Ovid Therapeutics Dosed Healthy Volunteers with OV329 in Phase 1 Trial

A Potential, Next-Generation GABA-Aminotransferase Inhibitor for Rare Epilepsies with Limited Treatment Options

NEW YORK, Jan. 05, 2023 (GLOBE NEWSWIRE) — Ovid Therapeutics Inc. (Nasdaq: OVID), a biopharmaceutical company developing medicines designed to conquer epilepsies and meaningfully improve the lives of people affected by brain disorders, dosed healthy volunteers with OV329 in late December 2022, as part of a Phase 1 study. The Company is conducting the study to evaluate the safety and target engagement associated with single and repeated doses of OV329.

“Inhibiting the GABA-aminotransferase (GABA-AT) enzyme is a validated mechanism for reducing seizures,” said Dr. Claude Nicaise, Head of Research & Development at Ovid. “We believe OV329 is an improved, potent GABA-AT inhibitor that may confer robust seizure reduction, an improved safety profile, and preferred dosing relative to the current medicine in the class. We thank the epilepsy community and individuals participating in this Phase 1 study. Results will inform our development plans, and hopefully, enable us to rapidly advance into trials with people experiencing treatment-resistant seizures.”

The Phase 1 study of OV329 is a first-in-human, randomized, double-blind, placebo-controlled trial that is being conducted at Duke University School of Medicine. The trial is structured in two parts, beginning with a single-ascending dose portion and followed by a multiple-ascending dose portion. Both parts are cohort gated. This study in healthy volunteers will explore several endpoints including safety, tolerability, pharmacokinetics, and target engagement levels as measured by magnetic resonance spectrometry (MRS). GABA-AT target engagement levels may be a potential indicator of OV329’s potential therapeutic effect because specified increases in GABA have previously been correlated to seizure reduction.^1,2,3 Ovid expects to enroll more than 60 individuals in this trial and anticipates final results in the first half of 2024.

“We are thrilled to see continued innovation for rare epilepsies, as many of the children and their families in our community do not find current treatments satisfactory,” said Amy E. Brin, CEO of the Child Neurology Foundation. “Families living with rare forms of epilepsy, such as infantile spasms and seizures associated with tuberous sclerosis complex, would benefit from more treatment options, and we look forward to seeing results from these studies.”

To learn more about the OV329 healthy volunteer study, contact Info@OvidRx.com.

About OV329

OV329 is a next-generation GABA-aminotransferase (GABA-AT) inhibitor being developed for the potential treatment of rare and treatment-resistant forms of epilepsy and seizures, such as seizures associated with tuberous sclerosis complex, infantile spasms, and conditions with focal onset seizures. Low levels of GABA, the primary inhibitory neurotransmitter in the brain, have been linked to neuronal hyperexcitability. OV329 is believed to work by reducing the activity of GABA-AT, thereby increasing levels of GABA in the brain, and potentially suppressing neuronal hyperexcitability known to cause seizures. OV329 may be a potential best-in-class GABA-AT inhibitor that could offer enhanced efficacy, an improved safety profile and more optimal dosing.

About Ovid Therapeutics

Ovid Therapeutics Inc. is a New York-based biopharmaceutical company striving to conquer seizures and brain disorders with courageous science. Ovid’s pipeline of small molecule and genetic medicines candidates seek to meaningfully improve the lives of people and families affected by epilepsies. Ovid is developing OV329, a GABA-aminotransferase inhibitor, for treatment-resistant seizures, and OV350, a direct activator of the KCC2 transporter, for potential treatment of epilepsies. In addition, Ovid maintains a significant financial interest in the future regulatory development and potential commercialization of soticlestat, which Takeda is responsible for advancing globally. Soticlestat is a cholesterol 24-hydroxylase inhibitor, which is currently in Phase 3 trials for Dravet and Lennox-Gastaut syndromes. For more information about these and other Ovid research programs, please visit www.ovidrx.com.

Forward-Looking Statements
This press release includes certain disclosures that contain “forward-looking statements,” including, without limitation: statements regarding the potential development and use of OV329; the potential timing of OV329’s Phase 1 Trial, the likelihood that data for OV329 will support future development and therapeutic potential; the potential use and development of OV350 and status of Takeda’s two pivotal Phase 3 trials evaluating soticlestat for Lennox-Gastaut and Dravet syndromes. You can identify forward-looking statements because they contain words such as “anticipates,” “believes,” “expected,” “intends,” “plan,” “potentially,” and “will,” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances). 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 include, without limitation, uncertainties inherent in the preclinical and clinical development and regulatory approval processes, the risk that Ovid may not be able to realize the intended benefits of its technology. Additional risks that could cause actual results to differ materially from those in the forward-looking statements are set forth under the caption “Risk Factors” in Ovid’s Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on November 8, 2022, and in future filings Ovid makes with the SEC. Any forward-looking statements contained in this press release speak only as of the date hereof, and Ovid assumes no obligation to update any forward-looking statements contained herein, whether because of any new information, future events, changed circumstances or otherwise, except as otherwise required by law.

Contacts:

Investors and Media:
Ovid Therapeutics Inc.
Meg Alexander
917-943-6681
malexander@ovidrx.com

OR

Investors:
Argot Partners
Maeve Conneighton
212-596-7231
ovid@argotpartners.com

¹Petroff OA, Rothman DL, Behar KL, Collins TL, Mattson RH. Human brain GABA levels rise rapidly after initiation of vigabatrin therapy. Neurology. 1996 Dec;47(6):1567-71. doi: 10.1212/wnl.47.6.1567. PMID: 8960747.
² Petroff OA, Rothman DL, Behar KL, Mattson RH. Human brain GABA levels rise after initiation of vigabatrin therapy but fail to rise further with increasing dose. Neurology. 1996 May;46(5):1459-63. doi: 10.1212/wnl.46.5.1459. PMID: 8628502.
³Prescot AP, Miller SR, Ingenito G, Huber RS, Kondo DG, Renshaw PF. In Vivo Detection of CPP-115 Target Engagement in Human Brain. Neuropsychopharmacology. 2018 Feb;43(3):646-654. doi: 10.1038/npp.2017.156. Epub 2017 Jul 25. PMID: 28741622; PMCID: PMC5770752.