CSL EXHIBIT 1071
`
`— P
`
`R&D Briefing
`
`age 1 of 101
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`Page 1 of 101
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`CSL EXHIBIT 1071
`
`

`

`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
`nu
`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 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.
`
`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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`Agenda December 2012 R&D Briefing
`
`- Welcome
`
`-
`
`-
`
`Introduction & Highlights
`
`Immunoglobulins & Specialty Products
`
`Mark Dehring
`
`Andrew Cuthbertson
`
`- Clinical Development
`
`- Commercial Opportunities
`
`Russell Basser
`
`Lutz Bonacker
`
`- Q&A
`
`- Break
`
`- Coagulation/Haemophilia
`
`- 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
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`Andrew Cuthbertson
`
`CSE
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`Page 3 of 101
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`

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`age 4 of 101
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`— P
`
`Introduction and Highlights
`
`Page 4 of 101
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`

`

`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
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`CSIL
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`Page 5 of 101
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`

`

`Immunoglobulins Strategy
`
`Breakthrough
`Medicines
`
`Products
`
`Immunoglobulins
`
`Specialty
`Products
`
`Haemophilia
`
`Maintaining leadership
`position through focus on:
`
`- Patient convenience
`
`- Yield
`
`- Label
`
`- Formulation science
`
`- Specialty Igs
`
`
`
`Page 6 of 101
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`

`

`Specialty Products Strategy
`
`,_
`
`- Novel indications
`
`
`
`Leveraging high quality, broad
`product portfolio through:
`Breakthrough
`
`Madicmes
`- New markets
`
`Specialty
`° Novel modes of
`Products
`administration
`
`Immunogmbums
`
`
`
`Haemophilia
`Products
`
`Page 7 of 101
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`csr; 1
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`

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`Haemophilia Strategy
`
`Haemophilia
`
`Products
`
`Supporting and enhancing
`plasma products and
`developing novel
`recombinant portfolio with
`focus on:
`
`- Scientific and product
`innovafion
`
`° Patient benefit
`
`Page 8 of 101
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`CSIL
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`Breakthrough Medicines Strategy
`
`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
`
`‘
`
`lmmunoglobulins
`
`specialty
`Products
`
`Haemophilia
`Products
`
`MM CSIL
`
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`

`

`Leveraging Global Capabilities
`
`Organisation by Site
`
`
`Marburg
`Broad -
`King of
`Parkvllle
`meadows
`Prussia
`
`
`up Global project
`
`management
`
`Integration via DFOIC‘C! nmnagmnem process
`
`OrganisationbyFunction
`
`
`
`
`
`
`Recombinant protein
`manufacturing capabilities ’
`
`10
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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.
`
`CSE
`
`07-08
`
`08-09
`
`09-10
`
`10—11
`
`11-12
`
`11
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`

