Phase 2 Study of VX-765 for Epilepsy

Vertex announced results from its completed Phase 2 study of VX-765 for treatment-resistant epilepsy. This double-blind, randomized, placebo-controlled Phase 2a study of VX-765 enrolled 60 patients with treatment-resistant partial onset epilepsy who did not benefit from the use of at least two currently available medicines. The primary efficacy endpoint was safety and tolerability, and results from the study showed a similar safety profile for VX-765 as compared with placebo. Secondary endpoints and additional analyses evaluated the clinical activity of VX-765, and results support the initiation of a larger and longer duration Phase 2b study of VX-765 in people with treatment-resistant epilepsy.

VX-765 is designed to inhibit Caspase-1, an enzyme involved in the production of interleukin-1 (IL-1) beta and linked to a wide range of immune and inflammatory responses. VX-765 has been shown to inhibit acute partial seizures in preclinical models and has shown activity in preclinical models of chronic partial epilepsy that do not respond to currently available medicines for epilepsy. 

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