How does PTI-125 work?
Proper folding of a protein is vital for it to work as intended. Alzheimer’s disease is a complex brain disorder thought to be caused by the incorrect folding of several proteins. These misfolded proteins clump together in the brain, creating plaques that impede brain function and lead to inflammation, degeneration, and atrophy in nerve cells.
Filamin A is one of the misfolded proteins found in Alzheimer’s disease. Filamin A is a scaffolding protein, a type of protein that helps the proper folding of other proteins. Correctly functioning filamin A is essential for the proper folding and working of amyloid-beta and tau, two proteins that play a crucial role in the development of Alzheimer’s. In its altered form, filamin A prevents the proper folding of the amyloid-beta and tau proteins, causing them to aggregate and form plaques and tangles in the brain. Misfolded filamin A also disrupts how nerve cells work, leading to the neurodegeneration observed in people with Alzheimer’s disease.
PTI-125 is an investigational small molecule that targets the misfolded filamin A protein. It binds to this filamin A and aims to restore its function, potentially alleviating the symptoms of Alzheimer’s disease.
PTI-125 in clinical trials
This study is enrolling 60 patients, ages 50 to 85, at eight U.S. sites. Participants will be randomly given either 50 mg or 100 mg oral tablets of PTI-125, or a placebo, twice a day. After 28 days of treatment or placebo, a cerebrospinal fluid (the fluid surrounding the brain and spinal cord) sample will be assessed for biomarkers of Alzheimer’s disease. Researchers will also evaluate markers of neurodegeneration and neuroinflammation in all patient groups. Secondary study goals are blood levels of disease biomarkers, and evidence of changes in learning skills and memory.
Two people have begun receiving treatment, Cassava reported in September 2019.
A previous Phase 2a open-label clinical trial (NCT03748706) investigated the safety and pharmacokinetics of PTI-125 in a similar group of mild-to-moderate Alzheimer’s patients. Study results showed that treatment, 28 days of PTI-125 at 100 mg twice daily, was well-tolerated, and these 13 patients had markedly lower disease-specific biomarkers of neuroinflammation and neurodegeneration.
PTI-125 was also shown to be safe and well-tolerated in a Phase 1 clinical trial (NCT03784300) that evaluated its safety and pharmacokinetics (movement in the body) in healthy volunteers.
Last updated: Oct. 08, 2019
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