Primary-source record
Decision-gradeTopline results
Ultragenyx: UX143 Phase 3 Orbit/Cosmic final analysis expected end of 2025
The document states that the Phase 3 Orbit and Cosmic studies of setrusumab (UX143) in osteogenesis imperfecta are progressing toward final analyses after patients have been on therapy for at least 18 months. Data from these studies are expected around the end of 2025.
Key facts
- Filed
- Nov 4, 2025, 09:12 PM
- Event
- Topline results
- Direction
- Neutral
- Company
RAREUltragenyx- Asset
- setrusumab
- Source
- SEC 8-K
- Confidence
- Sourced
Source excerpt
EX-99.1 2 rare-ex99_1.htm EX-99.1 EX-99.1 Exhibit 99.1 Contacts Ultragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Ultragenyx Reports Third Quarter 2025 Financial Results and Corporate Update Third quarter total revenue of $160 million, Crysvita® revenue of $112 million and Dojolvi® revenue of $24 million Reaffirm 2025 Revenue Guidance: Total revenue between $640 million to $670 million, Crysvita revenue of $460 million to $480 million, and Dojolvi revenue of $90 million to $100 million Bolstered balance sheet with $400 million proceeds from sale of portion of Crysvita royalty interest NOVATO, Calif. – November 04, 2025 – Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today reported its financial results for the quarter ended September 30, 2025. “We are a global commercial company with multiple products generating meaningful growth that is expected to accelerate from anticipated launches from our late-stage clinical pipeline,” said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “We announced today that we bolstered our balance sheet with a royalty financing ahead of pivotal milestones expected over the next year to support multiple late-stage data readouts, multiple regulatory submissions, and multiple launches. This includes the highly anticipated phase 3 study readout