Aktiespararna Sundsvall
17 January 2019
Linda Holmström, Senior IR Manager
Swedish Orphan Biovitrum AB (Publ) (SobiTM)
Forward looking statements
1
In order to utilise the ‘Safe Harbor’ provisions of the United States Private Securities Litigation Reform Act of 1995, Swedish Orphan Biovitrum AB (publ) is providing the following cautionary statement. This presentation contains forward-looking statements with respect to the financial condition, results of operations and businesses of Swedish Orphan Biovitrum AB (publ), By their nature, forward-looking statements and forecasts involve risk and uncertainty because they relate to events and depend on circumstances that will occur in the future. There are a number of factors that could cause actual results and developments to differ materially from that expressed or implied by these forward-looking statements. These factors include, among other things, the loss or expiration of patents, marketing exclusivity or trade marks; exchange rate fluctuations; the risk that R&D will not yield new products that achieve commercial success; the impact of competition, price controls and price reductions; taxation risks; the risk of substantial product liability claims; the impact of any failure by third parties to supply materials or services; the risk of delay to new product launches; the difficulties of obtaining and maintaining governmental approvals for products; the risk of failure to observe ongoing regulatory oversight; the risk that new products do not perform as we expect; and the risk of environmental liabilities.
Swedish Orphan Biovitrum AB (Publ) (SobiTM)
A long history of innovation
2
Pharmacia
Kabi/Kabi Pharmacia
Biovitrum
Sobi
Swedish Orphan
Sobi in brief
• Business areas; Haemophilia, Immunology and Specialty Care
• Integrated biopharma company with a focus on rare diseases
• Including R&D and in-house production of biological drug substances
• Delivering treatments to patients in over 70 countries world wide
• SEK 6,511 M in revenues 2017, SEK 6,568 M Jan-Sep 2018
• About 850 employees
3
4
Life-threatening and chronic
EU : 1 on 2 000
US : Less than 200 000 people
More than 7 000 know diagnosis
400 approved treatments
Roughly 400 million people worldwide could be affected by a rare disease (1 of 15)
Worldwide Orphan Drug Sales forecast to grow +11% 2018-2024 to USD 262 Bn., double the rate forecast for the non-orphan drug market*
Why rare diseases?
*EvaluatePharma Orphan Drug Report 2018
Swedish Orphan Biovitrum AB (Publ) (SobiTM)
Product portfolio
Haemophilia Specialty CareImmunology
5
Sobi’s vision is to become global leaders in rare diseases
Increasing access to our treatments for haemophilia
Enlarging our Specialty Care franchise
Expanding our position in EMENAR,
and growing in North America
Strengthening R&D pipeline
6
Vision
To be recognised as a
global leader in
providing innovative
treatments that
transform lives for
individuals with rare
diseases
Swedish Orphan Biovitrum AB (Publ) (SobiTM)
Strong growth over the past years
7
Main growth drivers
• Extremely successful Haemophilia
launch
• Kineret strong double-digit growth
Growth opportunities
• Further growth opportunities in
Haemophilia and penetration and
internationalisation
• Increase US market presence and
footprint
• Broaden Specialty Care portfolio in
areas of expertise
Sobi revenue1
SEK M
2013
3,228
2014
2,177
20172015 2016
2,607
5,204
6,511
33% p.a.
2018E
8,900-9,0002
1. Sobi Annual Reports 2013–2017
2. Revised outlook published on 31 October 2018
Haemophilia
8
Swedish Orphan Biovitrum AB (Publ) (SobiTM)
Haemophilia – the most common rare disease in the world
• A a genetic disorder in which the ability of a person’s blood to clot is impaired.
• Haemophilia A occurs in about one in 5,000 male births annually, and more rarely in females.
• Haemophilia B occurs in about one in 28,000 male births annually, and more rarely in females.
• Estimated that 185,000 people are currently diagnosed with haemophilia world-wide
• People with haemophilia A or B experience bleeding episodes that can cause pain, irreversible joint damage and life-threatening haemorrhages.
