Alnylam 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)
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY) 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.
“We continue to focus on the clinical advancement of ALN-TTR02, the lead effort in our ‘Alnylam 5x15' product strategy. With dosing completed in the Phase I study, we are on track to report results early in the third quarter. Our Phase II study is now underway, and will evaluate a once-monthly multi-dose regimen in ATTR patients,” said Akshay K. Vaishnaw, M.D., Ph.D., Senior Vice President and Chief Medical Officer of Alnylam. “ALN-TTR02 utilizes our proprietary second-generation LNP delivery technology which has shown significant potency improvements in both pre-clinical and clinical studies, including recently reported results from our ALN-PCS hypercholesterolemia program. We look forward to continuing to share clinical data from our ALN-TTR02 program, and, assuming positive results in the current Phase II study, we plan to advance to a pivotal trial in 2013.”
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