Today Alnylam reported a preliminary assessment of a small Phase I study (20 patients) showing statistically significant results in sharply reducing the levels of bad cholesterol in patients suffering from severe hypercholesterolemia. Alnylam also touted the use of second-generation lipid nanoparticles to effectively deliver an RNAi treatment (called ALN-PCS) in this study.
"...we believe is the first ever demonstration of efficacy for an RNAi therapeutic toward a clinically validated endpoint, namely LDL-C, a defined risk factor for coronary artery disease and acute myocardial infarction....," said Akshay Vaishnaw, M.D., Ph.D., Senior Vice President and Chief Medical Officer of Alnylam.
The new drug was well-tolerated at a variety of doses, and no one has dropped out of the study because of side effects, although a mild rash was observed in patients on the treatment, the company said. The study is still ongoing.
Albeit with a very small patient population and in aa early phase I trial, this is the first positive news from the clinic for an RNAi treatment in some time -- and comes on the heels of several Big Pharma players giving up on RNAi last year. Kudos for now. See Fierce Biotech and Xconomy.
Posted by Bruce Lehr Jan 4th 2012