Amsterdam Molecular Therapeutics Receives Opinion on Glybera(R) Marketing Authorisation Application

June 24, 2011

AMSTERDAM, June 24, 2011 /PRNewswire/ –

– Dossier Re-Examination Process Initiated

Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field
of human gene therapy, today announced that it has received an opinion on
its Marketing Authorisation Application (MAA) for Glybera(R) (alipogene
tiparvovec) as a potential therapy for Lipoprotein Lipase Deficiency
(“LPLD”). Following a recent meeting with the European Medicines Agency’s
(EMA) Committee for Medicinal Products for Human Use (CHMP), AMT has been
notified that, at this time, Glybera is not approvable.

Subsequent to a review of the CHMP’s letter, AMT believes that Glybera
can receive a positive opinion subject to generating additional data from
existing patients. AMT has therefore decided to ask for a re-examination of
the clinical data package.

“From what we know today, despite the disappointment, we believe that
there is an indication from the CHMP that Glybera could receive approval and
that the current opinion at this time is a reflection of insufficient proof
of clinical benefit of Glybera as a result of low patient numbers measured
for chylomicron handling for at least 12 months post treatment. The CHMP
also indicated that if certain additional data from already treated patients
would confirm current results by the end of 2011, an approval may be
possible,” noted Jorn Aldag, CEO of AMT. “In the dossier, we provided
important data showing Glybera is safe and prevents episodes of
pancreatitis, the major clinical complication of LPLD. We appreciate the
CHMP’s responsibility for caution on such an advanced therapy, so we will
work diligently to generate more information and, we hope, ensure that
Glybera will still reach patients.”

Next steps

As part of the MAA, AMT presented data to the CHMP on Glybera showing
evidence in reducing the risk of pancreatitis. Also, the company
demonstrated a clear signal of patients’ ability to break down large
chylomicron molecules, the accumulation of which, after intake of dietary
fat, seems to be responsible for the occurrence of pancreatitis in LPLD
patients. AMT is initiating study CT-AMT-011-04 to examine further how
already treated patients handle chylomicrons over time. In addition, AMT
will continue to collect follow up data on pancreatitis incidence. AMT’s
available funds will support its existing operations beyond the completion
of the re-examination process into 2012.

“Based on communication from EMA, we understand that our technology
platform using adeno-associated virus (AAV) vectors is approvable,” Mr.
Aldag added. “While we pursue the re-examination of Glybera, we will also
continue development of other products in our pipeline such as hemophilia B
and GDNF gene therapy for Parkinson’s disease and Huntington’s disease. We
will also continue our preparations for registrations in Canada and the US.”

For investors, AMT will conduct a conference call at 9:00 AM CET on June
24, 2011. To participate in the conference call, please call one of the
following telephone numbers 15 minutes prior to the event, using access code
2788745: +44-(0)20-7136-2054 for the UK; +1-718-247-0881 for the US; and +
31-(0)20-201-5468 for the Netherlands. A pdf version of the presentation is
available in the news and investor relations sections of the corporate
website. Following the presentation, the lines will be opened for a question
and answer session. A replay of the call will be available following the

About Glybera

AMT has developed Glybera as a treatment for patients with the genetic
disorder lipoprotein lipase deficiency. 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. Excess chylomicrons result in recurrent and severe acute
inflammation of the pancreas, called pancreatitis, the most debilitating
complication of LPLD. Glybera(R) has orphan drug status in the EU and US.

About Amsterdam Molecular Therapeutics

AMT is a world leader in the development of human gene based therapies.
In addition to Glybera, AMT has a product pipeline of several gene therapy
products in development for hemophilia B, Duchenne muscular dystrophy, acute
intermittent porphyria, Parkinson’s disease and SanfilippoB. Using
adeno-associated viral (AAV) derived vectors as the delivery vehicle of
choice for therapeutic genes, the company has been able to design and
validate probably the world’s first stable and scalable AAV manufacturing
platform. This proprietary platform can be applied to a large number of rare
(orphan) diseases caused by one faulty gene and allows AMT to pursue its
strategy of focusing on this sector of the industry. AMT was founded in 1998
and is based in Amsterdam. Further information can be found at


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 AMT 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. AMT expressly
disclaims any intent or obligation to update any forward-looking statements
herein except as required by law.


SOURCE Amsterdam Molecular Therapeutics B.V

Source: newswire

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