Ovid Therapeutics and NeuroPointDx said today they will partner to identify novel biomarkers of Angelman syndrome by analyzing metabolomic profile data, as part of Ovid’s Phase II STARS clinical trial designed to assess its lead candidate OV101 (gaboxadol).

“The metabolic profile of this syndrome is not well understood, and we believe this biomarker study will produce critical data to fill this gap and inform us about the impact of OV101 as a potential treatment option,” Ovid president and CSO Matthew During, M.D., D.Sc., FACP, FRACP, said in a statement. “This collaboration is the first step in Ovid’s broader rare neurological disorder biomarker strategy to identify molecular markers of treatment responders and guide enrollment of participants in our clinical trials.”

The collaboration is designed to apply NeuroPointDX’s expertise in metabolomics to identify biomarkers associated with Angelman syndrome, a rare neuro-genetic disorder. In STARS, the analysis is intended to provide molecular insights into disease's mechanism and assess the potential response to OV101 to help understand the physiological impact of the compound in people with Angelman syndrome. The analysis may help identify individuals that are most likely to respond to treatment, Ovid and NeuroPointDx said.

Ovid has initiated patient enrollment in STARS, with plans to enroll approximately 75 people with Angelman syndrome who are at least 18 years old to assess OV101.

OV101 is a delta (δ)-selective gamma-aminobutyric acid A (GABAA) receptor agonist being developed for Angelman as well as for Fragile X syndromes. According to Ovid, OV101 is believed to be the first investigational drug to target the disruption of tonic inhibition, a key mechanism allowing a healthy human brain to decipher excitatory and inhibitory neurological signals correctly without being overloaded.

The FDA granted its orphan drug designation to OV101 for Angelman syndrome in September. In preclinical models, OV101 has been able to selectively activate the δ-subunit of GABAA receptors, which are found in the extrasynaptic space, and helped to regulate neuronal activity through tonic inhibition.

NeuroPointDX uses its metabolomics platform technology to identify differences in children with autism spectrum disorders compared to neurotypical children and between subgroups of children on the spectrum for earlier diagnosis and more precise treatment.

NeuroPoinDX is the diagnostics division of Stemina Biomarker Discovery, a University of Wisconsin–Madison spinoff that has developed a proprietary platform for identifying biomarkers by screening blood samples using analytical equipment and its proprietary platform technology. In September 2015, Stemina launched a 1500-patient study, the Children’s Autism Metabolome Project (CAMP), funded by a $2.7 million grant from the National Institutes of Mental Health and funding from the Nancy Lurie Marks Family Foundation.

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