Xenon Pharmaceuticals outlines key milestone opportunities for 2023

Xenon Pharmaceuticals (XENE) outlined recent progress in its clinical programs and key milestones for 2023. Ian Mortimer, Xenon’s President and Chief Executive Officer, stated, "We enter 2023 in a strong position with increasing momentum in our XEN1101 Phase 3 program, which represents the most advanced potassium channel modulator in clinical development for multiple indications. With our X-TOLE2 study underway in focal onset seizures, we are excited to announce the initiation of our Phase 3 X-ACKT clinical trial in primary generalized tonic clonic seizures." Highlights and Anticipated Milestones: XEN1101 – XEN1101 for Focal Onset Seizures: Xenon has initiated its XEN1101 Phase 3 development program, which includes two identical Phase 3 clinical trials to be run in parallel, called X-TOLE2 and X-TOLE3, that are designed closely after the Phase 2b X-TOLE clinical trial. XEN1101 for Primary Generalized Tonic Clonic Seizures: Xenon has initiated a Phase 3 clinical trial, called X-ACKT, to support potential regulatory submissions in an additional epilepsy indication of primary generalized tonic clonic seizures. XEN1101 for Major Depressive Disorder: Based on promising pre-clinical data with XEN1101 and published clinical data generated using ezogabine, Xenon is evaluating the clinical efficacy, safety and tolerability of XEN1101 administered as monotherapy in approximately 150 patients with MDD in a Phase 2 clinical trial called X-NOVA. Topline results from the X-NOVA study are anticipated in the third quarter of this year. In addition, Xenon is collaborating with the Icahn School of Medicine at Mount Sinai to support an ongoing investigator-sponsored Phase 2 proof-of-concept, randomized, parallel-arm, placebo-controlled multi-site study of XEN1101 for the treatment of MDD in approximately 60 subjects. The primary objective of the study is to investigate the effect of XEN1101 on the brain reward circuit as measured by the change in bilateral ventral striatum activity as assessed by functional MRI. Additional Programs and Corporate Updates – XEN496: XEN496, a Kv7 potassium channel opener, is a proprietary pediatric formulation of the active ingredient ezogabine being developed for the treatment of KCNQ2 developmental and epileptic encephalopathy. Xenon anticipates that the EPIK study will be completed in 2024. NBI-921352: Xenon has an ongoing collaboration with Neurocrine Biosciences (NBIX) to develop treatments for epilepsy. Neurocrine Biosciences has an exclusive license to XEN901, now known as NBI-921352, a selective Nav1.6 sodium channel inhibitor. Neurocrine Biosciences is conducting a Phase 2 clinical trial evaluating NBI-921352 in adult patients with focal onset seizures, with data expected in the second half of this year. Cash and cash equivalents and marketable securities were $752.2 million as of September 30, 2022.