Science & Pipeline

Ion channels are proteins that control the flow of ions across cell membranes, playing a crucial role in neuronal excitability—the basis for communication within the nervous system. Neuronal hyperexcitability, a hallmark of many central nervous system (CNS) and other disorders, often stems from ion channel dysfunction. When certain neurons exhibit increased activity this can, for example, manifest as seizures in epilepsy, or as painful sensations. or other neurological symptoms in many common diseases, reflecting an imbalance between excitatory and inhibitory signaling in the brain.

Xenon is focused on advancing a novel ion channel product pipeline to address areas of high unmet medical need:

• Epilepsy and seizures can develop in any person at any age, and approximately 1 in 26 people will develop epilepsy in their lifetime. Despite the availability of multiple anti-seizure medications, a substantial unmet medical need exists with up to 50% of epilepsy patients requiring additional treatment options.
• Major Depressive Disorder (MDD) is highly prevalent, with an estimated 21 million adults in the US affected by MDD. Many who are taking anti-depressant medications may still have unresolved symptoms, indicating the need for novel mechanisms that offer efficacy.

Our portfolio focuses on developing highly potent and selective ion channel therapeutics that aim to restore proper neuronal function. Our lead candidate, azetukalner, is a novel, potent Kv7 potassium channel opener in advanced clinical trials for epilepsy and major depressive disorder.

Beyond azetukalner, candidates focus on other ion channels, such as Nav1.1 and Nav1.7 sodium channels, to advance our commitment to providing a better future for patients suffering from a range of psychiatric and neurological disorders.