Ultragenyx reports interim LC-FAOD data

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) fell $7.40 to $82.49 on Tuesday after releasing interim results from a Phase IIb study of triheptanoin ( UX007) to treat long-chain fatty acid oxidation disorders. The company said triheptanoin increased muscle performance and exercise tolerance in cycling and walking tests, but data were insufficient to show effects on liver and cardiac disease. Ultragenyx said that the study's design "limits definitive conclusions about efficacy and safety," but it plans to use the results to determine the appropriate patient population and endpoints of a Phase III study.

Ultragenyx reported a 60% mean increase in workload in seven patients who participated in a cycle ergometry test after 24 weeks of triheptanoin treatment. It also reported a 28% mean increase in walking distance in the eight patients who completed a 12-minute walk test after 18 weeks. The study included LC-FAOD patients ranging from 10 months to 58 years old. ...