CSL V. Shire
`
`CSL EXHIBIT 1071
`
`Page 1 of 101
`
`CSL EXHIBIT 1071
`CSL v. Shire
`
`Page 1 of 101
`
`

`

`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 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
`71“
`71“
`
`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 approval of our products
`
`as well as their decisions regarding label claims; 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,
`
`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.
`
`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 fon/vard looking statements in these materials to reflect any
`
`change in expectations in relation to any fonNard 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.
`
`Trademarks
`
`Except where otherwise noted, brand names designated by a T" or ® throughout this presentation are trademarks either owned by and/or
`licensed to CSL or its affiliates.
`
`Page 2 of 101
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`Page 2 of 101
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`

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`Agenda December 2012 R&D Briefing
`
`Welcome
`
`Introduction & Highlights
`
`Immunoglobulins & Specialty Products
`
`- Clinical Development
`
`0 Commercial Opportunities
`
`- Q&A
`
`- Break
`
`Coagulation/Haemophilia
`
`Mark Dehring
`
`Andrew Cuthbertson
`
`Russell Basser
`
`Lutz Bonacker
`
`- Introduction & Technical Approach
`
`Andrew Cuthbertson
`
`- Clinical Development
`
`- Commercial Opportunities
`
`Russell Basser
`
`Lutz Bonacker
`
`Breakthrough Medicines & Licensing
`
`Andrew Cuthbertson
`
`Summary
`
`Q&A
`
`Page 3 of 101
`
`Andrew Cuthbertson
`
`CSIL
`
`Page 3 of 101
`
`

`

`Introduction and Highlights
`
`
`
`Page 4 of 101
`
`

`

`CSL R&D Strategy
`
`- Maintain commitment
`
`to extracting maximum
`value from existing assets
`and supporting and
`improving current products
`
`commercial capabilities
`
`- Develop new protein-based
`therapies for treating
`serious illnesses focusing
`on products that align with
`our technical and
`
`Page 5 of 101
`
`Page 5 of 101
`
`

`

`lmmunoglobulins Strategy
`
`Breakthrough
`Medicines
`
`Products
`
`Maintaining leadership
`position through focus on:
`
`- Patient convenience
`
`- Yield
`
`- Label
`
`- Formulation science
`
`- Specialty lgs
`
`Specialty
`Products
`
`I
`
`l b I'
`mmunogo u ms
`
`Haemophilia
`
`
`
`Page 6 of 101
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`Page 6 of 101
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`

`

`Specialty Products Strategy
`
`
`
`_
`
`Leveraging high quality, broad
`product portfolio through:
`- New markets
`
`Breakthrough
`Medicines
`
`
`
`l
`
`I
`
`mmunog 0b” m
`
`I.
`
`k
`
`Specialty
`Products
`
`- Novel indications
`
`' Novel modes of
`.
`.
`.
`administration
`
`Haemophilia
`Products
`
`Page 7 of 101
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`Page 7 of 101
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`

`

`Supporting and enhancing
`plasma products and
`developing novel
`recombinant portfolio with
`focus on:
`
`- Scientific and product
`innovafion
`
`- Patient benefit
`
`Haemophilia Strategy
`
`Haemophilia
`
`Products
`
`Page 8 of 101
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`Page 8 of 101
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`

`

`Breakthrough Medicines Strategy
`
`products
`
`Haemophilia
`Products
`
`Leveraging clinical and
`technical insight in developing
`novel protein-based therapies
`
`- Significant unmet need
`
`- Multiple indications
`
`Optimising value of IP
`portfolio and assets
`
`- Partner high opportunity
`
`Breakthrough
`
`Medicines
`
`V
`
`lmmunoglobulins
`
`specialty
`Products
`
`I MPage 9 of 101
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`Page 9 of 101
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`

`

`Leveraging Global Capabilities
`
`
`
`Organisation by Site
`
`Marburg
`
`Broad -
`meadows
`
`King of
`Prussia
`
`Integration via proioct managmnenl process
`
`Parkvllle
`
`management
`
` .5 Global project
`
`
`OrganisationbyFunction
`
`Recombinant protein
`
`manufacturing capabilities I.
`
`10
`
`Page 10 of 101
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`Page 10 of 101
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`

`

`R&D Investment
`
`CSL RESEARCH AND DEVELOPMENT INVESTMENT
`
`(US$ MILLIONS)
`
`203
`
`226
`
`278
`
`323
`
`368
`
`- New Product Development activities focus on
`innovative new therapies for life-threatening diseases.
`
`- Market Development strategies seek to bring
`therapies to new markets and new indications.
`
`- Life Cycle Management ensures continuous
`improvement of existing products.
`
`07—08
`
`08—09
`
`09-10
`
`10-11
`
`11-12
`
`11
`
`Page 11 of101
`
`CSE
`
`Page 11 of 101
`
`

