Ultragenyx Pharmaceutical (RARE) investor relations material

Strategic approach and business model

• Focuses on rare disease medicine, developing first-ever treatments and innovative trial designs.

• Employs a lean commercial model emphasizing patient identification and support.

• Four commercial products approved, marketed in over 30 countries, with growing revenue.

• Five Phase 2/3 programs ongoing, with two approvals expected this year.

• Plans significant expense and headcount reductions to address financial challenges.

Setrusumab clinical trial results and analysis

• Two Phase 3 studies (Orbit and COSMIC) did not meet primary fracture reduction endpoints but showed significant bone mineral density (BMD) improvements.

• Orbit (placebo-controlled) showed improved pain and functional outcomes, but no fracture rate difference.

• COSMIC (active-controlled) showed a 59% reduction in vertebral fractures and strong BMD gains, especially in trabecular bone.

• Safety profile was favorable in both studies.

• Further data analysis ongoing to understand fracture outcomes and regulatory path.

Pipeline and upcoming milestones

• Sanfilippo syndrome gene therapy shows strong biomarker and clinical data; refile expected early in the year, aiming for mid-year approval.

• DTX401 for glycogen storage disease filed in December, with strong demand and positive trial results; potential approval in about eight months.

• GTX-102 for Angelman syndrome in Phase 3, with data expected in the second half of the year; multi-domain responder index and Bayley scores show meaningful improvements.

• Wilson disease gene therapy shows early promise; higher dose cohort data expected later this year.