@Bazsa,
You raise several interesting points..
"...however without data to support going with adults investors would not have given SI the cash needed."
I'm not sure how you reach the conclusion there's no data to go with adults. The company has RCT data from ARDS, Crohn disease, CHF and CLBP. All involve adults. Never has the company announced abandonment of any of these indications. They could have raised funds to continue studying children, and pursued one of these other indications as LEAD indication for a BLA filing. We've lost years and $$millions advancing an indication with poorly-controlled data. Nothing is cut in stone saying childhood GvHD had to be advanced first when it was.
."The problem with the potancy assay has been the major issue."
The company has never said this to my knowledge. They've told us "more data has been requested, which we are providing"..
"... no option other than put a stop to all development." / "... and actions that were in effect a cinical hold" Really? This is news to me. In October, 2022 the Company said this when they updated their IND file: "“The submission summarizes
controlled data providing further evidence of remestemcel-L’s ability to save lives” said Dr. Silviu Itescu, Chief Executive of Mesoblast. “Additionally, the improved process controls we have put in place to assure robust and consistent commercial product, together with a potency assay that predicts consistent survival outcomes, makes remestemcel-L a compelling treatment for these children.” See MESOBLAST SUBMITS NEW INFORMATION TO FDA IND FILE...
That doesn't sound like a company with a clinical hold, which is a serious matter that suspends all ongoing clinical trial activity and has to be reported to shareholders as an 8-K event.
"They could have said that we failed to connect our potancy with out comes and go do more trials to prove the connection. They did not." Well, based on the above quote from CEO Itescu...they did collect more trial data, though the origin and nature of the "controlled data" are obscure. Your assumption that it came from 001 is not stated anywhere.
"Could I suggest that 2 perhaps 3 batches found with a strong connection to positive survival data is not what the FDA would call strong data supporting the CQAs of Remectemcel. It is however good data." From where are you getting this?
On August 4 the Company said this: “FDA’s inspection of our manufacturing process resulted in no observed concerns, the Agency raised no safety issues across more than 1300 patients who have received remestemcel-L to date, and acknowledged improvements to our potency assay."
There's nothing in there about a clinical hold. But if there was, resolution of CMC issues could have been obtained without filing an ill-fated BLA#2 for childhood GvHD. Companies do it all the time when there IS a clinical hold. They satisfy the manufacturing issue, the clinical hold is lifted and the IND re-authorized. That never happened here, or if it did it was hidden in violation of 8-K rules. All evidence points to a CRL#2 rejection due to insufficient efficacy data, not CMC issues. Inexcusable. imo, Left-e
(20min delay)