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ADVANCING ORPHAN ONCOLOGY Ticker symbol: ACE Nasdaq Stockholm www.ascelia.com Orviglance® NDA Submitted to the FDA Q3 Report 2025 Conference call presentation on 5 Nov 2025, 10:00 CEST
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2 FORWARD LOOKING STATEMENTS This presentation, which includes all information and data on the following slides, any oral statements made when presenting these slides, and any other material distributed or statements made at, or in connection with, such presentation (the “Presentation”), relates to Ascelia Pharma AB (publ) (hereinafter, together with its subsidiaries, the “Company”) is furnished to you solely for your information and may not be reproduced or redistributed, in whole or in part, to any other person without the prior written consent of the Company. You should not rely upon it or use it to form the definitive basis for any decision, contract, commitment or action whatsoever, with respect to any transaction or otherwise. The information included in this Presentation may contain certain forward-looking statements relating to the business, financial performance and results of the Company and/or the industry in which it operates. Forward-looking statements concern future circumstances and results and other statements that are not historical facts, sometimes identified by the words “believes”, “expects”, “predicts”, “intends”, “projects”, “plans”, “estimates”, “aims”, “foresees”, “anticipates”, “targets”, and similar expressions. The forward-looking statements contained in this Presentation, including assumptions, opinions and views of the Company or cited from third party sources are solely opinions and forecasts which are subject to risks, uncertainties and other factors that may cause actual events to differ materially from any anticipated development. Neither the Company nor any of its affiliates, directors, employees or advisors provides any assurance that the assumptions underlying such forward-looking statements are free from errors nor do any of them accept any responsibility for the future accuracy of the opinions expressed in this Presentation or the actual occurrence of the forecasted developments. This Presentation speaks as of the applicable reporting date, and there may have been changes in matters which affect the Company subsequent to the date of this Presentation. Neither the issue nor delivery of this Presentation shall under any circumstance create any implication that the information contained herein is correct as of any time subsequent to the date hereof or that the affairs of the Company have not since changed, and the Company does not intend, and does not assume any obligation, to update or correct any information included in this Presentation. Each person should make their own independent assessment of the merits of the Company and should consult their own professional advisors. By receiving this Presentation, you acknowledge that you will be solely responsible for your own assessment of the market and the market position of the Company and that you will conduct your own analysis and be solely responsible for forming your own opinion of the potential future performance of the Company’s business.
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3 QUARTERLY REPORT Q3 2025 INVESTOR CONFERENCE CALL Agenda Recent key events Portfolio Financials and priorities ahead Presenters CEO - Magnus Corfitzen Deputy CEO - Julie Waras Brogren CSO – Andreas Norlin
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4 At Ascelia Pharma, we identify, develop and commercialize novel drugs that address unmet needs of people with rare cancer conditions Global outlook and Nordic roots Based in Malmö (Sweden), US entity in New Jersey (US) Listed on NASDAQ Stockholm (Ticker: ACE)
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ADVANCED ORPHAN ONCOLOGY PIPELINE 5 Drug candidate Indication Phase 1 Phase 2 Phase 3 Registration Market ORVIGLANCE Improved detection and visualization of focal liver lesions ▪ First-in-class contrast agent for use in liver MRI in patients with severely impaired kidney function ▪ FDA Orphan Drug Designation ▪ Global addressable market of USD 800 million Completed Ongoing NDA submitted 3 Sept 2025 ONCORAL Improved efficacy and safety of solid tumor treatment ▪ Daily, oral irinotecan chemotherapy ▪ Clinical collaboration with Taiho Oncology ▪ Opportunity in gastric cancer and other solid tumors Completed Ready
