Hi
@JB1975I enjoyed your well-structured post above - in particular the internal logical consistency of your argument presentation, the way you've substantiated your thoughts by referencing prior HC:MSB posts and going to the trouble of extracting the full narrative text of your focus of interest from
@Bazsa's earlier post, which provides essential context. As it happens, I followed the same process & reached a number of the same conclusions as you, although not all that you've listed.
Unfortunately, we HC posters post so furiously - fighting the good fight, into the breach etc - that we're constantly forgetting material already posted, and so we reinvent the wheel 5 or 6 times a day, rebuilding arguments almost from scratch. And sometimes we overlook others' well thought out prior work. I know I'm guilty of it & I could wish my memory was better and my research skills better trained. Of course thread abusers' minds work in a completely opposite manner - their lifeblood is trying to score cheap antagonistic points 'de novo' - so accepting anyone else's argument is anathema to them. They won't do it.
So apologies for reposting, but I need to refer you to some material linked on this
Post #:61778078, especially the comments by CMO Dr Rose made in response to John Hester, Bell Potter during the Q&A session towards the end of the Q3 Earnings Call Presentation (linked
here). I'm going to extract (from my extract) two particular comments made by Dr Rose, i.e:
"
Ithink, ultimately, approval will be based on a totality of the evidence. Andwe, as shown at the panel, the clinical data are quite convincing. (~42:15min)"
and then, after immediately shifting to talk about new potency assays and then reagents for release testing, Dr Rose concluded (importantly IMO, without indicating
any further or new clinical evidence):
"
And those 3 things, clinical effectiveness, potency assay and reagents to actually do the potency assay on our existing inventory, we think, will carry the day. (~42:34min)"
What I heard as I listened was Dr Rose' going through his refiling checklist for the Remestemcel-l BLA resubmission. I believe (although I'm not aware of the question being asked in the Q&A) MSB is still seeking a standard approval in the first instance (rather than falling back on an AA).
So, although agreeing with you on the key points, when you suggested above " ...
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." (underlining added for emphasis) , I tried to reconcile that argument with Dr Rose' comments.
I decided I couldn't, for two reasons - first, MSB's unconditional view (as expressed by Dr Rose) of the adequacy of the existing clinical evidence (which you'll recall still includes the 2 earlier clinical trials - data from which is being used in the new correlative analysis discussed with FDA) and second, Dr Rose' statement that the reagents that MSB has been having so many logistical difficulties with since November 2020 are now being used in assays on the existing inventory (which I understand to refer to the new potency assays already being up and running).
Which all seems to me to imply:
(a) that the pivotal trial doesn't need to be rescued by more clinical work at this time i.e. if the FDA correctly advised MSB that the new potency (release) assays' results correlating immunoactivity to available CQAs will be considered and are able to be accepted by the FDA as demonstrating potency etc; and
(b) that the integrity and relevance of lot release samples from the earlier trials (which have been cryo-preserved) has already been indicated to MSB as acceptable by the FDA for the purpose of MSB's ongoing inventory validation process. And we're just waiting for the results for the resubmission.
Food for thought. Getting exciting, eh.
(20min delay)