Amsterdam Molecular Therapeutics Reports Half-Year Results 2010

By Amsterdam Molecular Therapeutics B.v, PRNE
Monday, August 30, 2010

AMSTERDAM, The Netherlands, August 31, 2010 - Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field
of human gene therapy, today reported its results for the first half year of


    - Glybera(R):
    - EMA initiated MAA review in 01/2010
    - Approval progressing on schedule for decision mid 2011
    - Novel biomarker for Glybera(R) activity identified
    - Hemophilia B: Phase I/II started
    - Duchenne Muscular Dystrophy: to benefit from EUR 4 million
      innovation credit
    - sRNA: silencing gene therapy technology achieves 80% cholesterol
    - Supervisory Board nominations: 3 new industry professionals
      slated to join board
    - Key financial figures in line with guidance
    - Cash & cash equivalents of EUR 13.5 million at June 30, 2010

"We are very pleased with the key milestones we have achieved
in the first half year of 2010 in the Glybera(R) approval process which seems
firmly on track. Our dialogue with the EMA is very encouraging and we look
forward to the Agency's decision with confidence. We also continue to make
progress with our pipeline, not only on the partnership front for programs
such as Hemophilia B, but also in our early stage efforts," said Jorn Aldag,
chief executive officer of AMT.


Glybera(R) for Lipoprotein Lipase Deficiency (LPLD)

In January 2010, the European Medicines Agency (EMA) commenced
review of AMT's Marketing Authorization Application. In May 2010 AMT received
questions (Day 120 questions) regarding the application. In July we met with
the EMA, to clarify the questions they raised, enabling AMT to align its
response strategy. We are now working towards an official response to the EMA
Day 120 questions, due by the end of 2010.

As of today we remain confident in the approvability of
Glybera(R). Our assessment is based on the following:

    - Our response to the EMA does not require further clinical
      trials with additional new to be treated patients. We expect to be
      able to formulate our response satisfactorily by submitting data and
      further analyses from already treated patients.
    - More, highly relevant, data from our last clinical trial
      CT-AMT-011-02 AMT strongly suggest that Glybera's effects are lasting
      (one year) via a mechanism that causes clearance of chylomicrons, the
      fat carrying particles which are responsible for pancreatitis in LPLD
    - Overall we have developed a clear response strategy, which,
      if executed with no unforeseen adverse events or delays, should allow
      us to remain on track for a positive EMA decision in the middle of

Hemophilia B

Further to their 2009 agreement to co-develop a vector-gene
combination for the treatment of Hemophilia B, AMT and St. Jude Children's
Research Hospital in the USA have successfully transferred Factor IX to AMT's
manufacturing platform and have demonstrated proof of concept in animals in
2010. The multicenter, dose escalation study with this vector-gene
combination began in March, 2010 at University College London Hospital in the
United Kingdom guided by Prof. Amit Nathwani. The first patient has been
dosed successfully and demonstrated good results both in terms of clinical
benefit and side effects. Further enrolment of patients is expected in the
second half of 2010.

Duchenne Muscular Dystrophy

In support of its program to treat Duchenne Muscular
Dystrophy, AMT received an investment credit from SenterNovem (now, the Dutch government innovation agency, in January 2010. The
credit comprises a loan covering 35% of the costs of the project through to
2013 with a maximum of EUR 4 million. The loan is repayable only if AMT
successfully commercializes the program. AMT has shown proof of concept in a
pre-clinical model with its optimized construct for exon skipping using its
proprietary AAV technology.

Parkinson's Disease

Together with the University of Lund, Sweden, AMT is
diligently working on the preclinical development of a gene therapy for
delivery of the GDNF gene to the brain. Efficacy data in an animal model of
PD is anticipated to be available by the end of the current year


Elevated levels of cholesterol are a major risk factor and
contributor to the development of atherosclerosis and cardiovascular disease
(CVD). Early research at AMT demonstrates that after a single intravenous
injection of a silencing gene therapy in animal models, the serum cholesterol
levels were reduced by 80% with no signs of toxicity. It is therefore
reasonable to expect a similar effect in patients, resulting in reduced risk
for atherosclerosis or CVD. Such a long-term, perhaps life-long active gene
therapy could eliminate the need for maintenance statin therapy.

