AZD0120 / AstraZeneca - LARVOL DELTA

ALACRITY: A phase 1b/2 study of AZD0120 (BCMA/CD19 CAR-T cell therapy) in participants with relapsed or refractory light chain amyloidosis (AL) (ASH 2025) - P1/2 | "Daratumumab+CyBorD (cyclophosphamide, bortezomib, dexamethasone) is approved to treat newly diagnosed AL (ANDROMEDA study), with improved outcomes...This study is registered on ClinicalTrials.gov (NCT07081646). Conclusion This study aims to determine the safety, tolerability, and efficacy of AZD0120 in adult participants with relapsed or refractory AL."

CAR T-Cell Therapy • P1/2 data • Amyloidosis • Hematological Malignancies • Hepatology • Leukemia • Liver Failure • Multiple Myeloma

AZD0120 (GC012F), a BCMA/CD19 dual-targeting fastcar T-cell therapy, demonstrates potent preclinical efficacy in multiple myeloma (ASH 2025) - "The FasTCAR-based AZD0120, designed to target BCMA and/or CD19 and maximize in vivo expansion, demonstrated potent, dose-dependent, and antigen-specific cytotoxicity both in vitro and in vivo. The superior antitumor function of AZD0120 versus GC012C suggests benefit of the FasTCAR manufacturing platform and supports continued development of AZD0120 as a promising therapeutic option for pts with MM."

CAR T-Cell Therapy • IO biomarker • Preclinical • Hematological Malignancies • Multiple Myeloma • CXCR4 • IFNG

One-year follow-up of CD19/BCMA dual-targeting fastcar-T GC012F (AZD0120) therapy in patients with refractory systemic lupus erythematosus (ASH 2025) - P1/2 | "Patients not in remission still hadimproved disease activity. An ongoing phase 1/2 trial (NCT06530849) is further evaluating its safety andefficacy in a larger cohort."

Clinical • Glomerulonephritis • Immunology • Infectious Disease • Inflammatory Arthritis • Lupus • Lupus Nephritis • Nephrology • Renal Disease • Rheumatology • Systemic Lupus Erythematosus

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

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

AstraZeneca advances haematology and cell therapy ambition with largest-ever presence at ASH (AstraZeneca Press Release) - "Investigational T-cell engager, surovatamig, and CAR T-cell therapy, AZD0120, will show potential with initial data across multiple blood cancers; New results will showcase benefit of Calquence in patients with mantle cell lymphoma and chronic lymphocytic leukaemia."

Clinical data • B Acute Lymphoblastic Leukemia • Chronic Lymphocytic Leukemia • Diffuse Large B Cell Lymphoma • Follicular Lymphoma • Mantle Cell Lymphoma • Multiple Myeloma

Preliminary findings from the Phase Ib portion of the Phase Ib/II DURGA-1 trial were reported at the American Society of Hematology (ASH) meeting (Firstwordpharma Press Release) - "Of 15 efficacy-evaluable patients — 10 receiving dose level 1, and 5 at dose level 2 — the overall response rate was 100%; three patients in the dose level 1 cohort and two in the dose level 2 cohort experienced a complete response (CR). The median time to response for both dosages was 0.9 months. Five patients in the low dose and three receiving the high dose of AZD0120 were evaluable for minimal residual disease (MRD); all were MRD-negative as measured by next-generation sequencing."

P1 data • Multiple Myeloma

A Phase I, Open-label, Multicenter Study to Evaluate the Safety, Tolerability, Cellular Kinetics, Immunogenicity, Pharmacodynamics, and Preliminary Efficacy of AZD0120 in Participants With Multiple Myeloma (clinicaltrials.gov) - P1 | N=64 | Recruiting | Sponsor: AstraZeneca | N=20 ➔ 64

Enrollment change • Hematological Malignancies • Multiple Myeloma • Oncology

Study of GC012F, CAR-T Therapy Targeting CD19 and BCMA in Chinese Participants With Relapsed or Refractory AL Amyloidosis (clinicaltrials.gov) - P1 | N=9 | Recruiting | Sponsor: Gracell Biotechnologies (Shanghai) Co., Ltd.

