Ovid Therapeutics Announces Initiation of Phase 2 ROCKET Clinical Trial to Evaluate OV101 in Fragile X Syndrome
OV101 is a novel delta (δ)-selective GABAA receptor agonist that targets the disruption of tonic inhibition, a central physiological process of the brain that is thought to be the underlying cause of Fragile X syndrome and other neurodevelopmental disorders. It is believed that OV101 is the first investigational medicine to target the disruption of tonic inhibition.
"Fragile X syndrome is a rare neurogenetic condition associated with distinctive behavior characteristics and challenges, including anxiety and hyperactivity, and compounded with learning disabilities," said
"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," said
About the ROCKET Trial
The ROCKET trial is a Phase 2 randomized, double-blind, parallel-group, trial to evaluate the safety, tolerability and efficacy of OV101 in up to 30 males age 13 to 22 with a confirmed diagnosis of Fragile X syndrome. The primary endpoint of the study is safety and tolerability of OV101 over 12 weeks of treatment in three different cohorts. The secondary efficacy endpoint will evaluate changes in behavior during 12 weeks of treatment.
In addition to Fragile X syndrome, Ovid is also developing OV101 for the treatment of Angelman syndrome. Topline data from the company's Phase 2 STARS clinical trial investigating OV101 for the treatment of adults and adolescents with Angelman syndrome are expected in the third quarter of 2018.
About Fragile X Syndrome
Fragile X syndrome is the most common inherited form of intellectual disability and autism, with a prevalence of 1 in 3,600 to 4,000 males and 1 in 4,000 to 6,000 females in
In studies of individuals with Fragile X syndrome and in experimental models, extrasynaptic GABA levels are abnormally reduced, and there is also dysregulation of GABA receptors. This ultimately contributes to a decrease in tonic inhibition, causing the brain to become inundated with signals and lose the ability to separate background noise from critical information.
OV101 (gaboxadol) is believed to be the only delta (δ)-selective GABAA receptor agonist in development and the first investigational medicine to specifically target the disruption of tonic inhibition, a central physiological process of the brain that is thought to be the underlying cause of certain neurodevelopmental disorders. OV101 has been demonstrated in laboratory studies and animal models to selectively activate the δ-subunit of GABAA receptors, which are found in the extrasynaptic space (outside of the synapse), and thereby impact neuronal activity through tonic inhibition.
Ovid is developing OV101 for the treatment of Angelman syndrome and Fragile X syndrome to potentially restore tonic inhibition and relieve several of the symptoms of these disorders. In preclinical studies, it was observed that OV101 improved symptoms of Angelman syndrome and Fragile X syndrome. Gaboxadol has previously been tested in over 4,000 patients (with more than 1000 patient-years of exposure) and was observed to have favorable safety and bioavailability profiles.
For more information on Ovid, please visit http://www.ovidrx.com/.
This press release includes certain disclosures that contain "forward-looking statements," including, without limitation, statements regarding (i) progress, timing, scope and results of clinical trials for Ovid's product candidates, and (ii) the potential clinical benefit of OV101 to treat patients with Fragile X. You can identify forward-looking statements because they contain words such as "will," "believes" and "expects." Forward-looking statements are based on Ovid's current expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that may differ materially from those contemplated by the forward-looking statements, which are neither statements of historical fact nor guarantees or assurances of future performance. Important factors that could cause actual results to differ materially from those in the forward-looking statements are set forth in Ovid's filings with the
Ovid Therapeutics, Inc.
Senior Director, Investor Relations & Public Relations
Kelly Boothe, Ph.D.