CAMBRIDGE, Mass., Sept. 6 /PRNewswire-FirstCall/ -- Genzyme Corporation
(Nasdaq: GENZ) announced today that the first patient has been treated in a
new phase 3 clinical trial examining the safety and effectiveness of
Clolar(R) (clofarabine) in older patients with acute myelogenous leukemia
(AML). This is the first pivotal clinical study of clofarabine in adult
patients with AML, and is expected to provide substantial support for
expanding the current product label.
"We are very pleased to initiate treatment in this clinical study as we
seek to expand Clolar therapy to adult patients," stated Mark Enyedy,
senior vice-president and general manager of Genzyme Oncology. "Significant
data from investigator-sponsored clinical trials have already been
presented regarding the use of Clolar in adult AML and show very
encouraging results. The start of this new clinical study is an important
next step in our plan to broaden Clolar's label to benefit a larger patient
population and address multiple lines of adult AML."
Phase 3 Design and Focus on Adult AML
The trial, designed as a pivotal study for adult AML patients aged 60
and older previously treated with at least one, but not more than two prior
induction regimens, is a randomized, double-blind, controlled study that
will compare the combination of Clolar and cytarabine (Ara-C) to cytarabine
alone. The study will take place at approximately 100 medical centers in
the United States and Canada. It will enroll up to 376 patients with
refractory or relapsed AML.
"There is significant unmet medical need in relapsed, refractory adult
AML and clofarabine could represent an important therapeutic option for
these older patients," stated Stefan Faderl, M.D., The University of Texas
M. D. Anderson Cancer Center and principal investigator of the study. "This
clinical trial is a natural and important extension of M. D. Anderson's
experience with the clofarabine and cytarabine combination. It will help us
to further determine the position of clofarabine combinations in the
management of adult patients with AML."
In addition to safety, the trial will evaluate the effectiveness of one
dose of 40 mg/m2 per day of Clolar combined with 1 g/m2 per day of Ara-C,
compared with 1g/m2 per day of Ara-C combined with placebo. The primary
endpoint is improved overall survival. Other endpoints include overall
remission rate, duration of remission, disease-free survival and event-free
survival and safety and tolerability including hospitalizations. All
patients will be followed for at least two years after their end of
treatment visit.
A separate, phase 2 pivotal study of clofarabine in previously
untreated older AML patients for whom standard induction combination
chemotherapy is unlikely to be of benefit is expected to begin as early as
the fourth quarter of this year. A second phase 3 study of clofarabine
sponsored by the Eastern Cooperative Oncology Group is expected to begin
enrolling patients early next year. This study will focus on untreated AML
patients over the age of 60 who are considered suitable for standard
induction chemotherapy.
Clolar is indicated for the treatment of pediatric patients 1 to 21
years old with relapsed or refractory ALL after at least two prior
regimens. This use is based on the induction of complete responses.
Randomized trials demonstrating increased survival or other clinical
benefit have not been conducted.
Ongoing Clinical Development
Genzyme successfully entered the oncology market in 2005 with the
launch of Clolar, the first cancer treatment approved specifically for
pediatric patients in more than a decade. As in adults, Genzyme is
conducting parallel clinical studies in children. Clolar in combination
with existing therapies is being studied in two phase 1/2 studies, one of
which is underway. These studies are part of post-marketing commitments
that Genzyme made to the FDA following the accelerated approval of Clolar.
The first phase 1/2 trial is evaluating Clolar in combination with the
chemotherapy agents etoposide and cyclophosphamide in patients with no more
than two (AML) or three (ALL) prior induction regimens. The primary
objective of the phase 1 portion of the study is to define the maximum
tolerated dose, dose-limiting toxicities, and recommended phase 2 dose of
clofarabine in combination with these two agents. In the phase 2 portion of
the study involving ALL patients only, the primary objective is to assess
the efficacy of Clolar in combination with etoposide and cyclophosphamide,
as determined by the complete remission, complete remission without
platelet recovery, and overall remission rate. Thirty-nine patients are
expected to be treated during the enrollment period.
