Ascelia Pharma Ab · OMXSTO:ACE
Ascelia Pharma Ab — Q1 2024 Earnings Presentation
40 slides from Ascelia Pharma Ab's earnings presentation, May 16, 2024
Slide 1: Bringing Orviglance to Patients
ADVANCING ORPHAN ONCOLOGY Ticker symbol: ACE Nasdaq Stockholm www.ascelia.com Bringing Orviglance to Patients Investor Update Ascelia Pharma | Investor Update | 7 May 2024, 14:00h CEST Investor Update | 7 May2024
Slide 2: Taking Ascelia Pharma to the Next Level
ADVANCING ORPHAN ONCOLOGY Ticker symbol: ACE Nasdaq Stockholm www.ascelia.com Taking Ascelia Pharma to the Next Level Magnus Corfitzen, CEO Ascelia Pharma | Investor Update | 7 May 2024, 14:00h CEST Investor Update | 7 May2024
Slide 3: 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 4: Bringing Orviglance to Patients
Bringing Orviglance to Patients Presentations available Take Ascelia Pharma to the Next Level Magnus Corfitzen, CEO Advance Orviglance from Phase 3 to Approval Andreas Norlin, CSO Progress Commercialization Readiness Julie Waras Brogren, Deputy CEO Investor Update 7 May 2024 Live Q&A 4 Investor Upda
Slide 5: Objectives of today's presentation
Objectives of today’s Investor Update • Further explain the strong headline results • Share plans for the upcoming regulatory and commercialization activities • Outline value creation opportunities ahead for Ascelia Pharma 5
Slide 6: Our Mission
Our Mission At Ascelia Pharma, we identify, develop and commercialize novel drugs that address unmet needs of people with rare cancer conditions 6 Investor Update | 7 May2024
Slide 7: 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 8: Orviglance successfully meets primary endpoint
ORVIGLANCE SUCCESSFULLY MEETS PRIMARY ENDPOINT • Phase 3 study demonstrated strong superiority in visualization of focal liver lesions with Orviglance (CMRI) compared to unenhanced MRI • All three readers meet the primary endpoint (p<0.001) with statistical significance • The reliability of the data
Slide 9: 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 10: Substantial value creation opportunities
SUBSTANTIAL VALUE CREATION OPPORTUNITIES Advance Orviglance to Progress Orviglance Develop pipeline potential approval commercialization readiness Timely submission and approval by the Focused launch for well-defined Demonstrate Oncoral efficacy and US FDA as an orphan drug with an patient populatio
Slide 11: Creating value by building on our strengths
CREATING VALUE BY BUILDING ON OUR STRENGTHS Pursuing our vision… … with focused, strategic capabilities Identify and acquire assets that address unmet medical needs for patients At Ascelia Pharma, we Define optimal value proposition, development & market pathway identify, develop and commercialize n
Slide 12: Substantial value creation opportunities
SUBSTANTIAL VALUE CREATION OPPORTUNITIES Advance Orviglance to Progress Orviglance Develop pipeline potential approval commercialization readiness Full Clinical Study Report Advance launch readiness Initiate Phase 2 clinical study early Q4 2024 when financing allows Commercialization Conclusions fro
Slide 13: Advancing Orviglance from Phase 3 to Approval
ADVANCING ORPHAN ONCOLOGY Ticker symbol: ACE Nasdaq Stockholm www.ascelia.com Advancing Orviglance from Phase 3 to Approval Andreas Norlin, CSO Ascelia Pharma | Investor Update | 7 May 2024, 14:00h CEST Investor Update | 7 May2024
Slide 14: 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 15: Bringing Orviglance to Patients
Bringing Orviglance to Patients Presentations available Take Ascelia Pharma to the Next Level Magnus Corfitzen, CEO Advance Orviglance from Phase 3 to Approval Andreas Norlin, CSO Progress Commercialization Readiness Julie Waras Brogren, Deputy CEO Investor Update 7 May 2024 Live Q&A 15 Investor Upd
Slide 16: Strong superiority of Orviglance in Phase 3
STRONG SUPERIORITY OF ORVIGLANCE IN PHASE 3 Primary endpoint met successfully Orviglance clinical development completed Three out of three readers scored primary Primary efficacy and safety of Orviglance visualization variables significantly higher confirmed in pivotal Phase 3 with Orviglance than w
Slide 17: Pivotal Orviglance Phase 3 successfully completed
PIVOTAL ORVIGLANCE PHASE 3 SUCCESSFULLY COMPLETED Patients • Global study, 85 patients from 32 study sites in USA, Europe, and Latin America • Known or suspected focal liver lesions and severe kidney impairment Endpoints • Primary: Improved lesion visualization (Lesion border delineation + lesions c
Slide 18: Clinical data package for regulatory submission
CLINICAL DATA PACKAGE FOR REGULATORY SUBMISSION Phase 1 studies investigating safety, absorption and signal intensity 9 Total 4 studies with 126 healthy volunteers Phase 2 studies investigating efficacy and safety in patients with known metastases Total 4 studies with 75 patients Nine studies with c
Slide 19: Advancing Orviglance towards approval
ADVANCING ORVIGLANCE TOWARDS APPROVAL Clinical Nonclinical US FDA Timely submission and approval by the US FDA as an orphan drug with an optimal label for the CMC use in the target population 19 Investor Update | 7 May2024
Slide 20: Key milestones ahead for Orviglance
