Replies to post #215871 on Biotech Values

[bladerunner1717]

Clark,

Under what statutory authority does the FDA have the right to redact information regarding SAE's related to approved drugs?


Bladerunner

[mcbio]

SRPT -

And I would suggest the most interesting piece of this excerpt is what I put in italics since the two most common SAE for oligonucleotides are renal issues (almost universal) and blood disorders (although generally thrombocytopenia). In any case the FOIA results will be interesting - especially as the RCT populations get bigger.

Still assuming the biggest thing for SRPT is if their next gen platform (PPMO) gets around, or at least improves upon, any safety concerns for Etep.

Separately, there's still the GT programs as well. FWIW, I threw in the towel on SMMT's ezutromid as SRPT barely even mentions that drug on CCs and SMMT CEO has made remarks as well in CCs to the effect of potential concerns about whether the diagnostic they are using in the ongoing P2B will be adequate. Didn't really inspire confidence for me in the ongoing trial set to read out next year (they also significantly pushed back the readout of this trial which may well be another red flag).

[ghmm]

Charles Seife / SRPT:

I am really surprised you'd give this guy credibility! I stopped reading his article the first time because it lacked substance was pure tabloid. Sure you can infer conspiracy in everything if you like! He is just a "journalist" looking to make a story whether or not one exists. I skimmed through his other Scientific America and his style is similar pure conspiracy theory. He conveniently omits real facts like Sarepta's "plunge" caused by AE's and death was clarified as 3 deaths were ALL patients in their 20's I think 1 didn't even receive drug.

Specifically about the kidney issue. His reference is from study 201/202 given those kids are still on therapy its quite the stretch to say the drug caused kidney damage in them!

The choice of Journal alone should raise doubts about an rigor in his "analysis"!