Phase 1/2 Trial of GC012F in hard-to-treat SLE expected by 2024

CAR T-cell therapy is being tested in refractory patients in China

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The U.S. Food and Drug Administration (FDA) has given Gracell Biotechnologies the green light to start a Phase 1/2 clinical trial to test its experimental cell therapy GC012F in people with difficult-to-treat systemic lupus erythematosus (SLE).

The study’s Phase 1 portion should start in 2024 to assess the safety, tolerability, and pharmacokinetics of GC012F and determine the optimal dose for Phase 2 testing, according to Gracell. Pharmacokinetics refers to a therapy’s movement into, through, and out of the body.

GC012F is being tested in refractory, or treatment-resistant, SLE patients within an investigator-sponsored Phase 1 trial (NCT05846347) at a single site in China. That study may still be recruiting participants.

“We are excited to expand the clinical development of our lead FasTCAR asset, GC012F, for treatment of [refractory SLE] in the United States,” William Cao, PhD, founder, chairman, and CEO of Gracell, said in a company press release.

SLE is caused by the immune system attacking healthy tissue. B-cells, a type of immune cell responsible for making antibodies, are one of the main contributors in the autoimmune attacks that drive the disease.

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A next-generation CAR T-cell therapy

GC012F is a CAR T-cell therapy designed to destroy B-cells. It involves collecting T-cells, another type of immune cell, from a patient and modifying them to carry a human-made surface protein, called a chimeric antigen receptor, or CAR, which directs the cells toward a specific target.

With GC012F, the T-cells are equipped with a CAR that targets two proteins on the surface of B-cells, CD19 and BCMA. Because T-cells can target and eliminate cells based on the proteins at their surface, infusing the B-cell-targeting T-cells into a patient should reduce SLE severity.

GC012F is manufactured using FasTCAR, Gracell’s proprietary technology that lets a patient’s T-cells be equipped with the CARs within a day so they can be given to the patient quickly after being harvested.

“As a next-generation CAR-T therapy, GC012F combines the innovative CD19/BCMA dual-targeting approach and our breakthrough FasTCAR next-day manufacturing technology, both of which could potentially provide meaningful benefits to SLE patients,” Cao said.

The cell therapy is also being tested in trials of other diseases that are mediated by B-cells, including multiple myeloma, a rare cancer driven by B-cells’ uncontrolled growth.

GC012F has so far resulted in deep responses, with a favorable safety profile among the 60 people who’ve received it in trials, according to the company. None have experienced any neurological toxicity, a common safety problem with other CAR T-cell therapies, Cao said.

“What sets GC012F apart is its consistently favorable safety profile,” Cao said. “We look forward to developing GC012F as a transformative therapy for SLE patients, who are in urgent need of highly effective and safe treatment options.”