PTI-125 Fails to Lower Alzheimer’s Protein Levels in Phase 2 Trial

PTI-125 Fails to Lower Alzheimer’s Protein Levels in Phase 2 Trial
Cassava Sciences's investigational therapy PTI-125 failed to significantly lower the levels of Alzheimer's-relevant biomarkers present in fluid surrounding the brain and spinal cord of patients, according to results from a Phase 2b clinical trial. The company announced it will study these topline data in deciding next steps for PTI-125 as a potential treatment. Alzheimer's disease is characterized by the formation of aggregates (clumps) of misfolded proteins in the brain. PTI-125 is an investigational small molecule designed to reduce protein misfolding in the brain, easing Alzheimer's symptoms. A previous Phase 1 clinical trial (NCT03784300) in healthy volunteers showed the treatment to be safe and well-tolerated. Preliminary efficacy data from a small Phase 2a clinical trial (NCT03748706) suggested it could lower levels of disease markers of brain damage and inflammation. A larger
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