Actio Biosciences has begun the KYRON Phase Ib/II trial of ABS-1230 for potassium sodium-activated channel subfamily T member 1 (KCNT1)-related epilepsy.
The company is advancing ABS-1230, a selective oral KCNT1 inhibitor, to address the genetic mechanism in this severe, rare, often fatal paediatric developmental epileptic encephalopathy.
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In the Phase Ia healthy volunteer trial, all ABS-1230 doses, including multiple 20mg doses, were well-tolerated, and no serious adverse events occurred.
The ongoing KYRON Phase Ib/II study will examine the pharmacokinetics, safety and tolerability of oral or feeding tube-administered ABS-1230 in individuals aged one month to 21 years.
It will evaluate the effects on seizure activity and neurodevelopmental outcomes.
KYRON consists of three parts: a 12-week open-label single-arm study, a 12-week randomised, double-blind, placebo-controlled arm, and an optional longer-term extension.
Initial participants will be older children and young adults, while younger groups will be included based on the evaluation of safety and optimal dosing.
KCNT1-related epilepsy presents with frequent, treatment-resistant seizures from infancy, marked developmental delay, and limited response to general antiepileptic drugs.
The US Food and Drug Administration (FDA) has also accepted the therapy into its rare disease evidence principles (RDEP) process.
This framework aims to streamline the development of therapies for ultra-rare, genetically defined diseases.
Actio Biosciences CEO David Goldstein said: “The initiation of the KYRON trial marks a pivotal moment in our mission to bring a potentially disease-modifying, targeted therapy to children living with KCNT1-related epilepsy and their families.
“ABS-1230 is an investigational, precision-designed inhibitor of the overactive KCNT1 potassium ion channel that directly targets the underlying genetic driver of disease, and our preclinical and Phase Ia data reinforce our confidence in its potential to meaningfully reduce seizure burden.
“Acceptance into the FDA’s RDEP process is especially significant, as it provides a collaborative regulatory framework tailored to ultra-rare genetic diseases and may help clarify the path to bringing ABS-1230 to patients who urgently need new treatment options.”
In September 2025, Actio administered the initial dose to a subject in the Phase Ia clinical trial of ABS-1230.
