Heyuan Bio's CAR-T Therapy Yuanruida® (Najiorlun赛 Injection) Granted IND Approval for Autoimmune Indication in Refractory SLE-Related Immune Thrombocytopenia

Oct 12, 2024 10:12 CST Updated 10:12

Juventas

Innovative Cell Therapy Drug Developer

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October 11, 2024Information disclosed on the website of the Center for Drug Evaluation (CDE) under the National Medical Products Administration (NMPA) shows that Juventas' first original CAR-T cell therapy product, Yuan Ruida.^®(Juventas' Naciorl Injection) Receives Implied Permission for New IND Application in Autoimmune Disease Field（Application Number:CXSL2400450）, for the treatment of refractory systemic lupus erythematosus-associated immune thrombocytopenia (SLE-ITP),This is the first Investigational New Drug (IND) approval for Nakiolumab Injection in the field of autoimmune disease treatment.

Systemic Lupus Erythematosus (SLE) is a chronic systemic autoimmune disease that often causes multi-organ damage throughout the body. Abnormal B-cell activity and the production of autoantibodies play a crucial role in its pathogenesis. SLE can lead to immune thrombocytopenia, which occurs in approximately 16% of Chinese SLE patients. A significant proportion of these patients show poor responses to glucocorticoids, immunosuppressants, and biologics, resulting in poor prognosis and a long-term risk of major organ bleeding, posing a major challenge in SLE treatment.

In July this year, the international top journal "New England Journal of Medicine" (NEJM, IF: 96.2) published online a research paper by Professor Zeng Xiaofeng and Professor Li Mengtao from the Department of Rheumatology and Immunology, Peking Union Medical College Hospital, and Professor Zhou Daobin from the Department of Hematology. The paper, titled “Anti-CD19 CAR T cells in refractory immune thrombocytopenia of SLE”, reports on the study of an autologous T-cell injection targeting CD19 chimeric antigen receptor (CD19 CAR-T), known as Naciauclecel Injection, for the treatment of systemic lupus erythematosus-associated immune thrombocytopenia (SLE-ITP). The report demonstrates the favorable and lasting efficacy and safety of Naciauclecel Injection in treating SLE-associated ITP, highlighting the potential application of CAR-T in the treatment of SLE and ITP.

Yuan Ruida^®(Natcarlumab Injection) is a CD19-targeted CAR-T cell therapy product independently developed by Juventas, derived from the long-term technological innovation accumulation of the Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences. It features a globally unique CD19 scFv (HI19a) structure and internationally leading production and manufacturing processes. Yuanruida^®(Natrunaualen Injection) is the first CD19 CAR-T cell therapy product with full independent intellectual property rights in China. It has been developed for multiple indications, including adult relapsed or refractory B-cell acute lymphoblastic leukemia, relapsed or refractory large B-cell lymphoma, pediatric relapsed or refractory B-cell acute lymphoblastic leukemia, and various types of autoimmune diseases.The new drug application for the treatment of adult relapsed or refractory B-cell acute lymphoblastic leukemia was officially approved by the National Medical Products Administration (NMPA) in November 2023, and the new drug application for the treatment of relapsed or refractory large B-cell lymphoma was officially accepted by the NMPA in September 2024.

As the first CAR-T cell therapy product in China's leukemia treatment field, Yuan Ruida^®Post-marketing, it has been recommended by authoritative treatment guidelines such as CSCO (Chinese Society of Clinical Oncology) and the Chinese Medical Association; it has been used in 80 top hematology treatment hospitals in China, with its real-world efficacy and safety highly recognized by doctors and patients.

Professor Zeng Xiaofeng, Department of Rheumatology and Immunology, Peking Union Medical College Hospital:

"Systemic lupus erythematosus (SLE) is an autoimmune disease caused by a combination of genetic, environmental, and hormonal factors, leading to strong heterogeneity in the condition and high demand for personalized diagnosis and treatment. The harsh reality is that in China, patients with an onset age of around 30 years old have a mortality rate exceeding 50% by the age of 55 to 60. We are highly anticipating the rapid advancement of the clinical trial for the new drug, Naciorlenzep, in treating refractory systemic lupus erythematosus-associated thrombocytopenia, to benefit the vast number of patients severely affected by the disease."

Professor Mengtao Li, Department of Rheumatology and Immunology, Peking Union Medical College Hospital:

"In the exploratory clinical study we conducted on the use of Naciorlen Injection for the treatment of refractory systemic lupus erythematosus-associated immune thrombocytopenia, multiple patients have been successfully treated, with the first patient achieving sustained remission for over a year. We presented this groundbreaking global application of CD19 CAR-T cell therapy in treating systemic lupus erythematosus-associated immune thrombocytopenia in The New England Journal of Medicine. We are thrilled to see the approval of the clinical trial for Naciorlen as a new drug for treating refractory systemic lupus erythematosus-associated thrombocytopenia. The multidisciplinary team at Xiehe will continue to explore and deepen the application of CAR-T cell therapy in this disease, advancing progress in the field of autoimmune disease treatment to benefit more patients."

Professor Wang Jianxiang, Chief Clinical Expert of the Blood Diseases Hospital of the Chinese Academy of Medical Sciences (Institute of Hematology of the Chinese Academy of Medical Sciences) and Director of the National Clinical Research Center for Hematological Diseases：

"Sincere congratulations on the treatment of CD19 CAR-T drug, Najiolumab Injection, independently developed in China"Clinical Trial of New Drug for Refractory Systemic Lupus Erythematosus with Thrombocytopenia Approved! The injection of Naciorlens has shown good clinical effects in the field of hematological tumor treatment, winning wide recognition. It has also achieved positive clinical progress in exploratory clinical studies related to autoimmune diseases. We look forward to the expansion and approval of more indications for Naciorlens, benefiting more patients."

Source: Juventas