Clinical Edge

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Angelman Syndrome Treatment Safe, Effective in Exploratory Analyses

Key clinical point: OV101 (gaboxadol), a type A gamma-aminobutyric acid receptor agonist, was safe and well tolerated in a phase 2 trial, and improved clinical outcomes in exploratory analyses.

Major finding: Clinician-rated clinical global impressions of improvement were superior for gaboxadol versus placebo (P = .0006), informing further development of a phase 3 trial.

Study details: An analysis of data for 78 patients with Angelman syndrome who completed the STARS study, a 12-week, randomized, phase 2 trial that compared OV101 with placebo.

Disclosures: Funding for the study came from Ovid Therapeutics. Dr. Kolevzon reported disclosures related to Ovid Therapeutics, as well as Coronis Neurosciences, 5AM Ventures, SEMA4, LabCorp, and AMO Pharma, among others.