9
1. World Federation of Hemophilia. Report on the Annual Global Survey 2016. Available at: http://www1.wfh.org/publications/files/pdf-1690.pdf. Accessed on: 6 March 2018
Swedish Orphan Biovitrum AB (Publ) (SobiTM)
A strong heritage within Haemophilia
10
ITIBIVV001BIVV002
Haemophilia – Global vs. Sobi territory market
• Sobi’s European Haemophilia A market estimated at 3.5 Bn. USD today
• Sobi’s European Haemophilia B market estimated at 0.5 Bn. USD
11
Source: EvaluatePharma
Top 5 global haemophilia A product sales
estimated at $ 4.6 bn(2018e)
$9.3 bnby 2024
+100%
Haemophilia A is hereditary and the most common of rare diseases
Swedish Orphan Biovitrum AB (Publ) (SobiTM)
A distinctive position in Haemophilia with Elocta and Alprolix
• Well established safety and efficacy profiles - real-world experience from thousands of patients
• Replacing the missing factor – fundamental in haemophilia treatment
• Standard of care in many countries
• Suitability for all patient groups and ages and flexibility to truly match treatment to outcomes
• Creates possibilities to live an active life with less worry about the safety and effectiveness of their therapy
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Swedish Orphan Biovitrum AB (Publ) (SobiTM)
A year of prophylactic treatment with conventional FVIII vs EHL
13
Convential FVIII is usually dosed 3-4 times a week1, 2, 3
* Bild från M. Carcao1. Meunier et al. International Society on Thrombosis and Haemostasis, 29 June–4 July 2013, Amsterdam, Netherlands; 2. Hay et al. International Society on Thrombosis and Haemostasis, 20–25 June 2015, Toronto, Canada;3. Ahnström et al. Haemophilia 2004
Median dosing frequency with rFVIIIFc in the A-LONG study was 2times a week1
Swedish Orphan Biovitrum AB (Publ) (SobiTM)
Haemophilia – continued strong sales growth
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• Total Haemophilia Jan-Sep revenues of SEK 4,260 M (2,568)
• Revenue growth of 66 per cent (60 per cent increase at CER)
• Elocta Jan-Sep product revenues of SEK 2,316 M1 (1,018)
▪ Revenue growth of 128 per cent (115 per cent at CER)
• Alprolix Jan-Sep product revenues of SEK 671 M (232)
▪ Revenue growth of 190 per cent (172 per cent at CER)
Total Revenues (SEK M)
0
200
400
600
800
1 000
1 200
1 400
1 600
1 800
Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3
2016 2017 2018
Product revenues Royalty
Manufacturing One-time items
1 Revenues as well as EBITA were positively affected by SEK 56 M, in Q3 2018, related to adjusted pharmaceutical taxes in France from 2017, whereof SEK 52 M relates to Elocta.
Haemophilia growth strategy
Capitalise on strong position and substantial potential
• Achieve a leadership position in Europe, the Middle East and Africa
• Expand access to patients in markets where treatment not yet available
• Research into follow-on compounds and advancing care
• Strengthen and broaden R&D pipeline
15
Swedish Orphan Biovitrum AB (Publ) (SobiTM)
Reaching patients in 40 countriesDonation statistics July 2015 to December 2017
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By year-end 2017, Sobi and Bioverativ had donated
262million IUs of Elocta and Alprolix enabling the treatment of
15,000+people in
40countries by addressing
79,400bleeds and almost
1,500surgical procedures
Specialty Care
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Lifecycle management & indication expansione.g. turnaround of Kineret to solid 10%+ p.a. growth
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Active strategic partnership management in order to continuously introduce novel therapies into our key markets: moving from distribution to ownership
Expand portfolio by strengthening R&D pipeline and business development efforts in order to further drive growth of the Specialty Care business
Specialty Care – active portfolio management
Swedish Orphan Biovitrum AB (Publ) (SobiTM)
Products
Specialty CareImmunology
19
Swedish Orphan Biovitrum AB (Publ) (SobiTM)
Specialty Care - solid performance across portfolio
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• Total Specialty Care Jan-Sep revenues SEK 2,308 M (2,068)
• Revenue growth of 12 per cent (9 per cent at CER)
• Kineret Jan-Sep revenues SEK 985 M (835)
▪ Revenue growth of 18 per cent (15 per cent at CER)
• Orfadin Jan-Sep revenues SEK 645 M (594)
▪ Revenue growth of 9 per cent (decrease of 4 per cent at CER)
Sales Revenues (SEK M)
0
1 000
2 000
3 000
4 000
0
250
500
750
1 000
Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3
2016 2017 2018
Quarterly 4Q-rolling
Strategic transactions to further drive growth
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Swedish Orphan Biovitrum AB (Publ) (SobiTM)
Expansive strategy to build Immunology franchise and strengthen US business
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Develop
Specialty
Care
Expand
US business
Strengthen
R&D
pipeline
Drive Haemophilia
Penetration
Swedish Orphan Biovitrum AB (Publ) (SobiTM)
Components of the Synagis transaction
• Perpetual rights to Synagis® (palivizumab) in
the US
• Synagis, the only approved respiratory
syncytial virus (RSV) prophylaxis for high-risk
infants
• Clinical studies have shown reduced risk of RSV-related hospitalisations in high-risk infants (pre-mature, and/or cardiopulmonary disease)
