The drug is designed to block production of a protein that carries LDL cholesterol in the blood. It met its primary goal in a study of 51 patients with a rare genetic abnormality called homozygous familial hypercholesterolemia that increases their cholesterol level.
Patients who took mipomersen for about 26 weeks experienced a 25% reduction in their LDL cholesterol, also called bad cholesterol, as compared to only 3% for patients taking a placebo.
Besides meeting its main goal of lowering LDL cholesterol, the trial also met each of its three secondary goals of reduction in apolipoprotein B, total cholesterol and non-HDL cholesterol.
Richard A. Moscicki, Chief Medical Officer, Genzyme, said: “This is one of the largest studies of hoFH patients ever conducted, and we are very encouraged by these robust data and the emerging profile of the drug. With these results, we remain on-track with our development plan for mipomersen.”