Study Published in the Proceedings of the National Academy of Sciences
WALTHAM, Mass., Sept. 16 /PRNewswire-FirstCall/ -- Repligen Corporation
(Nasdaq: RGEN) today reported publication of a preclinical study
demonstrating that a novel histone deacetylase (HDAC) inhibitor improved
disease symptoms in a transgenic animal model of Huntington's disease. The
study, led by scientists at The Scripps Research Institute, demonstrated
that oral administration of the drug candidate to the mice after the onset
of symptoms slowed the progression of disease. Treated animals showed
superior motor performance by multiple measures, reduced loss of body
weight, reduced brain atrophy and improved overall appearance compared to
untreated animals. Huntington's mice were also analyzed for changes in the
hundreds of genes whose expression in the brain is altered in the mouse
model of Huntington's, as well as in humans with Huntington's disease.
Using gene microarrays, the researchers identified genes in three brain
regions whose expression was altered in the Huntington's mice and whose
expression was altered by treatment with the HDAC inhibitor. Treatment
partially normalized the expression level of approximately 90% of these
genes with 32% being restored to normal levels. These results suggest that
an HDAC inhibitor may be useful in treating Huntington's disease and that
specific genes may be useful as biomarkers in clinical trials. The research
was conducted using a compound that is covered by Repligen's exclusive
license from The Scripps Research Institute. The study entitled "The HDAC
Inhibitor 4b Ameliorates the Disease Phenotype and Transcriptional
Abnormalities in Huntington's Disease Transgenic Mice" will be published
the week of September 15, 2008 in the online version of the Proceedings of
the National Academy of Sciences.
"The marked reduction in symptoms achieved in the Huntington's disease
model without overt toxicity defines a second disease target for our HDAC
inhibitor program," stated Walter C. Herlihy, President and Chief Executive
Officer of Repligen Corporation. "We plan to continue to evaluate the
utility of our novel HDAC inhibitors as a potential treatment for both
Friedreich's ataxia and Huntington's disease."
Huntington's disease is caused by a trinucleotide repeat expansion in
the Huntington's disease gene (Htt) that results in production of a mutant
misfolded protein that is unable to function correctly. Huntington's
disease is characterized by dysregulation in the transcription of hundreds
of genes in the brain, leading to Huntington's symptoms ranging from jerky
and random movements to impaired thinking and perception. Huntington's
disease is a familial disease, passed from parent to child through a
mutation in the normal gene. Symptoms of Huntington's disease typically
emerge between the ages of 30 and 50 and fall into three categories: motor,
cognitive and psychiatric. Cognitive symptoms include slowed processing of
information in the brain, resulting in communication and planning
difficulties, while depression is the most common psychiatric symptom of
Huntington's disease. Motor symptoms include lack of coordination, muscle
spasms, and chorea. As the disease progresses, any function requiring
muscle control is affected, leading to severe disability, incapacitation or
loss of life due to complications 10 to 20 years after symptoms first
appear. There are approximately 30,000 people in the United States with
Huntington's disease and there is currently no safe and effective treatment
for the disease.
Repligen licensed the exclusive rights to intellectual property
covering HDAC inhibitors from The Scripps Research Institute in April 2007
following which Repligen established a HDAC development program for
Friedreich's ataxia. Over the past year, the Company has made significant
progress in advancing this program, resulting in the identification of
advanced lead compounds with improved potency and specificity. These lead
compounds are currently being assessed in pharmacology and toxicology
models in order to determine if one is suitable for clinical development.
In addition to Huntington's disease, Repligen is evaluating this family of
compounds for activity in preclinical models of other neurodegenerative
diseases including spinal muscular atrophy.
About The Scripps Research Institute
The Scripps Research Institute is one of the world's largest
independent, non-profit biomedical research organizations, at the forefront
of basic biomedical science that seeks to comprehend the most fundamental
processes of life. Established in its current configuration in 1961, it
employs approximately 3,000 scientists, postdoctoral fellows, scientific
and other technicians, doctoral degree graduate students, and
administrative and technical support personnel and is headquartered in La
Jolla, California.
About Repligen Corporation
Repligen Corporation is a biopharmaceutical company focused on the
development of novel therapeutics for neurological disorders. In addition,
we are the world's leading supplier of recombinant Protein A, the sales of
which partially fund the advancement of our development pipeline while
supporting our financial stability. Repligen's corporate headquarters are
located at 41 Seyon Street, Building #1, Suite 100, Waltham, MA 02453.
Additional information may be requested from http://www.repligen.com.
This press release contains forward-looking statements which are made
pursuant to the safe harbor provisions of Section 27A of the Securities Act
of 1933, as amended, and Section 21E of the Securities Exchange Act of
1934, as amended. The forward-looking statements in this release do not
constitute guarantees of future performance. Investors are cautioned that
statements in this press release which are not strictly historical
statements, including, without limitation, statements regarding current or
future financial performance and position, management's strategy, plans and
objectives for future operations, plans and objectives for product
development, plans and objectives for present and future clinical trials
and results of such trials, plans and objectives for regulatory approval,
litigation, intellectual property, product development, manufacturing plans
and performance such as the anticipated growth in the monoclonal antibody
market and our other target markets and projected growth in product sales,
constitute forward-looking statements. Such forward-looking statements are
subject to a number of risks and uncertainties that could cause actual
results to differ materially from those anticipated, including, without
limitation, risks associated with: the success of current and future
collaborative relationships, the market acceptance of our products, our
ability to compete with larger, better financed pharmaceutical and
biotechnology companies, new approaches to the treatment of our targeted
diseases, our expectation of incurring continued losses, our uncertainty of
product revenues and profits, our ability to generate future revenues, our
ability to raise additional capital to continue our drug development
programs, the success of our clinical trials, our ability to develop and
commercialize products, our ability to obtain required regulatory
approvals, our compliance with all Food and Drug Administration
regulations, our ability to obtain, maintain and protect intellectual
property rights for our products, the risk of litigation regarding our
intellectual property rights, our limited sales and manufacturing
capabilities, our dependence on third-party manufacturers and value added
resellers, our ability to hire and retain skilled personnel, our volatile
stock price, and other risks detailed in Repligen's filings with the
Securities and Exchange Commission. Repligen assumes no obligation to
update any forward-looking information contained in this press release or
with respect to the announcements described herein.
SOURCE Repligen Corporation
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Related links:
http://www.repligen.com
CONTACT:
Walter C. Herlihy, Ph.D., President and Chief
Executive Officer, +1-781-419-1900, or Laura Whitehouse, VP,
Market Development, +1-781-419-1812, both of Repligen Corporation
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