Ovid Therapeutics Expands Epilepsy Franchise With New KCC2 Activators

  • AstraZeneca Exclusive License Adds Unique Candidate and Large Compound Library to Ovid’s Franchise of Potential First-Class Antiepileptic Therapies
  • Collaboration with Dr Stephen Moss, founder of the Tufts Laboratory for Basic and Translational Neuroscience Research, and team of experts in neuropharmacology
  • Transaction is the first business development activity that aims to enhance Ovid’s CNS small molecule and genetic drug portfolio

NEW YORK, Jan. 03, 2022 (GLOBE NEWSWIRE) – Ovid Therapeutics (NASDAQ: OVID), a biopharmaceutical company committed to the development of drugs that transform the lives of people with rare neurological diseases, today announced the signing of an exclusive license agreement with AstraZeneca for an early stage small molecule library targeting the KCC2 transporter, including the lead candidate, OV350. The company seeks to optimize and accelerate the development of these activators of the KCC2 transporter in epilepsies and potentially other neuropathic conditions.

“The KCC2 transporter is an exciting and new target that we believe holds great promise in the treatment of epilepsies,” said Jeremy Levin, D.Phil, MB BChir, CEO of Ovid. “The compounds are a perfect fit for our franchise of small molecule epilepsy drugs, and they follow our history of successful partnerships with large pharmaceutical companies. “

Under the terms of the deal, AstraZeneca will receive an upfront payment of $ 5 million in cash and $ 7.5 million in Ovid common stock. AstraZeneca is eligible to receive potential clinical development milestones of up to $ 8 million and regulatory milestones of up to $ 45 million. Total commercial milestones could reach $ 150 million and installment royalty payments range from 10% on net sales. At the time of proof of clinical efficacy, AstraZeneca will have the right of first negotiation to opt for a strategic collaboration.

“Ovid’s focus in neuroscience and his experience in the development of novel antiepileptics make him an optimal choice for advancing KCC2 activators, including OV350,” noted Iain Chessell, Global Head of Neuroscience, BioPharmaceuticals R&D, AstraZeneca. “This transaction continues to align development resources with our stated areas of strategic interest. “

OV350 is an early stage compound that has shown encouraging in vitro and in vivo proof of concept in resistant forms of epilepsy. The compound is designed to directly target and activate KCC2, a potassium chloride co-transporter responsible for maintaining chloride homeostasis in neurons. By enhancing chloride homeostasis, OV350 is believed to inhibit neuronal hyperexcitability commonly associated with epilepsies. Research has shown that the presence of mutations and dysfunctions in KCC2 may contribute to the neuronal hyperexcitability commonly seen in epilepsies.1,2

The program was advanced through a collaboration between AstraZeneca and the Tufts Laboratory for Basic and Translational Neuroscience Research, which included Drs. Stephen Moss and Jamie Maguire. Ovid plans to continue a strategic collaboration with Drs. Moss and Maguire, who are leading authorities in GABA receptor research and neuropharmacology. Their associate, Dr Aaron Goldman of Harvard Medical School, will also collaborate with Ovid and translate his expertise in epilepsy drug resistance and the company’s targeted neurotherapy candidates.

“Despite therapeutic advances in recent decades, around one-third to half of people treated for epilepsy continue to have seizures.3,4,5 Therapies that activate KCC2, such as OV350, could become a powerful weapon for clinicians seeking to treat a potential underlying cause of epilepsies, ”according to Dr. Moss.

OV350 extends Ovid’s franchise of new anti-epileptic drugs. The Company is also developing OV329, a next-generation pregabalin for tuberous sclerosis and infantile spasms, which is expected to enter clinical trials in 2022. Earlier this year, Ovid cleared soticlestat, a new cholesterol 24-hydroxylase inhibitor, to Takeda . Soticlestat is currently being investigated in phase 3 trials for Dravet and Lennox Gastaut syndromes.

