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Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, today reported its consolidated financial results for the fourth quarter and full year 2013, and company highlights.
“The full year 2013 and the first weeks of 2014 were transformational for Alnylam and our continued efforts to advance RNAi therapeutics as a whole new class of medicines. We believe our recent alliance with Genzyme is a game changer in 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 the world. It also solidifies 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. “We’re also very pleased with our continued execution on our ‘Alnylam 5x15’ product strategy over the last several months, including initiation of new Phase 1, 2, and 3 clinical trials across three distinct programs, with multiple data read-outs expected in 2014. Specifically, we initiated our APOLLO Phase 3 trial with patisiran in ATTR patients with FAP; as the company’s first Phase 3 study, this is a very significant milestone in our history and also for the entire field of RNAi therapeutics. We’ve also advanced ALN-TTRsc – our first GalNAc-siRNA conjugate program to enter clinical development – into a Phase 2 trial in patients with TTR cardiac amyloidosis, with results expected later this year. We have also now started a Phase 1 clinical trial of ALN-AT3, an RNAi therapeutic targeting antithrombin for the treatment of hemophilia and rare bleeding disorders. ALN-AT3 is our second GalNAc-siRNA conjugate program to enter clinical testing, and we expect to share initial data from this Phase 1 trial later this year. Overall, we believe that our recent business and clinical accomplishments strengthen our efforts to build a leading, independent biopharmaceutical company that delivers value to our shareholders.”