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Ultragenyx Pharmaceutical (RARE) investor relations material
Ultragenyx Pharmaceutical Evercore ISI 8th Annual HealthCONx Conference summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.Evercore ISI 8th Annual HealthCONx Conference summary3 Dec, 2025
Current business and pipeline overview
Focused on developing and commercializing drugs for rare diseases, with commercial success from several products and anticipation for upcoming phase 3 data readouts, especially in Osteogenesis Imperfecta.
Recent royalty financing was pursued to strengthen the balance sheet after a delay in potential revenue from a program, resulting in a non-dilutive deal.
Clinical development and trial strategy
Orbit and Cosmic phase 3 study readouts for Osteogenesis Imperfecta will be presented together, as simultaneous disclosure is considered most effective.
Both studies are powered for success, with Cosmic designed to show superiority and Orbit powered to detect at least a 50% treatment effect with 159 patients.
Commercial viability is expected even with moderate effect sizes, as long as results surpass current standard treatments.
Dropouts and rescue treatments in trials are within expectations, with dropout rates described as a non-issue.
Commercial and market considerations
Pricing strategy for Osteogenesis Imperfecta is modeled after a previous successful launch, with duration of treatment expected to be lifelong due to the drug's mechanism.
Patient mix in trials is roughly 50% type 3/4 and 50% type 1, with commercial population estimates at 60% type 1 and 38% type 3/4.
What drove the royalty financing decision?
Is a low-end OI trial outcome commercially viable?
How can Angelman cognition be improved?
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Next Ultragenyx Pharmaceutical earnings date
Q4 202513 Feb, 2026
Next Ultragenyx Pharmaceutical earnings date
Q4 202513 Feb, 2026
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Frequently asked questions
Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company dedicated to developing and commercializing therapies for rare and ultra-rare genetic diseases. The company's focus includes treatments for metabolic disorders, bone diseases, and other conditions with significant unmet medical needs. Ultragenyx leverages innovative research and targeted approaches to address complex diseases that often lack effective therapies.The company is headquartered in Novato, California, and its shares are listed on the NASDAQ.
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