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ACTELION LTD
DELIVERING ON OUR STRATEGY
Company Presentation
February 2015
Copyright © 2015 Actelion Pharmaceuticals Ltd
The following information contains certain “forward-looking statements”, relating to the
company’s business, which can be identified by the use of forward-looking terminology such
as “estimates”, “believes”, “expects”, “may”, “are expected to”, “will”, “will continue”, “should”,
“would be”, “seeks”, “pending” or “anticipates” or similar expressions, or by discussions of
strategy, plans or intentions. Such statements include descriptions of the company’s
investment and research and development programs and anticipated expenditures in
connection therewith, descriptions of new products expected to be introduced by the company
and anticipated customer demand for such products and products in the company’s existing
portfolio. Such statements reflect the current views of the company with respect to future
events and are subject to certain risks, uncertainties and assumptions. Many factors could
cause the actual results, performance or achievements of the company to be materially
different from any future results, performances or achievements that may be expressed or
implied by such forward-looking statements. Should one or more of these risks or uncertainties
materialize, or should underlying assumptions prove incorrect, actual results may vary
materially from those described herein as anticipated, believed, estimated or expected.
2
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
TABLE OF CONTENTS
 Actelion at a Glance
 Actelion Today
 Strategy for Value Creation

Sustain & Grow the PAH Franchise

Build Additional Specialty Franchises

Optimize Profitability
 Management & Board
3
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
ACTELION
AT A GLANCE
4
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
ACTELION PHARMACEUTICALS LTD
ACTELION IS A FULLY-INTEGRATED BIOPHARMACEUTICAL
COMPANY WITH INNOVATION AT ITS CORE
Leader in the
science and medicine of
pulmonary arterial hypertension
(PAH)
Actelion Center, Allschwil
FOUNDED IN 1997 IN ALLSCHWIL, SWITZERLAND
Total employees (Dec 14)
• Drug Discovery
• Clinical Development
• Marketing & Sales
• Support Functions
2,470
371
611
1,066
422
Global reach with more than 30 affiliates worldwide
6 Products on the Market:
Opsumit®, Tracleer®, Veletri®, Ventavis®, Zavesca®, Valchlor®
2014 Sales: CHF 1.95 Billion
Core earnings: CHF 743 million
Over 50‘000 Patients currently treated with an Actelion
medication
Extensive Research & Development portfolio
5
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
STAGES OF COMPANY DEVELOPMENT
2020
2014
2007
1997
2001
Build pipeline
and commercial
Company formed,
infrastructure
development
& approval of
Tracleer
6
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Commercial
leverage
and prepare for
Tracleer LOE
Company presentation
Opsumit, selexipag
and development of
new franchises
PAH, Life Cycle
Management
and New
Franchises
ACTELION TODAY
7
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
ACTELION TODAY
A UNIQUE COMPANY
1 Based on innovation
2 Fully integrated and global
3 Highly profitable
4 Comprehensive infrastructure
5 Unencumbered assets
8
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
ACTELION TODAY
A UNIQUE COMPANY
1 Based on innovation
►
2 Fully integrated and global
►
3 Highly profitable
4 Comprehensive infrastructure
►
Searching only for innovative products
In-house research infrastructure from
discovery to clinical development
With a broad pipeline of interesting
projects on novel targets
5 Unencumbered assets
9
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
ACTELION TODAY
A UNIQUE COMPANY
1 Based on innovation
FULLY INTEGRATED AND GLOBAL
From Research to Commercialization
More than 30 operative affiliates worldwide
Product availability in >55 markets
2 Fully integrated and global
3 Highly profitable
4 Comprehensive infrastructure
5 Unencumbered assets
Commercial Operations
R & D Centers
10
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
ACTELION TODAY
A UNIQUE COMPANY
CORE EARNINGS
800
1 Based on innovation
700
2 Fully integrated and global
600
500
3 Highly profitable
400
4 Comprehensive infrastructure
200
300
5 Unencumbered assets
11
© 2015 Actelion Pharmaceuticals Ltd
February 2015
100
0
Company presentation
2008
2009
2010
2011
2012
2013
2014
ACTELION TODAY
A UNIQUE COMPANY
1 Based on innovation
►
Swiss company
2 Fully integrated and global
►
One discovery center in Switzerland
3 Highly profitable
►
Full global development capabilities
4 Comprehensive infrastructure
►
5 Unencumbered assets
12
© 2015 Actelion Pharmaceuticals Ltd
February 2015
►
Fully established infrastructure from
process to buildings
Focus on quality
Company presentation
ACTELION TODAY
A UNIQUE COMPANY
1 Based on innovation
2 Fully integrated and global
3 Highly profitable
►
Full rights to all products*
►
Strong balance sheet and financing capacity
►
No major alliances for own products
4 Comprehensive infrastructure
5 Unencumbered assets
*Cooperation with Nippon Shinyaku in
Japan for macitentan and selexipag
13
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
STRATEGY FOR VALUE
CREATION
SUSTAIN AND
GROW THE PAH
FRANCHISE
BUILD ADDITIONAL
SPECIALTY
FRANCHISES
OPTIMIZE PROFITABILITY
14
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
STRATEGIC PRINCIPLES
FOUR GOALS FOR ACTELION
Drive innovation forward
Pursue top quality science, internally
and externally, balanced with medical
need and commercial potential
Leverage our global presence
Expand innovative commercial
capabilities to new customers and
regions. Manage alliances putting the
product first
15
