Anavex Life Sciences, New York City, a clinical-stage biopharmaceutical company, has announced the publication of a study using artificial intelligence for the identification and confirmation of genomic biomarkers of response to an Alzheimer’s disease therapy.1

“This study highlights the relevance of phenotypic and genotypic precision medicine analyses of whole-exome sequencing and gene expression data in drug development, and in particular the potential to identify patients’ genetic variants and gene expression changes that may predict increased chances of success of Alzheimer’s disease treatments,” says Harald Hampel, MD, PhD, founding president of the Alzheimer Precision Medicine Initiative and lead author of the paper.

Alzheimer’s disease is a progressive, multifactorial disease and the most common cause of primary neurodegenerative dementias. In the United States the incidence of Alzheimer’s disease is approximately 5,700,000 patients. Worldwide, it is estimated that 50 million people live with dementia. The current annual cost of dementia is estimated at $1 trillion, a figure set to double by 2030, when the burden on the healthcare systems is expected to be dramatic.

Today, there are no commercially available therapies to address the underlying biological causes of Alzheimer’s progressive cognitive decline.

“We believe that the analysis platform described in this work opens the possibility of using big data-driven unbiased genome-wide patient selection marker identification early on in the drug development process of CNS diseases, including Alzheimer’s disease, which is currently applied more routinely in the field of oncology,” says Christopher U. Missling, PhD, president and chief executive officer of Anavex.”

Anavex is developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders, including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system diseases.

Findings from the recent study provided the basis for the testing of potential patient selection markers in the company’s ongoing Phase 2b/3 Alzheimer’s disease study, as well as Parkinson’s disease dementia and Rett syndrome studies using Anavex 2-73 (blarcamesine).


1. Hampel H, Williams C, Etcheto A, et al. A precision medicine framework using artificial intelligence for the identification and confirmation of genomic biomarkers of response to an Alzheimer’s disease therapy: analysis of the blarcamesine (Anavex‐73) Phase 2a clinical study. Alzheimer’s Dement (NY). 2020;6(1):e12013; doi: 10.1002/trc2.12013.

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