AZD0120 / AstraZeneca, Gracell - LARVOL DELTA

Safety and efficacy of AZD0120, a BCMA/CD19 dual-targeting CAR T-cell therapy, in relapsed/refractory multiple myeloma: Preliminary Results from the DURGA-1 Phase 1b/2 study (ASH 2025) - P1/2 | "Whileadvances such as BCMA-directed CAR T-cell therapy have improved outcomes, challenges persist,including disease relapse, treatment toxicities (CRS, ICANS, and non-ICANS neurotoxicities), and access.AZD0120 (formerly GC012F) is a first-in-class autologous BCMA/CD19 dual-targeting CAR T-cell therapyusing the FasTCAR rapid manufacturing platform that preserves the naive and central memory T-cellphenotypes with marked in vivo proliferative capacity...The median age was 64 y (range 44–78), median pLOT was 4 (range 3–7), 72% were triple-classrefractory, 20% had prior BCMA CAR T-cell therapy, 4% had prior teclistamab, 28% had high-riskcytogenetic features [del(13q), del(17p13), t(4; 14), t(14; 16), amp(1q)], and 8% had extramedullaryplasmacytomas...Median timeto CRS onset (DL1/DL2) was 9 d (range 2–11), with a median duration of 2 d (range 1–4); 12 pts (48%)received tocilizumab for CRS management and 12% received dexamethasone... Preliminary phase 1b results..."

CAR T-Cell Therapy • Clinical • P1/2 data • Gastroenterology • Gastrointestinal Disorder • Hematological Malignancies • Immunology • Multiple Myeloma • Plasmacytoma

Phase I Open-Label Single-Arm Study of BCMA/CD19 Dual-Targeting FasTCAR-T Cells (GC012F) As First-Line Therapy for Transplant-Eligible Newly Diagnosed High-Risk Multiple Myeloma (ASH 2022) - P1/2 | "Twelve pts received 2 cycles induction therapy of bortezomib, lenalidomide and dexamethasone (VRd), and one patient received 1 cycle bortezomib, epirubicin, and dexamethasone (PAD) and 1 cycle VRd prior to the infusion. GC012F was administered as a single infusion at 3 doses levels (DL) of 1x105/kg, 2x105/kg, or 3x105/kg, after receiving a conditioning chemotherapy consisting of cyclophosphamide and fludarabine...Conclusion In this phase I study for transplant-eligible newly diagnosed high-risk MM, BCMA-CD19 dual FasTCAR-T GC012F showed a very favorable safety profile, high efficacy with 100% ORR and 100% MRD negativity. The promising preliminary results warrants further assessment of GC012F for TE NDMM with more patients and longer follow-up."

CAR T-Cell Therapy • Clinical • P1 data • Hematological Malignancies • Inflammation • Multiple Myeloma • Oncology • Transplantation • CD19

DURGA-5: Study of AZD0120 in Newly Diagnosed Multiple Myeloma Ineligible for ASCT (clinicaltrials.gov) - P3 | N=750 | Recruiting | Sponsor: AstraZeneca

New P3 trial • Hematological Malignancies • Multiple Myeloma • Oncology

A dual targeting BCMA and CD19 fastcar-t (GC012F/AZD0120) as first-line therapy for newly diagnosed multiple myeloma (ASH 2025) - P1, P1/2 | "Here, we report the combined data of these twostudies to provide long term follow up data of GC012F/AZD0120 in NDMM pts.Methods Eligible NDMM pts received a single infusion of GC012F/AZD0120 following two cycles lenalidomide,bortezomib and dexamethasone (RVd) induction therapy...GC012F/AZD0120 was administered at 4 dose levels:1x105/kg (n=1), 1.5x105/kg (n=3), 2x105/kg (n=4), or 3x105/kg (n=22) after a standard 3-daylymphodepletion regimen of fludarabine and cyclophosphamide...3 pts were treated with one dose of tocilizumab and 1 pt was treated with one dose oftocilizumab and corticosteroids...These promising results highlight the potential ofGC012F/AZD0120 CAR-T therapy for treating NDMM patients with HR features and transplant ineligibleNDMM pts. Further research with a larger patient population and extended follow-up is needed tovalidate these findings and address this critically unmet medical need."

