Document and Entity Information
Document and Entity Information - shares | 3 Months Ended | |
Mar. 31, 2022 | May 02, 2022 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Mar. 31, 2022 | |
Document Fiscal Year Focus | 2022 | |
Document Fiscal Period Focus | Q1 | |
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 | 69,930,191 | |
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 | Mar. 31, 2022 | Dec. 31, 2021 |
Current assets: | ||
Cash and cash equivalents | $ 153,988 | $ 307,584 |
Marketable debt securities | 444,333 | 432,612 |
Accounts receivable, net | 28,369 | 28,432 |
Inventory | 17,809 | 16,231 |
Prepaid expenses and other current assets | 63,020 | 71,745 |
Total current assets | 707,519 | 856,604 |
Property, plant, and equipment, net | 177,073 | 141,247 |
Equity investments | 25,596 | 34,925 |
Marketable debt securities | 215,526 | 258,933 |
Right-of-use assets | 32,699 | 34,936 |
Intangible assets, net | 159,992 | 130,788 |
Goodwill | 44,406 | 44,406 |
Other assets | 21,218 | 20,558 |
Total assets | 1,384,029 | 1,522,397 |
Current liabilities: | ||
Accounts payable | 23,685 | 17,138 |
Accrued liabilities | 126,356 | 145,555 |
Contract liabilities | 5,867 | 7,609 |
Lease liabilities | 11,323 | 11,066 |
Total current liabilities | 167,231 | 181,368 |
Lease liabilities | 28,043 | 30,904 |
Contract liabilities | 0 | 1,467 |
Deferred tax liabilities | 33,306 | 33,306 |
Liability related to the sale of future royalties | 355,633 | 351,786 |
Other liabilities | 3,392 | 1,005 |
Total liabilities | 587,605 | 599,836 |
Stockholders’ equity: | ||
Preferred stock - 25,000,000 shares authorized; nil outstanding as of March 30, 2022 and December 31, 2021 | 0 | 0 |
Common stock - 250,000,000 shares authorized; 67,961,963 and 66,818,520 shares issued and outstanding as of March 31, 2021 and December 31, 2021, respectively | 70 | 69 |
Additional paid-in capital | 3,028,829 | 2,997,497 |
Accumulated other comprehensive loss | (6,554) | (1,404) |
Accumulated deficit | (2,225,921) | (2,073,601) |
Total stockholders’ equity | 796,424 | 922,561 |
Total liabilities and stockholders’ equity | $ 1,384,029 | $ 1,522,397 |
CONDENSED CONSOLIDATED BALANC_2
CONDENSED CONSOLIDATED BALANCE SHEETS (Parenthetical) - shares | Mar. 31, 2022 | Dec. 31, 2021 |
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 | 69,796,940 | 69,344,998 |
Common stock, shares outstanding | 69,796,940 | 69,344,998 |
CONDENSED CONSOLIDATED STATEMEN
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Revenues: | ||
Total revenues | $ 79,935 | $ 99,395 |
Operating expenses: | ||
Cost of sales | 6,100 | 5,188 |
Research and development | 143,155 | 147,518 |
Selling, general and administrative | 67,312 | 53,258 |
Total operating expenses | 216,567 | 205,964 |
Loss from operations | (136,632) | (106,569) |
Interest income | 494 | 639 |
Change in fair value of equity investments | (9,329) | (20,619) |
Non-cash interest expense on liability related to the sale of future royalties | (6,584) | (8,418) |
Other income (expense) | 289 | (795) |
Loss before income taxes | (151,762) | (135,762) |
Provision for income taxes | (558) | (379) |
Net loss | $ (152,320) | $ (136,141) |
Net loss per share, basic and diluted | $ (2.19) | $ (2.03) |
Weighted-average shares used in computing net loss per share, basic and diluted | 69,516,668 | 67,102,342 |
Collaboration and License | ||
Revenues: | ||
Total revenues | $ 48,413 | $ 79,010 |
Product Sales | ||
Revenues: | ||
Total revenues | 26,684 | 16,513 |
Non-cash Collaboration Royalty Revenue | ||
Revenues: | ||
Total revenues | $ 4,838 | $ 3,872 |
CONDENSED CONSOLIDATED STATEM_2
CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Statement of Comprehensive Income [Abstract] | ||
Net loss | $ (152,320) | $ (136,141) |
Other comprehensive income (loss): | ||
Foreign currency translation adjustments | (60) | (285) |
Unrealized loss on available-for-sale securities | (5,090) | (348) |
Other comprehensive loss: | (5,150) | (633) |
Total comprehensive loss | $ (157,470) | $ (136,774) |
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, 2020 | $ 1,154,375 | $ 67 | $ 2,773,195 | $ (689) | $ (1,619,576) |
Beginning balance, shares at Dec. 31, 2020 | 66,818,520 | ||||
Stock-based compensation | 24,220 | 24,220 | |||
Issuance of common stock under equity plan awards, net of tax | 12,761 | 12,761 | |||
Issuance of common stock under equity plan awards, net of tax, shares | 620,957 | ||||
Other comprehensive income (loss) | (633) | (633) | |||
Net loss | (136,141) | (136,141) | |||
Ending balance at Mar. 31, 2021 | 1,054,582 | $ 67 | 2,810,176 | 56 | (1,755,717) |
Ending balance, shares at Mar. 31, 2021 | 67,439,477 | ||||
Beginning balance at Dec. 31, 2021 | $ 922,561 | $ 69 | 2,997,497 | 1,404 | (2,073,601) |
Beginning balance, shares at Dec. 31, 2021 | 69,344,998 | 69,344,998 | |||
Issuance of common stock in connection with at-the-market offering, net of issuance costs, shares | 1,050,372 | ||||
Stock-based compensation | $ 29,578 | 29,578 | |||
Issuance of common stock under equity plan awards, net of tax | 1,755 | $ 1 | 1,754 | ||
Issuance of common stock under equity plan awards, net of tax, shares | 451,942 | ||||
Other comprehensive income (loss) | (5,150) | (5,150) | |||
Net loss | (152,320) | (152,320) | |||
Ending balance at Mar. 31, 2022 | $ 796,424 | $ 70 | $ 3,028,829 | $ (6,554) | $ (2,225,921) |
Ending balance, shares at Mar. 31, 2022 | 69,796,940 | 69,796,940 |
CONDENSED CONSOLIDATED STATEM_4
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Operating activities: | ||
Net loss | $ (152,320) | $ (136,141) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Stock-based compensation | 29,396 | 24,254 |
Amortization of premium on marketable debt securities, net | 1,894 | 940 |
Depreciation and amortization | 4,088 | 3,367 |
Change in fair value of equity investments | 9,329 | 20,619 |
Non-cash collaboration royalty revenue | (4,838) | (3,872) |
Non-cash interest expense on liability related to the sale of future royalties | 6,584 | 8,418 |
Other | 35 | 325 |
Changes in operating assets and liabilities: | ||
Accounts receivable | 33 | (1,993) |
Inventory | (1,475) | 474 |
Prepaid expenses and other assets | 9,126 | (10,656) |
Accounts payable, accrued, and other liabilities | (16,164) | (23,602) |
Contract liabilities | (3,209) | (41,479) |
Net cash used in operating activities | (117,521) | (159,346) |
Investing activities: | ||
Purchase of property, plant, and equipment | (32,184) | (16,414) |
Purchase of marketable debt securities | (203,710) | (405,230) |
Proceeds from sale of marketable debt securities | 39,422 | 14,980 |
Proceeds from maturities of marketable debt securities | 188,990 | 224,240 |
Purchase of mutual funds related to nonqualified deferred compensation plan | (2,458) | |
Payment for intangible asset | (30,000) | |
Other | 2,578 | |
Net cash used in investing activities | (37,362) | (182,424) |
Financing activities: | ||
Proceeds from the issuance of common stock equity plan awards, net | 1,755 | 12,761 |
Other | (143) | (112) |
Net cash provided by financing activities | 1,612 | 12,649 |
Effect of exchange rate changes on cash | (108) | (771) |
Net decrease in cash, cash equivalents and restricted cash | (153,379) | (329,892) |
Cash, cash equivalents and restricted cash at beginning of period | 309,585 | 726,294 |
