Evening, whoever is on, and welcome to EXACT Therapeutics market update. We're very excited to have a lot of news for you today. Before I introduce our speaker panel, we'll have a whole hour here in front of you with obviously market update, technology update, the clinical update, and some of the plans that EXACT Therapeutics have. Of course, a key part of this is the whole deal itself, the capital news that you have been reading about. So today, I am Anders Wold, chairman of the board. You have here on my side, it's the CEO, Per Walday. John Edvin is the CFO, and Kasper Fosgaard is our CBO. You'll hear from all of us today.
Let me first kick it off with a little bit background on what EXACT Therapeutics and what market we're operating in. Many of you, I'm sure I heard about ultrasound, and ultrasound, primarily from a diagnostic point of view, has been kind of a layman's diagnostic method, from screening to pure diagnostics to actually some gold standard over the years, because technology has improved dramatically. I've been in this business for a long, long time, from startup to industry with GE HealthCare, as you saw from the previous slide, but now it's moving on from diagnostics and moving into new areas, so new targeted non-invasive treatment. Treatment is the key word here, and that's where EXACT Therapeutics come into play.
It's about drug delivery as a modality, and we have seen pharma VCs and other key players are moving into this space and making very exciting and some examples is, Insightec, for instance, recently you have HistoSonics raised, a lot of funding into these different spaces. So we see major players in this, market is positioning. And, and not to mention, of course, today we, we are announcing the successful private placement with GE HealthCare, which is now a cornerstone investor. It's been successful from the point of view that we have been previously a spin-out of GE HealthCare and over the last 10 years been working on one particular product that, we have proof of concept with our phase II, phase I study. We'll see that in a moment. And this whole successful, private placement leads us to a partnering situation with GE HealthCare.
And partnering is important here because you have ultrasound as a hardware, diagnostic equipment as such. You have another component which is microbubbles, which is also in the GE HealthCare portfolio. You have GE HealthCare as a brand name, household name. They own more than one-third of the market in the diagnostic space and more than that in microbubbles. So there's a major player in the marketplace, and we have therefore secured two main elements of our industrial plan. And you'll hear more about that in a moment. So being a global player in medical technology, a massive operator, obviously, the leading one in this space. And some of the proofs recently have been that GE HealthCare has moved into therapy. An example would be Novo Nordisk's recent agreement in type 2 diabetes, obesity, etc.
So you will see some of that moving in this whole space. So ultrasound is moving from diagnostics to therapy, and that's where EXACT fits into this space. So with that, I'll hand it over to Per here to talk a little bit more about the whole EXACT story.
Thank you, Anders, and thank you for the introduction. Yes, we are building a company which is, a leading next-generation precision medicines company that are utilizing this combination of ultrasound and specific drugs that can enhance the therapeutic effect, enhance the therapy of what is being treated today in oncology. We have a very focused oncology development strategy, and we have recently, earlier this year, pre-presented our phase I, our first clinical study in oncology and interim results at the American Association for Cancer Research. This was presented in the spring this year, where we show a 10x increase in percent tumor shrinkage versus what is standard of care alone. And this has been done in patients with cancer in the liver, liver metastasis. Furthermore, with oncology drugs, safety is often the limiting factor for how effectively you can use them.
We haven't seen any safety events at all in our study. So even though it's not that many patients, it's only 9 patients that have been through both the interim read and the expansion, there seems to be a very clean safety profile and a very strong efficacy signal. We've used intrapatient control, so we have a control in relation to within the patient on those liver tumors that have been treated versus those that are not treated, and that is where we see a 10x increase. We are now moving into a locally advanced pancreatic cancer. This is a devastating disease, one of the worst cancers you can have, with only less than a year of median overall survival. This is what we're going to fund with the money that we've now raised through this private placement.
