Anavex Life Sciences Corp (AVXL)

[falconer66a]

Once again, as always, safety and efficacy.

What does this [very positive therapeutic results] tell us regarding the probability of success of this trial?

Well, the question, in another form, is, can these early-stage clinical results be accurately extrapolated for the study’s extended 204 weeks; or, on out to the big Phase 3 study that is concluding? The very positive results appeared in the reported, initial 57-week period. Any significance to these results?

In traditional perspectives, all of the conventional cautions and warnings would be in order.

A) A 57-week trial period may be too short to know the outcomes in long-term, chronic treatment regimens. The study is too short.

B) Apparently only 21 individuals participated in the study; a low number. By conventional protocols not enough to determine certainty. Even though the p-values were low and quite acceptable, (most less than 0.05) the study’s determined metrics are certainly not enough to allow the FDA to approve blarcamesine as a new Alzheimer’s therapeutic. That can’t happen until the top line data from the soon to be concluded big Alzheimer’s study are released, said to happen sometime this fall.

But the results of this short study, with only 21 subjects, for me, confirm that the final reports in the big Phase 3 study will be a profound success. For these reasons.

Most drugs acting in the central nervous system have all sorts of untoward side effects. They do provide positive therapeutic outcomes; well and good. But at the same time, because they are acting in the nervous system, they concomitantly change or affect non-targeted things; even outside of the CNS. But that has never been the case in any blarcamesine study, of any size, in any organism.

In no blarcamesine study have there been reports of genetic disruptions; no mutagenesis. Blarcamesine doesn’t change genes, causing cancers or other genetic pathologies.

No endocrine disruptions. Hormones and hormone signaling are unaffected.

Blarcamesine is active in chromosomes, which in many drugs causes adverse outcomes. Instead, blarcamesine favorably facilitates normal gene expression by modulating chromatin mechanics (proper unwrapping of chromatin enclosing DNA, to expose DNA to make proteins).

Lots of other side effects and molecular disruptions in other nerve-acting drugs. Simply, they don’t exist with blarcamesine. Read closely the details on both the murine (lab rodent) and human tests with blarcamesine. What were the reported adverse events, side effects? Only two types have been reported: transient [short-lived] dizziness and headache; all of which abated shortly after dosing began, or dosing levels were reduced (the dose titration process).

Once again, as always before, blarcamesine is both therapeutic (efficacious — it works) and safe (causes no side effects of concern). That’s what happened in this 57-week Phase 2 study in 21 patients with Alzheimer’s. There is no reason, no evidence whatsoever to believe that the results of the big, definitive Phase 3 study soon to be released won’t be the same.

The results of this Phase 2 study are: “Biomarkers with a significant impact on clinical outcomes were identified at week 57: mean plasma concentration of blarcamesine (slope MMSE:P < .041), genomic variants SIGMAR1 p.Gln2Pro (?MMSE:P < .039; ?ADCS ADL:P < .063) and COMT p.Leu146fs (?MMSE:P < .039; ?ADCS ADL:P < .063), and baseline MMSE score (slope MMSE:P < .015).”

If these results data were derived from the big Phase 3 study, which is much longer, with many more subjects, anyone want to contend that the FDA would not approve blarcamesine as a new Alzheimer’s therapeutic? Of course not. The numbers; especially compared to existing Standard of Care (SOC) Alzheimer’s drugs; are stunning.