Re:BMRN endpoints article.revisiting KOL comments on BMRN bleed free vs. FVIII % data
I'm not sure if I have this right, and I think I knew this at one point, and then forgot about it reading all of ryno's incessant posts about the quality of BMRN's data, but:
If a trail is attempting to advertise ABR as a primary endpoint, BUT patients on the initial gene therapy had to resume prophylaxis, can the ABR that you report really be considered reliable or comparable to any other trial, because the resumption of prophylaxis is likely preventing bleeds that would have shown up?
I would argue it's pretty clear the resumption of prophylaxis for some patients has clearly improved the ABR for the BMRN data.
However, that doesn't mean the BMRN drug isn't working really well. The totality of the picture painted is showing great benefit. It's just that the ABR story can be misconstrued, and isn't the be all end all in a vacuum.
Go figure that ryno would latch onto this so tightly (not really - eye roll).
PFE drug will use half the AAV when dosed (3E vs 6E), ramp to stable levels faster (10 weeks vs 6 months) and likely have comparable factor levels, which are directly correlated to bleeds, resuming prophylaxis, and ABR).
Since the trials are not going to prevent prophylaxis treatment for data sake, ABR in the context of allowing treatment becomes skewed.
I think some nice milestones are going to be coming SGMO's way over the next 1.5 years as this trial progresses.