MSB All-time low Party Thread, page-334

I'm not sure what you are apologising for - I was not posting to embarass you, (I kind of agree with bedger that this is not a great thread to encourage by posting in - it pretty much is a downramp on its face - but I saw your comment here and I didn't think leaving it go was doing you or anyone any favors and it might have been a disservice to someone who has sometimes provided good points and sources - I don't need an apology - but the facts matter - what MSB has written in their own announcements shouldn't sensibly be read as meaning something completely different.

It seems @dachopper, and some others still think your initial interpretation is the correct one and if you were apologising for not including your source rather than for over stating what your source says then perhaps you still think so as well.

If you look at the words on the end of your marked third key point in the December 2021 announcement they say -

"OTAT indicated that the in vitro immunomodulatory activity Mesoblast intends to measure for potency is a reasonable CQA for the product in the treatment of children with SR-aGVHD, and the relevance of the immunomodulatory activity to clinical outcomes should be established."

MSB wouldn't be writing "should be established" which has a different meaning to has been established if the second meaning was what they meant to convey.

One is a a statement about what OTAT suggests MSB should do (establish the relevance - a potency assay for immunological activity that isn't established as related to clinical outcomes is hardly a potency assay at all) and the other would be a simple direct statement about what MSB had already be told by the FDA (if it had already established the relevance to the satisfaction of the FDA).

There is a bunch of extra context that might make this important point more certain potentially - FDA meeting procedures - other MSB statements made at other times, perhaps even a recent statement by MSB's Rose in the KOL video, but really nothing in my opinion so clearly makes the point that the FDA has not yet agreed to the potency assay as being certainly relevant as the "should be established" wording from MSB's own statement.

Phaedrus warns about trusting anonymous sources and suggests MSB company officials would chose words in announcements carefully - okay - "should be established" are words company officials wrote into an official announcement - they aren't the words of an anonymous ramper.

In my opinion it is possible that MSB will succeed in getting a BLA approved without another trial. I can't say that it is certain but I can say that I think its possible with ST2, Reg3alpha and MAP surrogate biomarkers related to survival being used to shore up confidence in the single armed pediatric clinicial trial that has already taken place.

If MSB adds new potency assays besides TNFR1 and IL2Ralpha to the release assays though, which it could, unless those assays are somehow linked to the trial that has already taken place, (ST2, Reg3alpha and MAP could be linked because they are markers from the patients not the products) then I can't see how the new potency assays would help make the already completed trial be sufficient as an adequate and well controlled study.

I think the FDA needs to have an adequate and well controlled study (at least one) to grant a BLA so I think MSB has to rescue the last one to have at least one without having to do another one. So potency assays that are new and on the product side rather than one the patient side would seem to be too late to do that now. You can't use new potency assays on product that has already been administered in a trial already completed to further bulk up the credibility of that already completed trial. You can use new patient date though (ST2, Reg3alpha and MAP). In my opinion.