RegulationOrphan Drug (United States), Orphan Drug (European Union), Priority Review (China), Breakthrough Therapy (China), Conditional marketing approval (China), Regenerative Medicine Advanced Therapy (United States), PRIME (European Union)

Structure/Sequence

Sequence Code 1117836311

Source: *****

Clinical Trials associated with Zevorcabtagene Autoleucel

NCT06825845

/ Not yet recruitingNot Applicable

Long Term Follow-up Observational Study in Patients Treated with Gene-Modified T-Cell Therapy

This is a prospective long-term follow-up (LTFU) study to evaluate the long-term safety and survival benefit for patients who received at least one of CARsgen's GM T cell product in a clinical trial or as a commercially available product. In this study, patients will be followed for up to 15 years after last GM T-cell infusion for the evaluation of delayed adverse events (AEs), vector persistence, potential risk for integration, and survival time due to GM T-cell exposure.

Start Date31 Mar 2025

Sponsor / Collaborator

CARsgen Therapeutics Co. Ltd.

NCT06659770

/ Not yet recruitingNot Applicable

A Real-World Observational Study: Zevorcabtagene Autoleucel Injection in Patients with Relapsed/Refractory Multiple Myeloma

This study is a single-arm, open-label, multicenter, post-marketing, Phase IV, prospective, observational clinical trial to evaluate the efficacy and safety of the post-marketing product zevor-cel as treatment for subjects with R/R MM in the real world.

Start Date01 Oct 2024

Sponsor / Collaborator

CARsgen Therapeutics Co. Ltd.

NCT03915184

/ Active, not recruitingPhase 1/2

Open Label, Multi-center, Phase 1b/2 Clinical Trial to Evaluate the Safety and Efficacy of Autologous CAR BCMA T Cells (CT053) in Patients With Relapsed and/or Refractory Multiple Myeloma (LUMMICAR STUDY 2)

A phase 1b/2, open label, multi-center, Clinical Study of Chimeric Antigen Receptor T Cells targeting BCMA in patients with relapsed and or refractory multiple myeloma.

Start Date25 Sep 2019

Sponsor / Collaborator

CARsgen Therapeutics Co. Ltd.

100 Clinical Results associated with Zevorcabtagene Autoleucel

100 Translational Medicine associated with Zevorcabtagene Autoleucel

100 Patents (Medical) associated with Zevorcabtagene Autoleucel

Literatures (Medical) associated with Zevorcabtagene Autoleucel

01 Jul 2024·Molecular Diagnosis & Therapy

Zevorcabtagene Autoleucel: First Approval

Review

Author: Dhillon, Sohita

Zevorcabtagene autoleucel () is a fully humanised B cell maturation antigen (BCMA)-targeting specific chimeric antigen receptor (CAR) T-cell therapy being developed by CARsgen for the treatment of multiple myeloma. Zevorcabtagene autoleucel is an autologous CAR T cell comprising a fully human BCMA-specific scFv (25C2), a CD8α hinge region and transmembrane domain, a 4-1BB costimulatory domain and a CD3-ζ T cell activation domain. Zevorcabtagene autoleucel recognizes and induces selective toxicity against BCMA-expressing tumour cells leading to their elimination. In February 2024, zevorcabtagene autoleucel received its first approval in China for the treatment of adults with relapsed or refractory multiple myeloma who have progressed after ≥ 3 prior lines of therapy (including ≥ 1 proteasome inhibitor and an immunomodulatory agent). Clinical studies of zevorcabtagene autoleucel are underway in Canada and the US. This article summarizes the milestones in the development of zevorcabtagene autoleucel leading to this first approval for relapsed or refractory multiple myeloma.

01 Jul 2022·Blood reviewsQ2 · MEDICINE

Current state and next-generation CAR-T cells in multiple myeloma

Q2 · MEDICINE

Review

Author: Facon, Thierry ; Mitra, Suman ; Manier, Salomon ; Nudel, Morgane ; Prodhomme, Chloé ; Escure, Guillaume ; Ingegnere, Tiziano

Chimeric antigen receptor T cells (CAR-T cells) have emerged as a potentially transformative new approach to treating hematological malignancies. Ide-cel, an autologous B cell maturation antigen (BCMA) targeting CAR-T cells, has recently been approved to treat multiple myeloma (MM). Here, we review the main clinical trials of CAR-T cells in MM with the most advanced autologous BCMA-directed ide-cel and cilta-cel, the human CARs orva-cel and CT053, the alternative manufacturing process with P-BCMA-101 and bb21217, the dual CAR GC012F and the allogenic BCMA-directed CAR-T cells ALLO-715. In light of those clinical data, we provide an overview of CAR-T cells' main potential resistance mechanisms, including antigen loss, antigen spreading, anti-CAR antibodies, CAR-T cell exhaustion, and the emergence of a non-permissive microenvironment. Finally, we describe the principal area of research to build the next generation of CAR-T cells, with armored-, gated- or commuting-CARs, CARs associated with knock out of specific genes, and CAR-T cells made from γδT cells or NK cells.

