Document and Entity Information
Document and Entity Information - shares | 6 Months Ended | |
Jun. 30, 2019 | Jul. 26, 2019 | |
Document And Entity Information [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Jun. 30, 2019 | |
Document Fiscal Year Focus | 2019 | |
Document Fiscal Period Focus | Q2 | |
Trading Symbol | RARE | |
Entity Registrant Name | Ultragenyx Pharmaceutical Inc. | |
Entity Central Index Key | 0001515673 | |
Entity Current Reporting Status | Yes | |
Current Fiscal Year End Date | --12-31 | |
Entity Filer Category | Large Accelerated Filer | |
Entity Small Business | false | |
Entity Emerging Growth Company | false | |
Entity Common Stock, Shares Outstanding | 57,691,957 | |
Entity Shell Company | false | |
Entity File Number | 001-36276 | |
Entity Tax Identification Number | 27-2546083 | |
Entity Address, Address Line One | 60 Leveroni Court | |
Entity Address, City or Town | Novato | |
Entity Address, State or Province | CA | |
Entity Address, Postal Zip Code | 94949 | |
City Area Code | 415 | |
Local Phone Number | 483-8800 | |
Entity Interactive Data Current | Yes | |
Title of 12(b) Security | Common Stock, $0.001 par value | |
Security Exchange Name | NASDAQ | |
Entity Incorporation, State or Country Code | DE | |
Document Quarterly Report | true | |
Document Transition Report | false |
CONDENSED CONSOLIDATED BALANCE
CONDENSED CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands | Jun. 30, 2019 | Dec. 31, 2018 |
Current assets: | ||
Cash and cash equivalents | $ 131,337 | $ 113,432 |
Short-term investments | 486,949 | 346,274 |
Accounts receivable | 20,901 | 12,740 |
Inventory | 13,632 | 7,065 |
Prepaid expenses and other current assets | 46,612 | 42,858 |
Total current assets | 699,431 | 522,369 |
Property and equipment, net | 25,317 | 20,046 |
Investment in Arcturus equity securities | 24,167 | |
Intangible assets, net | 129,069 | 129,223 |
Goodwill | 44,406 | 44,406 |
Right-of-use assets | 33,356 | |
Other assets | 3,181 | 3,514 |
Total assets | 958,927 | 719,558 |
Current liabilities: | ||
Accounts payable | 8,743 | 12,275 |
Accrued liabilities | 59,264 | 62,450 |
Short-term lease liabilities | 6,999 | |
Total current liabilities | 75,006 | 74,725 |
Deferred tax liabilities | 31,166 | 31,166 |
Long-term lease liabilities | 32,968 | |
Other liabilities | 4,759 | |
Total liabilities | 139,140 | 110,650 |
Stockholders’ equity: | ||
Preferred stock — 25,000,000 shares authorized; nil outstanding as of June 30, 2019 and December 31, 2018 | ||
Common stock — 250,000,000 shares authorized; 57,665,375 and 50,860,588 shares issued and outstanding as of June 30, 2019 and December 31, 2018, respectively | 58 | 51 |
Additional paid-in capital | 2,045,685 | 1,639,773 |
Accumulated other comprehensive income (loss) | 255 | (633) |
Accumulated deficit | (1,226,211) | (1,030,283) |
Total stockholders’ equity | 819,787 | 608,908 |
Total liabilities and stockholders’ equity | $ 958,927 | $ 719,558 |
CONDENSED CONSOLIDATED BALANC_2
CONDENSED CONSOLIDATED BALANCE SHEETS (Parenthetical) - shares | Jun. 30, 2019 | Dec. 31, 2018 |
Statement Of Financial Position [Abstract] | ||
Preferred stock, shares authorized | 25,000,000 | 25,000,000 |
Preferred stock, shares outstanding | 0 | 0 |
Common stock, shares authorized | 250,000,000 | 250,000,000 |
Common stock, shares issued | 57,665,375 | 50,860,588 |
Common stock, shares outstanding | 57,665,375 | 50,860,588 |
CONDENSED CONSOLIDATED STATEMEN
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Revenues: | ||||
Total revenues | $ 24,149 | $ 12,794 | $ 42,321 | $ 23,471 |
Operating expenses: | ||||
Cost of sales | 766 | 141 | 1,218 | 366 |
Research and development | 96,045 | 76,835 | 174,150 | 152,339 |
Selling, general and administrative | 39,812 | 30,718 | 78,641 | 62,153 |
Total operating expenses | 136,623 | 107,694 | 254,009 | 214,858 |
Loss from operations | (112,474) | (94,900) | (211,688) | (191,387) |
Interest income | 4,063 | 2,448 | 7,149 | 4,185 |
Gain from sale of priority review vouchers | 40,322 | 170,322 | ||
Change in fair value of investment in Arcturus equity securities | 9,828 | 9,828 | ||
Other expense | (376) | (496) | (788) | (5,454) |
Loss before income taxes | (98,959) | (52,626) | (195,499) | (22,334) |
Provision for income taxes | (213) | (102) | (429) | (141) |
Net loss | $ (99,172) | $ (52,728) | $ (195,928) | $ (22,475) |
Net loss per share, basic and diluted | $ (1.72) | $ (1.06) | $ (3.54) | $ (0.46) |
Shares used in computing net loss per share, basic and diluted | 57,519,308 | 49,819,528 | 55,376,336 | 49,046,838 |
Collaboration and License | ||||
Revenues: | ||||
Total revenues | $ 19,247 | $ 10,519 | $ 33,485 | $ 19,881 |
Product Sales | ||||
Revenues: | ||||
Total revenues | $ 4,902 | $ 2,275 | $ 8,836 | $ 3,590 |
CONDENSED CONSOLIDATED STATEM_2
CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Statement Of Income And Comprehensive Income [Abstract] | ||||
Net loss | $ (99,172) | $ (52,728) | $ (195,928) | $ (22,475) |
Other comprehensive income (loss): | ||||
Foreign currency translation adjustments | 26 | 225 | 155 | (64) |
Transfer of cumulative translation adjustment for the substantial liquidation of foreign subsidiaries | 5,272 | |||
Unrealized gain (loss) on available-for-sale securities | 377 | 67 | 733 | (182) |
Other comprehensive income: | 403 | 292 | 888 | 5,026 |
Total comprehensive loss | $ (98,769) | $ (52,436) | $ (195,040) | $ (17,449) |
CONDENSED CONSOLIDATED STATEM_3
CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS' EQUITY (Unaudited) - USD ($) $ in Thousands | Total | Common Stock | Additional Paid-in Capital | Accumulated Other Comprehensive Income (Loss) | Accumulated Deficit |
Beginning balance at Dec. 31, 2017 | $ 383,454 | $ 44 | $ 1,221,762 | $ (5,680) | $ (832,672) |
Beginning balance, shares at Dec. 31, 2017 | 44,167,071 | ||||
Issuance of common stock in connection with underwritten public offering, net of issuance costs | 270,969 | $ 5 | 270,964 | ||
Issuance of common stock in connection with underwritten public offering, net of issuance costs, shares | 5,043,860 | ||||
Issuance of common stock in connection with at-the-market offering, net of issuance costs | 11,808 | $ 1 | 11,807 | ||
Issuance of common stock in connection with at-the-market offering, net of issuance costs, shares | 240,417 | ||||
Employee stock-based compensation | 38,360 | 38,360 | |||
Issuance of common stock under equity plan awards, net of tax | 16,829 | 16,829 | |||
Issuance of common stock under equity plan awards, net of tax, shares | 664,708 | ||||
Other comprehensive income | 5,026 | 5,026 | |||
Net loss | (22,475) | (22,475) | |||
Ending balance at Jun. 30, 2018 | 703,971 | $ 50 | 1,559,722 | (654) | (855,147) |
Ending balance, shares at Jun. 30, 2018 | 50,116,056 | ||||
Beginning balance at Mar. 31, 2018 | 723,657 | $ 50 | 1,526,972 | (946) | (802,419) |
Beginning balance, shares at Mar. 31, 2018 | 49,665,203 | ||||
Employee stock-based compensation | 19,563 | 19,563 | |||
Issuance of common stock under equity plan awards, net of tax | 13,187 | 13,187 | |||
Issuance of common stock under equity plan awards, net of tax, shares | 450,853 | ||||
Other comprehensive income | 292 | 292 | |||
Net loss | (52,728) | (52,728) | |||
Ending balance at Jun. 30, 2018 | 703,971 | $ 50 | 1,559,722 | (654) | (855,147) |
Ending balance, shares at Jun. 30, 2018 | 50,116,056 | ||||
Beginning balance at Dec. 31, 2018 | $ 608,908 | $ 51 | 1,639,773 | (633) | (1,030,283) |
Beginning balance, shares at Dec. 31, 2018 | 50,860,588 | 50,860,588 | |||
Issuance of common stock in connection with underwritten public offering, net of issuance costs | $ 330,415 | $ 6 | 330,409 | ||
Issuance of common stock in connection with underwritten public offering, net of issuance costs, shares | 5,833,333 | ||||
Issuance of common stock in connection with at-the-market offering, net of issuance costs | 24,828 | 24,828 | |||
Issuance of common stock in connection with at-the-market offering, net of issuance costs, shares | 468,685 | ||||
Employee stock-based compensation | 42,960 | 42,960 | |||
Issuance of common stock under equity plan awards, net of tax | 7,716 | $ 1 | 7,715 | ||
Issuance of common stock under equity plan awards, net of tax, shares | 502,769 | ||||
Other comprehensive income | 888 | 888 | |||
Net loss | (195,928) | (195,928) | |||
Ending balance at Jun. 30, 2019 | $ 819,787 | $ 58 | 2,045,685 | 255 | (1,226,211) |
Ending balance, shares at Jun. 30, 2019 | 57,665,375 | 57,665,375 | |||
Beginning balance at Mar. 31, 2019 | $ 886,729 | $ 57 | 2,013,859 | (148) | (1,127,039) |
Beginning balance, shares at Mar. 31, 2019 | 57,303,888 | ||||
Issuance of common stock in connection with at-the-market offering, net of issuance costs | 5,523 | 5,523 | |||
Issuance of common stock in connection with at-the-market offering, net of issuance costs, shares | 88,978 | ||||
Employee stock-based compensation | 22,490 | 22,490 | |||
Issuance of common stock under equity plan awards, net of tax | 3,814 | $ 1 | 3,813 | ||
Issuance of common stock under equity plan awards, net of tax, shares | 272,509 | ||||
Other comprehensive income | 403 | 403 | |||
Net loss | (99,172) | (99,172) | |||
Ending balance at Jun. 30, 2019 | $ 819,787 | $ 58 | $ 2,045,685 | $ 255 | $ (1,226,211) |
Ending balance, shares at Jun. 30, 2019 | 57,665,375 | 57,665,375 |
CONDENSED CONSOLIDATED STATEM_4
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS (Unaudited) - USD ($) $ in Thousands | 6 Months Ended | |
Jun. 30, 2019 | Jun. 30, 2018 | |
Operating activities: | ||
Net loss | $ (195,928) | $ (22,475) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Stock-based compensation | 42,428 | 38,360 |
Amortization of discount on investment securities, net | (2,960) | (913) |
Depreciation and amortization | 4,225 | 12,240 |
Foreign currency remeasurement loss | 567 | 5,846 |
Change in fair value of investment in Arcturus equity securities | (9,828) | |
Gain from sale of priority review vouchers | (170,322) | |
Other | (132) | (145) |
Changes in operating assets and liabilities: | ||
Accounts receivable | (8,161) | (12,253) |
Inventory | (6,040) | (2,585) |
Prepaid expenses and other current assets | (4,265) | (7,544) |
Right-of-use assets | (17,200) | |
Other assets | 1,010 | (102) |
Accounts payable | (3,644) | 1,376 |
Lease liabilities | 18,522 | |
Accrued liabilities and other liabilities | (3,432) | (7,039) |
Net cash used in operating activities | (184,838) | (165,556) |
Investing activities: | ||
Purchase of property and equipment | (8,299) | (1,852) |
Purchase of investments | (461,088) | (408,683) |
Purchase of investment in Arcturus equity securities | (14,339) | |
Proceeds from the sale of investments | 22,600 | 4,954 |
Proceeds from maturities of investments | 301,507 | 112,162 |
Proceeds from sale of priority review vouchers | 170,322 | |
Net cash used in investing activities | (159,619) | (123,097) |
Financing activities: | ||
Proceeds from the issuance of common stock in connection with underwritten public offerings, net | 330,415 | 270,969 |
