Phase 3 Study of VX-770 for Cystic Fibrosis

Vertex Pharmaceuticals announced final results from its Phase 3 STRIVE study of VX-770 for the treatment of cystic fibrosis (CF). The results of STRIVE showed a mean absolute improvement in FEV1 of 10.6% through Week 24 (primary study endpoint) and 10.5% through Week 48 (secondary study endpoint) among patients (n=83) treated with VX-770 vs. placebo. The mean relative improvement from baseline in FEV1 among patients (n=78) treated with VX-770 compared to placebo was 16.9% through Week 48.

Significant improvements in key secondary endpoints in this study were also reported through Week 48. Compared to placebo, patients who received VX-770 experienced a greater average weight gain (7lbs vs. 3.9lbs) and were 55% less likely to experience a pulmonary exacerbation. At Week 2, patients treated with VX-770 experienced an average reduction in sweat chloride of approximately 45 mmol/L which maintained through Week 48. Patients treated with the placebo maintained baseline sweat chloride levels of approximately 100 mmol/L through Week 48. The mean absolute improvement in sweat chloride among those treated with VX-770 was -48.1 mmol/L through Week 48 (P<0.0001).

VX-770 is an oral tablet that directly targets the defective CFTR protein known to cause CF. VX-770 increases the function of defective CFTR protein by increasing the gating activity, or ability to transport ions across the cell membrane, of CFTR once it reaches the cell surface.

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