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Key events in Q3 2025 New Drug Application (NDA) for Orviglance submitted to the US Food and Drug Administration (FDA) Directed share issue of approximately SEK 30 million completed Fenja Capital II A/S converted all outstanding convertibles of SEK 7.5 Million Updated timeline for submission of the Orviglance NDA to take place early September 2025 Key events after the period Management changes to support future growth 6 Q3 2025 PROGRESS
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7 SUBSTANTIAL ORVIGLANCE VALUE CREATION OPPORTUNITIES Looking ahead Objectives ✓ NDA submission September 2025 ▪ FDA communication of PDUFA dateNovember 2025 ▪ FDA approval following 10 months review July 2026 ▪ Advance launch readiness ▪ Establish commercialization partnership(s) Timely approval by the US FDA as an orphan drug for the use in liver MRI for patients with severe renal impairment or when gadolinium may otherwise be medically inadvisable Focused launch for well-defined patient population with 800 MUSD annual addressable market Partner driven global commercialization Advance Orviglance to approval Secure partnering and commercialization readiness
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ORVIGLANCE® Liver diagnostic imaging drug ONCORAL Daily, oral chemotherapy 8 PORTFOLIO
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A well-defined unmet need for liver imaging in cancer patients with impaired kidney function A global addressable market opportunity of USD 800 million Clinical development completed with 9 studies and strong phase 3 results Commercial scale manufacturing 9 NDA submitted to the FDA ATTRACTIVE ORVIGLANCE OPPORTUNITY Commercialization with partner
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ORVIGLANCE – FILLING AN UNMET NEED IN LIVER MRI 10 Liver metastases are critical in cancer care Contrast enhanced MRI is the gold standard Patients with healthy kidneys ▪ Receive MRI with gadolinium- based contrast agent (GBCA) Patients with severe kidney impairment ▪ Black Box warning for gadolinium contrast agents ▪ Risk of severe side effects, incl. Nephrogenic Systemic Fibrosis (NSF) Liver metastases are common in many cancer types and often the cause of mortality1-3 ▪ Colorectal cancer, metastatic breast cancer, gastric cancer 1) Riihimäki, M. et al. Patterns of metastasis in colon and rectal cancer. Sci. Rep. 6, 29765; doi: 10.1038/srep29765 (2016); Journal of Pathology, 2014, 232:23-31 2) Guy diSibio and Samuel W. French (2008) Metastatic Patterns of Cancers: Results From a Large Autopsy Study. Archives of Pathology & Laboratory Medicine: June 2008,Vol. 132, No. 6, pp. 931-939 3) Rahbari et al. Metastatic Spread Emerging From Liver Metastases of Colorectal Cancer: Does the Seed Leave the Soil Again? Annals of Surgery: February 2016 - Volume 263 - Issue 2 - p 345–352 A role for ORVIGLANCE in patients with severe kidney impairment Contrast enhanced MRI ▪ Detection and visualization ▪ Surgery & drug treatment plan ▪ Post-treatment surveillance Patient Landscape Treatments Unmet Need ORVIGLANCE Aims to be the imaging option without gadolinium-related safety risks in patients with severe kidney impairment ▪ Manganese based ▪ Liver specific
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ADDRESSABLE MARKET OF USD 800 MILLION ANNUALLY 11 Sources: Ascelia Pharma market research on real-world volumes with Decision Resources Group, 2020.. Ascelia Pharma market access research and analyses with Revenue Reimbursement Solutions and Charles River Associates (2020), Triangle (2022) and Trinity (2022), incl. 75 stakeholder and expoert interactions. 1) Final pricing strategy is subject to Phase 3 data, payer evidence, negotiations, discounts and access strategy US Rest of world Europe Japan Global addressable market of USD 800 million, half of this in the US Focused launch for well-defined patient population in liver imaging in cancer patients with severe kidney impairment Global commercialization through partners
