30 November 2017 - ArmaGen today reported that the U.S. FDA has granted fast track designation to AGT-181, a novel, investigational enzyme replacement therapy for the treatment of both somatic and cognitive symptoms in patients with Hurler syndrome (also known as mucopolysaccharidosis type I, or MPS I).
The initial results from an ongoing Phase 2 proof-of-concept study were presented in February 2017 at the 13th Annual WORLDSymposium in San Diego, California. The data suggested that AGT-181 improves neurocognitive function in patients with MPS I, demonstrating the ability of ArmaGen’s proprietary drug delivery technology to transport biopharmaceuticals across the blood-brain barrier. The study has since fully enrolled and final results will be presented at the 14th Annual WORLDSymposium in San Diego, California in February 2018.