Unprecedented Response to Enzyme Therapy for Pompe Disease in Mouse Model

Preliminary Data Suggest Complete Restoration of Skeletal Muscle Function

Signals Potential Therapy for Rare Form of Muscular Dystrophy



Apr 02, 2001, 01:00 ET from Novazyme Pharmaceuticals, Inc.

    ORLANDO, Fla., April 2 /PRNewswire/ -- 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 (Nasdaq:  
 NTEC).
 
     Novazyme Pharmaceuticals, Inc.
       John F. Crowley
       President & Chief Executive Officer
       609-683-4400, jcrowley@novazyme.com
 
 
     Noonan/Russo
       Stephen Gendel
       Vice President
       212-696-4455
       s.gendel@noonanrusso.com
 
 

SOURCE Novazyme Pharmaceuticals, Inc.
    ORLANDO, Fla., April 2 /PRNewswire/ -- 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 (Nasdaq:  
 NTEC).
 
     Novazyme Pharmaceuticals, Inc.
       John F. Crowley
       President & Chief Executive Officer
       609-683-4400, jcrowley@novazyme.com
 
 
     Noonan/Russo
       Stephen Gendel
       Vice President
       212-696-4455
       s.gendel@noonanrusso.com
 
 SOURCE  Novazyme Pharmaceuticals, Inc.