LONG-TERM VALUE FOR
PATIENTS, SOCIETY AND
INVESTORS
15
SCIENCE
DRIVEN
COMPANY
WITH A
FOCUSED
MIND
BRINGING
INNOVATION
TO PATIENTS
Positioned for Sustainable Revenue Growth
Potential Wave 2 pipeline not included
WAVE 1 Vyvanse
PIPELINE*1 Velcade WAVE 1 Entyvio
Azilva PIPELINE
Hemophilia Others
14
Others GLOBAL
14 BRANDS
GLOBAL
BRANDS
2018 2024 2029
PRO-FORMA
REVENUE
Note: The above chart represents conceptual changes in revenue through 2024 and 2029 demonstrating growth over time offsetting loss of exclusivities and achieving a single digit growth as compared to 2018 pro forma revenue
which represents the sum of Takeda revenue for FY2018 plus Shire revenue for the same period (not including the Legacy Shire oncology business, which was sold in August 2018), converted to JPY at the rate of $1 = 111 JPY, and
converted from US GAAP to IFRS. Actual future net sales achieved by our commercialized products and pipelines will be different, perhaps materially so, as there is a range of possible outcomes from clinical development, driven by a
number of variables, including safety, efficacy and product labelling. In addition, if a product is approved, the effect of commercial factors including the patient population, the competitive environment, pricing and reimbursement is 17
also uncertain. Sales estimate in Wave 1 Pipeline is non-risk adjusted.
R&D DAY AGENDA – NEW YORK, NOVEMBER 14, 2019
TIME AGENDA
Welcome and Opening Remarks
12:30 – 12:35
Sheelagh Cawley-Knopf, Head R&D Global Portfolio Strategy
Takeda: A Global Values-Based, R&D-Driven Biopharmaceutical Leader
12:35 – 12:45
Christophe Weber, President & CEO Takeda
Translating Science into Highly Innovative, Life-changing Medicines
12:45 – 13:20
Andy Plump, President R&D
Oncology and Cell Therapies with Spotlight on CAR-NK
13:20 – 13:45
Chris Arendt, Head Oncology Drug Discovery Unit
Spotlight on Oncology Opportunities
13:45 – 14:05 • TAK-788 : Rachael Brake, Global Program Lead
• Pevonedistat : Phil Rowlands, Head Oncology Therapeutic Area Unit
14:05 – 14:20 Break
Rare Diseases & Gene Therapy
14:20 – 14:45
Dan Curran, Head Rare Disease Therapeutic Area Unit
Spotlight on Orexin2R agonists
14:45 – 15:00
Deborah Hartman, Global Program Lead
Therapeutic Area Focus in GI with Spotlight on Celiac Disease
15:00 – 15:20
Asit Parikh, Head GI Therapeutic Area Unit
15:20 – 16:00 Panel Q&A Session
16:00 Drinks reception
19
TRANSLATING SCIENCE INTO HIGHLY INNOVATIVE
LIFE-CHANGING MEDICINES
Andy Plump MD, PhD
President R&D
Takeda Pharmaceutical Company Limited
New York, NY
November 14, 2019
WHAT YOU WILL HEAR TODAY
1 2 3
Our portfolio We are investing in We have cultivated
and pipeline will novel mechanisms an environment of
drive growth and and capabilities for empowerment,
offset key patent a sustainable accountability and
expirations future agility
21
WE ARE POSITIONED TO DELIVER NEAR-TERM & SUSTAINED GROWTH
WAVE 11 WAVE 22
TARGET CLINICAL-STAGE NMEs PLATFORMS
APPROVAL FY20 FY21 FY22 FY23 FY24 FY25 AND BEYOND
TAK-7883 TAK-007 TAK-924 TAK-164 TAK-252
2L NSCLC Hematologic AML GI malignancies Solid tumors TARGETED
malignancies CELL THERAPY INNATE NEXT-GEN
ONCOLOGY AND IMMUNE
ENGAGERS IMMUNE
CHECKPOINT
TAK-9243 TAK-788 TAK-573 TAK-981 MODULATION MODULATORS
HR-MDS 1L NSCLC R/R MM Multiple cancers
TAK-620 TAK-611 TAK-607 TAK-0794 TAK-754 TAK-755
CMV infect. in MLD (IT) Complications of MG, ITP HemA iTTP, SCD
Immunology
RARE Hematology
transplant prematurity GENE
THERAPY
DISEASES Metabolic
TAK-609 TAK-755 TAK-531
Hunter CNS (IT) cTTP Hunter CNS
TAK-935 Orexin2R-ag TAK-341 Orexin2R-ag TAK-041
DEE (TAK-925/994) Parkinson’s Sleep Disorders CIAS NS OTHER
Narcolepsy T1 Disease GENE PLATFORMS
THERAPY RNA Modulation
NEUROSCIENCE TAK-418 TAK-653 TAK-831 Antibody Transport
Vehicle
Kabuki Syndrome TRD CIAS NS
WVE-120101 WVE-120102
Huntington’s Huntington’s
Disease Disease
TAK-721 Kuma062 TAK-101 TAK-018 TAK-671
EoE Celiac Disease Celiac Disease Crohn’s Disease Acute
GASTRO- (post-op and ileitis) Pancreatitis GENE
MICROBIOME
CELL
THERAPY THERAPY
ENTEROLOGY TAK-954 TAK-906 TAK-951
POGD Gastroparesis Nausea &
vomiting
TAK-003 TAK-214 TAK-426 TAK-021
VACCINES Dengue Vaccine Norovirus Zika Vaccine EV71 vaccine
Vaccine
1. Projected timing of approvals depending on data read-outs; some of these Wave 1 target approval dates assume accelerated approval Orphan potential in at least one indication
2. Some Wave 2 assets could be accelerated into Wave 1 if they have breakthrough data 22
Estimated dates as of November 14, 2019
3. Projected approval date assumes filing on Phase 2 data
4. TAK-079 to be developed in Rare Diseases indications myasthenia gravis (MG) and immune thrombocytopenic purpura (ITP) (FPI projected in each indication in 2H FY19)
2019: A WATERSHED YEAR FOR TAKEDA
INTEGRATION OF SHIRE EXPANSION OF OUR GLOBAL BRANDS UNPRECEDENTED NMEs
• 18 assets added to the clinical pipeline* • VARSITY study demonstrated head-to-head • 17 NMEs in Phase 2 and Phase 3
superiority of Entyvio vs Humira and published
• Creation of a Rare Diseases Therapeutic Area in New England Journal of Medicine • Potentially curative novel mechanisms (e.g.
