Ultragenyx (RARE) investor relations material

Executive summary

• FDA approvals of GENGLYCOS and FAYUVI expanded the portfolio to six approved therapies and launched its first two gene therapies.

• GTX-102 failed to demonstrate Phase III efficacy and was wound down; UX701 was terminated after failing advancement criteria, as part of broad cost reductions and portfolio prioritization.

• 2027 GAAP net income profitability and positive operating cash flow remain targets, supported by commercial growth, new launches and lower spending.

Financial highlights

• Q3 revenue was $173M vs. $160M year over year; product sales were $105M vs. $95M and royalty revenue was $68M vs. $65M.

• Q3 operating expenses were $283M vs. $331M, operating loss was $110M vs. $171M, and net loss was $118M vs. $180M.

• Nine-month revenue was $523M vs. $466M; operating loss was $354M vs. $422M, net loss was $395M vs. $446M, and loss per share was $3.89 vs. $4.55.

• Reaffirmed 2026 revenue guidance of $730M–$760M, excluding GENGLYCOS and FAYUVI launches; CRYSVITA guidance is $500M–$520M and DOJOLVI guidance is $100M–$110M.

• Combined 2026 R&D and SG&A expense guidance remains flat to down low single digits vs. 2025; it includes $30M of first-half restructuring charges and ~$25M expected in Q4.

Outlook and guidance

• 2027 R&D expense is expected below $350M, over 50% below 2025; SG&A is expected below $375M, with full-year revenue guidance expected early in the year.

• DTX301 Phase III data are expected in H1 2027; spending priorities include completing the study, determining the path for UX143 and measured investment in early-stage programs.

• Management expects to sustain roughly five programs annually within profitability constraints; most 2027 R&D spending will support commercial products, disease monitoring and regulatory filings.

• Setrusumab showed activity, including fracture reduction in patients with more than three baseline fractures; FDA discussions will guide its development path and any investment.