AEGERION'S CHOLESTEROL DRUG STUDY PUBLISHED IN NEJM

Aegerion Pharmaceuticals has announced the publication of positive results from a second Phase II clinical trial of its lead cholesterol-lowering compound, AEGR-733, in the New England Journal of Medicine (NEJM).

The results demonstrate that AEGR-733 reduced the levels of LDL by 51 percent from baseline in subjects with homozygous familial hypercholesterolemia (FH), a genetic condition characterized by dangerously high LDL cholesterol levels. AEGR-733 was also effective in reducing total cholesterol levels by 58 percent, triglyceride levels by 65 percent and apolipoprotein B levels by 56 percent from baseline.

Researchers conducted a dose-escalation study examining the efficacy, safety and tolerability of AEGR-733 in six patients with homozygous FH who were titrated to the study's highest dose. Patients received AEGR-733 at four different doses, each for four weeks, and returned for a final visit after an additional four-week drug washout period. Analysis of lipid levels, safety laboratory analyses and magnetic resonance imaging of the liver for hepatic fat content were performed throughout the study. Elevation of liver transaminase levels and accumulation of hepatic fat were seen in some, but not all, of the study patients.

AEGR-733 is a novel proprietary MTP inhibitor under development for the treatment of dyslipidemia. Inhibiting the MTP enzyme reduces blood levels of cholesterol and triglyceride by limiting the production of lipoproteins from the intestine and liver, according to Aegerion.