TAK-101, Orexin2R-ag, CAR-NK)
• Access to world-class Gene Therapy • TAKHZYRO indication expansions in bradykinin
capabilities mediated angioedema • Momentum in Cell Therapies, including new
partnership with MD Anderson
• Expecting >15 approvals in China over the next
5 years
23
* Including approved products with ongoing R&D investment
PATIENT-DRIVEN AND SCIENCE-FIRST IN 3 CORE AREAS
INNOVATIVE BIOPHARMA
ONCOLOGY RARE DISEASES NEUROSCIENCE GASTROENTEROLOGY
PLASMA DERIVED THERAPIES VACCINES BUSINESS UNIT
Complementing our Differentiated
rare disease focus Dengue vaccine
24
WE ARE DOING MORE FOR OUR PATIENTS
8
POTENTIAL BIC/FIC NMEs IN
~40
NEW MOLECULAR
~4,500
R&D EMPLOYEES
PIVOTAL STUDIES1 ENTITY CLINICAL GLOBALLY
STAGE ASSETS
DIVERSIFIED
MODALITIES
IN RESEARCH
~70% ~50%
PIPELINE WITH
ORPHAN DRUG
DESIGNATION2
200+
ACTIVE PARTNERSHIPS
1. BIC/FIC Best-In-Class/First-In-Class (incl. relugolix). Three NMEs in pivotal studies in 2018 25
2. 31 Orphan Drug Designations in at least one indication for assets in Phase 1 through LCM in 2019 versus 15 in 2018
WE ARE TAKING COURAGEOUS RISKS TO MAKE A CRITICAL DIFFERENCE
5
“There is a considerable need
for improved treatments for Accelerated programs
Cell Tx
individuals with NT1, which is Gene Tx
20
caused by the loss of orexin- Biologics
producing neurons in the brain” Peptides
~70% NME stage-ups since FY18
Dr. Makoto Honda, Sleep Oligonucleotide
19
Disorders Project Leader,
Tokyo Metropolitan
Microbiome Indications terminated or
Institute of Medical Science
Small Molecule externalized since FY18
Data presented at World Sleep conference
NOVEL TARGET MODALITY FAST GO / NO-GO
MECHANISMS WITH DIVERSIFICATION DECISION MAKING
HUMAN VALIDATION
26
WE ARE CULTIVATING THE BEST SCIENCE THROUGH
DIFFERENTIATED PARTNERSHIPS…
Select partnerships*
Access to Innovation
RESEARCH IN-LICENSE Risk-Sharing
COLLABORATIONS
~ 110 ~ 50
Expanding Capacity
JOINT NEWCO
DEVELOPMENT FORMATION
~ 20 ~ 20
Total Value in Public & Private Equity
>$1B
* Externalizations and venture investments are not included
27
WE ARE NURTURING INNOVATION WHEREVER IT OCCURS
CHARACTERISTICS TAKEDA PARTNER-SOURCED
TAKEDA GREATER VALIDATION TAK-925, TAK-994 Narcolepsy TAK-573 Multiple Myeloma
DEVELOPS & AND / OR LOWER
COMMERCIALIZES DEVELOPMENT COST TAK-951 Vomiting Syndromes CD19 1XX (CAR-T)
TAK-924 Myelodysplastic Syndrome Kuma-062 Celiac
TAKEDA/PARTNER UNCERTAIN SCIENCE Psychiatry Assets Alzheimer Disease
SHARE DEVELOPMENT AND / OR HIGH
& COMMERCIALIZATION DEVELOPMENT COST
28
Representative examples only
TO DRIVE HIGHER RETURN ON OUR $4.5B ANNUAL R&D INVESTMENT
PRIORITIZED R&D PORTFOLIO FLEXIBLE R&D FUNDING MODEL
BALANCED SPEND TARGETED POPULATIONS PARTNERSHIP MODEL
Minimize internal spend and Smaller trials, lower costs, potential Success driven milestone
infrastructure longer exclusivity payments
29
A RESEARCH ENGINE FUELING A SUSTAINABLE PIPELINE
POTENTIAL NME PIVOTAL STUDY STARTS BY YEAR IMPROVED PRODUCTIVITY
• Research momentum building
Projected
with a projected ~18 portfolio
Clinical Replenishment
Research entries in FY19
Pipeline Engine
• Productivity likely to increase with
11
expansion of cell and gene
8 therapy capabilities
6
4 4 • Leveraging partnerships to access
2
the best clinical or preclinical
FY 2019 2020 2021 2022 2023 2024 innovation
Note: Projections assume successful data readouts
30
PIPELINE INVESTMENTS SUPPORTING NEAR-TERM GROWTH
WAVE 1
INNOVATIVE EXPANSIONS
NEW MOLECULAR ENTITIES
31
WE ARE DRIVING EXPANSION OF OUR GLOBAL BRANDS
SELECT GLOBAL GROWTH BRANDS
TAU Therapies New Indications / Geographic Expansions Target (FY)
Alunbrig 1L Non Small Cell Lung Cancer 2020
ONC Ninlaro ND MM Maintenance (non-SCT and post-SCT) 2020 / 2022
Entyvio Bradykinin Mediated Angioedema 2024
Rare * Prophylactic Treatment of von Willebrand Disease 2021
Alofisel Ulcerative Colitis, Crohn’s Disease (subcutaneous formulation) 2019 / 2020
Takhzyro Graft versus Host Disease (prophylaxis) 2022
GI
Complex Perianal Fistulas 2021
SELECT REGIONAL EXPANSIONS
Region Therapies Region Therapies
relugolix, cabozantinib,
China Japan niraparib
32
ND MM: newly diagnosed multiple myeloma * VONVENDI is emerging as a global brand
SCT: stem cell transplant Estimated dates as of November 14, 2019
