CAMBRIDGE, Mass., March 5 /PRNewswire-FirstCall/-- Transkaryotic
Therapies, Inc. (Nasdaq: TKTX) announced today that its pivotal clinical trial
evaluating iduronate-2-sulfatase (I2S) has completed patient enrollment. I2S
is TKT's investigational enzyme replacement therapy for the treatment of
Hunter syndrome (MPS II), a rare and often fatal disease. The twelve-month
trial, referred to as the AIM study (Assessment of Iduronate-2-sulfatase in
MPS II), enrolled ninety-six patients in six months, exceeding its initial
target of ninety patients. TKT expects top line results from the AIM study in
the second quarter of 2005 and, if positive, the company expects to submit
applications for marketing approval in both the United States and Europe in
the second half of 2005.
"We received tremendous support from families, physicians, and patient
organizations all over the world toward this study and are excited to complete
this important step in the development process of I2S," said Mr. Michael J.
Astrue, President and Chief Executive Officer of TKT. "We look forward to
completing the trial next year in our effort to help patients suffering from
the debilitating effects of Hunter syndrome."
Enrollment in the AIM study began in September 2003 and reached its target
of ninety patients in late February 2004. Enrollment was briefly extended to
allow Japanese patients to participate in the study. TKT believes inclusion of
four patients from Japan in the AIM study could help fulfill some of the
requirements for Japanese approval.
The study 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. Patients
will receive a total of fifty-two 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.
In November 2003, TKT reported long-term data from the Phase I/II clinical
trial evaluating I2S in twelve patients with Hunter syndrome. Results of the
study showed that I2S was generally well-tolerated and demonstrated evidence
of clinical activity in several aspects of the disease, including
stabilization of respiratory capacity, improvement in distance walked,
improvements in several joint motions, reduction in liver and spleen volumes,
and reductions in urinary glycosaminoglycan excretion. Infusion-related
reactions occurred in eight patients. The majority of infusion reactions
were mild and were successfully managed by lengthening the infusion time and
with pre-medications in some patients. All twelve patients continue to
receive I2S in the open-label extension study. A copy of the full poster
presentation is available in the News Bureau section of the TKT website at
http://www.tktx.com.
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
remains 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.
This press release contains forward-looking statements that involve a
number of risks and uncertainties, including statements regarding the clinical
progress and regulatory status of TKT's enzyme replacement therapy for Hunter
syndrome, as well as statements containing the words "believes,"
"anticipates," "plans," "expects," "estimates," "intends," "should," "could,"
"will," "may," and similar expressions. There are a number of important
factors that could cause the company's actual results to differ materially
from those indicated by such forward-looking statements, including whether I2S
will be safe and effective as a treatment for Hunter syndrome, whether TKT
will successfully manufacture adequate supply for, and otherwise complete,
clinical trials of I2S, whether the results of clinical trials, such as the
results referenced in this release, will be indicative of results obtained
during later clinical trials, whether future trials of I2S will be conducted,
whether future trials of I2S will commence on a timely basis, whether the FDA
and equivalent regulatory authorities will approve I2S on a timely basis, or
at all, whether, if approved, this product will achieve commercial success,
whether competing products will reduce any market opportunity that may exist
for I2S, 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 is on file with the Securities and
Exchange Commission and are incorporated herein by reference. While the
company may elect to update forward-looking statements at some point in the
future, the company specifically disclaims any obligation to do so, even if
its expectations change.
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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Company News On-Call:
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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
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