| Size | Price | Stock | Qty |
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| 5mg |
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| 10mg |
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| Other Sizes |
| Targets |
Filamin A (FLNA); NMDA receptor (NMDAR); α7 nicotinic acetylcholine receptor (α7 nAChR). Simufilam is an orally active modulator of filamin A (FLNA) that restores NMDA receptor signaling and Arc expression. It interacts with filamin and controls the actin cytoskeleton. By targeting altered filamin A, simufilam prevents and reverses the binding of Aβ42 to α7 nAChR, thereby reducing amyloid and tau formation.
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| ln Vitro |
Simufilam is a potent, orally active small molecule against Alzheimer's disease that interacts with filamin and controls the actin cytoskeleton. It prevents and reverses the binding of Aβ42 to α7 nAChR, reducing amyloid and tau formation. Its in vitro activities include restoration of NMDA receptor signaling and Arc expression.
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| ln Vivo |
In vivo, simufilam is an orally active modulator of filamin A. It has been evaluated in preclinical models of Alzheimer's disease for its ability to restore NMDA receptor signaling, reduce amyloid and tau formation, and improve cognitive function. The compound has shown low toxicity in preclinical studies.
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| Enzyme Assay |
Binding of simufilam to filamin A can be assessed using biophysical methods such as surface plasmon resonance (SPR) or isothermal titration calorimetry (ITC). Effects on NMDA receptor signaling and Arc expression are assessed in cell-based or biochemical assays. Standard analytical methods including HPLC, NMR, and mass spectrometry are used for compound characterization.
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| Cell Assay |
Cell-based assays for simufilam use neuronal cell lines to assess its effects on filamin A conformation, NMDA receptor signaling, Arc expression, and protection against Aβ42 toxicity. The compound's ability to prevent and reverse Aβ42 binding to α7 nAChR is assessed in cell culture models.
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| Animal Protocol |
In vivo studies of simufilam are conducted in transgenic mouse models of Alzheimer's disease. The compound is administered orally. Cognitive function is assessed using behavioral tests (e.g., Morris water maze). Brain tissue is collected for analysis of amyloid plaques, tau pathology, and synaptic markers. Pharmacokinetic parameters are determined from blood samples.
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| ADME/Pharmacokinetics |
Simufilam has a molecular formula of C₁₅H₂₁N₃O and a molecular weight of 259.35 g/mol. It is an orally active small molecule. The compound should be stored under recommended conditions. It is a novel drug candidate for Alzheimer's disease and is intended for research use only.
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| Toxicity/Toxicokinetics |
Simufilam has shown low toxicity in preclinical studies. Standard laboratory safety practices should be followed when handling this compound.
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| Additional Infomation |
An oral small molecule drug candidate that binds to and reverses the conformational changes of the scaffold protein filament protein A in the brain of Alzheimer's disease.
See also: Zonampanel (related). Simufilam (PTI-125) is a novel small-molecule drug candidate developed to treat Alzheimer's disease by targeting altered filamin A (FLNA). It is an orally active modulator of filamin A that restores NMDA receptor signaling and Arc expression. By preventing and reversing the binding of Aβ42 to α7 nAChR, simufilam reduces amyloid and tau formation. It is for research use only. |
| Molecular Formula |
C15H21N3O
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|---|---|
| Molecular Weight |
259.346743345261
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| Exact Mass |
259.168
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| CAS # |
1224591-33-6
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| Related CAS # |
Simufilam hydrochloride;2480226-07-9;Simufilam dihydrochloride;2480226-06-8
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| PubChem CID |
46195331
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| Appearance |
White to off-white solid powder
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| LogP |
1.1
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| Hydrogen Bond Donor Count |
1
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| Hydrogen Bond Acceptor Count |
3
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| Rotatable Bond Count |
2
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| Heavy Atom Count |
19
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| Complexity |
330
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| Defined Atom Stereocenter Count |
0
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| SMILES |
CN1CCC2(CC1)NCC(=O)N2CC3=CC=CC=C3
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| InChi Key |
BSQPTZYKCAULBH-UHFFFAOYSA-N
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| InChi Code |
InChI=1S/C15H21N3O/c1-17-9-7-15(8-10-17)16-11-14(19)18(15)12-13-5-3-2-4-6-13/h2-6,16H,7-12H2,1H3
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| Chemical Name |
4-benzyl-8-methyl-1,4,8-triazaspiro[4.5]decan-3-one
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| HS Tariff Code |
2934.99.9001
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| Storage |
Powder -20°C 3 years 4°C 2 years In solvent -80°C 6 months -20°C 1 month |
| Shipping Condition |
Room temperature (This product is stable at ambient temperature for a few days during ordinary shipping and time spent in Customs)
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| Solubility (In Vitro) |
May dissolve in DMSO (in most cases), if not, try other solvents such as H2O, Ethanol, or DMF with a minute amount of products to avoid loss of samples
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| Solubility (In Vivo) |
Note: Listed below are some common formulations that may be used to formulate products with low water solubility (e.g. < 1 mg/mL), you may test these formulations using a minute amount of products to avoid loss of samples.
