Chimeric Therapeutics Limited (ASX:CHM)
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May 8, 2026, 4:10 PM AEST
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Investor Update

Oct 8, 2025

Operator

Followed by Q&A. If you have a question you'd like to submit, please do so using the Q&A function within Zoom. Presenting for Chimeric today, we have the CEO, Dr. Rebecca McQualter, and CMO, Dr. Jason Litten. To begin, I'll hand it over to Rebecca.

Rebecca McQualter
CEO, Chimeric Therapeutics

Thank you so much, Matt, and thank you everyone for joining us today. We wanted to have this webinar because we really wanted to connect, contextualize the announcements that we put out last week. I'm really delighted to be joined by my CMO, Dr. Jason Litten, all the way from California. Thank you for dialing in so late, Dr. Jason.

Jason Litten
CMO, Chimeric Therapeutics

My pleasure to be here. Thanks, Bec.

Rebecca McQualter
CEO, Chimeric Therapeutics

A disclaimer really quickly. We will be making forward-looking statements in this presentation. We've recently just had a webinar, so I'm going to skip through some of the corporate things. As you're aware, we have four Phase 1 clinical trials running over three different assets. We've got a fantastic leadership team, and I'm really proud of our achievements with a small and nimble team, what we're able to achieve. We've also got a few first-in-class assets. As I mentioned, a small but mighty leadership team, and I'm missing my Head of Manufacturing there, who's now completed 10/ 10 manufacturing runs, which is fantastic. We've all worked on all of the listed cell therapies. Obviously, as you're aware, I've grown up in the cell therapy sector here in Melbourne. I'm really delighted to be joined by Jason today.

Just a quick refresher, we are going to cover CHM CDH17 and CHM CORN-K today. We won't be giving a chlorotoxin update today. With that, I'll hand over to Dr. Litten to present our updated CHM CDH17 results.

Jason Litten
CMO, Chimeric Therapeutics

Yeah, thanks, Bec. Just to bring everybody up to date, this is our really exciting clinical stage program targeting CDH17 with an autologous CAR- T cell asset. We're currently in Phase 1, and this is the first and most advanced CDH17 directed CAR-T cell therapy that's ongoing in clinical trials. We have four really prestigious, top-notch sites that we're collaborating with to advance the program: Sarah Cannon, University of Pennsylvania, University of Chicago, and the most recent addition, Emory University. Our IND was initially cleared in November 2023, and our clinical program was launched in mid-2024. At the end of last year, we achieved, I'm sorry, in the middle of this year, actually, in April of this year, we achieved fast track designation from the U.S. FDA. We have our orphan designation that's currently under review.

As Beth mentioned previously, we've attempted and successfully completed 10 manufacturing runs that were GMP compliant and have delivered an investigational product that's advancing our ongoing clinical trial. That clinical trial is currently open and recruiting patients with gastrointestinal malignancies. We've recruited 10 patients, eight of whom have already been treated. I'll describe them in the coming slides. There are two additional patients who we look forward to treating in the coming weeks. We intend to complete Phase 1 and initiate our Phase 2 activities after enrolling and treating 15 subjects. We're approaching that really pivotal moment for the clinical program that'll really drive us into a potentially registrational opportunity. This is the study schema that describes from left to right how we intend to pursue a registration for this program.

Currently, we're in the 3+3 Dose Exploration Phase on the left side, where we've demonstrated the safety of 50 million cells given as a single dose at dose level 1. We've advanced to dose level 2, which is 150 million T cells that are modified with our CAR-T construct. We've now demonstrated that that dose level is also safe. The dotted lines there represent an opportunity for us to continue exploration at dose level 2, which is an opportunity that we're now considering, or go to the next higher dose level, which is dose level 3, and continue dose escalation at that point. We intend to enroll additional patients likely at both dose levels two and three, as consistent with regulatory expectations, to really help us to determine what that recommended Phase 2 dose is in that orange box there in the middle of the slide.

