Anavex Life Sciences Corp (AVXL)

[Talon38]

Dear ladies and gentlemen there is a lot of negative noise on the board today, mainly from those investors (some previous that have taken to the lifeboats) who have a shorter perspective than we do. IMHO the longer perspective is validated by the Sigma-1 science documented by the posts below. Then we are continuing a very successful AZ P2 which will soon be joined by a precision P3 focused on those participants that Anavex will have high confidence of positive and safe response. I think we will have a more the even chance that this P3 will be conducted under the auspices of the ACTC, giving us financial and technical support. When we add to this the impending RS P2 which the Rett Syndrome Foundation will be fully supporting with their Natural History Study data, Research Centers of Excellence and the patient experience from the Neuren "Trofinetide" trial, the momentum will be gathering. We will really gain speed (and visibility) with the start of the PK P2 in Europe and the follow-on AZ P3 "down under". With positive results will come a clamor for trials in all the CNS diseases wanting to see what the Sigma-1 platform can do for them... ( IS, FX, EP, MS, AM, ALS) and you name it. The FDA wants a safe, effective winner in the CNS disease arena.

At the very worst pessimistically 2-37 will serve a sector (genome wise) in all of these diseases, will be a great drug for insomnia. and will be backed up by three or four excellent drugs for neurodegenerative and cancer conditions.

Patience and Perspective. John McCain and all the men in the "Hanoi Hilton" went for years not knowing what went on out side the walls of their prison......but they kept the faith in their God, themselves and their country. In time, a great white and silver bird with American flags on its tail carried them home.

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Mapman and many others have supplied a number of peer reviewed abstracts on Sigma-1 research while the the pre-clinical/clinical research on 2-73 for Alzheimer's, Parkinson's, Fragile X, Angelman's and Epilepsy has been conducted by very credible academic, medical and governmental entities like the University of Montpellier, Dresden University, The German Health Authority, Rosekamp Institute, Australian Health Authority, FDA, Melbourne/Alfred Health, University of South Florida, National Academy of Science, NIH, MJFF, FRAXA Research Foundation, Foundation for Angelman Syndrome, Baylor College of Medicine, Rett Syndrome Foundation, Lund University, MaGill University, and Wayne State University.

Three National Health Authorities, three Health Agencies, seven Universities, and four foundations along with presentations at a number of prestigious medical conferences says a lot about the vetting of the Sigma-1 concept and our drug.........that Anavex is carefully and thoroughly developing 2-73's wide application in a significant number of neurodegenerative diseases is very hard to dispute.

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From Mapman's and my earlier posts

i found this interesting paper on PubMed that the more scientifically inclined may find worthwhile.

Quote:
This review will focus on the role of MAMs in amyotrophic lateral sclerosis (ALS) and hereditary motor and sensory neuropathy, two neurodegenerative diseases particularly affecting neurons with long projecting axons. We will discuss how defects in MAM signaling may impair neuronal calcium homeostasis, mitochondrial dynamics, ER function, and autophagy, leading eventually to axonal degeneration. The possible impact of MAM dysfunction in glial cells, which may affect the capacity to support neurons and/or axons, will also be described. Finally, the possible role of MAMs as an interesting target for development of therapeutic interventions aiming at delaying or preventing neurodegeneration will be highlighted.


Quote:
Furthermore, MAM dysregulation occurs in several neurological pathologies including Alzheimer’s disease, Parkinson’s disease, and motoneuron diseases10–12.


Quote:
SIGMAR1 (sigma-1 receptor) recessive mutations were identified in both adult-onset137 and juvenile-onset ALS (ALS16)33,36 and also in patients affected with distal HMN (dHMN)34,35. SIGMAR1 is an ER chaperone protein present in MAMs and strongly expressed in motoneurons38,160. The mutated SIGMAR1 protein is unstable and non-functional, probably acting in a loss-of-function manner36.


https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5832431/

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other abstracts

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5603014/

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5832754/