Actio Biosciences announced that the U.S. Food and Drug Administration has cleared the investigational new drug application and granted Fast Track designation for ABS-1230, an expected first-in-class, orally administered small molecule KCNT1 inhibitor for treating KCNT1-related epilepsy. The rare, severe and often fatal pediatric developmental epileptic encephalopathy affects approximately 2,500 individuals in the United States.
The San Diego-based clinical-stage biotechnology company also received conditional approval from the Human Research Ethics Committee in Australia for human clinical evaluation of ABS-1230, which recently obtained both rare pediatric and orphan drug designations from the FDA.
Addressing Critical Unmet Medical Need
KCNT1-related epilepsy represents a devastating condition where patients experience frequent treatment-resistant seizures beginning in early infancy, accompanied by profound developmental delays and neurological impairments. Children with this condition can experience upwards of 20 seizures per day, with most unable to walk, talk, or feed themselves.
"With no disease-modifying therapies available today, we designed ABS-1230 to address the root cause of disease," said David Goldstein, Ph.D., co-founder and CEO of Actio. The company demonstrated ABS-1230's ability to inhibit all recurrently observed pathogenic mutations in the KCNT1 gene and rapidly suppress seizures in preclinical models.
Current general antiepileptic drugs provide limited benefit for patients with this genetic epilepsy, often causing debilitating side effects and increasing treatment resistance over time.
Clinical Development Timeline
Actio plans to initiate the healthy volunteer portion of a Phase 1 clinical trial of ABS-1230 in fall 2025, with expansion into a proof-of-concept Phase 1b study in KCNT1-related epilepsy patients planned for early 2026. The oral formulation offers potential convenience as a once-daily pill or solution, contrasting with therapies requiring clinical site visits for administration.
The Fast Track designation facilitates development and expedites review of investigational drugs for serious conditions with demonstrated potential to address unmet medical needs. This designation enables more frequent FDA interactions regarding development plans and potential eligibility for priority review and accelerated approval if supported by clinical data.
Community Impact
Justin West, M.D., co-founder of the KCNT1 Epilepsy Foundation and father of an 8-year-old with KCNT1 epilepsy, emphasized the significance of this milestone. "The FDA's approval of Actio's Phase 1a trial for ABS-1230 marks a historic milestone—not just for science, but for our entire community," West stated. "This trial represents more than hope. It is the beginning of a future we've spent years working toward—one where treatments exist, where lives are changed and where families like mine can finally breathe."
About the Therapeutic Candidate
ABS-1230 functions as a selective small molecule KCNT1 inhibitor designed to target the underlying genetic cause of KCNT1-related epilepsy. In preclinical studies, the compound demonstrated inhibition of all tested pathogenic mutations in the KCNT1 gene, indicating potential suitability for treating all patients with KCNT1-related epilepsy regardless of their specific mutation.
Actio Biosciences leverages advances in precision medicine to develop therapeutics targeting shared genetics in rare and common diseases. The company applies expertise in genetics, drug discovery, and data sciences to identify programs where biological and technical risks can be minimized, streamlining drug development for precisely targeted small molecule therapeutics.
