Shire’s Hunter Syndrome Drug Disappoints in Late-Stage Study

Dec. 20, 2017

Shire announced that its experimental treatment for children with Hunter syndrome and cognitive impairment, SHP609, failed to meet the main and secondary goals of a late-stage clinical trial.

SHP609 is being tested in combination with Elaprase, Shire’s existing drug that treats Hunter syndrome but not cognitive impairment.

According to Shire, the study’s primary endpoint evaluated the difference in cognition between the SHP609-treated and control groups, as measured by change from baseline in General Conceptual Ability (GCA) scores in children with Hunter syndrome after 12 months of treatment. The key secondary endpoint evaluated the difference between the SHP609-treated and control groups as measured by the change from baseline in Adaptive Behavior Composite (ABC) score.

Hunter syndrome is a severely debilitating, rare lysosomal storage disorder that affects almost exclusively males.