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Data Suggest Strong Treatment Effect for Patients Using Campath;
Serious Adverse Events in Trial Require Comprehensive Risk Management Plan
CAMBRIDGE, Mass. and Berlin, Sept. 16 /PRNewswire-FirstCall/ -- Genzyme
Corporation (Nasdaq: GENZ) and Schering AG Germany (FSE: SCH; NYSE: SHR) today
announced 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 announced today derive from a pre-specified efficacy
and safety interim analysis conducted after one year of treatment for all
patients in the planned three-year trial. This review was conducted in
conjunction with an independent data safety monitoring board.
Analysis of the primary endpoints after one year of treatment showed a
large treatment effect in favor of alemtuzumab. Review of the data also showed
that three confirmed cases of severe idiopathic thrombocytopenic purpura (ITP)
occurred in the trial. Based on these results, and after consultation with the
U.S. Food and Drug Administration, the companies will continue to collect both
efficacy and safety data from this trial while preparing to initiate a Phase 3
trial. Dosing with alemtuzumab in this trial has been suspended while the
companies work closely with regulatory authorities and clinical investigators
to ensure that a comprehensive approach is in place to manage patient safety.
Campath continues to be available in its current labeled indication for the
treatment of B-cell chronic lymphocytic leukemia.
The Phase 2 trial randomized 334 patients with active relapsing-remitting
multiple sclerosis at 49 medical centers in Europe and the United States.
Patients were treated with alemtuzumab at one of two doses administered in
once a year intravenous infusion regimens, or interferon beta-1a administered
three times per week as indicated in its product label. The randomized, open-
label trial compared the safety and efficacy of alemtuzumab with interferon
beta-1a, examining two primary endpoints: the rate of relapse of MS symptoms,
and the time to progression of clinically significant disability (time to
Sustained Accumulated Disability at six months as measured by Expanded
Disability Status Score [EDSS]). EDSS assessments were blinded and treatment
groups were comparable at baseline for all key demographic and clinical
parameters.
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 one year 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 the high and low
doses according to the p-value (p=0.00267) assigned for the one-year interim
analysis.
In the other co-primary endpoint, patients treated with the high and low
doses of alemtuzumab experienced at least a 60 percent reduction in the risk
for progression of clinically significant disability (p<0.05) when compared to
patients treated with interferon beta-1a. This result did not achieve
statistical significance according to the p-value (p=0.00015) assigned for the
one-year interim analysis.
Safety Results and Risk Management Program
In the trial, serious adverse events related to treatment occurred in two
patients on interferon beta-1a, four patients on the low dose of alemtuzumab,
and five patients on the high dose of alemtuzumab.
Idiopathic thrombocytopenic purpura is a condition in which patients
experience a low platelet count that can result in abnormal bleeding. Of the
three documented cases of ITP, two occurred in the high dose alemtuzumab
group, and one in the low dose group. One case of ITP in the trial resulted in
a fatality. In the two remaining cases, patients have responded to treatment
and are being appropriately managed by their physicians using accepted
treatment regimens.
As expected, common non-serious adverse events included infusion reactions
in the alemtuzumab patients, and flu-like symptoms in patients using
interferon beta-1a.
Genzyme and Schering's risk management plan for ITP has included
notification of regulatory authorities, trial sites and patients, and
consultation with a panel of hematologists with expertise in ITP to advise on
risk management. The companies have moved forward to implement a series of
provisions in the study, including more frequent hematological monitoring, and
patient education about the signs and symptoms of ITP. Genzyme and Schering
are also working to update informed consent forms, to conduct a thorough
review of patient laboratory data, and to seek indicators that might help
identify those at risk for developing these types of problems. The companies
are currently in discussions with the FDA about what additional steps might be
needed to protect patient safety.
Nearly all alemtuzumab patients in the trial have received their second
year's dose. In the coming months, Genzyme and Schering will evaluate the
necessity and timing of the third planned dose. Because the high dose appears
to offer no efficacy advantage compared to that achieved by the low dose
group, the companies will no longer use this dose.
