Ultragenyx Pharmaceutical Inc. Q4 FY2025 earnings call

• 2026 is a significant year with potential approvals in MPS IIIA, Glycogen Storage Disease Type Ia, and pivotal data in Angelman syndrome. • UX111 data for MPS IIIA showed sustained improvement in neurologic endpoints. • Resubmitted UX111 BLA received a complete response letter, with FDA requiring additional documentation. • 2025 commercial business grew 20%, with presence in over 35 countries. • Implemented strategic restructuring plan to reduce operating expenses, including 10% workforce reduction.

For 2025, total revenue was $673 million. Crysvita contributed $481 million, with 17% growth over 2024, including $275 million from North America, $177 million from Latin America and Turkey, and $29 million from Europe. Dojolvi contributed $96 million, a 9% growth over 2024. Evkeeza contributed $59 million, an 84% growth over 2024. MEPSEVII contributed $37 million.

• 2026 total revenue expected between $730 million and $760 million (8%-13% growth over 2025), excluding new product launches. • Crysvita revenue expected $500 million-$520 million. • Dojolvi revenue expected $100 million-$110 million. • 2026 R&D and SG&A expenses flat to low single digits vs 2025; 2027 combined R&D and SG&A expenses expected down at least 15% vs 2025. • 2027 R&D expenses to decrease 38% driven by completion of Phase III studies and reduced early-stage research.

• FDA's request for additional documentation in UX111 BLA resubmission may impact timeline. • Variability in revenue due to uneven ordering patterns, particularly in Latin America. • Uncertainty around regulatory decisions for ongoing clinical programs, such as UX111 and others.

Q: What is the primary endpoint for the Angelman study and what percentage of patients have rolled over into the long-term extension?

A: The primary endpoint is Bayley-4 cognition. There was very high retention, with patients continuing in the extension study as parents recognize the opportunity for their children to develop.

Q: How does Ultragenyx plan to achieve profitability in 2027?

A: Revenue growth from existing products in double digits and contribution from upcoming launches, with expense cuts: 2026 R&D and SG&A flat to low single digits vs 2025, and 2027 combined R&D and SG&A down at least 15% vs 2025, along with potential PRVs.