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Ultragenyx Setrusumab Miss: Fracture Endpoint Failure

Ultragenyx Plunges After Setrusumab Miss: When “Better BMD” Doesn’t Mean Fewer Fractures

A rare-disease biotech just got cut in half after a Phase 3 miss. Ultragenyx’s setrusumab improved bone density, but didn’t deliver what investors really needed: fewer fractures.

What Happened

Ultragenyx ($RARE) said its late-stage studies of setrusumab (UX143) in osteogenesis imperfecta (brittle-bone disease) failed to meet the primary endpoint: a statistically significant reduction in annualized clinical fracture rate versus placebo or bisphosphonates.

Shares dropped sharply on the news, and the company…

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