Lots of tidbits from the Stifel conference

[biopearl123]

1. “Potential” is no longer a modifier. Statements as to the disease modification aspects of Rytello apparent, other treatments are palliative. Rytello has the capability to change the course of the disease including reducing risk of transformation to AML.

2. Moving up the line to 2nd and 1st line. Greater acceptance and understanding of “benefit” of cytopenias.

3. Continued growth and addition of new prescribing sites.

4. Interim analysis a possible mid term catalyst. This is a big one. Dr. Eid has removed one area of anxiety, namely the risk of crossover contamination of the BAT arm: crossovers capped and programmed delays related to wash out and time to actual crossover. This should minimize the compression of the K-M curves. At interim two paths, stop for efficacy continue to conclusion, nothing new there but RWD does help in anticipation. Risk of futility stop almost nonexistent. Patients are living longer in the study but we don’t know if this applies to both arms equally or one arm differentially.

Very positive presentation.

[rccola335]

thanks for all you do - getting frustrating and losing interest - we could really use some good news

[biopearl123]

RC, thank you for the kind words. I have to agree some good news would be most welcome. I do think the interim analysis will give just that.

[biopearl123]

RC, was thinking about your comment and thought I would try to send a few words of encouragement. You have plenty of of company. Some posters frustrations have been directed at me privately. My response always has to be that I don't give investment advice, people can make their own decisions and deal with the consequences. Clearly "the faithful" have curtailed their posting here and while the reader numbers remain high, sometimes it does feel Sisyphean and a bit isolating. But as you know I will continue to moderate until I feel I have kept my promise to Fish Sr and honored the work he put into this board years ago. OK. Having said that, here are the words of encouragement:

1. Documented reversal of bone marrow fibrosis
2. The interim analysis in the second half, I put the most weight on this: we know that the study will not be stopped for futility, that would have happened long ago. That leaves two choices: A. it stops for efficacy or B It continues to completion because of a favorable trend. Both are good one obviously is better. Here's why you won't have to wait too long. The RWD from Kuykendall looks like a HR of .5 if confirmed that should get us over the top. We know from Dr. Eid that crossover effect is accounted for and minimized. The study has not reached interim as we have discussed either because patients are just living longer, that can only mean they are living longer in the BAT arm, the imetelstat arm or both. We have a pretty good idea of how to calculate EOS using the Dr. K data and estimate why the study is taking so long including factoring in the slow early enrollment numbers. Mr. G and Ryan carrying the ball for us here. And here's the last part of the argument. The results at interim are so important to the company and its future that if the interim is positive they will have to announce right away, maybe not with specifics but if the study stops for efficacy that will be enough reason for renewed enthusiasm for the stock. So a long winded word of encouragement, if the study is positive, you will know right away. If it's promising they probably will still have to announce but soften the language. In any case it is unlikely that they would wait for ASH to announce if it's positive and then they would get specific at a meeting. So even if the deadline for late breakers is exceeded (unlikely), we could and likely would get almost immediate word. Anyway it's unlikely you will have to wait too long from here. So let's all take heart and know that soon we will know if we made a colossal mistake or an excellent investment decision that will change treatment of MF. I neglected to add that Dr. M is looking at front line and that should also go in the plus column. Best Wishes, bp

[rccola335]

it has been a much longer road than i ever expected - i feel the delay in a european partner/launch is because the plan is to sell and that will happen after the interim analysis - histology will be immensely important

[Ryan]

The story is never ending, but the narrative has yet to change. Imetelstat / Rytelo is safe and efficacious in MDS and MF (and ET, let’s not forget that near 100% CRs). Remains to be seen in other hemes and other disorders.

I read how Mark Cuban sold his Bitcoin because “his narrative” changed, or his last narrative didn’t come to be. That *still has not happened here. It seemed so whne Janssen bailed, and that was the time to get out and lick wounds, but crazily the narrative had not actually changed, and some strong clinicians (hat tip drs Rizzo, Feller, Scarlett) proved that out.

So here on the cusp of Summer2026, the question remains, am I correct in my narrative? Frustratingly, the answer is crystal clear.

This remains a sooner or later thing. For those who are trying to time it, much respects to that. I plan on timing it wrong until I timed it right.

[biopearl123]

Ryan, other than presumed improving sales and market penetration, "timing" is preordained and binary for the MF PIII study and none of us can know when that last patient triggers the interim analysis. I do think that this will have to be seen as "material" to the company and be followed by a PR if the (not public) trigger is met. We just don't know when. As Yogi said: "it's difficult to make predictions, especially about the future." Good luck with timing, let us know how you do!

[Ryan]

Good Yogiism , my timing is to hold and hold (and hold). (That’s why I’ve been wrong, and eventually will be right.) Been buying more, that’s why I am psyched up a bit. Never thought I would be able to accumulate down here.

$820 Million market cap. Won’t be for long. MF interim readout will be positive, as you noted, will be half or continue. Both are positive.

There are also about 10 investigator sponsored trials out there. As Yogi also said, “it’s getting late early”.

[biopearl123]

At least one of those trials has to be Dr. Bruedigam’s xenograft lab animals which were living a long time on combination therapy. We should hear an update at ASH.