CAMBRIDGE, Mass. A drug for treating a genetic cholesterol disorder decreased levels of “bad” LDL cholesterol in patients, according to results of a late-stage clinical trial announced Wednesday.
Genzyme Corp. and Isis Pharmaceuticals announced results of a 124-patient phase 3 study of mipomersen for treating heterozygous familial hypercholesterolemia, a disease in which the body is unable to process LDL cholesterol. Patients with the disease carry a high risk of premature cardiovascular disease and death.
According to the study results, mipomersen reduced LDL cholesterol levels by 28%, thus meeting the trial’s primary goal. All of the patients in the study had pre-existing coronary artery disease and were taking statins and other lipid-lowering drugs. The companies said the trial also met its three secondary goals, with reductions of apolipoprotein B – a major component of LDL cholesterol – total cholesterol and non-HDL cholesterol.
“We are excited by these strong data in the second phase 3 trial of mipomersen,” Genzyme chief medical officer Richard Moscicki said. “This therapy has the potential to make a major difference in the lives of patients who are in great need of new treatment options.”