CARsgen Therapeutics Announces Promising Preliminary Data for Allogeneic BCMA CAR-T Therapy CT0596 in Relapsed/Refractory Multiple Myeloma

SHANGHAI – May 11, 2025 – CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on innovative CAR T-cell therapies, has announced preliminary clinical data for CT0596, an allogeneic BCMA-targeted CAR-T developed using their THANK-u Plus™ platform. This early exploratory clinical study is evaluating the safety and preliminary efficacy of CT0596 in patients with relapsed/refractory multiple myeloma (R/R MM) or relapsed/refractory plasma cell leukemia (R/R PCL).

As of May 6, 2025, eight R/R MM patients who had received at least three prior lines of therapy were enrolled and infused with CT0596 following lymphodepletion. Key findings from up to four months of follow-up include:

• Favorable Safety Profile: 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. No patients discontinued treatment due to adverse events.
• Encouraging Early Efficacy:

  • 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.
  • Early efficacy data from 2 patients at Day 14 showed reductions in measurable lesions by ≥92% and ≥65%, respectively.
  • 1 patient had not yet reached the protocol-specified efficacy assessment timepoint.

• Durable Responses: All sCR/CR patients remained in ongoing responses, including the first patient who completed the four-month follow-up.

Based on these preliminary safety and efficacy data, CT0596 demonstrated favorable tolerability and encouraging efficacy signals in R/R MM patients across all tested dose levels, with observed CAR-T expansion. CARsgen believes these findings warrant further investigation in R/R MM, other plasma cell malignancies, and autoimmune diseases mediated by autoreactive plasma cells. The company plans to present detailed clinical data at upcoming scientific conferences and anticipates submitting an Investigational New Drug (IND) application for CT0596 in the second half of 2025.