Aegerion Pharmaceuticals, Inc. announced that 56-week data for its lead compound, lomitapide, in a pivotal phase III clinical trial, were consistent with the data seen in the same study at 26 weeks. The single-arm, open label trial is designed to evaluate the efficacy and long-term safety of lomitapide for the treatment of patients with homozygous familial hypercholesterolemia (HoFH). HoFH is a rare and often fatal condition characterized by severely elevated levels of Low-Density Lipoprotein Cholesterol, or LDL-C, leading to life-threatening cardiovascular events. Lomitapide has been designated by FDA as an orphan drug to treat this condition. The data show that lomitapide reduced cholesterol substantially in patients with HoFH.

As previously reported, the phase III study enrolled 29 patients with a mean LDL-C of 336mg/dL (352 mg/dL for completers) on a variety of background lipid-lowering therapies.

For Week 26, Intention-to-Treat (Last Observation Carried Forward) is followed by completer analysis in brackets. For Week 56, ITT and Completer Analysis are the same.

Thirteen of the 23 patients were able to have their background lipid-lowering therapy reduced during the safety phase of the study (between Week 26 and Week 56). Overall, the lipid profile at Week 56 is consistent with the data at Week 26, with LDL-C and triglyceride levels dropping substantially and HDL-C levels returning to baseline levels.

“These phase III filing data will be at the core of our anticipated NDA and MAA submissions, and we are pleased that it is consistent with week 26 data,” said Marc Beer, CEO of Aegerion Pharmaceuticals. “We will now turn our attention to ensuring that these submissions are completed in a high quality and timely fashion.”

As previously announced, of the 29 patients enrolled in the trial, three patients discontinued the study due to gastrointestinal adverse events and three patients withdrew consent to participate. Since last reported, no additional patients have discontinued therapy. Mild-to-moderate gastrointestinal adverse events have been the most commonly reported side effect in this trial. The majority of these events occurred during the first days following the introduction of a higher dose. As previously reported, 4 patients experienced consecutive aminotransferase (ALT or AST) elevations of between five times to eleven times the Upper Limit of Normal (ULN). Since last reported, no additional patients have experienced consecutive aminotransferase elevations above five times ULN. No patients have discontinued treatment due to Liver Function Test (LFT) elevations.

Lomitapide is a small molecule microsomal triglyceride transfer protein inhibitor being developed as an oral, once-a-day treatment for patients with severe lipid disorders. It is being evaluated for its ability to reduce LDL-C levels in patients with HoFH and reduce triglyceride levels in patients with familial chylomicronemia, or FC. It reduces lipid levels in the blood by preventing the liver and intestines from secreting lipids into the blood stream.

The pivotal, phase III trial is designed to evaluate the efficacy and long-term safety of lomitapide for the treatment of patients with HoFH. Enrollment completed in March 2010 with a total of 29 patients. The trial is a single-arm, open-label study being conducted at 11 sites in four countries. The patients are adult males and females with a mean age of 31 years. After a six week run-in period on current lipid lowering therapy to determine baseline measurements, patients received ascending doses of lomitapide titrated over the first 26 weeks of the trial to a maximum tolerated dose of 60 mg/day. Patients remain on their highest tolerated dose of lomitapide for an additional 52 week safety phase. The efficacy and safety phases combined will last 78 weeks.

Aegerion Pharmaceuticals, Inc. is an emerging biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat severe lipid disorders.