GVHD in US, page-52

Silviu Itescu

Yes, Mark, first of all GI bleeding and hospitalization is not a surrogate endpoint. It is a key clinically meaningful endpoint that the FDA has identified as very important and a major unmet need in these patients. So the real question is whether we have sufficient data in two trials that have been confirmatory of each other. One with a post talk analysis the 30 patient study. And one with a pre-specified analysis in 159 patients.

We'll be sitting down with the FDA and have exactly that discussion with a full dataset in front of us. I think the key elements here are that it is a major unmet need it's probably the number one cause of morbidity in this patient population. The patient population per se is an orphan one. And so it is not easy to reproduce these large trials in such an orphan population. And the results have been very confirmatory and very similar in nature. And so I think that will be the argument.


The question then will become what kind of -- if we're fortunate enough to have a positive outcome from the discussion with the FDA, what kind of post-marketing obligations would we have. It could very well be another confirmatory study or it could a registry study. And those are all options under the RMAT designation.

Source : Mesoblast (MESO) CEO Silviu Itescu on Q1 2019 Results - Earnings Call Transcript