PTI-125 Reduces Neurodegeneration After 28 Days of Treatment, Data Show

PTI-125 Reduces Neurodegeneration After 28 Days of Treatment, Data Show
PTI-125, Cassava Sciences’ lead therapeutic candidate for the treatment of Alzheimer’s disease, significantly reduced the levels of disease biomarkers, brain inflammation, and neurodegeneration after 28 days of treatment, top-line data from a Phase 2 trial show. "Based on these encouraging biomarker results, this new treatment could be an important part of the research dialogue in Alzheimer’s disease," Jeffrey Cummings, research professor of the Department of Brain Health at the University of Nevada, Las Vegas and director of the Center for Neurodegeneration and Translational Neuroscience of the Cleveland Clinic Lou Ruvo Center for Brain Health, said in a press release. "This drug candidate appears to target some of the more toxic components of the illness. Results will need to be replicated in larger studies to prove it’s a definitive advance in the field," Cummings said. PTI-125 is an investigational therapy that has been designed to bind to an abnormal form of a protein called Filamin A (FLNA) to restore its normal structure and function. FLNA is misfolded in patients with Alzheimer’s disease and is thought to contribute to the buildup of toxic plaques inside nerve cells. Its effects in patients with mild to moderate Alzheimer’s disease have been evaluated in this open-label, Phase 2a trial (NCT03748706) sponsored by the National Institutes of Health (NIH). The study enrolled 13 patients who received 100 mg of PTI-125 orally twice a day for 28 days. The main goal of the trial was to evaluate the effects of PTI-125 on the levels of several disease biomarkers in patients’ cerebrospinal fluid (CSF, the liquid that circulates in the brain and spinal cord) before and after treatment. Top-line data from the trial has shown that: All patien
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