Oncopeptides Ab · OMXSTO:ONCO
Oncopeptides Ab — Investor Day (November 2020)
56 slides from Oncopeptides Ab's investor day presentation, Nov 30, 2020
Slide 1: The Transformation of Oncopeptides
THE TRANSFORMATION OF ONCOPEPTIDES Capital Markets Day November 30, 2020
Slide 2: Disclaimer
DISCLAIMER IMPORTANT: You must read the following before continuing. The following applies to this document, the oral presentation of the information in this document by Oncopeptides AB (the “Company”) or any person on behalf of the Company, and any question-and-answer session that follows the oral
Slide 3: Introduction and Strategy for Value Growth
Introduction and Strategy for Value Growth Marty J Duvall, CEO Oncopeptides 3
Slide 4: Welcome to Capital Markets Day
WELCOME TO CAPITAL MARKETS DAY KEY TAKEAWAY MESSAGES • Global company with R&D roots in Sweden • Large unmet needs in multiple myeloma • Melflufen has potential to become a backbone treatment in relapsed refractory multiple myeloma • Organization fully ready for US launch • PDC platform enables us t
Slide 5: The Transformation of Oncopeptides - Program
THE TRANSFORMATION OF ONCOPEPTIDES PROGRAM Introduction and strategy for value growth o Marty J Duvall, CEO Oncopeptides Myeloma remains one of the largest unmet medical needs within hematology o Assistant Professor Joshua Richter, MD. Icahn School of Medicine at Mount Sinai Hospital, New York Panel
Slide 6: Oncopeptides Company History
ONCOPEPTIDES COMPANY HISTORY PEPTIDE DRUG CONJUGATE (PDC) TECHNOLOGY PLATFORM • Founded in Stockholm, Sweden in 2000 • Focused on anticancer drug development based on PDC platform • Collaborations with leading cancer research institutions • US Offices in Boston and San Francisco Stockholm, Sweden Ph
Slide 7: Oncopeptides Team
ONCOPEPTIDES TEAM STRONG AND DIVERSE TEAM UNIFIED AND FOCUSED TO DRIVE VALUE Our core values • Science • Passion • Collaboration • Courage • Trust 7
Slide 8: Oncopeptides at a Glance
ONCOPEPTIDES AT A GLANCE TRANSFORMATION INTO A FULLY INTEGRATED BIOPHARMA COMPANY Discovery and Portfolio Development and Commercial launch and IND generation Life Cycle Management geographic expansion 8
Slide 9: Peptide Drug Conjugate
PEPTIDE DRUG CONJUGATE PDC TECHNOLOGY PLATFORM • Drug conjugates use innovative technology that attaches a cytotoxic agent onto a tumor-targeting element.1 • The PDC platform allows concentration of a toxic payload into cancer cells by exploiting the difference in peptidase activity between cancer c
Slide 10: Discovery and IND Generation
DISCOVERY AND IND GENERATION LEVERAGING PDC PLATFORM AND BUILDING A PIPELINE • Solna-based laboratory acquired in June 2020 • Expanded capabilities to harness the potential of metabolic enzymes in cancer cells - Team of passionate and externally focused scientists - State of the art medicinal chemis
Slide 11: Portfolio Development and Life Cycle Management
PORTFOLIO DEVELOPMENT AND LIFE CYCLE MANAGEMENT LEAD PROGRAM EXPANDING IN MULTIPLE MYELOMA AND BEYOND • Strong differentiation versus other alkylators • Broad pre-clinical activity • Multiple Myeloma focus - Triple-class refractory or heavily pre-treated patients Melfufen potential in different canc
Slide 12: Launch Investment and Geographic Expansion: Multiple Myeloma
LAUNCH INVESTMENT AND GEOGRAPHIC EXPANSION MULTIPLE MYELOMA - A HEMATOLOGICAL CANCER WITH NO CURE Rapid market growth Significant unmet needs remain $23B • Survival increasing with new drug classes $19B • Most patients are treated with - Immunomodulatory drugs (IMiD) - Proteasome inhibitors (PI) ROW