`

`Global R&D Portfolio D
`
`December 2011
`
`
`
`
`
`lmmunoglobulins
`
`Influenza Vaccine
`
`
`Privigenfi‘ClDP EU
`-Fifi! -FEEH'
`Hizentra® us
`New Indications
`Aortic Surgery —rW Hizentra® EU
`
`FXIH US
`I E:
`mug-v Self Admin
`
`._—1r;——__
`New indications
`
`
`Novel Plasma
`Proteins
`
`CSL689 rVIla-FP
`’
`
`erF-FP
`Rec Coagulation
`Factors
`
`CSL654 rlX-FP
`
`CSL627 erll-SC
`
`Partnered Vaccine
`Programs‘
`
`Partnered Vaccine Partnered Vaccine Partnered Vaccine
`Programs‘
`Programs‘
`Programs‘
`
`P gingivalis POD
`CRC_OHS/Sanofi *
`
`.
`Discovery
`
`CSL362 IL-3R
`
`CSL324 G-CSFR
`
`VEGFB
`
`CSL112
`reconstituted HDL
`
`CAM3001
`GM-CSFR - AZ‘
`
`Life Cycle
`Management
`
`Market
`Development
`
`
`
`New PrOdUCt
`Development
`
`Core Capabilifies
`
`Immunoglobulins
`
`Haemophilia
`
`Breakthrough Medicines
`
`Vaccines} lP
`
`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
`
`n-
`
`‘ 5'
`
`Page 12 of 101
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`Progress through Stage Gates in 2012
`
`N
`
`ew
`Product
`Opportunity
`
`Research
`
`Product
`Development
`& GLP
`Toxicology
`
`Phase I
`(FIH)
`
`
`
`.
`V
`Registration
`
`POS‘
`Re istration
`9
`
`EZ‘:;|:::::
`5 GLP Tax
`
`Enter
`Phase I
`
`ééé»
`
`Hizentra
`Canada, EU
`Berinert
`Se" Admin
`
`Enter
`Phase II
`
`Enter
`Phase III
`
`Register
`Launch
`
`.
`Hrzentra CIDP
`
`Fibrrnogen
`Aomc EU
`
`Subcur
`
`p
`Privigen CIDP
`7
`EU
`
`Benplex US
`Bleeding
`
`I
`
`CSIL
`
`Enter
`Research
`
`13
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`Global R&D Portfolio D
`
`December 2012
`
`.
`
`.
`
`
`
`.
`
`.
`
`Commercial!
`
`
`
`lmmunoglobulins
`
`Haemophilia
`
`Specialty Products
`Influenza Vaccine
`
`Hizentraté‘ US/EU
`
`“-3.1”-
`SelfAdmin
`
`
`
`Hizentrafii? CIDP
`
`Privigeéfi? CIDP
`Biostatexfi" EU
`
`.
`Zemaira® EU
`
`.
`
`.
`Berlplex® US
`
`Bennert® SC
`
`
`
`Ham
`Aortic EU New Indications
`New Indications
`
`
`
`Life Cycle
`Management
`
`Market
`
`Development
`
`New Product
`
`Development
`
`Novel Plasma
`Proteins
`
`WWPFP
`
`Rec Coagulation
`Factors
`
`Partnered Vaccine
`Prerams‘
`P gingivalis POD
`CRC-OHS/Sanofi *
`
`CSLGRQ er Ia FP
`
`CSL627 erllrSC
`
`CSL654 rIX-FP
`
`Partnered Vaccine Partnered Vaccine Partnered Vaccine
`
`
`
`PrOJECtS
`
`CSL346 VEGFB
`CSL334 lL-13R
`
`CSL324 G-CSFR
`
`CSL362 IL-3R
`
`CAM3001
`
`
`
`csu12
`reconstituted HDL
`
`Core Capabilities
`
`lmmunoglobulins
`
`Haemophilia
`
`Breakthrough Medicines
`
`Vaccines} lP
`
`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
`
`n-
`
`‘ 5'
`
`Page 14 of 101
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`age 15 of 101
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`— P
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`Immunoglobulins
`
`Page 15 of 101
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`

`Immunoglobulins
`
`Maintaining leadership
`position through focus on:
`- Patient convenience
`
`' Yield
`
`- Label
`
`- Formulation science
`
`Key FOCUS
`- Hizentra®
`
`- Privigen®
`
`
`
`Breakthrough
`medimnes
`
`Immunoglobulins
`
`Products
`
`,
`
`_
`
`- Specialty lgs Haemophiiia
`
`iMM csr
`
`H
`
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`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®
`CS[
`
`17
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`Cytogam®
`
`CYTOGAM’
`
`Cytomegaluvims Immune Globulin
`Intravenous (Human) [CMV-lVlGl
`
`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 J}! g:
`commenced December 2011
`«~va
`.
`a
`- CSL donating Cytogam®
`3-3:;
`. Primary analysis expected 2016
`
`18
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`CSIL
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`Hizentra®
`
`Hizéntra"
`
`Immune Globulin Subcmanews
`lHumanl
`20% Limid
`
`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
`
`
`:
`)-
`
`
`
`Ifil E
`
`- supports safety and efficacy of Hizentra® for PID
`
`- new drug application submitted to PMDA in Sept 12
`
`19
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`CS[
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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
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`Hare”
`
`immune doom Subcmanews
`[Human]
`20%Litpid
`
`CSE
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`Page 20 of 101
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`

