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news.US-based biotechnology firm ArmaGen has received acceptance from the US Food and Drug Administration (FDA) for an investigational new drug application (IND) for its second product candidate AGT-181, an investigational enzyme replacement therapy (ERT), to treat Hurler syndrome.
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The company intends to begin a Phase I trial in the second quarter of 2015 to evaluate the safety and tolerability of AGT-181 in adult patients with mucopolysaccharidosis type I (MPS I).
Hurler syndrome, the most severe form of MPS I, is a rare, hereditary lysosomal storage disorder that affects the brain and spinal cord in children, resulting in a wide range of debilitating symptoms.
The trial’s primary objective is to evaluate the safety and determine a well-tolerated dose of AGT-181 in adult patients with Hurler-Scheie and Scheie, which are less severe forms of MPS I.
A total of nine patients 18 years and older will be enrolled in the trial, and they will be treated with AGT-181 for a total of eight weeks.
AGT-181 is designed to use the body’s natural system for transporting products non-invasively across the blood-brain barrier (BBB) by targeting the receptor that delivers insulin to all cells of the body.
ArmaGen vice president of Clinical Affairs Steven Schoenfeld said: "The FDA’s acceptance of the IND allows us to begin activating clinical sites in the US and enrolling patients into the study.
"We are looking forward to working hand-in-hand with physicians and the patient community to conduct the Phase I study of AGT-181, with the goal of eventually providing a treatment option for currently unaddressed neurological complications of severe Hurler syndrome."
After securing results from this Phase I trial, the company intends to conduct subsequent studies in a broader population of patients with MPS I, including those with Hurler syndrome.
ArmaGen chief executive officer James Callaway said: "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.
"This development marks a significant milestone for ArmaGen and our pipeline of therapies for severe neurological disorders."