UX 007 fails Phase III trial to treat patients with glucose transporter type-1 deficiency syndrome. Ultragenyx

Ultragenyx Pharmaceutical Inc. ,announced its Phase III study of UX 007 in patients with glucose transporter type-1 deficiency syndrome (Glut1 DS) experiencing disabling paroxysmal movement disorders did not achieve its primary endpoint of demonstrating a statistically significant reduction in the frequency of paroxysmal movement events with UX 007 treatment compared to placebo, and did not demonstrate a meaningful difference between treatment groups. The study also did not meet its key secondary endpoints. The safety profile observed in this study was consistent with what has been previously reported with UX 007. Ultragenyx plans to discontinue the Glut1 DS development program, and will work with investigators and patients on a reasonable transition plan for patients with Glut1 DS who are still on UX 007. Ultragenyx has a separate program evaluating UX 007 in long-chain fatty oxidation disorders (LC-FAOD), which continues on track. The FDA has accepted the Company�s proposal to submit a New Drug Application (NDA) for UX 007 for the treatment of LC-FAOD based on existing data, and a pre-NDA meeting with the FDA will take place before the end of 2018. In the EU, Ultragenyx will discuss these data with the European Medicines Agency and expects to have an update this year.