EHA 2025 Highlights Breakthroughs in Cell Therapy: JNJ-4496, CT071, and CTD402 Demonstrate High Efficacy in Hematologic Malignancies

The 30th Annual Meeting of the European Hematology Association (EHA) in 2025 was grandly convened on June 12 local time in Milan, Italy.

At the conference, multiple CAR-T cell therapies demonstrated breakthrough potential in anti-tumor treatment.

Johnson & Johnson's CD19/CD20 Dual-Target CAR-T Achieves 100% Objective Response Rate

Johnson & Johnson Discloses Clinical Data for the First Time on CD19/CD20 Dual-Target CAR-T Therapy JNJ-90014496 (JNJ-4496).

In a Phase 1b trial involving patients with relapsed or refractory large B-cell lymphoma, 22 patients who received the recommended Phase 2 dose (RP2D, i.e., 75 million CAR+T cells) were included in the efficacy analysis (median follow-up of 4 months). Among them, 10 patients who had received one prior treatment achieved an objective response rate (ORR) of 100%, with 8 patients showing complete disappearance of cancer signs, resulting in a complete response rate (CRR) of 80%. For the 12 patients who had received two or more prior treatments, the ORR was 92%, and the complete response rate was 75%.

In terms of safety, 84% of the 25 patients in the RP2D safety group experienced grade 3 or 4 treatment-related adverse events, and 28% experienced serious TEAEs. No grade 3 or 4 cytokine release syndrome occurred in the cohort. One patient developed grade 1 immune effector cell-associated neurotoxicity syndrome (ICANS), and another patient with central nervous system lymphoma developed grade 3 ICANS.

Notably, this drug was acquired by Johnson & Johnson in 2023 from AbelZeta (CBMG). According to the agreement, Johnson & Johnson paid an upfront fee of $245 million to obtain exclusive rights outside of China for JNJ-4496 (known as C-CAR039 in China) and another CD20-targeted CAR-T therapy, C-CAR066.

Promising Prospects for CARsgen's GPRC5D CAR-T in NDMM Patients

CARsgen Pharma Announces Preliminary Results from Phase I Study of GPRC5D-Targeted CAR-T Cell Therapy CT071 in High-Risk Newly Diagnosed Multiple Myeloma (NDMM).

As of January 2, 2025, a total of 10 patients had received CT071 infusion, and none of the patients had received bridging therapy. The median follow-up time was 3.4 months (range: 1.8 to 5.9 months).

In terms of safety, 7 patients (70%) experienced grade 1 cytokine release syndrome (CRS), all of whom have recovered. No dose-limiting toxicity (DLT), immune effector cell-associated neurotoxicity syndrome (ICANS), or treatment-related adverse events (TRAE) leading to death occurred.

In terms of efficacy, the overall response rate (ORR) was 100%, with 7 cases (70%) achieving stringent complete response (sCR), 2 cases (20%) achieving very good partial response (VGPR), and 1 case (10%) achieving partial response (PR). Notably, 5 patients reached sCR by the 4th week. All 10 patients achieved minimal residual disease (MRD) negativity at the 10-6 threshold.

Preliminary results indicate that CT071 can induce deep remission in high-risk newly diagnosed multiple myeloma patients, with a favorable safety profile, warranting further clinical research evaluation.

North Heng Bio's Universal CAR-T Product Brings Long-Term Survival Benefits

Betah Bio announced the Phase I/II pooled analysis results of its off-the-shelf CAR-T product CTD402 for the treatment of relapsed or refractory (R/R) T-ALL/LBL patients in an oral presentation.

From December 2021 to November 2023, a total of 62 patients with R/R T-ALL/LBL (including 46 cases of ALL and 16 cases of LBL) were enrolled and received treatment.

In terms of safety, CTD402 demonstrated favorable safety characteristics. Cytokine Release Syndrome (CRS): The overall incidence rate was 79.0%, but predominantly low-grade, with the incidence of Grade 3 or higher CRS being only 4.8% (3/62). Neurotoxicity (ICANS): Only 2 cases were observed. Graft-versus-Host Disease (GVHD): Only 2 cases were observed. Other adverse events: The most common ≥Grade 3 AE was hematological toxicity, with a low incidence of infection events.

In terms of efficacy, as of March 13, 2025, the median follow-up time reached 18.4 months. Among 59 evaluable patients in total, the complete response (CR) rate was 64.4% (38/59), and the bone marrow/peripheral blood response rate was 81.4% (48/59). For the 34 patients with baseline extramedullary lesions, the extramedullary response rate was 50% (17/34). Depth and durability of response: Of the patients achieving CR, 94.7% (36/38) achieved minimal residual disease (MRD) negativity. The median duration of response (DOR) was 16.3 months (95% CI, 5.0-NE).

In the recommended Phase II dose (RP2D, 4×10^8 CAR-T cells, n=25) group, superior efficacy was observed. The CR rate in this group reached 68.0% (17/25), the bone marrow/peripheral blood response rate was 80.0% (20/25), and the extramedullary lesion response rate was 57.1% (8/14). Importantly, the median DOR in this dose group has not yet been reached (95% CI, 8.8-NE), demonstrating the potential for durable responses.

Long-term Benefits: Among the 38 patients who achieved CR, 22 (58%) underwent consolidative hematopoietic stem cell transplantation. Compared with those who did not receive transplantation, the median overall survival (OS) and median leukemia-free survival (LFS) were significantly prolonged in the transplantation group (OS: 29.4 months vs 8.7 months, p=0.011; LFS: 29.4 months vs 3.9 months, p<0.001), indicating that consolidative transplantation can provide long-term survival benefits for patients.

According to a press release from Beiheng Bio, CTD402 is an allogeneic CAR-T (UCAR-T) cell therapy product targeting CD7 derived from healthy donors. The product avoids fratricide, graft-versus-host disease (GvHD), and host-versus-graft rejection (HvG) through genetic modification, while enhancing anti-tumor activity. CTD402 allows for single-batch production for multiple patients, providing an "off-the-shelf" solution for patients in need of CAR-T cell therapy. In the second half of 2025, Beiheng Bio will initiate U.S. registration clinical trials for this product, enrolling pediatric and adult patients with R/R T-ALL/LBL.

Editor: Liuli

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