Ascelia Pharma AB (publ)

Welcome everyone to the webcast for Acelia Pharma's Q4 report for 2025. On this call, we will be making forward-looking statements. On today's call, we will start with an overview of Acelia Pharma, recent key events, and then head into the portfolio update before moving on to financials and priorities ahead. After the presentation, we will open up for questions as usual. I'm Magnus Corfertsen, CEO of Acelia Pharma. And with me today, I have Julie Varas Brogren, Deputy CEO, Andreas Norlin, Chief Scientific Officer, and Anton Hansson, CFO. I'm pleased to welcome Anton to his first quarterly call as Chief Financial Officer. This will also be the last quarterly call with Julie, as she will be moving on to new opportunities, as we announced a few weeks ago. At Acelia Pharma, we identify, develop, and commercialize novel drugs that address unmet medical needs across rare cancer conditions. We're based in Malmö, Sweden, and are listed on NASDAQ Stockholm. We have two drugs in our pipeline. Orviglans is a diagnostic drug that is now under FDA review with a Biduva date of July 3rd this year. We will spend the majority of this call talking about Orviglans. Oncoral is a patented tablet formulation of ibuprofen with encouraging results in phase one and potential to treat a range of solid tumors. We are very excited about OrbeGlance and here's why. OrbeGlance is addressing a well-defined unmet medical need for a subgroup of people living with cancer. This translates into an $800 million addressable global market opportunity, and OrbeGland is a first-in-class product to target this market and has orphan drug designation from the FDA. We have commercial scale manufacturing in place, and we have strong data from nine different clinical studies, including very compelling phase three data. As mentioned, the OrbeGland new drug application has been submitted to the FDA, and the review process is ongoing. The key milestone in the fourth quarter of 2025 was the FDA acceptance of the Overglans NDA in mid-November, the day 74 letter. This means that the FDA has reviewed the NDA file and decided it has the quality that warrants a full review. The FDA has also set a PDUFA date of July 3rd this year. This is the date when they expect to have made a decision on approval. In Q4, we submitted a new patent application for manufacturing of oviglands, which may extend the exclusivity period for oviglands. In November, we announced the appointment of Anton Hansson as our new chief financial officer, and he started a few weeks ago. Anton has a background in corporate finance and most recently worked with KPMG. I'm very happy to have him join our team. The long-term incentive program of 2022 vested and 53,000 shares were awarded in November to employees who had purchased and held Acelia shares throughout the three-year period. Also in Q4, the nomination committee was appointed by the largest shareholders in Acelia. After the close of the quarter earlier this, in January, we announced that Julie will be leaving Acelia Pharma and I would like to thank her for her contributions to Acelia throughout the years and wish her best of luck in her new role. There are two major value-creating milestones for AuricLens, and we are on track to meet both of them. The first objective is the FDA approval of AuricLens. And the key milestones of this is the submission we made in September last year. In mid-November, the FDA set a PDUFA date, which is July 3rd this year. And this is, as mentioned, the date when they expect to make a decision on approval. Our activities for the NDA review, including the interactions with the FDA, are progressing well, and we continue to be on track for approval. The second objective is to progress over glance for commercialization. This is a two-stringed approach. One is to continue our work to advance launch readiness by ensuring manufacturing and supply chain is as ready for launch as possible, as well as working with medical experts, including key opinion leaders, payers, and other key stakeholders. The other track that we are also working on is to make a deal with a partner who will drive the commercialization. Julie will follow up on our progress there. I'm very happy with the progress we are making on these important milestones, and I'm optimistic for a very strong 2026 for Celia. We will start the portfolio section of our presentation with OrbiGlance, and I'll hand over to Julie.

