What to Expect at CTAD 2019?
We fret that this company has no respect from anybody but we know with all our hearts that this is the drug. The press taunts us with “next miracle drug soon to help the sick” meantime short-sellers drive the stock price up and down in order to squeeze few dollars from the hopefull “millionaires” as the $AVXL faithful look at their statements and wish the worst upon the short-sellers. To add insult to injury $BIIB goes up by $15 billion in market value over a day on news that its failed drug can marginally slow the disease.
Fear no more since Dr. Missling goes into the offensive.
In the CTAD 2019 program, there is an oral presentation LB-20 by $AVXL. It references in description to data provided by Alzheimer’s Disease Neuroimaging Initiative ADNI . The ADNI collects and disseminates data on Alzheimer’s patients including imaging and seeks to identify early-onset dementia and Alzheimer’s disease biomarkers. This data collection involves also documenting the progression of the disease, that can be used to validates the biomarkers. Information covers PET (using a radioactive marker for amyloid plaque imaging), FDG-PET (glucose activity in the brain) and structural MRI (cortex thickness) and I believe staple of Alzheimer’s research; cognition and daily living tests and DNA data. The activities are broadly based in many research venues and rigorous proceedures are applied in collecting data for the industry. What is most important the data can be used to create a comprehensive statistical and clinical profile of disease progresion.
So it seems that $AVXL wants to validate Phase2a data in two ways. One is by comparing the cognitive performance of the Phase2a patients to some data substitute for the placebo arm. The above mentioned ADNI data is to be taped with this purpose in mind. The other part of it is adjusting this data for “propensity” (DNA profile: SIGMAR1 and COMT genes), and again ADNI data is used. Since no other data was collected (?) save the one on cognition the validation only includes cognition. This is adding to the data virtual placebo arm and eliminating the 20% who do not respond to Blarcamasine making the study virtually statistically significant. I have spent some time here making educated guesses on Blarcamasine performance. I was thwarted by the company always formating the data in such a way that comparing it with the previous set of data involved multiple assumptions.
Now to the main course
The other thing is that Frederico Goodsaid from Regulatory Pathfinders (US) is listed as one of the authors. For more info form the horse’s mouth visit Regulatory Pathfinders Webpage. I think this quotation from the webpage should ring the bell:
The value of medical product development knowledge is maximized when it can be shared between its source and regulatory agencies and integrated into coherent information.
Strategic Regulatory Intelligence is a useful tool to drive regulatory discussions about novel precision medicine products. In addition to this tool, however, the global strategy for regulatory approval of precision medicine products may require the identification of regulatory paths which do not have a prescriptive designation associated with current regulations. Regulatory pathfinding identifies regulatory paths which are not prescriptively defined :
I venture to say that Regulatory Pathfinders are very likely to negotiate with FDA on behalf of $AVXL. The implications are that the “augmented” performance of Blarcamesine has a good chance to be used to convince the “new” FDA to grant accelerated or conditional approval.
I say, fantastic, but will not guarantee the approval by Christmas!
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