>>VACAVILLE, Calif.--(BUSINESS WIRE)--Jan. 20, 2004--Large Scale Biology Corporation (Nasdaq:LSBC - News) today announced that its plant-produced human therapeutic enzyme, alpha-galactosidase A, showed positive results in preclinical studies using an animal model of Fabry disease. The results were published this week by researchers at the National Institutes of Health's National Institute of Neurological Disorders and Stroke (NIH/NINDS) and LSBC scientists. The studies demonstrated potential for using LSBC's plant-manufactured alpha-galactosidase in enzyme replacement therapy for Fabry disease and suggest further development of the product towards evaluation of its safety and efficacy in humans.
Fabry disease is an X-linked lysosomal storage disorder resulting in the accumulation of lipids in multiple organs, including the heart, liver and kidney. This life-long affliction impacts male patients in particular, with high morbidity and mortality by the time they reach middle age. The published manuscript, entitled "Preclinical Studies with Plant-Produced alpha-Galactosidase A in Fabry Mice Show Potential for Replacement Therapy," authored by M.P. Gelderman et al, and published in PreClinica, Jan/Feb 2004, profiles preclinical research on the characteristics of a plant-made alpha galactosidase in the mouse model for Fabry disease pioneered by Dr. Roscoe Brady, M.D., Chief, Developmental and Metabolic Neurology Branch at NIH/NINDS. The collaborative effort, conducted under a Cooperative Research and Development Agreement (CRADA) between LSBC and Dr. Brady's laboratory, demonstrated that a potentially clinically relevant enzyme can be made in plants. The study showed that significant reductions of excess lipids (GB3), characteristic of Fabry disease, can be achieved in target organs. Tissue distribution studies showed proper targeting of the enzyme to affected organs and no apparent toxicity or tissue damage in the animal model. Also, no neutralizing antibodies were observed in extended infusion studies, suggesting that LSBC's therapeutic enzyme does not elicit undesirable immune responses in the animal model.
"The highly productive CRADA between Dr. Brady's laboratory and LSBC has yielded excellent results in our alpha-galactosidase program," stated Kevin Ryan, LSBC's President and CEO. "The results of this collaborative research have encouraged further development of our alpha-galactosidase product as a near-term clinical target. Our ability to manufacture large quantities of this enzyme cost-effectively provides the basis for developing an improved product to satisfy unmet needs, such as the prophylactic treatment of Fabry patients," said Mr. Ryan. "Concern in the Fabry community over continued unavailability of Fabry therapeutics for the earlier treatment of patients, including women and children, will guide LSBC's clinical plans," Mr. Ryan added.
In 2003, the US Food and Drug Administration granted LSBC's alpha-galactosidase enzyme Orphan Drug designation. This year, LSBC plans to initiate GLP safety studies as a step toward filing an IND and initiating clinical trials. <<
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