01 Aug 2018·Journal of clinical oncology : official journal of the American Society of Clinical OncologyQ1 · MEDICINE

T Cells Genetically Modified to Express an Anti–B-Cell Maturation Antigen Chimeric Antigen Receptor Cause Remissions of Poor-Prognosis Relapsed Multiple Myeloma

Q1 · MEDICINE

Article

Author: Maric, Irina ; Lam, Norris ; Mikkilineni, Lekha ; Brudno, Jennifer N ; Stroncek, David F ; Hansen, Brenna G ; Yuan, Constance ; Pavletic, Steven ; Wang, Michael ; Patel, Rashmika ; Gress, Ronald E ; Lawrence, Judith ; Stetler-Stevenson, Maryalice ; Hartman, Steven D ; Rose, Jeremy J ; Kanakry, Jennifer A ; Salem, Dalia ; Kochenderfer, James N ; Ali, Syed Abbas ; Hakim, Frances ; Feldman, Steven A

Purpose:

Therapies with novel mechanisms of action are needed for multiple myeloma (MM). T cells can be genetically modified to express chimeric antigen receptors (CARs), which are artificial proteins that target T cells to antigens. B-cell maturation antigen (BCMA) is expressed by normal and malignant plasma cells but not normal essential cells. We conducted the first-in-humans clinical trial, to our knowledge, of T cells expressing a CAR targeting BCMA (CAR-BCMA).

Patients and Methods:

Sixteen patients received 9 × 106 CAR-BCMA T cells/kg at the highest dose level of the trial; we are reporting results of these 16 patients. The patients had a median of 9.5 prior lines of MM therapy. Sixty-three percent of patients had MM refractory to the last treatment regimen before protocol enrollment. T cells were transduced with a γ-retroviral vector encoding CAR-BCMA. Patients received CAR-BCMA T cells after a conditioning chemotherapy regimen of cyclophosphamide and fludarabine.

Results:

The overall response rate was 81%, with 63% very good partial response or complete response. Median event-free survival was 31 weeks. Responses included eradication of extensive bone marrow myeloma and resolution of soft-tissue plasmacytomas. All 11 patients who obtained an anti-MM response of partial response or better and had MM evaluable for minimal residual disease obtained bone marrow minimal residual disease–negative status. High peak blood CAR+ cell levels were associated with anti-MM responses. Cytokine-release syndrome toxicities were severe in some cases but were reversible. Blood CAR-BCMA T cells were predominantly highly differentiated CD8+ T cells 6 to 9 days after infusion. BCMA antigen loss from MM was observed.

Conclusion:

CAR-BCMA T cells had substantial activity against heavily treated relapsed/refractory MM. Our results should encourage additional development of CAR T-cell therapies for MM.

News (Medical) associated with Zevorcabtagene Autoleucel

24 Mar 2025

·www.prnewswire.com

CARVYKTI Continued Performance Reflects Growing Confidence in CAR-T Therapies for Multiple Myeloma | DelveInsight

