Xenon Pharmaceuticals (XENE) Investor presentation summary

Strategic overview

• Neuroscience-focused small-molecule ion-channel pipeline spans potassium and sodium channels; azetukalner (AZK), a Kv7 opener, is in late-stage development for epilepsy and neuropsychiatric disorders.

• Cash, cash equivalents and marketable securities totaled $1.2B, with runway into 2029.

• Strategy centers on AZK approval and launch in epilepsy, expanding AZK into neuropsychiatry, and advancing early-stage pain candidates.

Epilepsy

• FDA NDA submitted in Q3 2026 for focal seizures, based on positive X-TOLE and X-TOLE2 randomized trials; FDA review and approval would precede launch.

• In X-TOLE2, 25 mg AZK produced a 42.7% placebo-adjusted reduction in monthly seizure frequency over 12 weeks; RR50 was 54.8% vs. 20.8% with placebo.

• X-TOLE2 TEAEs occurred in 82.3% with AZK 25 mg vs. 62.4% with placebo; serious adverse events were 5.6% vs. 2.4%, respectively.

• In the X-TOLE open-label extension, monthly seizure frequency was reduced 90.9% from double-blind baseline at month 48; long-term data cutoff was October 6, 2025.

• X-TOLE3 and X-ACKT Phase 3 studies are ongoing; X-FIRST, a Phase 3b/4 first add-on study, is expected to initiate in Q1 2027.

Neuropsychiatry

• In Phase 2 X-NOVA, 20 mg AZK showed a 3.04-point MADRS improvement vs. placebo at week 6, not statistically significant (p=0.135); HAM-D17 and SHAPS differences were nominally significant (p=0.042 and p=0.046).

• X-NOVA2 enrollment completed at ~360 participants; topline data are expected in December 2026. New enrollment in X-NOVA3 and the MDD open-label extension is temporarily paused.

• The Phase 3 MDD program includes three placebo-controlled studies, with a planned sNDA supported by two positive studies; the Phase 3 BPD program includes two studies, with new enrollment temporarily paused.