Cassava Sciences’ lead drug candidate, PTI-125, has been found to significantly decrease key biomarkers of neuroinflammation and neurodegeneration in Alzheimer’s.
The company reported top-line results, saying that the “new treatment could be an important part of the research dialogue in Alzheimer’s disease.”
The Phase IIa study achieved a 100% responder rate, with all patients responding to PTI-125. A key objective of this first-in-patient study was to measure drug effects on biomarkers in the brain (i.e., in cerebrospinal fluid, or CSF) before and after 28 days of treatment with PTI-125.
Dr. Jeffrey Cummings, research professor of the department of Brain Health, UNLV went on to say that “This drug candidate appears to target some of the more toxic components of the illness. Results will need to be replicated in larger studies to prove it’s a definitive advance in the field.”
The study consisted of patients with mild-to-moderate Alzheimer’s disease, age 50-85, who received 100 mg oral PTI-125 twice daily for 28 days.
Alzheimer's is a complex disease and one of the largest challenges facing healthcare today, with lots of failed treatments in past pipelines.
Earlier this year Biogen and Eisai stopped two global Phase III trials of the Alzheimer's drug aducanumab, after interim analyses indicated that the agent was ineffective and would not meet the primary endpoint.
Novartis, Amgen and Banner Alzheimer's Institute also recently decided to discontinue investigation of the BACE1 inhibitor CNP520 (umibecestat), citing that potential benefit for participants in the studies did not outweigh the risk.
Over 850,000 people in the UK are currently living with dementia. The condition is caused by brain diseases, most commonly Alzheimer’s, which cause brain cells to die and impair the brain’s ability to function. With few treatments available, and none that can tackle the course of the underlying disease, Alzheimer’s and other dementias are now the country’s leading cause of death.