Hello, welcome back to ABGSC Investor Days. My name is Georg Tigalonov-Bjerke. I'm an equity research healthcare analyst at ABG. With us now, we have Magnus Corfitzen , the CEO of Ascelia Pharma. With that, Magnus, I give you the word.
Thank you, Georg. Great to be here. Thank you for the invitation from ABG to present to all of you today, talk about Ascelia Pharma and our very special year, 2026, with major milestones coming up. In my presentation, I will be making forward-looking statements. In Ascelia, we're focused on developing, or sort of identifying, developing, and commercializing novel drugs that address unmet medical needs within rare cancer. Our company is based in Malmö in Sweden. We've been listed on NASDAQ Stockholm since 2019. Our pipeline consists of two products. The lead product is Orviglance. I'm going to spend the majority of the time here today talking about Orviglance and its diagnostic drug. I'll come into that a lot later. We've gone through the clinical development.
We have a PDUFA date, which means the day that the FDA will be making a decision on approvability coming up in early July, getting excited about the upcoming milestone. The other program we have, I'm going to touch upon that in the end. It's a therapeutic drug, completed phase I development, also very interesting opportunities within a number of different solid tumors. Let's start with Orviglance, our diagnostic drug. I'll start with why we are so excited about Orviglance and what it does. It's a diagnostic drug that addresses a well-defined unmet medical need in a patient population that have poor options today. That translates into an $800 million global market opportunity, half of that in the U.S., a very attractive opportunity for us to transition into commercial stage. We have manufacturing capacity in place to supply the market.
We have completed nine clinical studies with consistent good results, that has led to the submission to the FDA that we did in September last year, and which is now why we expect a decision in early July on approvability. We also have a clear strategy. We are a development stage company. We're not a commercial stage company, so we will partner, and we are having a process with various potential partners, in terms of finding the right one that will drive commercialization of Orviglance. I'll come back to that. First let me go a step back in terms of the well-defined unmet medical need. Cancer is obviously a major problem for a lot of people, and it's often a cause of death. Within cancer in the liver is a major problem. Very many cancer types spread to the liver, it metastasizes to the liver.
The liver is actually one of the organs that have the most metastasis to it, also very often the first metastasis, which has important implications for how the doctor should treat the patient. The way to deal with that in clinical practice is to do medical imaging of the liver. The best way to do medical imaging of the liver to determine cancer or follow up on cancer is an MRI, [Non-English content] in Swedish. To get the best MRI image, today you need to inject the patient with a gadolinium-based chelate into the bloodstream that will provide enhancement of the liver. In most patients, that works fine. In some patients with severe kidney problems, they're unable to excrete the gadolinium as fast as people with normal kidney function. They are at risk of something known as nephrogenic systemic fibrosis, which is a potentially fatal side effect.
That's why the regulatory agencies have put in place black box warnings to highlight this safety concern for these patients. Those are the patients that we're going to make a difference for. Orviglance is based on manganese, so we have no gadolinium-associated safety issues. We have a liver-specific agent that goes to the liver, minimal systemic exposure, so almost nothing in the bloodstream, which makes us a very efficient and targeted product for these patients. We have an orphan drug designation from the FDA for this patient population. That population translates into the $800 million market opportunity I mentioned before. Almost half of that is in the U.S. In our development, we have prioritized the U.S., and prioritized the conversations with the FDA, but we've also had conversations with other regulatory agencies, such as the EMA in Europe.
The patients are very sick because they both have cancer and severe renal disease, and they are typically treated in the major medical hospitals. That creates a focused launch that the partner will be driving. Zooming in a little bit more on the U.S. opportunity. Based on very extensive market research, we've been looking into how many procedures are actually taking place in the U.S. that are billed in the system. These are based on real imaging procedures happening in real patients and being paid for. These data tell us that about 100,000 procedures, and these are 2019 numbers, around 100,000 procedures annually in our target patient population. It also tells us that 75% of those imaging procedures were taking place in the largest 400 accounts.
