Picking, Using Only Favorable Clinical Participants
Do not presume the variable results of the Phase 1 / 2 Australian trial will be those of the big Phase 3 clinical study.
The two studies are not parallel, by any means. Offhand, it could be presumed (as many here do) that the new Alzheimer’s trial will be simply a bigger one, with a much larger n=, number of participants. With that (were it the only thing to consider), statistical significance of any of the measured outcomes will not be in question — as they are with the Australian study.
That study yielded a variety of results; most of which trended very favorably. Cognition was either maintained, or, for some, actually enhanced, improved.
So, were those data merely a result of happenstance, where by chance a good number of people were favorable responders to the drug; or were there just by chance people who would keep or improve their cognition regardless? Chance, or direct cause?
Did Anavex 2-73 cause any or all of the favorable outcomes; or, was it only by chance, with no real cause/effect connections? Since the number of participants was small (32?), many (here) will argue the by-chance causation, not an authentic, unique efficacy of Anavex 2-73 against mid- to mild-stage Alzheimer’s disease.
Now here’s the big thing; where the upcoming big n= Phase 3 will be utterly different. The difference will not be merely size, number of participants (200?). Rather, it’s not how many in the trail, but who.
Anavex Life Sciences Corp has engaged firms who have precisely and minutely detected any number of personal variables that affected favorability to positive Anavex 2-73 outcomes. This time, before any trial participant is administered her first Anavex 2-73 tablet, Anavex has a high-probability understanding how each individual will respond.
As per the new FDA drug testing protocols, (precision medicine), Anavex is going to be highly discriminatory. To participate, one must have more than diagnosed Alzheimer’s disease. The person must also have genetic, metabolic, or other detected traits that assure the highest, best response to Anavex 2-73.
In fact, it may be only 25% of the general Alzheimer’s population with high levels of Anavex 2-73 response. Those will be the people allowed to participate. People with traits negating or suppressing a strong response to our drug will not be allowed to participate. Again, profound discrimination; stacking the deck in Anavex’s favor.
And, creating the best chances for eventual FDA approval. Remember, again, to gain approval Anavex merely needs to show equivalent safety and better efficacy of any of the existing four Alzheimer’s drugs. The safety profile, from the Australian study, is exceptionally strong. It should be replicated in the new study. All four of the existing drugs merely slow symptomatic progression, and only for short to moderate periods. If only the best responders in the Australian study are the types allowed in the Phase 3 study, Anavex 2-73 approval is unquestioned. Poor responders won’t be in this trial. Strong responder data sets will greatly exceed those of the existing drugs.
Because of precise analysis of the Australian data, and because of new FDA drug testing rules allowing or encouraging precision medicine (matching specific drugs with particularly favorable populations) Anavex will have just the best patients to prove both the safety and profound efficacies of Anavex 2-73 against mid- to mild-level Alzheimer’s patients.
