Genzyme Corp.
(Nasdaq: GENZ) today announced that it has successfully completed its phase
3 trial of Mozobil(TM) (plerixafor) in non-Hodgkin's lymphoma (NHL), and
that the trial has robustly met its primary and secondary endpoints.
The randomized, double-blind, placebo-controlled trial included 298
patients who were undergoing a hematopoietic stem cell transplant (HSCT)
for NHL at medical centers in the United States and Canada. It examined the
effectiveness of Mozobil in increasing the number of hematopoietic stem
cells collected for a transplant. The study compared the hematopoietic stem
cell yield from patients treated with Mozobil in combination with G-CSF to
patients treated with G-CSF in combination with placebo. G-CSF is the
standard of care for stimulating the mobilization of stem cells from the
bone marrow; Mozobil is designed to allow for the more rapid and effective
release of those stem cells from the marrow into the circulating blood for
collection by apheresis.
In the primary efficacy endpoint, 59 percent of patients treated with a
combination of Mozobil and G-CSF achieved the target threshold for
collection of at least 5 million CD34+cells/kg from the peripheral blood
with four or fewer days of apheresis sessions, compared with 20 percent of
patients in the G-CSF/placebo group. The three-fold increase was highly
statistically significant in favor of the Mozobil-treated patients
(p
"These are very impressive results with far-reaching clinical
importance for patients undergoing a stem cell transplant for lymphoma,"
said Principal Investigator John F. DiPersio, M.D., Ph.D., professor,
Washington University, St. Louis. "Current literature suggests that
increasing the number of stem cells in circulation and the number collected
at the time of apheresis may improve the outcomes of patients undergoing a
stem cell transplant, reduce the costs associated with stem cell collection
and, more importantly, broaden the pool of patients for whom
transplantation is an option."
Based on these results Genzyme expects to file for US and European
approval in lymphoma in the first half of 2008. In addition, Genzyme is
completing a second phase 3 trial of Mozobil in multiple myeloma, and
results are expected in the coming weeks.
About Mozobil
Mozobil, a novel small molecule CXCR4 chemokine antagonist, has been
shown in multiple earlier studies to rapidly and effectively increase the
number of stem cells in circulation in the blood. Once circulating in the
blood, stem cells can be collected for use in a stem cell transplant.
Mozobil has been granted special protocol assessment and orphan drug status
in the United States and European Union and the pivotal trials have
undergone Special Protocol Assessment by the FDA and Protocol Assistance by
the EMEA. Genzyme intends to commercialize Mozobil through its existing
global transplant business to hematologists and hematopoietic stem cell
transplant centers in more than 50 countries throughout the world. Genzyme
has been developing Mozobil since its acquisition of AnorMED, Inc. in 2006.
Approximately 55,000 stem cell transplants are performed each year for
multiple myeloma, Hodgkin's and non-Hodgkin's lymphoma, and other
conditions in markets where Genzyme has a commercial infrastructure,
including the United States, Europe, Latin America and the Asian Pacific
countries.
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.
This press release contains forward-looking statements, including the
statements regarding: the anticipated clinical importance for patients
undergoing stem cell transplants, the anticipated improvements with respect
to patient outcomes, the potential increase in the number of patients for
whom transplantation may become available, the timing expectations
associated with the multiple myeloma phase 3 clinical trial results,
Genzyme's anticipated timing associated with regulatory submissions for US
and European regulatory approvals for Mozobil and Genzyme's global
commercialization plans for Mozobil and its ability to leverage its
existing commercial infrastructure. These statements are subject to risks
and uncertainties that could cause actual results to differ materially from
those projected in these forward-looking statements. These risks and
uncertainties include, among others, the possibility of unfavorable
multiple myeloma phase 3 clinical trial results, the failure of Mozobil to
receive regulatory approvals for the label or on the schedule expected, the
uncertainties of launching a new product on a global scale following
receipt of applicable regulatory approvals due to misestimates of the time
and resources required to do so, or for other reasons, the failure of
Mozobil to receive favorable pricing or reimbursement; the possible
inaccuracies of Genzyme's analysis with respect to markets and number of
potential patients for Mozobil; 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 the information under the heading "Factors Affecting Future
Operating Results" in the Management's Discussion and Analysis of Financial
Condition and Results of Operations section of the Genzyme Quarterly Report
on Form 10-Q for the quarter ending 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.
Genzyme(R) is a registered trademark and Mozobil TM is a trademark of
Genzyme Corporation. All rights reserved.
Genzyme Corp.
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