Csl Limited · ASX:CSL
Csl — Investor Day (December 2012)
101 slides from Csl's investor day presentation, Dec 6, 2012
Slide 1: R&D Briefing
R&D Briefing December 6, 2012
Slide 2: Legal Notice
Legal Notice Forward looking statements The materials in this presentation speak only as of the date of these materials, and include forward looking statements about CSL’s financial results and estimates, business prospects and products in research, all of which involve substantial risks and uncerta
Slide 3: Agenda December 2012 R&D Briefing
Agenda December 2012 R&D Briefing • Welcome Mark Dehring • Introduction & Highlights Andrew Cuthbertson • Immunoglobulins & Specialty Products • Clinical Development Russell Basser • Commercial Opportunities Lutz Bonacker • Q&A • Break • Coagulation/Haemophilia • Introduction & Technical Approach An
Slide 4: Introduction and Highlights
Introduction and Highlights
Slide 5: CSL R&D Strategy
CSL R&D Strategy • Maintain commitment Breakthrough to extracting maximum Medicines value from existing assets and supporting and improving current products Specialty Immunoglobulins Products • Develop new protein-based therapies for treating serious illnesses focusing Haemophilia on products that a
Slide 6: Immunoglobulins Strategy
Immunoglobulins Strategy Maintaining leadership position through focus on: • Patient convenience • Yield • Label Immunoglobulins • Formulation science • Specialty Igs 6
Slide 7: Specialty Products Strategy
Specialty Products Strategy Leveraging high quality, broad product portfolio through: • New markets • Novel indications Specialty • Novel modes of Products administration 7
Slide 8: Haemophilia Strategy
Haemophilia Strategy Supporting and enhancing plasma products and developing novel recombinant portfolio with focus on: • Scientific and product innovation • Patient benefit Haemophilia Products 8
Slide 9: Breakthrough Medicines Strategy
Breakthrough Medicines Strategy Leveraging clinical and technical insight in developing Breakthrough novel protein-based therapies Medicines • Significant unmet need • Multiple indications Optimising value of IP portfolio and assets • Partner high opportunity products 9
Slide 10: Leveraging Global Capabilities
Leveraging Global Capabilities Global project management Recombinant protein manufacturing capabilities 10
Slide 11: R&D Investment
R&D Investment CSL RESEARCH AND DEVELOPMENT INVESTMENT (US$ MILLIONS) 203 226 Mi New Product Development activities focus on innovative new therapies for life-threatening diseases. M8 Market Development strategies seek to bring therapies to new markets and new indications. MS Life Cycle Management e
Slide 12: Global R&D Portfolio December 2011
Global R&D Portfolio December 2011 Immunoglobulins Haemophilia Specialty Products Influenza Vaccine Fibrinogen Fibrinogen Privigen®CIDP EU Biostate® EU Hizentra® US New Indications Aortic Surgery Zemaira® EU Berinert ® Hizentra® EU PCC Self Admin FXIII US Beriplex ® US New Indications Riastap® US Ri
Slide 13: Progress through Stage Gates in 2012
Progress through Stage Gates in 2012 Hizentra rVIIa-FP Hizentra CIDP Canada, EU Berinert rVIII-SC rIX-FP Self Admin Fibrinogen CSL362 Aortic EU Hizentra Berinert Japan Subcut Privigen CIDP CSL112 EU Biostate EU Beriplex US Bleeding 13
Slide 14: Global R&D Portfolio December 2012
Global R&D Portfolio December 2012 Immunoglobulins Haemophilia Specialty Products Influenza Vaccine Privigen® CIDP Hizentra® CIDP Hizentra® US/EU EU Biostate® EU Fibrinogen Berinert ® New Indications Zemaira® EU Beriplex® US Self Admin PCC Fibrinogen Berinert® SC New Indications Aortic EU Novel Plas
Slide 15: Immunoglobulins
Immunoglobulins
Slide 16: Immunoglobulins
Immunoglobulins Maintaining leadership position through focus on: • Patient convenience • Yield • Label Immunoglobulins • Formulation science • Specialty Igs Key Focus • Hizentra® • Privigen® 16
Slide 17: Innovation to Drive Growth
