Ultragenyx Pharmaceutical (RARE) Barclays 28th Annual Global Healthcare Conference summary

Company overview and commercial strategy

• Focuses on rare diseases with high unmet medical need, leveraging diverse platforms including enzyme replacement, small molecule, and gene therapy.

• Built a broad global commercial footprint, maintaining direct control in North America, Europe, Japan, and South America.

• Relies heavily on medical affairs expertise rather than a traditional large commercial footprint.

Financial outlook and profitability

• Targeting profitability by 2027, driven by growth in commercialized products and late-stage pipeline approvals.

• Profitability depends on successful approval and launch of late-stage programs and cost reductions as trials transition from phase III to commercialization.

• Priority review vouchers (PRVs) from GSD1A, MPS III, and potentially Angelman are factored into financial modeling.

Pipeline and development programs

• Near-term approvals expected for GSD1A (PDUFA in August) and Sanfilippo, with Angelman phase III data readout later this year.

• Additional programs include OI, OTC, and Wilson gene therapies, with ongoing research to expand the pipeline.