`

`Chronic Inflammatory Polyneuropathy (CIDP)
`
`Amman,
`
`- A chronic peripheral nerve
`
`disease with progressive
`
`muscle weakness and loss
`
`of sensation, usually
`
`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.
`
`
`
`"ms/hands
`
`Myelin [heath - The protective
`covering around nerve fibers.
`which allows the messages
`to move quickly.
`
`Peripheral nerve:
`
`21
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`‘
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`csr;
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`Potential benefits of Hizentra® in Patients
`
`with CIDP
`
`Current maintenance
`
`
`Oral
`
`steroids
`
`
`
`
`Adverse
`Less CONVemence
`
`
`effects with
`
`
`long term use Levels show peaks
`& troughs
`
`Hospital visits
`
`
`Plasma
`Exchange
`
`Invasive therapy
`_
`_
`_
`..
`lelted aVallabllltY
`Short term efficacy
`
`A 'd d
`VOI s
`route
`
`f.
`k
`b
`raw ac so |.V.
`
` Hizentra®
`
`Reduced volume
`
`Increases patient autonomy
`_
`.
`Less systemic Slde effects
`More stable lgG levels
`
`therapies
`
`
`
`
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`The PATH Trial
`
`: Hizentra® in CIDP
`
` L‘
`m" 0—* *
`
`!>
`
`
`
`
`
`- 150 patients
`
`- 2 doses vs placebo
`
`- Study approved by
`FDA, EMA, PMDA
`
`- Recruiting in US &
`EU
`
`23
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`CSE
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`PrIVIgen®
`
`P2115
`
`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
`
`- Phase III study in ClDP completed in Europe
`
`Ga
`[JPN—TWO
`
`- Study shows treatment with Privigen® improved function
`in patients with chronic ClDP
`
`- Dossier submitted to EMA in May 12
`
`24
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`CS[
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`Privigen® in CIDP: Rate of Response
`
`100 ~
`
`
`
`Responderrate(%)
`
`90 *
`
`8O *
`
`70 7
`
`60 *
`
`50 7
`
`10*
`
`40*
`
`30-
`
`20*
`
`Total
`
`IVIg pretreated
`
`IVIg untreated
`
`CSE
`
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`age 26 of 101
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`— P
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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
`
`- Novel modes of Immunoglobuiins
`
`SpeCIaity
`Products
`
`administration
`
`Haemophilia
`Products
`
`Key Focus
`
`' Ber|P|6X®
`0 Fibrinogen
`
`- Zemaira®
`
`- Berinert®
`
`
`
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`

`Beriplex®
`
`BeripleXP/N!
`
`- Prothrombin Complex Concentrate = PCC
`
`. vitamin K-dependent coagulation factors (F ll, 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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`(SI:
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`

`Fibrinogen®
`
`”i957”?
`
`fllrlrvi'zaqew Cancem/dre
`
`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
`
`- ln dialogue with FDA
`
`29
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`(SI:
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`Zemaira®
`
`(j Z:
`
`alpha1-prole
`
`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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`Beri nert®
`
`Bsnfié‘fir-
`
`Plasma derived, pasteurised & nanofiltered concentrate of C1 Esterase
`
`Inhibitor indicated for the treatment of acute abdominal or facial attacks
`
`
`
`
`
`Genetic Mutation
`
`C1-lNH Deficiency
`
`‘
`
`‘\'
`
`Vascular Permeability
`
`Plasma Cascade
`
`Dysregulation
`
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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
`
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`Berinert®
`
`Bsnffié-‘hr
`
`Plasma derived, pasteurised & nanofiltered concentrate of C1 Esterase
`
`lnhibitor 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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`CS[
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`

`Overcoming Challenges in Long-term
`Prophylaxis of HAE Attacks
`
`Current prophylactic
`
`therapies
`- Low volume for s.c. administration
`
`
`Oral
`androgens
`. Limited by
`adverse
`.
`efieCts.’
`espeCIally in
`women and
`
`children
`
`Intravenous
`High concentration
`C1 -lNH
`subcutaneous (sc) Berinert®
`. Inconvenience Q - Avoids drawbacks of iv.
`and risks of
`remitted "Y'
`- Builds on well—established safety
`administration
`.
`profile
`
`‘
`
`34
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`Berinert® Subcutaneous Prophylaxis Program
`
`COmPQCT
`
`Clinical Studies for 0pt:ma| Management in
`Preventing Angioedema with low-volume
`subcutaneous Ci -inhib:tor Replacement Theiapy
`
`Safety and
`pharmacokinetic
`study
`
`- due to commence 2H 2013
`
`- Study ongoing in US and Germany - due to complete 1H
`2013
`
`.
`,
`.
`_
`_
`- Select safe and efficacrous dosmg for clinical efficacy trial
`
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`age 36 of 101
`
`— P
`
`Commercial Opportunities
`
`and Activities
`
`Page 36 of 101
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`

