Clinical Trial Summary
May 2021

OVERVIEW OF CLINICAL TRIAL SUMMARYLCM1ONCOLOGYRARE
GENETIC &HEMATOLOGYPDTVACCINES

1 |1.

WAVE 2

ALUNBRIG 1L ALK+ NSCLCALUNBRIG 2L ALK+NSCLC H2H with alectinib
ICLUSIG TKI res. Chronic phase CML
ICLUSIG 1L Ph+ ALL

mobocertinib 2L NSCLC w/EGFR exon 20 insertion mutation
mobocertinib 1L NSCLC w/EGFR exon 20 insertion mutation
pevonedistat HR-MDS
pevonedistat Unfit AML

TAK-981 Multiple cancers
TAK-981 Non-Hodgkin’s lymphoma
TAK-981 Solid tumors
TAK-981 R/R multiple myeloma

NINLARO Maintenance ND MM post-SCT (MM3)

TAK-007 CD19+ Heme malignancies

TAK-573 Solid tumors

NINLARO Maintenance ND MM no-SCT (MM4)
NINLARO Maintenance no-SCT (MM6)ADYNOVATE Pediatric Hemophilia A
VONVENDI vWD Adult prophylaxis, Peds
TAKHZYRO HAE Pediatric
TAKHZYRO Bradykinin-mediated angioedema
OBIZUR Acquired Hemophilia A

NEUROSCIENCEGIWAVE 1ENTYVIO GvHD Prophylaxis
ENTYVIO UC/CD SCENTYVIO Pediatric UC/CD
Alofisel Complex perianal fistulas in CD
Vonoprazan H. Pylori China
HYQVIA CIDPHYQVIA Pediatric PID
GLASSIA A1P1 deficient patients

TAK-573 R/R multiple myeloma
maribavir R/R CMV infection in HSCT and SOT
maribavir 1L CMV infection In HSCT
TAK-755 cTTP
TAK-611 MLD (IT)TAK-609 Hunter CNS (IT)TAK-994 Orexin 2-ag NT1 and NT2
TAK-925 Narcolepsy NT1 and other sleep disorders
Soticlestat Rare epilepsies – LGS, DS
TAK-721 Eosinophilic Esophagitis

mezagitamab (TAK-079) ITP, MG
TAK-607 Complications of prematurity
TAK-755 iTTP
TAK-755 SCD

TAK-003 Dengue vaccine
TAK-919 SARS-CoV-2 vaccine
TAK-019 SARS-CoV-2 vaccine

TAK-214 Norovirus vaccine
TAK-426 Zika vaccine

TAK-605 Multiple cancers
TAK-676 STING agonist solid tumors
TAK-252 Bispecific solid tumors
TAK-102 CAR-T solid tumors
TAK-940 CAR-T CD19+ Hememalignancy
TAK-186 EGFR expressing malignancies

TAK-341 Parkinson's DiseaseTAK-071 Parkinson's Disease
TAK-951 Nausea & Vomiting
TAK-510 Nausea & Vomiting
TAK-906 Gastroparesis
TAK-954 POGD
sibofimloc Post-Op CD

LCM = Life cycle management programs or marketed assets in development seeking new indications, new geographic expansions, fulfillment of regulatory requirements, new formulations/method of use, and/or enhancement incommercial/competitive profile.

OVERVIEW OF CLINICAL TRIAL SUMMARYONCOLOGY

2 |2

RARE GENETIC & HEMATOLOGY 19

PLASMA-DERIVED THERAPIES

47

NEUROSCIENCE

30

VACCINES

52

GASTROENTEROLOGY (GI)37

Oncology

ALUNBRIG (BRIGATINIB): ALK INHIBITOR
Study

NCT02737501

NCT03596866

Indication

ALK-positive advanced lung cancer

ALK-positive non-small-cell lung cancer (NSCLC)

Phase

Phase III
ALTA-1L

Phase III
ALTA-3# of Patients

N = 275

N = 246

Target Patients

ALK+ locally advanced or metastatic NSCLC patients who have notpreviously been treated with an ALK inhibitor

Patients with ALK+ locally advanced or metastatic NSCLC who haveprogressed on crizotinib

Arms/Intervention• Arm A: Brigatinib 180 mg QD with 7-day lead-in at 90 mg• Arm B: Crizotinib 250 mg BID• Arm A: Brigatinib 90 mg to 180 mg QD• Arm B: Alectinib 600 mg PO BID with foodPrimary endpoint and key
secondary endpoint(s)Progression-Free Survival (PFS) as assessed by blinded Independent
Review Committee (bIRC)

Progression-Free Survival (PFS) as assessed by blinded Independent
Review Committee (bIRC)• Study start date: April 2016
• Primary completion date: June 2019

Status3 | 1.

Publications:• Camidge DR, et al. N Engl J Med 2018;379(21): 2027-2039• Camidge DR, Kim HR, Ahn MJ, et al. J Clin Oncol 2020;38: 1-13

The primary endpoint, PFS, is event driven and changes in event rate can lead to a change in the primary completion date.

• Study Start Date: April 2019
• Estimated primary completion date1: FY23

Oncology

ICLUSIG (PONATINIB): BCR-ABL INHIBITOR
Study

NCT02467270

NCT03589326

Indication

Chronic myeloid leukemia (CML)

Ph+ acute lymphoblastic leukemia (ALL)

Phase

Phase II
OPTIC

Phase III
Ph+ALLCON# of Patients

N = 276

N = 230 (max)Target Patients

Patients with resistant chronic phase chronic myeloid leukemia

Patients with newly-diagnosed Ph+ ALL

Arms/Intervention• Ponatinib 45 mg once daily• Ponatinib 30 mg once daily• Ponatinib 15 mg once daily• Cohort A: Ponatinib/reduced intensity chemotherapy until
progressive disease (PD) or stem cell transplant (SCT)• Cohort B: Imatinib/reduced intensity chemotherapy until PD or SCT

Primary endpoint and key
secondary endpoint(s)≤1% BCR-ABL1 at 12 months (time frame: 12 months)• Primary: Number of participants with Minimal Residual Disease(MRD) -Negative Complete Remission (CR)[Time frame: From Cycle 1 through Cycle 3 (approximately 3 months)(Cycle length is equal to 28 days)]• Secondary: EFS

Status• Study start date: June 2015
• Primary completion date: April 2020
• Estimated study completion: September 2021

• Study start date: January 2019
• Estimated primary completion date1: FY24

4 | 1.

The key secondary endpoint, EFS, is event driven and changes in event rate can lead to a change in the primary completion date.

NINLARO (IXAZOMIB): ORAL PROTEASOME INHIBITOR

Oncology

Study

NCT02181413

NCT02312258

Indication

Multiple myeloma (MM) maintenance post-stem cell transplant

Multiple myeloma (MM) maintenance non-stem cell transplant

Phase

Phase III
TOURMALINE-MM3

Phase III
TOURMALINE-MM4# of Patients

N = 652

N = 761

Target Patients

Patients with multiple myeloma following autologous stem celltransplantPatients with newly-diagnosed MM not treated with stem celltransplantation

Arms/Intervention

Arm A: Ixazomib• Cycles 1-4: Ixazomib 3.0 mg PO days 1, 8, 15 / 28-day cycle• Cycles 5-26: Ixazomib 3.0 or 4.0 mg PO days 1, 8, 15 / 28-day cycle
Arm B: Placebo• Cycles 1-4: Placebo 3.0 mg PO days 1, 8, 15 / 28-day cycle• Cycles 5-26: Placebo 3.0 or 4.0 mg PO days 1, 8, 15 / 28-day cycle

Arm A: Ixazomib• Cycles 1-4: Ixazomib 3.0 mg PO days 1, 8, 15 / 28-day cycle• Cycles 5-26: Ixazomib 3.0 mg or 4.0 mg PO days 1, 8, 15 / 28-day cycle
Arm B: Placebo• Cycles 1-4: Placebo 3.0 mg PO days 1, 8, 15 / 28-day cycle• Cycles 5-26: Placebo 3.0 or 4.0 mg PO days 1, 8, 15 / 28-day cycle

Primary endpoint and key
secondary endpoint(s)• Primary: Progression Free Survival (PFS)• Secondary: Overall Survival (OS)• Primary: Progression Free Survival (PFS)• Secondary: Overall Survival (OS)Status• Study start date: July 2014
• Primary completion date: April 2018
• Interim OS analysis1: FY21; Final: FY24/25
Publications:• Dimopoulos MA, et al. Lancet. 2019 Jan 19;393(10168): 253-264• Kaiser M, et al. Ann Hematol. 2020 Aug;99(8): 1793-1804• Goldschmidt H, et al. Leukemia. 2020 Nov;34(11): 3019-3027• Paiva B, et al. Presentation at EHA 2020

• Study start date: April 2015
• Primary completion date: August 2019
• Interim OS analysis1: FY20; Final FY24
Publications:• Bringhen S, et al. Presentation at ASH 2020
• Paiva B, et al. Presentation at ASH 2020
• Dimopoulos MA, et al.
https://ascopubs.org/doi/full/10.1200/JCO.20.02060

5 | 1.

