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

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.