Document and Entity Information
Document and Entity Information - shares | 6 Months Ended | |
Jun. 30, 2024 | Jul. 29, 2024 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Jun. 30, 2024 | |
Document Fiscal Year Focus | 2024 | |
Document Fiscal Period Focus | Q2 | |
Trading Symbol | RARE | |
Entity Registrant Name | ULTRAGENYX PHARMACEUTICAL INC. | |
Entity Central Index Key | 0001515673 | |
Entity Current Reporting Status | Yes | |
Current Fiscal Year End Date | --12-31 | |
Entity Filer Category | Large Accelerated Filer | |
Entity Small Business | false | |
Entity Emerging Growth Company | false | |
Entity Common Stock, Shares Outstanding | 92,165,190 | |
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, $0.001 par value | |
Security Exchange Name | NASDAQ | |
Entity Incorporation, State or Country Code | DE | |
Document Quarterly Report | true | |
Document Transition Report | false |
CONDENSED CONSOLIDATED BALANCE
CONDENSED CONSOLIDATED BALANCE SHEETS (Unaudited) - USD ($) $ in Thousands | Jun. 30, 2024 | Dec. 31, 2023 |
Current assets: | ||
Cash and cash equivalents | $ 480,693 | $ 213,584 |
Marketable debt securities | 283,128 | 363,625 |
Accounts receivable, net | 105,982 | 73,390 |
Inventory | 40,137 | 33,969 |
Other assets | 54,700 | 47,616 |
Total current assets | 964,640 | 732,184 |
Property, plant, and equipment, net | 278,707 | 290,566 |
Marketable debt securities | 110,669 | 199,901 |
Intangible assets, net | 166,243 | 166,271 |
Goodwill | 44,406 | 44,406 |
Other assets | 53,772 | 57,685 |
Total assets | 1,618,437 | 1,491,013 |
Current liabilities: | ||
Accounts payable | 59,807 | 42,114 |
Accrued liabilities | 154,421 | 196,486 |
Lease liabilities | 11,717 | 12,595 |
Liabilities for sales of future royalties | 46,921 | 29,242 |
Total current liabilities | 272,866 | 280,437 |
Lease liabilities | 25,741 | 30,574 |
Deferred tax liabilities | 30,058 | 30,058 |
Liabilities for sales of future royalties | 842,157 | 862,325 |
Other liabilities | 15,197 | 12,205 |
Total liabilities | 1,186,019 | 1,215,599 |
Stockholders’ equity: | ||
Preferred stock, par value of $0.001 per share -25,000,000 shares authorized; nil outstanding in 2024 and in 2023 | 0 | 0 |
Common stock, par value of $0.001 per share -250,000,000 shares authorized; outstanding - 92,135,384 in 2024 and 82,315,590 in 2023 | 92 | 82 |
Treasury stock, at cost, 65,063 in 2024 and 9,559 in 2023 | (3,395) | (432) |
Deferred compensation obligation | 3,395 | 432 |
Additional paid-in capital | 4,123,364 | 3,662,346 |
Accumulated other comprehensive income (loss) | (1,095) | 647 |
Accumulated deficit | (3,689,943) | (3,387,661) |
Total stockholders’ equity | 432,418 | 275,414 |
Total liabilities and stockholders’ equity | $ 1,618,437 | $ 1,491,013 |
CONDENSED CONSOLIDATED BALANC_2
CONDENSED CONSOLIDATED BALANCE SHEETS (Parenthetical) (Unaudited) - $ / shares | Jun. 30, 2024 | Dec. 31, 2023 |
Statement of Financial Position [Abstract] | ||
Preferred stock, par value | $ 0.001 | $ 0.001 |
Preferred stock, shares authorized | 25,000,000 | 25,000,000 |
Preferred stock, shares outstanding | 0 | 0 |
Common stock, par value | $ 0.001 | $ 0.001 |
Common stock, shares authorized | 250,000,000 | 250,000,000 |
Common stock, shares outstanding | 92,135,384 | 82,315,590 |
Treasury stock , shares | 65,063 | 9,559 |
CONDENSED CONSOLIDATED STATEMEN
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2024 | Jun. 30, 2023 | Jun. 30, 2024 | Jun. 30, 2023 | |
Revenues: | ||||
Total revenues | $ 147,026 | $ 108,309 | $ 255,859 | $ 208,805 |
Operating expenses: | ||||
Cost of sales | 21,280 | 9,914 | 38,813 | 22,171 |
Research and development | 161,503 | 164,949 | 339,990 | 330,647 |
Selling, general and administrative | 80,604 | 81,403 | 158,764 | 158,049 |
Total operating expenses | 263,387 | 256,266 | 537,567 | 510,867 |
Loss from operations | (116,361) | (147,957) | (281,708) | (302,062) |
Interest income | 7,401 | 5,964 | 16,225 | 12,254 |
Change in fair value of equity investments | (3,991) | 261 | (245) | (73) |
Non-cash interest expense on liabilities for sales of future royalties | (15,960) | (15,375) | (31,807) | (31,011) |
Other expense | (1,829) | (1,989) | (3,434) | (1,681) |
Loss before income taxes | (130,740) | (159,096) | (300,969) | (322,573) |
Provision for income taxes | (858) | (732) | (1,313) | (1,227) |
Net loss | $ (131,598) | $ (159,828) | $ (302,282) | $ (323,800) |
Net loss per share, basic | $ (1.52) | $ (2.25) | $ (3.54) | $ (4.58) |
Net loss per share, diluted | $ (1.52) | $ (2.25) | $ (3.54) | $ (4.58) |
Shares used in computing net loss per share, Basic | 86,580,516 | 70,897,991 | 85,433,443 | 70,639,015 |
Shares used in computing net loss per share, Diluted | 86,580,516 | 70,897,991 | 85,433,443 | 70,639,015 |
Product sales | ||||
Revenues: | ||||
Total revenues | $ 73,805 | $ 42,179 | $ 136,294 | $ 86,408 |
Royalty revenue | ||||
Revenues: | ||||
Total revenues | 73,221 | 46,331 | 119,565 | 51,213 |
Collaboration and license | ||||
Revenues: | ||||
Total revenues | $ 0 | $ 19,799 | $ 0 | $ 71,184 |
CONDENSED CONSOLIDATED STATEM_2
CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2024 | Jun. 30, 2023 | Jun. 30, 2024 | Jun. 30, 2023 | |
Statement of Comprehensive Income [Abstract] | ||||
Net loss | $ (131,598) | $ (159,828) | $ (302,282) | $ (323,800) |
Other comprehensive income (loss): | ||||
Foreign currency translation adjustments | (219) | 293 | (90) | 437 |
Unrealized gain (loss) on available-for-sale securities | (445) | 576 | (1,652) | 3,038 |
Other comprehensive income (loss): | (664) | 869 | (1,742) | 3,475 |
Total comprehensive loss | $ (132,262) | $ (158,959) | $ (304,024) | $ (320,325) |
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 | Treasury Stock | Deferred Compensation Obligation |
Beginning balance at Dec. 31, 2022 | $ 352,494 | $ 70 | $ 3,140,019 | $ (6,573) | $ (2,781,022) | ||
Beginning balance, shares at Dec. 31, 2022 | 70,197,297 | ||||||
Issuance of common stock in connection with at-the-market offering, net of issuance costs | 28,507 | 28,507 | |||||
Issuance of common stock in connection with at-the-market offering, net of issuance costs, shares | 564,302 | ||||||
Stock-based compensation | 64,535 | 64,535 | |||||
Issuance of common stock under equity plan awards, net of tax | 3,819 | $ 1 | 3,818 | ||||
Issuance of common stock under equity plan awards, net of tax, shares | 703,825 | ||||||
Deferred compensation | $ (381) | $ 381 | |||||
Other comprehensive income (loss) | 3,475 | 3,475 | |||||
Net Income (Loss) | (323,800) | (323,800) | |||||
Ending balance at Jun. 30, 2023 | 129,030 | $ 71 | 3,236,879 | (3,098) | (3,104,822) | (381) | 381 |
Ending balance, shares at Jun. 30, 2023 | 71,465,424 | ||||||
Beginning balance at Mar. 31, 2023 | 220,481 | $ 71 | 3,169,371 | (3,967) | (2,944,994) | (356) | 356 |
Beginning balance, shares at Mar. 31, 2023 | 70,660,249 | ||||||
Issuance of common stock in connection with at-the-market offering, net of issuance costs | 28,507 | 28,507 | |||||
Issuance of common stock in connection with at-the-market offering, net of issuance costs, shares | 564,302 | ||||||
Stock-based compensation | 34,432 | 34,432 | |||||
Issuance of common stock under equity plan awards, net of tax | 4,569 | 4,569 | |||||
Issuance of common stock under equity plan awards, net of tax, shares | 240,873 | ||||||
Deferred compensation | (25) | 25 | |||||
Other comprehensive income (loss) | 869 | 869 | |||||
Net Income (Loss) | (159,828) | (159,828) | |||||
Ending balance at Jun. 30, 2023 | 129,030 | $ 71 | 3,236,879 | (3,098) | (3,104,822) | (381) | 381 |
Ending balance, shares at Jun. 30, 2023 | 71,465,424 | ||||||
Beginning balance at Dec. 31, 2023 | $ 275,414 | $ 82 | 3,662,346 | 647 | (3,387,661) | (432) | 432 |
Beginning balance, shares at Dec. 31, 2023 | 82,315,590 | 82,315,590 | |||||
Issuance of common stock and pre-funded warrants in connection with the underwritten public offering, net | $ 380,866 | $ 9 | 380,857 | ||||
Issuance of common stock and pre-funded warrants in connection with the underwritten public offering, net, shares | 8,782,051 | ||||||
Stock-based compensation | 76,147 | 76,147 | |||||
Issuance of common stock under equity plan awards, net of tax | 4,015 | $ 1 | 4,014 | ||||
Issuance of common stock under equity plan awards, net of tax, shares | 1,037,743 | ||||||
Deferred compensation | (2,963) | 2,963 | |||||
Other comprehensive income (loss) | (1,742) | (1,742) | |||||
Net Income (Loss) | (302,282) | (302,282) | |||||
Ending balance at Jun. 30, 2024 | $ 432,418 | $ 92 | 4,123,364 | (1,095) | (3,689,943) | (3,395) | 3,395 |
Ending balance, shares at Jun. 30, 2024 | 92,135,384 | 92,135,384 | |||||
Beginning balance at Mar. 31, 2024 | $ 140,264 | $ 83 | 3,698,957 | (431) | (3,558,345) | (3,332) | 3,332 |
Beginning balance, shares at Mar. 31, 2024 | 83,094,037 | ||||||
Issuance of common stock and pre-funded warrants in connection with the underwritten public offering, net | 380,866 | $ 9 | 380,857 | ||||
Issuance of common stock and pre-funded warrants in connection with the underwritten public offering, net, shares | 8,782,051 | ||||||
Stock-based compensation | 39,476 | 39,476 | |||||
Issuance of common stock under equity plan awards, net of tax | 4,074 | $ 0 | 4,074 | ||||
Issuance of common stock under equity plan awards, net of tax, shares | 259,296 | ||||||
Deferred compensation | (63) | 63 | |||||
Other comprehensive income (loss) | (664) | (664) | |||||
Net Income (Loss) | (131,598) | (131,598) | |||||
Ending balance at Jun. 30, 2024 | $ 432,418 | $ 92 | $ 4,123,364 | $ (1,095) | $ (3,689,943) | $ (3,395) | $ 3,395 |
Ending balance, shares at Jun. 30, 2024 | 92,135,384 | 92,135,384 |
CONDENSED CONSOLIDATED STATEM_4
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS (Unaudited) - USD ($) $ in Thousands | 6 Months Ended | |
Jun. 30, 2024 | Jun. 30, 2023 | |
Operating activities: | ||
Net Income (Loss) | $ (302,282) | $ (323,800) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Stock-based compensation | 76,297 | 66,606 |
Amortization of discount on marketable debt securities, net | (6,703) | (6,213) |
Depreciation and amortization | 17,707 | 10,597 |
Change in fair value of equity investments | 245 | 73 |
Non-cash royalty revenue | (44,352) | (22,152) |
Non-cash interest expense on liabilities for sales of future royalties | 31,807 | 31,011 |
Other | (748) | 2,055 |
Changes in operating assets and liabilities: | ||
Accounts receivable | (22,750) | (17,586) |
Inventory | (6,251) | (1,507) |
Other assets | (449) | 11,594 |
Accounts payable, accrued, and other liabilities | (10,211) | (23,706) |
Net cash used in operating activities | (267,690) | (273,028) |
