Positive Initial Phase 1/2 Clinical Results for ALN-CC5 for the Treatment of Complement-Mediated Diseases

Positive Initial Phase 1/2 Clinical Results for ALN-CC5 for the Treatment of Complement-Mediated Diseases

We reported positive initial clinical results from our Phase 1/2 trial of ALN-CC5, an investigational RNAi therapeutic targeting complement component C5 for the treatment of complement-mediated diseases. The new clinical data are being presented at the 20th Congress of the European Hematology Association (EHA) held June 11 – 14, 2015. Initial study results from 12 healthy volunteer subjects showed that subcutaneous administration of a single dose of ALN-CC5 resulted in potent, dose-dependent, durable, and statistically significant knockdown of serum C5 of up to 96%. In addition, single dose administration of ALN-CC5 achieved inhibition of serum complement activity of up to 92%, including an up to 61% inhibition of serum hemolytic activity. Further, ALN-CC5 has been found to be generally well tolerated to date.




We find these initial proof-of-concept results encouraging, and we expect – based on our experience in non-human primates, or NHPs – that this level of single-dose activity should meet our target profile as we progress in multi-dose cohorts. We believe that ALN-CC5 – as a first-in-class C5 synthesis inhibitor – represents an innovative, differentiated, and well-validated approach for the treatment of complement-mediated diseases. Further, we believe that a new medicine to treat excessive complement activity that could be given by infrequent, subcutaneous administration would be a welcome addition to the treatment landscape, and we look forward to the continued clinical advancement of this novel investigational therapeutic toward patients.



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