New Clinical Data Analyses Show Metreleptin Reduced Blood Glucose and Triglyceride Levels in Patients with Rare Forms of Lipodystrophy
Results from an Ongoing Clinical Study Presented at ENDO 2012
HOUSTON, June 25, 2012 — Amylin Pharmaceuticals, Inc.
(Nasdaq: AMLN) today announced findings from an ongoing clinical
study (expanded access protocol) that shows treatment with
metreleptin, an investigational medication under evaluation for the
treatment of diabetes and/or hypertriglyceridemia (high levels of
triglycerides in the bloodstream) in patients with rare forms of
inherited or acquired lipodystrophy, reduced blood glucose and
triglyceride levels. These findings were presented today in two
separate posters at the Endocrine Society’s 94th Annual
Meeting and Expo (ENDO) in Houston, Texas.
Lipodystrophy is an “ultra-orphan” rare disease
estimated to affect a few thousand people worldwide, which often
manifests with an early age of onset, has life-threatening
metabolic complications, and for which there is a significant unmet
medical need as there are no approved drugs that effectively treat
the underlying cause(s) of the disease.
“The data analyses presented in patients with rare forms of
lipodystrophy show that metreleptin lowered blood glucose and
triglyceride levels regardless of leptin levels and associated
autoimmune diseases,” said Elif Oral, M.D., University of
Michigan, Ann Arbor, Michigan, and a principal investigator in the
expanded access protocol for metreleptin. “In patients with
lipodystrophy, fat typically accumulates in the blood and organs
such as liver and muscle, which can lead to life-threatening
complications including insulin-resistant diabetes and
hypertriglyceridemia. If approved, metreleptin would be the first
therapy indicated specifically for the treatment of diabetes and/or
hypertriglyceridemia in patients with inherited or acquired
lipodystrophy.”
Findings from the first poster titled, “Metabolic Effects of
Metreleptin Treatment in Familial Partial Lipodystrophy
(FPL),” included data from an ongoing expanded access
protocol of metreleptin administration in patients with rare forms
of inherited or acquired lipodystrophy. This analysis included 19
patients with diabetes and/or high triglyceride levels who were
diagnosed clinically with FPL, one of the major subtypes of
lipodystrophy, without using a threshold leptin level as an
eligibility criterion. Overall, A1C, a measure of average blood
sugar over three months, and triglyceride levels improved with
metreleptin treatment, and metabolic benefits were observed across
a range of leptin levels. In patients with A1C ≥7% (14/19), A1C
decreased by 0.4±0.6% at six months (n=10) and
1.0±0.7% at 12 months (n=7). In patients with triglyceride
levels ≥200 mg/dL at baseline (12/19), triglyceride levels
decreased by 31±28 mg/dL at six months (n=6) and
184±127 mg/dL at 12 months (n=5). While on metreleptin, 2 of
12 patients on oral antidiabetic agents (OAD) were able to
discontinue an OAD, and 4 of 9 patients on insulin reduced their
insulin dose by 20% or more. Metreleptin was generally well
tolerated.
Findings from the second poster titled, “Metreleptin
Treatment in Acquired Generalized Lipodystrophy: Consistent
Metabolic Effects in Three Patients Presenting with Distinct
Autoimmune Conditions,” focused on three pediatric patients
with acquired generalized lipodystrophy (AGL) who were studied
under the same expanded access protocol. AGL is another major
lipodystrophy subtype that is commonly associated with autoimmune
diseases. These three patients with AGL also had active autoimmune
disease, including juvenile dermatomyositis, autoimmune hepatitis,
or Graves’ disease (a form of auto-immune thyroid disease).
With metreleptin treatment, each patient experienced substantial
reductions in lipodystrophy-related metabolic abnormalities, such
as diabetes or high triglyceride levels, with no evidence of
worsening of their respective autoimmune diseases.
About Lipodystrophy
Fat tissue is a major endocrine organ producing important metabolic
hormones such as leptin. People with lipodystrophy lack the
required fat tissue for normal metabolic function. This can be
partial, affecting select areas of the body, or generalized,
affecting nearly the entire body. A lack of fat tissue can lead to
relative deficiency of leptin.
Without adequate leptin function, the metabolic system, which
regulates food intake and the storage and break-down of dietary fat
and carbohydrates, falls out of balance. As a result, fat
accumulates in the blood and organs such as liver and muscle, which
can lead to life-threatening complications including
insulin-resistant diabetes and hypertriglyceridemia (high levels of
triglycerides in the bloodstream).
There are no approved drugs that address the underlying relative
leptin deficiency that is believed to contribute in large part to
the metabolic abnormalities that occur in lipodystrophy. Currently
available therapies for diabetes and hypertriglyceridemia are often
rendered marginally effective due to the severity of the condition.
More information on lipodystrophy can be found at
www.mylipodystrophy.com.
About Metreleptin
Metreleptin, an analog of the human hormone leptin, is a unique
potential therapy for certain metabolic disorders in patients with
rare forms of inherited or acquired lipodystrophy. Metreleptin is
believed to work in part by reducing fat accumulation in organs,
thereby improving insulin sensitivity. Clinical studies have been
conducted by investigators at the National Institutes of Health
(NIH) and other academic institutions in the U.S., Europe, and
Japan to determine whether metreleptin can improve glycemic control
and hypertriglyceridemia in patients with lipodystrophy.
In December 2010, Amylin initiated its rolling biologics license
application (BLA) submission to the U.S. Food and Drug
Administration (FDA) for the use of metreleptin to treat diabetes
and/or hypertriglyceridemia in pediatric and adult patients with
rare forms of lipodystrophy. In April 2012, Amylin submitted the
chemistry, manufacturing and controls section of the BLA. Amylin
recently announced that the FDA is seeking updated data from the
submitted clinical studies that remain ongoing. Amylin is committed
to responding in a timely fashion to enable the FDA to complete its
evaluation of the rolling BLA submission.
About Amylin Pharmaceuticals
Amylin Pharmaceuticals is a biopharmaceutical company dedicated to
improving lives of patients through the discovery, development, and
commercialization of innovative medicines. Amylin is committed to
delivering novel therapies that transform the way diabetes and
other metabolic disorders are treated. Amylin is headquartered in
San Diego, Calif. and has a commercial manufacturing facility in
Ohio. More information about Amylin Pharmaceuticals is available at
www.amylin.com.
This press release contains forward-looking statements about
Amylin, which involve risks and uncertainties. Our actual results
could differ materially from those discussed herein due to a number
of risks and uncertainties, including risks that metreleptin as a
potential treatment option for diabetes and/or hypertriglyceridemia
in patients with rare forms of lipodystrophy will not be approved
by the FDA; risks that the FDA will not accept the BLA for filing;
risks that our clinical trials will not be completed when planned,
may not replicate previous results, may not be predictive of real
world use or may not achieve desired end-points; risks that our
preclinical studies may not be predictive; and other risks inherent
in the drug development and commercialization process. These and
additional risks and uncertainties are described more fully in the
Company's recently filed Form 10-Q. Amylin disclaims any obligation
to update these forward-looking statements.
Posted: June 2012