And John will talk a little bit more about the financial runway in a few seconds. We have also for our technology, which is called Acoustic Cluster Therapy, ACT, or ACT, broad IP coverage. We recently were actually granted a first patent in immuno-oncology, which is the greatest revenue generator in oncology these days. And we have also recently established a collaboration with Agenus, which is an immunotherapy company in the U.S. So our focus is clearly on oncology, but CNS and the blood-brain barrier constitute the potential additional therapeutic vertical, and we'll come back to why this is potentially an important opportunistic path that we are also looking into.
To deliver all of this, as you can hear from and see from the experience of the management team, we have a very good industry team with strong industry experience and taking drugs from research all the way to commercialization. So what we have done now, we have received $7 million in investment from GE HealthCare. They are, as Anders said, the global leader in medical technology and the market leader in ultrasound, which is the component that we need for our treatment. It's a four-year collaboration which is focused on abdominal cancers and first then pancreatic cancer. They participated as a cornerstone investor, and in total we raised $13 million in this private placement.
This will fund our phase II study where we aim to show proof of concept in pancreatic cancer that we can treat and provide clinical benefit to patients with locally advanced pancreatic cancer. For this, GE HealthCare will be our partner, develop and supply the probes and the consoles that we need for our clinical studies. With this, I'll just leave it over to John Edvin Pedersen, the CFO, to talk a little bit more about the money we have and the financial runway and how we're going to use it.
Yep, thank you, Per.
Just, John, before you start, just one reminder for everyone. It's possible to put questions across, as usual in the webcast, so please do that, and we will deal with the questions at the end of the presentation. John?
Yep, thank you. As mentioned here, we now have secured the private placement, announced this week, raising $13 million, NOK 145 million in kroner. For us, that is a pretty important accomplishment because this will fund the EnACT study in the pancreatic cancer. Where we are now, we're leaving 2024, with the private placement in the bank, and going forward, we'll see that when we reach the interim readout in the first half of 2026, that will be the main milestone and the proof of concept. But going forward, we also see that the funding that we now have raised will take us all the way to the top-line results of the EnACT study, securing the clinical development of the company for now.
Okay, so, shall we move over to Kasper Fosgaard, the Chief Business Officer, to talk a little bit about our strategic focus and the upcoming milestones or news flow?
So, EXACT, we have a very focused strategy. Our primary focus is to execute on the EnACT phase II trial where our private placement has enabled that with the income flow of the $13 million. It's building on our strong data from both preclinically and clinically from our ACTivate trial. Second pillar is our immuno-oncology activities where we are executing on the research collaboration. We have Agenus and other companies and research universities. And here, immuno-oncology is the largest market opportunity there is within the oncology space. Opportunistically, we also have activities ongoing in the CNS space, blood-brain barrier space, where all the research and treatments within the brain is severely hampered by the blood-brain barrier. So to open that is a very large, and treating diseases of the brain is a very, very large market opportunity.
That leads us to all the milestones we are looking to and news flow we're looking to generate in the first half of 2025. So first of all, we are finalizing the EnACT study, and here we'll see the final results, including the three last patients here during the first half of 2025. Next is the execution of the EnACT trial. So as that matures, we'll see a range of milestones and news flow. Initially, the approval of the IND, so the application of running the trial in the U.S. from the FDA, we'll have the first patient dosed, and then we'll see the initial safety readout from the first three patients expected again mid-next year, which again is linked to the warrants that was mentioned by our CFO.
Further, we are also expecting to initiate the trial in Europe, so we'll also see milestones coming out from that. In parallel, we have various preclinical activities. So we have the immuno-oncology activities, as mentioned, our second pillar, and we'll have the updates on the CNS/blood-brain barrier activities, which was mentioned as our third pillar. Thank you.
I'll come back to in some further slides, thank you, Kasper. I'll come back to in some further slides later about what this really entails with regard to the EnACT study and also to our preclinical programs. But before we go to that, now we've sort of covered this from a high-level perspective. Let's have a look at the technology and what this really does. What is Acoustic Cluster Therapy that we are developing? It is a proprietary patented technology which is completely unique. We have a way of defeating biological barriers that hamper drug delivery within the body. When you give a medicine that needs to act locally to a patient, it acts locally in an organ like the pancreas, for example, then only a fraction of that medicine will actually reach the pancreatic cells or the tumor cells that need to be treated.
