On 15 May, the biopharmaceutical company Cassava Sciences, developing novel solutions to detect and treat neurodegenerative diseases such as Alzheimer’s disease (AD) reported top-line results from its Phase II study of PTI-125 in AD. PTI-125 is a small molecule drug targeting the altered form of filamin A found in the AD brain to restore its normal shape and function.
Top-line results showed that the study did not meet its primary endpoint, which was a statistically significant effect of PTI-125 on cerebrospinal fluid (CSF) levels of tau protein and other biomarker assessments from baseline to Day 28. The drug was safe, well-tolerated and significantly reduced a secondary endpoint, CSF levels of IL1-beta, a core biomarker of neuroinflammation, from baseline to Day 28. The company reported that analyses of biomarker data revealed high variability in levels of CSF biomarkers over 28 days.
The Phase IIa clinical study was a double-blind, randomised and placebo-controlled study evaluating PTI-125 in people with mild-to-moderate AD. A total of 64 participants (50-85 years old) received PTI-125 or placebo twice a day for 28 days.
