The FDA has been taken to task by various industry bodies for...

The FDA has been taken to task by various industry bodies for failure to provide accurate guidance. The image above was a slide presented by Peter Marks and Matthew Klinker of the FDA, at a forum meeting in 2022. For those of you saying that Mesoblast should have done another trial because it was recommended by the first CRL rather than “required” , at least now have the decency to accept the guidance was at best opaque ! Read the unattributed commentaries of the industry participants who attended the forum whose names can be found in the appendices..they seem exasperated with the FDA.

https://alliancerm.org/wp-content/uploads/2023/03/Addressing-potency-assay-related-development-days-for-cell-and-gene-therapies-March-22.pdf

It is worth noting that Gamida Cell had received a CRL , slashed staff expenses and cancelled three clinical programmes before the FDA finally gave them approval. Maybe you should all read the review notes leading up to this approval (see link) . Look at the “unexplained mechanism of action”, the 37 patient size of the pivotal trial etc., etc
https://www.fda.gov/media/168198/download
Then look at the struggle faced by Kadmon Pharma to have its CGVHD product approved over so many years…despite very impressive results.
How can the FDA assign responsibility for CMC and potency assays to OTAT …then leave two years for Mesoblast to get technical sign off from this division …then allow Mesoblast to provide a further four months advance sight of the revised BLA before its submission so that any obvious deficiencies (such as shortage of data ) could be pointed out….before they then accepted the BLA submission …and then said it was effectively incomplete six months later because they (the FDA) had not set “requirements “ previously in a completely new area of regenerative medicine. It’s double standards over and over again with these clowns . They do not seem to mind that autologous senescent cells or allogenic cord blood can be used on patients with low success rates. When are they going to realise that it is the inflammatory milieu of each individual patient who receives allogenic MSCs in vivo which plays an important part in determining the efficacy of the therapy..not just the friggin potency of the individual cell !

Moving forward…Silviu has his work cut out. Why did no one want to talk about the accelerated approval pathway tonight ? Surely its the elephant in the room . Is this something the Company can formally ask the FDA for guidance on in the Type A meeting ? Or do they want to exhaust all other possibilities first ? Remember after the first CRL , Silviu suggested he would avail himself of the accelerated approval pathway.

The Company currently struggles to present itself as a going concern as it only has funds until Q1 24…but this is a Catch 22. If funded with say US$100m , IMO,the valuation appears absurdly low …without further funding, it’s anyone’s guess, until we know how the funding gap is resolved . Markets do not like uncertainty..but someone must surely step in and capitalise on the opportunity which presents itself. Lets be honest, if all Mesoblast had was CLBP…that’s enough to justify over a $1bn valuation right now ? as all they need to do is repeat the findings of a previous Phase 3 trial with a 12 month primary endpoint of pain…and this time they can limit enrolment to patients with less than a 5 year old condition which should boost overall success. Remember what Grunenthal was prepared to pay for a slice of other markets which still required their own trials !

This stock is definitely not for widows and orphans …but the cells most certainly are? I hope the Company secures the finding it needs because I sincerely believe in the technology. Good luck to all .OP


Please do not rely on the facts or opinions provided in the above post when making an investment decision