Ultragenyx (RARE) Corporate presentation summary

Portfolio and strategy

• Portfolio includes 6 commercial products, 4 ongoing clinical programs and 3 Phase 2/3 programs; 12 years since IPO and targeting GAAP profitability in 2027.

• Two gene therapies, FAYUVI and GENGLYCOS, received U.S. approval in 2026, expanding the commercial portfolio.

Approved gene therapy launches

• FAYUVI received standard full approval on September 17, 2026, for MPS IIIA; approval was based on neuro endpoints, and the therapy received a Priority Review Voucher (PRV).

• FAYUVI trial and long-term follow-up showed a 23.5-point higher mean Bayley-III cognitive raw score versus natural history from ages 24 to 60 months (p<0.0001); it is a single IV infusion.

• GENGLYCOS received accelerated approval on August 19, 2026, to reduce daily cornstarch intake in patients aged 8 and older with GSDIa; it received a PRV.

• GENGLYCOS Phase 1/2 and Phase 3 included 52 treated patients with over 8 years of follow-up; Phase 3 showed significant cornstarch reductions versus placebo while maintaining glycemic control.

• Launch support includes Gene Therapy Guides, Qualified Treatment Centers, and disease monitoring for 2 years for GENGLYCOS and up to 10 years for FAYUVI.

Clinical pipeline

• UX143 Phase 3 ORBIT and COSMIC missed primary annualized fracture rate endpoints but showed significant BMD increases; additional analyses reported vertebral fracture and patient-reported outcome improvements.

• In post-hoc analyses of patients under 18 with more than 3 baseline fractures, ORBIT showed numerically lower AFR versus placebo (p=0.063); COSMIC showed lower AFR versus IV-BP (rate ratio 0.47, nominal p=0.009).

• DTX301 Phase 3 showed an 18% reduction in plasma ammonia AUC0-24 versus placebo at Week 36 (p=0.018); complete-responder analysis is expected in 1H 2027.

• DTX301 patients showed clinically important overall improvement in OTCD in 86% of cases versus 13% for placebo at Week 24; treatment was well tolerated, with hepatic reactions the most common treatment-emergent adverse events.

• UX701 Stage 1 data from Cohorts 1–4 are expected in 4Q 2026; 6 of 15 patients were completely off chelators and/or zinc therapy, with one additional patient tapering standard care.