Ultragenyx's UX143 Phase 3 Orbit passes DMC safety review; final analysis year-end 2025

Ultragenyx Pharmaceutical Inc.

DMC confirms acceptable safety profile for UX143 in pediatric/young adult OI patients; study continues to final analysis.
Final analysis of Phase 3 Orbit trial expected around end of 2025, with statistical threshold p<0.04.
Safety in younger patients consistent with prior studies; no data from Cosmic study analyzed at interim.
Patients continue dosing; final analyses after at least 18 months therapy.

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