WAVE 1 NEW MOLECULAR ENTITIES HAVE POTENTIAL
TO DELIVER >$10B AGGREGATE PEAK SALES…
TARGET APPROVAL1 FY20 FY21 FY22 FY23 FY24
TAK-7882 TAK-007 TAK-924
Hematologic
2L NSCLC malignancies AML
ONCOLOGY
TAK-9242 TAK-788
HR-MDS 1L NSCLC
14 potential NME
TAK-620 TAK-611 TAK-607
launches which
represent
CMV infect. in MLD (IT) Complications of
Immunology transplant prematurity
RARE Hematology
DISEASES Metabolic
TAK-609
Hunter CNS (IT)
TAK-755
cTTP
best-in-class
or
NEUROSCIENCE TAK-935
DEE
Orexin2R-ag
(TAK-925/994)
first-in-class
therapies
Narcolepsy T1
GASTRO- TAK-721 to advance patient
standard of care
ENTEROLOGY EoE
VACCINES TAK-003
Dengue Vaccine
Peak sale estimate of >$10B is non-risk adjusted Orphan potential in at least one indication 33
1. Projected timing of approvals depending on data read-outs; some of these Wave 1 target approval dates assume accelerated approval
2. Projected approval date assumes filing on Phase 2 data Estimated dates as of November 14, 2019
…AND ARE EXPECTED TO DELIVER LIFE-CHANGING MEDICINES
POTENTIAL FIRST-IN-CLASS OR BEST-IN-CLASS NMEs
TARGET ADDRESSABLE ADDRESSABLE
PRODUCT MECHANISM INDICATION APPROVAL DATE POPULATION POPULATION
(FY)1 (IN US)2 (WW)2,3
TAK-788 EGFR inhibitor (exon 20) NSCLC – 2L / 1L 20214 / 2023 ~2k ~20 - 30k
pevonedistat (TAK-924) NAE inhibitor HR-MDS / AML 20214 / 2024 ~7k / ~12k 15 - 20k / 20 - 25k
ONCOLOGY
TAK-007 CD19 CAR-NK Hematologic malignancies 2023 ~9k ~15 - 25k
TAK-609 ERT / I2S replacement Hunter CNS (IT) 2021 ~250 ~1 - 1.5k
RARE maribavir (TAK-620) UL97 kinase inh CMV infect. in transpl. 2021 ~7 - 15k ~25 - 45k
DISEASES
TAK-607 IGF-1/ IGFBP3 Complications of prematurity 20245 ~25k ~80 - 90k
Immunology
Hematology TAK-611 ERT / arylsulfatase A MLD (IT) 2023 ~350 ~1 - 2k
Metabolic
TAK-755 ERT/ ADAMTS-13 cTTP / iTTP 2023 / 2025 ~500 / ~2k 2 - 6k / 5 - 18k
Orexin programs Orexin 2R agonist Narcolepsy Type 1 2024 70 - 140k 300k - 1.2M
NEUROSCIENCE
TAK-935 CH24H inhibitor Developmental and Epileptic 2023 ~50k ~70 - 90k
Encephalopathies (DEE)
GASTRO-
ENTEROLOGY TAK-721 Oral anti-inflammatory Eosinophilic Esophagitis 2020 ~150k Under evaluation
VACCINES TAK-003 Vaccine Dengue 2021 ~32M ~1.8B
1. Projected timing of approvals depending on data read-outs; some of these target approval dates assume accelerated approval 4. Projected approval date assumes filing on Phase 2 data
2. Estimated number of patients projected to be eligible for treatment in markets where the product is anticipated to be 5. Currently in a non-pivotal Ph 2; interim stage gates may advance program into pivotal trial for 34
commercialized, subject to regulatory approval target approval by 2024
3. For TAK-788, TAK-924, TAK-007, TAK-607 and TAK-620 the addressable population represent annual incidence Currently in pivotal study or potential for registration enabling Ph-2 study (note: table excludes relugolix)
IN SUMMARY: ROBUST NEAR-TERM GROWTH
TAK-609 Hunter CNS (IT) Potential NME Approval
Eosinophilic
TAK-721
Esophagitis1
TAK-003 Dengue vaccine
Potential Global Brand Extension
UC/CD, CN maribavir
ENTYVIO sc UC/CD, US, EU, JP2 TAK-620
CMV transplant Potential Regional Brand Extension
NDMM nSCT, US, pevonedistat
NINLARO HR-MDS
EU TAK-924
1L NSCLC, US, EU
ALUNBRIG TAK-788 2L NSCLC3
2L NSCLC, JP
GATTEX SBS, JP TAKHZYRO HAE, JP GATTEX SBS, CN TAK-755 cTTP 5
1L NSCLC, CN NDMM SCT, US, Hematologic
TAKHZYRO HAE, CN ALUNBRIG NINLARO TAK-007
2L NSCLC, CN EU malignancies
Gaucher Disease, H2H alectinib, EU
VIPRIV ALUNBRIG ALUNBRIG H2H alectinib, US TAK-611 MLD (IT)
CN Post-2Gen, US, EU
sc UC, US NDMM, US, EU, JP
ENTYVIO FIRAZYR HAE CN NINLARO ENTYVIO GvHD, EU TAK-935 DEE4
CD, JP NDMM nSCT, JP
Complications of
GATTEX Pediatric, US REPLAGAL Fabry Disease, CN ALOFISEL CPF, JP VONVENDI Peds, US, EU, JP TAK-788 1L NSCLC4,5 TAK-607
prematurity
Ovarian 1L, 2L, JP CPF, US
NINLARO NDMM SCT, JP niraparib cabozantinib 1L RCC, JP ICLUSIG 1L Ph+ ALL, US ALOFISEL Orexin 2R ag Narcolepsy T1
Ov Salvage 1L, JP CCF
pevonedistat
ADCETRIS FL PTCL, JP VONVENDI VWD, JP vonoprazan OD ARD, JP ADYNOVATE HemA, CN VONVENDI Prophy, JP AML5
TAK-924
cabozantinib 2L RCC, JP ADCETRIS FL PTCL, EU relugolix Prostate, JP relugolix Prostate, CN ICLUSIG 1L Ph+ ALL, EU, JP TAKHZYRO BMA, US
REGIONAL Acid Reflux Dis.
