#USA #CAR-T therapy #West Palm Beach #EXUMA Biotech #HER2 Targeting

EXUMA Biotech Presents Phase 1 Results at ASCO GI 2025

EXUMA Biotech, a pioneering biotechnology firm focused on innovative cell and gene immunotherapies, made headlines at the ASCO Gastrointestinal Cancers Symposium 2025. The company shared significant findings from a completed Phase 1 trial investigating their autologous HER2-targeted CAR-T therapy, revealing promising efficacy and robust safety across participant cohorts.

The trial involved 12 patients with HER2-positive Stage IV solid tumors, showcasing the drug's potential in treating various cancer types, including gastroesophageal cancer. Patients were administered a single dose of CAR-T cells, obtained through a meticulous 12-day manufacturing process that followed lymphodepletion with cyclophosphamide and fludarabine. Impressively, the trial faced no manufacturing failures, an important milestone for the development of this therapeutic approach.

Key Findings

Safety Insights

The reported results highlight a strong safety profile with no dose-limiting toxicities or serious adverse events linked to the therapy. Out of the 12 patients, only one experienced Grade 2 cytokine release syndrome (CRS), which resolved without any complications. Most notably, the trial reported no on-target, off-tumor toxicity (OTOTT), a crucial finding as it minimizes risks often associated with CAR-T therapies.

Efficacy Results

In terms of efficacy, the data demonstrated a remarkable deep partial response, particularly in a patient with gastroesophageal cancer, marked by a 100% reduction in the sum of the longest diameters (SLD) of their tumors, which has remained ongoing at a 36-week follow-up. Additionally, the survival rate observed in the third cohort at the 12-month mark reached 80%, contrasting sharply with the overall survival rate of 45.5% observed across all three cohorts combined.

Wendy Li, MD, the Chief Medical Officer at EXUMA Biotech, expressed her excitement regarding the trial's outcomes. She remarked on the ongoing need for safe yet effective treatments for patients diagnosed with HER2-positive gastroesophageal cancers and highlighted how their approach showcases significant anti-tumor activity without the adverse effects commonly seen in other immunotherapy methods.

Advancements and Future Directions

As they look ahead, EXUMA Biotech is dedicated to advancing its innovative solid tumor CAR-T treatments through its GCAR™ platform and SCL mRNA technology. The company plans to complete studies involving non-human primates in the first half of 2025. These studies aim to provide a clearer understanding of various pharmacological effects and guide future regulatory discussions regarding their first in vivo CAR-T product.

Gregory Frost, Ph.D., Chairman and CEO, shared his enthusiasm for the promising data. He emphasized how it aligns with their long-term vision of safely broadening the usage of CAR-T therapies into the realm of solid tumors. The intention to leverage novel technologies such as the FITNESS DRIVER and synthetic CAR ligand (SCL) reiterates their goal of providing effective solutions for patients in need.

Conclusion

As the company continues to innovate within the biotechnology sector, the Phase 1 results of their HER2-directed CAR-T product position EXUMA Biotech at the forefront of cancer treatment research, with hopes to significantly alter the treatment landscape for solid tumors. Collectively, these advancements echo the potential to deliver safer, effective therapies that meet the pressing medical needs posed by cancer.