Takeda Pharmaceutical Co. Ltd. · TSE:4502
Takeda Pharmaceutical — Investor Day (December 2020)
162 slides from Takeda Pharmaceutical's investor day presentation, Dec 8, 2020
Slide 1: Wave 1 Pipeline Market Opportunity Call
WAVE 1 PIPELINE MARKET OPPORTUNITY CALL December 8, 2020 (ET) / December 9, 2020 (JST) Takeda Pharmaceutical Company Limited
Slide 2: Important notice and forward-looking statements
For the purposes of this notice, “presentation” means this document, any oral presentation, any question and answer session and any written or oral material discussed or distributed by Takeda Pharmaceutical Company Limited (“Takeda”) regarding this presentation. This presentation (including any oral
Slide 3: Agenda
AGENDA TIME (EST) TIME (JST) AGENDA 17:00 – 17:10 07:00 – 07:10 Takeda: A Global Values-Based, R&D-Driven Biopharmaceutical Leader Christophe Weber, President & CEO 17:10 – 17:25 07:10 – 07:25 Following Through On Our Commitment To Deliver Innovative Medicines To Patients: Spotlight On Select Wave 1
Slide 4: Agenda
AGENDA TIME (EST) TIME (JST) AGENDA 17:00 – 17:10 07:00 – 07:10 Takeda: A Global Values-Based, R&D-Driven Biopharmaceutical Leader Christophe Weber, President & CEO 17:10 – 17:25 07:10 – 07:25 Following Through On Our Commitment To Deliver Innovative Medicines To Patients: Spotlight On Select Wave 1
Slide 5: A Values-Based and R&D-Driven Biopharmaceutical Leader
A VALUES-BASED AND R&D-DRIVEN BIOPHARMACEUTICAL LEADER 4 | Better health for people, brighter future for the worldPURPOSE Discover and deliver life-transforming treatments, guided by our commitment to patients, our people and the planet VISION IMPERATIVES PATIENT • Responsibly translate science into
Slide 6: Transformation to Top Global R&D-Driven Biopharma Company
TRANSFORMATION TO TOP 10 GLOBAL R&D-DRIVEN BIOPHARMA COMPANY 2014 Today Next 10 Years Strategic Evolution We Are One Takeda Accelerating Growth & Patient Impact GLOBALIZATION R&D TRANSFORMATION VALUES BASED, R&D-DRIVEN GLOBAL BIOPHARMA COMPANY 5 KEY BUSINESS AREAS AND 14 GLOBAL BRANDS 12 NEW MOLECUL
Slide 7: Global Brands Potential to Deliver Significant Growth
14 GLOBAL BRANDS HAVE POTENTIAL TO DELIVER SIGNIFICANT GROWTH WITH INCREMENTAL REVENUE OPPORTUNITY OF >$8B BY FY2024 1. Includes Albumin Glass, Flexbumin and Kenketsu Albumin. 2. USD included for reference calculated at JPY/USD of 107 yen. 3. New peak revenue estimates for these products are based o
Slide 8: Product indication, market opportunity and peak revenue potential
PRODUCT INDICATION FULL MARKET OPPORTUNITY2 TAKEDA’S PEAK REVENUE POTENTIAL3 Orexin programs4 Narcolepsy type 1 (NT1) Narcolepsy type 2 (NT2) Idiopathic hypersomnia $3,000 – 4,000MN (NT1) $1,000 – 2,000MN (NT2 + IH) soticlestat (TAK-935) Lennox-Gastaut syndrome, Dravet syndrome and other indications
Slide 9: Positioned for organic and sustainable revenue growth
PTS ADJUSTED4 FY2019-FY2030 Revenue CAGR: Low single digit 4,000 6,000 5,000 0 1,000 2,000 3,000 FY2024 14 Global Brands 14 Global Brands Hemophilia Wave 2 PTS adjusted3,4 Vyvanse/Velcade/Azilva All Others FY2019 Actual Wave 2 non-PTS adjusted1,3 Wave 1 non-PTS adjusted1 Wave 1 PTS adjusted4 14 Glob
Slide 10: Today's Speakers
TODAY’S SPEAKERS CHRISTOPHE WEBER President & CEO ANDY PLUMP President, Research & Development RAJEEV VENKAYYA President, Global Vaccine Business Unit Available for Q&A COSTA SAROUKOS Chief Financial Officer TERESA BITETTI President, Global Oncology Business Unit RAMONA SEQUEIRA President, USBU & Gl
Slide 11: Agenda
AGENDA TIME (EST) TIME (JST) AGENDA 17:00 – 17:10 07:00 – 07:10 Takeda: A Global Values-Based, R&D-Driven Biopharmaceutical Leader Christophe Weber, President & CEO 17:10 – 17:25 07:10 – 07:25 Following Through On Our Commitment To Deliver Innovative Medicines To Patients: Spotlight On Select Wave 1
Slide 12: Following Through On Our Commitment to Deliver Innovative Medicines
Following Through On Our Commitment to Deliver Innovative Medicines To Patients Spotlight on Select Wave 1 Programs Andy Plump President, R&D
Slide 13: R&D Focus and Innovative Pipeline
PLASMA-DERIVED THERAPIES VACCINES CELL THERAPY GENE THERAPY DATA SCIENCES ONCOLOGY GASTROENTEROLOGY (GI)NEUROSCIENCE RARE GENETIC & HEMATOLOGY R&D FOCUS INNOVATIVE BIOPHARMA INNOVATIVE PIPELINE • 12 Wave 1 NMEs 5 programs with BTD, 3 with FTD and 1 with Sakigake designation • ~30 Wave 2 NMEs A GLOBA
Slide 14: Accessing innovation through a network of partners
Total Upfront Investment ~$1B IN FY19/20 Access to Innovation Risk-Sharing Expanding Capacity IN-LICENSE RESEARCH COLLABORATIONS NEWCO FORMATION JOINT DEVELOPMENT ~ 150 ~ 50 ~ 20 ~ 25 WE ARE ACCESSING INNOVATION BY INTEGRATING TAKEDA’S WORLD CLASS LABORATORY WITH A NETWORK OF PARTNERS Non-Viral Gene
Slide 15: Momentum in our dynamic pipeline based on emerging data
MOMENTUM IN OUR DYNAMIC PIPELINE BASED ON EMERGING DATA 1. Projected approval dates depend on data read-outs; some Wave 1 target approval dates assume accelerated approval 2. Potential for data driven acceleration of some Wave 2 programs into Wave 1 3. Approval date assumes filing on Phase 2 data 4.
