Alnylam Pharmaceuticals this week said that it has begun dosing patients in a phase I study of its siRNA-based transthyretin-mediated amyloidosis treatment ALN-TTR02.
The trial is a randomized, single-blind, placebo-controlled, single-ascending dose study that will enroll about 32 healthy volunteers, the company said.
The primary endpoint is safety and tolerability of a single dose of ALN-TTR02, and study subjects will be enrolled in five sequential cohorts of increasing doses ranging from 0.01mg/kg to 0.50 mg/kg. Secondary objectives include serial measurement of circulating TTR serum levels through at least day 56 following a single dose.
ALN-TTR02 is a second-generation version of ALN-TTR01, which was found to safely trigger statistically significant reductions in serum levels of TTR protein, which characterizes TTR amyloidosis (GSN 12/1/2011).
The new drug uses a different delivery technology, the so-called MC3 lipid, which Alnylam has said is proprietary to the company. Tekmira Pharmaceuticals, which developed the lipid nanoparticles used in ALN-TTR01, has sued Alnylam over the rights to the MC3 technology.