New data back Ovid’s Angelman drug, say analysts

Jeremy Levin - Credit: Kelly Boothe, agent of Ovid Therapeutics - Own work - CC BY-SA 4.0
CEO Jeremy Levin. (Kelly Boothe, agent of Ovid Therapeutics - Own work - CC BY-SA 4.0)

After reporting mixed results in a midstage trial earlier this year, Ovid Therapeutics has new data for its OV101 drug for rare disease Angelman syndrome that it says are looking a lot stronger.  

The STARS trial—reported at the American Academy of Child and Adolescent Psychiatry (AACAP) conference this week—is the first industry-sponsored trial in Angelman, a disorder caused by a defect in the UBE3A gene that leads to delayed development, seizures, sleep problems and others medical issues.

Ovid reported preliminary results from the STARS trial in August, with improvements across a range of measures that helped it achieve a significant improvement in Clinical Global Impression of Improvement (CGI-I) scale from a single 15mg dose of OV101 given in the evening, and backed its safety and tolerability.


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There was some debate about the earlier readout, with analysts at Ladenburg Thalman saying it “suggested minimal benefits in sleep, motor, behavior or quality of life.” However, they now suggest the new data at AACAP has firmed up OV101’s clinical profile with “additional secondary endpoints with statistically significant or trends toward improvements.”

OV101 is a selective extra-synaptic GABA A receptor agonist vying to become the first-ever approved therapy for Angelman, which occurs in around 1 in 15,000 live births and is often misdiagnosed as cerebral palsy or autism.

CEO Jeremy Levin said on a conference call that with the STARS data in hand, the plan now is to start an open-label extension study before the end of the year for all OV101-treated patients, using the 15mg once daily dose.

“We believe the data…will support an end-of-phase 2 meeting with the FDA by the end of the year,” he continued, and that should clarify the clinical requirements for a regulatory filing.

Ladenburg Thalman think the likelihood is a second phase 2 trial in 2019 with a phase 3 study the following year, but suggest that Ovid may be able to proceed with a single phase 2/3 trial “as there is nothing approved or adequately used today to treat the problematic behaviors of Angelman.”

Other companies targeting Angelman include GeneTx Biotherapeutics, a startup launched by the Foundation for Angelman Syndrome Therapeutics (FAST) earlier this year that is focusing on the developing of antisense drugs for the disorder, and Marinus Pharma, which has a drug called ganaxolone in preclinical development for Angelman that has a similar mechanism of action to OV101. Other players include Sage Therapeutics and Zynerba Pharma.

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