Ultragenyx doses first patient in Aurora study of GTX-102 for Angelman; Aspire Phase 3 fully enrolled

Ultragenyx Pharmaceutical Inc.

First patient dosed in global Aurora study (NCT07157254) evaluating GTX-102 (apazunersen) for Angelman syndrome across all genotypes and ages 1 to <65.
Aurora will enroll ~60 participants in four cohorts based on age and genotype; primary endpoints include Bayley-4 and MDRI.
Phase 3 Aspire study fully enrolled with 129 participants (ages 4-17, UBE3A deletion), data expected H2 2026.
Aurora expands patient population beyond deletion-type to include UPD, ICD, and mutation genotypes.
Cohorts A, B, C single-arm; Cohort D randomized 2:1 GTX-102 vs no treatment with crossover at Week 24.

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