SAN DIEGO, June 4, 2011 /PRNewswire/ -- Amylin
Pharmaceuticals, Inc. (Nasdaq: AMLN) today announced results from a
study that showed treatment with metreleptin, an investigational
treatment that is an analog of the human hormone leptin, improved
diabetes and lipid control in patients with partial lipodystrophy.
Data from this study, the first findings from patients receiving
metreleptin through Amylin's lipodystrophy expanded access program,
were presented at the 93rd Annual Meeting and Expo of The Endocrine
Society (ENDO) in Boston.
In this study, being conducted under a treatment IND authorized
by the U.S. Food and Drug Administration (FDA), metreleptin is made
available to patients with rare forms of lipodystrophy who have one
or more metabolic abnormalities, including diabetes and
hypertriglyceridemia (high levels of triglycerides in the
bloodstream). This analysis focused on patients with partial
lipodystrophy who had received treatment for at least six months.
Treatment with metreleptin resulted in improvements from baseline
in A1C (a measure of average blood sugar over three months) and
triglycerides. Further, the majority of patients receiving
metreleptin were able to reduce or discontinue treatment with
pre-existing diabetes medications, including insulin.
"We are committed to assisting patients who are living with
lipodystrophy, a chronic and often debilitating disease that is not
adequately managed by existing therapies," said Christian Weyer, M.D., senior vice president,
research and development, Amylin Pharmaceuticals. "Our expanded
access program enables us to provide patients with metreleptin
while we continue working with the FDA to make the medicine more
broadly available to patients with this rare disorder."
Results of this analysis were presented by Suma Amarnath, M.D., a member of the research
team led by Elif Oral, M.D. during an oral presentation today at
ENDO 2011. Dr. Oral was instrumental in initiating the first
studies to investigate the therapeutic utility of leptin
replacement in lipodystrophy while she was working at the National
Institutes of Health (NIH). She is currently the Medical Director
of the University of Michigan Health
System (UMHS) Bariatric Surgery Program and Director of the UMHS'
Metabolism, Endocrinology and Diabetes (MEND) Obesity and Metabolic
Disorder Program.
Dr. Oral's team will also be presenting a poster at ENDO 2011
entitled, "Treatment of Severe Lipodystrophy with Metreleptin in a
Patient with Active Juvenile Dermatomyositis" on Sunday, June 5 at 1:30
p.m. ET.
Study Findings from Oral Presentation (OR07-3)
The findings of this study involve an analysis of nine patients
with partial lipodystrophy who received metreleptin treatment for
more than six months. At baseline, 89 percent were not achieving
adequate glycemic control (A1C greater than or equal to 7 percent),
and 89 percent had hypertriglyceridemia (triglycerides greater than
or equal to 150 mg/dL). Metreleptin treatment resulted in a
reduction in mean A1C from 8.1 percent at baseline to 6.8 percent
at six months, which was sustained through 15 months. Additionally,
at six months, patients who were taking insulin experienced an
average reduction of 110 units in their total daily insulin dose.
Similarly, mean triglyceride concentrations were reduced from 318
mg/dL at baseline to 169 mg/dL at 15 months.
Safety observations were generally consistent with those
observed in other studies, with the most common adverse events
being fatigue and nausea.
These results complement findings obtained in other studies
which, collectively, provide evidence to help support the potential
efficacy and safety of metreleptin across a range of rare,
generalized and partial lipodystrophy syndromes.
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.
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 has
had 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 the Metreleptin Expanded Access Program
Consistent with the severity and rare nature of this disorder,
Amylin has received both orphan drug designation from FDA's Office
of Orphan Products Development, as well as fast track designation
for use of metreleptin in patients with lipodystrophy.
Because metreleptin is not available for routine clinical use,
and because of the high unmet medical need of these patients,
Amylin has made this investigational medication available now under
an expanded access pathway, an FDA-authorized treatment IND
protocol. The treatment IND mechanism allows for access to
investigational medications in special cases of unmet medical need.
For more information on this program, please contact the Amylin
Customer Support Center at (800) 868-1190.
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.