Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY) reported Q1 EPS of ($0.39), $0.15 better than the analyst estimate of ($0.54). Revenue for the quarter came in at $8.28 million versus the consensus estimate of $10.8 million.

“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.”

“In addition to highlights noted, we also made advancements with other pipeline programs and in other business development efforts. First, we are pleased to announce today that we have selected our DC for ALN-CC5, an RNAi therapeutic targeting complement C5 for the treatment of complement-mediated diseases, and are on track to file our IND for this program in late 2014. In addition, we have named a DC for our ALN-AAT program, an RNAi therapeutic targeting alpha-1 antitrypsin (AAT) for the treatment of AAT deficiency-associated liver disease, with the goal of filing an IND for this program in mid-2015. As with ALN-AT3 and ALN-PCSsc, these two programs employ our Enhanced Stabilization Chemistry (ESC) GalNAc-conjugate technology, which enables subcutaneous dosing with increased potency, durability, and a wide therapeutic index. We are very encouraged by the potent knockdown and durability results emerging from our programs utilizing this improved technology,” said Barry Greene, President and Chief Operating Officer of Alnylam. “Regarding additional progress in our business efforts, we made a bold and decisive move acquiring Merck’s RNAi assets, including their Sirna Therapeutics subsidiary. We believe this acquisition will complement and extend our own progress and cement our continued leadership in RNAi therapeutics, including our efforts with GalNAc-siRNA conjugate delivery technologies. All told, our recent progress on pipeline advancement and business development provide what we believe to be a solid foundation for value creation through Alnylam’s continued efforts in advancing RNAi therapeutics to patients.”

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