Ascelia Pharma AB (publ) ($ACE)

Welcome to today's event. We have the pleasure to present Ascelia Pharma [indiscernible] presentation and answer questions in the end. We are joined by CEO, Magnus Corfitzen. Today's agenda is, of course, your Q1 '26 results, maybe they are a little bit pale through all the news flow and the very important time you're going into with about 2 months to potential PDUFA date. I think we will cover that probably more, and there's a lot of questions on the partner side. That we will also take in the end of the presentation. There's a box down below, already claim a lot in on the partner side, see if you have something a new angle then ask, but I think we are pretty good covered there. For now, I will hand the call over to you, Magnus.

Yes. Thank you, Michael, and it's great to be here talking more about our Q1 update that we have. I'll be making a number of forward-looking statements during the presentation and the Q&A session. At Ascelia, we identify, develop and commercialize novel drugs that address unmet medical needs within rare cancer conditions. We're based in Malmo, Sweden and traded on Nasdaq Stockholm since 2019. We have 2 drugs in our pipeline. We have Orviglance, which is a first-in-class diagnostic drug for a subset of patients undergoing liver MRI imaging with orphan drug designation, and we've completed the development, submitted an NDA application for approval to the FDA. They have set a PDUFA date on July 3, a little less than 2 months from here, where they will make a decision on approvability. Very optimistic, and I'm going to spend the most of my time here today talking about Orviglance and what we do there. We also have Oncoral, a very exciting program, daily dosing of irinotecan. I think that's a great opportunity to look into. We have some good early data from Phase I. For the treatment of solid tumors, this is an interesting asset to watch. In the Q1 was very much focused on operations in terms of moving forward on our key strategic objectives on approval and partnering. I'll come back to that. We announced that we have an oral presentation of Orviglance data at the ESGAR Conference, the European Society of Gastrointestinal and Abdominal Radiology, taking place in France this year, Montpellier. That was a very good piece of news. The announced changes in our management team were sort of implemented Anton Hansson started as our CFO and Julie Brogren moved on to a new role outside of CEO. After the close of the quarter, we raised SEK 20 million in a directed share issue. That extends our financial runway into next year. That was an important element in terms of making sure that we are not getting under, you could say, having a negotiation disadvantage because we would be running out of money in early Q4. That was a good transaction to do, and I think very much in the interest of shareholders. I'll now go into the portfolio. I'll start with Orviglance. This is why we are so excited about Orviglance. It's a really unique opportunity with a well-defined patient group that have a widely recognized unmet medical need, patients undergoing liver imaging with severely impaired kidney function. That translates into an $800 million global addressable market opportunity. We have commercial scale manufacturing in place. We have completed the clinical and non-clinical development program, 9 clinical studies in total, very consistent safety and efficacy result, everything pointing in the direction that the benefit that Orviglance offers to these patients is strong. The NDA has been submitted. They are reviewing the application and the review process is going well. We have a clearly stated strategy to commercialize with a partner. Partnership is not in place yet, but we are making good progress on that. All in all, we've taken this asset from back in the early days and now on the verge of approval and getting it out to patients and helping them building a good solid business at the same time. I think that's a very attractive journey. Let me take one step back and talk about what problem we're solving. I mean, cancer is a major burden on human health and cancer in the liver is an important issue within cancer care. Many different cancer types metastasize or spread to the liver. They start in one organ, could be the colon, could be the breast, lung, other highly frequent cancer types. Then they metastasize, they spread to the liver. There are also some tumors that start in the liver, but when we look at cancer in the liver, overall, about 90% of the cancers in the liver spreads to the liver does not start there. Very often, the liver -- it's one of the organs where metastases occur more frequently. Very often, it's the first organ of metastasis and very often also the cause of death. Understanding the cancer burden in the liver and treating it aggressively is important for patient outcomes. The way that is done is in medical imaging. The best medical imaging for cancer in the liver is an MRI imaging procedure. That's both for -- you take initial diagnosis, planning surgery, follow-ups for surgery, monitoring patient response to therapy. When they get drugs to treat the cancer, is the tumor shrinking, yes or no. Then post-treatment surveillance is the cancer coming back. All of that requires medical imaging. A patient will undergo a number of imaging procedures throughout the treatment of their disease. As mentioned, MRI is the preferred method is the best method. To get the best quality MRI, the patient will have an injection of a gadolinium-based contrast. Gadolinium is a toxic heavy metal, so it's chelated to bound in a chemical structure to minimize the toxicity and get the right profile of the drug and excretion and it works well in the vast majority of patients. Unfortunately, patients with severe kidney problems, they have risk