SAN DIEGO, April 15, 2011 /PRNewswire/ -- Amylin
Pharmaceuticals, Inc. (Nasdaq: AMLN) today announced results from a
new analysis of an ongoing, long-term research study of the
investigational drug metreleptin, an analog of the human hormone
leptin, for the treatment of lipodystrophy. The study is being
conducted by investigators from the National Institute of Diabetes
and Digestive and Kidney Diseases (NIDDK) at the National
Institutes of Health (NIH). Results from the analysis will be
presented by Christian Weyer, M.D.,
senior vice president, research and development, Amylin
Pharmaceuticals, at a late-breaking oral session on Sunday, April 17 at 7:55
a.m. PT at the 20th Annual Meeting and Clinical Congress of
the American Association of Clinical Endocrinologists (AACE) in
San Diego.
In the study, which has been ongoing for more than 10 years,
researchers at the NIH are examining the effect of metreleptin on
several metabolic abnormalities, including diabetes and
hypertriglyceridemia (high levels of triglycerides in the
bloodstream), in patients with rare inherited or acquired forms of
lipodystrophy. Treatment with metreleptin resulted in robust
reductions from baseline in both A1C and triglycerides. (A1C is a
measure of average blood sugar over three months; triglycerides are
the major form of fat stored in the body.) These improvements were
apparent at four months and generally sustained for up to several
years of treatment.
"Patients with rare forms of lipodystrophy are often affected by
the disease early in life, are at high risk of acute and chronic
complications and, currently, have very limited therapeutic
options," said Dr. Weyer. "The results of our analysis reaffirm the
potential of metreleptin as an important advance for people living
with this chronic and often debilitating metabolic disease. We
continue to work diligently toward bringing this potential therapy
to market."
Study Findings
The findings involve an analysis of 55 lipodystrophy patients
who received metreleptin treatment – the largest lipodystrophy
cohort reported to date. At baseline, 75 percent of patients had
diabetes and were not achieving adequate glycemic control (A1C
greater than or equal to 7 percent), and 75 percent of patients had
hypertriglyceridemia (triglycerides greater than or equal to 200
mg/dL). Metreleptin treatment resulted in robust reductions from
baseline in both measures, an effect that was evident within four
months post treatment initiation and sustained for up to several
years of treatment. In patients with diabetes, mean A1C decreased
from 9.4 percent at baseline to under 7.0 percent at year three; in
patients with hypertriglyceridemia at baseline, median triglyceride
concentrations decreased from 500 mg/dL at baseline to under 200
mg/dL at year three.
Adverse events were generally consistent with known co-morbid
conditions of lipodystrophy (pancreatitis, proteinuria,
autoimmune/chronic hepatitis) or expected pharmacological effects
of metreleptin (weight loss or insulin-induced hypoglycemia in the
setting of improved insulin sensitivity in patients on high doses
of insulin).
About Lipodystrophy
Lipodystrophy syndromes are characterized by abnormalities in
adipose (fat) tissue distribution. Because patients with
lipodystrophy do not have enough fat tissue, they typically also
have a deficiency of leptin, a hormone secreted by fat cells that
plays a key role in regulating metabolism. Beginning typically in
childhood or adolescence, patients affected by lipodystrophy
experience a loss of subcutaneous fat, which can result in
multiple, often severe metabolic abnormalities, including extreme
insulin resistance, very high triglyceride levels,
difficult-to-control diabetes and hepatic steatosis (excess fat
accumulation in the liver). These abnormalities put patients at a
high risk for serious medical problems such as acute pancreatitis,
accelerated atherosclerosis, and blood vessel and nerve damage from
diabetes and liver cirrhosis, which can markedly reduce quality of
life and life expectancy. Metreleptin works to treat metabolic
abnormalities in patients with lipodystrophy.
It is estimated that there are a few thousand patients worldwide
with this condition, although robust epidemiological data are not
available, as is common with rare diseases. There are no therapies
currently indicated specifically for the treatment of metabolic
abnormalities associated with lipodystrophy. Presently, patients
may receive a combination of dietary modification, anti-diabetic
medications and lipid-lowering agents. These traditional treatment
approaches do not address the underlying cause of the metabolic
abnormalities in lipodystrophy and are often rendered marginally
effective due to the severity of the condition.
About Metreleptin for Lipodystrophy
Data from clinical studies conducted by investigators at the NIH
and other academic institutions in the U.S., Europe and Japan, have demonstrated that metreleptin can
have profound effects on improving insulin sensitivity, high
trigylcerides, hyperglycemia and liver fat in patients with
lipodystrophy who are not responsive to conventional lipid and
glucose-lowering agents, even those undergoing intensive insulin
therapy.
Globally, approximately 150 patients with lipodystrophy are
being treated with metreleptin under investigator-sponsored trials
and expanded access programs.
In 2010, Amylin submitted the non-clinical and clinical sections
of a rolling submission for a Biologics License Application (BLA)
to the U.S. Food and Drug Administration (FDA) for the use of
metreleptin to treat diabetes and/or hypertriglyceridemia in
patients with rare inherited or acquired forms of lipodystrophy.
The Company plans to submit the chemistry, manufacturing and
controls (CMC) section of the BLA by the end of this year, which
will complete the application. If approved, metreleptin would be
the first therapy indicated specifically for the treatment of
diabetes and high triglycerides in patients with lipodystrophy, and
the first approved therapeutic use of metreleptin.
About Amylin Pharmaceuticals, Inc.
Amylin Pharmaceuticals is a biopharmaceutical company dedicated
to improving lives of patients through the discovery, development
and commercialization of innovative medicines. Amylin has developed
and gained approval for two first-in-class medicines for diabetes,
SYMLIN® (pramlintide acetate) injection and BYETTA® (exenatide)
injection. Amylin's research and development activities leverage
the Company's expertise in metabolism to develop potential
therapies to treat diabetes and obesity. Amylin is headquartered in
San Diego, California. Further
information on Amylin Pharmaceuticals is available at
www.amylin.com.
This press release contains forward-looking statements about
Amylin which involve risks and uncertainties. The actual
results for Amylin could differ materially from those discussed due
to a number of risks and uncertainties, including that the CMC
section of the metreleptin BLA mentioned in this press release may
not be submitted in a timely fashion, the estimate of the number of
lipodystrophy patients mentioned in this press release may not be
accurate, clinical trials or studies may not start when planned,
confirm previous results, be predictive of real world use or
achieve intended clinical endpoints; preclinical studies may not be
predictive; our product candidates, including the product candidate
mentioned in this press release, may not receive regulatory
approval; and inherent scientific, regulatory and other risks in
the drug development and commercialization process. These and
additional risks and uncertainties are described more fully in
Amylin's most recently filed SEC documents, including its Annual
Report on Form 10-K. Amylin undertakes no duty to update these
forward-looking statements.
SOURCE Amylin Pharmaceuticals, Inc.