MESOBLAST LIMITED (ASX:MSB) - MSB 2023 - awaiting for Ryoncil approval, 66218508, page-539 - HotCopper | ASX Share Prices, Stock Market & Share Trading Forum

Originally posted by JB1975: ↑

So what did Mesoblast submit? New data to support effectiveness of Ryoncil, linking MOA to survival of patients in the phase 3 trial. So some may conclude that Mesoblast have given the FDA what they asked for... is it exactly what they asked for? No. But it does address what the FDA want... more data to support effectiveness.

You've underlined what Mesoblast submitted (in your opinion) - but you haven't put it your own words what those general descriptions that you've underlined could actually be referring to in practice.

Its hard to link mechanism of action to survival of patients in the phase 3 trial when the mechanism of action is based on lot data (that's TNFR1 expression and IL2Ralpha inhibition on co-cultured t cells) (so few (matches of the lot data with the patient data - survival or otherwise) are uncontaminated by other confounding factors) and the good results.

I think you and I are in agreement that the FDA needed (after the CRL to see substantial new data). Where you and I seem to part company is that you don't seem to have a detailed understanding or to have put one on the record as to how sufficient new data could possibly be brought together without a new trial being done to generate it. Sufficient evidence in the view or an independent science-based and statistics based decision making body (the FDA should be that body - it should not give in to politics or special pleading - it didn't previously and it shouldn't this time either) ought and should be in existence before a BLA is granted.

If sufficiently persuasive new data could have been generated without a new trial - perhaps through running laboratory tests again to show up correlations that weren't clear in potency assays previously then - perhaps the phase three Study001 could have been rescued as a necessary "at least one adequate and well controlled trial". But such a rescue was clearly necessary. The FDA's statements at ODAC made that clear. They weren't seeing the correlations they expected to see on the data they'd been given (some of which data didn't get included in MSB's ODAC presentation) as the FDA made clear.

The longevity data (four years) is based on the same limited amount of donors and lots (only from three donors) that went into study001. It was good looking data for survival even at ODAC - it doesn't greatly help that folk that had largely survived then (about 25 patient got compared to 27 others after the trial) are still alive now in my opinion because they'd have been expected to keep surviving.

To my mind, and I think to the FDA's collective mind, what matters is whether the product that went into those Study001 patients will be as good in other future patients when the potency assay that is to be used is used.

Without a valid potency assay - you can't predict that. The FDA needs a valid potency assay to predict that. It would be bad policy and a bad precedent for stem cells and regenerative medicine to give the go ahead on a product that its going to be sold to parents of sick kids if that product can't be reliable shown to be potent. The temptation to go with a weaker version (but still harmless version of the product) would be too great.

Goosing old data doesn't increase your sample sizes.

I've no argument against using laboratory tests to provide evidence instead of second or third trials if the science and stats evidence stacks up doing it that way. I've no argument that random controlled trials must be done.

I don't want MSB to fail ultimately. But I don't want them to get an undeserved pass conceded either. I don't think the science and the stats stacks MSB have presented stacks up yet. Not on the data that MSB have put in the public domain. And I don't think they can have a lot of data in the private domain that makes enough difference to the small amount of samples that link putative mode of action with survival (or serum markers or anything else).

I think MSB needs another trial to get enough data not because another trial absolutely was required if they hadn't had problems in the potency assay.

Unless there is some amazing stuff in the data MSB has not made public then I think good science and good stats as I understand those terms simply requires MSB generate new data in the interest of good medicine.

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Hey sponge_, I don't want to encourage you because you're annoying AF but just wondering whether you spell check this guy's MSB-hating essays? Or like most of us, do you also not bother reading them?

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Last updated 14.54pm 14/06/2024 ?

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