of a potential fatal side effect known as nephrogenic systemic fibrosis, which can be fatal. As a consequence, there is a Black Box warning on all gadolinium products for this safety profile and patients with severe renal disease are identified as the patient group with the highest risk of this condition. Those patients are the targets for Orviglance. Orviglance is based on manganese, so there's no gadolinium-associated risks. It's liver-specific, which gives us a great imaging of the liver as opposed to many of the gadolinium products, which are injectable and have a systemic exposure. We think we have a very strong value proposition to the target patient population with Orviglance. When we think about what that value proposition translates into from a commercial perspective, that's an $800 million addressable market globally. Almost half of that is in the U.S. Our market research also tells us that these patients, because of their multiple diseases, they are most often treated at the large medical centers, which means that the commercial launch, the commercial infrastructure needed for serving this patient population is rather focused, providing good economics. Our strategy, as I mentioned, is to have a partner drive the commercialization in all geographies. We have prioritized the U.S. because that's the single biggest market. Our market research demonstrate based on real-world data that about 100,000 procedures are performed in our patients annually. We also see that they are concentrated in about 400 medical accounts or at least 75% of them are concentrated in the 400 largest accounts. Sort of a nice commercial opportunity. We've done extensive payer research, market research in terms of pricing and reimbursement, and there are some good benchmarks in terms of high-value innovative diagnostic drug at around $3,000 to $4,500. We think this translates into a very tangible near-term opportunity. We are focusing on the Black Box warning patients, the patients with renal disease. There are also some other concerns with using gadolinium, it's deposited in -- whenever you have an injection of gadolinium regardless of the kidney function, there is a gadolinium deposition. Most of the gadolinium leads the body again rather quickly, but some of it is accumulated in especially bone and skin, but also the brain. Studies are underway to sort of look at long-term consequences of that deposition. Another problem is the water contamination that when patients are injected, the gadolinium, most of the gadolinium is excreted through the urine. It goes into our sewer systems. Unfortunately, our filtration systems for water is unable to clear the gadolinium, which means that there is an accumulation of gadolinium in our drinking water, because we kind of filtrate and reuse that the level of gadolinium has gone up quite significantly over the last few decades, and the curve is looking to continue. These are some of the key concerns about gadolinium and the dynamics in the marketplace. When we look at what are the trends, what is the industry doing to address those concerns, we're very happy to be sort of leading the alternative to gadolinium space with our manganese agent. We've seen that GE Healthcare, one of the large gadolinium manufacturers are developing an injectable manganese compound. It's found a chelate. In many ways, similar to the gadolinium products on the market, but using manganese instead of gadolinium. They have completed their Phase I study, have recently started a Phase II/III study, and we think this is a great development. This is not based on what we know, a liver-specific product, which means that it enhances the blood vessels, whereas Orviglance enhances the healthy liver cells. These products are complementary, and we are happy to have other companies talk about the benefits of using manganese instead of any other products such as gadolinium. Some of the other gadolinium manufacturers are developing lower dose gadolinium. Again, the overall trend is that we should minimize -- gadolinium is not good for patients on the environment. Using less gadolinium or no gadolinium is the way to go. That is a clear macro trend in our space. Again, happy to be leading the manganese ratio. As mentioned, we're focusing on research and development. We do that well. We have completed the development. We've submitted an NDA to the authorities. We are in the process of securing a partnership for commercialization initially with a focus on the U.S., but could also be sort of a larger geography than just the U.S. We have different conversations and different geographical scopes. We offer a very attractive asset, Orviglance derisked because we brought it very far. There's a high well-documented unmet medical need, and that transcends into a payer value based on our market research. We have identified the key hospitals to target, and we think this is a really good package also with the derisking. That is what we're discussing with the partners that's progressing, and we're happy with the outlook and optimistic on getting a deal in place. The other part of, you could say, the process here is that we've come very far. Obviously, the FDA approval is a major milestone. It's based on an extensive clinical development and non-clinical development. We have consistency in the data from between the trials showing Orviglance significantly improved medical imaging versus unenhanced. We see more lesions, especially the small ones, which are important for medical decision-making. We also see a consistency on the safety profile, which is we consider very favorable in terms of this patient population. Overall, strong data package and well recognized in the clinical community and also having presented in publications and at various medical conferences. Here is an example from a patient in the