Slide 1: Orviglance Completes Clinical Development with Successful Phase 3 Study
ADVANCING ORPHAN ONCOLOGY Ticker symbol: ACE Nasdaq Stockholm www.ascelia.com Orviglance® Completes Clinical Development with Successful Phase 3 Study Half Year Report 2024 Conference call presentation on 15 August 2024, 10:00 CET
Slide 2: Forward Looking Statements
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 As
Slide 3: Quarterly Report Q2 2024 Investor Conference Call
QUARTERLY REPORT Q2 2024 INVESTOR CONFERENCE CALL Agenda Ascelia Pharma highlights Recent key events Portfolio Financials and priorities ahead Presenters CEO - Magnus Corfitzen Deputy CEO - Julie Waras Brogren CSO - Andreas Norlin 3
Slide 4: Ascelia Pharma - Highlights
ASCELIA PHARMA - HIGHLIGHTS Pipeline ORVIGLANCE® – Registration phase ▪ 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 ▪ Phase 3 study successful and clinical developme
Slide 5: Q2 2024 Progress
Q2 2024 PROGRESS Key events in Q2 2024 SPARKLE image reading completed with expected headline results first half of May 2024 Ascelia Pharma draws down SEK 15 million second tranche under existing loan Primary endpoint successfully met with strong headline results in Orviglance phase 3 study
Slide 6: Rights Issue to Capitalize on Phase 3 Results
RIGHTS ISSUE TO CAPITALIZE ON PHASE 3 RESULTS Allowing all shareholders to take part of the value Key dates creation opportunities ahead EGM to approve the Rights Issue 14 Aug 2024 • Raising up to 105 MSEK Last day of trading in shares including the right to receive unit rights 14 Aug 2024 • Investm
Slide 7: Attractive Orviglance Opportunity
ATTRACTIVE ORVIGLANCE OPPORTUNITY 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 Orviglance
Slide 8: Substantial Value Creation Opportunities
SUBSTANTIAL VALUE CREATION OPPORTUNITIES Advance Orviglance to approval Progress Orviglance commercialization Develop pipeline potential readiness Timely submission and approval by the Focused launch for well-defined Demonstrate Oncoral efficacy and Objectives US FDA as an orphan drug with an patien
Slide 9: Portfolio
ORVIGLANCE® Liver diagnostic imaging drug ONCORAL Daily, oral chemotherapy PORTFOLIO 9
Slide 10: Orviglance - Filling an Unmet Need in Liver MRI
ORVIGLANCE – FILLING AN UNMET NEED IN LIVER MRI Patient Landscape Treatments Unmet Need Liver metastases are Contrast enhanced MRI A role for ORVIGLANCE critical in cancer care is the gold standard in patients with severe kidney impairment Patients with healthy kidneys ▪ Receive MRI with gadolinium-
Slide 11: Strong Superiority of Orviglance in Phase 3
STRONG SUPERIORITY OF ORVIGLANCE IN PHASE 3 Primary Endpoint Met Successfully Phase 3 study demonstrated strong superiority in visualization of focal liver lesions with Orviglance (CMRI) compared to unenhanced MRI Visualization scored significantly higher with Orviglance than without for all three r
Slide 12: Clinical Development Completed
CLINICAL DEVELOPMENT COMPLETED Phase 1 studies demonstrated safety, absorption and signal intensity 9 Total 4 studies with 126 healthy volunteers Phase 2 studies demonstrated efficacy and safety in patients with known metastases Total 4 studies with 75 patients Nine studies with consistent positive
Slide 13: Advancing Orviglance Towards Approval
ADVANCING ORVIGLANCE TOWARDS APPROVAL Clinical US FDA Timely submission and approval by the US FDA as an orphan drug with an optimal label for Nonclinical use in the target population • Full Clinical Study Report early Q4 2024 • Conclusions from FDA pre-submission CMC meeting by Q1 2025 • NDA submis
Slide 14: Addressable Market of USD 800 Million Annually
ADDRESSABLE MARKET OF USD 800 MILLION ANNUALLY Japan Global addressable market of USD 800 million, Europe half of this in the US Focused launch for well-defined patient population in liver US imaging in cancer patients with severe kidney impairment Rest of Global commercialization through partners w
Slide 15: Attractive US Opportunity
ATTRACTIVE US OPPORTUNITY Abdominal imaging procedures in cancer patients ~100,000 with severe kidney impairment based on epidemiology and real-world data1 procedures annually Around 400 healthcare provider accounts serve ~400 accounts 75% of kidney impaired patients4 Pricing range benchmarks based