New P1 trial • Amyloidosis

CD19/BCMA Dual-Targeting Fastcar-T GC012F for Patients with Relapsed/Refractory B-Cell Non-Hodgkin's Lymphoma: An Update (ASH 2023) - "Patients received a median of 2 prior lines (range 2 - 3) of therapy including rituximab and anthracyclines. GC012F CAR-T cells were detected in the tumor biopsies, indicating the infiltration of CAR-T cells into the tumor lesions. Overall, based on the safety and efficacy results, CD19-BCMA dual targeting CAR-T product GC012F is a promising therapy for patients with refractory/ relapsed DLBCL."

Clinical • B Cell Lymphoma • B Cell Non-Hodgkin Lymphoma • Bone Marrow Transplantation • Diffuse Large B Cell Lymphoma • Hematological Disorders • Hematological Malignancies • Large B Cell Lymphoma • Leukopenia • Lymphoma • Neutropenia • Non-Hodgkin’s Lymphoma • Oncology • Thrombocytopenia • CD19

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

Autologous B Cell Maturation Antigen (BCMA) and CD19 Dual Targeting Fastcar-T Cells (GC012F/AZD0120) As First-Line Therapy for Elderly Patients with Newly Diagnosed Multiple Myeloma Patients (ASH 2024) - P1, P1/2 | "After enrollment, patients were permitted to receive up to two cycles of bortezomib, lenalidomide and dexamethasone (VRd) induction therapy before or after apheresis, at the discretion of investigators...All pts recovered from CRS within 5 days and only 1 pt received tocilizumab...Conclusion Consistent with the data from previous RRMM and transplant-eligible NDMM studies of GC012F, data from this study demonstrate that GC012F/AZD0120 resulted in a very favorable safety profile and promising, deep responses in elderly NDMM pts. Our results suggest that age alone should not preclude patients from receiving highly effective treatments aimed at cure or long-term disease control."

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

ZENITH: An Open-label Study of AZD0120 in Adults With Multiple Sclerosis (clinicaltrials.gov) - P1 | N=18 | Not yet recruiting | Sponsor: AstraZeneca

New P1 trial • CNS Disorders • Multiple Sclerosis

CD19/BCMA Dual-Targeting FasTCAR-T Cells GC012F (AZD0120) in patients with refractory Systemic Lupus Erythematosusm: an open-label, single-center Phase I study (ACR Convergence 2025) - P1, P1/2 | "All patients received a single infusion of autologous CD19-BCMA dual chimeric antigen receptor (CAR) T cells at one of three different dose levels following preconditioning with fludarabine and cyclophosphamide. GC012F CAR-T cell therapy was well tolerated and demonstrated high efficacy in patients with refractory SLE. A multicenter phase1/2 study (NCT06530849) is ongoing to further evaluate the safety and efficacy of GC012F in a broader range of patients with SLE."

CAR T-Cell Therapy • Clinical • P1 data • Fibrosis • Immunology • Infectious Disease • Inflammation • Inflammatory Arthritis • Lupus • Renal Disease • Systemic Lupus Erythematosus

GC012F Injection in the Treatment of Refractory Generalized Myasthenia Gravis(24103) (clinicaltrials.gov) - P1 | N=6 | Recruiting | Sponsor: Daishi Tian | Not yet recruiting ➔ Recruiting

Enrollment open • CNS Disorders • Myasthenia Gravis

GC012F Injection in Refractory Idiopathic Inflammatory Myopathy (clinicaltrials.gov) - P1 | N=12 | Not yet recruiting | Sponsor: Daishi Tian

New P1 trial • Myositis

GC012F Injection in the Treatment of Refractory Generalized Myasthenia Gravis(24103) (clinicaltrials.gov) - P1 | N=6 | Not yet recruiting | Sponsor: Daishi Tian

New P1 trial • CNS Disorders • Myasthenia Gravis

ALACRITY: A Phase 1b/2 Study of CAR T Cell Therapy Targeting CD19 and BCMA in Participants With Relapsed or Refractory AL Amyloidosis. (clinicaltrials.gov) - P1/2 | N=91 | Recruiting | Sponsor: Alexion Pharmaceuticals, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Amyloidosis

ALACRITY: A Phase 1b/2 Study of CAR T Cell Therapy Targeting CD19 and BCMA in Participants With Relapsed or Refractory AL Amyloidosis (clinicaltrials.gov) - P1/2 | N=91 | Not yet recruiting | Sponsor: Alexion Pharmaceuticals, Inc.

