Genzyme Completes Enrollment In Pivotal, Phase 2 Clinical Trial Of Clofarabine In Adult AML

Genzyme Corp.
(Nasdaq: GENZ) announced that the company has completed enrollment of
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. Data from this study are expected to provide substantial
evidence for expanding the current product label into adult AML.


"We are very pleased to have reached this milestone and to have done so
in a timeframe that we expect will allow us to present preliminary data at
ASCO next spring," stated Mark Enyedy, president of Genzyme Oncology. "The
completion of patient enrollment in this clinical study is another
important step in our plan to broaden the Clolar label to benefit a larger
patient population and address multiple lines of adult AML."



Genzyme has held preliminary discussions with the U.S. Food and Drug
Administration in advance of submitting a supplemental new drug application
(sNDA) for clofarabine as an initial treatment in older adults with AML.
The company expects to file this sNDA in the second half of next year.



Significant Unmet Medical Need



The clinical trial, known as CLASSIC II, 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. Older AML patients often have
disease features such as unfavorable cytogenetics and prior blood disorders
that result in lower response rates and poorer outcomes to standard
induction chemotherapy. In addition, standard therapy is poorly tolerated
in older patients and early induction mortality exceeds 30 percent in
patients with poor risk factors.



This study 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.



Study Design



The CLASSIC II trial was designed to enroll 109 patients at 20 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 disorder such as myelodysplastic syndromes (MDS),
poor health performance, or intermediate or unfavorable cytogenetics.
















The primary endpoint is overall remission rate measured as either
complete remission or complete remission with incomplete platelet recovery.
Secondary endpoints include duration of remission, disease free survival,
overall survival, safety and thirty-day mortality rate.



Patients receive an induction cycle of intravenous clofarabine
administered as 30mg/m2 per day for five consecutive days then, based on
response, receive up to five additional cycles of treatment at a dose of 20
mg/m2 per day for five consecutive days. The first stage of the CLASSIC II
trial required at least 11 responses in the first 59 patients to continue
to the second stage of the study. In September, Genzyme announced that the
number of responding patients had exceeded this requirement.



Clolar Clinical Development



A separate, phase 3 pivotal study (CLASSIC I) of clofarabine in adult
AML patients aged 55 and older and previously treated with at least one,
but not more than two, prior induction regimens is underway. It is a
randomized, double-blind, controlled study that will compare the
combination of clofarabine 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 compare clofarabine to standard therapy in 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.


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
statements regarding the potential administration, dosing and therapeutic
benefit of Clolar in various cancer indications; the expected results of
the data generated from the Clolar clinical 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; the timing and content of decisions by
the FDA related to clinical trials and approval of Clolar in additional
indications; the actual efficacy and safety of Clolar for the indications
in which it is being tested; and the risks and uncertainties described in
reports filed by Genzyme with the U.S. Securities and Exchange Commission,
including without limitation the factors discussed under the caption "Risk
Factors" in Genzyme's Quarterly Report on Form 10-Q for the quarter ended
September 30, 2007. We caution investors not to place undue reliance on the
forward-looking statements contained in this press release. These
statements speak only as of the date of this press release, and we
undertake no obligation to update or revise the statements.



Genzyme(R) and Clolar(R) are registered trademarks of Genzyme
Corporation. All rights reserved.


Genzyme Corp.

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