KEY MILESTONES AHEAD FOR ORVIGLANCE Early Q4 2024 Q1 2025 Mid-2025 Advancing Orviglance to approval FDA pre-NDA NDA NDA NDA Full SPARKLE FDA review interactions finalization Submission Approval report 10 months standard review Progressing commercialization readiness Secure launch readiness Establish
Slide 21: Substantial value creation opportunities
SUBSTANTIAL VALUE CREATION OPPORTUNITIES Advance Orviglance to Progress Orviglance Develop pipeline potential approval commercialization readiness Full Clinical Study Report Advance launch readiness Initiate Phase 2 clinical study early Q4 2024 when financing allows Commercialization Conclusions fro
Slide 22: Progressing Commercialization Readiness
ADVANCING ORPHAN ONCOLOGY Ticker symbol: ACE Nasdaq Stockholm www.ascelia.com Progressing Commercialization Readiness Julie Waras Brogren, Deputy CEO Ascelia Pharma | Investor Update | 7 May 2024, 14:00h CEST Investor Update | 7 May2024
Slide 23: 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 24: Bringing Orviglance to Patients
Bringing Orviglance to Patients Presentations available Take Ascelia Pharma to the Next Level Magnus Corfitzen, CEO Advance Orviglance from Phase 3 to Approval Andreas Norlin, CSO Progress Commercialization Readiness Julie Waras Brogren, Deputy CEO Investor Update 7 May 2024 Live Q&A 24 Investor Upd
Slide 25: Well defined, attractive opportunity
WELL DEFINED, ATTRACTIVE OPPORTUNITY Unique commercial opportunity Clear unmet need for well-defined patient population Orphan drug designation A strong value proposition for regulators, payers, physicians and patients Attractive market Clear ambition Focused, ambitious launch Liver imaging for canc
Slide 26: Orviglance - filling an unmet need in liver MRI
ORVIGLANCE – FILLING AN UNMET NEED IN LIVER MRI Patient landscape Clinical practice 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 gado
Slide 27: Attractive addressable market
ATTRACTIVE ADDRESSABLE MARKET 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 world with potenti
Slide 28: 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 29: Unenhanced MRI preferred for target patients today
UNENHANCED MRI PREFERRED FOR TARGET PATIENTS TODAY 78% of physicians prefer MRI without or with partial dose … even more for patients with acute contrast for patients with chronic kidney disease (CKD) kidney injury (AKI) 86% 78% Percentage of responses 75% 62% 22% 16% 14% 11% Severe kidneyGFR 15-29
Slide 30: 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 31: 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 32: On track for optimal commercialization
ON TRACK FOR OPTIMAL COMMERCIALIZATION Strategic objectives for commercialization Global commercialization through partners • Optimal balance between investment with potential for Ascelia Pharma led launch in the US required and future revenues • Maximize value with shared global launch strategy Sec
Slide 33: Clear roadmap for optimal launch
CLEAR ROADMAP FOR OPTIMAL LAUNCH Progress launch readiness securing VALUE CREATION Build network & advocacy with KOLs Secure evidence early access for PAYERS Develop advocacy for PATIENTS Target clinical DECISION MAKERS Secure availability of PRODUCT Build launch EXECUTION 33 Investor Update | 7 May
Slide 34: Building clinical insights and network with leading experts
Progress launch readiness securing VALUE CREATION Building clinical insights and network with leading experts Leading US experts in liver disease and imaging share their experience and advice, at Ascelia Pharma Advisory Board Meeting, New York May 2023 • Dr. Alessandro Furlan (Radiology, University
Slide 35: Building patient insights and policy advocates
Progress launch readiness securing VALUE CREATION Building patient insights and policy advocates Patient Roundtable with support from Expert Advisors and Patient Advocacy Group “I don’t want to go on dialysis because then my life changes forever. I don’t know if I can ride a horse and that’s my life
Slide 36: Supply chain on track
Progress launch readiness securing VALUE CREATION SUPPLY CHAIN ON TRACK Ensuring quality with optimal ramp-up and seamless customer decisions Manufacturing partner, Specialty distribution partners Halo Pharmaceuticals, New Jersey (US) Distribution for seamless procurement Supply chain strategy
Slide 37: Reimagine imaging for people with poor kidney function
Progress launch readiness securing VALUE CREATION Reimagine imaging for people with poor kidney function. 37
Slide 38: 84% US healthcare professionals will use Orviglance
84% US HEALTHCARE PROFESSIONALS WILL USE ORVIGLANCE LIKELIHOOD OF USING ORVIGLANCE FOR TARGET PATIENTS (market research) 16% Likely to definitely (5-7 of 7) Not likely to somewhat likely (1-4 of 7) 84% Market research for Ascelia Pharma conducted in Q4 2021/Q1 2022 by Two Labs Pharma Services N =254
Slide 39: Substantial value creation opportunities
SUBSTANTIAL VALUE CREATION OPPORTUNITIES Advance Orviglance to Progress Orviglance Develop pipeline potential approval commercialization readiness Full Clinical Study Report Advance launch readiness Initiate Phase 2 clinical study early Q4 2024 when financing allows Commercialization Conclusions fro
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