Ovid to Present Data on Potential Angelman, Fragile X Therapy During Quebec Workshop

Ovid Therapeutics will present new data from its lead investigational drug, OV101, to treat neurodevelopmental symptoms of diseases like Angelman syndrome and Fragile X syndrome, at the 18^th International Fragile X and Related Neurodevelopmental Disorders Workshop.

The conference begins today in Saint-Alexis-des-Monts, Quebec. On Oct. 15, Dr. Brett Abrahams, Ovid’s director and head of preclinical biology, will present the poster, “Gaboxadol normalizes behavioral abnormalities in a mouse model of Fragile X Syndrome,” the company announced.

OV101, also known as gaboxadol, is a potent and orally available agonist of the delta-selective GABA_A receptor, derived from a mushroom called Amanita muscaria. Neurons use a molecule called GABA and their GABA_A receptors to dampen down excessive activity between neurons, filtering sensory information and fine-tuning neuronal function. This process is called tonic inhibition.

In Angelman, the loss of the UBE3A gene function leads to an accumulation of GABA transporters, which mop up the GABA molecules from neuronal synapses. This deficiency in GABA leads to a loss of tonic inhibition, believed to be a fundamental mechanism underlying the symptoms of neurodevelopmental disorders like Angelman.

But OV101 can selectively activate the delta-subunit of GABA_A receptors, restoring neuronal activity balance by triggering tonic inhibition. This effect on GABA_A receptors improved walking capacity and balance in mice models of Angelman, and reduced hyperactivity and sound sensitivity in mice models of Fragile X syndrome, said New York-based Ovid.

Ovid’s drug candidate is currently under clinical development for both indications, in a Phase 1 and a Phase 2 trial.

A Phase 1 clinical trial (NCT03109756) is evaluating the effects of a single dose of OV101 in teens 13 to 17 years old with Angelman or Fragile X syndrome. The study aims to identify the optimal therapeutic dose of OV101 to be used in future trials. Topline data is expected in late 2017.

The STARS Phase 2 trial (NCT02996305) is assessing the safety and efficacy of twice-daily OV101 versus placebo in adults with Angelman  The trial is still recruiting participants, and interim results are expected during 2018. The company is also planning to test OV101 in a younger pediatric population. This future trial is pending completion of the Phase 1 trial and additional preclinical toxicity studies.

Ovid’s OV101 received orphan drug designation by the U.S. Food and Drug administration (FDA) to treat Angelman in September 2016, and to treat Fragile X syndrome in October 2017.