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.

Our lead program is a novel, selective and differentiated brain penetrant PPAR gamma agonist (leriglitazone), which is currently in clinical development for X-ALD and other CNS diseases. Leriglitazone demonstrated strong preclinical proof-of-concept in relevant animal models of disease and successfully completed phase 1 clinical trials. Leriglitazone was evaluated in a phase 2/3 study in adrenomyeloneuropathy (AMN) in EU and US with an open-label extension still ongoing. Leriglitazone significantly reduced the progression of cerebral lesions and myelopathy symptoms. The marketing authorization application (MAA) for adult male X-ALD patients is under review by the EMA and the launch preparations in the EU are underway. Also, discussions are currently ongoing with the FDA to define the next steps for its US approval path. In parallel, a separate study is ongoing on pediatric cALD patients in EU.

Additionally, a proof of concept study in Friedreich’s Ataxia (FRDA) showed clinical benefit in this population and a phase 3 confirmatory study is under discussion with the  EMA and FDA.

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 for X-ALD from the US FDA. In addition, leriglitazone has been designated as Rare Pediatric Disease Designation by FDA for both X-ALD and FRDA.