Aegerion Pharmaceuticals Initiates Phase 3 Clinical Trial in Japan to Evaluate Lomitapide for Treatment of Homozygous Familial Hypercholesterolemia (HoFH)

Multi-Site Trial Studying the Safety and Efficacy of Lomitapide in the Japanese HoFH Patient Population

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| Source: Aegerion Pharmaceuticals, Inc.

CAMBRIDGE, Mass., April 9, 2014 (GLOBE NEWSWIRE) -- Aegerion Pharmaceuticals, Inc. (Nasdaq:AEGR), a biopharmaceutical company dedicated to the development and commercialization of novel therapies for patients with debilitating rare diseases, today announced that patient enrollment has been initiated in Japan in a clinical trial of lomitapide as an adjunct treatment to reduce low-density lipoprotein cholesterol (LDL-C) in Japanese patients with homozygous familial hypercholesterolemia (HoFH). Last year, Aegerion received orphan drug designation from Japan's Ministry of Health, Labour and Welfare for lomitapide.

The phase three, open-label trial in Japan is similar in design to the completed Phase 3 trial conducted in the United States and other countries. Estimated enrollment is between five and 10 adult HoFH patients who are receiving concomitant lipid-lowering therapies, including, in some cases, apheresis. After a six week run-in period, patients will receive lomitapide for 26 weeks, starting at 5 mg/day and escalating to a maximum dose of 60 mg based on tolerability. After 26 weeks, patients will enter a 30 week safety phase. The primary endpoint is % change in LDL-C levels from baseline to week 26. Secondary endpoints include % change in lipid parameters, long-term safety, and changes in hepatic fat from baseline to week 56. The Japanese regulatory authorities (PMDA) have indicated they will allow the new drug application to be filed following completion of the 26 week efficacy phase.

"This pivotal trial marks an important milestone for Aegerion and Japanese HoFH patients," said Mark Sumeray, MD, Chief Medical Officer. "Japan is a well-characterized region for patients with severe hypercholesterolemia and investigators there have shown a high level of enthusiasm for the trial, recognizing the unmet clinical need for their patients living with this serious disease."

"Initiation of the phase three trial in Japan allows us to sharpen focus on our regulatory timeline as we seek approval in this key market," said Martha Carter, Chief Regulatory Officer. "We currently hope to submit the filing for marketing approval in Japan as early as mid-2015."

HoFH is a serious, rare genetic disease inherited from both parents that impairs the function of the receptor responsible for removing LDL-C ("bad" cholesterol) from the body. A loss or impairment of LDL receptor function results in severe elevation of blood cholesterol levels. HoFH patients often develop premature and progressive atherosclerosis, a narrowing or blocking of the arteries.

About Aegerion Pharmaceuticals

Aegerion Pharmaceuticals is a biopharmaceutical company dedicated to the development and commercialization of innovative therapies for patients with debilitating rare diseases. For more information about the company, please visit www.aegerion.com.

Forward-Looking Statements

This press release contains forward-looking statements, including statements regarding the clinical development and regulatory pathway for approval of lomitapide in Japan and the potential for lomitapide as a treatment for HoFH. These forward-looking statements are neither promises nor guarantees of future performance, and are subject to a variety of risks and uncertainties, many of which are beyond our control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, the risks and uncertainties include, among other factors: the risk that we may not be able to enroll and maintain in the Japanese clinical study a sufficient number of HoFH patients to complete the study on a timely basis; the risk that clinical development of lomitapide in Japan may generate different results than those generated in the Phase 3 study of lomitapide that was the basis for approval in other countries; the risk of unexpected safety data arising in the course of the Japanese study; the risk of other unexpected hurdles or delays in the conduct of the study; the risk that regulatory authorities in Japan may require us to generate more data prior to filing for regulatory approval of lomitapide in Japan or may not agree with or accept our interpretation of the results of the study; and the other risks inherent in drug development. For additional disclosure regarding these and other risks we face, see the disclosure contained in our public filings with the U.S. Securities and Exchange Commission (available on the SEC's website at http://www.sec.gov), including the "Risk Factors" section of our most recent Annual Report on Form 10-K for 2013. We undertake no obligation to update or revise the information contained in this press release, whether as a result of new information, future events or circumstances or otherwise.

Investor Contact:
Aegerion Pharmaceuticals, Inc.
Amanda Murphy, Manager, IR
(857) 242-5024