VERY STRONG START
TO 2016
Financial Reporting Investor Webcast
21 April 2016
Copyright © 2016 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.
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© 2016 Actelion Pharmaceuticals Ltd
Q1 2016 Financial Results
Q1
2016
KEY HIGHLIGHTS: A VERY STRONG
START TO 2016
 Opsumit: Continues its outstanding launch trajectory
 Uptravi: Off to an excellent start in the US
 Other products: Performing well
 Pipeline: Significant recruitment drive into Phase III programs
 Discovery: 3 new drugs delivered to the clinic
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© 2016 Actelion Pharmaceuticals Ltd
Q1 2016 Financial Results
CONSOLIDATE AND GROW OUR
PAH FRANCHISE
High clinical utility
Demonstrated long-term benefits
More to come with ongoing studies
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© 2016 Actelion Pharmaceuticals Ltd
Q1 2016 Financial Results
CONSOLIDATE AND GROW OUR
PAH FRANCHISE
US: FDA APPROVAL 21 DEC 2015
US: LAUNCH 04 JAN 2016
EU: CHMP POSITIVE OPINION 29 JAN 2016
Uptravi® (selexipag) is an orally available,
selective IP prostacyclin receptor agonist,
targeting and activating the prostacyclin
pathway.
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© 2016 Actelion Pharmaceuticals Ltd
Q1 2016 Financial Results
DEVELOP OTHER LANDMARK
THERAPIES
Cadazolid: Clostridium difficile associated diarrhea
Ponesimod: Multiple sclerosis
Clazosentan: Reversal of vasospasm post-aSAH
Cenerimod: Systemic lupus erythematosus
Other projects: Progressing well
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© 2016 Actelion Pharmaceuticals Ltd
Q1 2016 Financial Results
2016: A YEAR TO SEIZE
OPPORTUNITY
With the confidence of our shareholders:
We can create one of the most successful
biotech companies in the world!
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© 2016 Actelion Pharmaceuticals Ltd
Q1 2016 Financial Results
CONTINUE TO GROW PROFIT
High single-digit percentage core
operating income growth, at
constant exchange rates and barring
unforeseen events
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© 2016 Actelion Pharmaceuticals Ltd
Q1 2016 Financial Results
TURBO-CHARGING THE
PORTFOLIO TRANSFORMATION
9
© 2016 Actelion Pharmaceuticals Ltd
Q1 2016 Financial Results
Q1
2016
STRONG PERFORMANCE
515
Q1 2015
10
© 2016 Actelion Pharmaceuticals Ltd
+11% at CER
589
Q1 2016
Q1 2016 Financial Results
Q1 2015
Q1 2016
US
49%
56%
Europe
31%
28%
Japan
8%
8%
RoW
11%
8%
Q1
2016
ACCELERATING THE TRANSFORMATION
Q1 2015
CHF 515 m
Tracleer
11
Opsumit
© 2016 Actelion Pharmaceuticals Ltd
Q1 2016
CHF 589 m
Veletri
Uptravi
Others
Q1 2016 Financial Results
OPSUMIT + UPTRAVI
+ VELETRI
=
40% OF SALES
OPENING THE PROSTACYCLIN
PATHWAY TO MANY
MORE PATIENTS
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© 2016 Actelion Pharmaceuticals Ltd
POWERFUL PREPOSITIONING
Unaided vs. Aided Awareness
Unaided
Incremental Aided
76%
Total Awareness 86%
Source: In-house qualitative market research
with 50 participants in total
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© 2016 Actelion Pharmaceuticals Ltd
Familiarity with Uptravi
Familiar to very familiar
Heard of, not familiar
10%
46%
Little to somewhat familiar
Never heard of
32%
8% 14%
HIGH PROPENSITY TO PRESCRIBE
Oral prostacyclin analogue
(n=30)
Inhaled prostacyclin analogue (n=30)
67% Extremely / very suitable
80% Extremely / very suitable
20%
53%
Source: In-house qualitative market research
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© 2016 Actelion Pharmaceuticals Ltd
27%
7% 20% 7%
23%
43%
A POWERFUL START
Sales of
CHF 35m
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© 2016 Actelion Pharmaceuticals Ltd
ENGINE OF GROWTH
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© 2016 Actelion Pharmaceuticals Ltd
GLOBAL NET SALES EVOLUTION
CHF million
US sales
ex-US sales
42
53
58
33
27
14
5
Q4 2013
17
9
2
13
29
Q1 2014
Q2 2014
© 2016 Actelion Pharmaceuticals Ltd
22
44
46
Q3 2014
Q4 2014
68
Q1 2015
80
Q2 2015
105
109
Q3 2015
Q4 2015
120
Q1 2016
I.V. THERAPY PRESERVING
QUALITY OF LIFE DIMENSIONS
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© 2016 Actelion Pharmaceuticals Ltd
CONTINUED SIGNIFICANT GROWTH
+20% CER Growth

Growth momentum due to:
– Launch (June 2015) momentum in
France continuing in 2016
CHF million
19
24
– Japan* +16% despite a 12% price cut
on 1 March 2016
– US/EU achieved in most markets
> 80% new i.v. Epo patient share
– US «Ready Sette Go» program
expanded
Q1 2015
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© 2016 Actelion Pharmaceuticals Ltd
Q1 2016
*Trade
name Epoprostenol “ACT”
TRANSITIONING TO OPSUMIT
- 14% in Units
-18% at CER
CHF million
344
Q1 2015
20
© 2016 Actelion Pharmaceuticals Ltd
290
Q1 2016