Clinical • Hematological Malignancies • Inflammation • Multiple Myeloma • Plasmacytoma • PLAAT3

Updated Results of a Phase I Open-Label Single-Arm Study of Dual Targeting BCMA and CD19 Fastcar-T Cells (GC012F) As First-Line Therapy for Transplant-Eligible Newly Diagnosed High-Risk Multiple Myeloma (ASH 2023) - P1/2 | "Of the 22 pts, 21 pts received 2 cycles induction therapy of bortezomib, lenalidomide and dexamethasone (VRd), and one patient received 1 cycle bortezomib, epirubicin, and dexamethasone (PAD) and 1 cycle VRd prior to the infusion. GC012F was administered as a single infusion at 3 doses levels (DL) of 1x105/kg (n=1), 2x105/kg (n=4), or 3x105/kg (n=17), after a standard 3-day lymphodepletion consisting of cyclophosphamide and fludarabine...The promising preliminary results achieved with GC012F demonstrate potential of CAR-T therapy in newly-diagnosed MM pts. Further research with larger patient population and longer follow-up shall bring the hope to this unmet medical need."

CAR T-Cell Therapy • Clinical • P1 data • Hematological Malignancies • Inflammation • Multiple Myeloma • Oncology • Transplantation

BCMA/CD19 dual-targeting CAR T cell therapy in older patients with newly diagnosed multiple myeloma: a phase I study. (PubMed, Blood Adv) - P1 | "GC012F, now renamed AZD0120, is an autologous B-cell maturation antigen (BCMA) and CD19 dual-targeting CAR T cell product manufactured on the novel FasTCAR® platform...In this single-arm phase 1 study, NDMM patients age ≥70 received two cycles of VRD (bortezomib/lenalidomide/dexamethasone) induction therapy followed by AZD0120 infusion at the dose of 1.5x105 or 3x105 cells/kg...AZD0120 dual-targeting fast CAR T shows promising preliminary efficacy and a manageable safety profile for older patients with NDMM. NCT05840107."

Journal • P1 data • Hematological Disorders • Hematological Malignancies • Inflammation • Leukopenia • Multiple Myeloma • Neutropenia • Oncology

Safety and Efficacy of GC012F/AZD0120, an Autologous CD19/BCMA Dual-Targeting FasTCAR T Cell Therapy, in Refractory Systemic Lupus Erythematosus: 96-Week Long-term Follow-up (ACR Convergence 2026) - No abstract available

CAR T-Cell Therapy • Clinical • Immunology • Inflammatory Arthritis • Lupus • Systemic Lupus Erythematosus

Safety and Efficacy of AZD0120 (GC012F), a Dual-Targeting CD19/BCMA CAR-T Cell Therapy, in Refractory Systemic Lupus Erythematosus: Phase 1/2 Interim Results (ACR Convergence 2026) - No abstract available

CAR T-Cell Therapy • Clinical • P1/2 data • Immunology • Inflammatory Arthritis • Lupus • Systemic Lupus Erythematosus

Updated results of a multicenter first-in-human study of BCMA/CD19 dual-targeting fast CAR-T GC012F for patients with relapsed/refractory multiple myeloma (RRMM). (ASCO 2022) - P1 | "BCMA-CD19 dual FasT CAR-T GC012F continues to provide deep and durable responses with a favorable safety profile in additional RRMM pts across all dose levels demonstrating a very high MRD negativity rate including in pts refractory to anti-CD38, PI and IMIDs. GC012F is currently being studied in earlier lines of therapy as well as additional indications."

Clinical • P1 data • Hematological Malignancies • Infectious Disease • Inflammation • Leukemia • Multiple Myeloma • Novel Coronavirus Disease • Oncology • Transplantation • CD19

Updated results of a phase I, open-label study of BCMA/CD19 dual-targeting fast CAR-T GC012F for patients with relapsed/refractory multiple myeloma (RRMM). (ASCO 2023) - P1 | "The updated results showed GC012F continues to provide deep and durable responses, and a very high MRD negativity ratein RRMM pts, including in pts refractory to anti-CD38, PIs and IMIDs. Based on these promising results of the study of GC012F for RRMM, further clinical studies will be conducted to confirm the efficacy of GC012F. Clinical trial information: NCT04236011; NCT04182581."

