Caribou Biosciences (CRBU) investor relations material

Program updates and clinical data

• Advancing two off-the-shelf CAR-T therapies: vispa-cel (CD19, lymphoma) and CB-011 (BCMA, multiple myeloma), both showing promising phase I data with best-in-class potential.

• Proprietary chRDNA CRISPR platform enables highly specific gene editing, supporting unique cell therapy armoring strategies.

• Both programs will have oral presentations at EHA, with new data updates expected.

• Vispa-cel uses PD-1 knockout for enhanced anti-tumor activity; CB-011 employs immune cloaking to prolong persistence.

• Phase I data for vispa-cel show durable responses, with some patients in remission for over four years.

Phase III trial design and strategy

• Phase III pivotal trial for vispa-cel targets 2nd line LBCL patients ineligible for auto CAR T or transplant, with 1:1 randomization against chemoimmunotherapy regimens.

• Control arm includes four regimens, two with polatuzumab and two without, to reflect real-world treatment options.

• Primary endpoint is progression-free survival; crossover allowed for control arm patients upon progression.

• Study is unblinded due to treatment differences; 250 patients planned to ensure high statistical power.

• Recruitment leverages both academic and community hospitals, aiming for rapid global enrollment.

Commercial and manufacturing considerations

• Off-the-shelf approach addresses access and timing barriers of autologous CAR T, enabling broader patient reach.

• Manufacturing is fully scaled for vispa-cel, with cost of goods expected to be 96% lower than autologous CAR T.

• HLA matching strategy allows 99% of lymphoma patients to be served with just 10 donor lots.

• Community hospitals can deliver allo CAR T using existing bispecific protocols, simplifying logistics.