Ovid Therapeutics has initiated the Phase 2 ROCKET clinical trial to assess the safety, tolerability, and efficacy of OV101 (gaboxadol) as a treatment for adolescents and young adults with fragile X syndrome.
The study is expected to enroll about 30 male patients with clinically diagnosed fragile X syndrome, between the ages of 13 and 22. Participants will be randomized to receive one of three pre-selected doses of OV101 for up to 12 weeks.
The trial’s main goal is to evaluate the incidence of adverse events in the three different treatment groups. Researchers will also assess changes in behavior during the 12 weeks of treatment compared to baseline.
“We are pleased to advance our Fragile X program into a Phase 2 trial and to have taken the next important step in potentially developing a much needed treatment option for people living with this condition,” Amit Rakhit, chief medical and portfolio management officer at Ovid Therapeutics, said in a press release.
OV101 was designed to specifically inhibit the delta-selective GABAA receptor in the brain, which is believed to be involved in various neurodevelopmental disorders. Ovid is currently developing this compound to potentially restore tonic inhibition — a key feature of the brain to filter real neural signals from background brain activity — and improve symptoms in Angelman syndrome and fragile X syndrome patients.
In March, the U.S. Food and Drug Administration granted OV101 fast-track designation for the treatment of fragile X syndrome. This added to its previously granted orphan drug designation for the same indication. These actions are expected to accelerate development and regulatory review of the investigational therapy, and ultimately obtain its approval.
“At Ovid, we understand the critical role of tonic inhibition in neurological disorders and believe that our deep insights provide us the opportunity to develop OV101 as a potentially transformative medicine for the Fragile X community,” Rakhit said.
Preclinical data presented earlier this year at the 2018 Annual Meeting of the American Academy of Neurology showed that treatment with OV101 significantly reduced anxiety, hyperactivity, irritability, and aggressive behavior. The investigational therapy also effectively prevented animals from displaying restricted and repetitive behaviors.
The investigational drug already was studied in a Phase 1 clinical trial (NCT03109756) that enrolled 12 participants, ages 13 to 17, and diagnosed with Angelman or fragile X syndrome. The study revealed that a single 5 mg dose of OV101 had a similar safety and tolerability profile in both adolescents and young adults.
“Despite [fragile X syndrome being] the most common inherited form of intellectual disability, there are currently no approved therapies for the disorder. Current standard of care … is limited, relying primarily on symptomatic treatments,” said Randi Hagerman, MD, principal investigator and medical director of the MIND Institute at the University of California Davis. “Through this study, we hope to confirm the safety of OV101 and observe effects on behavioral endpoints.”