Slide 1: CSL's Annual R&D Investor Briefing 2020
For personal use only For immediate release 20 October 2020 CSL’s Annual R&D Investor Briefing 2020 CSL Limited (ASX:CSL; USOTC:CSLLY) – CSL will hold its annual Research and Development briefing today; the presentation is attached for the information of investors. Amongst other achievements, CSL is
Slide 2: R&D Investor Briefing 2020
For personal use only R&D Investor Briefing 2020 20th October 2020
Slide 3: Forward looking statements
For personal use only Forward looking statements The information in this presentation is current as of the date of this presentation, and includes forward looking statements about CSL Limited and its related bodies corporate’s (CSL) financial results and estimates, business prospects and product can
Slide 4: Introduction
For personal use only Introduction William Mezzanotte MD Executive Vice President, Head of Research and Development and CMO CSL Behring 3
Slide 5: Agenda
Agenda For personal use only Topic Presenter Welcome Mark Dehring Introduction and Highlights Bill Mezzanotte Research - Protein Therapies, Gene Therapies & Vaccines Andrew Nash Immunology Highlights & COVID-19 Response Mittie Doyle Commercial Bill Campbell Transplant Highlights Laurie Lee Summary B
Slide 6: Global Research and Development Footprint
Global Research and Development Footprint For personal use only King of Prussia US Amsterdam Netherlands Liverpool UK Kankakee US Marburg Germany London UK Cambridge US Tokyo Japan Siena Italy Pasadena US Bern Switzerland Summit US Wuhan China Shanghai China Holly SpringsUS CSL Behring Seqirus M
Slide 7: Key Global Research Partnerships for Early Innovation Access
Key Global Research Partnerships for Early Innovation Access For personal use only 6
Slide 8: Commitment to Research and Development
Commitment to Research and Development For personal use only 922 832 New Product Development activities focus on innovative new 667 702 therapies for life-threatening 614 diseases Market Development strategies seek to bring therapies to new markets and new indications Life Cycle Management ensures c
Slide 9: Focus Through Our Therapeutic Areas and Platforms
Focus Through Our Therapeutic Areas and Platforms For personal use only 8
Slide 10: R&D Portfolio – December 2019
R&D Portfolio – December 2019 Registration/ For personal use only Research Pre-Clinical Phase I Phase II Phase III Post-Registration Discovery Projects CSL510 CSL730 Garadacimab HIZENTRA® PRIVIGEN® Modified Fibrinogen rFc Multimer Anti-FXIIa (HAE) mAb (DM) (PID) JP Discovery Projects CSL787 CSL324 H
Slide 11: R&D Portfolio Highlights - FY20
R&D Portfolio Highlights - FY20 Immunology Acquisitions & For personal use only Respiratory • HIZENTRA® Phase III DM study initiated Alliances • CSL311 (Anti-Beta Common) Phase I study in • Alliance with Seattle Children’s Research • HAEGARDA® Phase III HAE study in Japan mild asthmatic patients ini
Slide 12: Key Past Launches from R&D Portfolio
Key Past Launches from R&D Portfolio For personal use only 2015 2016 2017 2018 2019 2020 HIZENTRA® PRIVIGEN® CIDP (US) HIZENTRA® CIDP (JP) PRIVIGEN® Ig IsoLo® Immunology CIDP PID/SID (JP) HAEGARDA® (US) PRIVIGEN® CIDP (JP) IDELVION® IDELVION® 21-Day (EU)^ Haematology IDELVION® AFSTYLA® 21-Day (JP) R
Slide 13: Notable Regional Regulatory Action July 2019 – October 2020
Notable Regional Regulatory Action 1 July 2019 – 1 October 2020 For personal use only CSL889 65yrs+ 6mo+ (treatment SCD*) Garadacimab (Bk-AE*) 65yrs+ CSL889 (treatment SCD*) 21d dosing CSL964 (prevention GvHD***) Human Albumin 20% CSL964 (treatment GvHD***) Extension to register in Clazakizumab (CAB
