Equecabtagene Autoleucel

SHANGHAI and NANJING, China and SAN JOSE, Calif., June 15, 2024 /PRNewswire/ -- IASO biotechnology ("IASO Bio"), a biopharmaceutical company engaged in discovering, developing, manufacturing and marketing innovative cell therapies and antibody products, presented clinical data on the use of Equecabtagene Autoleucel (Eque-cel), the world's first approved fully human CAR-T product, for the treatment of transplant-ineligible patients with high-risk newly diagnosed multiple myeloma (NDMM) in an oral presentation at the 2024 European Hematology Association (EHA) Annual Congress. Presentation Title: Eque-cel, A Novel Fully Human BCMA-Targeting CAR-T Therapy in Patients with High Risk Newly Diagnosed Multiple Myeloma Presentation Type: Oral report Session Date and Time: June 15, 2024, 16:30 - 17:45 (Central European Summer Time) Location: Madrid, Spain Publication Number: S206 Presenter: Professor Bing Chen, Nanjing Drum Tower Hospital The FUMANBA-2 study is a multicenter, open-label, phase I, single-arm study initiated by researchers, with the principal investigators being Professor Lijuan Chen from Jiangsu Province Hospital and Professor Bing Chen from Nanjing Drum Tower Hospital. This study aims to assess the efficacy, safety, and pharmacokinetics/pharmacodynamics characteristics of Eque-cel for the treatment of high-risk NDMM. Subjects must complete four cycles of induction treatment before the infusion of Eque-cel. After the third induction treatment cycle, patients deemed unsuitable for autologous hematopoietic stem cell transplantation (ASCT) by the researcher will undergo peripheral blood mononuclear cell collection and subsequently receive Eque-cel treatment, with an infusion dose of 1×106 CAR-T/kg. The primary endpoints were the proportion of minimal residual disease (MRD) negative subjects and progression-free survival (PFS), and secondary endpoints included overall response rate (ORR), duration of response (DOR), safety, and pharmacokinetics/pharmacodynamics (PK/PD). As of January 25th, 2024, 16 subjects received Eque-cel therapy. High-risk cytogenetics were detected in all subjects, including 62.5 percent double-hit and 12.5 percent triple-hit. 25 percent subjects had extramedullary disease. 37.5 percent subjects had R-ISS stage III disease, among whom 6.3 percent with double-hit and 6.3 percent with triple-hit. Efficacy: After the infusion of Eque-cel, the median follow-up time was 7.46 months (range: 2.8-18.1), the median PFS had not been reached, the 12-month PFS rate was 84.4% (95% CI: 49.31-96.00), all subjects achieved MRD negativity, of which 71.4% (95% CI: 25.8-92.0) maintained MRD negativity for more than 12 months; the overall response rate (ORR) was 100%, with 93.8% achieving stringent complete response (sCR). Safety: After the infusion of Eque-cel, the incidence of grade 1-2 cytokine release syndrome (CRS) was 68.8%, no grade 3 or above CRS was observed, and no immune effector cell-related neurotoxicity syndrome (ICANS) or other neurological toxicities occurred. The median time of CRS occurrence was the 7th day after infusion (range: 2-9 days), and the median duration of CRS was 3 days (range: 1-8 days). The most common grade 3 or above drug-related adverse event wase blood cell count reduction, and the incidence of grade 3 or above infectious disease adverse events was 25.0%. PK/PD: The median peak time of CAR copy number in peripheral blood was 10 days after infusion (range: 7-21 days), with a median peak level of 79,681.299 copies/μg DNA. 81.25% of subjects achieved clearance of free B-cell maturation antigen (sBCMA) within one month after infusion; the median peak time of cytokine detection IL-6 and CRP after infusion were the 7th and 10th day, respectively, and the serum ferritin level did not change significantly. Professor Lijuan Chen from Jiangsu Province Hospital stated: " Eque-cel, as a novel fully human BCMA CAR-T therapy, has shown encouraging efficacy and safety in high-risk patients with newly diagnosed multiple myeloma who are ineligible for transplantation. This is the world's first report on CAR-T therapy being used as a first-line treatment in this specific patient population. For NDMM patients who are not suitable for transplantation, the application of CAR-T therapy as a first-line treatment is expected to further improve the remission rate, extend survival, and improve patient prognosis compared to traditional chemotherapy and other targeted drug treatments. This allows us to see the application potential of Eque-cel in the front-line treatment of MM. Advancing CAR-T therapy to the first line will provide patients with more diverse and promising treatment options. With further research and the continuous improvement of treatment strategies, we look forward to CAR-T therapy benefiting more patients in the