XEN007 for Orphan Neurological Indications

XEN007, A CaV2.1 Calcium Channel Inhibitor

XEN007 (active ingredient flunarizine) is a CNS-acting calcium channel modulator that modulates Cav2.1 and T-type calcium channels. Other reported mechanisms include dopamine, histamine and serotonin inhibition. Flunarizine is available in certain countries outside of the United States, and has been reported to have clinical benefit in treating migraine and other neurological disorders, including hemiplegic migraine, or HM, alternating hemiplegia of childhood, or AHC, vertigo, and as adjunctive treatment in certain epilepsies.

The FDA has granted a rare pediatric disease, or RPD, designation for the treatment of AHC with XEN007. We previously received ODD from the FDA for XEN007 for the treatment of both AHC and HM. In addition, 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. Various development strategies for XEN007 are under consideration, including the support of at least one Phase 2 (or later stage) clinical trial in an orphan neurological indication, with initiation anticipated in 2019.