Simufilam, 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.
MedKoo Cat#: 414720
Name: Simufilam free base
CAS#: 1224591-33-6 (free base)
Chemical Formula: C15H21N3O
Exact Mass: 259.1685
Molecular Weight: 259.35
Elemental Analysis: C, 69.47; H, 8.16; N, 16.20; O, 6.17
The following data is based on the product molecular weight 259.35 Batch specific molecular weights may vary from batch to batch due to the degree of hydration, which will affect the solvent volumes required to prepare stock solutions.
Concentration / Solvent Volume / Mass | 1 mg | 5 mg | 10 mg |
---|---|---|---|
1 mM | 1.15 mL | 5.76 mL | 11.51 mL |
5 mM | 0.23 mL | 1.15 mL | 2.3 mL |
10 mM | 0.12 mL | 0.58 mL | 1.15 mL |
50 mM | 0.02 mL | 0.12 mL | 0.23 mL |