Rare Daily Staff

Takeda said it would discontinue development of its experimental Hunter syndrome therapy TAK-609 after years of extensive regulatory discussions.

The company made the announcement as part of its quarterly earnings report.

Hunter syndrome (also known as mucopolysaccharidosis II or MPS II) is a severely debilitating, rare lysosomal disease 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, causing a range of disease-related signs and symptoms. Roughly two of every three patients with Hunter syndrome are also affected with progressive cognitive decline. 

TAK-609 is an experimental formulation of idursulfase administered intrathecally for a new potential indication for the treatment of pediatric patients with Hunter syndrome and cognitive impairment.

Takeda began developing TAK-609 after it acquired the experimental therapy through its acquisition of Shire, completed in 2019. At the end of 2017, Shire reported top-line results from its phase 2/3 clinical trial and reported that the study failed to 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.

The company said it remains committed to developing therapies for Hunter syndrome and other lysosomal storage disorders.

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