A key secondary analysis, OS, is event driven and changes in event rate can lead to a change in the interim and final analysis.

NINLARO (IXAZOMIB): ORAL PROTEASOME INHIBITOR

6 |

Study

NCT03173092

Indication

Non-transplant eligible patients with newly diagnosed multiple myeloma

Phase

Phase IV
MM6

# of Patients

N = 160

Target Patients

Patients with multiple myeloma previously receiving a bortezomib-based induction. In-class (proteasome inhibitor) transition after 3cycles of bortezomib-based therapy.

Arms/Intervention• Ixazomib 4 mg + lenalidomide 25 mg + dexamethasone 40 mg• Transition from a bortezomib based regimen to IRD (ixazomib, lenalidomide, dexamethasone) may allow the long-term proteasome
inhibition to be maximized while maintaining a manageable safety profile.

Primary endpoint and key
secondary endpoint(s)Progression Free Survival (PFS).
Key secondary endpoints: time to next therapy (TTNT), relative dose intensity (RDI) of the oral regimen, overall survival (OS), electronic
patient reported outcomes (ePRO) and actigraphy (activity/sleep) data.

Status• Study start date: September 2017
• Primary completion date: FY25
Publications:• Kambhampati, et al., Presentation at AVAHO 2020
• Manda S, et al., Clin Lymphoma Myeloma Leuk. 2020 Nov;20(11):e910-e925• Girnius, et al., Presentation at ASH 2020

Oncology

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 II
EXCLAIM

Phase III
EXCLAIM-2# of Patients

N = 334

N = 318

Target Patients

2L+ NSCLC harboring EGFR in-frame exon 20 insertion mutations

1L NSCLC harboring EGFR in-frame exon 20 insertion mutations

Arms/Intervention• Single arm: Mobocertinib 160 mg QD• Arm A: Mobocertinib 160 mg QD• Arm B: Platinum-based chemotherapy

Primary endpoint and key
secondary endpoint(s)Confirmed ORR assessed by IRC

PFS as assessed by blinded Independent Review Committee (IRC)

Status• Study start date: June 2016
• Primary completion date: May 2020

• Study start date: January 2020
• Estimated primary completion date1: FY22

7 | 1.

The primary endpoint, PFS, is event driven and changes in event rate can lead to a change in the primary completion date.

Oncology

PEVONEDISTAT (TAK-924):
NEDD8-ACTIVATING ENZYME (NAE) INHIBITOR
Study

NCT03268954

NCT04090736

Indication

HR MDS

Unfit AML

Phase

Phase IIIPANTHER

Phase IIIPEVOLAM# of Patients

N = 450

N = 466

Target Patients

Patients with higher risk myelodysplastic syndromes (HR MDS),
chronic myelomonocytic leukemia or low-blast acute myelogenous
leukemia (LB AML)

Patients with acute myeloid leukemia (AML) not eligible for
INTENSIVE chemotherapy

Arms/Intervention• Arm A: Pevonedistat 20 mg/m2 (IV) on days 1, 3, 5; Azacitidine (AZA)
75 mg/m2 (IV or SC) on a 5-on/2-off [weekend]/2-on schedule in 28day cycles• Arm B: AZA 75 mg/m2 (IV or SC) on a 5-on/2-off [weekend]/2-onschedule in 28-day cycle• Arm A: Pevonedistat 20 mg/m2 (IV) on days 1, 3, 5; Azacitidine (AZA)
75 mg/m2 (SC) on a 5-on/2-off [weekend]/2-on schedule in 28-day
cycles• Arm B: AZA 75 mg/m2 SC on a 5-on/2-off [weekend]/2-on schedulein 28-day cycle (IV AZA can be administered for any patients who
have non-tolerated local reactions)Primary endpoint and key
secondary endpoint(s)Primary: Event Free Survival (EFS)
Secondary: Overall Survival (OS)Overall Survival (OS)Status• Study start date: December 2017
• Primary completion date: September 2021

• Study start date: August 2019
• Estimated primary completion date1: FY24

8 | 1.

The primary endpoint, OS, is event driven and changes in event rate can lead to a change in the primary completion date.

OncologyTAK-007: CD19 CAR NK
Study

NCT030563391

Indication

Relapsed refractory B-lymphoid malignancies

Phase

Phase I/II

# 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 300 mg/m2 by vein on days -5 to -3• iC9/CAR.19/IL15-Transduced CB-NK Cells: Infusion of iC9/CAR.19/IL15-transduced CB-NK cells on Day 0 by vein; starting dose: 10E5
• AP1903: If participant has graft-versus-host disease (GvHD) or cytokine release syndrome after the NK cell infusion, they will receive
AP1903 0.4 mg/kg administered as an intravenous infusion.

Primary endpoint and key
secondary endpoint(s)Safety and efficacy• Study start date: June 2017

Status

9 | 1.

Publication:• Liu E, Marin D, Banerjee P, et al. N Engl J Med 2020;382(6): 545-553

Trial managed by MD Anderson Cancer Center. The CD19 CAR-NKs evaluated in this study are generated with the same CAR construct used for TAK-007 but with a different formulation and cannot be equated with TAK-007.

Oncology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 = 80N = 130

Target Patients

Adult participants with advanced or metastatic solid tumors or
relapsed/refractory hematologic malignancies

Patients with relapsed/refractory CD-20 positive NHL

Arms/Intervention• TAK-981, intravenously, administered as 60 minute-infusion, once
on Days 1, 4, 8, and 11 for 2 consecutive weeks, followed by 1
week rest in a 21-day treatment cycle

•
•
•
•

Primary endpoint and key
secondary endpoint(s)Safety, tolerability and PK

Safety, tolerability and RP2D

Status• Study start date: October 2018
• Estimated primary completion date: December 2022

• Study start date: October 2019
• Estimated primary completion date: September 2022

10 | 1.

SUMO = Small ubiquitin-like modifier

Oncology

Phase 1, aNHL/iNHL: TAK-981 (10-160 mg) + rituximab 375 mg/m2
Phase 2, Cohort A: r/r DLBCL progressed to CAR T-cell therapy
Phase 2, Cohort B: r/r DLBCL with no CAR T-cell prior therapy
Phase 2, Cohort C: r/r FL progressed to systemic therapies

TAK-981: SUMO-ACTIVATING ENZYME1 INHIBITOR

Oncology

Study

NCT04381650

NCT04776018

Indication

Solid tumors

Multiple Myeloma

Phase

Phase Ib/II

Phase Ib/II# of Patients

N = 101

N= 81

Target Patients

Patients with select advanced or metastatic solid tumors

Patients with relapsed and/or refractory multiple myeloma

Arms/Intervention• Escalating doses of TAK-981 with starting dose of 40
mg, intravenous (IV) infusion, on Days 1, 4, 8 and 11 ineach 21-day treatment cycle and pembrolizumab 200
mg, IV infusion, as a fixed dose every 3 weeks in 21-day
treatment cycle until RP2D is determined (for a
maximum of 24 months).
• TAK-981 at RP2D as IV infusion on Days 1, 4, 8 and 11 ineach 21-day treatment cycle up to disease progression
or 12-months and pembrolizumab 200 mg IV infusion
as a fixed dose every 3 weeks in 21-day treatment cycle
for a maximum of 24 months.

• Phase 1b: Dose escalation of TAK-981 in combination with fixed doses ofmezagitamab or daratumumab and hyaluronidase-fihj, respectively inpatients with RRMM.
• In Phase 1b each 28-day treatment cycle will consist of TAK-981
administered IV in one of the following schedules:• BIW on Days 1, 4, 8, 11, and 15 during Cycles 1 and 2, then once
every 2 weeks during Cycles 3 through 6, followed by monthly
dosing, OR• QW on Days 1, 8, 15, 22 during Cycles 1 and 2, then once every 2
weeks during Cycles 3 through 6, followed by monthly dosing
thereafter until PD.
• Phase 2: Explore the efficacy and safety of TAK-981 in combination withan anti-CD38 antibody (mezagitamab or daratumumab and hyaluronidasefihj) in patients with RRMM. A schedule will be selected for continued
evaluation based on data from Phase 1b,

Primary endpoint and key
secondary endpoint(s)Safety and tolerability

Safety, tolerability and RP2D

Status• Study start date: August 2020
• Estimate primary completion date: October 2022

• Study start date: April 2021
• Estimated primary completion date: October 2024

11 | 1.

