A recent independent and open access paper from the excellent...

A recent independent and open access paper from the excellent Karolinska Institutet, published in Nature in May 2023 sums up the current mess CBER finds itself in .



“Multiple studies conducted around the world have shown an exceptional safety profile of MSCs and indicated their efficacy in treating GvHD. However, an assay that quantifies the in vivo immunosuppressive action of MSCs has remained elusive.

Clearly, clinical responsiveness depends on the interactions between the MSC product and host immune cells that take place in the inflammatory milieu of the recipient (Fig. 1). Implementation of late-stage trials with predefined clinical criteria has been difficult and has raised ethical concerns for patients with a lethal disease. In addition, such studies risk categorizing patients with similar clinical symptoms but vastlydifferent underlying biology into the same category, a possible explanation for the diverse reported outcomes. It could be speculated that patients with severe lymphodepletion due to GvHD have lost responsiveness to MSCs. An exhausted lymphocyte pool could potentially result from not only immune reactions characteristic of a particular patient’s GvHD but also multiple immunosuppressive treatment regimens prior to MSC treatment. We recommend continued immune profiling of patients both before and after MSC treatment to identify the patients who, as observed by many investigators, will clearly benefit from MSC treatment of GvHD.”


https://www.nature.com/articles/s41423-023-01022-z

In my opinion ,CBER has cocked up completely. They have approved a therapy in Ruxolitinib ,that approx 40% of patients (mostly with GI severe disease with the highest mortality rates )are refractory to and has limited median durability of response (11 months)…whilst halting the progress of a perfectly safe and effective alternative in Remestemcel….because they want an RCT, which would have compared patients that will respond to identical MSC treatment in totally different ways, dependent on their own internal immune responses. Meanwhile the Magic Consortium of teaching hospitals for aGVHD worldwide , offers a much more accurate proteomics based algorithm to separate the non responders with different biology…and it has been ignored.


Meanwhile, the comedy continues….are the FDA beginning to see the light ?

The FDA now re issues guidance notes specifically for steroid refractory acute GVHD stating it might approve a therapy from a single label trial.


Futhermore, they appear to have no problem with our updated potency assay. The foremost authority on acute GVHD, the BM CTN, is now prepared to conduct the Phase 3 adult trial on our behalf , funded by the NIH no less…….but still many of us are now understandably cynical and bitter from our investment losses In my opinion, we have a battle of egos with the FDA wanting to conjoin Mesoblast to a confirmatory trial with their sanctioned potency assay before allowing paediatric approval…to now save face.

See Page 2 of the Report and Accounts:





The Chairman’s message tell us there are “planned initiatives” to fund the Company. In a totally dysfunctional equity market the obvious first port of call is to try and securitise the revenue receipts from Temcell, to keep our heads above water and then top up with a small equity component , so that larger sums can be raised against a backdrop of regulatory approvals or licensing initiatives. The sums recently secured by Roivant for its promising Phase 2 therapy for Crohns/UC are breathtaking. Although Mesoblast has plenty of clinical work to do , the Phase1/2a studies at the Cleveland Clinic , particularly in peri anal fistula using our MSCs are in my opinion equally promising…although we may need to establish an extended treatment protocol to maintain remission rates.

https://www.biospace.com/article/roche-pays-7-1b-to-roivant-for-us-rights-to-telavant-s-ibd-candidate/#:~:text=Roche%20on%20Monday%20announced%20a,ulcerative%20colitis%20and%20Crohn%27s%20disease.

LAST BUT NOT LEAST….I don't want to spoil the party for the shorters …but I will make the following statement …based on publicly available information on the internet (which i will reference in due course, post publication of the findings ) , I believe we will all be talking about another small randomised controlled study …from a prestigious academic organisation very soon…I will not elaborate any further..but it makes me incredibly proud of this Company. Watch this space ! OP





Please do not rely on any of the facts or opinions contained in the above post when making any form of investment decision. Do your own research and remember that Mesoblast may well require additional equity funding to continue as a going concern. I could be the office cleaner so consult with your investment advisor.