On April 28, 2020 Gracell Biotechnologies Co., Ltd. ("Gracell"), a clinical-stage immune cell and gene therapy company, reported data of its first-in-human clinical trial for Universal TruUCAR GC027 in relapsed or refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL) patients (Press release, Gracell Biotechnologies, APR 28, 2020, View Source [SID1234556719]). The data was presented in the Adoptive Cell Transfer Therapy section during the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting held on April 28.

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T-ALL, a form of acute lymphoblastic leukemia, consisting of 20-25% of adult ALL, and 12-15% of pediatric ALL, represents a hard-to-treat disease with a high unmet clinical need.[1] Outcome of relapsed and refractory T-ALL remains poor, with very limited treatment options available. Most T-ALL patients relapse within two years after multi-agent chemotherapy regimens. The study of TruUCAR GC027 in relapsed and refractory T-ALL reports early efficacy outcomes of five patients treated in this area of high unmet medical need.

The clinical investigator initial trial (IIT) intends to evaluate safety and efficacy of TruUCAR GC027, the first-in-human, universal CAR-T therapy for R/R T-ALL. As of February, the study enrolled a total of five patients with R/R T-ALL, with median prior lines of therapy 5 (range 1-9). Baseline bone marrow tumor burden was 38.2% (range 4-80.2). All patients received a single infusion of TruUCAR GC027 in one of three dose levels: 0.6*10^7cells/kg, 1.0*10^7cells/kg or 1.5*10^7cells/kg. Notably, these patients were not HLA matched, and no one accepted post-infusion hematopoietic stem cell transplantation (HSCT).

Treatment efficacy was assessed in five patients with 28 days of follow-up, of which:

Five (100%) achieved a complete remission with or without complete blood count recovery (CR/CRi);
Four (80%) achieved minimum residual disease negative complete remission (MRD-CR).
All five patients tolerated the single infusion of TruUCAR GC027 with no neurotoxicity events or acute graft-versus-host disease (aGvHD) observed. Cytokine release syndrome (CRS) presented in all patients at any grade.

"We are delighted to report the outcome on the first five patients treated with TruUCAR GC027. These promising preliminary results are encouraging and warrant further evaluation of the therapy in this area of high unmet clinical need." said Dr. Martina Sersch, CMO of Gracell.

About TruUCAR

TruUCAR is Gracell’s proprietary and patented platform technology, with selected genes being edited to avoid GvHD and immune rejection without using strong immunosuppressive drugs. In addition to T-ALL antigen, the platform technology can also be implemented for other targets of hematological malignancies.

About GC027

GC027 is an investigational, off-the-shelf CAR-T cell therapy, redirected to CD7 for the treatment of T cell malignancies. GC027 was manufactured from T cells of human leukocyte antigen (HLA) unmatched healthy donors using TruUCAR technology, which is expected to improve efficacy and reduce production time, available for off-the-shelf use in a timely manner.

About T-ALL

T – Lymphoblastic Leukemia (T-ALL) is an aggressive form of acute lymphoblastic leukemia, with a diffuse invasion of bone marrow and peripheral blood. In 2015, T-ALL affected around 876,000 people globally and resulted in 110,000 deaths worldwide. T-ALL compromises about 15%-20% of all children and adult acute lymphoblastic leukemia[2]. Current standard of care therapies for T-ALL are chemotherapy and stem cell transplantation. 40-50% of patients will experience relapse within two years following front line therapy with limited treatment options available[3][4]. Treatment of relapsed and refractory T-ALL remains a high unmet medical need.