2. To be as brave as the people we help.
2
THE “SAFE HARBOR” STATEMENT UNDER THE PRIVATE SECURITIES
LITIGATION REFORM ACT OF 1995
Statements included in this announcement that are not historical facts are forward-looking statements. Forward-looking statements involve a number
of risks and uncertainties and are subject to change at any time. In the event such risks or uncertainties materialize, Shire’s results could be
materially adversely affected. The risks and uncertainties include, but are not limited to, that:
• Shire’s products may not be a commercial success;
• revenues from ADDERALL XR are subject to generic erosion and revenues from INTUNIV will become subject to generic competition starting in
December 2014;
• the failure to obtain and maintain reimbursement, or an adequate level of reimbursement, by third-party payors in a timely manner for Shire's
products may impact future revenues, financial condition and results of operations;
• Shire conducts its own manufacturing operations for certain of its Rare Diseases products and is reliant on third party contractors to manufacture
other products and to provide goods and services. Some of Shire’s products or ingredients are only available from a single approved source for
manufacture. Any disruption to the supply chain for any of Shire’s products may result in Shire being unable to continue marketing or developing
a product or may result in Shire being unable to do so on a commercially viable basis for some period of time.
• the development, approval and manufacturing of Shire’s products is subject to extensive oversight by various regulatory agencies. Submission of
an application for regulatory approval of any of our product candidates, such as our planned submission of a New Drug Application to the FDA for
Lifitegrast as a treatment for the signs and symptoms of dry eye disease in adults may be delayed for any number of reasons and, once
submitted, may be subject to lengthy review an ultimately rejected. Moreover, regulatory approvals or interventions associated with changes to
manufacturing sites, ingredients or manufacturing processes could lead to significant delays, increase in operating costs, lost product sales, an
interruption of research activities or the delay of new product launches;
• the actions of certain customers could affect Shire's ability to sell or market products profitably. Fluctuations in buying or distribution patterns by
such customers can adversely impact Shire’s revenues, financial conditions or results of operations;
• investigations or enforcement action by regulatory authorities or law enforcement agencies relating to Shire’s activities in the highly regulated
markets in which it operates may result in the distraction of senior management, significant legal costs and the payment of substantial
compensation or fines;
• adverse outcomes in legal matters and other disputes, including Shire’s ability to enforce and defend patents and other intellectual property rights
required for its business, could have a material adverse effect on Shire’s revenues, financial condition or results of operations;
• Shire faces intense competition for highly qualified personnel from other companies, academic institutions, government entities and other
organizations. Shire is undergoing a corporate reorganization and the consequent uncertainty could adversely impact Shire’s ability to attract
and/or retain the highly skilled personnel needed for Shire to meet its strategic objectives;
• failure to achieve Shire’s strategic objectives with respect to the acquisition of ViroPharma Incorporated may adversely affect Shire’s financial
condition and results of operations;
and other risks and uncertainties detailed from time to time in Shire’s filings with the U.S. Securities and Exchange Commission, including its most
recent Annual Report on Form 10-K.
3. To be as brave as the people we help.
3
We have created a platform to deliver sustainable
growth and superior patient outcomes
Sustainable
Growth
Operational
Efficiency;
Commercial
Excellence;
Scalable Model
Leading
Franchises;
Innovative
Portfolio;
Disciplined M&A
Simple Structure and
High Performing Management
Superior
Patient
Outcomes
4. To be as brave as the people we help.
4
We have accelerated our growth
1 Product sales are from continuing operations, and exclude DERMAGRAFT which is treated as a discontinued operation following divestment in January 2014.