Innovation to Drive Growth • Efficient and competitive cost structure • Ig yield improvements • Product differentiation • Patient convenience • Clinical Use and Indications • Clinical efficacy • Expansion into Neurology • Alzheimer’s Disease opportunity • Prevention of vertical transmission of CMV b
Slide 18: Cytogam
Cytogam® The only registered CMV immunoglobulin in the US indicated for the prevention of CMV disease associated with transplantation • CMV infection is the leading known cause of birth abnormalities in developed countries • Partnership with US National Institutes of Health (NIH) to determine effica
Slide 19: Hizentra
Hizentra® The first 20% high concentration low volume SCIG for convenient self administration providing steady-state Ig levels and an established long-term safety record with chronic administration Global Introductions Continue • Launched in US since 2010 • Broad approvals in EU and Canada • Japan P
Slide 20: Alternate schedules for Hizentra
Alternate schedules for Hizentra® • Hizentra® is indicated weekly for patients with PID • Enhancing patient options through provision of additional schedules • Efficacy expected to be maintained • Safety not expected to be different 20
Slide 21: Chronic Inflammatory Polyneuropathy (CIDP)
Chronic Inflammatory Polyneuropathy (CIDP) • A chronic peripheral nerve disease with progressive muscle weakness and loss of sensation, usually occuring in elderly patients • Most common chronic autoimmune neuropathy 21
Slide 22: Potential benefits of Hizentra in Patients with CIDP
Potential benefits of Hizentra® in Patients with CIDP Current maintenance therapies Oral IVIG Plasma Hizentra® steroids Exchange • Avoids drawbacks of i.v. route • Reduced volume Adverse Less convenience Invasive therapy • Increases patient autonomy effects with long term use Levels show peaks Limit
Slide 23: The PATH Trial: Hizentra in CIDP
The PATH Trial: Hizentra® in CIDP • 150 patients • 2 doses vs placebo • Study approved by FDA, EMA, PMDA • Recruiting in US & EU 23
Slide 24: Privigen
Privigen® The first and only 10% liquid intravenous immunoglobulin (IVIg) therapy that is proline stabilised with room temperature storage up to 36 months Building Capacity to Address Patient Needs Globally • Privigen approved broadly in US, Europe, South America • New Ig manufacturing facility in B
Slide 25: Privigen in CIDP: Rate of Response
Privigen® in CIDP: Rate of Response 100 90 80 70 Responder rate (%) 60 50 40 77% 30 47% 61% 20 10 0 Total IVIg pretreated IVIg untreated
Slide 26: Specialty Products
Specialty Products
Slide 27: Specialty Products
Specialty Products Leveraging high quality, broad product portfolio through: • New markets • Novel indications • Novel modes of Specialty administration Products Key Focus • Beriplex® • Fibrinogen • Zemaira® • Berinert® 27
Slide 28: Beriplex
Beriplex® • Prothrombin Complex Concentrate = PCC • vitamin K-dependent coagulation factors (FII, FVII, FIX, FX) Seeking approval for use of Beriplex® to reverse the effects of vitamin K antagonists for: • Bleeding related to over-anticoagulation • Patients needing surgery 2 large randomised, contro
Slide 29: Fibrinogen
Fibrinogen® The first and only treatment approved by the US FDA for acute bleeding episodes in patients with congenital fibrinogen deficiency Europe • Peri-/post-operative control of coagulopathic bleeding • REPLACE Phase III study • 200 subjects – recruitment commenced Jan 2012 • Aim to complete re
Slide 30: Zemaira
Zemaira® Zemaira is the first highly purified alpha-1 augmentation therapy approved by the FDA for chronic augmentation and maintenance therapy of adults with Alpha-1 and emphysema Seeking to broaden commercial reach through launch in EU, Canada, Brazil • EU requires demonstration of a clinical outc
Slide 31: Berinert
Berinert® Plasma derived, pasteurised & nanofiltered concentrate of C1 Esterase Inhibitor indicated for the treatment of acute abdominal or facial attacks of hereditary angioedema (HAE) in adults and adolescents 31
Slide 32: What Happens to Patients?
