CEO Itescu needs to go, page-747

@DV, for someone who wants this thread to die, you sure like to ramble on about it. Not much of substance, but at least your dull repetitiveness can be pricked by paradox: "The surprising thing is that they are OK with using adult trial (results) for efficacy in children".

Yes, given the content of your musings, I can see how you might be surprised by something that should not be a surprise. Maybe if you had done your homework... which is to list for us all those medications approved for children, but not adults, for indications that occur primarily in adults. In other words, out of the global spectrum of maladies, hundreds upon hundreds, that befall human beings, find us one that occurs primarily in adults... BUT the MEDICATION to treat it was first advanced in children.

I'll start the Old McDonald had a Farmacopoeia for you:

1) Magic Fairy Dust

Advancing rem-L to treat children for SR-aGvHD, which is a complex, evolving, multi-faceted disease primarily of adults, was based on a not-statistically-significant signal from a trial of Prochymal that failed. Yes, the product has been improved with ex-vivo fucosylation and the like, but the decision to advance it for a first approval in children without benefit of solid RCT data... was a mistake. imo. And then persisting with a dispute for three years when the FDA specifically asked for RCT data falls into the category of Big Fat Mistake (or worse). Everyone can make a mistake, but it takes a special combo of power, hubris and blinders to make big fat ones.

The entire rem-L program needs a deep-dive reassessment, starting with an assessment of upper management.

If we line up all the trials that have been completed by Mesoblast to date, GvHD no longer rises to the top. Two CRL's are a hard pill to swallow especially for ensconced management. The "quick" targeted trial in adults could take 2-3 years and could fail. Getting a limited-indication, long-shot AA to treat a rare condition involving a small number of cases per year could jeopardize the whole company if it were ever suspended. They can always come back to it later... that's the rational path other companies have followed for GvHD. For a first approval there are better risk-reward odds with CLBP or CHF which already have established phase 3 RCT data. imho, Left-e