Minoryx’s lead program is a selective, differentiated PPAR gamma agonist (leriglitazone) under development for the treatment of X-linked Adrenoleukodystrophy (X-ALD); it has the potential to treat both the severe cerebral form of ALD (cALD) and the chronic form, adrenomyeloneuropathy (AMN), as well as other orphan CNS diseases, such as Friedreich's Ataxia (FRDA) and Rett's disease..

Leriglitazone demonstrated robust preclinical proof-of-concept in relevant animal models of disease and successfully completed phase 1 clinical trials. Leriglitazone completed a phase 2/3 clinical study in adult patients with X-ALD (ADVANCE) in the EU and US showing a significant reduction of cerebral lesion progression and a reduction of incidence of progressive cALD and myelopathy symptoms. Additionally, a separate study in paediatric patients with cALD (NEXUS) is currently ongoing in EU and after 24 weeks of treatment, all evaluable patients in NEXUS were clinically stable and radiologically demonstrated disease arrest or lesion growth stabilization. Finally, a phase 3 study in adult male patients with progressive cALD (CALYX) is currently recruiting in the US. The marketing authorization application (MAA) for adult male X-ALD patients is currently under review by the EMA. 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. Leriglitazone is also being investigated for other orphan CNS diseases.