China clears Phase 1/2 trial of GC012F in difficult-to-treat SLE

China’s regulatory body has given Gracell Biotechnologies the green light to launch a Phase 1/2 clinical trial testing GC012F, an experimental dual-targeting CAR T-cell therapy, in people with difficult-to-treat systemic lupus erythematosus (SLE).

The decision by China’s National Medical Products Administration comes shortly after the U.S. Food and Drug Administration cleared an investigational new drug application for another Phase 1/2 trial, expected to start this year.

“This milestone marks our rapid progress in advancing development of GC012F in rSLE [refractory, or treatment-resistant, SLE], an autoimmune disease with high unmet need,” William Cao, PhD, Gracell’s founder, chairman, and CEO, said in a press release.

In SLE, autoimmune attacks are caused by self-reactive antibodies produced by immune B-cells against the body’s own tissues. The goal of treatment is to keep the immune system in check, but some people fail to respond to immunosuppressants or other medications.

GC012F is a form of CAR T-cell therapy specifically engineered to eliminate B-cells. The process involves retrieving T-cells, a different type of immune cell, from the patient and altering them to bear a synthetic surface protein known as a chimeric antigen receptor (CAR). This CAR guides the modified cells toward a predefined target CD19 and BCMA, two proteins on the surface of B-cells, which depletes B-cells and eases the symptoms of SLE.

“We’re relentlessly pursing clinical development of an innovative CD19/BCMA dual-targeting approach, which seeks to offer differentiated efficacy over other investigational therapies that only target CD19,” Cao said.

GC012F is being tested for difficult-to-treat SLE in an open-label, investigator-initiated Phase 1 clinical trial (NCT05846347) that is underway at a single site in Zhejiang University in Hangzhou, China. It is expected to conclude in 2025.