Actio Biosciences Announces First Participant Dosed in Phase 1 Clinical Trial of ABS-1230, a KCNT1 Inhibitor for the Treatment of KCNT1-Related Epilepsy

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SAN DIEGO--(BUSINESS WIRE)--Sep 23, 2025--

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, today 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.

About ABS-1230

ABS-1230 is an anticipated first-in-class orally administered, selective small molecule KCNT1 inhibitor for the treatment of KCNT1-related epilepsy. In preclinical studies, ABS-1230 inhibited all tested pathogenic mutations in the KCNT1 gene, indicating suitability to treat all patients with KCNT1-related epilepsy. ABS-1230 has the potential to provide patients with the convenience of an at-home, orally administered therapy. The initial Phase 1a study is being conducted under a U.S. FDA Investigational New Drug (IND) application with Ethics Committee approval to conduct the study in Australia. ABS-1230 has U.S. FDA Fast Track, Rare Pediatric Disease (RPD) and Orphan Drug (ODD) designations.

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 identifies programs where both biological and technical risk can be minimized to streamline drug development and bring forward exceptionally potent and precisely targeted small molecule therapeutics. Actio is advancing two precision drug candidates – 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, and with potential expansion in overactive bladder and other genetic epilepsies. 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.

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KEYWORD: UNITED STATES NORTH AMERICA CALIFORNIA

INDUSTRY KEYWORD: HEALTH NEUROLOGY GENETICS GENERAL HEALTH CLINICAL TRIALS PHARMACEUTICAL BIOTECHNOLOGY

SOURCE: Actio Biosciences

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PUB: 09/23/2025 09:00 AM/DISC: 09/23/2025 08:59 AM

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