Sirtris Receives Orphan Drug Designation from U.S. Food and Drug Administration for Resveratrol in Mitochondrial Disorder, MELAS Syndrome
CAMBRIDGE, Mass. - Sirtris Pharmaceuticals,Â a biopharmaceutical company focused on discovering and developing small molecule drugs to treat diseases of aging, announced that the United States Food and Drug Administration has granted the company orphan-drug designation for resveratrol in the treatment of MELAS syndrome (Mitochondrial myopathy, encephalopathy, lactic acidosis, and stroke-like episodes). Orphan-drug designation for resveratrol in MELAS syndrome provides Sirtris with seven years of marketing exclusivity upon receipt of FDA approval.
â€œMELAS can have a devastating effect on the quality of life of patients and their families,â€ says Professor Patrick Chinnery of Newcastle University, who is leading a MELAS Phase 1b clinical trial in the United Kingdom with SRT501, Sirtrisâ€™ proprietary formulation of resveratrol. â€œFinding treatments for MELAS and the many other mitochondrial-related illnesses is essential,â€ says Chinnery, whose focus is the treatment and research of mitochondrial disorders.
â€œMany diseases of aging, such as Type 2 Diabetes, exhibit impaired mitochondrial function,â€ says Peter Elliott, PhD, Sirtris Senior Vice President of Development. â€œAs we target SIRT1, a gene tied to the aging process and improved mitochondrial function, we hope to develop new therapies for diseases like MELAS and Type 2 Diabetes.â€
"We are extremely pleased to have received orphan-drug designation in MELAS," says Christoph Westphal, MD, PhD, CEO and Vice Chair, Sirtris Pharmaceuticals, Inc. â€œClinical testing with SRT501 in this mitochondrial disorder offers the potential to provide an exciting, novel treatment option for the future.â€
MELAS is a progressive and fatal disorder with no known treatments. The earliest symptoms include muscle weakness, fatigue, recurrent headaches and seizures. The reported age of onset varies between 3 and 40 years, with most patients presenting between the ages of 5 to 15 years. The syndrome can manifest as stroke-like episodes in patients under 20 years of age. Seizures, dementia, impaired muscular function and neurodegeneration can be observed as the disease progresses. MELAS patients also have high glucose levels and approximately 30 percent have Type 2 Diabetes.
MELAS is caused by a point mutation in mitochondrial DNA, leading to the development of poorly functioning mitochondria, which supply cellular energy. The diagnosis can be confirmed through genetic testing.
In preclinical testing, activation of the SIRT1 enzyme with SRT501 has been shown to increase the number and function of mitochondria. SRT501 has also been shown to be safe and well-tolerated in two earlier human Phase 1a clinical trials. In a Phase 1b clinical trial with Type 2 Diabetes patients naÃ¯ve to treatment, 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.
The Phase 1b trial for MELAS conducted at Newcastle University is designed to test the primary endpoints of safety and pharmacokinetics of SRT501 in patients with MELAS. SRT501 is being administered to a group of 15 patients once daily for three months and an additional group of five patients will receive a placebo. Secondary endpoints include exercise tolerance, and fasting blood glucose and insulin levels. Sirtris expects data from this trial in the first half of 2009.
The United States Orphan Drug Act of 1983 was created to provide incentives for companies to develop and market treatments for diseases affecting fewer than 200,000 people in the United States. Under the Orphan Drug Act, more than 300 new drugs have been developed and approved.
Sirtris plans to seek orphan drug status for SRT501 in Europe.
About Sirtris Pharmaceuticals
Sirtris Pharmaceuticals 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. Our drug candidates are designed to mimic certain beneficial health effects of calorie restriction, without requiring a change in eating habits, by activation of sirtuins, a recently discovered class of enzymes that the Company believes control the aging process. Sirtris Pharmaceuticals is engaged in human clinical trials for Type 2 Diabetes, and is planning similar trials in cancer, another age-related disease. Sirtris is also engaged in a human clinical trial for MELAS, a mitochondrial disorder. The company's headquarters are in Cambridge, Massachusetts.
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, the potential therapeutic effects of SIRT1 expression and activation for diseases of aging, such as Type 2 Diabetes and MELAS; the progress, timing and results of preclinical and clinical studies of SIRT1 activators; the success of new therapies for Type 2 Diabetes and MELAS; and the potential of sirtuin modulators to receive regulatory approval. These forward-looking statements about future expectations, plans and prospects of Sirtris Pharmaceuticals involve significant risks, uncertainties and assumptions, including risks related to the lack of results that would provide a basis for predicting whether any of the Company's product candidates will be safe or effective, or receive regulatory approval, the possibility that results of pre-clinical studies are not necessarily predictive of clinical trial results, the Company's potential inability to initiate and complete pre-clinical studies and clinical trials for its product candidates, the fact that none of the Company's product candidates has received regulatory approvals, the potential inability of the Company to gain market acceptance of the Company's product candidates, and those other risks factors that can be found in the Company's filings with the Securities and Exchange Commission. Actual results may differ materially from those Sirtris Pharmaceuticals contemplated by these forward-looking statements. Sirtris Pharmaceuticals does not undertake to update any of these forward-looking statements to reflect a change in its views or events or circumstances that occur after the date of this release.