TKTX Transkaryotic Therapies (MM)

TKT Begins Clinical Trial for Gaucher Disease CAMBRIDGE, Mass., April 26 /PRNewswire-FirstCall/ -- Transkaryotic Therapies, Inc. today announced that it has initiated a clinical trial to evaluate the safety and clinical activity of GA-GCB, its enzyme replacement therapy for the treatment of Gaucher disease. The clinical trial will involve twelve patients with Type I Gaucher disease, who will receive treatment for nine months. TKT expects the trial will be completed in 2005. "This trial was designed to replicate key results of the study the FDA and other regulatory agencies relied upon in approving the first enzyme replacement therapy for Gaucher disease," said Paul M. Martha, M.D., Vice President, Clinical Affairs at TKT. "We are pleased to take this step toward offering patients with Gaucher disease an alternative enzyme replacement therapy product." GA-GCB is a human glucocerebrosidase product developed using TKT's proprietary gene activation technology and is the first potential competing enzyme replacement therapy for Gaucher disease to be tested in patients. TKT intends to seek a partner for its GA-GCB product. Gaucher disease is caused by deficiency of the enzyme glucocerebrosidase, resulting in accumulation of a toxic substrate, specifically the glycolipid glucocerebroside. The symptoms of Gaucher disease range in severity and may include liver and spleen enlargement, skeletal weakening, and disorders associated with low blood cell counts, such as anemia. 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, outside of the United States. 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 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 and when the company's GA-GCB trial will be completely enrolled; whether the number of patients who ultimately meet the entry criteria will elect to enroll in the trial; whether the company's GA-GCB trial will be successfully completed; 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 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 "Certain Factors Which May Affect Future Results" in the company's Annual Report on Form 10-K for the year ended December 31, 2003, 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. CONTACTS: Justine E. Koenigsberg Daniella M. Lutz Director, Corporate Communications Corporate Communications Specialist (617) 349-0271 (617) 349-0205 http://www.newscom.com/cgi-bin/prnh/19990913/TKTLOGODATASOURCE: Transkaryotic Therapies, Inc. CONTACT: Justine E. Koenigsberg, Director, Corporate Communications, +1-617-349-0271, or Daniella M. Lutz, Corporate Communications Specialist, +1-617-349-0205, both for Transkaryotic Therapies, Inc. Web site: http://www.tktx.com/ Company News On-Call: http://www.prnewswire.com/comp/120657.html

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