Injection Formulations
Injection Formulation 1: DMSO : Tween 80： Saline = 10 : 5 : 85 (i.e. 100 μL DMSO stock solution → 50 μL Tween 80 → 850 μL Saline)(e.g. IP/IV/IM/SC) *Preparation of saline: Dissolve 0.9 g of sodium chloride in 100 mL ddH ₂ O to obtain a clear solution. Injection Formulation 2: DMSO : PEG300 ：Tween 80 : Saline = 10 : 40 : 5 : 45 (i.e. 100 μL DMSO → 400 μLPEG300 → 50 μL Tween 80 → 450 μL Saline) Injection Formulation 3: DMSO : Corn oil = 10 : 90 (i.e. 100 μL DMSO → 900 μL Corn oil) Example: Take the Injection Formulation 3 (DMSO : Corn oil = 10 : 90) as an example, if 1 mL of 2.5 mg/mL working solution is to be prepared, you can take 100 μL 25 mg/mL DMSO stock solution and add to 900 μL corn oil, mix well to obtain a clear or suspension solution (2.5 mg/mL, ready for use in animals). View More
Injection Formulation 4: DMSO : 20% SBE-β-CD in saline = 10 : 90 [i.e. 100 μL DMSO → 900 μL (20% SBE-β-CD in saline)] Oral Formulations
Oral Formulation 1: Suspend in 0.5% CMC Na (carboxymethylcellulose sodium) Oral Formulation 2: Suspend in 0.5% Carboxymethyl cellulose Example: Take the Oral Formulation 1 (Suspend in 0.5% CMC Na) as an example, if 100 mL of 2.5 mg/mL working solution is to be prepared, you can first prepare 0.5% CMC Na solution by measuring 0.5 g CMC Na and dissolve it in 100 mL ddH2O to obtain a clear solution; then add 250 mg of the product to 100 mL 0.5% CMC Na solution, to make the suspension solution (2.5 mg/mL, ready for use in animals). View More
Oral Formulation 3: Dissolved in PEG400 (Please use freshly prepared in vivo formulations for optimal results.) |
| Preparing Stock Solutions | 1 mg | 5 mg | 10 mg | |
| 1 mM | 3.8558 mL | 19.2790 mL | 38.5579 mL | |
| 5 mM | 0.7712 mL | 3.8558 mL | 7.7116 mL | |
| 10 mM | 0.3856 mL | 1.9279 mL | 3.8558 mL |
*Note: Please select an appropriate solvent for the preparation of stock solution based on your experiment needs. For most products, DMSO can be used for preparing stock solutions (e.g. 5 mM, 10 mM, or 20 mM concentration); some products with high aqueous solubility may be dissolved in water directly. Solubility information is available at the above Solubility Data section. Once the stock solution is prepared, aliquot it to routine usage volumes and store at -20°C or -80°C. Avoid repeated freeze and thaw cycles.
Calculation results
Working concentration： mg/mL；
Method for preparing DMSO stock solution： mg drug pre-dissolved in μL DMSO (stock solution concentration mg/mL). Please contact us first if the concentration exceeds the DMSO solubility of the batch of drug.
Method for preparing in vivo formulation:：Take μL DMSO stock solution, next add μL PEG300, mix and clarify, next addμL Tween 80, mix and clarify, next add μL ddH2O，mix and clarify.
(1) Please be sure that the solution is clear before the addition of next solvent. Dissolution methods like vortex, ultrasound or warming and heat may be used to aid dissolving.
(2) Be sure to add the solvent(s) in order.
Link: https://clinicaltrials.gov/ct2/show/NCT05026177
Conditions:Alzheimer DiseaseLink: https://clinicaltrials.gov/ct2/show/NCT05352763
Conditions:Alzheimer's DiseaseLink: https://clinicaltrials.gov/ct2/show/NCT04994483
Conditions:Alzheimer Disease
Title:Open-label Extension for Phase 3 Clinical Trials of Simufilam
Status:Terminated
updateDate:2025-05-23
Ctid:NCT05575076
Link: https://clinicaltrials.gov/ct2/show/NCT05575076
Conditions:Alzheimer DiseaseLink: https://clinicaltrials.gov/ct2/show/NCT04388254
Conditions:Alzheimer DiseaseLink: https://clinicaltrials.gov/ct2/show/NCT06195319
Conditions:Absorption|Metabolism|ExcretionLink: https://clinicaltrials.gov/ct2/show/NCT06390410
Conditions:Hepatic InsufficiencyLink: https://clinicaltrials.gov/ct2/show/NCT04932655
Conditions:Healthy VolunteersLink: https://clinicaltrials.gov/ct2/show/NCT04079803
Conditions:Alzheimer DiseaseLink: https://clinicaltrials.gov/ct2/show/NCT03748706
Conditions:Alzheimer DiseaseLink: https://clinicaltrials.gov/ct2/show/NCT03784300
Conditions:Alzheimer Disease, Early Onset|Alzheimer DiseaseProducts are for research use only; We do not sell to patients
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