What Happens to Patients? • Recurrent episodes of swelling, sometime with a rash • Unpredictable and occur anywhere in the body • Life-threatening if laryngeal swelling • Attacks caused by stress, infection, menstruation, some drugs, unknown causes 32
Slide 33: Berinert
Berinert® Plasma derived, pasteurised & nanofiltered concentrate of C1 Esterase Inhibitor indicated for the treatment of acute abdominal or facial attacks of hereditary angioedema (HAE) in adults and adolescents US and European approved label expansion for self administration of HAE • As part of US
Slide 34: Overcoming Challenges in Long-term Prophylaxis of HAE Attacks
Overcoming Challenges in Long-term Prophylaxis of HAE Attacks Current prophylactic therapies Oral Intravenous High concentration androgens C1-INH subcutaneous (sc) Berinert® • Limited by • Inconvenience • Avoids drawbacks of i.v. adverse and risks of • Low volume for s.c. administration effects, rep
Slide 35: Berinert Subcutaneous Prophylaxis Program
Berinert® Subcutaneous Prophylaxis Program • Study ongoing in US and Germany - due to complete 1H Safety and 2013 pharmacokinetic study • Select safe and efficacious dosing for clinical efficacy trial - due to commence 2H 2013 35
Slide 36: Commercial Opportunities and Activities
Commercial Opportunities and Activities
Slide 37: Immunoglobulins
Immunoglobulins
Slide 38: The Immunoglobulin Market is attractive
The Immunoglobulin Market is attractive The 2012 IG market is a > $6 B opportunity • Market includes IVIG, SCIG and Hyperimmunes CSL • Growing Market • CSL is well positioned 38
Slide 39: CSL's Immunoglobulin Portfolio
CSL’s Immunoglobulin Portfolio Specific IG • Globalise portfolio SCIG • Expand into neurology Privigen • Increase convenience IVIG Other $US1,722M 39
Slide 40: Ig Portfolio Positioning
Ig Portfolio Positioning First and only Proline-stabilised IVIG • High purity 10% liquid IVIG • Optimal dimer formulation throughout shelf life for improved tolerability First and only 20% Proline-stabilised SCIG • Low administration volume increases efficiency • Convenient – few sites & fast infusi
Slide 41: Globalise Portfolio
Globalise Portfolio Privigen & Hizentra (PL) Privigen & Hizentra (HU) Privigen (BR) Privigen (TW) Privigen & Hizentra (CZ) Privigen (TR) Privigen (MY) Privigen (SG) Privigen (MX) Privigen (RU) Hizentra (BR) Hizentra (TR) Hizentra (AR) Privigen (HK) Hizentra (JAP) Hizentra (MX) 2012 2013 2014 2015 •
Slide 42: Expand into Neurology
Expand into Neurology Hizentra 20% Privigen 10% CIDP CIDP (EU) (EU, CH, CAN) Hizentra 20% CIDP (US) 2012 2013 2014 2015 2016+ • Strengthen presence in neurological segment • Launch Privigen® in CIDP in the EU • Develop Hizentra® in CIDP in the US, the EU and RoW 42
Slide 43: Increase Convenience
Increase Convenience Privigen 40g Presentation (US, CH, EU) Hizentra Convenience Initiatives Hizentra 10g (US, EU) 2012 2013 2014 2015 • Increase dosing flexibility • Differentiate through convenience launches: • 10 g vial Hizentra® • 40 g vial Privigen® • Ongoing convenience initiatives 43
Slide 44: Infusion Steps
Clean surface Sanitize your hands Inspect each vial of Hizentra Gather your supplies Prepare syringe Infusion Steps 4 preparation steps per vial Prepare syringe Reducing number of vials (continued) increases convenience Write in your journal Prepare syringe (continued) Start infusion 44 Prepare inje
Slide 45: Cytogam
