On June 2, 2022 IASO Biotherapeutics ("IASO Bio"), a clinical-stage biopharmaceutical company engaged in discovering, developing, and manufacturing innovative cell therapies and antibody products, and Innovent Biologics, Inc. ("Innovent," HKEX: 01801), reported that the China National Medical Products Administration (NMPA) has formally accepted the New Drug Application (NDA) for Equecabtagene Autoleucel (IASO Bio R&D code: CT103A, Innovent R&D code: IBI326), a fully human anti-B cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T-cell therapy for the treatment of relapsed and/or refractory multiple myeloma (R/R MM) (Press release, IASO BioMed, JUN 2, 2022, View Source [SID1234615477]).
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Equecabtagene Autoleucel is the first CAR-T therapy in China that is self-developed with proprietary whole-process product development and the first BCMA-targeting CAR T-cell therapy in China with its NDA formally accepted by the NMPA. It is an innovative therapy co-developed by IASO Bio and Innovent. In February 2021, Equecabtagene Autoleucel was granted "Breakthrough Therapy Designation（BTD）" by the NMPA.
The acceptance of NDA is based on data from a single-arm, open-label, multi-center phase 1/2 study being conducted in China. Study results showed that Equecabtagene Autoleucel has excellent safety and efficacy profiles, low immunogenicity given a fully-human scFv, robust expansion and prolonged persistence in vivo. It will potentially offer a breakthrough treatment option for patients with R/R MM. The data from the phase 1/2 clinical study of Equecabtagene Autoleucel was presented in an oral presentation at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (Abstract #547) and the updated data was accepted as an oral presentation at the 27th Annual Congress of the European Hematology Association (EHA) (Free EHA Whitepaper)（EHA）Virtual Meeting (Abstract #S187), held on June 9-12, 2022.
"Multiple Myeloma (MM) is the second-most-common hematologic malignancy. Although the survival in MM patients has been dramatically extended to 7-10 years on average with recent drug development, the disease is still incurable and relapse or refractory after standard therapies is common for most MM patients. The later lines of treatment the patients are receiving, the shorter of survival time for those patients. Usually, the median progression-free survival of MM patients who had received at least third-line of prior therapy is only 3-6 months, and the overall survival time is less than 1 year. In recent years, there have been some encouraging breakthroughs in drugs and therapeutic interventions for the treatment of MM. The most exciting progress is BCMA CAR-T cell immunotherapy." said the two principal investigators at the primary study sites – Prof. Lugui Qiu, MD, from the Chinese Academy of Medical Science Hematology Hospital and Prof. Chunrui Li, MD, PhD, from Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology. "At the 63rd Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in 2021, we reported the results of the clinical study on Equecabtagene Autoleucel injection. The study was conducted in 14 clinical centers and enrolled 79 patients with MM who had received at least third-line of prior therapy. Of the 79 patients, the overall response rate(ORR) was 94.9% and the complete response/stringent complete response (CR/sCR) rate was 58.2%. These study results showed that Equecabtagene Autoleucel has excellent safety and efficacy profiles. In addition, Equecabtagene Autoleucel also demonstrated favorable efficacy in patients with extramedullary multiple myeloma and patients who had received prior CAR-T therapy. These results suggest that Equecabtagene Autoleucel is potentially a new and effective immunotherapy treatment option for patients with MM. We hope that Equecabtagene Autoleucel can be launched in China soon, bringing long-term benefits to patients."
"IASO Bio currently has more than 10 innovative pipeline products under development. Equecabtagene Autoleucel is China’s first domestically developed CAR T-cell therapy with global intellectual property rights and the first BCMA-targeting CAR T-cell therapy with its NDA formally accepted by the NMPA. This is a significant milestone for IASO Bio. IASO Bio’s over 100,000 ft² manufacturing facility in Nanjing, which has end-to-end manufacturing capability that covers the entire CAR-T production process, received its drug manufacturing license earlier this year, will be the future commercial production site for Equecabtagene Autoleucel." said Wen (Maxwell) Wang, M.D., Ph.D., Chief Executive Officer and Chief Medical Officer of IASO Bio.
"In 2018, Professor Jianfeng Zhou of Tongji Hospital, Tongji Medical College led a team of clinicians and biologists to initiate clinical study of the world’s first fully-human BCMA CAR T-Cell therapy (Equecabtagene Autoleucel) for the treatment of multiple myeloma. The first patient of the study has maintained strict complete remission (sCR) for over 40 months. "Maxwell added, "Many thanks to Professor Jianfeng Zhou for his unremitting efforts to promote the development of novel cell therapies and provide the impetus for the continuous innovation of CAR-T therapy. We look forward to the commercialization of Equecabtagene Autoleucel to bring hope to more multiple myeloma patients."
Dr. Yongjun Liu, President of Innovent, said, "We are glad about the NDA acceptance of Equecabtagene Autoleucel, a product candidate co-developed by Innovent and IASO Bio, and it will potentially to be the domestic first approved and launch-to-market BMCA CAR-T therapy for multiple myeloma. In the clinical studies, Equecabtagene Autoleucel demonstrated impressive efficacy and favorable safety profiles. We hope that this breakthrough therapy could be approved in the near future and we will actively coordinate with all parties including the government authorities, hospitals, commercial insurance and charity funds to bring benefit to more multiple myeloma patients.
IASO Bio and Innovent are actively advancing the clinical development of Equecabtagene Autoleucel. In February 2022, it was granted"Orphan Drug Designation (ODD)" by the Office of Orphan Products Development (OOPD) of the U.S. Food and Drug Administration (FDA). In January 2022, IASO Bio and Innovent have jointly granted non-exclusive commercial rights of the fully-human BCMA CAR construct used in Equecabtagene Autoleucel to Sana Biotechnology (NASDAQ: SANA), a company focused on creating and delivering engineered cells as medicines, for use in its in vivo gene therapy and ex vivo hypoimmune cell therapy applications. Sana’s clinical and commercial development could further enhance the potential value of CT103A, benefitting a broader patient population. In addition to multiple myeloma, the NMPA has accepted IND application of Equecabtagene Autoleucel for the new expanded indication of Neuromyelitis Optica Spectrum Disorder (NMOSD).
About Multiple Myeloma (MM)
In the United States, MM accounts for nearly 2% of new cancer cases, and more than 2% of all cancer-related deaths. According to Frost & Sullivan, the number of new MM cases in the United States rose from 30,300 in 2016 to 32,300 in 2020 and is expected to increase to 37,800 by 2025. Additionally, the total number of patients diagnosed with MM increased from 132,200 in 2016 to 144,900 in 2020 and is expected to rise to 162,300 by 2025. In China, the number of new MM cases rose from 18,900 in 2016 to 21,100 in 2020 and is expected to increase to 24,500 by 2025. The total number of patients diagnosed with MM in China increased from 69,800 in 2016 to 113,800 in 2020 and is expected to rise to 182,200 by 2025.