Brief Extension Allows Inclusion of Patients From Japan
CAMBRIDGE, Mass., Feb. 23 /PRNewswire-FirstCall/ -- Transkaryotic
Therapies, Inc. (Nasdaq: TKTX) today announced that it will briefly extend
enrollment in its pivotal clinical trial evaluating iduronate-2-sulfatase
(I2S) enzyme replacement therapy for the treatment of Hunter syndrome (MPS
II), a rare and often fatal disease. TKT had intended to close enrollment in
February 2004. However, with eighty-four patients currently enrolled and
commitments to additional patients, the company believes it will reach its
target of ninety patients by early March 2004. The extension allows the
company to fulfill commitments to four families from Japan who are relocating
to the United States to participate in the trial, referred to as the AIM study
(Assessment of I2S in MPS II). These families are expected to arrive in the
United States during the last week of February 2004. This effort, conducted
with the help of TKT's partner for Japan, Genzyme Corporation, may result in a
slight over-enrollment.
"Our team of investigators has been doing an exceptional job enrolling the
largest clinical trial ever for a lysosomal storage disorder in what will be
record time," said Michael J. Astrue, President and Chief Executive Officer of
TKT. "We are grateful to these young patients and their families for their
sacrifices and dedication, which we hope will help bring this treatment to
patients all over the world."
TKT continues to expect top-line data from the AIM study to be available
in the second quarter of 2005 and, if the results are positive, the company
expects to submit applications for marketing approval in the U.S. and Europe
during the second half of 2005. Additionally, TKT believes, depending on
randomization, the inclusion of patients from Japan in the AIM study may help
fulfill some requirements for Japanese approval.
Mr. Astrue will provide a detailed discussion of the Hunter syndrome
program including its plans for closing enrollment of the AIM study at the BIO
CEO & Investor Conference in New York on Tuesday, February 24, 2004 at 11:30
a.m. The live audio webcast of this presentation will be available at
http://www.tktx.com on TKT's Investor Information website under the Events category.
The AIM study, which began enrolling patients in September, is designed to
evaluate safety and efficacy of weekly and every-other week infusions of I2S,
administered at a dose of 0.5 mg/kg. Ninety patients with Hunter syndrome are
expected to receive a total of 52 infusions of either I2S (patients randomized
to the weekly dosing regimen), I2S alternating with placebo (patients
randomized to the every other week regimen), or placebo. The AIM study is a
twelve month, randomized, double-blind, placebo-controlled trial being
conducted at nine sites around the world. The primary efficacy endpoint in
the trial is a single composite variable which combines two clinical
measurements: forced vital capacity as a measure of respiratory function and
the six-minute walk test as a measure of functional capacity. Additional
efficacy endpoints include measurements of joint range of motion and combined
liver and spleen size. A summary of the AIM study protocol is available
online at http://www.clinicaltrials.gov.
About I2S
I2S is a human iduronate-2-sulfatase produced by genetic engineering
technology intended for long-term treatment of Hunter syndrome. The rationale
for the therapy is that I2S would replace enzyme that is deficient in patients
with Hunter syndrome and either stop or reverse disease progression. I2S has
been designated an orphan drug in both the United States and Europe and is the
only known enzyme replacement therapy in development for the treatment of
Hunter syndrome.
About Hunter Syndrome
Hunter syndrome is a genetic disorder, also referred to as MPS II. This
hereditary disorder is characterized by the body's inability to produce the
enzyme iduronate-2-sulfatase, which is essential in the continuous process of
replacing and breaking down glycosaminoglycans (GAG). As a result, GAG remain
stored in cells in the body causing progressive damage. The symptoms of
Hunter syndrome are usually not visible at birth, but usually start to become
noticeable after the first year of life. Often the first symptoms may include
hernias, frequent ear infections, runny noses, and abnormal facial appearance.
As the disease progresses, a variety of symptoms appear including, enlarged
liver and spleen, heart failure, obstructive airway disease, sleep apnea,
joint stiffness, and, in the severe form, central nervous system involvement.
In severe cases, the life expectancy for patients with Hunter syndrome is
typically 10-15 years of age. However, in the attenuated form of the disease,
patients can survive into the fifth or sixth decade of life. TKT believes
there are up to 2,000 patients worldwide afflicted with Hunter syndrome in
jurisdictions where reimbursement may be possible.
About TKT
Transkaryotic Therapies, Inc. is a biopharmaceutical company committed to
developing treatments for rare diseases caused by protein deficiencies with a
core focus on lysosomal storage diseases. The company markets one product,
Replagal(TM), an enzyme replacement therapy for Fabry disease in Europe and
other countries and is developing treatments for Hunter syndrome and Gaucher
disease. TKT's research pipeline focuses on rare diseases where a significant
unmet medical need exists. TKT was founded in 1988 and is headquartered in
Cambridge, Massachusetts, with additional operations in Europe, Canada and
Latin America. Additional information about TKT is available on the company's
website at http://www.tktx.com.
This press release contains forward-looking statements, including
statements regarding the anticipated number of patients to be enrolled in the
AIM study, the anticipated date of completing enrollment in the AIM study, the
anticipated timing of the availability of data from the AIM study, and other
statements containing the words "believes," "anticipates," "plans," "expects,"
"will" and similar expressions. These forward-looking statements are based on
the company's opinion and expectations as of the date hereof and are subject
to risks and uncertainties that could cause actual results to differ
materially. Important factors that could cause results to differ include, but
are not limited to: when the AIM study is completely enrolled; the number of
patients who ultimately meet the entry criteria and elect to enroll in the AIM
study; the timing of availability of data from the AIM study; and other
factors set forth under the caption "Certain Factors That May Affect Future
Results" in the company's Quarterly Report on Form 10-Q for the quarter ended
September 30, 2003 which risk factors are on file with the Securities and
Exchange Commission and are incorporated herein by reference. The company
does not undertake any obligation to update any forward-looking statements.
Replagal(TM) is a trademark of Transkaryotic Therapies, Inc.
Contacts:
Justine E. Koenigsberg
Director, Corporate Communications
(617) 349-0271
Daniella M. Lutz
Corporate Communications Specialist
(617) 349-0205
SOURCE Transkaryotic Therapies, Inc.
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http://www.tktx.com
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CONTACT:
Justine E. Koenigsberg, Director, Corporate
Communications, +1- 617-349-0271, or Daniella M. Lutz, Corporate
Communications Specialist, +1- 617-349-0205 both of Transkaryotic
Therapies, Inc.
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