puzolcabtagene autoleucel (pCAR-19B) / Chongqing Precision Biotech - LARVOL DELTA

Autologous CD19-directed CAR-T exhibit promising safety and efficacy in refractory primary immune thrombocytopenia (ASH 2025) - P1/2 | "The patients receivedconventional three-day FC lymphodepletion preconditioning chemotherapy with fludarabine 30mg/m2and cyclophosphamide 300mg/m2 on days -5, -4, and -3 except one patient. Our study suggested that anti-CD19 CAR-T cell therapy had manageable safety and promising efficacy intreating refractory primary ITP. However, the patients had quite different responses to therapy, showingthat a single B cell-eradicating intervention may not be enough for some patients. The success ofdaratumumab use for pt1 indicated the importance of plasma cell-eradication for the treatment ofrefractory primary immune thrombocytopenia."

Clinical • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Inflammation • Inflammatory Arthritis • Thrombocytopenia • Thrombocytopenic Purpura

China Approves First CAR-T Therapy Specifically for Pediatric r/r B-ALL: Chongqing Precision Biotech’s Puzolcabtagene Autoleucel Injection (pCAR-19B) Receives NMPA Approval (Chongqing Precision Biotech Press Release) - "The clinical development of pCAR-19B followed a single-arm, open-label study design. Phase 1 was led by Prof. Zhou Jianfeng at Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, serving as Principal Investigator (PI). Phase 2 pivotal study was co-led by Prof. Wang Tianyou of Beijing Children’s Hospital, Capital Medical University, and Prof. Zhang Yicheng of Tongji Hospital, in collaboration with more than ten top tertiary hospitals in China."

China approval • B Acute Lymphoblastic Leukemia

Reduced Intensity Lymphodepletion and Dose Optimization of Autologous CD19 Chimeric Antigen Receptor T Cell (CAR-T) Therapy in Pediatric Refractory Systemic Lupus Erythematosus (KIDNEY WEEK 2025) - "All patients underwent lymphodepleting chemotherapy with fludarabine and cyclophosphamide, with reduced-intensity lymphodepletion allowed. Three patients (13.6 %) relapsed 5–9 months post-infusion. Conclusion This study revealed that autologous CD19 CAR-T cell therapy with reduced lymphodepletion and lower cell doses is effective and safe for pediatric patients with refractory SLE."

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

A Phase 2 Clinical Trial of Anti-CD19 CAR-T (pCAR-19B) in Chinese Pediatric and Young Adult with Relapsed/Refractory (R/R) CD19+ B-ALL: The First Pivotal Study in an Asian Population (ASH 2024) - P2 | "The chimeric antigen receptor (CAR-T) cell therapy has revolutionized the treatment of relapsed/refractory (R/R) B-cell ALL, with the first FDA-approved product, Kymriah (tisagenlecleucel) developed by Novartis, significantly improving remission rates and survival times. pCAR-19B is an autologous CAR-T cell product with an optimized humanized CD19-specific single-chain variable fragment (scFv) designed to address safety concerns...Conclusions : Despite a heavy burden of BM blasts and a high risk of genetic variations, pCAR-19B demonstrated high remission rates, achieved a high rate of MRD-negative remission, durable responses, and a tolerable safety profile. This study represents the first pivotal clinical trial of childhood B-ALL in an Asian population, offering a new treatment option for Chinese pediatric and young adult patients with R/R B-ALL."

Clinical • P2 data • Acute Lymphocytic Leukemia • Childhood B Acute Lymphoblastic Leukemia • Hematological Malignancies • Leukemia • Oncology • Pediatrics • Solid Tumor

Frontline therapy combining with intrathecal dexamethasone effectively alleviate immune effector cell-associated neurotoxicity syndrome in pediatric relapsed B-ALL receiving pCAR-19B-cases report (BMC Pediatr) - "This case is registered on Clinicaltrials.gov under the number NCT05334823. Table 1 outlines the patient characteristics...For Case 1, steroid therapy lasted 10 days, after which ICANS returned to grade 2. The cumulative steroids administered reached 348 mg, but no significant effect on CAR copy number amplification was observed. For Case 2, steroid therapy lasted 3 days, and ICANS returned to grade 2 by the 3rd day post-steroid treatment. The cumulative steroid dose was 22.9 mg, with no impact on CAR amplification. Both patients are currently alive following bone marrow remission bridging. Their progression-free survival (PFS) was 150 days for Case 1 and 290 days for Case 2. As of October 1, 2023, their overall survival (OS) was 234 days for Case 1 and 305 days for Case 2."