Cytogam® The only registered CMV immunoglobulin in the US indicated for the prevention of CMV disease associated with transplantation Center for Disease Control1: Potential Opportunity • CMV is the most common viral infection that infants are born with Assuming Screening at week 23 in the United Sta
Slide 46: Specialty Products
Specialty Products
Slide 47: Specialty Products
Specialty Products Wound Healing Other Specialty Products Tachocomb Beriplast Kybernin Berinert Zemaira Fibrogammin Streptase Beriplex Perioperative Bleeding $US618 M Riastap • Increase clinical data set • Add indications • Expand regionally 47
Slide 48: Beriplex
Beriplex® In the US 3.8 M patients are on warfarin1 with a major bleeding rate of 3.1-3.6%2 per year • In US launch in warfarin reversal indication • US surgical indication: Patients requiring emergency surgery needing urgent reversal of warfarin • Understand the use in Factor Xa inhibitor reversal
Slide 49: Blood Products vs. Concentrates
Blood Products vs. Concentrates FFP Fibrinogen concentration at ≈2.3g / L Not virus inactivated Frozen, requires time (<50 minutes) to thaw Red Blood Cells Need to be matched to blood type Not virus inactivated Concentrated, virus inactivated, room temperature storage, Fibrinogen concentration Plate
Slide 50: Fibrinogen
Fibrinogen® • Obtain WEU & US acquired label • Initiate acquired label expansion • Generate, publish & communicate data 50
Slide 51: Bring to Europe
Bring to Europe Pivotal trial objective: Zemaira slows the progression of emphysema Demonstration of a clinical outcome (disease modification) • Publish data in 2013: • American Thoracic Society • European Respiratory Society • Recognition in treatment guidelines • Demonstrate economic benefit • Rei
Slide 52: Berinert
Berinert® Berinert treats the fundamental cause of HAE symptoms by providing C1- INH deficient patients with the missing human protein1. Berinert has demonstrated that it provides fast relief of pain and swelling within 30 minutes2 • Obtain Prophylaxis indication • Increase convenience with s.c. tre
Slide 53: Berinert - Current Life Cycle Activities
Berinert® - Current Life Cycle Activities • Low-volume, high Product improvement concentration, formulation • Short-term prophylaxis, Expand 2013 (EU/Can) indication • Further initiatives ongoing New method of • Prophylaxis Indication use with s.c. formulation 53
Slide 54: Q&A
Q&A
Slide 55: Break
Break
Slide 56: Haemophilia Products
Haemophilia Products
Slide 57: Haemophilia
Haemophilia Supporting and enhancing plasma products and developing novel recombinant portfolio with focus on: • Scientific and product innovation • Patient benefit Key Focus Haemophilia • Long acting rIX-FP Products • Long acting rVIIa-FP • rVIII-Single Chain • Research into long acting rVWF-FP 57
Slide 58: Innovation to Drive Growth
Innovation to Drive Growth • Patient convenience primary Scientific Edge driver of innovation • Albumin fusion technology Improved half life, rAlbumin Precise engineering extended as fusion of specially • rIX-FP, rVIIa-FP, rVWF-FP dosing platform designed interval linker • Factor VIII High VWF affin
Slide 59: rIX-FP (CSL654)
59 =, PROLONG 9 FP rlX-FP (CSL654) CSL
Slide 60: rIX-FP (CSL654) Clinical Program
rIX-FP (CSL654) Clinical Program PUP study Phase Phase 1/2 Phase 2/3 Phase 3 Paediatric 1 PK PK PK Long-term safety Long-term safety 7d prophylaxis 7-14d prophylaxis Extension safety On-demand On-demand Surgical prophylaxis 60
