At the 2025 Annual Meeting of the American Society of Hematology (ASH), IASO Bio presented updated clinical data for equecabtagene autoleucel, its fully human anti-BCMA CAR-T cell therapy, in patients with high-risk newly diagnosed multiple myeloma (NDMM) who were ineligible for transplantation.
The data, presented in an oral session on December 6, showed that equecabtagene autoleucel produced deep and durable responses in this high-risk patient population, with a manageable safety profile.
FUMANBA-2: Updated Clinical Data
The presentation was based on Abstract #8596, titled:
“Efficacy and Safety of Fully Human BCMA CAR-T Cell Therapy in Transplant-Ineligible Patients with High-Risk Newly Diagnosed Multiple Myeloma: Updated Data from the Open-Label, Single-Arm Phase 1 FUMANBA-2 Study.”
The primary endpoints of the study were minimal residual disease (MRD) negativity and progression-free survival (PFS).
As of May 13, 2025, a total of 16 patients with high-risk NDMM had received an infusion of equecabtagene autoleucel. Eleven patients had double-hit disease, while two had triple-hit disease. Two patients had both R-ISS stage III disease and double-hit features, and one had R-ISS stage III disease with triple-hit features. Four patients had extramedullary disease.
After a median follow-up of 27.04 months, median PFS had not yet been reached. The estimated PFS rates at 12, 18 and 24 months were 87.5%, 80.2% and 74.5%, respectively.
All patients achieved MRD negativity within one month after infusion, and 80% remained MRD-negative for more than 24 months. The objective response rate (ORR) was 100%, with 93.8% achieving a stringent complete response (sCR).
Manageable Safety Profile
Following infusion, 11 patients (68.8%) experienced cytokine release syndrome (CRS). All CRS events were grade 1 or 2. The median time to CRS onset was seven days, with a median duration of three days.
No neurotoxicity or immune effector cell-associated neurotoxicity syndrome (ICANS) was observed.
Potential for Earlier Use in High-Risk Myeloma
The updated FUMANBA-2 results presented at ASH are encouraging, particularly given the high-risk characteristics of the patients enrolled in the study.
Among all enrolled high-risk patients, equecabtagene autoleucel achieved a 93.8% stringent complete response rate, while 80% maintained MRD negativity for more than 24 months. The treatment also demonstrated sustained disease control in patients with particularly aggressive disease features, including double-hit or triple-hit disease, R-ISS stage III disease, and extramedullary disease.
The 24-month PFS rate reached 74.5%, suggesting that equecabtagene autoleucel may have the potential to become an earlier-line treatment option capable of inducing deep responses following induction therapy in high-risk NDMM.
“We are pleased to present the encouraging progress of equecabtagene autoleucel in patients with high-risk NDMM at the ASH Annual Meeting,” said IASO Bio. “The latest FUMANBA-2 data further support the ability of this therapy to provide deep and durable responses in this patient population. No ICANS was observed, and all CRS events were limited to grade 1 or 2. We will continue long-term follow-up of the study and look forward to bringing this innovative therapy to more patients in need at an earlier stage of treatment.”
The Growing Global Burden of Multiple Myeloma
Multiple myeloma continues to represent a significant global health burden. The number of newly diagnosed MM cases worldwide increased from approximately 164,300 in 2019 to 197,200 in 2024, and the number is projected to reach approximately 273,600 by 2040.
In China, annual new cases increased from approximately 28,100 in 2019 to 31,800 in 2024. It is estimated that 30–40% of patients will eventually reach third-line treatment, highlighting the ongoing need for effective treatment options that can deliver deep and durable responses earlier in the course of disease.
With its high response rates, sustained MRD negativity and encouraging PFS outcomes in a particularly high-risk NDMM population, the FUMANBA-2 findings add to the growing clinical evidence surrounding BCMA-directed CAR-T cell therapy in multiple myeloma.
Reference
