Document and Entity Information
Document and Entity Information - shares | 6 Months Ended | |
Jun. 30, 2021 | Jul. 28, 2021 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Jun. 30, 2021 | |
Document Fiscal Year Focus | 2021 | |
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 | 67,836,060 | |
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, 2021 | Dec. 31, 2020 |
Current assets: | ||
Cash and cash equivalents | $ 290,875 | $ 713,526 |
Marketable debt securities | 554,881 | 488,007 |
Accounts receivable, net | 24,720 | 23,093 |
Inventory | 15,083 | 13,048 |
Prepaid expenses and other current assets | 63,933 | 57,630 |
Total current assets | 949,492 | 1,295,304 |
Property, plant, and equipment, net | 104,568 | 73,515 |
Equity investments | 103,710 | 155,375 |
Marketable debt securities | 128,031 | 10,506 |
Right-of-use assets | 38,619 | 40,524 |
Intangible assets, net | 130,950 | 131,113 |
Goodwill | 44,406 | 44,406 |
Other assets | 12,668 | 8,812 |
Total assets | 1,512,444 | 1,759,555 |
Current liabilities: | ||
Accounts payable | 21,790 | 12,923 |
Accrued liabilities | 91,060 | 108,491 |
Contract liabilities | 3,912 | 59,219 |
Lease liabilities | 10,305 | 8,976 |
Total current liabilities | 127,067 | 189,609 |
Contract liabilities | 7,349 | |
Lease liabilities | 35,861 | 39,251 |
Deferred tax liabilities | 33,306 | 33,306 |
Liability related to the sale of future royalties | 344,039 | 335,665 |
Total liabilities | 540,273 | 605,180 |
Stockholders’ equity: | ||
Preferred stock — 25,000,000 shares authorized; nil outstanding as of June 30, 2021 and December 31, 2020 | ||
Common stock — 250,000,000 shares authorized; 67,764,950 and 66,818,520 shares issued and outstanding as of June 30, 2021 and December 31, 2020, respectively | 68 | 67 |
Additional paid-in capital | 2,850,016 | 2,773,195 |
Accumulated other comprehensive income | 232 | 689 |
Accumulated deficit | (1,878,145) | (1,619,576) |
Total stockholders’ equity | 972,171 | 1,154,375 |
Total liabilities and stockholders’ equity | $ 1,512,444 | $ 1,759,555 |
CONDENSED CONSOLIDATED BALANC_2
CONDENSED CONSOLIDATED BALANCE SHEETS (Parenthetical) - shares | Jun. 30, 2021 | Dec. 31, 2020 |
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 | 67,764,950 | 66,818,520 |
Common stock, shares outstanding | 67,764,950 | 66,818,520 |
CONDENSED CONSOLIDATED STATEMEN
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2021 | Jun. 30, 2020 | Jun. 30, 2021 | Jun. 30, 2020 | |
Revenues: | ||||
Total revenues | $ 86,975 | $ 61,709 | $ 186,370 | $ 98,018 |
Operating expenses: | ||||
Cost of sales | 3,136 | 1,803 | 8,324 | (1,700) |
Research and development | 113,205 | 80,709 | 260,723 | 193,670 |
Selling, general and administrative | 53,410 | 42,252 | 106,668 | 89,768 |
Total operating expenses | 169,751 | 124,764 | 375,715 | 281,738 |
Loss from operations | (82,776) | (63,055) | (189,345) | (183,720) |
Interest income | 441 | 1,797 | 1,080 | 4,716 |
Change in fair value of equity investments | (31,046) | 95,200 | (51,665) | 102,868 |
Non-cash interest expense on liability related to the sale of future royalties | (8,517) | (8,429) | (16,935) | (16,511) |
Other income (expense) | (67) | 217 | (862) | (239) |
Income (loss) before income taxes | (121,965) | 25,730 | (257,727) | (92,886) |
Provision for income taxes | (463) | (415) | (842) | (824) |
Net income (loss) | $ (122,428) | $ 25,315 | $ (258,569) | $ (93,710) |
Net income (loss) per share: | ||||
Basic | $ (1.81) | $ 0.42 | $ (3.84) | $ (1.59) |
Diluted | $ (1.81) | $ 0.41 | $ (3.84) | $ (1.59) |
Weighted-average shares used in computing net income (loss) per share: | ||||
Basic | 67,607,752 | 59,995,617 | 67,356,443 | 58,996,278 |
Diluted | 67,607,752 | 61,146,231 | 67,356,443 | 58,996,278 |
Collaboration and License | ||||
Revenues: | ||||
Total revenues | $ 63,940 | $ 50,161 | $ 142,950 | $ 77,376 |
Product Sales | ||||
Revenues: | ||||
Total revenues | 18,346 | 8,066 | 34,859 | 14,545 |
Non-cash Collaboration Royalty Revenue | ||||
Revenues: | ||||
Total revenues | $ 4,689 | $ 3,482 | $ 8,561 | $ 6,097 |
CONDENSED CONSOLIDATED STATEM_2
CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE INCOME (LOSS) (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2021 | Jun. 30, 2020 | Jun. 30, 2021 | Jun. 30, 2020 | |
Statement Of Income And Comprehensive Income [Abstract] | ||||
Net income (loss) | $ (122,428) | $ 25,315 | $ (258,569) | $ (93,710) |
Other comprehensive income (loss): | ||||
Foreign currency translation adjustments | 103 | 89 | (182) | 40 |
Unrealized gain (loss) on available-for-sale securities | 73 | 2,740 | (275) | 1,391 |
Other comprehensive income (loss): | 176 | 2,829 | (457) | 1,431 |
Total comprehensive income (loss) | $ (122,252) | $ 28,144 | $ (259,026) | $ (92,279) |
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, 2019 | $ 653,764 | $ 58 | $ 2,086,863 | $ (147) | $ (1,433,010) |
Beginning balance, shares at Dec. 31, 2019 | 57,838,220 | ||||
Ending balance at Mar. 31, 2020 | 609,146 | $ 59 | 2,162,667 | (1,545) | (1,552,035) |
Ending balance, shares at Mar. 31, 2020 | 59,488,873 | ||||
Beginning balance at Dec. 31, 2019 | 653,764 | $ 58 | 2,086,863 | (147) | (1,433,010) |
Beginning balance, shares at Dec. 31, 2019 | 57,838,220 | ||||
Issuance of common stock in connection with license agreement, net of issuance costs | 55,268 | $ 1 | 55,267 | ||
Issuance of common stock in connection with license agreement, net of issuance costs, shares | 1,243,913 | ||||
Issuance of common stock in connection with at-the-market offering, net of issuance costs | 20,391 | 20,391 | |||
Issuance of common stock in connection with at-the-market offering, net of issuance costs, shares | 283,333 | ||||
Stock-based compensation | 42,585 | 42,585 | |||
Issuance of common stock upon exercise of warrants and under equity plan awards, net of tax | 15,858 | $ 1 | 15,857 | ||
Issuance of common stock upon exercise of warrants and under equity plan awards, net of tax, shares | 1,048,290 | ||||
Other comprehensive income (loss) | 1,431 | 1,431 | |||
Net income (loss) | (93,710) | (93,710) | |||
Ending balance at Jun. 30, 2020 | 695,587 | $ 60 | 2,220,963 | 1,284 | (1,526,720) |
Ending balance, shares at Jun. 30, 2020 | 60,413,756 | ||||
Beginning balance at Mar. 31, 2020 | 609,146 | $ 59 | 2,162,667 | (1,545) | (1,552,035) |
Beginning balance, shares at Mar. 31, 2020 | 59,488,873 | ||||
Issuance of common stock in connection with at-the-market offering, net of issuance costs | 20,391 | 20,391 | |||
Issuance of common stock in connection with at-the-market offering, net of issuance costs, shares | 283,333 | ||||
Stock-based compensation | 22,428 | 22,428 | |||
Issuance of common stock under equity plan awards, net of tax | 15,478 | $ 1 | 15,477 | ||
Issuance of common stock under equity plan awards, net of tax, shares | 641,550 | ||||
Other comprehensive income (loss) | 2,829 | 2,829 | |||
Net income (loss) | 25,315 | 25,315 | |||
Ending balance at Jun. 30, 2020 | 695,587 | $ 60 | 2,220,963 | 1,284 | (1,526,720) |
Ending balance, shares at Jun. 30, 2020 | 60,413,756 | ||||
Beginning balance at Dec. 31, 2020 | $ 1,154,375 | $ 67 | 2,773,195 | 689 | (1,619,576) |
Beginning balance, shares at Dec. 31, 2020 | 66,818,520 | 66,818,520 | |||
Stock-based compensation | $ 51,319 | 51,319 | |||
Issuance of common stock under equity plan awards, net of tax | 25,503 | $ 1 | 25,502 | ||
Issuance of common stock under equity plan awards, net of tax, shares | 946,430 | ||||
Other comprehensive income (loss) | (457) | (457) | |||
Net income (loss) | (258,569) | (258,569) | |||
Ending balance at Jun. 30, 2021 | $ 972,171 | $ 68 | 2,850,016 | 232 | (1,878,145) |
Ending balance, shares at Jun. 30, 2021 | 67,764,950 | 67,764,950 | |||
Beginning balance at Mar. 31, 2021 | $ 1,054,582 | $ 67 | 2,810,176 | 56 | (1,755,717) |
Beginning balance, shares at Mar. 31, 2021 | 67,439,477 | ||||
Stock-based compensation | 27,099 | 27,099 | |||
Issuance of common stock under equity plan awards, net of tax | 12,742 | $ 1 | 12,741 | ||
Issuance of common stock under equity plan awards, net of tax, shares | 325,473 | ||||
Other comprehensive income (loss) | 176 | 176 | |||
Net income (loss) | (122,428) | (122,428) | |||
Ending balance at Jun. 30, 2021 | $ 972,171 | $ 68 | $ 2,850,016 | $ 232 | $ (1,878,145) |
Ending balance, shares at Jun. 30, 2021 | 67,764,950 | 67,764,950 |
CONDENSED CONSOLIDATED STATEM_4
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS (Unaudited) - USD ($) $ in Thousands | 6 Months Ended | |
Jun. 30, 2021 | Jun. 30, 2020 | |
Operating activities: | ||
Net loss | $ (258,569) | $ (93,710) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Stock-based compensation | 51,345 | 42,558 |
Amortization of premium (discount) on marketable debt securities, net | 2,511 | (149) |
Depreciation and amortization | 6,488 | 6,036 |
Change in fair value of equity investments | 51,665 | (102,868) |
Non-cash collaboration royalty revenue | (8,561) | (6,097) |
Non-cash interest expense on liability related to the sale of future royalties | 16,935 | 16,511 |
Other | 511 | (14) |
Changes in operating assets and liabilities: | ||
Accounts receivable | (1,669) | 9,482 |
Inventory | (2,174) | 521 |
Prepaid expenses and other assets | (9,002) | (6,136) |
Accounts payable, accrued, and other liabilities | (11,526) | (8,833) |
Contract liabilities | (62,656) | 134,898 |
Net cash used in operating activities | (224,702) | (7,801) |
Investing activities: | ||
Purchase of property, plant, and equipment | (36,485) | (18,179) |
Purchase of marketable debt securities | (664,347) | (456,331) |
Purchase of equity investments | (9,600) | |
Proceeds from sale of marketable debt securities | 70,548 | 28,850 |
Proceeds from maturities of marketable debt securities | 406,614 | 273,996 |
Net cash used in investing activities | (223,670) | (181,264) |
Financing activities: | ||
Proceeds from the issuance of common stock in connection with the license agreement, net | 55,268 | |
Proceeds from the issuance of common stock in connection with at-the-market offering, net | 20,391 | |
Proceeds from the issuance of common stock from exercise of warrants and equity plan awards, net | 25,503 | 15,858 |
Other | (226) | (91) |
Net cash provided by financing activities | 25,277 | 91,426 |
Effect of exchange rate changes on cash | (323) | (198) |
Net decrease in cash, cash equivalents and restricted cash | (423,418) | (97,837) |
Cash, cash equivalents and restricted cash at beginning of period | 726,294 | 436,244 |
Cash, cash equivalents and restricted cash at end of period | 302,876 | 338,407 |
Supplemental disclosures of non-cash information: | ||
Acquired lease liabilities arising from obtaining right-of-use assets | $ 2,301 | $ 10,403 |
Organization
Organization | 6 Months Ended |
Jun. 30, 2021 | |
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 operates as one reportable segment. The Company has three commercially approved products. Crysvita® (burosumab) is approved in the United States by the U.S. Food and Drug Administration (FDA) and in Canada for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients one year of age and older, and is approved in the European Union (EU) and the United Kingdom, for the treatment of XLH with radiographic evidence of bone disease in children one year of age and older, adolescents, and adults. In Brazil, Colombia, and Mexico, Crysvita is approved for treatment of XLH in adult and pediatric patients one year of age and older. Crysvita is also approved in the United States by the FDA for the treatment of fibroblast growth factor 23 (FGF23)-related hypophosphatemia in tumor-induced osteomalacia (TIO), associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized in adults and pediatric patients 2 years of age and older. The Company has also received FDA approval for Mepsevii ® Dojolvi ® In addition to the approved products, the Company has the following ongoing clinical development programs: • • • • GTX-102 is an antisense oligonucleotide (ASO), which the Company is collaborating on the development with GeneTx Biotherapeutics LLC (GeneTx) for the treatment of Angelman syndrome, a debilitating and rare neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene; • UX701 is an AAV type 9 gene therapy designed to deliver stable expression of a truncated version of the ATP7B copper transporter following a single intravenous infusion to improve copper distribution and excretion from the body and reverse pathological findings of Wilson liver disease; and • UX053 is a messenger RNA (mRNA) product candidate designed for the treatment of patients with Glycogen Storage Disease Type III (GSDIII), a disease caused by a glycogen debranching enzyme (AGL) deficiency that results in glycogen accumulation in the liver and muscle. 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 that the Company will need to raise additional capital to fully implement its business plans. Through June 30, 2021, the Company has relied primarily on its sale of equity securities, its revenues from commercial products, its sale of future royalties, and strategic collaboration arrangements, to finance its operations. The Company will likely 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 would need to reevaluate its operating plans. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 6 Months Ended |