SUMO = Small ubiquitin-like modifierTAK-605: ONCOLYTIC VIRUS ENCODING TRANSGENES FOR FLT3
LIGAND, ANTI-CTLA-4 ANTIBODY, AND IL-12 CYTOKINE

Oncology

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 administered alone by direct injection into tumor(s) x 4. Booster injections of TBio-6517 are
permitted for up to 24 months.
• Arm B: TBio-6517 and pembrolizumab Dose escalation of TBio-6517 administered in combination with pembrolizumab. TBio-6517 will
be directly injected into tumor(s) x 4. Booster injections of TBio-6517 are permitted for up to 24 months. Pembrolizumab will beadministered beginning at Day 8 via intravenous (IV) infusion every 3 weeks for up to 24 months.
• TBio-6517 and pembrolizumab in MSS-CRC Doses of TBio-6517 will be administered by direct injection into tumor(s) x 4 in combination
with pembrolizumab beginning at Day 8 given every 3 weeks for up to 24 months in patients with microsatellite stable colorectal
carcinoma (MSS-CRC). Booster injections of TBio-6517 are permitted for up to 24 months.
• TBio-6517 and pembrolizumab in TNBC Doses of TBio-6517 will be administered by direct injection into tumor(s) x 4 in combination withpembrolizumab beginning at Day 8 given every 3 weeks for up to 24 months in patients with triple negative breast cancer (TNBC).
Booster injections of TBio-6517 are permitted for up to 24 months.

Primary endpoint and key
secondary endpoint(s)Recommended Phase 2 dose (RP2D)

Status• Study start date: August 2020

12 | 1.

Trial managed by Turnstone Biologics

TAK-573: FIRST-IN-CLASS ANTI-CD38/ATTENUATED
IFNa FUSION PROTEIN

13 |Oncology

Study

NCT04157517

NCT03215030

Indication

Solid tumors

Relapsed/refractory multiple myeloma

Phase

Phase I/II

Phase I/II

# of Patients

N = 143

N = 151

Target Patients

Patients with locally advanced or metastatic solid tumors

Patients with relapsed/refractory multiple myeloma

Arms/Intervention• TAK-573 0.1 to 6 milligram per kilogram (mg/kg), infusion,
intravenously, once on Day 1 of each 21-days treatment cycle for up
to 1 year.
• Phase 2 Dose Expansion in combination with pembrolizumab:➢ Unresectable/metastatic cutaneous melanoma withprimary resistance or acquired resistance to no more than 2prior lines of anti-PD1 containing treatments.
➢ Unresectable/metastatic cutaneous melanoma naïve toprior anti-PD1 containing treatments.

• Phase 1 cohort: TAK-573 0.001 to 14 milligram per kilogram(mg/kg), infusion, intravenously, once on Days 1, 8, 15 and 22 ofeach 28-day treatment cycle up to 1 year.
• Phase 2 cohort: TAK-573 TBD as a single agent. Participants in atleast 1 cohort will receive TAK-573 TBD and dexamethasone 40 mg,
orally, once weekly of each 28-day treatment cycle until treatment
discontinuation.

Primary endpoint and key
secondary endpoint(s)Safety and tolerability

Safety and tolerability

Status• Study start date: December 2019
• Estimated primary completion date: Q3 2023

• Study start date: October 2017
• Estimated primary completion date: Oct 2021

TAK-676: STING AGONIST

14 |Oncology

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 21-day treatment cycles, andcapping at 2.5 mg IV on Days 1, 8 and 15 in a 21-day cycle.
• Arm 2: Dose escalating TAK-676 along the above parameters in combination with fixed dose pembrolizumab at 200 mg IV administeredon D1 in a 21-day cycle.

Primary endpoint and key
secondary endpoint(s)• Primary endpoints: Safety and tolerability• Secondary objectives: Recommended Phase 2 dose (RP2D), overall response rate (ORR)

Status• Study start date: August 2020

TAK-252: PD1-FC OX40L ARC

Oncology

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• Escalating doses of TAK-252 (SL-279252) with starting dose of 0.0001 mg/kg, intravenous (IV) infusion, on Days 1, 8, and 15 in the first28-day treatment cycle, followed by IV infusion on D1 and D15 of each 28-day cycle.
• Escalating doses of TAK-252 (SL-279252) with starting dose of 0.3 mg/kg, intravenous (IV) infusion, administered once weekly on Days
1, 8, 15 and 22 of each 28-day treatment cycle.

Primary endpoint and key
secondary endpoint(s)Safety, maximum tolerated dose (MTD).
Recommended Phase 2 dose (RP2D), preliminary antitumor activity by iRECIST, immunogenicity and PK characterization of TAK-252

Status• Study start date: March 2019
• Estimated primary completion date: February 2022

15 | 1.

Trial managed by Shattuck LabsTAK-102: GPC3 CAR-T
Study

NCT044057781

Indication

Solid tumors

Phase

Phase I

# of Patients

N = 18Target 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• Cohort 3: 1 × 10^9 CAR (+) cells/bodyPrimary endpoint and key
secondary endpoint(s)• Primary endpoint: Incidence of dose-limiting toxicities, treatment-emergent adverse events (AEs) and AEs of clinical interest• Primary objective: To evaluate the safety and tolerability of TAK-102 and to determine the recommended Phase 2 dose of TAK-102

Status• Study start date: July 202016 | 1.

In collaboration with Noile-Immune Biotech Inc. Enrollment in Japan.

OncologyTAK-940: CD19 CAR-T

Oncology

Study

NCT044642001

Indication

Relapsed/refractory B-cell cancers

Phase

Phase I

# of Patients

N = 30Target Patients

Adult patients with relapsed or refractory CD19+ B lymphoid malignancies

Arms/Intervention• 19(T2)28z1xx CAR T cells Cohorts of 3-6 patients will be infused with escalating doses of 19(T2)28z1xx CAR T cells to establish the RP2D.
There are 4 planned flat-dose levels: 25x10^6, 50 x 10^6, 100 x 10^6, and 200 x 10^6 CAR T cells and one de-escalation dose: 12.5 x
10^6 CAR T cells. A standard 3+3 dose escalation design will be implemented starting from dose 1.

Primary endpoint and key
secondary endpoint(s)Primary: Safety and Recommended Phase 2 dose (RP2D)
Secondary: Efficacy and CKStatus• Study start date: August 202017 | 1.

Trial managed by Memorial Sloan Kettering Cancer CenterTAK-186: T-CELL ENGAGER
Study

NCT048440731

Indication

Solid tumors

Phase

Phase I/II

# of Patients

N = 68Target Patients

Patients with unresectable, locally advanced or metastatic cancer

Oncology

Single-arm, open label, MVC-101 - An EGFR x CD3 COnditional Bispecific Redirected Activation (COBRA™) Protein

Arms/Intervention

This Phase 1/2, open-label study will characterize safety, dose-limiting toxicities (DLTs), and maximum tolerated/ recommended phase 2
dose (MTD/RP2D) of MVC-101.
Dose escalation will occur in a 1+3 and then 3+3 design in patients with advanced solid tumors. Once the MTD/RP2D is determined, aCohort Expansion Phase will be enrolled to further characterize safety and initial antitumor activity in patients with HNSCC, CRC or NSCLC.
Primary Endpoint: Safety based upon incidence of treatment-emergent adverse events.

Primary endpoint and key
secondary endpoint(s)Status18 | 1.

Trial managed by Maverick Therapeutics

Secondary Endpoints: Pharmacokinetics, Pharmacodynamics, Immunogenicity measured by plasma anti-drug antibodies, andRadiographic anti-tumor activity• Study start date: March 17, 2021

OVERVIEW OF CLINICAL TRIAL SUMMARYONCOLOGY

19 |2

RARE GENETIC & HEMATOLOGY 19

PLASMA-DERIVED THERAPIES

47

NEUROSCIENCE

30

VACCINES

52

GASTROENTEROLOGY (GI)37

ADYNOVATE (TAK-660): RECOMBINANT, PEGYLATEDANTIHEMOPHILIC FACTOR

20 |Rare Genetic and Hematology

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 determine safety including immunogenicity of Adynovate (TAK-660/BAX 855) based on the incidence of
inhibitor development to FVIII (≥ 0.6 Bethesda unit (BU)/mL using the Nijmegen modification of the Bethesda assay).
Safety1. To determine the immunogenicity of Adynovate in terms of binding IgG and IGM antibodies to FVIII, PEG-FVIII and PEG
2. To determine the safety of Adynovate based on adverse events (AEs) and serious adverse events (SAEs)
Hemostatic Efficacy
3. To assess the efficacy of prophylactic treatment with Adynovate
4. To characterize the efficacy of Adynovate in the control of bleeding episodes
Pharmacokinetics
6. To determine the incremental recovery (IR) of Adynovate at baseline and over time
7. To determine half-life of Adynovate at baseline (optional)

Status• Study start date: November 2015
• Final report expected Q1 2025

VONVENDI (TAK-577):
RECOMBINANT VON WILLEBRAND FACTOR

21 |Rare Genetic and Hematology

Study

NCT02973087

NCT02932618

Indication

Adult Prophylaxis

Pediatric On-demand and Elective Surgery

Phase

Phase III

Phase III# of Patients

N = 22N = 27 (On-demand)
N = 12 (Elective Surgery)