Cash, cash equivalents and restricted cash at end of period | 156,206 | $ 396,402 |
Supplemental disclosures of non-cash information: | ||
Acquired lease liabilities arising from obtaining right-of-use assets | 132 | |
Non-cash interest expense on liability related to the sale of future royalties capitalized into ending property, plant and equipment | $ 2,101 |
Organization
Organization | 3 Months Ended |
Mar. 31, 2022 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Organization | 1. Organization Ultragenyx Pharmaceutical Inc. (the Company) is a biopharmaceutical company incorporated in Delaware. 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 four commercially approved products. Crysvita® (burosumab) is approved in the United States (U.S.) 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 U.S. 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. Mepsevii® (vestronidase alfa) is approved by the FDA as the first medicine for the treatment of children and adults with mucopolysaccharidosis VII (MPS VII), also known as Sly syndrome. In the European Union and the United Kingdom, Mepsevii is approved under exceptional circumstances for patients of all ages for the treatment of non-neurological manifestations of MPS VII. In Brazil, Mepsevii is approved for the treatment of MPS VII for patients of all ages. Dojolvi® (triheptanoin) is approved in the U.S., Canada, and Brazil for the treatment of pediatric and adult patients severely affected by long-chain fatty acid oxidation disorders (LC-FAOD). On January 7, 2022, the Company announced a collaboration with Regeneron Pharmaceuticals (Regeneron) to commercialize Evkeeza® (evinacumab) outside of the U.S. Evkeeza is approved in the U.S. and the European Economic Area (EEA) for the treatment of homozygous familial hypercholesterolemia (HoFH). In addition to the approved products, the Company has the following ongoing clinical development programs: • DTX401 is an adeno-associated virus 8 (AAV8) gene therapy product candidate for the treatment of patients with glycogen storage disease type Ia (GSDIa); • DTX301 is an AAV8 gene therapy product candidate in development for the treatment of patients with ornithine transcarbamylase (OTC) deficiency, the most common urea cycle disorder; • UX143 (setrusumab), which is subject to the Company's collaboration agreement with Mereo BioPharma 3 (Mereo), is a fully human monoclonal antibody that inhibits sclerostin, a protein that acts on a key bone-signaling pathway and inhibits the activity of bone-forming cells for the treatment of patients with osteogenesis imperfect (OI); • 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 likely need to raise additional capital to fully implement its business plans. Through March 31, 2022, 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 | 3 Months Ended |
Mar. 31, 2022 | |
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 Condensed 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 15, 2022 (Annual Report) with the United States Securities and Exchange Commission (the SEC). The results of operations for the three months ended March 31, 2022 are not necessarily indicative of the results to be expected for the year ending December 31, 2022. The Condensed Consolidated Balance Sheet as of December 31, 2021 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 Condensed Consolidated Financial Statements have been prepared in accordance with GAAP. The preparation of the Condensed 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 Condensed 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, revenue recognition, 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 Condensed Consolidated Balance Sheets that sum to the total of the same such amounts shown in the Condensed Consolidated Statement of Cash Flows (in thousands): March 31, 2022 2021 Cash and cash equivalents $ 153,988 $ 383,794 Restricted cash included in prepaid expenses and 217 10,687 Restricted cash included in other assets 2,001 1,921 Total cash, cash equivalents, and restricted cash $ 156,206 $ 396,402 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 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. There were no material credit losses recorded for receivables and available-for-sale debt securities for the three months ended March 31, 2022 and 2021. 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 , which 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 Condensed 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 order to record collaboration revenue, the Company utilizes certain information from its collaboration partners, 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 Condensed 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. These reserves are based on estimates of the amounts earned or to be claimed on the related sales and are reviewed periodically and adjusted as necessary. 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. If actual results vary, the Company may need to adjust these estimates, which could have an effect on earnings in the period of the adjustment. |
Financial Instruments
Financial Instruments | 3 Months Ended |
Mar. 31, 2022 | |
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) and Solid Biosciences Inc. (Solid) by using the quoted market prices, which are Level 1 fair value measurements. 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): March 31, 2022 Level 1 Level 2 Level 3 Total Money market funds $ 113,600 $ — $ — $ 113,600 Certificate of deposits and time deposits — 27,101 — 27,101 Corporate bonds — 384,474 — 384,474 Commercial paper — 120,685 — 120,685 Asset-backed securities — 12,875 — 12,875 U.S. Government Treasury and agency securities 7,172 106,344 — 113,516 Debt securities in government-sponsored entities — 19,328 — 19,328 Investments in Arcturus and Solid common stock 22,871 — — 22,871 Other — 3,400 — 3,400 Total $ 143,643 $ 674,207 $ — $ 817,850 December 31, 2021 Level 1 Level 2 Level 3 Total Money market funds $ 266,765 $ — $ — $ 266,765 Certificate of deposits and time deposits — 16,000 — 16,000 Corporate bonds — 349,691 — 349,691 Commercial paper — 187,624 — 187,624 Asset-backed securities — 41,245 — 41,245 U.S. Government Treasury and agency securities — 87,435 — 87,435 Debt securities in government-sponsored entities — 19,549 — 19,549 Investments in Arcturus and Solid common stock 32,200 — — 32,200 Other — 942 — 942 Total $ 298,965 $ 702,486 $ — $ 1,001,451 |
Balance Sheet Components
Balance Sheet Components | 3 Months Ended |
Mar. 31, 2022 | |