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ATTRACTIVE US OPPORTUNITY 12 Sources: 1) Ascelia Pharma market research with Decision Resources Group, 2020. Literature on prevalence and epidemiology of kidney disease, cancer and liver metastases. 2) Ascelia Pharma market access research and analyses with Revenue Reimbursement Solutions and Charles River Associates(2020), Triangle (2022) and Trinity (2022) 3) Final pricing strategy is subject to Phase 3 data, payer evidence, negotiations, discounts and access strategy 4) Ascelia Pharma analysis based on market research with Decision Resources Group, 2020 Around 400 healthcare provider accounts serve 75% of kidney impaired patients4 ~400 accounts Pricing range benchmarks based on innovative diagnostics, payer and expert input and price testing2, 3 $3,000-4,500 Abdominal imaging procedures in cancer patients with severe kidney impairment based on epidemiology and real-world data1 ~100,000 procedures annually
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UNMET NEED RECOGNIZED IN CLINICAL PRACTICE 13 Source: Independent research by Two Labs Pharma Services for Ascelia Pharma in the USA conducted in Q4 2021/Q1 2022 included 16 in depth interviews and a survey of 254 healthcare professionals (HCPs), including 154 radiologists, 50 nephrologists and 50 oncologists. Participants in the study were asked about their choices of imaging and contrast agents in patients with cancer +90% of HCPs are concerned by issues relating to GBCAs (including NSF) +16% of providers have experienced GBCA-induced NSF NSF* risk with warnings for target population *nephrogenic systemic fibrosis “Those of us who have seen NSF are frightened by it... you’ll get buy-in from a lot of nephrologists…”. - Head of Renal section at US university hospital (from Ascelia Pharma Advisory Board meeting) ““The college [American Colleague of Radiology]…have a growing sense of responsibility and accountability about using these agents in high-risk patients.… our perception of which agents are “safe” has changed… this is another place where practice needed to evolve” - SPARKLE Investigator and Head of Radiology at US university hospital
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MOMENTUM FOR CHANGE ALSO BEYOND RENAL IMPAIRMENT 14 1) Brünjes R. et al. Anthropogenic gadolinium in freshwater and drinking water systems, Water Research, Volume 182, 2020.. Other sources include: Macke et al. Fast and automated monitoring of gadolinium-based contrast agents in surface waters, Water Research, Volume 207, 2021. Oluwasola et al, Gadolinium based contrast agents (GBCAs): Uniqueness, aquatic toxicity concerns, and prospective remediation. Journal of Contaminant Hydrology, Volume 250, 2022. M. Nicholl. Seeking alternatives to gadolinium-based contrast agents. Healthcareineurope.com. July 22022 Bang G. Gadolinium Deposition in Brain: Current Scientific Evidence and Future Perspectives. Mol. Neurosci., 20 September 2018. Gadolinium excreted in urine is discharged into our environment and drinking water New category for Symptoms Associated with Gado. Exposure (SAGE, Am. College of Rad., 2022) Multiple-GBCA effect on movement and mental skills study requested by the FDA (ODYSSEY, 2020) Risk of potentially fatal side effects of gadolinium, incl. nephrogenic systemic fibrosis, in patients with severe renal impairment Water contamination scrutiny of environmental impact Deposition in brain & organs concerns around safety for all patients Manganese Orviglance, a first in class oral manganese agent targeting patients with severe renal impairment (Ascelia Pharma) Completion of Phase 1 of full-body IV manganese-based contrast agent (GE HealthCare) Gadolinium Low dose full-body gadolinium contrast agents pursued by GBCA players with one approved by the FDA (Guerbet/Bracco) and another in regulatory review (Bayer) Black-box warning for use in renally impaired patients Current challenges with gadolinium use A future with less gadolinium
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OPTIMAL COMMERCIALIZATION THROUGH PARTNERS 15 Attractive partner opportunity De-risked asset in registration phase Unmet need with high value for payers – high value per patient Clear decision maker value - patients, physicians and payers Focused launch effortsfor hospital/imaging units Wide synergy potential within e.g. radiology/nephrology/oncology or high-value/orphan drug Partner led commercialization Maximizes overall value of Orviglance by leveraging established capabilities Optimizes capital requirements vs. future revenues Dialogue with potential partners progressing
