All right. Welcome, everyone, to 2025 Jefferies Global Healthcare Conference. My name is Roger Song, one of the SMICA Biotech Analysts at Jefferies. It's my great pleasure to introduce our first presenter company and the first for the entire conference for the public side, IO Biotech, Mai-Britt, CEO. And then this is the presentation. And then if we have time for questions in the end, then we will take that. Thank you.
Thank you,
As always, it's a great pleasure to be here at the Jefferies Healthcare Conference in New York. Let me just start off with our forward-looking statements before moving into what I'm really excited about presenting for you today. Cylembio. The power of our approach is the ability to combine innovation with speed, accessibility, and scalability, making it possible to break boundaries and ignite changes. This is what we are here for. Cylembio is the U.S. brand name of our lead product, the IO-102, IO-103, our lead investigational immune modulatory off-the-shelf cancer vaccine. We view Cylembio as not just a potential medical breakthrough, but really a possibility to redefine the possibilities for patients in need of treatments.
The platform we are working on, the Twin Technology Platform, is truly innovative, making our way through to the tumor microenvironment with our cancer vaccines, where we are able to really change and modulate the environment and thereby also reducing the tumor cells. We see this that it's giving us an unlimited possibility to truly make changes and fill an unmet need in many different indications. We are here today presenting these pipelines that we have, where we have already seen great hope for patients in need of these treatments. Let me just go through the patients. This is the most important part of why we are all working in this space. Patients in need of better and more effective treatments.
They come in, and when they get their diagnosis, they really are filled up with anxiety, nervousness, and also, of course, uncertainty about the journey that is in front of them. What we are focusing on with our lead product candidate is really to come in in the first line metastatic setting, where people are diagnosed with unresectable or metastatic melanoma. This is here where we are seeing that there is still a full unmet medical need. Patients that are treated today with standard of care are not benefiting well with the treatments that are out there. Also, some of the patients that are treated and benefiting well, they actually get adverse events needing to step off the treatment and therefore, of course, not benefiting at all. What we are seeing is that we can come in with a treatment that is really offering a much better effect.
At the same time, we have a clear safety profile, and therefore patients can stay longer on the treatment that we are offering. We see a great potential for our product candidate, Cylembio, and I will share more details as I'm going through the presentation here today. Let me just start here first with some highlights of the company. This is, again, I want to highlight break boundaries, ignite change. This is really our statement that we have here for the Twin Technology Platform and our opportunities we see that we have with our product candidates. We're working with the Twin Technology Platform. It's really a platform that is driving T cells to mediate changes in the tumor microenvironment and eradicate the cancer cells. We have already three pipeline programs in development.
We have our lead, Cylembio, IO-102, IO-103, and I will go through this one in much more details. Then we have the two newer targets, IO-112 and IO-170, that are coming up and moving towards IND as well. Those two newer candidates, we are seeing the same effect as we have already seen with our lead program, Cylembio, and we will have the opportunity with those two to move into more hard-to-treat cancer indications, really broadening up our pipeline. The indications we are working in right now is melanoma. This is our lead, and we have already presented data in lung and head and neck as well. I will go through these data. We are seeing really excitement also there with our lead product.
Really, what made a change for the company was the data that we saw in our proof of concept trial, the phase I, II trial that was published back in 2021 in Nature Medicine and gave us the breakthrough therapy designation granted by the FDA. 80% overall response rate, 50% CR, and an impressive 25.5 month median PFS. We saw that, as I have mentioned before, without adding any safety concerns to the patients treated. We are very excited when we brought this further into the pivotal phase III trial that is now reading out here in Q3 2025. This year, it's really a transformational year for IO Biotech. We are getting our data from the phase three trial, and it gives us the opportunity to potential file a BLA towards the end of this year.
It gives us the opportunity to bring the product to the market already in 2026. Again, I want to highlight Cylembio. This is our U.S. brand name for our IO-102, IO-103. Moving forward, let me just go into more details around the market that we are working in as our first indication. We all know that melanoma is growing. It is the fifth largest cancer in the U.S., and it is growing in numbers every year. We are also therefore seeing that the market is growing, giving us a great opportunity to move in and take a good position in this market with the data that we have already seen. It gives us the reason to believe that we can take a strong position and a huge market share of this market. You see the numbers here.
