MIN-102 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).
It has successfully completed a phase 1 clinical trial with excellent results. A pivotal phase 2/3 trial in adult AMN patients is currently ongoing and new clinical studies on other orphan CNS diseases are under preparation.