LOS ANGELES, Nov. 6 /PRNewswire-FirstCall/ -- Transkaryotic Therapies,
Inc. (Nasdaq: TKTX) today announced that long-term data from its Phase I/II
study with its investigational enzyme replacement therapy, iduronate-2-
sulfatase (I2S) for the treatment of Hunter syndrome, were presented at the
American Society of Human Genetics 53rd Annual Meeting being held in Los
Angeles, California. The results, which were presented by the lead study
investigator, Dr. Joseph Muenzer of the University of North Carolina at Chapel
Hill, showed that treatment with I2S is clinically active in patients with
Hunter syndrome. Hunter syndrome, often referred to as MPS II, is a life-
threatening disorder for which there is currently no effective therapy.
"I have been managing patients with Hunter syndrome for nearly 20 years,
but now, for the first time, we have the potential to alter the normal
progression of the disease," said Dr. Joseph Muenzer. "Some patients
participating in this trial had significant improvements in how they felt.
Moreover, their overall activity level, as measured by joint mobility and
walking, increased. These results give me hope that I2S enzyme replacement
therapy will provide benefits to patients with Hunter syndrome and that, for
the first time, I will be able to treat the physical disease and not just
manage symptoms."
The Phase I/II study was designed to evaluate safety and clinical activity
of I2S in 12 patients with Hunter syndrome in a randomized, double-blind,
placebo-controlled clinical trial. Three dosages of I2S (0.15 mg/kg, 0.5
mg/kg and 1.5 mg/kg) were studied. Within each dose group, patients were
randomized to receive either I2S (3 patients) or placebo (one patient) by IV
infusions biweekly for 6 months. All 12 patients successfully completed the
six-month double-blind study and elected to participate in the open-label
extension study.
Long-term data, which includes six months of I2S treatment in the Phase
I/II placebo-controlled trial as well as an additional six months of treatment
in the extension study, showed that:
-- Urinary glycosaminoglycan excretion was reduced by 45% from baseline
after one year of I2S treatment.
-- Liver and spleen volumes were also reduced by 27% and 26%,
respectively, after one year of therapy from baseline.
-- Respiratory capacity, as measured by forced vital capacity, has
remained stable.
-- Improvements in the 6-minute walk test (12% and 28% improvement in
distance covered in the mid and high dose groups, respectively) were
observed after one year of treatment.
Improvements in joint range of motion were observed after one year of
treatment. Improvements were seen in four to seven joint motions in seven of
nine patients who received treatment since the start of the trial, including
internal shoulder rotation, hip abduction and wrist extension.
Infusion-related reactions occurred in 8 patients and have been
successfully managed by slowing the infusion rate and using pre-medications.
IgG antibodies developed in one patient receiving the high dose during the
first six months. An additional 5 patients in the mid and high dose groups
developed IgG antibodies after 12 months. The development of these low-titer
antibodies appears not to have any clinical consequences.
"These data are extremely encouraging and supported our decision to
advance the product into a pivotal clinical trial," said Michael J. Astrue,
President and Chief Executive Officer of TKT. "We began enrolling patients in
our pivotal trial in September and expect the trial will be fully accrued by
February 2004."
AIM Study
In September 2003, TKT commenced the largest clinical trial to date for a
lysosomal storage disease, referred to as the AIM study, Assessment of I2S in
MPS II. The 90 patient, twelve month study is expected to be completed by the
first quarter 2005. The company expects that enrollment will begin closing at
certain sites as early as December 2003. North American families interested in
learning about this trial should contact Diane Towle, RN of the University of
North Carolina at Chapel Hill at 919-843-5729. Families outside of North
America should contact Leanne Torrie, RN of TKT at 617-613-4499.
Investor Event and Webcast
In conjunction with the American Society of Human Genetics Annual Meeting,
TKT will hold an investor meeting tomorrow, Friday, November 7, 2003 from 8:30
to 9:30 a.m. Pacific Time at the Omni Los Angeles Hotel to discuss long-term
Phase I/II data of I2S. In addition, a live audio webcast can be accessed in
the Investor Information section of TKT's website. A replay of the webcast
will be archived on the TKT website for one week.
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 the severe form, 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 have a normal
lifespan, but most die prematurely due to physical disease progression. TKT
believes there are about 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 TKT, +1-617-349-0271
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