Ultragenyx reports prelim Q3 rev $96-100M; UX143 cuts fractures 67% in Phase 2

Ultragenyx Pharmaceutical Inc.

Prelim Q3 total rev $96-100M; Crysvita $74-76M (impacted by inventory shift to KKC); cash $525M.
Setrusumab (UX143) cut annualized fracture rate 67% (p=0.042) in Phase 2; BMD +13-16% at 6 months.
GTX-102 for Angelman: clinically meaningful improvements vs natural history; no new SAEs since Nov 2022.
UX701 for Wilson: 4/5 low-dose patients tapering off chelators; 2 completely off standard therapy.
Gene therapy: DTX401 Phase 3 fully enrolled; UX111 fully enrolled; DTX301 enrollment expected H1 2024.

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