By Matthew Willett

Novazyme Pharmaceuticals Inc. plans to develop its manose-6 phosphate uptake enhancement enzyme therapy program with proceeds from an $8 million private placement with selected investors.

The Oklahoma City-based company said it will use the proceeds to further fund its lead lysosomal storage device disease program, which focuses on the biologic pathway for uptake of manose-6 phosphate.

Investors in the Series A preferred stock financing included Catalyst Health and Technology Partners, of Boston; HealthCare Ventures, of Princeton, N.J.; and Perseus-Soros Biopharmaceutical Fund, of New York.

Company officials say the funding should cover about one year's advancement for their program against Pompe's disease. That should bring the company to human trials in mid-2001, according to founder and Chairman William Canfield and Vice President of Drug Development Anthony McKinney.

"Obviously, we're very pleased," Canfield told BioWorld Today. "It demonstrates the promise and potential of our technology, its recognition by the venture capital folk, and it gives us some running time, about a year."

McKinney said the technology is the only program in development that utilizes a controlled phosphatization method for treatment of the 49 lysosomal storage disease programs. The company's lead candidate, NZ1001, is undergoing preclinical animal testing.

"For 48 of all the 49 lysosomal storage diseases, we can utilize the manose-6 phosphate receptor in order to effectively and efficiently gain access to the lysome mechanism using our technology controlling phospatization," McKinney said.

For those diseases, including Pompe's, Hurler's, Fabry's and Tay-Sachs diseases, McKinney said orphan designation is likely. He added that the company will pursue orphan designation from the FDA for NZ1001.

"To put this into perspective," McKinney explained, "the one lysosomal storage disease that's effectively being treated is Gaucher's disease, by our friends at Genzyme. It's about as frequent as Pompe's disease, and these are clearly orphan indications. There are about 5,000 or 6,000 patients in the U.S., and about an equal number in other Western countries.

"For Pompe's disease, there is currently not a treatment approved for that 5,000-patient population, which varies in age from infancy to adulthood," he said. "Some people who get this disease, for example, infants who are born with it, die within the first year, so clearly this a group of very sick people, but a small one, and the FDA clearly views it as an orphan indication."

Genzyme General, of Cambridge, Mass., is developing a therapy for Pompe's disease, an enzyme replacement technique using a recombinant alpha-glucosidase produced in Chinese hamster ovary cells.

Genzyme purchased development and commercialization rights to the program, dubbed Pompase, for $19.5 million from Synpac Inc., of Durham, N.C.

Novazyme's technology came to it from the University of Oklahoma Health Sciences Center, where company founder Canfield is a faculty member.

"The problem we have is that the science is going so well that we're expanding so rapidly in programs, that it's likely the major difficulty for us will be in our needs for future programs," Canfield said.

He added his company will pursue further funding options as program needs dictate. Novazyme is currently partnered with Neose Technologies Inc., of Horsham, Pa., for development of NZ1001 in a deal that retained marketing rights for Novazyme and called for the formation of a joint venture for development between the companies upon a trigger centered in Novazyme's animal research program.

Novazyme was formed this summer through funding by Neose.

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