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
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, https://www.carsgen.com. 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.

 

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