Trial status • B Acute Lymphoblastic Leukemia

Study of Therapeutic Efficacy of Anti-CD19 CAR-T Cells in Refractory Systemic Lupus Erythematosus (clinicaltrials.gov) - P1 | N=22 | Active, not recruiting | Sponsor: The Children's Hospital of Zhejiang University School of Medicine | Recruiting ➔ Active, not recruiting | Trial primary completion date: Dec 2026 ➔ Apr 2025

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

AUTOLOGOUS CD19-DIRECTED CAR-T EXHIBIT PROMISING SAFETY AND EFFICACY IN REFRACTORY PRIMARY IMMUNE THROMBOCYTOPENIA (EHA 2025) - P1/2 | "After conventional three-day FC lymphodepletion preconditioning chemotherapy with fludarabine 30mg/m2 and cyclophosphamide 300mg/m2 on days -5, -4, and -3, a single dose of 1.5x105/kg or 3x105/kg CAR-T cells were infused intravenously. Our study suggested that anti-CD19 CAR-T cell therapy had manageable safety and promising efficacy in treating refractory primary ITP. However, the patients had quite different responses to therapy, showing that a single B cell-eradicating intervention may not be enough for some patients. The success of daratumumab use for pt1 indicated the importance of plasma cell-eradication for the treatment of refractory primary immune thrombocytopenia."

Clinical • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Inflammation • Thrombocytopenia • Thrombocytopenic Purpura

Clinical Trial of CD19 Targeted CAR-T Cell in Refractory Adult SLE (clinicaltrials.gov) - P1 | N=12 | Not yet recruiting | Sponsor: Chongqing Precision Biotech Co., Ltd

New P1 trial • Immunology • Inflammatory Arthritis • Lupus • Systemic Lupus Erythematosus

…blockbuster new drugs expected to be approved for marketing in China in 2025 [Google translation] (Sohu.com) - "Pcar-19B: This is a CAR-T cell immunotherapy product for malignant blood system diseases originating from CD19-positive B cells. It is used to treat patients aged 3 to 21 years old with CD19-positive relapsed or refractory B-cell acute lymphoblastic leukemia. The drug's marketing application has been accepted by the CDE and is expected to be approved in 2025."

China approval • B Acute Lymphoblastic Leukemia

Total remission rate exceeds 90%! Accurate Biology announces the latest clinical results of CAR-T products for leukemia [Google translation] (bydrug.pharmcube.com) - P2 | N=100 | NCT05334823 | Sponsor: Chongqing Precision Biotech Co., Ltd | "Among them, the Phase 2 clinical study data of pCAR-19B (Puqilunsai injection), a CAR-T cell therapy drug targeting CD19 by Precision Biology, was selected for oral presentation. The data showed that the best objective response rate (ORR) of Puqilunsai injection for Chinese children and young people aged 3 to 21 with CD19-positive relapsed or refractory B-cell acute lymphoblastic leukemia (R/R B-ALL) was 90.63%. According to the announcement on the ASH official website, this study represents the first key clinical trial of pediatric B-ALL in the Asian population, and is expected to provide a new treatment option for Chinese children and adolescents with R/R B-ALL."

P2 data • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Hematological Malignancies • Leukemia • Oncology

Safety and Efficacy of Low-Dose CD19-Targeted CAR T Cells in Refractory Pediatric Systemic Lupus Erythematosus (SLE) (KIDNEY WEEK 2024) - P1 | "Most patients(pts) were preconditioned with fludarabine (30 mg/m2) and cyclophosphamide (300 mg/m2) daily for 3 days, while some received dosage reduction due to infections or intolerance. The treatment of refractory pediatric SLE with a low dose of CD19-targeted CAR-T Cells (MC-1-50) demonstrated an excellent safety profile while exhibiting high efficacy."

