Caribou Biosciences (CRBU) Jefferies 2024 Global Healthcare Conference summary

Recent clinical data and technology updates

• Presented new data from four clinical-stage off-the-shelf CAR-T cell therapy programs, focusing on CB-010 for B-cell lymphoma and lupus.

• Ended Q1 with $346M cash, providing runway into Q1 2026 for ongoing trials.

• CB-010 leverages next-gen CRISPR genome editing (chRDNA) for enhanced specificity and three key edits, including PD-1 knockout.

• CB-011 (multiple myeloma) and CB-012 (AML) are advancing in phase I trials, with initial data for CB-011 expected by year-end.

• All programs utilize strategies like checkpoint disruption and immune cloaking to improve anti-tumor activity.

CB-010 clinical trial progress and findings

• Dose escalation and expansion in the ANTLER trial included 46 patients, with 80M CAR-T cells selected as the recommended phase II dose.

• Partial HLA matching (≥4 alleles) between donor and patient led to enhanced efficacy and a median PFS of 14.4 months.

• New 20-patient cohort will prospectively confirm HLA matching benefits, with data expected in H1 2025.

• Safety profile is favorable: no Grade 3+ CRS or GvHD, and lower rates of severe ICANS compared to Yescarta.

• FDA approved outpatient administration of CB-010; a new cohort will include patients relapsed after prior CD19 therapy.

Strategic and operational plans

• Phase III pivotal trial in second-line large B-cell lymphoma planned for H2 2025, with inventory strategy to ensure HLA-matched doses for ~90% of patients.

• HLA matching strategy incorporated into both oncology and lupus trials.

• Initial lupus trial (GALLOP) to start by year-end, leveraging B-cell aplasia data for de-risking.

• CB-011 and CB-012 trials progressing, with first data for CB-011 expected by year-end and ongoing enrollment for CB-012.

• Two key CB-010 data readouts (HLA-matched and CD19-relapsed cohorts) expected in H1 2025 to support phase III initiation.