Ultragenyx Pharmaceutical Inc. Q3 FY2025 earnings call

Ultragenyx received $400 million nondilutive capital from OMERS through the sale of a portion of Crysvita royalties, with payments starting in January 2028, bolstering the balance sheet for pivotal data readouts. Clinically, GTX-102's pivotal Aspire study for Angelman syndrome enrolled 129 patients with data expected in H2 2026, and the Phase II/III Aurora study dosed its first patient. For UX143 in osteogenesis imperfecta, Phase III Orbit and Cosmic studies are ongoing with data on track for end of 2025 (Dec/Jan). Commercially, Crysvita Latin America saw 50 new start forms with ~875 patients on therapy, while Dojolvi in the US had ~700 new start forms and ~625 patients on reimbursed therapy. Other programs like UX111 (MPS IIIA) had a BLA resubmission planned for early 2026, and DTX401 (GSD Ia) had positive 96-week study results with a rolling BLA submission ongoing.

In the third quarter of 2025, Ultragenyx reported total revenue of $160 million, representing 15% growth over Q3 2024 and 18% growth for the first 9 months of 2025 compared to the same period in 2024. Crysvita was the largest product, contributing $112 million in Q3 2025 with $57 million from North America, $47 million from Latin America and Turkey, and $8 million from Europe. Dojolvi contributed $24 million, Evkeeza $17 million, and Mepsevii $7 million. Crysvita's revenue growth is expected in various regions, while Dojolvi, Evkeeza, and Mepsevii also contribute to the financial base with steady growth trajectories.

Total revenue for 2025 is expected to be between $640 million and $670 million, representing 14% to 20% growth from 2024. Crysvita revenue is forecasted to be between $460 million and $480 million (12% to 17% growth), and Dojolvi revenue between $90 million and $100 million (2% to 14% growth). The company remains on track to achieve profitability in 2027, leveraging revenue growth, expense management, and PRV monetization.

Uncertainty exists in clinical trial outcomes for Phase III studies like UX143 and GTX-102. Competition from other rare disease products could impact market share. Regulatory hurdles in BLA submissions or approvals pose risks. Variability in physician and patient adoption of new products may affect revenue growth.

Q: Walk through logics for Orbit and Cosmic data expected year-end 2025, timing and data combination A: Data for Orbit and Cosmic studies will be released together in December or January as the database locking and analysis process is completed.

Q: Open-label extension from Phase II for OI, quantitative perspective on fracture reduction A: No additional quantitative data on Phase II open-label extension is available, but Phase II results are consistent with prior data, and a fracture reduction range of 40% to 70% is expected in Phase III.

Q: UX111 and DTX401 submissions timing A: DTX401's rolling BLA submission is ongoing and expected to be completed next month, while UX111's BLA resubmission is planned for early 2026 due to FDA requirements.

Q: Physician thoughts on initiating setrusumab, prioritizing patients A: Early adoption is likely for most severe patients, but setrusumab will be used across the spectrum, including adults, with younger and more severely affected patients expected to have more immediate access.

Q: Angelman program competitive landscape, decision factors for patients A: Decision factors include data-driven outcomes, potency, safety, and support programs, with the company's ASO showing strong long-term data and patient support being a key factor.