CARGO Therapeutics (CRGX) 43rd Annual J.P. Morgan Healthcare Conference 2025 summary

Strategic vision and program updates

• Mission centers on developing next-generation, potentially curative cell therapies for cancer, with three programs advancing: two clinical-stage and one entering IND-enabling stage in 2024-2025.

• Lead program firi-cel (CD22-targeted CAR T) is in a pivotal Phase 2 trial, with interim results expected in H1 2024; 71 patients dosed by end of December 2023.

• CRG-023, a trispecific CAR T targeting CD19, CD20, and CD22, cleared IND and will begin Phase 1 by mid-2024.

• Announced a novel allogeneic universal vector platform, with lead candidate selection expected in H1 2024.

• Robust cell therapy expertise and manufacturing capabilities underpin program advancement.

Market opportunity and patient need

• Large B-cell lymphoma patient population relapsed/refractory to CD19 CARs expected to reach 8,000 in US/EU5 by 2030; 39,000 CAR-naive patients projected.

• Firi-cel aims to address high unmet need in patients with poor outcomes after CD19 CAR T therapy, where median survival is only 5.5 months.

• Stanford Phase 1 data for firi-cel showed 66% overall response, 52% complete response, and 25.7 months median overall survival.

• Tolerability profile for firi-cel is favorable compared to CD19 CARs; dose level one selected for Phase 2 due to lower toxicity.

Clinical development and milestones

• Phase 2 firi-cel study is single-arm, multi-center, targeting relapsed/refractory large B-cell lymphoma post-CD19 CAR; 71 patients dosed, interim data on track for H1 2024.

• Key efficacy benchmark is six-month durability of response; interim analysis will focus on three-month follow-up for FDA discussions.

• Manufacturing success rate remains high, with vein-to-vein time at 20 days and scalable, portable processes in place.

• Three CDMOs engaged, supporting clinical and potential commercial supply for up to two years.