Ovid Therapeutics Inc. (NASDAQ: OVID) shares were crushed to start the week after the company announced positive midstage results for its Angelman syndrome study. Specifically, the data came from the Phase 2 STARS trial of OV101, which achieved its primary endpoint of safety and tolerability.

Angelman syndrome is a rare, lifelong, genetic disorder that affects one in 15,000 people in the United States. It is characterized by severe impairment in behavior, learning, verbal communication, motor skills and sleep, and there are no FDA-approved medicines or an established treatment paradigm for this condition.

Overall, OV101 showed a statistically significant improvement compared to placebo in the physician-rated clinical global impressions of improvement. Further analysis was conducted in the hierarchy of symptoms on a prespecified subset of scales across the domains of behavior, sleep and gait. However, the analysis of these prespecified subsets did not show a statistically significant difference from placebo, but full data analyses on these domains are ongoing and will be communicated in the future, says the company.

Ovid intends to discuss these data with regulatory authorities to determine the next steps for a registrational pathway. Based on these data, the company plans to initiate in the fourth quarter of 2018 an open-label extension study (named ELARA).

Ron Thibert, DO, MsPH, chair, STARS clinical trial steering committee, director, Angelman syndrome clinic at MassGeneral Hospital for Children, and assistant professor Harvard Medical School, commented:

The data reported today are the first data in Angelman syndrome to show a compound specifically targeting the syndrome having a clinical effect. Ovid is the first company to have conducted a double-blind, placebo-controlled study in Angelman syndrome, providing important clinical and scientific data. Based on these data, I believe OV101 has the potential to offer a clinically meaningful benefit specific to people living with Angelman syndrome.

Shares of Ovid were last seen down about 33% at $6.38, with a consensus analyst price target of $23.50 and a 52-week trading range of $5.28 to $12.44.

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