PCSK9 Trial Shows LDL Improvement

Sanofi‘s and Regeneron Pharmaceuticals‘ alirocumab, an investigational MAb targeting PCSK9 (proprotein convertase subtilisin/kexin type 9), has met its primary efficacy endpoint in a Phase III trial as a monotherapy. The mean low-density lipoprotein-cholesterol (LDL-C) reduction from baseline to week 24 was significantly greater in patients randomized to alirocumab, as compared to patients randomized to Zetia (ezetimibe): 47.2% vs. 15.6%.

Most of the companies’ Phase III trials of alirocumab are in combination with lipid-lowering drugs, but this monotherapy trial generated strong results. A total of 12 Phase III trials that have been initiated so far for treatment, with more than 23,000 patients enrolled in the program.

The percentage of patients in this trial who reported treatment emergent adverse events was 78.4% in the ezetimibe group and 69.2% in the alirocumab group. The most common class of adverse events was infections (39.2% with ezetimibe vs. 42.3% with alirocumab), which included nasopharyngitis, influenza, and upper respiratory tract infection. Injection-site reactions occurred in less than 2% of patients in both groups. Muscle-related adverse events occurred in 3.9% of patients treated with ezetimibe and 3.8% of patients treated with alirocumab.

Detailed results from the trial study will be presented at a medical conference in 2014.