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Ovid Therapeutics to Present at the American Academy of Neurology 71st Annual Meeting

Emerging Science platform presentation highlights efficacy and safety data of OV101 in Angelman syndrome from the STARS study 

Research from Ovid’s Fragile X program shows new insights for outcome assessments

NEW YORK, April 18, 2019 (GLOBE NEWSWIRE) -- Ovid Therapeutics Inc. (OVID) continues to build on its neuroscience research platform with the presentation of data from the OV101 (gaboxadol) investigational program for rare neurodevelopmental conditions.  Ovid’s presentations on Angelman syndrome and Fragile X syndrome are part of the program at the 71st annual meeting of the American Academy of Neurology (AAN) in Philadelphia (May 4 - 10). The platform presentation and posters will highlight the data from OV101 in adolescents and adults with Angelman syndrome (STARS study) as well as frameworks to assess outcomes in Fragile X syndrome as a precursor to data that Ovid expects to announce in 2H 2019 (ROCKET study). 

“Ovid’s research efforts are focused on finding treatments for rare diseases, which are defined in the US as conditions affecting fewer than 200,000 people, and specifically, we seek to improve the lives of the approximately 8 million people in the US alone living with neurologic manifestations of rare diseases1,” said Amit Rakhit, MD, MBA, Chief Medical Officer and Head of Research & Development at Ovid. “The data we are presenting at AAN reflect the dedication of our R&D teams to investigate the use of OV101 as a potential treatment for Angelman syndrome and Fragile X syndrome, with each condition having high unmet medical need and no approved therapies.”

Ovid Therapeutics continues to expect the initiation of NEPTUNE, a global Phase 3 study of OV101 in children with Angelman syndrome in 2H 2019.

Details of the presentations are listed below.

Title: Methodology for Development of Indication-Specific Outcome Measures in Rare Disease Trials: An Innovative Research Approach
Poster ID: P1.9-073 
Poster Session 1: Research Methodology, Clinical Outcome and Quality Improvement 
Date and Time: Sunday, May 5, 2019, 11:30 a.m. – 6:30 p.m. ET

Title: Development of a Conceptual Model to Inform a Clinical Outcome Assessment Strategy in Adolescents and Young Adults with Fragile X Syndrome
Poster ID: P1.9-067  
Poster Session 1: Research Methodology, Clinical Outcome and Quality Improvement 
Date and Time: Sunday, May 5, 2019, 11:30 a.m. – 6:30 p.m. ET

Title: STARS: Results from a Safety and Efficacy Study of OV101 (gaboxadol) in Adults and Adolescents with Angelman Syndrome
Session: Emerging Science
Date and Time: Tuesday, May 7, 2019, 11:45 a.m. – 12:20 p.m. ET; Poster presentation from 12:20 p.m. – 12:45 p.m. ET

About Ovid Therapeutics
Ovid Therapeutics (OVID) is a New York-based biopharmaceutical company using its BoldMedicine™ approach to develop medicines that transform the lives of patients with rare neurological disorders. Ovid has a broad pipeline of potential first-in-class medicines. The company’s most advanced investigational medicine, OV101, is currently in clinical development for the treatment of Angelman syndrome and Fragile X syndrome. Ovid is also developing OV935/TAK-935 in collaboration with Takeda Pharmaceutical Company Limited for the potential treatment of rare developmental and epileptic encephalopathies (DEE).

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 thereby address the core symptoms of these disorders. In both these syndromes the underlying pathophysiology includes disruption of the tonic inhibition modulated through the δ-subunit of GABAA receptors. In preclinical studies, it was observed that OV101 improved symptoms of Angelman syndrome and Fragile X syndrome. This compound has also previously been tested in over 4,000 patients (over 1,000 patient-years of exposure) and was observed to have favorable safety and bioavailability profiles. In 2018 Ovid announced the successful completion of its Phase 2 (STARS) trials of OV101 in adults and adolescents with Angelman syndrome. In 2H 2019, Ovid expects to initiate a pivotal Phase 3 (NEPTUNE) clinical trial in children aged 4-12 years old. In addition, Ovid is conducting a Phase 2 clinical trial (ROCKET) in Fragile X with results expected in 2H 2019.

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 and Fragile X syndrome using OV101. The issued patents expire in 2035 without regulatory extensions.

About Angelman Syndrome
Angelman syndrome is a rare genetic disorder that is characterized by a variety of signs and symptoms. Characteristic features of this disorder include delayed development, intellectual disability, severe speech impairment, problems with movement and balance, seizures, sleep disorders and anxiety. The most common cause of Angelman syndrome is the loss of function of the gene that codes for ubiquitin protein ligase E3A (UBE3A), which plays a critical role in nerve cell communication, resulting in impaired tonic inhibition. Individuals with Angelman syndrome typically have normal lifespans but are unable to live independently, therefore, they require constant support from a network of specialists and caregivers. Angelman syndrome affects approximately 1 in 12,000 to 1 in 20,000 people in the U.S.

There are no FDA-approved therapies for Angelman syndrome, and treatment primarily consists of behavioral interventions and pharmacologic management of symptoms.

Angelman syndrome is associated with a reduction in tonic inhibition, a function of the delta (δ)-selective GABAA receptor that allows a human brain to decipher excitatory and inhibitory neurological signals correctly without being overloaded. If tonic inhibition is reduced, the brain becomes inundated with signals and loses the ability to separate background noise from critical information.

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. We believe this in turn affects GABA synthesis and appears to impact tonic inhibition.

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. We believe 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. Like the pathophysiology seen in Angelman syndrome, that of Fragile X is associated with a reduction in tonic inhibition, a function of the delta (δ)-selective GABAA receptor that allows a human brain to decipher excitatory and inhibitory neurological signals correctly without being overloaded.

1 Source: National Institute of Neurological Disorders and Stroke (NINDS) website.

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 advancing Ovid’s product candidates, progress, timing, scope and results of clinical trials for Ovid’s product candidates, and the reporting of clinical data regarding Ovid’s product candidates. 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 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:
Investor Relations & Public Relations
Ovid Therapeutics Inc.
irpr@ovidrx.com

Alex Gray
Burns McClellan, Inc.
agray@burnsmc.com
(212) 213-0006

Media:
Investor Relations & Public Relations
Ovid Therapeutics Inc.
irpr@ovidrx.com