Ascelia Pharma AB (publ)

Welcome everyone to the webcast for Acelia Pharma's Q1 report for 2026. On this call, we will be making forward looking statements. On today's call, we will start with an overview of Acelia Pharma and recent key events, and then we'll head into the portfolio update before moving to financials and priorities ahead. After the presentation, we will open up for questions. At Acelia Pharma, we identify, develop, and commercialize novel drugs that address unmet medical needs within rare cancer conditions. Our company is based in Malmö, Sweden, and we are listed on Nasdaq Stockholm. We have two drugs in our pipeline. Orbiglans is a diagnostic drug that is now under FDA review with a PDUFA date of 3rd of July, 2026. We will spend the majority of this call talking about Orbiglans. Oncrol is a patented tablet formulation of ibuprofen with encouraging results in phase 1 and potential to treat a range of solid tumors. On the progress of the first quarter this year, at the European Society for Gastrointestinal and Abdominal Radiology Conference, also shortened ESCAR, there will be an oral presentation about overglans by Professor Dominic Geisel from the Charité Hospital in Berlin. Dr. Geisel was an investigator in our Phase 3 study, and Charité is one of the largest hospitals in Europe. During the first quarter, our previously communicated changes in the management team took place as Anton Hansson started as our chief financial officer and Julie Brogan left Asilia. After the close of the quarter in April, we made a directed share issue and raised 20 million Swedish kronor before cost, which extends our cash runway into 2027. We're focused on our two key strategic goals, Orbeecland's approval and Orbeecland's partnering. Happy to say that we're on track on both of them. For the approval, the sub-milestones is that the submission of the NDA on September 3rd last year, and in mid-November last year, the FDA set the PDUFA date of 3rd of July, 2026. This is the date, the PDUFA date is the date where the FDA expects to make a decision on the approval of the Orbitlan's new drug applications. Our NDA activities, including the interactions with the FDA, are progressing well, and we continue to be on track for the approval. The second objective is to progress or relance for commercialization. This is a two-stringed approach. One is to continue to advance the launch readiness by ensuring manufacturing and supply chain is ready for launch, as well as working with medical experts and key opinion leaders, payers, and other key stakeholders. to ensure the product will have a successful launch whenever that happens for the partner. And that is the other track that is to enter into a deal with a partner who will lead and drive the commercialization. I'm proud of the team and the progress we are making on these important milestones, and I'm optimistic for a very strong 2026 for Acelia. We will start the portfolio section of our presentation with all the answers. We are very excited about OrbitGlance and here's why. OrbitGlance is addressing a well-defined unmet medical need for a subgroup of people living with cancer. This is an $800 million addressable global market opportunity and OrbitGlance is the first in class product to target this and has orphan drug designation from the FDA. We have commercial scale manufacturing in place and we have strong data from nine clinical studies, including compelling phase three data. As mentioned, the OrbeGlance new drug application has been submitted to the FDA, and the Pidufidate is coming up in less than two months. OrbeGlance is an attractive asset that can help patients, and we will work with a partner to drive the commercialization. Let's talk about how OrbeGlance can help patients. Cancer in the liver is a significant challenge in cancer care. Many different cancer types metastasize to the liver, meaning they start in one organ and spread to the liver. This accounts for roughly 90% of cancer in the liver in the US and Europe. The remaining 10% of cancer in the liver is when the cancer originates and starts in the liver. The liver is often the first organ where the cancer spreads to and also often the cause of death. That is why cancer in the liver is a major issue in cancer care. And therefore, it is important to perform high quality medical imaging to determine if the cancer has spread to the liver and how the cancer in the liver is responding to therapy or for surgical planning. The best way to do this is with liver MRI and to use a gadolinium-based contrast agent. For most patients, this works well. However, for patients with severe renal disease, they are at risk of a potentially fatal gadolinium-induced side effect known as nephrogenic systemic fibrosis. Regulatory agencies have requested black box warning on gadolinium products because of this risk. It is exactly these patients that we aim to help with OrbeGlance. OrbeGlance provides high quality imaging, and because it is based on manganese, there are none of the gadolinium associated risks. This unmet medical need translates into a global addressable market opportunity of $800 million on an annual basis. The US market is almost half of this, and therefore we have prioritized the US market. Celia will work with partners to drive the commercialization of OrbeGlance. Let's talk a bit more about the key metrics for the U.S. market. Based on real-world data, there are about 100,000 relevant procedures in the over-the-lens target population every year in the U.S. Because of their disease and comorbidities, these patients are mostly treated at the major medical centers, and 75% of these imaging procedures are made at the 400 largest medical accounts. Based on market research, the pricing benchmark for high-value diagnostic drugs can be at $3,000 to $4,500 per dose. OrbicLens is addressing a well-recognized medical need. As mentioned previously, OrbicLens is targeting patients with severe renal disease because of the gadolinium-associated risks. The medical community is well aware of this, and our market research demonstrates that more than 90% of healthcare professionals are concerned about gadolinium-associated risk, and a number of them have also experienced nephrogenic systemic fibrosis in patients. OrderGlance is targeting patients with severe renal impairment, which is the highest risk population defined by the Black Box 1. But there are also other concerns with gadolinium. Every gadolinium injection, there is also a deposition of gadolinium in tissue in the body, including in the brain. The consequences of this continues to be evaluated in the scientific community, including long-term effects of the deposition. Another issue is the gadolinium accumulation in our water supply. After the gadolinium contrast injection, the gadolinium is excreted in the urine, which goes into our sewer system. QN filtration systems are unable to remove the gadolinium, and as a consequence, gadolinium content in drinking water has increased over the last decades. These are important drivers for the future of MRI. Acelia is leading the change with OrbitLens being based on manganese and under FDA review. If and when approved, it will be the only manganese product on the market. The gadolinium manufacturers recognize the gadolinium challenges. GE Healthcare is developing an injectable manganese agent. They have completed a phase one study and has recently started a phase two slash three study. We consider this a positive development with increased focus on manganese agents. And based on what we know, these two products are complementary to each other. The other gadolinium manufacturers have developed gadolinium products with lower doses of gadolinium to reduce the impact. These underlying developments support the need for a product such as OrbeGlance, and we believe the timing for bringing OrbeGlance to the market is good. We have a clearly stated strategic goal of entering into a partnership with a company that will lead the commercialization of OrbeGlance. OrbeGlance represents an attractive commercial opportunity, and we are making good progress in our partnering dialogues. Now we'll transition to the scientific and regulatory section, and Andreas, please go ahead. Thank you, Magnus.