Thank you, Manus. OverGlance is a first-in-class liver MRI contrast agent, which addresses an unmet medical need for cancer patients for whom there are no good alternatives available today. Liver metastases are common in many cancer types, and adequate visualization of liver tumors and metastases is critical for making timely and appropriate treatment decisions. Orviglans based on manganese aims to be the preferred liver magnetic resonance imaging contrast agent for patients with severe kidney impairment. These patients have the highest risk of developing the serious and potentially fatal condition nephrogenic systemic fibrosis after exposure to the gadolinium-based contrast agents normally used today. The addressable market for overglans has a global value of $800 million annually. The U.S. represents almost half of this. This market opportunity for overglans addresses the unmet need for a well-defined patient population, cancer patients who need imaging of their liver and who also have severely impaired kidney function. Our strategy for commercialization is to launch through partners. This supports our ambition to secure the optimal balance between future revenues and investment required. Our focused, ambitious launch strategy and plans are built on advanced market insights and are already in place to support this partnering strategy and launch. As mentioned, the US is the largest commercial opportunity for Overglance. In the US alone, our real world data, i.e. data from realized procedures in our target patient population, show that every year 100,000 abdominal imaging procedures are performed in around 50,000 patients that fall under the black box warning for gadolinium contrast agents, i.e. our target patient population. This is about 4% of people with cancer undergoing abdominal imaging. The well-defined patient population with a clear unmet need also drives an attractive pricing opportunity. And we have extensive input from market access and pricing experts with whom we have tested different pricing levels and collected insights on the evidence needed to support access and reimbursement. And we have investigated pricing and access benchmarks of other innovative diagnostic drugs in the U.S. And 90% of healthcare professionals are concerned with issues related to gadolinium contrast agents, including the severe side effect associated with our target patient population, NSF. In fact, 16% of providers have experienced cases of NSF in patients exposed to gadolinium. When speaking with experts, whether in radiology or nephrology, they confirmed that an alternative to gadolinium for our target patient population would address concerns of today with the potential to become a valuable addition to their clinical practice. And the momentum for options for patients without gadolinium is strong. Beyond the risk of NSF in kidney impaired patients, gadolinium is well known to be retained in the brain and other tissue in all patients. And scrutiny over the possible safety effects is a key concern of regulators and medical bodies. It's also well known that gadolinium is excreted by other kidneys and urine. And because it's difficult to remove in our sewage system, it's discharged into the environment and into our drinking water. There's an urgency from regulators and medical bodies to find a viable alternative to the growing use of toxic gadolinium. An alternative that is not associated with these potential safety and environmental concerns for patients and for the environment with gadolinium. and the industry is responding, overglans will be the first in class liver MRI contrast agent for a future with less gadolinium and improved outcomes for patients. Recent developments from other large gadolinium manufacturers are focused on an early stage injectable manganese-based contrast agent, which is not liver specific like overglans, or focused on smaller doses of gadolinium. And we are excited that we have a head start with the upcoming approval and launch of Overglance. The go-to-market strategy for Overglance is to launch with commercialization partners. This strategy supports our objective to maximize the value of Overglance. OverGlance is an attractive partnering opportunity. With clinical development completed, we offer a de-risked asset, which is on track for FDA approval this summer. There's a clear unmet need for a focused launch with a well-defined patient population. So there are synergies for many types of commercial stage companies working with hospital decision makers. Partnering dialogues progress with additional momentum and clarity now with the regulatory timeline firmly established. In summary, we're excited about the opportunity to bring OverGlance to approval and to market with a partner. A market representing 800 million US dollars annual in addressable market for an unmet need of a very vulnerable patient population. We've come a long way with the successful completion of clinical development and our data supporting efficiency and safety of over glands received in the medical community. I will hand it over to Andreas to talk about the NDA's timeline and the review process with the FDA.

Thank you very much, Julie. I would like to start with a few highlights from the development program of Oroglance. So we have a quite extensive clinical development program, including a total of 286 patients and healthy volunteers across nine clinical studies. And 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 3 study SPARQL demonstrated that visualization of focal liver lesions measured by the co-primary efficacy variables border delineation and lesion contrast clearly improved with orboglans 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 orboglans than without. So in summary, with this strong data package supporting the use of water glands in a patient population with a unique and well-defined medical need, the NDA was, as we have heard before, successfully submitted and accepted for filing by the FDA last year. And now we are very excited that the FDA is proceeding with a review as planned according to their internal schedule and with the target of an approval on 3rd of July this year. So while still prioritizing our resources for successful approval and partnering for OrbeGlass, we also have Oncoral in our portfolio. Oncoral is a tablet formulation of Irinotecan, a well-established intravenous chemotherapeutic agent. A tablet formulation enables a frequent daily and low dose dosing regimen that could offer potential advantages on both efficacy and safety compared to the infrequent high dose intravenous administration 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 Irinotecan 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 regimen. This supports the continued development of OncOral as a daily oral formulation of irinotecan. Our plan is to bring OncOral into clinical phase 2 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 our strategy is to start Oncoral development in gastric cancer. which is today a $3 billion market. And Irinotecan is a well-established chemotherapy with recognized anti-tumor effect in many types of solid tumors. And therefore, we see opportunities for developing Oncoral in other indications as well, where daily dosing could positively impact the efficacy and safety outcomes for the patients. And we are assessing these opportunities as part of our ongoing strategic planning of Oncoral. So with that, let's move on to the update on the financials, and I hand it over to Anton.

Thank you, Andreas. I will now move to the update on our financials and priorities ahead. In Q4, our operating results amounted to a loss, i.e. costs of 16 million kroner. The costs are lower in Q4 compared to the costs per quarter in the first half of the year, as well as year over year. And that is due to the completion of the NDA. At the end of December, we had 15 million kronos in liquid assets. In September, we successfully completed a directed share issue, raising 30 million SEK before costs. And we now have a cash runway into Q4 2026, well beyond the expected FDA approval date of order dates. And to wrap up, we have substantial value creation opportunities ahead, both for Orviglands and Acelia Pharma. With Orviglands, we are bringing a first-in-class diagnostic drug to market, addressing an $800 million market for patients with a high unmet need. We have two key objectives. One is timely approval of Orviglands. And here we submitted the NDA early September. In mid-November, FDA accepted Orviglands NDA for review, and with a standard 10-month review, we can expect approval early July 2026. And our second objective is to progress Orviglands for commercialization for patients in need entering into a partnering agreement for the launch, and these efforts continuing to progress. We look forward to continuing our journey with opportunities ahead for growing Acilia Pharma in 2026 and beyond.