future." Professor Bing Chen from Nanjing Drum Tower Hospital stated: "High-risk newly diagnosed multiple myeloma patients have a poor prognosis in standard first-line treatment. For high-risk NDMM patients who do not meet the conditions for ASCT, Eque-cel has shown superior efficacy and safety, achieving deep and sustained remission, with all patients achieving MRD negativity. This opens up a new approach to reverse the poor prognosis of high-risk myeloma patients. Moreover, compared with relapsed/refractory multiple myeloma (RRMM) patients, the incidence and severity of CRS in high-risk NDMM patients treated with Eque-cel are lower, showing a more favorable safety profile. We will further investigate the clinical benefits of Eque-cel for high-risk newly diagnosed multiple myeloma patients with longer follow-up." About IASO Bio IASO Bio is a biopharmaceutical company engaged in the discovery and development of novel cell therapies and biologics for oncology and autoimmune diseases. IASO Bio possesses comprehensive capabilities spanning the entire drug development process, from early discovery to clinical development, regulatory approval, and commercial production. The pipeline in the company includes a diversified portfolio of over 10 novel products, including Equecabtagene Autoleucel (a fully human BCMA CAR-T injection). Equecabtagene Autoleucel received New Drug Application (NDA) approval from China's National Medical Products Administration (NMPA) and U.S. FDA IND approval for the treatment of RRMM. Leveraging its strong management team, innovative product pipeline, GMP production, as well as integrated manufactural and clinical capabilities, IASO aims to deliver transformative, curable, and affordable therapies that fulfil unmet medical needs to patients in China as well as around the world. For more information, please visit or . SOURCE IASO Bio

SHANGHAI, NANJING, China, and SAN JOSE, Calif., May 10, 2024 /PRNewswire/ -- On May 10, 2024, the impactful international academic journal, Science Immunology, a sub-journal of Science, published a research paper titled "Single-cell analysis of anti-BCMA CAR T cell therapy in patients with central nervous system autoimmunity". This paper focuses on the single-cell multi-omics analysis of the fully human BCMA targeting autologous CAR-T cell injection (Equecabtagene Autoleucel, Eque-cel, R&D code: CT103A) in the treatment of patients with neuromyelitis optica spectrum disorder (NMOSD). This paper described for the first time in the world the dynamic evolutionary trajectories of CAR-T cells in patients with autoimmune diseases, analyzed the molecular characteristics of central infiltration of CAR-T cells, revealed the mechanism of immune remodeling in the central nervous system during CAR-T therapy for autoimmune diseases of the central nervous system, and elucidated the molecular differences of CAR-T cells between patients with autoimmune diseases and cancers at the cellular and molecular levels. In 2022, IASO Bio published the interim results of an investigator-initiated phase I clinical study of Eque-cel in the treatment of neuromyelitis optica spectrum disorder (NMOSD) on the Signal Transduction and Targeted Therapy (IF = 38.1), the sub-journal of Nature, which preliminarily demonstrated the good tolerability and safety, durable pathogenic antibody clearance, and potential clinical efficacy of Eque-cel in NMOSD. However, the cell kinetics and immunological characteristics of CAR-T cells in the treatment of autoimmune diseases of the central nervous system are still unclear. This is an investigator-initiated open-label study to evaluate the safety and efficacy of Eque-cel for the treatment of relapsed/refractory antibody-mediated idiopathic inflammatory disorders of the nervous system (NCT04561557). It was conducted by Prof. Wei Wang's team at Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology. In this study, single-cell multi-omics analysis was performed on blood and cerebrospinal fluid (CSF) samples from 5 patients with relapsed or refractory AQP4-positive NMOSD and 5 patients with multiple myeloma treated with Eque-cel to study the in vivo characteristics of CAR-T cells in patients with autoimmune diseases. The study found that there was only a 13% overlap in the B cell immunoglobulin heavy chain variable region (IgVH) sequences between the peripheral blood and CSF of patients with NMOSD, suggesting that B cells in the CSF are mostly derived from B cells intrinsic to the central nervous system rather than from the peripheral blood. CAR-T cells with chemotactic properties can cross the blood-brain barrier and enter the central nervous system to directly kill abnormal plasma cells in the central nervous system. The action facilitates the restoration of central immunity as it reduces the secretion of autoantibodies within sheaths