Hunter Syndrome: Shire Collaboration And FDA Acceptance Of ArmaGen’s IND

Orphan Druganaut Blog

ArmaGen, a Calabasas, California-based clinical stage biotechnology company  developing therapies for severe neurological disorders, announces December 11th, that the FDA’s Investigational New Drug (IND) application for the company’s lead product candidate, AGT-182 for Hunter Syndrome (Muchopolysaccharidosis II or MPS II), is accepted and is now active. This will enable a Phase I clinical trial, to assess the safety and tolerability of AGT-182 in adult male patients with Hunter Syndrome, to start in the first quarter of 2015.

Hunter Syndrome is a progressive, rare, and severe Lysosomal Storage Disorder (LSD), caused by inadequate activity of the enzyme that breaks down complex sugars – Iduronate-2-Sulfatase (IDS). Current treatments are not expected to cross the Blood-Brain Barrier (BBB) in relevant amounts, and thus do not deal with the neurological complications of Hunter Syndrome.

AGT-182 is an investigational Enzyme Replacement Therapy (ERT) for Hunter Syndrome. What is unique about AGT-182 is the proprietary…

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