UX003: Preliminary Phase I/II data

Preliminary data from 3 patients ages 5-30 years 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 2, 6 and 12 weeks, respectively, led to no serious adverse events and no infusion-associated reactions. Additionally, urinary GAG excretion decreased by about 30-50% beginning at week 2. At the 12-week assessment of the first patient, absolute liver size was reduced by about 11%. A dose-exploration and long-term extension will follow the 12-week primary analysis phase. The trial is enrolling up to 5 patients. Data were presented at the American College of Medical Genetics and Genomics meeting in Nashville. ...