CAMBRIDGE, Mass., May 29, 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, announced today that an oral presentation summarizing the final 78-week results from its pivotal Phase III study of lomitapide in patients with adult Homozygous Familial Hypercholesterolemia (HoFH) was presented yesterday at the 80th European Atherosclerosis Society (EAS) Congress in Milan, Italy.
The oral presentation, entitled "A Phase III Study of Lomitapide, a Microsomal Triglyceride Transfer Protein (MTP) Inhibitor, in Patients with Homozygous Familial Hypercholesterolemia," was presented by Marina Cuchel, M.D., Ph.D, who is the Assistant Professor of Medicine and Member, Institute for Translational Medicine and Therapeutics at the University of Pennsylvania School of Medicine. The EAS Congress is an annual conference attended by approximately two thousand medical professionals throughout the world who specialize in the causes, treatment and prevention of atherosclerosis.
The Phase III study evaluated the safety and efficacy of lomitapide as an adjunct to a low-fat diet and other lipid-lowering therapies to reduce LDL-C levels in HoFH patients. HoFH patients were maintained on current lipid lowering therapy from six weeks prior to baseline LDL-C measurement through Week 26. The lomitapide dose was escalated from 5 mg to a maximum tolerated dose of up to 60 mg / day. The primary efficacy endpoint was mean percentage change in LDL-C from baseline at Week 26, after which patients remained on lomitapide for safety follow up through Week 78.
An electronic copy of the abstract of the oral presentation can be found at the following link: http://www.abstractserver.com/EAS2012/planner/index.php?go=abstract&action=abstract_iplanner&absno=1007&EAS2012=sh25mf0838998d0lgqsgn4lehe86ke0j&EAS2012=sh25mf0838998d0lgqsgn4lehe86ke0j
About Lomitapide
Lomitapide is a small molecule microsomal triglyceride transfer protein inhibitor (MTP-I) that Aegerion is developing as a once-day oral therapeutic for the treatment of patients with certain severe lipid disorders, including HoFH and familial chylomicronemia (FC). MTP exists in both the liver and intestines where it plays a role in the formation of lipoproteins containing cholesterol and triglycerides. Currently, there is no MTP-I approved by the FDA for any indication.
Lomitapide has been evaluated in fourteen Phase I and eight Phase II clinical trials, as well as the pivotal Phase III clinical trial completed in 2011, with an extension study as a separate protocol. Approximately 940 patients have been treated with lomitapide as part of these clinical trials.
In the first quarter of 2012, Aegerion submitted a New Drug Application (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, to reduce LDL-C in patients with HoFH. Both applications have been accepted for review. Lomitapide holds orphan drug designation for the treatment of HoFH in the United States (US), and for the treatment of FC in the US and European Union (EU).
About HoFH
HoFH is a rare genetic lipid disorder usually caused by defects in the low-density lipoprotein, or LDL, receptor genes, resulting in impaired or total loss of function in the LDL receptor. The LDL receptor is a protein on the surface of cells that is responsible for binding to and removing LDL from the blood. A loss of LDL receptor function results in accumulation of LDL-C in the blood.
Untreated HoFH patients have extremely high LDL-C levels, typically between 400 mg/dL and 1,000 mg/dL. These patients are at severely high risk of experiencing premature cardiovascular events, such as heart attack or stroke, often experiencing their first cardiovascular event in their twenties. If untreated, patients with HoFH generally die before the age of thirty.
About Aegerion Pharmaceuticals, Inc.
Aegerion Pharmaceuticals, Inc. (Nasdaq:AEGR) is committed to changing the way rare, life-threatening disorders are treated. We are an emerging biopharmaceutical company focused on the development and commercialization of novel, breakthrough therapeutics to treat debilitating and often fatal rare diseases. Aegerion is motivated by its commitment to patients first. We are also attentive to our core values of integrity, innovation, responsibility to healthcare providers, development of employees and always – scientific and clinical excellence.