Amsterdam, The Netherlands - September 18, 2008 - Amsterdam Molecular
Therapeutics (Euronext: AMT), a leader in the field of human gene
therapy, today announced that it obtained a license from Amgen to use
their GDNF gene for the development of a gene therapy treatment for
Parkinson's disease. The combination of this gene with AMT's
proprietary adeno-associated virus (AAV) gene therapy platform could
potentially allow the development of an effective, long-term
treatment for this progressive and crippling disease.
Parkinson's disease is the second most common neurodegenerative
disease. It usually affects people over 65 with an estimated total of
4.5 million patients worldwide. Due to increasing life expectancy of
the general population, the number of patients with Parkinson's
disease is expected to double to around 9 million patients between
now and the year 2030.
Patients with Parkinson's disease slowly lose control of their
muscles, resulting in tremors, stiffness, slowness of movement, and
lack of coordination and thus in a serious loss of quality of life.
Parkinson's is caused by degeneration and death of nerve cells in a
specific part of the brain. These cells produce dopamine, a substance
necessary for communication between nerve cells involved in the
coordination of movement. Current therapies are limited to treatment
of symptoms. There are no therapies available that slow down or halt
the progression of the disease.
A new way to deliver the GDNF gene
"This license from Amgen offers us a unique opportunity to combine
our gene technology and know-how with the GDNF gene as a tool to
create a potential breakthrough in the treatment for this common and
severely debilitating disease," said Ronald Lorijn, CEO of AMT "We
believe our gene therapy approach could be an effective way to
deliver the gene to the regions of the brain affected by Parkinson's
disease."
Protect and improve nerve cells with GDNF
The GDNF gene contains the information for a protein necessary for
the development and survival of nerve cells. AMT will combine this
gene with its own proprietary technology to develop a gene therapy
treatment that aims to protect and enhance the function of the nerve
cells that produce dopamine. The positive effect of GDNF on nerve
cells has been shown in several animal studies, making it an
attractive candidate for the treatment of Parkinson's disease.. AMT
believes that its gene-delivery platform may potentially provide a
solution for delivering GDNF to the brain.
About Amsterdam Molecular Therapeutics
AMT has a unique gene therapy platform that to date appears to
circumvent many if not all of the obstacles that have prevented gene
therapy from becoming a mainstay of clinical medicine. Using
adeno-associated viral (AAV) vectors as the delivery vehicle of
choice for therapeutic genes, the company has been able to design and
validate what is probably the first stable and scalable AAV
production platform. As such, AMT's proprietary platform holds
tremendous promise for thousands of rare (orphan) diseases,
especially the ones that are caused by one faulty gene. AMT currently
has a product pipeline with seven products at different stages of
development.
About Amgen
Amgen discovers, develops, manufactures and delivers innovative human
therapeutics. A biotechnology pioneer since 1980, Amgen was one of
the first companies to realize the new science's promise by bringing
safe and effective medicines from lab, to manufacturing plant, to
patient. Amgen therapeutics have changed the practice of medicine,
helping millions of people around the world in the fight against
cancer, kidney disease, rheumatoid arthritis, and other serious
illnesses. With a deep and broad pipeline of potential new medicines,
Amgen remains committed to advancing science to dramatically improve
people's lives. To learn more about our pioneering science and our
vital medicines, visit www.amgen.com.
For information
André Verwei Rob Janssen
CFO Director Corporate Communications
& Investor Relations
+31 20 566 5686 +31 20 566 7509
a.verwei@amtbiopharma.com r.janssen@amtbiopharma.com
www.amtbiopharma.com
Certain statements in this press release are "forward-looking
statements" including those that refer to management's plans and
expectations for future operations, prospects and financial
condition. Words such as "strategy," "expects," "plans,"
"anticipates," "believes," "will," "continues," "estimates,"
"intends," "projects," "goals," "targets" and other words of similar
meaning are intended to identify such forward-looking statements.
Such statements are based on the current expectations of the
management of Amsterdam Molecular Therapeutics only. Undue reliance
should not be placed on these statements because, by their nature,
they are subject to known and unknown risks and can be affected by
factors that are beyond the control of AMT. Actual results could
differ materially from current expectations due to a number of
factors and uncertainties affecting AMT's business, including, but
not limited to, the timely commencement and success of AMT's clinical
trials and research endeavors, delays in receiving U.S. Food and Drug
Administration or other regulatory approvals (i.e. EMEA, Health
Canada), market acceptance of AMT's products, effectiveness of AMT's
marketing and sales efforts, development of competing therapies
and/or technologies, the terms of any future strategic alliances, the
need for additional capital, the inability to obtain, or meet,
conditions imposed for required governmental and regulatory approvals
and consents. AMT expressly disclaims any intent or obligation to
update these forward-looking statements except as required by law.
For a more detailed description of the risk factors and uncertainties
affecting AMT, refer to the prospectus of AMT's initial public
offering on June 20, 2007, and AMT's public announcements made from
time to time.
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