vonoprazan cabozantinib HCC, JP VONVENDI Prophy, US, EU OBIZUR CHAWI, EU OBIZUR CHAWI, US NINLARO NDMM nSCT, CN
JP, CN
FY19 FY20 FY21 FY22 FY23 FY24
1. China approval in 2023 Potential approvals by fiscal year as of November 14, 2019
2. US approval for sc CD, EU approval for sc UC & CD, Japan approval for sc CD The target dates are estimates based on current data and subject to change
3. Includes approval in China
4. China approval in 2024 35
5. New indication for currently unapproved asset
SUSTAINED GROWTH BEYOND FY25
WAVE 2
NOVEL MECHANISMS
NEXT-GENERATION PLATFORMS
36
DRIVEN BY A CLINICAL PIPELINE OF NOVEL MECHANISMS…
TARGET APPROVAL1 FY25 AND BEYOND
TAK-164 TAK-252
GI malignancies Solid tumors
ONCOLOGY
TAK-573 TAK-981
R/R MM Multiple cancers
Rich early
TAK-0792 TAK-754 TAK-755
MG, ITP HemA iTTP, SCD
RARE Immunology
Hematology
DISEASES Metabolic TAK-531
Hunter CNS clinical
TAK-341
Parkinson’s
Orexin2R-ag TAK-041
pipeline of
potentially
Disease Sleep Disorders CIAS NS
TAK-418 TAK-653 TAK-831
NEUROSCIENCE Kabuki Syndrome TRD CIAS NS
transformative
WVE-120101 WVE-120102
Huntington’s
Disease
Huntington’s
Disease and curative
Kuma062
Celiac Disease
TAK-101
Celiac Disease
TAK-018
Crohn’s Disease
TAK-671
Acute
NMEs
GASTRO- (post-op and ileitis) Pancreatitis
ENTEROLOGY TAK-954 TAK-906 TAK-951
POGD Gastroparesis Nausea &
vomiting
TAK-214 TAK-426 TAK-021
VACCINES Norovirus Zika Vaccine EV71 Vaccine
Vaccine
1. Some Wave 2 assets could be accelerated into Wave 1 if they have breakthrough data Orphan potential in at least one indication
2. TAK-079 to be developed in Rare Diseases indications myasthenia gravis (MG) and immune thrombocytopenic purpura (ITP) (FPI
projected for 2H FY19) Estimated dates as of November 14, 2019
37
…AND WITH OUR NEXT-GENERATION PLATFORMS
TARGET APPROVAL FY25 AND BEYOND
CELL THERAPIES AND TARGETED INNATE IMMUNE NEXT-GEN CHECKPOINT
IMMUNE ENGAGERS MODULATION MODULATORS
CAR-T GammaDelta
Attenukine Agonist-redirected checkpoints
Teva Shattuck
ONCOLOGY MSKCC, Noile-
Immune
CAR-T
GammaDelta Tx STING Humabodies
T-CiRA, Takeda Conditional T cell CuraDev, Takeda Crescendo
CAR-NK engagers SUMOylation
MD Anderson Maverick Takeda
RARE
DISEASES
Immunology
Hematology
Metabolic
GENE THERAPY
Hemophilia
Harnessing the
potential of cell and
Lysosomal Storage Diseases
gene therapies and
NEUROSCIENCE
GENE THERAPY
Neurodegenerative Diseases
OTHER PLATFORMS
RNA Modulation
Wave, Skyhawk
other diverse
StrideBio Antibody Transport Vehicle
Denali modalities
MICROBIOME
GASTRO- GENE THERAPY FIN-524 CELL THERAPY
Liver FInch
ENTEROLOGY Ambys Ambys
Microbial Consortia
NuBiyota
Some Wave 2 assets could be accelerated into Wave 1 if they have breakthrough data Estimated dates as of November 14, 2019
38
INVESTING IN CAPABILITIES TO POSITION US FOR SUCCESS
• 5 clinical programs by end of FY20
Cell Therapy • Disruptive platforms, including off-the-
shelf cell-therapies
• World-class gene therapy
manufacturing
Gene Therapy
• Accessing innovation through
partnerships (e.g. Stridebio, Ambys)
• Accelerate clinical development with
real world data (e.g. TAK-788)
Data Sciences
• Use machine learning to identify rare
disease patients
39
COMMITTED TO OUR PEOPLE
40
LIVING OUR VALUES THROUGHOUT THE INTEGRATION PROCESS
December 2018
Leadership Team and Proposed R&D
Operating Model Announced
April 2019
Prioritization of Combined
Pipeline and Portfolio
August 2019
R&D Employees Informed
of Employment Status*
41
* Where legally cleared
STRONG LEADERSHIP EXECUTING ON OUR VISION
ASIT PARIKH PHIL ROWLANDS DAN CURRAN EMILIANGELO RATTI SARAH SHEIKH New hire
Head, Gastroenterology Head, Oncology Head, Rare Diseases Head, Neuroscience Head, Neuroscience
Therapeutic Area Unit Therapeutic Area Unit Therapeutic Area Unit Therapeutic Area Unit Therapeutic Area Unit*
*Sarah Sheik to succeed Emiliangelo Ratti
upon his retirement beginning November 25
†includes Regulatory, Global Patient Safety
Evaluation, Development Operations, and
Clinical Supply Chain
STEVE HITCHCOCK NENAD GRMUSA GEORGIA KERESTY ANNE HEATHERINGTON WOLFRAM NOTHAFT
Head, Research Head, Center for R&D Chief Operating Officer Head, Data Sciences Institute Chief Medical Officer
External Innovation
STEFAN WILDT JEREMY CHADWICK WOLFGANG HACKEL ERIKA MARDER COLLEEN BEAUREGARD TOSHIO FUJIMOTO
Head, Pharmaceutical Sciences Head, Global Development Head, Global R&D Finance Head, Global R&D Human Head, Global R&D General Manager, Shonan
and Translational Engine, Cell Office† Resources Communications Health Innovation Park (iPark)
Therapies
42
OUR COMMITMENT TO OUR PEOPLE IS BEING RECOGNIZED
43
WE ARE POSITIONED TO DELIVER NEAR-TERM & SUSTAINED GROWTH
WAVE 11 WAVE 22
TARGET CLINICAL-STAGE NMEs PLATFORMS
APPROVAL FY20 FY21 FY22 FY23 FY24 FY25 AND BEYOND
TAK-7883 TAK-007 TAK-924 TAK-164 TAK-252
2L NSCLC Hematologic AML GI malignancies Solid tumors TARGETED
malignancies CELL THERAPY INNATE NEXT-GEN
ONCOLOGY AND IMMUNE
ENGAGERS IMMUNE
CHECKPOINT
TAK-9243 TAK-788 TAK-573 TAK-981 MODULATION MODULATORS