Phase III trial. You see to the left, the image to the furthest to the left, you see the unenhanced. You see some black dots that are clearly visible. They are very large tumors in this case. When the patient gets Orviglance, the manganese goes into all the healthy hepatocytes and that provides a much, much clearer image of the liver. You see a clear delineation between the size of the tumor. When you do this in a follow-up setting where the patient could undergo drug therapy for 3 months and then you would hope to see the tumor shrinking, when you have that high accurate, highly accurate image, you're able to assess whether the tumor is indeed shrinking or it's growing. You don't have that kind of variability, same level of variability. We also see another marked by the Oncoral and improved detection. We see a lesion that is not visible on the unenhanced, but clearly visible on the OCA enhanced. Again, one example, but to the first right, we have the outcome on the scoring scale that all 3 readers, highly statistical significant improvement and the 95% error bars here show that we are very far from 0, which is kind of comparability. Again, solid data that has gone into the NDA that we submitted on 3rd of September last year. We got the data in the full letter acceptance review and set a PDUFA date of July 3. Very excited about that. We have orphan drug designation, which will provide 7 years of market exclusivity post approval in the U.S. On top of that, we have issued patents on the next-generation version of Orviglance. We have filed additional patents on using together with flu and also on manufacturing, which we think are very important to extend exclusivity quite significantly. Very excited about the outlook for Orviglance, both on the regulatory approvability path and for the partnering outlook. With that, I'll be transitioning into Oncoral and spend a couple of minutes talking about that opportunity. Oncoral is a tablet formulation of irinotecan. Irinotecan is used today in clinical cancer -- care cancer as an injectable drug given every second or third week, very high dose, providing a lot of side effects initially because of the high concentration and the toxicity of the molecule, but it has important cancer treating effects. What we can do with Oncoral is that we can take this molecule, put it into a tablet, get it absorbed in a good way that we have demonstrated in the Phase I studies and using the daily exposure to treat the tumor in a different way and providing both potential for better efficacy and better safety. The data from Phase I showed that we could get the patient dosed and we get a good absorption of it. We also show that we can -- in that study, we reduced the side effect -- improved the side effect profile compared to the intravenous one based on what is out there in the literature. We also saw some patients who had previously been injected with -- had infusions with IV version, so the high dose every third week. Based on that treatment regimen, the tumor had grown. When we gave them these very sick patients, daily dosing, they had stable disease for a period of time, which in these very sick patients is an encouraging sign, not proof of efficacy, but an encouraging sign nevertheless. We think that data puts us on a good path for further development. We have a plan for Phase II in gastric cancer, where we have identified LONSURF as a drug that would probably work very well in combination. You can see some preclinical data to the left here showing the synergistic effect between the 2 compounds. We have a small collaboration with Thai Oncology for their support into the study. I think that's an interesting opportunity and a sign that daily dosing is indeed something that is interesting. As mentioned, we are initially looking at gastric cancer, but there are a number of other solid tumor indications where daily dosing of irinotecan could play an important role. It's not just a single indication. This is a multiple indication opportunity. With that, I'd like to transition to the financial part of our Q1 update. In Q1, our operating loss was SEK 17 million, sort of in the same range as the last few quarters and significantly less than last year prior to finalizing the NDA submission. At the end of the quarter, we had Sek 34 million in cash. Then Hansson as mentioned earlier, one of the highlights after the close of the quarter, we raised SEK 20 million before cost, which will extend our financial runway into next year. Putting us in a good negotiation position versus the potential partnership discussion or the partnership discussions and not having sort of a short-term squeeze on that. I'd like to round up before moving into the questions, talking about our key strategic objective. We set them up to say we have 2 things we want to achieve. We want to get Orviglance approved and we want to have a partnership in place. On the approval, we've met the milestones on the submission and data before letter. We are on track for getting an approval in July. Very excited about that. On the partnering side, we continue to work with the clinical community, our key opinion leader networks and other activities as well as the product supply to ensure that the product is available to be sold once we are at the launch date. The other part, which is obviously a major milestone as well is the partnership. We want to establish a partnership, so a company to drive the commercialization at least in the U.S., but could be additional geographical areas as well. Those conversations are progressing nicely. They are doing diligence on the asset, and we are negotiating and we are having, you would say, discussions around that. Again, we think that's a very interesting and optimistic outlook on the partnership opportunity as well. With that, I'd like to open up for questions.