`

`age 37 of 101
`
`— P
`
`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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`

`

`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
`
`
`
`b.
`DPIVIQEH“
`
`i\J!i.x Vin
`
`Huanm Norcram i'nn noowglbl
`[W Hintfi.
`suiiizplw
`
`First and onl Proline-stabilised IVIG
`y
`- High purity 10% liquid IVIG
`- Optimal dimer formulation throughout shelf life for
`improved tolerability
`
`
`/ ‘ First and only 20% Proline-stabilised SCIG
`H izentra®
`- Low administration volume increases efficiency
`
`lmmuneGlobulin Subculaneous(Human))20%Liquid
`
`. Convenient _ few sites & fast infusion
`- Broad indications - Reconstitution options
`
`" Lyophilised IVIG
`
`- Long track record of safety & reliability
`
`Sandoglobulins/ ' l 9.25.
`
`40
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`Page 40 of 101
`
`

`

`Globalise Portfolio
`
`Privigen 8. Hizentra
`(PL)
`
`Privigen & Hizentra
`(HU)
`
`Hizentra (MX)
`
`Privigen & Hizentra
`(CZ)
`
`Privigen (TR)
`
`Privigen (MY)
`
`Privigen (SG)
`
`Privigen (BR)
`
`Privigen (TW)
`
`Privigen (MX)
`
`Privigen (RU)
`_
`
`Hizentra (TR)
`Hizentra (BR)
`_ _
`
`Hizentra (AR)
`
`Privigen (HK)
`
`Hizentra (JAP)
`
`- Privigen® currently registered in 55 countries
`
`- Hizentra® currently registered in 33 countries
`
`- Continue global launches for Privigen® and Hizentra®
`
`41
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`CSIL
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`

`

`Expand into Neurology
`
`
`Hizentra 20%
`
`CIDP (EU)
`
`Privigen 10% CIDP
`(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
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`CSIL
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`
`

`

`Increase Convenience
`
`Privigen 409 Presentation
`
`(US, CH, EU)
`
`
`
`Hizentra Convenience Initiatives
`
`Hizentra109
`(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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`CSIL
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`Page 43 of 101
`
`

`

`Clean surface
`
`‘
`.
`_
`Sanrtlze your hands
`
`
`
`Gather your supplies
`
`Inspect each vial
`of Hizentra
`
`Prepare syringe
`
`Hiz'entra“
`
`Immune kafln Sulxxrranews (Human) 20%qu
`
`Infusion Steps
`
`4 preparation steps per vial
`
`
`
`
`
`
`‘9'
`
`
`
`HNIHIUUuu
`
`
`-J 1‘ I
`(ll 04
`:6:
`
`
`
`Write in your journal
`
`44
`
`Page 44 of 101
`
`Reducing number of vials
`9 increases convenience
`
`
`Prepare syn
`
`(continu
`
`
`
`Start infusion
`
`
`
`Prepare syringe
`
`(continued)
`
`
`
`'
`
`'
`
`I
`
`":5;
`
`'
`
`Insert Sub-Q needle(s)
`
`Prepare injection s'rte(s)
`
`..
`
`CSL
`
`Page 44 of 101
`
`

`

`Cytogam®
`
`CYTDGAM’
`
`Cvtumegalwima mrmne Gobulin
`Immatum [Hurranl (CMV-ME!
`
`The only registered CMV immunoglobulin in the US indicated for the
`
`prevention of CMV disease associated with transplantation
`
`Center for Disease Controll:
`
`. CMV is the most common viral
`
`Pmemia' Opp°nunity
`
`infection that infants are born with
`.
`_
`In the United States
`
`.
`_
`Assuming 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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`

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`age 46 of 101
`
`— P
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`Specialty Products
`
`Page 46 of 101
`
`

