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."
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).
Amylin Pharmaceuticals, Inc.