Replies to post #180818 on Biotech Values

[masterlongevity]

That explains why the rate of success in rare disease is so much higher, right? If $SRPT thinks their phase 2 data is good enough for acc approval, then why not use same incl criteria as ph 2? They can always exclude those not doing well once the trial starts(TIC). Companies like STPT popping up all over because single mutation disease easier to crack. Shorter R&D timelines, more lenient TX by FDA and EA, cheaper to develop, higher probability of success, charger whatever the hell they want.

I am not criticizing because it's a great ROI business model and rare disease needs focused research. But barrier to entry and prob of success is higher, and in my book, that means easier. Easier is a good thing so no need to be defensive about it

[iwfal]

SRPT

If you really think not much has learned in the past two years about trial designs from various Ataluren, Drisapersen, eteplirsen trials, well, I don't know what else to say except sorry I waited time. ... Drisapersen ph3 failure changed many things

By focusing on RNA and PTCT I would suggest you are acknowledging not much learned from their own trial per se. I would agree that drafting behind others does work. But it also means their own trials aren't very informative - not awesome for justifying approval.