We have a technology with ultrasound and a specific microdroplet microbubble, which is our proprietary agent, PS101, where we can watch or look at the area where we want to increase the delivery of drug with ultrasound and increase that and enhance that drug delivery to the tumor while the patient is being treated with standard of care. We don't want to disrupt what the treatment is today, so the patient flow should go as it is. We only put us on top. So when a patient comes in, gets standard of care therapy, we give an intravenous injection, we locate PS101, we locate the tumor with the ultrasound, and we do our treatment, which then will enhance how much of that standard of care therapy is actually taken up into the tumor and providing the therapeutic effect that we are wanting to see.
So how does this work then, with this microbubble, a gas-filled lipid thing, and microdroplet? It's a kind of an oil drop that are connected together, that is injected. The way this works is that when we have located the area where we want to increase the delivery, then we inject PS101. When PS101, these droplets and bubbles pass through the ultrasound beam, they will be activated, and this activation leads to a much larger bubble which will get trapped in the area where we want the delivery to to increase. What we do then, and this is done with normal, low-energy, high-frequency ultrasound that is being used normally in diagnostic for diagnostic purposes in all hospitals today.
But what we do then is we adjust the frequency to a lower frequency, which is closer to what is called the resonance frequency of these larger bubbles, which means they start to expand and shrink and expand and shrink. And what happens is that they create waves and break up the pore and create pores in these small blood vessels so that the drug that is in the blood can actually come out and be spread into the tissue where it needs to be to work. So this is the simple mechanism, a very basic biological mechanism that works across a number of different drugs and across a number of different tumors, as we have shown in preclinical studies. So we are moving into pancreatic cancer, and pancreatic cancer is one of the worst cancers you can get. It is one of the deadliest cancers.
It's the 12th most common cancer, so it's not the most common. It's actually an orphan indication, which I'll come back to a bit later. But there is also a growing patient population, and in particular in younger population groups. I think the important thing is that even if it's not, even if it's only the 12th most common, it's actually the 3rd most common cancer death. So it's a really deadly cancer. We want to treat we have a local treatment technology where we can increase the uptake locally. So we want to treat those patients who have locally or local or regional cancers in the pancreas. So that is approximately the dark green and the slightly lighter green, 30%-40% of the patients.
We have done studies in preclinical models, in animal models with pancreatic cancer to see how does our technology work with the standard of care that is being used today. We have done this together with Dr. Daniel Von Hoff, a professor at TGen in Phoenix, in Arizona, one of the or maybe the main leading key opinion leader within pancreatic cancer in the U.S. This is the statement that he said in their own press release after publishing our results, that this are one of the most encouraging results that he has seen in preclinical models of pancreatic cancer, and that they look forward to being involved in the further development.
He is really experienced, has done a lot of preclinical work, and has been involved and led the clinical trial work that has led to the approval of three of the four drug combinations that are actually today approved for treatment in pancreatic cancer. So he was part of the advisory board that we established with both U.S. and European key opinion leaders in pancreatic cancer to design and decide how do we develop this to make this work as good as possible. What's the clinical population? What's the design of the study, etc.? That is the EnACT study, the study that we will now fund with the money that we have raised in this private placement.
It is a potential orphan drug designation that you can get for this because it's not a very common cancer, even though it's a devastating and the third most killing cancer, and which means that you would get seven years of market exclusivity on the market, after this has been approved in the U.S., and potentially 10 years in Europe if it, if an orphan drug designation is achieved also in Europe. The statement of Daniel Von Hoff was based on the results from this study. What you can see here are two standard-of-care treatments that are being used today within pancreatic cancer. What you see on the right-hand side is the tumor growth, and you can see the black figures here, black on the top. They show what happens if you don't treat at all.
