Clinical Trials

Date: 2015-10-10

Type of information: Initiation of development program

phase: 1

Announcement: initiation of development program

Company: Vertex Pharmaceuticals (USA - MA)

Product: VX-440

Action mechanism: CFTR potentiator/cystic fibrosis transmembrane regulator (CFTR) protein modulator

Disease: cystic fibrosis

Therapeutic area: Rare diseases - Genetic diseases

Country:

Trial details:

Latest news:

• • On October 10, 2015, Vertex Pharmaceuticals announced that it is advancing two next-generation correctors from its research program into clinical development. Known as VX-152 and VX-440, these next-generation correctors will be evaluated alone and in combination with VX-661/ivacaftor in Phase 1 studies in healthy volunteers beginning in November 2015 . Pending results of these studies, Vertex plans to initiate Phase 2 studies in people with CF evaluating VX-440 or VX-152 in combination with VX-661/ivacaftor in the second half of 2016. The studies of a triple combination (VX-152/VX-661/ivacaftor and VX-440/VX-661/ivacaftor) planned for the second half of 2016 are expected to enroll people with CF who have two copies of the F508del mutation and people who have one copy of the F508del mutation and a second mutation that results in minimal CFTR function. VX-152 and VX-440 are designed to further improve processing and trafficking of the CFTR protein to the cell surface, beyond that observed with a single corrector combined with ivacaftor, which may enable increased CFTR chloride transport, a measure of the function of the CFTR protein at the cell surface.
• In human bronchial epithelial (HBE) cells with two copies of the F508del mutation and in HBE cells with one copy of the F508del mutation and one copy of a mutation known to result in minimal CFTR function, the triple combinations (VX-152/VX-661/ivacaftor and VX-440/VX-661/ivacaftor) resulted in chloride transport (percent of normal) that was approximately three-fold greater than the use of the lumacaftor/ivacaftor combination in these cells. A significant increase in cilia beat frequency was also observed with triple combination therapy as compared to the use of the lumacaftor/ivacaftor combination in these cells. These in vitro data suggest that a triple combination of a next-generation corrector with VX-661/ivacaftor may improve CFTR function in cells with two copies of the F508del mutation and cells with one copy of the F508del mutation and one copy of a mutation known to result in minimal CFTR function.

Is general: Yes