Gracell’s CAR T-Cell Therapy to be Evaluated in Lupus


GC012F recently yielded a 100% ORR in an investigator-initiated trial in patients with newly diagnosed multiple myeloma.

The FDA has granted investigational new drug (IND) clearance to Gracell Biotechnologies for its chimeric antigen receptor (CAR) T-cell therapy GC012F to be evaluated in a phase 1/2 trial in patients with refractory systemic lupus erythematosus (rSLE).1

“We are excited to expand the clinical development of our lead FasTCAR asset, GC012F, for treatment of rSLE in the United States,” William Cao, PhD, founder, chairman and chief executive officer, Gracell, said in a statement.1 “This progress marks the second US IND clearance for GC012F, a notable milestone. 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. Additionally, what sets GC012F apart is its consistently favorable safety profile demonstrated by the absence of neurotoxicity in 60 patients treated across three investigator-initiated (IIT) studies. 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.”

Gracell plans to initiate the phase 1 portion of the trial in 2024. This initial part of the trial will evaluate the safety and tolerability of the GC012F, determine the recommended phase 2 dose, and characterize pharmacokinetics in patients with rSLE. SLE is a B-cell-mediated autoimmune disease and immunosuppressants are used as the current standard of care.

GC012F is an autologous therapy developed with the use of Gracell’s proprietary FasTCAR next-day manufacturing platform and dual targets B cell maturation antigen (BCMA) and CD19. The IND clearance marks Gracell’s first US foray into autoimmune disease, although GC012F has been evaluated in multiple trials. These trials include the phase 1b/2 IND study for the treatment of relapsed/refractory multiple myeloma (RRMM) in the US, and in 4 IIT studies for the treatment of rSLE, RRMM, newly-diagnosed MM, and B-cell non-Hodgkin lymphoma.

READ MORE: Nkarta Enters the Lupus Nephritis CAR Cell Therapy Race With NKX019 IND Clearance

Gracell will present updated clinical results from the newly diagnosed MM IIT trial (NCT04935580) at the 65ᵗʰ American Society of Hematology Annual Meeting & Exposition in December 2023, but shared that the new data include an overall response rate (ORR) of 100% and minimal residual disease negative stringent complete response (MRD- sCR) rate of 95.5%.

Most recently, Gracell announced data from that trial current as of an August 1, 2023 cut-off date in which all 19 treated patients (100%) achieved an sCR with minimal residual disease negativity, with a sensitivity of 10-6 assessed by Euroflow. The patients’ follow-up ranged from 3.1 to 24.5 months (median, 15.3) and the median duration of response was not reached as of the cut-off. Gracell noted that the trial included patients with NDMM who were transplant eligible and that each of the patients, who were treated at 3 different dose levels (1x105 cells/kg, 2x105 cells/kg, and 3x105 cells/kg), had high-risk disease features. These included classification as stage II or stage III on the Revised International Staging System in 89% of patients and extramedullary plasmacytoma in 63% of patients.2

1. Gracell Biotechnologies announces FDA clearance of IND application for phase 1/2 clinical trial of FasTCAR-T GC012F for the treatment of refractory systemic lupus erythematosus. News release.
2. Gracell Biotechnologies reports updated clinical data for FasTCAR-T GC012F for high-risk, newly diagnosed multiple myeloma, demonstrating 100% stringent complete response rate. News release. Gracell Biotechnologies Inc. September 27, 2023. Accessed October 2, 2023.
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