Ultragenyx Pharmaceutical (RARE) Corporate presentation summary

Strategic vision and business model

• Focuses on developing first-ever treatments for rare diseases, emphasizing potent biology and patient-centric approaches.

• Utilizes adaptive trial designs and novel endpoints to accelerate development and value creation.

• Operates a lean commercial team to efficiently commercialize products and support patients.

• Maintains a diverse clinical pipeline with multiple late-stage programs targeting high unmet medical needs.

Commercial portfolio and pipeline

• Four commercial products: Crysvita, Evkeeza, Dojolvi, and Mepsevii, with strong revenue growth.

• Five Phase 2/3 programs with two potential approvals anticipated in 2026.

• Late-stage pipeline includes gene therapies and antisense oligonucleotides for diseases like MPS IIIA, GSDIa, Angelman syndrome, Wilson disease, and osteogenesis imperfecta.

Key clinical program highlights

• UX111 for MPS IIIA showed substantial and durable reductions in CSF HS and improvements in developmental skills; BLA resubmission planned.

• DTX401 for GSDIa met primary and key secondary endpoints in Phase 3, significantly reducing cornstarch dependence and improving patient-reported outcomes; BLA submitted, PDUFA expected 3Q 2026.

• GTX-102 for Angelman syndrome is in Phase 3 with data expected in 2H 2026; broad enrollment across genotypes and ages.

• UX701 for Wilson disease showed clinical activity with some patients off chelation therapy; Stage 1 data expected in 2026.

• UX143 (setrusumab) for osteogenesis imperfecta increased bone mineral density and reduced vertebral fractures but did not meet primary fracture reduction endpoints.