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Ovid Therapeutics Announces OV101 Granted Fast Track Designation by U.S. FDA for Treatment of Fragile X Syndrome

Second Fast Track Designation for OV101 within Three Months

NEW YORK, March 15, 2018 (GLOBE NEWSWIRE) -- Ovid Therapeutics Inc. (NASDAQ:OVID), a biopharmaceutical company committed to developing medicines that transform the lives of people with rare neurological diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to OV101 for the treatment of Fragile X syndrome.  

“Fragile X syndrome continues to have a high unmet medical need and has no FDA-approved therapies available. Fast Track designation enables us to work closely with the FDA to accelerate our efforts to potentially provide an impactful therapy to people with Fragile X syndrome,” said Amit Rakhit, M.D., MBA, chief medical and portfolio management officer of Ovid Therapeutics. “This important designation by the FDA comes on the heels of several other clinical and regulatory milestones for our OV101 program in recent months, including positive Phase 1 data, orphan drug designation for Fragile X syndrome, and Fast Track designation for Angelman syndrome. We are committed to exploring the potential of OV101 to become a transformative medicine and we look forward to announcing further progress this year.”

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 FDA throughout the drug development and review process, often leading to faster drug approval and patient access.

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 the United States. Individuals with Fragile X syndrome often have a range of behavioral challenges, such as cognitive impairment, anxiety, mood swings, hyperactivity, attention deficit, poor sleep, self-injury and heightened sensitivity to various stimuli, such as sound. Additionally, individuals with Fragile X syndrome are prone to comorbid medical issues including seizures and sleep disturbance. Fragile X syndrome results from mutations in the FMR1 gene, which blocks expression of the Fragile X Mental Retardation Protein (FMRP), an important protein in GABA synthesis. There are no FDA-approved therapies for Fragile X syndrome, and treatment primarily consists of behavioral interventions and pharmacologic management of symptoms.

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.

About OV101 
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.

About Ovid Therapeutics
Ovid Therapeutics (NASDAQ:OVID) is a New York-based biopharmaceutical company using its BoldMedicine™ approach to develop therapies that transform the lives of patients with rare neurological disorders. Ovid’s drug candidate, OV101, is currently in development for the treatment of Angelman syndrome and Fragile X syndrome. Ovid initiated the Phase 2 STARS trial of OV101 in people with Angelman syndrome in 2017 and completed a Phase 1 trial in adolescents with Angelman syndrome or Fragile X syndrome. Ovid is also developing OV935 in collaboration with Takeda Pharmaceutical Company Limited for the treatment of epileptic encephalopathies and in 2017 initiated a Phase 1b/2a trial of OV935.

For more information on Ovid, please visit http://www.ovidrx.com/.

Forward-Looking Statements
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 Securities and Exchange Commission, including its Quarterly Report on Form 10-Q for the quarter ended September 30, 2017, under the caption “Risk Factors.” Ovid assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available. 

Contacts
Investors:
Burns McClellan
Steve Klass, 212-213-0006
Sklass@burnsmc.com   

Media:
W2O pure
Katie Engleman, 910-509-3977
kengleman@w2ogroup.com  

 

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