R&D Briefing December 8, 2011

Disclaimer

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 uncertainties, many of which are outside the control of, and are unknown to, CSL. You can identify these forward looking statements by the fact that they use words such as “anticipate,” “estimate,” “expect,” “project,” “intend,” “plan,” “believe,” “target,” “may,” “assume,” and other words and terms of similar meaning in connection with any discussion of future operating or financial performance. Factors that could cause actual results to differ materially include: the success of research and development activities, decisions by regulatory authorities regarding whether and when to approve our products as well as their decisions regarding labeling and other matters that would affect the commercial potential of our products; competitive developments affecting our products; the ability to successfully market new and existing products; difficulties or delays in manufacturing; trade buying patterns and fluctuations in interest and currency exchange rates; legislation or regulations that affect product production, distribution, pricing, reimbursement or access; litigation or government investigations, including legal costs, settlement costs and the risk of adverse decisions or settlements; and CSL’s ability to protect its patents and other intellectual property. The statements being made in this presentation do not constitute an offer to sell, or solicitation of an offer to buy, any securities of CSL.

No representation, warranty or assurance (express or implied) is given or made in relation to any forward looking statement by any person (including CSL). In particular, no representation, warranty or assurance (express or implied) is given in relation to any underlying assumption or that any forward looking statement will be achieved. Actual future events may vary materially from the forward looking statements and the assumptions on which the forward looking statements are based. Given these uncertainties, readers are cautioned to not place undue reliance on such forward looking statements.

Subject to any continuing obligations under applicable law or any relevant listing rules of the Australian Securities Exchange, CSL disclaims any obligation or undertaking to disseminate any updates or revisions to any forward looking statements in these materials to reflect any change in expectations in relation to any forward looking statements or any change in events, conditions or circumstances on which any such statement is based. Nothing in these materials shall under any circumstances create an implication that there has been no change in the affairs of CSL since the date of these materials.

Agenda December 2011 R&D Briefing

8.30am: Sign in and coffee

Welcome
Introduction & Highlights

Mark Dehring Andrew Cuthbertson

Immunoglobulins
Specialty Bleeding Products
Clinical Development

Russell Basser

Commercial Opportunities

Ingolf Sieper

Q&A and Break
Coagulation/Haemophilia
New Product Development

Val Romberg

Commercial Opportunities

Ingolf Sieper

Licensing

Andrew Cuthbertson

Summary
Q&A

Noon: Finish

Introduction and Highlights

CSL R&D Strategy

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Breakthrough
Medicines
Specialty
Immunoglobulins
Products
Haemophilia
Products
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Maintain commitment to extracting maximum value from existing assets and supporting and improving current products
Develop new protein-based therapies for treating serious illnesses focusing on products that align with our technical and commercial capabilities

Immunoglobulins Strategy

Supporting and enhancing current portfolio

Patient convenience
Yield
Label

Immunoglobulins

Formulation science

Specialty Products Strategy

Expanding use of specialty plasma products through:

New markets

Specialty Products

Novel indications
Novel modes of administration

Haemophilia Strategy

Supporting and enhancing portfolio and developing new products

Plasma products
Recombinant analogs
Coagulation research

Haemophilia Products

Breakthrough Medicines Strategy

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Breakthrough
Medicines
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Developing new protein-based therapies

Significant unmet need
Multiple indications

Licensing Strategy

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Breakthrough
Medicines
Specialty
Immunoglobulins Products
Haemophilia
Products
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Optimise value of IP Portfolio and assets

Partner high opportunity products
Continue broad licensing strategy for ISCOMATRIX[®] adjuvant

