New oral medication to treat seizures in patients with mesial temporal lobe epilepsy
Official title: Development of Oral KCC2 Potentiator AXN-027 as a Novel Anti-Epileptic Drug for Mesial Temporal Lobe Epilepsy
Funded at Axonis Therapeutics, INC. · NIH-10922018 · Data updated 2026-11-12
This study is testing a new pill called AXN-027 to help people with mesial temporal lobe epilepsy (mTLE) by making a brain protein work better, which could lead to fewer seizures for those who haven't found relief with current treatments.
Quick facts
| Grant type | Sbir 1 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Axonis Therapeutics, INC. NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | September 1, 2024 |
| Project end | August 31, 2025 |
| Funding year | 2024 |
| Award (this year) | $323,001 |
| NIH project number | 1R43NS137942-01 |
| Project ID | NIH-10922018 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with mesial temporal lobe epilepsy who have not responded adequately to standard anti-epileptic medications.
✕Less likely to help
Patients with epilepsy types other than mesial temporal lobe epilepsy or those who have not experienced drug resistance may not benefit from this research.
What this research studies
This research focuses on developing a new oral medication called AXN-027, which aims to treat mesial temporal lobe epilepsy (mTLE) by enhancing the function of a specific protein, KCC2, that helps regulate neuronal excitability. Many patients with mTLE experience seizures that do not respond to existing treatments, and this research seeks to address that challenge. By improving the balance of excitation and inhibition in the brain, AXN-027 could potentially reduce the frequency of seizures. The approach involves pharmacologically potentiating KCC2 to restore its normal function, which is crucial for effective seizure control.
Why it matters
Potential benefit: If successful, this research could provide a new treatment option for patients with drug-resistant mesial temporal lobe epilepsy, potentially reducing their seizure frequency and improving their quality of life.
How similar studies have gone: While the approach of targeting KCC2 for epilepsy treatment is novel, previous research has shown promise in enhancing KCC2 function to reduce seizure activity in animal models.
Where this research is happening
-
Axonis Therapeutics, INC.
Boston, United States Active
Researchers
- Principal investigator: Kadam, Shilpa Dattatray — Axonis Therapeutics, INC.
- Study coordinator: Kadam, Shilpa Dattatray
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About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.