Triheptanoin: Phase II data

Interim data from an open-label, international Phase II trial in 29 patients ages 10 months to 58 years with moderate to severe long-chain fatty acid oxidation disorders showed that UX007 improved cycle ergometry workload by 60% from baseline to week 24 in 7 evaluable patients. Specifically, cycle ergometry workload increased by a mean of 446.8 watts at week 24 from 744.6 watts at baseline. No patient who completed all 40 minutes of the cycle ergometry test at baseline and at week 24 had a reduced duration of work. In the 3 patients who were not able to complete all 40 minutes at baseline, mean duration of work increased by 11.1 minutes at week 24 from 11.5 minutes at baseline. In 8 evaluable patients who completed the 12-minute walk test (12MWT) at baseline and at week 18, UX007 led to a mean increase of 188 meters at week 18 from 673.4 meters at baseline. Seventeen patients were either too young to complete the exercise testing or were unable to complete the testing due to other physical constraints. In the 6 adults who participated in the cycle ergometry and 12MWT, UX007 significantly improved both the physical and mental health composite scores and all subscores of the Short Form-12 Health Survey (SF-12) at week 24. ...