FDA Fast Tracks CAR-T Therapy for Lupus

The US Food and Drug Administration (FDA) has granted Fast Track Designation to Adicet Bio’s ADI-001 for the potential treatment of adult patients with refractory systemic lupus erythematosus (SLE) with extrarenal involvement.1

Announced on February 5, 2025, the investigational allogeneic gamma delta chimeric antigen receptor (CAR) T cell therapy has now been granted Fast Track Designation for the treatment of relapsed/refractory class III or class IV lupus nephritis, and SLE with extrarenal involvement.1,2

ADI-001 targets CD20 for the treatment of autoimmune diseases and is being advanced across 6 autoimmune indications. In the phase 1 GLEAN trial, ADI-001 was shown to target B-cells via an anti-CD20 CAR and demonstrated robust exposure and complete CD19+ B-cell depletion both in peripheral blood and secondary lymphoid tissue.1

In November 2024, the first patient with lupus nephritis was dosed in the phase 1 clinical trial evaluating ADI-001 in autoimmune diseases, and patient enrollment is ongoing. According to a press release from Adicet, patient enrollment in SLE, systemic sclerosis, idiopathic inflammatory myopathy, and stiff person syndrome is expected to be initiated in the first quarter of 2025.3

The phase 1 study has 4 arms, enrolling lupus nephritis and SLE patients into one arm, systemic sclerosis patients into a second arm, idiopathic inflammatory myopathy and stiff person syndrome patients in a third arm and autoantibody associated vasculitis patients into a fourth arm. Enrolled patients will receive a single dose of ADI-001, with a dose-limiting toxicity window of 28 days.3

The primary objectives of the study are to evaluate the safety and tolerability of ADI-001. Secondary objectives include measuring cellular kinetics, pharmacodynamics, changes in autoantibody titers, and appropriate disease activity scores in each indication.3

“With clinical sites open for enrollment and additional sites that are expected to open in the near future, we anticipate sharing preliminary clinical data from the trial in the first half of 2025,” Francesco Galimi, MD, PhD, senior vice president and chief medical officer of Adicet Bio, said at the time of the dosing of the first patient.3

References

1. Adicet Bio. Adicet Bio Receives FDA Fast Track Designation for ADI-001 for the Treatment of Refractory Systemic Lupus Erythematosus (SLE) with Extrarenal Involvement. February 5, 2025. Accessed February 5, 2025. https://investor.adicetbio.com/news-releases/news-release-details/adicet-bio-receives-fda-fast-track-designation-adi-001-treatment
2. Adicet Bio. Adicet Bio Receives FDA Fast Track Designation for ADI-001 in Lupus Nephritis. June 5, 2024. Accessed February 5, 2025. https://investor.adicetbio.com/news-releases/news-release-details/adicet-bio-receives-fda-fast-track-designation-adi-001-lupus
3. Adicet Bio. Adicet Bio Announces First Lupus Nephritis Patient Dosed in Phase 1 Clinical Trial of ADI-001 in Autoimmune Diseases. November 18, 2024. Accessed February 5, 2025. https://investor.adicetbio.com/news-releases/news-release-details/adicet-bio-announces-first-lupus-nephritis-patient-dosed-phase-1