Target Patients

Severe von Willebrand Disease

Severe von Willebrand Disease

Arms/Intervention• Arm A: Transitioning from on-demand• Arm B: Switching from prophylactic treatment with pdVWF• Arm A: On-demand• Arm B: Elective and emergency surgery

Primary endpoint and key
secondary endpoint(s)• Annualized Bleed Rate (ABR) - comparing subject’s historical andon-study ABR for spontaneous bleeding episodes• Key secondary endpoint: Safety and additional efficacy measuresof prophylactic treatment• Hemostatic efficacy and safety of rVWF, with or without ADVATE, inthe treatment and control of nonsurgical bleeding events• Key secondary endpoint: Hemostatic efficacy assessed after thelast perioperative rVWF infusion

Status• Topline data available• Study start date: October 2016
• Estimated primary completion date: FY22

TAKHZYRO (LANADELUMAB):
PLASMA KALLIKREIN (PKAL) INHIBITOR

22 |Rare Genetic and Hematology

Study

NCT04070326

NCT04206605

Indication

Hereditary angioedema (HAE) pediatricNon-histaminergic angioedemawith normal C1-Inhibitor

Phase

Phase IIISPRING

Phase IIICASPIAN# of Patients

N = 20N = 75

Target Patients

Type I and Type II hereditary angioedema, ages 2 to <12 yo

Non-histaminergic bradykinin-mediated angioedema (BMA) withnormal C1-inhibitor

Arms/Intervention• Lanadelumab 150mg; q4wks ages 2 to < 6, q2wks ages 6 to <12 yo• Lanadelumab 300mg q2wks

Primary endpoint and key
secondary endpoint(s)• Primary: Safety and pharmacokinetics• Key secondary: Clinical outcomes, pharmacodynamics• Primary: Number of investigator-confirmed angioedema attacks
during the treatment period of Day 0 through Day 182• Key secondary: Number of participants achieving attack-freestatus during the treatment period of Day 0 through Day 182

Status• Study start date: August 2019
• Estimated primary completion date: FY22

• Study start date: August 2020
• Estimated primary completion date: FY23

MARIBAVIR (TAK-620):
ORAL VIRAL PROTEIN KINASE INHIBITOR

23 |Rare Genetic and Hematology

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 = 351

N = 550

Target Patients

Treatment of CMV infection refractory or resistant to ganciclovir,
valganciclovir, cidofovir or foscarnet in solid organ transplant (SOT)and stem cell transplant patients

Treatment of asymptomatic CMV infection in stem cell transplantpatients

Arms/Intervention

Arm A: Maribavir
Arm B: Investigator-assigned treatment

Arm A: Maribavir
Arm B: Valganciclovir

Primary endpoint and key
secondary endpoint(s)• Primary: Confirmed clearance of plasma CMV DNA (CMV viremia
clearance) at the end of Study Week 8• Secondary: Achievement of CMV viremia clearance and resolution
or improvement of tissue invasive CMV disease or CMV syndrome
for subjects symptomatic at baseline or achievement of clearanceof viremia and no symptoms of tissue invasive CMV disease or
CMV syndrome for subjects asymptomatic at baseline at the end of
Study Week 8, followed by maintenance of this treatment effectfor an additional 8 weeks off treatment• Primary: Confirmed clearance of plasma CMV DNA (CMV viremia
clearance) at the end of Study Week 8• Secondary: Maintenance of confirmed CMV viremia clearance
achieved at the end of Study Week 8 through Week 16 having
received exclusively a study-assigned treatment.

Status• Study start date: December 2016
• Actual primary completion date: Q3FY20• Study met its primary and secondary endpoint.
• Regulatory discussions ongoing for global filings.
• Phase 2: Papanicolaou GA, et al. Clin Infect Dis. 2019 Apr
8;68(8):1255-1264.

• Study start date: April 2017
• Estimated primary completion date: FY21
• Phase 2: Maertens J, et al. N. Engl J Med 2019;381:1136-47.

TAK-755: REPLACEMENT OF THE
DEFICIENT-ADAMTS13 ENZYME

24 |Rare Genetic and Hematology

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 = up to 68

N = 30N = 56

Target Patients

Patients diagnosed with severe cTTP
in prophylactic and on-demand treatment

Adult patients diagnosed with iTTP

Adult patients with sickle cell disease at baseline
health and during acute vaso-occlusive crisis(VOC)

Arms/Intervention

Prophylaxis Treatment Cohort: 6 + 6 months
cross over of TAK-755 vs SoC followed by 6months TAK-755 extension• Arm 1: TAK-755 followed by SOC• Arm 2: SOC followed by TAK-755(Patients are also eligible to enter theprophylaxis study upon completion of acutetreatment)• Arm 1: TAK-755 High dose + SOC• Arm 2: TAK-755 Low dose + SOC• Arm 3: Placebo + SOC• Part A: TAK-755 (three dose levels) or placebo
administered at baseline healthPrimary endpoint
and key secondary
endpoint(s)Incidence of acute TTP episodes in subjects
receiving prophylactic treatment with either
TAK-755 or SoC

ADAMTS-13 activity, ADAMTS-13 binding andinhibitory antibodies, Platelet count, and LDH
levelsSAEs/AEs, changes in vital signs and laboratory
parameters, and incidence of binding andinhibitory antibodies to ADAMTS-13

Status• Study start date: October 2017
• Estimated primary completion date: FY22

• Study start date: October 2019
• Estimated primary completion date: FY21

• Study start date: October 2019

TAK-611: RHASA1 ENZYME REPLACEMENTTHERAPY FOR MLD, INTRATHECAL (IT)Rare Genetic and Hematology

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 = 23

N = 42

Target PatientsChildren with Metachromatic Leukodystrophy (MLD)

Late Infantile Metachromatic Leukodystrophy (MLD)

Arms/Intervention

Open Label with 4 Cohorts:• Cohort 1 – 10 mg dose level• Cohort 2 – 30 mg dose level• Cohort 3 – 100 mg dose level• Cohort 4 – 100 mg dose level (Process B)

Open Label with 6 Groups:• Group A - GMFC-MLD level of 1 or 2
• Group B - GMFC-MLD level of 3
• Group C - GMFC-MLD level of 4
• Group D - younger siblings of enrolled subjects, and have the same
ASA allelic constitution• Group E - GMFC-MLD level of 1 or 2 ( ≥12 to <18 mons of age)
• Group F - GMFC-MLD level of 5 or 6

Primary endpoint
and key secondary
endpoint(s)Primary - Safety will be measured by the following endpoints:• Reporting of treatment-emergent adverse events (TEAEs)• Change from baseline in clinical laboratory testing (serum chemistry
including liver function tests, hematology, and urinalysis)• Change from baseline in vital signs, physical examinations, and CSF
chemistry (including cell counts, glucose, albumin, and protein)• Determination of the presence of anti-HGT-1110 antibodies in CSF and/or
serum

Primary - The primary efficacy endpoint is response in Group A,
defined as maintenance of gross motor function at 2 years (Week106), evaluated as no greater than 2 levels decline from baseline in
GMFC-MLD. If suitable controls cannot be matched despite thesponsor’s best efforts, change from baseline results of GMFC-MLD at
Week 106 may be compared with a prespecified objective thresholdto evaluate primary efficacy for this study.

Status• Study start date: May 2013

• Study start date: May 2019
• Estimated primary completion date: FY23

25 | 1.

rhASA = recombinant human arylsulfatase A

TAK-609: CNS REPLACEMENT OF THE
DEFICIENT-IDS1 ENZYME, INTRATHECAL (IT)Rare Genetic and Hematology

Study

NCT01506141

NCT02412787

Indication

Hunter Syndrome with Cognitive Impairment

Hunter Syndrome with Cognitive Impairment

Phase

Phase I/II (extension of HGT-HIT-045)
HGT-HIT-046

Phase II/III (extension of HGT-HIT-094)
SHP609-302# of Patients

N = 14

N = 56 (including sub-study)

Target Patients

Pediatric participants that completed HGT-HIT-045 with Hunter
syndrome and cognitive Impairment

Pediatric participants that completed study HGT-HIT-094 to continue
receiving Elaprase treatment in conjunction with IdS IT or to continue
receiving Elaprase treatment and begin concurrent IT treatment for
those that did not receive IdS IT treatment in study HGT-HIT–094.

Arms/Intervention

All 56 participants will receive 10 mg of IdS IT once every 28 days.
All participants will receive Idursulfase-IT once monthly at the dose
Participants who are younger than 3 years of age will receive anused in study HGT-HIT-045 via intrathecal drug delivery device (IDDD). adjusted dose of 7.5 mg (>8 months to 30 months of age) and 10 mg(>30 months to 3 years of age).

Primary endpoint and key
secondary endpoint(s)Extension study of HGT-HIT-045 evaluating long-term safety andclinical outcomes of intrathecal idursulfase in conjunction withintravenous Elaprase

An open label extension of study HGT-HIT-094 evaluating long term
safety and clinical outcomes of intrathecal idursulfase administered inconjunction with Elaprase

Status• Study start date: August 2010, recruitment completed
Publication:• Muenzer J, et al. Genet. Med. 2016 Jan; 18(1):73-81.

