Novazyme Pharmaceuticals Receives Orphan Drug Designation For Mucopolysaccharidosis (MPS I) Therapy

- Therapy for MPS I Is the Company's Second of Two Drugs Expected to Enter

Clinicals in 2001 -



Apr 17, 2001, 01:00 ET from Novazyme Pharmaceuticals, Inc.

    OKLAHOMA CITY, April 17 /PRNewswire Interactive News Release/ -- Novazyme
 Pharmaceuticals, Inc. today announced that the Company was granted orphan drug
 status for its proprietary treatment for Mucopolysaccharidosis I (MPS I).
 This notification from the FDA represents the second orphan designation for
 Novazyme's technology platforms with which the Company is addressing the unmet
 needs of lysosomal storage disease (LSD) patients.
     LSDs are a family of over 50 rare, genetic diseases that are often fatal
 in childhood. These include MPS I and Pompe Disease, a type of muscular
 dystrophy.  Formal notification came from the Office of Orphan Products
 Development at the Food and Drug Administration. Utilizing Novazyme's
 proprietary technologies, the Company has developed recombinant human
 iduronidase (IDUA) that is nearly identical to the enzyme present in healthy
 persons.  The enzyme is produced in a complex process that adds phosphate and
 modifies sugar molecules attached to the enzyme. Novazyme's process eliminates
 complex sugar molecules on the enzyme which can interfere with enzyme
 activity.  The modifications enable the enzyme to be effectively targeted to
 the lysosome, the location within the body where it is needed to work.
     MPS I disorders are characterized by the body's inability to break down
 sugar-like molecules called mucopolysaccharides, which accumulate in the cells
 of the body causing progressive damage to various organs.  This accumulation
 causes systemic problems characteristic of the disease including corneal
 clouding, mental retardation, bone and joint disease, cardiac involvement,
 respiratory compromise and reduced lifespan in nearly all cases.
     In its application for orphan status, filed on January 8, 2001, Novazyme
 submitted pre-clinical evidence demonstrating that its highly phosphorylated
 and properly glycosylated   product for MPS I, designated as NZ-1002, provides
 greatly enhanced enzyme uptake into affected cells.  With more efficient
 uptake of replacement enzymes, patients may benefit by a greater response to
 these therapies and by reduced side effects, such as decreased immune
 reactions to the product.  Enhanced enzyme uptake is widely viewed as the key
 to the treatment of all lysosomal storage diseases.
     "We are extremely pleased with this validation of our technology and the
 efficiency of which it was granted from the FDA," stated John F. Crowley,
 President and Chief Executive Officer of Novazyme Pharmaceuticals. "MPS I is a
 severely debilitating disease and we look forward to rapidly moving NZ-1002
 into human clinical trials by the end of 2001."
     Orphan drug designation qualifies Novazyme to receive certain benefits
 such as tax credits and marketing exclusivity from the Government in exchange
 for developing NZ-1002.  The FDA may grant orphan drug designation to drugs
 intended to treat a rare disease or condition.  If approved, a drug with
 orphan designation generally receives seven years of market exclusivity.
     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).
 
     CONTACT:
 
     Novazyme Pharmaceuticals, Inc.
     John F. Crowley
     President & Chief Executive Officer
     609-683-4400
 
     E-mail:  jcrowley@novazyme.com
 
     Noonan/Russo
     Elinor Kline
     Account Coordinator
     212-696-4455 ext. 254
     E-mail:  e.kline@noonanrusso.com
 
     Web site: http://www.novazyme.com
 
                     MAKE YOUR OPINION COUNT -  Click Here
                http://tbutton.prnewswire.com/prn/11690X56957677
 
 

SOURCE Novazyme Pharmaceuticals, Inc.
    OKLAHOMA CITY, April 17 /PRNewswire Interactive News Release/ -- Novazyme
 Pharmaceuticals, Inc. today announced that the Company was granted orphan drug
 status for its proprietary treatment for Mucopolysaccharidosis I (MPS I).
 This notification from the FDA represents the second orphan designation for
 Novazyme's technology platforms with which the Company is addressing the unmet
 needs of lysosomal storage disease (LSD) patients.
     LSDs are a family of over 50 rare, genetic diseases that are often fatal
 in childhood. These include MPS I and Pompe Disease, a type of muscular
 dystrophy.  Formal notification came from the Office of Orphan Products
 Development at the Food and Drug Administration. Utilizing Novazyme's
 proprietary technologies, the Company has developed recombinant human
 iduronidase (IDUA) that is nearly identical to the enzyme present in healthy
 persons.  The enzyme is produced in a complex process that adds phosphate and
 modifies sugar molecules attached to the enzyme. Novazyme's process eliminates
 complex sugar molecules on the enzyme which can interfere with enzyme
 activity.  The modifications enable the enzyme to be effectively targeted to
 the lysosome, the location within the body where it is needed to work.
     MPS I disorders are characterized by the body's inability to break down
 sugar-like molecules called mucopolysaccharides, which accumulate in the cells
 of the body causing progressive damage to various organs.  This accumulation
 causes systemic problems characteristic of the disease including corneal
 clouding, mental retardation, bone and joint disease, cardiac involvement,
 respiratory compromise and reduced lifespan in nearly all cases.
     In its application for orphan status, filed on January 8, 2001, Novazyme
 submitted pre-clinical evidence demonstrating that its highly phosphorylated
 and properly glycosylated   product for MPS I, designated as NZ-1002, provides
 greatly enhanced enzyme uptake into affected cells.  With more efficient
 uptake of replacement enzymes, patients may benefit by a greater response to
 these therapies and by reduced side effects, such as decreased immune
 reactions to the product.  Enhanced enzyme uptake is widely viewed as the key
 to the treatment of all lysosomal storage diseases.
     "We are extremely pleased with this validation of our technology and the
 efficiency of which it was granted from the FDA," stated John F. Crowley,
 President and Chief Executive Officer of Novazyme Pharmaceuticals. "MPS I is a
 severely debilitating disease and we look forward to rapidly moving NZ-1002
 into human clinical trials by the end of 2001."
     Orphan drug designation qualifies Novazyme to receive certain benefits
 such as tax credits and marketing exclusivity from the Government in exchange
 for developing NZ-1002.  The FDA may grant orphan drug designation to drugs
 intended to treat a rare disease or condition.  If approved, a drug with
 orphan designation generally receives seven years of market exclusivity.
     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).
 
     CONTACT:
 
     Novazyme Pharmaceuticals, Inc.
     John F. Crowley
     President & Chief Executive Officer
     609-683-4400
 
     E-mail:  jcrowley@novazyme.com
 
     Noonan/Russo
     Elinor Kline
     Account Coordinator
     212-696-4455 ext. 254
     E-mail:  e.kline@noonanrusso.com
 
     Web site: http://www.novazyme.com
 
                     MAKE YOUR OPINION COUNT -  Click Here
                http://tbutton.prnewswire.com/prn/11690X56957677
 
 SOURCE  Novazyme Pharmaceuticals, Inc.