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Last updated on April 18, 2014 at 7:49 EDT

Start of Phase I/II Gene Therapy Clinical Trial for Hemophilia B

March 10, 2010

AMSTERDAM, March 10, 2010 /PRNewswire-FirstCall/ — Amsterdam Molecular
Therapeutics (Euronext: AMT), a leader in the field of human gene therapy,
announced today that the first patient has been dosed in the Phase I/II
exploratory clinical trial with a gene therapy product for hemophilia B, a
seriously debilitating and potentially lethal disease.

The trial is an open label dose-escalation study using a vector-gene
combination developed at the renowned St. Jude Children’s Research Hospital.
Dr. Arthur W. Nienhuis of St. Jude is the principal investigator of the
on-going trial. The work was initiated at St. Jude more than a decade ago by
Drs. Andrew Davidoff and Amit Nathwani and the collaboration has continued
following Dr. Nathwani’s return to London. The collaboration involves St.
Jude and University College London and other institutions in the US and
Britain. The objective of the trial is to assess the safety and efficacy of
different doses of hemophilia B gene therapy. Hemophilia B is an inherited
condition in which patients may have repeated and sometimes life threatening
bleeds after accidental trauma or medical interventions, because they do not
have sufficient functioning of an essential blood clotting factor, called
Factor IX.

AMT will build on the outcome of this exploratory trial and is preparing
for additional clinical development to establish safety, tolerability and
proof-of-concept with Factor IX gene therapy produced using AMT’s
proprietary, clinically validated production system. AMT has the exclusive
commercialization rights to the Factor IX gene used in the St. Jude trial and
has the ability to produce gene therapy product for hemophilia B at high
quality on a commercial scale. Additional developmental work using AMT’s
production system is on-going at St. Jude with AMT support.

Jorn Aldag CEO of AMT said: “Dr. Andrew Davidoff and his group at St.
Jude, together with Professor Nathwani in London, have done very important
scientific work on hemophilia B. We are really looking forward to the results
of the trial for continuing our collaboration, aiming for a real cure for
patients with this bleeding disorder. Use of the Factor IX gene fits
perfectly with AMT’s proprietary gene therapy platform and our business
strategy of developing cures for seriously debilitating orphan diseases. “

This hemophilia B gene therapy, administered once, will introduce the
functional gene for the Factor IX protein into the patient’s liver cells with
the goal to restore blood clotting functionality long-term. In pre-clinical
studies, Factor IX gene therapy resulted in long-term production of Factor IX
protein at a therapeutically significant level after a single administration.
If this approach is successful, the long term efficacy of one time
administered hemophilia B gene therapy is expected to be perceived as a
significant advance over the current regular dosing of recombinant Factor IX.
In addition, the efficacy profile of this gene therapy is anticipated to
exceed that of current therapy, as the gene therapy should lead to stable
Factor IX levels whereas current recombinant protein treatment causes peaks
and troughs. It is hoped that hemophilia B gene therapy, therefore, can
potentially replace all recombinant Factor IX products.

The UK Medicines and Healthcare products Regulatory Agency as well as the
US Food and Drug Administration have approved the current trial.

About the Disease

Hemophilia B is characterized by severe episodes of external and internal
bleeding, resulting in significant morbidity. The episodes cause long-term
damage, for instance to the joints, and may be fatal if they occur in the
brain. The defect in blood clotting in hemophilia B is caused by the absence
of functional clotting Factor IX as a result of mutations in the gene
encoding this protein. The factor IX gene is located on the X chromosome. It
is an X-linked recessive trait, which explains why only males are usually
affected.

Hemophilia B is a rare disease, occurring in 1 in 30,000 people, almost
always in males. The total number of patients in Europe and the USA together
is estimated to be between 35,000 and 40,000.

Currently, frequent intravenous administrations of recombinant Factor IX
are required to stop or prevent bleeding. Protein replacement therapy is
costly, cumbersome, and does not completely prevent bleeding.

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 LPLD,
Hemophilia B, DMD, Acute Intermittent Porphyria and Parkinson’s Disease at
different stages of research or development.

For information

AMT will be presenting at Bio Europe Spring 2010 Conference at the Centre
Convencions Internacional, Barcelona, at 1200 (CET) on Wednesday March 10,
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