CAMBRIDGE, Mass., Jan. 12, 2015 (GLOBE NEWSWIRE) -- Aegerion
Pharmaceuticals, Inc. (Nasdaq:AEGR) today announced that it has
completed the acquisition of MYALEPT(R) (metreleptin) for injection, an
orphan drug product that is indicated to treat complications of leptin
deficiency in patients with generalized lipodystrophy (GLD). Execution
of the asset purchase agreement setting forth the terms of the
acquisition was announced on November 6, 2014. Aegerion paid $325
million upfront to acquire the global rights to develop, manufacture
and commercialize MYALEPT, subject to an existing distributor license
with Shionogi covering Japan, South Korea and Taiwan.
MYALEPT is approved in the United States for the treatment of GLD, and
it has orphan drug designation in the United States, European Union and
Japan. MYALEPT is a recombinant analogue of human leptin, indicated in
the US as an adjunct to diet as replacement therapy to treat the
complications of leptin deficiency in patients with congenital or
acquired generalized lipodystrophy.
Marc Beer, chief executive officer of Aegerion, commented: "We are
excited to have an opportunity to build on AstraZeneca's progress and
commitment to delivering MYALEPT to GLD patients, with a continuing
commitment to excellence in patient support."
About Generalized Lipodystrophy
Lipodystrophy is a group of rare syndromes characterized by loss of fat
tissue. In some patients, it is genetic, and in others it may be
acquired for different pathophysiological, and in some cases unknown,
reasons. Generalized lipodystrophy is characterized by a widespread
loss of fat tissue under the skin. This loss of fat tissue causes a
deficit in the hormone leptin leading to multiple metabolic
complications.
MYALEPT(TM) (metreleptin) for injection
INDICATION and IMPORTANT SAFETY INFORMATION for MYALEPT(TM)
(metreleptin) for injection
INDICATION
MYALEPT(TM) (metreleptin) for injection is a recombinant human leptin
analog indicated as an adjunct to diet as replacement therapy to treat
the complications of leptin deficiency in patients with congenital or
acquired generalized lipodystrophy.
LIMITATIONS OF USE
-- The safety and effectiveness of MYALEPT (metreleptin) for injection for
the treatment of complications of partial lipodystrophy or for the
treatment of liver disease, including nonalcoholic steatohepatitis
(NASH), have not been established.
-- MYALEPT is not indicated for use in patients with HIV-related
lipodystrophy or in patients with metabolic disease, including diabetes
mellitus and hypertriglyceridemia, without concurrent evidence of
congenital or acquired generalized lipodystrophy.
IMPORTANT SAFETY INFORMATION
WARNING: RISK OF ANTI-METRELEPTIN ANTIBODIES WITH NEUTRALIZING ACTIVITY
AND RISK OF LYMPHOMA
-- Anti-metreleptin antibodies with neutralizing activity have been
identified in patients treated with MYALEPT. The consequences of these
neutralizing antibodies are not well characterized but could include
inhibition of endogenous leptin action and/or loss of MYALEPT efficacy.
Severe infection and/or worsening metabolic control have been reported.
Test for anti-metreleptin antibodies with neutralizing activity in
patients who develop severe infections or show signs suspicious for loss
of MYALEPT efficacy during treatment. Contact 1-866-216-1526 for
neutralizing antibody testing of clinical samples.
-- T-cell lymphoma has been reported in patients with acquired generalized
lipodystrophy, both treated and not treated with MYALEPT. Carefully
consider the benefits and risks of treatment with MYALEPT in patients
with significant hematologic abnormalities and/or acquired generalized
lipodystrophy.
-- Because of these risks associated with the development of
anti-metreleptin antibodies that neutralize endogenous leptin and/or
MYALEPT and the risk for lymphoma, MYALEPT is available only through a
restricted program under a Risk Evaluation and Mitigation Strategy (REMS)
called the MYALEPT REMS PROGRAM.
CONTRAINDICATIONS
MYALEPT (metreleptin) for injection is contraindicated in patients
with:
-- General obesity not associated with congenital leptin deficiency. MYALEPT
has not been shown to be effective in treating general obesity, and the
development of anti-metreleptin antibodies with neutralizing activity has
been reported in obese patients treated with MYALEPT
-- Prior severe hypersensitivity reactions to metreleptin or to any of the
product components. Known hypersensitivity reactions have included
urticaria and generalized rash.
WARNINGS AND PRECAUTIONS
Risk for Development of Antibodies that Neutralize Endogenous Leptin
and/or MYALEPT
Anti-metreleptin antibodies with in vitro neutralizing activity to
leptin associated with adverse events consistent with loss of
endogenous leptin activity and/or loss of efficacy have been identified
in two patients with generalized lipodystrophy treated with MYALEPT
(severe infections, increases in HbA1c and triglycerides), and in three
patients without lipodystrophy who received MYALEPT in clinical studies
(excessive weight gain, development of glucose intolerance or diabetes
mellitus). The clinical implications associated with development of
anti-metreleptin antibodies with neutralizing activity are not
well-characterized at this time due to the small number of reports.
