The opportunity to redefine the treatment of major diseases is what drives everyone at Vertex. Our development portfolio represents the fruit of these efforts. Spend some time here and look around. Click on the pipeline chart to get information on our development programs and links to selected published articles. Go further and see how, just maybe, disease challenges may be overcome with innovation.

VX-765
(Epilepsy)

VX-765 is a novel Caspase-1 inhibitor being investigated for the treatment of epilepsy.

VX-765 has been shown to inhibit acute seizures in preclinical models of acute epilepsy and has shown activity in preclinical models of chronic epilepsy that do not respond to standard anti-epileptic drugs.

Vertex recently initiated a Phase 2 proof-of-concept clinical trial of VX-765 in patients with epilepsy, which could result in interim clinical data being available as early as the second half of 2010. The Phase 2 trial for VX-765 is expected to enroll approximately 75 patients with treatment-resistant epilepsy.

The double-blind, randomized, placebo-controlled trial will evaluate the safety, tolerability and clinical activity of VX-765. Patients will receive six weeks of treatment with VX-765 following a six-week baseline period to monitor seizure frequency. The primary endpoints of the trial are safety and tolerability. The secondary endpoints will evaluate the clinical efficacy of VX-765 based on the percent reduction in seizure frequency and percent of patients with a 50 percent or greater reduction in seizure frequency, known as the responder-rate.