Member Info for Haywain

If you had any sense you would not be posting "notposting" - anyway I don't want to affect your sense of entitlement in a public forum so I will leave it there.

DH-Hull - I hope so too : though if the timing doesn't suit - which it may not there are plenty of other opportunities that reach the same audience in the new year - Or maybe a new England journal of medicine publication?

Someone dose protest very loudly about a share they don't own. Anyway I would suggest that instead of asking A.I to counter what these nasty people are saying that you actually do some research of own. Go an take a look at what other oncology focused CAR-T share price did following external validation of positive early clinical readouts (DSMB being the first). I didn't say that DSMB would announce publicly but I would think it is fairly reasonable to assume that their conclusion verbatim might be RNS'd. The language is usually fairly bland but there are certain terms within that indicate responses across the dose cohort. I will leave it at that before you wet yourself.

Let it roll - that is exactly how I see it too - big pharma moving at POC early phase1 and increasing the upfront to a median of $300M and that is where Hemo CAR-T currently is - & DSMB confirms it .

Oh there you go - we have got the full set - wait until the efficacy is presented in black and white and then come and speak to me about apologies.

Aldebaran - you have been involved in this share for 35 minutes and shot-gunned so many diametrically opposed opinions out there that from the range of your scatter the probability of one being correct was actually quite high. I don't have to be right about timing's that is a traders game and I only offer an opinion when asked - I just have to be right about the science which I think you will find if you read back through my posting history everything that I have predicted (against much consternation from those short) has come to fruition.

If HG-CT-01 is performing as the evidence suggests, then once the DSMB issues its formal conclusions the market cap should move swiftly into the £150–£250m range even before any conference disclosures, licensing discussions, or term sheets. The reason for that is simple: it will be the first time efficacy can be presented at a cohort level with independent verification, putting the data in black and white and removing the “yeah? show me then” overhang. At that point Hemogenyx should be valued like a normal clinical-stage CAR-T developer rather than a speculative micro-cap as it currently sits. In my view.

One swallow does not a summer make - but 3 is time for emergency factor 50 ;)

I believe because of the tiny set it should be relatively straight forward and I would expect it to arrive in the coming weeks - ie around about Halloween/ Bonfire night - If Stu was not faceless I would make an effigy of him for the guy and enjoy watching him go up in flames whilst I toast hemogenyx and HG-CT-1. But in reality it can be relatively quick following data availability (day 28 for patient 3) - 3/4 weeks for data prep, committee review, formal &recommendation- so could come anytime really.

That’s because institutions are waiting for definitive, independently verified data — which will come via the DSMB’s forthcoming dose-escalation recommendation. As HFH noted, that moment will mark the first time the company can formally discuss efficacy at a cohort level. The logic is already clear: long-term remission in the first two patients, as confirmed by the company, implies eradication of leukaemic blasts. This was explicitly reinforced in the RNS stating that the third patient’s AML blasts were undetectable by standard methods. In other words, what was initially inferred from durable patient survival — deep clearance of AML — has already been demonstrated; we’re now simply awaiting formal, independent verification. Which I believe will open the door to the first of the institutional investors.

The only logical explanation for what we’re seeing in patients 1 and 2 is that HG-CT-01 has wiped out the leukaemic progenitor population. In FLT3-positive AML, relapse almost always comes from a tiny pool of FLT3-high, CD34⁺, CD38⁻ progenitors that survive conventional therapy. Drugs like gilteritinib or midostaurin can knock back the blasts but rarely touch that root cause — which is why remissions are fleeting and rapid relapse (weeks to couple of months) almost inevitable without HSCT.

HG-CT-01 is different. It targets FLT3 directly, not just on circulating blasts but on those progenitors themselves. If the CAR-T cells reach the marrow and engage that population, they can destroy the disease at its source. That’s the key difference between temporary blast clearance and genuine eradication.

The results speak for themselves. Patient 1 is now around eight months post-treatment and patient 2 roughly five and a half, both relapse-free without HSCT or follow-up therapy. In relapsed/refractory FLT3⁺ AML that is simply unheard of. The most plausible explanation is deep molecular remission and durable CAR-T persistence — exactly what you see when a therapy crosses the line from effective to curative. I believe this is the signal that FDA saw to grant paediatric inclusion following treatment of two adult patients - land mark decision can only be justified by landmark data.