Slide 16: Unmet Need Recognized in Clinical Practice
UNMET NEED RECOGNIZED IN CLINICAL PRACTICE “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 NSF* risk (from Ascelia Pharma Advisory Board meeting) with warnings for target population +90% +16% of
Slide 17: Unenhanced MRI Preferred Today; 84% of US Physicians Likely to Use Orviglance
UNENHANCED MRI PREFERRED TODAY; 84% OF US PHYSICIANS LIKELY TO USE ORVIGLANCE 78% of physicians prefer MRI without or … even more for patients with Likelihood of Using Orviglance with partial dose contrast for patients acute kidney injury (AKI) for Target Patients with chronic kidney disease (CKD) 8
Slide 18: Momentum for an Alternative to Gadolinium
MOMENTUM FOR AN ALTERNATIVE TO GADOLINIUM Deposition in brain & organs concerns around safety for all patients New safety category recommended for Symptoms Associated with Gadolinium exposure (SAGE), by Am. College of Rad. (2022) Water contamination Multiple-GBCA effect on body movement scrutiny of
Slide 19: On Track for Optimal Commercialization
ON TRACK FOR OPTIMAL COMMERCIALIZATION Strategic objectives for commercialization Global commercialization through partners • Optimal balance between investment required and future revenues • Leverage established commercialization Secure launch readiness capabilities • Maximize value with global lau
Slide 20: Reimagine imaging for people with poor kidney function
Reimagine imaging for people with poor kidney function. 20
Slide 21: Portfolio
ORVIGLANCE® Liver diagnostic imaging drug ONCORAL Daily, oral chemotherapy PORTFOLIO 21
Slide 22: Improving Irinotecan Efficacy and Tolerability
IMPROVING IRINOTECAN EFFICACY and TOLERABILITY Irinotecan intravenous bolus dosing irinotecan ONCORAL – irinotecan oral daily dosing High-dose IV infusion Oncoral low-dosing every 3 weeks daily tablet Serious side-effects Potential for improved efficacy limit efficacy with reduced side-effects Tumor
Slide 23: Oncoral Phase 2 in Gastric Cancer
ONCORAL PHASE 2 IN GASTRIC CANCER STRONG RATIONALE FOR GASTRIC CANCER LONSURF AND IRINOTECAN COMBINATION • High unmet need and clinically demonstrated RANDOMIZED CONTROLLED PHASE 2 STUDY • ~100 patients with metastatic gastric cancer • Potential for synergistic effect between Lonsurf and irinotecan
Slide 24: High Value Opportunity in Gastric Cancer and Expansion
HIGH VALUE OPPORTUNITY IN GASTRIC CANCER AND EXPANSION POTENTIAL FOR ORAL, DAILY DOSING OF IRINOTECAN3 Global 3 000 000 Annual A WELL-ESTABLISHED CHEMOTHERAPY Incidence with recognized anti-tumor effect in solid tumors 2 500 000 Lung Colorectal Current focus: Gastric cancer • Clinically demonstrated
Slide 25: Financials & Outlook
FINANCIALS & OUTLOOK 25
Slide 26: Rights Issue to Capitalize on Strong Phase 3 Results
RIGHTS ISSUE TO CAPITALIZE ON STRONG PHASE 3 RESULTS Rights Issue overview Use of proceeds • Raising up to 105 MSEK • NDA submission to FDA • Investment commitments and guarantees • Phase 3 full Clinical Study Report, Q4 2024 amounting to 70 MSEK • Conclusions from FDA pre-submission meeting, Q1 202
Slide 27: Operating Result - Maintained Low Operating Expenses
OPERATING RESULT– MAINTAINED LOW OPERATING EXPENSES Operating result (EBIT) (SEK million) SG&A costs Operating loss of SEK 11.3 million in Q2 2024 R&D costs Slightly decreased loss (costs) compared to -41.8 Q1 2024 driven by completion of SPARKLE re-evaluation 10,6 -21.4 -16.7 7,4 31,2 -11.1 5,9 -11
Slide 28: Liquidity - Cash Runway Beyond NDA Submission Mid-2025
LIQUIDITY - CASH RUNWAY BEYOND NDA SUBMISSION MID-2025 Liquid assets including marketable securities (SEK million) Liquid assets of 30 MSEK (30 Jun 2024), including 35 MSEK fully drawn Fenja Capital financing of loan and convertibles 71 Runway with minimum 70 MSEK Rights Issue financing 39 22 27 30
Slide 29: Substantial Value Creation Opportunities
SUBSTANTIAL VALUE CREATION OPPORTUNITIES Advance Orviglance to approval Progress Orviglance commercialization Develop pipeline potential readiness Timely submission and approval by the Focused launch for well-defined Demonstrate Oncoral efficacy and Objectives US FDA as an orphan drug with an patien
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