Heyuan Bio Announces China's First Homegrown CD19 CAR-T Therapy Yuanruida® (Inaticabtagene Autoleucel Injection) Approved for Relapsed/Refractory B-ALL
* Nakiolencel Injection is the first CD19 CAR-T cell therapy product with full independent intellectual property rights in China, and also the first CAR-T cell therapy product for leukemia treatment in China.
* Pivotal clinical study data for nakiolecabtagene autoleucel injection in the treatment of adult relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL) demonstrate durable high response rates and a significant reduction in the severity of CAR-T therapy-related toxicities, representing a major breakthrough in the treatment of adult r/r B-ALL.
* With its superior efficacy and safety profile, Nakiolencel Injection has been designated as a “Breakthrough Therapy” by the National Medical Products Administration (NMPA) and approved for market launch through the “Priority Review” pathway. The approval of Nakiolencel Injection provides adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL) with a breakthrough clinical treatment option in terms of efficacy, safety, and convenience of administration.
On November 8, 2023, Healios Biopharmaceuticals (Tianjin) Co., Ltd. (“Healios Biopharma”), a biopharmaceutical company dedicated to becoming a global leader driven by cutting-edge innovative technologies in cell and gene therapy, announced thatThe National Medical Products Administration (NMPA) has officially approved the Company’s immune cell CAR-T therapy product, Yuanruida.®(New Drug Application (NDA) for Inaticabtagene Autoleucel Injection (CNCT19 Cell Injection) for the treatment of adult relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL).)The successful approval and market launch of nakiolemab injection will bring breakthrough clinical treatment options for adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL) in terms of efficacy, safety, and treatment convenience. November 8 has also been designated by Heyuan Biopharma as its annual “Acute Lymphoblastic Leukemia Day.”
(NMPA Website News) Yuanruida®(Nakioluncept Injection) isSelf-developed CD19-targeted CAR-T cell therapy product,Featuring a globally unique CD19 scFv (HI19a) structure and internationally leading manufacturing processes, it achieved a 100% production success rate in registrational clinical studies for adult relapsed/refractory B-cell acute lymphoblastic leukemia (r/r B-ALL). Leukemia is one of the most common hematologic malignancies. Adult B-ALL has a high relapse rate after initial treatment, with approximately 60% of patients progressing to the relapsed or refractory stage (r/r B-ALL). Patients with adult r/r B-ALL have an extremely poor prognosis, posing a severe threat to life. Due to the lack of effective therapeutic options, their median survival is only 2–6 months. There has been no significant improvement in survival outcomes over the past three decades, indicating a substantial unmet clinical need. There is an urgent demand for novel, effective treatments to extend patient survival and improve quality of life.
The approval for the market launch of this new drug is based on the results of a single-arm, open-label, multicenter pivotal clinical study (NCT04684147) evaluating Nakiolencel Injection for the treatment of adult relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL). The 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 and Peking Union Medical College) serving as the principal investigator. At the 64th American Society of Hematology (ASH) Annual Meeting held in December 2022, Heyuan Biopharma presented these pivotal clinical study data to the global community via an oral presentation (Oral Presentation, #0660).These pivotal clinical study data demonstrate durable high response rates and a significant reduction in the severity of CAR-T therapy-related toxicities:
1. Sustained High Remission Rates: The overall response rate (ORR) was 82.1%. At a median follow-up of 9.3 months, the median duration of response (DOR) had not been reached; among patients still in response at 3 months, an estimated 80% were expected to maintain their response at 1 year. Furthermore, patients demonstrated sustained responses and long-term survival benefits regardless of whether they subsequently underwent hematopoietic stem cell transplantation.
2. Significantly Reduced Severity of CAR-T Therapy-Related Toxicity: The incidence of grade ≥3 cytokine release syndrome (CRS) was 10.3%; the incidence of grade ≥3 immune effector cell-associated neurotoxicity syndrome (ICANS) was 7.7%, with no adverse events attributable to off-target effects of CD19-directed CAR-T therapy observed.
3. The durable high response rate and safety profile are significantly superior to those of existing treatments,Represents a major breakthrough in the treatment of adult r/r B-ALL.
