Member Info for Haywain

The DSMB doesn’t only look at safety in a cellular therapy trial. It reviews all available clinical data: laboratory markers, marrow findings, adverse events

& emerging signs of efficacy. If anything had deteriorated clinically the DSMB would not have authorised dose escalation.

So yes “preliminary efficacy” is the formal regulatory wording, but the biological reality of AML means that maintaining remission for months without HSCT already represents meaningful activity at the lowest dose. None of this is speculation — it is simply what is publicly disclosed, interpreted in the context of well-established AML clinical behaviour. undetectable blasts within the safety window for patient 3 - sounds like complete response to me - which is as good as it gets.

"The efficacy part of the the study is 'preliminary efficacy', meaning does it even work? It may be that at the lowest dose the treatment doesn't show any efficacy, but might at higher doses. " - It could be that (could it - really?) that would fly in the face of what is known about the disease biology of FLT3+ve r/r AML. Patients do not go more than a few weeks/couple of months without relapse especially if they are not receiving HSCT to consolidate the treatment. The only way that happens is if your have removed the root cause of relapse ie leukaemic stem cell/ progenitors from the deep bone niches - and if that is the case it is a curative level of signal not just a marginal improvement on the standard of care. The fact that DSMB gave them the nod post 3 patients also means its safe at this dose level - which means no cytopenias : ie it is selectively killing leukaemic precursors without killing haematopoetic stem cells/precursors - that is exciting and its exactly what happened in the chimeric mouse model all those years ago and why it was predicted to do so in humans.

i can infer that the clinical trials are going well because i understand r/r aml biology - patients do not go on to durable (beyond a few weeks) remission free survival in the absence of hsct - in order for that to happen you have to control or remove the leukaemic precursors/ stem cells. in addition in order for that to happen without prolonged cytopenias it has to selectively clear the leukaemic compartment without impacting the normal hsc’s. this is the only logical conclusion even in a small data set this signal should be apparent so your arguement about low patient numbers is bull****. roll on efficacy read outs.

Look at that share chart and tell me it reflects anything about the science. It doesn’t. It reflects one thing: a coordinated effort to walk the price down so the right people can get in cheap before the real HG-CT-1 data hits daylight. If the programme were failing, there’d be no need for the psychological warfare, the forward-sold warrants, the daily deramping, the synthetic drops, or the panic-baiting. This level of manufactured pressure is being applied because underlying asset is too valuable to let retail hold it at a fair price. The share price chart is a distortion -the clinical reality is the opposite IMO

You do realise that because we own the IP for HG-Ct-1 it can be converted into an in-vivo CAR-T right ? The fact is we have a functioning anti-FLT3 CAR-T in clinical trials were there is very little competition and it appears to be working.

“ I realise it totally destroys HG-CT-1 but you won't care about that will you.” totally different indication : B-cell malignancies - but don’t let that get in the way of your fear mongering - stick to insider trading thanks

They’ve already lost on the science, and they’ll lose on the financials too — because the two are inseparable. That’s why they’ve been screaming into the void every day on here. Clinical data rewrites the entire narrative. Tick-tock.

Some of the clearest evidence of Insider trading I have ever seen Not posting and Aldebaron should be locked up - They have been signposting some event for weeks and low and behold this turns up which they see as the big opportunity to trade. Criminal is the word I would use. Anyway makes no difference to the science and the probability HG-CT-1 will change the treatment landscape of AML. I see the IG shorts closing now - the rats running away

Yes Alde 😆

The main difference between the two in my opinion is that Hemogenyx is a "science first, communication second company" and Avacta is a "communication first science second company" - the differential can only be proven by results but once printed you will see the difference between the two - it will be crystal clear.

The charts don’t tell you what is on the FDA’s desk / being viewed under NDA. I am more than happy to wait for the verified data to be released to market- which won’t be long, tick tock.

Well one invests in potential and I happen to agree with Pingu - I see similar potential to what he intimated.

Think split dosing is allowed in theory but must be specified in the protocol and clinical investigators brochure - so I think we would have to do a protocol amendment before that could be done with HG-CT-1

It’s also possible that we are already close to the optimal dose, and that the next dose level will simply confirm this if response kinetics begin to plateau. It’s important to remember that CAR-T cells typically expand 10- to 100-fold in vivo following administration as they encounter their target antigen . The magnitude of that expansion depends on both the fitness of the patient’s T cells and the abundance and density of FLT3 expression on leukaemic cells. Consequently doubling the administered dose does not necessarily translate into a doubling of total in-vivo CAR-T exposure or activity nor does it equate linearly to the various adverse events like a small molecule chemotherapeutic agent might. So we could be at optimal dose and if so a dose escalation will confirm it.

Name them then

I wouldnt worry -Per the published AML CAR-T literature to date, I am not aware of another programme that has publicly reported a DSMB (or equivalent) authorising dose escalation after only three treated patients at the starting dose. That makes HG-CT-1’s DSMB decision notable in the AML CAR-T landscape. Historically AML CAR-T programmes have required larger safety cohorts or extended observation periods because of concerns relating to myelotoxicity, cytokine-release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), and prolonged cytopenias. So DSMB's dose escalation decision is actually early proof of concept that HG-CT-1 is a) safe and b) Demonstrating objective biological activity , consistent with target engagement of FLT3-expressing leukaemic progenitors and stem cells per its intended design. In short its doing what it was intended to do. This is inline with the FDA's landmark decision to greenlight paediatric expansion after reviewing data from 2 adult patients - which is also a first.

I hear your "years away comments" and agree that it can be the case for certain programs (not naming any in particular), however I would counter that with the fact that Hemogenyx isn't generic - It is sitting on a de-risked first-in-class CAR-T in an area where any hint of durable remission is front-page material. It is clear that Vlad would do a deal - the only question mark is how long can Pharma afford to wait - if a curative signal is released publicly there will be immediate interest from multiple big pharma.

It’s like a real life example of the children’s psychology experiment where children are offered a single sweet now or if they can wait 5 minutes without eating the single sweet they can have the whole packet. Surprising how may of them can’t resist taking the single sweet. I would rather eat the whole bag at my leisure but each to their own.

What has been achieved to date versus any comparitor CAR-T company in this indication tells me that you could add a zero to the market cap without blushing - everything else is noise until the results are released.

Because Vlad is in talks with big pharma - which means data rooms and NDA’s.