多发性骨髓瘤 (MM) 是一种表达 B 细胞成熟抗原 (BCMA) 的浆细胞恶性肿瘤。 Elranatamab 是一种双特异性抗体，可结合 MM 上的 BCMA 和 T 细胞上的 CD3。 MagnetisMM-1 试验评估了其安全性、药代动力学和功效。达到了主要终点，包括剂量限制性毒性的发生率以及客观缓解率（ORR）和缓解持续时间（DOR）。次要疗效终点包括无进展生存期（PFS）和总生存期（OS）。 88 名复发或难治性 MM 患者接受了 elranatamab 单药治疗，55 名患者接受了有效剂量的 elranatamab。患者之前平均接受过五种治疗方案； 90.9% 为三级难治性，29.1% 具有高细胞遗传学风险，23.6% 之前接受过 BCMA 定向治疗。在剂量递增过程中未观察到剂量限制性毒性。不良事件包括血细胞减少和细胞因子释放综合征。暴露量与剂量成正比。中位随访时间为 12.0 个月，ORR 为 63.6%，达到完全缓解或更好的患者为 38.2%。对于应答者来说，中位 DOR 为 17.1 个月。所有 13 名可评估微小残留病的患者均呈阴性。即使在之前的 BCMA 定向治疗之后，53.8% 的患者也取得了缓解。对于所有 55 名患者，中位 PFS 为 11.8 个月，中位 OS 为 21.2 个月。 Elranatamab 为 MM 患者实现了持久的缓解、可控的安全性和有希望的生存。 ClinicalTrials.gov 标识符：NCT03269136 .© 2023。作者。

Multiple myeloma (MM) is a plasma cell malignancy expressing B cell maturation antigen (BCMA). Elranatamab, a bispecific antibody, engages BCMA on MM and CD3 on T cells. The MagnetisMM-1 trial evaluated its safety, pharmacokinetics and efficacy. Primary endpoints, including the incidence of dose-limiting toxicities as well as objective response rate (ORR) and duration of response (DOR), were met. Secondary efficacy endpoints included progression-free survival (PFS) and overall survival (OS). Eighty-eight patients with relapsed or refractory MM received elranatamab monotherapy, and 55 patients received elranatamab at efficacious doses. Patients had received a median of five prior regimens; 90.9% were triple-class refractory, 29.1% had high cytogenetic risk and 23.6% received prior BCMA-directed therapy. No dose-limiting toxicities were observed during dose escalation. Adverse events included cytopenias and cytokine release syndrome. Exposure was dose proportional. With a median follow-up of 12.0 months, the ORR was 63.6% and 38.2% of patients achieving complete response or better. For responders, the median DOR was 17.1 months. All 13 patients evaluable for minimal residual disease achieved negativity. Even after prior BCMA-directed therapy, 53.8% achieved response. For all 55 patients, median PFS was 11.8 months, and median OS was 21.2 months. Elranatamab achieved durable responses, manageable safety and promising survival for patients with MM. ClinicalTrials.gov Identifier: NCT03269136 .© 2023. The Author(s).