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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

Together, these pharmacokinetic and safety data support inclusion of VX-809 400mg (q12h) in combination with ivacaftor 250mg (q12h) in the Phase 3 program to evaluate the effect that higher exposures of VX-809 have on efficacy and safety.

The pattern of lung function response observed in Cohort 3 was similar to that observed in the 600mg QD dose group in Cohort 2, with a decline in FEV 1 during the VX-809 monotherapy dosing period followed by a statistically significant increase in FEV 1 during the VX-809 and ivacaftor combination dosing period. The within-group mean absolute improvement in FEV 1 observed during the combination-dosing period in Cohort 3 was 6.6 percentage points, compared to 6.1 percentage points for the 600mg QD dose group in Cohort 2.

Additional lung function results for Cohort 3 are provided below:

Cohort 3
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 (400mg q12h) + ivacaftor (250mg q12h)    

Within-Group

Absolute

Relative

   

-4.3 (p=0.04)

-6.3 (p=0.08)

   

+6.6 (p=0.01)

+8.8 (p=0.01)

   

+1.9 (p=0.57)

+2.5 (p=0.67)

Study in People with One Copy of the F508del Mutation

In addition to the Phase 3 studies in people with two copies of the F508del mutation, Vertex plans to conduct an 8-week exploratory Phase 2 study of VX-809 in combination with ivacaftor in people 12 and older with one copy (heterozygous) of the F508del mutation on one allele and a second mutation that is not expected to respond to either ivacaftor or VX-809 alone. This study is designed to provide additional safety and lung function data on the combination in heterozygous patients, and will evaluate the twice daily (q12h) combination of VX-809 (400mg) and ivacaftor (250mg).

VX-809 and ivacaftor were discovered as part of a collaboration with Cystic Fibrosis Foundation Therapeutics, Inc., the non-profit drug discovery and development affiliate of the Cystic Fibrosis Foundation.

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