Epygenix Submits IND Application for EPX-100 for Dravet Syndrome

Epygenix Therapeutics announced 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," said Dr. Hahn-Jun Lee, M.Sc., Ph.D., president and CEO of Epygenix Therapeutics, Inc.

"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. Jules Mitchel, Ph.D., President at Target Health Inc., the lead Regulatory and Clinical CRO for the EPX-100 development program said.

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.

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