Proceeds from the issuance of common stock in connection with at-the-market offering, net | 24,828 | 11,789 |
Proceeds from the issuance of common stock from equity plan awards, net of tax | 7,716 | 16,829 |
Net cash provided by financing activities | 362,959 | 299,587 |
Effect of exchange rate changes on cash | (30) | (647) |
Net increase in cash, cash equivalents and restricted cash | 18,472 | 10,287 |
Cash, cash equivalents and restricted cash at beginning of period | 115,525 | 103,041 |
Cash, cash equivalents and restricted cash at end of period | 133,997 | $ 113,328 |
Supplemental disclosures of non-cash information: | ||
Acquired lease liabilities arising from obtaining right-of-use assets | $ 21,515 |
Organization
Organization | 6 Months Ended |
Jun. 30, 2019 | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | |
Organization | 1. Organization Ultragenyx Pharmaceutical Inc. (the Company) is a biopharmaceutical company 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 serious rare and ultra-rare genetic diseases. The Company has two approved therapies. Crysvita ® In addition to the approved treatments for XLH and MPS VII, the Company has four ongoing clinical development programs. Crysvita is being studied for the treatment of tumor induced osteomalacia (TIO), a rare disease that impairs bone mineralization. UX007 is being studied 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. The Company has two gene therapy pipeline candidates: DTX301 is an adeno-associated virus 8 (AAV8) gene therapy product candidate in development for the treatment of patients with ornithine transcarbamylase (OTC) deficiency, the most common urea cycle disorder; and DTX401 is an AAV8 gene therapy product candidate for the treatment of patients with glycogen storage disease type Ia (GSDIa). The Company operates as one reportable segment. The Company has sustained operating losses and expects such annual losses to continue over the next several years. The Company’s ultimate success depends on the outcome of its research and development and commercialization activities, for which it expects to incur additional losses in the future. Management recognizes the need to raise additional capital to fully implement its business plan. Through June 30, 2019, 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 | 6 Months Ended |
Jun. 30, 2019 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies Basis of Presentation The accompanying unaudited condensed consolidated financial statements include the accounts of the Company and its wholly-owned subsidiaries and have been prepared in accordance with U.S. generally accepted accounting principles (GAAP) for interim financial information and in accordance with the instructions to Form 10-Q and Rule 10-01 of Regulation S-X. Accordingly, they do not include all of the information and footnotes required by GAAP for complete financial statements. The unaudited interim consolidated financial statements have been prepared on the same basis as the annual financial statements. In the opinion of management, the accompanying unaudited condensed consolidated financial statements reflect all adjustments (consisting only of normal recurring adjustments) considered necessary for a fair presentation. These financial statements should be read in conjunction with the audited financial statements and notes thereto for the preceding fiscal year contained in the Company’s Annual Report on Form 10-K filed on February 19, 2019 with the United States Securities and Exchange Commission (SEC). The results of operations for the three and six months ended June 30, 2019 are not necessarily indicative of the results to be expected for the year ending December 31, 2019. The condensed consolidated balance sheet as of December 31, 2018 has been derived from audited financial statements at that date, but does not include all of the information required by GAAP for complete financial statements. Use of Estimates The accompanying consolidated financial statements have been prepared in accordance with 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, Cash Equivalents and Restricted Cash Restricted cash primarily consists of money market accounts as collateral for the Company’s obligations under its facility leases. The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the consolidated balance sheets that sum to the total of the same such amounts shown in the consolidated statement of cash flows (in thousands): June 30, 2019 2018 Cash and cash equivalents $ 131,337 $ 110,854 Restricted cash included in prepaid expenses and other current assets 161 652 Restricted cash included in other assets 2,499 1,822 Total cash, cash equivalents, and restricted cash shown in the statements of cash flows $ 133,997 $ 113,328 Investment in Equity Securities In June 2019, the Company entered into an amendment to the Research Collaboration and License Agreement and an Equity Purchase Agreement with Arcturus Therapeutics Holdings Inc. (“Arcturus”). Pursuant to the Equity Purchase Agreement, the Company purchased 2,400,000 shares of common stock, or approximately 18.2% of Arcturus’s outstanding shares of common stock as of the closing date of the transaction and received an option to purchase an additional 600,000 shares of common stock. The investment was accounted for using the equity method of accounting as it was determined that the Company has significant influence over, but does not control the significant activities of Arcturus. The Company elected to apply the fair value option to account for the equity investment in Arcturus. The decision to elect the fair value option is irrevocable and is determined on an instrument by instrument basis. The option to purchase additional stock was accounted for at fair value using Black-Scholes option pricing method. The changes in fair value of the equity investment and option to purchase additional stock are included in the Condensed Consolidated Statements of Operations. See “Note 6. License and Research Agreements” for additional details on the Arcturus transaction. Revenue Recognition Collaboration and license revenue The Company has certain license and collaboration agreements that are within the scope of Accounting Standards Codification (ASC) 808, Collaborative Agreements provides guidance on the presentation and disclosure of collaborative arrangements. Generally, the classification of the transactions under the collaborative arrangements is determined based on the nature of contractual terms of the arrangement, along with the nature of the operations of the participants. The Company records its share of collaboration revenue, net of transfer pricing related to net sales in the period in which such sales occur, if the Company is considered as an agent in the arrangement. The Company is considered an agent when the collaboration partner controls the product before transfer to the customers and has the ability to direct the use of and obtain substantially all of the remaining benefits from the product. Funding received related to research and development services and commercialization costs is generally classified as a reduction of research and development expenses and selling, general and administrative expenses, respectively, in the consolidated statement of operations, because the provision of such services for collaborative partners are not considered to be part of the Company’s ongoing major or central operations. The also receives royalty revenues under certain of the Company’s license or collaboration agreements in exchange for license of intellectual property. If the Company does not have any future performance obligations for these license or collaboration agreements, royalty revenue is recorded as the underlying sales occur. In to record collaboration revenue, the Company utilizes certain information from its , including revenue from the sale of the product, associated reserves on revenue, and costs incurred for development and sales activities. For the periods covered in the financial statements presented, there have been no material changes to prior period estimates of revenues and expenses. The terms of the Company’s collaboration agreements may contain multiple performance obligations, which may include licenses and research and development activities. The Company evaluates these agreements under ASC 606, Revenue from Contracts with Customers (ASC 606), to determine the distinct performance obligations. The Company analogizes to ASC 606 for the accounting for distinct performance obligations for which there is a customer relationship. Prior to recognizing revenue, the Company makes estimates of the transaction price, including variable consideration that is subject to a constraint. Amounts of variable consideration are included in the transaction price to the extent that it is probable that a significant reversal in the amount of cumulative revenue recognized will not occur and when the uncertainty associated with the variable consideration is subsequently resolved. Total consideration may include nonrefundable upfront license fees, payments for research and development activities, reimbursement of certain third-party costs, payments based upon the achievement of specified milestones, and royalty payments based on product sales derived from the collaboration. If there are multiple distinct performance obligations, the Company allocates the transaction price to each distinct performance obligation based on its relative standalone selling price. The standalone selling price is generally determined based on the prices charged to customers or using expected cost plus margin. The Company estimates the efforts needed to complete the performance obligation and recognizes revenue by measuring the progress towards complete satisfaction of the performance obligation using an input measure. Product sales The Company sells its approved products through a limited number of distributors. Under ASC 606, revenue from product sales is recognized at the point in time when the delivery is made and when title and risk of loss transfers to these distributors. The Company also recognizes revenue from sales of certain products on a “named patient” basis, which are allowed in certain countries prior to the commercial approval of the product. Prior to recognizing revenue, the Company makes estimates of the transaction price, including any variable consideration that is subject to a constraint. Amounts of variable consideration are included in the transaction price to the extent that it is probable that a significant reversal in the amount of cumulative revenue recognized will not occur and when the uncertainty associated with the variable consideration is subsequently resolved. Product sales are recorded net of estimated government-mandated rebates and chargebacks, estimated product returns, and other deductions. Provisions for returns and other adjustments are provided for in the period the related revenue is recorded, as estimated by management. Leases The Company adopted Accounting Standards Update (ASU) No. 2016-02, Leases (Topic 842) Leases (Topic 840) The Company determines if an arrangement includes a lease at inception. Right-of-use assets and lease liabilities are recognized based on the present value of the future minimum lease payments over the lease term at the commencement date. The right-of-use asset includes any lease payments made and excludes lease incentives. Incremental borrowing rate is used in determining the present value of future payments. The Company applies a portfolio approach to the property leases to apply an incremental borrowing rate to leases with similar lease terms. The lease terms may include options to extend or terminate the lease. The Company recognizes the options to extend the lease as part of the right-of-use assets and lease liabilities only if it is reasonably certain that the option would be exercised. Lease expense for minimum lease payments is recognized on a straight-line basis over the non-cancelable lease term. Prior period amounts have not been adjusted and continue to be reported in accordance with the Company’s historical accounting under previous lease guidance, Topic 840. As a result of the adoption of the new guidance, the Company recorded a right-of-use asset of $16.2 million, a short-term lease liability of $4.5 million, and a long-term lease liability of $17.0 million and no cumulative effect adjustment was made to the retained earnings as of the adoption date. In addition, as of the adoption date, the Company derecognized a net deferred rent obligation of $5.2 million. See Note 11 for further disclosure. Recently Issued Accounting Pronouncements In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments — Credit Losses, (Topic 326): Measurement of Credit Losses on Financial Instruments, |
Financial Instruments
Financial Instruments | 6 Months Ended |
Jun. 30, 2019 | |
Fair Value Disclosures [Abstract] | |
Financial Instruments | 3. Financial Instruments Financial assets and liabilities are recorded at fair value. The carrying amount of certain financial instruments, including cash and cash equivalents, accounts receivable, 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 following tables set forth the fair value of the Company’s financial assets remeasured on a recurring basis based on the three-tier fair value hierarchy (in thousands): June 30, 2019 Level 1 Level 2 Level 3 Total Money market funds $ 92,414 $ — $ — $ 92,414 Time deposits — 10,000 — 10,000 Corporate bonds — 58,742 — 58,742 Commercial paper — 132,947 — 132,947 Asset-backed securities — 40,901 — 40,901 U.S. Government Treasury and agency securities 112,200 157,135 — 269,335 Investment in Arcturus equity securities 22,656 — 1,511 24,167 Total $ 227,270 $ 399,725 $ 1,511 $ 628,506 December 31, 2018 Level 1 Level 2 Level 3 Total Money market funds $ 72,999 $ — $ — $ 72,999 Time deposits — 10,000 — 10,000 Corporate bonds — 179,926 — 179,926 Commercial paper — 50,198 — 50,198 Asset-backed securities — 22,587 — 22,587 U.S. Government Treasury and agency securities — 99,034 — 99,034 Total $ 72,999 $ 361,745 $ — $ 434,744 The Company determines the fair value of the Arcturus common stock by using the quoted market price on June 30, 2019, which is a Level 1 fair value measurement. The change in fair value of the Arcturus common stock for the three and six months ended June 30, 2019 was $8.8 million, which was recognized in the Condensed Consolidated Statements of Operations. T he The change in fair value of the option to purchase additional Arcturus common stock for the three and six months ended June 30, 2019 was $1.0 million, which was recognized in the Condensed Consolidated Statements of Operations. See “Note 6. License and Research Agreement” for additional details on the Arcturus transaction. |
Balance Sheet Components
Balance Sheet Components | 6 Months Ended |
Jun. 30, 2019 | |
Investments Debt And Equity Securities [Abstract] | |
Balance Sheet Components | 4. Balance Sheet Components Cash Equivalents and Investments The fair values of cash equivalents and short-term investments classified as available-for-sale securities consisted of the following (in thousands): June 30, 2019 Gross Unrealized Amortized Cost Gains Losses Estimated Fair Value Money market funds $ 92,414 $ — $ — $ 92,414 Time deposits 10,000 — — 10,000 Corporate bonds 58,692 50 — 58,742 Commercial paper 132,947 — — 132,947 Asset-backed securities 40,834 67 — 40,901 U.S. Government Treasury and agency securities 269,023 312 — 269,335 Total $ 603,910 $ 429 $ — $ 604,339 December 31, 2018 Gross Unrealized Amortized Cost Gains Losses Estimated Fair Value Money market funds $ 72,999 $ — $ — $ 72,999 Time deposits 10,000 — — 10,000 Corporate bonds 180,167 — (241 ) 179,926 Commercial paper 50,198 — — 50,198 Asset-backed securities 22,597 — (10 ) 22,587 U.S. Government Treasury and agency securities 99,087 2 (55 ) 99,034 Total $ 435,048 $ 2 $ (306 ) $ 434,744 At June 30, 2019, the remaining contractual maturities of available-for-sale securities were less than one year. There have been no material realized gains or losses on available-for-sale securities for the periods presented. Inventory Inventory consists of the following (in thousands): June 30, December 31, 2019 2018 Work-in-progress $ 11,064 $ 5,384 Finished goods 2,568 1,681 Total inventory $ 13,632 $ 7,065 Accrued Liabilities Accrued liabilities consist of the following (in thousands): June 30, December 31, 2019 2018 Research, clinical study, and manufacturing expenses $ 18,932 $ 16,912 Payroll and related expenses 28,998 36,443 Other 11,334 9,095 Total accrued liabilities $ 59,264 $ 62,450 |
Revenue
Revenue | 6 Months Ended |
Jun. 30, 2019 | |
Revenue From Contract With Customer [Abstract] | |
Revenue | 5. Revenue The following table disaggregates total revenues from external customers by collaboration and license revenue and product sales (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 Collaboration and license revenue: KKC (Crysvita) $ 19,179 $ 1,577 $ 33,133 $ 1,592 Bayer 68 8,942 352 18,289 Total collaboration and license revenue 19,247 10,519 33,485 19,881 Product sales: Crysvita 1,006 26 1,594 26 Mepsevii 3,240 2,007 5,913 3,126 UX007 656 242 1,329 438 Total product sales 4,902 2,275 8,836 3,590 Total revenues $ 24,149 $ 12,794 $ 42,321 $ 23,471 The following table disaggregates total revenues based on geographic location (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 United States $ 20,163 $ 11,838 $ 34,618 $ 21,852 Europe 2,821 930 5,732 1,593 All other 1,165 26 1,971 26 Total revenues $ 24,149 $ 12,794 $ 42,321 $ 23,471 The following table presents changes in the contract assets (liabilities) (in thousands): Six Months Ended June 30, 2019 2018 Balance of contract assets (liabilities) at beginning of period $ 2,979 $ (5,986 ) Additions 352 18,877 Deductions (3,386 ) (14,135 ) Balance of contract liabilities at end of period $ (55 ) $ (1,244 ) The Company’s largest accounts receivable balance accounted for 92% and 88% of the total accounts receivable balance as of June 30, 2019 and December 31, 2018, respectively, and was due from a collaboration partner. |
License and Research Agreements
License and Research Agreements | 6 Months Ended |
Jun. 30, 2019 | |
Research Grant Agreement [Abstract] | |
License and Research Agreements | 6 . License and Research Agreements Kyowa Kirin Collaboration and License Agreement In August 2013, the Company entered into a collaboration and license agreement with Kyowa Kirin Co., Ltd. (KKC or formerly Kyowa Hakko Kirin Co., Ltd. or KHK). Under the terms of this collaboration and license agreement, as amended, the Company and KKC will collaborate on the development and commercialization of Crysvita in the field of orphan diseases in the United States and Canada, or the profit share territory, and in the European Union and Switzerland, 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 KKC, the Company will be the lead party for development activities in the profit share territory and in the European territory until the applicable transition dates; 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 KKC. 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 KKC, and KKC shall be responsible for 100% of the costs for development activities in Japan and Korea. On the applicable transition dates in the profit share territory and the European territory, KKC 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 KKC. Crysvita was approved in the European Union in February 2018 and was approved by the FDA in April 2018. The collaboration and license agreements are within the scope of ASC 808, which provides guidance on the presentation and disclosure of collaborative arrangements. Collaboration revenue related to sales in profit share territory The Company and KKC share commercial responsibilities and profits in the profit share territory until the applicable transition date. Under the collaboration agreement, KKC will manufacture and supply Crysvita for commercial use in the profit share territory. The remaining profit or loss after supply costs from commercializing products in the profit-share territory, until the applicable transition date, are shared between the Company and KKC 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 The Company is considered the agent in the profit share territory as KKC controls the product before transfer to the customers and has the ability to direct the use of and obtain substantially all of the remaining benefits from the product. The Company recognizes a pro-rata share of collaboration revenue, net of supply costs, in the period the sale occurs. The Company concluded that its portion of KKC’s sales in the profit share territory is analogous to a royalty and therefore recorded its share as collaboration revenue, similar to a royalty. Royalty revenue related to sales in European territory KKC has the commercial responsibility for in the European territory. The Company receives a royalty of up to 10% on net sales in the European territory, which is recognized as the underlying sales occur The Company’s Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 Company's share of collaboration revenue in profit share territory $ 17,248 $ 1,065 $ 29,187 $ 1,065 Royalty revenue in European territory 1,931 512 3,946 527 Total $ 19,179 $ 1,577 $ 33,133 $ 1,592 Product revenue related to sales in other territories The Company is responsible for commercializing in Latin America and Turkey. The Company is considered the principal in the arrangement as the Company controls the product before it is transferred to the customer. Accordingly, the Company records revenue on a gross basis related to the sale of once the product is delivered and the risk and title of the product is transferred to the distributor. For the three and six months ended June 30, 2019, the Company recorded product sales of $1.0 million and $1.6 million, respectively, net of estimated product returns and other deductions. The Company recorded a nominal amount of product sales for the three and six months ended June 30, 2018. Under KKC manufactures and supplies for sales in the above territories. The Company also pays to KKC a low single-digit royalty on net sales in Latin America. One of the wholly-owned subsidiaries of KKC has the option to assume responsibility for commercialization efforts in Turkey from the Company, after a certain minimum period. Cost sharing payments Under the collaboration agreement, KKC and the Company shares certain development and commercialization costs. Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 Research and development $ 5,895 $ 9,420 $ 12,994 $ 17,656 Selling, general and administrative 5,163 4,068 10,376 7,850 Total $ 11,058 $ 13,488 $ 23,370 $ 25,506 Collaboration receivable The Company had accounts receivable from KKC in the amount of $19.3 million and $11.2 million, from profit share revenue and royalties, and other receivables recorded in prepaid and other current assets of $13.8 million and $11.1 million from commercial and development activity reimbursements, as of June 30, 2019 and December 31, 2018, respectively. Bayer HealthCare LLC The Company has an agreement with Bayer Healthcare LLC (Bayer) to research, develop and commercialize AAV gene therapy products for treatment of hemophilia A (DTX 201). Under this agreement, Bayer has been granted an exclusive license to develop and commercialize one or more novel gene therapies for hemophilia A. The agreement requires that Bayer use commercially reasonable efforts to conduct and fund a proof-of-concept (POC) clinical trial and any subsequent clinical trials and commercialization of gene therapy products for treatment of hemophilia A. Bayer will have worldwide rights to commercialize the potential future product. Bayer is responsible to fund certain research and development services performed by the Company in the performance of its obligations under the annual research plan and budget. Under the terms of the agreement with Bayer, the Company is eligible to receive development and commercialization milestone payments of up to $232.0 million, as well as, royalty payments ranging in the high single-digit to low double-digit percentages, not exceeding the mid-teens, of net sales of licensed products. The Company achieved the first milestone in December 2017, the second milestone in April 2018, and has received $15.0 million for such milestones to date. As of the acquisition date of Dimension Therapeutics, Inc. on November 7, 2017, the Company valued the Bayer contract under ASC 805, Business Combinations, The Company evaluated the agreement under ASC 606 and recorded a contract liability as of November 7, 2017 of $2.5 million. It was determined that the performance obligations under the agreement include (i) research and development services to be provided over the research term, (ii) a development and commercialization license, and (iii) the Company’s participation in certain committees. It was determined that these performance obligations are not distinct in the context of the contract and therefore are a single performance obligation. The Company calculated the transaction price by including the unconstrained milestones along with the estimated payments for research and development services and recorded $0.1 million and $0.4 million as collaboration and license revenue for the three and six months ended June 30, 2019, respectively, and $9.0 million and $18.3 million for the three and six months ended June 30, 2018, respectively, by measuring the progress toward complete satisfaction of the performance obligation using an input measure. The performance obligation under the contract is expected to be substantially complete by end of 2019. As of June 30, 2019 and December 31, 2018, the Company had a $0.1 million contract liability and a $3.0 million contract asset, respectively. Arcturus The Company has a Research Collaboration and License Agreement with Arcturus to research and develop therapies for select rare diseases. Pursuant to the agreement, the Company incurred $0.4 million and $0.6 million for the three and six months ended June 30, 2019, respectively, and $0.5 million and $0.9 million for the three and six months ended June 30, 2018, respectively, in research and development expense for the funding of certain research services received from Arcturus. As of June 30, 2019 and December 31, 2018, the Company has a balance of none and $0.5 million, respectively, in prepaid expenses and other current assets, and a balance of none and $0.4 million, respectively, in accrued liabilities related to Arcturus. In June 2019, the Company entered into an Equity Purchase Agreement and an amendment to the Research Collaboration and License Agreement to expand the field of use and increase the number of disease targets to include mRNA, DNA and siRNA therapeutics for up to 12 rare diseases. Pursuant to the agreements, the Company paid $6.0 million in cash upfront to Arcturus and purchased 2,400,000 shares of Arcturus’ common stock at a stated value of $10.00 per share, resulting in a total of $30.0 million of consideration paid at the close of the transaction. As a result, the Company received expanded license rights; the Arcturus common stock; an option to purchase an additional 600,000 shares of Arcturus’ common stock at $16.00 per share, which may be exercised up to two years after the agreement effective date, with certain restrictions; in addition to other changes as noted in the agreement. The period for the Company to exercise its option to purchase the additional stock may also be extended under certain circumstances as specified in the Equity Purchase Agreement. The Company is restricted from selling the 2,400,000 shares of common stock for a period of two years from the purchase date. The additional stock, if purchased, are also restricted from sale for a period of time as specified in the agreement. The Company also received the right to nominate one member to the Arcturus Board of Directors as well as one Board observer. Under the amended license agreement, certain early-stage milestone payments are reduced and the total potential milestone payments are increased due to the expanded number of targets. Arcturus is also entitled to reimbursement of related research expenses and royalties on commercial sales. Immediately after the purchase, the Company held 18.2% of Arcturus’ outstanding common stock, based on Arcturus’ outstanding common stock balance as of the transaction date. The recorded the common stock investment at $13.9 million on the transaction date, which was based on the quoted market price on the closing date. As a result of the equity ownership and the right to nominate a board member, it was determined that the Company has significant influence over Arcturus. The Company elected to apply the fair value option to account for the equity investment in Arcturus. The Company also accounts for the option to purchase additional shares of Arcturus common stock at fair value, which was recorded at $0.5 million on the transaction date based on the Black-Scholes option pricing method. The remaining $15.6 million of the total $30.0 million paid as consideration was attributed to the additional license rights obtained and was recorded as in-process research and development expense. For the three and six months ended June 30, 2019, the Company recorded an increase in the fair value of Arcturus common stock of $8.8 million and an increase in fair value of the option to purchase additional shares of Arcturus common stock of $1.0 million in its Condensed Consolidated Statement of Operations. As of June 30, 2019, the fair value of the Company’s investment in Arcturus common stock was $ 22.7 million based on the quoted market price on that date and the fair value of the Company’s option to purchase additional share s of Arcturus common stock was $ 1.5 million based on the Black-Scholes option pricing method. |
Stock-Based Awards
Stock-Based Awards | 6 Months Ended |
Jun. 30, 2019 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Stock-Based Awards | 7 . Stock-Based Awards The 2014 Incentive Plan (the 2014 Plan) provides for automatic annual increases in shares available for grant, beginning on January 1, 2015 through January 1, 2024. As of June 30, 2019 were 2,582,049 shares reserved under the 2014 Plan for the future issuance of equity awards and 2,740,058 shares reserved for the 2014 Employee Stock Purchase Plan. The table below sets forth the stock-based compensation expense for the periods presented (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 Cost of sales $ 51 $ — $ 85 $ — Research and development 12,032 11,644 23,262 22,891 Selling, general and administrative 10,124 7,919 19,081 15,469 Total stock-based compensation expense $ 22,207 $ 19,563 $ 42,428 $ 38,360 |
Net Loss Per Share
Net Loss Per Share | 6 Months Ended |
Jun. 30, 2019 | |
Earnings Per Share [Abstract] | |
Net Loss Per Share | 8 . Net Loss Per Share Basic net loss per share has been computed by dividing the net loss by the weighted-average number of shares of common stock outstanding during the period. Diluted net loss per share is calculated by dividing net loss by the weighted-average number of shares of common stock and potential dilutive securities outstanding during the period. The following weighted-average outstanding common stock equivalents were excluded from the computation of diluted net loss per share for the periods presented because including them would have been antidilutive: Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 Options to purchase common stock and restricted stock units 8,220,733 7,735,231 7,825,615 7,479,526 Employee stock purchase plan 7,857 9,369 3,950 4,710 Common stock warrants 149,700 149,700 149,700 149,700 8,378,290 7,894,300 7,979,265 7,633,936 |
Equity Transactions
Equity Transactions | 6 Months Ended |
Jun. 30, 2019 | |
Equity [Abstract] | |
Equity Transactions | 9 . Equity Transactions In July 2017, the Company entered into an At-The-Market, or ATM, sales agreement with Cowen and Company, LLC (Cowen), whereby the Company can sell up to $150.0 million in aggregate proceeds of common stock from time to time, through with Cowen as its sales agent. During the three and six months ended June 30, 2019, the Company sold 88,978 and 468,685 shares of common stock, respectively, resulting in net proceeds of approximately $5.5 million and $24.8 million, respectively, after commissions and other offering costs. During the three and six months ended June 30, 2018, the Company sold 240,417 shares of common stock, resulting in net proceeds of approximately $11.8 million after commissions and other offering costs. In February 2019, the Company completed an underwritten public offering in which 5,833,333 shares of common stock were sold, which included 760,869 shares purchased by the underwriters pursuant to an option granted to them in connection with the offering, at a public offering price of $60.00 per share. The total proceeds that the Company received from the offering were approximately $330.4 million, net of underwriting discounts and commissions. |
