CAMBRIDGE, Mass.--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the initiation of a Phase 2a clinical trial for VX-809, an investigational Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) corrector that targets the defective CFTR protein that causes cystic fibrosis (CF). The trial is designed primarily to evaluate the safety and tolerability of multiple doses of VX-809 in patients with CF. In addition to assessing safety, the trial will also evaluate the potential effect of VX-809 on measures of CFTR function. The trial is expected to enroll approximately 90 patients homozygous for the F508del mutation in the CFTR gene, the most common mutation in CF patients. VX-809 was discovered as part of a collaboration with Cystic Fibrosis Foundation Therapeutics Inc. (CFFT) to develop novel CFTR correctors. CFFT is the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation. Vertex retains worldwide rights to develop and commercialize VX-809 and its CFTR potentiator VX-770.