Slide 16: Upcoming regulatory and data milestones
DEVELOPMENT STAGE PROGRAM INDICATION NEXT MILESTONE EXPECTED TIMING Regulatory Milestones TAK-721 Eosinophilic esophagitis Approval Q4FY201 TAK-003 Prevention of dengue fever Submission Q4FY20 TAK-609 Hunter syndrome CNS Submission Q4FY20 mobocertinib NSCLC exon 20 insertion mutation (2L) Submission
Slide 17: First in a series of investor events
TODAY IS THE FIRST IN A SERIES OF INVESTOR EVENTS TO CONNECT CLINICAL DATA TO THE MARKET POTENTIAL OF OUR WAVE 1 PIPELINE 1. Date to be confirmed TODAY’S EVENT APRIL 6, 20211 Regulatory filing for Dengue vaccine in endemic region US NDA submission for eosinophilic esophagitis PART 1 WAVE 1 MARKET OP
Slide 18: Agenda
AGENDA TIME (EST) TIME (JST) AGENDA 17:00 – 17:10 07:00 – 07:10 Takeda: A Global Values-Based, R&D-Driven Biopharmaceutical Leader Christophe Weber, President & CEO 17:10 – 17:25 07:10 – 07:25 Following Through On Our Commitment To Deliver Innovative Medicines To Patients: Spotlight On Select Wave 1
Slide 19: Entyvio Case Study
Entyvio Case Study: Building A Gold Standard Therapy Along The Patient Journey Ramona Sequeira President, USBU & Global Portfolio Commercialization
Slide 20: Entyvio: The Gold Standard Therapy for IBD
19 | ENTYVIO: THE GOLD STANDARD THERAPY FOR IBD illustrative FY2019 Actual SUBCUTANEOUS LAUNCH1 Peak revenue estimate4 Leadership in IBD $5.5-6.5BN $3.2BN3 1. Per approval by country Regulatory Authority 2. Based on head-to-head trial that compared clinical remission rates for Entyvio and adalimumab
Slide 21: Global Multi-Pronged Growth Strategy for Entyvio
20 | GLOBAL MULTI-PRONGED GROWTH STRATEGY TO MAXIMIZE POTENTIAL OF ENTYVIO 1. Per approval by country Regulatory Authority 2. Based on head-to-head trial that compared clinical remission rates for Entyvio and adalimumab in adults with moderately to severely active UC SUBCUTANEOUS (SC) LAUNCH1 GEOGRA
Slide 22: Robust research portfolio and real world evidence
Source: Sands et al. Vedolizumab versus Adalimumab for Moderate-to-Severe Ulcerative Colitis. N Engl J Med 2019; 381:1215-1226 Lukin D, et al; on behalf of VICTORY Collaboration. Comparative safety and effectiveness of vedolizumab to tumour necrosis factor antagonist therapy for ulcerative colitis.
Slide 23: Bringing our pipeline to life
22 | Bringing our pipeline to life GLOBAL CAPABILITIES to deliver LIFE TRANSFORMING TREATMENTS LAUNCH EXCELLENCE Patient Journey & Diagnosis Data, Insights & Analytics Patient Services Value Based Partnerships Digital Evidence Generation
Slide 24: Agenda
AGENDA 23 | TIME (EST) TIME (JST) AGENDA 17:00 – 17:10 07:00 – 07:10 Takeda: A Global Values-Based, R&D-Driven Biopharmaceutical Leader Christophe Weber, President & CEO 17:10 – 17:25 07:10 – 07:25 Following Through On Our Commitment To Deliver Innovative Medicines To Patients: Spotlight On Select W
Slide 25: TAK-721 Deep Dive
TAK-721 Deep Dive: Potential To Be First FDA-Approved Agent With Indication For Eosinophilic Esophagitis Michael Nedham TAK-721 Global Program Lead, Global Product & Launch Strategy
Slide 26: Key Takeaways for TAK-721 in EoE
KEY TAKEAWAYS FOR TAK-721 IN EOE (EOSINOPHILIC ESOPHAGITIS) 1 32 TAK-721 is an investigational treatment and has not been approved for use by the U.S. Food and Drug Administration or other regulatory authorities. Neither Breakthrough Therapy Designation nor Orphan Drug Designation guarantee approval
Slide 27: What is EoE?
Over time, untreated chronic inflammation can progress to fibrotic disease, narrowing of the esophagus, and associated food impactions. Diagnosed through biopsy confirming high eosinophil count (>15/hpf) in the esophagus. EoE is a rare, chronic, inflammatory, immune-mediated disease of the esophagus
Slide 28: What is it like for patients living with EoE?
WHAT IS IT LIKE FOR PATIENTS LIVING WITH EOE? Physical PAIN Staying NOURISHED Social ISOLATION 27 | “I sometimes go for days or weeks without an impaction, but I always feel like I’m living on the edge of another choking incident. I wish I could go back to not having to constantly think about food.”
Slide 29: Patient journey involves multiple specialties and long diagnosis times
PATIENT JOURNEY INVOLVES MULTIPLE SPECIALTIES AND LONG DIAGNOSIS TIMES 28 | PRE-EOE DIAGNOSIS (6 MONTHS) EOE DIAGNOSIS PROCEDURE 1ST EOE DIAGNOSIS TREATMENT INITIATION PERSISTENCE • 72% of patients are diagnosed with endoscopy • 25% have had an endoscopy prior but may not have had a biopsy Rxs: • 50
Slide 30: EoE is relatively new and rare, but prevalence is growing
EOE IS STILL RELATIVELY NEW AND RARE, BUT PREVALENCE IS GROWING 29 | Approximately 1 in 2000 people in the US live with EoE1-5 Emerging evidence suggests environmental factors such as microbes, early-life events affecting the microbiome, and other factors may be contributing to the rise in prevalenc
Slide 31: Academic interest and research activities ramping up
ACADEMIC INTEREST AND RESEARCH ACTIVITIES RAMPING UP First international consensus recommendations on EoE Initial series characterizing EoE in adults AGA/AAAAI joint taskforce guidelines Case reports of EoE in adults Topical steroid therapy prescribed in children ICD-9 classification for EoE FDA dru
Slide 32: Current U.S. treatment approaches are limited
CURRENT U.S. TREATMENT APPROACHES ARE LIMITED 31 | Patient Adaptive Behaviors Active Diet Management Esophageal Dilation Off-Label Treatments Homemade “slurry” Swallowing inhaled steroids Compounding pharmacy PPIs PPI: Proton Pump Inhibitor.
Slide 33: Summarizing the opportunity in EoE
SUMMARIZING THE OPPORTUNITY IN EOE • Increasing prevalence and incidence of EoE • Limited access to high quality treatment options • Challenge in consistency in managing disease, but high interest and excitement from HCPs • Low overall patient awareness and education with long diagnosis times • Need
Slide 34: TAK-721 is a potential solution to the unmet need in EoE
TAK-721 is a synthetic second-generation, non- halogenated corticosteroid having potent topical anti-inflammatory and glucocorticoid activity TAK-721 has a wide range of inhibitory activities against multiple cell types Topically active, oral viscous suspension of budesonide, formulated specifically
Slide 35: Pioneering clinical development program of firsts for EoE
34 | Orphan Drug Designation in EoE (2006) Breakthrough Therapy Designation in EoE (2016) NDA submitted on EoE (2020) Comprehensive development program of EoE specific Ph2 & Ph3 studies, aligned with FDA, and largest completed studies specific to EoE To develop and validate with FDA a Patient Report
Slide 36: ORBIT 1 study: co-primary endpoints met
1.0% 53.1% 0 10 20 30 40 50 60 Pr op or ti on o f p at ie nt s ( % ) Placebo (n = 105) 2 mg BID (n = 213) p < 0.001 39.1% 52.6% 0 10 20 30 40 50 60 Pr op or ti on o f p at ie nt s ( % ) Placebo (n = 105) 2 mg BID (n = 213) p = 0.024 Results presented at presidential plenary at ACG, Texas, Oct 2019 3
Slide 37: Key secondary efficacy endpoint met
-7.6 -55.2 -80 -60 -40 -20 0 LS m ea n (S EM ) c ha ng e Placebo (n = 92) 2 mg BID (n = 201) p < 0.001 -9.1 -13.0 -15 -10 -5 0 LS m ea n (S EM ) c ha ng e Placebo (n = 89) 2 mg BID (n=197) p = 0.015 KEY SECONDARY EFFICACY ENDPOINT MET 36 | LS: least squares, SEM: standard error of the mean Change in
Slide 38: Endoscopic appearance (EREFS) endpoint met
ENDOSCOPIC APPEARANCE (EREFS) ENDPOINT MET AT 12 WEEKS Individual patient example from ORBIT 1 study 37 | Change to total EREFS score from baseline to week 12 of therapy -2.2 -4.0 -5 -4 -3 -2 -1 0 LS m ea n (S EM ) c ha ng e Placebo (n = 93) 2 mg BID (n = 202) p < 0.001
Slide 39: TAK-721 was well tolerated
TAK-721 was well tolerated Treatment-emergent adverse events (TEAEs) were similar for TAK-721 (61.0%) and placebo (61.0%) groups after 12 weeks Most TEAEs reported were mild or moderate in severity SAFETY RESULTS CONSISTENT WITH KNOWN SAFETY PROFILE OF BUDESONIDE 38 |
Slide 40: A leadership path to 1st potential U.S. approval
A LEADERSHIP PATH TO 1ST POTENTIAL U.S. APPROVAL NEXT YEAR Orphan Drug Designation granted (2006) Breakthrough Therapy Designation granted (2016) ORBIT 1 Ph3 study (completed 2019) ORBIT 2 Ph3 study (completed 2020) NDA submission (FY2020 Q3) Ph2 studies (Completed 2015) 1st FDA product (if approved
Slide 41: Potential launch as 1st FDA approved product in EoE
TAK-721 1st FDA product (if approved) 40 | 1st line Therapy Later Line TherapyBiologic Treatments Currently in trials TAK-721 POTENTIAL LAUNCH AS 1ST FDA APPROVED PRODUCT IN EOE TO SET STANDARD AS 1ST LINE THERAPY
Slide 42: Broad access for patients to an approved treatment
WE AIM TO ACHIEVE BROAD ACCESS FOR PATIENTS TO AN APPROVED TREATMENT Broad Patient Access 41 | Strong value proposition for payers Many patients currently pay full out-of-pocket for off-label treatments Objective to make TAK-721 accessible to as many patients as possible Services and solutions to en
Slide 43: U.S. business unit well prepared for launching TAK-721 brand
U.S. BUSINESS UNIT WELL PREPARED FOR LAUNCHING TAK-721 BRAND 42 |
Slide 44: U.S. business unit well prepared for launching TAK-721 brand
U.S. BUSINESS UNIT WELL PREPARED FOR LAUNCHING TAK-721 BRAND 43 | HCP PATIENT Field Force • Medical, Thought Leader Liaisons and Sales personnel already hired and established to support EoE launch Disease State Education • New Digital approach to bolster EoE awareness with the launch of SeeEoE.com K
Slide 45: Leadership in GI
44 | Leadership in Inflammatory Bowel Disease (IBD) Leadership in Short Bowel Syndrome (SBS) Leadership in Motility & Acid Related Disorders Leadership in EOE TOGETHER. BEYOND.