• Study start date: October 2015, recruitment completed

26 | 1.

IdS = iduronate-2-sulfatase

MEZAGITAMAB (TAK-079): ANTI-CD38 ANTIBODY
Study

NCT04278924

NCT04159805

Indication

Persistent/Chronic Primary
Immune 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 years of age with generalized Myasthenia Gravis•

Arms/Intervention

27 |Rare Genetic and Hematology•

Part A: 2 dose groups and placebo added to stable backgroundtherapy• Arm A1: Matching placebo (n = 12 patients)• Arm A2: TAK-079 100 mg (n = 12 patients)• Arm A3: TAK-079 300 mg (n = 12 patients)
Part B: Following interim analysis. 1 dose group and placebo (600mg) added to stable, standard background therapy.
• Arm B1: Matching placebo (n = 6 patients)• Arm B2: TAK-079 600 mg (n = 12 patients)• 2 dose groups and placebo added to stable background therapy• TAK-079 300 mg (n = 12 patients)• TAK-079 600 mg (n = 12 patients)• Matching placebo (n = 12 patients)

Primary endpoint and key
secondary endpoint(s)The primary endpoint is the percentage of patients with TEAEs
including Grade 3 or higher events, SAEs, and AEs leading to TAK-079
discontinuation.

The primary endpoint is the percentage of patients with TEAEs
including Grade 3 or higher events, SAEs, and AEs leading to TAK-079
discontinuation.

Status• Estimated study start date: November 2020

Study start date: January 2020

MECASERMIN RINFABATE (TAK-607): REPLENISHESINSULIN LIKE GROWTH FACTOR-1, IV

28 |Rare Genetic and Hematology

Study

NCT03253263

Indication

Preventing Chronic Lung Disease in 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 infusion of SHP607 250 μg/kg/24 hours• ~159 subjects randomized toto continuous IV infusion of SHP607 400 μg/kg/24 hours• ~159 subjects randomized to standard neonatal care

Primary endpoint and key
secondary endpoint(s)Time to final weaning off respiratory technology support (RTS) from Day 1 (i.e., randomization) through 12 months corrected age (CA),Incidence of Bronchopulmonary Dysplasia (BPD) or Death through Postmenstrual age (PMA) 36 Weeks

Status• Study start date: May 2019

OBIZUR (TAK-672): RECOMBINANT PORCINE FACTOR
VIII

29 |

Study

NCT04580407

Indication

Acquired Hemophilia A

Phase

Phase II/III# of Patients

N=5

Target PatientsJapanese subjects ≥18 years of age with AHA

Arms/Intervention• Single group assignment

Primary endpoint and key
secondary endpoint(s)The primary objective is to evaluate the efficacy and safety of TAK-672 for the treatment of serious bleeding
events in Japanese subjects with AHA.

Status• Estimated study start date: May 2021

Rare Genetic and Hematology

OVERVIEW OF CLINICAL TRIAL SUMMARYONCOLOGY

30 |2

RARE GENETIC & HEMATOLOGY 19

PLASMA-DERIVED THERAPIES

47

NEUROSCIENCE

30

VACCINES

52

GASTROENTEROLOGY (GI)37

Neuroscience

TAK-994: OREXIN 2R AGONIST, ORAL

31 |

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 = 18Target Patients

Patients with Narcolepsy Type 1 (with cataplexy, NT1) or Narcolepsy
Type 2 (without cataplexy, NT2)

Healthy male participants

Arms/Intervention• Part A: Patients with NT1 treated for 28 days (TAK-994 dose 1 orplacebo in 2:1 ratio). Second cohort with dose 2 TBD.
• Part B: Dose ranging study in NT1 for 56 days (TAK-994 dose 1-3 orplacebo in 1:1:1:1 ratio)• Part C: China specific cohort in NT1 for 56 days (TAK-994 orplacebo in 2:1 ratio)• Part D: Patients with NT2 treated for 28 days (TAK-994 or placebo
in 2:1 ratio). Second cohort with dose 2 TBD.

Primary endpoint and key
secondary endpoint(s)• Maintenance of Wakefulness Test (MWT)• Epworth Sleepiness Scale (ESS)• Weekly Cataplexy Rate (WCR)• Maintenance of Wakefulness Test (MWT)• Safety, PK/PD

Status• Study start date: July 2020
• Part A completed, as reported at April 2021 Takeda WAVE 1 pipeline
investor call

• Study start date: September 2020
• Recruitment completed• Actual Primary Completion Date: December 2020

Randomization to 1 of 3 treatment sequences with a washout periodof at least 7 days in between each treatment period:• TAK-994 Dose A, Placebo, and TAK-994 Dose B• TAK-994 Dose B, TAK-994 Dose A, and Placebo• Placebo, TAK-994 Dose B, and TAK-994 Dose A

Neuroscience

TAK-925: OREXIN 2R AGONIST, IV

32 |

Study

NCT03332784

NCT03748979

Indication

Narcolepsy type 1

Narcolepsy type 1 andNarcolepsy type 2

Phase

Phase I

Phase I

# of Patients

N = 58N = 57

Target Patients

Patients with narcolepsy type 1 andhealthy volunteers

Patients with narcolepsy type 1,
patients with narcolepsy type 2 andhealthy volunteers

Arms/Intervention• Part 1: Healthy participants and healthy elderly participants• Part 2: Patients with narcolepsy type 1: TAK-925 5 mg, 11.2 mg,
44.8mg or placebo with cross-over• Part A: Healthy participants• Part B: TAK-925 (Dose Levels 11mg, 44mg ) vs. placebo in NT1
patients• Part C: TAK-925 (Dose Levels 44mg, 112mg ) vs. placebo in NT2
patients• Part A’: TAK-925 (Dose Levels 112mg) in healthy participants.

Primary endpoint and key
secondary endpoint(s)Sleep Latency in Maintenance of Wakefulness Test (MWT)
Karolinska Sleepiness Scale (KSS)

Sleep Latency in Maintenance of Wakefulness Test (MWT)
Epworth Sleepiness Scale (ESS)

Status• Study start date: November 2017
• Study primary completion date: September 2018
Publication:
https://www.professionalabstracts.com/ws2019/iPlanner/#/presenta
tion/1832• Study start date: November 2018
• Study primary completion date: October 2019
Publication:
https://onlinelibrary.wiley.com/toc/13652869/2020/29/S1

Neuroscience

TAK-925: OREXIN 2R AGONIST, IV

33 |

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 = 40Target Patients

Patients with obstructive sleep apnea who are experiencing excessive
daytime sleepiness despite adequate use of CPAP

Patients with Idiopathic Hypersomnia (IH)

Arms/Intervention• 3 period, 3 treatment crossover: TAK-925 High Dose, Low dose andplacebo• 2 period, 2 treatment crossover: TAK-925 and placebo

Primary endpoint and key
secondary endpoint(s)• Maintenance of Wakefulness Test (MWT)• Karolinska Sleepiness Scale (KSS)• Maintenance of Wakefulness Test (MWT)• Karolinska Sleepiness Scale (KSS)• Safety, PK/PD

Status• Study start date: November 2019
• Study primary completion date: April 2020
• Results in-house awaiting publication at a future conference• Study start date: January 2020
• Actual Primary Completion Date: November 2020

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 ≥3 convulsive or ≥4 drop
seizures, respectively, per month during the 3 months immediately prior to screening

Arms/Intervention• 51 DS subjects (1:1 soticlestat:placebo randomization ratio)• And 90 LGS subjects (1:1 soticlestat:placebo randomization ratio)
Primary: Percent change from baseline in seizure frequency (convulsive for DS and drop for LGS)

Primary endpoint and key
secondary endpoint(s)Status34 | 1.

In collaboration with Ovid Therapeutics Inc.

Key secondary endpoints:• Clinician’s Clinical Global Impression of Severity and Change• Caregiver Global Impression of Change (GI-C) responses• Plasma 24S-hydroxycholesterol (24HC) levels• Safety and tolerability endpoints• Study start date: August 2018
• Study completion date: July 2020
• Press release August 25, 2020: https://www.takeda.com/newsroom/newsreleases/2020/phase-2-elektra-study-of-soticlestat-tak935ov935-meets-primary-endpoint-reducing-seizure-frequency-in-children-with-dravet-syndrome-or-lennox-gastaut-syndrome/

NeuroscienceNeuroscience

TAK-3411: ALPHA-SYNUCLEIN ANTIBODY, IV
Study

NCT03272165

NCT04449484

Indication

Parkinson’s Disease

Parkinson’s Disease

Phase

Phase I

Phase I

# of Patients

N = 48N = 36

Target Patients

Healthy volunteers

Patients with Parkinson’s Disease

Arms/Intervention• TAK-341 (MEDI1341) IV at a single ascending dose
• Placebo IV

Three cohorts of 12 patients treated over 8 weeks with three 60
minute IV infusions• Dose A of TAK-341/MEDI1341 over 8 weeks, with 4 weeks
intervals• Dose A of TAK-341/MEDI1341 over 8 weeks, with 4 weeks
intervals• Matched placebo over 8 weeks, with 4 weeks intervals

Primary endpoint and key
secondary endpoint(s)• Safety and tolerability• Secondary endpoint: PK and PD (alpha-synuclein concentrations inplasma and CSF)

• Safety and tolerability

Status• Study start date: October 2017
• Recruitment Completed• Study start date: August 202035 | 1.

