Wednesday, Genzyme Corp. (GENZ) and Isis Pharmaceuticals, Inc. (ISIS) announced commencing a second phase 3 study of mipomersen in patients with heterozygous familial hypercholesterolemia, or heFH, a genetic disorder that causes exceptionally high levels of LDL cholesterol.
The new trial will evaluate the safety and efficacy of mipomersen in patients who have heFH and coronary artery disease. The primary endpoint will be percent reduction in LDL cholesterol, with data expected to be available in 2010. The companies anticipate U.S. filing for the indication during the second half of 2010.
Genzyme and Isis plan to begin three additional trials evaluating mipomersen's safety and efficacy in reducing LDL cholesterol in high-risk patients during the second half of 2008.
In 2008, Genzyme and Isis completed a licensing agreement that provides Genzyme with exclusive worldwide rights to mipomersen, which was discovered and initially developed by Isis.
Mipomersen is a second-generation antisense drug that reduces the production of apoB-100, a protein critical to the synthesis and transport of "bad" cholesterol.
The companies also announced that the United States Patent and Trademark Office have granted a patent that broadly covers the use of antisense compounds targeting the apoB messenger RNA except a ribozyme.
Stanley Crooke, CEO of Isis said, "This comprehensive patent covers methods of inhibiting apoB by targeting anywhere on the messenger RNA including the site to which mipomersen binds, and all therapeutic uses that might result from lowering apoB."
Genzyme closed Wednesday's regular trading at $79.03, up $0.95 or 1.22%, while Isis closed at $17.33, down $0.20 or 1.14%.
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