NEWS

The 30th Annual Congress of the European Hematology Association (EHA) for the year 2025 commenced with great ceremony on June 12th, local time, in Milan, Italy. At this event, Bioheng Therapeutics presented the latest clinical data on its universal CAR-T product, CTD402, for the treatment of patients with relapsed or refractory (R/R) T-cell acute lymphoblastic leukemia/lymphoma (T-ALL/LBL), in the form of an oral report. The research data indicates that CTD402 not only achieves a high initial remission rate and a high rate of MRD negativity, but also provides long-term survival benefits for patients following consolidative transplantation, with a favorable safety profile.

The report at this EHA Congress detailed the results of a multi-center, investigator-initiated Phase I/II pooled analysis of CTD402 for the treatment of R/R T-ALL/LBL. Between December 2021 and November 2023, a total of 62 patients with R/R T-ALL/LBL (including 46 with ALL and 16 with LBL) were enrolled and received treatment. The median age of the patients was 23 years, and they had received a median of 3 prior lines of therapy. The median baseline blast percentage in the bone marrow of the patients was 45.1%, and 58.1% (36 out of 62) of the patients presented with extramedullary disease (EMD).

Safety

CTD402 demonstrated a favorable safety profile in this study:

• Cytokine Release Syndrome (CRS): The overall incidence was 79.0%, but cases were predominantly low-grade. The incidence of Grade 3 or higher CRS was only 4.8% (3/62).
• Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS): Only 2 cases were observed.
• Graft-versus-Host Disease (GVHD): Only 2 cases were observed.
• Other Adverse Events (AEs): The most common Grade 3 or higher AEs were hematologic toxicities. The incidence of infection events was low.

Efficacy

As of March 13, 2025, the median follow-up time was 18.4 months.

In the 59 evaluable patients overall, the efficacy outcomes were positive:

• Response Rate: The complete remission (CR) rate was 64.4% (38/59), and the bone marrow/peripheral blood remission rate was 81.4% (48/59). For the 34 patients with baseline extramedullary disease (EMD), the extramedullary remission rate was 50% (17/34).
• Depth and Duration of Remission: Among patients who achieved CR, 94.7% (36/38) achieved minimal residual disease (MRD) negativity. The median duration of remission (DOR) was 16.3 months (95% CI, 5.0-NE).

In the Recommended Phase 2 Dose (RP2D, 4×108 CAR-T cells, n=25) cohort, superior efficacy was observed:

• In this group, the CR rate reached 68.0% (17/25), the bone marrow/peripheral blood remission rate was 80.0% (20/25), and the EMD remission rate was 57.1% (8/14).
• Notably, the median DOR for this dose group has not yet been reached (95% CI, 8.8-NE), indicating potential for durable remission.

Long-term Benefit: Consolidative Transplantation Significantly Extends Survival

For patients who achieve remission, the subsequent treatment strategy is crucial. The study found that among the 38 patients who achieved a complete remission (CR), 22 (58%) received consolidative hematopoietic stem cell transplantation (HSCT). Compared to patients who did not undergo transplantation, the median overall survival (OS) and median leukemia-free survival (LFS) of the transplant group were both significantly prolonged (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 provides long-term survival benefits for these patients.

For more information about the oral presentation and the EHA Annual Congress, please visit the official EHA website.

https://congress-apps.ehaweb.org/eha2025/en-GB/pag/presentation/566395

About CTD402

CTD402 is an allogeneic, CD7-targeting Universal CAR (UCAR) T-cell therapy product derived from healthy donors for the treatment of T-ALL/LBL and other T-cell hematological malignancies. The product is gene-edited to prevent fratricide, graft-versus-host disease (GvHD), and host-versus-graft (HvG) rejection, while simultaneously enhancing its anti-tumor activity. CTD402 can be manufactured from a single batch to treat multiple patients, providing an "off-the-shelf" solution for patients in need of CAR-T cell therapy.