GALNS: Extension study preliminary data

BioMarin reported preliminary data from patients with MPS-IVA who have reached 36 and 48 weeks of total treatment time with GALNS in the double-blind, international Phase III MOR-005 extension study of the double-blind, international Phase III MOR-004 trial in 176 patients. Patients receiving once-weekly 2 mg/kg IV GALNS had mean increases in 6-minute walk distance (6MWD) from baseline of 46.6 meters at week 36 (n=27) and 45.4 meters at week 48 (n=13). Patients receiving 2 mg/kg GALNS given every other week had mean increases in 6MWD from baseline of 17 meters at week 36 (n=27) and 3.8 meters at week 48 (n=18).

Once-weekly GALNS improved endurance as measured by the 3-minute stair climb test by 6.5 stairs per minute at week 36 (n=27) and 7.1 stairs per minute at week 48 (n=16). GALNS given every other week improved 3-minute stair test scores by 5.3 stairs per minute at week 36 (n=27) and 5.8 stairs per minute at week 48 (n=18). Additionally, reductions in mean urinary keratan sulfate levels from baseline for once-weekly GALNS were 45.8% at week 36 (n=19) and 45.9% at week 48 (n=10). GALNS given every other week led to reductions in mean urinary keratan sulfate levels from baseline of 30.2% at week 36 (n=25) and 31.7% at week 48 (n=9). ...