HR-MDS 1L NSCLC R/R MM Multiple cancers
TAK-620 TAK-611 TAK-607 TAK-0794 TAK-754 TAK-755
CMV infect. in MLD (IT) Complications of MG, ITP HemA iTTP, SCD
Immunology
RARE Hematology
transplant prematurity GENE
THERAPY
DISEASES Metabolic
TAK-609 TAK-755 TAK-531
Hunter CNS (IT) cTTP Hunter CNS
TAK-935 Orexin2R-ag TAK-341 Orexin2R-ag TAK-041
DEE (TAK-925/994) Parkinson’s Sleep Disorders CIAS NS OTHER
Narcolepsy T1 Disease GENE PLATFORMS
THERAPY RNA Modulation
NEUROSCIENCE TAK-418 TAK-653 TAK-831 Antibody Transport
Vehicle
Kabuki Syndrome TRD CIAS NS
WVE-120101 WVE-120102
Huntington’s Huntington’s
Disease Disease
TAK-721 Kuma062 TAK-101 TAK-018 TAK-671
EoE Celiac Disease Celiac Disease Crohn’s Disease Acute
GASTRO- (post-op and ileitis) Pancreatitis GENE
MICROBIOME
CELL
THERAPY THERAPY
ENTEROLOGY TAK-954 TAK-906 TAK-951
POGD Gastroparesis Nausea &
vomiting
TAK-003 TAK-214 TAK-426 TAK-021
VACCINES Dengue Vaccine Norovirus Zika Vaccine EV71 vaccine
Vaccine
1. Projected timing of approvals depending on data read-outs; some of these Wave 1 target approval dates assume accelerated approval Orphan potential in at least one indication
2. Some Wave 2 assets could be accelerated into Wave 1 if they have breakthrough data 44
Estimated dates as of November 14, 2019
3. Projected approval date assumes filing on Phase 2 data
4. TAK-079 to be developed in Rare Diseases indications myasthenia gravis (MG) and immune thrombocytopenic purpura (ITP) (FPI projected in each indication in 2H FY19)
R&D DAY AGENDA – NEW YORK, NOVEMBER 14, 2019
TIME AGENDA
Welcome and Opening Remarks
12:30 – 12:35
Sheelagh Cawley-Knopf, Head R&D Global Portfolio Strategy
Takeda: A Global Values-Based, R&D-Driven Biopharmaceutical Leader
12:35 – 12:45
Christophe Weber, President & CEO Takeda
Translating Science into Highly Innovative, Life-changing Medicines
12:45 – 13:20
Andy Plump, President R&D
Oncology and Cell Therapies with Spotlight on CAR-NK
13:20 – 13:45
Chris Arendt, Head Oncology Drug Discovery Unit
Spotlight on Oncology Opportunities
13:45 – 14:05 • TAK-788 : Rachael Brake, Global Program Lead
• Pevonedistat : Phil Rowlands, Head Oncology Therapeutic Area Unit
14:05 – 14:20 Break
Rare Diseases & Gene Therapy
14:20 – 14:45
Dan Curran, Head Rare Disease Therapeutic Area Unit
Spotlight on Orexin2R agonists
14:45 – 15:00
Deborah Hartman, Global Program Lead
Therapeutic Area Focus in GI with Spotlight on Celiac Disease
15:00 – 15:20
Asit Parikh, Head GI Therapeutic Area Unit
15:20 – 16:00 Panel Q&A Session
16:00 Drinks reception
45
TAKEDA ONCOLOGY: INNOVATIVE CELL THERAPIES
& NEW FRONTIERS IN IMMUNO-ONCOLOGY
Chris Arendt, PhD
Head of Oncology Drug Discovery Unit
Takeda Pharmaceutical Company Limited
New York, NY
November 14, 2019
A CURATIVE-INTENT IMMUNO-ONCOLOGY PIPELINE IS TAKING SHAPE
WAVE 1 WAVE 2
NMEs that complement our global brands Leading platforms in immuno-oncology and cell therapies
Hematologic Hematologic
Malignancies Immuno-Oncology Malignancies
TAK-924
FY21 target approval
TAK-007
FY23 target approval
Lung Cancer & Lung Cancer &
Solid Tumors Solid Tumors
TAK-788
FY21 target approval
47
PARTNERSHIPS DRIVE OUR DIFFERENTIATED EARLY CLINICAL PIPELINE
Unique Differentiated
Partnership Portfolio
Model
• Innovative, disruptive platforms • Harness innate immunity
• Agility in ‘open lab’ model • Eye towards solid tumors
48
THE FIRST BREAKTHROUGHS IN CANCER IMMUNOTHERAPY
TARGET T CELLS
T CELL CHECKPOINT INHIBITORS FIRST-GEN CAR-Ts
PD-1 T cell
CAR
T cell
CTLA-4
Cancer cell death
49
Adapted from Chen & Mellman, Immunity 2013
OUR FOCUS IS ON NOVEL MECHANISMS IN THE
CANCER-IMMUNITY CYCLE
Novel-scaffold immune
2 checkpoint platforms
Next-gen cell therapy &
3 immune engager platforms
Innate immuno-
1 modulation
Cancer cell death
50
Adapted from Chen & Mellman, Immunity 2013
EMERGING STRENGTH IN TARGETED INNATE IMMUNE
1
MODULATION
HIGH UNMET Patients refractory/ unresponsive to current
NEED immunotherapies
OUR Systemic therapies leveraging innate immunity to
DIFFERENTIATED enhance response breadth, depth & durability
APPROACH
Cancer cell death
PLATFORM PARTNER MECHANISM-OF-ACTION PROGRAMS PRE-CLINICAL PH 1
TAK-676 (STING agonist)
STING agonism • Innate-to-adaptive priming
Targeted STING agonist
TAK-981
SUMOylation • Innate immune enhancer
TAK-981 (ADCC combo)
TAK-573 (CD38-AttenukineTM)
AttenukineTM • Targeted attenuated IFN-α
Next-gen AttenukineTM
51
ADCC = Antibody-dependent cellular cytotoxicity = first-in-class
ATTENUKINETM PLATFORM ELICITS BOTH DIRECT
1
TUMOR KILL AND IMMUNE ACTIVATION
TARGETED ATTENUATED TYPE I IFN PAYLOAD TAK-573 POM IN ONGOING PHASE 1 R/R MM STUDY
TAK-573
Activation of CD8+ T cells in bone marrow
Binds CD38 Immunomodulation in
Baseline Cycle 1 Day 16 Cycle 2 Day 2
preclinical models
7.3% 18.4% 28.8%
Includes CD8+ T cell
CD8+ T cells
Human IgG4 Fc
migration / activation
Attenuated IFNα2b
Activation Marker (CD69+)
NEXT-GEN Binds innate
immune
ATTENUKINETM target
EXPECTED 2019 2020
MILESTONES (FY) Ph1 FPI in solid Ph1b MM (incl.