`

`Global R&D Portfolio D
`
`December 2011
`
`Commercial]
`
`Phase IV
`ill
`
`lmmunoglobulins
`
`Haemophilia
`
`Influenza Vaccine
`
`Hizentra® US
`
`ill
`
`er
`
`ex
`
`Privigen®ClDP EU
`_ hm"
`'
`ti
`| d'
`ew l‘l
`ICG OHS
`AOI'th Surgery W Hizentra® EU
`T
`,
`Self Admin
`Fxm US
`_,
`New Indications
`a
`El
`
`Life Cycle
`Management
`
`Market
`
`Development
`
`
`
`New Product
`
`Development
`
`N
`
`Novel Plasma
`Proteins
`erF-FP
`
`Rec Coagulation
`Factors
`
`CSL689 erla-FP
`'
`'
`
`CSL654 rlX-FP
`CSL627 rVIll-SC
`
`Partnered Vaccine
`Programs‘
`
`Partnered Vaccine Partnered Vaccine
`Programs‘
`Programs‘
`
`Partnered Vaccine
`Programs‘
`
`CSL112
`reconstituted HDL
`
`CAM3001
`GM-CSFR - AZ'
`
`P gingivalis POD
`CRC_OHS/sanofi *
`
`Discovery
`Projects
`
`CSL362 IL-3R
`
`CSL324 G-CSFR
`
`VEGFB
`
`IL-13R
`
`Core Capabilities
`
`lmmunOQIObulinS
`
`Haemophilia
`
`Breakthrough Medicines
`
`VaccinesilP
`
`12
`
`“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
`
`c
`
`Page 12 of 101
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`Page 12 of 101
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`

`

`Progress through Stage Gates in 2012
`
`New
`Product
`
`Research
`
`Product
`
`Development
`8 GLP
`Toxicology
`
`Opportunity
`AA
`
`Registration
`Launch
`PhaseIV
`
`Post
`
`Registration
`
`Enter
`Research
`
`grgferlggg:
`& GU, Tax
`
`Entor
`Phase I
`
`Enter
`Phase II
`
`Enter
`Phase III
`
`Register
`Launch
`
`.
`Hizentra CIDP
`
`Fibrmogen
`Aomc EU
`
`Hizentra
`Canada‘ EU
`
`Berinert
`Seif Admin
`
`Berinert
`Subcut
`
`Hizentra
`
`Japan
`
`Privigen CIDP
`7
`EU
`
`->
`
`Beiiplex US
`Bleeding
`
`13
`
`Page 13 of101
`
`CSE
`
`Page 13 of 101
`
`

`

`Global R&D Portfolio D
`
`December 2012
`
`mmmm Commercial]
`
`Phase IV
`
`
`Beriplex® us
`
`lmmunoglobulins
`
`Haemophilia
`
`specialty Products
`
`influenza Vaccine
`
`Privigen® ClDP
`EU
`
`Biostateié‘? EU
`
`Hizentra® USIEU
`
`wS
`
`elf Admin
`
`Life Cycle
`Management
`
`Market
`
`Development
`
`
`
`New Product
`
`Development
`
`Novel Plasma
`Proteins
`
`Rec Coagulation
`Factors
`
`Partnered Vaccine
`Programs‘
`
`
`
`P gingivalis POD
`CRC-OHS/Sanofi *
`
`Hizentra® ClDP
`
`Berinert® SC
`
`Zemaira® EU
`
`
` 'W
`
`Aortic EU
`
`CSL654 rlX-FP
`
`WN
`
`ew Indications
`
`W N
`
`indications
`
`
`
`
`
`CSL627 erllSC
`CSLBBQ rVIlarFP
`
`Partnered Vaccine Partnered Vaccine
`Programs‘
`Programs“
`
`Partnered Vaccine
`Programs'
`
`Discovery
`Projects
`
`CSL346 VEGFB
`
`CSL334 lL-13R
`
`CSL324 G-CSFR
`
`CSL362 lL-3R
`
`CAM3001
`GM-CSFR - AZ'
`
`reconstituted HDL
`
`CSL112
`
`Core Capabilities
`
`Immunoglobulins
`
`Haemophilia
`
`Breakthrouh Medicines
`
`Vaccines {IF
`
`14
`
`“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
`
`csL'-
`
`Page 14 of 101
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`Page 14 of 101
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`

`

`Immunoglobulins
`
` Page 15 oflOl
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`Page 15 of 101
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`

`

`Immunoglobulins
`
`Breakthrough
`Medicmes
`
`“
`
`_
`
`Maintaining leadership
`position through focus on:
`- Patient convenience
`
`- Yield
`
`Immunoglobulins
`
`-
`.
`Speaa“
`Product
`
`- Label
`
`_
`,
`- Formulation scuence
`
`- Privigen®
`
`- Specialty lgs
`
`Haemophilia
`Products
`
`_
`
`Key Focus
`° Hizentra®
`
`fl
`
`Page 16 of 101
`
`/'
`
`CSIL
`
`Page 16 of 101
`
`

`

`Innovation to Drive Growth
`
`- Efficient and competitive cost structure
`
`- lg 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 by Cytogam®
`
`17
`
`Page 17 of 101
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`CSIL
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`Page 17 of 101
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`