Clinical • Bone Marrow Transplantation • Hematological Malignancies • Inflammation • Multiple Myeloma • Oncology • Transplantation

A PHASE I OPEN-LABEL SINGLE-ARM STUDY OF DUAL TARGETING BCMA AND CD19 FASTCAR-T (GC012F) AS FIRST-LINE THERAPY FOR TRANSPLANT-ELIGIBLE NEWLY DIAGNOSED HIGH-RISK MULTIPLE MYELOMA (EHA 2024) - P1/2 | "Consistent with the previous RRMM cohort treated with GC012F, initial data from this study demonstrated thatGC012F resulted in deep and durable response in TE HR NDMM pts with a very favorable safety profile. Allthree dose groups achieved MRD negativity and 100% ORR. The promising preliminary results achieved withGC012F demonstrate the potential of CAR-T therapy in treating NDMM pts."

Clinical • P1 data • Hematological Malignancies • Inflammation • Multiple Myeloma • Oncology • Transplantation

B-Cell Maturation Antigen/CD19 Dual-Targeting Immunotherapy in Newly Diagnosed Multiple Myeloma. (PubMed, JAMA Oncol) - "The findings of this single-arm, open-label phase 1 cohort study suggest that GC012F may be a safe treatment associated with positive health and survival outcomes for patients with high-risk NDMM eligible for transplant. Owing to the small sample size, further studies with larger cohorts and longer follow-up durations are needed."

Journal • Hematological Malignancies • Inflammation • Multiple Myeloma • Oncology • Transplantation

AZD0120, A DUAL BCMA/CD19 CAR-T CELL THERAPY, DEMONSTRATES A NAIVE/CENTRAL MEMORY PHENOTYPE AND INDUCES A RAPID DECLINE IN SOLUBLE BCMA IN RELAPSED/REFRACTORY MULTIPLE MYELOMA (EHA 2026) - P1/2 | "These findings support AZD0120 as a promising treatment for RRMM. Updated clinical data on safety and efficacy will be presented at EHA2026."

CAR T-Cell Therapy • IO biomarker • Hematological Malignancies • Inflammation • Multiple Myeloma • CRP • CXCL8 • IL10 • IL15 • IL6

ACE-CAR T: Autoimmune Cytopenia Elimination by Chimeric Antigen Receptor T-cell Therapy (clinicaltrials.gov) - P1 | N=16 | Not yet recruiting | Sponsor: Saurabh Dahiya

New P1 trial • Hematological Disorders • Immunology

AZD0120: Data from P3 DURGA-4 trial (NCT07391657) for r/r multiple myeloma post 2027 (AstraZeneca) - H1 and Q2 2026 Results: Data from P2 DURGA-3 trial (NCT06235229) for r/r multiple myeloma post 2027

P2 data • P3 data • Hematological Malignancies • Multiple Myeloma • Oncology

A Phase III, Multicentre Study to Evaluate Consolidation Treatment With AZD0120 Compared With ASCT, in Participants With Newly Diagnosed Multiple Myeloma Who Are Transplant Eligible (DURGA-6). (clinicaltrials.gov) - P3 | N=750 | Not yet recruiting | Sponsor: AstraZeneca

New P3 trial • Hematological Malignancies • Multiple Myeloma • Oncology • Transplantation

AZD0120: Data from P1/2 DURGA-1 trial (NCT05850234) for r/r multiple myeloma in H2 2027 (AstraZeneca) - H1 and Q2 2026 Results: Data from P1 DURGA-2 trial (NCT07073547) for newly diagnosed and r/r multiple myeloma H2 2026

