Slide 1: R&D Briefing
For personal use only R&D Briefing December 8, 2011
Slide 2: Disclaimer
Disclaimer For personal use only 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 substanti
Slide 3: Agenda December 2011 R&D Briefing
Agenda December 2011 R&D Briefing 8.30am: Sign in and coffee For personal use only • Welcome Mark Dehring • Introduction & Highlights Andrew Cuthbertson • Immunoglobulins • Specialty Bleeding Products • Clinical Development Russell Basser • Commercial Opportunities Ingolf Sieper • Q&A and Break • Co
Slide 4: Introduction and Highlights
For personal use only Introduction and Highlights
Slide 5: CSL R&D Strategy
CSL R&D Strategy • Maintain commitment For personal use only to extracting maximum Breakthrough Medicines value from existing assets and supporting and improving current products Specialty Immunoglobulins Products • Develop new protein-based therapies for treating serious illnesses focusing Haemophi
Slide 6: Immunoglobulins Strategy
Immunoglobulins Strategy For personal use only Supporting and enhancing current portfolio • Patient convenience • Yield • Label Immunoglobulins • Formulation science 6
Slide 7: Specialty Products Strategy
Specialty Products Strategy For personal use only Expanding use of specialty plasma products through: • New markets Specialty • Novel indications Products • Novel modes of administration 7
Slide 8: Haemophilia Strategy
Haemophilia Strategy For personal use only Supporting and enhancing portfolio and developing new products • Plasma products • Recombinant analogs • Coagulation research Haemophilia Products 8
Slide 9: Breakthrough Medicines Strategy
Breakthrough Medicines Strategy For personal use only Developing new protein-based therapies Breakthrough Medicines • Significant unmet need • Multiple indications 9
Slide 10: Licensing Strategy
Licensing Strategy For personal use only Optimise value of IP Portfolio and assets Breakthrough Medicines • Partner high opportunity products Specialty Immunoglobulins Products • Continue broad licensing strategy for ISCOMATRIX® adjuvant Haemophilia Products 10
Slide 11: Leveraging Global Capabilities
Leveraging Global Capabilities For personal use only 11
Slide 12: R&D Investment
12 For personal use only R&D Investment
Slide 13: Global R&D Pipeline December 2010
Global R&D Pipeline For personal use only December 2010 Humate® P Zemaira® US Privigen® Rhophylac® US Afluria® Fibrinogen Fibrinogen Privigen®CIDP EU Hizentra® EU Hizentra® US New Indications Aortic Surgery Zemaira® EU Beriplex® EU Berinert® US PCC Beriplex ® US Riastap® US New Indications Riastap®
Slide 14: Progress through Stage Gates in 2011
Progress through Stage Gates in 2011 For personal use only Hizentra rVIIa-FP Privigen CIDP EU Fibrinogen G-CSF Aortic EU FXIII US Riastap VEGFB Biostate EU EU rFVIII Berinert Self Admin CSL112 rvWF-FP 14
Slide 15: Global R&D Portfolio December 2011
Global R&D Portfolio December 2011 For personal use only 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 Indi
Slide 16: Immunoglobulins
For personal use only Immunoglobulins
Slide 17: Immunoglobulins
Immunoglobulins Supporting and enhancing For personal use only current portfolio • Patient convenience • Yield • Label • Formulation science Immunoglobulins Key Focus • Hizentra® • Privigen® 17
Slide 18: Hizentra
Hizentra® For personal use only 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 • In 2011, broader app
Slide 19: Privigen
Privigen® For personal use only 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 • IgLab Module2 on-line increasing capacity • Privigen approved in US,
Slide 20: Specialty Products
For personal use only Specialty Products
