International Phase 3 Trials of This Non-Antibiotic Therapy Progressing
CAMBRIDGE, Mass., Aug. 15 /PRNewswire-FirstCall/ -- Genzyme Corporation
(Nasdaq: GENZ) announced today that results from its phase 2 trial of
tolevamer, an investigational polymer therapy for patients with Clostridium
difficile-associated diarrhea (CDAD), have been published in the August 15
issue of Clinical Infectious Diseases.
Tolevamer is a novel therapy that could be the first non-antibiotic
treatment approved for CDAD, a sometimes deadly form of infectious diarrhea
caused by the bacterium C. difficile. Tolevamer is designed to bind and
remove from the body toxins released by C. difficile that damage the large
intestine. In an era of increasing concern about the overuse of antibiotics
and the emergence of antibiotic resistance and "superbugs," tolevamer has
the potential not only to treat CDAD, but also to reduce its rate of
recurrence through a non-antibiotic mechanism of action that does not harm
the normal intestinal bacteria that provide protection against C.
difficile.
"We are pleased to have these tolevamer study results featured in a
leading, international infectious disease journal," said John P. Butler,
president and general manager of Genzyme's renal business unit, which is
developing tolevamer. "Our phase 3 trials of tolevamer are making excellent
progress, and we believe this non-antibiotic therapy holds great promise in
addressing a large and growing unmet medical need for a new treatment
approach to CDAD."
The phase 2 trial enrolled nearly 300 patients at 58 sites throughout
the United States, Canada and the United Kingdom in a randomized,
double-blind, active-controlled study to determine the safety and efficacy
of tolevamer versus the standard prescribed dose of the antibiotic
vancomycin. Vancomycin is the only therapy approved in the United States
and Europe for treatment of CDAD. In the phase 2 study, tolevamer
demonstrated similar treatment outcomes as vancomycin in terms of time to
resolution of diarrhea, and a strong trend toward a reduced recurrence rate
of CDAD.
C. difficile is a bacterium found widely in the environment, and is a
frequent contaminant in hospitals and long-term care facilities. It is the
most common cause of infectious diarrhea among hospitalized patients, and
data published recently in the Centers for Disease Control and Prevention
journal Emerging Infectious Diseases show that the rate of reported C.
difficile cases in the United States nearly doubled between 2000 and 2003.
C. difficile is estimated to affect approximately one percent of all
hospitalized patients, and to result in more than 400,000 cases of diarrhea
and colitis, and more than 5,000 deaths annually in the United States. C.
difficile is a worldwide problem. The U.K. Health Protection Agency last
month published a report showing that cases of C. difficile infection in
patients aged 65 and older increased by 17 percent in England over the last
year.
Patients are at risk of developing CDAD when they are treated with
antibiotics that alter the normal, protective bacteria that reside in the
colon. Virtually all antibiotics have been implicated in causing CDAD.
Several deadly outbreaks of CDAD have occurred in the United States, Canada
and Europe in recent years, and a new, more virulent strain of C. difficile
recently was identified and tied to these outbreaks.
Phase 3 Studies Ongoing
Tolevamer is currently being studied in two concurrent clinical trials,
the largest of their kind ever to be conducted for CDAD. One trial is
taking place in North America, and the other in Europe and Australia. These
studies, which began in April 2005, will involve more than 1,000 patients
at approximately 300 sites.
In these trials, tolevamer is being compared to two antibiotic
therapies, vancomycin and metronidazole, which are the most commonly
prescribed treatments for CDAD. Because tolevamer is a non-antibiotic and
will not harm the normal protective intestinal bacteria that prevent C.
difficile proliferation, it is expected to reduce the rate of recurrent
CDAD. Aside from testing tolevamer's efficacy, these are also the first
large, rigorously designed, multi-center clinical studies comparing the
efficacy of the current standards of care.
Genzyme expects to complete the tolevamer trials in 2007 and, pending
regulatory review, the first commercial approval is anticipated in 2008.
Tolevamer has been given fast-track designation by the U.S. Food and Drug
Administration for the treatment of CDAD.
Genzyme is a worldwide leader in developing polymer-based therapies for
serious diseases, with two proven products already on the market.
Renagel(R) (sevelamer hydrochloride) controls serum phosphorus in patients
with chronic kidney disease on dialysis, and WelChol(R) (colesevelam
hydrochloride) reduces LDL cholesterol in certain patients with elevated
LDL levels. In each case, the polymers are designed to provide clinical
benefit by binding and removing unwanted substances from the body.
Tolevamer has been evaluated in several clinical studies to date,
including the phase 2 study, two open-label clinical trials in CDAD
patients, a phase 1 trial in healthy volunteers and an additional phase 1
tolerability study evaluating a new liquid formulation.
About Clostridium difficile
C. difficile proliferates in the setting of altered normal colonic
bacterial flora, most commonly due to the administration of broad-spectrum
antibiotics. Once established in the colon, C. difficile produces toxins
that disrupt the intestinal lining, causing cell death and inflammation
that result in diarrhea and colitis.
Even after successful treatment with the current standard of care,
approximately 20 percent of patients experience a recurrence of CDAD which
may require repeat hospitalization. In addition, a subset of patients with
CDAD develop multiple recurrences of the disease, with symptoms that may
persist for years.
About Genzyme Corporation
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
without limitation statements regarding: the potential benefits of
tolevamer for CDAD; the size of the CDAD patient population; clinical trial
plans for tolevamer; and the anticipated timing of completion of clinical
trials for tolevamer and regulatory approval of tolevamer. 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: the accuracy of
Genzyme's information concerning the CDAD patient population; additional
analysis of the tolevamer phase 2 data; the results of other studies and
whether such results are consistent with the phase 2 tolevamer trial data;
the actual efficacy and safety of tolevamer for CDAD; the actual timing and
results of phase 3 clinical trials; the outcome of discussions with the FDA
and the EMEA regarding clinical studies and the approval of tolevamer and
the timing of such discussions; the timing and content of submissions to
and decisions made by regulatory authorities; and the risks and
uncertainties described in reports filed by Genzyme with the Securities and
Exchange Commission under the Securities Exchange Act of 1934, as amended,
including without limitation the information under the heading "Factors
Affecting Future Operating Results" in Genzyme's Quarterly Report on Form
10-Q for the period ended June 30, 2006. 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.
Renagel(R) is a registered trademark of Genzyme. WelChol(R) is a
registered trademark of Sankyo Pharma, 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:
Erin Emlock Sally Curley
(617) 768-6923 (617) 768-6140
SOURCE Genzyme Corporation
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CONTACT:
Erin Emlock, Media Contact, +1-617-768-6923,
or Sally Curley, Investor Contact, +1-617-768-6140, both of
Genzyme Corporation
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