simufilam

Simufilam dihydrochloride (PTI-125 dihydrochloride) is a low toxicity, orally active filamin A (FLNA) activator, which can be used for the research of Alzheimer's disease. Simufilam dihydrochloride preferentially binds altered FLNA and restores its native conformation, restoring receptor and synaptic activities and reducing its α7nAChR TLR4 associations and downstream pathologies.

Simufilam (PTI-125) (hydrochloride) is an orally active filamin A (FLNA) activator with low toxicity that can be used in the Alzheimer's disease research. Simufilam (hydrochloride) preferentially binds to altered FLNA and restores its native conformation, restores receptor and synaptic activities, and reduces its a7nAChR TLR4 associations and downstream pathologies [1].

Simufilamum (also known as PTI-125) is an oral small-molecule compound candidate that targets altered conformations of filamin A. It binds and reverts the misfolded filamin A in the brains of Alzheimer's disease patients, thereby slowing the disease's pathological progression. Additionally, PTI-125 reduces tau protein hyperphosphorylation, decreases Aβ42 aggregation and deposition, neurofibrillary tangles, and neuroinflammation. The compound demonstrates concentration-dependent efficacy in vitro, starting at 1 picomolar (pM), in both control groups and post-mortem hippocampal slices from Alzheimer's disease (AD) patients. PTI-125 is the first therapeutic candidate to preferentially bind and reverse pathogenic protein conformations.

Simufilam (PTI-125) is a low toxicity, orally active filamin A ( FLNA ) activator. Simufilam preferentially binds altered FLNA and restores its native conformation, restores receptor and synaptic activities, reduces its a7nAChR/TLR4 associations and downstream pathologies. Simufilam can be used in Alzheimer's disease research[1].