HOUSTON, June 25, 2012 — Amylin Pharmaceuticals, Inc. (Nasdaq: AMLN) today announced findings from an ongoing clinical study (expanded access protocol) that shows treatment with metreleptin, an investigational medication under evaluation for the treatment of diabetes and/or hypertriglyceridemia (high levels of triglycerides in the bloodstream) in patients with rare forms of inherited or acquired lipodystrophy, reduced blood glucose and triglyceride levels. These findings were presented today in two separate posters at the Endocrine Society’s 94th Annual Meeting and Expo (ENDO) in Houston, Texas.
Lipodystrophy is an “ultra-orphan” rare disease estimated to affect a few thousand people worldwide, which often manifests with an early age of onset, has life-threatening metabolic complications, and for which there is a significant unmet medical need as there are no approved drugs that effectively treat the underlying cause(s) of the disease.
“The data analyses presented in patients with rare forms of lipodystrophy show that metreleptin lowered blood glucose and triglyceride levels regardless of leptin levels and associated autoimmune diseases,” said Elif Oral, M.D., University of Michigan, Ann Arbor, Michigan, and a principal investigator in the expanded access protocol for metreleptin. “In patients with lipodystrophy, fat typically accumulates in the blood and organs such as liver and muscle, which can lead to life-threatening complications including insulin-resistant diabetes and hypertriglyceridemia. If approved, metreleptin would be the first therapy indicated specifically for the treatment of diabetes and/or hypertriglyceridemia in patients with inherited or acquired lipodystrophy.”
Findings from the first poster titled, “Metabolic Effects of Metreleptin Treatment in Familial Partial Lipodystrophy (FPL),” included data from an ongoing expanded access protocol of metreleptin administration in patients with rare forms of inherited or acquired lipodystrophy. This analysis included 19 patients with diabetes and/or high triglyceride levels who were diagnosed clinically with FPL, one of the major subtypes of lipodystrophy, without using a threshold leptin level as an eligibility criterion. Overall, A1C, a measure of average blood sugar over three months, and triglyceride levels improved with metreleptin treatment, and metabolic benefits were observed across a range of leptin levels. In patients with A1C =7% (14/19), A1C decreased by 0.4±0.6% at six months (n=10) and 1.0±0.7% at 12 months (n=7). In patients with triglyceride levels =200 mg/dL at baseline (12/19), triglyceride levels decreased by 31±28 mg/dL at six months (n=6) and 184±127 mg/dL at 12 months (n=5). While on metreleptin, 2 of 12 patients on oral antidiabetic agents (OAD) were able to discontinue an OAD, and 4 of 9 patients on insulin reduced their insulin dose by 20% or more. Metreleptin was generally well tolerated.
Findings from the second poster titled, “Metreleptin Treatment in Acquired Generalized Lipodystrophy: Consistent Metabolic Effects in Three Patients Presenting with Distinct Autoimmune Conditions,” focused on three pediatric patients with acquired generalized lipodystrophy (AGL) who were studied under the same expanded access protocol. AGL is another major lipodystrophy subtype that is commonly associated with autoimmune diseases. These three patients with AGL also had active autoimmune disease, including juvenile dermatomyositis, autoimmune hepatitis, or Graves’ disease (a form of auto-immune thyroid disease). With metreleptin treatment, each patient experienced substantial reductions in lipodystrophy-related metabolic abnormalities, such as diabetes or high triglyceride levels, with no evidence of worsening of their respective autoimmune diseases.
Fat tissue is a major endocrine organ producing important metabolic hormones such as leptin. People with lipodystrophy lack the required fat tissue for normal metabolic function. This can be partial, affecting select areas of the body, or generalized, affecting nearly the entire body. A lack of fat tissue can lead to relative deficiency of leptin.
Without adequate leptin function, the metabolic system, which regulates food intake and the storage and break-down of dietary fat and carbohydrates, falls out of balance. As a result, fat accumulates in the blood and organs such as liver and muscle, which can lead to life-threatening complications including insulin-resistant diabetes and hypertriglyceridemia (high levels of triglycerides in the bloodstream).
There are no approved drugs that address the underlying relative leptin deficiency that is believed to contribute in large part to the metabolic abnormalities that occur in lipodystrophy. Currently available therapies for diabetes and hypertriglyceridemia are often rendered marginally effective due to the severity of the condition. More information on lipodystrophy can be found atwww.mylipodystrophy.com.
Metreleptin, an analog of the human hormone leptin, is a unique potential therapy for certain metabolic disorders in patients with rare forms of inherited or acquired lipodystrophy. Metreleptin is believed to work in part by reducing fat accumulation in organs, thereby improving insulin sensitivity. Clinical studies have been conducted by investigators at the National Institutes of Health (NIH) and other academic institutions in the U.S., Europe, and Japan to determine whether metreleptin can improve glycemic control and hypertriglyceridemia in patients with lipodystrophy.
In December 2010, Amylin initiated its rolling biologics license application (BLA) submission to the U.S. Food and Drug Administration (FDA) for the use of metreleptin to treat diabetes and/or hypertriglyceridemia in pediatric and adult patients with rare forms of lipodystrophy. In April 2012, Amylin submitted the chemistry, manufacturing and controls section of the BLA. Amylin recently announced that the FDA is seeking updated data from the submitted clinical studies that remain ongoing. Amylin is committed to responding in a timely fashion to enable the FDA to complete its evaluation of the rolling BLA submission.
About Amylin Pharmaceuticals
Amylin Pharmaceuticals is a biopharmaceutical company dedicated to improving lives of patients through the discovery, development, and commercialization of innovative medicines. Amylin is committed to delivering novel therapies that transform the way diabetes and other metabolic disorders are treated. Amylin is headquartered in San Diego, Calif. and has a commercial manufacturing facility in Ohio. More information about Amylin Pharmaceuticals is available at www.amylin.com.
This press release contains forward-looking statements about Amylin, which involve risks and uncertainties. Our actual results could differ materially from those discussed herein due to a number of risks and uncertainties, including risks that metreleptin as a potential treatment option for diabetes and/or hypertriglyceridemia in patients with rare forms of lipodystrophy will not be approved by the FDA; risks that the FDA will not accept the BLA for filing; risks that our clinical trials will not be completed when planned, may not replicate previous results, may not be predictive of real world use or may not achieve desired end-points; risks that our preclinical studies may not be predictive; and other risks inherent in the drug development and commercialization process. These and additional risks and uncertainties are described more fully in the Company's recently filed Form 10-Q. Amylin disclaims any obligation to update these forward-looking statements.