Actio Biosciences has administered the initial dose to a subject in the Phase Ia clinical trial of ABS-1230, a potassium sodium-activated channel subfamily T member 1 (KCNT1) inhibitor aimed at treating epilepsy.
The Phase Ia trial represents a double-blind, randomised, placebo-controlled study that examines single and multiple ascending doses, as well as the effects of food, in healthy participants.
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It is being carried out in Australia and will evaluate the pharmacokinetics, safety and tolerability of ABS-1230.
Actio intends to progress to a proof-of-concept Phase Ib/IIa trial involving subjects with KCNT1-related epilepsy in the US, starting in 2026.
Epilepsy impacts around 2,500 people in the US and is classified as a rare and severe form of developmental epileptic encephalopathy in children, often resulting in fatal outcomes.
Actio CEO David Goldstein said: “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.
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By GlobalData“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.
“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.”
Preclinical trials have demonstrated that ABS-1230 inhibits all pathogenic mutations tested in the KCNT1 gene, suggesting its potential as a treatment option for all individuals with epilepsy.
Subjects with this condition suffer from frequent seizures that are resistant to treatment, starting in early infancy.