Jun. 30, 2021 | |
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 12, 2021 with the United States Securities and Exchange Commission (SEC). The results of operations for the three and six months ended June 30, 2021 are not necessarily indicative of the results to be expected for the year ending December 31, 2021. The Condensed Consolidated Balance Sheet as of December 31, 2020 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, stock-based compensation, and the liability related to the sale of future royalties. 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 used as collateral for the Company’s obligations under its facility leases and the gene therapy building construction project. 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, 2021 2020 Cash and cash equivalents $ 290,875 $ 335,639 Restricted cash included in prepaid expenses and other current assets 10,000 847 Restricted cash included in other assets 2,001 1,921 Total cash, cash equivalents, and restricted cash shown in the statements of cash flows $ 302,876 $ 338,407 Credit Losses The Company is exposed to credit losses primarily through receivables from customers and collaborators and through its available-for-sale debt securities. For trade receivables and other instruments, the Company uses a new forward-looking expected loss model that generally results in the earlier recognition of allowances for losses. For available-for-sale debt securities with unrealized losses, the losses are recognized as allowances rather than as reductions in the amortized cost of the securities. The Company’s expected loss allowance methodology for the receivables is developed using historical collection experience, current and future economic market conditions, a review of the current aging status and financial condition of the entities. Specific allowance amounts are established to record the appropriate allowance for customers that have a higher probability of default. Balances are written off when determined to be uncollectible. The Company’s expected loss allowance methodology for the debt securities is developed by reviewing the extent of the unrealized loss, the size, term, geographical location, and industry of the issuer, the issuers’ credit ratings and any changes in those ratings, as well as reviewing current and future economic market conditions and the issuers’ current status and financial condition. The Company considered the current and expected future economic and market conditions surrounding the novel coronavirus (COVID-19) pandemic and determined that the estimate of credit losses was not significantly impacted. There was no material allowance recorded for losses on receivables and available-for-sale debt securities which were attributable to credit risk for the three and six months ended June 30, 2021 and 2020. 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. 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 Company sold the right to receive certain royalty payments from net sales of Crysvita to RPI Finance Trust (RPI), an affiliate of Royalty Pharma, as further described in Note 7. The Company records the royalty revenue from the net sales of Crysvita in the applicable European territories on a prospective basis as non-cash royalty revenue in the Consolidated Statements of Operations over the term of the arrangement. The terms of the Company’s collaboration and license 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 obligations and recognizes revenue by measuring the progress towards complete satisfaction of the performance obligations using input measures. 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. The Company’s estimates of government mandated rebates, chargebacks, estimated product returns, and other deductions depends on the identification of key customer contract terms and conditions, as well as estimates of sales volumes to different classes of payors. |
Financial Instruments
Financial Instruments | 6 Months Ended |
Jun. 30, 2021 | |
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 Company determines the fair value of its equity investments in Arcturus Therapeutics Holdings Inc. (Arcturus) Solid Biosciences Inc. (Solid) 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, 2021 Level 1 Level 2 Level 3 Total Money market funds $ 206,674 $ — $ — $ 206,674 Certificate of deposits and time deposits — 17,501 — 17,501 Corporate bonds — 237,296 — 237,296 Commercial paper — 341,522 — 341,522 Asset-backed securities — 55,020 — 55,020 U.S. Government Treasury and agency securities 12,500 42,109 — 54,609 Debt securities in government-sponsored entities — 28,751 — 28,751 Investments in Arcturus and Solid common stock 103,091 — — 103,091 Total $ 322,265 $ 722,199 $ — $ 1,044,464 December 31, 2020 Level 1 Level 2 Level 3 Total Money market funds $ 598,392 $ — $ — $ 598,392 Time deposits — 10,000 — 10,000 Corporate bonds — 193,802 — 193,802 Commercial paper — 173,859 — 173,859 Asset-backed securities — 11,225 — 11,225 U.S. Government Treasury and agency securities 167,967 17,661 — 185,628 Investments in Arcturus and Solid common stock 154,756 — — 154,756 Total $ 921,115 $ 406,547 $ — $ 1,327,662 In July 2020, the Company invested $2.5 million in a private diagnostic company, in the form of a convertible promissory note that matures in two years, if not converted earlier. The Company was also issued a warrant to purchase up to $1.0 million of the entity’s preferred stock. The fair value of the warrant to purchase shares of the entity was based on unobservable inputs that are significant to the measurement of the fair value of the asset and is supported by little or no market data; accordingly, the warrant is considered a Level 3 financial asset and is remeasured on a nonrecurring basis. The Company measured the fair value of the warrant by applying the Black-Scholes option pricing method and utilizing the following inputs: stock price, strike price, volatility, risk-free interest rate, and expected term. The Company recognizes the interest income on the convertible promissory note based on the effective interest rate method over the life of the note. As of June 30, 2021, the balance of the convertible promissory note was $2.2 million, including $0.4 million in interest receivable, and was recorded in other assets, In December 2020, the Company invested $1.4 million in Mazi Therapeutics, Inc. (Mazi), a private pharmaceutical company founded by a current employee of the Company, in the form of a convertible promissory note that matures in two years, if not converted earlier. As of June 30, 2021, the balance of the convertible promissory note was $1.4 million. |
Balance Sheet Components
Balance Sheet Components | 6 Months Ended |
Jun. 30, 2021 | |
Investments Debt And Equity Securities [Abstract] | |
Balance Sheet Components | 4. Balance Sheet Components Cash Equivalents and Investments The fair values of cash equivalents and investments classified as available-for-sale securities consisted of the following (in thousands): June 30, 2021 Gross Unrealized Amortized Cost Gains Losses Estimated Fair Value Money market funds $ 206,674 $ — $ — $ 206,674 Certificate of deposits and time deposits 17,501 — — 17,501 Corporate bonds 237,310 45 (59 ) 237,296 Commercial paper 341,522 — — 341,522 Asset-backed securities 55,015 11 (6 ) 55,020 U.S. Government Treasury and agency securities 54,605 7 (3 ) 54,609 Debt securities in government-sponsored entities 28,761 4 (14 ) 28,751 Total $ 941,388 $ 67 $ (82 ) $ 941,373 December 31, 2020 Gross Unrealized Amortized Cost Gains Losses Estimated Fair Value Money market funds $ 598,392 $ — $ — $ 598,392 Time deposits 10,000 — — 10,000 Corporate bonds 193,610 209 (17 ) 193,802 Commercial paper 173,859 — — 173,859 Asset-backed securities 11,224 1 — 11,225 U.S. Government Treasury and agency securities 185,561 67 — 185,628 Total $ 1,172,646 $ 277 $ (17 ) $ 1,172,906 At June 30, 2021, the remaining contractual maturities of available-for-sale securities were less than five years. There have been no significant realized gains or losses on available-for-sale securities for the three and six months ended June 30, 2021 and 2020. All marketable securities with unrealized losses at have been in a loss position for less than twelve months and were temporary in nature. Inventory Inventory consists of the following (in thousands): June 30, December 31, 2021 2020 Work-in-process $ 9,457 $ 7,184 Finished goods 5,626 5,864 Total inventory $ 15,083 $ 13,048 Accrued Liabilities Accrued liabilities consist of the following (in thousands): June 30, December 31, 2021 2020 Research, clinical study, and manufacturing expenses $ 22,551 $ 25,875 Payroll and related expenses 41,545 58,176 Other 26,964 24,440 Total accrued liabilities $ 91,060 $ 108,491 |
Revenue
Revenue | 6 Months Ended |
Jun. 30, 2021 | |
Revenue From Contract With Customer [Abstract] | |
Revenue | 5. Revenue The following table disaggregates total revenues from customers (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2021 2020 2021 2020 Collaboration and license revenue: Crysvita collaboration revenue in profit- share territory $ 41,756 $ 29,806 $ 78,016 $ 57,021 Crysvita royalty revenue in European territory 228 1,498 228 1,498 Daiichi Sankyo 21,956 18,857 64,706 18,857 Total collaboration and license revenue 63,940 50,161 142,950 77,376 Product sales: Crysvita 2,900 2,549 8,772 4,159 Mepsevii 5,399 4,185 9,006 7,610 Dojolvi 10,047 1,332 17,081 2,776 Total product sales 18,346 8,066 34,859 14,545 Crysvita non-cash collaboration royalty revenue 4,689 3,482 8,561 6,097 Total revenues $ 86,975 $ 61,709 $ 186,370 $ 98,018 The following table disaggregates total revenues based on geographic location (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2021 2020 2021 2020 North America $ 75,567 $ 51,931 $ 163,309 $ 82,288 Europe 6,700 6,548 12,337 10,705 Latin America 4,708 3,230 10,724 5,025 Total revenues $ 86,975 $ 61,709 $ 186,370 $ 98,018 The following table presents changes in the contract liabilities (in thousands): Six Months Ended June 30, 2021 2020 Balance of contract liabilities at beginning of period $ 66,568 $ — Additions 2,050 153,755 Deductions (64,706 ) (18,857 ) Balance of contract liabilities at end of period $ 3,912 $ 134,898 See Note 6 for additional details on contract liabilities activities. The Company’s largest accounts receivable balance accounted for 58% and 71% of the total accounts receivable balance as of June 30, 2021 and December 31, 2020, respectively, and was due from a collaboration partner. |
License and Research Agreements
License and Research Agreements | 6 Months Ended |
Jun. 30, 2021 | |
