▎WuXi AppTec Content Team Report

Myasthenia Gravis (MG) is an autoimmune-mediated neuromuscular junction transmission disorder, with the main clinical manifestations being a decrease in muscle strength in local or systemic muscles. It can affect important muscle groups such as the eye muscles, respiratory muscles, and limb muscles, significantly negatively impacting the patient's quality of life. Myasthenic crisis-induced swallowing or breathing difficulties can be life-threatening. The main pathogenic antibodies of MG includeAcetylcholine Receptor (AChR)、Muscle-Specific Receptor Tyrosine Kinase (MuSK) and Low-Density Lipoprotein Receptor-Related Protein 4 (LRP4)Antibody.

Previously, an investigator-initiated exploratory clinical studyEvaluation of IASO Bio's Cilta-cel Injection TreatmentRelapsed/Refractory Antibody-Mediated Idiopathic Inflammatory Neurological DiseasesSafety and EffectivenessIn this study, two subjects with refractory MG were enrolled. 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 antibody therapy. The other subject was a 60-year-old female who was positive for MuSK-IgG4, had a 20-year history of the disease, and had previously received various ineffective treatments including hormones, immunosuppressants, and targeted CD20 monoclonal antibodies. The two subjects were each administered 1.0×10⁶Single infusion treatment with IASO Bio's CAR-T/Kg Ixazomib Injection.

The research results were published in an international academic journal this February.EMBO Molecular MedicineOfficialPublish。Efficacy data showed that clinical symptoms of 2 subjects continued to improve for more than 18 months.。In terms of safety,In 2 subjects, only 1 subject experienced Grade 1 cytokine release syndrome (CRS), and no immune effector cell-associated neurotoxicity syndrome (ICANS) occurred.ICANS), ≥Grade 3 cytopenia all recovered within 4 weeks after infusion.Compared with the safety profile in the study of multiple myeloma indications, no new safety risks were identified, and the safety...Better.

The principal investigator of this clinical study is from Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology.Professor Wang WeiSaid: "The current treatment options for autoimmune diseases are limited, difficult to cure, and prone to relapse, imposing a significant physical and psychological burden on patients and their families. In collaboration with IASO Bio, we have pioneered the application of BCMA CAR-T, an advanced cell therapy, in various severe autoimmune diseases, including MG, achieving excellent clinical outcomes and publishing multiple research papers in authoritative international academic journals. The recent approval of the U.S. IND for the autoimmune indication of severe myasthenia gravis using Ixcellen 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."

[1] IASO Bio's Icarus Cell Injection for Myasthenia Gravis Autoimmune Indication Receives US IND Approval.Retrieved Apr 5 , 2024 ,from https://mp.weixin.qq.com/s/r44QTgnpj_YevvclG8z3QQ