#United States #Santa Monica #Gilead #Kite #anito-cel

Introduction

Gilead Sciences and Kite, a subsidiary of Gilead, recently disclosed promising findings from the iMMagine-1 trial during the 67th Annual Meeting of the American Society of Hematology (ASH) 2025. This pivotal Phase II study evaluates anito-cel, a novel CAR T-cell therapy targeting refractory and relapsed multiple myeloma (RRMM). The results indicate significant efficacy and a favorable safety profile, providing hope for improved outcomes among patients suffering from this challenging blood cancer.

Efficacy of anito-cel

The iMMagine-1 trial involved 117 patients, all of whom had previously undergone multiple lines of treatment. The median follow-up was approximately 15.9 months, during which striking results were observed: a remarkable overall response rate (ORR) of 96%, with 74% achieving stringent complete response (sCR) or complete response (CR) according to International Myeloma Working Group (IMWG) criteria. Furthermore, out of the 96 patients who could be assessed for minimal residual disease (MRD), 95% achieved MRD negativity within a median time of just one month.

Across the study population, 87% had triple-class exposure, indicating they had been previously treated with at least three different classes of therapies, showcasing the challenging nature of these patients' condition. However, the majority showed rapid responses, often within the first month post-treatment. The median time to best response was 4.8 months, whereas the median duration for attaining sCR or CR was 3.2 months.

Survival Rates

Long-term survival rates were also encouraging. The 12-month progression-free survival (PFS) rate was reported at 82.1%, with 67.4% at 18 months and 61.7% at 24 months. Overall survival (OS) rates were similarly robust, showing 94% OS at the 12-month mark, dropping to 88% at 18 months, and 83% at 24 months. These results suggest the treatment provides a sustained benefit, with a significant number of patients remaining free from progression after two years.

Safety Profile

One of the most reassuring aspects of the study was the safety profile of anito-cel. Notably, no cases of late-onset neurotoxicity or significant immune effector cell-associated colitis were reported during the trial. The treatment's side effects showed consistency with previous studies, with 86% of patients experiencing cytokine release syndrome (CRS), primarily mild and manageable. Serious adverse events related to hematologic toxicity were observed, including neutropenia in 71% of patients, but severe infections were reported in only 9%.

Expert Opinions

Krina Patel, an associate professor at The University of Texas MD Anderson Cancer Center and the trial's principal investigator, emphasized the data's importance, acknowledging how these results mark a significant advance in RRMM treatment. Patel highlighted the need for treatments that provide durable and meaningful efficacy with predictable safety profiles, an area where anito-cel shows promise.

Cindy Perettie, Executive Vice President at Kite, acknowledged the necessity for effective options as resistance increases in advanced RRMM cases. She stated, “With the confirmed depth and durability of responses observed in iMMagine-1 along with a manageable safety profile and reliable manufacturing, anito-cel holds the potential to redefine treatment protocols.”

Future Perspectives

Anito-cel employs an innovative D-Domain technology aimed at efficiently targeting BCMA-positive cells. Preliminary non-clinical research has demonstrated its unique mechanism of action involving rapid binding and dissociation from BCMA, helping in effectively eradicating myeloma cells while potentially alleviating inflammation. The ongoing exploration around enhancing these mechanisms might lead to breakthrough insights into CAR T-cell therapy efficacy.

As Gilead and Kite look towards regulatory submissions and market introduction of anito-cel in the U.S. by 2026, the landscape for RRMM treatments may significantly shift, offering new hope and improved outcomes for patients worldwide.

Conclusion

With the combination of encouraging efficacy, promising safety data, and a novel approach to targeting myeloma cells, anito-cel stands as a potential game changer in the treatment of RRMM. As the field of oncology continues to evolve, developments like the iMMagine-1 trial insights are crucial in addressing the needs of patients and enhancing therapeutic options in this challenging disease landscape.