Good afternoon, everyone, and welcome back to HC Wainwright's 27th Annual Global Investment Conference on September 8 to 10, 2025. My name is Patrick Truchio. I'm a Senior Healthcare Analyst at HC Wainwright. It's my pleasure to introduce our next company, Marker Therapeutics. Marker is focused on the development and commercialization of next-generation T-cell-based immunotherapies for patients with a wide range of blood and solid tumor cancers. The novel multi-TAA technology uses non-genetically engineered T cells designed to recognize and kill multiple tumor targets for precise and safer therapies. From the company, it's my pleasure to introduce CEO Juan Vera. Welcome to the conference.
Thank you. Thank you so much for the invitation.
Maybe you can start by introducing Marker Therapeutics and the mar T-cell technology.
Yeah, absolutely. I think that this technology is quite unique, right? We, Marker Therapeutics, are the only company that is developing this strategy. The technology was developed at Baylor College of Medicine and relies really on millions of years of natural evolution to design T cells that can target multiple different targets present within the tumor, right? Our technology is capable of doing that by mediating a recognition via the native T cell receptor. In that manner, you can engage both CD4 and CD8 T cells and recognize a very broad array of epitopes present within the tumor. When you compare this with other technologies that have shown good clinical activity, such as chimeric T cell receptors or bispecific antibodies, you're basically talking about a different modality from target recognition with a very broad spectrum of target profile.
That's helpful. Maybe you could talk a little bit more about what differentiates mar T cells from CAR T cells and bispecific antibodies.
Yes, absolutely. One of the major differences is the mechanism of antigen recognition, right? When you think about bispecifics and also chimeric T cell receptors, they're restricted by the mechanism of recognition of a monoclonal antibody. As a consequence, you are restricted to a quite narrow type of targets. In the context of DLBCL, you're limited to C19, 20, or 22. All these types of modalities are competing literally for the same targets, right? Now, with our technology, the recognition is via the MHC molecule. As a consequence, you can recognize intracellular proteins. That opens this candidate for us to basically now target multiple different tumor-associated antigens. With our technology, these antigens are not only expressed in cancer cells, but they also have driver components, right? They are also involved in the cancer biology, right?
From that perspective, our technology is able to recognize antigens in a mechanism that it is very different. In addition to that, I think that the unique mechanism of action, that it is natural, also has translated into an excellent safety profile. I think we actually have seen that when administered to patients, they are very well tolerated. We haven't seen the same evidence of cytokine release syndrome nor neurotoxicities. I think that, again, a significant difference in the mechanism of action and how they recognize that now we're also seeing translated into the clinic.
Right. What do you see as the biggest unmet need today in cell therapy, and how do mar T cells aim to address them?
Yeah, that's an excellent question and kind of builds very well from your previous one. When you look into the space, I think that we can all agree that there are multiple different players in that particular arena, it's very competitive. Multiple technologies are narrowed by the target recognition profile of monoclonal antibodies and the targets available to that end. As a consequence, you basically have multiple technologies competing for C19, 20/22. When you look into diffuse large B-cell lymphoma (DLBCL), particularly after CAR T relapse, bispecific relapse, you basically have patients that have failed C19 targeted therapies, that have failed C20 targeted therapies, and these patients have nothing else available. You're talking about a patient population with a high unmet medical need, right? That's basically where we come in, right? We're basically advancing our drug MT-601 in this group of patients.
I believe that our drug will be able to fill a very specific niche.
Right. How do you think about the scalability and accessibility of mar T cells relative to CAR T?
Yes, excellent question. I think that we can also agree that, when you're talking about these types of technologies, manufacturing plays a very important role, right? I think that having a simple manufacturer that we can characterize is going to become a critical component of our success. We have spent a lot of time improving, optimizing our manufacturing process that was developed at Baylor College of Medicine. I believe we have done so in a very successful manner, right? Now you're talking about a simple manufacturing process that is only nine days. Our vein-to-vein is about 20 to 25 days, right? We are able to manufacture a product without any type of genetic engineering, right? It's a very simple manufacturing process that we have now shown is reproducible across multiple patients, including patients that are heavily pretreated, that have received CAR T cells, that have received bispecifics.
You have to take into account that the quality of the starting material is not the same as a patient that is naive. Nevertheless, we are able to produce products and deliver to the clinic.
Can you give us an overview of your pipeline and which programs you're most excited about?
Yes, that's a very good question. I'm excited about all the different programs, right? I think that our lead asset and we are concentrating our efforts at the moment is MT-601 in DLBCL. The reason for that is because we consider that with the clinical data that we have seen, and as we discussed earlier, the high unmet medical need, this will be an area that will be able to get accelerated approvals and bring this into the market in a very short period of time, right? In addition to that, we're also advancing the same asset, MT-601, into solid tumors, which is quite exciting given the fact that it highlights one of the unique elements of our technology, right? It's the fact that you can use the same asset and put that to work outside of hematologic malignancies.
