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Cortexyme, pressing forward after its novel approach to Alzheimer’s disease failed in a mid-stage trial, believes a lower dose of the medicine might safely treat a subset of patients. But detailed data, presented at a medical conference Thursday, suggest the path ahead could be difficult.

In the roughly 650-patient study, Cortexyme’s drug proved no better than placebo at slowing patients’ decline, as measured by tests of cognition and ability to complete basic activities. However, the failed trial taught the company how to succeed next time, said Michael Detke, Cortexyme’s chief medical officer, at the Clinical Trials on Alzheimer’s Disease meeting in Boston.


The plan now is to start a second study, this time enrolling only the Alzheimer’s patients most likely to benefit and treating them with a lower dose of Cortexyme’s drug, an effort to minimize some of the worrying side effects seen in the prior trial. The first trial failed, but “by following the data we were able to find the right population and the right dose to move forward,” Detke said.

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