SHANGHAI, China, June 16, 2025, 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 newly diagnosed multiple myeloma (NDMM) in an investigator-initiated trial (NCT06407947), were presented in a poster session at the 30th European Hematology Association (EHA) Congress. The poster was titled “A phase I study of GPRC5D targeting CAR T-cell therapy CT071 for high-risk newly diagnosed multiple myeloma” (Abstract # PF1164).
This single-arm, single-center, open-label exploratory clinical trial aimed to evaluate the safety and preliminary efficacy of CT071 in patients with high-risk (HR) newly diagnosed multiple myeloma (NDMM). As of January 2, 2025, 10 patients had received CT071 infusion. Of these, 6 (60%) patients had high-risk cytogenetics, 3 (30%) had EMD, and 7 (70%) were at R2-ISS III or IV. Bridging therapy was not required for any of the patients. The median follow-up time was 3.4 months (range 1.8 to 5.9).
Regarding safety: 7 patients (70%) experienced cytokine release syndrome (CRS), all at Grade 1 and resolved. No dose-limiting toxicities (DLTs), immune effector cell-associated neurotoxicity syndrome (ICANS), or death due to treatment-related adverse event (TRAE) occurred.
Regarding efficacy: The overall response rate (ORR) was 100%, including stringent complete response (sCR) rate of 70% (7/10). Two (20%) patients achieved very good partial response (VGPR) and one (10%) achieved partial response (PR). Notably, 5 patients achieved sCR by week 4. All 10 patients achieved MRD negativity at 10⁻6 threshold. A robust expansion of CAR-T cells was observed in all patients with a median Cmax of 54,812.0 copies/μg gDNA (range 9,885 to 82,600) and Tmax was 10 days (range 10 to 14).
Preliminary results indicate that CT071 induces deep responses in high-risk newly diagnosed multiple myeloma with a favorable safety profile, warranting further clinical investigation.
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 multiple myeloma or relapsed/refractory plasma cell leukemia (PCL). An investigator-initiated trial (NCT05838131) is ongoing in China to evaluate the preliminary safety and efficacy of CT071 for the treatment of relapsed/refractory multiple myeloma or plasma cell leukemia. Another investigator-initiated trial (NCT06407947) is ongoing in China for the treatment of newly diagnosed multiple myeloma (NDMM). CT071 received IND clearance from the FDA for the treatment of patients with relapsed/refractory MM or relapsed/refractory primary PCL.
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.
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