• Access to a proven US commercial
infrastructure
• Right to participate in 50 per cent of the future
earnings in the US
• A follow-on candidate to Synagis being
investigated for the prevention of lower
respiratory tract infection (LRTI) caused by
RSV in a larger patient population
• Currently in an ongoing Phase IIb study
• The current development plan includes initiation of a Phase III trial in healthy full-term and late pre-term infants
15
MEDI8897
RSV infection may be associated with significant morbidity
• Respiratory syncytial virus (RSV) is a major cause of respiratory illness in young children
• Almost all children have been infected by 4 years of age
• Symptoms may be like common cold and can develop into bronchiolitis and pneumonia
• Severe disease in children most often occurs in premature children and/or children with cardiopulmonary disease
• For the care of high-risk infants, prevention is critical
24Centers for Disease Control and Prevention. Respiratory Syncytial Virus Infection (RSV): Trends and Surveillance;Hall CB et al. The burden of respiratory syncytial virus infection in young children. New Engl J Med. 2009.Merck Manual 2018.
Swedish Orphan Biovitrum AB (Publ) (SobiTM)
The main theses of the transaction
Build a business on the combination of a unique biologic with access to the 50% of earnings of a follow on compound in a much broader patient population
Synagis is a product that connects with our DNA, meaning focus on rare conditions and expertise in the paediatric field
Double our commercial footprint in the US –allowing Sobi to expand its presence beyond Synagis
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Longitudinal earnings stream
Synagis connects with Sobi’s DNA
Access to talent poolto build US platform
Swedish Orphan Biovitrum AB (Publ) (SobiTM)
Pro forma financial impact
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Revenues
EBITA3 Margin
Key Financials
FY2018E (SEK)
8.95 billion1 2.4 billion2 11.4 billion
>40%
Combined
Pro forma
38-39%1
Enhances financial capacity
1. Revised outlook published on 31 October 2018. EBITA expected in the range of SEK 8.9-9.0 M.
2. Based on Synagis US LTM 30 June 2018 revenues reported by AstraZeneca, converted at a USD/SEK FX of 9.05
3. Financial measure not defined according to IFRS (alternative performance measure). EBITA is earnings before interest, tax and amortisation
>60%
Key Financials
LTM 30 June 2018 (SEK)
Swedish Orphan Biovitrum AB (Publ) (SobiTM)
Emapalumab - a highly attractive late stage orphan drug candidate
• Strategic partnership to develop and commercialise emapalumab
• A fully human monoclonal antibody that targets IFN-gamma, a central mediator of inflammation
• Addresses a high unmet medical need in Haemophagocytic lymphohistiocytosis (HLH)
• Emapalumab in review for rare disease primary HLH
• FDA approval 20 November 2018
• MAA filing to EMA validated Aug 2018
• Near-term commercial opportunity with sales potential from 2019 onwards
• Purchase price of CHF 450 M (CHF 50 M cash upon signing)
• The license agreement to emapalumab is expected to add of around SEK 200 M to the Sobi cost base in 2018 preparing for a successful launch
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Haemophagocytic lymphohistiocytosis (HLH)
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High unmet medical need
• Fatal with median survival of <2 months without
treatment
• No approved drug treatments
• Current widely accepted treatment protocols
apply a combination of dexamethasone and
chemo-immunotherapy (incl. etoposide)
HLH is a rare and life-threatening
syndrome of extreme immune activation
ca. 5,000+Patients in the
US, EU and Japan
(primary and
secondary HLH)
Two patient segments
Primary HLH (pHLH)
genetic disease
Secondary HLH (sHLH)
to infections, autoimmune
diseases or malignancyInitial filing
Source: [patient population data]
Swedish Orphan Biovitrum AB (Publ) (SobiTM)
Emapalumab – an excellent fit with our strategy
Late stage orphan drug in the Immunology area
Provides an attractive near-term commercial opportunity with USD 300 M peak sales potential
Expected to support sustainable sales growth from 2019 onwards
Full global rights to emapalumab
Utilises Sobi’s strengths in market access and product launch
Potential to significantly expand sales in the US market
PDUFA date 20 November 2018MAA filing to EMA validated Aug 2018
A late stage asset with studies in follow-on indications planned
29
Builds Immunology franchise and long-term
earnings streams
Expands
US business and
extend position in
EMENAR
Strengthens R&D pipeline
Swedish Orphan Biovitrum AB (Publ) (SobiTM)
Strategic rationale
30
Diversify Sobi’s
revenue base
into Immunology
Accelerate build
up of Sobi’s US
commercialisation
platform
Enhance
financial
capacity
R&D pipeline
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1 Extension trial for an already approved indication 2 PUP – Previously Untreated Patients
3 A Bioverativ, a Sanofi-company, development programme. Sobi has elected to add the programmes to the collaboration agreement with Bioverativ but has not yet opted-in. 4. Pending regulatory acceptance of acquisition.
