US-based biopharmaceutical firm Cassava Sciences has started a Phase IIb clinical trial to assess PTI-125 for the treatment of Alzheimer’s disease.

PTI-125 is an investigational small molecule developed to target the neuroinflammation and neurodegeneration that occurs in the brain of Alzheimer’s patients.

It acts on the modified form of a scaffolding protein called filamin A (FLNA), which was found to hinder the normal neuronal functions and cause Alzheimer’s pathology, neurodegeneration and neuroinflammation.

The drug candidate exhibits an anti-neuroinflammatory effect and restores FLNA’s normal shape and function.

In the Phase IIb study, PTI-125’s safety, tolerability and effects will be investigated using validated biomarkers of the neurodegenerative disease.

It will involve around 60 patients with mild-to-moderate Alzheimer’s.

The randomised, placebo-controlled, multi-centre, multi-dose trial will treat participants with twice daily PTI-125 100mg, 50mg or placebo over 28 days. Cassava Sciences noted that the initial two subjects in the trial have been dosed.

The primary endpoint of the trial is the improvement in Alzheimer’s biomarkers from baseline to day 28.

The Phase IIb study is supported by a National Institutes of Health (NIH) research grant and comes after positive findings in a prior Phase IIa trial.

Phase IIa data showed a 100% responder rate and a statistically significant reduction in important biomarkers of the disease pathology, neuroinflammation and neurodegeneration.

Cassava Sciences president and CEO Remi Barbier said: “There is a profound and timely need to develop new drug therapies for this devastating disease.

“We are pleased to advance PTI-125 into this randomised, placebo-controlled study in approximately 60 Alzheimer’s patients, and are eager to report how results of this study will expand upon the clinical data set for PTI-125.”

The company is also working on an investigational blood test for Alzheimer’s under a programme named PTI-125Dx.