molecules of the month


oral PPARg agonist for CNS diseases

Ph. II and III ongoing for AMN and cALD

one of the metabolites of pioglitazone

Science Translational Medicine

Minoryx Therapeutics S.L., Barcelona, ES

1 min read

14. The Minoryx peroxisome proliferator-activated receptor gamma (PPARg) agonist, leriglitazone, is one of the several metabolites of pioglitazone, a member of the thiazolidinedione (glitazone) drug class for diabetes. It is instead being developed for a rare peroxisomal neurodegenerative disease, X-linked adrenoleukodystrophy (X-ALD). It completed a Ph. I clinical trial (135 and 270 mg/day, 8 days) showing good safety, tolerability and CNS engagement of PPAR gamma receptors at levels equivalent to those required for efficacy in preclinical models, and a Ph. II/III study is currently ongoing to elucidate whether leriglitazone can preventing or delaying the advancement of adrenomyeloneuropathy (AMN) into cALD (childhood cerebral adrenoleukodystrophy). It is an interesting example of rationally repurposing a species of an established drug class for an…

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