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Maximize the value of innovation
Develop projects ourselves and seek
partners or out-license when necessary to
maximize value
Insist on the highest quality in all we do
Quality is crucial and needs to be
ingrained across all functions
Company presentation
KEY HIGHLIGHTS OF 2014
ONCE AGAIN WE DELIVERED
 Strong operating performance leading to 25% core earnings growth at CER*
 Core earnings growth of 20% at CER - Excluding US rebate reversals
 Successful launch of Opsumit in 18 countries
 Opsumit filing in Japan in June
 Selexipag (Uptravi) positive morbidity/mortality Phase III
 Selexipag (Uptravi) filing in US and EU end of 2014
 Tracleer – PIP Compliance Statement and Commission Decision issued in
Europe
*CER = constant exchange rates
16
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
STRATEGY FOR VALUE
CREATION
SUSTAIN AND
GROW THE PAH
FRANCHISE
BUILD ADDITIONAL
SPECIALTY
FRANCHISES
OPTIMIZE PROFITABILITY
17
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
PULMONARY ARTERIAL HYPERTENSION
A LIFE-THREATENING AND OFTEN MISUNDERSTOOD CONDITION
 Disease of the blood vessels carrying blood from the heart to the lungs
- the pulmonary arteries
 When PAH develops, blood circulating through these vessels becomes
restricted, and the right side of the heart is put under increasing strain to pump
blood through the lungs
Normal artery
18
© 2015 Actelion Pharmaceuticals Ltd
Artery showing
vasoconstriction
February 2015
Company presentation
Diseased artery showing tissue
thickening and fibrosis
CLINICAL SEVERITY OF PAH
CLASSIFIED BY WORLD HEALTH ORGANIZATION (WHO)
This system grades PAH severity according to the functional status of the patient
FUNCTIONAL
CLASS
SYMPTOMATIC PROFILE
I
Patients with pulmonary hypertension but without resulting limitation of physical activity.
Ordinary physical activity does not cause dyspnoea or fatigue, chest pain, or near
syncope.
II
Patients with pulmonary hypertension resulting in slight limitation of physical activity.
They are comfortable at rest. Ordinary physical activity causes undue dyspnoea or
fatigue, chest pain, or near syncope.
III
Patients with pulmonary hypertension resulting in marked limitation of physical activity.
They are comfortable at rest. Less than ordinary activity causes undue dyspnoea or
fatigue, chest pain, or near syncope.
IV
Patients with pulmonary hypertension with inability to carry out any physical activity
without symptoms. These patients manifest signs of right heart failure. Dyspnoea and/or
fatigue may even be present at rest. Discomfort is increased by any physical activity.
19
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
TREATMENT PATHWAYS IN PAH
ENDOTHELIN RECEPTOR
ANTAGONISTS (ERA)
20
© 2015 Actelion Pharmaceuticals Ltd
February 2015
PHOSPHODIESTERASE-5INHIBITORS (PDE-5i)
Company presentation
PROSTACYCLIN
RECEPTOR AGONISTS
SIGNIFICANT PROGRESS IN THE FIELD OF PAH
PAH targeted therapies
1st
1st
PGI2
1990
INCREASING DISEASE
AWARENESS
PRECLINICAL/
CLINICAL RESEARCH
21
Oral PDE-5i
2000
IMPROVEMENT IN SYMPTOMS,
MEASURED BY 6MWD
ERA:
PDE-5i:
PGI2:
Oral ERA
1st
NATIONAL
NETWORKS
endothelin receptor antagonist
phosphodiesterase-5 inhibitor
prostacyclin
© 2015 Actelion Pharmaceuticals Ltd
February 2015
PATIENT
ASSOCIATIONS
DISEASE
REGISTRIES
therapies in 2010
2010
DISEASE WORSENING, MEASURED BY
TIME TO CLINICAL WORSENING
REFERENCE
CENTERS
Multiple approved
CONTROLLED
CLINICAL TRIALS
EVIDENCE-BASED
GUIDELINES
PROCEEDINGS FROM
3RD WORLD
CONGRESS 2003
ESC 2004
GUIDELINES
Company presentation
DISEASE PROGRESSION OVER YEARS,
MEASURED BY MORBIDITY/MORTALITY
SCREENING
HIGH-RISK GROUPS
PROCEEDINGS FROM
4TH WORLD
CONGRESS 2008
ESC/ERS 2009
GUIDELINES
ORAL THERAPIES IN PAH
RANDOMIZED CONTROLLED TRIALS
Drug
Study
Duration
Primary endpoint
No. of
patients
Study-3511,2
12 weeks
6-MWD
32
BREATHE-13
16 weeks
6-MWD
213
EARLY4
24 weeks
PVR, 6-MWD
185
Sildenafil
SUPER-15
12 weeks
6-MWD
277
Tadalafil
PHIRST6
16 weeks
6-MWD
405
ARIES-17,8
12 weeks
6-MWD
202
ARIES-27,9
12 weeks
6-MWD
192
AMBITION10
78.6 weeks
Clinical failure
610
Macitentan
SERAPHIN11
103.9 weeks
Morbidity/Mortality
742
Selexipag
GRIPHON12
76.4 weeks
Morbidity/Mortality
1,156
Bosentan
Ambrisentan
Short-term
fixed
treatment
period trial
design
1. Channick RN, et al. Lancet 2001. 2. Badesch D, et al. Curr Ther Res 2002.
3. Rubin LJ, et al. N Engl J Med 2002. 4. Galiè N, et al. Lancet 2008.
5. Galiè N, et al. N Engl J Med 2005. 6. Galiè N, et al. Circulation 2009.
7. Galiè N, et al. Circulation 2008. 8. Oudiz R, et al. Chest 2006.
9. Oudiz RJ, et al. J Am Coll Cardiol 2009. 10. Galiè N, et al. Eur Respir J 2014.
11. Pulido, et al. N Engl J Med 2013. 12. Actelion press release, June 2014.
22
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
EVOLUTION OF THE TREATMENT GUIDELINES
FROM RANDOMISED CONTROLLED TRIALS TO EVIDENCE-BASED
GUIDELINES
 A wealth of data concerning PAH management has emerged in recent years
–
Not only from RCTs, but also clinical practice, including disease registries
–
This has led to published management guidelines1, updated
recommendations2, and approval of multiple therapies
1. Galiè N, et al. Eur Heart J 2009. 2. Galiè N, et al. J Am Coll Cardiol 2013.
23
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
TRACLEER SUCCESS
CONTINUES
24
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
TRACLEER®: FIRST ORAL PRODUCT IN PAH
THE FIRST DECADE OF SHAPING PAH TREATMENT
Tracleer (bosentan) is an orally available
endothelin receptor antagonist (ERA) approved
for the treatment of PAH in over 60 countries, including
the United States in November 2001, the European Union
in May 2002 and Japan in April 2005
25
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
TRACLEER®
SOLID VOLUME GROWTH IN NON-OPSUMIT MARKETS

