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Aegerion Pharmaceuticals Announces Publication in the Lancet of Data From Its Pivotal Phase III Study of Lomitapide in Adult Patients with Homozygous Familial Hypercholesterolemia (HoFH)


CAMBRIDGE, Mass., Nov. 2, 2012 (GLOBE NEWSWIRE) -- Aegerion Pharmaceuticals, Inc. (Nasdaq: AEGR), an emerging biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat debilitating and often fatal rare diseases, today announced publication in the November 2nd online version of the Lancet of the 78 week data from the Company's pivotal Phase III study of lomitapide in adults patients with homozygous familial hypercholesterolemia (HoFH).

HoFH is a rare, hereditary disease that causes extreme elevation of blood cholesterol levels. Patients with this disease develop progressive atherosclerosis at a young age and are at high risk of experiencing premature cardiovascular events including heart attack or stroke. Despite current treatments, many patients with HoFH do not survive beyond their mid-30's. 

"We are pleased that the Lancet recognizes the importance of this research and the relevance of the findings in this rare disease with significant medical needs," said Marc Beer, Chief Executive Officer at Aegerion.

 About Lomitapide

Lomitapide is a microsomal triglyceride transfer protein (MTP) inhibitor that Aegerion is developing as a once-daily capsule for the treatment of patients affected with HoFH.  MTP exists in both the liver and intestines where it plays a key role in the formation of lipoproteins containing cholesterol and triglycerides. Inhibiting MTP has been shown to reduce the level of cholesterol that the liver and intestines assemble and secrete into the bloodstream. Currently, there is no MTP inhibitor approved by the FDA for any indication.

In addition to this pivotal Phase III clinical trial in HoFH completed in 2011, lomitapide has been evaluated in fourteen Phase I and eight Phase II clinical trials, including one Phase II trial in HoFH patients.  An extension study of the Phase III study is ongoing to assess long-term safety.  Over 900 patients have been treated with lomitapide as part of these clinical trials.

The most frequent adverse events in the Phase III clinical trial were gastrointestinal, and were generally mild to moderate. These events typically decreased after the patients were established on the maximally tolerated dose. Elevations in liver enzymes and hepatic fat were also observed in the Phase III trial. Four patients experienced elevations in liver enzymes of between five times to eleven times the upper limit of normal. Hepatic fat increased from a baseline of 1% to 8.3% at week 26, and then stabilized through week 78. 

Aegerion submitted an NDA to the Food and Drug Administration (FDA), and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA), requesting approval to market lomitapide as an adjunct to a low-fat diet and other lipid-lowering therapies, with or without apheresis, to reduce LDL-C, total cholesterol, apolipoprotein B, and triglycerides in adults with HoFH.

About Homozygous Familial Hypercholesterolemia (HoFH)

HoFH is a rare genetic lipid disorder that, if left untreated, results in extremely high cholesterol levels, typically between 400 mg/dL and 1,000 mg/dL. Those affected are at very high risk of experiencing premature cardiovascular events, such as heart attack or stroke, often experiencing their first cardiovascular event in their twenties. Despite current treatments, many patients with HoFH do not survive beyond their mid-30's.

The disease is usually caused by genetic defects that affect the function of the low-density lipoprotein (LDL) receptor, resulting in impaired or total loss of function. The LDL receptor is a protein on the surface of cells that is responsible for binding to and removing cholesterol from the blood. A loss of LDL receptor function results in elevated accumulation of cholesterol.

About Aegerion Pharmaceuticals

Aegerion Pharmaceuticals is an emerging biopharmaceutical company focused on the development and commercialization of novel, life-altering therapeutics to treat debilitating and often fatal rare diseases. The company's lead drug candidate, lomitapide, is in late-stage development for the treatment of homozygous familial hypercholesterolemia (HoFH), a rare life-threatening disease characterized by severely elevated cholesterol levels.  

Aegerion is motivated by its commitment to patients first, as well as its core values of integrity, innovation, responsibility to healthcare providers and development of employees, with a constant focus on scientific and clinical excellence. For more information, visit

CONTACT: Aegerion Pharmaceuticals, Inc. Michael Lawless, VP, IR (857) 242-5028
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