Investing activities: | ||
Purchase of property, plant, and equipment | (5,220) | (38,972) |
Purchase of marketable debt securities | (25,301) | (165,305) |
Proceeds from sale of marketable debt securities | 3,022 | 14,627 |
Proceeds from maturities of marketable debt securities | 197,104 | 407,221 |
Payments for intangible asset | (12,500) | (2,500) |
Other | (2,106) | (4,349) |
Net cash provided by investing activities | 154,999 | 210,722 |
Financing activities: | ||
Proceeds from the issuance of common stock and pre-funded warrants in connection with the underwritten public offering, net | 380,866 | 0 |
Proceeds from the issuance of common stock in connection with at-the-market offering, net | 0 | 28,507 |
Proceeds from the issuance of common stock under equity plan awards, net of tax | 4,015 | 3,819 |
Other | 0 | (28) |
Net cash provided by financing activities | 384,881 | 32,298 |
Effect of exchange rate changes on cash | (1,327) | 75 |
Net increase (decrease) in cash, cash equivalents and restricted cash | 270,863 | (29,933) |
Cash, cash equivalents and restricted cash at beginning of period | 219,399 | 137,601 |
Cash, cash equivalents and restricted cash at end of period | 490,262 | 107,668 |
Supplemental disclosures of non-cash information: | ||
Stock-based compensation capitalized into ending inventory | 3,004 | 2,303 |
Costs of property, plant and equipment included in accounts payable, accrued, and other liabilities | 899 | 6,063 |
Non-cash interest expense on liabilities for sales of future royalties capitalized during the year into ending property, plant and equipment | $ 0 | $ 7,969 |
Pay vs Performance Disclosure
Pay vs Performance Disclosure - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2024 | Jun. 30, 2023 | Jun. 30, 2024 | Jun. 30, 2023 | |
Pay vs Performance Disclosure | ||||
Net Income (Loss) | $ (131,598) | $ (159,828) | $ (302,282) | $ (323,800) |
Insider Trading Arrangements
Insider Trading Arrangements | 3 Months Ended |
Jun. 30, 2024 shares | |
Trading Arrangements, by Individual | |
Material Terms of Trading Arrangement | I tem 5. Other Information During the three months ended June 30, 2024, the following directors and Section 16 officers adopted a Rule 10b5-1 trading arrangement intended to satisfy the affirmative defense conditions of Rule 10b5-1(c). Name and Title Date Adopted Aggregate Number of Shares of Common Stock to be Sold (Subject to Certain Conditions) Plan End Date Dennis Huang EVP, Chief Technical Operations Officer, Gene Therapy Research & Development May 7, 2024 Up to 75,000 shares, all of which are shares to be acquired upon the exercise of stock options July 15, 2025 John Pinion Chief Quality Operations Officer and EVP, Translational Sciences May 7, 2024 Up to 109,730 shares, 107,800 of which are shares to be acquired upon the exercise of stock options May 7, 2025 |
Dennis Huang | |
Trading Arrangements, by Individual | |
Name | Dennis Huang |
Title | Chief Technical Operations Officer, Gene Therapy Research & Development |
Rule 10b5-1 Arrangement Adopted | true |
Adoption Date | May 7, 2024 |
Arrangement Duration | 434 days |
Aggregate Available | 75,000 |
Trading Arrangement Expiration Date | July 15, 2025 |
John Pinion | |
Trading Arrangements, by Individual | |
Name | John Pinion |
Title | Chief Quality Operations Officer and EVP, Translational Sciences |
Rule 10b5-1 Arrangement Adopted | true |
Adoption Date | May 7, 2024 |
Arrangement Duration | 365 days |
Aggregate Available | 109,730 |
Trading Arrangement Expiration Date | May 7, 2025 |
Plan 1 | John Pinion | |
Trading Arrangements, by Individual | |
Aggregate Available | 107,800 |
Organization
Organization | 6 Months Ended |
Jun. 30, 2024 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Organization | 1. Organization Ultragenyx Pharmaceutical Inc., or 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 ultrarare genetic diseases. The Company operates as one reportable segment and has four commercially approved products. Crysvita® (burosumab) is approved in the United States, or U.S., the European Union, or EU, and certain other regions for the treatment of X-linked hypophosphatemia, or XLH, in adult and pediatric patients one year of age and older. Crysvita is also approved in the U.S. and certain other regions for the treatment of fibroblast growth factor 23, or FGF23-related hypophosphatemia in tumor-induced osteomalacia, or 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 in the U.S., the EU and certain other regions, as the first medicine for the treatment of children and adults with mucopolysaccharidosis VII, or MPS VII, also known as Sly syndrome. Dojolvi® (triheptanoin) is approved in the U.S. and certain other regions for the treatment of pediatric and adult patients severely affected by long-chain fatty acid oxidation disorders, or LC-FAOD. Evkeeza® (evinacumab) is approved in the U.S. and the European Economic Area, or EEA, for the treatment of homozygous familial hypercholesterolemia, or HoFH. The Company has exclusive rights to commercialize Evkeeza® (evinacumab) outside of the U.S . In addition to the approved products, the Company has the following ongoing clinical development programs: • UX111 (formerly ABO-102) is an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; • DTX401 is an adeno-associated virus 8, or AAV8, gene therapy product candidate for the treatment of patients with glycogen storage disease type Ia, or GSDIa; • DTX301 is an AAV8 gene therapy product candidate in development for the treatment of patients with ornithine transcarbamylase, or OTC deficiency, the most common urea cycle disorder; • UX143 (setrusumab), which is subject to the Company ’ s collaboration agreement with Mereo BioPharma 3, or 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 Imperfecta, or OI; • GTX-102 is an antisense oligonucleotide, or ASO 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; and • UX701 is an adeno-associated virus 9, or AAV9, 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. 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. Through June 30, 2024, 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 may need to raise additional capital to fully implement its business plans through the issuance of equity, borrowings, or strategic alliances with partner companies. However, if such financing is not available at adequate levels, the Company would need to reevaluate its operating plans. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 6 Months Ended |
Jun. 30, 2024 | |
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, or 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 21, 2024, or Annual Report, with the United States Securities and Exchange Commission, or the SEC. The results of operations for the three and six months ended June 30, 2024 are not necessarily indicative of the results to be expected for the year ending December 31, 2024. The Condensed Consolidated Balance Sheet as of December 31, 2023 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 liabilities for sales 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 to guarantee the fulfillment of certain sales orders to certain government-sponsored customers. 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): June 30, 2024 2023 Cash and cash equivalents $ 480,693 $ 102,059 Restricted cash included in other current assets 6,172 1,696 Restricted cash included in other non-current assets 3,397 3,913 Total cash, cash equivalents, and restricted cash $ 490,262 $ 107,668 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 which were attributable to credit risk for the three and six months ended June 30, 2024 and 2023. Revenue Recognition 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 control is transferred 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 a material effect on earnings in the period of the adjustment. Collaboration, License, and Royalty Revenue The Company has certain license and collaboration agreements that are within the scope of Accounting Standards Codification, or 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 Statements of Operations, because the provision of such services for collaborative partners are not considered to be part of the Company’s ongoing major or central operations. The Company utilizes certain information from its collaboration partners to record collaboration revenue, 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 also records royalty revenues under certain of the Company’s license or collaboration agreements in exchange for licensing of intellectual property. The Company sold the right to receive certain royalty payments from net sales of Crysvita in certain territories to RPI Finance Trust, or RPI, an affiliate of Royalty Pharma, and to OCM LS23 Holdings LP, an investment vehicle for Ontario Municipal Employees Retirement System, or OMERS, as further described in “Note 8. Liabilities for Sales of Future Royalties.” The Company records the royalty revenue from the net sales of Crysvita in the applicable territories on a prospective basis as non-cash royalty revenue in the Condensed Consolidated Statements of Operations over the term of the applicable 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, or 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. |
Financial Instruments
Financial Instruments | 6 Months Ended |
Jun. 30, 2024 | |
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 investment in Solid Biosciences Inc., or 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 and liabilities remeasured on a recurring basis based on the three-tier fair value hierarchy (in thousands): June 30, 2024 Level 1 Level 2 Level 3 Total Financial Assets: Money market funds $ 427,247 $ — $ — $ 427,247 Certificates of deposit and time deposits — 15,986 — 15,986 Corporate bonds — 169,365 — 169,365 Commercial paper — 12,226 — 12,226 Asset-backed securities — 369 — 369 U.S. Government Treasury and agency securities 30,732 176,728 — 207,460 Investment in Solid common stock 2,959 — — 2,959 Deferred compensation assets — 13,712 — 13,712 Total financial assets $ 460,938 $ 388,386 $ — $ 849,324 Financial Liabilities: Deferred compensation liabilities $ — $ 13,959 $ — $ 13,959 December 31, 2023 Level 1 Level 2 Level 3 Total Financial Assets: Money market funds $ 162,289 $ — $ — $ 162,289 Certificates of deposit and time deposits — 17,986 — 17,986 Corporate bonds — 215,166 — 215,166 Commercial paper — 20,620 — 20,620 Asset-backed securities — 2,712 — 2,712 U.S. Government Treasury and agency securities 57,437 259,605 — 317,042 Investment in Solid common stock 3,204 — — 3,204 Deferred compensation assets — 10,220 — 10,220 Total financial assets $ 222,930 $ 526,309 $ — $ 749,239 Financial Liabilities: Deferred compensation liabilities $ — $ 10,365 $ — $ 10,365 |
