Countering skeptics, Alnylam claims pioneering first in RNAi cholesterol study

Facing off against some deep-seated skepticism about the future of RNAi drug development, Alnylam ($ALNY) has pushed out a preliminary assessment of a small Phase I study demonstrating some dramatic results in sharply reducing the levels of bad cholesterol in patients suffering from severe hypercholesterolemia. And tackling doubts about being able to effectively deliver an RNAi treatment, Alnylam touted the use of second-generation lipid nanoparticles in the study.

The bottom line on the study is impressive: Patients taking ALN-PCS experienced a reduction of LDL-C of 50% or more. And the biotech unabashedly claimed an historic first in the study.

"We are extremely pleased with these data from our ALN-PCS trial which represent what 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. Indeed, preliminary results from our ongoing study show robust, statistically significant, and dose-dependent lowering of both PCSK9 and LDL-C levels," said Akshay Vaishnaw, M.D., Ph.D., Senior Vice President and Chief Medical Officer of Alnylam. "The RNAi effects were rapid and durable after a single dose, exemplifying a compelling profile for RNAi therapeutics that we have now established in man for two disease programs."

The RNAi world has been rocked by a number of high-level defections in the past two years as pharma companies assessed the time, costs and efforts needed to get a treatment through clinical trials and on to an approval. Encouraging efficacy data from a small Phase I, while helpful, is unlikely to dramatically change anyone's mind about that. But it is a step forward for a field that has been forced to contend with a bitter chill.

- here's the Alnylam release