As a BCMA-targeting CAR T-cell therapy, CARVYKTI addresses a high unmet need in relapsed or refractory multiple myeloma patients. With promising clinical efficacy, durable responses, and growing adoption in advanced treatment lines, CARVYKTI is poised for substantial revenue growth. LAS VEGAS, March 24, 2025 /PRNewswire/ -- DelveInsight's " CARVYKTI Market Size, Forecast, and Market Insight Report" highlights the details around CARVYKTI, a BCMA-directed, genetically modified autologous T-cell immunotherapy, which involves reprogramming a patient's T cells with a transgene encoding a CAR that identifies and eliminates cells that express BCMA. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of CARVYKTI. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Janssen's CARVYKTI (ciltacabtagene autoleucel) Overview CARVYKTI is an autologous T-cell immunotherapy that targets B-cell maturation antigen (BCMA). It works by genetically modifying a patient's T cells with a transgene that encodes a chimeric antigen receptor (CAR). This CAR helps the T cells recognize and destroy cells expressing BCMA, which is primarily found in malignant multiple myeloma B-lineage cells, late-stage B cells, and plasma cells. The CARVYKTI CAR protein includes two single-domain antibodies designed to bind strongly to human BCMA, enhancing T-cell activation, proliferation, and the destruction of target cells upon binding. Currently, CAR-T cell therapies are only available to UK patients through clinical trials. Janssen recently decided not to proceed with seeking approval for cilta-cel (CARVYKTI) for myeloma patients in the UK through the National Institute for Health and Care Excellence (NICE). This decision means cilta-cel will not be available on the NHS for now, although it remains accessible through clinical trials. Janssen's decision does not impact ongoing trials. The drug is currently being evaluated in Phase III trials: CARTITUDE-6 for frontline multiple myeloma transplant eligible vs ASCT and CARTITUDE-5 for frontline multiple myeloma TNI. In 2024, CARVYKTI generated sales of USD 963 million across the world. CARVYKTI Dosage and Administration CARVYKTI is administered as a single infusion containing a suspension of CAR-positive, viable T cells in one infusion bag. The recommended dosage ranges from 0.5 to 1.0 × 10⁶ CAR-positive viable T cells per kilogram of body weight, with a maximum limit of 1 × 10⁸ CAR-positive viable T cells per infusion. Learn more about CARVYKTI projected market size for multiple myeloma @ CARVYKTI Market Potential CAR-T cell immunotherapy is emerging as a revolutionary treatment for multiple myeloma, offering new hope to patients who have exhausted conventional treatment options. Currently, only two CAR-T cell therapies are approved for multiple myeloma: ABECMA (idecabtagene vicleucel) from Bristol-Myers Squibb and CARVYKTI (ciltacabtagene autoleucel) from Johnson & Johnson Innovative Medicine. Their approval highlights the potential of CAR-T therapy to transform multiple myeloma treatment. A key advantage of CAR-T therapies is their "one-and-done" nature, requiring a single administration, unlike bispecific antibodies such as TECVAYLI and TALVEY, which need ongoing dosing. Present CAR-T therapies use a patient's own T cells (autologous), but research is underway to develop allogeneic CAR-T cells derived from healthy donors. Allogeneic therapies could offer off-the-shelf solutions, enhancing accessibility and lowering costs. The approval of CAR-T therapies has created new opportunities for companies focused on advanced-stage multiple myeloma (fourth line and beyond). Several companies are progressing with their CAR-T candidates at different stages of development. The CAR-T cell therapy market for multiple myeloma is expected to grow significantly between 2024 and 2034, driven by its high efficacy and potential for expanded use. Collaboration between pharmaceutical companies and research institutions, along with ongoing innovation, is expected to accelerate market growth. However, challenges such as manufacturing complexity and pricing will need to be addressed to unlock the full potential of CAR-T therapies. Discover more about the CAR T-cell therapy for multiple myeloma market in detail @ CAR T-cell Therapy for Multiple Myeloma Market Report Emerging Competitors of CARVYKTI Some of the CAR-Ts in the multiple myeloma pipeline that will give fierce competition to CARVYKTI include Anito-cel (Arcellx), PHE885 (Novartis), BMS-986393 (Bristol-Myers Squibb), Zevorcabtagene Autoleucel (CARsgen Therapeutics), and GLPG5301 (Galapagos), among others In December 2024, Arcellx announced encouraging new data from its Phase II pivotal iMMagine-1 trial for anito-cel in multiple myeloma. The results were presented during an oral session at the 66th American Society of Hematology (ASH) Annual Meeting on Monday, December 9, 2024, at 5:30 p.m. PT. Also at ASH 2024, BMS shared the first overall survival (OS) and progression-free survival (PFS) results for arlo-cel in an oral presentation. The Phase I trial involved patients with relapsed or refractory multiple myeloma who had previously undergone at least three treatments, including a proteasome inhibitor, an immunomodulatory drug, and anti-CD38 therapy. Among 79 patients evaluable for efficacy, with a median follow-up of 16.1 months (range: 2.8–25.2), arlo-cel showed an overall response rate (ORR) of 87%, indicating durable responses. Minimal residual disease (MRD) was assessed as an exploratory measure, with 57% (48 of 84) of MRD-evaluable patients included. Of these, 46% (22 of 48) were MRD-negative and achieved a complete response (CR) or stringent CR (sCR). Across all treated patients, 27% (23 of 84) were MRD-negative and achieved a CR. Median PFS was 18.3 months (95% CI: 11.8–21.9), while the median OS had not yet been reached. To know more about the number of competing drugs in development, visit @ CARVYKTI Market Positioning Compared to Other Drugs Key Milestones of CARVYKTI In April 2024, Johnson & Johnson announced that the US FDA approved CARVYKTI (ciltacabtagene autoleucel; cilta-cel) for the treatment of adult patients with RRMM who have received at least one prior line of therapy, including a proteasome inhibitor and an immunomodulatory agent, and are refractory to lenalidomide. In March 2024, Johnson & Johnson announced that the US FDA Oncologic Drugs Advisory Committee (ODAC) recommends CARVYKTI for the treatment of adult patients with RRMM who have received at least one prior line of therapy, including a proteasome inhibitor (PI) and an immunomodulatory agent (IMiD) and who are refractory to lenalidomide. In February 2024, Johnson & Johnson announced that the Committee for Medicinal Products for Human Use (CHMP) of the EMA recommended the approval of a Type II variation for CARVYKTI for the earlier treatment of RRMM. In September 2022, Legend