Again, that shows a concentrated, you could say, location of the patients, which means that the commercial footprint needed to drive commercialization successfully is a lot smaller, opening up to a wider range of partnering opportunities. We've also done extensive pricing research to understand the value we bring and how that can be priced into the healthcare context and have some good benchmarks and pricing feedback from peer representatives. All in all, a very interesting opportunity on the dynamics starting in the U.S. Again, just to come back to the regulatory agency, you have the warnings on the gadolinium products. It's also something that is well-recognized in the clinical community. The doctors are quietly aware that there are risks with gadolinium.
Some of them, a fair number, have even experienced nephrogenic systemic fibrosis, even though it's a rare condition, partly because many of these patients are not getting gadolinium in the first place. Sometimes that is necessary in the current practice when there is not a good non-gadolinium agent available. I think the fact that they know it tells to the, you could say, severity of the side effect. We are targeting, as mentioned, and that's why our orphan drug designation is the black box warning patients, the patient with severe renal disease. Some of the other concerns with gadolinium is the fact that every time gadolinium is injected, there's deposition of gadolinium in the tissue. That means some of the gadolinium stays in the body. It does not leave. It happens primarily in bone and in skin, but it's also deposited in the brain.
Obviously, heavy metal deposition in the brain following a medical procedure is not something you would want. There's a lot of research going on to understand what are the long-term consequences. There's also increasing focus on the environmental impact. When the patients have been injected with the gadolinium, it leaves through the urine, goes into our sewage system, and there it increases. There are numerous publications where scientists have measured gadolinium content in drinking water over the last few decades, and it has risen tremendously. Still not one of the major concerns on drinking water, but I think it's important for a number of reasons to minimize the heavy metal deposition in drinking water overall. All these major trend drivers are favoring Orviglance. We're leading the way to get a manganese agent on the market here with the PDUFA date coming up.
We have GE Healthcare , one of the large gadolinium manufacturers. They are developing an injectable manganese agent. They've completed phase I, recently started a phase II/III study, which we think is a great event. Our products, based on the data we have, is that they are complementary. They have a manganese agent that is injected into the bloodstream, stays in the bloodstream. Our agent is a liver-specific agent that the patient will drink in a glass of water like this, and then it's taken up from the small intestine to the liver. The manganese does almost not get into the bloodstream, which gives us a very unique safety profile. It provides a liver enhancement that, based on the data we have, the GE agent does not have.
I think that's a good development that some of the large companies in the space are also saying manganese is a part of the future. Some of the other gadolinium manufacturers have developed lower dose, they've taken their older products and been able to reduce the amount of gadolinium per scan, the patient would get less gadolinium to have a scan, they still get gadolinium. Sometimes a patient can have severe side effects after even a small dose. Some patients will have a number of high doses throughout their lifetime, they will not experience anything. It's very difficult to predict in general who will have side effects. The severe renal impairment patients are well-defined as being the highest-risk patients.
Overall, the conclusion of this message is really when you look at the trends in radiology and in particular in the MRI space, Orviglance is really well-positioned and in sync with the developments at this point in time. We're really pleased with that. That brings us to the partnering. We are approaching the approval date. We are also having conversations with a number of companies that could be strong commercialization partners. Those conversations progress, and obviously, it's long discussions and a lot of things that we need to cover in those conversations. We bring to the table basically a product that has on the brink of FDA approval. There's a clear, well-defined unmet medical need. It's well-recognized both in the medical and regulatory and other societies that there is a need for a better product for these patients. We're excited about where we are.
We're not there yet, but we're working every day to get there. The other part of getting there is obviously getting the product approved. That's the other key milestone we have. As mentioned, we have completed the clinical development with nine different clinical studies. High level of consistency in safety and efficacy. We have had conversations with the regulatory agency throughout the development and are very happy with where we are now. Just to give sort of on the phase III data, and some of you who have been following Ascelia for a while have probably seen this image before from our phase III study. It just illustrates the enhancement and the value that we provide with Orviglance.
To the left, you see the unenhanced scan, and our market research tells us that for the target patient, the patients we go after, the majority of them, according to our research, gets the left-hand side scan, which has inferior quality. That means that there is a risk that they will miss cancer in the liver. There is a risk that they will not interpret the development, the patient's response to drug therapy in the right way. If they're getting chemotherapy or antibodies for treating the tumor, if they are not able to measure growth correctly, they may take the wrong decisions in terms of continuing on therapy or changing to something new. With these images, we have high quality, and you can also see that on the scoring, which is a little bit complicated on a 4-point scale.