Leveraging Global Capabilities

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R&D Investment

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Global R&D Pipeline

December 2010

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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
New Indications Beriplex ® US Riastap® US
Biostate® EU Riastap® EU
Rec Coagulation CSL689 rVIIa-FP
Factors
CSL654 rIX-FP
Novel Plasma
Proteins
Vaccines– Merck Vaccines– Merck Vaccines– Merck Partnered Vaccine
Vaccines – Pfizer Programs
Vaccines– Pfizer
Vaccines– Abbott
P gingivalis POD CSL425
CRC-OHS/Sanofi * 2009 H1N1 Flu
CSL112 CAM3001
CSL401
reconstituted HDL GM-CSFR - AZ
H5N1 Flu
CSL362 IL-3R
CSL324 G-CSFR
Discovery
Projects IL-13R
Core Capabilities Plasma Proteins Haemophilia Specialty Products Breakthrough Medicines Vaccines & IP
Partnered Projects
13
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Progress through Stage Gates in 2011

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Hizentra
rVIIa-FP Privigen CIDP
EU
Fibrinogen
G-CSF FXIII US
Aortic EU
Riastap
VEGFB
Biostate EU EU
rFVIII Berinert
Self Admin
CSL112
rvWF-FP
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Global R&D Portfolio December 2011

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Immunoglobulins
Haemophilia
Specialty Products
Influenza Vaccine
Fibrinogen Fibrinogen Privigen®CIDP EU Biostate® EU Hizentra® US
New Indications Aortic Surgery
Zemaira® EU Berinert ® Hizentra® EU
New Indications PCC Beriplex ® US Self Admin FXIII US
Riastap® US
Riastap® EU
Novel Plasma CSL689 rVIIa-FP
Proteins CSL654 rIX-FP
rvWF-FP CSL627 rFVIII
Rec Coagulation
Factors
Partnered Vaccine Partnered Vaccine Partnered Vaccine Partnered Vaccine
Programs Programs Programs Programs
P gingivalis POD CSL112 CAM3001
CRC-OHS/Sanofi * reconstituted HDL GM-CSFR - AZ
CSL362 IL-3R
CSL324 G-CSFR
VEGFB
Discovery
Projects IL-13R
Core Capabilities Immunoglobulins Haemophilia Specialty Products Breakthrough Medicines Vaccines & IP
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*Partnered Projects

LCM includes direct post marketing commitments as well as pathogen safety, capacity expansions, yield improvements, new packages and sizes for all registered products

Immunoglobulins

Supporting and enhancing current portfolio

Patient convenience
Yield
Label

Immunoglobulins

Formulation science

Key Focus

Hizentra[®]
Privigen[®]

Hizentra[®]

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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 approvals in EU and Canada
Japan Phase III licensing study complete

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Exploring Chronic Use of Hizentra in Immunomodulation

Pursuing SCIG for the treatment of CIDP
Anticipated study initiation in 2012

Privigen[®]

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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, Europe, South America with additional country registrations underway

Strengthening Presence in Neurology Market

Phase III study in CIDP conducted in Europe
LPLV Completed Nov 2011
Anticipate filing in EU in Q2 2012

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Specialty Products

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Specialty
Products
21
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Expanding use of specialty plasma products e.g.

Berinert[®]
• Beriplex[®]
• Riastap[®]
• Zemaira[®]
Key Focus • Acquired bleeding • Perioperative bleeding

Correcting problems that lead to bleeding

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Acquired Bleeding Disorders

Coagulation factor deficiencies can occur because of drugs, surgery, trauma, liver disorders, other diseases
Current treatment options
Traditional blood products – platelets, fresh frozen plasma (FFP), cryoprecipitate
Specific factor concentrates such as those in CSL portfolio
Limitations with traditional approaches
Sensitivity reactions
Large volume
Time taken to administer
Storage not straightforward
Consume a lot of donated blood
Limited lifespan

Fibrinogen (Riastap / Haemocomplettan[®] ) for Complex Cardiac Surgery

Fibrinogen in cardiopulmonary bypass surgery


Coagulation factors are consumed bleeding

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Hannover proof-of-concept study in aortic repair

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

Complex Cardiac Surgery

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Fibrinogen Development

Europe / Global
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
Dose-finding required by FDA
Considering options for broader indication

Beriplex[®] to Reverse Anti-coagulation

Challenges with anti-coagulation

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
Bleeding can occur related to excessive anti-coagulation
Need to urgently reverse if trauma, surgery immediately required
~3M people currently on warfarin in the US
- ~100K patients in need of urgent reversal annually

What is Beriplex[®] ?

