Anito-Cel Therapy Achieves 97% Response Rate in Myeloma Patients

In a groundbreaking development in cancer treatment, anitocabtagene autoleucel, branded as Anito-Cel, has demonstrated a remarkable overall response rate of 97% in patients suffering from relapsed/refractory multiple myeloma (R/R MM) after undergoing at least three prior lines of therapy. This data was presented during the European Hematology Association (EHA) Congress held from June 12 to 15, 2025, in Milan, Italy, highlighting the potential of this novel CAR T-cell therapy in managing a challenging hematologic malignancy.

The clinical trial, labeled iMMagine-1 and registered under NCT05396885, involved 117 evaluable participants, with a median patient age of 64 years. These patients had previously been refractory to multiple treatment regimens, including an anti-CD38 antibody, an immunomodulatory drug, and a proteasome inhibitor. Notably, the trial reported a stringent complete response rate of 68%. The findings also indicated a minimal residual disease (MRD) negativity rate of 93% among evaluable patients, which is a critical factor for long-term remission in cancer treatment.

Dr. Gurbakhash Kaur, an assistant professor of medicine in hematology and medical oncology at Mount Sinai Hospital in New York, who is the lead author of the study, emphasized the significance of the D-Domain, a novel synthetic antigen-binding domain utilized in Anito-Cel. "This innovative design enhances transduction efficiency, allowing for a higher density of CAR-positive T cells, which contributes to the therapy's efficacy and safety profile," said Dr. Kaur during her presentation.

The safety data collected during the trial indicated manageable adverse effects, with cytokine release syndrome (CRS) occurring in 70% of patients at grade 1 severity and 15% at grade 2. Importantly, the majority of CRS cases resolved quickly, with a median onset time of four days. Furthermore, only 8% of patients experienced immune effector cell-associated neurotoxicity syndrome (ICANS), which further supports the therapy's safety.

Prior studies, including a phase 1 trial (NCT04155749), have shown that Anito-Cel leads to a median progression-free survival (PFS) of 30.2 months, indicating its potential for long-term effectiveness in R/R MM patients. The ongoing phase 3 trial, iMMagine-3 (NCT06413498), aims to further evaluate Anito-Cel against standard-of-care therapies in patients with R/R MM who have received one to three prior lines of therapy.

The implications of these findings are significant, as multiple myeloma remains a challenging condition to treat, particularly in patients who have exhausted existing therapies. According to the American Cancer Society, approximately 34,920 new cases of multiple myeloma were expected in the United States in 2025, with a five-year survival rate of only 54%. The introduction of Anito-Cel could provide a new lifeline for patients battling this disease.

In summary, the promising results from the iMMagine-1 trial underscore the potential of Anito-Cel as a transformative treatment option for patients with relapsed/refractory multiple myeloma. With ongoing trials and research, the future looks optimistic for incorporating CAR T-cell therapies into standard oncology practices, potentially improving patient outcomes significantly.