Ann: Update on BLA for Graft Versus Host Disease, page-143

Approval would have been better but the basis of this CRL is actually very good for us. They have not knocked it back on manufacturing or batch consistency or uncertainty about the mechanism of action. Those might have been very hard to deal with. But rather they have over-ruled the ODAC on proof of efficacy, gone with the one dissenting vote and said we need a randomised controlled trial. (Obviously that will be undertaken in adults, not children, despite them contemplating the dead-babies option.)

That will be much easier for us to deal with because we have very near-term read-outs in three gold-standard, double-blind trials: Covid ARDS, chronic heart failure and chronic lower back pain. If those show the cells don't work, then we have backed the wrong horse and we can tear up our tickets. But if they show efficacy, then the path is clear because MOA, manufacturing and consistency seem to have dropped away as issues.

The market will over-react, of course, but as I said before the ODAC meeting, this is a side-show.