Mipomersen: Phase III data

A double-blind, U.S. and Canadian Phase III trial in 158 patients with hypercholesterolemia and at high risk of developing coronary heart disease (CHD) showed that once-weekly subcutaneous injections of 200 mg mipomersen for 26 weeks met the primary endpoint of significantly reducing LDL-C from baseline to week 28 vs. placebo (37% vs. 5%, p<0.001). Specifically, mipomersen reduced LDL-C from an average of 123 mg/dL at baseline to 75 mg/dL at week 28, with 50% of patients achieving LDL-C <70 mg/dL. Mipomersen also met the secondary endpoints of significantly reducing APOB, total cholesterol and non-HDL cholesterol vs. placebo (p<0.001 for each). Patients were already receiving a maximally tolerated statin regimen.

Additionally, 26 patients receiving mipomersen and 2 patients receiving placebo discontinued treatment due to adverse events, including elevations in liver alanine transaminase (ALT) levels, injection-site reactions and flu-like symptoms. Specifically, 10% of patients treated with mipomersen had persistent ALT levels that were 3 times the upper limit of normal vs. an undisclosed percentage for placebo. The partners said the nature of the AEs were "generally similar" to what was seen in earlier trials. None of the patients had changes in other laboratory tests that would indicate hepatic dysfunction. ...