Solid growth ex-US – driven by
Digital Ulcer indication in Europe and PAH
emerging markets (China, Russia, Asian
and Latin America business)

Global units sales -2% due to erosion
in Opsumit markets and increasing Gx
competition

Positive impact of US rebate reversals
and 2013/2014 US price increases

Successful Gx defense in Canada,
Turkey, Mexico

Stayveer launched by Marklas in Poland
and Czech Republic
CHF million
-1% at CER
1,532
2013
26
© 2015 Actelion Pharmaceuticals Ltd
1,481
2014
February 2015
Company presentation
ACTELION’S PAH PORTFOLIO
27
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
ACTELION’S PAH FRANCHISE
* Selexipag (Uptravi) is an investigational drug submitted to health authorities but not approved for use.
28
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
TRANSFORMING OUR PAH PORTFOLIO
MOVING TO OUTCOME-BASED THERAPY
* Selexipag (Uptravi) is an investigational drug submitted to health authorities but not approved for use.
29
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
UNIQUELY POSITIONED TO SERVE PAH
COVERING CONTINUUM OF CARE WITH OUTCOME-BASED MEDICINES
FC II
FC III
* Selexipag (Uptravi) is an investigational drug submitted to health authorities but not approved for use.
30
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
FC IV
ENGINE OF
TRANSFORMATION
31
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
OPSUMIT®
ENGINE OF TRANSFORMATION
Opsumit (macitentan) is an orally available
endothelin receptor antagonist (ERA) approved
for the treatment of PAH in over 30 countries,
including the United States in October 2013 and the
European Union in December 2013
32
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
OPSUMIT: A LANDMARK IN PAH
 The effect of macitentan to reduce combined morbidity/mortality events
33
–
a multi-center, event driven long-term, placebo controlled study
–
average duration of exposure approximately 2 years,
–
in 742 patients
–
with symptomatic PAH
–
WHO functional class (FC) II-III
–
who were randomized to placebo (n=250), 3mg macitentan (n=250),
or 10mg macitentan (n=242) once daily
–
Patients were treated with Opsumit® monotherapy or in combination with
phosphodiesterase-5 inhibitors or inhaled prostanoids
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
IMPORTANT DATA CONFIRMING THE
CLINICAL UTILITY OF OPSUMIT
 Macitentan efficacy in incident & prevalent patients
 Change in 6MWD vs. long-term outcomes
 Change in WHO FC vs. long-term outcomes &
WHO FC subgroup analysis
 Macitentan and PAH related hospitalization
 All cause hospitalization
 Macitentan and quality of life in PAH
 Macitentan hemodynamic sub-study
 Macitentan in patients with and w/o anticoagulant
 Macitentan in patients with and w/o RV function
34
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
Oral
presentations
9
Poster
presentations
8
OPSUMIT® STRONG LAUNCH TRAJECTORY
ENGINE OF TRANSFORMATION