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): March 31, 2022 Gross Unrealized Amortized Gains Losses Estimated Money market funds $ 113,600 $ — $ — $ 113,600 Certificates of deposit and time deposits 27,101 — — 27,101 Corporate bonds 389,619 1 ( 5,146 ) 384,474 Commercial paper 120,686 — ( 1 ) 120,685 Asset-backed securities 12,924 — ( 49 ) 12,875 U.S. Government Treasury and agency securities 114,445 29 ( 958 ) 113,516 Debt securities in government-sponsored entities 19,577 — ( 249 ) 19,328 Total $ 797,952 $ 30 $ ( 6,403 ) $ 791,579 December 31, 2021 Gross Unrealized Amortized Gains Losses Estimated Money market funds $ 266,765 $ — $ — $ 266,765 Certificates of deposit and time deposits 16,000 — — 16,000 Corporate bonds 350,667 3 ( 979 ) 349,691 Commercial paper 187,624 — — 187,624 Asset-backed securities 41,282 1 ( 38 ) 41,245 U.S. Government Treasury and agency securities 87,642 1 ( 208 ) 87,435 Debt securities in government-sponsored entities 19,612 — ( 63 ) 19,549 Total $ 969,592 $ 5 $ ( 1,288 ) $ 968,309 At March 31, 2022 , the remaining contractual maturities of available-for-sale securities were less than three years . There have been no significant realized gains or losses on available-for-sale securities for the three months ended March 31, 2022 and 2021. The unrealized losses on the Company’s investments in marketable debt securities were caused by interest rate increases. The contractual terms of these investments do not permit the issuers to settle the securities at a price less than the par value. Accordingly, it is expected that the securities would not be settled at a price less than the amortized cost basis of these investments. The Company does not intend to sell the investments and it is not more likely than not that the Company will be required to sell the investments before recovery of their amortized cost basis. All marketable securities with unrealized losses at March 31, 2022 have been in a loss position for less than twelve months. Inventory Inventory consists of the following (in thousands): March 31, December 31, 2022 2021 Work-in-process $ 11,989 $ 10,504 Finished goods 5,820 5,727 Total inventory $ 17,809 $ 16,231 Accrued Liabilities Accrued liabilities consist of the following (in thousands): March 31, December 31, 2022 2021 Research, clinical study, and manufacturing expenses $ 40,092 $ 40,880 Payroll and related expenses 41,638 62,591 Other 44,626 42,084 Total accrued liabilities $ 126,356 $ 145,555 |
Revenue
Revenue | 3 Months Ended |
Mar. 31, 2022 | |
Revenue from Contract with Customer [Abstract] | |
Revenue | 5. Revenue The following table disaggregates total revenues from customers (in thousands): Three Months Ended March 31, 2022 2021 Collaboration and license revenue: Crysvita collaboration revenue in profit- $ 45,164 $ 36,260 Daiichi Sankyo 3,249 42,750 Total collaboration and license revenue 48,413 79,010 Product sales: Crysvita 9,394 5,872 Mepsevii 4,861 3,607 Dojolvi 12,429 7,034 Total product sales 26,684 16,513 Crysvita non-cash collaboration royalty 4,838 3,872 Total revenues $ 79,935 $ 99,395 The following table disaggregates total revenues based on geographic location (in thousands): Three Months Ended March 31, 2022 2021 U.S. and Canada $ 61,917 $ 87,742 Europe 8,227 5,637 Latin America 9,791 6,016 Total revenues $ 79,935 $ 99,395 The following table presents changes in the contract liabilities (in thousands): Three Months Ended March 31, 2022 2021 Balance of contract liabilities at beginning of period $ ( 9,076 ) $ ( 66,568 ) Additions ( 40 ) ( 1,271 ) Deductions 3,249 42,750 Balance of contract liabilities at end of period $ ( 5,867 ) $ ( 25,089 ) See Note 6 for additional details on contract liabilities activities. The Company’s largest accounts receivable balance accounted for 61 % and 71 % of the total accounts receivable balance as of March 31, 2022 and December 31, 2021, respectively, and was due from a collaboration partner. |
License and Research Agreements
License and Research Agreements | 3 Months Ended |
Mar. 31, 2022 | |
Research Grant Agreement [Abstract] | |
License and Research Agreements | 6. License and Research Agreements Kyowa Kirin Collaboration and License Agreement In August 2013, the Company entered into a collaboration and license agreement with Kyowa Kirin Co., Ltd. (KKC or formerly Kyowa Hakko Kirin Co., Ltd. or KHK). Under the terms of this collaboration and license agreement, as amended, the Company and KKC collaborate on the development and commercialization of Crysvita in the field of orphan diseases in the U.S. 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. 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. 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 Crysvita 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. The Company’s share of collaboration and royalty revenue related to Crysvita was as follows (in thousands): Three Months Ended March 31, 2022 2021 Company's share of revenue in profit- $ 45,164 $ 36,260 Non-cash royalty revenue in European 4,838 3,872 Total $ 50,002 $ 40,132 Product revenue related to sales in other territories The Company is responsible for commercializing Crysvita 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 Crysvita once the product is delivered and the risk and title of the product is transferred to the distributor. The Company recorded product sales of $ 9.4 million and $ 5.9 million for the three months ended March 31, 2022 and 2021, 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 the collaboration agreement, KKC manufactures and supplies Crysvita, which is purchased by the Company for sales in its 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. As a result, the Company was reimbursed for these costs and operating expenses were reduced as follows (in thousands): Three Months Ended March 31, 2022 2021 Research and development $ 4,619 $ 6,185 Selling, general and administrative 9,196 7,495 Total $ 13,815 $ 13,680 Collaboration receivable and payable The Company had accounts receivable from KKC in the amount of $ 17.4 million and $ 20.2 million from profit-share revenue and royalties and other receivables recorded in prepaid expenses and other current assets of $ 3.8 million and $ 16.0 million and accrued liabilities of $ 4.2 million and $ 2.3 million from commercial and development activity reimbursements, as of March 31, 2022 and December 31, 2021, respectively. 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, which was recorded as an in-process research and development expense. In April 2022, the parties entered into an amendment to the Unitholder Option Agreement (the “Amendment”) which provides the Company with an additional, earlier option to acquire GeneTx for a payment of $ 75.0 million based on the earlier of receipt of interim data in the Phase 1/2 study or a specified date (such option, the “Interim Option”). After such date, the Company has the right to acquire GeneTx for a payment of $ 125.0 million until the earlier of 30 months from the first dosing of a patient in the 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 one additional three month period, by paying an additional extension fee. Pursuant to the Amendment, in the event the Company acquires GeneTx by exercising the Interim Option, the Company will be required to make a milestone payment to GeneTx in an amount up to $ 30.0 million upon achievement of the earlier of a Phase 3 clinical study start and product approvals in Canada and the U.K. 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 would 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. 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 Pinnacle PCL TM producer cell line platform (Pinnacle PCL Platform), and HEK293 transient transfection manufacturing technology platforms for AAV-based gene therapy products. Under the terms of the License Agreement, Daiichi Sankyo made an upfront payment of $ 125.0 million and an additional $ 25.0 million upon completion of the technology transfer of the Pinnacle PCL Platform and HEK293 platform. Daiichi Sankyo reimbursed the Company for all costs associated with the transfer of the manufacturing technology and will pay single-digit royalties on net sales of products manufactured in either system. The Company also entered into a Stock Purchase Agreement (SPA) with Daiichi Sankyo, pursuant to which Daiichi Sankyo purchased 1,243,913 shares of the Company’s common stock in exchange for $ 75.0 million in cash during the first quarter of 2020. 