Two Measures of Efficacy in Alzheimer’s Trials. Blarcamesine $AVXL $SAVA $ANVS

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There are two measures by which go trials of Alzheimer’s/dementia. The most popular and the most expeditious is the average increase/decrease in MMSE or ADAS-Cog points of the dosed cohort vs. the placebo. The other is taking much more time and is probably more telling of the drugs potential. It is the number of dropouts or remaing patients in the dose cohort over time vs. the placebo. Why do I make a statement like this? It is because the average change is scores can be much easier a statistical event (fluke) during the 18 months long trial than the dropout rate during few years of study. Again, I use the information in the reference https://pubmed.ncbi.nlm.nih.gov/10404988/ to make the point. In this study 372 subjects were followed over the natural progresion of the disease from diagnosis to droping out. The event of disappearing from the study rolls was connected with severe disease or natural death, almost exclusively. There was a plot of the changes in MMSE scores in consecutive one year periods. If indeed, the change in a patient was in given year over 10 points then we can reasonably believe that he might become soon a dropout due to severe AD and find himself institutionalized. This rate of those declining 10 or more points MMSE is almost steady during the 6 years of the study. The picture of the disease I see in this graph is that of a disease which accelarates rapidly during the final year in the study leading to removal from the study. Notable is that the first year results do not confirm this phenomenon and as such are the event which confirms the phenomenon taking place later in the study, as the initail scores are still not in the domain of severe Alzheimer’s/dementia leading to dropping out. The average decline in MMSE scores for this study was -3.8 SD +/-4.3. This is visible as the line under the zero MMSE line. The average rate is almost constant. Also remember that a dot stands for score not a patinets and can represent few patients.

The overwhelming efficacy of the drug can be seen in the ability to stop or at least control this phenomenon of rapid and accelerated decline and in effect lowering drop out rate.

During the latest Conference Call Q1 2021, Dr. Missling said that the number of remaing patinets in Open Label Extesion after 5 years is between 10 and 21. Parsing it a bit, any number in between is technicly true now so we were not given an answer but a clever way of confounding us. 10 patients left and we still are much better than placebo from the study. 21 patients might be equally true and we are shining. See my last few post on the possible meaning and interpretation of that data. I shall refrain from further interpratation of the Conference Call statment by Dr. Missling. Hat tip to Tom Bishop from BI Research for asking this question. Does anybody know what BI Research wrote about $AVXL if ever?

Currently not company can boast data that deep and rich as $AVXL. I am waiting for data from $SAVA and $ANVS.