Minoryx Therapeutics

Minoryx is a clinical-stage biotechnology company focused on the discovery and development of new therapies for serious orphan genetic diseases of the central nervous system (CNS) with high unmet medical needs. Our lead program is a novel, selective, and differentiated PPARγ agonist (leriglitazone), currently in clinical development for X-ALD and other CNS disorders. Leriglitazone has demonstrated robust preclinical proof-of-concept in relevant animal models of disease and successfully completed a Phase 1 clinical trial. Leriglitazone has been evaluated in a Phase 2/3 study in patients with neurologic disease validated in the EU and the US, with an ongoing open-label extension. Leriglitazone significantly reduced the progression of brain neurodegeneration and myelopathy symptoms and is currently under regulatory discussion for the approval pathway for AMN patients. Additionally, a separate study in pediatric CALD patients in the EU is ongoing.