Slide 46: Reinforcing leadership in GI with potential first FDA approved therapy in EoE
REINFORCING LEADERSHIP IN GI WITH POTENTIAL FIRST FDA APPROVED THERAPY IN EOE 45 | 1 32 Unmet need in EoE is high, leadership needed Eohilia is specifically designed to tackle EoE Takeda is strongly positioned to launch Eohilia TAK-721 is an investigational treatment and has not been approved for us
Slide 47: Agenda
AGENDA TIME (EST) TIME (JST) AGENDA 17:00 – 17:10 07:00 – 07:10 Takeda: A Global Values-Based, R&D-Driven Biopharmaceutical Leader Christophe Weber, President & CEO 17:10 – 17:25 07:10 – 07:25 Following Through On Our Commitment To Deliver Innovative Medicines To Patients: Spotlight On Select Wave 1
Slide 48: TAK-003 Deep Dive
TAK-003 Deep Dive: Live-Attenuated Tetravalent Vaccine For Prevention Of Dengue Disease Rajeev Venkayya President, Global Vaccine Business Unit
Slide 49: Dengue is a top ten threat to global health
48 | 1. World Health Organization (WHO). Top ten threats to global health in 2019. https://www.who.int/news- room/spotlight/ten-threats-to-global-health- in-2019 2. WHO. Dengue and Severe Dengue. https://www.who.int/news-room/fact-sheets/detail/dengue-a nd-severe- dengue DENGUE IS A TOP TEN THREAT T
Slide 50: Fastest-spreading mosquito-borne viral disease
49 | 1. WHO. https://www.who.int/images/default-source/departments/ntd-libra ry/dengue/infographics-and- illustrations/dengue-infographic.png?sfvrsn=ae8ce604_8 2. WHO. Promoting dengue vector surveillance and control https://www.who.int/activities/promoting-dengue- vector-surveillance-and-control 3.
Slide 51: Geographical range of dengue is expanding
50 | GEOGRAPHICAL RANGE OF DENGUE IS EXPANDING More than 90% of dengue cases occur in Middle Income Countries* (MICs)2,3,4 • Brazil: 2 million cases, 20195 • Philippines, ~400,000 cases, 20196 Dengue is found mostly in urban and semi-urban areas in tropical and sub-tropical climates. Globalization,
Slide 52: Societal impact of dengue outbreaks
51 | 1. PLOS. Neglected Tropical Disease. Societal impact of dengue outbreaks: Stakeholder perceptions and related implications. A qualitative study in Brazil, 2015 2. WHO scales up response to worldwide surge in dengue https://www.who.int/news- room/feature-stories/detail/who-scales-up-response-to-
Slide 53: The economic impact of dengue is broad
52 | THE ECONOMIC IMPACT OF DENGUE IS BROAD1–4 Health care expenditures Direct medical and non-medical costs Care-related productivity Indirect costs: time off work & school for patients and their caregivers Vector control, surveillance and communication costs Dengue outbreak control costs Additiona
Slide 54: Large eligible population for dengue vaccination
Larger and more diverse cohorts • Urban and rural dwellers • Children, adolescents, adults • Men and women LARGE ELIGIBLE POPULATION FOR DENGUE VACCINATION Vaccine Eligible Population In Endemic Countries For A Dengue Vaccine Far Exceeds Any Recently Launched Vaccine1 % of Overall Population that is
Slide 55: Plan to maximize access in endemic countries
4 9 14 19 24 29 34 39 44 49 54 59 64Age $1500 $600 $300 $60 Pe r ca pi ta A ve ra ge M on th ly In co m e (U SD ) Po pu la ti on p er ce nt ag e (% ) 3% 59% 26% 10% 2% Mexico example PLAN TO MAXIMIZE ACCESS IN ENDEMIC COUNTRIES Statistical data from: Pew Research Center analysis of data from the Wor
Slide 56: Plan to maximize access in endemic countries
4 9 14 19 24 29 34 39 44 49 54 59 64Age $1500 $600 $300 $60 Pe r ca pi ta A ve ra ge M on th ly In co m e (U SD ) Po pu la ti on p er ce nt ag e (% ) 3% 59% 26% 10% 2% Mexico example PLAN TO MAXIMIZE ACCESS IN ENDEMIC COUNTRIES Statistical data from: Pew Research Center analysis of data from the Wor
Slide 57: Plan to maximize access: national immunization programs
4 9 14 19 24 29 34 39 44 49 54 59 64Age $1500 $600 $300 $60 Expanded National Immunization Programs • Government Financing • Procurement modalities Pe r ca pi ta A ve ra ge M on th ly In co m e (U SD ) Po pu la ti on p er ce nt ag e (% ) 3% 59% 26% 10% 2% Mexico example PLAN TO MAXIMIZE ACCESS IN EN
Slide 58: Plan to maximize access: private sector activation
4 9 14 19 24 29 34 39 44 49 54 59 64Age $1500 $600 $300 $60 Private Sector Activation • Individual Financing options Pe r ca pi ta A ve ra ge M on th ly In co m e (U SD ) Po pu la ti on p er ce nt ag e (% ) 3% 59% 26% 10% 2% Mexico example PLAN TO MAXIMIZE ACCESS IN ENDEMIC COUNTRIES Expanded Nation
Slide 59: Plan to maximize access: access to medicines
4 9 14 19 24 29 34 39 44 49 54 59 64Age $1500 $600 $300 $60 Private Sector Activation • Individual Financing options Access to Medicines • Patient Assistance Programs • Vulnerable populations Pe r ca pi ta A ve ra ge M on th ly In co m e (U SD ) Po pu la ti on p er ce nt ag e (% ) 3% 59% 26% 10% 2%
Slide 60: Utility for travel and non-endemic markets
UTILITY FOR TRAVEL AND NON-ENDEMIC MARKETS Map is illustrative of trips traveled from the US, Canada and Europe, and does not detail specific numbers or timeframes More than 90 million arrivals* from the US, Canada and Europe to dengue endemic countries in 20183 Dengue is a leading cause of fever am
Slide 61: TAK-003 potential to address the global burden of dengue
Based on a live-attenuated dengue serotype 2 virus, which provides the genetic “backbone” for four dengue serotypes represented in the vaccine Designed to stimulate multiple arms of the immune system TAK-003 is being developed by Takeda to protect children and adults against all four virus serotypes
Slide 62: Pivotal Phase 3 trial design
