CEO Itescu needs to go, page-784

@otherperspective,

If I didn't believe Study 001 gave positive results I wouldn't be invested here. Nor have I "mixed up" primary and secondary endpoints. My comments were focused on the slide deck presentation to investors where the company, not me, chose to emphasize mortality reduction at 100 days rather than ORR at 28 days. While those results are indeed positive, ie a reduction in mortality to 26% from the null hypothesis rate of 43%, that reduction is nowhere close to being statistically significant let alone extraordinarily significant, with an N=55 study size. I posted a detailed analysis above showing why that is... if you think some other statistical method gives a fairer analysis, please post it - rather than accusing me of failing to use it. Note that my analysis grants the null hypothesis mortality rate - which rate also could be questioned since data were historical.

You'll also find no disagreement from me on using MAGIC biomarker scores to distinguish high-risk patients. Yes, it's a grading method. But that doesn’t preclude RCTs to analyze the response to an experimental therapy. A score above 0.29 becomes a criterion for entry into the study, much the same as a low PF ratio is used for entry into an ARDS study. Nothing there that prohibits randomization into a blinded study. I suspect that's part of the plan to be discussed at the Type A meeting for the adult study. .

The link you gave is worth a full read to get a better understanding of the FDA position on Mesoblast's data from all 3 studies.

Bottom line on GvHD - it's too compex an illness with too many variables to expect to prove efficacy with small non-prospectively controlled "studies".

You think the FDA treated Mesoblast unfairly... others have said FDA is capricious... I'm not so sure... certainly Krause was there to judge/navigate that and I think Califf was probably on our side given his training - but I'm willing to grant the possibility that all of you are correct. Let's say the FDA IS capricious, corrupt, under the thumb of big pharma and really has it in for stem cells sponsored by immigants to Australia who trained at prestigious US universities, whatever. Knowing that and it's indeed his job to know the lay of the land... what should SI have done as CEO on his LEAD indication for a first-in-category product when his data wobbled a bit from not being prospectively controlled ? Please answer that question - and send your binomial p-value calculation, we'll discuss it. Left-e