It is interesting that the FDA meeting happened on 21 February, and they received the minutes back in less than 2 and half weeks. This is very unusual. Usually FDA wait a whole month before releasing minutes. This surely means another meeting is imminent (I think in the next week) for aGVHD.
Now I believe I understand what Dr Perin is talking about, when he says "if we get approval":
https://youtu.be/qm9uQGHs0y4HiPlease listen to above video from 56:30 to 59:30
The end-stage HFrEF with left ventricular assist devices (LVADs) indication was covered by RMAT. The company went to the FDA under RMAT. This is an orphan therapy designation that shall now very likely get accelerated approval without further studies. The MOA for REVASCOR (Rexlemestrocel-L) was confirmed to a high degree of certainty in the DREAM HF trial.
Repeating what I have said before, the HUGE indication is the ischemic class II/III HFrEF2 patients (with high inflammation levels).
Now they shall almost certainly find a partner to fund the DREAM "TWO" HF trial with the goal to get earlier approval based on a surrogate endpoint. Dr Perin is talking about 2-years, which sounds about right. The FDA and Mesoblast must first be aligned on the surrogate endpoint, which should be the next step:
LVEF improvement at 12 months should be an appropriate early surrogate endpoint. Targeting the key mechanism of HFrEF which is inflammation must be a major priority. Understanding mechanisms that drive HFrEF is a key objective of the FDA, and Mesoblast has now provided the FDA with results from three randomized controlled trials in class II/III HFrEF and in end-stage HFrEF with left ventricular assist devices (LVADs), which ALL support the idea of a common mechanism of action (MOA) by which rexlemestrocel-L reverses inflammation-related endothelial dysfunction and reduces adverse clinical outcomes across the spectrum of HFrEF patients.
This is a pivotal year for Mesoblast and I sincerely hope they strike a huge partnership deal for CHF. Remember that Mesoblast reported that effects on LVEF and MACE outcomes were even more pronounced in 301 HFrEF patients with high baseline levels of inflammation as measured by hsCRP. This should be the Data that pushes the FDA to approve the trial with the surrogate endpoint and recruitment criteria of high baseline levels of inflammation, as the data showed that increased LVEF preceded long-term reduction in major adverse cardiovascular events (MACE) and associated recurrent hospitalizations for non-fatal heart attack or stroke.
What’s a partnership worth for a Multi Billion CHF Blockbuster indication?
Mesoblast spent hundreds of millions on the prior trials and research, so I would say a $500 million usd upfront payment is reasonable and then additional payments subject to certain milestones and then a double digit royalty on all sales. With this payment, Mesoblast can fund all their other trials and programs for future indications.
Please do not consider the above as investment advice.
(20min delay)