That was our Q4 presentation. Now we would like to open up for questions.

If you wish to ask a question, please dial pound key five on your telephone keypad to enter the queue. If you wish to withdraw your question, please dial pound key six on your telephone keypad.

Hello, everyone. Thank you for taking questions. Maria here from D&D Carnegie. I was wondering, I have two questions for now. So we have five months-ish left for a PDUFA date. Do you have any updates for us on your discussion regarding pricing strategy and maybe payer engagement?

Julie, do you want to take that one?

yeah um we've over the years developed both uh research or insights from from payers and experts and you can say the the roadmap for a launch so we we are very fine in having a strategy ready um and we expect a partner to to to drive the execution of this um but we really have the the insights and and the strategies in place for a partner to to immediately ramp up on that. Does that answer your question? It includes testing the value messages and testing how to approach each partner in each setting and so forth. So it is, yes, an important area and we have done a lot of preparations.

Thank you. Thank you, Julie. And the next one is, so cash runway into the fourth quarter of 2026. But looking ahead, and if we're looking at it optimistically with a potential approval, how would it affect your cost base in the second half of the year? What do you see in front of you?

So we don't have a... you would say, revenue or cost guidance per se. So what we're saying is that with the current cash we have and the expected cost going forward, we will have cash into Q4. We're also very clear that we have a strategy to partner. And as we say, we are optimistic on the outcome of that process. And we think that's going to contribute with capital to Asilia that will change, you could say, the outlook.

All right, thank you. That's it for me for now. I'll get back in the queue. Thanks.

Yeah, thank you, Maria.

And this is your operator speaking. The next speaker is Fredrik Thor from Redeye.

Hello and thank you. Maybe a follow up to Maria. How will the distribution between R&D costs and administrative costs be for the rest of the year, roughly?

We don't guide specifically for sort of the split, but I think what you see is probably, with the current outlook, not going to change dramatically.

Okay, got it. And the next question was about, you mentioned or you released about a new patent application for Orbeglons. Can you elaborate a bit on this and overall the patent strategy for Orbeglons?

Yeah, so happy to talk on that. So we have orphan drug designation in the US, which means that the FDA will grant seven years of market exclusivity of all the glands, which gives us a very strong protection that we are very satisfied with. So what we also have is we have additional patent applications. I mean, it's well established that manganese enhances MRI signal. So that's not an IP space we can be in. But we have... patent application on the food effect and we have now filed an additional patent application related to the manufacturing and how to do that in a good way and control the process. We have some unique insights that we have filed in the patent that of course it's not published yet so we will not go into details but we think that was a very nice application that we put in place.

Got it. And maybe a final question. In your slide about addressable market, the rest of the world was quite a bit of the total market. What would you say are the key markets in the rest of the world beyond Europe, Japan, and the US?

I mean, some of the key markets are Asian markets. where it's a very mature healthcare system and the awareness about the use of gadolinium and the risks is very high. There are also other very mature markets in the Middle East and some in Latin America and so forth where there's a a willingness to pay for very vulnerable patients. So it's some of the sort of mature markets in Asia, Middle East, and Latin America primarily.

Got it. Thank you. That's all for me.

Thank you. As a reminder, if you wish to ask a question, please dial pound key five on your telephone keypad.

We also got some questions in the chat here and we will start. There's one in terms of pointing to our strategies to make an agreement with a partner. And there's a question in terms of what we could share on the timeline for that. I think the question is that we are very clearly dedicated to get a partnership agreement with OrbitGlance. As Julie mentioned, the day 74 letter and the due for date has certainly been helpful for those conversations. It's, you could say, a lengthy process. It always is, and especially when we are late stage in development with a lot of data for the partners to review. So I would say we will not say anything specific on the timing. We have a very optimistic outlook on getting a deal done. But we don't want to put ourselves in sort of a timeline squeeze where that may be exploited by a partner. We don't think that's in the interest of the company. So we're dedicated to getting a deal. We're very optimistic on the outlook. And things are progressing very well. So I think that's what we can share at this point with our investors. We understand the importance and we are fully aligned on that objective with our investors.

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. So another question that has come up is that we have mentioned that we are talking to multiple partners and whether we're still doing that. I think it's fair to say that we are speaking to multiple partners and entertaining those conversations and processes. And yeah, I guess we are optimistic that we will find a partner. Another question here is whether the European authorities accept an FDA approval or whether we need to do additional studies. So the structure of an application for approval in Europe is very similar to the US application. So the vast, vast majority of the NDA file can be reused for Europe, but there are some specific European requirements that would need to be adjusted in that submission. And obviously, at the end of the day, it would be the European Medicinal Agency that will make a decision whether any further studies are needed. That's a kind of end of review decision. But we don't expect any additional studies are needed for approval in Europe. Those were the questions. So thank you very much for joining our quarterly call. We look forward to updating you on our progress in 2026. Very excited about the outlook for this year. Thank you and have a good day.