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Ovid begins late-stage trial for rare disease treatment, defying Wall Street’s expectations

Dr. Jeremy Levin, the CEO of Ovid Therapeutics, is working to convince Wall Street that a controversial measure for testing a treatment is valid. Ovid

Ovid Therapeutics may be one step closer to vindication with a rare disease treatment whose polarizing results led Wall Street to all but abandon the small drug company, which is based in New York and has an office in Boston.

After a meeting with the Food and Drug Administration, Ovid is taking its therapy for the debilitating Angelman syndrome into a late-stage trial, one that will use the same definition of success employed in a prior study. The company said Thursday that the FDA had agreed with its approach to determining whether Angelman patients are getting better. And that means Ovid could win FDA approval by replicating what it saw in an earlier trial.


There are currently no treatments for Angelman, and approval would bring hope to the thousands of patients with the inherited neurological disorder, many of whom are unable to walk, talk, or sleep through the night. It would also mark a dramatic change to the narrative surrounding Ovid.

In 2018, the company disclosed data from an 88-patient trial in which its drug, OV101, beat placebo on one measure of the disease but failed on a host of others. To Ovid, it was a positive result that lit the way to a confirmatory study. To many investors, it looked like dumb luck that would never convince the FDA. The company’s stock price has traded at an 80 percent discount ever since.

At issue is Ovid’s chosen measurement. It’s called the clinical global impressions of improvement, or CGI-I, and it’s a seven-point scale doctors use to score whether patients’ symptoms are improving. According to Dr. Jeremy Levin, Ovid’s CEO, that makes it ideal for a disease like Angelman, in which symptoms can vary in both type and severity. One patient may struggle to sleep but be able to walk; another could be virtually immobile but capable of speech.


That logic wasn’t terribly convincing on Wall Street, but it has since resonated with the FDA, Levin said.

“They agreed to it, I think, in large measure because they understand from physicians that this disorder affects all of the parts of the brain, and there are so many different symptoms that it’s not enough to just affect any one of them,” he said. “You’ve got to have a totality of an effect to know that the medicine is going to be beneficial.”

To Dr. Christopher Keary, a psychiatrist who treats Angelman patients at Massachusetts General Hospital’s Lurie Center for Autism, CGI-I’s strength is its breadth. Each patient gets evaluated on nine key symptoms of the disease, including sleep and motor skills, at the outset of the trial and then again after 12 weeks. That gives doctors a holistic view on whether OV101 is having a positive effect on a complex disease, said Keary, who has served as a paid adviser to Ovid.

“In my mind, it is a very useful assessment tool because it really comes the closest to what I’m asking myself as a caregiver: Is this person better or worse or no different because of a treatment I gave?” he said. “I don’t think there’s a better measure for capturing all that is affected in Angelman syndrome.”

In the coming months, Ovid will enroll 60 Angelman patients between the ages of 4 and 12, randomizing them to receive either OV101 or placebo. The primary goal is improving CGI-I, and Ovid expects to have data in the middle of 2020.


If everything goes well, Ovid will use the results of the latest trial and the prior one, which included adults and teenagers, to make its case for an FDA approval that covers all Angelman patients.

In the meantime, the company has its work cut out for it with investors. Ovid’s share price has dawdled below $2 for most of 2019. The company is hosting an analyst event in New York on Thursday to make the case for OV101 and the other neurological treatments in its pipeline. Levin, who previously led the multibillion-dollar generics company Teva Pharmaceutical, is confident he can bring financiers over to his — and the FDA’s — way of thinking.

“The breadcrumbs have been laid down, and we’re making it easier for them to understand it,” Levin said. “The bottom line is we absolutely need to deliver. That’s what we need to do.”

Damian Garde can be reached at Follow him on Twitter @damiangarde. Follow Stat on Twitter: @statnews.