Genzyme, Isis Post Phase 3 Data For Heart Drug Kynamro At AHA

Sanofi company Genzyme and its partner Isis Pharmaceuticals presented new two-year data from the Phase 3 extension study of Kynamro at the American Heart Association meeting in Chicago.

Kynamro (mipomersen sodium) injection is a first-in-class, oligonucleotide inhibitor of apolipoprotein B-100 synthesis. The drug is an adjunct to lipid-lowering drugs and proper diet for the reduction of low density lipoprotein-cholesterol (LDL-C) and certain cholesterols in patients with homozygous familial hypercholesterolemia (HoFH). Kynamro lowers LDL-C by hindering the formation of atherogenic lipoproteins, particles that carry cholesterol through the bloodstream. Kynamro is being jointly developed by Sanofi and Isis Pharmaceuticals and was approved by the U.S. Food and Drug Administration (FDA) in January 2013.

Retrospective analysis of the randomized, placebo controlled, Phase 3 study data show that patients who were treated with Kynamro for two years experienced significant decrease in Major Adverse Cardiovascular Events (MACE) as compared to two years before they received therapy. MACE were defined as myocardial infarction (MI), stroke, unstable angina (UA), and revascularization procedures (PCI/CABG). Two years prior to taking Kynamro therapy, 62 percent of patients had MACE. About two years later, only 9 percent of Kynamro-treated patients experienced MACE. The company noted that the reduction in MACE coincided with reduction in LDL cholesterol levels.

Homozygous familial hypercholesterolemia (HoFH) is a rare genetic disease marked by extreme cholesterol levels. Due to their body’s inability to naturally clear cholesterol, patients with HoFH are at risk for cardiovascular disease. “Kynamro is approved for use in these patients in the U.S. to reduce LDL-cholesterol, apoB, total cholesterol, and non-HDL cholesterol as an adjunct to lipid lowering medication and diet. The data presented today are encouraging and add to the broad lipid lowering profile observed in these patients,” said Dr. Sotirios Tsimikas, VP of clinical development and leader of the cardiovascular franchise at Isis.

“This analysis is very encouraging as the rate of MACE declined sevenfold after two years of treatment with Kynamro in patients with homozygous and heterozygous familial hypercholesterolemia (FH),” observes Dr. John Kastelein, professor of medicine and chairman of the department of vascular medicine, Academic Medical Center, University of Amsterdam. Dr. Kastelein added that the analysis supports further study of Kynamro.