Slide 13: Launch Investment and Geographic Expansion
LAUNCH INVESTMENT AND GEOGRAPHIC EXPANSION DRIVEN TO MAXIMIZE VALUE FOR SHAREHOLDERS AROUND THE GLOBE Launch Phase Regulatory Phase Early Reg Phase • “Go at it alone” strategy • “Go at it alone” strategy • Likely partnering strategy • Boston-based HQ for US • Targeting “conditional • Gap analysis un
Slide 14: Myeloma Remains one of the Largest Unmet Medical Needs Within Hematology
Myeloma Remains one of the Largest Unmet Medical Needs Within Hematology Assistant Professor Joshua Richter MD, Icahn School of Medicine at Mount Sinai Hospital, New York 14
Slide 15: Relapsed refractory multiple myeloma and melflufen – clinical experience
Relapsed refractory multiple myeloma and melflufen – clinical experience with melflufen Panel discussion 15
Slide 16: Relapsed Refractory Multiple Myeloma and Melflufen
RELAPSED REFRACTORY MULTIPLE MYELOMA AND MELFLUFEN CLINICAL EXPERIENCE WITH MELFLUFEN Klaas Bakker, M.D. Ph.D. Maria-Victoria Mateos, M.D. Ph.D. Paul G. Richardson, M.D. Chief Medical Officer Associate Professor of Medicine R.J. Corman Professor of Medicine Oncopeptides AB University of Salamanca Ha
Slide 17: Understanding how melflufen can help patients the most
Understanding how melflufen can help patients the most The PDC platform and the clinical development program for melflufen Jakob Lindberg, CSO Oncopeptides 17
Slide 18: Melflufen - A First in Class Drug Candidate
MELFLUFEN - A FIRST IN CLASS DRUG CANDIDATE A PEPTIDE-DRUG CONJUGATE TARGETING AMINOPEPTIDASES PDC mechanism has potential to add several benefits Amino-peptidase Alkylating payload • Increased potency of linked toxin due to aminopeptidase binding domain targeting with subsequent hydrolysis • Potenc
Slide 19: Aminopeptidases Are Excellent Cancer Targets
AMINOPEPTIDASES ARE EXCELLENT CANCER TARGETS KEY ROLE IN CANCER CELL SURVIVAL, PROLIFERATION AND MIGRATION Amino-peptidases play a key role in protein homeostasis, and in other critical functions such as cell-cycle progression, programmed cell death and cell migration I Amino-peptidases are over-exp
Slide 20: Novel Drug Conjugates Targeting Multiple Myeloma
NOVEL DRUG CONJUGATES TARGETING MULTIPLE MYELOMA TWO DIFFERENT TECHNOLOGIES TO ACHIEVE SAME OBJECTIVE 1 Antibody–drug conjugate Multiple myeloma cell Binding to Antibody cell surface antigen Cytotoxic drug payload Lysosomal degradation Linker Endocytosis of ADC-antigen Release of active complex cyto
Slide 21: The Evolving Myeloma Landscape – A Few Years Ago
THE EVOLVING MYELOMA LANDSCAPE – A FEW YEARS AGO… NEWLY DIAGNOSED MARKET DOMINANT – 3x RRMM EVENT LATE Newly Diagnosed MM RMM RRMM Triple-Class Refractory Event 21
Slide 22: The Evolving Myeloma Landscape – Current
THE EVOLVING MYELOMA LANDSCAPE – CURRENT RRMM GROWING WITH MAJORITY OF PATIENTS BEING 3x RRMM Newly Diagnosed MM RMM RRMM Triple-Class Refractory Event 22
Slide 23: Melflufen – Current Clinical Development Program
MELFLUFEN – CURRENT CLINICAL DEVELOPMENT PROGRAM FULL CLINICAL DEVELOPMENT PROGRAM IN RRMM O-12-M1 HORIZON US US EU EU OCEAN ANCHOR Phase 1 Phase 2 BRIDGE AL-amyloidosis LIGHTHOUSE PORT 2016 2017 2018 2019 2020 2021 2022 Phase 1 and 2: Phase 2: single-arm Phase 3: Phase 1 and 2: Phase 2: study in Ph
Slide 24: Details – Melflufen Current Clinical Studies
