TKTX Transkaryotic Therapies (MM)

TKT's Research Findings on Intrathecal Delivery of I2S Presented at ASHG TORONTO, Oct. 28 /PRNewswire-FirstCall/ -- Transkaryotic Therapies, Inc. (NASDAQ:TKTX) today released research findings at the American Society of Human Genetics 54th Annual Meeting being held in Toronto, Canada evaluating intrathecal delivery of iduronate-2-sulfatase (I2S) in an animal model. The results, presented by Dr. Justin Lamsa of TKT, showed that repeated injections of I2S were well-tolerated and resulted in the accumulation of enzyme in various cells of the central nervous system (CNS). I2S is TKT's investigational enzyme replacement therapy for the treatment of Hunter syndrome, also referred to as MPS II, a rare and often fatal disease. In addition to the I2S CNS program, TKT is conducting a pivotal trial known as the AIM study (Assessment of I2S for MPS II) which is evaluating I2S as a treatment for the non-CNS aspects of Hunter syndrome through repeated intravenous infusion. TKT expects top-line data from the AIM study in the second quarter of 2005. "We are encouraged with these early research findings and intend to advance this program further to better understand optimal dosing and methods to deliver enzyme into the central nervous system. Since many Hunter syndrome patients will develop central nervous system involvement, we are committed to finding a way to safely administer this missing enzyme to the brain of Hunter syndrome patients to complement our product currently under development for non-CNS aspects of Hunter syndrome," said Michael Heartlein, Ph.D., TKT's Vice President, Research. The primary goal of TKT's I2S CNS research initiative was to determine if I2S could be successfully delivered to cells of the central nervous system by way of intrathecal injections. I2S was administered as 1 ml bolus injections via the cisterna magna. Key research findings include: * Intracisternal injection of I2S results in a dose-dependent accumulation of I2S in the brain. In contrast, intravenous injection of I2S does not elevate CNS I2S levels. * I2S is taken up primarily by the meninges after intracisternal injection, but also by perivascular cells, neurons and glial cells. * I2S was detected in lumbar cerebrospinal fluid after intracisternal injection with a peak at four to six hours and an elimination half-life of two to three hours. * I2S injected into the cisterna magna distributes throughout the cerebrospinal fluid and penetrates into the parenchyma of the brain. TKT has advanced a program evaluating delivery of I2S directly into the central nervous system into preclinical development. If data from the preclinical testing are positive, TKT expects to file an Investigational New Drug Application to commence human clinical trials of its I2S CNS program in the first half of 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. Outside 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 the company's 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 findings will be indicative of results obtained in other clinical trials, whether future trials of I2S CNS will be conducted; whether future trials of I2S CNS will commence on a timely basis; 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 http://www.newscom.com/cgi-bin/prnh/19990913/TKTLOGO http://photoarchive.ap.org/ DATASOURCE: Transkaryotic Therapies, Inc. CONTACT: Justine E. Koenigsberg, Senior Director, Corporate Communications, +1-617-349-0271, Daniella M. Lutz, Corporate Communications Manager, +1-617-349-0205 Web site: http://www.tktx.com/ Company News On-Call: http://www.prnewswire.com/comp/120657.html

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