Our science is based on stabilizing a critical protein in the brain.
PTI-125 is an investigational drug that represents an entirely new approach to treat Alzheimer’s disease.
PTI-125 improves the function of multiple brain receptors and exerts powerful anti-neuroinflammatory effects.
In 2018, we initiated a Phase II clinical program in patients with Alzheimer’s disease using PTI-125 backed by research grant awards from NIH.
Over the past ten years, we have conducted basic research to understand the underlying causes of neurodegeneration.
We are now in clinical studies to test a new and promising scientific approach for the treatment and diagnosis of 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.
Our lead therapeutic product candidate, called PTI-125, is a small molecule drug with a novel mechanism of action. The target of PTI-125 is an altered form of filamin A (FLNA). FLNA is a scaffolding protein found throughout the body. The function of a scaffolding protein is to bring multiple proteins together and to ensure they interact properly. However, an altered and highly toxic form of FLNA is found in the Alzheimer's brain. Altered FLNA disrupts the normal function of neurons, leading to neurodegeneration and brain inflammation.
Our investigational drug candidate, PTI-125, restores the normal shape of FLNA in the brain. This improves the function of multiple brain receptors and exerts powerful anti-neuroinflammatory effects.
In animals models of disease, chronic treatment with PTI-125 results in dramatic improvements in brain health, such as improved insulin receptor signaling; improved learning and memory; significant reductions in levels of inflammatory cytokines in the brain; and reduced amyloid and tau deposits. Importantly, since PTI-125 has a unique mechanism of action, we believe its potential therapeutic effects may be additive or synergistic with those of other therapeutic candidates against neurodegeneration.