CAMBRIDGE, Mass., July 26 /PRNewswire-FirstCall/ -- Genzyme Corporation
(Nasdaq: GENZ) today announced that it has begun to treat patients in a
Phase 2 clinical trial of Genz-112638, an oral therapy being developed for
the treatment of Gaucher disease. Genzyme will conduct the study in
patients with Type 1 Gaucher disease at medical centers in Europe, Israel,
South America and the United States.
Initiation of this trial reflects Genzyme's strong commitment to
developing a next-generation therapy for patients with Gaucher disease that
has the potential to decrease or eliminate their need for enzyme
replacement therapy, the current standard of care.
"We are proud to advance our small molecule program into Phase 2
testing, which will help enable us to determine the potential of this
therapy to have a meaningful impact on the lives of patients with Gaucher
disease," said Rich Moscicki, MD, chief medical officer, Genzyme. "We
remain committed to continued innovation on behalf of people with Gaucher
disease, through our efforts to develop new treatment approaches and to
further improve upon the care patients receive with enzyme replacement
therapy."
Genz-112638, a ceramide analog given as an oral capsule, is designed to
inhibit the enzyme glucosylceramide synthase, which results in reduced
production of glucocerebroside. This is the substance that builds up in the
cells and tissues of people with Gaucher disease. In preclinical studies,
the molecule has shown high potency and specificity. In addition to Gaucher
disease, there are a variety of other conditions that can be caused by
malfunctions in the pathway targeted by this molecule, such as Tay-Sachs,
Sandhoff, and Fabry diseases.
The open-label trial will evaluate patient response for one year
according to several primary endpoints, including changes in hemoglobin,
platelet levels and spleen volume. Other endpoints to be investigated
include change in liver volume and a series of biomarkers and quality of
life indicators relevant to Gaucher disease.
Initiation of the Phase 2 program follows completion of an extensive
pre-clinical research effort and a Phase 1 program that involved more than
120 subjects in three separate studies. The first public presentation of
the Genz-112638 pre-clinical and Phase 1 data occurred July 19 at the
European Working Group on Gaucher Disease Workshop in Cambridge, UK.
Genzyme's Commitment to Gaucher Disease
Development of Genz-112638 reflects Genzyme's long-standing commitment
to improving the care of patients with Gaucher disease. Since introducing
enzyme replacement therapy more than 15 years ago, Genzyme has engaged in
extensive basic science research to further improve its safety and
effectiveness. Post- approval clinical trials have helped to demonstrate
the impact enzyme replacement therapy can have on different manifestations
of the disease, such as bone involvement. Management of the Gaucher
Registry, the largest database of its kind, has yielded insights that have
shaped how physicians and their patients manage Gaucher disease. Genzyme
has also performed extensive research into gene therapy for single gene
disorders like Gaucher disease.
About Genzyme
One of the world's leading biotechnology companies, Genzyme is
dedicated to making a major positive impact on the lives of people with
serious diseases. This year marks the 25th anniversary of Genzyme's
founding. Since 1981, the company has grown from a small start-up to a
diversified enterprise with more than 8,500 employees in locations spanning
the globe and 2005 revenues of $2.7 billion. Genzyme has been selected by
FORTUNE as one of the "100 Best Companies to Work for" in the United
States.
With many established products and services helping patients in more
than 80 countries, Genzyme is a leader in the effort to develop and apply
the most advanced technologies in the life sciences. The company's products
and services are focused on rare inherited disorders, kidney disease,
orthopaedics, cancer, transplant and immune diseases, and diagnostic
testing. Genzyme's commitment to innovation continues today with a
substantial development program focused on these fields, as well as heart
disease and other areas of unmet medical need.
This press release contains forward-looking statements, including
statements regarding clinical trial plans for GENZ-112638; potential
indications for GENZ-112638, including Gaucher disease; and expectations
regarding the potency and specificity of GENZ-112638. These statements are
subject to risks and uncertainties that could cause actual results to
differ materially from those forecast in these forward-looking statements.
These risks and uncertainties include, among others, the results and timing
of preclinical and clinical studies of GENZ-112638 for Gaucher disease and
other potential indications; the enrollment rate for the Phase 2 trial of
GENZ-112638; Genzyme's ability to obtain regulatory approvals and the
timing of receipt of such approvals; and the risks and uncertainties
described in Genzyme's SEC reports filed under the Securities Exchange Act
of 1934, including the factors discussed under the caption "Factors
Affecting Future Operating Results" in Genzyme's Quarterly Report on Form
10-Q for the quarter ended March 31, 2006. Genzyme cautions investors not
to place substantial reliance on the forward-looking statements contained
in this press release. These statements speak only as of the date of this
press release and Genzyme undertakes no obligation to update or revise the
statements.
Genzyme(R) is a registered trademark of Genzyme Corporation. All rights
reserved.
Genzyme's press releases and other company information are available at
http://www.genzyme.com and by calling Genzyme's investor information line
at 1-800-905-4369 within the United States or 1-703-797-1866 outside the
United States.
Media Contact: Investor Contact:
Dan Quinn Sally Curley
(617) 768-6849 (617) 768-6140
SOURCE Genzyme Corporation
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http://www.genzyme.com/
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CONTACT:
Dan Quinn (Media Contact), +1-617-768-6849;
or Sally Curley (Investor Contact), +1-617-768-6140, both of
Genzyme Corporation
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