Slide 14: R&D Portfolio Progression in 2020
R&D Portfolio Progression in 2020 For personal use only Pre-Clinical/ Registration/ Phase I Phase II Phase III Post-Registration CSL888 CSL346 Garadacimab PRIVIGEN® Haptoglobin (SAH) Anti-VEGF-B mAb Anti-FXIIa mAb(HAE) (PID) JP CSL787 Garadacimab HAEGARDA® IDELVION® Nebulised Ig Anti-FXIIa mAb (ARDS
Slide 15: CSL112 ApoA-1
CSL112 ApoA-1 For personal use only • All countries and sites reactivated CSL112 particles • Japan now active and enrolling well • 1st futility analysis in 2020 passed ABCA1 transporter Intracellular cholesterol 1° Endpoint : MACE MACE Follow Up D90 D180 D365 >17,000 AMI subjects 6g CSL112 ≥18yrs of
Slide 16: R&D Portfolio – October 2020
R&D Portfolio – October 2020 For personal use only Registration/ Research Pre-Clinical Phase I Phase II Phase III Post-Registration Gene Therapy CSL888 CSL324 HIZENTRA® HIZENTRA® PRIVIGEN® Treatments Haptoglobin (SAH) Anti-G-CSFR mAb (HS) (SSc) (DM) (PID) JP PID CSL510 CSL730 CSL630 HAEGARDA® IDELVI
Slide 17: Research: Protein Therapies, Gene Therapies, Vaccines
For personal use only Research Protein Therapies Gene Therapies Vaccines Andrew Nash PhD Senior Vice President, Research and CSO CSL Behring 16
Slide 18: CSL Research
CSL Research For personal use only • Global team exploiting internal and external expertise and 4 drug discovery CSL Behring CSL Behring platforms to deliver innovative Research Research Bern development opportunities across CSL therapeutic areas Melbourne Swiss Inst. for Translational & Entrepreneu
Slide 19: CSL Behring Research – Sourcing Innovation
CSL Behring Research – Sourcing Innovation For personal use only Research External Innovation Strategy - the competition for innovation Walter and Eliza Hall Institute (WEHI) Bioinformatics Alliance Seattle Children’s Institute Gene Therapy Alliance Strategic Partnerships (universities, Research Acc
Slide 20: Growing the Development Portfolio
Growing the Development Portfolio For personal use only Platforms Candidates Indications Plasma PID & SID proteins CSL888 Neutrophilic dermatoses CSL889 ANCA vasculitis CSL787 SLE COPD Asthma CSL312 ARDS CSL324 Pulmonary fibrosis Recombinant Thrombotic conditions CSL346 proteins SCD crisis CSL311 CS
Slide 21: Collaboration Delivers Innovation
Collaboration Delivers Innovation For personal use only Collaboration leads to the Antagonist and agonist discovery that BTN2A1 is required monoclonal antibodies for for the activation of γδ T cells use in autoimmune disease and immuno-oncology Source: Rigau, M. et al., (2020) Science 367(642) 20
Slide 22: Targeting Complement Regulation
Targeting Complement Regulation For personal use only Complement Pathways Potential indications Chronic Indications, Classical/Lectin Pathway (Anti-C2 mAb) C1INH Anti-C2 C1INH Acute Indications, Classical/Lectin Pathway (CSL040) Cyclic Acute ex vivo Pathway (CSL040) CSL040 solCR1 Anti-C5 Alternative
Slide 23: CSL040 Complement Receptor Inhibitor
CSL040 Complement Receptor 1 Inhibitor For personal use only CR1 Truncation Mutant, rCR1(1392) / CSL040 1908 1504 1540 1028 1605 1534 1763 1481 1310 1152 447 860 897 509 702 578 959 252 410 56 S P LHR-A LHR-B LHR-C LHR-D rCR1(1971) / TP-10 1 42 489 939 1392 1971 S P LHR-A LHR-B LHR-C rCR1(1392) = CS
Slide 24: CSL040 Complement Receptor Inhibitor
CSL040 Complement Receptor 1 Inhibitor For personal use only Renal Ischemia-reperfusion Injury (IRI) Mouse Model C57BL/6 Remove right kidney Sacrifice at 24 hrs Inject treatment or vehicle Clamp blood supply to Unclamp i.v. 60 mg/kg CSL040 left kidney for 20' (reperfuse) 1 hr pre-IRI and 3 hr later
