Xenon Presents Clinical and Regulatory Milestones at J.P. Morgan Healthcare Conference
The company is advancing its lead KV7 potassium channel modulator azetukalner through multiple Phase III trials in epilepsy and mood disorders, and planning its transition to commercial-stage growth.
Neuroscience-focused biopharmaceutical company, Xenon Pharmaceuticals, has set out an ambitious set of late-stage clinical and regulatory objectives for year during the 2026 J.P. Morgan Healthcare Conference in San Francisco (1). Central to these objectives is the company’s lead KV7 potassium channel modulator, for the treatment of epilepsy, major depressive disorder (MDD), and bipolar depression (BPD), azetukalner.
Azetukalner is designed to work by opening potassium channels in the central nervous system (CNS), which allows potassium ions to flow and hyperpolarize neurons, reducing excessive neuronal firing — a key contributor to several neurologic and psychiatric disorders. The drug is the only KV7 potassium channel opener in development for multiple indications that is backed by long-term efficacy and safety data in epilepsy patients and proof-of-concept data in MDD patients.
Short term focus for the company centers on X-TOLE2 — a Phase III trial evaluating azetukalner as an adjunctive therapy for adults with focal onset seizures — with topline results anticipated in March 2026. Positive data from X-TOLE2 are intended to underpin a planned new drug application (NDA) in the second half of 2026, which, if accepted, would start the formal U.S. review process for Xenon’s first potential commercial product.
“We’re approaching an important inflection point as we expect to report topline data for our X-TOLE2 Phase III study of azetukalner in focal onset seizures later this quarter, a major milestone on the path toward regulatory submission, and ultimately, approval and our first commercial launch,” said Ian Mortimer, President and Chief Executive Officer of Xenon, in a company press release (1).
Beyond X-TOLE2, Xenon is running a total of five placebo-controlled Phase III studies of azetukalner across epilepsy and depression indications, representing one of the more extensive late-stage programs for a single small-molecule CNS asset.
In epilepsy, additional trials include studies designed to expand the label into Primary Generalized Tonic-Clonic Seizures (PGTCS) and to further characterize safety and efficacy across broader focal epilepsy populations. In parallel, the company is advancing Phase III programs in major depressive disorder and bipolar depression that could translate into therapeutic benefits beyond seizure control.
“We are excited about the anticipation in the epilepsy community and our potential opportunity to bring a different mechanism to the epilepsy treatment paradigm,” added Mortimer in the press release (1). “We continue to build momentum through progress across five additional placebo-controlled Phase III studies of azetukalner in epilepsy and neuropsychiatry, and through our maturing pipeline with multiple studies underway in our early-stage pain portfolio.”
Xenon’s leadership also pointed to growing interest in mechanistically novel treatments in mood disorders, an area where many patients fail to respond adequately to standard antidepressants and mood stabilizers.
Underpinning the late-stage portfolio is a maturing early pipeline of ion channel modulators targeting pain, including KV7 and NaV1.7 programs now in Phase I development. These assets are designed to leverage Xenon’s long-standing expertise in ion channel biology, with the goal of producing non-opioid treatments that can address chronic pain with improved specificity and tolerability compared with older sodium channel–blocking drugs.
The company expects multiple clinical readouts and trial initiations from its pain portfolio over the next 12 to 24 months, which could diversify its opportunities beyond epilepsy and psychiatry if the data are supportive. The timing of future financings will depend on factors such as data outcomes, regulatory interactions, and market conditions; however, Xenon has demonstrated that its current balance sheet provides sufficient runway to execute on the immediate clinical agenda.
Reference
Xenon Pharma. Xenon Outlines Key Upcoming Milestones at the 2026 J.P. Morgan Healthcare Conference. Press Release, Jan. 12, 2026.