`

`Specialty Products
`
`2011 2012
`
`Other Specialty Products
`
`‘
`
`’-
`
`. Wound Healing
`
` ‘
`
`Kybernin
`
`Bednen
`
`Zemaira
`
`Streptase
`
`Fibrogammin
`
`Beriplex
`
`Riastap
`
`I $U3618 M
`
`- Increase clinical data set
`
`. Add indications
`
`- Expand regionally
`
`47
`
`Page 47 of 101
`
`Perioperative Bleeding
`
`CSIL
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`Page 47 of 101
`
`

`

`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
`
`IMS data July 2012
`
`Connolly et al NJEM 2009, Patel et al NEJM 2011, Granger et al NEJM 2011
`
`1)
`
`2)
`
`48
`
`CSIL
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`Page 48 of 101
`
`

`

`Blood Products vs. Concentrates
`
`'
`«-
`Err—1'sHP'
`2
`f
`QM; itm% flmg
`
`Not virus inactivated
`
`' Frozen‘ requires time (<50 minutes) to thaw
`
`Red Blood Cells
`
`Need to be matched to blood type
`Not virus inactivated
`
` Fibrinogen concentration at =2.39 / L
`
`Concentrated, virus inactivated room
`temperature storage, Fibrinogen concentration
`20g / L
`
`'
`
`"
`
`‘
`
`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
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`Page 49 of 101
`
`

`

`Fibrinogen®
`
`Rinsmpé '
`
`F/b/mogen (anal/71732?/
`
`- Obtain WEU & US acquired label
`
`. Initiate acquired label expansion
`
`- Generate, publish & communicate
`
`data
`
`INTERNATIONAL SYMPOSIUM OF INYENSIVE CARE AND EMERGENCY MEDICINE
`
`[mammal
`
`E uropean
`Sooety of
`Anaesthesxology
`
`A
`
`
`
`50
`
`Page 50 of 101
`
`
`
`CSE
`
`Page 50 of 101
`
`

`

`Bring <\,£/Ihira“ 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
`
`. Reimbursement
`
`. Enhanced testing & diagnosis
`
`Page 51 of 101
`
`Page 51 of 101
`
`

`

`Berinert®
`
`BERIDERT’
`
`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
`
`
`
`Product
`
`improvement
`
`- Low—volume, high
`.
`.
`concentration, formulation
`
`.
`
`Be
`
`W P . "‘5'"
`' ”M ‘
`
`._
`
`I
`
`llllll
`
`Expand
`indication
`
`
`
`oShort-term prophylaxis,
`
`2013 (EU/Can)
`- Further initiatives ongoing
`
`compmc
`
`New
`method of
`use
`
`.Prophylaxis Indication
`with so. formulation
`
`
`
`53
`
`Page 53 of 101
`
`’ lr‘ll
`uh
`vl
`l'.0l,1'lle1;l:lfl€l'
`"ii
`M C H :W '2“
`
`CSIL
`
`Page 53 of 101
`
`

`

`age 54 of 101
`
`— P
`
`Page 54 of 101
`
`

`

`age 55 of 101
`
`— P
`
`Page 55 of 101
`
`

`

`age 56 of 101
`
`— P
`
`Haemophilia Products
`
`Page 56 of 101
`
`

`

`Haemophilia
`
`Supporting and enhancing
`plasma products and developing
`
`novel recombinant portfolio with
`
`
`focus on:
`Breakthrough
`“
`_
`_
`_
`° Screntrfrc and product
`innovafion
`
` Medicines
`
`lmmunoglobulmc
`/
`
`X
`
`SPeC'a'ty
`\\ Products
`
`/
`
`\._
`
`Haemophilia
`Products
`
`° Patient benefit
`
`Key Focus
`' Long actlng rIX-FP
`- Long acting erla-FP
`
`- erll-Single Chain
`
`- Research into long acting
`rVWF-FP
`
`
`
`Page 57 of 101
`
`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
`
`rAIbumin
`as fusion
`platform
`
`Precise
`engineering
`of specially
`designed
`linker
`
`- Factor VIII
`
`High VWF affinity
`
`- biobetter erlI-SingleChain
`
`58
`
`Page 58 of 101
`
`Improved molecular stability
`
`Opportunity for Extended
`Dosing interval
`
`CSIL
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`

`

`rlX-FP (CSL654)
`
`
`
`CS[
`
`%P
`
`ROLONG 9 PF
`
`59
`
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`Page 59 of 101
`
`

