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VX-509
(Inflammatory Diseases)

VX-509 is a novel Janus kinase 3 (JAK3) inhibitor being investigated for the potential treatment of multiple immune-mediated inflammatory diseases.

In in vitro studies, VX-509 has been shown to be greater than 1000-fold more selective for JAK3 compared to non-JAK kinases and approximately 25- to 150-fold more selective for JAK3 compared to other JAK isotypes in cell-based assays.

Results of a Phase 1, 14-day dose-ranging study of VX-509 in healthy volunteers completed in the first quarter suggested a promising safety profile. In this study, VX-509 showed a dose-dependent and reversible reduction in PSTAT-5, a specific biomarker of JAK3 activity, and a high degree of selectivity for JAK3 over JAK2, consistent with observations from previous in vitro studies.

Vertex recently initiated a Phase 2 proof-of-concept clinical trial of VX-509, which is expected to enroll approximately 200 patients with moderate to severe rheumatoid arthritis (RA). The company expects to obtain interim clinical data, including measurements of safety, tolerability and clinical activity, as early as the second half of 2010.

The double-blind, randomized, placebo-controlled trial will evaluate the safety, tolerability and clinical activity of four doses of VX-509. Patients will receive 12 weeks of treatment with VX-509 dosed twice daily compared to placebo.

The primary endpoints of the trial are to evaluate safety and to measure the improvement in clinical signs and symptoms of RA in patients after 12 weeks of treatment. Efficacy assessments will include the American College of Rheumatology criteria (ACR20, ACR50 and ACR70) for defining clinical improvement in RA patients. ACR20, ACR50 and ACR70 are standardized measures of the number of patients who achieve at least a 20, 50 or 70 percent improvement, respectively, in ACR-specified measures of RA activity. The trial will also utilize disease activity score (DAS) and European League Against Rheumatism (EULAR) response criteria as additional efficacy assessments.