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, formerly Kyowa Hakko Kirin Co., Ltd. or KHK). Under the terms of this collaboration and license agreement, as amended, the Company and KKC 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, United Kingdom, and Switzerland, or the European territory, and the Company has 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 May 2017, the Company entered into an agreement with a wholly-owned subsidiary of KKC pursuant to which it was granted the right to commercialize Crysvita in Turkey. Development Activities In the field of orphan diseases, and except for ongoing studies being conducted by KKC, the Company is the lead party for development activities in the profit-share territory and in the European territory until the applicable transition date. The Company shares 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. In April 2023, which is the transition date for the profit-share 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 and United Kingdom 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 April 2023. Under the collaboration agreement, KKC manufactures and supplies Crysvita for commercial use in the profit-share territory and charges the Company the transfer price of 35% of net sales through December 31, 2022, and 30% thereafter. The remaining profit or loss after supply costs from commercializing products in the profit-share territory are shared between the Company and KKC on a 50/50 basis until April 2023. Thereafter, the Company will be entitled to receive a tiered double-digit revenue share in the mid-to-high 20% range. As KKC is the principal in the sale transaction with the customer, the Company recognizes a pro-rata share of collaboration revenue, net of transfer pricing, 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. In December 2019, the Company sold its right to receive royalty payments based on sales in the European territory to Royalty Pharma, effective January 1, 2020, as further described in Note 7. Prior to the Company’s sale of the royalty, the Company received a royalty of up to 10% on net sales in the European territory, which was recognized as the underlying sales occur. Beginning in 2020, the Company recorded the royalty revenue as non-cash royalty revenues. During the three months ended June 30, 2021 and 2020, there was a change in the estimate of the revenue reserves related to sales made prior to January 1, 2020, as a result of which, the Company recorded $0.2 million and $1.5 million, respectively, as royalty revenue in European territory. The Company’s Three Months Ended June 30, Six Months Ended June 30, 2021 2020 2021 2020 Company's share of revenue in profit- share territory $ 41,756 $ 29,806 $ 78,016 $ 57,021 Royalty revenue in European territory 228 1,498 228 1,498 Non-cash royalty revenue in European territory 4,689 3,482 8,561 6,097 Total $ 46,673 $ 34,786 $ 86,805 $ 64,616 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 these territories 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. The Company recorded product sales of $2.9 million and $8.8 million for the three and six months ended June 30, 2021, respectively, and $2.5 million and $4.2 million for the three and six months ended June 30, 2020, respectively, net of estimated product returns and other deductions. KKC has the option to assume responsibility for commercialization efforts in Turkey from the Company, after a certain minimum period. Under KKC manufactures and supplies for sales in the above territories and is based on 35% of the net sales through December 31, 2022 and 30% thereafter. The Company also pays to KKC a low single-digit royalty on net sales in Latin America Cost sharing payments Under the collaboration agreement, KKC and the Company share certain development and commercialization costs. Three Months Ended June 30, Six Months Ended June 30, 2021 2020 2021 2020 Research and development $ 5,171 $ 5,168 $ 11,356 $ 10,658 Selling, general and administrative 7,576 5,965 15,071 13,017 Total $ 12,747 $ 11,133 $ 26,427 $ 23,675 Collaboration receivable and payable The Company had accounts receivable from KKC in the amount of $14.7 million and $16.4 million from profit-share revenue and royalties and other receivables recorded in prepaid and other current assets of $3.8 million and $9.6 million and accrued liabilities of $2.7 million and $2.4 million from commercial and development activity reimbursements, as of June 30 , 2021 Bayer HealthCare LLC The Company has an agreement with Bayer Healthcare LLC (Bayer) to research, develop and commercialize AAV gene therapy products for the 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 has worldwide rights to commercialize the potential future product. Bayer was responsible for funding 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, 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. The Company has no further obligations under the contract. The Company may record future milestone payments as revenue, if it becomes probable that a significant reversal in the amount of revenue recognized will not occur and when the uncertainty associated with the variable consideration is subsequently resolved. Arcturus In October 2015, the Company entered into a Research Collaboration and License Agreement with Arcturus whereby the Company and Arcturus would collaborate on the research and development of therapies for select rare diseases. Arcturus has the primary responsibility for conducting certain research services, funded by the Company, and the Company is responsible for development and commercialization costs. On a product-by-product basis, the Company may be obligated to make development and regulatory milestone payments of up to $24.5 million, and commercial milestone payments of up to $45.0 million, if certain milestones are achieved. The Company may also be obligated to pay Arcturus royalties on any net sales of products incorporating the licensed intellectual property that range from a mid single-digit to low double-digit percentage. For the three and six months ended June 30, 2021 and 2020, there were no research and development expenses incurred for the funding of certain research services received from Arcturus. In May 2020, the Company exercised an option to purchase 600,000 shares of Arcturus’ common stock at $16.00 per share, or a total purchase price of $9.6 million. In December 2020, the Company sold 800,000 shares of Arcturus’ common stock at a weighted-average price of $100.81 per share and received net proceeds of $79.8 million. The Company has elected to apply the fair value option to account for the equity investment in Arcturus. As of June 30 The changes in the fair value of the Company’s equity investment in Arcturus securities were as follows (in thousands): Arcturus common stock Option to purchase additional shares of Arcturus common stock Total December 31, 2019 $ 26,088 $ 1,664 $ 27,752 Change in fair value 113,978 23,948 137,926 Transfer of value upon option exercise 35,212 (25,612 ) 9,600 Sale of shares (79,842 ) — (79,842 ) December 31, 2020 95,436 — 95,436 Change in fair value (20,988 ) — (20,988 ) June 30, 2021 $ 74,448 $ — $ 74,448 The Company recorded $16.4 million and $21.0 million in unrealized losses for the three and six months ended June 30, 2021, respectively, and $95.2 million and $102.9 million in unrealized gains for the three and six months ended June 30, 2020, respectively, from the fair value adjustments on the equity investment in Arcturus securities. GeneTx In August 2019, the Company entered into a Program Agreement and a Unitholder Option Agreement with GeneTx to collaborate on the development of GeneTx’s GTX-102, an ASO for the treatment of Angelman syndrome. Pursuant to the terms of the Unitholder Option Agreement, the Company made an upfront payment of $20.0 million for an exclusive option to acquire GeneTx, which was exercisable any time prior to 30 days following FDA acceptance of the IND for GTX-102. Pursuant to the agreement, upon acceptance of the IND, which occurred in January 2020, the Company elected to extend the option period by paying an option extension payment of $ 25.0 million (option extension premium) during the quarter ended March 31, 2020. The Company has the right to acquire GeneTx for a payment of $ 125.0 million, at any time, until the earlier of 30 months from the first dosing of a patient in a planned Phase 1/2 study (subject to extensions) or 90 days after results are available from that study. This exclusive option to acquire GeneTx can be extended under certain circumstances, by up to four additional three-month periods, by paying an additional extension fee for each three-month period. During the exclusive option period, GeneTx is responsible for conducting the program based on the development plan agreed between the parties and, subject to the terms in the Program Agreement, has the decision-making authority on all matters in connection with the research, development, manufacturing and regulatory activities with respect to the Program. The Company will provide support, at its discretion, including strategic guidance and clinical expertise. The Company and GeneTx will collaborate on the management of the Phase 1/2 study in patients with Angelman syndrome. If the Company acquires GeneTx, the Company will then be responsible for all development and commercialization activities from the date of acquisition. The Company would also be required to make payments upon achievement of certain development and commercial milestones, as well as royalties, depending upon the success of the program. Although GeneTx is a variable interest entity, the Company is not the primary beneficiary as it currently does not have the power to direct the activities that would most significantly impact the economic performance of GeneTx. Prior to product regulatory approval, all consideration paid to GeneTx represents rights to potential future benefits associated with GeneTx’s in-process research and development activities, which have not reached technological feasibility and have no alternative future use. Accordingly, for the three months ended March 31, 2020, the Company recorded the option extension payment of $25.0 million as an in-process research and development expense. REGENXBIO, Inc. In March 2020, the Company executed a License Agreement with REGENXBIO, Inc. (REGENEX), for an exclusive, sublicensable, worldwide license to REGENX’s NAV AAV8 and AAV9 Vectors for the development and commercialization of gene therapy treatments for a rare metabolic disorder. In return for these rights, the Company made an upfront payment of $7.0 million, which was recorded as an in-process research and development expense during the three months ended March 31, 2020. The Company will pay certain annual fees of $0.1 million, milestone payments of up to $14.0 million, and royalties on any net sales of products incorporating the licensed intellectual property that range from a high single-digit to low double-digit royalty Daiichi Sankyo In March 2020, the Company executed a License and Technology Access Agreement (the License Agreement) with Daiichi Sankyo Co., Ltd. (Daiichi Sankyo). Pursuant to the License Agreement, the Company granted Daiichi Sankyo a non-exclusive license to intellectual property, including know-how and patent applications, with respect to its HeLa PCL and HEK293 transient transfection manufacturing technology platforms for AAV-based gene therapy products. The Company retains the exclusive right to use the manufacturing technology for its current target indications and additional indications identified now and in the future. The Company will provide certain technical assistance and technology transfer services during the technology transfer period of three years to enable Daiichi Sankyo to use the technologies for its internal gene therapy programs. Daiichi Sankyo has an option to extend the technology transfer period including know-how improvements by two additional one-year periods by paying a fixed amount for each additional year. Daiichi Sankyo will be responsible for the manufacturing, development, and commercialization of products manufactured with the licensed technology; however, the Company has the option to co-develop and co-commercialize rare disease products at the IND stage. Ultragenyx may also provide strategic consultation to Daiichi Sankyo on the development of both AAV-based gene therapy products and other products for rare diseases. Under the terms of the License Agreement, Daiichi Sankyo