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Epygenix Therapeutics Announces First Patient Successfully Dosed in Phase 2 Study for the Treatment of Dravet Syndrome With EPX-100

PARAMUS, N.J. - November 12, 2020 - (

​​​Epygenix Therapeutics, Inc., a privately held biopharmaceutical company developing precision medicine for Dravet Syndrome, announced today that the first patient has been successfully dosed in a Phase 2, 20-week Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial of EPX-100 as Adjunctive Therapy in Children with Dravet Syndrome. This study will evaluate the efficacy and safety of EPX-100 in children suffering from Dravet Syndrome. The Company is in the middle of recruiting 24 patients to complete the study. Detailed study and site information is available at and

Dravet Syndrome is a rare, catastrophic, lifelong form of epilepsy which begins in the first year of life with frequent or prolonged seizures. Intellectual disability, behavioral abnormalities, gait and motor dysfunction, and increased mortality are commonly observed as the disease progresses. Patients suffer from life-threatening seizures that cannot be adequately controlled with available medications and face an increased risk of SUDEP (Sudden Unexplained Death in Epilepsy), seizure-related accidents such as drowning, or infections.

Dr. Hahn-Jun Lee, M.Sc., Ph.D., President and CEO of Epygenix Therapeutics, Inc. stated, "We are very excited to announce that first patient has been successfully dosed in our trial. First patient dosing is an extremely important clinical milestone of EPX-100, as this is the crucial first step to evaluate the efficacy and safety of EPX-100 in patients. We have made great efforts so far and will continuously work to obtain meaningful clinical outcomes for Dravet patients."

Dr. Scott C. Baraban, Ph.D., Professor & William K. Bowes Jr. Endowed Chair in Neuroscience Research at UCSF and Chair of the Scientific Advisory Board at Epygenix Therapeutics, Inc. issued a joint comment that "Beginning the process of evaluating EPX-100 in Dravet Syndrome patients is the culmination of a very exciting journey from the 'aquarium' to the 'bedside'. We are optimistic that Epygenix drugs will offer a safe and effective treatment option for these children."

Alex Yang, J.D., LLM, President and CEO of Mstone Partners Hong Kong and Chair of the Board at Epygenix Therapeutics also stated that "Given our robust science background and superb safety profile, we are cautiously optimistic that EPX-100 will yield excellent results in this trial and take another step towards the best-in-class drug for refractory epilepsies."   

About Epygenix Therapeutics, Inc.


Epygenix Therapeutics, Inc. is a precision medicine-based biopharmaceutical company focused on discovering and developing drugs to treat rare and intractable genetic epilepsy in childhood, such as Dravet Syndrome. Epygenix is currently focused on developing EPX-100, -200, and -300. These candidates abolish convulsive behavior and electrographic seizure activity and were discovered in a zebrafish Dravet Syndrome model which mimics the human pathology and confirmed its validity by the human efficacy with EPX-200 and -300. For more information, please visit


 About EPX-100


EPX-100 is a first-generation antihistamine and found to be a powerful suppressor of spontaneous convulsive behavior and electrographic seizures in zebrafish models for Dravet Syndrome. Its antiepileptic action, however, is not through a histaminergic mechanism of action, but via modulation of serotonin (5HT) signaling pathways. Its safety has been confirmed with the completed Phase I, Placebo-Controlled, Double-Blind, 2-Period Study to assess safety and pharmacokinetics of escalating single and multiple oral doses of EPX-100 in fasting healthy subjects and following a high-fat meal. 


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Hahn-Jun Lee, M.Sc., Ph.D.

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Original Source: Epygenix Therapeutics Announces First Patient Successfully Dosed in Phase 2 Study for the Treatment of Dravet Syndrome With EPX-100

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Last Updated: 07-Jan-2021