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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
Current program
Expand geographical usage
Evaluate potential for correcting bleeding due to new anti-

coags

Program to licence Beriplex[®] in US

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
Surgical study due to be completed mid 2012
BLA submission planned for Q1 2012

Effects of Beriplex[®] on specific clotting factor levels in Bleeding Study

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Factor X Beriplex (n=98)
level in Plasma (n=104)
patients
Time
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Effects of Beriplex[®] on bleeding test in Bleeding Study

INR Beriplex (n=98) in patients Plasma (n=104) Time

Potential of Beriplex[®] for bleeding related to new anti-coagulants

Preclinical data – rat bleeding model

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Globalisation of Perioperative Bleeding (POB)

Surgical or Coagulopathic Bleeding?

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What is the Challenge?

BOF Syndrome!

BOF Syndrome

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BLOOD
ON
FLOOR
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Coagulation Cascade

Intrinsic System

Extrinsic System

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PL XII XI VII TTPL
VIII
IX
X
V
Stabilized through FXIII
II
Fibrinogen Fibrin
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Treatment Paradigm in Central Europe

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 normalisation and
- confirmation of impaired thrombin generation
Subsequently: Impairment of fibrinolysis post-
- surgery
- First line –Fibrogammin >12 – 24 hours post-surgery

Short-Term Strategy to Accelerate the Global Penetration of POB

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 medical education
Allaboutbleeding website

Fibrinogen Registration Status 2011/12

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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, Ireland, Italy, Norway, Slovakia, Spain, Sweden, United Kingdom,
USA
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Target Fibrinogen Registration Status 2015/16

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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, Spain, Russia, Slovakia,
Sweden, Switzerland, Taiwan, Turkey,
Congenital Indication USA , Mexico
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Beriplex[®]

Main Indications for Beriplex[®] /PCC

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8% [6% 4%]
9%
50%
10%
13%
Source: CSLB CEU PCC Market Research 2009
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Anti-Coagulant Reversal (ACR) Bleeding caused by coagulopathy other than ACR Liver disease Postoperative bleeding Dilution coagulopathy (e.g. trauma) DIC Vit. K deficiency

Indications for Beriplex[®] US

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 replacement of vitamin K-dependent coagulation factors and proteins C and S in patients on Vitamin K antagonist (warfarin) requiring emergency surgery or invasive intervention

Primary Target Customer Segments

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 Bankers / Hematologists blood products Need cost effective treatments to manage pharmacy Pharmacists budget while satisfying the needs of hospital physicians

Beriplex[®] Registration Status 2012

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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, Taiwan,
United States
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Summary Commercial Opportunity POB

Perioperative Bleeding

Specialty Critical Care FY11 $367m

PCC: Beriplex[®]
Fibrinogen: Riastap[®] /Haemocomplettan[®]
FXIII: Fibrogammin[®] /Corifact[®]

Kybernin / Berinert

POB 60%

Treatment paradigm for POB has shifted in Central Europe
Rest of Europe has started to follow
In terms of registration we are 3+ years ahead
Growth Potential POB
~20% per annum mid-term

Q&A

Break

Haemophilia Products

Haemophilia

Supporting and enhancing portfolio and developing new products

Plasma products
Recombinant analogs
Coagulation research

Key Focus

Haemophilia Products

Long acting rIX-FP
Long acting rVIIa-FP
rVIII-Single Chain
Research into long acting rvWF-FP

Coagulation Cascade

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Not all products are licensed in all countries
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
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Innovations in Coagulation

