What clinical trials have you conducted in nonsense mutation cystic fibrosis (nmCF) and what were the results?
After successfully completing two Phase 1 clinical trials with healthy volunteers, we conducted three Phase 2 trials in patients with nmCF in the U.S., Israel, France, and Belgium. Each of the 77 patients (47 adults and 30 children) enrolled in these trials received ataluren in two repeated 28-day cycles, each consisting of 14 days on therapy and 14 days off therapy. In one cycle, participants received a lower dose regimen of ataluren, and in the other cycle, they received a higher dose of ataluren. One goal of the CF clinical program was to determine whether ataluren increases the production of cystic fibrosis transmembrane conductance regulator (CFTR) in cells. CF is caused by a lack of CFTR. Because ataluren is designed to permit the production of CFTR in patients with nmCF, we monitored CFTR levels. This was accomplished by swabbing a small number of cells from out of the nose of participants in the trial and analyzing the cells for CFTR protein using a microscope. Lack of CFTR cau
