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EMA Starts Formal Review of Glybera(R) Dossier

January 25, 2010

AMSTERDAM, The Netherlands, January 25 /PRNewswire-FirstCall/ –
Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of
human gene therapy, has reached another important milestone in the official
marketing authorisation process for its lead product Glybera(R), AMT’s
proprietary product for lipoprotein lipase deficiency (LPLD). The submission
of the Glybera(R) Marketing Authorisation Application (MAA), announced
earlier, has cleared the validation stage with The European Medicines Agency
(EMA, formerly known as EMEA). The EMA will now commence its formal review of
Glybera(R).

AMT has concluded two clinical studies for LPLD, in Europe and Canada,
and long term follow-up from both of these is ongoing, as is a third clinical
study in Canada. In these three studies Glybera(R) has shown a sizeable
decrease in the incidence of pancreatitis, or acute inflammation of the
pancreas, the most debilitating complication of LPLD. In addition, these
studies indicate that Glybera(R) has an excellent safety profile.

“The acceptance of the Glybera(R) dossier by EMA is a significant step
towards marketing approval for Glybera(R). Moreover, it demonstrates AMT’s
development capability. A future approval of the MAA for Glybera would fully
validate our gene therapy approach and our adeno-associated viral (AAV)
vector delivery platform. We believe this step offers hope to many patients,
as gene therapy may become the therapeutic approach of choice for inherited
disorders” said Jorn Aldag, Chief Executive Officer of AMT.

The EMA formal review will be conducted via the centralised procedure,
which is the standard route for all advanced therapies. During 2010, AMT
expects to provide further updates on the results from its follow-up and
ongoing studies, in accordance with reporting regulations.

About the Disease

LPLD is a seriously debilitating, and potentially lethal, 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 can result not only in pancreatitis, but also in
difficult-to-treat diabetes, and is associated with significant morbidity and
mortality.

About Amsterdam Molecular Therapeutics

AMT, founded in 1998 and based in Amsterdam, is a leader in the
development of human gene based therapies. Using AAV 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 safe and efficacious proprietary platform offers a unique
manufacturing capability which 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 LPL
Deficiency, Hemophilia B, DMD, Acute Intermittent Porphyria and Parkinson’s
Disease at different stages of research or development.

For information

AMT will be presenting at the BioCEO & Investor Conference,
Waldorf-Astoria, New York City, at 9:30 am (EST) on Tuesday, February 9, 2010.

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.

SOURCE Amsterdam Molecular Therapeutics B.V


Source: newswire



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