2026 IMS | IASO Bio Presented First-in-Human Data of BCMA-Targeted In Vivo CAR-T Cell Therapy IASO206

— Late-breaking oral presentation of first-in-human data (LBA-11) showed that a single intravenous infusion of IASO206 generated functional BCMA CAR-T cells in vivo without lymphodepleting chemotherapy. The study demonstrated a 90% objective response rate and a 90% MRD negativity rate in a population in which 90% of patients had high-risk and 70% had ultra-high-risk cytogenetic features; both rates reached 100% in the high-dose cohort.

SHANGHAI, NANJING, China and PLEASANTON, Calif., Sept. 26, 2026 /PRNewswire/ -- IASO Biotechnology ("IASO Bio"), a commercial-stage biopharmaceutical company focused on the discovery, development, manufacturing, and commercialization of innovative cell therapies and biologics for hematologic malignancies and autoimmune diseases, announced today the presentation of first-in-human (FIH) Phase 1 clinical data for IASO206, its internally developed BCMA-targeted in vivo CAR-T cell therapy, in patients with relapsed/refractory multiple myeloma (R/R MM) at the 2026 International Myeloma Society (IMS) Annual Meeting.

IASO206 is designed to generate functional BCMA-directed CAR-T cells directly in patients following a single intravenous infusion, eliminating the need for ex vivo cell manufacturing and lymphodepleting chemotherapy. The results were presented by Dr. Wenqiang Yan of the Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences, during a late-breaking oral session at the IMS Annual Meeting^[1] (Abstract No. LBA-11).

Patient Baseline and Study Design

This investigator-initiated trial (IIT) enrolled patients with R/R MM who had received at least two prior lines of therapy and had relapsed or refractory disease following treatment with proteasome inhibitors, immunomodulatory agents, and/or anti-CD38 antibodies.

As of September 26, 2026, a total of 10 patients had received IASO206. Patients had received a median of three prior lines of therapy (range, 2–8); 70% were triple-class exposed, 90% had high-risk cytogenetic abnormalities, and 70% had ultra-high-risk cytogenetic features.

Three dose levels were evaluated, expressed in transducing units (TU): 1×10⁸, 3×10⁸, and 9×10⁸ TU.

Robust In Vivo Expansion and Deep Clinical Responses

Pharmacokinetic analyses demonstrated robust expansion of CAR-positive cells in the absence of lymphodepleting chemotherapy. Peak vector copy number (VCN) occurred between Days 12 and 21 and most remained detectable beyond 60 days and up to six months after treatment.

Among the 10 efficacy-evaluable patients, the objective response rate (ORR) was 90% (9/10), including three stringent complete responses (sCRs). The minimal residual disease (MRD) negativity rate was 90%. One patient in the low-dose cohort who did not achieve MRD negativity still benefit with a very good partial response (VGPR). In the high-dose cohort, both the ORR and MRD negativity rate reached 100%. Follow-up remains limited, and responses in several patients continued to deepen over time.

Manageable Safety Profile

IASO206 demonstrated an overall manageable safety profile. No dose-limiting toxicities (DLTs), immune effector cell-associated neurotoxicity syndrome (ICANS), or treatment-related deaths were reported.

Infusion-related reactions were mild and consisted primarily of fever, chills, or rigors. All resolved on the day of infusion with standard supportive care.

Two patients experienced no CRS, seven patients experienced Grade 1 CRS, and one patient experienced Grade 2 CRS. The median duration of CRS was 2.5 days (range, 1–3 days).

Most Grade ≥3 hematologic toxicities were recovered within a relatively short period following treatment. Two patients experienced Grade ≥3 viral infections due to hypogammaglobulinemia, both of whom recovered following IVIG treatment.

Conclusion

The first-in-human data from IASO206 demonstrated that a single intravenous administration, without lymphodepleting chemotherapy, could generate functional BCMA-directed CAR-T cells in vivo. IASO206 showed encouraging anti-myeloma activity together with an overall manageable safety profile, supporting its continued clinical development and further evaluation of the optimal dose.

Professor Gang An

Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences

"Patients with relapsed/refractory multiple myeloma who have failed multiple lines of therapy, particularly those with triple-class exposure and high-risk disease, continue to face a poor prognosis and substantial unmet medical need.

"As an in vivo CAR-T cell therapy, IASO206 is administered as a single intravenous infusion without the need for apheresis or lymphodepleting chemotherapy. In this first-in-human study, IASO206 achieved a 90% objective response rate and a 90% MRD negativity rate, with both rates reaching 100% in the high-dose cohort.

"Notably, this was a particularly high-risk patient population: 90% had high-risk and 70% had ultra-high-risk cytogenetic features. Some patients also had adverse biological characteristics, including concurrent loss of TP53 and RB1 or low BCMA expression, which are associated with aggressive disease and poor prognosis. Despite these challenging baseline characteristics, meaningful clinical benefit was observed across the study population, providing encouraging preliminary evidence of the therapeutic potential of IASO206. With continued follow-up, we look forward to further characterizing the depth and durability of response."

Yongke Zhang, MD, PhD

Chief Scientific Officer and Senior Vice President, IASO Bio

"The selection of IASO206's first-in-human data for a late-breaking oral presentation at this year's IMS Annual Meeting represents another important milestone in the clinical translation of InTelliCAR™, IASO Bio's proprietary in vivo CAR-T platform, following U.S. Food and Drug Administration clearance of the Investigational New Drug application for IASO208.

"The first-in-human study of IASO206 has provided encouraging preliminary evidence that deep antitumor responses can be achieved with an overall manageable safety profile, even in a heavily pretreated R/R MM population enriched for high-risk and ultra-high-risk cytogenetic abnormality. These findings provide an important foundation for further characterizing the dose–response and dose–safety relationships and for determining the recommended Phase 2 dose (RP2D).

"IASO Bio has established a broad portfolio of investigational therapies based on the InTelliCAR™ platform. We will continue to advance the clinical development of IASO206, IASO208, and subsequent pipeline candidates while exploring their potential across additional disease settings. By combining the potential for efficacy with manageable toxicity and reduced treatment complexity, in vivo CAR-T therapy may ultimately enable CAR-T treatment to move beyond later-line settings and be evaluated earlier in the treatment paradigm, including potentially in first-line disease."

SOURCE IASO Bio