CARsgen Therapeutics Holdings Limited  a company focused on developing innovative CAR T-cell therapies, announces preliminary clinical data for CT0596, an allogeneic BCMA-targeted CAR-T developed using the THANK-u Plus™ platform. CT0596 is currently being evaluated in an early exploratory clinical study for relapsed/refractory multiple myeloma (R/R MM) or relapsed/refractory plasma cell leukemia (R/R PCL) to assess its safety, and preliminary efficacy.

As of May 6, 2025, 8 patients with R/R MM who had received at least three prior lines of therapy were enrolled and infused with CT0596 following lymphodepletion with the FC regimen (fludarabine 22.5-30 mg/m² and cyclophosphamide 350-500 mg/m²). Key findings from up to four months of follow-up include:

• No dose-limiting toxicities (DLTs), ≥Grade 3 cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), or graft-versus-host disease (GVHD) were reported. The product demonstrated a favorable safety profile, with no patients discontinuing treatment due to adverse events.
• 1) Among 5 patients who completed the first efficacy assessment at Week 4, 3 patients (60%) achieved stringent complete response/complete response (sCR/CR), and 4 patients (80%) achieved minimal residual disease (MRD)-negativity in the bone marrow. 2) Early efficacy data from 2 patients at Day 14 showed reductions in measurable lesions by ≥92% and ≥65%, respectively. 3) 1 patient had not yet reached the protocol-specified efficacy assessment timepoint.
• All sCR/CR patients remained in ongoing responses, including the first patient who completed the four-month follow-up.

Based on the preliminary safety and efficacy data, CT0596 demonstrated favorable tolerability and encouraging efficacy signals in R/R MM patients across all predefined dose levels, with CAR-T expansion observed. These findings warrant further exploration not only in R/R MM, but also in other plasma cell malignancies and autoimmune diseases mediated by autoreactive plasma cells. CARsgen plans to present detailed clinical data at upcoming scientific conferences. The company anticipates submitting an Investigational New Drug (IND) application for this product candidate in the second half of 2025.