MSB to dispute FDA finding in Type A meeting, page-300

Thanks @stockrock and @Zenox

Wondered if this might also be of interest

https://www.bio.org/sites/default/files/legacy/bioorg/docs/Clinical%20Development%20Success%20Rates%202006-2015%20-%20BIO,%20Biomedtracker,%20Amplion%202016.pdf

Amplion Clinical Development success rates 2016

On p12 and Figure 5 this describes the time to approval and percent approved for New Drugs Application (NDA)/Biologics License Applications (BLA). You can look at the break down eg for haematology which would include aGVHD.

This shows clearly that success rate increases from 60% upon 1st review, to 76% and 90% with 2nd and 3rd reviews.

The comment is also made that the introduction of expedited approval pathways and associated interactions with the FDA such as Accelerated Approval have contributed to faster approval.

This suggests that it is very likely that following the path suggested by Si should have a good chance of success.However, I have always been more convinced that the alignment with the need for a COVID-19 therapeutic would put pressure on the FDA for approval, if the phase 3 is positive, as GVHD as a small niche approval, with the lack of a control arm, would be unlikely to be sufficient for them to want to turn over a whole new area of medicine to a small Australian biotech.

It goes without saying that it would be great if the 45% read-out of the COVID ARDS trial is positive, which is due at the same time as the 30d Type A FDA meeting, although we don’t know that yet, and we may be waiting for the full trial results until Christmas according to Si on Friday. Of course, Si will be hoping to discuss COVID data with the FDA at that meeting, as then he will have a placebo controlled trial to discuss and may well be able to address a number of translational questions, based around TNF, proof of product quality, as well as efficacy.

But there are a number of additional questions that come to mind which I don’t think have been raised although I might have missed them, apologies if so:

In my experience, price is an issue that is relevant and can be part of reimbursement authority decision delays but is never made public. Prices tossed around in the HC forum (I have no idea what prices are being discussed or implied at the FDA) seem to be on the higher end for Rem-L. Given that there is a pandemic and that there are multiple potential indications to follow, even though there are also a number of products and price tweaking could occur in the future, it is possible that price wrangling could also be part of what the FDA want. I am curious as to why that has not been raised at on HC? Certainly it has not been mentioned by the FDA or by Si or by any official body, but that is to be expected. But surely it could be part of the mix, for such a novel approval, for something pitched to change medicine forever? The FDA will be aware that MSB are bringing a line of products for which they hold a strangle hold on the IP till 2040 (discussed in my next post). This must impact price negotiations.

Thanks for your insights

Another post to follow regarding intangibles.