First of all congratulations to all MSB holders! All well deserved outcome for shareholders for sitting tight and hopefully accumulating more after the SP plummeted to below 30c and participating in the CR!
What we have now witnessed was in my opinion a political move rather than one that was based on science as we have seen it in the past (based on CRL1 transcripts in the public domain that laid out what the FDA was expecting to see). I suspected that, hence was happy to enter into a bet with you
@JB1975. I can't fault your science approach and you were right twice, but this time was different. I have posted some re-Marks from Marks, director of the FDA’s Center for Biologics Evaluation and Research (CBER).
The approach we have now witnessed is likely the new Marks-FDA version of the Japanese Sakigake designation:
1. Is it safe? Yes.
2. Is there an alternative therapy on the market? No.
3. Are there signs that this therapy COULD help these patients? Yes.
= Move straight to Go and collect your BLA approval.
You can see that the FDA still wants to see more evidence, but you get approval first based on on the original 54 patient single arm P3 trial:
https://www.fda.gov/media/184605/download?attachmentI called it "saving face" at the time after CRL2 and you picked me up on that phrase, JB. The writing was on the wall this time.
What investors will hopefully realise, the Marks-FDA-Sakigake way to obtain approval bodes well for other therapies including Cynata's approach.
Our safety record will be on the record (SR-aGvHD, MEND, DFU, OA fully enrolled without any safety concerns reported, HR-aGvHD so far). We have seen sogns of efficacy in SR-aGvHD and the company is optimistic that we see good results in HR-aGvHD.
There is however an approved therapy (no, not Ryoncil, but Rux - with horrible side effects).
Now that the heavy lifting is done by MSB and a someone in charge that is putting more weight on future potential of a therapy, and given that we are running a multinational randomised clinical trial at a much lower MSC dose per patient than what has already been approved now in SR-aGvHD, with a lot less side effects compared to Rux, maybe, just maybe, we can be quietly optimistic that if the results are stellar, approval could be possible after randomised P2 trial.
We will see if the market picks up on it or of I am simply delusional and missed jumling the ship years ago.
(20min delay)