EXUMA Biotech Reports Promising Phase 1 Results for Novel HER2-Targeted CAR-T Therapy in Solid Tumors

EXUMA Biotech has unveiled Phase 1 clinical trial results for its novel HER2-directed CAR-T cell therapy, CCT303-406, designed specifically for patients with metastatic HER2-positive solid tumors. The findings were presented at ASCO GI 2025, marking a significant advancement in solid tumor immunotherapy.

Novel Approach to Solid Tumor Treatment

The investigator-initiated trial evaluated CCT303-406, an autologous CAR-T product that incorporates EXUMA's proprietary tumor metabolism regulated (TMR) targeting technology. This innovative approach focuses CAR-T activity specifically within the tumor microenvironment (TME), potentially addressing a critical challenge in CAR-T therapy for solid tumors – the risk of on-target, off-tumor toxicity.

Clinical Significance and Target Population

HER2 overexpression, a characteristic feature of various cancers including breast, stomach, bladder, and colon tumors, remains a significant therapeutic target. While many patients initially respond to conventional HER2-targeted antibody treatments, they often develop resistance while still maintaining HER2 overexpression. CCT303-406 presents a promising alternative for these patients, particularly showing encouraging results in gastroesophageal cases.

Future Development Pipeline

Dr. Gregory Frost, Chairman and CEO of EXUMA Biotech, expressed optimism about the results: "We are pleased with these data that support our decade-long vision to expand the safety and efficacy of CAR-T into solid tumors."

The company is simultaneously advancing its in vivo CAR-T platform, GCAR™, with non-human primate studies scheduled for completion in the first half of 2025. These studies will evaluate various critical parameters including:

• Vector selectivity and tropism
• Biodistribution patterns
• Pharmacokinetic and pharmacodynamic profiles
• Safety assessments
• Effects of SCL mRNA administration on GCAR™ cellular pharmacokinetics

Platform Technology Innovation

EXUMA's technology portfolio includes several proprietary innovations:

• The GCAR™ platform for in vivo CAR-T delivery
• Synthetic CAR ligand (SCL) technology
• FITNESS DRIVER technology for enhanced targeting

These combined technologies aim to create an off-the-shelf solution that could potentially revolutionize CAR-T therapy by reducing cost and complexity while maintaining or improving efficacy and safety profiles.