Azetukalner (XEN1101) - (X-TOLE2)

May 7, 2026: Xenon Pharmaceuticals Inc. (NASDAQ: XENE) provided a pipeline update and Q1 2026 financial results. Lead program azetukalner (KV7 potassium channel opener) reported positive Phase 3 X-TOLE2 topline data in focal onset seizures (FOS) (announced March 2026): the study met the primary endpoint (median percent change in monthly FOS frequency to week 12) for both 25 mg and 15 mg vs placebo (MPC -53.2%, -34.5% vs -10.4%; p<0.0001 for both). Xenon plans an FDA NDA submission in Q3 2026, supported by X-TOLE2 and prior X-TOLE results. X-TOLE2 data were presented as late-breaking at the AAN Annual Meeting (Apr 18–22, 2026); 48-month open-label extension data were also presented.

Ongoing Phase 3 azetukalner studies: X-TOLE3 in FOS (to support ex-U.S. filings; enrollment outside Japan expected to complete in 2026), X-ACKT in primary generalized tonic-clonic seizures, X-NOVA2/X-NOVA3 in major depressive disorder (X-NOVA2 topline expected H1 2027), and X-CEED in bipolar depression.

Pain R&D: Phase 1 SAD/MAD studies of XEN1701 (NaV1.7) and XEN1120 (KV7) expected to complete H2 2026 to support Phase 2 acute pain proof-of-concept. Partnered with Neurocrine, Phase 1b data for NBI-921355 (NaV1.2/NaV1.6 inhibitor) are expected in 2027.