| Description | 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. |
| In vitro | Simufilam dihydrochloride (1 pM~1 nM)以剂量依赖的方式增加NMDA/甘氨酸诱导的Arc表达。Simufilam dihydrochloride (1 nM, 1小时)大大恢复了filamin A到其原生构象，并有效地正常化了AD前额皮层中的受体活性。Simufilam dihydrochloride 以剂量依赖的方式降低了Ab42诱导的FLNA与a7nAChR及TLR4的耦合，在Ab42处理的对照组和AD海马体中均有此现象。Simufilam dihydrochloride 降低了Ab42对a7nAChR的结合亲和力，从而阻止了Ab42的信号传导和在a7nAChRs上的进一步积累。 |
| In vivo | Simufilam dihydrochloride（22 mg/kg；口服）显著减少FLNAea7nAChR/TLR4的关联。Simufilam dihydrochloride显著改善空间记忆力。 |
| Synonyms | PTI-125 dihydrochloride |
| molecular weight | 332.3 |
| Molecular formula | C15H23Cl2N3O |
| CAS | 2480226-06-8 |
| Storage | Powder: -20°C for 3 years | In solvent: -80°C for 1 year | Shipping with blue ice. |
| Solubility | DMSO: 65 mg/ml (195.61 mM) |
| References | 1. Wang HY, et al. PTI-125 binds and reverses an altered conformation of filamin A to reduce Alzheimer's disease pathogenesis. Neurobiol Aging. 2017;55:99-114. |