made an upfront payment of $125.0 million and will pay an additional $25.0 million upon completion of the technology transfer of the HeLa PCL and HEK293 platforms, as well as single-digit royalties on net sales of products manufactured in either system. Daiichi Sankyo will reimburse the Company for all costs associated with the transfer of the manufacturing technology. The Company also entered into a Stock Purchase Agreement (SPA) with Daiichi Sankyo, pursuant to which Daiichi Sankyo purchased 1,243,913 The fair market value of the common stock issued to Daiichi Sankyo was $55.3 million based on the stock price of $44.43 per share on the date of issuance, resulting in a $19.7 million premium on the SPA. In June 2020, the Company executed a subsequent license agreement (the Sublicense Agreement) with Daiichi Sankyo for transfer of certain technology in consideration for an upfront payment of $8.0 million and annual maintenance fees, The License Agreement, the Sublicense Agreement, and the SPA are being accounted for as one arrangement because they were entered into at or near the same time and negotiated in contemplation of one another. The Company evaluated the License Agreement and the Sublicense Agreement under ASC 606 and determined that the performance obligations under the agreements are (i) intellectual property with respect to its HeLa PCL and HEK293 transient transfection manufacturing technology platforms together with the initial technical assistance and technology transfer services, which are expected to be completed in the fourth quarter of 2021, and (ii) the transfer of any know-how and improvements after the completion of the initial technology transfer through the end of the three year technology transfer period ending March 2023. As of June 30, 2021, the Company has determined that the total transaction price of the License Agreement was $183.6 million which was comprised of the $19.7 million premium from the SPA, the $125.0 million upfront payment, the $25.0 million in unconstrained milestone payments, $8.0 million from the Sublicense Agreement, and the $5.9 million estimated reimbursement amount for delivering the license and technology services The Company allocated the total transaction price to the two performance obligations on a relative stand-alone selling price basis. Revenue allocated to the intellectual property and the technology transfer services will be recognized over an initial period which is estimated to end in the fourth quarter of 2021, measuring the progress toward complete satisfaction of the individual performance obligation using an input measure. Revenue for know-how and improvements after the completion of technology transfer will be recognized on a straight-line basis over the remaining technology transfer period, which ends in March 2023, as it is expected that Daiichi Sankyo will receive and consume the benefits consistently throughout the period. The estimated period to complete the technology transfer services and the related milestones payments, if any, are subject to revised estimates which could be impacted by limitations or delays from the COVID-19 pandemic, successful scale-up of the manufacturing, and other changes that may impact timing. Royalties from commercial sales will be accounted for as revenue upon achievement of such sales, assuming all other revenue recognition criteria are met. The Company recognized $22.0 million and $64.7 million for the three and six months ended June 30 Accordingly, the Company had recorded $3.9 million as contract liabilities, net, as of . Solid Biosciences, Inc. In October 2020, the Company entered into received an exclusive license for any pharmaceutical product that expresses Solid’s proprietary microdystrophin construct from AAV8 and variants thereof in clade E for use in the treatment of Duchenne muscular dystrophy and other diseases resulting from lack of functional dystrophin, including Becker muscular dystrophy. The Company will collaborate to develop products that combine Solid’s differentiated microdystrophin construct, the Company’s HeLa PCL manufacturing platform, and the Company’s AAV8 variants. Solid will also provide development support and was granted an exclusive option to co-invest in products the Company develops for profit share participation in certain territories. On a product-by-product basis, the Company may be obligated to make development milestone payments of up to $25.0 million, regulatory milestone payments of up to $65.0 million, and commercial milestone payments of up to $165.0 million, if such milestones are achieved, as well as royalties on any net sales of products incorporating the licensed intellectual property that range from a low to mid-double-digit percentage. The royalty rate changes to mid to high double-digit percentage if Solid decides to co-invest in the product. The Company also entered into a Stock Purchase Agreement and the Investor Agreement with Solid, pursuant to which, the Company purchased 7,825,797 and (iii) certain other specified events. The Company also agreed to customary standstill restrictions in accordance with the terms of the Investor Agreement until the earlier of (a) 24 months after the closing of the transaction and (b) certain specified events. The Company’s investment in Solid is being accounted at fair value, as the fair value is readily determinable. The Company recorded the common stock investment at $26.8 million on the transaction date, which was based on the quoted market price on the closing date. Although Solid is a variable interest entity, the Company is not the primary beneficiary as it does not have the power to direct the activities that would most significantly impact the economic performance of Solid. Prior to the achievement of certain development milestones, all consideration paid to Solid represents rights to potential future benefits associated with Solid’s in-process research and development activities, which have not reached technological feasibility and have no alternative future use. Accordingly, the remaining $13.2 million of the total $40.0 million paid as consideration was attributed to the license rights obtained and was recorded as in-process research and development expense during the year ended December 31, 2020. The changes in the fair value of the Company’s investment in Solid’s common stock were as follows (in thousands): Solid common stock Acquisition of investment in Solid common stock in October 2020 $ 26,843 Change in fair value 32,477 December 31, 2020 59,320 Change in fair value (30,677 ) June 30, 2021 $ 28,643 The Company recorded $14.6 million and $30.7 million in unrealized losses for the three and six months ended June 30, 2021, respectively, from the fair value adjustments on the equity investment in Solid securities. Mereo BioPharma 3 Limited In December 2020, the Company entered into a License and Collaboration Agreement with Mereo BioPharma 3 Limited, or Mereo, to collaborate on the development of setrusumab. Under the terms of the agreement, the Company will lead future global development of setrusumab in both pediatric and adult patients with Osteogenesis Imperfecta, or OI. The Company was granted an exclusive license to develop and commercialize setrusumab in the U.S., Turkey, and the rest of the world, excluding the European Economic Area, United Kingdom, and Switzerland, or the Mereo territory, where Mereo retains commercial rights. Each party will be responsible for post-marketing commitments and commercial supply in their respective territories. Upon the closing of the transaction in January 2021, the Company made a payment of $50.0 million to Mereo and will be required to make payments of up to $254.0 million upon the achievement of certain clinical, regulatory, and commercial milestones. The Company will pay for all global development costs as well as tiered double-digit percentage royalties to Mereo on net sales in the U.S., Turkey, and the rest of the world (excluding the Mereo Territory), and Mereo will pay the Company a fixed double-digit percentage royalty on net sales in the Mereo Territory. Although Mereo is a variable interest entity, the Company is not the primary beneficiary as it does not have the power to direct the activities that would most significantly impact the economic performance of Mereo. Prior to the achievement of certain development milestones, all consideration paid to Mereo represents rights to potential future benefits associated with Mereo’s in-process research and development activities, which have not reached technological feasibility and have no alternative future use. Accordingly, for the three months ended March 31, 2021, the Company recorded the upfront payment of $50.0 million as in-process research and development expense. |
Liability Related to the Sale o
Liability Related to the Sale of Future Royalties | 6 Months Ended |
Jun. 30, 2021 | |
Deferred Revenue Disclosure [Abstract] | |
Liability Related to the Sale of Future Royalties | 7. Liability Related to the Sale of Future Royalties In December 2019, the Company entered into a Royalty Purchase Agreement with RPI. Pursuant to the agreement, RPI paid $320.0 million to the Company in consideration for the right to receive royalty payments effective January 1, 2020, arising from the net sales of Crysvita in the European Union, the United Kingdom, Proceeds from the transaction were recorded as a liability (liability related to sale of future royalties on the Consolidated Balance Sheets). The Company amortizes $320.0 million, net of transaction cost of $5.8 million using the effective interest method over the estimated life of the arrangement. In order to determine the amortization of the liability, the Company is required to estimate the total amount of future royalty payments to be received by the Company and paid to RPI, subject to the capped amount, over the life of the arrangement. The excess of future estimated royalty payments (subject to the capped amount), over the $314.2 million of net proceeds, is recorded as non-cash interest expense over the life of the arrangement. Consequently, the Company estimates an imputed interest on the unamortized portion of the liability and records interest expense relating to the transaction. The Company records the royalty revenue arising from the net sales of Crysvita in the applicable European territories as non-cash royalty revenue in the Consolidated Statements of Operations over the term of the arrangement. The Company periodically assesses the expected royalty payments using a combination of historical results, internal projections and forecasts from external sources. To the extent such payments are greater or less than the Company’s initial estimates or the timing of such payments is materially different than its original estimates, the Company will prospectively adjust the amortization of the liability and the effective interest rate. The Company’s effective annual interest rate was approximately 9.8% for the three months ended June 30 There are a number of factors that could materially affect the amount and timing of royalty payments from KKC in the applicable European territories, most of which are not within the Company’s control. Such factors include, but are not limited to, the success of KKC’s sales and promotion of Crysvita, changing standards of care, delays or disruptions related to the COVID-19 pandemic, the introduction of competing products, pricing for reimbursement in various European territories, manufacturing or other delays, intellectual property matters, adverse events that result in governmental health authority imposed restrictions on the use of Crysvita, significant changes in foreign exchange rates as the royalty payments are made in U.S. dollars (USD) while significant portions of the underlying European sales of Crysvita are made in currencies other than USD, and other events or circumstances that could result in reduced royalty payments from European sales of Crysvita , all of which would result in a reduction of non-cash royalty revenue and the non-cash interest expense over the life of the arrangement. Conversely, if sales of Crysvita in Europe are more than expected, the non-cash royalty revenue and the non-cash interest expense recorded by the Company would be greater over the term of the arrangement. The following table shows the activity within the liability account (in thousands): Liability related to the sale of future royalties December 31, 2019 $ 315,369 Non-cash collaboration royalty revenue (12,995 ) Non-cash interest expense 33,291 December 31, 2020 335,665 Non-cash collaboration royalty revenue (8,561 ) Non-cash interest expense 16,935 June 30, 2021 $ 344,039 |
