KCNT1 Therapeutic Development News & Updates

Stage 1: Discovery & Development

Several companies and labs are in the discovery and development stage which spans basic research in the lab to animal model research. During this step a potential therapeutic is identified and tested in many ways in the lab to show that it works (called efficacy), and a most promising version of the therapeutic (often called the lead candidate) is chosen for next stages. This stage may involve 5-10 years of testing.

Stage 2: Preclinical Research & Development

After a lead candidate has been selected in the discovery stage, the candidate therapeutic must be tested more carefully and strenuously for safety and efficacy in cell culture and animal models. These tests follow regulatory agency mandated requirements. This stage can last 3-5 years and if successful, results in an Investigational New Drug Application (IND) application to the Food and Drug Administration (FDA), which is a request to begin clinical trials. To date, no KCNT1 therapeutic has moved from the preclinical phase to the clinical phase, though we hope clinical trials may initiate in late 2025.

Stage 3: Clinical Trials – Phase 1, 2, and 3

Once an Investigational New Drug (IND) application is approved by the FDA, the therapeutic enters the clinical trial phase, which involves testing in humans. These trials are conducted in three progressive phases.

• Phase 1 involves a small group of healthy volunteers or affected individuals and is primarily focused on determining safety, dosage range, and side effects.
• Phase 2 expands the testing to a larger group of affected individuals to evaluate efficacy and continue monitoring safety.
• Phase 3 includes even larger populations—often hundreds of patients across multiple clinical sites—to confirm effectiveness, monitor side effects, and compare the drug to standard or placebo treatments.

For rare diseases like KCNT1-related epilepsy, patient recruitment is challenging and may require international collaboration and innovative trial designs. The full clinical trial process may span 5–7 years or longer depending on the availability of patients, trial design, and regulatory review. If successful, the company can then submit a New Drug Application (NDA) to the FDA for marketing approval.

Stage 4: FDA Review, Approval & Post-Market Monitoring (Phase 4)

If the Phase 3 results support the therapeutic’s safety and effectiveness, the FDA will review the NDA, which includes all data from laboratory, animal, and human studies. This regulatory review period typically takes 6–10 months. If approved, the therapeutic may be sold and prescribed to patients. However, the process doesn’t end here.

In Phase 4 (post-marketing surveillance), the therapeutic continues to be monitored for long-term safety, side effects, and effectiveness in the broader population. This stage is crucial for identifying rare adverse events that may not have appeared during earlier phases and may lead to updated warnings, label changes, or in rare cases, withdrawal from the market. For KCNT1, post-market studies could help identify long-term impacts on neurodevelopment or seizure burden and inform ongoing care strategies.