Transcription Or Translation: Two Sides of Blarcamesine Rett Syndrome $AVXL $ACAD

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It Seems Blarcamesine Can Works On Either Of These Or Both…

The SIGMAR1 receptors agonists can work on either of these or both but at different optimal doses. In the Trascription Complex, reading of genes is affected or regulation of genes, and the familial form (genetic cause) of a disease like Alzheimer’s can have a therapeutic response as well as the sporadic which might be more up to Translation Complex fault. This might be the reason for the fact that Blarcamesine works in seemingly disconnected etiologically diseases groups like Alzheimers, Parkinson’s Dementia or Parkinson’s Disease (neurodegenerative) as well Rett Syndrome and others alike (neurodevelopmental). In the former it is the Translation part which is primary and the response or rather required dose is much larger (30-50mg/kg) than in the case of the latter (5mg/kg), that we can presume to be more likely connected to the Transcription Complex. After the first trial in Rett producing the extraordinary good results with just the 1/10 of dose of the corresponding efficacy in AD, PDD and PD the distinction becomes clearer, Transcription vs. Translation. The expectations are that with higher dose Rett Syndrone could be stopped or reversed by just increasing the dose. These expectations might not pan out as for some reason the homeostasis can wind them down or level off even with sharply increased dosage.

If this is the case, it is not failure of the drug but rather the other nature of the so often called out homeostasis, that down regulates certain physiological processes when they could become excesive or destructive. Would this be the case with AVATAR, which on clinicaltrial.gov is listed as still recruiting? I truly don’t know but it is sill with in the realm of possibilities.

The design of all the Rett Syndrome trials for Blarcamesine points to very gingerly approach to dosing as compared to AD. Yet, this might not be the case entirely, because the dose could have been established during the murine Rett Syndrome tests. Nevertheless, the existence of AVATAR points to very measured approach seeking to establish optimal dose after the safety initial dose has been documented in the first trial. In biotech measured analysis is lacking and most investors are triggered by buzzwords. One unfortunate phrase can sink the stock price to unbelievely cheap levels wheras the value is intact.

The latest Corporate Presentation December 2021 have few slide with the results of competition’s ($ACAD) drug.

Had Blarcamesine kept its eficacy or extended it in EXCELLENCE study, by the virtue of demographics, that is having population including pediatric patients, which are expected to have a more robust response, then it still would have been ahead of Trofinetide by a country mile. Just one look at the “Age, y[ear], median” gives us the called for insight into the future expectations for the EXCELLENCE trial. If Rett Syndrome is a neurodevelopmental disease, which it is, one should expect, even with AVATAR limiting the therapetic dose to 5mg/kg, much better results from the third and final trial (EXCELLENCE). I believe that Dr Missling is trying to temper the expectations for a miracle (expectations for AVATAR results are very high) where we have just only a drug, which can be proven further in EXCELLENCE to be the best drug at present.

On a lighter note, homeostasis can be a bitch! I have witnessed another drug to work at a lower doses but not at a higher ($GALT). This is counterintuitive since our minds are biased toward linear extrapolation of physical world, just like in stone throwing. This intuition thwarts our understanding when there is a need to develop insight into the complexity of physiological phenomena.