Ann: Quarterly Activities/Appendix 4C Cash Flow Report, page-169

@Blackkkkkkk@Diane325 @reginaldp Many thanks for the links and information. I'll subscribe and listen.

Blackkkkkkk, I think it must have been your post I remembered. (I didn’t give you a TU but I fixed that.) I think SI saying few children are non responders is important because he’s not given to overstating the efficacy of our products and has shown an understanding of immunology over the years. (IMO one important reason we attracted PK and GG.)

I'm not saying revenue from paediatric aGVHD could be a billion US this year (or maybe even next) because it will take time to ramp up sales but there's nothing approved for under 12s and nothing effective for gut GvHD. Just a year ago, members of the forum I belong to were asking if anyone knew of an IV product for gut GvHD; @Sven62 You mentioned Axatilimab, which has recently been approved for cGvHD. Do you understand the difference between chronic and acute? Patients are told it can take up to months to see a difference with this drug.

SI said at one AGM the price was high because of being in the steroid-refractory position. Surely it figures the price would be higher still because many patients will also have been treated with Rux? Those referred to the EAP have been in a very poor state. That’s why I think it likely that a number of children - I don’t know how many - will require more than a full course. I also believe the incidence and severity of aGvHD are underestimated for several reasons, which I’ll go through in a later post.

Based on the company estimates (which IMO are conservative), the cost of treating a child with sr-aGvHD in ICU can be around a million US. That’s why, before pricing was announced, I estimated up to one million reimbursement. @Stellowe Yes, I saw the interview SI did with Alan Kohler. That’s the reason I thought the reimbursement could be significantly larger.

I don’t know much about US insurers but I’ve been told they’re notorious for trying to avoid paying out; however, there’s a context here. Ryoncil is curative. It's not just SI’s opinion but (I didn’t know this) he said there are teams that work out the true economic value of such a drug. This bigger picture, together with SI’s statement that there are very few non responders if they’re provided with more prolonged use, has IMO any potentially reluctant payers tied up. I think they’d have got the broad message anyway.

I believe that’s why Peter Marks was pushed out. It’s about doing right by children. Stellowe, you also tagged me about the interview with Marty Makary. Was it the one with Megyn Kelly? There’s a lot to unpack there but relevant to this post, Makary said Marks pushed out the two top vaccine experts (not his opinion but on record). They had concerns over mandates and repeat dosing, particularly of children, for a virus that John Ioannidis (whom Malhotra calls “the Stephen Hawking of medicine”) estimated had a low infection fatality rate in people under 70. Makary commissioned a study of 48000 children which found no deaths from Covid of a healthy child under 18. These data were supported by studies from Germany, Sweden and the UK:

https://www.nature.com/articles/d41586-021-01897-w

In that same interview, Makary mentioned Professor Vinay Prasad (who’s written several articles praising Dr. Krause). IMO Marks is just a pawn but his fate was sealed when he approved mRNA boosters last fall for babies as young as six months and Prasad vented his fury on X.

I think there will be support for a company that has done right by children from the get-go. (I joined the dots before Dr. Krause came to us. Having worked out what this revolution is fundamentally about, they were the obvious ones to join.)

Finally, I just wanted to give my two bucks worth about our CEO. I watched the Bloomberg interview and I’ve never seen one person do so much in such a short period of time! SI got his shovel out and dug a deep moat around our company. He took his cue and brought out the halo effect, expanded it and threw it around the moat. Then he hurled some large rocks (I saw the beginning of the end for the mabs and nibs) SI stressed over and over the consistency of our product. Every patient gets the same product. Not stated but shown. Every. Single. Vial. (subtext: as opposed to…Dr Krause is not just up on immunology but also a stickler for manufacturing consistency)

SI was pressured to state the likely revenue for Ryoncil. He said he’d expect the cost incurred for treatment would be reimbursed by major healthcare providers. Carol could have been giving her opinion, but I rather think she was delivering a message: “It will be”

Keep going Silviu