Aegerion Pharmaceuticals Announces Interim Lomitapide (AEGR-733) Phase III Data Demonstrates Significant Lowering of LDL Cholesterol with Promising Hepatic Safety Profile
Webcast of Company-Sponsored Symposium to Review Clinical Data Scheduled for June 16th 2009, 12:30-2:00 pm ET at the ISA (International Symposium on Atherosclerosis) in Boston, MA
BRIDGEWATER, N.J.--(BUSINESS WIRE)--Jun 15, 2009 - Aegerion Pharmaceuticals, Inc., a biopharmaceutical company focused on the treatment of cardiovascular and metabolic disease, today announced interim data from its ongoing pivotal Phase III trial involving its lead cholesterol management compound, lomitapide (AEGR-733), which is a microsomal triglyceride transfer protein (MTP) inhibitor small molecule drug. This pivotal trial is designed to evaluate the 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. Preliminary data from this trial indicate that lomitapide significantly reduced patients' LDL-C vs. baseline, was well tolerated and demonstrated a promising safety profile, including low levels of hepatic fat accumulation.
There are 19 patients currently enrolled in the ongoing Phase III trial, which is studying lomitapide in HoFH patients, and the interim analysis examined 10 patients who had been in the trial and on lomitapide for a minimum of six months. In this trial, patients are titrated up to a maximum tolerated dose of lomitapide (up to 60 mg/day). On average, at their maximum tolerated dose, these 10 patients have experienced an additional reduction in LDL-C of 44% on top of existing background therapy. Average baseline LDL-C levels in this trial were 351 mg/dl and four of the 10 patients achieved an LDL-C level below 100 mg/dl. The reduction in LDL-C levels is consistent with the more than 50% reduction seen when lomitapide was used as a monotherapy in a separate Phase II study, the results of which were published in the New England Journal of Medicine in January 2007.
This trial continues to suggest a promising safety and tolerability profile. Of the 19 patients currently enrolled in the trial, two have discontinued due to GI side effects. There have been no discontinuations due to liver enzyme elevations. The trial also includes magnetic resonance spectroscopy to quantify the level of the patients' hepatic fat. The average hepatic fat level after six months of treatment was approximately 8%. This preliminary data compares favorably to what the Company believes are the relevant clinical benchmarks for hepatic fat.
Bill Sasiela, Chief Medical Officer of Aegerion Pharmaceuticals, said, “We are pleased with these promising interim results that suggest the ability of lomitapide to significantly and safely lower the LDL-C levels of the most severe and refractory patients. We are hopeful that these results will continue over the course of this pivotal Phase III trial and believe that lomitapide could become an important part of the treatment regimen for severe dyslipidemic patients who have limited treatment options today. Importantly, while this trial focuses on the most severe patients, we are continuing to develop lomitapide for other appropriate populations, including patients with severe, refractory heterozygous familial hypercholesterolemia, such as those eligible for lipid apheresis.”
In conjunction with the ISA in Boston, MA, an Aegerion-sponsored Symposium to review its clinical data is scheduled for June 16th 2009 from 12:30-2:00 pm ET at the Sheraton Boston Hotel in the Republic Ballroom. The Symposium will be webcast and can be accessed through the following link:
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.
Aegerion Pharmaceuticals, Inc.
Will Lewis, 908-707-2100
Co-Founder and Chief Financial Officer
Sard Verbinnen & Co.
Jim Barron/Lesley Bogdanow/Nick Lawler
Posted: June 2009