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BioMarin Announces Initiation of Clinical Assessment Program for Morquio A Syndrome
Date:11/3/2008

NOVATO, Calif, Nov. 3 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today the initiation of the Morquio Clinical Assessment Program (MorCAP) for patients with the lysosomal storage disease Mucopolysaccharidosis Type IVA (MPS IVA), or Morquio A Syndrome. MorCAP is designed to augment available data on the disease by measuring endurance and respiratory function and other parameters in affected patients. BioMarin expects to follow the MorCAP program with a Phase 1b clinical trial of enzyme replacement therapy beginning in the first quarter of 2009. The primary objectives of the Phase 1b study will be to evaluate safety and to establish the optimal dose of enzyme based on pharmacokinetic and pharmacodynamic parameters.

"After successfully advancing two enzyme replacement therapies in approximately five years each from IND filing to FDA approval, we plan to leverage our clinical, manufacturing and regulatory expertise to develop a treatment for Morquio A syndrome," said Emil Kakkis, M.D., Ph.D., Chief Medical Officer of BioMarin. "Preliminary studies are promising and indicate that our drug candidate binds naturally to bone matrix and can adequately reach the growth cartilage after IV infusion. We recently have also shown that GALNS can reduce keratan sulfate storage in Morquio chondrocytes. This is important as the skeletal system is a primary concern in the treatment of this disease."

"We are excited to be the first site to enroll patients in the BioMarin Clinical Assessment Program for MPS IVA patients. This study is crucial to developing a deeper understanding of the clinical outcomes for this rare disorder, which will help lead to better disease management and therapy options, said Barbara B
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SOURCE BioMarin Pharmaceutical Inc.
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