SHANGHAI, Oct. 7, 2025 /PRNewswire/ -- CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on developing innovative CAR T-cell therapies, announces that the results of CT071 (an autologous CAR T-cell product targeting GPRC5D) for the treatment of relapsed/refractory multiple myeloma (R/R MM) in an investigator-initiated trial (NCT05838131) have been published in The Lancet Haematology. The article was titled "GPRC5D-targeted CAR T-cell therapy (CT071) in patients with relapsed or refractory multiple myeloma: a first-in-human, single-centre, single-arm, phase 1 trial".
This trial aimed to assess the safety, preliminary activity of CT071 in R/R MM. 20 patients received CT071 infusion. Patients had received a median of 5 prior lines of therapy (IQR 3.0-6.5); 19 (95%) were double-class refractory, 13 (65%) were triple-class refractory, 5 (25%) were penta-drug refractory, 10 (50%) had received autologous stem cell transplantation (ASCT), and 5 (25%) had relapsed after CAR T-cell therapies targeting BCMA or BCMA/CD19. Four (20%) patients had extramedullary disease (EMD), 14 (70%) had =1 high-risk cytogenetics and 19 (95%) had a Revised International Staging System (R-ISS) 2 or 3 disease at baseline.
No dose-limiting toxicities (DLTs) were observed. The recommended phase 2 dose was determined at 0.1×106 CAR T cells/kg. Cytokine release syndrome occurred in 12 patients (60%), all graded as 1 or 2. No Grade =3 cytokine release syndrome (CRS) occurred. One patient (5%) experienced Grade 3 immune effector cell-associated neurotoxicity syndrome (ICANS). No treatment-related deaths occurred.
With a median follow-up of 10.71 months (IQR 6.13-12.02), the objective response rate (ORR) was 100% (95%CI, 83.2-100). 10 (50%) patients achieved stringent complete response (sCR), 4 (20%) had very good partial response (VGPR) and 6 (30%) had partial response (PR). One patient with a large EMD (125 mm×99 mm at baseline) achieved a 67.6% reduction at month 10 with ongoing PR. All 5 patients previously treated with an anti-BCMA CAR T (n=1) or anti-BCMA/CD19 CAR T (n=4) responded; 2 achieved PR, 1 achieved VGPR and 2 achieved sCR. 18 of 20 (90%) evaluable patients achieved MRD negativity at 10-6 including all 10 with CR or sCR. Median time to MRD negativity was 29 days (IQR 29-29). Median duration of response (DoR), progression-free survival (PFS) and overall survival (OS) were not reached. The data cutoff for all analyses was December 9, 2024.
About CT071
CT071 is a CAR T-cell therapy candidate developed utilizing the proprietary CARcelerate® platform targeting GPRC5D for the treatment of relapsed/refractory multiple myeloma (R/R MM) or plasma cell leukemia (PCL). Initiated trials include an investigator-initiated trial for R/R MM or PCL in China (NCT05838131), and an investigator-initiated trial for newly diagnosed multiple myeloma (NDMM) in China (NCT06407947).
About CARsgen Therapeutics Holdings Limited
CARsgen is a biopharmaceutical company focusing on developing innovative CAR T-cell therapies to address the unmet clinical needs including but not limited to hematologic malignancies, solid tumors and autoimmune diseases. CARsgen has established end-to-end capabilities for CAR T-cell research and development covering target discovery, preclinical research, product clinical development, and commercial-scale production. CARsgen has developed novel in-house technologies and a product pipeline with global rights to address challenges faced by existing CAR T-cell therapies. Efforts include improving safety profile, enhancing the efficacy in treating solid tumors, and reducing treatment costs, etc. CARsgen's mission is to be a global biopharmaceutical leader that provides innovative and differentiated cell therapies for patients worldwide and makes cancer and other diseases curable.
Forward-looking Statements
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