|
< back to headlines
Aegerion Pharmaceuticals, Inc. Announces AEGR-733 Phase II
Data Demonstrates Significant Lowering of LDL Cholesterol
with Promising Hepatic Safety Profile
Preliminary Phase III Data Also Shows Signs of Efficacy,
Safety and Tolerability in
Most Severe Patient Population
BRIDGEWATER, NJ
(November 6, 2008) — Aegerion Pharmaceuticals,
Inc., a biopharmaceutical company focused on the treatment of
cardiovascular and metabolic disease, today announced top-line
data
from three separate Phase II trials involving its lead
cholesterol management compound, AEGR-733, which is a
microsomal triglyceride transfer protein (MTP) inhibitor.
All three trials were designed to evaluate the
efficacy, safety and tolerability of low doses of AEGR-733
alone and in combination with other lipid lowering agents such
as Lipitor, Zetia and fenofibrate.
The largest trial also included magnetic resonance
spectroscopy (MRS) to quantify patients’ hepatic fat levels.
Preliminary data from the trials indicates
statistically significant reductions in patients’
low-density-lipoprotein cholesterol (LDL-C) vs. baseline,
while at the same time suggesting a promising safety and
tolerability profile, including low levels of hepatic fat
accumulation.
The three Phase II 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 lipid levels in the bloodstream. During the trials,
AEGR-733 was administered alone as a once-daily pill in doses
ranging from 2.5 mg to 10 mg and also in combination with
other lipid lowering agents.
At the high end of the dose range evaluated in these
Phase II trials, the drug reduced LDL-C in patients up to 35%
from baseline when used as a monotherapy, and up to 66% from
baseline when administered in combination with Lipitor.
In addition to reducing LDL-C, patients also
experienced a reduction in their triglyceride levels by up to
50% and weight loss of up to 3% after 12 weeks on therapy.
The trials also suggested a promising safety and tolerability
profile. In one of
the trials, which was designed to evaluate patients’ hepatic
fat levels while treated with AEGR-733 alone and in
combination with Lipitor, Zetia and fenofibrate, the average
hepatic fat levels after 12 weeks of exposure across doses of
AEGR-733 ranging from 2.5 to 10 mg were approximately 7% with
no arm exceeding 10%.
This preliminary data compares favorably to what the
Company believes are the relevant clinical benchmarks for
hepatic fat.
Across all trials, less than 2% of the patients experienced
adverse events related to elevated liver enzyme levels where
such events resulted in their discontinuation from the
studies. This
compares favorably to what the Company believes are the
relevant clinical benchmarks for elevated liver enzymes,
statins and fenofibrate.
When titrated from a starting dose of 2.5 mg of
AEGR-733, less than 5% of patients experienced
gastrointestinal adverse events leading to discontinuation
from the trial, which the Company believes highlights the
importance of titration as an important component of
administration of AEGR-733 to reduce such adverse events.
Bill Sasiela, Chief Medical Officer of Aegerion
Pharmaceuticals, said, “We are pleased with the results of
these trials because they suggest the ability of AEGR-733 at
low doses to significantly reduce LDL cholesterol and
triglycerides with what we believe to be an acceptable safety
and tolerability profile.
We hope to confirm and extend these observations in our
Phase III studies and believe that AEGR-733 could become an
important part of the treatment regimen for dyslipidemic
patients who have limited treatment options today.”
The top-line findings described above represent preliminary
results from these studies. The Company expects to present
more complete trial results at appropriate scientific meetings
and in peer-reviewed publications at a later date.
Aegerion also released preliminary results from its ongoing
open-label Phase III study of AEGR-733.
This pivotal study focuses on patients suffering from a
rare genetic disorder characterized by high cholesterol levels
known as homozygous familial hypercholesterolemia (HoFH).
In light of this patient population’s extremely high
LDL-C levels, the Company believes higher doses of AEGR-733
may be an appropriate therapeutic option for these patients.
In this ongoing study, patients are being treated with
AEGR-733 in doses titrated up to 60 mg per day.
Preliminary data
reveals LDL-C reductions of greater than 50% (beyond the
existing reductions patients experienced on background
therapy, such as statins and cholesterol absorption
inhibitors) in the majority of patients that have reached high
dosages in the study.
One patient was able to discontinue plasma apheresis
(mechanical filtration of the blood used to temporarily remove
extremely high levels of LDL-C).
The reduction in LDL-C levels is consistent with the
more than 50% reduction seen when AEGR-733 was used as a
monotherapy in a separate Phase II study, the results of which
were published in the New England Journal of Medicine in
January 2007. To date, safety and tolerability in these
patients have been promising with no discontinuations from the
study. Final results from this ongoing Phase III trial will be
forthcoming upon study completion.
Will Lewis, Co-Founder and Chief Financial Officer of Aegerion,
said, “We are encouraged by the promising results of these
Phase II and Phase III trials, and we look forward to
maximizing the potential of this therapy across the spectrum
of patient need.
We will be exploring several options in the immediate future
to enable development of what the Company believes to be a
promising new therapeutic option with the ability to safely
lower LDL-C and triglycerides and contribute to weight loss.”
About AEGR-733
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.
About Aegerion
Pharmaceuticals, Inc.
Aegerion Pharmaceuticals, Inc. is a privately held
biopharmaceutical company focused on the development and
commercialization of promising pharmaceuticals to treat
cardiovascular and metabolic disease. The Company’s primary
focus is on hyperlipidemia. Its most advanced products have
demonstrated significant LDL lowering activity in human trials
and are currently in Phase III and Phase II testing.
Company Contact:
Aegerion Pharmaceuticals, Inc.
Will Lewis, Co-Founder and Chief Financial Officer
(908) 707-2100
Media Contact:
Sard Verbinnen & Co.
Jim Barron/Lesley Bogdanow/Nick Lawler
(212) 687-8080
###
|
