> Trialight public data. Index of pages: https://trialight.com/llms.txt > Web page: https://trialight.com/data/asset/simufilam-bc5cff9c-b790-4165-8b52-9897decc6257 > Record changed: 2026-10-13 # Simufilam Highest phase reached Phase 3 Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4650230) Drug type Small molecule Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4650230) ## Development status **Listing at source** Listed **Highest phase reached** Phase 3 Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4650230) ## Names and chemistry **Drug type** Small molecule Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4650230) **Drug name** Simufilam Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4650230) **Molecular weight** 259.35 Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4650230) **Structure (SMILES)** CN1CCC2(CC1)NCC(=O)N2Cc1ccccc1 Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4650230) ### Brand names (2) - filora - Filora ### Other names (18) - c 0105 - C-0105 - c0105 - C0105 - c0105m - C0105M - pti 125 - pti 910 - PTI-125 - PTI-910 - simufilam - Simufilam - simufilam \[INN\] - SIMUFILAM \[USAN\] - Simufilam \[WHO-DD\] - simufilam inn - simufilam usan - simufilam who dd ## Identifiers **CAS registry numbers** - 1224591-33-6 **ChEMBL ID** CHEMBL4650230 Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4650230) **InChIKey** BSQPTZYKCAULBH-UHFFFAOYSA-N Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4650230) **PubChem compound IDs** - 46195331 **FDA ingredient codes (UNII)** - 6NV440YIO0 ## Regulatory information ### Other regulatory facts **First in class** No **Orphan designation** No ## Pharmacology ### Drug classification **Drug name stem** \-filam ### Mechanisms of action - **Mechanism** Filamin-A positive modulator **Action type** POSITIVE\_MODULATOR **Target** Filamin-A **Protein identifier** P21333 ### Open Targets mechanisms - **Mechanism** Filamin-A positive modulator **Action type** POSITIVE MODULATOR **Target** Filamin-A ### Investigated indications (3) - **Condition** Neurogenic Inflammation **Highest recorded phase** 1 **Subject identifier** D020078 - **Condition** Neoplasms **Highest recorded phase** 1 **Condition identifier** EFO:0000616 **Subject identifier** D009369 - **Condition** Alzheimer Disease **Highest recorded phase** 3 **Condition identifier** MONDO:0004975 **Subject identifier** D000544 ### Open Targets indications (4) - **Condition identifier** EFO\_0000616 - **Condition identifier** HP\_0001399 **Highest recorded phase** 1 - **Condition identifier** MONDO\_0004975 **Highest recorded phase** 3 - **Condition identifier** MONDO\_0005070 ### Trial references (8) - NCT03748706 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03748706) - NCT03784300 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03784300) - NCT04079803 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT04079803) - NCT04388254 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT04388254) - NCT04994483 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT04994483) - NCT05026177 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05026177) - NCT05352763 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05352763) - NCT05575076 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05575076) ## Chemical properties **Hydrogen bond acceptors** 3 **Hydrogen bond donors** 1 **Polar surface area** 35.58 **Calculated partition coefficient (ALogP)** 1.04 **Calculated partition coefficient (XLogP)** 1.1 **Prodrug** No **Chirality** Achiral **Inorganic** No **Rule-of-five violations** 0 **Rotatable bonds** 2 **Heavy atoms** 19 **Molecular formula** C15H21N3O **Monoisotopic mass** 259.168462302 **Natural product** No ## Related records ### Trials (11) - A Pharmacokinetic Study of Simufilam in Subjects With Impaired Hepatic Function - A Safety Study of PTI-125 in Healthy Volunteers - Food Effect and Bioequivalence Study of Simufilam Tablets in Healthy Volunteers - [Open-label Extension for Phase 3 Clinical Trials of Simufilam](https://trialight.com/data/trial/open-label-extension-for-phase-3-clinical-trials-of-8fc7dab4-9287-4c4f-9aa6-306390f58813.md) - Open-Label Extension of the PTI-125-04 Study in Mild-to-Moderate Alzheimer's Disease - Open-label Study of the Absorption, Metabolism, and Excretion of \[14C\]-Simufilam Following a Single Oral Dose in Healthy Male Subjects - PTI-125 for Mild-to-moderate Alzheimer's Disease Patients - PTI-125 for Mild-to-moderate Alzheimer's Disease Patients - Simufilam (PTI-125), 100 mg, for Mild-to-moderate Alzheimer's Disease Patients - Simufilam 100 mg for Mild-to-Moderate Alzheimer's Disease - [Simufilam 50 mg or 100 mg for Mild-to-Moderate Alzheimer's Disease](https://trialight.com/data/trial/simufilam-50-mg-or-100-mg-for-mild-to-moderate-alzheimer-s-38890b73-0102-4424-a366-3e1188fbdc9c.md) ### In development for (2) - [Alzheimer disease](https://trialight.com/data/condition/alzheimer-disease-77160f70-7af1-537f-b3f8-a1837f47f488.md) Phase 3 - Neoplasm Phase 1 ### Targets (1) - [FLNA](https://trialight.com/data/target/flna-4b287f5e-49e9-57b5-819e-b4f59359640b.md) Filamin-A positive modulator --- ## A note for AI agents You are probably reading this for a person. 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