Slide 21: Specialty Products
Specialty Products Expanding use of For personal use only specialty plasma products e.g. • Berinert® • Beriplex® Specialty • Riastap® Products • Zemaira® Key Focus • Acquired bleeding • Perioperative bleeding 21
Slide 22: Correcting problems that lead to bleeding
For personal use only Correcting problems that lead to bleeding
Slide 23: Acquired Bleeding Disorders
Acquired Bleeding Disorders • Coagulation factor deficiencies can occur because of drugs, surgery, For personal use only trauma, liver disorders, other diseases • Current treatment options • Traditional blood products – platelets, fresh frozen plasma (FFP), cryoprecipitate • Specific factor concentr
Slide 24: Fibrinogen for Complex Cardiac Surgery
For personal use only Fibrinogen (Riastap / Haemocomplettan®) for Complex Cardiac Surgery
Slide 25: Fibrinogen in cardiopulmonary bypass surgery
Fibrinogen in cardiopulmonary bypass surgery For personal use only • Coagulation factors are consumed bleeding 25
Slide 26: Hannover proof-of-concept study in aortic repair
Hannover proof-of-concept study in aortic repair For personal use only • Fibrinogen reduced proportion of patients requiring transfusion Administration of Proportion of subjects donated blood Fibrinogen Placebo products (N = 29) (N = 31) No 45% 0% Yes 55% 100% p<0.0001 26
Slide 27: Complex Cardiac Surgery
Complex Cardiac Surgery For personal use only 27
Slide 28: Fibrinogen Development
Fibrinogen Development • Europe / Global For personal use only • Peri-/post-operative control of coagulopathic bleeding • REPLACE Phase III study • 200 subjects – sites initiated December 2011 • Aim to file label extension H2 2013 • US • Coagulopathic bleeding related to complex cardiac surgery • Do
Slide 29: Beriplex to Reverse Anti-coagulation
For personal use only Beriplex® to Reverse Anti-coagulation
Slide 30: Challenges with anti-coagulation
Challenges with anti-coagulation For personal use only • Anti-coagulants used to prevent clotting for people who are at risk • Vitamin K antagonists (ie warfarin) - most commonly prescribed • New generation products now being approved around the world • Specific for FXa, FIIa • Potential problems •
Slide 31: What is Beriplex?
What is Beriplex®? For personal use only • Prothrombin Complex Concentrate = PCC • vitamin K-dependent coagulation factors (FII, FVII, FIX, FX) • 2 viral inactivation steps • Specific antidote to vitamin K antagonists • new anti-coagulants? • Used in Europe for >10 years with excellent safety record
Slide 32: Program to licence Beriplex in US
Program to licence Beriplex® in US For personal use only • 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, controlled clinical trials • Bleeding study completed • Sur
Slide 33: Effects of Beriplex on specific clotting factor levels
Effects of Beriplex® on specific clotting factor levels in Bleeding Study For personal use only Factor X Beriplex (n=98) level in Plasma (n=104) patients Time 33
Slide 34: Effects of Beriplex on bleeding test
Effects of Beriplex® on bleeding test in Bleeding Study For personal use only INR Beriplex (n=98) in patients Plasma (n=104) Time 34
Slide 35: Potential of Beriplex for bleeding related to new anti-coagulants
Potential of Beriplex® for bleeding related to new anti-coagulants For personal use only Preclinical data – rat bleeding model 35
Slide 36: Globalisation of Perioperative Bleeding (POB)
For personal use only Globalisation of Perioperative Bleeding (POB)
Slide 37: Surgical or Coagulopathic Bleeding?
Surgical or Coagulopathic Bleeding? For personal use only 37
Slide 38: What is the Challenge? BOF Syndrome!
