Heyjulucel (CNCT19) Achieves Primary Endpoint in Pivotal Clinical Trial for Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia

On October 11, 2022, Hesyuan Biotech (Tianjin) Co., Ltd. (hereinafter referred to as “Hesyuan Biotech”) and the Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences (Institute of Hematology, Chinese Academy of Medical Sciences) jointly announced that Inaticabtagene Autoleucel Injection (proposed name) (CNCT19 Cell Injection, Inaticabtagene Autoleucel), developed by Hesyuan Biotech, had met its primary endpoint in a pivotal clinical study for the treatment of adult relapsed or refractory acute B-lymphoblastic leukemia (r/r B-ALL). This single-arm, open-label, multicenter pivotal clinical study was conducted across 10 clinical centers in China, with Professor Wang Jianxiang from the Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences (Institute of Hematology, Chinese Academy of Medical Sciences) serving as the principal investigator. The study aimed to evaluate the efficacy and safety of Inaticabtagene Autoleucel Injection, an independently innovated CD19-targeted CAR-T therapy, in treating adult patients with r/r B-ALL. The primary efficacy endpoint was the overall response rate (ORR) at three months, as assessed by an Independent Review Committee (IRC). The results demonstrated that Inaticabtagene Autoleucel Injection exhibited excellent clinical efficacy and a favorable safety profile in adult patients with r/r B-ALL. Detailed study results will be published at international academic conferences or in peer-reviewed journals.

Acute B-cell lymphoblastic leukemia (B-ALL) is one of the common hematologic malignancies, with adult patients having worse overall survival compared to pediatric patients. The relapse rate after initial treatment for adult B-ALL is high, with approximately 60% of patients eventually progressing to relapsed/refractory (r/r) B-ALL. Patients with r/r B-ALL have an extremely poor prognosis; there is a lack of effective clinical treatments, and their survival period is only 2–6 months. There has been no significant improvement in survival among Chinese patients over the past three decades. Adult r/r B-ALL poses a severe life-threatening risk and represents a substantial unmet clinical need, urgently requiring new effective therapies to extend patient survival and improve quality of life. Currently, no CAR-T products for the treatment of adult r/r B-ALL have been approved for marketing in China.

Hejilunsai Injection is a CD19-targeted CAR-T cell therapy product with independent intellectual property rights owned by Heyuan Biologics. It features a globally unique CD19 scFv (HI19a) structure and internationally leading manufacturing processes, and has been granted multiple national invention patents. Due to its excellent clinical efficacy, Hejilunsai Injection has received the “Breakthrough Therapy Drug” designation from the Center for Drug Evaluation of China’s National Medical Products Administration and Orphan Drug Designation (ODD) from the U.S. FDA. This product is the first CAR-T therapy in the field of leukemia in China to achieve clinical study endpoints, and is poised to become the first fully independently developed domestic CAR-T product targeting CD19.

Dr. Chen Changting, Chief Medical Officer (CMO) of Heyuan Biologics (Changting C. Haudenschild, M.D., stated: “The pivotal clinical study of Hejilunsai Injection for the treatment of highly aggressive adult relapsed/refractory B-cell acute lymphoblastic leukemia (r/r B-ALL) has met its primary efficacy endpoint. The results demonstrate that CNCT19 exhibits significant clinical efficacy in adult r/r B-ALL, with robust effectiveness across studies and durable disease remission. We look forward to the imminent market launch of Hejilunsai Injection to provide an effective therapeutic option for adult r/r B-ALL. Meanwhile, registrational clinical studies of Hejilunsai Injection for pediatric and adolescent r/r B-ALL and relapsed/refractory non-Hodgkin lymphoma (r/r NHL) are ongoing, with the expectation that it will benefit a broader patient population.”

Dr. Wang Yongzeng, Chief Technology Officer (CTO) of Heyuan BiologyIt stated: “Hejilunsai Cell Injection features a fully independently developed advanced manufacturing process, with key parameters reaching internationally leading levels. In the registrational clinical studies for the treatment of relapsed/refractory B-cell acute lymphoblastic leukemia (r/r B-ALL), the manufacturing success rate of the cell therapy product reached 100%. We will further enhance our end-to-end medical quality control system and scalable production and supply infrastructure to safeguard the clinical application of Hejilunsai Cell Injection.”

