AXN-xxx

AXN-xxx refers to a series of oral small molecule KCC2 potentiators being developed by Axonis Therapeutics for the treatment of various neurological disorders. These compounds are designed to enhance the activity of the potassium-chloride cotransporter 2 (KCC2), a neuron-specific transporter essential for maintaining low intracellular chloride levels, which is critical for effective GABAergic inhibitory neurotransmission. By restoring the excitatory/inhibitory (E/I) balance in the central nervous system, AXN-xxx aims to address conditions characterized by E/I imbalance, such as refractory epilepsy, chronic pain, and spinal cord injury. This program follows the lead candidate AXN-027, which is transitioning into clinical development.