So what is the MOA?
If you recall, the February 11 PR noted,
"Researchers at the University of California San Diego have identified the underlying cause of Alzheimer's disease in neurons. They discovered that changes in the structure of chromatin are responsible. ["Chromatin is a complex of DNA, protein and RNA found in eukaryotic cells. Its primary function is packaging long DNA molecules into more compact, denser structures." Wikipedia] Sigma-1 Receptor (SIGMAR1), the direct target which gets activated with ANAVEX 2-73 demonstrated to restore chromatin structures."
Today's PR:
Anavex "today reported predictive biomarker of response established with SIGMAR1 mRNA expression correlates significantly with responses in primary clinical efficacy endpoints" in the first Rett trial.
From Wikipedia's entry on mRNA:
"mRNA is created during the process of transcription . . . . Transcription is when RNA is copied from DNA. During transcription, RNA polymerase makes a copy of a gene from the DNA to mRNA as needed."
It seems, from my reading, that 2-73 acts directly upon Sigma-1 receptors, which in turn restore chromatin so that it can do its job in facilitating transcription of a cell's DNA into the mRNA operating instructions. AVXL references this function in terms of increasing Sigma1-R presence/activity so that various intracellular functions involving, for instance, calcium transport and waste recycling work as intended. Healthy conditions then ameliorate or can even reverse CNS pathologies.
This seems to be the MOA, supported by research and now by clinical trial. However, I cannot see why 2-73's chromatin repair should only be focused on CNS-related mRNA. I can see why AVXL would not make any statements beyond what has been said (which is a lot). But I am not bound by SEC constraints -- this looks like a universal therapeutic.
We appear to have re-entered Too Good To Be True Territory.
I would be interested in our honest broker experts MayoMobile and Falconer have to say.
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