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rIX-FP
rVIIa-FP
Me [++]
A1 A2 A3 C1 C2
rVIII-SingleChain
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Albumin Fusion Technology

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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
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Recombinant Albumin Fusion Proteins

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 maintain molecular activity
Optimised to minimise immunogenicity
Demonstrated by in vitro / in vivo testing

Development of rIX-FP, CSL654

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 and on-demand study recruitment complete
Phase II/III study commencing early 2012

*German Society of Thrombosis and Haemostasis

rIX-FP has a half life ~3x longer than BeneFIX in haemophilia B dogs

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Mean values and fitted curves
1 CSL654 100 IU/kg (n=3)
0.50 BeneFIX® 100 IU/kg (n=2)
0.20
0.10
0.05
0.02
0.01
0.005
0 1 2 3 4 5 6 7 8
Days after administration
Faxtor IX antigen (IU/mL)
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rIX-FP Activity in Haemophilia B Patients

~~rIX-FP rFIX pdFIX~~

FIX activity

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Time
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rVIIa-FP is active and has a half life ~6 times longer than FVIIa

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100
10
1
rFVIIa rVIIa-FP
0.1
0 2 4 6 8 10 12 14 16 18 20 22 24
Time (hours)
% of initial dose
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Weimer et. al. rVIIa albumin fusion

 rVIIa-FP has an improved half-life in rat model

Recombinant Albumin Fusion Proteins Summary

Significant half-life extension demonstrated
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
rIX-FP: Phase I PK trial complete and data being evaluated Presentation of Phase I data at GTH* Congress Feb 2012 Phase I/II on-going
rVIIa-FP: Pharmacology and toxicology studies completed
rvWF-FP : Research program for long acting vWF initiated

*German Society of Thrombosis and Haemostasis

rVIII-SingleChain: approach for improved FVIII

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 molecule in the manufacturing environment
Potential for dissociation which leads to loss of procoagulant activity of FVIII ~~[[++]]~~

~~Me[[++]]~~ A1 A2 A3 C1 C2 rVIII-SingleChain

Aim: Improve molecular stability
Appropriate molecule originated by SK Chemicals

rVIII-SingleChain (CSL627)

Increased heavy/light chain stability
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 binding to VWF

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Me [++]
A1 A2 A3 C1 C2
Covalent Bond
Wild Type FVIII rVIII-SingleChain
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Short-term reconstituted stability at 25°C

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110
100
90
pd FVIII
80
full-length rFVIII
single-chain rFVIII
70
0 1 2 3 4 5 6 7 8
66 time (days)
compared to t= 0 (%)
FVIII activity (CS method)
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rVIII-SingleChain: high affinity for vWF

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Binding to plasma-derived (pd) VWF Comparison of VWF affinity constants
250
20
18
200
16
14
150
12
10
8
100
6
4
50
2
Single chain rFVIII (CSL627)
Full length rFVIII 0
rVIII-SingleChain Full length rFVIII
0
(CSL627)
0 500 1,000 1,500
Surface plasmon resonance (SPR) analysis Time (s)
CSL Behring. Data on file
Relative units
Affinity constant (1/(nm)
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rVIII-SingleChain: PK properties in mice and rats

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PK in haemophilia A mice PK in rats
5 6,000
5,000
4
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
Minutes
Hours
rVIII-SingleChain (CSL627)
Full length rFVIII
CSL Behring. Data on file
FVIII:Ag (mIU/mL)
FVIII plasma level (IU/mL)
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rVIII-SingleChain: PK parameters in cynomolgus monkeys

	Single chain rFVIII (CSL627) (250 IU/kg)	Full length rFVIII (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
T½β (h)	9.7	6.8

CSL Behring. Data on file

Development of rVIII-SingleChain, CSL627

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
Phase III component to commence within 15 months

rVIII-SingleChain: Summary

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

Pharmacology and toxicology studies completed
Animal PK studies show promising results
• Phase I/III clinical trial sites initiated

