extending legacy of leadership in...
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Extending Legacy Of Leadership In Hematology
Brian Goff President, Hematology
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Deliver transformative and personalized
therapies to improve the lives of patients with blood disorders
Our Strategic Priority:
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Key takeaways
• Strengthening our hematology leadership for sustainable and profitable growth
• Building personalized care through patient-centric innovation
• Elevating access to treatment and standards of care globally
• Diversifying to transform care in adjacent blood disorders by leveraging our core expertise
Poised For 3% - 5% Sales CAGR Across Hematology Business Through 2020
Extending Legacy Of Leadership In Hematology
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Disease description & pathology
• Hemophilia is a congenital bleeding disorder caused by a deficiency or absence of FVIII or FIX
• Most common complication is development of chronic arthropathy due to repeated bleeding into joints
• Hemorrhages into skin, muscles, soft tissue and mucous membranes
• Intracranial hemorrhage, a serious life-threatening complication, occurs in ~2% of patients1
• ~$10B market size (Hemophilia A & B and Inhibitors) growing to ~$13B by 2020 at CAGR of 4% - 6%
Hemophilia Is A Rare Bleeding Disorder Affecting ~480,000 People Worldwide
Data Source: World Federation of Hemophilia; Baxalta internal analysis, 1 Witmer, C., et al. (2011). Br J Haematol 152(2): 211-216.
80-85% Hemophilia A:
deficiency of FVIII
15-20% Hemophilia B:
deficiency of FIX
Over 75% of Hemophilia A & B
patients are in developing countries
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Challenges and opportunities
• Improvement in diagnosis and treatment, especially in developing countries
• Adequate diagnosis and care for patients with inhibitors:
– 30% of Hemophilia A and 5% of Hemophilia B patients develop inhibitors to factor treatment
• Wider adoption of prophylaxis
• Optimization of outcomes through personalization of care
• Sustained high trough levels to enhance quality of life
Significant Opportunity To Raise Global Standard Of Care
Majority Of Hemophilia Patients Worldwide Lack Adequate Treatment
30% Treated
480,000 Worldwide
Hemophilia A & B population
40% Diagnosed
8% Prophylaxis
3% Achieve Zero Annual
Bleeds
Data Source: World Federation of Hemophilia Annual Global Survey 2013; Baxalta internal analysis
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Baxalta Hematology Revenue
Hematology: Delivering Sustainable Growth Of 3% - 5%
Strengthening Hemophilia leadership
through innovation
Raising standard of care for patients with
Inhibitors
Expanding global access and improving treatment standards
Accelerating game-changing therapies in
blood disorders
~$3.5B
$3.5 $3.8 $4.1
~$4.3B
$0
$2
$4
$6
2015Expectation*
HemophiliaA & B
InhibitorTherapy
BloodDisorders
2020Outlook
$ In
Bill
ions
CAGR 3% - 5%
New Products $1B+
BAX 855 BAX 817 OBIZUR RIXUBIS VONVENDI** BAX 555
*Actual rates **VONVENDI = BAX 111
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Therapy advancements have made zero annual bleeds a
reality for some patients
Strengthening Hemophilia leadership through innovation
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Market dynamics
• Competitive entries will double
• Growing austerity measures
Baxalta Hemophilia Revenue*
Hemophilia A & B: Solid Foundation For Sustainable Growth
A high bar with ADVATE-based therapy • Direct factor replacement to address
well-understood pathophysiology
• Proven safety and effectiveness
• Zero annual bleeds through prophylaxis
• Improving outcomes through personalization of care
~$2.7B
$0.0
$1.0
$2.0
$3.0
2015Expectation
BaseFVIII & FIX
BAX 855 2020Outlook
CAGR 1%-3%
*Reflects sales of FVIII and FIX therapies **At actual rates
**
~$3.0B
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ADVATE: The Worldwide rFVIII Leader Approved In Over 65 Countries
1 Iorio A, Marcucci M, Cheng J, et al. Patient data meta-analysis of Post-Authorization Safety Surveillance (PASS) studies of haemophilia A patients treated with rAHF-PFM. Haemophilia. 2014:1-7
Proven Safety in the Real World
Proven Effectiveness in the Real World
Personalization with Reliable Pharmacokinetic
(PK)-based dosing
1.66 Annual Bleed Rate (ABR) Real World1
0.15% incidence of de novo inhibitors in severe Previously Treated
Patients (PTPs)1
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Building On A Well-Understood Scientific Platform With Direct Factor Replacement