tumors combinations)
Attenuated IFNα2b
FPI = first patient in R/R MM = Relapsed / refractory multiple myeloma POM = proof-of-mechanism
52
1 NOVEL SCAFFOLD NEXT-GENERATION CHECKPOINT MODULATORS
HIGH UNMET Current checkpoint modulators fail to improve
NEED overall survival in majority of patients
OUR New classes of checkpoint inhibitors designed
DIFFERENTIATED to increase breadth and depth of responses
APPROACH
Cancer cell death
PLATFORM PARTNER MECHANISM-OF-ACTION PROGRAMS PRE-CLINICAL PH 1
Concept 1
Humabody Vh • Unique pharmacology
Concept 2
Agonist-redirected • Co-inhibition & co- TAK-252 / SL-279352 (PD1-Fc-OX40L)
checkpoints stimulation TAK-254 / SL-115154 (CSF1R-Fc-CD40L)
53
= first-in-class
Vh = Variable heavy domain
BRINGING 5 NOVEL CELL THERAPY PLATFORMS
1
TO THE CLINIC BY THE END OF FY20
HIGH UNMET Current CAR-T therapies have significant
NEED challenges & fail to address solid tumors
OUR Leverage novel cell platforms & engineering to
DIFFERENTIATED address shortcomings in liquid & solid tumors
APPROACH
Cancer cell death
INNATE IMMUNE PLATFORMS Innate tumor sensors & effectors
• Multiple mechanisms of tumor killing NK &
γδT Engineered CAR
• ‘Off-the-shelf’
cells
• Utility in solid tumors
Fc-mediated killing
54
NK = Natural killer
A NETWORK OF TOP INNOVATORS IS
1
FUELING TAKEDA’S CELL THERAPY ENGINE
CUTTING-EDGE ENGINEERING & CELL PLATFORMS
IPSC γδT cell Armored Next-gen IPSC CAR-NK
expertise platform CAR-Ts CARs CAR-Ts platform
Shinya Adrian Koji Michel Shin Katy
Yamanaka Hayday Tamada Sadelain Kaneko
Kaneko Rezvani
2016
2017
2018
2019
Dec 2015 May 2017 Sept 2017 July 2018 April 2019 Nov 2019
Takeda Cell Therapy First Development-Stage
Translational Engine Partnership
IPSC = Induced pluripotent stem cell NK = Natural killer
55
Dr. Sadelain is a co-inventor on patents relative to next-gen CARs, intellectual property that MSK has licensed to Takeda. As a
result of these licensing arrangements, Dr. Sadelain and MSK have financial interests related to these research efforts.
TAKEDA IS EMBARKING ON A TRANSFORMATIVE CAR-NK
1
PARTNERSHIP THAT COULD ENTER PIVOTAL TRIALS IN 2021
IL-15 Activating
NK receptor
NK CAR
Platform
Multiple mechanisms
of tumor killing
Potentiation of innate
& adaptive immunity
CAR19
56
1 FOUR NOVEL, OFF-THE-SHELF CAR-NK THERAPIES IN DEVELOPMENT
PATIENT VALUE PROPOSITION PLATFORM VALUE INFLECTIONS
Rapid and deep responses with a short-time-to-treatment, FY
safe, off-the-shelf CAR-NK available in outpatient & Ongoing maturation of clinical data: Efficacious dose,
community settings
2H 2020 durability, partial vs. full allo, cryopreserved product
Initial opportunity in G7 countries (CD19)* Manufacturing process complete
3L+ DLBCL ~8,000
2021 Pivotal trials in r/r DLBCL / CLL / Indolent NHL
3L+ CLL ~5,000
3L+ iNHL ~6,000
Potential to move into earlier lines of therapy 2023 BLA filing
PLATFORM PARTNER MECHANISM-OF-ACTION PROGRAMS PRECLINICAL PH 1
TAK-007 (CD19 CAR-NK)
CAR-NK • Non-autologous NK cell therapy BCMA CAR-NK
(allo cord blood) Platform expansion
Dr. Katy Rezvani
= first-in-class
CLL = Chronic lymphocytic leukemia DLBCL = Diffuse large B-cell lymphoma iNHL = Indolent non-Hodgkin’s lymphoma 57
*Estimated number of patients projected to be initially eligible for treatment in G7 markets, subject to regulatory approval
1 DRAMATIC COMPLETE RESPONSE IN FIRST PATIENT TREATED
47-YEAR OLD MALE WITH RELAPSED TRANSFORMED KINETICS OF CAR-NK VERSUS ENDOGENOUS T AND B
DOUBLE-HIT (C-MYC / BCL-2) DLBCL CELLS IN PERIPHERAL BLOOD
CAR-NK cells
% positive cells
Days post-CAR-NK infusion
X1000/ml of blood
T cells
B cells
Baseline scan Days post-CAR-NK infusion
Day 30 post CAR19-NK
58
Data from Dr. Katy Rezvani, MD Anderson Cancer Center
1 IMPRESSIVE RESPONSES IN OTHER HEAVILY PRETREATED PATIENTS
61-YEAR OLD MALE CLL/RICHTER’S TRANSFORMATION 60-YEAR OLD FEMALE WITH CLL / ACCELERATED CLL
(5 PRIOR LINES OF THERAPY) (5 PRIOR LINES OF THERAPY)
CAR-NK
Baseline scan Day 30 post CAR19-NK Baseline scan Day 30 post CAR19-NK
CR in Richter’s; SD in CLL 59
CLL = Chronic lymphocytic leukemia CR = Complete response SD = Stable disease
Data from Dr. Katy Rezvani, MD Anderson Cancer Center
CAR-NK CELLS PERSIST IN PATIENTS AND DO NOT TRIGGER
1
CYTOKINE RELEASE SYNDROME (CRS)
CAR-NK CELLS PERSIST UP TO 4 MONTHS POST INFUSION IL-6 LEVLS POST CAR-NK INFUSION DO NOT INDICATE CRS
Median IL-6 level in grade 2-5 CRS post-CAR-T treatment*
Vector transgene copy per µg of genomic DNA
IL-6 (pg/ml)
Time from infusion (days) Time from infusion (days)
CRS = Cytokine Release Syndrome 60
*Turtle et al. 2017
Data from Dr. Katy Rezvani, MD Anderson Cancer Center
1 CAR-NK EFFICACY & TOXICITY TREATING MULTPLE DIAGNOSES
