Xenon Pharmaceuticals (XENE) Jefferies London Healthcare Conference 2024 summary

Portfolio overview and clinical focus

• Lead asset azetukalner is a potassium channel modulator with ongoing phase 3 trials in epilepsy and planned expansion into psychiatry, specifically major depressive disorder (MDD).

• Three phase 3 trials are underway for epilepsy (focal onset and primary generalized tonic-clonic seizures), and three phase 3 trials in MDD are planned, with the first starting by year-end and two more in 2025.

• Early-stage pipeline includes multiple Kv7 candidates and sodium channel programs (NaV1.1 for Dravet syndrome and NaV1.7 for pain), with several IND filings expected in 2025.

• The company is well-funded, with over $800 million in cash, supporting operations and pipeline maturation into 2027.

Key milestones and upcoming data

• X-TOLE2 phase 3 epilepsy study will unblind in the second half of 2025, enabling a potential NDA filing and U.S. commercial launch for focal onset seizures.

• The first phase 3 MDD trial will initiate by year-end, with two more in 2025; an investigator-sponsored MDD study will report results in the first half of 2025.

• Multiple IND filings and phase 1 initiations for preclinical Kv7 and NaV1.7 programs are expected in 2025.

• Regular annual updates and potential indication expansion for Kv7 assets are anticipated as the pipeline matures.

Clinical strategy and regulatory plans

• X-TOLE2 data will drive the NDA submission, targeted roughly six months after top-line results; X-TOLE3 is designed for ex-U.S. regulatory submissions.

• U.S. commercialization will be handled directly, while ex-U.S. markets will be accessed through partners.

• Phase 3 epilepsy studies maintain consistent design and high statistical power, aiming to replicate strong phase 2 results.

• Phase 3 MDD trials will use HAM-D17 as the primary endpoint, with increased severity criteria and 450 subjects to ensure robust statistical power.