Biotechnology company Cassava Sciences has concluded enrolment and started dosing the last patient in a Phase IIb clinical trial of PTI-125 to treat Alzheimer’s disease.

The randomised, double-blind, placebo-controlled trial recruited 64 participants with mild to moderate Alzheimer’s in the US.

PTI-125 is a small molecule designed to act on the modified form of a scaffolding protein called filamin A (FLNA). In the brain, the altered protein negatively impacts neuron functions and results in the development of Alzheimer’s pathology, neuroinflammation, and neurodegeneration.

The drug candidate works to re-establish FLNA’s normal shape and function in the brain. In turn, the mechanism improves the function of some receptors, mitigates neurodegeneration, and has anti-neuroinflammatory effects.

During the Phase IIb study, a 50mg and 100mg twice-a-day dose of PTI-125 will be compared to placebo over 28 days. The trial aims to assess the drug candidate’s safety and effectiveness on disease biomarkers.

The primary endpoint of the trial is the improvement in disease biomarkers from baseline to day 28, while secondary outcomes include cognitive assessment using three different measures.

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By GlobalData

The trial is being conducted by the company with the National Institutes of Health (NIH)’s scientific and financial support. Cassava commenced the study in September last year.

Cassava Sciences president and CEO Remi Barbier said: “Our previously announced results of a Phase IIa study with PTI-125 generated promising biomarker data in Alzheimer’s patients. Based on those positive results, we believe our ongoing Phase IIb study is a critical point of clinical validation for PTI-125.”

The company is also working on PTI-125Dx, an investigational diagnostic to identify Alzheimer’s using a blood test.