Ultragenyx Pharmaceutical Inc

Latest Results (17.05.2026):

Ultragenyx Pharmaceutical Inc.'s fourth quarter 2025 and full-year 2025 results show continued commercial growth but sustained losses: Q4 2025 total revenue was $207 million (up 25% year-over-year) and full-year 2025 total revenue was $673 million (up 20% year-over-year), while the company reported a Q4 net loss of $129 million ($1.29 per share) and a 2025 net loss of $575 million ($5.83 per share). Management reiterated a path to profitability in 2027 while initiating a strategic restructuring to reduce expenses and refocus resources.

Revenue

Q4 2025 total revenue: $207M (25% YoY growth). Key product contributions in Q4: Crysvita $145M (includes $40M product sales in Latin America & Türkiye), Dojolvi $32M, Evkeeza $17M. Full-year 2025: total revenue $673M (20% growth), Crysvita $481M (17% growth; $177M from Latin America & Türkiye), Dojolvi $96M, Evkeeza $59M (84% growth).

Profitability and Operating Expenses

Q4 2025 total operating expenses: $321M; full-year 2025 operating expenses: $1,208M (including $153M non-cash stock-based compensation). Q4 net loss: $129M ($1.29/sh); 2025 net loss: $575M ($5.83/sh). Loss from operations for FY2025 was $535M.

Cash, Cash Flow and Balance Sheet

Cash, cash equivalents and marketable securities totaled $737M at December 31, 2025. Net cash used in operations: $100M in Q4 2025 and $466M for full-year 2025. Working capital was $567M and total assets $1.532B.

2026 Financial Guidance

2026 revenue guidance (from current products, excluding potential new launches): $730M–$760M (up 8%–13% vs 2025). Crysvita guidance $500M–$520M; Dojolvi guidance $100M–$110M.

Strategic Restructuring and Path to Profitability

Ultragenyx announced a strategic restructuring to reduce expenses and refocus on priority programs, including a ~10% workforce reduction (about 130 roles). The company expects combined R&D+SG&A to be flat to down low-single digits in 2026 vs 2025, with ~ $50M of one-time severance, manufacturing and restructuring charges in 2026. It targets profitability in 2027, with 2027 R&D expected to fall ~38% (~$280M) from 2025 and combined R&D+SG&A down at least 15% vs 2025.

Clinical and Regulatory Catalysts for 2026

Key near-term clinical/regulatory events cited: DTX401 (GSDIa) BLA rolling submission completed; anticipated PDUFA action in Q3 2026. UX111 (MPS IIIA) BLA resubmitted in Jan 2026 but received an IRL requesting additional CMC-related documentation. GTX-102 (Angelman syndrome) pivotal Phase 3 Aspire readout expected in H2 2026. UX701 (Wilson disease) enrollment in the dose-finding stage is complete and data are expected in 2026.