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Press release
The AFM and
Transgene Release Results of the first Phase I Gene Therapy Trial on Duchenne
and Becker Muscular Dystrophies
at the American Society of Gene Therapy Annual Meeting
Evry / Strasbourg, France, June 5, 2003 - The French Association against
Muscular Dystrophies (Association Française contre les Myopathies, AFM) and
Transgene (Nouveau Marché: TRANSGENE or 6274 - NASDAQ: TRGNY) announced that
the results of their Phase I trial on gene transfer for Duchenne/Becker's
Muscular Dystrophy will be presented today at the Conference of the American
Society of Gene Therapy in Washington, D.C.
This trial involved the administration of a plasmid (non-viral vector)
containing the whole sequence of the human dystrophin gene in patients with
Duchenne’s and Becker's Muscular Dystrophies.
These genetic muscular diseases are characterized by the lack of, or
incomplete expression of the dystrophin protein, resulting in serious
progressive muscular degeneration.
Duchenne’s dystrophy, the most frequent neuromuscular disease in
children, affects approximately one male out of 4,000. Becker’s dystrophy has a
later onset and affects one teenager out of 50,000. No curative treatment has
been found to date for these disorders.
This trial was initiated through the impetus given by AFM, a patient
association, and was mostly funded with proceeds from the Telethon, AFM’s
annual fund-raising event. It is the first completed gene-transfer trial in
humans for a neuromuscular disease. The vector used in the trial is a plasmid constructed
by Transgene, sponsor of the trial, under its long-standing partnership with
AFM. The trial was conducted in Paris at the Myology Institute and in the
Department of Internal Medicine of the Pitié-Salpêtrière Hospital, under the
direction of Professors Michel Fardeau and Serge Herson. It started in
September 2000 after obtaining regulatory approvals on November 30, 1999.
Objectives and methodology :
This clinical trial was designed to demonstrate the vector-induced
expression of the normal protein in Duchenne/Becker’s Muscular Dystrophy
patients and the absence of toxicity and rejection of this protein by their
immune systems.
Nine patients were enrolled according to
specific genetic and histological criteria.
The patients were divided into three cohorts, the first two of which
received a single injection of 200 µg or 600 µg of plasmid-dystrophin, while
patients in the third cohort received two injections of 600 µg, two weeks
apart. A sample of the muscle segment which received the injection (radial
muscle) was collected 21 days after the first (or single) injection. As a precautionary measure, patients were
sequentially enrolled in the trial, with an independent committee examining the
results of each patient before enrolling the next one.
Results :
The plasmid-dystrophin administered has been
found in muscle samples from all patients.
Expression of the dystrophin has been detected, generally in a small
quantity in one to ten percent of muscle fibers, in three out of six
patients in the first two cohorts, and in all three patients in the third
cohort. No immune reaction against the
plasmid or the newly expressed dystrophin was detected. The clinical safety profile of the plasmid
administration was always excellent.
These results demonstrate for the first time
that it is possible to obtain local expression of dystrophin in patients with
Duchenne/Becker’s Muscular Dystrophies following the administration of a
plasmid containing the human dystrophin gene, without any detectable immune
reaction observed under the trial’s experimental dosing and time conditions.
The AFM and Transgene are currently working
on continued development of this approach with the goal of increasing the
protein expression level and extending the targeted muscle regions.
About AFM
Created in 1958, the French Muscular Dystrophy Association (AFM) is a non-profit association registered under French law which includes patients and families affected by neuromuscular diseases. Its mission is to find a cure for these seriously disabling - and currently incurable- diseases, most of which have a genetic origin, and also help people affected by them. Funding comes from a Telethon, the Association’s annual fund-raising event held on the first week-end in December. Thanks to the generosity of millions of donors, AFM has become a major player in genetic research in France. In 1990, it created the Genethon laboratory which produced the first maps of the human genome, thus paving the way for the sequencing of the human genome. It was also a partner in the first successful gene therapy for bubble-boys announced by Pr Alain Fischer in 2000. AFM has supported more than 7,500 research programs since 1987 and has contributed to the discovery of the genes responsible for more than 700 diseases.
About Transgene
Transgene, based in Strasbourg, France, is a biopharmaceutical company
dedicated to the discovery and development of therapeutic vaccines, gene
therapy products, and delivery technologies for the treatment of diseases for
which there is no cure or adequate treatment at present, with a focus on the
treatment of cancer. Transgene has five
products in clinical development, two of which are in Phase II clinical trials
and three of which have completed Phase I clinical trials. Transgene’s proprietary vector technology
platform consists of multiple vector families with an emphasis on adenovirus,
poxvirus and non-viral vectors.
This press release contains forward-looking
statements, including statements regarding
the efficiency and safety of and potential market for Transgene’s
product candidates and prospects.
Statements that are not historical facts are based on Transgene’s
current expectations, beliefs, estimates, forecasts and assumptions. The statements contained in this release are
not guarantees of future performance and involve certain risks, uncertainties
and assumptions which are difficult to predict. Accordingly, actual outcomes and results may differ materially
from what is expressed in those forward-looking statements. Important factors
which may affect Transgene’s future operating results include the following:
Transgene’s product candidates may not demonstrate therapeutic efficacy after
initial promising results, Transgene may be unable to obtain regulatory
approval for its product candidates, Transgene may be unable to conduct its
clinical trials as quickly as it has predicted, Transgene may not have
sufficient resources to complete the research and commercialization of any of
its product candidates, competitors may develop technologies or products
superior to Transgene’s technologies or products, and other important factors
described in Transgene's Annual Report on Form 20-F for the year ended December
31, 2001 filed with the U.S. Securities and Exchange Commission, including
those factors described in the section entitled “Risk Factors.”