Ovid fails to transform gaboxadol’s fortunes

Ovid Therapeutics’ bid to repurpose the failed sedative gaboxadol as a rare disease drug hit a snag yesterday with unconvincing data in Angelman syndrome. Still, with a lack of options in the disorder, the company appears to be pressing ahead with the project, which it calls OV101. Ovid said that the phase II Stars trial met its primary endpoint, with OV101 showing a similar safety profile to placebo – notable as adverse events previously scuppered gaboxadol. However, five seizures in the OV101 groups raised eyebrows; although Angelman patients often have seizures, none were seen in the placebo arm. But it is efficacy where the project really fell down. Ovid claimed a win on what it called the first prespecified efficacy endpoint, the CGI-I score, but OV101 failed to show a benefit on a huge list of other measures – on clinicaltrials.gov CGI-I is listed as the sixteenth of 17 secondary endpoints. Ovid said it would seek an audience with regulators and begin an open-label study on the back of the Stars data but investors were less convinced, sending its shares down 36% yesterday. Ovid is also developing OV101 in fragile X syndrome, where it recently began the phase II Rocket trial.