TAK-007 / Takeda, UT MD Anderson Cancer Center - LARVOL DELTA

A Study of TAK-007 in Adults With Refractory Lupus Nephritis (LN) or Refractory Systemic Sclerosis (SSc) (clinicaltrials.gov) - P1 | N=0 | Withdrawn | Sponsor: Takeda | N=20 ➔ 0 | Trial completion date: Apr 2029 ➔ Sep 2030 | Not yet recruiting ➔ Withdrawn | Trial primary completion date: Apr 2029 ➔ Sep 2030

Enrollment change • Trial completion date • Trial primary completion date • Trial withdrawal • Glomerulonephritis • Immunology • Inflammatory Arthritis • Lupus • Lupus Nephritis • Nephrology • Scleroderma • Systemic Sclerosis

Allogeneic CAR-NK Therapy TAK-007 Achieves Responses in R/R B-NHL (CGTLive) - P2 | N=27 | NCT05020015 | Sponsor: Takeda | "Takeda’s TAK-007...has demonstrated safety and efficacy in a phase 2 clinical trial (NCT05020015) evaluating the therapy for the treatment of patients with relapsed/refractory (r/r) large B-cell lymphoma (LBCL) or indolent nonHodgkin lymphoma (iNHL).1 The data were presented by Justin M. Darrah...at the 66th American Society of Hematology (ASH) Annual Meeting, held December 7-10, 2024, in San Diego, California....The overall response rate (ORR) for the total group of 26 patients was 53.8%, with the complete response rate (CRR) being 30.8% and the partial response rate (PRR) being 23.1%. For patients with LBCL who received the high dose, the ORR was 50.0%, with a 21.4% CRR and a 28.6% PRR....Darrah pointed out that the responses seen in the trial were not particularly durable, however, with a median duration of response (DOR) of 2.6 months observed for the patients with LBCL and a DOR of 4.9 months observed for the patients with iNHL."

P2 data • Indolent Lymphoma • Large B Cell Lymphoma • Non-Hodgkin’s Lymphoma

Efficacy and Safety of TAK-007, Cord Blood-Derived CD19 CAR-NK Cells, in Adult Patients with Relapsed/Refractory (R/R) B-Cell Non-Hodgkin Lymphoma (NHL) (ASH 2024) - P2 | "Treatment included 3 consecutive days of IV lymphodepleting chemotherapy (LDC) with 300 mg/m2 cyclophosphamide and 30 mg/m2 fludarabine, followed by a single dose of TAK-007 >2 days later. TAK-007, an allogeneic off-the-shelf CD19 CAR-NK cell therapy, demonstrated early efficacy at 800M and a favorable safety profile in a heavily pretreated pt population, and may be a safer and simpler treatment option with a shorter turnaround time compared with autologous CD19-targeting CAR-T cells. The use of >1 dose of TAK-007 at 800M may further increase efficacy and durability of response."

Clinical • Anemia • B Cell Lymphoma • B Cell Non-Hodgkin Lymphoma • Bone Marrow Transplantation • Febrile Neutropenia • Indolent Lymphoma • Infectious Disease • Large B Cell Lymphoma • Leukopenia • Lymphoma • Neutropenia • Non-Hodgkin’s Lymphoma • Septic Shock • Thrombocytopenia

A Study of TAK-007 in Adults With Refractory Lupus Nephritis (LN) (clinicaltrials.gov) - P1 | N=20 | Not yet recruiting | Sponsor: Takeda | Initiation date: Nov 2024 ➔ May 2025

Trial initiation date • Glomerulonephritis • Immunology • Inflammatory Arthritis • Lupus • Lupus Nephritis • Nephrology

A Study of TAK-007 in Adults With Relapsed or Refractory (r/r) B-cell Non-Hodgkin Lymphoma (NHL) (clinicaltrials.gov) - P2 | N=27 | Active, not recruiting | Sponsor: Takeda | N=265 ➔ 27 | Recruiting ➔ Active, not recruiting

Enrollment change • Enrollment closed • Follicular Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • BCL2 • BCL6

A Study of TAK-007 in Adults With Refractory Lupus Nephritis (LN) (clinicaltrials.gov) - P1 | N=20 | Not yet recruiting | Sponsor: Takeda

New P1 trial • Glomerulonephritis • Immunology • Inflammatory Arthritis • Lupus • Lupus Nephritis • Nephrology

MD Anderson’s CAR/NK Therapy Shows Improved Safety Profile in B-Cell Malignancies (CGTLive) - P1/2 | N=49 | NCT03056339 | "Investigators from MD Anderson found that CAR19/IL-15 CBU-NK (CAR19/IL-15) cells had a similar efficacy profile and an improved safety profile when compared with autologous CAR19 T cells in patients with CD19+ B cell malignancies...Data from 37 patients in a phase 1/2 trial (NCT03056339) were published in Nature Medicine. Rezvani and colleagues found that CAR19/IL-15 was not associated with any notable toxicities such as cytokine release syndrome, neurotoxicity or graft-versus-host disease....Trial participants had an overall response (OR) rate of 48.6% at days 30 and 100, overall survival of 68% at 1 year, and progression-free survival of 32% at 1 year. Responders had higher levels and longer persistence of CAR-NK cells than nonresponders."