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. Daiichi Sankyo is also subject to a three-year standstill and restrictions on sale of the shares (subject to customary exceptions or release). 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, milestone payments, and royalties on any net sales of products incorporating the licensed intellectual property. 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 Pinnacle PCL Platform and HEK293 transient transfection manufacturing technology platform together with the initial technical assistance and technology transfer services, which was completed in the first quarter of 2022, 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 March 31, 2022 , the Company has determined that the total transaction price of the License Agreement was $ 183.3 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 $ 5.6 million estimated reimbursement amount for delivering the license and technology services. Total revenue recognized under the license agreement through March 31, 2022 was $ 177.5 million. 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 was recognized over an initial period which was completed during the first quarter of 2022, 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 is being 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. 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 $ 3.2 million and $ 42.8 million for the three months ended March 31, 2022 and 2021, respectively, in revenue related to this arrangement. Accordingly, the Company had recorded $ 5.9 and $ 9.1 million of contract liabilities, net, as of March 31, 2022 and December 31, 2021, respectively. The Company recorded an accounts receivable related to the above agreements of nil and $ 0.1 million as of March 31, 2022 and December 31, 2021, respectively. 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. Regeneron Pharmaceuticals, Inc. In January 2022, the Company announced a collaboration with Regeneron Pharmaceuticals, Inc., or Regeneron, to commercialize Evkeeza for HoFH outside of the U.S. Evkeeza is approved in the U.S., where it is marketed by Regeneron and in the European Economic Area, or EEA, as a first-in-class therapy for use together with diet and other low-density lipoprotein-cholesterol- lowering therapies to treat adults and adolescents aged 12 years and older with HoFH. Pursuant to the terms of the agreement, the Company received the rights to develop, commercialize and distribute the product for HoFH in countries outside of the U.S. The Company will share in certain costs for global trials led by Regeneron and also have the right to opt into other potential indications. The Company may be obligated to pay up to $ 63.0 million in future milestone payments, contingent upon the achievement of certain regulatory and sales milestones. The collaboration agreement is within the scope of ASC 808 which provides guidance on the presentation and disclosure of collaborative arrangements. As the Company would be the principal in future sale transactions with the customer, the Company will recognize product sales and cost of sales in the period the related sale occurs and the related revenue recognition criteria are met. Under the collaboration agreement, Regeneron will supply the product and will charge the Company a transfer price from the low 20 % range up to 40 % on net sales, which will be recognized as cost of sales in the Company's Statement of Operations. Upon the closing of the transaction in January 2022, the Company paid Regeneron a $ 30.0 million upfront payment. As the upfront payment was related to the Company's usage of intellectual property related to Evkeeza for HoFH, the upfront payment was recorded on the Consolidated Balance Sheet as an intangible asset, which is being amortized over its useful life of 10.5 years. The Company recorded costs of goods sold of $ 0.7 million for the three months ended March 31, 2022 , related to the amortization of the intangible asset. Further, the Company reimbursed Regeneron for certain development costs of $ 0.6 million for the three months ended March 31, 2022 . No sales of Evkeeza were recorded for the three months ended March 31, 2022. Other Arrangements The Company has also entered into several collaborations and/or licensing arrangements in prior periods. Except as disclosed above, there have not been material changes in these arrangements as disclosed in Note 7 to the Consolidated Financial Statements in the Annual Report. Under the financial terms of these arrangements, the Company may be required to make payments upon achievement of developmental, regulatory, and commercial milestones, which could be significant. Future milestone payments, if any, will be reflected in the Condensed Consolidated Statements of Operations upon the occurrence of the contingent event. In addition, the Company may be required to pay royalties on future sales if products related to these arrangements are commercialized. The payment of these amounts, however, is contingent upon the occurrence of various future events, which have a high degree of uncertainty of occurrence. As described in the Annual Report, in connection with the Company’s collaborations with Solid Biosciences, Inc. (Solid) and Arcturus Therapeutic Holding, Inc. (Arcturus), the Company holds certain equity interests in each entity. The changes in the fair value of the Company’s equity investments in the common stock of Solid and Arcturus were as follows (in thousands ): Common stock As of December 31, 2020 154,756 Change in fair value ( 42,713 ) Sale of shares ( 79,843 ) December 31, 2021 32,200 Change in fair value ( 9,329 ) March 31, 2022 $ 22,871 |
Liability Related to the Sale o
Liability Related to the Sale of Future Royalties | 3 Months Ended |
Mar. 31, 2022 | |
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, and Switzerland under the terms of the Company’s Collaboration and License Agreement with KKC dated August 29, 2013, as amended. 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 . 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 9.6 % as of March 31, 2022. 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, 2020 $ 335,665 Non-cash collaboration royalty revenue ( 17,951 ) Non-cash interest expense 34,072 December 31, 2021 351,786 Non-cash collaboration royalty revenue ( 4,838 ) Non-cash interest expense 8,685 March 31, 2022 $ 355,633 |
Stock-Based Awards
Stock-Based Awards | 3 Months Ended |
Mar. 31, 2022 | |
Share-based Payment Arrangement [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 March 31, 2022 , there were 2,008,628 shares reserved under the 2014 Plan for the future issuance of equity awards, 4,698,895 shares reserved for the 2014 Employee Stock Purchase Plan, and 436,063 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 2022 2021 Cost of sales $ 175 $ 379 Research and development 16,907 13,489 Selling, general and administrative 12,305 10,430 Total stock-based compensation expense $ 29,387 $ 24,298 |
Net Loss Per Share
Net Loss Per Share | 3 Months Ended |
Mar. 31, 2022 | |
