Caribou Biosciences has linked its CAR-T cell therapy candidate to 17.1-month median progression-free survival (PFS), adding to evidence that the off-the-shelf treatment can match the efficacy of autologous products in lymphoma patients.
Last year, Caribou posted phase 1 data showing its allogeneic anti-CD19 CAR-T therapy achieves similar response and 12-month PFS rates to Bristol Myers Squibb’s Breyanzi and Novartis’ Yescarta. At the time, the clinical trial was yet to reach its median PFS, leaving questions about how Caribou’s vispacabtagene regedleucel (vispa-cel) would compare to Breyanzi and Yescarta on that key yardstick.
Caribou shared the median PFS result ahead of an oral presentation at the 2026 European Hematology Association Annual Meeting on Friday. At 17.1 months, Caribou’s result numerically beat Breyanzi’s 14.8 months and Yescarta’s 14.9 months.
The vispa-cel data come from 27 second-line large B cell lymphoma (LBCL) patients who received a single dose of 80 million optimized CAR-T cells. Caribou optimized the therapy by taking cells from donors aged under 30 with at least two human leukocyte antigen alleles that match the patient.
None of the patients had graft-versus-host disease or grade 3 or higher immune effector cell-associated neurotoxicity syndrome, respectively concerns with off-the-shelf treatments and CAR-T cell therapies in general. There was one grade 3 or higher case of cytokine release syndrome, another known side effect of CAR-T therapies. There were six infections and five prolonged cytopenias of at least grade 3 severity.
One vispa-cel-related death, which Caribou disclosed in an earlier update, occurred because of immune effector cell-associated HLH-like syndrome. A death from progressive multifocal leukoencephalopathy was possibly related to the CAR-T cell therapy.
The safety and efficacy data contain little evidence that vispa-cel is a better treatment than Breyanzi and Yescarta, which were approved in 2021 and 2017, respectively. But Caribou is betting that matching the outcomes achieved by the autologous cell therapies will be enough for its allogeneic prospect to carve out a piece of the market.
About 25% of second-line LBCL patients receive an autologous CAR-T cell therapy, despite the products being the “undisputed gold standard therapeutic approach,” Caribou CEO Rachel Haurwitz, Ph.D., said at a Jefferies event last week. In a planned phase 3 trial, Caribou will target patients who are ineligible for autologous therapy or cannot access the drugs, for example, because they live too far from a care center.
Medically ineligible patients typically cannot wait for an autologous therapy to be made from their cells, Haurwitz said. As an off-the-shelf treatment, vispa-cel could eliminate the lag between prescription and treatment. Equally, Caribou plans to lower access barriers by including community hospitals in its phase 3 trial, expanding uptake beyond the academic centers that administer autologous cell therapies.
While Caribou sees a clear opportunity for vispa-cel, the company has struggled to drum up investor enthusiasm. Caribou’s market cap was below $180 million at the close of trading Wednesday. And with the biotech forecasting its cash runway will end in the second half of next year, management is looking for ways to fully fund the study. Caribou ended March with $118.6 million in the bank.