DETAILS – MELFLUFEN CURRENT CLINICAL STUDIES EU EU LIGHTHOUSE OP201 sEAPort PHASE 3 PHASE1/2 PHASE 3 RANDOMIZED TRIPLE RANDOMIZED PHASE 1/2 PHASE 2 PHASE 2 COMPARATIVE COMBINATION PHASE 2 COMPARATIVE SINGLE-ARM STUDY PHASE 2 Expanded Access SINGLE-ARM STUDY SINGLE-ARM STUDY SUPERIORITY STUDY STUDY S
Slide 25: Planned New Studies in 2021 - RRMM
PLANNED NEW STUDIES IN 2021 - RRMM • RRMM patients with extramedullary disease (EMD) represents one of RRMM patients with the largest unmet medical needs in multiple myeloma EMD • Melflufen showed highly encouraging data in EMD patients in HORIZON • Planned study start during 2021 • Strong clinical
Slide 26: Melflufen Development
MELFLUFEN DEVELOPMENT DESIGNED TO SUPPORT USE AFTER IMiD AND PI FAILURE MUST HAVE CHARACTERISTICS MELFLUFEN Single agent +/- steroid activity in multi-refractory patients O-12-M1 showed an ORR of 31%, HORIZON an ORR of of >20% Overall Response Rate (ORR) 29% in multi-refractory patients OCEAN pivota
Slide 27: Developing Melflufen for RRMM Patients
DEVELOPING MELFLUFEN FOR RRMM PATIENTS US MARKET – CURRENT PATIENT NUMBERS Clinical Program Drives Label Expansion 3x RRMM Anticipated label in triple-class 20,000+ refractory patients 1-2x RRMM 1-2x RRMM Head-to-head comparison study Single drug use Comb. use with the most used regimen in 25,000 20
Slide 28: Competitive Melflufen Data
COMPETITIVE MELFLUFEN DATA TRIPLE CLASS REFRACTORY MULTIPLE MYELOMA PATIENTS Melflufen Xpovio Blenrep Oncopeptides Karyopharm GSK US NDA, June 30, 2020 US approval, July 2019 US Approval, Aug 6, 2020 Number of patients studied 119 122 95 Overall Response/Clinical Benefit Rate 26%/39% 25%/39% 31%/36%
Slide 29: Pivotal Trials
PIVOTAL TRIALS OCEAN AND LIGHTHOUSE • Head-to-head comparison with the most used drug in RRMM – pomalidomide • Pomalidomide is perceived as the drug with the best combination of efficacy and safety in RRMM • Both non-inferiority and superiority outcome from OCEAN is a positive • Based on good combin
Slide 30: OCEAN Study - Head to Head vs Pomalidomide
ENROLLMENT COMPLETED IN PHASE 3 OCEAN STUDY 495 PATIENTS RECRUITED – TOP-LINE RESULTS IN H1 2021 Head to Head study versus pomalidomide Melflufen + Primary N = 495 Randomization dexamethasone endpoint: Lenalidomide- PFS refractory Secondary multiple myeloma Pomalidomide + endpoint: patients dexameth
Slide 31: Pomalidomide Shares Resistance Mechanism with Lenalidomide
POMALIDOMIDE SHARES RESISTANCE MECHANISM WITH LENALIDOMIDE Average IMiD free period significant in pomalidomide Pomalidomide efficacy decreases registration study for recent lenalidomide failures • Only 29% received lenalidomide as last treatment Median overall survival (months) 20 Lenalidomide used
Slide 32: Phase 3 OCEAN Study
PHASE 3 OCEAN STUDY TWO WAYS TO MEET PRIMARY ENDPOINT • OCEAN meets its primary endpoint with a superiority or non-inferiority result • Pomalidomide is the largest treatment option in RRMM with very good physician perception OCEAN OUTCOME FDA EMA Primary endpoint met - Superiority ✓ ✓ Primary endpoi
Slide 33: LIGHTHOUSE Study - Based on Positive ANCHOR Data
LIGHTHOUSE STUDY - BASED ON POSITIVE ANCHOR DATA CONFIRMATORY PHASE 3 STUDY – AWAITING GO-AHEAD Phase 3 study with melflufen in multiple myeloma • Melflufen + daratumumab vs daratumumab randomized 1:1 • Subcutaneous version of daratumumab Objectives • Expand market potential – expand label for melfl
Slide 34: Peptide Drug Conjugate Platform
PEPTIDE DRUG CONJUGATE PLATFORM THERAPEUTIC ACTIVITY IN MOST CANCERS PDC Potentiation • Melflufen is focused on multiple myeloma and AL-amyloidosis • New molecules are based on PDC platform • Potential broadening of indications in AML, Non-Hodgkin Lymphoma and breast cancer 34
