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Vertex cystic fibrosis drug combination passes phase 3 trials

Vertex Pharmaceuticals Inc.'s tezacaftor/Kalydeco combination therapy met its primary endpoints in two phase 3 trials in patients with cystic fibrosis.

In the EVOLVE study, the drug combination showed statistically significant improvements in lung function by 4 percentage points versus placebo. The EXPAND study showed that the drug combination improved lung function by 6.8 percentage points versus placebo.

The EVOLVE study evaluated the combination treatment in people who have two copies of the F508del mutation, while the EXPAND study evaluated the treatment in people who have one mutation that results in residual cystic fibrosis transmembrane conductance regulator function and one F508del mutation.

Vertex said the treatment also showed statistically significant improvements in multiple key secondary endpoints, including a 35% reduction in the annualized rate of pulmonary exacerbations versus placebo, and an absolute change in the Cystic Fibrosis Questionnaire-Revised respiratory domain score from baseline to average versus placebo.

Rates of discontinuations due to adverse events were low and similar between placebo and treatment groups, while rates of respiratory adverse events were similar between placebo and treatment groups in both trials.

Vertex plans to submit a new drug application to the U.S. FDA and a marketing authorization application to EMA in the third quarter of 2017 on the basis of these results.