Unicar-Therapy's Breakthrough CAR-T Therapy ssCART-19 Showcased at ESMO 2024 with Promising Safety and Efficacy in Relapsed/Refractory B-ALL

Sep 20, 2024 08:00 CST Updated 08:00

Apricot capital

Venture Capital Institution

Unicar-Therapy

Developer of Immune Cell and Gene Therapy Products

The 2024 European Society for Medical Oncology (ESMO) Annual Meeting was held in Barcelona, Spain, from September 13 to 17 local time. Currently, the ESMO conference official website has disclosed the abstract results selected for the Mini Oral presentations.SHANGHAI UNICAR-THERAPY BIOMED-PHAMACEUTICAL TECHNOLOGY CO., LTD ConductedssCART-19 for the Treatment of Refractory/Relapsed Acute Lymphoblastic Leukemia (Including Central Nervous System Leukemia)The latest clinical research progress has been successfully selected.

ssCART-19 is aTargeted CD19-engineered autologous T-cell injection with IL-6 silencing expression function. In May this year, the product's treatment for refractory/recurrent acute lymphoblastic leukemia has been accepted by the Center for Drug Evaluation (CDE) of the China National Medical Products Administration.IncludedBreakthrough Therapy Drug。

According to the abstract introduction disclosed on the ESMO conference official website, although CAR-T therapy has shown better efficacy in the treatment of hematologic malignancies in recent years, severe cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) are still limiting its wide application, especially in patients with acute lymphoblastic leukemia. Therefore, SHANGHAI UNICAR-THERAPY BIOMED-PHAMACEUTICAL TECHNOLOGY CO., LTD developed an autologous CD19-specific CAR-T cell product, ssCART-19.It can innovatively combine shRNA technology to silence the IL-6 gene, reducing the overall intensity of cytokine release, thereby decreasing the incidence of severe CRS/ICANS, and ultimately improving the clinical safety of CAR-T therapy.。

The oral report selected for this ESMO conference is ssCART-19 in Chinese adult R/R B-cellAcute Lymphoblastic Leukemia (ALL)A single-arm, open-label phase 1 study conducted in patients. All patients received a lymphodepletion regimen prior to ssCART-19 infusion. The primary endpoint was safety, and the secondary endpoint was efficacy.

In the study, ssCART-19 at 1×10⁶/kg、5×10⁶/kg and 10×10⁶/kg at three dose levels (DL). As of the data cutoff date of October 31, 2023 (median follow-up of 19.6 months), 17 patients received ssCART-19 treatment, and 10 patients (58.8%) had a cell count exceeding 50%.Safety data show,The most common AEs were lymphocytopenia (94.2%), transient neutropenia (88.2%), leukopenia (88.2%), anemia (70.6%), and thrombocytopenia (58.8%).CRS occurred in 13 of 17 patients (76.5%),No CRS of grade 4 or above occurred.In the DL1 group, there was 1 case (10.0%) of Grade 3 CRS. Notably,No patients developed ICANS, and no deaths due to CRS or ICANS were reported.。

Efficacy data showed that, in evaluable patients (n=16),The ORR within 3 months reached 87.5%., Complete Remission (CR) RateAt 62.5%,Complete Remission with Incomplete Hematologic Recovery (CRi) RateIs 25.0%The 6-month median duration of response (DOR) rates for DL1, DL2, and DL3 were 100.0%, 80.0%, and 0.0%, respectively. Among them, seven patients had a sustained response for more than six months. Additionally,Patients with grade 2 central nervous system infiltration before ssCART-19 infusion did not develop ICANS and remained in remission after ssCART-19 treatment.。

Researchers believe that ssCART-19 has achieved a high remission rate in adult R/R B-cell ALL patients, and it can significantly improve the safety of CAR-T therapy, especially in patients with central nervous system infiltration.

About SHANGHAI UNICAR-THERAPY BIOMED-PHAMACEUTICAL TECHNOLOGY CO., LTD

SHANGHAI UNICAR-THERAPY BIOMED-PHAMACEUTICAL TECHNOLOGY CO., LTD. ("UNICAR-THERAPY" for short) is a leading biopharmaceutical company dedicated to cutting-edge cell therapy technology innovation and translation. It owns several original core technology platforms, including the SMART small nucleotide gene regulation empowerment technology platform, MADDS antibody innovative drug development technology platform, and the 6H production quality control platform. Currently, the company has established a billion-level gene and cell therapy research and industrial center in the North Shanghai Biomedical Park, creating an internationally first-class immunotherapy tumor innovation technology translation center and an advanced, high-end genetically engineered immune cell industry chain center. UNICAR-THERAPY has successfully treated thousands of advanced cancer patients through collaborations with many well-known medical institutions both domestically and internationally. UNICAR-THERAPY’s product pipeline is extensive, covering autologous CAR-T technology, universal CAR-T technology, and in vivo CAR technology, with indications spanning hematologic tumors, solid tumors, and autoimmune diseases. Currently, three first-in-class/first-in-safety products are in the registration clinical research stage. ssCART-19 injection can be used as a breakthrough treatment for CAR-T contraindicated central nervous system leukemia (commonly known as "brain leukemia"), having received FDA orphan drug designation and being included in the NMPA breakthrough therapy list, now entering the pivotal clinical stage; U16 injection is a non-CD19 target lymphoma treatment product with significant efficacy, soon to enter the pivotal clinical stage; U87 injection is a fully human CAR-T product, with proposed indications covering multiple malignant tumors such as lung cancer, triple-negative breast cancer, pancreatic cancer, nasopharyngeal cancer, and colorectal cancer, currently in Phase I of the registration clinical trial.