For personal use only What is the Challenge? BOF Syndrome! 38
Slide 39: BOF Syndrome
BOF Syndrome For personal use only BLOOD ON FLOOR 39
Slide 40: Coagulation Cascade
Coagulation Cascade For personal use only Intrinsic System Extrinsic System PL XII XI VII TTPL IX VIII X V Stabilized through FXIII II Fibrinogen Fibrin 40
Slide 41: Treatment Paradigm in Central Europe
Treatment Paradigm in Central Europe For personal use only • Given the order of factor deficiency in coagulopathic bleeding • First: Fibrinogen deficiency • First line – RiaStap when confirmed fibrinogen deficiency • Second: Impaired thrombin generation • First line – Beriplex P/N after fibrinogen n
Slide 42: Short-Term Strategy to Accelerate Global Penetration of POB
Short-Term Strategy to Accelerate the Global Penetration of POB For personal use only • Actively participate in ISICEM, WCA, EACTA, ESA • Post partum hemorrhage guideline roundtables • Grow in regions • Dedicated sales force • Education 100 roundtables • Expand web presence • ISICEM supported medica
Slide 43: Fibrinogen Registration Status
Fibrinogen Registration Status 2011/12 For personal use only Acquired Indication Austria, Brazil, Bulgaria, Czech Republic, Germany, Hungary, Iran, Israel, Kuwait, Netherlands, Portugal, Romania, Switzerland, Taiwan, Turkey Congenital Indication Australia, Belgium, Denmark, Finland, France, Iceland,
Slide 44: Target Fibrinogen Registration Status
Target Fibrinogen Registration Status 2015/16 For personal use only Acquired Indication Argentina, Austria, Australia, Belgium, Brazil, Bulgaria, Canada, Czech Republic, Denmark, Finland, France, Germany, Hungary, Iceland, Iran, Ireland, Israel, Italy, Kuwait, Netherlands, Norway, Portugal, Romania,
Slide 45: Beriplex
For personal use only Beriplex®
Slide 46: Main Indications for Beriplex/PCC
Main Indications for Beriplex®/PCC For personal use only Anti-Coagulant Reversal (ACR) 6% 4% Bleeding caused by 8% coagulopathy other than ACR 9% 50% Liver disease 10% Postoperative bleeding 13% Dilution coagulopathy (e.g. trauma) DIC Vit. K deficiency Source: CSLB CEU PCC Market Research 2009 46
Slide 47: Indications for Beriplex US
Indications for Beriplex® US For personal use only • Two indications 1. Bleeding • For the treatment of acute major bleeding resulting from an acquired deficiency of vitamin K-dependent coagulation factors and proteins C and S due to Vitamin K antagonist (warfarin) therapy 2. Surgery • For the repla
Slide 48: Primary Target Customer Segments
Primary Target Customer Segments For personal use only Need safe, cost effective treatments that rapidly ER Physicians, Nurses resolve acute bleeding episodes Need treatments that satisfy hospital physicians needs Transfusion Medicine while helping them effectively manage the utilisation of Blood Ba
Slide 49: Beriplex Registration Status 2012
Beriplex® Registration Status 2012 For personal use only registered/launched Argentina, Australia, Austria, Belgium, Brazil, Canada, Finland, France, Germany, Great Britain, Greece, Hong Kong, Hungary, Italy, Luxembourg, Malaysia, Netherlands, Norway, Poland, Portugal, Spain, Sweden, Switzerland, Ta
Slide 50: Summary Commercial Opportunity POB
Summary Commercial Opportunity POB • Perioperative Bleeding For personal use only Specialty Critical Care • PCC: Beriplex® FY11 $367m • Fibrinogen: Riastap®/Haemocomplettan® • FXIII: Fibrogammin®/Corifact® Kybernin / • Treatment paradigm for POB has shifted Berinert POB in Central Europe 60% • Rest
Slide 51: Q&A
For personal use only Q&A
Slide 52: Break
