AMT Receives EUR 1.1 Million Funding for Acute Intermittent Porphyria Gene Therapy as part of EU Consortium Grant

January 30, 2011

AMSTERDAM, The Netherlands, January 31, 2011 /PRNewswire-FirstCall/ –
Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of
human gene therapy, announced today that the European Union (EU) has
finalized a EUR 3.3 million grant to the AIPGENE consortium, of which AMT is
a member, for the development of a gene therapy product for Acute
Intermittent Porphyria (AIP). AIP is a severe and progressive disease caused
by the inability of the body to produce the heme protein, a component of
hemoglobin, as well as other important proteins.

From the grant, made under the EU’s FP7 program, AMT will receive EUR 1.1
. The grant will cover approximately 75% of AMT’s overall development
costs to bring this product forward to completion of a Phase I/II study in
humans. AMT holds the commercialization rights to the AIP gene therapy
(AMT-021). The AIPGENE consortium is led by Fundacion para la Investigation
Medica Aplicada (FIMA, Spain), and in addition to AMT, it includes Clinica
Universidad de Navarra (Spain), Stockholms Lans Landsting (Sweden), Deutsches
Krebsforschungszentrum (Germany), Digna Biotech (Spain) and Servicio
Madrileno de Salud (Spain).

“This grant allows us to prioritize the development of our AIP gene
therapy program for this progressive and devastating disease within AMT’s
pipeline, as well as initiating the next step in advancing a potentially more
effective, long-term treatment for patients,” said Jorn Aldag, Chief
Executive Officer of AMT.

AIP results from mutations in the PBDG gene, which encodes for the enzyme
porphobilinogen deaminase, a liver protein necessary for the production of
heme. AMT-021 is intended to provide long-term normalization of the PBGD
protein in order to prevent acute attacks and their complications. AMT has
demonstrated that AMT-021 produces this normalization of the PBGD protein in
an animal model of AIP. In addition, it completely prevented the occurrence
of attacks and significantly ameliorated the neuropathy that develops in
untreated animals. AMT’s partner at FIMA has shown persistence of expression
of genes in the liver for more than a year using AAV-mediated delivery
methods similar to AMT-021. With the support of the all the AIPGENE partners,
AMT anticipates AIP patient enrolment in a clinical trial to begin in 2012.

On 28 May 2008, the European Medicines Agency (EMA) granted Orphan Drug
Designation to AMT’s gene therapy product for the treatment of AIP. This
entitles AMT to ten years of market exclusivity to treat AIP in Europe
following marketing approval, provided that this product candidate is the
first such approved new drug with a major medical benefit.

About Acute Intermittent Porphyria

Acute intermittent porphyria is a rare genetic disease where mutations in
the porphobilinogen deaminase (PBGD) gene, results in insufficient activity
of a protein necessary for heme synthesis. This leads to the accumulation of
toxic intermediate metabolites that produce a wide variety of problems
including acute and severe abdominal pains, psychiatric and neurological
illnesses. There is currently no cure for this condition and the disease is
typically progressive.

About Amsterdam Molecular Therapeutics

AMT is a world leader in the development of human gene based therapies.
The company’s lead product Glybera(R), a gene therapy for the treatment of
lipoprotein lipase deficiency (LPLD), is currently under review by the
European Medicines Agency (EMA). If approved, Glybera will be the first gene
therapy product to be marketed in Europe. AMT also has a product pipeline of
several gene therapy products in development for hemophilia B, Duchenne
muscular dystrophy, acute intermittent porphyria, and Parkinson’s disease.
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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