Document and Entity Information
Document and Entity Information - USD ($) | 12 Months Ended | ||
Dec. 31, 2018 | Feb. 25, 2019 | Jun. 30, 2018 | |
Document And Entity Information [Abstract] | |||
Document Type | 10-K | ||
Amendment Flag | false | ||
Document Period End Date | Dec. 31, 2018 | ||
Document Fiscal Year Focus | 2,018 | ||
Document Fiscal Period Focus | FY | ||
Trading Symbol | MYOK | ||
Entity Registrant Name | MyoKardia Inc | ||
Entity Central Index Key | 1,552,451 | ||
Current Fiscal Year End Date | --12-31 | ||
Entity Well-known Seasoned Issuer | No | ||
Entity Current Reporting Status | Yes | ||
Entity Voluntary Filers | No | ||
Entity Filer Category | Large Accelerated Filer | ||
Entity Small Business | false | ||
Entity Emerging Growth Company | false | ||
Entity Ex Transition Period | false | ||
Entity Shell Company | false | ||
Entity Common Stock, Shares Outstanding | 40,319,855 | ||
Entity Public Float | $ 1,944,784,343 |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) $ in Thousands | Dec. 31, 2018 | Dec. 31, 2017 |
Current assets | ||
Cash and cash equivalents | $ 246,122 | $ 224,571 |
Short-term investments | 68,564 | 31,933 |
Receivable from collaboration partner | 1,013 | |
Prepaid expenses and other current assets | 4,760 | 1,876 |
Total current assets | 319,446 | 259,393 |
Property and equipment, net | 5,138 | 3,147 |
Long-term investments | 80,148 | 19,900 |
Restricted cash and other | 2,521 | 368 |
Total assets | 407,253 | 282,808 |
Current liabilities | ||
Accounts payable | 2,946 | 2,301 |
Accrued liabilities | 20,758 | 11,639 |
Prepayment from collaboration partner | 12,973 | 4,432 |
Deferred revenue | 33,558 | |
Total current liabilities | 36,677 | 51,930 |
Other long-term liabilities | 9 | 202 |
Total liabilities | 36,686 | 52,132 |
Commitments and contingencies (Note 6) | ||
Stockholders’ equity | ||
Preferred stock, $0.0001 par value; 5,000,000 shares authorized; none issued and outstanding | ||
Common stock, $0.0001 par value, 150,000,000 shares authorized at December 31, 2018 and 2017; 40,288,949 and 35,812,791 shares issued and outstanding at December 31, 2018 and 2017, respectively | 4 | 4 |
Additional paid-in capital | 573,183 | 365,719 |
Accumulated other comprehensive loss | (67) | (192) |
Accumulated deficit | (202,553) | (134,855) |
Total stockholders’ equity | 370,567 | 230,676 |
Total liabilities and stockholders’ equity | $ 407,253 | $ 282,808 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - $ / shares | Dec. 31, 2018 | Dec. 31, 2017 |
Statement Of Financial Position [Abstract] | ||
Preferred stock, par value | $ 0.0001 | $ 0.0001 |
Preferred stock, shares authorized | 5,000,000 | 5,000,000 |
Preferred stock, shares issued | 0 | 0 |
Preferred stock, shares outstanding | 0 | 0 |
Common stock, par value | $ 0.0001 | $ 0.0001 |
Common stock, shares authorized | 150,000,000 | 150,000,000 |
Common stock, shares issued | 40,288,949 | 35,812,791 |
Common stock, shares outstanding | 40,288,949 | 35,812,791 |
Consolidated Statements of Oper
Consolidated Statements of Operations and Comprehensive Loss - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2017 | Sep. 30, 2017 | Jun. 30, 2017 | Mar. 31, 2017 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Income Statement [Abstract] | |||||||||||
Collaboration and license revenue | $ 33,558 | $ 11,442 | $ 41,971 | ||||||||
Operating expenses: | |||||||||||
Research and development, net | 68,774 | 48,136 | 36,215 | ||||||||
General and administrative | 38,435 | 21,973 | 16,289 | ||||||||
Total operating expenses | $ 30,281 | $ 26,867 | $ 26,130 | $ 23,931 | $ 13,700 | $ 20,245 | $ 18,771 | $ 17,393 | 107,209 | 70,109 | 52,504 |
Loss from operations | (73,651) | (58,667) | (10,533) | ||||||||
Interest and other income, net | 5,953 | 1,657 | 153 | ||||||||
Net loss | (67,698) | (57,010) | (10,380) | ||||||||
Other comprehensive income (loss) | 125 | (200) | 8 | ||||||||
Comprehensive loss | $ (67,573) | $ (57,210) | $ (10,372) | ||||||||
Net loss per share, basic and diluted | $ (0.39) | $ (0.39) | $ (0.49) | $ (0.50) | $ (0.28) | $ (0.50) | $ (0.50) | $ (0.47) | $ (1.76) | $ (1.74) | $ (0.38) |
Weighted average number of shares used to compute net loss per share, basic and diluted | 40,259,575 | 40,116,644 | 37,440,024 | 35,827,235 | 35,684,201 | 33,525,567 | 31,200,773 | 31,089,310 | 38,386,906 | 32,832,514 | 27,475,792 |
Consolidated Statements of Stoc
Consolidated Statements of Stockholders' Equity - USD ($) $ in Thousands | Total | Follow-on Offering | Common Stock | Common StockFollow-on Offering | Additional Paid-In Capital | Additional Paid-In CapitalFollow-on Offering | Accumulated Other Comprehensive Income/ (Loss) | Accumulated Deficit |
Beginning balance at Dec. 31, 2015 | $ 93,873 | $ 3 | $ 158,555 | $ (64,685) | ||||
Beginning balance, shares at Dec. 31, 2015 | 27,053,156 | |||||||
Impact of implementation/ Cumulative effect adjustment upon adoption | ASU 2014-09 | (2,772) | (2,772) | ||||||
Issuance of common stock during offering, net of issuance costs, value | $ 61,110 | $ 61,110 | ||||||
Issuance of common stock during offering, net of issuance costs, shares | 4,370,000 | |||||||
Issuance of common stock upon the exercise of options and purchases under employee stock purchase plan, value | 506 | 506 | ||||||
Issuance of common stock upon the exercise of options and purchases under employee stock purchase plan, shares | 71,826 | |||||||
Repurchase of early exercised stock options, value | (48) | (48) | ||||||
Repurchase of early exercised stock options, shares | (65,984) | |||||||
Vesting of early exercised stock options and restricted stock | 272 | 272 | ||||||
Stock-based compensation | 2,813 | 2,813 | ||||||
Unrealized gains/(losses), net of tax benefits | 8 | $ 8 | ||||||
Net loss | (10,380) | (10,380) | ||||||
Ending balance at Dec. 31, 2016 | 145,382 | $ 3 | 223,208 | 8 | (77,837) | |||
Ending balance, shares at Dec. 31, 2016 | 31,428,998 | |||||||
Impact of implementation/ Cumulative effect adjustment upon adoption | ASU 2016-09 | 8 | (8) | ||||||
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 upon the exercise of options and purchases under employee stock purchase plan, value | 2,365 | 2,365 | ||||||
Issuance of common stock upon the exercise of options and purchases under employee stock purchase plan, 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 | ||||||
Unrealized gains/(losses), net of tax benefits | (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 upon the exercise of options and purchases under employee stock purchase plan, value | 6,208 | 6,208 | ||||||
Issuance of common stock upon the exercise of options and purchases under employee stock purchase plan, 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), net of tax benefits | 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 |
Consolidated Statements of St_2
Consolidated Statements of Stockholders' Equity (Parenthetical) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Common Stock | Follow-On-Offering | |||
Issuance costs | $ 12,233 | $ 9,025 | $ 4,441 |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Cash flows from operating activities: | |||
Net loss | $ (67,698) | $ (57,010) | $ (10,380) |
Adjustments to reconcile net loss to net cash used in operating activities: | |||
Stock-based compensation expense | 19,341 | 6,138 | 2,813 |
Depreciation | 1,567 | 1,294 | 1,111 |
Amortization of premiums on investments | (279) | 85 | |
Loss on disposal of equipment | 4 | ||
Changes in operating assets and liabilities: | |||
Receivable from collaboration partner | 1,013 | 43,987 | (45,000) |
Prepaid expenses and other current assets | (2,884) | (482) | (112) |
Other long-term assets | (296) | (59) | (24) |
Accounts payable | 671 | 367 | (434) |
Accrued liabilities | 8,945 | 2,968 | 3,250 |
Prepayment from collaboration partner | 8,541 | 4,432 | |
Other long-term liabilities | (178) | (120) | (109) |
Deferred revenue | (33,558) | (11,442) | 28,029 |
Net cash used in operating activities | (64,815) | (9,838) | (20,856) |
Cash flow from investing activities: | |||
Purchases of investments | (132,475) | (44,044) | (16,060) |
Sales of investments | 8,000 | 4,000 | |
Maturities of investments | 28,000 | 4,000 | |
Purchases of property and equipment | (3,373) | (1,517) | (1,083) |
Proceeds from sale of equipment | 10 | ||
Net cash used in investing activities | (99,848) | (37,551) | (17,143) |
Cash flow from financing activities: | |||
Proceeds from issuance of common stock during follow-on offerings, net of issuance costs | 181,863 | 133,862 | 61,148 |
Proceeds from exercise of stock options and employee stock purchase plan | 6,208 | 2,365 | 506 |
Payments of prior period offering costs | (38) | (153) | |
Net cash provided by financing activities | 188,071 | 136,189 | 61,501 |
Net increase in cash and cash equivalents | 23,408 | 88,800 | 23,502 |
Cash, cash equivalents and restricted cash at beginning of period | 224,857 | 136,057 | 112,555 |
Cash, cash equivalents and restricted cash at end of period | 248,265 | 224,857 | 136,057 |
Non-cash investing and financing activities: | |||
Vesting of early exercised options and restricted stock | 53 | 184 | 272 |
Unpaid portion of property and equipment purchases included in period-end accounts payable and accrued liabilities | $ 468 | $ 283 | 103 |
Unpaid financing-related costs included in period-end accounts payable and accrued liabilities | $ 38 |
Organization
Organization | 12 Months Ended |
Dec. 31, 2018 | |
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 heritable cardiomyopathies, a group of rare, genetically driven forms of heart failure that result from biomechanical defects in cardiac muscle contraction. The Company has used its precision medicine platform to generate a robust pipeline of therapeutic programs. MyoKardia’s most advanced program, mavacamten, is in four clinical trials, including a pivotal Phase 3 study for the treatment of hypertrophic cardiomyopathy. A second clinical-stage candidate, MYK-491, is in a Phase 2a multiple-ascending dose study in patients with stable systolic heart failure. The Company was incorporated on June 8, 2012 in Delaware and its corporate headquarters and operations are located in South San Francisco, California. Liquidity The Company has incurred significant operating losses since inception and has an accumulated deficit of $202.6 million as of December 31, 2018. 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 will no longer record revenues from Sanofi in 2019 nor receive 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. The Company expects to incur additional losses and negative cash flows for the foreseeable future and it 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. There is no assurance that such financing will be available on terms acceptable to the Company, if at all. On March 8, 2018, the Company filed a Shelf Registration Statement (“2018 Shelf Registration Statement”) covering the potential offering, issuance, and sale of an indeterminate amount of shares of common stock at a price of $49.00 per share. In June 2018, the Company sold an additional 211,147 shares of common stock directly to the underwriters when they partially exercised their over-allotment option at the price of $49.00 per share. As of December 31, 2018, the Company had $394.8 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 this Form 10-K. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 12 Months Ended |
Dec. 31, 2018 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies Basis of Presentation and 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”). During 2015, the Company established a wholly-owned foreign subsidiary, MyoKardia Australia Pty Ltd 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 revenue recognition, clinical trial accruals, income taxes 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, 2018 and 2017, the Company’s cash and cash equivalents were comprised of funds held in checking accounts and interest-bearing money market accounts and money market funds. Restricted Cash Restricted cash at December 31, 2018 and 2017 comprises cash balances primarily held as security in connection with the Company’s facility lease agreements and is included in restricted cash and other on the consolidated balance sheets. 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 and cash equivalents and the restricted cash as presented on the consolidated balance sheets. Cash as reported in the consolidated statements of cash flows consists of (in thousands): As of December 31, 2018 2017 2016 Cash and cash equivalents $ 246,122 $ 224,571 $ 135,797 Restricted cash - noncurrent 2,143 286 260 Cash balance in consolidated statements of cash flows $ 248,265 $ 224,857 $ 136,057 Short-term and Long-term Investments All investments have been classified as “available-for-sale” and are carried at fair value as determined 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. 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 the receivable from collaboration partner, accounts payable and accrued liabilities and other current liabilities approximate fair value due to their short-term maturities. Concentration of Credit Risk Financial instruments that potentially subject the Company to a concentration of credit risk consist of cash and 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. Risk and Uncertainties The Company’s future results of operations involve a number of risks and uncertainties. Factors that could affect the Company’s future operating results and cause actual results to vary materially from expectations include, but are not limited to, uncertainty of results of clinical trials and reaching milestones, uncertainty of regulatory approval of the Company’s potential drug candidates, uncertainty of market acceptance of any of the Company’s product candidates that receive regulatory approval, competition from substitute products and larger companies, securing and protecting proprietary technology, strategic relationships and dependence on key individuals and source suppliers. Products developed by the Company require approvals from the U.S. Food and Drug Administration (“FDA”) or other international regulatory agencies prior to commercial sales. There can be no assurance that any of the Company’s product candidates will receive the necessary approvals. If the Company is denied approval, approval is delayed, or the Company is unable to maintain approvals, it could have a materially adverse impact on the Company. The Company expects to incur substantial operating losses for the next several years and will need to obtain additional financing in order to complete clinical trials and launch and commercialize any product candidates for which it receives regulatory approval. There can be no assurance that such financing will be available or will be on terms acceptable by the Company. 