Biotech announced that Japan's Ministry of Health, Labour and Welfare (MHLW) had approved CARVYKTI (ciltacabtagene autoleucel) for the treatment of adults with relapsed or refractory multiple myeloma, limited to cases meeting both of the following conditions including patients having no history of CAR-positive T-cell infusion therapy targeting BCMA and patients who have received three or more lines of therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 monoclonal antibody, and in whom multiple myeloma has not responded to or has relapsed following the most recent therapy. In May 2022, Janssen announced that the European Commission (EC) granted conditional marketing authorization for CARVYKTI for the treatment of adults with relapsed and refractory multiple myeloma (RRMM) who have received at least three prior therapies, including an immunomodulatory agent (IMiD), a proteasome inhibitor (PI) and an anti-CD38 antibody, and have demonstrated disease progression on the last therapy. In February 2022, Janssen announced that the US FDA had approved CARVYKTI (ciltacabtagene autoleucel; cilta-cel) for the treatment of adults with relapsed or refractory multiple myeloma (RRMM) after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. In August 2020, Legend Biotech announced that the China Center for Drug Evaluation, National Medical Products Administration (CDE, NMPA) had recommended BTD for ciltacabtagene autoleucel (cilta-cel; LCAR-B38M CAR-T cells) for the treatment of adults with relapsed or refractory multiple myeloma. In December 2019, Janssen announced receipt of a BTD from the US FDA for cilta-cel. In April 2019, the EMA granted a PRIME (PRIority MEdicines) designation for the company's investigational BCMA CAR-T therapy. In February 2019, the US FDA granted ODD for cilta-cel (JNJ-4528), and in February 2020, the European Commission also granted orphan designation for this drug. In December 2017, Janssen entered a worldwide collaboration and license agreement with Legend Biotech to jointly develop and commercialize LCAR-B38M in multiple myeloma Discover how CARVYKTI is shaping the multiple myeloma treatment landscape @ CARVYKTI CAR-T CARVYKTI Market Dynamics The market for CARVYKTI is driven by the increasing prevalence of multiple myeloma and the growing demand for innovative therapies that can address the limitations of existing treatments, such as proteasome inhibitors and immunomodulatory drugs. The competitive landscape includes other BCMA-targeting therapies, such as Bristol Myers Squibb's ABECMA (idecabtagene vicleucel), which was the first BCMA-directed CAR-T therapy approved for multiple myeloma. CARVYKTI's strong clinical efficacy, coupled with its potential for long-term remission, gives it a competitive edge, but challenges related to manufacturing scalability, logistical complexities, and high treatment costs could impact its market penetration and patient accessibility. Strategic partnerships and ongoing clinical development will play a key role in expanding CARVYKTI's market reach. Janssen and Legend Biotech are actively working to improve manufacturing processes to reduce vein-to-vein time and address supply chain bottlenecks. Furthermore, ongoing trials, such as CARTITUDE-4, are exploring CARVYKTI's use in earlier lines of therapy, which could significantly expand its patient base and market potential. If successful in earlier settings, CARVYKTI could shift the treatment paradigm for multiple myeloma and increase adoption among physicians and healthcare providers. Despite its promising clinical profile, CARVYKTI faces challenges related to reimbursement and healthcare infrastructure. The high cost of CAR-T therapies often limits patient access, particularly in markets with strict reimbursement policies. Moreover, the complex nature of CAR-T administration, which requires specialized centers and trained personnel, adds to the barriers. However, continued clinical success, regulatory support, and improvements in manufacturing and delivery infrastructure could strengthen CARVYKTI's market position and establish it as a cornerstone therapy in the multiple myeloma treatment landscape. Dive deeper to get more insight into CARVYKTI's strengths & weaknesses relative to competitors @ CARVYKTI Market Drug Report Table of Contents Related Reports Relapsing Refractory Multiple Myeloma Market Relapsing Refractory Multiple Myeloma Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key RRMM companies including AbbVie, Genentech, Amgen, Onyx Therapeutics Inc., Bristol-Myers Squibb, MedImmune LLC, Novartis Pharmaceuticals, Incyte Corporation, Takeda, among others. Relapsing Refractory Multiple Myeloma Pipeline Relapsing Refractory Multiple Myeloma Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key RRMM companies, including Bristol-Myers Squibb, I-MAB Biopharma, Pfizer, Arcellx, Gilead Sciences, Novartis, Array Biopharma, Hrain Biotechnology Co., Ltd., Cartesian Therapeutics, Xencor, Takeda, Sorrento Therapeutics, Heidelberg Pharma AG, Ichnos Sciences, Allogene Therapeutics, Harpoon Therapeutics, Cellectis, Poseida Therapeutics, Regeneron Pharmaceuticals, ONK Therapeutics, TeneoOne, iTeos Therapeutics, Oricell Therapeutics, Anaveon AG, Luminary Therapeutics, Seagen Inc., Trillium Therapeutics Inc., Virtuoso BINco, Inc., Seagen Inc., Trillium Therapeutics Inc., among others. Multiple Myeloma Market Multiple Myeloma Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key multiple myeloma companies including Sanofi, Karyopharm Therapeutics, AbbVie, Takeda Pharmaceutical, Celgene, Bristol-Myers Squibb, RAPA Therapeutics, Pfizer, Array Biopharma, Cellectar Biosciences, BioLineRx, Celgene, Aduro Biotech, ExCellThera, Janssen Pharmaceutical, Precision BioSciences, Takeda, Glenmark (Ichnos Sciences SA), Poseida Therapeutics, Molecular Partners AG, Chipscreen Biosciences, AbbVie, Genentech (Roche), Janssen Biotech, Nanjing Legend Biotech, Merck Sharp & Dohme Corp., among others. Multiple Myeloma Pipeline Multiple Myeloma Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key multiple myeloma companies, including CASI Pharmaceuticals, Carsgen Therapeutics, Cartesian Therapeutics, Gracell Biotechnology Shanghai Co., Ltd., Sorrento Therapeutics, TeneoOne, Karyopharma Therapeutics, Arcellx, Poseida Therapeutics, Ichnos Sciences, Nerviano Medical Sciences, Bristol Myers Squib, Ascentage Pharma, Ionis Pharmaceuticals, Chongqing Precision Biotech Co., Ltd., CRISPR Therapeutics, AstraZeneca, IGM Biosciences, Novartis, GlaxoSmithKline, Innovent Biologics, Keymed Biociences, Starton Therapeutics, Takeda, Fate Therapeutics, Gilead Sciences, Jiangsu Chia Tai Fenghai Pharmaceutical Co., Ltd., Janssen Pharmaceutical, Nanjing IASO Biotechnology Co., Ltd., GPCR Therapeutics, Chimerix, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Clinical ResultImmunotherapyOrphan DrugDrug ApprovalPhase 3

