Ultragenyx stumbles with UX007 PhIII, shares plunge
Rare disease specialist Ultragenyx Pharma $RARE has abandoned its experimental drug, UX007, as a treatment for Glut1 DS – a debilitating disease characterized by seizures, developmental delay, and movement disorders. The drugmaker, whose stock fell sharply in early trading, said the drug failed to demonstrate a statistically significant improvements in a Phase III trial.
UX007, also dubbed triheptanoin, was designed to address glucose transporter type-1 deficiency syndrome — or Glut1 DS — by getting glucose into the brains of the genetically afflicted group. Without adequate glucose, patients have a tendency to suffer from repeated seizures. Roughly 500 cases have been reported worldwide since the disorder was first identified in 1991, according to the NIH.
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