Ultragenyx Pharmaceutical (RARE) Goldman Sachs 45th Annual Global Healthcare Conference summary

Strategic outlook and pipeline development

• Achieved over $500 million in revenue with five global product approvals and a robust rare disease pipeline.

• Orbit and Cosmic programs in phase III, with phase II data expected later this year and interim/final assessments by late 2024 or 2025.

• Angelman program showed strong phase II data; phase III trial to start later this year with a 120-patient, 48-week design.

• Two gene therapy programs (Sanfilippo and GSDIa) completed phase III, with regulatory filings anticipated.

• Potential for three BLAs to be filed within just over a year, positioning for multiple global launches from 2025 to 2026.

Clinical results and regulatory progress

• Setrusumab phase II in OI showed a 67% median reduction in fracture frequency, with many patients experiencing zero fractures.

• Phase III OI study includes interim analyses, with less than 50% chance of early completion but strong FDA support.

• Angelman phase II data demonstrated consistent, durable improvements and manageable safety profile after protocol amendments.

• Regulatory alignment for Angelman phase III focuses on Bayley cognition as primary endpoint and 48-week duration.

• Wilson disease gene therapy progressing, with three-cohort data on urinary copper and ceruloplasmin activity expected later this year.

Commercial strategy and market opportunity

• OI launch will leverage existing Crysvita sales force, with 36 reps and patient diagnosis liaisons to maximize reach.

• OI represents a larger, more urgent commercial opportunity than XLH, with an estimated 60,000 patients in key territories.

• GSDIa and Sanfilippo gene therapies address urgent needs, with strong patient demand and commercial potential.

• Commercial launches expected to be more successful than previous products due to higher unmet need and improved strategies.