Ultragenyx Pursues a Tough Turnaround with Broad Restructuring

Ultragenyx Pharmaceutical reported a double-digit rise in revenue for 2025, but the biopharma still posted deep losses as it moves to restore profitability by 2027. The company unveiled a far-reaching overhaul that includes a sizable reduction in staff, adding pressure from FDA regulatory snags that cloud the path to nearer-term milestones.

• Revenue 2025: $673 million (+20% versus 2024)
• Net loss: $575 million (-$5.83 per share)
• Headcount reduction: 10% (about 130 employees)
• Guidance 2026: Revenue seen between $730 million and $760 million

Strategic pivot aimed at profitability

The fourth quarter outperformed on the top line, up 25% to $207 million, yet the quarterly loss per share rose to $1.29, above expectations. Crysvita remains the main growth driver, contributing $481 million to the annual tally. In response, Ultragenyx will trim roughly 130 roles as part of a broader plan to slash research and development spending significantly through 2027.

The restructuring will incur one-off costs of roughly $50 million, including severance payments and the early termination of certain manufacturing contracts. Ultragenyx’ cash cushion stood at about $737 million, which the company said should be sufficient to bridge the company through the transition to sustained profitability. Management signaled that, while 2026 operating and development costs are expected to remain flat, 2027 targets include a 38% reduction in R&D spending versus the 2025 level.

Regulatory delays and clinical milestones ahead

Beyond the earnings figures, FDA-related news weighed on sentiment. The gene therapy UX111 received an Incomplete Response Letter, with the agency requesting additional documentation on manufacturing and quality controls (CMC). The setback delays the regulatory path for the Sanfilippo syndrome Type A therapy.

Looking ahead to 2026, several pivotal events are on Ultragenyx’ calendar. In the third quarter, the FDA’s PDUFA decision date for DTX401, a treatment for glycogen storage diseases, is anticipated. In the second half of the year, phase 3 results for GTX-102, aimed at Angelman syndrome, are due and are expected to be a key stock driver for the company.

Summary outlook

• 2025 produced revenue growth but felt the weight of continued losses.
• A broad restructuring targets operating and development efficiency, with a heavy emphasis on reducing R&D spend through 2027.
• Near-term regulatory headwinds persist, even as the company advances other clinical programs and seeks meaningful milestones in 2026.

@ boerse-global.de | US90400D1081 ULTRAGENYX