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CSL Behring. Data on file

Commercial Opportunities and Activities

Haemophilia General Market Trends

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 in surgery and age related complications
- Geographic expansion
Therapy type
Treatment categories remain “plasma derived” and “recombinant”
Within recombinant segment a category with improved PK parameters will establish itself. This opens an opportunity for differentiation

CSL Haemophilia Portfolio Overview

	Haemophilia A	Haemophilia B	VWD	FVIII/ FIX Inhibitors	FVIII/ FIX Inhibitors
				Bleed Management	ITT
Plasma-derived	Beriate® Monoclate-P®	Mononine® Berinin®	Biostate® Haemate® P Humate-P®	-	Haemate® P Biostate®
Recombinant	Helixate®
Recombinant Bio-Better
Recombinant Half life extended

CSL Haemophilia Portfolio Overview

	Haemophilia A	Haemophilia B	VWD	FVIII/ FIX Inhibitors	FVIII/ FIX Inhibitors
				Bleed Management	ITT
Plasma-derived	Beriate® Monoclate-P®	Mononine® Berinin®	Biostate® Haemate® P Humate-P®	-	Haemate® P Biostate®
Recombinant	Helixate®
Recombinant Bio-Better	CSL627 rVIII-SC
Recombinant Half life extended		CSL654 rIX-FP	Research rvWF-FP	CSL689 rVIIa-FP

Status of Haemophilia A Products

Growing Market

Total market size in IU FVIII only (excl. VWD) 7.3BIU ($5.04B)

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CSL market leader in plasma derived products
Significant opportunity to grow our own rFVIII

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CSL Behring rec. FVIII CSL Behring pd. FVIII

Status of Haemophilia B Products

Total market size in IU 1.1BIU ($0.84B)

Anticipated $1Bn market in next 4-5 years

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CSL Behring pd. FIX

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In longer term 2/3 of market likely to shift to half life extended products
CSL has the potential to be a major player

Status of Haemophilia Inhibitor Bleeding: rFVIIa

Only bleeding management

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100%
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Total market size $1.05Bn

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rFVIIa Market

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Most of the market likely to shift to extended half life products
CSL is developing a potentially very competitive product

Product Features

CSL654 (rIX-FP) Features


Attribute	Ideal TPP*	CSL654 (rIX-FP)
Range of vials	Wide	
Human protein in manufacturing/ formulation process?	No	
Animal protein in purification process?	No	
Half life extension technology	Low immunogenicity	
PK: Half life extension	> three fold	
PK: Recovery	> 1.2 (IU/mL)/(IU/kg)	
Cell line	CHO	
Reconstitution volume	Low	
Reconstitution device	Simple, needle-free	

*Target Product Profile

CSL689 (rVIIa-FP) Features

SL689 (rVIIa-FP) Features	SL689 (rVIIa-FP) Features	SL689 (rVIIa-FP) Features

Attribute	Ideal TPP	CSL689 (rVIIa-FP)
Range of vials	Wide	
Animal / Human protein in manufacturing/ formulation process?	No	
Half life extension technology	Low immunogenicity	
PK: Half life extension	> 6 fold	
Cell line	CHO	
Reconstitution volume	Low	
Reconstitution device	Simple, needle-free	

*Target Product Profile

CSL627 (rVIII-SingleChain) Features

SL627 (rVIII-SingleChain) Features	SL627 (rVIII-SingleChain) Features	SL627 (rVIII-SingleChain) Features

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	Approx. 1.3 times
Cell line	CHO	
Immunogenicity risk	Low	
Reconstitution volume	Low	
Reconstitution device	Simple, needle-free	

*Target Product Profile

Advantages of Albumin as a Fusion Partner

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Proof of
Concept for
complex
proteins
established
Long half-
life with
understood
clearance
Albumin
mechanism
Abundant, fusion
natural proteins
carrier, low expressed
immunog. as a single
potential entity
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CSL654 (rIX-FP) & CSL689 (rVIIa-FP)

