CAMBRIDGE (MASSACHUSETTS) (dpa-AFX) - Vertex Pharmaceuticals Inc. (VRTX) announced final data from a Phase 2 study of VX-809 and KALYDECO (ivacaftor) that exhibited statistically significant improvements in lung function among adults with cystic fibrosis who have two copies of the most common mutation in the cystic fibrosis transmembrane conductance regulator gene, F508del.
The study randomized homozygous F508del patients to three treatment groups that assessed 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, noted the company.
The greatest improvements in lung function were observed in patients who received 600mg of VX-809, the highest dose evaluated in the study, in combination with KALYDECO. Vertex stated that these data support its 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.
Besides, the study included an exploratory treatment group that looked at a subset of heterozygous patients who have one copy of the F508del mutation and a second mutation that is not expected to respond to KALYDECO dosed as monotherapy. This group of patients received VX-809 (600mg) and KALYDECO on the same dosing schedule as homozygous patients. Heterozygous patients who were treated with the combination experienced a mean absolute improvement in lung function compared to placebo from day 28 to 56.
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