Biotech Values

[iwfal]

RTRX AUPH

Will they (RTRX) try to file for accelerated approval based on this data? it seems fairly strong and a rare disease

And my original response:

BTW - I'd give it 30% chance that in an outcomes trial (e.g. Time to dialysis and monitor of OS) that Sparsentan does worse than SOC. The primary argument wrt protenurea benefit in FSGS is an argument based upon MOA-like arguments or natural histories. And that is, IMO, is too tenuous an argument to fully counter all the history of worse outcomes with more drugs, despite lower proteinurea.

Just another followup to this as I sweep through recent nephropathy data - the recent AUPH results in Lupus Nephritis is yet another example of proteinurea benefit (as created by drug) mapping very badly to clinical outcome. The same arm that showed stat sig proteinurea benefit vs placebo also showed much worse mortality. A very different etiology than diabetic nephropathy and yet same issue.


With some more thought I would actually up the chances that the RTRX drug actually induces clinical harms to well over 50% - and my guess is that they can see some of them in their short, very small trial (my guess is a GFR spike the wrong direction). Really proteinurea has to one of the worst surrogates for drug benefit in common use in published papers anywhere in medicine. There are very good reasons that the FDA hasn't, to date, routinely allowed it. MDs and paper writers like it because it is quick and easy - but clearly almost always misleading.

BTW - What is so intriguing about the Vadadustat results is precisely that the data so far released really is very very different than the norm.