Ultragenyx reports positive Phase 1/2 GTX-102 data in Angelman syndrome with rapid, sustained improvements

Ultragenyx Pharmaceutical Inc.

Expansion Cohorts A&B (24 patients) at Day 170 showed clinically meaningful improvements across cognition, behavior, sleep, motor, and communication domains vs natural history.
Multi-domain responder index (MDRI) net response of +2.0 (p<0.0001) at Day 170; majority of patients achieved +2 to +4 domain improvement.
Dose-escalation Cohorts 4-7 (15 patients) at Day 758 showed sustained long-term benefit; MDRI net response +2.0 (p=0.0007) at Day 338.
No unexpected serious adverse events; three patients had mild-moderate lower extremity weakness (resolved, no recurrence on re-dosing).
FDA notified of safety events; no issues raised or additional actions required; data to be presented at AAN 2024.

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