CAMBRIDGE, Mass., Nov. 27 /PRNewswire-FirstCall/ -- Genzyme Corporation
(Nasdaq: GENZ) announced today that it has begun treating patients in a
phase 2 clinical trial examining the safety and effectiveness of Clolar(R)
(clofarabine) in previously untreated, older adult patients with acute
myelogenous leukemia (AML) who are unlikely to benefit from standard
induction therapy. This is Genzyme's second pivotal clinical study of
clofarabine in adult patients with AML to commence this year, and it is
expected to provide substantial support for expanding the current product
label.
"We are very pleased to begin 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 another
important step in our plan to broaden Clolar's label to benefit a larger
patient population and address multiple lines of adult AML."
The trial is designed to address a high unmet medical need among older
AML patients who currently have limited treatment options. According to the
American Cancer Society, each year approximately 6,500 people over the age
of 60 are diagnosed with AML in the U.S. The median survival for those
receiving therapy can vary from one to thirteen months, and the five-year
survival rate over the past three decades remains at less than 15 percent.
Standard therapy is poorly tolerated and early induction mortality exceeds
30 percent in patients with poor risk factors.
"While the outlook for children and young adults with AML has improved
during the last three decades due to advances in chemotherapy and bone
marrow transplant, the majority of AML patients are over the age of sixty
years and not able to tolerate these curative approaches," stated Harry
Erba, M.D., Ph.D., of the University of Michigan Comprehensive Cancer
Center. "This Genzyme-sponsored trial of clofarabine represents an attempt
to develop a new effective therapy for this aggressive leukemia that can be
tolerated by older patients."
Hagop M. Kantarjian, M.D. of The University of Texas M. D. Anderson
Cancer Center, added that complete remission and response rates remain low
among older AML patients. "Standard therapies for AML in this population
are unsatisfactory. Older patients are often faced with treatment choices
at either end of the spectrum: either toxic combination chemotherapy that
has a high chance of treatment-related morbidity and mortality despite a
low chance of cure, or very shortened survival with supportive care alone."
The clinical trial, known as CLASSIC II, builds on promising results
from two phase 2 studies of clofarabine in previously untreated older
patients with AML deemed unfit for chemotherapy. These studies were
conducted by Alan Burnett, M.D., of Cardiff University in the United
Kingdom. Dr. Burnett will present updated results from his UK clinical
trials at the annual American Society of Hematology meeting taking place
next month in Orlando, Florida.
Study Design
The CLASSIC II trial will enroll 109 patients at approximately 30 sites
in the U.S. Patients must have AML, be 60 years or older and have at least
one of the following adverse prognostic factors: age greater than or equal
to 70, prior hematological disease such as myelodysplastic syndrome, poor
health performance, or intermediate or unfavorable cytogenetics.
The primary endpoint is overall response measured as either complete
response or complete response with incomplete platelet recovery. Secondary
endpoints include duration of remission, disease free survival, overall
survival, safety and thirty-day mortality rate.
Patients will receive an induction cycle of intravenous clofarabine
administered as 30mg/m2 per day for five consecutive days, and based on
response may receive up to five additional cycles of treatment at a dose of
20 mg/m2 per day for five consecutive days.
Clolar Clinical Development
A separate, phase 3 pivotal study (CLASSIC I) of clofarabine in adult
AML patients aged 60 and older and previously treated with at least one,
but not more than two, prior induction regimens began earlier this year. It
is a randomized, double-blind, controlled study that will compare the
combination of Clolar and cytarabine (Ara-C) to cytarabine alone.
A second phase 3 study of clofarabine sponsored by the Eastern
Cooperative Oncology Group is expected to begin enrolling patients 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.
Genzyme also is actively exploring additional therapeutic indications
for Clolar, including in 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. Suppression of
bone marrow function, which is usually reversible and dose dependent,
should be anticipated and is likely to increase the risk of infection,
including severe sepsis. Administration of Clolar results in a rapid
reduction of peripheral leukemia cells. Patients should be evaluated and
monitored for signs and symptoms of tumor lysis syndrome and cytokine
release (e.g., tachypnea, tachycardia, hypotension, pulmonary edema) that
could develop into systemic inflammatory response syndrome (SIRS)/capillary
leak syndrome, and organ dysfunction. Clolar should be discontinued
immediately in the event of clinically significant signs or symptoms of
SIRS or capillary leak syndrome.
The most common side effects seen after Clolar treatment, regardless of
causality, were gastrointestinal tract symptoms, including vomiting,
nausea, and diarrhea; hematologic effects including anemia, leukopenia,
thrombocytopenia, neutropenia, and febrile neutropenia; and infection.
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 primarily through the kidneys. Concomitant use of
medications known to induce hepatic toxicity should be avoided. 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.
Clolar may cause fetal harm when administered to a pregnant woman.
Women of childbearing potential should be advised to avoid becoming
pregnant and avoid breast feeding while receiving treatment with Clolar.
For more information about Clolar, please call 1-800-RX CLOLAR or visit
http://www.CLOLAR.com.
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.
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 September 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.
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 Carol Miceli
(617) 768-6690 (617) 768-6602
SOURCE Genzyme Corporation
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CONTACT:
Media Contact: Maria Cantor, +1-617-768-6690,
or Investor Contact: Carol Miceli, +1-617-768-6602, both of
Genzyme Corporation
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