Ultragenyx's UX007 fails Phase III in glucose transporter disorder

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) said it will discontinue development of UX007 to treat glucose transporter type-1 deficiency syndrome (Glut1 DS, De Vivo disease) after the therapy missed the primary and key secondary endpoints in a Phase III trial. Ultragenyx lost $10.74 (18%) to $48.62 Friday.

The trial's primary endpoint measured the change from baseline in the frequency of disabling paroxysmal movement disorder events at week 22. Secondary endpoints included the duration of those disabling events; walking capacity and endurance; and patient-reported quality of life assessments. The double-blind, crossover trial enrolled 44 patients to receive UX007 followed by placebo, or placebo followed by UX007...