1. business and industrial
2. company
3. earnings

Sobi: Update and Perspective
Geoffrey McDonough, CEO
JP Morgan Conference
San Francisco January 2013
Forward Looking Statements
In order to utilize the ‘Safe Harbor’ provisions of the United States Private Securities Litigation Reform Act
of 1995, Swedish Orphan Biovitrum 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. 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.
2
Who We Are
Sobi is an international specialty
healthcare company dedicated to
rare diseases.
Our mission is to develop and
deliver innovative therapies that
improve the lives of patients.
3
Quick Facts
• Listing: NASDAQ OMX (STO:SOBI)
- Outstanding shares: 265.2 M
- Sponsored ADR through BNY Mellon (SWTUY)
• Market Cap: $1 508 M
- Share price, close 2 January 2012: SEK 38.1
- 52-week range: SEK 11.40 – 45.50
• Net Revenues FY 2011: $285 M
• EBITA1 FY 2011: $19 M
• Net Debt, September 30 2012: $42 M
• Employees: ~ 480
• Ownership Summary:
30 September 2012
% of capital
Sweden
65
of which Investor AB
40
Rest of World
35
1
4
Before non-recurring items.
Figures restated in US dollars @ SEK 6.7/dollar
What We Do
Our key therapeutic areas are Inflammation and Genetic
diseases, with a growing focus on Hemophilia and
Neonatology.
We deliver products to specialist physicians and their patients
through our integrated and focused team approach to sales
and marketing, medical affairs and patient access.
We leverage our world-class capabilities in protein
biochemistry and biologics manufacturing to develop next
generation biological products.
5
Last Year at JP Morgan
6
A Diverse Commercial Portfolio + Strong Pipeline…
Development
Programs
Key Therapeutic
Areas
+12%2
at CER
US$ 4 Billion
Market Potential
in Pipeline
Neonatology
Hemophilia
Inflammation
Genetics
+10%2
at CER
+8%2
at CER
Base
Business
-4%2
Partner Products
ReFacto AF®
1) 2011 figures in USD, excluding co-promotion revenues
2) YTD growth 2012 vs. 2011, adjusted for discontinued products and ReFacto/BeneFIX co-promotion
7
US$ 120M
US$ 150M1
YTD Consolidated Revenues and Net Debt
Merger with
Swedish Orphan
300
250
USD M
USD M
200
Biovitrum
stand-alone
150
200
0,5
160
0,4
120
0,3
80
0,2
40
0,1
0
0,0
Revenues
8
EBITA before non-recurring items
Net debt
Net debt / Equity
Q3-12
2012
YTD
Sept
Q2-12
2011
Q1-12
2010
Q4-11
2009
Q3-11
2008
Q2-11
2007
Q1-11
0
Q4-10
50
Q3-10
100
Outlook 2012 On Track
Revenues
Total revenues expected to be about SEK 100 M lower than 2011, reflecting the
divestment of the co-promotion rights.
Gross Margin
Gross margin expected to be in line with 2011 margin of 54% after adjustment for
the balance sheet write-downs and the divestment of co-promotion rights.
Costs related to the transfer of Kineret production are estimated at SEK 60 M
impacting gross margin primarily in the first half of the year.
OPEX
Operating expenses estimated at or below SEK 950 M.
Milestone Payment
Milestone payment to Amgen of USD 55 M expected in Q4 2012 or in Q1 2013.
The outlook was first published in the Q4 report on 23 February 2012.
9
Calendar Highlights 2012
Event
H1
2012
H2
2012
Orfadin Liquid Formulation PIP Response
Kineret CAPS PIP Response
Complete Tech Transfer Kineret Manufacturing
Kineret NOMID Filing FDA
Top-Line Data for rFIXFc Program (BIIB)
Kineret CAPS Filing EMA
Top-Line Data for rFVIIIFc Program (BIIB)
Kiobrina Complete Phase 3 Enrollment
Kineret NOMID Filing FDA Approval
□
Management Team Additions
Geoffrey McDonough, Chief Executive Officer
Previous positions: Genzyme Corporation since 2002, latest as President of Europe, Middle East and Africa.
