Shire hit by a setback as Hunter syndrome drug fails late-stage study

Shire’s attempt to develop a new formulation of Elaprase that could be used to prevent cognitive decline in patients with rare cases of Hunter syndrome has just slammed into a late-stage wall.

Joseph Muenzer

Researchers say the drug, SHP609, failed both the primary as well as key secondary endpoints. The primary focused on a measure of cognition for the children in the study after 12 months of treatment, while the key secondary was an evaluate of the Adaptive Behavior Composite for the drug and control arms.

Elaprase is an IV drug that can’t cross the blood-brain barrier, leaving Shire $SHPG to see if this new formulation of the drug could make it into the brain through cerebrospinal fluid.

We don’t have the data today, just the top-line results. And researchers still plan to do follow-up work examining the results. But a late-stage failure like this doesn’t bode well for Shire, which has been concentrating on developing new therapies for rare diseases.

“Hunter syndrome is a severely debilitating rare genetic disorder caused by an enzyme deficiency which typically presents in early childhood,” said Joseph Muenzer, professor of pediatric genetics and metabolism genetics at the University of North Carolina Chapel Hill School of Medicine. “Two out of three patients exhibit progressive cognitive decline which is a high unmet need. This can be devastating for patients and their families as it severely diminishes a child’s functional ability and typically leads to death in the teenage years.”

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