Test for anti-metreleptin antibodies with neutralizing activity in
patients who develop severe infections or show signs suspicious for
loss of MYALEPT efficacy during treatment.
Lymphoma
-- Three cases of T-cell lymphoma have been reported in the MYALEPT
lipodystrophy program; all three patients had acquired generalized
lipodystrophy. Two of these patients were diagnosed with peripheral
T-cell lymphoma while receiving MYALEPT. Both had immunodeficiency and
significant hematologic abnormalities including severe bone marrow
abnormalities before the start of MYALEPT treatment. A separate case of
anaplastic large cell lymphoma was reported in a patient receiving
MYALEPT (metreleptin) for injection who did not have hematological
abnormalities before treatment.
-- Lymphoproliferative disorders, including lymphomas, have been reported in
patients with acquired generalized lipodystrophy not treated with
MYALEPT. A causal relationship between MYALEPT treatment and the
development and/or progression of lymphoma has not been established.
Acquired lipodystrophies are associated with autoimmune disorders, and
autoimmune disorders are associated with an increased risk of
malignancies including lymphomas.
-- The benefits and risks of MYALEPT treatment should be carefully
considered in patients with acquired generalized lipodystrophy and/or
those with significant hematologic abnormalities (including leukopenia,
neutropenia, bone marrow abnormalities, lymphoma and/or lymphadenopathy).
MYALEPT REMS Program
MYALEPT is available only through a restricted distribution program
under a REMS, called the MYALEPT REMS Program, because of the risks
associated with the development of anti-metreleptin antibodies that
neutralize endogenous leptin and/or MYALEPT and the risk for lymphoma
[see Warnings and Precautions section].
Further information is available at www.myaleptrems.com or
1-855-6MYALEPT.
About Aegerion
Aegerion Pharmaceuticals is a biopharmaceutical company dedicated to
the development and commercialization of innovative therapies for
patients with debilitating rare diseases. For more information about
the company, please visit www.aegerion.com.
Forward-Looking Statements
This press release contains forward-looking statements, including
statements regarding: Aegerion's opportunity to build the MYALEPT
business. These forward-looking statements are neither promises nor
guarantees of future performance, and are subject to a variety of risks
and uncertainties, many of which are beyond Aegerion's control, which
could cause actual results to differ materially from those contemplated
in these forward-looking statements. In particular, the risks and
uncertainties related to building the MYALEPT business include, among
others: our ability to smoothly transition MYALEPT operations; our
ability to build and to maintain market acceptance for MYALEPT in the
U.S. for the treatment of GLD; observation of a side effect profile for
MYALEPT in commercial use and in further clinical studies that
continues to be manageable, and that is generally consistent, in scope
and severity, with the side effect profile observed in the pivotal
study, including the percentage of patients experiencing
anti-metreleptin antibody formation with MYALEPT in commercial use
being not significantly different than that observed in the pivotal
study; the prevalence of GLD being consistent with management's
estimates of one person in a million which is based on assumptions
which may prove not to be accurate; our ability to be able to sell
MYALEPT on a named patient sales basis in key markets outside the
United States, based on the U.S. approval, and the amount of revenues
generated from such sales; the willingness of payers and government
entities to provide reimbursement for MYALEPT at the prices we intend
to offer the product; our ability to gain regulatory approval for
MYALEPT as a treatment for GLD in key countries outside the U.S.
without restrictions that are substantially more onerous than those
imposed in the U.S., and without the need to conduct further clinical
studies; our ability to successfully develop MYALEPT in additional
indications; our ability to have sufficient quantities of each strength
of MYALEPT to meet demand; the strength of the patent portfolio and
marketing and data exclusivity for MYALEPT; the continued lack of
competitive challenges for MYALEPT in the treatment of GLD; and other
risks inherent in integration of a new product, and in
commercialization, drug development and the regulatory approval
process. Existing and prospective investors are cautioned not to place
undue reliance on these forward-looking statements, which speak only as
of the date of this press release.
For disclosure regarding other risks faced by Aegerion, see the
disclosure contained in the "Risk Factors" section of Aegerion's
Quarterly Report on Form 10-Q filed on November 10, 2014, and our other
public filings with the Securities and Exchange Commission, available
on the SEC's website at http://www.sec.gov.
Except as required by law, Aegerion undertakes no obligation to update
or revise the information contained in this press release, whether as a
result of new information, future events or circumstances or otherwise.
CONTACT: AEGERION CONTACT
Investors & Media
Amanda Murphy
Associate Director of Investor Relations & Public Relations
857-242-5024
[email protected]