Welcome to Ascelia Pharma Q3 2025 report presentation. For the first part of the conference, participants will be in listen-only mode. During the Q&A session, participants are able to ask questions by dialing pound key five on their telephone keypad. Now I will hand the conference over to CEO Magnus Corfitzen, CSO Andreas Norlin, and Deputy CEO Julie Waras Brogren. Please go ahead.
Hi everyone, and welcome to the webcast for Ascelia Pharma Q3 financial report 2025. On this call, we will be making forward-looking statements. On today's call, we will start with recent key events and then head into our portfolio update before moving to financials and priorities ahead. After the presentation, we will open up for questions as usual. At Ascelia Pharma, we identify, develop, and commercialize novel drugs that address unmet medical needs within rare cancer conditions. We're based in Malmö, Sweden, and are listed on Nasdaq Stockholm. We have two drugs in our pipeline. OrbiGlans is in the registration phase as we have successfully completed the pivotal Phase III clinical study SPIRAL and have submitted the new drug application to the FDA. The FDA is currently reviewing the application. OrbiGlans has orphan drug designation from the FDA and is targeting an addressable market opportunity of $800 million.
Our other asset, OncRol, is a patented tablet formulation of irinotecan with encouraging results in Phase I and potential to treat a range of solid tumors. We have a clinical collaboration with Tidal Oncology and are initially targeting treatment of gastric cancer. The key milestone in the third quarter was our submission of the OrbiGlans NDA to the FDA, which took place in early September. Also in September, Fenja Capital converted their SEK 7.5 million convertible, and at the end of September, we completed a directed share issue of SEK 30 million before cost based on inbound investor interest. With these financing events, we now have a clean balance sheet and a stronger cash balance. On Monday this week, we announced the appointment of Anton Hansson as our new Chief Financial Officer. Anton has a background in corporate finance and most recently worked with KPMG.
In conjunction with the appointment of Anton, we made some changes to the management team to prepare our organization for the next phase of growth for Ascelia Pharma. We are focused on some key value creation opportunities, in particular with OrbiGlans. The first objective is a timely submission and approval of OrbiGlans with the optimal label. The key milestones to reach this objective are, first, the submission of the OrbiGlans NDA, which was done on the 3rd of September. This means we expect to receive a PDUFA date from the FDA, and this is likely to be shared with us in the middle of November. The approval of the OrbiGlans NDA is expected after a standard 10-month review by the FDA. As we submitted in early September, that would imply a timeline around early July. In parallel, the second objective is to progress OrbiGlans for commercialization.
The key activities are to continue to advance our launch readiness by ensuring manufacturing and supply chains are ready for launch, as well as working with medical experts and key opinion leaders, payers, patient advocacy groups, and other key stakeholders. Another important part of that is entering into a commercialization partnership. I'm very happy with the progress we're making in Ascelia Pharma and the efforts made by our team to ensure we will meet our objectives and create value for our shareholders. Now we will move into the portfolio section of our presentation, and we'll start with OrbiGlans. We are very excited about OrbiGlans, and here's why. OrbiGlans is addressing a well-defined unmet medical need for a subgroup of people living with cancer. This is a $800 million global market opportunity, and OrbiGlans is a first-in-class product to target this. It has an orphan drug designation from the FDA.
We have commercial-scale manufacturing in place, and we have strong data from nine different clinical studies, including compelling Phase III data. As mentioned, the OrbiGlans new drug application has been submitted to the FDA, and the review is ongoing. Now we'll go further into the OrbiGlans opportunity, and I'd like to hand the word over to Julie. Please go ahead.
Yeah, thank you, Magnus. OrbiGlans is a first-in-class MRI contrast agent. It addresses an unmet 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 and for following up on the effect of treatment. Contrast-enhanced MRI is the gold standard procedure for examination of patients with suspected unknown tumors or metastases. The most used contrast agents are based on the toxic heavy metal gadolinium. In patients with severe renal impairment, the use of gadolinium-based contrast agents has been associated with an increased risk of a very severe side effect called NSF, nephrogenic systemic fibrosis, which may even have a lethal outcome.