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. Impairment of Long-lived Assets 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, 2018, there have been no such impairment charges. Deferred Offering Costs Deferred offering costs, consisting of legal, accounting and other fees and costs relating to the Initial Public Offering (“IPO”) and follow-on public offerings were capitalized. The deferred offering costs were offset against the proceeds received upon the closing of the IPO and follow-on offerings. There were $0.4 million of deferred offering costs capitalized during 2017 upon the completion of the 2017 follow-on public offering, which were offset against the $134.3 million of proceeds received, net of underwriting discounts and commissions. There were $0.6 million of deferred offering costs capitalized during 2018 upon the completion of the 2018 follow-on public offering, which were offset against the $182.5 million of proceeds received, net of underwriting discounts and commissions. As of December 31, 2018 and 2017, there were no deferred offering costs capitalized on the consolidated balance sheets. Revenue Recognition The Company generates revenue from collaboration and license agreements for the development and commercialization of its products. Collaboration and license agreements may include non-refundable upfront license fees, partial or complete reimbursement of research and development costs, contingent consideration payments based on the achievement of defined collaboration objectives and royalties on sales of commercialized products. To date, the Company has not recognized revenue from sales of its product candidates. Effective January 1, 2018, the Company adopted Accounting Standards Codification Topic 606, Revenue from Contracts with Customers Licenses of intellectual property : Upon the inception of a Collaboration Agreement, if the license to the Company’s intellectual property is determined to be distinct from the other performance obligations identified in the arrangement, the Company recognizes revenue from non-refundable, up-front fees allocated to the license when the license is transferred to the customer and the customer is able to use and benefit from the license. For licenses that are bundled with other promises, the Company utilizes judgement to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or at a point in time and, if over time, the appropriate method of measuring proportional performance for purposes of recognizing revenue from non-refundable, up-front fees. The Company evaluates the measure of proportional performance each reporting period and, if necessary, adjusts the measure of performance and related revenue recognition. Milestone payments : At the inception of each arrangement that includes development, regulatory or commercial milestone payments, the Company evaluates whether the milestones are considered probable of being reached and estimates the amount to be included in the transaction price. ASC 606 suggests two alternatives to use when estimating the amount of variable consideration: the expected value method and the most likely amount method. Under the expected value method, an entity considers the sum of probability-weighted amounts in a range of possible consideration amounts. Under the most likely amount method, an entity considers the single most likely amount in a range of possible consideration amounts. Whichever method is used, it should be consistently applied throughout the life of the contract; however, it is not necessary for the Company to use the same approach for all contracts. The Company expects to use the most likely amount method for development and regulatory milestone payments. If it is probable that a significant revenue reversal would not occur, the associated milestone value is included in the transaction price. Milestone payments that are not within the control of the Company or the licensee, such as regulatory approvals, are not considered probable of being achieved until those approvals are received. The transaction price is then allocated to each performance obligation on a relative stand-alone selling price basis. The Company recognizes revenue when the performance obligations under the contract are satisfied. At the end of each subsequent reporting period, the Company re-evaluates the probability or achievement of each such milestone and any related constraint, and if necessary, adjusts its estimates of the overall transaction price. Any such adjustments are recorded on a cumulative catch-up basis, which would affect revenues and earnings in the period of adjustment. Royalties : For arrangements that include sales-based royalties, including milestone payments based on the level of sales, and the license is deemed to be the predominant item to which the royalties relate, the Company recognizes revenue at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied). To date, the Company has not recognized any royalty revenue resulting from its collaboration arrangement. Up-front payments and fees are recorded as deferred revenue upon receipt or when due and may require deferral of revenue recognition to a future period until the Company performs its obligations under these arrangements. Amounts payable to the Company are recorded as accounts receivable when the Company’s right to consideration is unconditional. The Company does not assess whether a contract has a significant financing component if the expectation at contract inception is such that the period between payment by the customer and the transfer of the promised goods or services to the customer will be one year or less. 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. Amounts incurred in connection with collaboration and license agreements are also included in research and development expense. Under the terms of the Collaboration Agreement, the Company and Sanofi are sharing qualified research and development expenses that are jointly incurred in order to register mavacamten. Such cost sharing ends June 30, 2019, the end of the Collaboration agreement for the mavacamten registration program plan activities (“RPP”). Sanofi is reimbursing the Company 100% of preapproved costs incurred in the development of MYK-224 through March 31, 2019, the end of the Collaboration Agreement program term for this compound. Qualified costs consist of internal and external research and development expenses including employee costs and direct out-of-pocket costs that are specifically identifiable or reasonably and directly related to the development of mavacamten and MYK-224. Examples of qualified costs include those incurred for clinical trials, preparation for regulatory approval, manufacture or purchase of product for use in trials and development of manufacturing processes. The Company reduces its research and development expenses during the period by the amounts expected to be received from Sanofi. Preclinical Study and Clinical Trial Accruals The Company’s preclinical study and clinical trial accruals 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 multiple research institutions and clinical research organizations (“CROs”) that conduct and manage clinical trials on the Company’s behalf. The Company estimates preclinical study and clinical trial expenses 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. The Company estimates these expenses based on discussions with internal clinical management personnel and external service providers as to the progress or stage of completion of trials or services and the contracted fees to be paid for such services. If the actual timing of the performance of services or the level of effort varies from the original estimates, the Company will adjust the accrual accordingly. Payments made to third parties under these arrangements in advance of the performance of the related services by the third parties are recorded as prepaid expenses until the services are rendered. 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 is based on the probability of achieving certain performance criteria, as defined in the individual option grant agreement. The Company estimates the number of performance options ultimately expected to vest and recognizes stock-based compensation expense for those options expected to vest when it becomes probable that the performance criteria will be met and the options vest. The Company has also granted stock options to purchase common stock that vest upon the achievement of market-based stock price targets. The fair value of a stock-based award is recognized over the period during which an optionee 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. As permitted under ASU 2016-09, beginning January 1, 2017, the Company has elected to recognize forfeitures as they occur, and no longer estimates a forfeiture rate when calculating the stock-based compensation for our equity awards. Through December 31, 2016, stock-based compensation expense recognized at fair value included the impact of estimated forfeitures as the Company estimated future forfeitures at the date of grant and revised the estimates, if necessary, in subsequent periods if actual forfeitures differed from those estimates. Equity instruments issued to non-employees are recorded at their fair value on the measurement date and are subject to periodic adjustments as the underlying equity instruments vest. The fair value of options granted to consultants is expensed when vested. 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. 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. 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. Comprehensive Income (Loss) Comprehensive income (loss) is defined as a change in equity of a business enterprise during a period resulting from transactions from non-owner sources. The Company’s comprehensive income and losses are primarily due to unrealized gains and losses from its available-for-sale securities that are excluded from reported net loss, which qualified as other comprehensive income (loss) presented in the consolidated statements of operations and comprehensive loss. Net Loss per Share of Common Stock Basic net loss per common share is calculated by dividing the net loss by the weighted-average number of common shares outstanding during the period, without consideration of potentially dilutive securities. Diluted net loss per share is computed by dividing the net loss by the weighted-average number of common shares and potentially dilutive common share equivalents outstanding during the period. 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 all periods. Adopted Accounting Pronouncements – Revenue Recognition As previously noted, effective January 1, 2018, the Company adopted 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. Impact of Adoption – ASC 606 The Company entered into a license and collaboration agreement that became effective in August 2014, which is within the scope of ASC 606, under which it has licensed certain rights to its HCM-1 (which includes mavacamten and MYK-224), HCM-2 and DCM-1 (which includes MYK-491) programs to Sanofi and may enter into other such arrangements in the future. The terms of the arrangement include payment to the Company of one or more of the following: non-refundable, up-front license fees, development and regulatory and commercial milestone payments, and royalties on net sales of licensed products. The Company has applied the five-step model of the new standard to the Company’s contract with Sanofi, as it is the only contract that will be impacted by the adoption of the new revenue standards. The Company has implemented the new revenue standard using the full retrospective transition method and has revised its comparative financial statements as if ASC 606 had been effective for those periods. The license and collaboration agreement provided for upfront, milestone and continuation payments, which the Company has recognized in revenues in full as of December 31, 2018. The following tables summarize the financial statement line items that were affected due to the Company’s implementation of ASC 606 : Consolidated Balance Sheets December 31, 2017 As Originally Reported Effect of Change As Revised Deferred revenue - current $ 22,500 $ 11,058 $ 33,558 Accumulated deficit $ (123,797 ) $ (11,058 ) $ (134,855 ) Consolidated Statements of Operations and Comprehensive Losses Year Ended December 31, 2017 As Originally Reported Effect of Change As Revised Collaboration and license revenue $ 22,500 $ (11,058 ) $ 11,442 Operating expenses: Research and development 48,136 — 48,136 General and administrative 21,973 — 21,973 Total operating expenses 70,109 — 70,109 Loss from operations (47,609 ) (11,058 ) (58,667 ) Interest and other income, net 1,657 — 1,657 Net loss (45,952 ) (11,058 ) (57,010 ) Other comprehensive loss (200 ) — (200 ) Comprehensive loss (46,152 ) (11,058 ) (57,210 ) Net loss per share, basic and diluted $ (1.40 ) $ (0.34 ) $ (1.74 ) Weighted average number of shares used to compute net loss per share, basic and diluted 32,832,514 — 32,832,514 Year Ended December 31, 2016 As Originally Reported Effect of Change As Revised Collaboration and license revenue $ 39,199 $ 2,772 $ 41,971 Operating expenses: Research and development 36,215 — 36,215 General and administrative 16,289 — 16,289 Total operating expenses 52,504 — 52,504 Loss from operations (13,305 ) 2,772 (10,533 ) Interest and other income, net 153 — 153 Net loss (13,152 ) 2,772 (10,380 ) Other comprehensive gain 8 — 8 Comprehensive loss (13,144 ) 2,772 (10,372 ) Net loss per share, basic and diluted $ (0.48 ) $ 0.10 $ (0.38 ) Weighted average number of shares used to compute net loss per share, basic and diluted 27,475,792 — 27,475,792 Consolidated Statements of Cash Flows Year Ended December 31, 2017 As Originally Reported Effect of Change As Revised Net loss $ (45,952 ) $ (11,058 ) $ (57,010 ) Adjustments to reconcile net loss to net cash used in operating activities: Deferred revenue $ (22,500 ) $ 11,058 $ (11,442 ) Cash, cash equivalents and restricted cash, beginning of period $ 135,797 $ 260 $ 136,057 Cash, cash equivalents and restricted cash, end of period $ 224,571 $ 286 $ 224,857 Year Ended December 31, 2016 As Originally Reported Effect of Change As Revised Net loss $ (13,152 ) $ 2,772 $ (10,380 ) Adjustments to reconcile net loss to net cash used in operating activities: Deferred revenue $ 30,801 $ (2,772 ) $ 28,029 Cash, cash equivalents and restricted cash, beginning of period $ 112,265 $ 290 $ 112,555 Cash, cash equivalents and restricted cash, end of period $ 135,797 $ 260 $ 136,057 The changes to net loss and deferred revenue in the consolidated statement of cash flows, as revised above, reflects: (i) for the year ended December 31, 2017, the Company’s determination that the Sanofi continuation payment of $45.0 million received in January 2017 relates to three performance obligations that were previously accounted for as one combined unit of accounting, (ii) for the year ended December 31, 2016, the Company’s determination that the Sanofi up-front payment of $35.0 million received in August 2014 relates to three performance obligations that were previously accounted for as one combined unit of accounting and, (iii) the result of the Company utilizing a cost-based input method to measure proportional performance for both fiscal years, instead of straight-line. As discussed in “Adopted Accounting Pronouncements – Other” below, the change to cash, cash equivalents and restricted cash in the table above reflects the Company’s implementation of ASU 2016-18 whereby amounts generally described as restricted cash and restricted cash equivalents should be included with cash and cash equivalents when reconciling the beginning of period and end of period total amounts shown on the statement of cash flows. The Company implemented this change using a retrospective transition method. Adopted Accounting Pronouncements – Other In May 2017, the Financial Accounting Standards Board (“ ASU No. 2017-09—Compensation—Stock Compensation (Topic 718): Scope of Modification Accounting. The amendments in this ASU provide guidance about which changes to the terms or conditions of a share-based payment award require an entity to apply modification accounting in Topic 718 to address diversity in practice. An entity should account for the effects of a modification unless all the three specified conditions are met. The current disclosure requirements in Topic 718 apply regardless of whether an entity is required to apply modification accounting under the amendments in this ASU. The amendments in this ASU are effective for all entities for annual periods, and interim periods within those annual periods, beginning after December 15, 2017, with early adoption permitted. The amendments in this ASU should be applied prospectively to an award modified on or after the adoption date. The adoption of this standard did not have a material impact on the Company’s consolidated financial statements In November 2016, the FASB issued ASU No. 2016-18 (Topic 230), Restricted Cash, Statement of Cash Flows ment of cash flows explain the change during the period in the total of cash, cash equivalents, and amounts generally described as restricted cash or restricted cash equivalents. Therefore, amounts generally described as restricted cash and restricted cash equivalents should be included with cash and cash equivalents when reconciling the beginning-of-period and end-of-period total amounts shown on the statement of cash flows. ASU 2016-18 is effective for public business entities for fiscal years beginning after December 15, 2017, and interim periods within those fiscal years, with early adoption permitted. The amendments in this ASU should be applied using a retrospective transition method to each period presented. Other than the change in presentation in the accompanying consolidated statements of cash flows, the adoption of this guidance had no effect on the Company’s financial position, results of operations or liquidity. Recent Accounting Pronouncements From time to time, new accounting pronouncements are issued by the FASB or other standard setting bodies and adopted by the Company as of the specified effective date. Unless otherwise discussed, the impact of recently issued standards that are not yet effective will not have a material impact on the Company’s consolidated financial statements upon adoption. In November 2018, the FASB issued ASU 2018-18 (Topic 808), Clarifying the Interaction Between Topic 808 and Topic 606 In August 2018, the FASB issued ASU 2018-13 (Topic 820), Fair Value Measurement In February 2016, the FASB issued ASU No. 2016-02, Leases In June 2018, the FASB issued ASU No. 2018-07 (Topic 718), Compensation – Stock Compensation In February 2018, the FASB issued ASU No. 2018-05 (Topic 740) Income Taxes |