Erosion driven by Opsumit and Gx
impact in selected markets

DU growth (i.e. Japan, Germany, UK)
mitigates erosion

Gx Update:

Q1 2016 Financial Results
–
Spain: very aggressive generics
–
Portugal: Actelion prevailed in legal
case vs Zentiva
No price cut in Japan
WELL MANAGED PERFORMANCE
-18% CER Variance
CHF million
 24% decline in units shipped due
to competitive pressure
 Volume decline expected to
31
accelerate due to competitive
situation
27
 No Gx entry expected in 2016
Q1 2015
21
© 2016 Actelion Pharmaceuticals Ltd
Q1 2016
Q1 2016 Financial Results
CONTINUED GROWTH IN NP-C
+26% CER Growth
CHF million
 Baseline effect in the US
 Increasing generic competition for
GD1 in selected EU markets
20
25
 Strong growth in NP-C indication
Ex-US – underlying volume up 15%
 Potential miglustat generic entry in
US during H2 2016
Q1 2015
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© 2016 Actelion Pharmaceuticals Ltd
Q1 2016
Q1 2016 Financial Results
CONSISTENT PROGRESS
 Q1 2016 sales: CHF 9 million
W
1,400
Monthly tubes shipped to patients (US)
1,200
 ATU FR proceeding well: > 230
PoD end of March 2016,
CHF 0.6 m sales
1,000
800
 Regulatory process ongoing – EU*
600
decision anticipated early in 2017
400
200
0
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*Trade name Ledaga®
© 2016 Actelion Pharmaceuticals Ltd
Q1 2016 Financial Results
PRIORITIES 2016:
FOCUS ON EXCELLENCE OF EXECUTION
Continue Opsumit trajectory
Build on strong early launch trajectory for Uptravi in the US
Prepare for Uptravi launches ex-US
Manage generic exposures
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© 2016 Actelion Pharmaceuticals Ltd
Q1 2016 Financial Results
STRONG FINANCIAL
PERFORMANCE
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© 2016 Actelion Pharmaceuticals Ltd
Q1 2016 Financial Results
Q1
2016
EXCELLENT START TO 2016
Variance
Q1 2015
Q1 2016
CHF
CER
Product sales
515
589
14%
11%
Core operating income
218
249
14%
8%
Core diluted EPS
1.61
1.98
23%
17%
Operating income
190
208
10%
3%
US GAAP diluted EPS
1.38
1.64
19%
11%
CHF million
CHF million
CHF
CHF million
CHF
26
© 2016 Actelion Pharmaceuticals Ltd
Q1 2016 Financial Results
Q1
2016
PRODUCT SALES
INTRINSIC GROWTH + 11%
17
58
CHF million
515
Q1'15 Sales
27
© 2016 Actelion Pharmaceuticals Ltd
Q1 '16 intrinsic
sales growth
Q1 2016 Financial Results
573
589
FX
Q1'16 Sales
Q1
2016
CORE OPERATING INCOME
INTRINSIC GROWTH + 8%
13
18
CHF million
218
Q1'15 Core operating income
28
© 2016 Actelion Pharmaceuticals Ltd
Q1 '16 intrinsic growth
Q1 2016 Financial Results
236
249
FX
Q1'16 Core operating income
Q1
2016
CORE OPERATING INCOME
CHF million
46
126
169
589
Q1 '15 Product sales
29
© 2016 Actelion Pharmaceuticals Ltd
Cost of sales
R&D
Q1 2016 Financial Results
248
249
SG&A
Q1 '16 Core operating
income
Q1
2016
US GAAP OPERATING INCOME
22
CHF million
16
249
Q1'16 Core operating
income
30
© 2016 Actelion Pharmaceuticals Ltd
D&A
Stock-based
compensation
Q1 2016 Financial Results
3
208
208
Other
Q1'16 Operating
Income
190
Q1'15 Operating
Income
Q1
2016
US GAAP NET INCOME
1
CHF million
29
0.4
208
Q1'16 Operating
Income
31
Financial result
© 2016 Actelion Pharmaceuticals Ltd
Income taxes
178
178
Non-controlling
interest
Q1'16 Net Income
Q1 2016 Financial Results
159
Q1'15 Net Income
Q1
2016
EARNINGS PER SHARE
Variance
Q1 2015
Q1 2016
CHF
CER
Core Net income
185
215
16%
10%
Core Diluted EPS
1.61
1.98
115.3
108.9
23%
17%
US GAAP Net income
159
178
12%
5%
US GAAP Diluted EPS
1.38
1.64
115.3
108.9
19%
11%
CHF million
Number of shares in calculation (m)
CHF million
Number of shares in calculation (m)
32
© 2016 Actelion Pharmaceuticals Ltd
Q1 2016 Financial Results
Q1
2016
CASH POSITION & CASH FLOW
172
CHF million
37
69
405
Net cash
31 Dec 15
33
Operating
cash flow
© 2016 Actelion Pharmaceuticals Ltd
2nd line share
repurchase
6
6
471
471
471
472
1st line share
purchase
ESOP
proceeds
Capex,
other items
Net cash
31 March 2016
Q1 2016 Financial Results
2016
FINANCIAL GUIDANCE
High single-digit percentage core
operating income growth, at
constant exchange rates and barring
unforeseen events
34
© 2016 Actelion Pharmaceuticals Ltd
Q1 2016 Financial Results
2016: A YEAR TO SEIZE
OPPORTUNITY
With the confidence of our shareholders:
We can create one of the most successful
biotech companies in the world!
35
© 2016 Actelion Pharmaceuticals Ltd
Q1 2016 Financial Results