On December 10, 2022 Gracell Biotechnologies Inc. ("Gracell" or the "Company", NASDAQ: GRCL), a global clinical-stage biopharmaceutical company dedicated to developing highly efficacious and affordable cell therapies for the treatment of cancer, reported that the clinical data from its ongoing Phase 1, investigator-initiated trial (IIT) in China evaluating FasTCAR-enabled GC012F as first-line therapy in transplant-eligible, high-risk, newly diagnosed multiple myeloma (NDMM) patients (Press release, Gracell Biotechnologies, DEC 11, 2022, View Source [SID1234625037]). Data has been presented in an oral session at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, held Dec. 10-13 in New Orleans. Patients in the study achieved a 100% overall response rate (ORR) and 100% minimal residual disease (MRD) negativity in all dose levels.

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GC012F is an autologous CAR-T therapeutic candidate dual-targeting B cell maturation antigen (BCMA) and CD19, and utilizes Gracell’s proprietary FasTCAR next-day manufacturing platform.

As of the Oct. 14, 2022 data cutoff date, 16 transplant-eligible NDMM patients had received GC012F infusion in the clinical trial. All patients had multiple high-risk features. After receiving a conditioning lymphodepletion regimen of cyclophosphamide and fludarabine, patients were treated with GC012F as a single infusion with one of three dose levels: 1×105 cells/kg, 2×105 cells/kg and 3×105 cells/kg.

As of Oct. 14, 2022, among the 16 evaluable patients with the median follow-up time of eight months (ranging from 1.3 to 15.4 months):

ORR was 100%
87.5% (14/16) of patients achieved stringent complete response (sCR). Patients continue to be followed for deepening responses
100% of evaluable patients achieved MRD negativity in all dose levels
100% of evaluable patients achieved MRD negativity at months 1, 6 and 12
100% of patients experienced robust CAR-T cell expansion with long persistence in all dose levels
The clinical data also demonstrated an excellent safety profile:

Only 25% (4/16) of patients experienced Grade 1-2 cytokine release syndrome (CRS); no patients experienced Grade 3-5 CRS
No immune effector cell-associated neurotoxicity syndrome (ICANS) or other neurotoxicity of any grade had been observed
"This clinical data brings us great optimism. GC012F has demonstrated an impressive 100% ORR, 100% MRD negativity and 87.5% sCR, as well as an outstanding safety profile, among newly-diagnosed multiple myeloma patients, showing tremendous potential for substantial improvement over currently available therapies," said Dr. Wendy Li, Gracell’s Chief Medical Officer. "We are pleased to share this data with the leading experts in hematology and oncology at ASH (Free ASH Whitepaper) 2022. We believe that the data underscores GC012F’s significant potential as a safe and effective therapy for NDMM patients. In addition, GC012F is developed using our proprietary FasTCAR next-day manufacturing platform, which could greatly expedite the delivery of this much-needed therapy to patients."

About GC012F

GC012F is a FasTCAR-enabled BCMA/CD19 dual-targeting CAR-T product candidate that is currently being evaluated in IIT studies in China for the treatment of multiple myeloma and B-cell non-Hodgkin’s lymphoma. GC012F simultaneously targets CD19 and BCMA to drive fast, deep and durable responses, which can potentially improve efficacy and reduce relapse in multiple myeloma and B-NHL patients.

About FasTCAR

CAR-T cells manufactured on Gracell’s proprietary FasTCAR platform appear younger, less exhausted and show enhanced proliferation, persistence, bone marrow migration and tumor cell clearance activities as demonstrated in preclinical studies. With next-day manufacturing, FasTCAR is able to significantly improve cell production efficiency which may result in meaningful cost savings, and, together with fast release time, enables enhanced accessibility of cell therapies for cancer patients.