Part 3Part 2 PIVOTAL PHASE 3 TRIAL DESIGN DEN-301: 20,099 children (aged 4–16 years) were randomized 2:1 to receive either TAK-003 or placebo in endemic countries in Latin America and Asia; subjects were balanced across geographies1 Primary endpoint: Overall vaccine efficacy Secondary efficacy endpo
Slide 63: TAK-003 efficacious against dengue illness with strong safety profile
TAK-003 EFFICACIOUS AGAINST DENGUE ILLNESS WITH A STRONG SAFETY PROFILE 80.2% (73.3 to 85.3; p<0.001) overall vaccine efficacy (VE) * across serotypes and serostatus1 90.4% (82.6 to 94.7; p<0.001) VE against hospitalized dengue across serotypes and serostatus2 TAK-003 was generally well tolerated, w
Slide 64: Hospitalizations reduced by approximately 90%
Overall VE 89.2% (82.4, 93.3) Baseline Sero- 87.0% (70.1, 94.3) Baseline Sero+ 90.0% (81.9, 94.5) HOSPITALIZATIONS REDUCED BY APPROXIMATELY 90% Through 24 Months1 63 | Seronegative at baseline: seronegative to all four dengue serotypes; Seropositive at baseline: reciprocal neutralizing antibody tite
Slide 65: Dengue illness reduced by more than 70%
Overall VE 72.7% (67.1 to 77.3) Baseline Sero- 67.0% (53.6, 76.5) Baseline Sero+ 74.8% (68.6, 79.8) DENGUE ILLNESS REDUCED BY MORE THAN 70% Through 24 Months1 64 | Seronegative at baseline: seronegative to all four dengue serotypes; Seropositive at baseline: reciprocal neutralizing antibody titer >=
Slide 66: Efficacy varied by serotype
EFFICACY VARIED BY SEROTYPE WITH GREATEST REDUCTIONS IN DENGUE ILLNESS OBSERVED FOR THE MOST PREVALENT SEROTYPE, DENV-2* Through 24 Months1 69.0% 90.8% 51.4% 50.4% 0 10 20 30 40 50 60 70 80 90 100 DENV-1 DENV-2 DENV-3 DENV-4‡ VE a ga in st V CD % (57.1, 77.5) (85.6, 94.1) (34.0, 64.2) (-19.3, 79.3)
Slide 67: Safety and tolerability of TAK-003
SAFETY AND TOLERABILITY OF TAK-003 Through 24 Months No evidence of disease enhancement in seronegative individuals1,2,3 Slight increase in dengue fever* in the DENV-3 seronegative population at 18 months. Stabilized at 24 months1,3 Strong safety profile up to 24 months after second dose of TAK-003
Slide 68: Strong efficacy and safety profile two years after vaccination
STRONG EFFICACY AND SAFETY PROFILE TWO YEARS AFTER VACCINATION Continued efficacy against dengue, with some waning efficacy seen between year 1 and year 2; evaluating potential for a booster1 Robust reduction of hospitalized dengue1 Similar efficacy regardless of previous dengue exposure1 The observ
Slide 69: Expected milestones
EXPECTED MILESTONES Launch in EU Approval in EU Regulatory filing in US First approvals in 1st Wave endemic countries and US FY21FY20 FY22 FY23 FY24 FY25 FY26 2nd Wave countries: first approvals and launches 3rd Wave countries: approvals and launches (TBD) DEN-301 36-month data readout • 1st Wave: I
Slide 70: Summary
1 32 Takeda building up capacity to fulfill 50+ million doses per year, meeting urgent need for a new dengue vaccine Estimate the dengue vaccine market could reach USD 1.5-2 billion annually by 20291 First regulatory filings in endemic countries (Article 58), Europe and US expected in 2021 SUMMARY 6
Slide 71: Agenda
TIME (EST) TIME (JST) AGENDA 17:00 – 17:10 07:00 – 07:10 Takeda: A Global Values-Based, R&D-Driven Biopharmaceutical Leader Christophe Weber, President & CEO 17:10 – 17:25 07:10 – 07:25 Following Through On Our Commitment To Deliver Innovative Medicines To Patients: Spotlight On Select Wave 1 Progra
Slide 72: Today's Speakers
TODAY’S SPEAKERS 71 | CHRISTOPHE WEBER President & CEO ANDY PLUMP President, Research & Development RAJEEV VENKAYYA President, Global Vaccine Business Unit Available for Q&A COSTA SAROUKOS Chief Financial Officer TERESA BITETTI President, Global Oncology Business Unit RAMONA SEQUEIRA President, USBU
Slide 73: Appendix 1: Wave 1 Pipelines One-Pager Summaries
Appendix 1 Wave 1 Pipelines One-Pager Summaries
Slide 74: Mobocertinib one-pager
MECHANISM EGFR TKI specifically designed for Exon20 insertions PATIENT JOURNEY/ UNMET NEED Patients with EGFR Exon20 insertion mutations have no approved targeted therapy • Approved EGFR TKIs are not designed to treat Exon20 insertions • Current treatment approaches including chemotherapy, approved
Slide 75: Pevonedistat one-pager
MECHANISM NEDD8-activating enzyme (NAE) inhibitor PATIENT JOURNEY/ UNMET NEED Patients with HR-MDS have a poor prognosis, diminished QoL, higher chance of transformation to AML and limited treatment options • Outcomes are poor and, even with current treatment options, mortality rates remain high. Me
Slide 76: TAK-007 one-pager
TAK-007 Potential Transformative “Off-the-shelf” Cell Therapy For Multiple Cancers MECHANISM CD19 CAR-NK cell therapy PATIENT JOURNEY/ UNMET NEED Significant need for an efficacious, off-the-shelf cell therapy with an improved toxicity profile • Patients with CD19 positive B-cell malignancies who ca
Slide 77: Maribavir (TAK-620) one-pager
MARIBAVIR (TAK-620) Potential 1st Approved Treatment In Over 10 Years For Patients With Post-transplant CMV Infection MECHANISM Maribavir is an oral benzimidazole riboside with activity against cytomegalovirus, that blocks nuclear egress of viral capsids through the inhibition of protein kinase UL97
Slide 78: TAK-609 one-pager
TAK-609 Potential To Be The First Product For Cognitive Impairment In Hunter Syndrome In US/EU MECHANISM Recombinant human iduronate-2-sulfatase unique formulated for intrathecal injection directly into the CNS through a surgically implanted port to circumvent the blood brain barrier. PATIENT JOURNE
Slide 79: TAK-611 one-pager
TAK-611 Potential As The Only Therapy To Halt Rapid Progression Of Symptomatic Late Infantile MLD MECHANISM Recombinant human arylsulfatase A (rhASA) unique formulated for intrathecal injection directly into the CNS through a surgically implanted port to circumvent the blood brain barrier PATIENT JO
