IASO Biotechnology, a prominent biopharmaceutical company specializing in innovative cell therapy and antibody products, is excited to announce the approval of the Investigational New Drug application by the U.S. Food and Drug Administration (FDA) for its BCMA CAR-T therapy, Equecabtagene Autoleucel (Eque-cel), for use in U.S. clinical trials targeting generalized myasthenia gravis (gMG). The Chinese IND for Eque-cel for this indication was also granted approval by the National Medical Products Administration (NMPA) earlier this year.

An investigator-initiated open-label study enrolled two subjects with refractory MG to evaluate the safety and efficacy of Eque-cel in treating relapsed/refractory antibody-mediated idiopathic inflammatory disorders of the nervous system. One subject, a 33-year-old female, tested positive for AChR-IgG and Titin-IgG, while the other subject, a 60-year-old female, tested positive for MuSK-IgG4. Both had extensive treatment histories, including thymectomy, but had not achieved clinical remission. The subjects received a single infusion of Eque-cel at a dose of 1.0×106 CAR-T/Kg. The results of this study were published in EMBO Molecular Medicine in February of this year.

Safety assessments revealed that only one subject experienced grade 1 Cytokine Release Syndrome (CRS), with no occurrences of Immune effector Cell-Associated Neurotoxicity Syndrome (ICANS). Hemocytopenia of grade ≥3 recovered within four weeks post-infusion. Notably, the safety profile demonstrated superiority compared to previous indications, such as multiple myeloma.

Regarding effectiveness, both subjects showed continued improvement in clinical symptoms over 18 months. Significant enhancements in limb strength, vital capacity, and sustained improvements in various MG-related scores were observed starting from three months post-infusion.

Pharmacokinetic/pharmacodynamic (PK/PD) analyses indicated robust expansion of Eque-cel post-infusion. There was a rapid decrease and sustained low levels of anti-AChR antibodies, anti-Titin antibodies, and anti-MuSK antibodies in both subjects. B cells and plasma cells decreased to undetectable levels within two months post-infusion, gradually recovering thereafter. At 18 months post-infusion, B cells returned to normal levels, predominantly comprising naïve B cells, while plasma cells remained at low levels, suggesting the potential long-term efficacy of CAR-T cell therapy.

Professor Wei Wang of Tongji Hospital, Tongji Medical School of Huazhong University of Science and Technology, the principal investigator of the study, expressed, "Autoimmune diseases pose significant challenges with limited treatment options. Through collaboration with IASO Bio, we have made significant strides in applying BCMA CAR-T therapy to severe autoimmune diseases like MG, demonstrating promising clinical efficacy."

Ms. Jinhua Zhang, Founder and Chairman of IASO Bio, expressed gratitude towards the clinicians, patients, and families involved in the clinical trial. She emphasized the company's commitment to advancing Eque-cel's clinical development in the U.S., aiming to provide more effective and durable treatment options for MG patients globally. Additionally, she highlighted the company's dedication to expanding breakthrough cell therapies to address a broader spectrum of autoimmune diseases, with the ultimate goal of improving treatment outcomes for patients worldwide.

MG is an autoantibody-mediated neuromuscular junction disorder characterized by muscle weakness, which can significantly impact quality of life and, in severe cases, lead to life-threatening complications like dysphagia or respiratory distress. The main pathogenic antibodies in MG include AChR, MuSK, and LRP4 antibodies. MG can affect individuals of any age, with a slightly higher prevalence in women. In the United States, the incidence rate of MG is estimated at 3.1/100,000 people, with a prevalence rate of 37.0/100,000 people.

 

Source: prnewswire.com

 

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