Huaxia Yingtai's LILRB4-Targeted STAR-T Cell Therapy YTS104 Receives IND Approval from China’s NMPA for Phase I Clinical Trial

On January 23, 2024, "YTS104 Cell Injection," a LILRB4-specific innovative target developed by Huaxia Yingtai (Beijing) Biotechnology Co., Ltd. based on its proprietary STAR-T platform, received the clinical trial implied permission from the Center for Drug Evaluation of the National Medical Products Administration for treating indications such as relapsed/refractory acute myeloid leukemia (AML) and chronic myelomonocytic leukemia (CMML). This marks another milestone breakthrough for the company following the IND approval of its CD19/CD20 dual-target STAR-T product.

YTS104 Cell Injection is an autologous cell therapy innovative drug developed by Huaxia Yingtai (Beijing) Biotechnology Co., Ltd. based on the STAR-T technology co-pioneered with Tsinghua University.The product advantage lies in the use of two independently screened LILRB4 nanobodies with different epitopes, which are connected to the α and β chains of the STAR structure, respectively. This is expected to provide better antigen recognition and killing effects. YTS104 cell injection showed positive data in early studies, effectively killing target-positive tumor cells, and was presented as a poster at the 2023 American Association for Cancer Research (AACR). Previously, YTS104 cell injection received "Orphan Drug" designation from the U.S. FDA in December 2022.

YTS104 targets the LILRB4 (also known as ILT3, CD85k), a myeloid immune inhibitory receptor., which is highly expressed on monocytic AML blasts (FAB M4 and M5 AML subtypes), making it an ideal therapeutic target for monocytic AML. At the same time, the LILRB4 target represents a differentiated exploration from the current CAR-T cell therapy research, which is more concentrated on targets such as CD19 and BCMA in hematologic malignancies.

The indications for YTS104 cell injection include relapsed/refractory acute myeloid leukemia (AML), chronic myelomonocytic leukemia (CMML), and other diseases.Among them, AML is one of the common malignant tumors of the hematopoietic system; AML has strong heterogeneity, with limited current clinical treatment options and poor prognosis. Primary drug resistance is common. Existing adoptive cellular immunotherapies, such as CAR-T therapy, have demonstrated significant anti-tumor activity in B-cell malignancies and multiple myeloma. However, due to the heterogeneity of AML cells, their similarity to normal hematopoietic cells, and the lack of leukemia cell-specific targets, the efficacy of CAR-T therapy for AML is very limited. CMML is a type of chronic myeloid leukemia that exhibits dual characteristics of myelodysplastic syndromes/myeloproliferative neoplasms (MDS/MPN); CMML is the most aggressive form of chronic leukemia, with a short median overall survival and a high risk of transformation into acute leukemia. Currently, there are no highly effective treatments available.

The IND approval of YTS104 cell injection not only marks a significant milestone for Huaxia Yingtai(Beijing)Biotechnology Co., Ltd., but also represents a differentiated exploration of new targets in cell therapy in China, offering a new treatment option for many patients with advanced AML and CMML in China and globally. As research progresses, Huaxia Yingtai will further promote the application of the STAR-T technology platform to treat more indications, addressing unmet clinical needs.