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Aegerion Pharmaceuticals, Inc. Announces AEGR-733 Phase II Data Demonstrates Significant Lowering of LDL Cholesterol With Promising Hepatic Safety Profile

November 6, 2008

Aegerion Pharmaceuticals, Inc., a biopharmaceutical company focused on the treatment of cardiovascular and metabolic disease, today announced top-line data from three separate Phase II trials involving its lead cholesterol management compound, AEGR-733, which is a microsomal triglyceride transfer protein (MTP) inhibitor. All three trials were designed to evaluate the efficacy, safety and tolerability of low doses of AEGR-733 alone and in combination with other lipid lowering agents such as Lipitor, Zetia and fenofibrate. The largest trial also included magnetic resonance spectroscopy (MRS) to quantify patients’ hepatic fat levels. Preliminary data from the trials indicates statistically significant reductions in patients’ low-density-lipoprotein cholesterol (LDL-C) vs. baseline, while at the same time suggesting a promising safety and tolerability profile, including low levels of hepatic fat accumulation.

The three Phase II trials ranged in duration from 8 to 12 weeks and collected clinical data on more than 460 patients who suffer from dyslipidemia, a condition in which there are abnormal lipid levels in the bloodstream. During the trials, AEGR-733 was administered alone as a once-daily pill in doses ranging from 2.5 mg to 10 mg and also in combination with other lipid lowering agents. At the high end of the dose range evaluated in these Phase II trials, the drug reduced LDL-C in patients up to 35% from baseline when used as a monotherapy, and up to 66% from baseline when administered in combination with Lipitor. In addition to reducing LDL-C, patients also experienced a reduction in their triglyceride levels by up to 50% and weight loss of up to 3% after 12 weeks on therapy.

The trials also suggested a promising safety and tolerability profile. In one of the trials, which was designed to evaluate patients’ hepatic fat levels while treated with AEGR-733 alone and in combination with Lipitor, Zetia and fenofibrate, the average hepatic fat levels after 12 weeks of exposure across doses of AEGR-733 ranging from 2.5 to 10 mg were approximately 7% with no arm exceeding 10%. This preliminary data compares favorably to what the Company believes are the relevant clinical benchmarks for hepatic fat.

Across all trials, less than 2% of the patients experienced adverse events related to elevated liver enzyme levels where such events resulted in their discontinuation from the studies. This compares favorably to what the Company believes are the relevant clinical benchmarks for elevated liver enzymes, statins and fenofibrate. When titrated from a starting dose of 2.5 mg of AEGR-733, less than 5% of patients experienced gastrointestinal adverse events leading to discontinuation from the trial, which the Company believes highlights the importance of titration as an important component of administration of AEGR-733 to reduce such adverse events.

Bill Sasiela, Chief Medical Officer of Aegerion Pharmaceuticals, said, “We are pleased with the results of these trials because they suggest the ability of AEGR-733 at low doses to significantly reduce LDL cholesterol and triglycerides with what we believe to be an acceptable safety and tolerability profile. We hope to confirm and extend these observations in our Phase III studies and believe that AEGR-733 could become an important part of the treatment regimen for dyslipidemic patients who have limited treatment options today.”

The top-line findings described above represent preliminary results from these studies. The Company expects to present more complete trial results at appropriate scientific meetings and in peer-reviewed publications at a later date.

Aegerion also released preliminary results from its ongoing open-label Phase III study of AEGR-733. This pivotal study focuses on patients suffering from a rare genetic disorder characterized by high cholesterol levels known as homozygous familial hypercholesterolemia (HoFH). In light of this patient population’s extremely high LDL-C levels, the Company believes higher doses of AEGR-733 may be an appropriate therapeutic option for these patients. In this ongoing study, patients are being treated with AEGR-733 in doses titrated up to 60 mg per day. Preliminary data reveals LDL-C reductions of greater than 50% (beyond the existing reductions patients experienced on background therapy, such as statins and cholesterol absorption inhibitors) in the majority of patients that have reached high dosages in the study. One patient was able to discontinue plasma apheresis (mechanical filtration of the blood used to temporarily remove extremely high levels of LDL-C). The reduction in LDL-C levels is consistent with the more than 50% reduction seen when AEGR-733 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. To date, safety and tolerability in these patients have been promising with no discontinuations from the study. Final results from this ongoing Phase III trial will be forthcoming upon study completion.

Will Lewis, Co-Founder and Chief Financial Officer of Aegerion, said, “We are encouraged by the promising results of these Phase II and Phase III trials, and we look forward to maximizing the potential of this therapy across the spectrum of patient need. We will be exploring several options in the immediate future to enable development of what the Company believes to be a promising new therapeutic option with the ability to safely lower LDL-C and triglycerides and contribute to weight loss.”

About AEGR-733

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 and Phase II testing.




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