Yes. Let's jump into it, Magnus. Could you discuss the trade-off between maximizing deal terms of Orviglance and the timing required to defend your orphan drug status? That's one.

Yes. As mentioned, we have the -- and I think the question refers to the orphan exclusivity where you have 7 years, you can extend it to 7.5 if you have pediatric addition to that. I think a very important, you could say, question. We think this is a multistep opportunity and really a franchise where we can have oral manganese a franchise that we can hopefully dominate for a long time to come. The market exclusivity from the orphan designation is important, and that gives us a good start. We think the additional IP coverage we have on the manufacturing and the food effect provides us with some good protection. I think we've done some very good -- some good IP applications there. Then we have the next-generation opportunity that I think would also potentially differentiate that product from the current version. With our knowledge and expertise in this area, we could hopefully extend the exclusivity period significantly beyond the 7 years.

Then there's a question that you haven't received a deal, should we take that as a sign that you're holding out on the negotiation that you want a better deal that reflects the long-term potential of the assets. Has that been a part of the considerations why you haven't broke a deal yet?

No, I think it's always a lot of conversations. I think different partners have different objectives, right? They have different risk profiles. They have different things happen. The companies we speak to are mostly significantly larger than Ascelia because they have commercial infrastructure. I think for each of the companies we speak to, there are different time line and priorities. Our objective is to make Orviglance available to patients and build, you would say, a strong business around that. I think we're doing well. I think we have some very interesting conversations ongoing and processes. I think it's a little bit of mix in terms of, you would say, where we are and how we get a strong value. I would say it's not like we have 10 signed final negotiations, and we're just kind of waiting and trying to do something. We have some very good conversations that have matured quite far. Some are very far, some are catching up. Our objective is to make sure we get a very good deal. It's not like we want to say, okay, but we want to have final agreements negotiated with everybody and then we decide. I think it's kind of a sort of a balanced approach. I think securing a strong partner who is committed to invest in the launch and who has -- where it's a good strategic fit and a good collaboration because it's not just a one-off, it's a partnership. We want to work well with the partner also to explore next-generation opportunities. All of these building the value, not just in the first couple of quarters, that's important, but the most important is building the long-term value. That's part of the process. I think we're having some really good conversations with various companies.

There's a question here. Can you come up the last 6 months concrete steps? Have they been through the technical due diligence, have there been due diligence? Have you had the data room? Has there been some -- can you elaborate a little bit on the concrete steps that you have been done in this process with different partners?

