Cell therapy ADI-001 reduces disease activity in lupus patients
Phase 1 trial data could set stage for launch of subsequent study in 2026
A single infusion of the experimental cell therapy ADI-001 led to reductions in disease activity for seven lupus patients in a clinical trial, according to preliminary data announced by its developer Adicet Bio. No serious side effects reported.
“These preliminary results reinforce our belief that ADI-001 has the potential to transform the treatment landscape for autoimmune diseases,” Chen Schor, president and CEO of Adicet, said in a company press release.
Adicet is planning to meet with the U.S. Food and Drug Administration (FDA) to go over the data in early 2026. Pending feedback from the regulatory agency, the company hopes to soon launch a pivotal Phase 2 study that could form the basis for applications seeking the approval of ADI-001 as a lupus treatment.
“With these data in hand, we plan to engage with the FDA in the first quarter of 2026 to discuss the design of a potentially pivotal study that we anticipate initiating in the second quarter of 2026,” Schor said.
ADI-001 works by using T-cells to target B-cells
Lupus is driven by antibodies that target the body’s own healthy tissues. Antibodies are produced by B-cells, a type of immune cell. ADI-001 is designed to deplete or lower the number of B-cells, thereby reducing disease activity.
ADI-001 works by using T-cells, another type of immune cell, which are equipped with a chimeric antigen receptor (CAR) that targets a B-cell protein called CD20. Other CAR T-cell therapies are in development for lupus, but they mostly work by collecting T-cells from a patient, reprogramming them in a lab, and then transfusing them back into the patient. By contrast, ADI-001 uses T-cells from healthy donors, with the aim of creating an off-the-shelf therapy that can simplify treatment.
The new data came from the first seven lupus patients treated with ADI-001 as part of an ongoing Phase 1 clinical trial (NCT06375993). That trial is still enrolling patients at several sites, and Adicet said that lupus patients will continue to be enrolled until the pivotal study starts enrolling participants.
“We have now activated over 25 clinical sites globally, which is a testament to our team’s focus and execution as well as demonstrated investigator interest in ADI-001,” said Julie Maltzman, MD, Adicet’s chief medical officer.
Disease activity reduced in all 7 patients
Of the seven initial lupus patients, five had lupus nephritis, a severe manifestation of lupus marked by kidney damage. The other two had systemic lupus erythematosus (SLE), the most common form of lupus, which can affect tissues and organs throughout the body.
Each of the seven patients received a single infusion of ADI-001. As of the latest follow-up, all seven patients have either stopped taking corticosteroids or have reduced their corticosteroid dose to very low doses, and all have discontinued other immunosuppressant medications.
According to Adicet, the latest data show that all seven patients have had rapid and sustained reductions in standard assessments of disease activity, namely the SLE Disease Activity Index (SLEDAI-2K) score and the Physician’s Global Assessment. Follow-up times ranged from two to nine months.
In the five lupus nephritis patients, ADI-001 also led to reductions in the severity of kidney disease, with three of the five patients achieving a complete renal response and going into remission. This essentially means they no longer showed signs of disease activity and kidney dysfunction.
“We are extremely encouraged to share preliminary results highlighting disease remissions or a halting of disease progression in the first seven patients treated in our Phase 1 trial,” Maltzman said.
‘ADI-001 has also demonstrated a favorable safety and tolerability profile’
Safety data have overall been positive, with no serious side effects reported. One participant had a mild respiratory infection, and two experienced mild cytokine release syndrome, an inflammatory reaction associated with CAR T-cell therapies that can cause flu-like symptoms such as fever.
“ADI-001 has also demonstrated a favorable safety and tolerability profile … which is promising and has the potential to support outpatient administration of ADI-001. In addition, ADI-001’s potential availability as an off-the-shelf therapy may allow for far broader accessibility to many more patients and treating physicians,” Maltzman said.
Available biomarker data from the Phase 1 study have indicated that ADI-001 treatment can destroy disease-driving B-cells and make room for new B-cells to grow, indicating that the therapy can effectively reset the immune system to reduce lupus disease activity.
“We observed clear evidence of immune reset with subsequent emergence of naïve B cell repertoire following a single treatment of ADI-001,” Schor said. “ADI-001’s potential to reset the immune system with a one-time, off-the-shelf therapy and a generally favorable safety profile could be practice-changing for patients and providers alike.”
The FDA has previously granted ADI fast track status for both lupus nephritis and treatment-resistant SLE. This designation aims to speed the development of new therapies that have the potential to fill unmet medical needs for serious conditions.
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