Denufosol tetrasodium: Phase III data

In the double-blind, international Phase III TIGER-2 trial in 466 CF patients, denufosol missed the primary endpoint of significantly improving FEV1 at 48 weeks vs. placebo (40 vs. 32 mL, p=0.742). Denufosol also missed the secondary endpoints of rate of change in FEV1 at 48 weeks (-2.3% vs. -3.02%, p=0.41), change from baseline in FEF at 48 weeks (-0.034 vs. -0.018 L/sec, p=0.728) and time to first pulmonary exacerbation, defined as IV antibiotic treatment for at least one respiratory sign or symptom (p=0.132). The incidence of serious adverse events in the denufosol arm was similar to placebo, with cough as the most common adverse event.

Last year, the company reported that a post-hoc subgroup analysis of patients taking 0-2 classes of concomitant medications in the Phase III TIGER-1 trial showed that denufosol non-significantly improved FEV1 from baseline to week 24 vs. placebo (see BioCentury, Oct. 4, 2010). In 2008, TIGER-1 met the primary endpoint of a significant improvement in baseline FEV1 after 24 weeks compared with placebo (see BioCentury, June 9, 2008). ...