Xenon Pharmaceuticals Inc. (XENE) has unveiled positive results from its Phase 3 X-TOLE2 study of Azetukalner (AZK) in adults with focal onset seizures (FOS), presented at the American Academy of Neurology (AAN) 2026 Annual Meeting in Chicago.
The late-breaking data showed that patients receiving Azetukalner 25 mg achieved a 53.2% reduction in monthly seizure frequency compared to 10.4% with placebo. Importantly, increased rates of complete seizure freedom were observed over the 12-week double-blind period, underscoring the drug's potential to deliver durable seizure control.
The X-TOLE2 study enrolled 380 adults with highly treatment-resistant epilepsy, including patients who had previously tried or discontinued Cenobamate. Results showed dose-dependent improvements across weekly and monthly seizure measures, with some patients achieving complete seizure control.
Long-term findings from the ongoing X-TOLE open-label extension (OLE) study further reinforced these results. At 48 months, nearly 40% of participants achieved at least 12 months of seizure freedom, while one in four maintained seizure freedom for 24 months or longer. Extended treatment showed seizure reductions approximately 90% at 48 months, with safety remaining stable across more than 775 patient-years of exposure.
Azetukalner is a novel KV7 potassium channel opener designed to reduce excessive neuronal firing without the need for titration, simplifying epilepsy management for patients and physicians. The safety profile remained consistent with earlier studies, with dizziness, headache, and fatigue as the most common side effects, and no major cardiovascular or retinal concerns reported.
Real-world findings presented at AAN highlighted the burden of titration in epilepsy care. Patients and physicians reported that no-titration therapies like Azetukalner could reduce stress, improve adherence, and simplify management.
Xenon's leadership emphasized that Azetukalner demonstrated the strongest placebo-adjusted efficacy ever observed in a pivotal focal seizure study. The company plans to submit a New Drug Application (NDA) to the FDA in the third quarter of 2026, aiming to bring this once-daily, no-titration therapy closer to patients living with uncontrolled seizures.
Epilepsy affects nearly three million adults in the U.S., with focal onset seizures being the most common form. Despite multiple available treatments, many patients continue to struggle with uncontrolled seizures, highlighting the urgent need for innovative therapies. Xenon's data position Azetukalner as a promising candidate to reshape epilepsy care.
XENE has traded between $28.19 and $63.95 over the past year. The stock closed Friday's trading (April 17, 2026) at $59.70, up 2.17%. During the overnight trading session, the stock traded at $59.50, down 0.34%.
For comments and feedback contact: editorial@rttnews.com
Business News
April 17, 2026 15:29 ET The ongoing conflict in the Middle East continues to raise concerns for policymakers who worry about the impact of the supply shock and high energy prices on the real economy. Producer price data and various survey results on the housing market were the main news from the U.S. this week. In Europe, industrial production data for the euro area gained attention. GDP figures out of China and the policy move by the Singapore central bank were in focus in Asia.