Two-Year, Pre-Planned Interim Analysis Demonstrates Robust, Statistically
Significant Treatment Effect of Campath Compared to Rebif(R)
CAMBRIDGE, Mass., Sept. 14 /PRNewswire-FirstCall/ -- Genzyme
Corporation (Nasdaq: GENZ) today announced two-year interim results from a
Phase 2 trial comparing Campath(R) (alemtuzumab) with Rebif(R) (interferon
beta-1a) for the treatment of multiple sclerosis. The results derive from a
pre-specified analysis conducted after two years of treatment for 334
patients in the planned three-year trial. This review was conducted in
conjunction with an independent data and safety monitoring board.
As previously announced, dosing of alemtuzumab in this study was
suspended in September 2005 after three patients developed immune
thrombocytopenic purpura (ITP), a treatable condition in which patients
experience a low platelet count as a result of an immune response directed
against the platelets. At that time, most patients had received two cycles
of therapy with alemtuzumab. Treatment with Rebif in the control arm has
continued without interruption. The trial remains on clinical hold in the
United States, and Genzyme is working closely with clinical investigators
and regulatory agencies to complete the study and ensure that the risk of
ITP is well understood and managed. The company discourages physicians and
patients from using alemtuzumab for MS outside of a clinical trial setting
in which procedures are in place for managing ITP risk.
Efficacy Results
Analysis of the first co-primary endpoint showed that patients taking
alemtuzumab at high and low doses experienced at least a 75 percent
reduction in the risk for relapse after at least two years of follow up
when compared to patients treated with interferon beta-1a. This difference
was statistically significant in favor of the alemtuzumab patients at both
high and low doses, with a p-value less than the pre-specified value
(p=0.00328) assigned for the two-year interim analysis.
Analysis of the other co-primary endpoint showed that patients taking
alemtuzumab at high and low doses experienced at least a 65 percent
reduction in the risk for progression of clinically significant disability
when compared to patients treated with interferon beta-1a. This difference
was statistically significant in favor of the alemtuzumab patients at both
high and low doses, with a p-value less than the pre-specified value
(p=0.01194) assigned for the two-year interim analysis.
Results of additional secondary and tertiary efficacy endpoints,
including MRI data, functional assessments, and quality of life measures,
support the findings seen in the co-primary endpoints.
"These results continue to demonstrate that alemtuzumab has great
potential to make a meaningful impact on the treatment of multiple
sclerosis," said Richard A. Moscicki, MD, chief medical officer for
Genzyme. "We will work with regulatory agencies in the United States and
Europe, as well as our clinical investigators, to successfully complete
this important trial and to prepare for the initiation of a Phase 3 trial
in the first half of 2007."
Genzyme has requested a meeting with the U.S. Food and Drug
Administration to present these data and to address the next steps in the
development of alemtuzumab. The company has already received scientific
advice from the European Medicines Agency for moving forward with a Phase 3
study.
Safety Results and Risk Management
Dosing of alemtuzumab in this study was suspended in the United States
in September 2005 after three patients were diagnosed with ITP. The first
patient to present with ITP died from the disease. Genzyme immediately
implemented enhanced monitoring for ITP and has since created a
comprehensive risk management plan to help physicians and patients
participating in the trial detect ITP early and minimize the risk of
complications. These efforts have been effective and have enabled the
identification of all five additional patients with ITP symptoms. All
patients requiring medical treatment for ITP have responded well. To date,
a total of six patients have been diagnosed with ITP in this trial.
Other than ITP, serious adverse events related to treatment occurred
among four patients treated with the low dose of alemtuzumab and four
treated with the high dose. Two patients treated with interferon beta-1a
experienced serious adverse events related to treatment. Common non-serious
adverse events included infusion reactions in the alemtuzumab patients and
flu-like symptoms in patients using interferon beta-1a. The incidence of
all thyroid adverse events, including Graves' disease, was less than
expected compared to reports in the literature on the use of Campath in MS.