That this is the delta, basically anything above zero means that Orviglance is better. You see the 95% error bars. Really strong level of significance on this primary endpoint. In terms of where we are, we have the mention, we submitted in September last year. We got a Discipline Review Letter in November. The review process is on track. We have a dialogue with the FDA that is, I would say, customary as one would expect in terms of they're asking questions and doing all the other things, including audits that they should be doing. We're excited about where we are and looking forward to the PDUFA date. That's an important milestone. Another important element is obviously when we get to market, the orphan drug designation will provide us with seven years of market exclusivity in the U.S.
The FDA will not approve a similar product for the same indication, giving us a strong natural protection. We've also filed IP, both on a food effect. It's a product you drink. We have demonstrated that you can have certain kinds of food in certain amounts prior to the administration of Orviglance. We hope to get that patent. We also filed some manufacturing patents, where we've not disclosed more than manufacturing, which we are also very excited about in terms of, you say, extending exclusivity for the Orviglance franchise. We have issued patents for next generation Orviglance product. Today, Orviglance is a powder that is mixed with the water. This IP, these patents cover taking the Orviglance product into a tablet. It could be a dissolving tablet, it could be an effervescent tablet.
That could be a next generation product that would have exclusivity. Again, one thing is the near term, but the value we provide with Orviglance is, for the long term, finding better solutions for patients over the next decades. With that, I'll switch gears and go to Oncoral before we get to questions in the end. Oncoral is a therapeutic drug. It's based on irinotecan. Irinotecan is being used every day, throughout the world, for treating cancers, in particular colorectal and pancreatic cancers. Irinotecan today is given as an injection or as an infusion. The patient will typically go to the hospital every second, third week, get a very high dose, have very high level of side effects in the first couple of days, and then as they gradually recover from the side effects, they're scheduled to get the next dose.
The good thing is that irinotecan is very good at treating the cancer, but it's a very difficult regimen, and many patients do not tolerate. Therefore, we see a significant improvement opportunity by giving daily dosing of irinotecan. Giving a lower dose every single day, so you don't get to this excessive level of drug exposure that cause these massive side effects. Over the course of the treatment, it will be potentially better safety, better efficacy. In the phase I trials we did, illustrated here, you see the concentration of the active metabolite, SN-38. That's an important marker for how, you could say, exposure to the tumor of this active metabolite, and we think there are definitively benefits of that. We saw that on the safety profiles on these early studies. Studies were in very sick patients, so not designed for any efficacy signals.
Still, it was encouraging to see a number of patients having stable disease. These patients had been tried, I think, three to six different kind of drug treatments before coming to the trial. Some of them even had stable disease when they've had growing tumors with injectable irinotecan. Proving that the constant exposure, constantly having anti-cancer drug in the bloodstream is helpful and much better than giving a high dose all at once. We're very excited to move forward with this in due time. We are initially focusing on gastric cancer, but there are many different solid cancer types where irinotecan could play an important role. As mentioned, to date, it's approved in colorectal cancer and pancreatic cancer. Before going to questions, I'll just round up with our focus on the milestones, right? We have two milestones.
One is getting Orviglance approved. The other one is getting a deal on Orviglance. That's what we focus on. We are well on track for both of them. We are making good progress on the regulatory process. There's a timetable. We expect in early July to have a decision from the FDA. We're optimistic for a positive outcome based on the interactions we've had to date. On the partnering, we are continuing to work with our key opinion leader network in terms of preparing a lot of the market launch activities. We are also having some serious conversations with a number of companies that we see as potentially very strong commercialization partners that we can work with and support so they can drive the adoption of Orviglance to patients who need it. With that, I'd like to open up for questions.
Thank you for that nice presentation, Magnus. We have a few questions. First, you touched upon these things, but perhaps you can give some more details, favor. First of all, on the FDA regulatory process, especially the PDUFA action date on the 3rd of July. How has it been and what will happen going forward also?
