Caribou Biosciences (CRBU) investor relations material

Key insights from presentations

• Vispacel, an off-the-shelf CAR T cell therapy for large B cell lymphoma, shows efficacy and safety on par with autologous CAR T, with potential for outpatient and community use.

• Around 75% of second-line large B cell lymphoma patients do not benefit from autologous CAR T, and 60% are ineligible for both auto CAR T and stem cell transplant due to clinical and logistical barriers.

• The pivotal phase 3 trial for Vispacel will target second-line, dual-ineligible patients, using a randomized controlled design with endpoints including PFS, response, durability, OS, and quality of life.

• Clinical data from the Antler study show high response rates and durable remissions, especially with young donor T cells, and a manageable safety profile similar to liso-cel.

• Community oncologists and academic leaders agree that off-the-shelf CAR T could address major access issues, enabling more patients to receive potentially curative therapy locally.

Barriers and unmet needs

• Major barriers to CAR T access include referral challenges, patient reluctance to travel, socioeconomic factors, and long wait times for manufacturing and insurance approval.

• Many eligible patients decline or cannot access CAR T due to distance from academic centers, with up to 50-60% in some regions not receiving optimal therapy.

• Rapidly progressive disease and manufacturing failures further limit timely access to autologous CAR T, highlighting the need for immediate, off-the-shelf options.

• Community centers are prepared to deliver intensive regimens and manage CAR T toxicities, especially with experience from bispecific antibody programs.

Physician perspectives and future outlook

• Physicians see Vispacel as a practice-changing therapy that could shift the treatment paradigm, especially for patients unable or unwilling to travel.

• Academic centers anticipate that off-the-shelf CAR T will relieve volume pressures and allow them to focus on innovation, while community centers expand access.

• If efficacy is non-inferior to autologous CAR T or superior to bispecifics, uptake is expected to be high; logistical ease and immediate availability are key advantages.

• The platform's flexibility and potential for further innovation are seen as major strengths for future cellular therapies.

• Expansion into other indications, including multiple myeloma and solid tumors, is underway, with new data expected on CB-011 targeting BCMA.