So before I talk about the NDA process, just a few words about the development program of Oroglands. So we have an extensive clinical development program, including a total of 286 patients and healthy volunteers across nine clinical studies. Together, these studies have shown a strong, consistent, positive benefit-risk profile, supporting the intended use in patients in need of a liver MRI or severe renal impairment. The phase three study, SPARQL, demonstrated that visualization of focal liver lesions measured by the co-primary efficacy variables border delineation and lesion contrast clearly improved with orbiglans compared to unenhanced imaging with a high degree of statistical significance. We could also see that more lesions, especially smaller ones, could be detected in the liver with orbiglans than without. So to summarize, we've built a strong and compelling data package supporting the use of Orviglance in a patient population with a clearly defined and significant unmet medical need. Based on that, we successfully submitted our NDA in September last year. And since then, the review has progressed as expected with a target date for FDA's final decision on 3rd of July. Because Orviglance has orphan drug designation for its intended population, we expect to benefit from seven years of regulatory exclusivity following approval. In addition, we filed new patent applications that could extend protection well into the 2040s, further enhancing the long-term commercial opportunity for the product. Overall, we're really pleased with where we are and are very much looking forward to the final stretch leading up to FDA's expected final decision on July 3rd. While our primary focus right now is on securing a successful approval and partnering for OverGlance, we also have Oncoral in our portfolio. That program remains an important asset and we are looking forward to re-initiation when the timing is right. I'd like to say a few words about that opportunity as well. Oncoral is a tablet formulation of Arenotecan, a well-established intravenous chemotherapeutic agent. A tablet formulation enables a frequently daily low-dose dosing regimen that could offer potential advantages on both efficacy and safety compared to the infrequent high-dose intravenous administration that is used today. The opportunity of improving efficacy and safety by administration of daily low doses, also called metronomic dosing, has been demonstrated for Irinotecan and other chemotherapeutic agents. In our phase one studies with Oncoral, we've seen encouraging results that suggest that oral daily administration of arenotecan could offer meaningful benefits to patients. Importantly, the Oncoral studies have demonstrated an acceptable safety profile and that the uptake of the drug after oral administration is well suited for a metronomic dosing approach. This supports the continued development of Oncoral as a daily oral formulation of arenotecan. Our plan is to bring Oncoral into clinical phase two in gastric cancer. Animal data has demonstrated a synergistic effect of Irinotecan when combined with Lonesurf, another oral cancer treatment already approved for gastric cancer, which makes this all oral combination very interesting. The plan is to demonstrate clinical proof of concept for the combination of Oncoral and Lonesurf in gastric cancer. And we start there, and this is today a $3 billion market. Arenotecan is a well-established chemotherapy with recognized antitumor effects in many types of solid tumors. Therefore, we see opportunities for developing on-crawl in other indications too, where daily dosing could positively impact efficacy and safety outcomes for the patient. And we are assessing these opportunities as part of our ongoing strategic planning for on-crawl. So let's move on to an update of the financial. Anton?

