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 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 is conducting 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 in 2011.