Trials managed by AstraZeneca

Neuroscience

TAK-071: M1 PAM, ORAL

36 |

Study

NCT04334317

Indication

Parkinson’s Disease

Phase

Phase II

# of Patients

N = 64

Target Patients

Parkinson's Disease patients with cognitive impairment and an elevated risk of falls

Arms/Intervention• Participants aged 40 to less than or equal to (<=) 65 years will be randomly assigned to one of the two treatment
sequences in a crossover design:• TAK-071 7.5 mg + Placebo• Placebo + TAK-071 7.5 mg• A sentinel cohort in healthy volunteers (n=10) will provide PK and safety data, to extend the enrollment topatients in older age groups.

Primary endpoint and key secondary
endpoint(s)• Primary: Change from Baseline in Gait Variability during a 2-minute Dual-Task Walking Test
• Key Secondary:• Change from Baseline in Global Cognition Profile• PK

Status• Study start date: October 21, 2020

OVERVIEW OF CLINICAL TRIAL SUMMARYONCOLOGY

37 |2

RARE GENETIC & HEMATOLOGY 19

PLASMA-DERIVED THERAPIES48

NEUROSCIENCE

30

VACCINES

54

GASTROENTEROLOGY (GI)38

ENTYVIO (VEDOLIZUMAB): GUT-SELECTIVE
ANTI-a4b7 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 undergoing allogeneic hematopoietic stem celltransplantation (Allo-HSCT) in the prophylaxis of intestinal
acute GvHD (aGvHD)

Patients with UC or CD who received vedolizumab SC in a prior vedolizumab SC
study – long-term open-label extension• Arm 1: Vedolizumab 300 mg at Days -1 (baseline), +13,
+41, +69, +97, +125, and +153
• Arm 2: Placebo at Days -1 (baseline), +13, +41, +69,
+97, +125, and +153

• Group A: Vedolizumab SC 108 mg Q2W - patients from studies VISIBLE 1(NCT02611830) and VISIBLE 2 (NCT02611817) who completed theMaintenance Period (Week 52) or were not randomized into Maintenance
Period and achieved response at Week 14 after having received a third
vedolizumab IV infusion at Week 6• Group B: Vedolizumab SC 108 mg QW - patients from studies VISIBLE 1 andVISIBLE 2 who withdrew early from the Maintenance Period due to treatment
failure or patients from current study who enrolled on Q2W dosing but
experienced treatment failure while on study and were dose escalated to QW
dosing.

Primary endpoint and key
secondary endpoint(s)Intestinal aGvHD-free survival by Day +180 after Allo-HSCT

Percentage of participants with study drug related treatment emergent adverse
events (AEs) and serious AEs
Key secondary endpoints: long term clinical response and remission rates for UC
and CD

Status• Study start date: February 2019
• Estimated primary completion date: FY22

• Study start date: April 2016

Arms/Intervention

38 |GASTROENTEROLOGYENTYVIO (VEDOLIZUMAB): GUT-SELECTIVE
ANTI-a4b7 INTEGRIN MAB

39 |GASTROENTEROLOGY

Study

NCT03196427

Indication

Ulcerative Colitis or Crohn’s disease
in pediatric patients IV

Phase

Phase II(Long-term safety study)# of Patients

N = 90Target Patients

Pediatric patients with Ulcerative Colitis or Crohn’s disease between 2 to 17 years old at the time of randomization for Study NCT03138655.

Arms/Intervention• Arm 1 ( ≥30 kg weight cohort): Vedolizumab 300 mg or 200 mg (Q8W)• Arm 2 ( <30 kg weight cohort): Vedolizumab 150 mg or 100 mg (Q8W)

Primary endpoint and key
secondary endpoint(s)Percentage of participants with Treatment-Emergent Adverse Events (TEAEs)

Status• Phase 2 start date: July 2018
• Study completion date: May 2020
• Pediatric Phase 3 to start 2021

ALOFISEL/CX601 (DARVADSTROCEL): ALLOGENEIC EXPANDEDADIPOSE-DERIVED STEM CELLS (ASC)40 |GASTROENTEROLOGY

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
immunosuppressants, anti TNF, ustekinumab

Arms/Intervention• Arm 1: Cx601, adult allogeneic expanded adipose-derived stem cells (eASC 120 million cells (5 million cells per milliliter)) administered onceby intralesional injection• Arm 2: Placebo-matching eASCs cells administered once by intralesional administration

Primary endpoint and key
secondary endpoint(s)Primary: Combined Remission, defined as:• The clinical assessment of closure of all treated external openings at week 24, and• Absence of collections >2 cm (in at least 2 dimensions) confirmed by blinded central MRI assessment at Week 24.
Key Secondary:• Clinical Remission at weeks 24 and 52• Time to Clinical Remission at weeks 24 and 52

Status• Study start date: September 2017
• Estimated primary completion date: FY22

VONOPRAZAN: POTASSIUM-COMPETITIVE ACID BLOCKER, ORAL

41 |

Study

NCT04198363

Indication

Acid related disease (adjunct to Helicobacter pylori eradication)

Phase

Phase III
China

# of Patients

N = 510Target Patients

Helicobacter pylori (HP)-positive participants who require HP eradication

Arms/Intervention• Experimental: Vonoprazan 20 mg in combination with bismuth containing quadruple therapy• Active Comparator: Esomeprazole 20 mg in combination with bismuth containing quadruple therapy

Primary endpoint and key
secondary endpoint(s)Percentage of Helicobacter pylori positive (HP+) participants with successful HP eradication at week 4 post-treatment

Status• Study start date: April 2020
• Estimated primary completion date: FY21

GASTROENTEROLOGYTAK-721: GLUCOCORTICOSTEROID, ORAL

42 |GASTROENTEROLOGY

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) (0.2 milligrams/mL) 2mg twice daily

Primary endpoint and key
secondary endpoint(s)To evaluate the long-term safety and tolerability of budesonide oral suspension･# of participants with treatment-emergent adverse events (TEAEs)･# of participants with clinically relevant changes in physical examinations, vital signs and clinical laboratory assessments･Change from baseline in bone mineral density (BMD) for adolescents assessed by dual-energy x-ray absorptiometry (DXA) scan
･Change from baseline in adrenocorticotropic hormone (ACTH) stimulation level

Status• Study start date: October 2017
• Estimated Study Completion Date: October 2023

GASTROENTEROLOGY

TAK-951: PEPTIDE AGONIST, SC

43 |

Study

NCT04486950

NCT04557189

Indication

Nausea & Vomiting

Nausea & Vomiting

Phase

Phase I

Phase IIa

# of Patients

N = 40N = 100

Target Patients

Healthy participants

Surgical patients under general anesthesia with 3 or more Apfel risk factors

Arms/Intervention• Cohort 1: TAK-951 20 mcg or matching placebo
infusion (intravenous (IV)) over 60 minutes• Cohort 2: TAK-951 (dose TBD) or matching placebo
infusion (IV) over 60 minutes• Cohort 3: TAK-951 (dose TBD) or matching placebo
infusion (IV) < 60 minutes• Group A: Ondansetron placebo-matching intravenous (IV) injection, once
immediately before induction of anesthesia and prophylaxis followed by TAK951 4 mg subcutaneous (SC) injection once 30 to 45 mins before the end of
surgery;• Group B: Ondansetron IV 4 mg once immediately before induction of anesthesia
followed by TAK-951 placebo-matching injection SC administered 30 to 45
minutes before the end of surgery

Primary endpoint and key
secondary endpoint(s)Safety and tolerability of IV administered TAK-951 in
healthy participantsComplete response in the immediate postoperative period (time frame: 6 hours
post surgery)
Percentage of participants with complete response, defined as no emesis (vomiting
or retching) and no need for rescue therapy (indicated if vomiting/retching and/or
nausea score ≥4 or upon participant's request), will be reported.
The severity of nausea will be scored using a self-reported, 11-point numerical
Verbal Rating Scale (VRS), where 0 represents "no nausea" and 10 represents the"worst nausea possible." Significant nausea is defined as a VRS score ≥4

Status• Study start date: July 2020

• Study start date: October 2020

TAK-510: PEPTIDE AGONIST, SC

44 |GASTROENTEROLOGY

Study

NCT04731922

Indication

Nausea & Vomiting

Phase

Phase I

# of Patients

N = 160

Target Patients

Healthy participants

Arms/Intervention• Part 1 (Cohort 1-12): TAK-510 single rising dose
• Part 2 (Cohort 13-17): TAK-510 multiple rising dose
• Part 3 (Cohort 18-20): TAK-510 dose titration and redosing cohorts

Primary endpoint and key secondary
endpoint(s)Safety and tolerability, pharmacokinetic, and immunogenicity of SC administered TAK-510 in healthy
participants

Status• Study start date: Feb 2021

TAK-906: DOPAMINE D2/D3 RECEPTOR ANTAGONIST, ORAL

45 |GASTROENTEROLOGY

Study

NCT03544229

Indication

Gastroparesis

Phase

Phase II

# of Patients

N = 205

Target Patients

Patients who have symptomatic idiopathic or diabetic gastroparesis.

