First scientific presentation of OV-935 (TAK-935) safety and tolerability data in developmental and epileptic encephalopathies from the Phase 1b/2a clinical trial
Study met primary safety and tolerability endpoints and showed initial evidence of potential efficacy in seizure reduction
Data encouraging for plasma 24HC as a potential biomarker for assessing treatment outcome and disease management
NEW YORK, May 23, 2019 /PRNewswire/ -- Ovid Therapeutics Inc. , a biopharmaceutical company committed to developing medicines that transform the lives of people with rare neurological diseases, will present data on safety, tolerability, exploratory and secondary outcomes from the Phase 1b/2a clinical trial of OV935/TAK-935 in adult patients with developmental and epileptic encephalopathies (DEE). The data will be presented as a poster at the 12(th) International Epilepsy Colloquium (IEC), taking place May 26-28 in Lyon, France.
"DEE refers to a group of rare conditions characterized by cognitive impairment and severe epilepsy.(1) Each of these conditions can worsen over time and cause significant morbidities for patients and their families so there is an urgent need for new medicines in this area," said Amit Rakhit, MD, MBA, Chief Medical Officer and Head of Research & Development at Ovid. "Together with Takeda, we believe that OV935 may have the potential to treat rare epilepsies and are committed to making a difference for patients living with these diseases."
The randomized, double blind, placebo-controlled Phase 1b/2a clinical trial achieved its primary endpoint of safety and tolerability and showed OV935 was generally well tolerated. During the open-label period, 11 patients (68.8%) experienced a treatment-emergent adverse event (TEAE), of which the majority were mild. There were five serious TEAEs, all seizure cluster related. Overall, the data are consistent with a favorable safety and tolerability profile and support the continued clinical development of OV935.
Ovid is committed to grounding innovative research by first understanding biological pathways and their central role in rare neurological disorders in order to develop clinically-relevant endpoints that measure the efficacy of investigational treatments and deliver tangible patient benefits. Exploratory endpoints in the Phase 1b/2a trial included changes in seizure frequency and plasma 24S-hydroxycholesterol (24HC) levels. OV935 showed initial evidence of potential efficacy with a reduction in seizure frequency and a reduction in mean 24HC levels. This preliminary evidence suggests that plasma 24HC may be a promising biomarker for assessing treatment outcome and disease management.
Details of the presentation are listed below.
Title: A phase 1b/2a study of TAK-935 (OV935) as adjunctive therapy in patients with developmental and epileptic encephalopathies (DEE) Poster Number: 45 Presentation Date and Time: Sunday, May 26, 2019 at 1:30 p.m. - 3:30 p.m. CEST
Previously announced by Ovid in its December 17, 2018 press release, these results were also presented as an invited speakership at the 15(th) Antiepileptic Drug and Device Trials Conference, May 22-24, Miami, Fl. The IEC presentation and the invited speakership demonstrate a burgeoning interest in rare epilepsies and the hope that OV935 might be a potential treatment option.
About Developmental and Epileptic Encephalopathies The term developmental and epileptic encephalopathy includes a group of epilepsy syndromes associated with severe cognitive and behavioral disturbances. The International League Against Epilepsy (ILAE) defines an epileptic encephalopathy as a condition in which "the epileptiform EEG abnormalities themselves are believed to contribute to a progressive disturbance in cerebral function."
These epilepsies cause significant morbidities for patients beyond what might be expected from the known underlying pathology alone and can worsen over time. Developmental and epileptic encephalopathies typically present early in life and are often associated with severe cognitive and developmental impairment in addition to frequent treatment-resistant seizures throughout the person's lifetime. These disorders vary in age of onset, developmental outcomes, etiologies, neuropsychological deficits, electroencephalographic (EEG) patterns, seizure types, and prognosis.
Despite the availability of medicines for epilepsy, few treatment options are available for epileptic encephalopathies, and novel therapies are needed.
About Investigational OV935/TAK-935 OV935/TAK-935 is a potent, highly-selective, first-in-class inhibitor of the enzyme cholesterol 24-hydroxylase (CH24H) being investigated as an anti-epileptic drug (AED). CH24H is predominantly expressed in the brain, where it plays a central role in cholesterol homeostasis. CH24H converts cholesterol to 24-hydroxycholesterol (24HC), which then exits the brain into the peripheral circulation. Glutamate is one of the main neurotransmitters in the brain and has been shown to play a role in the initiation and spread of seizure activity. Recent literature indicates CH24H is involved in over-activation of the glutamatergic pathway through modulation of the NMDA channel, implying its potential role in central nervous system diseases such as epilepsy. In addition, preclinical data suggest OV935 has an anti-inflammatory and glial modulatory function with resultant anti-epileptogenic not simply a direct seizure suppressive activity. Ovid and Takeda believe that the novel mechanism of action of OV935 may potentially treat rare epilepsies by modifying disease pathology over time and might ultimately be considered an early treatment option in view of this mechanism. To Ovid and Takeda's knowledge, OV935 is the only molecule with this mechanism of action in clinical development. OV935 is an investigational drug, not approved for commercial use.
The United States Food and Drug Administration (FDA) has granted orphan drug designation to OV935 for the treatment of both Dravet syndrome and LGS.
About the Ovid/Takeda Collaboration Ovid and Takeda entered into a global development and commercialization collaboration in January 2017 to evaluate OV935/TAK-935 across a range of rare epilepsy syndromes. Under the terms of the agreement, the companies share in the development and commercialization costs on a 50/50 basis and, if successful, the companies will share in the profits on a 50/50 basis. Takeda will lead commercialization in Japan and has the option to lead in Asia and other selected geographies. Ovid leads clinical development activities and commercialization in the United States, Europe, Canada and Israel.
About Ovid Therapeutics Ovid Therapeutics is a New York-based biopharmaceutical company using its BoldMedicine(TM) 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).
(1)Source: I.E. Scheffer, S. Berkovic, G. Capovilla, et al. ILAE classification of the epilepsies: Position paper of the ILAE Commission for Classification and Terminology. Epilepsia; 58 (2017): 512-521. https://onlinelibrary.wiley.com/doi/full/10.1111/epi.13709 [https://c212.net/c/link/?t=0&l=en&o=2477015-1&h=2756295029&u=https%3A%2F%2Fonlinelibrary.wiley.com%2Fdoi%2Ffull%2F10.1111%2Fepi.13709&a=https%3A%2F%2Fonlinelibrary.wiley.com%2Fdoi%2Ffull%2F10.1111%2Fepi.13709]
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