Ultragenyx announces 'positive' data from UX007 Phase 2 study
Ultragenyx Pharmaceutical announced positive 78-week data from the Phase 2 study of UX007 in patients with long-chain fatty acid oxidation disorder, or LC-FAOD. The frequency and duration of major medical events were reduced significantly during treatment with UX007, and patients demonstrated improved exercise tolerance and quality of life during the study. "The reduction in frequency and duration of hospitalizations and other major medical events suggests a clinically meaningful improvement for patients and is consistent with the data from the prior retrospective compassionate use study," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "These results are encouraging and we continue to further develop the Phase 3 study design and endpoints before meeting with regulators and initiating the study in 2017."