Actio Biosciences to Present New Preclinical
Actio Biosciences to Present New Preclinical Data for ABS-1230, a KCNT1 Inhibitor for the Treatment of Epilepsy
SAN DIEGO -- 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.
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. 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 Actio Biosciences
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.
