Simufilamum Dihydrochloride Monohydrate - CAS 2375909-85-4

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.

2375820

C15H25Cl2N3O2

350.28

O=C1N(C2(CCN(CC2)C)NC1)CC3=CC=CC=C3.Cl.Cl.O

-20°C

Ice Packs

Small Compound