Ann: Mesoblast Maintains Momentum with FDA, page-67

Hi @Wilba32,

I was surprised that a number of posters thought there was either no or no significant information content in the announcement.

First, there was the confirmation that the Type B meeting for REVASCOR was held almost a week ago. It's no longer a matter for speculation - on HC or anywhere else. Well done Mesoblast. That means the relationship with FDA is working.

Second, there was confirmation that Mesoblast personnel were given by FDA the opportunity to 'discuss' face-to-face with relevant FDA personnel the preliminary responses from FDA i.e. to the questions that MSB was required to outline in its briefing package in advance of the meeting. The information value of iterative discussion at the meeting enhances the information value of the preliminary responses. Yes, apart from the outcomes themselves it's off-the-record and confidential (which is great) and yes, it went to the key elements of Mesoblast's 'moat' i.e. potency assay for commercial release, and CMC items.

Third, there was confirmation that the meeting addressed the primary endpoint and "design" for the confirmatory trial. This was critically good news, because it means that the FDA has formed and discussed with MSB its view of an acceptable trial endpoint to pursue in relation to an RMAT designated product. That could only happen under the umbrella of a discussion of the entirety of a proposed clinical trial, which in turn suggests that Mesoblast provided all the key elements of a clinical trial protocol to FDA in advance of the meeting, and everything from eligibility to controls to powering & so on was covered. And so would be able to be discussed by MSB in confidence right now with 3rd parties. Y'know - the prospective collaborative licensees who are going to fund it.

Fourth, there was confirmation that the Type B meeting was held under MSB's RMAT, which is indirect confirmation that the FDA continue to allow the possibility of Accelerated Approval of the BLA for REVASCOR when filed. If the Type B was not under RMAT (or a prior confirmation of AA availability from FDA) the assumption that AA remains an option immediately available to MSB wouldn't arise. We'd otherwise be assuming a need to pursue a lengthier standard approval process for REVASCOR. If there was any genuine remaining doubt on this score, the use of the word "confirmatory" stripped it away it. So, it follows that the FDA is prepared to align on everything required for AA & put REVASCOR into the US market "in short order".

Fifth, there was confirmation of "general alignment" on key matters that were pre-specified by MSB as targets for the Type B meeting. That's important because of the way that a "general alignment" arises with the FDA. Prior to the meeting the FDA holds an internal meeting with relevant FDA personnel to discuss the content of Mesoblast's meeting package and to gain its own internal alignment on the preliminary responses. Only then do they sit down with MSB personnel. If "general alignment" resulted from the meeting as well, then we should expect that MSB and the relevant FDA personnel are, at the very least, in agreement that the BLA for REVASCOR should proceed. Hence the corporation's undertaking to update the market on timelines for potential filing subject only to receipt of the FDA's minutes of the meeting in due course.

And then there was all the additional & quite separate & also very meaningful stuff about the upcoming RYONCIL meeting and commercial launch progress.

All good, as I see things.

Cheers
GLTALTH