@ArtephiusLet me save your blushes .
Mesoblast already has an estimated three years supply of validated inventory for Ryoncil for use in paediatric sr aGVHD. It has been checked and validated for all the issues you have brought up. As an orphan indication there is no need to use mass production methods such as the 3D bioreactors which would be needed for the larger indications. Just look at JCR Pharmaceuticals who have commercialised our licenced IP over in Japan, for
paediatric and
adult patients for over five years with Temcell. JCR have been paying in excess of 20% royalties by my calculations and are still making healthy profits There are many granted patents which show Mesoblast is fully appraised of the latest science in micro carriers , hydrogels , not to mention ascorbic acid chains acting as xeno free alternatives for FBS .
Unlike CYP, Mesoblast have patient data.. lots and lots of it . The have also passed an FDA Pre Licence Manufacturing Inspection without any 483 being issued . The latter is probably the most important factor of all. It means Mesoblast has orchestrated numerous manufacturing runs to the FDAs satisfaction to required manufacturing standards. You have to bring yourself up to date.
Any reader with a modicum of sense , appreciates Lonza’s Singapore plant , is a world class facilty and has passed every regulatory inspection . Your basic level of criticism is not backed up by reality in the commercial world. There are many contract manufacturers of MSCs supplying to independent laboratories all over the world. To be fair to you , manufacturing is absolutely key.
According to Liu,S., Yang, W., Li, Y et al, in an article published in a Feb 23 edition of Nature who evaluated all the main six suppliers of FBS to laboratories…they found that different brands of FBS had varying influences on the background expression of IL-8, an inflammatory factor that has been extensively studied , but
not on the on the expression of
IL-
1b and
TNFa in epithelial cells…. owing to amino acid metabolism
https://doi.org/10.1038/s41598-023-29060-7
Mesoblast’s foundation patents make it perfectly clear our bone marrow cell immunoselection is based on extremely rare “bright” stromal cells drawn from the iliac crest. Once the FDA approve Ryoncil , expect to see a series of manufacturing upgrades come through with regards to ascorbic acid . Mesoblast has already patented its xeno free substitute which may increase productivity by up to fivefold. It is breakthroughs such as these which will give them the IP advantage in commercial exploitation when all the dust has settled . Indeed Mesoblast has spent many millions perfecting seeding densities to determine the appropriate cell confluence rates to optimise yields and potency for the very latest 3D bioreactors which will help drive down costs to below 10% of COGS. I doubt very much that they will stick with a single supply agreement as they will want to keep there supply chain honest .
In summary, I believe the points you make have been dealt with to the satisfaction of the FDA . There will always be room for improvement.
In my opinion , Mesoblast shares in the next 12 months are about to go on a run of epic proportions,
provided we are granted approval for the Ryoncil BLA resubmission. The latter is being used as a “bellwether “ indication for a whole host of inflammatory orphan designation diseases as well as some “blockbuster “ indications. The catalyst , for a big chunk of this rerating apart from establishing much needed credibility and validation…is that reality has set in at the FDA. Peter Marks should be given credit for reappraising the Accelerated Pathway so that it stimulates investment into unmet needs. This will allow early commercialisation of perfectly safe regenerative medicines who are falling slightly short of clinical trial requirements which are more appropriate for small molecules therapies which are generally immunosuppressive rather than immunomodulators. Regenerative medicine , has had the added burden of needing potency assays to ensure consistency of product so that every patient receives a uniform product regardless of individual donor profile . IMO,
if Mesoblast has finally cracked the potency assay issues it will become one of the most sought after stocks in healthcare portfolios. Within five years , it would not surprise me if the market capitalisation was closer to US$100bn. I know the latter comment will leave me open to ridicule but just remember the market for chronic lower back pain alone is at least US$ 3bn based on a lowball $15k wholesale price per outpatient procedure . If the FDA is serious about accelerated approval then they must surely apply that process to the best potential therapy for solving the opioid crises in the USA ! Go figure ! OP
Please do not rely on the facts or opinions expressed in the above post when making an investment decision. I am not medically qualified .
(20min delay)