Sumifilam Lowers Levels of Multiple Biomarkers of Disease Activity, Trial Shows

Sumifilam Lowers Levels of Multiple Biomarkers of Disease Activity, Trial Shows
Treatment with sumifilam significantly lowered the levels of multiple biomarkers of disease activity, neurodegeneration, and inflammation in people with mild-to-moderate Alzheimer’s after 28 days of treatment, according to final data from a Phase 2b trial. Sumifilam, Cassava Sciences’ lead investigational candidate for Alzheimer’s disease, also was found to be safe and well-tolerated, and to improve patients’ cognitive function, compared with a placebo. “Other than a few drugs to help ease the decline, there’s really nothing out there to treat people with Alzheimer’s,” Remi Barbier, chairman, president and CEO of Cassava Sciences, said in a press release. “The improvement on multiple biomarkers in this clinical study is a first and offers hope that sumifilam has potential to become a transformative treatment for people with Alzheimer’s disease,” Barbier said. Sumifilam, formerly known as PTI-125, is a proprietary small oral molecule that has been designed to bind and restore the normal shape and function of filamin A (FLNA), a protein that is misfolded in patients with Alzheimer’s and is thought to contribute to the accumulation of toxic protein clumps (aggregates) inside nerve cells. Data from a previous open-label Phase 2a study (NCT03748706) had shown that, when given at a dose of 100 mg twice per day, sumifilam was able to lower the levels of several disease biomarkers in 13 patients with mild-to-moderate Alzheimer’s within a period of 28 days. These promising findings led the company to launch a larger, double-blind, placebo-controlled Phase 2b trial (NCT04079803) to confirm the positive effects of sumifilam on Alzheimer’s disease biomarkers. Funded by the National Institutes of Health (NIH), the study enrolled
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