First Alzheimer’s Patients Dosed with PTI-125 in New Phase 2b Clinical Trial

First Alzheimer’s Patients Dosed with PTI-125 in New Phase 2b Clinical Trial
The first two patients with Alzheimer's disease have been dosed with Cassava Sciences’ investigational small molecule PTI-125 in a new Phase 2b clinical study. PTI-125 is a small molecule that has been designed to bind to an abnormal form of a protein called Filamin A (or FLNA), and restore its normal structure and activity. FLNA is found misfolded in the brains of many patients with Alzheimer’s disease, and is believed to be an important contributor to neuroinflammation and the buildup of toxic plaque inside nerve cells. Supported by the National Institutes of Health (NIH), the trial (NCT04079803) will evaluate the safety, tolerability, and therapeutic activity of PTI-125, as well as its effect on known disease biomarkers, in approximately 60 people with mild-to-moderate Alzheimer’s disease. Recruitment is ongoing in the United States, and participants will be randomly assigned to oral administration of 50 mg or 100 mg of PTI-125, or a dose-matching placebo, twice daily for 28 consecutive days. For more information about study locations and contacts, click here. “There is a profound and timely need to develop new therapies for this devastating disease,” Remi Barbier, president and CEO of Cassava Sciences, said in a news release. “We are pleased to adva
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