Slide 35: Planned New Clinical Studies 2021 Outside Multiple Myeloma
PLANNED NEW CLINICAL STUDIES 2021 OUTSIDE MULTIPLE MYELOMA KEY AREAS OF EXPLORATION • Significant unmet medical need in AML Relapsed/refractory AML • Strong pre-clinical data with melflufen in AML patients • Phase I/II trial planned to be initiated in 2021 • Significant unmed medical need in sub-gro
Slide 36: Developing Peptide-Drug-Conjugate Platform
DEVELOPING PEPTIDE-DRUG-CONJUGATE PLATFORM FROM PRE-CLINICAL TO CLINICAL DEVELOPMENT 2020/21 EXPLORATORY LATE PRECLINICAL DEVELOPMENT DEVELOPMENT PHASE 1 PHASE 2 PHASE 3 REGISTRATION MARKET Melflufen OPD 5 New CD 36
Slide 37: Successfully Making the Drug Available for Patients in the US
Successfully Making the Drug Available for Patients in the US Overview of US launch of melflufen Marty J Duvall, CEO Oncopeptides 37
Slide 38: Melflufen Product Positioning
MELFLUFEN PRODUCT POSITIONING ASPIRATIONAL POSITIONING IN MULTIPLE MYELOMA Melflufen, used alone and in combination, is a first in class peptide-drug conjugate that leverages aminopeptidases and rapidly releases alkylating agents into tumor cells • “First in class” supports a novel product and diffe
Slide 39: Improved Outcomes Lead to Fast Growth in Treated Patients
IMPROVED OUTCOMES LEAD TO FAST GROWTH IN NUMBER OF TREATED PATIENTS IN LATER LINES OF THERAPY Projected US multiple myeloma patients by line of therapy Q3 2017 Q3 2020 60000 CAGR: 6% 50000 CAGR: 9% 40000 CAGR: 9% 30000 CAGR: 9% 20000 10000 0 1L 2L 3L 4L+ Source: Intrinsiq MAT Q3 2020 39 Note: 3-yr a
Slide 40: Newer Products on Top of Older as Survival Improves
NEWER PRODUCTS ON TOP OF OLDER AS SURVIVAL IMPROVES NEED OF NEW TREATMENT OPTIONS US MM # of Total Patients by Product 70000 Revlimid 60000 Velcade Darzalex 50000 Pomalyst 40000 Kyprolis Ninlaro 30000 Cytoxan 20000 Empliciti melphalan 10000 Farydak 0 Xpovio Jul-17 Oct-17 Jan-18 Apr-18 Jul-18 Oct-18
Slide 41: Triple-Class Refractory Multiple Myeloma
TRIPLE-CLASS REFRACTORY MULTIPLE MYELOMA AN INDICATION WITH GROWING UNMET MEDICAL NEED Triple-class refractory % patients after each LoT 40% 29% 30% 30% 27% Estimated >20,000 Triple- 20% class refractory 15% 16% patients in the 11% US and 10% growing 4% 6% 3% 0% 2016 2017 2018 2L 3L 4L+ 41
Slide 42: Become a Leading Choice in TRC MM and Expand Market
BECOME A LEADING CHOICE IN TRC MM AND EXPAND MARKET TWO-PRONGED STRATEGIC APPROACH Driving change in today’s RRMM treatment paradigm Common Practice to “recycle” drugs within existing classes as patients progress EXISTING CLASSES New MOAs NEW CLASSES/ MOAs Become leading choice New MOAs 1 Selinexor
Slide 43: Paving the Way for a Successful Launch ... The Team
PAVING THE WAY FOR A SUCCESSFUL LAUNCH ... THE TEAM US LEADERSHIP ORGANIZATION WITH SIGNIFICANT ONCOLOGY LAUNCH EXPERIENCE Mohamed Ladha, General Manager US Business Unit Paula O’Connor, MD, Head of Medical Affairs US 17 years in industry with extensive oncology launch expertise 17 years industry ex
Slide 44: Paving Way to Ensure Best Customer Experience
ONCOPEPTIDES IS PAVING WAY TO ENSURE BEST CUSTOMER EXPERIENCE ”ONE ONCOPEPTIDES” TEAM IN MOTION • An integrated and coordinated field force team Onc. Medical Science Nurse Educators • Oncology Account Managers spearhead the tactical Liaisons execution and pulls in appropriate resources Overall accou
Slide 45: Putting Patients First by Eliminating Barriers to Therapy
