Member Info for Haywain

Just based on timing - but if I were Vlad I would be pretty keen to get it out there.

Thanks pawnking - alternatively try next week - first dose cohort update

I view most of the market movements as noise - try and find another company developing a CAR-T for AML with any degree of clinical success for 20-30M : you won’t. they are all in the 100’s M- so I am comfortable to wait and allow the market/ big pharma assign the actual value through a licensing deal.

London South East - complicit in aiding and abetting the snaggle toothed Tom Winnifrith - surely not….. ? Does have a bit of a putrid whiff about it considering we are trialing a potentially curative CAR-T in terminal paediatric cancer: not sure that is a good look.

Well at least for patient 1 &2 the probability for surviving without relapse for 6M and 4M respectively and without HSCT to consolidate treatment is very low in r/rAML to the point where CR and or CMR is probable - median time to relapse with FLT3 positive r/r AML (if not consolidated by HSCT) is counted in weeks to 2-3 months (Pearl et al. (Blood 2017 & NEJM 2019 quzartinib/gilteritinib studies) not 4-6M or 6M+ even if treated with FLT3 inhibitors. I believe the explanation for this is in the FLT3 targeting and the fact that AML leukaemic progenitors & stem cells (the cells that cause the relapse) also express FLT3 and therefore are also cleared by HG-CT-01. If that is the case then expect remissions to continue. Perhaps the surprising aspect of this is that not only does it appear to be demonstrating efficacy, it also appears to be doing so with a clean safety profile which is unique with respect to any other CAR-T attempted treatment of AML - as usually associated with significant side affects. If the above is true and we see a similar profile in patient 3 - then it becomes groundbreaking and undeniable as soon as the data is published. FDA at least believe this is plausible otherwise in my opinion they woudl not have green lighted paediatrics after only 2 adult doses. This is potentially the data set that will be available next weds/thurs 3/3 safety & 3/3 efficacy ? . I suspect MD Anderson will keep this under wraps for a big splash late breaking submission to ASH.

Thanks Pumpky- that has always been my hope and why I have defended the attacks over the years - most of which didn’t have grounding . Anyway have a good weekend.

Pumpky- That argument is completely wrong – NICE do not apply some arbitrary £50k ceiling, they assess cost per QALY gained, and in end-of-life settings that threshold goes up to £50–60k. Every CAR-T already on the NHS (Kymriah, Yescarta, Tecartus) costs in the region of £250–280k per patient and all were approved because the benefit relative to relapsed patients with no options was so dramatic. HSCT, which is the current “gold standard”, already costs the NHS £120–200k up front, and that’s before you add years of expensive complications such as GVHD, re-hospitalisations and repeat procedures. If HG-CT-01 really is curative in a proportion of patients without the need for transplant then not only is it clinically superior, it could actually be cost-saving for the NHS, because one infusion avoids both the upfront transplant cost and years of downstream management. So the idea that patients would need to pay privately is simply wrong – NICE will absolutely assess it through the same pathway as every other CAR-T, and if the early results hold, it will walk straight through on cost-effectiveness grounds.

Great question Mr Jinx - I believe the paediatric starting dose will be calculated on body mass - ie same number of cells per kg - but I am not entirely sure as this isn’t my area of expertise - I have however seen this approach been taken in other trials

Stu on this board is like a recurring malignancy — always trying to make the company sick. And just like a teratoma, he surrounds himself with a backing band of toxic cells that at first glance may look like normal posters, but are nothing more than cancerous growths in disguise. Think of him as the lead singer, fronting Stu and the Teratomas — same tired tune &same false notes

Just as HG-CT-01 must succeed by not only debulking AML blasts but also eradicating the leukemic progenitors and stem cells while sparing healthy HSCs, so too will the malignancies that have lurked in the deep niches of this board be brought into the light and cleared- just a matter of time.

You can apologise when you read it in a company RNS - suspect it may come shortly after the licensing deal is announced.