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16 Reimagine imaging for people with poor kidney function Orviglance data accepted for 4 oral and 5 poster presentations at major conferences since Phase 3
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ORVIGLANCE NDA SUBMITTED TO THE FDA 17 Obtain approval for Orviglance as a liver MRI contrast agent for patients with severe kidney impairment or when gadolinium may be otherwise medically inadvisable NDA submitted 3 September 2025 ▪ Attractive benefit-risk profile ▪ Established commercial-scale manufacturing ▪ Orphan Drug Designation granted, offering regulatory and commercial benefits
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Nine studies with consistent positive efficacy and safety results1-8 286 patients and healthy volunteers Superior efficacy compared to unenhanced imaging • Superior visualization of focal liver lesions • More lesions detected, in particular small lesions (< 1cm) • Consistent improvement of visualization across main patient groups • Efficacy further supported by secondary endpoints across studies NDA SUPPORTED BY ROBUST CLINICAL PROGRAM 18 1) Thomsen HS et al, Acad Radiol 2004: 11: 630-636 2) Thomsen HS et al. Eur Radiol 2007, 17: 273-278 3) Rief M et al. Invest Radiol. 2010; 45: 565-71 4) Brismar TB et al.. Eur Radiol 2012; 22:633-41 5) Albiin N et al. MAGMA. 2012; 25:361-368 6) Brismar TB, et al., Invest Radiol 2025: Apr 8. doi: 10.1097/RLI.0000000000001184. Online ahead of print. 7) Study CMC-P005, primary objective to study of Orviglance for imaging of bile ducts (not published) 8) Results from Phase 1 and 2 and Food Effect and Hepatic Impairment Studies presented at RSNA and ESGAR conferences between 2022 and 2023 9 Favorable safety profile • Robust non-clinical and clinical safety data with no concerning findings • Minimal systemic exposure of manganese • Mild GI-related adverse reactions most frequently reported • No serious drug-related reactions
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Without Orviglance (T1, Pre-contrast) With Orviglance (T1, post-contrast) SUPERIORITY OF ORVIGLANCE DEMONSTRATED IN PHASE 3 19 *Visualization assessed by 3 independent readers as the improvement of Lesion border delineation (LBD) and Lesion contrast (LC) on combined Orviglance-enhanced + unenhanced (CMRI) images compared to unenhanced (UMRI) images for all matched lesions, using a 4-point scale (from 1 (“poor”) to 4 (“excellent”)). Data presented as mean paired differences for matched lesions per patient for CMRI and UMRI with 95% Confidence Intervals. One-sided paired t-test (=0.025). Total N=85, n=number of patients with matched lesions (per reader). Improved visualization of focal liver lesions with Orviglance -1,5 -1 -0,5 0 0,5 1 1,5 Mean paired difference in* LBD (●) and LC (●) score (CMRI - UMRI) Favors OrviglanceFavors unenhanced Reader 1 (n=61) p<0.001 Reader 2 (n=53) p<0.001 Reader 3 (n=61) p<0.001 Example from a patient with metastases in the SPARKLE Phase 3 study Improved visualization Improved visualization Improved detection
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ORVIGLANCE PROGRESSES TOWARDS US APPROVAL IN 2026 20 Objective: Timely approval by the US FDA as an orphan drug for the use in liver MRI for patients with severe renal impairment or when gadolinium may otherwise be medically inadvisable Milestones: 3 Sept 2025: NDA submission (review ongoing) November 2025: Day 74 (PDUFA announced) July 2026: Approval (10 months review) Future opportunities: Apply for marketing approval in EU and other ex-US regions
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21 PORTFOLIO ORVIGLANCE® Liver diagnostic imaging drug ONCORAL Daily, oral chemotherapy
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22 irinotecan Oncoral low-dosing daily tablet Serious side-effects limit efficacy Potential for improved efficacy with reduced side-effects High-dose IV infusion every 3 weeks Tumor cells Tumor cells IMPROVING IRINOTECAN EFFICACY AND TOLERABILITY Irinotecan intravenous bolus dosing ONCORAL – irinotecan oral daily dosing
