Document and Entity Information
Document and Entity Information - USD ($) $ in Billions | 12 Months Ended | ||
Dec. 31, 2016 | Feb. 13, 2017 | Jun. 30, 2016 | |
Document And Entity Information [Abstract] | |||
Document Type | 10-K | ||
Amendment Flag | false | ||
Document Period End Date | Dec. 31, 2016 | ||
Document Fiscal Year Focus | 2,016 | ||
Document Fiscal Period Focus | FY | ||
Trading Symbol | RARE | ||
Entity Registrant Name | Ultragenyx Pharmaceutical Inc. | ||
Entity Central Index Key | 1,515,673 | ||
Current Fiscal Year End Date | --12-31 | ||
Entity Well-known Seasoned Issuer | Yes | ||
Entity Current Reporting Status | Yes | ||
Entity Voluntary Filers | No | ||
Entity Filer Category | Large Accelerated Filer | ||
Entity Common Stock, Shares Outstanding | 41,726,736 | ||
Entity Public Float | $ 1.9 |
CONSOLIDATED BALANCE SHEETS
CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands | Dec. 31, 2016 | Dec. 31, 2015 |
Current assets: | ||
Cash and cash equivalents | $ 161,120 | $ 93,569 |
Short-term investments | 219,028 | 343,428 |
Restricted cash | 1,411 | 150 |
Prepaid expenses and other current assets | 20,136 | 13,060 |
Total current assets | 401,695 | 450,207 |
Property and equipment, net | 17,055 | 7,373 |
Restricted cash | 2,076 | 2,135 |
Long-term investments | 117,963 | 99,259 |
Other assets | 1,837 | 595 |
Total assets | 540,626 | 559,569 |
Current liabilities: | ||
Accounts payable | 5,364 | 2,942 |
Accrued liabilities | 54,554 | 24,784 |
Deferred rent—current portion | 341 | 192 |
Total current liabilities | 60,259 | 27,918 |
Other liabilities | 6,393 | 561 |
Total liabilities | 66,652 | 28,479 |
Commitments and contingencies (Note 10) | ||
Stockholders’ equity: | ||
Preferred stock, par value of $0.001 per share—25,000,000 shares authorized; nil outstanding as of December 31, 2016 and 2015 | ||
Common stock, par value of $0.001 per share—250,000,000 shares authorized; 41,240,230 and 38,882,394 shares issued and outstanding as of December 31, 2016 and 2015 | 41 | 39 |
Additional paid-in capital | 1,003,561 | 816,578 |
Accumulated other comprehensive income (loss) | 905 | (868) |
Accumulated deficit | (530,533) | (284,659) |
Total stockholders’ equity | 473,974 | 531,090 |
Total liabilities and stockholders’ equity | $ 540,626 | $ 559,569 |
CONSOLIDATED BALANCE SHEETS (Pa
CONSOLIDATED BALANCE SHEETS (Parenthetical) - $ / shares | Dec. 31, 2016 | Dec. 31, 2015 |
Statement Of Financial Position [Abstract] | ||
Preferred stock, par value | $ 0.001 | $ 0.001 |
Preferred stock, shares authorized | 25,000,000 | 25,000,000 |
Preferred stock, shares outstanding | 0 | 0 |
Common stock, par value | $ 0.001 | $ 0.001 |
Common stock, shares authorized | 250,000,000 | 250,000,000 |
Common stock, shares issued | 41,240,230 | 38,882,394 |
Common stock, shares outstanding | 41,240,230 | 38,882,394 |
CONSOLIDATED STATEMENTS OF OPER
CONSOLIDATED STATEMENTS OF OPERATIONS - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | |
Income Statement [Abstract] | |||
Revenue | $ 133 | ||
Operating expenses: | |||
Research and development | 183,204 | $ 114,737 | $ 45,967 |
General and administrative | 64,936 | 33,001 | 10,811 |
Total operating expenses | 248,140 | 147,738 | 56,778 |
Loss from operations | (248,007) | (147,738) | (56,778) |
Other income (expense), net: | |||
Interest income | 3,789 | 2,320 | 608 |
Other expense, net | (1,621) | (200) | (3,632) |
Total other income (expense), net | 2,168 | 2,120 | (3,024) |
Loss before income taxes | (245,839) | (145,618) | (59,802) |
Income tax provision | (35) | ||
Net loss | (245,874) | (145,618) | (59,802) |
Net loss attributable to common stockholders | $ (245,874) | $ (145,618) | $ (64,610) |
Net loss per share attributable to common stockholders, basic and diluted | $ (6.21) | $ (3.96) | $ (2.25) |
Shares used in computing net loss per share attributable to common stockholders, basic and diluted | 39,586,908 | 36,782,603 | 28,755,758 |
CONSOLIDATED STATEMENTS OF COMP
CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | |
Statement Of Income And Comprehensive Income [Abstract] | |||
Net loss | $ (245,874) | $ (145,618) | $ (59,802) |
Other comprehensive income (loss): | |||
Foreign currency translation adjustments | 1,322 | ||
Unrealized gain (loss) on available-for-sale securities | 451 | (694) | (185) |
Other comprehensive income (loss): | 1,773 | (694) | (185) |
Total comprehensive loss | $ (244,101) | $ (146,312) | $ (59,987) |
CONSOLIDATED STATEMENTS OF CONV
CONSOLIDATED STATEMENTS OF CONVERTIBLE PREFERRED STOCK AND SHAREHOLDERS' EQUITY (DEFICIT) - USD ($) $ in Thousands | Total | Series A Redeemable Convertible Preferred Stock | Series B Convertible Preferred Stock | Common Stock | Additional Paid-in Capital | Accumulated Other Comprehensive Income (Loss) | Accumulated Deficit |
Beginning balance at Dec. 31, 2013 | $ (74,821) | $ 4 | $ 11 | $ (74,836) | |||
Beginning balance, shares at Dec. 31, 2013 | 3,766,289 | ||||||
Beginning balance at Dec. 31, 2013 | $ 51,001 | $ 73,929 | |||||
Beginning balance, shares at Dec. 31, 2013 | 34,349,894 | 27,081,680 | |||||
Accretion on convertible preferred stock | $ 4,430 | ||||||
Accretion on convertible preferred stock | (4,430) | $ (27) | (4,403) | ||||
Conversion of convertible preferred stock to common stock | 129,360 | $ (55,431) | $ (73,929) | $ 19 | 129,341 | ||
Conversion of convertible preferred stock to common stock, shares | (34,349,894) | (27,081,680) | 19,598,486 | ||||
Reclassification of preferred stock warrant liability | 6,743 | 6,743 | |||||
Issuance of common stock in connection with initial public offering, net of issuance costs and preferred stock dividend | 121,711 | $ 7 | 121,704 | ||||
Issuance of common stock in connection with initial public offering, net of issuance costs and preferred stock dividend, shares | 6,624,423 | ||||||
Issuance of common stock in connection with underwritten public offering, net of issuance costs | 60,239 | $ 2 | 60,237 | ||||
Issuance of common stock in connection with underwritten public offering, net of issuance costs, shares | 1,613,879 | ||||||
Employee stock-based compensation | 5,394 | 5,394 | |||||
Issuance of common stock upon exercise of stock options and warrants | $ 736 | 736 | |||||
Issuance of common stock upon exercise of stock options and warrants, shares | 305,090 | 331,605 | |||||
Other comprehensive income (loss) | $ (185) | (185) | |||||
Net loss | (59,802) | (59,802) | |||||
Ending balance at Dec. 31, 2014 | 184,945 | $ 32 | 324,128 | (174) | (139,041) | ||
Ending balance, shares at Dec. 31, 2014 | 31,934,682 | ||||||
Issuance of common stock in connection with underwritten public offering, net of issuance costs | 461,136 | $ 6 | 461,130 | ||||
Issuance of common stock in connection with underwritten public offering, net of issuance costs, shares | 5,980,000 | ||||||
Employee stock-based compensation | 24,884 | 24,884 | |||||
Issuance of common stock upon exercise of stock options and warrants | $ 7,355 | $ 1 | 7,354 | ||||
Issuance of common stock upon exercise of stock options and warrants, shares | 795,825 | 957,587 | |||||
Restricted stock units vested during the period, net | $ (918) | (918) | |||||
Restricted stock units vested during the period, net of shares | 10,125 | ||||||
Other comprehensive income (loss) | (694) | (694) | |||||
Net loss | (145,618) | (145,618) | |||||
Ending balance at Dec. 31, 2015 | $ 531,090 | $ 39 | 816,578 | (868) | (284,659) | ||
Ending balance, shares at Dec. 31, 2015 | 38,882,394 | 38,882,394 | |||||
Issuance of common stock in connection with at-the-market offering, net of issuance costs | $ 79,486 | $ 1 | 79,485 | ||||
Issuance of common stock in connection with at-the-market offering, net of issuance costs, shares | 1,159,415 | ||||||
Issuance of common stock in connection with collaboration agreement, net of issuance costs | 52,272 | $ 1 | 52,271 | ||||
Issuance of common stock in connection with collaboration agreement, net of issuance costs, shares | 727,120 | ||||||
Put option grant in connection with collaboration agreement | (916) | (916) | |||||
Employee stock-based compensation | 48,309 | 48,309 | |||||
Issuance of common stock upon exercise of stock options and warrants | $ 9,096 | 9,096 | |||||
Issuance of common stock upon exercise of stock options and warrants, shares | 425,922 | 438,811 | |||||
Restricted stock units vested during the period, net | $ (1,262) | (1,262) | |||||
Restricted stock units vested during the period, net of shares | 32,490 | ||||||
Other comprehensive income (loss) | 1,773 | 1,773 | |||||
Net loss | (245,874) | (245,874) | |||||
Ending balance at Dec. 31, 2016 | $ 473,974 | $ 41 | $ 1,003,561 | $ 905 | $ (530,533) | ||
Ending balance, shares at Dec. 31, 2016 | 41,240,230 | 41,240,230 |
CONSOLIDATED STATEMENTS OF CASH
CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | |
Operating activities: | |||
Net loss | $ (245,874) | $ (145,618) | $ (59,802) |
Adjustments to reconcile net loss to net cash used in operating activities: | |||
Stock-based compensation | 48,309 | 24,884 | 5,394 |
Amortization of premium on investment securities, net | 4,842 | 5,637 | 3,600 |
Depreciation and amortization | 3,424 | 1,384 | 684 |
Revaluation of convertible preferred stock warrant liability | 0 | 0 | 3,324 |
Foreign currency remeasurement loss | 1,322 | ||
Non-cash license fee from collaboration arrangement | 700 | ||
Changes in operating assets and liabilities: | |||
Prepaid expenses and other current assets | (7,147) | (7,131) | (4,081) |
Other assets | (1,242) | 179 | (411) |
Accounts payable | 2,502 | (1,982) | 3,177 |
Accrued liabilities and other liabilities | 32,189 | 16,670 | 3,481 |
Net cash used in operating activities | (160,975) | (105,977) | (44,634) |
Investing activities: | |||
Purchase of property and equipment | (10,188) | (4,955) | (2,149) |
Purchase of investments | (442,490) | (624,226) | (208,972) |
Proceeds from sale of investments | 140,556 | 89,321 | 5,002 |
Proceeds from maturities of investments | 403,239 | 249,050 | 82,972 |
Increase in restricted cash | (1,202) | (1,541) | (293) |
Net cash provided by (used in) investing activities | 89,915 | (292,351) | (123,440) |
Financing activities: | |||
Proceeds from the issuance of common stock in connection with underwritten public offerings, net | 461,136 | 188,581 | |
Proceeds from the issuance of common stock in connection with at-the-market offering, net | 79,486 | ||
Proceeds from the issuance of common stock in connection with collaboration agreement, net | 51,356 | ||
Proceeds from the issuance of common stock from equity awards, net | 7,834 | 6,437 | 736 |
Payment of preferred stock dividend | (4,346) | ||
Net cash provided by financing activities | 138,676 | 467,573 | 184,971 |
Effect of exchange rate changes on cash | (65) | ||
Net increase in cash and cash equivalents | 67,551 | 69,245 | 16,897 |
Cash and cash equivalents at beginning of year | 93,569 | 24,324 | 7,427 |
Cash and cash equivalents at end of year | 161,120 | 93,569 | 24,324 |
Supplemental disclosures of non-cash investing and financing information: | |||
Costs of property and equipment included in accounts payable and accrued liabilities | 147 | $ 769 | 243 |
Tenant improvement allowance | $ 3,467 | ||
Reclassification of warrant liability to equity upon conversion to common stock warrants | 6,743 | ||
Conversion of Series A and Series B preferred stock to common stock | $ 129,360 |
Organization and Basis of Prese
Organization and Basis of Presentation | 12 Months Ended |
Dec. 31, 2016 | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | |
Organization and Basis of Presentation | 1. Organization and Basis of Presentation Ultragenyx Pharmaceutical Inc. (the Company) is a biopharmaceutical company and was incorporated in California on April 22, 2010. The Company subsequently reincorporated in the state of Delaware in June 2011. The Company is focused on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. The Company is currently conducting a Phase 3 study of aceneuramic acid extended-release (Ace-ER) in patients with GNE myopathy, which is also known as hereditary inclusion body myopathy, a progressive muscle-wasting disorder; a Phase 3 study of recombinant human beta-glucuronidase (rhGUS) in patients with mucopolysaccharidosis 7 (MPS 7), a rare lysosomal storage disease; a Phase 2 clinical study for UX007 in patients with glucose transporter type-1 deficiency syndrome (Glut1 DS), a brain energy deficiency; a Phase 2 clinical study of UX007 in patients severely affected by long-chain fatty acid oxidation disorders (LC-FAOD), a genetic disorder in which the body is unable to convert long chain fatty acids into energy; and Phase 2 and Phase 3 studies of KRN23, an antibody targeting fibroblast growth factor 23, or FGF23, in patients with X-linked hypophosphatemia (XLH) and tumor-induced osteomalacia (TIO), both rare diseases that impair bone mineralization. The Company operates as one reportable segment. In the course of its research activities, the Company has sustained operating losses and expects such losses to continue over the next several years. The Company’s ultimate success depends on the outcome of its research and development activities. Management expects to incur additional losses in the future to conduct product research and development and recognizes the need to raise additional capital to fully implement its business plan. Through December 31, 2016, the Company has relied primarily on the proceeds from equity offerings to finance its operations. The Company intends to raise additional capital through the issuance of equity, borrowings, or strategic alliances with partner companies. However, if such financing is not available at adequate levels, the Company will need to reevaluate its operating plans. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 12 Months Ended |
Dec. 31, 2016 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies Basis of Consolidation The consolidated financial statements include the accounts of Ultragenyx Pharmaceutical Inc. and our wholly-owned subsidiaries. All intercompany balances and transactions have been eliminated. Use of Estimates The accompanying consolidated financial statements have been prepared in accordance with U.S. generally accepted accounting principles (“GAAP”). The preparation of the consolidated financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, disclosure of contingent liabilities and the reported amounts of expenses in the consolidated financial statements and the accompanying notes. On an ongoing basis, management evaluates its estimates, including those related to clinical trial accruals, fair value of assets and liabilities, 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. Cash and Cash Equivalents The Company considers all highly liquid investments with original maturities of three months or less from the date of purchase to be cash equivalents. Cash equivalents consist primarily of amounts invested in money market accounts and corporate bonds. Investments All investments have been classified as “available-for-sale” and are carried at estimated fair value as determined based upon quoted market prices or pricing models for similar securities. Management determines the appropriate classification of its investments in debt securities at the time of purchase and reevaluates such designation as of each balance sheet date. Investments with a maturity of one year or less from the balance sheet date are reported as short-term investments and investments with a maturity of greater than one year from the balance sheet date are reported as long-term investments. Unrealized gains and losses are excluded from earnings and are reported as a component of comprehensive loss. Realized gains and losses and declines in fair value judged to be other than temporary, if any, on available-for-sale securities are included in other expense, net. The cost of securities sold is based on the specific-identification method. Interest on investments is included in interest income. Concentration of Credit Risk and Other Risks and Uncertainties Financial instruments that potentially subject the Company to a concentration of credit risk consist of cash, cash equivalents, and investments. The Company’s cash, cash equivalents, and investments are held by financial institutions that management believes are of high credit quality. The Company’s investment policy limits investments to fixed income securities denominated and payable in U.S. dollars such as U.S. government obligations, money market instruments and funds, corporate bonds, and asset-backed securities and places