Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data from its RNAi therapeutic program for the treatment of hemophilia and other bleeding disorders at the 54 th American Society of Hematology Annual Meeting being held December 8-11, 2012 in Atlanta. Alnylam scientists presented data showing that ALN-AT3, a subcutaneously administered RNAi therapeutic targeting antithrombin (AT), yields potent, dose-dependent, and durable knockdown of AT in non-human primates (NHPs) with an up to four-fold increase in thrombin generation. Alnylam’s program in hemophilia comprises part of its ‘Alnylam 5x15’ product strategy, by which the company aims to advance five programs in clinical development, including programs in advanced stages, by the end of 2015.
“Our ALN-AT3 program is central to our ‘Alnylam 5x15’ strategy, which is aimed at bringing innovative medicines to patients, with a focus on RNAi therapeutics toward genetically defined targets for diseases with very high unmet medical need. Hemophilia and other bleeding disorders exemplify these types of diseases, as there remains significant need for new treatment options,” said Akshay Vaishnaw, M.D., Ph.D., Executive Vice President and Chief Medical Officer of Alnylam. “The new data with ALN-AT3 demonstrate potent and sustained knockdown of serum antithrombin and marked increases in thrombin generation in non-human primates. ALN-AT3 utilizes our GalNAc-siRNA conjugate delivery approach enabling subcutaneous dose administration, and is on track for an investigational new drug filing in mid-2013.”
“New therapeutic options are needed for patients with hemophilia, including those with inhibitors to their replacement factors, to prevent bleeding and the associated pathology. In addition, there is growing recognition of unmet need in patients with other rare bleeding disorders where congenital deficiencies of blood coagulation factors result in impaired thrombin generation and bleeding diatheses,” said Claude Negrier, M.D., head of the Hematology Department and director of the Haemophilia Comprehensive Care Centre at Edouard Herriot University Hospital in Lyon. “Human genetic data on co-inheritance of thrombophilic traits in patients with hemophilia support the hypothesis that inhibition of endogenous anticoagulant proteins, such as AT, can improve hemostasis. In aggregate, I am very encouraged by the emerging pre-clinical data on ALN-AT3. Indeed, availability of a subcutaneously administered therapeutic with a long duration of action could represent an exciting opportunity for hemophilia patients including those with inhibitors to their replacement factor, and patients with other rare bleeding disorders.”
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