Genzyme and Isis say mipomersen meets goals in 2 late-stage cholesterol studies

Genzyme, Isis say cholesterol drug meets goals

NEW YORK — Genzyme Corp. and Isis Pharmaceuticals Inc. said Wednesday their cholesterol drug candidate mipomersen proved effective at significantly cutting patients’ bad cholesterol levels in two late-stage studies.

The companies said mipomersen reduced LDL-C, or total cholesterol, by 36 percent in patients with severe high cholesterol, or hypercholesterolemia, compared with a 13 percent increase for patients taking a placebo. Meanwhile, the drug prompted a 37 percent reduction in total cholesterol for patients with high cholesterol at high cardiovascular risk, compared with a 5 percent drop for those taking the placebo.

Both studies met those key goals and all their secondary endpoints. Frequently observed side effects included injection site reactions, flu-like symptoms, and elevations in liver enzymes.

The results come months after positive results from a study focusing on patients with a genetic disorder called heterozygous familial hypercholesterolemia, that raises “bad” cholesterol. At that time in February, Genzyme said it wanted to finish other studies and get the drug on the market before it seeks approval for that illness. One such condition includes homozygous familial hypercholesterolemia, which is rarer than the heterozygous form.

Overall, the companies have now completed four late-stage studies on the drug for various types of cholesterol conditions. Genzyme expects to start seeking Food and Drug Administration approvals during the first half of 2011.

Mipomersen was discovered by Isis, of Carlsbad, Calif., and licensed to Genzyme, which is based in Cambridge, Mass.

Shares of Genzyme fell 35 cents to $69.85 in morning trading. Isis shares fell 53 cents, or 5.3 percent, to $9.46.