The green and the orange shows what kind of delay in growth can you get if you treat with standard of care, and the red and the blue shows what happens when you add ACT on top of this. So 50% of the animals got completely rid of their cancers. This is the amazing result that he was talking about in his quotation in his quote on the previous slide. So what we're moving into is the EnACT
While if you cannot do that, you only go for what's called palliative treatment to try to delay the growth of the cancer. So moving, converting a patient from being inoperable to operable, from unresectable to resectable, that's a very important endpoint to measure. We will measure that in this phase II study where we add our technology on top of standard of care. In addition, we will also look, of course, at overall survival and see whether we can prolong that survival in the patients, even if they don't become resectable. Today, without surgery, these patients have a median overall survival of only 10 months. As I mentioned, the target population we're talking about is 30%-40% of the pancreatic cancer patients, as you can see in the red box, on the bottom left, bottom right side of this slide.
It also, as mentioned before, is a potential orphan drug designation for this indication, and there is also a potential to move into oligometastatic pancreatic cancer patients. Those who have spread but only a few metastases, only a few new additional tumors, and they normally first spread to the liver. And we've shown already in the EnACT study a 10x increase in shrinkage of tumors in the liver, so we know that we can treat the liver. So there's a potential also to expand from locally advanced to those with oligometastatic pancreatic cancer, but that's not the EnACTed study. That is a potential expansion at the latest stage. The EnACT phase II study is a proof of concept study where we include 25 patients with locally advanced pancreatic cancer.
The planned geographies for this are the United States and Europe, and we are in the process of, as Kasper mentioned, opening an IND with the FDA in the US to start that clinical study. We are looking at both safety, of course, and efficacy in first-line borderline resectable and locally advanced pancreatic cancer. 3 patients to start with as a safety run-in because it's a new organ. We've never used ACT on the pancreas before, just to ensure that we don't see any safety signs. And then we have the safety review, and that safety review is for opening up so that all sites can include patients at full speed, and then to include a total of 25 patients on the standard of care plus our treatment.
An interim analysis is planned on the first half of 2026, and the final analysis will be available in the first half of 2027. And the safety review here is where the warrants that were also given to those who invested, if that is positive, they can then execute the warrants to, buy further shares and support the company further with those warrants. We also mentioned that we are working preclinically in immuno-oncology. So why is this important? This is the greatest revenue generator in oncology. It is a potentially, it's it's a treatment that, patients, when they respond to the treatment, they often respond for a very long period, and it's something that can almost make, oncology to become a chronic disease if it works over a long period.
What this really is is that you engage and enable the body's own immune system to actually attack the cancer so that you don't use a drug that kills the cancer cells, but you enable the immune cells in the body to go in and kill the cancer cells. There are very large sales drugs like Keytruda from Merck and Opdivo from BMS that are providing many billions of dollars in sales in 2023. So it's a huge area. The problem with this is that there are few patients that actually respond. Those who respond are lucky, but there are few patients that respond to this. And if you can increase the response somehow, that is really an area where you can improve the clinical benefit of these, what's called checkpoint inhibitors, these immunotherapies.
We have been working in this area to see what can we do with our technology, and we were granted our first immuno-oncology patent in the first territory, a few weeks ago. And this patent is covering EXACT proprietary Acoustic Cluster Therapy in combination with immunotherapy in cancer and in autoimmune diseases. We also have a preclinical research collaboration with an immunotherapy company, Agenus, in the U.S., who are developing this kind of immunotherapeutic checkpoint inhibitors, and also with The Institute of Cancer Research in London and TGen in Phoenix, Arizona. This is a potential expansion area that we are looking at preclinically. Another expansion area that we are looking at more opportunistically is can we do something in the CNS to temporarily break down the blood-brain barrier and allow drugs to actually come into the brain and have their therapeutic effect.