06 Nov 2024

·www.prnewswire.com

CARsgen® to Present Zevor-cel, CT071 and CT0590 at ASH 2024 Annual Congress

SHANGHAI, Nov. 6, 2024 /PRNewswire/ -- CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on innovative CAR T-cell therapies for the treatment of hematologic malignancies and solid tumors, announces that the Company will present the clinical data of zevorcabtagene autoleucel, CT071, and CT0590 as posters at the 66th Annual Congress of the American Society of Hematology ("ASH") which is due to take place between Dec 7 – 10, 2024. The abstracts are available on ASH official website. "CARsgen is advancing therapeutic options for hematologic malignancies with robust CAR T-cell pipeline, which includes the autologous CAR T-cell therapy zevor-cel, the single-day-culture CT071, and the allogeneic CAR T-cell candidate CT0590. These initiatives underscore CARsgen's strong commitment to innovation in hematology. Leveraging proprietary technology platforms such as CARcelerate® and THANK-uCAR®, CARsgen is focused on developing differentiated CAR T-cell therapies to address the critical challenges faced by the clinical community. We are excited to share new data and are confident in the potential of our CAR T-cell therapies to benefit patients worldwide," said Raffaele Baffa, M.D., Ph.D., Chief Medical Officer of CARsgen Therapeutics. Subgroup Analyses of Phase 2 Study: Evaluating the Efficacy of Fully Human BCMA-Targeting CAR T Cells (Zevorcabtagene Autoleucel) in Patients with Relapsed/Refractory Multiple Myeloma Publication Number: 4762 Presentation Time: 6:00 PM - 8:00 PM, Monday, December 9, 2024 (PST) In 102 patients with RRMM who had received at least 3 prior lines of therapy including an immunomodulatory drug and a proteasome inhibitor, the objective response rate (ORR) was 92.2%, the stringent complete response (sCR) or complete response (CR) was 71.6%. The ORR or the CR/sCR rate was not affected by any of the baseline characteristics tested. With a median follow-up of 20.3 (range: 0.4 to 27) months, the median duration of response (DOR), progression-free survival (PFS), and overall survival (OS) data were not mature and therefore, 18-month (18m) and estimated 30-month (30m) event free rates were used as efficacy outcomes for subgroup analyses. The DOR, PFS and OS were not impacted by age or ISS. These subgroup analyses indicate that baseline characteristics have minimal impact on the clinical efficacy of zevorcabtagene autoleucel, demonstrating that even RRMM patients with poor prognostic factors can benefit from zevorcabtagene autoleucel. GPRC5D-Targeted CAR T-Cell Therapy CT071 for the Treatment of Refractory/Relapsed Multiple Myeloma Publication Number: 3451 Presentation Time: 6:00 PM - 8:00 PM, Sunday, December 8, 2024 (PST) CT071 is a fully human GPRC5D-targeting autologous CAR T-cell product manufactured using an expedited CARcelerate® platform which shortens the manufacturing process to around 30 hours resulting in shorter vein-to-vein time. Patients with RRMM who had previously received ≥ 3 prior lines of therapy (LOT) or patients who experienced progression or lack of response having been treated with a proteasome inhibitor and an immunomodulatory agent or those who were double class-refractory, all with ECOG score of 0-2 were enrolled. In 17 patients who had been dosed with CT071, 11 patients (64.7%) experienced cytokine release syndrome (CRS), all at Grade 1 (n=8) or 2 (n=3). No immune effector cell-associated neurotoxicity syndrome (ICANS) was observed. No dose limiting toxicity (DLT) occurred. The overall response rate (ORR) was 94.1% (16/17), and the stringent complete response (sCR) rate was 52.9% (9/17). Notably, 7 patients achieved complete response or better at week 4. All 4 patients with previous exposure to BCMA or BCMA/CD19 CAR T responded to CT071 (2 with sCR and 2 with partial response). A First-in-Human Study of CT0590, a Triple Knock-out, Allogeneic CAR T-Cell Therapy Targeting BCMA and NKG2A, in Subjects with Relapsed/Refractory Multiple Myeloma Publication Number: 4843 Presentation Time: 6:00 PM - 8:00 PM, Monday, December 9, 2024 (PST) CT0590 is an allogeneic dual CAR T-cell therapy deploying THANK-uCAR® technology that targets both BCMA and NKG2A (a membrane protein expressed in NK and T cells), featuring a triple knockout for TRAC/B2M/NKG2A which mitigates against graft-versus-host disease (GvHD), host immune rejection and fratricide. This is a first-in-human (FIH), open-label, single center, phase I study of CT0590 in subjects with RRMM to evaluate the safety, tolerability, pharmacokinetics and preliminary efficacy of CT0590 (NCT05066022). As of 22-Apr-2024, 5 subjects were enrolled (4 subjects with RRMM and 1 subject with primary plasma cell leukemia [pPCL] under compassionate use). No ≥ 3 grade cytokine release syndrome (CRS) was observed. No cases of immune effector cell-associated neurotoxicity syndrome (ICANS) or GvHD were observed. No dose limiting toxicities were reported and there were no withdrawal from the study or deaths due to adverse events. With a median follow-up time of 16.6 months (range: 5.1, 24.2), 3 subjects achieved confirmed responses including 2 subjects with stringent complete response (sCR) and 1 subject with partial response (PR). The 2 subjects with sCR include1 RRMM subject [sCR ongoing] with a DOR longer than 23 months as of data cut-off date and 1 pPCL subject with a duration of response (DOR) of 20 months. In the 2 subjects with sCR, CAR copies peaked at > 280,000 copies/µg genomic DNA. Preliminary results of this FIH study of the CT0590 allogeneic CAR T-cell therapy demonstrated a manageable safety profile while achieving deep and durable clinical responses. About Zevorcabtagene Autoleucel Zevorcabtagene autoleucel is a fully human, autologous BCMA CAR T-cell product for the treatment of Multiple Myeloma (MM). Zevorcabtagene autoleucel was approved by the NMPA on February 23, 2024 for the treatment of adult patients with R/R MM who have progressed after at least 3 prior lines of therapy (including a proteasome inhibitor and an immunomodulatory agent). CARsgen is conducting a separate Phase 1b/2 LUMMICAR STUDY 2 clinical trial in North America to evaluate the safety and efficacy of zevorcabtagene autoleucel in R/R MM. Zevorcabtagene autoleucel received Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug designations from the U.S. FDA in 2019, as well as Priority Medicines (PRIME) and Orphan Medicinal Product designations from the European Medicines Agency (EMA) in 2019 and 2020, respectively. About CT071 CT071 is a CAR T-cell therapy candidate developed utilizing the proprietary CARcelerate® platform of CARsgen targeting GPRC5D for the treatment of relapsed/refractory MM or relapsed/refractory plasma cell leukemia (PCL). An IIT (NCT05838131) is ongoing in China to evaluate the preliminary safety and efficacy of CT071 for the treatment of patients with relapsed/refractory multiple myeloma or plasma cell leukemia. A separate investigator-initiated trial (NCT06407947) is ongoing in China for the treatment of patients with newly diagnosed multiple myeloma (NDMM). About CT0590 CT0590 is a BCMA-targeting allogeneic CAR T-cell product candidate deploying CARsgen's THANK-uCAR® technology. An IIT is ongoing in China to evaluate the preliminary safety and efficacy of CT0590 for the treatment of R/R MM. About CARsgen Therapeutics Holdings Limited CARsgen is a biopharmaceutical company with operations in China and the U.S., focusing on innovative CAR T-cell therapies for the treatment of hematologic malignancies and solid tumors. CARsgen has established a comprehensive CAR T-cell research and development platform that covers target discovery, innovative CAR T-cell development, clinical trials, and commercial-scale production. Internally, CARsgen has developed novel technologies and a product pipeline with global rights to address significant challenges faced by existing CAR T-cell therapies. Efforts include improving safety profile, enhancing the efficacy in treating solid tumors, and reducing treatment costs. CARsgen's mission is to become a global biopharmaceutical leader that provides innovative and differentiated cell therapies for cancer patients worldwide and makes cancer curable. Forward-looking Statements All statements in this press release that are not historical fact or that do not relate to present facts or current conditions are forward-looking statements. Such forward-looking statements express the Group's current views, projections, beliefs and expectations with respect to future events as of the date of this press release. Such forward-looking statements are based on a number of assumptions and factors beyond the Group's control. As a result, they are subject to significant risks and uncertainties, and actual events or results may differ materially from these forward-looking statements and the forward-looking events discussed in this press release might not occur. Such risks and uncertainties include, but are not limited to, those detailed under the heading "Principal Risks and Uncertainties" in our most recent annual report and interim report and other announcements and reports made available on our corporate website, . No representation or warranty is given as to the achievement or reasonableness of, and no reliance should be placed on, any projections, targets, estimates or forecasts contained in this press release. For more information, please visit SOURCE CARsgen Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Cell TherapyClinical ResultPhase 1ASHImmunotherapy

