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

AMSTERDAM, June 24, 2011 -

- 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® (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 Jörn 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 event.

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® 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
href="www.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 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. 

PRN NLD

For further enquiries: Jörn Aldag, CEO, AMT, Tel : +31-20-566-7394, j.aldag at amtbiopharma.com; Mike Sinclair,
Partner, Halsin Partners, Tel : +44-20-7318-2955, msinclair at halsin.com