Company Presentation
DELIVERING ON OUR STRATEGY Copyright © 2014 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 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation DELIVERING ON OUR STRATEGY OUR THREE KEY PRIORITIES SUSTAIN AND GROW THE PAH FRANCHISE BUILD ADDITIONAL SPECIALTY FRANCHISE OPTIMIZE PROFITABILITY 3 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation KEY FINANCIAL AND OPERATIONAL HIGHLIGHTS 9M 2014 –STRONG MOMENTUM MAINTAINED Strong financial performance – Sales of CHF 1,488 million, up 17% at CER(1) – 12% excluding US rebate reversals – Core earnings CHF 630 million, increase of 34% at CER – 21% excluding US rebate reversals Opsumit® launch momentum remains strong – Strong Opsumit launch momentum continues – Approvals and well-differentiated label around the world Selexipag data - as planned – 1 4 Regulatory filing process on track Constant Exchange Rates © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation DELIVERING ON OUR STRATEGY SUSTAIN AND GROW THE PAH FRANCHISE BUILD ADDITIONAL SPECIALTY FRANCHISE OPTIMIZE PROFITABILITY 5 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation THE FIRST DECADE OF SHAPING PAH TREATMENT TRACLEER: FIRST ORAL PRODUCT IN PAH 6 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation GLOBAL LEADERSHIP IN PAH IN SECOND DECADE EXPANDING THE TREATMENT OPTIONS * Selexipag is an investigational drug and not approved for use. 7 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation DEFINING THE FUTURE OF PAH TREATMENT COVERING CONTINUUM OF CARE AND GROWING PAH FRANCHISE FCII FCIII (in Monotherapy and/or combination with PDE5) * Selexipag is an investigational drug and not approved for use. 8 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation FC IV OPSUMIT® 9 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation ENGINE OF TRANSFORMATION: OPSUMIT® HIGHLIGHTS Standard setting SERAPHIN trial with ground-breaking outcome data Highly differentiated label in US and EU. Re. Liver enzyme monitoring: Label language at par with Ambrisentan Launched in 16 countries by Sept 2014 including US, several EU countries, Switzerland, and Canada Blockbuster potential Included in the Nice guidelines published in Journal of the American College of Cardiology* – Dec 2013 * Galiè N, Corris PA, Frost A, et al. Updated Treatment Algorithm of Pulmonary Arterial Hypertension. J Am Coll Cardiol 2013;62:D60–72 10 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation STRATEGIC PRIORITIES Upgrade ERA & 1st Line Co New treatment option for patients on an ERA today priority Early combination with PDE5-i Patients on PDE5 monotherapy today - to be treated in combination with Opsumit LAUNCH 1st priority 1st choice ERA 11 © 2014 Actelion Pharmaceuticals Ltd December 2014 ERA naive patients who would be treated with Tracleer or Letairis today Company presentation OPSUMIT® LAUNCH MOMENTUM CONTINUES CHF million Strong launch momentum maintained – YTD 112 Mio SFR sales Global roll-out update: – Launched in: US, Germany, Austria, Switzerland, Canada, UK, Ireland, Denmark, Norway, Sweden and NL – QIII launches: Italy, Belgium, Luxembourg and Australia – QIV launches: Mexico*(private market) and Finland 59 38 15 5 Q4 2013 Q1 2014 Q2 2014 Q3 2014 Registration process proceeding well in Japan; filed in other markets * Trade name Zependo 12 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation MACITENTAN DEVELOPMENT UPDATE Macitentan 13 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation OBJECTIVES OF MACITENTAN CLINICAL EXPANSION Better characterize Macitentan benefits in patients with PAH (e.g. Eisenmenger Syndrome) Extend use beyond PAH in other forms of Pulmonary Hypertension Develop for diseases beyond PH (e.g. Glioblastoma) Development program for pediatric use in PAH 14 © 2014 Actelion Pharmaceuticals Ltd December 2014 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 15 © 2014 Actelion Pharmaceuticals Ltd December 2014 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 16 © 2014 Actelion Pharmaceuticals Ltd December 2014 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 17 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation TRACLEER® 18 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation TRACLEER® SOLID VOLUME GROWTH IN NON-OPSUMIT MARKETS CHF million Positive impact of US rebate reversals and 2013/2014 US price increases +5% CER Growth Global units sales flat – despite erosion in Opsumit markets and increasing Gx competition (Poland, Czech, Mexico) 1,138 1,153 Stayveer launched by Marklas in Poland and Czech Republic Solid growth ex-US – driven by 19 9M 2013 9M 2014 © 2014 Actelion Pharmaceuticals Ltd December 2014 Digital Ulcer and PAH emerging markets (China, Russia, Asian and Latin America business) Company presentation VELETRI® 20 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation VELETRI® BECOMING A TRULY GLOBAL ASSET CHF million +93% CER Growth Accelerated growth trajectory Ongoing global launches - most recently available in Australia and NZ Continued strong uptake in Japan* - adversely affected going forward by price reduction March 01 2014 46 25 9M 2013 9M 2014 * Trade name