Ultragenyx Reports Third Quarter 2026 Financial Results, Announces Substantial Expense Reductions, and Reaffirms Profitability in 2027

Key Points:

• Ultragenyx reported third quarter 2026 total revenue of $173 million, with CRYSVITA® generating $109 million and DOJOLVI® $30 million, reaffirming full-year 2026 revenue guidance of $730-$760 million excluding new product launches.
• The FDA approved BLAs for two gene therapies, GENGLYCOS™ for glycogen storage disease type Ia and FAYUVI™ for Sanfilippo syndrome type A, with the company selling associated Priority Review Vouchers (PRVs) for a combined $413 million.
• Ultragenyx is implementing substantial expense and headcount reductions, including a 20% workforce cut, aiming to reduce 2027 R&D expenses by at least 50% compared to 2025, and expects to achieve GAAP profitability in 2027 and beyond.
• The company is discontinuing development of UX701 for Wilson disease due to insufficient efficacy, while continuing clinical progress on other programs such as DTX301 for OTC deficiency and UX143 for osteogenesis imperfecta.
• Cash and marketable securities stood at $401 million as of September 30, 2026, with net cash used in operations at $43 million for the quarter; restructuring charges of approximately $25 million are expected in Q4 2026.