Replies to post #142493 on Biotech Values

[exwannabe]

...this is not unheard of for disease with serious unmet medical need, I can think of one example. The initial SPA of ipilimumab was for a single-arm study demonstrating 10% response rate.

Yeah, response rate is OK in a single arm.

PFS (and even OS) is not because there is no way to evaluate it.

I can certainly find an example where the FDA would not even consider PFS data for exactly this reason (in a very similar setting). Can anybody name a case where a drug was approved on PFS single arm?

[BTH]

Heck, even CRIS/RHHBY got pushed through with a small 100 pt open-label Phase 2 in mBCC, aBCC.
Certainly not unheard of, if they target a specific unmet pt population.

They need a real partner for this though; if they do it alone, I can really see them screwing this up badly - heck, they couldnt even check the right box at ASCO.