Pivotal Trial Underway
CAMBRIDGE, Mass., Sept. 24 /PRNewswire-FirstCall/ -- Transkaryotic
Therapies, Inc. (Nasdaq: TKTX) today announced that one-year experience data
from its Phase I/II clinical trial evaluating iduronate-2-sulfatase (I2S) will
be presented at the American Society of Human Genetics 53rd Annual Meeting in
Los Angeles. I2S is being developed as an enzyme replacement therapy for the
treatment of Hunter syndrome, also referred to as MPS II, a disease for which
there is currently no effective therapy. Results of the study showed that I2S
was generally well-tolerated and demonstrated evidence of clinical activity in
several aspects of the disease. The Phase I/II study results will be
presented by the lead study investigator, Dr. Joseph Muenzer of the University
of North Carolina at Chapel Hill at a poster session from 4:30 to 6:30 pm
Pacific Time on Thursday, November 6, 2003 in Yorty Hall A of the Los Angeles
Convention Center. A copy of the abstract to be presented is available online
at http://www.ashg.org. Based on the positive results of the Phase I/II clinical
trial, TKT has commenced a pivotal clinical trial, referred to as the AIM
study (Assessment of I2S in MPS II), to support regulatory approval of I2S.
The first patient in the AIM study was dosed last week.
"These study results continue to support our efforts to bring enzyme
replacement therapy forward as a treatment for patients with Hunter syndrome,
for whom there is currently no effective therapy," said Joseph Muenzer, M.D.,
lead investigator of the study. "In this study, I2S was found to be well-
tolerated and to improve the overall activity levels of patients, including
important clinical areas such as respiratory function."
The Phase I/II clinical trial was a randomized, double-blind, placebo-
controlled study, involving 12 Hunter syndrome patients. The trial evaluated
three doses (0.15 mg/kg, 0.5 mg/kg, and 1.5 mg/kg) and within each dose group
patients were randomized to receive either I2S or placebo for six months. All
12 patients successfully completed this portion of the study and elected to
enroll in the open-label extension study where all patients were switched to
the 0.5 mg/kg dose administered bi-weekly.
In the extension study, improvements in the 6-minute walk and joint range
of motion occurred and treatment with I2S significantly reduced liver and
spleen volumes. Data also showed significant reductions in urinary GAG
excretion. Any infusion-related reactions that occurred were successfully
managed by slowing the infusion rate and using pre-medications. One patient
receiving 1.5 mg/kg of I2S developed an IgG antibody during the first six
months of treatment and an additional five patients developed IgG antibodies
after 12 months. The development of low-titer antibodies appears not to have
any clinical consequences. Results from the Phase I/II placebo-controlled
study were previously reported at the American Society of Human Genetics
Annual Meeting in October 2002.
About I2S Enzyme Replacement Therapy
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 the European
Union 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
Mucopolysaccharidosis type II, or 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 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. Currently, there is no effective treatment.
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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