CEO Itescu needs to go, page-385

@Zenox,

You said, "SI shot down use of a placebo and said he will check with the FDA about an adequate control group. Neither of these is consistent with an RCT".

It's why we have to be careful with what SI reports back. The issue is whether controls have to be prospective, randomized and blinded as typically implemented in a phase 3 RCT. To remain blinded the study has to be placebo-controlled, because you have to put something in the infusions received by the control group. If not rem-L, then... a placebo (ruxilitinib is given orally). However, in adults rem-L could be pitted against Standard of Care which includes FDA-approved ruxilitinib. So, that might ease the ethical concerns that arise in children where there is no approved therapy. You might want to read the FDA publication, "22 case studies where phase 2 and phase 3 trials had divergent results" to get a feel for why the FDA is probably going to require a phase 3 RCT for the trial to be pivotal. The publication helps explain why cobbling together 3 small studies that relied mostly on non-prospective, non-randomized, non-blinded or historical control groups or other forms of "RWE/RWD" didn't cut it with the FDA for this illness. And probably won't going forward in the future. And yes, info from CRL #2 is needed by investors. It appears we're not going to get it.