Accumulated Other Comprehensive
Accumulated Other Comprehensive Income (Loss) | 6 Months Ended |
Jun. 30, 2019 | |
Other Comprehensive Income Loss Net Of Tax Period Increase Decrease [Abstract] | |
Accumulated Other Comprehensive Income (Loss) | 10 . Accumulated Other Comprehensive Income (Loss) Total accumulated other comprehensive income (loss) consisted of the following (in thousands): June 30, December 31, 2019 2018 Foreign currency translation adjustments $ (174 ) $ (329 ) Unrealized gain (loss) on securities available-for-sale 429 (304 ) Total accumulated other comprehensive income (loss) $ 255 $ (633 ) |
Leases
Leases | 6 Months Ended |
Jun. 30, 2019 | |
Lessee Disclosure [Abstract] | |
Leases | 11 . Leases As described in “Note 2. Summary of Significant Accounting Policies”, the Company adopted Topic 842 Topic 840 The Company leases office space and research, testing and manufacturing laboratory space in various facilities in Novato and Brisbane, California, in Cambridge and Woburn, Massachusetts, and in certain foreign countries, under operating agreements expiring at various dates through 2028. 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. None of these optional periods have been considered in the determination of the right-of-use asset or the lease liability for the leases as the Company did not consider it reasonably certain that it would exercise any such options. The Company recognizes lease expense on a straight-line basis over the non-cancelable term of its operating leases. The variable lease expense primarily consists of common area maintenance and other operating costs. The components of lease expense were as follows (in thousands): Three Months Ended Six Months Ended June 30, 2019 June 30, 2019 Operating lease expense $ 2,373 $ 4,330 Variable lease expense 669 1,321 Total lease expense $ 3,042 $ 5,651 Cash paid for amounts included in the measurement of lease liabilities for the three and six months ended June 30, 2019 was $2.2 million and $4.2 million, respectively, and was included in net cash provided by operating activities in the Consolidated Statements of Cash Flows. Future minimum lease payments under non-cancellable leases as of June 30, 2019 were as follows (in thousands): Year Ending December 31, Leases 2019 (remaining) $ 4,724 2020 9,639 2021 7,561 2022 7,433 2023 7,541 Thereafter 13,153 Total future lease payments 50,051 Less: Amount representing interest (10,084 ) Present value of future lease payments $ 39,967 Less: Short-term lease liabilities (6,999 ) Long-term lease liabilities $ 32,968 Lease liabilities are based on the net present value of the remaining lease payments over the remaining lease term. As of June 30, 2019, the weighted-average remaining lease term was 5.98 years and the weighted-average discount rate used to determine the lease liability was 7.5%. |
Gain from Sale of Priority Revi
Gain from Sale of Priority Review Voucher | 6 Months Ended |
Jun. 30, 2019 | |
Goodwill And Intangible Assets Disclosure [Abstract] | |
Gain from Sale of Priority Review Voucher | 12 . Gain from Sale of Priority Review Vouchers In January 2018, the Company completed the sale of a Rare Pediatric Disease Priority Review Voucher (PRV) it received in connection with the approval of Mepsevii for $130.0 million. In June 2018, the Company also completed the sale of the PRV it received in connection with the approval of Crysvita for $80.6 million, net, which was shared equally with KKC. As the PRVs did not have a carrying value, the gain recognized was equal to the net proceeds received. The Company recorded its portion of the net proceeds in other income of $40.3 million and $170.3 million for the three and six months ended June 30, 2018, respectively, as a gain from the sale of the PRVs. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 6 Months Ended |
Jun. 30, 2019 | |
Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation The accompanying unaudited condensed consolidated financial statements include the accounts of the Company and its wholly-owned subsidiaries and have been prepared in accordance with U.S. generally accepted accounting principles (GAAP) for interim financial information and in accordance with the instructions to Form 10-Q and Rule 10-01 of Regulation S-X. Accordingly, they do not include all of the information and footnotes required by GAAP for complete financial statements. The unaudited interim consolidated financial statements have been prepared on the same basis as the annual financial statements. In the opinion of management, the accompanying unaudited condensed consolidated financial statements reflect all adjustments (consisting only of normal recurring adjustments) considered necessary for a fair presentation. These financial statements should be read in conjunction with the audited financial statements and notes thereto for the preceding fiscal year contained in the Company’s Annual Report on Form 10-K filed on February 19, 2019 with the United States Securities and Exchange Commission (SEC). The results of operations for the three and six months ended June 30, 2019 are not necessarily indicative of the results to be expected for the year ending December 31, 2019. The condensed consolidated balance sheet as of December 31, 2018 has been derived from audited financial statements at that date, but does not include all of the information required by GAAP for complete financial statements. |
Use of Estimates | Use of Estimates The accompanying consolidated financial statements have been prepared in accordance with 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, Cash Equivalents, and Restricted Cash | Cash, Cash Equivalents and Restricted Cash Restricted cash primarily consists of money market accounts as collateral for the Company’s obligations under its facility leases. The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the consolidated balance sheets that sum to the total of the same such amounts shown in the consolidated statement of cash flows (in thousands): June 30, 2019 2018 Cash and cash equivalents $ 131,337 $ 110,854 Restricted cash included in prepaid expenses and other current assets 161 652 Restricted cash included in other assets 2,499 1,822 Total cash, cash equivalents, and restricted cash shown in the statements of cash flows $ 133,997 $ 113,328 |
Investment in Equity Securities | Investment in Equity Securities In June 2019, the Company entered into an amendment to the Research Collaboration and License Agreement and an Equity Purchase Agreement with Arcturus Therapeutics Holdings Inc. (“Arcturus”). Pursuant to the Equity Purchase Agreement, the Company purchased 2,400,000 shares of common stock, or approximately 18.2% of Arcturus’s outstanding shares of common stock as of the closing date of the transaction and received an option to purchase an additional 600,000 shares of common stock. The investment was accounted for using the equity method of accounting as it was determined that the Company has significant influence over, but does not control the significant activities of Arcturus. The Company elected to apply the fair value option to account for the equity investment in Arcturus. The decision to elect the fair value option is irrevocable and is determined on an instrument by instrument basis. The option to purchase additional stock was accounted for at fair value using Black-Scholes option pricing method. The changes in fair value of the equity investment and option to purchase additional stock are included in the Condensed Consolidated Statements of Operations. See “Note 6. License and Research Agreements” for additional details on the Arcturus transaction. |
Revenue Recognition | Revenue Recognition Collaboration and license revenue The Company has certain license and collaboration agreements that are within the scope of Accounting Standards Codification (ASC) 808, Collaborative Agreements provides guidance on the presentation and disclosure of collaborative arrangements. Generally, the classification of the transactions under the collaborative arrangements is determined based on the nature of contractual terms of the arrangement, along with the nature of the operations of the participants. The Company records its share of collaboration revenue, net of transfer pricing related to net sales in the period in which such sales occur, if the Company is considered as an agent in the arrangement. The Company is considered an agent when the collaboration partner controls the product before transfer to the customers and has the ability to direct the use of and obtain substantially all of the remaining benefits from the product. Funding received related to research and development services and commercialization costs is generally classified as a reduction of research and development expenses and selling, general and administrative expenses, respectively, in the consolidated statement of operations, because the provision of such services for collaborative partners are not considered to be part of the Company’s ongoing major or central operations. The also receives royalty revenues under certain of the Company’s license or collaboration agreements in exchange for license of intellectual property. If the Company does not have any future performance obligations for these license or collaboration agreements, royalty revenue is recorded as the underlying sales occur. In to record collaboration revenue, the Company utilizes certain information from its , including revenue from the sale of the product, associated reserves on revenue, and costs incurred for development and sales activities. For the periods covered in the financial statements presented, there have been no material changes to prior period estimates of revenues and expenses. The terms of the Company’s collaboration agreements may contain multiple performance obligations, which may include licenses and research and development activities. The Company evaluates these agreements under ASC 606, Revenue from Contracts with Customers (ASC 606), to determine the distinct performance obligations. The Company analogizes to ASC 606 for the accounting for distinct performance obligations for which there is a customer relationship. Prior to recognizing revenue, the Company makes estimates of the transaction price, including variable consideration that is subject to a constraint. Amounts of variable consideration are included in the transaction price to the extent that it is probable that a significant reversal in the amount of cumulative revenue recognized will not occur and when the uncertainty associated with the variable consideration is subsequently resolved. Total consideration may include nonrefundable upfront license fees, payments for research and development activities, reimbursement of certain third-party costs, payments based upon the achievement of specified milestones, and royalty payments based on product sales derived from the collaboration. If there are multiple distinct performance obligations, the Company allocates the transaction price to each distinct performance obligation based on its relative standalone selling price. The standalone selling price is generally determined based on the prices charged to customers or using expected cost plus margin. The Company estimates the efforts needed to complete the performance obligation and recognizes revenue by measuring the progress towards complete satisfaction of the performance obligation using an input measure. Product sales The Company sells its approved products through a limited number of distributors. Under ASC 606, revenue from product sales is recognized at the point in time when the delivery is made and when title and risk of loss transfers to these distributors. The Company also recognizes revenue from sales of certain products on a “named patient” basis, which are allowed in certain countries prior to the commercial approval of the product. Prior to recognizing revenue, the Company makes estimates of the transaction price, including any variable consideration that is subject to a constraint. Amounts of variable consideration are included in the transaction price to the extent that it is probable that a significant reversal in the amount of cumulative revenue recognized will not occur and when the uncertainty associated with the variable consideration is subsequently resolved. Product sales are recorded net of estimated government-mandated rebates and chargebacks, estimated product returns, and other deductions. Provisions for returns and other adjustments are provided for in the period the related revenue is recorded, as estimated by management. |