Lines of CRS / Complete
Diagnosis HLA Match
Treatment Neurotox Response
3
DLBCL - Relapsed transformed double-hit
Incl. ASCT
Partial match None
Dose
DLBCL - Refractory 7 Partial match None PD
Level 1
4
CLL
Incl. ibrutinib & venetoclax
Partial match None
CLL 4 Partial match None PD
Incl. ibrutinib
Dose
CLL/Richter’s transformation 5
Incl. ibrutinib
Partial match None *
Richter’s
Level 2 5
CLL/Accelerated CLL
Incl. ibrutinib & venetoclax
Partial match None CLL = Chronic lymphocytic leukemia
4
CLL
Incl. ibrutinib
Partial match None
CRS = Cytokine release syndrome
DLBCL = Diffuse large B-cell lymphoma
11
DLBCL - Refractory
Incl. ASCT
Partial match None
ASCT = Autologous stem cell transplant
4
HLA = Human leukocyte antigen
Dose DLBCL - Relapsed transformed double-hit Partial match None
Incl. ASCT PD = Progressive disease
Level 3 4
Follicular lymphoma - Relapsed Mismatch None PD *Complete response for Richter’s
Incl. ASCT
Follicular lymphoma - Relapsed 4 Mismatch None
61
Data from Dr. Katy Rezvani, MD Anderson Cancer Center
FAST-TO-CLINIC CELL THERAPY ENGINE WILL MAXIMIZE
1
LEARNINGS ON MULTIPLE ‘DISRUPTIVE’ PLATFORMS
5 CLINICAL-STAGE PROGRAMS EXPECTED BY END OF FY20
FY19 FY20 FY21+:
Other cell
TAK-007 Off-the-shelf TAK-102 Cytokine + therapy
CAR-NK product
chemokine candidates
armed CAR-T
CD19 1XX-CAR-T
Next-gen CART
signaling domain
Hematology
GDX012 Gamma-delta
T cells
Solid tumors
GCC CAR-T Colorectal
Cancer
62
A RICH AND POTENTIALLY TRANSFORMATIVE EARLY CLINICAL
1
ONCOLOGY PIPELINE
PLATFORM PARTNER(S) MECHANISM-OF-ACTION PROGRAMS PRECLINICAL PH1
TAK-676 (STING agonist) UNDISCLOSED
STING agonism • Innate-to-adaptive priming
Targeted STING agonist TARGETS
TAK-981
SUMOylation • Innate immune enhancer
TAK-981 (ADCC combo)
AttenukineTM • Targeted attenuated IFN-α TAK-573 (CD38-AttenukineTM)
Agonist-redirected TAK-252 / SL-279353
• Co-inhibition & co-stimulation
checkpoints TAK-254 / SL-115154
Shiga-like toxin A • Novel cytotoxic payload TAK-169 (CD38-SLTA)
IGN toxin • Solid tumor-targeted ADC TAK-164 (GCC-ADC)
Conditional T
• Novel solid tumor platform MVC-101 (EGFR COBRATM)
cell engagers
Cell therapy TAK-007 (CD19 CAR-NK)
• Off-the-shelf cell therapies
platforms 5 cell therapies expected in clinic by end of FY20
= first-in-class
Hematology Solid tumors 63
NME MILESTONES ACHIEVED IN FY19 AND LOOKING AHEAD TO
OTHER POTENTIAL MILESTONES1 THROUGH FY20
PIVOTAL STUDY STARTS, APPROVALS
MLD PEVONEDISTAT AML EoE
TAK-611
Ph 2 start2 TAK-924 Ph 3 start
TAK-721
Approval
cTTP 1L NSCLC Huntington’s Disease
TAK-755
Ph 3 start
TAK-788
Ph 3 start
mHTT ASO
Pivotal start
1H FY 2019 2H FY 2019 1H FY 2020 2H FY 2020
Narcolepsy PEVONEDISTAT HR-MDS 2L NSCLC
TAK-925
POC TAK-924 Ph 2 Overall Survival
TAK-788
Ph 2 Pivotal
TAK-620
R/R CMV SOT & HSCT
Ph 3 data
TAK-721
EoE
Ph 3 data (induction) TAK-007
Hem. Malignancies
POC
TAK-573
R/R MM, Solid Tumor
POC
TAK-755
iTTP
POC
Celiac Disease Hunter (IT) DEE
TAK-101
POC
TAK-609
Ph 3 data 2yr extension
TAK-935
POC
Huntington’s Disease Gastroparesis
mHTT ASO TAK-906
POC POC
EoE Nausea & Vomiting
TAK-721 TAK-951
Ph 3 data (maintenance) POC
Oncology
Rare Disease
Neuroscience
Gastroenterology
Denotes milestones that have been achieved.
KEY DATA READOUTS
64
1. Potential key milestone dates as of November 14, 2019. The dates included herein are estimates based on current data and are subject to change
2. Potentially registration enabling
SUMMARY
1 2 3
Total transformation Differentiated Multiple near-term
of preclinical & early opportunities in IO catalysts informing
clinical pipeline leveraging innate momentum towards
immunity & cell solid tumors
therapies
65
R&D DAY AGENDA – NEW YORK, NOVEMBER 14, 2019
TIME AGENDA
Welcome and Opening Remarks
12:30 – 12:35
Sheelagh Cawley-Knopf, Head R&D Global Portfolio Strategy
Takeda: A Global Values-Based, R&D-Driven Biopharmaceutical Leader
12:35 – 12:45
Christophe Weber, President & CEO Takeda
Translating Science into Highly Innovative, Life-changing Medicines
12:45 – 13:20
Andy Plump, President R&D
Oncology and Cell Therapies with Spotlight on CAR-NK
13:20 – 13:45
Chris Arendt, Head Oncology Drug Discovery Unit
Spotlight on Oncology Opportunities
13:45 – 14:05 • TAK-788 : Rachael Brake, Global Program Lead
• Pevonedistat : Phil Rowlands, Head Oncology Therapeutic Area Unit
14:05 – 14:20 Break
Rare Diseases & Gene Therapy
14:20 – 14:45
Dan Curran, Head Rare Disease Therapeutic Area Unit
Spotlight on Orexin2R agonists
14:45 – 15:00
Deborah Hartman, Global Program Lead
Therapeutic Area Focus in GI with Spotlight on Celiac Disease
15:00 – 15:20
Asit Parikh, Head GI Therapeutic Area Unit
15:20 – 16:00 Panel Q&A Session
16:00 Drinks reception
66
TAK-788: PURSUING A FAST-TO-PATIENT STRATEGY
