The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending the granting of a marketing authorisation under exceptional circumstances for NEZGLYAL (leriglitazone), Minoryx’s lead program, as treatment for of Cerebral Adrenoleukodystrophy (cALD) in males with Adrenoleukodystrophy (ALD) aged 2 to 12 years with non-gadolinium (Gd) enhancing lesions (i.e. Gd negative) in brain Magnetic Resonance Imaging (MRI), with a Neurological Functional Score (NFS) of 0 or 1.

Leriglitazone is a selective, differentiated PPAR gamma agonist, which has shown preclinical proof-of-concept in animal models of multiple CNS diseases by modulating pathways leading to neuroinflammation, demyelination, mitochondrial dysfunction, oxidative stress, and axonal degeneration. In clinical trials, 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. More than 170 patients with cALD have received treatment to date.. Additionally, a phase 3 study in adult male patients with progressive cALD (CALYX) is currently ongoing. Leriglitazone offers a strong potential for indication expansion into other CNS diseases. In this regard, 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 is also being investigated for other orphan CNS diseases.