MPSII Clinical Trial Update – First potential treatment for children with Hunter disease and cognitive impairment misses its primary endpoints
The Society for Mucopolysaccharide Diseases (MPS Society) is disappointed to learn that Shire’s SHP609 clinical trial has missed its Primary Endpoints and failed to demonstrate Stabilization of Cognitive Decline in Children with Hunter Disease as stated in their press release below:
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 paediatric 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 Behaviour Composite (ABC) score.
“Shire is disappointed that the top-line data from this study did not meet the primary and key secondary endpoints and remains committed to patients and families living with MPS II,” said Howard Mayer, M.D., Senior Vice President and Global Head of R&D (ad-interim), Shire. “We are grateful to the children, their families and healthcare providers for participating in this challenging trial and will continue our ongoing dialogue with the community as we conduct an analysis of the full data set. Further analysis of the data will be presented at forthcoming congresses.”
“Hunter syndrome is a severely debilitating rare genetic disorder caused by an enzyme deficiency which typically presents in early childhood,” said Joseph Muenzer, M.D., Ph.D., Professor of Pediatric Genetics and Metabolism Genetics, 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.”
The MPS Society encourages Shire to share its learning from this clinical trial for the benefit of all those affected by Hunter disease and cognitive decline.
For Further information please contact:
MPS SOCIETY – 0345 389 9901
Bob Stevens 07786 271125
Sophie Thomas 07920 234802
About the Society for Mucopolysaccharide Diseases (MPS)
The MPS Society was established in 1982 and now provides support and advocacy to over 1300 individuals with Mucopolysaccharide and related diseases, their families and carers. The Society provides an information service and brings about public awareness of these devastating conditions. Over the last 20 years the Society has funded over £5 million in research that is hoped will lead to treatments for all of those affected.