Both the European and U.S. regulatory authorities issued warnings for the use of gadolinium-based contrast agents in this group of patients. Patients with impaired kidney function will therefore typically receive an MRI without contrast, which can result in liver images of suboptimal quality, risking that their cancer is not managed in the best possible way. We envision that OrbiGlans, which is based on manganese, not gadolinium, will address this unmet medical need and in the future become an efficacious non-gadolinium liver imaging contrast agent for cancer patients with impaired kidney function. The addressable market for OrbiGlans has a global value of $800 million annually. The U.S. represents almost half of this. This market opportunity for OrbiGlans addresses the unmet need for a well-defined patient population, the 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 strategy supports our ambition to secure the optimal balance between future revenues and investment required. Our focused, ambitious launch strategy and the plans are built on advanced market insights, and they are ready and in place to support this partnering strategy and the launch of OrbiGlans. As mentioned, the U.S. is the largest commercial opportunity. In the U.S. 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. Patients that fall under the black box warning for gadolinium contrast agents, and 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.
We have extensive input from market access and pricing experts with whom we've tested different pricing levels and collected insights on the evidence needed to support access and reimbursement. We have investigated pricing and access benchmarks of other innovative diagnostics in the U.S. 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. These insights come from market research with 270 U.S. healthcare professionals, and the insights confirm the concerns with gadolinium in clinical practice and the unmet need for OrbiGlans.
When speaking with experts, whether in radiology or nephrology, they confirmed that an attractive 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. 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 regulatory and medical bodies. It's also well known that gadolinium is excreted via the kidneys in urine, and because it's difficult to remove in our sewer 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's not associated with these potential safety and environmental concerns for patients and for the environment with gadolinium. The industry is responding. OrbiGlans will be the first-in-class liver MRI contrast agent for a future with less gadolinium and improved outcomes for our target patient population. Recent developments from large gadolinium manufacturers are focused on an early-stage injectable manganese contrast agent, which is not liver-specific like OrbiGlans, or focused on smaller doses of gadolinium. We're excited that we have a head start with the upcoming approval and launch of OrbiGlans. The go-to-market strategy for OrbiGlans is to launch with commercialization partners. This strategy supports our objective to maximize the value of OrbiGlans. OrbiGlans is an attractive partnering opportunity. With clinical development completed, we offer a de-risked asset in the process of FDA approval. There's a clear unmet need.
With a focused launch with synergies from many types of commercial-stage companies working with hospital decision-makers. Our dialogue with potential partners for the successful commercialization of OrbiGlans is progressing. In summary, we are excited about the opportunity to bring OrbiGlans to approval and to market with a partner, a market representing $800 million US dollars annually in an addressable market for an unmet need for very vulnerable patients. We have come a long way with the successful completion of clinical development, and our data supporting efficacy and safety of OrbiGlans is well received by the medical community. With this, I will hand over to Andreas to talk about our NDA submission and the review process ahead with the FDA.
Thank you, Julie. We submitted the NDA, the marketing approval application, to the FDA on the 3rd of September. As explained by Magnus and Julie, the aim is to obtain an approval for the use of OrbiGlans as a liver contrast agent in patients where gadolinium contrast may be medically inadvisable, including those patients who have severely impaired kidney function. We have completed a comprehensive clinical development program, including a total of 286 patients and healthy volunteers across nine clinical studies. These studies have shown an improved visualization of focal liver lesions, and the safety assessment has demonstrated that the most common adverse reactions relate to the GI tract and are generally mild and transient in nature. Together, the data shows a positive risk-benefit profile. The Phase III study, SPARCL.
