IPA - International Pompe Association

Novazyme Announces Successful Results with Animal Model

Preliminary Data Suggest Complete Restoration of Skeletal Muscle Function Signals Potential Therapy for Rare Form of Muscular Dystrophy

Date: April 2, 2001

Orlando, Florida, April 2, 2001 - Researchers from the University of Florida, in conjunction with Novazyme Pharmaceuticals, Inc., demonstrated for the first time that a specially engineered, recombinant human enzyme restores normal muscle function in laboratory animals with a rare type of muscular dystrophy called Pompe disease. Pompe disease is caused by a deficiency of the enzyme acid alpha-glucosidase (GAA), which breaks down glycogen.

NZ-1001 or highly phosphorylated recombinant human acid alpha-glucosidase (HP-rhGAA), the modified GAA developed and produced by Novazyme Pharmaceuticals, Inc., successfully treated experimental mice engineered to have Pompe disease. The investigators treated animals with two separate doses, one week apart resulting in restoration of normal levels of GAA to both skeletal and cardiac muscles, the organs most affected by the disease. NZ-1001 also cleared accumulated glycogen from the muscles of these mice. Most importantly, treatment with NZ-1001 restored normal skeletal muscle function in these diseased mice.

Utilizing Novazyme’s proprietary technologies, the company has developed a recombinant human GAA that is nearly identical to the enzyme present in healthy persons. The GAA is produced in a complex process that adds phosphate and modifies sugar molecules attached to the enzyme. The modifications enable the enzyme to be effectively targeted to the lysosome, the location within the muscle cells where it is needed to work.

Barry Byrne, M.D., Ph.D., a pediatric cardiologist in the Departments of Pediatrics, Molecular Genetics and Microbiology and Associate Director of the Powell Gene Therapy Center at the University of Florida, presented the data at the Experimental Biology 2001 conference sponsored by the Federation of American Societies for Experimental Biology (FASEB) - one the world’s largest meetings for basic science researchers.

“These studies indicate that NZ-1001 reaches affected organs, enters the target cells, clears accumulated glycogen and restores function in affected animals.” said Barry Byrne, M.D. Ph.D.

Pompe disease is a rare, fatal, genetic disorder caused by a deficiency of the enzyme, acid alpha-glucosidase (GAA). Without this enzyme, glycogen accumulates in the lysosome of cells and rapidly destroys muscle fibers. Patients with Pompe disease experience severe muscle weakness, difficulty breathing and cardiac insufficiency. Ultimately, patients require wheel chair assistance and mechanical ventilation and succumb to cardiopulmonary failure. There is currently no approved therapy for Pompe disease. President and CEO of Novazyme, John F. Crowley said, “We are delighted with the results reported by Dr. Byrne. The data demonstrates that phosphorylation combined with proper glycosylation is critical to reach target tissues in this cruel disease. If we can replicate these results in Pompe patients, we expect a dramatic improvement in their muscle function that, hopefully, will halt and perhaps even reverse the progression of the disease and lead to measurable improvements in quality of life. Novazyme will move NZ-1001 rapidly forward into human clinical trials later this year.”

Novazyme is a pharmaceutical company developing biotherapies for the treatment of lysosomal storage disorders. These biotherapies are based on Novazyme’s proprietary technologies for the targeted delivery of the missing enzymes critical for the treatment of these diseases. The technologies were developed by William M. Canfield, M.D., Ph.D. in his laboratories at the University of Oklahoma Health Sciences Center. Dr. Canfield founded Novazyme in 1999. Dr. Canfield currently serves as the company’s chairman and chief scientific officer. Novazyme’s headquarters are located in Oklahoma City, Oklahoma. The company’s principal investors include: Catalyst Health & Technology Partners (Boston); HealthCare Ventures (Princeton); the Perseus-Soros Biopharmaceutical Fund (New York); and Neose Technologies (NasdaqNM: NTEC). CONTACT: Novazyme Pharmaceuticals, Inc. John F. Crowley President & Chief Executive Officer (609) 844--7570 E-mail: Noonan/Russo Stephen Gendel Vice President (212) 696-4455 E-mail:

CONTACTS: Novazyme Pharmaceuticals, Inc. John F. Crowley President and Chief Executive Officer (405) 271-8144 or (609) 683-4400 E-mail: Noonan/Russo Stephen Gendel Vice President (212) 696-4455 E-mail: Web site: http://www.novazyme.com