Xenon Pharmaceuticals (XENE) 2026 Bloom Burton & Co. Healthcare Investor Conference summary

Strategic and financial overview

• Raised capital to reach $1.4 billion in pro forma cash, supporting operations through 2029 and first commercialization of lead asset.

• Strategic goal is to become a fully integrated neurology company, with first commercial launch expected in the next few years.

• Commercial infrastructure is being built, with leadership roles filled and teams preparing for launch in the U.S.

Lead asset: Azetukalner in epilepsy

• Azetukalner, a Kv7 potassium channel modulator, showed best-in-class efficacy in phase III X-TOLE2 for focal onset seizures.

• Demonstrated rapid onset of efficacy, with significant seizure reduction from week one and deepening response over time.

• Long-term open-label data show up to 90% seizure reduction after four years, with 40% of patients achieving 12 months of seizure freedom.

• Safety profile is consistent and well-tolerated, with common CNS-related adverse events and few serious events.

• NDA filing for focal onset seizures planned for Q3, with phase III ongoing in primary generalized tonic-clonic seizures.

Expansion into psychiatry

• Azetukalner is being developed for major depressive disorder and bipolar depression, leveraging its novel mechanism.

• Phase II data showed dose-dependent improvements in depression and anhedonia, with rapid onset and favorable safety profile.

• Three phase III trials are ongoing in psychiatry, with first readout expected in the first half of 2027.