After we determine that recommended Phase 2 dose, we intend to take our clinical programs into three or more indication-specific cohorts where we'll be able to describe how well our therapy works in specific indications and begin to map out a registrational opportunity with U.S. regulators and then begin to pursue a more global strategy. The indications listed there are gastric cancer, colorectal cancer, and neuroendocrine tumors of the mid gut or the hind gut. Those are the indications that we're currently pursuing in Phase 1. We look forward to potentially starting that sometime early next year. To go into a little bit more detail about what we've seen to date, and this is based on a database snapshot of October 8, 2025. Dose level 1, as I mentioned, has been completed with four subjects treated and one subject who remains on study.

We'll talk about that patient in a little bit more detail. As I mentioned, Dose level 2, 150 million cells, is ongoing with four subjects treated. We recently described in a press release that our safety monitoring committee deemed that safety level safe for ongoing exploration and dose escalation. The opportunity to explore 450 million cells is currently under consideration. We have two subjects that we may or may not treat at that dose level. All of the activity that I'm representing there is based on the successful manufacturing runs, which in the business that we're involved in, genetically modifying people's human cells, this is a real measure of how well we do our job. It's a real testament to the work that is coming out of our CMC group. Going a little bit deeper, these are the subjects treated at Dose level one.

We have three patients here that we can describe as being valuable. One with a neuroendocrine tumor who demonstrated really nice expansion and persistence of her cells. I'll show you that in just a slide or two. She, unfortunately, her disease progressed after about five months, which is something that we do in this advanced setting, unfortunately, see sometimes. What's really exciting, though, is patient number three there listed in bold achieved stable disease. She also showed nice expansion and continued persistence of her CAR-T cells, which really kind of gives us a lot of hope that her stable disease that we've now seen at 10 months out may continue and may actually deepen into something that we hope can be described as a partial response.

Finally, as is oftentimes the case in Phase 1 trials, we did have a patient that progressed at day 28, but fortunately was treated safely and was able to establish the safety of the Dose level for additional exploration. Dose level two, as I mentioned, is continuing to be something that's showing activity for us. The first two patients treated demonstrated what is, by our registrational standards, deemed stable disease, but in both cases demonstrated anti-tumor activity and shrinkage of their tumors, in one case by more than 10%. In the case of patient number six, across five of six of that patient's measurable lesions, the tumor shrinkage or the diminution of the tumor burden ranged from 6%- 37%, meaning that those tumors all got meaningfully smaller and hopefully contributed to that patient feeling better and living longer.

We're eagerly anticipating the results of patients seven and eight who have been treated safely and have led to our anticipation and excitement for what we're starting to see at Dose level two. Finally, as I mentioned, we have two patients with neuroendocrine tumors who will be treated in the coming weeks. Just to share a little bit of the translational or scientific data that we hope to generate in a study like this, what this graph shows is what our cells are doing in each patient. Each of those individual colors that describe a line going from zero up to approaching a million is describing the number of copies of our CAR or of the gene that we have transduced into these cells that we can measure in patients' blood. For all of these patients, they start at zero because they don't have any of these engineered cells.

What you can see from left to right on the x-axis is from day zero out to now day 200+ in that patient that I described previously to you, we're seeing that the cells can be measured and expanding up to a peak in most of these patients at somewhere between 10 days and 25 days out, where they then compress and contract to a population that we expect to see in the context of the approved CAR-T cell treatments that are available for patients with hematologic diseases, that a population of cells is going to persist into perpetuity. What those cells do in patients is they provide continued anti-tumor surveillance and anti-tumor activity that can result in a deepening of a response that we see over time. It actually provides maybe the most important function, which is preventing the tumor from coming back and from growing over time.

One of the really unique and important things that needs to be remembered in the context of what we're trying to do with CHM CDH17 is provide that one-time therapy that expands exactly the way these cells do in this figure, but then also go on to persist as we're starting to see in each of the patients now that we're able to measure. What's probably important to note here is those lines that end sooner than 150 days out or less are not patients that we can't measure yet, but those patients that we don't yet have assessments for. We're hopeful that those lines will continue out for much longer for all of those patients. We just don't have that information yet. It's that one patient with the line that goes out past 200 days that has us really excited about what this therapy could deliver for patients.