Stock-Based Awards
Stock-Based Awards | 6 Months Ended |
Jun. 30, 2021 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Stock-Based Awards | 8 . 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, 2021, there were 4,103,718 shares reserved under the 2014 Plan for the future issuance of equity awards, 3,956,907 shares reserved for the 2014 Employee Stock Purchase Plan, and 488,223 shares reserved for the Employment Inducement 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, 2021 2020 2021 2020 Cost of sales $ 223 $ 112 $ 602 $ 180 Research and development 15,094 12,856 28,583 23,785 Selling, general and administrative 11,825 9,441 22,255 18,616 Total stock-based compensation expense $ 27,142 $ 22,409 $ 51,440 $ 42,581 |
Net Income (Loss) Per Share
Net Income (Loss) Per Share | 6 Months Ended |
Jun. 30, 2021 | |
Earnings Per Share [Abstract] | |
Net Income (Loss) Per Share | 9 . Net Income (Loss) Per Share Basic net income (loss) per share has been computed by dividing the net income (loss) by the weighted-average number of shares of common stock outstanding during the period. Diluted net income (loss) per share is calculated by dividing net income (loss) by the weighted-average number of shares of common stock and potential dilutive securities outstanding during the period. For the three and six months ended June 30, 2021 and the six months ended June 30, 2020, there were no differences between basic and diluted net loss per share since the effect of the dilutive securities would have been antidilutive and therefore were excluded from the diluted net loss per share calculation. The following table sets forth the computation of the basic and diluted net income (loss) per share (in thousands, except share and per share data): f Three Months Ended June 30, Six Months Ended June 30, 2021 2020 2021 2020 Net income (loss), basic and diluted $ (122,428 ) $ 25,315 $ (258,569 ) $ (93,710 ) Weighted-average shares used in computing net income (loss) per share, basic 67,607,752 59,995,617 67,356,443 58,996,278 Weighted-average effect of dilutive securities: Options to purchase common stock and RSUs — 1,150,614 — — Weighted-average shares used in computing net income (loss) per share, diluted 67,607,752 61,146,231 67,356,443 58,996,278 Net income (loss) per share: Basic $ (1.81 ) $ 0.42 $ (3.84 ) $ (1.59 ) Diluted $ (1.81 ) $ 0.41 $ (3.84 ) $ (1.59 ) The following weighted-average outstanding common stock equivalents were excluded from the computation of diluted net income (loss) per share for the periods presented because including them would have been antidilutive: Three Months Ended June 30, Six Months Ended June 30, 2021 2020 2021 2020 Options to purchase common stock and restricted stock units 8,491,447 5,204,036 8,287,698 8,643,439 Employee stock purchase plan 8,621 10,952 4,334 5,476 Common stock warrants — — — 59,222 8,500,068 5,214,988 8,292,032 8,708,137 |
Equity Transactions
Equity Transactions | 6 Months Ended |
Jun. 30, 2021 | |
Equity [Abstract] | |
Equity Transactions | 10 . Equity Transactions In October 2020, the Company completed an underwritten public offering in which 5,111,110 shares of common stock were sold, which included 666,666 shares purchased by the underwriters pursuant to an option granted to them in connection with the offering, at a public offering price of $90.00 per share. For the year ended December 31, 2020, the total proceeds that the Company received from the offering were approximately $435.6 million, net of underwriting discounts and commissions. In May 2021, the Company entered into an Open Market Sale Agreement with Jefferies LLC, or Jefferies, pursuant to which the Company may offer and sell shares of the Company’s common stock having an aggregate offering proceeds up to $350.0 million, from time to time, in at-the-market offerings through Jefferies. As of June 30, 2021, the Company has not sold any shares under the arrangement. |
Accumulated Other Comprehensive
Accumulated Other Comprehensive Income | 6 Months Ended |
Jun. 30, 2021 | |
Other Comprehensive Income Loss Net Of Tax Period Increase Decrease [Abstract] | |
Accumulated Other Comprehensive Income | 11 . Accumulated Other Comprehensive Income Total accumulated other comprehensive income consisted of the following (in thousands): June 30, December 31, 2021 2020 Foreign currency translation adjustments $ 247 $ 429 Unrealized gain (loss) on available-for-sale securities (15 ) 260 Total accumulated other comprehensive income $ 232 $ 689 |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 6 Months Ended |
Jun. 30, 2021 | |
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 12, 2021 with the United States Securities and Exchange Commission (SEC). The results of operations for the three and six months ended June 30, 2021 are not necessarily indicative of the results to be expected for the year ending December 31, 2021. The Condensed Consolidated Balance Sheet as of December 31, 2020 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, stock-based compensation, and the liability related to the sale of future royalties. 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 used as collateral for the Company’s obligations under its facility leases and the gene therapy building construction project. 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, 2021 2020 Cash and cash equivalents $ 290,875 $ 335,639 Restricted cash included in prepaid expenses and other current assets 10,000 847 Restricted cash included in other assets 2,001 1,921 Total cash, cash equivalents, and restricted cash shown in the statements of cash flows $ 302,876 $ 338,407 |
Credit Losses | Credit Losses The Company is exposed to credit losses primarily through receivables from customers and collaborators and through its available-for-sale debt securities. For trade receivables and other instruments, the Company uses a new forward-looking expected loss model that generally results in the earlier recognition of allowances for losses. For available-for-sale debt securities with unrealized losses, the losses are recognized as allowances rather than as reductions in the amortized cost of the securities. The Company’s expected loss allowance methodology for the receivables is developed using historical collection experience, current and future economic market conditions, a review of the current aging status and financial condition of the entities. Specific allowance amounts are established to record the appropriate allowance for customers that have a higher probability of default. Balances are written off when determined to be uncollectible. The Company’s expected loss allowance methodology for the debt securities is developed by reviewing the extent of the unrealized loss, the size, term, geographical location, and industry of the issuer, the issuers’ credit ratings and any changes in those ratings, as well as reviewing current and future economic market conditions and the issuers’ current status and financial condition. The Company considered the current and expected future economic and market conditions surrounding the novel coronavirus (COVID-19) pandemic and determined that the estimate of credit losses was not significantly impacted. There was no material allowance recorded for losses on receivables and available-for-sale debt securities which were attributable to credit risk for the three and six months ended June 30, 2021 and 2020. |
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. 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 Company sold the right to receive certain royalty payments from net sales of Crysvita to RPI Finance Trust (RPI), an affiliate of Royalty Pharma, as further described in Note 7. The Company records the royalty revenue from the net sales of Crysvita in the applicable European territories on a prospective basis as non-cash royalty revenue in the Consolidated Statements of Operations over the term of the arrangement. The terms of the Company’s collaboration and license 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 obligations and recognizes revenue by measuring the progress towards complete satisfaction of the performance obligations using input measures. 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. The Company’s estimates of government mandated rebates, chargebacks, estimated product returns, and other deductions depends on the identification of key customer contract terms and conditions, as well as estimates of sales volumes to different classes of payors. |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 6 Months Ended |
Jun. 30, 2021 | |
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, 2021 2020 Cash and cash equivalents $ 290,875 $ 335,639 Restricted cash included in prepaid expenses and other current assets 10,000 847 Restricted cash included in other assets 2,001 1,921 Total cash, cash equivalents, and restricted cash shown in the statements of cash flows $ 302,876 $ 338,407 |
Financial Instruments (Tables)
Financial Instruments (Tables) | 6 Months Ended |
Jun. 30, 2021 | |
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, 2021 Level 1 Level 2 Level 3 Total Money market funds $ 206,674 $ — $ — $ 206,674 Certificate of deposits and time deposits — 17,501 — 17,501 Corporate bonds — 237,296 — 237,296 Commercial paper — 341,522 — 341,522 Asset-backed securities — 55,020 — 55,020 U.S. Government Treasury and agency securities 12,500 42,109 — 54,609 Debt securities in government-sponsored entities — 28,751 — 28,751 Investments in Arcturus and Solid common stock 103,091 — — 103,091 Total $ 322,265 $ 722,199 $ — $ 1,044,464 December 31, 2020 Level 1 Level 2 Level 3 Total Money market funds $ 598,392 $ — $ — $ 598,392 Time deposits — 10,000 — 10,000 Corporate bonds — 193,802 — 193,802 Commercial paper — 173,859 — 173,859 Asset-backed securities — 11,225 — 11,225 U.S. Government Treasury and agency securities 167,967 17,661 — 185,628 Investments in Arcturus and Solid common stock 154,756 — — 154,756 Total $ 921,115 $ 406,547 $ — $ 1,327,662 |
Balance Sheet Components (Table
Balance Sheet Components (Tables) | 6 Months Ended |
Jun. 30, 2021 | |
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 investments classified as available-for-sale securities consisted of the following (in thousands): June 30, 2021 Gross Unrealized Amortized Cost Gains Losses Estimated Fair Value Money market funds $ 206,674 $ — $ — $ 206,674 Certificate of deposits and time deposits 17,501 — — 17,501 Corporate bonds 237,310 45 (59 ) 237,296 Commercial paper 341,522 — — 341,522 Asset-backed securities 55,015 11 (6 ) 55,020 U.S. Government Treasury and agency securities 54,605 7 (3 ) 54,609 Debt securities in government-sponsored entities 28,761 4 (14 ) 28,751 Total $ 941,388 $ 67 $ (82 ) $ 941,373 December 31, 2020 Gross Unrealized Amortized Cost Gains Losses Estimated Fair Value Money market funds $ 598,392 $ — $ — $ 598,392 Time deposits 10,000 — — 10,000 Corporate bonds 193,610 209 (17 ) 193,802 Commercial paper 173,859 — — 173,859 Asset-backed securities 11,224 1 — 11,225 U.S. Government Treasury and agency securities 185,561 67 — 185,628 Total $ 1,172,646 $ 277 $ (17 ) $ 1,172,906 |
Summary of Inventory | Inventory consists of the following (in thousands): June 30, December 31, 2021 2020 Work-in-process $ 9,457 $ 7,184 Finished goods 5,626 5,864 Total inventory $ 15,083 $ 13,048 |
Accrued Liabilities | Accrued liabilities consist of the following (in thousands): June 30, December 31, 2021 2020 Research, clinical study, and manufacturing expenses $ 22,551 $ 25,875 Payroll and related expenses 41,545 58,176 Other 26,964 24,440 Total accrued liabilities $ 91,060 $ 108,491 |
