Genzyme, Isis begin phase 3 trial for lipid-lowering drug

Genzyme and Isis Pharmaceuticals have begun a phase 3 study of mipomersen in patients with heterozygous familial hypercholesterolemia (heFH), a genetic disorder that causes high levels of LDL cholesterol.

It is the first of four new trials the companies plan to initiate by the end of this year, and the second late-stage study of mipomersen, a lipid-lowering drug that utilizes antisense technology, according to the Cambridge, Mass.-based Genzyme and the Carlsbad, Calif.-based Isis. 

The companies also announced that the U.S. Patent and Trademark Office granted the patent entitled "Antisense Modulation of Apolipoprotein B Expression," U.S. Patent No. 7,407,943. The patent covers the use of antisense compounds targeting the apoB messenger RNA except a ribozyme.   

Isis and Genzyme said the new trial will evaluate the safety and efficacy of mipomersen in patients who have heFH and coronary artery disease. It is a randomized, double-blind, placebo-controlled study taking place at approximately 30 sites in the U.S. and Canada, with an anticipated total enrollment of around 100 patients. Patients on a stable dose of other lipid-lowering agents are being randomized 2:1 to receive a 200 mg dose of mipomersen or placebo weekly for 26 weeks. The primary endpoint will be percent reduction in LDL cholesterol, and data are expected to be available in 2010. 

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