Xenon Pharmaceuticals (XENE) 44th Annual J.P. Morgan Healthcare Conference summary

Strategic objectives and leadership

• Focus on becoming a fully integrated biopharma company with expertise in CNS ion channels and small molecule drug discovery.

• Majority of resources allocated to azetukalner, aiming for first drug approval following phase III data.

• Expansion plans include neuropsychiatry and early-stage pipeline development.

• Recent hires bring commercial and financial expertise to support forward integration.

Corporate and financial overview

• Neuroscience-focused biopharma with $555.3M in cash and equivalents as of September 30, 2025, with runway into 2027.

• Pipeline includes sodium and potassium channel modulators for epilepsy, pain, and neuropsychiatry.

Clinical development and pipeline updates

• Six ongoing phase III trials for azetukalner in epilepsy and psychiatry, with first readout (X-TOLE2) expected in March 2026.

• X-TOLE2 and X-TOLE3 trials in focal onset seizures mirror successful phase IIb design, with extended double-blind period.

• Japanese subjects incorporated into global phase III, eliminating need for separate trials in Japan.

• XEN1701, a Nav1.7 inhibitor, is in Phase 1 for pain, with completion expected in 2026.

• Early-stage pipeline includes Nav1.7 and Kv7 programs, with Nav1.7 showing promising potency and selectivity in phase I.