Alan Raffensperger, Chief Operating Officer
Previous positions: CEO of Benechill Inc.,USA, Executive Director, Head of Nephrology at Amgen 2008-2010,
General Manager of the Nordic and Baltic region at Amgen 2005-2008, Sales and Marketing Director at Roche
Pharmaceuticals, Sweden 1999-2004, Vice President, Global Marketing Diabetes Care, Roche Diagnostics 19961998, Business Director Europe, Diabetes Care at Boehringer Mannheim 1994-1996. He has also held leading
positions within Pharmacia in Sweden and USA.
Birgitte Volck , Chief Medical Officer
Previous positions: Various senior positions within Amgen since 2007, latest as Executive Development
Director, Bone, Neuroscience & Inflammation at Amgen Limited in Uxbridge, UK. Prior to Amgen she was
Nordic Medical Director & Project Director at Genzyme A/S in Denmark 2004-2007, and Vice President, Clinical
Development & Medical Affairs at Pharmexa A/S in Denmark 2001-2004.
Wills Hughes-Wilson, Vice President, Chief Patient Access Officer
Honors graduate in Law from University of Durham in the UK.
Previous positions: Genzyme Corporation, now part of the French Sanofi Group, where she was Vice President
Health/Market Access Policy, Europe. Prior to Genzyme she was Executive Director of Emerging
Biopharmaceutical Enterprises (EBE), a specialized group of the European Federation of Pharmaceuticals
Industries & Associations (EFPIA) representing the interest of biotechnology companies in Europe.
11
Twelve Months of Progress
12
Today’s Portfolio is a Platform…
Development
Programs
Key Therapeutic
Areas
Base
Business
13
Neonatology
Hemophilia
Inflammation
Partner Products
Genetics
ReFacto AF®
Near-Term Focus on Operating Businesses…
Pre-clinical Development Programs
Hemophilia
Key
Therapeutic
Areas
Neonatology
Inflammation
Partner Products
Base
Business
14
ReFacto
Continued focus
to maintain current
momentum.
Genetics
Segment as
near-term growth driver,
evolution and streamlining
of business model.
Steady growth and
leverage in key source of
cash flow.
3
2
1
1
Quarterly revenues (SEK M)
150
600
100
400
50
200
Quarterly
15
Q3-12
Q1-12
Q3-11
Q1-11
Q3-10
0
Q1-10
0
4 quarters rolling
4 quarters rolling revenues (SEK M)
Kineret® – Progress
• Sales of Kineret in the third quarter were
USD 17 M, an increase of 12%
• No impact from currency y/y
• Priority review by FDA for application for
Kineret for NOMID indication has
resulted in FDA approval in late
December 2012
• Increased efficiency in the production
following tech transfer will further
improve gross margins going forward
1
Evolving Position of Kineret in IL-1 Diseases
Identifying subsets of
RA population
Where Kineret’s short half life
and safety profile uniquely suitable
Entering the Pediatric
inflammation segment
Emerging evidence for role
of IL-1 in disesases with
unmet medical need
• Gout and pseudogout
High Rrisk Pts on Biologicals1
(200 000)
• Post-myocardial infarction
heart failure
Dynamic Patients 2,3
(100 000)
Prone to
Infections
Kineret Opportunity
= 10.000 FYP4
16
• Skin disease (hidradenitis
suppurativa, acne)
Diabetes
CV
1 Datamonitor 2010
2 IMS Health 2011
3 Opticom MR 2011
4 EU 27 and US (est)
• Stroke
•
Filed for CAPS in EU in Q4, 2012
• Type 1 & 2 diabetes
•
Critical to allow support
practice in children
• Dry eye syndrome
• Malignanacies
Treating inflammation by blocking interleukin-1
in a broad spectrum of diseases
Charles A. Dinarello, Anna Simon and Jos W. M.
