On November 3, new clinical data were released for a universal BCMA-targeted CAR-T therapy candidate (CT0596) in patients with relapsed/refractory multiple myeloma (R/R MM). The results show favorable preliminary safety and encouraging efficacy outcomes.
About the Study
CT0596 is a universal CAR-T cell therapy targeting BCMA, currently under evaluation in an investigator-initiated trial (NCT06718270) for malignant plasma cell disorders. The study is in the dose-expansion phase, exploring higher cell doses to determine the recommended dose.
As of June 24, 2025, eight patients with R/R MM were enrolled during the dose-escalation phase. The median number of prior therapy lines was 4.5 (range: 3–9). Five had received triple-class therapy (PI, IMiD, and anti-CD38 mAb), and five had undergone autologous stem cell transplantation. The CAR-T doses were 1.5×10⁸ (1 patient), 3×10⁸ (5 patients), and 4.5×10⁸ (2 patients).
Efficacy and Safety
All eight patients were evaluable for efficacy, with a median follow-up of 2.56 months (range: 0.9–5.9 months).
- Responses: 5 patients achieved partial response (PR) or better.
- 3 achieved complete/stringent complete response (CR/sCR)
- 1 achieved PR
- 1 achieved very good partial response (VGPR)
- MRD negativity: 6 patients achieved MRD-negative status by week 4.
Durability: No disease progression observed; one patient maintained sCR and MRD negativity for nearly six months.
All patients showed CAR-T expansion. No dose-limiting toxicity, treatment discontinuation, or deaths occurred. Four patients experienced grade 1 cytokine release syndrome (CRS), all resolving within 2–10 days.
Background
In October 2025, early results in two patients with relapsed/refractory primary plasma cell leukemia (pPCL) showed both achieved sCR after CT0596 treatment, highlighting its potential even in highly aggressive disease.
