Phase 2 STARS Trial Results Demonstrate Efficacy of OV101 for Treating Angelman Syndrome

Ovid Therapeutics Inc., a biopharmaceutical company, recently announced that the phase 2 STARS trial, which tested the efficacy of the investigational drug OV101, achieved its primary endpoint of safety and tolerability for treating Angelman syndrome, according to press release.

OV101 is an investigational medicine that is the only selective extrasynaptic GABA_A receptor agonist in development shown to mediate tonic inhibition, which is a known underlying pathophysiological mechanism of Angelman syndrome—a rare genetic disorder that is characterized by severe impairment in behavior, learning, verbal communication, motor skills, and sleep.

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The study represented the first industry-sponsored, randomized, double-blind, placebo-controlled clinical trial specifically for Angelman syndrome. The study involved 88 patients who were divided into 3 groups: once-daily dose of OV101, twice-daily daily dose of OV101, or placebo.

“We are excited by these data, as this is the first demonstration of positive clinical effect on overall symptomology in Angelman syndrome,” said Jeremy Levin, DPhil, MB, BChir, chairman and chief executive officer of Ovid Therapeutics. “In collaboration with the Angelman community, we designed a robust study to evaluate prespecified endpoints that may pave the way for a registrational pathway for a disorder that has no previously approved medicines. These data are a tribute to the patients and their families, and we thank them.”

Over the course of 12 weeks of treatment, the researchers found that OV101 resulted in statistically significant improvement when compared to the placebo. The study used the physician-rated clinical global impressions of improvement (CGI-I), a measure that allows the physician to capture a constellation of clinical symptoms in order to measure the efficacy of the OV101 for treating the disease.

“These initial data from the STARS study are encouraging, particularly the statistically significant improvement in overall symptoms that we see in the CGI-I scale in the once-daily dosing group. Angelman syndrome is a complex disorder, and the CGI-I scale captures the totality of global neurological deficits and helps to define the impact of medicines on the individual and their families,” said Ron Thibert, DO, MsPH, chairperson of the STARS clinical trial steering committee and director of the Angelman syndrome clinic at Massachusetts General Hospital for Children and assistant professor Harvard Medical School. “Based on these data, I believe OV101 has the potential to offer a clinically meaningful benefit specific to people living with Angelman syndrome.”

Subsequent analyses were conducted on prespecified subsets, such as the domains of behavior, sleep, and gait, and did not show a statistically significant difference from the placebo. However, full data analysis on these specific domains are still ongoing.

The researchers noted that this study represents an advanced understanding of relevant endpoints to evaluate Angelman syndrome and demonstrates that a once-daily dose of OV101 could be effective for patients with this disease.