Slide 25: Garadacimab: Global leaders in FXII biology
Garadacimab For personal use only Global leaders in FXII biology – new opportunities for Garadacimab FXI → FXIa FXIIa - Auto-activation FXII +FXIIa/β FXII Hemostasis PK → KAL* βFXIIa Plasminogen Fibroblast/ HK → BK→ BR2 C1qr,s → C1qr,s Plasmin endothelial cell/immune cells Vasodilation Complement Fi
Slide 26: Garadacimab: Pulmonary Fibrosis
Garadacimab Plasma FXII levels are higher in IPF patients with progressive disease For personal use only Pulmonary Fibrosis Garadacimab reduces fibrosis in the mouse bleomycin model of IPF Garadacimab from D0 Garadacimab from D5 FXII expression is higher in the IPF lung Translation to human disease
Slide 27: Gene Therapy
Gene Therapy For personal use only AMT-061 (EtranaDez) gene Seattle Children’s Research Direct delivery Cell-based delivery therapy (GT) for the treatment Institute (SCRI) – world leading of Haemophilia B Treatment or Treatment or preclinical & clinical experience missing gene missing gene with Lent
Slide 28: Fc Mimetics and Anti-FcRn mAbs
Fc Mimetics and Anti-FcRn mAbs For personal use only Novel Applications CSL730 Clinical IVIg & SCIg Usage for IgFc Mimetics Development Immuno- Immuno- replacement modulation Surrogate CSL730 is effective in a model of glomerulonephritis* PID Autoimmune & SID disease + - + - CSL / Momenta Responds t
Slide 29: CSL – COVID-19 Vaccines
CSL – COVID-19 Vaccines For personal use only UQ/CSL V451 AZD1222 Partners University of Queensland, AstraZeneca Coalition for Epidemic Preparedness Innovations (CEPI) Vaccine Format Recombinant virus spike protein Adenovirus vector designed to (molecular clamp technology) express spike protein of C
Slide 30: CSL – COVID-19 Vaccines
CSL – COVID-19 Vaccines For personal use only Classical Platforms Next-Gen Platforms AZD1222 7 8 Candidates in clinical development - 42 Candidates in pre-clinical development - 151 4 13 6 UQ/CSL V451 subunit vaccine 2 plus 2 live replicating viral vectors # In Clinical Development Source: van Riel,
Slide 31: CSL – Production of UQ/CSL V451
CSL – Production of UQ/CSL V451 For personal use only CSL Biotech. Manufacturing Facility, Broadmeadows 2000L Cell Culture Harvest by Depth Drug Drug Product Filling, to be Vaccination Filtration Substance Formulated with MF59® • Process scaled up and industrialised from Ph I as required • Productio
Slide 32: Immunology & COVID-19 Response
For personal use only Immunology & COVID-19 Response Mittie Doyle MD Vice President, R&D Immunology CSL Behring Christal: a nurse living with chronic inflammatory demyelinating polyneuropathy (CIDP) 31
Slide 33: For personal use only
For personal use only 1. FOUNDERS 2. MEMBERS 3. CONTRIBUTORS 4. SUPPORTERS 32
Slide 34: Collaborative Hyperimmune Ig Trial in COVID-19
Collaborative Hyperimmune Ig Trial in COVID- 19 For personal use only Placebo + Remdesivir Screen 500 Randomize Day 1 Patients 1:1 H-Ig + Remdesivir Anti-COVID-19 Hyperimmune Globulin (H-Ig) will be provided Day 0 Day 7 Day 28 by four manufacturers Primary Endpoint H-IG Administration Secondary Endp
Slide 35: Hyperimmune Program for Australia
Hyperimmune Program for Australia For personal use only • Convalescent plasma collected by the Australian Red Cross Lifeblood • CSLB has manufactured a clinical batch ready for clinical testing • A single centre, Phase I, study of the Australian H-Ig product in 24 healthy volunteers • Leverage globa
Slide 36: Potential Benefits of Blocking Factor XIIa in COVID-19