Arms/Intervention

•
•
•
•

Primary endpoint and key
secondary endpoint(s)To assess the efficacy of treatment with 2 dose levels of TAK-906 in adult subjects with gastroparesis compared with placebo during 12 weeks
of treatment

Status• Study start date: October 2018; recruitment completed: March 2021
• Estimated Study Completion Date: July 2021

TAK-906 5 mg capsule BID: approximately 25 subjects prior to discontinuation of randomization into this dose arm
TAK-906 25 mg capsule BID: n = 60
TAK-906 50 mg capsule BID: n = 60
Placebo capsule BID: n = 60

TAK-954: 5-HT4-HYDROXYTRYPTAMINE RECEPTOR AGONIST, IV

46 |GASTROENTEROLOGY

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 over 60 minutes) pre-operation and daily post-operation until return of upper and lower GI
function (ie, resolution of POGD) or for up to 10 days.
• Regimen 3: TAK-954 (0.5 mg/100 mL infusion over 60 minutes) pre-operation and daily post-operation until return of upper and lower GI
function or for up to 10 days.
• Regimen 5: TAK-954 (0.5 mg/100 mL infusion over 60 minutes) pre-operation and daily placebo infusions post-operation until return ofupper and lower GI function or for up to 10 days.

Primary endpoint and key
secondary endpoint(s)To assess the efficacy and safety of intravenous (IV) TAK-954 for accelerating the recovery of GI function post-surgery in patients undergoing
open or laparoscopic-assisted partial small- or large-bowel resection.

Status• Study start date: March 2018
• A blinded interim analysis was conducted in January of 2021 leading to the recommendation of an independent monitoring committee todrop two of the 5 arms (regimen 2 & 4). The study will continue with regimens 1, 3 & 5.

SIBOFIMLOC (TAK-018): FIMH ANTAGONIST, ORAL

47 |

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 0.30 g Low Dose BID for up to 26 weeks• Cohort 2: TAK-018 1.5 g High Dose BID for up to 26 weeks• PlaceboPrimary endpoint and key
secondary endpoint(s)% of participants with endoscopic recurrence of CD as assessed by Rutgeerts Grading Scale at Week 26

Status• Study start date: August 2020

GASTROENTEROLOGYOVERVIEW OF CLINICAL TRIAL SUMMARYONCOLOGY

48 |2

RARE GENETIC & HEMATOLOGY 19

PLASMA-DERIVED THERAPIES

45

NEUROSCIENCE

30

VACCINES

50GASTROENTEROLOGY (GI)38

HYQVIA (TAK-771): IMMUNE GLOBULIN INFUSION 10% (HUMAN)
WITH RECOMBINANT HUMAN HYALURONIDASE

49 |

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 Patients

Adult subjects with a confirmed diagnosis of CIDP and who haveremained on a stable dosing regimen of IV immunoglobulin G (IGIV)
therapy for at least 12 weeks prior to screening.

Adult subjects who have completed Epoch 1 of Study NCT02549170
without CIDP worsening.

Arms/Intervention• Epoch 1: SC Treatment Period – Double blind assignment of
HYQVIA/HyQvia or 0.25% albumin placebo solution with
rHuPH20 6 months or until relapse.
• Epoch 2: IV Treatment Period - Open-label phase providing IGIV
for subjects who meet relapse criteria during Epoch 1.

• Subjects remain on same dosing regimen they were administered in
Epoch 1 of study 161403 (1 to 2 g/kg body weight every 4 weeks).
The first infusion will be at the subject’s full dose; there will be no
ramp-up of dose.

Primary endpoint and key
secondary endpoint(s)To evaluate the efficacy of HYQVIA/HyQvia as a maintenancetherapy for CIDP to prevent relapse of neuromuscular disability andimpairment. Safety and tolerability.

To evaluate the long-term safety, tolerability, and immunogenicity of
HYQVIA/HyQvia.

Status• Study start date: April 2016
• Estimated primary completion date (LPO): December 2021

• Study start date: December 2016
• Estimated primary completion date (LPO): September 2023

PDT

HYQVIA (TAK-771): IMMUNE GLOBULIN INFUSION 10% (HUMAN) WITH
RECOMBINANT HUMAN HYALURONIDASE
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 immunodeficiency
diseases in the US

Pediatric subjects with primary immunodeficiency diseases in the EU

Arms/Intervention

Primary endpoint and key
secondary endpoint(s)Status50 |Single-Group:• Epoch 1: HyQvia SC dose and ramp up for all patients; up to 6
weeks duration; patients were previously treated with IVIG orother SC immunoglobulin• Epoch 2: HYQVIA treatment (final dosing); 1-3 years• For IV-pre-treated subjects: every three or four weeks,
depending on the subject’s previous IV dosing schedule.
• For SC-pre-treated subjects: every three or four weeks,at the discretion of investigator and subject.
• Epoch 3: Safety Follow-Up: up to 1 year, if needed
Primary: Efficacy - rate of acute serious bacterial infections per
participant per year.
Secondary: Safety, tolerability, immunogenicity, efficacy, PK,
health-related Quality of Life.

• Study start date: Oct 2017
• Estimated primary completion date (LPO): Original date - May
2023 (Team is evaluating acceleration options with potential forInterim Analysis as early as Q1 FY2021)

Single-Group:• Epoch 1: HyQvia SC dose and ramp up for patients previously nottreated with HyQvia• Epoch 2: HyQvia dose once every three or four weeks; 1-3 years• For IV-pretreated subjects: every three or four weeks,
depending on the subject’s previous IV dosing schedule.
• For SC-pretreated subjects: every three or four weeks, at thediscretion of investigator and subject• For HyQvia pre-treated subjects: No change in frequency ofadministration• Epoch 3: Safety Follow-Up: up to 1 year, if needed
Primary: Safety
Secondary: Tolerability, immunogenicity, efficacy, health-related Qualityof Life.

• Study start date: June 2017
• Primary completion date (LPO): Jan 2021

PDT

CUVITRU (TAK-664): IMMUNE GLOBULIN SUBCUTANEOUS (HUMAN),
20% SOLUTION (IGSC, 20%) IN JAPANESE SUBJECTS WITH PID
Study

NCT04346108, JapicCTI-205162

Indication

Primary Immunodeficiency Diseases (PIDD)

Phase

Phase III# of Patients

N = 16

Target PatientsJapanese Subjects with PIDD

Arms/Intervention• Epoch 1 (13 weeks): IGIV: IGIV will be administered via IV infusions every 3 or 4 weeks, as per local product label, at the same dose asduring pre-study period (equivalent to approximately 200 - 600 mg/kg BW at 3- or 4- week intervals).
• Epoch 2 (24 weeks): approximately 50 - 200 mg/kg of Immune Globulin Subcutaneous (Human), 20% Solution (IGSC, 20%), will beadministered subcutaneously once a week. The dose in Epoch 2 will be adjusted so that it is an equivalent weekly dose of the dose
administered in Epoch 1.
• Epoch 3 (12 weeks): approximately 100 - 400 mg/kg of Immune Globulin Subcutaneous (Human), 20% Solution (IGSC, 20%), will beadministered subcutaneously once every 2 weeks in a subset of 7 subjects. The dose in Epoch 3 will be twice the dose in Epoch 2.

Primary endpoint and key
secondary endpoint(s)1. To assess serum trough IgG concentrations following weekly administration of IGSC, 20% (Epoch 2) and serum trough IgG concentration
after biweekly administration of IGSC, 20% (Epoch 3), in Japanese subjects with PID.
2. To assess serum trough IgG concentrations following every 3-week or every 4-week administration of IGIV (Epoch 1) in Japanese subjects
with PID.
3. To characterize the pharmacokinetic (PK) profiles of IGSC, 20% in Japanese subjects with PID following weekly subcutaneous (SC)administration (Epoch 2).
4. To evaluate the safety and tolerability of IGSC, 20% (Epoch 2, Epoch 3) and of intravenous immunoglobulin (IGIV) (Epoch 1) in Japanese
subjects with PID.
5. To evaluate the efficacy of IGSC, 20% (Epoch 2, Epoch 3) and of IGIV (Epoch 1) in Japanese subjects with PID.
6. To assess quality of life aspects, treatment satisfaction, and treatment preference of Japanese subjects with PID (Epoch 1, Epoch 2, Epoch3).

