Ultragenyx Pharmaceutical (RARE -3.6%) announces positive 78-week data from an open-label 29-subject Phase 2 clinical trial assessing UX007 in patients with long-chain fatty acid oxidation disorder (LC-FAOD). Treatment with UX007 reduced the frequency and duration of major medical events while improving participants' exercise tolerance and quality of life.
Specifically, there was a 48.1% reduction in the mean annualized rate of major clinical events (events that led to hospitalization, emergency room visit or an emergency intervention at home).
At baseline, eight patients performed the 12-minute walk test (12MWT). At week 60, average distance improved 29.7%. The cycle ergometry test at week 78 was inconclusive and uninterpretable due to missing data.
Five patients discontinued treatment over the course of the study, four for reasons not attributed to treatment with UX007 and one due to diarrhea at week 1 which resolved after treatment was stopped.
LC-FAOD is a group of autosomal recessive genetic disorders in which sufferers cannot convert long-chain fatty acids into energy. This leads to glucose depletion and serious liver, muscle and heart disease. Current standard-of-care treatment includes the avoidance of fasting, low-fat/high carb diets and dietary supplements carnitine and medium-chain triglyceride oil.
UX007 is a purified pharmaceutical-grade form of a triglyceride compound called triheptanoin that provides patients with medium-length odd-chain fatty acids which increase the intermediate substrates in the Krebs cycle, a key energy-generating process, which produces new glucose.
The company intends to advance UX007 into Phase 3 development.