restrictions on maturities and concentrations by type and issuer. Such deposits may, at times, exceed federally insured limits. The Company has not experienced any losses on its deposits of cash and cash equivalents and its accounts are monitored by management to mitigate risk. The Company is exposed to credit risk in the event of default by the financial institutions holding its cash and cash equivalents, corporate bond issuers and other financial instruments to the extent recorded in the balance sheets . Property and Equipment Property and equipment are stated at cost, less accumulated depreciation and amortization. Depreciation and amortization is computed using the straight-line method over the estimated useful lives of the respective assets. Depreciation and amortization begins at the time the asset is placed in service. Maintenance and repairs are charged to operations as incurred. Upon sale or retirement of assets, the cost and related accumulated depreciation are removed from the balance sheet and the resulting gain or loss, if any, is reflected in operations. The useful lives of the property and equipment are as follows: Research and development equipment 5 years Furniture and office equipment 5 years Computer equipment 3 years Software 3-5 years Leasehold improvements Shorter of lease term or estimated useful life Impairment of Long-Lived Assets The Company evaluates its long-lived assets, including property and equipment, for impairment whenever events or changes in circumstances indicate that the carrying value of these assets may not be recoverable. Recoverability of these assets is measured by comparison of the carrying amount of each asset to the future undiscounted cash flows expected to result from the use of the asset and its eventual disposition. If the asset is considered to be impaired, the amount of any impairment is measured as the difference between the carrying value and the fair value of the impaired asset. The Company has not recorded impairment of any long-lived assets since inception. Restricted Cash Restricted cash primarily consists of money market accounts as collateral for its obligations under its facility leases of the Company’s corporate headquarters in Novato, California and for its facilities in Brisbane, California. Accruals of Research and Development Costs The Company records accruals for estimated costs of research, preclinical and clinical studies and manufacturing development. These costs are a significant component of the Company’s research and development expenses. A substantial portion of the Company’s ongoing research and development activities are conducted by third-party service providers, including contract research organizations. The Company accrues the costs incurred under its agreements with these third parties based on actual work completed in accordance with agreements established with these third parties. The Company determines the actual costs through discussions with internal personnel and external service providers as to the progress or stage of completion of the services and the agreed-upon fee to be paid for such services. The Company makes significant judgments and estimates in determining the accrual balance in each reporting period. As actual costs become known, the Company adjusts its accruals. The Company has not experienced any material deviations between accrued clinical trial expenses and actual clinical trial expenses. However, actual services performed, number of patients enrolled, and the rate of patient enrollment may vary from the Company’s estimates, resulting in adjustments to clinical trial expense in future periods. Changes in these estimates that result in material changes to the Company’s accruals could materially affect the Company’s results of operations. Revenue The Company recognizes revenue when persuasive evidence of an arrangement exists, delivery has occurred, the price is fixed or determinable, and collectability is reasonably assured. Revenue is recognized once all revenue recognition criteria are met. During the year ended December 31, 2016, the Company recognized revenue from sales of rhGUS (UX003) on a “named patient” basis which are allowed in certain European countries prior to the commercial approval of the product in the territory. Due to the Company’s limited sales and collection history to date, revenue has been recognized upon receipt of payment. Leases The Company enters into lease agreements for its office and laboratory facilities. These leases are classified as operating leases. Rent expense is recognized on a straight-line basis over the term of the lease and, accordingly, the Company records the difference between cash rent payments and the recognition of rent expense as a deferred rent liability. Incentives granted under the Company’s facilities leases, including allowances to fund leasehold improvements, are deferred and are recognized as adjustments to rental expense on a straight-line basis over the term of the lease. Comprehensive Loss Comprehensive loss is the change in stockholders’ equity from transactions and other events and circumstances other than those resulting from investments by stockholders and distributions to stockholders. The Company’s other comprehensive loss is comprised of unrealized gains and losses on investments in available-for-sale securities and foreign currency translation adjustments. Research and Development Research and development costs are expensed as incurred and consist of salaries and benefits, stock-based compensation expense, lab supplies and facility costs, as well as fees paid to other nonemployees and entities that conduct certain research and development activities on the Company’s behalf. Amounts incurred in connection with license agreements are also included in research and development expense. Nonrefundable advance payments for goods or services to be received in the future for use in research and development activities are deferred and capitalized. The capitalized amounts are expensed as the related goods are delivered or the services are performed. Stock-Based Compensation Stock-based awards issued to employees, including stock options and restricted stock units (RSUs), are recorded at fair value as of the grant date and recognized as expense on a straight-line basis over the employee’s requisite service period (generally the vesting period). Because noncash stock compensation expense is based on awards ultimately expected to vest, it is reduced by an estimate for future forfeitures. Forfeitures are estimated at the time of grant and revised, if necessary, in subsequent periods if actual forfeitures differ from estimates. Income Taxes The Company uses the liability method of accounting for income taxes. Under this method, deferred tax assets and liabilities are determined based on the differences between the financial reporting and the tax bases of assets and liabilities and are measured using the enacted tax rates and laws that will be in effect when the differences are expected to reverse. The Company must then assess the likelihood that the resulting deferred tax assets will be realized. A valuation allowance is provided when it is more likely than not that some portion or all of a deferred tax asset will not be realized. Due to the Company’s lack of earnings history, the net deferred tax assets have been fully offset by a valuation allowance. The Company recognizes benefits of uncertain tax positions if it is more likely than not that such positions will be sustained upon examination based solely on their technical merits, as the largest amount of benefit that is more likely than not to be realized upon the ultimate settlement. The Company’s policy is to recognize interest and penalties related to the underpayment of income taxes as a component of income tax expense or benefit. To date, there have been no interest or penalties charged in relation to the unrecognized tax benefits. Foreign Currency Assets and liabilities of non-U.S. subsidiaries that operate in a local currency environment, where that local currency is the functional currency, are translated to U.S. dollars at exchange rates in effect at the balance sheet date, with the resulting translation adjustments directly recorded to a separate component of accumulated other comprehensive loss. Income and expense accounts are translated at average exchange rates for the period. Remeasurement adjustments are recorded in other income (expense), net. Net Loss per Share Attributable to Common Stockholders Basic net loss per share attributable to common stockholders is calculated by dividing the net loss attributable to common stockholders by the weighted-average number of shares of common stock outstanding during the period, without consideration for common stock equivalents. Diluted net loss per share attributable to common stockholders is the same as basic net loss per share attributable to common stockholders, since the effects of potentially dilutive securities are antidilutive. In periods when we have incurred a net loss, convertible preferred stock, options and warrants to purchase common stock and convertible preferred stock warrants are considered common stock equivalents, but have been excluded from the calculation of diluted net loss per share attributable to common stockholders, as their effect is antidilutive. Recent Accounting Pronouncements In May 2014, the FASB, issued ASU 2014-09, Revenue from Contracts with Customers (Topic 606) Revenue from Contracts with Customers: Principal versus Agent Considerations Revenue from Contracts with Customers: Identifying Performance Obligations and Licensing Revenue from Contracts with Customers: Narrow-Scope Improvements and Practical Expedients to provide supplemental adoption guidance and clarification to ASU 2014-09 Technical Corrections and Improvements to Topic 606, Revenue from Contracts with Customers In February 2016, the FASB issued ASU No. 2016-02, Leases (Topic 842), which requires an entity that is a lessee to record a right of use asset and a corresponding lease liability on the balance sheet for all leases with terms longer than 12 months. This guidance also requires disclosures about the amount, timing, and uncertainty of cash flows arising from leases. This guidance is effective for annual reporting periods beginning after December 15, 2018, and interim periods within those annual periods, using a modified retrospective approach, and early adoption is permitted. The Company is evaluating the effect that this guidance will have on its Consolidated Financial Statements and related disclosures. In March 2016, the FASB issued ASU 2016-09, Compensation — Stock Compensation (Topic 718): Improvements to Employee Share-Based Payment Accounting accounting for employee share-based payments, including income tax consequences, application of award forfeitures to expense, classification on the statement of cash flows, and classification of awards as either equity or liabilities. This guidance is effective for annual reporting periods beginning after December 15, 2016, and interim periods within those annual periods. In October 2016, the FASB issued ASU 2016-16, Income Taxes - Intra-Entity Transfers of Assets Other Than Inventory |
Fair Value Measurements
Fair Value Measurements | 12 Months Ended |
Dec. 31, 2016 | |
Fair Value Disclosures [Abstract] | |
Fair Value Measurements | 3. Fair Value Measurements Financial assets and liabilities are recorded at fair value. The carrying amount of certain financial instruments, including cash and cash equivalents, accounts payable and accrued liabilities approximate fair value due to their relatively short maturities. Assets and liabilities recorded at fair value on a recurring basis in the balance sheets are categorized based upon the level of judgment associated with the inputs used to measure their fair values. Fair value is defined as the exchange price that would be received for an asset or an exit price that would be paid to transfer a liability in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants on the measurement date. The authoritative guidance on fair value measurements establishes a three-tier fair value hierarchy for disclosure of fair value measurements as follows: Level 1 —Inputs are unadjusted, quoted prices in active markets for identical assets or liabilities at the measurement date; Level 2 —Inputs are observable, unadjusted quoted prices in active markets for similar assets or liabilities, unadjusted quoted prices for identical or similar assets or liabilities in markets that are not active, or other inputs that are observable or can be corroborated by observable market data for substantially the full term of the related assets or liabilities; and Level 3 —Unobservable inputs that are significant to the measurement of the fair value of the assets or liabilities that are supported by little or no market data. The Company’s financial instruments consist of Level 1 and Level 2 assets. Where quoted prices are available in an active market, securities are classified as Level 1. Money market funds are classified as Level 1. Level 2 assets consist primarily of corporate bonds, asset backed securities, commercial paper and U.S. Government agency securities based upon quoted market prices for similar movements in active markets, quoted prices for identical or similar instruments in markets that are not active and model-based valuation techniques for which all significant inputs are observable in the market or can be corroborated by observable market data for substantially the full term of the assets. Where applicable these models project future cash flows and discount the future amounts to a present value using market-based observable inputs obtained from various third party data providers, including but not limited to, benchmark yields, interest rate curves, reported trades, broker/dealer quotes and reference data. The following table sets forth the fair value of the Company’s financial assets and liabilities remeasured on a recurring basis based on the three-tier fair value hierarchy (in thousands): December 31, 2016 Level 1 Level 2 Level 3 Total Financial Assets: Money market funds $ 123,536 $ — $ — $ 123,536 Corporate bonds — 207,726 — 207,726 Commercial paper — 11,970 — 11,970 Asset-backed securities — 27,713 — 27,713 U.S. Government Treasury and agency securities — 111,931 — 111,931 Total financial assets $ 123,536 $ 359,340 $ — $ 482,876 December 31, 2015 Level 1 Level 2 Level 3 Total Financial Assets: Money market funds $ 53,254 $ — $ — $ 53,254 Corporate bonds — 370,445 — 370,445 Commercial paper — 13,887 — 13,887 Asset-backed securities — 29,302 — 29,302 U.S. Government Treasury and agency securities — 47,452 — 47,452 Total financial assets $ 53,254 $ 461,086 $ — $ 514,340 |
Balance Sheet Components
Balance Sheet Components | 12 Months Ended |
Dec. 31, 2016 | |
Investments Debt And Equity Securities [Abstract] | |
Balance Sheet Components | 4. Balance Sheet Components Cash Equivalents and Investments The fair values of cash equivalents, short-term investments, and long-term investments classified as available-for-sale securities, consisted of the following (in thousands): December 31, 2016 Gross Unrealized Amortized Cost Gains Losses Estimated Fair Value Money market funds $ 123,536 $ — $ — $ 123,536 Corporate bonds 207,909 14 (197 ) 207,726 Commercial paper 11,970 — — 11,970 Asset-backed securities 27,712 3 (2 ) 27,713 U.S. Government Treasury and agency securities 112,166 10 (245 ) 111,931 Total $ 483,293 $ 27 $ (444 ) $ 482,876 December 31, 2015 Gross Unrealized Amortized Cost Gains Losses Estimated Fair Value Money market funds $ 53,254 $ — $ — $ 53,254 Corporate bonds 371,098 11 (664 ) 370,445 Commercial paper 13,887 — — 13,887 Asset-backed securities 29,356 — (54 ) 29,302 U.S. Government Treasury and agency securities 47,613 — (161 ) 47,452 Total $ 515,208 $ 11 $ (879 ) $ 514,340 At December 31, 2016, the remaining contractual maturities of available-for-sale securities were less than two years. There have been no significant realized gains or losses on available-for-sale securities for the periods presented. All marketable securities with unrealized losses at December 31, 2016 have been in a loss position for less than twelve months or the loss is not material and were temporary in nature. We do not intend to sell the investments that are in an unrealized loss position before recovery of their amortized cost basis. Property and Equipment, net Property and equipment, net consists of the following (in thousands): December 31, 2016 2015 Research and development equipment $ 2,154 $ 1,302 Furniture and office equipment 2,595 944 Computer equipment 2,013 628 Software 3,931 1,622 Leasehold improvements 12,486 3,466 Construction-in-progress 37 2,276 Property and equipment, gross 23,216 10,238 Less accumulated depreciation (6,161 ) (2,865 ) Property and equipment, net $ 17,055 $ 7,373 Depreciation expense for the years ended December 31, 2016, 2015 and 2014 was $3.4 million, $1.4 million and $0.7 million respectively. Amortization of leasehold improvements and software is included in depreciation expense. Accrued Liabilities Accrued liabilities consist of the following (in thousands): December 31, 2016 2015 Research and clinical study expenses $ 18,593 $ 9,764 Payroll and related expenses 17,226 9,423 Repayment liability under collaboration agreement 13,650 — Other 5,085 5,597 Total accrued liabilities $ 54,554 $ 24,784 |
