Actio Biosciences to Present New Preclinical Data for ABS-1230, a KCNT1 Inhibitor for the Treatment of KCNT1-Related Epilepsy, at the American Epilepsy Society Annual Meeting

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SAN DIEGO--(BUSINESS WIRE)--Nov 24, 2025--

Actio Biosciences, a clinical-stage biotechnology company advancing the translation of genetic insights into novel precision medicines, today announced it will present new preclinical data from its ABS-1230 program for the treatment of KCNT1-related epilepsy in a platform presentation and poster session at the American Epilepsy Society (AES) Annual Meeting, being held December 5-9 in Atlanta. KCNT1-related epilepsy is a rare and often fatal pediatric developmental epileptic encephalopathy with a U.S. prevalence of approximately 2,500 individuals.

“We’re excited to share new preclinical data on ABS-1230, our potent and selective orally available small-molecule inhibitor of KCNT1, now being evaluated in a Phase 1a trial in healthy adult volunteers and soon to be evaluated in a Phase 1b/2a trial in participants with KCNT1-related epilepsy,” said David Goldstein, Ph.D., CEO of Actio Biosciences. “By directly addressing the pathogenic KCNT1 hyperactivity that drives this epilepsy, ABS-1230 has the potential to deliver a meaningful benefit for patients and families. We look forward to engaging with the scientific community at AES as we work toward bringing this potentially transformative therapy to patients.”

Details of the platform and poster presentations are as follows:

Title:ABS-1230, a selective oral small molecule inhibitor of KCNT1 broadly inhibits pathogenic mutants and reduces seizures in a mouse model of KCNT1-related epilepsy.

Platform Presentation Session Number: Platform C.06
Platform Presentation Session Time/Date: Friday, December 5, 2025, 3:30-5:45 PM
Platform Presentation Location: GWCC; B312, Level 3
Poster Session Number: 1.366
Poster Presentation Time/Date: Saturday, December 6, 2025
Poster Presentation Location: GWCC, Exhibit Hall B2
Presenter: David Breckenridge, Ph.D., CSO, Actio Biosciences

About ABS-1230

ABS-1230 is designed to be a potent and selective orally available small molecule inhibitor of the KCNT1 ion channel and is initially in clinical development for the treatment of KCNT1-related epilepsy. The KCNT1 gene provides instructions for making potassium channels that allow potassium ions to flux out of cells, especially neurons in the brain. Gain-of-function mutations in the KCNT1 gene are the known genetic cause of KCNT1-related epilepsy — a rare, severe and often fatal form of pediatric epilepsy. The company has initiated a Phase 1a trial of ABS-1230 in healthy adult volunteers. Actio is also conducting preclinical studies to evaluate the therapeutic potential of ABS-1230 in more prevalent genetic epilepsies with related underlying biology. The FDA has granted ABS-1230 Rare Pediatric Disease, Fast Track and Orphan Drug designations for the treatment of KCNT1-related epilepsy.

About KCNT1-related Epilepsy

KCNT1-related epilepsy is a rare and often fatal pediatric developmental epileptic encephalopathy with a U.S. prevalence of approximately 2,500 patients. KCNT1-related epilepsy patients typically have a normal prenatal course before developing sporadic, asynchronous focal seizures. Seizure onset is often in the first six months of life, and the median age of onset is approximately 3.5 weeks. Seizure frequency tends to increase over time and can reach up to 50 seizures per day (15-20 per day on average). Other common symptoms and complications of KCNT1-related epilepsy include developmental delays and profound cognitive impairments and neurological deficits. There are currently no approved therapies for KCNT1-related epilepsy.

About Actio Biosciences

Actio Biosciences is a clinical-stage biotechnology company advancing the translation of genetic insights into novel precision medicines. The company applies deep expertise in Mendelian genetics, drug discovery and data science to identify targets whose biology underlies rare diseases with high unmet need and holds promise for the treatment of more prevalent indications. By precisely targeting the root causes of rare diseases Actio generates biological insights to enable expansion of development into more prevalent indications.

The company is advancing two lead clinical-stage programs, ABS-1230, initially being developed for the treatment of KCNT1-related epilepsy, and ABS-0871, initially being developed for the treatment of Charcot-Marie-Tooth disease type 2C including other TRPV4-related neuromuscular disorders (collectively, CMT2C). Actio plans to pursue development of both lead programs in more prevalent indications such as additional genetic epilepsies for ABS-1230 and overactive bladder for ABS-0871. For more information, please visit ActioBiosciences.com and follow the company on LinkedIn and X.

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SOURCE: Actio Biosciences

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PUB: 11/24/2025 09:00 AM/DISC: 11/24/2025 09:01 AM

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