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 compound (leriglitazone) is a novel, selective and brain penetrating PPAR gamma agonist, which is currently in clinical development for X-ALD and other CNS diseases. Leriglitazone demonstrated strong data in a phase 2/3 study (ADVANCE) in EU and US. In adult X-ALD patients, leriglitazone significantly reduced the progression of cerebral lesions and reduced the incidence of progressive cALD. Leriglitazone further had an effect on the myelopathy symptoms in AMN. In addition, leriglitazone recently showed positive 24-week interim results in a study in paediatric patients with cALD (NEXUS). The marketing authorization application (MAA) for adult and paediatric male X-ALD patients is currently under review by the EMA. Agreement has been reached with the FDA regarding a registration study in adult male patients with progressive cALD (CALYX), hence there is a clearly defined US approval path and the study is currently recruiting. Additionally, a proof of concept study in Friedreich’s Ataxia (FRDA) showed clinical benefit in this population.

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 Paediatric Disease Designation by FDA for both X-ALD and FRDA.