Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, today reported its consolidated financial results for the first quarter 2014, and company highlights.
“The first quarter and recent period have been game changing for Alnylam. Specifically, we believe our alliance with Genzyme strengthens our efforts to bring RNAi therapeutics to patients with rare diseases as potential breakthrough genetic medicines. The new collaboration crystallizes Alnylam’s strategy to develop and commercialize our products in North America and Western Europe while Genzyme advances our products in the rest of world. It also bolsters our balance sheet, enabling an increased investment in an expanded number of RNAi therapeutic programs while securing a cash runway that we believe provides us with financial independence to develop and launch multiple products,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “In the recent period, we also presented a steady stream of clinical and pre-clinical data and advanced our overall pipeline. While we enroll patients in our APOLLO Phase 3 trial with patisiran, we were pleased to report positive initial data from our Phase 2 open-label extension study with patisiran, showing sustained knockdown of serum TTR protein levels at an 80% target level through 168 days. Our ALN-TTRsc Phase 2 study in TTR cardiomyopathy is enrolling ahead of schedule, and we now intend to expand the study to collect additional data in advance of our expected Phase 3 trial start later this year. Beyond our efforts in TTR amyloidosis, we initiated our Phase 1 study with ALN-AT3 – an RNAi therapeutic targeting antithrombin for the treatment of hemophilia – and look forward to providing an update from the ALN-AT3 program in the coming week. Finally, our new pre-clinical results with ALN-PCSsc demonstrate robust knockdown of PCSK9 and reductions in LDL-C, with the potential for a once-monthly and possibly once-quarterly subcutaneous dosing regimen, which we believe could be highly competitive with anti-PCSK9 monoclonal antibodies. In sum, we are very excited about our overall pipeline progress and the accumulation of these robust data sets that are defining the potential for a very attractive product profile for our RNAi therapeutic candidates, including the ability to clamp down disease targets in a predictable, sustainable, and durable manner. We look forward in the coming weeks to sharing additional updates from our pipeline.”