Bridging Market Insights To Science To Drive Innovation
• Freedom from pain
• To engage in activities of daily living
• A treatment that works for them and fits their lifestyle
• A treatment that is safe
• A cure
• Efficacy profile to achieve zero bleeds
• Reliable assays for optimum dose-tailoring
• Lifestyle-aligned personalization
• Well established long-term safety with low inhibitor risk
• Sustained multi-year elevated trough levels with innovation in gene therapy
Patient Needs Our Solution
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We Strive For A Bleed-Free World… One Person At A Time
EVERY BLEED MATTERS
PROPHYLAXIS FOR ALL
PERSONALIZED FOR ONE
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Right Product + Right Dosing Regimen = Personalized Outcomes
High Patient Variability Drives Need For Personalized Treatment Patient factors Treatment requirements
Variability in patient PK parameters: Product half-life and dosing regimen to achieve optimum factor activity levels between infusions
PK Response to Clotting Factor
Bleed Risk/Phenotype
Patient Lifestyle/
Physical Activity Levels
Frequency and intensity of physical activity: Peak factor protection and time spent in ‘safe zone’ at time of physical activity
Bleed risk and joint health: Patient-specific baseline factor activity levels and time spent in ‘safe zone’
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Annual Number Of Joint Bleeds According To FVIII Activity
Higher Baseline FVIII Activity Level Is Associated With Fewer Annual Joint Bleeds
0
2
4
6
0 5 10 15 20
Annu
al N
umbe
r Of J
oint
Ble
eds
% FVIII Activity (IU dL-1)
Baseline activity levels of over 10% were shown to be associated with significant decrease in annual joint
bleeds
(N= 377)
Hemophilia Severity
Severe (<1%)
Moderate (1% - 5%)
Mild (5% - 40%)
Adapted from den Uijl, et al, 2011
den Uijl IEM et al. Haemophilia. 2011, 17: 849-853
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Annual Number Of Joint Bleeds According To FVIII Activity
Changing Treatment Paradigm With Gene Therapy
Portfolio Strategy To Elevate Baseline FVIII Activity Level
0
2
4
6
0 5 10 15 20
Annu
al N
umbe
r Of J
oint
Ble
eds
% FVIII Activity (IU dL-1)
Base FVIII
Factor Replacement Therapies
Extended Half-Life
Gene Therapy
Adapted from den Uijl, et al, 2011
Hemophilia Severity
Severe (<1%)
Moderate (1% - 5%)
Mild (5% - 40%)
den Uijl IEM et al. Haemophilia. 2011, 17: 849-853
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• High structural and functional consistency
• Extensively tested for safety and efficacy prior to clinical development
• Successful Phase III clinical trial
– 1.4-1.5 fold increase in ADVATE’s half-life
– 95% reduction in median ABR vs. on-demand
– No patients developed inhibitors
• Accelerated product development and robust manufacturing positions Baxalta for competitive market entry
• U.S. BLA filed in Q4 2014; Japan Q2 2015
• U.S. pediatric and EU filing expected in 2016
BAX 855 Is Built On The Proven Safety And Efficacy Of ADVATE
Building An ADVATE-Based Family Beginning With BAX 855
BAX 855
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• Polysialylation of full-length rFVIII with no mutation in the protein structure
• Prolong ADVATE half-life; potential for once-weekly or less frequent dosing
• Leveraging real world long-term safety and efficacy of ADVATE-based direct factor replacement
• Expect to initiate Phase I clinical trial end of 2015
Prolong ADVATE Half-Life And Reduce Dosing Frequency With BAX 826
Expanding The ADVATE-Based Family With BAX 826
BAX 826
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Gene therapy programs in Hemophilia
• Acquired Asklepios BioPharmaceuticals (Chatham Therapeutics) Hemophilia Gene Therapy program in 2014
• BAX 888 (FVIII) and BAX 335 (FIX) induces the body to produce factor
• BAX 888 FVIII gene construct selection under evaluation
• Phase I/II trial in progress to assess safety and optimal dosing level of BAX 335
• 7 patients treated in three dosing cohorts; FIX activity level ~10% or above in two patients; no bleeding events or inhibitor development
• Additional data at ISTH in June 2015
Shifting The Paradigm In Hemophilia Care With Sustained High Factor Levels
Our Gene Therapy Pipeline Offers Potential For Multi-Year Treatment
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Raising standard of care for patients with Inhibitors
Only 35% of patients with Inhibitors are on prophylaxis
in developed countries
Data Source: Baxalta internal analysis