Balance Sheet Components
Balance Sheet Components | 6 Months Ended |
Jun. 30, 2024 | |
Investments, Debt and Equity Securities [Abstract] | |
Balance Sheet Components | 4. Balance Sheet Components Cash Equivalents and Marketable Debt Securities The fair values of cash equivalents and marketable debt securities classified as available-for-sale securities consisted of the following (in thousands): June 30, 2024 Gross Unrealized Amortized Gains Losses Estimated Money market funds $ 427,247 $ — $ — $ 427,247 Certificates of deposit and time deposits 15,986 — — 15,986 Corporate bonds 169,414 139 ( 188 ) 169,365 Commercial paper 12,226 — — 12,226 Asset-backed securities 369 — — 369 U.S. Government Treasury and agency securities 207,810 1 ( 351 ) 207,460 Total $ 833,052 $ 140 $ ( 539 ) $ 832,653 December 31, 2023 Gross Unrealized Amortized Gains Losses Estimated Money market funds $ 162,289 $ — $ — $ 162,289 Certificates of deposit and time deposits 17,986 — — 17,986 Corporate bonds 214,792 711 ( 337 ) 215,166 Commercial paper 20,620 — — 20,620 Asset-backed securities 2,715 — ( 3 ) 2,712 U.S. Government Treasury and agency securities 316,160 982 ( 100 ) 317,042 Total $ 734,562 $ 1,693 $ ( 440 ) $ 735,815 At June 30, 2024 , 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 periods presented. Inventory Inventory consists of the following (in thousands): June 30, December 31, 2024 2023 Work-in-process $ 25,252 $ 18,859 Finished goods 14,885 15,110 Total inventory $ 40,137 $ 33,969 Accrued Liabilities Accrued liabilities consist of the following (in thousands): June 30, December 31, 2024 2023 Research, clinical study, and manufacturing expenses $ 36,325 $ 65,326 Payroll and related expenses 61,052 82,936 Revenue related reserves 27,972 17,029 Other 29,072 31,195 Total accrued liabilities $ 154,421 $ 196,486 |
Revenue
Revenue | 6 Months Ended |
Jun. 30, 2024 | |
Revenue from Contract with Customer [Abstract] | |
Revenue | 5. Revenue The following table disaggregates total revenues from external customers by product sales, royalty revenue, and collaboration and license revenue (in thousands): Three Months Ended Six Months Ended June 30, June 30, 2024 2023 2024 2023 Product sales: Crysvita $ 40,449 $ 16,884 $ 76,690 $ 38,118 Mepsevii 6,145 8,439 12,756 16,919 Dojolvi 19,355 16,491 35,717 30,794 Evkeeza 7,856 365 11,131 577 Total product sales 73,805 42,179 136,294 86,408 Crysvita royalty revenue 73,221 46,331 119,565 51,213 Collaboration and license revenue: Crysvita collaboration revenue in Profit- — 19,799 — 69,705 Daiichi Sankyo — — — 1,479 Total collaboration and license revenue — 19,799 — 71,184 Total revenues $ 147,026 $ 108,309 $ 255,859 $ 208,805 The following table disaggregates total revenues based on geographic location (in thousands): Three Months Ended Six Months Ended June 30, June 30, 2024 2023 2024 2023 North America $ 88,889 $ 79,289 $ 146,571 $ 146,421 Latin America 38,929 19,068 74,107 41,867 Europe, Middle East, and Africa, or EMEA 16,157 9,952 32,130 19,339 Asia-Pacific, or APAC 3,051 — 3,051 1,178 Total revenues $ 147,026 $ 108,309 $ 255,859 $ 208,805 The Company’s largest accounts receivable balance was from a collaboration partner and accounted for 63 % and 53 % of the total accounts receivable balance as of June 30, 2024 and December 31, 2023, respectively. A separate customer accounted for 12 % and 2 %, respectively, of the total accounts receivable balance as of June 30, 2024 and December 31, 2023, respectively. |
GeneTx Acquisition
GeneTx Acquisition | 6 Months Ended |
Jun. 30, 2024 | |
Business Combinations [Abstract] | |
Acquisition | 6. GeneTx Acquisition In August 2019, the Company entered into a Program Agreement and a Unitholder Option Agreement with GeneTx Biotherapeutics LLC, or GeneTx, to collaborate on the development of GeneTx’s GTX-102, an ASO for the treatment of Angelman syndrome. In July 2022, pursuant to the terms of the Unitholder Option Agreement, as amended, the Company exercised the option to acquire GeneTx and entered into a Unit Purchase Agreement, or the Purchase Agreement, pursuant to which the Company purchased all the outstanding units of GeneTx. In accordance with the terms of the Purchase Agreement, the Company paid the option exercise price of $ 75.0 million and an additional $ 15.6 million to acquire the outstanding cash of GeneTx, and adjustments for working capital and transaction expenses of $ 0.6 million, for a total purchase consideration of $ 91.2 million. The Company may be required to make payments of up to $ 190.0 million upon the achievement of certain milestones, including up to $ 30.0 million in milestone payments upon achievement of the earlier of initiation of a Phase 3 clinical study or product approvals in Canada and the U.K., up to $ 85.0 million in additional regulatory approval milestones for the achievement of U.S. and EU product approvals, and up to $ 75.0 million in commercial milestone payments based on annual worldwide net product sales. The Company will also pay tiered mid- to high single-digit percentage royalties based on licensed product annual net sales. If the Company receives and resells an FDA priority review voucher, or PRV, in connection with a new drug application approval, GeneTx is entitled to receive a portion of proceeds from the sale or a cash payment from the Company if the Company choses to retain the PRV. As part of the Company's acquisition of GeneTx, the Company assumed a License Agreement with Texas A&M University, or TAMU. Under this agreement, TAMU is eligible to receive from the Company up to $ 23.5 million upon the achievement of various future milestones, a nominal annual license fee that may increase up to a maximum of $ 2.0 million, as well as royalties in the mid-single-digits of net sales. The transaction was accounted as an asset acquisition, as substantially all of the fair value of the gross assets acquired was concentrated in a single identifiable in-process research and development intangible asset. Prior to the achievement of certain development and regulatory milestones, the acquired in-process research and development intangible asset has not yet reached technological feasibility and has no alternative future use. Accordingly, to date, amounts paid to GeneTx, net of cash and working capital acquired, were classified as in-process research and development expense. |
License and Research Agreements
License and Research Agreements | 6 Months Ended |
Jun. 30, 2024 | |
Research Grant Agreement [Abstract] | |
License and Research Agreements | 7. License and Research Agreements Kyowa Kirin Co., Ltd. In August 2013, the Company entered into a collaboration and license agreement with Kyowa Kirin Co., Ltd., or KKC. 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, except for ongoing studies being conducted by KKC, the Company was the lead party for development activities in the Profit-Share Territory and in the European Territory until the applicable transition date. The Company shared 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 was the transition date for the Profit-Share Territory, KKC became the lead party and became responsible for the costs of the subsequent development activities. However, the Company will continue to equally share in the costs of the studies with KKC that commenced prior to the applicable transition date. The collaboration and license agreements are within the scope of ASC 808, which provides guidance on the presentation and disclosure of collaborative arrangements. Collaboration and Royalty Revenue for Sales in the Profit-Share Territory The Company and KKC shared commercial responsibilities and profits in the Profit-Share Territory until April 2023. Under the collaboration agreement, KKC manufactured and supplied Crysvita for commercial use in the Profit-Share Territory and charged the Company a transfer price of 30 % of net sales in 2023, and 35 % prior to December 31, 2022. The remaining profit or loss after supply costs from commercializing products in the Profit-Share Territory was shared between the Company and KKC on a 50 / 50 basis until April 2023. In April 2023, commercialization responsibilities for Crysvita in the Profit-Share Territory transitioned to KKC. Thereafter, the Company is entitled to receive a tiered double-digit revenue share from the mid- 20 % range up to a maximum rate of 30 %. In 2022, the Company entered into an amendment to the collaboration agreement which granted the Company the right to continue to support KKC in commercial field activities in the U.S. through April 2024, subject to the limitations and conditions set forth in the amendment. The parties subsequently mutually agreed to extend the Company's right to continue to support KKC in commercial field activities in the U.S. through December 31, 2024, and as a result, the Company will continue to support commercial field efforts in the U.S. through a cost share arrangement through December 2024. After December 31, 2024, the Company’s rights to promote Crysvita in the U.S. will be limited to medical geneticists and the Company will solely bear its expenses for the promotion of Crysvita in the Profit-Share Territory. As KKC was the principal in the sale transaction with the customer during the profit-share period, the Company recognized a pro-rata share of collaboration revenue, net of transfer pricing, in the period the sale occurred. The Company concluded that its portion of KKC’s sales in the Profit-Share Territory prior to April 2023 was analogous to a royalty and therefore recorded its share as collaboration revenue, similar to a royalty. Starting in April 2023, the Company began to record the royalty revenue as the underlying sales occurred. In July 2022, the Company sold to OMERS its right to receive 30 % of the future royalty payments due to the Company based on net sales of Crysvita in the U.S. and Canada, subject to a cap, beginning in April 2023, as further described in Note 8. The Company records this revenue as royalty revenue. Product Sales Revenue for 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 for the sale of Crysvita once the product is delivered and the risk and title of the product is transferred to the distributor. 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 Latin American territories and charges the Company a transfer price of 30 % of net sales. The transfer price on these sales was 35 % prior to December 31, 2022. The Company also pays to KKC a low single-digit royalty on net sales in Latin America. Royalty Revenue for Sales in the 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 8. 