Clinical Benefits of OV101 in Adolescents and Adults With Angelman Syndrome

Angelman syndrome
Angelman syndrome
Investigators found that OV101 (gaboxadol) was clinically beneficial in adults and adolescents with Angelman syndrome.

The following article is part of conference coverage from the 2019 American Academy of Neurology Annual Meeting (AAN 2019) in Philadelphia, PA. Neurology Advisor’s staff will be reporting breaking news associated with research conducted by leading experts in neurology. Check back for the latest news from AAN 2019.

PHILADELPHIA –  Investigators found that OV101 (gaboxadol) was clinically beneficial in adults and adolescents with Angelman syndrome (AS) according to research presented at the 2019 American Academy of Neurology Annual Meeting, held May 4-10, 2019, in Philadelphia, Pennsylvania.

AS is a neurodevelopmental disorder caused by a deficient UBE3A allele, which can result in seizures, speech impairments, behavioral issues, intellectual disability, and impairments in motor skills. Investigators conducted a phase 2, randomized, double-blinded, placebo-controlled trial to determine the safety, tolerability, and efficacy of OV101 ( Identifier: NCT02996305), an extrasynaptic delta-selective GABAA receptor agonist, in adolescents and adults with AS.  Participants were randomly assigned either OV101 10 mg daily, OV101 twice daily (10mg and 15 mg), or placebo. The primary outcome was to determine safety and tolerability in a 12-week period. To determine efficacy, investigators conducted assessments to evaluate motor function, sleep, and behavior using the Clinical Global Impressions-Improvement scale (CGI-I) and domain specific instruments.

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Of the total participants, 78 completed the study.  At week 12, there was global improvement observed according to the CGI-I scale with OV101 daily vs placebo (P =.0006).  Post-hoc analysis demonstrated improvement in motor function and overall sleep, including sleep onset latency. Those participants who demonstrated clinically meaningful improvements on the CGI-I (≤2), also showed improvements in challenging behavior, anxiety, and communication. Adverse events were mild between all groups.

Investigators concluded that “STARS is the first trial to demonstrate clinical benefit of OV101 in patients with AS, with improvements observed in sleep, motor, and behavior. These data suggest that CGI-I may be particularly well suited for assessing clinical improvements in a highly heterogeneous disorder like AS and may serve as a primary endpoint in future clinical trials.”  In addition, the CGI-I scale is a good tool to measure symptom improvement across multiple domains.

This study was funded by Ovid Therapeutics.  Multiple investigators have reported disclosures; please refer to the original article for a complete list.

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Bird L, Ochoa-Lubinoff C, Tam, W, et al. STARS: Results from a safety and efficacy study of OV101 (gaboxadol) in adults and adolescents with Angelman syndrome. Presented at: 2019 American Academy of Neurology Annual Meeting; May 4-10, 2019; Philadelphia, PA