Dive Brief:

• An experimental drug from New York biotech Ovid Therapeutics has failed a Phase 3 trial in Angelman’s syndrome, a rare genetic disorder with no approved treatment.

• The Ovid drug, called OV101, did no better than placebo on a 7-point scale evaluating patients’ condition over three months of treatment, the study’s main goal. Angelman patients on OV101 experienced a 0.7-point improvement, while placebo patients’ scores climbed by 0.8 points. There was no difference between OV101 and placebo on secondary measures either.

• In a statement Tuesday, Ovid said it will evaluate future steps, “if any,” for OV101 in Angelman’s and focus instead on another experimental treatment, OV935, that is set to enter late-stage testing for multiple rare forms of epilepsy. The failure likely marks a disappointing end of a six-year effort by Ovid to repurpose an old drug for Angelman’s, a quest spearheaded by former Teva and Bristol Myers Squibb executive Jeremy Levin. Shares fell by 52% in post-marketing trading Tuesday.

Dive Insight:

After resigning from Teva in 2013, Jeremy Levin — who’d spent years as a pharmaceutical executive — unexpectedly emerged two years later as the CEO of a small biotech startup. His plan at the fledgling company, Ovid, was to find promising drug candidates gathering dust elsewhere, license them and develop them for rare brain diseases.

Soon after, Levin brokered a deal with Lundbeck to grab rights to an overlooked drug called gaboxadol. The drug had been tested for a range of conditions, but after disappointing as a potential non-addictive sleeping pill, Lundbeck and then-partner Merck stopped development in 2007.

Ovid co-founder Matthew During, a former molecular virology professor at Ohio State University and a neuroscience specialist, saw potential in gaboxadol as a treatment for Angelman’s disease. The drug boosts levels of an inhibitory neurotransmitter, which During thought could be helpful for Angelman, a condition characterized by a host of neurological, cognitive, and motor problems. After licensing the drug from Lundbeck, Ovid renamed it OV101 and went public in 2017, a move that gave it the funds to test its theory.

The company worked closely with patient groups to help inform its trial design for Angleman, aiming to better understand what kind of benefit would make the most difference for patients. Ovid consulted with doctors and caregivers, for example, to develop a scale to evaluate motor, sleep, communication and behavioral abilities.

While Ovid saw promising signs for OV101 in an 88-patient, Phase 2 trial in adults with Angelman, others were skeptical of data that showed the drug didn’t lead to clear improvement over placebo on some measures.

The skepticism appears to have been warranted, given Tuesday’s results from a Phase 3 trial called Neptune in children with the disease. Though Ovid will continue giving patients OV101 in an open-label extension study, Levin said the company would pause the rest of its work in Angelman “pending a full understanding of this outcome and discussions with regulators and investigators.”

The outcome is a “major disappointment,” wrote Brian Abrahams, an analyst at RBC Capital Markets, in a note. “It is possible the heterogeneous population was too challenging overcome and/or that the potential signals in adults did not translate to the pediatric population tested in Neptune,” he added, predicting a “low likelihood” that development will continue.

Ovid shares lost half their value on the news, although the company still has another drug it’s advancing into late-stage testing for a group of rare epilepsies. The biotech splits rights to that drug, OV935, with the Japanese pharmaceutical firm Takeda.