BLA 125706 Remestemcel-L FDA briefing document, page-16

The key difficulty as I said in my post of a few days ago is that MAGIC study was aptly named.

It turns out MSB didn’t provide a SAP to the FDA for this study and nor have they provided the data. The MAGIC estimate to bolster the control estimate in the single-arm study has appeared just like magic.

This will be a difficult challenge to overcome.

On the plus side the original control estimate of 45% was more rigorously derived than the journal publication for the trial suggested and what the Lassman comment would have you believe.

But the FDA are not happy with it; and basically, just don’t accept it.

I think this is potentially arguable. In adopting this position the FDA is very close to saying that trial was doomed right from start because the null hypothesis was not sustainable. In other words the trial lacked scientific integrity.

But this view was not shared by the numerous IRBs with independent experts who reviewed the protocol. So I don’t think the FDA can so easily avoid making an effort to interpret the result from the trial substantively. Despite its methodological weaknesses.

As you guys are discovering however, as with QRX, the communication between the FDA and MSB (which is kept confidential) is critical.

It appears that the FDA told MSB that the historical control estimate wasn’t satisfactory and that MSB should do a parallel group RCT in adults. But MSB have decided to chance their luck. I doubt investors would have had quite the enthusiasm here if that was publicly known.

Gut feeling.

Within 5 minutes of the FDA QRX AdCom briefing materials being made public US fund managers were receiving abusive phone calls from clients, RT of NEU fame had concluded the case was hopeless and LW was coming to terms with dropping $12m. The actual meeting was a waste of time; it was irretrievable; everybody knew that.

I don’t get the same flavour here; but very challenging.

I know we all have out for and against positions which we battle out on HC. And seeing a messiah effect with posts receiving 200 TU is enough to make any self-respecting contrarian jump out of a window.

But HC is not actually the real world. In the real world we should all be hoping for a success here; Australia can’t rely on digging stuff out of the ground forever; so getting smart is our best chance. I hope MSB can pull it off.