Initial Evidence for Potential Correction of the Hemophilia Phenotype in Phase 1 Study of ALN-AT3

Initial Evidence for Potential Correction of the Hemophilia Phenotype in Phase 1 Study of ALN-AT3

We announced updated clinical results from the ongoing Phase 1 study of ALN-AT3, a subcutaneously administered, investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders (RBD).  New results were presented at the 2015 Goring Coagulation Conference in London.  Specifically, data were presented from the study’s second dose cohort in hemophilia subjects (n=3), where subcutaneous administration of ALN-AT3 resulted in an up to 70% knockdown of AT.  New results provide initial evidence for potential correction of the hemophilia phenotype associated with ALN-AT3 administration and AT knockdown.  Specifically, ALN-AT3 administration resulted in an increase in thrombin generation of up to 334% and a marked improvement in whole blood clotting.  In addition, the most advanced severe hemophilia A subject in the cohort has remained bleed free for 47 days without replacement factor prophylaxis as of the January 6, 2015 data cut-off date. In addition, ALN-AT3 administration remains generally well tolerated.



We believe that these new results from our ongoing Phase 1 study with ALN-AT3 provide initial evidence for potential disease-modifying effects in hemophilia.  While early and based on a limited number of subjects, we believe that these new data support human proof-of-concept for ALN-AT3, a promising and innovative strategy for the treatment of hemophilia and rare bleeding disorders.



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