Yes. I think just in the interest of -- I understand the interest from the investor community because this is a major milestone that's going to drive a lot of value. I'm also aware that investors are listening to this, but our potential partners, the companies we speak to are also listening to this. I think in the interest of preserving the value for Ascelia, I think that's just a limit on how much I can say or want to say at this -- we are fully lined up for the process, right? We have the full setup processes, information available, everything is lined up. It's not like we're not ready for doing that. It's about managing the process, making sure that we bring the companies along and that we are aligned because it's important to get the deal, but it's also important that we are aligned on what needs to happen with the drug and how to move forward to the patients. Any potential issues we want to resolve before signing the deal rather than after because if we have a partnership and the partnership does not work, then that's going to be extremely problematic. We want to make sure that we have a good alignment with the partner, and they are comfortable with the information and the opportunity. We get good feedback on the opportunity. I think yes, I think we're doing a good job. I think the timing, we'll see when that happens.

Actually, you said keeping the partner in the loop. Have you shared your discussions with the FDA with a potential partner so they could get some kind of -- they also get an insight into the process up to the approval.

Yes. The parties that are furthest along have a good understanding of -- a detailed understanding of where we are with the FDA for sure, because that's a prerequisite for them to sign a deal. We do this as any other proven company would do is that you could say you enter the dialogue based on the assumption that everything is fine because we don't want to give them all companies access to our conversations and detail with the FDA. It's kind of a staged approach because we also want to make sure that we have this with we end up with at least one partner. The more information we share with the rest of the universe of companies, the more information we have is available for potential competitors, and we don't want to have that too much. If they are seriously interested and demonstrated that they're seriously interested, they have access to information about where we are with the FDA.

Then regarding the FDA, have you had any questions from them that surprised you or you might say needed extra work for you to kind of address? Or has it been like you expected the question. That's kind of an indication on the process, how well that is going.

Yes. I'd say there are no surprises in terms of the questions. I think they are across the different -- various different topics that you -- that we would expect. From our side, we think the FDA is doing a prudent and diligent job in terms of stress testing the application and to validate that this provides a benefit for patients with a good risk profile. I think that our experience is they do a good job. They ask questions that we think are very relevant, and I'm very happy with also our responses to those questions. No surprises. That's really nice.

Yes. Let's end the partner discussion. Firstly, what should we take out of the -- you're taking in capital, which could have been with a partner from maybe some upfront payment? Secondly, the direct answer, what is your belief before or after the approval date?

I work in scenarios. I don't have like kind of a belief of saying this date or the other one, right? I think there is a scenario where it happens before the signing because obviously, you can get an edge on the other -- if you're a company looking to partner here, you can get an edge by signing prior to approval. Then you structure that obviously, there is a nice milestone at approval. I can also see dynamics where companies say we -- for internal reasons and we'd hate to sign term sheet or a final agreement announced it 3 days before the PDUFA date and the FDA says for highly unexpected, but basically it comes down, right? Then you look like a full and nobody likes to look like a full. I think there are different scenarios, and depending on the partner, there could be different, you would say, risk profiles. We work according to each and every dialogue in terms of how do we maximize the -- get the best possible process. Then obviously, our job is also to evaluate the different partners to see which ones are most attractive in terms of capabilities, motivation and deal terms. I think that's how we do it. We don't have sort of a set kind of is it one day or the other. It's more about managing different scenarios, and we're prepared for it all.

That's the financial, I guess, in that sense, the last sentence you said we should look at the capital raise. Is that correct?

Yes, exactly. I think if we are in the scenario where the deal is happening after the approval, then we said we had financial runway into -- going into early Q4. Negotiations take a long time, including layer time, and that's also important to get the right agreements, and they are not -- that's fairly expensive. That's quite expensive. We want to progress the dialogue with as many partners as possible in parallel. I think that's just to ensure that there are no kind of runway issues or having to change our financial runway guidance in the middle of negotiations, I think, is also not helpful for Ascelia and our situation. I think that was a really good opportunity, I think, a small dilution, but giving us a much stronger hand in the partnering negotiations to finalize that part of the process.

