Phase 1 Studies Begin for EPX-100 for Dravet’s Syndrome

  • Dravet syndrome
  • Epilepsy
  • Seizures

The Food and Drug Administration has approved the Investigational New Drug Application for EPX-100 (Epygenix Therapeutics, Inc., Paramus, NJ), allowing a phase 1 study to being. This phase 1 placebo-controlled, 2-period study of EPX-100 will test safety and pharmacokinetics of escalating single and multiple doses of in healthy individuals. 

A first-generation antihistamine, EPX-100 was safely used from 1959 to 1976 to treat itch. More recently, the agent was found to be a powerful suppressor of spontaneous convulsive behavior in zebrafish models for Dravet’s syndrome. Antiepileptic action of EPX-100, however, is not through a histaminergic mechanism, but through modulation of 5HT signaling pathways. 

If the phase 1 trial is successful, phase 2 trials in people with Dravet’s syndrome will begin. 

“With this IND approval for EPX-100 from the FDA, we are now able to validate our unique zebrafish-to-human approach,” said Scott C. Baraban, PhD, professor and William K. Bowles, Jr. Endowed Chair in Neuroscience Research at the University of California, San Francisco; and chair of the scientific advisory board at Epygenix Therapeutics. “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.”

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