I am not going to do this much longer because your rambling waffle is not worth reading . But It’s precisely because no other anti-FLT3 CAR-T has produced transplant-free survival with clean safety in r/r AML that Patient 1 is so significant — pointing to median survival stats or CD7/CD19 trials in very different biology is a red herring. The company must use cautious regulatory language, but the facts are simple: a patient with FLT3 positive r/r AML is alive and well beyond six months post-treatment without HSCT or toxicity, something unseen in this indication before. That is not “ramp,” it’s observable clinical reality, and it’s why the FDA has already approved paediatric expansion after only two adults. If that isn’t game-changing, then what exactly is?

One for you to ponder - how can it be “ramping” to request an entirely independent emotionless (robotic) independent assessment of the facts that we know them to date - through inputting these HG-Ct-01 facts into A.I and for A.I to come up with exactly what I have been saying for the last 5 years . Hmm - ramping?

is it like the last 50 papers you have posted -? because if so not interesting at all and largely irrelevant where the conclusions i came to . but whatever - i can’t stop you being a ******** so go head

We will just have to wait for MD Anderson’s new paper about HG-CT-01 to be published - then I will happily post the link in here

I can’t post a paper, Stu, because in CAR-T for r/r AML nobody has yet witnessed this type of clinical profile — which is exactly why it’s a game changer. The literature shows median survival in this setting is usually measured in months, and remissions typically require an allogeneic transplant to hold. Here, you have a patient alive beyond six months, with a clean safety profile and without HSCT. That isn’t something you’ll find in a prior paper because it hasn’t been achieved before. By definition, this is new ground.

And if you don’t belive Mr I or me for that matter then ask A.I - in the case of FLT3 positive r/rAmL is it normal for a patients to have extended survival past 6M following treatment with an anti-FLT3 CAR-T and for those patients to have a clean safety profile and not require HSCT. Ask it if this survival continues how much that might be worth to big pharma and how that might change the treatment landscape of r/r AMl

I don’t need to point you to an RnS Stu I can point you to the statistical probability of a patient with FLT3 positive replaced refractory AmL surviving past 6M without HSCt to consolidate - should there not have been the efficacy I am describing

Thanks for the post but the comparison doesn’t hold. The ASH 2024 CD19 CAR-T trial you cite was in the very rare AML subgroup, where some patients aberrantly express CD19 – this applies to maybe 2–3% of all AML cases at most. Even in that tiny niche, most responders still went on to allogeneic HSCT, so the OS/LFS outcomes reflected transplant consolidation rather than durable CAR-T activity alone. That is exactly the pattern across prior AML CAR-Ts: CRs that look impressive on paper but relapse quickly without HSCT and are often accompanied by serious toxicities. What makes Hemogenyx different – and why CRs in a small cohort are game-changing – is that their first two patients are alive and well months out with no HSCT rescue, no CRS, no ICANS and no cytopenias, and the target, FLT3, is expressed in a much higher percentage of AML patients including the leukaemic progenitors that drive relapse. That combination of breadth, safety and transplant-free durability has never been seen before in AML CAR-T, which is why this data cannot be dismissed as “just another CR.”

since you continue to bring this up - avacta is essentially a reformulation of an ancient chemotherapy product doxyrubicin adding their proprietary precision linker to make the treatment more directional and therefore safer and potentially more efficacious - it isn't a potential cure - still requires lengthy clinical studies because the benefit is incremental regulators expect larger longer studies to show statistical improvements in survival or reduced toxicity when compared to standard doxyrubicin. ****genyx's offering is in an entirely different league its a state of the art novel cell therapy in an area of catastrophic unmet clinical need - if it shows deep remissions with clean safety (as early data suggests) it qualifies for accelerated regulatory pathways - proof of concept can be established in just a handful of patients because the signal to noise ratio is enormous (curative responses where none are expected) - avacta's endpoints are progression free survival (long term) hemogenyx endpoints may be measurable in 28 days to 3 m (cr or mrd-ve) - regulators can move very quickly on orphan/ultra rare diseases where responses are unambiguous and also impact paediatrics. so please as there is no comparison stop talking about avacta on the hemo board.