FDA grants fast track designation to CAR T-cell therapy for SLE

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FDA grants fast track designation to CAR T-cell therapy for SLE

Caribou's CB-010 candidate designed for treatment-resistant lupus

by Andrea Lobo, PhD

The U.S. Food and Drug Administration (FDA) has granted fast track designation to Caribou Biosciences‘ CAR T-cell therapy CB-010 for systemic lupus erythematosus (SLE) — the most common form of lupus — that’s resistant to available treatments, the company announced in a press release.

This designation is granted to accelerate the development of new treatments that have the potential to fill unmet medical needs in serious medical conditions. It gives Caribou access to certain benefits, including more frequent communications with the FDA during the development process, as well as eligibility for programs enabling faster regulatory reviews if certain criteria are met.

A Phase 1 trial evaluating CB-010’s safety and early efficacy in people with lupus nephritis and extrarenal lupus, two subcategories of SLE, is on track to start by year’s end, Caribou said.

Additionally, the FDA granted fast track designation to CB-012, the company’s CAR T-cell therapy candidate for relapsed or refractory (hard-to-treat) acute myeloid leukemia, a type of blood and bone marrow cancer.

“We are pleased to receive fast track designations from the FDA. This is a testament to the significant unmet need [in these two conditions] and the potential of CB-010 and CB-012 as readily available, off-the-shelf CAR-T cell therapies,” said Tina Albertson, MD, PhD, Caribou’s chief medical officer.

Fast track designation follows FDA green light for Phase 1 trial

In lupus, immune B-cells produce self-reactive antibodies that attack the body’s own healthy tissues. Lupus nephritis, a common complication of lupus characterized by kidney inflammation and damage, develops in about half of patients, with most failing to respond to treatment with standard immunosuppressant therapies.

CB-010 is being developed to treat both lupus nephritis and extrarenal lupus, a form of the disease in which other organs are affected.

As a CAR T-cell therapy, CB-010 uses immune T-cells collected from healthy donors, which are modified in the lab to be equipped with a chimeric antigen receptor (CAR) that specifically targets CD19, a protein found on the surface of B-cells. Once equipped with this CAR, the T-cells can specifically target and destroy the B-cells that are implicated in lupus.

Moreover, CB-010 cells are modified to lack a protein called PD-1. This is meant to reduce CAR T-cell exhaustion, a process by which modified T-cells lose their ability to target and kill B-cells.

The therapy is considered off-the-shelf because collected cells come from donors instead of patients, and they can be stored and used as needed. Also, cells collected from one donor can be used to treat several patients.

In the upcoming Phase 1 trial, called GALLOP, adults with lupus will receive a single dose of the therapy after completing a cycle of treatment with two different chemotherapy agents to wipe out their immune cells and make room for the modified cells.

The trial was given the green light by the FDA earlier this year.

We are eager to expand the clinical development program for CB-010 into [lupus nephritis and extrarenal lupus], prevalent and severe autoimmune diseases.

CB-010 also is being tested as a potential treatment for refractory B-cell non-Hodgkin lymphoma, a type of blood cancer, in a Phase 1 clinical trial dubbed ANTLER (NCT04637763).

“Based on previously reported data in our ongoing trial for patients with relapsed or refractory B cell non-Hodgkin lymphoma, CB-010 has shown encouraging safety, efficacy, and prolonged B cell [reduction] following a single dose,” Albertson said. “We are eager to expand the clinical development program for CB-010 into [lupus nephritis and extrarenal lupus], prevalent and severe autoimmune diseases.”

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