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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

Phase 2 Data Supporting Phase 3 Trial Design

The Phase 3 studies announced today are supported by data from a Phase 2 study of VX-809 in combination with ivacaftor. The two combination dosing regimens selected for evaluation in Phase 3 were evaluated in Cohorts 2 and 3 of the Phase 2 study.

Cohort 2: As previously reported, the once-daily (QD) 600mg dose of VX-809 in combination with ivacaftor (250mg q12h) was evaluated in 21 patients in the second part (Cohort 2) of the Phase 2 study and resulted in statistically significant improvements in lung function (within group and versus placebo) during the combination dosing period, as noted below:

Cohort 2
Mean Absolute and Relative Changes in Percent Predicted FEV 1     Day 0 – 28; VX-809 Alone     Day 28 – 56; VX-809 + ivacaftor     Day 0 - 56
VX-809 (600mg QD) + ivacaftor (250mg q12h)    

Within-Group

Absolute

Relative

   

-2.9 (p=0.07)

-3.5 (p=0.13)

   

+6.1 (p<0.001)

+9.7 (p<0.001)

   

+3.4 (p=0.03)

+5.3 (p=0.02)

   

Versus Placebo

Absolute

Relative

   

-2.0 (p=0.36)

-3.9 (p=0.21)

   

+8.6 (p<0.001)

+12.8 (p<0.001)

   

+6.7 (p=0.002)

+9.2 (p=0.004)

Cohort 3: Vertex also evaluated a 400mg twice-daily (q12h) dosing regimen of VX-809 in combination with ivacaftor in a third cohort of patients in the Phase 2 study. Cohort 3 evaluated 11 patients who received VX-809 (400mg q12h) for 28 days followed by VX-809 (400mg q12h) in combination with ivacaftor (250mg q12h) for 28 days. This cohort was designed to evaluate safety and pharmacokinetics of the 400mg q12h dose of VX-809 to support inclusion of this dose in the Phase 3 program. Cohort 3 also included the randomization of four patients to placebo to allow for a blinded safety assessment. Three patients completed treatment in the placebo group. A pharmacokinetic model suggested that 400mg dosing every 12 hours (q12h) of VX-809 would provide a higher total exposure (AUC; area under the curve) compared to 600mg QD dosing, and data from Cohort 3 were consistent with this model.

Safety results from the 400mg (q12h) dose group were similar to that of the 600mg (QD) dose group. In both dose groups, VX-809 was generally well-tolerated alone and in combination with ivacaftor. The most common adverse events in both groups were respiratory in nature. In Cohort 3, one patient in the treatment group discontinued treatment because of a pulmonary adverse event.

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