PUTTING PATIENTS FIRST BY ELIMINATING BARRIERS TO THERAPY PROVIDING CONTINUOUS SUPPORT ALONG THE PATIENT JOURNEY ACCESS – No Patient Left Behind • Payer engagement for “prior authorization” • Support any “appeals” processes • Uninsured patients (Free Drug program) • Quickstart program AFFORD – Reduc
Slide 46: Engaging with Payers Representing >95% of Covered Lives
ENGAGING WITH PAYERS REPRESENTING >95% OF COVER MEDICAL LIVES MARKET ACCESS TEAMING WITH MEDICAL AFFAIRS TO LAY THE GROUNDWORK UnitedHealth Group/OPTUM BlueCross BlueShield North Carolina • Medical Affairs leads Anthem Cambia Health Solutions (Regence) melfufen clinical profile Aetna & CVS Caremark
Slide 47: Distribution and Supply Chain
DISTRIBUTION AND SUPPLY CHAIN SLIDE US ORGANIZATION SET FOR LAUNCH Supply Chain and distribution Third Party Logistics (3PL) in place to Traditional Specialty Distributor model strategy aims to optimize access, ensure supply and distribution of familiar to end-customers (IDNs, control and customer s
Slide 48: Materials Ready and Tailored for Targeted Customers
MATERIALS READY AND TAILORED FOR TARGETED CUSTOMERS* DRIVE BROAD AWARENESS APPROVAL OF NOVEL TREATMENT OPTION FOR MM • Now approved in relapsed or refractory multiple myeloma • The first and only anticancer peptide-drug conjugate HIGHLIGHT EFFICACY RESULTS IN DIFFICULT TO TREAT PATIENTS • Clinically
Slide 49: Customer Information Management System
CUSTOMER INFORMATION MANAGEMENT SYSTEM PROVIDING REAL-TIME CUSTOMER INSIGHTS TO IMPROVE SERVICE Leveraging integrated healthcare patient-level data and delivering weekly insights Rules Based Alert Development AI/ML Predictive Modeling with Alerts AI= Artificial Intelligence ML=Machine Learning 49
Slide 50: Medical Affairs
MEDICAL AFFAIRS US ORGANIZATION SET FOR LAUNCH Medical Information • Majority of KOLs and Large Scientific Exchange Data Generation & Communication Community practices engaged and profiled • Call Center • MSL and Nurse • Investigator Initiated • Compendia & dossiers Educator Teams Trial (IIT) progra
Slide 51: Melflufen Awareness Continues to Increase in US
MELFLUFEN AWARENESS CONTINUES TO INCREASE IN US MOMENTUM IS GROWING AFTER SCIENTIFIC MEETINGS % MDs likely or very likely to use % MDs with awareness of melflufen product X by line of therapy 80% 53% TBD 49% 60% 60% 38% 38% 50% 40% 20% 20% 20% 11% 10% 0% EHA 2019 ASH 2019 EHA 2020 October 2020 ASH 2
Slide 52: Effective RRMM Drugs Without Major Side Effects Have Significant Potential
EFFECTIVE RRMM DRUGS WITHOUT MAJOR SIDE EFFECTS OR COMORBIDITY LIMITATIONS HAVE A SIGNIFICANT POTENTIAL Q4 2019-Q3 2020 Worldwide Sales $M, (YoY Growth %) $3 767 (37%) $2 927 CD38 mAb (22%) IMiD PI Other $1 120 $776 (4%) $384 (20%) $10 $20 $73 $74 (16%) (n/a*) (n/a*) (12%) (n/a*) Blenrep Sarclisa Fa
Slide 53: News Flow
NEWS FLOW VALUE DRIVERS AND MAJOR MILESTONES Q3 2020 Q4 2020 Q1 2021 Q2 2021 H2 2021 / H1 2022 First patient in PORT Expanded Access First patient in EU-submission Potential conditional study Program (US) opened LIGHTHOUSE conditional approval approval in EU First patient in Intent to file for EU Fi
Slide 54: In Summary
IN SUMMARY KEY TAKEAWAY MESSAGES • Global company with R&D roots in Sweden • Large unmet needs in multiple myeloma • Melflufen has the potential to become a backbone treatment in relapsed refractory multiple myeloma • Organization fully ready for US launch • PDC platform enables us to broaden our po
Slide 55: Q/A session
55 Q/A session © oncopeptides
Slide 56: © oncopeptides
© oncopeptides