Earnings Per Share [Abstract] | |
Net Loss Per Share | 9. Net Loss Per Share Basic net loss per share has been computed by dividing the net loss by the weighted-average number of shares of common stock outstanding during the period. Diluted net loss per share is calculated by dividing net loss by the weighted-average number of shares of common stock and potential dilutive securities outstanding during the period. The following weighted-average outstanding common stock equivalents were excluded from the computation of diluted net loss per share for the periods presented because including them would have been antidilutive: Three Months Ended 2022 2021 Options to purchase common stock, 9,363,681 8,084,585 Employee stock purchase plan 23,276 33,636 9,386,957 8,118,221 |
Equity Transactions
Equity Transactions | 3 Months Ended |
Mar. 31, 2022 | |
Equity [Abstract] | |
Equity Transactions | 10. Equity Transactions In May 2021, the Company entered into an Open Market Sale Agreement with Jefferies LLC, (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 (ATM) offerings through Jefferies. As of March 31, 2022 , the Company has sold 1,050,372 shares under the arrangement resulting in net proceeds of approximately $ 78.9 million. No shares were sold under the arrangement for the three months ended March 31, 2022 . |
Accumulated Other Comprehensive
Accumulated Other Comprehensive Loss | 3 Months Ended |
Mar. 31, 2022 | |
Other Comprehensive Income (Loss), Net of Tax [Abstract] | |
Accumulated Other Comprehensive Loss | 11. Accumulated Other Comprehensive Loss Total accumulated other comprehensive loss consisted of the following (in thousands): March 31, December 31, 2022 2021 Foreign currency translation adjustments $ ( 181 ) $ ( 121 ) Unrealized loss on available-for-sale securities ( 6,373 ) ( 1,283 ) Total accumulated other comprehensive loss $ ( 6,554 ) $ ( 1,404 ) |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 3 Months Ended | |
Jun. 30, 2022 | Mar. 31, 2022 | |
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 Condensed 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 15, 2022 (Annual Report) with the United States Securities and Exchange Commission (the SEC). The results of operations for the three months ended March 31, 2022 are not necessarily indicative of the results to be expected for the year ending December 31, 2022. The Condensed Consolidated Balance Sheet as of December 31, 2021 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 Condensed Consolidated Financial Statements have been prepared in accordance with GAAP. The preparation of the Condensed 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 Condensed 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, revenue recognition, 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 Condensed Consolidated Balance Sheets that sum to the total of the same such amounts shown in the Condensed Consolidated Statement of Cash Flows (in thousands): March 31, 2022 2021 Cash and cash equivalents $ 153,988 $ 383,794 Restricted cash included in prepaid expenses and 217 10,687 Restricted cash included in other assets 2,001 1,921 Total cash, cash equivalents, and restricted cash $ 156,206 $ 396,402 | |
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 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. There were no material credit losses recorded for receivables and available-for-sale debt securities for the three months ended March 31, 2022 and 2021. | |
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 , which 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 Condensed 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 order to record collaboration revenue, the Company utilizes certain information from its collaboration partners, 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 Condensed 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. These reserves are based on estimates of the amounts earned or to be claimed on the related sales and are reviewed periodically and adjusted as necessary. 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. If actual results vary, the Company may need to adjust these estimates, which could have an effect on earnings in the period of the adjustment. |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
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 Condensed Consolidated Balance Sheets that sum to the total of the same such amounts shown in the Condensed Consolidated Statement of Cash Flows (in thousands): March 31, 2022 2021 Cash and cash equivalents $ 153,988 $ 383,794 Restricted cash included in prepaid expenses and 217 10,687 Restricted cash included in other assets 2,001 1,921 Total cash, cash equivalents, and restricted cash $ 156,206 $ 396,402 |
Financial Instruments (Tables)
Financial Instruments (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
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): March 31, 2022 Level 1 Level 2 Level 3 Total Money market funds $ 113,600 $ — $ — $ 113,600 Certificate of deposits and time deposits — 27,101 — 27,101 Corporate bonds — 384,474 — 384,474 Commercial paper — 120,685 — 120,685 Asset-backed securities — 12,875 — 12,875 U.S. Government Treasury and agency securities 7,172 106,344 — 113,516 Debt securities in government-sponsored entities — 19,328 — 19,328 Investments in Arcturus and Solid common stock 22,871 — — 22,871 Other — 3,400 — 3,400 Total $ 143,643 $ 674,207 $ — $ 817,850 December 31, 2021 Level 1 Level 2 Level 3 Total Money market funds $ 266,765 $ — $ — $ 266,765 Certificate of deposits and time deposits — 16,000 — 16,000 Corporate bonds — 349,691 — 349,691 Commercial paper — 187,624 — 187,624 Asset-backed securities — 41,245 — 41,245 U.S. Government Treasury and agency securities — 87,435 — 87,435 Debt securities in government-sponsored entities — 19,549 — 19,549 Investments in Arcturus and Solid common stock 32,200 — — 32,200 Other — 942 — 942 Total $ 298,965 $ 702,486 $ — $ 1,001,451 |
Balance Sheet Components (Table
Balance Sheet Components (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
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): March 31, 2022 Gross Unrealized Amortized Gains Losses Estimated Money market funds $ 113,600 $ — $ — $ 113,600 Certificates of deposit and time deposits 27,101 — — 27,101 Corporate bonds 389,619 1 ( 5,146 ) 384,474 Commercial paper 120,686 — ( 1 ) 120,685 Asset-backed securities 12,924 — ( 49 ) 12,875 U.S. Government Treasury and agency securities 114,445 29 ( 958 ) 113,516 Debt securities in government-sponsored entities 19,577 — ( 249 ) 19,328 Total $ 797,952 $ 30 $ ( 6,403 ) $ 791,579 December 31, 2021 Gross Unrealized Amortized Gains Losses Estimated Money market funds $ 266,765 $ — $ — $ 266,765 Certificates of deposit and time deposits 16,000 — — 16,000 Corporate bonds 350,667 3 ( 979 ) 349,691 Commercial paper 187,624 — — 187,624 Asset-backed securities 41,282 1 ( 38 ) 41,245 U.S. Government Treasury and agency securities 87,642 1 ( 208 ) 87,435 Debt securities in government-sponsored entities 19,612 — ( 63 ) 19,549 Total $ 969,592 $ 5 $ ( 1,288 ) $ 968,309 |
Summary of Inventory | Inventory consists of the following (in thousands): March 31, December 31, 2022 2021 Work-in-process $ 11,989 $ 10,504 Finished goods 5,820 5,727 Total inventory $ 17,809 $ 16,231 |