Rebecca McQualter
CEO, Chimeric Therapeutics

This is hot off the press, I might add. I just want to make it clear that the patient IDs here do not match the patient IDs presented earlier. We only got this graph this morning. Patient number one is not patient number one, and they're clinical trial site IDs, just to make that really clear.

Jason Litten
CMO, Chimeric Therapeutics

Thank you. That is really important.

Rebecca McQualter
CEO, Chimeric Therapeutics

Anything else to add before I move on?

Jason Litten
CMO, Chimeric Therapeutics

No, that's all. Thanks.

Rebecca McQualter
CEO, Chimeric Therapeutics

Right. We're really excited about what we're seeing here. We're really delighted to see that the cells still remain in the body for multiple months after their only infusion. We're really excited to see what comes from the results that we get after these are processed. Very exciting. I think on the last webinar, I mentioned the market size that we're chasing here with this particular asset. Last year it was $27 billion. Unfortunately, I think I've mentioned before, bowel cancer is the fastest growing cancer currently in the segment of 20-year-olds to 40-year-olds. This is likely attributed to the high processed food diet that we now have no choice but to be exposed to. This is only going to continue to grow. I think it's really important to state here that we've got quite a few companies that are looking at us right now.

There's a lot of commercial interest in this particular asset. Because it's a CAR-T, there's a few gaps in some CAR-T manufacturing facilities around the world, particularly at Novartis that I used to work for. They've got a manufacturing facility sitting there that's only used for 30% of their capacity. There's a big gap there that we think we can fill. We're really excited to continue our progress and to show these types of results and this type of persistence and these responses so that we have all these big boys looking at us, which is really exciting. In summary, for CHM CDH17, we've treated eight patients. There are two more waiting to be treated. We've manufactured for 10 patients, which, as Dr. Jason Litten mentioned, is a really important measure of our success on how well we can manufacture. We've seen tumors shrink.

I think a question that came up during the week was, why did we describe it as a mixed response? I don't know if you want to answer that now, Jason, but if you can answer that now, that would be great.

Jason Litten
CMO, Chimeric Therapeutics

I'm happy to. It's a great question because it kind of gets to some of the nuance of the work that we're doing and how we need to have lots of different lenses for how we describe success. Per RECIST, R-E-C-I-S-T, which has a regulatory standard for how a response is defined, it requires that all of the measurable lesions are diminished in size by greater than 30%. In addition to that, it requires that any of the lesions that are not measurable but present do not increase in size and that no new lesions emerge. Those are the three qualifications. In the case where we've described and continue to describe ongoing stable disease per RECIST, that is per that very strict regulatory definition that includes all three of those criteria.

What we've seen, however, and what's really important in the pursuit of progress and the pursuit of, frankly, cure in this context is that we've fulfilled two-thirds of these requirements in a really impactful way. The first is we've not seen the emergence of any new lesions in any of these patients. That's one kind of clear objective measure of success. The second is the non-measurable lesions have not grown in size in any significant way. Our focus becomes narrowly, what about those lesions that we can measure with a caliper in an imaging scan on a patient with cancer? In every case where we've been able to measure tumors in patients, they've either not grown sufficiently to be described as progressive disease, or PD, as we say it, or they have shrunk in size that is not yet 30% less than what would be required.

In some cases, we have seen tumors that are greater than 30% lesser in size than when treated. Unfortunately, the other tumors that we're measuring don't fulfill that criteria as well. We're being conservative and we're being rigorous and scientific in the way that we talk about this when we say it's a mixed response, because what we're saying is some of the tumors are getting smaller. That's really, really important. That's a signpost for us along the way to say that we're pursuing something that is disease modifying. Now we can say that objectively in human patients. What we now need to do is continue to do that and demonstrate that more consistently, either by treating additional patients at this dose level or potentially pursuing a higher dose level. This is progress, though. We're excited by what we're seeing.

