CARGO Therapeutics (CRGX) Jefferies 2024 Global Healthcare Conference summary

Lead program overview and clinical progress

• Lead program firi-cel (CRG-022) targets CD22 antigen in large B-cell lymphoma, focusing on patients relapsed/refractory to CD19 CAR therapy.

• Phase I Stanford study dosed 38 high-risk patients, showing a 68% overall response rate and 53% complete response rate, with favorable safety and no grade 3 ICANS.

• Median overall survival at dose level 1 reached 25.7 months, with durable responses in the majority of complete responders after 30 months of follow-up.

• Over 20 patients have been dosed in the ongoing pivotal phase II FIRCE-1 study, with strong manufacturing success.

• Interim analysis is planned for the first half of 2025 to enable FDA discussions and inform commercialization investments.

Financial and operational updates

• Recently closed an oversubscribed $110 million PIPE, providing resources for robust BLA preparation and extending runway through 2026.

• Increased resources will also support progress on the CRG-023 program, which is in IND-enabling studies.

• The company is leveraging CDMOs for manufacturing, with plans to have two at launch and scalable processes for commercial readiness.

• Commercial process improvements and comparability packages have been submitted and cleared by the FDA.

Market opportunity and strategic outlook

• The addressable population for post-CD19 CAR therapy in large B-cell lymphoma is estimated at 7,600–7,700 patients in the US, EU, and UK by 2030.

• Market opportunity is considered multi-billion dollar, with potential to expand as CD19 CAR use grows and moves to earlier lines of therapy.

• The company is focused on the US market but is open to ex-US partnerships to accelerate global access.

• Manufacturing strategy includes optionality for scaling, with the ability to add CDMOs or invest in internal capacity as needed.