Accrued Liabilities | Accrued liabilities consist of the following (in thousands): March 31, December 31, 2022 2021 Research, clinical study, and manufacturing expenses $ 40,092 $ 40,880 Payroll and related expenses 41,638 62,591 Other 44,626 42,084 Total accrued liabilities $ 126,356 $ 145,555 |
Revenue (Tables)
Revenue (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
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 March 31, 2022 2021 Collaboration and license revenue: Crysvita collaboration revenue in profit- $ 45,164 $ 36,260 Daiichi Sankyo 3,249 42,750 Total collaboration and license revenue 48,413 79,010 Product sales: Crysvita 9,394 5,872 Mepsevii 4,861 3,607 Dojolvi 12,429 7,034 Total product sales 26,684 16,513 Crysvita non-cash collaboration royalty 4,838 3,872 Total revenues $ 79,935 $ 99,395 The following table disaggregates total revenues based on geographic location (in thousands): Three Months Ended March 31, 2022 2021 U.S. and Canada $ 61,917 $ 87,742 Europe 8,227 5,637 Latin America 9,791 6,016 Total revenues $ 79,935 $ 99,395 |
Summary of Changes in Contract Liabilities | The following table presents changes in the contract liabilities (in thousands): Three Months Ended March 31, 2022 2021 Balance of contract liabilities at beginning of period $ ( 9,076 ) $ ( 66,568 ) Additions ( 40 ) ( 1,271 ) Deductions 3,249 42,750 Balance of contract liabilities at end of period $ ( 5,867 ) $ ( 25,089 ) |
License and Research Agreemen_2
License and Research Agreements (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
Share of Collaboration and Royalty Revenue Related to Crysvita | The Company’s share of collaboration and royalty revenue related to Crysvita was as follows (in thousands): Three Months Ended March 31, 2022 2021 Company's share of revenue in profit- $ 45,164 $ 36,260 Non-cash royalty revenue in European 4,838 3,872 Total $ 50,002 $ 40,132 |
Schedule of Cost Sharing Payments | Under the collaboration agreement, KKC and the Company share certain development and commercialization costs. As a result, the Company was reimbursed for these costs and operating expenses were reduced as follows (in thousands): Three Months Ended March 31, 2022 2021 Research and development $ 4,619 $ 6,185 Selling, general and administrative 9,196 7,495 Total $ 13,815 $ 13,680 |
Solid and Arcturus | |
Schedule of Changes in Fair Value of Equity Investment | The changes in the fair value of the Company’s equity investments in the common stock of Solid and Arcturus were as follows (in thousands ): Common stock As of December 31, 2020 154,756 Change in fair value ( 42,713 ) Sale of shares ( 79,843 ) December 31, 2021 32,200 Change in fair value ( 9,329 ) March 31, 2022 $ 22,871 |
Liability Related to the Sale_2
Liability Related to the Sale of Future Royalties (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
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, 2020 $ 335,665 Non-cash collaboration royalty revenue ( 17,951 ) Non-cash interest expense 34,072 December 31, 2021 351,786 Non-cash collaboration royalty revenue ( 4,838 ) Non-cash interest expense 8,685 March 31, 2022 $ 355,633 |
Stock-Based Awards (Tables)
Stock-Based Awards (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
Share-based Payment Arrangement [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 2022 2021 Cost of sales $ 175 $ 379 Research and development 16,907 13,489 Selling, general and administrative 12,305 10,430 Total stock-based compensation expense $ 29,387 $ 24,298 |
Net Loss Per Share (Tables)
Net Loss Per Share (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
Earnings Per Share [Abstract] | |
Outstanding Shares of Common Stock Equivalents Excluded from Computation of Diluted Net Loss per Share | The following weighted-average outstanding common stock equivalents were excluded from the computation of diluted net loss per share for the periods presented because including them would have been antidilutive: Three Months Ended 2022 2021 Options to purchase common stock, 9,363,681 8,084,585 Employee stock purchase plan 23,276 33,636 9,386,957 8,118,221 |
Accumulated Other Comprehensi_2
Accumulated Other Comprehensive Loss (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
Other Comprehensive Income (Loss), Net of Tax [Abstract] | |
Schedule of Total Accumulated Other Comprehensive Loss | Total accumulated other comprehensive loss consisted of the following (in thousands): March 31, December 31, 2022 2021 Foreign currency translation adjustments $ ( 181 ) $ ( 121 ) Unrealized loss on available-for-sale securities ( 6,373 ) ( 1,283 ) Total accumulated other comprehensive loss $ ( 6,554 ) $ ( 1,404 ) |
Organization - Additional Infor
Organization - Additional Information (Details) | 3 Months Ended |
Mar. 31, 2022Segment | |
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 | Mar. 31, 2022 | Dec. 31, 2021 | Mar. 31, 2021 | Dec. 31, 2020 |
Accounting Policies [Abstract] | ||||
Cash and cash equivalents | $ 153,988 | $ 307,584 | $ 383,794 | |
Restricted cash included in prepaid expenses and other current assets | $ 217 | $ 10,687 | ||
Restricted Cash and Cash Equivalents, Current, Asset, Statement of Financial Position [Extensible List] | Prepaid expenses and other current assets | Prepaid expenses 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 | $ 156,206 | $ 309,585 | $ 396,402 | $ 726,294 |
Financial Instruments - Summary
Financial Instruments - Summary of Financial Assets Measured on Recurring Basis (Details) - USD ($) $ in Thousands | Mar. 31, 2022 | Dec. 31, 2021 |
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | $ 817,850 | $ 1,001,451 |
Level 1 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 143,643 | 298,965 |
Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 674,207 | 702,486 |
Money Market Funds | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 113,600 | 266,765 |
Money Market Funds | Level 1 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 113,600 | 266,765 |
Asset-backed Securities | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 12,875 | 41,245 |
Asset-backed Securities | Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 12,875 | 41,245 |
Certificate of Deposits and Time Deposits | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 27,101 | 16,000 |
Certificate of Deposits and Time Deposits | Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 27,101 | 16,000 |
Corporate Bonds | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 384,474 | 349,691 |
Corporate Bonds | Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 384,474 | 349,691 |
Commercial Paper | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 120,685 | 187,624 |
Commercial Paper | Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 120,685 | 187,624 |
U.S. Government Treasury and Agency Securities | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 113,516 | 87,435 |
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 | 7,172 | |
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 | 106,344 | 87,435 |
Debt Securities in Government Sponsored Entities | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 19,328 | 19,549 |
Debt Securities in Government Sponsored Entities | Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 19,328 | 19,549 |
Investments in Arcturus and Solid Common Stock | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 22,871 | 32,200 |
