Company Overview - Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company focused on developing and commercializing novel therapies for serious rare and ultra-rare genetic diseases [3] - The company has established a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical needs [3] Management and Strategy - The management team of Ultragenyx is experienced in the development and commercialization of rare disease therapeutics [4] - The company's strategy emphasizes time- and cost-efficient drug development to deliver safe and effective therapies to patients urgently [4] Upcoming Events - Howard Horn, the chief financial officer and executive vice president of Ultragenyx, will participate in a fireside chat at Bank of America's 2025 Healthcare Conference on May 13, 2025, at 8:00 a.m. PT [1] - A live and archived webcast of the panel will be available on the company's website [2]

Core Insights - Ultragenyx Pharmaceutical Inc. reported total revenue of $139 million for Q1 2025, reflecting a 28% increase compared to Q1 2024, with Crysvita revenue at $103 million, a 25% increase year-over-year [4][8] - The company reaffirmed its 2025 financial guidance, projecting total revenue between $640 million to $670 million, with Crysvita revenue expected to be between $460 million to $480 million, and Dojolvi revenue between $90 million to $100 million [10][12] - The investigational treatment UX111 for Sanfilippo syndrome is on track for a Biologics License Application (BLA) action date set for August 18, 2025 [14] Financial Performance - Total revenues for Q1 2025 were $139 million, up from $108.8 million in Q1 2024, with significant contributions from Crysvita and Dojolvi [3][4] - Crysvita's product sales in Latin America and Türkiye reached $55 million, marking a 52% increase compared to the previous year [4][5] - Operating expenses totaled $282 million in Q1 2025, with research and development costs at $165.8 million [6][7] Net Loss and Cash Position - The net loss for Q1 2025 was $151 million, or $1.57 per share, an improvement from a net loss of $171 million, or $2.03 per share, in Q1 2024 [8][9] - As of March 31, 2025, the company had cash, cash equivalents, and marketable securities totaling $563 million, down from $745 million at the end of 2024 [9][29] Clinical Developments - The company is progressing with multiple clinical trials, including UX143 for osteogenesis imperfecta and GTX-102 for Angelman syndrome, with key interim analyses expected in mid-2025 [10][11] - The Phase 3 study for DTX401 is anticipated to submit a BLA in mid-2025, following positive results from the GlucoGene study [15][16] Strategic Focus - Ultragenyx aims to manage expenses while prioritizing investments in upcoming commercial launches and advancing multiple Phase 3 programs, which is expected to reduce net cash used in operations in 2025 compared to 2024 [10][12]

NOVATO, Calif., April 30, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call at 5:00 p.m. ET on Tuesday, May 6, 2025, to discuss its financial results and corporate update for the quarter ending March 31, 2025. The live and replayed webcast of the call will be available through the company’s w ...