Replies to post #327454 on Anavex Life Sciences Corp (AVXL)

[falconer66a]

From the new paper’s abstract. Rather definitive:

The present findings support the viability of S1R as a therapeutic target in FXS, and the clinical potential of blarcamesine in FXS and other neurodevelopmental disorders.

Then, from the paper itself (my text markings):

"In conclusion, the present findings confirm the dose-dependent receptor occupancy of the S1R with blarcamesine and, combined with the therapeutic response observed at low doses in the tested preclinical model, emphasize the viability of S1R as a therapeutic target in FXS and the clinical potential of blarcamesine in FXS and other neurological disorders. Indeed, pre-clinical studies in a mouse model of Rett syndrome showed similar positive effects on multiple clinically relevant neurobehavioral phenotypes21. Furthermore, clinical efficacy was demonstrated in a placebo-controlled Phase 2 study in Rett syndrome (NCT03758924) and previously in a smaller PK cohort of patients with this neurodevelopmental disorder39, as well as significant cognitive improvements in a Phase 2 trial in Parkinson’s disease dementia (NCT03774459). Late-stage clinical studies of blarcamesine in adult and pediatric patients with Rett syndrome (NCT03941444, NCT04304482) and Alzheimer’s disease (NCT02756858, NCT03790709) are currently ongoing. Continued findings from these clinical studies with blarcamesine, combined with the presented data strengthens the rationale for potentially a dependable and effective treatment strategy for FXS and other neurological disorders targeting the S1R with blarcamesine."

On every point, the efficacy of blarcamesine, where it activates the sigma1- receptor, is substantiated. Of course, all mammals have sigma-1 receptor proteins, both mice and people.

I’m ever more confident that the up-coming results from the on-going Rett syndrome trial in humans will be strongly positive. Later will be a trial substantiating blarcamesine for fragile X syndrome and other forms of autism.

Game-changing.