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.
Amylin Pharmaceuticals, Inc.