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Zagociguat is an orally administered, CNS-penetrant small molecule that acts as a stimulator (agonist) of soluble guanylate cyclase (sGC). By amplifying nitric oxide–cyclic guanosine monophosphate (NO–cGMP) signaling in the central nervous system, it is designed to improve mitochondrial function, cerebral blood flow, and cognitive performance. Zagociguat has received orphan drug designation from the FDA for mitochondrial diseases and is being investigated primarily for MELAS syndrome (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes), as well as Alzheimer's disease. The drug was originally developed by Ironwood Pharmaceuticals and further developed by Cyclerion Therapeutics and Tisento Therapeutics[2][4][5][6].
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