`

`Cytogam®
`
`CYTOGAM”
`
`Cytomegalovims Immune Globulin
`Intravenous (Human) [CMV-IVIG}
`
`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 efficacy of CMV immunoglobulin in preventing
`mother to baby transmission
`- Large multi-site clinical trial screening >150,000 women
`
`23'
`
`commenced December 2011
`
`c CSL donating Cytogam®
`
`- Primary analysis expected 2016
`
`18
`
`Page 18 of 101
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`Page 18 of 101
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`

`

`Hizentra®
`
`Hiz/entra°
`
`Immune Globulln Subculamis
`
`lHumanl
`
`20% Liquid
`
`The first 20% high concentration low volume SCIG for convenient self
`
`administration providing steady-state lg 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 Phase III licensing study complete
`
`
`
`- supports safety and efficacy of Hizentra® for PID
`
`- new drug application submitted to PMDA in Sept 12
`
`19
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`Page 19 of101
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`C
`
`SI;
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`Page 19 of 101
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`

`

`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
`
`2°
`
`Page 20 of 101
`
`Hizgttra
`
`Immune Globulm Subcmanews
`{Human}
`2095mm
`
`c
`
`SI;
`
`Page 20 of 101
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`

`

`Chronic Inflammatory Polyneuropathy (CIDP)
`
`Axon Sheath
`
`- A chronic peripheral nerve
`
`disease with progressive
`
`muscle weakness and loss
`
`ofsensaflon,usuaHy
`
`occuring in elderly patients
`
`- Most common chronic
`
`autoimmune neuropathy
`
`Axon - A long nerVe fiber that carries
`messages to and from the brain, such
`
`
`as telling a muscle when to move.
`
`
`Myelin sheath - The protective
`covering around nerve fibers.
`which allows the messages
`to move quickly.
`
`Peripheral nerves
`arms/hands
`
`21
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`Page 21 of 101
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`CSE
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`Page 21 of 101
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`

`

`Potential benefits of Hizentra® in Patients
`
`with CIDP
`
`Current maintenance
`
`therapies
`
`
`
`LeSS convenience
`
`Adverse
`effects with
`long term use Levels show peaks
`8‘ troughs
`Hospital visits
`
`'"VaSiVetherapy
`_
`_
`_
`__
`lelted aVaI'ablllty
`Short term efficacy
`
`22
`
`Page 22 of 101
`
`’ Hizentra®
`
`- Avoids drawbacks of iv.
`
`° More same '96 levels
`
`route
`
`- Reduced volume
`
`- Increases patient autonomy
`_
`_
`- Less systemic snde effects
`
` Oral
`steroids
`
`Plasma
`
`Exchange
`
`
`
`Page 22 of 101
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`

`

`The PATH Trial
`
`: Hizentra® in CIDP
`
` w
`K. .* .*
`
`is
`
`*
`
`
`
`
`
`- 150 patients
`
`- 2 doses vs placebo
`
`0 Study approved by
`FDA, EMA, PMDA
`
`- Recruiting in US &
`EU
`
`23
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`Page 23 of 101
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`CSE
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`Page 23 of 101
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`

`

`Privigen®
`
`6-3
`[Dr—‘I VMt; therapy
`
`The first and only 10% liquid intravenous immunoglobulin (lVlg) 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 lg manufacturing facility in Broadmeadows
`
`Strengthening Presence in Neurology Market fig
`° Phase III study in ClDP completed in Europe
`' Study shows treatment with Privigen® improved function
`in patients with chronic ClDP
`
`- Dossier submitted to EMA in May 12
`
`24
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`Page 24 of 101
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`CSIL
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`Page 24 of 101
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`

`

`Privigen® in CIDP: Rate of Response
`
`E a
`
`,
`
`E ‘
`
`5
`'O
`
`Sa
`
`.
`«I:
`
`0I
`
`!
`
`100 i
`
`90 7
`
`80 7
`
`70 A
`
`60 -
`
`50 -
`
`40 ~
`
`30 v
`
`20 i
`
`10 7
`
`
`
`0 w
`
`Total
`
`IVlg pretreated
`
`lVlg untreated
`
`Page 25 of 101
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`

`

`Specialty Products
`
`
`
`Page 26 of 101
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`

`

`Specialty Products
`
`Breakthrough
`Medicines
`
`Leveraging high quality,
`broad product portfolio
`through:
`
`° New markets
`
`- Novel indications
`
`Immunoglobuiins Key Focus
`
`SPECIBW
`Products
`
`- Novel modes of
`administration
`
`Haemophilia
`Products
`
`° Ber'pleX®
`- Fibrinogen
`
`- Zemaira®
`
`
`Page 27 of 101
`
`- Berinert®
`
`Page 27 of 101
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`

`

`Beriplex®
`
`Beriplexn/Ng
`
`- Prothrombin Complex Concentrate = PCC
`
`- vitamin K-dependent coagulation factors (Fll, FVll, 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, controlled clinical trials
`
`- Bleeding study completed
`
`- Surgical study recruitment completed
`
`BLA submitted in US for acute bleeding
`
`' Accepted for standard review
`
`28
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`Page 28 of 101
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`CSIL
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`Page 28 of 101
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`