Slide 61: Compared with in market rFIX
Compared with in market rFIX • 5.3-fold longer half-life (92hrs) • ~ 45% higher incremental recovery • ~7-fold larger AUC • ~7-fold slower clearance 61
Slide 62: Efficacy of rIX-FP in Phase 1/2 Trial
Efficacy of rIX-FP in Phase 1/2 Trial • 13 subjects treated weekly for up to 48 weeks • previously on prophylaxis no increase in weekly FIX consumption • switched from on-demand to weekly prophylaxis >90% reduction in bleeding rate • Subjects treated on-demand (85 bleeds) • 88% of episodes contr
Slide 63: rIX-FP (CSL654) Safety Data
rIX-FP (CSL654) Safety Data • Excellent safety profile in completed studies • Well tolerated • No inhibitors • No adverse events related to CSL654 63
Slide 64: rIX-FP (CSL654) Further Development
rIX-FP (CSL654) Further Development • Enrolment of Phase 2/3 study due to be completed early 2013 • Paediatric study has commenced • Prolonged half life exploring treatment intervals longer than every second week 64
Slide 65: rVIIa-FP (CSL689)
rVila-FP (CSL689) PROLONG 7 Fre CSL
Slide 66: Development of rVIIa-FP (CSL689)
Development of rVIIa-FP (CSL689) • Phase 1 in 40 healthy volunteers • First-in-man dose escalation study in healthy volunteers completed • No SAEs, one related mild AE • Pivotal Phase 2/3 Trial in Hemophilia A & B patients with Inhibitors • Dose finding, Safety and Efficacy on-demand therapy • Compl
Slide 67: Phase 1 Study of rVIIa-FP in Healthy Volunteers
Phase 1 Study of rVIIa-FP in Healthy Volunteers Lines represent different doses of CSL689 (no placebo) Half-life 3-4 fold longer than rFVIIa 67
Slide 68: Potential of rVIIa-FP (CSL689)
Potential of rVIIa-FP (CSL689) • For patients with inhibitors • Single dose for treatment of bleeding • Prevention of bleeding in patients undergoing surgery • Prophylaxis • Other indications • Congenital Factor VII deficiency • Acquired hemophilia • Glanzmann's thrombasthenia 68
Slide 69: rVIII-SingleChain (CSL627)
rVIII-SingleChain (CSL627) 69
Slide 70: rVIII-SingleChain: approach for improved FVIII
rVIII-SingleChain: approach for improved FVIII • FVIII’s physiological partner in plasma is von Willebrand factor (vWF) • FVIII/vWF complex is important role in the physiological activity and clearance of FVIII • Aim - improve binding to VWF • FVIII is an unstable molecule in the manufacturing envir
Slide 71: rVIII-SingleChain: high affinity for vWF
rVIII-SingleChain: high affinity for vWF Binding to plasma-derived (pd) VWF Comparison of VWF affinity constants 250 20 18 200 Affinity constant (1/(nm) 16 14 Relative units 150 12 10 100 8 6 4 50 2 Single chain rFVIII (CSL627) Full length rFVIII 0 rVIII-SingleChain Full length rFVIII 0 (CSL627) 0 5
Slide 72: rVIII-SingleChain: PK profiles in rodents
rVIII-SingleChain : PK profiles in rodents PK in haemophilia A mice PK in rats 5 6,000 FVIII:C plasma level (IU/mL) 5,000 4 FVIII:Ag (mIU/mL) 200 IU/kg FVIII:C 100 IU/kg FVIII:C 4,000 n=3/timepoint 3 geometric means ± SD n=5/timepoint 3,000 geometric means ± SD 2 2,000 1 1,000 0 0 0 20 40 60 80 0 12
Slide 73: rVIII-SingleChain Phase 1/3 Study Design
rVIII-SingleChain Phase 1/3 Study Design Interim analysis Planned Dec 2012 Part 1 Part 2 CSL627 & Octagog alfa CSL627 repeat-dose, on-demand or Extension study single-dose PK (n=30) prophylaxis (n=30) Part 3 CSL627 repeat-dose, on-demand or prophylaxis (n=78 evaluable subjects) Single-dose PK rVIII-