It's an exciting process and this is my first PDUFA, but fortunately we have a number of people in my team who have been through several. Basically, once you submit the FDA review team, they're reviewing and they will ask questions and then we'll say, "We need that answered by Thursday next week," or kind of. We get scrambling going from doing some other stuff and then putting everything aside and answering those, responding to the FDA. That is the process that goes on throughout the review period. Typically they will also do, I mean, almost always, I guess, do audits and it could be the company, it could be Ascelia, it could be manufacturing, it could be hospitals that are participating in clinical trials, could be CROs that have been involved.
Everything can be audited by the FDA, and then you get the audit reports, and then if there are any sort of major critical findings, you will need to respond to those, so that the FDA can be confident that things have been done in the right way. That's kind of the cycle. It's basically, you don't know anything for sure until you get the approval letter. Obviously we see it as a positive sign that the process, the interactions we've had, the questions we've had, and our responses to that, everything is pointing in the right direction. I think based on where we are, we couldn't be much more happy than we are at this point.
Exciting. Very exciting days coming up. There's also one other very important milestone that you have spoken about, and that is finding a partner to do the commercialization with. Can you say some words about what you're in particular looking for in a partner? How you are going to drive the most value post-approval as well?
Absolutely. We're a small company, and we will not be able to do a good launch ourselves. We would need to build everything from scratch, and that's not in the interest of Ascelia Pharma and shareholders. We have a clearly stated strategy, we want to bring in a partner. Obviously, us taking the product through the clinical and regulatory development means that what we need from a partner is somebody who's really good at getting approved drugs used in patients in a hospital setting. The partners that we are thinking about would be the optimal partners and the companies we speak to are companies that have sales forces targeting U.S. hospitals, in particular, the large hospitals. You could say they are in the databases, in the IT systems, they have procurement, they have the distribution chain set up.
What they need is, they need Orviglance and they need the experience we have and our key opinion leader network and other key people who can support them in bringing the product to market. They would take more and more over. It's a broad range of companies. We are speaking to companies that you would expect for a radiology product. We're talking to those companies. We're also talking to specialty pharma companies that have hospital-focused sales forces in many different therapeutic areas. Some of them that are very adjacent to what we have here. Some of them who say, "This could be an important product in the strategy that we have in terms of focusing more on these specialties." It's really, I would say, a diverse group of companies we speak to. I think it's also different structures.
Some are interested in having global rights. Some companies are interested in having U.S. only. Then if that ends up being a U.S.-only deal, we'll work with that partner in the U.S. and then we'll find partners for Europe and Asia and other geographies. It's really an exciting period of time trying to put, you could say, the pieces in the jigsaw puzzle together and make sure that we get it done right and bring on a partner who's committed and really willing to make the investment into the launch. Also a partner where this is a strategically important product for them.
What would the role of Ascelia be in this partnership going forward? What kind of support are typically partners looking for from you and what are you able to provide there?
Yeah.
Yeah.
That's a good question. Again, it depends who the partner is, what they come with and what we come with. Obviously, we know the most about Orviglance. We know more about Orviglance than anyone else. We want to share that with the partner. We want to help the partner succeed. Again, depending very much on the deal structure. If it's a global right, they need to bring it into the global organization. We'll be busy trying to support all the different regions. If it's a U.S. only, we'll be focusing on U.S. and trying to help the U.S. team do it in a really good way. They will drive everything from commercialization and onwards. Depending on geography and others, we may still have manufacturing. We may be working on the next generation opportunities.
One thing is obviously getting the approval and getting the deal. Those are the key objectives we have. I think a very important element is also to work with the partner after signing the deal and helping maximize the revenue stream for many years to come. I think that is a really important parameter. Right now we're focused on the execution on the two major milestones right now. We're also preparing to say after those two milestones, what does Ascelia look like? How are we utilizing our knowledge and our capabilities in the best way to drive long-term value, not just for 2026, but also the years beyond? I think that's a very exciting opportunity, and I think we are well prepared for that.
Great. Time is running very fast here, so I think we'll leave it at that.
Yeah.
Thank you very much, Magnus.
Thank you.