and the abnormal activation of immune cells. As a result, the immune disorder in the central nervous system of the NMOSD patients can be rectified and its immune system can be reset. The study shows that CAR-T cells predominantly exhibit a CD8+ cycling CAR-T cell phenotype in patients with immune diseases, and the cytotoxic function of CAR-T cells is reduced compared with the control group. The above characteristics explain the relatively low grade of CRS and relatively short survival time of CAR-T cells in patients with immune diseases after CAR-T therapy, which is conducive to earlier immune remodeling in patients, and further confirms the good safety of Eque-cel in autoimmune diseases. Professor Wei Wang from Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, the principal investigator of this study, said " With the continuous emergence of innovative clinical studies of CAR-T cells in the treatment of autoimmune diseases in different countries and regions around the world, experts around the world have attached increasing importance to and recognized the prospects of such innovative cell therapy for patients with relapsed and refractory immune diseases. This study is the first in-depth single-cell multi-omics analysis of multiple types of body fluids from NMOSD patients in the world, depicting the dynamic evolutionary trajectories of CAR-T cells in patients with immune diseases at the cellular and molecular level. In particular, we found that CAR-T cells with chemotactic properties can more easily cross the blood-brain barrier and enter the central nervous system to directly kill abnormal immune cells in the central nervous system. This discovery is very critical for the treatment of immune abnormalities in the central nervous system. We also noted different characteristics in CAR-T cells between autoimmune patients and tumor patients, which provided scientific insights into refining CAR-T cell therapies for autoimmune diseases." Ms. Jinhua Zhang, the Founder and Chief Executive Officer of IASO Bio, said, "I am very pleased that another research achievement of Eque-cel for the treatment of autoimmune diseases has been published in Science Immunology. This is the third academic article in the field of autoimmunity published in internationally impactful journals through the collaboration between IASO Bio and the team led by Professor Wei Wang at Tongji Hospital in Wuhan this year. This research achievement has once again created a "world first", further validating the efficacy, safety and persistence of CAR-T therapy in the treatment of autoimmune diseases, and once again firming the determination of IASO Bio to continue to focus on the development of CAR-T products in the field of autoimmunity. Currently, IASO Bio has obtained INDs for multiple autoimmune diseases, including NMOSD and MG, in both China and the United States, and plans to accelerate the progress of these projects. In terms of BD collaboration in the field of autoimmunity, we entered into a licensing agreement with Cabaletta Bio, a US cell therapy company, in 2022. We granted Cabaletta exclusive rights globally to develop, produce, and commercialize CAR-T therapies for autoimmune diseases using our clinically validated, fully human CD19 binder. Currently, the product has received IND approvals for a number of autoimmune indications, including systemic lupus erythematosus (SLE) / lupus nephritis (LN), idiopathic inflammatory myopathies (IIM), systemic sclerosis（SSc）and generalized myasthenia gravis（gMG）, and is steadily progressing through clinical trials. We are actively seeking to expand our global BD collaborations in the autoimmune area to accelerate product development so that autoimmune patients worldwide would be able to use safer and more effective treatments at an early date." About IASO Bio IASO Bio is a biopharmaceutical company engaged in the discovery and development of novel cell therapies and biologics for oncology and autoimmune diseases. IASO Bio possesses comprehensive capabilities spanning the entire drug development process, from early discovery to clinical development, regulatory approval, and commercial production. The pipeline in the company includes a diversified portfolio of over 10 novel products, including Equecabtagene Autoleucel (a fully human BCMA CAR-T injection). Equecabtagene Autoleucel received New Drug Application (NDA) approval from China's National Medical Products Administration (NMPA) and U.S. FDA IND approval for the treatment of RRMM. Leveraging its strong management team, innovative product pipeline, GMP production, as well as integrated manufactural and clinical capabilities, IASO aims to deliver transformative and curable therapies that fulfil unmet medical needs to patients in China as well as around the world. For more information, please visit or . SOURCE IASO Bio