Slide 80: TAK-755 one-pager
MECHANISM Recombinant ADAMTS-13 enzyme replacement therapy aiming to reduce the abnormally high von Willebrand factor activity seen in TTP PATIENT JOURNEY/ UNMET NEED • Standard of care with on-demand or prophylactic plasma infusions is highly burdensome. Replacement of ADAMTS13 to achieve sufficien
Slide 81: Soticlestat (TAK-935) one-pager
MECHANISM Cholesterol 24-hydroxylase (CH24H) enzyme inhibitor PATIENT JOURNEY/ UNMET NEED Developmental and Epileptic Encephalopathies (DEEs) are highly treatment resistant to multiple antiepileptic drugs, with few FDA-approved therapies • Over 50% of patients suffer from treatment-resistant seizure
Slide 82: Orexin agonist one-pager
MECHANISM Orexin 2 receptor (OX2R) agonist PATIENT JOURNEY/ UNMET NEED Current treatments do not address the underlying orexin deficiency in NT1 patients • Backbone of care is a combination of wake promoting agents/stimulants for excessive daytime sleepiness (EDS), anti-depressants for cataplexy and
Slide 83: TAK-721 one-pager
MECHANISM Viscous budesonide oral suspension for eosinophilic esophagitis (EoE) PATIENT JOURNEY/ UNMET NEED No U.S.-approved medication exists for EOE • EoE often results in dysphagia (difficulty swallowing) and heartburn, and in adolescents often presents with vomiting and GI pain. Symptoms can rep
Slide 84: TAK-003 one-pager
MECHANISM Tetravalent Dengue Vaccine Candidate based on a live-attenuated dengue serotype 2 virus PATIENT JOURNEY/ UNMET NEED Dengue is endemic in more than 100 countries. Each year, dengue is estimated to cause 390 million infections1. • Severe dengue is a leading cause of serious illness and death
Slide 85: Appendix 2: Epidemiology Data
Appendix 2 Epidemiology Data
Slide 86: Overview of epidemiology data
OVERVIEW OF EPIDEMIOLOGY DATA ONCOLOGY GASTROENTEROLOGY (GI) RARE GENETIC & HEMATOLOGY 85 |
Slide 87: Epidemiology: Ninlaro, Alunbrig, Adcetris & Iclusig
EPIDEMIOLOGY – NINLARO, ALUNBRIG, ADCETRIS & ICLUSIG Product Indication Total Global Eligible Population (FY20) Geographies included R/R 2L+ Multiple Myeloma ~105,0001 Japan, US, EU5, China ALK+ NSCLC ~14,000 [+ ~25,000 in China]2 Japan, US, EU5, [China] HL – Front Line, ASCT Consolidation, 2L+; TCL
Slide 88: Overview of epidemiology data
OVERVIEW OF EPIDEMIOLOGY DATA ONCOLOGY GASTROENTEROLOGY (GI) RARE GENETIC & HEMATOLOGY 87 |
Slide 89: Epidemiology: Ulcerative colitis
EPIDEMIOLOGY ULCERATIVE COLITIS (UC) 770K 755K 453K 83K 1,100K 850K 423K 131K 488K 303K 160K 24K 299K 287K 103K 19K ~2,195K ~1,138K ~257K GEM (incl. China) EUCAN US Japan Diagnosed Prevalence Treated Treated Moderate to Severe Patients on biologic therapies ~2,658K 88 | Source: Takeda estimate. Note
Slide 90: Epidemiology: Crohn's disease
804K 788K 473K 193K 888K 741K 461K 216K 262K 160K 103K 50K 53K 52K 23K 14K ~473K ~1,060K ~1,740K EPIDEMIOLOGY CROHN’S DISEASE (CD) GEM (incl. China) EUCAN US Japan Diagnosed Prevalence Treated Treated Moderate to Severe Patients on biologic therapies ~2,007K 89 | Source: Takeda estimate. Note: Numbe
Slide 91: Epidemiology: Complex perianal fistula
EPIDEMIOLOGY COMPLEX PERIANAL FISTULA (CPF) 780K 193K 114K 21K 792K 119K 62K 21K 58K 27K 14K 2K EUCAN US Japan Crohn’s Disease (CD) Patients Alofisel Eligible Refractory CPF Patients ~1,630K CD Patients with Perianal Fistula Complex Perianal Fistula (CPF) Patients 90 | Source: Takeda estimate. Note:
Slide 92: Epidemiology: Short bowel syndrome adult
14K 6K 6K 1K 10K 5K 2K EPIDEMIOLOGY SHORT BOWL SYNDROME (SBS-IF)-ADULT INDICATION ~8K ~1.7K ~11K Ex-US (mainly EUCAN) US Diagnosed Treated ~24K Diagnosed Eligible1 Access to Gattex/Revestive 91 | 1. Revestive/Gattex Eligible patients: excluding SBS caused by malignancies that occurred less than 5 ye
Slide 93: Epidemiology: Short bowel syndrome pediatric
3K 3K 3K 3K 2K 1K EPIDEMIOLOGY SHORT BOWL SYNDROME (SBS-IF)-PEDIATRIC INDICATION Ex-US (mainly EUCAN) US Diagnosed Treated ~6K Diagnosed Eligible1 Access to Gattex/Revestive ~5K ~4K ~0.2K 92 | 1. Revestive/Gattex Eligible patients: excluding SBS caused by malignancies that occurred less than 5 years
Slide 94: Overview of epidemiology data
OVERVIEW OF EPIDEMIOLOGY DATA ONCOLOGY GASTROENTEROLOGY (GI) RARE GENETIC & HEMATOLOGY 93 |
Slide 95: Epidemiology: Hereditary angioedema
EPIDEMIOLOGY HEREDITARY ANGIOEDEMA 64K 21K 18K Prevalence Diagnosed Treated Global 94 | Source: Takeda estimates. Note: Numbers are rounded. 1. ORE HAE: Global overview of Hereditary Angioedema Epidemiology (http://207.154.195.35/) 2. Dx and Tx to be reviewed with the country * Geographies considere
Slide 96: Epidemiology: Hunter's disease
EPIDEMIOLOGY HUNTER’S DISEASE: GLOBAL, INDIA/CHINA NOT INCLUDED 5K 2-3K 2K MPS II Patients Prevalence Diagnosed Treated Global 95 | Source: Takeda estimates. Note: Numbers are rounded.
Slide 97: Epidemiology: Gaucher's disease
EPIDEMIOLOGY GAUCHER’S DISEASE: GLOBAL, CHINA/INDIA NOT INCLUDED Prevalence Diagnosed ~35K prevalent patients ~9K diagnosed patients (26%) ~26K un-diagnosed patients (74%) 96 | Source: Takeda estimates, US Census Bureau, Shire/CRA developed epi rate (2016). Note: Numbers are rounded.
Slide 98: Epidemiology: Fabry's disease
EPIDEMIOLOGY FABRY’S DISEASE: EX-US REGION, CHINA/INDIA NOT INCLUDED Prevalence Diagnosed ~60K prevalent Fabry patients ~13K diagnosed Fabry patients (21%) ~47K un-diagnosed patients (79%) 97 | Source: Takeda estimates, US Census Bureau, Shire/CRA developed blended epi rate (2016): 1.28,500. Note: N
Slide 99: Epidemiology: Chronic hypoparathyroidism
EPIDEMIOLOGY CHRONIC HYPOPARATHYROIDISM US + EUROPE GEM, Japan And China Not Included Prevalent cHypoPT cases ~350k cases of cHypoPT 98 | Sources: Takeda estimates. Note: Numbers are rounded.
