January 6, 2012

Aegerion Pharmaceuticals Announces Lomitapide 78-Week Phase III Clinical Data Consistent With Earlier Results

Efficacy and Safety Profile Maintained

Company to Webcast Presentation at the 30th Annual J.P. Morgan Healthcare Conference

CAMBRIDGE, Mass., Jan. 6, 2012 (GLOBE NEWSWIRE) -- Aegerion Pharmaceuticals, Inc. (Nasdaq:AEGR), an emerging biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat severe lipid disorders, today provided a clinical update on its lead investigational therapeutic, lomitapide.

Aegerion reported that the 78-week data from its pivotal Phase III clinical trial are consistent with data previously reported at the 26- and 56-week time points. The Phase III study was a single-arm, open label trial, designed to evaluate the efficacy and long-term safety of lomitapide for the treatment of patients with homozygous familial hypercholesterolemia (HoFH). The data demonstrate that the reduction in LDL-C cholesterol from baseline was maintained at 78-weeks.

"We are pleased that the 78-week data are consistent with our 56-week data," said Marc D. Beer, Chief Executive Officer. "We expect to submit our applications containing the 56-week data to the FDA and EMA this quarter and continue to build our commercial organization in preparation for our anticipated launch."

Lomitapide is a small molecule microsomal triglyceride transfer protein inhibitor, or MTP-I, in clinical development as an oral, once-a-day therapeutic for HoFH. Lomitapide is being evaluated for its ability to reduce low density lipoprotein (LDL-C) or bad cholesterol levels in patients with HoFH. HoFH is a rare and often fatal condition characterized by severely elevated levels of LDL-C leading to life-threatening cardiovascular events. Lomitapide has been designated by FDA as an orphan drug to treat this condition.

The Phase III study enrolled 29 patients with a mean LDL-C of 337 mg/dL (352 mg/dL for completers) on a variety of background lipid-lowering therapies. Patients were 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 escalated over the first 26 weeks of the trial to a maximum tolerated dose of up to 60 mg/day. Patients remained on their highest tolerated dose of lomitapide for an additional 52 weeks, to complete 78 weeks of treatment.

The results for all patients through week 78 are summarized below:  

Protocol Phase
Week 26
(Efficacy Phase)
Week 56
(Safety Phase)
Week 78
(Safety Phase)
Background Therapy
N 29 [23] 23 23
Average Dose (mg) 38.4 [44.6] 40.2 40.7
(mean % change from baseline)
-40.1 [-50.2]
Hepatic Fat (mean %)
(Baseline 1.0)
(Baseline 1.2)
(Baseline 1.2)

For Week 26, Intention-to-Treat (Last Observation Carried Forward) is followed by Completer Analysis in brackets. For Weeks 56 and 78, ITT and Completer Analysis are the same.


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. Between week 26 and week 78, no additional patients permanently discontinued therapy. 

Mild-to-moderate gastrointestinal adverse events have been the most commonly reported side effect in this trial. The frequency of these events decreased after the dose escalation period was finished and patients were established on their maximum tolerated dose. As previously reported, in the first 56 weeks of the Phase III trial, four patients experienced consecutive aminotransferase (ALT or AST) elevations of between five times to eleven times the upper limit of normal (ULN). Between week 56 and week 78, no additional patients experienced consecutive ALT or AST elevations of greater than five times ULN. No patients permanently discontinued treatment during the 78-week study due to liver function test (LFT) elevations. Mean hepatic fat remained stable between week 56 and week 78.

J.P. Morgan Healthcare Conference Presentation and Webcast

Aegerion will participate in the 30th Annual J.P. Morgan Healthcare Conference in San Francisco. CEO, Marc D. Beer, is scheduled to present on Thursday, January 12, 2012, at 11:30 a.m. PST (2:30 p.m. EST). A live webcast of the presentation will be available and can be accessed for 90 days following the conference by visiting the "Investors" section of Aegerion's website, www.aegerion.com.

About Aegerion Pharmaceuticals, Inc.

Aegerion Pharmaceuticals aspires to change the way rare, genetic lipid disorders are treated. We are an emerging biopharmaceutical company focused on the development and commercialization of a novel life-altering therapeutic for debilitating and often fatal orphan 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.

Forward-Looking Statements

This press release contains forward-looking statements which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including statements regarding timing of the Company's planned regulatory filings with respect to lomitapide, and the potential for regulatory approval and launch of lomitapide. 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 the Company's 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 unexpected technical or regulatory issues may delay filing; the risk that the Company's regulatory filings may not be accepted by the applicable regulatory authorities or that such acceptance may be delayed; the risk that FDA or other applicable regulatory authorities may ask for additional data, information or studies to be completed or provided prior to approval; the risk that the FDA or other applicable regulatory authorities may require additional work related to the commercial manufacturing process to be completed prior to approval or may, in the course of the inspection of manufacturing facilities, identify issues to be resolved; the risks that the FDA or other applicable regulatory authorities may not be satisfied with the safety profile of lomitapide; and the risk that the Company does not receive approval of lomitapide in the U.S. or EU on a timely basis or at all. For additional disclosure regarding these and other risks faced by the Company, see the disclosure contained in the Company's public filings with the Securities and Exchange Commission, including the Company's most recent Quarterly Report on Form 10-Q under the heading "Risk Factors" and available on the SEC's website at http://www.sec.gov. The Company undertakes 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.

CONTACT: Aegerion Pharmaceuticals, Inc.


         Mark Fitzpatrick, CFO

         +1 (617) 500-6235


         LaVoie Group, Inc.

         Investors & Media

         Donna LaVoie or Amanda Murphy

         +1 (978) 745-4200 x103/107



Close window | Back to top

Copyright 2016 Aegerion Pharmaceuticals, Inc.