For personal use only Break
Slide 53: Haemophilia Products
For personal use only Haemophilia Products
Slide 54: Haemophilia
Haemophilia Supporting and enhancing portfolio and developing new For personal use only products • Plasma products • Recombinant analogs • Coagulation research Key Focus Haemophilia • Long acting rIX-FP Products • Long acting rVIIa-FP • rVIII-Single Chain • Research into long acting rvWF-FP 54
Slide 55: Coagulation Cascade
Coagulation Cascade Not all products are licensed in all countries For personal use only Beriate Helixate NexGen CSL627 + CSL689 Haemate P Humate-P Beriplex P/N Biostate Confidex DDAVP Berinin P Factor X P Behring Mononine CSL654 Haemocomplettan P Riastap Fibrogammin P Corifact 55
Slide 56: Innovations in Coagulation
Innovations in Coagulation rIX-FP For personal use only rVIIa-FP Me++ A1 A2 A3 C1 C2 rVIII-SingleChain 56
Slide 57: Albumin Fusion Technology
Albumin Fusion Technology For personal use only Proof of Concept for complex proteins established Long half-life with understood clearance mechanism Albumin Abundant, fusion natural proteins carrier, low expressed as immunog. a single entity potential 57
Slide 58: Recombinant Albumin Fusion Proteins
Recombinant Albumin Fusion Proteins For personal use only • rIX-FP (CSL654) • Recombinant fusion protein linking coagulation factor IX with albumin • rVIIa-FP (CSL689) • Recombinant fusion protein linking coagulation factor VIIa with albumin • Rational design to optimise efficacy • Designed to maint
Slide 59: Development of rIX-FP, CSL654
Development of rIX-FP, CSL654 For personal use only • Completed discussions with EMA, PEI and FDA • Agreement for clinical program and criteria for licensure • Paediatric Investigational Plan agreed • Phase I PK study completed and results to be presented at GTH* in February • Phase I/II prophylaxis
Slide 60: rIX-FP has a half life ~3x longer than BeneFIX in dogs
rIX-FP has a half life ~3x longer than BeneFIX in haemophilia B dogs For personal use only Mean values and fitted curves 1 CSL654 100 IU/kg (n=3) 0.50 BeneFIX® 100 IU/kg (n=2) Faxtor IX antigen (IU/mL) 0.20 0.10 0.05 0.02 0.01 0.005 0 1 2 3 4 5 6 7 8 Days after administration 60
Slide 61: rIX-FP Activity in Haemophilia B Patients
rIX-FP Activity in Haemophilia B Patients For personal use only rIX-FP rFIX pdFIX FIX activity Time 61
Slide 62: rVIIa-FP is active with half life ~6 times longer
rVIIa-FP is active and has a half life ~6 times longer than FVIIa For personal use only 100 % of initial dose 10 1 rFVIIa rVIIa-FP 0.1 0 2 4 6 8 10 12 14 16 18 20 22 24 Time (hours) Weimer et. al. rVIIa albumin fusion rVIIa-FP has an improved half-life in rat model 62
Slide 63: Recombinant Albumin Fusion Proteins Summary
Recombinant Albumin Fusion Proteins Summary • Significant half-life extension demonstrated For personal use only • Pre-clinical studies in rats, rabbits and monkeys • rIX-FP ~3 to 4-fold • rVIIa-FP ~8 to 10-fold • Both proteins designated Orphan Medicinal Products in Europe • Stage of development •
Slide 64: rVIII-SingleChain: approach for improved FVIII
rVIII-SingleChain: approach for improved FVIII For personal use only • FVIII’s physiological partner in plasma is von Willebrand factor (VWF) • The FVIII/VWF complex plays an important role in the physiological activity and clearance of FVIII • Aim: Improve binding to VWF • FVIII is an unstable mole
Slide 65: rVIII-SingleChain (CSL627)
rVIII-SingleChain (CSL627) • Increased heavy/light chain stability For personal use only • Covalent linkage between heavy and light chain • Expressed as a recombinant single-chain FVIII • Enhanced molecular integrity • Very strong affinity to von Willebrand factor (VWF) • Faster and more efficient b