Very clearly demonstrated that visualization of focal liver lesions measured by the co-primary efficacy variables border delineation and lesion contrast were improved with OrbiGlans compared to unenhanced imaging, with a very high degree of statistical significance. We could also see that more lesions could be detected in the liver with OrbiGlans than without. In summary, the NDA for OrbiGlans was submitted to the FDA in early September with a very strong data package. We expect to pass the important Day 74 milestone by mid-November. We continue with a standard FDA process with a timeline to completion of the review at around 10 months from submission. While still prioritizing our resources for successful approval and partnering of OrbiGlans, we also have OncRol in our portfolio. OncRol is a tablet formulation of irinotecan, 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 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 I studies with OncRol, we've seen encouraging results that suggest that oral daily administration of irinotecan could offer meaningful benefits to patients. Importantly, the OncRol 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 potential and the continued development of OncRol as a daily oral formulation of irinotecan. Our plan is to bring OncRol into clinical Phase II in gastric cancer.
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 combination very interesting for us. The planned Phase II study is designed to demonstrate clinical proof of concept for the combination of OncRol and Lonsurf in gastric cancer. Our strategy is to start OncRol development in gastric cancer, which is today a $3 billion market. Irinotecan is a well-established chemotherapy with recognized anti-tumor effects in many types of solid cancers or tumors. Therefore, we see opportunities for developing OncRol in other indications too, where daily dosing could positively impact the efficacy and safety outcomes for the patients. We are assessing these opportunities as part of our ongoing strategic planning for OncRol. With that, back to Julie again.
Thank you, Andreas. I will now move to the update on our financials and priorities ahead. In Q3, our operating result was a loss, i.e., cost of SEK 15 million. The costs are lower than in Q2 this year due to the finalization of the NDA. At the end of September, we had SEK 72 million in liquid assets. This quarter, we considerably strengthened our balance sheet. In September, Fenja converted all outstanding convertibles of SEK 7.5 million, and later in the month, we successfully completed a directed share issue, raising SEK 30 million before cost. With this fundraise, we broaden and anchor our investor base. We now have a cash runway into Q4 2026, well beyond the expected FDA approval date of OrbiGlans. To wrap up our call today, we have substantial value creation opportunities ahead for OrbiGlans and Ascelia Pharma.
With OrbiGlans, we're bringing to market a first-in-class diagnostic drug addressing an $800 million market for patients with a high unmet need. We have two key objectives. One is the timely approval of OrbiGlans with the optimal label. We submitted the NDA early September. Mid-November, we expect communication from the FDA regarding the expected review completion and approval date. With a standard 10-month review, we can expect an approval early July 2026. Our other objective is to progress OrbiGlans for commercialization for patients in need by entering into a partnering agreement for the launch and by securing that a partner and OrbiGlans is ready for launch by approval. These efforts continue to progress. All in all, we've progressed well in Q3 with the OrbiGlans NDA submission and the strengthening of our balance sheet.
We're excited to advance OrbiGlans through the FDA review process and to advance the partnering process for the launch of OrbiGlans. We look forward to continuing our journey with opportunities for growing Ascelia Pharma into 2026 and beyond. That was the end of our presentation.
Thank you, everyone, for listening. We'd be happy to take any questions now.
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. The next question comes from Maria Karrlson Osipova from DNB Carnegie. Please go ahead.
Hello, everyone. Thank you for taking my question. Maria here from DNB Carnegie. I wanted to take the first one on the cost profile. You've mentioned no commercial preparation costs in the report that you've posted now this morning. When do you expect those costs to start appearing in the P&L? Basically, in general, how should we think about the cost picture going forward?
Yes, thank you for your question. Since our strategy is to launch with a partner, we do not expect any significant launch preparation costs for OrbiGlans. Our focus in terms of preparing launches is to make sure the manufacturing is ready for a partner. There are some costs, of course, associated with that, but not significant. In terms of the cost picture going forward, I mean, we have SEK 72 million end of the quarter in cash, and we have communicated that our cash runway is into Q4 next year. That, of course, is towards the approval date mid next year. That is our current business plan, and then you can sort of project the expected costs based on that. Of course, costs have gone down now after the submission of the NDA, and that is a typical pattern we can expect also going forward.
