FDA puts CAR T-cell therapy ADI-001 on fast track

Phase 1 study for lupus nephritis to begin soon, Adicet says

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The U.S. Food and Drug Administration (FDA) has granted fast track designation to CAR T-cell therapy ADI-001 as a potential treatment for lupus nephritis, a lupus complication marked by kidney damage and inflammation.

The FDA gives fast track status to experimental treatments that have the potential to fill unmet medical needs in the treatment of serious conditions. The designation is intended to speed the development of needed therapies, and gives ADI-001’s developer, Adicet Bio, access to certain perks, such as more frequent communication with the FDA throughout the drug development process.

“The FDA’s decision to grant ADI-001 fast track designation for lupus nephritis underscores the urgent need for new therapies for this chronic disease,” Chen Schor, president and CEO of Adicet, said in a company press release.

CAR T-cell therapy is a type of cell therapy that’s attracted substantial interest as a potential treatment for lupus. These therapies use T-cells — immune cells that have the ability to destroy other cells —  that are engineered to be equipped with chimeric antigen receptor, or CAR, that directs them to attack and eliminate other cells having specific protein markers.

ADI-001 specifically uses a subset of T-cells, called gamma delta T-cells, which naturally home in to tissues. This makes them an attractive choice for autoimmune diseases that attack and damage specific organs and tissues, including the kidneys. These T-cells are equipped with a CAR that targets CD20, a protein found on the surface of B-cells — another type of immune cell that plays a central role in the autoimmune attack that drives lupus. By depleting B-cells, ADI-001 is expected to reduce disease activity.

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CAR T-cell therapy with donor-derived cells

Most CAR T-cell therapies are autologous treatments that involve harvesting a patient’s own T-cells, engineering them in a lab to equip them with the intended CAR, and then infusing them back into the patient. ADI-001, however, is designed to be an allogeneic, or off-the-shelf, therapy. This means that rather than being harvested from the patient who is to receive treatment, T-cells are isolated from healthy donors. After being modified and expanded in the lab, donor-derived T-cells can be used to treat multiple patients.

ADI-001 is expected to enter clinical testing for lupus nephritis in the coming weeks. “We plan to initiate our Phase 1 clinical study in lupus nephritis later this month,” Schor said.

Adicet is also developing ADI-001 as a potential treatment for certain blood cancers that are characterized by the uncontrolled growth of B-cells. According to Schor, early clinical data from cancer patients has indicated that the off-the-shelf therapy is able to destroy B-cells just as well as autologous CAR T-cell therapies.

“We believe we are well positioned to expand our autoimmune program to address additional indications beyond lupus nephritis,” Schor said, noting that Adicet will provide its investors with a “comprehensive update” about ADI-001 and its other experimental therapies for autoimmune diseases “in the near term.”