FOR NSCLC PATIENTS WITH EGFR EXON 20 INSERTIONS
Rachael L Brake, PhD
Global Program Leader, Oncology
Takeda Pharmaceutical Company Limited
New York, NY
November 14, 2019
THE SIZE OF THE LUNG CANCER CHALLENGE IS VAST
Survival of Lung cancer is amongst
228,0001 the lowest of all cancers
New Lung cancer
cases / year
Male Female
10% 13%
143,0001 survival survival
Lung cancer deaths/ yr
More than breast, colon,
and prostate cancer
combined 5 yr survival estimates among adults diagnosed with
lung cancer between 2007-20112
1. American Cancer Society; Cancer facts and figures 2019 68
2. Office for National Statistics UK (www.ons.gov.uk)
EXON 20 INSERTIONS ARE A RARE SUBSET OF EGFR MUTANT NSCLC
Non-Sq NSCLC EGFR Exon 20 insertions
200,000 pts/yr1 2,000 pts/yr2
EGFR Sensitizing Mutations 19.4%
No Mutations 1.2%
EGFR exon18 4%
UMD 12.0% EGFR exon19 45%
EGFR exon21 41%
Other Drivers 2.9%
Insertion variants
PTEN loss 0.7%
CDKN2A loss 1.9%
BRAF nonV600E 1.3%
EGFR 28% 1. V769_D770insASV (≈20%)
NF1 loss 1.9% 2. D770_N771insSVD (≈19%)
EGFR T790M 5.5%
3. H773_V774insH (≈8%)
EGFR exon20 2.1% 4. A763_Y764insFQEA (≈7%)
KRAS 25.3% EGFR WT Amp 1.0% 5. H773_v774insPH (≈5%)
ALK fusion 3.8% 6. H773_V774insNPH (≈4%)
KRAS 25.3%
ROS1 fusion 2.6% 7. N771_P772insN (≈3%)
RET fusion 1.7%
8. H773_V774insAH (≈3%)
BRAF V600E 2.1%
MET splice 3.0% 9. Other (≈31%)
FGFR ½ ½ 0.7% MET Amp 1.4%
NRAS 1.2% ERBB2 Amp 1.4%
PIK3CA 2.0% BRCA ½ loss 1.3%
MAP2K1 0.7% TSC ½ loss 0.7%
ERBB2 Mut 2.3%
Sources: Leduc C et al., Ann Oncol 2017; Jorge S et al. Braz J Med Biol Res 2014; Kobayashi Y & Mitsudomi 1. Estimated US annual incidence of non-squamous NSCLC 69
T. Cancer Sci 2016; Arcila M et al. Mol Cancer Ther 2013; Oxnard G et al. J Thorac Oncol 2013 2. Represents annual incidence of the US addressable patient population
PATIENTS WITH EGFR EXON 20 INSERTIONS HAVE NO EFFECTIVE THERAPY
POOR RESPONSE TO EXISTING TKIs 1 POOR RESPONSE TO ANTI PD-1/PDL-1 THERAPY 2
EGFR exon 20 insertions do not demonstrate EGFR exon 20 ins patients demonstrate limited
significant PFS benefit with 1st and 2nd gen EGFR TKIs benefit to anti PD-1 directed therapy
100 100%
Classic EGFR EGFR exon 20
80%
80
Progression-free survival (%)
Best change in target lesions (%)
60%
60 Hazard ratio
40%
= 12.3 (p<0.0001)
40 20%
0%
20
-20%
0
10 20 30 40 50 -40%
Time (months)
-60% Individual patient responses
Group Median PFS (months) Group Median PFS (months) PDL-1 expression ≥1%
EGFR exon 20 ins (n=9) 2.0 EGFR exon 20 ins (n=20) 2.7 (1.7-3.8) 40%
Classical EGFR mut (n=129) 12.0 Classical EGFR mut (n=22) 1.8 (1.2-2.4) 25%
1. Robichaux et al., WCLC 2016. 70
2. Adapted from Negrao et al., WCLC 2019
OVERCOMING THE DRUG DEVELOPMENT CHALLENGE
IN EXON 20 INSERTIONS
EGFR exon 20 NPG insertion L858R EGFR mutation
Wild type EGFR Wild type EGFR Classical EGFR
mutation
L858R
TAK-788
TAK-788
EGFR exon 20 insertion
EGFR D770 ins NPG
EGFR exon 20 insertion mutations Classical EGFR mutations
have a similar structure and similar affinity for Significantly alter both structure and affinity
ATP to wild type EGFR for ATP compared to wild type EGFR 71
Source. TAK-788 bound to EGFR kinase domain containing D770 ins NPG, crystal structure (data on file)
TAK-788 PROOF OF CONCEPT DATA IN EGFR EXON 20 INSERTIONS
• Confirmed ORR: 12/28 patients: 43% (24.5-62.8%) • Median PFS: 7.3 months (4.4 mo - NR)
ANTITUMOR ACTIVITY IN EGFR EXON 20 INS AT 160 MG DAILY SAFETY SUMMARY IN PATIENTS TREATED WITH TAK-788
All Patients
80 N (%)
160 mg qd (n=72)
Best change in target lesions (%)
60
40 Treatment-related AE
20
0 Any grade 68 (94)
-20 Grade ≥3 29 (40)
-40
-60 Dose reduction due to AE 18 (25)
-80 Dose interruption due to AE 36 (50)
-100
Discontinuation due to treatment-
Individual patient responses 10 (14)
related AE
Prior TKI: N N N N Y N N N N N N N Y N N N N N N N N N Y Y N
Prior IO: N Y Y N Y N N N N Y Y N Y Y N Y Y Y N N Y N Y Y Y
TAK-788 has not been approved for the use or indications under investigation in the clinical trials (and there is no guarantee it will be approved for such use or indication). Claims of safety and effectiveness can only be
made after regulatory review of the data and approval of the labeled claims. 72
Adapted from Riley et al. ASCO. 2019
ENCOURAGING EFFICACY AND SAFETY HAS BEEN OBSERVED
WITH TAK-788
Select signs of efficacy
Clinical feature TAK-788 1 Poziotinib 2 Afatinib 3 Osimertinib 4
n=28 n=50 n=23 n=15
ITT confirmed ORR (%) 43% NR 8.7% 0%
Evaluable confirmed ORR (%) NR 43% NR NR
ITT median PFS (months) 7.3 5.5 2.7 3.5
Select treatment related adverse events attributable to wild type EGFR inhibition
Grade ≥ 3 Adverse event TAK-788 1 Poziotinib 2 Afatinib 5 Osimertinib 6