Vertex Pharmaceuticals announced positive results from the phase III STRIVE study of VX-770, an oral medicine that targets the defective protein that causes cystic fibrosis (CF). STRIVE was designed to evaluate people with a mutation in the CF gene known as G551D. This study showed profound improvements in lung function through week 24 and sustained through week 48 among those who received VX-770 (n=83) compared to those treated with placebo (n=78). Improvements in all key secondary endpoints were also observed through week 48 among those who received VX-770.

Data from the study showed a mean absolute improvement in lung function from baseline compared to placebo through week 24 of 10.6% among those treated with VX-770 (p

Highly statistically significant improvements in key secondary endpoints in this study were also reported through week 48. People who received VX-770 were 55% less likely to experience a pulmonary exacerbation (periods of worsening in signs and symptoms of the disease requiring treatment with antibiotics) and, on average, gained nearly seven pounds (3.1 kilograms) through 48 weeks. Significant reduction in the amount of salt in the sweat (sweat chloride) among people treated with VX-770 was evidenced. Increased sweat chloride is a diagnostic hallmark of CF and marker of CFTR protein dysfunction, which is the underlying molecular mechanism responsible for CF.