Irony......
Genzyme General and Pharming Group Report Results From First Two Clinical Trials for Pompe Disease
CAMBRIDGE, Mass., and LEIDEN, the Netherlands, Oct. 5 /PRNewswire/ --
Genzyme General (Nasdaq: GENZ) and Pharming Group N.V. (AEX: PHAR and
Easdaq: PHAR) announced today that results from the first two clinical trials
ever conducted for Pompe disease were presented this week at the American
Society of Human Genetics (ASHG) meeting in Philadelphia. Genzyme and
Pharming are working in partnership to develop a treatment for Pompe disease
and are associated with both clinical studies.
There is currently no effective therapy for patients who have Pompe
disease, a rare and fatal genetic disorder caused by the lack of the lysosomal
enzyme alpha-Glucosidase, which breaks down glycogen into glucose. The
accumulation of glycogen in the heart and skeletal muscles of patients results
in severe organ and tissue degradation. Pompe disease manifests itself in
early or late-onset forms. Infants generally die before reaching 12 months of
age, and the disease is typically fatal for those who develop symptoms as
juveniles. Pompe disease affects an estimated 5,000-10,000 people in the
developed world.
"The very encouraging clinical results achieved in both of these studies
give us tremendous confidence about moving forward to complete the development
of the first treatment for patients who are suffering from Pompe disease,"
said Henri A. Termeer, chairman and chief executive officer of Genzyme Corp.
"These results reveal the great progress that has been made and give us hope
that a therapy for all patients with this devastating disease will be
available in the near future."
On Tuesday, Y.T. Chen, M.D., Ph.D., of Duke University presented results
from an ongoing Phase 1-2 study being conducted at Duke Medical Center, in
which three infants with Pompe disease have received enzyme replacement
therapy for over one year. The patients have been receiving intravenous
infusions of a recombinant form of the enzyme human alpha-Glucosidase produced
in CHO cells.
The preliminary results from the trial indicate that the product is
capable of improving cardiac and skeletal muscle functions. In two patients,
left ventricular mass measurements decreased during therapy. The infants have
each passed the critical age of one year (currently 15-, 17-, and 20-months
old) and continue to show improved cardiac function, thus averting the
progressive cardiomyopathy and heart failure that normally lead to death prior
to age one in untreated patients.
Improvements of skeletal muscle functions have also been noted, although
the significance and extent have been more variable than with improvements in
cardiac function. One patient showed marked improvement and currently has
normal muscle tone and strength, as well as normal neurological and motor
development evaluations. Muscle biopsies confirmed that significant
reductions in glycogen accumulation occurred in one patient after enzyme
replacement therapy. The study data showed that the CHO-cell product was
generally well tolerated.
Also at the ASHG meeting, Dr. Ans T. van der Ploeg of Sophia Children's
Hospital in Rotterdam presented the results from a 36-week Phase 2 clinical
trial of transgenic human alpha-Glucosidase. The trial results were published
in The Lancet in July. The study was sponsored by Genzyme and Pharming.
In the trial, four infants with Pompe disease ranging in age from 2.5 to 8
months received weekly infusions of transgenic human alpha-Glucosidase. The
starting dose was 15-20 milligrams of enzyme per kilogram of body weight. The
dose was later increased to 40 mg/kg.
Results showed that transgenic human alpha-Glucosidase lowered lysosomal
glycogen storage and improved tissue morphology. Total tissue glycogen
content did not change. Skeletal muscle and strength improved, most
significantly for the patient who had the least severe disease at the start of
treatment. This infant reached milestones that are beyond expectations for a
patient with the disease, including crawling and standing with the support of
one arm at the age of 12 months. Improvements in motor function were also
observed for all patients.
The most prominent effect of treatment was on the heart. By 36 weeks,
cardiac size had decreased significantly to less than 30 percent of baseline.
Left-ventricular posterior-wall thickness and left-ventricular mass index
decreased in all patients.
All patients remain on therapy and have been treated for over a year.
Adverse events such as fever, rashes, and flushing were transient and
manageable by adaptation of the infusion rate and premedication.
Patient enrollment in the clinical trial evaluating the use of transgenic
alpha-Glucosidase has been completed. All patients will continue to receive
treatment with the transgenic product and will have the option of
transitioning to the CHO-cell derived product.
Earlier this year, Genzyme obtained exclusive, worldwide rights from
Synpac (North Carolina) Inc. to develop and commercialize the human
alpha-Glucosidase product derived from CHO cells, shifting its focus from the
development of human alpha-Glucosidase produced in the milk of transgenic
rabbits. The decision to pursue the CHO-cell product was based on
manufacturing considerations. Genzyme and Pharming believe that the
CHO-derived product can be scaled to commercial production levels, qualified
for use, and made accessible to patients faster than the transgenic product.
This decision was not based on efficacy or safety concerns with either
product. Clinical results obtained to date suggest that the CHO-derived and
transgenic products show comparable clinical effects.
Genzyme and Pharming plan to initiate a second clinical trial of the CHO-
cell product by the end of 2000 in patients with infantile-onset Pompe
disease. The companies are currently in discussions with the U.S. Food and
Drug Administration on the design of the trial, which is expected to involve
medical centers in both the United States and Europe. Further details will be
announced later this fall once the trial's protocol has been finalized.
Genzyme General develops and markets therapeutic products and diagnostic
products and services. Genzyme General has three therapeutic products on the
market and a strong pipeline of therapeutic products in development focused
primarily on the treatment of genetic diseases. A division of the
biotechnology company Genzyme Corporation, Genzyme General has its own common
stock intended to reflect its value and track its economic performance.
Pharming focuses on the development, production and commercialization of
human therapeutic proteins to be used in highly innovative therapies.
Pharming has developed a proprietary production platform of transgenic
animals, capable of producing human therapeutic proteins at high levels in
their milk. The company's product portfolio is aimed at treatments for
genetic disorders, surgery and trauma, infectious and inflammatory diseases,
tissue and bone repair and blood-related disorders. Pharming has operations
in Belgium, Finland, the Netherlands and the USA and currently employs more
than 200 people.
This press release contains forward-looking statements, including
statements about: the estimated Pompe disease patient population; plans to
complete development of a treatment for Pompe disease; and the expected timing
and design of a clinical trial. Actual results may materially differ due to
numerous factors, including: the actual timing and content of decisions made
by the FDA and other regulatory authorities; the results of clinical trial;
the actual safety and efficacy of the therapy; the ability to manufacture
sufficient quantities of product for development and commercialization
activities; the accuracy of Genzyme's information about the Pompe disease
patient population; and the risks and uncertainties described in reports filed
by Genzyme with the Securities and Exchange Commission under the Securities
Exchange Act of 1934, as amended, including without limitation Exhibit 99.2 to
Genzyme's 1999 Annual Report on Form 10-K, as amended. GENZ stock is a series
of common stock of Genzyme Corporation. Therefore, GENZ shareholders are
subject to all of the risks and uncertainties described in the aforementioned
reports.
Genzyme's releases are available on the World Wide Web at
genzyme.com. They are also available from Genzyme's fax-on-demand
service at 1-800-436-1443 within the United States or 1-201-521-1080 outside
the United States.
SOURCE Genzyme General
Web Site: genzyme.com
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