`

`F'b '
`I
`
`n 0 g e n
`
`®
`
`Rinsmpf _
`Millage/2 (once/)t/ale
`
`The first and only treatment approved by the US FDA for acute
`
`bleeding episodes in patients with congenital fibrinogen deficiency
`
`Europe
`
`- Peri-lpost-operative control of coagulopathic bleeding
`
`- REPLACE Phase III study
`
`- 200 subjects — recruitment commenced Jan 2012
`
`- Aim to complete recruitment end 2013
`
`US
`
`- Coagulopathic bleeding related to complex cardiac surgery
`
`- In dialogue with FDA
`
`29
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`Page 29 of 101
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`CSIL
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`Page 29 of 101
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`

`

`Zemaira®
`
`<j z.
`
`alpha1-prote
`
`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 outcome (disease
`modification)
`
`- Increase diagnosis and treatment
`
`Anticipating pivotal efficacy data early 2013
`
`30
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`Page 30 of 101
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`CS[
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`Page 30 of 101
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`

`

`Berinert®
`
`8597153157.
`
`Plasma derived, pasteurised & nanofiltered concentrate of C1 Esterase
`
`Inhibitor indicated for the. treatment of acute abdominal or facial attacks
`
`
`
`
`
`Genetic Mutation
`
`Cl—lNH Deficiency
`
`‘
`
`
`
`‘\v 7
`
`Plasma Cascade
`Dysregulation
`
`
`
`Vascular Permeability
`
`317
`
`__'u h“: “7
`Page 31 of 101
`
`777-7
`
`7’7 “WI—7
`
`A
`
`r
`
`A
`
`_
`
`'-
`
`Page 31 of 101
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`

`

`Unpredictable and occur
`anywhere in the body
`
`Life-threatening if laryngeal
`swelling
`
`Attacks caused by stress,
`
`infection, menstruation, some
`
`What Happens to Patients?
`
`Recurrent episodes of swelling,
`sometime with a rash
`
`drugs, unknown causes
`
`32
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`Page 32 of 101
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`

`

`Berinert®
`
`Benznsnr
`
`.0.04
`
`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 label expansion Berinert® now also indicated to
`treat life-threatening laryngeal HAE attacks, as well as facial
`and abdominal attacks
`
`33
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`Page 33 of 101
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`CSIL
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`Page 33 of 101
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`

`

`Overcoming Challenges in Long-term
`Prophylaxis of HAE Attacks
`
`Current prophylactic
`
`Oral
`androgens
`. Limited by
`adverse
`:gggtgéuy in
`women and
`children
`
`High concentration
`subcutaneous (sc) Berinert®
`- Avoids drawbacks of iv.
`- Low volume for so. administration
`- Builds on well-established safety
`prof'le
`
`therapies
`
`
`
`Intravenous
`C1_|NH
`. Inconvenience ‘
`and risks _of
`gzfigtgfiggbn
`
`i
`
` *
`
`34
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`Page 34 of 101
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`CSIL
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`Page 34 of 101
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`

`

`Berinert® Subcutaneous Prophylaxis Program
`
`COmPQCT
`
`Clinical Studies for Opttmal Management in
`Preventing Angioedema with lowrvolume
`subcutaneous Ci inhibitor Replacement Therapy
`
`safety and
`pharmacokinetic
`study
`
`- due to commence 2H 2013
`
`- Study ongoing in US and Germany - due to complete 1H
`2013
`
`.
`.
`_
`_
`- Select safe and efficaCIous dosmg for clinical efficacy trial
`
`35
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`Page 35 of 101
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`'-
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`Page 35 of 101
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`

`

`Commercial Opportunities
`
`and Activities
`
`Page 36 of 101
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`Page 36 of 101
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`

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`Immunoglobulins
`
`
`
`Page 37 of 101
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`

`

`The Immunoglobulin Market is attractive
`
`The 2012 IG market is a > $6 B
`
`opportunity
`
`- Market includes IVIG, SCIG
`
`- CSL is well positioned
`
`- CSL
`
`and Hyperimmunes
`
`- Growing Market
`
`38
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`Page 38 of 101
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`CSE
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`

`

`CSL’s Immunoglobulin Portfolio
`
`- Globalise portfolio
`
`- Expand into neurology
`
`- Increase convenience
`
`2011/12
`
`Privigen
`
`
`
`39
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`Page 39 of 101
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`

`

`lg Portfolio Positioning
`
`
`
`»’ irst and only Proline-stabilised IVIG
`
`- High purity 10% IIqUId IVIG
`- Optimal dimer formulation throughout shelf life for
`improved tolerability
`
`
`
`
`‘ First and only 20% Proline-stabilised SCIG
`
`- Low administration volume increases efficiency
`
`0 Convenient — few sites & fast infusion
`
`' Lyophilised lVlG
`
`- Reconstitution options
`
`- Long track record of safety & reliability
`
`- Broad indications
`
`[:Jl—‘IVIQEI—Vo
`rno.,f‘ifif”,l‘fffflTittitftlii?
`
`Hie/mid
`
`Immune Globulin Subcutaneous (Human) 20%Liquid
`
`Sandoglobulinsl '
`
`5‘ ..‘:~.i NF
`
`40
`
`Page 40 of 101
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`Page 40 of 101
`
`

