Rare Disease Report

Successful Phase 2 Results in Fast Track Designation for Potential Hurler Therapy

NOVEMBER 30, 2017
Mathew Shanley
Initial results from an ongoing Phase 2 proof-of-concept (POC) study have led to the U.S. Food and Drug Administration (FDA) granted Fast Track designation to AGT-181, a novel, investigational enzyme replacement therapy for the treatment of symptoms in patients with mucopolysaccharidosis type I (MPS I; Hurler Syndrome).

Results from the study were presented in February at the 13th Annual WORLDSymposium in San Diego, and suggested that AGT-181, developed by ArmaGen, Inc. improves neurocognitive function in patients with Hurler syndrome. The development exhibits the capability of the company’s proprietary drug delivery technology to transport biopharmaceuticals across the blood-brain barrier (BBB). The study has since been fully enrolled and final results will be presented at the 14th Annual WORLDSymposium in 2018.

The Fast Track designation will allow for expedited review of the potential therapy.

Hurler syndrome arises from a mutation in the iduronidase (IDUA) enzyme, which is necessary for the breakdown of complex sugars. Without a properly functioning IDUA enzyme, large sugar molecules called glycosaminoglycans (GAGs) accumulate within the lysosomes, causing different organs and tissues within the body to become enlarged.

AGT-181 targets the insulin receptor and takes advantage of the body’s natural system for transporting proteins and other large molecules non-invasively across the BBB, in this case, by binding the same receptor that transports insulin across the BBB into the brain. This process is intended to alleviate the neurological complications associated with Hurler syndrome.

"FDA's decision to grant Fast Track designation will allow us to interact with the Agency in the most efficient manner in order to advance AGT-181 into a pivotal trial and future registration," said Mathias Schmidt, Ph.D., chief executive officer of ArmaGen in a press release. "AGT-181 has the potential to provide patients with MPS I a treatment option that addresses the unmet cognitive disease burden facing these patients."

At present, there are no approved cures for Hurler syndrome, but bone marrow transplant and enzyme replacement therapy have been proven to help manage the symptoms of the condition.

ArmaGen is also currently conducting a Phase 1 Trial to evaluate the safety and determine a well-tolerated dose of its investigational therapy AGT-182 in male patients ages 18 years and older with MPS II, or Hunter syndrome.

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