Slide 100: Epidemiology: Hemophilia A
EPIDEMIOLOGY HEMOPHILIA A 17K 17K 17K 30K 25K 18K 5K 5K 5K Prevalence Diagnosed Treated 52K *Note: In the graph, prevalence refers to the number of patients with Hemophilia A without inhibitors. Calculated prevalence for US based on occurrence per x population is high as is does not consider high mo
Slide 101: Appendix 3: Clinical Trial Summary
Appendix 3 Clinical Trial Summary
Slide 102: Wave 1 and Wave 2 pipeline overview
LCM1 WAVE 1 WAVE 2 ONCOLOGY ALUNBRIG 1L ALK+ NSCLC mobocertinib 2L NSCLC w/EGFR exon 20 insertion mutation TAK-981 Multiple cancers TAK-169 R/R Multiple myeloma ALUNBRIG 2L ALK+NSCLC H2H with alectinib mobocertinib 1L NSCLC w/EGFR exon 20 insertion mutation TAK-981 Non-Hodgkin’s lymphoma TAK-252 Bis
Slide 103: Overview of clinical trial summary
OVERVIEW OF CLINICAL TRIAL SUMMARY ONCOLOGY RARE GENETIC & HEMATOLOGY NEUROSCIENCE GASTROENTEROLOGY (GI) PLASMA-DERIVED THERAPIES VACCINES 102 |
Slide 104: Alunbrig (brigatinib) trials
ALUNBRIG (BRIGATINIB): ALK INHIBITOR Study NCT02737501 NCT03596866 Indication ALK-positive advanced lung cancer ALK-positive non-small-cell lung cancer (NSCLC) Phase Phase IIIALTA-1L Phase III ALTA-3 # of Patients N = 275 N = 246 Target Patients ALK+ locally advanced or metastatic NSCLC patients who
Slide 105: Iclusig (ponatinib) trials
ICLUSIG (PONATINIB): BCR-ABL INHIBITOR Study NCT02467270 NCT03589326 Indication Chronic myeloid leukemia (CML) Ph+ acute lymphoblastic leukemia (ALL) Phase Phase IIOPTIC Phase III Ph+ALLCON # of Patients N = 276 N = 230 - 320 Target Patients Patients with resistant chronic phase chronic myeloid leuk
Slide 106: Ninlaro (ixazomib) trials
NINLARO (IXAZOMIB): ORAL PROTEASOME INHIBITOR Study NCT02181413 NCT02312258 Indication Multiple myeloma (MM) maintenance post-stem cell transplant Multiple myeloma (MM) maintenance non-stem cell transplant Phase Phase IIITOURMALINE-MM3 Phase III TOURMALINE-MM4 # of Patients N = 652 N = 761 Target Pa
Slide 107: Ninlaro (ixazomib) MM6 trial
NINLARO (IXAZOMIB): ORAL PROTEASOME INHIBITOR Study NCT03173092 Indication Non-transplant eligible patients with newly diagnosed multiple myeloma Phase Phase IVMM6 # of Patients N = 160 Target Patients Patients with multiple myeloma previously receiving a bortezomib-based induction. In-class (protea
Slide 108: Mobocertinib (TAK-788) trials
MOBOCERTINIB (TAK-788): EGFR/HER2 EXON 20 INHIBITOR Study NCT02716116 NCT04129502 Indication 2L NSCLC exon 20 insertion mutation 1L NSCLC exon 20 insertion mutation Phase Registration enabling Phase IIEXCLAIM Phase III EXCLAIM-2 # of Patients N = 341 N = 318 Target Patients 2L+ NSCLC harboring EGFR
Slide 109: Pevonedistat (TAK-924) trials
PEVONEDISTAT (TAK-924): NEDD8-ACTIVATING ENZYME (NAE) INHIBITOR Study NCT03268954 NCT04090736 Indication HR MDS Unfit AML Phase Phase IIIPANTHER Phase III PEVOLAM # of Patients N = 450 N = 466 Target Patients Patients with higher risk myelodysplastic syndromes (HR MDS), chronic myelomonocytic leukem
Slide 110: TAK-007: CD19 CAR NK
TAK-007: CD19 CAR NK Study NCT030563391 Indication Relapsed refractory B-lymphoid malignancies Phase Phase I # of Patients N = 36 Target Patients Patients with relapsed and refractory CD19+ B lymphoid malignances Arms/Intervention • Fludarabine 30 mg/m2 by vein on days -5 to -3 • Cyclophosphamide 30
Slide 111: TAK-981 trials
TAK-981: SUMO-ACTIVATING ENZYME1 INHIBITOR Study NCT03648372 NCT04074330 Indication Solid tumors, hematologic malignancies Non-Hodgkin’s lymphoma (NHL) Phase Phase I Phase I/II # of Patients N = 80 N = 130 Target Patients Adult participants with advanced or metastatic solid tumors or relapsed/refrac
Slide 112: TAK-981 solid tumors trial
TAK-981: SUMO-ACTIVATING ENZYME1 INHIBITOR Study NCT04381650 Indication Solid tumors Phase Phase Ib/II # of Patients N = 101 Target Patients Patients with select advanced or metastatic solid tumors Arms/Intervention • Escalating doses of TAK-981 with starting dose of 40 mg, intravenous (IV) infusion
Slide 113: TAK-605 oncolytic virus
TAK-605: ONCOLYTIC VIRUS ENCODING TRANSGENES FOR FLT3 LIGAND, ANTI-CTLA-4 ANTIBODY, AND IL-12 CYTOKINE Study NCT043010111 Indication Solid tumors Phase Phase I/IIa # of Patients N = 84 Target Patients Patients with advanced solid tumors Arms/Intervention • Arm A: TBio-6517 (TAK-605) dose escalation
Slide 114: TAK-573 trials
TAK-573: FIRST-IN-CLASS ANTI-CD38/ATTENUATED IFNα FUSION PROTEIN Study NCT04157517 NCT03215030 Indication Solid tumors Relapsed/refractory multiple myeloma Phase Phase I Phase I/2 # of Patients N = 86 N = 151 Target Patients Patients with locally advanced or metastatic solid tumors Patients with rel
Slide 115: TAK-676: STING agonist
TAK-676: STING AGONIST Study NCT04420884 Indication Solid tumors Phase Phase I # of Patients N = 76 Target Patients Adult patients with advanced or metastatic solid tumors Arms/Intervention • Arm 1: Dose escalating single agent TAK-676, starting with a safety lead-in at 0.1 mg IV on Days 1, 8, 15 in
Slide 116: TAK-169 trial
TAK-169: ANTI-CD38 ANTIBODY-SLTA1 TOXIN Study NCT04017130 Indication Relapsed or refractory multiple myeloma Phase Phase I # of Patients N = 102 Target Patients Patients with relapsed or refractory multiple myeloma Arms/Intervention • Dose escalation arms: TAK-169 50 mcg/kg Once Weekly; TAK-169 100
Slide 117: TAK-252 trial
TAK-252: PD1-FC OX40L ARC Study NCT038946181 Indication Advanced solid tumors or lymphomas Phase Phase I # of Patients N = 87 Target Patients Patients with advanced solid tumors or lymphomas Arms/Intervention • TAK-252 (SL-279252) is a first-in-class agonist redirected checkpoint (ARC) fusion protei
Slide 118: TAK-102: GPC3 CAR-T
TAK-102: GPC3 CAR-T Study NCT044057781 Indication Solid tumors Phase Phase I # of Patients N = 18 Target Patients Adult patients with GPC3-expressing previously treated solid tumors Arms/Intervention • Cohort 1: 1 × 10^7 CAR (+) cells/body [starting dose] • Cohort 2: 1 × 10^8 CAR (+) cells/body • Co
Slide 119: TAK-940: CD19 CAR-T
TAK-940: CD19 CAR-T Study NCT044642001 Indication Relapsed/refractory B-cell cancers Phase Phase I # of Patients N = 30 Target Patients Adult patients with relapsed or refractory B-dell malignancies Arms/Intervention • 19(T2)28z1xx CAR T cells Cohorts of 3-6 patients will be infused with escalating
Slide 120: Overview of clinical trial summary
OVERVIEW OF CLINICAL TRIAL SUMMARY RARE GENETIC & HEMATOLOGY NEUROSCIENCE GASTROENTEROLOGY (GI) ONCOLOGY PLASMA-DERIVED THERAPIES VACCINES 119 |
Slide 121: Adynovate trial in hemophilia A
