Member Info for Haywain

aldebaran - with all due respect ; **** off back to your 50 year old doxyrubicin packaging company - no comparisons can be drawn to a ground breaking cell therapy and your ancient chemotherapy- end of

You got me boom - there are no flies on you

I thought Stu had me blocked anyway - well that’s what you said yesterday: As pingu said - I said it was plausible that if you meet the clinical end points that you could arrange a type B meeting with FDA and push for phase 2 initiation - which is true: I didn’t say that was going to happen as it is always better to do dose escalation : more that they could decide to this - anyway I am not the one jumping up and down : I stand by what I say - and admit when I am wrong

My thoughts are that patients are alive is as much as the company can say until MD Anderson release the efficacy news publically - I would say though that the fact that the patients are alive - already exceeds what one might expect under normal circumstance - remember that no HSCT was delivered to these patients - Typically patients with FLT3 positive r/rAML have very poor outcomes particularly without HSCT to consolidate the available front line treatments. I, like you would love to see actual efficacy data but I am afraid we will have to wait - It’s all looking very good from my perspective

It should be very obvious Stu to all who read this who or what you are by now - however the RNS reads to me like paediatrics and adult population will immediately move forward in parallel following confirmation of the safety period of the first patient - so unfortunately for you no public apology needed - you sanctimonious pr-pick

i agree mickey - i just can’t help myself ; i can barely contain my excitement sometimes - will try not to post unless i see bull****.

I get the Avacta comparison — both have unprecedented early data in diseases with no effective standard of care, yet the market cap doesn’t reflect the potential. But there are key differences. AVA6000’s results are mainly stable disease with some partial responses, which is promising, but it’s not the same as deep, durable remission. Regulators still need larger, later-phase confirmation, so FDA fast-tracking isn’t realistic yet. HG-CT-01 in r/r AML is playing in a far harsher arena — median survival is only 4–5 months without transplant, and relapse is almost inevitable. Durable remission in even one patient without HSCT raises eyebrows; here we have two consecutive patients with sustained benefit, one very liley on path to MRD-negative and the other vlikely on the same path, both treated at the lowest dose with no dose-limiting toxicities — unheard of for AML CAR-T. The market’s muted reaction in both cases comes down to lack of a pharma partner so far, early-stage small patient numbers, and general investor unfamiliarity with how unusual these results are. The difference is HG-CT-01 already has a major piece of regulatory validation — the FDA has expanded to pediatrics after just two adults, something that doesn’t happen in AML without a benefit–risk profile that’s overwhelmingly positive. In terms of commercial potential, Avacta’s AVA6000 in metastatic salivary gland cancer might realistically be a ~$250M/year product if approved, with additional upside from expansion into other tumours. HG-CT-01, if it achieves curative potential in r/r AML and expands into earlier lines, paediatrics, and other FLT3⁺ malignancies (like BPDCN and subsets of ALL), could be addressing a $3–5B/year global market. Yet Avacta is valued at ~£200M for a program still proving clinical impact, while Hemogenyx sits at ~£6M despite having potentially paradigm-shifting data in one of the hardest cancers to treat. That disconnect won’t last once a partner or licensing deal validates the science — HG-CT-01 isn’t “further behind,” it’s arguably more de-risked in its field than AVA6000 is in solid tumours, and positioned for a deal well before Phase 1 is complete.

"The FDA are notoriously slow" after having granted paediatric extension after 2 adult patients? Have you heard of the RACE for children Act? the US law passed in 2017 and implemented by FDA in 2020 - Every FDA acceleration possible will be granted to the paediatric AML trial.

It comes down to indication and modality : Aldebaran, in most oncology indications you’d be right that two patients wouldn’t mean much, but relapsed/refractory AML isn’t “most oncology.” This is one of the most aggressive and difficult-to-treat cancers known, with median survival of around 4–5 months after relapse without a transplant. In FLT3⁺ r/r AML especially, even with the best available targeted agents, long-term remission without HSCT is vanishingly rare. That’s why when you have a first-in-human CAR-T in AML with two consecutive patients showing durable remissions so far without any follow-up AML therapy, and on top of that the FDA expands the trial to include pediatric patients after only those two adults, it’s extremely unusual. In this indication, you don’t need 30 patients to know you’re seeing something outside the normal clinical pattern — durable remission in even two late-stage r/r AML patients without HSCT is already statistically extraordinary, especially at the lowest CAR-T dose. That’s why there’s “fascination” here: the signal is already strong enough that big pharma will take note well before this gets to commercial stage, because they know how rare these outcomes are in this disease.

