Vertex Pharma Announces Data From 12-Week Phase 2 Safety Study Of VX-661

(RTTNews) - Vertex Pharmaceuticals Inc. (VRTX) announced data from a 12-week Phase 2 study evaluating VX-661 in combination with ivacaftor in 39 people with CF ages 18 and older who have two copies of the F508del mutation.

The study evaluated two doses of VX-661 (100 mg once daily or 50 mg every 12 hours) in combination with ivacaftor (150 mg every 12 hours).

The primary endpoint of the study was safety. The study showed that the combination regimen was generally well tolerated, and all patients completed 12 weeks of treatment.

The most common adverse events were pulmonary exacerbation, which occurred in 38 percent of all patients who received VX-661 and 44 percent of those who received placebo, and cough, which occurred in 33 percent of all patients who received VX-661 and 39 percent of those who received placebo.

Secondary endpoints evaluated the effect of the combination on lung function (percent predicted forced expiratory volume in one second; ppFEV1), and the mean within-group absolute improvement from baseline in ppFEV1 for those who received 100 mg of VX-661 in combination with ivacaftor (n=15) was 4.4 (p=0.009) and 3.0 (p=0.026) percentage points at week 4 and through 12 weeks of treatment, respectively.

Consistent with prior Phase 2 studies that evaluated 4 weeks of treatment with VX-661 in combination with ivacaftor, this study showed a rapid improvement in lung function within four weeks of treatment, and after patients completed treatment, lung function returned to baseline.

These safety and efficacy data, together with other data from multiple previously completed Phase 2 studies of VX-661, support Vertex's ongoing Phase 3 program of VX-661 in combination with ivacaftor. The Phase 3 program is evaluating VX-661 (100 mg once daily) in combination with ivacaftor (150 mg every 12 hours) and consists of four Phase 3 studies, including a study in people with two copies of the F508del mutation that began enrollment in February.

The company noted that the other three studies will enroll people with CF who have one copy of the F508del mutation and a second mutation that is either a gating mutation, residual function mutation or a mutation that results in minimal CFTR function.