Ultragenyx Pharmaceutical (RARE) investor relations material

Executive summary

• Q1 2026 revenue reached $136 million, with strong performance across all commercial products and continued double-digit annual revenue growth expected to exceed $700 million for the year.

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

• Multiple late-stage programs are progressing, including two gene therapies (DTX401 and UX111) with PDUFA dates in August and September 2026, and phase III data for GTX-102 in Angelman syndrome expected in the second half of 2026.

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

• The company is preparing for two major product launches and expects to achieve profitability in 2027.

Financial highlights

• Q1 2026 total revenue was $136 million: CRYSVITA $93 million, DOJOLVI $18 million, EVKEEZA $18 million (64% YoY growth), and MEPSEVII $7 million.

• Operating expenses totaled $305 million, including $30 million in non-cash stock-based compensation and $30 million in restructuring costs.

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

• Cash and equivalents stood at $534 million as of March 31, 2026.

• Net cash used in operations was $197 million, expected to decrease in subsequent quarters.

Outlook and guidance

• 2026 revenue guidance reaffirmed at $730–$760 million, representing 8–13% growth over 2025, excluding potential 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 versus 2025; at least 15% decrease expected in 2027 versus 2025.

• Profitability targeted for 2027.

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