Thinly traded nano cap Cassava Sciences (NASDAQ:SAVA) is up 41% premarket on increased volume in reaction to new data from a Phase 2a clinical trial evaluating lead drug PTI-125 in patients with mild-to-moderate Alzheimer’s disease (AD). The results were presented at the Clinical Trials on Alzheimer’s Disease Conference in San Diego.
Consistent improvements in AD biomarkers in plasma and lymphocytes were noted including evidence that each patient showed biomarker responses to PTI-125.
Significant reductions in both nitrated and phosphorylated forms of tau protein were observed.
Early clinical validation of the drug target, altered filamin A, as a facilitator protein between amyloid beta and both neuroinflammation and tau pathology.
Topline data are expected in 2020.
Small molecule PTI-125 targets an altered form of filamin A, a scaffolding protein found throughout the body. A highly toxic form of the protein is present in the brains of AD sufferers which disrupts the normal function of neurons, leading to neurodegeneration and brain inflammation. PTI-125 is designed to restore the normal shape of filamin A in the brain, improving the function of multiple brain receptors and dampening neuroinflammation.
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