Now, targeted delivery to the brain is very difficult, because this blood-brain barrier is there to protect the brain so that toxic substances cannot enter from the blood into the brain. But it also means that medicines are hampered in the delivery into the brain. It's difficult to get medicines over the blood-brain barrier into the brain, and only 2% of small molecule drugs and almost no biological drugs can pass this blood-brain barrier and enter into the brain. So it's a formidable barrier for medical delivery. What we have shown with our technology is that we can with Acoustic Cluster Therapy temporarily open up the blood-brain barrier and provide delivery of what you saw at the top level here, a gadolinium contrast agent, an MR image, and at the bottom, even larger micelles that are being delivered.
So large structures can be delivered with our ACT technology, and this can be done without seeing any hemorrhage that's bleeding into the brain, and no severe inflammatory reactions to this. So an opportunistic area that we're looking into because of the high unmet medical need and the very good technical fit with our technology. So coming to conclusion and key takeaways, we have some really encouraging clinical and preclinical proof of concept for Acoustic Cluster Therapy, ACT. The EnACT study has shown a 10x increase in the shrinkage, in the percent shrinkage of tumors, that were treated with our technology. And we have done studies across a number of animal models with a number of different drugs and consistently shown that we can dramatically and significantly increase the effect of these drugs in these models. We have a very clear and focused oncology strategy.
The phase II EnAct study in pancreatic cancer is our first priority. We have now, with this private placement, the cash, and we have the experienced management to execute on this strategy, to go forward with this study to proof of concept, to show clinical benefit in this high unmet medical need of pancreatic cancer. We also have number one medical equipment company as an invested partner going with us, as a strategic partner going with us, providing the medical equipment partner that we need to be able to realize our strategy. As you have seen, a rich news flow and many near-term milestones, across the clinical and the preclinical work that we're doing. We are planning to have a Capital Markets Day, in the spring, sometime in the spring 2025, to give a further update on this.
So I think this is taking us to the end of this presentation, after 30 minutes, which is according to plan. And then, do we have questions that we have received from the webcast?
Yeah, we have some questions from the audience, and yeah, feel if you feel like further questions, please do send them in. So, first question, I guess, would be to Anders. What is GE HealthCare's intention behind their investment?
I'd be a bit speculative. That's okay, that's interesting. Of course, we would like to have GE HealthCare here to respond to the question, but so we can speculate a little bit. However, I would say there's obviously GE HealthCare becoming a partner, before now a vested partner and a significant partner.
We already know quite a bit about the background and the whys and the intention to some extent. So from a GE HealthCare perspective, being in a diagnostic space, a major player, you don't want to lose the opportunity with the market is developing on different fronts. That's been the essence of how ultrasound has developed from day one, say maybe 30 years ago, where there was like a very basic stuff and there's a lot of hardware and clinical development, and you move forward with all these different things. For the last 10 years, there's been some movement in using ultrasound as a diagnostic tool and visualization tool for therapy. So this step here is moving to the next step again to enter into the therapy market.
Combined with the microbubbles from GE HealthCare, obviously, you don't want to miss out the next step, which is a true therapy area and partner with that area. So that's, I would say, number one, to not miss out the market. That's a number one piece. The second piece is to be leading in the ultrasound area because you probably want to develop further hardware ultrasound technology to make certain it fits and it evolves into this space as well. As you now go into radiology application like the pancreatic one, you want to go into the blood-brain barrier. So you want to go in different organ, different places. That is also giving some opportunity for a hardware company to develop new technology in that space. So you want to don't want to miss out on that either. So that's not the intention.
I also say, obviously for GE HealthCare being probably the number one medical player, the medical diagnostic player in this space, you want to protect your brand position and be around that. Of course, that means the network that company is working on is expecting that you actually lean forward and be in the forefront of many of these developments. So I think some of these are different things. Obviously, to be an investor, it's not normal for GE HealthCare to invest in a public company, but doing this, obviously, there will be an opportunity to create additional value for the company itself and its own shareholders. And that is obviously what we are looking for too, that we want to build all of this somewhat together. I would say that's probably summing up.
