Epygenix Therapeutics has submitted an investigational new drug application (IND) to the U.S. Food and Drug Administration (FDA) to assess the safety and efficacy of EPX-100, its investigational therapy for Dravet syndrome.
If accepted, Epygenix expects to initiate a Phase 1 trial with healthy volunteers by August. The company has also requested fast track designation for EPX-100.
A new drug application is an essential first step in launching a clinical trial; a fast track designation allows for an accelerated FDA review process for treatments that could alleviate serious conditions and fill an unmet medical need.
“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,” Hahn-Jun Lee, M.Sc., PhD, president and CEO of Epygenix Therapeutics, said in a press release.
“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,” said Jules Mitchel, PhD, president at Target Health, the lead regulatory and clinical research organization for the EPX-100 development program.
EPX-100 is a repurposed antihistamine, originally known as clemizole, used to treat itching in the 1950s and 1960s. EPX-100 has shown potential to suppress seizures by modulating serotonin, a mechanism that differs from its antihistaminic properties.
Serotonin is a chemical messenger present in many parts of the brain. Scientists think that people with Dravet syndrome may have alterations in the serotonin signaling pathway, but exact problems are not known. Likewise, precisely how EPX-100 affects serotonin systems in the brain to improve seizures is still unclear.
The therapy’s potential to halt seizures was discovered in a study that screened over 3,000 approved medications in zebrafish models of Dravet’s syndrome. The study, “Drug screening in Scn1a zebrafish mutant identifies clemizole as a potential Dravet syndrome treatment,” was published in the journal Nature Communications in 2013.
“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,” said Scott C. Baraban, PhD, professor at UC San Francisco and chair of the scientific advisory board at Epygenix Therapeutics.