Collaboration and License Agree
Collaboration and License Agreement | 12 Months Ended |
Dec. 31, 2018 | |
Collaboration And License Agreement Disclosure [Abstract] | |
Collaboration and License Agreement | 3. Collaboration and License Agreement Sanofi (Aventis Inc.) Agreement Overview and Termination In August 2014, the Company entered into an exclusive License and Collaboration Agreement (“Collaboration Agreement”) with Aventis Inc., a wholly-owned subsidiary of Sanofi, 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. During the period August 2014 through December 2018, Sanofi paid the Company a total of $105.0 million in cash to perform research and development on the development of such products, as well as for granting to Sanofi certain royalty bearing licenses. Of the $105.0 million, $0.7 million was attributed to a freestanding convertible preferred stock call option and $104.3 million was recognized as revenue during the period from August 2014 through December 31, 2018, the date on which the Company received a notice of termination of the Collaboration Agreement. 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. The continuation would have committed Sanofi to specific research and development activities in support of the commercialization of the Company’s products as well as resulted in a continuation of its obligation under the cost sharing portion of the collaboration to co-fund development as discussed further below. On December 31, 2018 Sanofi notified the Company it was terminating the Collaboration Agreement. Under the terms of the termination: • Sanofi will reimburse the Company for certain research and development costs through June 30, 2019, after which such time such reimbursements will discontinue; • the Company recovered global rights to all programs in its portfolio, including lead clinical-stage candidates, mavacamten and MYK-491; and • Sanofi will remain eligible to receive royalties associated with any potential HCM-1 products that will range from mid-single to low-double digits in the U.S. (there is no royalty obligation to Sanofi for sales outside the U.S.). The Company has determined that Sanofi was a related party of the Company due to its previous collaborative relationship, certain royalty bearing license obligations on future sales of HCM-1 in the United States, and that it is the Company’s only partner. As of December 31, 2018, Sanofi was also a beneficial shareholder of the Company’s common stock. History of the Collaboration Agreement Under the Collaboration Agreement, the Company granted Sanofi royalty-bearing licenses to develop and commercialize products resulting from its lead candidate programs HCM-1, HCM-2 and DCM-1. The licenses provide Sanofi with worldwide rights in the case of DCM-1 and rights outside the United States with respect to the HCM-1 and HCM-2 programs. The terms of the Collaboration Agreement also state that the Company is responsible for conducting research and development activities through early human efficacy studies for all three programs, except for specified research activities to be conducted by Sanofi. Upon entering into this agreement, the Company received an up-front non-refundable cash payment of $35.0 million and Sanofi made an up-front equity purchase of $10.0 million (additional equity investments from Sanofi totaling $26.5 million were received subsequent to the effective date of the Collaboration Agreement). The Company was also eligible to receive additional payments and services, as follows: • a one-time, non-refundable payment of $25.0 million contingent upon submission of an Investigational New Drug (“IND”) application before certain regulatory authorities for its DCM-1 program; • a non-refundable continuation payment of $45.0 million contingent upon Sanofi’s notification of its decision to continue the agreement beyond December 31, 2016; and • up to $15.0 million in research and development funding for the lead compound in each program if studies leading to proof-of-concept (“POC”) were extended beyond December 31, 2018; • up to $45.0 million in funding from Sanofi of approved in-kind research and clinical activities. During the fourth quarter of 2016, the Company submitted an IND application to the U.S. Food and Drug Administration and as a result, the Company received the $25.0 million milestone payment from Sanofi. In December 2016, Sanofi provided notice to the Company of its election to continue the collaboration through December 31, 2018 pursuant to the terms of the Collaboration Agreement. In connection with Sanofi’s decision to continue the collaboration, in January 2017 the Company received the $45.0 million milestone payment. Under the terms of the agreement, the Company was entitled to receive tiered royalties ranging from the mid-single digits to the mid-teens on net sales of certain HCM-1, HCM-2 and DCM-1 finished products outside the United States and on net sales of certain DCM-1 finished products in the United States. Revenue Recognition In the implementation of ASC 606 “Revenues from Contracts with Customers” using the full retrospective transition method effective January 1, 2016, the Company evaluated the Collaboration Agreement under ASC 606 and determined that it had the following performance obligations: 1. the licenses of intellectual property for each of the HCM-1, HCM-2 and DCM-1 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 through an extended term ending December 31, 2018 and made the $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 do 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 which at contract inception consisted of the upfront cash payment, valued at $34.3 million, net of the fair value of $0.7 million allocated to the option provided to Sanofi to acquire equity, and variable consideration of $25.0 million, subject to an IND application. Sanofi paid the Company the $25.0 million milestone payment upon the Company’s application for the IND. In 2016, after the IND application was made and when the Company determined it was deemed probable that significant reversal in the amount of cumulative revenue recognized will not occur, the Company included this amount in the transaction price. As of December 31, 2016, all performance obligations associated with the initial term were satisfied. 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. As previously noted above, the Collaboration Agreement also 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, 2018, 2017 and 2016, the Company recognized $33.6 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 years ending December 31, 2018 and 2017 (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 ) $ — Year Ended December 31, 2017 Balance at Beginning of Period Additions Deductions Balance at End of Period Contract assets: Receivable from collaboration partner $ 45,000 $ — $ (45,000 ) $ — Contract liabilities: Deferred revenue $ 45,000 $ — $ (11,442 ) $ 33,558 Cost Sharing During the years ended December 31, 2018 and 2017 the Company has 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 the termination date, Sanofi has 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 is established for the lead compound under each of the HCM-1 and HCM-2 programs; and (ii) if the Company has initiated a clinical trial of a compound under a proof-of-concept development plan and not terminated its development thereof and if another additional compound is identified as a development candidate for the same program, the Company is entitled to full reimbursement of pre-proof-of-concept (“pre-POC”) 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, Sanofi is sharing RPP costs for the mavacamten program until June 30, 2019 pursuant to the Collaboration Agreement termination terms. Registration program costs are subject to review and approval by the Company and Sanofi and include amounts incurred relating to clinical trials, development and manufacturing of, and obtaining regulatory approvals for mavacamten, and include direct employee costs and direct out-of-pocket costs incurred, by or on behalf of a party, that are 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 has incurred in the development of the MYK-224 compound, which has been identified as an additional compound under the HCM-1 program. Eligible costs are subject to review and approval under the same procedures as under the RPP program; reimbursable costs consist 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 will continue to be received from Sanofi until March 31, 2019, in accordance to the Collaboration Agreement termination terms. Estimated reimbursements are invoiced to Sanofi before each interim period based on budgeted amounts. For the RPP program, these estimates consist of one half of the Company’s mavacamten development budget in excess of Sanofi’s mavacamten development budget each interim period. For the MYK-224 compound, these estimates consist of all of the Company’s research and development budget related to the compound for the forthcoming quarter. After each period end, a review of the actual expenses incurred is performed and any adjustments are carried forward to future invoices. Actual amounts received from Sanofi are applied to the applicable interim period to reduce the Company’s research and development expenses. The Company recorded zero The following table presents the Sanofi research and development reimbursement receivables and related prepayment activity during the years ended December 31, 2018 and 2017 (in thousands): Year Ended December 31, 2018 2017 Receivable from collaboration partner Balance at beginning of year $ 1,013 $ — Additions 3,994 1,013 Deductions (5,007 ) — Balance at end of year $ - $ 1,013 Prepayment from collaboration partner for mavacamten Balance at beginning of year $ 4,432 $ — Additions for advance billings - 1,013 Payments received from Sanofi 31,659 10,697 Actual expenses incurred (23,118 ) (7,278 ) Balance at end of year $ 12,973 $ 4,432 |
Fair Value Measurements
Fair Value Measurements | 12 Months Ended |
Dec. 31, 2018 | |
Fair Value Disclosures [Abstract] | |
Fair Value Measurements | 4. Fair Value Measurements Financial assets and liabilities are recorded at fair value. 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. 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, 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 — $ 393,906 $ 245,194 $ 148,712 $ — Classified as (with contractual maturities): Cash equivalents (due within 90 days) $ 245,194 Short-term investments (due within one year) 68,564 Long-term investments (due between one and two years) 80,148 $ 393,906 Fair Value Measurements at December 31, 2017 Total Level 1 Level 2 Level 3 Assets Money market funds $ 223,568 $ 223,568 $ — $ — U.S. government agency obligations 27,878 — 27,878 — Corporate securities 23,955 — 23,955 — $ 275,401 $ 223,568 $ 51,833 $ — Classified as (with contractual maturities): Cash equivalents (due within 90 days) $ 223,568 Short-term investments (due within one year) 31,933 Long-term investments (due between one and two years) 19,900 $ 275,401 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 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 $ 393,968 $ 98 $ (160 ) $ 393,906 Fair Amortized Cost Unrealized Gain Unrealized Loss Fair Value Cash equivalents (due within 90 days) $ 223,568 $ — $ — $ 223,568 Short-term investments (due within one year) 32,010 — (77 ) 31,933 Long-term investments (due between one and two years) 20,010 — (110 ) 19,900 $ 275,588 $ — $ (187 ) $ 275,401 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, 2018 and 2017. 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, 2018 | |
Balance Sheet Related Disclosures [Abstract] | |
Balance Sheet Components | 5. Balance Sheet Components Property and Equipment Property and equipment consists of the following (in thousands): As of December 31, 2018 2017 Scientific equipment $ 9,126 $ 5,935 Furniture and equipment 1,248 1,064 Capitalized software 302 278 Leasehold improvements 451 331 Total 11,127 7,608 Less: Accumulated depreciation (5,989 ) (4,461 ) Property and equipment, net $ 5,138 $ 3,147 Depreciation expense was $1.6 million, $1.3 million and $1.1 million for the years ended December 31, 2018, 2017 and 2016, respectively. Accrued Liabilities Accrued liabilities consist of the following (in thousands): As of December 31, 2018 2017 Clinical research and development $ 10,903 $ 5,981 Payroll-related liabilities 8,151 4,412 Other 1,704 1,246 Total accrued liabilities $ 20,758 $ 11,639 |
Commitments and Contingencies
Commitments and Contingencies | 12 Months Ended |
Dec. 31, 2018 | |
Commitments And Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 6. 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. Facilities As of December 31, 2018, the Company has operating leases for facilities consist of approximately 48,400 square feet of office and lab space in two facilities in South San Francisco, California, which will expire in January 2020. In October 2018, the Company entered into a 15-month noncancelable operating lease for approximately 22,100 square feet of additional space in South San Francisco, California, which commenced in February 2019. Future minimum rental payments are $1.1 million in the aggregate excluding taxes and operating expenses. In September 2018, the Company entered into a noncancelable operating lease for approximately 129,800 square feet of space in Brisbane, California which is currently under construction. The date on which the Company will become responsible for paying rent will be the date the premises are ready for occupancy, currently anticipated to be January 2020, and the lease will expire 10 years after that date. Included in the lease is one option to extend for an additional 10-year period. Future minimum rental payments are $93.2 million in the aggregate plus taxes and operating expenses payable to the landlord. In September 2018, the Company provided a standby letter of credit of $1.9 million as security for its obligations under this Lease. Standby letters of credit are classified as long-term assets within restricted cash and other on the consolidated balance sheet. Future annual minimum lease payments due under the new and existing operating leases at December 31 of each year are as follows (in thousands): Year ending December 31: Amount 1 2019 2,752 2020 5,831 2021 8,461 2022 8,757 2023 9,063 Thereafter 61,444 Total $ 96,308 (1) Rent expense, net was $2.1 million, $1.4 million and $1.3 million for the years ended December 31, 2018, 2017 and 2016, respectively. 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. 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, 2018 and 2017. 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, 2018 | |
Stockholders Equity Note [Abstract] | |
Stockholders’ Equity | 7. Stockholders’ Equity On March 8, 2018, the Company filed the 2018 Shelf Registration Statement covering the potential offering, issuance, and sale of an indeterminate amount of shares of common stock at a price of $49.00 per share. In June 2018, the Company sold an additional 211,147 shares of common stock directly to the underwriters when they partially exercised their over-allotment option at the price of $49.00 per share. 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, 2018 December 31, 2017 Options and awards issued and outstanding 3,864,407 2,964,549 Shares available for issuance under 2015 Stock Option and Incentive Plan 904,785 863,538 Shares available for issuance under 2015 Employee Stock Purchase Plan 780,716 449,444 Total 5,549,908 4,277,531 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, 2018 and 2017, no preferred stock was issued or outstanding. |
Stock-Based Compensation
Stock-Based Compensation | 12 Months Ended |