van der Meer, Nature Reviews, Aug, 2012
2
Partner Portfolio – Growing European Platform
Offers small and midsized companies an
integrated solution for commercialization
of niche and specialty products:
• Market leader in Nordics, Baltics, CEE
Oncology
Emergency
Medicines &
Antidotes
• Expanding to all Europe model and Russia
• Portfolio of more than 40 specialty
pharmaceuticals, 30+ partners
• High rates of capital return in this segment of the
business with attractive growth risk profile
 Focused on renewed deal flow in line with this
model
17
Other
Specialty
Areas
Hematology &
Hemophilia
3
250
1 000
200
800
150
600
100
400
50
200
ReFacto Manufacturing
4 quarters rolling
18
Q3-12
Q1-12
Q3-11
Q1-11
Q3-10
0
Q1-10
0
ReFacto Royalty
4 quarters rolling revenues (SEK M)
Quarterly revenues (SEK M)
Revenues from ReFacto
• Solid volume growth in ReFacto
manufacturing revenues
• Adjusted for validation batches in 2011 (SEK
42 M), revenues increased 3% YTD
• Higher utilization of production
facility compared to last year
• Scale-up of downstream production
process will have a positive effect on
gross margin going forward
• ReFacto royalty revenues impacted by
difference in timing between quarters
… and Mid-Term Launch of Our Pipeline
Development Programs
Hemophilia
Key
Therapeutic
Areas
Base
Business
Neonatology
Inflammation
Partner Products
ReFacto
19
Genetics
Hemophilia
Hemophilia A + B: A Combined USD $6B+ Market
Global sales, 2010 ($ M)
FIX
BeneFIX
(Pfizer)
Plasma Derived Factor
(Various)
1000
Source: Benefix - 2010 Pfizer Annual Report. Plasma Derived - Fall 2010 Citigroup projections.
Global sales, 2010 ($ M)
FVIII
Kogenate FS
(Bayer)*
Advate/Recombinate
(Baxter)
0
200 0
1000
Plasma Derived
Helixate FS Xyntha/
(CSL Behring) Refacto AF Factor (Various)
400 0
2000
600 0
3000
Source: 2010 Annual reports for Bayer, Pfizer, Baxter. Internal estimates
and secondary reports for remainder.
*Excludes sales to CSL Behring
21
(Pfizer)
800 0
4000
100 00
120 00
5000
B-LONG only
Factor Use Relatively High in Sobi Territory
Per Capita consumption of FVIII (IU/Inhab.)
~55% of global factor sales
are in the Sobi territory – an
approx. ~$3.4 B market
Sweden
Germany
Ireland
Hungary
France
Italy
USA
Spain
Norway
Poland
Switzerla…
Russia
Portugal
Greece
Czech
Lithuania
Bulgaria
Turkey
Romania
0
Source: WFH 2009
22
2
4
6
8
10
World Federation of Hemophilia Annual Global Survey
Hemophilia Patient Segmentation
~100,000
Total Haemophilia
Treated
Patients
Treated Patients (A&B)
(A&B)
24% <18 years
1/3 pts
2/3 pts
76%> 18 years
10% Surgery
Induction approx 0,3%
Elective and Emergency
60% Prophylaxis
20 % Prophylaxis
Per Treatment
Per Surgery
100k$ /year/pt
300k$ /year/pt
600k$/year/pt
100k$/surgery/pt
40% On Demand
40k$/year/pt
80% On Demand
100k$ /year/pt
Source: WFH Annual Global Survey 2010
UK Haemophilia Centre Doctors Organisation Data – Report 2010
Data varies by country and territory
23
2% Immune
Tolerance
Hemophilia Programs: Positive Top Line Data
Factor 9: B-LONG
Safety Summary
• No inhibitors to rFIXFc were detected
• No cases of anaphylaxis were reported
1.
Prophylactic regimens resulted in lowsingle-digit annualized bleeding rates
2.
