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Shire genetic disease drug fails in phase 2/3 trial

Shire plc said the phase 2/3 trial of SHP609 in children with Hunter syndrome and cognitive impairment failed to meet its main and secondary goals.

Hunter syndrome is a rare, genetic disorder caused by an enzyme deficiency that develops in early childhood. It diminishes a child's functional ability and is typically fatal.

SHP609 is a formulation of Shire's Elaprase administered intrathecally, or through an injection into the spinal canal.

Shire said the treatment missed the study's main goal, which evaluated the difference in cognition of children treated with SHP609 and those in control groups after 12 months of treatment.

SHP609 also failed to meet the secondary goal, which evaluated the difference between the SHP609-treated children and the control group children as measured by the Adaptive Behavior Composite score.