PTI-125 is an experimental therapy being developed by Cassava Sciences to treat Alzheimer’s disease, a progressive and neurodegenerative condition.

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

An ongoing and recruiting Phase 2b clinical trial (NCT04079803) is evaluating the safety, tolerability, and effectiveness of two doses of PTI-125 in people with mild-to-moderate Alzheimer’s disease.

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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Alzheimer’s News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified healthcare providers with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

Vijaya Iyer is a freelance science writer for BioNews Services. She has contributed content to their several disease-specific websites, including cystic fibrosis, multiple sclerosis, muscular dystrophy, among others. She holds a PhD in Microbiology from Kansas State University, where her research focused on molecular biology, bacterial interactions, metabolism, and animal models to study bacterial infections. Following the completion of her PhD, Dr. Iyer went on to complete three postdoctoral fellowships at Kansas State University, University of Miami and Temple University. She joined BioNews Services to utilize her scientific background and writing skills to help patients and caregivers remain abreast with important scientific breakthroughs.
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Özge has a MSc. in Molecular Genetics from the University of Leicester and a PhD in Developmental Biology from Queen Mary University of London. She worked as a Post-doctoral Research Associate at the University of Leicester for six years in the field of Behavioural Neurology before moving into science communication. She worked as the Research Communication Officer at a London based charity for almost two years.
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Vijaya Iyer is a freelance science writer for BioNews Services. She has contributed content to their several disease-specific websites, including cystic fibrosis, multiple sclerosis, muscular dystrophy, among others. She holds a PhD in Microbiology from Kansas State University, where her research focused on molecular biology, bacterial interactions, metabolism, and animal models to study bacterial infections. Following the completion of her PhD, Dr. Iyer went on to complete three postdoctoral fellowships at Kansas State University, University of Miami and Temple University. She joined BioNews Services to utilize her scientific background and writing skills to help patients and caregivers remain abreast with important scientific breakthroughs.
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