Use of real-world data/evidence in MM drug approvals
The role of real-world data/evidence (RWD/E) in regulatory applications was a key topic of discussion at the 2024 IMS-European Medicines Agency (EMA) Joint Meeting. An editorial published in Blood Cancer Journal digs deeper into this topic. A Special Interest Session at the 2025 American Society of Hematology (ASH) Annual Meeting (ASH 2025), chaired by IMS Board Member Saad Z. Usmani, MD, will explore RWD use in MM drug approvals.
Early data for novel in vivo CAR-T cell-generating MM therapy to be shared at ASH 2025
The Late-Breaking Abstracts Session at ASH 2025 will feature two MM studies. In addition to findings from the phase 3 MajesTec-3 trial, early data from MMyCAR, a first-in-human phase 1 study of KLN-1010 in patients with relapsed/refractory myeloma, will be presented. KLN-1010 is a novel in vivo gene therapy for generating BCMA-targeted CAR-T cells.
Scientific session at ASH 2025 will spotlight MM origins and evolution
At ASH 2025, a session chaired by Salomon Manier, MD, PhD, will track the biological and genetic trajectory of myelomagenesis. Multiple speakers will discuss various aspects of MM evolution; 2024 IMS Young Investigator Awardee Romanos Sklavenitis-Pistofidis, MD, will speak about “Early Molecular and Immune Deregulations in Premalignant Conditions.”
EMN recommendations on sequencing immunotherapies and functional high-risk MM
The European Myeloma Network (EMN) published a report in Hemaspehere outlining practical guidance on sequencing BCMA- and GPRC5D-directed immunotherapies. An EMN consensus statement on functional high-risk (FHR) MM—myeloma that relapses early and unexpectedly in the absence of baseline clinical/molecular risk factors—was also published recently, in the American Journal of Hematology.
Hong Kong Department of Health approves fully human anti-BCMA CAR-T therapy
Equecabtagene autoleucel (eque-cel), a fully human anti-BCMA CAR-T therapy, gained approval from the Hong Kong Department of Health. The approval was based on results of the FUMANBA-1 trial; 3-year follow-up data were presented at the 2025 IMS Annual Meeting. Eque-cel is approved in China (Mainland and Macau) and is under review in Singapore and Saudi Arabia.
Challenges persist in myeloma care in rural America
Managing Relapsed/Refractory Multiple Myeloma in Rural America
(MedPage Today) “The treatment landscape for relapsed and refractory multiple myeloma (RRMM) has changed dramatically over the past decade, resulting in more therapeutic options for various stages of relapse…However, obtaining the benefits of this clinical progress is contingent on access to the therapies, which remains uneven in the U.S.”
Overweight status predicts worse CAR-T therapy outcomes
Patients with relapsed/refractory myeloma who were overweight had worse outcomes with anti-BCMA CAR-T therapy, compared to those with normal weight or obesity, according to a retrospective analysis published in Blood Immunology & Cellular Immunotherapy. The authors noted that the “U-shaped relationship between BMI and CAR T-cell outcomes in MM” warrants further validation and mechanistic studies, to uncover potential targets or modifiable factors that may help boost the efficacy of CAR-T therapy.
