Ovid Therapeutics shares slump on Phase 2 Angelman drug results

Shares in the thinly traded biopharma group Ovid Therapeutics Inc (NASDAQ:OVID) slumped in midday trade upon the release of questionable results from a mid-stage clinical trial evaluating its lead drug OV101 in patients with Angelman syndrome.

The company said patients with Angelman taking OV101, also known as gaboxadol, saw a statistically significant improvement in a Phase 2 trial compared to a placebo when measured according to a scale called clinical global impressions of improvement (CGI-I), which  allows physicians to assess several symptoms together.

However, more specific analyses of measures of changes in behavior, sleep and gait failed to achieve statistical significance in these patients.

The company said additional research into these areas will continue.

“While the analysis of these prespecified subsets did not show a statistically significant difference from placebo, full data analyses on these domains are ongoing and will be communicated in the future,” the company said.

In the wake of the results, investors sent Ovid shares down by nearly 35% to US$6.19.

Phase 2 study marks first trial for Angelman syndrome

The Phase 2 study is the first randomized, double-blind, placebo-controlled clinical trial for Angelman syndrome, a rare genetic disorder that hits 1 in 15,000 people in the US.

Angelman is characterized by severe impairment in behavior, learning, verbal communication, motor skills and sleep. No FDA-approved medicines currently exist for the condition.

The Phase 2 study lasted 12 weeks and included the evaluation of 88 patients with Angelman between the ages of 13 and 49, which took place at trial sites in the US and Israel.

Investors might have been less than impressed by the trial’s results, but Jeremy Levin, Ovid’s chairman and CEO, was enthused about their holistic nature.

“We are excited by these data, as this is the first demonstration of positive clinical effect on overall symptomology in Angelman syndrome,” Levin said in a statement.

OV101 is a type of inhibitor of brain receptors known as GABA, which targets neurological dysfunction. The FDA has granted Orphan drug and Fast Track designations for OV101 for both the treatment of Angelman syndrome and Fragile X syndrome.

Contact Ellen Kelleher at [email protected]

Story by ProactiveInvestors