Supervisory Board changes

During the period ended June 30, 2010, Alexander Ribbink and
George Morstyn retired from the Supervisory Board and AMT thanks them for
their substantial contributions. On April 28, 2010, AMT's co-founder Sander
van Deventer
was appointed to the Supervisory Board, and in addition Joseph
M. Feczko
, Steven H. Holtzman and Francois Meyer were nominated to the
Supervisory Board for consideration at the Extraordinary General Meeting to
be held on September 20, 2010.


Results comparison

Total net loss for the period ended June 30, 2010 amounted to
EUR 9.4 million, in line with the net loss for the period ended June 30, 2009
which also amounted to EUR 9.4 million.

The main item within operating costs reflects the investment
in Glybera(R) to support the registration process. Development of our
Duchenne Muscular Dystrophy program, which is 35% funded by a research credit
from SenterNovem through to completion of a Phase I clinical study continues.
Expenditure on our other development projects has been reduced as we are
constrained by our current resources and are focusing on the successful
completion of the Glybera registration process. Research and development
costs increased to EUR 8.1 million for the period ended June 30, 2010 from
EUR 7.1 million in the same period of 2009. At the same time, general and
administrative costs decreased to EUR 1.8 million in the period ended June
30, 2010
from EUR 2.9 million in the same period of 2009.

Net interest income/(cost) decreased to EUR (0.0) million for
the period ended June 30, 2010 from EUR 0.5 million in the same period in
2009 as a result of the Company's decreasing cash balance combined with
continuing low market interest rates for deposits.

Cash and cash equivalents amounted to EUR 13.5 million at June
30, 2010
, a decrease of EUR 9.1 million compared to EUR 22.6 million at
December 31, 2009. The decrease in cash and cash equivalents mainly stems
from the operational cash outflow which amounted to EUR 8.9 million for the
period ended June 30, 2010 (compared to an operating cash outflow of EUR 9.5
for the period ended June 30, 2009).


The Company's expenditure continues in line with budget.
However, as AMT has not yet reached the point of generating significant
revenues that could fund operations we continue to explore additional
opportunities for funding, including non-dilutive sources such as grants
and/or collaborations with partners. In addition, AMT is also tracking
opportunities for raising additional capital in conjunction with its bankers.
The outlook for the year remains unchanged.

Conference call and webcast presentation

AMT will conduct a conference call open to the public today at
3.30 p.m. CET, which will also be webcast. Netherlands dial in:
+31(0)20-712-1304; US dial in: +1-718-354-1361; UK dial in:
+44(0)20-7138-0821. Confirmation Code: 1128246

To listen to the conference call live via the internet, visit the
investor relations portion of the AMT website at Please
go to the website 15 minutes prior to the call to register, download and
install the necessary audio software.

The archived webcast also will be available for replay shortly
after the close of the call.

About Amsterdam Molecular Therapeutics

AMT is a leader in the development of human gene based
therapies. 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 what is probably the first stable and scalable AAV production
platform. This proprietary platform can be applied to a large number of rare
(orphan) diseases that are caused by one faulty gene. Currently, AMT has a
product pipeline with several AAV-based gene therapy products in LPLD,
Hemophilia B, Duchenne Muscular Dystrophy, Acute Intermittent Porphyria, and
Parkinson's Disease at different stages of research or development. AMT was
founded in 1998 and is based in Amsterdam.


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. EMA, 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.


For further enquiries: Ellen Roest, AMT Communications, Mobile: +31-6-2900-6179, e.roest at; Mike Sinclair, Halsin Partners, Mobile: +44-7968-022075, msinclair at; Jorn Aldag CEO, Tel: +31-20-566-7394, j.aldag at

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