Genzyme Announces Phase 3 Trial of Mozobil in non-Hodgkin'
Meets Primary Endpoint
July 19, 2007: 08:59 AM EST
CAMBRIDGE, Mass., July 19 /PRNewswire-
today announced that it has successfully completed its phase 3 trial
of Mozobil(TM) (plerixafor) in non-Hodgkin'
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<0.0001). The 40 percent absolute difference
between the two treatment groups was nearly double the target that
Genzyme prospectively defined in the protocol for the study, which
was reviewed by FDA as part of the Special Protocol Assessment
process.
In the secondary efficacy endpoint, nearly 87 percent of patients
treated with Mozobil and G-CSF achieved the minimum level of stem
cells generally associated with a successful transplant (2 million
CD34+cells/kg) in four or fewer days of apheresis sessions, compared
with approximately 47 percent in the placebo arm. This result was
also highly statistically significant in favor of the Mozobil-treated
patients (p<0.0001).
The other secondary efficacy endpoints were supportive of these
findings, including analysis of the number of days needed to reach
target ranges for stem cell mobilization, the success of engraftment,
the number of days needed to engraft, and the durability of the
engraftment for the first 100 days.
Mozobil was well tolerated in the trial, with the most common adverse
events being mild gastrointestinal effects and redness at the site of
injection. There were two related serious adverse events seen in the
Mozobil plus G-CSF arm, and one in the G-CSF plus placebo arm.
"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'
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's press releases and other company information are available
at www.genzyme.
at 1-800-905-4369 within the United States or 1-678-999-4572 outside
the United States.
Media Contact: Investor Contact:
Dan Quinn Sally Curley
(617) 768-6849 (617) 768-6140
http://money.
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StemCells subscribers may also be interested in these sites:
Children's Neurobiological Solutions
http://www.CNSfoundation.org/
Cord Blood Registry
http://www.CordBlood.com/at.cgi?a=150123
The CNS Healing Group
http://groups.yahoo.com/group/CNS_Healing
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