UX001: Additional Phase II data
Additional data from a 48-week, double-blind, placebo-controlled, U.S. and Israeli Phase II trial in 46 patients ages 18-65 years showed that once-daily 6 mg UX001 for 24 or 48 weeks led to a "modest" increase from baseline in upper extremity muscle strength. Ultragenyx said the improvement with the high dose was significant compared to the decline in upper extremity muscle strength observed in patients receiving a once-daily 3 mg dose of UX001 for 24 or 48 weeks. Patients received low or high dose UX001 for 48 weeks or placebo for 24 weeks followed by either dose of UX001 for an additional 24 weeks.
In patients who received high-dose UX001 for 48 weeks, the increase in upper extremity muscle strength observed at 24 weeks was sustained at week 48, while patients who received low-dose UX001 for 48 weeks experienced a further decline in upper extremity muscle strength from week 24 to week 48. ...