Potential Benefits of Blocking Factor XIIa in COVID-19 For personal use only Primary Drivers of ARDS in COVID-19 • Inflammation • Thrombosis Thrombosis Vasodilation, Complement Cytokine • Vascular Permeability vascular permeability activation production ARDS – Acute Respiratory Distress Syndrome 35
Slide 37: Garadacimab in COVID-19
Garadacimab in COVID-19 For personal use only Garadacimab + Supportive Care Screening Dosing Primary endpoint Day -2 to 1 1:1 Day 1 Day 2 Day 7 Day 14 Day 21 Day 28 ICF Placebo + Supportive Care • Population - 124 patients with severe COVID-19 complications • Primary objective - prevent progression
Slide 38: Garadacimab in HAE: The Vanguard Program
Garadacimab in HAE: The Vanguard Program For personal use only Phase II Study Autosomal dominant genetic condition Phase III Study Design 1 in 10,000 – 50,000 people Unregulated protein cascade → elevated levels of bradykinin → fluid release into tissues → swelling in specific parts of body Unpredic
Slide 39: Garadacimab and Factor XIIa in HAE
Garadacimab and Factor XIIa in HAE For personal use only Garadacimab Vascular Permeability Angiodema Landelumab 38
Slide 40: Monthly SC Garadacimab Markedly Reduces Mean HAE Attack Rate
Monthly SC Garadacimab Markedly Reduces Mean HAE Attack Rate (Phase II Study Results) Primary Endpoint For personal use only 5 4.24 Mean Number of HAE Attacks/Month 88.68% 98.94% 90.50% (2.74–5.75) 4 Mean Reduction* Mean Reduction* Mean Reduction* 3 Mean Reduction in HAE Attacks per Month vs Placebo
Slide 41: VANGUARD Garadacimab Pivotal Phase III Study
VANGUARD Garadacimab Pivotal Phase III Study For personal use only Double-Blind, Placebo-Controlled, n=60 Run-in Period Up~13 toweeks 1 month 1-2 ~13months weeks 6 months 200 mg Garadacimab N=36 once monthly SC* 3:2 ≥12 years of age type Baseline attack rate 1 or 2 HAE ≥1 attack/month N=24 Placebo o
Slide 42: HAE in Japan
HAE in Japan For personal use only No ethnic differences No drugs approved for worldwide* long-term prophylaxis Prevalence ~1/50,000, Investigating both 2,400 estimated Garadacimab and patients in Japan; HAE HAEGARDA® for long type 1 (85%), type 2 term prophylaxis (15%) * Source: Zuraw, B. (2010) Wo
Slide 43: Investigating HAEGARDA for HAE in Japan
Investigating HAEGARDA® for HAE in Japan For personal use only Open-label, single-arm Phase III study in ≥ 8 patients with HAE1+2 • Twice-weekly subcutaneous administration of 60 IU/kg HAEGARDA® • Primary Endpoint: HAE attack rate during treatment vs during Run-in period Follow-up Screening Run-in p
Slide 44: Dermatomyositis – a Severe Autoimmune Disease
Dermatomyositis – a Severe Autoimmune Disease For personal use only • Incidence 11 per 1,000,000 • Prevalence rate 14 per 100,000 • Increases with age (peak ages 70-79)* Presents with proximal weakness, characteristic rash and systemic manifestations Mortality rate 10-30% (5y), high comorbidity Curr
Slide 45: RECLAIIM Phase III Study of HIZENTRA in Dermatomyositis
RECLAIIM Phase III Study of HIZENTRA® in Adults with Dermatomyositis For personal use only Weeks 1 to 53 Active DM w/without Steroid rescue if worsening occurs Muscle Weakness Randomization A HIZENTRA® 0.5 g/kg Safety Follow-up HIZENTRA® 0.5 g/kg Telephone Call Screening Forced Steroid taper startin
Slide 46: Commercial
For personal use only Commercial Bill Campbell Executive Vice President and Chief Commercial Officer CSL Behring John, a police officer, and his daughter, Emma, are both living with bleeding disorders. 45
Slide 47: FY20 Highlights
