FDA Accepts ArmaGen’s IND for its Hurler Syndrome Candidate

Privately-held biotech ArmaGen, Inc. announced today that US health regulators have accepted for review its investigational New Drug (IND) application for its drug for a rare lysosomal storage disease.

The company, focused on developing novel therapies to treat severe neurological disorders, said that the US Food and Drug Administration (FDA) has accepted for review its IND for AGT-181 for the treatment of Hurler syndrome. This is the company’s second product candidate.

AGT-181 is an investigational enzyme replacement therapy (ERT) for the treatment of Hurler syndrome. Hurler syndrome is the most severe form of mucopolysaccharidosis type I (MPS I). It is a rare, hereditary lysosomal storage disorder that affects the brain and spinal cord in children, resulting in a wide range of debilitating symptoms. Currently, commercially available treatments for the condition do not penetrate the blood-brain barrier (BBB), and therefore do not address the severe and progressive neurological complications of the disease. ArmaGen’s AGT-181 is designed to use the body’s natural system for transporting products non-invasively across the BBB by targeting the receptor that delivers insulin to all cells of the body.

“The FDA’s acceptance of the IND allows us to begin activating clinical sites in the U.S. and enrolling patients into the study,” said Steven L. Shoenfeld, MD, Vice President of Clinical Affairs at ArmaGen. “We are looking forward to working hand-in-hand with physicians and the patient community to conduct the Phase 1 study of AGT-181, with the goal of eventually providing a treatment option for currently unaddressed neurological complications of severe Hurler syndrome.”

ArmaGen said that it expects to begin a Phase I trial evaluating AGT-181 in adult patients with MPS I in the second quarter of 2015. The primary objective of the trial is to test the safety and determine a well-tolerated dose of AGT-181 in adult patients with Hurler-Sheie and Scheie, which are less severe forms of MPS I. The company plans to enroll nine adult patients into the study for a total of eight weeks. All study participants will receive AGT-181.

“This is our second investigational product to enter clinical trials for the treatment of lysosomal storage disease, and we are proud to be independently advancing a potential therapy for patients with Hurler syndrome that may offer a potentially important alternative to experimental treatments such as stem cell transplantation,” said James Callaway, PhD, Chief Executive Officer of ArmaGen. “This development marks a significant milestone for ArmaGen and our pipeline of therapies for severe neurological disorders.”

In December, ArmaGen announced that the FDA accepted its IND for AGT-182 for the treatment of Hunter syndrome. The company is developing AGT-182 in partnership with Shire as part of a worldwide licensing and collaboration agreement.

Source: ArmaGen, Inc.

Last updated: 4/8/15; 10:10am EST


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