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 records the royalty revenue as non-cash royalty revenues. Total Crysvita revenue was as follows (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 Revenue in Profit-Share Territory: Royalty revenue $ 46,932 $ 29,061 $ 75,213 $ 29,061 Non-cash royalty revenue 20,113 12,454 32,234 12,454 Collaboration revenue — 19,799 — 69,705 Total revenue in Profit-Share Territory 67,045 61,314 107,447 111,220 Product sales 40,449 16,884 76,690 38,118 Non-cash royalty revenue in European Territories 6,176 4,816 12,118 9,698 Total Crysvita revenue $ 113,670 $ 83,014 $ 196,255 $ 159,036 Collaboration Cost Sharing and Payments Under the collaboration agreement, KKC and the Company share certain development and commercialization costs and as a result, the Company was reimbursed for these costs and operating expenses were reduced. Additionally, KKC is owed a transfer price fee and royalties on certain revenues and the Company recorded amounts owed to KKC in cost of sales. These amounts were recognized in the Company’s Condensed Consolidated Statements of Operations in connection with the collaboration agreement with KKC as follows (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 Research and development $ ( 1,045 ) $ ( 1,453 ) $ ( 1,938 ) $ ( 3,725 ) Selling, general and administrative ( 1,024 ) ( 4,206 ) ( 2,286 ) ( 13,286 ) Cost of sales 12,626 3,986 23,703 9,528 Collaboration Receivable and Payable The Company had accounts receivable from KKC in the amount of $ 67.2 million and $ 39.2 million from profit-share revenue and royalties and other receivables recorded in other current assets of $ 2.3 million and $ 1.1 million and accrued liabilities of $ 12.9 million and $ 5.3 million from inventory, commercial, and development activity reimbursements, as of June 30, 2024 and December 31, 2023, respectively. Mereo In December 2020, the Company entered into a License and Collaboration Agreement with Mereo to collaborate on the development of setrusumab. Under the terms of the agreement, the Company leads 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 is responsible for post-marketing commitments and commercial supply in their respective territories. Upon the closing of the transactions under the License and Collaboration Agreement with Mereo in January 2021, the Company made a payment of $ 50.0 million to Mereo. In July 2023, the Company made a $ 9.0 million payment to Mereo upon the achievement of a clinical milestone. Going forward, the Company may be required to make payments of up to $ 245.0 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, 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. Regeneron In January 2022, the Company announced a collaboration with Regeneron Pharmaceuticals, 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 EU and U.K. 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 is obligated to pay up to $ 63.0 million in future milestone payments, contingent upon the achievement of certain regulatory and sales milestones. The Company may share in certain costs for global trials led by Regeneron and also received the right to opt into other potential indications. 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 recognizes product sales and cost of sales in the period the related sales occur and the related revenue recognition criteria are met. Under the collaboration agreement, Regeneron supplies the product and charges the Company a transfer price from the low 20 % range up to 40 % on net sales, which is recognized as cost of sales in the Company’s Condensed Consolidated Statement of Operations. The Company paid Regeneron a $ 30.0 million upfront payment upon the closing of the transaction in January 2022, a $ 10.0 million regulatory milestone payment in January 2024, and a $ 2.5 million regulatory milestone payment in May 2024. As these payments are for the Company’s use of intellectual property for Evkeeza for HoFH, they were recorded as intangible assets. Under the collaboration agreement, the Company was reimbursed by Regeneron for development costs of $ 3.3 million and $ 2.8 million, net, for the three and six months ended June 30, 2024 , respectively, recorded as offsets to research and development expense on the Condensed Consolidated Statements of Operations. The Company paid Regeneron $ 1.8 million and $ 5.0 million under this agreement for the three and six months ended June 30, 2023, respectively, recorded as research and development expense on the Condensed Consolidated Statements of Operations. The Company had collaboration receivables for this arrangement included in other current assets on the Condensed Consolidated Balance Sheets of $ 2.4 million and nil , and collaboration payables for this arrangement included in accrued liabilities on the Condensed Consolidated Balance Sheets of $ 1.0 million and $ 10.6 million as of June 30, 2024 and December 31, 2023, respectively. A dditionally, Regeneron is owed a transfer price fee and royalties of $ 2.0 million and $ 2.8 million was recorded in cost of sales on the Condensed Consolidated Statements of Operations for the three and six months ended June 30, 2024 , respectively, and $ 0.1 million for the three and six months ended June 30, 2023. Other Arrangements The Company has also entered into several collaborations and/or licensing arrangements in prior periods. Except as disclosed above, there have been no material changes in these arrangements during the three and six months ended June 30, 2024 as compared to those disclosed in "Note 8. License and Research Agreements" 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, the Company holds an equity interest in Solid in connection with its collaboration arrangement. The changes in the fair value of the Company’s equity investment in the common stock of Solid were as follows (in thousands): Common stock As of December 31, 2022 $ 2,807 Change in fair value 397 As of December 31, 2023 3,204 Change in fair value ( 245 ) As of June 30, 2024 $ 2,959 |
Liabilities for Sales of Future
Liabilities for Sales of Future Royalties | 6 Months Ended |
Jun. 30, 2024 | |
Deferred Revenue Disclosure [Abstract] | |
Liabilities for Sales of Future Royalties | 8. Liabilities for Sales 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 EU, the U.K., and Switzerland under the terms of the Company’s Collaboration and License Agreement with KKC dated August 29, 2013, as amended, or the KKC Collaboration Agreement. 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, or when aggregate royalty payments received by RPI are equal to $ 800.0 million. In July 2022, the Company entered into a Royalty Purchase Agreement with OMERS. Pursuant to the agreement, OMERS paid $ 500.0 million to the Company in consideration for the right to receive 30 % of the future royalty payments due to the Company from KKC based on net sales of Crysvita in the U.S. and Canada under the terms of the KKC Collaboration Agreement. The calculation of royalty payments to OMERS is based on net sales of Crysvita beginning in April 2023 and will expire upon the earlier of the date on which aggregate payments received by OMERS equals $ 725.0 million or the date the final royalty payment is made to the Company under the KKC Collaboration Agreement. Proceeds from these transactions were recorded as liabilities for sales of future royalties on the Condensed Consolidated Balance Sheets. Upon inception of the respective arrangements, the Company recorded $ 320.0 million and $ 500.0 million, net of transaction costs of $ 5.8 million and $ 9.1 million for RPI and OMERS, respectively, using the effective interest method over the estimated life of the applicable arrangement. In order to determine the amortization of the liabilities, the Company is required to estimate the total amount of future royalty payments to be received by the Company and paid to RPI and OMERS, subject to the capped amount, over the life of the arrangements. The excess of future estimated royalty payments (subject to the capped amount to RPI and OMERS), over the $ 314.2 million and $ 491.0 million, respectively, of net proceeds, is recorded as non-cash interest expense over the life of the arrangements. Consequently, the Company estimates an imputed interest on the unamortized portion of the liabilities and records interest expense relating to the transactions. The Company records the royalty revenue arising from the net sales of Crysvita in the applicable territories as royalty revenue in the Condensed Consolidated Statements of Operations over the term of the arrangements. Royalties earned under the RPI and OMERS arrangements from inception to June 30, 2024 have been $ 85.5 million and $ 80.8 million, respectively. 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 liabilities and the effective interest rate. The Company’s effective annual interest rates were 6.2 % and 7.7 %, for RPI and OMERS, respectively, as of June 30, 2024. There are a number of factors that could materially affect the amount and timing of royalty payments from KKC in the applicable 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, macroeconomic and inflationary pressures, the introduction of competing products, pricing for reimbursement in various 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, or USD, while significant portions of the underlying sales of Crysvita are made in currencies other than USD, and other events or circumstances that could result in reduced royalty payments from sales of Crysvita, all of which would result in a reduction of royalty revenue and the non-cash interest expense over the life of the arrangement. Conversely, if sales of Crysvita in the relevant territories are more than expected, the royalty revenue and the non-cash interest expense recorded by the Company would be greater over the term of the arrangements. The following table shows the activity within the liability account (in thousands): Liabilities for Sales of Future Royalties RPI OMERS Total December 31, 2022 $ 365,189 $ 510,250 $ 875,439 Royalty revenue ( 20,783 ) ( 38,524 ) ( 59,307 ) Non-cash interest expense 32,235 43,200 75,435 December 31, 2023 376,641 514,926 891,567 Royalty revenue ( 12,118 ) ( 22,178 ) ( 34,296 ) Non-cash interest expense 11,915 19,892 31,807 June 30, 2024 $ 376,438 $ 512,640 $ 889,078 |
Stock-Based Awards
Stock-Based Awards | 6 Months Ended |
Jun. 30, 2024 | |
Share-Based Payment Arrangement [Abstract] | |