Dec. 31, 2018 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Stock-Based Compensation | 8. 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, 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 (the “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 period, and the fair value is the market price on the date of grant . 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, 2019 and 2018, the Company reserved an additional 402,889 358,127 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, 2016 2,141,868 $ 6.42 8.4 $ 14,963 Options granted 1,735,875 15.41 Options exercised (333,822 ) 4.81 Options canceled (579,372 ) 8.11 Balance at December 31, 2017 2,964,549 11.54 8.3 90,780 Options granted 1,370,381 52.53 Options exercised (489,153 ) 10.61 Options canceled (144,316 ) 22.67 Balance at December 31, 2018 3,701,461 26.40 8.0 88,524 Options outstanding and exercisable as of December 31, 2018 1,519,637 14.35 7.3 52,979 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 $21.1 million, $9.8 million and $337,000 for the years ended December 31, 2018, 2017 and 2016, respectively. The total estimated grant date fair value of options vested during the years ended December 31, 2018, 2017 and 2016 was $13.4 million, $4.8 million and $2.1 million, respectively. 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, 2017 — $ — — $ — RSUs awarded 171,766 53.27 RSUs released (3,250 ) 48.93 RSUs forfeited (5,570 ) 52.83 Balance at December 31, 2018 162,946 53.37 2.0 7,962 The aggregate intrinsic value of RSUs was calculated using the estimated fair value of common stock times the number of RSUs. The total grant date fair value of RSUs vested during the years ended December 31, 2018, 2017 and 2016 was $159,000, nil and nil, respectively. Stock-Based Compensation Stock-based compensation expense, net of forfeitures, as applicable for the years ended December 31, 2018, 2017 and 2016, is reflected in the statements of operations and comprehensive loss as follows (in thousands): Year Ended December 31, 2018 2017 2016 Research and development $ 8,144 $ 2,752 $ 1,252 General and administrative 11,197 3,386 1,561 Total stock-based compensation $ 19,341 $ 6,138 $ 2,813 As of December 31, 2018, total unamortized stock-based compensation relating to options was $45.9 million, which is expected to be recognized over the average remaining vesting period of 3.1 As of December 31, 2018, total unamortized stock-based compensation relating to RSUs was $7.0 million, which is expected to be recognized over the average remaining vesting period of 3.2 In relation to stock options and awards that vest upon the achievement of performance criteria, $248,000, $174,000 and $180,000 in stock-based compensation expense was recorded for the years ended December 31, 2018, 2017 and 2016, respectively. The weighted‑average grant date fair value of options granted under the Company’s stock plans in the years ended December 31, 2018, 2017 and 2016 was $34.70, $ 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, 2018 2017 2016 Risk-free interest rate 2.54%-2.98% 1.92%-2.27% 1.05%-2.10% Expected life (in years) 5.3-6.1 5.3-6.1 5.3-6.1 Volatility 69%-75% 71%-75% 71%-73% Dividend yield 0% 0% 0% In relation to stock options that vest upon the achievement of market-based stock price target, the Company estimated the fair value on the original grant date using a Monte-Carlo simulation model. Since its IPO, the Company has recognized the stock-based compensation expense on a straight-line basis over the implicit service period as derived under that simulation model. Liability for Early Exercise of Stock Options As of December 31, 2018 and 2017, there were 9,790 and 81,373, respectively, of unvested common shares outstanding that were issued upon the early exercise of stock options prior to the vesting of the underlying shares and subject to repurchase by the Company at the original issuance price upon termination of the stockholders’ services. The right to repurchase these shares generally lapses with respect to 25% of the shares underlying the option after one year of service to the Company and 1/48 of the shares underlying the original grant per month for 36 months thereafter. The shares purchased by the employees pursuant to the early exercise of stock options are not deemed, for accounting purposes, to be issued until those shares vest. The cash received in exchange for exercised and unvested shares related to stock options granted is recorded as a liability for the early exercise of stock options on the consolidated balance sheets and will be reclassified to common stock and additional paid-in capital as the shares vest. As of December 31, 2018, and 2017, the Company recorded $15,000 and $68,000, respectively, within accrued liabilities and other long-term liabilities associated with shares issued subject to repurchase rights. The Company repurchased 997 and 41,961 shares of unvested early exercised stock in the years ended December 31, 2018 and 2017, for $1,000 and $45,000, respectively. |
Net Loss per Share
Net Loss per Share | 12 Months Ended |
Dec. 31, 2018 | |
Earnings Per Share [Abstract] | |
Net Loss per Share | 9. 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, 2018 2017 2016 As revised (Note 2) Numerator Net loss $ (67,698 ) $ (57,010 ) $ (10,380 ) Denominator Weighted average shares outstanding 38,466,233 33,098,571 28,104,991 Less: weighted average shares subject to repurchase (79,327 ) (266,057 ) (629,199 ) Weighted average shares used to compute basic and diluted net loss per share 38,386,906 32,832,514 27,475,792 Net loss per share, basic and diluted $ (1.76 ) $ (1.74 ) $ (0.38 ) 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, 2018 2017 2016 Common stock subject to repurchase 9,790 81,373 409,839 Options and awards issued and outstanding 3,864,407 2,964,549 2,141,868 As of December 31, 2018, the Company has contributions from plan participants of $272,000 under the 2015 ESPP, which if converted, would be equivalent to 5,703 shares based on 85% of the stock price at the beginning of the offering period. As of December 31, 2017, the Company had contributions from plan participants of $133,000 under the 2015 ESPP, which if converted, would be equivalent to 4,030 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, 2018 | |
Employee Benefit Plans [Abstract] | |
Employee Benefit Plan | 10. 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, 2018 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | 11. Income Taxes For each of the years ended December 31, 2018, 2017 and 2016, the effective income tax rate and tax provision from continuing operations was zero percent for all periods, primarily attributable to losses generated which are not more likely than not to be realized. The Company adopted ASC 606 on January 1, 2018 and has revised the following information for the years ending December 31, 2017 and 2016 accordingly. The following is the reconciliation between the statutory federal income tax rate and the Company’s effective tax rate: Year Ended December 31, 2018 2017 2016 As revised (Note 2) Federal statutory income tax rate 21.0 % 34.0 % 34.0 % State taxes (tax effected) 8.6 7.1 8.4 Non-deductible expenses and other 2.5 2.0 (12.9 ) Research and development credits 5.9 6.0 17.8 Tax Act – net deferred tax rate change — (25.1 ) — Change in valuation allowance (38.0 ) (24.0 ) (47.3 ) Total — % — % — % As of December 31, 2018 and 2017, the components of the Company’s deferred tax assets are as follows (in thousands): As of December 31, 2018 2017 As revised (Note 2) Deferred tax assets: Net operating loss carryforwards $ 49,689 $ 22,246 Research and development credits carryforwards 12,908 8,631 Stock-based compensation 4,349 890 Deferred revenue — 9,391 Start-up costs 1,237 1,354 Depreciation (959 ) (457 ) Other 1,858 1,327 Total deferred tax assets 69,082 43,382 Less: valuation allowance (69,082 ) (43,382 ) Net deferred tax assets $ — $ — In December 2017, the Tax Act was signed into law making significant changes to the Internal Revenue Code. The Tax Act reduced the corporate income tax rate from 34% to 21% effective January 1, 2018. The Company re-measured its U.S. deferred tax assets and liabilities, which resulted in a reduction of net deferred tax assets with a corresponding adjustment to the valuation allowance. As a result, no tax expense was recorded related to the enactment of the Tax Act. The Company’s primary deferred tax asset of $49.7 million at December 31, 2018 and $22.2 million at December 31, 2017 relates 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 valuation allowance to offset the net deferred tax assets at December 31, 2018 and December 31, 2017, respectively. As of December 31, 2018, the Company had approximately $177.0 million and $179.4 million of federal and state net operating losses, respectively, that will begin to expire in 2032. As of December 31, 2018, the Company had approximately $4.5 million and $3.8 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, 2018, the Company had approximately $9.0 million of federal orphan tax credit carryovers, which will begin to expire in 2036 if not utilized. The valuation allowance increased by approximately $25.7 million, $ 13.7 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 percentage point change (by value) in its equity ownership over a rolling three-year period, the corporation’s ability to use its pre-change net operating loss carryforwards and other pre-change tax attributes (such as research tax credits) to offset its post-change income or taxes may be limited. Similar rules may apply under the laws of the state of California. The annual limitation generally is determined by multiplying the value of the Company’s stock at the time of such ownership change (subject to certain adjustments) by the applicable “long-term tax-exempt rate.” Such limitations may result in expiration of a portion of the NOLs and other tax attributes before utilization. Each ownership change resulted in an annual limitation, but all NOLs and other tax attributes generated prior to the ownership changes on April 20, 2015 and August 14, 2017 can be utilized prior to expiration if the Company earns sufficient taxable income. The Company’s follow-on stock offering completed in June 2018 did not result in an ownership change. As of December 31, 2018, and 2017, 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, 2018, and 2017, 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 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 2018, remain open to U.S. federal and California tax examinations. The statute of limitation in Australia is four years. There were no interest or penalties accrued at December 31, 2018 and 2017. At December 31, 2018, 2017 and 2016, the Company's reserve for unrecognized tax benefits is approximately $3.8 million, $2.5 million and $1.5 million, respectively. Due to the full valuation allowance at December 31, 2018, 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, 2018 2017 2016 As revised (Note 2) Beginning balance $ 2,471 $ 1,474 $ 719 Increases (decreases) of unrecognized tax benefits related to prior year 289 (97 ) — Increases of unrecognized tax benefits related to current year 1,050 1,094 755 Ending balance $ 3,810 $ 2,471 $ 1,474 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, 2018 | |
Quarterly Financial Data [Abstract] | |
Quarterly Financial Data (unaudited) | 12. 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 per share amounts) First Second Third Fourth Quarter Quarter Quarter Quarter 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 2017, As Revised (Note 2) Revenues $ 2,410 $ 3,001 $ 3,077 $ 2,954 Total operating expenses 17,393 18,771 20,245 13,700 Net loss (14,762 ) (15,461 ) (16,721 ) (10,066 ) Net loss per common share, basic and diluted $ (0.47 ) $ (0.50 ) $ (0.50 ) $ (0.28 ) Weighted average number of shares, basic and diluted 31,089,310 31,200,773 33,525,567 35,684,201 |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 12 Months Ended |
Dec. 31, 2018 | |
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”). During 2015, the Company established a wholly-owned foreign subsidiary, MyoKardia Australia Pty Ltd |
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 revenue recognition, clinical trial accruals, income taxes 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, 2018 and 2017, the Company’s cash and cash equivalents were comprised of funds held in checking accounts and interest-bearing money market accounts and money market funds. |
Restricted Cash | Restricted Cash Restricted cash at December 31, 2018 and 2017 comprises cash balances primarily held as security in connection with the Company’s facility lease agreements and is included in restricted cash and other on the consolidated balance sheets. |
Cash as Reported in Consolidated Statements of Cash Flows | 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 and cash equivalents and the restricted cash as presented on the consolidated balance sheets. Cash as reported in the consolidated statements of cash flows consists of (in thousands): As of December 31, 2018 2017 2016 Cash and cash equivalents $ 246,122 $ 224,571 $ 135,797 Restricted cash - noncurrent 2,143 286 260 Cash balance in consolidated statements of cash flows $ 248,265 $ 224,857 $ 136,057 |
Short-term and Long-term Investments | Short-term and Long-term Investments All investments have been classified as “available-for-sale” and are carried at fair value as determined 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. |
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 the receivable from collaboration partner, accounts payable and accrued liabilities and other current liabilities approximate fair value due to their short-term maturities. Financial assets and liabilities are recorded at fair value. 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. |
Concentration of Credit Risk | Concentration of Credit Risk Financial instruments that potentially subject the Company to a concentration of credit risk consist of cash and cash equivalents and 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. |
Risk and Uncertainties | Risk and Uncertainties The Company’s future results of operations involve a number of risks and uncertainties. Factors that could affect the Company’s future operating results and cause actual results to vary materially from expectations include, but are not limited to, uncertainty of results of clinical trials and reaching milestones, uncertainty of regulatory approval of the Company’s potential drug candidates, uncertainty of market acceptance of any of the Company’s product candidates that receive regulatory approval, competition from substitute products and larger companies, securing and protecting proprietary technology, strategic relationships and dependence on key individuals and source suppliers. Products developed by the Company require approvals from the U.S. Food and Drug Administration (“FDA”) or other international regulatory agencies prior to commercial sales. There can be no assurance that any of the Company’s product candidates will receive the necessary approvals. If the Company is denied approval, approval is delayed, or the Company is unable to maintain approvals, it could have a materially adverse impact on the Company. The Company expects to incur substantial operating losses for the next several years and will need to obtain additional financing in order to complete clinical trials and launch and commercialize any product candidates for which it receives regulatory approval. There can be no assurance that such financing will be available or will be on terms acceptable by the Company. |
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. |
Impairment of Long-Lived Assets | Impairment of Long-lived Assets 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, 2018, there have been no such impairment charges. |
Deferred Offering Costs | Deferred Offering Costs Deferred offering costs, consisting of legal, accounting and other fees and costs relating to the Initial Public Offering (“IPO”) and follow-on public offerings were capitalized. The deferred offering costs were offset against the proceeds received upon the closing of the IPO and follow-on offerings. There were $0.4 million of deferred offering costs capitalized during 2017 upon the completion of the 2017 follow-on public offering, which were offset against the $134.3 million of proceeds received, net of underwriting discounts and commissions. There were $0.6 million of deferred offering costs capitalized during 2018 upon the completion of the 2018 follow-on public offering, which were offset against the $182.5 million of proceeds received, net of underwriting discounts and commissions. As of December 31, 2018 and 2017, there were no deferred offering costs capitalized on the consolidated balance sheets. |