It's growing from, if we look at 2023 all the way to 2030, it's growing with 9% up to $30 billion. I talked about the challenges we have in these indications in my introduction, but let me just highlight again. We have standard of care with 50% of the patients that do not respond. For the responding patients, we have additional 50% of patients that actually do not benefit because they have adverse events. It gives us a huge opportunity to come in with a treatment where we both are able to offer effect and reduce the safety concerns as well. We see our well-positioned to come in and take a huge market share of this first line opportunity. We do that based on the data that we published, as I mentioned, back in 2021 in Nature Medicine.
I don't want to highlight the data again. I gave them already in my introductions under the highlight. I just want to mention here in this slide that we also updated the data with a publication in JITC in May 2023, where we confirmed the data that we had published in 2021. The current trial that we have been executing on during the last many years is shown here. This is the trial design. We enrolled 407 patients. We randomized towards pembrolizumab with IO-102, IO-103 in combination with pembrolizumab, one-to-one randomization. PFS is the primary readout. We are getting the data here in Q3 this year, as I mentioned. We have already had multiple IDMCs, all with the same recommendation to the company to continue without any modification.
That shows us that we still continue to have a very clear safety profile with these recommendations from the IDMCs. Of course, we are out there talking with KOLs, discussing our data and testing the excitements as well from both treating physicians, key opinion leaders, and the feedback that we are getting is very positive and very exciting for us. Of course, patients are looking for better treatments with more effect and also with lower safety profile. As we know, this is also what the treating physicians are looking for. We are looking forward to come and offer this opportunity both to the patients, but also to the treating physicians. As we are preparing for the data readout and for the BLA filing, of course, we are also preparing to launch. We're excited about where we are right now.
In those preparations, we are working on fully establishing the belief in this potentially transformative product, Cylembio. Also, we are working on the value by ensuring that we have the product available for all the eligible patients out there that would have the opportunity to get our product. The launch model that we are working on is pragmatic, and we are working on focusing on the top treaters of the melanoma community, both at the academic centers and also in the community settings. With that, let me just walk you through our pipeline and discuss a little bit more also the details around lung and head and neck and the neoadjuvant-adjuvant, the perioperative settings that we're working on as well. We have front and center here in the top of the pipeline diagram, we have the phase III trial.
This is what I have just gone through and where we are expecting data here in Q3. Next to that trial, we have also embarked on II phase two basket trials, one in first line solid tumors and one in the perioperative settings, neoadjuvant-adjuvant solid tumors. We are working on lung and head and neck in the first line solid tumor settings. Here we have already seen exciting data, which we published last year at some of the key clinical conferences. We will also come with updates from this trial later on. In addition to this, we have also executed on a phase II trial in the perioperative settings. This trial recruited beyond our expectations. We took all the patients in within a year, both in the melanoma and the head and neck cohort.
We actually saw so much interest for that trial that we made an extension of the clinical design, including also a cohort C, which were a randomized cohort in the melanoma settings. I mentioned in my introduction that we are extending also the work on the Twin Technology Platform with additional targets. We have IO-112 coming up, targeting arginase. This is hugely overexpressed from multiple different cell types in some of the harder-to-treat cancer indications like ovarian, colorectal, and so forth. We are also working on IO-170, TGF-beta, which is a little bit earlier in the development, but we are seeing very interesting and exciting data from this target as well. As we know, TGF-beta has been a target that has been attempted to be dropped many, many times from different types of platforms.
What we are seeing is still a clean safety profile in the early preclinical models with this target. If we meet our expectations, of course, we're excited also to bring this target into the clinical settings as well. Looking at the market, both in lung and head and neck, we are seeing similar growth as we saw also when I introduced the opportunity with melanoma. Lung is a very large and huge indication. It is the second most diagnosed cancer form, and it is still growing. We see a 10% growth from 2023- 2030, growing all the way up to a $60 billion market opportunity in 2030. With our data, we intend to take a good market share of this indication as well. It still remains a clear unmet need despite the opportunities that are for these patients as of today.
We have numbers of 40%-60% of the patients not responding to the first line opportunities that are offered today. Head and neck is a slightly smaller indication compared both to melanoma and to lung. It is the sixth most diagnosed cancer worldwide and also with growing in the numbers. We see a 6% growth coming from 2023- 2030 and going all the way up to a $5 billion market opportunity at 2030. It remains a clear unmet need with 60% of the patients failing to respond to the treatment options as we are seeing today. I'm sure that many of you were excited together with me when we saw the updates for the ASCO coming out with new and great results for these indications. We are excited about our own data as well in these indications.
