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 chronic form, adrenomyeloneuropathy (AMN), as well as other orphan CNS diseases, such as Friedreich's Ataxia (FRDA).

Leriglitazone was evaluated in a double-blind, placebo-controlled phase 2/3 clinical study (ADVANCE) in adult AMN patients in EU and US, overall showing a significant effect on reducing progression of cerebral lesions and progression of myelopathy symptoms. An open-label extension is ongoing to generate long term safety and efficacy data, and currently, Minoryx is under discussions with regulatory authorities on the approval path for AMN population. A separate study in pediatric cALD patients is currently ongoing in EU (NEXUS) to further assess the impact of leriglitazone on the progression of cerebral lesions.

Leriglitazone is also being investigated for other orphan CNS diseases, and a proof of concept study in patients with FRDA (FRAMES) was completed showing clinical benefit in this population. The design of the confirmatory phase 3 study in this population is now under discussion with regulatory authorities.