"Based on these results, we will be moving this program forward with a
tremendous sense of urgency," said Henri A. Termeer, chairman and chief
executive officer, Genzyme Corporation. "Both companies are fully committed to
advancing this treatment as intensively, thoughtfully and responsibly as
possible."
"Our early analysis of efficacy from this study is very encouraging. We
have a long history of commitment to advancing therapeutic options for
patients with MS, and both companies will be working hard in order to move
forward in the best interest of patients, balancing potential benefits with
the possible risk of serious side effects," said Marc Rubin, MD, Member of the
Board of Executive Directors, Schering AG, with responsibility for
Development.
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 for injection), 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. Determination of the effectiveness of Campath is based on overall
response rates. Comparative, randomized trials demonstrating increased
survival or clinical benefits such as improvement in disease-related symptoms
have not yet been conducted. 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 and Schering are co-developing alemtuzumab in oncology and other
indications, with Schering having exclusive responsibility for the development
and commercialization of alemtuzumab for solid organ transplantation. Schering
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.
Phase 2 Trial Detail
A total of 334 patients in the trial were randomized to receive either a
low dose of alemtuzumab (12 mg/day for five days), a high dose of alemtuzumab
(24 mg/day for five days), or interferon beta-1a (44 mcg administered three
times per week). At 12 months, patients on alemtuzumab received a dose of 12
or 24 mg/day for three days. Data reviews are scheduled at 12, 24, and 36
months for the co-primary endpoints and a series of secondary endpoints.
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. Founded in 1981, Genzyme has grown from a small start-up to a
diversified enterprise with 7,600 employees in locations spanning the globe,
and 2004 revenues of $2.2 billion. 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, orthopedics, 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.
About Schering AG
Schering AG is a research-based pharmaceutical company. Its activities are
focused on four business areas: Gynecology & Andrology, Oncology, Diagnostic
Imaging as well as Specialized Therapeutics for disabling diseases. As a
global player with innovative products Schering AG aims for leading positions
in specialized markets worldwide. With in-house R&D and supported by an
excellent global network of external partners, Schering AG is securing a
promising product pipeline. Using new ideas, Schering AG aims to make a
recognized contribution to medical progress and strives to improve the quality
of life: making medicine work.
This press release contains forward-looking statements, including
statements about clinical trial results, regulatory plans and expected
timelines for Campath, including the initiation of a Phase 3 trial in MS
patients and the timing thereof, and the ability to manage patient safety and
recommence Campath 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 Campath 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, 2005 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(R), Campath(R), and MabCampath(R) are registered trademarks of
Genzyme Corporation. Rebif(R) is a registered trademark of Ares Trading, S.A.
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.
Conference Call Information
There will be a conference call today at 10:00 a.m. Eastern. If you would
like to participate in the call, please dial (800) 843-7880 or (706) 679-8722
just prior to the start of the call. This call also will be Webcast live on
the investor events section of http://www.genzyme.com. A replay of this call
will be available from 1:30 p.m. Eastern today through midnight on September
22, 2005 by dialing (800) 642-1687 or (706) 645-9291. Please refer to
Conference ID # 9647796.
Schering Contacts: Genzyme Contacts:
Oliver Renner (media) Dan Quinn (media)
+49 30 468 124 31 (617) 768-6849
Marcy Funk (media)
(973) 487-2095 Sally Curley (investors)
(617) 768-6140
Peter Vogt (investors)
+49 30 468 128 38
Joanne Marion (investors)
(973) 487-2164
SOURCE Genzyme Corporation
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CONTACT:
Oliver Renner (media), +49 30 468 124 31 or
Marcy Funk (media), +1-973-487-2095, or Peter Vogt (investors),
+49 30 468 128 38, or Joanne Marion (investors), +1-973-487-2164,
all of Schering; or Dan Quinn (media), +1-617- 768-6849, or Sally
Curley (investors), +1-617-768-6140, both of Genzyme
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