Cell Therapy News/Articles, page-775

RE 1/ Not withstanding the fact that MSB did not reveal the gist of the FDA meeting prior to the P2 LVAD results being released, it is clear the independent researchers were testing something the FDA never considered was clinically meaningful. You are quite correct, it was not a path to a BLA but then the trials were not paid for or designed by Mesoblast, the purpose was not aimed at creating a path to a BLA. I have wondered for a while what the MOU covers.....

"2) Reduction in GI Bleeding isn't a result for repair of the heart" no, it is a fix for a side effect of LVADs. But in terms of weaning

"For instance, among LVAD patients whose cardiomyopathy was due to ischemic events, there was a significant improvement in the wean rate (P=0.02). There was no significant difference among patients whose cardiomyopathy was caused by non-ischemic events."

Now whether or not weaning ability is a result of repair to the heart, well you can play with that how you like. Doesn't matter to the FDA, frequency of MACE is another matter altogether, as per the Mesoblast CHF P3 trial.

Also "patients whose cardiomyopathy was due to ischemic events" is said to cover a high percentage of those in the CHF P3 trial.

Re 3/ They aren't running it to prove how good the results of the last trial were, because they weren't. So has this confirmatory trial begun, has the hypothesis been put forward, or the trial design or the Primary and Secondary endpoints? I'd be quite certain the trial is not testing how good the results of the preceding 2 P2 trials were. I believe the confirmatory trial will be testing for GI bleeding outcomes, not LVAD weaning.