An experimental therapy that families of children with Angelman syndrome had followed for years failed its final-stage trial, missing both the primary endpoint and the key secondary endpoint. There is still no approved treatment that addresses the underlying cause of the condition.
Ultragenyx reported that its Phase 3 Aspire study of apazunersen, previously known as GTX-102, did not achieve improvement in Bayley-4 cognitive raw scores from baseline, nor in a composite measure called the Multidomain Responder Index. The company said the randomized groups were comparable at baseline and that no differences emerged between the treated and control groups that could support efficacy.
