The 22nd International Myeloma Society (IMS) Annual Meeting in 2025 took place from September 17 to 20 in Toronto, Canada. Preliminary statistics show that Chinese experts have contributed 5 studies selected for Oral Presentations and over 80 studies for Poster Presentations this year, fully demonstrating the international influence of Chinese hematology research.
At this conference, Professor Qiu Lugui from the Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences, presented a study titled “Three-Year Follow-Up of FUMANBA-1: a Phase 1b/2 Study of Fully Human Anti-BCMA CAR-T Equecabtagene Autoleucel in Patients with Relapsed/Refractory Multiple Myeloma” (Abstract Number: OA-08).
The FUMANBA-1 study is a multicenter, single-arm Ib/II trial designed to evaluate the long-term safety and efficacy of Eque-cel in heavily pretreated relapsed/refractory multiple myeloma (R/R MM) patients, including those with high-risk features, providing evidence-based insights for optimizing R/R MM treatment strategies.
Background
Multiple myeloma (MM) is a malignant plasma cell disorder. Despite diverse existing treatment options, most patients still experience relapse, and some further progress to a relapsed/refractory state, highlighting the urgent need for safer and more effective treatment options. Previous studies have shown that Equecabtagene Autoleucel (Eque-cel), a BCMA-targeted CAR-T cell therapy, holds great potential for treating relapsed/refractory multiple myeloma (R/R MM). This therapy has been approved in China for the treatment of adult R/R MM patients who have received at least three prior lines of therapy. However, the efficacy and safety of Eque-cel in R/R MM still require further clinical investigation.
Study Methods and Key Results
1. Study Methods
- This was a multicenter, single-arm Ib/II trial. As of December 31, 2024, 109 R/R MM patients were enrolled. Patients underwent three consecutive days of lymphodepletion therapy (cyclophosphamide 500 mg/m² combined with fludarabine 30 mg/m²), followed by a single infusion of CAR-T cells at a dose of 1×10⁶ cells/kg body weight.
- Efficacy endpoints included overall response rate (ORR), complete response/stringent complete response rate (CR/sCR), progression-free survival (PFS), overall survival (OS), minimal residual disease (MRD) negativity rate, and duration of MRD negativity. Safety endpoints included the incidence and severity of cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), and others.
2. Study Results
- In terms of efficacy, among 107 evaluable patients, the ORR was 96.3%, with a CR/sCR rate of 83.2%. Among CAR-T-naïve patients, the ORR and CR/sCR rates were 98.9% and 88.4%, respectively. The MRD negativity rate was 95.3%, with a median duration of MRD negativity of 36.5 months.
- Among the 109 patients treated with Eque-cel, the median PFS was 30.5 months, while in CAR-T-naïve patients, the median PFS extended to 35.9 months. The median OS has not yet been reached.
- Regarding safety, the incidence of CRS was 93.6%, with only one case being grade ≥3. The median time to onset of CRS was 6 days (range: 1–13 days), and the median duration was 5 days (range: 2–30 days). Only two patients experienced ICANS, both of grade 1–2. No delayed neurotoxicity or secondary malignancies were observed.
- Pharmacokinetic analysis showed that CAR transgene persistence was detected in 52% (39/75) and 37.5% (21/56) of patients at 12 and 24 months, respectively. The incidence of anti-CAR antibodies was 24.8%.
This multicenter, single-arm Ib/II trial aimed to investigate the safety and efficacy of Eque-cel in heavily pretreated R/R MM patients, enrolling 109 patients. The results demonstrate that Eque-cel was well-tolerated, with the main adverse events being low-grade cytokine release syndrome and a low incidence of immune effector cell-associated neurotoxicity syndrome. Additionally, it exhibited potent and durable antitumor activity, with an overall response rate of 96.3%, and 95.3% of patients achieved minimal residual disease negativity, with a median duration of MRD negativity exceeding 36 months. This study suggests that Eque-cel offers a promising new treatment option for heavily pretreated R/R MM patients, including those with high-risk features.