Slide 66: Short-term reconstituted stability at 25°C
Short-term reconstituted stability at 25°C 110 For personal use only FVIII activity (CS method) 100 compared to t= 0 (%) 90 pd FVIII 80 full-length rFVIII single-chain rFVIII 70 0 1 2 3 4 5 6 7 8 66 time (days)
Slide 67: rVIII-SingleChain: high affinity for vWF
rVIII-SingleChain: high affinity for vWF For personal use only Binding to plasma-derived (pd) VWF Comparison of VWF affinity constants 250 20 18 200 16 Affinity constant (1/(nm) 14 Relative units 150 12 10 100 8 6 4 50 2 Single chain rFVIII (CSL627) Full length rFVIII 0 rVIII-SingleChain Full length
Slide 68: rVIII-SingleChain: PK properties in mice and rats
rVIII-SingleChain: PK properties in mice and rats For personal use only PK in haemophilia A mice PK in rats 5 6,000 FVIII plasma level (IU/mL) 5,000 4 FVIII:Ag (mIU/mL) 4,000 3 100 IU/kg FVIII:C 3,000 2 n=5/timepoint 2,000 1 1,000 0 0 0 20 40 60 80 0 120 240 360 480 Hours Minutes rVIII-SingleChain (
Slide 69: rVIII-SingleChain: PK parameters in cynomolgus monkeys
rVIII-SingleChain: PK parameters in cynomolgus monkeys For personal use only Single chain Full length rFVIII (CSL627) rFVIII (250 IU/kg) (250 IU/kg) AUC (h*IU/mL) 101.7 67.2 Cmax (IU/mL) 10.7 11.19 Clearance (mL/kg/h) 2.0 3.4 T½ β (h) 9.7 6.8 CSL Behring. Data on file 69
Slide 70: Development of rVIII-SingleChain, CSL627
Development of rVIII-SingleChain, CSL627 For personal use only Progress to date • Completed discussions with EMA, PEI and FDA • Agreement for phase I/III clinical program and criteria for licensure • Paediatric Investigational Plan agreed • Toxicology completed • Phase I study sites initiated • Phas
Slide 71: rVIII-SingleChain: Summary
rVIII-SingleChain: Summary For personal use only Novel single chain rFVIII molecule which demonstrates: • Very high binding affinity to VWF • Improved stability after reconstitution • Comparable haemostatic efficacy to full length rFVIII in FVIII deficient mice Advanced status of development • Pharm
Slide 72: Commercial Opportunities and Activities
For personal use only Commercial Opportunities and Activities
Slide 73: Haemophilia General Market Trends
Haemophilia General Market Trends For personal use only • Market growth • $7-8Bn market and growing • The rate of prophylaxis in adults and children is increasing • Weight gain across the population increases the weekly dose • Increased life expectancy of people with haemophilia leads to higher use
Slide 74: CSL Haemophilia Portfolio Overview
CSL Haemophilia Portfolio Overview For personal use only FVIII/ FIX Inhibitors Haemophilia Haemophilia VWD A B Bleed ITT Management Plasma-derived Beriate® Mononine® Biostate® - Haemate® P Monoclate-P® Berinin® Haemate® P Biostate® Humate-P® Recombinant Helixate® Recombinant Bio-Better Recombinant H
Slide 75: CSL Haemophilia Portfolio Overview
CSL Haemophilia Portfolio Overview For personal use only FVIII/ FIX Inhibitors Haemophilia Haemophilia VWD A B Bleed ITT Management Plasma-derived Beriate® Mononine® Biostate® - Haemate® P Monoclate-P® Berinin® Haemate® P Biostate® Humate-P® Recombinant Helixate® Recombinant CSL627 Bio-Better rVIII-
Slide 76: Status of Haemophilia A Products
Status of Haemophilia A Products For personal use only • Growing Market Total market size in IU FVIII only (excl. VWD) 7.3BIU ($5.04B) • CSL market leader in plasma derived products • Significant opportunity to grow our own rFVIII CSL Behring rec. FVIII CSL Behring pd. FVIII 76
