Ultragenyx Pharmaceutical (RARE) investor relations material

Executive summary

• Achieved $166M in Q2 2025 revenue, a 13% year-over-year increase, with double-digit growth in commercial therapies and strong performance from Crysvita and Dojolvi.

• Maintained focus on rare diseases with four commercial products and a robust clinical pipeline, including key programs for osteogenesis imperfecta and Angelman syndrome.

• Celebrated 15th anniversary with five Phase III programs fully enrolled or at BLA submission stage and significant regulatory progress.

• Net loss for Q2 2025 was $115M ($1.17 per share), improved from $132M ($1.52 per share) in Q2 2024.

• Cash, cash equivalents, and marketable securities totaled $539M as of June 30, 2025, supporting ongoing operations and development.

Financial highlights

• Q2 2025 total revenue reached $166M, up from $147M in Q2 2024; first half 2025 revenue up 20% year-over-year.

• Crysvita contributed $120M in Q2, with $79M from North America, $35M from Latin America and Turkey, and $7M from Europe.

• Dojolvi revenue was $23M, Akiza/Evkeeza $15M, and Mepsevii $8M in Q2.

• Gross margin for Q2 2025 was $143M (total revenue $166M minus cost of sales $23M).

• Net loss per share improved to $1.17 from $1.52 year-over-year.

Outlook and guidance

• 2025 total revenue guidance reaffirmed at $640M–$670M, representing 14%–20% growth over 2024.

• Crysvita revenue expected between $460M–$480M (12%–17% growth); Dojolvi revenue expected between $90M–$100M (2%–14% growth).

• Path to GAAP profitability targeted for 2027, with continued focus on revenue growth and expense prioritization.

• Key regulatory submissions for pipeline assets anticipated in late 2025, with several pivotal trial readouts expected by year-end.

• Monetization of priority review vouchers from UX111, DTX401, and UX143 expected to support liquidity.