About the OV350
OV350 is a small molecule that directly activates the KCC2 transporter, which is important for seizure control. In vivo studies have shown that KCC2 activity results in reduced seizure susceptibility and seizure-induced mortality. Preclinical mechanistic studies also demonstrated that OV350 was well tolerated and did not induce sedation. OV350 has the potential to be developed for multiple epilepsies and other CNS indications, including neurodevelopmental and neurodegenerative diseases.

About Ovid Therapeutics
Ovid Therapeutics Inc. is a New York-based biopharmaceutical company that uses its BoldMedicine® approach to develop drugs that transform the lives of patients with neurological disorders. Ovid seeks to combine deep CNS experience with emerging advances in genetics and brain pathways to create a leading next-generation neuroscience pipeline. Ovid’s current pipeline programs include: OV329, a small molecule GABA aminotransferase inhibitor for seizures associated with tuberous sclerosis and infantile spasms; OV882, a short hairpin RNA therapy approach for Angelman syndrome; OV815, a gene therapy approach for neurological disorders associated with KIF1A; and other research targets. Additionally, Ovid retains a significant financial interest in the future regulatory development and potential commercialization of soticlestat, which Takeda is responsible for advancing globally. Two phase 3 trials for soticlestat in Dravet syndrome and Lennox-Gastaut syndrome are actively recruiting patients. For more information about Ovid, 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 development and acceleration of Ovid’s product candidate pipeline, Ovid’s strategic approach and business development intentions and opportunities and the ability to derive the desired benefits, Ovid’s ability to identify acquisition targets, potential therapeutic benefits of Ovid’s current or future product candidates, clinical and regulatory development and the potential commercialization of soticlestat, OV329, OV350 or any of Ovid’s current or future product candidates, and Ovid’s eligibility for potential milestone and royalty payments. You can identify forward-looking statements because they contain words such as “will”, “appears”, “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 uncertainties, risks and inherent changes in circumstances which 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 forward-looking statements include, but are not limited to, uncertainties inherent in the preclinical and clinical development and regulatory approval processes, risks related to the ability to ” Ovid to achieve its financial goals, the risk that Ovid may not be able to realize the expected benefits of its technology, the risks associated with Ovid’s ability to identify acquisition targets or strategic partners, to conclude strategic transactions on favorable terms, or to consume and realize the benefits of any strategic transaction or acquisition and the risks to Ovid or Takeda’s ability to meet the expected timelines and milestones presented by the COVID-19 pandemic In progress. Additional risks that could cause actual results to differ materially from those of forward-looking statements are set out under “Risk Factors” in Ovid’s Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission ( SEC) on November 10. , 2021, and in future filings that Ovid will make with the SEC. All 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 as a result of new information, future events, changes in circumstances or otherwise, unless otherwise required by law.


1 Ellender, TJ, Raimondo, JV, Irkle, A., Lamsa, KP and Akerman, CJ (2014). the excitatory effects of parvalbumin-expressing interneurons maintain epileptiform activity of the hippocampus via synchronous discharges afterwards. J. Neurosks. 34, 15208-15222. doi: 10.1523 / JNEUROSCI.1747-14.2014
2 Magloire, V., Cornford, J., Lieb, A., Kullmann, DM and Pavlov, I. (2019). Overexpression of KCC2 prevents the paradoxical action of promoting seizures of somatic inhibition. Nat. Commmon. 10: 1225. doi: 10.1038 / s41467-019-08933-4
3 Kwan, P., and Brodie, MJ (2000). Early identification of refractory epilepsy. N. Engl. J. Med. 342, 314-319. doi: 10.1056 / NEJM200002033420503
4Shorvon, S., and Luciano, AL (2007). Prognosis of chronic and newly diagnosed epilepsy: revisiting the temporal aspects. Course. Opinion. Neurol. 20, 208-212. doi: 10.1097 / wco.0b013e3280555175
5 Cascino, GD (2008). When drugs and surgery don’t work. Epilepsy 49, 79-84. doi: 10.1111 / j.1528-1167.2008.01930.x


Investors and media:
Ovid Therapeutics Inc.
Meg Alexander
[email protected]


Argot Partners
Dawn Schottlandt
[email protected]

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