I would like to add one more comment. It's not related to the question, but we have also signed up a joint development agreement with GE HealthCare, which protects both parties in terms of new development, in terms of IP, both hardware for us with the PS101, etc., and so on. So that's going to lead us through the 2 years, all the phase II and next study, and also positioning us for the next phase where we want to commercialize a product. So I think those areas are kind of tied really at the hip here and how we are set up for the future.
And a second question from the audience, how fast can you be on the market and when are you going to be income-generating and profitable?
So how fast we can be on the market depends on what the results look like, of course, because the studies you need to do is, the smaller the effect you have, the bigger the study is. But if we look at what we've seen so far, we've seen really good effect size in the preclinical models, and we've seen a, as I mentioned, a 10x increase in shrinkage of the tumors, in percent shrinkage of tumors in the Enact clinical trial. If we see really good effect sizes, and this is a very high unmet medical need, as I've mentioned several times, then it's possible to discuss with the regulators whether we can get an accelerated approval of this because there is such a high unmet medical need.
If we if we get that, the good results and we can get discussions with the authorities around this to convert it into what's called a pivotal study that leads to registration, then we can probably be on the market in 2028, which is relatively quick when thinking that we're moving into 2025 now. But this is of course due to the high unmet medical need and a good effect size in the treatment. So when can we be revenue-generating? As soon as we are on the market, probably 2029 should be a revenue-generating area, year for that. And I can add that being a small biotech is not part of our strategy to build our own sales force, but rather partner with a large pharma partner.
Yeah, but that's, I mean, we're open to discuss any potential possibilities, but the normal biotech growth is to find a good commercialization partner that licenses the product after approval concept.
And talking about the phase II and next study, we have a question. Do you have a CRO, a clinical research organization, as a partner to conduct the trial?
Of course. This is a complex undertaking. We are a small biotech. There are experts in all the different aspects of these clinical trials and global clinical trials. So we have a global CRO that we have engaged to this, and we've been working with them now to prepare for the trial. So they are ready to go, as soon as we get the regulatory approvals that we need to start the study. And we have a question for the CFO.
Will there be a so-called repair emission for existing shareholders? That is a little bit dependent on how we see the volume of the shares trading now. We see that it's been quite high volume over the today and yesterday, but it is a decision that the board will make shortly.
Last question for the moment. What does Orphan drug designation do for the pancreatic cancer indication and its market opportunity?
It is an important factor because it gives you and provides you from approval a market exclusivity within that area for your drug. It also provides normal very high price tag for your for your drug. Orphan drugs are priced up to, yeah, $500,000 per patient per year. So it can be extremely high value products that come out of Orphan designations.
Normally in oncology, I think it is around between $200,000-$300,000 that is what you normally get paid for these kind of drugs per patient per year. So it's important both for the price tag that you can get for the, for the product and also for the market exclusivity and the protection in addition to the patent that you have. With that, if there are no more questions from the audience, we can give it 10 seconds. Otherwise, I think we will conclude this session
. Okay, maybe while you guys are thinking about any potential questions, let me maybe wrap up our session here. First of all, we're extremely excited about the opportunity here that this whole fundraising has given us. That's number one.
Very excited to have GE HealthCare as a strong partner and not only as an investor, but really as a partner from everything from technology to clinical, etc., and branding, etc., although we are agnostic to the future. So that's an interesting setup, well covered financially to execute our EnAct study, ready to go. So that will be the primary opportunity. But also you heard that our solution here is not just specifically pancreatic. It is actually a platform from a delivery of drugs to drive more efficiency and in treatment. So that's the core message there. We'll set up with our team here in terms of competency, expertise, and connections to the marketplace. So we're very happy about that.
As Per mentioned, we will give frequent updates as we go through these milestones, going forward. We'll give an update in the springtime with the market day for all investors, etc. So, stay tuned for that. In the meantime, feel free to connect with the company if you have more questions. Thank you all for your attention. Thank you.