Our Science
We are developing first- and best-in-class oral therapeutics for KCC2, a currently undrugged but highly validated CNS-specific target that can restore balance to disinhibited brain circuits to treat neurological disorders without disabling side effects.
Platform & Pipeline
KCC2 DISCOVERY ENGINE TO UNLOCK FULL THERAPEUTIC POTENTIAL OF THIS SPACE
We have built a proprietary and specialized discovery engine to unlock the neuro-blockbuster drug potential for KCC2 treatments of neurological disorders. World leading experts in KCC2 biology within and outside of Axonis have collaborated to optimize and develop these customized assays, which are being used to identify and advance the best candidate compounds for therapeutic evaluation in humans.
PORTFOLIO OF NOVEL COMPOUNDS TO DEVELOP FIRST-IN-CLASS ORAL KCC2 POTENTIATORS
Axonis’ lead candidate AXN-027 is a first- and best-in-class oral KCC2-potentiating drug currently being evaluated in a Phase 1 clinical trial, and is being developed for the treatment of refractory epilepsy, chronic pain, and spinal cord injury. Additionally, a portfolio of KCC2 potentiating drugs are under development for multiple neurological disorders.
