Phase 1 trial of CAR T-cell therapy will soon enroll AAV patients
New Adicet Bio study testing ADI-001 for lupus, other autoimmune diseases
A Phase 1 clinical trial evaluating the safety and preliminary efficacy of Adicet Bio’s CAR T-cell therapy ADI-001 in people with autoimmune diseases, including ANCA-associated vasculitis (AAV), has started recruiting certain participants — and it’s expected to enroll AAV patients by the end of the year — the company announced.
The newly launched trial (NCT06375993) is now open to adults with lupus nephritis, a type of kidney damage that is a common complication of lupus, another autoimmune disease.
Adicet said it expects to start recruiting people with systemic lupus erythematosus (SLE), the most common form of lupus, in the fourth quarter, or last three months, of this year, along with individuals with systemic sclerosis (SSc) and AAV.
“We expect to have several additional sites open for enrollment by the end of … 2024, and further increase the number of active sites during the first [months] of 2025,” Francesco Galimi, MD, PhD, Adicet’s senior vice president and chief medical officer, said in a company press release. “We look forward to reporting preliminary clinical data from this trial of ADI-001 [across all indications] in the first half of 2025.”
The announcement of the start of enrollment for this early trial follows the U.S. Food and Drug Administration’s green light earlier this year to expand the clinical development of ADI-001 beyond lupus nephritis, to include SLE, SSc, and AAV.
Phase 1 trial to test safety, tolerability of ADI-001
In people with autoimmune conditions like lupus and AAV, the body’s immune system wrongly launches an attack against healthy parts of the body.
AAV is caused by the production of self-reactive antibodies, called ANCAs, that ultimately result in inflammation and damage to small blood vessels. The disease can cause many different symptoms, including kidney, lung, and nervous system problems.
As a CAR T-cell therapy, ADI-001 involves the collection of T-cells — immune cells able to promote the death of other cells — from healthy donors. These cells are treated in the lab to produce a chimeric antigen receptor, or CAR, that directs them to attack and eliminate cells with specific protein markers.
ADI-001 specifically uses a subtype of T-cells, called gamma delta T-cells, that are present in many body tissues, making them an attractive choice for autoimmune diseases that attack and damage specific tissues, including small blood vessels.
In this therapy, T-cells carry a CAR-targeting CD20, a protein found at the surface of B-cells, the immune cells that produce ANCAs and other self-reactive antibodies. By reducing B-cell levels, ADI-001 is expected to reduce disease activity.
The favorable safety profile … and [data thus far position] ADI-001 to potentially bring a paradigm shift in the treatment of autoimmune diseases.
The therapy is also being tested as a potential treatment for B-cell cancers, with Phase 1 trial data showing a favorable safety profile, as well as CAR T-cell expansion, and preliminary efficacy.
“The favorable safety profile … and B cell depletion in peripheral blood and secondary lymphoid tissue [tissues where immune cells are activated] demonstrated with ADI-001 clinical experience to date, [position] ADI-001 to potentially bring a paradigm shift in the treatment of autoimmune diseases,” Galimi said.
In the new Phase 1 trial for autoimmune diseases, participants will receive a single dose of ADI-001, and undergo regular safety and response evaluations for up to two years.
The trial’s main goal is to evaluate the treatment’s safety and tolerability. Secondary goals will be assessing ADI-001’s cellular expansion, its effects on the body, and changes in the levels of self-reactive antibodies and disease activity scores for each indication.
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