DD re: discussion of genome sequencing database:
This is not a drill. This is a real post based upon real information - DD.
Suppose I were to tell the board that the AD trial (with 32-25 participants) which was conducted and is ongoing has no material impact on the future phase trial whatsoever.
Suppose that the size of the sample (32-25) was designed small because it was a test of very little relevance in the broader scope of precision medicine and applied use of scientific method.
Other than to make sure that no participants had severe adverse events when administered our drug, the rest was immaterial. The super responders were not planned for, expected, nor necessary.
Please google the Illumina website.
There is a database of 50,000 genome sequences specific to Alzheimer’s patients - data housed in the UK, which will reach 100,000 by end of February 2018.
A local biotech CEO in the exact field as Illumina (please google Illumina and read their PR’s and watch their webinar) shares an SAB member with Anavex. If not for that conflict, he and his company might well have had some dealings with Anavex. The application of genome sequencing and the role it plays in weeding out noise, making for precise inclusion/exclusion criteria is exceptional beyond belief. The fact that other AD trials never used this approach is beyond belief but our very great fortune.
Using Illumina’s AD database (they have one for Parkinson’s which is much larger - and much to my surprise there is almost zero overlap between AD and PD in the genes affected - go figure. Interesting! And they have one (genome sequencing database) for rare diseases.)
Bottom line: our super responders are not dose dependent, rather, they all have a common genetic thread - never mind the dose/concentration those are immaterial. The patients in our trial - a very small cohort - were necessary to confirm that the theoretical science of the compound (crossing BBB within MTD, no safety issues within MTD, tolerability in a human with AD) would work in applied conditions on real live humans with AD.
Since that has been confirmed, the rest can be done using the database only, I repeat, even with no responders at all, Anavex has the ability to summon a cohort which will respond to the administration of A2-73. Its true. The reason 32 patients were needed was because the FDA would surely not have allowed a trial for approval without going through the lower levels of the process.
The SAB members know what is available and they sit on boards of synergistic companies. They “go golfing” together. They know the best resources available and our company is using them.
Please google Illumina’s website.
This is not data mining or post hoc analysis - good lord 5 people???? Have you lost your mind? (Even if analyzed until the end of time that would yield little and nothing reliable.) There is data available on tens of thousands of patients (which has yielded much and is very reliable / listen to the webinar). We have access.
If 5 of ours returned positive outcomes, that’s great and they will match what has already been observed. Cohort done. If none had been positive (it would have been very difficult to justify funding a next phase) but we still would have had the ability to tap the known data and succeed.
Please google Illumina.
This is not a crap shoot. This is not a failed phase. This is unbelievable. This is the new world of medicine. “The boy in the bubble and the baby with the baboon heart.”
As for Parkinson’s the data sequencing turned up so many gene biomarkers...unfathomable...
Please google Illumina and listen to the webinar.
The key is mitochondrial dysfunction. Who would have thought?
They state it plain as day.
This thing was designed to fail quickly if the compound could not be tolerated in human AD participants. Once that did not happen, and with each passing day, it was designed with cohorts already in mind - not from our trial! From the genome data. This design was ahead of its time, and well constructed. This is the scientific method in action and has a logical way of proceeding to a logical outcome. Don’t know who gets the credit but, well done, I say!
Further note: according to my source, CEO is aware of how to make shorts cover (the anticipated burn you all are blood-lusting for is one thing a CEO has control over. I thought M wanted no parts of burning them but I was wrong. There is a very funny/graphic German/Bavarian expression for our term “burning the shorts” which I cannot print here.)
Take this post for what it’s worth - AFTER googling Illumina.
We have a winner here, imo.
Then why the wait, you ask? The 50,000 mark of the genome database for AD was just passed - the UK and US are working together on this. 100,000 mark coming - EOFeb. Fully funded by UK govt. We are going to get this right, no error. Google Illumina.
Note: The trial participants do not need to be in the existing database. They can swab anyone to test against the database for similar markers. This is how drugs for rare diseases which have never occurred before are developed and cohorts are formed, based upon targets housed in the database and finding similar matches.
Please google Illumina:)
(Also, if you want to blow your mind, google Multi-modal imaging. This is another tool Dr M mentioned when discussing how trials would be designed. These scans can detect inflammation in the brain in specific areas. Pretty cool way to provide evidence of a decrease along with better test scores....
“This is the age of miracles and wonder, this is the long distance call...”
Best,
Bio
