New Data on Lysosomal Storage Disease Programs to be Presented
CAMBRIDGE, Mass., Sept. 20 /PRNewswire-FirstCall/ -- Transkaryotic
Therapies, Inc. (Nasdaq: TKTX) today announced that its research findings
evaluating intrathecal delivery of iduronate-2-sulfatase (I2S) in an animal
model will be presented at the American Society of Human Genetics 54th Annual
Meeting in Toronto. I2S is TKT's investigational enzyme replacement therapy
being developed for the treatment of Hunter syndrome, also referred to as MPS
II, a rare and often fatal disease. These research findings will be presented
by Dr. Justin Lamsa of TKT at a poster session from 5:00 - 7:00 p.m. on
Thursday, October 28, 2004. Recently, TKT made the decision to advance a
program evaluating delivery of I2S directly into the central nervous system
into preclinical development. The company expects to file an Investigational
New Drug Application to commence human clinical trials of its I2S CNS program
in the first half of 2006.
"As part of our ongoing efforts to broadly serve patients suffering from
rare diseases, we are committed to understanding how best to administer
treatments targeting the central nervous system. Our hope is that intrathecal
injection or other delivery methods may be feasible in treating patients
affected with Hunter syndrome who develop central nervous system
complications," said Michael Heartlein, Ph.D., Vice President, Research at
TKT.
A number of additional abstracts relating to TKT's leading three lysosomal
storage disease programs have been accepted for poster presentation at the
ASHG meeting. A list of these abstracts to be presented include:
Hunter Syndrome
-- One year data from a natural history study describing progression in
118 patients with Hunter syndrome will be presented by Dr. Alan Kimura
of TKT on Thursday, October 28, 2004 from 5:00 - 7:00 p.m.
-- The effect of weekly and biweekly dosing of I2S on glycosaminoglycan
reduction in MPS II mouse models will be presented by Mr. Antony Garcia
of TKT on Thursday, October 28, 2004 from 5:00 - 7:00 p.m.
Fabry Disease
-- Pharmacokinetic results of Replagal in patients with end stage renal
disease will be presented by Dr. John Flaherty of TKT on Wednesday,
October 27, 2004 from 4:30 - 6:30 p.m.
-- Professor Michael Beck of The University of Mainz in Germany will
present results from the Fabry Outcome Survey, or FOS, describing the
overall treatment effects of Replagal, on Thursday, October 28, 2004
from 5:00 - 7:00 p.m.
-- Dr. Atul Mehta of Royal Free Hospital in London will present the
relationship between genotype and phenotype in Fabry disease on
Thursday, October 28, 2004 from 5:00 - 7:00 p.m.
-- Pharmacokinetic results of Replagal in pediatric patients will be
presented by Dr. Kenneth Loveday of TKT on Friday, October 29, 2004
from 1:30 - 3:30 p.m.
Gaucher Disease
-- Preliminary safety and pharmacokinetic data of GA-GCB for the treatment
of Gaucher disease will be presented by the lead study investigator,
Professor Ari Zimran of Shaare Zedek Medical Center in Jerusalem,
Israel. The poster presentation is scheduled for Friday, October 29,
2004 from 8:00 - 10:00 a.m.
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.
This press release contains forward-looking statements regarding
scientific research findings, 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 research or clinical trial results will be indicative of results
obtained in other clinical trials, and other factors set forth under the
caption "Certain Factors Which May Affect Future Results" in the company's
Quarterly Report on Form 10-Q filed August 9, 2004, with the Securities and
Exchange Commission and 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
Corporate Communications Manager
(617) 349-0205
SOURCE Transkaryotic Therapies, Inc.
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http://www.tktx.com
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CONTACT:
Justine E. Koenigsberg, Senior Director,
Corporate Communications, +1-617- 349-0271, or Daniella M. Lutz,
Corporate Communications Manager, +1-617-349-0205, both of TKT
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