Strong launch momentum maintained
– 2014 sales of CHF 180 million

Over 6,300 patients benefiting from Opsumit

Global roll-out update:
CHF million
–
59
68
38
5
Q4 2013
35

15
Q1 2014
Launched in: US, Germany, Austria,
Switzerland, Canada, UK, Ireland,
Denmark, Norway, Sweden, NL, Italy,
Belgium, Luxembourg, Australia,
Mexico*(private market), Iceland and
Finland
Registration process proceeding well
in Japan; filed in other markets
* Trade name Zependo
Q2 2014
© 2015 Actelion Pharmaceuticals Ltd
Q3 2014
February 2015
Q4 2014
Company presentation
I.V. THERAPY MADE A
LITTLE EASIER
36
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
VELETRI®
I.V. THERAPY MADE A LITTLE EASIER
Veletri (Epoprostenol for Injection)
is intravenous prostacyclin.
Unlike other epoprostenol formulations
approved for PAH, Veletri is stable at room
temperature (77 F, 25 C) for up to 48 hours when
administered immediately upon reconstitution and dilution,
making the use of frozen gel packs unnecessary.
Approved in 15 countries including the United States since
2010 and some European markets since 2013
37
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
VELETRI®
BECOMING A TRULY GLOBAL ASSET
CHF million
+84% CER Growth

Accelerated growth trajectory

Global asset – available in 14 markets
around the globe

Continued strong uptake in Japan*
- adversely affected going forward by
price reduction March 01 2014
64
37
2013
38
© 2015 Actelion Pharmaceuticals Ltd
2014
February 2015
* Trade name Epoprostenol “ACT”
Company presentation
OPEN THE PROSTACYCLIN
PATHWAY TO MORE
PATIENTS
Selexipag (Uptravi) is an investigational drug
submitted to health authorities
but not approved for use
39
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
PROSTACYCLINS ARE UNDER UTILIZED TODAY
PROSTACYCLIN MARKET US VS EU5 *
Inhaled
(no
US
EU 5
Intravenous & Sub-Cutaneous
patients)
(no patients)
Total: ~ 4’500
Total: ~ 770
US
Total: ~ 4’000
EU 5
Total: ~ 1’000
Only about 20% of European patients and 50% of US patients initiated in
FC II - III receive a prostacyclin at some point in their PAH treatment**
* Source: Actelion Assessment based on Decision Resources Forecast, IMS sales data, CIA Worldfact book accessed in 2014
** Source: Actelion 2014 Quantitative Physician Survey
40
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
WHAT DRIVES PROSTACYCLIN UTILIZATION TODAY?
ACTELION 2014 QUANTITATIVE PHYSICIAN SURVEY
EFFICACY
TREATMENT
BURDEN
PATIENT
REFUSAL
PATIENT
ATTRIBUTES
41
© 2015 Actelion Pharmaceuticals Ltd
“
Prostacyclins are the most potent drug class.
“
The administration is a burden.
There is the risk of line infections.
“
Patients sometimes resist [PC] therapy because they view it as
a last resort that signals the end is near.
“
If a patient does not follow the hygiene rules you cannot give
them parenteral therapy. Infections are a severe threat.
”
Cardiologist, Italy
”
Consultant for PH and Rheumatology, UK
”
February 2015
Cardiologist, US
”
Pulmonologist, France
Company presentation
WHAT CAN CHANGE THIS DYNAMIC?
OPENING THE PROSTACYCLIN PATHWAY TO MORE PATIENTS
Are prostacyclin's more than a “last resort” option?
Is there an easy way to determine the best effective dose for each individual
patient?
Can an effective dose be reached with acceptable tolerability?
Is there long-term outcome data – including in double and triple combination
therapy?
42
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
SELEXIPAG (UPTRAVI) RESULTS
FIRST SELECTIVE ORAL IP PROSTACYCLIN RECEPTOR AGONIST
 Positive study – decreased the risk of a morbidity/mortality event versus placebo
by 39% (p<0.0001)
 Efficacy observed was consistent across key subgroups, ie. age, gender, WHO
Functional Class, PAH etiology and background PAH therapy
 The most common adverse events in GRIPHON that occurred with higher
frequency on selexipag than placebo were in-line with those known in
prostacyclin therapies
 First regulatory filings with US FDA & EMA initiated in December 2014
 Data to be presented at American College of Cardiology Congress (ACC.15)
–
12:00 hrs, 15 March 2015 – San Diego, California
* Selexipag (Uptravi) is an investigational drug submitted to health authorities but not approved for use.
43
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
SUSTAINING OUR
BUSINESS
MARKETED BY ACTELION IN THE US ONLY
44
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
VENTAVIS®
Ventavis (inhaled iloprost) is an
inhaled formulation of iloprost, a synthetic
compound that is structurally similar to prostacyclin
(PGI2), a naturally occurring molecule that causes blood
vessels to dilate, limits cellular hypertrophy, and inhibits
platelet aggregation.
MARKETED BY ACTELION IN THE US ONLY
45
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
VENTAVIS®
SUSTAINING OUR BUSINESS
CHF million
46
+3% CER Variance
110
112
2013
2014
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation

Increasing competitive pressure

17% decline in units shipped

Top line maintained through price increase
and positive effects of US rebate accrual
reversals

Ready to defend our position in the US
market
EXPANDING THE CLINICAL
UTILITY OF MACITENTAN
MACITENTAN
47
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
OBJECTIVES OF MACITENTAN CLINICAL PROGRAM
 Better characterize macitentan in specific PAH patient population
 Extend use beyond PAH in other forms of Pulmonary Hypertension
 Develop for diseases beyond PH
48
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
CLINICAL CLASSIFICATION OF
PULMONARY HYPERTENSION (PH) – 2009
1. PAH
1.1 Idiopathic PAH (iPAH)
1.2 Heritable PAH
1.3 Drugs and toxin induced
1.4 Associated with (APAH):
1.4.1 Connective tissue disease
1.4.2 HIV infection
1.4.3 Portal hypertension
1.4.4 CHD
1.4.5 Schistosomiasis
1.4.6 Chronic hemolytic anemia
1.5 Persistent pulmonary hypertension of
the newborn
1’. Pulmonary veno-occlusive disease
and/or pulmonary capillary
hemangiomatosis
Galiè et al. Eur Heart J 2009
© 2015 Actelion Pharmaceuticals Ltd
February 2015
2. PH due to
left heart disease
3. PH due to lung disease
and/or hypoxemia
4. Chronic
thromboembolic
pulmonary hypertension
5. PH with unclear and/or
multifactorial mechanisms
5.1 Hematological disorders
5.2 Systemic disorders
5.3 Metabolic disorders
5.4 Other
EXPANDING THE CLINICAL UTILITY OF OPSUMIT
MANAGING THE LIFE CYCLE
 OPUS (US observational, drug registry of Opsumit new users in clinical practice)
 SYMPHONY (psychometric validation of QoL questionnaire – USA)
 ORCHESTRA (psychometric validation of QoL questionnaire – FR, IT, ES)
 MAESTRO (Eisenmenger Syndrome)
 MERIT (CTEPH - Chronic Thromboembolic Pulmonary Hypertension)
 MELODY (CpcPH-LVD - Combined Pre- and Post-capillary Pulmonary
Hypertension due to Left Ventricular Dysfunction)
 Phase I program in new and recurring glioblastoma
50
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
EXTEND USE IN PULMONARY HYPERTENSION
CHRONIC THROMBOEMBOLIC PULMONARY HYPERTENSION
(MERIT STUDY)
 Chronic thromboembolic pulmonary hypertension (CTEPH) is a form of
pulmonary hypertension caused by old blood clots in the lungs (pulmonary
embolism)
 Phase II study “MERIT-1” initiated
 Goal is assessment of efficacy and safety of macitentan in CTEPH
51
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
EXTEND USE IN PULMONARY HYPERTENSION
COMBINED PRE- AND POST-CAPILLARY PULMONARY HYPERTENSION
DUE TO LEFT VENTRICULAR DYSFUNCTION (CpcPH)
 CpcPH is pulmonary hypertension secondary to left ventricular dysfunction
based on the difference between the diastolic pulmonary artery pressure (dPAP)
and the pulmonary artery wedge pressure (PAWP), or diastolic pulmonary
vascular pressure gradient (DPG).
 Phase II “MELODY” study enrolling patients
 Goal is assessment of efficacy and safety of macitentan in CpcPH
52
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
MACITENTAN - POTENTIAL INDICATIONS BEYOND PH
GLIOBLASTOMA: AREA OF HIGH UNMET MEDICAL NEED
 Glioblastoma (GBM) most common and aggressive malignant brain tumor, with
approximately 5 cases per 100’000
 Macitentan in GBM (with TMZ) Phase I tolerability study ongoing
(dose of 150 mg reached), no safety issues raised to date
 Further steps to be discussed with Health Authorities
53
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
STRATEGY FOR VALUE
CREATION
SUSTAIN AND
GROW THE PAH
FRANCHISE
BUILD ADDITIONAL
SPECIALTY
FRANCHISES
OPTIMIZE PROFITABILITY
54
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
BUILD ADDITIONAL SPECIALTY
FRANCHISE
MARKETED BY ACTELION IN THE US ONLY
55
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
VALCHLOR®
Valchlor (mechlorethamine) gel 0.016%
is applied topically once-a-day and dries
on the skin. Valchlor is the only US FDA
approved topical formulation of mechlorethamine,
a chemotherapeutic agent for the treatment of early stage
mycosis fungoides, a type of Cutaneous T-Cell Lymphoma.
Launched in the US in November 2013
56
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
MYCOSIS FUNGOIDES
EXPANDING OUR SPECIALTY BUSINESS
 Mycosis fungoides is the most common type of
Cutaneous T-Cell Lymphoma (CTCL), a rare form of
non-Hodgkin's lymphoma
 The cause of mycosis fungoides remains unknown and
there is no known cure
 Unlike most non-Hodgkin's lymphomas, mycosis
fungoides is caused by a mutation of T-cells. The
malignant T-cells in the body initially migrate to the skin,
causing various lesions to appear
 These lesions typically begin as what appears to be a
rash and may progress to form plaques and disfiguring
tumors
57
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
VALCHLOR®
A YEAR OF EXTENSIVE LEARNING
USD million
3.9
1.2
58
Q2 2014
Q3 2014
© 2015 Actelion Pharmaceuticals Ltd
February 2015
FY 2014 sales: CHF 11 million