FY20 Highlights Strong underlying demand across the portfolio For personal use only Balanced regional & key market growth New products contributing significantly to growth Sales of $7.7Bn; increased by 8%1 Ig growth well above market Continuing to invest in foundational tools for future growth Succe
Slide 48: FY20: Strong Performance Across the Portfolio
FY20: Strong Performance Across the Portfolio For personal use only 20% growth1in 21% growth1 in revenue YoY; revenue YoY; Continued Growth3 in nearly all growth2 in PID, CIDP launched markets 34% growth1 in revenue YoY and clear SCIg market leader2 New launches in globally Transitioned EU, APAC and
Slide 49: Targeted Protein Therapeutic Market
Targeted Protein Therapeutic Market For personal use only Albumin $4.1B Immunoglobulins $12.8B Total Global Market Value Haemophilia ~$33.5B $11.9B Specialty $4.7B Source: Analyst Reports, Company Annual Reports, data on file; Haemophilia mkt includes Inhibitor mkt 48
Slide 50: Haemophilia
Haemophilia • Transformational product Recombinant Coags +18%1 Sales increased by 8%1 For personal use only • Leadership position in several key markets2 FY20 Sales • Approval of 21-day dosing in EU^, CH, JP & CA • Patient retention strategies and ongoing switches in competitive environment • New ma
Slide 51: AFSTYLA Share of rFVIII Prophylaxis
AFSTYLA® Share of rFVIII Prophylaxis – Growing Steadily For personal use only ® AFSTYLA rFVIII Prophylaxis Patient Share by Country 12% 11% 10% 9% Patient Share (%) 8% 6% 5% 5% 4% 4% 3% 2% 2% 2% 0% Q3 16 Q4 16 Q1 17 Q2 17 Q3 17 Q4 17 Q1 18 Q2 18 Q3 18 Q4 18 Q1 19 Q2 19 Q3 19 Q4 19 Q1 20 Q2 20 German
Slide 52: IDELVION Share of rFIX Prophylaxis
IDELVION® Share of rFIX Prophylaxis – Significant Shares For personal use only ® 70% IDELVION rFIX Prophylaxis Patient Share by Country 59% 60% 58% 57% 50% 44% Patient Share (%) 43% 40% 41% 30% 29% 20% 10% 0% Q1 16 Q2 16 Q3 16 Q4 16 Q1 17 Q2 17 Q3 17 Q4 17 Q1 18 Q2 18 Q3 18 Q4 18 Q1 19 Q2 19 Q3 19 Q
Slide 53: Immunoglobulin Market
Immunoglobulin Market For personal use only Global Ig Volume by Indication Market Dynamics • Market growth above historical rates • Growth in PID & CIDP • Expanding usage for SID • Market supply tightness pre-COVID-19 • COVID-19: Impact on plasma collection • Shifting preference to SCIg and home adm
Slide 54: Immunoglobulins
Immunoglobulins1 Sales increased by 22%2 For personal use only • Increased disease awareness & *IVIg +16%2 FY20 Sales improved diagnosis in chronic therapies (PID & CIDP) • Expansion of SID usage • Launched PID/SID in Japan $4,014 • Market leader SCIg +34%2 • Increased preference for home administra
Slide 55: HIZENTRA: Continued Strong Performance in SCIg
HIZENTRA®: Continued Strong Performance in SCIg Segment For personal use only 80% Share of SCIg Market (Volume) Total 7MM 70% 62% 61% 61% 60% 61% 60% 60% 60% 58% 57% 57% 58% 59% 57% 60% 50% 40% 30% 20% 10% 0% Q1 17 Q2 17 Q3 17 Q4 17 Q1 18 Q2 18 Q3 18 Q4 18 Q1 19 Q2 19 Q3 19 Q4 19 Q1 20 Q2 20 ® Hizen
Slide 56: CSL Behring Well-Positioned in CIDP
CSL Behring Well-Positioned in CIDP For personal use only 40% 35% 30% Patient Share (Total 7MM) 25% 20% 15% 10% 5% 0% Q1 17 Q2 17 Q3 17 Q4 17 Q1 18 Q2 18 Q3 18 Q4 18 Q1 19 Q2 19 Q3 19 Q4 19 Q1 20 Q2 20 CSLB Competitor A Competitor B Competitor C Competitor D Source: Data on file 7MM refers to US, DE
Slide 57: Specialty Products
Specialty Products Sales increased by 10%1 For personal use only FY20 Sales $1,697 Ig Haemophilia Specialty Albumin Other Peri-Operative Other Bleeding +10%1 Specialty +9%1 1. Growth percentages shown at constant currency to remove the impact of exchange rate movements, facilitating comparability of
Slide 58: HAEGARDA Continues to Deliver in the US
