HeyGene Bio's CNCT19 Cell Injection Receives Breakthrough Therapy Designation from China NMPA for Relapsed/Refractory Acute Lymphoblastic Leukemia

China Finance Online, December 23 (Reporter Du Ding): Today, the website of the Center for Drug Evaluation (CDE) under the National Medical Products Administration showed that Juventas Biotechnology (Tianjin) Co., Ltd. (“Juventas”)’s CNCT19 Cell Injection (anti-CD19 chimeric antigen receptor T-cell injection) has been approved for inclusion in the “Breakthrough Therapy” program (CDE Acceptance Number: CXSL1800106).

　　Indications: Relapsed or Refractory Acute Lymphoblastic Leukemia

The indication included in the “Breakthrough Therapy” designation is relapsed or refractory acute lymphoblastic leukemia. Currently, there are no CAR-T products approved globally for the indication of adult relapsed or refractory acute lymphoblastic leukemia, and there is an urgent unmet clinical need.

CAR-T therapy is a treatment that uses genetic engineering technology to "super-arm" T cells, thereby killing tumor cells. It is characterized by high remission rates and long-term survival rates in the treatment of relapsed or refractory acute lymphoblastic leukemia.

Preliminary clinical study data indicate that CNCT19 Cell Injection demonstrates superior efficacy and safety in the treatment of adult relapsed or refractory acute lymphoblastic leukemia, with a complete remission rate exceeding 90% and the longest survival duration surpassing three years. It is poised to become one of the most promising clinical therapeutic agents for adult relapsed or refractory acute lymphoblastic leukemia.

It is understood that CNCT19 Cell Injection is a CD19-targeted CAR-T cell therapy product with independent intellectual property rights owned by Juventas, derived from the long-term accumulation of technological innovations at the Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences (Institute of Hematology, Chinese Academy of Medical Sciences). On November 29, 2019, CNCT19 Cell Injection received approval from the National Medical Products Administration for two new drug clinical trials: one for the treatment of relapsed or refractory acute lymphoblastic leukemia (Application No.: CXSL1800106) and the other for the treatment of relapsed or refractory aggressive B-cell non-Hodgkin lymphoma (Application No.: CXSL1800107). Currently, both clinical trials have advanced into the registrational Phase II stage, placing them at the forefront of clinical research progress. Juventas is accelerating the advancement of these clinical studies.

　　Experts State That the Process of Clinical Research Will Be Accelerated

“Breakthrough Therapy Drugs” represent a “green channel” established by the National Medical Products Administration (NMPA) at the review and approval stage for innovative drugs. Through policy optimization, this initiative encourages new drug development to address challenges in treating critical diseases. On July 8, 2020, the NMPA issued the “Procedures for the Review of Breakthrough Therapy Drugs (Trial).” For drugs under research and development that demonstrate significant clinical advantages, applicants may assess the scope of application and eligibility criteria during clinical trials and submit an application to the Center for Drug Evaluation (CDE) of the NMPA. The scope of breakthrough therapy drugs includes: (1) drugs intended to treat diseases that pose a serious threat to life; and (2) innovative drugs for which sufficient evidence demonstrates significant clinical advantages compared to existing treatment options.

Professor Wang Jianxiang, Director of the National Clinical Research Center for Hematologic Diseases and Deputy Director of the Institute of Hematology & Blood Diseases Hospital (Institute of Hematology, Chinese Academy of Medical Sciences), stated that adult patients with relapsed or refractory acute lymphoblastic leukemia have an extremely poor prognosis, with existing treatment modalities yielding minimal efficacy and a 5-year overall survival rate of less than 10%, highlighting an urgent unmet clinical need. The CNCT19 Cell Injection brings breakthrough therapeutic options and hope for cure to this patient population. “Currently, no CAR-T products have been formally approved for clinical application worldwide for adult relapsed or refractory acute lymphoblastic leukemia. The inclusion of CNCT19 Cell Injection in the Breakthrough Therapy Designation by the Center for Drug Evaluation of the National Medical Products Administration fully reflects the urgency of this clinical need. We will accelerate the clinical research process to ensure patients benefit from this breakthrough therapy as soon as possible.”

According to Dr. He Ruyi, Chief Scientist of SDIC Merchants Medicine and Health, the National Medical Products Administration’s Breakthrough Therapy Drug Work Program reflects the regulatory authorities’ purpose and principles of encouraging innovation and addressing urgent clinical needs. The inclusion of CNCT19 Cell Injection as a breakthrough therapy drug will facilitate its research and development process under enhanced regulatory guidance. “We look forward to the early completion of clinical studies and the subsequent clinical application of CNCT19 Cell Injection, so that this innovative medicine can benefit patients.”

“The inclusion of CNCT19 Cell Injection in the Breakthrough Therapy Designation program by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) marks another significant milestone in the overall development of CNCT19. The indication covered by this designation currently has no approved comparable products available globally, and CNCT19 Cell Injection is specifically designed to address this urgent clinical need,” stated Dr. Lv Lulu, CEO of Juventas. “Driven by the supportive regulatory policy of Breakthrough Therapy Designation, we will accelerate the process of registrational clinical studies to achieve market approval as soon as possible, thereby meeting the urgent needs of patients in China and worldwide.”