Company will review data; analyze positive trends beyond initial results
Shire announced today that top-line data from its phase II/III clinical trial for MPS II did not meet primary or key secondary endpoints. The endpoints looked at General Conceptual Ability (GCA) and Adaptive Behavior Composite (ABC) scores in Hunter syndrome patients.
The National MPS Society is disappointed in the news shared today for our Hunter patient community but remains optimistic for future possibilities.
“We know the strain clinical trials put on families and how disappointing outcomes, such as this one, can impact our community,” said Terri Klein, National MPS Society interim CEO. “Still, with hope, we are encouraged by Shire’s commitment to review individual subset data and identify potential positive trends in patients. This data could unlock a positive path to regulatory and the FDA – time will tell.”
In addition, Shire said patients currently enrolled in the clinical trial will retain access to SHP609, which is an investigational formulation of idursulfase administered intrathecally. The Society will continue our conversations with Shire and with SHP609 families and patients already receiving this treatment.
“It is important that we take the time to thank our children and their families, as well as the medical community committed to this clinical trial,” Klein said. “Without your many efforts, these opportunities could not exist and change the future for those living with rare diseases. We are committed to finding viable options for cognitive issues in Hunter Syndrome.”
For more information, please visit Shire’s website.