`

`rlX-FP (CSL654) Clinical Program
`
`Phase 1/2
`PK
`
`Phase 2/3
`PK
`
`Phase3
`Paediatric
`
`Long-term safety
`
`Long-term safety
`
`Surgical prophylaxis
`
`7d prophylaxis
`
`On-demand
`
`7-14d prophylaxis
`
`On—demand
`
`Extension
`
`’—/
`
`PROLONG 9 FP
`
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`

`

`blood Prepublished online August 2, 2012:
`
`doi: 10 1182/blood-2012-05429688
`
`2012 120: 2405-2411
`
`Safety and pharmacokinetics of a novel recombinant fusion protein
`linking coagulation factor IX with albumin (rlX-FP)In hemophilia B
`patients
`Elena Santagostino Claude Ne rier, Robert Klamroth, Andreas Tiede. Ingrid Pabinger-Fasching,
`5U
`Christine Voigt Iris Jacobs and
`assimo Morfini
`
`
`
`FIXActivity(IUIdLl
`
`
`
`FIXAdiviyllU/dl)
`
`70'
`
`+50lU/kg rlX-FP 01:1?)
`—0— 50 Iu/kg lrlK (n-a)
`-r- 50 mm; delX (MI
`- - -‘ 5% trough
`
`O
`
`24
`
`48
`
`72
`
`$6
`
`120
`
`I“
`
`168
`Hours
`
`132
`
`216
`
`240
`
`164
`
`288
`
`312
`
`336
`
`
`
`
`—o—25 IU/ks rIX-FP ("=7)
`+50 lU/kg rlX-FP(n=13l
`—.— 75 Iu/kg rIX<FP In=8i
`---- 5% iruugh
`....... 295 Waugh
`
`Compared with in market rFlX
`
`- 5.3-fold longer half-life (92hrs)
`
`- ~ 45% higher incremental recovery
`
`- ~7-fold larger AUC
`
`- ~7-fold slower clearance
`
`_‘
`
`1’v
`
`PROLONG 9 PF
`
`61
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`Page 61 of 101
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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 PF
`
`62
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`Page 62 of 101
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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 FP
`
`63
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`Page 63 of 101
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`

`

`rlX-F P (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
`
`PROLONG FP
`
`64
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`Page 64 of 101
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`

`

`rVIIa-FP (CSL689)
`
`
`
`F’RDLDNG 7 F9
`
`65
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`

`

`Development of erla-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 PEI
`
`. Briefing documents to FDA/ EMA
`_,_\
`
`__-/
`>
`pRDl—DNG 7 F9
`
`66
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`Page 66 of 101
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`

`

`Phase 1 Study of rVIIa-FP in Healthy Volunteers
`
`
`
`
`
`CorrectedplasmaFVIIactivity[mU/mL]
`
`
`
`
`
`
`
`Half-life 3-4 fold longer than rFVlla
`
`/
`
`,- ‘
`
`‘
`
`.5/
`PROLONG 7 FF’
`
`67
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`Page 67 of 101
`
`

`

`Potential of rVIla-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
`
`EJ”
`PROLONG 7 Fl:
`
`68
`
`Page 68 of 101
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`CS[
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`Page 68 of 101
`
`

`

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

`

`erll—SingleChain: approach for improved FVIII
`
`- 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
`
`. 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
`
`~200
`
`r"""""
`
`l - I
`
`""I
`
`80 kDa
`
`erll-SingleChain
`
`CSIL
`
`7°
`
`Page 70 of 101
`
`Page 70 of 101
`
`

`

`rVIII-SingleChain: high affinity for vWF
`
`Binding to plasma-derived (pd) VWF
`
`Comparison of VWF affinity constants
`
`250
`
`200
`
`
`
`Relativeunits
`
`150
`
`100
`
`50
`
`
`
`— Single chain rFVlll (CSL627)
`
`— Full length rFVlll
`
`0
`
`500
`
`1 ,000
`
`1 ,500
`
`Surface plasmon resonance (SPR) analysis Time (S)
`CSL Behring. Data on file
`
`71
`
`Page 71 of 101
`
`
`
`
`
`Affinityconstant(1/(nm)
`
`20
`
`18
`
`16
`
`14
`
`12
`
`10
`
`ON-hmm
`
`
`
`erll-SingleChain
`(CSL627)
`
`Full length rFVlll
`
`CSIL
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`