I mean, as they are not going much further down, but yeah.
All right. Thank you, Julie. To keep us on the FDA track, that's the most imminent and interesting trigger upcoming, of course, and you expect the standard 10-month review timeline. How confident are you in that the dataset is submitted that fully meets their expectations, maybe based on your pre-NDA meetings? Are there any pitfalls here? Any outstanding clarifications that may come? Could you shed some light on that?
Yeah, thank you for that question. Yeah, we are confident that we have submitted a very strong data package. Nothing has changed in the period from submission until now. As a normal process, FDA have asked a few clarifying questions, but nothing that is indicating of any concerns. It is more to make sure that they are preparing themselves to complete the review in an adequate way.
Yeah. You do not see any scenarios where the date for the PDUFA communication can be delayed as of now? We have talked about this before, I think, but just a quick recap.
Nothing has changed in our assessment of this application.
Okay. Thank you. I'll get back in the queue and see if other people have questions as well. Thank you very much.
As a reminder, if you wish to ask a question, please dial pound key five on your telephone keypad. The next question comes from Maria Karllson Osipova from DNB Carnegie. Please go ahead.
All right, then I'll continue with another one. You've mentioned the dose reduction trend in gad that's out there. On that topic, when you are out there and talking about your product's competitive edge, are you hearing any objections from KOLs? Because we hear a lot of positive things, obviously, but do you hear any feedback on objections that might come regarding the potential of your product in this context?
Thank you for asking that question. I think the dynamic has actually changed over the last years towards an increased discussion around the safety of gadolinium, even from those big manufacturers. Going from the black box warnings, where gadolinium manufacturers were saying, "Well, we're not too concerned. These are very few patients." Now, what is happening with those half-dose launches is that they are also bringing into the discussion at conferences and advisory boards, so forth. We don't really know. The risk is still there. We should be careful about using gadolinium. We think it's definitely also to our advantage that the safety around gadolinium is a topic for all players in the industry. It can help us. We don't think it's a concern for OrbiGlans. Also, there's no data supporting that risk of gadolinium is dependent on dose of gadolinium.
Anyways, we think it's good for patients and for OrbiGlans that the safety is a discussion topic for all of us.
Yes. Thank you. The main goal is partnering, of course, but could you shed some light on what are your main criteria when you're evaluating potential partners? Is it the commercial reach, the radiology footprint maybe, or oncology focus, or anything else that you're thinking about?
Yeah, I think you're pointing to synergies. It's important. I mean, it makes good sense that there are commercial synergies for either the operations of the launch or the portfolio strategy of the partner. That's one part. Another part is we want, of course, a partner where OrbiGlans is a meaningful contribution to the P&L or their strategy. Or both. It is a combination of how it fits to the portfolio and the synergies. Yeah, I think those are the key criteria. Of course, the terms that they put on the table.
Oh, of course. That will be all from me in the interest of time. Thank you very much.
Thank you.
Yeah.
There are no more phone questions at this time. I hand the conference back to the speakers for any written questions and closing comments.
Yeah. We have received some questions in the chat. Let's start with this one. That's a regulatory question related to EU and other ex-U.S. EU regions. In terms of when we apply and how long that would take. Andreas, do you want to start on that one?
We have focused on the FDA process and making sure that we get an approval there. The EU and other regions is very much dependent on the progress and collaboration with your partners. The exact timing there is a matter of synchronization, if you like, with other processes here.
