FDA clears ArmaGen's AGT-182 IND application for treatment of Hunter syndrome

ArmaGen, Inc., a privately held biotechnology company focused on developing novel therapies to treat severe neurological disorders, announced today that the Investigational New Drug (IND) application for the company's lead product candidate, AGT-182 for the treatment of Hunter syndrome, has been accepted by the U.S. Food and Drug Administration (FDA) and is now active. This will enable ArmaGen to initiate a Phase 1 clinical trial, which is expected to begin in the first quarter of 2015, to assess the safety and tolerability of AGT-182 in adult male patients with Hunter syndrome.

AGT-182 is an investigational enzyme replacement therapy (ERT) for the treatment of Hunter syndrome. Also known as mucopolysaccharidosis type II, or MPS II, Hunter syndrome is a rare, severe, progressive and life-limiting lysosomal storage disorder.

"Commercially available treatments for Hunter syndrome lack the ability to penetrate the blood-brain barrier in clinically relevant amounts, and therefore do not address the severe and progressive neurological complications of the disease," said James Callaway, Ph.D., Chief Executive Officer of ArmaGen. "Now that the IND is active, we hope to soon be able to demonstrate the safety of AGT-182 and eventually fill a significant unmet need for the Hunter syndrome community."

AGT-182 is designed to utilize the body's natural system for transporting products across the blood-brain barrier (BBB) by targeting the receptor that delivers insulin to all cells of the body.

"We encourage patients and caregivers living with Hunter syndrome to learn more about ongoing clinical trials of investigational therapies," said Barbara Wedehase, MSW, CGC, Executive Director of the National MPS Society. "Delivery of enzyme replacement therapeutics into the central nervous system has long been a priority for physicians, patients and caregivers seeking to address the developmental delays and other neurological symptoms experienced by many patients with MPS syndromes."

As previously announced, ArmaGen entered into a worldwide licensing and collaboration agreement with Shire plc valued at $225 million to develop AGT-182 for the treatment of both the central nervous system (CNS) and somatic (body-related) manifestations of Hunter syndrome. ArmaGen is responsible for conducting the Phase 1 study of AGT-182. Under the terms of the agreement, ArmaGen will receive R&D funding, development and sales milestones, and future royalties from Shire.