`

`rVIII-SingleChain : PK profiles in rodents
`
`PK in haemophilia A mice
`
`pK in rats
`
`6,000
`
`3
`E 4,000
`2
`E
`2:
`'5'
`>
`u.
`
`3 000
`'
`2,000
`
`1,000
`
`100 lU/kg FV|||:C
`_
`.
`.
`n—5/timepomt
`geometric means 1 SD
`
`5
`
`4
`
`3
`
`3 E
`
`a
`3
`>
`a:
`3
`g
`a
`<_.>_
`..=.
`>u.
`
`5,000
`
`200 lU/kg FV|||:C
`n=3/timepoint
`.
`geometric means i SD
`
`
`
`0
`
`0
`
`20
`
`40
`
`60
`
`80
`
`0
`
`120
`
`240
`
`360
`
`480
`
`Time (hrs)
`
`— CSL627
`
`_ Full-length rFVIII
`
`Time (min)
`
`CSL627 has ~ 50% increase in terminal half-life compared to full-length rFVllI
`
`72
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`Page 72 of 101
`
`

`

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

`

`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)
`
`-
`
`.
`
`Phase III“ 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
`
`CSIL
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`Page 74 of 101
`
`

`

`age 75 of 101
`
`— P
`
`Commercial Opportunities
`
`and Activities
`
`Page 75 of 101
`
`

`

`Coagulation Sales
`
`2011 2012
`
`
`
`
`I 51181 ,05‘8M
`
`- Launch rlX-FP in 2015
`
`. Launch rVIll—SingleChain as bio-better in 2016
`
`- Grow pd FVIII
`
`76
`
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`
`
`
`CSIL
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`Page 76 of 101
`
`

`

`Coagulation: Total Market Size
`Key Market Segments and Products
`
`- Beriate
`
`- Helixate
`
`-
`
`,
`
`..
`
`‘
`
`Target
`Segments
`
`management
`
`a
`
`'
`
`- Haemate P
`
`
`
`. Biostate
`
`- HaemateP
`
`- Mononine
`
`‘ - Berlnln
`
`77
`
`CSL Market Estimate 2012
`
`Page 77 of 101
`
`CSIL
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`Page 77 of 101
`
`

`

`--
`
`Chain
`
`- Helixate
`
`NexGenIFSI81
`
`- erll-Single
`
`Coagulation:
`Key Market Segments and Products.
`
`- Beriate
`
`
`
`
`
`-
`
`- erIa-FP
`
`..
`
`_.
`"
`
`‘
`
`I
`
`v
`
`‘
`
`Target
`Segments
`
`pd ITT
`minggggnwfn
`
`t
`
`.
`
`'
`Blostate
`- Haemate P
`
`_
`. Blostate EU
`
`-
`
`rVWF-FP
`
`- Mononine
`. Berinin
`
`78
`
`CSL Market Estimate 2012
`
`Page 78 of 101
`
`(SI:
`
`Page 78 of 101
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`

`

`Recombinant Coagulation Portfolio
`
`W' Fl $051";
`
`Berinln" P
`
`
`
`
`
`. Differentiate recombinant albumin fusion platform and launch rIX-FP
`
`. Differentiate and launch erll-SingleChain
`
`. Strong support for Helixate and growth of rVIIl—SingIeChain
`
`79
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`CSIL
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`Page 79 of 101
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`

`

`rIX-FP (CSL654) & rVIIa-FP (CSL689)
`
`Scientific Edge
`
`”mm
`
` PROLONG
`rCoags Slide Kit Jan 2012
`
`Improved half
`life, extended
`doang
`interval
`
`Recombinant
`Albumin as
`fugon
`parhter
`
`Specifically
`designed
`aner
`
`Innovations
`
`in Coagulation
`
`80
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`Page 80 of 101
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`Page 80 of 101
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`

`

`rlX-FP: The Scientific Edge
`
`Phase 1 data
`
`rFlX- PEGylated 2
`
`rFlX-Fc fusion 3
`
`rIX-FP 1
`(CSL Behring —
`
`Albumin Fusion)
`
`
`
`PROLONG
`
`FP
`
`ProphyIaXIs 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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`Page 81 of 101
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`Page 81 of 101
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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 clinical
`
`development which enhances the dur