For Immediate Release -- December 9, 1996
Media Contact: Cheryl Greenhouse 617-252-7785
Genzyme Transgenics Begins U.S. Phase II Trial with ATIII
Framingham, Mass. -- Genzyme Transgenics Corp. announced today that it has begun a phase II clinical trial in the United States with its lead product, the transgenic form of human antithrombin III (ATIII), a plasma protein that helps prevent harmful blood clotting in many serious medical conditions.
It is the first clinical trial in which patients will be treated with a product made in the milk of a transgenic animal.
The product is being developed jointly with Genzyme Corp.'s General Division (Nasdaq:GENZ), which is providing funding and clinical trial support for transgenic ATIII in exchange for worldwide co-marketing rights, except for Asia.
The initiation of the trial follows the U.S. Food and Drug Administration's acceptance of the company's Investigational New Drug application, which was filed in October.
Transgenic proteins are produced by inserting human DNA into animals' cells so that the targeted protein, or drug, is secreted in the milk of female offspring. Genzyme Transgenics produces ATIII in the milk of transgenic goats at its 168-acre commercial production facility in central Massachusetts. Three goats at the facility are milked three times a day to produce enough of the protein for the clinical trial. Each liter of milk contains two grams of ATIII which is more than is required at the highest dose tested in the trial.
In the phase II trial, which is being conducted at Emory University, Atlanta, transgenic ATIII will be administered to 27 patients undergoing coronary artery bypass grafting (CABG). The trial is a dose escalation study to test the safety of transgenic ATIII in this patient population.
During this surgery, plasma levels of ATIII drop below normal levels. This may impair the anticoagulant response to heparin -- a drug routinely administered during this type of surgery to prevent clotting while the patient is on cardiopulmonary bypass. Better anticoagulation while the patient is on the bypass pump may preserve critical blood components necessary for blood clotting once the surgical procedure is completed.
Increasing the patient's ATIII levels should increase the anticoagulant effect of heparin without interfering with the blood's ability to clot naturally after surgery and may thereby reduce the need for blood product transfusions that could complicate CABG surgery. Genzyme Transgenics expects to complete the trial in the first half of 1997.
The safety of transgenic ATIII was previously tested in a phase I study conducted in the United Kingdom. In that trial, 20 healthy male volunteers received an intravenous infusion of the protein. A review of all safety parameters of that study -- including tests of blood, urine, and vital signs -- showed no clinically significant changes in any group and no differences between subjects who received ATIII and those who received the placebo.
Once the safety of the product is confirmed in CABG patients and an appropriate dosing regimen has been determined, a phase III trial will be initiated to test the efficacy of transgenic ATIII in reducing bleeding and the subsequent need for blood product transfusions in CABG patients.
Plasma-derived ATIII is currently on the market for a number of acquired ATIII deficiency indications in Europe and Japan. Examples of conditions where an acquired ATIII deficiency can occur include liver diseases, disseminated intravascular coagulation, septicemia, shock, burns, multiple trauma, bone marrow and other organ transplantation, hip and knee replacement, and other surgical procedures.
The transgenic form of the protein is being developed to expand the available indications. It ultimately could replace the plasma version. Genzyme Transgenics believes the transgenic form of the drug will be safer and more cost-effective than the current plasma-derived drug.
In addition to treating both acquired and hereditary ATIII deficiencies, the company believes new uses may increase the potential market for the product well beyond the current market of more than $200 million worldwide.
Genzyme Transgenics is engaged in the application of transgenic technology to enable the development and production of recombinant proteins and monoclonal antibodies for medical uses. The company's contract research organization businesses provide non-clinical development and testing services to pharmaceutical, biotechnology, medical device, and other companies. Genzyme Corp.'s General Division (Nasdaq:GENZ) owns approximately 42 percent of the outstanding stock of Genzyme Transgenics.
This news release contains forward-looking information, including statements about the product's cost-effectiveness and the potential market size. Actual results may differ materially from these projections depending on the accuracy of the company's market research and its information about potential new indications.
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