Aegerion Pharmaceuticals announced additional data from its ongoing Phase 3 trial involving lomitapide (AEGR-733) for the treatment of patients with Homozygous Familial Hypercholesterolemia (HoFH), a rare and extremely serious condition resulting in severely elevated levels of low-density lipoprotein cholesterol (LDL-C).

There are 22 patients currently enrolled in the ongoing Phase 3 trial. At the time of this most recent analysis, 14 of the patients had been in the trial and on lomitapide for a minimum of 26 weeks and seven of the patients had been treated with lomitapide for 56 weeks. In this trial, patients are titrated up to a maximum tolerated dose of lomitapide (up to 60 mg/day). The 14 patients treated with lomitapide for 26 weeks experienced a mean reduction in LDL-C of 49% on top of maximum tolerated background therapy. Average baseline LDL-C levels in this trial were 351 mg/dl and six of the 14 patients achieved an LDL-C level below 100 mg/dl, with ten of the 14 patients achieving LDL-C levels below 165 mg/dl.

At 26 weeks, patients experienced a modest increase in hepatic fat from 1.0% to 7.8%; however, all patients that have reached 56 weeks of treatment have seen their hepatic fat levels reduced from the levels seen at 26 weeks, with a mean hepatic fat level of 3.7% at 56 weeks. Additionally, mild to moderate gastrointestinal adverse events have been the most commonly reported side effect to date for patients at these high doses of lomitapide. Only two of the 14 patients experienced transaminase elevations which required a dose reduction and none of the patients required drug discontinuation due to liver function test elevations.

Lomitapide (AEGR-733) is a MTP-inhibitor under development for the treatment of dyslipidemia (abnormal lipid levels in the bloodstream). Inhibiting the MTP enzyme reduces blood levels of cholesterol and triglyceride by limiting the production of lipoproteins from the intestine and liver.

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