License and Research Agreements
License and Research Agreements | 12 Months Ended |
Dec. 31, 2016 | |
Research Grant Agreement [Abstract] | |
License and Research Agreements | 5. License and Research Agreements Nobelpharma License Agreement In September 2010, the Company entered into a collaboration and license agreement with Nobelpharma Co., Ltd. (Nobelpharma), which was amended in August 2015. Under the terms of this collaboration and license agreement, each party granted the other party a worldwide exclusive license under certain of that party’s intellectual property related to the compound identified as N-acetylneuraminic acid, also known as sialic acid, to develop, manufacture, and commercialize products. Nobelpharma’s licensed territory includes Japan and certain other Asian countries, and the Company’s licensed territory includes the rest of the world. The Company is required to make certain payments to Nobelpharma based upon achievement of certain development and approval milestones. The Company may make future payments that aggregate up to 200 million Yen (approximately $1.7 million based on the exchange rate at December 31, 2016) that are contingent upon attainment of various development and approval milestones. The Company will pay a mid-single digit royalty on net sales in the Company’s territory and will receive a mid-single digit royalty on net sales in the Nobelpharma territory, excluding Japan, if such product sales are ever achieved. Saint Louis University License Agreement In November 2010, the Company entered into a license agreement with Saint Louis University (SLU). Under the terms of this license agreement, SLU granted the Company an exclusive worldwide license to make, have made, use, import, offer for sale, and sell therapeutics related to SLU’s beta-glucuronidase product for use in the treatment of human diseases. The Company will be required to make a milestone payment of $0.1 million upon approval of a glucuronidase-based enzyme therapy for treatment of MPS 7. Additionally, upon reaching a certain level of cumulative worldwide sales of the product, the Company will be required to pay to SLU a low single-digit royalty on net sales of the licensed products in any country or region, if such product sales are ever achieved. Alcami Corporation License Agreement In March 2011, the Company entered into a license agreement with Alcami Corporation (Alcami). Under the terms of this license agreement, Alcami granted the Company a fully paid-up, royalty-free, exclusive, perpetual, and irrevocable license to research, develop, make, have made, use, import, offer for sale, and sell products incorporating Alcami’s controlled release matrix solid dose oral tablet. Under the license agreement, the Company will pay a mid-single digit percentage of any sublicense revenue received by Ultragenyx related to the sublicense of Alcami technology that had been initially licensed by Ultragenyx. HIBM Research Group In April 2012, the Company entered into an exclusive license agreement with HIBM Research Group (HRG). Under the terms of this license agreement, HRG granted the Company an exclusive worldwide license to certain intellectual property related to the treatment of HIBM. The Company may make future payments that aggregate up to $0.3 million that are contingent upon attainment of various development and approval milestones. Additionally, the Company will pay to HRG a royalty of less than 1% of net sales of the licensed products in the licensed territories, if such product sales are ever achieved. St. Jude Children’s Research Hospital License Agreement In September 2012, the Company entered into a license agreement with St. Jude Children’s Research Hospital (St. Jude). Under the terms of this license agreement, St. Jude granted the Company an exclusive license under certain know-how to research, develop, make, use, offer to sell, import, and otherwise commercialize and exploit St. Jude’s protective protein, cathepsin, a protein product to treat, prevent, and/or diagnose galactosialidosis and other monogenetic diseases. The Company will pay to St. Jude a royalty of less than 1% on net sales of the licensed products in the licensed territories, if such product sales are ever achieved. Baylor Research Institute License Agreement In September 2012, the Company entered into a license agreement with Baylor Research Institute (BRI). Under the terms of this license agreement, BRI exclusively licensed to the Company certain intellectual property related to triheptanoin for North America. In June 2013, the Company notified BRI that it was exercising its option pursuant to the agreement to license the rights to triheptanoin in all territories outside of the United States, Canada and Mexico and paid the option exercise fee of $0.8 million. The Company may make future payments of up to $10.5 million contingent upon attainment of various development milestones and $7.5 million contingent upon attainment of various sales milestones. Additionally, the Company will pay to BRI a mid-single digit royalty on net sales of the licensed product in the licensed territories, if such product sales are ever achieved. Kyowa Hakko Kirin Collaboration and License Agreement In August 2013, the Company entered into a collaboration and license agreement with Kyowa Hakko Kirin Co., Ltd. (KHK). Under the terms of this collaboration and license agreement, as amended, the Company and KHK will collaborate on the development and commercialization of certain products containing KRN23 in the field of orphan diseases in the United States and Canada, or the profit share territory, and in the European Union, Switzerland, and Turkey, or the European territory, and the Company will have the right to develop and commercialize such products in the field of orphan diseases in Mexico and Central and South America, or Latin America. In the field of orphan diseases, and except for ongoing studies being conducted by KHK, the Company will be the lead party for development activities in the profit share territory and in the European territory until the applicable transition date; the Company will also be the lead party for core development activities conducted in Japan and Korea, for which the core development plan is limited to clinical trials mutually agreed to by the Company and KHK. The Company will share the costs for development activities in the profit share territory and the European territory conducted pursuant to the development plan before the applicable transition date equally with KHK, and KHK shall be responsible for 100% of the costs for development activities in Japan and Korea. On the applicable transition date in the profit share territory and the European territory, KHK will become the lead party and be responsible for the costs of the development activities. However, the Company will continue to share the costs of the studies commenced prior to the applicable transition date equally with KHK. The Company has the primary responsibility for conducting certain research and development services. The Company is obligated to provide assistance in accordance with the agreed upon development plan as well as participate on various committees. If KRN23 is approved, the Company and KHK will share commercial responsibilities and profits in the profit share territory until the applicable transition date, KHK will commercialize KRN23 in the European territory, and the Company will develop and commercialize KRN23 in Latin America. KHK will manufacture and supply KRN23 for clinical use globally and will manufacture and supply KRN23 for commercial use in the profit share territory and Latin America. The remaining profit or loss from commercializing products in the profit-share territory, until the applicable transition date, will be shared between the Company and KHK on a 50/50 basis. Thereafter, the Company will be entitled to receive a tiered double-digit revenue share in the mid-to-high 20% range in the profit share territory, intended to approximate the profit share. The Company will also be entitled to receive a royalty of up to 10% on net sales in the European territory. In Latin America, the Company will pay to KHK a low single-digit royalty on net sales. The Company is accounting for the agreement as a collaboration arrangement as defined in ASC 808, Collaborative Agreements. As of December 31, 2016 and 2015, the Company had receivables in the amount of $8.6 million and $3.8 million, respectively, for this collaboration arrangement. Arcturus Research Collaboration and License Agreement In October 2015, the Company entered into a Research Collaboration and License Agreement with Arcturus Therapeutics, Inc. (Arcturus). The Company and Arcturus will collaborate on the research and development of therapies for select rare diseases. As consideration for entering into the arrangement, the Company paid Arcturus an upfront fee of $10.0 million. Arcturus will have the primary responsibility for conducting certain research services, funded by the Company, and the Company will be responsible for development and commercialization costs. Takeda License and Collaboration and Purchase Agreements In June 2016, the Company executed a collaboration and license agreement with Takeda Pharmaceutical Company Limited (Takeda). Pursuant to the agreement, which became effective in July 2016, the Company obtained an exclusive license for a pre-clinical compound from Takeda in a pre-determined field of use, which includes an option to an additional field of use for this product with the terms to be negotiated, and an option to a specified product candidate (identified option product). The Company is responsible for the development costs for the pre-clinical compound and the identified option product pursuant to an initial development plan. Because the license to the pre-clinical compound has no alternative future use, the estimated fair value of $0.7 million was recorded as a research and development expense upon acquisition. Under the license for the pre-clinical compound, the Company may be required to make future milestone payments to Takeda of up to $7.5 million if certain development milestones are met, $75.0 million if certain regulatory milestones are met, and $150.0 million if certain commercial milestones are met, as well as royalties with respect to net sales in the high-single digits to low-teens. Any products resulting from the pre-clinical compound or the identified option product is referred to in this report as the “licensed product.” A s part of the agreement, the Company and Takeda established a five-year research collaboration whereby the parties may mutually agree to add additional option products candidates to the collaboration, in which case the Company will bear the cost of the development activities, with certain exceptions. In July 2016, the Company consummated a common stock purchase agreement, executed in conjunction with the collaboration and license agreement, whereby Takeda purchased 374,590 shares of the Company’s common stock for $40.0 million in cash. The fair market value of the common stock issued to Takeda was $27.3 million, based on the closing stock price of $72.95 on the date of issuance, resulting in a $12.7 million premium paid to the Company. The Company also received a put option to require Takeda to purchase an additional $25.0 million in shares of the Company’s common stock at the then-current 30-day volume-weighted average price (VWAP). The put option was exercised in October 2016, whereby Takeda purchased 352,530 shares of the Company’s common stock for $25.0 million in cash. The Company also received a second put option, which is contingent upon meeting certain development milestones on the identified option product, to require Takeda to purchase an additional $10.0 million in shares of the Company’s common stock at the then-current 30-day VWAP. Takeda was subject to a 180-day lock-up provision as a result of the initial purchase of common stock, and a 90-day lock-up provision as a result of its purchase under the first put option, is subject to a five-year standstill (subject to customary exceptions or release), and has registration rights for purchases of common stock. The Company estimated the fair value of the put options to be $0.9 million and recorded the put options in additional paid-in capital. The valuation was performed using a Monte Carlo simulation approach using the term of the put options and an equity volatility of approximately 70%. The Company also granted Takeda an exclusive option for Asian rights, for a limited period, to any licensed products and any additional products resulting from the collaboration, as well as an option to exclusively license one of the Company’s products for development and commercialization in Japan. If Takeda exercises any of its option rights to license a product pursuant to the agreement, Takeda will pay for the development costs within the licensed territory, will share in a portion of the global development costs, and will make a milestone payment upon regulatory approval. Takeda will also owe royalties on net sales in the licensed territory for any licensed product, depending on the development stage when the product is licensed as well as sales levels. The royalties related to the option to license the Company’s product, as well as the additional product are subject to future good faith negotiations at the time that the option is exercised. The research and license agreement and the stock purchase agreement are being accounted for as one arrangement because they were entered into at the same time. The arrangements were evaluated pursuant to ASC 605-25, Multiple-Element Arrangements In January 2017, the Company terminated the option to the identified option product. As a result, the second put option to require Takeda to purchase an additional $10.0 million in shares of the Company’s common stock was terminated. |
Equity
Equity | 12 Months Ended |
Dec. 31, 2016 | |
Equity [Abstract] | |
Equity | 6. Equity Equity Transactions In July 2016, the Company entered into an At-The-Market (ATM) sales agreement, with Cowen and Company, LLC (Cowen), whereby the Company may sell up to $150.0 million in aggregate proceeds of common stock from time to time, through Cowen as our sales agent. During the year ended December 31, 2016, the Company sold 1,159,415 shares of common stock, resulting in net proceeds of approximately $79.5 million, after commissions and other offering costs. During the period of January 1, 2017 through February 15, 2017, the Company sold an additional 658,532 shares of common stock, resulting in net proceeds of approximately $48.4 million, after commissions and other offering costs. Common Stock Warrants In connection with various financing activities, the Company issued preferred stock warrants. The fair value ascribed to these warrants upon their issuance was $0.2 million, which was determined using an option-pricing method to allocate the equity value of the Company to the warrants based on the Company’s capital structure. Upon the closing of the Company’s IPO in February 2014, the warrants were converted into warrants to purchase common stock. Accordingly, the warrants were reclassified from a liability to permanent equity and were no longer subject to remeasurement. As of December 31, 2016 and 2015, there were 83,167 and 66,533 common stock warrants outstanding that were issued in June 2010 and June 2011, respectively, with a contractual term of 10 years and an exercise price of $3.006. |
Stock-Based Awards
Stock-Based Awards | 12 Months Ended |
