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Ultragenyx Pharmaceutical announced that its Phase 3 Aspire study for apazunersen (GTX-102) in Angelman syndrome…
RAREUltragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical announced that its Phase 3 Aspire study for apazunersen (GTX-102) in Angelman syndrome failed to achieve the primary endpoint of change from baseline in Bayley-4 cognitive raw score or the key secondary endpoint of net response in the Multidomain Responder Index. The safety profile was consistent with earlier phases, but no efficacy differences supported approval. The company will evaluate the program's future and implement significant expense reductions while supporting its commercial business. Ultragenyx Pharmaceutical Inc. operates in the biopharmaceutical industry, developing treatments for rare genetic diseases.