Additional Results of TKT's Replagal(TM) Enzyme Replacement Therapy Published in Circulation
Reversal of Cerebral Blood Flow Abnormalities Demonstrated in Clinical Study
CAMBRIDGE, Mass., Sep 25, 2001 /PRNewswire via COMTEX/ -- Transkaryotic Therapies, Inc. (Nasdaq: TKTX chart, msgs) today announced the publication of clinical data evaluating Replagal(TM) (agalsidase alfa) enzyme replacement therapy as a treatment for Fabry disease. In an article entitled "Regional Cerebral Hyper-Perfusion and Nitric Oxide Pathway Dysregulation in Fabry Disease: Reversal by Enzyme Replacement Therapy," results of the randomized, double-blind, placebo-controlled trial of 26 patients with Fabry disease are discussed. Patients enrolled in the study, conducted at the National Institutes of Health (NIH) under the direction of Drs. Raphael Schiffmann and Roscoe Brady, were administered 0.2 mg/kg of Replagal by a 40 minute intravenous infusion every other week for six months.
Clinical Outcome Measures
The NIH investigators reported that patients receiving Replagal demonstrated a reversal in cerebral blood flow abnormalities. Patients receiving Replagal had a significant improvement in cerebral blood flow compared to patients receiving placebo (p=0.0258). The investigators believe that the reversal of this abnormality could lead to a decrease in the incidence of stroke in patients with Fabry disease, a leading cause of morbidity and mortality.
"We previously reported in The Journal of the American Medical Association (JAMA) that Replagal enzyme replacement therapy had a marked effect in treating Fabry disease," said Dr. Schiffmann. "The results in JAMA discussed improvements in reducing severe, debilitating pain, the stabilization and improvement of kidney function and the improvement of cardiac function. Today, in Circulation, we report on improvement in cerebral blood flow, which we believe could reduce the incidence of stroke. We have previously demonstrated that Replagal enzyme replacement therapy has resulted in functional improvement in several organs and tissues, with an associated impact on improving quality of life in these patients," he added.
"As we continue to evaluate the clinical trial data for Replagal, it is becoming increasingly clear that Replagal does not simply reduce lipid levels," said Thomas J. Schuetz, M.D., Ph.D., Vice President of Clinical Affairs at TKT. "The European Summary of Product Characteristics states that therapy with Replagal reduces pain, improves renal function, and reduces cardiac mass. The results published today further expand the data supporting therapeutic efficacy of Replagal by showing improvement in blood flow to the brain."
Update on Replagal(TM) Development
TKT is committed to a broad clinical program for Replagal. In a pivotal Phase II study conducted at the NIH, twenty-six patients participated in a 24-week, randomized, double-blind, placebo-controlled trial. In a separate pivotal Phase II study conducted at the Royal Free Hospital in the United Kingdom, fifteen patients also participated in a randomized, double-blind, placebo-controlled study. Investigators have reported that data from these pivotal trials suggest that treatment with Replagal is effective in reducing neuropathic pain, reducing cardiac mass and stabilizing or improving kidney function and that treatment with Replagal is safe and well-tolerated. TKT is evaluating Replagal in female patients and patients who have received renal transplants. Additional studies are planned, including a trial to evaluate the effect of Replagal in pediatric patients.
About Replagal(TM) Enzyme Replacement Therapy
Replagal is a human alpha-galactosidase A produced by genetic engineering technology in a human cell line. Patients receive 0.2 mg/kg of Replagal every other week over a 40 minute intravenous infusion. In the United States and Europe, many patients receive Replagal at home rather than in a hospital setting.
Replagal is approved for marketing in the fifteen countries of the European Union and Norway. In Europe, Replagal will be distributed and marketed by TKT Europe-5S AB, a majority-owned subsidiary of TKT headquartered in Sweden. TKT Europe-5S has a presence in all major European countries and, through its country managers, is providing a range of services to patients and physicians. Approximately 200 patients are receiving Replagal worldwide, of which approximately 150 patients are in Europe.
About Fabry Disease
Fabry disease is an inherited rare genetic disorder caused by deficient activity of the lysosomal enzyme alpha-galactosidase A. In patients with Fabry disease, Gb3 accumulates in various organs and tissues of the body due to the deficiency of alpha-galactosidase A. Many cells are damaged by Gb3 including epithelial cells of the kidney, myocardial cells, cells of the autonomic nervous system, endothelial, perithelial, and smooth muscle cells in the large vessels. As a result, the deposits of this material can result in extreme pain, severe kidney damage, cardiovascular disease, and stroke. Due to its rarity and vast array of symptoms, diagnosis is often difficult and affected individuals have a significantly reduced quality of life and a greatly shortened life expectancy. TKT estimates that approximately 5,000 patients worldwide are affected by Fabry disease.
About TKT
Transkaryotic Therapies, Inc. (TKT) is a biopharmaceutical company dedicated to the development and commercialization of products based on its three proprietary development platforms: Niche Protein(TM) products, Gene- Activated(R) proteins, and Gene Therapy. The Company's Niche Protein product platform is based on protein replacement for the treatment of rare genetic diseases, a group of disorders characterized by the absence of certain metabolic enzymes. TKT's gene activation technology is a proprietary approach to the large-scale production of therapeutic proteins, which does not require the cloning of genes and their subsequent insertion into non-human cell lines. The Company's Gene Therapy technology, known as Transkaryotic Therapy(TM), is focused on the commercialization of non-viral, ex vivo gene therapy products for the long-term treatment of chronic protein deficiency states.
This press release contains forward-looking statements that involve a number of risks and uncertainties. For this purpose, any statements contained herein that are not statements of historical fact may be deemed to be forward- looking statements. Without limiting the foregoing, the words, "believes," "anticipates," "plans," "expects," "estimates," "intends," "should," "could," "will," "may," and similar expressions are intended to identify forward- looking statements. 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 set forth under the caption "Risk Factors" in the Company's Quarterly Report on Form 10-Q for the quarter ended June 30, 2001 which is on file with the Securities and Exchange Commission and incorporated herein by reference. These include, without limitation, the following: (1) whether any of the Company's Niche Protein, Gene-Activated protein or gene therapy product candidates will advance in the clinical trial process, (2) whether such clinical trials will proceed in a timely manner, (3) whether the clinical trial results will warrant continued product development, (4) whether the required regulatory filings, such as Investigational New Drug applications and Biologics License Applications, are made in a timely manner, (5) whether the Company's products will receive approval from the U.S. Food and Drug Administration or equivalent foreign regulatory agencies, (6) if such products receive approval, whether they will be successfully distributed and marketed, (7) whether patent litigation in which the Company is involved or may become involved are resolved in a manner adverse to the Company, (8) the effects of competitive products on the Company's proposed products, and (9) the Company's dependence on third parties, including collaborators, manufacturers and distributors.
Gene-Activated(R) is a registered trademark and Niche Protein(TM), Replagal(TM), TKT(TM), and Transkaryotic Therapy(TM) are trademarks of Transkaryotic Therapies, Inc.
Please visit our web site at www.tktx.com for additional information about Transkaryotic Therapies, Inc.
CONTACT:
Daniel E. Geffken
Senior Vice President, Finance
and Chief Financial Officer
(617) 349-0581
Source: Transkaryotic Therapies, Inc.
Contact:
Daniel E. Geffken, Senior Vice President, Finance and Chief
Financial Officer of Transkaryotic Therapies, Inc., +1-617-349-0581
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