Business Description Aegerion Pharmaceuticals is an emerging biopharmaceutical company focused on the
development and commercialization of drug products consisting of single
molecules, called small-molecule therapeutics, to treat cardiovascular and
metabolic disease. Our two product candidates are microsomal triglyceride
transfer protein inhibitors that limit secretion of cholesterol and
triglycerides, collectively referred to as lipids, from the intestine and liver
and have demonstrated in humans an ability to significantly reduce low-density
lipoprotein cholesterol, or LDL-C. We believe these compounds have the potential
to address significant unmet medical needs for patients who seek to lower their
cholesterol levels, either on a stand-alone basis or in combination with
existing therapies, such as statins and cholesterol absorption inhibitors.
Lipid-lowering therapeutics represent one of the largest markets in the
pharmaceutical industry, with as many as 52 million patients in the United
States alone eligible for therapy. Worldwide revenues for lipid-lowering
therapeutics were approximately $30 billion for the twelve months ended
December 31, 2006. However, significant unmet medical needs remain for patients
with elevated lipid levels, in particular LDL-C. Recent clinical trials
conducted by others add to the substantial evidence that there is a direct
relationship between lower LDL-C levels and decreased risk for major
cardiovascular events. These findings prompted the National Cholesterol
Education Program in 2004 to introduce optional LDL-C guidelines, endorsed by
the American Heart Association, the American College of Cardiology and others,
advising physicians to consider more intensive treatment options for people with
high and moderately high-risk of a cardiovascular event. These options include
setting lower treatment goals for LDL-C levels (including a LDL-C level of <70
mg/dL as a therapeutic option for very high-risk patients and a treatment goal
of <100 mg/dL for all high-risk patients) and initiating cholesterol-lowering
drug therapy at lower LDL-C thresholds. We believe these recommendations reflect
a trend toward more aggressive treatment of LDL-C levels in both high and
moderately high-risk patient populations, a significant percentage of whom will
not be able to achieve the lower LDL-C goals using current treatments. We
believe that this unmet medical need will become even more pronounced as lower
LDL-C goals become more widely adopted by the medical community.
Several recent clinical trials have shown that combination therapy with
multiple, differently acting compounds is the most effective way to achieve
these more aggressive LDL-C goals. For example, one recent clinical trial
demonstrated that use of Vytorin, a fixed-dose combination of the statin Zocor
(simvastatin) and the cholesterol absorption inhibitor Zetia (ezetimibe),
results in a greater percentage of high-risk patients reaching their LDL-C goal
than does the use of the leading marketed statin, Lipitor (atorvastatin),
alone. Despite the increased LDL-C lowering effect of Vytorin over statin
monotherapy, we believe millions of people may be unable to reach their optimal
level of LDL-C, and the number will increase significantly if the National
Cholesterol Education Program lowers the guidelines even further for people with
high and moderately high-risk of a cardiovascular event. We believe our product
candidates have the potential to take advantage of this trend towards
combination therapy in the lipid-lowering market.
One of our two product candidates, AEGR-733, is a microsomal triglyceride
transfer protein inhibitor that we are developing for the oral, once-a-day
treatment of individuals with high lipid levels, or hyperlipidemia, in
particular in those patients with high cholesterol, or hypercholesterolemia,
and/or high triglyceride levels, or hypertriglyceridemia, who are unable to meet
specified target goals. We believe AEGR-733 has the potential to address unmet
medical needs in a variety of patient populations, including the approximately
one-third of all patients in the United States at high-risk for coronary heart
disease, or approximately 8 million people, we estimate would not be able to
achieve a target LDL-C level of <70 mg/dL, even if the most efficacious forms of
currently available drug therapies were prescribed. In addition, there are an
estimated 1.5 million patients in the United States who are statin-intolerant
and unable to achieve goals on non-statin therapy, such as Zetia (ezetimibe),
one of the most effective non-statin treatments currently approved for the
treatment of high cholesterol. In a recently completed Phase II clinical trial,
AEGR-733 in doses of 5 mg to 10 mg in combination with Zetia (ezetimibe) 10 mg
resulted in an average reduction in LDL-C of 34.5% to 46.2% in patients with
hypercholesterolemia (versus approximately 20% for Zetia (ezetimibe) alone).