This is truly an indication where we have not seen much impression of the data through many, many years, but now it seems like many of us start understanding the biology in this treatment as well and giving the patients a hope that we are able to move forward with some great drugs for this indication as well. Going into the clinical trial design, it is shown here. This was a phase II basket trial, and we took in patients in the lung cohort with PD-L1 TPS score above 50%. We treated those with IO-102, IO-103, Cylembio in combination with pembrolizumab. In the head and neck indication, we took patients in with a CPS score above 20%.
We have already shared data at some of the conferences, as I mentioned, and I want to share the data with you also here today, what we shared at CTC in 2024. This is from the lung cohort of the trial. We saw 48% confirmed overall response rate and 55% unconfirmed. We also saw a 12-month PFS rate of 48% and a median PFS of 8.1 months. This is very encouraging data, and we are working on maturing these data and coming out with an update on these data later on. Similar, what we saw in the head and neck setting where we met the primary endpoint of 44% of overall response rate, and we had a 6.6-month median PFS in this trial. As I already mentioned, this is really great data.
This is doubling up compared to standard of care, what we see with pembrolizumab alone in these indications. Of course, something that we will intend to work on as we move forward. Really very excited to see what we are able to do with the lead product candidate that we have in our pipeline in this indication. I tend to say in this slide that there is only so much luck that you can have. We have great data in melanoma. We have already shared that in our publications in Nature Medicine. We have worked on our phase III trial to fully enroll and execute this trial with the patients. We took in 407 patients, and we are having the PFS readout here in Q3. Now we also have data in both lung and head and neck.
We truly believe that we have a drug because seeing effect in three individual indications is not to us just a luck. It is truly reflecting that we potentially have a drug here. We are very excited to move forward with Cylembio across these multiple tumor types that we already have worked on, but also going beyond. This is not a product that is limited to these three indications. It truly can be also effectful in other indications as we move forward. Let me just finish this part of my presentation going through all the clinical highlight, also highlighting the perioperative trial that we are running in the neoadjuvant-adjuvant setting. We are excited, as many others, to move into the earlier clinical stages and also to try to test the value of our lead product in those settings. We have already enrolled fully this trial.
It took no time, one year. Then we were fully enrolled in all the three indications that we are working on. We have enrolled patients in cohort A, the melanoma resectable stage three settings. Here we enrolled 15 patients and treated patients prior to surgery with three rounds of three cycles of IO-102, IO-103 in combination with pembrolizumab, then surgery. After surgery, moving into the adjuvant phase, patients were treated with up to 15 cycles of IO-102, IO-103 in combination with pembrolizumab. Similar treatment was done in the cohort B, where we enrolled patients with head and neck resectable stage three, stage four. 15 patients were enrolled. We opened up the trial to include a cohort C, where we enrolled patients in a randomized setting, again with melanoma resectable stage three disease. We combined IO-102, IO-103 with pembrolizumab.
We had an arm also where we treated only with pembrolizumab prior to the surgery. Then patients were treated as well after surgery. This is data, or we will have data from this trial coming out probably later this year. We will be excited to share this as well when we have those data. Looking at the totality of what I have shared today, we see a great number of addressable patients in those indications that we are working on. We will anchor with our product launch in first line advanced melanoma. There is a huge number of patients that we could target, 15,000 patients, as we have mentioned here on this slide. We are very excited about this opportunity. We are also excited about the ability to actually move beyond melanoma as the first indication.
This is where we will advance into both head and neck and first line metastatic lung. The number of addressable patients we have mentioned here is all the way up to 40,000. When we expand further out to the third cycle, we have the perioperative settings where I have just gone through with the head and neck and also the melanoma indications. Here the number of patients goes beyond 100,000. Truly big numbers and really very exciting. We look forward to all of this, of course. Before I end this part of the presentation, let me just go through what we have in the preclinical pipeline as well. We are excited about what we are seeing already with the arginase IO-112. We are seeing similar data as we have seen for IO-102, IO-103.