Dec. 31, 2016 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Stock-Based Awards | 7. Stock-Based Awards Equity Plan Awards In 2011, the Company adopted the 2011 Equity Incentive Plan (the 2011 Plan). The 2011 Plan provides for the granting of stock-based awards to employees, directors, and consultants under terms and provisions established by the board of directors. In 2014, the Company adopted the 2014 Incentive Plan (the 2014 Plan), which became effective upon the closing of the Company’s IPO in February 2014. The 2014 Plan had 2,250,000 shares of common stock available for future issuance at the time of its inception, which included 655,038 shares available under the 2011 Plan, which were transferred to the 2014 Plan upon adoption. No further grants subsequent to the IPO were made under the 2011 Plan. The 2014 Plan provides for automatic annual increases in shares available for grant, beginning on January 1, 2015 through January 1, 2024. Under the terms of the 2014 Plan, awards may be granted at an exercise price not less than fair market value. For employees holding more than 10% of the voting rights of all classes of stock, the exercise prices for awards must be at least 110% of fair market of the common stock on the grant date, as determined by the board of directors. The term of an award granted under the 2014 Plan may not exceed ten years. Typically, the vesting schedule for option grants to the employees provides that 1/4 of the grant vests upon the first anniversary of the date of grant, with the remainder of the shares vesting monthly thereafter at a rate of 1/48 of the total shares subject to the option. The vesting schedule for RSU grants provides that 1/4 of the grant vests upon the annual anniversary of the date of grant over the period of four years. As of December 31, 2016, an aggregate of 7,478,107 shares of common stock have been authorized for issuance under all equity award plans. Stock Option Activity The following table summarizes activity under the Company’s stock option plans, including the 2011 Plan and the 2014 Plan and related information: Options Outstanding Number of Options Weighted- Average Exercise Weighted-Average Remaining Contractual Term Aggregate Intrinsic Value (In Outstanding — December 31, 2013 2,228,883 $ 3.41 8.91 $ 19,468 Options granted 932,555 41.12 Options exercised (305,090 ) 2.14 Options cancelled (116,873 ) 8.92 Outstanding — December 31, 2014 2,739,475 $ 16.15 8.43 $ 79,840 Options granted 2,013,350 90.60 Options exercised (795,825 ) 8.65 Options cancelled (130,037 ) 31.49 Outstanding — December 31, 2015 3,826,963 $ 56.36 8.58 $ 217,386 Options granted 1,435,995 67.00 Options exercised (425,922 ) 21.21 Options cancelled (407,564 ) 70.87 Outstanding — December 31, 2016 4,429,472 $ 61.85 8.22 $ 79,135 Vested and exercisable — December 31, 2016 1,620,443 $ 44.96 7.24 $ 55,537 Vested and expected to vest — December 31, 2016 4,264,788 $ 61.33 8.18 $ 78,412 The aggregate intrinsic values of options outstanding, vested and exercisable, and vested and expected to vest were calculated as the difference between the exercise price of the options and the fair value of the Company’s common stock. The total intrinsic value of options exercised during the years ended December 31, 2016, 2015, and 2014 was $22.2 million, $59.0 million and $12.1 million, respectively. Cash received from the exercise of options was $9.0 The weighted-average estimated fair value of stock options granted was $40.49, $54.90 and $26.58 per share of the Company’s common stock during the years ended December 31, 2016, 2015, and 2014, respectively. The total estimated fair value of options vested during the years ended December 31, 2016, 2015, and 2014 was $43.8 million, $10.7 million, and $2.0 million, respectively. Restricted Stock Units The following table summarizes activity under the Company’s Restricted Stock Units (RSUs) from the 2014 Plan and related information: RSUs Outstanding Number of Shares Weighted- Average Grant Date Fair Value Unvested — December 31, 2013 — $ — RSUs granted 31,000 53.23 Unvested — December 31, 2014 31,000 $ 53.23 RSUs granted 187,260 89.67 RSUs released (18,209 ) 54.72 RSUs cancelled (2,900 ) 84.89 Unvested — December 31, 2015 197,151 $ 87.24 RSUs granted 477,816 66.83 RSUs released (52,273 ) 82.59 RSUs cancelled (48,950 ) 78.04 Unvested — December 31, 2016 573,744 $ 71.45 The fair value of the RSUs is determined on the grant date based on the fair value of the Company’s common stock. The fair value of the RSUs is recognized as expense ratably over the vesting period of one to four years. The total fair value of the 52,273 shares vested during 2016 was approximately $4.3 million with an aggregate intrinsic value of the shares of $3.3 million. Employee Stock Purchase Plan In January 2014, the Company adopted the 2014 Employee Stock Purchase Plan (ESPP) which became effective upon the closing of our IPO in February 2014. The Company had reserved a total of 600,000 shares of common stock for issuance under the ESPP. The ESPP provides for automatic annual increases in shares available for grant, beginning on January 1, 2015 through January 1, 2024. Eligible employees may purchase common stock at 85% of the lesser of the fair market value of common stock on the offering date or the purchase date with a six-month look-back feature. ESPP purchases are settled with common stock from the ESPP’s previously authorized and available pool of shares. Stock-Based Compensation Expense Total stock-based compensation recognized was as follows (in thousands): Year Ended December 31, 2016 2015 2014 Research and development $ 29,412 $ 17,100 $ 4,116 General and administrative 18,897 7,784 1,278 Total stock-based compensation expense $ 48,309 $ 24,884 $ 5,394 As of December 31, 2016, the total unrecognized compensation expense related to unvested equity awards, net of estimated forfeitures, was $136.5 million, which the Company expects to recognize over an estimated weighted-average period of 2.78 years. In determining the estimated fair value of the stock-based awards, the Company uses the Black-Scholes option-pricing model and assumptions discussed below. Each of these inputs is subjective and generally requires significant judgment to determine. Expected Term —The Company’s expected term represents the period that the Company’s stock-based awards are expected to be outstanding and is determined using the simplified method (based on the mid-point between the vesting date and the end of the contractual term). Expected Volatility —As the Company does not have sufficient historical stock price information from the Company to meet the expected life of the stock-based awards, our approach to estimating expected volatility is to phase in our own common stock trading history and supplement the remaining historical information with a blended volatility from the trading history from the common stock of the set of comparable publicly traded biopharmaceutical companies. When selecting comparable publicly traded biopharmaceutical companies on which to base the expected stock price volatility, we selected companies with comparable characteristics to it, including enterprise value, risk profiles, position within the industry, and with historical share price information sufficient to meet the expected life of the stock-based awards. The historical volatility data was computed using the daily closing prices for the selected companies’ shares during the equivalent period of the calculated expected term of the stock-based awards. The average volatility for comparable publicly traded biopharmaceutical companies over a period equal to the expected term of the stock option grants is used to supplement the Company’s historical volatility. We will continue to apply this process until a sufficient amount of historical information regarding the volatility of our own stock price becomes available. Risk-Free Interest Rate —The risk-free interest rate is based on the U.S. Treasury zero coupon issues in effect at the time of grant for periods corresponding with the expected term of option. Expected Dividend —The Company has never paid dividends on its common stock and has no plans to pay dividends on its common stock. Therefore, the Company used an expected dividend yield of zero. The fair value of stock option awards was estimated at the date of grant using a Black-Scholes option-pricing model with the following weighted-average assumptions: Options Year Ended December 31, 2016 2015 2014 Expected term (years) 6.23 6.23 6.23 Expected volatility 65% 65% 70% Risk-free interest rate 1.5% 1.8% 1.9% Expected dividend rate 0.0% 0.0% 0.0% |
Defined Contribution Plan
Defined Contribution Plan | 12 Months Ended |
Dec. 31, 2016 | |
Defined Contribution Pension And Other Postretirement Plans Disclosure [Abstract] | |
Defined Contribution Plan | 8. Defined Contribution Plan In March 2013, the Company began to sponsor a 401(k) retirement plan, in which substantially all of its full-time employees are eligible to participate. Eligible participants may contribute a percentage of their annual compensation to this plan, subject to statutory limitations. Prior to 2015, the Company had not provided any contributions to the plan. In 2015, the Company began to make contributions to the Plan for eligible participants, and recorded $1.5 million and $0.6 million and as contribution expenses for the years ended December 31, 2016 and 2015, respectively. |
Income Taxes
Income Taxes | 12 Months Ended |
Dec. 31, 2016 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | 9. Income Taxes The components of the Company’s loss before income taxes were as follows (in thousands): Year Ended December 31, 2016 2015 2014 Domestic $ 192,287 $ 145,618 $ 59,802 Foreign 53,552 — — Total loss before income taxes $ 245,839 $ 145,618 $ 59,802 The components of the Company’s income tax provision were as follows (in thousands): Year Ended December 31, 2016 2015 2014 Federal $ — $ — $ — State — — — International 35 — — Total income tax provision $ 35 $ — $ — The Company has incurred net operating losses since inception. The Company has not reflected any benefit of such net operating loss carryforwards in the accompanying financial statements. The Company has established a full valuation allowance against its deferred tax assets due to the uncertainty surrounding the realization of such assets. The effective tax rate of our provision for income taxes differs from the federal statutory rate as follows: Year Ended December 31, 2016 2015 2014 Federal statutory income tax rate 34.0 % 34.0 % 34.0 % State income taxes, net of federal benefit 1.3 7.5 4.6 Federal tax credits 13.7 5.9 6.7 Other (0.3 ) 0.8 — Nondeductible permanent items — — (1.2 ) Stock-based compensation (1.4 ) (1.3 ) (1.4 ) Uncertain tax positions 2.0 (7.7 ) — Change in valuation allowance (41.9 ) (39.2 ) (42.7 ) Foreign rate differential (7.4 ) — — Provision for income taxes — — — The tax effect of temporary differences that give rise to significant portions of the deferred tax assets is presented below (in thousands): Year Ended December 31, 2016 2015 Deferred tax assets: Net operating loss carryforwards $ 102,960 $ 61,503 Tax credits 78,324 33,881 Stock options 16,084 6,680 Accruals and reserves 10,039 2,541 Fixed assets and intangibles 3,014 4,442 Other 1,950 432 Total deferred tax assets 212,371 109,479 Valuation allowance (212,371 ) (109,479 ) Net deferred tax assets $ — $ — As of December 31, 2016 and 2015, the Company had approximately $315.5 million and $225.5 million of federal net operating loss carryforwards available to reduce future taxable income that will begin to expire in 2030. As of December 31, 2016 and 2015, the Company had approximately $259.8 million and $255.0 million of state net operating loss carryforwards available to reduce future taxable income that will begin to expire in 2030. As of December 31, 2016 and 2015, the Company had federal research tax credit carryforwards of approximately $2.5 million and $1.7 million available to reduce future tax liabilities that will begin to expire in 2030. As of December 31, 2016 and 2015, the Company had state research credit carryforwards of approximately $6.7 million and $4.3 million available to reduce future tax liabilities that will be carried forward indefinitely. As of December 31, 2016 and 2015, the Company had federal Orphan Drug Credits of approximately $84.3 million and $35.2 million available to reduce future tax liabilities that will begin to expire in 2031. The Company’s ability to use net operating loss and tax credit carryforwards to reduce future taxable income and liabilities may be subject to annual limitations pursuant to Internal Revenue Code Sections 382 and 383 as a result of ownership changes in the past and future. As a result of ownership changes in 2012 and 2011, $3.6 million of federal net operating loss carryforwards, $3.6 million of state net operating loss carryforwards, and $0.2 million of federal tax credits are permanently limited. Deferred tax assets for net operating losses and tax credits have been reduced and a corresponding adjustment to the valuation allowance has been recorded. The valuation allowance increased by $102.9 million and $58.1 million during the year ended December 31, 2016 and 2015, respectively. The Company recorded unrecognized tax benefits for uncertainties in income taxes. A reconciliation of the Company’s unrecognized tax benefits for the years ended December 31, 2016, 2015, and 2014 is as follows (in thousands): December 31, 2016 2015 2014 Balance at beginning of year $ 24,010 $ 7,275 $ 3,725 Additions based on tax positions related to current year 6,777 15,628 3,550 Additions for tax positions of prior years 877 5,505 — Reductions for tax positions of prior years (18,159 ) (4,398 ) — Balance at end of year $ 13,505 $ 24,010 $ 7,275 The entire amount of the unrecognized tax benefits would not impact the Company’s effective tax rate if recognized. The Company has elected to include interest and penalties as a component of tax expense. During the years ended December 31, 2016 and 2015, the Company did not recognize accrued interest and penalties related to unrecognized tax benefits. The Company does not anticipate that the amount of existing unrecognized tax benefits will significantly increase or decrease during the next 12 months. It is our intention to reinvest the earnings of our non-U.S. subsidiaries in their operations. As of December 31, 2016, the Company had not made a provision for U.S. income taxes or foreign withholding taxes on approximately $0.1 million of the excess of the amount of net income for financial reporting over the tax basis of investments in foreign subsidiaries that are essentially permanent in duration. Generally, such amounts become subject to U.S. taxation upon repatriation as dividends and under certain other circumstances. If these earnings were repatriated to the U.S., the deferred tax liability associated with these temporary differences would result in a nominal amount. The Company files income tax returns in the U.S. federal, California, and other state tax jurisdictions. The federal and state income tax returns from inception to December 31, 2016 remain subject to examination. |
Commitments and Contingencies
Commitments and Contingencies | 12 Months Ended |
Dec. 31, 2016 | |
