TKT Achieves GA-GCB Clinical Milestone
April 20 2005 - 10:43AM
PR Newswire (US)
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
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