If these remissions hold past twelve months, it’ll be very difficult to argue that HG-CT-01 is anything less than a potential cure for a subset of FLT3-positive AML. The data are already rewriting the rulebook — the market just hasn’t caught up yet.

Yes Vander - it is not the DSMB’s role - they review the clinical safety data and efficacy data and perform a risk benefit analysis that leads to a decision to dose escalate or not - that is it.

Just to be clear - it was actually pre-clinical I had assumed phase 2 because of the 201 tag - but no BMS paid 1.5 billion for a preclinical asset and platform.

No worries Tennantry - if you want to understand how collaborations between a biotech sponsor and a research hospital actually function. In simple terms, Hemogenyx owns the product, the IND, and the core clinical data generated under that IND, because they are the regulatory sponsor. MD Anderson acts as the clinical site and scientific collaborator under a contractual research agreement. They don’t own the data generated as part of the defined clinical protocol, but they do have academic rights to analyse, publish, and explore the results, provided this is done in line with the sponsor’s oversight and confidentiality obligations.

Where it becomes a little more nuanced is when MD Anderson conduct additional or exploratory testing beyond what was originally defined in the protocol – for example, deeper molecular analyses, immune profiling, or follow-up assays that they decide to run as part of their own scientific curiosity. In those cases, MD Anderson technically own the data arising from that additional research, because it wasn’t part of the sponsor-funded trial deliverables. However, such data can still be shared with or used by Hemogenyx if MD Anderson permit it, usually under a data-sharing agreement or with the sponsor’s acknowledgement in publications. It’s a collaborative balance: Hemogenyx retain ownership of the core clinical dataset and all commercial IP rights, while MD Anderson retain academic ownership and credit for any supplementary findings they generate independently.

If Dr Short or his team were to identify a new mechanistic insight, a biomarker, or a potential expanded indication from their additional research, that discovery would typically lead to co-authorship and scientific recognition for MD Anderson, while Hemogenyx would hold any commercial rights or file joint IP where relevant. The physicians and institution don’t receive direct financial reward from the data itself – their benefit is academic prestige, reputation, and future research opportunities

DH- are you asking about OTX-201 rna liposomal -T-Cell activator for B-Cell lymphoma ? If so different indication and in pahse 2

Tenantry - I don’t know why the concept is so weird to you. MDA are a research hospital that perform clinical trials in oncology. The clue is in the “research” part thier plaudits (and funding) require them to conduct world class research - they get rewarded based on the quality of the papers they write and the presentations they deliver at large conferences. An early discovery of a new oncology agent that is showing signs that it might be better than the standard of care is career defining for those involved.

Well - anything in addition to the protocol is MD A’s bag - personally I would be quite surprised if Dr Short was not doing MRD testing (not defined in protocol) as he is a world renowned expert in MRD testing.

The answer to your question is both do actually- Hemogenyx owns everything that is defined by the protocol. However the protocol does not state MRD-ve testing in relation to efficacy it states morphological assessment per Doner et.al. That is not a complete assessment of efficacy. My belief is that MD A are conducting much more sensitive tests in the background as exploratory endpoints (not defined by protocol) in which case they would be funding these tests and MDA would own the data. I say this because although the morphological testing is expected by regulatory requirement - additional tests are commonplace and far more sensitive and predictive of patient response : Flow cytometry and quantitative PCR can detect down to 0.1% - if MDA (which I suspect) have and own this data then it ca not be used by hemogenyx until MDA release via a publication or presentation. Doner et al can only test to < 5% blasts which in the context of FLT3 +ve r/r AML is meaningless - shows some form of efficacy “encouraging signs of efficacy” deep sensitive tests are required to predict MRD negativity and or complete molecular response.

Within the next 2 weeks would be my guess but could be any time now

Mickey - DH-Hull is talking about the CLN shares when they come to market - will obviously dilute a little bit but crucially they are in sticky hands - as sticky as mine are.