Caribou Biosciences Inc. (NASDAQ:CRBU) shares skyrocketed 100% on Monday after the company released promising Phase 1 clinical data for its CB-011 therapy in patients with relapsed or refractory multiple myeloma.
The CRISPR-based genome-editing company reported a 92% overall response rate and a 75% complete response rate or better among 12 patients treated with the recommended expansion dose in its ongoing CaMMouflage Phase 1 trial. CB-011 is an off-the-shelf anti-BCMA CAR-T cell therapy, designed to target and destroy malignant plasma cells.
Notably, 91% of evaluable patients achieved minimal residual disease (MRD) negativity, and seven out of 12 patients remained in very good partial response or better six months after a single infusion. The longest responding patient has maintained a stringent complete response for 15 months following treatment.
“We are very encouraged by the compelling results from the CaMMouflage Phase 1 trial, which demonstrate that CB-011 is delivering deep, durable responses in high-risk, heavily pretreated multiple myeloma patients with a manageable safety profile,” said Rachel Haurwitz, PhD, Caribou’s president and chief executive officer.
CB-011 showed a manageable safety profile across all dose levels, with no cases of graft-versus-host disease reported. However, the company noted some adverse events, including one treatment-related grade 5 immune effector cell-associated hematotoxicity.
Caribou plans to initiate dose expansion by the end of 2025, with additional data expected in 2026.
According to Dr. Adriana Rossi, a trial investigator, the therapy could help close a major treatment gap, since only about 10% of eligible multiple myeloma patients currently receive autologous CAR-T therapy.
The results position CB-011 as a potential breakthrough allogeneic cell therapy in the fight against multiple myeloma, offering renewed hope for patients with limited treatment options.