Rebecca McQualter
CEO, Chimeric Therapeutics

Really great. Thank you for that. Hopefully, that makes sense to our shareholders on the call. If I move into FY26 deliverables, we're really keen to complete Dose level two, which we're almost there if it's appropriate. As Dr. Jason Litten mentioned, move up to Dose level three if we feel like it's appropriate. There's no need for us to do that. We can hang out at Dose level two if we like what we see. Because we've seen that shrinkage by 37%, we really like what we see. As the Safety Monitoring Committee has deemed this dose safe, we're really excited to see how the next two patients read out. We'll make a decision about the patients nine and ten, how we're going to treat them. We will conclude Phase 1 in FY26. We're really, really close. We'll get ready for Phase 2, which is really exciting.

We don't need to go back to the FDA. I think that's really important to note because we have Phase 2 approval already. Dr. Jason Litten did such a great job of designing our clinical trial that we don't need to go back to them, which is fantastic. Once we pick a dose that we feel is safe and efficacious, we can move straight into Phase 2, which is very exciting. With that, I will finish CHM CDH17 and move on to our next asset. It's very nice to have two performing assets, CHM CORN-K. There's a couple of points I want to make about CHM CORN-K really quickly before I hand over to Jason to take us through the results. CHM CORN-K is an off-the-shelf allogeneic product. We manufacture this product from universal donors. We can manufacture about 200 doses from one patient, one healthy donor.

I just wanted to say thank you again to our manufacturing team because these particular NK cells that my manufacturing team makes are sensational. They're really going in and beating up that leukemia really quickly. We're really impressed with what we're seeing. Having said all of that, these two trials are not our trials. These are investigator-initiated trials. MD Anderson came to us and said, can we please use your amazing NK cells? We said yes. What that means is we don't have control over how the studies run because they're paying $5 million and we pay a small fee for manufacturing. We really rely on our good relationship to make sure that we have open lines of communication. We connect with the team every two weeks and make sure that we have a continuous flow of information to maintain our continuous disclosure obligations.

It is really up in the hands of MD Anderson. This is their study. I really wanted to make that clear before I hand over to Jason that we do our best to maintain our continuous disclosure obligations. We've got a great relationship with MD Anderson to make that possible. With that, I'll hand over to Jason to take us through the results.

Jason Litten
CMO, Chimeric Therapeutics

Thanks, Bec. That's a really nice introduction. I think, you know, when they say in the horse racing business, you know, different horses for different courses, the prior asset, which is an autologous asset, it's highly bespoke. It's hoping in the hopes that it requires something highly bespoke to be effective in solid tumors, where we've already seen that CAR-T cells can be really effective. In AML, on the other hand, CAR-T cells have not yet, autologous CAR-T cells have not yet demonstrated success. This is a different horse for a very different course. AML is a very challenging disease for us. What we've done with this program is support the advancement of the CHM CORN-K program, most specifically to be used in combination with other drugs that look to be potentially transformational in this horrible disease.

The first of these that we talked about, I think on the prior slide, was it, Beth, was the combination with Vactocertib that is being evaluated at Case Western Reserve University. That one continues to accrue patients in CML, but also in AML, where the focus has really shifted there. Dr. Tomlinson, our partner there, has taken the lead and has done a great job to really drive the science and the clinical enrollment of AML patients. That study is ongoing. In addition, Dr. Abhishek Maiti at MD Anderson has been advancing a really exciting new study where he's shown preclinically and through really exciting support through the literature that the established frontline therapy for patients who are not appropriate to go to transplant, combining with NK cells, could really deepen and extend the activity that we see from that combination.

That's what we see here. Azacitidine and venetoclax are really the standard therapy for patients with frontline AML who aren't suitable for transplant. This combination does have activity, but unfortunately, it's completely inadequate for what these patients need. Dr. Maiti is advancing the combination now with the CORN-K cells that I'll describe in just a minute to really try to not only increase the number of patients who might benefit from this combination but also extend the durability of the activity that we see there. Once these patients progress from this frontline therapy, their options become very, very limited. Those options, when they do work, don't work for very long. This is maybe, in many cases, the only shot patients are going to get. On the next slide, I can describe what Dr. Maiti has now shown in his ongoing clinical trial.

