Investigational New Drug Application Approved for Inaticabtagene Autoleucel Treating Autoimmune Diseases

On October 11, 2024, Juventas Cell Therapy Ltd. announces China's National Medical Product Administration (NMPA) has approved its Investigational New Drug （IND）application for Inaticabtagene Autoleucel (CNCT19 Injection), a CD19-targeted autologous chimeric antigen receptor (CAR)-T cell therapy, to treat  Immune thrombocytopenia of systemic lupus erythematosus (SLE - ITP) . This is the first IND of Inaticabtagene Autoleucel approved for autoimmune disease in China.

SLE is a systemic autoimmune disease, usually leading to multiple organ damage. Abnormal B cells and autoantibody play a critical role in SLE pathogenesis. SLE could cause excessive platelet destruction, namely SLE-ITP. SLE-ITP occurs in approximately 16% of SLE Chinese patients, most of the SLE-ITP patients respond very poorly to steroids, immunoinhibitors, and biological drugs, and often suffer major organ bleeding risk，while their long-term survival is rather poor.

In July of this year, the prestigious *The New England Journal of Medicine* (NEJM, IF: 96.2) published a study "Anti-CD19 CAR T cells in refractory immune thrombocytopenia of SLE," co-authored by Professor Zeng Xiaofeng and Professor Li Mengtao from the Rheumatology Department and Professor Zhou Daobin from the Hematology Department of Peking Union Medical College Hospital. The study showcased the long-lasting efficacy and safety of Inaticabtagene Autoleucel in treating SLE-ITP, highlighting the huge potential of CAR-T cell therapy.

Inaticabtagene Autoleucel is a CD19 CAR-T cell therapy product, featured with a unique CD19 scFv（HI19a）and advanced manufacturing processes. Inaticabtagene Autoleucel is the first proprietary CD19-targeted CAR-T product homegrown in China, and the first commercialized CAR-T cell therapy product for B-ALL in China . Inaticabtagene Autoleucel is expanding its application for several indications, including adult r/r B-ALL, r/r DLBCL, pediatric and adolescent r/r B-ALL, and various autoimmune diseases. Its New Drug Application (NDA) of treating Relapsed or Refractory Large B-Cell Lymphoma has been accepted by NMPA in September 2024.

As the first CAR-T product for adult r/r B-ALL treatment in China, Inaticabtagene Autoleucel is recommended as priority treatment option by multiple clinical guidelines, including those from the Chinese Society of Clinical Oncology (CSCO) and the Chinese Medical Association. So far, Inaticabtagene Autoleucel has been adopted by over 80 leading hematology treatment centers across China, with real-world data validating its efficacy and safety, gaining endorsement from physicians and patients alike.

Professor Zeng Xiaofeng from the Rheumatology Department of Peking Union Medical College Hospital commented:

" SLE is an autoimmune disease caused by genetic, environmental, and hormonal factors, resulting in high heterogeneity and requiring individualized treatment. The harsh reality is that more than half of Chinese patients who develop SLE around age 30 die by age 55–60. We are eagerly looking forward to the commencement of the clinical trials to benefit more patients in struggle."

Professor Li Mengtao from the Rheumatology Department of Peking Union Medical College Hospital stated:

"Following the world first report in *The New England Journal of Medicine* on the use of CD19 CAR-T cell therapy treating SLE-ITP, we are very pleased to see the approval of this IND application. Our multidisciplinary team at Peking Union Medical College Hospital will continue to explore the application of CAR-T cell therapy in SLE, advancing the treatment of autoimmune diseases and benefiting more patients."

Professor Jianxiang Wang, chief clinical expert at the Institute of Hematology and Blood Diseases Hospital (CAMS) and director of the National Clinical Research Center for Hematologic Diseases, expressed his congratulations on the approval of this application. He highlighted the significant clinical benefits demonstrated by Inaticabtagene Autoleucel in both clinical trials and real-world practice for blood cancer, he is looking forward to the success of Inaticabtagene Autoleucel’s clinical trial in treating autoimmune diseases.

Dr. Lu Lulu, CEO of Juventas said,

"We are extremely excited about the approval of the IND application for Inaticabtagene Autoleucel in the treatment of refractory SLE-ITP. This marks a new milestone in expanding Inaticabtagene Autoleucel application into the field of autoimmune diseases, following its application in adult relapsed or refractory B-cell acute lymphoblastic leukemia, relapsed or refractory large B-cell lymphoma, and pediatric and adolescent relapsed or refractory B-cell acute lymphoblastic leukemia. Inaticabtagene Autoleucel is also undergoing clinical trail and studies for various other autoimmune diseases, including lupus nephritis and autoimmune hemolytic anemia. We will push forward the clinical research and application to benefit more patients with this innovative therapy."

Name: Lu ZHOU
Email: zhoulu@juventas.cn
Organization: Juventas Cell Therapy
Website: www.juventas.cn/en.php