MSB 2022 - The road to commercialisation, page-1430

Ha … yes Techinvestor. But the one thing everyone can look forward to with some certainty is the GVHD resubmission with the biomarker analyses.

This produces a sugar hit with a high stakes binary outcome in “in short order”.

The FDA may accept the resubmission for review. This could be interpreted very positively for ultimate acceptance as it would tend to suggest that the FDA are willing to entertain the possibility that the biomarker analyses can reverse their current “go away and do an adult RCT” position.

The catch is other than JB1975 who is going to spend time drilling down to try and understand the probability of success for a biomarker study. The whole thing is inscrutable.

I think JB1975 makes a good point about the paucity of data – which is obvious because sub-grouping is everywhere - but it is also the quality … everything is confounded by patients receiving other treatments.

So I think people are going to fall back on a simple heuristic; we could call the dogma of correlations.

Companies that are good at clinical trial design, choosing endpoints, producing reproducible results that are accepted as methodologically rigorous by regulators will also be likely to be good at producing convincing results from their biomarker studies where the processes for establishing validity are less well established than those in clinical trials.

With the reverse being true. And so what do we think should happen when MSB travels less well established pathways? For example, where it is has to posit a hypothetical control rate in a single arm trial? Or where it conducts a registry MAGIC study? Dumpster fire in a train wreck springs to mind.

So that will be the general flavour I think.

The contrarian position is that the ODAC 9:1 vote must count for something and does provide some sort of anchor point for efficacy from which to attach your biomarker analyses. This anchor point was lost when the control group estimate and MAGIC study were rejected by the FDA.

FDA acceptance for review could be interpreted very positively as it would suggest that the FDA are willing to entertain the possibility that the biomarker data can reverse the “go away and do a adult RCT” position.

So like insurance - someone will take on the risk - at a price. The only thing that is perhaps certain is that the price will never be disclosed to the likes of us.