According to Tekmira Pharmaceuticals, a specialist in RNA interference (RNAi) therapeutics, Alnylam Pharmaceuticals has reported positive results on its Phase I clinical study of ALN-PCS, a target-specific RNAi therapeutic for PCSK9 called ALN-PCS that uses Tekmira Pharmaceuticals’ lipid nanoparticle technology to treat severe hypercholesterolemia.
Alnylam Pharmaceuticals presented the latest results at the Arteriosclerosis, Thrombosis, and Vascular Biology 2012 Scientific Sessions conducted by the American Heart Association in Chicago, Illinois. The results demonstrated that a single dose administration of ALN-PCS without concomitant lipid-lowering agents like statins lowered PCSK9 plasma levels by up to 84% and bad cholesterol or low-density lipoprotein cholesterol by up to 50%. The data also showed that ALN-PCS was safe and well tolerated in this trial.
The President and Chief Executive Officer at Tekmira Pharmaceuticals, Dr. Mark J. Murray stated that the latest human clinical study data on ALN-PCS is another testament proving that the company’s lipid nanoparticle technology allows RNAi activity in humans and is well tolerated. Since the proof of positive results within the RNAi realm is ever-growing, the company believes that its lipid nanoparticle technology will play a key role in advancing the field to commercialize RNAi therapeutics for numerous clinical indications.
In Tekmira's lipid nanoparticle technology, formerly known as stable nucleic acid-lipid particles or SNALP, siRNAs are efficiently encapsulated in homogeneous lipid nanoparticles, which can effectively deliver RNAi medicines to diseased spots in several preclinical models. The company uses a reproducible and scalable proprietary method to synthesize lipid nanoparticle formulations.