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BiotechTracker
- Aryplase Enters Phase III |
July 08, 2003 BioMarin (NASDAQ: BMRN) announced its preparedness to enroll approximately 36 study subjects for randomized, double-blind, placebo-controlled, phase III clinical investigation of Aryplase (recombinant arylsulfatase B) as enzyme replacement therapy in the treatment of muccopolysaccarhidosis VI (MPS VI, Maroteaux-Lamy Syndrome). The company hopes to complete the double-blind phase of the study “within approximately nine months,” with success leading to filing U.S. and E.U. regulatory applications “in the second half of 2004.” Mid-phase results from this program, although limited in scope, are encouraging.
The phase III program’s primary endpoint will be the change in endurance as measured by the distance walked in 12 minutes. Secondary endpoints will include change in urinary glycosaminoglycan (GAG) excretion and the change in the number of stairs climbed in three minutes.
MPS VI is an inborn genetic error of metabolism caused by a deficiency of the intracellular enzyme arylsulfatase B, an enzyme responsible for the breakdown of specific GAGs. GAGs accumulate in the cell’s digestive organelles, the lysosomes, leading to progressive cell, tissue, and organ system dysfunction. Typically, deterioration occurs in cardiac and pulmonary function, physical development, and impaired vision and hearing, and patients experience skeletal and joint deformities, sleep apnea, and progressively reduced endurance. Most patients die from disease-related complications between childhood and early adulthood.
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