TKTX Transkaryotic Therapies (MM)

TKT Completes Enrollment in Clinical Trial for Gaucher Disease CAMBRIDGE, Mass., July 26 /PRNewswire-FirstCall/ -- Transkaryotic Therapies, Inc. (NASDAQ:TKTX) announced today that it has completed enrollment of an open-label Phase I/II study to evaluate the safety and clinical activity of GA-GCB, its investigational enzyme replacement therapy for the treatment of Gaucher disease. The study enrolled twelve patients with Type I Gaucher disease from several countries. TKT expects to release top- line data in the second half of 2005. The lead investigator of the study, Professor Ari Zimran of Shaare Zedek Medical Center in Jerusalem, Israel stated, "I am honored and excited to lead the first Gaucher study evaluating an alternative enzyme replacement therapy for Gaucher patients. Since this therapeutic protein is produced in human cells, I believe it may have important effects in treating the disease." About Gaucher Disease Gaucher disease is the most common lysosomal storage disease and is caused by an inherited deficiency of the enzyme glucocerbrosidase. This deficiency results in the accumulation of the substrate glucocerbroside in the phagocytic cells of the liver, spleen and bone marrow. The symptoms of Gaucher disease range in severity and may include liver and spleen enlargement, bleeding tendency (due to low platelet counts), fatigue (due to anemia), bone pains and fractures. Currently, there is one enzyme replacement therapy approved for the treatment of Gaucher disease. 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 Latin 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 development of GA-GCB for Gaucher disease, 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 forward- looking statements, including, whether GA-GCB will be safe and effective as a treatment for Gaucher disease; whether TKT will adequately manufacture adequate supply for, and otherwise complete, clinical trials of GA-GCB; whether future trials of GA-GCB will be conducted; whether future trials of GA-GCB will commence on a timely basis; whether the trial described in this press release will be sufficient for regulatory approval or additional studies will be required; whether the FDA and equivalent authorities will approve GA- GCB on a timely basis, or at all; whether if approved, this product will achieve commercial success; whether competing products will reduce any market opportunity that may exist for GA-GCB; whether TKT will be successful in partnering GA-GCB; and other factors set forth under the caption "Risk Factors" in the company's Current Report on Form 8-K filed April 29, 2004, which is on file with the Securities and Exchange Commission and which risk 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 Aventis Pharmaceuticals, Inc. 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, or Daniella M. Lutz, Corporate Communications Manager, +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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