On 9 September, the biopharmaceutical company Cassava Sciences, developing novel solutions to detect and treat neurodegenerative diseases such as Alzheimer’s disease (AD) announced positive results of its lead drug candidate PTI-125. It is a small molecule drug targeting the altered form of filamin A found in the AD brain to restore its normal shape and function.
The Phase IIa clinical study was an open-label and multicentre trial evaluating the safety, pharmacokinetics and effect on biomarkers of PTI-125 in people with mild-to-moderate AD. The study was conducted with support from the National Institutes of Health (NIH). A total of 13 US participants (50-85 years old) received 100 mg oral tablets of PTI-125 twice a day for 28 days.
The company reported that all participants responded to the experimental drug, achieving thus a 100% responder rate. PTI-125 has been found to significantly decrease key biomarkers of AD pathology, neuroinflammation and neurodegeneration. The total Tau and the neurogranin, a marker for cognitive decline, decreased significantly by 20% and 32% respectively in participants.
It is anticipated that data will be presented at upcoming meetings, such as the Clinical Trials on Alzheimer’s disease (CTAD) conference (San Diego, December 2019). Furthermore, the company plans to initiate a Phase IIb study of PTI-125. Approximately 60 participants with mild-to-moderate AD will receive PTI-125 (50 or 100 mg) or placebo twice daily for 28 days.
