Shire Hunter syndrome treatment drug clinical phase II/III failure
Shire Hunter syndrome treatment drug clinical phase II/III failure
December 20, 2017 Source: Sina Pharmaceutical
Window._bd_share_config={ "common":{ "bdSnsKey":{ },"bdText":"","bdMini":"2","bdMiniList":false,"bdPic":"","bdStyle":" 0","bdSize":"16"},"share":{ }};with(document)0[(getElementsByTagName('head')[0]||body).appendChild(createElement('script')) .src='http://bdimg.share.baidu.com/static/api/js/share.js?v=89860593.js?cdnversion='+~(-new Date()/36e5)];On December 19th, Shire, the world leader in rare diseases, announced its final results of Phase II/III clinical trials evaluating SHP609 (formerly known as HGT-2310). SHP609 is an experimental preparation of the drug idursulfase, a new potential indication for the treatment of Hunter's syndrome (mucopolysaccharidosis type II or MPS II) and cognitive impairment by intrathecal injection.
Phase II/III clinical trials were a controlled, randomized, open-label, multicenter, and evaluator blinded trial that did not reach the primary or critical secondary endpoint. The primary endpoint was to assess the cognitive difference between the SHP609 treatment group and the control group, and the difference was measured by baseline changes in the general concept ability (GCA) score after 12 months of treatment in Hunter's syndrome patients. Key secondary endpoints The differences between the SHP609 treatment group and the control group were assessed by measuring baseline changes in the Adaptive Behavior Synthesis (ABC) score.
Dr. Howard Mayer, senior vice president and global research director (temporary) of Shire, said: "Shire is disappointed that clinical data is not at the primary and secondary endpoints, but is still committed to helping patients with mucopolysaccharidosis type II and their families. Thanks to the children, families and medical institutions involved in this challenging trial, we will continue to engage in dialogue with the group to analyze the entire set of data. Further data analysis will be presented at the upcoming conference."
Dr. Joseph Muenzer, professor of pediatric genetics and metabolic genetics at the University of North Carolina's Chapel Hill School of Medicine, said: "Hunt syndrome is a rare and debilitating genetic disorder that is usually caused by early childhood enzyme deficiency. Two-thirds of patients show a progressive decline in cognitive ability, which can be devastating for patients and their families, as this will severely reduce a child's functional ability and often lead to death in adolescence."
Previously, ELAPRASE (idursulfase), which has been approved by more than 70 countries around the world, needs intravenous injection, and it will continue to be an important drug for patients with Hunter's syndrome in terms of its current indications, and is not affected by these results.
One in 162,000 survivors is affected by Hunter's syndrome and is almost always male. This is a severely debilitating lysosomal storage disease (LSD) caused by a 2-sulfatase deficiency, which is an enzyme required to break down glycosaminoglycans (GAGs) in the body. . Without this enzyme, GAG accumulates and damages various organs, causing a range of signs and symptoms associated with the disease, such as decreased hearing, decreased heart function, obstructed airway disease, hepatosplenomegaly, and reduced range of motion and activity. Physical manifestations may include significant facial features, and the head and abdomen become larger. In many cases, the central nervous system may also be affected.
About 2/3 of Hunter's syndrome patients are affected by a decline in progressive cognitive decline. SHP609 was specifically developed to be administered directly by injecting cerebrospinal fluid as a means of delivering the drug to the central nervous system (intrathecal). (Sina Pharmaceutical Compilation / Bernardo)
Article, image reference source: Shire's Hunter Syndrome Treatment Failed Phase II/III Goals
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