Aegerion Pharmaceuticals Announces Final Lomitapide (AEGR-733) Phase II Data

Main Category: Cholesterol
Also Included In: Clinical Trials / Drug Trials
Article Date: 18 Jun 2009 - 0:00 PDT

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Aegerion Pharmaceuticals, Inc., a biopharmaceutical company focused on the treatment of cardiovascular and metabolic disease, announced final data from three separate Phase II trials involving its lead cholesterol management compound, lomitapide (AEGR-733), which is a microsomal triglyceride transfer protein inhibitor (MTP-I) small molecule drug. The final data was recently presented at the American Diabetes Association's "69th Scientific Sessions" in New Orleans, LA on June 7, 2009 and the International Symposium on Atherosclerosis (ISA) in Boston, MA on June 15, 2009.

The three 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 levels of lipids in the bloodstream. All three Phase II trials examined the efficacy, safety and tolerability of lomitapide when administered in low doses, ranging from 2.5mg to 10mg, both alone and in combination with lipid lowering therapies (LLTs) such as Lipitor, Zetia and fenofibrate.

The final data confirm the preliminary results Aegerion announced in November 2008 and demonstrate the strong efficacy, safety and tolerability profile of lomitapide, including:

- Significant reductions in patients' low-density-lipoprotein cholesterol (LDL-C) when administered at the high end of the tested dose range; patients experienced reductions of up to 35% vs. baseline when used as a monotherapy and up to 66% from baseline in combination with Lipitor

- Good additivity in LDL-C reduction when combined with Zetia and fenofibrate

- Strong decreases in triglyceride levels of up to 40% when used as a monotherapy and up to 50% in combination with Lipitor

- Weight loss of up to 3.0% in generally overweight patients (BMIs of 25-30 kg/m2) from the study after 12 weeks on therapy

- A rate of discontinuation due to liver function test (LFT) elevations of less than 2.0%

- Modest increases in hepatic fat with mean levels of 5.1-9.8% and median levels of 2.4-8.0%

- Minimal gastrointestinal adverse event discontinuations (less than 5.0%) when dose titration is utilized

Bill Sasiela, Chief Medical Officer of Aegerion Pharmaceuticals, said, "We are pleased to receive confirmation of our original findings from November, which suggest that lomitapide can safely and effectively lower LDL-C and triglycerides in patients with elevated lipids. Importantly, the full collection of Phase II trials conducted in the low end of lomitapide's dose range, which involved nearly 550 patients, indicate that patients can achieve significant reductions in LDL-C when using lomitapide with statin therapies like Lipitor. Additionally, these data show that combining lomitapide with other non-statin drugs like Zetia could provide meaningful LDL-C reductions in patients that cannot tolerate statin therapy. In both cases, we see an improved safety profile over prior high dose MTP-I development efforts."

As announced on June 15, 2009, Aegerion is currently conducting a Phase III trial designed to evaluate the efficacy, safety and tolerability of lomitapide for the treatment of patients with Homozygous Familial Hypercholesterolemia (HoFH), a rare and extremely serious condition resulting in severely elevated levels of LDL cholesterol which leads to life-threatening cardiovascular events. The preliminary data from the trial indicate statistically significant reductions in patients' LDL-C vs. baseline, while at the same time reporting a promising safety and tolerability profile, including low levels of hepatic fat accumulation. For more information on this announcement, please visit http://www.aegerion.com.

About LOMITAPIDE (AEGR-733)

Lomitapide (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.

Source
Aegerion Pharmaceuticals, Inc.