GA-GCB, I2S and Dynepo Top-Line Results Expected in the Coming Months
CAMBRIDGE, Mass., April 20 /PRNewswire-FirstCall/ -- Transkaryotic
Therapies, Inc. (Nasdaq: TKTX) today announced that the last patient has
completed treatment in its open-label Phase I/II clinical trial evaluating its
investigational enzyme replacement therapy for Gaucher disease, Gene-
Activated(R) glucocerebrosidase, or GA-GCB. The Phase I/II clinical trial
evaluated the safety of GA-GCB as well as its clinical activity in 12
patients. The company expects to report top-line results of the study in the
second half of 2005. Assuming the results are favorable, the company intends
to commence a pivotal trial in 2006, designed to support an approval of GA-GCB
in 2008.
In March 2005, TKT completed its pivotal clinical trial evaluating its
investigational enzyme replacement therapy Iduronate-2-Sulfatase, I2S, for the
treatment of Hunter syndrome in 96 patients. The company is currently
gathering and verifying the final data and expects to unblind the study and
report top-line results in June 2005. If the results are positive, TKT
intends to file for regulatory approval in both the United States and Europe
during the second half of 2005. From May 23, 2005 until it reports top-line
results TKT will suspend its investor relations activities.
TKT is expecting to receive results of a Phase III study evaluating Dynepo
as a treatment for anemia associated with cancer chemotherapy that was
previously completed by Sanofi-Aventis. Under a September 2004 agreement,
Sanofi-Aventis has agreed to conduct the analysis and provide TKT with the
data after the FDA provides final input on the statistical analysis plan.
TKT previously expected to report top-line results from this study in the
first half of 2005, however the company now expects the results to be
available in June or in the third quarter of 2005.
"We are very pleased that the treatment phase of the GA-GCB Phase I/II
clinical trial and the I2S pivotal study are now completed. Many of the
patients in these trials relocated internationally in order to gain access to
our investigational products; and we are very grateful to these patients and
their families for their many sacrifices," said Michael J. Astrue, President
and Chief Executive Officer of TKT. "We intend to continue executing on our
established clinical and regulatory objectives throughout 2005, which we hope
will allow us to achieve our goal of generating revenue from three commercial
products in 2006."
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 certain products, including I2S, GA-
GCB and Dynepo, 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
any of the company's products will achieve the commercial success anticipated
by the company; the timing of submissions to and decisions by regulatory
authorities regarding clinical trials and marketing and other applications;
whether the FDA, the EMEA and equivalent regulatory authorities will grant
marketing approval for the company's products on a timeline consistent with
TKT's expectations, or at all; whether TKT will be able to complete clinical
trials of its products on a timely basis; whether the results of clinical
trials will be consistent with the results of earlier clinical trials of the
company's products and warrant further clinical trials or submission of
applications for regulatory approval for such products to the FDA and
equivalent regulatory authorities; whether TKT and its third party
manufacturers will be able to complete the manufacturing development necessary
to satisfy regulatory requirements on a timeline consistent with TKT's
expectations or at all 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 the company's products; whether competing products will reduce
any market opportunity that may exist; whether TKT will be able to enter into
a collaboration agreement for Dynepo in Europe and whether such collaboration
will be successful; results of ongoing litigation and the risks of future
litigation; whether competitors will be able to limit access to markets in
which TKT is attempting to sell Dynepo, through legal maneuvering or
otherwise; and other factors set forth under the caption "Certain Factors That
May Affect Future Results" in the company's annual report on Form 10-K for the
year ending December 31, 2004, 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 Communication
(617) 349-0271
Daniella M. Lutz
Manager, Corporate Communications
(617) 349-0205
SOURCE Transkaryotic Therapies, Inc.
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Related links:
http://www.tktx.com
Photo Notes:
NewsCom:
http://www.newscom.com/cgi-bin/prnh/19990913/TKTLOGO
AP Archive: http://photoarchive.ap.org
PRN Photo Desk, photodesk@prnewswire.com
Company News On-Call:
http://www.prnewswire.com/comp/120657.html
CONTACT:
Justine E. Koenigsberg, Senior Director,
Corporate Communication, +1-617-349-0271, or Daniella M. Lutz,
Manager, Corporate Communications, +1-617-349-0205, both of
Transkaryotic Therapies, Inc.
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