Axonis Therapeutics is developing first-in-class oral KCC2 potentiator drugs, specifically targeting the KCC2 protein to restore functional inhibition in the brain. This approach aims to address neurological disorders such as epilepsy and pain by enhancing inhibitory signaling within the central nervous system.
Funding
$115M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
+2Founders
Product
Problem
Many neurological disorders, such as epilepsy and pain, are characterized by impaired inhibitory signaling within the central nervous system. Current treatments often fail to fully restore this inhibition, leading to suboptimal outcomes for patients.
Solution
Axonis Therapeutics is developing a new class of oral drugs called KCC2 potentiators that target the KCC2 protein, a key regulator of inhibitory neurotransmission in the brain. By enhancing KCC2 activity, these drugs aim to restore functional inhibition and address the underlying causes of neurological disorders. The lead drug candidate, AXN-027, is designed to improve neuronal function and provide better control over conditions like epilepsy and pain.
Target Audience
The primary target audience includes individuals suffering from neurological disorders such as epilepsy and chronic pain, as well as the physicians who treat them.
Features
- First-in-class oral KCC2 potentiator drugs
- Specifically targets the KCC2 protein to enhance inhibitory signaling
- Designed to restore functional inhibition in the brain
- Potential application in treating epilepsy, pain, and other CNS pathologies