Genzyme and Isis Initiate Phase III Study of Cholesterol Drug
Genzyme and Isis Pharmaceuticals have initiated a Phase III study of mipomersen in patients with heterozygous familial hypercholesterolemia, a genetic disorder that causes exceptionally high levels of low-density lipoprotein cholesterol.
The new trial will evaluate the safety and efficacy of mipomersen in patients who have heterozygous familial hypercholesterolemia and coronary artery disease. It is a randomized, double-blind, placebo-controlled study taking place at approximately 30 sites in the US 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 200mg dose of mipomersen or placebo weekly for 26 weeks. The primary endpoint will be percent reduction in low-density lipoprotein cholesterol, and data are expected to be available in 2010. The trial is expected to add to the body of clinical data on mipomersen and the overall product profile.
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.
Stanley Crooke, chairman and CEO of Isis, said: “This is an exciting moment for mipomersen, combining the initiation of the Phase III trial in heFH with a very broad patent allowance that expands the patent coverage for the apoB franchise.”