It certainly is big in the sense that it would appear they know exactly what the FDA is willing to accept as trial results before they would file an NDA in a few years. The great news for longs on that stock is that by having he money and FDA support to run concurrent P3s, is that they will save at least a year in their process. It also shows what is clearly expected by the FDA for mild to moderate alzheimer's. Those that expect to take a shortcut will likely be disappointed.
While its probably been the best "trading" stock I have ever stumbled across, its still a coin flip as to their drug IMO. Their is plenty of research to show the role FLNA plays in AD pathology and they used this to develop biomarkers dealing with neurodegeneration and neuroinflammation as well as other studied AD pathologies. They met all endpoints on those biomarkers as in every patient met them, and commenced and open label study that has now shown improved test scores at six months with an update on those 50 sometime this summer when they hit the 12 month mark.
All of the above is positive no doubt but I imagine almost any drugs going through the AD approval process would have their own biomarkers just as bryostatin could measure pkce levels, bdnf and other growth factors, etc which are known reduce inflammation and neurodegeneration. In other words IMO biomarkers aren't nearly as important as actual results. Their results are positive, but open label studies without placebo are notorious for not painting an accurate picture. Their 12 month results, if positive, would be another step in the right direction, but first placebo controlled results are a long way off.
Just for comparison sake, pkce is upstream of the SAVA target. That alone has very little to do with a drug being more or less successful compared to another drug however. Whether your drug actually produces results is all the really matters, although some may think the further upstream is a cure all but that's likely not the case. Research does exist to the positive impact bryostatin plays in the FLNA process though and I've posted here. Also Alkon pointed out at the investor conference that one potential explanation of why SAVAs drug may be of some benefit is that is does have some indirect effects of PKCe as opposed to bryostatin's direct effect, but then again it may all be about the FLNA.
Reality is that bryostatin has improved test scores now in multiple trials, and initial patients in the current trial are likely now on cycle 2 of the drug. We won't know results until the second half of next year (later in the year). At that point we'll see what the FDA will require for this moderately severe population. At that point they will have data from nearly 350 patients in an indication that only we are trying to treat. The current trial requires a final data check point months after last dosing. My guess is the FDA asked for this as part of safety precautions. I'm certainly hoping that the likely additional P3 trial for us will just be a single trial and hopefully not more than 500 patients. Just my take anyway.
