Ultragenyx's Q1 2025 Earnings Call: Navigating Contradictions in Trial Success, Therapy Duration, and Data Timelines

Confidence in the second interim analysis and trial success, duration of therapy for OI, timeline for Orbit and Cosmic data, setrusumab interim analysis and fracture reduction, and Angelman Syndrome data and endpoints are the key contradictions discussed in Ultragenyx Pharmaceutical's latest 2025Q1 earnings call.



Revenue Growth and Regional Performance:
- Ultragenyx reported Q1 2025 revenue of $139 million, representing 28% growth over the same period in 2024.
- The growth was driven by strong performance in Latin America, notably in Crysvita, and increased new patient starts in the U.S. for Dojolvi and Crysvita.

Crysvita's International Success:
- Crysvita contributed $103 million to Ultragenyx's Q1 2025 revenue, with 25% growth over 2024.
- The growth in Latin America was supported by successful negotiations with Brazilian and Mexican authorities for reimbursement, and continued expansion in Central and South American countries.

Dojolvi's Steady Growth:
- Dojolvi contributed $17 million to Ultragenyx's Q1 2025 revenue, maintaining a steady growth trajectory.
- The growth was sustained by the increasing number of new starts and prescribers in the U.S., with approximately 600 patients on reimbursed therapy.

Upcoming Clinical Milestones:
- Ultragenyx emphasized key clinical data readouts expected in 2025, including BLA submissions for DTX401 and UX111, and UX143's second interim analysis.
- The company's development teams are advancing large and late-stage programs, with significant progress in Phase 3 trials for UX701 and GTX-102, positioning Ultragenyx for future success.