Our purposeWe enable people with life-altering conditions to lead better lives.
Innovation
Exane BNP Paribas 14th Healthcare Conference
10th May 2012
Sylvie GrégoirePresident, Human Genetic Therapies
To be as brave as the people we help.
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The Start of Our Journey: Shire’s First Headquarters (1988)
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Transformation continues
2004
• ADDERALL XR and ADHDthe prime focus
• Small molecules• Oral drug delivery (SLI)• Hatch Waxman dependent• US the dominant market• Presence in Canada and
6 EU markets
2012
• Leading Specialty Biopharmaceutical Company
• Business based on:• Small molecules• Peptides• Biologics
• Technology platforms• Human cell line biologics• Carrier Wave• Locked Nucleic Acid Technology
• ADHD, GI, HGT and Regenerative Medicine• Balanced product portfolio
• Products with global rights• Strong pipeline• Robust Intellectual property• Presence in 29 countries and growing
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Global Expansion and Some Success (2012)
Lexington, MA
Basingstoke, UK
Chesterbrook, PA
Nyon, Switzerland
71%
68%
5%Sao Paolo, Brazil San Diego, CA
2012 Eurordis Company AwardExemplary work in the field of rare diseases
2011 Company of the Year (2nd year)European Medicine Awards
2011 ISPE Facility of the Year AwardHonorable Mention
Named one of the top women-led businesses in Massachusetts (2011)
Named one of Massachusetts High Tech’s “Tech Citizens” for
our philanthropic work (2010)
2010 UK Prix Galien award for FIRAZYR
2010 Partners in Progress Award for Accelerated Development of VPRIV
National Organization for Rare Disorders (NORD)
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Shire HGT has an Extensive Global Footprint: Direct Presence in 29 Countries with Sales in 48
Direct PresenceKey Partner Distribution Sites
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Human Genetic Therapies (HGT)Orphan Diseases
Specialty Pharmaceuticals (SP)Attention Deficit Hyperactivity Disorder (ADHD) ,
Gastrointestinal (GI), Renal, Hematology
Advanced BioHealing (ABH)Regenerative Medicine
Shire: Three Decentralized BusinessesFocus on symptomatic diseases and individual patient needs is key to Shire’s success
Acquisition of ABH adds a strategic platform in Regenerative Medicine
Vascugel®
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KEYPRODUCTS
LATE STAGE PIPELINE
EARLY STAGE PIPELINEVALIDATING TECHNOLOGY PLATFORMS
RESOLOR
LIALDA
VYVANSE
ELAPRASE
REPLAGAL
VPRIV
INTUNIV
FIRAZYR
DERMA-GRAFT
VYVANSEEx-US
Investing to deliver growth now and into the future, supported by strong cash generation
VYVANSEMDDVYVANSE
BED
INTUNIVEx-US
Specialty PharmaVYVANSE NSS&EDS
CarrierwaveHematology
Movetis assets
HGT Hunter CNSSanfilippo ASanfilippo B
DMDMLD
Emer
ging
Res
earc
h As
sets
SPD602IRON
OVERLOADRMVASCUGELRegen Med
assets
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NMEs per $B R&D spent (inflation adjusted)
Structure of DNA
Restrictionenzymes
DNA sequencing
RecombinantDNA
Humaninsulin
Dolly theSheep
Humangenome v1
0.1
1
10
100
1950 1960 1970 1980 1990 2000 2010
Note: R&D costs are estimated from PhRMA annual survey 2009; NMEs are the total number of small molecule and biologic approvals by the FDASource: Bernstein Research “The Long View – R&D Productivity” (September 30, 2010), Source Boston Consulting Group
Pharma R&D Productivity Consistently Declining:A Broken Business Model
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Charles Darwin
It is not the strongest of the species that survives, nor the most intelligent that survives. It is the one that is the most adaptable to change.
