Simufilam

Summary

Simufilam (CHEMBL4650230) is a phase-3 clinical-stage small molecule; indicated across 3 conditions including alzheimer disease and neoplasm.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Indications: 3 conditions
• Clinical trials: 8
• Chemistry: 259.35 Da · C15H21N3O

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4650230
Name	Simufilam
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	46195331
Molecular formula	C15H21N3O
Molecular weight	259.35
InChIKey	BSQPTZYKCAULBH-UHFFFAOYSA-N

SMILES: CN1CCC2(CC1)NCC(=O)N2CC3=CC=CC=C3

IUPAC name: 4-benzyl-8-methyl-1,4,8-triazaspiro[4.5]decan-3-one

Also known as: C-0105, C0105, C0105M, Filora, PTI-125, PTI-910, Simufilam, SIMUFILAM

Parent form; salt/anhydrous children: CHEMBL4650231

Patent coverage: 50 distinct patent families (133 SureChEMBL compound mentions), from 1 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

No target linkage available.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

3 indications (0 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
Alzheimer disease	3	MONDO:0004975	MONDO:0004975
neoplasm	1	MONDO:0005070	EFO:0000616

1 further indication record had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 8.

Phase distribution

Phase	Trials
PHASE1	4
PHASE3	3
PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT04994483	PHASE3	COMPLETED	Simufilam 100 mg for Mild-to-Moderate Alzheimer’s Disease
NCT05026177	PHASE3	TERMINATED	Simufilam 50 mg or 100 mg for Mild-to-Moderate Alzheimer’s Disease
NCT05575076	PHASE3	TERMINATED	Open-label Extension for Phase 3 Clinical Trials of Simufilam
NCT05352763	PHASE2	TERMINATED	Open-Label Extension of the PTI-125-04 Study in Mild-to-Moderate Alzheimer’s Disease
NCT03784300	PHASE1	COMPLETED	A Safety Study of PTI-125 in Healthy Volunteers
NCT04932655	PHASE1	COMPLETED	Food Effect and Bioequivalence Study of Simufilam Tablets in Healthy Volunteers
NCT06195319	PHASE1	COMPLETED	Open-label Study of the Absorption, Metabolism, and Excretion of [14C]-Simufilam Following a Single Oral Dose in Healthy Male Subjects
NCT06390410	PHASE1	COMPLETED	A Pharmacokinetic Study of Simufilam in Subjects With Impaired Hepatic Function

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).