`

`Globalise Portfolio
`
`Privigen & Hizentra
`
`(PL)
`
`Privigen & Hizentra
`(HU)
`
`Privigen & Hizentra
`
`Privigen (BR)
`
`Privigen (TW)
`
`a“
`
`(CZ)
`
`Privigen (TR)
`
`Privigen (MY)
`
`Privigen (SG)
`
`Privigen (MX)
`
`Privigen (RU)
`_
`
`Hizentra (TR)
`Hizentra (BR)
`- —
`
`Hizentra (AR)
`
`Privigen (HK)
`
`Hizentra (JAP)
`
`Hizentra (MX)
`
`z'rr'
`
`V ;-
`
`‘a .
`
`I“ r-
`_-.
`._.‘.
`..4 , - ;
`
`‘
`
`- Privigen® currently registered in 55 countries
`
`- Hizentra® currently registered in 33 countries
`
`- Continue global launches for Privigen® and Hizentra®
`
`4‘
`
`Page 41 of 101
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`CSIL
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`

`

`Expand into Neurology
`
`
`Hizentra 20%
`Privigen 10% CIDP
`CIDP (EU)
`
`
`(EU, CH, CAN) Hizentra 20%
`
`CIDP (US)
`
`- Strengthen presence in neurological segment
`
`- Launch Privigen® in CIDP in the EU
`
`- Develop Hizentra® in CIDP in the US, the EU and RoW
`
`42
`
`Page 42 of 101
`
`"
`
`Page 42 of 101
`
`

`

`Increase Convenience
`
`Privigen 409 Presentation
`
`(US, CH, EU)
`
`
`Hizentra Convenience Initiatives
`
`Hizentra 109
`(US, EU)
`
`- Increase dosing flexibility
`
`- Differentiate through convenience launches:
`- 10 g vial Hizentra®
`- 40 g vial Privigen®
`- Ongoing convenience initiatives
`
`43
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`Page 43 of 101
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`

`

`
`
`
`Reducing number of vials
`9 increases convenience
`
`Prepare syringe
`
`(continued)
`
`of Hizentra
`
`Inspect each vial
`
`Gather your supplies
`
`Hiz'entra“
`
`Immune GmJIin Stlbunmems (Human) 20%qu
`
`Infusion Steps
`
`4 preparation steps per vial
`
`
`
`ntnxuuuun
`I
`d ‘1
`49‘“
`
`,
`
`
`
`Start infusion
`
`44
`
`Page 44 of 101
`
`
`
`r»: }
`
`"
`
`'
`
`Insert Sub-Q needle(s)
`
`Prepare '"Ject'm sue“)
`
`v-
`
`Page 44 of 101
`
`

`

`Cytogam®
`
`CYTDGAM'
`
`cutaneme mrmna Gobulin
`lmr'avamm [Hurmnl (CMV-NIB]
`
`The only registered CMV immunoglobulin in the US indicated for the
`
`prevention of CMV disease associated with transplantation
`
`
`Center for Disease Control1:
`
`- CMV is the most common viral
`
`P°tentia' opp°nunity
`
`infection that infants are born with
`_
`_
`In the United States
`
`.
`.
`Assumlng Screening at week 23
`95% screening uptake
`
`- About 1 in 150 children is born with
`
`Number of Patients: 25 thousand
`
`congenital CMV infection
`
`- About 1 in 750 children in the US is
`
`Total market (volume): ~ 1,000 kg
`
`born with or develops permanent
`
`Total US. Market: ~ $US 300-500 M
`
`problems due to congenital CMV
`
`infection
`
`1) http://www.cdc.gov/cmv/trends-stats.html
`
`45
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`Page 45 of 101
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`CS"
`L
`
`Page 45 of 101
`
`

`

`Specialty Products
`
`
`
`Page 46 of 101
`
`

`

`Specialty Products
`
`2011 2012
`
`Other Specialty Products
`
`' a Wound Healing
`
` ‘
`
`Kybernin
`
`Bennen
`
`Zemaira
`
`Streptase
`
`I $U3618 M
`
`Fibrogammin
`
`Beriplex
`
`Riastap
`
`Perioperative Bleeding
`
`o Increase clinical data set
`
`- Add indications
`
`- Expand regionally
`
`47
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`

`

`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
`
`- Include in perioperative bleeding management algorithm
`
`1)
`
`2)
`
`48
`
`IMS data July 2012
`
`Connolly et al NJEM 2009, Patel et al NEJM 2011, Granger et al NEJM 2011
`
`Page 48 of 101
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`

