IASO Biotherapeutics’ Equecabtagene Autoleucel, the World’s First CAR-T for Treatment of NMOSD, Receives IND Application Acceptance by NMPA
IASO Biotherapeutics (IASO Bio), a clinical-stage biopharmaceutical company engaged in discovering, developing, and manufacturing innovative cell therapies and antibody products, announced today that China’s National Medical Products Administration (NMPA) has accepted its investigational new drug (IND) application for the new extended indication of Neuromyelitis Optica Spectrum Disorder (NMOSD) for a fully human BCMA chimeric antigen receptor autologous T (CAR-T) cell Injection (Equecabtagene Autoleucel，CT103A). This NMPA acceptance, the world’s first IND application for CAR-T in NMOSD.
IASO Bio’s IND application is based on the Equecabtagene Autoleucel Investigator Initiated Trial (IIT) enrolling patients with refractory NMOSD who were positive for Aquaporin 4 antibodies (AQP4). Subjects enrolled were patients with poor symptom control despite at least one year of treatment with at least one immunosuppressant. The primary endpoint of this study was to observe the safety and tolerability, as well as preliminary efficacy, pharmacokinetic (PK), and pharmacodynamic (PD) related indicators in subjects with relapsed/refractory NMOSD treated with Equecabtagene Autoleucel. As of March 20, 2022 cut-off date, 12 subjects received Equecabtagene Autoleucel cell reinfusion, including three in the 0.5×106 CAR-T cells/kg dose group, three in the 1.0×106 CAR-T cells/kg dose group in the dose escalation stage and six subjects in the expansion stage.
The preliminary data presented safety and efficacy of Equecabtagene Autoleucel in the 0.5×106 CAR-T cells/kg and 1.0×106 CAR-T cells/kg dose groups in treatment of NMOSD patients suffering from conditions that were poorly controlled by conventional therapies. All of them experienced 1~2 CRS and no ICANS events prescribed in the protocol were found in the dose exploration period of 12 subjects. Equecabtagene Autoleucel generally reduced the EDSS (Expanded Disability Status Scale) scores of NMOSD subjects, with improved visual acuity in 50% subjects, improved walking ability in 67% subjects, and improved bladder function in 75% subjects.
“The use of CAR-T cell products has been widely accepted in the treatment of hematologic malignancy but has been rarely reported in the treatment of autoimmune diseases. As one of the first company to use CAR-T cell to treat autoimmune diseases, IASO Bio submits the first IND application in China, marking the initiation of the expansion of their indication from oncology to autoimmune diseases, which is of great important for the cell therapy field. BCMA-directed CAR-T cell products targeting plasmocyte are available as a new option to treat the autoimmune diseases caused by plasmocyte-produced autoantibody. Neuromyelitis optica spectrum disorder (NMOSD) is characterized by high recurrence and disability rates, etc., and often cause blindness, paralysis, defecation and other serious physical disorders in a later stage” said Dr. Wen Wang, the Chief Executive Officer and Chief Medical Officer of IASO Bio. “In contrast, the new therapeutic strategy offered by IASO Bio with Equecabtagene Autoleucel can provide an opportunity to reverse and ameliorate organ damage and improve the quality of life in NMOSD patients. It may provide a new treatment option for patients with poorly controlled NMOSD by existing drugs. We expect that this product candidate will be available soon to NMOSD patients in China and around the world.”
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