Actio Biosciences has launched a major clinical push in rare disease drug development.

 

The biopharma powerhouse has announced the initiation of its KYRON Phase 1b/2 trial of ABS-1230 for KCNT1-related epilepsy, a devastating pediatric neurological disorder marked by severe, treatment-resistant seizures and early-life onset.

 

The company also confirmed that ABS-1230 has been accepted into the U.S. Food and Drug Administration’s Rare Disease Evidence Principles (RDEP) process — a regulatory pathway designed to streamline development for ultra-rare genetic diseases with urgent unmet need.

 

ABS-1230 is an oral small molecule designed to selectively inhibit the overactive KCNT1 potassium ion channel, targeting what researchers believe is the root genetic driver of the disease.

 

“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,” said David Goldstein, CEO of Actio Biosciences. 

 

“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 1a 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.”

 

KCNT1-related epilepsy typically begins in infancy and is associated with relentless seizures, profound developmental impairment, and high medical fragility. Existing anti-epileptic treatments often provide limited benefit.

 

“For families in this community, including mine, the focus each day is simple and urgent: keeping our children alive. Relentless seizures, medical fragility and profound developmental challenges define daily life,” said Justin West, M.D., co-founder and president of the KCNT1 Epilepsy Foundation. 

 

“The start of Actio’s KYRON trial represents real progress and renewed hope for families who have waited far too long for a treatment designed specifically for KCNT1-related epilepsy. We are deeply grateful to the patients, caregivers and investigators who are driving this work forward, and we are encouraged by what this program could mean for reducing seizure burden and improving quality of life for these children.”

 

Earlier Phase 1a data in healthy volunteers showed the drug was well tolerated across all tested doses, including multiple 20 mg administrations, with no serious adverse events reported.

 

The KYRON study will enroll children and young adults from one month to 21 years old and evaluate safety, tolerability, pharmacokinetics, and seizure outcomes. It will run in three stages: an initial 12-week open-label phase, a randomized placebo-controlled phase, and a long-term extension for eligible participants.

Initial dosing will begin with older children and young adults, with younger age groups added after safety and dosing are confirmed.

 

If successful, the therapy could represent one of the first precision medicines targeting the underlying genetic mechanism of KCNT1-related epilepsy — a condition with few effective treatment options and high unmet medical need.