Alnylam Pharmaceuticals, a company specializing in RNAi therapeutics, has begun the Phase I clinical trial of ALN-TTR02, which uses the company’s in-house second-generation lipid nanoparticle technology utilizing the MC3 lipid.
ALN-TTR02 is an RNAi therapeutic targeting the transthyretin (TTR) gene for treating TTR-mediated amyloidosis (ATTR). Alnylam Pharmaceuticals is conducting ALN-TTR02’s Phase I study as a placebo-controlled, single-blind, randomized, single-ascending dose study involving 32 healthy volunteers in the United Kingdom. The primary objective of the trial is to assess the tolerability and safety of a single dosage of ALN-TTR02 in the volunteers. The second objective of the study is to assess the clinical activity of ALN-TTR02 by measuring the serum levels of TTR, a protein that causes disease in ATTR patients. Moreover, ALN-TTR02’s urine and plasma pharmacokinetics will also be studied. Alnylam Pharmaceuticals plans to release the study results during Q3 of 2012.
Alnylam Pharmaceuticals’ Senior Vice President and Chief Medical Officer, Akshay K. Vaishnaw stated that the initiation of the Phase I clinical trial of ALN-TTR02 is a key landmark in the company’s ‘Alnylam 5x15’ product strategy. The company plans to start a Phase II study later in 2012 and a pivotal study in 2013. ALN-TTR02 uses the company’s lipid nanoparticle technology, which has demonstrated meaningful potency enhancements in pre-clinical and clinical studies.
Unidade Clinica de Paramiloidose’s Director, Teresa Coelho stated that Alnylam Pharmaceuticals’ Phase I study of ALN-TTR01 has demonstrated positive results by knocking down serum levels of TTR. These results are crucial as TTR suppression can halt or even reverse disease pathology in patients with ATTR.