Ultragenyx Says Phase 3 Study on Rare Neurogenetic Disorder Missed Primary, Secondary Endpoints; Shares Fall After-Hours
MT Newswires Live
Sep 03
Ultragenyx Pharmaceutical (RARE) said late Wednesday the phase 3 Aspire study of apazunersen in Angelman syndrome, a rare, neurogenetic disorder, did not achieve its primary endpoint or its key secondary endpoint.
The late-stage study did not reach the primary endpoint of change from Baseline in Bayley-4 cognitive raw score nor the key secondary endpoint of net response in Multidomain Responder Index, according to a statement.
The safety profile for the phase 3 trial remained consistent with phase 1 and phase 2, the company said.
Ultragenyx said it will assess the apazunersen program following this outcome and make a decision on its disposition.
Shares of Ultragenyx were down about 45% in after-hours trading.
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