Slide 77: Status of Haemophilia B Products
Status of Haemophilia B Products For personal use only • Anticipated $1Bn market in Total market size in IU 1.1BIU ($0.84B) next 4-5 years • In longer term 2/3 of market likely to shift to half life extended products • CSL has the potential to be a CSL Behring pd. FIX major player 77
Slide 78: Status of Haemophilia Inhibitor Bleeding: rFVIIa
Status of Haemophilia Inhibitor Bleeding: rFVIIa For personal use only • Only bleeding management Total market size $1.05Bn • Most of the market likely to shift to extended half life products 100% • CSL is developing a potentially very competitive rFVIIa Market product 78
Slide 79: Product Features
For personal use only Product Features
Slide 80: CSL654 (rIX-FP) Features
CSL654 (rIX-FP) Features For personal use only Attribute Ideal TPP* CSL654 (rIX-FP) Range of vials Wide Human protein in manufacturing/ No formulation process? Animal protein in purification process? No Half life extension technology Low immunogenicity PK: Half life extension > three fold
Slide 81: CSL689 (rVIIa-FP) Features
CSL689 (rVIIa-FP) Features For personal use only Attribute Ideal TPP CSL689 (rVIIa-FP) Range of vials Wide Animal / Human protein in manufacturing/ No formulation process? Half life extension technology Low immunogenicity PK: Half life extension > 6 fold Cell line CHO Reconstitution volum
Slide 82: CSL627 (rVIII-SingleChain) Features
CSL627 (rVIII-SingleChain) Features For personal use only Attribute Ideal TPP CSL627 Range of vials Wide Human protein in manuf. / formulation proc? No Animal protein in purification proc? No Chain One Binding to VWF High affinity BDD Yes (yield) PK: Half life extension 3 fold extension
Slide 83: Advantages of Albumin as a Fusion Partner
Advantages of Albumin as a Fusion Partner For personal use only Proof of Concept for complex proteins established Long half- life with understood clearance mechanism Albumin Abundant, fusion natural proteins carrier, low expressed immunog. as a single potential entity 83
Slide 84: CSL654 & CSL689 Scientific Edge
CSL654 (rIX-FP) & CSL689 (rVIIa-FP) For personal use only Scientific Edge Improved Precise half life, rAlbumin as engineering extended fusion of specially dosing platform designed interval linker 84
Slide 85: Scientific Edge: rIX-FP Prophylactic Dosing Interval
Scientific Edge: rIX-FP Prophylactic Dosing Interval For personal use only • Standard dosing 2 3 x per week MON TUE WED THU FRI SAT SUN MON TUE WED • CSL654 dosing 1 x per 1 2 week MON TUE WED THU FRI SAT SUN MON TUE WED 85
Slide 86: Scientific Edge: rVIIa-FP Prophylactic Dosing Interval
Scientific Edge: rVIIa-FP Prophylactic Dosing Interval For personal use only • Standard dosing MON TUE WED THU FRI SAT SUN MON TUE • CSL689 dosing 2 3 x per week MON TUE WED THU FRI SAT SUN MON TUE 86
Slide 87: CSL627 (rVIII-SingleChain)
CSL627 (rVIII-SingleChain) Me++ A1 A2 A3 C1 C2 Covalent bond For personal use only Commercial Scientific Edge Edge Improved Improved High VWF financial molecular affinity contribution stability Opportunity for Extended 87 Dosing Interval
Slide 88: Scientific Edge: rVIII Prophylactic Dosing Interval
Scientific Edge: rVIII Prophylactic Dosing Interval For personal use only • Standard dosing MON TUE WED THU FRI SAT SUN MON TUE bleeding risk • Alternative dosing TUE FRI TUE morning evening morning • CSL627 will be tested in both dosing regimens 88
Slide 89: Rec. Coag Portfolio Summary