Commitments And Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 10. Commitments and Contingencies Facilities In December 2015, the Company entered into a lease agreement for office facilities in Brisbane, California, which provided for a tenant improvement allowance of up to $3.7 million. This operating lease commenced in May 2016 and expires 122 months after the commencement date. At the end of the lease term, the Company has the option to extend the lease for two additional consecutive terms of five years each. As provided in the lease agreement, monthly lease payments are subject to annual increases as defined in the lease agreement. The Company leases office space and research, testing and manufacturing laboratory space in various facilities in Novato, California, under operating agreements expiring at various dates through 2020 and include tenant improvement allowance up to $1.5 million. Certain of the leases provide for options by the Company to extend the lease for multiple five-year renewal periods and also provide for annual minimum increases in rent, usually based on a consumer price index or annual minimum increases. The Company recognizes rent expense on a straight-line basis over the noncancelable term of its operating lease. Rent expense was $3.3 million, $1.4 million, and $0.6 million during the years ended December 31, 2016, 2015 and 2014, respectively. Other Commitments The Company has various manufacturing, clinical, research, and other contracts with vendors in the conduct of the normal course of its business. Other than as noted below, contracts are terminable, with varying provisions regarding termination. If a contract with a specific vendor were to be terminated, the Company would only be obligated for the products or services that the Company had received at the time the termination became effective. As of December 31, 2016, the aggregate future minimum lease payments under the noncancelable operating lease arrangements and future payments under contractually binding manufacturing and service agreements are as follows (in thousands): Year Ending December 31, Leases Manufacturing and Services 2017 $ 3,956 $ 1,956 2018 4,534 810 2019 3,553 265 2020 2,867 62 2021 2,346 — Thereafter 11,446 — $ 28,702 $ 3,093 Contingencies While there are no material legal proceedings the Company is aware of, the Company may become party to various claims and complaints arising in the ordinary course of business. Management does not believe that any ultimate liability resulting from any of these claims will have a material adverse effect on its results of operations, financial position, or liquidity. However, management cannot give any assurance regarding the ultimate outcome of these claims, and their resolution could be material to operating results for any particular period, depending upon the level of income for the period. Guarantees and Indemnifications The Company indemnifies each of its directors and officers for certain events or occurrences, subject to certain limits, while the 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. The term of the indemnification period lasts as long as a director may be subject to any proceeding arising out of acts or omissions of such director in such capacity. The maximum amount of potential future indemnification is unlimited; however, the Company currently holds director liability insurance. This insurance allows the transfer of risk associated with the Company’s 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, it has not recognized any liabilities relating to these obligations for any period presented. |
Net Loss per Share Attributable
Net Loss per Share Attributable to Common Stockholders | 12 Months Ended |
Dec. 31, 2016 | |
Earnings Per Share [Abstract] | |
Net Loss per Share Attributable to Common Stockholders | 11. Net Loss per Share Attributable to Common Stockholders The following table sets forth the computation of the basic and diluted net loss per share attributable to common stockholders during the years ended December 31, 2016, 2015 and 2014 (in thousands, except share and per share data): Year Ended December 31, 2016 2015 2014 Numerator: Net loss $ (245,874 ) $ (145,618 ) $ (59,802 ) Accretion and dividends on convertible preferred stock — — (4,808 ) Net loss attributable to common stockholders $ (245,874 ) $ (145,618 ) $ (64,610 ) Denominator: Weighted-average shares used to compute net loss per share attributable to common stockholders, basic and diluted 39,586,908 36,782,603 28,755,758 Net loss per share attributable to common stockholders, basic and diluted $ (6.21 ) $ (3.96 ) $ (2.25 ) The following weighted-average outstanding common stock equivalents were excluded from the computation of diluted net loss per share attributable to common stockholders for the periods presented because including them would have been antidilutive: Year Ended December 31, 2016 2015 2014 Convertible preferred stock — — 1,610,834 Options to purchase common stock and RSUs 4,699,111 3,368,507 2,582,444 Convertible preferred stock warrants — — 29,051 Employee stock purchase plan 7,933 — — Common stock warrants 149,700 195,762 318,666 4,856,744 3,564,269 4,540,995 |
Accumulated Other Comprehensive
Accumulated Other Comprehensive Income (Loss) | 12 Months Ended |
Dec. 31, 2016 | |
Other Comprehensive Income Loss Net Of Tax Period Increase Decrease [Abstract] | |
Accumulated Other Comprehensive Income (Loss) | 12. Accumulated Other Comprehensive Income (Loss) Total accumulated other comprehensive income (loss) consisted of the following (in thousands) : Unrealized Gain (Loss) on Securities Available-for-Sale Foreign Currency Translation Adjustments Accumulated Other Comprehensive Income (Loss) December 31, 2013 $ 11 $ — $ 11 Current period other comprehensive loss (185 ) — (185 ) December 31, 2014 (174 ) — (174 ) Current period other comprehensive loss (694 ) — (694 ) December 31, 2015 (868 ) — (868 ) Current period other comprehensive income 451 1,322 1,773 December 31, 2016 $ (417 ) $ 1,322 $ 905 |
Quarterly Financial Data (unaud
Quarterly Financial Data (unaudited) | 12 Months Ended |
Dec. 31, 2016 | |
Quarterly Financial Data [Abstract] | |
Quarterly Financial Data (unaudited) | 13. Quarterly Financial Data (unaudited) The following table presents certain unaudited quarterly financial information. This information has been prepared on the same basis as the audited financial statements and includes all adjustments (consisting only of normal recurring adjustments) necessary to present fairly the unaudited quarterly results of operations set forth herein (in thousands, except share and per share data): 2016 March 31, June 30, September 30, December 31, Revenue $ — $ 17 $ 111 $ 5 Operating expenses 53,622 58,070 65,894 70,554 Net loss attributable to common stockholders (52,757 ) (56,923 ) (64,907 ) (71,287 ) Net loss per share attributable to common stockholders, basic and diluted $ (1.35 ) $ (1.46 ) $ (1.64 ) $ (1.75 ) 2015 March 31, June 30, September 30, December Operating expenses $ 21,502 $ 30,142 $ 39,936 $ 56,158 Net loss attributable to common stockholders (21,379 ) (29,787 ) (39,232 ) (55,220 ) Net loss per share attributable to common stockholders, basic and diluted $ (0.63 ) $ (0.83 ) $ (1.03 ) $ (1.42 ) |
Summary of Significant Accoun21
Summary of Significant Accounting Policies (Policies) | 12 Months Ended |
Dec. 31, 2016 | |
Accounting Policies [Abstract] | |
Basis of Consolidation | Basis of Consolidation The consolidated financial statements include the accounts of Ultragenyx Pharmaceutical Inc. and our wholly-owned subsidiaries. All intercompany balances and transactions have been eliminated. |
Use of Estimates | Use of Estimates The accompanying consolidated financial statements have been prepared in accordance with U.S. generally accepted accounting principles (“GAAP”). The preparation of the consolidated financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, disclosure of contingent liabilities and the reported amounts of expenses in the consolidated financial statements and the accompanying notes. On an ongoing basis, management evaluates its estimates, including those related to clinical trial accruals, fair value of assets and liabilities, 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. |
Cash and Cash Equivalents | Cash and Cash Equivalents The Company considers all highly liquid investments with original maturities of three months or less from the date of purchase to be cash equivalents. Cash equivalents consist primarily of amounts invested in money market accounts and corporate bonds. |
Investments | Investments All investments have been classified as “available-for-sale” and are carried at estimated fair value as determined based upon quoted market prices or pricing models for similar securities. Management determines the appropriate classification of its investments in debt securities at the time of purchase and reevaluates such designation as of each balance sheet date. Investments with a maturity of one year or less from the balance sheet date are reported as short-term investments and investments with a maturity of greater than one year from the balance sheet date are reported as long-term investments. Unrealized gains and losses are excluded from earnings and are reported as a component of comprehensive loss. Realized gains and losses and declines in fair value judged to be other than temporary, if any, on available-for-sale securities are included in other expense, net. The cost of securities sold is based on the specific-identification method. Interest on investments is included in interest income. |
Concentration of Credit Risk and Other Risks and Uncertainties | Concentration of Credit Risk and Other Risks and Uncertainties Financial instruments that potentially subject the Company to a concentration of credit risk consist of cash, cash equivalents, and investments. The Company’s cash, cash equivalents, and investments are held by financial institutions that management believes are of high credit quality. The Company’s investment policy limits investments to fixed income securities denominated and payable in U.S. dollars such as U.S. government obligations, money market instruments and funds, corporate bonds, and asset-backed securities and places restrictions on maturities and concentrations by type and issuer. Such deposits may, at times, exceed federally insured limits. The Company has not experienced any losses on its deposits of cash and cash equivalents and its accounts are monitored by management to mitigate risk. The Company is exposed to credit risk in the event of default by the financial institutions holding its cash and cash equivalents, corporate bond issuers and other financial instruments to the extent recorded in the balance sheets . |
Property and Equipment | Property and Equipment Property and equipment are stated at cost, less accumulated depreciation and amortization. Depreciation and amortization is computed using the straight-line method over the estimated useful lives of the respective assets. Depreciation and amortization begins at the time the asset is placed in service. Maintenance and repairs are charged to operations as incurred. Upon sale or retirement of assets, the cost and related accumulated depreciation are removed from the balance sheet and the resulting gain or loss, if any, is reflected in operations. The useful lives of the property and equipment are as follows: Research and development equipment 5 years Furniture and office equipment 5 years Computer equipment 3 years Software 3-5 years Leasehold improvements Shorter of lease term or estimated useful life |
Impairment of Long-Lived Assets | Impairment of Long-Lived Assets The Company evaluates its long-lived assets, including property and equipment, for impairment whenever events or changes in circumstances indicate that the carrying value of these assets may not be recoverable. Recoverability of these assets is measured by comparison of the carrying amount of each asset to the future undiscounted cash flows expected to result from the use of the asset and its eventual disposition. If the asset is considered to be impaired, the amount of any impairment is measured as the difference between the carrying value and the fair value of the impaired asset. The Company has not recorded impairment of any long-lived assets since inception. |
Restricted Cash | Restricted Cash Restricted cash primarily consists of money market accounts as collateral for its obligations under its facility leases of the Company’s corporate headquarters in Novato, California and for its facilities in Brisbane, California. |
Accruals of Research and Development Costs | Accruals of Research and Development Costs The Company records accruals for estimated costs of research, preclinical and clinical studies and manufacturing development. These costs are a significant component of the Company’s research and development expenses. A substantial portion of the Company’s ongoing research and development activities are conducted by third-party service providers, including contract research organizations. The Company accrues the costs incurred under its agreements with these third parties based on actual work completed in accordance with agreements established with these third parties. The Company determines the actual costs through discussions with internal personnel and external service providers as to the progress or stage of completion of the services and the agreed-upon fee to be paid for such services. The Company makes significant judgments and estimates in determining the accrual balance in each reporting period. As actual costs become known, the Company adjusts its accruals. The Company has not experienced any material deviations between accrued clinical trial expenses and actual clinical trial expenses. However, actual services performed, number of patients enrolled, and the rate of patient enrollment may vary from the Company’s estimates, resulting in adjustments to clinical trial expense in future periods. Changes in these estimates that result in material changes to the Company’s accruals could materially affect the Company’s results of operations. |
Revenue | Revenue The Company recognizes revenue when persuasive evidence of an arrangement exists, delivery has occurred, the price is fixed or determinable, and collectability is reasonably assured. Revenue is recognized once all revenue recognition criteria are met. During the year ended December 31, 2016, the Company recognized revenue from sales of rhGUS (UX003) on a “named patient” basis which are allowed in certain European countries prior to the commercial approval of the product in the territory. Due to the Company’s limited sales and collection history to date, revenue has been recognized upon receipt of payment. |
Leases | Leases The Company enters into lease agreements for its office and laboratory facilities. These leases are classified as operating leases. Rent expense is recognized on a straight-line basis over the term of the lease and, accordingly, the Company records the difference between cash rent payments and the recognition of rent expense as a deferred rent liability. Incentives granted under the Company’s facilities leases, including allowances to fund leasehold improvements, are deferred and are recognized as adjustments to rental expense on a straight-line basis over the term of the lease. |
Comprehensive Loss | Comprehensive Loss Comprehensive loss is the change in stockholders’ equity from transactions and other events and circumstances other than those resulting from investments by stockholders and distributions to stockholders. The Company’s other comprehensive loss is comprised of unrealized gains and losses on investments in available-for-sale securities and foreign currency translation adjustments. |
Research and Development | Research and Development Research and development costs are expensed as incurred and consist of salaries and benefits, stock-based compensation expense, lab supplies and facility costs, as well as fees paid to other nonemployees and entities that conduct certain research and development activities on the Company’s behalf. Amounts incurred in connection with license agreements are also included in research and development expense. Nonrefundable advance payments for goods or services to be received in the future for use in research and development activities are deferred and capitalized. The capitalized amounts are expensed as the related goods are delivered or the services are performed. |
Stock-Based Compensation | Stock-Based Compensation Stock-based awards issued to employees, including stock options and restricted stock units (RSUs), are recorded at fair value as of the grant date and recognized as expense on a straight-line basis over the employee’s requisite service period (generally the vesting period). Because noncash stock compensation expense is based on awards ultimately expected to vest, it is reduced by an estimate for future forfeitures. Forfeitures are estimated at the time of grant and revised, if necessary, in subsequent periods if actual forfeitures differ from estimates. |
Income Taxes | Income Taxes The Company uses the liability method of accounting for income taxes. Under this method, deferred tax assets and liabilities are determined based on the differences between the financial reporting and the tax bases of assets and liabilities and are measured using the enacted tax rates and laws that will be in effect when the differences are expected to reverse. The Company must then assess the likelihood that the resulting deferred tax assets will be realized. A valuation allowance is provided when it is more likely than not that some portion or all of a deferred tax asset will not be realized. Due to the Company’s lack of earnings history, the net deferred tax assets have been fully offset by a valuation allowance. The Company recognizes benefits of uncertain tax positions if it is more likely than not that such positions will be sustained upon examination based solely on their technical merits, as the largest amount of benefit that is more likely than not to be realized upon the ultimate settlement. The Company’s policy is to recognize interest and penalties related to the underpayment of income taxes as a component of income tax expense or benefit. To date, there have been no interest or penalties charged in relation to the unrecognized tax benefits. |
