Ultragenyx fully enrolls Phase 3 Aspire study (129 patients) for GTX-102 in Angelman Syndrome

Ultragenyx Pharmaceutical Inc.

Phase 3 Aspire study fully enrolled with ~129 patients ages 4-17 with maternal UBE3A deletion.
Participants randomized 1:1 to GTX-102 intrathecal injection or sham comparator for 48 weeks; sham can cross over after Week 48.
Primary endpoint: Bayley-4 cognitive raw score improvement; key secondary: MDRI across five domains.
Study completion expected H2 2026; company plans to move to topline data and regulatory submission.

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