Heyuan Biosciences' Inaticabtagene Autoleucel Granted Breakthrough Therapy Designation for Pediatric Relapsed/Refractory B-cell Acute Lymphoblastic Leukemia

Acute lymphoblastic leukemia (ALL) is the most common hematologic malignancy in children. Although combination chemotherapy and precise stratified treatment have significantly improved overall survival rates, 20%-25% of pediatric ALL cases remain refractory or relapsed. The effective rate of salvage therapy for primary refractory patients is only 31%, with a median overall survival (OS) of less than one year. As the number of treatment lines increases, the prognosis for pediatric ALL worsens and the cure rate declines. Relapsed B-ALL patients exhibit strong chemoresistance, with a chemotherapy efficacy rate of approximately 25% and a median survival of 3.7 months, while also facing increased risks of treatment-related toxicity. Ultimately, children die from disease relapse or treatment-related adverse effects. Currently, relapsed or refractory B-ALL remains a serious threat to the lives of pediatric B-ALL patients in China, and there is still no unified standard treatment regimen. Clinically, there is an urgent need for more effective and safer therapies to improve patient outcomes. With its unique CD19 scFv, CAR structure, advanced manufacturing process, and quality system, the investigational drug Naciorlen Cell Injection has demonstrated remarkable clinical efficacy and safety in early-phase clinical studies and registrational clinical trials. It has received Breakthrough Therapy Designation from the Center for Drug Evaluation (CDE), which will further expedite its approval process for use in pediatric acute lymphoblastic leukemia.

As China’s first CD19 CAR-T product with full independent intellectual property rights, the injection of Naciaurelase has been developed for multiple indications, including adult and pediatric relapsed or refractory B-cell acute lymphoblastic leukemia, lymphoma, and various types of autoimmune diseases. Its adult indication was approved for marketing in November 2023, making it the first CAR-T product in China approved for leukemia treatment. The inclusion of the pediatric relapsed or refractory B-cell acute lymphoblastic leukemia indication into the breakthrough therapy designation will further promote the recognition of this therapy in the field of pediatric leukemia, offering more precise and efficient treatment options for young patients.

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

Congratulations to Nakio Cell Injection for obtaining the Breakthrough Therapy Designation from the Center for Drug Evaluation of the National Medical Products Administration for the treatment of children with relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL). This fully recognizes the clinical value demonstrated by the product in pediatric ALL patients. It also highlights the clinical significance of China-developed CAR-T drugs across various patient populations, including adults with acute lymphoblastic leukemia, children with acute lymphoblastic leukemia, non-Hodgkin lymphoma, and autoimmune diseases. These advancements are expected to improve treatment outcomes, enhance quality of life, and help patients return to normal lives.

Professor Zhu Xiaofan, Clinical Chief Expert of the Pediatric Hematology and Oncology Treatment Center at the Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences, stated:

Acute Lymphoblastic Leukemia (ALL) is the most common hematologic malignancy in children. Despite a relatively high overall cure rate, children with relapsed or refractory ALL still face an extremely high risk of mortality. Conventional chemotherapy often proves ineffective at this stage, offering poor outcomes and significant toxicity, placing enormous physical, emotional, and financial burdens on families. Naciorl Injection, a CAR-T product independently developed in China, has demonstrated high remission rates and durable efficacy in clinical studies. Its recent designation as a breakthrough therapy is a long-awaited milestone in the field of pediatric leukemia treatment in China!

Dr. Lulu Lv, CEO of Juventas, stated:

Juventas' Naciorl Injection has been included in the breakthrough therapy drug program by the Center for Drug Evaluation of the National Medical Products Administration, marking a significant advancement in the field of pediatric leukemia treatment in China. This therapy is expected to reshape the treatment approach for pediatric acute lymphoblastic leukemia (ALL), offering a healthier future for more children with leukemia. We are about to submit a new drug marketing application for Naciorl Injection to treat pediatric acute lymphoblastic leukemia to the National Medical Products Administration. This will become the third approved indication for Naciorl Injection after treating adult acute lymphoblastic leukemia and non-Hodgkin lymphoma. The company will continue to expand the therapeutic potential of China’s domestically developed CAR-T product, Naciorl Injection, for more hematological malignancies and autoimmune diseases, allowing more patients to benefit from this innovative therapy.