SHANGHAI, Dec. 7, 2025 /PRNewswire/ — CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on developing innovative CAR T-cell therapies, announced that clinical data from its allogeneic BCMA-targeted CAR T-cell product candidate, CT0596, for the treatment of relapsed/refractory multiple myeloma (R/R MM) was presented in a poster at the 67th American Society of Hematology (ASH) Annual Meeting. The poster was titled “A First-in-Human Study of CT0596, an Allogeneic CAR T-Cell Therapy Targeting BCMA, in Patients with Relapsed/Refractory Multiple Myeloma.” The publication number was 2296.
This clinical trial (NCT06718270) enrolled 8 patients with R/R MM in the dose-escalation phase who received CT0596 infusion. The median number of prior lines of therapy was 4.5 (range: 3-9). Enrollment was not restricted by NKG2A expression levels. Regarding lymphodepletion, 6 patients received full-dose lymphodepletion with fludarabine (30 mg/m²/day) and cyclophosphamide (500 mg/m²/day) for 3 consecutive days, while 2 patients received reduced-dose lymphodepletion. CT0596 was administered at dose levels of 1.5×10⁸ (n=1), 3×10⁸ (n=5), and 4.5×10⁸ CAR-T cells (n=2), with one patient receiving two infusions.
As of August 31, 2025, all 8 infused patients were evaluable for efficacy, with a median follow-up of 4.14 months (range: 0.9-7.9 months). Six patients achieved a partial response (PR) or better: 3 achieved complete response/stringent complete response (CR/sCR) (all in the full-dose lymphodepletion group), 1 achieved very good partial response (VGPR), and 2 achieved PR. Among the 6 patients who received full-dose lymphodepletion, 5 achieved PR or better. Six patients in the full-dose lymphodepletion group achieved minimal residual disease (MRD)-negativity at Week 4. Patient 01 maintained ongoing sCR and MRD-negativity as of Month 8. Patient 04 achieved PR with resolution of extramedullary disease following the second infusion. CAR-T cell expansion was observed in all 8 patients. Among the two patients who received the 4.5×10⁸ dose, one achieved sCR, and the other exhibited deepening response to VGPR.
CT0596 demonstrated a manageable safety profile. Four patients experienced Grade 1 cytokine release syndrome (CRS); no Grade 2 or higher CRS was observed. No immune effector cell-associated neurotoxicity syndrome (ICANS) or graft-versus-host disease (GVHD) was reported. No dose-limiting toxicities, treatment discontinuations, or deaths were observed.
The study is still in the dose-exploration phase. The lymphodepletion regimen has been determined, and higher cell doses are being explored to further define the recommended dose (RD). The company plans to initiate a Phase 1b registrational study for CT0596 in 2026.
About CT0596
CT0596 is an allogeneic BCMA-targeted CAR-T therapy developed using CARsgen’s proprietary THANK-u Plus™ platform. It is currently being evaluated in investigator-initiated trials for relapsed/refractory multiple myeloma (R/R MM) or plasma cell leukemia (PCL). CT0596 demonstrated preliminary favorable tolerability and encouraging efficacy signals. Further investigation is planned in additional plasma cell malignancies and autoimmune diseases mediated by autoreactive plasma cells. The company anticipates submitting an Investigational New Drug (IND) application in the second half of 2025.
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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