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Raising The Bar On Outcomes For Patients With Inhibitors
Addressing Key Unmet Needs For Fragile Patient Segment
Acute Bleed Control
• Rare autoimmune disease
• Unmet needs: delay in diagnosis and treatment, high patient mortality (20%)
ACUT
E/
EMER
GEN
CY
Acquired Hemophilia A
(~10,500 patients/y) 2
1 World Federation of Hemophilia Annual Global Survey 2013, 2 Collins PW, et al. Blood. 2007 Mar 1; 109(5):1870-7
CHRO
NIC
/ LO
NG-
TERM
Prophylaxis
Congenital Hemophilia
with Inhibitors (~5,000 diagnosed
patients) 1
• Development of anti-bodies that neutralize clotting factor
• Unmet needs: Low prophylaxis rates, limited choice of by-pass therapy agents
PIPELINE
BAX 817 (rFVIIa)
Anti-TFPI
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Key studies
• FEIBA PROOF Study: Prospective, randomized, open label study of FEIBA prophylaxis vs. on-demand therapy showed significant reduction in bleeds1
• FEIBA GO Study: Long-term prophylaxis observational study to track outcomes; interim results expected in 2017
FEIBA prophylaxis
• Only bypass therapy indicated for prophylaxis • Growth driven by increase in global
prophylaxis; opportunity due to low (~35%) prophylaxis rate
Differentiated Label With FEIBA Prophylaxis
Strong Bleed Reduction With FEIBA Prophylaxis
Reduced median
ABR for all bleeds by 72.5%
(P=0.0003)
When compared with FEIBA on-demand treatment, FEIBA prophylaxis 85±15 U/kg given every other day
Global FEIBA Revenue
$0.0
$0.5
$1.0
2012 2013 2014
$ In
Bill
ions
~10% CAGR*
1 Antunes SV,et al. Haemophilia. 2014;20(1):65-72. *Constant Currency
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OBIZUR meets a significant unmet need for patients • Only therapy for AHA that allows for
measurement of FVIII
• Elevates clinician confidence by providing faster response time, better safety and optimum dosing
• 100% of bleeds successfully treated since U.S. launch
• Regulatory review ongoing in EU, Colombia, and Canada
• Exploring additional indications
$250M Peak Sales Opportunity
OBIZUR: First And Only Clinically Approved Treatment For Acquired Hemophilia A
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70% of Hemophilia patients worldwide do not receive
any or adequate treatment
Data Source: World Federation of Hemophilia, Baxalta internal analysis
Expanding access and improving global treatment standards
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Global Patients By Regimen & Severity Global Patients By Regimen & Severity
Majority Of Hemophilia Patients Do Not Have Access To Prophylaxis Treatment
0%
25%
50%
75%
100%
Mild Moderate Severe
Prophylaxis On-Demand
% O
f Pat
ient
s
~15% Of Patients On Prophylaxis Treatment
% O
f Pat
ient
s
~30% Of Patients On Prophylaxis Treatment
0%
25%
50%
75%
100%
Mild Moderate Severe
Prophylaxis On-Demand
Data Source: Baxalta internal analysis
Hemophilia A Hemophilia B
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% Prophylaxis Penetration Severe Hemophilia A Patients
Expanding Prophylaxis Opportunity In Developed Markets
98% 90% 87% 84%
70% 71%
51%
68%
Germany U.S. France Japan
Pediatric (0-18 years) Adult (18+ years)
Opportunity
Data Source: Baxalta internal analysis
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Treatment Rates Recombinant Conversion
>20 Billion IU Potential If UK Standard Of Care Is Achieved
Achieving Higher Standard Of Care In Developing Countries
Data Source: World Federation of Hemophilia Annual Global Survey 2013, China - 2012 MRB report; Baxalta Internal Analysis
0%
25%
50%
75%
100%
UK Russia Turkey Brazil China India
0
2
4
6
8
UK Russia Turkey Brazil China India
IU/C
apita
% rF
VIII
By U
nits
UK: 7.7 IU/Capita UK: 90% Recombinant FVIII
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Strategic initiatives
Leveraging Global Presence And Strong Relationships In Local Markets To Raise Standards Of Care
Implementing Diverse And Localized Strategies In Developing Countries
RUSSIA • Local manufacturing agreement for Hemophilia therapies • ADVATE launched in 2014; FEIBA included in national tender
TURKEY • ADVATE launched in 2014: 400+ patients added since 2014
BRAZIL • 20-year exclusive partnership with Hemobrás • 4,000+ patients now have access to ADVATE
CHINA • Improving affordability and access to ADVATE with patient
access programs • Local partnerships with city-based funding organizations
INDIA • Awareness and education among stakeholders • Secure Government funded access; improve infrastructure
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Brazil Conversion To rFVIII
