Ascelia Pharma AB (publ)

Thank you and welcome everyone to the webcast for Celia Pharma's Q3 report for 2024. 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. At Acelia Pharma, we identify, develop, and commercialize novel drugs that address unmet medical needs within rare cancer conditions. We have two drugs in our pipeline. Orvigland is in registration phase, as we have successfully completed the pivotal phase three clinical study sparkle, and we're preparing the NDA submission. Orvigland has orphan drug designation from the FDA and is targeting an addressable market opportunity of $800 million. Oncoral is ready to start phase two in the treatment of gastric cancer based on encouraging results in phase one and a high level of unmet medical needs. Acelia Pharma is based in Malmö, Sweden, and we are listed on nasdaqs.com. So communications in Q3 were dominated by the right tissue that has now been completed. The rights issue was fully subscribed to 105 million Swedish kronor. And as part of this transaction, we repaid half the convertible loan to Fenja Capital. So it is now at 7.5 million. Since the end of Q3, there have been a number of activities that we have reported. The phase three primary endpoint results from SPACL have been accepted as a cutting edge or late breaker oral presentation at the RSNA, which is the largest radiology conference in the world. SPARQL results have also been presented as a late-breaking abstract at Kidney Week 2024, which is a large nephrology conference in the U.S. This is important as the target indication for Orvi glands include people with severe renal disease who regularly see a nephrologist to manage their kidney disease. We're pleased that Mariana Koch has been elected to the Board of Directors at Acilia Pharma, which happened at an extraordinary general meeting on October 30th. Mariana has a long experience from pharma in executive roles, and including work within drug development, regulatory affairs, and commercialization. Yesterday, we announced the completion of the full study report for SPARQL, which reinforces the successful outcome of the pivotal phase three study. Andreas will talk about this in a few minutes. We're very excited about Overglance, and here's why. Orbig Lens is addressing a well-defined, unmet medical need for a subgroup of people living with cancer. This is an $800 million global market opportunity, and Orbig Lens is a first-in-class product to target this and has often dropped designation from the FDA. We have strong data from nine clinical studies, and manufacturing has been upscaled to commercial scale. With the strong Phase III data, Orbig Lens has now been advanced to regulatory phase, and we are preparing for an NDA submission. We're pursuing some important value creation opportunities with OverGlance. The first objective is a timely submission and approval of OverGlance with the optimal label. The key steps on the way are completion of the SPARQL clinical study report here in early Q4, which is now in place with our announcement last night. The next milestone is the conclusions from the pre-submission meeting with the FDA, which is expected in Q1. And submission of the formal application of BNDA to the FDA in the middle of next year. The second key objective is to progress all the glands for commercialization. The key activities here are to continue to advance our launch readiness by ensuring manufacturing and supply chain is ready for launch, as well as working with medical experts, including key opinion leaders, payers, patient advocacy groups, and other key stakeholders. The other part of that is to entering into a commercialization partnership, which is also a process that we are continuing our efforts in. I'm very happy with the progress we're making in Acelia Pharma and the efforts made by our team to ensure we will meet our objectives and create value for shareholders. We will now start the portfolio section of our presentation with OrbeGlance, and I'd like to hand over to Andreas to continue the presentation.

