Cellectis (ALCLS) JMP Hematology and Oncology Summit 2024 summary

Strategic partnerships and investments

• AstraZeneca invested $220 million for a 44% stake, supporting joint development of cell and gene therapies across oncology, immune, and genetic disorders.

• Three initial programs launched: CAR-T for hematological malignancies, CAR-T for solid tumors, and a gene therapy for genetic disorders.

• Collaboration leverages innovation in allogeneic CAR-T and advanced internal manufacturing capabilities.

• Funding extends cash runway to 2027, enabling progress on both partnered and wholly owned clinical programs.

Clinical program updates and data

• UCART22 (BALLI-01 trial) targets heavily pretreated ALL patients, with full Phase I data expected in 2025.

• Internally manufactured product shows improved expansion and efficacy over externally sourced versions.

• Enrollment is progressing, with recommended Phase II dose expected to be identified in 2025.

• Safety profile is favorable, with low-grade CRS/ICANS and manageable adverse events, even in frail populations.

• Plans to engage FDA and EMA in 2025 for pivotal trial design and registration pathway.

Dual-target CAR-T and future directions

• UCART20x22 dual CAR-T showed three responses in three patients, including two MRD-negative, at initial dose.

• Safety remains acceptable during dose escalation, with no major concerns anticipated.

• Product positioned for broad use in hematological malignancies and potential in autoimmune indications.

• Next data update and regulatory feedback for UCART20x22 expected in 2025 after current cohort completion.