Idecabtagene Autoleucel (CT103A/IBI326) NDA Accepted by China's NMPA for Relapsed/Refractory Multiple Myeloma

Nanjing, Shanghai, and San Jose, CaliforniaJune 2, 2022PR Newswire -- IASO Bio, an innovative biopharmaceutical company at the clinical stage dedicated to the development and industrialization of cell therapy and antibody drugs, today announced together with Innovent Bio (stock code: 01801 on the Hong Kong Stock Exchange), a biopharmaceutical company committed to the research, production, and sales of innovative drugs in major disease areas such as oncology, autoimmune diseases, metabolism, and ophthalmology,National Medical Products Administration (NMPA)NMPA) officially accepted the Ixcellen injection (IASO Bio's R&D code:CT103A; Innovent Bio R&D Code:IBI326`) Treatment Recurrence`/Refractory Multiple Myeloma (R/R MM) Marketing Authorization Application (NDA）。

Equecabtagene Autoleucel Injection is the first fully self-developed product in China.CAR-TCell Therapy Products, jointly developed by IASO Bio and Innovent Bio.This is the first domestic company to submit a new drug for marketing approval and is expected to become the first approved targeted drug in China.BCMAAutologous Chimeric Antigen ReceptorTCellular Immunotherapy Products。The product was granted the "Breakthrough Therapy Designation (BTD)" by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) in February 2021.

The submission of this NDA is based on the results of a phase 1/2 registrational clinical trial (NCT05066646) of Ixalotenib injection: Ixalotenib injection has demonstrated excellent safety and efficacy in humans. The fully human BCMA antibody sequence ensures minimal immunogenicity, with long-lasting CAR-T expansion and persistence in the body, offering the potential to become a breakthrough treatment for patients with relapsed or refractory multiple myeloma. IASO Bio and Innovent Bio presented the clinical study results at the 63rd American Society of Hematology (ASH) Annual Meeting in 2021 in an oral presentation format (Abstract #547). Updated phase 1/2 clinical research data will be presented in an oral report format (Abstract #S187) during the virtual meeting of the 2022 European Hematology Association (EHA).

The Leading Unit of This Study Professor Qiu Lugui from the Blood Disease Hospital of the Chinese Academy of Medical Sciences and Professor Li Chunrui from Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology both stated:

"Multiple Myeloma (MM) is the second most common malignant tumor in the hematologic system. Although the survival period of MM patients has been continuously extended with the widespread application of new drugs, the median survival period of MM patients receiving systematic and standardized treatment can reach 7-10 years. However, this disease remains incurable, and relapse and refractory progression to a state of no available treatment is the outcome for most MM patients. With an increasing number of relapses or treatment lines, the patient's survival time becomes progressively shorter. Typically, the median progression-free survival (PFS) of MM patients who have experienced three relapses/progressions is only 3-6 months, with an overall survival (OS) of about one year. In recent years, there have been some new breakthroughs in drugs and treatment methods for multiple myeloma, among which BCMA CAR-T cell immunotherapy has advanced the most rapidly. At the 63rd American Society of Hematology (ASH) Annual Meeting in 2021, we reported clinical research data on Innovent Bio's Icaritin injection from 79 heavily pretreated MM patients across 14 clinical research centers: The overall response rate (ORR) was 94.9%, and the complete response/stringent complete response rate (CR/sCR) was 58.2%, demonstrating the excellent safety and efficacy of Icaritin injection. In patients with extramedullary multiple myeloma (EMM) and those previously treated with CAR-T therapy, Icaritin injection still showed favorable efficacy. These results suggest that Icaritin injection holds promise as a novel cancer immunotherapy for potentially curing multiple myeloma. We hope that Icaritin injection will be launched soon to bring long-term survival hope to patients."