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Scientific Edge
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Precise
rAlbumin as engineering
fusion of specially
platform designed
linker
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Improved half life, extended dosing interval

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Scientific Edge: rIX-FP Prophylactic Dosing Interval

• 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

Scientific Edge: rVIIa-FP Prophylactic Dosing Interval

• 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

CSL627 (rVIII-SingleChain)

~~Me[++]~~ A1 A2 A3 C1 C2

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Covalent bond
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Commercial
Scientific Edge
Edge
Improved Improved
High VWF
financial molecular
affinity
contribution stability
Opportunity for Extended
Dosing Interval
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Scientific Edge: rVIII Prophylactic Dosing Interval

• 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

Rec. Coag Portfolio Summary

2015

CSL654 (rIX-FP)
Recombinant fusion protein genetically linking coagulation factor IX with recombinant human albumin

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2016

CSL627 (rVIII-SingleChain)

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Recombinant single - chain factor VIII
2017
CSL689 (rVIIa-FP)
Recombinant fusion protein genetically linking coagulation VIIa with recombinant human albumin

Sharing our Results

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Summary Commercial Opportunities Haemophilia Products

$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 experienced commercial organisations in the coagulation business, with a proven track record in Haemophilia and other coagulation related disorders like POB

Licensing and Collaborations

Licensing

Optimise value of IP Portfolio and assets

Breakthrough Medicines Specialty Immunoglobulins Products

Partner high opportunity products
GARDASIL[®] IP
GM-CSFRa (Medi/AZ)
Periodontal disease (Sanofi)
Continue broad licensing strategy for ISCOMATRIX[®] adjuvant

Haemophilia Products

GARDASIL[®]

Impact of Australian HPV Vaccination Program

Significant reduction in genital warts and high grade abnormalities[1,2]
Long term protection
Studies indicate no break through disease 6 & 7 yrs post immunisation
Male clinical data
Efficacy against external genital lesions established
Efficacy against anal disease under review by the TGA (approved in US & Canada)
V503: 9-Valent HPV Vaccine
Pivotal efficacy study completed (N=14,000)
Anticipated global regulatory filings mid 2012

1 Donovan et al, Lancet Nov 2010 2. Brotherton et al, Lancet 2011

CAM3001 / Mavrilimumab

Rheumatoid arthritis

common chronic inflammatory disease of the joints
market opportunity – DMARD / biological DMARD
inadequate responders

CAM3001

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a
fully human mAb targeting the GM-CSFR
licensed to MedImmune / AstraZeneca
CSL to receive milestones and royalties
phase II study commenced in 2010
study results presented at ACR meeting, October 2011

Mavrilimumab Phase IIa Study

3 mnth sequential ascending dose study, dosing every 2nd wk

Proportion of subjects achieving a change of 1.2 from baseline DAS28-CRP

233 subjects with active RA
Mavrilimumab showed a rapid (< 2 wks) and significant clinical effect compared with placebo, especially in the higher (100 mg) dose cohort
Clinical activity maintained at 12 wks

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Weeks
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p<0.05; p<0.01
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Short-term safety profile to support further clinical development

ISCOMATRIX[®] Adjuvant Partnering Activities

Major partners continue to advance vaccine development programs using ISCOMATRIX[®] adjuvant
Merck
Additional field
into clinic
Pfizer
Program in GLP tox study
~40 fields across all partners

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Value
Broad Exclusive
Licenses Manufacture
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NIH Multi-site CMV Trial

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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) to determine efficacy of CMV immunoglobulin in preventing mother to baby transmission

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Large multi-site clinical trial involving >150,000 women commencing December 2011
CSL donating Cytogam[®] , the only registered CMV immunoglobulin in the US.
Primary analysis expected 2016

