Actio Biosciences Update:
First-in-Human Trial Underway

A message for caregivers

Families affected by KCNT1-related epilepsy know how urgent the need is for new treatment options. No currently approved therapies specifically target KCNT1, but research is advancing to try to change that. ABS-1230, an investigational drug developed by Actio Biosciences, is currently being studied in its first clinical trial in human healthy volunteers.

This page is designed to give you a factual, non-promotional update on the status of the program, what type of therapy is being tested, and what you can expect in the near future. This is not an advertisement and does not suggest that the medicine is proven safe or effective. Please consult your clinician for any medical decisions.

About the investigational drug

Type of therapy

Small-molecule medicine (taken orally).

Clinical Trials (Interventional Studies)

Designed to inhibit the overactive KCNT1 potassium channel that causes the disease. Clinical trials will investigate whether ABS-1230 can calm the excessive activity caused by KCNT1 mutations, reduce seizures, and improve related symptoms.

Status

Investigational. The drug is not yet proven to be safe or effective and has not been approved by any regulatory agency.

Regulatory Designations

ABS-1230 has received the following designations from the United States Food and Drug Administration (U.S. FDA). Health authority designations allow for additional support from an agency; they do not imply that a drug is effective or that it will be approved for prescription use:

  • Fast Track Designation (FTD): Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patients faster.
  • Orphan Drug Designation (ODD): Designation granted for a drug or biological product that is intended to prevent, diagnose or treat a rare disease or condition.
  • Rare Pediatric Disease Designation (RPDD): Designation granted for a drug or biological product that is intended to prevent, diagnose or treat a rare pediatric disease or condition.

Current phase of development

Phase 1 trial (2025)

Help researchers track symptoms, development, and quality of life over time by participating in the KCNT1 International Registry. Sign up

In September 2025, Actio Biosciences began enrolling healthy adult volunteers in a Phase 1a clinical trial.
The Phase 1a study is being conducted in Australia with approval to proceed from both the Australia Therapeutic Goods Administration (TGA) and the United States Food and Drug Administration (U.S. FDA). The study is registered on ClinicalTrials.gov under NCT07156201.

Why Phase 1 Matters

This first step looks at safety, tolerability, and how the body processes the drug. It does not assess whether the medicine reduces seizures.

What comes next

If the Phase 1a study results support continued development, Action plans to initiate a Phase 1b/2a clinical trial in patients with KCNT1-related epilepsy in early 2026 in the U.S., subject to regulatory feedback and operational progress. The trial intends to evaluate safety, tolerability, how the body processes the investigational drug and if the drug reduces seizures and other symptoms.

What caregivers can expect now

No Direct Access Yet

The drug is not available outside of clinical trials. Expanded access (“compassionate use”) is not currently in place.⁴

Next Updates

Initial Phase 1a results will inform whether studies in patients with KCNT1-related epilepsy can begin in 2026.

How to stay informed

You can sign up below to receive updates when new information becomes available. Sponsors, like Actio Biosciences, often share key milestones – such as trial start, first-participant dosed, or trial results – through press releases at their website and with the KCNT1 Epilepsy Foundation.

Stay connected

If you would like updates from the KCNT1 Epilepsy Foundation when new trial phases are announced, please share your name and email:

Sign up for Email List for Trial Updates here.

KCNT1 Caregiver Update — Key Points

KCNT1 disorders are rare, severe epilepsies caused by variants in the KCNT1 gene.

Investigational medicine (ABS-1230): The drug is not yet proven to be safe or effective and has not been approved by any regulatory agency.

Next steps:

  • Phase 1a (healthy volunteers) – the study is ongoing.
  • Phase 1b/2a (small patient study) planned for 2026.

Important: ABS-1230 is not approved anywhere; safety and effectiveness are still being studied.

Stay informed: Caregivers can sign up for non-promotional updates about news and resources.

References

  1. Orphanet. KCNT1-related developmental and epileptic encephalopathy. Link
  2. Actio Biosciences. Actio Biosciences Receives FDA IND Clearance and Fast Track Designation for KCNT1 Developmental and Epileptic Encephalopathy Program. Press release, July 30, 2025. Link
  3. Actio Biosciences. Actio Biosciences Announces $55 Million Series B Financing to Advance Precision Neuroscience Programs. Press release, June 18, 2025. Link
  4. Actio Biosciences. Expanded Access Policy. Link
  5. U.S. Food and Drug Administration (FDA). Prescription Drug Advertising: Questions and Answers. Link
  6. U.S. Food and Drug Administration (FDA). Prescription Drug Advertising Basics. Link
  7. Directive 2001/83/EC of the European Parliament and of the Council on the Community code relating to medicinal products for human use. Link
  8. European Medicines Agency (EMA). Rules governing medicinal products in the European Union, Volume 2A, Chapter 7: Advertising. Link

This page is structured to comply with U.S. and EU regulations that restrict advertising of investigational medicines to the public. It provides non-promotional, informational content only.