FDA Grants Fast Track Designation to ADI-001 for Treatment of Refractory Systemic Lupus Erythematosus

The FDA has granted fast track designation to ADI-001 for the treatment of refractory systemic lupus erythematosus (SLE) for extrarenal involvement, according to a news release from Adicet Bio. This new designation will allow for expedited review and optimized development of ADI-001, which could fill a major unmet need for this patient population and provide pharmacists with more options for patient treatment.¹

Lupus can cause debilitating rashes and other difficult complications. | Image Credit: © Alessandro Grandini | stock.adobe.com

An investigational allogeneic gamma delta chimeric antigen receptor (CAR) T cell therapy that targets CD20 for the treatment of autoimmune conditions, ADI-001 has also been granted FTD by the FDA for the treatment of relapsed/refractory class III or class IV lupus nephritis (LN).¹ A phase 1 multicenter, open-label, dose-finding, and dose-expansion clinical trial (NCT06375993) is currently ongoing to investigate the impacts of ADI-001 in patients with SLE, in addition to patients with systemic sclerosis, idiopathic inflammatory myopathy, and stiff person syndrome.^2,3

Patients enrolled in the study receive a single dose of ADI-001, with safety assessments conducted on day 28 and at months 3, 6, 9, 12, 18, and 24. The trial’s primary objective is to evaluate the tolerability and safety of ADI-001, while secondary objectives involve determining pharmacodynamics, cellular kinetics, and accurate disease activity scores for each indication.²

Previously, ADI-001 was evaluated in a study for treatment of adults with relapsed/refractory advanced B-cell lymphoma. Investigators determined that CAR T cells generated from ADI-001 were well-tolerated and elicited a favorable safety profile among patients, along with encouraging preliminary efficacy. In an important development, patients who achieved a complete response at day 28 appeared disease-free at the 3-month follow-up point.⁴

SLE is a chronic autoimmune disease that occurs when a patient’s immune system, which typically assists the body in protection from disease and infection, attacks its own tissue. These attacks can cause inflammation and possibly permanent, widespread tissue damage. This damage can be spread throughout the entire body, affecting the skin, heart, lungs, kidneys, circulating blood cells, brain, and other areas.⁵

Symptoms can vary based on the patient and can range from mild to severe. They also may vary regarding the location; they could affect one area of the body, or symptoms may spread and affect multiple locations at once. However, commonly reported symptoms include arthritis, fevers, a rash, sores, or swelling, among others. Inflammation that occurs throughout the body is a major contributor to poor outcomes and can cause kidney damage or heart problems.⁵

Patients who may be experiencing these symptoms should report them to their pharmacist or other trusted health care professional, where they can be counseled on the appropriate course of treatment. Pharmacists should be prepared to educate patients on the signs and symptoms of SLE and note the clinical development of ADI-001 for future use in this patient population.

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. News Release. Released February 5, 2025. Accessed February 5, 2025. https://www.businesswire.com/news/home/20250205155838/en/Adicet-Bio-Receives-FDA-Fast-Track-Designation-for-ADI-001-for-the-Treatment-of-Refractory-Systemic-Lupus-Erythematosus-SLE-with-Extrarenal-Involvement

2. ClinicalTrials.gov. A phase 1 study of ADI-001 in autoimmune disease. National Library of Medicine. Last Updated January 13, 2025. Accessed February 5, 2025. https://clinicaltrials.gov/study/NCT06375993

3. Adicet Bio. Adicet Bio announces first lupus nephritis patient dosed in phase 1 clinical trial of ADI-001 in autoimmune diseases. News Release. Released November 18, 2024. Accessed February 5, 2025. https://www.businesswire.com/news/home/20241118011893/en/Adicet-Bio-Announces-First-Lupus-Nephritis-Patient-Dosed-in-Phase-1-Clinical-Trial-of-ADI-001-in-Autoimmune-Diseases

4. Neelapu SS, Hamadani M, Miklos DB, et al. A phase 1 study of ADI-001: Anti-CD20 CAR-engineered allogeneic gamma delta (γδ) T cells in adults with B-cell malignancies. Hematologic Malignancies. 2022;40(16). doi:10.1200/JCO.2022.40.16_suppl.7509

5. National Institute of Arthritis and Musculoskeletal and Skin Diseases. Overview of systemic lupus erythematosus (lupus). Last Updated October 2022. Accessed February 5, 2025. https://www.niams.nih.gov/health-topics/lupus#:~:text=Systemic%20lupus%20erythematosus%20(lupus)%20is,disease%2C%20attacks%20its%20own%20tissues.