Xenon Pharmaceuticals (XENE) investor relations material

Clinical data and product profile

• Phase III X-TOLE2 study showed 43% placebo-adjusted seizure reduction at highest dose and 7% complete remission in refractory focal onset epilepsy patients.

• Efficacy in phase III exceeded phase II results, with strong performance in a heavily pretreated population, including those who failed cenobamate.

• Azetukalner offers a novel Kv7 mechanism, once-daily dosing, no titration, minimal drug interactions, and strong efficacy in both double-blind and open-label periods.

• Open-label extension data show nearly 40% of long-term patients achieving at least 12 months of seizure freedom, with high rollover rates and sustained benefit.

• Four doses (10, 15, 20, 25 mg) are planned for the label, with most physicians expected to start at 15 mg due to efficacy and safety.

Regulatory and launch plans

• NDA submission for azetukalner is targeted for Q3, with most nonclinical and CMC sections ready and focus now on integrating safety data.

• Regular FDA interactions and a pre-NDA meeting are planned; standard review is expected rather than priority review.

• Commercial infrastructure is being built, including a field force of about 75 reps and total commercial FTEs around 150.

• Targeting 7,500–8,000 core physicians (epileptologists and select general neurologists) for initial launch.

• Pre-launch activities include MSL engagement, market access preparation, and leadership team buildout.

Market opportunity and positioning

• U.S. has about 1.8 million focal onset epilepsy patients; focus is on those with partial or poor control after multiple therapies.

• Aim is to move azetukalner upstream as an early add-on, not as frontline therapy.

• Ease of use, novel mechanism, and efficacy resonate with both specialists and general neurologists.

• Feedback from KOLs highlights demand for new mechanisms and long-term efficacy.