There was no difference in discontinuation rates between the patients treated
with the combination of AEGR-733 + Zetia (ezetimibe) 10 mg versus those treated
with Zetia (ezetimibe) alone. We believe the results of this trial demonstrate
the potential of AEGR-733, when used in combination with other non-statin
therapies such as Zetia (ezetimibe), to offer statin-like LDL-C lowering
efficacy for statin-intolerant patients.
We also believe that AEGR-733 may have advantages over Zetia (ezetimibe), both
as a monotherapy and particularly when used in combination with other drug
therapies. We believe the potential advantages may include: superior LDL-C
lowering efficacy; the ability to administer AEGR-733 at increasing doses for
greater LDL-C lowering efficacy due to a significant dose response curve,
whereas Zetia (ezetimibe) is only available at a single marketed dose; superior
triglyceride lowering efficacy; and the potential to induce weight loss. In a
recently completed Phase II clinical trial, AEGR-733 10 mg when used as a
monotherapy resulted in significantly greater LDL-C lowering efficacy than Zetia
(ezetimibe) 10 mg (29.9% vs. 19.9%, respectively). In 2007, we intend to
initiate additional Phase II trials of AEGR-733 to further evaluate its
potential role in reducing LDL-C, both as a monotherapy and in combination with
Lipitor (atorvastatin) and other lipid-lowering therapies.
We also believe AEGR-733 has the potential to address unmet medical needs in
niche patient populations, such as the approximately 30,000 patients in the
United States with high cholesterol levels that are resistant to treatment, a
condition known as severe refractory hypercholesterolemia. Patients with this
condition have elevated and refractory hypercholesterolemia to the point that
they are eligible to receive periodic plasma apheresis, or the mechanical
filtration of LDL-C particles from the blood, and often die at an early age. Of
these 30,000 patients, approximately 300 patients in the United States have a
rare genetic disorder called homozygous familial hypercholesterolemia resulting
in severely elevated levels of LDL-C (400-800 mg/dL). In January 2007, The New
England Journal of Medicine published the results of a Phase II clinical trial
of AEGR-733 in patients with homozygous familial hypercholesterolemia. In this
trial, patients receiving the highest dose of AEGR-733 (1.0 mg per kg of body
weight) as monotherapy achieved on average a 51% reduction in LDL-C. We expect a
Phase III clinical trial, to be conducted by the University of Pennsylvania
School of Medicine on our behalf, will be initiated in the second half of 2007,
to further study the effects of AEGR-733 in this patient population. In this
trial, AEGR-733 will be used in conjunction with other lipid-lowering therapies.
We expect AEGR-733 to be eligible for orphan drug designation for this patient
population and we intend to pursue such designation with the FDA.
Our second product candidate, AEGR-427 (implitapide), is also a microsomal
triglyceride transfer protein inhibitor that we are developing for the oral,
once-a-day treatment of hyperlipidemia. In the Phase II trial conducted by Bayer
Healthcare AG, implitapide 40 mg was shown to lower LDL-C by an average of 22%
and triglycerides by an average of 11%, with a discontinuation rate the same as
placebo (2%). In addition, based on prior clinical trials, we believe that
AEGR-427 (implitapide) may also have an impact on weight loss. In the second
half of 2007, we plan to initiate a pharmacokinetic trial, which involves
measuring the drug-to-drug interaction in the blood of healthy volunteers, to
study AEGR-427 (implitapide) in combination with a variety of statin therapies
as well as a dose ranging trial to evaluate its efficacy and safety/tolerability
profile in doses ranging from 30 mg to 50 mg.
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We were founded in 2005 as a Delaware corporation. Our principal executive
offices are located at CenterPointe IV, 1140 Route 22 East, Suite 304,
Bridgewater, New Jersey 08807, and our telephone number is (908) 704-1300. Our
web site address is www.aegerion.com. |