We've told you at previous webinars that the dose escalation Phase of the study had been completed and had demonstrated safety across six patients. It's important to note that all of those patients had previously received the combination that is being offered here in a prior line of therapy. It wasn't very surprising to see that the activity here was not very impressive. In this heavily pretreated and, frankly, very fragile patient population, this combination could be delivered safely. Dr. Maiti was able to show that by the end of next year and open the frontline Phase of this study that is continuing to enroll patients at MD Anderson. It has recently opened at Case Western Reserve University with Dr. Tomlinson as the PI there.

We released last week the fact that now more than half of the patients treated, four of the seven patients treated, have experienced responses to this therapy. This is actually more than what we might have expected with the azacitidine-venetoclax combination on its own. With the way that Dr. Maiti is delivering these cells on the schedule and at the dose that he's delivering it, the expectation is that we won't contribute negatively to the safety profile that these drugs deliver, but that we can really deepen and increase the durability of the activity that these patients get to experience. We will be looking at responses over time and reporting them to you as we see them. The importance to me, and I think the importance to patients, is that we really look at the durability and the depth of those responses over time.

My hope is that we can not only maintain the numbers that we're seeing here, but potentially show that the responses that we see become deeper. That's the hope. Any questions about that, Bec, before we move on?

Rebecca McQualter
CEO, Chimeric Therapeutics

No.

Jason Litten
CMO, Chimeric Therapeutics

OKkay, thank you.

Rebecca McQualter
CEO, Chimeric Therapeutics

I'll do the summary. As I mentioned, for CHM CORN-K, this is an investigator-initiated trial. That means that we do our best to get the results. We received the results on Saturday. We got the press release back to them to approve, and we were able to get that approval at 5:00 A.M. on Thursday morning. We got it out before market open. We're doing our best. We've completed dose escalation. The first-line dosing is underway. As far as we're aware, we're the only company that's supporting a first-line cell therapy trial in the world at the moment. Please correct me if I'm wrong if anybody finds out that someone else is doing first-line. This is before anyone's had any single treatments. They're getting our NK cells first, which is really exciting. I know that the team at MD Anderson are really excited about this front-line dosing.

We've treated seven patients, and we can go up to 20 patients per the study design, which is really great. There's also no safety issues, which has been really important to see. For us, for FY26, our deliverable, really the catalyst for FY26 for us is just to complete that dosing of the 20 subjects at MD Anderson. However, because this is run by MD Anderson, that's really up to them. We trust in their partnership, being the biggest cancer center in the world. We're really happy with our relationship with them, which is great. Very delighted to see this progress, and we hope we see more complete responses in the weeks to come. Please get your questions ready. I'm just going to summarize everything we've spoken about today, and Jason and I are very happy to take your questions.

Just to reiterate on CHM CDH17, we've treated eight patients, and we're shrinking tumors, which is so exciting. We've had very 10 / 10 successful manufacturing runs with very, very tight confidence intervals. You can tell I'm very proud about that. I think the most important thing for us in a Phase 1 right now is that we have seen no safety issues to date. Really importantly, particularly for the target of CDH17, we haven't seen any off-target effects. We're really happy with what we're seeing, and we hope that we can see tumors shrinking even more in the coming patients that we treat. For CORN-K, we've finished dose escalation. First-line dosing is underway. We're at 7 / 20 right now. We've got four complete responses, one partial response, and no safety issues to date.

I'm really happy about our progress to date, and I really want to say thank you to my team for running the show over there in the U.S., and they're doing such a great job. With that, we will open for questions. Thank you, Matt.

Operator

Thank you. As you mentioned, we'll move on to some questions and get through as many as we can over the next 10 minutes or 15 minutes. I'll just jump straight into it. Firstly, I know you touched on this, Bec, but are you engaged in discussions with potential partners following these recent clinical updates?

Rebecca McQualter
CEO, Chimeric Therapeutics

Yeah, so because of the big market that we're chasing with seven different types of tumors that we can target with this particular asset, and because of the uniqueness of it being a CAR-T targeting CDH17, there's a lot of antibodies targeting CDH17, but we're the only CAR-That we're aware of. Because of those two unique features about our particular asset, we have had a lot of different discussions with potential partners. We're really looking forward to progressing those discussions as we get more results in. We're working on it.

