Alnylam Pharma announces top-line results of Phase IIb clinical trial of ALN-RSV01; says study in RSV-infected lung transplant patients narrowly misses primary 'Intent-to-Treat' endpoint (ALNY) 10.12 : Co announced today top-line results from a Phase IIb trial with ALN-RSV01 for the treatment of respiratory syncytial virus infection in lung transplant patients. The primary endpoint of the study was the incidence of new or progressive bronchiolitis obliterans syndrome at 180 days after RSV infection. The study missed the primary endpoint of reduced BOS in an "intent-to-treat" analysis of confirmed RSV infected patients, but achieved statistically significant reductions in prospectively defined analyses of ITTc patients with their "last observation carried forward" with a p-value of 0.028, and of ITTc patients treated "per protocol" with a p-value of 0.025. In all analyses, ALN-RSV01 treatment was associated with a clinically meaningful treatment effect, with a reduction of over 50% in the incidence of day 180 BOS as compared with placebo. Top-line results are detailed in the table below. In the ITTc analysis, ALN-RSV01 narrowly missed the primary endpoint of new or progressive BOS at 180 days. ALN-RSV01 treatment was associated with a statistically significant reduction in the incidence of day 180 BOS in the prospectively defined LOCF and PP analyses. In all analyses, treatment with ALN-RSV01, as compared with placebo, was associated with a clinically meaningful reduction in the incidence of BOS with a treatment effect ranging from approximately 54% to 65%. Following central laboratory testing by PCR analysis, 10 patients could not be confirmed as infected for RSV; these patients happened to include nine patients randomized to receive placebo and one patient randomized to receive ALN-RSV01. ALN-RSV01 was found to be generally safe and well tolerated in the study, with a comparable incidence of reported adverse events in placebo and study drug treatment arms. There were three deaths in the study, two in placebo and one in ALN-RSV01, all of which were determined to be unrelated to treatment. Additional study results are expected to be presented at the European Respiratory Society Annual Congress taking place September 1-5, 2012 in Vienna.