Thank you, Andreas. So I will now move on to an update of our financials. And in Q1, our operating results amounted to a loss of 17 million kroners and the costs are lower in the quarter compared to Q1 2025 due to the completion of the NDA. At the end of March, we had 34 million in liquid assets. And as Magnus mentioned earlier, after the quarter ended, we successfully completed a directed share issue, raising 20 million SEK before costs in April. And we have now extended our cash runway into 2027. And to summarize, we have significant value creation opportunities ahead. With OrviGlands, we are bringing a first-in-class diagnostic drug to market, addressing an $800 million market for patients with high unmet need. And our two key objectives remain. The first timely approval of Orviglans with the optimal label. And here in early September, we submitted the NDA. And then FDA accepted the Orviglans NDA for review in mid-November. And based upon a standard 10-month review process, we can expect an approval early July 2026. Our second objective is to progress Orviglans for commercializations for patients in need by entering into a partnering agreement for the launch. These efforts continue to advance. So overall, we continue to progress in Q1 and are well positioned to deliver on our key milestones. And we look forward to continuing our journey with these opportunities for growing Acilia Pharma.

That concludes our quarterly presentation. Now we'd like to open up for questions.

If you wish to ask a question, please dial pound key 5 on your telephone keypad. To enter the queue, if you wish to withdraw your question, please dial pound key 6 on your telephone keypad. The next question comes from Maria Karlsson-Osipova from DNB Carnegie. Please go ahead.

Maria Karlsson- Hello. Good morning, affiliate team. I have two short questions. Thank you for the presentation and of course obviously all eyes on the PDUFA date. And in the report you state that you're working actively to answer all of the FDA supplementary questions. And I mean in previous calls and reports you have described this as a standard back and forth process with the agency. So basically my question is has the nature or volume of the questions changed somehow as you're now entering the final stretch? And if you could maybe characterize the types of areas that the agency is now focused on?

Thank you for that question. I think the simple answer is that we have received questions across the board. And that is, of course, what you expect if they are reviewing all sections, efficacy, safety, CMC, non-clinical. And we have received questions all over. At the end of the review period you enter the negotiation or discussion about the label. And that is what we expect also here at the end. But everything has been flowing nicely so far.

That's good to hear. And another short one. If the FDA approves this on July 3rd, what happens on July 4th? I mean, I think it's Friday, so what happens on July 6th then? Can you walk us through the process? What are the coming length of your days or so? What's already in place and what still depends on the potential partner maybe?

Yeah, so you could say, at least as I understand it, we get an approval letter, which we'll probably hang up on. Yeah, so have a copy on the wall to celebrate. I think that's obviously a major transformative milestone. There are some, you could say, administrative things for setting everything in place. But you could say the next step is getting ready for launch and all the activities that goes into that. And as we've said, we want to work with a partner who will drive the commercializations. We have done a lot of work to prepare that. So we have some roadmaps and blueprints and a lot of market insights that is going to help the partner get moving fast. So I think we will work with the partner to make sure everything is lined up for the launch.

All right. Thank you. I don't have any more questions. Thank you very much.

Thank you, Maria.

As a reminder, if you wish to ask a question, please dial pound key five on your telephone keypad. The next question comes from Frederick Thor from Red Eye. Please go ahead.

Hello. Thank you. I have a follow-up question to Maria regarding the approval date in July. Are there any one of costs related to the approval process that will or one of not one of but costs that will are related to the approval process that will yeah but you won't have any more often approval so basically will the cost development be in a specific way after the approval or will it be quite yeah quite stable cost development after the approval potential approval

Yeah, well, thank you for the question. We're not providing sort of specific cost guidance. I think obviously responding to questions and requests is both, you could say, internal assailant team and external experts. And so obviously it's not for free to be in this process. On the other hand, we also envision other activities after the approval. So I think at this point, we're not sort of expecting major significant changes in our cost structure.

Got it. That's my question. Thank you very much.

There are no more phone questions at this time, so I hand the conference back to the speakers for any written questions and closing comments.

Yeah. There's a question here on whether it's a partnering strategy to complete the negotiations after positive PDUFA feedback. So I think the overall strategy that we have is to line up for partnering. We have completed the clinical development. We have finalized the NDA and we're now running the FDA review process. And as we said, we are on track for approval. which I think, you know, I'm very proud of the team that can, you know, meet these, you know, very challenging milestones. And I think that's a great achievement so far. I think that Those achievements also generate value and create a good dynamic in the partnering conversations. The partners are, you would say, focused on preparing of what would their commercial team look like and the commercial opportunity, what's required. And I would say significantly less on the scientific part. because the FDA is basically doing the diligence on that part. So I think that is creating some good dynamics, whether we sign an agreement before the FDA decision or after, you know, it's hard to tell. Obviously, from a partner perspective, you have lower risk after the approval, but on the other hand, you could kind of structure the deal in a way that where you take out some of the risk on the... on the FDA decision. But I think it could be either or, you know, time will tell where we end up. There are no more questions. So thank you all for joining our Q1 call. And, you know, we are, as mentioned, on track for FDA approval and on our partnering process. So a very exciting 2026 for Acilia Pharma. And we look forward to updating you on our progress as the year grows on.