Simufilam dihydrochloride  (Synonyms: PTI-125 dihydrochloride)

Simufilam dihydrochloride (PTI-125 dihydrochloride) is an orally active FLNA modulator. Simufilam dihydrochloride restores NMDAR signaling and Arc expression. Simufilam dihydrochloride inhibits overactive mTOR signaling by restoring the normal conformation of FLNA, improves insulin sensitivity, reduces Aβ₄₂-induced neuroinflammation and tau protein hyperphosphorylation. Simufilam dihydrochloride can be used for research of Alzheimer's disease^[1][2][3].

Simufilam (1 nM；1 h) dihydrochloride 可减少死后人类 AD 脑切片中 FLNA-α7nAChR/TLR4 结合，解离 Aβ₄₂-α7nAChR 复合物，恢复 α7nAChR/NMDAR/IR 功能，并降低 tau 磷酸化^[2]。
Simufilam (1 pM-1 nM; 1 h) dihydrochloride 剂量依赖性地降低死后人类 AD 海马切片中的 FLNA-α7nAChR/TLR4 结合、Aβ₄₂-α7nAChR 复合物、tau 磷酸化和 tau 硝化，同时恢复 NMDAR 信号传导和 Arc 表达^[3]。

MCE has not independently confirmed the accuracy of these methods. They are for reference only.

Simufilam (10 mg/kg；腹腔注射；每日一次；2 周) dihydrochloride 可减少 ICV Aβ₄₂ 输注小鼠 FLNA-α7nAChR/TLR4 关联、tau 磷酸化、Aβ₄₂ 聚集和炎症细胞因子，同时恢复α7nAChR/NMDAR/IR 功能^[2]。
Simufilam dihydrochloride (30 mg/kg/d；口服；2 个月) 可减少 3xTg AD 小鼠 FLNA-α7nAChR/TLR4 关联、tau 磷酸化、Aβ₄₂ 沉积和 NFT，改善 NMDAR/IR 功能和突触可塑性，并增强空间/工作记忆和筑巢行为^[3]。

MCE has not independently confirmed the accuracy of these methods. They are for reference only.

332.27

C₁₅H₂₃Cl₂N₃O

2480226-06-8

Room temperature in continental US; may vary elsewhere.

Please store the product under the recommended conditions in the Certificate of Analysis.

• [1]. Wang HY, et al. Simufilam suppresses overactive mTOR and restores its sensitivity to insulin in Alzheimer's disease patient lymphocytes. Front Aging. 2023 Jun 29;4:1175601.  [Content Brief]

  [2]. Wang HY, et al. Reducing amyloid-related Alzheimer's disease pathogenesis by a small molecule targeting filamin A. J Neurosci. 2012 Jul 18;32(29):9773-84.  [Content Brief]

  [3]. Wang HY, et al. PTI-125 binds and reverses an altered conformation of filamin A to reduce Alzheimer's disease pathogenesis. Neurobiol Aging. 2017 Jul;55:99-114.  [Content Brief]