Final results from Vertex VX-509 Phase 2a study on RA

Vertex Pharmaceuticals IncorporatedNov 6 2011

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the final results from a Phase 2a study of the investigational selective oral JAK3 inhibitor VX-509. The data showed substantial and statistically significant improvements in multiple measurements of rheumatoid arthritis (RA) activity. The 12-week study met its two primary endpoints, defined as a statistically significant improvement in the proportion of people who achieved at least a 20 percent improvement in the signs and symptoms of RA, also known as ACR₂₀, and a statistically significant improvement from baseline in Disease Activity Score 28 (DAS28). For the two highest dose groups of the study, statistically significant ACR₂₀, ACR₅₀, ACR₇₀, and DAS28 responses were also observed as compared to placebo, and more than one-third of people in these groups achieved clinical remission (DAS28 remission). Overall, the most frequently reported class of adverse event in the VX-509 and placebo arms was infections. The data from the study will be presented in a poster session on November 8 at the 2011 Annual Meeting for the American College of Rheumatology (ACR) in Chicago.

"People with RA experience frequent debilitating pain and discomfort in joints, which significantly impacts their lives and underscores the need for new RA medicines," said Roy Fleischmann, M.D., Clinical Professor of Medicine at the University of Texas Southwestern Medical Center and an investigator for the Phase 2 study of VX-509. "People in this study showed significant improvement in the signs and symptoms of RA after treatment with VX-509 - an encouraging step toward the future treatment of RA by selectively targeting an underlying mechanism of the disease."

Based on the Phase 2a data to be presented at ACR, a six-month Phase 2b study of VX-509 in RA is expected to begin in early 2012. This study, which will be run by Vertex, will evaluate once-daily (QD) and twice-daily (BID) doses of VX-509 in combination with methotrexate, a commonly prescribed disease-modifying antirheumatic drug (DMARD) for RA that is frequently used in combination with other RA medicines. The study is expected to enroll approximately 350 people with moderate to severe RA.