AMT to Supply cGMP Manufacturing to Consortium Led by Institut Pasteur for Clinical Development of Gene Therapy for Rare Genetic Disorder in Children

January 10, 2011

AMSTERDAM, January 10, 2011 /PRNewswire-FirstCall/ — Amsterdam Molecular
Therapeutics (Euronext: AMT), a leader in human gene therapy, announced today
that it has entered into an agreement with Institut Pasteur, Paris, France,
and a group of French research institutes (together the “Consortium”) to
support clinical development of a novel gene therapy to treat Sanfilippo B.
This rare genetic disease affecting new-born children leads to progressive
neuronal degeneration and death. There is no approved therapy currently

On behalf of the Consortium, Institut Pasteur will lead the development
program and will also sponsor the initial Phase I/II clinical study of a gene
therapy to replace an enzyme (alpha-N-acetylglucosaminidase) that is missing
in brain cells of SanfilippoB patients. This enzyme is specifically required
for the degradation of heparan sulfate glycosaminoglycans (GAGs), essential
carbohydrate molecules used to build tissue. The accumulation of incompletely
degraded GAG molecules triggers a cascade of pathological events leading to
neuronal dysfunction and death.

AMT will manufacture and supply the adeno-associated viral 5 (AAV5) gene
therapy product to the Consortium. Thanks to donations collected during the
French Telethon, the French Muscular Dystrophy Association (AFM), a
Consortium member, will fully fund the development program through to
completion of the Phase I/II clinical study, including all AMT manufacturing
costs. The overall manufacturing contract entails payments to AMT of EUR1.8
. If the Consortium successfully demonstrates proof of concept in the
Phase I/II study, AMT will have an option to acquire full commercial rights
for the program. The Phase I/II clinical study is scheduled to begin before

“This partnership leverage’s AMT’s proven expertise in cGMP manufacturing
of gene therapy products and our experience in progressing these products
through clinical development and the regulatory processes needed for
successful commercialization,” said Jorn Aldag, CEO of AMT. “In addition, we
will have an option to acquire full commercial rights for the program on
completion of the Phase I/II study, which supports our strategy to build a
pipeline of orphan and ultra-orphan indications.”

Muriel Eliaszewicz, Medical Director of Institut Pasteur, said: “AMT is
one of the only companies in the world that has a proven ability in
manufacturing cGMP quality gene therapy products, not only in batches
sufficient for clinical development but also in support of a potential
regulatory approval. We are delighted that we now have the ability to
commence the difficult process of developing a cure for patients with this
very challenging disease.”

AMT has a cGMP-licensed, 375 m2 manufacturing facility to produce its AAV
vectors for gene therapy products. This highly specialized facility is fully
validated for commercial production and has a capacity capable of producing
enough material to supply its European and North American target markets with
AMT’s lead product Glybera(R), currently under review at the European
Medicines Agency (EMA) for treatment of lipoprotein lipase deficiency (LPLD)
and for the next phases of clinical development of all other programs
currently in the pipeline.

In December 2010, Consortium researchers published preclinical data on
the Sanfilippo B gene therapy in Molecular Therapy, the official publication
of the American Society of Gene and Cell Therapy*. This data demonstrated
safety and efficient spreading of the AAV5 gene vector particles throughout
the brain in models of the disease. The gene vectors also exhibited long-term
viability within the cells and an improvement of histological and biochemical

AMT to present today at Biotech Showcase in San Francisco

Amsterdam Molecular Therapeutics will present today, Monday, Jan. 10,
, at 2 p.m. Pacific time, at the Biotech Showcase Conference in San
. Jorn Aldag, chief executive officer, will make a formal
presentation at the conference, which runs parallel to the 28th Annual J.P.
Morgan Healthcare Conference, and takes place at Parc 55 Wyndham San
Francisco – Union Square. The presentation takes places in the Stockton room.

*Molecular Therapy 18, 2041 (December 2010) |10.1038/mt.2010.265

About the Disease

Sanfilippo B is a rare autosomal recessive lysosomal storage disease,
which manifests in young children. Initial symptoms include a slowing of
development and/or behavioral problems, followed by progressive intellectual
decline resulting in severe dementia and progressive motor disease. In the
final phase of the illness, children become increasingly immobile and
unresponsive, often require wheelchairs, and develop swallowing difficulties
and seizures. The life-span of an affected child does not usually extend
beyond late teens to early twenties. Currently there is no approved therapy
for this disease. The estimated number of new cases of SanFilippo B syndrome
in the European Union is 14 per year.

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

About Institut Pasteur

Louis Pasteur created the Institut Pasteur in 1887 as a private
non-profit foundation that rapidly became world-renowned for its biomedical
research. The main aim of the Institut Pasteur is understanding and
preventing diseases throughout the world through excellent scientific and
public health research, teaching and other activities. Together with its
major contribution to a deeper understanding of fundamental aspects of life,
the Institut Pasteur continues to devote a large part of its efforts to
infectious diseases, inherited disorders, neurodegenerative diseases and
certain cancers. Close to 2,600 people work on its main campus in Paris,
which is at the heart of an international network of 32 research institutes
on 5 continents. Over the years, 10 Institut Pasteur researchers have
received the Nobel Prize.


About the French Muscular Dystrophy Association (AFM)

AFM, a Consortium member, has become a major player in biomedical
research on rare diseases in France and around the world. Thanks to donations
from France’s annual Telethon, the AFM is currently funding 36 clinical
trials on 30 different genetic diseases: neuromuscular or neurological
diseases, and diseases of the blood, eyes, skin and immune system.

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.

    For further enquiries:

    Jorn Aldag
    Tel : +31-20-566-7394

    Mike Sinclair
    Halsin Partners
    Tel : +44-20-7318-2955

SOURCE Amsterdam Molecular Therapeutics B.V

Source: newswire

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