Epoprostenol “ACT” 21 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation VENTAVIS® 22 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation VENTAVIS® SUSTAINING OUR BUSINESS CHF million 8% CER Variance Increasing competitive pressure 16% decline in units shipped Top line maintained through price 83 increase and positive effects of US rebate accrual reversals 86 Ready to defend our position in the market 23 9M 2013 9M 2014 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation OPEN THE PROSTACYCLIN PATHWAY TO MORE PATIENTS Selexipag Selexipag is an investigational drug and not approved for use 24 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation SELEXIPAG*: STRENGTHENING OUR PAH FRANCHISE FIRST SELECTIVE ORAL IP PROSTACYCLIN RECEPTOR AGONIST Originally discovered and synthesized by Nippon Shinyaku Selexipag selectively targets the prostacyclin receptor (also called IP-receptor) The IP receptor is one of 5 types of prostanoid receptor Selexipag, unlike prostacyclin analogs, is selective for the IP receptor over other prostanoid receptors Globally around 10,000-12,000 patients are receiving prostacyclin or prostacyclin analog therapy * Selexipag is an investigational drug not approved for use. 25 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation GRIPHON IN PAH A PIVOTAL PHASE III OUTCOME STUDY A multicenter, double-blind, placebo-controlled Phase III study assessing the safety and efficacy of selexipag on morbidity and mortality in patients with pulmonary arterial hypertension * Selexipag is an investigational drug and not approved for use. 26 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation GRIPHON - STUDY OBJECTIVES ► PRIMARY To demonstrate the effect of selexipag on time to first morbidity and mortality event in patients with pulmonary arterial hypertension (PAH) ► To evaluate the effect of selexipag on exercise capacity and other secondary and exploratory efficacy endpoints in patients with PAH ► To evaluate the safety and tolerability of selexipag in patients with PAH SECONDARY * Selexipag is an investigational drug and not approved for use. 27 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation GRIPHON – STUDY OVERVIEW POPULATION ► 1156 adult WHO Group I PAH patients STUDY PERIOD ► December 2009 – April 2014 ► Patients randomized 1:1 ► Patients were treated for up to 4.3 years ► February 2014, the study reached the total number of DETAILS primary endpoint events required to close the study * Selexipag is an investigational drug and not approved for use. 28 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation DOSING IN GRIPHON Uptitration of selexipag allows each patient's maintenance dose to be individualized based on tolerability. Selexipag or placebo twice daily Individual Maintenance Dose 1600 mcg 1400 mcg 1200 mcg 1000 mcg 800 mcg 600 mcg 400 mcg 200 mcg Visit 1 (randomization) * Selexipag is an investigational drug and not approved for use. 29 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation PERMITTED BACKGROUND THERAPY ORAL MEDICATIONS SPECIFIC FOR PAH When on a stable dose for at least 3 months prior to enrollment patients could receive: – endothelin receptor antagonist (ERA) – phosphodiesterase-5 (PDE-5) inhibitor – or a combination of the two * Selexipag is an investigational drug and not approved for use. 30 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation GRIPHON: PRIMARY ENDPOINT TIME TO FIRST MORBIDITY/MORTALITY EVENT Death (all-cause mortality) Patients in FC II or III at baseline: OR ↓ in 6MWD from Baseline (≥15%, confirmed by 2 tests on different days within 2 weeks) Hospitalization for worsening of PAH OR Time to 1st morbidity or mortality event Worsening of FC Worsening of PAH resulting in need for lung transplantation or balloon atrial septostomy OR Initiation of IV or SC prostanoids or chronic O2 therapy for worsening PAH OR * Selexipag is an investigational drug and not approved for use. 31 © 2014 Actelion Pharmaceuticals Ltd Disease Progression December 2014 AND Company presentation Patients in FC III or IV at baseline: ↓ in 6MWD from Baseline (≥15%, confirmed by 2 tests on different days within 2 weeks) AND Need forAND additional PAH treatment(s) All events adjudicated by a blinded critical events committee (CEC) PRIMARY EFFICACY ENDPOINT MET PATIENTS WERE TREATED FOR UP TO 4.3 YEARS. Selexipag – decreased the risk of a morbidity/mortality event versus placebo by 39% – p<0.0001 Efficacy observed was consistent across key subgroups: – age, – gender, – WHO Functional Class, – PAH etiology and – background PAH therapy * Selexipag is an investigational drug and not approved for use. 32 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation SAFETY AND TOLERABILITY The overall tolerability profile of selexipag in GRIPHON was consistent with prostacyclin therapies. 