`

`Blood Products vs. Concentrates
`
`COFiFaCT(73
`
`Factoerll (attenuate (Human)
`
`
`
`Confidex 4,
`toe\‘
`
`‘..\¢\\\‘\§§\\2‘I
`
`i_~E:JTIE.3“?
`
`Q ~
`
`Concentrated, virus inactivated, room
`temperature storage, Fibrinogen concentration
`209 / L
`
`_
`
`Fibrinogen concentration at =2.3g / L
`Not virus inactivated
`
`Frozen, requires time (<50 minutes) to thaw
`
`Need to be matched to blood type
`
`Not virus inactivated
`
`Platelets
`Short shelf life (5 days)
`Risk of bacterial
`
`-
`
`' contamination
`
`Cryo
`
`- Frozen, require time to thaw
`
`- Pooled from 10 bags of FFP in the blood
`bank
`
`- Average Fibrinogen concentration = 69 /L
`
`49
`
`Page 49 of 101
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`Page 49 of 101
`
`

`

`Fibrinogen®
`
`Ring/5P5
`
`Hui/709m Concentrate
`
`
`
`- Obtain WEU & US acquired label
`
`- Initiate acquired label expansion
`
`- Generate, publish & communicate
`
`data
`
`
`
`INTERNATIONAL SYMPOSIUM OF INTENSIVE CARE
`AND EMERGENCY MEDICINE
`
`55f; 9%”
`
`50
`
`Page 50 of 101
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`CSE
`
`Page 50 of 101
`
`

`

`Bring ("Zeifiirar 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
`
`51
`
`- Demonstrate economic benefit
`
`o Reimbursement
`
`- Enhanced testing & diagnosis
`
`Page 51 of 101
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`Page 51 of 101
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`

`Be rinert®
`
`BERInERT’
`
`.0...
`
`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 so treatment option
`
`- Continue geographical expansion
`
`- Continuous Life Cycle Management to improve product profile
`
`1)Agostini et al. J Allergy Clin Immunol. 2004
`
`2) Craig et al. J Allergy Clin Immunol 2009
`
`52
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`

`

`Berinert® - Current Life Cycle Activities
`
`P
`
`roduc
`improvement
`
`t
`
`oLow-volume, high
`.
`.
`concentration, formulation
`
`
`
`Expand
`indication
`
`- Short—term prophylaxis,
`2013 (EU/Can)
`- Further initiatives ongoing
`
`COmPQCT
`
`in l: e; ".‘l 0p -' ml Mril agierre’ti
`.
`.
`'il‘i‘llutl
`
`:lii
`
`ill
`
`‘11-.C'
`
`iliil
`
`ill’lpl
`
`New
`
`method of
`use
`
`- Prophylaxis Indication
`with so. formulation
`
`53
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`

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`Page 54 of 101
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`Page 54 of 101
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`

`

` Page 55 of 101
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`Page 55 of 101
`
`

`

`Haemophilia Products
`
` Page 56 of 101
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`Page 56 of 101
`
`

`

`Haemophilia
`
`
`
`Breakthrough
`
`_
`
`"
`
`Supporting and enhancing
`plasma products and developing
`novel recombinant portfolio with
`focus on:
`I
`_
`.
`- Scnentlfic and product
`innovafion
`
`Immunoglobulin;
`/"
`
`/
`
`”
`
`Specialty
`‘\ Products
`\,
`
`\\
`
`Haemophilia
`
`Products
`
` Medicines
`
`- Patient benefit
`
`Key Focus
`
`- Long acting rlX-FP
`
`- Long acting erla-FP
`
`- erll-Single Chain
`
`- Research into long acting
`rVWF-FP
`
`
`Page 57 of 101
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`Page 57 of 101
`
`

`

`Innovation to Drive Growth
`
`- Patient convenience primary
`
`driver of innovation
`
`- Albumin fusion technology
`
`- rIX-FP, rVIIa-FP, rVWF-FP
`
`Improved
`half life.
`extended
`dosing
`interval
`
` Scientific Edge
`
`rAlbumin
`as fusion
`platform
`
`Precise
`engineering
`of specially
`designed
`linker
`
`- Factor Vlll
`
`High VWF affinity
`
`- biobetter rVIll-SingleChain
`
`58
`
`Page 58 of 101
`
`Improved molecular stability
`
`Opportunity for Extended
`Dosing Interval
`
`CSIL
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`

`rIX-FP (CSL654)
`
`
`
`CSE
`
`%P
`
`ROLONG 9 PP
`
`59
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`Page 59 of 101
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`

`

`rlX-FP (CSL654) Clinical Program
`
`Extension
`
`Phase 1/2
`
`Phase 2/3
`
`Phase 3
`
`Paediatric
`
`PK
`
`PK
`
`Long-term safety
`
`Long—term safety
`
`7d prophylaxis
`
`On-demand
`
`7—14d prophylaxis
`
`On-demand
`
`Surgical prophylaxis
`
`’—/
`
`‘V
`
`PROLONG 9 FF’
`
`60
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`