Summary

Global R&D Portfolio December 2011

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*Partnered Projects

LCM includes direct post marketing commitments as well as pathogen safety, capacity expansions, yield improvements, new packages and sizes for all registered products

100

Expected Progress in next 12 Months

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Hizentra
rIX-FP
ROW
rVIIa-FP Hizentra
CIDP
CSL362
Privigen CIDP
Berinert Biostate
SC EU
Beriplex
CSL112
US
Hi
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101

Short to Mid-term Target Launch Dates

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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 Capabilities Immunoglobulins Haemophilia Specialty Products Breakthrough Medicines Vaccines & IP
Partnered Projects
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*Calendar Years

102

AI assistant

CSL Ltd. — Investor Presentation 2011

17854_rns_2011-12-07_e25ebbb5-d1f4-4ab6-a7ab-997cd272cf04.pdf

R&D Briefing December 8, 2011

Disclaimer

Forward looking statements

Agenda December 2011 R&D Briefing

8.30am: Sign in and coffee

Introduction and Highlights

CSL R&D Strategy

Immunoglobulins Strategy

Specialty Products Strategy

Haemophilia Strategy

Breakthrough Medicines Strategy

Licensing Strategy

Leveraging Global Capabilities

R&D Investment

﻿ ﻿ Global R&D Pipeline

December 2010

Progress through Stage Gates in 2011

﻿ Global R&D Portfolio ﻿ December 2011

LCM includes direct post marketing commitments as well as pathogen safety, capacity expansions, yield improvements, new packages and sizes for all registered products

Immunoglobulins

Immunoglobulins

Hizentra[®]

Global Introductions Continue

Privigen[®]

Building Capacity to Address Patient Needs Globally

Strengthening Presence in Neurology Market

Specialty Products

Specialty Products

Acquired Bleeding Disorders

Fibrinogen in cardiopulmonary bypass surgery

Hannover proof-of-concept study in aortic repair

Complex Cardiac Surgery

Fibrinogen Development

• US

Beriplex[®] to Reverse Anti-coagulation

Challenges with anti-coagulation

What is Beriplex[®] ?

Program to licence Beriplex[®] in US

Effects of Beriplex[®] on specific clotting factor levels in Bleeding Study

Effects of Beriplex[®] on bleeding test in Bleeding Study

Potential of Beriplex[®] for bleeding related to new anti-coagulants

Globalisation of Perioperative Bleeding (POB)

Surgical or Coagulopathic Bleeding?

What is the Challenge?

BOF Syndrome!

BOF Syndrome

Coagulation Cascade

Intrinsic System

Extrinsic System

Treatment Paradigm in Central Europe

Short-Term Strategy to Accelerate the Global Penetration of POB

Fibrinogen Registration Status 2011/12

Target Fibrinogen Registration Status 2015/16

Main Indications for Beriplex[®] /PCC

Indications for Beriplex[®] US

1. Bleeding

2. Surgery

Primary Target Customer Segments

Beriplex[®] Registration Status 2012

Summary Commercial Opportunity POB

Q&A

Break

Haemophilia Products

Haemophilia

Coagulation Cascade

Innovations in Coagulation

Albumin Fusion Technology

Recombinant Albumin Fusion Proteins

Development of rIX-FP, CSL654

rIX-FP has a half life ~3x longer than BeneFIX in haemophilia B dogs

rIX-FP Activity in Haemophilia B Patients

rVIIa-FP is active and has a half life ~6 times longer than FVIIa

Recombinant Albumin Fusion Proteins Summary

rVIII-SingleChain (CSL627)

Short-term reconstituted stability at 25°C

rVIII-SingleChain: high affinity for vWF

rVIII-SingleChain: PK properties in mice and rats

CSL Ltd. Investor Presentation 2011

Global R&D Pipeline

Global R&D Portfolio December 2011

Global R&D Portfolio December 2011

Short to Mid-term Target Launch Dates