CAR T-Cell Therapy • Clinical • Late-breaking abstract • Immunology • Infectious Disease • Inflammatory Arthritis • Lupus • Pediatrics • Renal Disease • Systemic Lupus Erythematosus

Clinical Research of CD19 Targeted CAR-T Cell in Relapsed/ Refractory B-ALL (clinicaltrials.gov) - P1 | N=24 | Not yet recruiting | Sponsor: Chongqing Precision Biotech Co., Ltd

CAR T-Cell Therapy • New P1 trial • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Hematological Malignancies • Leukemia • Oncology

Clinical Trial of CD19-targeted CAR-T Therapy for Refractory Juvenile Dermatomyositis (clinicaltrials.gov) - P1 | N=10 | Recruiting | Sponsor: Chongqing Precision Biotech Co., Ltd

New P1 trial • Dermatomyositis • Immunology • Myositis

A Study of pCAR-19B in the Treatment of CD19-positive Relapsed/Refractory B-ALL in Children and Adolescents (clinicaltrials.gov) - P2 | N=100 | Recruiting | Sponsor: Chongqing Precision Biotech Co., Ltd | Trial primary completion date: Dec 2023 ➔ Dec 2024

Trial primary completion date • Acute Lymphocytic Leukemia • Hematological Malignancies • Leukemia • Oncology • Pediatrics • CD19

Precision Biopharmaceuticals' CAR-T product application for listing has been accepted [Google translation] (new.qq.com) - "On July 20, the official website of the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) announced that Chongqing Precision Biotech's pCAR-19B cell autologous infusion preparation (Pulkilumai injection) has submitted a new drug application and has been accepted....According to the China Clinical Trial Registration and Information Disclosure Platform, pCAR-19 B cell autologous reinfusion preparations are conducting two Phase 2 clinical studies, which are used to treat patients with CD19-positive relapsed refractory acute lymphoblastic leukemia aged 3-21 years old, and patients with CD19-positive relapsed/refractory B-cell acute lymphoblastic leukemia aged 22-70 years old. The two studies plan to recruit 200 patients, 100 in each study."

China approval • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Hematological Malignancies • Leukemia • Oncology

New frontier observations | CAR-T for younger people received priority review; new stem cell drug approved for clinical trials [Google translation] (Sohu.com) - "Recently, according to the official website of CDE, Chongqing Precision Biotechnology Co., Ltd.'s pCAR-19 B cell autologous infusion preparation (Pulkilumab injection) is planned to be included in the priority review variety, and is intended to be used to treat CD19-positive relapsed or refractory B-cell acute lymphoblastic leukemia patients aged 3 to 21 years old...According to the CDE official website, there are currently two clinical trials of pCAR-19B underway. The conditions of the patients enrolled in the two trials are highly similar....The obvious difference between the two trials is that one trial population is 3-21 years old, and the other trial population is 22-70 years old. The current clinical priority review qualification for 3-21 years old may be related to the policy of encouraging the development of innovative drugs at a younger age."

Priority review • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Hematological Malignancies • Leukemia • Oncology

AUTOLOGOUS CD19-DIRECTED CAR T CELLS PRODUCED BY NOVEL BEDSIDE-MANUFACTURE PLATFORM EXHIBIT SAFETY AND EFFICACY IN R/R B-ALL (EHA 2024) - P1/2 | "Here, we describethe preliminary results of the first clinical study of CD19 CAR-T manufactured by the OlyCAR Platform inrelapsed/refractory B-cell acute lymphocytic leukemia (r/r B-ALL) patients (NCT05932173)... Our study has demonstrated that this novel bedside-manufactured CAR-T has good safety profiles andpromising efficacy in treating r/r B-ALL. It can significantly shorten the vein-to-vein time, greatly reduce costs,and improve accessibility, making CAR-T therapy an off-the-shelf treatment option."