Operator

OK, we'll stick with CORN-K to start with. With regards to CORN-K, do you know when we might see a full data, a full readout?

Rebecca McQualter
CEO, Chimeric Therapeutics

That's really up to MD Anderson. They're working on their timeline, but it will probably be mid next year, I assume. Jason, do you have any more information on that?

Jason Litten
CMO, Chimeric Therapeutics

We'll continue to provide updates along the way. As I mentioned, I think really the opportunity for this program to deliver is in the long term, taking those subjects who have responded and deepen and extend the duration of those responses. The results will really start to get interesting in the middle of next year. As we get toward the end of next year, I think our ability to demonstrate something really exciting could present itself.

Operator

Thank you. The next question is, how does the CORN-K data in frontline compare with historical data?

Jason Litten
CMO, Chimeric Therapeutics

That's what I was kind of suggesting too, as I mentioned the fact that azacitidine and venetoclax are an established part of frontline therapy for an important population of AML patients. The initial responses that we would hope to see have reported anywhere from about 35% to upwards of, in some cases, in highly selective patients, greater than 50%. The fact that we're progressing now at a response rate that's north of 50% suggests to us that we're on the right track. As I've said now a couple of times, our real ability to demonstrate success is prolonging the survival of these patients, where survival is oftentimes measured in weeks or months.

Operator

Thank you. Another NK cells related question is, could there be an FDA hurdle regarding inter-donor variation with the final product, given that only 200 doses per donor can be manufactured?

Jason Litten
CMO, Chimeric Therapeutics

Yeah, so it's one of the things that I can probably uniquely speak to because my former company was an NK cell company that used cord blood-derived NK cells as the donor source. This is something that we thought about quite a bit. It provides challenges, but those that the regulatory agencies are interested in navigating with us because it's a challenge that all the allogeneic programs are confronting. The bottom line is if these are transformative medicine, the regulators will work with us to get them approved.

Operator

Thank you. Next question, I have now going to CDH17. When will updates on patients seven and eight, when are they expected?

Rebecca McQualter
CEO, Chimeric Therapeutics

Same.

Operator

Very good. Now, moving on, what factors will lead to the decision not to proceed with those three?

Jason Litten
CMO, Chimeric Therapeutics

It's actually something that I'm quite happy to have the luxury of considering. We have seen evidence of expansion and persistence of our cells in every patient that's been treated, even the patients that have been treated at the lowest dose level. With that expansion, we see in the context of autologous CAR-T cells a safety finding known as CRS, or cytokine release syndrome. With the higher doses that we give, the higher likelihood it is that patients may experience higher grades of cytokine release syndrome. We'd like to deliver the lowest effective dose that we can that provides the right amount of anti-tumor activity for our patients. We have described a dose of 150 million cells, which is shrinking tumors and is continuing to shrink tumors that may actually deepen, as I've suggested, over time. We're eagerly anticipating and watching every scan that we take.

We now have the luxury to go up to that higher dose that may provide that additional punch if we decide that it's required. Because we're enrolling patients at the top cancer and cell therapy institutions in the country, we'll be able to manage the potential risk to patients of that higher dose. If we don't have to take it, we'd prefer not to.

Operator

Thank you. Again, related question, what will lead to the designation that Phase 2 for CDH17 will be registrational trials? What are the implications?

Jason Litten
CMO, Chimeric Therapeutics

That's the crystal ball that I don't, at this moment, see clearly into. I can tell you it's the right combination of safety and activity in each of the indications that we're talking about. For each of those indications, it's a little bit different because the biology of those diseases is a little bit different. If we're shrinking tumors and those tumors are staying shrunken, if I can say that, then we will have a regulatory path for any of the indications in which we're showing that.

Rebecca McQualter
CEO, Chimeric Therapeutics

I think it's worth noting as well that all of the cell therapies in the market right now have been registered in the middle or at the end of Phase 2. Because it's a bespoke treatment, as Jason said, it's easy for us to get like a regenerative medicine designation because we're taking the human tissue and engineering it. We're making the drug product for you. All of our predecessors have been registered in Phase 2. There's no need for us to do a big Phase 3. We can be registered at the end of Phase 2. If I think about the patient population, these are really sick patients that really need, you know, this is their last opportunity for a treatment. To be registered in Phase 2 is where we want to be.