Stock-Based Awards | 9. Stock-Based Awards As of June 30, 2024 , there were 1,711,846 shares available under the 2023 Incentive Plan, 6,567,545 shares available under the Amended & Restated 2014 Employee Stock Purchase Plan, and 23,673 shares available under the Employment Inducement Plan for the future issuance of equity awards. The table below sets forth the stock-based compensation expense for the periods presented (in thousands): Three Months Ended Six Months Ended 2024 2023 2024 2023 Cost of sales $ 269 $ 325 $ 685 $ 671 Research and development 21,674 19,138 42,215 37,258 Selling, general and administrative 17,420 15,190 33,397 28,663 Total stock-based compensation expense $ 39,363 $ 34,653 $ 76,297 $ 66,592 |
Net Loss Per Share
Net Loss Per Share | 6 Months Ended |
Jun. 30, 2024 | |
Earnings Per Share [Abstract] | |
Net Loss Per Share | 10. 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, pre-funded warrants, and treasury stock for deferred compensation obligations required to be settled in shares of common stock. Diluted net loss per share is calculated by dividing net loss by the weighted-average number of shares of common stock outstanding, pre-funded warrants, and treasury stock for deferred compensation obligations required to be settled in 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 Six Months Ended 2024 2023 2024 2023 Options to purchase common stock, 16,992,495 14,919,562 16,031,785 13,876,326 Employee stock purchase plan 24,405 23,966 12,002 12,049 17,016,900 14,943,528 16,043,787 13,888,375 |
Equity Transactions
Equity Transactions | 6 Months Ended |
Jun. 30, 2024 | |
Equity [Abstract] | |
Equity Transactions | 11. Equity Transactions At-the-Market Offerings In February 2024, the Company entered into a Sales Agreement with Cowen and Company, LLC, or Cowen, 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, or ATM, offerings through Cowen. No shares were sold under this agreement during the three and six months ended June 30, 2024. Underwritten Public Offering In October 2023, the Company completed an underwritten public offering in which 9,833,334 shares of common stock were sold, including the exercise in full by the underwriters of their option to purchase an additional 1,500,000 shares, at a public offering price of $ 30.00 per share. In connection with the offering, the Company sold to certain investors pre-funded warrants, in lieu of common stock, to purchase 1,666,722 shares of common stock at a purchase price of $ 29.999 per pre-funded warrant, which equals the public offering price per share of common stock less the $ 0.001 exercise price per share of each pre-funded warrant. The total proceeds that the Company received from the offering were $ 326.5 million, net of underwriting discounts and commissions. In June 2024, the Company completed an underwritten public offering in which 8,782,051 shares of common stock were sold, including the exercise in full by the underwriters of their option to purchase an additional 1,346,153 shares, at a public offering price of $ 39.00 per share. In connection with the offering, the Company sold to certain investors pre-funded warrants, in lieu of common stock, to purchase 1,538,501 shares of common stock at a purchase price of $ 38.999 per pre-funded warrant, which equals the public offering price per share of common stock less the $ 0.001 exercise price per share of each pre-funded warrant. The total proceeds that the Company received from the offering were $ 380.9 million, net of underwriting discounts and commissions. The pre-funded warrants were classified as a component of permanent equity in the Company's Condensed Consolidated Balance Sheets as they are freestanding financial instruments that are immediately exercisable, do not embody an obligation for the Company to repurchase its own shares and permit the holders to receive a fixed number of shares of common stock upon exercise. All of the shares underlying the pre-funded warrants have been included in the weighted-average number of shares of common stock used to calculate net loss per share, basic and diluted, attributable to common stockholders because the shares may be issued for little or no consideration, are fully vested, and are exercisable after the original issuance date of the pre-funded warrants. As of June 30, 2024 , no ne of the pre-funded warrants had been exercised. The table below summarizes the pre-funded warrants activity: Pre-funded warrants As of December 31, 2022 — Issuance of pre-funded warrants 1,666,722 As of December 31, 2023 1,666,722 Issuance of pre-funded warrants 1,538,501 As of June 30, 2024 3,205,223 |
Related Party Transaction
Related Party Transaction | 6 Months Ended |
Jun. 30, 2024 | |
Related Party Transactions [Abstract] | |
Related Party Transaction | 12. Related Party Transaction In July 2022, the Company entered into an agreement with a non-profit foundation in which two members of the Company’s board of directors, including the Company’s Chief Executive Officer, at the time also served as board members of the foundation, whereby an aggregate $ 1.0 million contribution is being paid to the foundation over a four-year period, beginning in the third quarter of 2022, to support rare disease education and awareness. As a result, the Company recorded nil and $ 0.3 million as research and development expense for this agreement for the three and six months ended June 30, 2024 , respectively, and $ 0.3 million for the three and six months ended June 30, 2023 . A total of $ 0.8 million has been recorded as research and development expense for this agreement to date. |
Accumulated Other Comprehensive
Accumulated Other Comprehensive Income (Loss) | 6 Months Ended |
Jun. 30, 2024 | |
Other Comprehensive Income (Loss), Net of Tax [Abstract] | |
Accumulated Other Comprehensive Loss | 13. Accumulated Other Comprehensive Income (Loss) Total accumulated other comprehensive income (loss) consisted of the following (in thousands): June 30, December 31, 2024 2023 Foreign currency translation adjustments $ ( 696 ) $ ( 606 ) Unrealized gain (loss) on available-for-sale securities ( 399 ) 1,253 Total accumulated other comprehensive income (loss) $ ( 1,095 ) $ 647 |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 6 Months Ended |
Jun. 30, 2024 | |
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, or 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 21, 2024, or Annual Report, with the United States Securities and Exchange Commission, or the SEC. The results of operations for the three and six months ended June 30, 2024 are not necessarily indicative of the results to be expected for the year ending December 31, 2024. The Condensed Consolidated Balance Sheet as of December 31, 2023 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 liabilities for sales 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 to guarantee the fulfillment of certain sales orders to certain government-sponsored customers. 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): June 30, 2024 2023 Cash and cash equivalents $ 480,693 $ 102,059 Restricted cash included in other current assets 6,172 1,696 Restricted cash included in other non-current assets 3,397 3,913 Total cash, cash equivalents, and restricted cash $ 490,262 $ 107,668 |
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 which were attributable to credit risk for the three and six months ended June 30, 2024 and 2023. |
Revenue Recognition | Revenue Recognition 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 control is transferred 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 a material effect on earnings in the period of the adjustment. Collaboration, License, and Royalty Revenue The Company has certain license and collaboration agreements that are within the scope of Accounting Standards Codification, or 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 Statements of Operations, because the provision of such services for collaborative partners are not considered to be part of the Company’s ongoing major or central operations. The Company utilizes certain information from its collaboration partners to record collaboration revenue, 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 also records royalty revenues under certain of the Company’s license or collaboration agreements in exchange for licensing of intellectual property. The Company sold the right to receive certain royalty payments from net sales of Crysvita in certain territories to RPI Finance Trust, or RPI, an affiliate of Royalty Pharma, and to OCM LS23 Holdings LP, an investment vehicle for Ontario Municipal Employees Retirement System, or OMERS, as further described in “Note 8. Liabilities for Sales of Future Royalties.” The Company records the royalty revenue from the net sales of Crysvita in the applicable territories on a prospective basis as non-cash royalty revenue in the Condensed Consolidated Statements of Operations over the term of the applicable 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, or 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. |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 6 Months Ended |
Jun. 30, 2024 | |
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): June 30, 2024 2023 Cash and cash equivalents $ 480,693 $ 102,059 Restricted cash included in other current assets 6,172 1,696 Restricted cash included in other non-current assets 3,397 3,913 Total cash, cash equivalents, and restricted cash $ 490,262 $ 107,668 |
Financial Instruments (Tables)
Financial Instruments (Tables) | 6 Months Ended |
Jun. 30, 2024 | |
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 and liabilities remeasured on a recurring basis based on the three-tier fair value hierarchy (in thousands): June 30, 2024 Level 1 Level 2 Level 3 Total Financial Assets: Money market funds $ 427,247 $ — $ — $ 427,247 Certificates of deposit and time deposits — 15,986 — 15,986 Corporate bonds — 169,365 — 169,365 Commercial paper — 12,226 — 12,226 Asset-backed securities — 369 — 369 U.S. Government Treasury and agency securities 30,732 176,728 — 207,460 Investment in Solid common stock 2,959 — — 2,959 Deferred compensation assets — 13,712 — 13,712 Total financial assets $ 460,938 $ 388,386 $ — $ 849,324 Financial Liabilities: Deferred compensation liabilities $ — $ 13,959 $ — $ 13,959 December 31, 2023 Level 1 Level 2 Level 3 Total Financial Assets: Money market funds $ 162,289 $ — $ — $ 162,289 Certificates of deposit and time deposits — 17,986 — 17,986 Corporate bonds — 215,166 — 215,166 Commercial paper — 20,620 — 20,620 Asset-backed securities — 2,712 — 2,712 U.S. Government Treasury and agency securities 57,437 259,605 — 317,042 Investment in Solid common stock 3,204 — — 3,204 Deferred compensation assets — 10,220 — 10,220 Total financial assets $ 222,930 $ 526,309 $ — $ 749,239 Financial Liabilities: Deferred compensation liabilities $ — $ 10,365 $ — $ 10,365 |
