Caribou Biosciences Expands Clinical Development Of CB-010 With FDA Clearance Of IND In Lupus

Author: Benzinga Newsdesk | April 04, 2024 04:01pm

-- FDA has cleared Caribou's IND application for CB-010 in lupus nephritis and extrarenal lupus; GALLOP Phase 1 clinical trial expected to initiate by YE 2024 --

 

-- Driven by encouraging initial safety and efficacy in the ongoing ANTLER trial for r/r B-NHL, CB-010 clinical development has expanded to include autoimmune diseases --

-- Advancing ANTLER Phase 1 trial for 2L LBCL; initial dose expansion data to be shared at a medical congress in Q2 2024 --

-- Conference call and webcast scheduled for today at 5:00 pm ET --

BERKELEY, Calif., April 04, 2024 (GLOBE NEWSWIRE) -- Caribou Biosciences, Inc. (NASDAQ:CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, today announced that it received clearance of its Investigational New Drug (IND) application from the U.S. Food and Drug Administration (FDA) for CB-010, an allogeneic anti-CD19 CAR-T cell therapy with a PD-1 knockout (KO), for the treatment of lupus nephritis (LN) and extrarenal lupus (ERL). The Phase 1, multicenter, open label GALLOP clinical trial of CB-010 in patients with LN and ERL is expected to initiate by year-end 2024.

"CB-010 has demonstrated encouraging initial safety and efficacy in patients with relapsed or refractory B cell non-Hodgkin lymphoma, and we are excited to expand CB-010's clinical development to include autoimmune diseases," said Rachel Haurwitz, PhD, Caribou's president and chief executive officer. "By targeting CD19-positive B cells involved in the production of autoantibodies and the perpetuation of the autoimmune response, our off-the-shelf CAR-T cell therapy CB-010 has the potential to greatly improve the standard of care for patients with lupus, a prevalent and severe autoimmune disease."

CB-010 is the lead clinical-stage product candidate from Caribou's allogeneic CAR-T cell therapy platform. As previously reported, CB-010 has demonstrated encouraging initial safety and efficacy from the dose escalation portion of the ongoing ANTLER Phase 1 trial in patients with relapsed or refractory B cell non-Hodgkin lymphoma (r/r B-NHL). Caribou continues to enroll patients with large B cell lymphoma (LBCL) in the second-line setting, and initial dose expansion data from ANTLER will be presented at a medical congress in Q2 2024.

"Despite treatment advancements, fatigue, organ damage, and low health-related quality of life often remain life-long characteristics of lupus," said Richard Lafayette, MD, professor of medicine, Stanford Medicine Health Care. "An allogeneic anti-CD19 CAR-T cell therapy from healthy donor T cells has the potential to revolutionize lupus treatment, offering a readily available treatment for patients who need new therapeutic options."

Lupus is an autoimmune disease characterized by widespread inflammation that damages tissues and organs throughout the body. B cells, which normally produce antibodies that protect from infection, can play a devastating role in lupus by producing autoantibodies that cause the immune system to attack healthy tissues. CB-010 targets CD19, a protein on the surface of B cells, and has a PD-1 knockout (KO) that reduces CAR-T cell exhaustion. CB-010 holds the potential for deep depletion of disease-causing B cells which could reset the immune system, leading to sustained drug-free remission. In the ongoing ANTLER trial, depletion and recovery of patients' B cells is on par with the duration of B cell aplasia recently reported by Müller et al.1 Unlike many autologous and allogeneic CAR-T cell therapies, the manufacture of CB-010 does not rely on lentiviral or other retroviral vectors, which the FDA has recently identified may lead to risk of secondary malignancy potentially due to random integration of the chimeric antigen receptor (CAR) construct. Instead, the chRDNA technology allows for precise insertion of the CAR at an intended location within the T cell genome. The GALLOP trial will include partial HLA matching between donor sources and patients, which may lead to improved clinical outcomes based on data from the ongoing ANTLER trial.

"The human leukocyte antigen, or HLA, system acts as our body's identity card to know one's ‘self' from ‘not self.' In stem cell transplants, it has been shown that a close HLA match between patients and donors significantly reduces the rejection of the therapy. This same logic can be applied to allogeneic CAR-T cells as well, so that the activity of the therapy persists long enough to target and destroy the diseased cells," said Mehdi Hamadani, MD, professor of medicine, section chief of hematologic malignancies at Medical College of Wisconsin and investigator for the ongoing ANTLER Phase 1 trial. "Intriguing data from the ongoing ANTLER trial support incorporating an HLA matching strategy into Caribou's CB-010 trials, an innovative clinical approach that can potentially improve outcomes for patients. I am pleased to be part of the ANTLER trial to advance the development of CB-010 as an off-the-shelf CAR-T cell therapy that aims to address the limitations of currently approved treatment options."

Caribou continues to expect the $372.4 million cash, cash equivalents, and marketable securities, as of December 31, 2023, to fund the current operating plan into Q1 2026.

Posted In: CRBU