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 Phase 2b clinical trial (NCT04079803) was sponsored by Cassava in collaboration with the National Institute on Aging to further test PTI-125's safety, tolerability, and efficacy. This trial enrolled 64 people between the ages of 50 and 85. All participants had mild-to-moderate Alzheimer's disease, as evidenced by middling scores on the Mini-Mental State Exam (MMSE), a tool used to measure cognitive function. Participants were randomized to one of two doses of PTI-125 (50 or 100 mg) or a placebo, taken orally twice daily for 28 days. Its primary measure of efficacy was changes in the levels of Alzheimer's-related biomarkers in patients' cerebrospinal fluid (CSF) — the fluid that surrounds the brain and spinal cord. Biomarker levels were measured by labs independent of Cassava. Relative to placebo, PTI-125 treatment did not result in statistically significant changes
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