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Alnylam Pharmaceuticals Inc presents new pre-clinical data with development candidate for ALN-CC5

Monday, 9 Jun 2014 08:00am EDT 

Alnylam Pharmaceuticals Inc:Says 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.New data demonstrate that ALN-CC5 led to up to 98.7 pct knockdown of serum C5 and an up to 96.8 pct inhibition of complement activity in non-human primates (NHP) with weekly subcutaneous dose administration.Alnylam believes that ALN-CC5 - part of company's 'Alnylam 5x15' product strategy - could represent novel approach for treatment of complement-mediated diseases, with potentially competitive profile compared with intravenously administered anti-C5 monoclonal antibody therapy.ALN-CC5 utilizes 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.Company is on track to file its ALN-CC5 IND or IND equivalent in late 2014, and is guiding that it expects to present initial clinical results in mid-2015. 

Company Quote

-0.49 -0.45%
11:38am EDT