Internal
Medicine
GI
Neuro-
science
Rare
Diseases
Q1 2014
1,308
Q1 2013
1,098
1Q 2014 Product sales growth1
$ Million; Percent growth
+19%
+39%
+16%
+17%
(11)%
Improved Rare Diseases
competitive performance
Added ViroPharma, including
CINRYZE in Rare Diseases
Management actions
Sharpened Neuroscience
customer focus
Achieved #1 position
in 5-ASA market
Divested DERMAGRAFT to
a more natural owner
5. To be as brave as the people we help.
5
Q1 2014: Strategy delivers strong results
• Serve patients with high unmet needs
in select, commercially attractive
specialty therapeutic areas
• Drive optimum performance of our
In-line marketed products
• Build our pipeline of innovative
treatments through both R&D and BD
activities
• Ensure efficient use of resources
through a simplified corporate
structure
Our refined strategy
2014 Q1 EBITDA 41%
2014 Q1 Revenue 19%
2014 Q1 Cash Generation 29%
Full-year 2014 earnings guidance
upgraded following Q1 results
6. To be as brave as the people we help.
6
Human Genetic
Therapies
Specialty
Pharmaceuticals
Regenerative
Medicine
Organizational transition
1Q 2013
One
Shire
Current
4x commercially focused
business units
1x integrated
R&D organization
Significant operating leverage
1x international infrastructure
with presence in 50+ countries
Core components
Streamlined supporting functions
Rapid transition to simplified structure has
increased accountability and agility
7. To be as brave as the people we help.
7
Significant increase in 1Q EBITDA margin(1)
$ Million; Percent of product sales
Program details
(1) Results exclude DERMAGRAFT which is treated as a discontinued operation following divestment in January 2014.
(2) Excluding royalties and other revenues.
Integrating R&D under a
single leadership team
Created a single, consolidated
international structure
Streamlined other
corporate functions
Halted programs that did not fit
strategy or commercial criteria
Divested underperforming
businesses (DERMAGRAFT)
SG&A
EBITDA(2)
100%
R&D
COGS
Q1 2014
1,308
Q1 2013
1,098
We have created a step change in efficiency
8. To be as brave as the people we help.
8
S&P 500
FTSE 100
BioPharma
Peers
Source: Company reports, Capital IQ as of 15 May 2014
(1) Median of peers including Alexion, Allergan, Amgen, Biogen, BioMarin, Celgene, Gilead, Merck KGaA, Novo Nordisk, UCB, Valeant and Vertex.
Driving material share price outperformance
Total shareholder return from May 1, 2013 to May 15, 2014
(1)
Accelerated
growth
Increased
efficiency
Disciplined
M&A
Immediate impact
Enhanced
pipeline
These initiatives have had an immediate
impact on share price performance
9. To be as brave as the people we help.
9
$30 billion global market(7)
Market growth estimated to
be 5% from 2013-20(7)
#1 position in 5-ASA market(8)
#2 sales force as ranked by
U.S. GI’s(8)
Sample Pipeline Programs:
Leading positions in attractive therapeutic areas
Shire’s largest business unit
~$75 billion market size
(orphan drug sales) allows
for significant headroom(4)
Market growth estimated to
be 11-12% from 2012-17(4)
#1 position in HAE market(5)
Sample Pipeline Programs:
$8 billion U.S. ADHD market
We expect product sales to
double by 2020 through BED,
SHP465 and geographic
expansion(6)
Sample Pipeline Programs:
VPRIV (Japan)
Gaucher
SHP611
MLD
CINRYZE IV
(low vol)
HAE Prophylaxis
SHP609
Hunter CNS
SHP610
Sanfilippo A
SHP613
Acute Vascular
Repair
LDX*
BED
INTUNIV
(Japan)
ADHD
INTUNIV® (EU)
ADHD
SHP465
ADHD
LUM 001
Cholestatic Liver
Disease
LUM 002
Non-Alcoholic
Steatohepatitis
SHP555 (US)
Chronic
Constipation
$2B in Rare Diseases(1) $1.6B in Neuroscience(2) $0.8B in GI(3)
* Lisdexamfetamine dimesylate, active ingredient in
VYVANSE/ELVANSE.
(1) 2013 product sales on a pro-forma basis including CINRYZE
(2) 2013 product sales exclude ADDERALL XR sales, which is
managed in our Internal Medicine BU (3) 2013 product sales
(4) Kalorama Worldwide Market for Orphan Drugs
(5) Seeking Alpha “Dyax And ViroPharma: An Overview Of HAE Space”
(6) Subject to FDA approval for BED and SHP465
(7) Evaluate Pharma
(8) IMS
10. To be as brave as the people we help.
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Multiple drivers of double digit sales performance
