Gut microbiota DNA sequencing analysis of Alzheimer’s disease patients

July 22, 2019

Anavex Life Sciences Corp., a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental diseases including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) diseases, reported results from a study evaluating the gut microbiota of patients in the ongoing ANAVEX2-73 Phase 2a extension study of ANAVEX2-73, a selective sigma-1 receptor agonist.

“This is the first gut microbiota DNA sequencing analysis of Alzheimer’s disease patients treated with ANAVEX2-73, which might help to identify potential biomarkers of response for ANAVEX2-73,” said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex. “We continue to advance the ANAVEX2-73 Alzheimer’s disease program through the ongoing Phase 2b/3 Alzheimer’s disease clinical study and in other ongoing ANAVEX2-73 clinical trials focused on serious neurology diseases with high unmet needs, including Rett syndrome and Parkinson’s disease dementia.”

The analysis was conducted using Ariana Pharma’s KEM Artificial Intelligence, an FDA-tested technology to systematically explore combinations of biomarkers. Results revealed that patients treated with ANAVEX2-73 had high levels of two gut microbiota families, Ruminococcaceae and Porphyromonadaceae, which were associated with improved activities of daily living (ADCS-ADL) at week 148 (p<0.01 and p<0.04, respectively).

Communication between gut microbiota and the brain has been shown to be a critical requirement of a healthy brain function. The reduction in gut microbiota diversity has become one of the hallmarks of aging, and disturbances in its composition are associated with several age-related neurological conditions, including Alzheimer’s disease. These changes in the gut microbiota composition induce increased permeability of the gut barrier and immune activation leading to systemic inflammation, which in turn may impair the blood-brain barrier and promote neuroinflammation, neural injury, and ultimately neurodegeneration.

Numerous pre-clinical studies demonstrate beneficial effects of SIGMAR1 (S1R) agonists on neuroinflammation, including with ANAVEX2-73. The effect might potentially be reversal of the microbiota imbalances and might have a homeostatic effect on the brain-gut-microbiota axis.

The oral presentation at AAIC 2019, titled, Exploring gut microbiota as a source of potential biomarkers: Initial results from the ANAVEX2-73 Alzheimer’s disease clinical study [O4-02-04]  will be presented by the lead author of the study, Mohammad Afshar, MD, PhD, CEO of Ariana Pharma, and will be available at

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