Far to knowledgeable and clever for you obviously Bazsa.
You might want to re check the Grade 3 patients who reacted best to treatment. Many CRs. MSCs work better in sicker GvHD patients is my view, and also supported by your own cherry picked trial.
Here's a good take for you to mull over from a once big MSB fan Left-E
"Stop blaming the FDA for management FU's. FDA rules, which are founded in US law, are very clear. If you want a new medicine approved, start by showing you can make pure product, then post two large, prospective, blinded, well-controlled phase 3 clinical trials in a specific illness that demonstrate safety and efficacy beyond 95% certainty. Same rules for everyone. There are shortcuts and alternative pathways, strewn with thistles, rockfalls and detours, for exceptional situations. Management of this company, for whatever reason, chose an alternative pathway - using children with an illness that occurs primarily in adults, using open label and historical controls, using product with shifting purity/potency - and then spent several years and $$MM protesting when the FDA asked for more data. They've raised over $1B from investors and incurred significant debt. That should have been enough to run the requisite focused trials. Focus on the problem, which is in the C-suite, imho.
@OP, I never said SI broke any FDA rules... but he did opt to follow an alternative pathway, a side route to approval. The foundation for that decision rests on cherry-picked peds cases from a larger GvHD trial run by Osiris that failed. And yes, the FDA authorized a protocol... authorization to run a study does not guarantee approval of results. Especially in a shape-shifting illness like GvHD where multiple other immune modulating treatments are used off-label. There's nothing cut in stone saying he had to pursue GvHD as a lead indication for a first-in-class treatment... but if he's going to pursue that with his new, improved formulation of rem-L, the first thing to do (not the last thing) is to reach agreement on the potency assay - so that data collected are not declared invalid. Then let it face off against rux in adults and resolve the efficacy issue for all to see. Rather than running an uncontrolled, costly EAP using children as a foil. Go back 5 years, go back 10 years. He made sophomoric decisions that led to 2 CRL's. A seasoned CEO versed in dealing with the FDA would have done better. The efficacy issue is still open to attack with his wobbly uncontrolled data, and he has yet to do what he promised, which is to run a controlled trial in adults. And where's the Crohn's data he's sat on for a decade? caveat investor."
CYP Price at posting:
32.5¢ Sentiment: Buy Disclosure: Held