Thank you, Magnus. OrbeGlance is a first-in-class liver MRI contrast agent which addresses a very specific unmet medical need for which there are no good alternatives available today. Treatment of liver cancer and liver metastasis is an important challenge within oncology. Adequate visualization of liver tumors and metastases is critical for making the right treatment decisions. Contrast-enhanced MRI is the gold standard procedure for examination of patients with suspected or known tumors or metastases. The most used contrast agents are all based on the heavy metal gadolinium. In patients with severe kidney impairment, 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 lethal outcome. Both the European and US regulatory authorities have for that reason issued warnings for the use of gadolinium-based contrast agents in this group of patients. The consequence is that patients with impaired kidney function typically will get an MRI without contrast, which will result in liver images of suboptimal quality, with the risk for that their cancer is not managed in the best possible way. We envision that Orviglance, which is based on manganese, will address this unmet medical need and in the future become an efficacious non-gadolinium contrast agent for liver MRI in patients with impaired kidney functions. In May, we shared the positive results from the SPARQL Phase 3 study. We found that overglans-enhanced liver MRI were superior to unenhanced MRIs. All three readers of the images reported strong, statistically significant results. We also noted that the common side effects were related to the gastrointestinal tract, which aligns with findings from previous studies on overglans. We have now finished the full study report for SPARQL, and I'd like to share some of the key data and conclusions. First, let's have a look at the primary endpoint, the enhancement of focal level lesion visualization assessed through two co-primary variables, border delineation and lesion contrast. These were rated on a scale of one to four by three independent readers. The mean scores for each patient aggregated across all readers are displayed in these bar charts. In the upper panel are the border delineation and in the lower panel you can see the lesion contrasts. The height of each bar indicates the percentage of all patients receiving that score. Blue bars represent unenhanced imaging results while orange bars show the combined evaluation of unenhanced and over-glance enhanced images. For unenhanced images, scores predominantly range from 2.1 to 3. In contrast, the combined assessment revealed a shift towards scores of 3 to 4, clearly indicating improved lesion visualization with OrvaGlass. The statistical evaluation compared overglance-enhanced scores with unenhanced scores within patients, and the mean pair differences along with their 95% confidence intervals are presented here. Notably, all mean pair differences are positive, indicating that overglance MRI is superior to unenhanced images for visualizing focal lever lesions. The analysis yielded a p-value of less than 0.01 for both variables across all readers, underscoring the significant advantage of combined MRI. To further validate our findings, subgroup analysis were conducted, and in every subgroup, combined MRI was preferred over unenhanced MRI. The study also included various secondary efficacy analysis and safety evaluations. The secondary endpoints aimed to assess different aspects of overglance performance, reinforcing the primary conclusion that overglance enhances lesion visualization. Among the secondary endpoints, we observed that overglance increased the detection of lesions measured as the proportion of patients with at least one additional lesion identified compared to unenhanced. Additionally, Orviglance improved the ability to detect and measure smaller lesions, with the mean size of the smallest lesion being on average 2 mm smaller with Orviglance compared to unenhanced imaging. The results from other secondary endpoints consistently demonstrate the superiority of Orviglance over unenhanced MRI, with no analysis favoring unenhanced MRI. These endpoints include a quantitative assessment of signal intensity in the images, recommendations for the next step in treatment, and reader confidence in detecting and localizing lesions. Importantly, the performance of overglance alone was similar to when both overglance-enhanced and unenhanced images were evaluated together. The primary safety analysis indicated that no serious adverse drug reactions were observed, and more than 80% of reported reactions were mild and short-lived, primarily related to the gastrointestinal tract, including nausea, diarrhea, and vomiting. The safety results from this vulnerable patient population were consistent with previous studies. With SPARQL, we have completed the clinical development The comprehensive clinical program includes a total of 286 patients and healthy volunteers in nine clinical studies. Taken together, these studies have consistently demonstrated positive efficacy and safety of warboglans. With the phase three SPARCA study confirming the superior visualization of focal liver lesions in the target population of patients with severe kidney impairment and an adverse event profile consistent with what were observed in the other studies, We are moving ahead with the submission of the Marketing Approval Application, the NDA, for Overglans. We are on track to submit the NDA for Overglans by mid 2025. Having completed the full study report for SPARQL, we are now focusing on finalizing our pre-NDA interaction with the FDA by the first quarter of next year. This step is crucial for laying the groundwork for our NDA submission and review, ensuring alignment with FDA and to facilitate a smooth review process. After submission, we expect the standard 10-month review period from the FDA. So in summary, the strong results from the SPARQL study show that Orviglans provides superior imaging compared to unenhanced MRI, allowing us to move forward into the registration phase of Orviglans. So now I will hand over to Julie.

Thank you, Andreas. The addressable market for OverGlands has a global value of $800 million annually. The US represents almost half of this. This market opportunity for OverGlands 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 strategy supports our ambition to secure the optimal balance between future revenues and investment required. Our focused ambitious launch strategy and plans built on advanced market insights are in place to support this partnering strategy and the launch. As mentioned, the US is the largest commercial opportunity. 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 50,000 patients that fall under the black box warning for gadolinium contrast agents. This is about 4% of people with cancer undergoing abdominal imaging. The well-defined patient population with this clear unmet need also drives an attractive value and 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 US. 90% of healthcare professionals are concerned with issues related to gadolinium contrast agents, including the severe side effects 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 US healthcare professionals and answers from radiologists, nephrologists and oncologists. And the insights confirm the concerns with gadolinium in clinical practice and the unmet need for overgans. When speaking to 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. In clinical practice, this need is indeed well recognized. Physicians tell us that patients who need liver imaging and fall under the black box warning for gadolinium, today receive MRIs that balance the trade-off between being either without a contrast agent, i.e. an inferior image quality, or by being half-dose gadolinium. These are around 80% of the patients. Or by being a full-dose gadolinium, with the awareness that gadolinium is not recommended for these patients due to the black box warning. In addition, we're both excited and optimistic that almost all physicians tell us that they would like to use OrbitGlance when it's available on the market. Beyond the risk of NSF in kidney impaired patients, gadolinium is well known to be retained in the brain and in 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 sewage systems, it is discharged into the environment and into our drinking water. There's an urgency 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. In short, the momentum is getting better and better, and the industry is responding. Recent developments from the large gadolinium manufacturers are focused on smaller doses of gadolinium, and there's even an early stage injectable manganese-based contrast agent, which is not liver specific like orbic glands. We are excited that we have a head start and that OverGlance is expected to be a first in class to lead a future with less gadolinium and improved outcomes for our target patients. The go-to-market strategy for OverGlance is to launch with commercialization partners and our dialogue with these potential partners is progressing. This supports our objective to secure the optimal balance between future revenues and investment required. This strategy also allows us to leverage commercial capabilities already established by a partner. The focus of Acelia is to continue the dialogue with potential partners and reach an attractive agreement for the commercialization of organs. We're also working to ensure that OverGlance and the partner is ready to launch upon approval. We're excited to announce recently that the headline data from SPARQL, the phase three study for OverGlance, was accepted in the late breaking or cutting edge categories for key conferences. RSNA for radiology and kidney week for nephrology. Our efforts to publish SPARQL data and to build relationships in the medical community continue as we progress the NDA and our commercialization with partners. In summary, OverGlance addresses a well-defined unmet need and represents an attractive commercial opportunity. Our focus is to ensure that OverGlance is ready for launch with a partner and our efforts to help patients to launch over glands to help patients and to launch over glands are well recognized by experts and partners in the industry and with this i will hand over to andreas to talk about encore thank you julie so yeah let's talk about encore the other asset in our development portfolio