leriglitazone

Leriglitazone is a novel, orally bioavailable, blood-brain-barrier penetrant small molecule that acts as a selective peroxisome proliferator-activated receptor gamma (PPARγ) agonist. It is the active metabolite (M4) of pioglitazone and has been specifically developed for central nervous system (CNS) diseases. Leriglitazone modulates gene expression involved in mitochondrial biogenesis and function via activation of the PPARγ/PGC-1α pathway. This leads to restoration of mitochondrial function and energy production, reduction in oxidative stress and neuroinflammation, protection against demyelination and axonal degeneration, increased neuronal survival, improved myelination through oligodendrocyte differentiation/survival, and overall improvement in motor function. The drug is being developed primarily for X-linked adrenoleukodystrophy (X-ALD), including adrenomyeloneuropathy (AMN) and cerebral adrenoleukodystrophy (cALD), as well as Friedreich’s ataxia. Leriglitazone has demonstrated efficacy in preclinical models for other CNS diseases such as Rett syndrome and NBIA disorders[1][2][5][6][7].

Brand names

Nezglyal

Other names

1-hydroxy pioglitazoneleriglitazone hydrochlorideM4 of pioglitazoneM-4 of pioglitazoneM 4 of pioglitazone