BioMarin Phase 3 Study of GALNS Meets Primary Endpoint

BioMarin Pharmaceutical BMRN announced today that the pivotal Phase 3 study of GALNS met the primary endpoint of change in six-minute walk distance compared with placebo at 24 weeks in subjects receiving weekly infusions of GALNS at the dose of 2 mg/kg (p=0.0174). MOR-004 was a randomized, double-blind, placebo-controlled study evaluating two doses of GALNS (BMN-110, N- acetylgalactosamine-6-sulfatase) for the treatment of patients with the rare lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), also called Morquio A Syndrome. Patients dosed with GALNS at 2 mg/kg every other week did not show a meaningful or statistically significant change from baseline compared to placebo. The company also announced preliminary data from the MOR-005 extension study which suggests that clinical benefits continue to improve with further dosing with GALNS. Only a limited number of patients have reached the 36 or 48 week points of total time on treatment in the extension study, and the results will be updated when the study is completed. The company confirmed that based on the results See full press release