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Xenon Pharmaceuticals (XENE) investor relations material

Xenon Pharmaceuticals Study Update summary

Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.
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Study Update summary6 Oct, 2025

Corporate and Pipeline Overview

  • Focus on neuroscience and ion channel drug discovery, with expertise in potassium (Kv7) and sodium (Nav1.7) channels for pain and neurological disorders.

  • Lead molecule azetukalner is in phase III for epilepsy and depression, with over 700 patient years of data.

  • Lead pain compounds XEN1120 (Kv7 opener) and XEN1701 (Nav1.7 inhibitor) are both in phase I clinical studies.

  • Early-stage pipeline includes Kv7 openers and Nav1.7 inhibitors for pain, both in phase I, with strong patent protection to at least 2040.

  • Additional programs target Nav1.1 for Dravet Syndrome and collaborations on Nav1.6/1.2 inhibitors for epilepsy.

Scientific Rationale and Discovery

  • Human genetics underpin the selection of ion channel targets, with Nav1.7 loss-of-function mutations causing congenital insensitivity to pain.

  • Kv7 and Nav1.7 channels are critical in pain transduction, transmission, and relay, making them compelling non-opioid pain targets.

  • Preclinical models show Kv7 openers and Nav1.7 inhibitors reduce pain signaling at multiple pathway points.

  • Advanced in-house capabilities include electrophysiology, computational chemistry, and AI-driven drug design.

  • Pain signals travel from peripheral tissues through DRG neurons to the spinal cord and brain.

Kv7 Pain Program Highlights

  • Kv7 channels suppress repetitive firing; dysfunction is linked to pain states.

  • Kv7 openers decrease excitability in DRG and spinal cord, blocking pain signaling.

  • XEN1120 shows efficacy in preclinical models, including acute sensory pain where most non-opioids are ineffective.

  • Phase 1 study of XEN1120 is underway, with additional Kv7 compounds advancing toward IND-enabling studies.

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Frequently asked questions

Xenon Pharmaceuticals Inc. is a clinical-stage biopharmaceutical company focused on developing innovative therapeutics for neurological disorders. The company leverages its expertise in ion channel biology to advance a pipeline of small-molecule therapies aimed at treating conditions such as epilepsy, rare neurological diseases, and other central nervous system disorders. The company is headquartered in Burnaby, Canada, and its shares are listed on the NASDAQ.

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