• CINRYZE delivered $86m since Jan 24
• Over 1,100 patients on therapy in the US
• FIRAZYR sales +80%
• Strong US growth continues – more than 1,800 patients have
used FIRAZYR since launch
• ELAPRASE sales +13%
• Driven by continued growth in the number of treated patients
• LIALDA strong performance continues, +28%
• Continued market share gains in the US
• Strong TRx performance continues through 1Q 2014
• VYVANSE sales +18%
• US growth driven by price and volume
• International sales show strong growth
Operational
Discipline
11. To be as brave as the people we help.
11
BED – Under diagnosed and undertreated patient population with
significant morbidity
• Approximately 3MM(1) patient in the US suffering with the disorder
• In a survey of US adults, less than 7% of BED patients reported receiving
treatment over a 12 month period(1)
• Per DSM-5: Condition is characterized by marked distress
• No FDA approved treatment
Targeting 2nd half of 2015 launch - Substantive market analysis is well
underway
• Phase 3 studies complete – achieved primary endpoint in both trials
• On track for a 2nd half of 2014 NDA submission
Binge Eating Disorder – a significant growth
opportunity for VYVANSE
(1) Hudson et al,, 2012, National Comorbidity Survey Replication 2001-2003
Commercial
Excellence
12. To be as brave as the people we help.
12
SHP465: A new product to meet an adult ADHD
market need
• Demonstrated efficacy at 16 hours post-dosing in registration trials
• Planning for a launch in the first half of 2015
• We expect SHP465 to have 3-years of Hatch-Waxman exclusivity and we expect to have
at least two patents listed in the Orange Book expiring as late as May 2023
SHP465
We believe with SHP465 as part of our portfolio we can add
3%-5% to our existing share of the overall ADHD market
Commercial
Excellence
13. To be as brave as the people we help.
13
Lifitegrast for dry eye disease - next steps
Following FDA meeting, preparing for
a Q1 2015 filing
Complete remaining chemistry and
manufacturing work
Build BU / Finalize commercial strategy
2016 Launch
(subject to FDA approval)
Assess the need for gathering additional clinical
data in support of the US filing and potential
international regulatory submissions
Strategy
Shift
14. To be as brave as the people we help.
14
CINRYZE IV
(low vol)
HAE Prophylaxis
LUM 001
Rare Hepatic
Diseases
LUM002
NASH
SHP607
(PREMIPLEX®)
Prevention of ROP
SHP606
(lifitegrast)
Dry eye disease
Reinforce
Core TAs
M&A Strategy
Expand into high
value adjacent TAs
Rationale Recent examples
• Existing infrastructure or expertise
creates “ownership” advantage
• Generate synergies (revenue, cost,
operational) to substantiate control
premium and value creation
• Informed entry into adjacent TAs
with long-term growth potential
where Shire has expertise or can
build core competencies
Disciplined M&A strategy focused on acquiring
in-line and pipeline assets in core and adjacent TAs
Strategy
Shift
15. To be as brave as the people we help.
15
ViroPharma: integration progressing successfully
Drive revenue
from marketed
products
Ensure
uninterrupted
supply
Realize cost
and revenue
synergies
Leverage talent
Extract value
from pipeline
programs
• Cinryze proforma revenue growth Q1 +16%
• Sales force in US integrated with minimal turnover; driving strong
new patient growth
• Resolving outstanding manufacturing issues
• Leveraging Shire’s manufacturing expertise
• Leveraging opportunities in HAE
• Integration of ViroPharma into One Shire organization
• Rapid transition of G&A activities; Commercial and R&D over
longer-term; on track to deliver $150M in savings in 2015
• Leveraging ViroPharma expertise in HAE and strength in patient
advocacy area
• Pipeline programs reviewed and key decisions made
5 key success factors:
Operational
Discipline
16. To be as brave as the people we help.
16
FSGS has severe consequences for
patients and significant societal costs
• ~75,000 patients with primary FSGS in the G6
• Affects children and adults of all ages and
races; increased risk in people of African
ancestry
• In children, it is the second most common
cause of nephropathic syndrome and the
second leading cause of kidney failure
• 60-75% of patients are nephrotic at diagnosis
and eventually progress to ESRD, typically in
about 8 years
• ~25% of patients relapse after treatment
• High recurrence rates in patients receiving
kidney transplants (30% in adults, up to 80%
in children)
The human face of FSGS
“We have a massive unmet medical need,
but we are stuck with histological
diagnosis and working with steroids”
- FSGS KOL
Sources: KOL interview; http://www.nephcure.org/fsgs-facts - site accessed Jan 11th 2014