Brazil: Long-Term Growth Strategy Through Hemobrás Partnership
Amauri has been on ADVATE prophylaxis since it was launched in Brazil
0% ~25%
40%+ 50%+
65%
1.68
2.55 2.79
2.95
4.10
2012 2013 2014 2015E 2020E
% rF
VIII
Patie
nts
IU/Capita
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Accelerating game-changing therapies in other blood disorders
Bringing paradigm shift to current therapies
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Building Depth And Breadth To Compete In A $16B+ Market
Leveraging Heritage In Hemophilia To Expand In Other Blood Disorders
Current therapy areas in Hemophilia Future growth in Blood Disorders
Hemophilia A Hemophilia with inhibitors
Sickle Cell Disease
Hereditary TTP*
Disease Population ~400,000 ~80,000 ~20,000-
40,000 ~70 M
(~700K require treatment)
~3,000-4,000 ~200,000 (U.S. and EU)
Hemophilia B von Willebrand Disease
Depth Breadth
Segment Size in 2020 = ~$13B
Under-penetrated Segment Potential = ~$3.0B
*Thrombotic Thrombocytopenic Purpura
Data Source: World Federation of Hemophilia, CDC, NIH, EMA, answeringttp.org, Baxalta internal analysis
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VONVENDI (BAX 111): The First Recombinant Pure von Willebrand Factor
VONVENDI (BAX 111) For Hereditary von Willebrand Disease (vWD)
Disease Description
• Most common inherited bleeding disorder; prevalence of symptomatic disease:1in 10,0001
• Low prophylaxis (~20%) in Severe Type 3 • Currently treated with desmopressin and/or combination pdFVIII/vWF
Product Attributes and Targeted Indications
• First highly purified recombinant vWF
• Can be used with or without FVIII therapy for added flexibility
• Advanced manufacturing to preserve the vWF profile including ultra-large multimers
Studies and Timelines
• Phase III met its primary efficacy endpoint
• U.S. BLA submission Q4 2014
• Surgery study initiated Q4 2014
Potential
• ~$1.0B peak sales opportunity
• Significantly under-developed segment
• Exploring additional indications
1 Ng C,et al. Blood. 2015 Feb 23.
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BAX 930: First Disease-Specific Treatment For hTTP
BAX 930 For Hereditary Thrombotic Thrombocytopenic Purpura (TTP)
Disease Description
• Life-threatening coagulation disorder affecting 3,000 - 4,000 patients globally
• Currently treated with infusions of fresh frozen plasma (FFP)
Product Attributes and Targeted Indications
• First recombinant factor replacement therapy with rADAMTS13
• For on-demand treatment of TTP episodes and prophylactic treatment of hereditary TTP
Studies and Timelines
• Preclinical IND-enabling studies completed
• Phase I initiated in 2014
Potential • ~$1.0B including additional indications
• New therapy options expected to outgrow the market
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BAX 555: A Potential New Treatment Option For Patients With Sickle Cell Disease
BAX 555 For Sickle Cell Disease
Disease Description
• World’s most common blood disorder with ~200,000 patients diagnosed in U.S./Europe
• Currently only one approved drug — low adoption rates and requires frequent monitoring
Product Attributes
• First-in-class, oral, small molecule compound • Early studies indicate the compound may reduce the sickling of red blood cells
Studies and Timelines
• Phase I studies completed • First cohort of Phase II study initiated in 2014 • Received FDA Orphan designation; eligible in EU
Potential • ~$1.0B • New therapy options expected to expand market
potential
![Page 33: Extending Legacy Of Leadership In Hematologys2.q4cdn.com/643609687/files/doc_events/2015/Hematology_FINAL.pdf · • Strengthening our hematology leadership for sustainable and profitable](https://reader031.vdocuments.site/reader031/viewer/2022030510/5ab9e5977f8b9ad5338e7a4f/html5/thumbnails/33.jpg)
Key takeaways
• Strengthening our hematology leadership for sustainable and profitable growth
• Building personalized care through patient-centric innovation
• Elevating access to treatment and standards of care globally
• Diversifying to transform care in adjacent blood disorders by leveraging our core expertise
Poised For 3% - 5% Sales CAGR Across Hematology Business Through 2020
Extending Legacy Of Leadership In Hematology
![Page 34: Extending Legacy Of Leadership In Hematologys2.q4cdn.com/643609687/files/doc_events/2015/Hematology_FINAL.pdf · • Strengthening our hematology leadership for sustainable and profitable](https://reader031.vdocuments.site/reader031/viewer/2022030510/5ab9e5977f8b9ad5338e7a4f/html5/thumbnails/34.jpg)
Baxalta Incorporated 2015 Investor Conference