Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it has initiated a Phase 1 study with ALN-AT3, a subcutaneously administered RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders (RBD). ALN-AT3 has demonstrated efficacy in animal models of hemophilia, including in non-human primate models of induced hemophilia. Moreover, pre-clinical studies of ALN-AT3 support a wide therapeutic index in the hemophilia setting. ALN-AT3 is a key program in the company’s “Alnylam 5x15” product strategy, which is aimed at advancing multiple RNAi therapeutic genetic medicine programs into clinical development. Alnylam recently announced that it expects to exceed its original “Alnylam 5x15” guidance with six to seven genetic medicine programs in clinical development by the end of 2015, including at least two programs in Phase 3 and five to six programs that will have achieved human proof-of-concept results supporting further development.
“Hemophilia and other rare bleeding disorders are characterized by deficiencies in specific clotting factors that ultimately lead to inadequate thrombin generation and a bleeding diathesis. ALN-AT3 is aimed at correcting these bleeding disorders by knockdown of AT - an endogenous anticoagulant - thus, increasing thrombin generation and improving hemostasis,” said Akshay Vaishnaw, M.D., Ph.D., Executive Vice President and Chief Medical Officer of Alnylam. “Our pre-clinical results have demonstrated a promising activity profile for ALN-AT3 in hemophilia animal models, and our results in non-human primate studies are particularly encouraging in light of the excellent human translation we have seen with RNAi therapeutics. In addition, we have shown in preclinical studies that ALN-AT3 administration is associated with a very wide therapeutic index in the hemophilia setting. We are excited to be advancing this product candidate – notably, our second GalNAc-conjugate program – into a Phase 1 trial, and look forward to sharing initial data by the end of the year.”