Member Info for Haywain

Actually the mice are more than 'humanised' they are 'Advanced peripheral blood Hematopoietic Chimeric mice" ie they actually have a human immune system rather than the normal 'Humanised' mouse that are transplanted/ engrafted with human mononuclear cells. The significance of this is that they are far better at predicting unwanted adverse human immune responses to therapeutic agents as typical humanised mice suffer from graft versus host disease ie they are permanently trying to reject the human engraftment which means it is difficult to read actual signals related to the immunogenicity of the therapeutic agent over the underlying GvHD. Which in fact is why Hemogenyx developed the mice in the first place they are a much better prediction of how a therapeutic agent will interact with human immune system.

Https://www.youtube.com/watch?v=LFH95Zw8e6Y&t=1168s - Worthwhile having another watch

The regulatory consultants that Hemo have employed to write the IND submission have over 70 successful IND's under their belt : I think they will know what they are doing.

That last comment is in particularly poor taste 1:1- have a word with yourself ; it’s just money.

May I just add - I do this for a living : I have 4 INDs currently ongoing + 2 that are approved and commercial products in similar indications ie I have been through the process several times. I have seen phase 1 through to phase 3 and onto the market with the caveat being I work on biotherapeutic proteins (not car-t) . I am saying that I like what Hemo are developing and the timelines are not too surprising to me. I also know that most senior people in the company need to be reigned in for practical reasons i.e Vlad leaking his expectations without understanding practically what needs to be done from a regulatory point of view. I don't see any scientific reason why this doesn't work and if the FDA accept the IND then presumably they don't either - delays are due to dotting the i's and crossing the t's : These things take time but if it works and I don't see any reason why this will not then the rewards are big. Wishing you all well and chill out!

I think most majors would normally wait until Safety data is available at least probably also initial efficacy- but the pre-clinical CAR-T space has been quite hot recently so I wouldnt rule it out altogether

Nice to see that all of the speculative misery over the last month or so was all for naught. Onwards and upwards towards IND approval and being a clinical company :)

I could do with a good news RNS - nice little paddy's day treat

Who knows - Maybe a partner to be announced with IND submission :)

Looking forward to the pot of gold

Condoms - those charts dont make any sense to me either- I find it hard to believe that a preclinical asset would be worth 500m and drop to 75 m in a year- its like random noise generation unless you know how the data was selected behind the chart - so would take it with a pinch of salt. I do however have no doubt that Hemo-cart is worth 100million plus if it makes it through phase 1. First things first IND submitted - IND approved (or at least not object to) would be a start. Best of luck all! H

might be Wuxi themselves given that they developed the lentiviral vector but it could be any number of medium-big pharma companies

notice the reports from the video are in threes - this looks like the batch data from the third party test reports : I would say that we are now very close. IMO

A month here a month there is absolutely no time at all in the biopharma world - it will go in when it goes in. I had always thought end of Q1 at the earliest. Any sooner than that would be a bonus

Also the need for stability data can be the kicker - I dont know for CAR-T's what is required here but they have to demonstrate that the product would be safe and effective during any storage periods that might be involved in the products clinical use - this normally means storing under defined conditions with interval testing to demonstrate that you understand how the car-t's retain their expected quality profile over time in the proposed container: Shouldnt be rate limiting for the IND as I imagine they will have done this with PQ 1 and 2 - they will put PQ3 on some form of stability but probably they will just file the PQ3 release data in the IND.

It is a little bit more involved than the document you shared hedge on the CMC side because they will have a separate module 3 for the lentiviral vector as well as the CAR-T process and analyses but we are talking 100 pages tops most of which could be written ahead of final PQ data which presumably we have now received. Essentially the IND would be about 10th of the size of the final BLA package. They will want to cross all the T's and dot all the i's though so it does take a bit of time to get right especially being their first one.

Havn't sold a bean - Would rather have heard more news before placing but hey ho the money is needed and hopefully this should give us a new platform to build from. Onwards and upwards.

HFH - if Hemo release news that the CAR-M works in-vivo I am all in as they say- hopefully soon! All the best

You are right - I was more thinking in terms of recruitment to the study being relatively rare disease- However with Van Besien as MD I am sure that recruitment will not be an issue. Either way it is on the verge of being clinical and that is good enough for me.

I wasn't sure if it would be donor blood for the PQ batches for the purpose of demonstrating that the process is capable prior to IND filing with AML patient samples following IND approval - The CAR-T itself can be frozen and stored for months at a time so it is possible that the PQ batches are actually generated from AML patient samples. Interesting nod from Vlad to the article from Novartis saying that they are focusing on a 7 day process rather than allogenic (off the shelf) CAR-T cells- Novartis would be a good partner for Hemo CAR-T and seem to be betting that the patient specific process (as per Hemo CAR-T) is the way forward.

Perhaps vlad is giving a clue that they are actually working on Patient samples for the PQ batches which means that we should be literally dosing patients as soon as the IND is approved - that would be great : think JHFH suggested that this might be the case before