Foreign Currency | Foreign Currency Assets and liabilities of non-U.S. subsidiaries that operate in a local currency environment, where that local currency is the functional currency, are translated to U.S. dollars at exchange rates in effect at the balance sheet date, with the resulting translation adjustments directly recorded to a separate component of accumulated other comprehensive loss. Income and expense accounts are translated at average exchange rates for the period. Remeasurement adjustments are recorded in other income (expense), net. |
Net Loss per Share Attributable to Common Stockholders | Net Loss per Share Attributable to Common Stockholders Basic net loss per share attributable to common stockholders is calculated by dividing the net loss attributable to common stockholders by the weighted-average number of shares of common stock outstanding during the period, without consideration for common stock equivalents. Diluted net loss per share attributable to common stockholders is the same as basic net loss per share attributable to common stockholders, since the effects of potentially dilutive securities are antidilutive. In periods when we have incurred a net loss, convertible preferred stock, options and warrants to purchase common stock and convertible preferred stock warrants are considered common stock equivalents, but have been excluded from the calculation of diluted net loss per share attributable to common stockholders, as their effect is antidilutive. |
Recent Accounting Pronouncements | Recent Accounting Pronouncements In May 2014, the FASB, issued ASU 2014-09, Revenue from Contracts with Customers (Topic 606) Revenue from Contracts with Customers: Principal versus Agent Considerations Revenue from Contracts with Customers: Identifying Performance Obligations and Licensing Revenue from Contracts with Customers: Narrow-Scope Improvements and Practical Expedients to provide supplemental adoption guidance and clarification to ASU 2014-09 Technical Corrections and Improvements to Topic 606, Revenue from Contracts with Customers In February 2016, the FASB issued ASU No. 2016-02, Leases (Topic 842), which requires an entity that is a lessee to record a right of use asset and a corresponding lease liability on the balance sheet for all leases with terms longer than 12 months. This guidance also requires disclosures about the amount, timing, and uncertainty of cash flows arising from leases. This guidance is effective for annual reporting periods beginning after December 15, 2018, and interim periods within those annual periods, using a modified retrospective approach, and early adoption is permitted. The Company is evaluating the effect that this guidance will have on its Consolidated Financial Statements and related disclosures. In March 2016, the FASB issued ASU 2016-09, Compensation — Stock Compensation (Topic 718): Improvements to Employee Share-Based Payment Accounting accounting for employee share-based payments, including income tax consequences, application of award forfeitures to expense, classification on the statement of cash flows, and classification of awards as either equity or liabilities. This guidance is effective for annual reporting periods beginning after December 15, 2016, and interim periods within those annual periods. In October 2016, the FASB issued ASU 2016-16, Income Taxes - Intra-Entity Transfers of Assets Other Than Inventory |
Summary of Significant Accoun22
Summary of Significant Accounting Policies (Tables) | 12 Months Ended |
Dec. 31, 2016 | |
Accounting Policies [Abstract] | |
Summary of Useful Lives of Property and Equipment | The useful lives of the property and equipment are as follows: Research and development equipment 5 years Furniture and office equipment 5 years Computer equipment 3 years Software 3-5 years Leasehold improvements Shorter of lease term or estimated useful life |
Fair Value Measurements (Tables
Fair Value Measurements (Tables) | 12 Months Ended |
Dec. 31, 2016 | |
Fair Value Disclosures [Abstract] | |
Summary of Financial Assets Measured on Recurring Basis | The following table sets forth the fair value of the Company’s financial assets and liabilities remeasured on a recurring basis based on the three-tier fair value hierarchy (in thousands): December 31, 2016 Level 1 Level 2 Level 3 Total Financial Assets: Money market funds $ 123,536 $ — $ — $ 123,536 Corporate bonds — 207,726 — 207,726 Commercial paper — 11,970 — 11,970 Asset-backed securities — 27,713 — 27,713 U.S. Government Treasury and agency securities — 111,931 — 111,931 Total financial assets $ 123,536 $ 359,340 $ — $ 482,876 December 31, 2015 Level 1 Level 2 Level 3 Total Financial Assets: Money market funds $ 53,254 $ — $ — $ 53,254 Corporate bonds — 370,445 — 370,445 Commercial paper — 13,887 — 13,887 Asset-backed securities — 29,302 — 29,302 U.S. Government Treasury and agency securities — 47,452 — 47,452 Total financial assets $ 53,254 $ 461,086 $ — $ 514,340 |
Balance Sheet Components (Table
Balance Sheet Components (Tables) | 12 Months Ended |
Dec. 31, 2016 | |
Investments Debt And Equity Securities [Abstract] | |
Summary of Fair Values of Cash Equivalents, Short-Term Investments and Long-Term Investments Classified as Available-for-Sale Securities | The fair values of cash equivalents, short-term investments, and long-term investments classified as available-for-sale securities, consisted of the following (in thousands): December 31, 2016 Gross Unrealized Amortized Cost Gains Losses Estimated Fair Value Money market funds $ 123,536 $ — $ — $ 123,536 Corporate bonds 207,909 14 (197 ) 207,726 Commercial paper 11,970 — — 11,970 Asset-backed securities 27,712 3 (2 ) 27,713 U.S. Government Treasury and agency securities 112,166 10 (245 ) 111,931 Total $ 483,293 $ 27 $ (444 ) $ 482,876 December 31, 2015 Gross Unrealized Amortized Cost Gains Losses Estimated Fair Value Money market funds $ 53,254 $ — $ — $ 53,254 Corporate bonds 371,098 11 (664 ) 370,445 Commercial paper 13,887 — — 13,887 Asset-backed securities 29,356 — (54 ) 29,302 U.S. Government Treasury and agency securities 47,613 — (161 ) 47,452 Total $ 515,208 $ 11 $ (879 ) $ 514,340 |
Summary of Property and Equipment | Property and equipment, net consists of the following (in thousands): December 31, 2016 2015 Research and development equipment $ 2,154 $ 1,302 Furniture and office equipment 2,595 944 Computer equipment 2,013 628 Software 3,931 1,622 Leasehold improvements 12,486 3,466 Construction-in-progress 37 2,276 Property and equipment, gross 23,216 10,238 Less accumulated depreciation (6,161 ) (2,865 ) Property and equipment, net $ 17,055 $ 7,373 |
Accrued Liabilities | Accrued liabilities consist of the following (in thousands): December 31, 2016 2015 Research and clinical study expenses $ 18,593 $ 9,764 Payroll and related expenses 17,226 9,423 Repayment liability under collaboration agreement 13,650 — Other 5,085 5,597 Total accrued liabilities $ 54,554 $ 24,784 |
Stock-Based Awards (Tables)
Stock-Based Awards (Tables) | 12 Months Ended |
Dec. 31, 2016 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Summary of Activity Under Stock Option Plans Including 2011 Plan and 2014 Plan | Stock Option Activity The following table summarizes activity under the Company’s stock option plans, including the 2011 Plan and the 2014 Plan and related information: Options Outstanding Number of Options Weighted- Average Exercise Weighted-Average Remaining Contractual Term Aggregate Intrinsic Value (In Outstanding — December 31, 2013 2,228,883 $ 3.41 8.91 $ 19,468 Options granted 932,555 41.12 Options exercised (305,090 ) 2.14 Options cancelled (116,873 ) 8.92 Outstanding — December 31, 2014 2,739,475 $ 16.15 8.43 $ 79,840 Options granted 2,013,350 90.60 Options exercised (795,825 ) 8.65 Options cancelled (130,037 ) 31.49 Outstanding — December 31, 2015 3,826,963 $ 56.36 8.58 $ 217,386 Options granted 1,435,995 67.00 Options exercised (425,922 ) 21.21 Options cancelled (407,564 ) 70.87 Outstanding — December 31, 2016 4,429,472 $ 61.85 8.22 $ 79,135 Vested and exercisable — December 31, 2016 1,620,443 $ 44.96 7.24 $ 55,537 Vested and expected to vest — December 31, 2016 4,264,788 $ 61.33 8.18 $ 78,412 |
Summary of Activity Under Restricted Stock Units (RSUs) from 2014 Plan | The following table summarizes activity under the Company’s Restricted Stock Units (RSUs) from the 2014 Plan and related information: RSUs Outstanding Number of Shares Weighted- Average Grant Date Fair Value Unvested — December 31, 2013 — $ — RSUs granted 31,000 53.23 Unvested — December 31, 2014 31,000 $ 53.23 RSUs granted 187,260 89.67 RSUs released (18,209 ) 54.72 RSUs cancelled (2,900 ) 84.89 Unvested — December 31, 2015 197,151 $ 87.24 RSUs granted 477,816 66.83 RSUs released (52,273 ) 82.59 RSUs cancelled (48,950 ) 78.04 Unvested — December 31, 2016 573,744 $ 71.45 |
Summary of Stock-Based Compensation Recognized | Total stock-based compensation recognized was as follows (in thousands): Year Ended December 31, 2016 2015 2014 Research and development $ 29,412 $ 17,100 $ 4,116 General and administrative 18,897 7,784 1,278 Total stock-based compensation expense $ 48,309 $ 24,884 $ 5,394 |
Fair Value of Stock Option Awards Estimated Using Black-Scholes Option-Pricing Model | The fair value of stock option awards was estimated at the date of grant using a Black-Scholes option-pricing model with the following weighted-average assumptions: Options Year Ended December 31, 2016 2015 2014 Expected term (years) 6.23 6.23 6.23 Expected volatility 65% 65% 70% Risk-free interest rate 1.5% 1.8% 1.9% Expected dividend rate 0.0% 0.0% 0.0% |
Income Taxes (Tables)
Income Taxes (Tables) | 12 Months Ended |
Dec. 31, 2016 | |
Income Tax Disclosure [Abstract] | |
Components of Company's Loss Before Income Taxes | The components of the Company’s loss before income taxes were as follows (in thousands): Year Ended December 31, 2016 2015 2014 Domestic $ 192,287 $ 145,618 $ 59,802 Foreign 53,552 — — Total loss before income taxes $ 245,839 $ 145,618 $ 59,802 |
Components of Company's Income Tax Provision | The components of the Company’s income tax provision were as follows (in thousands): Year Ended December 31, 2016 2015 2014 Federal $ — $ — $ — State — — — International 35 — — Total income tax provision $ 35 $ — $ — |
Effective Tax Rate of Provision for Income Taxes from Federal Statutory Rate | The effective tax rate of our provision for income taxes differs from the federal statutory rate as follows: Year Ended December 31, 2016 2015 2014 Federal statutory income tax rate 34.0 % 34.0 % 34.0 % State income taxes, net of federal benefit 1.3 7.5 4.6 Federal tax credits 13.7 5.9 6.7 Other (0.3 ) 0.8 — Nondeductible permanent items — — (1.2 ) Stock-based compensation (1.4 ) (1.3 ) (1.4 ) Uncertain tax positions 2.0 (7.7 ) — Change in valuation allowance (41.9 ) (39.2 ) (42.7 ) Foreign rate differential (7.4 ) — — Provision for income taxes — — — |
Schedule of Tax Effect of Temporary Differences to Significant Portions of Deferred Tax Assets | The tax effect of temporary differences that give rise to significant portions of the deferred tax assets is presented below (in thousands): Year Ended December 31, 2016 2015 Deferred tax assets: Net operating loss carryforwards $ 102,960 $ 61,503 Tax credits 78,324 33,881 Stock options 16,084 6,680 Accruals and reserves 10,039 2,541 Fixed assets and intangibles 3,014 4,442 Other 1,950 432 Total deferred tax assets 212,371 109,479 Valuation allowance (212,371 ) (109,479 ) Net deferred tax assets $ — $ — |
Unrecognized Tax Benefits | A reconciliation of the Company’s unrecognized tax benefits for the years ended December 31, 2016, 2015, and 2014 is as follows (in thousands): December 31, 2016 2015 2014 Balance at beginning of year $ 24,010 $ 7,275 $ 3,725 Additions based on tax positions related to current year 6,777 15,628 3,550 Additions for tax positions of prior years 877 5,505 — Reductions for tax positions of prior years (18,159 ) (4,398 ) — Balance at end of year $ 13,505 $ 24,010 $ 7,275 |
Commitments and Contingencies (
Commitments and Contingencies (Tables) | 12 Months Ended |
Dec. 31, 2016 | |
Commitments And Contingencies Disclosure [Abstract] | |
Summary of Future Minimum Lease Payments under Non-Cancellable Operating Leases Arrangements and Future Payments under Contractually Binding Manufacturing and Service Agreements | As of December 31, 2016, the aggregate future minimum lease payments under the noncancelable operating lease arrangements and future payments under contractually binding manufacturing and service agreements are as follows (in thousands): Year Ending December 31, Leases Manufacturing and Services 2017 $ 3,956 $ 1,956 2018 4,534 810 2019 3,553 265 2020 2,867 62 2021 2,346 — Thereafter 11,446 — $ 28,702 $ 3,093 |
Net Loss per Share Attributab28
Net Loss per Share Attributable to Common Stockholders (Tables) | 12 Months Ended |
Dec. 31, 2016 | |
Earnings Per Share [Abstract] | |
Computation of Basic and Diluted Net Loss per Share | The following table sets forth the computation of the basic and diluted net loss per share attributable to common stockholders during the years ended December 31, 2016, 2015 and 2014 (in thousands, except share and per share data): Year Ended December 31, 2016 2015 2014 Numerator: Net loss $ (245,874 ) $ (145,618 ) $ (59,802 ) Accretion and dividends on convertible preferred stock — — (4,808 ) Net loss attributable to common stockholders $ (245,874 ) $ (145,618 ) $ (64,610 ) Denominator: Weighted-average shares used to compute net loss per share attributable to common stockholders, basic and diluted 39,586,908 36,782,603 28,755,758 Net loss per share attributable to common stockholders, basic and diluted $ (6.21 ) $ (3.96 ) $ (2.25 ) |
Outstanding Shares of Common Stock Equivalents Excluded from Computation of Diluted Net Loss per Share | The following weighted-average outstanding common stock equivalents were excluded from the computation of diluted net loss per share attributable to common stockholders for the periods presented because including them would have been antidilutive: Year Ended December 31, 2016 2015 2014 Convertible preferred stock — — 1,610,834 Options to purchase common stock and RSUs 4,699,111 3,368,507 2,582,444 Convertible preferred stock warrants — — 29,051 Employee stock purchase plan 7,933 — — Common stock warrants 149,700 195,762 318,666 4,856,744 3,564,269 4,540,995 |
Accumulated Other Comprehensi29
Accumulated Other Comprehensive Income (Loss) (Tables) | 12 Months Ended |
Dec. 31, 2016 | |
Other Comprehensive Income Loss Net Of Tax Period Increase Decrease [Abstract] | |
Schedule of Total Accumulated Other Comprehensive Income (Loss) | Total accumulated other comprehensive income (loss) consisted of the following (in thousands) : Unrealized Gain (Loss) on Securities Available-for-Sale Foreign Currency Translation Adjustments Accumulated Other Comprehensive Income (Loss) December 31, 2013 $ 11 $ — $ 11 Current period other comprehensive loss (185 ) — (185 ) December 31, 2014 (174 ) — (174 ) Current period other comprehensive loss (694 ) — (694 ) December 31, 2015 (868 ) — (868 ) Current period other comprehensive income 451 1,322 1,773 December 31, 2016 $ (417 ) $ 1,322 $ 905 |
Quarterly Financial Data (una30
Quarterly Financial Data (unaudited) (Tables) | 12 Months Ended |
Dec. 31, 2016 | |
Quarterly Financial Data [Abstract] | |
Summary of Unaudited Quarterly Financial Information | The following table presents certain unaudited quarterly financial information. This information has been prepared on the same basis as the audited financial statements and includes all adjustments (consisting only of normal recurring adjustments) necessary to present fairly the unaudited quarterly results of operations set forth herein (in thousands, except share and per share data): 2016 March 31, June 30, September 30, December 31, Revenue $ — $ 17 $ 111 $ 5 Operating expenses 53,622 58,070 65,894 70,554 Net loss attributable to common stockholders (52,757 ) (56,923 ) (64,907 ) (71,287 ) Net loss per share attributable to common stockholders, basic and diluted $ (1.35 ) $ (1.46 ) $ (1.64 ) $ (1.75 ) 2015 March 31, June 30, September 30, December Operating expenses $ 21,502 $ 30,142 $ 39,936 $ 56,158 Net loss attributable to common stockholders (21,379 ) (29,787 ) (39,232 ) (55,220 ) Net loss per share attributable to common stockholders, basic and diluted $ (0.63 ) $ (0.83 ) $ (1.03 ) $ (1.42 ) |