“
”
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Delivering Value in a Challenging Environment
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Our Core Focus
Profoundly impacting the lives of patients who suffer from devastating rare diseases
Provide financial return to shareholders
Clearly address the rapidly changing needs of stakeholders in the Circle of Value
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R&D as an Engine of Growth and Change
Time
Gro
wth
Time
Gro
wth
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Research-Process Development Partnership: Joint Accountability to Deliver Therapies More Quickly
Knowledge
Time
Early Development
Process Development
Knowledge
Time
Early Development
Process Development
Partnering
Over The Fence
Discovery
Discovery
Transition
Transition
• Less duplication• Faster• Consistency• Flexibility• Reduced risk• Leverage resources for
new tech platforms• Co-location of Res and
PD personnel
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• Increased intensity of focus on vendor management• Align incentives• Demand high quality and performance from vendors
• Nimbler internal processes• Speed of decision-making• Enhanced partnership with procurement
• Leverage increasing scale• Relationships across HGT, SP, ABH• Establish global strategies (e.g. for clinical studies)
• Preferred relationships• Opportunities for broad commitments with the right suppliers
• Creativity• New models which align with key strategic needs• Social networking
New Supplier Relationships
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Innovation and Creativity Focused on Business Needs
• Creative Preclinical and Clinical Science
• Creative engagement of Regulatory authorities
• Creative external partnerships
• Creative ways to decrease time to approval
• Creative partnerships within Shire
• Creative people development opportunities
• Create value to Payers
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Creativity Focused on Business Needs: Intrathecal Technology
• Challenge Paradigms
• Apply Technology In New Ways
• New Business Relationships
• Profound Impact OnCurrent and New Patients
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Creativity Focused on Business Needs:Manufacturing Technology
• Challenge Paradigms
• Apply Technology In New Ways
• New Business Relationships
• Profoundly Impact OnCurrent and New Patients
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Creative External Partnerships: Alliance with Atlas Venture
We recently announced that R&D has formed an alliance with Atlas Venture
Objective• To identify and invest in early-stage companies formed around single assets for which
Shire will retain an exclusive optionScope• Rare disease therapeuticsAdvantage• Combining Atlas Venture’s entrepreneurial network and experience in new company
formation with HGT’s expertise in rare disease research, development, and commercialization
We believe that multiple investments across project-focused companies will mitigate early-stage development risk
Retaining rights to acquire these assets after de-risking allows for expansion of the pipeline while maintaining organizational flexibility
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Creative External Partnerships:Strategic Investment Group (SIG)
Focusing on the diagnosis and treatment of food allergies such as peanut and milk allergies. DBV Technologies’ novel approach to food allergy diagnosis and treatment is focused on eliciting an immune response by targeting antigen to the skin epidermis. The antigens (allergens) are delivered to the skin using DBV Technologies’ unique worldwide patented delivery system, VIASKIN®www.dbvtechnologies.com
Advancing treatment of neurodegenerative diseases through a novel approach to disaggregating proteins using plaque technology. NPT001, the lead molecule, has been shown in animal models to disaggregate amyloidplaques.www.neurophage.com
Prexa develops proprietary oral small molecule therapeutics targeting treatment of neuropsychological disorders. Prexa’s proprietary chemistry approach is focused on discovery of highly differentiated tri-reuptake inhibitors with unique dopamine-biased neurotransmitter transport selectivity profiles and rationally designed CNS PK. Prexa is also testing mono- and di-reuptake inhibitors for activity in ADHD animal models.www.adventhealthcareventures.com/prexa
Naurex Inc. is a clinical-stage company developing novel therapies for depression and other CNS disorders based on a new mechanism of action for modulating the N-methyl-D-aspartic acid receptor (NMDAR). Using these discoveries, Naurex researchers have generated novel chemical drug classes known as glycine-site functional partial agonists (GFPAs), which modulate the receptor in a different way than existing NMDAR agents. The company’s drug development programs include the first-generation molecule, GLYX-13, the second-generation NRX-1050 small molecule series and additional compounds from Naurex’s platform of novel NMDAR modulators. www.naurex.com
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Creative External Engagement:Social Networking
• Patient recruitment through CRO’s• Patient education• Patient engagement (e.g. patientslikeme)• Internal Shire engagement• Problem solving (e.g. exploring open innovation with Harvard)• ………
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Delivering Value in a Challenging Environment
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Balanced product portfolio provides foundation for future growth
Delivering good revenue and earnings growth
Demonstrating value to stakeholders in a changing healthcare environment
Expanding the pipeline through new acquisitions and partnerships