Revenue (Tables)
Revenue (Tables) | 6 Months Ended |
Jun. 30, 2021 | |
Revenue From Contract With Customer [Abstract] | |
Summary of Disaggregation of Total Revenues | The following table disaggregates total revenues from customers (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2021 2020 2021 2020 Collaboration and license revenue: Crysvita collaboration revenue in profit- share territory $ 41,756 $ 29,806 $ 78,016 $ 57,021 Crysvita royalty revenue in European territory 228 1,498 228 1,498 Daiichi Sankyo 21,956 18,857 64,706 18,857 Total collaboration and license revenue 63,940 50,161 142,950 77,376 Product sales: Crysvita 2,900 2,549 8,772 4,159 Mepsevii 5,399 4,185 9,006 7,610 Dojolvi 10,047 1,332 17,081 2,776 Total product sales 18,346 8,066 34,859 14,545 Crysvita non-cash collaboration royalty revenue 4,689 3,482 8,561 6,097 Total revenues $ 86,975 $ 61,709 $ 186,370 $ 98,018 The following table disaggregates total revenues based on geographic location (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2021 2020 2021 2020 North America $ 75,567 $ 51,931 $ 163,309 $ 82,288 Europe 6,700 6,548 12,337 10,705 Latin America 4,708 3,230 10,724 5,025 Total revenues $ 86,975 $ 61,709 $ 186,370 $ 98,018 |
Summary of Changes in Contract Liabilities | The following table presents changes in the contract liabilities (in thousands): Six Months Ended June 30, 2021 2020 Balance of contract liabilities at beginning of period $ 66,568 $ — Additions 2,050 153,755 Deductions (64,706 ) (18,857 ) Balance of contract liabilities at end of period $ 3,912 $ 134,898 |
License and Research Agreemen_2
License and Research Agreements (Tables) | 6 Months Ended |
Jun. 30, 2021 | |
Share of Collaboration and Royalty Revenue Related to Crysvita | The Company’s Three Months Ended June 30, Six Months Ended June 30, 2021 2020 2021 2020 Company's share of revenue in profit- share territory $ 41,756 $ 29,806 $ 78,016 $ 57,021 Royalty revenue in European territory 228 1,498 228 1,498 Non-cash royalty revenue in European territory 4,689 3,482 8,561 6,097 Total $ 46,673 $ 34,786 $ 86,805 $ 64,616 |
Schedule of Cost Sharing Payments | Under the collaboration agreement, KKC and the Company share certain development and commercialization costs. Three Months Ended June 30, Six Months Ended June 30, 2021 2020 2021 2020 Research and development $ 5,171 $ 5,168 $ 11,356 $ 10,658 Selling, general and administrative 7,576 5,965 15,071 13,017 Total $ 12,747 $ 11,133 $ 26,427 $ 23,675 |
Solid Biosciences, Inc. | |
Schedule of Changes in Fair Value of Investment in Securities | The changes in the fair value of the Company’s investment in Solid’s common stock were as follows (in thousands): Solid common stock Acquisition of investment in Solid common stock in October 2020 $ 26,843 Change in fair value 32,477 December 31, 2020 59,320 Change in fair value (30,677 ) June 30, 2021 $ 28,643 |
Arcturus | |
Schedule of Changes in Fair Value of Equity Investment | The changes in the fair value of the Company’s equity investment in Arcturus securities were as follows (in thousands): Arcturus common stock Option to purchase additional shares of Arcturus common stock Total December 31, 2019 $ 26,088 $ 1,664 $ 27,752 Change in fair value 113,978 23,948 137,926 Transfer of value upon option exercise 35,212 (25,612 ) 9,600 Sale of shares (79,842 ) — (79,842 ) December 31, 2020 95,436 — 95,436 Change in fair value (20,988 ) — (20,988 ) June 30, 2021 $ 74,448 $ — $ 74,448 |
Liability Related to the Sale_2
Liability Related to the Sale of Future Royalties (Tables) | 6 Months Ended |
Jun. 30, 2021 | |
Deferred Revenue Disclosure [Abstract] | |
Schedule of Activity Within Liability Related to Sale of Future Royalties | The following table shows the activity within the liability account (in thousands): Liability related to the sale of future royalties December 31, 2019 $ 315,369 Non-cash collaboration royalty revenue (12,995 ) Non-cash interest expense 33,291 December 31, 2020 335,665 Non-cash collaboration royalty revenue (8,561 ) Non-cash interest expense 16,935 June 30, 2021 $ 344,039 |
Stock-Based Awards (Tables)
Stock-Based Awards (Tables) | 6 Months Ended |
Jun. 30, 2021 | |
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, 2021 2020 2021 2020 Cost of sales $ 223 $ 112 $ 602 $ 180 Research and development 15,094 12,856 28,583 23,785 Selling, general and administrative 11,825 9,441 22,255 18,616 Total stock-based compensation expense $ 27,142 $ 22,409 $ 51,440 $ 42,581 |
Net Income (Loss) Per Share (Ta
Net Income (Loss) Per Share (Tables) | 6 Months Ended |
Jun. 30, 2021 | |
Earnings Per Share [Abstract] | |
Computation of Basic and Diluted Net Income (Loss) per Share | The following table sets forth the computation of the basic and diluted net income (loss) per share (in thousands, except share and per share data): f Three Months Ended June 30, Six Months Ended June 30, 2021 2020 2021 2020 Net income (loss), basic and diluted $ (122,428 ) $ 25,315 $ (258,569 ) $ (93,710 ) Weighted-average shares used in computing net income (loss) per share, basic 67,607,752 59,995,617 67,356,443 58,996,278 Weighted-average effect of dilutive securities: Options to purchase common stock and RSUs — 1,150,614 — — Weighted-average shares used in computing net income (loss) per share, diluted 67,607,752 61,146,231 67,356,443 58,996,278 Net income (loss) per share: Basic $ (1.81 ) $ 0.42 $ (3.84 ) $ (1.59 ) Diluted $ (1.81 ) $ 0.41 $ (3.84 ) $ (1.59 ) |
Outstanding Shares of Common Stock Equivalents Excluded from Computation of Diluted Net Income (Loss) per Share | The following weighted-average outstanding common stock equivalents were excluded from the computation of diluted net income (loss) per share for the periods presented because including them would have been antidilutive: Three Months Ended June 30, Six Months Ended June 30, 2021 2020 2021 2020 Options to purchase common stock and restricted stock units 8,491,447 5,204,036 8,287,698 8,643,439 Employee stock purchase plan 8,621 10,952 4,334 5,476 Common stock warrants — — — 59,222 8,500,068 5,214,988 8,292,032 8,708,137 |
Accumulated Other Comprehensi_2
Accumulated Other Comprehensive Income (Tables) | 6 Months Ended |
Jun. 30, 2021 | |
Other Comprehensive Income Loss Net Of Tax Period Increase Decrease [Abstract] | |
Schedule of Total Accumulated Other Comprehensive Income | Total accumulated other comprehensive income consisted of the following (in thousands): June 30, December 31, 2021 2020 Foreign currency translation adjustments $ 247 $ 429 Unrealized gain (loss) on available-for-sale securities (15 ) 260 Total accumulated other comprehensive income $ 232 $ 689 |
Organization - Additional Infor
Organization - Additional Information (Details) | 6 Months Ended |
Jun. 30, 2021Segment | |
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, 2021 | Dec. 31, 2020 | Jun. 30, 2020 | Dec. 31, 2019 |
Accounting Policies [Abstract] | ||||
Cash and cash equivalents | $ 290,875 | $ 713,526 | $ 335,639 | |
Restricted cash included in prepaid expenses and other current assets | $ 10,000 | $ 847 | ||
Restricted Cash and Cash Equivalents, Current, Asset, Statement of Financial Position [Extensible List] | Prepaid and Other Current Assets | Prepaid and Other Current Assets | ||
Restricted cash included in other assets | $ 2,001 | $ 1,921 | ||
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 | $ 302,876 | $ 726,294 | $ 338,407 | $ 436,244 |
Financial Instruments - Summary
Financial Instruments - Summary of Financial Assets Measured on Recurring Basis (Details) - USD ($) $ in Thousands | Jun. 30, 2021 | Dec. 31, 2020 |
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | $ 1,044,464 | $ 1,327,662 |
Level 1 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 322,265 | 921,115 |
Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 722,199 | 406,547 |
Money Market Funds | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 206,674 | 598,392 |
Money Market Funds | Level 1 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 206,674 | 598,392 |
Asset-backed Securities | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 55,020 | 11,225 |
Asset-backed Securities | Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 55,020 | 11,225 |
Certificate of Deposits and Time Deposits | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 17,501 | |
Certificate of Deposits and Time Deposits | Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 17,501 | |
Corporate Bonds | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 237,296 | 193,802 |
Corporate Bonds | Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 237,296 | 193,802 |
Commercial Paper | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 341,522 | 173,859 |
Commercial Paper | Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 341,522 | 173,859 |
U.S. Government Treasury and Agency Securities | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 54,609 | 185,628 |
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 | 12,500 | 167,967 |
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 | 42,109 | 17,661 |
Debt Securities in Government Sponsored Entities | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 28,751 | |
Debt Securities in Government Sponsored Entities | Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 28,751 | |
Investments in Arcturus and Solid Common Stock | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 103,091 | 154,756 |
Investments in Arcturus and Solid Common Stock | Level 1 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | $ 103,091 | 154,756 |
Time Deposits | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 10,000 | |
Time Deposits | Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | $ 10,000 |
Financial Instruments - Additio
Financial Instruments - Additional Information (Details) - Convertible Promissory Note - USD ($) | 1 Months Ended | 6 Months Ended | |
Dec. 31, 2020 | Jul. 31, 2020 | Jun. 30, 2021 | |
Schedule Of Available For Sale Securities [Line Items] | |||
Investment amount | $ 2,500,000 | ||
Debt instrument maturity period | 2 years | ||
Debt instrument, face amount | $ 2,200,000 | ||
Interest receivable | 400,000 | ||
Allocated fair value of warrant | 600,000 | ||
Mazi Therapeutics, Inc. | |||
Schedule Of Available For Sale Securities [Line Items] | |||
Investment amount | $ 1,400,000 | ||
Debt instrument maturity period | 2 years | ||
Debt instrument, face amount | $ 1,400,000 | ||
Maximum | Dia Carta Ltd | |||
Schedule Of Available For Sale Securities [Line Items] | |||
Warrants issued to purchase preferred stock | $ 1,000,000 |
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, 2021 | Dec. 31, 2020 |
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | $ 941,388 | $ 1,172,646 |
Gross Unrealized Gains | 67 | 277 |
Gross Unrealized Losses | (82) | (17) |
Estimated Fair Value | 941,373 | 1,172,906 |
Money Market Funds | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 206,674 | 598,392 |
Estimated Fair Value | 206,674 | 598,392 |
Asset-backed Securities | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 55,015 | 11,224 |
Gross Unrealized Gains | 11 | 1 |
Gross Unrealized Losses | (6) | |
Estimated Fair Value | 55,020 | 11,225 |
Certificate of Deposits and Time Deposits | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 17,501 | |
Estimated Fair Value | 17,501 | |
Corporate Bonds | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 237,310 | 193,610 |
Gross Unrealized Gains | 45 | 209 |
Gross Unrealized Losses | (59) | (17) |
Estimated Fair Value | 237,296 | 193,802 |
Commercial Paper | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 341,522 | 173,859 |
Estimated Fair Value | 341,522 | 173,859 |
U.S. Government Treasury and Agency Securities | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 54,605 | 185,561 |
Gross Unrealized Gains | 7 | 67 |
Gross Unrealized Losses | (3) | |
Estimated Fair Value | 54,609 | 185,628 |
Debt Securities in Government Sponsored Entities | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 28,761 | |
Gross Unrealized Gains | 4 | |
Gross Unrealized Losses | (14) | |
Estimated Fair Value | $ 28,751 | |
Time Deposits | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 10,000 | |
