Simufilam

Simufilam
Clinical data
Other names	PTI-125, PTI-910
ATC code	None;
Pharmacokinetic data
Elimination half-life	4.5 hours
Identifiers
	IUPAC name 1-benzyl-8-methyl-1,4,8-triazaspiro(4.5)decan-2-one;
CAS Number	1224591-33-6;
PubChem CID	46195331;
UNII	6NV440YIO0;
KEGG	D12003;
ChEMBL	ChEMBL4650230;
Chemical and physical data
Formula	C15H21N3O
Molar mass	259.353 g·mol−1
3D model (JSmol)	Interactive image;
	SMILES CN1CCC2(CC1)NCC(=O)N2Cc1ccccc1;
	InChI 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; Key:BSQPTZYKCAULBH-UHFFFAOYSA-N;

Simufilam (PTI-125) is an experimental medication for the treatment of Alzheimer's disease. It was being developed by the American pharmaceutical firm Cassava Sciences. Development of simufilam was discontinued in November 2024 after it failed to show clinical benefit during phase III clinical trials.

The US Food and Drug Administration (FDA) received a citizen petition in August 2021 to stop the clinical trials and investigate Cassava Sciences. Scientists questioned the preclinical results, citing the small sample size, alleged methodological flaws in an in vitro technique, alleged manipulations of western blot images and potential conflict of interest.

After the FDA said that the citizen petition was the improper procedure to request an investigation, Reuters reported in July 2022 that a criminal investigation of Cassava Sciences was started by the United States Department of Justice (DOJ) over research results related to the experimental drug. The U.S. Securities and Exchange Commission (SEC), the U.S. National Institutes of Health (NIH), and City University of New York (CUNY) were also investigating whether Cassava or individuals manipulated data. In June 2024, Hoau-Yan Wang was charged by the DOJ with fraud for falsifying data on $16 million in NIH grant applications related to simufilam.