Actio Biosciences Announces U.S. FDA Clearance of Investigational New Drug Application and Fast Track Designation for ABS-1230, a KCNT1 Inhibitor for the Treatment of KCNT1-Related Epilepsy

SAN DIEGO--(BUSINESS WIRE)--Actio Biosciences, a clinical-stage biotechnology company leveraging a novel approach to genetics and precision medicine to develop new therapeutics that target shared underlying biology in both rare and common diseases, today announced that the United States (U.S.) Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application and granted Fast Track designation for ABS-1230, an expected first-in-class, orally administered small molecule KCNT1 inhibitor, 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. Additionally, ABS-1230 received conditional approval from the Human Research Ethics Committee (HREC) in Australia for human clinical evaluation.

“Regulatory approval to initiate clinical evaluation of ABS-1230 is a significant moment for Actio and the children who are living with this devastating form of epilepsy that can lead to upwards of 20 seizures a day,” said David Goldstein, Ph.D., co-founder and CEO of Actio. “With no disease-modifying therapies available today, we designed ABS-1230 to address the root cause of disease, having demonstrated ABS-1230’s ability to inhibit all recurrently observed pathogenic mutations in the KCNT1 gene and rapidly suppress seizures in preclinical models. We look forward to initiating our Phase 1 trial in healthy volunteers soon and further determining the potential therapeutic potential of ABS-1230 for patients with KCNT1-related epilepsy. The Fast Track designation underscores the serious unmet need. We look forward to working closely with regulatory agencies to advance this program as rapidly as possible.”

“As a father to an 8-year-old with KCNT1 epilepsy and president of the KCNT1 Epilepsy Foundation, I know firsthand how devastating this disease is for the children who suffer daily seizures, and for the families who care for them around the clock,” said Justin West, M.D., co-founder of the KCNT1 Epilepsy Foundation. “Most of our children can’t walk, talk or feed themselves. But today, we get to celebrate. 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. We’re profoundly grateful to the team at Actio for their tireless work, their compassion and their belief in this fight. 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.”

Actio plans to initiate the healthy volunteer portion of a Phase 1 clinical trial of ABS-1230 in fall of 2025 with plans to expand into a proof-of-concept Phase 1b study in KCNT1-related epilepsy patients in early 2026. ABS-1230 was recently granted both rare pediatric and orphan drug designations from the U.S. FDA.

Fast Track is a process designed by the FDA to facilitate the development and expedite the review of investigational drugs intended to treat serious conditions and for which nonclinical or clinical data demonstrate the potential to address unmet medical need. A therapeutic candidate that receives Fast Track designation may be eligible for more frequent interactions with the FDA to discuss the candidate’s development plan. Therapeutic candidates with Fast Track designation may also be eligible for priority review and accelerated approval if supported by clinical data.

About ABS-1230

ABS-1230 is an expected first-in-class orally administered, selective small molecule KCNT1 inhibitor for the treatment of KCNT1-related epilepsy. In preclinical studies, ABS-1230 has been shown to inhibit all tested pathogenic mutations in the KCNT1 gene, indicating suitability to treat all patients with KCNT1-related epilepsy. ABS-1230 has the potential to offer patients the convenience of a once-daily pill or solution, in contrast to therapies that require visits to clinical sites for administration. Actio plans to initiate the healthy volunteer portion of a Phase 1 clinical trial of ABS-1230 in fall of 2025 with plans to expand into a proof-of-concept Phase 1b study in KCNT1-related epilepsy patients in early 2026.

About KCNT1-related Epilepsy

KCNT1-related epilepsy is a rare and often fatal pediatric epileptic encephalopathy. Patients with KCNT1-related epilepsy experience frequent treatment-resistant seizures that typically begin in early infancy and are accompanied by profound developmental delays and neurological impairments. General antiepileptic drugs have limited benefit in patients with this genetic epilepsy, with side effects that can be debilitating, and the risk of increasing refractoriness over time. It is estimated that approximately 2,500 individuals in the U.S. have KCNT1-related epilepsy.

About Actio Biosciences

Actio Biosciences is a clinical-stage company leveraging advances in precision medicine to develop new therapeutics that target shared genetics in rare and common diseases—bringing meaningful medicines from one to many. Applying its expertise in genetics, drug discovery and data sciences, Actio seeks to identify programs where both biological and technical risk can be minimized to streamline the drug development process and bring forward exceptionally potent and precisely targeted small molecule therapeutics. Actio is advancing two precision medicines – ABS-0871 and ABS-1230 – with first-in-class potential for the treatment of Charcot-Marie-Tooth disease, type 2C (CMT2C) and KCNT1-related epilepsy, respectively. Founded in October 2021, the San Diego-based company is guided by leaders in genetics and drug development and backed by top healthcare investors. For more information, please visit ActioBiosciences.com and follow the company on LinkedIn and X.