MIN-102 (leriglitazone) is a novel, orally bioavailable and selective PPAR gamma agonist with a superior profile for CNS related diseases. It is a metabolite of pioglitazone which shows a superior brain penetration and safety profile, allowing PPAR gamma engagement in the CNS above the level that can be safely achieved with pioglitazone and other glitazones. It showed robust preclinical proof of concept in several animal models.

In X-ALD, mutations on ABCD1 trigger a cascade of events leading to mitochondrial dysfunction, oxidative stress, neuroinflammation, demyelination and axonal degeneration. MIN-102, through its PPAR gamma activity, prevents such dysfunctions, thus it has the potential to treat both adrenomyeloneuropathy (AMN) and cerebral ALD (cALD). Thanks to its capacity to modulate several pathways central for neurodegeneration, MIN-102 also showed promising efficacy in models of other CNS diseases such as Friedreich’s Ataxia.

It has successfully completed a phase 1 clinical trial with excellent results showing that contrary to other PPAR gamma agonists, MIN-102 is able to reach the CNS and modulate PPAR gamma at equivalent levels than those required for efficacy in preclinical models. A pivotal phase 2/3 trial in AMN and a phase 2 in FRDA are currently ongoing and new clinical studies on other orphan CNS diseases are under preparation.