A phase 1/1b study of the BCMA-targeting bispecific T-cell engager pavurutamab for relapsed/refractory multiple myeloma
Details
Publication Year 2026-07-09,Volume 148,Issue #2,Page 199-212
Journal Title
Blood
Publication Type
Research article
Abstract
The B-cell maturation antigen (BCMA)-targeting bispecific T-cell engager pavurutamab (AMG 701) directs the cytotoxic T-cell response toward multiple myeloma (MM) cells. This phase 1/1b, open-label, dose-exploration and dose-expansion study evaluated the safety, tolerability, and efficacy of pavurutamab monotherapy in patients with triple-class relapsed/refractory MM (RRMM). Pavurutamab (5-18 000 μg) was administered IV weekly with step-up dosing in week 1. Overall, 172 patients received at least 1 dose of pavurutamab; 73 received the recommended phase 2 dose (RP2D; 18 000 μg), with 2 different step-up dosing regimens in phase 1b. Twelve patients had dose-limiting toxicities, which included cytokine release syndrome (CRS) and increased transaminases; none of which occurred at the RP2D. The most frequently reported treatment-emergent adverse events included CRS (74.4%), anemia (61.0%), neutropenia (47.1%), and hypophosphatemia (45.3%). Grade ≥3 infections were noted in 60 patients (34.9%). The overall response rate (ORR) was 46.5% among all patients and 65.8% (very good partial response [VGPR] or better, 60.3%) among 73 patients treated at the RP2D. At median follow-up of 17.2 months, median duration of response (DOR) was 36.6 months (95% confidence interval [CI], 22.3 to not estimable). Median progression-free survival (PFS) for all patients was 5.5 months (95% CI, 2.8-10.1) and 16.8 months (95% CI, 5.0 to not estimable) with the RP2D. Pavurutamab exposure generally increased in a dose-proportional manner, with high soluble BCMA levels correlating with lower exposure level. The acceptable safety profile, pharmacokinetics, and preliminary efficacy of pavurutamab supports anti-BCMA T-cell engager therapy in heavily pretreated patients with RRMM. This trial was registered at www.clinicaltrials.gov as NCT03287908.
Publisher
American Society of Hematology
Keywords
Humans; *Multiple Myeloma/drug therapy/pathology/immunology/mortality; Female; *Antibodies, Bispecific/therapeutic use/adverse effects/administration &; dosage/pharmacokinetics; *B-Cell Maturation Antigen/antagonists & inhibitors/immunology; Male; Middle Aged; Aged; Aged, 80 and over; Adult; Neoplasm Recurrence, Local/drug therapy; *Antineoplastic Agents/therapeutic use/adverse effects; T-Lymphocytes/immunology; Recurrence
Department(s)
Haematology
Open Access at Publisher's Site
https://doi.org/10.1182/blood.2025032044
Terms of Use/Rights Notice
Refer to copyright notice on published article.


Creation Date: 2026-04-28 05:52:36
Last Modified: 2026-08-04 04:43:07
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