01 Nov 2024

·endpts.com

FDA removes hold on three CARsgen CAR-T trials; Ono passes on itolizumab

Plus, news about Kymera Therapeutics, Nanoscope, Eupraxia, Alpha Cognition and EyePoint: FDA lifts hold of CARsgen’s CAR-T therapies: The FDA on Thursday lifted a clinical hold on three of CARsgen Therapeutics’ CAR-T therapies. The trials were halted in December due to issues found at the company’s manufacturing facilities in Durham, NC. The drugs — CT053, CT041 and CT071 — are being investigated against BCMA, Claudin18.2 and GPRC5D, respectively. — Anna Brown Ono lets option for itolizumab expire: The company signed an agreement with Equillium in 2022 to potentially purchase an IgG1 monoclonal antibody in graft versus host disease. But Ono decided not to acquire the drug, with Equillium’s stock $EQ closing 37% lower on Thursday. Equillium also reorganized its pipeline and paused enrollment of a Phase 3 study of itolizumab, according to an SEC filing . — Max Gelman Kymera Therapeutics ends oncology work: The biotech will instead focus on its immunology pipeline , which includes STAT6, IRAK4 and TYK2 degrader programs. Two cancer drugs will not proceed past Phase 1 development. Kymera said it is “evaluating partnership opportunities” for them. — Max Gelman Nanoscope’s two-year data for eye disease gene therapy: The program, MCO-010, is being developed for permanent and severe vision loss from advanced retinitis pigmentosa. The high-dose group had a statistically significant improvement in patients being able to read an eye chart after 52 and 76 weeks. There were no serious side effects, and the most commonly reported ones were hypertension in different parts of the eye. — Max Gelman Eupraxia discloses $32M private placement: The Canadian company plans to use the funding to support research into its experimental treatment for eosinophilic esophagitis. — Jaimy Lee Alpha Cognition’s $25M offering: The Vancouver biotech, which makes neurodegenerative disease treatments, also plans to uplist to the Nasdaq . — Kyle LaHucik EyePoint ended up raising $161 million in its upsized public offering. — Jaimy Lee

