Vertex Announces Phase 2 Results of Combination Treatment in People with One Copy of the F508del CF Mutation

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

Positive results from Vertex F508del Phase 3 Study with Lumacaftor in Combination with Ivacaftor

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

Combined effects of VX-770 and VX-809 on several functional abnormalities of F508del-CFTR channels

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

Proteostasis Therapeutics Announces Extension of Funded Collaboration with Cystic Fibrosis Foundation Therapeutics for Novel Therapies Targeting Most Common CF Mutation

Second stage of collaboration aims to identify drug development candidate with a goal of IND filing in 2015 Preclinical results demonstrate ability of compounds to increase functional activity in ΔF508 human bronchial epithelial cells  Proteostasis Therapeutics, Inc., a company developing … Continued

Proteostasis Therapeutics Presents Promising Preclinical Data at the 27th Annual North American Cystic Fibrosis Conference

Proteostasis Therapeutics, Inc., today announced the presentation of data on the Company’s lead preclinical candidates and their potential for use as combination therapies for cystic fibrosis (CF) at the 27th Annual North American Cystic Fibrosis Conference in Salt Lake City, … Continued