Impact:

PTI-125 is a clinical-stage small molecule drug candidate to treat Alzheimer’s disease derived from collaborative work with Pain Therapeutics, Inc. (Austin, Texas). PTI-125 has a novel mechanism of action: it binds an altered shape of the scaffolding protein and restores its normal shape. Altered scaffolding protein links to the brain receptors, enabling aberrant signaling by the Alzheimer’s disease pathological factor, amyloid-β through these receptors. By restoring the native shape of the scaffolding protein and blocking these toxic cascades, PTI-125 drastically reduced Alzheimer’s disease brain pathologies, and improved neuronal plasticity and function of the brain receptors. These effects translate to cognitive improvements in Alzheimer’s disease mouse model and suggest the possibility of disease modification in humans. Based on PTI-125’s underlying science, a blood-based diagnostic for Alzheimer’s disease, PTI-125Dx, is also under development.