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Alnylam Presents New Pre-Clinical Data With Development Candidate For ALN-CC5, A Subcutaneously Administered RNAi Therapeutic Targeting Complement Component C5 In Development For The Treatment Of Complement-Mediated Diseases

Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), a leading RNAi therapeutics company, announced today new pre-clinical results with its Development Candidate (DC) for ALN-CC5, a subcutaneously administered investigational RNAi therapeutic targeting complement component C5 in development for the treatment of complement-mediated diseases. These results were presented at the 7 th International Conference on Complement Therapeutics being held June 6 – 11, 2014, in Olympia, Greece. New data demonstrate that ALN-CC5 led to an up to 98.7% knockdown of serum C5 and an up to 96.8% inhibition of complement activity in non-human primates (NHP) with weekly subcutaneous dose administration. Alnylam believes that ALN-CC5 – part of the company’s “Alnylam 5x15” product strategy – could represent a novel approach for the treatment of complement-mediated diseases, with a potentially competitive profile compared with intravenously administered anti-C5 monoclonal antibody therapy. ALN-CC5 utilizes the company’s Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate technology, which enables subcutaneous dosing with increased potency and durability and a wide therapeutic index. ESC-GalNAc conjugates are a clinically validated platform based on  recent preliminary Phase 1 study results from the company's ALN-AT3 program in development for the treatment of hemophilia. The company is on track to file its ALN-CC5 IND or IND equivalent in late 2014, and is now guiding that it expects to present initial clinical results in mid-2015.

“These new pre-clinical data with our recently selected Development Candidate for ALN-CC5 demonstrate potent C5 knockdown and robust inhibition of complement activity in NHPs with weekly subcutaneous dosing. We believe that these are promising results since an over 80% inhibition of complement activity has been shown to be associated with clinical benefit. Further, comparative studies in a mouse arthritis model showed ALN-CC5 to be as effective as an anti-C5 antibody in reducing disease activity, demonstrating a necessary and sufficient role for liver-expressed C5 in localized complement-mediated disease,” said Akshay Vaishnaw, M.D., Ph.D., Executive Vice President and Chief Medical Officer of Alnylam. “ALN-CC5 employs our ESC-GalNAc-siRNA conjugate platform that has recently been validated in preliminary data from human studies and where we observe a further ten-fold increase in potency as compared with studies in NHPs. Accordingly, we believe we are seeing the emergence of a compelling therapeutic profile for ALN-CC5, which could make it competitive with anti-C5 monoclonal antibody therapy. We look forward to filing an IND or IND-equivalent by the end of this year, and are now guiding that we expect to present initial clinical results in mid-2015.”

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