@col69
74% of patients in this trial had already previously received ( and presumably become unresponsive to) Ruxolitinib ! Maybe the FDA’s obsession with T and B cell mediated mechanisms of action for GVHD needs refining. The efficacy of Axatilimab is attributed to targeting inflammation and fibrosis through the inhibition of disease associated macrophages. Shock horror. Has the FDA cocked up again forcing its view of MOA despite clinical results now showing an alternative explanation . Lets us also remember that in this trial "the median duration of response was not reached " and that only 60% of patients maintained a response over a period of 12 months.Now I want you to ask yourself. What percentage of patients treated with Ryoncil for acute sr aGVHD go on to develop medium to severe grades for Chronic GVHD? Second question, what results did Kurtzberg get when she undertook an investigator lead trial into using Ryoncil for patients with cGVHD? I suspect we are going to find out more detail very soon...we already know from the 4 year mortality data that tumour relapse and chronic GVHD percentages in patients from GVHD001 are likely to be much lower than current approved treatments.
I would like to make a general point in the lead up to the decision of the FDA regarding Ryoncil.
Many of you here might well be waiting for a positive announcement to “sell the news”. That is perfectly understandable and in biotechs is often a sensible strategy. The difference this time is that Mesoblast will , post an approval , start to generate decent cash flows . It is unlikely to remain independent if the financial markets ignore this inflexion point. My own view is that the share price movement might initially be quite constrained until the reimbursement pricing is agreed and whilst there is profit taking…but i genuinely see substantial upside on a 3-12 month view. I will elaborate in greater detail shortly ….I think people will be quite shocked as institutions do their due diligence and start to build up holdings in a first in class therapy. I suspect Mesoblast will apply for accelerated approval for severe grade (C/D )adult acute steroid refractory GVHD…and have a straightforward royalty deal to negotiate for entry into Europe. I believe they also already have some good efficacy data for chronic . My back of the envelope calculations suggest that it would take about 24-30 months to reach about a US$7bn market cap for Mesoblast.. just for this
one indication based on the likely addressable market . It would not surprise me if the Company starts signing royalty deals post BLA approval to repair the balance sheet and provide growth capital. If the back trial starts this quarter , that could well add $US billions to the market cap over a period of time as we approach various read outs. Do not ignore the fact that the previous trials will give them excellent clarity over trial design and the Company already enjoys RMAT status for back and CHF.
Post a successful BLA application , Mesoblast will have a market cap of US$650 at these levels for a first in class, platform technology, with most of the key foundational patents secure…which is plainly ridiculous. Sanofi bought Kadmon months after its cGVHD approval for almost $2bn.. I think exactly the same will happen to Mesoblast . I would prefer Mesoblast to restructure its debt and j/v with a major Pharma …this would allow the Company to accelerate is expansion of the label into many different orphan diseases. Let’s wait and see. OP
Please do not rely on the opinions or facts in the above post when making an investment decision.
... phase 3 trials under its belt; extensive MSC data; huge patent portfolio; etc etc ... what big pharma won't want to own the next paradigm shift in medicine ? At what price remains to be seen ?
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