Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today complete results from its Phase IIb trial with ALN-RSV01 for the treatment of respiratory syncytial virus (RSV) infection in lung transplant patients. The data were presented at the European Respiratory Society’s (ERS) Annual Congress held in Vienna, September 1-5, 2012. As reported previously, the study narrowly missed the primary endpoint of reduced day 180 BOS in an “intent-to-treat” (ITTc) analysis of confirmed RSV infected patients, but achieved statistically significant reductions in prospectively defined analyses of ITTc patients with their “last observation carried forward” (LOCF), and of ITTc patients treated “per protocol” (PP). At the ERS meeting, new results were presented on secondary endpoints and certain post-hoc analyses that support the efficacy of ALN-RSV01 in this setting. Further, and as reported earlier, ALN-RSV01 was found to be generally safe and well tolerated in the study. Alnylam plans to discuss these complete results with U.S. and European regulatory authorities, and communicate future development plans for ALN-RSV01 at year’s end.
“Our Phase IIb study results demonstrate that inhaled ALN-RSV01 reduces the incidence of new or progressive BOS in RSV-infected lung transplant patients. The complete results presented at the ERS meeting continue to show that ALN-RSV01 is associated with a significant treatment effect, including results of a multivariate logistic regression analysis where treatment with ALN-RSV01 showed an over eight-fold reduced risk in developing day 180 BOS. Further, we showed a statistically significant effect on the secondary endpoint of day 90 BOS, and demonstrated a particularly strong effect of over 80% in patients receiving ALN-RSV01 within five days of symptom onset,” said Akshay Vaishnaw, M.D., Ph.D., Executive Vice President and Chief Medical Officer of Alnylam. “Our plans are to discuss these results with U.S. and European regulatory authorities later this year and then communicate next steps, if any, for our ALN-RSV program. In the meanwhile, we continue to execute on our ‘Alnylam 5x15’ product strategy with a focus on our transthyretin-mediated amyloidosis and hemophilia programs.”