Largest prospective efficacy study conducted in the orphan disease AMN
Leriglitazone significantly reduces progression of cerebral lesions and myelopathy symptoms
Minoryx preparing for discussions with regulators for approval of leriglitazone for AMN patients
Mataró, Barcelona, Spain, January 26, 2021 - Minoryx Therapeutics, a Phase 3 clinical stage biotech company focused on the development of differentiating treatment options in orphan central nervous system (CNS) disorders, today announces topline results from its Phase 2/3 ADVANCE clinical trial. The study evaluated leriglitazone, a novel, selective PPARγ agonist, in male patients with adrenomyeloneuropathy (AMN), a neurodegenerative disease causing progressive spastic paraparesis and autonomic nervous system dysfunction. Additionally, AMN patients are at risk of developing progressive cerebral lesions, a devastating form of the disease leading to rapid severe disability and fatal if left untreated.