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Pre-Clinical Results from ALN-CC5 for the Treatment of Complement-Mediated Diseases

We reported new pre-clinical results in animal models of myasthenia gravis (MG) from ALN-CC5, an investigational RNAi therapeutic in development for the treatment of complement-mediated diseases, at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting, held September 14-17 in New Orleans, Louisiana.


Data from two rat experimental autoimmune myasthenia gravis (EAMG) models showed that ALN-CC5 administration resulted in significant reductions in hemolytic activity and MG disease activity scores. In the passive model, ALN-CC5 was administered as a pretreatment, resulting in about 70% reduction in hemolytic activity and near complete prevention of disease at the highest dose level. In the active model, ALN-CC5 was administered after symptoms developed, resulting in about 80% reduction in hemolytic activity and reversal of disease progression.

We are encouraged by these data supporting the hypothesis that circulating C5 has a key role in MG pathogenesis, and we look forward to the continued development of ALN-CC5 for the treatment of MG and other complement-mediated diseases.