Revenue Recognition | Revenue Recognition The Company generates revenue from collaboration and license agreements for the development and commercialization of its products. Collaboration and license agreements may include non-refundable upfront license fees, partial or complete reimbursement of research and development costs, contingent consideration payments based on the achievement of defined collaboration objectives and royalties on sales of commercialized products. To date, the Company has not recognized revenue from sales of its product candidates. Effective January 1, 2018, the Company adopted Accounting Standards Codification Topic 606, Revenue from Contracts with Customers Licenses of intellectual property : Upon the inception of a Collaboration Agreement, if the license to the Company’s intellectual property is determined to be distinct from the other performance obligations identified in the arrangement, the Company recognizes revenue from non-refundable, up-front fees allocated to the license when the license is transferred to the customer and the customer is able to use and benefit from the license. For licenses that are bundled with other promises, the Company utilizes judgement to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or at a point in time and, if over time, the appropriate method of measuring proportional performance for purposes of recognizing revenue from non-refundable, up-front fees. The Company evaluates the measure of proportional performance each reporting period and, if necessary, adjusts the measure of performance and related revenue recognition. Milestone payments : At the inception of each arrangement that includes development, regulatory or commercial milestone payments, the Company evaluates whether the milestones are considered probable of being reached and estimates the amount to be included in the transaction price. ASC 606 suggests two alternatives to use when estimating the amount of variable consideration: the expected value method and the most likely amount method. Under the expected value method, an entity considers the sum of probability-weighted amounts in a range of possible consideration amounts. Under the most likely amount method, an entity considers the single most likely amount in a range of possible consideration amounts. Whichever method is used, it should be consistently applied throughout the life of the contract; however, it is not necessary for the Company to use the same approach for all contracts. The Company expects to use the most likely amount method for development and regulatory milestone payments. If it is probable that a significant revenue reversal would not occur, the associated milestone value is included in the transaction price. Milestone payments that are not within the control of the Company or the licensee, such as regulatory approvals, are not considered probable of being achieved until those approvals are received. The transaction price is then allocated to each performance obligation on a relative stand-alone selling price basis. The Company recognizes revenue when the performance obligations under the contract are satisfied. At the end of each subsequent reporting period, the Company re-evaluates the probability or achievement of each such milestone and any related constraint, and if necessary, adjusts its estimates of the overall transaction price. Any such adjustments are recorded on a cumulative catch-up basis, which would affect revenues and earnings in the period of adjustment. Royalties : For arrangements that include sales-based royalties, including milestone payments based on the level of sales, and the license is deemed to be the predominant item to which the royalties relate, the Company recognizes revenue at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied). To date, the Company has not recognized any royalty revenue resulting from its collaboration arrangement. Up-front payments and fees are recorded as deferred revenue upon receipt or when due and may require deferral of revenue recognition to a future period until the Company performs its obligations under these arrangements. Amounts payable to the Company are recorded as accounts receivable when the Company’s right to consideration is unconditional. The Company does not assess whether a contract has a significant financing component if the expectation at contract inception is such that the period between payment by the customer and the transfer of the promised goods or services to the customer will be one year or less. Revenue Recognition In the implementation of ASC 606 “Revenues from Contracts with Customers” using the full retrospective transition method effective January 1, 2016, the Company evaluated the Collaboration Agreement under ASC 606 and determined that it had the following performance obligations: 1. the licenses of intellectual property for each of the HCM-1, HCM-2 and DCM-1 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. Amounts incurred in connection with collaboration and license agreements are also included in research and development expense. Under the terms of the Collaboration Agreement, the Company and Sanofi are sharing qualified research and development expenses that are jointly incurred in order to register mavacamten. Such cost sharing ends June 30, 2019, the end of the Collaboration agreement for the mavacamten registration program plan activities (“RPP”). Sanofi is reimbursing the Company 100% of preapproved costs incurred in the development of MYK-224 through March 31, 2019, the end of the Collaboration Agreement program term for this compound. Qualified costs consist of internal and external research and development expenses including employee costs and direct out-of-pocket costs that are specifically identifiable or reasonably and directly related to the development of mavacamten and MYK-224. Examples of qualified costs include those incurred for clinical trials, preparation for regulatory approval, manufacture or purchase of product for use in trials and development of manufacturing processes. The Company reduces its research and development expenses during the period by the amounts expected to be received from Sanofi. |
Preclinical Study and Clinical Trial Accruals | Preclinical Study and Clinical Trial Accruals The Company’s preclinical study and clinical trial accruals 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 multiple research institutions and clinical research organizations (“CROs”) that conduct and manage clinical trials on the Company’s behalf. The Company estimates preclinical study and clinical trial expenses 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. The Company estimates these expenses based on discussions with internal clinical management personnel and external service providers as to the progress or stage of completion of trials or services and the contracted fees to be paid for such services. If the actual timing of the performance of services or the level of effort varies from the original estimates, the Company will adjust the accrual accordingly. Payments made to third parties under these arrangements in advance of the performance of the related services by the third parties are recorded as prepaid expenses until the services are rendered. |
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 is based on the probability of achieving certain performance criteria, as defined in the individual option grant agreement. The Company estimates the number of performance options ultimately expected to vest and recognizes stock-based compensation expense for those options expected to vest when it becomes probable that the performance criteria will be met and the options vest. The Company has also granted stock options to purchase common stock that vest upon the achievement of market-based stock price targets. The fair value of a stock-based award is recognized over the period during which an optionee 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. As permitted under ASU 2016-09, beginning January 1, 2017, the Company has elected to recognize forfeitures as they occur, and no longer estimates a forfeiture rate when calculating the stock-based compensation for our equity awards. Through December 31, 2016, stock-based compensation expense recognized at fair value included the impact of estimated forfeitures as the Company estimated future forfeitures at the date of grant and revised the estimates, if necessary, in subsequent periods if actual forfeitures differed from those estimates. Equity instruments issued to non-employees are recorded at their fair value on the measurement date and are subject to periodic adjustments as the underlying equity instruments vest. The fair value of options granted to consultants is expensed when vested. 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. |
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. 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. |
Comprehensive Income (Loss) | Comprehensive Income (Loss) Comprehensive income (loss) is defined as a change in equity of a business enterprise during a period resulting from transactions from non-owner sources. The Company’s comprehensive income and losses are primarily due to unrealized gains and losses from its available-for-sale securities that are excluded from reported net loss, which qualified as other comprehensive income (loss) presented in the consolidated statements of operations and comprehensive loss. |
Net Loss per Share of Common Stock | Net Loss per Share of Common Stock Basic net loss per common share is calculated by dividing the net loss by the weighted-average number of common shares outstanding during the period, without consideration of potentially dilutive securities. Diluted net loss per share is computed by dividing the net loss by the weighted-average number of common shares and potentially dilutive common share equivalents outstanding during the period. 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 all periods. |
Adopted Accounting Pronouncements | Adopted Accounting Pronouncements – Revenue Recognition As previously noted, effective January 1, 2018, the Company adopted 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. Impact of Adoption – ASC 606 The Company entered into a license and collaboration agreement that became effective in August 2014, which is within the scope of ASC 606, under which it has licensed certain rights to its HCM-1 (which includes mavacamten and MYK-224), HCM-2 and DCM-1 (which includes MYK-491) programs to Sanofi and may enter into other such arrangements in the future. The terms of the arrangement include payment to the Company of one or more of the following: non-refundable, up-front license fees, development and regulatory and commercial milestone payments, and royalties on net sales of licensed products. The Company has applied the five-step model of the new standard to the Company’s contract with Sanofi, as it is the only contract that will be impacted by the adoption of the new revenue standards. The Company has implemented the new revenue standard using the full retrospective transition method and has revised its comparative financial statements as if ASC 606 had been effective for those periods. The license and collaboration agreement provided for upfront, milestone and continuation payments, which the Company has recognized in revenues in full as of December 31, 2018. The following tables summarize the financial statement line items that were affected due to the Company’s implementation of ASC 606 : Consolidated Balance Sheets December 31, 2017 As Originally Reported Effect of Change As Revised Deferred revenue - current $ 22,500 $ 11,058 $ 33,558 Accumulated deficit $ (123,797 ) $ (11,058 ) $ (134,855 ) Consolidated Statements of Operations and Comprehensive Losses Year Ended December 31, 2017 As Originally Reported Effect of Change As Revised Collaboration and license revenue $ 22,500 $ (11,058 ) $ 11,442 Operating expenses: Research and development 48,136 — 48,136 General and administrative 21,973 — 21,973 Total operating expenses 70,109 — 70,109 Loss from operations (47,609 ) (11,058 ) (58,667 ) Interest and other income, net 1,657 — 1,657 Net loss (45,952 ) (11,058 ) (57,010 ) Other comprehensive loss (200 ) — (200 ) Comprehensive loss (46,152 ) (11,058 ) (57,210 ) Net loss per share, basic and diluted $ (1.40 ) $ (0.34 ) $ (1.74 ) Weighted average number of shares used to compute net loss per share, basic and diluted 32,832,514 — 32,832,514 Year Ended December 31, 2016 As Originally Reported Effect of Change As Revised Collaboration and license revenue $ 39,199 $ 2,772 $ 41,971 Operating expenses: Research and development 36,215 — 36,215 General and administrative 16,289 — 16,289 Total operating expenses 52,504 — 52,504 Loss from operations (13,305 ) 2,772 (10,533 ) Interest and other income, net 153 — 153 Net loss (13,152 ) 2,772 (10,380 ) Other comprehensive gain 8 — 8 Comprehensive loss (13,144 ) 2,772 (10,372 ) Net loss per share, basic and diluted $ (0.48 ) $ 0.10 $ (0.38 ) Weighted average number of shares used to compute net loss per share, basic and diluted 27,475,792 — 27,475,792 Consolidated Statements of Cash Flows Year Ended December 31, 2017 As Originally Reported Effect of Change As Revised Net loss $ (45,952 ) $ (11,058 ) $ (57,010 ) Adjustments to reconcile net loss to net cash used in operating activities: Deferred revenue $ (22,500 ) $ 11,058 $ (11,442 ) Cash, cash equivalents and restricted cash, beginning of period $ 135,797 $ 260 $ 136,057 Cash, cash equivalents and restricted cash, end of period $ 224,571 $ 286 $ 224,857 Year Ended December 31, 2016 As Originally Reported Effect of Change As Revised Net loss $ (13,152 ) $ 2,772 $ (10,380 ) Adjustments to reconcile net loss to net cash used in operating activities: Deferred revenue $ 30,801 $ (2,772 ) $ 28,029 Cash, cash equivalents and restricted cash, beginning of period $ 112,265 $ 290 $ 112,555 Cash, cash equivalents and restricted cash, end of period $ 135,797 $ 260 $ 136,057 The changes to net loss and deferred revenue in the consolidated statement of cash flows, as revised above, reflects: (i) for the year ended December 31, 2017, the Company’s determination that the Sanofi continuation payment of $45.0 million received in January 2017 relates to three performance obligations that were previously accounted for as one combined unit of accounting, (ii) for the year ended December 31, 2016, the Company’s determination that the Sanofi up-front payment of $35.0 million received in August 2014 relates to three performance obligations that were previously accounted for as one combined unit of accounting and, (iii) the result of the Company utilizing a cost-based input method to measure proportional performance for both fiscal years, instead of straight-line. As discussed in “Adopted Accounting Pronouncements – Other” below, the change to cash, cash equivalents and restricted cash in the table above reflects the Company’s implementation of ASU 2016-18 whereby amounts generally described as restricted cash and restricted cash equivalents should be included with cash and cash equivalents when reconciling the beginning of period and end of period total amounts shown on the statement of cash flows. The Company implemented this change using a retrospective transition method. Adopted Accounting Pronouncements – Other In May 2017, the Financial Accounting Standards Board (“ ASU No. 2017-09—Compensation—Stock Compensation (Topic 718): Scope of Modification Accounting. The amendments in this ASU provide guidance about which changes to the terms or conditions of a share-based payment award require an entity to apply modification accounting in Topic 718 to address diversity in practice. An entity should account for the effects of a modification unless all the three specified conditions are met. The current disclosure requirements in Topic 718 apply regardless of whether an entity is required to apply modification accounting under the amendments in this ASU. The amendments in this ASU are effective for all entities for annual periods, and interim periods within those annual periods, beginning after December 15, 2017, with early adoption permitted. The amendments in this ASU should be applied prospectively to an award modified on or after the adoption date. The adoption of this standard did not have a material impact on the Company’s consolidated financial statements In November 2016, the FASB issued ASU No. 2016-18 (Topic 230), Restricted Cash, Statement of Cash Flows ment of cash flows explain the change during the period in the total of cash, cash equivalents, and amounts generally described as restricted cash or restricted cash equivalents. Therefore, amounts generally described as restricted cash and restricted cash equivalents should be included with cash and cash equivalents when reconciling the beginning-of-period and end-of-period total amounts shown on the statement of cash flows. ASU 2016-18 is effective for public business entities for fiscal years beginning after December 15, 2017, and interim periods within those fiscal years, with early adoption permitted. The amendments in this ASU should be applied using a retrospective transition method to each period presented. Other than the change in presentation in the accompanying consolidated statements of cash flows, the adoption of this guidance had no effect on the Company’s financial position, results of operations or liquidity. |
