Caribou Phase 1 trial testing safety, efficacy of CAR T-cell therapy
CB-010 aims to treat patients with lupus affecting kidneys, other organs
Caribou Biosciences has launched a Phase 1 clinical trial to evaluate the safety and efficacy of CB-010, its CAR T-cell therapy for lupus that affects the kidneys and other organs.
CB-010 is being developed as a treatment for people with lupus nephritis, a common complication of lupus that causes kidney damage and inflammation, and those with extrarenal lupus, in which other tissues and organs are affected.
In the new Phase 1 GALLOP trial (NCT06752876), approximately 20 patients with hard-to-treat, or refractory, lupus will receive a single infusion of CB-010 and be followed for a period of two years. During that time, researchers will assess the treatment’s safety and effectiveness.
The trial is not yet recruiting participants and study sites have not been announced.
“We are excited to share we have initiated the GALLOP Phase 1 trial. This milestone is a testament to the significant momentum we achieved across … clinical programs throughout 2024,” Rachel Haurwitz, PhD, president and CEO of Caribou, said in a company press release.
Phase 1 trial expects to enroll 20 patients, plans 2 years of follow-up
In lupus, immune cells called B-cells produce self-reactive antibodies that target healthy tissues, driving inflammation and disease symptoms.
Approximately half of all lupus patients develop lupus nephritis, and most with this complication do not respond adequately to immunosuppressant therapies. That’s led scientists to seek other types of treatment.
A promising form of treatment designed for cancers and other diseases involving abnormal immune functioning, like lupus, is CAR T-cell therapy.
CAR T-cell therapies use immune cells, called T-cells, that are isolated from either patients or healthy donors. Once isolated, these cells are genetically modified to have a lab-made protein on their surface known as a chimeric antigen receptor, or CAR. This receptor enables the modified T-cells to recognize specific proteins on the surface of other cells and selectively kill those cells once they are infused back into a patient.
In CB-010, the CAR is designed to recognize CD19, a protein found on the surface of B-cells. By directing T-cells to target and eliminate B-cells, the therapy is expected to lower the levels of the self-reactive antibodies that drive the disease.
The therapy is considered to be off the shelf, because the engineered T-cells that are harvested from healthy donors can be used to treat multiple patients.
[Caribou is] excited to be on the forefront of a new era for … CAR T-cell therapies.
The GALLOP trial aims to enroll patients with refractory lupus who have not responded well to glucocorticoids and at least two immunosuppressive therapies. CB-010 will be given to two groups of patients, one with lupus nephritis and a second with extrarenal lupus.
Patients with lupus nephritis must have been diagnosed as either class III or IV. For people with class III lupus nephritis, less than 50% of kidney glomeruli — the tiny blood vessels in the kidneys responsible for filtering blood — are affected by the disease. In class IV lupus nephritis, more than 50% of glomeruli are impacted. Both of these classes are considered severe forms of lupus kidney disease and have a high risk of progressing to kidney failure.
Participants who enroll in the trial should expect to be in the study for approximately two years after they receive their infusion of CB-010. During those two years, patients will be monitored for adverse reactions to the treatment to ensure the therapy is safe. Additionally, researchers will assess CB-010’s pharmacokinetics, or how the therapy moves into, through, and out of the body.
The U.S. Food and Drug Administration granted fast track designation to CB-010 as a lupus treatment in the fall of last year. This designation is intended to encourage the development, and speed the review, of therapies for serious conditions that meet an unmet medical need. As part of this designation, CB-010 may be eligible for programs enabling faster regulatory reviews if certain criteria are met.
CB-010 is also being tested in the ongoing Phase 1 ANTLER trial (NCT04637763) as a treatment for relapsed or refractory B-cell non-Hodgkin lymphoma, a B-cell-driven form of cancer.
According to the release, “Caribou plans to provide updates as the GALLOP Phase 1 clinical trial … advances.”
Haurwitz noted that the company is “excited to be on the forefront of a new era for … CAR T-cell therapies.”
