banter and General Discussion, page-8267

@ddwn,

I'm having some difficulty getting HC to accept a reply to your earlier post. The HC internal reference URL (for your Post #: 71757088) doesn't seem to like being used in the header of my response. Anyway, below is what I meant to respond with earlier:

----------------------------------------------------------------------------------------

Hi @ddwn,

I think MSB want standard approval for the paediatric indication. As relayed by CEO Itescu, the GVHD-001 trial was acknowledged as being adequate to do that. There's just one thing standing in the way. And it would be the same thing standing in the way for an accelerated approval, as I see it.

That's why it would have been germane for BMT CTN to say to the FDA, 'look, we aren't going to do this adult trial - whether you want or are asked to consider it as confirmatory or standalone - unless and until you tell us that potency assay (Potency 1b) is 'suitable' i.e. fit for purpose'.

That's it, IMO. Once that's agreed to by the FDA, MSB has a clear path to paediatric approval IMO. Now, if MSB don't get the assay comparability results IMO they're looking for right now in Q1 2024, then that path disappears. And a potency connection to clinical outcomes will need to come from the results of that adult trial IMO. I understand that's what Dr Krause said in the Earnings Call on August 30, 2023, viz:

" ...Now the CRL then went on to say that if that can't be accomplished, in other words, this demonstration, then the only way forward would then be another clinical trial in either adults or pediatrics, which would then allow this connection between potency assay and clinical outcomes to be made and thus allow future product to be related to product that was shown to be effective in a clinical trial... "

So, I'm not seeing a way to jump that forward in that case or allow any earlier product release than eventual trial results (or at least top-line results). Good thing we're going for the main chance.

Cheers
Have a great weekend.