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SMALL BUSINESS
First Patient Enrolled in Phase 3 Trials of Genzyme Oral Capsule for Gaucher Disease
Business Wire
Genzyme
Corporation (NASDAQ: GENZ) today announced that the company has
begun enrollment in the first of two global, multi-center, phase 3
trials of Genz-112638, a potential new oral therapy for Gaucher disease
type 1. The two multi-national, multi-center trials are being conducted
to evaluate the safety and efficacy of the small molecule.
The first trial, known as ENCORE, has enrolled its first patient. ENCORE
is a randomized, open-label study for adult patients with Gaucher
disease type 1 designed to compare Genz-112638 to Cerezyme®
(imiglucerase for injection). Adult patients who have previously
received Cerezyme for at least three years and have reached their
therapeutic goals may qualify for this trial. Genzyme expects to enroll
nearly 200 patients in the ENCORE trial, with a treatment duration of
one year.
The ENGAGE trial is a randomized, double-blind, placebo-controlled study
for patients with a confirmed diagnosis of Gaucher disease type 1.
Patients with intact spleens who have not been treated in the last 12
months for Gaucher disease may qualify for this study. Genzyme expects
to enroll 36 patients in the ENGAGE trial, with a treatment duration of
nine months.
Currently over 35 centers in more than 20 countries are participating in
these trials. Genzyme expects that the number of recruiting centers will
expand, as more sites receive regulatory approval to proceed. At the
conclusion of both trials, patients will be offered the option of
continuing on therapy with Genz-112638 as part of the extension phase of
the program.
“We remain very enthusiastic about this potential new therapy for
Gaucher disease, which could offer flexibility and choice to patients
and to physicians,” said Genzyme Senior Vice President Geoff McDonough,
M.D. “Our studies to date for Genz-112638 suggest a potent and
highly-specific therapy that was well-tolerated and that we believe
could impact patients in a meaningful, positive way.”
To learn more about these trials for Genz-112638, contact Genzyme
Medical Information at
medinfo@genzyme.com
or 1-800-745-4447. More information can also be found at
www.clinicaltrials.gov.
About Genz-112638
Genz-112638, a novel glucosylceramide analog given orally, is designed
to partially inhibit the enzyme glucosylceramide synthase, which results
in reduced production of glucosylceramide. Glucosylceramide 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 February, Genzyme reported that the phase 2 clinical
trial of Genz-112638 met its composite primary efficacy endpoint: a
clinically meaningful response in at least two of three endpoints
(improvements in spleen size, hemoglobin and platelet levels) in
individual patients after the 52-week study period. The safety analysis
for the phase 2 trial demonstrated that Genz-112638 was well tolerated.
Related adverse events in the phase 2 study included infrequent
abdominal discomfort and diarrhea, as well as transient palpitations and
headache. Patients continue to be treated in the extension phase of the
study and have now completed two years of treatment.
Based on its mechanism of action, which is independent of genotype,
Genz-112638 is a potential therapy for all adult patients with Gaucher
disease type 1. The compound was developed with James A. Shayman, M.D.
from the University of Michigan.
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 11,000 employees in locations
spanning the globe and 2008 revenues of $4.6 billion.
With many established products and services helping patients in
approximately 100 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
disease, and diagnostic testing. Genzyme's commitment to innovation
continues today with a substantial development program focused on these
fields, as well as cardiovascular disease, neurodegenerative diseases,
and other areas of unmet medical need.
This press release contains forward-looking statements regarding
Genzyme’s clinical development plan for Genz-112638 as a potential
investigational therapy for Gaucher disease, including that it expects
to fully enroll its Phase 3 clinical studies for Genz- 112638, expects
to expand its recruiting capacity; and believes Genz-112638 could offer
flexibility and choice to patients and physicians and could be a potent,
highly specific and well tolerated therapy that could impact patients in
a positive and meaningful way. These statements are subject to risks and
uncertainties that could cause actual results to differ materially from
those forecasted. These risks and uncertainties include, among others:
Genzyme’s ability to pursue its Phase 3 studies expeditiously and in a
broad group of Gaucher type I patients, Genzyme’s ability to develop
Genz-112638 into a meaningful treatment option for Gaucher type I
patients, 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 10Q for the second quarter of 2009. Genzyme
cautions investors not to place substantial reliance on the
forward-looking statements contained in this press release. These
statements speak only as of today’s date and Genzyme undertakes no
obligation to update or revise the statements.
Genzyme
® and Cerezyme
® are registered trademarks
of Genzyme Corporation or its subsidiaries. All rights reserved.
Genzyme’s press releases and other company information are available at
www.genzyme.com
and by calling Genzyme’s investor information line at 1-800-905-4369
within the United States or 1-678-999-4572 outside the United States.
Copyright Business Wire 2009
2009-10-20 14:01:00
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