Safety and efficacy of AZD0120, a BCMA/CD19 dual-targeting CAR T-cell therapy, in relapsed/refractory multiple myeloma: Preliminary Results from the DURGA-1 Phase 1b/2 study

作者：Shambavi Richard, Mahmoud Gaballa, Tara Gregory, Saurabh Chhabra, Larry D. Anderson, Luciano da Fontoura Costa, Caitlin Costello, Scott Goldsmith, Doris Hansen, Sridevi Rajeeve, Shaji Kumar, Aravind Ramakrishnan, Minoo Battiwalla, Ajay K. Nooka, Hira Shaikh, Meiyue Grace Hong, Steven Wang, Patricia Cheung, Liang Li, Binod Dhakal · 发表于：Blood · 年份：2025 · DOI：10.1182/blood-2025-269 · 被引用次数：8 · 研究领域：CAR-T cell therapy research、Multiple Myeloma Research and Treatments、Protein Degradation and Inhibitors

Abstract Introduction: Multiple myeloma (MM) accounts for nearly 20% of all hematologic cancers in the US and remains a substantial clinical burden, with patients (pts) requiring multiple lines of therapy (LOT). While advances such as BCMA-directed CAR T-cell therapy have improved outcomes, challenges persist, including disease relapse, treatment toxicities (CRS, ICANS, and non-ICANS neurotoxicities), and access. AZD0120 (formerly GC012F) is a first-in-class autologous BCMA/CD19 dual-targeting CAR T-cell therapy using the FasTCAR rapid manufacturing platform that preserves the naive and central memory T-cell phenotypes with marked in vivo proliferative capacity. Phase 1 investigator-initiated trials in China demonstrated deep, durable responses and a favorable safety profile following single-infusion AZD0120 in newly diagnosed and relapsed/refractory MM (RRMM) (Du J, et al. EHA 2024 and ASCO 2023). Here, we report for the first time preliminary phase 1b results from DURGA-1, an ongoing phase 1b/2 clinical trial evaluating the safety and efficacy of AZD0120 in pts with RRMM. Methods: This open-label, single-arm, US-based, multicenter study (NCT05850234) evaluated the safety/tolerability of 2 dose levels (DL) of AZD0120 in pts with RRMM along with preliminary efficacy. Eligible ptswere aged ≥18 y with RRMM (3+ prior LOT [pLOT] including a proteasome inhibitor, immunomodulatory drug, and anti-CD38 antibody), ECOG PS 0–1, and documented evidence of progressive disease. Exposure t...