Simufilamum, also known as PTI-125, is a filamin A altered conformations binder. PTI-125 is an oral small molecule drug candidate that binds and reverses an altered conformation of the scaffolding protein filamin A found in Alzheimer's disease brain. PTI-125 binds and reverses an altered conformation of filamin A to reduce Alzheimer's disease pathogenesis. PTI-125 also reduced tau hyperphosphorylation, aggregated Aβ42 deposition, neurofibrillary tangles, and neuroinflammation. Efficacy in postmortem AD and Aβ42-treated age-matched control hippocampal slices was concentration-dependent starting at 1 picomolar (pM) concentration. PTI-125 is the first therapeutic candidate to preferentially bind an altered protein conformation and reverse this proteopathy.
Cat.No
DC74974
Name
Simufilamum
Chemical Properties
CAS
1224591-33-6
Formula
C15H21N3O
MW
259.35
Storage
2 years -20°C Powder, 2 weeks 4°C in DMSO, 6 months -80°C in DMSO
References
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