Recent Accounting Pronouncements | Recent Accounting Pronouncements From time to time, new accounting pronouncements are issued by the FASB or other standard setting bodies and adopted by the Company as of the specified effective date. Unless otherwise discussed, the impact of recently issued standards that are not yet effective will not have a material impact on the Company’s consolidated financial statements upon adoption. In November 2018, the FASB issued ASU 2018-18 (Topic 808), Clarifying the Interaction Between Topic 808 and Topic 606 In August 2018, the FASB issued ASU 2018-13 (Topic 820), Fair Value Measurement In February 2016, the FASB issued ASU No. 2016-02, Leases In June 2018, the FASB issued ASU No. 2018-07 (Topic 718), Compensation – Stock Compensation In February 2018, the FASB issued ASU No. 2018-05 (Topic 740) Income Taxes |
Contract Term | 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 through an extended term ending December 31, 2018 and made the $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 do 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 | 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 which at contract inception consisted of the upfront cash payment, valued at $34.3 million, net of the fair value of $0.7 million allocated to the option provided to Sanofi to acquire equity, and variable consideration of $25.0 million, subject to an IND application. Sanofi paid the Company the $25.0 million milestone payment upon the Company’s application for the IND. In 2016, after the IND application was made and when the Company determined it was deemed probable that significant reversal in the amount of cumulative revenue recognized will not occur, the Company included this amount in the transaction price. As of December 31, 2016, all performance obligations associated with the initial term were satisfied. 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. As previously noted above, the Collaboration Agreement also 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, 2018 and 2017 the Company has 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 the termination date, Sanofi has 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 is established for the lead compound under each of the HCM-1 and HCM-2 programs; and (ii) if the Company has initiated a clinical trial of a compound under a proof-of-concept development plan and not terminated its development thereof and if another additional compound is identified as a development candidate for the same program, the Company is entitled to full reimbursement of pre-proof-of-concept (“pre-POC”) 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, Sanofi is sharing RPP costs for the mavacamten program until June 30, 2019 pursuant to the Collaboration Agreement termination terms. Registration program costs are subject to review and approval by the Company and Sanofi and include amounts incurred relating to clinical trials, development and manufacturing of, and obtaining regulatory approvals for mavacamten, and include direct employee costs and direct out-of-pocket costs incurred, by or on behalf of a party, that are 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 has incurred in the development of the MYK-224 compound, which has been identified as an additional compound under the HCM-1 program. Eligible costs are subject to review and approval under the same procedures as under the RPP program; reimbursable costs consist 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 will continue to be received from Sanofi until March 31, 2019, in accordance to the Collaboration Agreement termination terms. Estimated reimbursements are invoiced to Sanofi before each interim period based on budgeted amounts. For the RPP program, these estimates consist of one half of the Company’s mavacamten development budget in excess of Sanofi’s mavacamten development budget each interim period. For the MYK-224 compound, these estimates consist of all of the Company’s research and development budget related to the compound for the forthcoming quarter. After each period end, a review of the actual expenses incurred is performed and any adjustments are carried forward to future invoices. Actual amounts received from Sanofi are applied to the applicable interim period to reduce the Company’s research and development expenses. |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Revenue Initial Application Period Cumulative Effect Transition [Line Items] | |
Summary of Cash Reported in Consolidated Statements of Cash Flows | Cash as reported in the consolidated statements of cash flows consists of (in thousands): As of December 31, 2018 2017 2016 Cash and cash equivalents $ 246,122 $ 224,571 $ 135,797 Restricted cash - noncurrent 2,143 286 260 Cash balance in consolidated statements of cash flows $ 248,265 $ 224,857 $ 136,057 |
Accounting Standards Update 2014-09 | |
Revenue Initial Application Period Cumulative Effect Transition [Line Items] | |
Summary of Financial Statement Line Items Affected due to Implementation of Topic 606 | The following tables summarize the financial statement line items that were affected due to the Company’s implementation of ASC 606 : Consolidated Balance Sheets December 31, 2017 As Originally Reported Effect of Change As Revised Deferred revenue - current $ 22,500 $ 11,058 $ 33,558 Accumulated deficit $ (123,797 ) $ (11,058 ) $ (134,855 ) Consolidated Statements of Operations and Comprehensive Losses Year Ended December 31, 2017 As Originally Reported Effect of Change As Revised Collaboration and license revenue $ 22,500 $ (11,058 ) $ 11,442 Operating expenses: Research and development 48,136 — 48,136 General and administrative 21,973 — 21,973 Total operating expenses 70,109 — 70,109 Loss from operations (47,609 ) (11,058 ) (58,667 ) Interest and other income, net 1,657 — 1,657 Net loss (45,952 ) (11,058 ) (57,010 ) Other comprehensive loss (200 ) — (200 ) Comprehensive loss (46,152 ) (11,058 ) (57,210 ) Net loss per share, basic and diluted $ (1.40 ) $ (0.34 ) $ (1.74 ) Weighted average number of shares used to compute net loss per share, basic and diluted 32,832,514 — 32,832,514 Year Ended December 31, 2016 As Originally Reported Effect of Change As Revised Collaboration and license revenue $ 39,199 $ 2,772 $ 41,971 Operating expenses: Research and development 36,215 — 36,215 General and administrative 16,289 — 16,289 Total operating expenses 52,504 — 52,504 Loss from operations (13,305 ) 2,772 (10,533 ) Interest and other income, net 153 — 153 Net loss (13,152 ) 2,772 (10,380 ) Other comprehensive gain 8 — 8 Comprehensive loss (13,144 ) 2,772 (10,372 ) Net loss per share, basic and diluted $ (0.48 ) $ 0.10 $ (0.38 ) Weighted average number of shares used to compute net loss per share, basic and diluted 27,475,792 — 27,475,792 Consolidated Statements of Cash Flows Year Ended December 31, 2017 As Originally Reported Effect of Change As Revised Net loss $ (45,952 ) $ (11,058 ) $ (57,010 ) Adjustments to reconcile net loss to net cash used in operating activities: Deferred revenue $ (22,500 ) $ 11,058 $ (11,442 ) Cash, cash equivalents and restricted cash, beginning of period $ 135,797 $ 260 $ 136,057 Cash, cash equivalents and restricted cash, end of period $ 224,571 $ 286 $ 224,857 Year Ended December 31, 2016 As Originally Reported Effect of Change As Revised Net loss $ (13,152 ) $ 2,772 $ (10,380 ) Adjustments to reconcile net loss to net cash used in operating activities: Deferred revenue $ 30,801 $ (2,772 ) $ 28,029 Cash, cash equivalents and restricted cash, beginning of period $ 112,265 $ 290 $ 112,555 Cash, cash equivalents and restricted cash, end of period $ 135,797 $ 260 $ 136,057 |
Collaboration and License Agr_2
Collaboration and License Agreement (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
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 years ending December 31, 2018 and 2017 (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 ) $ — Year Ended December 31, 2017 Balance at Beginning of Period Additions Deductions Balance at End of Period Contract assets: Receivable from collaboration partner $ 45,000 $ — $ (45,000 ) $ — Contract liabilities: Deferred revenue $ 45,000 $ — $ (11,442 ) $ 33,558 |
Schedule of Research and Development Reimbursement Receivables and Related Prepayments from Collaboration Partner | The following table presents the Sanofi research and development reimbursement receivables and related prepayment activity during the years ended December 31, 2018 and 2017 (in thousands): Year Ended December 31, 2018 2017 Receivable from collaboration partner Balance at beginning of year $ 1,013 $ — Additions 3,994 1,013 Deductions (5,007 ) — Balance at end of year $ - $ 1,013 Prepayment from collaboration partner for mavacamten Balance at beginning of year $ 4,432 $ — Additions for advance billings - 1,013 Payments received from Sanofi 31,659 10,697 Actual expenses incurred (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, 2018 | |
Fair Value Disclosures [Abstract] | |
Summary of Fair Value Assets and Liabilities Measured on Recurring Basis | 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, 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 — $ 393,906 $ 245,194 $ 148,712 $ — Classified as (with contractual maturities): Cash equivalents (due within 90 days) $ 245,194 Short-term investments (due within one year) 68,564 Long-term investments (due between one and two years) 80,148 $ 393,906 Fair Value Measurements at December 31, 2017 Total Level 1 Level 2 Level 3 Assets Money market funds $ 223,568 $ 223,568 $ — $ — U.S. government agency obligations 27,878 — 27,878 — Corporate securities 23,955 — 23,955 — $ 275,401 $ 223,568 $ 51,833 $ — Classified as (with contractual maturities): Cash equivalents (due within 90 days) $ 223,568 Short-term investments (due within one year) 31,933 Long-term investments (due between one and two years) 19,900 $ 275,401 |
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 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 $ 393,968 $ 98 $ (160 ) $ 393,906 Fair Amortized Cost Unrealized Gain Unrealized Loss Fair Value Cash equivalents (due within 90 days) $ 223,568 $ — $ — $ 223,568 Short-term investments (due within one year) 32,010 — (77 ) 31,933 Long-term investments (due between one and two years) 20,010 — (110 ) 19,900 $ 275,588 $ — $ (187 ) $ 275,401 |
Balance Sheet Components (Table
Balance Sheet Components (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Balance Sheet Related Disclosures [Abstract] | |
Summary of Property and Equipment | Property and equipment consists of the following (in thousands): As of December 31, 2018 2017 Scientific equipment $ 9,126 $ 5,935 Furniture and equipment 1,248 1,064 Capitalized software 302 278 Leasehold improvements 451 331 Total 11,127 7,608 Less: Accumulated depreciation (5,989 ) (4,461 ) Property and equipment, net $ 5,138 $ 3,147 |
Summary of Accrued Liabilities | Accrued liabilities consist of the following (in thousands): As of December 31, 2018 2017 Clinical research and development $ 10,903 $ 5,981 Payroll-related liabilities 8,151 4,412 Other 1,704 1,246 Total accrued liabilities $ 20,758 $ 11,639 |
Commitments and Contingencies (
Commitments and Contingencies (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Commitments And Contingencies Disclosure [Abstract] | |
Schedule of Future Annual Minimum Lease Payments Due Under New and Existing Operating Leases | Future annual minimum lease payments due under the new and existing operating leases at December 31 of each year are as follows (in thousands): Year ending December 31: Amount 1 2019 2,752 2020 5,831 2021 8,461 2022 8,757 2023 9,063 Thereafter 61,444 Total $ 96,308 (1) |
Stockholders_ Equity (Tables)
Stockholders’ Equity (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
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, 2018 December 31, 2017 Options and awards issued and outstanding 3,864,407 2,964,549 Shares available for issuance under 2015 Stock Option and Incentive Plan 904,785 863,538 Shares available for issuance under 2015 Employee Stock Purchase Plan 780,716 449,444 Total 5,549,908 4,277,531 |
Stock-Based Compensation (Table
Stock-Based Compensation (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
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, 2016 2,141,868 $ 6.42 8.4 $ 14,963 Options granted 1,735,875 15.41 Options exercised (333,822 ) 4.81 Options canceled (579,372 ) 8.11 Balance at December 31, 2017 2,964,549 11.54 8.3 90,780 Options granted 1,370,381 52.53 Options exercised (489,153 ) 10.61 Options canceled (144,316 ) 22.67 Balance at December 31, 2018 3,701,461 26.40 8.0 88,524 Options outstanding and exercisable as of December 31, 2018 1,519,637 14.35 7.3 52,979 |
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, 2017 — $ — — $ — RSUs awarded 171,766 53.27 RSUs released (3,250 ) 48.93 RSUs forfeited (5,570 ) 52.83 Balance at December 31, 2018 162,946 53.37 2.0 7,962 |
Schedule of Stock-Based Compensation Expense, Net of Forfeitures | Stock-based compensation expense, net of forfeitures, as applicable for the years ended December 31, 2018, 2017 and 2016, is reflected in the statements of operations and comprehensive loss as follows (in thousands): Year Ended December 31, 2018 2017 2016 Research and development $ 8,144 $ 2,752 $ 1,252 General and administrative 11,197 3,386 1,561 Total stock-based compensation $ 19,341 $ 6,138 $ 2,813 |
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, 2018 2017 2016 Risk-free interest rate 2.54%-2.98% 1.92%-2.27% 1.05%-2.10% Expected life (in years) 5.3-6.1 5.3-6.1 5.3-6.1 Volatility 69%-75% 71%-75% 71%-73% Dividend yield 0% 0% 0% |
Net Loss per Share (Tables)
Net Loss per Share (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
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, 2018 2017 2016 As revised (Note 2) Numerator Net loss $ (67,698 ) $ (57,010 ) $ (10,380 ) Denominator Weighted average shares outstanding 38,466,233 33,098,571 28,104,991 Less: weighted average shares subject to repurchase (79,327 ) (266,057 ) (629,199 ) Weighted average shares used to compute basic and diluted net loss per share 38,386,906 32,832,514 27,475,792 Net loss per share, basic and diluted $ (1.76 ) $ (1.74 ) $ (0.38 ) |
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, 2018 2017 2016 Common stock subject to repurchase 9,790 81,373 409,839 Options and awards issued and outstanding 3,864,407 2,964,549 2,141,868 |
Income Taxes (Tables)
Income Taxes (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
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, 2018 2017 2016 As revised (Note 2) Federal statutory income tax rate 21.0 % 34.0 % 34.0 % State taxes (tax effected) 8.6 7.1 8.4 Non-deductible expenses and other 2.5 2.0 (12.9 ) Research and development credits 5.9 6.0 17.8 Tax Act – net deferred tax rate change — (25.1 ) — Change in valuation allowance (38.0 ) (24.0 ) (47.3 ) Total — % — % — % |
Components of Company's Deferred Tax Assets | As of December 31, 2018 and 2017, the components of the Company’s deferred tax assets are as follows (in thousands): As of December 31, 2018 2017 As revised (Note 2) Deferred tax assets: Net operating loss carryforwards $ 49,689 $ 22,246 Research and development credits carryforwards 12,908 8,631 Stock-based compensation 4,349 890 Deferred revenue — 9,391 Start-up costs 1,237 1,354 Depreciation (959 ) (457 ) Other 1,858 1,327 Total deferred tax assets 69,082 43,382 Less: valuation allowance (69,082 ) (43,382 ) 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, 2018 2017 2016 As revised (Note 2) Beginning balance $ 2,471 $ 1,474 $ 719 Increases (decreases) of unrecognized tax benefits related to prior year 289 (97 ) — Increases of unrecognized tax benefits related to current year 1,050 1,094 755 Ending balance $ 3,810 $ 2,471 $ 1,474 |
