AXN-027

AXN-027 is an oral, first‑in‑class small‑molecule potentiator of the neuron‑specific potassium‑chloride co‑transporter 2 (KCC2; solute carrier family 12 member 5) being developed by Axonis Therapeutics for refractory epilepsy, chronic/neuropathic pain, and spinal cord injury.[5][9][11] By allosterically enhancing KCC2 function, AXN-027 increases neuronal chloride extrusion and restores inhibitory GABAergic signaling in hyperexcitable CNS circuits, aiming to control seizures and reduce pathological pain and spasticity without the global CNS depression and sedation associated with many existing antiepileptic and analgesic drugs.[4][9][11] The drug is formulated as an oral salt (capsules and tablets) and is currently in Phase 1 clinical trials in healthy volunteers and patients, including a two‑part study assessing safety, tolerability, pharmacokinetics, cerebrospinal fluid penetration, and food effects.[1][3][5][11]