Positive new Phase III data on Ultragenyx' UX003 drug candidate
US ultra-rare diseases focussed biotech firm Ultragenyx Pharmaceutical (Nasdaq: RARE) has announced positive top-line data from the pivotal Phase III study of recombinant human beta-glucuronidase (rhGUS, UX003), an investigational therapy for the treatment of Mucopolysaccharidosis 7 (MPS 7, Sly syndrome).
The study met its primary endpoint of reducing urinary GAG (dermatan sulfate) excretion after 24 weeks of treatment, demonstrating a reduction from baseline of 64.8% (p<0.0001). The data are being presented at the 14th International Symposium on MPS and Related Diseases.
The company’s shares traded up 7.33% during midday trading on Friday, reaching $53.17. Wedbush analysts currently have an “Outperform” rating and a $92.00 target price on the stock.
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