Quarterly Financial Data (una_2
Quarterly Financial Data (unaudited) (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
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 per share amounts) First Second Third Fourth Quarter Quarter Quarter Quarter 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 2017, As Revised (Note 2) Revenues $ 2,410 $ 3,001 $ 3,077 $ 2,954 Total operating expenses 17,393 18,771 20,245 13,700 Net loss (14,762 ) (15,461 ) (16,721 ) (10,066 ) Net loss per common share, basic and diluted $ (0.47 ) $ (0.50 ) $ (0.50 ) $ (0.28 ) Weighted average number of shares, basic and diluted 31,089,310 31,200,773 33,525,567 35,684,201 |
Organization - Additional Infor
Organization - Additional Information (Details) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | 3 Months Ended | 12 Months Ended | |||
Jun. 30, 2018 | May 31, 2018 | Jun. 30, 2018 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Organization Consolidation And Presentation Of Financial Statements [Line Items] | ||||||
Date of incorporation | Jun. 8, 2012 | |||||
Accumulated deficit | $ 202,553 | $ 134,855 | ||||
Proceeds from offering, net of underwriting discounts and commissions and offering expenses | 181,863 | $ 133,862 | $ 61,148 | |||
Cash, cash equivalents and short and long-term investments | $ 394,800 | |||||
Follow-On-Offering | ||||||
Organization Consolidation And Presentation Of Financial Statements [Line Items] | ||||||
Common stock shares issued | 3,750,000 | |||||
Common stock shares issued, price per share | $ 49 | |||||
Proceeds from offering, net of underwriting discounts and commissions and offering expenses | $ 181,900 | |||||
Over Allotment Option | ||||||
Organization Consolidation And Presentation Of Financial Statements [Line Items] | ||||||
Common stock shares issued | 211,147 | |||||
Common stock shares issued, price per share | $ 49 | $ 49 | ||||
Proceeds from offering, net of underwriting discounts and commissions and offering expenses | $ 9,700 |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies - Additional Information (Details) | 1 Months Ended | 12 Months Ended | ||
Jan. 31, 2017USD ($) | Dec. 31, 2018USD ($)Segment | Dec. 31, 2017USD ($)PerformanceObligation | Dec. 31, 2016USD ($)PerformanceObligation | |
Summary Of Significant Accounting Policies [Line Items] | ||||
Number of reportable and operating segment | Segment | 1 | |||
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 | |||
Impairment of long-lived assets | $ 0 | |||
Deferred offering costs | 0 | $ 0 | ||
Proceeds from offering, net of underwriting discounts and commissions and offering expenses | $ 181,863,000 | 133,862,000 | $ 61,148,000 | |
Sanofi (Aventis Inc.) | Collaborative Agreement | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Research and development expense reimbursing of preapproved costs | 100.00% | |||
Continuation payment, amount received | $ 45,000,000 | $ 45,000,000 | $ 35,000,000 | |
Number of performance obligations | PerformanceObligation | 3 | 3 | ||
Follow-on Public Offering | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Deferred offering costs | $ 600,000 | $ 400,000 | ||
Proceeds from offering, net of underwriting discounts and commissions and offering expenses | $ 182,500,000 | $ 134,300,000 | ||
Minimum | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Property plant and equipment useful life | 2 years | |||
Maximum | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Property plant and equipment useful life | 5 years | |||
Maximum | ASU 2016-02 | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Short-term leases term | 12 months | |||
Leasehold improvements | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Property plant and equipment estimated useful lives | shorter of their estimated useful lives or the related lease term |
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, 2018 | Dec. 31, 2017 | Dec. 31, 2016 |
Accounting Policies [Abstract] | |||
Cash and cash equivalents | $ 246,122 | $ 224,571 | $ 135,797 |
Restricted cash - noncurrent | 2,143 | 286 | 260 |
Cash balance in consolidated statements of cash flows | $ 248,265 | $ 224,857 | $ 136,057 |
Summary of Significant Accoun_6
Summary of Significant Accounting Policies - Consolidated Balance Sheets (Details) - USD ($) $ in Thousands | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 |
Revenue Initial Application Period Cumulative Effect Transition [Line Items] | |||
Deferred revenue - current | $ 33,558 | $ 45,000 | |
Accumulated deficit | $ (202,553) | (134,855) | |
As Originally Reported | Accounting Standards Update 2014-09 | |||
Revenue Initial Application Period Cumulative Effect Transition [Line Items] | |||
Deferred revenue - current | 22,500 | ||
Accumulated deficit | (123,797) | ||
Effect of Change | Accounting Standards Update 2014-09 | |||
Revenue Initial Application Period Cumulative Effect Transition [Line Items] | |||
Deferred revenue - current | 11,058 | ||
Accumulated deficit | $ (11,058) |
Summary of Significant Accoun_7
Summary of Significant Accounting Policies - Consolidated Statements of Operations and Comprehensive Losses (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2017 | Sep. 30, 2017 | Jun. 30, 2017 | Mar. 31, 2017 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Revenue Initial Application Period Cumulative Effect Transition [Line Items] | |||||||||||
Collaboration and license revenue | $ 12,400 | $ 9,188 | $ 6,639 | $ 5,331 | $ 2,954 | $ 3,077 | $ 3,001 | $ 2,410 | $ 11,442 | $ 41,971 | |
Operating expenses: | |||||||||||
Research and development | $ 68,774 | 48,136 | 36,215 | ||||||||
General and administrative | 38,435 | 21,973 | 16,289 | ||||||||
Total operating expenses | $ 30,281 | $ 26,867 | $ 26,130 | $ 23,931 | $ 13,700 | $ 20,245 | $ 18,771 | $ 17,393 | 107,209 | 70,109 | 52,504 |
Loss from operations | (73,651) | (58,667) | (10,533) | ||||||||
Interest and other income, net | 5,953 | 1,657 | 153 | ||||||||
Net loss | (67,698) | (57,010) | (10,380) | ||||||||
Other comprehensive, gain (loss) | 125 | (200) | 8 | ||||||||
Comprehensive loss | $ (67,573) | $ (57,210) | $ (10,372) | ||||||||
Net loss per share, basic and diluted | $ (0.39) | $ (0.39) | $ (0.49) | $ (0.50) | $ (0.28) | $ (0.50) | $ (0.50) | $ (0.47) | $ (1.76) | $ (1.74) | $ (0.38) |
Weighted average number of shares used to compute net loss per share, basic and diluted | 40,259,575 | 40,116,644 | 37,440,024 | 35,827,235 | 35,684,201 | 33,525,567 | 31,200,773 | 31,089,310 | 38,386,906 | 32,832,514 | 27,475,792 |
As Originally Reported | Accounting Standards Update 2014-09 | |||||||||||
Revenue Initial Application Period Cumulative Effect Transition [Line Items] | |||||||||||
Collaboration and license revenue | $ 22,500 | $ 39,199 | |||||||||
Operating expenses: | |||||||||||
Research and development | 48,136 | 36,215 | |||||||||
General and administrative | 21,973 | 16,289 | |||||||||
Total operating expenses | 70,109 | 52,504 | |||||||||
Loss from operations | (47,609) | (13,305) | |||||||||
Interest and other income, net | 1,657 | 153 | |||||||||
Net loss | (45,952) | (13,152) | |||||||||
Other comprehensive, gain (loss) | (200) | 8 | |||||||||
Comprehensive loss | $ (46,152) | $ (13,144) | |||||||||
Net loss per share, basic and diluted | $ (1.40) | $ (0.48) | |||||||||
Weighted average number of shares used to compute net loss per share, basic and diluted | 32,832,514 | 27,475,792 | |||||||||
Effect of Change | Accounting Standards Update 2014-09 | |||||||||||
Revenue Initial Application Period Cumulative Effect Transition [Line Items] | |||||||||||
Collaboration and license revenue | $ (11,058) | $ 2,772 | |||||||||
Operating expenses: | |||||||||||
Loss from operations | (11,058) | 2,772 | |||||||||
Net loss | (11,058) | 2,772 | |||||||||
Comprehensive loss | $ (11,058) | $ 2,772 | |||||||||
Net loss per share, basic and diluted | $ (0.34) | $ 0.10 |
Summary of Significant Accoun_8
Summary of Significant Accounting Policies - Consolidated Statement of Cash Flows (Details) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2017 | Sep. 30, 2017 | Jun. 30, 2017 | Mar. 31, 2017 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Revenue Initial Application Period Cumulative Effect Transition [Line Items] | |||||||||||
Net loss | $ (15,676) | $ (15,789) | $ (18,413) | $ (17,820) | $ (10,066) | $ (16,721) | $ (15,461) | $ (14,762) | $ (67,698) | $ (57,010) | $ (10,380) |
Adjustments to reconcile net loss to net cash used in operating activities: | |||||||||||
Deferred revenue | (33,558) | (11,442) | 28,029 | ||||||||
Cash, cash equivalents and restricted cash at beginning of period | 224,857 | 136,057 | 224,857 | 136,057 | 112,555 | ||||||
Cash, cash equivalents and restricted cash at end of period | $ 248,265 | 224,857 | 248,265 | 224,857 | 136,057 | ||||||
As Originally Reported | Accounting Standards Update 2014-09 | |||||||||||
Revenue Initial Application Period Cumulative Effect Transition [Line Items] | |||||||||||
Net loss | (45,952) | (13,152) | |||||||||
Adjustments to reconcile net loss to net cash used in operating activities: | |||||||||||
Deferred revenue | (22,500) | 30,801 | |||||||||
Cash, cash equivalents and restricted cash at beginning of period | 224,571 | 135,797 | 224,571 | 135,797 | 112,265 | ||||||
Cash, cash equivalents and restricted cash at end of period | 224,571 | 224,571 | 135,797 | ||||||||
Effect of Change | Accounting Standards Update 2014-09 | |||||||||||
Revenue Initial Application Period Cumulative Effect Transition [Line Items] | |||||||||||
Net loss | (11,058) | 2,772 | |||||||||
Adjustments to reconcile net loss to net cash used in operating activities: | |||||||||||
Deferred revenue | 11,058 | (2,772) | |||||||||
Cash, cash equivalents and restricted cash at beginning of period | $ 286 | $ 260 | $ 286 | 260 | 290 | ||||||
Cash, cash equivalents and restricted cash at end of period | $ 286 | $ 286 | $ 260 |
Collaboration and License Agr_3
Collaboration and License Agreement - Additional Information (Details) - USD ($) | Aug. 31, 2014 | Jan. 31, 2017 | Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2017 | Sep. 30, 2017 | Jun. 30, 2017 | Mar. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2018 | Mar. 31, 2018 | Dec. 31, 2018 |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||
Collaboration and license revenue | $ 33,558,000 | $ 11,442,000 | $ 41,971,000 | ||||||||||||||
Collaboration and license revenue | $ 12,400,000 | $ 9,188,000 | $ 6,639,000 | $ 5,331,000 | $ 2,954,000 | $ 3,077,000 | $ 3,001,000 | $ 2,410,000 | 11,442,000 | 41,971,000 | |||||||
Deferred revenue | 33,558,000 | $ 45,000,000 | 33,558,000 | 45,000,000 | |||||||||||||
Short-term receivable from collaboration partner | 1,013,000 | 45,000,000 | 1,013,000 | 45,000,000 | |||||||||||||
Prepayment from collaboration partner | (8,541,000) | (4,432,000) | |||||||||||||||
Collaborative Agreement | Sanofi (Aventis Inc.) | |||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||
Payment received for research and development | $ 105,000,000 | ||||||||||||||||
Collaboration and license revenue | $ 104,300,000 | ||||||||||||||||
Type of Revenue [Extensible List] | myok:ResearchAndDevelopmentMember | ||||||||||||||||
Research and development payment received attributed to redeemable convertible preferred stock call option | $ 700,000 | ||||||||||||||||
Upfront cash received under collaboration agreement | $ 35,000,000 | ||||||||||||||||
Up-front equity investment | 10,000,000 | ||||||||||||||||
Additional equity investments received | $ 26,500,000 | ||||||||||||||||
Eligible to receive one-time, non-refundable contingent payment | 25,000,000 | ||||||||||||||||
Project non-refundable continuation payment | 45,000,000 | ||||||||||||||||
Milestone payment received | 25,000,000 | $ 25,000,000 | |||||||||||||||
Continuation payment, amount received | $ 45,000,000 | 45,000,000 | 35,000,000 | ||||||||||||||
Upfront cash payment | 34,300,000 | ||||||||||||||||
Net fair value allocated to equity | 700,000 | ||||||||||||||||
Variable consideration | 25,000,000 | ||||||||||||||||
Fixed fee | $ 45,000,000 | ||||||||||||||||
Transaction price for extended term | 45,000,000 | ||||||||||||||||
Collaboration and license revenue | 33,600,000 | 11,400,000 | 42,000,000 | ||||||||||||||
Deferred revenue | 0 | 0 | 0 | 0 | |||||||||||||
Short-term receivable from collaboration partner | $ 0 | $ 1,013,000 | 0 | 1,013,000 | $ 0 | $ 0 | |||||||||||
Prepayment from collaboration partner | 13,000,000 | 4,400,000 | |||||||||||||||
Reduction in research and development expenses due to RPP reimbursements | $ 23,100,000 | $ 7,300,000 | $ 0 | ||||||||||||||
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 | ||||||||||||||||
Additional research and development funding for collaboration | $ 15,000,000 |
Collaboration and License Agr_4
Collaboration and License Agreement - Changes in Contract Assets and Liabilities (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Contract liabilities: | |||
Deferred revenue, Balance at Beginning of Period | $ 33,558 | $ 45,000 | |
Deferred revenue, Deductions | (33,558) | (11,442) | $ 28,029 |
Deferred revenue, Balance at End of Period | 33,558 | 45,000 | |
Contract assets: | |||
Receivable from collaboration partner, Balance at Beginning of Period | $ 1,013 | 45,000 | |
Receivable from collaboration partner, Deductions | (45,000) | ||
Receivable from collaboration partner, Balance at End of Period | $ 1,013 | $ 45,000 |
Collaboration and License Agr_5
Collaboration and License Agreement - Schedule of Receivables and Related Prepayments From Collaboration Partner (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Receivable from collaboration partner, Balance at Beginning of Period | $ 1,013 | $ 45,000 |
Receivable from collaboration partner, Balance at End of Period | 1,013 | |
Prepayment from collaboration partner for mavacamten, Balance at Beginning of Period | 4,432 | |
Prepayment from collaboration partner for mavacamten, Balance at End of Period | 12,973 | 4,432 |
Collaborative Agreement | Sanofi (Aventis Inc.) | ||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Receivable from collaboration partner, Balance at Beginning of Period | 1,013 | |
Additions | 3,994 | 1,013 |
Deductions | (5,007) | |
Receivable from collaboration partner, Balance at End of Period | 0 | 1,013 |
Prepayment from collaboration partner for mavacamten, Balance at Beginning of Period | 4,432 | |
Additions for advance billings | 1,013 | |
Payments received from Sanofi | 31,659 | 10,697 |
Actual expenses incurred | (23,118) | (7,278) |
Prepayment from collaboration partner for mavacamten, Balance at End of Period | $ 12,973 | $ 4,432 |
Fair Value Measurements - Summa
Fair Value Measurements - Summary of Fair Value Assets and Liabilities Measured on Recurring Basis (Details) - USD ($) $ in Thousands | Dec. 31, 2018 | Dec. 31, 2017 |
Assets | ||
Assets fair value | $ 393,906 | $ 275,401 |
Money market funds | ||
Assets | ||
Assets fair value | 245,194 | 223,568 |
U.S. government agency obligations | ||
Assets | ||
Assets fair value | 85,033 | 27,878 |
Corporate securities | ||
Assets | ||
Assets fair value | 63,679 | 23,955 |
Cash equivalents (due within 90 days) | ||
Assets | ||
Assets fair value | 245,194 | 223,568 |
Short-term investments (due within one year) | ||
Assets | ||
Assets fair value | 68,564 | 31,933 |
Long-term investments (due between one and two years) | ||
Assets | ||
Assets fair value | 80,148 | 19,900 |
Fair Value Measurements on Recurring Basis | Level 1 | ||
Assets | ||
Assets fair value | 245,194 | 223,568 |
Fair Value Measurements on Recurring Basis | Level 1 | Money market funds | ||
Assets | ||
Assets fair value | 245,194 | 223,568 |
Fair Value Measurements on Recurring Basis | Level 2 | ||
Assets | ||
Assets fair value | 148,712 | 51,833 |
Fair Value Measurements on Recurring Basis | Level 2 | U.S. government agency obligations | ||
Assets | ||
Assets fair value | 85,033 | 27,878 |
Fair Value Measurements on Recurring Basis | Level 2 | Corporate securities | ||
Assets | ||
Assets fair value | $ 63,679 | $ 23,955 |
Fair Value Measurements - Sum_2
Fair Value Measurements - Summary of Fair Value Measurement of Available-for-sale Securities (Details) - USD ($) $ in Thousands | Dec. 31, 2018 | Dec. 31, 2017 |
