Actio Biosciences Targets Rare Pediatric Epilepsy

Actio Biosciences has initiated the KYRON Phase Ib/II clinical trial for ABS-1230, a targeted therapy for KCNT1-related epilepsy. This rare and severe pediatric developmental epileptic encephalopathy is characterized by treatment-resistant seizures and developmental delays from infancy.

ABS-1230, an oral selective KCNT1 inhibitor, aims to address the genetic mechanism of the disease. Previous Phase Ia trials in healthy volunteers demonstrated that all tested doses of ABS-1230 were well-tolerated without serious adverse events.

The KYRON study is evaluating the pharmacokinetics, safety, and tolerability of ABS-1230 in individuals aged one month to 21 years. It includes an open-label phase, a placebo-controlled arm, and a long-term extension, with younger age groups to be included following safety evaluations.

The US Food and Drug Administration (FDA) has accepted ABS-1230 into its rare disease evidence principles (RDEP) process, a framework designed to expedite the development of therapies for ultra-rare, genetically defined diseases. This marks a significant step towards providing urgently needed treatment options for affected children and their families.