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 | 22,871 | 32,200 |
Other | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 3,400 | 942 |
Other | Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | $ 3,400 | $ 942 |
Balance Sheet Components - Summ
Balance Sheet Components - Summary of Cash Equivalents and Investments Classified as Available For Sale Securities (Details) - USD ($) $ in Thousands | Mar. 31, 2022 | Dec. 31, 2021 |
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | $ 797,952 | $ 969,592 |
Gross Unrealized Gains | 30 | 5 |
Gross Unrealized Losses | (6,403) | (1,288) |
Estimated Fair Value | 791,579 | 968,309 |
Money Market Funds | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 113,600 | 266,765 |
Estimated Fair Value | 113,600 | 266,765 |
Asset-backed Securities | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 12,924 | 41,282 |
Gross Unrealized Gains | 1 | |
Gross Unrealized Losses | (49) | (38) |
Estimated Fair Value | 12,875 | 41,245 |
Certificate of Deposits and Time Deposits | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 27,101 | 16,000 |
Estimated Fair Value | 27,101 | 16,000 |
Corporate Bonds | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 389,619 | 350,667 |
Gross Unrealized Gains | 1 | 3 |
Gross Unrealized Losses | (5,146) | (979) |
Estimated Fair Value | 384,474 | 349,691 |
Commercial Paper | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 120,686 | 187,624 |
Gross Unrealized Losses | (1) | |
Estimated Fair Value | 120,685 | 187,624 |
U.S. Government Treasury and Agency Securities | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 114,445 | 87,642 |
Gross Unrealized Gains | 29 | 1 |
Gross Unrealized Losses | (958) | (208) |
Estimated Fair Value | 113,516 | 87,435 |
Debt Securities in Government Sponsored Entities | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 19,577 | 19,612 |
Gross Unrealized Losses | (249) | (63) |
Estimated Fair Value | $ 19,328 | $ 19,549 |
Balance Sheet Components - Addi
Balance Sheet Components - Additional Information (Details) | 3 Months Ended |
Mar. 31, 2022 | |
Maximum | |
Schedule Of Available For Sale Securities [Line Items] | |
Available-for-sale securities remaining contractual maturities | 3 years |
Balance Sheet Components - Su_2
Balance Sheet Components - Summary of Inventory (Details) - USD ($) $ in Thousands | Mar. 31, 2022 | Dec. 31, 2021 |
Investments, Debt and Equity Securities [Abstract] | ||
Work-in-process | $ 11,989 | $ 10,504 |
Finished goods | 5,820 | 5,727 |
Total inventory | $ 17,809 | $ 16,231 |
Balance Sheet Components - Accr
Balance Sheet Components - Accrued Liabilities (Details) - USD ($) $ in Thousands | Mar. 31, 2022 | Dec. 31, 2021 |
Payables and Accruals [Abstract] | ||
Research, clinical study, and manufacturing expenses | $ 40,092 | $ 40,880 |
Payroll and related expenses | 41,638 | 62,591 |
Other | 44,626 | 42,084 |
Total accrued liabilities | $ 126,356 | $ 145,555 |
Revenue - Summary of Disaggrega
Revenue - Summary of Disaggregation of Total Revenues from Customers (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Total revenues | ||
Total revenues | $ 79,935 | $ 99,395 |
Collaboration and License | ||
Total revenues | ||
Total revenues | 48,413 | 79,010 |
Collaboration and License | Crysvita | ||
Total revenues | ||
Total revenues | 45,164 | 36,260 |
Collaboration and License | Daiichi Sankyo | ||
Total revenues | ||
Total revenues | 3,249 | 42,750 |
Product Sales | ||
Total revenues | ||
Total revenues | 26,684 | 16,513 |
Product Sales | Crysvita | ||
Total revenues | ||
Total revenues | 9,394 | 5,872 |
Product Sales | Mepsevii | ||
Total revenues | ||
Total revenues | 4,861 | 3,607 |
Product Sales | Dojolvi | ||
Total revenues | ||
Total revenues | 12,429 | 7,034 |
Non-cash Collaboration Royalty Revenue | ||
Total revenues | ||
Total revenues | 4,838 | 3,872 |
Non-cash Collaboration Royalty Revenue | Crysvita | ||
Total revenues | ||
Total revenues | $ 4,838 | $ 3,872 |
Revenue - Summary of Disaggre_2
Revenue - Summary of Disaggregation of Total Revenues (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Disaggregation Of Revenue [Line Items] | ||
Total revenues | $ 79,935 | $ 99,395 |
U.S. and Canada [Member] | ||
Disaggregation Of Revenue [Line Items] | ||
Total revenues | 61,917 | 87,742 |
Europe | ||
Disaggregation Of Revenue [Line Items] | ||
Total revenues | 8,227 | 5,637 |
Latin America | ||
Disaggregation Of Revenue [Line Items] | ||
Total revenues | $ 9,791 | $ 6,016 |
Revenue - Summary of Changes in
Revenue - Summary of Changes in Contract Liabilities (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Disaggregation of Revenue [Abstract] | ||
Balance of contract liabilities at beginning of period | $ (9,076) | $ (66,568) |
Additions | (40) | (1,271) |
Deductions | 3,249 | 42,750 |
Balance of contract liabilities at end of period | $ (5,867) | $ (25,089) |
Revenue - Additional Informatio
Revenue - Additional Information (Details) | Mar. 31, 2022 | Dec. 31, 2021 |
Disaggregation of Revenue [Abstract] | ||
Percentage of gross accounts receivable balance | 61.00% | 71.00% |
License and Research Agreemen_3
License and Research Agreements - Additional Information (Details) - USD ($) | Jan. 01, 2023 | Apr. 30, 2022 | Jan. 31, 2022 | Jan. 31, 2021 | Mar. 31, 2020 | Aug. 31, 2019 | Mar. 31, 2022 | Mar. 31, 2021 | Mar. 31, 2020 | Dec. 31, 2022 | Dec. 31, 2021 | Sep. 30, 2021 |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Collaboration and license revenue | $ 79,935,000 | $ 99,395,000 | ||||||||||
Daiichi Sankyo | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Deferred revenue | 5,900,000 | $ 9,100,000 | ||||||||||
Revenue recognized | 3,200,000 | 42,800,000 | ||||||||||
Receivable related to Daiichi Sankyo license agreement | $ 0 | 100,000 | ||||||||||
Regeneron Pharmaceuticals, Inc. | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Upfront payments of collaboration | $ 30,000,000 | |||||||||||
Intangible assets, remaining amortization period | 10 years 6 months | |||||||||||
Eligible future milestone payments | $ 63,000,000 | |||||||||||
Costs of goods sold related to amortization of intangible asset | $ 700,000 | |||||||||||
Development costs | 600,000 | |||||||||||
Sales | $ 0 | |||||||||||
Regeneron Pharmaceuticals, Inc. | Maximum | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Profit loss sharing percentage on net sales | 40.00% | |||||||||||
Regeneron Pharmaceuticals, Inc. | Minimum | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Profit loss sharing percentage on net sales | 20.00% | |||||||||||
Product Sales | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Collaboration and license revenue | $ 26,684,000 | 16,513,000 | ||||||||||
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 | Prepaid Expenses and Other Current Assets | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
License agreement other receivables | 3,800,000 | 16,000,000 | ||||||||||
Kyowa Kirin Collaboration | License Agreement | Product Sales | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Collaboration and license revenue | 9,400,000 | $ 5,900,000 | ||||||||||
Kyowa Kirin Collaboration | License Agreement | Profit Share Revenue | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
License agreement accounts receivable | 17,400,000 | 20,200,000 | ||||||||||
