Transkaryotic Therapies (NASDAQ:TKTX)
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TKT Achieves GA-GCB Clinical Milestone
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
http://www.newscom.com/cgi-bin/prnh/19990913/TKTLOGO
http://photoarchive.ap.org/
DATASOURCE: Transkaryotic Therapies, Inc.
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.
Web site: http://www.tktx.com/
Company News On-Call: http://www.prnewswire.com/comp/120657.html