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Metronomic low-daily dose (Oncoral) Infrequent high-dose IV • Gastrointestinal and hematological side effects • ~30% severe or life- threatening (grade 3 or 4)1 ENCOURAGING SAFETY PROFILE IN PHASE 1 23 PLASMA LEVELS OF IRINOTECAN 1) Camptosar prescribing information 2) Furman et al, J Clin Oncol 17:1815, 1999 3) Perez et al. J Clin Oncol, 22:2849, 2004 4) Allegrini et al, Br J Cancer 98:1312, 2008 5) Bocci et al, Br J Cancer 98:1619, 2008 6) Kümler et al, Cancer Chemother Pharmacol 83:169, 2019 Frequent (metronomic) low-dose irinotecan • Several studies show potential for improved efficacy and tolerability2-5 • Daily dosing – adjust quickly if acute toxicity Oncoral Phase 1 results6 • Well tolerated, no unexpected side-effects • Hematological toxicities mild-moderate (grade 1 or 2) • 36% of patients with stable disease, in patients previously treated with IV irinotecan • Pharmacokinetic profile suitable for daily dosing SN-38 concentration (ng/mL) Time (days) Source: Simulation of Oncoral vs. IV Camptosar
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ONCORAL PHASE 2 IN GASTRIC CANCER 24 STRONG RATIONALE FOR GASTRIC CANCER • High unmet need and clinically demonstrated • Potential for synergistic effect between Lonsurf and irinotecan Control arm Lonsurf (TAS-102) IV irinotecan (CPT-11) Lonsurf + IV irinotecan Efficacy study in an animal model of gastric cancer1 (Relative Tumor Volume, RTV) 1) Nukatsuka et al, Anticancer Res 35:1437, 2015 PHASE 2 STUDY OUTLINE Clinical collaboration with LONSURF is approved for treatment of metastatic gastric cancer and metastatic colorectal cancer • ~100 patients with metastatic gastric cancer • Study arms: Oncoral + Lonsurf vs. Lonsurf • Endpoints: Progression Free Survival (Primary), Response Rate, PK, Safety (Secondary) and Overall Survival (follow-up) • IND approved in the US
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HIGH VALUE OPPORTUNITY IN GASTRIC CANCER AND EXPANSION 25 1) International Agency for Research on Cancer (IARC, 2021) 2) GlobalData - Gastric and Gastroesophageal Junction Adenocarcinoma – Global Drug Forecast and Market Analysis to 2024 3) Globocan 2020, WHO, Cancer Research UK • Clinically demonstrated • Guidelines recognized • 3rd highest cancer deaths1 • Orphan disease (US and EU) • $3-4bn market2 Colorectal Pancreatic Gastric Lung Esophagus Cervical Ovarian Liver NHL LeukemiaKidney - 500 000 1 000 000 1 500 000 2 000 000 2 500 000 3 000 000 0% 20% 40% 60% 80% 100% Indications where IV irinotecan are clinically demonstrated & guidelines recognized Indications where IV irinotecan are clinically demonstrated Approved indications for IV irinotecan Current focus: Gastric cancer POTENTIAL FOR ORAL, DAILY DOSING OF IRINOTECAN3 Global Annual Incidence Median 5-year Survival Rate A WELL-ESTABLISHED CHEMOTHERAPY with recognized anti-tumor effect in solid tumors
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26 FINANCIALS & OUTLOOK
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14,2 18,3 15,7 18,7 10,9 3,6 3,6 4,6 4,4 4,4 Q3 2024 Q4 2024 Q1 2025 Q2 2025 Q3 2025 -20.3 -23.0 -15.3 -17.8 -21.9 Operating loss of SEK 15 million in Q3 2025 Costs lower than Q2 due to finalization of the NDA. 27 Operating result (EBIT) (SEK million) Notes: Other operating income and other operating costs added to SG&A 24.7 SG&A costs R&D costs OPERATING RESULT– MAINTAINED LOW OPERATING EXPENSES
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96 75 57 60 72 Liquid assets including marketable securities (SEK million) Liquid assets of SEK 72 million (30 Sept 2025) Clean balance sheet following Fenja conversion of all outstanding convertibles of SEK 7.5 million (2 Sept 2025). Directed issue raising SEK 30 million before cost based on inbound investor interest (22 Sept 2025). Cash runway into Q4 2026 well beyond expected FDA approval date of Orviglance. 28 LIQUIDITY - CASH RUNWAY INTO Q4 2026 31 Mar 2025 30 Jun 2025 30 Sep 2025 30 Sep 2024 31 Dec 2024
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29 SUBSTANTIAL ORVIGLANCE VALUE CREATION OPPORTUNITIES Looking ahead Objectives ✓ NDA submission September 2025 ▪ FDA communication of PDUFA dateNovember 2025 ▪ FDA approval following 10 months review July 2026 ▪ Advance launch readiness ▪ Establish commercialization partnership(s) Timely approval by the US FDA as an orphan drug for the use in liver MRI for patients with severe renal impairment or when gadolinium may otherwise be medically inadvisable Focused launch for well-defined patient population with 800 MUSD annual addressable market Partner driven global commercialization Advance Orviglance to approval Secure partnering and commercialization readiness
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