BioCentury
ARTICLE | Clinical News

GALNS: Phase III data

November 12, 2012 8:00 AM UTC

The double-blind, international Phase III MOR-004 trial in 176 patients with MPS-IVA showed that once-weekly 2 mg/kg IV GALNS met the primary endpoint of improving 6MWD from baseline to week 24 vs. placebo, while 2 mg/kg GALNS given every other week missed the endpoint. Specifically, once-weekly GALNS led to a mean increase of 22.5 meters in 6MWD over placebo (p=0.0174). Observed mean improvements in 6MWD from baseline to week 24 were 13.5 meters for placebo, 36.5 meters for once-weekly GALNS and 14.9 meters for GALNS given every other week. BioMarin said the p-values are based on a pre-specified primary statistical analysis model and that the mean changes in the endpoints vs. placebo are the treatment effects based on the model and may differ slightly from the means of the observed data.

Once-weekly GALNS also met the secondary endpoint of reducing mean urinary keratan sulfate levels from baseline to week 24 vs. placebo (40.7% reduction over placebo, p<0.0001). Observed reductions in mean urinary keratan sulfate levels from baseline to week 24 were 4.4% for placebo, 45.1% for once-weekly GALNS and 35.2% for GALNS given every other week. Once-weekly GALNS missed the secondary endpoint of improving endurance as measured by the 3-minute stair climb test vs. placebo (improvement of 1.1 additional stairs per minute over placebo, p=0.4935). Observed improvements in the 3-minute stair climb test from baseline to week 24 were 3.6 stairs per minute for placebo, 4.8 stairs for once-weekly dosing and 3.4 stairs for dosing every other week. ...