The second phase 1/2 trial is sponsored by the Children's Oncology
Group. This trial is evaluating Clolar in combination with cytarabine. Its
primary objective is to measure overall response rate in AML and ALL
patients 30 years of age and younger who have recurrent disease or are
refractory to induction therapy. Eighty-seven patients are expected to be
enrolled in this study over the course of 24 months. Based upon the
experience with these two studies, Genzyme will continue to consult with
the U.S. Food and Drug Administration regarding the final appropriate
design of a randomized phase 3 study to demonstrate clinical benefit in the
pediatric population with ALL.
Genzyme seeks to explore additional therapeutic indications for Clolar
through its support of several investigator initiated studies.
Additionally, Genzyme is enrolling patients in its phase 1 trial of
clofarabine in solid tumors and continues to consider development in
myelodysplastic syndrome (MDS).
About Clolar
Clolar has Orphan Drug designation for adult and pediatric ALL, and
seven years of market exclusivity in the United States for
relapsed/refractory pediatric ALL. The FDA also granted six months of
extended market exclusivity to Clolar under the Best Pharmaceuticals for
Children Act.
Clolar should be administered under the supervision of a qualified
physician experienced in the use of antineoplastic therapy. The most common
side effects seen after Clolar treatment, regardless of causality, are
vomiting, nausea, diarrhea, low blood counts, fever and infection. Careful
monitoring of blood counts during therapy is important since Clolar can
cause reversible and dose dependent suppression of bone marrow function,
which may increase the risk of infection, including severe sepsis.
Administration of Clolar results in a rapid reduction in peripheral
leukemia cells. Patients should be evaluated and monitored for signs and
symptoms of tumor lysis syndrome and cytokine release that could develop
into systemic inflammatory response syndrome (SIRS)/capillary leak
syndrome, and organ dysfunction. Liver and kidney function should be
assessed prior to and during treatment with Clolar, as the liver is a
target organ for Clolar toxicity and Clolar is excreted primary through the
kidneys. Cardiac disorders, including tachycardia, pericardial effusion,
and left ventricular systolic dysfunction, have been noted in up to 35% of
pediatric patients treated with Clolar. However, the presence of these
disorders in patients prior to Clolar administration and/or previous
therapy or concurrent illness in patients receiving Clolar makes the
etiology of these disorders unclear.
For more information about Clolar, please call 1-800-RX CLOLAR or visit
http://www.CLOLAR.com.
This press release contains forward-looking statements, including
statements regarding the potential administration, dosing and therapeutic
benefit of Clolar in various cancer indications; the planned timetable and
enrollment for clinical trials of Clolar and the sites of such trials; and
the requirements and plans for regulatory filings and approvals for Clolar
in additional indications. These risks and uncertainties include, among
others: the timing of discussions with the FDA regarding clinical studies
and approval of Clolar in additional indications; Genzyme's ability to
engage cooperative groups to conduct clinical trials of Clolar and the
ability of all clinical trial sites to enroll patients in the applicable
study; the timing and content of decisions by institutional review boards
and the FDA related to clinical trials and approval of Clolar in additional
indications; and the actual efficacy and safety of Clolar for the
indications in which it is being tested. Please refer to the risks and
uncertainties described in reports filed by Genzyme with the Securities and
Exchange Commission under the heading "Factors Affecting Future Operating
Results" in the Management's Discussion and Analysis of Financial Condition
and Results of Operations section of Genzyme's Quarterly Report on Form
10-Q for the period ended June 30, 2006 for a more complete discussion of
the risks associated with Genzyme's business. 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 these statements.
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. This year marks the 25th anniversary of Genzyme's
founding. Since 1981, the company has grown from a small start-up to a
diversified enterprise with more than 8,500 employees in locations spanning
the globe and 2005 revenues of $2.7 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 more
than 80 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 diseases, and diagnostic
testing. Genzyme's commitment to innovation continues today with a
substantial development program focused on these fields, as well as heart
disease and other areas of unmet medical need.
Genzyme(R) and Clolar(R) are registered trademarks of Genzyme
Corporation. All rights reserved.
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 1-703-797-1866 outside the
United States.
Media Contact: Investor Contact:
Maria Cantor Sally Curley
(617) 768-6690 (617) 768-6140
SOURCE Genzyme Corporation
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Related links:
http://www.genzyme.com
CONTACT:
Maria Cantor, Media Contact, +1-617-768-6690,
or Sally Curley, Investor Contact, +1-617-768-6140, both of
Genzyme
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