A rare disease pipeline with increasing valueTherapeutic area / Indication
MPSIIIA
Anti-C5
Anti-IL-1
Haemophilia B
Pre-clinical
Haemophilia A
Phase 1
Acute gout
Phase 2
Haemophilia A
Haemophilia B
Still’s disease
Alkaptonuria
Phase 3
Haemophilia A
Haemophilia A
Haemophilia A
Haemophilia A and B
Product / Project
SOBI003
SOBI005
SOBI006
BIVV0023/EXTEN-B
BIVV0013/EXTEN-A
Anakinra/anaGO
Elocta/PUP1,2
Alprolix/B-YOND1
Alprolix/PUP12
Anakinra/anaSTILLs
Nitisinone/SONIA2
Elocta/ASURE
Elocta/reITIrate
Elocta/verITI8
Elocta/Alprolix/PREVENT
Phase 4
Haemophilia B
Primary HLH Emapalumab
Secondary HLH Emapalumab
HA
EMO
PH
ILIA
SPEC
IALT
Y C
AR
E
RSV Prevention MEDI88974
SOBI003 for Mucopolysaccharidosis IIIA (MPS IIIA)
• A rare systemic disease with a significant CNS component due to incomplete breakdown and increased lysosomal storage of heparan sulfate(HS)
• Increased morbidity and mortality
• Caused by mutations in gene for sulfamidaseenzyme
• Up to about 2000 people are estimated to live with MPS IIIA in the EU and US
• There is currently no treatment available for MPS IIIA
• SOBI003: a recombinant sulfamidase using proprietary Modifa™ technology with potential to address an unmet needs in MPS IIIA
33
SOBI003 - chemically modified recombinant human sulfamidase
Summary
34
Strong growth and positive outlook in the rare disease market segment
35
HAEMOPHILIA
Strong product offering
Continued market expansion
High entry barriers limit
competition
SPECIALTY CARE
Move towards proprietary
drugs and drug candidates
Acquisitions to
- Leverage European platform
- Create synergies with existing
portfolio
- Expand North American
footprint
LONG TERM VALUE
CREATION
Profitable growth
Strong cash flow
Increasing financial
flexibility
WORLD WIDE COMMERCIAL PLATFORM
• Proven track record of bringing Haemophilia products to the market
• Robust marketing and distribution infrastructure
Sobi’s vision is to become global leaders in rare diseases
Further internationalisationand commercialisation of Haemophilia
Build Specialty Care as the preferred partner
Strengthen positionin the US and EMENAR
Build pipeline and self-sustained R&D
36
Vision
To be recognised as a
global leader in
providing innovative
treatments that
transform lives for
individuals with rare
diseases
Swedish Orphan Biovitrum AB (Publ) (SobiTM)
Executive Committee
37
Ramin LevinHead of North America
Guido OelkersCEO
Torbjörn HallbergGeneral Counsel, Head of Legal Affairs
Fredrik WetterlundHead of HR
Henrik StenqvistCFO
Philip WoodHead of Haemophilia
Norbert OppitzHead of Specialty Care
Milan ZdravkovicHead of R&D, CMO
Armin ReiningerHead of Medical and Scientific Affairs
Christian DregerHead of NorthernEurope, Middle East& Russia
Anne Marie de Jonge SchuermansHead of Technical Operations
Paula TreutigerHead ofCommunications and IR
Sofiane FahmyHead of Southern and Western Europe & North Africa