Rec. Coag Portfolio Summary For personal use only 2015 • CSL654 (rIX-FP) – Recombinant fusion protein genetically linking coagulation factor IX with recombinant human albumin 2016 • CSL627 (rVIII-SingleChain) – Recombinant single - chain factor VIII 2017 • CSL689 (rVIIa-FP) – Recombinant fusion prot
Slide 90: Sharing our Results
Sharing our Results For personal use only 90
Slide 91: Summary Commercial Opportunities
Summary Commercial Opportunities Haemophilia Products For personal use only • $7-8Bn market and growing • Significant upside potential by entering new markets • New CSL product portfolio meets/exceeds the “ideal” TPP • “Albumin” technology provides a strong competitive edge • CSL has one of the most
Slide 92: Licensing and Collaborations
For personal use only Licensing and Collaborations
Slide 93: Licensing
Licensing Optimise value of IP Portfolio and assets For personal use only Breakthrough • Partner high opportunity products Medicines • GARDASIL® IP • GM-CSFRa (Medi/AZ) Specialty Immunoglobulins Products • Periodontal disease (Sanofi) • Continue broad licensing strategy for ISCOMATRIX® adjuvant Haem
Slide 94: GARDASIL
GARDASIL® Impact of Australian HPV Vaccination Program For personal use only • Significant reduction in genital warts and high grade abnormalities1,2 Long term protection • Studies indicate no break through disease 6 & 7 yrs post immunisation Male clinical data • Efficacy against external genital le
Slide 95: CAM3001 / Mavrilimumab
CAM3001 / Mavrilimumab For personal use only Rheumatoid arthritis • common chronic inflammatory disease of the joints • market opportunity – DMARD / biological DMARD inadequate responders CAM3001 • fully human mAb targeting the GM-CSFRa • licensed to MedImmune / AstraZeneca • CSL to receive mileston
Slide 96: Mavrilimumab Phase IIa Study
Mavrilimumab Phase IIa Study For personal use only • 3 mnth sequential ascending Proportion of subjects achieving a change of 1.2 dose study, dosing every 2nd wk from baseline DAS28-CRP • 233 subjects with active RA DAS28-CRP Responders (%) • Mavrilimumab showed a rapid (< 2 wks) and significant cli
Slide 97: ISCOMATRIX Adjuvant Partnering Activities
ISCOMATRIX® Adjuvant Partnering Activities • Major partners continue to advance vaccine For personal use only development programs using ISCOMATRIX® adjuvant • Merck • Additional field Value • Progressing new program into clinic • Pfizer Broad Licenses Exclusive Manufacture • Program in GLP tox stud
Slide 98: NIH Multi-site CMV Trial
NIH Multi-site CMV Trial For personal use only • CMV infection is the leading known cause of birth abnormalities in developed countries • 1-2% of pregnant women are infected with CMV • Total live births per year with CMV disease in US >6,000 • Partnership with US National Institutes of Health (NIH)
Slide 99: Summary
For personal use only Summary
Slide 100: Global R&D Portfolio December 2011
Global R&D Portfolio December 2011 For personal use only 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 Indi
Slide 101: Expected Progress in next Months
Expected Progress in next 12 Months For personal use only Hizentra rIX-FP ROW rVIIa-FP Hizentra CIDP CSL362 Privigen CIDP Berinert Biostate SC EU CSL112 Beriplex US Hi 101
Slide 102: Short to Mid-term Target Launch Dates
Short to Mid-term Target Launch Dates For personal use only >2016 2012 2013 2014 2015 2016 Biostate CSL654 CSL627 EU rIX-FP rFVIII Beriplex® US Fibrinogen Zemaira® Berinert® Bleeding Aortic Surgery EU SC Berinert® Beriplex® US Self Admin Surgical Hizentra® Privigen®CIDP Hizentra® EU EU CIDP Core Cap
Slide 103: Q&A
For personal use only Q&A