Estimated Fair Value | $ 10,000 |
Balance Sheet Components - Addi
Balance Sheet Components - Additional Information (Details) - USD ($) | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2021 | Jun. 30, 2020 | Jun. 30, 2021 | Jun. 30, 2020 | |
Schedule Of Available For Sale Securities [Line Items] | ||||
Significant realized gains or losses on available-for-sale securities | $ 0 | $ 0 | $ 0 | $ 0 |
Maximum | ||||
Schedule Of Available For Sale Securities [Line Items] | ||||
Available-for-sale securities remaining contractual maturities | 5 years |
Balance Sheet Components - Su_2
Balance Sheet Components - Summary of Inventory (Details) - USD ($) $ in Thousands | Jun. 30, 2021 | Dec. 31, 2020 |
Investments Debt And Equity Securities [Abstract] | ||
Work-in-process | $ 9,457 | $ 7,184 |
Finished goods | 5,626 | 5,864 |
Total inventory | $ 15,083 | $ 13,048 |
Balance Sheet Components - Accr
Balance Sheet Components - Accrued Liabilities (Details) - USD ($) $ in Thousands | Jun. 30, 2021 | Dec. 31, 2020 |
Payables And Accruals [Abstract] | ||
Research, clinical study, and manufacturing expenses | $ 22,551 | $ 25,875 |
Payroll and related expenses | 41,545 | 58,176 |
Other | 26,964 | 24,440 |
Total accrued liabilities | $ 91,060 | $ 108,491 |
Revenue - Summary of Disaggrega
Revenue - Summary of Disaggregation of Total Revenues from Customers (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2021 | Jun. 30, 2020 | Jun. 30, 2021 | Jun. 30, 2020 | |
Total revenues | ||||
Total revenues | $ 86,975 | $ 61,709 | $ 186,370 | $ 98,018 |
Collaboration and License | ||||
Total revenues | ||||
Total revenues | 63,940 | 50,161 | 142,950 | 77,376 |
Collaboration and License | Crysvita | ||||
Total revenues | ||||
Total revenues | 41,756 | 29,806 | 78,016 | 57,021 |
Collaboration and License | Royalty Revenue in European Territory | Crysvita | ||||
Total revenues | ||||
Total revenues | 228 | 1,498 | 228 | 1,498 |
Collaboration and License | Daiichi Sankyo | ||||
Total revenues | ||||
Total revenues | 21,956 | 18,857 | 64,706 | 18,857 |
Product Sales | ||||
Total revenues | ||||
Total revenues | 18,346 | 8,066 | 34,859 | 14,545 |
Product Sales | Crysvita | ||||
Total revenues | ||||
Total revenues | 2,900 | 2,549 | 8,772 | 4,159 |
Product Sales | Mepsevii | ||||
Total revenues | ||||
Total revenues | 5,399 | 4,185 | 9,006 | 7,610 |
Product Sales | Dojolvi | ||||
Total revenues | ||||
Total revenues | 10,047 | 1,332 | 17,081 | 2,776 |
Non-cash Collaboration Royalty Revenue | ||||
Total revenues | ||||
Total revenues | 4,689 | 3,482 | 8,561 | 6,097 |
Non-cash Collaboration Royalty Revenue | Crysvita | ||||
Total revenues | ||||
Total revenues | $ 4,689 | $ 3,482 | $ 8,561 | $ 6,097 |
Revenue - Summary of Disaggre_2
Revenue - Summary of Disaggregation of Total Revenues (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2021 | Jun. 30, 2020 | Jun. 30, 2021 | Jun. 30, 2020 | |
Disaggregation Of Revenue [Line Items] | ||||
Total revenues | $ 86,975 | $ 61,709 | $ 186,370 | $ 98,018 |
North America | ||||
Disaggregation Of Revenue [Line Items] | ||||
Total revenues | 75,567 | 51,931 | 163,309 | 82,288 |
Europe | ||||
Disaggregation Of Revenue [Line Items] | ||||
Total revenues | 6,700 | 6,548 | 12,337 | 10,705 |
Latin America | ||||
Disaggregation Of Revenue [Line Items] | ||||
Total revenues | $ 4,708 | $ 3,230 | $ 10,724 | $ 5,025 |
Revenue - Summary of Changes in
Revenue - Summary of Changes in Contract Liabilities (Details) - USD ($) $ in Thousands | 6 Months Ended | |
Jun. 30, 2021 | Jun. 30, 2020 | |
Disaggregation Of Revenue [Abstract] | ||
Balance of contract liabilities at beginning of period | $ 66,568 | |
Additions | 2,050 | $ 153,755 |
Deductions | (64,706) | (18,857) |
Balance of contract liabilities at end of period | $ 3,912 | $ 134,898 |
Revenue - Additional Informatio
Revenue - Additional Information (Details) | Jun. 30, 2021 | Dec. 31, 2020 |
Disaggregation Of Revenue [Abstract] | ||
Percentage of gross accounts receivable balance | 58.00% | 71.00% |
License and Research Agreemen_3
License and Research Agreements - Additional Information (Details) | Jan. 01, 2023 | Jan. 31, 2021USD ($) | Dec. 31, 2020USD ($)$ / sharesshares | Oct. 31, 2020USD ($)shares | May 31, 2020USD ($)$ / sharesshares | Mar. 31, 2020USD ($) | Aug. 31, 2019USD ($) | Oct. 31, 2015USD ($) | Jun. 30, 2021USD ($)$ / sharesshares | Mar. 31, 2021USD ($) | Jun. 30, 2020USD ($) | Mar. 31, 2020USD ($)shares | Jun. 30, 2021USD ($)Quarter$ / sharesshares | Jun. 30, 2020USD ($)shares | Dec. 31, 2020USD ($)$ / sharesshares | Dec. 31, 2022 |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
Collaboration and license revenue | $ 86,975,000 | $ 61,709,000 | $ 186,370,000 | $ 98,018,000 | ||||||||||||
Research and development | $ 113,205,000 | 80,709,000 | $ 260,723,000 | 193,670,000 | ||||||||||||
Common stock, shares issued | shares | 66,818,520 | 67,764,950 | 67,764,950 | 66,818,520 | ||||||||||||
Proceeds from issuance of common stock in connection with license agreement | 55,268,000 | |||||||||||||||
Issuance of common stock in connection with license agreement, net of issuance costs | 55,268,000 | |||||||||||||||
Common stock investment value | $ 67,000 | $ 68,000 | $ 68,000 | $ 67,000 | ||||||||||||
Daiichi Sankyo | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
Deferred revenue | 3,900,000 | 3,900,000 | ||||||||||||||
Revenue recognized | 22,000,000 | 18,900,000 | 64,700,000 | $ 18,900,000 | ||||||||||||
Receivable related to Daiichi Sankyo license agreement | 800,000 | 800,000 | ||||||||||||||
Solid Biosciences, Inc. | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
Business acquisition, consideration transferred | 40,000,000 | |||||||||||||||
Common stock investment value | $ 26,800,000 | |||||||||||||||
Remaining consideration paid | 13,200,000 | |||||||||||||||
Common Stock | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
Issuance of common stock in connection with license agreement, net of issuance costs, shares | shares | 1,243,913 | |||||||||||||||
Issuance of common stock in connection with license agreement, net of issuance costs | $ 1,000 | |||||||||||||||
Common Stock | Solid Biosciences, Inc. | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
Unrealized gain (loss) | (14,600,000) | (30,700,000) | ||||||||||||||
Product Sales | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
Collaboration and license revenue | 18,346,000 | 8,066,000 | 34,859,000 | 14,545,000 | ||||||||||||
License and Collaboration Agreement | Mereo | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
Upfront payment for in-process research and development expense | $ 50,000,000 | |||||||||||||||
Payments made under agreement | $ 50,000,000 | |||||||||||||||
License and Collaboration Agreement | Maximum | Mereo | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
Future contingent milestone payments | $ 254,000,000 | |||||||||||||||
Kyowa Kirin Collaboration | License Agreement | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
Collaboration and license revenue | 200,000 | 1,500,000 | ||||||||||||||
Kyowa Kirin Collaboration | License Agreement | Prepaid and Other Current Assets | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
License agreement other receivables | 9,600,000 | 3,800,000 | 3,800,000 | 9,600,000 | ||||||||||||
Kyowa Kirin Collaboration | License Agreement | Product Sales | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
Collaboration and license revenue | 2,900,000 | 2,500,000 | 8,800,000 | 4,200,000 | ||||||||||||
Kyowa Kirin Collaboration | License Agreement | Profit Share Revenue | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
License agreement accounts receivable | 16,400,000 | 14,700,000 | 14,700,000 | 16,400,000 | ||||||||||||
Kyowa Kirin Collaboration | License Agreement | Commercial and Development Activity Reimbursements | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
License agreement accrued liabilities | $ 2,400,000 | 2,700,000 | $ 2,700,000 | $ 2,400,000 | ||||||||||||
Kyowa Kirin Collaboration | License Agreement | Scenario Forecast | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
Profit loss sharing percentage on net sales | 30.00% | 35.00% | ||||||||||||||
Kyowa Kirin Collaboration | License Agreement | Profit Share Territory | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
Remaining profit or loss share percentage on commercializing products | 50.00% | |||||||||||||||
Tiered double-digit revenue share percentage entitled to receive | 20.00% | |||||||||||||||
Kyowa Kirin Collaboration | License Agreement | Royalty Revenue in European Territory | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
Percentage of royalty on net sales receives | 10.00% | |||||||||||||||
Research, Develop and Commercialize AAV Gene Therapy Products | Bayer HealthCare LLC | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
Milestone payments received | $ 15,000,000 | |||||||||||||||
Research, Develop and Commercialize AAV Gene Therapy Products | Bayer HealthCare LLC | Maximum | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
Future contingent milestone payments | 232,000,000 | 232,000,000 | ||||||||||||||
Arcturus | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
Research and development | $ 0 | 0 | $ 0 | 0 | ||||||||||||
Number of shares purchased on exercise of common stock option | shares | 600,000 | |||||||||||||||
Option exercise price | $ / shares | $ 16 | |||||||||||||||
Purchase price of shares purchased on exercise of common stock option | $ 9,600,000 | |||||||||||||||
Common stock shares sold | shares | 800,000 | |||||||||||||||
Common stock. per share | $ / shares | $ 100.81 | $ 100.81 | ||||||||||||||
Net proceeds from sale of common stock | $ 79,800,000 | |||||||||||||||
Common stock, shares issued | shares | 2,200,000 | 2,200,000 | ||||||||||||||
Arcturus | Common Stock | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
Unrealized gain (loss) | $ (16,400,000) | $ 95,200,000 | $ (21,000,000) | $ 102,900,000 | ||||||||||||
Arcturus | Maximum | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
Development milestone payment | $ 24,500,000 | |||||||||||||||
Regulatory milestone payment | 24,500,000 | |||||||||||||||
Commercial milestone payment | $ 45,000,000 | |||||||||||||||
Program Agreement and Unitholder Option Agreement | GeneTx Biotherapeutics LLC | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
Research and development | $ 25,000,000 | |||||||||||||||
Upfront payment for exclusive option to acquisition | $ 20,000,000 | |||||||||||||||
Business acquisition option exercise period, description | the Company made an upfront payment of $20.0 million for an exclusive option to acquire GeneTx, which was exercisable any time prior to 30 days following FDA acceptance of the IND for GTX-102. | |||||||||||||||
Option extension Premium | 25,000,000 | |||||||||||||||
Business acquisition, consideration transferred | $ 125,000,000 | |||||||||||||||
Exercise of option extension, right to acquire condition | The Company has the right to acquire GeneTx for a payment of $125.0 million, at any time, until the earlier of 30 months from the first dosing of a patient in a planned Phase 1/2 study (subject to extensions) or 90 days after results are available from that study. | |||||||||||||||
Program Agreement and Unitholder Option Agreement | GeneTx Biotherapeutics LLC | Maximum | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
Extension period available for right to acquisition | Quarter | 4 | |||||||||||||||
License Agreement | REGENXBIO, Inc. | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
Upfront payment for in-process research and development expense | $ 7,000,000 | |||||||||||||||
Annual fees payable | 100,000 | |||||||||||||||
License Agreement | REGENXBIO, Inc. | Maximum | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
Future contingent milestone payments | $ 14,000,000 | $ 14,000,000 | ||||||||||||||
License and Technology Access Agreement | Daiichi Sankyo | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
Upfront payment received | $ 125,000,000 | |||||||||||||||
Deferred revenue | 25,000,000 | 25,000,000 | ||||||||||||||