For topical treatment of mycosis
fungoides–type cutaneous t-cell lymphoma
(a rare form of non-Hodgkin’s lymphoma)
in patients who have received prior skindirected therapy

Approx. 18,000 patients in US of which
30-40% considered candidates for
Valchlor treatment

Sales force expansion complete

The registration process for other
countries is under evaluation
4.2
2.3
Q1 2014

Q4 2014
Company presentation
BUILD ADDITIONAL SPECIALTY
FRANCHISE
59
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
ZAVESCA®
Miglustat, the active ingredient of
Zavesca, is an orally available molecule
with a large volume of distribution
Zavesca is approved for the treatment of
Niemann-Pick type C disease in 43 countries, including the
European Union since 2009 and Japan since 2012.
Zavesca is approved for the treatment of mild to moderate
type 1 Gaucher disease in 43 countries, including the US
and the European Union since 2003
60
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
NIEMANN-PICK TYPE C DISEASE (NP-C)
A RARE AND DIFFICULT TO DIAGNOSE GENETIC LYSOSOMAL
STORAGE DISORDER
 Devastating neurological genetic disorder which is ultimately fatal
 Onset from early childhood until adult age
 Pathophysiology
–
Abnormal intracellular lipid transport
–
Cytotoxic accumulation of glycosphingolipids in neurons
 Symptoms become progressively more severe and include:
61
–
Severe disabilities in swallowing, ambulation, eye movements, language,
cognition, muscle control
–
Lipid accumulation can also lead to an enlarged liver and/or spleen.
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
TYPE 1 GAUCHER DISEASE (GD1)
A RARE GLYCOSPHINGOLIPID DISORDER
 An inherited metabolic lysosomal storage disorder
 Characterized by an accumulation of lycosphingolipids
 The accumulation leads to multiple clinical manifestations:
–
an enlarged spleen and liver
–
anemia and a low platelet count
–
bone pain and bone deterioration
 Symptoms can appear at any age
62
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
ZAVESCA®
NIEMANN-PICK TYPE C INDICATION DRIVING GROWTH EX-US
CHF million
+11% CER Growth
96
2013
63
© 2015 Actelion Pharmaceuticals Ltd
103
2014
February 2015

Underlying volume growth +14%

Driven by continued growth in the
Niemann-Pick Type C (NP-C) indication
outside US and new markets (i.e.
Japan*)

