Epygenix Therapeutics Submits Investigational New Drug Application for EPX-100 to the U.S. FDA for the Treatment of Patients With Dravet Syndrome
PARAMUS, N.J., June 24, 2019 - Epygenix Therapeutics, Inc., a privately held biopharmaceutical company developing precision medicines for Dravet Syndrome, announced today that the Company has submitted Investigational New Drug Application (IND) and Fast Track Designation Request for EPX-100 to the U.S. Food and Drug Administration (FDA) for the treatment of patients with Dravet syndrome. Epygenix anticipates that Phase 1 will be initiated in August 2019.
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 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.
"This IND filing for EPX-100 is one of the company's significant development milestones. We will test its safety and efficacy in a prompt manner with an eye toward helping many patients who suffer from this disorder," 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., the lead Regulatory and Clinical CRO for the EPX-100 development program states that: "We are very pleased to collaborate with Epygenix to support the IND filing for EPX-100, as well as to manage the Phase 1 study in normal volunteers."
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 "the IND filing for EPX-100 is an exciting leap to the clinical stage, and a significant milestone in validating our unique zebrafish-to-human approach to precision medicine."
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 1950 and 1970. 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.