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; – headache, diarrhea, nausea, jaw pain, vomiting, pain in extremity, myalgia, nasopharyngitis and flushing. The proportion of patients discontinuing treatment due to adverse events was 14% on selexipag and 7% on placebo. * Selexipag is an investigational drug and not approved for use. 33 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation GRIPHON RESULTS Positive study – primary endpoint met 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 upcoming congress * Selexipag is an investigational drug and not approved for use. 34 © 2014 Actelion Pharmaceuticals Ltd DELIVERING ON OUR STRATEGY SUSTAIN AND GROW THE PAH FRANCHISE BUILD ADDITIONAL SPECIALTY FRANCHISE OPTIMIZE PROFITABILITY 35 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation VALCHLOR® 36 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation VALCHLOR® EXPANDING OUR SPECIALTY BUSINESS 9M 2014 sales: CHF 6.6 million USD 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 skin-directed therapy 3.8 Approx. 18,000 patients in US of which 30-40% considered candidates for Valchlor treatment 2.3 1.2 Q1 2014 Sales force expansion complete Q2 2014 Q3 2014 The registration process for other countries is under evaluation 37 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation ZAVESCA® 38 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation ZAVESCA® NIEMANN-PICK TYPE C INDICATION DRIVING GROWTH EX-US CHF million +15% CER Growth Underlying volume growth +18% Driven by continued growth in the Niemann-Pick Type C (NP-C) indication outside US and new markets (i.e. Japan*) 73 81 US growth driven by positive price effect Gx miglustat approved in Spain and 9M 2013 Denmark for the GD1 indication only – more EU markets expected to follow 9M 2014 * Trade name Brazaves 39 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation BUILD ADDITIONAL SPECIALTY FRANCHISE CADAZOLID: Clostridium difficile-associated diarrhea Cadazolid is investigational, in development and not approved or marketed in any country. 40 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation CADAZOLID: OUR NOVEL ANTIBIOTIC For treatment of Clostridium difficile-associated diarrhea (CDAD) Clostridium difficile is a spore-forming bacteria that is best known for causing antibiotic-associated diarrhea Cadazolid: 41 – 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 – Negligible absorption, safe and well tolerated – US FDA designated cadazolid as both a Qualified Infectious Disease Product (QIDP) and a Fast Track development program © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation CADAZOLID: PROGRESSING AS PLANNED Large 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 42 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation BUILD ADDITIONAL SPECIALTY FRANCHISE S1P RECEPTOR MODULATION Ponesimod is investigational, in development and not approved or marketed in any country. 43 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation S1P RECEPTOR MODULATORS: PONESIMOD Continued belief in the therapeutic value of ponesimod 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 44 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation S1P1 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 45 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation LOOKING TO THE FUTURE: OUR PIPELINE PROMISING PROJECTS TO DELIVER FUTURE SUCCESS Phase I Phase II Selexipag PAH Cadazolid Clostridium difficile assoc. diarrhea Macitentan Eisenmenger syndrome Ponesimod Multiple Sclerosis Macitentan CTEPH Macitentan CpcPH Lucerastat Lipid storage disorders Macitentan Glioblastoma S1P1 follow-up Immunological Disorders 46 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation Phase III Regulatory 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 47 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation DELIVERING ON OUR STRATEGY OUR THREE KEY PRIORITIES SUSTAIN AND GROW THE PAH FRANCHISE BUILD ADDITIONAL SPECIALTY FRANCHISE OPTIMIZE PROFITABILITY 48 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation FIRST NINE MONTHS 2014 – FINANCIAL OVERVIEW Variance Product sales CHF million 9M 2013 9M 2014 CHF CER 1,322 1,488 13% 17% 496 630 27% 34% 397 519 31% 39% 3.54 4.77 35% 42% Core earnings CHF million Operating income CHF million Core EPS CHF 49 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation NINE MONTHS 2014 – CORE EARNINGS CORE EARNINGS UP 21% AT CER - EX REBATE REVERSALS CHF million 34 105 63 664 630 630 FX 9M'14 CE as reported 496 9M '13 CE 50 Net impact of rebate reversals © 2014 Actelion Pharmaceuticals Ltd 9M '14 intrinsic CE 9M '14 CE at CER growth December 2014 Company presentation NINE MONTHS 2014 – EARNINGS PER SHARE Variance 9M 2013 9M 2014 CHF CER 304 566 86% 97% Basic EPS 2.73 5.09 Number of shares in calculation (m) 111.6 111.2 87% 98% Fully diluted EPS 2.65 4.87 Number of shares in calculation (m) 114.9 116.2 84% 95% Net income CHF million 51 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation FINANCIAL GUIDANCE AT CONSTANT EXCHANGE RATES 2014 Core earnings Growth low twenties percentage range 2014 Core earnings ex US rebate reversals Growth mid-teen percentage range 2015 Core earnings Due to higher 2014 base, 2015 guidance will be reviewed early next year 52 © 2014 Actelion Pharmaceuticals Ltd December 2014 Company presentation DELIVERING ON OUR STRATEGY Copyright © 2014 Actelion Pharmaceuticals Ltd
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