Balance Sheet Components (Table
Balance Sheet Components (Tables) | 6 Months Ended |
Jun. 30, 2024 | |
Investments, Debt and Equity Securities [Abstract] | |
Summary of Cash Equivalents and Marketable Debt Securities Classified as Available For Sale Securities | The fair values of cash equivalents and marketable debt securities classified as available-for-sale securities consisted of the following (in thousands): June 30, 2024 Gross Unrealized Amortized Gains Losses Estimated Money market funds $ 427,247 $ — $ — $ 427,247 Certificates of deposit and time deposits 15,986 — — 15,986 Corporate bonds 169,414 139 ( 188 ) 169,365 Commercial paper 12,226 — — 12,226 Asset-backed securities 369 — — 369 U.S. Government Treasury and agency securities 207,810 1 ( 351 ) 207,460 Total $ 833,052 $ 140 $ ( 539 ) $ 832,653 December 31, 2023 Gross Unrealized Amortized Gains Losses Estimated Money market funds $ 162,289 $ — $ — $ 162,289 Certificates of deposit and time deposits 17,986 — — 17,986 Corporate bonds 214,792 711 ( 337 ) 215,166 Commercial paper 20,620 — — 20,620 Asset-backed securities 2,715 — ( 3 ) 2,712 U.S. Government Treasury and agency securities 316,160 982 ( 100 ) 317,042 Total $ 734,562 $ 1,693 $ ( 440 ) $ 735,815 |
Summary of Inventory | Inventory consists of the following (in thousands): June 30, December 31, 2024 2023 Work-in-process $ 25,252 $ 18,859 Finished goods 14,885 15,110 Total inventory $ 40,137 $ 33,969 |
Summary of Accrued Liabilities | Accrued liabilities consist of the following (in thousands): June 30, December 31, 2024 2023 Research, clinical study, and manufacturing expenses $ 36,325 $ 65,326 Payroll and related expenses 61,052 82,936 Revenue related reserves 27,972 17,029 Other 29,072 31,195 Total accrued liabilities $ 154,421 $ 196,486 |
Revenue (Tables)
Revenue (Tables) | 6 Months Ended |
Jun. 30, 2024 | |
Revenue from Contract with Customer [Abstract] | |
Summary of Disaggregation of Total Revenues | The following table disaggregates total revenues from external customers by product sales, royalty revenue, and collaboration and license revenue (in thousands): Three Months Ended Six Months Ended June 30, June 30, 2024 2023 2024 2023 Product sales: Crysvita $ 40,449 $ 16,884 $ 76,690 $ 38,118 Mepsevii 6,145 8,439 12,756 16,919 Dojolvi 19,355 16,491 35,717 30,794 Evkeeza 7,856 365 11,131 577 Total product sales 73,805 42,179 136,294 86,408 Crysvita royalty revenue 73,221 46,331 119,565 51,213 Collaboration and license revenue: Crysvita collaboration revenue in Profit- — 19,799 — 69,705 Daiichi Sankyo — — — 1,479 Total collaboration and license revenue — 19,799 — 71,184 Total revenues $ 147,026 $ 108,309 $ 255,859 $ 208,805 The following table disaggregates total revenues based on geographic location (in thousands): Three Months Ended Six Months Ended June 30, June 30, 2024 2023 2024 2023 North America $ 88,889 $ 79,289 $ 146,571 $ 146,421 Latin America 38,929 19,068 74,107 41,867 Europe, Middle East, and Africa, or EMEA 16,157 9,952 32,130 19,339 Asia-Pacific, or APAC 3,051 — 3,051 1,178 Total revenues $ 147,026 $ 108,309 $ 255,859 $ 208,805 |
License and Research Agreemen_2
License and Research Agreements (Tables) | 6 Months Ended |
Jun. 30, 2024 | |
Share of Collaboration and Royalty Revenue Related to Crysvita | Total Crysvita revenue was as follows (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 Revenue in Profit-Share Territory: Royalty revenue $ 46,932 $ 29,061 $ 75,213 $ 29,061 Non-cash royalty revenue 20,113 12,454 32,234 12,454 Collaboration revenue — 19,799 — 69,705 Total revenue in Profit-Share Territory 67,045 61,314 107,447 111,220 Product sales 40,449 16,884 76,690 38,118 Non-cash royalty revenue in European Territories 6,176 4,816 12,118 9,698 Total Crysvita revenue $ 113,670 $ 83,014 $ 196,255 $ 159,036 |
Schedule of Cost Sharing Payments | These amounts were recognized in the Company’s Condensed Consolidated Statements of Operations in connection with the collaboration agreement with KKC as follows (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 Research and development $ ( 1,045 ) $ ( 1,453 ) $ ( 1,938 ) $ ( 3,725 ) Selling, general and administrative ( 1,024 ) ( 4,206 ) ( 2,286 ) ( 13,286 ) Cost of sales 12,626 3,986 23,703 9,528 |
Solid and Arcturus | |
Schedule of Changes in Fair Value of Equity Investment | The changes in the fair value of the Company’s equity investment in the common stock of Solid were as follows (in thousands): Common stock As of December 31, 2022 $ 2,807 Change in fair value 397 As of December 31, 2023 3,204 Change in fair value ( 245 ) As of June 30, 2024 $ 2,959 |
Liabilities for Sales of Futu_2
Liabilities for Sales of Future Royalties (Tables) | 6 Months Ended |
Jun. 30, 2024 | |
Deferred Revenue Disclosure [Abstract] | |
Schedule of Activity Within Liability for Sale of Future Royalties | The following table shows the activity within the liability account (in thousands): Liabilities for Sales of Future Royalties RPI OMERS Total December 31, 2022 $ 365,189 $ 510,250 $ 875,439 Royalty revenue ( 20,783 ) ( 38,524 ) ( 59,307 ) Non-cash interest expense 32,235 43,200 75,435 December 31, 2023 376,641 514,926 891,567 Royalty revenue ( 12,118 ) ( 22,178 ) ( 34,296 ) Non-cash interest expense 11,915 19,892 31,807 June 30, 2024 $ 376,438 $ 512,640 $ 889,078 |
Stock-Based Awards (Tables)
Stock-Based Awards (Tables) | 6 Months Ended |
Jun. 30, 2024 | |
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 Six Months Ended 2024 2023 2024 2023 Cost of sales $ 269 $ 325 $ 685 $ 671 Research and development 21,674 19,138 42,215 37,258 Selling, general and administrative 17,420 15,190 33,397 28,663 Total stock-based compensation expense $ 39,363 $ 34,653 $ 76,297 $ 66,592 |
Net Loss Per Share (Tables)
Net Loss Per Share (Tables) | 6 Months Ended |
Jun. 30, 2024 | |
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 Six Months Ended 2024 2023 2024 2023 Options to purchase common stock, 16,992,495 14,919,562 16,031,785 13,876,326 Employee stock purchase plan 24,405 23,966 12,002 12,049 17,016,900 14,943,528 16,043,787 13,888,375 |
Equity Transactions (Tables)
Equity Transactions (Tables) | 6 Months Ended |
Jun. 30, 2024 | |
Equity [Abstract] | |
Summary of Pre- Funded Warrants Activity | The table below summarizes the pre-funded warrants activity: Pre-funded warrants As of December 31, 2022 — Issuance of pre-funded warrants 1,666,722 As of December 31, 2023 1,666,722 Issuance of pre-funded warrants 1,538,501 As of June 30, 2024 3,205,223 |
Accumulated Other Comprehensi_2
Accumulated Other Comprehensive Income (Loss) (Tables) | 6 Months Ended |
Jun. 30, 2024 | |
Other Comprehensive Income (Loss), Net of Tax [Abstract] | |
Schedule of Total Accumulated Other Comprehensive Income (Loss) | Total accumulated other comprehensive income (loss) consisted of the following (in thousands): June 30, December 31, 2024 2023 Foreign currency translation adjustments $ ( 696 ) $ ( 606 ) Unrealized gain (loss) on available-for-sale securities ( 399 ) 1,253 Total accumulated other comprehensive income (loss) $ ( 1,095 ) $ 647 |
Organization - Additional Infor
Organization - Additional Information (Details) | 6 Months Ended |
Jun. 30, 2024 Segment | |
Organization And Nature Of Business [Line Items] | |
Number of reportable segments | 1 |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies - Additional Information (Details) - USD ($) $ in Thousands | 6 Months Ended | |
Jun. 30, 2024 | Jun. 30, 2023 | |
Accounting Policies [Abstract] | ||
Credit losses recorded for receivables | $ 0 | $ 0 |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies - Summary of Reconciliation of Cash, Cash Equivalents and Restricted Cash (Details) - USD ($) $ in Thousands | Jun. 30, 2024 | Dec. 31, 2023 | Jun. 30, 2023 | Dec. 31, 2022 |
Accounting Policies [Abstract] | ||||
Cash and cash equivalents | $ 480,693 | $ 213,584 | $ 102,059 | |
Restricted cash included in other current assets | $ 6,172 | $ 1,696 | ||
Restricted Cash and Cash Equivalents, Current, Asset, Statement of Financial Position [Extensible List] | Other assets | Other assets | ||
Restricted cash included in other non-current assets | $ 3,397 | $ 3,913 | ||
Restricted Cash and Cash Equivalents, Noncurrent, Asset, Statement of Financial Position [Extensible List] | Other assets | Other assets | ||
Total cash, cash equivalents, and restricted cash shown in the statements of cash flows | $ 490,262 | $ 219,399 | $ 107,668 | $ 137,601 |
Financial Instruments - Summary
Financial Instruments - Summary of Financial Assets Measured on Recurring Basis (Details) - USD ($) $ in Thousands | Jun. 30, 2024 | Dec. 31, 2023 |
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | $ 849,324 | $ 749,239 |
Level 1 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 460,938 | 222,930 |
Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 388,386 | 526,309 |
Money Market Funds | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 427,247 | 162,289 |
Money Market Funds | Level 1 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 427,247 | 162,289 |
Asset-backed Securities | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 369 | 2,712 |
Asset-backed Securities | Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 369 | 2,712 |
Certificates of Deposit and Time Deposits | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 15,986 | 17,986 |
Certificates of Deposit and Time Deposits | Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 15,986 | 17,986 |
Corporate Bonds | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 169,365 | 215,166 |
Corporate Bonds | Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 169,365 | 215,166 |
Commercial Paper | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 12,226 | 20,620 |
Commercial Paper | Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 12,226 | 20,620 |
U.S. Government Treasury and Agency Securities | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 207,460 | 317,042 |
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 | 30,732 | 57,437 |
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 | 176,728 | 259,605 |
Investments in Solid Common Stock | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 2,959 | 3,204 |
Investments in Solid Common Stock | Level 1 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 2,959 | 3,204 |
Deferred Compensation Assets | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 13,712 | 10,220 |
