Vertex Pharmaceuticals Inc today announced positive data from Phase 1 and Phase 2 studies of three different triple combination regimens in people with cystic fibrosis (CF) who have one F508del mutation and one minimal function mutation (F508del/Min).
These are the first data to demonstrate the potential to treat the underlying cause of CF in these patients, who have a severe and difficult-to-treat type of the disease.
Data from the Phase 2 studies in these patients showed mean absolute improvements in percent predicted FEV1 of 9.7 and 12.0 percentage points from baseline for the triple combination regimens with VX-152 (200mg q12h) or VX-440 (600mg q12h), respectively.
Initial data from a Phase 1 study showed a mean absolute improvement in ppFEV1 of 9.6 percentage points from baseline for the triple combination regimen of VX-659, tezacaftor and ivacaftor in people with one F508del mutation and one minimal function mutation.
The company also announced today initial data showing improvements in mean absolute ppFEV1 of 7.3 and 9.5 percentage points when VX-152 or VX-440 was added in people with two copies of the F508del mutation, who were already receiving tezacaftor and ivacaftor.