A new Irish Trader? Hat tip to Paul C

[biopearl123]

Reposted from Paul C on Stocktwits:

“ I’m currently on this drug trial in the UK. I was diagnosed 4 years ago low risk watch and wait. 6 months later my bloods dropped and i started on weekly Epo & GCSF injections, these worked for around 18 months. I then began having blood transfusions every 4 weeks and then every 3 weeks, 2 units each time. I was then enrolled on this Imetalstat trial Jan 2023 . I have this drug via infusion every 4 weeks and since I have been on this drug my bloods have gone from 75 to 120 and holding steady. I also have not had any blood transfusions in 11 months. I had my latest Imetalstat infusion yesterday and all my bloods are holding steady. I have not felt this good or had this much energy in over 5 years. I can only speak for myself but this drug has worked wonders for me with very little side effects which my trial team have now erased with a few tweeks to my pre meds.”

[biopearl123]

I am assuming he meant a rise in hemoglobin from 7.5 to 12.0 gm. Pretty impressive.

[biopearl123]

This guy had to wait 5 years to feel good. The implications for earlier intervention and maybe front line may become obvious with more experience with the drug.

[kmall]

Bp - It was mentioned that this post on Stocktwits was reposted from the MDS Facebook group.

[biopearl123]

Thanks Kmall, missed the attribution. I am not on Facebook. I get enough flak from other sources.

[kmall]

I can't think of a better endorsement for the patient community then this. I would imagine that this sort of testimony spreads like fire amongst these groups. Simply incredible that Imetelstat hadn't received priority review or accelerated approval. -Kmall

[LWS]

"I have not felt this good or had this much energy in over 5 years. I can only speak for myself but this drug has worked wonders for me with very little side effects --- " -- from Paul C.

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In these days of misinformation, it is hard to know what is real and what is not. Assuming that Paul C. is real and can be properly vetted, his story should stand out as the 'poster-case' for why Imetelstat should be approved (NDA) now (post haste) by the FDA, as an extension of the limited use that is now available (EAP). There are likely many people with similar experiences. This would be an interesting discussion to have with John Scarlett at the upcoming fireside chat (B. Riley -- Thursday, 1/18/24).

[jayfish101]

And I was just ruminating about why we have not heard from patients who were granted access to Imetelstat. I wonder if Geron gets feedback on those patients.

[Ryan]

There is little doubt that this is a true testimonial from a real patient… given the details supplied about treatments before Imetelstat in particular.

And I wouldn’t see this as surprising either, the data we have all seen has shown this type of improvement. It is great to see a real story and live their recovery vicariously, it’s great to see!

Of course, the note on (little) side effects also encouraging given the doubts around that aspect.

[kmall]

Of course every patient and situation is unique, but it is noteworthy that this patient has an Imetelstat transfusion every 4 weeks for the past year. Do we have any idea how long a patient like the one described here needs to be getting transfusions, and do the patients transfusions diminish over an exceed period of time?

I know this is most likely an impossible question to answer at this point, but if the success shown here is indictive of Imetelstat's efficacy in patients, I would imagine Geron's manufacturing end will be extremely busy in the very near future. -Kmall

[biopearl123]

Kmall, I think I missed the point of your question. Maybe rephrase? Did you mean infusion in stead of transfusion?

[biopearl123]

Kmall, am going to assume you meant Imetelstat infusion. It’s a good question. As the bone marrow recovers (assuming the patient responded to treatment) and the blood picture improves I think you are asking if the time between treatments can be increased or the dose of Imet can be reduced. In looking over the slides in the past I was curious as to how many patients remained on treatment over time and why they would have stopped since the numbers of patients on treatment clearly go down over time. It seems that about 20% of patients have a sustained freedom from transfusion and about 60% or more have a substantial increase in hemoglobin, yet there is a reduction in the number of patients on treatment after say one to two years. So what we need to get at is, if their treatment is stopped because the bone marrow has normalized and they don’t need it anymore (this seems doubtful) or there are other reasons to stop treatment. These would include progression of disease, severe infection, transformation to H/R or AML or the development of other conditions of old age which would have happened anyway (heart attacks,CHF, peripheral vascular disease, stroke, dementia, who knows?) Or in some patients the drug stopped working or never worked in the first place. (?Time for a combination regimen which might increase efficacy and the number of responders.) In short I don’t know the answer to your question but we should find out as the data continues to be milked (transformation rates, longevity) and future publications address the issues you raise specifically.

[kmall]

Bp - yes to infusion.....my mistake. And I think you basically answered my question, which was poorly structured to begin with. It is difficult to come to any concrete answers on the subject since it's still a work in progress with each patient example different. Thank you for taking the time to sort it all out. -Kmall

[CKTC]

I’m trying to figure out how this individual could have started on imetelstat in January 2023. IMerge Phase 3 enrolled 178 patients between September 2019 and October 2021. There was an expansion phase that allowed patients to continue on imetelstat, which is still ongoing. However, this individual could not have been part of the main Phase 3 study. Recruitment for the Ventricular Repolarization substudy started in February 2022 and ran through September 2023. I guess this individual is participating in the substudy?

Regarding patients in the IMerge Phase 3 stopping treatment, 91 of 118 (77%) of those receiving imetelstat discontinued treatment by data cut-off (October 2022). If those patients were benefiting from imetelstat and had wanted to, they could have continued receiving the drug in the expansion phase. The likely reasons for their discontinuations are all noted by biopearl123.

[biopearl123]

Great question and the enrollment times don’t really work out. He could as you suggest be in the ventricular repolarization sub study which was separate and ongoing and perhaps subject to different enrollment dates. This latter study does allow for crossover from placebo if there is no benefit after two doses. There has never been an issue with QT interval prolongation with this drug that has been reported and the repolarization study appeared to be a clever back door that allowed later enrollment of more patients.

[biopearl123]

Dr. Scarlett confirmed today that the ventricular repolarization substudy is ongoing and continues to enroll. That could address the issues raised by CKTC re enrollment timeline.

[rccola335]

https://www.youtube.com/watch?v=8Mnyrt7bAQQ

what a strong endorsement for AML for a treated patient - i am looking forward to seeing that data

[biopearl123]

Thanks for posting this RC, definitely a little more detailed and “produced” (music etc.) and a beaming patient who seemed very grateful. Nonetheless devoid of actual detailed patient experience. We still have to wait for that but probably not too much longer. Lots of Cheshire Cat in the Lane group, they are clearly happy about something.