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March 27, 2014
09:06 EDTRAREUltragenyx announces preliminary data from phase 1/2 study of rhGUS, UX003
Ultragenyx Pharmaceutical announced the presentation of preliminary data from the Phase 1/2 study of recombinant human beta-glucuronidase, or rhGUS, UX003, an investigational therapy for the treatment of mucopolysaccharidosis 7. The Phase 1/2 open-label clinical study is assessing the safety, efficacy, and dose of rhGUS administered every other week via intravenous infusion in a 12-week primary analysis phase, which will be followed by dose-exploration and long-term extension. Preliminary results from three patients who have been administered 2 mg/kg of rhGUS every other week for two, six, and 12 weeks show evidence of clearance of lysosomal storage as indicated by the decline in urinary glycosaminoglycan excretion beginning at two weeks of treatment of approximately 30-50%. At the 12 week assessment of the first patient, absolute liver size was reduced by approximately 11%. This represents a 46% decrease in the excess liver size above normal for age and gender. The remaining patients have not yet reached the 12 week time point for liver size assessment. No serious adverse events were observed during up to 12 weeks of treatment, and no infusion-associated reactions were observed after a total of 13 infusions to date in these three subjects. The study will continue and additional 12-week interim data are expected in 2014. If the efficacy and safety results for all treated subjects are supportive and the dose confirmed, the company intends to initiate a pivotal Phase 3 study enrolling at least 12 patients in 2014.
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