We are targeting not only the tumor cells directly, but we are also targeting the tumor microenvironment, modifying the tumor microenvironment, giving us the ability to really give a much cleaner effect on the tumor cells themselves. Similarly, we are seeing the same data also with IO-170, the TGF-beta. Last but not least, and this is not something we are working on right now in the pipeline, is the opportunity to expand beyond the IO indications. The Twin Platform can truly be also applied to indications outside of oncology. For example, we have the opportunity to go into infectious diseases as well. Something that in the future we see as an opportunity for the future application of the Twin Platform. With that, let me just go through quickly some of the steps that we have with the mechanism of action and our technology.
It's truly novel biology that we are working on. We are giving the vaccines as a subcutaneous injection. We are giving it as peptides. This will allow T cells to be activated with dual mechanism of action. The T cells will home out to the tumor microenvironment and target both tumor cells and also immune suppressive cells that are positive for the targets that we are working on. Here we can exemplify that with IDO and PD-L1. This will also lead to a modification and an inflammation of the tumor microenvironment and thereby further enable the tumor killing effect. Of course, we have also worked on the product. It's really important for us that we have not only patient convenience.
It should be convenient for patients to come in and get treatment, but it should also be the best possible way from the product manufacturer all the way out to the hospital settings. We have secured now commercial scale of our product. We have also secured a convenient storage of 2 degrees- 8 degrees, which we do think is really a great opportunity for us to make sure that we have the product available for the patients in need of treatment at a timely fashion. We have very many milestones in front of us. Of course, the most important one I have always already gone through. This is the readout of the primary endpoint of our phase III. I want to say that we are in a position with our cash to secure that we have time after that.
On the back of that readout, we have a cash position that takes us into the second quarter of 2026. You might have seen the recent press releases of our EIB loan facility where we have drawn down some of the tranches from this facility. We also have some of the other data coming out from the other programs that I have gone through that will come out here in the second half of 2025. This is truly an exciting next half of the year that we are going into. We are very excited, of course, about the data readout that we have in our phase III trial. I want to end my presentation here today just by going through some of the key points or the key highlights that I have already mentioned.
We do see a significant unmet need in the indications that we are working on, not only melanoma, but also head and neck and lung. It is large and growing markets going from what we are seeing as of today and growing with a 10% growth rate towards 2030. Our opportunity is off the shelf. It is really giving the patients to be treated when they are in need of the treatment fast and effective. We see an opportunity to come in with the data that we have already achieved to potentially be transforming the treatment paradigm, not only in melanoma, as we have already shared, but also in the other indications. We do see a great future for Cylembio, which is the U.S. brand name that we have here for our first lead product candidate.
I would like to thank all of you for listening in to this early morning presentation from IO Biotech. Thank you.
We have a couple of minutes.
Kasim.
It's open.
Yeah, maybe thank you, Mai-Britt and Kasim. Welcome. Maybe just a quick one because I know you were at ASCO and then coming back from Chicago. Since you were expecting this pivotal data very soon, what have been the feedback you are getting considering this is standard of care combination and then how the physician will use if this gets approval?
Yeah, I can start and then Kasim can elaborate. ASCO this year was exciting for us. We are truly seeing a lot of expectations to the readout from our phase three trial. Everybody we are speaking with are just so excited with us. Maybe Kasim, you can elaborate.
Yeah, I think as Mai-Britt just mentioned, we saw a lot of excitement. I think most of our key opinion leaders, medical experts now see almost the final stages of it. They are all excited. They're looking forward. They all still believe that the data that we have as of now is hopefully going to reflect into the results from phase three.
Great. Right now we have a few options in the first line. Silent Bio is combining the current standard of care. I see some slides you mentioned if the OR is better than EpiNevo, it will be the new standard of care. Maybe just elaborate on this and what are the people looking for in the data in order to change the practice?
Yeah. I think as you know, with the current standard of care and Mai-Britt elaborated already, there is still a high unmet need even with the combination of Yervoy and Opdivo now becoming, especially in the U.S., sort of the emerging standard of care. Safety profile still remains a big issue for them. Within the sort of domain of median PFS, they are like 10-11 months maximum. With our base case target product profile, we believe that we would have a result better than both of them. Eventually when we show the overall survival data, plus the safety analysis that we have had so far, are quite encouraging. The benchmark obviously is in front of us. We are also looking at what's coming next. For example, Regeneron's phase 3 is about to read out as well, which will be another leg three.
Keeping all of those into consideration, we believe, and also that's how we've estimated that we could be the next standard of care if we meet our plans.
Excellent. Thank you.
Thank you.