Slide 74: Recombinant Coagulation Portfolio Summary
Recombinant Coagulation Portfolio Summary Target Launch Dates CSL654 (rIX-FP) • Pivotal Phase II/III study commenced 2015 • Phase I data demonstrate >5x half life extension • Orphan drug status granted by US FDA CSL627 (rVIII-SingleChain) 2016 • Phase I/III trial commenced • Early clinical data supp
Slide 75: Commercial Opportunities and Activities
Commercial Opportunities and Activities
Slide 76: Coagulation Sales
Coagulation Sales Helixate pd Coag $US1,058M • Launch rIX-FP in 2015 • Launch rVIII-SingleChain as bio-better in 2016 • Grow pd FVIII 76
Slide 77: Coagulation: Total Market Size
Coagulation: Total Market Size Key Market Segments and Products • Beriate • Helixate • Factor X P Hem A FX P ~ $5B ~ $3M pd ITT management • Biostate Inhibitor bleed ~ $300M treatment Target • Haemate P ~ $1B Segments • Mononine VWD Hem B • Berinin • Biostate ~$500M ~$700M • Haemate P 77 CSL Market
Slide 78: Coagulation: Key Market Segments and Products
Coagulation: Key Market Segments and Products. • Beriate • Helixate NexGen/FS/81 • Factor X P Hem A • rVIII-Single ~ $5B Chain FX P ~ $ 3M pd ITT management • Biostate • rVIIa-FP Inhibitor bleed ~ $300M treatment Target • Haemate P ~ $1B Segments • Mononine • Biostate EU VWD Hem B • Berinin • Haemat
Slide 79: Recombinant Coagulation Portfolio
Recombinant Coagulation Portfolio 2012/13 & • Differentiate recombinant albumin fusion platform and launch rIX-FP • Differentiate and launch rVIII-SingleChain • Strong support for Helixate and growth of rVIII-SingleChain 79
Slide 80: rIX-FP (CSL654) & rVIIa-FP (CSL689) Scientific Edge
rIX-FP (CSL654) & rVIIa-FP (CSL689) Scientific Edge Improved half Recombinant Specifically life, extended Albumin as designed dosing fusion linker interval partner 80
Slide 81: rIX-FP: The Scientific Edge
rIX-FP: The Scientific Edge Phase 1 data rIX-FP 1 rFIX- PEGylated 2 rFIX-Fc fusion 3 (CSL Behring – Albumin Fusion) Half life x 5.3 x5 ~ x3 extension vs rFIX • Half life supports dosing every 2+ weeks 1 Santagostino et al, Blood. 2012; 120 (12): 2405 – 2411 2 Negrier et al., Blood 2011, 118(10): 269
Slide 82: rVIIa-FP: The Scientific Edge
rVIIa-FP: The Scientific Edge T/2 extension rVIIa-FP (CSL Behring-Albumin Fusion) Phase 1 being analysed Half life vs rFVIIa x 3-4 • The only half life extension technology currently in clinical development which enhances the duration of effect of native rFVIIa • Half life supports single dose manag
Slide 83: rVIII-SingleChain (CSL627)
rVIII-SingleChain (CSL627) Me++ A1 A2 B A3 C1 C2 Commercial Covalent bond Scientific Edge Edge Improved Improved High VWF financial molecular affinity contribution stability Opportunity for Extended Dosing Interval 83
Slide 84: Presenting Data: Active Scientific Presence
Presenting Data: Active Scientific Presence 30 25 20 15 10 5 0 Albumin Fusion rVIIa-FP rVIII-SingleChain rIX-FP Technology Abstract/Poster Publication 84
Slide 85: Breakthrough Medicines
Breakthrough Medicines
Slide 86: Breakthrough Medicines
Breakthrough Medicines Leveraging clinical and technical insight in developing Breakthrough novel protein-based therapies Medicines • Significant unmet need • Multiple indications Key Focus • CSL112 (Apo AI) • CSL362 (anti-IL-3R mAb) • CSL346 (anti-VEGF-B mAb) 86
Slide 87: CSL112 (Apolipoprotein A-I)
