Rare Disease Report

New Data on AGT-182 for Hunter Syndrome Treatment

JULY 14, 2016
Andrew Black
New information was presented at the 14th International Symposium on MPS and Related Diseases located in Bonn, Germany in the Phase 1/2a clinical trial of AGT-182, an enzyme replacement therapy (ERT) tin development to treat Hunter syndrome (also known as MPII).
The trial is an open-label multi-dose study with the primary endpoint of safety and tolerability in the dosing of AGT-182. The investigational ERT is a compound that utilizes the body’s natural system for delivering products across the blood-brain barrier (BBB) in which in targets the receptor that transports insulin to the body’s cells.
ArmaGen’s new data of a small cohort (n=4), dosing of AGT-182 at 1.0-mg/kg to patients weekly, administered as an intravenous (IV) infusion to attenuated Hunter patients, was overall well-tolerated. Because of the safety, clinical, and bioanalytical data, an independent Data Monitoring Committee (DMC) has recommended ArmaGen proceeds to study's second a cohort, in which adult patients are to receive a weekly IV infusion starting at a dose of 3 mg/kg. As with the first cohort, patients enrolled in the second cohort will be age 18 and over.
Data of four adult male patients with Hunter syndrome who received weekly IV infusions of the ERT was also presented at MPS 2016. With the eight-week assessment, significant decreases in liver and spleen volume in two out of the 4 patients occurred. Along with levels of urinary glycosaminoglycans (GAGs), a marker for Hunter syndrome, also showed clinically significant decreases in all four patients.
ArmaGen will proceed to do a cohort 2 due to the DMC’s recommendation for patients to receive dosing of AGT-182 at 3 mg/kg
In March of 2015, we interviewed Steven L Schoenfeld, MD, vice president, clinical affairs at ArmaGen about AGT-182. 

Hunter Syndrome

Hunter syndrome, also known as mucopolysaccharidosis type II, or MPS II, is a lysosomal storage disorder caused by inadequate activity of the enzyme iduronate-2-sulfatase (IDS), which is needed to break down complex sugars produced by the body. The buildup of these complex sugars, known as mucopolysaccharides, interferes with functioning of certain cells and organs, leading to serious complications including developmental delays and mental impairment.

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