Organization and Basis of Pre31
Organization and Basis of Presentation - Additional Information (Details) | 12 Months Ended |
Dec. 31, 2016Segment | |
United States of America | |
Organization And Nature Of Business [Line Items] | |
Number of reportable segments | 1 |
Summary of Significant Accoun32
Summary of Significant Accounting Policies - Summary of Useful Lives of Property and Equipment (Details) | 12 Months Ended |
Dec. 31, 2016 | |
Research and Development Equipment | |
Property Plant And Equipment [Line Items] | |
Property plant and equipment useful life | 5 years |
Furniture and Office Equipment | |
Property Plant And Equipment [Line Items] | |
Property plant and equipment useful life | 5 years |
Computer Equipment | |
Property Plant And Equipment [Line Items] | |
Property plant and equipment useful life | 3 years |
Software | Minimum | |
Property Plant And Equipment [Line Items] | |
Property plant and equipment useful life | 3 years |
Software | Maximum | |
Property Plant And Equipment [Line Items] | |
Property plant and equipment useful life | 5 years |
Leasehold Improvements | |
Property Plant And Equipment [Line Items] | |
Property plant and equipment useful life | Shorter of lease term or estimated useful life |
Summary of Significant Accoun33
Summary of Significant Accounting Policies - Additional Information (Details) | 12 Months Ended |
Dec. 31, 2016USD ($) | |
Accounting Policies [Abstract] | |
Unrecognized tax benefits related to interest or penalties | $ 0 |
Fair Value Measurements - Summa
Fair Value Measurements - Summary of Financial Assets and Liabilities Measured on Recurring Basis (Details) - USD ($) $ in Thousands | Dec. 31, 2016 | Dec. 31, 2015 |
Financial Assets: | ||
Fair value | $ 482,876 | $ 514,340 |
Money Market Funds | ||
Financial Assets: | ||
Fair value | 123,536 | 53,254 |
Asset Backed Securities | ||
Financial Assets: | ||
Fair value | 27,713 | 29,302 |
Corporate Bonds | ||
Financial Assets: | ||
Fair value | 207,726 | 370,445 |
Commercial Paper | ||
Financial Assets: | ||
Fair value | 11,970 | 13,887 |
US Government Treasury and Agencies Securities | ||
Financial Assets: | ||
Fair value | 111,931 | 47,452 |
Level 1 | ||
Financial Assets: | ||
Fair value | 123,536 | 53,254 |
Level 1 | Money Market Funds | ||
Financial Assets: | ||
Fair value | 123,536 | 53,254 |
Level 2 | ||
Financial Assets: | ||
Fair value | 359,340 | 461,086 |
Level 2 | Asset Backed Securities | ||
Financial Assets: | ||
Fair value | 27,713 | 29,302 |
Level 2 | Corporate Bonds | ||
Financial Assets: | ||
Fair value | 207,726 | 370,445 |
Level 2 | Commercial Paper | ||
Financial Assets: | ||
Fair value | 11,970 | 13,887 |
Level 2 | US Government Treasury and Agencies Securities | ||
Financial Assets: | ||
Fair value | $ 111,931 | $ 47,452 |
Balance Sheet Components - Summ
Balance Sheet Components - Summary of Fair Values of Cash Equivalents, Short-Term Investments and Long-Term Investments Classified as Available-for-Sale Securities (Details) - USD ($) $ in Thousands | Dec. 31, 2016 | Dec. 31, 2015 |
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | $ 483,293 | $ 515,208 |
Gross Unrealized Gains | 27 | 11 |
Gross Unrealized Losses | (444) | (879) |
Estimated Fair Value | 482,876 | 514,340 |
Money Market Funds | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 123,536 | 53,254 |
Estimated Fair Value | 123,536 | 53,254 |
Commercial Paper | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 11,970 | 13,887 |
Estimated Fair Value | 11,970 | 13,887 |
Corporate Bonds | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 207,909 | 371,098 |
Gross Unrealized Gains | 14 | 11 |
Gross Unrealized Losses | (197) | (664) |
Estimated Fair Value | 207,726 | 370,445 |
Asset Backed Securities | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 27,712 | 29,356 |
Gross Unrealized Gains | 3 | |
Gross Unrealized Losses | (2) | (54) |
Estimated Fair Value | 27,713 | 29,302 |
U.S. Government Treasury and Agency Securities | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 112,166 | 47,613 |
Gross Unrealized Gains | 10 | |
Gross Unrealized Losses | (245) | (161) |
Estimated Fair Value | $ 111,931 | $ 47,452 |
Balance Sheet Components - Addi
Balance Sheet Components - Additional Information (Details) - USD ($) | 12 Months Ended | ||
Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | |
Schedule Of Available For Sale Securities [Line Items] | |||
Significant realized gains or losses on available-for-sale securities | $ 0 | ||
Depreciation and amortization | $ 3,424,000 | $ 1,384,000 | $ 684,000 |
Maximum | |||
Schedule Of Available For Sale Securities [Line Items] | |||
Available-for-sale securities remaining contractual maturities | 2 years |
Balance Sheet Components - Su37
Balance Sheet Components - Summary of Property and Equipment (Details) - USD ($) $ in Thousands | Dec. 31, 2016 | Dec. 31, 2015 |
Property Plant And Equipment [Line Items] | ||
Property and equipment, gross | $ 23,216 | $ 10,238 |
Less accumulated depreciation | (6,161) | (2,865) |
Property and equipment, net | 17,055 | 7,373 |
Research and Development Equipment | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, gross | 2,154 | 1,302 |
Furniture and Office Equipment | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, gross | 2,595 | 944 |
Computer Equipment | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, gross | 2,013 | 628 |
Software | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, gross | 3,931 | 1,622 |
Leasehold Improvements | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, gross | 12,486 | 3,466 |
Construction-in-progress | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, gross | $ 37 | $ 2,276 |
Balance Sheet Components - Accr
Balance Sheet Components - Accrued Liabilities (Details) - USD ($) $ in Thousands | Dec. 31, 2016 | Dec. 31, 2015 |
Payables And Accruals [Abstract] | ||
Research and clinical study expenses | $ 18,593 | $ 9,764 |
Payroll and related expenses | 17,226 | 9,423 |
Repayment liability under collaboration agreement | 13,650 | |
Other | 5,085 | 5,597 |
Total accrued liabilities | $ 54,554 | $ 24,784 |
License and Research Agreemen39
License and Research Agreements - Additional Information (Details) $ / shares in Units, $ in Thousands, ¥ in Millions | 1 Months Ended | 12 Months Ended | ||||||||
Oct. 31, 2016USD ($)shares | Jul. 31, 2016USD ($)$ / sharesshares | Jun. 30, 2016USD ($) | Oct. 31, 2015USD ($) | Jun. 30, 2013USD ($) | Dec. 31, 2016USD ($)shares | Dec. 31, 2015USD ($)shares | Dec. 31, 2014USD ($)shares | Jan. 31, 2017USD ($) | Dec. 31, 2016JPY (¥)shares | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||
Research and development | $ 183,204 | $ 114,737 | $ 45,967 | |||||||
Common stock purchased | shares | 41,240,230 | 38,882,394 | 41,240,230 | |||||||
Common stock value | $ 121,711 | |||||||||
Common stock issued upon option exercised in period, shares | shares | 425,922 | 795,825 | 305,090 | |||||||
Common stock issued upon option exercised in period, value | $ 9,096 | $ 7,355 | $ 736 | |||||||
Liability under collaboration arrangement | $ 13,650 | |||||||||
Common Stock | ||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||
Common stock value | $ 7 | |||||||||
Common stock issued upon option exercised in period, shares | shares | 438,811 | 957,587 | 331,605 | |||||||
Common stock issued upon option exercised in period, value | $ 1 | |||||||||
Nobelpharma License Agreement | Maximum | ||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||
Aggregate future payments | $ 1,700 | ¥ 200 | ||||||||
Saint Louis University License Agreement | ||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||
Future contingent milestone payments | 100 | |||||||||
H I B M Research Group License Agreement | Development and Approval Milestones | ||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||
Future contingent milestone payments | $ 300 | |||||||||
H I B M Research Group License Agreement | Maximum | ||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||
Payments for royalty on net sales | 1.00% | |||||||||
St. Jude Children's Research Hospital License Agreement | Maximum | ||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||
Payments for royalty on net sales | 1.00% | |||||||||
Baylor Research Institute License Agreement | ||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||
Payment of option exercise fee | $ 800 | |||||||||
Baylor Research Institute License Agreement | Development Milestones | ||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||
Future contingent milestone payments | $ 10,500 | |||||||||
Baylor Research Institute License Agreement | Sales Milestones | ||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||
Future contingent milestone payments | 7,500 | |||||||||
Kyowa Hakko Kirin Collaboration | License Agreement | ||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||
Reduction in research and development cost | 26,800 | 10,800 | ||||||||
License agreement receivable | $ 8,600 | $ 3,800 | ||||||||
Kyowa Hakko Kirin Collaboration | License Agreement | Profit Share Territory | ||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||
Remaining profit or loss share percentage on commercializing products | 50.00% | |||||||||
Tiered double-digit revenue share percentage entitled to receive | 20.00% | |||||||||
Kyowa Hakko Kirin Collaboration | Maximum | License Agreement | European Territory | ||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||
Percentage of royalty on net sales entitled to receive | 10.00% | |||||||||
Arcturus Research Collaboration and License Agreement | ||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||
Research and development | $ 10,000 | |||||||||
Takeda License and Collaboration and Purchase Agreements | ||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||
Estimated fair value of license recorded as research and development expense | $ 700 | |||||||||
Research collaboration agreement, period | 5 years | |||||||||
Common stock purchased | shares | 374,590 | |||||||||
Common stock value | $ 40,000 | |||||||||
Common closing stock price per share | $ / shares | $ 72.95 | |||||||||
Common stock shares issued premium amount | $ 12,700 | |||||||||
Option to purchase additional common stock, value | $ 25,000 | |||||||||
Volume weighted average price, period | 30 days | |||||||||
Second option to purchase additional common stock, value | $ 10,000 | |||||||||
Common stock purchase agreement, terms | Takeda was subject to a 180-day lock-up provision as a result of the initial purchase of common stock, and a 90-day lock-up provision as a result of its purchase under the first put option, is subject to a five-year standstill (subject to customary exceptions or release), and has registration rights for purchases of common stock. | |||||||||
Total arrangement consideration received | 14,300 | |||||||||
Initial development plan period | Approximately one and a half years | |||||||||
Liability under collaboration arrangement | $ 14,300 | |||||||||
Takeda License and Collaboration and Purchase Agreements | Subsequent Event | ||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||
Second option to purchase additional common stock, value | $ 10,000 | |||||||||
Takeda License and Collaboration and Purchase Agreements | Put Option | ||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||
Estimated fair value of future options | $ 900 | |||||||||
Common stock issued upon option exercised in period, shares | shares | 352,530 | |||||||||
Common stock issued upon option exercised in period, value | $ 25,000 | |||||||||
Takeda License and Collaboration and Purchase Agreements | Put Option | Monte Carlo Simulation | ||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||
Equity volatility percentage | 70.00% | |||||||||
Takeda License and Collaboration and Purchase Agreements | Common Stock | ||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||
Fair market value of shares issued | $ 27,300 | |||||||||
Takeda License and Collaboration and Purchase Agreements | Maximum | ||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||
Estimated approximate cost for expected work performed | 15,500 | |||||||||
Takeda License and Collaboration and Purchase Agreements | Maximum | Development Milestones | ||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||
Future contingent milestone payments | $ 7,500 | |||||||||
Takeda License and Collaboration and Purchase Agreements | Maximum | Regulatory Milestones | ||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||
Future contingent milestone payments | 75,000 | |||||||||
Takeda License and Collaboration and Purchase Agreements | Maximum | Commercial Milestones | ||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||
Future contingent milestone payments | $ 150,000 | |||||||||
Takeda License and Collaboration and Purchase Agreements | Minimum | ||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||
Estimated approximate cost for expected work performed | $ 10,000 |
Equity - Additional Information
Equity - Additional Information (Details) - USD ($) | 1 Months Ended | 2 Months Ended | 12 Months Ended | ||||
Jul. 31, 2016 | Feb. 15, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | Jan. 30, 2014 | Jun. 30, 2011 | |
Stockholders Equity [Line Items] | |||||||
Net proceeds from sale of common stock | $ 461,136,000 | $ 188,581,000 | |||||
Fair value of warrants | $ 6,700,000 | $ 200,000 | |||||
Revaluation of convertible preferred stock warrant liability | $ 0 | $ 0 | $ 3,324,000 | ||||
June 2010 Common Stock Warrant [Member] | |||||||
Stockholders Equity [Line Items] | |||||||
Common stock warrants outstanding | 83,167 | 83,167 | |||||
Common stock warrants, issued date | 2010-06 | 2010-06 | |||||
Common stock warrants, contractual term | 10 years | 10 years | |||||
Common stock warrants, exercise price | $ 3.006 | $ 3.006 | |||||
June 2011 Common Stock Warrant [Member] | |||||||
Stockholders Equity [Line Items] | |||||||
Common stock warrants outstanding | 66,533 | 66,533 | |||||
Common stock warrants, issued date | 2011-06 | 2011-06 | |||||
Common stock warrants, contractual term | 10 years | 10 years | |||||
Common stock warrants, exercise price | $ 3.006 | $ 3.006 | |||||
Subsequent Event | |||||||
Stockholders Equity [Line Items] | |||||||
Common stock shares sold | 658,532 | ||||||
Net proceeds from sale of common stock | $ 48,400,000 | ||||||
ATM Sales Agreement | |||||||
Stockholders Equity [Line Items] | |||||||
Common stock shares sold | 1,159,415 | ||||||
Net proceeds from sale of common stock | $ 79,500,000 | ||||||
Maximum | ATM Sales Agreement | |||||||
Stockholders Equity [Line Items] | |||||||
Option to sell common stock for cash | $ 150,000,000 |
Stock-Based Awards - Additional
Stock-Based Awards - Additional Information (Details) - USD ($) $ / shares in Units, $ in Millions | 12 Months Ended | |||
Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | Feb. 05, 2014 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Total intrinsic value of options exercised | $ 22.2 | $ 59 | $ 12.1 | |
Cash received from exercise of options | $ 9 | $ 6.9 | $ 0.7 | |
Weighted-average estimated fair value of stock option | $ 40.49 | $ 54.90 | $ 26.58 | |
Total estimated fair value of options vested | $ 43.8 | $ 10.7 | $ 2 | |
Total unrecognized compensation cost | $ 136.5 | |||
Weighted-average period to recognize cost | 2 years 9 months 11 days | |||
Restricted Stock Units | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Shares vested during the period | 52,273 | 18,209 | ||
Total fair value of shares vested | $ 4.3 | |||
Aggregate intrinsic value | $ 3.3 | |||
Maximum | Restricted Stock Units | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Stock based awards vesting period | 4 years | |||
Minimum | Restricted Stock Units | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Stock based awards vesting period | 1 year | |||
2014 Incentive Plan | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Share of common stock authorized for future issuance | 2,250,000 | |||
Automatic increases in shares available for grant effective date | Jan. 1, 2015 | |||
Shares available for grant, ending date | Jan. 1, 2024 | |||
2014 Incentive Plan | Employee Stock Option | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Minimum percentage of voting rights of all classes of stock | 10.00% | |||
Percentage of statutory stock awards | 110.00% | |||
