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News from Genzyme May 16, 2007
Genzyme Announces Positive
Initial Observations in Trial Evaluating Novel Oral Treatment for
Gaucher Disease
Date: May 16 2007
Genzyme Corp. (Nasdaq: GENZ) announced today that it has completed
enrollment in the ongoing Phase 2 trial of Genz-112638, a novel oral
therapy being developed for the treatment of Gaucher disease. Based
on positive results seen in the trial to date, Genzyme intends to
meet with regulatory agencies in the coming weeks to discuss an
expedited development strategy.
Initial observations from the first five patients suggest that
Genz-112638 may produce a rapid and meaningful impact on important
clinical endpoints including reductions in spleen and liver volume,
and an increase in platelet counts and hemoglobin concentration.
Safety observations from all patients enrolled to date suggest that
the only drug-related adverse events seen in the trial have been
mild and transient in nature, including one possibly related serious
adverse event that is currently being investigated. These early
findings will be presented today at Genzyme’s Analyst Day, and full
trial results will be available in mid-2008.
If these early improvements continue and are observed in other
patients enrolled in the trial, Genz-112638 may represent a
promising novel approach to treating patients with Gaucher disease.
“We are very encouraged by our first observations from this trial,”
said David Meeker MD, president of the Lysosomal Storage Disorder
business. “Cerezyme has had a remarkable effect on the lives of
patients with Gaucher Disease. We have set a very high bar for
ourselves in trying to develop a convenient oral therapy that can
provide a safe and effective choice for patients. We look forward to
developing this molecule further and exploring the role it may play
in the treatment of patients with Gaucher disease.”
The open-label trial has enrolled patients with Type 1 Gaucher
disease at medical centers in Europe, Israel, North America and
South America.
About Genz-112638
Genz-112638, a novel ceramide analog given orally, 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 Fabry disease, and Genzyme
intends to explore studies in this area.
Initiation of the Phase 2 program followed completion of an
extensive pre-clinical research effort and a Phase 1 program that
involved more than 120 subjects in three separate studies.
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 Ceredase in 1991, Genzyme has engaged in extensive
basic science research to further improve the management of Gaucher
patients. Post-approval experience has helped to demonstrate the
positive and enduring impact Ceredase and Cerezyme can have on
different manifestations of the disease, such as anemia,
thrombocytopenia, hepatosplenomegaly, and bone involvement. Data
generated by the Gaucher Registry, the largest database of its kind,
have yielded insights that have shaped how physicians and their
patients manage Gaucher disease. Genzyme has also conducted
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. Since 1981, the company has grown from a
small start-up to a diversified enterprise with more than 9,000
employees in locations spanning the globe and 2006 revenues of $3.2
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
nearly 90 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
diagnostic testing. Genzyme's commitment to innovation continues
today with a substantial development program focused on these fields,
as well as immune disease, infectious disease, and other areas of
unmet medical need.
Forward Looking Statements
This press release contains forward-looking statements regarding
Genzyme’s business plans and strategies, including: Genzyme’s
intention to meet with regulatory authorities to discuss an
expedited regulatory strategy and the expected timing of that
meeting; the potential of Genz-112638 to treat Gaucher disease; and
Genzyme’s intention to pursue studies of Genz-112638 for treatment
of other diseases. 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, Genzyme's ability to
successfully complete clinical development of, and secure regulatory
approvals for, Genz-112638 for the treatment of Gaucher disease;
that early improvements in patients are not observed in new patients
and/or over a longer period of time; the willingness of regulatory
authorities to meet with Genzyme and approve an expedited regulatory
strategy; 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 "Risk Factors" in Genzyme's
Quarterly Report on Form 10-Q for the period ended March 31, 2007.
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
http://www.genzyme.com:80/corp/investors/GENZ
PR-051607.asp
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