Median dosing interval was 14 days in
the individualized interval
prophylaxis arm during last 6 months
3.
Greater than 90% of bleeding
episodes were controlled by
a single injection of rFIXFc
24
• Most common adverse events (incidence of
≥5%) occurring outside of the perioperative
management arm were nasopharyngitis,
influenza, arthralgia (joint pain), upper
respiratory infection, hypertension and
headache
• One serious adverse event was assessed to
be possibly related to drug by the
investigator. The patient experienced
obstructive uropathy in the setting of
hematuria; he continued rFIXFc treatment
and the event resolved with medical
management
B-LONG Trial Summary
Sequential PK*
BeneFIX®
rFIXFc
Nonsequential PK
rFIXFc
MEDIAN ABR 2.95
Arm 1
Weekly prophylaxis regimen
Arm 2
Individualized interval
prophylaxis regimen
Up to 52 weeks
Optimize
Dose
Weekly
Up to 52 weeks
Optimize
Interval
N=63
MEDIAN ABR 1.38
N=29
MEDIAN ABR 17.69
Arm 3
Episodic treatment
Arm 4
Perioperative management
N=27
N=12
*PK subgroup (n=16) dosed with BeneFIX, then crossed over to rFIXFc.
rFIXFc=recombinant factor IX Fc fusion protein; PK=pharmacokinetics.
NCT01027364. Available at http://www.clinicaltrials.gov/ct2/show/NCT01027364?term=rFIXFc&rank=1. Accessed September 24, 2012.
25
Up to 52 weeks On-Demand
Comparison
10 surgeries
in ≥5 patients
Hemophilia Programs: Positive Top Line Data
Safety Summary
• No inhibitors to rFVIIIFc were detected
1.
Individualized and weekly prophylactic
regimens resulted in low-single-digit
annualized bleeding rates
2.
Median dosing interval was 3.5 days in
the individualized prophylaxis arm
3.
98% of bleeding episodes were
controlled by one or two injections of
rFVIIIFc
26
• No cases of anaphylaxis were reported
• The most common adverse events
(incidence of ≥5%) occurring outside of the
perioperative management arm were
nasopharyngitis, arthralgia, headache and
upper respiratory tract infection
• No serious adverse events were assessed
to be related to drug by the investigator
A-LONG Trial Summary
Sequential PK
(Advate® vs rFVIIIFc)
MEDIAN ABR 1.6
Arm 1
Individualized prophylaxis
Optimize
ABR / Protection
N=118
Surgery subgroup
MEDIAN ABR 3.6
Screening
Arm 2
Weekly prophylaxis
Randomization
N=24
Surgery subgroup
MEDIAN ABR 33.6
Arm 3
Episodic treatment
N=23
Surgery subgroup
Advate (Baxter) is an antihemophilic factor (recombinant) plasma/albumin-free method.
PK=pharmacokinetics; rFVIIIFc=recombinant factor VIII Fc fusion protein.
NCT01181128. Available at http://clinicaltrial.gov/ct2/show/NCT01181128. Accessed September 24, 2012.
27
Offer Weekly
Option for
On-Demand
What Does Shifting the Curve Mean?
ILLUSTRATION ONLY – NOT DATA
2X per week
3X per week
Relative
Number
Of
Patients
1
2
Number of
Infusions Per Week
28
3
4
Launch in Sobi Territories will follow US
2010
rFIXFc
B-LONG
2011
2012
H1
2016
H2
Start of Pediatric
studies H1
A-LONG
Start of
Phase III
studies
H1
Launch
in
Sobi Territory
H2
Data
Read
Out
Start of Pediatric
studies H1
29
2014
Data
Read
Out
Start of
Phase III
studies
rFVIIIFc
2013
B-LONG only
Kiobrina
Kiobrina – Oral ERT for Premature Infants
Kiobrina is a recombinant
human lipase (BSSL) given
together with formula or
pasteurized milk to pre-term
infants, in development:
• To increase growth rate
• To reduce length of stay in the NICU,
• To improve long-term development
outcomes.