Leases | Leases The Company adopted Accounting Standards Update (ASU) No. 2016-02, Leases (Topic 842) Leases (Topic 840) The Company determines if an arrangement includes a lease at inception. Right-of-use assets and lease liabilities are recognized based on the present value of the future minimum lease payments over the lease term at the commencement date. The right-of-use asset includes any lease payments made and excludes lease incentives. Incremental borrowing rate is used in determining the present value of future payments. The Company applies a portfolio approach to the property leases to apply an incremental borrowing rate to leases with similar lease terms. The lease terms may include options to extend or terminate the lease. The Company recognizes the options to extend the lease as part of the right-of-use assets and lease liabilities only if it is reasonably certain that the option would be exercised. Lease expense for minimum lease payments is recognized on a straight-line basis over the non-cancelable lease term. Prior period amounts have not been adjusted and continue to be reported in accordance with the Company’s historical accounting under previous lease guidance, Topic 840. As a result of the adoption of the new guidance, the Company recorded a right-of-use asset of $16.2 million, a short-term lease liability of $4.5 million, and a long-term lease liability of $17.0 million and no cumulative effect adjustment was made to the retained earnings as of the adoption date. In addition, as of the adoption date, the Company derecognized a net deferred rent obligation of $5.2 million. See Note 11 for further disclosure. |
Recently Issued Accounting Pronouncements | Recently Issued Accounting Pronouncements In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments — Credit Losses, (Topic 326): Measurement of Credit Losses on Financial Instruments, |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Accounting Policies [Abstract] | |
Summary of Reconciliation of Cash, Cash Equivalents and Restricted Cash | The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the consolidated balance sheets that sum to the total of the same such amounts shown in the consolidated statement of cash flows (in thousands): June 30, 2019 2018 Cash and cash equivalents $ 131,337 $ 110,854 Restricted cash included in prepaid expenses and other current assets 161 652 Restricted cash included in other assets 2,499 1,822 Total cash, cash equivalents, and restricted cash shown in the statements of cash flows $ 133,997 $ 113,328 |
Financial Instruments (Tables)
Financial Instruments (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Fair Value Disclosures [Abstract] | |
Summary of Financial Assets Measured on Recurring Basis | The following tables set forth the fair value of the Company’s financial assets remeasured on a recurring basis based on the three-tier fair value hierarchy (in thousands): June 30, 2019 Level 1 Level 2 Level 3 Total Money market funds $ 92,414 $ — $ — $ 92,414 Time deposits — 10,000 — 10,000 Corporate bonds — 58,742 — 58,742 Commercial paper — 132,947 — 132,947 Asset-backed securities — 40,901 — 40,901 U.S. Government Treasury and agency securities 112,200 157,135 — 269,335 Investment in Arcturus equity securities 22,656 — 1,511 24,167 Total $ 227,270 $ 399,725 $ 1,511 $ 628,506 December 31, 2018 Level 1 Level 2 Level 3 Total Money market funds $ 72,999 $ — $ — $ 72,999 Time deposits — 10,000 — 10,000 Corporate bonds — 179,926 — 179,926 Commercial paper — 50,198 — 50,198 Asset-backed securities — 22,587 — 22,587 U.S. Government Treasury and agency securities — 99,034 — 99,034 Total $ 72,999 $ 361,745 $ — $ 434,744 |
Balance Sheet Components (Table
Balance Sheet Components (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Investments Debt And Equity Securities [Abstract] | |
Summary of Cash Equivalents and Investments Classified as Available For Sale Securities | The fair values of cash equivalents and short-term investments classified as available-for-sale securities consisted of the following (in thousands): June 30, 2019 Gross Unrealized Amortized Cost Gains Losses Estimated Fair Value Money market funds $ 92,414 $ — $ — $ 92,414 Time deposits 10,000 — — 10,000 Corporate bonds 58,692 50 — 58,742 Commercial paper 132,947 — — 132,947 Asset-backed securities 40,834 67 — 40,901 U.S. Government Treasury and agency securities 269,023 312 — 269,335 Total $ 603,910 $ 429 $ — $ 604,339 December 31, 2018 Gross Unrealized Amortized Cost Gains Losses Estimated Fair Value Money market funds $ 72,999 $ — $ — $ 72,999 Time deposits 10,000 — — 10,000 Corporate bonds 180,167 — (241 ) 179,926 Commercial paper 50,198 — — 50,198 Asset-backed securities 22,597 — (10 ) 22,587 U.S. Government Treasury and agency securities 99,087 2 (55 ) 99,034 Total $ 435,048 $ 2 $ (306 ) $ 434,744 |
Summary of Inventory | Inventory consists of the following (in thousands): June 30, December 31, 2019 2018 Work-in-progress $ 11,064 $ 5,384 Finished goods 2,568 1,681 Total inventory $ 13,632 $ 7,065 |
Accrued Liabilities | Accrued liabilities consist of the following (in thousands): June 30, December 31, 2019 2018 Research, clinical study, and manufacturing expenses $ 18,932 $ 16,912 Payroll and related expenses 28,998 36,443 Other 11,334 9,095 Total accrued liabilities $ 59,264 $ 62,450 |
Revenue (Tables)
Revenue (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Revenue From Contract With Customer [Abstract] | |
Summary of Disaggregation of Total Revenues | The following table disaggregates total revenues from external customers by collaboration and license revenue and product sales (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 Collaboration and license revenue: KKC (Crysvita) $ 19,179 $ 1,577 $ 33,133 $ 1,592 Bayer 68 8,942 352 18,289 Total collaboration and license revenue 19,247 10,519 33,485 19,881 Product sales: Crysvita 1,006 26 1,594 26 Mepsevii 3,240 2,007 5,913 3,126 UX007 656 242 1,329 438 Total product sales 4,902 2,275 8,836 3,590 Total revenues $ 24,149 $ 12,794 $ 42,321 $ 23,471 The following table disaggregates total revenues based on geographic location (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 United States $ 20,163 $ 11,838 $ 34,618 $ 21,852 Europe 2,821 930 5,732 1,593 All other 1,165 26 1,971 26 Total revenues $ 24,149 $ 12,794 $ 42,321 $ 23,471 |
Summary of Changes in Contract Assets (Liabilities) | The following table presents changes in the contract assets (liabilities) (in thousands): Six Months Ended June 30, 2019 2018 Balance of contract assets (liabilities) at beginning of period $ 2,979 $ (5,986 ) Additions 352 18,877 Deductions (3,386 ) (14,135 ) Balance of contract liabilities at end of period $ (55 ) $ (1,244 ) |
License and Research Agreemen_2
License and Research Agreements (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Research Grant Agreement [Abstract] | |
Share of Collaboration Revenue Related to Crysvita | The Company’s Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 Company's share of collaboration revenue in profit share territory $ 17,248 $ 1,065 $ 29,187 $ 1,065 Royalty revenue in European territory 1,931 512 3,946 527 Total $ 19,179 $ 1,577 $ 33,133 $ 1,592 |
Schedule of Cost Sharing Payments | Under the collaboration agreement, KKC and the Company shares certain development and commercialization costs. Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 Research and development $ 5,895 $ 9,420 $ 12,994 $ 17,656 Selling, general and administrative 5,163 4,068 10,376 7,850 Total $ 11,058 $ 13,488 $ 23,370 $ 25,506 |
Stock-Based Awards (Tables)
Stock-Based Awards (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Summary of Stock-Based Compensation Expense | The table below sets forth the stock-based compensation expense for the periods presented (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 Cost of sales $ 51 $ — $ 85 $ — Research and development 12,032 11,644 23,262 22,891 Selling, general and administrative 10,124 7,919 19,081 15,469 Total stock-based compensation expense $ 22,207 $ 19,563 $ 42,428 $ 38,360 |
Net Loss Per Share (Tables)
Net Loss Per Share (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Earnings Per Share [Abstract] | |
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 for the periods presented because including them would have been antidilutive: Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 Options to purchase common stock and restricted stock units 8,220,733 7,735,231 7,825,615 7,479,526 Employee stock purchase plan 7,857 9,369 3,950 4,710 Common stock warrants 149,700 149,700 149,700 149,700 8,378,290 7,894,300 7,979,265 7,633,936 |
Accumulated Other Comprehensi_2
Accumulated Other Comprehensive Income (Loss) (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
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): June 30, December 31, 2019 2018 Foreign currency translation adjustments $ (174 ) $ (329 ) Unrealized gain (loss) on securities available-for-sale 429 (304 ) Total accumulated other comprehensive income (loss) $ 255 $ (633 ) |
Leases (Tables)
Leases (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Lessee Disclosure [Abstract] | |
Summary of Components of Lease Expense | The components of lease expense were as follows (in thousands): Three Months Ended Six Months Ended June 30, 2019 June 30, 2019 Operating lease expense $ 2,373 $ 4,330 Variable lease expense 669 1,321 Total lease expense $ 3,042 $ 5,651 |
