Hunter Syndrome: Shire Collaboration And FDA Acceptance Of ArmaGen’s IND
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 technology designed to “take advantage of the body’s natural system for transporting products across the BBB, by binding to the same receptor that delivers insulin to the brain”. AGT-182 has Orphan Drug Designation (ODD) from both the FDA (May 2013) and the European Medicines Agency (EMA).
In July 2014, ArmaGen enters into a worldwide licensing and collaboration agreement with Shire plc to develop AGT-182 for treatment of both the central nervous system and somatic manifestations of Hunter Syndrome. Under the agreement:
“… Shire receives worldwide commercialization rights for AGT-182. ArmaGen will receive payments up to approximately $225 million, including an initial upfront payment of $15 million in cash and equity, an additional equity investment, R&D funding, development and sales milestones, in addition to future royalties up to double digits. The collaboration between ArmaGen and Shire on AGT-182 will be managed by a joint steering committee, with representatives from both companies. ArmaGen will be responsible for conducting a Phase 1/2 study of AGT-182 … Shire will be responsible for further clinical development, including Phase 3 trials, registration and commercialization of AGT-182 worldwide.”
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