Ann: Mesoblast Completes Resubmission of BLA to FDA for SR-aGVHD, page-206

SI told me at the AGM that they had not discussed not doing another trial with the FDA. The CRL 'recommended' a double blinded placebo controlled trial that MSB and treating physicians indicate is not ethical (therefore possible). Recommend does not imply mandatory, but it is risky not to do so. To me this remains the biggest risk. MSB have addressed all the other aspects, but cannot do another trial as recommended. Presumably Krause (who should know) is strongly of the view that approval is still likely with the remaining CRL issues addressed and with the addition of the four year survival data.

The context in which the approval was assessed has changed with Ruxolitinib showing no efficacy, whereas at the time of the CRL it had recently been approved. I conclude that MSB is relying on survival data showing that it is a cure, in a context where there is no alternative to get them over the line without another trial. Here's hoping they are right.

I suspect that MSB is waiting on partnership agreements for chronic heart failure and back pain till approval of Ryoncil so that they can negotiate from a position of financial strength. If we don't get approval, then we will have to take what we can get to survive. The Oaktree Capital loan will have to be repaid before we can get near any other approval.