NV354

NV354 is an orally bioavailable small molecule succinate prodrug in development for the treatment of primary mitochondrial diseases with severe neurological symptoms, such as Leigh syndrome. It is designed to restore cellular energy production by delivering succinate directly to mitochondria and modulating mitochondrial oxidative phosphorylation. Preclinical studies have shown that NV354 can reach the brain and improve mitochondrial function in models of Leigh syndrome. The drug has been granted orphan drug designation by the FDA for mitochondrial disease and is being advanced towards clinical trials. The developer is Abliva (formerly NeuroVive Pharmaceutical), which was acquired by Pharming Technologies B.V.

Other names

Methyl 4-{[2-(acetamino)ethyl]sulfanyl}-4-oxobutanoateButanoic acid, 4-[[2-(acetylamino)ethyl]thio]-4-oxo-, methyl ester