Biotech Tracker News - BioMarin’s Aryplase Moves Toward Phase III
March 14, 2003 BioMarin’s (NASDAQ: BMRN) Aryplase (recombinant human arylsulfatase B) generated open-label, phase II data supporting its advancement to pivotal investigation as an enzyme replacement therapy for the management of mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome). BioMarin plans to initiate a double-blind, placebo-controlled, phase III trial of Aryplase in 2003.  10 patients were assessed for 24 weeks, receiving Aryplase 1.0 mg/kg infusions. Aryplase administration was associated with the following improved outcomes:  average 6-minute walk test distance – 62%  average 12-minutes walk test distance – 98%  average stairs climbed in 3 minutes – 110%  joint pain – 57%  joint stiffness – 54%  shoulder range of motion for flexion, extension, and lateral rotation.  These clinical improvements were associated with an average decrease in urinary glycosaminoglycan (GAG) excretion of 71% in 24 weeks. Such a decrease, in the context of the clinical improvements, confirms the clinically relevant mechanism of Aryplase action reduced carbohydrate storage in a clinically meaningful way.  Other endpoints were not met with statistical significance, but BioMarin hopes that a longer treatment duration might at least have an impact on: forced vital capacity, pinch and grip strength test, physical activity, oxygenation level during sleep, expanded time to get up and go test, and a set of tasks reflecting quality of life. The impact each of these endpoints will have on approvability remains to be explored. Aryplase’s tolerability and adverse effect profiles were manageable.  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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