The randomized, placebo-controlled single-dose escalation Phase I study enrolled patients with ATTR into seven sequential cohorts of increasing doses ranging from 0.01 to 1.0mg/kg.
Patients administered with ALN-TTR01 showed significant reductions in serum TTR protein levels, including both wild-type and mutant TTR protein.
ALN-TTR01 also demonstrated a dose-dependent reduction in serum TTR levels with a mean reduction of 38% at approximately day 7 to 10 in the 1mg/kg group.
Alnylam Pharmaceuticals Clinical Research senior director Jared Gollob said the Phase I data from the ALN-TTR01 clinical study demonstrate rapid, dose-dependent, and durable lowering of TTR protein levels after a single dose in ATTR patients.
"We believe these data with ALN-TTR01 provide key human proof of concept as we advance ALN-TTR02 as our ‘go-to-market’ RNAi therapeutic for the treatment of ATTR, a debilitating orphan genetic disease," Gollob added.
The company plans to commence a Phase II multi-dose study of ALN-TTR02 in the second half of 2012.