Yeah. Another question comes to why we did the share issue. What the implications are in terms of also relation to partnering. I can start on that on my own, and maybe others can chip in here. You would say we received some inbound interest, investor interest, and we thought that would be a good opportunity to strengthen the balance sheet. That would give us a longer financial runway, so we would be on the other side, could finance the company to the other side of the expected approval from the FDA. We think that also would give us a much better negotiation position in the partnering discussions. I think overall the conclusion for us was that this was a good opportunity to put us in a better place for the partnering and the NDA process.
Another question here. Is related to sort of the timing for preparing a U.S. launch, including sort of reimbursement and when we can expect sort of reimbursement after the approval. Julie, can you walk us through some thoughts there?
I mean, reimbursement in particular in the U.S. is a gradual process. We have come a long way in preparing the roadmap and working with payer and reimbursement experts. It is ready for execution, especially now that we also have our clinical data. I mean, not long after approval, OrbiGlans can be in the market, and then gradually, of course, the bigger payers can adopt OrbiGlans so it is a standard. I do not see OrbiGlans being particularly different. Anything from the day after the product is ready in the hand of a physician till some of the processes take three-six months. It is standard and no different for OrbiGlans. The key thing is that we have really prepared the strategy also for the payer environment and for reimbursement, tested it several times, and the roadmap is ready to be executed by a partner.
There's a question here. We had similar questions in the previous calls, but in terms of any update on the partnering progress. Also, sort of a backup in terms of if we're not getting a partnership. Maybe I can start with the latter one and say the plan is to get a partner, and we continue along that track, and that is the plan. We expect to be able to get a partnership in place. Julie, in terms of, can you provide?
Yeah.
It's difficult.
Yeah. I mean, we have. Of course, we've been asked by you and others, and I mean, we completely understand the curiosity and the interest in the partnering process. What is key is OrbiGlans is a very attractive asset and commercial opportunity, and we continue to progress those discussions. It's not in the interest of Ascelia or in the interest of U.S. shareholders to provide the details because then we limit our opportunities to negotiate the best possible deal. As discussed in my presentation, the key is that we've successfully taken OrbiGlans very far, and this is a de-risked opportunity for many types of partners. That's the key to us, and we strongly believe in the strategy. Yeah.
Any thoughts on, sort of, the—we have an upcoming milestone. Any thoughts in terms of partnership?
No, for sure. I mean, I think definitely the continuation of this de-risking after day 74, the asset is further de-risked. From a partner perspective, that is very soon, mid-November. Every step in that process brings more value to us and de-risks the asset for a partner. Yeah, it will definitely support also the partnering discussions.
Yeah. We have a question here in terms of why we focused on patients with severe renal impairment and others who are, or gadolinium may be medically inadvisable or cannot be administered. A question also relating to this is a small subset of the overall patient population. Will you start, Julie?
Yeah. The key is that for this patient population, there is the highest unmet need due to the risks associated with the use of gadolinium in this patient population. When there's a high unmet need, it also means that there's a higher value potential per patient. It means that we can obtain a higher price per dose for OrbiGlans than with a gadolinium compound or with addressing the volume market. The gadolinium compounds have been on the market for years. It's also a volume, a competitive, you could say, market in the sense it's a volume game played by some very big companies, GE, Bracco, so forth.
We actually think the best opportunity for OrbiGlans in terms of helping patients, in terms of the value for Ascelia, in terms of the commercialization strategy, is really to focus on these patients, which also means we have an orphan drug designation from the FDA. That has led to this, you can say, leaner and faster clinical development program and also a lot of support from the FDA to our decision-making along development. For many reasons, both in terms of development time and cost, and in terms of the overall commercial opportunity and the execution of that, this is the most attractive strategy.
Just as a reminder, our agent is based on manganese and not the gadolinium, which is out there. We are really making a difference for these patients that the existing products cannot do in any of the same way because it is a highly differentiated first-in-class product.
Yeah. I think these are.
Yeah. I think this concludes the list of questions. Thank you for joining our Q3 webcast. Happy to have given you this update and look forward to continue to update you as we progress. Thank you and have a wonderful day.
Thank you, Operator.