First Patient Dosed in Largest-Ever Clinical Trial for a Lysosomal Storage
Disease
CAMBRIDGE, Mass., Sept. 19 /PRNewswire-FirstCall/ -- Transkaryotic
Therapies, Inc. (Nasdaq: TKTX) today announced that it has dosed the first
patient in the Assessment of Iduronate-2-Sulfatase in MPS II (AIM) Pivotal
Trial. The AIM study is a randomized, double-blind, placebo-controlled,
clinical trial to evaluate the effect of iduronate-2-sulfatase (I2S) over
twelve months in patients with Hunter syndrome, also referred to as MPS II.
Ninety patients with Hunter syndrome will be enrolled at eight sites around
the world. If the results are positive, TKT expects to file applications for
market approval in the United States and Europe in 2005. There is currently
no effective therapy for Hunter syndrome.
"Patients in the initial trial saw significant improvements in overall
activity levels and treatment was generally well-tolerated," said Dr. Joseph
Muenzer of the University of North Carolina at Chapel Hill and lead
investigator of the study. "We are excited to expand the scope of clinical
testing for I2S and are hopeful this study will demonstrate robust clinical
benefit in a much wider patient population."
The primary objective of the study is to determine safety and efficacy of
I2S as a treatment for Hunter syndrome. Ninety patients will be randomized
equally to three treatment groups receiving either weekly or every other week
infusions of I2S at a dose of 0.5 mg/kg or weekly infusions of placebo for
twelve months. Efficacy outcomes will be measured at baseline and at four
month intervals. There will not be an interim analysis performed in the
study. The primary efficacy endpoint will be a single composite variable.
The composite variable will combine 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 will be available shortly online at
http://www.clinicaltrials.gov.
"It is devastating for families to learn that a child has Hunter syndrome.
However, today's news provides reassurance that we are one step closer to
turning the hope of treatment into a reality through the development of I2S,"
said Christine Lavery of the United Kingdom MPS Society. "If successful, I2S
will be the first treatment to clinically address Hunter syndrome and we are
hopeful that all patients will have access to this important therapy."
TKT previously conducted a Phase I/II randomized, double-blind, placebo-
controlled clinical trial to evaluate the safety of I2S and its clinical
activity in twelve patients affected with Hunter syndrome. Three doses were
studied and within each dose group three patients were randomized to receive
I2S and one patient to receive placebo bi-weekly for six months. Results of
the study showed that I2S was generally well-tolerated and demonstrated
evidence of clinical activity in several critical aspects of the disease,
including respiratory function and functional capacity. Data from the six
month Phase I/II study was presented at the American Society of Human Genetics
Annual Meeting in October 2002. Long-term results from the open-label
extension portion of the study were presented earlier this month at the Ninth
International Congress of Inborn Errors of Metabolism meeting in Australia and
additional data from this study will be presented in November 2003 at the
American Society of Human Genetics 53rd Annual Meeting.
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
TKT is a biopharmaceutical company developing therapeutics for the
treatment of rare genetic diseases caused by protein deficiencies. The company
currently markets one product, Replagal(TM) (agalsidase alfa) for the
treatment of Fabry disease in the European Union and certain other countries.
TKT is headquartered in Cambridge, Massachusetts and has a majority-owned
subsidiary in Sweden, TKT Europe-5S AB, which is responsible for European
sales and marketing activities of Replagal. Additional information on TKT is
available on the company's website at http://www.tktx.com.
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 accrue patients and 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 June 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.
CONTACT:
Justine E. Koenigsberg
Director, Corporate Communications
(617) 349-0271
SOURCE Transkaryotic Therapies, Inc.
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CONTACT:
Justine E. Koenigsberg, Director, Corporate
Communications of Transkaryotic Therapies, Inc., +1-617-349-0271
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