Alnylam Pharmaceuticals Completes Enrollment in Phase I Clinical Trial and Initiates Phase II Clinical Trial of ALN-TTR02, an RNAi Therapeutic Targeting Transthyretin (TTR) for the Treatment of Transthyretin-Mediated Amyloidosis (ATTR)

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it has completed enrollment in its Phase I trial with ALN-TTR02, an RNAi therapeutic targeting the transthyretin (TTR) gene for the treatment of TTR-mediated amyloidosis (ATTR). Alnylam also announced today that it has initiated a Phase II trial with ALN-TTR02 aimed at evaluating clinical activity, safety, and tolerability of multiple once-monthly doses of ALN-TTR02 in ATTR patients. Specifically, the study will evaluate the clinical activity of ALN-TTR02 based on measurement of serum levels of TTR, the disease-causing protein in patients with ATTR. ALN-TTR02 utilizes the company’s proprietary second-generation lipid nanoparticle (LNP) delivery technology using the “MC3” lipid. The company expects to present data from the Phase I trial early in the third quarter of 2012.