Vertex reports interim data from VX-809 and KALYDECO combination Phase 2 study for cystic fibrosis

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today an interim analysis of data from an ongoing Phase 2 study of VX-809 and KALYDECO™ (ivacaftor) that showed significant improvements in lung function (FEV₁) 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. A planned interim analysis was conducted after approximately half of the study patients had completed 56 days of treatment. Today's results are based on data from 37 homozygous F508del patients who completed treatment in the 56-day study and 11 patients with one or two copies of the F508del mutation who received placebo. There was a statistically significant improvement in lung function (absolute change in percent predicted FEV₁) across the combined treatment groups relative to baseline compared to placebo.

“While we still eagerly await the results from the remainder of the trial, we are definitely encouraged that this interim analysis showed a significant improvement in lung function in people with two copies of the F508del mutation who received VX-809 and KALYDECO.”

The study is ongoing and complete data, including statistical analyses for all patient groups, will be available in mid-2012. Vertex plans to start a pivotal study of VX-809 and KALYDECO in people with two copies of the F508del mutation, pending final study results and discussions with regulatory agencies. 

Cystic fibrosis is a rare, life threatening genetic disease affecting approximately 30,000 people in the United States and 70,000 people worldwide. Nearly half of those with CF are estimated to have two copies of the F508del mutation.

"For the past 14 years, our teams have focused on learning about the underlying cause of cystic fibrosis so we can develop new medicines to help as many patients as possible. Today we believe we're one step closer to this goal," said Chris Wright, M.D., Ph.D., Vertex's Senior Vice President, Global Medicines Development and Medical Affairs. "People with two copies of the F508del mutation have one of the most severe forms of cystic fibrosis. In these patients, the combination of VX-809 and KALYDECO led to significant improvements in lung function that exceeded our expectations. We look forward to beginning discussions with regulatory agencies later this year when we have full data from the study, with the goal of moving forward with a pivotal study as quickly as possible."

"Lung function is the single most important marker of disease progression for people with cystic fibrosis, and improvement in lung function is the goal of every new CF therapy," said Michael P. Boyle, M.D, FCCP, Associate Professor of Medicine, Director of the Johns Hopkins Adult Cystic Fibrosis Center, and lead investigator for this study. "While we still eagerly await the results from the remainder of the trial, we are definitely encouraged that this interim analysis showed a significant improvement in lung function in people with two copies of the F508del mutation who received VX-809 and KALYDECO."