CAMBRIDGE, Mass., April 26 /PRNewswire-FirstCall/ -- Transkaryotic
Therapies, Inc. (Nasdaq: TKTX) today announced that it has initiated a
clinical trial to evaluate the safety and clinical activity of GA-GCB, its
enzyme replacement therapy for the treatment of Gaucher disease. The clinical
trial will involve twelve patients with Type I Gaucher disease, who will
receive treatment for nine months. TKT expects the trial will be completed in
2005.
"This trial was designed to replicate key results of the study the FDA and
other regulatory agencies relied upon in approving the first enzyme
replacement therapy for Gaucher disease," said Paul M. Martha, M.D., Vice
President, Clinical Affairs at TKT. "We are pleased to take this step toward
offering patients with Gaucher disease an alternative enzyme replacement
therapy product."
GA-GCB is a human glucocerebrosidase product developed using TKT's
proprietary gene activation technology and is the first potential competing
enzyme replacement therapy for Gaucher disease to be tested in patients. TKT
intends to seek a partner for its GA-GCB product.
Gaucher disease is caused by deficiency of the enzyme glucocerebrosidase,
resulting in accumulation of a toxic substrate, specifically the glycolipid
glucocerebroside. The symptoms of Gaucher disease range in severity and may
include liver and spleen enlargement, skeletal weakening, and disorders
associated with low blood cell counts, such as anemia.
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. Outside its
focus on rare diseases, TKT intends to commercialize Dynepo(TM), its
Gene-Activated(R) erythropoietin product for anemia related to kidney disease,
outside of the United States. TKT was founded in 1988 and is headquartered in
Cambridge, Massachusetts, with additional operations in Europe, Canada and
Latin America. Additional information about TKT is available on the company's
website at http://www.tktx.com.
This press release contains forward-looking statements regarding GA-GCB
for Gaucher disease, 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 forward-looking statements, including, whether and
when the company's GA-GCB trial will be completely enrolled; whether the
number of patients who ultimately meet the entry criteria will elect to enroll
in the trial; whether the company's GA-GCB trial will be successfully
completed; whether GA-GCB will be safe and effective as a treatment for
Gaucher disease; whether TKT will adequately manufacture adequate supply for,
and otherwise complete, clinical trials of GA-GCB; whether future trials of
GA-GCB will be conducted; whether future trials of GA-GCB will commence on a
timely basis; whether the FDA and equivalent authorities will approve GA-GCB
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 GA-GCB; whether TKT will be successful in
partnering GA-GCB; and other factors set forth under the caption "Certain
Factors Which May Affect Future Results" in the company's Annual Report on
Form 10-K for the year ended December 31, 2003, which is on file with the
Securities and Exchange Commission and which risk 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
Aventis.
CONTACTS:
Justine E. Koenigsberg Daniella M. Lutz
Director, Corporate Communications Corporate Communications Specialist
(617) 349-0271 (617) 349-0205
SOURCE Transkaryotic Therapies, Inc.
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Related links:
http://www.tktx.com
Photo Notes:http://www.newscom.com/cgi-bin/prnh/19990913/TKTLOGO
Company News On-Call:
http://www.prnewswire.com/comp/120657.html
CONTACT:
Justine E. Koenigsberg, Director, Corporate
Communications, +1-617-349-0271, or Daniella M. Lutz, Corporate
Communications Specialist, +1-617-349-0205, both for
Transkaryotic Therapies, Inc.
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