Amsterdam (ots/PRNewswire) - Amsterdam Molecular Therapeutics
(Euronext: AMT), a leader in the field of human gene therapy,
announced today the treatment of the first patient in a
preregistration clinical trial with Glybera(TM). This gene therapy
product targets lipoprotein lipase deficiency (LPLD), a seriously
debilitating and potentially lethal disease.
The randomized controlled trial has been designed to gather
additional data on the effects of Glybera on lipid metabolism and the
mechanisms underlying the prevention of pancreatitis attacks. The
trial is being performed under a Clinical Trial Application approved
by Health Canada.
The new clinical trial builds on positive data obtained from two
previous clinical trials in which a total of 22 LPLD patients were
treated. These data indicate that a single treatment with Glybera
results in a long-term, statistically significant and clinically
important reduction in the incidence of acute pancreatitis in LPLD
patients. The longest follow-up of individual patients is well over
three years, and the cumulative follow-up of all patients is more
than 45 years. The therapy was well tolerated and no drug-related
severe adverse events or unexpected side-effects have been observed.
AMT will include the data from the new trial in the Marketing
Authorization Application for Glybera. The submission of the dossier
to the European Medicines Agency is planned for the second half of
About the Disease
LPLD is an orphan disease, for which no treatment exists today.
The disease is caused by mutations in the LPL gene, resulting in
highly decreased or absent activity of LPL protein in patients. This
protein is needed in order to break down large fat-carrying particles
that circulate in the blood after each meal. When such particles,
called chylomicrons, accumulate in the blood, they may obstruct small
blood vessels. This results in recurrent and severe acute
inflammation of the pancreas, called pancreatitis, the most
debilitating complication of LPLD. The disease can result in
difficult-to-treat diabetes and is associated with significant
morbidity and mortality.
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 those that are caused by one faulty gene. Currently, AMT
has a product pipeline with nine products at different stages of
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.
ots Originaltext: Amsterdam Molecular Therapeutics B.V
Im Internet recherchierbar: http://www.presseportal.ch
For Information, André Verwei, CFO, Tel +31(0)20-5667394,