TITLE: Genzyme General Clarifies Position in Gaucher's Disease Market
DATE: June 23, 1998
Genzyme General (Nasdaq:GENZ) today clarified the competitive position of its
Ceredase(R) and Cerezyme(R) enzyme-replacement products to treat Gaucher's
disease.
On Friday, a Furman Selz analyst downgraded Genzyme General's stock to a "hold"
from a "buy" on the basis of statements made by Transkaryotic Therapies Inc.
(Nasdaq:TKTX) that it plans to develop an enzyme-replacement product for
Gaucher's disease. Separately, Oxford GlycoSciences (LSE:OGS) in a June 10
press release commented on it's plans to develop a product intended
to treat the same disease.
"We have a very strong position in the treatment of Gaucher's disease," said
Genzyme Corp.'s Chairman and Chief Executive Officer Henri A. Termeer. "We
believe our intellectual property, regulatory position, manufacturing expertise,
and close relationships with Gaucher's patients and the physicians who
successfully treat them around the world will allow us to protect and develop
this position for many years to come. In fact, we are working aggressively to
expand this position to include related genetic disorders and gene therapy
approaches to treat these diseases.
"We also believe that public comments and resulting premature speculation
regarding potentially competitive products understate the risks, costs, and
timelines to develop, gain approval, and market a safe and effective product to
treat Gaucher's disease."
Ceredase is a modified version of an enzyme called beta-glucocerebrosidase
(GCR) harvested from human placentas. It has been marketed by Genzyme since
1991.
In 1994, Genzyme introduced Cerezyme, a therapeutically equivalent version of
the enzyme made through recombinant DNA technology. Since the company can
produce Cerezyme in virtually unlimited quantities, Genzyme is well along in
converting patients from Ceredase to Cerezyme throughout the world.
Barriers to Entry
Cerezyme has orphan drug status, providing it with market exclusivity in the
United States until May 2001. It also has patents protecting its manufacturing
method (until 2011) and its composition (until 2013).
In addition to orphan drug status and intellectual property, several other
characteristics of Cerezyme will make entry into this market complex,
time-consuming, and expensive.
For example, the choice of cells in which to produce the product can present
safety and regulatory challenges. Genzyme produces Cerezyme in Chinese
hamster ovary (CHO) cells because these cells have been in widespread use for
the production of recombinant products. This cell line has well-documented
production and safety characteristics and is known not to harbor infectious
agents. One of Genzyme's patents covers the use of CHO cells for the
production of Cerezyme.
GCR is not normally secreted from cells. It is produced, and has its
therapeutic effect, in immune system cells called macrophages. This fact has
two important consequences:
* The enzyme, normally locked within the cells, is difficult and expensive to
harvest and purify in the quantities needed to support the needs of the patient
population. Genzyme applied a considerable amount of expertise and capital to
achieve the large-scale, high-yield manufacturing system for Cerezyme.
* The natural form of the enzyme has no therapeutic effect when injected into
patients because it does not get into the macrophage cells. To develop
Cerezyme, Genzyme scientists created a modified form of the enzyme that enters
the macrophages, where it can reverse the disease process. This remodeled
enzyme is protected by a composition patent.
Oxford GlycoSciences' product functions by a different mechanism from Cerezyme. Genzyme's product is an enzyme that selectively breaks down excessive amounts of a lipid called glucocerebroside. Oxford GlycoSciences' product is intended to inhibit the body's production of glucocerebroside, but it also inhibits the synthesis of other lipids in the body, possibly resulting in toxic side-effects. The product reportedly entered a phase I trial in December 1997, and therefore its long-term safety and effectiveness has not been demonstrated.
Expanding the Product Offerings
Gaucher's disease is one of a family of some 30 genetic diseases known as lysosomal storage disorders. Genzyme is actively pursuing programs to develop treatments for several other such disorders.
"We are committed to making further improvements in the treatment of Gaucher's disease and to developing treatments for several other lysosomal storage disorders," Termeer said.
On June 2, Genzyme General and privately held BioMarin Pharmaceutical Inc. announced their intention to form a joint venture to develop and commercialize BioMarin's lead product, alpha-L-iduronidase, a recombinant enzyme designed to treat mucopolysaccharidosis I (MPS I). The best known and most severe form of MPS I, Hurler's syndrome, is a crippling and fatal disease that affects young children.
Genzyme General has a proprietary program to develop an enzyme called alpha-galactosidase for the treatment of Fabry's disease, another serious lysosomal storage disorder. Genzyme has an exclusive worldwide license to a broad patent covering the over-expression of alpha-galactosidase. The product has recently entered a phase I/II clinical trial.
Genzyme is also developing gene therapies for lysosomal storage disorders. If successful, gene therapies are expected to supplement or supercede enzyme-replacement therapies. The company is sponsoring a phase I trial of a gene therapy for Gaucher's disease at the University of Pittsburgh.
Genzyme General develops and markets therapeutic and surgical products and diagnostic products and services. A division of the biotechnology company Genzyme Corp., Genzyme General has its own common stock intended to reflect its value and track its performance.
This news release contains forward-looking statements about Genzyme General's competitive position in the Gaucher's disease market and the potential use of gene therapies. Actual results may differ materially, depending on the safety, efficacy, cost-effectiveness, and market acceptance of competitive products; the timing and content of decisions made by regulatory authorities; the ability of Genzyme to maintain a competitive position through patents, trade secrets, know-how, and technological innovation; and the results of clinical trials of gene therapies. There can be no assurance that the proposed joint venture with BioMarin will be formed.
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Please call Genzyme's Corporate Communications department at
1-617-252-7570 for additional information.
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