Ultragenyx Pharmaceutical(RARE) - 2024 Q4 - Annual Report

Financial Performance - For the year ended December 31, 2024, total revenues increased to $560.2 million, compared to$434.2 million for the same period in 2023, representing a growth of approximately 29% driven by higher demand for approved products [378]. - Net losses for the years ended December 31, 2024 and 2023 were $569.2 million and$606.6 million, respectively, indicating a reduction in losses of about 6.2% year-over-year [377]. - Total revenues for the year ended December 31, 2024, were $560.2 million, up 29$434.2 million in 2023, driven by a $105.0 million increase in product sales [410]. - Product sales for Crysvita increased by 78$134.7 million in 2024, while Dojolvi sales rose by 25% to $88.2 million [410]. - Selling, general and administrative expenses increased by$11.8 million, or 4%, to $321.6 million for the year ended December 31, 2024, compared to$309.8 million in 2023 [420]. - Interest income rose by $9.8 million, or 37$36.5 million for the year ended December 31, 2024, primarily due to higher marketable debt securities balances [422]. - The company recognized an income tax provision of $1.6 million for the year ended December 31, 2024, compared to an income tax benefit of$4.8 million in 2023, reflecting a change of 188% [426]. Cash and Investments - As of December 31, 2024, the company had $745.0 million in available cash, cash equivalents, and marketable debt securities [378]. - Cash used in operating activities for the year ended December 31, 2024, was$414.2 million, reflecting a net loss of $569.2 million [434]. - Cash provided by financing activities for the year ended December 31, 2024, was$399.2 million, primarily from a public offering that generated $381.0 million in net proceeds [439]. - The investment portfolio is primarily composed of low-risk, investment-grade debt instruments, including U.S. government treasury and agency securities [449]. - A hypothetical 100 basis point change in interest rates would not have had a material impact on the fair market value of cash equivalents and marketable debt securities as of December 31, 2024 [449]. - The company has not experienced a loss of principal on any investments to date and did not record any allowance for credit loss as of December 31, 2024 [449]. Research and Development - The company has incurred substantial costs related to research and development, which are a significant component of its net losses [377]. - Research and development expenses rose by 8$697.9 million in 2024, with significant increases in gene therapy programs and biologic and nucleic acid programs [418]. - The company expects annual research and development expenses to moderate as product candidates advance through clinical development [419]. - Upfront license, acquisition, and milestone fees increased by 238% to $30.5 million in 2024, primarily due to a clinical enrollment milestone achievement for the GTX-102 program [418]. Product Portfolio - The company has four commercially approved products, including Crysvita® and Evkeeza®, targeting serious rare genetic diseases [375]. - The company has a diverse pipeline of product candidates, including biologics and gene therapies, aimed at treating serious genetic diseases [375]. - The company transitioned commercial responsibilities for Crysvita in the Profit-Share Territory to KKC in April 2023, impacting revenue recognition [411]. Royalty Agreements - In December 2019, the company entered into a Royalty Purchase Agreement with RPI for$320.0 million for future royalty payments on Crysvita, with a capped amount of $608.0 million [397]. - In July 2022, the company entered into a Royalty Purchase Agreement with OMERS for$500.0 million for 30% of future royalty payments from Crysvita, with a cap of $725.0 million [398]. - The effective annual interest rates for the royalty agreements with RPI and OMERS were 6.2$158.1 million, an increase from $135.2 million in 2023, reflecting a growth of 17.5$76.7 million in 2024, primarily due to increased demand for approved products, especially Crysvita and Evkeeza [413]. - The change in fair value of equity investments recorded a net decrease of $1.1 million for the year ended December 31, 2024, compared to a net increase of$0.4 million in 2023, reflecting a change of 381% [423]. - Non-cash interest expense on liabilities for sales of future royalties decreased by $3.0 million, or 4$66.0 million for the year ended December 31, 2024 [424]. - Other expenses increased by $3.6 million for the year ended December 31, 2024, primarily due to fluctuations in foreign exchange rates [425]. Future Outlook - The company anticipates continued annual losses in the near term as it develops and seeks regulatory approvals for product candidates [441]. - Total gross deferred tax assets as of December 31, 2024, were$1,213.7 million, fully offset by a valuation allowance due to uncertainties in generating future taxable income [409]. Foreign Exchange Risk - The company faces foreign exchange risk due to transactions in currencies other than U.S. dollars, with a majority of revenue, expenses, and capital expenditures denominated in U.S. dollars for the year ended December 31, 2024 [451]. - A hypothetical 10% change in foreign exchange rates would not have had a material impact on the company's Consolidated Financial Statements [451].