By Randall Osborne
Genzyme Corp. said it would invest $14 million in Pharming Group NV, as the companies agreed to form a joint venture — Genzyme's second in two months — aimed at developing alpha glucosidase, a human enzyme, to treat Pompe's disease.
"The joint venture terms are being negotiated," said Bo Piela, spokesman for Cambridge, Mass.-based Genzyme. "We've signed a letter of intent, and we're proceeding."
Pharming, of Leiden, the Netherlands, produces transgenic rabbits, which secrete alpha glucosidase in their milk.
Pompe's disease affects 5,000 to 10,000 people in the Western world and occurs in three forms — infant, juvenile and adult.
"They all lead to death, because the disease causes a buildup of glycogen in the muscles," Piela said. "Respiratory complications are the principal cause of death."
Patients stricken as infants typically show symptoms in the first few months after birth. They often die before age 2. Those afflicted as juveniles, who develop progressive muscle weakness, usually succumb before age 30. The adult form appears between ages 20 and 60.
Genzyme markets Cerezyme and Ceredase, enzyme replacements for Gaucher's disease which, like Pompe's disease, is classified as a lysosomal storage disorder. In 1997, Cerezyme and Ceredase, Genzyme's two lead products, recorded sales of $333 million.
"That's an important point," Piela said. "We have the know-how to treat diseases of this type."
Last month, Genzyme disclosed its plan for a joint venture with BioMarin Pharmaceutical Inc., of Novato, Calif., to develop an enzyme replacement treatment for mucopolysaccharidosis I (MPS I), a fatal childhood genetic disorder of which the best known form is Hurler syndrome. (See BioWorld Today, June 4, 1998, p. 1.)
In May, Genzyme raised $250 million through a private placement to boost its cardiovascular gene therapy program and surgical products business. (See BioWorld Today, May 21, 1998, p. 1.)
The company's stock (NASDAQ:GENZ) closed Wednesday at $27.25, up $1.75. *