`

`blood 2012 120: 2405-2411
`
`Prepublished online August 2, 2012:
`doi:10.1 182/blood-2012-05-429688
`
`Safety and pharmacokinetics of a novel recombinant fusion protein
`linking coagulation factor IX with albumin (rIX-FP) in hemophilia B
`patients
`
`Elena Santagostino. Claude Nearer, Robert Klamroth, Andreas Tiede. Ingrid Pabinger-Fasching.
`assimo Morfini
`6U
`Christine Voigt, Iris Jacobs and
`
`70-
`
`so
`3 SD
`E
`E 40
`E 30 .
`20
`
`10
`
`+SOIU/kurlX-FHM13)
`—0— SOlU/kg rFIK (n-B)
`-— SOIU/kg delX (11:4)
`- - m 5%tvough
`
`o
`
`24
`
`43
`
`72
`
`96
`
`120
`
`144
`
`168
`Hours
`
`192
`
`216
`
`240
`
`26:
`
`288
`
`312
`
`336
`
`
`
`*25 Ill/kg rlX—FP [n=7)
`+50 lU/kg rlX-FP (n=13)
`-i— 75 IU/kg rlX-FP [n=8)
`---- 5% trough
`-----"2% trough
`
`
`
`nxActiviy(nu/an
`
`Compared with in market rFlX
`
`- 5.3-fold longer half-life (92hrs)
`
`- ~ 45% higher incremental recovery
`
`- ~7-fold larger AUC
`
`- ~7-fold slower clearance
`
`V
`
`I,
`
`\
`
`PROLONG 9 PP
`
`61
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`

`

`Efficacy of rlX-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 controlled by a single injection (the
`
`rest by only one additional injection)
`
`PROLONG
`
`9
`
`FF’
`
`62
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`

`

`rlX-FP (CSL654) Safety Data
`
`- Excellent safety profile in completed studies
`
`- Well tolerated
`
`- No inhibitors
`
`- No adverse events related to CSL654
`
`PROLONG
`
`9
`
`FP
`
`63
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`

`

`rlX-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
`
`_
`
`,/
`\
`I
`\/-\
`PROLONG 9 FF’
`
`64
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`

`

`erIa-FP (CSL689)
`
`
`
`PROLONG 7 PF
`
`65
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`

`Development of rVIla—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
`
`- Completed discussions with PEl
`
`- Briefing documents to FDA/ EMA
`
`/
`
`__r—\
`
`,‘-/
`
`i
`
`pROLDNG 7 PP
`
`66
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`

`

`Phase 1 Study of erla—FP in Healthy Volunteers
`
`100000
`
`1 0000
`
`
`
`
`
` 10 CorrectedplasmaFVIIactivity[mU/mL]
`
`
`
`
`
`1000
`
`100
`
`Half—life 3-4 fold longer than rFVlla
`
`, A
`
`v//
`
`PROLONG 7 FF
`
`67
`
`Page 67 of 101
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`(SSE
`
`Page 67 of 101
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`

`

`Potential of erla-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
`
`‘
`
`7/
`
`,
`
`,
`
`PROLONG 7 PF
`
`68
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`Page 68 of 101
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`SI;
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`Page 68 of 101
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`

`

`erII-SingleChain (CSL627)
`
`
`
`C3AFFINITY
`
`erll-SingleChain Clinical Trial Program
`
`69
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`

`

`erIl-SingleChain: approach for improved FVlll
`
`- FVlll’s physiological partner in plasma is von Willebrand
`factor (vWF)
`
`- FVIII/VWF complex is important role in the physiological activity
`and clearance of FVlll
`
`o Aim - improve binding to VWF
`
`- FVlll is an unstable molecule in the manufacturing
`environment
`
`- Potential for dissociation and loss of procoagulant activity of FVIII
`
`- Aim - improve molecular stability
`
`~2oo
`
`:r_______"-m'!
`
`80 kDa
`
`7°
`
`Page 70 of 101
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`

`

`rVIlI-SingleChain: high affinity for vWF
`
`Binding to plasma-derived (pd) VWF
`
`Comparison of VWF affinity constants
`
`20
`
`18
`
`
`
`
`
`Affinityconstant(1I(nm)
`
`A —
`
`Single chain rom (CSL627)
`
`250
`
`200
`
`150
`
`100
`
`50
`
`
`
`Relativeunits
`
`16
`
`14
`
`12
`
`10
`
`Oh)th
`
`— Full length rFVlll
`
`0
`
`0
`
`500
`
`1,000
`
`1,500
`
`erIl-SingleChain
`(CSL627)
`
`Full length rFVlll
`
`Surface plasmon resonance (SPR) analysis Time (s)
`CSL Behring. Data on file
`
`71
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`

`

`rVIII-SingleChain : PK profiles in rodents
`
`PK in haemophilia A mice
`
`pK in rats
`
`6,000
`
`
`
`100 lU/kg va:c
`_
`.
`.
`n—5/tlmep0int
`geometric means i SD
`
`5,000
`
`3
`g 4,000
`—
`E
`3 000
`2)
`’
`.'.:.'
`2,000
`>
`u.
`
`1,000
`
`0
`
`200 lU/kg va:c
`n=3ltimepoint
`geometric means i SD
`
`5
`
`4
`
`3 i
`
`3 g
`
`3
`E
`a:
`-;
`g
`g
`9
`5
`L
`
`>I
`
`0
`
`20
`
`40
`
`60
`
`so
`
`0
`
`120
`
`240
`
`360
`
`480
`
`Time (hrs)
`
`— CSL627
`
`— Full-length rFVlll
`
`Time (min)
`
`CSL627 has ~ 50% increase in terminal half-life compared to full-length rFVlll
`
`72
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`

