Lumacaftor / VX-809

An excellent roundup of in development CFTR modulators, covering Ivacaftor (Kalydeco), Lumacaftor (VX-809), VX-661, Ataluren (PTC124), 4PBA, VRT-532, and N6022. Full text is here – http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4103577/

Vertex Pharmaceuticals Inc. announced results from a Phase 2 clinical trial of the cystic fibrosis drug ivacaftor (Kalydeco™) in combination with another potential therapy, lumacaftor, in people with one copy of the F508del mutation, ages 18 and older. In the … Continued

Two 24-Week Phase 3 Studies of Lumacaftor in Combination with Ivacaftor Met Primary Endpoint with Statistically Significant Improvements in Lung Function (FEV1) in People with Cystic Fibrosis who have Two Copies of the F508del Mutation Combination of lumacaftor and ivacaftor … Continued

BACKGROUND: The most common cystic fibrosis-associated mutation, the deletion of phenylalanine 508 (F508del), results in channels with poor membrane expression and impaired function. VX-770, a clinically approved drug for treatment of CF patients carrying the G551D mutation, and VX-809, a … Continued

Enrollment complete for TRAFFIC and TRANSPORT Phase 3 studies of lumacaftor (VX-809) in combination with ivacaftor for people with two copies of the F508del mutation (homozygous); data expected in mid-2014 12-week Phase 2 study of VX-661 in combination with ivacaftor … Continued

Vertex presented data in two talks at the European Cystic Fibrosis Society Conference last week. “VX-661, an investigational CFTR corrector, in combination with ivacaftor, a CFTR potentiator, in patients with CF and homozygous for the F508Del-CFTR mutation: interim analysis.” and … Continued

Vertex Pharmaceuticals announced the design of two phase 3 studies for its combination therapy to treat the most common form of cystic fibrosis, df508. The studies will each run for six months, so results could be ready as early as the … Continued

Excellent videos from CFUnite’s conference on Kalydeco and the future