AcquisitionGene TherapyPhase 3Cell TherapyImmunotherapy

100 Deals associated with Zevorcabtagene Autoleucel

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Multiple Myeloma	China	Kaixing Life Technology (Shanghai) Co., Ltd.	23 Feb 2024

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Relapse multiple myeloma	Phase 2	China	CARsgen Therapeutics Co. Ltd.	10 Jun 2019
B-Cell Leukemia	Clinical	China	CARsgen Therapeutics Co. Ltd.	29 Dec 2017
Gastroesophageal junction adenocarcinoma	Clinical	China	CARsgen Therapeutics Co. Ltd.	29 Dec 2017
Hepatocellular Carcinoma	Clinical	China	CARsgen Therapeutics Co. Ltd.	29 Dec 2017
Pancreatic Cancer	Clinical	China	CARsgen Therapeutics Co. Ltd.	29 Dec 2017

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03975907 (LUMMICAR STUDY 1, ASH2024) Manual	Phase 2	Multiple Myeloma	102	Zevorcabtagene Autoleucel	hppjvtwqux(csdblljtdq) = fchkwvqvxz gicdoagreo (dpqmvpydfi, 85.13 - 96.55) View more	Positive	09 Dec 2024
NCT03975907 (LUMMICAR STUDY 1, ASH2024) Manual	Phase 2	Multiple Myeloma	102	Zevorcabtagene Autoleucel (<65 years)	hppjvtwqux(csdblljtdq) = fcatrqjdai gicdoagreo (dpqmvpydfi ) View more	Positive	09 Dec 2024
NCT03975907 (LUMMICAR-1 \| LUMMICAR STUDY 1, EHA 12024 \| EHA 22024) Manual	Phase 2	Multiple Myeloma Last line	102	ZEVORCABTAGENE AUTOLEUCEL	yoikcofsby(dcrhhlbvkh) = uvhjzowcju dgtahigjpn (hrgsmbsrhs, 85.13 - 96.55) View more	Positive	14 May 2024
NCT03975907 (LUMMICAR STUDY1, ASH 12022 \| ASH 22022) Manual	Phase 2	Multiple Myeloma Last line	102	Zevorcabtagene autoleucel	hvxypgdzqv(fyrqbuxbjy) = thgqwyteqj pvxaicmbml (wdpxmcwaic ) View more	Positive	15 Nov 2022
NCT03915184 (LUMMICAR STUDY 2, Biospace) Manual	Phase 2	Relapse multiple myeloma	17	Fludarabine Phosphate+Cyclophosphamide+Zevorcabtagene autoleucel	ztspcuqspo(fhzsbhhkoe) = hrioltuqyw voctgmidco (qhohdinhmj ) View more	Positive	21 Sep 2022
NCT03975907 (Lummicar Study 1, ASH 12021 \| ASH 22021) Manual	Phase 1	Relapse multiple myeloma Last line	-	CT053 (median age of 54 years (range 34-62))	vwdwsrnefv(xxianyvngm) = ximkhimwgt ugoamveaqk (qvflkkebkj ) View more	Positive	05 Nov 2021
NCT03915184 (PRNewswire) Manual	Phase 1	Multiple Myeloma	24	Zevorcabtagene autoleucel	hialbssklm(lxcckydebc) = pebphvirpf knblfwchxf (meivudlspj ) View more	Positive	06 Dec 2020
NCT03915184 (Lummicar-2, ASH 12020 \| ASH 22020) Manual	Phase 1	Relapse multiple myeloma	20	cyclophosphamide+fludarabine+CT053	zuvwexefpo(kqpcxkaodq) = No grade 3 or higher CRS or neurotoxicity was observed. Twelve of 14 subjects (86%) experienced grade 1 or 2 CRS, including 2 subjects who experienced grade 2 CRS and 1 subject who had grade 2 neurotoxicity. lpdnsfkffs (elcasnpyky ) View more	Positive	05 Nov 2020
NCT03975907 (ASH 12020 \| ASH 22020) Manual	Phase 1	Relapse multiple myeloma Last line	14	CT053	lldczruwjl(peugcvywzw) = None qndjfagier (pwlbmzfdfe ) View more	Positive	05 Nov 2020
NCT03716856 (Lummicar Study 1, ASH 12018 \| ASH 22018) Manual	Phase 1	Relapse multiple myeloma	16	fludarabine+cyclophosphamide+CT053	tcwuxyesaw(hjblahobcr) = none jbljfqpuij (qafkjcgyru ) View more	Positive	29 Nov 2018

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

Chat with Hiro

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis