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Sirtris Pharmaceuticals, Inc. (SIRT) Receives Orphan Drug Designation from FDA for Treatment of MELAS

Sirtris Pharmaceuticals (SIRT), based in Cambridge, Massachusetts, is a biopharmaceutical company focused on discovering and developing proprietary, orally available, small molecule drugs with the potential to treat diseases associated with aging, including metabolic diseases such as Type 2 diabetes. The company’s drug candidates are designed to mimic certain beneficial health effects of calorie restriction, without requiring a change in eating habits, by activation of sirtuins. Sirtuins are a recently discovered class of enzymes that Sirtris believes control the aging process. The company is also engaged in a human clinical trial for MELAS, a mitochondrial disorder.

The company was recently granted orphan-drug designation by the United States Food and Drug Administration (FDA) for resveratrol in the treatment of MELAS (Mitochondrial myopathy, Encephalopathy, Lactic Acidosis, and Stroke-like episodes) syndrome. Orphan-drug designation for resveratrol in MELAS syndrome provides the company with seven years of marketing exclusivity upon FDA approval. MELAS is a progressive and fatal disorder with no known treatments. Symptoms include muscle weakness, fatigue, recurrent headaches and seizures. The disease is caused by a point mutation in mtDNA, leading to the development of poorly functioning mitochondria, and can be confirmed through genetic testing.

In preclinical testing, activation of the SIRT1 (a gene tied to the aging process and improved mitochondrial function) enzyme with SRT 501 (resveratrol) has been shown to increase the number and function of mitochondria. It has also been shown to be safe and well-tolerated in two earlier human phase 1a clinical trials. For patients with Type 2 Diabetes, SRT501 was shown to be safe and to significantly lower glucose at the two-hour time point in an oral glucose tolerance test conducted as part of the 28 day trial.

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