Monday, Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) released results from its once-daily vanzacaftor/tezacaftor/deutivacaftor (vanza triple) program for cystic fibrosis (CF), a progressive, multi-organ disease caused by dysfunction of the CFTR protein.
In SKYLINE 102 and SKYLINE 103, the primary endpoint of absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1, measurement of lung function) through week 24 was met and showed that treatment with vanza triple was non-inferior to treatment with Trikafta.
Concurrently, the company released its fourth quarter 2023 financial results.
Goldman Sachs analysts acknowledge the debate on the clinical meaningfulness of sweat chloride, as physicians do not routinely measure it; it is the direct marker of CFTR protein function (which underlies disease pathophysiology).
The analyst reiterates the Buy rating with a price target ...