Schedule of Future Minimum Lease Payments under Non-cancellable Leases | Future minimum lease payments under non-cancellable leases as of June 30, 2019 were as follows (in thousands): Year Ending December 31, Leases 2019 (remaining) $ 4,724 2020 9,639 2021 7,561 2022 7,433 2023 7,541 Thereafter 13,153 Total future lease payments 50,051 Less: Amount representing interest (10,084 ) Present value of future lease payments $ 39,967 Less: Short-term lease liabilities (6,999 ) Long-term lease liabilities $ 32,968 |
Organization - Additional Infor
Organization - Additional Information (Details) | 6 Months Ended |
Jun. 30, 2019Segment | |
United States of America | |
Organization And Nature Of Business [Line Items] | |
Number of reportable segments | 1 |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies - Summary of Reconciliation of Cash, Cash Equivalents and Restricted Cash (Details) - USD ($) $ in Thousands | Jun. 30, 2019 | Dec. 31, 2018 | Jun. 30, 2018 | Dec. 31, 2017 |
Accounting Policies [Abstract] | ||||
Cash and cash equivalents | $ 131,337 | $ 113,432 | $ 110,854 | |
Restricted cash included in prepaid expenses and other current assets | $ 161 | $ 652 | ||
Restricted Cash and Cash Equivalents, Current, Asset, Statement of Financial Position [Extensible List] | us-gaap:PrepaidExpensesAndOtherCurrentAssetsMember | us-gaap:PrepaidExpensesAndOtherCurrentAssetsMember | ||
Restricted cash included in other assets | $ 2,499 | $ 1,822 | ||
Restricted Cash and Cash Equivalents, Noncurrent, Asset, Statement of Financial Position [Extensible List] | us-gaap:OtherNoncurrentAssetsMember | us-gaap:OtherNoncurrentAssetsMember | ||
Total cash, cash equivalents, and restricted cash shown in the statements of cash flows | $ 133,997 | $ 115,525 | $ 113,328 | $ 103,041 |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies - Additional Information (Details) - USD ($) | 1 Months Ended | ||
Jun. 30, 2019 | Jan. 01, 2019 | Dec. 31, 2018 | |
Summary Of Significant Accounting Policies [Line Items] | |||
Common stock purchased | 57,665,375 | 50,860,588 | |
Right-to-use-asset | $ 33,356,000 | ||
Short-term lease liabilities | 6,999,000 | ||
Long-term lease liability | $ 32,968,000 | ||
Accounting Standards Update 2016-02 | |||
Summary Of Significant Accounting Policies [Line Items] | |||
Right-to-use-asset | $ 16,200,000 | ||
Short-term lease liabilities | 4,500,000 | ||
Long-term lease liability | 17,000,000 | ||
Adjustment to retained earnings | 0 | ||
Deferred rent derecognized, net | $ 5,200,000 | ||
Arcturus Research Collaboration and License Agreement | |||
Summary Of Significant Accounting Policies [Line Items] | |||
Common stock purchased | 2,400,000 | ||
Percentage of outstanding common shares | 18.20% | ||
Option to purchase additional common stock, shares | 600,000 |
Financial Instruments - Summary
Financial Instruments - Summary of Financial Assets Measured on Recurring Basis (Details) - USD ($) $ in Thousands | Jun. 30, 2019 | Dec. 31, 2018 |
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | $ 628,506 | |
Total fair value | 604,339 | $ 434,744 |
Level 1 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 227,270 | |
Total fair value | 72,999 | |
Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 399,725 | |
Total fair value | 361,745 | |
Level 3 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 1,511 | |
Money Market Funds | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 92,414 | |
Total fair value | 92,414 | 72,999 |
Money Market Funds | Level 1 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 92,414 | |
Total fair value | 72,999 | |
Asset-backed Securities | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 40,901 | |
Total fair value | 40,901 | 22,587 |
Asset-backed Securities | Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 40,901 | |
Total fair value | 22,587 | |
Time Deposits | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 10,000 | |
Total fair value | 10,000 | 10,000 |
Time Deposits | Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 10,000 | |
Total fair value | 10,000 | |
Corporate Bonds | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 58,742 | |
Total fair value | 58,742 | 179,926 |
Corporate Bonds | Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 58,742 | |
Total fair value | 179,926 | |
Commercial Paper | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 132,947 | |
Total fair value | 132,947 | 50,198 |
Commercial Paper | Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 132,947 | |
Total fair value | 50,198 | |
U.S. Government Treasury and Agency Securities | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 269,335 | |
Total fair value | 269,335 | 99,034 |
U.S. Government Treasury and Agency Securities | Level 1 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 112,200 | |
U.S. Government Treasury and Agency Securities | Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 157,135 | |
Total fair value | $ 99,034 | |
Investment in Arcturus Equity Securities | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 24,167 | |
Investment in Arcturus Equity Securities | Level 1 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 22,656 | |
Investment in Arcturus Equity Securities | Level 3 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | $ 1,511 |
Financial Instruments - Additio
Financial Instruments - Additional Information (Details) - Arcturus Research Collaboration and License Agreement - USD ($) $ in Millions | 3 Months Ended | 6 Months Ended |
Jun. 30, 2019 | Jun. 30, 2019 | |
Schedule Of Available For Sale Securities [Line Items] | ||
Change in fair value of common stock | $ 8.8 | $ 8.8 |
Change in fair value of additional common stock | $ 1 | $ 1 |
Balance Sheet Components - Summ
Balance Sheet Components - Summary of Cash Equivalents and Investments Classified as Available For Sale Securities (Details) - USD ($) $ in Thousands | Jun. 30, 2019 | Dec. 31, 2018 |
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | $ 603,910 | $ 435,048 |
Gross Unrealized Gains | 429 | 2 |
Gross Unrealized Losses | (306) | |
Estimated Fair Value | 604,339 | 434,744 |
Money Market Funds | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 92,414 | 72,999 |
Estimated Fair Value | 92,414 | 72,999 |
Asset-backed Securities | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 40,834 | 22,597 |
Gross Unrealized Gains | 67 | |
Gross Unrealized Losses | (10) | |
Estimated Fair Value | 40,901 | 22,587 |
Time Deposits | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 10,000 | 10,000 |
Estimated Fair Value | 10,000 | 10,000 |
Corporate Bonds | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 58,692 | 180,167 |
Gross Unrealized Gains | 50 | |
Gross Unrealized Losses | (241) | |
Estimated Fair Value | 58,742 | 179,926 |
Commercial Paper | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 132,947 | 50,198 |
Estimated Fair Value | 132,947 | 50,198 |
U.S. Government Treasury and Agency Securities | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 269,023 | 99,087 |
Gross Unrealized Gains | 312 | 2 |
Gross Unrealized Losses | (55) | |
Estimated Fair Value | $ 269,335 | $ 99,034 |
Balance Sheet Components - Su_2
Balance Sheet Components - Summary of Inventory (Details) - USD ($) $ in Thousands | Jun. 30, 2019 | Dec. 31, 2018 |
Investments Debt And Equity Securities [Abstract] | ||
Work-in-progress | $ 11,064 | $ 5,384 |
Finished goods | 2,568 | 1,681 |
Total inventory | $ 13,632 | $ 7,065 |
Balance Sheet Components - Accr
Balance Sheet Components - Accrued Liabilities (Details) - USD ($) $ in Thousands | Jun. 30, 2019 | Dec. 31, 2018 |
Payables And Accruals [Abstract] | ||
Research, clinical study, and manufacturing expenses | $ 18,932 | $ 16,912 |
Payroll and related expenses | 28,998 | 36,443 |
Other | 11,334 | 9,095 |
Total accrued liabilities | $ 59,264 | $ 62,450 |
Revenue - Summary of Disaggrega
Revenue - Summary of Disaggregation of Total Revenues from External Customers (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Total revenues | ||||
Total revenues | $ 24,149 | $ 12,794 | $ 42,321 | $ 23,471 |
Bayer HealthCare LLC | ||||
Total revenues | ||||
Total revenues | 100 | 9,000 | 400 | 18,300 |
Collaboration and License | ||||
Total revenues | ||||
Total revenues | 19,247 | 10,519 | 33,485 | 19,881 |
Collaboration and License | Kyowa Kirin Collaboration | ||||
Total revenues | ||||
Total revenues | 19,179 | 1,577 | 33,133 | 1,592 |
Collaboration and License | Bayer HealthCare LLC | ||||
Total revenues | ||||
Total revenues | 68 | 8,942 | 352 | 18,289 |
Product Sales | ||||
Total revenues | ||||
Total revenues | 4,902 | 2,275 | 8,836 | 3,590 |
Product Sales | Crysvita | ||||
Total revenues | ||||
Total revenues | 1,006 | 26 | 1,594 | 26 |
Product Sales | Mepsevii | ||||
Total revenues | ||||
Total revenues | 3,240 | 2,007 | 5,913 | 3,126 |
Product Sales | UX007 | ||||
Total revenues | ||||
Total revenues | $ 656 | $ 242 | $ 1,329 | $ 438 |
Revenue - Summary of Disaggre_2
Revenue - Summary of Disaggregation of Total Revenues (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Disaggregation Of Revenue [Line Items] | ||||
Total revenues | $ 24,149 | $ 12,794 | $ 42,321 | $ 23,471 |
United States | ||||
Disaggregation Of Revenue [Line Items] | ||||
Total revenues | 20,163 | 11,838 | 34,618 | 21,852 |
Europe | ||||
Disaggregation Of Revenue [Line Items] | ||||
Total revenues | 2,821 | 930 | 5,732 | 1,593 |
All other | ||||
Disaggregation Of Revenue [Line Items] | ||||
Total revenues | $ 1,165 | $ 26 | $ 1,971 | $ 26 |
Revenue - Summary of Changes in
Revenue - Summary of Changes in Contract Assets (Liabilities) (Details) - USD ($) $ in Thousands | 6 Months Ended | |
Jun. 30, 2019 | Jun. 30, 2018 | |
Disaggregation Of Revenue [Abstract] | ||
Contract assets | $ 2,979 | |
Contract liabilities | $ (5,986) | |
Additions | 352 | 18,877 |
Deductions | (3,386) | (14,135) |
Contract liabilities | $ (55) | $ (1,244) |
Revenue - Additional Informatio
Revenue - Additional Information (Details) | Jun. 30, 2019 | Dec. 31, 2018 |
Disaggregation Of Revenue [Abstract] | ||
Percentage of gross accounts receivable balance | 92.00% | 88.00% |
License and Research Agreemen_3
License and Research Agreements - Additional Information (Details) | Nov. 07, 2017USD ($) | Jun. 30, 2019USD ($)DiseaseTarget$ / sharesshares | Jun. 30, 2019USD ($)$ / sharesshares | Jun. 30, 2018USD ($) | Jun. 30, 2019USD ($)$ / sharesshares | Jun. 30, 2018USD ($) | Dec. 31, 2018USD ($)shares |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||
Collaboration and license revenue | $ 24,149,000 | $ 12,794,000 | $ 42,321,000 | $ 23,471,000 | |||
Research and development | 96,045,000 | 76,835,000 | 174,150,000 | 152,339,000 | |||
Contract asset | $ 2,979,000 | ||||||
Prepaid expenses and other current assets | $ 46,612,000 | 46,612,000 | 46,612,000 | 42,858,000 | |||
Accrued liabilities | $ 59,264,000 | $ 59,264,000 | $ 59,264,000 | $ 62,450,000 | |||
Common stock purchased | shares | 57,665,375 | 57,665,375 | 57,665,375 | 50,860,588 | |||
Common stock investment value | $ 58,000 | $ 58,000 | $ 58,000 | $ 51,000 | |||
