TKT Reports Encouraging Results in First Human Investigation Of Hemophilia A Gene Therapy
- Data Presented at American Society of Hematology Meeting -
SAN FRANCISCO, Dec 4, 2000 /PRNewswire/ -- Transkaryotic Therapies, Inc. (Nasdaq: TKTX chart, msgs) reported today Phase I results of the first clinical trial evaluating Factor VIII gene therapy in patients with hemophilia A. Data from six patients participating in the study demonstrated that non-viral delivery of Factor VIII using patients' genetically modified cells is safe and well-tolerated. Furthermore, four of the first six patients showed clinical benefit with two patients having no spontaneous bleeds for close to a year following treatment. The results were presented by Dr. David A. Roth from Beth Israel Deaconess Medical Center and Harvard Medical School at the 42nd Annual Meeting of the American Society of Hematology.
"We have seen encouraging biochemical and clinical results in patients, which suggests this approach may become a practical option for the treatment of hemophilia. These results are exciting since we were cautious in the number of cells returned to the patient initially and in the amount of Factor VIII the cells were producing, yet several experienced an improvement in their hemophilia," said David A. Roth, M.D., Director, Hemophilia Clinical Research, Beth Israel Deaconess Medical Center and Assistant Professor of Medicine, Harvard Medical School. "Since the initiation of this program, we have taken a conservative approach with this new technology, and accordingly, I am pleased to note that the therapy not only shows clinical activity but also has been very well-tolerated."
The Phase I open-label study was initiated in December 1998 at Beth Israel Deaconess Medical Center in Boston, Massachusetts to assess the safety of Factor VIII gene therapy in twelve patients with hemophilia A, utilizing Transkaryotic Therapy(TM). Following implantation of 100 to 400 million genetically-modified cells, four of six patients demonstrated decreased bleeding frequency and/or Factor VIII usage. In addition, two patients, while still having occasional trauma-related bleeds, have not experienced spontaneous bleeds for close to one year for the first time in their lives, while occasional injury-related bleeds may have occurred. There were no serious adverse events related to the study material or the implantation procedure. Only minor anticipated adverse experiences related to the implantation procedure were observed.
"We are very hopeful that our non-viral approach to gene therapy will be appropriate for long term delivery of Factor VIII in patients with hemophilia A," stated Richard F Selden, M.D., Ph.D., President and Chief Executive Officer of TKT. "We are very excited by the clinical activity we have seen this early on in the program, but it is important to emphasize to the hemophilia community that we still have a great deal of work ahead to develop a safe and effective treatment."
About Transkaryotic Therapy
In Transkaryotic Therapy, the Company's non-viral gene therapy system, a small sample of a patient's cells are removed in an outpatient procedure and sent to the Company's manufacturing facility where the cells are genetically- engineered to produce the desired protein for extended periods of time. After the cells and the protein have been tested to ensure both safety and functionality, an appropriate number of genetically-engineered cells are returned to the physician and injected back into the patient.
TKT believes Transkaryotic Therapy is well suited to allow safe and long-term delivery of therapeutic proteins for the treatment of chronic protein deficiency states, including anemia, hemophilia, hypercholesterolemia, and diabetes. This technology is designed to take advantage of a patients' ability to synthesize therapeutic proteins for extended periods, potentially providing improved therapeutic outcome, a reduction of side effects due to overdosing or underdosing of conventional proteins, improved patient compliance, and significant reductions in cost due to less frequent administration.
About Hemophilia A
Hemophilia A is an X-linked, hereditary bleeding disorder caused by the absence or defect of a blood clotting protein Factor VIII. As a result, when a person with hemophilia A has a bleeding episode, the bleeding may be prolonged due to the body's inability to form blood clots. Patients affected with hemophilia A experience acute, debilitating, and often life threatening bleeding episodes and may suffer from spontaneous internal, joint, or soft tissue bleeding episodes due to Factor VIII deficiency. It is estimated that approximately 50,000 individuals worldwide are affected with hemophilia A.
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," "intends," and similar expressions are intended to identify forward-looking statements. Important factors that could cause actual results to differ materially from the expectations described in these forward-looking statements are set forth under the caption "Certain Factors That May Affect Future Results" in the Company's Annual Report on Form 10-K for the year ended December 31, 1999 and the Company's Quarterly Report on Form 10-Q for the quarter ended September 30, 2000 which are on file with the Securities and Exchange Commission and incorporated herein by reference. These important factors include risks as to whether TKT's products will advance in the clinical trials process, the timing of such clinical trials, whether the clinical trial results will warrant continued product development, and whether the Company's products will receive approval from the U.S. Food and Drug Administration or equivalent regulatory agencies, and, if such products receive approval, whether they will be successfully marketed; the results of any patent litigation in which the Company is involved or may become involved; competition; and the Company's dependence on collaborators.
Gene-Activated(R) is a registered trademark, Niche Protein(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
Manager, Corporate Communications
(617) 349-0271
Additional Contact:
Bill Schaller, Communications,
Beth Israel Deaconess Medical Center
(617) 632-8062
Source: Transkaryotic Therapies, Inc.
Contact:
Justine E. Koenigsberg, Manager, Corporate Communications of
Transkaryotic Therapies, Inc., 617-349-0271; or Bill Schaller, Communications
of Beth Israel Deaconess Medical Center, 617-632-8062
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