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

Strategic objectives and leadership

• Aims to become a fully integrated biopharma company, focusing on discovering, developing, and commercializing CNS ion channel molecules.

• Recent hires in commercial and financial leadership bring experience in transitioning to a commercial organization.

• Early investment in building relationships within the epilepsy community and assembling a team with deep epilepsy expertise.

Corporate and financial overview

• Neuroscience-focused biopharma with expertise in small molecule, ion channel drug discovery and development.

• $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.

Lead asset: azetukalner clinical progress

• Azetukalner is a potent Kv7 channel opener, the most advanced Kv7 modulator, in six ongoing phase III trials: three in epilepsy and three in neuropsychiatry.

• Over 800 patient-years of efficacy and safety data for azetukalner.

• X-TOLE2 phase III trial in focal onset seizures completed randomization; top-line data expected in March 2026.

• X-TOLE3 includes Japanese subjects, enabling global regulatory alignment without separate Japanese trials.

• Phase III trial in primary generalized tonic-clonic seizures is ongoing.