SHANGHAI, NANJING, China and SAN JOSE, Calif., Jan. 31, 2024 /PRNewswire/ -- On Jan. 30, 2024,
PNAS published the research paper titled "Single-cell analysis of refractory anti-SRP necrotizing myopathy treated with anti-BCMA CAR T-cell therapy." The paper analyzed results using the fully human B cell maturation antigen (BCMA)-targeting chimeric antigen receptor (CAR)-antologous T cell (BCMA CAR-T) injection (Equecabtagene Autoleucel injection, R&D code: CT103A) for the treatment of IMNM, which showed durable pathogenic antibody clearance, and potentially persistent clinical efficacy with good tolerability and safety. This study provides a potentially new therapeutic option for antibody-mediated autoimmune disorders. IMNM is an autoimmune-mediated skeletal muscle disorder belonging to an idiopathic inflammatory disease. IMNM mediated by human anti-signal recognition particle (SRP) antibody, an IMNM-specific autoantibody, is mainly characterized by symmetrical weakness of limb proximal muscles, prominent dysphagia, and significantly elevated serum creatine kinase, with features of acute onset, severe conditions, and rapid progression. Traditional pharmacological approaches have varying and often limited effects. A 25-year-old male patient with a 7-year history of SRP antibody-positive refractory IMNM was enrolled in this study. The patient had been subject to repeated relapses and persistent injuries even after receiving multiple prior therapies including steroids, calcineurin inhibitors, folic acid antagonists, CD20 monoclonal antibodies, interleukin-6 (IL-6) receptor antagonistsinterleukin-6 (IL-6) receptor antagonists, inosine monophosphate dehydrogenase (IMPDH) inhibitors, alkylating agents, plasmapheresis, intravenous immunoglobulin injection, and mesenchymal stem cell infusion. Before enrollment, the patient was still paralyzed and bedridden. He was not able to lift his arms overhead and his serum creatine kinase was up to 4806 IU/L (Normal range ≤ 190 U/L) even under regular combination therapies with steroids, IL-6 receptor antagonists, folic acid antagonists, and intravenous immunoglobulin injection. Efficacy: During the 18-month follow-up after infusion of Equecabtagene Autoleucel, the patient's clinical symptoms and imaging characteristics continuously improved. Three months after infusion of Equecabtagene Autoleucel, the patient's strength of extremities improved significantly. He could lift his arms without much effort, and regained his ability to walk. Manual Muscle Testing-8 (MMT-8) score improved from 96 points at baseline to 137 points at the last visit (18 months after infusion). Serum creatine kinase level decreased from 4778 IU/L before infusion to 260 IU/L at the last visit, and myoglobin level decreased from 837 ng/mL before infusion to 66.2 ng/mL at the last visit. Significant improvements were also observed in other quality of life scales. There was no concomitant use of other immunomodulatory therapies during the follow-up. The principal investigator of this study,
IASO Bio is a biopharmaceutical company engaged in the discovery and development of novel cell therapies and biologics for oncology and autoimmune diseases. IASO Bio possesses comprehensive capabilities spanning the entire drug development process, from early discovery to clinical development, regulatory approval, and commercial production. The pipeline in the company includes a diversified portfolio of over 10 novel products, including Equecabtagene Autoleucel (a fully human BCMA CAR-T injection). Equecabtagene Autoleucel received New Drug Application (NDA) approval from China's National Medical Products Administration (NMPA) and U.S. FDA IND approval for the treatment of RR MM. Leveraging its strong management team, innovative product pipeline, GMP production, as well as integrated manufactural and clinical capabilities, IASO aims to deliver transformative, curable, and affordable therapies that fulfil unmet medical needs to patients in China as well as around the world. For more information, please visit http://www.iasobio.com or www.linkedin.com/company/iasobiotherapeutics.