Well 28 days completes the safety data set - I presume efficacy will be observed from day 14 (peak T-Cell expansion) onwards - observed in the sense that bone marrow tests (at least morphological per doner et al) are completed at day 14, day 28 …. Etc -if the third patient follows the trend of the first two and clears safety : that for me is significant

Yes Pingu - it is fair to say that P3 is a big deal 😂

Let me tell you a little bit about the significance of P3. With Hemo CAR-T, the first two patients were both treated at the lowest dose in a first-in-human AML CAR-T trial, showed zero CRS, zero ICANS, zero cytopenias, and are still doing well months later without HSCT — an almost unheard-of safety and durability profile in relapsed/refractory FLT3+ AML. These results were strong enough that the FDA already approved a pediatric expansion after seeing only these two cases, which is itself extraordinary. However, two patients, no matter how impressive, can still be dismissed by sceptics as a statistical fluke. A third patient with the same depth and durability of response closes the minimum 3+3 safety cohort, confirming that the dose level is safe and expansion-ready, and shifts the data from anecdote to pattern. Statistically, going from 2/2 to 3/3 deep remissions in this population reduces the probability of results being due to chance from 0.25% to 0.0125% — a twenty-fold strengthening of the evidence. For big pharma, this can be the tipping point from interest to urgency, as it provides a reproducibility signal with almost no safety risk and an efficacy profile never before seen in AML CAR-T. In short, P3 is not just another data point — it’s the moment the story moves from exceptional promise to statistically undeniable, regulator-validated, and potentially deal-triggering.

Not what I said HT - but you believe what you want - how about that?

There are quite a few plausible reasons actually HT - infection being most plausible - infection needs to be treated and lymphodepletion repeated - interventions (these are patients at the end of the day) interim treatment could have knocked them out of the protocol inclusion criteria window - these are very sick patients and each one is different to the next and the medics running the clinical trial will make the best decision with respect to the patients specific circumstances

You believe that P3 has “sadly deteriorated” because you want that to be the case to support your narrative. Which I have to agree with JHFH is rather sick. I believe what you believe is irrelevant as has been proven time and time again. One of these days you will get it right but it’s a bit of a shot gun approach in the hope that at least one pellet hits your target - A company developing life saving products for terminally ill patients soon to include paediatric patients.

It isn’t delsusions Boom - I am quite grande

I rest my case

Yes Boom - correct I didn't know anything about the financial side - But unlike you I have a brain that is still capable of learning - I am in fact a scientist but i had to learn about the financial side because there was zero correlation between the science and the market cap - In spotting this anomaly it peaked my interest.

As far as I can tell, Hemogenyx main missteps have been financial, not scientific. They floated too early without sufficient financial backing and have at times shown financial naivete - Particularly in sharing sensitive information with individuals they believed they could trust, who turned out to be toxic traders exploiting that trust to manipulate the market. Such as Stu - who knew when and the price of every placing and traded accordingly. These are financial misjudgments. They say nothing about the scientific merit or potential value of the treatment being developed - Which for me is what it is all about.

In fact - to point to how ridiculous this has become: Hemogenyx could very soon meet their Phase 1 Clinical endpoint with just three patients treated. The third patient could be dosed at any moment - and if they follow the same safety and efficacy trends as the first two , that would represent a statistically and clinically meaningful signal. If that happens hemogenyx could reasonably request a type B meeting with the FDA to discuss initiating a Phase 2 study or fast track development depending on the durability and depth of response. This isn't just theoretical - its supported by precedent in rare disease trials, especially where early safety is clean and efficacy is unusually strong. You tell me in what world is their current 6-8M market cap justified when you have a look around at the competitive landscape. You cant undose a patient once they have been dosed - the data is real and getting stronger as time moves on.