CAR T-Cell Therapy • Clinical • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Immunology • Leukemia • Oncology

Study of Therapeutic Efficacy of Anti-CD19 CAR-T Cells in Children With Refractory Systemic Lupus Erythematosus (clinicaltrials.gov) - P1 | N=19 | Recruiting | Sponsor: The Children's Hospital of Zhejiang University School of Medicine

CAR T-Cell Therapy • New P1 trial • Immunology • Inflammatory Arthritis • Lupus • Systemic Lupus Erythematosus

a Phase I Clinical Research of CD19 Targeted CAR-T Cell in Relapsed/Refractory B Cell Lymphoma (clinicaltrials.gov) - P1 | N=24 | Recruiting | Sponsor: Chongqing Precision Biotech Co., Ltd

CAR T-Cell Therapy • New P1 trial • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology

Clinical Study of Chimeric Antigen Receptor T Lymphocytes (CAR-T) in the Treatment of Relapsed/Refractory Non-Hodgkin Lymphoma (clinicaltrials.gov) - P1 | N=54 | Recruiting | Sponsor: Chongqing Precision Biotech Co., Ltd | Trial completion date: Sep 2024 ➔ Sep 2025 | Trial primary completion date: Sep 2023 ➔ Sep 2024

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

Safety and Efficacy of CD19 Targeted CAR-T Therapy for Refractory Autoimmune Disease (clinicaltrials.gov) - P1 | N=24 | Recruiting | Sponsor: Chongqing Precision Biotech Co., Ltd

New P1 trial • ANCA Vasculitis • Dermatomyositis • Fibrosis • Immunology • Inflammatory Arthritis • Lupus • Myositis • Scleroderma • Sjogren's Syndrome • Systemic Lupus Erythematosus • Systemic Sclerosis • Vasculitis • IFIH1

Precision Biotechnology’s two CAR-T cell drugs have received IND acceptance [Google translation] (pharnexcloud.com) - "...Chongqing Precision Biotechnology Co., Ltd. (hereinafter referred to as 'Precision Biotechnology') has obtained IND applications for 'C-13-60 Cell Preparation' and 'MC-1-50 Cell Preparation' accepted."

New trial • Hematological Malignancies • Oncology • Solid Tumor

Safety and efficacy profile of a very low dose of MC-1-50 for treatment of r/r NHL. (ASCO 2023) - P1/2 | "Pts were pre-conditioned with fludarabine (25-30 mg/m2) and cyclophosphamide (200-300 mg/m2) daily for 3 days. The PRIMCAR platform could produce CAR T cells quickly with a high percentage of Tscm. Treatment of r/r B-NHL at very low dose resulted in excellent safety profiles while exhibiting a high and durable efficacy. Clinical trial information: NCT04271410."

Clinical • IO biomarker

Examining safety and durable disease remission in R/R B-ALL after autologous CD19-directed CAR T cells produced by novel PRIMCAR manufacture platform. (ASCO 2023) - P1/2 | " In this Phase I /II study (NCT04271410), MC-1-50 CAR T cells were manufactured by the PRIMCAR platform, which reduces manufacture periods to about 2 days...Pts were pre-conditioned with fludarabine (25-30 mg/m2) and cyclophosphamide (200-300 mg/m2) daily for 3 days... Treatment of r/r B-ALL at a very low dose resulted in excellent safety profiles while exhibiting a high and durable efficacy. That makes this PRIMCAR platform potential for outpatient administration in the future. Clinical trial information: NCT04271410."

CAR T-Cell Therapy • Clinical • IO biomarker • Oncology • CD19

Safety and Efficacy of CD19-Targeted CAR-T Therapy for Relapsed/Refractory CD19+ B Cell Leukemia and Lymphoma (clinicaltrials.gov) - P1/2 | N=40 | Recruiting | Sponsor: Chongqing Precision Biotech Co., Ltd | Trial completion date: Jul 2023 ➔ Jul 2024 | Trial primary completion date: Dec 2022 ➔ Dec 2023

Trial completion date • Trial primary completion date • Chronic Lymphocytic Leukemia • Hematological Malignancies • Leukemia • Lymphoma • Oncology • CD19