US growth driven by positive price effect

Generic miglustat approved in several
European markets for GD1 indication
only
* Trade name Brazaves
Company presentation
BUILD ADDITIONAL SPECIALTY
FRANCHISE
CADAZOLID
CLOSTRIDIUM DIFFICILE-ASSOCIATED
DIARRHEA
Cadazolid is investigational, in development and not approved or marketed in any country.
64
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
International, Multi-center Program Assessing
Cadazolid Treatment in patients suffering from
Clostridium difficile-associated diarrhea (CDAD)
mpact
65
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
CADAZOLID: OUR NOVEL ANTIBIOTIC
 Investigated for the treatment of Clostridium difficile-associated diarrhea (CDAD)
 Clostridium difficile is a spore-forming bacteria that is best known for
causing antibiotic-associated diarrhea
 Cadazolid:
–
Strong inhibitor of Clostridium difficile protein synthesis leading to strong
suppression of both toxin and spore formation
–
Narrow spectrum – very limited effect on normal gut microflora – potential for
selective treatment for Clostridium difficile in the gut = less recurrence
–
In vitro tests demonstrate low propensity for resistance development
–
Early results indicate it may be safe and well tolerated with negligible
absorption
–
US FDA designated cadazolid as both a Qualified Infectious Disease
Product (QIDP) and a Fast Track development program
Cadazolid is investigational, in development and not approved or marketed in any country.
66
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
CADAZOLID: PROGRESSING AS PLANNED
PHASE III PROGRAM
 Two identical multi-center, randomized, double-blind studies designed to
demonstrate:
–
Non-inferior clinical response with cadazolid compared to vancomycin
–
Superior sustained clinical response with cadazolid compared to vancomycin
–
Efficacy on hypervirulent strains
 Enrollment of >1,250 patients worldwide commenced in Q4 2013
 Results expected in 2016
Cadazolid is investigational, in development and not approved or marketed in any country.
67
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
BUILD ADDITIONAL SPECIALTY
FRANCHISE
PONESIMOD
S1P RECEPTOR
IMMUNOMODULATION
Ponesimod is investigational, in development and not approved or marketed in any country.
68
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
PONESIMOD: KEY PROPERTIES
 Profile suitable for once-daily oral dosing
 Selective S1P1 receptor modulator
 Prevents lymphocytes from leaving lymph
nodes
 Lymphocyte reduction is rapid, dose-
dependent and reversible
 Rapidly reversible upon discontinuation
 Potential in multiple autoimmune diseases
69
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
PONESIMOD IN MULTIPLE SCLEROSIS
 Long-term treatment in the multiple sclerosis Phase II extension study under
evaluation
 Opportunity to proceed with an innovative treatment paradigm under
assessment
 Any further development will carefully balance:
– Clinical risk and investment
– Medical need and commercial opportunity
– Incorporating health authority input
70
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
S1P RECEPTOR MODULATORS: FOLLOW UP
IMMUNOLOGICAL DISORDERS
 Currently evaluated in Phase I
 Tolerability data in human volunteers suggest differentiation from products on
the market or compounds in development
– After Phase I program is completed, decision on future direction will be
made
71
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
EXTENSIVE RESEARCH
& DEVELOPMENT
72
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
A CHAIN OF EXPERTISE
356 PROFESSIONALS (SEPT. 2014)
Pharmacologists
Toxicologists
Cell Biologists
Molecular Biologists
DRUG
DISCOVERY
ORGANIZATION
Biochemists
Process Research Chemists
© 2015 Actelion Pharmaceuticals Ltd
Formulation Specialists
Clinical Scientists
Medicinal Chemists
73
Pharmacokineticists
February 2015
Company presentation
Structural Biologists
ACTELION’S DRUG DISCOVERY STRATEGY
All important research functionalities in-house
(e.g. MedChem, AssayTech, DMPK, Pharmacology)
Highly regulated service activities outsourced
(e.g. Toxicology, Production, Formulation)
74
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
THE BASE FOR HIGH DISCOVERY EFFICIENCY
CULTURE OF INNOVATION
•
•
•
•
•
•
75
Single-center approach
Fully integrated research informatics
Focus on small molecules
Few platforms of expertise
Multiple therapeutic areas
High medical input
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
CLINICAL DEVELOPMENT ORGANIZATION
594 PROFESSIONALS (SEPT. 2014)
Clinical Science
Life Cycle Management
Clinical Pharmacology
CLINICAL
DEVELOPMENT
