ADDING MULTIMEDIA Sirtris Receives Orphan Drug Designation from U.S. Food and Drug Administration for Resveratrol in Mitochondri
April 02 2008 - 12:00PM
Business Wire
Sirtris Pharmaceuticals, Inc. (NASDAQ: SIRT), 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. �MELAS can have a
devastating effect on the quality of life of patients and their
families,� says Professor Patrick Chinnery of Newcastle University,
who is leading a MELAS Phase 1b clinical trial in the United
Kingdom with SRT501, Sirtris� proprietary formulation of
resveratrol. �Finding treatments for MELAS and the many other
mitochondrial-related illnesses is essential,� says Chinnery, whose
focus is the treatment and research of mitochondrial disorders.
�Many diseases of aging, such as Type 2 Diabetes, exhibit impaired
mitochondrial function,� says Peter Elliott, PhD, Sirtris Senior
Vice President of Development. �As 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.�
"We are extremely pleased to have received orphan-drug designation
in MELAS," says Christoph Westphal, MD, PhD, CEO and Vice Chair,
Sirtris Pharmaceuticals, Inc. �Clinical testing with SRT501 in this
mitochondrial disorder offers the potential to provide an exciting,
novel treatment option for the future.� 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 na�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.
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