Ultragenyx Pharmaceutical (RARE) investor relations material

Executive summary

• Q1 2026 revenue was $136 million, with Crysvita contributing $93 million and Dojolvi $18 million; the company operates in rare and ultra-rare genetic diseases with four approved products and a robust pipeline.

• Net loss for Q1 2026 was $185 million ($1.84 per share), up from $151 million ($1.57 per share) in Q1 2025, driven by higher R&D and restructuring costs.

• 2026 is expected to be transformative, with revenue projected to exceed $700 million and multiple new drug approvals anticipated; two gene therapies (DTX401 and UX111) have PDUFA dates in August and September 2026.

• Strategic restructuring plan implemented in February 2026, including a 10% workforce reduction and $30 million in charges.

• Commercial business generates revenue in over 35 countries, with disciplined execution and a path to profitability in 2027.

Financial highlights

• Q1 2026 total revenue was $136 million: Crysvita $93M, Dojolvi $18M, Evkeeza $18M (64% YoY growth), Mepsevii $7M.

• Total operating expenses were $305 million, including $30M cost of sales, $275M R&D and SG&A, $30M non-cash stock-based compensation, and $30M restructuring expenses.

• Net loss for the quarter was $185 million, or $1.84 per share; operating loss was $169 million.

• Cash, cash equivalents, and marketable securities at quarter-end were $534 million; net cash used in operations was $197 million.

• Gross margin for Q1 2026 was approximately 78%.

Outlook and guidance

• 2026 revenue guidance reaffirmed at $730–$760 million, representing 8–13% growth over 2025, excluding new product launches.

• CRYSVITA revenue expected between $500–$520 million; DOJOLVI between $100–$110 million.

• Combined R&D and SG&A expenses for 2026 expected to be flat to down low single digits vs. 2025; at least 15% decrease expected in 2027 vs. 2025.

• Profitability targeted for 2027; management expects annual losses to continue in the near term.

• Guidance includes continued investment in pipeline and commercial launches, with sufficient liquidity for at least the next 12 months.