Xenon Pharmaceuticals (NasdaqGM:XENE) 2025 Conference Transcript

Xenon Pharmaceuticals Conference Call Summary Company Overview - Company: Xenon Pharmaceuticals (NasdaqGM:XENE) - Focus: Development of innovative therapies for epilepsy, psychiatry, and pain management Key Themes Discussed 1. Lead Program - Azetukalner in Epilepsy - Near-term phase 3 data expected in early 2026 from the fully enrolled EXTOL-two program [2][4] - Positive phase 2b data showing best efficacy in focal onset seizure studies with a severe patient population [6][7] - High confidence in phase 3 readout due to consistent inclusion-exclusion criteria and experienced clinical sites [10][11] 2. Expansion into Psychiatry - Ongoing phase 3 trials for major depressive disorder (MDD) and recently initiated trials for bipolar depression [2][3] - Phase 2 proof of concept study (ExNOVA) showed clear separation between active and placebo with a three-point difference on clinical scales [25][26] 3. Emerging Early-Stage Portfolio in Pain - Two phase 1 assets targeting NaV1.7 and Kv7, with plans to advance to phase 2 proof of concept studies in 2026 [3][50] - NaV1.7 target based on genetic studies showing patients with loss of function do not feel pain [36][37] Important Data and Insights - Azetukalner Efficacy: Placebo-adjusted efficacy in phase 2b ranged from teens to low 30s in monthly seizure reduction [9] - Commercial Strategy: Engaging with the epilepsy community and preparing for a successful launch with a Chief Commercial Officer and medical science liaisons in place [14][16] - Market Opportunity: Approximately 1 million patients in the U.S. with focal onset seizures are not achieving good seizure control [18] Competitive Landscape - Market Dynamics: Acknowledgment of multiple existing drugs in the focal onset seizure market, but belief in the unique profile of Azetukalner to differentiate from competitors [34][35] - Regulatory Path: Plans to file NDA next year, with Exfil and Exfil II studies included in the filing package [12][13] Pipeline Developments 1. NaV1.7 and Kv7 Programs - NaV1.7 in phase I study with a focus on achieving high receptor occupancy and safety [39][40] - Kv7 program showing potential analgesic effects similar to opioids [50] 2. NaV1.1 for Dravet Syndrome - Development of an oral small molecule to potentiate NaV1.1 channels, aiming to protect against seizures and improve survival in animal models [51][53] - Plans to initiate human clinical trials next year [54] Financial Position - Cash Reserves: Over $500 million in cash at the end of the last quarter, expected to sustain operations into 2027 [55]