Deferred Compensation Assets | Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 13,712 | 10,220 |
Deferred Compensation Liabilities | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | 13,959 | 10,365 |
Deferred Compensation Liabilities | Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total fair value | $ 13,959 | $ 10,365 |
Balance Sheet Components - Summ
Balance Sheet Components - Summary of Cash Equivalents and Marketable Debt Securities Classified as Available For Sale Securities (Details) - USD ($) $ in Thousands | Jun. 30, 2024 | Dec. 31, 2023 |
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | $ 833,052 | $ 734,562 |
Gross Unrealized Gains | 140 | 1,693 |
Gross Unrealized Losses | (539) | (440) |
Estimated Fair Value | 832,653 | 735,815 |
Money Market Funds | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 427,247 | 162,289 |
Estimated Fair Value | 427,247 | 162,289 |
Asset-backed Securities | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 369 | 2,715 |
Gross Unrealized Losses | (3) | |
Estimated Fair Value | 369 | 2,712 |
Certificates of Deposit and Time Deposits | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 15,986 | 17,986 |
Estimated Fair Value | 15,986 | 17,986 |
Corporate Bonds | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 169,414 | 214,792 |
Gross Unrealized Gains | 139 | 711 |
Gross Unrealized Losses | (188) | (337) |
Estimated Fair Value | 169,365 | 215,166 |
Commercial Paper | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 12,226 | 20,620 |
Estimated Fair Value | 12,226 | 20,620 |
U.S. Government Treasury and Agency Securities | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 207,810 | 316,160 |
Gross Unrealized Gains | 1 | 982 |
Gross Unrealized Losses | (351) | (100) |
Estimated Fair Value | $ 207,460 | $ 317,042 |
Balance Sheet Components - Addi
Balance Sheet Components - Additional Information (Details) | 6 Months Ended |
Jun. 30, 2024 | |
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 | Jun. 30, 2024 | Dec. 31, 2023 |
Inventory, Net [Abstract] | ||
Work-in-process | $ 25,252 | $ 18,859 |
Finished goods | 14,885 | 15,110 |
Total inventory | $ 40,137 | $ 33,969 |
Balance Sheet Components - Su_3
Balance Sheet Components - Summary of Accrued Liabilities (Details) - USD ($) $ in Thousands | Jun. 30, 2024 | Dec. 31, 2023 |
Payables and Accruals [Abstract] | ||
Research, clinical study, and manufacturing expenses | $ 36,325 | $ 65,326 |
Payroll and related expenses | 61,052 | 82,936 |
Revenue related reserves | 27,972 | 17,029 |
Other | 29,072 | 31,195 |
Total accrued liabilities | $ 154,421 | $ 196,486 |
Revenue - Summary of Disaggrega
Revenue - Summary of Disaggregation of Total Revenues from Customers (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2024 | Jun. 30, 2023 | Jun. 30, 2024 | Jun. 30, 2023 | |
Disaggregation Of Revenue [Line Items] | ||||
Total revenues | $ 147,026 | $ 108,309 | $ 255,859 | $ 208,805 |
Collaboration and license | ||||
Disaggregation Of Revenue [Line Items] | ||||
Total revenues | 0 | 19,799 | 0 | 71,184 |
Collaboration and license | Crysvita | ||||
Disaggregation Of Revenue [Line Items] | ||||
Total revenues | 0 | 19,799 | 0 | 69,705 |
Collaboration and license | Daiichi Sankyo | ||||
Disaggregation Of Revenue [Line Items] | ||||
Total revenues | 0 | 0 | 0 | 1,479 |
Product Revenue | ||||
Disaggregation Of Revenue [Line Items] | ||||
Total revenues | 73,805 | 42,179 | 136,294 | 86,408 |
Product Revenue | Crysvita | ||||
Disaggregation Of Revenue [Line Items] | ||||
Total revenues | 40,449 | 16,884 | 76,690 | 38,118 |
Product Revenue | Mepsevii | ||||
Disaggregation Of Revenue [Line Items] | ||||
Total revenues | 6,145 | 8,439 | 12,756 | 16,919 |
Product Revenue | Dojolvi | ||||
Disaggregation Of Revenue [Line Items] | ||||
Total revenues | 19,355 | 16,491 | 35,717 | 30,794 |
Product Revenue | Evkeeza | ||||
Disaggregation Of Revenue [Line Items] | ||||
Total revenues | 7,856 | 365 | 11,131 | 577 |
Royalty revenue | ||||
Disaggregation Of Revenue [Line Items] | ||||
Total revenues | 73,221 | 46,331 | 119,565 | 51,213 |
Royalty revenue | Crysvita | ||||
Disaggregation Of Revenue [Line Items] | ||||
Total revenues | $ 73,221 | $ 46,331 | $ 119,565 | $ 51,213 |
Revenue - Summary of Disaggre_2
Revenue - Summary of Disaggregation of Total Revenues (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2024 | Jun. 30, 2023 | Jun. 30, 2024 | Jun. 30, 2023 | |
Disaggregation Of Revenue [Line Items] | ||||
Total revenues | $ 147,026 | $ 108,309 | $ 255,859 | $ 208,805 |
North America | ||||
Disaggregation Of Revenue [Line Items] | ||||
Total revenues | 88,889 | 79,289 | 146,571 | 146,421 |
Latin America | ||||
Disaggregation Of Revenue [Line Items] | ||||
Total revenues | 38,929 | 19,068 | 74,107 | 41,867 |
Europe, Middle East, and Africa, or EMEA | ||||
Disaggregation Of Revenue [Line Items] | ||||
Total revenues | 16,157 | 9,952 | 32,130 | 19,339 |
Asia-Pacific, or APAC | ||||
Disaggregation Of Revenue [Line Items] | ||||
Total revenues | $ 3,051 | $ 0 | $ 3,051 | $ 1,178 |
Revenue - Additional Informatio
Revenue - Additional Information (Details) | Jun. 30, 2024 | Dec. 31, 2023 |
Customer One | ||
Disaggregation of Revenue [Line Items] | ||
Percentage of gross accounts receivable balance | 63% | 53% |
Customer Two | ||
Disaggregation of Revenue [Line Items] | ||
Percentage of gross accounts receivable balance | 12% | 2% |
GeneTx Acquisition - Additional
GeneTx Acquisition - Additional Information (Details) $ in Millions | 1 Months Ended |
Jul. 31, 2022 USD ($) | |
GeneTx [Member] | |
Business Acquisition [Line Items] | |
Business acquisition, consideration transferred | $ 75 |
Outstanding Cash and Cash Equivalents Acquired | 15.6 |
Amount on adjustments for working capital and transaction expenses | 0.6 |
Payments to Acquire Businesses, Gross | 91.2 |
GeneTx [Member] | Maximum | |
Business Acquisition [Line Items] | |
Future contingent milestone payments | 190 |
Additional Milestone Payments | 30 |
Regulatory Approval Milestones | 85 |
Commercial Milestone Payment | 75 |
Texas AM University Member | Maximum | |
Business Acquisition [Line Items] | |
Future contingent milestone payments | 23.5 |
Annual license fee payment | $ 2 |
License and Research Agreemen_3
License and Research Agreements - Additional Information (Details) - USD ($) $ in Thousands | 1 Months Ended | 3 Months Ended | 6 Months Ended | 113 Months Ended | ||||||||
Jul. 01, 2022 | May 31, 2024 | Jan. 31, 2024 | Jul. 31, 2023 | Jan. 31, 2022 | Jan. 31, 2021 | Jun. 30, 2024 | Jun. 30, 2023 | Jun. 30, 2024 | Jun. 30, 2023 | Dec. 31, 2022 | Dec. 31, 2023 | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Research and development | $ 161,503 | $ 164,949 | $ 339,990 | $ 330,647 | ||||||||
Cost of sales | 21,280 | 9,914 | 38,813 | 22,171 | ||||||||
Regeneron Pharmaceuticals, Inc. | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Upfront payments of collaboration | $ 30,000 | |||||||||||
Eligible future milestone payments | $ 63,000 | |||||||||||
Reimbursed development costs | 3,300 | 2,800 | ||||||||||
Development costs | 1,800 | 5,000 | ||||||||||
AccruedCollaborationPayables | 1,000 | 1,000 | $ 10,600 | |||||||||
Regulatory milestone payments achieved | $ 2,500 | $ 10,000 | ||||||||||
Cost of sales | 2,000 | 100 | 2,800 | 100 | ||||||||
Regeneron Pharmaceuticals, Inc. | Maximum | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Profit loss sharing percentage on net sales | 40% | |||||||||||
Regeneron Pharmaceuticals, Inc. | Minimum | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Profit loss sharing percentage on net sales | 20% | |||||||||||
Other Current Assets | Regeneron Pharmaceuticals, Inc. | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
License agreement other receivables | 2,400 | 2,400 | 0 | |||||||||
License and Collaboration Agreement | Mereo | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Payments made under agreement | $ 50,000 | |||||||||||
Research and development | $ 9,000 | |||||||||||
License and Collaboration Agreement | Maximum | Mereo | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Future contingent milestone payments | 245,000 | 245,000 | ||||||||||
Kyowa Kirin Collaboration | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Cost of sales | 12,626 | $ 3,986 | $ 23,703 | $ 9,528 | ||||||||
Kyowa Kirin Collaboration | License Agreement | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Profit loss sharing percentage on net sales | 30% | 35% | ||||||||||
Kyowa Kirin Collaboration | License Agreement | Maximum | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Tiered double-digit revenue share percentage entitled to receive | 30% | |||||||||||
Kyowa Kirin Collaboration | License Agreement | Minimum | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Tiered double-digit revenue share percentage entitled to receive | 20% | |||||||||||
Kyowa Kirin Collaboration | License Agreement | Other Current Assets | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
License agreement other receivables | 2,300 | $ 2,300 | 1,100 | |||||||||
Kyowa Kirin Collaboration | License Agreement | Profit Share Revenue | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
License agreement accounts receivable | 67,200 | 67,200 | 39,200 | |||||||||
Kyowa Kirin Collaboration | License Agreement | Commercial and Development Activity Reimbursements | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
License agreement accrued liabilities | $ 12,900 | $ 12,900 | $ 5,300 | |||||||||
Kyowa Kirin Collaboration | License Agreement | Profit Share Territory | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Percentage of right to receive royalty payments on net sales | 30% | |||||||||||
Remaining profit or loss share percentage on commercializing products | 50% | |||||||||||
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% |
License and Research Agreemen_4
License and Research Agreements - Share of Collaboration and Royalty Revenue Related to Crysvita (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2024 | Jun. 30, 2023 | Jun. 30, 2024 | Jun. 30, 2023 | |
Disaggregation Of Revenue [Line Items] | ||||
Total Crysvita revenue | $ 147,026 | $ 108,309 | $ 255,859 | $ 208,805 |
Royalty revenue | ||||
Disaggregation Of Revenue [Line Items] | ||||
Total Crysvita revenue | 73,221 | 46,331 | 119,565 | 51,213 |
Royalty revenue | Kyowa Kirin Collaboration | Profit Share Territory | ||||
Disaggregation Of Revenue [Line Items] | ||||
Total Crysvita revenue | 46,932 | 29,061 | 75,213 | 29,061 |
Non-cash royalty revenue | Kyowa Kirin Collaboration | Profit Share Territory | ||||
