Shire Hunter syndrome drug fails to meet primary and secondary endpoints

Shire said on Tuesday that clinical trials for a drug to treat Hunter syndrome in children, SHP609, failed to meet their primary and secondary endpoints.

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.

Howard Mayer, senior vice president and global Head of R&D, said: "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.

"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 lysosomal storage disorder (LSD) caused by a deficiency of iduronate-2-sulfatase, an enzyme that is needed to break down substances in the body called glycosaminoglycans (GAGs). Without this enzyme, GAGs can build up in and damage various organs, causing a range of disease-related signs and symptoms such as hearing loss, declined cardiac function, obstructive airway disease, enlargement of the liver and spleen and decreased range of motion and mobility.

Also on Tuesday, Shire said the US Food and Drug Administration has granted 510k marketing clearance to a free web-based software that is the first and only FDA-cleared pharmacokinetic (PK) dosing software for haemophilia.

Shire said the myPKFiT for ADVATE software represents an innovative approach to estimating a patient’s PK curve, a key measure for assessing drug exposure over time.

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