In a move to strengthen its rare-disease drug pipeline, the specialty pharmaceutical company Shire plc has formed a worldwide licensing and collaboration agreement with ArmaGen, a US privately held biotechnology company, for AGT-182, an investigational enzyme replacement therapy for the potential treatment of both the central nervous system (CNS) and somatic manifestations in patients with Hunter syndrome. 

Under the terms of the agreement, Shire will obtain worldwide commercialization rights for AGT-182 in exchange for payments of approximately $225 million to ArmaGen, including an initial upfront payment of $15 million in cash and equity, an additional equity investment, R&D funding, development milestones, sales milestones, and  royalty payments. As part of the agreement, ArmaGen will be responsible for conducting and completing the Phase I/II study which it expects to initiate before the end of 2014, after which point Shire will be responsible for further clinical development, including Phase III trials, and commercialization.

Shire researched, developed and commercialized the first treatment approved for Hunter syndrome, according to the company. This agreement with ArmaGen expands Shire's commitment to finding treatments for Hunter syndrome, which also includes SHP-609, Shire's product currently being investigated to treat the CNS manifestations associated with Hunter syndrome.

AGT-182, which has received orphan drug designation from both the US Food and Drug Administration and the European Medicines Agency, is designed to take advantage of the body's natural system for transporting products across the blood brain barrier (BBB) by using the same receptor that delivers insulin to the brain. AGT-182 is engineered by the fusion of the replacement IDS enzyme to an antibody that binds to a receptor on the BBB. The IDS enzyme is designed to cross the BBB attached to that antibody.

Shire has made the deal following the announced acquisition by AbbVie of Shire for $55 billion. Pending shareholder approval and customary closing and regulatory approvals, that deal is expected to close in the fourth quarter of 2014. 

See related story, “AbbVie and Shire: Examining the Proposed Combined Company”

Source: Shire