Profile: Aegerion Pharmaceuticals Inc (AEGR.O)

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Aegerion Pharmaceuticals, Inc. (Aegerio), incorporated in February 2005, is a biopharmaceutical company focused on the development and commercialization of therapeutics to treat lipid disorders. As of December 31, 2011, the Company developed its first product candidate, lomitapide, as an oral, once-a-day treatment for patients with a inherited lipid disorder called homozygous familial hypercholesterolemia (HoFH). The Company conducted a 78-week Phase III clinical trial of lomitapide in the treatment of adult patients with HoFH. The 56-week results of the trial were announced in May 2011. It completed the trial during the year ended December 31, 2011, and in January 2012, announced the 78-week results of the trial, which were consistent with the 56-week results. In 2011, the United States Food and Drug Administration (FDA) granted orphan drug designation for lomitapide in the treatment of HoFH. The Company’s lead product compound, lomitapide, is a small molecule microsomal triglyceride transfer protein (MTP), inhibitor (MTP-I), that the Company developed as an oral once-a-day treatment for patients with certain severe lipid disorders in 2011. Lomitapide has been evaluated in 14 Phase I and eight Phase II clinical trials, as well as the Phase III clinical trial completed in 2011. Approximately 940 patients have been treated with lomitapide as part of these clinical trials. As of December 31, 2011, it developed lomitapide as an oral, once-a-day treatment for patients with HoFH and familial chylomicronemia (FC).

The Company’s Phase III clinical trial studying lomitapide in the treatment of HoFH was a single-arm, dose titration, open-label clinical trial with a 78-week treatment period. The trial was conducted at 11 sites in four countries. A total of 29 patients enrolled in the trial. The patients in the trial were adult males and females with a mean age of 31. After a six week run-in period to stabilize lipid lowering therapy (including apheresis if applicable) and diet, patients were given ascending doses of lomitapide beginning at 5 milligram per day, and then escalated individually up to their maximum tolerated dose, not to exceed 60 milligram per day, over the first 26 weeks of the clinical trial. Patients were then maintained at their maximum tolerated dose for an additional 52-week safety phase. The efficacy and safety phases combined lasted 78 weeks. After this time, eligible patients were given the option to enroll in a separate protocol for a long-term, open-label extension trial to evaluate the long-term efficacy and safety of lomitapide at the maximum tolerated dose beyond 78 weeks. For patients who did not enter the optional open-label extension trial, there was a six-week wash-out period during which lomitapide was discontinued, and patients remained on concomitant lipid-lowering therapy.

In October 2011, the last patient enrolled in the study completed the 78-week treatment period, and the trial concluded. The primary efficacy endpoint of this trial was percent change in LDL-C at the maximum tolerated dose compared to baseline after 26 weeks of treatment in combination with other lipid lowering therapies. The secondary endpoints of this trial included the evaluation of other lipid parameters, including percent change in TG levels from baseline, long-term safety, and percent change in hepatic fat, as measured by magnetic resonance spectroscopy (MRS). Of the 29 patients originally enrolled in the trial, three patients withdrew their consent to participate in the trial and three patients discontinued treatment due to gastrointestinal adverse events in each case during the first 26 weeks. The 23 patients remaining in the trial completed the 26-week period of therapy after which the primary efficacy endpoint was measured, the 56-week period of therapy during which the 56-week results were collected, and the entire 78 weeks of the trial. In 2011, the Company treated one patient with lomitapide for severe hypertriglyceridemia under the FDA’s compassionate use program, through which a licensed physician may request from a manufacturer an investigational drug for the diagnosis, monitoring or treatment of a serious disease or condition in an individual patient. Based on the TG reductions seen in this, patient and the TG reductions seen in other clinical trials of lomitapide, lomitapide had the potential for treating patients suffering from high levels of TGs leading to pancreatitis. In March 2011, the FDA also granted orphan drug designation for lomitapide in the treatment of FC.

Company Address

Aegerion Pharmaceuticals Inc

1 Main St Ste 800
CAMBRIDGE   MA   02142-1531
P: +1617.5007867
F: +1617.9457968

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