Estimates Peak Worldwide Transplant Opportunity of Over $400 Million
Annually
CAMBRIDGE, Mass., Aug. 2 /PRNewswire-FirstCall/ -- Genzyme Corp.
(Nasdaq: GENZ) today announced that its second phase 3 trial of Mozobil(TM)
(plerixafor) has successfully met its primary and secondary endpoints,
demonstrating positive results in multiple myeloma (MM) similar to those
reported two weeks ago in non-Hodgkin's lymphoma.
The combined strength of these two trials -- in which patients with two
types of cancer achieved more rapid and effective mobilization of stem
cells in preparation for transplant than patients treated with current
therapies -- will support Mozobil's regulatory approval, commercialization,
and likely adoption as a standard of care in transplantation.
The randomized, double-blind, placebo-controlled trial included 302
patients who were undergoing a hematopoietic stem cell transplant (HSCT)
for multiple myeloma at medical centers in the United States, Canada, and
Europe. Like the previous trial, it examined the effectiveness of Mozobil
in increasing the number of hematopoietic stem cells collected for a
transplant, comparing the stem cell yield from patients treated with
Mozobil following G- CSF to patients treated with placebo following G-CSF.
In the primary efficacy endpoint, 72 percent of patients treated with
Mozobil and G-CSF achieved the target threshold for collection of at least
6 million CD34+cells/kg from the peripheral blood with two days or fewer of
apheresis sessions, compared with 34 percent of patients in the
G-CSF/placebo group. This two-fold increase was statistically significant
in favor of the Mozobil-treated patients (p<0.0001). Like the previous
trial, these results exceed the 20 percent absolute difference
prospectively defined through the FDA's Special Protocol Assessment as a
successful result.
The median number of days to achieve the target of 6 million cells was
one day for the Mozobil/G-CSF group and four days for the G-CSF/placebo
group. Over half of patients treated with Mozobil and G-CSF reached the
target cells in the first day of apheresis. By comparison, it took four
days for a similar percentage of the G-CSF/placebo group to reach this
threshold.
Secondary outcomes were consistent with the primary endpoint, showing a
statistically significant result in favor of Mozobil in the number of
patients who reached the target threshold of at least 6 million
CD34+cells/kg from the peripheral blood with four days of apheresis, and
the number of patients who reached at least two million cells collected in
four days.
Other secondary outcomes were also supportive, including the success of
engraftment, the number of days needed to engraft, and the durability of
the engraftment for the first 100 days. The trial also robustly met the
primary endpoint specified by the European Agency for the Evaluation of
Medicinal Products (EMEA) -- a composite of successful mobilization and
engraftment.
Mozobil was well tolerated in the trial, with the most common adverse
events being mild gastrointestinal effects and redness at the site of
injection. The only serious adverse event deemed related to treatment
occurred in the G-CSF/placebo group.
"Taken together, these two pivotal trials paint a dramatic picture of
the role that Mozobil will play in the treatment of patients with lymphoma
and multiple myeloma," said Henri A. Termeer, chairman and chief executive
officer, Genzyme. "Mozobil has shown the ability to quickly and predictably
prepare a patient for a transplant, providing a strong clinical and
economic argument in favor of its use in all autologous transplant
procedures. We will work aggressively to gain regulatory approval in these
transplant settings, while also pressing forward to unlock the full
potential of Mozobil in allogeneic transplantation, chemosensitization,
cardiovascular disease, and other promising applications."
Commercialization plans
Based on these results Genzyme expects to file for US and European
regulatory approval in both multiple myeloma and lymphoma in the first half
of 2008. The company anticipates receiving priority review, and marketing
approval by the end of 2008. Genzyme plans to launch the product early in
2009. Mozobil will be marketed by Genzyme's existing transplant business,
which has a commercial presence in more than 50 countries worldwide.
Approximately 55,000 autologous and allogeneic 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. Genzyme believes that over time Mozobil will be
used in the majority of these procedures, fueling peak sales of the product
in the transplant setting of more than $400 million annually.
In addition to its currently studied use in autologous HSCT, Genzyme is
initiating a number of phase 1/2 trials and investigator-initiated trials
to study its use in other settings. An investigator-initiated trial in
allogeneic HSCT is already underway, examining whether Mozobil can improve
the stem cell yield from healthy unrelated donors. In addition, studies
examining whether Mozobil can improve the efficacy of chemotherapy and/or
immunotherapy in various forms of chronic lymphocytic leukemia and acute
myelogenous leukemia will also begin later this year. Genzyme is also
pursuing preclinical work to explore the role that Mozobil may play in cord
blood transplantation, solid organ transplantation, cardiovascular disease,
HIV, renal ischemic disease, and prevention of metastasis in oncology.
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 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.
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 9,500 employees in locations
spanning the globe and 2006 revenues of $3.2 billion. In 2007, Genzyme was
chosen to receive the National Medal of Technology, the highest honor
awarded by the President of the United States for technological innovation.
In 2006 and 2007, Genzyme was 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: Genzyme's expectations regarding peak sales of
Mozobil, the value of the successful multiple myeloma and non-Hodgkin's
lymphoma trials to Mozobil's regulatory, commercialization and standard of
care prospects, the role Mozobil will play in the treatment of patients
with lymphoma and multiple myeloma, the strength of the clinical and
economic arguments favoring use of Mozobil, Genzyme's ability to work
aggressively to gain regulatory approval, Genzyme's ability to unlock the
full potential of Mozobil in allogeneic transplantation,
chemosensitization, graft-versus-host disease and other potential
applications, Genzyme's anticipated timing associated with regulatory
submissions for US and European regulatory approvals, the expectation of
priority review and marketing approval for Mozobil, Genzyme's global
commercialization plans for Mozobil, the size of the transplant market, the
expectation that Mozobil will be used in a majority of stem cell
transplants, Genzyme's ongoing and planned research, preclinical and
clinical studies relating to autologous and allogeneic HSCT, cord blood
transplantation, solid organ transplantation, peripheral arterial disease,
HIV, renal ischemic disease, and prevention of metastasis in oncology.
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 failure of Mozobil to receive regulatory approvals for multiple
myeloma or non- Hodgkin's lymphoma, a possible delay in regulatory
approvals, 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 possibility
that Mozobil will not meet current expectations with respect to adoption in
the transplant market, the possible inaccuracies of Genzyme's analysis with
respect to markets and number of potential patients for Mozobil, the risks
associated with research, preclinical and clinical studies for other
applications of 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.
Conference Call Information
Genzyme Corporation will host a conference call today at 11:00 a.m.
Eastern Time to discuss Mozobil. To participate in the call, please dial
773-799-3828 and refer to pass code "Genzyme." A replay of this call will
be available by dialing 203-369-0672. This call will also be Webcast live
on the investor events section of http://www.genzyme.com. Replays of the call and
the Webcast will be available until midnight on August 9, 2007.
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 1-678-999-4572 outside the
United States.
Media Contact: Investor Contact:
Dan Quinn Sally Curley
(617) 768-6849 (617) 768-6140
SOURCE Genzyme Corp.
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Related links:
http://www.genzyme.com/
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CONTACT:
Media, Dan Quinn, +1-617-768-6849, or
Investors, Sally Curley, +1-617-768-6140, both of Genzyme
Corporation
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