Yes. Then that's a little bit about the competitive situation. yourself, GEs, manganese via low-dose approach. Do they limit some of your possibilities to get to larger players? Do you need the large incumbents in this space? Or do you need to go for smaller players, maybe this -- is there anything -- have you seen any reactions by the partners by this communication from GE moving manganese and via the lower dose? Do they see a limited market or does it matter anything to whether you can speak to the large complement in this space? Or do you need to look more down to maybe more smaller partners with more special sales forces?

Overall, I'm very positive on this development. I mean if we go back, let's say, 5 years, the conversation with getting manufacturers was mostly kind of this is not a big problem with real disease. There's a back warning, but it's not so many patients. It's kind of a nonissue, more or less. What we experienced today is that all of them put into their communication to the medical community that gadolinium has side effects. There are concerns about gadolinium. Firstly, obviously, the Black Box warning in renal disease patients, which are the ones we address, but also the brain deposition, the environmental issues. I think it's great for Orviglance that we have the large gadolinium manufacturers educating the medical community that using less gadolinium is a good thing. That makes our job of bringing out Orviglance as a non-gadolinium agent, bringing that out into the medical community, it's much easier because it's highly consistent with what everybody else is saying. When GE is investing a lot of money into an injectable manganese version, they show that manganese can provide -- they show that they have confidence in manganese providing strong images, and it's good from a safety and efficacy point of view. Again, highly supportive. As I said, their agent is an injectable version. As we know, there is no liver-specific elements of that. These products would be complementary. In theory, you could almost transition with that, and there are some other in early developments based on injectable manganese. You could see a transition so that gadolinium is no longer needed in the future. We could get rid of the environmental issues and the patient safety issues. I think that's not where we see it in the cards. I think that's certainly not needed for glance. I think what we come in with product with manganese in the high-risk patient for liver imaging. This is a very strong value proposition. The value proposition is supported by what the large gadolinium manufacturers are communicating.

There's a little bit about the FDA and the label. Have you had any discussion with them? If you had any discussion with them, I can see here on the slide, you haven't changed your market estimates, but I guess the label will have some effect on the market size or could have. A little bit -- have you discussed the label with the FDA? Does it give you any consideration about talking about the current market size?

I'm not going to disclose exactly what we have discussed with the FDA, yes and no. I can say we are very optimistic on the outlook. The label we intend -- that we filed for is based on the orphan drug designation, which is used in patients where you -- where gadolinium may be medically adviceable, which is obviously the renal impairment, so renal disease. That also would include segments such as severe allergies to gadolinium. Based on that, we continue to believe that is an appropriate label for the product, and that's covered by our orphan drug designation. I think that's where we stay at that. You never know what the final label is until you get the approval. That needs to wait until July 3, where we hopefully have the approval. Based on where we are, we continue with that base case.

Then I will leave you with the last question, yes or no, and I know there is no yes and no in this business. There is someone highlighting all the potentials if you do this yourself, if a partner doesn't appreciate and can give you the value. There's a very direct question. Could you, in some circumstances, consider doing this without a partner, commercializing this yourself if the partner would not -- if you can't find a partner that will give you the value you think this product has. I know it's not a yes and no, but let's end with that one, Magnus.

It's obviously a feasible strategy solution. We had that strategy a couple of years ago. Then we changed our strategy and said we want to work with a partner. I would say, based on the conversations we have with the potential partners, we focus on getting the partnership in place.

Yes. I think that was the last question. Thank you, Magnus, for taking us through your results, you can say, the investment case or your product category and of course, answering questions, and I know what must be a difficult situation, balancing investor community wanting to know everything and probably keeping your priority rights and your negotiating power with partners. I think, yes, we at least tried it here. Thank you, Magnus, and thank you for the audience listening in.

Yes. Thank you, everyone, for joining. Happy to keep you updated on our progress.