Xenon Pharmaceuticals (XENE) TD Cowen 6th Annual Novel Mechanisms in Neuropsychiatry Summit summary

Key program updates

• Filed first NDA for azetukalner in focal-onset seizures, supported by robust phase IIb and II studies showing strong efficacy and safety, with over 1,500 patient-years of exposure and long-term data up to 60 months to be updated at the American Epilepsy Society meeting in December 2026.

• Commercial preparations include hiring a Chief Commercial Officer and building a leadership team, with expectations for broad adoption due to simple dosing (once daily, no titration needed) and a favorable safety profile.

• Four dose strengths (10, 15, 20, 25mg) filed for azetukalner, with 15mg expected as the likely starting dose due to its strong tolerability and efficacy.

• NDA review expected to follow a standard 12-month timeline, with DEA scheduling to add about three months post-approval.

• Commercial launch anticipated to be gradual but with potential for broader reach than previous ASM launches, targeting both specialists and general neurologists.

Psychiatry program developments

• Phase III psychiatry program for azetukalner paused to improve tolerability via dose escalation after emerging safety profile in MDD and BPD mirrored epilepsy studies.

• X-NOVA2 phase III study in MDD over 80% enrolled; unblinded efficacy and safety data expected in Q1 2027 to inform benefit-risk assessment and future program adjustments.

• Dose escalation strategies being developed, leveraging clinical pharmacology and open-label epilepsy data to enhance tolerability for psychiatric populations.

• No changes to the epilepsy program; continuous updates planned as psychiatry studies are adjusted and restarted.

Early-stage and pain portfolio

• Three molecules advanced into phase I over the past 15 months, with lead pain programs (Nav1.7 inhibitor XEN1701 and KV7 drug XEN1120) set for phase II proof-of-concept studies in 2027.

• Nav1.7 program aims to achieve sufficient receptor occupancy for analgesic effect, with phase I data supporting safety and efficacy targets.

• KV7 program shows strong preclinical efficacy; phase I achieved target exposures, with more data to be shared before phase II initiation.

• Acute pain phase II studies (e.g., bunionectomy, abdominoplasty) expected to yield rapid readouts.