April 5, 2024, Nanjing, Shanghai, China, and San Jose, California, USA — IASO Bio, a biopharmaceutical company dedicated to the research, development, manufacturing, and commercialization of innovative cell therapies, announcedIASO Bio's self-developed fully human BCMA-targeted chimeric antigen receptor autologous T-cell injection (Idecabtagene Vicleucel Injection, R&D code CT103A) has received the FDA's IND tacit approval for the treatment of refractory generalized myasthenia gravis (gMG)., the IND for this indication in China was approved by the NMPA in January this year.Based on an investigator-initiated, open-label exploratory clinical study evaluating the safety and efficacy of infusing Ixcellence (generic name: Idecabtagene Vicleucel) injection for the treatment of relapsed/refractory antibody-mediated idiopathic inflammatory neurological diseases (NCT04561557), this study enrolled two subjects with refractory Myasthenia Gravis (MG). One subject was a 33-year-old female who was positive for AChR-IgG and Titin-IgG. She underwent thymectomy 21 months prior to enrollment and had not achieved clinical remission despite treatments with cholinesterase inhibitors, glucocorticoids, immunosuppressants, and targeted CD20 monoclonal antibodies. The other subject was a 60-year-old female who was positive for MuSK-IgG4, with a 20-year history of the disease, and had previously received various ineffective treatments including steroids, immunosuppressants, and targeted CD20 monoclonal antibodies. The two subjects were infused with 1.0×106A single infusion treatment with IASO Bio's CAR-T/kg Olverembatinib injection. The study results were officially published in the international authoritative academic journal "EMBO Molecular Medicine" this February.Safety:In the 2 subjects, only 1 subject experienced Grade 1 Cytokine Release Syndrome (CRS), and no Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS) occurred. The ≥Grade 3 cytopenia recovered within 4 weeks after infusion. Compared with the safety profile in the multiple myeloma indication study, no new safety risks were identified, and the safety was better.Effectiveness:Clinical symptoms of 2 subjects continued to improve for over 18 months. Beginning 3 months after the infusion of Icarus Bio's Icibio Injection, patients showed significant improvement in limb strength and vital capacity. Scores on the Myasthenia Gravis-Activities of Daily Living (MG-ADL), Quantitative Myasthenia Gravis (QMG), Myasthenia Gravis-Quality of Life (MG-QOL), and modified Rankin Scale (mRS) also demonstrated sustained improvement.PK/PD：After infusion, Ixcellen injection showed good expansion in the subjects. The levels of anti-AChR and anti-Titin antibodies, as well as anti-MuSK antibodies, rapidly decreased and remained at very low levels over the long term in two subjects. B cells and plasma cells in the two subjects became undetectable within two months post-infusion and gradually recovered afterward. At 18 months post-infusion, B cell counts in both subjects had returned to normal levels, with approximately 80% being naive B cells, while plasma cells remained at low levels. These results suggest that the long-term efficacy of CAR-T cell therapy may be related to the reconstitution of B cells predominantly with a naive phenotype and the sustained depletion of plasma cells.Professor Wang Wei, the principal investigator of this clinical study from Tongji Hospital affiliated with Tongji Medical College of Huazhong University of Science and Technology, stated"Current treatment options for autoimmune diseases are limited, making them difficult to cure and prone to recurrent episodes, which imposes a significant physical and psychological burden on patients and their families. In collaboration with IASO Bio, we have pioneered the application of the advanced BCMA CAR-T cell therapy in various severe autoimmune diseases, including MG, achieving excellent clinical outcomes and publishing multiple research papers in internationally authoritative academic journals. The recent approval of the U.S. IND for the autoimmune indication of severe myasthenia gravis using Iberdomide Injection represents another endorsement by the FDA of the potential clinical value of our research direction. We will further explore the application of BCMA CAR-T in more refractory autoimmune diseases."Ms. Jin Hua Zhang, Founder and CEO of IASO Bio"We are very pleased that the clinical trial for the myasthenia gravis indication of Ixazomib Cilostazol Injection has been approved in the United States. This is the first CAR-T application in the autoimmune field that IASO Bio has obtained clinical approval for in the U.S. This not only represents international recognition of our R&D capabilities in cell therapy for autoimmune diseases and the potential value of our products, but also marks an important milestone for IASO Bio as we embark on expanding into the international autoimmune market. We would like to thank the clinical physician teams who have assisted us in conducting the clinical trials, as well as the patients and their families who have placed their trust in IASO Bio. Seeing patients with severe autoimmune diseases, who have been repeatedly afflicted by their conditions and unable to care for themselves, return to a normal life after treatment with Ixazomib Cilostazol is our greatest motivation for development. We will actively advance the clinical progress of Ixazomib Cilostazol in the United States, providing more effective and long-lasting treatment options for myasthenia gravis patients worldwide. At the same time, we will strive to expand groundbreaking cell therapies into a broader range of autoimmune disease areas, aiming to improve the treatment landscape for autoimmune diseases globally and help more patients with serious autoimmune conditions regain a healthy life."About Myasthenia Gravis (MG)Myasthenia Gravis (MG) is an autoimmune-mediated neuromuscular junction transmission disorder. The main clinical manifestations of MG include a decrease in muscle strength in local or systemic muscles, which can affect important muscle groups such as ocular, respiratory, and limb muscles, significantly negatively impacting the patient's quality of life. Moreover, myasthenic crisis-induced swallowing or breathing difficulties can be life-threatening. The primary pathogenic antibodies in MG include AChR, MuSK, and LRP4 antibodies, while a small number of patients have no detectable levels of the aforementioned antibodies in their serum.1MG can occur at any age, with a slightly higher prevalence in women than in men.2According to relevant studies, the incidence rate of MG in the United States is 3.1 per 100,000 people, and the prevalence rate is 37.0 per 100,000 people.3It is estimated that there will be approximately 10,000 new cases of MG in the United States in 2023, with a total of about 124,000 people affected.IASO Bio is a biopharmaceutical company dedicated to the research, production, and commercialization of innovative cell-based drugs. With a focus on developing cellular therapies and antibody drugs for hematological malignancies as its innovation cornerstone, the company has expanded into autoimmune diseases, possessing comprehensive capabilities across the entire process from early discovery, clinical development, regulatory submission to commercial-scale manufacturing.
The company currently has more than 10 innovative drug candidates at various stages of development. Among them, the injection of Ixazomib (a fully human BCMA CAR-T product) has been approved for marketing by the National Medical Products Administration (NMPA) in China and has also received FDA approval for registration clinical trials in the United States for the treatment of relapsed/refractory multiple myeloma.
IASO Bio, with its strong management team, innovative product pipeline, self-owned GMP manufacturing, and powerful clinical development capabilities, aims to provide transformative and curative innovative therapies, bringing hope of a cure to patients in China and around the world.
For more information, please visit the company's official website: www.iasobio.com or LinkedIn account: www.linkedin.com/company/iasobiotherapeutics.
References:
[1]Chinese Guidelines for the Diagnosis and Treatment of Myasthenia Gravis (2020 Edition)
[2] Incidence, mortality, and economic burden of myasthenia gravis in China: A nationwide population-based study - The Lancet Regional Health – Western Pacific.
[3]Rodrigues, E. et al. Incidence and prevalence of myasthenia gravis in the United States: A claims-based analysis. Muscle Nerve 69, 166–171 (2024).
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