Minoryx is a registration-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), a novel, selective and brain penetrating PPAR gamma agonist, has received a positive CHMP opinion to become the first pharmacological treatment for male cALD patients, ages 2-12years with Gadolinium (Gd) negative brain lesions.. It has shown clinical benefit in both paediatric cALD patients in the NEXUS clinical trial and adult cALD patients in the ADVANCE trial. Results from NEXUS demonstrate that paediatric cALD patients are clinically and radiologically stable after over 96 weeks of treatment or at a visit prior to Haematopoietic Stem Cell Transplantation (HSCT). Data from ADVANCE showed that leriglitazone reduced cALD progression. It is expected that the European Commission will grant formal Marketing Authorization approval by end September 2026. In the US, and agreement was 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 ongoing. Additionally, a proof of concept study in Friedreich’s Ataxia (FRDA) showed clinical benefit in this population, and by the end of 2026 we will obtain read-out from our phase 2 trial (TREE) in Rett syindrome.

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.