Promising results for new cholesterol control mechanism

Data from a phase 2 clinical trial show that a treatment targeting the PCSK9 (proprotein convertase subtilisin/kexin type 9) antibody could be a new treatment for high cholesterol. Over an 8 to 12 week period, the new drug, developed by Sanofi and Regeneron Pharmaceuticals, reduced mean low-density lipoprotein-cholesterol (LDL, or ‘bad’ cholesterol) by 40 to 72 percent in patients with elevated LDL. PCSK9 is an example of how the study of genetics can identify new targets for therapies -- its role in lipid metabolism was discovered a few years ago in population studies. “Genetic data have shown that patients with natural loss-of-function mutations in PCSK9 have significantly lower LDL-C and a lower risk of coronary heart disease,” said Dr. Elias Zerhouni, President, Global Research & Development, Sanofi. “Based on this finding and the results of our Phase 2 trials, Sanofi and Regeneron plan to initiate the … Phase 3 program in the second quarter.”