Ultragenyx (RARE) Corporate presentation summary

Commercial portfolio and launches

• Portfolio spans six commercial products, three ongoing clinical programs and two Phase 3 programs; the presentation targets $1B in revenue.

• FAYUVI received standard full approval for MPS IIIA on Sept. 17, 2026, with a PRV issued; GENGLYCOS received accelerated approval for GSDIa on Aug. 19, 2026, also with a PRV.

• FAYUVI is a single-infusion gene therapy that restores SGSH enzyme production; approval was based on neuro endpoints. Estimated prevalence is 3,000–5,000 in commercially accessible geographies.

• GENGLYCOS reduces daily cornstarch intake in adults and children aged 8+ with GSDIa; manufacturing is in-house at the Bedford, MA plant.

• Gene therapy launches use dedicated patient guides, qualified treatment centers and monitoring programs lasting 2 years for GENGLYCOS and up to 10 years for FAYUVI.

Clinical pipeline

• DTX301 reduced plasma ammonia AUC 0–24 by 18% vs placebo at Week 36 (p=0.018); treated patients maintained generally normal ammonia with 27% lower scavenger medication use and ~13% higher protein intake.

• At Week 24, 86% of DTX301-treated patients had clinically important improvement in overall OTCD vs 13% on placebo; Phase 3 complete-responder data are expected in 1H 2027.

• UX143 Phase 3 studies missed their primary annualized fracture-rate endpoints but showed significant BMD gains; an exploratory COSMIC high-fracture subgroup analysis showed lower AFR vs IV bisphosphonates (p=0.009; not adjusted for multiplicity).

• FDA discussions are underway on prospective data needed to support a UX143 BLA; UX016 GNE myopathy Phase 1/2 IND clearance and first-patient-in are anticipated in 2H 2026.

Financial outlook

• 2025 revenue was $673M, up 20%; 2026 revenue guidance is $730–760M, excluding growth from GENGLYCOS and FAYUVI launches.

• 2026 guidance includes CRYSVITA revenue of $500–520M, up 4–8%, and DOJOLVI revenue of $100–110M, up 4–14%.

• 2027 GAAP profitability is targeted, supported by growth across four existing products, gene therapy launches and expense discipline; planned 2027 R&D is below $350M and SG&A below $375M.

• Expense plans include a workforce reduction of about 20%; 2027 R&D is expected to be 50% below 2025.

• Cash and investments totaled $401M at Sept. 30, 2026, excluding PRVs; two PRV sales are expected to provide ~$350M net, with the first closed and the second expected to close in November.