Dr. Wang Wen, CEO and Chief Medical Officer of IASO Bio, stated:

"IASO Bio currently has more than ten competitive innovative pipeline products. The injection of Equecabtagene Autoleucel is the first CAR-T cell therapy product with independent intellectual property rights in China to file for NDA and be accepted, as well as the first CAR-T product targeting BCMA in China to file for NDA. This milestone achievement is a source of pride for all members of IASO Bio. The company’s nearly 10,000-square-meter commercial production base in Nanjing possesses complete capabilities for plasmid, lentiviral vector, and CAR-T cell production, along with quality control for corresponding products. It has obtained the Drug Manufacturing License for CAR-T cell therapy products, which will be used for the production of Equecabtagene Autoleucel after its market launch. In 2018, Professor Jianfeng Zhou from the Department of Hematology at Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, led a clinical team and a team of bioscientists to successfully apply the world's first fully human BCMA CAR-T (Equecabtagene Autoleucel) in clinical research on multiple myeloma. The first subject has maintained a stringent complete response (sCR) for up to 40 months. We express our gratitude to Professor Jianfeng Zhou for his relentless efforts in promoting the development of the immune cell drug industry and providing momentum for continuous innovation in CAR-T therapy. We look forward to the early market launch of this candidate product, bringing hope of a cure to more patients."

Dr. Yongjun Liu, President of Innovent Bio, stated:

"Ireluce Injection, a fully human BCMA CAR-T cell therapy product co-developed by Innovent Bio and IASO Bio, is also expected to become the first approved cell therapy product in China for the treatment of multiple myeloma. Ireluce Injection has demonstrated outstanding and durable efficacy as well as excellent safety in previous clinical studies. As an entirely new approach to cancer treatment, we look forward to this product being approved for marketing in China as soon as possible. At that time, we also plan to actively collaborate with governments at all levels, hospitals, commercial insurance companies, and charitable funds to explore innovative payment models, bringing breakthrough treatment options to patients with multiple myeloma in China."

The overseas development layout of Idecabtagene Vicleucel Injection is proceeding actively. In February 2022, Idecabtagene Vicleucel Injection was granted "Orphan Drug Designation (ODD)" by the Office of Orphan Products Development (OOPD) of the U.S. Food and Drug Administration (FDA) for the treatment of relapsed/refractory multiple myeloma. Additionally, in January 2022, IASO Bio and Innovent Bio granted Sana Biotechnology (NASDAQ: SANA) non-exclusive commercial rights to the BCMA CAR construct for use in Sana’s specific in vivo gene therapy and ex vivo hypoimmune cell therapy product development. Besides R/R MM, the Investigational New Drug (IND) application for the new extended indication—antibody-mediated Neuromyelitis Optica Spectrum Disorder (NMOSD)—has been officially accepted by the National Medical Products Administration (NMPA).

About Multiple Myeloma (MM）

Multiple Myeloma (MM) is one of the most common blood cancers and is a malignant disease characterized by the clonal proliferation of abnormal plasma cells. For newly diagnosed multiple myeloma patients, commonly used first-line treatments include proteasome inhibitors, immunomodulatory drugs, and alkylating agents. While the majority of patients achieve disease stability for 3-5 years with first-line treatment, a small proportion exhibit primary resistance at initial treatment, resulting in uncontrolled disease progression. Most patients who initially respond to treatment will inevitably enter a relapsed or refractory phase after a period of disease stability. Therefore, there remains an unmet need for patients with relapsed/refractory multiple myeloma. In the United States, MM accounts for nearly 2% of all cancer cases and over 2% of cancer-related deaths. According to a Frost & Sullivan report: The number of new MM cases in China increased from 18,900 in 2016 to 21,100 in 2020 and is expected to rise to 24,500 by 2025. The prevalence of MM in China grew from 69,800 in 2016 to 113,800 in 2020 and is projected to reach 182,200 by 2025. In the U.S., the number of new MM cases increased from 30,300 in 2016 to 32,300 in 2020 and is expected to rise to 37,800 by 2025. The number of prevalent MM cases in the U.S. rose from 132,200 in 2016 to 144,900 in 2020 and is projected to increase to 162,300 by 2025.