Actio Biosciences, a clinical-stage biotechnology company leveraging a novel approach to genetics and precision medicine to develop new small molecule therapeutics that target shared underlying biology in both rare and common diseases,  announced that the first participant has been dosed in the Phase 1a healthy volunteer clinical trial of ABS-1230 for the treatment of KCNT1-related epilepsy. KCNT1-related epilepsy is a rare, severe and often fatal pediatric developmental epileptic encephalopathy with a U.S. prevalence of approximately 2,500 individuals.

“Advancing ABS-1230 into the clinic marks a historic milestone for Actio and the patients living with KCNT1-related epilepsy, a debilitating and severe epilepsy with no disease-modifying therapies available today. Designed to address the root cause of this devastating disease, ABS-1230 has the potential to offer patients an option that could meaningfully reduce seizures and improve daily quality of life,” said David Goldstein, Ph.D., CEO of Actio. “This marks our second clinical study initiation this year, underscoring Actio’s commitment to sustained momentum in the pursuit of transformational therapies for patients with rare genetic diseases.”

The Phase 1a trial is a randomized, double-blind, placebo-controlled, single- and multiple-ascending dose and food effect study in healthy volunteers that will evaluate safety, tolerability, and pharmacokinetics of ABS-1230. The study is being conducted in Australia. Actio plans to expand into a proof-of-concept Phase 1b/2a study in KCNT1-related epilepsy patients in the U.S. in early 2026.