Premium from stock purchase agreement | 19,700,000 | |||||||||||||||
Transaction price of license agreement | 183,600,000 | |||||||||||||||
Estimated reimbursement of delivering license and technology services | 5,900,000 | |||||||||||||||
License and Technology Access Agreement | HeLA PCL Platform | Daiichi Sankyo | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
Deferred revenue | $ 25,000,000 | 25,000,000 | ||||||||||||||
License and Technology Access Agreement | Sublicense Agreement | Daiichi Sankyo | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
Revenue from sublicense agreement | 8,000,000 | |||||||||||||||
License and Technology Access Agreement | License Agreement | Daiichi Sankyo | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
Revenue recognized | 153,900,000 | |||||||||||||||
Stock Purchase Agreement | Daiichi Sankyo | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
Issuance of common stock in connection with license agreement, net of issuance costs, shares | shares | 1,243,913 | |||||||||||||||
Proceeds from issuance of common stock in connection with license agreement | $ 75,000,000 | |||||||||||||||
Issuance of common stock in connection with license agreement, net of issuance costs | $ 55,300,000 | |||||||||||||||
Stock price | $ / shares | $ 44.43 | $ 44.43 | ||||||||||||||
Premium from stock purchase agreement | $ 19,700,000 | |||||||||||||||
Sublicense Agreement | Daiichi Sankyo | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
Upfront payment received | $ 8,000,000 | |||||||||||||||
Collaboration and License Agreement | Maximum | Solid Biosciences, Inc. | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
Development milestone payment | 25,000,000 | |||||||||||||||
Regulatory milestone payment | 65,000,000 | |||||||||||||||
Commercial milestone payment | $ 165,000,000 | |||||||||||||||
Collaboration Agreement | Solid Biosciences, Inc. | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||
Number of common stock purchased | shares | 7,825,797 | |||||||||||||||
Aggregate purchase price | $ 40,000,000 |
License and Research Agreemen_4
License and Research Agreements - Share of Collaboration and Royalty Revenue Related to Crysvita (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2021 | Jun. 30, 2020 | Jun. 30, 2021 | Jun. 30, 2020 | |
Disaggregation Of Revenue [Line Items] | ||||
Collaboration and license revenue | $ 86,975 | $ 61,709 | $ 186,370 | $ 98,018 |
Collaboration and Royalty | Kyowa Kirin Collaboration | ||||
Disaggregation Of Revenue [Line Items] | ||||
Collaboration and license revenue | 46,673 | 34,786 | 86,805 | 64,616 |
Collaboration and Royalty | Kyowa Kirin Collaboration | Profit Share Territory | ||||
Disaggregation Of Revenue [Line Items] | ||||
Collaboration and license revenue | 41,756 | 29,806 | 78,016 | 57,021 |
Collaboration and Royalty | Kyowa Kirin Collaboration | Royalty Revenue in European Territory | ||||
Disaggregation Of Revenue [Line Items] | ||||
Collaboration and license revenue | 228 | 1,498 | 228 | 1,498 |
Non-cash Collaboration Royalty Revenue | ||||
Disaggregation Of Revenue [Line Items] | ||||
Collaboration and license revenue | 4,689 | 3,482 | 8,561 | 6,097 |
Non-cash Collaboration Royalty Revenue | Kyowa Kirin Collaboration | Royalty Revenue in European Territory | ||||
Disaggregation Of Revenue [Line Items] | ||||
Collaboration and license revenue | $ 4,689 | $ 3,482 | $ 8,561 | $ 6,097 |
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, 2021 | Jun. 30, 2020 | Jun. 30, 2021 | Jun. 30, 2020 | |
Cost Sharing Payments [Line Items] | ||||
Research and development | $ 5,171 | $ 5,168 | $ 11,356 | $ 10,658 |
Selling, general and administrative | 7,576 | 5,965 | 15,071 | 13,017 |
Total | $ 12,747 | $ 11,133 | $ 26,427 | $ 23,675 |
License and Research Agreemen_6
License and Research Agreements - Schedule of Changes in Fair Value of Equity Investment (Details) - USD ($) $ in Thousands | 6 Months Ended | 12 Months Ended |
Jun. 30, 2021 | Dec. 31, 2020 | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Common stock, Beginning balance | $ 1,327,662 | |
Common stock, Ending balance | 1,044,464 | $ 1,327,662 |
Arcturus | ||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Fair value of option to purchase additional shares of common stock, Beginning balance | 1,664 | |
Fair value of option to purchase additional shares of common stock, Change in fair value | 23,948 | |
Fair value of option to purchase additional shares of common stock, Transfer of value upon option exercise | (25,612) | |
Common stock, Beginning balance | 95,436 | 27,752 |
Common stock, Change in fair value | (20,988) | 137,926 |
Common stock, Transfer of value upon option exercise | 9,600 | |
Common stock, Sale of shares | (79,842) | |
Common stock, Ending balance | 74,448 | 95,436 |
Arcturus | Common Stock | ||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Common stock, Beginning balance | 95,436 | 26,088 |
Common stock, Change in fair value | (20,988) | 113,978 |
Common stock, Transfer of value upon option exercise | 35,212 | |
Common stock, Sale of shares | (79,842) | |
Common stock, Ending balance | $ 74,448 | $ 95,436 |
License and Research Agreemen_7
License and Research Agreements - Schedule of Changes in Fair Value of Investment in Securities (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended |
Dec. 31, 2020 | Jun. 30, 2021 | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Common stock, Beginning balance | $ 1,327,662 | |
Common stock, Ending balance | $ 1,327,662 | 1,044,464 |
Common Stock | Solid Biosciences, Inc. | ||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Common stock, Beginning balance | 59,320 | |
Common stock, Acquisition of investment in securities | 26,843 | |
Common stock, Change in fair value | 32,477 | (30,677) |
Common stock, Ending balance | $ 59,320 | $ 28,643 |
Liability Related to the Sale_3
Liability Related to the Sale of Future Royalties - Additional Information (Details) - Royalty Purchase Agreement - RPI Finance Trust (RPI) - USD ($) | 1 Months Ended | 6 Months Ended |
Dec. 31, 2019 | Jun. 30, 2021 | |
Liability Related To Sale Of Future Royalties [Line Items] | ||
Proceeds from sale of future royalties | $ 320,000,000 | |
Royalty payment termination description | The agreement with RPI will automatically terminate, and the payment of royalties to RPI will cease, in the event aggregate royalty payments received by RPI are equal to or greater than $608.0 million prior to December 31, 2030, or in the event aggregate royalty payments received by RPI are less than $608.0 million prior to December 31, 2030, when aggregate royalty payments received by RPI are equal to $800.0 million. | |
Royalties transaction costs net | 5,800,000 | |
Proceeds from the sale of future royalties, net | 314,200,000 | |
Effective annual interest rate | 9.80% | |
Minimum [Member] | ||
Liability Related To Sale Of Future Royalties [Line Items] | ||
Royalty agreement termination threshold amount | 608,000,000 | |
Maximum | ||
Liability Related To Sale Of Future Royalties [Line Items] | ||
Royalty agreement termination threshold amount | $ 800,000,000 |
Liability Related to the Sale_4
Liability Related to the Sale of Future Royalties - Schedule of Activity Within Liability Related to Sale of Future Royalties (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | 12 Months Ended | ||
Jun. 30, 2021 | Jun. 30, 2020 | Jun. 30, 2021 | Jun. 30, 2020 | Dec. 31, 2020 | |
Deferred Revenue Disclosure [Abstract] | |||||
Liability related to the sale of future royalties at beginning of year | $ 335,665 | $ 315,369 | $ 315,369 | ||
Non-cash collaboration royalty revenue | (8,561) | (6,097) | (12,995) | ||
Non-cash interest expense | $ 8,517 | $ 8,429 | 16,935 | $ 16,511 | 33,291 |
Liability related to the sale of future royalties at end of year | $ 344,039 | $ 344,039 | $ 335,665 |
Stock-Based Awards - Additional
Stock-Based Awards - Additional Information (Details) | 6 Months Ended |
Jun. 30, 2021shares | |
2014 Incentive Plan | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
Share reserved for future issuance | 4,103,718 |
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 | 3,956,907 |
Employment Inducement Plan | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
Share reserved for future issuance | 488,223 |
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, 2021 | Jun. 30, 2020 | Jun. 30, 2021 | Jun. 30, 2020 | |
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||||
Stock-based compensation expense | $ 27,142 | $ 22,409 | $ 51,440 | $ 42,581 |
Cost of sales | ||||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||||
Stock-based compensation expense | 223 | 112 | 602 | 180 |
Research and development | ||||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||||
Stock-based compensation expense | 15,094 | 12,856 | 28,583 | 23,785 |
Selling, general and administrative | ||||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||||
Stock-based compensation expense | $ 11,825 | $ 9,441 | $ 22,255 | $ 18,616 |
Net Income (Loss) Per Share - C
Net Income (Loss) Per Share - Computation of Basic and Diluted Net Income (Loss) per Share (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2021 | Jun. 30, 2020 | Jun. 30, 2021 | Jun. 30, 2020 | |
Schedule Of Basic And Diluted Net Income Loss Per Share [Line Items] | ||||
Net income (loss) | $ (122,428) | $ 25,315 | $ (258,569) | $ (93,710) |
Weighted-average shares used in computing net income (loss) per share, basic | 67,607,752 | 59,995,617 | 67,356,443 | 58,996,278 |
Weighted-average effect of dilutive securities: | ||||
Weighted-average shares used in computing net income (loss) per share, diluted | 67,607,752 | 61,146,231 | 67,356,443 | 58,996,278 |
Net income (loss) per share: | ||||
Basic | $ (1.81) | $ 0.42 | $ (3.84) | $ (1.59) |
Diluted | $ (1.81) | $ 0.41 | $ (3.84) | $ (1.59) |
Options to Purchase Common Stock and RSUs | ||||
Weighted-average effect of dilutive securities: | ||||
Weighted-average effect of dilutive securities | 1,150,614 |
Net Income (Loss) Per Share - O
Net Income (Loss) Per Share - Outstanding Shares of Common Stock Equivalents Excluded from Computation of Diluted Net Income (Loss) per Share (Details) - shares | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2021 | Jun. 30, 2020 | Jun. 30, 2021 | Jun. 30, 2020 | |
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||||
Common stock equivalents excluded from computation of diluted net income (loss) per share | 8,500,068 | 5,214,988 | 8,292,032 | 8,708,137 |
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 income (loss) per share | 8,491,447 | 5,204,036 | 8,287,698 | 8,643,439 |
Common Stock Warrants | ||||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||||
Common stock equivalents excluded from computation of diluted net income (loss) per share | 59,222 | |||
Employee Stock Purchase Plan | ||||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||||
Common stock equivalents excluded from computation of diluted net income (loss) per share | 8,621 | 10,952 | 4,334 | 5,476 |
Equity Transactions - Additiona
Equity Transactions - Additional Information (Details) - USD ($) $ / shares in Units, $ in Millions | 1 Months Ended | 3 Months Ended | 12 Months Ended | |
May 31, 2021 | Oct. 31, 2020 | Jun. 30, 2021 | Dec. 31, 2020 | |
Underwritten Public Offering | ||||
Stockholders Equity [Line Items] | ||||
Common stock shares sold | 5,111,110 | |||
Shares purchased by underwriters | 666,666 | |||
Public offering price | $ 90 | |||
Net proceeds from sale of common stock | $ 435.6 | |||
Open Market Sale | ||||
Stockholders Equity [Line Items] | ||||
Common stock shares sold | 0 | |||
Option to sell common stock aggregate offering price | $ 350 |
Accumulated Other Comprehensi_3
Accumulated Other Comprehensive Income - Schedule of Total Accumulated Other Comprehensive Income (Details) - USD ($) $ in Thousands | Jun. 30, 2021 | Dec. 31, 2020 |
Accumulated Other Comprehensive Income Loss Net Of Tax [Abstract] | ||
Foreign currency translation adjustments | $ 247 | $ 429 |
Unrealized gain (loss) on available-for-sale securities | (15) | 260 |
Total accumulated other comprehensive income | $ 232 | $ 689 |