RhGUS: Additional Phase I/II data

Additional data from 3 patients ages 5-30 with MPS VII in an open-label, U.K. Phase I/II trial showed that 2 mg/kg IV UX003 given every other week for 12 weeks led to 40-50% reductions in urinary GAG excretion from baseline at week 12. Additionally, UX003 reduced liver size in 2 patients with enlarged livers at baseline. No serious adverse events or infusion-associated reactions were reported. A dose-exploration and long-term extension will follow the 12-week primary analysis phase. Data were presented at the Society for the Study of Inborn Errors of Metabolism meeting in Innsbruck, Austria. Ultragenyx reported preliminary data in March (see BioCentury, April 21). ...