AMT Provides Business Update for the First Quarter 2011

May 19, 2011

AMSTERDAM, May 19, 2011 /PRNewswire-FirstCall/ — Amsterdam Molecular
Therapeutics (Euronext Amsterdam: AMT), a leader in the field of human gene
therapy, today provides its non-audited business update in compliance with
the EU transparency directive. This report summarizes material events and
AMT’s financial position for the first quarter of 2011.

1Q 2011 Highlights

– Glybera(R):

– Responses to Day 180 questions of the EMA/CHMP were submitted on

– EMA/CHMP opinion still expected mid-2011

– Collaboration with Institut Pasteur-led Consortium to develop
SanfilippoB gene therapy product for cGMP manufactured material; worth up to
EUR 1.8 million to AMT

EUR 1.1 million funding for Acute Intermittent Porphyria gene therapy
product as part of EU Consortium

– Grant from Dutch Parents Association for Duchenne Muscular Dystrophy
gene therapy

– Key financial figures in line with guidance

– Cash & cash equivalents of EUR 13.1 million at March 31, 2011, in line
with budget

Business Update

AMT has continued to diligently work towards the regulatory approval of
its lead product, Glybera, for lipoprotein lipase deficiency (LPLD). If
approved, Glybera would be the first gene therapy to be successfully
developed in the western world. AMT would target the initial launch in
France, Germany and the UK. In addition to LPLD, AMT believes there are many
hundreds of rare diseases that could be treated using AMT’s proprietary gene
therapy platform.

AMT has also continued to secure alternative funding sources through
grants and collaborations. This quarter, this has resulted in grants from the
EU as well as from the Dutch Parents Association for Duchenne Muscular

Across the remainder of AMT’s portfolio, the Company continues to make
encouraging progress. The Hemophilia B program is in a Phase I/II study,
while the Company’s Acute Intermittent Porphyria program is slated to start a
Phase I/II trial in patients in early 2012. The Duchenne Muscular Dystrophy
program has shown proof of concept in preclinical models, demonstrating
effective transgene delivery and distribution in the heart; loss of heart
function is the most significant contributor to mortality in this condition.
In GDNF, additional preclinical studies are continuing in Parkinson’s Disease
with further data expected by mid-2011. The collaboration with Institut
Pasteur has commenced well and initial batches have been manufactured and
supplied for evaluation.

AMT’s cash position* on March 31, 2011 amounted to EUR 13.1 million
compared to EUR 17.9 million on December 31, 2010. The cash outflow in the
first quarter of 2011, amounting to EUR 4.8 million, compared to EUR 4.9
in the prior year, mainly represented operational cash flow and is in
line with budget. AMT employed 87 persons as of March 31, 2011. Total
expenses in the first quarter of 2011 were EUR 4.5 million compared to EUR
5.1 million
in the same period last year.

    *The Company's cash position is composed of cash and cash equivalents.
    Material events after March 31, 2011
    Since March 31, 2011 there have been no material events.
    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,
Parkinson’s Disease, and SanfilippoB 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
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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