Potential Treatment for Dravet Syndrome Receives FDA IND Clearance for Further Study

The Food and Drug Administration (FDA) and the Clinical Trial Approval (CTA) scheme from the Australian Therapeutic Goods Administration have cleared the investigational new drug (IND) application for ETX101 (Encoded Therapeutics, South San Francisco, CA), an AAV9-mediated candidate gene regulation therapy to be studied for the treatment of infants and children with SCN1A+ Dravet syndrome. ETX101 has been engineered to restore the function of inhibitory GABAergic interneurons in those with Dravet syndrome by upregulating the expression of Nav1.1 sodium channels.

The IND clearance enables Encoded Therapeutics to initiate ENDEAVOR (NCT05419492), a phase 1/2 clinical trial in the United States, and WAYFINDER, a phase 1/2 clinical trial in Australia. These trials are part of POLARIS, Encoded Therapeutics global clinical development program which aims to assess the safety and efficacy of ascending doses of ETX101 in infants and young children, including the initial effects of ETX101 on seizure burden and the potential long-term improvements in neurodevelopment.

“Dravet syndrome is a devastating disorder that necessitates a paradigm shift from conventional symptomatic management towards precise and targeted interventions at the genetic level. I’m hopeful that gene therapy will not just alleviate symptoms but address the root cause of Dravet syndrome, marking a potentially transformative approach in our quest to address the ongoing medical burden of families living with Dravet syndrome,” said Joseph Sullivan, MD, Principal Investigator on the ENDEAVOR study.