Rare Daily Staff

Ovid Therapeutics said topline data from a mid-stage trial showed its experimental therapy OV101 for the rare condition Angelman syndrome met its primary endpoint of safety and tolerability, but raised concerns over the efficacy of the treatment.

Angelman syndrome is characterized by symptoms that include delayed development, intellectual disability, severe speech impairment, problems with movement and balance, seizures, sleep disorders, and anxiety. The most common cause of Angelman syndrome is the loss of function of a gene that codes for a protein that plays a critical role in nerve cell communication. This loss of function results in impaired tonic inhibition, a central physiological process of the brain that is thought to be the underlying cause of certain neurodevelopmental disorders.

OV101 is believed to be the first investigational drug to specifically target the disruption of tonic inhibition. OV101 has been demonstrated in laboratory studies and animal models to impact neuronal activity through tonic inhibition. Ovid is developing OV101 for the treatment of Angelman syndrome and Fragile X syndrome to potentially restore tonic inhibition and relieve several of the symptoms of these disorders.

The company said the mid-stage study of OV101 showed a favorable safety profile and that the experimental therapy was well tolerated in adults and adolescents with Angelman syndrome.

At the prespecified efficacy analysis at 12 weeks of treatment, OV101 showed a statistically significant improvement compared to placebo in the physician-rated clinical global impressions of improvement (CGI-I) – a measure commonly used in clinical trials that allows the physician to capture a constellation of clinical symptoms.

But subsequent analyses were conducted on a prespecified subset of measures of behavior, sleep, and gait. The analysis of those prespecified subsets did not show a statistically significant difference from placebo. The company said full data analyses on these areas are ongoing and will be communicated in the future.

“The STARS study was designed to provide information to allow us to progress the development of OV101,” said Amit Rakhit, chief medical and portfolio management officer of Ovid Therapeutics. “With these findings, we have advanced our understanding of relevant endpoints to evaluate key symptoms of Angelman syndrome.

Furthermore, we demonstrated that a once-daily dose of OV101 could be sufficient to drive clinically meaningful benefit to patients. We look forward to discussing the data with regulatory authorities to inform our future development plans.”

The FDA has granted Orphan Drug and Fast Track designations for OV101 for both the treatment of Angelman syndrome and Fragile X syndrome.

August 6, 2018
Photo: Amit Rakhit, chief medical and portfolio management officer of Ovid

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