Amylin Submits Clinical and Nonclinical Sections of Rolling Biologics License Application for Metreleptin to Treat Rare Forms of Lipodystrophy

SAN DIEGO, Dec. 20, 2010 /PRNewswire-FirstCall/ -- Amylin Pharmaceuticals, Inc. (Nasdaq: AMLN) today announced that it has submitted the initial 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 (high levels of triglycerides in the bloodstream) in patients with rare forms of lipodystrophy. Consistent with the severity and rare nature of the disorder, Amylin has received both orphan drug designation from FDA's Office of Orphan Products Development, as well as Fast Track designation for the use of metreleptin in patients with lipodystrophy. The focus of this marketing application is on rare inherited and acquired forms of lipodystrophy.

In the first part of its rolling submission, the Company submitted the nonclinical and clinical sections of the BLA. The Company plans to submit the chemistry, manufacturing and controls (CMC) section of the BLA by the end of 2011, which will complete the submission.

"It is gratifying to see that, after years of research focused on leptin as an effective therapy for lipodystrophy, we are now closer to bringing this important and innovative medicine to patients who are in dire need of better treatments," said Phillip Gorden, M.D., Director Emeritus, Senior Investigator, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) at the National Institutes of Health (NIH). Dr. Gorden is the principal investigator of an ongoing NIH clinical study evaluating the long-term efficacy of metreleptin treatment in lipodystrophy.

Lipodystrophy syndromes are characterized by abnormalities in adipose (fat) tissue distribution with loss of subcutaneous fat, and often manifest in childhood or adolescence. Patients with lipodystrophy can have multiple 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 result in a high risk for serious medical problems such as acute pancreatitis, accelerated atherosclerosis, vessel and nerve damage from diabetes and liver cirrhosis, which can markedly reduce quality of life and life expectancy. 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. Metreleptin therapy, an analog of the human hormone leptin, can substantially reduce high glucose and triglyceride levels in these patients. 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 leptin.

"We are proud to advance metreleptin to this regulatory milestone, and look forward to working with the FDA to make this important treatment more broadly available to patients with lipodystrophy," said Daniel M. Bradbury, President and Chief Executive Officer at Amylin. "The use of metreleptin to treat this rare and debilitating condition represents another example of the tremendous potential for peptide and protein science to be translated into therapies that improve the lives of patients with metabolic disorders."

About Metreleptin for Lipodystrophy

There are no therapies currently indicated specifically for the treatment of metabolic abnormalities associated with lipodystrophy. Presently, patients 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. 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.

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. Globally, approximately 150 patients with lipodystrophy are being treated with metreleptin under investigator-sponsored trials and expanded access programs.

About Leptin

Leptin, a fat cell hormone that plays a key role in regulating metabolism, was first discovered in 1994 by Dr. Jeffrey Friedman of The Rockefeller University in New York.  Dr. Friedman has won numerous awards during his career and recently won the 2010 Lasker Award in basic medical research for his work.  Metreleptin (recombinant methionyl human leptin), an analog of human leptin, has also been studied as a potential treatment for obesity and diabetes.

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 http://www.amylin.com.

This press release contains forward-looking statements about Amylin, which involve risks and uncertainties.  Amylin's actual results could differ materially from those discussed herein 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 Form 10-Q.  Amylin undertakes no duty to update these forward-looking statements.

SOURCE Amylin Pharmaceuticals, Inc.