Our science is based on stabilizing a critical protein in the brain.
Simufilam (formerly known as PTI-125) is an investigational drug that represents an entirely new approach to treat Alzheimer’s disease.
Importantly, we do not seek to clear amyloid out of the brain. Our science is based on stabilizing a critical protein in the brain.
Simufilam is a proprietary, small molecule (oral) drug that restores the normal shape and function of altered filamin A (FLNA), a scaffolding protein, in the brain.
The underlying science for simufilam is published in peer-reviewed journals, including Journal of Neuroscience, Neurobiology of Aging, Journal of Biological Chemistry, Neuroimmunology and Neuroinflammation and Journal of Prevention of Alzheimer’s Disease.
We are now in Phase III clinical studies to test a new and promising scientific approach for the treatment and diagnosis of Alzheimer's disease. If you are interested in being notified about clinical trials, click here.
Simufilam was developed in-house. The early development of our lead drug candidate, simufilam, was substantially funded by peer-review research grant awards from the National Institutes of Health (NIH).