CARGO Therapeutics (CRGX) Corporate Presentation summary

Business overview and market opportunity

• Focused on developing next-generation CAR T-cell therapies for large B-cell lymphoma (LBCL) and other B-cell malignancies, targeting significant unmet needs post-CD19 CAR T therapy.

• Autologous CAR T revenue in DLBCL projected to reach $3.3B by 2030, with patient access expanding as more centers offer CAR T treatments.

• Approximately 60% of LBCL patients do not achieve durable response with CD19 CAR T, representing a high unmet need and poor survival outcomes.

• By 2030, around 7,600 patients in the US, EU4, and UK are expected to require treatment post-CD19 CAR T therapy.

Clinical development and results

• FIRCE-1 Phase 2 study for firi-cel (CD22 CAR T) in R/R LBCL is ongoing, with >20 patients dosed and 26 sites activated; interim analysis expected 1H 2025.

• Independent Data Monitoring Committee recommended continuation of FIRCE-1 without protocol modifications due to strong safety and manufacturing success.

• Stanford Phase 1 study of firi-cel in heavily pretreated R/R LBCL patients showed 68% ORR and 53% CR rate at median 31.4 months follow-up, with no new relapses among CRs and mOS of 25.7 months for DL1.

• Firi-cel demonstrated a favorable safety profile with low rates of grade 3+ cytokine release syndrome and no grade 3+ neurotoxicity.

Pipeline and platform innovation

• Proprietary cell engineering platform enables development of multi-specific CAR T programs to address resistance mechanisms such as antigen escape and loss of costimulation.

• CRG-023, a tri-specific CAR T targeting CD19, CD20, and CD22 with a novel CD2 costimulatory domain, is in IND-enabling studies and has shown superior in vivo activity in mouse models.

• CARGO is the only cell therapy company in the post-CD19 space with a potentially pivotal Phase 2 clinical study underway.