SAN DIEGO– Actio Biosciences, a clinical-stage biotechnology company focused on genetics and precision medicine, announced Tuesday that the first participant has been dosed in its Phase 1a clinical trial of ABS-1230, an investigational KCNT1 inhibitor being developed to treat KCNT1-related epilepsy.
KCNT1-related epilepsy is a rare and often fatal pediatric developmental epileptic encephalopathy affecting an estimated 2,500 individuals in the United States. No disease-modifying therapies are currently available.
“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,” said David Goldstein, Ph.D., CEO of Actio. “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. 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 study being conducted in Australia. It will evaluate the safety, tolerability, and pharmacokinetics of single and multiple ascending doses of ABS-1230, as well as food effects.
Actio said it plans to expand the program into a Phase 1b/2a proof-of-concept study in U.S. patients with KCNT1-related epilepsy in early 2026.