Safety information from the study related both to ITP and thyroid disorders
will be presented in two weeks at the annual meeting of the European
Committee for Treatment and Research in Multiple Sclerosis.
Phase 2 Trial Design
The phase 2 trial randomized 334 patients with active
relapsing-remitting multiple sclerosis at 49 medical centers in Europe and
the United States. Patients in the trial were treated with alemtuzumab at
one of two doses (12 or 24/mg per day for five days at initial treatment,
and three days of re-treatment), or interferon beta-1a (44 mcg administered
three times per week, as indicated in its product label). The alemtuzumab
regimen was administered once per year by intravenous infusion, while the
interferon beta-1a regimen was administered three times per week by
subcutaneous injection. The randomized trial compares the safety and
efficacy of alemtuzumab with interferon beta-1a using two primary
endpoints: the rate of relapse of MS symptoms, and the time to progression
of clinically significant disability (time to Sustained Accumulated
Disability over six months as measured by Expanded Disability Status Scale
[EDSS]). Although treating physicians are aware of which treatment patients
received, independent (blinded) neurologists assessed the disability
efficacy endpoint.
About Multiple Sclerosis
Multiple Sclerosis (MS) is a chronic, debilitating disease in which the
immune system attacks the person's brain and spinal cord. The disease
causes a wide range of symptoms including fatigue, difficulty walking,
numbness, and vision problems, and can progress to cause severe disability.
Relapsing-remitting MS is the most common form of this disease.
About Campath
Campath (alemtuzumab), is indicated in the United States for the
treatment of B-cell chronic lymphocytic leukemia (B-CLL) in patients who
have been treated with alkylating agents and who have failed fludarabine
therapy. Campath continues to be available for its labeled indication in
leukemia. Determination of the effectiveness of Campath is based on overall
response rates. A randomized, controlled phase 3 trial demonstrating
clinical benefits in previously untreated patients with B-CLL has been
completed, and final safety and efficacy results have been submitted for
presentation at a medical meeting later this year. Campath is a humanized
monoclonal antibody that binds to a specific target, CD52, on cell surfaces
directing the body's immune system to destroy malignant cells. It is the
first and only monoclonal antibody approved by the FDA for the treatment of
patients with B-CLL.
Genzyme is developing alemtuzumab in oncology, multiple sclerosis and
other indications. Schering AG holds exclusive worldwide marketing and
distribution rights to Campath. The product is marketed in the U.S. by
Berlex Laboratories, a U.S. affiliate of Schering AG. Campath was launched
in the U.S. in June 2001, and in Europe, where it is named MabCampath(R),
in August 2001.
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 about clinical trial results, regulatory plans and expected
timelines for alemtuzumab, including the initiation of a Phase 3 trial in
MS patients and the timing thereof, and the ability to manage patient
safety and recommence alemtuzumab administration in the ongoing Phase 2
clinical trial in MS. 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: that final results of the clinical
trial demonstrate safety and efficacy comparable to the interim data that
have emerged to date, the actual timing and content of submissions to and
decisions made by the regulatory authorities, institutional review boards,
data safety monitoring boards and treating physicians regarding the
continued administration of alemtuzumab to MS patients, Genzyme's ability
to develop and obtain approval of a patient safety plan, and the other
risks and uncertainties described in reports filed by Genzyme with the
Securities and Exchange Commission. Please see the disclosure 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 quarter ended
June 30, 2006 for a more complete discussion of these and other risks.
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 and Campath(R) are registered trademarks of Genzyme
Corporation. Rebif(R) is a registered trademark of Serono, Inc.
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:
Dan Quinn Sally Curley
(617) 768-6849 (617) 768-6140
SOURCE Genzyme Corporation
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CONTACT:
Dan Quinn (Media), +1-617-768-6849; or Sally
Curley (Investor), +1-617-768-6140, both of Genzyme Corporation
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