Phase 3 Study of GALNS for Mucopolysaccharidosis Type IVA

BioMarin Pharmaceuticals announced that the pivotal Phase 3 study of GALNS for the treatment of patients with the rare lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA; also called Morquio A Syndrome) met its primary endpoint.

The MOR-004 study was a randomized, double-blind placebo-controlled clinical trial designed to evaluate the efficacy and safety of 2mg/kg/week and 2mg/kg/every other week of GALNS (BMN-110, N-acetylgalactosamine-6-sulfatase) in patients with MPS IVA.

The primary endpoint of the study, change in six-minute walk distance at 24 weeks, was statistically significant in patients dosed with GALNS at 2mg/kg every week with a mean increase of 22.5 meters (P=0.0174) over placebo. Patients dosed at 2mg/kg every week showed an improvement in six-minute walk distance at Week 12 compared to baseline and showed continued improvement at Week 24.

On the secondary endpoint of three-minute stair climb, patients dosed with GALNS at 2mg/kg every week showed a trend toward improvement at 24 weeks of 1.1 additional stairs per minute over placebo. Patients dosed with GALNS at 2mg/kg every week showed an improvement in three-minute stair climb performance at Week 12 compared to baseline and showed continued improvement at Week 24. In the other secondary endpoint, urinary keratan sulfate (KS) levels, patients dosed with GALNS at 2mg/kg every week showed consistent and robust reduction in urinary KS with a mean difference from baseline as compared to placebo of 40.7% (P<0.0001).

Pulmonary function, as defined by maximum voluntary ventilation (MVV) was measured at 24 weeks; patients dosed with GALNS at 2mg/kg every week showed a trend toward improvement from baseline of 10.3% over placebo. Pulmonary function, as defined by forced vital capacity (FVC) was also measured at 24 weeks; patients dosed with GALNS at 2mg/kg every week showed a trend toward improvement from baseline of 3.3% over placebo. 

MPS IVA is a disease characterized by deficient activity of N-acetylgalactosamine 6-sulfatase (GALNS) causing excessive lysosomal storage of KS. This excessive storage causes a systemic skeletal dysplasia, short stature, and joint abnormalities, which limit mobility and endurance.  BioMarin's GALNS therapy is intended as an enzyme replacement to treat MPS IVA.

For more information call (877) 695-8826 or visit www.bmrn.com.