Dr. Li Zhou, Chief Scientific Officer (CSO) of Heyuan BiologicsHechuan Bio stated: “With the rapid advancement of the development of Heqilunse injection, Hechuan Bio has established an efficient technical platform and R&D system. We will deepen and focus on promoting technological innovation, continuously delivering differentiated autologous and universal pipeline products with international competitiveness covering disease areas such as hematologic malignancies and solid tumors, enabling more patients to benefit from innovative cell therapies.”

Dr. Lu Lulu, CEO of Heyuan Biologics, stated: “Hejilunsel Cell Injection is the company’s first core product, and we will formally submit a New Drug Application to the Center for Drug Evaluation of the National Medical Products Administration in the near future. We believe that the launch of this product in China will transform the current landscape where domestic CAR-T therapies rely on imported technologies and products, addressing the challenges of accessibility and affordability for Chinese patients. Furthermore, it will help propel Chinese CAR-T therapies into the global competitive arena and strengthen the ‘Chinese force’ in the innovation and development of novel cell-based therapies.”

Professor Wang Jianxiang, Deputy Director of the Institute of Hematology & Blood Diseases Hospital (Institute of Hematology, Chinese Academy of Medical Sciences) and Director of the National Clinical Research Center for Blood System Diseasesstated: “There is a significant unmet clinical need in adult relapsed or refractory acute lymphoblastic leukemia (r/r ALL). Hejiluncai Injection has demonstrated substantial clinical value in the ongoing multicenter registrational clinical trials in China, holding the promise to transform the current treatment landscape for Chinese adult patients with r/r B-cell precursor ALL (r/r B-ALL) and offer them hope for long-term survival. We also extend our gratitude to the Hejiluncai Cellular Injection R&D team, the clinical investigator team, and especially the trial participants for their dedicated efforts!”

Healios Biopharma was founded in June 2018. As a next-generation biopharmaceutical company driven by innovative cell and gene technologies, it collaborates extensively with top-tier national research institutes and clinical research centers. The company has established an international new drug R&D innovation system centered on its CAR technology platform, iPSC technology platform, and gene editing technology platform. Focused on the R&D and commercialization of innovative therapies such as immune cell treatments, Healios Biopharma is committed to building an industry-leading platform for the development, clinical translation, and commercialization of cell therapy drugs.

The company’s first core product, Hejilunsai Injection (proposed name) (CNCT19 Cell Injection, Inaticabtagene Autoleucel), is a CAR-T cell therapy product targeting CD19 with independent intellectual property rights. On November 29, 2019, it received two new drug clinical trial approvals from the National Medical Products Administration (NMPA): one for the treatment of relapsed or refractory acute lymphoblastic leukemia (Acceptance No.: CXSL1800106) and the other for the treatment of relapsed or refractory aggressive B-cell non-Hodgkin lymphoma (Acceptance No.: CXSL1800107). Both clinical trials were among the first to enter the Phase II registration stage. The product has been granted “Breakthrough Therapy Designation” by the Center for Drug Evaluation (CDE) under the NMPA and Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA), positioning it to potentially become the first marketed CD19-targeting CAR-T product with independent intellectual property rights. The third expanded indication for Hejilunsai Injection received implicit approval for clinical trials from the CDE under the NMPA on August 19, 2022, for the treatment of pediatric and adolescent patients (aged 3–18 years) with relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL).

Guided by a commitment to meeting clinical needs, the Company has established rigorous production and quality management systems for cell therapy products to deliver safe, efficacious, and accessible immune cell therapies to patients. It continues to build a highly scalable and internationally competitive innovative pipeline covering hematologic malignancies, solid tumors, and non-oncologic conditions such as autoimmune diseases. Furthermore, the Company boasts world-class R&D technology platforms, process development capabilities, quality control systems, and commercial-scale manufacturing facilities. The Company holds multiple invention patents and has been selected for the National Key R&D Program of the Ministry of Science and Technology of China, specifically the “Science and Technology Supporting Economy 2020” Key Special Project.