inaticabtagene autoleucel 治疗成人复发/难治性 B 细胞急性淋巴细胞白血病

Before November 2023, CD19 chimeric antigen receptor (CAR) T-cell therapies had not been approved in China for patients with relapsed or refractory B-cell acute lymphoblastic leukemia (R/R B-ALL), leaving a significant unmet need. In response, inaticabtagene autoleucel (Inati-cel), a novel CD19 CAR T-cell therapy with a distinct single-chain variable fragment (HI19 ), was developed and showed promising efficacy in preliminary clinical research.

We conducted a phase 2, single-arm, multicenter study of Inati-cel in adult CD19+ R/R B-ALL in China. The primary end point was the overall remission rate (ORR) at the end of month 3. Forty-eight patients who underwent Inati-cel infusion were evaluated for both efficacy and safety. Among them, 34 patients achieved and maintained remission beyond 3 months, with a 3-month ORR of 70.

8% (95% confidence interval [CI], 55. 9-83. 1). The best ORR was 85. 4%, with all responders reaching minimal residual disease negativity. With a median follow-up of 23. 7 months, the median duration of remission was 20. 7 months (95% CI, 6. 4 to not reached), and the median overall survival was not reached (95% CI, 13. 0 months to not reached).

Additionally, grade 3 cytokine release syndrome and neurologic events occurred in 12. 5% and 6. 2% of patients, respectively. The 2-year follow-up data suggest that Inati-cel demonstrates encouraging and durable responses with manageable safety profiles in R/R B-ALL. Based on the data from this pivotal trial, Inati-cel was approved as the first CAR T-cell therapy for adult R/R B-ALL in China and underscores its potential therapeutic benefits for this patient population. This trial was registered at www. ClinicalTrials. gov as #NCT04684147.