Cancer Treatment New Drug Developer



On September 13, 2024, IASO Bio announced that it will present a poster on September 15 (Orlando, USA time) during the 2024 American Neurological Association (ANA) Annual Meeting, showcasing the research results of Equecabtagene Autoleucel Injection for the treatment of three neurological autoimmune diseases.
The contents of the three posters are all based on the results of an investigator-initiated exploratory clinical study (NCT04561557) evaluating the safety and efficacy of a single infusion of Icarus bio's Iciclenin injection in treating recurrent/refractory antibody-mediated neuroinflammatory diseases. The study is being conducted at Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, by a research team led by Professor Wei Wang from the Department of Neurology at Tongji Hospital.
Abstract Number: LB-S1.005
Title: Single-cell analysis of BCMA CAR-T cell therapy in patients with central nervous system autoimmune diseases
The study enrolled 12 subjects with aquaporin-4 (AQP4) antibody-positive relapsing/refractory neuromyelitis optica spectrum disorder (NMOSD). The enrolled subjects had been treated with at least one immunosuppressant for more than a year but had poor symptom control. The study results showed,A single infusion of IASO Bio's BCMA CAR-T cell therapy can durably eliminate NMOSD-related pathogenic antibodies and demonstrates significant clinical efficacy, with good tolerability and safety. BCMA CAR-T cells can cross the blood-brain barrier into the central nervous system, directly targeting and killing abnormal plasma cells., reduce the secretion of intrathecal autoantibodies and the abnormal activation of immune cells, thereby correcting the immunological disorder status in the central nervous system of NMOSD patients, which is beneficial for the central immune reconstruction of patients.
Abstract Number: LB-S1.006
Abstract Title: BCMA CAR-T Therapy for the Treatment of Refractory Severe Myasthenia Gravis
The study enrolled 2 subjects with refractory severe myasthenia gravis (MG), of which only 1 experienced grade 1 cytokine release syndrome (CRS), and no immune effector cell-associated neurotoxicity syndrome (ICANS) occurred. Three months after a single infusion of Icaritamab, the subject showed significant improvement in limb strength and vital capacity, with continuous improvement in Myasthenia Gravis-Activities of Daily Living score (MG-ADL), Quantitative Myasthenia Gravis score (QMG), Myasthenia Gravis-Quality of Life score (MG-QOL), and modified Rankin Scale score (mRS).Pathogenic antibodies related to 2 subjects both rapidly turned negative after CAR-T cell reinfusion and remained negative, while clinical symptoms continuously improved for over 2 years.During the follow-up period, no other immunomodulatory treatments were combined except for low-dose bromopyridostigmine (with a tapering dose, discontinued two years after reinfusion).
Abstract Number: LB-S1.007
Abstract Title: BCMA CAR-T Therapy for the Treatment of Refractory SRP-Positive Necrotizing Myopathy
The study enrolled a 25-year-old male subject with refractory immune-mediated necrotizing myopathy (IMNM) who was positive for anti-SRP antibodies. The subject had a medical history of 7 years and had previously received various treatments but still experienced multiple relapses and ongoing damage. Prior to enrollment, despite receiving regular treatment, the subject remained bedridden due to paralysis, unable to raise his arms above his head, with a serum creatine kinase level as high as 4806 IU/L (reference value ≤190 U/L).Nine months after a single infusion of IASO Bio's equecabtagene autoleucel, the subject's neurological examination results were nearly normal, with only mild weakness in the proximal muscles of the lower limbs. Muscle strength in the neck, distal lower limbs, and all upper limb muscles had returned to normal.Clinical and imaging improvements persisted until 2 years post-infusion, during which no immunosuppressants or hormone treatments were combined. The serum level of anti-SRP autoantibodies rapidly decreased after cell infusion and remained negative for 2 years post-infusion. Single-cell sequencing analysis revealed the normalization of the immune microenvironment after CAR-T cell infusion, including B-cell reconstitution, T-cell subset reconstruction, and suppression of overactivated immune cells.