Jason Litten
CMO, Chimeric Therapeutics

Yeah, and Bec, maybe actually, if you can put the study schema slide up for a minute, I can provide a little bit more clarity on what our regulatory strategy will be because it folds right out of our first in human study. You'll see, as I mentioned when we were talking earlier, that we have our Phase 2 indication-specific cohorts. Those cohorts will be enrolled in two Phases. They're described as Simon two-stage design, where the first 10 patients will really serve to describe to us whether or not there is a signal there that we want to pursue. Importantly, though, we've designated an additional 19 patients to be kind of our efficacy confirmation cohort. That really provides an opportunity for us to do two things.

It provides an opportunity for us to go to the regulators and discuss, A, what the size of that Phase 2 cohort needs to be to truly be registrational, and B, and a requirement of FDA kind of in the recent past and certainly going forward is what does our confirmational study need to look like in order to support a full approval. It is possible here that just stemming from this particular study design, the second part of those Phase 2 cohorts, depending on the indication and the level of efficacy that we're able to describe, those cohorts in and of themselves could be registrational. This study design provides that opportunity for us to adaptively redesign that Phase 2 cohort in order to be a size that's sufficient to describe the requirements that the FDA wants to see. I hope that's clear. Thanks for the opportunity.

Operator

Thank you. The next question, I think this was touched on slightly again, but what could be the market size for this version of a frontline therapy in this patient group? Has the company applied for orphan drug designation?

Rebecca McQualter
CEO, Chimeric Therapeutics

Which drug is that? Is that for CHM CORN-K or CHM CDH17? I can answer both. For CHM CDH17, we've applied for orphan. That's under review right now. I'm sure that you're aware, with the new administration in the U.S., things are pretty messy. That's usually a 60-day review, or now it's a 90-day review, or something's happening like that. We've got a really great regulatory guy on it at the moment. That's for CDH17. For CORN-K, we haven't applied for anything just yet. We just want to see how it performs. We're really covered with the manufacturing patent because that's where the gold lies in that particular asset. I've seen a lot of questions about the Case Western Reserve University trial with the Vactocertib study.

Because of the new Trump administration, he's taken away a lot of resources from the universities, and that includes Case Western Reserve University. Because that's, again, another investigator-initiated trial that they're paying for, there hasn't been too much progress on that trial because they're trying to figure out how to run their university without any money anymore. We've kind of just left it until we've got the money to contribute and speak up. We kind of have just left them. Now there's a new investigator who will get his feet under the desk. I'm sure that he'll be underway shortly. Jason's got a really great relationship with him. I'm confident that we'll see some updates shortly. I think because it's so messy over there right now, we've kind of just let it be. We've got two really good performing studies right now.

If I had no option, I'd be going over there to see what we can do to help. I think right now we're just kind of waiting for them to sort out their stuff and leaving them to it because they're paying for the majority of the study.

Jason Litten
CMO, Chimeric Therapeutics

Yeah, and I think it's also probably important to recognize that while the MD Anderson azacitidine-venetoclax study is incorporating standard treatment for frontline patients, that's actually a relatively common patient population to MD Anderson. That's easy to find and easy to kind of identify as being appropriate here. The Vactocertib study is involving our NK cells, which are considered an investigational agent, and Vactocertib, which is also investigational. The value proposition for each individual patient being treated for advanced AML may or may not be a little bit different. That may lead it to be a little bit harder to find patients appropriate for that study as compared to the MD Anderson study. The accrual there has been a little slower than what we would have hoped. I think all the reasons that Beth just described have contributed to it. As she mentioned, Dr. Tomlinson is a great partner. He's going to be working with us to advance that program.

Operator

Okay, we've got time for just a couple more. The clients can, it was mentioned during the presentation, can target seven different targets. Can they be licensed individually, or given the administration route and cells being the same, would it be one license?