31
Kiobrina – Physiology
1.
Bile Salt Stimulated Lipase (BSSL) is needed in the
infant GI tract to digest long chain fatty acids (LCFA)
2.
BSSL naturally occurs in mother’s milk
3.
Premature infants do not produce BSSL
a.
4.
Lack of BSSL is correlated with slower growth in
neonatal period
BSSL not present in pasteurized milk or formula
 Kiobrina aims to replace normal quantity and activity
of native BSSL
32
Phase II: Potentially Significant Clinical Value
Growth model for a 1kg pre-term infant to reach 1.8kg
Growth Velocity ( g/kg/d)
18
16.9*
16
14
13.9
7 days
* p<0.001
12
Placebo
(Formula /pasteurized milk)
Kiobrina +
Formula/pasteurized milk
The phase II Kiobrina program was designed as two parallel prospective
randomized double-blind crossover studies where Kiobrina, or placebo,
was administered in pasteurized milk, or preterm infant formula, during
one week of treatment. All infants were born before week 32 of
gestational age.
33
Source: Internal modeling; Sobi.
Phase III Pivotal Trial Underway in Europe
• Ongoing Phase III study primary endpoint is
Growth Velocity after four weeks treatment
• 12 months total observation period
• A 24 month extension study has been initiated to
capture longer term development outcomes
* Design and timeline to be finalized.
34
Cross-Sectional Data: Growth Velocity & Risk of Impairment
% Patients w/ Cerebral Palsy
% Patients w/ Impaired Neurodevelopment
25
60
50
20
40
15
30
10
20
5
10
0
12 g*kgˉ¹*dˉ¹
16 g*kgˉ¹*dˉ¹
18 g*kgˉ¹*dˉ¹
21 g*kgˉ¹*dˉ¹
Growth Velocity
Ehrenkranz et al. Pediatrics, 2006; 117;1253
35
0
12 g*kgˉ¹*dˉ¹
16 g*kgˉ¹*dˉ¹
18 g*kgˉ¹*dˉ¹
Growth Velocity
21 g*kgˉ¹*dˉ¹
Kiobrina Could Help >275,000 Patients Globally*
86,000
Filing Based on
EU Dossier
24,000
US
Latin America*
178,000
Korea
Australia
MENA*
China
US Pivotal Study
*Lat AM: Brazil, Mexico, Argentina, Venezuela
*MENA: Saudi, Iran, Israel, Egypt, Turkey
36
Current Phase III Study
Russia
20,000
EU
Bridging Studies
(based on EU File)
Strategic Priorities
1.
Near term focus on growth in key
therapeutic areas, with sustainable
positive cash flow from operations.
2.
Medium-term investments to ensure
successful commercialization of our
late-stage pipeline.
3.
Long-term growth will come
organically and through acquisitions
in key therapeutic areas.
Q4 and Full Year Earnings: 21 February 2013
37
Reference
38
100
400
75
300
50
200
25
100
Quarterly
39
Q3-12
Q1-12
Q3-11
Q1-11
Q3-10
0
Q1-10
0
4 quarters rolling
4 quarters rolling revenues (SEK M)
Quarterly revenues (SEK M)
Orfadin® – On Track Year to Date
• Sales of Orfadin in the third quarter
were USD 12 M, an increase of 3%
• Adjusted for currency:
• Revenues increased by 6% y/y
• Revenues increased by 10% YTD
• North America and Central Eastern
Europe contributing to growth
• Development in EMENA affected by
negative currency effects and purchasing
pattern
• Geographic expansion
• Sobi Middle East established in Dubai
• New orphan legislation in Russia in place
Orfadin® – New Formulation Meets Market Need
Rationale
1.
Enables precise dosing
2.
Expands compliance
3.
IP Extension (orphan status EU 2017)
Status
• Manufacturing scale-up underway
• Clinical program underway
• EMA PIP approved March 2012
17