P1 data • P1/2 data • Hematological Malignancies • Multiple Myeloma • Oncology

TRIAL IN PROGRESS: A PHASE 3 STUDY COMPARING AZD0120, A BCMA/CD19 DUAL-TARGETING CAR-T CELL THERAPY, VERSUS STANDARD REGIMENS IN PATIENTS WITH RELAPSED/REFRACTORY MULTIPLE MYELOMA (DURGA-4) (EHA 2026) - P3 | "Eligible patients will be randomized to receive either a single infusion of AZD0120 or standard therapy including DKd (daratumumab, carfilzomib, and dexamethasone), DPd (daratumumab, pomalidomide, and dexamethasone), PVd (pomalidomide, bortezomib, and dexamethasone), or Kd (carfilzomib and dexamethasone)...Results This is a trial in progress and is expected to enroll ~500 patients in ~17 countries, with first patient enrollment planned in Q1 2026. Summary/Conclusion We hypothesize that in this Phase 3 study AZD0120 will demonstrate superior PFS and MRD negativity versus standard regimens in patients with RRMM."

CAR T-Cell Therapy • Clinical • P3 data • Hematological Malignancies • Inflammation • Multiple Myeloma

D8311C00001: A trial to learn how well AZD0120 works and how safe it is in people with relapsed or refractory multiple myeloma. (clinicaltrialsregister.eu) - P2/3 | N=153 | Not yet recruiting | Sponsor: AstraZeneca AB | N=38 ➔ 153

Enrollment change • Hematological Malignancies • Multiple Myeloma • Oncology

A Study of GC012F Injection in Subjects With Refractory Systemic Lupus Erythematosus (clinicaltrials.gov) - P1/2 | N=20 | Active, not recruiting | Sponsor: Gracell Biotechnologies (Shanghai) Co., Ltd. | Recruiting ➔ Active, not recruiting | N=118 ➔ 20

Enrollment change • Enrollment closed • Immunology • Inflammatory Arthritis • Lupus • Systemic Lupus Erythematosus

Characterization of AZD0120, a BCMA/CD19 Dual-Targeting Autologous CAR T-cell Therapy Generated from PBMCs of Patients with Rheumatologic Disease (EULAR 2026) - "The presence of plasma cells in rheumatologic diseases suggests that simultaneous targeting of CD19 and BCMA may deepen responses and broaden applicability across autoimmune indications. Image(s), graphic(s), or table(s)"

CAR T-Cell Therapy • Clinical • IO biomarker • Hematological Disorders • Hematological Malignancies • Immunology • Inflammatory Arthritis • Lupus • Multiple Myeloma • Myositis • Rheumatoid Arthritis • Scleroderma • Systemic Lupus Erythematosus • Systemic Sclerosis • IFNG

Dual Target CAR-T Cell Treatment for Refractory Systemic Lupus Erythematosus (SLE) Patients (clinicaltrials.gov) - P1 | N=18 | Recruiting | Sponsor: RenJi Hospital | Trial completion date: May 2025 ➔ Jun 2029 | Trial primary completion date: Nov 2023 ➔ Aug 2028

Trial completion date • Trial primary completion date • Immunology • Inflammatory Arthritis • Lupus • Systemic Lupus Erythematosus

D8311C00001: A trial to learn how well AZD0120 works and how safe it is in people with relapsed or refractory multiple myeloma. (clinicaltrialsregister.eu) - P2/3 | N=153 | Not yet recruiting | Sponsor: AstraZeneca AB

New P2/3 trial • Hematological Malignancies • Multiple Myeloma • Oncology

D8311C00001: A trial to learn how well AZD0120 works and how safe it is in people with relapsed or refractory multiple myeloma. (clinicaltrialsregister.eu) - P2/3 | N=38 | Not yet recruiting | Sponsor: AstraZeneca AB

New P2/3 trial • Hematological Malignancies • Multiple Myeloma • Oncology

CAR-T Therapy Targeting CD19 and BCMA in Highly Sensitized Kidney Transplant Participants (clinicaltrials.gov) - P1 | N=12 | Recruiting | Sponsor: Tongji Hospital | Not yet recruiting ➔ Recruiting

Enrollment open • IO biomarker • Chronic Kidney Disease • Nephrology • Renal Disease • Transplantation • CXCL8 • IFNG • IL12A • IL13 • IL4 • SDC1