Study NCT02615691 Indication Hemophilia A Phase Phase III # of Patients N = 120 Target Patients Previously untreated patients (PUPs) < 6 years with severe hemophilia A (FVIII < 1%) Arms/Intervention • Single group assignment Primary endpoint and key secondary endpoint(s) The primary objective is to
Slide 122: Vonvendi (TAK-577) trials
VONVENDI (TAK-577): RECOMBINANT VON WILLEBRAND FACTOR Study NCT02973087 NCT02932618 Indication Adult Prophylaxis Pediatric On-demand and Elective Surgery Phase Phase III Phase III # of Patients N = 22 N = 27 (On-demand)N = 12 (Elective Surgery) Target Patients Severe von Willebrand Disease Severe vo
Slide 123: Takhzyro (lanadelumab) trials
TAKHZYRO (LANADELUMAB): PLASMA KALLIKREIN (PKAL) INHIBITOR Study NCT04070326 NCT04206605 Indication Hereditary angioedema (HAE) pediatric Non-histaminergic angioedemawith normal C1-Inhibitor Phase Phase IIISPRING Phase III CASPIAN # of Patients N = 20 N = 75 Target Patients Type I and Type II heredi
Slide 124: Maribavir (TAK-620) trials
MARIBAVIR (TAK-620): ORAL VIRAL PROTEIN KINASE INHIBITOR Study NCT02931539 NCT02927067 Indication Treatment of Resistant/Refractory Post-Transplant Cytomegalovirus (CMV) Infection Treatment of CMV infection in Hematopoietic Stem Cell Transplant Recipients Phase Phase III Phase III # of Patients N =
Slide 125: TAK-755 trials
TAK-755: REPLACEMENT OF THE DEFICIENT-ADAMTS13 ENZYME Study NCT03393975 NCT03922308 NCT03997760 Indication Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Immune Thrombotic Thrombocytopenic Purpura (iTTP) Sickle Cell Disease Phase Phase III Phase II Phase I # of Patients N = 68 N = 30 N = 56 T
Slide 126: Metachromatic leukodystrophy trials
Study NCT01887938 NCT03771898 Indication Treatment of patients with motor symptoms in Metachromatic Leukodystrophy (MLD) Treatment of patients with motor symptoms in Metachromatic Leukodystrophy (MLD) Phase Phase I/II Extension Trial (Of HGT-MLD-070) Registration Enabling Phase IIb # of Patients N =
Slide 127: Hunter syndrome trials
Study NCT01506141 NCT02412787 Indication Hunter Syndrome with Cognitive Impairment Hunter Syndrome with Cognitive Impairment Phase Phase I/IIHGT-HIT-045 Phase II/III HGT-HIT-094 # of Patients N = 14 N = 56 Target Patients Pediatric participants that completed HGT-HIT-045 with Hunter syndrome and cog
Slide 128: Mezagitamab (TAK-079) trials
MEZAGITAMAB (TAK-079): ANTI-CD38 ANTIBODY Study NCT04278924 NCT04159805 Indication Persistent/Chronic PrimaryImmune Thrombocytopenia (ITP) Myasthenia Gravis Phase Phase II Phase II # of Patients N = 54 N = 36 Target Patients Patients ≥18 years of age with persistent/chronic primary ITP Patients ≥18
Slide 129: SHP607 trial in premature infants
Study NCT03253263 Indication Disease Complications of Extremely Premature Infants Phase Phase IIb # of Patients N = 477 Target Patients Extremely premature infants (birth>23 weeks to < 28 weeks of gestational age) Arms/Intervention 3 Arms 1:1:1 Ratio • ~159 subjects randomized to continuous IV infus
Slide 130: Obizur (TAK-672) trial
Study NCT02895945 Indication Congenital Hemophilia A with Inhibitors (CHAWI) patients who are undergoing major or minor elective surgical, dental, or other invasive procedures Phase Phase III # of Patients N = 12 Target Patients CHAWI patients Arms/Intervention • Single arm study with individualized
Slide 131: Overview of clinical trial summary
OVERVIEW OF CLINICAL TRIAL SUMMARY ONCOLOGY RARE GENETIC & HEMATOLOGY NEUROSCIENCE GASTROENTEROLOGY (GI) PLASMA-DERIVED THERAPIES VACCINES 130 |
Slide 132: TAK-994: Orexin 2R agonist, oral
TAK-994: OREXIN 2R AGONIST, ORAL Study NCT04096560 NCT04551079 Indication Narcolepsy with or without cataplexy (NT1 or NT2) Acute sleep phase delay paradigm in healthy male participants Phase Phase IISPARKLE-1501 Phase I # of Patients N = up to 202 N = 18 Target Patients Patients with Narcolepsy Typ
Slide 133: TAK-925: Orexin 2R agonist, IV
TAK-925: OREXIN 2R AGONIST, IV Study NCT03332784 NCT03748979 Indication Narcolepsy type 1 Narcolepsy type 1 and Narcolepsy type 2 Phase Phase I Phase I # of Patients N = 58 N = 57 Target Patients Patients with narcolepsy type 1 andhealthy volunteers Patients with narcolepsy type 1, patients with nar
Slide 134: TAK-925 trials in OSA and idiopathic hypersomnia
TAK-925: OREXIN 2R AGONIST, IV Study NCT04091425 NCT04091438 Indication Excessive Daytime sleepiness in subjects with Obstructive Sleep Apnea Idiopathic Hypersomnia Phase Phase 1 Phase 1 # of Patients N = 25 N = 40 Target Patients Patients with obstructive sleep apnea who are experiencing excessive
Slide 135: Soticlestat (TAK-935) ELEKTRA trial
SOTICLESTAT (TAK-935): CH24H INHIBITOR, ORAL Study NCT036504521 Indication Dravet Syndrome (DS) and Lennox–Gastaut syndrome (LGS) Phase Phase IIELEKTRA # of Patients N = 141 Target Patients Pediatric patients between the ages of 2 and < 18 years of age with the diagnosis of DS or LGS demonstrating ≥
Slide 136: WVE-120101/120102: MHTT ASO
WVE-120101/120102: MHTT ASO Study NCT032258331 NCT032258461 Indication Huntington’s Disease Huntington’s Disease Phase Phase I/IIPRECISION-HD1 Phase I/II PRECISION-HD2 # of Patients N = 60 N = 60 Target Patients Adult patients with early manifest Huntington's disease (HD) who carry a targeted single
Slide 137: TAK-341: Alpha-synuclein antibody
TAK-3411: ALPHA-SYNUCLEIN ANTIBODY, IV Study NCT03272165 NCT04449484 Indication Parkinson’s Disease Parkinson’s Disease Phase Phase I Phase I # of Patients N = 48 N = 36 Target Patients Healthy volunteers Patients with Parkinson’s Disease Arms/Intervention • TAK-341 (MEDI1341) IV at a single ascendi
Slide 138: Overview of clinical trial summary
OVERVIEW OF CLINICAL TRIAL SUMMARY ONCOLOGY RARE GENETIC & HEMATOLOGY GASTROENTEROLOGY (GI) NEUROSCIENCE PLASMA-DERIVED THERAPIES VACCINES 137 |
Slide 139: Entyvio (vedolizumab) trials
ENTYVIO (VEDOLIZUMAB): GUT-SELECTIVE ANTI-α4β7 INTEGRIN MAB Study NCT03657160 NCT02620046 Indication Graft-versus-Host Disease (GvHD) prophylaxis IV Ulcerative Colitis (UC) or Crohn’s disease (CD) subcutaneous (SC) Phase Phase III Phase III # of Patients N = 558 N = 692 Target Patients Patients unde
Slide 140: Entyvio pediatric trial
ENTYVIO (VEDOLIZUMAB): GUT-SELECTIVE ANTI-α4β7 INTEGRIN MAB Study NCT03196427 Indication Ulcerative Colitis or Crohn’s disease in pediatric patients IV Phase Phase II(Long-term safety study) # of Patients N = 90 Target Patients Pediatric patients with Ulcerative Colitis or Crohn’s disease between 2
Slide 141: ADMIRE-CD II trial
