simufilam

Simufilam is a proprietary, oral small molecule drug developed for the treatment of Alzheimer's disease. Its mechanism of action centers on binding to an altered form of filamin A (FLNA), a scaffolding protein in the brain that is abnormally configured in Alzheimer's disease. By restoring FLNA’s normal shape and function, simufilam disrupts aberrant linkages between FLNA and several receptors implicated in Alzheimer’s pathology—including the α7 nicotinic acetylcholine receptor (α7nAChR) and inflammatory receptors such as Toll-like receptor 4 (TLR4), TLR2, CXCR4, CCR5, and CD4. This disruption blocks toxic signaling from soluble amyloid beta 1-42 via α7nAChR that leads to tau hyperphosphorylation—a key event in neurodegeneration—and reduces neuroinflammation by modulating inflammatory pathways. Simufilam has also been shown to normalize overactive mTOR signaling and improve insulin receptor function in Alzheimer's models[1][3][5][6][8]. The drug does not target amyloid clearance but instead aims to stabilize critical protein interactions upstream of tau aggregation and inflammation. Simufilam is currently being evaluated in Phase III clinical trials for Alzheimer’s disease[1][5].

Brand names

simufilam

Other names

1224591-33-6