Summary slide of ECFS presentation for VX-661/770 Phase II
Summary and Conclusions
●Reductions in sweat chloride were observed with both VX-661 alone and in combination with ivacaftor
●Improvements in pulmonary function (FEV1) were observed at all combination doses studied, and were greatest at the VX-661 100mg and 150mg dose levels
–At the end of the 28-day washout period, FEV1 returned to near baseline levels.
●VX-661 alone and in combination with ivacaftor was generally well tolerated
–The most common adverse events were respiratory in nature
–The frequency of serious adverse events was similar in the treatment and placebo groups
●These data support the concept of a combined corrector and potentiator approach for treatment of patients who are homozygous for the F508del-CFTR mutation
Please find the slide set from ECFS website and look at the charts, particularly Slides #9, 10, and 13. Slide #9 plots the sweat chloride changes. You will see the 661 dose dependence and 'washout' response of % predicted FEV1 in Slide #10. #13 slide gives the 'waterfall' plot. The #13 slide shows that 12 out of 31 treated with two highest doses achieved 10% or greater relative change in FEV1. This corresponds to 39%, while the group receiving placebo achieved the same in 3 out of 23 (=13%). The data are clearly more positive than the data from treatment with VX-809/770.