Pipeline
Translating ion channel science into meaningful medicines
With deep expertise in ion channel biology, Xenon is advancing a differentiated pipeline led by our late‑stage investigational candidate, azetukalner, which is being studied in multiple Phase 3 trials in epilepsy, major depressive disorder (MDD), and bipolar depression (BPD). Our early-stage pipeline features Kv7 potassium channel openers and Nav sodium channel modulators being advanced for select high‑need indications.
Our pipeline of ion channel modulators
Reflecting our commitment to a sustainable pipeline, our diverse portfolio of candidates is positioned to realize the broad potential of ion channel science.
Xenon Programs
This graphic displays pipeline drug candidates undergoing testing in a variety of disease indications. The safety and efficacy of these investigational drug candidates have not been fully evaluated, and they have not yet been approved for use by any regulatory authorities.
Our approach in epilepsy
We are developing a novel, potent Kv7 potassium channel opener, azetukalner, which is in Phase 3 clinical trials for the treatment of focal and generalized seizures. If approved, azetukalner would be the only potassium channel opener among antiseizure medications.
With azetukalner, we are also exploring the Kv7 potassium channel as a key regulator of neuronal excitability that may play a role in both epilepsy and depression, which is increasingly being recognized as a neuronal circuit disorder.
Our approach in depression
Supported by azetukalner’s Kv7-targeting mechanism that reduces neuronal hyperexcitability, we have expanded its potential use into depression. Following completion of the Phase 2 X-NOVA™ study in MDD, we are advancing a Phase 3 clinical program that includes both MDD and BPD.
Our approach in pain
We are exploring pain indications by leveraging our expertise in ion channels and heritage in human genetics. We are evaluating two distinct approaches: 1) blocking the activity of the sodium channels (Nav1.7 inhibition) that trigger pain signaling; and 2) enhancing the activity of the potassium channels (Kv7 openers) that stop pain signals.
We have extensive history researching the sodium channel Nav1.7 as a novel pain target, both through multiple industry collaborations and our own discovery research. This history started more than 20 years ago with the identification of a loss of function in the sodium channel Nav1.7 as the cause of congenital indifference to pain (CIP), an extremely rare autosomal recessive disorder whose carriers are unable to feel pain but are otherwise healthy. Since then, we have focused on optimizing chemistry to address the limitations of earlier drug candidates targeting Nav1.7 and believe we are now on the precipice of realizing the potential of this promising pain target with our XEN1701 clinical program, which is being investigated in a Phase 1 study.
We have also initiated a Phase 1 study of our first Kv7 program in pain, XEN1120.