Study NCT03279081 Indication Complex perianal fistula(s) in patients with Crohn’s disease Phase Phase IIIADMIRE-CD II # of Patients N = 554 Target Patients Patients with Crohn’s disease who have complex perianal fistula(s), previously treated and have shown an inadequate response to immunosuppressan
Slide 142: Vonoprazan trial in China
Study NCT04198363 Indication Acid related disease (adjunct to Helicobacter pylori eradication) Phase Phase III China # of Patients N = 510 Target Patients Helicobacter pylori (HP)-positive participants who require HP eradication Arms/Intervention • Experimental: Vonoprazan 20 mg in combination with
Slide 143: TAK-721 extension study
Study NCT03245840 Indication Eosinophilic Esophagitis (EoE) Phase Phase III # of Patients N = 133 Target Patients Subjects with EoE who have completed participation in both the SHP621-301 and SHP621-302 studies – extension study Arms/Intervention Open Label Study:• Budesonide oral suspension (BOS) (
Slide 144: TAK-951: Peptide agonist, SC
TAK-951: PEPTIDE AGONIST, SC Study NCT04486950 NCT04557189 Indication Nausea & Vomiting Nausea & Vomiting Phase Phase I Phase IIa # of Patients N = 40 N = 100 Target Patients Healthy participants Surgical patients under general anesthesia with 3 or more Apfel risk factors Arms/Intervention • Cohort
Slide 145: TAK-906 gastroparesis trial
Study NCT03544229 Indication Gastroparesis Phase Phase II # of Patients N = 205 Target Patients Patients who have symptomatic idiopathic or diabetic gastroparesis. Arms/Intervention • TAK-906 5 mg capsule BID: approximately 25 subjects prior to discontinuation of randomization into this dose arm • T
Slide 146: TAK-954 post-operative GI dysfunction trial
Study NCT03827655 Indication Post-Operative Gastrointestinal Dysfunction (POGD) Phase Phase II # of Patients N = 180 Target Patients Participant is scheduled to undergo a laparoscopic-assisted or open partial small- or large-bowel resection. Arms/Intervention • Regimen 1: Placebo (NS 100 mL infusion
Slide 147: Sibofimloc (TAK-018) trial
Study NCT03943446 Indication Prevention of the Recurrence of Postoperative Crohn's Disease (CD) Phase Phase II # of Patients N = 96 Target Patients Documented diagnosis of CD confirmed by endoscopic biopsy before resection or by tissue obtained at resection. Arms/Intervention • Cohort 1: TAK-018 0.3
Slide 148: Overview of clinical trial summary
OVERVIEW OF CLINICAL TRIAL SUMMARY ONCOLOGY RARE GENETIC & HEMATOLOGY PLASMA-DERIVED THERAPIES NEUROSCIENCE GASTROENTEROLOGY (GI) VACCINES 147 |
Slide 149: HyQvia (TAK-771) CIDP trials
HYQVIA (TAK-771): IVIG WITH RECOMBINANT HUMAN HYALURONIDASE, SC Study NCT02549170 NCT02955355 Indication Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Phase Phase III Phase III # of Patients N = 174 N = 120 Target Pa
Slide 150: HyQvia PIDD pediatric trials
HYQVIA (TAK-771): IVIG WITH RECOMBINANT HUMAN HYALURONIDASE, SC Study NCT03277313 NCT03116347 Indication Primary Immunodeficiency Diseases (PIDD) Primary Immunodeficiency Diseases (PIDD) Phase Phase III Phase IV # of Patients N = 44 N = 42 Target Patients Pediatric subjects with primary immunodefici
Slide 151: Glassia A1PI trial
Study NCT02525861 Indication Chronic augmentation and maintenance therapy in adults with clinically evident emphysema due to severe congenital deficiency of Alpha1-Proteinase Inhibitor (A1PI) Phase Phase III/IV # of Patients N = 36 Target Patients A1PI deficient subjects Arms/Intervention • Arm 1: G
Slide 152: CoVIg-19 trial in COVID-19
Study NCT045465811 Indication Treatment of COVID-19 in hospitalized patients with moderate disease Phase Phase III # of Patients N = 500 Target Patients Adult hospitalized COVID-19 patients with moderate disease with duration of symptoms ≤ 12 days Arms/Intervention • Arm A: Hyperimmune globulin to S
Slide 153: Overview of clinical trial summary
OVERVIEW OF CLINICAL TRIAL SUMMARY ONCOLOGY RARE GENETIC & HEMATOLOGY VACCINESNEUROSCIENCE GASTROENTEROLOGY (GI) PLASMA-DERIVED THERAPIES 152 |
Slide 154: TAK-003 TIDES Phase 3 trial
Study NCT02747927 Indication The prevention of dengue disease caused by any dengue virus serotype in individuals 4 years to 60 years of age Phase Phase IIITetravalent Immunization against Dengue Efficacy Study (TIDES) # of Patients N = 20,100 Target Patients Healthy children aged 4 to 16-year-old i
Slide 155: TAK-214: Norovirus vaccine
TAK-214: NOROVIRUS GI.1/GII.4 BIVALENT VIRUS-LIKE PARTICLE VACCINE Study NCT02669121 NCT03039790 Indication For active immunization for the prevention of acute gastroenteritis caused by norovirus (NoV) For active immunization for the prevention of acute gastroenteritis caused by norovirus (NoV) Phas
Slide 156: Zika vaccine Phase 1 trial
Study NCT03343626 Indication For active immunization for prevention of disease caused by Zika virus (ZIKV) Phase Phase I # of Patients N = 271 Target Patients Healthy Adult Participants aged 18-49-years of age Arms/Intervention • Placebo: TAK-426 placebo-matching injection, intramuscular, once on Da
Slide 157: Appendix: Reconciliation Tables, Glossary
Appendix Reconciliation Tables, Glossary
Slide 158: Underlying Growth definition
Takeda uses the concept of Underlying Growth for internal planning and performance evaluation purposes. Underlying Growth compares two periods (fiscal quarters or years) of financial results under a common basis and is used by management to assess the business. These financial results are calculated
Slide 159: Reconciliation from reported to core/underlying core FY2014
Billion yen FY2013 FY2014 Growth Revenue 1,691.7 1,778.8 +5.1% Fx effects 6.0 (40.0) Divestments (22.1) (16.0) Underlying Revenue 1,675.7 1,721.9 Underlying Growth +2.8% Operating Profit 139.3 -129.3 — Actos one off 274.1 Amortization of intangibles 119.7 123.8 Impairment of intangibles 23.1 63.5 Di
Slide 160: Reported to core to underlying core reconciliation
(BN JPY) REPORTED REPORTED TO CORE ADJUSTMENTS CORE CORE TO UNDERLYING CORE ADJ. UNDERLYING CORE Amortization & impairment of intangible assets Other operating income/ expense Shire acquisition related costs Shire purchase accounting adjustments Swiss Tax Reform Teva JV related accounting adjustment
Slide 161: Glossary of abbreviations
GLOSSARY OF ABBREVIATIONS 160 | PBS phosphate buffered saline PCAB potassium competitive acid blocker Ph+ ALL Philadelphia chromosome-positive acute lymphoblastic leukemia PID primary immunodeficiency PK pharmacokinetics POC proof of concept POGD post-operative gastrointestinal dysfunction POI post-
Slide 162: Copyright notice
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