Vertex Announces Final Data from Phase 2 Combination Study of VX-809 and KALYDECO™ (ivacaftor) Showed Statistically Significant Improvements in Lung Function in People with Cystic Fibrosis Who Have Two Copies of the F508del Mutation

Vertex Pharmaceuticals Incorporated VRTX announced today final data from a Phase 2 study of VX-809 and KALYDECO™ (ivacaftor) that showed statistically significant improvements in lung function among adults with cystic fibrosis (CF) who have two copies (homozygous) of the most common mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, F508del. The study randomized homozygous F508del patients to three treatment groups that evaluated increasing doses of VX-809 (200mg, 400mg or 600mg; QD) alone for 28 days, followed by VX-809 in combination with KALYDECO (250mg; q12h) from Day 28 to 56. When patients received the two medicines in combination (Day 28 to 56), there was a statistically significant improvement in lung function (percent predicted forced expiratory volume in one second, FEV1) in each of the homozygous treatment groups compared to placebo. The greatest improvements in lung function were observed in patients who received 600mg of VX-809, the highest dose evaluated in this study, in combination with KALYDECO. These data support Vertex's plans to initiate a pivotal program in early 2013, which is expected to evaluate VX-809 (600mg) in combination with KALYDECO (250mg) in homozygous patients, pending discussions with regulatory agencies. Most adverse events observed during the 56-day study were mild to moderate in severity across all treatment groups and similar between treatment and placebo groups.