Dravet Syndrome, a catastrophic form of epilepsy, affects one in 15,700 children and adults in the United States.
Epygenix Therapeutics, Inc., a privately held biopharmaceutical company developing precision medicines for Dravet Syndrome, announced today that the Company has received an Investigational New Drug Application (IND) approval from the U.S. Food and Drug Administration (FDA) for the treatment of patients with Dravet syndrome. Epygenix will start a Phase I, placebo-controlled, double-blind, 2-period study to assess the safety and pharmacokinetics of escalating single (and multiple) oral doses of EPX-100 in fasting healthy subjects following a high-fat meal. Immediately following Phase 1 studies, Epygenix will initiate Phase 2 studies with Dravet Syndrome patients.
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. In most cases, the disease is caused by heterozygous de novo mutation or deletion of SCN1A, a gene encoding a brain voltage-gated sodium channel (Nav1.1). In a phenotype-based preclinical screen of over 3500 compounds, EPX-100 was identified as a drug that abolished spontaneous seizures and convulsive behavior in a zebrafish model for Dravet Syndrome.
“We are very excited with EPX-100 IND approval from the FDA, and this allows us to test EPX-100 in humans, and eventually Dravet patients. Our team will rigorously monitor all safety and efficacy tests for EPX-100 as we advance this drug to the clinic,” says Dr. Hahn-Jun Lee, M.Sc., Ph.D., President and CEO of Epygenix Therapeutics, Inc.
Dr. Jules Mitchel, Ph.D., President at Target Health, Inc., lead CRO for Phase 1 EPX-100 trials commented that: “This is an exciting moment for us to move EPX-100 from preclinical trials into human testing. We are looking forward to the continuation of our collaboration with Epygenix to support successful completion of EPX-100 Phase 1 studies.”
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 comment that, “With this IND approval for EPX-100 from the FDA, we are now able to validate our unique zebrafish-to-human approach. This is a very big step toward our overall goal of using zebrafish in a precision medicine pipeline for the development of new therapies for Dravet syndrome and related genetic 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, EPX-200, and EPX-300. These drug candidates abolish convulsive behavior and electrographic seizure activity and were discovered in a zebrafish Dravet Syndrome model which mimics the human pathology. For more information, please visit www.epygenix.com.
EPX-100 is a first-generation antihistamine which was safely used to treat itch between 1959 and 1976. EPX-100 was found to be a powerful suppressor of spontaneous convulsive behavior and electrographic seizures in zebrafish models for Dravet Syndrome. EPX-100 antiepileptic action, however, is not through a histaminergic mechanism of action, but via modulation of serotonin (5HT) signaling pathways.
Hahn-Jun Lee, M.Sc., Ph.D.
SOURCE Epygenix Therapeutics, Inc.