New P1/2 trial • Amyloidosis

GC012F in Patients With Autoimmune Diseases (clinicaltrials.gov) - P1 | N=15 | Not yet recruiting | Sponsor: Qiong Fu

New P1 trial • Immunology • Myositis • Scleroderma • Systemic Sclerosis

DURGA-3: A Study of GC012F in Patients With Relapsed/Refractory Multiple Myeloma (clinicaltrials.gov) - P1/2 | N=110 | Recruiting | Sponsor: Gracell Biotechnologies (Shanghai) Co., Ltd. | Trial completion date: Jun 2026 ➔ Mar 2026

Trial completion date • Hematological Malignancies • Multiple Myeloma • Oncology

A Study of GC012F (AZD0120), a CAR T Therapy Targeting CD19 and BCMA in Early-Line Treatment in Subjects With Multiple Myeloma (clinicaltrials.gov) - P1 | N=18 | Recruiting | Sponsor: AstraZeneca

New P1 trial • Hematological Malignancies • Multiple Myeloma • Oncology

An Exploratory Clinical Study of GC012F Injection for Refractory gMG (clinicaltrials.gov) - P1 | N=0 | Withdrawn | Sponsor: Zhejiang University | N=18 ➔ 0 | Not yet recruiting ➔ Withdrawn

Enrollment change • Trial withdrawal • CNS Disorders • Myasthenia Gravis

UPDATED RESULTS OF A PHASE I OPEN-LABEL SINGLE-ARM STUDY OF DUAL TARGETING BCMA AND CD19 FASTCAR-T (GC012F/AZD0120) AS FIRST-LINE THERAPY FOR TRANSPLANT-ELIGIBLE NEWLY DIAGNOSED HIGH-RISK MULTIPLE MYELOMA (EHA 2025) - P1/2 | "TE NDMM patients aged 18 to 70 were eligible if they had at least one of the following high risk features: R-ISS stage II or III; del (17p), t (4; 14), t (14; 16), or ≥ 4 copies of 1q21; extramedullary disease (EMD); IgD or IgE subtype; LDH levels above the upper limit of normal; any of the HR criterion defined by mSMART3.0.GC012F/AZD0120 was administered as a single infusion at 3 dose levels: 1x105/kg (n=1), 2x105/kg (n=4), or 3x105/kg (n=17), following a standard 3-day lymphodepletion regimen of fludarabine and cyclophosphamide.A total of 22 patients with a median age of 59 years old were enrolled and evaluable...Two pts received tocilizumab and 1 pt received tocilizumab and corticosteroid... Consistent with findings from the previous relapsed/ refractory MM cohort treated with GC012F/AZD0120, initial data from this study demonstrates that GC012F/AZD0120 induced deep and durable responses in TE HR NDMM pts, with a highly favorable safety profile. All patients..."

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

AUTOLOGOUS B CELL MATURATION ANTIGEN (BCMA) AND CD19 DUAL TARGETING FASTCAR-T CELLS (GC012F/AZD0120) AS FIRST-LINE THERAPY FOR ELDERLY PATIENTS WITH NEWLY DIAGNOSED MULTIPLE MYELOMA PATIENTS (EHA 2025) - P1, P1/2 | "After enrollment, patients were permitted to receive up to two cycles of bortezomib, lenalidomide and dexamethasone (VRd) induction therapy before or after apheresis, at the discretion of investigators...All pts recovered from CRS within 5 days and only 1 pt received tocilizumab... GC012F/AZD0120 exhibited a high clinical efficacy, and a favorable safety profile in this study of patients aged > 70. Given these excellent efficacy and safety data, chronological age should not be used as a criterion to exclude patients from CAR T cell clinical trials or from receiving anti-BCMA CAR T-cell therapy in a real-world setting. Future studies with larger cohorts are needed to confirm this approach as a standard of care."

CAR T-Cell Therapy • Clinical • Geriatric Disorders • Hematological Malignancies • Inflammation • Multiple Myeloma • Oncology