Date: 2015-09-02
Type of information: Treatment of the first patient
phase: 1
Announcement: treatment of the first patient
Company: Armagen (USA - CA)
Product: AGT-181 (fusion protein of anti-human insulin receptor monoclonal antibody and alpha-L-iduronidase; HIRMAb-IDUA)
Action
mechanism: enzyme replacement therapy/fusion protein. AGT-181 is a novel, investigational enzyme replacement therapy (ERT) for the treatment of neurological complications in patients with Hurler syndrome. AGT-181 is a fusion protein containing alpha-L-Iduronidase that is intended to deliver the enzyme peripherally and to the brain, when administered intravenously. The most severe form of MPS I, Hurler syndrome is a rare, hereditary lysosomal storage disorder that affects the brain and spinal cord in children, resulting in a wide range of debilitating symptoms. Commercially available treatments for Hurler syndrome do not penetrate the blood-brain barrier (BBB), and therefore do not address the severe and progressive neurological complications of the disease. AGT-181 is designed to utilize 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 fusion protein binds to insulin receptors located on the surface of the BBB, enabling its passage into the brain.
Disease: Hurler syndrome or mucopolysaccharidosis type I (MPS I)
Therapeutic area: Rare diseases - Genetic diseases
Country: USA
Trial
details: This study is a safety and dose ranging study to obtain safety and exposure data, as well as information on the biological activity of the investigational drug. (NCT02371226)
Latest
news: * On September 2, 2015, ArmaGen, a privately held biotechnology company focused on developing revolutionary therapies to treat severe neurological disorders, announced that the first patient has been dosed in its multi-center Phase 1/2a clinical trial of AGT-181 in patients with attenuated mucopolysaccharidosis type I (MPS I).Pending the results of the Phase 1/2a trial, ArmaGen plans to conduct subsequent studies in a pediatric population of patients with MPS I, including those with Hurler syndrome. AGT-181 is Armagen's second investigational product to enter the clinic in 2015. * On April 8, 2015, ArmaGen announced that the Investigational New Drug Application (IND) for the company’s second product candidate, AGT-181 for the treatment of Hurler syndrome, has been accepted by the FDA and is now active. ArmaGen expects to initiate a Phase 1 clinical trial in the second quarter of 2015 to assess the safety and tolerability of AGT-181 in adult patients with mucopolysaccharidosis type I (MPS I). The primary objective of the Phase 1 trial is to test 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. ArmaGen plans to enroll nine patients 18 years and older into the study. These patients will be treated for a total of eight weeks, with all enrolled patients receiving AGT-181. Pending the results of the Phase 1 trial, ArmaGen plans to conduct subsequent studies in a broader population of patients with MPS I, including those with Hurler syndrome.
