CAMBRIDGE, Mass., May 31, 2011 (GLOBE NEWSWIRE) -- Aegerion Pharmaceuticals, Inc. (Nasdaq:AEGR), today 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. The results are summarized below for all patients through week 56:
| Protocol Phase |
Week 26 (Efficacy) |
Week 56 (Safety) |
||
| Background Therapy | Fixed | Reduction Allowed | ||
| N | 29 | 23 | ||
| Average Dose (mg) | 44.6 | 40.2 | ||
|
LDL-C (mean % change from baseline) |
-40.1 [-50.2] |
-44.0 |
||
|
Triglycerides (median % change from baseline) |
-44.6 [-56.1] |
-33.3 |
||
|
Hepatic Fat (mean %) |
9.0* (Baseline 1.0) |
7.3** (Baseline 1.2) |
||
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.
*N=22
**N=21
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.
About Lomitapide
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. Lomitapide 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.
About the Lomitapide Phase III Clinical Trial
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.
About Aegerion Pharmaceuticals, Inc.
Aegerion Pharmaceuticals, Inc. (Nasdaq:AEGR) is an emerging biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat severe lipid disorders. The Company's lead product, lomitapide, is initially being developed to treat patients with a rare genetic lipid disorder called homozygous familial hypercholesterolemia, or HoFH, and is currently in Phase III development for this indication. The Company also plans to initiate a clinical program for lomitapide to treat patients with a severe genetic form of hypertriglyceridemia called familial chylomicronemia.
Forward-Looking Statements
This press release contains forward-looking statements which are made pursuant to the safe harbor provisions of Private Securities Litigation Reform Act of 1995, including statements regarding expected regulatory filings for and commercialization of the Company's lead product candidate, lomitapide. The forward-looking statements in this release do not constitute guarantees of future performance. These statements are neither promises nor guarantees, 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. The risks and uncertainties include, the Company's history of operating losses; the Company's potential need for additional capital to fund operations and develop its product candidates; uncertainties associated with the clinical development and associated regulatory filings of the Company's product candidates, including the risk that the Company's regulatory filings may be delayed or may not be accepted by the applicable regulatory authorities; the risk that the Company's product candidates may not be approved for any indication, or if approved; the risk that the finally approved definition of the targeted patient populations for the Company's product candidates may be narrower than expected; risks associated with undesirable side effects experienced by some patients in clinical trials for the Company's product candidates; risks associated with the Company's lack of sales and marketing experience; the highly competitive industry in which the Company operates; risks associated with the Company's intellectual property rights and the extent to which such intellectual property rights protect the Company's product candidates; the risk that third parties may allege that the Company infringes their intellectual property rights or that the Company has failed to comply with the provisions of its in-license agreements; risks associated with the Company's reliance on third parties, in particular clinical research organizations and contract manufacturers; risks associated with the Company's ability to recruit, hire and retain qualified personnel; and risks associated with volatility in the Company's stock price as a newly public company. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. 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. 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 recent Annual Report on Form 10-K under the heading "Risk Factors" and available on its investor relations website at http://www.aegerion.com and on the SEC's website at http://www.sec.gov.