributions converted shares under benefit plan | 10,000 | 5,700 |
Percentage of stock price at the beginning of offering period | 85.00% | 85.00% |
Income Taxes - Additional Infor
Income Taxes - Additional Information (Details) - USD ($) | 12 Months Ended | |||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Income Taxes [Line Items] | ||||
Effective income tax rate and tax provision from continuing operations | 0.00% | 0.00% | 0.00% | |
Deferred tax assets, relates to net operating loss carryforwards | $ 98,445,000 | $ 49,689,000 | ||
Deferred tax assets, operating loss foreign | 351,100,000 | |||
Deferred tax assets, operating loss state | $ 354,000,000 | |||
Operating loss expiration date | 2032 | |||
Research and development tax credit | $ 23,061,000 | 12,908,000 | ||
Increase in valuation allowance | $ 85,800,000 | 25,700,000 | $ 13,700,000 | |
Time period over percentage change of ownership | 3 years | |||
Unrecognized tax benefits related to liability | $ 0 | 0 | ||
Unrecognized tax benefits related to penalties or interest accrued | 0 | 0 | ||
Unrecognized tax benefits that would impact effective tax rate | $ 0 | |||
Minimum percentage of likelihood of realization of tax benefits | 50.00% | |||
Reserve for unrecognized tax benefits | $ 7,504,000 | $ 3,810,000 | $ 2,471,000 | $ 1,474,000 |
Internal Revenue Service | ||||
Income Taxes [Line Items] | ||||
Operating loss carryforwards adjustments period | 3 years | |||
California Franchise Tax Board | ||||
Income Taxes [Line Items] | ||||
Operating loss carryforwards adjustments period | 4 years | |||
Australia | ||||
Income Taxes [Line Items] | ||||
Uncertain tax positions statute of limitation | 4 years | |||
Minimum | ||||
Income Taxes [Line Items] | ||||
Change of ownership percentage in equity ownership | 50.00% | |||
Federal | ||||
Income Taxes [Line Items] | ||||
Research and development tax credit | $ 7,200,000 | |||
Research and development tax credit carryforward, expiration year | 2032 | |||
Orphan tax credit carryovers, expiration beginning year | 2036 | |||
Orphan tax credit carryovers | $ 18,200,000 | |||
State | ||||
Income Taxes [Line Items] | ||||
Research and development tax credit | $ 5,900,000 |
Income Taxes - Schedule of Reco
Income Taxes - Schedule of Reconciliation between Statutory Federal Income Tax Rate and Effective Tax Rate (Details) | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Income Tax Disclosure [Abstract] | |||
Federal statutory income tax rate | 21.00% | 21.00% | 34.00% |
State taxes (tax effected) | 7.30% | 8.60% | 7.10% |
Non-deductible expenses and other | (0.50%) | 2.50% | 2.00% |
Research and development reimbursements | 3.20% | 5.90% | 6.00% |
Change in valuation allowance | (31.00%) | (38.00%) | (24.00%) |
Tax Act – net deferred tax rate change | (25.10%) | ||
Total | 0.00% | 0.00% | 0.00% |
Income Taxes - Schedule of Sign
Income Taxes - Schedule of Significant Deferred Tax Assets (Details) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Deferred tax assets: | ||
Net operating loss carryforwards | $ 98,445 | $ 49,689 |
Research and development reimbursement carryforwards | 23,061 | 12,908 |
Stock-based compensation | 8,861 | 4,349 |
Start-up costs | 22,896 | 1,237 |
Depreciation | (1,027) | (959) |
Other | 2,646 | 1,858 |
Total deferred tax assets | 154,882 | 69,082 |
Less: valuation allowance | $ (154,882) | $ (69,082) |
Income Taxes - Reconciliation o
Income Taxes - Reconciliation of Beginning and Ending Amount of Unrecognized Tax Benefits (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Income Tax Disclosure [Abstract] | |||
Beginning balance | $ 3,810 | $ 2,471 | $ 1,474 |
Increases (decreases) of unrecognized tax benefits related to prior year | 380 | 289 | (97) |
Increases of unrecognized tax benefits related to current year | 3,314 | 1,050 | 1,094 |
Ending balance | $ 7,504 | $ 3,810 | $ 2,471 |
Quarterly Financial Data (una_3
Quarterly Financial Data (unaudited) - Summary of Unaudited Quarterly Financial Data (Details) - USD ($) | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Quarterly Financial Data [Abstract] | |||||||||||
Revenues | $ 12,400,000 | $ 9,188,000 | $ 6,639,000 | $ 5,331,000 | $ 0 | $ 33,558,000 | $ 11,442,000 | ||||
Total operating expenses | $ 61,411,000 | $ 145,118,000 | $ 41,564,000 | $ 39,741,000 | 30,281,000 | 26,867,000 | 26,130,000 | 23,931,000 | 287,834,000 | 107,209,000 | 70,109,000 |
Net loss | $ (58,767,000) | $ (141,802,000) | $ (38,174,000) | $ (37,470,000) | $ (15,676,000) | $ (15,789,000) | $ (18,413,000) | $ (17,820,000) | $ (276,213,000) | $ (67,698,000) | $ (57,010,000) |
Net loss per common share, basic and diluted | $ (1.27) | $ (3.07) | $ (0.83) | $ (0.93) | $ (0.39) | $ (0.39) | $ (0.49) | $ (0.50) | $ (6.17) | $ (1.76) | $ (1.74) |
Weighted average number of shares, basic and diluted | 46,278,409 | 46,133,068 | 46,065,901 | 40,506,313 | 40,259,575 | 40,116,644 | 37,440,024 | 35,827,235 | 44,765,496 | 38,386,906 | 32,832,514 |