BRISBANE, Australia, Sept. 4 /PRNewswire-FirstCall/--
Transkaryotic Therapies, Inc. (Nasdaq: TKTX) today announced that its lead
clinical investigator, Joseph Muenzer, M.D., Ph.D., of the University of North
Carolina at Chapel Hill, presented long-term data from TKT's Phase I/II
clinical trial evaluating iduronate-2-sulfatase (I2S) enzyme replacement
therapy for the treatment of Hunter syndrome, also referred to as MPS II, a
debilitating disease for which there is currently no effective therapy. The
one-year data showed improvements in a variety of clinical measures and
continued to demonstrate a favorable safety profile. These findings were
presented at the IXth International Congress of Inborn Errors of Metabolism
meeting being held this week in Australia. Additional data from this study
will be presented in November 2003 at the American Society of Human Genetics
53rd Annual Meeting.
"The significant clinical improvements observed in these patients treated
with I2S at both six and twelve months indicates that this product provides a
clinical benefit and appears to represent a promising treatment for Hunter
syndrome," said Dr. Joseph Muenzer, Associate Professor of Pediatrics,
Division of Genetics and Metabolism, Department of Pediatrics at the
University of North Carolina at Chapel Hill.
After successfully completing the six-month randomized, double-blind,
placebo-controlled study evaluating three doses of I2S (0.15 mg/kg, 0.5 mg/kg,
and 1.5 mg/kg) bi-weekly for 24 weeks, all 12 patients elected to enroll in
the open-label extension study. The six-month data showed a significant
reduction in urinary glycosaminglycan (GAG) excretion, significant reductions
in liver and spleen volumes, as well as reductions in left ventricular mass
and improvements in forced vital capacity. Patients receiving placebo that
crossed over to I2S also showed a similar treatment response. Patients showed
a greater improvement in their joint range of motion and with the six-minute
walk test at one year. Any significant infusion-related reactions that
occurred were successfully managed by slowing the infusion rate and using pre-
medications. Data from the six-month Phase I/II study were previously
presented at the American Society of Human Genetics Annual Meeting in October
2002.
TKT is preparing to commence a pivotal study of I2S in September 2003.
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 to
three treatment groups to receive either weekly or every other week infusions
of I2S at a dose of 0.5 mg/kg or weekly infusions of placebo. The primary
endpoint will be a single composite variable designed to evaluate the efficacy
of I2S therapy across multiple outcomes. 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.
"The final preparations for the pivotal trial in patients with Hunter
syndrome are being made and we are on track to begin enrolling patients in the
trial this month," said Michael J. Astrue, President and Chief Executive
Officer of TKT.
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 for breaking down
glycosaminglycans (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
their fifth or sixth decade of life. TKT believes there are approximately
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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