Alnylam Pharmaceuticals Files Clinical Trial Application for ALN-TTR02, an RNAi Therapeutic Targeting Transthyretin for the Treatment of Transthyretin-Mediated Amyloidosis

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it has filed a Clinical Trial Application (CTA) with the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase I clinical trial with ALN-TTR02, an RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR). ALN-TTR02 is a systemically delivered RNAi therapeutic, targeting the TTR gene, that comprises an siRNA formulated in a second-generation lipid nanoparticle (LNP). Following clearance of the CTA, Alnylam expects to initiate the ALN-TTR02 Phase I study in the first half of 2012 with data expected to be reported in the third quarter of 2012.