2014 Incentive Plan | Maximum | Employee Stock Option | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Award expiration period | 10 years | |||
2011 Equity Incentive Plan | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Share of common stock authorized for future issuance | 655,038 | |||
2011 Equity Incentive Plan | Restricted Stock Units | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Option vesting rights, percentage | 25.00% | |||
Stock based awards vesting period | 4 years | |||
2011 Equity Incentive Plan | First Anniversary | Employee Stock Option | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Option vesting rights, percentage | 25.00% | |||
2011 Equity Incentive Plan | Shares Vesting Monthly After First Anniversary | Employee Stock Option | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Option vesting rights, percentage | 2.08% | |||
2011 Equity Incentive Plan and 2014 Incentive Plan | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Share of common stock authorized for future issuance | 7,478,107 | |||
2014 Employee Stock Purchase Plan | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Automatic increases in shares available for grant effective date | Jan. 1, 2015 | |||
Shares available for grant, ending date | Jan. 1, 2024 | |||
Percentage of statutory stock awards | 85.00% | |||
Share of common stock available for future issuance | 600,000 |
Stock-Based Awards - Summary of
Stock-Based Awards - Summary of Activity Under Stock Option Plans Including 2011 Plan and 2014 Plan (Details) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | |||
Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | Dec. 31, 2013 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | ||||
Number of Options, Outstanding, Beginning Balance | 3,826,963 | 2,739,475 | 2,228,883 | |
Number of Options, Options granted | 1,435,995 | 2,013,350 | 932,555 | |
Number of Options, Options exercised | (425,922) | (795,825) | (305,090) | |
Number of Options, Options cancelled | (407,564) | (130,037) | (116,873) | |
Number of Options, Outstanding, Ending Balance | 4,429,472 | 3,826,963 | 2,739,475 | 2,228,883 |
Number of Options, Vested and exercisable — December 31, 2016 | 1,620,443 | |||
Number of Options, Vested and expected to vest — December 31, 2016 | 4,264,788 | |||
Weighted Average Exercise Price, Outstanding, Beginning Balance | $ 56.36 | $ 16.15 | $ 3.41 | |
Weighted Average Exercise Price, Options granted | 67 | 90.60 | 41.12 | |
Weighted Average Exercise Price, Options exercised | 21.21 | 8.65 | 2.14 | |
Weighted Average Exercise Price, Options cancelled | 70.87 | 31.49 | 8.92 | |
Weighted Average Exercise Price, Outstanding, Ending Balance | 61.85 | $ 56.36 | $ 16.15 | $ 3.41 |
Weighted Average Exercise Price, Vested and exercisable — December 31, 2016 | 44.96 | |||
Weighted Average Exercise Price, Vested and expected to vest — December 31, 2016 | $ 61.33 | |||
Weighted Average Remaining Contractual Term (Years), Outstanding | 8 years 2 months 19 days | 8 years 6 months 29 days | 8 years 5 months 5 days | 8 years 10 months 28 days |
Weighted Average Remaining Contractual Term (Years), Vested and exercisable — December 31, 2016 | 7 years 2 months 27 days | |||
Weighted Average Remaining Contractual Term (Years), Vested and expected to vest — December 31, 2016 | 8 years 2 months 5 days | |||
Aggregate Intrinsic Value, Outstanding | $ 79,135 | $ 217,386 | $ 79,840 | $ 19,468 |
Aggregate Intrinsic Value, Vested and exercisable — December 31, 2016 | 55,537 | |||
Aggregate Intrinsic Value, Vested and expected to vest — December 31, 2016 | $ 78,412 |
Stock-Based Awards - Summary 43
Stock-Based Awards - Summary of Activity Under Restricted Stock Units (RSUs) from 2014 Plan (Details) - Restricted Stock Units - $ / shares | 12 Months Ended | ||
Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||
RSUs Outstanding, Number of Shares, Unvested, Beginning Balance | 197,151 | 31,000 | |
Number of Shares, RSUs granted | 477,816 | 187,260 | 31,000 |
Number of Shares, RSUs released | (52,273) | (18,209) | |
Number of Shares, RSUs cancelled | (48,950) | (2,900) | |
RSUs Outstanding, Number of Shares, Unvested, Ending Balance | 573,744 | 197,151 | 31,000 |
RSUs Outstanding, Weighted-Average Grant Date Fair Value, Unvested, Beginning Balance | $ 87.24 | $ 53.23 | |
Weighted-Average Grant Date Fair Value, RSUs granted | 66.83 | 89.67 | $ 53.23 |
Weighted-Average Grant Date Fair Value, RSUs released | 82.59 | 54.72 | |
Weighted-Average Grant Date Fair Value, RSUs cancelled | 78.04 | 84.89 | |
RSUs Outstanding, Weighted-Average Grant Date Fair Value, Unvested, Ending Balance | $ 71.45 | $ 87.24 | $ 53.23 |
Stock-Based Awards - Summary 44
Stock-Based Awards - Summary of Stock-Based Compensation Recognized (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | |
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | |||
Stock-based compensation expense | $ 48,309 | $ 24,884 | $ 5,394 |
Research and development | |||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | |||
Stock-based compensation expense | 29,412 | 17,100 | 4,116 |
General and administrative | |||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | |||
Stock-based compensation expense | $ 18,897 | $ 7,784 | $ 1,278 |
Stock-Based Awards - Fair Value
Stock-Based Awards - Fair Value of Stock Option Awards Estimated Using Black-Scholes Option-Pricing Model (Details) - Employee Stock Option | 12 Months Ended | ||
Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||
Expected term (years) | 6 years 2 months 23 days | 6 years 2 months 23 days | 6 years 2 months 23 days |
Expected volatility | 65.00% | 65.00% | 70.00% |
Risk-free interest rate | 1.50% | 1.80% | 1.90% |
Expected dividend rate | 0.00% | 0.00% | 0.00% |
Defined Contribution Plan - Add
Defined Contribution Plan - Additional Information (Details) - USD ($) $ in Millions | 12 Months Ended | |
Dec. 31, 2016 | Dec. 31, 2015 | |
Defined Contribution Pension And Other Postretirement Plans Disclosure [Abstract] | ||
Contribution expenses | $ 1.5 | $ 0.6 |
Income Taxes - Components of Co
Income Taxes - Components of Company's Loss Before Income Taxes (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | |
Income Tax Disclosure [Abstract] | |||
Domestic | $ 192,287 | $ 145,618 | $ 59,802 |
Foreign | 53,552 | ||
Total loss before income taxes | $ 245,839 | $ 145,618 | $ 59,802 |
Income Taxes - Components of 48
Income Taxes - Components of Company's Income Tax Provision (Details) $ in Thousands | 12 Months Ended |
Dec. 31, 2016USD ($) | |
Income Tax Disclosure [Abstract] | |
International | $ 35 |
Total income tax provision | $ 35 |
Income Taxes - Effective Tax Ra
Income Taxes - Effective Tax Rate of Provision for Income Taxes from Federal Statutory Rate (Details) | 12 Months Ended | ||
Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | |
Effective Income Tax Rate Continuing Operations Tax Rate Reconciliation [Abstract] | |||
Federal statutory income tax rate | 34.00% | 34.00% | 34.00% |
State income taxes, net of federal benefit | 1.30% | 7.50% | 4.60% |
Federal tax credits | 13.70% | 5.90% | 6.70% |
Other | (0.30%) | 0.80% | |
Nondeductible permanent items | (1.20%) | ||
Stock-based compensation | (1.40%) | (1.30%) | (1.40%) |
Uncertain tax positions | 2.00% | (7.70%) | |
Change in valuation allowance | (41.90%) | (39.20%) | (42.70%) |
Foreign rate differential | (7.40%) |
Income Taxes - Schedule of Tax
Income Taxes - Schedule of Tax Effect of Temporary Differences to Significant Portions of Deferred Tax Assets (Details) - USD ($) $ in Thousands | Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2012 | Dec. 31, 2011 |
Deferred tax assets: | ||||
Net operating loss carryforwards | $ 102,960 | $ 61,503 | $ 200 | $ 200 |
Tax credits | 78,324 | 33,881 | ||
Stock options | 16,084 | 6,680 | ||
Accruals and reserves | 10,039 | 2,541 | ||
Fixed assets and intangibles | 3,014 | 4,442 | ||
Other | 1,950 | 432 | ||
Total deferred tax assets | 212,371 | 109,479 | ||
Valuation allowance | (212,371) | (109,479) | ||
Net deferred tax assets | $ 0 | $ 0 |
Income Taxes - Additional Infor
Income Taxes - Additional Information (Details) - USD ($) | 12 Months Ended | |||
Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2012 | Dec. 31, 2011 | |
Tax Credit Carryforward [Line Items] | ||||
Deferred tax assets net operating loss carryforwards | $ 102,960,000 | $ 61,503,000 | $ 200,000 | $ 200,000 |
Valuation allowance increased | 102,900,000 | 58,100,000 | ||
Unrecognized tax benefits, income tax penalties and interest accrued | 0 | 0 | ||
Undistributed earnings of foreign subsidiaries | 100,000 | |||
Federal | ||||
Tax Credit Carryforward [Line Items] | ||||
Deferred tax assets operating loss carryforwards subject to expiration | $ 315,500,000 | 225,500,000 | ||
Operating loss carryforwards expiration year | 2,030 | |||
Tax credit carryforwards | $ 2,500,000 | 1,700,000 | ||
Tax credit carryforwards expiration year | 2,030 | |||
Deferred tax assets net operating loss carryforwards | 3,600,000 | 3,600,000 | ||
Federal | Orphan Drug Credits [Member] | ||||
Tax Credit Carryforward [Line Items] | ||||
Deferred tax assets operating loss carryforwards subject to expiration | $ 84,300,000 | 35,200,000 | ||
Operating loss carryforwards expiration year | 2,031 | |||
State | ||||
Tax Credit Carryforward [Line Items] | ||||
Deferred tax assets operating loss carryforwards subject to expiration | $ 259,800,000 | 255,000,000 | ||
Operating loss carryforwards expiration year | 2,030 | |||
Tax credit carryforwards | $ 6,700,000 | $ 4,300,000 | ||
Deferred tax assets net operating loss carryforwards | $ 3,600,000 | $ 3,600,000 |
Income Taxes - Unrecognized Tax
Income Taxes - Unrecognized Tax Benefits (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | |
Income Tax Disclosure [Abstract] | |||
Balance at beginning of year | $ 24,010 | $ 7,275 | $ 3,725 |
Additions based on tax positions related to current year | 6,777 | 15,628 | 3,550 |
Additions for tax positions of prior years | 877 | 5,505 | |
Reductions for tax positions of prior years | (18,159) | (4,398) | |
Balance at end of year | $ 13,505 | $ 24,010 | $ 7,275 |
Commitments and Contingencies -
Commitments and Contingencies - Additional Information (Details) $ in Thousands | 1 Months Ended | 12 Months Ended | ||
Dec. 31, 2015USD ($)Option | Dec. 31, 2016USD ($) | Dec. 31, 2015USD ($) | Dec. 31, 2014USD ($) | |
Disclosure Commitments And Contingencies Additional Information Details [Line Items] | ||||
Tenant improvement allowance | $ 3,467 | |||
Operating lease commencement date | 2016-05 | |||
Rent expense | $ 3,300 | $ 1,400 | $ 600 | |
Brisbane Office Facility | ||||
Disclosure Commitments And Contingencies Additional Information Details [Line Items] | ||||
Tenant improvement allowance | $ 3,700 | |||
Operating lease expiration period | 122 months | |||
Number of options to extend lease | Option | 2 | |||
Lease renewal term | 5 years | |||
Novato Office Facility | ||||
Disclosure Commitments And Contingencies Additional Information Details [Line Items] | ||||
Tenant improvement allowance | $ 1,500 | |||
Lease renewal term | 5 years | |||
Operating lease expiration year | 2,020 |
Commitments and Contingencies54
Commitments and Contingencies - Summary of Future Minimum Lease Payments under Non-Cancellable Operating Leases Arrangements and Future Payments under Contractually Binding Manufacturing and Service Agreements (Details) $ in Thousands | Dec. 31, 2016USD ($) |
Commitments And Contingencies Disclosure [Abstract] | |
Leases, 2017 | $ 3,956 |
Leases, 2018 | 4,534 |
Leases, 2019 | 3,553 |
Leases, 2020 | 2,867 |
Leases, 2021 | 2,346 |
Leases, Thereafter | 11,446 |
Leases, Total | 28,702 |
Manufacturing and Services, 2017 | 1,956 |
Manufacturing and Services, 2018 | 810 |
Manufacturing and Services, 2019 | 265 |
Manufacturing and Services, 2020 | 62 |
Manufacturing and Services, Total | $ 3,093 |
Net Loss per Share Attributab55
Net Loss per Share Attributable to Common Stockholders - Computation of Basic and Diluted Net Loss per Share (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2016 | Sep. 30, 2016 | Jun. 30, 2016 | Mar. 31, 2016 | Dec. 31, 2015 | Sep. 30, 2015 | Jun. 30, 2015 | Mar. 31, 2015 | Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | |
Numerator: | |||||||||||
Net loss | $ (245,874) | $ (145,618) | $ (59,802) | ||||||||
Accretion and dividends on convertible preferred stock | (4,808) | ||||||||||
Net loss attributable to common stockholders | $ (71,287) | $ (64,907) | $ (56,923) | $ (52,757) | $ (55,220) | $ (39,232) | $ (29,787) | $ (21,379) | $ (245,874) | $ (145,618) | $ (64,610) |
Denominator: | |||||||||||
Weighted-average shares used to compute net loss per share attributable to common stockholders, basic and diluted | 39,586,908 | 36,782,603 | 28,755,758 | ||||||||
Net loss per share attributable to common stockholders, basic and diluted | $ (1.75) | $ (1.64) | $ (1.46) | $ (1.35) | $ (1.42) | $ (1.03) | $ (0.83) | $ (0.63) | $ (6.21) | $ (3.96) | $ (2.25) |
Net Loss per Share Attributab56
Net Loss per Share Attributable to Common Stockholders - Outstanding Shares of Common Stock Equivalents Excluded from Computation of Diluted Net Loss per Share (Details) - shares | 12 Months Ended | ||
Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | |
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | |||
Common stock equivalents excluded from computation of diluted net loss per share | 4,856,744 | 3,564,269 | 4,540,995 |
Convertible Preferred Stock | |||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | |||
Common stock equivalents excluded from computation of diluted net loss per share | 1,610,834 | ||
Options to Purchase Common Stock and RSUs | |||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | |||
Common stock equivalents excluded from computation of diluted net loss per share | 4,699,111 | 3,368,507 | 2,582,444 |
Convertible Preferred Stock Warrants | |||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | |||
Common stock equivalents excluded from computation of diluted net loss per share | 29,051 | ||
Common Stock Warrants | |||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | |||
Common stock equivalents excluded from computation of diluted net loss per share | 149,700 | 195,762 | 318,666 |
Employee Stock Purchase Plan | |||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | |||
Common stock equivalents excluded from computation of diluted net loss per share | 7,933 |
Accumulated Other Comprehensi57
Accumulated Other Comprehensive Income (Loss) - Schedule of Total Accumulated Other Comprehensive Income (Loss) (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | |
Accumulated Other Comprehensive Income (Loss) [Line Items] | |||
Beginning balance | $ 531,090 | $ 184,945 | $ (74,821) |
Current period other comprehensive income (loss) | 1,773 | (694) | (185) |
Ending balance | 473,974 | 531,090 | 184,945 |
Unrealized Gain (Loss) on Securities Available-for-Sale | |||
Accumulated Other Comprehensive Income (Loss) [Line Items] | |||
Beginning balance | (868) | (174) | 11 |
Current period other comprehensive income (loss) | 451 | (694) | (185) |
Ending balance | (417) | (868) | (174) |
Foreign Currency Translation Adjustments | |||
Accumulated Other Comprehensive Income (Loss) [Line Items] | |||
Current period other comprehensive income (loss) | 1,322 | ||
Ending balance | 1,322 | ||
Accumulated Other Comprehensive Income (Loss) | |||
Accumulated Other Comprehensive Income (Loss) [Line Items] | |||
Beginning balance | (868) | (174) | 11 |
Current period other comprehensive income (loss) | 1,773 | (694) | (185) |
Ending balance | $ 905 | $ (868) | $ (174) |
Quarterly Financial Data (una58
Quarterly Financial Data (unaudited) - Summary of Unaudited Quarterly Financial Information (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2016 | Sep. 30, 2016 | Jun. 30, 2016 | Mar. 31, 2016 | Dec. 31, 2015 | Sep. 30, 2015 | Jun. 30, 2015 | Mar. 31, 2015 | Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | |
Selected Quarterly Financial Information [Abstract] | |||||||||||
Revenue | $ 5 | $ 111 | $ 17 | $ 133 | |||||||
Operating expenses | 70,554 | 65,894 | 58,070 | $ 53,622 | $ 56,158 | $ 39,936 | $ 30,142 | $ 21,502 | 248,140 | $ 147,738 | $ 56,778 |
Net loss attributable to common stockholders | $ (71,287) | $ (64,907) | $ (56,923) | $ (52,757) | $ (55,220) | $ (39,232) | $ (29,787) | $ (21,379) | $ (245,874) | $ (145,618) | $ (64,610) |
Net loss per share attributable to common stockholders, basic and diluted | $ (1.75) | $ (1.64) | $ (1.46) | $ (1.35) | $ (1.42) | $ (1.03) | $ (0.83) | $ (0.63) | $ (6.21) | $ (3.96) | $ (2.25) |