TKT Reports Positive Top-Line Results of Hunter Syndrome Pivotal Trial
June 20 2005 - 9:01AM
PR Newswire (US)
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
http://photoarchive.ap.org/ 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/ Company News On-Call:
http://www.prnewswire.com/comp/120657.html
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