FDA grants fast track status to CAR T-cell therapy CABA-201 for lupus
Cabaletta Bio to test treatment in lupus patients in Phase 1/2 trial
CABA-201, an experimental cell therapy for lupus and other autoimmune disorders, has been granted fast track status by the U.S. Food and Drug Administration (FDA).
The FDA awards this designation to therapies with the potential to address unmet medical needs in the treatment of serious conditions. Fast track status is intended to speed the development of potentially important therapies with various perks.
CABA-201 is being developed by Cabaletta Bio, which licensed the therapy from IASO Biotherapeutics last year. This new designation for CABA-201 came barely a month after the FDA gave Cabaletta the all-clear to launch a Phase 1/2 clinical trial testing the therapy in people with lupus.
“We believe the FDA’s decision to grant Fast Track Designation for CABA-201 underscores the unmet need for a treatment that has the potential to provide deep and durable responses for people living with lupus,” David Chang, MD, Cabaletta’s chief medical officer, said in a company press release.
Perks of fast track status aim to speed therapy development
Lupus is caused by the immune system erroneously attacking the body’s own healthy tissue. CABA-201 is designed to destroy B-cells — a type of antibody-producing immune cell that plays a central role in driving the autoimmune attack. The aim of the therapy is to be a one-time treatment that temporarily, but completely, eliminates all of a patient’s B-cells, allowing the immune system to be reset.
The therapy works by using a different type of immune cell: T-cells. With CABA-201, T-cells are collected from a patient, then taken to a laboratory, where scientists engineer them to express a chimeric antigen receptor (CAR) — a protein that directs T-cells to attack a specific target. In this case, T-cells are equipped with a CAR that leads them to attack CD19, a protein that is found on the surface of B-cells.
In addition to the CD19-CAR protein, CABA-201 cells also are engineered to express 4-1BB, a stimulatory molecule that enhances their ability to kill target cells. A study published last year reported that a similar type of CAR T-cell therapy, given under a compassionate use program, led to complete disease remission in five people with lupus who had not responded to other treatments.
The upcoming Phase 1/2 study is expected to enroll six people with systemic lupus erythematosus (SLE) who have lupus nephritis, or kidney inflammation. Another six SLE patients with no kidney involvement also will be enrolled. All participants will be treated with CABA-201 at an initial dose of 1 million cells per kilogram of body weight — the same dose that was used in the compassionate use program.
Prior to the infusion of the cell therapy, patients will undergo a preconditioning regimen with the chemotherapy agents fludarabine and cyclophosphamide. The goal of this initial treatment is to destroy existing immune cells in the patient’s body, making room for the modified CAR T-cells.
We believe the FDA’s decision to grant Fast Track Designation for CABA-201 underscores the unmet need for a treatment that has the potential to provide deep and durable responses for people living with lupus.
“We look forward to initiating the Phase 1/2 trial for CABA-201 and further evaluating its therapeutic potential for patients in need,” Chang said.
The new fast track status designation for CABA-201 will qualify Cabaletta for certain benefits, including more frequent meetings with the FDA during the clinical development process. It also gives the therapy potential eligibility for accelerated approval and/or priority review.