Caribou Biosciences(CRBU) - 2025 Q3 - Quarterly Results

Financial Position - As of September 30, 2025, Caribou Biosciences expects cash, cash equivalents, and marketable securities to be approximately $159.2 million[7]. - As of September 30, 2025, the Company reported preliminary unaudited cash, cash equivalents, and marketable securities[38]. Clinical Trials and Results - The ANTLER phase 1 trial for vispacabtagene regedleucel ("vispa-cel") reported an overall response rate (ORR) of 82% and a complete response (CR) rate of 64% among 22 patients[17]. - The 12-month progression-free survival (PFS) rate for the confirmatory cohort of vispa-cel was 51%[17]. - In the optimized product cohort of 35 patients, the ORR was 86% and the CR rate was 63%[22]. - The 12-month PFS for the optimized cohort was 53%[22]. - The ANTLER trial has enrolled a total of 84 patients, including a confirmatory cohort of 22 patients[15]. - In the ongoing CaMMouflage phase 1 trial for CB-011, 48 patients were enrolled, with a recommended dose for expansion of 450x10 viable CAR-T cells following a selected lymphodepletion regimen[29][31]. - The 12-patient BCMA-naïve cohort treated with the recommended dose showed a 92% overall response rate (ORR) and a 75% stringent complete response (sCR) rate[35]. - Notable adverse events in the CaMMouflage trial included infections (49% any grade) and cytokine release syndrome (31% any grade) across all treated patients[37]. - The longest responding patient in the BCMA-naïve cohort is in stringent complete response at 15 months post-infusion[31]. Regulatory Designations and Plans - Vispa-cel has received regenerative medicine advanced therapy (RMAT) designation and fast track designation from the FDA for relapsed or refractory large B cell lymphoma (r/r LBCL)[13]. - The FDA has recommended a randomized, controlled trial design for vispa-cel, which the Company believes provides a straightforward regulatory path to full approval[28]. - The Company plans to initiate a pivotal phase 3 trial for vispa-cel in second-line large B cell lymphoma patients who are ineligible for transplant[8]. - The Company plans to conduct a pivotal phase 3 trial for vispa-cel in 2L LBCL CD19-naïve patients, evaluating approximately 250 patients, with a primary endpoint of progression-free survival (PFS) and an interim analysis planned[28]. - The Company anticipates initiating dose expansion of the CaMMouflage trial before the end of 2025 and reporting data in 2026[37]. - The Company plans to further refine its pivotal phase 3 clinical trial design through continued engagement with the FDA prior to trial initiation[28]. Safety Profile - The safety profile of vispa-cel showed treatment emergent adverse events (TEAEs) in 25% or more of patients, including thrombocytopenia (62%) and cytokine release syndrome (55%)[25]. - The Company has not completed any head-to-head trials comparing vispa-cel or CB-011 with autologous CAR-T therapies, making cross-trial comparisons challenging[42].