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@starcahier 1 month ago

Ultragenyx Phase 3 results for setrusumab missed primary endpoints

Ultragenyx Phase 3 results for setrusumab missed primary endpoints

Ultragenyx (RARE) released results from their Phase 3 Orbit and Cosmic studies for setrusumab in Osteogenesis Imperfecta. Neither study met the primary endpoint of reducing annualized fracture rates compared to placebo or bisphosphonates. They did see statistically significant improvements in bone mineral density, which was a secondary endpoint, but that didn't result in the fracture reduction they were looking for. The safety profile remained unchanged. The CEO expressed disappointment since the Phase 2 data looked more promising. As a result, the company announced they will be implementing significant expense reductions while they evaluate the data and next steps.
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