GVHD the first step, page-7

We are almost there. A one hundred thousand page BLA document is extremely close to filing.
The long term mortality data of GVHD001 trumps any Phase 3 surrogate endpoint of 28 days . The Reach 2 trials showed Ruxolitinib (Incyte/Novartis) extending median survival by a mere four months to eleven , over that achieved by best available therapy. There is simply no contest.
In Grade D sr aGVHD , Remestemcel has shown about a 50% greater efficacy over any other reported therapy ..
The most accurate biomarker, ST2, shows that Remestemcel is far superior in relation to the all important MAP scores …..BUT…

This is NOT a rolling submission.

It is part of a dispute resolution process where only one final BLA submission is allowed .

There is supposed to be a 14 day response from CEBR after the imminent filing. As OTAT/OTP has already been given about a three month head start to give feedback to the Company , I will see the actual filing date as extremely significant. It is the POINT OF NO RETURN AND MESOBLAST KNOW THAT ! I expect the PDUFA date given to suggest up to a six month timetable , but I also feel confident that with so many boxes already ticked from the previous submission, i would be surprised if we had to wait more than 3-4 months to learn the outcome…
If the submission addresses the issues raised in the Complete `Response Letter and the FDA are satisfied with the audit of Lonza’s Singapore manufacturing facility , we are good to go !
I think Mesoblast has painstakingly accumulated and supplied all the necessary data…they speak extremely confidently about their potency assay linking in vitro to in vivo outcomes. The have now backtested the patients from their EAP programme…to show the results over a much wider patient population.

I suppose , the FDA has achieved their pound of flesh. They can legitimately argue that they have set a very high bar for approval of allogenic MSc therapies having now received four year survival data. Mesoblast, having been counselled by Philip Krause, have taken more time than any of us would have liked ..but I think we are all going to like the end result. I am extremely confident .



Please do not take my recent absence from these threads as showing a lack of interest. I have been extremely busy with other tasks but have carefully followed developments . It was obvious that Mesoblast needed time to compile new data sets for the FDA…arguing the toss with a bunch of clueless down rampers on HC .. is not my style.
I am back because I feel the moment is nearly upon us. Good luck to all . OP



Please do not rely on the facts or opinions given in the above post when making an investment decision,