Actio Biosciences Announces Preclinical Data Highlighting Ability of ABS-1230 to Potently and Selectively Inhibit KCNT1 and Significantly Reduce Seizure Activity
Actio Biosciences Announces Preclinical Data Highlighting Ability of ABS-1230 to Potently and Selectively Inhibit KCNT1 and Significantly Reduce Seizure Activity
Data to be Presented During an Oral Platform Presentation at the American Epilepsy Society Annual Meeting
SAN DIEGO–(BUSINESS WIRE)–Actio Biosciences, a clinical-stage biotechnology company advancing the translation of genetic insights into novel precision medicines, today announced preclinical data related to the company’s co-lead candidate ABS-1230, a selective small molecule KCNT1 inhibitor for the treatment of KCNT1-related epilepsy. The data are being presented today, December 5, in a platform presentation by Actio CSO David Breckenridge, Ph.D., from 3:30-5:45 p.m. at the American Epilepsy Society (AES) Annual Meeting in Atlanta.
The preclinical data support the therapeutic potential of ABS-1230 to reduce seizures by selectively inhibiting mutations of the KCNT1 gene, which cause overactivation of the potassium channel and abnormal electrical brain activity, leading to a severe, early-onset epileptic encephalopathy. Actio is currently advancing ABS-1230 in a Phase 1a healthy volunteer clinical trial and plans to initiate a Phase 1b/2a clinical trial in patients with KCNT1-related epilepsy in 2026.
“Development of treatments for KCNT1-related epilepsy has been limited by the large number of distinct pathogenic mutations, many of which are resistant to known inhibitors,” said David Goldstein, Ph.D., CEO of Actio. “In our preclinical work, ABS-1230 has consistently demonstrated its ability to potently and selectively inhibit KCNT1 and pathogenic mutations leading to notable reductions in seizures in a mouse model. We’re pleased to share these data today, which support ABS-1230 as a targeted therapy with the potential to address the immense burden and unmet need for patients with KCNT1-related epilepsy. Our Phase 1a trial of ABS-1230 in healthy volunteers is ongoing, and we look forward to reporting data from that study in early 2026 and advancing into a first-in-patient trial next year.”
Presentation Highlights
In preclinical human and mouse models, administration of ABS-1230 led to:
- Potent and selective inhibition of KCNT1 and known pathogenic mutations
- High oral bioavailability and brain penetration across species
- Significant dose-dependent reduction of seizures in a KCNT1 mouse model relative to vehicle control treatment
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 Actio estimates to be 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. In addition to Actio’s two lead programs, Actio has also launched a third program for a rare genetic epilepsy. For more information, please visit ActioBiosciences.com and follow the company on LinkedIn and X.
Contacts
Contact
Katie Engleman, 1AB
katie@1abmedia.com
Investor Contact:
Renee Leck, THRUST
renee@thrustsc.com