What's Happening?
Actio Biosciences has announced the dosing of the first participant in its Phase 1 clinical trial for ABS-1230, a KCNT1 inhibitor aimed at treating KCNT1-related epilepsy. This rare and severe pediatric epileptic encephalopathy affects approximately 2,500 individuals in the U.S. The trial, conducted in Australia, will assess the safety, tolerability, and pharmacokinetics of ABS-1230 in healthy volunteers. Actio plans to expand into a Phase 1b/2a study in the U.S. in early 2026, targeting patients with KCNT1-related epilepsy.
Why It's Important?
The initiation of this clinical trial marks a significant step forward in addressing KCNT1-related epilepsy, a condition with limited treatment options. ABS-1230 has the potential to offer a disease-modifying therapy, which could significantly improve the quality of life for affected patients. The trial underscores Actio Biosciences' commitment to developing precision medicine solutions for rare genetic diseases, potentially paving the way for new treatments that target the root causes of such conditions.
What's Next?
Following the Phase 1 trial, Actio Biosciences plans to conduct a Phase 1b/2a proof-of-concept study in the U.S. in early 2026. This expansion will involve KCNT1-related epilepsy patients, aiming to further evaluate the efficacy and safety of ABS-1230. The company is also advancing other drug candidates, indicating a robust pipeline focused on rare genetic diseases.
