Transkaryotic Therapies (NASDAQ:TKTX)
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TKT Reports Positive Top-Line Results of Hunter Syndrome Pivotal
Trial
Primary Endpoint Achieves Statistical Significance
CAMBRIDGE, Mass., June 20 /PRNewswire-FirstCall/ -- Transkaryotic Therapies,
Inc. (NASDAQ:TKTX) today announced positive top-line results from the company's
pivotal Phase III clinical trial evaluating its investigational human enzyme
replacement therapy, iduronate-2-sulfatase (I2S), for the treatment of patients
with Hunter syndrome. Hunter syndrome, also known as MPS II, is a rare,
life-threatening genetic disorder with no available treatment. In the trial,
patients who received 0.5 mg/kg of I2S on a weekly basis showed a statistically
significant improvement in the primary efficacy endpoint (p=0.0049) compared to
patients receiving placebo. Based on these results, TKT expects to file for
regulatory approval of I2S in both the United States and Europe in the fourth
quarter of 2005.
The primary efficacy endpoint of the trial, also referred to as the AIM study
("Assessment of I2S in MPS II") was a composite endpoint of two clinical
measures previously used to assess clinical benefit in MPS disorders - forced
vital capacity and six-minute walk test. The mean improvement from baseline to
week 53 in percent predicted forced vital capacity was 3.4% in patients
receiving I2S compared to 0.8% in patients receiving placebo. The mean
increase from baseline to week 53 in the distance walked by patients receiving
I2S was 44 meters as compared to 7 meters in the placebo group.
Joseph Muenzer, M.D., Ph.D., of the University of North Carolina at Chapel
Hill, an internationally recognized leader in the diagnosis and treatment of
MPS disorders and the lead investigator of the AIM study said, "These findings
are very encouraging for the medical and patient communities as we believe
enzyme replacement therapy can bring new hope for patients and families
addressing many of the symptoms associated with Hunter syndrome."
Treatment with I2S was generally well-tolerated by patients in the trial. The
most common adverse events observed were associated with the clinical
manifestations of Hunter syndrome. Of the adverse events considered possibly
related to I2S, infusion related reactions were the most common and were
generally mild. No patient withdrew from the trial due to an adverse event
considered related to I2S.
"We are extremely excited about the outcome of the study. In addition, we are
very thankful to all the patients and their families who participated in this
one year trial. Their commitment to this program was instrumental in
generating the data which we believe will support regulatory approval of I2S,"
said Kip Martha, M.D., Senior Vice President and Chief Medical Officer of TKT.
TKT expects full data will be presented at a medical meeting in the autumn of
2005.
Trial Design
The AIM study was a Phase III double-blind, placebo-controlled clinical trial
conducted at nine sites around the world, including the United States, the
United Kingdom, Germany and Brazil. The primary goal of the study was to
evaluate the safety and efficacy of 0.5 mg/kg of I2S administered weekly
compared to placebo. Additionally, the trial evaluated 0.5 mg/kg of I2S every
other week compared to placebo. Ninety-six patients with Hunter syndrome were
randomized to one of three groups with each patient receiving a total of 52
infusions of either I2S, I2S alternating weekly with placebo, or placebo. Of
the 96 who enrolled, 94 completed the study and they all elected to participate
in the open-label extension study of I2S at a dose of 0.5 mg/kg weekly.
Conference Call/Webcast
TKT will host a conference call and live webcast today, June 20, 2005, at 9:00
a.m. to discuss the AIM study. To participate by telephone, dial (913)
981-4905. A live audio webcast can be accessed on the TKT website at
http://www.tktx.com/ within the Investor Information section. A replay of the
call will be available for two weeks by dialing (719) 457-0820 and using the
access code: 3246105. A replay of the webcast will be archived on the TKT
website under Events in the Investor Information section.
