Ovid Therapeutics Announces OV101 Granted Fast Track Designation by U.S. FDA for Treatment of Fragile X Syndrome
“Fragile X syndrome continues to have a high unmet medical need and has no
OV101 is a 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.
Ovid recently announced successful completion of a Phase 1 pharmacokinetic (PK) and safety study in patients with Fragile X syndrome and Angelman syndrome, demonstrating that OV101 has a similar safety and tolerability profile in adolescents as in young adults. These results will inform dosing in future clinical trials and support advancing the development of OV101 for the treatment of adolescents with Fragile X syndrome.
Ovid is moving forward with the clinical development program in Fragile X syndrome, demonstrating momentum for the overall OV101 program. Ovid intends to initiate a Phase 2 clinical trial in 2018, investigating OV101 for the treatment of males ages 13 to 22 diagnosed with Fragile X syndrome.
The FDA’s Fast Track process is designed to expedite the development and review of drugs used to treat serious conditions and fill an unmet medical need. Fast Track designation enables the company to have early and frequent communication with the
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 second half 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 drug 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 (approximately 950 patient-years of exposure) and was observed to have favorable safety and bioavailability profiles.
The FDA has granted orphan drug and Fast Track designations for OV101 for both the treatment of Angelman syndrome and Fragile X syndrome. The U.S. Patent and Trademark Office has granted Ovid patents directed to methods of treating Angelman syndrome using OV101. The issued patents expire in 2035 for Angelman syndrome.
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 progress, timing, scope and results of clinical trials for Ovid’s product candidates, the timing of clinical data, the development of therapies for younger patients, the provision of access to effective therapies, and the FDA Fast Track process leading to faster drug approval and patient access. 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