Again, we are able to do so due to the broad target profile. It de-risked the development significantly given the fact that you share all the CMC section and share all the experience in the drug development. Finally, and not least, we also have an off-the-shelf program. I'm very excited, and I think that that could become the future of the company where you have a technology that we are planning to test now in patients for the first time in the second part of this year to identify if we can deliver a simple, nevertheless effective off-the-shelf technology that can be provided to patients by a very simple strategy and just providing a partially matching, creating an inventory of cells that can be given to patients in a very short period of time.
Right. Maybe moving on to MT-601, let's talk a bit about MT-601. How did you select the targeted antigens, and why do you think targeting six tumor antigens could change the treatment landscape in lymphoma?
Yes, that's an excellent question. I think that when you have a technology like this, the selection of the target antigens plays a very important role. For our approach, we actually have followed a statistical analysis looking into the antigen spectrum across multiple different histologies, right? Across multiple different types of oncological conditions, not just hematologic malignancies, we identified an array of antigen 6, which are actually expressed in a high prevalence in DLBCL. As a consequence, with that statistical approach, you are basically throwing a very broad net. In that manner, you are creating a coverage for patients. One of the things that is very important to highlight, we normally don't talk about this, is that the enrollment is antigen-agnostic, right? If you're developing a C19, C20, you cannot say that.
In many cases, you actually have to take the tumor and be sure that C19 or 20 is actually expressed. We are actually antigen-agnostic, and we can actually enroll patients independently of a prior biopsy sample to confirm expression of the target. The reason we do that is simply because of the broad target profile that our product is able to create.
What is the design and objective of the Phase 1 Apollo trial?
Yeah, for our Apollo Phase 1 trial, it's a quite interesting study that has two components: dose escalation followed by a dose expansion. Really excited as we recently have completed our dose escalation. Our dose escalation, we explored doses ranging from 100 to 400 million cells. We're really excited to report that all doses have shown to be well tolerated and safe. In the first part of the study, this was, in fact, open to patients with different types of histopathologies. The primary objective of this was to address the safety and what was the maximum tolerated dose. We have seen not only that the product is safe, but also we have seen a clear signal of efficacy. As a consequence, we're now moving since June 17 into the second part of the study, which is the dose expansion.
That is now concentrated in enrollment in patients that are diffuse large B-cell lymphoma (DLBCL), CAR T, and/or bispecific relapse that are going to be treated with the highest tolerated dose level, which is 400 million cells.
Got it. How do mar T cells perform in heavily pretreated patients, including those who failed CAR T and bispecific antibodies?
Yeah, I mean, I think that that's the question, right? I mean, we're basically advancing this into a patient population that we consider with high unmet medical need. We're really excited with the results that we're seeing so far. We reported, if we look into the data so far, of 12 patients that are non-Hodgkin lymphoma, we're reporting a complete response rate of 50% and an overall response rate of 66%. If you look particularly into those patients, the DLBCL and those patients that are CAR relapse and bispecific relapse, we actually have seen one of their best responses with this group of patients. Really excited to see that even though we're treating patients that have been heavily pretreated, have failed CAR T cells and bispecifics, we're able to see exciting objective responses with an excellent safety profile.
Right. Great. Safety is a key differentiator of the product candidates. How do you expect the safety profile to change exploring now higher doses in the dose expansion portion of the program?
Yeah, I mean, I think that that is one of the elements that makes this treatment different, right? It's the safety profile, right? I think that despite now going into the highest dose level ever tested in humans, which has been 400 million cells, we actually have seen an excellent tolerability, right? We basically have seen no evidence of ICANS. The only thing that has been reported as CRS has been two level 1 CRS that have been fever that responded in absence of any treatment. I think that speaks in terms of how unique this technology actually is.
What is the benefit of using lymph node depletion in your study? What have you learned from your recent immunomodulatory data in terms of mar T cell persistence and expansion in vivo?
Yeah, absolutely. I think that that was a big decision for us to incorporate lymph node depletion, given the fact that a lot of the clinical experience from Baylor College of Medicine was in absence of. The reason to do that is really to try to provide the best available capacity for T cells to expand and persist once administered to the clinic. I think that with the data that we have seen today, that is actually correct, right? It's not like we are inventing the wheel here, right? I mean, I think that in the context of chimeric T cell receptors, it has been very well demonstrated that lymph node depletion is needed in order to promote the expansion and persistence of the T cells. We really were able to extract some of that knowledge from the field.
When we tested it in our own study, we were able to confirm the same benefits, right? The patients that now we have treated with lymph node depletion, we're seeing a very similar kinetics of better expansion, better persistence, and therefore we anticipate to see better clinical activities. The thing that is remarkable is that despite going to now incorporate lymph node depletion and despite going to the higher dose levels, we haven't seen anything detrimental from a safety perspective. Yes, we're seeing neutropenia at the rise of lymph node depletion, that it is transitory, but outside of that, we have not changed the excellent safety profile of the drug.
