Ascelia Pharma (Stockholm:ACE) Q1 2026 Earnings Call

0:00 Welcome everyone to the webcast for Ascelia 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 Ascelia 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 Ascelia Pharma, we identify, develop and commercialize novel drugs that address unmet medical needs within rare cancer conditions. Our company is based in Malmo, Sweden and we are listed on NASDAQ's Defy, develop and commercialize novel drugs that address unmet medical needs within rare cancer conditions. Our company is based in Malmo, Sweden and we are listed on Nasdaq Stockholm.

0:49 We have two drugs in our pipeline. OrbiGlambs is a diagnostic drug that is now under FDA review with a PDUFA date of 07/03/2026. We will spend the majority of this call talking about OrbiGlans. Oncrol is a patented tablet formulation of ibreenutrikin with encouraging results in phase one and potential to treat a range of solid tumors. On the progress, of the first quarter this year, we at the European Society for Gastrointestinal and Abdominal Radiology Conference also shortened ESCAR. There will be an oral presentation about Orbeglands by Professor Dominic Geisel from the Charity Hospital in Berlin. Doctor Geisel was an investigator in our phase three study and Charity is one of the largest hospitals in Europe.

1:37 During the first quarter our previously communicated changes in the management team took place as Anton Hansen started as our Chief Financial Officer and Julie Brogren left Ascelia. After the close of the quarter in April, we made a directed share issue and raised 20,000,000 Swedish kronor before cost, extends our cash runway into 2027. We are focused on our two key strategic goals. OrbiGlans approval and OrbiGlans 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 third last year and in mid November last year, the FDA set the set the PDUFA date of 07/03/2026.

2:23 This is the date, the PDUFA date is the date where the FDA expects to make a decision on the approval of the organ glands new drug application. 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 Orbiglans for commercialization. This is a two string 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 with 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.

3:11 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 Ascelia. We will start the portfolio section of our presentation with Orbitrans. We are very excited about Orbitrans and here is why. Orbitrans is addressing a well defined unmet medical need for subgroup of people living with cancer. This is an $800,000,000 addressable global market opportunity for and Orlevance 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 OrbitLance new drug application has been submitted to the FDA and the PDUFA date is coming up in less than two months.

4:01 OrbitLance is an attractive asset that can help patients and we will work with a partner to drive the commercialization. Let's talk about how OrbiLance 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 spreads to the liver. This accounts for roughly ninety percent of cancer in the liver in The US and Europe. The remaining ten percent 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.

4:57 The best way to do this is with liver MRI and to use a gadolinium based counterstages. 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 OrbitLance. OrbitLance provides high quality imaging and because it is based on manganese, are none of the gadolinium associated risks. This unmet medical need translates into a global addressable market opportunity of $800,000,000 on an annual basis.

5:43 The US market is almost half of this and therefore we have prioritized The US market. Ascelia will work with partners to drive the commercialization of organelles. Let's talk a bit bit more about the key metrics for The US market. Based on real world data, there are about 100,000 relevant procedures in the organ transplant population every year in The US. Because of their disease and comorbidities, these patients are mostly treated at the major medical centers and seventy five percent of these imaging procedures are made at the 400 largest medical accounts. Based on market research, pricing benchmark for high value diagnostic drugs can be at $3,000 to $4,500 per dose. Is addressing a well recognized medical need. As mentioned previously, OrigLance is targeting patients with severe renal disease because of the gadolinium associated risks.

6:39 The medical community is well aware of this and our market research demonstrates that more than ninety percent of healthcare professionals are concerned about gadolinium associated risk and a number of them have also experienced nephrogenic systemic fibrosis in patients. Orbitrans is targeting patients with severe renal impairment, which is the highest risk population defined by the black box warning. But there are also other concerns with gadolinium. Every gadolinium injection, there is also a deposition of gadolinium and 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.

7:26 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. Ascelia is leading the change with Orbitlance 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 twothree study.

8:11 We consider this a positive development with increased focus on manganese agents. And based on what we know, 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 Orbiglans, and we believe the timing for bringing Orbiglans 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 Orbiglans. Orbiglans 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.

8:57 Thank you Magnus. So before I talk about the NDA process, just a few words about the development program of oral glands. So we have an extensive clinical development program including a total of two eighty six 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 and with severe renal impairment. The phase three study SPARCL demonstrated that visualization of focal liver lesions measured by the co primary efficacy variables, border delineation and lesion contrast clearly improved with OrbeGlance compared to unenhanced imaging with a high degree of statistical significance.

9:49 We could also see that more lesions, especially smaller ones could be detected in the liver with OrbeGlance than without. So to summarize, we've built a strong and compelling data package supporting the use of OrbeGlance 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. As is then, the review has progressed as expected with a target date for FDA's final decision on July 3. Because OrbeGlass 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.

10:44 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 3. While our primary focus right now is on securing a successful approval and partnering for Overglance, we also have Oncrawl in our portfolio. That program remains an important asset and we are looking forward to reinitiation 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 frequent 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.

