Ultragenyx Reports Third Quarter 2025 Financial Results and Corporate Update

Core Insights - Ultragenyx Pharmaceutical Inc. reported total revenue of $160 million for Q3 2025, marking a 15% increase compared to Q3 2024 [4][5][11] - The company reaffirmed its 2025 revenue guidance, expecting total revenue between $640 million to $670 million, with Crysvita revenue projected at $460 million to $480 million and Dojolvi revenue between $90 million to $100 million [1][16] - A significant balance sheet improvement was noted with $400 million raised from the sale of a portion of Crysvita royalty interest [1][9] Financial Performance - Total revenues for Q3 2025 were $159.9 million, up from $139.5 million in Q3 2024 [5][30] - Crysvita revenue for Q3 2025 was $112 million, including $47 million from Latin America and Türkiye [4][30] - Dojolvi generated $24 million in revenue during the same period [4][30] - Operating expenses totaled $331 million for Q3 2025, which included $37 million in non-cash stock-based compensation [6][30] - The net loss for Q3 2025 was $180 million, or $1.81 per share, compared to a net loss of $134 million, or $1.40 per share, in Q3 2024 [7][30] Cash Position - As of September 30, 2025, the company had cash, cash equivalents, and marketable debt securities amounting to $447 million [8][35] - Net cash used in operations for Q3 2025 was $91 million, with a total of $366 million used in the first nine months of 2025 [10][11] Clinical Developments - The company is preparing for pivotal milestones in its late-stage clinical pipeline, including the Phase 3 study readouts for UX143 in osteogenesis imperfecta expected by the end of 2025 [2][12] - Enrollment for the Phase 3 Aspire study of GTX-102 for Angelman syndrome has been completed, with data expected in the second half of 2026 [13] - The company plans to resubmit the Biologics License Application (BLA) for UX111 early in 2026 [15][18] Strategic Updates - The company has engaged in discussions with the FDA following a Complete Response Letter (CRL) and plans to include additional clinical data in the BLA resubmission [17][18] - The rolling submission of the BLA for DTX401 is expected to be completed in Q4 2025, with significant data supporting its efficacy [19][20]