Since its establishment in 2018, Heyuan Biopharma has been guided by clinical needs and national policies, leveraging the momentum of the “14th Five-Year Plan” for Bioeconomic Development to advance key areas in immune cell therapy. Adhering to independent innovation, the company has strengthened its core capabilities in the drug development pathway for immune cell therapies and continuously delivers innovative products driven by cutting-edge cell and gene technologies to benefit patients. Heyuan Biopharma was selected for the National Key R&D Program of the Ministry of Science and Technology, specifically the “Science and Technology Supporting Economy 2020 Key Special Project,” and in June 2021, it obtained the first Drug Manufacturing License for cell therapy products in the Beijing-Tianjin-Hebei region.”
Professor Wang Jianxiang
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 Hematologic Diseases, stated:
“Under conventional treatment modalities, adult patients with relapsed or refractory acute B-lymphoblastic leukemia face a threat of death within months, representing a significant unmet clinical need in China and globally. Yuanruida”®As the first CAR-T cell therapy product for leukemia treatment in China, and also the first independently developed Chinese CAR-T product targeting CD19, it demonstrates significant clinical value and outstanding efficacy, bringing deep and durable remission to patients, with the potential to reshape the current treatment landscape for adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia (r/r B-ALL) in China. Yuanruida®"The approval provides clinicians with a breakthrough treatment option, offering hope for long-term survival in patients with relapsed or refractory B-cell acute lymphoblastic leukemia."
Dr. Lu Lulu
Dr. Lu Lulu, CEO of Heyuan Biologics, stated:
“It is highly inspiring to welcome Yuanruida, the first CAR-T product targeting CD19 independently developed in China.”®The successful approval and market launch mark a significant milestone for HeYuan Biopharma and a source of pride for the entire HeYuan team. Upholding our steadfast commitment to “clinical value orientation and patient-centricity,” we have jointly achieved this milestone with the care and support from all sectors of society. In YuanRuiDa®In multi-center registrational clinical trials, we have observed a growing number of patients achieving sustained remission and long-term survival following a single infusion of CAR-T cells. Their quality of life continues to improve, with most returning to normal daily activities. This represents a significant breakthrough in the field of leukemia treatment in China. We will remain true to our original mission, continue to collaborate with all stakeholders, and constantly enhance Yuanruida®accessibility, establishing a patient-centric, personalized medical and quality control system for the entire cell therapy process, enabling more patients to benefit from Naqiolorsen treatment. Unlocking source power, igniting hope!”
Healios Biopharma was founded in June 2018 and has grown into a leader in independent innovation within China’s cell therapy industry, committed to becoming a globally leading next-generation biopharmaceutical enterprise driven by innovative cell and gene technologies. The company’s first core product, Yuanruida®(Nakiolunse Injection, CNCT19) was officially approved for marketing by the National Medical Products Administration in November 2023. This product is the first CAR-T therapy in China for the treatment of leukemia and the first fully independently developed CD19 CAR-T drug in China. The company has established deep collaborations with leading national research institutions to build an international new drug R&D system centered on core platforms including CAR technology, induced pluripotent stem cells (iPSCs), and gene editing. It currently has more than 10 pipeline products under development, covering disease areas such as hematologic malignancies, solid tumors, and autoimmune diseases.
Yuanruida®(Naticabtagene Autoleucel Injection, CNCT19) is a CD19-targeted CAR-T cell therapy product with independent intellectual property rights. It features a globally unique CD19 scFv (HI19a) structure and internationally leading manufacturing processes. The product has successively obtained three Investigational New Drug (IND) approvals from the National Medical Products Administration (NMPA) for the treatment of adult relapsed or refractory acute lymphoblastic leukemia, relapsed or refractory aggressive B-cell non-Hodgkin lymphoma, and relapsed or refractory B-cell acute lymphoblastic leukemia in children and adolescents. It has been designated as a “Breakthrough Therapy” by the Center for Drug Evaluation of the NMPA and has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA). In December 2022, the NMPA formally accepted the New Drug Application (NDA) for naticabtagene autoleucel injection for the treatment of adult r/r B-ALL and included it in the priority review program. In March 2023, the IND application for naticabtagene autoleucel injection for the treatment of adult r/r B-ALL was also approved by the U.S. FDA. In November 2023, Yuanruida®(Nakio-lunse Injection) has been officially approved for market launch by the National Medical Products Administration (NMPA) of China.
Guided by a commitment to meeting clinical needs, the Company has established rigorous production and quality management systems for cell therapy products to provide patients with safe, effective, and accessible immune cell therapies. The Company boasts world-class R&D technology platforms, process development capabilities, quality control systems, and commercial-scale manufacturing facilities. In June 2021, it obtained Tianjin’s first Drug Manufacturing License for cell-based pharmaceuticals. The Company holds multiple invention patents and was 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.”