Cytokine release syndrome • P1/2 data • Hematological Malignancies • Oncology

A Study of TAK-007 in Adults With Relapsed or Refractory (r/r) B-cell Non-Hodgkin Lymphoma (NHL) (clinicaltrials.gov) - P2 | N=265 | Recruiting | Sponsor: Takeda | Trial completion date: Oct 2029 ➔ Jun 2030 | Trial primary completion date: Oct 2029 ➔ Oct 2025

Trial completion date • Trial primary completion date • Follicular Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • BCL2 • BCL6

A Study of TAK-007 in Adults With Relapsed or Refractory (r/r) B-cell Non-Hodgkin Lymphoma (NHL) (clinicaltrials.gov) - P2; N=242; Recruiting; Sponsor: Takeda; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open • Follicular Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • BCL2 • BCL6 • IL10 • IL1B • IL6 • TNFA

A Study of TAK-007 in Adults With Relapsed or Refractory (r/r) B-cell Non-Hodgkin Lymphoma (NHL) (clinicaltrials.gov) - P2; N=242; Not yet recruiting; Sponsor: Takeda

Clinical • New P2 trial • Follicular Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • BCL2 • BCL6 • IL10 • IL1B • IL6 • TNFA

Takeda Provides Pipeline Update and Shares Goal to Increase Revenue 50% by FY2030 at the 39th Annual J.P. Morgan Healthcare Conference (Takeda Press Release) - "In the ongoing phase 1/2 trial, CAR-NK therapy has not been associated with the severe cytokine release syndrome (CRS) or neurotoxicity observed with existing CAR-T cell therapies and has the potential to be administered in an outpatient setting...A TAK-007 pivotal study is expected to begin enrolling patients with relapsed or refractory B-cell malignancies in 2021."

Cytokine release syndrome • New trial • Trial status • Chronic Lymphocytic Leukemia • Hematological Malignancies • Non-Hodgkin’s Lymphoma • Oncology

Takeda Opens New R&D Cell Therapy Manufacturing Facility to Support Expansion of Next-Generation Clinical Programs (Takeda Press Release) - "Takeda Pharmaceutical Company Limited...announced the expansion of its cell therapy manufacturing capabilities with the opening of a new 24,000 square-foot R&D cell therapy manufacturing facility at its R&D headquarters in Boston...Takeda and MD Anderson are developing a potential best-in-class allogeneic cell therapy product (TAK-007), a Phase 1/2 CD19-targeted chimeric antigen receptor-directed natural killer (CAR-NK) cell therapy with potential for off-the-shelf use being studied in patients with relapsed or refractory non-Hodgkin’s lymphoma (NHL) and chronic lymphocytic leukemia (CLL). Two additional Phase 1 studies of Takeda cell therapy programs were also recently initiated: 19(T2)28z1xx CAR T cells (TAK-940)...to treat relapsed/refractory B-cell cancers, and a cytokine and chemokine armored CAR-T (TAK-102)....to treat GPC3-expressing previously treated solid tumors."

Clinical • Trial status • Chronic Lymphocytic Leukemia • Hematological Malignancies • Non-Hodgkin’s Lymphoma • Oncology • Solid Tumor

Takeda Announces FY2020 Q1 Results; Confirms Management Guidance & Raises Reported Operating Profit And Reported Net Profit for the Full Year (Businesswire) - "In Oncology, TAK-007 for the treatment of hematologic malignancies on an outpatient basis, with encouraging Phase 1/2 data and cohort expansion ongoing for a pivotal study planned for next year and potential approval in FY2023; Mobocertinib (TAK-788) has the potential to set a new standard of care for a subset of Non-Small Cell Lung Cancer (NSCLC) patients with EGFR exon 20 insertions; Pevonedistat (TAK-924) has the potential to be the first novel HR-MDS therapy in over a decade; anticipated Phase 3 PANTHER trial in HR-MDS readout in second half of FY2020; Reported revenue and underlying revenue growth rates for notable Q1 FY2020 contributors include: ALUNBRIG JPY 2.0 billion +26%; NINLARO JPY 22.9 billion +31%"

Clinical • P3 data • Regulatory • Sales • Hematological Malignancies • Lung Cancer • Myelodysplastic Syndrome • Non Small Cell Lung Cancer • Oncology