Disaggregation Of Revenue [Line Items] | ||||
Total Crysvita revenue | 20,113 | 12,454 | 32,234 | 12,454 |
Non-cash royalty revenue | Kyowa Kirin Collaboration | Royalty Revenue in European Territories | ||||
Disaggregation Of Revenue [Line Items] | ||||
Total Crysvita revenue | 6,176 | 4,816 | 12,118 | 9,698 |
Collaboration Revenue | Kyowa Kirin Collaboration | Profit Share Territory | ||||
Disaggregation Of Revenue [Line Items] | ||||
Total Crysvita revenue | 0 | 19,799 | 0 | 69,705 |
Collaboration and Royalty | Kyowa Kirin Collaboration | ||||
Disaggregation Of Revenue [Line Items] | ||||
Total Crysvita revenue | 113,670 | 83,014 | 196,255 | 159,036 |
Collaboration and Royalty | Kyowa Kirin Collaboration | Profit Share Territory | ||||
Disaggregation Of Revenue [Line Items] | ||||
Total Crysvita revenue | 67,045 | 61,314 | 107,447 | 111,220 |
Product sales | ||||
Disaggregation Of Revenue [Line Items] | ||||
Total Crysvita revenue | 73,805 | 42,179 | 136,294 | 86,408 |
Product sales | Kyowa Kirin Collaboration | ||||
Disaggregation Of Revenue [Line Items] | ||||
Total Crysvita revenue | $ 40,449 | $ 16,884 | $ 76,690 | $ 38,118 |
License and Research Agreemen_5
License and Research Agreements - Schedule of Cost Sharing Payments (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2024 | Jun. 30, 2023 | Jun. 30, 2024 | Jun. 30, 2023 | |
Cost Sharing Payments [Line Items] | ||||
Cost of sales | $ 21,280 | $ 9,914 | $ 38,813 | $ 22,171 |
Kyowa Kirin Collaboration | ||||
Cost Sharing Payments [Line Items] | ||||
Research and development | (1,045) | (1,453) | (1,938) | (3,725) |
Selling, general and administrative | (1,024) | (4,206) | (2,286) | (13,286) |
Cost of sales | $ 12,626 | $ 3,986 | $ 23,703 | $ 9,528 |
License and Research Agreemen_6
License and Research Agreements - Schedule of Changes in Fair Value of Equity Investment (Details) - USD ($) $ in Thousands | 6 Months Ended | 12 Months Ended |
Jun. 30, 2024 | Dec. 31, 2023 | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Common stock, Beginning balance | $ 749,239 | |
Common stock, Ending balance | 849,324 | $ 749,239 |
Solid and Arcturus | Common Stock | ||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Common stock, Beginning balance | 3,204 | 2,807 |
Common stock, Change in fair value | (245) | 397 |
Common stock, Ending balance | $ 2,959 | $ 3,204 |
Liabilities for Sales of Futu_3
Liabilities for Sales of Future Royalties - Additional Information (Details) - Royalty Purchase Agreement - USD ($) $ in Millions | 1 Months Ended | 6 Months Ended | 55 Months Ended | |
Jul. 31, 2022 | Dec. 31, 2019 | Jun. 30, 2024 | Jun. 30, 2024 | |
Liability For Sale Of Future Royalties [Line Items] | ||||
Profit loss sharing percentage on net sales | 30% | |||
RPI Finance Trust (RPI) | ||||
Liability For 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, or 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 | 6.20% | 6.20% | ||
Non-cash collaboration royalty revenue | $ 85.5 | |||
OMERS | ||||
Liability For Sale Of Future Royalties [Line Items] | ||||
Proceeds from sale of future royalties | $ 500 | |||
Royalty payment termination description | In July 2022, the Company entered into a Royalty Purchase Agreement with OMERS. Pursuant to the agreement, OMERS paid $500.0 million to the Company in consideration for the right to receive 30% of the future royalty payments due to the Company from KKC based on net sales of Crysvita in the U.S. and Canada under the terms of the KKC Collaboration Agreement. The calculation of royalty payments to OMERS is based on net sales of Crysvita beginning in April 2023 and will expire upon the earlier of the date on which aggregate payments received by OMERS equals $725.0 million or the date the final royalty payment is made to the Company under the KKC Collaboration Agreement. | |||
Royalty agreement termination threshold amount | $ 725 | |||
Payment from sale of future royalties | 500 | |||
Royalties transaction costs net | 9.1 | |||
Proceeds from the sale of future royalties, net | $ 491 | |||
Effective annual interest rate | 7.70% | 7.70% | ||
Non-cash collaboration royalty revenue | $ 80.8 | |||
Minimum | RPI Finance Trust (RPI) | ||||
Liability For Sale Of Future Royalties [Line Items] | ||||
Royalty agreement termination threshold amount | $ 608 | 608 | ||
Maximum | RPI Finance Trust (RPI) | ||||
Liability For Sale Of Future Royalties [Line Items] | ||||
Royalty agreement termination threshold amount | $ 800 | $ 800 |
Liabilities for Sales of Futu_4
Liabilities for Sales of Future Royalties - Schedule of Activity Within Liability Related to Sale of Future Royalties (Details) - USD ($) $ in Thousands | 6 Months Ended | 12 Months Ended |
Jun. 30, 2024 | Dec. 31, 2023 | |
Liability For Sale Of Future Royalties [Line Items] | ||
Liability for sale of future royalties at beginning of year | $ 891,567 | $ 875,439 |
Royalty revenue | (34,296) | (59,307) |
Non-cash interest expense | 31,807 | 75,435 |
Liability for sale of future royalties at end of year | 889,078 | 891,567 |
RPI Finance Trust (RPI) | ||
Liability For Sale Of Future Royalties [Line Items] | ||
Liability for sale of future royalties at beginning of year | 376,641 | 365,189 |
Royalty revenue | (12,118) | (20,783) |
Non-cash interest expense | 11,915 | 32,235 |
Liability for sale of future royalties at end of year | 376,438 | 376,641 |
OMERS | ||
Liability For Sale Of Future Royalties [Line Items] | ||
Liability for sale of future royalties at beginning of year | 514,926 | 510,250 |
Royalty revenue | (22,178) | (38,524) |
Non-cash interest expense | 19,892 | 43,200 |
Liability for sale of future royalties at end of year | $ 512,640 | $ 514,926 |
Stock-Based Awards - Additional
Stock-Based Awards - Additional Information (Details) | Jun. 30, 2024 shares |
Amended & Restated 2014 Employee Stock Purchase Plan | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
Share reserved for future issuance | 6,567,545 |
Employment Inducement Plan | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
Share reserved for future issuance | 23,673 |
2023 Plan | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
Share reserved for future issuance | 1,711,846 |
Stock-Based Awards - Summary of
Stock-Based Awards - Summary of Stock-Based Compensation Expense (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2024 | Jun. 30, 2023 | Jun. 30, 2024 | Jun. 30, 2023 | |
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||||
Stock-based compensation expense | $ 39,363 | $ 34,653 | $ 76,297 | $ 66,592 |
Cost of sales | ||||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||||
Stock-based compensation expense | 269 | 325 | 685 | 671 |
Research and development | ||||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||||
Stock-based compensation expense | 21,674 | 19,138 | 42,215 | 37,258 |
Selling, general and administrative | ||||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||||
Stock-based compensation expense | $ 17,420 | $ 15,190 | $ 33,397 | $ 28,663 |
Net Loss Per Share - Outstandin
Net Loss Per Share - Outstanding Shares of Common Stock Equivalents Excluded from Computation of Diluted Net Loss per Share (Details) - shares | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2024 | Jun. 30, 2023 | Jun. 30, 2024 | Jun. 30, 2023 | |
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||||
Common stock equivalents excluded from computation of diluted net loss per share | 17,016,900 | 14,943,528 | 16,043,787 | 13,888,375 |
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 | 16,992,495 | 14,919,562 | 16,031,785 | 13,876,326 |
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 | 24,405 | 23,966 | 12,002 | 12,049 |
Equity Transactions - Additiona
Equity Transactions - Additional Information (Details) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | 3 Months Ended | 6 Months Ended | 12 Months Ended | |||
Jun. 30, 2024 | Feb. 29, 2024 | Oct. 31, 2023 | Jun. 30, 2024 | Jun. 30, 2024 | Jun. 30, 2023 | Dec. 31, 2023 | |
Stockholders Equity [Line Items] | |||||||
Net proceeds from at-the-market offering | $ 0 | $ 28,507 | |||||
Pre Funded Warrant | |||||||
Stockholders Equity [Line Items] | |||||||
Underwritten Public Offering Shares | 1,538,501 | 1,666,722 | |||||
Number of warrants exercised | 0 | 0 | 0 | ||||
Open Market Sale | |||||||
Stockholders Equity [Line Items] | |||||||
Common stock shares sold | 0 | 0 | |||||
Option to sell common stock aggregate offering price | $ 350,000 | ||||||
Underwritten Public Offering [Member] | |||||||
Stockholders Equity [Line Items] | |||||||
Common stock shares sold | 8,782,051 | 9,833,334 | |||||
Shares purchased by underwriters | 1,346,153 | 1,500,000 | |||||
Public offering price | $ 39 | $ 30 | $ 39 | $ 39 | |||
Underwritten Public Offering [Member] | Pre Funded Warrant | |||||||
Stockholders Equity [Line Items] | |||||||
Underwritten Public Offering Shares | 1,538,501 | 1,666,722 | |||||
Pre-funded warrant price | $ 38.999 | $ 29.999 | 38.999 | 38.999 | |||
Common stock warrants, exercise price | $ 0.001 | $ 0.001 | $ 0.001 | $ 0.001 | |||
Net proceeds from sale of common stock | $ 380,900 | $ 326,500 |
Equity Transactions - Summary o
Equity Transactions - Summary of Pre- Funded Warrants Activity (Details) - Pre Funded Warrant - shares | 6 Months Ended | 12 Months Ended |
Jun. 30, 2024 | Dec. 31, 2023 | |
Stockholders Equity [Line Items] | ||
Beginning Balance | 1,666,722 | 0 |
Issuance of pre-funded warrants | 1,538,501 | 1,666,722 |
Ending Balance | 3,205,223 | 1,666,722 |
Related Party Transaction - Add
Related Party Transaction - Additional Information (Details) - USD ($) $ in Thousands | 1 Months Ended | 3 Months Ended | 6 Months Ended | 24 Months Ended | ||
Jul. 31, 2022 | Jun. 30, 2024 | Jun. 30, 2023 | Jun. 30, 2024 | Jun. 30, 2023 | Jun. 30, 2024 | |
Related Party Transaction [Line Items] | ||||||
Research and development | $ 161,503 | $ 164,949 | $ 339,990 | $ 330,647 | ||
Contribution amount paid | $ 1,000 | |||||
Contribution period | 4 years | |||||
Non Profit Foundation [Member] | ||||||
Related Party Transaction [Line Items] | ||||||
Research and development | $ 0 | $ 300 | $ 300 | $ 300 | $ 800 |
Accumulated Other Comprehensi_3
Accumulated Other Comprehensive Income (Loss) - Schedule of Total Accumulated Other Comprehensive Loss (Details) - USD ($) $ in Thousands | Jun. 30, 2024 | Dec. 31, 2023 |
Accumulated Other Comprehensive Income (Loss), Net of Tax [Abstract] | ||
Foreign currency translation adjustments | $ (696) | $ (606) |
Unrealized gain (loss) on available-for-sale securities | (399) | 1,253 |
Total accumulated other comprehensive income (loss) | $ (1,095) | $ 647 |