Right. How are you thinking about next steps for the program, and what's the regulatory pathway and anticipated timing for a pivotal trial?
Yes, excellent point. I think that our plan is to advance this for a pivotal study for early next year. Our anticipation is that given the fact that this is a patient population with a high unmet medical need and the results that we're seeing, we'll be successful doing so in a single arm phase 2B clinical study that we calculate would be about 70 patients, right? We anticipate to start enrollment on such a study early next year.
Turning to solid tumors, you know, with MT-601 also being investigated in pancreatic cancer, can you walk us through the rationale for this indication?
Yeah, absolutely. I think that one of the complex scenarios, especially when you have a drug like this that can play in multiple different areas, is how to select which indication to pursue, right? I think that what we are trying to do here is, one, identify whether this drug could also work in solid tumors. The reason we selected pancreatic cancer is because this is a huge area that, despite multiple efforts, the treatment paradigm has not changed for many years, right? You're talking about another group of patients with a high unmet medical need, a group population that is predicted to continue to increase. If you look into the mortality rate for pancreatic cancer, particularly for 2030, it's predicted to actually take many of the other conditions, including prostate cancer, as they're now coming into decline due to better screening strategies.
Without a doubt, pancreatic cancer is and will be a huge healthcare issue that we want to address. The reason of going into this area is also rooted out of proof of concept clinical data from Baylor College of Medicine. We have tested this technology at Baylor College of Medicine. We are seeing good results from the Baylor College of Medicine clinical study, where a publication now is going to be imminent, where we'll be characterizing these results, where we have seen complete responses, partial responses in patients with metastatic pancreatic cancer. Our objective with our study MT-601 is to recapitulate these studies, to build in that success by now adding our drug MT-601, higher dose level, lymph node depletion, and hope to further improve the clinical activity.
How does the mechanism of mar T lend itself to tackling heterogeneous antigen-adaptive solid tumors?
Wow, that's an excellent question. I think that is perhaps one of the things that makes treating cancer so difficult, right? When you look from patient to patient, every cancer is different. That alone is very challenging. In addition to that, as you treat the tumor, that tumor is built to evolve and adapt from that heterogeneous makeup, right? I think that we actually have seen that within our own models, right? When you put an immunological pressure, those tumor clones that do not express those target antigens are meant to escape. I think we now see that very clearly in the context of diffuse large B-cell lymphoma, CAR T, and CD19 negative tumors, right? I think that the unique elements of our technology derive from the multi-targeted approach that basically is able to minimize that potential and is able also to adapt in vivo once given to the patient.
We have seen evidence of that, which is very interesting.
What are the next milestones investors should look for in this program?
We're definitely concentrating our activity into our MT-601 study. We seek to continue providing clinical data and reports into the enrollment, clinical activity, and safety profile that we're seeing into our drug. Definitely continue updates on MT-601, followed by updates into our off-the-shelf study. We anticipate to treat our first patient in the second part of the year and also enroll our first patient into our solid tumor program in the second part of this year.
Moving on to manufacturing, it's a critical challenge for cell therapies. What advantage does your simpler non-genetically engineered process provide, and how does it differ from other cell therapies?
Yeah, I think simplicity is key, right? Simplicity is going to allow to have a process that can be reproduced, right? One of the things from our current clinical data is that this data is already generated from two different vendors. We have seen that despite having two different vendors, we have one successfully perform a tech transfer. We have products that can be characterized equally and products that are able to yield objective responses. Our objective now, we recently entered into a relationship with Cellipont. We're in the mix of validating this new vendor with the objective of having additional capacity as we enter into a pivotal study.
Maybe just as a final question, as you kind of outline the milestones and expectations over the next 6 to 12 months, what will you be looking for to determine success for Marker? What should investors be looking for? How should we think about the company, say, in the next 6 months, 12 months, but then also over the next 3 years?
Yeah, absolutely. I mean, from a viewpoint, the success of the company is binary, right? It's basically rooted out of MT-601, DLBCL, right? From there, I think what is very important is to characterize and understand what is the benchmark in that area. When you look at DLBCL, fourth line, there's really nothing available, right? The benchmark is relatively low, right? I think what we basically want to show is a clear success of complete responses that are well tolerated. That will anchor the success of MT-601 for approval in that area. If you look beyond that, I think that you could be looking to a technology that can be extended to solid tumors in such pancreatic cancer, as well as evolve into the future as an off-the-shelf technology, right? Those things are things that we want to bring once we are able to ensure success into our DLBCL program.
Terrific. Thank you so much. Thanks to Marker Therapeutics for attending the conference. Thanks to our audience for being with us. Have a great rest of your day and a great rest of your conference.
Thank you.