Global Drug Safety
Global Drug Regulatory Affairs
76
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Biometry
Global Clinical Operations
Strategic Clinical Development
Company presentation
OUR CARDIOVASCULAR PIPELINE
PROMISING PROJECTS TO DELIVER FUTURE SUCCESS
Phase I
Phase II
Selexipag
PAH
Macitentan
Eisenmenger syndrome
Macitentan
CTEPH
Macitentan
CpcPH
Selexipag
o
Raynaud’s 2 to SSc
New Chemical Entity
Cardiovascular disorders
77
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
Phase III
Filing
OUR SPECIALTY PIPELINE
PROMISING PROJECTS TO DELIVER FUTURE SUCCESS
Phase I
Phase II
Cadazolid
Clostridium difficile assoc. diarrhea
Ponesimod
Multiple Sclerosis
Ponesimod
Immunological Disorders
Lucerastat
Lipid storage disorders
S1P1 follow-up
Immunological Disorders
Macitentan
Glioblastoma
78
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
Phase III
Filing
OUR RICH DISCOVERY PIPELINE
 >15 promising projects advancing in Drug Discovery
 Focus towards specialty markets and rare diseases with high unmet medical
need
 Current clinical pipeline to build solid portfolio for future revenue growth
79
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
STRATEGY FOR VALUE
CREATION
SUSTAIN AND
GROW THE PAH
FRANCHISE
BUILD ADDITIONAL
SPECIALTY
FRANCHISES
OPTIMIZE PROFITABILITY
80
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
FINANCIAL OVERVIEW
BY REPORTING PERIOD
81
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
FULL YEAR 2014 – FINANCIAL OVERVIEW
Variance
FY 2013
FY 2014
CHF
CER
Product sales
1,784
1,956
10%
12%
Core earnings
619
743
20%
25%
Operating income
482
570
18%
24%
Core EPS
4.41
5.58
27%
33%
CHF million
CHF million
CHF million
CHF
82
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
CORE EARNINGS
BY REPORTING PERIOD
83
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
FY 2014 – CORE EARNINGS
CHF million
800
743
66
619
32
600
677
588
400
2013
2014
Core earnings ex US rebate reversals
84
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
US rebate reversals
EARNINGS PER SHARE
(EPS) BY REPORTING
PERIOD
85
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
FY 2014 – EARNINGS PER SHARE
Variance
FY 2013
FY 2014
CHF
CER
Net income
453
594
31%
38%
Basic EPS
4.06
5.34
Number of shares in calculation (m)
111.5
111.2
32%
39%
Fully diluted EPS
3.92
5.11
115.4
116.2
30%
37%
CHF million
Number of shares in calculation (m)
86
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
SHAREHOLDER
RETURNS
87
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
DELIVERING ON OUR STRATEGY CREATES VALUE
SHARE PRICE PERFORMANCE
CASH RETURNED TO SHAREHOLDERS
588
358
133
2012
2013
2014
2015 – CASH DISTRIBUTION
New second-line share repurchase: up to 10 million shares within 3 years*
Board proposal: Increased dividend to CHF 1.30 per share
* Subject to regulatory approval
88
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
FINANCIAL GUIDANCE
JANUARY
2015
89
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
2015 FINANCIAL GUIDANCE
AT CONSTANT EXCHANGE RATES
From higher 2014 base (excluding US rebate reversals):
Low-single digit core earnings growth
743
2014 inc US
rebates
677
625
2014 ex US
rebates
2014 ex US
rebates at post
SNB FX*
* assumes USD 0.93, EUR 1.05, JPY 0.78, all other foreign currencies -5%
90
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
MANAGEMENT &
BOARD
91
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation
THE RIGHT PEOPLE FOR THE NEXT GROWTH PHASE
ACTELION MANAGEMENT TEAM
Jean-Paul Clozel
Founder, CEO
Joined in 1997
Martine Clozel
Founder, CSO
Joined in 1997
Otto Schwarz
COO
Joined in 2008
Nicholas Franco
Chief BD Officer
Joined in 2011
Marian Borovsky
General Counsel
Joined in 2003
92
© 2015 Actelion Pharmaceuticals Ltd
February 2015
Christian Albrich
Head of Global HR
Joined in 2005
Company presentation
André Muller
CFO
Joined in 2013
Guy Braunstein
Head of Global CD
Joined in 2009
Andrew Weiss
Head of IR & CC
Joined in 2014
THE RIGHT PEOPLE FOR THE NEXT GROWTH PHASE
ACTELION BOARD OF DIRECTORS
Jean-Pierre Garnier
Chairman
Joined in 2011
Juhani Anttila
Joined in 2005
Carl Feldbaum
Joined in 2005
Robert J. Bertolini
Joined in 2011
John J. Greisch
Joined in 2013
Werner Henrich
Joined in 2000
93
© 2015 Actelion Pharmaceuticals Ltd
Michael Jacobi
Joined in 2009
February 2015
Company presentation
Jean-Paul Clozel
Joined in 2000
Peter Gruss
Joined in 2012
Jean Malo
Joined in 2004
THANK YOU FOR YOUR
INTEREST IN ACTELION
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© 2015 Actelion Pharmaceuticals Ltd
February 2015
Company presentation

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