CSL112 (Apolipoprotein A-I) • CSL112 is natural apolipoprotein A-I (apoA-I) the chief protein component of HDL • Rapidly and robustly enhances capacity of plasma to promote cholesterol efflux • Global Phase 2b development program to initiate mid-2013 • Potential to address significant gap in acute c
Slide 88: CSL362 (anti-IL-3Ra mAb)
CSL362 (anti-IL-3Ra mAb) • Initial indication: Acute myeloid leukaemia • Enhanced recruitment of tumour killing NK cells • Targeting patients in CSL362 - Improved killing of patient leukaemia cells with autologous NK cells remission with high risk of relapse • Phase I trial in progress at 4 sites •
Slide 89: CSL346 (anti-VEGF-B mAb)
CSL346 (anti-VEGF-B mAb) • Type 2 Diabetes • Fat accumulation within tissues leads to insulin resistance and failure to control blood glucose • Most patients progress to insulin dependence CSL346 treatment preserves b cell • VEGF-B controls fat uptake into tissues insulin production in diabetic mice
Slide 90: Licensing and Collaborations
Licensing and Collaborations
Slide 91: Licensing
Licensing Optimising value of IP Portfolio and assets Breakthrough Breakthrough Medicines Medicines • Partner high opportunity products • GARDASIL® Specialty Immunoglobulins Specialty Immunoglobulins Products • Mavrilimumab (GM- Products CSFRa - Medi/AZ) Haemophilia • Periodontal disease Products (S
Slide 92: GARDASIL
GARDASIL® • Impact of Australian HPV Vaccination Program • 93% reduction in genital warts in females less than 21 years • Adolescent male funding • Extension to Australian NIP to include 12-13 yr males, with 2 year catch up for Year 9 males, from 2013 • Long term protection • No break through diseas
Slide 93: Mavrilimumab (GM-CSFRa)
Mavrilimumab (GM-CSFRa) Phase II EARTH Study completed • 264 subjects with moderate-to-severe RA • bi-weekly dosing for 12weeks (10, 30, 50, 100mg) • Primary endpoint • DAS28-CRP decrease >1.2 at wk12 • Secondary endpoints • DAS28-CRP remission • ACR20/50/70 & HAQ-DI • Safety profile Outcomes • rapi
Slide 94: Mavrilimumab
Mavrilimumab Safety profile - Most Common Adverse events (≥3%) 94
Slide 95: Mavrilimumab
Mavrilimumab Safety profile – serious adverse events 95
Slide 96: ISCOMATRIX Adjuvant Partnering Activities
ISCOMATRIX® Adjuvant Partnering Activities • Major partners continue to advance vaccine development programs • Merck Research Laboratories initiated Dengue clinical study • Strong neutralising antibodies against all 4 serotypes in NHPs even at low antigen doses 10000 LiCor 50 Microneut. Titers Salin
Slide 97: Summary
Summary
Slide 98: Global R&D Portfolio December 2012
Global R&D Portfolio December 2012 Immunoglobulins Haemophilia Specialty Products Influenza Vaccine Hizentra® CIDP Privigen® CIDP Hizentra® US/EU Biostate® EU Fibrinogen Berinert ® New Indications Zemaira® EU Beriplex® US Self Admin PCC Fibrinogen Berinert® SC New Indications Aortic EU Novel Plasma
Slide 99: Expected Progress in next 12 Months
Expected Progress in next 12 Months rVWF-FP rVIIa-FP Zemaira EU Beriplex Beriplex P gingivalis Japan Surgery US Partnered vaccine Privigen CIDP programs EU Berinert EU rVIII-SC Prophylaxis Berinert Beriplex SC Bleeding US 99
Slide 100: Significant Short-Midterm Target Launch Dates
Significant Short-Midterm Target Launch Dates >2016 2013 2014 2015 2016 2017 CSL654 CSL627 CSL689 rIX-FP rFVIII rVIIa-FP Berinert® Beriplex® US Beriplex® US SubCut EU Berinert® Bleeding Surgical SubCut US Fibrinogen EU Aortic Surgery Zemaira® EU Privigen® CIDP Hizentra® Hizentra® EU Japan CIDP Core
Slide 101: Q&A
Q&A