FSGS is a rare renal disorder affecting both children and adults
and has significant commercial opportunity given high unmet need
Strategy
Shift
17. To be as brave as the people we help.
17
• Expands Shire’s pipeline with a novel clinical stage anti-fibrotic agent
• Strengthens our growing and innovative portfolio targeting renal and
fibrotic diseases
• FT011, the lead molecule, targets an innovative, novel and previously
undescribed mechanism of action and is currently completing a phase 1b
trial in patients with renal impairment
• A phase 2 trial is planned for 2015 in patients with Focal Segmental
Glomerulosclerosis (FSGS), a rare fibrotic kidney disease with high
unmet medical need
• The acquisition also diversifies our pipeline, giving us access to
Fibrotech’s library of novel molecules that are engineered to target fibrotic
mechanisms across multiple indications of high unmet need
Fibrotech: strategic step in strengthening
Shire’s Rare Disease pipeline
Strategy
Shift
18. To be as brave as the people we help.
18
Pediatric
Alagille Syndrome (ALGS)
• Present at 3 months
• No approved therapy
• ~13% bile diversion surgery, 21-31% liver
transplant
• Very intense pruritus
Progressive Familial
Intrahepatic Cholestasis (PFIC)
• Present at 3-6 months
• No approved therapy
• ~35% bile diversion surgery, ~50%
liver transplant
• Very intense pruritus
• Without surgery, fatal by 2nd decade
Adult
Primary Biliary Cirrhosis (PBC)
• >40 years old, 90% female
• Ursodeoxycholic acid (UDCA) approved for
therapy – only ~50% patients respond
• Obeticholic acid (OCA; Intercept) in Ph 3 (but
increases pruritus & CV issues have been
reported)
• Slow progression
• Intense pruritus
Primary Sclerosing Cholangitis (PSC)
• Mean age at diagnosis: 40 years, 70% Male
• No approved therapy
• Intense pruritus
• Aggressive, life expectancy 8-10 years from
diagnosis
LUM001 for Cholestatic Liver Disease
Strategy
Shift
19. To be as brave as the people we help.
19
ALGS PFIC
Before
Surgery(1)
After
Surgery(1)
Before
Surgery(2)
After
Surgery(2)
Bile Acids (µmol/L) 115 28 Bile Acids (µmol/L) 337 11
Bilirubin (mg/dL) 2.4 1.6 Bilirubin (mg/dL) 2.4 1.5
Itching (0 no scratching-
4 cutaneous mutilation)
4 1 Itching (0-4) 3 1
Rationale for an ASBTi in Cholestasis
Lowering Bile Acids Results in Significant Clinical Benefit
Emerick, BMC Gastroenterology (2008), Emeriick, Hepatology (2002), Schukfeh, J Ped Surgery (2012)
(1) Mean (2) Median
Removing bile acids through surgical intervention:
Reduces serum bile acids
Improves biochemical markers of liver disease
Rapidly reduces itching
Slows disease progression
x Disfiguring and associated with serious complications
Strategy
Shift
20. To be as brave as the people we help.
20
Replenishing our pipeline and pursuing further
strategic deals
Acquired first and only protein
replacement therapy being investigated
for Dystrophic Epidermolysis Bullosa
(pre-clinical)
Acquired Dry Eye Disease asset (phase 3),
which could provide the basis for a larger
ophthalmology business
Acquired Retinopathy of Prematurity (ROP)
asset (phase 2), providing entry point
into neonatology
Announced deal adds significant scale to
rare disease portfolio with on- market
hereditary angioedema (HAE) product
complementary to FIRAZYR®
Expands our portfolio with clinical stage
anti-fibrotic agent (rare & renal diseases)
Expands GI and Rare Disease portfolio
with clinical assets for Cholestatic Liver
Diseases
Well positioned for future M&A
• Strong cash generation
• $1.8 billion in 2013
• Balance sheet flexibility
• Net Debt of $1.4 billion
• Increased corporate debt
ceiling to $12 billion
• Developing integration as a core
strength, for rapid realization of
synergies
Strategy
Shift
Note: Fibrotech & Lumena deals have been signed but are still subject to closure.
21. To be as brave as the people we help.
21
Further refine therapeutic area strategy
Strengthen our portfolio organically and via M&A
Integrate ViroPharma
Maintain discipline and continue One Shire
program to drive further operational leverage
Operational
Discipline
Strategy
Shift
2014 Priorities: continue to reposition Shire for
future growth
Commercial
Excellence
Explore new opportunities to drive growth from
in-line portfolio
Maintain commercial focus to deliver continued
sales growth
22. Our purpose
We enable people with life-altering conditions to lead better lives.
Q&A Breakout: Broadway Room, 10AM
23. Our purpose
We enable people with life-altering conditions to lead better lives.
Investor relations contacts
Europe:
Sarah Elton-Farr Souheil Salah
T: +44 1256 894157 T: +44 1256 894160
E: seltonfarr@shire.com E: ssalah@shire.com
North America:
Jeff Poulton
T: +1 781 482-0945
E: jpoulton@shire.com