Kyowa Kirin Collaboration | License Agreement | Commercial and Development Activity Reimbursements | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
License agreement accrued liabilities | $ 4,200,000 | $ 2,300,000 | ||||||||||
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% | |||||||||||
Program Agreement and Unitholder Option Agreement | GeneTx Biotherapeutics LLC | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Additional milestone payments | $ 30,000,000 | |||||||||||
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 until the earlier of 30 months from the first dosing of a patient in the Phase 1/2 study (subject to extensions) or 90 days after results are available from that study. | |||||||||||
Program Agreement and Unitholder Option Agreement | Subsequent Event | GeneTx Biotherapeutics LLC | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Acquisition cost | $ 75,000,000 | |||||||||||
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 | |||||||||||
Premium from stock purchase agreement | 19,700,000 | |||||||||||
Transaction price of license agreement | 183,300,000 | |||||||||||
Estimated reimbursement of delivering license and technology services | 5,600,000 | |||||||||||
License and Technology Access Agreement | PCL and HEK293 Platform | Daiichi Sankyo | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Deferred revenue | $ 25,000,000 | |||||||||||
License and Technology Access Agreement | License Agreement | Daiichi Sankyo | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Revenue recognized | 177,500,000 | |||||||||||
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 | 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 | $ 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 |
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 | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Disaggregation Of Revenue [Line Items] | ||
Collaboration and license revenue | $ 79,935 | $ 99,395 |
Collaboration and Royalty | Kyowa Kirin Collaboration | Profit Share Territory | ||
Disaggregation Of Revenue [Line Items] | ||
Collaboration and license revenue | 45,164 | 36,260 |
Collaboration and Royalty | Kyowa Kirin Collaboration | Royalty Revenue in European Territory | ||
Disaggregation Of Revenue [Line Items] | ||
Collaboration and license revenue | 4,838 | 3,872 |
Non-cash Collaboration Royalty Revenue | ||
Disaggregation Of Revenue [Line Items] | ||
Collaboration and license revenue | 4,838 | 3,872 |
Non-cash Collaboration Royalty Revenue | Kyowa Kirin Collaboration | Royalty Revenue in European Territory | ||
Disaggregation Of Revenue [Line Items] | ||
Collaboration and license revenue | $ 50,002 | $ 40,132 |
License and Research Agreemen_5
License and Research Agreements - Schedule of Cost Sharing Payments (Details) - Kyowa Kirin Collaboration - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Cost Sharing Payments [Line Items] | ||
Research and development | $ 4,619 | $ 6,185 |
Selling, general and administrative | 9,196 | 7,495 |
Total | $ 13,815 | $ 13,680 |
License and Research Agreemen_6
License and Research Agreements - Schedule of Changes in Fair Value of Equity Investment (Details) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended |
Mar. 31, 2022 | Dec. 31, 2021 | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Common stock, Beginning balance | $ 1,001,451 | |
Common stock, Ending balance | 817,850 | $ 1,001,451 |
Solid and Arcturus | Common Stock | ||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Common stock, Beginning balance | 32,200 | 154,756 |
Common stock, Change in fair value | (9,329) | (42,713) |
Common stock, Sale of shares | (79,843) | |
Common stock, Ending balance | $ 22,871 | $ 32,200 |
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 ($) $ in Millions | 1 Months Ended | 3 Months Ended |
Dec. 31, 2019 | Mar. 31, 2022 | |
Liability Related To Sale Of Future Royalties [Line Items] | ||
Proceeds from sale of future royalties | $ 320 | |
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 | 5.8 | |
Proceeds from the sale of future royalties, net | 314.2 | |
Effective annual interest rate | 9.60% | |
Minimum | ||
Liability Related To Sale Of Future Royalties [Line Items] | ||
Royalty agreement termination threshold amount | 608 | |
Maximum | ||
Liability Related To Sale Of Future Royalties [Line Items] | ||
Royalty agreement termination threshold amount | $ 800 |
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 | 12 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | Dec. 31, 2021 | |
Deferred Revenue Disclosure [Abstract] | |||
Liability related to the sale of future royalties at beginning of year | $ 351,786 | $ 335,665 | $ 335,665 |
Non-cash collaboration royalty revenue | (4,838) | $ (3,872) | (17,951) |
Non-cash interest expense | 8,685 | 34,072 | |
Liability related to the sale of future royalties at end of year | $ 355,633 | $ 351,786 |
Stock-Based Awards - Additional
Stock-Based Awards - Additional Information (Details) | 3 Months Ended |
Mar. 31, 2022shares | |
2014 Incentive Plan | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
Share reserved for future issuance | 2,008,628 |
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 | 4,698,895 |
Employment Inducement Plan | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
Share reserved for future issuance | 436,063 |
Stock-Based Awards - Summary of
Stock-Based Awards - Summary of Stock-Based Compensation Expense (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||
Stock-based compensation expense | $ 29,387 | $ 24,298 |
Cost of sales | ||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||
Stock-based compensation expense | 175 | 379 |
Research and development | ||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||
Stock-based compensation expense | 16,907 | 13,489 |
Selling, general and administrative | ||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||
Stock-based compensation expense | $ 12,305 | $ 10,430 |
Net Loss Per Share - Outstandin
Net Loss Per Share - Outstanding Shares of Common Stock Equivalents Excluded from Computation of Diluted Net Loss per Share (Details) - shares | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Common stock equivalents excluded from computation of diluted net loss per share | 9,386,957 | 8,118,221 |
Options to Purchase Common Stock, RSUs, and PSUs | ||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Common stock equivalents excluded from computation of diluted net loss per share | 9,363,681 | 8,084,585 |
Employee Stock Purchase Plan | ||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Common stock equivalents excluded from computation of diluted net loss per share | 23,276 | 33,636 |
Equity Transactions - Additiona
Equity Transactions - Additional Information (Details) - USD ($) $ in Millions | 1 Months Ended | 3 Months Ended |
May 31, 2021 | Mar. 31, 2022 | |
Stockholders Equity [Line Items] | ||
Stock sold in at-the-market offering | 1,050,372 | |
Net proceeds from at-the-market offering | $ 78.9 | |
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 Loss (Details) - USD ($) $ in Thousands | Mar. 31, 2022 | Dec. 31, 2021 |
Accumulated Other Comprehensive Income (Loss), Net of Tax [Abstract] | ||
Foreign currency translation adjustments | $ (181) | $ (121) |
Unrealized loss on available-for-sale securities | (6,373) | (1,283) |
Total accumulated other comprehensive loss | $ (6,554) | $ (1,404) |