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Vertex Announces Initiation Of Pivotal Phase 3 Program Of VX-809 In Combination With Ivacaftor For The Treatment Of People With Cystic Fibrosis Who Have Two Copies Of The F508del Mutation

Cystic fibrosis is a rare, life-shortening genetic disease for which there is no cure. Approximately 70,000 people worldwide have CF, including 30,000 in the United States and 35,000 in Europe. Globally, nearly half of those with CF have two copies of the F508del mutation.

About the Phase 3 TRAFFIC and TRANSPORT Studies

Vertex plans to conduct two 24-week, randomized, double-blind, placebo-controlled Phase 3 studies of VX-809 in combination with ivacaftor. TRAFFIC and TRANSPORT will each enroll approximately 500 people with CF ages 12 and older who have two copies of the F508del mutation in the CFTR gene, for a total of 1,000 patients. The studies have the same design and together will be conducted at approximately 200 clinical trial sites in North America, Europe and Australia.

The primary endpoint of each study is relative improvement in lung function (percent predicted FEV 1) through 24 weeks of treatment, compared to placebo. Safety and tolerability will also be assessed through 24 weeks. Key secondary endpoints through 24 weeks include absolute improvement in FEV 1, change in body mass index (BMI) or weight gain, number of pulmonary exacerbations and improvements in patient-reported outcomes as measured by the CF Questionnaire Revised (CFQ-R), among others.

Each study will include two combination treatment groups and one placebo group. The treatment groups will evaluate two regimens of VX-809 (600mg QD or 400mg q12h) in combination with ivacaftor (250mg q12h). Fixed-dose tablets that contain both VX-809 and ivacaftor, or placebo, will be used in both studies. Vertex plans to follow the initial 24-week treatment period with a separate rollover double-blind extension study where all eligible patients, including those who received placebo, will receive one of the combination regimens for up to an additional 96 weeks. The design of the studies is as follows:
Treatment Group         24-Week Dosing Regimen
Group 1 (n=167)         VX-809 (600mg QD) + ivacaftor (250mg q12h)
Group 2 (n=167)         VX-809 (400mg q12h) + ivacaftor (250mg q12h)
Group 3 (n=167)         Placebo + Placebo

About the Study in Patients Ages 6 to 11

Vertex also plans to conduct a study of VX-809 in combination with ivacaftor in children with CF ages 6 to 11 who have two copies of the F508del mutation. The study will evaluate the pharmacokinetics and safety of the combination for up to 24 weeks. Vertex expects to use the data from this study, along with data from TRAFFIC and TRANSPORT, for registration of the combination in the United States in children ages 6 to 11, following registration in patients ages 12 and older. In Europe, the company is in discussions with regulatory agencies regarding patients in this age group.

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