XEN007, A CaV2.1 Calcium Channel Modulator

XEN007 (active ingredient flunarizine) is a CNS-acting Cav2.1 and T-type calcium channel modulator that is being studied in treatment-resistant absence seizures in pediatric patients and potentially other neurological disorders.

Various development strategies for XEN007 are under consideration. We have entered into key exclusive licensing agreements in order to access regulatory files and drug product manufacturing, both of which may enable advanced clinical development of XEN007.

The FDA granted Orphan Drug Designation (ODD) and rare pediatric disease (RPD) designation for the treatment of childhood absence epilepsy (CAE) with XEN007. The FDA grants the RPD designation for serious or life-threatening diseases that primarily affect children 18 years old or younger and affect fewer than 200,000 people nationwide.

Clinical Development

An investigator-led Phase 2 proof-of-concept study is ongoing to examine the potential clinical efficacy, safety, and tolerability of XEN007 as an adjunctive treatment in pediatric patients diagnosed with treatment-resistant absence seizures, including CAE and juvenile absence epilepsy (JAE). Promising interim data collected from a small number of patients was presented at the virtual annual meeting of the American Epilepsy Society in December 2020. The lead investigator has expanded the study to include an additional site, which is currently screening patients, and is also evaluating the addition of other sites. Additional results from a larger data set are anticipated by the end of 2021, which will inform Xenon’s decision regarding the future development of XEN007.