Study Finds Treatment with Metreleptin Improved Diabetes and Lipid Control in Patients with Partial Lipodystrophy

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.