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 pivotal phase 2/3 clinical trial (ADVANCE) in adult AMN patients and a phase 2 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 phase 2 study in patients with cerebral Adrenoleukodystrophy (cALD) has started.