CAR T-cell therapy, CB-011, on FDA fast track for hard-to-treat myeloma

Phase 1 trial underway in adults with relapsed or refractory disease

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The U.S. Food and Drug Administration (FDA) has given a fast track designation to CB-011, an experimental CAR T-cell therapy being developed by Caribou Biosciences for people with relapsed or refractory multiple myeloma.

Fast track status is given to investigational treatments with the potential to fill an unmet need in medical care. It allows the therapy’s developer to have earlier and more frequent communication with the regulatory agency throughout the drug development process.

Therapies placed on fast track by the FDA also may be eligible for future designations, like accelerated approval and priority review, if other conditions are met.

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“Fast track designation for CB-011 allows us instrumental interactions with the FDA as we progress our clinical development and regulatory plans for CB-011,” Syed Rizvi, MD, chief medical officer of Caribou, said in a company press release.

Caribou launched the two-part Phase 1 clinical trial, called CaMMouflage (NCT05722418), to evaluate CB-011 in up to 50 adults with relapsed or refractory multiple myeloma.

“This designation could not be more timely as we recently dosed our first patient in the … trial,” Rizvi said.

CaMMouflage participants must have received at least three prior lines of treatment, including a proteasome inhibitor, an immunomodulator, and an anti-CD38 monoclonal antibody therapy. The study still may be recruiting patients at sites in New York, Ohio, and Tennessee.

In the trial’s first part, participants will be given varying doses of CB-011, with the aim of identifying the highest dose that can be given without unacceptable safety issues. Its second part will assess how patients respond to the selected dose after a year of treatment.

CB-011 is a type of CAR T-cell therapy, a form of treatment that utilizes immune T-cells that are genetically modified to enhance their ability to destroy myeloma cells. While most of these therapies use T-cells that are taken from a patient and modified in a lab, CB-011 is an allogenic or “off-the-shelf” CAR T-cell therapy that uses donor cells.

Designed with Caribou’s genome-editing technology, called Cas12a CRISPR hybrid RNA-DNA, CB-011 cells produce a receptor protein that recognizes BCMA, a protein found at high levels on myeloma cells. Additional modifications aim to prevent the donor cells from being rejected by the patient’s immune system.

“Our goal is to develop CB-011 as a readily available off-the-shelf treatment option for patients with relapsed or refractory multiple myeloma to overcome the need for [blood filtering] or bridging therapy, variable quality and long manufacturing timelines, manufacturing failures, or the inability to bear the burden of treatments that require frequent dosing over several months,” Rizvi said.