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | $ 393,968 | $ 275,588 |
Unrealized Gain | 98 | |
Unrealized Loss | (160) | (187) |
Fair Value | 393,906 | 275,401 |
Cash equivalents (due within 90 days) | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 245,194 | 223,568 |
Fair Value | 245,194 | 223,568 |
Short-term investments (due within one year) | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 68,656 | 32,010 |
Unrealized Loss | (92) | (77) |
Fair Value | 68,564 | 31,933 |
Long-term investments (due between one and two years) | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 80,118 | 20,010 |
Unrealized Gain | 98 | |
Unrealized Loss | (68) | (110) |
Fair Value | $ 80,148 | $ 19,900 |
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 - Summ
Balance Sheet Components - Summary of Property and Equipment (Details) - USD ($) $ in Thousands | Dec. 31, 2018 | Dec. 31, 2017 |
Property Plant And Equipment [Line Items] | ||
Property and equipment | $ 11,127 | $ 7,608 |
Less: Accumulated depreciation | (5,989) | (4,461) |
Property and equipment, net | 5,138 | 3,147 |
Scientific Equipment | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment | 9,126 | 5,935 |
Furniture and Equipment | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment | 1,248 | 1,064 |
Capitalized Software | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment | 302 | 278 |
Leasehold Improvements | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment | $ 451 | $ 331 |
Balance Sheet Components - Addi
Balance Sheet Components - Additional Information (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Balance Sheet Related Disclosures [Abstract] | |||
Depreciation expense | $ 1,567 | $ 1,294 | $ 1,111 |
Balance Sheet Components - Su_2
Balance Sheet Components - Summary of Accrued Liabilities (Details) - USD ($) $ in Thousands | Dec. 31, 2018 | Dec. 31, 2017 |
Balance Sheet Related Disclosures [Abstract] | ||
Clinical research and development | $ 10,903 | $ 5,981 |
Payroll-related liabilities | 8,151 | 4,412 |
Other | 1,704 | 1,246 |
Total accrued liabilities | $ 20,758 | $ 11,639 |
Commitments and Contingencies -
Commitments and Contingencies - Additional Information (Details) | 1 Months Ended | 12 Months Ended | |||
Oct. 31, 2018USD ($)ft² | Sep. 30, 2018USD ($)ft² | Dec. 31, 2018USD ($)ft²Facility | Dec. 31, 2017USD ($) | Dec. 31, 2016USD ($) | |
Commitments And Contingencies Disclosure [Line Items] | |||||
Purchase commitment cancellation notice period | 30 days | ||||
Aggregate future minimum rental payments due | $ 96,308,000 | ||||
Rent expense net | 2,100,000 | $ 1,400,000 | $ 1,300,000 | ||
Contingent liability for accrual | $ 0 | $ 0 | |||
Facilities | |||||
Commitments And Contingencies Disclosure [Line Items] | |||||
Area of leased property | ft² | 22,100 | 129,800 | 48,400 | ||
Facilities available for operating lease | Facility | 2 | ||||
Lease expiration date | Jan. 31, 2020 | ||||
Lease period | 15 months | 10 years | |||
Lease commencement period | 2019-02 | ||||
Aggregate future minimum rental payments due | $ 1,100,000 | $ 93,200,000 | |||
Rent commencement date | 2020-01 | ||||
Additional period of extension in lease contract | 10 years | ||||
Standby letter of credit | $ 1,900,000 |
Commitments and Contingencies_2
Commitments and Contingencies - Future Minimum Commitments under Non-Cancelable Operating Leases (Details) $ in Thousands | Dec. 31, 2018USD ($) |
Commitments And Contingencies Disclosure [Abstract] | |
2,019 | $ 2,752 |
2,020 | 5,831 |
2,021 | 8,461 |
2,022 | 8,757 |
2,023 | 9,063 |
Thereafter | 61,444 |
Total | $ 96,308 |
Stockholders' Equity- Additiona
Stockholders' Equity- Additional Information (Details) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | 3 Months Ended | 12 Months Ended | |||
Jun. 30, 2018 | May 31, 2018 | Jun. 30, 2018 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Class Of Stock [Line Items] | ||||||
Proceeds from offering, net of underwriting discounts and commissions and offering expenses | $ 181,863 | $ 133,862 | $ 61,148 | |||
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 | 3,750,000 | |||||
Common stock shares issued, price per share | $ 49 | |||||
Proceeds from offering, net of underwriting discounts and commissions and offering expenses | $ 181,900 | |||||
Over Allotment Option | ||||||
Class Of Stock [Line Items] | ||||||
Common stock shares issued | 211,147 | |||||
Common stock shares issued, price per share | $ 49 | $ 49 | ||||
Proceeds from offering, net of underwriting discounts and commissions and offering expenses | $ 9,700 |
Stockholders' Equity - Schedule
Stockholders' Equity - Schedule of Common Stock Reserved for Issuance (Details) - shares | Dec. 31, 2018 | Jan. 01, 2018 | Dec. 31, 2017 |
Class Of Stock [Line Items] | |||
Shares reserved for future issuance, shares | 5,549,908 | 4,277,531 | |
Options and Awards Issued and Outstanding | |||
Class Of Stock [Line Items] | |||
Shares reserved for future issuance, shares | 3,864,407 | 2,964,549 | |
2015 Stock Option and Incentive Plan | |||
Class Of Stock [Line Items] | |||
Shares reserved for future issuance, shares | 904,785 | 1,432,511 | 863,538 |
2015 Employee Stock Purchase Plan | |||
Class Of Stock [Line Items] | |||
Shares reserved for future issuance, shares | 780,716 | 449,444 |
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, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | Jan. 01, 2019 | Jan. 01, 2018 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||||
Shares reserved for issuance, shares | 5,549,908 | 4,277,531 | |||||
Aggregate intrinsic value of options exercised | $ 21,100,000 | $ 9,800,000 | $ 337,000 | ||||
Estimated grant date fair value of options vested | 13,400,000 | 4,800,000 | 2,100,000 | ||||
Unamortized stock-based compensation | $ 45,900,000 | $ 16,900,000 | $ 7,500,000 | ||||
Stock option average expected recognition period | 3 years 1 month 6 days | 1 year 8 months 12 days | 2 years 8 months 12 days | ||||
Unrecognized share based compensation expense | $ 180,000 | $ 174,000 | $ 180,000 | ||||
Weighted average grant date fair value of options granted | $ 34.70 | $ 9.92 | $ 7.59 | ||||
Unvested common shares outstanding | 9,790 | 81,373 | |||||
Right of repurchase of shares, lapse rate | 25.00% | ||||||
Percentage of shares of original grant per month for 36 months thereafter | 2.083% | ||||||
Accrued liabilities and other long-term liabilities associated with shares issued subject to repurchase rights | $ 15,000 | $ 68,000 | |||||
Unvested Early Exercised Stock | |||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||||
Shares repurchased | 997 | 41,961 | |||||
Payments for repurchase of shares | $ 1,000 | $ 45,000 | |||||
Employee Stock Purchase Plan | |||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||||
Number of shares reserved for issuance under the option plan, description | An annual increase on January 1 of each year beginning on January 1, 2017 | ||||||
Stock Options | |||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||||
Shares reserved for issuance, shares | 3,864,407 | 2,964,549 | |||||
RSU | |||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||||
Total grant date fair value vested | $ 159,000 | ||||||
Stock option average expected recognition period | 3 years 2 months 12 days | ||||||
Unamortized stock-based compensation relating to RSUs | $ 7,000,000 | ||||||
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 | 904,785 | 863,538 | 1,432,511 | ||||
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 | 1,611,557 | ||||||
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 | 358,127 | |||||
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 | 402,889 |
Stock-Based Compensation - Summ
Stock-Based Compensation - Summary of Stock Option and Related Information (Details) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |||
Shares Subject to Outstanding Options, Beginning Balance | 2,964,549 | 2,141,868 | |
Shares Subject to Outstanding Options, granted | 1,370,381 | 1,735,875 | |
Shares Subject to Outstanding Options, exercised | (489,153) | (333,822) | |
Shares Subject to Outstanding Options, canceled | (144,316) | (579,372) | |
Shares Subject to Outstanding Options, Ending Balance | 3,701,461 | 2,964,549 | 2,141,868 |
Shares Subject to Outstanding Options, Options outstanding and exercisable | 1,519,637 | ||
Weighted Average Exercise Price per Share, Beginning Balance | $ 11.54 | $ 6.42 | |
Weighted Average Exercise Price Per Share, granted | 52.53 | 15.41 | |
Weighted Average Exercise Price per Share, exercised | 10.61 | 4.81 | |
Weighted Average Exercise Price Per Share, cancelled | 22.67 | 8.11 | |
Weighted Average Exercise Price per Share, Ending Balance | 26.40 | $ 11.54 | $ 6.42 |
Weighted Average Exercise Price Per Share, Options outstanding and exercisable | $ 14.35 | ||
Weighted Average Remaining Contractual Term | 8 years | 8 years 3 months 18 days | 8 years 4 months 24 days |
Weighted Average Remaining Contractual Term, Options outstanding and exercisable | 7 years 3 months 18 days | ||
Aggregate Intrinsic Value, Beginning Balance | $ 88,524 | $ 90,780 | $ 14,963 |
Options outstanding and exercisable as of December 31, 2018 | $ 52,979 |
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, 2018USD ($)$ / sharesshares | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
Shares Subject to Outstanding Awards, RSUs awarded | shares | 171,766 |
Shares Subject to Outstanding Awards, RSUs released | shares | (3,250) |
Shares Subject to Outstanding Awards, RSUs forfeited | shares | (5,570) |
Shares Subject to Outstanding Awards, Ending Balance | shares | 162,946 |
Weighted Average Grant Date Fair Value, RSUs awarded | $ / shares | $ 53.27 |
Weighted Average Grant Date Fair Value, RSUs released | $ / shares | 48.93 |
Weighted Average Grant Date Fair Value, RSUs forfeited | $ / shares | 52.83 |
Weighted Average Grant Date Fair Value, Ending Balance | $ / shares | $ 53.37 |
Weighted Average Remaining Contractual Term | 2 years |
Aggregate Intrinsic Value | $ | $ 7,962 |
Stock-Based Compensation - Sche
Stock-Based Compensation - Schedule of Stock-Based Compensation Expense, Net of Forfeitures (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | |||
Stock-based compensation expense | $ 19,341 | $ 6,138 | $ 2,813 |
Research and development | |||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | |||
Stock-based compensation expense | 8,144 | 2,752 | 1,252 |
General and administrative | |||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | |||
Stock-based compensation expense | $ 11,197 | $ 3,386 | $ 1,561 |
Stock-Based Compensation - Sc_2
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, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||
Risk-free interest rate, minimum | 2.54% | 1.92% | 1.05% |
Expected life (in years) | 5 years 3 months 18 days | 5 years 3 months 18 days | 5 years 3 months 18 days |
Volatility, minimum | 69.00% | 71.00% | 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 | 2.98% | 2.98% | 2.10% |
Expected life (in years) | 6 years 1 month 6 days | 6 years 1 month 6 days | 6 years 1 month 6 days |
Volatility, maximum | 75.00% | 75.00% |
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, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2017 | Sep. 30, 2017 | Jun. 30, 2017 | Mar. 31, 2017 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Numerator | |||||||||||
Net loss | $ (67,698) | $ (57,010) | $ (10,380) | ||||||||
Denominator | |||||||||||
Weighted average shares outstanding | 38,466,233 | 33,098,571 | 28,104,991 | ||||||||
Less: weighted average shares subject to repurchase | (79,327) | (266,057) | (629,199) | ||||||||
Weighted average shares used to compute basic and diluted net loss per share | 40,259,575 | 40,116,644 | 37,440,024 | 35,827,235 | 35,684,201 | 33,525,567 | 31,200,773 | 31,089,310 | 38,386,906 | 32,832,514 | 27,475,792 |
Net loss per share, basic and diluted | $ (0.39) | $ (0.39) | $ (0.49) | $ (0.50) | $ (0.28) | $ (0.50) | $ (0.50) | $ (0.47) | $ (1.76) | $ (1.74) | $ (0.38) |
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, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
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 | 409,839 |
Options and Awards Issued and Outstanding | |||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | |||
Antidilutive securities excluded from earnings per share | 3,864,407 | 2,964,549 | 2,141,868 |
Net Loss per Share - Additional
Net Loss per Share - Additional Information (Details) - 2015 ESPP - USD ($) | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Earnings Per Share [Line Items] | ||
ESPP contributions from plan participants | $ 272,000 | $ 133,000 |
Contributions converted shares under benefit plan | 5,703 | 4,030 |
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, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Income Taxes [Line Items] | ||||
Effective income tax rate and tax provision from continuing operations | 0.00% | 0.00% | 0.00% | |
Federal statutory income tax rate | 21.00% | 34.00% | 34.00% | |
Tax Cuts and Jobs Act, income tax expense (benefit) | $ 0 | |||
Deferred tax assets, relates to net operating loss carryforwards | 49,689,000 | $ 22,246,000 | ||
Deferred tax assets, operating loss foreign | 177,000,000 | |||
Deferred tax assets, operating loss state | $ 179,400,000 | |||
Operating loss expiration date | 2,032 | |||
Research and development tax credit | $ 12,908,000 | 8,631,000 | ||
Increase in valuation allowance | $ 25,700,000 | 13,700,000 | $ 4,900,000 | |
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 | |||
Reserve for unrecognized tax benefits | $ 3,810,000 | $ 2,471,000 | $ 1,474,000 | $ 719,000 |
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 | $ 4,500,000 | |||
Research and development tax credit carryforward, expiration year | 2,032 | |||
Orphan tax credit carryovers, expiration beginning year | 2,036 | |||
Orphan tax credit carryovers | $ 9,000,000 | |||
State | ||||
Income Taxes [Line Items] | ||||
Research and development tax credit | $ 3,800,000 |
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, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Income Tax Disclosure [Abstract] | |||
Federal statutory income tax rate | 21.00% | 34.00% | 34.00% |
State taxes (tax effected) | 8.60% | 7.10% | 8.40% |
Non-deductible expenses and other | 2.50% | 2.00% | (12.90%) |
Research and development credits | 5.90% | 6.00% | 17.80% |
Tax Act – net deferred tax rate change | (25.10%) | ||
Change in valuation allowance | (38.00%) | (24.00%) | (47.30%) |
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, 2018 | Dec. 31, 2017 |
Deferred tax assets: | ||
Net operating loss carryforwards | $ 49,689 | $ 22,246 |
Research and development credits carryforwards | 12,908 | 8,631 |
Stock-based compensation | 4,349 | 890 |
Deferred revenue | 9,391 | |
Start-up costs | 1,237 | 1,354 |
Depreciation | (959) | (457) |
Other | 1,858 | 1,327 |
Total deferred tax assets | 69,082 | 43,382 |
Less: valuation allowance | $ (69,082) | $ (43,382) |
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, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Income Tax Disclosure [Abstract] | |||
Beginning balance | $ 2,471 | $ 1,474 | $ 719 |
Increases (decreases) of unrecognized tax benefits related to prior year | 289 | (97) | |
Increases of unrecognized tax benefits related to current year | 1,050 | 1,094 | 755 |
Ending balance | $ 3,810 | $ 2,471 | $ 1,474 |
Quarterly Financial Data (una_3
Quarterly Financial Data (unaudited) - Summary of Unaudited Quarterly Financial Data (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2017 | Sep. 30, 2017 | Jun. 30, 2017 | Mar. 31, 2017 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Quarterly Financial Data [Abstract] | |||||||||||
Revenues | $ 12,400 | $ 9,188 | $ 6,639 | $ 5,331 | $ 2,954 | $ 3,077 | $ 3,001 | $ 2,410 | $ 11,442 | $ 41,971 | |
Total operating expenses | 30,281 | 26,867 | 26,130 | 23,931 | 13,700 | 20,245 | 18,771 | 17,393 | $ 107,209 | 70,109 | 52,504 |
Net loss | $ (15,676) | $ (15,789) | $ (18,413) | $ (17,820) | $ (10,066) | $ (16,721) | $ (15,461) | $ (14,762) | $ (67,698) | $ (57,010) | $ (10,380) |
Net loss per common share, basic and diluted | $ (0.39) | $ (0.39) | $ (0.49) | $ (0.50) | $ (0.28) | $ (0.50) | $ (0.50) | $ (0.47) | $ (1.76) | $ (1.74) | $ (0.38) |
Weighted average number of shares, basic and diluted | 40,259,575 | 40,116,644 | 37,440,024 | 35,827,235 | 35,684,201 | 33,525,567 | 31,200,773 | 31,089,310 | 38,386,906 | 32,832,514 | 27,475,792 |