19 dec 2017 : SHiRE Press Release

Shire Announces Top-Line Results for Phase II/III Clinical Trial in Children with Hunter Syndrome and Cognitive Impairment

Cambridge, Ma. – December 19, 2017 – Shire plc (LSE: SHP, NASDAQ: SHPG), the global leader in rare diseases, today announced top-line results from its Phase II/III clinical trial evaluating SHP609, previously known as HGT-2310. SHP609 is an investigational formulation of idursulfase administered intrathecally for a new potential indication for the treatment of pediatric patients with Hunter syndrome (mucopolysaccharidosis II or MPS II) and cognitive impairment.

The study did not meet either its primary or its key secondary endpoint. The 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.


Link naar de volledige press release op de Shire website: