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 4.5% and 4.8% 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.”