HAEGARDA® Continues to Deliver For personal use only in the US HAEGARDA® reduced HAE attacks Prophylactic Almost 25% of by 95%* market** all new patients grew by 25%1 came from newest product Rescue medication launch1 use was reduced by >99%†‡1 Finished with most patients on HAEGARDA® since launch1
Slide 59: Finished with Most Patients on HAEGARDA Since Launch
Finished with Most Patients on HAEGARDA® Since Launch For personal use only Patients Q3 17 Q4 17 Q1 18 Q2 18 Q3 18 Q4 18 Q1 19 Q2 19 Q3 19 Q4 19 Q1 20 Q2 20 ® HAEGARDA Patients Source: Data on file – represents US market only 58
Slide 60: KCENTRA: OAC Market & KCENTRA Utilization
KCENTRA® : OAC Market & KCENTRA® Utilization For personal use only US Clinical practice guidelines recommend KCENTRA® over FFP to reverse the effects of Warfarin* Oral Anticoagulant (OAC) Market FY 19/20 Warfarin Urgent/Major Bleed Reversal Shares Kcentra® Utilization MAT CY Q4’19 Total: 6.9M Patien
Slide 61: Successful Transition of Business Model in China
Successful Transition of Business Model in China For personal use only Obtained the GSP1 license Recruited and transferred all employees to GSP1 Entity Completed in ~1 Year Implemented IT systems, interfaces & training Developed and changed business processes for new affiliate Established direct rel
Slide 62: Commercial Summary
For personal use only Strong underlying demand Executing on strategies across the portfolio Commercial Summary Balanced regional & key New products contributing market growth significantly to growth Aligned therapeutic area Remain flexible and agile teams and strategy managing through COVID-19 61
Slide 63: Transplant
For personal use only Transplant Laurie Lee MD Vice President, R&D Transplant CSL Behring 62
Slide 64: Improve Outcomes for Transplant Recipients
Improve Outcomes for Transplant Recipients For personal use only Unmet needs in hematopoietic stem cell (HSCT) & solid organ transplantation Before & During Transplantation After Transplantation Lack of organs & optimally Inadequate long-term Need for less toxic post matched cells patient and graft
Slide 65: Three Ongoing Late-Phase Transplant Programs
Three Ongoing Late-Phase Transplant Programs For personal use only Inadequate long-term patient and graft survival Graft-versus-Host Disease (GvHD) Antibody-Mediated Rejection (AMR) Phase III Phase II/III Phase III AAT (CSL964) AAT (CSL964) Clazakizumab (CSL300) treatment study in MODULAATE IMAGINE
Slide 66: GvHD: Frequent Post-Transplantation Complication
GvHD: Frequent Post-Transplantation Complication with High Morbidity and Mortality For personal use only Clinical Manifestations Up to 50% of patients develop GvHD after allogeneic HSCT despite current prophylactic regimens Skin GI Liver Of those who develop acute GvHD, only 50% respond to treatment
Slide 67: Potential Mechanisms of AAT in GvHD
Potential Mechanisms of AAT in GvHD For personal use only Pre-Clinical Data AAT 1 • Protease inhibition protects DAMPs 2 tissue Conditioning regimen, Tissue Activation and chemo-therapy damage Chemokines proliferation of or radiation Teff cells • Reduces pro-inflammatory PAMPs APC Naïve T cell 3 cyt
Slide 68: Clinical Response to AAT in Steroid-Refractory acute GvHD
Clinical Response to AAT in Patients with Steroid-Refractory acute GvHD (SR-aGvHD) For personal use only Overall Response Rate (ORR) Treg Fold Change Prospective, open label, Phase II study of i.v. AAT in SR-aGvHD* • 40 subjects, steroid-refractory acute GvHD • AAT twice weekly x 4 weeks at % respon
Slide 69: AAT for GvHD Treatment Study: BMT CTN 1705
