VX-828 is a corrector, a type of CFTR modulator designed to fix the defective CFTR protein so that it can move to the proper place on the cell surface.

It is being studied in combination with tezacaftor, another CFTR corrector, and deutivacaftor, a potentiator that facilitates the opening of the chloride channel on the cell surface to allow chloride and sodium (salt) to move in and out of the cell.

Status

A Phase 1 study to test safety and tolerability of VX-828 in combination with tezacaftor and deutivacaftor is currently underway. The study is for adults with CF who have one copy of the F508del mutation and one copy of a minimal function mutation that is not responsive to ELX/TEZ/IVA (Trikafta) therapy.

Sponsor

This program is sponsored by Vertex Pharmaceuticals. It is being conducted within the Therapeutics Development Network.