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Alnylam Advances Development Candidate For ALN-AAT, A Subcutaneously Administered RNAi Therapeutic Targeting Alpha-1 Antitrypsin (AAT) In Development For The Treatment Of AAT Deficiency-Associated Liver Disease

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it is advancing its Development Candidate (DC) for ALN-AAT, an RNAi therapeutic targeting alpha-1 antitrypsin (AAT) in development for the treatment of AAT deficiency-associated liver disease. New pre-clinical data were presented in a Late-Breaking Abstract Session at Digestive Disease Week (DDW), held May 3 – 6, 2014 in Chicago, Illinois. ALN-AAT is one of Alnylam’s genetic medicine programs, which are RNAi therapeutics directed toward genetically defined targets for the treatment of diseases with high unmet medical need. AAT deficiency-associated liver disease is caused by accumulation of mutant AAT protein (“Z-allele” or “Z-AAT”) in liver tissue with subsequent liver injury, fibrosis, cirrhosis, and, in some cases, hepatocellular carcinoma. It is estimated that approximately 12,000 people with AAT deficiency in the U.S. and E.U. have associated liver pathology. The company now plans to initiate IND-enabling studies with the goal of filing an IND or IND equivalent for ALN-AAT in mid-2015.

“Our pre-clinical research efforts have now led to the selection of a DC in our ALN-AAT program, which is part of our genetic medicine pipeline. This is important progress since we believe ALN-AAT holds considerable promise as a novel therapeutic approach for the treatment of liver disease associated with AAT deficiency, an increasingly recognized problem where there is significant unmet need. Our pre-clinical results, including new data presented at the DDW meeting, demonstrate that RNAi therapeutics targeting AAT can reduce liver levels of mutant AAT, improve histopathology associated with mutant AAT expression, and reduce liver fibrosis and the incidence of tumor formation in a mouse model of disease; additional data have been generated in non-human primates,” said Rachel Meyers, Ph.D., Vice President, Research and RNAi Lead Development at Alnylam. “We very much look forward to the continued advancement of this program toward the clinic, including the expected filing of an IND in mid-2015. As such, ALN-AAT is positioned to become our seventh program in clinical development by the end of 2015, consistent with the up-scaled “Alnylam 5x15” guidance that we provided earlier this year.”

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