Anavex Life Sciences Corp (AVXL)

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But efficacy yet to be “proven.”

Each paragraph is packed with an abundance of information. And yet, it allows the reader to easily understand and appreciate the contents.

Thank you. I was a biology instructor, had to get across deep concepts to interested students (headed to top-line colleges and science training programs). I’m also experienced in science writing, for both professional and lay audiences.

But I believe my post told only why the blarcamesine clinical results will show safety. I didn’t much tell why they, too, should show efficacy. A drug that’s safe, but produces no symptomatic relief is no better therapy than a glass of orange juice. To use a castigated phrase, in regard to safety, “We got this.” But what about efficacy? Might a presumption of trial efficacies be, also, presumptuous? Or, is the preclinical evidence of efficacy as strong as that for safety?

Ever bit. There has never been any evidence showing safety problems with blarcamesine. In every trial, preclinical or early clinical, in both murines (lab rodents) and humans, blarcamesine has proven itself safe. But more importantly, in every single completed trial of the drug results have been positive. To some degree, perhaps controlled in some cases by dosages, therapeutic results have been favorable.

Over five years ago, after I had been following Anavex for a time, I came across what I wanted to see. Specifically, what does the drug do in diseased animals? I found a good number of early murine studies, many in transgenic animals, rats or mice that had human CNS disease genes inserted in them. The little rodents had human CNS diseases. The genetics and biochemistries were close analogues to human conditions.

Critics and skeptics would (as they still do) claim that mice aren’t men; that the little rodents, even transgenic ones with human disease genes, can’t be relied upon to accurately predict human dosings of blarcamesine. Understood; that’s why the three human trials will definitively decide the matter.

But, again, in every case, where transgenic murines were tested with blarcamesine, and when actual humans with the same disease were subsequently treated, the humans had the same favorable treatment outcomes. The mice-aren’t-men crowd is welcome to hold to that position. But so far, every sequence of blarcamesine in mice, then in people (in early Phase 1 trials), humans gained the same favorable (safe) outcomes as the rodents. The murines have been 100% predictive of human outcomes.

Here’s the definitive, predictive murine study with blarcamesine (Anavex 2-73) against Rett syndrome:

We tested blarcamesine in female heterozygous mice carrying one null allele of Mecp2 (HET) using a two-tier approach, with age-appropriate tests. Administration of the drug to younger and older adult mice resulted in improvement in multiple motor, sensory, and autonomic phenotypes of relevance to RTT [Rett syndrome]. The latter included motor coordination and balance, acoustic and visual responses, hindlimb clasping, and apnea in expiration. In line with previous animal and human studies, blarcamesine also showed a good safety profile in this mouse model of RTT.

https://www.sciencedirect.com/science/article/abs/pii/S0091305719302515

There are similar parallel outcomes in animal models of Alzheimer’s and Parkinson’s.

Readers may recall Dr. Randi Hagerman’s personal support and desire for blarcamesine availability, for both humans with CNS diseases, and as a prophylactic (prevention). She’s a world-class clinical researcher in CNS diseases; actually has conducted pre-clinical tests of blarcamesine on humans with Fragile-X syndrome. Dr. Hagerman knows the study-revealed biology of blarcamesine better than I. She’s scrutinized all of the journal articles, looking for the same disqualifying evidence that I did. Neither of us have found any. There isn’t any.

But, no, we don’t have this — yet. The ongoing clinical results will formally settle the matter. Based upon all that can be presently known, I expect them to be positive.