The phase 2 VX661/Kalydeco results have just been released:
Statistically significant improvements in lung function were seen in this study with CFs who have two copies of F508del
"Treatment with combination of VX-661 and ivacaftor for 28 days in two highest dose groups resulted in mean relative increases in lung function (percent predicted FEV1) of 9.0% (p=0.01) and 7.5% (p=0.02) versus placebo."
The absolute improvements were 4.8% and 4.5% versus placebo (p=0.01). A smaller, not statistically significant improvement was seen with VX661 alone.
"There were statistically significant mean absolute decreases in sweat chloride, both within-group and versus placebo, across the combination and monotherapy groups. These changes were generally modest and were variable across the dose groups."
"Vertex has advanced three correctors from research into development - VX-809, VX-661 and VX-983. VX-809 is Vertex's lead corrector and is currently being evaluated in combination with ivacaftor as part of two ongoing Phase 3 studies expected to enroll a total of approximately 1,000 people ages 12 and older with two copies of the F508del mutation. Vertex expects to obtain 24-week safety and efficacy data from these studies and to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in 2014, pending study results.
VX-661 is Vertex's second corrector to enter clinical development. Vertex plans to conduct additional studies of VX-661 to further evaluate its potential for late-stage development, pending regulatory discussions. VX-983 is the third corrector to enter clinical development and is currently being evaluated as part of a Phase 1 multiple-ascending-dose study in healthy volunteers. In the second half of 2013, Vertex plans to begin a 28-day study of VX-983 in combination with ivacaftor in people with two copies of the F508del CFTR mutation.
In addition to Vertex's development activities focused on combinations of a corrector with ivacaftor, the company has an active research program that has identified next-generation correctors that could be used as part of future combination regimens for people with CF."
http://investors.vrtx.com/releasedetail.cfm?ReleaseID=757597
Statistically significant improvements in lung function were seen in this study with CFs who have two copies of F508del
"Treatment with combination of VX-661 and ivacaftor for 28 days in two highest dose groups resulted in mean relative increases in lung function (percent predicted FEV1) of 9.0% (p=0.01) and 7.5% (p=0.02) versus placebo."
The absolute improvements were 4.8% and 4.5% versus placebo (p=0.01). A smaller, not statistically significant improvement was seen with VX661 alone.
"There were statistically significant mean absolute decreases in sweat chloride, both within-group and versus placebo, across the combination and monotherapy groups. These changes were generally modest and were variable across the dose groups."
"Vertex has advanced three correctors from research into development - VX-809, VX-661 and VX-983. VX-809 is Vertex's lead corrector and is currently being evaluated in combination with ivacaftor as part of two ongoing Phase 3 studies expected to enroll a total of approximately 1,000 people ages 12 and older with two copies of the F508del mutation. Vertex expects to obtain 24-week safety and efficacy data from these studies and to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in 2014, pending study results.
VX-661 is Vertex's second corrector to enter clinical development. Vertex plans to conduct additional studies of VX-661 to further evaluate its potential for late-stage development, pending regulatory discussions. VX-983 is the third corrector to enter clinical development and is currently being evaluated as part of a Phase 1 multiple-ascending-dose study in healthy volunteers. In the second half of 2013, Vertex plans to begin a 28-day study of VX-983 in combination with ivacaftor in people with two copies of the F508del CFTR mutation.
In addition to Vertex's development activities focused on combinations of a corrector with ivacaftor, the company has an active research program that has identified next-generation correctors that could be used as part of future combination regimens for people with CF."
http://investors.vrtx.com/releasedetail.cfm?ReleaseID=757597