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Aegerion Pharmaceuticals Announces Additional Lomitapide (AEGR-733) Phase III Data Demonstrates Significant Lowering of LDL Cholesterol with Promising Safety Profile

Results Will Be Presented Today At AHA Conference in Orlando, Florida

BRIDGEWATER, NJ (November 16, 2009) — Aegerion Pharmaceuticals, Inc., a biopharmaceutical company focused on the treatment of cardiovascular and metabolic disease, today announced additional data from its ongoing Phase III trial involving its lead cholesterol management compound, lomitapide (AEGR-733), which is a microsomal triglyceride transfer protein (MTP) inhibitor small molecule drug.  The trial is designed to evaluate the long-term efficacy, safety and tolerability of lomitapide 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), which leads to life-threatening cardiovascular events.  Patients afflicted by this condition face a severely curtailed life expectancy and limited treatment options.  The results of the trial continue to show that lomitapide significantly reduces patients’ LDL-C vs. baseline and that it is well tolerated and demonstrates a promising safety profile.

There are 22 patients currently enrolled in the ongoing Phase III 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. 

The Phase III trial also continues to demonstrate a promising safety and tolerability profile. 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.

Bill Sasiela, Chief Medical Officer of Aegerion Pharmaceuticals, said, “The promising results we announced today are another significant step forward in our efforts to develop successful treatment options for patients who suffer from severe forms of hypercholesterolemia.  These data continue to suggest the ability of lomitapide to produce substantial reductions in LDL-C in the most severe patients, who are typically refractory to drug treatments, while providing a promising safety and tolerability profile. We were incredibly encouraged to find that treatment with lomitapide is associated with minimal to moderate hepatic fat accumulation at 26 weeks that seems to stabilize or even decrease after one year of treatment.  We believe these results indicate that lomitapide could become a potential component of the treatment regimen for severe dyslipidemic patients who are faced with limited treatment options today, as well as other high risk patients that have high cholesterol while taking existing lipid-lowering medications.” 

Marina Cuchel, MD, PhD, Research Assistant Professor at the University of Pennsylvania School of Medicine, will present these results with additional data as an abstract poster at the American Heart Association’s “Scientific Sessions 2009” on Monday November 16, 2009 at 9:30 a.m. ET – 11:00 a.m. ET in Orlando, FL. The poster will be available in Poster Hall A2-A3, Core 2, Poster Board 2011. 

About LOMITAPIDE (AEGR-733)

Lomitapide (AEGR-733) is a novel proprietary 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.

About Aegerion Pharmaceuticals, Inc.

Aegerion Pharmaceuticals, Inc. is a privately held biopharmaceutical company focused on the development and commercialization of promising pharmaceuticals to treat cardiovascular and metabolic disease. The Company’s primary focus is on hyperlipidemia. Its most advanced products have demonstrated significant LDL lowering activity in human trials and are currently in Phase III testing.

Company Contact:

Aegerion Pharmaceuticals, Inc.
Christine Pellizzari, Senior Vice President, General Counsel
908) 707-2100

Media Contact:

Sard Verbinnen & Co.
Jim Barron/Lesley Bogdanow/Nick Lawler
(212) 687-8080