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Ultragenyx Phase 3 Aspire Trial Of Apazurersen Misses Key Goals In Angelman Syndrome; Shares Plunge

Source: Nasdaq

Healthcare & BiotechCompany FundamentalsAnalyst Insights

Ultragenyx (RARE) reported that its Phase 3 Aspire study of apazutersen (GTX-102) in Angelman syndrome did not meet the primary or key secondary endpoints. The clinical readout is a material setback for the program’s efficacy case and increases uncertainty around future development and potential commercialization.

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