CARGO Therapeutics (CRGX) Piper Sandler 36th Annual Healthcare Conference summary

Program overview and clinical progress

• FIRCE-1 is a phase II, potentially pivotal study of firi-cel in relapsed/refractory large B-cell lymphoma patients who failed CD19 CARs, addressing a high unmet need.

• Phase I data from Stanford showed a 52% complete response rate and strong durability, with 75% of complete responses durable at one year and 47% overall survival at three years.

• 57 patients have been dosed as of November 8, with all 31 sites activated and strong enrollment momentum.

• Interim analysis is planned for the first half of 2025, with the potential for regulatory discussions based on results.

• Three cohorts are included: primary CD19 relapsed/refractory, bispecific T-cell engager relapsed/refractory, and a novel out-of-spec product cohort.

Competitive landscape and manufacturing

• No standard of care exists for patients relapsed/refractory to CD19 CARs; bispecific T-cell engagers offer about 20% durable response, but CAR-T is preferred for higher cure rates.

• Manufacturing success is high, with only two out-of-spec doses among 57 patients, attributed to a robust CDMO strategy and in-house expertise.

• Reliable manufacturing and predictable supply have driven strong investigator and patient interest.

• The company is positioned ahead of competitors, with faster dosing and data generation compared to others still in phase I.

Safety, future plans, and pipeline expansion

• Firi-cel shows a favorable safety profile: very low to zero grade 3 CRS and 0% grade 3 ICANS, reducing patient and healthcare burden.

• Passed multiple safety reviews with no modifications; outpatient therapy and pediatric B-ALL studies are under consideration.

• Early phase I data in mantle cell and follicular lymphoma show 100% response in initial patients, supporting lifecycle management opportunities.

• Expansion into earlier lines of therapy and pediatric settings is planned, pending interim results and further FDA discussions.