Transave reports positive results from Phase II cystic fibrosis trial

The Phase II data indicated that Arikace, delivered once daily for 28 consecutive days, produced a significant improvement in lung function, was well-tolerated, and had a side-effect profile comparable to placebo.

The Phase II trial was a randomized, double-blind, placebo-controlled study of 64 patients from 15 centers in Europe. The intent-to-treat analysis for efficacy demonstrated that Arikace, when administered once daily either at 280mg or 560mg for 28 days, resulted in clinically significant improvement in lung function at the end of treatment.

This improvement was dose-dependent and was sustained at 28 days after completion of dosing, which was day 56 of the study. Pulmonary function (FEV1) increased significantly among patients receiving the 560mg dose of Arikace, with a sustained treatment effect of 224ml (p=0.004) and 17.6% (p=0.009) increase compared to placebo, at day 56.

At both the 280mg and 560mg doses, patients achieved a reduction in the density of Pseudomonas that was greater than the reduction achieved by placebo. Patients receiving the 560mg dose achieved a 2.2 log reduction in bacterial density.

Arikace was well tolerated, with no differences observed in the overall rates of adverse events or drug-related adverse events between groups. Arikace is a novel molecular entity comprised of the antibiotic amikacin, which is enclosed in nanocapsules of lipids called liposomes.