Fair value, investments | 628,506,000 | 628,506,000 | 628,506,000 | ||||
Dimension | |||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||
Business acquisition date | Nov. 7, 2017 | ||||||
Research and development | 100,000 | 4,400,000 | $ 200,000 | 8,800,000 | |||
Amortization completion year | 2019 | ||||||
Dimension | Contract Asset | |||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||
Business combination recognized intangible assets | $ 13,500,000 | ||||||
Bayer HealthCare LLC | |||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||
Collaboration and license revenue | 100,000 | 9,000,000 | $ 400,000 | 18,300,000 | |||
Contract liability | $ 2,500,000 | 100,000 | 100,000 | 100,000 | |||
Contract asset | 3,000,000 | ||||||
Product Sales | |||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||
Collaboration and license revenue | 4,902,000 | 2,275,000 | 8,836,000 | 3,590,000 | |||
Kyowa Kirin Collaboration | License Agreement | Prepaid and Other Current Assets | |||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||
License agreement other receivables | 13,800,000 | 13,800,000 | 13,800,000 | ||||
Kyowa Kirin Collaboration | License Agreement | Product Sales | |||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||
Collaboration and license revenue | 1,000,000 | 1,600,000 | |||||
Kyowa Kirin Collaboration | License Agreement | Profit Share Revenue | |||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||
License agreement accounts receivable | 19,300,000 | 19,300,000 | $ 19,300,000 | 11,200,000 | |||
Kyowa Kirin Collaboration | License Agreement | Commercial and Development Activity Reimbursements | |||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||
License agreement other receivables | 11,100,000 | ||||||
Kyowa Kirin Collaboration | Profit Share Territory | License Agreement | |||||||
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 Kirin Collaboration | European Territory | License Agreement | Maximum | |||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||
Percentage of royalty on net sales receives | 10.00% | ||||||
Research, Develop and Commercialize Adeno Associated Virus Gene Therapy Products | Bayer HealthCare LLC | |||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||
Milestone payments received | $ 15,000,000 | ||||||
Research, Develop and Commercialize Adeno Associated Virus Gene Therapy Products | Maximum | Bayer HealthCare LLC | |||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||
Future contingent milestone payments | 232,000,000 | 232,000,000 | 232,000,000 | ||||
Arcturus | |||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||
Research and development | 6,000,000 | 400,000 | $ 500,000 | 600,000 | $ 900,000 | ||
Prepaid expenses and other current assets | 0 | 0 | 0 | 500,000 | |||
Accrued liabilities | $ 0 | $ 0 | $ 0 | $ 400,000 | |||
Common stock purchased | shares | 2,400,000 | 2,400,000 | 2,400,000 | ||||
Common stock. per share | $ / shares | $ 10 | $ 10 | $ 10 | ||||
Consideration paid | $ 30,000,000 | ||||||
Additional option to purchase common stock | shares | 600,000 | ||||||
Additional option to purchase common stock value per share | $ / shares | $ 16 | $ 16 | $ 16 | ||||
Number of shares restricted for sale | shares | 2,400,000 | 2,400,000 | 2,400,000 | ||||
Number of years restricted for selling common stock | 2 years | ||||||
Option exercising period | 2 years | ||||||
Percentage of outstanding common shares | 18.20% | 18.20% | 18.20% | ||||
Common stock investment value | $ 13,900,000 | $ 13,900,000 | $ 13,900,000 | ||||
Fair value of additional option to purchase common stock | 500,000 | 500,000 | 500,000 | ||||
Remaining consideration paid | 15,600,000 | ||||||
Change in fair value of common stock | 8,800,000 | 8,800,000 | |||||
Increase decrease in fair value of additional common stock | 1,000,000 | 1,000,000 | |||||
Fair value, investments | 22,700,000 | 22,700,000 | 22,700,000 | ||||
Option to purchase additional common stock, value | $ 1,500,000 | $ 1,500,000 | $ 1,500,000 | ||||
Arcturus | Maximum | |||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||
Number of disease targets | DiseaseTarget | 12 |
License and Research Agreemen_4
License and Research Agreements - Share of Collaboration Revenue Related to Crysvita (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Disaggregation Of Revenue [Line Items] | ||||
Collaboration and license revenue | $ 24,149 | $ 12,794 | $ 42,321 | $ 23,471 |
Collaboration and License | ||||
Disaggregation Of Revenue [Line Items] | ||||
Collaboration and license revenue | 19,247 | 10,519 | 33,485 | 19,881 |
Collaboration and License | Kyowa Kirin Collaboration | ||||
Disaggregation Of Revenue [Line Items] | ||||
Collaboration and license revenue | 19,179 | 1,577 | 33,133 | 1,592 |
Collaboration and License | Kyowa Kirin Collaboration | Profit Share Territory | ||||
Disaggregation Of Revenue [Line Items] | ||||
Collaboration and license revenue | 17,248 | 1,065 | 29,187 | 1,065 |
Collaboration and License | Kyowa Kirin Collaboration | Royalty Revenue in European Territory | ||||
Disaggregation Of Revenue [Line Items] | ||||
Collaboration and license revenue | $ 1,931 | $ 512 | $ 3,946 | $ 527 |
License and Research Agreemen_5
License and Research Agreements - Schedule of Cost Sharing Payments (Details) - Kyowa Kirin Collaboration - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Cost Sharing Payments [Line Items] | ||||
Research and development | $ 5,895 | $ 9,420 | $ 12,994 | $ 17,656 |
Selling, general and administrative | 5,163 | 4,068 | 10,376 | 7,850 |
Total | $ 11,058 | $ 13,488 | $ 23,370 | $ 25,506 |
Stock-Based Awards - Additional
Stock-Based Awards - Additional Information (Details) | 6 Months Ended |
Jun. 30, 2019shares | |
2014 Incentive Plan | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
Share reserved for future issuance | 2,582,049 |
Automatic increases in shares available for grant effective date | Jan. 1, 2015 |
Shares available for grant, ending date | Jan. 1, 2024 |
2014 Employee Stock Purchase Plan | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
Share reserved for future issuance | 2,740,058 |
Stock-Based Awards - Summary of
Stock-Based Awards - Summary of Stock-Based Compensation Expense (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||||
Stock-based compensation expense | $ 22,207 | $ 19,563 | $ 42,428 | $ 38,360 |
Cost of sales | ||||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||||
Stock-based compensation expense | 51 | 85 | ||
Research and development | ||||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||||
Stock-based compensation expense | 12,032 | 11,644 | 23,262 | 22,891 |
Selling, general and administrative | ||||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||||
Stock-based compensation expense | $ 10,124 | $ 7,919 | $ 19,081 | $ 15,469 |
Net Loss Per Share - Outstandin
Net Loss Per Share - Outstanding Shares of Common Stock Equivalents Excluded from Computation of Diluted Net Loss per Share (Details) - shares | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||||
Common stock equivalents excluded from computation of diluted net loss per share | 8,378,290 | 7,894,300 | 7,979,265 | 7,633,936 |
Options to Purchase Common Stock and Restricted Stock Units | ||||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||||
Common stock equivalents excluded from computation of diluted net loss per share | 8,220,733 | 7,735,231 | 7,825,615 | 7,479,526 |
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 | 149,700 | 149,700 | 149,700 |
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 | 7,857 | 9,369 | 3,950 | 4,710 |
Equity Transactions - Additiona
Equity Transactions - Additional Information (Details) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | 3 Months Ended | 6 Months Ended | |||
Feb. 28, 2019 | Jul. 31, 2017 | Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Stockholders Equity [Line Items] | ||||||
Net proceeds from sale of common stock | $ 330,415 | $ 270,969 | ||||
ATM Sales Agreement | ||||||
Stockholders Equity [Line Items] | ||||||
Option to sell common stock for cash | $ 150,000 | |||||
Common stock shares sold | 88,978 | 240,417 | 468,685 | 240,417 | ||
Net proceeds from sale of common stock | $ 5,500 | $ 11,800 | $ 24,800 | $ 11,800 | ||
Underwritten Public Offering | ||||||
Stockholders Equity [Line Items] | ||||||
Common stock shares sold | 5,833,333 | |||||
Net proceeds from sale of common stock | $ 330,400 | |||||
Shares purchased by underwriters | 760,869 | |||||
Public offering price | $ 60 |
Accumulated Other Comprehensi_3
Accumulated Other Comprehensive Income (Loss) - Schedule of Total Accumulated Other Comprehensive Income (Loss) (Details) - USD ($) $ in Thousands | Jun. 30, 2019 | Dec. 31, 2018 |
Accumulated Other Comprehensive Income Loss Net Of Tax [Abstract] | ||
Foreign currency translation adjustments | $ (174) | $ (329) |
Unrealized gain (loss) on securities available-for-sale | 429 | (304) |
Total accumulated other comprehensive income (loss) | $ 255 | $ (633) |
Leases - Additional Information
Leases - Additional Information (Details) $ in Millions | 3 Months Ended | 6 Months Ended |
Jun. 30, 2019USD ($) | Jun. 30, 2019USD ($) | |
Lessee Disclosure [Abstract] | ||
Operating lease extension details | multiple five-year renewal periods | |
Operating lease, existence of option to extend | true | |
Operating lease expiry term | 2028 | |
Cash paid for measurement of lease liabilities | $ 2.2 | $ 4.2 |
Weighted average remaining lease term | 5 years 11 months 23 days | 5 years 11 months 23 days |
Weighted average discount rate | 7.50% | 7.50% |
Leases - Summary of Components
Leases - Summary of Components of Lease Expense (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended |
Jun. 30, 2019 | Jun. 30, 2019 | |
Lease Cost [Abstract] | ||
Operating lease expense | $ 2,373 | $ 4,330 |
Variable lease expense | 669 | 1,321 |
Total lease expense | $ 3,042 | $ 5,651 |
Leases - Summary of Future Mini
Leases - Summary of Future Minimum Lease Payments under Non-cancellable Leases (Details) $ in Thousands | Jun. 30, 2019USD ($) |
Lessee Disclosure [Abstract] | |
2019 (remaining) | $ 4,724 |
2020 | 9,639 |
2021 | 7,561 |
2022 | 7,433 |
2023 | 7,541 |
Thereafter | 13,153 |
Total future lease payments | 50,051 |
Less: Amount representing interest | (10,084) |
Present value of future lease payments | 39,967 |
Less: Short-term lease liabilities | (6,999) |
Long-term lease liability | $ 32,968 |
Gain from Sale of Priority Re_2
Gain from Sale of Priority Review Voucher - Additional Information (Details) - USD ($) $ in Thousands | 1 Months Ended | 3 Months Ended | 6 Months Ended | |
Jun. 30, 2018 | Jan. 31, 2018 | Jun. 30, 2018 | Jun. 30, 2018 | |
Finite Lived Intangible Assets [Line Items] | ||||
Sale of PRV | $ 170,322 | |||
Gain from sale of PRV | $ 40,322 | $ 170,322 | ||
Mepsevii | ||||
Finite Lived Intangible Assets [Line Items] | ||||
Sale of PRV | $ 130,000 | |||
Crysvita | ||||
Finite Lived Intangible Assets [Line Items] | ||||
Sale of PRV | $ 80,600 |