Ovid fails to transform gaboxadol’s fortunes

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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.

Stars trial – efficacy endpoints ordered as per Clinicaltrials.gov
Endpoint Pooled treatment groups Placebo p value
mPOMA-G (gait) Not given Not given Not significant
BSID-III (development) Not given Not given Not significant
PEDI-CAT (disability) Not given Not given Not significant
Sleep onset latency Not given Not given Not significant
Total sleep time Not given Not given Not significant
Wake after sleep onset Not given Not given Not significant
Nocturnal awakenings Not given Not given Not significant
Sedentary behaviour Not given Not given Not significant
Daily activity level Not given Not given Not significant
Aberrant behaviour checklist Not given Not given Not significant
Anxiety, depression and mood scales Not given Not given Not significant
EQ5D5L (quality of life) Not given Not given Not significant
Child health assessment questionnaire Not given Not given Not significant
Parent global impression Not given Not given Not significant
SF-36 (quality of life) Not given Not given Not significant
CGI-I 66.7% 39.3% 0.0206
CGI-S Not given Not given Not significant

mPOMA-G: Modified performance oriented mobility assessment-gait; BSID-III: Bayley scales of infant and toddler development; PEDI-CAT: Pediatric evaluation of disability inventory-computer adaptive test; EQ5D5L: EuroQol 5 dimension 5 level; SF-36: Short form health survey 36 items; CGI-I: Clinical global impression-improvement; CGI-S: Clinical global impression-severity.

Source: Clinicaltrials.gov.

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