CEO Itescu needs to go, page-110

@Phaedrus,

Yes, I understand that SI would like to do a "quick study" on adults with the highest risk of mortality.

The FDA standard for approval of a new medication is generally successful completion of two large prospective randomly controlled phase 3 trials demonstrating safety and statistically significant efficacy. (Yes, there have been exceptions with other meds in unusual circumstances) ... But doing "studies" that rely on historical controls or some other shortcut rather than prospectively and randomly assigned controls is pretty much what landed us in the current predicament. So, I'm not sure I take much comfort in the plan going forward.... especially knowing that SoC and the profile of patients with aGvHD is shifting as advances are made in bone marrow transplantion and new meds come on line. We were led to believe that additional data from "studies" involving MAGIC (the Mt Sinai aGvHD Consortium) would be adequate to get approval in children. That turned out not to be the case. So the question remains - whether an RCT or whether a controlled "study", why wasn't such a plan instituted after the first CRL? ie Why was the risky "well-established dispute resolution" route chosen. And Why oh why didn't it succeed?

Strike 1 was the first CRL. Strike 2 the Novartis fiasco.... I gave benefit of the doubt on both, though I would have liked to have had better explanations for what went wrong. Now this black eye and an additional 60% hit on market cap. This was totally botched and it's just not acceptable to say now we're going to do the study we should have done 3 years ago. All programs seem bogged down and stuck. As an investor I count on management to communicate clearly, make good decisions, and get results... No one is irreplacable. Time for the board to take action and bring in someone with a track record of getting new meds approved. Such people exist.