FDA grants ADI-001 fast track status for treatment-resistant SLE
Adicet Bio’s CAR T-cell therapy aims to reduce disease activity in patients
The U.S. Food and Drug Administration (FDA) has granted fast track status to ADI-001, Adicet Bio’s CAR T-cell therapy for systemic lupus erythematosus (SLE) that’s resistant to available treatments, the company announced in a press release.
The designation follows the FDA’s award last year of fast track status for lupus nephritis, a common complication of lupus marked by kidney damage and inflammation. The newly granted status covers SLE with extrarenal involvement, or symptoms affecting organs other than the kidneys.
Fast track designation is granted to facilitate and accelerate the regulatory review of therapies being developed for serious conditions for which there is an unmet medical need.
SLE is the most common type of lupus, an autoimmune disease in which the body’s immune cells produce autoantibodies that mistakenly attack healthy tissues. Depending on the tissue affected, the disease can lead to a wide range of symptoms.
Besides affecting the kidneys, the disease commonly leads to skin issues, joint problems, muscle pain, and fatigue. It may also affect the nervous system, eyes, lungs, heart, and digestive system.
Fast track status for SLE follows similar designation last year for lupus nephritis
To treat SLE, Adicet Bio is advancing ADI-001, its lead product candidate and a first-in-class CAR T cell therapy,
Such therapies use T-cells — immune cells that are capable of destroying other cells — engineered to harbor a chimeric antigen receptor, or CAR, that directs them to attack and eliminate other cells containing specific proteins.
ADI-001 specifically uses gamma delta T-cells, a type of T-cells that naturally home in on tissues, obtained from healthy donors. After being harvested, the cells are modified in the lab to be equipped with a CAR that targets CD20, a protein found on the surface of B-cells. These immune cells are those that produce the self-reactive antibodies that drive lupus. By lowering the number of B-cells, ADI-001 is expected to reduce disease activity.
The therapy’s safety and efficacy are being evaluated in a Phase 1 clinical trial (NCT06375993) that’s now recruiting adults with lupus nephritis at a single site in New York. Individuals with SLE and other autoimmune conditions are expected to start being enrolled in the study in the coming months.
Participants will receive a single infusion of ADI-001. Treatment response and safety will then be assessed after about one month, and for up to two years.
Before receiving treatment, patients will complete a short course of chemotherapy to kill their immune cells and make room for the CAR T-cells.
The trial’s main goal is to assess the incidence and severity of adverse events. Secondary goals include assessing the treatment’s pharmacological properties, particularly its ability to reduce the number of B-cells and the levels of self-reactive antibodies. Efficacy endpoints include evaluating the treatment’s ability to reduce disease activity and severity, using the SLE Disease Activity Index 2000.
ADI-001’s safety and tolerability were initially assessed in the Phase 1 GLEAN-1 trial (NCT04735471), which enrolled people with B-cell lymphoma, a type of blood cancer. The treatment was generally well tolerated and effectively reduced B-cell counts as intended.
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