AAT for GvHD Treatment Study: BMT CTN 1705 Collaboration opportunity with Blood and Marrow Transplant Clinical Trials Network For personal use only BMT CTN (NHLBI/NCI) AAT IV (120mg/kg) Twice weekly (x 8 doses) + CS Primary If Response: Acute GvHD Endpoint AAT or Randomization (1:1) Placebo (De Novo
Slide 70: MODULAATE AAT GvHD Prevention Phase II/III Study
MODULAATE AAT GvHD Prevention Phase II/III Study For personal use only Part 1: Part 2: Dose Finding – Open Label Randomized Double Blind Placebo Controlled 3 ascending cohorts Cohort 4 (n=15) for each cohort Selected dose from Part 1 (n=260, 1:1; AAT: Placebo) HSCT Adults & adolescents AAT/Placebo H
Slide 71: Solid Organ Transplantation
Solid Organ Transplantation For personal use only Transplants by Organ Type (US - 2015) Other 3% Lung 7% Heart 9% Liver Kidney 23% 58% • >500,000 patients are living with a transplanted kidney* * Scientific Registry of Transplant Recipients (SRTR) and European Renal Association – European Dialysis a
Slide 72: Antibody-Mediated Rejection is a Leading Cause of Long-Term Graft Loss
Antibody-Mediated Rejection (AMR) is a Leading Cause of Long-Term Graft Loss For personal use only Source: Sellarés et al., (2012) Am J Transplant. 12:388-99 Source: Halloran et al., (2015) J Am Soc Hephrol. 26:1711-1720 71
Slide 73: IL-6 Plays a Key Role in the Development of AMR
IL-6 Plays a Key Role in the Development of AMR For personal use only IL-6 Clazakizumab Donor Specific Antibodies IL-6 induces donor-specific antibodies (DSAs) leading to renal tissue damage IL-6 Producing Plasma Cell Plasmablast Anti-inflammatory and immune modulatory Germinal Center IL-6 Clazakizu
Slide 74: IMAGINE: Clazakizumab for chronic AMR treatment study
IMAGINE Clazakizumab for chronic AMR treatment study For personal use only Treatment Phase: Day 1 to Week 260 Dosing with Clazakizumab 12.5 mg SC injection, Q4W (until graft loss or death): N=175 V2 V68 Follow-up Day 1 Wk 260 (monthly) V1 BL EOS assessments assessments Screening Day -42 to -1 Dosing
Slide 75: Improve Outcomes for Transplant Recipients
Improve Outcomes for Transplant Recipients For personal use only Unmet needs in hematopoietic stem cell (HSCT) & solid organ transplantation Before & During Transplantation After Transplantation Lack of organs & optimally Inadequate long-term Need for less toxic post matched cells patient and graft
Slide 76: Summary
For personal use only Summary William Mezzanotte MD Executive Vice President, Head of Research and Development and CMO CSL Behring 75
Slide 77: R&D Portfolio – October 2020
R&D Portfolio – October 2020 For personal use only Registration/ Research Pre-Clinical Phase I Phase II Phase III Post-Registration Gene Therapy CSL888 CSL324 HIZENTRA® HIZENTRA® PRIVIGEN® Treatments Haptoglobin (SAH) Anti-G-CSFR mAb (HS) (SSc) (DM) (PID) JP PID CSL510 CSL730 CSL630 HAEGARDA® IDELVI
Slide 78: Significant Target Launch Dates
Significant Target Launch Dates For personal use only 2019 2020 2021 2022 2023-2025 HIZENTRA® CIDP PRIVIGEN® PID FLUCELVAX® QUAD HAEGARDA® CSL510 Japan Japan 9yrs+ AU Japan Modified Fibrinogen PRIVIGEN® CIDP IDELVION® UQ/CSL V451^ EtranaDez CSL112 Japan 21 Day Dosing (aCov2) Etranacogene ApoA-1 (ACS
Slide 79: R&D Portfolio Highlights – FY21
R&D Portfolio Highlights – FY21 Transplant • CSL964 (AAT) for prevention of GvHD - complete For personal use only Part 1, adaptive phase of study, and advance to confirmatory Part 2 Immunology Respiratory Influenza Vaccines • Garadacimab (Anti-FXIIa) initiate Phase III study • CSL311 (Anti-Beta Comm
Slide 80: Panel Q&A
For personal use only Session Panel Q&A