Caribou Biosciences (NASDAQ:CRBU) Chief Executive Officer Rachel Haurwitz said the company is advancing two CRISPR-enabled, off-the-shelf CAR T cell therapy programs, with its lymphoma candidate vispa-cel now “pivotal-ready” and its multiple myeloma program CB-011 continuing in Phase 1 development.
Speaking at a company news event, Haurwitz described Caribou as “a CRISPR genome editing company at its core” that is using its proprietary platform to develop allogeneic CAR T therapies for hematologic malignancies. She said vispa-cel has completed a Phase 1 study in non-Hodgkin lymphoma and has shown outcomes “on par with autologous CAR T cell therapies.” CB-011, she added, has generated early clinical activity in multiple myeloma, with dose expansion data expected later this year.
Vispa-cel pivotal trial targets patients without access to autologous CAR T
The study is designed for patients Haurwitz described as the “have-nots”: those who do not receive autologous CAR T therapy or autologous stem cell transplant in the second-line setting. She said commercial data indicate that 75% of second-line large B-cell lymphoma patients do not receive autologous CAR T, despite it being the “undisputed gold standard” approach.
According to Haurwitz, the FDA has endorsed enrollment of two categories of patients: those medically ineligible for autologous CAR T, often because they cannot wait for treatment, and those who may be medically eligible but face access barriers, such as travel and caregiver limitations.
The control arm will consist of investigator’s choice chemoimmunotherapy regimens. Haurwitz said those approaches have no demonstrated curative potential and cited median progression-free survival of about 4.5 months for the basket of regimens. She said Caribou expects physicians to choose from four control options, including regimens with and without polatuzumab, to reflect varied use of the drug in frontline therapy.
Haurwitz also said the FDA has allowed crossover in the United States. Patients assigned to the control arm who experience progressive disease may cross over to receive vispa-cel, a feature she said should help with enrollment.
Company sees off-the-shelf model addressing timing and access barriers
Haurwitz said vispa-cel’s off-the-shelf format could address the two main reasons many second-line patients do not receive autologous CAR T: urgent need for treatment and access challenges. She said patients treated in the community may face eight to 12 weeks or more to complete referral, apheresis, bridging therapy and dosing for autologous CAR T.
“With vispa-cel, it’s sitting in the freezer as we speak today,” Haurwitz said. She said that in the Phase 1 study, some patients moved from completing eligibility paperwork to beginning lymphodepletion on the same day.
The company plans to include both academic centers and “sophisticated community hospitals” in the trial. Haurwitz said these community sites often administer bispecific antibodies today but cannot deliver autologous CAR T. Caribou also expects ANTLER III to be a global study, focused on countries where similar access gaps exist.
Haurwitz said Caribou expects the study to run quickly, estimating approximately two years from initiation to data, based on the event-driven progression-free survival endpoint.
Market positioning and manufacturing economics discussed
Asked about the evolving second-line lymphoma landscape, including potential bispecific antibody approvals, Haurwitz said she does not believe future approvals would dramatically change the market opportunity for vispa-cel. She said autologous CAR T penetration has stabilized around 20% to 25%, despite manufacturing investments by companies in that space, reflecting real-world limits related to urgency and access.
Haurwitz said Caribou has not publicly provided quantitative market share estimates for vispa-cel. However, she said market research supports the company’s view that vispa-cel could become a market-leading second-line drug if its profile matches what was seen in Phase 1.
On pricing, Haurwitz said it is premature to provide specifics. She said Caribou’s manufacturing process is “commercial ready,” with each batch producing enough cells for 200 to 300 doses. She also said the company anticipates cost of goods sold at launch to be 96% lower than autologous CAR T therapies, giving Caribou “tremendous flexibility” on pricing strategy.
CB-011 data and development options
For CB-011 in multiple myeloma, Haurwitz said Caribou expects to present longer follow-up from the Phase 1 CaMMouflage trial at the European Hematology Association meeting in Stockholm. She said last year’s data included all 48 patients in dose escalation and helped the company select a lymphodepletion regimen and 450 million CAR T cells as the recommended dose for expansion.
In a cohort of 12 BCMA-naive patients at the recommended expansion dose, Haurwitz said Caribou observed response rates above 90% and a 75% complete response rate. She said the key question now is durability, with key opinion leader feedback suggesting a median progression-free survival benchmark of 12 to 15 months for late-line, high-risk, relapsed/refractory multiple myeloma patients.
Caribou is enrolling two expansion cohorts for CB-011: one for BCMA-naive patients and one for patients with prior BCMA exposure. Haurwitz said the company expects to share initial dose expansion data by the end of the year.
CB-011 received RMAT designation earlier this year. Haurwitz said that designation should support more proactive engagement with the FDA, and she hopes Caribou will have its first agency interaction before sharing dose expansion data later this year. She said future development could include larger patient populations, potentially requiring additional capital or a partner, or more focused approaches in later-line groups such as post-BCMA patients or those with extramedullary disease.
Capital needs and upcoming catalysts
Haurwitz said partnerships could play a role in the future of vispa-cel and/or CB-011, although she said the company is not relying on partnership as the only path forward. For vispa-cel, she said current cash is being used for initial trial startup activities, site readiness and supply, but Caribou will need to raise additional capital to fully fund the pivotal study.
She said potential financing options include equity capital markets and non-dilutive structured financing. Near-term catalysts include two podium presentations at EHA, one for vispa-cel and one for CB-011, as well as CB-011 dose expansion data expected by the end of the year.
About Caribou Biosciences (NASDAQ:CRBU)
Caribou Biosciences, Inc is a clinical-stage biopharmaceutical company that leverages its proprietary CRISPR-Cas gene-editing platform to develop transformative cell therapies and in vivo treatments for a range of cancers and genetic diseases. The company’s core technology enables precise modification of cellular genomes, allowing the design of engineered T-cell and NK-cell therapies aimed at improving safety, efficacy and persistence in patients with hematologic and solid tumor malignancies. Alongside its oncology portfolio, Caribou is advancing in vivo editing programs targeting monogenic disorders, with initiatives in areas such as Duchenne muscular dystrophy and familial amyloidosis.
Established in 2011 and headquartered in Berkeley, California, Caribou Biosciences was co-founded by Nobel laureate Jennifer Doudna, one of the pioneers of CRISPR gene-editing technology.