51 | Status• Study start date: Aug 2020
• Estimated primary completion date (LPO): Jan 2022

PDT

GLASSIA (TAK-670): HUMAN ALPHA1-PROTEINASE INHIBITOR, IV

52 |

Study

NCT02525861

Indication

Chronic augmentation and maintenance therapy in adults with clinically evident emphysema due to severe congenital deficiency of Alpha1Proteinase Inhibitor (A1PI)

Phase

Phase III/IV# of Patients

N = 36

Target Patients

A1PI deficient subjects

Arms/Intervention• Arm 1: GLASSIA lot with particle loads representing the high end within the normal range observed in GLASSIA lots manufactured• Arm 2: GLASSIA lot with particle loads representing the low end within the normal range observed in GLASSIA lots manufactured

Primary endpoint and key
secondary endpoint(s)1. To evaluate the effectiveness of the use of 5-micron in-line filter on the safety and potential immunogenicity of GLASSIA.
2. To determine the effects of weekly IV augmentation therapy with GLASSIA at a dosage of 60 mg/kg BW on antigenic and functional A1PI
levels in epithelial lining fluid (ELF) in subjects with congenital A1PI deficiency.
3. To collect additional safety information for GLASSIA.

Status• Study start date: April 2016
• Primary completion date (LPO): July 2020

PDT

OVERVIEW OF CLINICAL TRIAL SUMMARYONCOLOGY

53 |2

RARE GENETIC & HEMATOLOGY 19

PLASMA-DERIVED THERAPIES48

NEUROSCIENCE

30

VACCINES

54

GASTROENTEROLOGY (GI)38

TAK-003: LIVE ATTENUATED TETRAVALENT VACCINEFOR PREVENTION OF DENGUE DISEASE
Study

NCT02747927

Indication

The prevention of dengue fever of any severity caused by any dengue virus serotype in individuals 4 years to 60 years of age​

Phase

Phase III
Tetravalent Immunization against Dengue Efficacy Study (TIDES)# of Patients

N = 20,100

Target Patients

Healthy children aged 4 to 16-year-old in dengue-endemic countries in Latin America and Asia

Arms/Intervention• Randomized 2:1 to receive either TAK-003 or placebo on Day 1 and Day 90

Primary endpoint and key
secondary endpoint(s)• Efficacy: Onset of protection 30 days post 2nd dose in all (seronegative and seropositive)• Primary endpoint: ≥70% efficacy against all symptomatic dengue fever caused by any strain• Secondary endpoints:− ≥70% efficacy individual strains− ≥60% efficacy in seronegatives• Safety:• Comparable to other live attenuated viral vaccines (e.g., MMR, YF, Varicella)• No disease enhancement in partially protected individuals

Status

54 |Vaccines• Study start date: September 2016
• Primary completion date: July 2018
• Estimated completion date: FY24/25 (following booster evaluation)• 24-month data presented November 2020 at American Society of Tropical Medicine and Hygiene Annual Meeting
Publication:• Biswal S, et al. N Engl J Med. 2019; 381:2009-2019.
• Biswal S, et al. Lancet. 2020; 395(10234):1423-1433.
• López-Medina E, et al. The Journal of Infectious Diseases. 2020. jiaa761, https://doi.org/10.1093/infdis/jiaa761 [epub ahead of print].

TAK-214: NOROVIRUS GI.1/GII.4 BIVALENT
VIRUS-LIKE PARTICLE VACCINE

55 |Vaccines

Study

NCT02669121

NCT03039790

Indication

For active immunization for the prevention of acutegastroenteritis caused by norovirus (NoV)

For active immunization for the prevention of acute gastroenteritis
caused by norovirus (NoV)

Phase

Phase II

Phase II

# of Patients

N = 4176

up to N = 575

Target Patients

Healthy adults (18 to 49 years of age)

Healthy adults >18 years who received at least one dose of NoV
GI.1/GII.4 Bivalent Virus-Like Particle Vaccine in previous studies NOR107, NOR-210 and NOR-204

Arms/Intervention• Arm 1: NoV 15µg GI.1/50µg GII.4 bivalent virus-like particle(VLP) vaccine, 0.5 mL intramuscularly (IM), once, on Day 1• Arm 2: NoV vaccine placebo-matching solution (0.9% sodium
chloride), 0.5 mL intramuscularly (IM), once, on Day 1• No NoV vaccine injection administered.
• Long-Term Immunogenicity Follow-up Trial of Adult and Elderly
Subjects (followed up to 5y post-primary vaccination).
• Vaccine formulation according to parent trials.

Primary endpoint and key
secondary endpoint(s)• Primary endpoint: Percentage of Participants with Moderate or
Severe Acute Gastroenteritis (AGE) Occurring >7 Days After
Dosing Due to GI.1 or GII.4 NoV Strains (excluding Co-infection
due to Salmonella, Shigella, or Campylobacter)• Key secondary: Percentage of Participants with Moderate or
Severe AGE Occurring >7 Days After Dosing Due to Any NoVStrains (including/excluding Co-infection) and Due to GI.1 orGII.4 NoV Strains (including Co-infection)• Primary endpoint: Geometric Mean Blocking Titers 50 percent (%)(GMBT50) of Anti-norovirus GI.1 VLP / GII.4 VLP Antibodies asmeasured by the histo-blood group antigen (HBGA) blocking assay.
• Secondary endpoint: Geometric Mean Titers (GMT) of Anti-norovirus
GI.1 VLP / GII.4 VLP Antibodies as measured by total immunoglobulin(pan-Ig) enzyme-linked immunoassay (ELISA).

Status• Study start date: February 2016
• Study primary completion date: June 2018
Publication:• Sherwood J, et al. Vaccine 2020; 38(41):6442-6449.

• Study start date: February 2017
• Estimated completion date: Q4 CY 2021

TAK-426: PURIFIED INACTIVATED ZIKA VIRUS VACCINE PIZV

56 |

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

•
•
•
•

Primary endpoint and key
secondary endpoint(s)Safety, immunogenicity and dose ranging study

Status• Study start date: November 2017
• Presentation at ASTHM 2019 (Htay Htay Han #215, #1948)• https://www.astmh.org/ASTMH/media/2019-Annual-Meeting/ASTMH-2019-Abstract-Book.pdf• Estimated completion data: August 2021

Placebo: TAK-426 placebo-matching injection, intramuscular, once on Days 1 and 29
Low Dose: PIZV 2 microgram (mcg) (PIZV 0.5 milliliter (mL), 2 mcg antigen, injection, intramuscular, once on Days 1 and 29)
Medium Dose: PIZV 5 mcg (PIZV 0.5 mL, 5 mcg antigen, injection, intramuscular, once on Days 1 and 29)
High Dose: PIZV 10 mcg (PIZV 0.5 mL, 10 mcg antigen, injection, intramuscular, once on Days 1 and 29)

Vaccines

TAK-919: MESSENGER RIBONUCLEIC ACID (mRNA) VACCINE

Vaccines

Moderna vaccine candidate, mRNA-1273
Study

NCT04677660

Indication

Prevention of infectious disease caused by Severe Acute Respiratory Syndrome Coronavirus-2 (SARS-CoV-2)

Phase

Phase I/II and filed (Mar 5, 2021)# of Patients

N = 200

Target Patients

Healthy Japanese male and female adults aged 20 years and older

Arms/Intervention• Participants will be randomized to either receive two doses of the vaccine candidate (150), or placebo (50), at Day 1
and Day 29• TAK-919 0.5 mL• Matching placebo• Immunogenicity will be measured at Day 1, 29, 43, 57, 209 and 394• The study will include 12-months safety follow-up after the second dosePrimary endpoint and key
secondary endpoint(s)Safety and Immunogenicity of 2 doses of TAK-919 given 28 days apart

StatusStart date: Jan 21, 2021
Estimated completion date: March 11, 2022

57 |

TAK-019: RECOMBINANT SPIKE PROTEIN NANOPARTICLE VACCINEWITH Matrix-M™ ADJUVANT

Vaccines

Novavax vaccine candidate, NVX-CoV2373
Study

NCT04712110

Indication

Prevention of infectious disease caused by Severe Acute Respiratory Syndrome Coronavirus-2 (SARS-CoV-2)

Phase

Phase I/II

# of Patients

N = 200

Target Patients

Healthy Japanese male and female adults aged 20 years and older

Arms/Intervention• Participants will be randomized to either receive two doses of the vaccine candidate (n=150), or placebo (n=50), at Day
1 and Day 21• TAK-019 0.5 mL• Matching placebo• Immunogenicity will be measured at Day 1, 22, 36, 50, 202 and 387• The study will include 12-months safety follow-up after the second dosePrimary endpoint and key
secondary endpoint(s)Safety and Immunogenicity of 2 doses of TAK-019 given 21 days apart

StatusStart date: February 24, 2021
Estimated completion date: April 4, 2022

58 |