Alnylam Pharmaceuticals Presents Positive Preliminary Clinical Results for ALN-TTR01, an RNAi Therapeutic for the Treatment of Transthyretin-Mediated Amyloidosis

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today positive preliminary results from its Phase I clinical trial with ALN-TTR01, an RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR). The data were presented at the VIIIth International Symposium on Familial Amyloidotic Polyneuropathy being held November 20 - 22, 2011 in Kumamoto, Japan. Data from this study show that administration of ALN-TTR01 resulted in statistically significant reductions in serum TTR protein levels in ATTR patients. Lowering of TTR was found to be dose dependent, rapid, and durable after just a single dose. To date, ALN-TTR01 has been generally safe and well tolerated in this study.

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