About I2S and Hunter Syndrome
I2S is a human iduronate-2-sulfatase produced by genetic engineering technology
intended for long-term treatment of Hunter syndrome. TKT's I2S replaces an
enzyme that is deficient in patients with Hunter syndrome, and therefore could
potentially either stop or ameliorate the clinical manifestations of the
disease. TKT's I2S product has been designated an orphan drug in both the
United States and the European Union. There is currently no effective therapy
for Hunter syndrome.
Hunter syndrome is a hereditary disorder 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, decreased endurance, obstructive and
restrictive airway disease, sleep apnea, joint stiffness, and, in some cases,
central nervous system involvement. If central nervous system involvement
exists, the life expectancy for patients with Hunter syndrome is typically 10-
15 years of age, however, some patients can survive into the fifth or sixth
decade of life. TKT believes there are approximately 2,000 patients worldwide
afflicted with Hunter syndrome in countries where reimbursement may be
possible.
Additional information about Hunter syndrome is available online at
http://www.hunterpatients.com/.
About TKT
Transkaryotic Therapies, Inc. is a biopharmaceutical company primarily focused
on researching, developing and commercializing treatments for rare diseases
caused by protein deficiencies. Within this focus, the company markets
Replagal(TM), an enzyme replacement therapy for Fabry disease, and is
developing treatments for Hunter syndrome and Gaucher disease. In addition to
its focus on rare diseases, TKT intends to commercialize Dynepo(TM), its Gene-
Activated(R) erythropoietin product for anemia related to kidney disease, in
the European Union. TKT was founded in 1988 and is headquartered in Cambridge,
Massachusetts, with additional operations in Europe, Canada and South America.
Additional information about TKT is available on the company's website at
http://www.tktx.com/.
Forward-Looking Statements
This press release contains forward-looking statements including statements
regarding TKT's development of I2S, 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 TKT will be able to complete and file applications for the marketing
approval of I2S in the timeframes it anticipates; whether the FDA, the EMEA and
equivalent regulatory authorities will view the data related to I2S in the same
manner as TKT; whether such regulatory agencies will ask for additional
information about I2S, including the manufacturing processes for I2S; whether
such regulatory agencies will require additional clinical testing of I2S prior
to approving I2S for commercial sale; whether such regulatory agencies will
grant marketing approval for I2S on a timeline consistent with TKT's
expectations, or at all; whether I2S will achieve the commercial success
anticipated by TKT; whether the results of future clinical trials will be
consistent with the results of earlier clinical trials of I2S and warrant
further clinical trials or submission of applications for regulatory approval
for such products to the FDA, the EMEA and equivalent regulatory authorities;
whether TKT will be able to complete the manufacturing development necessary to
satisfy regulatory requirements on a timeline consistent with TKT's
expectations or at all, including with respect to repairing damage done to
TKT's Alewife manufacturing facility where I2S is currently manufactured, and
to manufacture sufficient quantities of TKT's products to satisfy both clinical
trial requirements and commercial demand, or to manufacture material at all, if
approved; the availability and extent of coverage from third party payors and
the timing and receipt of reimbursement approvals for I2S; whether competing
products will reduce any market opportunity that may exist; 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 ending March 31,
2005, which is on file with the Securities and Exchange Commission and which
factors 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.
Gene-Activated(R) is a registered trademark and Replagal(TM) is a trademark of
Transkaryotic Therapies, Inc. Dynepo(TM) is a trademark of Sanofi-Aventis SA.
For More Information Contact:
Justine E. Koenigsberg
Senior Director, Corporate Communications
(617) 349-0271
Daniella M. Lutz
Manager, Corporate Communications
(617) 349-0205
http://www.newscom.com/cgi-bin/prnh/19990913/TKTLOGO
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DATASOURCE: Transkaryotic Therapies, Inc.
CONTACT: Justine E. Koenigsberg, Senior Director, Corporate
Communications, +1-617-349-0271, Daniella M. Lutz, Manager, Corporate
Communications, +1-617-349-0205
Web site: http://www.tktx.com/
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