Minoryx is a clinical stage biotech company focused on the discovery and development of novel therapies for severe, orphan genetic diseases of the central nervous system (CNS) with high unmet medical need.

The company’s lead program is a novel, selective and differentiated PPAR gamma agonist (leriglitazone), which is currently in clinical development in multiple CNS diseases. Leriglitazone has demonstrated strong preclinical proof-of-concept in relevant animal models of disease and has successfully completed phase 1 clinical trials. Leriglitazone is currently in a registration enabling study in adrenomyeloneuropathy (AMN) in EU and US, a registration enabling study in cerebral Adrenoleukodystrophy (cALD) in EU and a proof of concept study in Friedreich Ataxia (FRDA).

Leriglitazone has been granted Orphan Drug Designation for X-ALD and for FRDA by both the European Commission (EC) and the US Food and Drug Administration (FDA),  as well as, Fast Track from the US FDA. In addition, leriglitazone has been designated as Rare Pediatric Disease Designation by FDA for X-ALD and FRDA.

The Company is also investigating the potential of leriglitazone in other CNS diseases.