Making Commercial Sense of Isis Pharmaceuticals' Cholesterol Drug Mipomersen

Isis Pharmaceuticals (ISIS-$10.31) sold development and marketing rights of its flagship antisense drug mipomersen to Genzyme in January 2008. Mipomersen, a first-in-class apo-B synthesis inhibitor, works by decreasing the production of apo-B, a protein critical to the synthesis and transport of LDL and VLDL cholesterol — the “bad” lipids involved in the buildup of plaque in the arteries and the development of heart disease — through the bloodstream.

Whether mipomersen rises to $1 billion-plus blockbuster status or remains a niche drug with $250 million peak sales will depend on how the FDA feels about approving the drug for use in a broad pool of high-risk patients (refractory to existing lipid-lowering therapy) with uncontrolled LDL.

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Editor David J Phillips does not hold a financial interest in any stocks mentioned in this article. The 10Q Detective has a Full Disclosure Policy.