Journal of the American Medical Association Publishes Results of TKT's Replagal(TM) Enzyme Replacement Therapy Demonstrating Clinical Benefit in Patients with Fabry Disease
CAMBRIDGE, Mass., Jun 5, 2001 /PRNewswire via COMTEX/ -- Transkaryotic Therapies, Inc. (Nasdaq: TKTX chart, msgs) today announced the publication in The Journal of the American Medical Association (JAMA) of pivotal clinical results evaluating Replagal(TM) (agalsidase alfa) enzyme replacement therapy as a treatment for Fabry disease.
The JAMA article, titled "Enzyme Replacement Therapy in Fabry disease," describes the results of a pivotal Phase II clinical trial in twenty-six patients with Fabry disease. The double-blind, placebo-controlled trial was conducted at the National Institutes of Health (NIH) under the direction of Drs. Raphael Schiffmann and Roscoe Brady to assess multiple clinical outcome measures. Patients received 0.2 mg/kg of Replagal by a 40 minute intravenous infusion every other week for six months. The primary clinical outcome measure was the effect of therapy on neuropathic pain. Additional measures were assessed including renal and cardiac function.
Clinical Outcome Measures
The NIH investigators reported that patients receiving Replagal had a clinically significant reduction in severe, debilitating pain compared to no change for the placebo group. Patients on treatment also experienced a reduction in the number of days off pain medication, with several patients permanently discontinuing pain medications for the duration of the trial. Patients also demonstrated improvement in their quality of life associated with the reduction of pain.
The investigators further reported that patient kidney function stabilized or improved in the group receiving Replagal, whereas patients in the placebo arm experienced a decline in kidney function consistent with the natural history of Fabry disease. This functional change was consistent with observed improvements in renal pathology in patients receiving Replagal. In particular, the fraction of normal glomeruli increased in these patients, while there was a decline in patients on placebo.
The article also indicates that patients experienced an improvement in cardiac function, as well as improvements in the underlying metabolic defects as reflected by a decrease in globtriaosylceramide (Gb3) storage and an increase in body weight. Treatment with Replagal was safe and well-tolerated with the most common adverse events being symptoms typically observed in patients with Fabry disease.
"We have demonstrated that treatment with Replagal had a marked effect in treating Fabry disease. We believe the functional improvements seen in multiple organs and tissues are also having an important impact on improving the quality of life of these patients which, in many cases, was significantly impaired prior to treatment," said Dr. Raphael Schiffmann, lead author of the JAMA publication and Principal Investigator of the study at the National Institutes of Health. "These patients continue to receive treatment, essentially all at home, and the long-term follow-up confirms and extends the results we have seen to date."
Update on Replagal(TM) Development
TKT is committed to a broad clinical program for Replagal. Several studies, designed to provide more insight into the potential role of Replagal as a treatment for Fabry disease, are underway in a variety of patient populations. In addition to the two independent pivotal Phase II studies (which formed the basis of applications for marketing approval in the U.S. and Europe), a study is ongoing to treat symptomatic females affected by Fabry disease. TKT is also evaluating Replagal in patients who have received renal transplants. Additional studies are planned, including a trial to evaluate the effect of Replagal in pediatric patients.
Approximately 150 patients are currently receiving Replagal enzyme replacement therapy in ten countries, and many of these patients have been on therapy for approximately two years. Based on the product's excellent safety profile, essentially all patients in the U.S. are now receiving Replagal(TM) at home, and patients in the U.K. are being transitioned to home therapy. In March 2001, the European Union's Committee of Proprietary Medicinal Products (CPMP) recommended granting marketing authorization of Replagal in Europe for long-term enzyme replacement therapy in patients with a confirmed diagnosis of Fabry Disease.
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.
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: Gene-Activated(r) proteins, Niche Protein(tm) products, and Gene Therapy. The Company'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. TKT'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. 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 "Certain Factors That May Affect Future Results" in the Company's Quarterly Report on Form 10-Q for the quarter ended March 31, 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 Gene- Activated protein, Niche 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:
Justine E. Koenigsberg
Director, Corporate Communications
(617) 349-0271
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Source: Transkaryotic Therapies, Inc.
Contact:
Justine E. Koenigsberg, Director, Corporate Communications of
Transkaryotic Therapies, Inc., 617-349-0271
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