Microsoft word - aegerion nejm jan 10.doc
New England Journal of Medicine Reports Aegerion's
Lead Compound Reduces LDL By 51% in Patients With a Severe Form of
Phase III Trials Planned in 2007 for
homozygous familial hypercholesterolemia patient population
Bridgewater, NJ (January 10, 2007) – Aegerion Pharmaceuticals, Inc., a specialty pharmaceutical
company focused on the treatment of cardiovascular and metabolic diseases, today announced the
publication of positive results from a second Phase II clinical trial of its lead cholesterol-lowering
compound AEGR-733 in the January 11, 2007 issue of the New England Journal of Medicine.
“The results of this study suggest that microsomal triglyceride transfer protein (MTP) inhibition with
AEGR-733 is a highly effective and potentially promising agent to treat these high-risk, hard-to-treat
patients, for whom there are few effective treatment options. It may also have applicability in a much
broader population of patients — those who are not sufficiently clinically responsive to standard therapy
or are intolerant of statins,” said Daniel J. Rader, MD, Director of the Clinical and Translational Research
Center at the University of Pennsylvania School of Medicine and the lead investigator for this study.
“There are many patients who are not able to get to recommended levels with the therapies that are
The results demonstrate that AEGR-733 (previously known as BMS-201038) reduced the levels of low-
density lipoprotein (LDL) or “bad cholesterol” by a remarkable 51% from baseline in subjects with
homozygous familial hypercholesterolemia (FH), a genetic condition characterized by dangerously high
LDL-C levels. AEGR-733 was also effective in reducing total cholesterol levels by 58%, triglyceride
levels by 65%, and apolipoprotein B levels by 56% from baseline.
Researchers at the University of Pennsylvania School of Medicine conducted a dose-escalation study
examining the efficacy, safety, and tolerability of AEGR-733 in 6 patients with homozygous FH who
were titrated to the study’s highest dose. Patients received AEGR-733 at 4 different doses, each for 4
weeks, and returned for a final visit after an additional 4-week drug washout period. Analysis of lipid
levels, safety laboratory analyses, and magnetic resonance imaging of the liver for hepatic fat content
were performed throughout the study. The most common adverse events were loose
stool/diarrhea. Elevation of liver transaminase levels and accumulation of hepatic fat were seen in
some, but not all, of the study patients.
As a result of their dangerously high LDL-C levels, patients with homozygous FH are at an increased risk
of premature cardiovascular disease and death, and existing agents are minimally effective in reducing
LDL-C in this population. AEGR-733 is an investigational MTP inhibitor, a new class of cholesterol-
lowering agents that inhibit the protein that produces low-density lipoprotein. AEGR-733 is expected to
enter Phase III trials later this year with continued development of the compound in the homozygous
FH population. A second developmental pathway will focus on the treatment of the broader
hypercholesterolemic population, who are either unable to achieve recommended cholesterol levels on
currently available therapies or cannot tolerate statin therapy.
Results from an earlier Phase II trial, involving lower doses of AEGR-733 alone or in combination with
ezetimibe (Zetia®)*, a cholesterol absorption inhibitor, in a hypercholesterolemic patient population
were presented at the American Heart Association Scientific Sessions Conference in November
2006. Patients treated with a combination of AEGR-733 plus ezetimibe achieved a 35% reduction in
LDL-C after 4 weeks. There was no difference in discontinuation rates between the patients who
received AEGR-733 in combination with ezetimibe or ezetimibe alone. In addition, AEGR-733 alone was
shown to be effective in reducing LDL-C. Ongoing Phase II development is aimed at continuing to
explore AEGR-733 in this broader hypercholesterolemic population.
“We are extremely encouraged by the results of this trial, as it provides a second proof of concept of
the potential of AEGR-733 to treat patients who are not able to reduce their cholesterol to
recommended levels with currently available agents,” said Jerry Wisler, President and Chief Executive
Officer of Aegerion Pharmaceuticals. “We are excited about the potential of AEGR-733, especially as
guidelines for target cholesterol levels continue to become more aggressive. Aegerion’s approach is to
develop compounds that complement existing therapies to help patients get to their goals. AEGR-733
shows promise as a potential new treatment for high cholesterol.”
* Zetia® (ezetimibe) is a registered trademark of MSP Singapore Company, LLC.
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. In the
United States alone, nearly 100 million adults have total blood cholesterol values of 200 mg/dL and
higher, and 34.5 million have levels of 240 and above. Current guidelines recommend that appropriate
LDL levels in patients at high risk of cardiovascular disease should be less than 100 mg/dL, or below 70
mg/dL for very high-risk patients. Under these guidelines, 36 million people would be eligible for
treatment. Standard therapies, such as statins and cholesterol absorption inhibitors, do not effectively
lower LDL-C to target levels in a significant number of patients.
Aegerion Pharmaceuticals, Inc. is a privately held specialty pharmaceutical company with a highly
experienced senior management team. The company is focused on the development and
commercialization of pharmaceuticals to treat cardiovascular and metabolic disease. Its most advanced
products are MTP inhibitors which have demonstrated significant clinical effects in Phase II trials in
patients with various forms of hyperlipidemia and whose cholesterol levels remain uncontrolled on
currently available dyslipidemia therapies. MTP inhibitors represent a potentially new therapeutic
approach for hyperlipidemia and a significant commercial opportunity for Aegerion.
CONTACT: Jeff McLaughlin, Vox Medica Health-care Public Relations Group
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