Rebecca McQualter
CEO, Chimeric Therapeutics

When we think about licensing, we'd like to license it as a whole to make the most money for our company. There's an opportunity for different indications under that license, so it's quite broad, I think, in that respect, which is really good. We'll wait until we see what we see at the end of Phase 1. Is it a different dose for different indications or is it not? Is it one dose fits all? We're still learning about that right now. It'll be one license for the product, yeah. I think I've mentioned before, it's really easy delivery. As soon as we get your cells, we manufacture them. They're frozen. We can ship them anywhere in the world. Basically, you hang a bag and you get your tiny little dose. It's like a half a shot, basically. It's really easy delivery. It's intravenous and the patient's monitored. That's about it. The application for this to be global is really easy. I can see it from a commercial perspective in that respect.

Operator

The last question is a more general one. What is the significance of frontline treatment?

Jason Litten
CMO, Chimeric Therapeutics

I think there are probably a few different ways to answer that. I think from a commercial standpoint, Beth is probably better suited. From a clinical and scientific standpoint, your opportunity there is actually potentially curative. That's what, as an oncologist, I'm always aiming for my patients being my former patients because they've moved on with their lives and they're not cancer patients anymore. That's really what the frontline opportunity provides. Azacitidine-venetoclax actually does provide cures for a minority of patients. Unfortunately, it's only a small minority. We think that there's good scientific rationale from the work that Dr. Maiti and others have done to believe, as I said, that we can get more responses that are deeper and more durable by combining NK cells with those two drugs and hopefully cure more patients.

The opportunity, just to kind of underline it, when cancer comes back, it is far more limited. That recalcitrant or relapsed or refractory cancer, any of the R words that we describe, it means just by virtue of it kind of withstanding and resisting standard therapies, you're now being treated with something that has been deemed secondary to what we would want most patients to have an opportunity to receive. It's also the reason why many of the CAR-T cells, while they were approved in the latest lines of therapy, are increasingly being approved in the earliest lines of therapy, where they can potentially deliver cures for the greatest number of patients.

Rebecca McQualter
CEO, Chimeric Therapeutics

From a commercial perspective, you know, typically, patients have to go through nasty chemos. They save the most expensive therapies for last. As Jason just mentioned, we're trying to move that up and demonstrate the benefit of having the most expensive therapy first. The patient's cells perform much better during manufacturing. The patients are actually much better. They don't get really sick and tired. The market potential of a first-line therapy is a lot bigger than the fourth, fifth, sixth line because the patients don't make it that far. That's a little bit callous, but that's the way that we view the market. I think having the frontline, and particularly for cell therapy, having a frontline therapy is really exciting because, yeah, like I said, we're not aware of anybody else trying that right now.

Jason Litten
CMO, Chimeric Therapeutics

One final perspective that honestly, Bec neither of us really represent, but is increasingly important, especially in the U.S., is demonstrating value to payers. At the end of the day, it's the payers that really matter. The ability to demonstrate the value of a frontline therapy, taking patients who, in later lines, may or may not have their lives extended by days or weeks, if we're able to demonstrate that we can cure more patients, that's a really easy way to demonstrate value there that actually translates to important economic value for patients and for society.

Rebecca McQualter
CEO, Chimeric Therapeutics

That's a really good point because in my last job, that's all I did was argue with our single payer here in Australia, the Australian government and the Health Minister. I know what that's like. We'll be able to beat him with this data.

Operator

Great. Thanks, everyone, for the questions. We had more than we could really handle. We'll come back to any others in due course. I'll just hand it back to Rebecca for a bit of a summary.

Rebecca McQualter
CEO, Chimeric Therapeutics

Thank you so much. Thank you so much to Dr. Jason Litten for joining me very late in California. We're really delighted with the progress that we're seeing. We're shrinking tumors. We're safe, which is really important. No off-target effects. We've got a really clear line on how we're going to finish this Phase 1 study for CDH17. For CORN-K, we're cruising. We're really delighted with the complete responses that we're seeing. I'm really happy with the position of the clinical operations of the business right now. I'm really delighted to share all of these updates with you. I look forward to sharing the next few updates with you soon.

Jason Litten
CMO, Chimeric Therapeutics

Thank you all.

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