The results were reported at the 2018 Alzheimer’s Association International Conference (AAIC) in Chicago.
The study, “Systematic Processing of Full Genomic Analysis of ANAVEX®2-73 Phase 2a Alzheimer’s Disease Study Identifies Biomarkers Enabling a Precision Medicine Approach,” was presented by Harald Hampel, the study’s first author and AXA Research Fund and Sorbonne University Excellence Chair at Sorbonne University in Paris.
Anavex 2-73, developed by Anavex Life Sciences, is an investigational therapy with the potential to modify the course of Alzheimer’s disease instead of acting on symptoms alone.
It binds to and activates the Sigma-1 receptor (SIGMAR1), known to have neuroprotective effects, resulting in reduced neuroinflammation, accumulation of beta amyloid and tau proteins, and oxidative stress. All are involved in Alzheimer’s disease and other neurodegenerative disorders.
Results from a randomized, open-label, Phase 2a study (NCT02244541) showed that one-year of treatment with Anavex 2-73 was safe and prevented further cognitive decline in 32 people with mild to moderate Alzheimer’s.
From those participants, 21 agreed to move to a two-year open-label Phase 2 extension study (NCT02756858) where they would continue to receive Anavex 2-73. The study aimed to evaluate the therapy’s long-term safety and therapeutic benefits.
Researchers conducted a detailed analysis on the entire DNA and RNA — the molecule produced from DNA that contains the instructions to produce a protein — of these patients to find biomarkers of response to Anavex 2-73.
After analyzing 33,311 genes and 860 pathways, the team identified several mutations associated with patients’ responses to Anavex 2-73. These included mutations in the SIGMAR1 gene, which produces the target protein of Anavex 2-73, and in the COMT gene, which generates a protein involved in memory.
Mutations in these two genes were found in about 20% of patients and were associated with significantly fewer improvements in cognition and everyday activities. The 80% of patients who had no mutations showed improvement in these areas, suggesting that most Alzheimer’s patients may potentially benefit from Anavex 2-73 treatment.
“This study represents an exciting step forward, away from the ‘magic bullet, one-size-fits-all’ drug development in Alzheimer’s, following the targeted therapy successes in the field of oncology,” Hampel, MD and PhD, said in a press release.
“Our vision is that a precision medicine approach will allow us to more precisely treat and prevent key features of the cause and progression of Alzheimer’s,” he said.
The company said these treatment response biomarkers will be used in a randomized, placebo-controlled, Phase 2b/3 study that aims to evaluate the safety and effectiveness of Anavex 2-73 in 450 patients with Alzheimer’s disease.
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