Xenon Pharmaceuticals (XENE) investor relations material

Lead program and clinical data

• Azetukalner, a potassium channel modulator, showed best-in-class efficacy in phase III studies for focal onset seizures, with rapid onset and no need for titration or drug-drug interaction adjustments.

• Up to 40% of long-term patients achieved 12 months of seizure freedom, significantly improving quality of life in a highly refractory population.

• NDA filing for epilepsy is planned for Q3, with a commercial launch targeted for the second half of 2027.

• Additional phase III analyses and five-year data from the open-label extension will be presented at the American Epilepsy Society meeting.

• X-TOLE3 phase III trial is focused on EU and Japan approval, with non-Japanese enrollment completing this year and Japanese sites starting soon.

Pipeline expansion and milestones

• Psychiatry program includes two phase III MDD trials (X-NOVA2, X-NOVA3) and one in bipolar depression; first MDD readout expected in H1 2027.

• Earlier-stage pain portfolio includes Kv7 (XEN1120) and NaV1.7 (XEN1701) molecules in phase I, with phase II proof-of-concept studies planned after phase I completion this year.

• NaV1.1 program for Dravet syndrome is in toxicology, aiming for clinical entry next year.

• Strong balance sheet with $1.3 billion in cash, providing runway into 2029 and supporting multiple upcoming milestones.

Regulatory and commercial strategy

• Standard FDA review is expected for azetukalner; breakthrough designation is unlikely, but therapy designation and priority review are possible.

• DEA scheduling will add about three months post-approval, but is not expected to impact commercial uptake.

• Commercial launch will target both epileptologists and general neurologists, leveraging the drug’s ease of use and differentiated profile.

• Sales force will start with about 75 reps, supported by non-personal promotion and data-driven distribution strategies.

• Pricing is expected to be above the current branded competitor, XCOPRI, reflecting a strong value proposition.