Minoryx’s lead program is a selective, differentiated PPAR gamma agonist (leriglitazone) being developed 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 milder form, adrenomyeloneuropathy (AMN), as well as other orphan CNS diseases, such as Friedreich's Ataxia (FRDA).

Leriglitazone is currently being evaluated in a registration enabling clinical trial (ADVANCE) in adult AMN patients in EU and US and a proof of concept trial (FRAMES) in patients with FRDA. Recruitment for both studies has been completed and results for both studies are expected by end 2020. In addition, recruitment of the registration enabling study in patients with cerebral Adrenoleukodystrophy (cALD) in EU has started.