Pre-Clinical Data with ALN-CC5 for Treatment of Complement Mediated Diseases Presented at ASH

We presented pre-clinical with ALN-CC5, an investigational RNAi therapeutic targeting complement component C5 for the treatment of complement-mediated diseases, at the American Society of Hematology (ASH) Meeting. Data showed an up to 99.2% knockdown of serum C5 and up to 96.2% inhibition of serum hemolytic activity in non-human primates (NHPs) with continued dosing for over seven months.

Although significant progress has been made in the treatment of complement-mediated diseases, including paroxysmal nocturnal hemoglobinuria (PNH), there is potential for significant improvements.  As a first-in-class C5 synthesis inhibitor, we believe that ALN-CC5 represents an innovative, differentiated, and well-validated approach for the treatment of complement-mediated diseases.  If our pre-clinical results can be extended to the clinical setting, we believe that they could represent an attractive therapeutic strategy and potential new treatment option for patients with PNH and other complement-mediated diseases.