Angelman syndrome is a rare neurodevelopmental disorder caused by a genetic mutation – usually a mutation in the ubiquitin protein ligase E3a (UBE3A) gene – that leads to a reduction in UBE3A protein. Symptoms include physical abnormalities (small head, specific facial appearance), severe intellectual disability, developmental disability, speaking problems, balance and movement problems, seizures, and sleep problems.

Persons with Angelman syndrome usually live a normal lifespan but there is currently no treatment specifically for the condition and therapy is focused on managing the various medical and developmental issues that arise in these individuals.

In this video, Amit Rahkit, MD, MBA, chief medical officer at Ovid Therapeutics discusses his company’s drug, OV101, a GABAA agonist that is showing promise in clinical trials.

Dr. Rahkit said, “We recently announced our phase II study results, the STARS results, which showed a very important indicator of improvement in Angelman syndrome and that kind of global functioning described by a measure called the clinical global impressions of improvement or CGI-I.”

“This study was highlighted in the American Academy of Neurology scientific sessions in 2019 and we’re very proud to say that it was chosen by the chair of the Scientific Committee as one of the top three presentations,” noted Dr. Rahkit, adding that the company is currently in discussions with various regulatory agencies to begin their phase III study, known as the NEPTUNE study.

The NEPTUNE study will enroll people with Angelman syndrome ages four to twelve years of age and compare OV101 given once-daily with placebo. The primary outcome measure will be the Angelman syndrome specific CGI-I scale. Results from that study are expected in mid 2020.

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