Ultragenyx Pharmaceutical (RARE) Corporate presentation summary

Strategic vision and business model

• Focuses on rare diseases with high unmet medical need, leveraging adaptive trial designs and patient-centric commercialization to accelerate value creation and reduce post-approval R&D costs.

• Operates four commercial products and maintains a diverse late-stage clinical pipeline, with two potential approvals targeted for 2026.

• Plans significant expense and headcount reductions to support profitability by 2027, while investing in launches for new products.

Clinical pipeline and late-stage programs

• Pipeline includes gene therapies and biologics for bone/endocrine, neurogenetic, and metabolic disorders, with multiple Phase 2/3 programs.

• Key programs: UX143 (osteogenesis imperfecta), UX111 (Sanfilippo syndrome), DTX401 (GSDIa), GTX-102 (Angelman syndrome), and UX701 (Wilson disease).

• Near-term catalysts include BLA submissions, pivotal data readouts, and potential regulatory approvals in 2026.

Key clinical results and regulatory updates

• UX143 missed primary fracture reduction endpoints but showed significant BMD gains, reduced vertebral fractures, and improved patient-reported outcomes in pediatric patients.

• UX111 demonstrated substantial and sustained reduction in CSF heparan sulfate, with significant cognitive and functional improvements in MPS IIIA patients; BLA resubmission planned for early 2026.

• DTX401 met primary and secondary endpoints in GSDIa, significantly reducing cornstarch dependence and improving quality of life; BLA submitted December 2025.

• GTX-102 Phase 3 Aspire data expected in 2H 2026; UX701 dose-finding data expected in 1H 2026, with early clinical activity observed.