On February 23, 2011, results were announced for one of the three studies involved in the potential cystic fibrosis drug compound VX-770 clinical trials – the Phase 2 DISCOVER study, according to a press release by the potential drug’s manufacturer, Vertex Pharmaceuticals Incorporated (“Vertex”). The potential drug VX-770 is a next generation medication designed to treat those affected by the lung disease, as it is meant to act upon a defective protein in cystic fibrosis patients, and opens a chloride channel in that cell, thereby allowing for the proper flow of salt and fluid in and out of the impaired CF protein.
DISCOVER, which began in August 2009, enrolled 140 patients with Cystic Fibrosis who had two copies of the CF gene mutation F508del, which prevents the CFTR protein, the underlying mechanism responsible for cystic fibrosis, from moving to its proper location at the cell surface. Participants in this study received either the VX-770 compound, or placebo. While the DISCOVER study was designed primarily as a safety study, other purposes of this 16 week study involved following changes in the participants’ lung function and sweat chloride output.
Although adverse events (cough, nausea, rash, and contact dermatitis) occurred more frequently for VX-770 patients versus those taking placebo, the increase was not serious enough to discontinue the VX-770 study. With regard to findings of lung function improvement of patients during this study, the benefits were not statistically significant. However, those involved in DISCOVER witnessed a small reduction of sweat chloride produced. Although this reduction was minor, it was statistically significant. A reduction in sweat chloride is important, as this suggests an improvement in the dysfunctional CFTR protein.
Results for two other legs of the VX-770 clinical study, the Phase 3 STRIVE and Phase 3 ENVISION trials, were announced by Vertex in June 2011.
Source:
Phase 3 Study of VX-770 Showed Profound and Sustained Improvements in Lung Function (FEV1) and Other Measures of Disease Among People With a Specific Type of Cystic Fibrosis , Vertex Pharmaceuticals Incorporated