Hi
@Wilba32thanks for the post. Good questions.
Your post was largely about side effects & the disease stage at which it will come in. I think we could see something special in this space on approval - because RYONCIL's treatment efficacy has been correlated to MAP scores, rather than a simple disease severity grading. In any case, you'd expect it to be looked at by anyone with Grade C or D assessed severity.
CEO Itescu has made clear that in the US we're not actually putting ourselves into 'competition' with Ruxolitinib (Jakafi/ Jakavi) for cGVHD or aGVHD or anything else in paediatric indications right now at all. And that's how the FDA will see it too.
But that's because there's
nothing approved by the FDA for marketing in
paediatric indications i.e. for cGVHD or aGVHD, much less for SR-aGVHD, and much much less for RR-aGVHD. SOC is still a dog's breakfast of off-label products being thrown at each kid, while physicians tear their hair out, as I understand it.
However, once we have a product that's approved for use in adult aGVHD indications (either SR-aGVHD or RR-aGVHD) we'll be in direct competition. Because for those indications, Ruxolitinib (Jakafi/ Jakavi) is approved for marketing, and has become part of SOC. So, one of the considerations in specifying a Remestemcel-l treatment is whether to confine it to a part of the adult market - the easier part to get into being the RR-aGVHD segment (where it would have no competition from Ruxolitinib, of course).
There's always off-label use, and many HC posters much better informed than me believe that will be significant in the adult market, commencing from approval for the paediatric market (in any indication).
This will all be sorted out in Q1 as I understand it, because that's when the adult trial protocol will be presented by MSB and the BMT CTN to the FDA. Mesoblast Ltd and the BMT CTN may be designing the adult population trial narrowly i.e. with more broad exclusions, and more limited inclusions - I'm not aware of CEO Itescu having said anything definitive about current trial scope & big chunks of it may depend on early FDA feedback (after FDA reconfirm & re-reconfirm & then re-re-reconfirm they're OK with the go-forward potency assays (joking, but not really)).
Personally, I think the BMT Clinical Trial Network will want to take Ruxolitinib head-on for SR-aGVHD, and not just leave Remestemcel-l's directly marketable utility to those patients who prove to be Ruxolitiinib resistant. And I'm pretty sure MSB would be thinking the same thing at that point, because that's the main market.
Hope that assists.
PS: If there were to be an election for Royal Tealady tomorrow, you'd have my vote!
Cheers
Have a good one
(20min delay)