`

`rVIII-SingleChain Phase 1/3 Study Design
`
`Planned Dec 2012
`
`Interim analysis
`
`CSL627 & Octagog alfa
`single-dose PK (n=30)
`
`CSL627 repeat-dose, on-demand or
`prophylaxis (n=30)
`
`Part 2
`
`CSL627 repeat-dose, on-demand or
`prophylaxis (n=78 evaluable subjects)
`
`Single-dose PK erIl-SingleChain (n213)
`
`Surgical sub-study
`
`Includes patients from Parts 2 & 3
`
`(n=5 with 210 major surgeries)
`
`
`
`Extensionstudy
`
` Part 1
`
`Study entry
`
`
`
`I
`
`I
`
`- Part 1 due to complete enrolment 2012
`- Part 3 to commence early 2013
`
`73
`
`erlI-Singlechain Clinical Trial Program
`
`Page 73 of 101
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`

`

`Recombinant Coagulation Portfolio Summary
`
`Target Launch Dates
`
`CSL654 (rlX-FP)
`
`2015
`
`
`
`74
`
`Page 74 of 101
`
`°
`-
`
`Pivotal Phase ll/lll study commenced
`Phase I data demonstrate >5x half life extension
`
`- Orphan drug status granted by US FDA
`
`CSL627 (erll-SingleChain)
`
`-
`
`o
`
`Phase |/lll trial commenced
`
`Early clinical data support potential half life extension
`
`CSL689 (erla-FP)
`
`-
`
`Initial pharmacokinetic data shows a 3-4x half life extension
`
`- Orphan drug status granted by US FDA
`
`CSL65O (rVWF-FP)
`
`- Candidate pre-clinical molecule shows a 5x half life extension
`
`Vi
`
`Page 74 of 101
`
`

`

`Commercial Opportunities
`
`and Activities
`
`Page 75 of 101
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`Page 75 of 101
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`Coagulation Sales
`
`
`
`2011 2012
`
`
`
`I $US‘1,05‘8M
`
`- Launch rIX-FP in 2015
`
`- Launch erll-SingIeChain as bio-better in 2016
`
`- Grow pd FVIII
`
`76
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`Page 76 of 101
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`CSE
`
`
`
`Page 76 of 101
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`

`

`Coagulation: Total Market Size
`Key Market Segments and Products
`
`- Haemate P
`
`I
`
`‘
`
`- Biostate
`
`I
`
`-
`
`V
`
`- Biostate
`
`- HaemateP
`
`77
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`77 of 101
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`Pa 6
`CSL MarkethstImate 2012
`
`Y.
`
`Page 77 of 101
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`

`

`Coagulation:
`Key Market Segments and Products.
`
`- Beriate
`
`- Helixate
`
`NexGen/FSI81
`
`Chain
`
`.
`.
`t
`minggggwfn ‘ Blostate
`- HaemateP
`
`- Mononine
`
`- Berinin
`
`
`
`
`
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`
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`
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`
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`
`,
`
`- Biostate EU
`
`- Haemate PI
`
`-
`
`rVWF-FP
`
`78
`
`78 of 101
`.
`Pa 6
`CSL MarkethstImate 2012
`
`~
`
`-
`
`rlX-FP
`
`Page 78 of 101
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`

`

`Recombinant Coagulation Portfolio
`
`W' P I ISISYAP'__
`
`miz‘n'n' P IW‘
`
`cum...»- — '
`mus-h.—
`Fm'xg‘m‘"
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`lulu-min- aux—-
`Mononiqe'
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`Berinin° P
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`Beriate"
`hmmmmwm Haemate’ P I Humate-P'
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`' Ml!-MM
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`Hum
`Helixateo NexGen I Helixate? F5
`
`
`
`
`
`
`
`- Differentiate recombinant albumin fusion platform and launch rIX-FP
`
`- Differentiate and launch erll-SingleChain
`
`- Strong support for Helixate and growth of erll-SingleChain
`
`79
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`Page 79 of 101
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`CSIL
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`Page 79 of 101
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`

`rCoags Slide Kit Jan 2012
`
`Specifically
`designed
`aner
`
`Innovations
`
`in Coagulation
`
`Scientific Edge
`
`Recombinant
`
`Albumin as
`
`fusion
`
`panner
`
`Improved half
`life, extended
`dosing
`interval
`
`80
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`

`rlX-FP: The Scientific Edge
`
`Phase 1 data
`
`rlX-FP 1
`(CSL Behring —
`
`rFIX- PEGylated 2
`
`rFIX-Fc fusion 3
`
`Albumin Fusion)
`
`PROLONG
`
`FP
`
`Prophylaxrs usmg longer half-life fusion protein
`
`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): 2695-2701
`3 Shapiro et al., Blood 2012, 119(3): 666-672
`
`81
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`rVIIa-FP: The Scientific Edge
`
`T/2 extension
`
`erla-FP
`
`Phase 1 being analysed
`
`(CSL Behring-Albumin Fusion)
`
`PROLONG
`
`PP
`
`The only half life extension technology currently in clinica