Ultragenyx (RARE) investor relations material

Executive summary

• Achieved record Q2 2026 revenue of $214 million, up 28% year-over-year, driven by strong commercial execution and global expansion of key products including Crysvita, DOJOLVI, Evkeeza, and MEPSEVII.

• Preparing for potential first-ever gene therapy launches for GSDIa (DTX-401) and Sanfilippo syndrome type A (UX-111), with PDUFA dates in August and September 2026.

• Strategic restructuring in February 2026 reduced workforce by 10% and focused resources on high-value drivers.

• Anticipating top-line Phase III data for GTX-102 in Angelman syndrome in September or October 2026.

• Confident in path to profitability by 2027, supported by continued product growth, disciplined expense management, and PRV monetization.

Financial highlights

• Q2 2026 total revenue reached $214 million, with Crysvita contributing $156 million, DOJOLVI $27 million, Evkeeza $21 million, and MEPSEVII $10 million.

• Net loss for Q2 2026 was $92 million ($0.90 per share), improved from $115 million ($1.17 per share) in Q2 2025.

• Operating expenses for Q2 2026 totaled $289 million, including $34 million in non-cash stock-based compensation.

• Cash, cash equivalents, and marketable securities stood at $436 million as of June 30, 2026.

• Net cash used in operations for Q2 2026 was $97 million, a significant decrease from Q1.

Outlook and guidance

• Reaffirmed 2026 total revenue guidance of $730–$760 million, excluding potential new product launches.

• Path to profitability in 2027 based on double-digit revenue growth, expense discipline, and monetization of priority review vouchers (PRVs) upon potential approvals.

• Launch investments for DTX-401 and UX-111 are included in current expense guidance.

• Combined R&D and SG&A expenses for 2026 expected to be flat to slightly down versus 2025; 2027 expected to decrease by at least 15%.

• Sufficient capital resources projected to fund operations for at least the next 12 months.