11:42 The opportunity of improving efficacy and safety by administration of daily low doses also called metronomic dosing has been demonstrated for arinotecan and other chemotherapeutic agents. In our phase one studies with Omporal, we've seen encouraging results that suggest that oral daily administration of areinotecan 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 Arinotecan. Our plan is to bring Oncoral into clinical phase two in gastric cancer.

12:29 Animal data has demonstrated a synergistic effect of Irinotecan when combined with Lonsurf, 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 long serve in gastric cancer. And we start there and this is today a $3,000,000,000 market. Arinotecan is a well established chemotherapy with recognized antitumor effects in many types of solid tumors. Therefore, we see opportunities for developing Oncrol in other indications too where daily dosing could positively impact efficacy and safety outcomes for the patients.

13:21 And we are assessing these opportunities as part of our ongoing strategic planning for Oncrol. So let's move on to an update of the financials. Anton? Thank you, Andreas. So I will now move on to an update of our financials. And in q one, our operating results amounted to a loss of 17,000,000 kronas, and the costs are lower in the quarter compared to q one twenty twenty five due to the completion of the NDA. At the end of the at the end of March, we had 34,000,000 in liquid assets. And as mentioned earlier, after the quarter ended, we successfully completed a directed share issue, right, raising 20,000,000 SEK before costs in April.

14:07 And we have now extended our cash runway into 2027. And to summarize, we have significant value creation opportunities ahead. With Orbiglance, we are bringing a first in class diagnostic drug to market, addressing an $800,000,000 market for for patients with high unmet need. And our two key objectives remain. The first, timely approval of Orbiglans with the optimal label. And here in early September, we submitted the NDA, and then FDA accepted the Orbiglans NDA for review in mid November. And based on a standard ten months review

14:44 process, we can expect an approval early July twenty twenty six. Our second objective is to progress Orbiglans 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 q one and are well positioned to deliver on our key milestones. And we look forward to continuing our journey with these opportunities for growing Ascelia Pharma. That concludes our, quarterly presentation. Now we'd like to open up for questions. If you wish to ask a question, please dial 5 on your telephone keypad The next question comes from Maria Carlson Ascepubl) from DNB Carnegie.

15:36 Please go ahead. Hello. Good morning, Ascelia team. I have two short questions. Thank you for the presentation and of course, obviously all eyes on 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 and it is a standard back and forth process with the agency. So basically my question is, has the nature of volume of the questions changed somehow as you're now entering the final stretch?

16:10 And if you could maybe characterize the types of areas that the agency is now focused on? Yeah, 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. FDA 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.

16:58 That's good to hear. And another short one. I mean if the FDA approves this on July 3, what happens on July 4? Or I mean, I think it's Friday. So what happens on July 6 then? Can you walk us through like the process? What are they up like the coming ninety 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 as I understand it, you know, we get we get an approval letter which will, you know, probably hang up. Yeah. So so so, you know, have a have a sort of a copy on on the wall to celebrate. I think that's obviously a major transformative milestone. There are some, you'd say, administrative things and, you know, for for setting everything in place.

17:46 But you could say the next step is is, you know, getting ready for launch and all the all the activities that that goes into that. And as

17:55 we've said, we want to work with a partner who will drive the commercialization. We have done a lot of work to prepare that. So we have, you know, some road maps and blueprints and and a little of market insights that is gonna help the partner get moving fast. So so I think that's we will work with a partner to make sure everything is lined up for for the launch.

18:22 Alright. Thank you. I don't have any more questions. Thank you very much. Yep. Thank you, Maria. The next question comes from Fredrik Thor from Redeye. Please go ahead. Hello, thank you. I have a follow-up question to Maria regarding the approval that in July. Are there any one off costs related to the approval process that will or not one off, but costs that will are related to the approval process that will that won't have any more after approval. So basically, will the cost development be in a specific way after the approval or will it be quite quite stable cost development after the approval, potential approval?

19:22 Yeah, well, thank you for the question. It's we're not providing sort of specific cost guidance. I think, obviously, responding to to questions and and requests is both, you could say, internal Ascelia team and and external experts. And and so obviously it's it's it's not for free to to be in this process. On the other hand, we also envision other activities after after the approval. So I think we'll at at this point, we're not sort of expecting

19:53 major significant changes in our cost structure. Got it. That's 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 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 is to to line up for for partnering. We have the kid we have, you know, 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 for approval.

20:40 Which I think, you know, I'm very proud of the team that that can, you know, meet these, you know, very, challenging milestones and I think, I think that's a great achievement, so far. I I think the that those achievements also generate value and create a a good dynamic in in the partnering conversations. The partners are, you would say, focused on on you preparing on what would their commercial team look like and and the commercial opportunity was required. And I would say significantly less on on the on the scientific part because the FDA is is basically doing the the the diligence on on that part. So I think that that is creating some some good dynamics whether we sign an agreement before the, you know, FDA decision or after. You know, it's it's hard to to tell.

21:29 Obviously, from a partner perspective, you can you have lower risk after the approval, but on the other hand, could kind of structure the deal in a way that where you take out some of the risk on the FDA decision. But I think it could be either or, 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 Ascelia Pharma and, we look forward to updating you on our progress as, as it, the year grows on.