Ovid Therapeutics $OVID says it picked up a snapshot of broad, positive efficacy in its Phase II study of a new drug for rare cases of Angelman syndrome and will now turn to the FDA to see about mapping out a registrational pathway with the FDA for a pivotal test. But the biotech has to clear some hurdles ahead if it expects to convince analysts of the drug’s potential. And its share price was crushed Monday morning as skeptics pounced.
The drug is OV101, which passed the primary goal on safety and tolerability and also scored what Ovid CEO Jeremy Levin called the first clearly promising data recorded in a Phase II study for Angelman syndrome.
“This is the first time that anything has shown it has an impact,” Levin told me early Monday. “This is terrific.”
The market, though, didn’t take the same view, with Ovid shares crashing after the results were met with some initial skepticism about some failures also reported by Ovid. The stock was down 30% in pre-market trading.
The key efficacy endpoint — in Levin’s view — was based on the physician-rated clinical global impressions of improvement (CGI-I), which clinicians use to track a variety of symptoms for a disease that can leave patients unable to communicate or crippled with anxiety. On that collective endpoint, Ovid came away with a highly statistically significant p value of 0.0006 for the once-daily drug arm — which is exactly what they wanted to see.
The twice-daily drug arm, though, did not achieve statistical significance, falling well short of the mark. But Levin says that was expected after preclinical studies demonstrated that too much drug — designed to clarify signal from noise — would fail to rein in symptoms. On a pooled basis, he says the data are still positive, with a p value of 0.02.
That position attracted plenty of hostile reaction on Twitter this morning, though, as some questioned whether Ovid had spelled out a likely failure for the higher dose. Also, CGI was listed as the 16th of 17 secondary endpoints on clinicaltrials.gov, raising further suspicions that the data were more negative than the company asserted.
Ovid execs say that the drug was also scored on “a prespecified subset of scales across the domains of behavior, sleep and gait,” but failed to demonstrate statistical significance. Levin noted those scores relied on caregivers’ assessments, which aren’t always the best way of gathering data in a study.
The patients will now be followed on an ongoing basis.
The drug is an extrasynaptic GABAA receptor agonist. There are no approved therapy for this condition.
The best place to read Endpoints News? In your inbox.
Comprehensive daily news report for those who discover, develop, and market drugs. Join 41,800+ biopharma pros who read Endpoints News by email every day.Free Subscription